Strategic Wins
Collaboration will focus on co-development of unique datasets to understand health in everyday life
January 10, 2022 06:00 AM Eastern Standard Time
SAN MATEO, Calif.--(BUSINESS WIRE)--Evidation, the company creating new ways to measure and improve health in everyday life, is expanding its decade-long collaboration with Sanofi to build upon their joint real-world data initiatives. This new phase will focus on the co-development of unique datasets to develop and validate new measures of health and wellness.
Evidation’s collaboration with Sanofi has delivered groundbreaking results to date, with over 20 studies conducted across 10 therapeutic areas, including diabetes and Type 2 Inflammation, more than 500,000 patients reached, and four studies published. This continued collaboration will further the work Evidation and Sanofi have pioneered to translate person-generated health data into quantified clinical and economic outcomes, a key priority for both companies.
“After nearly a decade of working with Sanofi, we are proud to expand this collaboration agreement to advance the role that real-world data and analysis can play in better understanding health and disease,” said Christine Lemke, Evidation co-founder and co-CEO. “Sanofi has led the way in garnering insights from real-world data in R&D and we’re excited to advance our work together into its next decade."
Sanofi and Evidation announced a prior expansion of their work together in 2017, in addition to Sanofi’s investment in Evidation in the same year.
“Real-world evidence is critical to help us better understand the patient’s health and wellness journey outside of traditional healthcare visits,” said Arnaud Robert, Executive Vice President, Chief Digital Officer, Sanofi. “Through our expanded collaboration with Evidation, we can further our ambition to transform the practice of medicine by connecting more closely with patients and citizens, expanding our geographic capabilities, and increasing diversity to better represent the global population.”
This announcement comes as biopharmaceutical companies, regulators, and payers are working to develop new guidelines on how real-world data should be incorporated into the development and approval of therapeutics. Evidation and Sanofi will continue to contribute to this conversation through similar industry-leading research.
The Evidation network is made up of more than 4.4 million individuals across all 50 states, representing 9 out of every 10 U.S. ZIP codes, allowing organizations like Sanofi access to a highly engaged, diverse population and privacy-conscious research platform.
ABOUT EVIDATION
Evidation measures health in everyday life and enables anyone to participate in ground-breaking research and health programs. Built upon a foundation of user privacy and control over permissioned health data, Evidation’s platform is trusted by millions of individuals—generating data with unprecedented speed, scale, and rigor. We partner with leading healthcare companies to understand health and disease outside the clinic walls. Guided by our mission to enable and empower everyone to participate in better health outcomes, Evidation is working to bring people individualized, proactive, and accessible healthcare—faster. Founded in 2012, Evidation is headquartered in California with additional offices around the globe. To learn more, visit evidation.com, or follow us on Twitter @evidation.
Contacts
MEDIA CONTACT
Matt Miller
press@evidation.com
Partnership to create new treatment delivery options for people facing serious diseases
BOSTON, MA—February 10, 2021—i2O Therapeutics, developers of a platform for oral delivery of traditionally injectable biological drugs, announced today a research collaboration with Sanofi to investigate the oral delivery of Sanofi's Nanobody®-based medicines, which are currently administered through intravenous or subcutaneous injections.
Nanobodies - proprietary therapeutic proteins based on camelid derived immunoglobulin single variable domains - have potential uses in the treatment of a range of serious and life-threatening diseases and are being developed in many therapeutic areas including inflammation, hematology, immuno-oncology, oncology and rare diseases. The research collaboration between i2O Therapeutics and Sanofi will explore a new oral route of administering nanobodies.
“Our mission at i2O Therapeutics is to develop safe and effective oral formulations of therapies traditionally limited to injections and we are excited to partner with Sanofi to advance this mission,” said Ravi Srinivasan, co-founder and director of i2O Therapeutics.
“i2O’s ionic liquid platform opens new opportunities to orally deliver biologics, and nanobodies represent an exciting application of this platform,” said Samir Mitragotri, co-founder of i2O Therapeutics.
i2O Therapeutics announced seed funding in April 2020, which was led by Sanofi Ventures, the corporate venture capital arm of Sanofi, and JDRF T1D Fund. The company also announced a strategic investment from Colorcon Ventures, the corporate venture capital fund of Colorcon, Inc. in December 2020.
About i2O Therapeutics
i2O Therapeutics is a biotechnology company developing safe and effective oral formulations of therapies traditionally limited to injections. Using an innovative ionic liquid technology, this platform leverages the benefits of protecting the drug cargo while also transiently enhancing permeation across the epithelial lining when administered orally. i2O is focused on creating the next generation of oral peptide and protein-based therapies. Visit us at www.i2OBio.com.
About Sanofi
Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions. With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Contact
Lauren Arnold
MacDougall
larnold@macbiocom.com
781-235-3060
- Kymera to receive $150 million upfront with more than $2 billion in potential milestones plus royalty payments
- Kymera to retain option during clinical development to participate equally in US cost and profit sharing
CAMBRIDGE, Mass. (July 9, 2020) – Kymera Therapeutics Inc. today announced the company has entered into a multi-program strategic collaboration with Sanofi (NASDQ: SNY) to develop and commercialize first-in-class protein degrader therapies targeting IRAK4 in patients with immune-inflammatory diseases. The companies will also partner on a second earlier stage program. Kymera will receive $150 million in cash upfront and may receive more than $2 billion in potential development, regulatory and sales milestones, as well as significant royalty payments. Kymera retains the option to participate in US development and commercialization for both programs. This includes the ability to participate equally in the costs, profits and losses after opt-in, and to co-promote partnered products in the US.
“This is an important collaboration for both companies and for the field of targeted protein degradation,” said Nello Mainolfi, Ph.D., co-founder, President and CEO of Kymera Therapeutics. “Kymera is becoming a fully integrated biotechnology company advancing a pipeline of novel therapies with the potential to transform treatment paradigms. We are excited to partner with Sanofi, an organization with world-class drug development and commercialization capabilities, to ensure maximal patient impact from two of our programs across multiple disease indications, while enabling Kymera to invest in key strategic areas to realize the broad potential of protein degrader therapies.”
Under terms of the collaboration, Sanofi will make an upfront payment of $150 million in cash to Kymera for global rights to develop its small molecule IRAK4 protein degraders in inflammation and immunology indications, and a second earlier stage undisclosed program. IRAK4 is believed to play a key role in multiple immune-inflammatory diseases, including hidradenitis suppurativa, atopic dermatitis and rheumatoid arthritis. Kymera will advance the IRAK4 program through Phase 1 clinical trials; Sanofi will assume clinical development and commercialization responsibilities thereafter. Sanofi will lead all clinical development activities for the second program. Kymera will have the option to participate in the development of both programs in the US during clinical development. Kymera will retain global rights to its IRAK4 program in oncology indications.
IRAK4 is a key protein involved in inflammation mediated by the activation of toll-like receptors (TLRs) and IL-1 receptors (IL-1Rs). While TLR and IL-1R signaling via IRAK4 is involved in the normal immune response, aberrant activation of these pathways is the underlying cause of multiple immune-inflammatory conditions. In pre-clinical studies, Kymera has shown oral daily administration of an IRAK4 degrader can lead to complete knockdown of IRAK4 in skin and immune cells in higher species and is well tolerated. Data presented at the most recent annual meetings of the American College of Rheumatology and the European Hidradenitis Suppurativa Foundation showed potent anti- inflammatory activity in both in vitro and in vivo preclinical models."
“Targeted protein degrada on is an exci ng modality. Kymera has developed an incredible drug discovery engine producing protein degraders with compelling and dieren ated pharmacology against targets that, to date, have not been op mally addressed with other therapeu c modali es,” said John Reed, Global Head of Research & Development at Sano. “We are excited to partner with the Kymera team to advance a new genera on of rst-in-class therapies with the poten al to eliminate underlying drivers of disease.”
Aquilo Partners, L.P. acted as Financial advisor to Kymera on this transac on.
# # #
About Kymera Therapeutics
Kymera Therapeutics is a biotechnology company pioneering a transformative new approach to treating previously untreatable diseases. The company is advancing the field of targeted protein degradation, accessing the body’s innate protein recycling machinery to degrade dysregulated, disease-causing proteins. Kymera’s Pegasus targeted protein degradation platform harnesses the body’s natural protein recycling machinery to degrade disease-causing proteins, with a focus on un-drugged nodes in validated pathways currently inaccessible with conventional therapeutics.
Kymera is accelerating drug discovery with an unmatched ability to target and degrade the most intractable of proteins, and advance new treatment options for patients. For more information, visit www.kymeratx.com.
About Sanofi
Sanofi is dedicated to supporting people through their health challenges. We are a global pharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions. With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe. Sanofi, empowering life. For more information, visit www.sanofi.com.
PARIS and NEW YORK – November 20, 2019 - Sanofi announced today an enterprise- wide collaboration with health care technology company Aetion that will integrate Sanofi’s real-world data platform, DARWIN, with the Aetion Evidence Platform® with the objective of advancing more efficient use of real-world evidence (RWE), facilitating regulatory-grade studies with deep transparency, and unlocking access to new real-world data.
Both companies have invested in RWE platforms, recognizing the pressing need for accurate, fast, and cost-effective research and the important role RWE could play in meeting this need. Sanofi’s DARWIN compiles and analyzes de-identified data from hundreds of millions of patients across disease states, while Aetion’s platform analyzes real-world data to produce transparent, rapid, and scientifically validated answers about the effectiveness, safety, and value of drugs. By combining these platforms, Sanofi is seeking to elevate its capabilities in conducting regulatory-grade analytics, opening new doors for the development and application of medical treatments.
“Today marks another important step in Sanofi’s digital transformation,” said Bernard Hamelin, MD, MSc, MBA, Global Head of Medical Evidence Generation, Sanofi. “By integrating these platforms, we strive to make faster, more informed decisions with the potential to lead to first-in-class and best-in-class treatments that could change the practice of medicine.”
Real-world evidence offers a view of clinical practice outside of the experimental setting, providing an opportunity to inform clinical trial development and supplement trial data with evidence of actual product use in the health care system.
“Our work with Sanofi further validates the value and potential for real-world evidence in drug development,” said Carolyn Magill, Chief Executive Officer of Aetion. “Our companies share a common goal of using the best available data to get the right treatment to the right patient as quickly and efficiently as possible.”
This collaboration between Sanofi and Aetion demonstrates leadership during a critical time. Real-world evidence is expected to play a key role in transforming the health care ecosystem, with the U.S. Food and Drug Administration (FDA) recently prioritizing efforts to incorporate RWE as a companion to clinical trial data to aid in regulatory decision making. The FDA will release its draft RWE guidance before the end of 2020.
About Aetion
Aetion is a health care technology company that delivers real-world evidence for life sciences companies, payers, at-risk providers, and regulatory agencies. The Aetion Evidence Platform analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on treatments, costs, and outcomes. Founded by Harvard Medical School faculty with decades of experience in epidemiology and health outcomes research, Aetion informs health care's most critical decisions — what works best, for whom, and when — to guide treatment development, commercialization, and payment innovation into health care's modern era. Aetion is based in New York City, and backed by investors including New Enterprise Associates (NEA), Flare Capital Partners, Lakestar, Town Hall Ventures, McKesson Ventures, Sanofi Ventures, Amgen Ventures, UCB, and Horizon Health Services, Inc. Learn more at aetion.com, and follow us at @aetioninc.
About Sanofi
Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Sanofi, Empowering Life
Sanofi Media Relations Contact
Anna Robinson
Tel.: +33 (0)1 53 77 46 46
mr@sanofi.com
Aetion Media Relations Contact
202.792.7200
press@aetion.com
Sanofi Investor Relations Contact
George Grofik
Tel.: +33 (0)1 53 77 45 45
ir@sanofi.com
Sanofi Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward- looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic conditions, the impact of cost containment initiatives and subsequent changes thereto, the average number of shares outstanding as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2018. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any
forward-looking information or statements.
Without clinical trials, new medicine may never make it from the research lab to patients in need. These carefully designed studies can provide important data that include proper dosage, benefit to patients, and potential side effects.
There is a growing challenge, however, in finding appropriate participants, especially for treatments that target highly specific conditions affecting narrower patient populations. Right now, there are more than 40,000 clinical studies recruiting patients in the U.S. alone, with some requiring thousands of participants, each of whom must meet precise criteria to join. So it’s not surprising that 80% of these important studies are delayed due to recruitment problems, according to a study by the Center for Information and Study on Clinical Research Participation (CISCRP).
Unfortunately, those delays mean it can take longer for innovative new medicines to be studied and approved, leaving patients to wait years for new treatment options. To tackle this growing problem, Sanofi is taking a digital approach to clinical trials, partnering with Science 37, a clinical research services and technology company based in California.
Leveraging mobile technology and telemedicine capabilities, this new approach will allow Sanofi to develop “site-less” or decentralized clinical trials that are more patient friendly: easier for them to access, and eliminating many of the common impediments to participation. Using digital technologies to streamline finding and retaining participants for the entire length of a study has the potential to reduce the time required for a typical trial by at least 30%, according to Science 37.
“After years invested in the lab on an innovative treatment, the clinical trials are where we finally obtain and analyze the relevant data that will let us understand how well a new treatment will benefit patients,” said Lionel Bascles, Global Head of Clinical Sciences and Operations of Sanofi. “With digital clinical trials we can get and analyze the data on how a new medicine works in the real world a lot sooner, which means patients get the medicines they need sooner.”
Going digital also eliminates a number of other hurdles to patient participation, including the most significant: geography.
Most people are eager to participate in relevant trials – 87% of patients want to do so, the CISCRP study found. Yet, 70% of potential participants live more than two hours away from the nearest study center. Because most clinical trials require patients to travel to those centers for regular tests and observations, sometimes several times each week for the duration of the trial, this distance is another challenge to patient access.
Science 37’s approach allows patients to be monitored and report to researchers via an Apple iPhone equipped with the company’s NORA® technology. Qualified study participants are provided with the phone, a data plan and any other sensors or connected devices needed for the trial, along with the medicines being researched. Participants can reach study staff at any time via the mobile device, while also remaining under the care of their local health care professionals. Mobile nurses are also sent to the participant’s home to provide services like blood draws when needed, and nearby hospitals or clinics are engaged for scans or other tests that require specialized equipment.
The patient’s data are sent securely to researchers who can immediately access information that would otherwise have to be collected by medical personnel through face-to-face interactions at study centers. This platform can also remind patients to take their study medications at the proper time, and let researchers know if participants are adhering to the study requirements.
“Our decentralized clinical trial model addresses critical shortcomings of traditional clinical trials, such as enrolling and retaining appropriate patients. Whether you live near a major research institution, or in a remote area, we make participation possible,” said Noah Craft, CEO of Science 37. “By utilizing a patient’s home in lieu of a physical trial site, we remove the burden of travel for those too sick or remote and provide access to qualified individuals who want to volunteer for a study but cannot because of geographic limitations.”
The Science 37 platform will also help engage patients who would normally not participate in clinical trials, “so our data will much more closely track the diversity of the population,” Bascles said. “In addition to reducing the burden for patients, decentralized clinical trials are far more likely to keep patients engaged for the full length of the trial, increasing the relevance and the acceptability of the data by regulators.”
Sanofi’s agreement with Science 37 covers use of its Metasite™ model and NORA technology across the U.S. with plans to expand internationally in the future. By eliminating months of searching for patients and long travel time to study sites, virtual clinical trials could reduce total trial time by as much as two years.
Partnering with Science 37 is the most recent strengthening of the relationship with Sanofi, which began last October when Sanofi’s venture capital fund, Sanofi-Genzyme BioVentures, made a minority investment in Science 37.
“Science 37 has a great track record, and they are smart and forward-thinking about developing the science around clinical trials that leverage digital technologies,” said Heather Bell, Global Head of Digital and Analytics for Sanofi. “As part of the scope of our digital strategy, we have expanded the scope of the venture fund to include digital investments, and Science 37 was our first investment since that change and we’re very excited about it.”
SEATTLE and SOUTH SAN FRANCISCO – October 16, 2014 Immune Design Corp. (NASDAQ: IMDZ), a clinical-stage immunotherapy company, today announced that it has entered into a broad collaboration for the development of a herpes simplex virus (HSV) immune therapy with Sanofi Pasteur, the vaccines division of Sanofi (EURONEXT: SAN and NYSE: SNY).
Sanofi Pasteur and Immune Design will each contribute product candidates to the collaboration: Sanofi Pasteur will contribute HSV-529, a clinical-stage replication-defective HSV vaccine product candidate, and Immune Design will contribute G103, its preclinical trivalent vaccine product candidate. The collaboration will explore the potential of various combinations of agents, including leveraging Immune Design’s GLAASTM platform, with the goal to select the best potential immune therapy for patients.
The two companies will develop the products jointly through Phase 2 clinical trials, at which point Sanofi Pasteur intends to continue development of the most promising candidate and be responsible for commercialization. Sanofi Pasteur will bear the costs of all preclinical and clinical development, with Immune Design providing a specific formulation of GLA from the GLAAS platform at its cost through Phase 2 studies. Immune Design will be eligible to receive future milestone and royalty payments on any product developed from the collaboration; other financial terms of the agreement have not been disclosed.
“Instead of being limited to a single approach, the companies are joining forces and combining multiple cutting-edge technologies with the goal to develop the most effective and safe immunotherapy to address HSV infection, a significant unmet medical need,” said Carlos Paya, M.D., Ph.D., President and Chief Executive Officer of Immune Design. “With other clinical and preclinical GLAAS-based product candidates in development, both with partners and internally at Immune Design, I believe this new collaboration continues to demonstrate the productivity and broad applicability of this platform.”
About G103 and GLAAS
G103 is a trivalent vaccine candidate consisting of recombinantly-expressed viral proteins adjuvanted with a specific formulation from Immune Design’s GLAAS platform. The combination of a novel molecular toll-like receptor 4 (TLR4) agonist with rationally selected antigens is designed to boost pre-existing T cells and trigger a broad antibody response, allowing for prophylactic and therapeutic immunization.
The GLAAS platform works in vivo and is based on a small synthetic molecule called GLA, which stands for glucopyranosyl lipid adjuvant. GLA selectively binds to the TLR4 receptor and causes potent activation of dendritic cells (DCs) leading to the production of cytokines and chemokines that drive a Th1-type immune response. When GLA is accompanied by an antigen and injected into a patient, the combination is taken up by DCs and leads to the production and expansion of immune cells called CD4 T helper lymphocytes with a Th1 phenotype. These CD4 T cells play a key role in boosting pre-existing cytotoxic T cells that are specific to the same antigen; and providing help to other immune cells, including B lymphocytes that are the precursor to antibodies, and natural killer cells that are also important in the overall immune response. Immune Design believes that GLAAS- based product candidates have the potential to target multiple types of cancer, as well as infectious, allergic and autoimmune diseases. Product candidates leveraging GLAAS’ core technology have now
been evaluated in over 1000 subjects in Phase 1 and Phase 2 trials.
About Immune Design
Immune Design (NASDAQ: IMDZ) is a clinical-stage immunotherapy company employing next- generation in vivo approaches to enable the body’s immune system to fight disease. The company’s technologies are engineered to activate the immune system’s natural ability to create and/or expand antigen-specific cytotoxic T cells, while enhancing other immune effectors, to fight cancer and other chronic diseases. Immune Design’s three on-going immuno-oncology clinical programs are the product of its two synergistic discovery platforms: ZVexTMand GLAASTM, the fundamental technologies of which were licensed from the California Institute of Technology and the Infectious Disease Research Institute (IDRI), respectively. Immune Design has offices in Seattle and South San Francisco. For more information, visit www.immunedesign.com.
Immune Design Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend", “believe” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Immune Design’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include statements regarding efforts to develop products under the collaboration, the potential receipt of milestone and royalty payments and the potential to develop new therapeutics. Factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Immune Design’s filings with the U.S. Securities and Exchange Commission (the “SEC”), including the "Risk Factors" section of Immune Design’s Quarterly Report on Form 10-Q filed with the SEC on September 8, 2014 and in any subsequent filings with the SEC. Except as required by law, Immune Design assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
# # #
For Immune Design:
Media Contact Julie Rathbun
Rathbun Communications
julie@rathbuncomm.com
206-769-9219
Investor Contact Robert H. Uhl
Westwicke Partners
robert.uhl@westwicke.com
858-356-5932
CAMBRIDGE, Mass. and SEATTLE and SOUTH SAN FRANCISCO, Calif., Aug. 7, 2014 (GLOBE NEWSWIRE) -- Sanofi
(EURONEXT:SAN) (NYSE:SNY) and Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company, today announced that they have entered into a licensing agreement for use of Immune Design's GLAASTM discovery platform to develop therapeutic agents to treat a selected food allergy.
The incidence of food allergies is increasing worldwide in both developed and undeveloped countries, and especially in children.1 Globally, experts believe 220-250 million people may suffer from food allergies.2,3 In the United States alone, as many as 15 million people have food allergies,4 with allergic reactions resulting in an emergency room visit every three minutes and averaging more than 200,000 emergency room visits per year.5
"This is an exciting time in the area of immunology research, and our relationship with Immune Design is a great example of how Sanofi has changed our approach to R&D," said Kurt Stoeckli, vice president and head of Global Bio Therapeutics Organization, Sanofi. "With this partnership, we are able to tap into breakthrough science that holds great potential to transform how food allergies are treated, and the lives of those people affected. This kind of innovation is central to our new approach."
Under terms of the agreement, Immune Design has granted Sanofi an exclusive license to discover, develop and commercialize products to treat a selected food allergy. The company has received an undisclosed upfront payment and will be eligible to receive development and commercialization milestones totaling US $168 million, as well as tiered royalties on sales of approved products.
"Our fourth agreement for the use of the GLAAS platform further demonstrates the broad applicability of this approach not only in cancer and infectious diseases, but now in allergic diseases as well," said Stephen Brady, chief business officer at Immune Design. "Due to the immune dysfunction leading to allergic diseases, GLAAS' mechanism of action is well suited to correct the imbalance, allowing for the potential of new therapeutics in the targeted indication that currently uses century-old technologies. We are pleased that Sanofi has decided to develop products for this often life-threatening and growing food allergy."
Under an existing collaborative research arrangement, Sanofi and Immune Design have generated a large set of preclinical data demonstrating that certain formulations within GLAAS, when given prophylactically or therapeutically, can shift the immune responses in a way that may result in significant protection and reduction from allergy symptoms.
About Sanofi
Sanofi, an integrated global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, and consumer healthcare, emerging markets, animal health and the new Genzyme. Sanofi is listed in Paris (EURONEXT:SAN) and in New York (NYSE:SNY).
About GLAAS
Immune Design's GLAAS platform works in vivo and is based on a small synthetic molecule called GLA, which stands for glucopyranosyl lipid adjuvant. GLA selectively binds to the TLR4 receptor and causes potent activation of dendritic cells (DCs) leading to the production of cytokines and chemokines that drive a Th1-type immune response. When GLA is accompanied by an antigen and injected into a patient, the combination is taken up by DCs and leads to the production and expansion of immune cells called CD4 T helper lymphocytes with a Th1 phenotype. These CD4 T cells play a key role in boosting pre- existing CTLs that are specific to the same antigen; and providing help to other immune cells, including B lymphocytes that are the precursor to antibodies, and natural killer cells that are also important in the overall immune response. Immune Design believes that GLAAS product candidates have the potential to target multiple types of cancer, as well as infectious, allergic and autoimmune diseases. GLAAS-based product candidates have now been evaluated in over 1000 subjects in Phase 1 and Phase 2 trials demonstrating an acceptable safety profile and efficacy.
About Immune Design
Immune Design (Nasdaq:IMDZ) is a clinical-stage immunotherapy company employing next-generation in vivo approaches to enable the body's immune system to fight disease. The company's technologies are engineered to activate the immune system's natural ability to create tumor-specific cytotoxic T cells, while enhancing other immune effectors, to fight cancer and other chronic diseases. Immune Design's three on-going Immuno-oncology clinical programs are the product of its two synergistic discovery platforms: DCVexTMand GLAASTM, the fundamental technologies of which were licensed from the California Institute of Technology and the Infectious Disease Research Institute, respectively. Immune Design has offices in Seattle, Washington and South San Francisco, California. For more information, visit www.immunedesign.com.
Sanofi Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, the Group's ability to benefit from external growth opportunities, trends in exchange rates and prevailing interest rates, the impact of cost containment policies and subsequent changes thereto, the average number of shares outstanding as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2013. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.
Immune Design Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend", "believe" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Immune Design's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include statements regarding the receipt of milestone and royalty payments, the potential to develop new therapeutics and the potential of any future products to prevent and reduce allergy symptoms. Factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Immune Design's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" contained therein. Except as required by law, Immune Design assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
References
- "Food Allergy - A Rising Global Health Problem," World Allergy Week 2013. 8-14 April 2013. http://www.worldallergy.org/UserFiles/file/WorldAllergyWeek2013final.pdf. Accessed online, July 28, 2014.
- Mills EN, Mackie AR, Burny P, Beyer K, Frewer L et al. "The prevalence, cost and basis of food allergy across Europe." Allergy 2007; 62:717-722.
- Fiocchi A, Sampson HA. "Food Allergy", Section 2.5, in WAO White Book on Allergy, Pawankar R, Canonica GW, Holgate ST, and Lockey RF, editors (Milwaukee, Wisconsin: World Allergy Organization, 2011), pp. 47-53.
- National Institute of Allergy and Infectious Diseases, National Institutes of Health. Report of the NIH Expert Panel on Food Allergy Research. 2006. Accessed online, July 25, 2014. http://www.niaid.nih.gov/topics/foodallergy/research/pages/reportfoodallergy.aspx
- 5. Clark S, Espinola J, Rudders SA, Banerji, A, Camargo CA. Frequency of US emergency department visits for food-
related acute allergic reactions. J Allergy ClinImmunol. 2011; 127(3):682-683.
CONTACT:
Amy BA, Ph.D.
Sanofi Global R&D Communications
Amy.Ba@sanofi.com
Tel: 646-207-4935
Julie Rathbun
Rathbun Communications (Immune Design)
julie@rathbuncomm.com
Tel: 206-769-9219
Latest News
Collaboration will focus on co-development of unique datasets to understand health in everyday life
January 10, 2022 06:00 AM Eastern Standard Time
SAN MATEO, Calif.--(BUSINESS WIRE)--Evidation, the company creating new ways to measure and improve health in everyday life, is expanding its decade-long collaboration with Sanofi to build upon their joint real-world data initiatives. This new phase will focus on the co-development of unique datasets to develop and validate new measures of health and wellness.
Evidation’s collaboration with Sanofi has delivered groundbreaking results to date, with over 20 studies conducted across 10 therapeutic areas, including diabetes and Type 2 Inflammation, more than 500,000 patients reached, and four studies published. This continued collaboration will further the work Evidation and Sanofi have pioneered to translate person-generated health data into quantified clinical and economic outcomes, a key priority for both companies.
“After nearly a decade of working with Sanofi, we are proud to expand this collaboration agreement to advance the role that real-world data and analysis can play in better understanding health and disease,” said Christine Lemke, Evidation co-founder and co-CEO. “Sanofi has led the way in garnering insights from real-world data in R&D and we’re excited to advance our work together into its next decade."
Sanofi and Evidation announced a prior expansion of their work together in 2017, in addition to Sanofi’s investment in Evidation in the same year.
“Real-world evidence is critical to help us better understand the patient’s health and wellness journey outside of traditional healthcare visits,” said Arnaud Robert, Executive Vice President, Chief Digital Officer, Sanofi. “Through our expanded collaboration with Evidation, we can further our ambition to transform the practice of medicine by connecting more closely with patients and citizens, expanding our geographic capabilities, and increasing diversity to better represent the global population.”
This announcement comes as biopharmaceutical companies, regulators, and payers are working to develop new guidelines on how real-world data should be incorporated into the development and approval of therapeutics. Evidation and Sanofi will continue to contribute to this conversation through similar industry-leading research.
The Evidation network is made up of more than 4.4 million individuals across all 50 states, representing 9 out of every 10 U.S. ZIP codes, allowing organizations like Sanofi access to a highly engaged, diverse population and privacy-conscious research platform.
ABOUT EVIDATION
Evidation measures health in everyday life and enables anyone to participate in ground-breaking research and health programs. Built upon a foundation of user privacy and control over permissioned health data, Evidation’s platform is trusted by millions of individuals—generating data with unprecedented speed, scale, and rigor. We partner with leading healthcare companies to understand health and disease outside the clinic walls. Guided by our mission to enable and empower everyone to participate in better health outcomes, Evidation is working to bring people individualized, proactive, and accessible healthcare—faster. Founded in 2012, Evidation is headquartered in California with additional offices around the globe. To learn more, visit evidation.com, or follow us on Twitter @evidation.
Contacts
MEDIA CONTACT
Matt Miller
press@evidation.com
Dr. Suliman brings over 25 years of drug development, deal-making and company building expertise as ReCode continues advancing its pipeline of novel genetic medicines using its powerful selective organ targeting (SORT) LNP delivery platform
Menlo Park, Calif. And Dallas, Texas – January 10, 2022 – ReCode Therapeutics (the “Company”), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the appointment of Shehnaaz Suliman, M.D., MBA, M.Phil., as chief executive officer and as a member of the board of directors effective today. Former CEO, David Lockhart, Ph.D., will transition to the role of chief scientific officer and will remain president and a member of the board of directors.
“I am excited about furthering the development of transformative therapeutics for patients with ReCode’s first-in-class LNP platform and promising genetic medicines pipeline,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer, ReCode Therapeutics. “There is vast untapped potential in RNA medicines and the delivery of genetic payloads to modify and potentially remedy a wide variety of life-limiting diseases. I am humbled to lead ReCode into its next stage of growth and look forward to partnering with Dr. Lockhart and the team to continue to execute our mission to develop a new generation of first-in-class mRNA-based and gene editing therapies for patients.”
“Dr. Suliman has an exceptional track record of building and transforming small and large life science companies into leading biotechnology companies. Her impressive business acumen, ability to advance assets through the clinic and competency in deal-making make her an outstanding fit for CEO as we enter our next chapter of growth,” said David Lockhart, Ph.D., president and chief scientific officer, ReCode Therapeutics. “I am personally very excited to partner with Dr. Suliman, and we are invigorated by her arrival and look forward to leveraging her expertise as we advance our powerful LNP platform and robust pipeline of treatments for patients with life-limiting genetic diseases.”
Dr. Suliman is a physician, drug developer and dealmaker with over 25 years of experience building and transforming small and large biopharmaceutical companies. She most recently served as president and chief operating officer of Alector, a leading immuno-neurology and immuno-oncology company where she played a critical role growing, scaling, raising capital and advancing a late-stage development pipeline. Prior to Alector, she served as senior vice president, corporate development and strategy at Theravance Biopharma from 2017 to 2019, where she helped deliver a $1B profit sharing partnership with Janssen for the company’s lead JAK inhibitor program. Prior to Theravance, Dr. Suliman was vice president and global head, immunology, infectious diseases and specialty care at Roche from 2015 to 2017. Dr. Suliman worked for Genentech as a group leader and project team leader in the R&D Portfolio Management and Operations Group from 2010 to 2015, where she oversaw an early development portfolio of more than 30 programs across multiple therapeutic areas. She held various management roles of increasing responsibility at Gilead Sciences, Inc. between 2005 and 2010 and played a significant role in forward-integrating Gilead into new therapeutic areas through M&A. Prior to Gilead, Dr. Suliman was an investment banker with Lehman Brothers and Petkevich & Partners, advising numerous public and private companies on buy- and sell-side transactions. She was named as one of the 2017 Fiercest Women in Life Sciences and as one of the National Diversity Council’s Power 50 in 2021 (Most Influential Women in Business). Dr. Suliman serves as an independent director on the Board of Directors of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and 10x Genomics (NASDAQ: TXG). Dr. Suliman received her M.D. (MB, ChB) from the University of Cape Town Medical School, South Africa, and holds an MBA, with distinction, and M.Phil. degrees from Oxford University, where she was a Rhodes Scholar.
About ReCode Therapeutics
ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using its powerful LNP delivery technology to target organs and tissues beyond the liver. The Company’s pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia. The Company is leveraging its proprietary LNP platform and nucleic acid technologies and utilizing systemic and direct delivery for mRNA-mediated replacement and gene editing/correction in target cells, including stem cells. For more information, visit www.recodetx.com and follow us on Twitter @ReCodeTx and LinkedIn.
Media Contact:
Will Zasadny
Canale Communications, Inc.
Will.zasadny@canalecomm.com
(619) 961-8848
Investor Contact:
Sarah McCabe
Stern Investor Relations
sarah.mcCabe@sternir.com
IR@recodetx.com
– Alain Baron, M.D., Escient Co-founder and CEO Retires –
SAN DIEGO, Calif., January 5, 2022 — Escient Pharmaceuticals, a clinical-stage company focused on discovering and developing novel, first-in-class therapies to address serious, unserved medical needs, today announced the appointment of Joshua Grass, MBA as Chief Executive Officer effective immediately. Mr. Grass will join the company’s Board of Directors. He succeeds Alain Baron, M.D., who recently retired and will remain a strategic advisor to Escient through the first quarter of 2022.
“I am very excited to be leading Escient during this next phase in its evolution,” said Mr. Grass. “The team has developed very elegant science in characterizing several Mrgprs as potentially important drug targets for large unmet needs. Now, with drug candidates against these targets, the company is poised to translate this science into important medicines for neuroinflammatory and immune activation diseases.”
Prior to joining Escient, Mr. Grass most recently served as CEO of Modis Therapeutics. In 2017, Mr. Grass launched ModisTx with a $30 million Series A financing while working at F- Prime Capital as an Entrepreneur in Residence. ModisTx was acquired by Zogenix in 2019 for $250 million upfront and $400 million in total consideration. Before joining F-Prime, Mr. Grass was a member of the BioMarin senior executive management team. During his 15-year tenure there, he led the acquisition and divestiture of many technologies and products and participated in the global development and launch of multiple products to treat rare genetic diseases. He started his career in biotech as a scientist at Cerus Corporation before holding roles in finance and equity research at boutique investment banks. Mr. Grass earned a Bachelor of Science in Biology from California Polytechnic State University, San Luis Obispo and an MBA in Finance and Entrepreneurship from the William E. Simon School of Business at the University of Rochester.
“Josh’s deep industry experience and impressive track record combined with his scientific and business background and dynamic leadership style make him the right leader for Escient. We are excited to have him at the helm and welcome him to the role.” said Marcus Boehm, Ph.D., Escient’s Co-founder and Chief Scientific Officer.
“On behalf of the Board of Directors and the entire team at Escient, I want to thank Alain for his leadership in building a world class company and team. Escient is well-positioned for the next stage of growth in Mrgpr drug discovery and clinical development,” added Kathleen Sereda Glaub, Chair of the Board of Directors at Escient.
About Escient Pharmaceuticals
Escient Pharmaceuticals is a clinical-stage company focused on discovering and developing novel, first-in-class therapies to address serious, unserved medical needs. The two most advanced programs are focused on new chemical entity antagonists targeting MrgprX4 and MrgprX2 for the treatment of cholestatic/uremic pruritus, and a broad range of mast-cell mediated disorders, respectively. Based in San Diego, Calif., Escient is led by an experienced team of biotechnology entrepreneurs with specific expertise in GPCR drug discovery and development and funded by top-tier life science investors, including The Column Group, 5AM Ventures, Sanofi Ventures, Cowen Healthcare Investors, Redmile Group, Perceptive Advisors, and Osage University Partners.
Visit www.escientpharma.com to learn more.
Contact:
Cory Tromblee
Scient Public Relations
cory@scientpr.com
Svetlana Makhni
Escient Pharmaceuticals, Inc.
info@escientpharma.com
Acquisition will give Aetion customers ability to tap into previously inaccessible, high utility, global health data to conduct transformational healthcare research.
NEW YORK, NY and OTTAWA, ON, January 4, 2022 — Aetion, the leading regulatory-grade real-world evidence (RWE) technology provider, today announced that it has acquired Replica Analytics, a pioneer in using artificial intelligence (AI) for synthetic health data generation.
Replica’s AI technology generates synthetic, privacy-protected copies of real-world data (RWD) that preserve the integrity and utility of source data. Through Replica, Aetion will open up new avenues for better understanding treatment pathways, safety and effectiveness, and improve standards of care.
“Replica enhances Aetion’s technology portfolio to open access to previously inaccessible RWD,” said Dr. Jeremy Rassen, Co-Founder and President of Aetion. “Our shared belief in rooting technology innovation in scientific rigor assures that together Replica and Aetion will accelerate the impact of RWE on improving access to higher quality, more affordable healthcare, globally.”
Replica’s advanced technology expands Aetion’s offerings, including its fit-for-purpose data services, by giving customers more privacy-protected data options when using the Aetion Evidence Platform®—an especially important feature in rare disease and specialty research. Fortune 50 companies already use Replica’s technology to enable fast and effective access to high utility, synthetic versions of health data across their organizations while meeting regulatory obligations and compliance with privacy laws like the Health Insurance Portability and Accountability Act (HIPAA) in the U.S. and the General Data Protection Regulation (GDPR) in Europe. These customers include some of the most prominent life sciences research organizations in the world.
“Despite the tremendous number of valuable RWD sources, the vast majority of the world’s health data is still inaccessible to researchers because of privacy concerns,” said Dr. Khaled El Emam, Replica Analytics’ Co-Founder and CEO, now SVP & General Manager of Replica Analytics at Aetion. “Replica’s mission is to protect that privacy while making the world’s health data responsibly accessible for decision-grade RWE. Becoming part of Aetion and its ecosystem enables us to accelerate and expand that mission by deploying our technology with more data sources and in new applications.”
Aetion’s acquisition of Replica Analytics follows a series of recent high-profile announcements for the company. Most recently, the European Medicines Agency (EMA) selected Aetion to support safety and efficacy research in Europe. Also in 2021, Aetion announced a collaboration with the U.S. Food and Drug Administration (FDA) to generate RWE on inpatient COVID-19 therapies, a partnership with Paris-based AI solutions company Quinten Health to reduce research timelines, and a collaboration with England’s National Institute for Health and Care Excellence (NICE) to explore how RWE can be used to study real-world clinical effectiveness.
About Aetion
Aetion is a healthcare analytics company that delivers real-world evidence for the manufacturers, purchasers, and regulators of medical treatments and technologies. The Aetion Evidence Platform® analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on safety, effectiveness, and value. Founded by Harvard Medical School faculty members with decades of experience in epidemiology and health outcomes research, Aetion informs healthcare’s most critical decisions—what works best, for whom, and when—to guide product development, commercialization, and payment innovation.
Aetion is based in New York City and backed by investors including Warburg Pincus, New Enterprise Associates (NEA), Flare Capital Partners, Greenspring Associates, Lakestar, B Capital, Foresite Capital, Town Hall Ventures, McKesson Ventures, Sanofi Ventures, EDBI, Johnson & Johnson Innovation—JJDC, Inc., UCB, Amgen Ventures, and Horizon Health Services, Inc. Learn more at aetion.com and follow us at @aetioninc.
About Replica Analytics
Replica Analytics is a developer of unique technologies for generating privacy protective synthetic data that maintains the statistical properties of real data. The Replica Synthesis software provides a full suite of synthetic data generation and evaluation capabilities that can solve multiple grand challenges facing the life sciences industry, and health research in general. Synthetic data enables rapid innovation by accelerating the development of AI models and accelerating clinical studies through data simulations. For more information, visit: https://replica-analytics.com/
Aetion
+1-844-823-8466
US: +1-646-513-2200
Europe: +33-983-887-249
New York
Headquarters 5 Penn Plaza 7th Floor
New York, NY 10001
Boston
Science & Technology Office
50 Congress Street
Suite 1025
Boston, MA 02109
Los Angeles
Science & Technology Office
840 Apollo Street
Suite 100
El Segundo, CA 90245
Barcelona
European Office
Can Rabia 3-5, Planta 4
08017 Barcelona
Spain
SAN CARLOS, Calif., December 17, 2021 – Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced that the company has received U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for GLM101 for the treatment of PMM2-CDG and has initiated dosing healthy volunteers in a Phase 1 clinical study. GLM101 is a mannose-1-phosphate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG Type Ia. PMM2-CDG is the most prevalent congenital disease of glycosylation but has no FDA-approved treatments.
“The initiation of this clinical study is an important milestone for Glycomine and for PMM2-CDG patients and their families,” said Peter McWilliams, Ph.D., CEO of Glycomine. “We have demonstrated in preclinical studies that GLM101 can restore the glycosylation pathway that is disrupted in PMM2-CDG, and we look forward to advancing our clinical studies to confirm the potential of GLM101 as an important disease-modifying therapy for PMM2-CDG patients.”
“PMM2-CDG is a serious rare disease with no therapeutic options and represents a critical unmet medical need,” said Horacio Plotkin, M.D., FAAP, Chief Medical Officer of Glycomine. “Glycomine has developed a proprietary approach to delivering mannose-1-phosphate intracellularly to overcome the deficiency that leads to PMM2-CDG. The open-label Phase 1 study will evaluate safety and tolerability in healthy volunteers. With successful completion, we look forward to opening enrollment for patients, which is planned for the second half of 2022.”
About GLM101, a Potential Treatment for PMM2-CDG (CDG-Ia)
Glycomine’s GLM101 is a mannose-1-phosphate replacement therapy in development to treat PMM2-CDG. PMM2-CDG is a severe multisystem disorder with symptoms such as coagulopathies, endocrinopathies, hypotonia, stroke-like episodes, as well as immune and nervous system disfunctions, and resulting mortality of 20% in the early years of life. PMM2-CDG is caused by genetic mutations that lead to a deficiency of the enzyme phosphomannomutase 2 (encoded by the PMM2 gene). PMM2 converts mannose-6-phosphate to mannose-1-phosphate, which is an essential sugar molecule in the N-glycosylation pathway and is crucially important for proper glycoprotein structure and function. GLM101 is designed to deliver mannose-1-phosphate directly into cells and thereby bypass the PMM2 enzyme deficiency and address all disease-causing PMM2 mutations to restore pathway function. GLM101 has received Orphan Drug Designation in the U.S. and Europe and Rare Pediatric Disease Designation in the U.S.
About Glycomine, Inc.
Glycomine is developing orphan drugs for serious rare disorders of metabolism and protein misfolding for which no other therapeutic options exist. The company's approach is to use replacement therapies – substrates, enzymes, or proteins – and to target those molecules to clinically relevant cellular compartments. The company is based in San Carlos, California and supported by leading international life sciences investors.
Corporate Contact: Peter McWilliams, Ph.D., info@glycomine.com
Media Contact: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com, +1.858.344.8091
- Study of lead candidate to provide important information on safety, pharmacokinetics and pharmacodynamics to inform further clinical development in chronic inflammatory diseases
- Highlights continued progress advancing a differentiated portfolio of novel NLRP3 inflammasome inhibitors to treat both peripheral- and neuro- inflammatory diseases
BOSTON, SEATTLE and CAMBRIDGE, UK – November 4, 2021 – NodThera, a clinical-stage biotechnology company developing a new class of medicines that inhibit the NLRP3 inflammasome to treat chronic inflammation, today announced that the first healthy volunteers have been dosed in a Phase 1 clinical trial of its lead investigational candidate, NT-0796.
NT-0796 is a small molecule NLRP3 inflammasome inhibitor with differentiated novel chemistry that provides unprecedented potency and potential for prolonged pharmacodynamic (PD) effect, with the ability to cross the blood brain barrier in preclinical species. NT-0796 selectively inhibits NLRP3, the upstream regulator of the body’s inflammation response, to reduce levels of both IL-1β and IL-18 – pro-inflammatory cytokines known to play a role in chronic inflammation underlying a wide range of chronic diseases. Pharmacokinetic (PK) and PD data from an ex vivo IL-1β/IL-18 stimulation assay and cerebrospinal fluid (CSF) sampling in the Phase 1 study will inform further clinical development.
“The recent convergence of key insights into innate immunity, IL-1β/IL-18 and the NLRP3 inflammasome have revolutionized our understanding of chronic disease,” said Adam Keeney, Ph.D., CEO of NodThera. “As one of the first companies to recognize the importance of NLRP3 in the inflammation cascade, we look forward to gathering important human data from the Phase 1 clinical study of our lead candidate NT-0796 so we can accelerate innovation for patients with limited treatment options.”
The primary objective of this study is to assess the safety and tolerability of NT-0796, while secondary objectives include assessment of PK and PD (ability to lower IL-1 and IL-18 levels) and CSF sampling to assess NLRP3 target engagement and compound exposure after single and multiple ascending doses.
“NT-0796 leverages novel chemistry that is unlike any other NLRP3 inflammasome inhibitor in the field. It is designed to deliver key advantages in PK and PD, with the potential to cross the blood brain barrier,” said Donald Johns, M.D., Chief Medical Officer of NodThera. “The NLRP3 inflammasome is a key driver of diseases that span different parts of the body, from common ailments such as osteoarthritis, to cardiovascular disease, Alzheimer’s Disease, cancer, and beyond. Unlocking this treatment potential provides the opportunity to impact many patients whose quality of life is negatively affected by chronic inflammatory disease.”
About NodThera
NodThera is a clinical-stage biotechnology company developing a new class of potent and selective NLRP3 inflammasome inhibitors for the treatment of diseases driven by chronic inflammation. Led by an experienced management team, NodThera is leveraging new insights into inflammasome biology and chemistry to build a portfolio of highly differentiated small molecule NLRP3 inflammasome inhibitors. The company was founded in 2016 and financed by 5AM Ventures, Cowen Healthcare Investments, Epidarex Capital, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera is headquartered in Lexington, MA, with additional locations in Cambridge, UK and Seattle, WA. Learn more at www.nodthera.com or follow us on LinkedIn.
Media Contact
Darby Pearson
Verge Scientific Communications
dpearson@vergescientific.com
Brings more than 15 years of CNS drug discovery and development experience
Copenhagen, Denmark and Boston, US, 1st November 2021 – Muna Therapeutics (“Muna”), pioneering the development of novel, first-in-class small molecule therapeutics for neurodegenerative diseases, today announces the appointment of Dr. Niels Plath as Chief Scientific Officer.
Dr. Plath is a molecular biologist and neuroscientist with more than 15 years of experience in academia and biopharma. He was previously the acting Head of Global Research at Lundbeck, leading drug discovery and development for neurologic and psychiatric diseases. Prior to this role, Dr. Plath was Vice President for Neuroscience, leading teams that brought several drug candidates into clinical development, including alpha-synuclein and tau targeting antibodies, idalopirdine and Nalmefene.
Rita Balice-Gordon, Chief Executive Officer of Muna Therapeutics, said: “We are delighted to welcome Niels as Muna’s Chief Scientific Officer. He shares our commitment to discover and develop disease modifying therapeutics to address the staggering unmet need experienced by patients with neurodegenerative disorders around the world.”
Henrijette Richter, Managing Partner at Sofinnova Partners, said: “Niels has broad biopharma R&D experience that will be invaluable as Muna continues to progress its innovative pipeline of potential disease-modifying medicines for neurodegenerative diseases. We are very pleased to be supporting Muna’s world-class global team.”
Dr. Niels Plath, Chief Scientific Officer of Muna Therapeutics, said: “I’m excited to be joining Muna Therapeutics to further develop its promising, cutting-edge pipeline of neurodegenerative disease therapeutics. I’m looking forward to working with this stellar team to advance promising treatments for patients to significantly improve their quality of life.”
Dr. Plath obtained a PhD in Neuroscience from the Free University of Berlin, Germany, focusing on the role of immediate early genes in neuronal plasticity, learning and memory. Following a postdoctoral fellowship supported by the Human Frontier Science Program, he joined biopharma in 2005 to pursue research and development of treatments for patients with CNS disorders. Dr. Plath has authored many scientific publications in peer-reviewed journals, is a guest lecturer in Neuroscience at the University of Copenhagen and has given numerous conference talks at meetings around the world.
ENDS
For more information please contact:
Muna Therapeutics
Rita Balice-Gordon, CEO
Email: balicegordon@munatherapeutics.com
Optimum Strategic Communications
Mary Clark, Stella Lempidaki, Zoe Bolt
Tel: +44 (0) 20 3922 1906
Email: muna@optimumcomms.com
About Muna Therapeutics
Muna Therapeutics is a private biopharmaceutical company founded in 2020 and based in Copenhagen, Denmark and Leuven, Belgium. Muna discovers and develops therapies that slow or stop devastating neurodegenerative diseases including Alzheimer’s, Frontotemporal Dementia and Parkinson’s. These disorders impact memory, movement, language, behavior and personality resulting in disability and death of millions of patients around the globe. We focus our groundbreaking science on identifying new medicines to preserve cognition and other brain functions and enhance resilience to neurodegenerative diseases. Our name reflects this focus: Muna means ‘to remember’ in Old Norse. www.munatherapeutics.com
NEW YORK--(BUSINESS WIRE)--Ovid Therapeutics Inc. (Ovid), a privately held biopharmaceutical company focused on developing therapies for rare and orphan diseases of the brain, today announced that Bart Friedman has joined the Company’s Board of Directors. Mr. Friedman is a senior partner at the Wall Street law firm Cahill Gordon & Reindel LLP and is Chairman of its Business Development Committee.
“We are delighted that Bart is joining our Board. Our goal is to build a world-leading neurology company with a Board of unparalleled and diverse capabilities. Bart’s broad experience, exceptional reputation, deep knowledge, and intellect are exactly what we require to achieve this goal,” said Dr. Jeremy Levin, Chief Executive Officer and Chairman of Ovid. “It is a privilege to have the opportunity to work with him as we seek to bring medicines to patients and help the families of those with these significant disorders.”
At Cahill Gordon & Reindel, Mr. Friedman advises leading financial institutions and global corporations, boards of directors, audit committees, and officers and directors of publicly held companies in significant corporate and securities matters, corporate compliance, and enforcement challenges. Mr. Friedman chairs the Board of Directors of the Sanford C. Bernstein Mutual Funds, is the Lead Independent Director of Allied World Assurance Holdings, serves on the Board of Trustees of The Brookings Institution and chairs its Audit Committee, and serves on the Board of Lincoln Center for the Performing Arts and chairs its Audit Committee. Mr. Friedman also serves on the Membership Committee of the Council on Foreign Relations. Earlier in his career, Mr. Friedman worked at the Securities and Exchange Commission as Special Counsel and later as Assistant Director. He earned his J.D. at Harvard Law School and was on the research faculty of Harvard Business School during and following his graduation from Harvard Law School and prior to joining Cahill.
Mr. Friedman commented, “I have been impressed with Ovid’s mission, strategy, and progress as it becomes a pre-eminent global, commercial company focused on orphan diseases of the brain. This is an important area of medical need, and Ovid is well-positioned to play a leading role. I am honored to serve on Ovid’s Board.”
About Ovid Therapeutics Inc.
Ovid Therapeutics Inc. is a privately held, New York-based, biopharmaceutical company committed to transforming the lives of patients with rare and orphan diseases of the brain. Ovid focuses on patients and their unmet medical needs. Using the significant operational, product development, and business development experience of its management team, Ovid aims to become a leading neurology company, with multiple products and a rich pipeline, coupled with compelling research and development. Ovid recently completed a substantially oversubscribed $75 Million Series B financing led by Fidelity Management and Research Company and including Cowen Private Investments, Sanofi-Genzyme BioVentures, Tekla Capital Management, Sphera Global Healthcare Fund, Jennison Associates (on behalf of certain clients), Redmile Group, DoubleLine Equity Healthcare fund, and Cormorant Asset Management, as well as additional blue chip mutual funds and leading life sciences investors.
For more information on Ovid, please visit http://www.ovidrx.com.
Contacts
Burns McClellan, on behalf of Ovid Therapeutics
Justin Jackson, 212-213-0006, ext. 327
jjackson@burnsmc.com
NEW YORK--(BUSINESS WIRE)--Click Therapeutics, Inc. (“Click”), a leader in Digital Therapeutics™ as prescription medical treatments, today announced that it has closed a $52 million upsized Series B financing. The financing round was co-led by new investors H.I.G. BioHealth Partners (“H.I.G. BioHealth”) and Accelmed Partners II (“Accelmed”), with participation from Health Catalyst Capital, Revelation Partners, and a top biotechnology hedge fund, and from existing investors Sanofi Ventures, K2 HealthVentures, Hikma Ventures, and Ridgetop Health. Proceeds from the Series B financing will be used to accelerate the development and commercialization of Click’s internal prescription digital therapeutic pipeline and advance Click’s platform capabilities.
Click’s pipeline of innovative digital therapeutics spans multiple therapeutic areas, from psychiatry and chronic pain to cardiometabolic and autoimmune disorders. In addition to its internal pipeline programs, Click has entered into landmark collaboration agreements with Otsuka to develop and commercialize a prescription digital therapeutic for treatment of Major Depressive Disorder (MDD), and with Boehringer Ingelheim to develop and commercialize a prescription-based digital therapeutic to aid in the treatment of schizophrenia.
“Closing our Series B is an important milestone for Click that will allow us to further scale our proprietary Click Neurobehavioral Intervention (CNI) Platform and fund the development of new and innovative digital therapeutics for patients in need,” said David Benshoof Klein, Co-Founder and CEO of Click. “We are excited to welcome our new investors and deepen our relationships with existing investors, all of whom share our vision of a new healthcare landscape in which prescription digital therapeutics play a prominent role alongside traditional pharmacological treatments.”
In conjunction with the Series B financing, Click’s Board of Directors will expand to include Alex Zisson, Managing Director at H.I.G. BioHealth, and Dr. Uri Geiger, Founder and Managing Partner of Accelmed.
“Digital therapeutics hold the promise to change the very paradigm of healthcare,” said Mr. Zisson. “Click has one of the very best platforms in this field, and we are excited to help the company improve patient outcomes and potentially lower healthcare costs.”
“Click is a prime example of the tremendous and growing potential for digital health software applications to enhance the lives of patients suffering from a variety of medical conditions,” commented Dr. Geiger. “Click is the ideal partner for Accelmed due to our shared belief that leveraging technology in healthcare improves patient outcomes and increases efficiencies throughout the healthcare system. We are excited to offer our financial and operational expertise and to work closely with David and the entire Click executive team to support the growth of Click’s innovative platform.”
About Click Therapeutics
Click Therapeutics, Inc. develops and commercializes software as prescription medical treatments for patients with unmet medical needs. Through cognitive and neurobehavioral mechanisms, Click’s Digital Therapeutics™ enable change within individuals, and are designed to be used independently or in conjunction with biomedical treatments. The Clickometrics® adaptive data science platform continuously personalizes user experience to optimize engagement and outcomes. Following a groundbreaking clinical trial, Click’s industry-leading smoking cessation program is available nationwide through a wide variety of payers, providers, and employers. Click’s lead prescription program has recently entered a pivotal, fully remote, randomized, controlled trial on the Verily platform for the treatment of Major Depressive Disorder in up to 540 adults. For more information on Click, visit ClickTherapeutics.com.
About H.I.G. BioHealth Partners
H.I.G. BioHealth Partners is the dedicated life-science investment affiliate of H.I.G. Capital. H.I.G. BioHealth Partners invests in a broad range of healthcare opportunities across sectors and stages, principally in companies developing therapeutic drugs, medical devices, and diagnostics for significant unmet medical needs. With approximately $400 million in committed capital, H.I.G. BioHealth Partners invests $5 million to $40 million per company over the life of an investment. For more information, please refer to the H.I.G. BioHealth Partners website at www.higbio.com.
About Accelmed Partners
Accelmed is a U.S.-based private equity firm focused on acquiring and investing in U.S. commercial stage, lower middle market HealthTech companies. Since 2009, Accelmed has deployed over $400 million into companies spanning medical devices, diagnostics, digital health and technology-enabled healthcare services. Accelmed seeks to accelerate value and scale innovation across the HealthTech field by bringing to bear the team’s industry experience, operational and financial expertise, and strong global relationships. For more information, please visit www.accelmed.com.
Contacts
Company Contact
Daniel Busby
VP, Investor Relations & Strategic Finance
dbusby@clicktherapeutics.com
Media Contact
Karen Sharma
ksharma@macbiocom.com
781-235-3060
The agency will use Aetion’s scientifically validated software platform to develop a system of studies that enables rapid evidence generation for COVID-19 and future pandemics
NEW YORK, NY, October 21, 2021 — Today, Aetion announces that it is expanding its relationship with the U.S. Food and Drug Administration (FDA) to use real-world evidence (RWE) to study COVID-19 interventions and advance regulatory science and innovation. FDA and Aetion will use Aetion Evidence Platform® (AEP), validated software that enables efficient, transparent, and reliable RWE research, to develop a framework and system of studies for the rapid assessment of COVID-19 inpatient medical products.
This project is designed to demonstrate how using a platform-based approach furthers regulatory learnings on the use of RWE to inform decision-making. The work will also provide a scalable infrastructure for the rapid development and evaluation of COVID-19 therapies, which can be applied for future public health emergencies. This contract will support the agency’s broader digital transformation efforts, which include its Technology and Data Modernization Action Plans as well as its recently announced Office of Digital Transformation.
“COVID-19 has created an urgent need to develop and apply innovative methods to assess novel interventions,” said Dr. Jeremy Rassen, co-founder and president of Aetion. “As FDA continues to advance its digital capabilities, Aetion is proud to partner with the agency in developing the rigorous scientific processes and RWE generation tools needed to quickly respond to future public health challenges.”
The aim of this project is to further data familiarity and protocol standards to support real-world data (RWD) analyses among the broader research community. FDA will work with Aetion to define and prioritize key research questions; identify fit-for-purpose data sources; develop appropriate, validated, and applicable measurement algorithms to capture key exposures, subgroups, confounding variables, and outcomes; design template epidemiological studies applicable to a range of treatments; implement studies and generate transparent reporting using AEP; and build and disseminate knowledge via peer-reviewed publications and other avenues.
Aetion and FDA will build on learnings from the research collaboration announced in May 2020, in which FDA and Aetion explored the utility of RWD to advance the understanding of and response to COVID-19. Since launching the collaboration, FDA and Aetion have developed mechanisms to assess data fitness for use; identified methodological good practices on working with RWD for COVID-19; and built the foundation for rapid-cycle analytics to address critical and emergent public health questions.
About Aetion
Aetion is a health care analytics company that delivers real-world evidence for the manufacturers, purchasers, and regulators of medical treatments and technologies. The Aetion Evidence Platform® analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on safety, effectiveness, and value. Founded by Harvard Medical School faculty members with decades of experience in epidemiology and health outcomes research, Aetion informs health care’s most critical decisions— what works best, for whom, and when—to guide product development, commercialization, and payment innovation.
Aetion is based in New York City and backed by investors including New Enterprise Associates (NEA), Warburg Pincus, Flare Capital Partners, Greenspring Associates, Lakestar, B Capital, Foresite Capital, Town Hall Ventures, McKesson Ventures, Sanofi Ventures, EDBI, Johnson & Johnson Innovation—JJDC, Inc., UCB, Amgen Ventures, and Horizon Health Services, Inc. Learn more at aetion.com and follow us at @aetioninc.
+1-844-823-8466
US: +1-646-513-2200
Europe: +33-983-887-249
New York
Headquarters
5 Penn Plaza 7th Floor
New York, NY 10001
Boston
Science & Technology Office
50 Congress Street
Suite 1025
Boston, MA 02109
Los Angeles
Science & Technology Office
840 Apollo Street Suite 100
El Segundo, CA 90245
Barcelona
European Office
Can Rabia 3-5, Planta 4
08017 Barcelona
Spain
- Financing co-led by Pfizer Ventures and EcoR1 Capital with participation from syndicate of world-class life sciences investors
- Company to advance mRNA and gene correction therapies into the clinic for cystic fibrosis and primary ciliary dyskinesia
- ReCode’s proprietary non-viral lipid nanoparticle (LNP) delivery platform to generate a deep pipeline of therapies that target the lung, liver and other extra-hepatic tissues
- Company to expand internal manufacturing capabilities to support research and clinical programs
Menlo Park, Calif. and Dallas, Texas – October 21, 2021 – ReCode Therapeutics (the “Company”), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the closing of an $80 million Series B financing round co-led by Pfizer Ventures and EcoR1 Capital. New investors include Sanofi Ventures, funds managed by Tekla Capital Management LLC, Superstring Capital and NS Investment. Existing investors who participated included OrbiMed, Vida Ventures, MPM Capital, Colt Ventures, Hunt Technology Ventures, L.P., and Osage University Partners (OUP). The proceeds from the Series B financing will be used to drive ReCode’s lead programs in primary ciliary dyskinesia (PCD) and cystic fibrosis (CF) into human clinical studies, expand the pipeline of treatments for patients with life-limiting genetic respiratory diseases, advance its LNP platform for organ-specific delivery of RNA and gene correction therapies and increase internal manufacturing capabilities.
“ReCode is working to unleash the power of genetic medicine by delivering therapies with our novel LNP platform, which has the potential to reach across a broad spectrum of diseases involving multiple organs and tissues,” said David Lockhart, Ph.D., CEO and president, ReCode Therapeutics. “The significant capital secured from such a respected group of investors, known for backing innovative biotechnology companies, enables us to accelerate delivery of impactful medicines to thousands of patients with genetic respiratory diseases in need of options, including those with CF and PCD.”
In connection with the closing of the financing, Rana Al-Hallaq, Ph.D., a partner at Pfizer Ventures and executive director for Pfizer Worldwide Business Development, has joined the ReCode board of directors. “Through this investment, we are excited to support ReCode in its development of these novel LNPs, which we believe, if successful, may significantly expand the potential of genetic medicine across therapeutic areas,” Al-Hallaq said.
Oleg Nodelman, founder and portfolio manager of EcoR1 Capital also joined ReCode’s Board of Directors in connection with the financing. “We are excited to co-lead ReCode’s Series B financing and to support the company as they advance their unique technology that enables the delivery of novel genetic medicines to target organs, tissues and cells. ReCode’s platform has the potential to unlock vast capabilities unaddressable by first-generation mRNA and gene editing programs and enable development of therapeutics for patients with diseases that have historically been untreatable.”
ReCode’s lead programs are focused on the genetic respiratory diseases PCD and CF. Recent preclinical data from the Company’s RNA-based CF program showed that its LNPs can deliver CFTR mRNA that restores cystic fibrosis transmembrane conductance regulator (CFTR) function in the CF patient-derived hBE cell model. Preclinical data from the Company’s inhaled mRNA-based program for the treatment of PCD demonstrated that its LNP formulations successfully delivered DNAI1 mRNA to target airway epithelial cells in hBEs, mice and NHPs, and that robust ciliary activity was restored in treated DNAI1- deficient hBE cells.
About ReCode Therapeutics
ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using its powerful LNP delivery technology to target organs and tissues beyond the liver. The Company’s pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia. The Company is leveraging its proprietary LNP platform and nucleic acid technologies and utilizing systemic and direct delivery for mRNA-mediated replacement and gene editing/correction in target cells, including stem cells. For more information, visit www.recodetx.com and follow us on Twitter @ReCodeTx and LinkedIn.
Media Contact:
Will Zasadny
Canale Communications, Inc.
will.zasadny@canalecomm.com
(619) 961-8848
Investor Contact:
Sarah McCabe
Stern Investor Relations
sarah.mccabe@sternir.com
IR@recodetx.com
BOSTON, MA—October 12, 2021—i2O Therapeutics, a developer of novel peptide and antibody based oral biologic products based on proprietary ionic liquid platform, announces the appointment of Kurt C. Graves as Executive Chairman. Mr. Graves brings 30 years of business leadership experience in leading global pharmaceutical and biotech companies.
“Kurt’s business acumen and expertise in setting vision, culture, and growth strategies across early, mid and late-stage bio-pharma companies will be invaluable at this pivotal time of corporate growth at i2O Therapeutics,” said Ravi Srinivasan, PhD, CEO of i2O Therapeutics. “Kurt has successfully led the development, launch, and build-out of multiple blockbuster brands and multi-billion dollar franchises, and his deep background in over 15 therapeutic areas, crafting major financings and partnerships, and working with peptides and biologics makes him the ideal selection to lead our Board.”
Over the last 10 years Mr. Graves has provided leadership to several innovative biotech companies including as Chairman, President and CEO of Intarcia Therapeutics, former Chairman of Radius Health, and as a Board member at Achillion Pharmaceuticals until it was acquired by Alexion, and at Seres Health. He was also E&Y’s New England Entrepreneur of the Year in 2015. Previously, Mr. Graves served as EVP, Head of Corporate & Business Development, Head of Strategic Drug Development and Program Management & Commercial at Vertex Pharmaceuticals. Prior to that, he was at Novartis Pharmaceuticals for nearly 10 years, most recently as the Global Head of the General Medicines Business & the first Global Chief Marketing Officer for the Pharmaceuticals division. Earlier in his career, at Merck and Astra-Merck he led the GI Business Unit responsible for Prilosec , Nexium , and Prilosec OTC .
“i20 Therapeutics has developed a transformational new platform for enabling the full potential of oral biologics including peptides, antibodies and nucleic acids. The company is focused on creating novel oral biologics that are also tunable and well-differentiated therapies that can provide superior clinical efficacy and safety benefits to patients. Our vision and scope of potential therapeutic impact is very exciting and directly aligns with my passion to help develop novel platform technologies and therapeutics that meaningfully enhance lives and real-world outcomes,” said Graves. “I look forward to working with the management team and Board and forming key external partnerships to help scale our platform, strengthen the organization, and advance a new generation of tunable oral biologics starting with programs in serious immune-mediated inflammatory diseases and metabolic therapeutic areas.
Mr. Graves received a B. S. in Biology from Hillsdale College and has attended educational and leadership development programs at Harvard University, University of Michigan, and the Wharton School of Management.
About i2O Therapeutics
i2O Therapeutics is the leader in exploiting the versatile properties of Ionic Liquids (ILs) for therapeutic development. i2O’s wholly owned pipeline consists of first-in-class and best-in-class transformative oral biologics with improved clinical benefits and a superior safety profile over current standard of care. With a focus on metabolic diseases, inflammatory diseases and other indications, i2O is delivering on the promise of oral biologics—considered the “holy grail biologic opportunity” by the pharmaceutical industry. Visit us at www.i2obio.com.
- Science 37 to debut on Nasdaq as a publicly traded company under ticker symbol “SNCE”
- Business combination will provide Science 37 with approximately $235 million in cash proceeds to support continued growth
- Science 37 to fuel its mission to enable universal access to clinical research which has proven to accelerate patient enrollment, minimize patient burden, and include underserved patient populations
- Investments targeted to enhance the Science 37 Operating System to decentralize clinical trial execution and enable more agile clinical trials
LOS ANGELES and NEW YORK, October 7, 2021 – Science 37, Inc., the Operating System for today’s agile clinical trials, announced today that it has completed its previously announced business combination with LifeSci Acquisition II Corp. (NASDAQ: LSAQ) (“LifeSci”), a blank check company targeting the biopharma, medical technology, digital health and healthcare services sectors. Shares of common stock of the combined company, named Science 37
Holdings, Inc. (“Science 37” or the "Company") will begin trading on the Nasdaq Global Market under the new ticker symbol "SNCE” today, October 7, 2021.
LifeSci shareholders approved the transaction at a special meeting on October 4, 2021. As a result of the transaction, Science 37 has received approximately $235 million total cash, net of fees and expenses paid in connection with the closing of the business combination, including the proceeds from the private placement completed in connection with the transaction. Science 37 intends to use the transaction proceeds to fund its decentralized trial technology platform, extend into new adjacencies, and power the next generation in clinical research. David Coman, Chief Executive Officer of Science 37, Inc., and Science 37 Inc.’s current executive team will continue to lead the combined company.
“The Science 37 Operating System has been proven to materially accelerate patient enrollment, provide meaningfully higher patient retention, significantly reduce patient burden and enable participation from underserved patient populations. This is all made possible through our full-stack, end-to-end technology platform and supported by specialized patient communities, telemedicine investigators, mobile nurses, and remote coordinators,” said Mr. Coman. “The additional capital from this transaction will help us deliver on our vision to be the category-defining operating system that powers every clinical trial as the industry shifts to more agile trial designs.”
Andrew McDonald, Ph.D., Chief Executive Officer of LifeSci, said, “Science 37’s extraordinary differentiation based on its technology platform and specialized networks represents the future of clinical trial operations. As a leader and innovator in agile clinical trials, Science 37 has the opportunity to transform the industry and impact positive change in millions of people's lives. We are proud to partner with David and his talented team as Science 37 begins its next chapter as a public company.”
Following the business combination, David Coman will serve on Science 37’s Board of Directors alongside Rob Faulkner, Managing Director at Redmile Group, as Chairman; John W. Hubbard, Ph.D., former Chief Executive Officer at Bioclinica and former Senior Vice President and Worldwide Head of Development Operations at Pfizer; Bhooshi de Silva, Senior Vice President, Global Head of Corporate Development, Strategy and Ventures, at PPD; Adam Goulburn, Ph.D., Partner at Lux Capital; Neil Tiwari, Partner of Private Healthcare Ventures at Magnetar Capital; and Emily Rollins, former Partner of Deloitte & Touche.
Advisors
Cowen and Perella Weinberg Partners LP served as financial advisors and Latham & Watkins LLP and DLA Piper LLP (US) served as legal advisors to Science 37. Cowen acted as sole placement agent to LifeSci Acquisition II Corp. in connection with the private placement. LifeSci Capital LLC acted as lead book-running manager to LifeSci Acquisition II Corp. in connection with its initial public offering in November 2020. Loeb & Loeb LLP served as legal advisor to LifeSci Acquisition II Corp. Cowen, William Blair, Baird, and Lake Street Capital Markets acted as capital markets advisors to Science 37.
About Science 37
Science 37's mission is to enable universal access to clinical research—making it easier for patients and providers to participate from nearly anywhere and helping to accelerate the development of treatments that impact patient lives. As a pioneer of decentralized clinical trials, the Science 37 Clinical Trial Operating System (OS) supports today’s more
agile clinical research designs with its full stack, end-to-end technology platform and specialized networks of patient communities, telemedicine investigators, mobile nurses, remote coordinators and connected devices. Configurable to enable any study type, the Science 37 OS can enable up to 15x faster enrollment, 28% better retention and 3x more diverse patient population with industry-leading workflow orchestration, evidence generation and data harmonization. For more information, visit https://www.science37.com.
About LifeSci Acquisition II Corp.
LifeSci Acquisition II Corp. (Nasdaq: LSAQ) is a blank check company formed for the purpose of entering into a merger, share exchange, asset acquisition, stock purchase, recapitalization, reorganization, or other similar business combination with one or more businesses or entities, pursuing targets that are focused on healthcare innovation in North America or Europe. For more information visit: https://lifesciacquisition.com/spac-2/.
Forward-Looking Statements
This press release contains certain forward-looking statements within the meaning of the federal securities laws, including statements regarding the benefits of the transaction between Science 37, Inc. and LifeSci, the services offered by Science 37 and the markets in which it operates, and Science 37’s projected future results. These forward- looking statements generally are identified by the words “believe,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “strategy,” “future,” “opportunity,” “plan,” “may,” “should,” “will,” “would,” “will be,” “will continue,” “will likely result” and similar expressions. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this press release, including but not limited to: (i) the ability to maintain the listing of Science 37’s securities on Nasdaq, (ii) volatility in the price of Science 37’s securities due to a variety of factors, including changes in the competitive and highly regulated industries in which Science 37 plans to operate, variations in performance across competitors, changes in laws and regulations affecting Science 37’s business and changes in its capital structure, (iii) the ability to implement business plans, forecasts, and other expectations, and to identify and realize additional opportunities, (iv) the risk that Science 37 may never achieve or sustain profitability, (v) the risk that Science 37 will need to raise additional capital to execute its business plan, which may not be available on acceptable terms or at all; and (vi) the potential adverse effects of the ongoing global COVID-19 pandemic. The foregoing list of factors is not exhaustive.
You should carefully consider the foregoing factors and the other risks and uncertainties described in the “Risk Factors” section of LifeSci’s definitive proxy statement/prospectus and registration statement on Form S-4 filed with the U.S. Securities and Exchange Commission (the “SEC”) and other documents filed by Science 37 from time to time with the SEC. These filings identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements, and Science 37 and LifeSci assume no obligation and do not intend to update or revise these forward- looking statements, whether as a result of new information, future events, or otherwise. Neither Science 37 nor LifeSci gives any assurance that either Science 37 or LifeSci will achieve their expectations.
Contacts For Media:
Margie Kooman
margie.kooman@science37.com
Nina Gill
Science37@10fold.com
For Investors:
Caroline Paul Gilmartin Group
investors@science37.com
- Completed enrolment of 150 pregnant women in a Phase 2 study in South Africa and dosing started in an additional 50 pregnant women in Uganda, as part of same study
- Phase 1 booster study started in the United Kingdom
- Regulatory approval obtained from Danish Medicines Agency and UK MHRA for the initiation of a Phase 2 study in pregnant women, in Denmark and United Kingdom
- Vaccine Expert and Biotech Entrepreneur Gerd Zettlmeissl appointed new Chairman of the Board of DirectorsLeading vaccine industry veterans join Scientific Advisory Board
Copenhagen, Denmark, 7 October 2021 – MinervaX, a privately held Danish biotechnology company developing a novel vaccine against Group B Streptococcus (GBS), today announces clinical progress on its maternal GBS vaccine as well as multiple additions to its leadership teams.
MinervaX is developing a maternal vaccine for the prevention of adverse pregnancy outcomes and life- threating infections caused by Group B streptococcus (GBS). GBS is responsible for nearly half of all life- threatening infections in newborns during the first 3 months of life as well as a portion of late-term abortions, premature deliveries, or stillbirths during pregnancy. Current preventative strategy is insufficient, and great medical needs exist, which may be addressed by a maternal GBS vaccine. MinervaX’s maternal GBS vaccine is based on adjuvanted proteins antigens covering close to 100% of clinical GBS isolates.
Clinical Update:
MinervaX announces today completed enrolment of 150 pregnant women in a Phase 2 study in South Africa, sponsored by the European Developing Countries Trial Partnership. Dosing of an additional 50 pregnant women in the same study has started in Uganda. The study is listed on clinicaltrials.gov under NCT04596878.
MinervaX has also started dosing healthy adult women previously receiving the Company’s GBS vaccine in a Phase 1 booster study in the United Kingdom. The study is listed on clinicaltrials.gov under NCT05005247.
Finally, MinervaX has received regulatory approval from the Danish Medicines Agency and UK MHRA to initiate a Phase 2 study in 270 pregnant women in Denmark and the United Kingdom.
Corporate Update:
Gerd Zettlmeissl joins MinervaX as new Chairman of the Board of Directors. Gerd brings a wealth of experience from the vaccine and broader biotech industry, including several successful biotech exits.
MinervaX announces the addition of Ralf Clemens, Jean-Paul Prieels, Peter Patriarca, Jean Smal, Clement
Lewin, and Inca Kusters as new members of its Scientific Advisory Board (SAB).
MinervaX ApS, Ole Maaløes Vej 3, DK-2200 Copenhagen N, Denmark VAT no. DK32673287
Commenting on the announcement, Per Fischer, Chief Executive Officer of MinervaX, said: “We are very pleased with the progress in clinical development of our maternal GBS vaccine since closing our last financing round in December 2020. The vaccine has to date demonstrated high immune responses even in individuals with low levels of preexisting immunity to GBS who are most at risk of invasive disease. The vaccine has also demonstrated a very promising safety profile in both non-pregnant and pregnant women in past and ongoing clinical studies. The progress represents a significant advancement towards initiating Phase 3 clinical studies”
“We are also very pleased to welcome Gerd Zettlmeissl as new Chairman of the Board of Directors as well as the members of the expanded Scientific Advisory Board. The additions bring valuable business and development expertise to the Company.”
Gerd Zettlmeissl, Chairman of MinervaX Board of Directors said: “I am very excited about the outstanding progress MinervaX is making in the development of one of the globally most promising GBS vaccine candidates and about the opportunity to support the company towards future success.”
Ralf Clemens, MinervaX Scientific Advisory Board member said: “Group B Streptococcus is a leading cause of neonatal sepsis and despite efforts since many years by academia and industry to develop a prophylactic vaccine progress so far is limited. The MinervaX phase 2 candidate has shown very promising immunogenicity data and an excellent safety profile, and the company has a clear plan of path to licensure. The studies in Uganda, UK and Denmark are a critical piece of that plan.”
ENDS
Enquiries
For more information on MinervaX, please contact:
Per Fischer, CEO: pbf@minervax.com
Funding to advance the company’s SMiRNA™ platform to identify clinical candidates in myotonic dystrophy type 1 (DM1), amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and tauopathies.
September 29, 2021 07:00 AM Eastern Daylight Time
BOSTON--(BUSINESS WIRE)--Expansion Therapeutics, Inc., a biotechnology company focused on developing transformative oral medicines for severe RNA-mediated diseases, today announced the close of an $80 million Series B financing. Cormorant Asset Management led the financing with participation from new investors Westlake Village BioPartners, Surveyor Capital (a Citadel company) and Logos Capital as well as Series A investors RA Capital Management, 5AM Ventures, Kleiner Perkins, Sanofi Ventures and Novartis Venture Fund. Proceeds from the financing will be used to advance the company’s small molecule RNA platform (SMiRNA™) to identify clinical candidates in myotonic dystrophy type 1 (DM1), amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and various tauopathies.
“Drugging RNA with small molecules has the potential to dramatically transform the lives of patients and we are excited to be supported by world-class investors who share our commitment to patients and our vision to develop innovative therapies to treat severe RNA-mediated diseases, including neurodegenerative and neurological disorders,” said Renato Skerlj, Ph.D., President and Chief Executive Officer of Expansion Therapeutics. “This financing milestone demonstrates significant investor confidence in the leadership team, the science and our mission to accelerate the preclinical and clinical development of our novel medicines utilizing our proprietary approach.”
Expansion focuses on structured RNA targets that are evolutionarily conserved or genetically demonstrated to cause disease. Disease-driving RNA structures amenable to ligand binding are identified by combining patient genetics with deep informatic analysis and then screened against Expansion’s proprietary RNA-focused libraries for small molecules that modulate the RNA target in a functionally relevant manner. Expansion’s novel structural biology SMiRNA™ platform is then utilized for the design and advancement of lead-like chemical matter that ultimately delivers an oral medicine to treat disease. Expansion is focused on neurological diseases where few treatment options are available for patients such as DM1 which is a system wide disorder affecting muscle, cardiac and brain function. A small molecule medicine that treats all aspects of the disease offers patients a significant benefit.
“Recent advances in understanding RNA structure and the science of targeting RNA with small molecules offer tremendous opportunities to transform the way RNA-mediated diseases are treated. We believe Expansion’s established focus on structured RNA targets position them as leaders in this emerging space,” said Andy Phillips, Ph.D., Managing
Director at Cormorant. “We’re extremely enthusiastic about the promise that the SMiRNA™ platform holds to deliverdisease-modifying therapies for a wide range of devastating neurological disorders, particularly neurodegenerativediseases,” added Raymond J. Kelleher, M.D., Ph.D., also Managing Director at Cormorant.
“The progression of Expansion’s lead programs, broad therapeutic potential of the platform, and the leadership team’sdemonstrated track record in the field of neuroscience compelled us to partner with this exceptionally strong group of drugdevelopers, as well as Cormorant and the rest of the investor syndicate,” said Andrew Levin, M.D., Ph.D., ManagingDirector at RA Capital Management. “We are confident in Expansion’s ability to leverage its differentiated platform topositively impact the lives of patients suffering from severe and debilitating neurological diseases who currently havelimited or no treatment options,” added Laura Tadvalkar Ph.D., Principal at RA Capital Management.
As part of its initiative to broaden its portfolio of small molecule medicines, Expansion also recently in-licensed two newresearch programs from Scripps Research, including a program targeting tau, an important driver of dementia disorders.The tau program builds on the observation that defective pre-mRNA encoding the production of tau protein is involved inthe formation of neurofibrillary tangles and has been linked to tauopathies such as Alzheimer’s disease, frontotemporaldementias, progressive supranuclear palsy and other neurodegenerative diseases.
Most recently, Expansion’s scientific founder, Matthew D. Disney, Ph.D. and colleague, Alicia Angelbello, Ph.D., wererecipients of the American Chemical Society’s Nobel Laureate Signature award for their pioneering work developing RNA-targeting medications for genetic diseases, including DM1.
About Expansion Therapeutics
Expansion Therapeutics is a biotechnology company focused on developing transformative oral medicines for severeRNA-mediated diseases including neurodegenerative disorders. Based on exclusive worldwide rights to groundbreakingresearch from the laboratory of Matthew D. Disney, Ph.D., at Scripps Research, Expansion has assembled the intellectualproperty, know-how, and proprietary enabling technologies and tools necessary to facilitate the creation of potent andspecific small molecule RNA modulators. Through this unique platform, Expansion is building a portfolio of novel RNA-targeted drug candidates with activity across a broad number of severe RNA-mediated disease indications. Headquarteredin Boston, Massachusetts, Expansion’s research facility is located in Jupiter, Florida. For more information, visitwww.expansionrx.com.
Contacts
Media:
Mike Beyer
Sam Brown Inc.
312-961-2502
mikebeyer@sambrown.com
CAMBRIDGE, Mass. – September 14, 2021 – ROME Therapeutics, a biotechnology company harnessing the power of the repeatome for drug development, today announced the completion of a $77 million Series B financing led by new investor Section 32. In addition, new investors Sanofi Ventures, Casdin Capital, Andreessen Horowitz and Alexandria Venture Investments participated in the round, alongside existing investors ARCH Ventures, GV and Mass General Brigham Ventures (formerly Partners Innovation Fund). Concurrent with the financing, Steven J. Kafka, Ph.D., managing partner at Section 32, and Jim Trenkle, Ph.D., head of investments at Sanofi Ventures, were appointed to ROME’s Board of Directors.
ROME’s mission is to harness the power of the repeatome – the roughly 60% of the human genome consisting of repetitive sequences of nucleic acids, known as repeats – to discover powerful new classes of medicines for cancer and autoimmune diseases. Repeats have long been considered part of the “dark genome,” but recent discoveries have demonstrated that the repeatome has pathological consequences in cancer, autoimmune disease and other therapeutic areas. Since launching in April 2020, ROME has identified multiple repeatome-derived targets and created an internal data science platform, called repeatomics, to analyze vast quantities of data available through the repeatome.
“In the short time since our founding, ROME has made significant progress uncovering the role and mechanism of the repeatome in cancer and autoimmune disease, which has led to our foundational repeatomics platform and identification of multiple tractable targets for drug discovery,” said Rosana Kapeller, M.D., Ph.D., president, chief executive officer and co-founder of ROME. “With the support of this group of premier investors, we are well-capitalized and resourced to continue advancing our lead programs into the clinic, expanding our pipeline of repeatome-derived programs and enhancing our repeatomics platform, as we seek to revolutionize the way cancer and autoimmune diseases are treated.”
“Traditional genomics has transformed how we think about drug development, diagnosis and treatment; however, for too long we have been unable to extend this approach to the ‘dark genome,’” said Dr. Kafka. “Leveraging the deep insights garnered by ROME’s experienced team of drug hunters and data scientists, ROME has developed innovative tools and techniques to generate novel insights from the repeatome in order to bring forward an entirely new and important class of disease-modifying medicines.”
About New Directors
Steven J. Kafka, Ph.D., is a managing partner at Section 32, a venture capital fund investing at the frontiers of technology and healthcare. Dr. Kafka serves as chairman of the board of Glympse Bio and a director at ImmuneID, as well as CEO and director of DA 32 Life Science Tech Acquisition Corp. Previously, Dr. Kafka served as both founding CEO and executive chairman of Thrive Earlier Detection, which was acquired by Exact Sciences in January 2021. Dr. Kafka also served as executive chairman of ArcherDX, Inc., which was acquired by Invitae in October 2020. Earlier, Dr. Kafka served as president and chief operating officer of Foundation Medicine, Inc., which was acquired by Roche in 2018, and held executive roles at multiple public and private oncology drug discovery and development companies. Dr. Kafka earned a Ph.D. in political economy and government from Harvard University and a B.A. in economics and political science from Stanford University.
Jim Trenkle, Ph.D., is currently head of investments at Sanofi Ventures and has a background in research and development, commercialization and early-stage biotech investing. Dr. Trenkle currently serves on the board of directors at Therini Bio, Glycomine and Veralox Therapeutics. Prior to joining Sanofi, Dr. Trenkle was part of the early team at Pivotal bioVenture Partners in San Francisco, where he served as Board observer for Entasis (ETTX), Inozyme (INZY), Akouos (AKUS), Fusion Pharmaceuticals (FSUN) and Karuna Therapeutics (KRTX). He began his career with Gilead Sciences where he held positions of increasing responsibility in medicinal chemistry, project and portfolio management and commercial strategy, largely focused on hepatitis C and other liver diseases. Dr. Trenkle holds a B.S. in honors chemistry from the University of Michigan, a Ph.D. in organic chemistry from Massachusetts Institute of Technology and an MBA from the University of California Berkeley, Haas School of Business.
About ROME
ROME Therapeutics is developing novel therapies for cancer and autoimmune diseases by harnessing the power of the repeatome – vast stretches of uncharted genetic material that have long been dismissed as “junk DNA.” With several drug targets identified and multiple discovery programs underway, ROME is moving rapidly to leverage this new frontier in biology. To lead this exploration, ROME has assembled a team of world-class leaders across fields including oncology, immunology, virology and machine learning. ROME is based in Cambridge, Mass. For more information, please visit www.rometx.com.
Media Contact
Lisa Qu
Ten Bridge Communications
678-662-9166
lqu@tenbridgecommunications.com
Investor Contact
Monique Allaire
THRUST Strategic Communications
monique@thrustsc.com
###
Industry leader with expertise in building successful biotechnology companies
Copenhagen, Denmark and Boston, US 13th September 2021 – Muna Therapeutics (“Muna”), pioneering the development of first-in-class small molecule therapeutics for neurodegenerative diseases, today announced the appointment of Dr. Donald Nicholson, as independent Chair of its Board of Directors.
Dr. Nicholson has deep experience across both biotech and large pharma. He was previously the CEO of Nimbus Therapeutics and was responsible for major transactions with Gilead, Celgene and Genentech. Prior to this, he spent 25 years at Merck, where he held various strategic, leadership and operational roles across diverse therapeutic areas including inflammation, immunology and neuroscience, amongst others. Dr. Nicholson began his career at Merck Frosst in Montreal as a senior research biologist and advanced through various positions of increasing responsibility, including vice president and site head of the Merck Neurosciences Research site in San Diego, vice president of immunology and infectious diseases and vice president and worldwide discovery head for the Bone, Respiratory, Immunology and Endocrine franchise based in New Jersey. He is Chair of the Board of Directors at NodThera, Disc Medicine and Jnana Therapeutics, and Board Director at Kymera Therapeutics (KYMR) and Generation Bio (GBIO).
Rita Balice-Gordon, Chief Executive Officer of Muna Therapeutics, said: “We are delighted to welcome Dr. Nicholson as independent Chair at Muna. He brings to Muna a wealth of experience in biopharma and biotech, insights into building high-functioning companies and boards, and a commitment to advancing therapies for patients with neurodegenerative diseases. We look forward to working with him and the Board as we move forward.”
Morten Graugaard Døssing, prior Chair of the Board of Directors of Muna Therapeutics said: “We are very pleased to welcome Dr. Nicholson to our Board of Directors as independent Chair. He brings to Muna his exceptional experience and leadership to support our mission to advance and strengthen our therapeutic pipeline.”
Donald Nicholson, incoming independent Chair of the Board of Directors of Muna Therapeutics said: “I am delighted to be joining Muna as Chair of the Board. Muna’s innovative approach to neurodegenerative disease therapeutics as well as its stellar leadership and scientific teams inspire great confidence in its future success.”
Dr. Nicholson has co-authored more than 150 publications in peer-reviewed scientific and medical journals and is internationally recognized for his contributions to the field of apoptotic cell death. He received his doctorate in biochemistry from the University of Western Ontario and trained as a Medical Research Council postdoctoral fellow at the University of Munich in Germany. He is the recipient of multiple academic and professional honors.
Muna Therapeutics previously raised US$ 73 million (EUR 60 million) in a Series A financing round co- led by Novo Holdings, Sofinnova Partners, Droia Ventures and LSP Dementia Fund, with Polaris Partners, Polaris Innovation Fund, Sanofi Ventures, V-Bio Ventures and VIB joining the round. The
recent raise will help Muna progress its innovative small molecule candidates to the clinic, further
develop its innovative drug discovery platform as well as expand its US presence.
ENDS
For more information please contact:
Muna Therapeutics
Rita Balice-Gordon, CEO
Email: balicegordon@munatherapeutics.com
Optimum Strategic Communications
Mary Clark, Stella Lempidaki, Zoe Bolt
Tel: +44 (0) 20 3922 1906
Email: muna@optimumcomms.com
About Muna Therapeutics
Muna Therapeutics is a private biopharmaceutical company founded in 2020 and based in Copenhagen, Denmark and Leuven, Belgium. Muna discovers and develops therapies that slow or stop devastating neurodegenerative diseases including Alzheimer’s, Frontotemporal Dementia and Parkinson’s. These disorders impact memory, movement, language, behavior and personality resulting in disability and death of millions of patients around the globe. We focus our groundbreaking science on identifying new medicines to preserve cognition and other brain functions and enhance resilience to neurodegenerative diseases. Our name reflects this focus: Muna means ‘to remember’ in Old Norse. www.munatherapeutics.com
Copenhagen, Denmark, 9th July 2021 – Muna Therapeutics (“Muna”), pioneering the development of first-in-class small molecule therapeutics for neurodegenerative diseases, today announced the successful closing of a US$ 73 million (EUR 60 million) Series A financing round. The investor syndicate was co-led by Novo Holdings, Sofinnova Partners, Droia Ventures and LSP Dementia Fund, with Polaris Partners, Polaris Innovation Fund, Sanofi Ventures, V-Bio Ventures and VIB joining the round.
Muna is the combination of two innovative European start-up companies. Muna was founded in 2020 by progranulin pathway thought leaders Professor Simon Glerup and his team from Aarhus University, Denmark, with investor Novo Holdings. Muna developed a strategic partnership on additional targets with Axxam SpA, based in Milan, Italy, which became a minority shareholder. Muna is part of Novo Seeds’ company creation efforts, where the Novo team and its entrepreneurs-in-residence help build new biotech companies based on groundbreaking new science.
Muna joined forces with K5 Therapeutics, co-founded in 2020 by Professor Bart De Strooper from VIBKU Leuven, Belgium, a pioneer in neurodegenerative diseases research, with investors Droia Ventures and VIB. The combined company - Muna Therapeutics - will be based in Copenhagen and Leuven and is led by seasoned pharma executives CEO Rita Balice-Gordon and COO Anders Hinsby, both entrepreneurs-in-residence of Novo Seeds.
Neurodegenerative diseases affect millions of individuals, with increasing global impact as the population ages. Palliative treatments are scarce, and no curative therapies are currently available. Muna is focused on addressing the staggering unmet need experienced by patients around the world with neurodegenerative disorders.
Muna’s innovative all-in-human target discovery and validation platform is based on proprietary insights into molecular pathways in different human brain cell types that underlie disease pathology and resilience to neurodegeneration, based on work from the De Strooper and Glerup laboratories. Muna has built a cutting-edge small molecule drug discovery engine that leverages high-resolution target structural approaches, AI-driven computational chemistry and cell-based screening. The financing will be used to advance Muna’s small molecule programs focused on repairing neuronal dysfunction, resolving neuroinflammation and restoring neuroprotection and resilience to disease to Investigational New Drug (IND) applications.
“We are in an era of rapid advancement in understanding how to slow or stop the relentless progression of neurodegenerative diseases like Alzheimer’s and Frontotemporal Dementia that devastate cognition and quality of life of patients as well as caregivers.” said Rita Balice-Gordon, Chief Executive Officer of Muna Therapeutics. “Our team is committed to leveraging our collective expertise to deliver impactful disease modifying small molecule therapeutics to patients as rapidly as possible.”
The Board of Directors includes: Morten Graugaard Døssing, Partner at Novo Holdings, current Chairman; Henrijette Richter, Managing Partner at Sofinnova Partners; Cillian King, Investment Manager at LSP; Luc Dochez, Partner at Droia Ventures; Isaac Ciechanover, Partner at Polaris Partners; Laia Crespo, Head of Investments at Sanofi Ventures; and Rita Balice-Gordon, CEO of Muna.
Morten Graugaard Døssing, Chairman of the Board and Partner at Novo Holdings,said: “Novo Seeds is delighted to welcome a global syndicate of first-class investors who strongly believe in Muna’s world-leading science, experienced leadership team, and its potential to develop innovative treatments for neurodegenerative diseases. We are honored to co-lead this round with Sofinnova Partners, Droia Ventures and LSP Dementia Fund – a tremendous joint effort to bring Muna to the next level.”
- Financing co-led by Novo Holdings, Sofinnova Partners, Droia Ventures and LSP Dementia Fund, with participation from Polaris Partners, Polaris Innovation Fund, Sanofi Ventures, V-Bio Ventures and VIB
- Developing small molecules to repair neuronal dysfunction and resolve neuroinflammation
- Innovative drug discovery platform leveraging insights from novel targets and pathways, resilience to neurodegeneration and all-in- human validation
Copenhagen, Denmark 30 June 2021 – Muna Therapeutics (“Muna”), pioneering the development of first-in-class small molecule therapeutics for neurodegenerative diseases, today announced the successful closing of a US$ 73 million (EUR 60 million) Series A financing round. The investor syndicate was co-led by Novo Holdings, Sofinnova Partners, LSP Dementia Fund, and Droia Ventures, with Polaris Partners, Polaris Innovation Fund, Sanofi Ventures, V-Bio Ventures and VIB joining the round.
Muna is the combination of two innovative European start-up companies. Muna was founded in 2020 by progranulin pathway thought leaders Professor Simon Glerup and his team from Aarhus University, Denmark, with investor Novo Holdings. Muna developed a strategic partnership on additional targets with Axxam SpA, based in Milan, Italy. Muna is part of Novo Seeds’ company creation efforts, where the Novo team and its entrepreneurs-in-residence help build new biotech companies based on groundbreaking new science.
Muna joined forces with K5 Therapeutics, co-founded in 2020 by Professor Bart De Strooper from VIBKU Leuven, Belgium, a pioneer in neurodegenerative diseases research, with investors Droia Ventures and VIB. The combined company - Muna Therapeutics - will be based in Copenhagen and Leuven and is led by seasoned pharma executives CEO Rita Balice-Gordon and COO Anders Hinsby, both
entrepreneurs-in-residence of Novo Seeds.
Neurodegenerative diseases affect millions of individuals, with increasing global impact as the population ages. Palliative treatments are scarce, and no curative therapies are currently available. Muna is focused on addressing the staggering unmet need experienced by patients around the world with neurodegenerative disorders.
Muna’s innovative all-in-human target discovery and validation platform is based on proprietary insights into molecular pathways in different human brain cell types that underlie disease pathology and resilience to neurodegeneration, based on work from the De Strooper and Glerup laboratories. Muna has built a cutting-edge small molecule drug discovery engine that leverages high-resolution target structural approaches, AI-driven computational chemistry and cell-based screening. The financing will be used to advance Muna’s small molecule programs focused on repairing neuronal dysfunction, resolving neuroinflammation and restoring neuroprotection and resilience to disease to Investigational New Drug (IND) applications.
“We are in an era of rapid advancement in understanding how to slow or stop the relentless progression of neurodegenerative diseases like Alzheimer’s and Frontotemporal Dementia that devastate cognition and quality of life of patients as well as caregivers.” said Rita Balice-Gordon, Chief Executive Officer of Muna Therapeutics. “Our team is committed to leveraging our collective expertise to deliver impactful disease modifying small molecule therapeutics to patients as rapidly as possible.”
The Board of Directors includes: Morten Graugaard Døssing, Partner at Novo Holdings, current Chairman; Henrijette Richter, Managing Partner at Sofinnova Partners; Cillian King, Investment Manager at LSP; Luc Dochez, Partner at Droia Ventures; Isaac Ciechanover, Partner at Polaris Partners; Laia Crespo, Head of Investments at Sanofi Ventures; and Rita Balice-Gordon, CEO of Muna.
Morten Graugaard Døssing, Chairman of the Board and Partner at Novo Holdings, said: “Novo Seeds is delighted to welcome a global syndicate of first-class investors who strongly believe in Muna’s world-leading science, experienced leadership team, and its potential to develop innovative treatments for neurodegenerative diseases. We are honored to co-lead this round with Sofinnova Partners, Droia Ventures and LSP Dementia Fund – a tremendous joint effort to bring Muna to the next level.”
For more information please contact:
Muna Therapeutics
Rita Balice-Gordon, CEO
Email: balicegordon@munatherapeutics.com
Optimum Strategic Communications
Mary Clark, Manel Mateus, Stella Lempidaki
Tel: +44 (0) 20 3922 1906
Email: muna@optimumcomms.com
About Muna Therapeutics
Muna Therapeutics is a private biopharmaceutical company founded in 2020 and based in Copenhagen, Denmark. Muna discovers and develops therapies that slow or stop devastating neurodegenerative diseases including Alzheimer’s, Frontotemporal Dementia and Parkinson’s. These disorders impact memory, movement, language, behavior and personality resulting in disability and death of millions of patients around the globe. We focus our groundbreaking science on identifying new medicines to preserve cognition and other brain functions and enhance resilience to neurodegenerative diseases. Our name reflects this focus: Muna means ‘to remember’ in Old Norse. www.munatherapeutics.com
Investor Syndicate
About Novo Holdings
Novo Holdings A/S is a private limited liability company wholly owned by the Novo Nordisk Foundation. It is the holding company of the Novo Group, comprising Novo Nordisk A/S and Novozymes A/S, and is responsible for managing the Novo Nordisk Foundation’s assets. Novo Holdings is recognized as a world-leading life science investor with a focus on creating long-term value. As a life sciences investor, Novo Holdings provides seed and venture capital to developmentstage companies and takes significant ownership positions in growth and well-established companies. Novo Holdings also manages a broad portfolio of diversified financial assets. https://www.novoholdings.dk/
About Sofinnova Partners
Sofinnova Partners is a leading European venture capital firm in life sciences, specializing in healthcare and sustainability. Based in Paris, London and Milan, the firm brings together a team of professionals from all over the world with strong scientific, medical and business expertise. Sofinnova Partners is a hands-on company builder across the entire value chain of life sciences investments, from seed to later-stage. The firm actively partners with ambitious entrepreneurs as a lead or cornerstone investor to develop transformative innovations that have the potential to positively impact our collective future. Founded in 1972, Sofinnova Partners is a deeply-established venture capital firm in Europe, with 50 years of experience backing over 500 companies and creating market leaders around the globe. Today, Sofinnova Partners has over €2 billion under management. www.sofinnovapartners.com
About Droia Ventures
Droia is a specialist biotech investor with an exclusive focus on therapeutics for oncology and genetic disease. Droia invests globally in newly founded or early-stage platform companies that apply novel science and innovative technologies to bring first-in-class drug candidates to patients. With our team of seasoned scientists, entrepreneurs and investment professionals we build great companies to save patient lives. www.droiaventures.com.
About LSP Dementia Fund
LSP is one of the largest European investment firms providing financing for life sciences and health care companies. LSP’s management has raised over €2 billion ($2.5 billion) and supported the growth of 300 companies since it started to invest in 1988, including signature deals such as argenx, Crucell and Neuravi. With offices in Amsterdam, Munich and Boston, LSP currently has the possibility to invest through five strategies, each having a distinctive investment scope and a dedicated team: LSP 6 invests in private early- to late-stage drug development and medical technology companies; LSP HEF 2 focuses on private late-stage medical technology companies; the LSP Dementia Fund invests in companies targeting neurodegenerative diseases; LSP Public targets public healthcare companies; and EBAC is the first healthcare SPAC to exclusively focus on European biotech. LSP is an active contributor to the global life sciences industry and the European life science eco-system by assuming for-profit and notfor- profit roles as initiators, founders and board members in various private and public bodies and organizations, for example being founder and board member of the Oncode Institute. www.lspvc.com.
About Polaris Partners and Polaris Innovation Fund
Polaris Partners has a 20-plus-year history of partnering with repeat entrepreneurs and world-class innovators who are improving the way we live and work. The multibillion-dollar firm manages specialty and diversified funds in healthcare and technology with investments across all stages. The Polaris Innovation Fund aims to accelerate the commercial and therapeutic potential of early-stage academic research. By partnering with passionate entrepreneurs with transformational science, the Polaris Innovation Fund fosters company creation and growth through an active investment model. Polaris has offices in Boston, San Francisco, and New York. www.polarispartners.com.
About Sanofi Ventures
Sanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are oncology, immunology, rare diseases, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. www.sanofiventures.com
About V-Bio Ventures
V-Bio Ventures is an independent venture capital firm specialized in building and financing young, innovative life science companies. V-Bio Ventures was established in 2015 and works closely with Belgium-based VIB, one of the world’s premier life science institutes. The fund invests throughout Europe in start-up and early-stage companies with high growth potential focusing on technologies that provide transformational improvements in the biopharmaceutical, pharmaceutical, diagnostics and agricultural sectors. www.v-bio.ventures
About VIB
VIB is an entrepreneurial research institute in life sciences located in Flanders, Belgium. VIB’s basic research leads to new and innovative insights into normal and pathological life processes. It unites the expertise of all its collaborators and research groups in a single institute, firmly based on its close partnership with 5 Flemish universities (Ghent University, KU Leuven, University of Antwerp, Vrije Universiteit Brussel and Hasselt University). We are supported by a solid funding program from the Flemish government. VIB has an excellent track record on translating basic scientific results into pharmaceutical, agricultural and industrial applications. Since its foundation in 1996, VIB has created 29 start-up companies, now employing over 900 people. www.vib.be
SAN CARLOS, Calif., June 29, 2021 – Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced the publication in Molecular Genetics and Metabolism of a key finding from a natural history study of phosphomannomutase 2-congenital disorder of glycosylation (PMM2- CDG), a rare pediatric orphan disease. In this study, the levels of morning cortisol and adrenocorticotropic hormone (ACTH) were measured in a cohort of patients and found to be significantly below normal, indicating PMM2-CDG patients are at risk for secondary adrenal insufficiency. These data provide key insights to improve standard of care, as early recognition of adrenal insufficiency and initiation of glucocorticoid replacement therapy and stress dosing could be lifesaving. The authors conclude that morning cortisol and ACTH levels should be evaluated at least annually for all patients with PMM2-CDG. If abnormal, a low dose ACTH stimulation test should follow to evaluate the hypothalamus, pituitary, adrenal (HPA) axis.
“Endocrinopathies in PMM2-CDG have not received the attention they deserve, especially considering that glycoproteins are involved in virtually every endocrine axis,” said Kyriakie Sarafoglou, M.D., Associate Professor in the Department of Pediatrics at University of Minnesota and lead author of the paper. “Through an international collaboration, this study was the first to identify that patients with PMM2-CDG are at risk for secondary adrenal insufficiency and to suggest that morning cortisol and ACTH monitoring should become part of standard care in these patients.”
The natural history study completed enrollment with 139 PMM2-CDG patients at 11 sites around the world (ClinicalTrials.gov Identifier: NCT03173300). Morning serum cortisol and ACTH levels were simultaneously measured in a cohort of 43 patients. In this cohort, 11 patients (25.6%) had cortisol below 5 μg/dl and low to normal ACTH levels, suggestive of secondary adrenal insufficiency. Secondary adrenal insufficiency has a prevalence of 1.5 to 2.8 in 10,000 (< 0.03%) in the general population. Since this finding, two of the 11 patients have been confirmed to have central adrenal insufficiency after low dose ACTH stimulation test results and are on hydrocortisone replacement and/or stress dosing during illness.
“We initiated this natural history study, which is the largest longitudinal study ever undertaken for this disease, to learn more about how PMM2-CDG affects patients and inform the design of our clinical trials for GLM101,” said Horacio Plotkin, M.D., FAAP, Chief Medical Officer of Glycomine. “Through thoughtfully and scientifically studying the clinical presentation and biochemistry of PMM2-CDG, our investigators have uncovered a significant risk factor that is often masked by other co-morbidities of the disease. We thank the investigators and the patients and families who participated in this study for their time and support as we advance into clinical trials GLM101. Good things happen when patients and families, investigators, and companies work together.”
“Even before we have started our first interventional clinical trial, we have uncovered a potential life-saving therapeutic intervention,” said Peter McWilliams, Ph.D., CEO of Glycomine. “While cortisol/ACTH monitoring and glucocorticoid therapy have the potential to be lifesaving for PMM2-CDG patients during stress or illness, they are still only addressing the symptoms of the disease. We look forward to initiating later this year our interventional clinical trials for GLM101 that has the potential to restore the deficiency in mannose-1-phosphate that causes PMM2-CDG.”
The research entitled, “Should patients with Phosphomannomutase 2-CDG (PMM2-CDG) be screened for adrenal insufficiency?” is available at this link: https://doi.org/10.1016/j.ymgme.2021.06.003.
SAN CARLOS, Calif., June 23, 2021 – Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced that it has closed a $68 million Series B financing. The proceeds of the financing will be used to advance Glycomine’s lead drug candidate, GLM101, through initial clinical trials in patients. GLM101 is a novel substrate replacement therapy in development to treat phosphomannomutase 2- congenital disorder of glycosylation (PMM2-CDG), a rare disease representing a critical unmet medical need.
The Series B financing includes $35 million of new funds in addition to the $33 million announced in August 2019. Today’s financing was led by new investors, Abingworth and Sanofi Ventures, and joined by RiverVest Venture Partners and Remiges Ventures. In addition, all previous Series B investors – Novo Holdings A/S, Asahi Kasei Pharma Ventures, Mission BioCapital, Sanderling Ventures, and Chiesi Ventures – participated.
Glycomine’s CEO, Peter McWilliams, Ph.D., said, “We are delighted to have expanded our syndicate with these high-quality, experienced life science investors. We have demonstrated in preclinical studies that GLM101 can restore the glycosylation pathways that are disrupted in PMM2-CDG. This additional funding will enable us to confirm in the clinic the potential of GLM101 as a therapy for all PMM2-CDG patients, regardless of genotype, and we are looking forward to executing on our clinical program with this new infusion of capital.”
PMM2-CDG is the most prevalent congenital disease of glycosylation but has no FDA-approved treatments. Glycomine’s GLM101 is a mannose-1-phosphate replacement therapy in development to treat PMM2-CDG, a disease caused by a deficiency of the enzyme phosphomannomutase 2 (PMM2). PMM2 converts mannose-6- phosphate to mannose-1-phosphate, which is an essential sugar molecule in the N-glycosylation pathway and is crucially important for proper glycoprotein structure and function. GLM101 is designed to deliver mannose-1- phosphate directly into cells and thereby bypass the PMM2 enzyme deficiency and address all disease-causing PMM2 mutations to restore pathway function. GLM101 has received Orphan Drug Designation in the U.S. and Europe and Rare Pediatric Disease Designation in the U.S.
“Glycomine’s novel and scientifically informed approach shows the potential of GLM101 to be a significant disease-modifying treatment,” added Bali Muralidhar, M.D., Ph.D., Managing Partner with Abingworth. “Glycomine has developed a proprietary approach to delivering mannose-1-phosphate intracellularly to replace the missing sugar molecule. Initial clinical studies of GLM101 will be an important proof-of-concept, and we are excited to support the company at this pivotal stage.”
“PMM2-CDG is an important unmet need with no therapeutic option,” added Jim Trenkle, Ph.D., US Head of Investments at Sanofi Ventures. “We are enthusiastic to support Glycomine’s work to advance transformative therapies for PMM2-CDG patients and their families and caregivers.”
In connection with the financing, Dr. Muralidhar, Dr. Trenkle, and Niall O’Donnell, Ph.D., Managing Director of RiverVest, have been appointed to Glycomine’s Board of Directors.
FREDERICK, Md – June 16, 2021 – Veralox Therapeutics, a biotechnology company developing first-in-class small molecule therapeutics that treat the underlying pathologies of diseases with significant unmet medical needs, today announced the closing of a $16.6 million Series A financing. The round was led by Hatteras Venture Partners with participation from Genesys Capital, Point Field Partners and Alexandria Venture Investments as well as support from previous Veralox investors including Sanofi Ventures, JDRF T1D Fund, Maryland Momentum Fund, VTC Innovation Fund and TEDCO. The Company also announced that Ben Scruggs, PhD, principal at Hatteras Venture Partners, and Jamie Stiff, MBA, managing director at Genesys Capital, will join the Veralox Therapeutics board of directors.
“In recent months, we have achieved significant momentum in our goal to build a robust pipeline of therapeutics targeting 12-lipoxygenase, which is associated with the onset and progression of a range of serious diseases and conditions,” said Jeffrey W. Strovel, PhD, chief executive officer of Veralox Therapeutics. “We are very pleased to have leading life sciences investors participating in our Series A financing, which will support our efforts to advance the development program for our lead product candidate VLX-1005 for the treatment of heparininduced thrombocytopenia (HIT).”
In January 2021, Veralox announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for VLX-1005, a first-in-class small molecule inhibitor of 12- lipoxygenase, for the treatment of HIT. The FDA also approved the company’s Investigational New Drug (IND) application for initiation of a Phase 1 clinical trial of VLX-1005 in HIT.
“Veralox’s approach has the potential to be a major advance for the treatment of heparininduced thrombocytopenia, and we believe targeting the underlying pathology holds promise to improve clinical outcomes for these patients,” said Ben Scruggs, PhD, principal at Hatteras Venture Partners. “Hatteras is delighted to support Veralox’s efforts to develop novel therapeutics for this area of significant clinical unmet need.”
Milpitas, CA, June 15 Milpitas, CA, June 15, 2021 – Bigfoot Biomedical announced today that it has acquired the intellectual property assets of Common Sensing, a Cambridge, MA-based company that develops and manufactures data-driven hardware and software solutions for people using injectable medicine, including Gocap.
“This acquisition will accelerate the innovative development work already underway at Bigfoot as we advance and expand our insulin-delivery portfolio,” said Jeffrey Brewer, CEO of Bigfoot Biomedical. “There’s tremendous momentum for smart and connected technology within the diabetes industry, and Bigfoot Biomedical is uniquely poised to deliver solutions that are simple, convenient and easy to use.”
“We’ve admired the maverick spirit of the Bigfoot Biomedical team for many years as both our companies have worked incredibly hard to develop solutions for the more than 20 million people using injectable medicine in the US and EU” said Kevin Schmid, CEO of Common Sensing. “Bigfoot has 6/16/21, 3:31 PM Page 2 of 9 proven itself as a company that can gain regulatory approval and effectively lead a manufacturing and implementation strategy. This is important as we fully expect the number of people needing to intensify their insulin therapy with multiple daily injections to grow especially with the advent of simple and accessible technologies.”
Last month, Bigfoot received FDA clearance for its Bigfoot Unity™ Diabetes Management System featuring first-of-its-kind smart pen caps for insulin pens used to treat Type 1 and Type 2 diabetes. Integrated with Abbott’s FreeStyle® Libre 2 system, the Bigfoot Unity System is the first and only solution to translate continuously monitored glucose data into on-demand insulin dose recommendations, based on HCP instructions, displayed right on the pen-cap screen for ease of use.
“For the millions of people with insulin-requiring Type 1 and Type 2 diabetes, there’s a huge need for solutions that are accessible in terms of cost and simplicity,” said Brewer. “We’re keeping our foot firmly on the gas pedal as we look to expedite the 6/16/21, 3:31 PM Page 3 of 9 incredible work being done currently by the Bigfoot team. Making smart acquisitions that are compatible with our mission and philosophy are key to our strategy of delivering valued innovations to the diabetes community.”
BOSTON and NEW YORK, May 11, 2021 /PRNewswire/ -- Health care technology company Aetion announced today a $110 million Series C fundraise, led by Warburg Pincus, a leading global growth equity rm, with additional investments from B Capital and Foresite Capital. Aetion's existing backers New Enterprise Associates (NEA) and Flare Capital Partners also joined the round. Aetion has raised a total of $212 million to date. The capital infusion supports Aetion's continued growth and industry leadership in the real-world evidence (RWE) space. In the last year alone, Aetion nearly doubled revenue and secured 100 percent of customer renewals while establishing valuable industry collaborations. Aetion became the rst RWE company to establish an FDA COVID-19 research collaboration agreement and was selected by ICER as its preferred RWE partner and platform.
"In a post-COVID era, we have an imperative to use rapid, rigorous evidence to inform health care decisions," said Aetion CEO, Carolyn Magill. "This investment reinforces Aetion's position as an RWE leader and our potential for future growth as we enable our customers to generate regulatory-grade evidence at scale."
The company will use the new funds to extend the capabilities of its Aetion Evidence Platform®, expand its European and Asian-Pacic footprint, and grow its commercial team in order to serve the growing demand from biopharma, payer, and medical device / diagnostics customers.
Just a few years after its launch, the Aetion Evidence Platform is used by the majority of the top global biopharma rms, leading payers, and regulatory and HTA agencies to inform decisions on the safety, effectiveness, and value of medical products.
Warburg Pincus is a leading investor in health care and technology, with signicant expertise in venture capital and growth investing. Since its inception, the rm has invested in excess of $12 billion and $22 billion into health care and technology companies, respectively. Current health care and health care IT investments include, Alignment Healthcare, Experity, Intelligent Medical Objects, Modernizing Medicine, Outset Medical, Qualifacts, Quantum Health, SOC Telemed, Summit Health, and WebPT.
"Aetion has shown incredible growth over the last year, scaling its platform and forming industry-leading partnerships. As the use of real-world evidence expands globally, we believe our investment will help advance Aetion's technology and further inect its growth," said T.J. Carella, Managing Director and Head of Healthcare at Warburg Pincus. "We are excited to back this excellent management team and platform to further accelerate the use of data in healthcare," added Amr Kronfol, Managing Director at Warburg Pincus.
Aetion's growth has been accompanied by recognition of its status as a premier talent destination. Last year, the company was named to Fast Company's top 10 Best Workplaces for Innovators and BuiltIn's Best Places to Work in New York City and Boston; Aetion CEO Carolyn Magill was named to Rock Health's 2021 Top 50 in Digital Health; and Co-Founder, President, and Chief Science Ofcer, Jeremy Rassen, was cited among FiercePharma's Most Inuential People in the Fight Against COVID-19. Also in 2020, Cathy Polinsky, technology leader at Shopify and former StichFix CTO, joined Aetion's board of directors.
SOUTH SAN FRANCISCO, Calif, May 10, 2021 /PRNewswire/ -- Therini Bio, Inc. announced it has completed an oversubscribed Seed Extension round of nancing. The capital will accelerate the development of Therini's lead program – a monoclonal antibody against brin – towards the clinic for patients with inammatory conditions associated with vascular damage.
Therini Bio was co-founded by Dr. Katerina Akassoglou, PhD, based upon discoveries from her laboratory at Gladstone Institutes and also UC San Francisco that a cryptic epitope on brin, a blood-clotting factor, drives toxic chronic inammation in the brain and the invention of an antibody to selectively target this brin cryptic epitope. This mechanism is believed to underlie damage in multiple neurodegenerative diseases including Alzheimer's disease and Multiple Sclerosis, in addition to peripheral inammatory conditions like colitis and kidney disease. The scientic team at Therini led by Chief Scientic Ofcer Dr. Jeff Stavenhagen, PhD, has advanced this pioneering work and developed an optimized therapeutic antibody appropriate for human use that attenuates brin-induced inammation without affecting critical clotting functions. In addition, Therini also has developed a suite of antibodies that could be utilized as imaging agents for diagnosis and clinical trial selection and has also discovered a broad set of novel human therapeutic antibody candidates that it is proling for potential use in a wide range of inammatory diseases. The Company is using the capital from this nancing for antibody manufacturing and IND enabling studies in anticipation of initiating human clinical trials in 2022.
"The upsized nancing validates the progress made by Therini's research team and reveals the signicant excitement that exists around the breadth and depth of the science behind this new biological approach to inammation. Having such prestigious corporate and institutional investors around the table provides the critical nancial and human capital needed to support Therini through this stage of development as we near human clinical trials. The round will allow us to begin developing our exciting portfolio of antibodies to treat and diagnose a wider array of clinical indications," stated Dan Burgess, President and CEO of Therini. The round was co-led by SV Health Investors' Impact Medicine Fund, MRL Ventures, and Sano Ventures who join existing investors including the Dementia Discovery Fund and Dolby Family Ventures, as well as new investor, Foundation for a Better World in support of the Company. With the closing of the nancing Dr. Christine Brennan, PhD from MRL Ventures and Dr. Jim Trenkle, PhD from Sano Ventures have joined the Therini board of directors.
LOS ANGELES & NEW YORK--(BUSINESS WIRE)--Science 37, Inc. (“Science 37”), developer of the Decentralized Clinical Trial Operating System™, and LifeSci Acquisition II Corp. (NASDAQ: LSAQ) (“LifeSci”), a blank check company targeting the biopharma, medical technology, digital health and healthcare services sectors, announced today that they have entered into a definitive business combination agreement. Upon closing of the proposed transaction, the combined company will operate as Science 37 and is expected to be listed on the NASDAQ under the ticker symbol “SNCE”. The proposed transaction values Science 37 at an initial enterprise value of approximately $1.05 billion and will provide the combined company with approximately $250 million of cash (assuming no redemptions from LifeSci’s trust account), to fuel continued growth.
“The clinical research industry is undergoing a dramatic transformation in which traditional development methods are being supplanted by technology fueled innovation,” said David Coman, Chief Executive Officer of Science 37. “Our clinical trial Operating System (OS) can enable significantly faster enrollment, retain patients at a meaningfully higher rate, and achieve higher enrollment among diverse patient populations. With this investment, we expect to advance our OS to further penetrate adjacent markets, and power the future of clinical research where we bridge between the traditional and decentralized approaches to enable a truly Agile Clinical Trial.”
Andrew McDonald, Ph.D., Chief Executive Officer of LifeSci Acquisition II Corp, said, “healthcare is increasingly transitioning to virtual and home-based environments, and we believe Science 37 is uniquely positioned as a pioneer in its approach to clinical trials. The company’s rapid growth is a testament to its truly disruptive technology and its immense market opportunity to change the way drugs are developed and go to market.”
SEATTLE--(BUSINESS WIRE)--Icosavax, Inc. today announced the close of a $100 million Series B Financing, led by RA Capital Management and joined by Janus Henderson Investors, Perceptive Advisors, Viking Global Investors, Cormorant Asset Management, Omega Funds, and Surveyor Capital (a Citadel company). Icosavax’s existing investors also participated, including Qiming Venture Partners USA, Adams Street Partners, Sanofi Ventures, and ND Capital. A previously announced funding from Open Philanthropy was included in this round. In conjunction with the financing, Peter Kolchinsky, Ph.D., founder and Managing Partner of RA Capital Management, will join the company’s Board of Directors.
The proceeds of the financing will support the development of Icosavax’s bivalent respiratory syncytial virus (RSV) and human metapneumovirus (hMPV) vaccine program through initial clinical studies, continued evaluation of its SARS-CoV-2 vaccine candidate, and further expansion of the company’s pipeline of VLP vaccine candidates focused on protecting older adults from life-threatening respiratory diseases.
“There are currently no approved vaccines against RSV and hMPV, two respiratory viruses that are disproportionately lifethreatening for the elderly. We are excited to lead this investment in Icosavax alongside a great syndicate to advance the combination RSV/hMPV candidate through development and into the clinic,” said Peter Kolchinsky, Ph.D., Managing Partner at RA Capital Management.
“We are delighted to have attracted a top-tier investor syndicate who recognize the potential of our VLP technology to create more effective and durable vaccines for at-risk populations, like the elderly, where traditional vaccines have reduced efficacy,” said Adam Simpson, Chief Executive Officer of Icosavax. “Based on preclinical data, we believe our vaccine candidates could offer significant protection against leading viral causes of pneumonia in older adults where no licensed vaccines currently exist.”
The company’s RSV vaccine candidate, IVX-121, incorporates a stabilized prefusion F antigen licensed from NIAID/NIH (DS-Cav1; Science 2019). In a Phase 1 clinical study conducted by NIAID/NIH, DS-Cav1 was shown to induce robust neutralizing antibody titers, higher than titers shown in previous studies with RSV postfusion F vaccine candidates. Extensive preclinical studies suggest that the VLP display of DS-Cav1 in IVX-121 induces higher and more durable Icosavax Closes $100 Million Series B Financing to Advance Bivalent RSV/hMPV Vaccine Candidate Into Clinical Trials Company’s novel computationally designed virus-like particle (VLP) technology has the potential to create safe, effective, and durable vaccines against life-threatening respiratory viruses in older adults neutralizing antibody titers compared to the DS-Cav1 antigen alone, and presentation of DS-Cav1 on the VLP confers improved stability. Icosavax plans to advance IVX-121 into clinical studies this year. Data from the first in human study of IVX-121 in young and older adults will inform and enable the transition to a bivalent RSV/hMPV clinical program.
For COVID-19, Icosavax is advancing a SARS-CoV-2 receptor binding domain VLP vaccine candidate, IVX-411, into initial clinical studies in 2021. Preclinical data on IVX-411 and precursor candidates in mice and non-human primates show induction of robust neutralizing antibody titers and protection from viral challenge (Cell 2020, Preprint 2021). Data from the IVX-411 clinical study will inform potential development paths in the rapidly evolving COVID vaccine landscape.
NEW YORK--(BUSINESS WIRE)--Click Therapeutics, Inc. (“Click”), a leader in Digital Therapeutics™ as prescription medical treatments, today announced three corporate governance additions to further position the organization for continued growth and advancing its mission to develop and commercialize software as treatments. Wall Street veteran Randall Stanicky has joined the company leadership as Chief Financial Officer; accomplished digital healthcare executive Lee Shapiro has joined the Board of Directors; and former biopharmaceutical executive Muzammil Mansuri, Ph.D. has expanded his role at Click through appointment as Board Chair.
“We are honored to have these industry experts on the Click team,” said David Benshoof Klein, co-founder and CEO of Click Therapeutics. “As we discover, develop and commercialize a broad pipeline of prescription digital therapeutics for the treatment of multiple medical conditions, the experience and respected tenures of Randall, Lee, and Muz solidify Click’s leadership role in digital therapeutics and in the broader digital health space.”
Joining as CFO with more than 20 years of extensive industry experience and most recently leading RBC Capital Markets’ large cap, specialty and generic pharmaceuticals team, Randall Stanicky brings the focused financial leadership necessary to expand Click’s clinical and commercial reach. Prior to RBC, Stanicky spent the majority of his Wall Street career in a similar role at Goldman Sachs. He holds a Bachelor of Commerce degree from the University of British Columbia and sits on the Board of Directors at the Children's Tumor Foundation where he serves as an officer and chair of the audit committee. "The transformative potential of digital therapeutics and the breadth of Click's platform is what drew me to the company," said Stanicky. "With clear opportunities to scale the organization and improve clinical outcomes in millions of users, the executive team and I are primed to succeed with our vision."
Lee Shapiro joins Click’s Board of Directors and will serve as chair of the audit committee. He is Managing Partner at 7wireVentures, an investment firm he co-founded over a decade ago. He recently served as Chief Financial Officer of Livongo Health until their successful $18.5B merger with Teladoc. Prior to Livongo, Mr. Shapiro was President of Allscripts from 2001 until the end of 2012. He has also served on the Board of Directors of leading health technology companies such as Medidata, ConsejoSano, Medisafe, HomeThrive and other 7wire portfolio companies. He is a member of the National Board of Directors of the American Heart Association, where he chairs the audit committee and serves on the business operations committee. He holds a J.D. from The University of Chicago Law School and a B.S. in accounting from the University of Illinois.
Dr. Muzammil Mansuri has expanded his role from Senior Strategic Advisor to Click’s Board to Chair of the Board. With over three decades of comprehensive experience in senior roles within the global biopharmaceutical industry, Dr. Mansuri’s deep knowledge of therapeutic and digital approaches to patient care is key as Click advances an industry-leading digital therapeutics pipeline. Dr. Mansuri currently serves as Partner at F-Prime Capital. Prior to joining F-Prime, Dr. Mansuri was a member of the Executive Committee at Sanofi, as Executive Vice President, Strategy, Business Development, and Click Therapeutics Expands its Leadership with Executive and Board Appointments Licensing. Before this, he was at Gilead Sciences as Senior Vice President, Research and Development Strategy and Business Development. In that capacity he led the R&D Strategy, Business Development, and M&A activities for the company. Dr. Mansuri has served as Chief Executive Officer of several biotech companies and as a General Partner at Flagship Ventures (now Flagship Pioneering). He began his career in the pharmaceutical industry as a bench medicinal chemist with Bristol Myers. He received his B.Sc. and Ph.D. in chemistry from the University College London and conducted postdoctoral research at UCLA and Columbia University.
The executive leadership and Board announcements follow Click’s strategic partnerships with Otsuka, Boehringer Ingelheim, and a variety of payers, employers, and thought leaders.
Shapiro stated, “I have had the privilege to work with and evaluate numerous organizations seeking to advance the future of digital health. Click is uniquely positioned to seize on this moment to provide a new type of digital care. I am thrilled to join the Board to help ensure the company succeeds in this opportunity, and to become an investor in the company along with my business partner Glen Tullman.”
Board Chair Mansuri added, “Clinically validated digital therapeutics are the real solutions needed to improve patients' health outcomes beyond what we have been able to achieve with pharmacotherapy alone. In a time when obtaining access to comprehensive care can be more difficult than ever, Click is positioned to change this paradigm.”
SAN MATEO, Calif.--(BUSINESS WIRE)--Evidation Health today announced the close of $153 million in Series E growth funding to rapidly expand its virtual health programs on Achievement , the largest digital health network in the United States. The round was co-led by OMERS Growth Equity and Kaiser Permanente Group Trust; existing investors, including McKesson Ventures and B Capital Group also participated. Teresa Lee, Managing Director, OMERS Growth Equity, joins Evidation’s Board of Directors.
Evidation will use this additional capital to fuel the expansion of virtual health programs on the Achievement platform, building on its trusted relationships with individuals and its experience synthesizing and analyzing person-generated health data for stakeholders across healthcare. Evidation’s new programs will provide personalized insights and tools to motivate and empower individuals to take evidence-supported actions to manage their health and conditions.
“Achievement has made it possible to rapidly understand health and therapeutic impact with speed and rigor at scale,” said Deborah Kilpatrick, PhD, Co-CEO and Executive Chair at Evidation. “The next era of Evidation will transform how individuals interact with the ecosystem of care, by providing anyone with evidence-supported guidance and actionable tools to better understand and improve their health. We are now the first company that can help digital health and biopharma companies generate evidence about their treatments, and then enable individuals to use that evidence to take actions to manage their health.”
Comprised of more than 4 million individuals, Achievement is uniquely positioned to power virtual health at scale. The privacy-forward app and platform powering Evidation’s research business, Achievement is the largest and most geographically and demographically diverse connected cohort in the United States, representing 50 states and nine out of every 10 ZIP codes nationwide. Launched as a research platform powered by individuals and their permissioned health data, Achievement has been the basis for pioneering real-world studies across diverse topics, from COVID-19 and Alzheimer’s disease to chronic pain and respiratory conditions. Among its biopharma customers, Evidation works with nine out of the 10 largest in the world, along with academic institutions, public health organizations, and medical specialty societies. To date, Evidation has conducted more than 100 real-world studies across therapeutic areas. In 2020, the company reached the milestone of more than 1 million individuals participating in research and health programs and expects to surpass 2 million later this year.
“We are excited to support Evidation as it continues to scale its individual-centric Achievement platform,” said Teresa Lee of OMERS Growth Equity. “Evidation has made rapid, virtual research at scale possible and the company’s innovative platform can be leveraged to guide patients on their health journeys. Evidation is positioned to be a key enabler of the feedback loop that translates into timely, appropriate care and health insights.”
Harnessing its experience in research and connection to individuals through person-generated health data, last year Evidation launched its first virtual health initiatives, including Achievement for Heart Health—a first-of-its-kind health program in partnership with the American College of Cardiology to help individuals monitor and improve their cardiovascular health outside clinic-based settings, with an initial focus on heart failure. The company also collaborated with Apple and the government of Singapore on LumiHealth, a personalized program that encourages healthy activity and behaviors using Apple Watch. Using a double opt-in, these privacy-safe health programs seamlessly connect research and care, accelerating the discovery and dissemination of evidence-supported practices that improve health outcomes.
Individual privacy and per-use consents to share data will remain core tenets as the company transforms the way everyday health is understood and expands the programs available through Achievement. To join Achievement and learn more about how you can contribute to innovative health research and participate in personalized health programs, visit myachievement.com.
UTRECHT, The Netherlands and PHILADELPHIA, March 24, 2021 (GLOBE NEWSWIRE) -- LAVA Therapeutics B.V. (Nasdaq: LVTX), a biotechnology company focused on applying its expertise in bispecific gamma-delta T cell engagers to transform cancer therapy, today announced the pricing of its initial public offering of 6,700,000 common shares at a public offering price of $15.00 per share. In addition, LAVA has granted the underwriters a 30-day option to purchase up to an additional 1,005,000 common shares at the initial public offering price, less underwriting discounts and commissions. All of the shares are being offered by LAVA. The shares are expected to begin trading on the Nasdaq Global Select Market on March 25, 2021 under the ticker symbol “LVTX.”
Gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by LAVA, are expected to be $100.5 million. The offering is expected to close on March 29, 2021, subject to the satisfaction of customary closing conditions.
J.P. Morgan, Jefferies and SVB Leerink are acting as joint book-running managers for the offering. Kempen & Co is acting as lead manager for the offering.
The registration statement relating to these securities became effective on March 24, 2021. The offering will be made only by means of a prospectus, copies of which may be obtained from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at prospectus_eqfi@jpmchase.com; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 821-7388 or by email at prospectus_department@Jefferies.com; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6105, or by email at syndicate@svbleerink.com.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.
Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding LAVA’s expectations regarding the commencement of trading of its shares on the Nasdaq Global Select Market and the completion and timing of the closing of offering. Forward-looking statements are based on LAVA’s current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to the satisfaction of customary closing conditions and the completion of the offering. These and other risks and uncertainties are described more fully in the section titled “Risk Factors” in the final prospectus related to the offering to be filed with the Securities and Exchange Commission. Forward-looking statements contained in this press release are made as of this date, and LAVA undertakes no duty to update such information except as required under applicable law.
Company Contact: IR@lavatherapeutics.com
Cambridge, MA, 17, March 2021
Amathus Therapeutics announced today a strategic collaboration with Merck, known as MSD outside the United States and Canada, to develop novel small molecule therapeutic candidates for neurodegenerative diseases.
Edward Holson, President and CSO of Amathus added that Amathus will focus on exploiting genetically defined pathways and core pathologies in multiple disease indications, “the Amathus team has demonstrated activation of molecular chaperones is possible and the potential to address core mitochondrial dysfunction with specificity holds tremendous therapeutic potential. The Merck neuroscience team is an ideal partner to translate this novel approach into potential first-in-class, disease modifying treatments for patients with neurodegenerative diseases.”
Under the terms of the agreement, Amathus will be responsible for identifying and progressing novel chaperone activators through preclinical discovery. Merck has the option to acquire Amathus Therapeutics and its pipeline of mitochondrial targeted candidates for the treatment of neurodegenerative disorders and renal diseases. Amathus will receive an upfront payment from Merck and, upon Merck’s exercise of its option, will be eligible for milestone payments associated with the successful development of candidates in excess of $500 million per program. Upon exercise of its option, Merck will be solely responsible for clinical development and commercialization.
“We are excited to be working with the Amathus team to investigate the company’s novel mitochondrial targeted candidates for the treatment of neurodegenerative conditions,” said Fiona Marshall, Senior Vice President, Head of Discovery Sciences and Translational Medicine, Merck Research Laboratories. “External collaborations that supplement and complement our internal pipeline programs remain a cornerstone of Merck’s discovery strategy.”
About Amathus Therapeutics
Amathus Therapeutics is a biopharmaceutical company and leader in the field of small molecule modulators of organelle specific chaperones across a variety of genetically defined diseases including Parkinson’s Disease (PINK1) and Polycystic Kidney Disease (ADPKD). The company’s proprietary platform enables highly specific modulation of mitochondrial and endoplasmic reticulum chaperones to exploit cellular stress response pathways and rescue key cellular functions associated with disease. Amathus was founded by Sanofi Ventures based on licensed discoveries from the Icahn School of Medicine at Mount Sinai, Mount Sinai Health System faculty and the laboratory of Professor Yiannis A. Ioannou. Amathus Therapeutics is funded by Sanofi Ventures and the Dementia Discovery Fund. Mount Sinai and Mount Sinai faculty, including Yiannis A. Ioannou, have a financial interest in Amathus.
Inquires: www.amathustx.com
About DDF
The Dementia Discovery Fund (DDF) is a specialist venture capital fund that invests in projects and creates biotech companies to deliver high impact therapeutics for dementia - including Alzheimer’s Disease (AD), Parkinson’s Disease Dementia (PDD), Frontotemporal Dementia (FTD), Amyotrophic Lateral Sclerosis (ALS), and Huntington’s Disease (HD). By making meaningful and sustained investments which are actively managed, the DDF enables the development of therapeutics addressing one of the world’s largest unmet medical needs and the generation of significant returns for its investors. Established in 2015, it has raised funds from an influential group including leading pharmaceutical companies (Biogen, Eli Lilly and Co., GSK, Johnson & Johnson, Otsuka (Astex), Pfizer and Takeda), along with AARP, Aegon, Bill Gates, British Patient Capital, NFL Players Association, Quest Diagnostics, UnitedHealth Group, the UK Government’s Department of Health and Social Care, and the charity Alzheimer’s Research UK. The Fund is managed by SV Health Investors. Learn more at www.TheDDFund.com
About Sanofi Ventures
Sanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are oncology, immunology, rare diseases, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. For more information, visit www.sanofiventures.com.
+++
Media Contact: Tandee Newman
Amathus Therapeutics, Inc.
617-953-4844
contact@amathustx.com
Veteran biopharma CFO brings over 20 years of experience leading finance functions at publicly traded pharmaceutical companies
Utrecht, The Netherlands and Philadelphia, USA – March 15, 2021 – LAVA Therapeutics B.V. (“LAVA”, or the “Company”), a biotechnology company focused on applying its expertise in bispecific gamma-delta T cell engagers to transform cancer therapy, today announced that it is expanding its management team with the appointment of Edward F. Smith as its chief financial officer.
Mr. Smith has served on management teams of publicly traded life science companies for the past 20 years, raising approximately $500 million, building finance organizations and supporting operations from early development stage into commercialization.
“I am excited to welcome Ed to the team as LAVA prepares to become a clinical stage company,” said Stephen Hurly, chief executive officer of LAVA. “We have built a highly experienced team committed to a culture of tenacious hard work, creativity, and collaboration to drive our platform of bispecific gamma-delta T cell engagers forward to serve cancer patients. Ed fits in perfectly and brings significant experience building finance and accounting functions to our team.”
“While first generation T-cell engagers had great promise in many areas, that promise has yet to be fully realized. I believe LAVA’s approach leveraging the unique attributes of gamma delta T-cells holds the potential to move the field forward and potentially transform the standard of care across many tumor types,” Mr. Smith said.
Prior to LAVA, Mr. Smith was CFO of Marinus Pharmaceuticals and PolyMedix, Inc., and prior to that was executive director of finance at InKine Pharmaceutical Company, Inc., where he assisted with the acquisition of that company by Salix Pharmaceuticals, Inc. Earlier in his career, he held various positions of increasing responsibility in public accounting, most recently in the audit practice at Deloitte & Touche, LLP. Mr. Smith is currently a member of the board of directors at Benitec Biopharma, Inc., a development-stage biotechnology company focused on the advancement of novel genetic medicines. Mr. Smith holds a B.S. in business administration from the University of Hartford and was licensed as a Certified Public Accountant in Pennsylvania.
BOSTON, Feb. 25, 2021 /PRNewswire/ -- Medisafe, a leading digital therapeutics company creating digital drug companions, today announced that it has raised $30 million in Series C funding with investments led by Sano Ventures and ALIVE Israel HealthTech Fund, joined by Leumi Partners, Menorah Mivtachim and Consensus Business Group, previous investors Pitango Ventures, 7wireVentures, Merck Ventures, Octopus Ventures, lool HealthTech, Triventures and Ourcrowd. With this new investment Medisafe will further expand its end-to-end solutions supporting patients managing medications and accelerate revenues growth. Cris De Luca, Global Head, Digital Investments at Sano Ventures and David Klein, Co-Founder, Managing Partner ALIVE Israel HealthTech Fund will join Medisafe's board of directors.
"The COVID-19 pandemic has proven to us that supporting patients digitally through virtual care and leveraging data-driven insights, is going to be foundational to future models of healthcare and improving outcomes," said Cris De Luca, Global Head, Digital Investments at Sano Ventures.
"We've been following Medisafe and witnessed its massive growth as the healthcare industry is rapidly deploying digital solutions," said David Klein, Co-Founder, Managing Partner ALIVE Israel HealthTech Fund. "We're excited to partner with the company, its investors and partners at this inection point to drive together to new heights."
Founded in 2012, Medisafe is the largest medication management platform with over 7M registered users globally. Medisafe's growth is rapidly accelerating as the healthcare industry turns to digital health solutions to support patients. Partnering with top global pharma companies and expanding opportunities across the health ecosystem, Medisafe deploys digital drug companions to holistically support patients throughout their unique therapy journey. Medisafe's advanced technology transforms patient behaviors powered by JITI™ (Justin-Time-Interventions), AI data-driven personalized engine providing support for patients.
Over the past eight years, Medisafe has amassed a database of over four billion dosage behaviors informing JITI to maximize engagement and driving outcomes. In 2020, Medisafe expanded its platform rst launching Medisafe Care Connector which links the all-critical human element into patient support programs digitally connecting patients with clinicians. Last September, Medisafe introduced Maestro, a proprietary no-code technology to build, deploy and optimize personalized journeys. Medisafe Maestro provides a scalable solution to activate patient journeys, orchestrate patient support interventions and increase overall speed to market.
"This investment allows Medisafe to expand holistic treatment support for patients to impact behavior change and ultimately outcomes. Medisafe is continuously advancing its technology to meet the dynamic needs of patients managing complex therapies," said Omri Shor, CEO and Co-Founder of Medisafe. "The future model of patient support is not purely digital but adaptable to empower human connections. In fact, we are seeing impressive results connecting clinicians and care givers into the support solutions with adoption rates 4x that of pure digital and increasing nurse-to-patient connectivity by 1.8x. We are excited to build out further capabilities to meet the evolving needs of the industry."
PRINCETON, N.J. & NEW YORK--(BUSINESS WIRE)--Otsuka Pharmaceutical Development & Commercialization, Inc. (Otsuka) and Click Therapeutics, Inc. announce the initiation of the Mirai study, a landmark fully remote clinical trial to investigate the effectiveness of digital therapeutics in reducing depressive symptoms in adults diagnosed with major depressive disorder (MDD) who are on antidepressant monotherapy.
The pivotal, randomized, controlled trial will enroll up to 540 patients nationwide. Trial participation will be 10 weeks and efficacy will be evaluated as a change from baseline in the Montgomery–Åsberg Depression Rating Scale (MADRS) total score.
“This landmark clinical trial demonstrates Otsuka’s unwavering commitment to the evolution of clinically validated, FDAcleared digital health solutions that support patients living with mental illnesses, like major depressive disorder,” said Kabir Nath, president and CEO, Otsuka North America Pharmaceutical Business Division, Otsuka America, Inc. “The COVID-19 pandemic has clearly demonstrated the need for digital treatments and fully remote e-clinical trials that go beyond the pill to empower patients, enhance connectivity between patients and their healthcare team, and ensure more diverse populations can participate in new clinical trials.”
According to the World Health Organization, depression is a leading cause of disability worldwide, and is a major contributor to the overall global burden of disease. Click and Otsuka are committed to bridging the gaps between the global deficiency of mental health treatment access and the millions of patients in need of safe, effective, convenient and accessible care.
“While awareness of mental illness has grown steadily over the last decade, clinically validated therapeutic options available to patients and providers have remained essentially the same,” said David Benshoof Klein, CEO of Click Therapeutics. “Now more than ever, there is a need for a scalable digital solution that can dramatically expand access to mental health treatment without sacrificing the rigors of clinical validation or a patient-centered focus on engagement and user experience.”
Otsuka and Click Therapeutics Initiate First-of-its-Kind Fully Remote Clinical Trial Using Digital Therapeutics as Adjunctive Therapy in Adults With Major Depressive Disorder Primary objective of the Mirai study is to evaluate the effectiveness of digital therapeutics to reduce depressive symptoms in adults diagnosed with major depressive disorder (MDD) who are on antidepressant monotherapy Trial will run fully remotely utilizing the Verily Project Baseline platform, a first-of-its-kind trial design for MDD Otsuka is committed to exploring digital therapeutics in patients with serious mental illnesses like MDD, which affects more than 17 million adults in the US1 2 / Otsuka and Click will collaborate with Verily, a subsidiary of Alphabet, to execute the trial as a fully remote trial. The collaboration with Verily provides tools and technology to engage patients and clinicians, in order to increase the pace of studies and collect higher quality, more comprehensive data in a more naturalistic setting. The collaboration also enables the trial to proceed efficiently and safely in the face of the unique market challenges presented by COVID-19.
Utrecht, The Netherlands and Philadelphia, USA – February 22, 2021 – LAVA Therapeutics B.V., an early clinical-stage biotechnology company focused on applying its expertise in bispecific gamma-delta T cell engagers to transform cancer therapy, today announced the appointment of Dr. Kapil Dhingra as chairman of its Board of Directors. Dr. Dhingra has more than 30 years of experience in oncology clinical research and drug development within the biotechnology and pharmaceutical industries, as a Board member at several successful oncology biotechnology companies, and as a clinician at academic research centers.
“We are delighted to have Dr. Dhingra join our Board as chairman at this moment in our company’s journey,” said Stephen Hurly, President and Chief Executive Officer. “In the coming year we expect two LAVA programs to enter Phase 1/2a studies in solid and hematologic malignancies. Dr. Dhingra’s deep expertise in cancer drug development and impressive track record of building successful oncology biotechnology companies will have an immediate and beneficial impact on our strategy and mission to bring our first-in-class cancer drugs to patients.”
Mr. Hurly added, “We look forward to benefiting from Dr. Dhingra’s expertise as we expand our preclinical pipeline. Our platform’s modularity enables rapid discovery and development of novel candidates, and Dr. Dhingra’s experience in translational medicine will provide important insights as we select high-value, innovative targets. I also want to thank Erik van den Berg for serving as chairman of LAVA’s Board of Directors since our founding. During his tenure, LAVA raised over $100M in capital from leading life science VCs which provides a solid foundation for the company’s continued growth.”
“I am excited to welcome Dr. Dhingra to the LAVA Board of Directors. His experience will be extremely valuable to LAVA as it transitions to become a clinical-stage company this year,” commented Erik van den Berg.
“LAVA’s novel T cell engager platform, which harnesses the unique attributes of gamma-delta T cells, has the potential to change the standard of care for many high unmet need cancer patient populations,” said Dr. Dhingra. “I look forward to working with the LAVA Board and management team. I also want to thank Erik van den Berg for his leadership of LAVA’s Board. He has been instrumental in growing LAVA into a company with two drug candidates poised to enter the clinic within a year.”
Dr. Dhingra is a medical oncologist and a physician-scientist with a proven track record in academic research, patient care, and drug development. He served as Vice President, Head of the Oncology Disease Biology Leadership Team and Head of Oncology Clinical Development at Hoffmann-La Roche (“Roche”), during which he led numerous drug approvals, including Herceptin®, Tarceva®, and Avastin®. Prior to joining Roche, he worked in the oncology clinical development group at Eli Lilly and Company. Dr. Dhingra has served as a faculty member at The University of Texas M.D. Anderson Cancer Center, Indiana University School of Medicine, and Memorial Sloan Kettering Cancer Center. Dr. Dhingra is currently a member of the Boards of Directors of Black Diamond Therapeutics, Inc., Replimune, Inc., Five Prime Therapeutics, Inc., Autolus Therapeutics plc, and Median Technologies, and he has previously served on the boards of several successful biotech companies, including Biovex, Micromet, Algeta, YM Biosciences, Epitherapeutics, Advanced Accelerator Applications, and Exosome Diagnostics. He is a member of the NCI Experimental Therapeutics Panel. Dr. Dhingra founded KAPital Consulting, LLC in 2008, a company dedicated to helping biotechnology, pharmaceutical, and diagnostic companies realize the www.LavaTherapeutics.com clinical and commercial advances in oncology. Dr. Dhingra obtained his M.B.B.S. degree from the All India Institute of Medical Sciences in New Delhi, India. He completed his residency in internal medicine at Lincoln Medical and Mental Health Center, New York Medical College and completed his fellowship in hematology and oncology at Emory University School of Medicine.
Partnership to create new treatment delivery options for people facing serious diseases
BOSTON, MA—February 10, 2021—i2O Therapeutics, developers of a platform for oral delivery of traditionally injectable biological drugs, announced today a research collaboration with Sanofi to investigate the oral delivery of Sanofi's Nanobody®-based medicines, which are currently administered through intravenous or subcutaneous injections.
Nanobodies - proprietary therapeutic proteins based on camelid derived immunoglobulin single variable domains - have potential uses in the treatment of a range of serious and life-threatening diseases and are being developed in many therapeutic areas including inflammation, hematology, immuno-oncology, oncology and rare diseases. The research collaboration between i2O Therapeutics and Sanofi will explore a new oral route of administering nanobodies.
“Our mission at i2O Therapeutics is to develop safe and effective oral formulations of therapies traditionally limited to injections and we are excited to partner with Sanofi to advance this mission,” said Ravi Srinivasan, co-founder and director of i2O Therapeutics.
“i2O’s ionic liquid platform opens new opportunities to orally deliver biologics, and nanobodies represent an exciting application of this platform,” said Samir Mitragotri, co-founder of i2O Therapeutics.
i2O Therapeutics announced seed funding in April 2020, which was led by Sanofi Ventures, the corporate venture capital arm of Sanofi, and JDRF T1D Fund. The company also announced a strategic investment from Colorcon Ventures, the corporate venture capital fund of Colorcon, Inc. in December 2020.
About i2O Therapeutics
i2O Therapeutics is a biotechnology company developing safe and effective oral formulations of therapies traditionally limited to injections. Using an innovative ionic liquid technology, this platform leverages the benefits of protecting the drug cargo while also transiently enhancing permeation across the epithelial lining when administered orally. i2O is focused on creating the next generation of oral peptide and protein-based therapies. Visit us at www.i2OBio.com.
About Sanofi
Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions. With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Contact
Lauren Arnold
MacDougall
larnold@macbiocom.com
781-235-3060
Strengthens senior leadership team with appointments of Donald Johns, M.D.,
as Chief Medical Officer and Katina Dorton, J.D., MBA, as Chief Financial Officer
CAMBRIDGE, UK, BOSTON and SEATTLE – December 15, 2020 – NodThera, a biotechnology company developing a new class of medicines that inhibit the NLRP3 inflammasome to treat chronic inflammation, today announced the expansion of its senior leadership team with the appointments of Donald Johns, M.D., as Chief Medical Officer and Katina Dorton, J.D., MBA, as Chief Financial Officer.
Dr. Johns is an accomplished drug development leader and board-certified clinical neurologist who previously served as Chief Medical Officer and Executive Vice President of Medical and Scientific Affairs at Syntimmune, prior to the company’s acquisition by Alexion Pharmaceuticals. Ms. Dorton is a recognized and internationally experienced financial executive, corporate director and public company CFO. Ms. Dorton’s industry expertise includes roles in healthcare, life sciences and investment banking, including experiences at Repare Therapeutics and AVROBIO, Inc.
NodThera is advancing a portfolio of potent and selective inhibitors of the NLRP3 inflammasome that reduce both IL-1β and IL-18, pro-inflammatory cytokines which are known to play a role in chronic inflammation underlying a wide range of diseases. The pipeline includes brain penetrant NLRP3 inhibitors for central nervous system (CNS) indications.
“We welcome Don and Katina to the team as we continue to advance our portfolio of differentiated NLRP3 inhibitors to capitalize on the opportunity to exploit the well-understood, but still untapped therapeutic potential of the NLRP3 inflammasome across a broad spectrum of diseases,” said Adam Keeney, Ph.D., President & Chief Executive Officer of NodThera. “Don is an accomplished clinician with an impressive track record who brings a wealth of innovative clinical development experience. Katina brings capital markets experience and has built financial, legal and operational functions to support companies through aggressive growth, including IPO preparation. Their combined contributions will add significant value as we continue to advance NodThera’s portfolio through clinical development and additional financing rounds.”
“The NLRP3 inflammasome is one of the most exciting emerging areas of therapeutic science. NodThera’s best-in-class molecules have great potential to address the unmet medical need in patients with a wide range of inflammatory disorders,” said Dr. Johns. Ms. Dorton adds, “By leveraging innate immunity, NodThera’s platform has the potential to blaze a new path forward in a wide range of disorders, and I am excited to join the rest of the experienced leadership team in this effort.”
Dr. Johns brings more than 25 years of experience in the development of novel treatments for serious diseases, including autoimmune and CNS disorders. Prior to his role at Syntimmune, Dr. Johns served in key leadership positions at Biogen and the Novartis Institutes for Biomedical Research (NIBR). He has contributed to numerous investigational new drug applications (INDs), first-in-human studies and conclusive proof-of-concept studies in a broad spectrum of CNS and autoimmune diseases, as well as four successful new drug applications (NDAs). Prior to joining the pharmaceutical industry, he was a neurologist clinician-scientist at Johns Hopkins and Harvard Medical School. Dr. Johns earned his B.A. from Vanderbilt University and his M.D. from the Yale University School of Medicine.
Ms. Dorton currently serves on the board of directors for Fulcrum Therapeutics (Nasdaq: FULC), Pandion Therapeutics, Inc. (Nasdaq: PAND) and US Ecology (Nasdaq: ECOL). She most recently served as Chief Financial Officer of Repare Therapeutics, a synthetic lethality and DNA repair-focused oncology company. Prior to Repare, Ms. Dorton served as Chief Financial Officer of AVROBIO, a lentiviral gene therapy company. Earlier in her career, she served as a managing director in investment banking for Morgan Stanley and Needham & Company and as an associate attorney at Sullivan & Cromwell. Ms. Dorton received her J.D. from the University of Virginia School of Law, her MBA from George Washington University and her B.A. from Duke University.
About NodThera
NodThera is a biotechnology company developing a new class of potent and selective NLRP3 inflammasome inhibitors for the treatment of diseases driven by chronic inflammation. Led by an experienced management team, NodThera is leveraging new insights into inflammasome biology and chemistry to build a platform of highly differentiated small molecule NLRP3 inhibitors. The company was founded by Epidarex Capital and further financed by 5AM Ventures, Cowen Healthcare Investments, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera was founded in 2016 and maintains offices in Cambridge, UK, Seattle, WA and Boston, MA. For more information please visit www.nodthera.com.
Media Contact
Gina Nugent
Ten Bridge Communications
(617) 460-3579
gina@tenbridgecommunications.com
- On the back of highly promising Phase Ib data, MinervaX has raised financing from leading investors to accelerate development of its novel vaccine through the end of Phase II trials and preparations for Phase III pivotal trials
- Sanofi Ventures, Wellington Partners, Adjuvant Capital, and Industrifonden join existing investors Novo Holdings REPAIR Impact Fund, Sunstone Life Science Ventures and LF Investment
- Group B Streptococcus (GBS) is one of the leading causes of stillbirth and infant mortality representing a significant unmet need globally, including in the US and Europe; nearly one in five women globally are colonized by GBS
Copenhagen, Denmark, 15 December 2020 – MinervaX, a privately held Danish biotechnology company developing a novel vaccine against Group B Streptococcus (GBS), announced today that it has raised an upsized EUR 47.4 million Series B financing. The round included new investors Sanofi Ventures, Wellington Partners, Adjuvant Capital, and Industrifonden, along with existing investors Novo Holdings REPAIR Impact Fund, Sunstone Life Science Ventures, and LF Investment. Proceeds will advance the clinical development of MinervaX’s novel GBS vaccine through Phase II clinical trials, as well as manufacturing and regulatory preparation for Phase III.
Concurrent with the financing, Christopher Gagliardi from Sanofi Ventures, Karl Nägler from Wellington Partners, Kabeer Aziz from Adjuvant Capital and Bita Sehat from Industrifonden will join MinervaX’s board of directors.
GBS is responsible for nearly half of all life-threatening infections in newborns. MinervaX’s protein-only GBS vaccine targets pregnant women for the prevention of adverse pregnancy outcomes and life-threatening neonatal infections associated with GBS. Globally, 15-25% of women are colonized with GBS, and they run the risk of transmitting the bacteria to their child in utero, during birth and / or during their first months of life. GBS colonization may lead to late-term abortions, premature delivery or stillbirth; and in newborn children may result in sepsis, pneumonia or meningitis, all of which carry a significant risk of severe morbidity, long-term disability or death.
Currently, the only preventative strategy available involves the use of intravenously delivered prophylactic antibiotics, which does not comprehensively prevent GBS infection in utero or protect against late-onset infection in newborns. As this approach is expensive and logistically challenging, it fails to cover all, including the most severe cases in the US and Europe, nor is it available in resource-limited settings.
Commenting on the financing, Per Fischer, CEO of MinervaX, said: “Prevention of GBS infections in pregnant women and newborns represents a large unmet medical need. The current preventive strategy is insufficient and involves excessive use of prophylactic antibiotics, which has resulted in the emergence of wide-spread antibiotic resistance.
“We are pleased to have received funding from such a strong investment syndicate. It is a significant endorsement of the potential of our vaccine. We look forward to advancing our novel vaccine candidate through Phase II clinical trials to develop a new standard of care in preventing GBS infections.”
Commenting on the investment, Christopher Gagliardi, Director of Investments at Sanofi Ventures, said: “Sanofi Ventures is tremendously excited by the first and best in class potential of MinervaX’s GBS vaccine. We are thrilled to invest alongside a top-tier investor syndicate while supporting Sanofi’s strategic goals and commitment to early stage companies advancing global public health.”
Karl Nägler, Managing Partner at Wellington Partners said: “We are proud and excited to back MinervaX’s GBS program that will address an unmet high medical need and represents a blockbuster commercial opportunity. Beyond prevention of GBS infections in newborns, we are eager to explore important further indications for this much needed vaccine.”
Emmanuelle Coutanceau, Partner at Novo Seeds and Board Member at MinervaX, added: “MinervaX is developing an important vaccine against a potentially fatal pathogen and, in doing so, is furthering the battle against antimicrobial resistance. This is a landmark for the Novo Holdings REPAIR Impact Fund with the first company in the fund moving to Phase II. We are also delighted to help bring together such a strong syndicate in a company where Novo was one of the first investors.”
MinervaX has completed Phase I studies across 300 healthy female subjects, generating compelling data to support advancing its novel vaccine candidate to Phase II trials. Studies to date have demonstrated a favourable safety profile, while generating high levels of long-lasting antibodies, which are capable of mobilizing the immune system against GBS bacteria and preventing invasion of epithelial and endothelial cell barriers.
The development of MinervaX’s novel GBS vaccine candidate is also endorsed by Group B Strep Support and Group B Strep International, and GBS has been prioritised by a number of public health organisations. Both increased uptake of immunisation among pregnant women and greater awareness of the implications of GBS suggest that a safe and effective vaccine targeting GBS would be well suited to address this unmet need.
Enquiries
For more information on MinervaX, please contact:
Optimum Strategic Communications
Supriya Mathur, Charlotte Hepburne-Scott
+44 (0) 203 922 0891, +44 (0) 787 687 2223
minervax@optimumcomms.com
About MinervaX
MinervaX is a Danish biotechnology company, established in 2010 in order to develop a prophylactic vaccine against Group B Streptococcus (GBS), based on research from Lund University. MinervaX is developing a GBS vaccine for maternal immunization, likely to have superior characteristics compared with other GBS vaccine candidates in development. The latter are based on traditional capsular polysaccharide (CPS) conjugate technology. By contrast, MinervaX’s vaccine is a protein-only vaccine based on fusions of highly immunogenic and protective protein domains from selected surface proteins of GBS (the Alpha-like protein family). Given the broad distribution of proteins contained in the vaccine on GBS strains globally, it is expected that MinervaX’s vaccine will confer protection against virtually 100% of all GBS isolates. www.minervax.com
About Group B Streptococcus (GBS)
GBS is responsible for nearly 50% of all life-threatening infections in newborns. At any given time, some 15-25% of women are spontaneously colonized with GBS, and they run the risk of transmitting the bacteria to their child in the womb, during birth and/or during the first months of life. GBS colonization may lead to late abortions, premature delivery or stillbirth and, in the newborn child, may result in sepsis, pneumonia or meningitis, all of which carry a significant risk of severe morbidity, long-term disability or death.
About Sanofi Ventures
Sanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are vaccines, oncology, immunology, rare diseases, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. For more information, visit www.sanofiventures.com.
About Novo Holdings A/S
Novo Holdings is recognized as a leading international life science investor, with a focus on creating long-term value. As a life science investor, Novo Holdings provides seed and venture capital to development-stage companies and takes significant ownership positions in growth and well-established companies. Novo Holdings also manages a broad portfolio of diversified financial assets. Further information: http://www.novoholdings.dk
About REPAIR Impact Fund
The Fund invests in start-ups, early-stage companies and corporate spin-outs around the world. It gives priority to first-in-class therapies, covering small molecules, biologics and new modalities, from the early stage of drug development (lead optimization) to later stages of clinical development (into Phase 2). It can invest as the sole investor or in a syndicate, with investments ranging from EUR 1 million to EUR 12 million.
The projects are selected through an investment process with support from a highly qualified Scientific Selection Board, comprising ten world-class experts. For more information about members of the Scientific Selection Board, see www.repair-impact-fund.com/people.
The Fund focuses on priority pathogens as defined by the World Health Organization and the United States Centers for Disease Control and Prevention, a catalogue of 18 families of bacterial and fungal pathogens that pose the greatest threat to human health. For more details about the investment process, see www.repair-impact-fund.com/investment-process.
REPAIR is an acronym: Replenishing and Enabling the Pipeline for Anti-Infective Resistance
About Wellington Partners
Wellington Partners is a leading European venture capital firm investing in early- and growth-stage life science companies. Wellington Partners is focused on investing in the most promising life science companies in the fields of biotechnology, therapeutics, medical technology, diagnostics and digital health. With funds totaling more than €1 billion, thereof €430 million committed to Life Sciences, Wellington Partners has been actively supporting world class private companies translating true innovation into successful businesses with exceptional growth. To date, Wellington Partners has invested in 46 innovative life science companies, including Actelion (acquired by J&J), Definiens (acquired by AZ), Invendo (acquired by Ambu), Rigontec (acquired by MSD), Symetis (acquired by Boston Scientific), and Themis (acquired by MSD). www.wellington-partners.com
About Adjuvant Capital
Adjuvant is a New York- and San Francisco-based life sciences investment fund built to accelerate the development of new technologies for the world's most pressing public health challenges. Backed by prominent healthcare investors such as Novartis, Merck, the International Finance Corporation, and the Bill & Melinda Gates Foundation, Adjuvant draws upon its global network of scientists, public health experts, biopharmaceutical industry veterans, and development finance professionals to identify new investment opportunities. Adjuvant invests in companies developing promising new vaccines, therapeutics, and diagnostics for historically overlooked indications targeting high-burden infectious diseases, maternal and child health, and antimicrobial resistance, with a commitment to make these interventions accessible to those who need them most in low- and middle-income countries. For more information, visit www.adjuvantcapital.com
About Industrifonden
Industrifonden is a Nordic venture capital investor based in Stockholm that invests in early-stage growth companies. Our areas of expertise include Life Sciences, Deep Tech and Transformative Tech. In the life science space, our focus is on biotech, heathtech and medtech, and our life-science portfolio includes companies like Oncopeptides, Calliditas and Bonesuppport. www.industrifonden.com
About Sunstone Life Science Ventures
Sunstone Life Science Ventures is an independent European venture capital investment firm founded in 2007 by an international team of industry experts with combined entrepreneurial, operational and financial experience. Sunstone Life Science Ventures focuses on developing and expanding early-stage Life Science companies with strong potential to achieve global success in their markets. Since the inception, Sunstone Life Science Ventures has invested in more than 50 companies in the areas of pharmaceuticals, medical technologies and diagnostics, and has completed more than 20 successful IPOs and large M&A transactions. Managing total funds of approx. €500 million, Sunstone Life Science Ventures is one of the largest Nordic venture capital investors. https://sunstone.eu/
LF Investment
LF Investment is an investment company fully owned by The Lauritzen Foundation. www.lauritzenfonden.com
Preclinical data published in Cell show the company’s COVID-19 vaccine candidate induces high neutralizing antibody titers after a single administration
SEATTLE, October 30, 2020 – Icosavax, Inc. today announced the launch of the company’s COVID-19 vaccine program with preclinical data on the company’s VLP vaccine candidate, IVX-411, which comprises a virus-like particle (VLP) displaying the SARS-CoV-2 receptor-binding domain (RBD) in a highly immunogenic array. Icosavax also announced that the Bill & Melinda Gates Foundation has provided a $10 million grant to support the company’s COVID-19 vaccine program through the first in human Phase 1 clinical trial in young and older adults, expected to initiate in mid-2021. In addition, Icosavax received $6.5 million from Open Philanthropy to support development of the company’s vaccine platform technology and COVID-19 vaccine candidate. The company is currently advancing the necessary studies to support regulatory filings and has initiated GMP manufacturing. To enable rapid progress of the company’s COVID-19 vaccine candidate to the clinic, Amgen has agreed to manufacture a key intermediate for initial clinical studies.
“This is truly a great example of the scientific community coming together in a time of exceptional need to fight this pandemic. We are grateful to the Bill & Melinda Gates Foundation and Open Philanthropy for their financial support and to Amgen for the supply of a key intermediate for manufacturing,” said Adam Simpson, Chief Executive Officer of Icosavax. “The team at Icosavax is dedicated to advancing vaccines against severe life-threatening respiratory diseases to protect our most at-risk populations. Because VLP vaccines have the potential to induce high-neutralizing antibody titers, our COVID-19 VLP vaccine candidate could be especially important for older adults with age-related declines in immunity.”
Developed by scientists at the University of Washington School of Medicine using structure-based vaccine design techniques invented at the Institute for Protein Design (IPD) at the UW Medicine, IVX-411, the lead vaccine candidate for COVID-19, is a self-assembling protein nanoparticle that displays 60 copies of the SARSCoV- 2 spike (S) glycoprotein receptor-binding domain in a highly immunogenic array. Preclinical data from UW researchers and their collaborators show IVX-411 induces high neutralizing antibody titers in mice after a single administration and further improvement after a second administration (Cell 2020). Titers after the second administration were ten-fold higher than those seen with the soluble SARS-CoV-2 spike protein that forms the basis of many other vaccine candidates. The data also show a strong B-cell response after immunization, critical for immune memory and a durable vaccine effect, with antibodies that target multiple distinct epitopes on the RBD, suggesting potential protection from escape mutations.
“These data highlight that while our VLP-based SARS-CoV-2 vaccine may not be first to market, it has the potential to be a best-in-class vaccine that safely delivers potent and durable immune protection,” said Icosavax co-founder Neil King, Ph.D. Dr. King is also inventor of the computationally designed VLP technology at the Institute for Protein Design, and assistant professor of biochemistry at the UW School of Medicine. “This technology is designed to drive higher neutralizing antibody titers than soluble protein approaches and to create safe, stable, and effective VLP vaccines with simple and scalable manufacturing.”
“It’s clear that we will need a variety of vaccine approaches to effectively fight this novel coronavirus,” said Jean-Paul Prieels, Ph.D., former Senior Vice President of Research and Development at GSK Vaccines and member of the scientific advisory board of Icosavax. “That’s why it’s important to advance not just the fastest but the best vaccine technologies that can deliver safe, effective, and durable protection.”
VLPs enable high-density, multivalent display of antigens in a manner that closely resembles viruses, with an important difference. VLPs contain no genetic material, so they are non-infectious and can provide a safer alternative to live-attenuated or inactivated vaccines. The high yield and stability of the protein components and assembled nanoparticles suggest that manufacture of the nanoparticle vaccines will be highly scalable.
Icosavax has a worldwide license with an exclusive option for IVX-411 in North America and Europe from the University of Washington.
About Icosavax
Icosavax is focused on developing safe and effective vaccines against infectious diseases that cause severe, life-threatening respiratory illnesses. In addition to the COVID-19 vaccine candidate, Icosavax is also advancing IVX-121 into clinical trials as a potential vaccine for respiratory syncytial virus (RSV) for older adults. The company was founded on breakthrough computationally-designed virus like particle technology developed at the Institute for Protein Design and exclusively licensed from the University of Washington. Icosavax is located in Seattle. For more information, please visit www.icosavax.com.
Media Contact:
Jessica Yingling, Ph.D.,
Little Dog Communications Inc.,
jessica@litldog.com,
+1.858.344.8091
Financing round led by Novo Ventures and includes new investors Cowen Healthcare
Investments and Sanofi Ventures, as well as existing investors 5AM Ventures, F-Prime Capital,
Sofinnova Partners and founding investor Epidarex Capital
Proceeds to support advancement of pipeline of small molecule NLRP3 inflammasome inhibitors,
including continued progression of NT-0167 through clinical development
CAMBRIDGE, UK, BOSTON & SEATTLE – June 3, 2020 – NodThera, a clinical stage biotechnology company developing a new class of medicines that inhibit the NLRP3 inflammasome to treat diseases driven by chronic inflammation, today announced that it has secured $55 million (£44 million) in a Series B financing. NodThera’s lead candidate, NT-0167, is being evaluated in a Phase 1 clinical trial in healthy volunteers.
“Millions of people live with diseases in which chronic inflammation plays a role,” said Nanna Lüneborg, Partner, Novo Ventures. “NodThera’s approach to selectively target the NLRP3 inflammasome without broadly suppressing the immune system is an enormously promising strategy for developing therapies that can help these patients.”
Proceeds from the financing will be used to advance NT-0167 through clinical studies, further progress the development of additional compounds — including brain-penetrant NLRP3 inflammasome inhibitors for central nervous system indications — and continued drug discovery efforts.
“The NLRP3 inflammasome is one of the most exciting emerging areas of drug discovery. Therapeutics that disrupt the NLRP3 inflammasome to inhibit damaging inflammatory processes have the potential to help patients with many chronic diseases,” said Henrijette Richter, Managing Partner at Sofinnova Partners. “NodThera has distinguished their science in a field that holds huge promise and we look forward to the company’s further progress.”
The first-in-human clinical study for NT-0167 is evaluating the candidate for safety and tolerability as well as pharmacokinetic and pharmacodynamic response. In preclinical studies, NT-0167 demonstrated potent and selective inhibition of the NLRP3 inflammasome resulting in reductions of IL-1β and IL-18, pro-inflammatory cytokines which are known to play a key role in chronic inflammation underlying a wide range of diseases.
“Continued advancement of our lead molecule through clinical development represents a significant achievement in our work to exploit the still untapped therapeutic potential of selectively targeting the NLRP3 inflammasome,” said Adam Keeney, President and Chief
Executive Officer of NodThera. “This financing from a world-class syndicate of life science investors speaks to the significant potential of our pipeline.”
Joining the NodThera Board as part of this Series B financing will be Nanna Lüneborg, Partner at Novo Ventures, Kevin Raidy, Managing Partner at Cowen Healthcare Investments and Alex Pasteur, Partner at F-Prime Capital.
About NodThera
NodThera is a biotechnology company developing a new class of potent and selective NLRP3 inflammasome inhibitors for the treatment of diseases driven by chronic inflammation. Led by an experienced management team, NodThera is leveraging new insights into inflammasome biology and chemistry to build a platform of highly differentiated small molecule NLRP3 inhibitors. The company was founded by Epidarex Capital and further financed by 5AM Ventures, Cowen Healthcare Investments, F-Prime Capital, Novo Ventures, Sanofi Ventures and Sofinnova Partners. NodThera maintains offices in Cambridge, UK, Seattle, WA and Boston, MA.
For more information please visit www.nodthera.com.
Media Contact
Matthew Corcoran
Ten Bridge Communications
(617) 866-7350
mcorcoran@tenbridgecommunications.com
- BIAL Biotech to be based in Cambridge, Massachusetts and will be a Research Center of Excellence dedicated to genetically-defined Parkinson’s disease
- LTI-291 clinical program and other research programs in Parkinson's disease acquired from Lysosomal Therapeutics, Inc
- R&D team led by Peter Lansbury, professor of neurology at Harvard Medical School
- Investment may add up to 130 million dollars depending on the accomplishment of downstream development, and several regulatory and commercial milestones
Porto / Cambridge, Mass, October 1, 2020 – BIAL, a pharmaceutical company based in Portugal with locations across Europe and dedicated to R&D in CNS diseases, announced today that it has established a new affiliate in the United States of America, BIAL Biotech Investments Inc. (BIAL Biotech). This new research center focused on genetically-defined Parkinson’s disease is based in Cambridge, Massachusetts, a prominent biotech hub in the world.
Simultaneously, BIAL announced that it has acquired worldwide rights of LTI-291 and all the Parkinson’s disease research programs of Lysosomal Therapeutics Inc. (LTI) and taken on the entire R&D team.
António Portela, executive president of BIAL, reveals “Our entry into the US with the creation of BIAL Biotech and the acquisition of the promising programs from LTI, is a decisive step towards the fulfilment of our mission to contribute to improving the quality of life of people worldwide. The development of this new research center in the US, is a landmark of enormous relevance for us. We are investing in science and research, through our direct presence in one of the most important research hubs in the world and in one of the most promising areas of medicine”.
This acquisition not only provides the company with a pipeline of new product candidates in Parkinson’s disease but also an experienced R&D team, led by Peter Lansbury, professor of neurology at Harvard Medical School and a recognized thought leader in the field of neurodegenerative diseases.
With this acquisition, BIAL is expanding its pipeline, namely with the integration of new compounds in neurodegeneration already in clinical development, specifically for Parkinson's disease, where the pharmaceutical company already has a significant market position.
The executive president of BIAL also points out: “The compounds we’ve acquired are based on genetics, a new field of research for us. The lead asset, which now has the code name 'BIA 28-6156/LTI-291', has an innovative mechanism of action and presents the potential of being a first disease-modifying therapy for a genetic subset of Parkinson’s disease. It has successfully completed a Phase I trial program and should be ready to start Phase II studies in 2021. We´re progressing from symptomatic treatment to an intervention in the mechanisms ase, which is very exciting for BIAL”.
“We are happy to be part of BIAL and take the lead on the growth story in the US” says Kees Been, former CEO of LTI and now CEO of BIAL Biotech. "With the commitment and resources of BIAL we will be able to accelerate our novel clinical and research programs as targeted and personalized treatment approaches for genetically-defined PD patients”.
“On behalf of the LTI shareholders, I want to express our enthusiastic endorsement of BIAL as a good home for the programs of LTI”, said Clay B. Thorp, General Partner, Hatteras Venture Partners, “This deal is a perfect fit and we have great confidence that the team, with the resources and commitment of BIAL, will advance LTI-291 and the other compounds rapidly to patients to treat this debilitating form of Parkinson’s disease.”
BIA 28-6156 / LTI-291 is a drug compound aimed at treating patients with GBA-Associated Parkinson’s diseases (GBA-PD) caused by an underlying mutation in the GBA1 gene. This genetic mutation affects approximately 10% to 15% of Parkinson’s disease patients (100,000-150,000 patients in the US) and is a validated risk factor that generally leads to an earlier age of disease onset and more rapid disease progression.
In addition to an upfront payment, the total value of the deal could reach up to 130 million dollars, based on attainment of a series of milestone payments as the assets progress through the clinic and into the market. Other financial terms will not be disclosed.
About BIAL Biotech
BIAL Biotech is a Center of Excellence for the development of genetically-defined Parkinson’s disease therapeutics located in the heart of the Boston biotech cluster. BIAL Biotech is a wholly owned subsidiary of BIAL, a pharmaceutical company based in Portugal with locations across Europe.
About BIAL
Founded in 1924, BIAL's mission is to research, develop and provide therapeutic solutions within the area of health. In the last decades, BIAL has focused strategically on quality, innovation and internationalisation. BIAL is strongly committed to therapeutic innovation, investing more than 20% of its annual turnover into research and development within neurosciences and the cardiovascular system. The company expects to introduce new drugs on the market in the coming years, strengthening its international presence based on proprietary drugs and achieving its goal of supplying innovative products to patients worldwide.
For more information on BIAL: www.bial.com
Media Contacts:
BIAL
Susana Vasconcelos
susana.vasconcelos@bial.com
US Media Contact:
Amanda Whelan
MacDougall Advisors
awhelan@macbiocom.com
Funding to support advancement of powerful bispecific gamma-delta T cell engager programs
for the treatment of a broad range of cancers
New investors include Novo Holdings, Sanofi Ventures, Redmile, Ysios and BB Pureos
Utrecht, The Netherlands and Philadelphia, USA – September 17, 2020 – LAVA Therapeutics, a biotech company pioneering the development of bispecific antibodies to engage gamma-delta T cells for cancer therapies, today announced the closing of an oversubscribed $83 million (€71 million) Series C financing to fund the advancement of its pipeline and platform. The financing was co-led by new investors Novo Ventures, the venture arm of Novo Holdings, and Sanofi Ventures, and included additional new investors Redmile Group, LLC, Ysios Capital and BB Pureos Bioventures. In addition, current investors Versant, Gilde Healthcare and MRL Ventures Fund, LLC participated significantly in the round.
As part of the transaction, Nanna Lüneborg, partner at Novo Ventures, Laia Crespo, EU head of investments at Sanofi Ventures, and Joël Jean-Mairet, managing partner and co-founder of Ysios, joined LAVA as members of the board of directors.
“We are grateful to have attracted a high-quality syndicate of new investors complementing strong continued support of our existing investors. This financing provides meaningful capital to advance our bispecific gamma-delta T cell engager portfolio into multiple proof-of-concept clinical trials expected to start in 2021 for the treatment of solid tumors and hematologic malignancies,” said Stephen Hurly, chief executive officer of LAVA Therapeutics. “We believe our targeted approach, leveraging the unique features of gamma9-delta2 T cells with innovative bispecific antibodies, will deliver novel T cell-based therapies offering advantages over today’s oncology treatments. In addition to the funding raised, the appointments of Nanna, Laia and Joël to our board further strengthen our team, and we look forward to benefiting from their insights and industry expertise.”
Gamma-delta T cells are the natural surveillance cells of the immune system, continuously patrolling the human body for the identification and targeting of tumor cells. These cells bridge the innate with the adaptive immune system and are a largely untapped opportunity in cancer treatment. LAVA’s bispecific gamma-delta T cell engager platform is harnessing the unique properties of these T cells creating a revolutionary truly tumor-targeted immunotherapy to improve outcomes for cancer patients.
“LAVA’s bispecific antibody approach to targeting and engaging gamma-delta T cells has the potential to transform the treatment of a wide range of cancers,” said Dr. Lüneborg. “We are impressed by the preclinical data generated by LAVA to date, which validate the company’s platform and support their transition into a clinical-stage organization. The team is highly experienced in drug development, and I look forward to working with them.”
“Gamma-delta T cells are an emerging field and an incredibly exciting area in oncology. Bispecific antibodies able to directly engage this type of cells offer the potential to significantly impact patients across the globe in these diseases with high morbidity and mortality,” said Dr. Crespo.
About LAVA Therapeutics
LAVA Therapeutics is developing a proprietary bispecific antibody platform that engages gamma-delta T cells for the treatment of hematological and solid cancers. The company’s firstin- class immuno-oncology approach activates Vγ9Vδ2 T cells upon binding to membraneexpressed tumor targets. LAVA was founded in 2016 based on intellectual property originating from the Amsterdam University Medical Center. The company has established a highly experienced antibody research and development team located in Utrecht, the Netherlands (headquarters) and Philadelphia, USA. For more information, please visit www.lavatherapeutics.com.
Contact
Alicia Davis
THRUST Strategic Communications
alicia@thrustsc.com
- Proceeds support clinical development for lead program in cholestatic and uremic pruritis and second program for mast cell-related diseases
- Financing round led by Sanofi Ventures and Cowen Healthcare Investments and included new investors Redmile Group and Perceptive Advisors
- Jason Hafler, Ph.D., of Sanofi Ventures and Kevin Raidy of Cowen Healthcare Investments join Escient board of directors
San Diego, CA – September 14, 2020 – Escient Pharmaceuticals, Inc., an industry-leading company developing Mas-related G Protein-Coupled Receptor (MRGPR)-targeted drugs to address serious, underserved medical needs across a broad range of therapeutic indications, today announced the completion of a $77.5 million Series B financing and the initiation of a Phase 1/1b clinical trial of EP547, a MRGPRX4-targeted product candidate to treat cholestatic and uremic pruritis. The round was led by Sanofi Ventures and Cowen Healthcare Investments (CHI) with participation by new investors Redmile Group and Perceptive Advisors. All of the company’s existing investors, including The Column Group, 5AM Ventures, and Osage University Partners, participated in the round. Escient discovered and is developing EP547 based on the novel finding of disease-related accumulation of key metabolites that specifically activate the MRGPRX4 itch receptor and will use these proceeds to support clinical development. The company is developing a portfolio of MRGPR-focused therapeutics including a second program targeting mast cell-based MRGPRX2 that is anticipated to play a role in a number of indications in various therapeutic areas.
“In just over two years since our launch, we’ve made significant progress developing our platform and pipeline. We have advanced both our understanding of the role MRGPRs in a number of therapeutic areas and our methods for drugging them to potentially treat several diseases based on novel and specific mechanisms of action,” said Alain Baron, M.D., Chief Executive Officer of Escient. “Through our work, we’ve identified promising drug candidates that we are advancing into clinical development with the potential to build a pipeline of class-leading, once-daily, oral therapeutics to address debilitating diseases.”
Effective treatments for severe pruritus associated with cholestasis and uremia are sorely needed. Drug development to address pruritus in these conditions has been hampered by the lack of basic biological knowledge of their specific mechanisms. Cholestasis occurs in chronic adult and pediatric conditions wherein 30-100% of patients experience moderate-to-severe itch. Hemodialysis and end-stage renal disease are largely adult conditions with up to 55% of the population experiencing itch.
“Current treatment options for cholestatic and uremic pruritis are non-specific, largely ineffective, and have significant side effects,” said Marcus F. Boehm, Ph.D., Chief Scientific Officer of Escient. “Based on Escient Series B Financing/Page 2 our preclinical discoveries, we believe we have the opportunity to develop a mechanism-based, well- tolerated, rapidly-acting and highly effective novel therapy for these patients with the potential to make a significant difference in their lives.”
The Phase 1/1b trial of EP547 is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of EP547 in healthy subjects and subjects with cholestatic or uremic pruritus.
In conjunction with the financing, Jason Hafler, Ph.D., of Sanofi Ventures and Kevin Raidy of CHI have joined Escient’s board of directors.
About Escient Pharmaceuticals
Escient Pharmaceuticals is a biotechnology company that is advancing first-in-class G Protein-Coupled Receptor (GPCR)-targeted drugs to address serious, unserved medical needs across a broad range of therapeutic indications. Focused on unleashing the therapeutic potential of specific orphan GPCRs, including the novel family of Mas-Related G-Protein Receptors (MRGPRs), Escient’s lead program targets MRGPRX4. Based in San Diego, Calif., Escient is led by an experienced team of biotechnology entrepreneurs with specific expertise in GPCR drug discovery and development, and funded by top-tier life science investors, including The Column Group, 5AM Ventures, Osage University Partners and Altitude Life Science Ventures. Visit www.escientpharma.com to learn more.
Contact:
Cory Tromblee
Scient Public Relations
cory@scientpr.com
William Hodder
Escient Pharmaceuticals, Inc.
info@escientpharma.com
In a significantly oversubscribed offering, Science 37 attracted capital to support its rapid growth, expand its technology platform and accelerate global expansion.
LOS ANGELES, August 20, 2020 — Science 37, the industry leader in decentralized clinical trials, today announced it closed an oversubscribed $40 million funding round. Existing investors Lux Capital, Redmile Group, and PPD, Inc. (Nasdaq: PPD) led the round, and are joined by existing investors Novartis, Amgen, Sanofi Ventures, GV, and Glynn Capital. Notable new investors include LifeSci Ventures and Mubadala Ventures.
Science 37 will use the new capital to support its rapid growth, expand its technology platform, and accelerate its global expansion efforts—further strengthening its ability to help sponsors execute decentralized trials and enable patients to participate in trials from anywhere, without the burden of traveling to a traditional clinical site.
“The Science 37 mission to democratize clinical research by bringing trials directly to the patients is not only the right thing to do, but it also has had a clear, measurable impact on sponsors’ ability to access a more diverse patient population, accelerate research, and bring new medicines to the market faster,” said David Coman, CEO of Science 37. “We are thrilled to have an incredibly strong, committed network of investors and plan to continue extending our leadership in the decentralized clinical trial market.”
The new investment builds upon immense progress from the company’s previous round, which afforded global expansion; a progressive, BYOD patient experience; world-class collaboration workflow; a unified regional nurse network; and advanced patient recruitment capability. The company also embedded clinical industry veterans in management and implemented a flexible business model to enable a choice between fully decentralized trials, a Science 37 Metasite™—a virtual arm to a traditional site network—and the ability to purchase Science 37 technology with optional services.
“We are in the unique position where the clinical trial industry absolutely has to adapt to a new model, today and going forward,” says Adam Goulburn, partner at Lux Capital. “Science 37 has been preparing for this day for years with an incredible head start. They are uniquely positioned to help sponsors conduct clinical research without the traditional barriers. With their foundation and impressive growth, Lux knows Science 37 will continue to lead the way.”
About Science 37
Science 37 is making the promise of virtual trials the new reality. By engaging with patients from the comfort of their own home, we provide access to patients who can never be reached by traditional site-based models. We have proven to enroll faster, retain patients at a higher rate, and reach a more representative population. Science 37 has conducted more decentralized, interventional trials than any other company, using an expansive network of telemedicine investigators and home-health nurses, who are supported by the industry’s most comprehensive, fully integrated, decentralized clinical trial platform. Learn more at Science 37, and follow Science 37 on Twitter, LinkedIn, and Facebook.
Contact:
Lawrence Lloyd Science 37
Phone: (984) 377-3737
Email: pr@science37.com
# # #
WATERTOWN, Mass., Aug. 20, 2020 (GLOBE NEWSWIRE) -- Kymera Therapeutics, Inc. (NASDAQ: KYMR), a biopharmaceutical company focused on discovering and developing novel small molecule therapeutics that selectively degrade disease-causing proteins by harnessing the body’s own natural protein degradation system, today announced the pricing of its upsized initial public offering of 8,684,800 common shares at a public offering price of $20.00 per share. All of the shares are being offered by Kymera. The gross proceeds of the offering, before deducting underwriting discounts and commissions, and other offering expenses payable by Kymera, are expected to be approximately $173.7 million. Kymera has granted the underwriters a 30-day option to purchase up to an additional 1,302,720 common shares at the initial public offering price.
The shares are expected to begin trading on the Nasdaq Global Market on August 21, 2020 under the ticker symbol “KYMR.” The offering is expected to close on August 25, 2020, subject to the satisfaction of customary closing conditions.
Morgan Stanley, BofA Securities, Cowen, and Guggenheim Securities, are acting as joint book-running managers for the offering.
A registration statement relating to these securities became effective on August 20, 2020. The offering will be made only by means of a prospectus. Copies of the final prospectus, when available, may be obtained from Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014, or by email at prospectus@morganstanley.com; BofA Securities, NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255- 0001 Attn: Prospectus Department, or by email at dg.prospectus_requests@bofa.com; Cowen and Company, LLC, c/o
Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY, 11717, Attention: Prospectus Department, by telephone at (833) 297-2926; Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, New York, NY 10017, by telephone at (212) 518-5548, or by email at GSEquityProspectusDelivery@guggenheimpartners.com.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state, province, territory or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state, province, territory or jurisdiction.
About Kymera
Kymera Therapeutics is a biopharmaceutical company focused on advancing the field of targeted protein degradation by accessing the body’s innate protein recycling machinery to degrade dysregulated, disease-causing proteins to address previously intractable disease targets.
Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding Kymera’s expectations regarding the commencement of trading of its shares on the Nasdaq Global Market, the completion and timing of the closing of the offering and the anticipated gross proceeds from the offering. Forward-looking statements are based on Kymera’s current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to the satisfaction of customary closing conditions and the completion of the offering, and the risks inherent in biopharmaceutical product development and clinical trials. These and other risks and uncertainties are described more fully in the section titled “Risk Factors” in the final prospectus related to the offering to be filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of this date, and Kymera undertakes no duty to update such information except as required under applicable law.
Contact:
Investors@kymeratx.com
Bruce Jacobs
Chief Financial Officer
(857) 285-5314
Integrates new capabilities for pharmaceutical manufacturers, PBMs and payers to simplify rebate negotiation and payment management along with real-time insights into contract performance.
PHILADELPHIA, July 20, 2020 /PRNewswire/ -- HealthVerity, the leader in privacy-protected data exchange, today announced that it has acquired Curisium, an innovator in cloud-based drug rebate processing and contract performance management. The combination of HealthVerity and Curisium will deliver a more complete and responsive infrastructure for high-governance, privacy- compliant data sharing between pharmaceutical manufacturers, pharmacy beneits managers (PBMs) and payers.
Current Curisium clients leverage cutting-edge technologies that streamline and consolidate contract negotiation, rebate validation and invoicing through a fully managed SaaS solution. Built upon a highly responsive, rules-driven solution, Curisium's platform is the operating system that delivers automated workflows standardizing contracting, adjudication, and payment processes, and replacing disparate analog processes and spreadsheets. HealthVerity is now positioned to deliver technologies that can dramatically improve the eficiency and transparency of the cost and complications of patients on a drug. For pharmaceutical manufacturers, brands can more eficiently track the funds flow on existing contracts, better incorporate third party data to understand the healthcare journey of patients on therapy and leverage more advanced technologies to optimize the design and ultimate success of value-based contracts. For PBMs and payers, HealthVerity now represents a turnkey solution for simpliication of the contracting and adjudication process, enabling privacy-protected data exchange that establishes trust between counterparties in pursuit of cost-effective treatment pathways, advanced analytics and superior patient outcomes.
HealthVerity has emerged as a leader and key partner to nearly 75% of top pharmaceutical manufacturers and payers, having fundamentally changed the way that key healthcare organizations secure, exchange and synthesize HIPAA-protected information at scale. With software and APIs that enable access to de-identiied data for more than 330 million individuals across the largest healthcare and consumer data ecosystem in the US, HealthVerity is well positioned to enable clients to signiicantly improve the way counterparties can derive more actionable clinical and inancial impacts.
"Curisium had the vision to build an innovative framework and SaaS platform that allows parties to seamlessly negotiate and visualize contractual relationships around drug utilization, which is fundamentally similar to the HealthVerity approach to making our data ecosystem available for data sharing or analytic applications," said Andrew Kress, CEO of HealthVerity. "The idea of creating a frictionless, standardized, scalable architecture to support privacy-protected data workflows is complementary to everything that we do. In light of the disruptions due to COVID-19 as well as the potential of proposed changes by CMS to value-based agreements, we believe this is an ideal time to scale additional tools that provide our data partners and our healthcare clients an entirely new way to transact around speciic contracts."
"We are excited to join HealthVerity and to continue our mission of empowering life sciences companies and payers with a more agile, progressive approach to healthcare contracting," said Peter Kim, Co-founder and CEO of Curisium. "Our clients will now have access to clinically relevant outcomes that have beneitted from these healthcare contracts, in addition to a greater understanding of the impact on patient lives. We see HealthVerity as being well positioned to become the premier network for privacy-protected data exchange across biopharma, payers, PBMs and pharmacies and we're excited to join forces in this cause."
Financial terms of the transaction were not released. HealthVerity was represented by Blank Rome LLP while Curisium was represented by Cooley LLP.
About HealthVerity
Powering the largest US healthcare and consumer data ecosystem, combined with best-in-class management and privacy solutions, HealthVerity is helping answer healthcare's most critical questions. Our technology platform serves as the foundation for the rapid creation, exchange and management of healthcare and consumer data in a fully-interoperable, privacy-protecting manner. Advantaged by highly sophisticated identity resolution and matching capabilities, HealthVerity is on a mission to increase transparency, forge interoperability and activate deeper insights. To learn more about HealthVerity's technology platform, visit www.healthverity.com.
Contact:
Abigail Stockwell
Senior Director, Marketing
HealthVerity
astockwell@healthverity.com
SOURCE HealthVerity
Related Links
- Kymera to receive $150 million upfront with more than $2 billion in potential milestones plus royalty payments
- Kymera to retain option during clinical development to participate equally in US cost and profit sharing
CAMBRIDGE, Mass. (July 9, 2020) – Kymera Therapeutics Inc. today announced the company has entered into a multi-program strategic collaboration with Sanofi (NASDQ: SNY) to develop and commercialize first-in-class protein degrader therapies targeting IRAK4 in patients with immune-inflammatory diseases. The companies will also partner on a second earlier stage program. Kymera will receive $150 million in cash upfront and may receive more than $2 billion in potential development, regulatory and sales milestones, as well as significant royalty payments. Kymera retains the option to participate in US development and commercialization for both programs. This includes the ability to participate equally in the costs, profits and losses after opt-in, and to co-promote partnered products in the US.
“This is an important collaboration for both companies and for the field of targeted protein degradation,” said Nello Mainolfi, Ph.D., co-founder, President and CEO of Kymera Therapeutics. “Kymera is becoming a fully integrated biotechnology company advancing a pipeline of novel therapies with the potential to transform treatment paradigms. We are excited to partner with Sanofi, an organization with world-class drug development and commercialization capabilities, to ensure maximal patient impact from two of our programs across multiple disease indications, while enabling Kymera to invest in key strategic areas to realize the broad potential of protein degrader therapies.”
Under terms of the collaboration, Sanofi will make an upfront payment of $150 million in cash to Kymera for global rights to develop its small molecule IRAK4 protein degraders in inflammation and immunology indications, and a second earlier stage undisclosed program. IRAK4 is believed to play a key role in multiple immune-inflammatory diseases, including hidradenitis suppurativa, atopic dermatitis and rheumatoid arthritis. Kymera will advance the IRAK4 program through Phase 1 clinical trials; Sanofi will assume clinical development and commercialization responsibilities thereafter. Sanofi will lead all clinical development activities for the second program. Kymera will have the option to participate in the development of both programs in the US during clinical development. Kymera will retain global rights to its IRAK4 program in oncology indications.
IRAK4 is a key protein involved in inflammation mediated by the activation of toll-like receptors (TLRs) and IL-1 receptors (IL-1Rs). While TLR and IL-1R signaling via IRAK4 is involved in the normal immune response, aberrant activation of these pathways is the underlying cause of multiple immune-inflammatory conditions. In pre-clinical studies, Kymera has shown oral daily administration of an IRAK4 degrader can lead to complete knockdown of IRAK4 in skin and immune cells in higher species and is well tolerated. Data presented at the most recent annual meetings of the American College of Rheumatology and the European Hidradenitis Suppurativa Foundation showed potent anti- inflammatory activity in both in vitro and in vivo preclinical models."
“Targeted protein degrada on is an exci ng modality. Kymera has developed an incredible drug discovery engine producing protein degraders with compelling and dieren ated pharmacology against targets that, to date, have not been op mally addressed with other therapeu c modali es,” said John Reed, Global Head of Research & Development at Sano. “We are excited to partner with the Kymera team to advance a new genera on of rst-in-class therapies with the poten al to eliminate underlying drivers of disease.”
Aquilo Partners, L.P. acted as Financial advisor to Kymera on this transac on.
# # #
About Kymera Therapeutics
Kymera Therapeutics is a biotechnology company pioneering a transformative new approach to treating previously untreatable diseases. The company is advancing the field of targeted protein degradation, accessing the body’s innate protein recycling machinery to degrade dysregulated, disease-causing proteins. Kymera’s Pegasus targeted protein degradation platform harnesses the body’s natural protein recycling machinery to degrade disease-causing proteins, with a focus on un-drugged nodes in validated pathways currently inaccessible with conventional therapeutics.
Kymera is accelerating drug discovery with an unmatched ability to target and degrade the most intractable of proteins, and advance new treatment options for patients. For more information, visit www.kymeratx.com.
About Sanofi
Sanofi is dedicated to supporting people through their health challenges. We are a global pharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions. With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe. Sanofi, empowering life. For more information, visit www.sanofi.com.
Sam Marwaha joins as Chief Commercial Officer; Company to deploy virtual
health programs for its network of nearly 4 million individuals later this year
SAN MATEO, Calif., July 1, 2020 — Evidation Health today announced the close of $45 million in Series D funding to power the expansion of its privacy-first, direct-to-person research platform, Achievement, to include virtual health. The company will use this new capital to build upon its existing evidence generation capabilities to offer real-time insights and digital tools to its network of nearly 4 million individuals.
B Capital Group led the round, which includes new investors McKesson Ventures and Section 32 as well as existing investors Revelation Partners, Rethink Impact, and SV Health Investors. B Capital’s Adam Seabrook has joined Evidation’s Board of Directors. Sam Marwaha, Senior Partner at Boston Consulting Group and long-time advisor to Evidation, joins the company as Chief Commercial Officer.
The company will leverage this investment to continue to rapidly grow its study solutions business and offer programs that enable Achievement members to gain clinically actionable insights into their health. These include relevant digital interventions, treatments, and tools to help individuals understand and take advantage of the latest evidence, in partnership with their care teams. The first of these new virtual health offerings is slated for release later this year. Evidation will continue to expand and diversify the Achievement population — already the largest and most demographically and geographically diverse connected cohort in the United States — to serve additional at-risk and disease-specific cohorts. Achievement already includes participants residing in all 50 states and in 9 out of every 10 ZIP codes nationwide.
“Participation in research is an activator for individuals to better understand their health and seek care proactively,” said Evidation CEO Deborah Kilpatrick, Ph.D. “The natural next step for Evidation is to decrease the latency between evidence and action by offering virtual health programs, while still keeping privacy and consent at the forefront. We are especially excited to have Sam Marwaha, a pioneer in real-world evidence and an experienced digital health strategist and operator, join us to lead our commercial endeavors as the company ushers in a new era of self-activated care.”
Marwaha will lead Evidation’s commercial strategy as it brings its new offerings to market. A 20-year industry veteran, Sam will strengthen and broaden partnerships with life sciences companies as they innovate with patients, providers, and payers. He will also scale Evidation’s marketing and business development teams and shape new opportunities to help people achieve better outcomes through virtual health.
“Evidation is a learning engine directly connected to four million people, making it possible to understand real-life indicators of health quality and disease progression, reflecting behavioral and social factors,” said Marwaha. “Evidation can drive personalized, consented engagement with individuals in the safety and convenience of their homes, empowering them to be partners in their own health.”
“Healthcare needs Evidation more than ever before,” said Raj Ganguly, co-Founder and Managing Partner at B Capital Group. “Evidation’s technology has the potential to realize new ways for individuals, providers, biopharma, and payers to understand and react to health in real life. Today’s announcement marks the company’s next step toward achieving that goal and we are pleased to continue our partnership with the company as investors.”
“We see tremendous value in generating real-world data for both clinical development and commercial life sciences use cases, ensuring the best possible understanding of a population inside and outside the clinic,” said Carrie Hurwitz Williams of McKesson Ventures. “The discipline, rigor and scale with which Evidation has built their technology makes it possible to garner relevant real-world data points and transform them into meaningful insights for pharma.”
This funding and C-suite hire caps off a fast-paced growth period for Evidation. Eight of the top 10 global biopharma companies rely on Evidation for person-generated health data (PGHD), including passive sensor data, electronic patient reported outcomes (ePROs), and at-home diagnostics. Johnson & Johnson, Apple, and Evidation recently launched the ongoing Heartline Study, the largest randomized trial in the history of cardiovascular disease. Evidation is also working with a diverse cross section of partners in industry, academia, government, and civil society, including the Icahn School of Medicine at Mount Sinai, the Biomedical Advanced Research and Development Authority (BARDA), and the Bill & Melinda Gates Foundation, on efforts to better detect, characterize and predict COVID-19 using PGHD.
“COVID-19 has shown us that accelerating real-world evidence development is critical,” said Mike Pellini, MD, Managing Partner of Section 32 and former CEO and Chairman of Foundation Medicine. “Evidation has led the way in health measurement and has proven out the potential of person-generated health data. The company is optimally positioned to lead the way into a new era of individualized healthcare.”
About Evidation
Evidation measures health in everyday life and enables anyone to participate in ground-breaking research and health programs. Built upon a foundation of user privacy and control over permissioned health data, Evidation’s Achievement platform is trusted by millions of
individuals—generating data with unprecedented speed, scale, and rigor. We partner with leading healthcare companies to understand health and disease outside the clinic walls. Guided by our mission to enable and empower everyone to participate in better health outcomes, Evidation is working to bring people individualized, proactive, and accessible healthcare—faster. Founded in 2012, Evidation Health is headquartered in California with additional offices around the globe. To learn more, visit evidation.com, or follow us on Twitter @evidation.
Media Contact
press@evidation.com
CAMBRIDGE, Mass., June 10, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (Nasdaq: FULC) today announced the closing of a $68.5 million private placement pursuant to a securities purchase agreement with a group of institutional investors and accredited investors.
The private placement includes a mix of new and existing investors, including EcoR1 Capital, LLC, Alyeska Investment Group, L.P., Boxer Capital, Casdin Capital LLC, Perceptive Advisors LLC, Samsara BioCapital, Monashee Investment Management LLC and Foresite Capital.
SVB Leerink acted as the exclusive placement agent to the Company in connection with the private placement.
In the private placement, the Company sold 4,029,411 shares of common stock at a price of $17.00 per share. The private placement priced on June 8, 2020.
The Company expects to use net proceeds from the private placement to fund research and development expenses, including the ongoing clinical development of losmapimod for facioscapulohumeral muscular dystrophy (FSHD), the initiation of clinical development of losmapimod for COVID-19 pending review of the Company’s recently filed investigational new drug application by the U.S. Food and Drug Administration, the advancement of its hemoglobinopathies program into clinical development and the advancement of both its discovery efforts and product engine capabilities, as well as working capital and other general corporate purposes.
The securities sold in the private placement have not been registered under the Securities Act of 1933, as amended (the “Securities Act”), or any state or other applicable jurisdiction’s securities laws, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws. The Company has agreed to file a registration statement with the U.S. Securities and Exchange Commission (the “SEC”) registering the resale of the shares of common stock issued in the private placement no later than the 15th day after the date of the securities purchase agreement for the private placement.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any offer, solicitation or sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful. Any offering of the securities under the resale registration statement will only be made by means of a prospectus.
About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forwardlooking statements include, but are not limited to, those regarding: the anticipated use of proceeds from the private placement; the filing of a registration statement to register the resale of the shares issued and sold in the private placement; and the Company’s plans, strategies and prospects for its business, including the Company’s plans to evaluate losmapimod as a potential treatment for COVID-19. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company’s strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum’s ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials, if at all; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the Company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date hereof and should not be relied upon as representing the Company’s views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company’s views to change. However, while the Company may elect to update these forwardlooking statements at some point in the future, the Company specifically disclaims any obligation to do so.
Contact:
Investors:
Christi Waarich
Director, Investor Relations and
Corporate Communications
617-651-8664
cwaarich@fulcrumtx.com
Stephanie Ascher
Stern Investor Relations, Inc.
stephanie.ascher@sternir.com
212-362-1200
Media:
Kaitlin Gallagher
Berry & Company Public Relations
kgallagher@berrypr.com
212-253-8881
Collaboration aims to develop predictive algorithm for symptoms of COVID-19 by monitoring first responders
SAN MATEO, Calif., June 4, 2020 – Evidation Health today announced a new effort to develop an early warning algorithm to detect symptoms of COVID-19 and to understand susceptibility to infection, funded by the Biomedical Advanced Research and Development
Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services (HHS) and the Bill & Melinda Gates Foundation.
The Evidation platform will analyze behavior, including sleep and activity patterns, alongside self-reported symptoms for 300 people at high risk of developing COVID-19. This builds on Evidation’s current, ongoing 150,000-person nationwide initiative tracking people’s health and attitudes during the pandemic, COVID-19 Pulse.
“Many infected individuals are asymptomatic but still able to spread the virus, making efforts to prevent and slow transmission of COVID-19 difficult,” said Luca Foschini, Ph.D., Evidation’s co-founder and chief data scientist. “This initiative will use novel behavioral and physiological data to more effectively identify when and where people may contract COVID-19 and can potentially enable real-time interventions to limit spread and monitor outcomes.”
The analysis, performed in collaboration with non-profit 4YouandMe, will use de-identified data generated by self-reporting and wearable devices to track symptoms of COVID-19 in those at particularly high risk, including health care workers and other first responders, in order to better understand susceptibility to SARS-CoV-2 infection. One potential outcome of this work is an early warning algorithm to help individuals better understand and monitor their respiratory disease symptoms and take precautions against their spread.
“The ability to self-monitor and be informed of health status will empower Americans in their decisions to help slow the spread of this pandemic and improve health outcomes for people with COVID-19,” said BARDA Acting Director, Gary Disbrow, Ph.D. “This pilot study is not only an early step in demonstrating the utility of models developed using person-generated health data but also may provide data to better understand the varied symptoms of COVID-19.”
This program follows Evidation’s work with BARDA to monitor individuals for respiratory infections, such as influenza. Evidation’s existing research on influenza utilizes person-generated health data and population-based models with the goal of improving real-time respiratory infection monitoring at the individual and population level. BARDA is contributing a $720,000 award as part of BARDA’s COVID-19 Rapidly Deployable Capabilities program to identify and pilot near-term innovative solutions for COVID-19. Support from the Bill & Melinda Gates Foundation is from the $250 million the foundation has committed to address the COVID-19 pandemic.
About Evidation
Evidation measures health in everyday life and enables anyone to participate in ground-breaking research and health programs. Built upon a foundation of user privacy and control over permissioned health data, Evidation's Achievement platform is trusted by millions of
individuals—generating data with unprecedented speed, scale, and rigor. We partner with leading healthcare companies to understand health and disease outside the clinic walls. Guided by our mission to enable and empower everyone to participate in better health outcomes, Evidation is working to bring people individualized, proactive, and accessible healthcare—faster. Founded in 2012, Evidation Health is headquartered in California with additional offices around the globe. To learn more, visit evidation.com, or follow us on Twitter @evidation.
About HHS, ASPR, and BARDA
HHS works to enhance and protect the health and well-being of all Americans, providing for effective health and human services and fostering advances in medicine, public health, and social services. The mission of ASPR is to save lives and protect Americans from 21st century health security threats. Within ASPR, BARDA invests in the advanced research and development, acquisition, and manufacturing of medical countermeasures – vaccines, drugs, therapeutics, diagnostic tools, and non-pharmaceutical products needed to combat health security threats. To date, 54 BARDA-supported products have achieved regulatory approval, licensure or clearance. Learn more about preparing for and responding to public health emergencies, from new infectious diseases to natural disasters and bioterrorism, by visiting the HHS public health emergency website, www.phe.gov. For more information on partnering with BARDA on developing medical countermeasures, visit www.medicalcountermeasures.gov.
Media Contact
press@evidation.com
- Proprietary tissue-targeted complement platform and best-in-class IL-7R antibody technology to treat patients with severe autoimmune and inflammatory disease; clinical trials begin in 2020
- Previously closed $46M Series A led by Atlas Venture included OrbiMed Advisors, Abingworth, and Sanofi Ventures
- Company appoints Michael Broxson as CEO; co-founder Shelia Violette as Chief Scientific Officer and President of Research
Cambridge, Mass. (May 27, 2020) – Q32 Bio, a biotechnology company developing biologics to restore healthy immune regulation, today announced the company’s pipeline and plans to enter the clinic this year with its lead candidate. Enabled by a $46M Series A financing led by Atlas Venture, and a world class team and Scientific Advisory Board, Q32 Bio is advancing a portfolio of biologics targeting the body’s innate and adaptive immune systems. The company has a robust product pipeline including a monoclonal antibody antagonist of the interleukin-7 receptor (IL-7R), and a complement therapeutics platform that has generated fusion proteins that downregulate complement activity specifically in disease-affected tissues. The company is led by industry veterans Michael Broxson as Chief Executive Officer, and co-founder, Shelia Violette, Ph.D., as Chief Scientific Officer and President of Research.
Q32 Bio was seeded and incubated by Atlas Venture with foundational science from renowned researchers in immunology Michael Holers, M.D. and Joshua Thurman, M.D., both from the University of Colorado, and Steven Tomlinson, Ph.D. from the Medical University of South Carolina. Dr. Holers serves as chairman of the Scientific Advisory Board, which is comprised of distinguished global experts in autoimmune and inflammatory disease.
The subsequent Series A financing included Atlas Venture, OrbiMed Advisors, Abingworth, Sanofi Ventures, University of Colorado and Children’s Hospital Colorado Center for Innovation.
“Autoimmune and inflammatory diseases are driven by dysregulation of the immune response,” said Mr. Broxson. “Q32 Bio has a preeminent team with expertise in both innate and adaptive immunity, a board of directors and scientific advisory board made up of leaders in our field, and a syndicate of blue-chip investors. This gives us a running start in developing therapies that may improve and save lives. I am thrilled to join Q32 Bio and look forward to advancing our first two immune-regulating therapies into first-in-human studies planned for 2020 and 2021.”
Integrated Approach to Restoring Healthy Immune Regulation
Q32 Bio’s most advanced program, ADX-914, is a fully human anti-IL-7R antibody licensed from Bristol Myers Squibb (NYSE: BMY) that re-regulates adaptive immune function. The cytokine IL-7 has demonstrated the ability to drive several T-cell mediated pathological processes. It stimulates T-effector and T-memory cells, lowering the threshold-response to disease related antigens; inhibits the immunosuppressive function of T-regulatory cells; and promotes pathogenic autoantibody production. Inhibition of IL-7R signaling has the potential to durably and safely restore healthy immune regulation in numerous autoimmune and inflammatory diseases. Q32 Bio is advancing plans for the first-in-human trial of ADX-914 while continuing to monitor the potential impact of COVID-19 on clinical operations. Pending confirmation that trial sites are able to operate and enroll patients safely, a biomarker-guided ADX-914 Phase 1 trial is planned to start in late 2020.
Q32 Bio is developing ADX-914 (formerly known as BMS-986265) as part of an exclusive worldwide licensing agreement with Bristol Myers Squibb. Under the terms of the agreement, Bristol Myers Squibb received an upfront payment and became a minority shareholder of Q32 Bio. Bristol Myers Squibb has the potential to earn additional milestone payments subject to the achievement of certain development, regulatory and sales-based milestones, as well as tiered annual net sales royalties. Q32 Bio is solely responsible for all future development and commercialization costs of ADX-914.
Q32 Bio’s lead program in innate immunity, ADX-097, is based on a groundbreaking platform providing tissue-targeted regulation of the complement system. Complement is an integral component of the innate immune system that provides a first line of defense for clearing pathogens and removing damaged cells. In a wide range of autoimmune and inflammatory diseases the complement system becomes hyperactivated, causing the immune system to attack and damage otherwise healthy tissue. Q32 Bio’s proprietary and unique platform has yielded a pipeline of novel protein therapeutics that provide potent inhibition of complement in diseased tissues without long-term systemic blockade – a key differentiator versus current complement therapeutics. Q32 Bio has initiated IND-enabling activities for ADX-097 and first-in-human dosing is planned for 2H 2021.
“Given their broad roles in mediating immunity, the complement system and IL-7 signaling pathways are high potential arenas for drug development,” said Dave Grayzel, MD, Partner, Atlas Venture and chairman of the Q32 Bio Board of Directors. “With a best-in-class IL-7R antibody and fusion proteins designed to target the complement system at sites of activation in a unique and differentiated manner, Q32 Bio is well positioned to make a meaningful impact for patients with serious inflammatory and autoimmune diseases.”
Experienced Leadership Team
Michael Broxson brings over 20 years of industry experience to Q32 Bio. He joined from Goldfinch Bio, where he was Chief Business and Operating officer. Prior to Goldfinch he served in leadership roles in business development, new product planning, strategy and
finance at Takeda Pharmaceuticals. He holds BA and MSPH degrees in Toxicology from Tulane University and an MBA from the University of Chicago’s Booth School of Business and is a CFA® charterholder.
Shelia Violette, Ph.D., joined Q32 Bio from Atlas Venture, where she was Entrepreneur in Residence and initially co-led the formation of Q32 Bio. Previously she served in leadership roles in the Immunology Research group at Biogen, joining Stromedix as Vice President of Research and returning to Biogen as Vice President of Research, spearheading the tissue injury and fibrosis therapeutic area. She holds a Ph.D. in Pharmacology from Yale University.
About Q32 Bio
Q32 Bio is a biotechnology company developing therapies targeting powerful regulators of the innate and adaptive immune systems, to re-balance immunity in severe autoimmune and inflammatory diseases. Q32 Bio’s lead programs, focused on the IL-7R pathway and complement system, address immune dysregulation to help patients take back control of their lives. For more information, please visit www.Q32bio.com.
About Atlas Venture
Atlas Venture is a leading biotech venture capital firm. With the goal of doing well by doing good, the company has been building breakthrough biotech startups for over 25 years. Atlas works side by side with exceptional scientists and entrepreneurs to translate high impact science into medicines for patients. Our seed-led venture creation strategy rigorously selects and focuses investment on the most compelling opportunities to build scalable businesses and realize value. For more information, please visit www.atlasventure.com.
About Abingworth
Abingworth is a leading transatlantic life sciences investment firm. We help transform cutting-edge science into novel medicines by providing capital and expertise to top caliber management teams and building world-class companies. With offices in Menlo Park (California), Boston and London, Abingworth has invested in approximately 160 life science companies, leading to more than 40 M&A/exits and over 65 IPOs since 1973.
About OrbiMed
OrbiMed is a leading healthcare investment firm, with $13 billion in assets under management. OrbiMed invests globally across the healthcare industry, from start-ups to large multinational corporations, utilizing a range of private equity funds, public equity funds, and royalty/credit funds. OrbiMed maintains offices in New York City, San Francisco, Shanghai, Hong Kong, Mumbai and Herzliya. OrbiMed seeks to be a capital provider of choice, providing tailored financing solutions and extensive global team resources to help build world-class healthcare companies.
About Sanofi Ventures
Sanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are oncology, immunology, rare diseases, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. For more information, visit www.sanofiventures.com.
Media Contact
Arleen Goldenberg
Verge Scientific Communications
agoldenberg@vergescientific.com
917.548.1582
Financing will support i20 Therapeutics’ growth and R&D of platform for oral delivery of injectable biologic drugs
BOSTON, MA—April 9, 2020—i2O Therapeutics, an innovative biotech company developing a platform for oral delivery of traditionally injectable biological drugs, announced today it has raised a $4 million seed funding round led by Sanofi Ventures and JDRF T1D Fund.
Founded by a team of researchers from Harvard University, i2O Therapeutics is focused on the development of safe and effective oral formulations of therapies that are conventionally limited to injections, e.g. biologics, large molecules, and peptide-based pharmaceuticals such as insulin. The foundational technology has been exclusively licensed to i2O by Harvard’s Office of Technology
Development. The company’s innovative platform enables drugs that traditionally wouldn’t survive the hostile environment of the digestive system to pass through safely by utilizing a unique coating that dissolves in the small intestine, thereby releasing the active drug. I2O’s initial focus is on developing a novel oral formulation for GLP-1 analogs.
The technology was initially developed in the Harvard lab of Samir Mitragotri, PhD, who is Hiller Professor of Bioengineering and Hansjorg Wyss Professor of Biologically Inspired Engineering at Harvard John A. Paulson School of Engineering and Applied Sciences and a Core Faculty member at Harvard’s Wyss Institute for Biologically Inspired Engineering.
“Our technology has the potential to enable the oral delivery of high-value drugs in a safer, more effective and patient-friendly way and also by easing the treatment burden for dozens of therapeutics that were previously restricted to intravenous or subcutaneous delivery,” commented Mitragotri, Cofounder of i2O Therapeutics.
“The support and partnership of Sanofi Ventures and JDRF T1D Fund marks a major milestone for i2O, and potentially millions of people around the world. This round of financing will enable us to rapidly expand our team and ramp up research and development at i2O as we seek to create the next generation of oral peptide and protein-based therapies,” commented Ravi Srinivasan, PhD, Co-founder of i2O Therapeutics.
“i2O Therapeutics is developing an extremely promising new platform for oral biologics with the potential to significantly ease treatment burden for countless patients who rely on drugs that can only be administered via injection,” said Christopher Gagliardi, PhD, Director of Investments at Sanofi Ventures.
“We are excited to partner with i2O Therapeutics, whose platform has the potential to revolutionize the way people with diabetes manage their disease,” commented Katie Ellias, Managing Director of the JDRF T1D Fund. “The possibility of an oral insulin product, among other exciting applications of the i2O platform, represents a significant commercial opportunity and more importantly, has the potential to improve glycemic management and decrease hypoglycemia risk over today’s injectable insulins.”
Both Christopher Gagliardi and Katie Ellias will join the i2O Board of Directors.
About i2O Therapeutics
i2O Therapeutics is a biotechnology company developing safe and effective oral formulations of therapies traditionally limited to injections. Using an innovative ionic liquid technology, this platform leverages the benefits of protecting the drug cargo while also transiently enhancing permeation across the epithelial lining when administered orally. i2O is focused on creating the next generation of oral peptide and protein-based therapies. Visit us at www.i2OBio.com.
About JDRF T1D Fund
The JDRF T1D Fund (https://t1dfund.org/) is a venture philanthropy fund accelerating life-changing solutions to cure, prevent and treat type 1 diabetes (T1D) through catalytic equity investments. Through its investments in partnership with private capital, including venture capital, corporations and foundations, the T1D Fund seeks to attract the private investment necessary to advance therapeutics, devices, diagnostics, and vaccines into the hands of those living with T1D. The T1D Fund invests in areas strategically aligned with JDRF, the leading global organization funding T1D research, with an exclusive focus on supporting the best commercial opportunities. The T1D Fund reinvests any realized gains into new investments to further its mission.
About Sanofi Ventures
Sanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are oncology, immunology, rare diseases, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. For more information, visit www.sanofiventures.com.
Contact
Gregory Johnson, PhD
MacDougall
Gjohnson@macbiocom.com
- First patient dosed in Phase 1 clinical trial evaluating IL-7R antibody ADX-914
- Financing round co-led by OrbiMed Advisors and Acorn Bioventures fuels advancement of portfolio
Cambridge, Mass. (October 29, 2020) – Q32 Bio, a biotechnology company developing biologic therapeutics to restore healthy immune regulation, today announced the completion of a $60 million Series B financing co-led by OrbiMed Advisors and Acorn Bioventures. The company also announced the commencement of a Phase 1 clinical trial evaluating its best-in-class IL-7R antibody ADX-914, a significant milestone marking Q32 Bio’s entry into clinical-stage development.
Proceeds from the Series B financing will support the advancement of ADX-914 through demonstration of proof-of-mechanism and propel the company’s groundbreaking tissue-targeted complement regulation platform. ADX-097 is the first fusion protein generated by Q32’s complement platform and is expected to enter Phase 1 in the fourth quarter of 2021. The financing also enables further refinement and expansion of both the company’s platform and pipeline.
“The closing of our Series B financing and entry into the clinic are pivotal milestones for Q32 Bio,” said Michael Broxson, CEO of Q32 Bio. “The initiation of the Phase 1 trial for ADX-914 represents an important achievement and a significant step toward bringing a powerful new treatment option to patients with autoimmune disease. We expect to continue to make major advances over the coming year as we move ADX-097 into the clinic.”
In addition to Orbimed Advisors and Acorn Bioventures, Series B investors include Osage University Partners, Atlas Venture, Abingworth, Sanofi Ventures, University of Colorado and Children’s Hospital Colorado Center for Innovation. In conjunction with the Series B financing, Isaac Manke (Acorn Bioventures) and Diyong Xu (OrbiMed Advisors) will join the Q32 Bio Board of Directors.
“We are thrilled to partner with a stellar group of investors to support Q32’s novel therapeutic platform,” said Stephen Squinto, Ph.D., Executive Partner, OrbiMed Advisors, member of the Q32 Board of Directors, and Scientific Advisor to the company. “With a premier investor syndicate and world class leadership team, Q32 Bio is well positioned to advance a differentiated portfolio of therapies targeting important regulators of both adaptive and innate immunity.”
“Q32 Bio is an ideal investment for Acorn Bioventures, which provides committed capital through multiple financing rounds to innovative and entrepreneurial biotech companies,” said Isaac Manke, Acorn Bioventures partner and Q32 Bio Board member. “We are delighted to partner with the company’s outstanding leadership team in Q32’s mission to create the next generation of immune-modulating therapies for patients.”
About Q32 Bio
Q32 Bio is a clinical stage biotechnology company developing therapies targeting powerful regulators of the innate and adaptive immune systems to re-balance immunity in severe autoimmune and inflammatory diseases. Q32 Bio’s lead programs, focused on the IL-7R pathway and complement system, address immune dysregulation to help patients take back control of their lives.
Q32 Bio’s approach centers on restoring healthy immune regulation in autoimmune and inflammatory disease, with a focus on pathways that are critical modulators of innate and adaptive immunity. The first-in-human trial for the company’s most advanced program, ADX-914, a fully human anti-IL-7R antibody, is designed to evaluate safety and tolerability and demonstrate proof-of-mechanism. IL-7 has been genetically and biologically validated to drive several T cell-mediated pathological processes in numerous autoimmune diseases.
Q32 Bio’s lead program for innate immunity, ADX-097, is based on a pioneering approach enabling tissue-targeted regulation of the complement system without long-term systemic blockade – a key differentiator versus current complement therapeutics. First-in-human dosing of ADX-097 is planned for 2H 2021.
For more information, please visit www.Q32bio.com.
###
Media Contact
Arleen Goldenberg
Verge Scientific Communications
agoldenberg@vergescientific.com
917.548.1582
PARIS and NEW YORK – November 20, 2019 - Sanofi announced today an enterprise- wide collaboration with health care technology company Aetion that will integrate Sanofi’s real-world data platform, DARWIN, with the Aetion Evidence Platform® with the objective of advancing more efficient use of real-world evidence (RWE), facilitating regulatory-grade studies with deep transparency, and unlocking access to new real-world data.
Both companies have invested in RWE platforms, recognizing the pressing need for accurate, fast, and cost-effective research and the important role RWE could play in meeting this need. Sanofi’s DARWIN compiles and analyzes de-identified data from hundreds of millions of patients across disease states, while Aetion’s platform analyzes real-world data to produce transparent, rapid, and scientifically validated answers about the effectiveness, safety, and value of drugs. By combining these platforms, Sanofi is seeking to elevate its capabilities in conducting regulatory-grade analytics, opening new doors for the development and application of medical treatments.
“Today marks another important step in Sanofi’s digital transformation,” said Bernard Hamelin, MD, MSc, MBA, Global Head of Medical Evidence Generation, Sanofi. “By integrating these platforms, we strive to make faster, more informed decisions with the potential to lead to first-in-class and best-in-class treatments that could change the practice of medicine.”
Real-world evidence offers a view of clinical practice outside of the experimental setting, providing an opportunity to inform clinical trial development and supplement trial data with evidence of actual product use in the health care system.
“Our work with Sanofi further validates the value and potential for real-world evidence in drug development,” said Carolyn Magill, Chief Executive Officer of Aetion. “Our companies share a common goal of using the best available data to get the right treatment to the right patient as quickly and efficiently as possible.”
This collaboration between Sanofi and Aetion demonstrates leadership during a critical time. Real-world evidence is expected to play a key role in transforming the health care ecosystem, with the U.S. Food and Drug Administration (FDA) recently prioritizing efforts to incorporate RWE as a companion to clinical trial data to aid in regulatory decision making. The FDA will release its draft RWE guidance before the end of 2020.
About Aetion
Aetion is a health care technology company that delivers real-world evidence for life sciences companies, payers, at-risk providers, and regulatory agencies. The Aetion Evidence Platform analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on treatments, costs, and outcomes. Founded by Harvard Medical School faculty with decades of experience in epidemiology and health outcomes research, Aetion informs health care's most critical decisions — what works best, for whom, and when — to guide treatment development, commercialization, and payment innovation into health care's modern era. Aetion is based in New York City, and backed by investors including New Enterprise Associates (NEA), Flare Capital Partners, Lakestar, Town Hall Ventures, McKesson Ventures, Sanofi Ventures, Amgen Ventures, UCB, and Horizon Health Services, Inc. Learn more at aetion.com, and follow us at @aetioninc.
About Sanofi
Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Sanofi, Empowering Life
Sanofi Media Relations Contact
Anna Robinson
Tel.: +33 (0)1 53 77 46 46
mr@sanofi.com
Aetion Media Relations Contact
202.792.7200
press@aetion.com
Sanofi Investor Relations Contact
George Grofik
Tel.: +33 (0)1 53 77 45 45
ir@sanofi.com
Sanofi Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward- looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic conditions, the impact of cost containment initiatives and subsequent changes thereto, the average number of shares outstanding as well as those discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2018. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any
forward-looking information or statements.
YTX-7739 represents a novel, first-in-class, potentially disease-modifying therapy
Data from Phase 1 study expected in the first quarter of 2020
CAMBRIDGE, Mass. – October 7, 2019 – Yumanity Therapeutics, a company focused on protecting the vitality of the mind by discovering and developing transformative brain-penetrating small molecule drugs to treat neurodegenerative diseases, today announced that the first subject cohort has been dosed in a Phase 1 clinical trial evaluating the safety and tolerability of YTX-7739 in healthy volunteers. YTX-7739, the company’s lead investigational therapy, is designed to inhibit Stearoyl-CoA-Desaturase (SCD), a validated biologic target that has recently shown potential in neurodegenerative diseases by protecting cells from a-synuclein toxicity, a major driver of Parkinson’s disease.
“Developing effective therapies for patients with devastating neurodegenerative diseases has been challenging because too few hypotheses and novel targets have been explored,” said Kenneth Rhodes, Ph.D., chief scientific officer at Yumanity Therapeutics. “We advanced YTX-7739, an orally-active SCD inhibitor, into clinical development because of recent evidence established at Yumanity Therapeutics demonstrating its promise to protect cells from a-synuclein toxicity. We look forward to fully characterizing the potential clinical use of YTX-7739, which is clearly differentiated from currently available Parkinson’s disease therapies that only address the symptoms, not the underlying causes.”
The double-blind, placebo-controlled, dose-escalation, crossover study is intended to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of YTX-7739 in adult healthy volunteers. A second study, exploring multiple ascending doses in adult healthy volunteers and patients with Parkinson’s, will follow. Approximately 40 participants will be enrolled in this Phase 1 single ascending dose study. Following completion of the Phase 1 studies, Yumanity Therapeutics expects to advance YTX-7739 into a Phase 1b proof-of-concept clinical trial in the second half of 2020.
“Since Yumanity Therapeutics’ inception, our goal has been to uncover novel pathways and targets to tackle significant medical challenges,” said Richard Peters, M.D., Ph.D., chief executive officer of Yumanity Therapeutics. “Moving from target identification of SCD to initial clinical development of YTX-7739 in just three years is a testament to the enormous potential of our discovery platform to reproducibly identify previously unexplored biology and new, druggable targets that have the potential to protect cells from neurodegeneration. This Phase 1 trial will provide important validation for the broad application of our technology to help address arguably the most important therapeutic challenges of our time.”
About YTX-7739
YTX-7739 is Yumanity Therapeutics’ proprietary lead investigational therapy designed to penetrate the blood-brain barrier and inhibit the activity of a novel target that plays an important and previously unrecognized role in the neurotoxicity caused by the a-synuclein protein, a major driver of Parkinson’s disease and related neurodegenerative disorders. Misfolding and aggregation of the a-synuclein protein triggers a cascade of events, ultimately resulting in neurotoxicity and the subsequent disorders in movement and cognition that affect patients living with these diseases. YTX-7739 has been shown to inhibit many of the key aspects of a-synuclein toxicity and the company is assessing its potential utility in Parkinson’s disease.
About Parkinson’s Disease
Parkinson’s disease is a progressive neurological disorder that affects the central nervous system and impacts both motor and non-motor functions. It is one of the most common age-related neurodegenerative diseases, affecting an estimated 0.5 to 1 percent of people 65 to 69 years of age, rising to 1 to 3 percent of the population over the age of 80.1 Symptom severity and disease progression differ between individuals, but typically include slowness of movement (bradykinesia), trembling in the extremities (tremors), stiffness (rigidity), cognitive or behavioral abnormalities, sleep disturbances and sensory dysfunction.2 There is no laboratory or blood test for Parkinson’s disease, so diagnosis is made based on clinical observation.3 Currently, there is no cure and available treatments only address the symptoms of Parkinson’s disease, not the underlying causes.
About Yumanity Therapeutics
Yumanity Therapeutics is transforming drug discovery for neurodegenerative diseases caused by protein misfolding. Formed in 2014 by renowned biotech industry leader, Tony Coles, M.D., and protein folding science pioneer, Susan Lindquist, Ph.D., the company is focused on discovering disease-modifying therapies for patients with Parkinson’s disease and related disorders, amyotrophic lateral sclerosis (ALS), and Alzheimer’s disease. Leveraging its proprietary discovery engine, Yumanity Therapeutics’ innovative new approach to drug discovery and development concentrates on reversing the cellular phenotypes and disease pathologies caused by protein misfolding. For more information, please visit yumanity.com.
# # #
Media Contact:
Rachel Eides
W2O pure
reides@purecommunications.com
Investor Relations Contact:
Caroline Corner, Ph.D.
Westwicke
caroline.corner@westwicke.com
- Two-component virus-like particle (VLP) technology developed at the Institute for Protein Design exclusively licensed from the University of Washington for a variety of infectious disease indications
- License secured from NIH for DS-Cav1 (clinically validated prefusion F antigen) for RSV indication
SEATTLE, October 3, 2019 – Icosavax, Inc. today announced the launch of the company with a $51 million Series A financing, led by Qiming Venture Partners USA and joined by Adams Street Partners, Sanofi Ventures and NanoDimension, with continuing support from its seed investors. The company was founded on computationally designed self-assembling virus-like particle (VLP) technology developed at the Institute for Protein Design (IPD) at University of Washington School of Medicine (Cell 2019, Preview). The proceeds of the financing will be used to advance the company’s first vaccine candidate, IVX-121, for respiratory syncytial virus (RSV) for older adults through Phase 1b clinical studies. Icosavax also announced today its leadership team, board of directors and key scientific advisors.
“Working closely with the Institute for Protein Design, we started Icosavax with seed funding from its philanthropic supporters,” said Adam Simpson, Chief Executive Officer of Icosavax. “This support allowed us to assemble a world-class team and to help translate the scientific insights from IPD into IVX-121, our lead candidate for RSV. We are thrilled with the quality of the investor syndicate we have built who provide both significant expertise and financial support to enable advancement of IVX-121 into the clinic and the application of our VLP technology to a whole class of vaccine targets with significant unmet medical needs.”
VLPs enable high-density, multivalent display of antigens in a manner that closely resembles viruses, with an important difference. VLPs contain no genetic material, so they are non-infectious and can provide a safer alternative to live-attenuated or inactivated vaccines. Naturally occurring VLPs have delivered successful vaccines, including Gardasil® and Cervarix® against human papillomavirus (HPV) and Engerix-B® and Recombivax HB® against Hepatitis B. However, VLPs have been difficult to use for the display of complex heterologous antigens, like in the case of RSV.
Mark McDade, who is managing partner of Qiming Venture Partners USA and was previously a founder, COO and director of Corixa Corporation (sold to GSK), said “We were extremely impressed with this novel approach using computational protein design to create VLP-based vaccines that have improved efficacy and are simple to manufacture. Our investment in Icosavax supports the value of science and technology to improve public health and our belief that preventing infection is preferable to treating illness.”
“Icosavax’s vaccine technology solves the problem of constructing and manufacturing VLPs displaying complex antigens by utilizing computationally designed proteins that separate the folding of individual protein subunits from the assembly of the final macromolecular structure. The individual proteins are expressed and purified using traditional recombinant technologies, and then self-assemble into VLPs when mixed together,” said Icosavax co-founder Neil King, Ph.D. VLPs are known to induce superior immunological responses compared to traditional soluble antigens, eliciting protective immune responses while reducing the need for strong adjuvants, which in some instances have been associated with side effects.
The company’s RSV vaccine candidate, IVX-121, incorporates a stabilized prefusion F antigen licensed from NIAID/NIH (DS-Cav1; Science 2019). Extensive preclinical studies conducted at IPD and Icosavax suggest that IVX-121 could increase the protective immunogenicity of RSV F compared to the DS-Cav1 antigen alone.
Tadataka (Tachi) Yamada, M.D., chair of Icosavax’s board of directors explained, “RSV can cause a life-threatening respiratory infection with consequences as severe as those associated with influenza in older adults. An effective RSV vaccine could have an impact on the lives of millions of people around the world.”
Tachi previously served as chief medical and scientific officer of Takeda Pharmaceuticals, where he was responsible for the acquisitions of Inviragen (dengue vaccine) and LigoCyte (norovirus vaccine). The board of directors also includes Terry Gould, a partner and head of Growth Equity Investments, Adams Street Partners; Jason Hafler, the U.S. Head of Investments at Sanofi Ventures; Eric Moessinger, a partner of NanoDimension, Mark McDade, and Adam Simpson.
Simpson added, “We are pleased to announce our leadership team who has extensive experience in successful vaccine development and our board of directors and key advisors, which include leaders in computational protein design and global vaccine programs, experts in successfully advancing novel technologies to vaccine products, and entrepreneurs who have helped companies develop and create commercially valuable medical products that benefit human health.”
The founding leadership team of Icosavax:
- Adam Simpson is the chief executive officer (CEO). He is also the CEO of PvP Biologics, another spinout from IPD. Previously he served as president and chief operating officer (COO) of Cypher Genomics (sold to Human Longevity), and chief business officer, Meritage Pharma (sold to Shire).
- Doug Holtzman, Ph.D., MPH is the chief scientific officer. Previously, he served as vice president, Discovery, Takeda, and deputy director, Childhood Pneumonia, Bill & Melinda Gates Foundation. Prior to his work in global health he worked in a number of innovative early-stage biotechnology companies, including Millennium Pharmaceuticals (sold to Takeda) and Ironwood.
- Niranjan Kanesa-thasan, M.D., MTMH, FIDSA, FASTMH is the chief medical officer and has contributed to the development of seven licensed vaccines. Previously, he served as the clinical franchise head, GSK Vaccines; chief medical officer, Americas, Novartis Vaccines; and vice president, Medical Affairs and Pharmacovigilance, Acambis (sold to Sanofi Pasteur).
- Charles Richardson, Ph.D. is the senior vice president, Technical Operations. Previously, he served as global head, CMC, Takeda Vaccines; senior vice president, R&D, LigoCyte (sold to Takeda); vice president, Manufacturing, Corixa (sold to GSK); and vice president, Pharma Discovery, RibiImmunoChem (sold to Corixa).
Icosavax’s founding scientists, scientific advisory board (SAB) members, and key advisors are:
- Neil King, Ph.D., is a co-founder of Icosavax, inventor of the computationally designed VLP technology, and chair of the SAB. He is a researcher at the Institute for Protein Design and an assistant professor of biochemistry at the UW School of Medicine.
- David Baker, Ph.D., is a co-founder of Icosavax. He is the director of the Institute for Protein Design, an endowed professor of biochemistry at the University of Washington School of Medicine, and a Howard Hughes Medical Institute (HHMI) investigator.
- Ralf Clemens, M.D., is a development advisor and SAB member and a leading expert in vaccinology with more than 30 years of experience in global vaccine development at Takeda, Novartis, and GSK. Ralf developed and brought to licensure more than 25 different vaccines and has published extensively on vaccines and public health.
- Christian Mandl, M.D., Ph.D., is a SAB member and has an accomplished academic career in molecular and clinical virology. Previously, he served as global head of Research, Early and Exploratory Clinical Development at Novartis Vaccines, leading more than 300 discovery and clinical researchers in the development of a broad range of viral and bacterial vaccines, adjuvants, and delivery platforms.
- Jean-Paul Prieels, Ph.D., is a SAB member and his career spans from basic research to process and product development. He previously served as a senior vice president of Research and Development at GlaxoSmithKline Biologicals (now GSK Vaccines). He was instrumental in developing several commercially available vaccines, such as rotavirus, human papilloma virus (HPV), pneumococcal conjugates, and others.
- Barney S. Graham, M.D., Ph.D., is a SAB member, and inventor of DS-Cav1, the clinically validated RSV F antigen incorporated into IVX-121. He is an immunologist, virologist, and clinical trials physician. He was one of the founding investigators for the National Institute of Allergy and Infectious Diseases (NIAID) Vaccine Research Center (VRC) at the National Institutes of Health (NIH), where he is now the deputy director and chief of the Viral Pathogenesis Laboratory and oversees the advanced development of VRC candidate vaccine products.
Icosavax’s in-licensed computationally designed VLP technology is a product of the IPD’s Translational Investigator Program, which enables entrepreneurial researchers to turn their first working versions of designed proteins into commercially viable assets. The license to this technology was negotiated with CoMotion at the University of Washington, UW’s collaborative innovation hub.
About Icosavax
Icosavax is focused on developing safe and effective vaccines against infectious diseases that address important unmet medical needs and reduce healthcare costs. The company was founded on breakthrough computationally-designed virus like particle technology, exclusively licensed for a variety of infectious disease indications from the Institute for Protein Design at the University of Washington. Icosavax is located in Seattle. For more information, please visit www.icosavax.com.
Media Contact:
Jessica Yingling, Ph.D.
Little Dog Communications Inc.
jessica@litldog.com
+1.858.344.8091
Funding to Advance Development of Drug Candidates Targeting
Heparin-Induced Thrombocytopenia and Type 1 Diabetes
FREDERICK, MD – September 5, 2019 – Veralox Therapeutics (“Veralox”), a preclinical stage company focused on accelerating the development of first-in-class therapeutics for unmet medical needs, today announced it has raised a $5.4 million seed funding round. The funding was led by the JDRF T1D Fund, Sanofi Ventures, and the VTC Innovation and VTC Seed Fund, with participation from the Maryland Momentum Fund, the University of Vermont Health Network and TEDCO.
Veralox is headquartered in Maryland and founded in 2017 by Drs. Jeffrey Strovel, David Maloney, and Matthew Boxer, three industry veterans with a track record of successful drug discovery and development. The company is currently developing therapeutics targeting 12- lipoxygenase (12-LOX). The new funding will be used to support the pre-clinical development of therapeutics to block the 12-LOX enzyme for several indications, including the rare hematological indication heparin-induced thrombocytopenia and thrombosis (HITT) and type 1 diabetes (T1D). Veralox’s lead candidate, VLX-1005, was discovered and developed in collaboration with University of California Santa Cruz, Thomas Jefferson University, NIH, and Eastern Virginia Medical School.
“We are excited to fuel our mission to advance the development of therapeutics for immunemediated disorders such as HITT and T1D,” said Jeffrey Strovel, CEO of Veralox Therapeutics. “With the support of the T1D Fund, Sanofi Ventures, and our other investors, we are well positioned to broaden the potential indications for our technology while ensuring sufficient capital to prepare for clinical studies in patients.”
“Beta cell therapies are a key strategic area of focus for the T1D Fund and we believe Veralox’s innovative approach to inhibiting beta cell stress has the potential to create a first-in-class drug targeting a novel pathway to address type 1 diabetes,” said Katie Ellias, Managing Director at the JDRF T1D Fund. “JDRF was an early supporter of work on this pathway, and we look forward to supporting the company and collaborating with Sanofi and the other investors to accelerate the development of this promising therapy.”
“Veralox fits within our strategic investing mandate and we are excited by the potential for 12-LOX inhibition in both T1D and HITT. We see the potential to impact patients across the globe in these diseases with high morbidity and mortality,” said Daniel Mansuri from Sanofi Ventures.
Both Daniel Mansuri and Katie Ellias will join the Board of Directors.
Veralox is pioneering the development of inhibitors of 12-LOX, which plays key a role in platelet activation and mediation of cellular stress signaling. Halting the immune driven activation of platelets reduces blood clot formation, offering the promise of an orphan hematological indication for the immune-mediated coagulation disorder known as heparin-induced thrombocytopenia and thrombosis (HITT). In T1D, beta cell stress leads to cell dysfunction and death. By keeping beta cells healthy and functional, Veralox’s inhibitor may halt T1D disease progression and has the potential to be used at all stages of the disease.
###
About Veralox Therapeutics
VERALOX Therapeutics Inc. (https://veralox.com/) is developing first-in-class therapeutics that target the underlying pathologies of thrombosis and type 1 diabetes. The company’s lead candidate, VLX-1005, will be developed initially to treat patients with heparin-induced thrombocytopenia (HIT) and HIT with thrombosis (HITT). Second generation therapeutic products are under development for type 1 diabetes (T1D) as well as other immune-mediated and inflammatory diseases.
JDRF T1D Fund
The JDRF T1D Fund (https://t1dfund.org/) is a venture philanthropy fund accelerating lifechanging solutions to cure, prevent and treat type 1 diabetes (T1D) through catalytic commercial investments. Through its investments in partnership with private capital, including venture capital, corporations and foundations, the T1D Fund seeks to attract the private investment necessary to advance drugs, devices, diagnostics, and vaccines into the hands of
those living with T1D. The T1D Fund invests in areas strategically aligned with JDRF, the leading global organization funding T1D research, with an exclusive focus on supporting the best commercial opportunities. The T1D Fund will reinvest any realized gains into new investments to further its mission.
Sanofi Ventures
Sanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in earlystage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are rare diseases, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. For more information, visit www.sanofiventures.com.
Media Contacts:
Contact@Veralox.com
301-360-3502
JDRF T1D Fund
Scott Lessne
Stanton
slessne@stantonprm.com
646-502-3569
NEW YORK – September 4, 2019 - Click Therapeutics, Inc. (“Click”), a leader in Digital Therapeutics™ solutions as prescription medical treatments, today announced the appointment of Ross J. Muken as Chief Financial Officer. Mr. Muken brings to Click over 18 years of Wall Street experience and a distinguished track record of success, having been broadly recognized as a thought leader across the healthcare sector. Mr. Muken joins the Click team from Evercore ISI, where he was a Senior Managing Director and Partner.
“We are thrilled to welcome Ross to the leadership team,” said David Benshoof Klein, co-founder and CEO of Click. “Ross has had unrivaled insight into our industry since inception. His deep expertise across the healthcare continuum, strong relationships within the community, and wealth of experience will help us to capitalize on our leading position as we transition toward the next stage of rapid growth.”
“I’ve had the privilege of covering a number of disruptive technology companies in the healthcare space throughout my career, and Click is the most exciting opportunity I have come across to-date, with a clear path to becoming the leader in the digital therapeutics space,” said Ross J. Muken. “I am extremely impressed by the tremendous progress that Click has made in the past year and am pleased to join Click at this pivotal time as it is advancing several first-line digital therapeutics into the market. I look forward to leveraging my experience to support Click’s future growth and financial strategy as it builds upon the industry’s most advanced digital therapeutics and patient engagement platform.”
Ross J. Muken was previously Partner, Senior Managing Director and Head of the Healthcare Services & Technology Research Team at Evercore ISI, widely recognized as the #1 independent equity research franchise on Wall Street. He provided thoughtful insights on over 50 companies across the Healthcare Technology & Distribution, Life Science Tools & Diagnostics, Managed Care & Facilities and Medical Supplies & Devices sub-sectors. Ranked amongst the top analysts by Institutional Investor over the past decade, Mr. Muken is well-known for his proprietary fundamental research and highly accurate financial modeling as well as his work with emerging and disruptive technologies across the healthcare continuum. Prior to joining Evercore ISI, Mr. Muken served as a Managing Director at Deutsche Bank Securities, where he helped build the healthcare platform into a top franchise at the company. Previously, Mr. Muken spent time in the Equity Research division of Thomas Weisel Partners and began his career as an M&A Investment Banker at Bank of America Securities. Mr. Muken earned his B.S. in Business Administration, magna cum laude, at Boston University.
About Click Therapeutics
Click Therapeutics, Inc. develops and commercializes software as prescription medical treatments for people with unmet medical needs. Through cognitive and neurobehavioral mechanisms, Click’s Digital Therapeutics™ enable change within individuals, and are designed to be used independently or in conjunction with biomedical treatments. The Clickometrics® adaptive data science platform continuously personalizes user experience to optimize engagement and outcomes. Following a groundbreaking clinical trial, Click’s industry-leading smoking cessation program is available nationwide through a wide variety of payers, providers, and employers. Click’s lead prescription program is entering into a multi-center, randomized, controlled, parallel-group, phase III FDA registration trial for the treatment of Major Depressive Disorder in adults. For more information, visit ClickTherapeutics.com.
101 Avenue of the Americas, 8th Floor
New York, NY 10013
www.clicktherapeutics.com
# # #
Company Contact
Sarah Jackson
Chief of Staff
sarah@clicktherapeutics.com
Media Contacts
Karen Sharma
ksharma@macbiocom.com
781-235-3060
Digital Care Leader’s Integrated Program Supports Individuals Dealing with Chronic
Disease, Mental Health Issues
San Francisco, CA; (June 26, 2019) - Omada Health today announced a $73 million round of funding led by Wellington Management Company LLP. Wellington Management is an independent investment firm with more than $1 trillion of client assets under management. Omada will use the funding to fuel the continued expansion of its digital care program, including support for those with type 2 diabetes and hypertension, as well as those dealing with anxiety and depression.
Omada works with more than 600 employers and health plans across all 50 states, delivering an integrated experience that adapts to participants health needs, and personalizes their journeys to create the best health outcomes. Omada’s dedicated coaches guide each individual throughout the program, achieving unparalleled engagement and sustainable behavior change. Omada’s proprietary coaching platform enables them to deliver personalized interventions at scale. The company proved the model for digital care as the nation’s largest CDC-recognized provider of the National Diabetes Prevention Program, and has now launched programs over the last twelve months for diabetes self-management, as well as anxiety and depression, to holistically address individuals’ health needs.
“Ten years from now, the most engaging and utilized healthcare provider in the U.S. will be digital. Omada is poised to be that provider, as we inspire and engage the more than 100 million patients who need additional care and support between physician visits,” said Omada Health CEO and co-founder Sean Duffy. “Today’s announcement -- and the incredible roster of investors participating in this round of fundraising -- will deepen our collaboration with health plans, employers, and health systems, and accelerate the development of our truly personalized program that helps our participants build patterns that spark lifelong health.”
“Omada has become a category-defining company in digital healthcare,” added Vijay Pande, General Partner at Andreessen Horowitz. “Since our first investment, the team’s vision, rigorous clinical focus, and intelligent application of sustainable behavior change across multiple conditions has raised the bar for what health plan buyers, and individuals, should expect from digital health.”
“Through our collaboration with Omada, we’re making it simple and convenient for our customers to take control of their health and well-being. By harnessing the power of personal coaching, peer support, digital engagement and personal accountability, we can better inspire and support people to achieve their individual health goals and prevent chronic health conditions,” said Joan Harvey, Cigna’s Senior Vice president of Consumer Health Engagement and Behavioral Health. “We’re excited to deepen our relationship with Omada, and further integrate its innovative program into our full suite of data-driven health services.”
Omada has published 11 peer-reviewed studies, and is currently running the largest-ever randomized controlled trial of digital diabetes prevention. The company’s approach is proven to deliver lasting clinical outcomes, as well as significant medical cost savings, across diverse populations. Omada’s program drives unprecedented levels of meaningful engagement by participants, directly leading to sustained improvements in health.
Also participating in the round were previous investors Cigna Ventures, Andreessen Horowitz, U.S. Venture Partners, Norwest Venture Partners, Kaiser Permanente Ventures, Sanofi Ventures, Civilization Ventures, and Providence Ventures.
About Omada
Omada is a digital care program that empowers people to achieve their health goals through sustainable lifestyle change. Working primarily through health plans, employers, and integrated health systems, the company delivers personalized interventions for individuals at risk for, or dealing with, type 2 diabetes and hypertension, as well as anxiety and depression. Combining data-powered human coaching, connected devices, proprietary technology platform, and curriculum tailored to an individual's specific conditions and circumstances, Omada has enrolled more than 250,000 participants to date. Omada partners include Cigna, Kaiser Permanente, Health Care Services Corporation (HCSC), Blue Cross Blue Shield Minnesota, and other leading health plans. For additional information, please visit www.omadahealth.com .
-Multi-year collaboration leveraging Kymera’s proprietary targeted protein degradation platform to develop novel medicines-
-Kymera to receive $70 million upfront, including equity investment, and potential additional milestone and royalty payments for up to six programs in the collaboration-
BOSTON, MA and CAMBRIDGE, MA – May 15, 2019 – Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) and Kymera Therapeutics today announced that the two companies have entered into a four-year strategic research and development collaboration to advance small molecule protein degraders against multiple targets. The collaboration will leverage Kymera’s expertise in targeted protein degradation and its proprietary PegasusTM drug discovery platform and Vertex’s scientific, clinical, and regulatory capabilities to accelerate the development of first-in-class medicines for people with serious diseases.
“This collaboration with Kymera will enhance our drug discovery capabilities and support our strategy of investing in scientific innovation to develop transformative medicines for serious diseases,” said Mark Bunnage, Senior Vice President and Site Head, Boston Research at Vertex. “We’ve been impressed by the Kymera team’s depth of knowledge in the field and compelling technology platform, and are excited to bring our research and development expertise to this promising new therapeutic modality.”
“We are thrilled to partner with Vertex to combine their deep understanding of human biology and genetics with Kymera’s state-of-the art protein degrader platform,” said Laurent Audoly, PhD, president and CEO, Kymera Therapeutics. “This strategic partnership will broaden the application of targeted protein degradation to address serious diseases beyond cancer with limited or no treatment options. This fits perfectly with Kymera’s vision to build a platform that is disease agnostic and delivers the broadest possible impact.”
About the Collaboration
Under the terms of the four-year agreement, Vertex will pay Kymera $70 million upfront including an equity investment in the company. Kymera will conduct research activities in multiple targets under the collaboration. Upon designation of a clinical development candidate, Vertex has the option to exclusively license molecules against the designated target. Kymera is also eligible to receive more than $1 billion in potential payments based upon the successful achievement of specified research, development, regulatory, and commercial milestones for up to six programs optioned as part of the collaboration. In addition, Vertex will pay tiered royalties on future net sales on any products that may result from this collaboration.
About Kymera Therapeutics
Kymera Therapeutics is a biotechnology company pioneering a transformative new approach to treating previously untreatable diseases. The company is advancing the field of targeted protein degradation, accessing the body’s innate protein recycling machinery to degrade dysregulated, disease-causing proteins. Powered by Pegasus™, a game-changing integrated degradation platform, Kymera is accelerating drug discovery with an unmatched ability to target and degrade the most intractable of proteins, and advance new treatment options for patients. For more information visit, www.kymeratx.com.
About Pegasus™
Pegasus™ is Kymera Therapeutics’ proprietary protein degradation platform, created by its team of experienced drug hunters to improve the effectiveness of targeted protein degradation and generate a pipeline of novel therapeutics for previously undruggable diseases. The platform consists of informatics driven target identification, novel E3 ligases, proprietary ternary complex predictive modeling capabilities and degradation tools.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex’s headquarters is now located in Boston’s Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Latin America. Vertex is consistently recognized as one of the industry’s top places to work, including being named to Science magazine’s Top Employers in the life sciences ranking for nine years in a row.
For additional information and the latest updates from the company, please visit www.vrtx.com.
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Bunnage’s statements in the second paragraph of the press release, Dr. Audoly’s statements in the third paragraph of the press release, and statements regarding future activities of the parties pursuant to the collaboration. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent Vertex’s beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, Vertex may not realize the anticipated benefits of the collaboration, and the other risks listed under Risk Factors in Vertex’s annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company’s website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
Vertex Contacts:
Investors:
Michael Partridge, 617.341.6108
Eric Rojas, 617.961.7205
Zach Barber, 617.341.6470
Media:
617.341.6992
mediainfo@vrtx.com
Kymera Contact:
Lissette Steele
Verge Scientific Communications
lsteele@vergescientific.c
Sanofi, UCB, McKesson Ventures, and Horizon Health Services Endorse Aetion's Vision for Real-World Evidence
NEW YORK, Feb. 5, 2019 /PRNewswire/ -- Aetion, the health care technology company that delivers the real-world analytics and evidence platform needed to engage in value-based care, announced it has attracted strategic investment from global leaders in biopharma and health care services, including Sanofi, UCB, McKesson Ventures, and Horizon Health Services, Inc., a Blue Cross Blue Shield of New Jersey company — four new strategic shareholders that join Amgen Ventures, an earlier investor. Existing venture capital investors also participated in this round, including NEA, Flare Capital, and Lakestar.
The latest strategic capital infusion of $27 million reflects Aetion's substantialgrowth in customer adoption over the past year. Coupled with its prior funding in early 2018, the investment caps the total Series B funding at $63 million. This brings Aetion's total funding to $77 million since its 2015 launch. Investment funds will be applied to strengthen the Aetion Evidence Platform™, extending its reach in therapeutic area-related intelligence, outcomes-based contracting for payers, and advancing the acceptance of standards for real-world evidence around the globe.
"The time for real-world evidence is now," said Carolyn Magill, Chief Executive Officer of Aetion. "We're entering a new era in which nearly the entire health care ecosystem — from biopharma and regulators to payers and technology companies— recognizes the importance of using real-world evidence to reduce the time and cost to bring new therapies to market. This funding demonstrates that the industry's leaders, who are using our technology to drive health care's most critical decisions, view Aetion as a trusted partner vital to their long-term success."
For Aetion, strategic backing from a sweep of biopharma, health care services, and payer organizations with investment capital reinforces its position as an unbiased thought-leader at the forefront of industry adoption of real-world evidence, and underscores growing confidence among its partners and clients in the Aetion Evidence Platform as the analytics tool of record.
"We are at an inflection point, in which the convergence of massive analytical advances and enormous amounts of new data are transforming what's possible in health care," said Ameet Nathwani, M.D., Chief Medical Officer at Sanofi. "Aetion is ideally positioned to lead this shift, given the power of its technology and extensive experience with biopharma leaders and the FDA. Sanofi is excited to join as a strategic investor as the company helps set the global standards for real-world evidence."
"Aetion's abilities represent the leading edge of member-centric, value-based care," said Bill Georges, Chief Strategy Officer at Horizon Healthcare Services, Inc. "Horizon has successfully deployed Aetion's analytics and leveraged real-world evidence to determine which treatments are most effective for specific patient populations with diabetes. Horizon shares this data with our members' doctors to help them deliver more effective treatment regimens that work to improve outcomes for our members and reduce their overall cost of care. We are thrilled to be collaborating with Aetion as a strategic investor and excited to expand the number of Horizon members who will benefit directly from Aetion's innovation."
Aetion is coming off a year of strong growth, and this funding continues the company's momentum. Today, the Aetion Evidence Platform is used by eight of the top 15 global biopharma firms, leading payers, academic institutions, the U.S. Food and Drug Administration (FDA) and other major international regulatory bodies. Since 2017, it has seen a 100 percent customer renewal rate.
"Aetion shares our ambition to transform the future for people living with severe diseases, and our passion for science-driven approaches that serve this goal," said Iris Loew-Friedrich, M.D., Ph.D., chief medical officer at UCB. "Aetion's transparent
evidence serves as a bridge between UCB and its payers, enabling us to apply realworld evidence to engage them in new ways. We're thrilled to build upon our strong relationship with Aetion as a strategic investor."
Also in 2018, Aetion began a partnership with the FDA to recreate 30 randomized clinical trials through real-world evidence, to demonstrate its value as an accelerant to drug approval and access decisions. The three-year study, named DUPLICATE, is referenced in the FDA's recently released Framework for its Real-World Evidence Program, which applies across its drug and biologic review programs — yet another clear signal that real-world evidence will modernize how treatments are developed, approved, and commercialized.
"Aetion is poised to be the partner of choice for biopharma companies, payers and regulators looking to leverage real-world evidence given its unique relationships across the health care ecosystem and its expertise in protocols and treatment based analytics," said Tom Rodgers, SVP and Managing Director at McKesson Ventures.
To learn more, visit Aetion's website and subscribe to its newsletter at www.aetion.com.
About Aetion
Aetion is a health care technology company that delivers real-world evidence for life sciences, payers, providers, and regulatory agencies. The Aetion Evidence Platform analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on treatments, costs, and outcomes. Founded by Harvard Medical School faculty members with decades of experience in epidemiology and health outcomes research, Aetion informs health care's most critical decisions — what works best, for whom, and when — to guide treatment development, commercialization, and payment innovation into health care's modern era.
Aetion is based in New York City, and backed by investors including New Enterprise Associates (NEA), Flare Capital Partners, Lakestar, Town Hall Ventures, McKesson Ventures, Sanofi Ventures, Amgen Ventures, UCB, and Horizon Health Services, Inc. Learn more at aetion.com, and follow us at @aetioninc.
SOURCE Aetion
Related Links
https://www.aetion.com
Safety Data and Potential Clinical Efficacy Signal Expected Mid-2019
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Navitor Pharmaceuticals, Inc., the leader in the development of mTORC1-targeted therapeutics designed to help patients live longer and healthier lives, announced today the initiation of Part B of its Phase 1 clinical study with its lead candidate, NV-5138, for treatment-resistant depression (TRD). NV-5138 is a first-in-class, orally-active small molecule that directly activates mTORC1, the gatekeeper of cellular metabolism and renewal, which is suppressed in the brain of people suffering from depression.
“NV-5138’s direct activation of mTORC1 may deliver significant advantages over antidepressants that only indirectly activate this master metabolic control switch, offering the potential for rapid-acting antidepressant benefits without the psychotomimetic side effects and abuse potential observed with many N-methyl-D-aspartic-acid (NMDA) receptor targeted therapeutics,” said Thomas E. Hughes, Ph.D., Chief Executive Officer of Navitor. “The positive results from the single ascending dose portion of our Phase 1 clinical study of NV-5138 in healthy volunteers support advancement into Part B, and we are now evaluating a single dose of the compound in patients suffering with TRD. We look forward to the initial top-line data from this study in the middle of 2019.”
The Phase 1, multicenter, two-part, double-blind, placebo-controlled study is evaluating the safety, tolerability and pharmacokinetics of NV-5138 in up to 88 subjects, including healthy volunteers and patients diagnosed with TRD. In Part A, the single-ascending-dose portion of the study, up to 48 healthy volunteers were randomly assigned to double-blind treatment in six cohorts. In Part B of the study, approximately 40 subjects diagnosed with TRD are being randomized to receive either a single dose of NV-5138 or placebo. Secondary efficacy outcome measures for Part B include standard depression rating and symptomology scores such as the Montgomery-Åsberg Depression Rating Scale (MADRS).
“In multiple standard preclinical models of depressive behavior and cognition, we have shown that a single dose of NV-5138 stimulates mTORC1, enhances protein expression within hours, and increases synaptic growth in key, relevant brain regions, resulting in sustained antidepressant behavioral responses,” stated George P. Vlasuk, Ph.D., President and Chief Scientific Officer of Navitor. “These behavioral changes and increases in synaptogenesis were consistent with the effects of NMDA receptor modulators such as ketamine; however, NV-5138 works through direct, post-synaptic activation of the mTORC1 signaling pathway and may therefore offer the potential for an improved safety and tolerability profile. Part B of our Phase 1 trial will offer important insights on the candidate’s potential in this difficult-to-treat patient population.”
About NV-5138
NV-5138 is an orally bioavailable, small molecule that directly and transiently activates mTORC1, the master modulator of cellular metabolism, which is often suppressed in the brain of patients suffering from depression. NV-5138 binds to and modulates sestrin, a newly discovered cellular sensor protein for the amino acid leucine, a potent natural activator of mTORC1. As opposed to many other organ systems like skeletal muscle, leucine is a poor activator of mTORC1 in the brain since it is principally used as a metabolic precursor for neurotransmitter and protein synthesis. NV-5138 was designed to avoid the metabolic fate of leucine in the brain and thus serves as an effective activator of mTORC1 in this tissue. Results from preclinical models demonstrate that oral administration of NV-5138 produces rapid upregulation of key synaptic proteins, synaptic remodeling in the prefrontal cortex and hippocampus, sustained antidepressant behavioral responses, cognitive improvements and compound-specific spectral power changes, as measured by quantitative electroencephalography (qEEG). Navitor’s strong intellectual property portfolio includes composition of matter patent protection for NV-5138 and related compounds.
About mTORC1
mTORC1, or Complex 1 of the mechanistic target of rapamycin, activity governs the pace and ability of the cell to synthesize protein and other cellular components. Increased mTORC1 activity contributes to a broad array of diseases of aging by increasing protein misfolding and driving cellular stress, inflammation, and fibrosis. In other disease states such as severe depression, inadequate mTORC1 activity contributes to disease pathology by limiting energy utilization and protein synthesis, leading to impaired function. Multiple preclinical studies have shown that mTORC1 activation is required for the efficacy of many rapid-acting antidepressant compounds, including but not limited to modulators of the N-methyl-D-aspartic-acid (NMDA)-mediated signaling pathway like ketamine.1
About Navitor
Navitor Pharmaceuticals, Inc. is the leader in the development of mTORC1-targeted therapeutics designed to help patients live longer and healthier lives. The Company’s proprietary platform enables true modulation of mTORC1, the gatekeeper of cellular metabolism and renewal, with the first-ever absolutely selective mTORC1 inhibition and the unique ability for mTORC1 activation. Navitor’s lead clinical-stage candidate, NV-5138, is a small molecule that directly activates mTORC1 and is being developed for treatment-resistant depression, with additional opportunities in cognition and memory. The Company’s NΛValog program, which provides unprecedented selectivity in mTORC1 inhibition, is initially targeting chronic kidney disease and has broad potential application for age-related diseases. For more information, please visit www.navitorpharma.com.
Approach combines Otsuka’s expertise in developing and commercializing treatments for mental health with Click’s record of discovering and validating digital technologies as prescription medical treatments
SAN FRANCISCO, C.A. and NEW YORK, N.Y. – January 3, 2019 – Otsuka America, Inc., and Click Therapeutics, Inc., announce today that the companies have signed a collaboration agreement to develop and commercialize a prescription digital therapeutic for treatment of Major Depressive Disorder (MDD), with the intent to address unmet medical needs among this patient population and to improve outcomes.
This collaboration will leverage Click’s demonstrated ability to discover and validate a software application and deploy it commercially, with Otsuka’s expertise in developing approved prescription therapies for patients with serious mental illnesses, including Otsuka’s established development and commercialization capabilities. The companies believe digital therapeutics align naturally with psychiatry and have significant potential to transform mental healthcare. Together, the companies aim to bring to market a new offering that will provide a novel treatment for patients with MDD.
Otsuka has agreed to commit capital to fully fund development of Click’s novel mobile application ‘CT-152’ for MDD, and to commercialize this application world-wide upon achievement of regulatory approvals. Otsuka will pay Click up to $10 million in upfront and regulatory milestone payments, along with an estimated $20 million in development funding. An additional $272 million in commercial milestone payments are contingent upon regulatory approvals. In addition, Click will receive tiered, double-digit royalties on global sales of the software and the digital therapeutic applications that result.
“This collaboration signals Otsuka’s commitment to meet patients’ unmet medical needs by developing solutions far beyond medication. Our goal is to deliver evidence-based cognitive therapies to a broader population of patients with MDD than is currently feasible, due to the challenges of a shortage of mental health professionals and limited time for them to conduct cognitive therapy,” said Kabir Nath, president and CEO, Otsuka North America Pharmaceutical Business Division, Otsuka America, Inc. “We are proud to be one of the few pharmaceutical companies that continues to invest in developing medicinal and digital products for the treatment of mental illnesses, and we are doing so by breaking down barriers and collaborating with leading therapeutic technology companies, such as Click, which share our vision.”
According to the World Health Organization, depression is the leading cause of disability worldwide, and is a major contributor to the overall global burden of disease.1 Otsuka and Click believe that new approaches are needed to address this condition, including pioneering ways to use technology and data in support of better patient outcomes.
“Otsuka has established itself as the leading innovator in digital medicine for psychiatry, and we are thrilled to collaborate with their clinical and commercial experts on our CT-152 digital therapeutic for the treatment of depression. This collaboration symbolizes the growing recognition that digital therapeutics are a new category of treatment with the potential to become a routine treatment option for physicians for their patients,” said David Benshoof Klein, Chairman and CEO of Click. “The potential value of these treatments is clear: they provide validated tools that may be an effective treatment option to bring the power and accessibility of digital technologies for the benefit of the patient and physician. This recognition is also shared by regulators helping to build new pre- market review programs uniquely suited for digital therapeutics, while still retaining the traditional level of rigor and scrutiny that clinical studies require.”
About the prescription digital therapeutic for MDD and the agreement between Otsuka and Click:
- CT-152 is a software application (app) that will leverage evidence-based cognitive therapy principles and Click’s patient engagement platform to treat patients either independently or in conjunction with prescribed pharmacotherapies.
- The intent is that the app will be classified as Software as a Medical Device (SaMD) and will fall under the FDA regulatory framework that supports innovation and commercialization of digital tools while protecting patient health.
About Otsuka Pharmaceutical
Otsuka Pharmaceutical is a global healthcare company with the corporate philosophy: “Otsuka-people creating new products for better health worldwide.” Otsuka researches, develops, manufactures and markets innovative products, with a focus on pharmaceutical products for the treatment of diseases and nutraceutical products for the maintenance of everyday health.
In pharmaceuticals, Otsuka is a leader in the challenging area of mental health and has research programs on several under-addressed diseases including tuberculosis, a significant global public health issue. These commitments illustrate how Otsuka is a “big venture” company at heart, applying a youthful spirit of creativity in everything it does.
Otsuka America, Inc. is a subsidiary of Otsuka Pharmaceutical Co., Ltd. headquartered in Tokyo, Japan. Both are part of Otsuka Holdings Co., Ltd. The Otsuka group of companies employed 46,000 people worldwide and had consolidated sales of approximately USD 11.1 billion in 2017.
All Otsuka stories start by taking the road less travelled. Learn more about Otsuka Pharmaceutical Company on its global website at https://www.otsuka.co.jp/en. Learn more about Otsuka in the U.S. at www.otsuka-us.com. Connect with us in the U.S. on Twitter at @OtsukaUS.
About Click Therapeutics
Click Therapeutics, Inc. develops and commercializes software as prescription medical treatments for people with unmet medical needs. Through cognitive and neurobehavioral mechanisms, Click’s Digital Therapeutics™ enable change within individuals, and are designed to be used independently or in conjunction with biomedical treatments. The Clickometrics® adaptive data science platform continuously personalizes user experience to optimize engagement and outcomes. Following a groundbreaking clinical trial, Click’s industry-leading smoking cessation program is available nationwide in the U.S through a wide variety of payers, providers, and employers. Click’s lead prescription program is entering into a multi-center, randomized, controlled, parallel-group, phase III FDA
December 2018 01US18EUC0370
December 2018 01US18EUC0370
registration trial for the treatment of Major Depressive Disorder in adults. For more information, visit ClickTherapeutics.com.
References:
1. World Health Organization. Depression Fact Sheet. Available at: www.who.int/news-room/fact- sheets/detail/depression
Media Contacts
Otsuka In U.S.
Robert Murphy
Corporate Communications
Otsuka America Pharmaceutical, Inc.
robert.murphy@otsuka-us.com
+1 609 249 7262
Otsuka in Japan
Jeffrey Gilbert (Outside the U.S.)
Leader, Pharmaceutical PR
Otsuka Pharmaceutical, Co., Ltd.
gilbert.jeffrey@otsuka.co.jp
+81 3 6361 7379, +81 80 8728 6039
Click Therapeutics
Contact Karen Sharma
ksharma@macbiocom.com
+1 781 235 3060
Series B financing led by 6 Dimensions Capital, Bessemer Venture Partners, and Pfizer Ventures
Financing to enable the advancement of lead program into clinical development and support the progression of additional programs
Cambridge, Mass. (Nov. 13, 2018) – Kymera Therapeutics Inc., a biotechnology company pioneering targeted protein degradation to create breakthrough medicines for patients, announced today that it has completed a $65 million Series B financing. The financing will support the advancement of its lead asset into clinical development, and to progress its therapeutic pipeline in oncology and immunology. The financing was co-led by 6 Dimensions Capital, Bessemer Venture Partners, and Pfizer Ventures with participation by MRL Ventures Fund, Sanofi Ventures, Hatteras Venture Partners, and Aju IB Investment, in addition to Kymera’s Series A investors. The company also announced Wei Li, PhD, of 6 Dimensions Capital, Andrew Hedin of Bessemer Venture Partners, and Elaine Jones, PhD, of Pfizer Ventures, will join Kymera Therapeutics’ Board of Directors.
“We are delighted to have the support of this exceptional group of both new and existing investors and experts that share our vision and excitement on our progress to date,” said Laurent Audoly, PhD, president and CEO, Kymera Therapeutics. “In the last year, we’ve made great strides in building preclinical data packages supporting both the drug-like properties of our assets and their differentiated pharmacology. This rapid advancement has been enabled by our focused investment in Kymera’s industry-leading protein degradation platform and a stellar team of drug hunters and seasoned executives.”
Since announcing its founding in 2016, Kymera Therapeutics has progressed its proprietary Pegasus™ integrated degradation platform, consisting of informatics-driven target identification coupled to ternary complex predictive modeling, new degradation tools, and novel E3 ligases and ligands. Kymera also entered into a two-year discovery collaboration agreement with GSK, leveraging innovations in small molecule-based targeted protein degradation and encoded library technologies.
“This year has been marked by the maturation of our pipeline with applications across diverse target types and disease indications as well as a deeper understanding of the pharmaceutical properties required to develop protein degrading drugs,” said Nello Mainolfi, PhD, co-founder and chief technology officer, Kymera Therapeutics. “These insights have allowed us to refine our drug discovery platform and improve our ability to identify first-in-class drug candidates.”
“Kymera is truly at the forefront of targeted protein degradation, one of the most exciting new areas of drug discovery and medicine,” said Bruce Booth, DPhil, Kymera co-founder, partner at Atlas Venture, and chairman of the board. “With an incredible team, advisors and syndicate and a singular focus on rational drug discovery, the company has quickly emerged as a leader in the field and is well-positioned to realize the potential of this transformational modality to impact patients.”
About Kymera Therapeutics
Kymera Therapeutics is a biotechnology company pioneering a transformative new approach to treating previously untreatable diseases. The company is advancing the field of targeted protein degradation, accessing the body’s innate protein recycling machinery to degrade dysregulated, disease-causing proteins. Powered by Pegasus™, a gamechanging integrated degradation platform, Kymera is accelerating drug discovery with an unmatched ability to target and degrade the most intractable of proteins, and advance new treatment options for patients. For more information visit, www.kymeratx.com.
About Pegasus™
Pegasus™ is Kymera Therapeutics’ proprietary protein degradation platform, created by its team of experienced drug hunters to improve the effectiveness of targeted protein degradation and generate a pipeline of novel therapeutics for previously undruggable diseases. The platform consists of informatics driven target identification, novel E3 ligases, proprietary ternary complex predictive modeling capabilities and degradation tools.
About 6 Dimensions Capital
6 Dimensions Capital is a leading healthcare focused investment firm with an in-depth focus and extensive coverage across China and the United States. It consists of a team of about 40 investment and operation professionals with offices in Shanghai, Hong Kong, Boston, and Palo Alto. The firm currently has US$1.6 billion assets under management through 4 US dollar-denominated and 3 RMB-denominated funds and has cultivated a portfolio of more than 80 companies. For more information, visit www.6dimensionscapital.com.
About Pfizer Ventures
Pfizer Ventures (PV), the venture capital arm of Pfizer Inc. was founded in 2004 and invests for return in areas of current or future strategic interest to Pfizer. PV seeks to remain at the forefront of life science advances, looking to identify and invest in emerging companies that are developing transformative medicines and technologies that have the potential to enhance Pfizer’s pipeline and shape the future of our industry. For more information, visit www.pfizerventures.com.
About Bessemer Venture Partners
Bessemer Venture Partners is the world’s most experienced early-stage venture capital firm, with a portfolio of more than 200 companies, including Pinterest, Betterment, Smule, Rocket Lab, Procore, PagerDuty, Intercom, Fiverr, ServiceTitan, Toast, Allscripts, and Bright Health. Bessemer partners early with visionary entrepreneurs and supports them throughout every stage of their growth, primarily focusing on consumer, enterprise, healthcare, and frontier technology companies. The firm has backed more than 120 IPOs, including Shopify, Yelp, LinkedIn, Skype, OvaScience, LifeLock, Twilio, SendGrid, DocuSign, Wix, and MindBody. Bessemer’s 14 partners operate from offices in Silicon Valley, San Francisco, New York City, Boston, Israel, and India. For more information, visit www.bvp.com.
Contact:
Lissette Steele
Verge Scientific Communications
202.930.4762
lsteele@vergescientific.c
NEW YORK, Oct. 22, 2018 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc.(NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced it has received the 2018 CDKL5 Forum Company Making a Difference Award for initiation of the Phase 2 ARCADE trial with OV935/TAK- 935, and its commitment to the CDKL5 deficiency disorder (CDD) patient community. The award was announced today in London, UK at the CDKL5 Forum, the largest annual conference on the advancement of science and therapeutic development for CDD.
CDD is a rare, severe, neurological disorder that causes frequent, treatmentresistent seizures in the first few months of life. CDD results in a constellation of severe challenges, including developmental delay and intellectual disability, movement disorder, difficulty sleeping, scoliosis, visual impairment, microcephaly and various gastrointestinal difficulties. There are currently no FDA-approved therapies for CDD.
“We hand out the Company Making a Difference Award annually to recognize industry partners for their commitment and support, and this year we are pleased to recognize the team at Ovid Therapeutics,” said Ana Mingorance, Ph.D., chief development officer of Loulou Foundation. “Since the day Ovid selected CDD as a condition to pursue, they have shown amazing support and care for our community, and we are excited to partner with such a humancentric organization.”
The ARCADE trial is a Phase 2 multi-center, open-label, pilot study designed to evaluate the treatment of OV935 in pediatric patients with epileptic seizures associated with CDD or Duplication 15q (Dup15q) syndrome. The first patients have already been enrolled into ARCADE. This study is part of a global collaboration with Takeda Pharmaceutical Company Limited.
“We are honored to be recognized for our dedication and efforts to bring a novel treatment option to the clinic for those with CDD and other rare epilepsies,” said Amit Rakhit, M.D., MBA, chief medical and portfolio management officer of Ovid Therapeutics. “It was important to everyone at Ovid that I travel to London to accept this award to show our unwavering support and partnership with the CDD community. We thank LouLou Foundation and the community and look forward to providing updates on our progress with the ARCADE study.”
The CDKL5 Forum is a unique community of collaboration and knowledge exchange, made up of leading scientists, clinicians and company representatives from around the world, united in the mission of better understanding the CDKL5 gene and disorder. The objective is to share current research on CDKL5 and to stimulate peer-group discussion and brainstorming around existing and future avenues of research and therapeutic approaches, in order to accelerate treatments and ultimately find cures for this neuro-genetic disorder. Now in its fourth year, the Forum represents the flagship annual event of the CDKL5 Program of Excellence, established by the Loulou Foundation and the Orphan Disease Center of the University of Pennsylvania’s Perelman School of Medicine.
About the ARCADE Trial
ARCADE is a Phase 2, multi-center, open-label, pilot study that will evaluate the treatment of OV935 in pediatric patients, aged 2 to 17 years old, with epileptic seizures associated with CDD or Dup15q syndrome. The primary endpoint is the change in motor seizure frequency in patients treated with OV935 by disorder (CDD and Dup15q). The key secondary endpoints include safety and tolerability, including percentage of participants considered treatment responders, change in CGI-S/C and correlation of OV935 concentration and plasma 24HC levels.
ARCADE is expected to enroll approximately 15 children with each condition at clinical trial sites in the United States. This study consists of a four to six week screening period to establish baseline seizure frequency followed by a 12-week treatment period (2-week dose titration and 10-week maintenance period.) To learn more about ARCADE visit clinicaltrials.gov or www.arcadestudy.com. At the end of treatment, eligible patients can roll over into the ENDYMION study. Additional details on the ENDYMION clinical trial can be found at www.clinicaltrials.gov.
About Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (CDD)
Cyclin-Dependent Kinase-Like 5 (CDKL5) deficiency disorder, also known as CDD, is a rare, severe, neurological disorder caused by mutations in the CDKL5 gene on the X-chromosome. The CDKL5 gene provides instructions for making a protein that is essential for normal brain and neuron development, and may play a role in regulating the activity of other genes. CDD causes early onset and treatment resistant epilepsy in the first few months of life. Other common features of CDD include severe developmental delay and intellectual disability, poor fine motor skills, difficulty sleeping, scoliosis, visual impairment, microcephaly and various gastrointestinal difficulties.
About Duplication 15q (Dup15q) Syndrome
Duplication 15q syndrome, also known as Dup15q syndrome, is a rare, severe, neurological disorder that results from duplications of chromosome 15q11.2-q13.1. In most cases, the chromosome mutation is not inherited but occurs during formation of reproductive cells or during embryonic development. Those with Dup15q syndrome experience seizures, hypotonia (poor muscle tone), developmental delays and intellectual disability. Difficult to control seizures are the most devastating symptom of Dup15qii. The severity of Dup15q and associated symptoms varies based on the size and location of the duplication and which genes are involved. There is insufficient demographic data to determine the prevalence of Dup15q in the general population.
About Investigational OV935/TAK-935
OV935/TAK-935 is a potent, highly-selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H) being investigated as an anti-epileptic drug (AED). CH24H is predominantly expressed in the brain, where it plays a central role in cholesterol homeostasis. CH24H converts cholesterol to 24-hydroxycholesterol (24HC), which then exits the brain into the blood plasma circulation. Glutamate is one of the main neurotransmitters in the brain and has been shown to play a role in the initiation and spread of seizure activity. Recent literature indicates CH24H is involved in over-activation of the glutamatergic pathway through modulation of the NMDA channel, implying its potential role in central nervous system diseases such as epilepsy. Ovid and Takeda believe that OV935’s novel mechanism of action may potentially treat rare epilepsies by inhibiting CH24H to decrease 24HC levels, effectively decreasing glutamate hyperactivity. To Ovid and Takeda’s knowledge, OV935 is the only molecule with this mechanism of action in clinical development. OV935 is an investigational drug, not approved for commercial use.
OV935 has successfully completed four Phase 1 clinical studies, which have assessed tolerability, PK and target engagement at doses believed to be therapeutically relevant. In preclinical models, a novel proprietary PET ligand was used to determine target occupancy of OV935 in the brain. OV935 is being co-developed by Ovid and Takeda Pharmaceutical Company Limited.
The United States Food and Drug Administration (FDA) has granted orphan drug designation to OV935 for the treatment of both Dravet syndrome and LGS.
About Ovid Therapeutics
Ovid Therapeutics (NASDAQ: OVID) is a New York-based biopharmaceutical company using its BoldMedicine™ approach to develop medicines that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of potential first-in-class medicines. The company’s lead investigational medicine, OV101, is currently in development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935/TAK-935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of rare developmental and epileptic encephalopathies (DEE).
For more information on Ovid, please visit http://www.ovidrx.com/.
Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding the potential clinical benefit of OV935 to treat patients with rare epilepsies, number of patients enrolled, the initiation, progress, timing, scope and results of clinical trials, and the effects of OV935 on efficacy, safety and tolerability. You can identify forward-looking statements because they contain words such as “will,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission under the caption “Risk Factors”. Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
Contacts:
Patient advocacy:
Luke Rosen
Ovid Therapeutics Inc
lrosen@ovidrx.com
Media:
Jerica Pitts
W2O Group
jpitts@w2ogroup.com
(312) 858-3469
Ovid Therapeutics Inc.
New capital will enable Evidation Health to further enhance platform capabilities that quantify real life patient outcomes at scale
SAN MATEO, Calif.--(BUSINESS WIRE)--Today Evidation Health announced that it has closed a $10 Million funding round led by Sanofi-Genzyme BioVentures—the corporate venture capital arm of Sanofi, a global healthcare company that discovers, develops and distributes therapeutic solutions focused on patients' needs. Existing investors GE Ventures and B Capital Group also participated in the round.
The new investment will enable Evidation Health to further enhance its capabilities in large scale behavioral analytics, health outcomes measurement, and digital biomarker development using real life patient data from hundreds of applications and devices.
“At Evidation Health, we focus on measuring behavior-driven outcomes to help our partners understand and maximize product impact in the real world,” says Deborah Kilpatrick, CEO of Evidation Health. “We are excited about the Sanofi investment, as it aligns to our creation of faster, better ways to quantify patient outcomes—which we believe will directly fuel innovation across healthcare and life sciences.”
Evidation Health combines expertise from consumer technology, data science, outcomes research, and healthcare economics to measure product impact at entirely new levels of scale. By identifying what truly impacts patient outcomes outside of clinic walls, the value of all sorts of interventions can be quantified in the digital era of medicine.
“With the transition to value-based care, it is increasingly important for pharma companies to understand realworld behavior of individual patients and populations outside of the traditional clinical trial setting,” says Bernard Davitian, Vice President and Managing Director of Sanofi-Genzyme BioVentures. “Evidation Health’s unique capabilities in behavior analytics and integrated patient data are big enablers of this understanding. We’ve been impressed with both the Evidation team and platform, and we believe that they have the potential to transform the way pharma companies interact with patients to deliver better outcomes.”
About Evidation Health
Evidation Health helps healthcare companies quantify outcomes in the digital era, with real life data from connected patients. The company developed its Real Life Study Platform to accelerate and enhance outcomes research through virtualized pragmatic trials at scale—quantifying the impact of digital and traditional interventions far more efficiently than conventional approaches. Evidation Health works across the healthcare ecosystem with top pharmaceutical companies, payers, providers, and digital health companies. Evidation
Health is a privately held company headquartered in San Mateo, CA. For more information, visit www.evidation.com.
About Sanofi-Genzyme BioVentures
Sanofi-Genzyme BioVentures (SGBV) is the corporate venture capital arm of Sanofi. SGBV invests in early stage companies developing innovative products or technologies of interest to Sanofi. Today, SGBV has assembled a portfolio of direct equity investments in a variety of promising innovative life-science and digital health companies. SGBV is an important component of Sanofi's broader global strategy to invigorate external innovation. For more information, visit: http://sanofigenzymebioventures.com/
-Complete Responses Observed in Three of Six Patients with relapsed or refractory CD20-positive B cell non-Hodgkin Lymphoma at Dose Level 1 of the ATTCK-20-03 Phase I Study -
- No SAEs of Cytokine Release Syndrome or Neurotoxicity Observed in Dose Level 1 -
- Study Enrollment Continues -
-Management to host conference call today at 8:30 a.m. EDT-
CAMBRIDGE, Mass., Sept. 17, 2018 (GLOBE NEWSWIRE) -- Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies based on its novel, universal Antibody-Coupled T cell Receptor (ACTR) technology platform, today announced that the Company will be presenting data on the first dose level (40x106 ACTR+ T cells) of its ATTCK-20-03 clinical trial evaluating ACTR707 in combination with rituximab in patients with relapsed or refractory CD20-positive B cell non-Hodgkin Lymphoma (r/r NHL). Three of the six patients treated at the first dose level achieved a complete response, two of which remained ongoing at the time of the most recent data cut off. No dose-limiting toxicities (DLTs) were observed in any of the four DLT-evaluable patients, and no serious or severe adverse events of cytokine release syndrome or neurotoxicity were observed in any patients. These data will be presented at the Fourth Annual CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference on September 30 in New York, New York.
“These preliminary clinical data suggest that complete responses may be achieved without cytokine release syndrome, further validating the potential of our proprietary ACTR technology platform,” said Chuck Wilson, Chief Executive Officer of Unum. “The ATTCK-20-03 trial is a key element of our strategy to develop novel therapeutics for patients with no available treatment options.”
“Complete responses observed in this heavily pre-treated patient population at the first dose level of the ATTCK-20-03 trial support the potential potency of ACTR T cells for these patients,” said Michael Vasconcelles, Chief Medical Officer of Unum. “Based upon this encouraging preliminary profile, we look forward to continuing the dose escalation phase of this study. We expect these and future data to support selection of an ACTR product candidate to progress into potential registration trials in patients with r/r NHL.”
Enrollment and ACTR707 dosing in the second dose cohort (60x106 ACTR+ T cells) of the ATTCK-20-03 trial has been completed and dose escalation is proceeding. The Company expects to present additional data from this study later this year.
Safety and Preliminary Efficacy of ACTR707, Autologous T Lymphocytes Expressing an Antibody-Coupled T Cell Receptor, in Combination with Rituximab in Subjects with Relapsed or Refractory CD20-Positive B-cell Lymphoma (Abstract #A003)
Presenter: Dr. Veronika Bachanova, University of Minnesota
Date: Sunday, September 30, 2018, 11:45am – 2:15pm
Location: New York Marriott Marquis, Westside Ballroom
ATTCK-20-03 is a Phase I, multi-center, open label, single arm clinical trial evaluating ACTR707 in combination with rituximab in patients with r/r NHL. Eligible patients for enrollment must have, among other criteria, received adequate prior anti-lymphoma therapy, including rituximab and chemotherapy, for their CD20-positive r/r NHL. Key eligibility criteria include: pre-specified eligible NHL subtypes, including DLBCL, disease progression following immediate prior therapy, adequate organ function and performance status, and measurable disease. The trial design includes a dose escalation phase using an adaptive design, followed by a cohort expansion phase. Primary study objectives are to characterize the safety of ACTR707 in combination with rituximab and to determine the maximum tolerated dose and proposed recommended Phase 2 dose. Secondary study objectives include: assessment of the anti-lymphoma activity of the combination, ACTR707 persistence, rituximab pharmacokinetics, and inflammatory markers and cytokine levels. Following leukapheresis, each patient receives lymphodepletion followed by the first infusion of rituximab and then a single infusion of ACTR707. Rituximab infusions continue on a regular, pre-specified schedule.
Data in the first cohort of the trial demonstrate complete responses at the first response assessment in 3/6 patients treated with ACTR707 in combination with rituximab, two of which remained ongoing at the time of the most recent data cut off. There were no serious or severe adverse events of cytokine release syndrome, neurotoxicity, or autoimmune events. Grade 3 or higher adverse events were mostly hematologic including neutropenia (n=2), febrile neutropenia (n=2), and thrombocytopenia (n=1). ACTR+ T cells were detectable in all subjects and ACTR+ T cells persisted in the presence of continued rituximab administration. These results support the continued dose escalation of ACTR707 in combination with rituximab.
Conference Call and Webcast
Unum will host a conference call and webcast at 8:30 a.m. EDT today to discuss the data. To participate in the conference call, please dial (866) 300-3411 (domestic) or (636) 812-6658 (international) and enter the conference code: 2958105. To join the live webcast, please visit the investor relations section of the Unum Therapeutics website at https://investors.unumrx.com/ at least 10 minutes before the event begins. A webcast replay will be available at the same location on the Unum Therapeutics website beginning approximately two hours after the event and will be archived for 90 days.
About ACTR707
ACTR707 is an investigational drug that may represent an important construct not only for adult patients with CD20+ r/r NHL, when used in combination with rituximab, but also for patients with other cancer types when used in combination with other antibodies. ACTR707 was identified through a comprehensive high-throughput screening effort aimed at identifying receptors with improved functional characteristics across several dimensions. In preclinical testing, ACTR707 demonstrated potent activity against a wide range of hematologic and solid tumor cancers. Given the challenges of the immunosuppressive solid tumor microenvironment, Unum believes that ACTR707’s increased activity may be particularly important in addressing solid tumor cancers. ACTR707 is currently being tested in combination with rituximab in patients with r/r NHL in a Phase I multi-center open label clinical trial, ATTCK-20-03. Testing is expected to be initiated later in 2018 in ATTCK-34-01, a Phase I multi-center open label clinical trial exploring the combination of ACTR707 with trastuzumab in patients with HER2+ advanced cancers.
About Unum Therapeutics
Unum Therapeutics is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel immunotherapy products designed to harness the power of a patient’s immune system to cure cancer. Unum’s novel proprietary technology, antibody-coupled T cell receptor (ACTR) is a universal, engineered cell therapy intended to be used in combination with a wide range of tumor-specific antibodies to target different tumor types. ACTR087 and ACTR707, each used in combination with rituximab, an anti-CD20 antibody, are Unum’s two most advanced product candidates, currently in Phase I clinical testing in adult patients with relapsed or refractory non-Hodgkin lymphoma (r/r NHL). The Company has an additional product candidate in Phase I clinical testing: ACTR087 used in combination with the novel antibody SEA-BCMA in adult patients with relapsed or refractory multiple myeloma. Finally, the Company has an active investigational new drug application (IND) for ACTR707 used in combination with trastuzumab, an anti-human epidermal growth factor receptor 2 (HER2) antibody, to treat patients with HER2+ advanced cancer. This Phase I trial is expected to be initiated by the end of 2018.
The Company is headquartered in Cambridge, MA.
Forward looking Statements
This press release contains forward-looking statements. Statements in this press release about our future expectations, plans and prospects, our long-term growth, the anticipated timing of our clinical trials and regulatory filings, the development of our product candidates, including the four lead ACTR product candidates, as well as other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar expressions, constitute forward-looking statements within the meaning of the safe harbor provisions of The Private Securities Litigation Reform Act of 1995. We may not actually achieve the forecasts disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results could differ materially from the projections disclosed in the forward-looking statements we make as a result of a variety of risks and uncertainties, including risks related to the accuracy of our estimates regarding expenses, future revenues, capital requirements, and the need for additional financing, the success, cost and timing of our product development activities and clinical trials, our ability to obtain and maintain regulatory approval for our product candidates, and the other risks and uncertainties described in the "Risk Factors" sections of our public filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent our views as of the date hereof. We anticipate that subsequent events and developments may cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date hereof.
Investor Contact:
Stephanie Ascher, 212-362-1200
stephanie@sternir.com
Media Contact:
Paul Kidwell, 617-680-1088
paul.kidwell@unumrx.com
Proceeds will help advance lead program in facioscapulohumeral muscular dystrophy (FSHD) and further support advancement of rare disease-focused pipeline
Cambridge, Mass., September 5, 2018 – Fulcrum Therapeutics, a company focused on discovering and developing small molecule therapies to unlock gene control and treat serious genetic diseases, today announced the closing of an $80 million Series B financing. Proceeds from the financing will be used to advance Fulcrum’s lead program in facioscapulohumeral muscular dystrophy (FSHD) into clinical testing, and to progress its pipeline of therapeutics for rare, genetically based neuromuscular, central nervous system and hematologic disorders.
The financing was led by Foresite Capital, with participation by [Fidelity Management and Research Company, 6 Dimensions Capital, Casdin Capital, Sanofi Ventures, Section 32, NS Investments, entities affiliated with Leerink Partners, and undisclosed institutional investors. Jim Tananbaum, M.D., of Foresite Capital will join the Board of Directors of Fulcrum.
“We are delighted to have the support of this exceptional group of investors as we continue to work towards our vision of bringing new futures to patients and families affected by debilitating genetic diseases,” said Robert J. Gould, Ph.D., Fulcrum’s President and Chief Executive Officer. “This funding will provide crucial support as we move rapidly towards the clinic with our lead drug candidate in FSHD, and further advance our pipeline of small molecule therapies.”
Fulcrum is pioneering a small molecule, precision medicine approach to address severe monogenic and prevalent diseases of gene misregulation. By focusing on disease-causative genes, the company is unlocking the druggable mechanisms that regulate disease to develop a new generation of therapies in multiple therapeutic areas. In partnership with the FSHD Clinical Trial Research Network (CTRN), the company has initiated a clinical trial readiness study in FSHD. The aim of the study is to standardize a set of tools and measurements for Fulcrum’s future clinical drug trials.
“Fulcrum’s uniquely patient-centered approach to treating severe genetic diseases is already delivering strong early results,” said Dr. Tananbaum. “We are confident that Fulcrum will continue to lead the way in developing novel medicines for patients and families who have no viable treatment options, and we look forward to supporting the team as it works to further advance its rare-disease focused pipeline.”
About Fulcrum Therapeutics
Fulcrum Therapeutics is a biotechnology company developing new medicines to deliver a new future to patients and their families by transforming gene regulation in disease. Fulcrum’s therapies are based on modulating gene regulation via control of genetic on and off switches of disease genes. Fulcrum, headquartered in Cambridge, Mass., was launched by Third Rock Ventures in 2016 and named a “Fierce 15” company later that year. For more information, please visit www.fulcrumtx.com.
CONTACTS:
Media: Sarah Sutton
Ten Bridge Communications
sarah@tenbridgecommunications.com
518-932-3680
T1D Sleep Pilot to study the relationship between low blood sugar at night and daily behavior, as measured by smartphones and connected sensors from wearables and medical devices
SAN MATEO, CALIF., August 23, 2018 – Evidation Health, a health and measurement company that helps life sciences and health care companies understand how everyday behaviors and health interact, announced today a new technology partnership with Tidepool, an open source, not-for-profit company focused on making diabetes data more accessible, actionable and meaningful for people with diabetes, their care teams and researchers.
Under the partnership, Evidation and Tidepool are joining together on a new research study, called the T1D Sleep Pilot, to capture and study real world data from people with type 1 diabetes. The research will develop insights from data generated by continuous glucose monitors and insulin pumps, in addition to sleep and activity trackers from smartphones and other connected sensors.
The observational study will explore linkages between nocturnal hypoglycemia, next-day behavior, sleep patterns, and heart rates in order to contribute to ongoing research on how everyday behavior data and diabetes complications interact.
“Linking real world data from connected devices with other medical data in virtual studies allow us to measure how behaviors — outside of the doctor’s office or hospital — affect health and impact outcomes,” said Evidation Health CEO Deborah Kilpatrick, Ph.D. “Tidepool has done a fantastic job of giving people with diabetes access to their own data, and this effort provides a new opportunity to share everyday data with researchers at scale and enable more people to contribute to diabetes innovation.”
The partnership brings together Evidation’s data platform, which analyzes and processes large-scale sensor and behavior data in clinically meaningful ways, with Tidepool’s device-agnostic consumer software. Tidepool joins a growing list of more than 100 individually-permissioned data sources that are linked to Evidation’s platform, including Apple Health, Blue Button, Dexcom, Epic and Fitbit.
“People with diabetes can use their individual data to play a key role in improving health,” said Howard Look, president, CEO, and founder of Tidepool. “Our study with Evidation gives people with diabetes a new way to share their data with researchers, and contribute to a better understanding of dangerous low-blood sugar levels, which can often occur more frequently while sleeping.”
Since 2012, Evidation Health has built the most diverse virtual pool of research participants through its proprietary app, Achievement. With more than 2 million individuals using the app, this cohort is unparalleled in clinical research today.
People with type 1 diabetes can learn more about joining the T1D Sleep Pilot at http://study.myachievement.com/t1dsleep.
About Evidation
Evidation Health is a new kind of health and measurement company that provides the world’s most innovative life sciences and health care companies the technology and expertise they need to understand how everyday behavior and health interact. The volume of behavior data generated from smartphones and connected sensors — including wearables and medical devices — has opened up new ways to analyze individuals’ behavior and health in real time, unlocking insights into what medicines and treatments work best and spotting significant changes in health earlier. The scale and utility of everyday behavior data has the potential to be one of the most transformative forces in medicine, and Evidation Health is leading the way. Over the years, Evidation has built the largest, most diverse virtual pool of research participants through its proprietary and popular app, Achievement. With a direct and trusted relationship with more than 2 million individuals, its deep research expertise, and its data platform, Evidation Health can undertake real world research for life sciences and health care companies — and, ultimately, transform how health is measured and how diseases are identified, treated, and monitored.
Founded in 2012, Evidation Health is headquartered in San Mateo, Calif., with additional offices in San Francisco and Santa Barbara, Calif. To learn more, visit evidation.com, or follow us on Twitter @evidation.
About Tidepool
Tidepool is an open source, not-for-profit company focused on liberating data from diabetes devices, supporting researchers, and providing great, free software to people with diabetes and their care teams. Through the Tidepool Clinical Studies Platform and the Tidepool Big Data Donation Project, Tidepool is empowering the next generation of diabetes research and innovation. To learn more, visit tidepool.org.
Share on LinkedIn Twitter
---- Deborah Kilpatrick, Ph.D.
CEO
Evidation Health | www.evidation.com
650.906.7641
- Poster presentation of STARS topline data: the first clinical trial to show a positive clinical
benefit on overall Angelman syndrome impairments -
NEW YORK, Aug. 16, 2018 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that an abstract on OV101 was accepted for poster presentation at the 65th American Academy of Child and Adolescent Psychiatry (AACAP) Annual Meeting taking place in Seattle, Wash., October 22 to 27, 2018. AACAP is the largest international gathering of child and adolescent psychiatrists.
Angelman syndrome is a rare, lifelong, genetic disorder that affects 1 in 15,000 people in the general population. It is characterized by severe impairment in behavior, learning, verbal communication, motor skills, and sleep, and there are no FDA-approved medicines or an established treatment paradigm for this condition. If approved, OV101 could be the first medicine to specifically target a key underlying neurological dysfunction of Angelman syndrome: impaired tonic inhibition, which is most commonly caused by a disruption of the ubiquitin protein ligase (UBE3A) gene.
Poster presentation details
Title: Topline Results from a Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol (OV101)
Session: New Research Poster Session 3
Date and Time: Thursday, October 25, 2018, 9:30 a.m. – 12:00 p.m. ET
Topline data from the STARS trial were announced on August 6, 2018.
About OV101
OV101 (gaboxadol) is believed to be the only delta (δ)-selective GABAA receptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has been demonstrated in laboratory studies and animal models to selectively activate the δ-subunit of GABAA receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through tonic inhibition.
Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and relieve several of the symptoms of these disorders. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. This compound has also previously been tested in over 4,000 patients (over 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles.
The FDA has granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. The U.S. Patent and Trademark Office has granted Ovid patents directed to methods of treating Angelman syndrome and Fragile X syndrome using OV101. The issued patents expire in 2035.
About Ovid Therapeutics
Ovid Therapeutics (NASDAQ: OVID) is a New York-based biopharmaceutical company using its BoldMedicine™ approach to develop medicines that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of first-in-class medicines. The company’s lead investigational medicine, OV101, is currently in development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935/TAK-935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of rare developmental and epileptic encephalopathies (DEE).
For more information on Ovid, please visit http://www.ovidrx.com/.
Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding (i) the potential clinical benefit of OV101 to treat patients with Angelman syndrome, and (ii) the timing and results of any discussions with regulatory authorities regarding the registrational path for OV101 and approval. You can identify forward-looking statements because they contain words such as “will,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission. Ovid assumes no obligation to update any forwardlooking statements contained herein to reflect any change in expectations, even as new information becomes available.
Contacts
Investors:
Lora Pike
Ovid Therapeutics Inc.
Senior Director, Investor Relations & Public Relations
lpike@ovidrx.com
Steve Klass
Burns McClellan, Inc
sklass@burnsmc.com
(212) 213-0006
Media:
Elliot Fox
W2O Group
efox@w2ogroup.com
(212) 257-6724
– First Solid Tumor Product Candidate Based on Unum’s universal ACTR Technology –
– Phase 1 Study Expected to Initiate by the End of 2018 –
CAMBRIDGE, Mass., Aug. 13, 2018 (GLOBE NEWSWIRE) -- Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies based on its novel, universal Antibody-Coupled T Cell Receptor (ACTR) technology platform, today announced that an investigational new drug (IND) application is now active for ACTR T cells in combination with trastuzumab for the treatment of patients with HER2+ advanced cancers. This represents the first solid tumor product candidate
based on Unum’s novel, universal ACTR technology, and the fourth clinical trial program for the Company.
“We are very happy to reach this important milestone for patients and for Unum,” said Chuck Wilson, Chief Executive Officer of Unum. “ACTR represents a promising novel technology that can be used to target different tumor types and it’s exciting to expand its application to target solid tumors. We are committed to developing ACTR for patients with HER2+ advanced cancers who need better treatment options.”
Under this IND, Unum is preparing to initiate a multi-center Phase I trial, called ATTCK-34-01, by the end of 2018 in patients with HER2+ advanced cancers. ATTCK-34-01 is designed as a dose escalation study where both the ACTR T cell drug product and trastuzumab doses are escalated in order to define the safety, tolerability, and anti-tumor activity of the combination. Expansion at the recommended Phase 2 dose is planned.
About Unum Therapeutics
Unum Therapeutics is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel immunotherapy products designed to harness the power of a patient’s immune system to cure cancer. Unum’s novel proprietary technology, antibody-coupled T cell receptor (ACTR), is a universal, engineered cell therapy intended to be used in combination with a wide range of tumor-specific antibodies to target different tumor types. ACTR087 used in combination with rituximab, an anti-CD20 antibody, is Unum’s most advanced product candidate, currently in Phase I clinical testing in adult patients with relapsed or refractory non-Hodgkin lymphoma (r/r NHL). The Company has two additional product candidates in Phase I clinical testing: ACTR087 used in combination with the novel antibody SEA-BCMA in adult patients with relapsed or refractory multiple myeloma and ACTR707, a modified ACTR construct, used in combination with rituximab in adult patients with r/r NHL. Finally, the Company has an active investigational new drug application (IND) for ACTR707 used in combination with trastuzumab, an anti- human epidermal growth factor receptor 2 (HER2) antibody, to treat patients with HER2+ cancers and expects to initiate the Phase 1 trial by the end of 2018.
The Company is headquartered in Cambridge, MA.
Forward looking Statements
This press release contains forward-looking statements. Statements in this press release about the Company’s future expectations, plans and prospects, including projections regarding the anticipated timing of its clinical trials and regulatory filings, the development of its product candidates, including the four lead ACTR product candidates, as well as other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar expressions, constitute forward-looking statements within the meaning of the safe harbor provisions of The Private Securities Litigation Reform Act of 1995. The Company may not actually achieve the forecasts disclosed in the Company’s forward-looking statements, and undue reliance should not be placed on its forward-looking statements. Actual results could differ materially from the projections disclosed in the forward-looking statements the Company makes as a result of a variety of risks and uncertainties, including risks related to the accuracy of its estimates regarding expenses, future revenues, capital requirements, and the need for additional financing, the success, cost and timing of its product development activities and clinical trials, its ability to obtain and maintain regulatory approval for its product candidates, and the other risks and uncertainties described in the "Risk Factors" sections of the Company’s public filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date hereof. The Company anticipates that subsequent events and developments may cause its views to change. However, while it may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date hereof.
Investor Contact:
Stern Investor Relations, Inc.
Mary T. Conway, 212-362-1200
mary@sternir.com
Media Contact:
Paul Kidwell, 617-680-1088
paul.kidwell@unumrx.com
Former CEO and President of Zafgen brings pharmaceutical development and public biotechnology company
leadership experience as Navitor builds product pipeline
George P. Vlasuk, Ph.D. to remain as President and appointed Chief Scientific Officer
CAMBRIDGE, Mass., August 2, 2018 – Navitor Pharmaceuticals, Inc., a biopharmaceutical company targeting the mTORC1 pathway to develop novel therapeutics that help patients live longer and healthier lives, today announced the appointment of Thomas E. Hughes, Ph.D., as Chief Executive Officer. Dr. Hughes is the former Chief Executive Officer of Zafgen (Nasdaq:ZFGN), a publicly‐traded biotechnology company developing medicines for metabolic diseases and has been a scientific advisor to Navitor since its seed stage. George P. Vlasuk, Ph.D., current President and CEO of Navitor, will remain President and will assume the newly‐created role of Chief Scientific Officer. Both Dr. Hughes and Dr. Vlasuk will serve on Navitor’s Board of Directors.
These moves come at an exciting time for Navitor, which recently initiated a Phase 1 clinical study of its lead candidate, NV‐5138, for treatment‐resistant depression (TRD) and is advancing its mTORC1 platform to address multiple therapeutic applications, including CNS, immuno‐metabolism, fibrosis and multiple rare diseases.
“We are delighted to bolster the leadership team at Navitor to further strengthen our ability to advance a multi‐product pipeline based on our proprietary mTORC1 platform. Tom brings extensive pharmaceutical development and public biotechnology company experience to lead this next stage of Navitor’s growth,” said Alan Crane, Co‐founder and Chairman of the Board of Navitor and Entrepreneur Partner at Polaris Partners.
“George has been instrumental in building Navitor to establish the industry‐leading capabilities of our mTORC1 platform. George had the foresight to propose bringing a CEO on board with public company and translational experience, while he looks to focus his efforts on continuing to advance the science of Navitor. We are thrilled to have both George and Tom as members of the Navitor leadership team and are confident that with their combined experience, we will be able to advance the company to its next stage of evolution,” continued Mr. Crane.
“I am excited to join Navitor and am eager to work with George and the team he has built to move Navitor through its next stages of growth,” said Dr. Hughes. “mTORC1 is a powerful biological node with proven and tractable approaches to drug development. With a strong platform of proprietary approaches to drugging the mTORC1 system both for selective inhibitors and activators, Navitor is well positioned to lead this field of biology with valuable new insights and therapeutic programs addressing diseases with high and unmet medical need. The recent clinical trial initiation of NV‐5138 is the first application of Navitor’s mTORC1 technology in its platform of programs that aim to bring new therapies to patients with limited treatment options.”
Navitor’s small molecule therapeutics are designed to selectively modulate the cellular signals that are aberrant in disease processes caused by the dysregulation of mTORC1 activation. Navitor was founded based on the groundbreaking discoveries related to the mTORC1 pathway and nutrient signaling mechanisms by Dr. David Sabatini at The Whitehead Institute for Biomedical Research.
Dr. Vlasuk commented, “I am excited to focus my efforts on further building the capabilities of Navitor’s mTORC1 platform, expanding our pipeline and shaping the long‐term direction of our drug discovery and development efforts. I look forward to working with Tom, whom I have known professionally for many years, to realize our Company’s vision.”
Dr. Tom Hughes, who has more than 30 years of industry experience in the development and commercialization of pharmaceutical products, most recently served as President and Chief Scientific Officer of Zafgen and previously led Zafgen as CEO from 2008 to 2017. During this time, Dr. Hughes established Zafgen as a leading biotechnology company working in the area of rare and prevalent metabolic disorders and led the company through its Initial Public Offering in 2014. Prior to Zafgen, Dr. Hughes held several positions at Novartis including Global Head of the Cardiovascular and Metabolic Diseases Therapeutic Area at the Novartis Institutes for BioMedical Research in Cambridge, MA. In these roles, he oversaw many drug discovery and development projects targeting major global aging‐related health issues including obesity, diabetes, and heart disease. Dr. Hughes currently serves as a member of the Board of Directors of miRagen Therapeutics, Inc., is an advisor to Atlas Venture, and is a member of several scientific and strategic advisory boards, including Broadview Ventures, HotSpot Therapeutics, and Nimbus Therapeutics. He holds a Ph.D. in nutritional biochemistry from Tufts University, an M.S. in zoology from Virginia Polytechnic Institute & State University and a B.A. in biology from Franklin and Marshall College.
About NV‐5138
NV‐5138 is an orally bioavailable, small molecule that is designed to directly and transiently activate mTORC1 activity by binding to and modulating a newly discovered cellular sensor protein for the amino acid leucine, which is a potent natural activator of mTORC1. Unlike leucine, oral administration of NV‐5138 results in significant mTORC1 pathway activation in the brain since it is not broken down or incorporated into new proteins. These properties make NV‐5138 a unique agent with which to evaluate the role of mTORC1 in brain disorders, such as depression, where mTORC1 activity is often suppressed. Results from preclinical models demonstrate that NV‐5138 produces rapid upregulation of key synaptic proteins, synaptogenesis and sustained antidepressant behavioral responses via the transient and direct activation of the mTORC1 signaling pathway. Since NV‐5138 does not directly modulate the NMDA receptor pathway, it may not have the side effects and abuse potential observed with several NMDA receptor therapeutics currently in development. NV‐ 5138 is currently being clinically studied for the treatment of major depressive disorder (MDD) with an initial focus on treatment‐resistant depression (TRD).
About Navitor
Navitor Pharmaceuticals, Inc.is realizing the potential of modulating mTORC1, the master regulator of cellular function, to develop a pipeline of therapeutics that help patients live longer and healthier lives. Our industry leading team is unlocking the promise of recent discoveries in mTORC1 biology to address a broad range of chronic diseases. Our initial clinical application is a first‐in‐class drug to address unmet needs in depression. For more information, please visit www.navitorpharma.com.
Contact:
The Yates Network
Kathryn Morris, 914‐204‐6412
kathryn@theyatesnetwork.com
Launches First, Purpose-Built Data Platform to Measure and Better Understand How Everyday Behavior Data, Medical Conditions, and Symptoms Interact
SAN MATEO, CALIF., August 1, 2018 – Evidation Health, a health and measurement company that helps life sciences and health care companies understand how everyday behaviors and health interact, announced today that it has raised $30 million in Series C funding.
The round was co-led by SV Health Investors, a new investor, and existing investor B Capital Group. It included participation from existing investors GE Ventures and Sanofi Ventures. With this funding, Evidation has raised a total of $61 million.
Evidation also announced that it launched a new data platform to enable its customers to analyze and process large-scale sensor and behavior data in clinically meaningful ways.
“Our new data platform makes it easier for statisticians and data scientists at life sciences and health care companies to take everyday behavior and health care data, analyze it, and create a new understanding of health,” said Evidation Health CEO Deborah Kilpatrick, Ph.D. “This will help Evidation speed a transformation in real world research and knowledge, so we can better treat, diagnose, and predict the onset of disease.”
By linking real world data from smartphones and connected sensors — including wearables and medical devices — with traditional medical data, Evidation measures how behaviors outside of the doctor’s office or hospital relate to health and impact outcomes. The new data platform — which has been battle tested in a pilot program with a leading, global pharmaceutical company — can ingest individually-permissioned data from more than 100 sources ranging from Apple Health to Fitbit, Epic, Blue Button, and Dexcom.
The platform marries this information with traditional data from insurance claims, electronic health records, and diagnostic reports, alongside patient-reported outcomes collected directly through Evidation’s platform. This provides a new opportunity to analyze individual behavior and health in real time, not just when someone checks in with a physician or stops to record what they did or felt.
“Evidation’s data platform is opening up exciting new possibilities in medical research and the way we treat and prevent disease,” said P. Murali Doraiswamy, M.D., professor of psychiatry and medicine at Duke University Health System, and an advisor to Evidation. “With its unique analytics and methods, Evidation is transforming data into the kind of findings that traditional clinical trials may need substantially longer time frames, maybe even decades, to achieve. This will help identify health risks earlier and better treat the kinds of chronic diseases that scientists are working to cure.”
The secure, private platform currently processes more than 1 trillion data points each year across millions of individuals. It is built to manage the scale of high volume, continuous data streams and eliminates the need for infrastructure, data, and feature engineering on the part of Evidation customers, allowing them to focus entirely on analytics.
Already, the platform is being used to invent new ways to measure health across diabetes and cognitive decline. This complements the work that the company is performing with partners across multiple therapeutic areas ranging from cardiovascular disease to chronic pain, rheumatoid arthritis, migraine, depression and anxiety, fatigue, heart failure, and asthma — to name a few. In addition to supporting the new data platform, the funding will be used to develop partnerships, adding to Evidation’s vast array of real world data sources, and to launch novel research studies to better understand and measure how everyday behavior and health interact.
“We’re excited to deepen our partnership with Evidation Health and continue supporting the team as they build the leading platform to generate clinical relevance from everyday behavior data in real world populations. Evidation’s ability to link patient behavior to medical outcomes is truly revolutionary and will help transform how we measure health in everyday life,” said Raj Ganguly, co-founder and partner of B Capital Group.
“Evidation stands out not only because of its powerful data intake, aggregation, and analytics capabilities, but also because of the strength and vision of the company’s leadership team with a rare combination of deep technology and health care expertise. Evidation represents a unique platform company with the ability to meaningfully improve clinical development and, by extension, drug delivery, treatment, and outcomes,” added SV Health Investors Managing Partner Michael Balmuth, who is joining Evidation’s board of directors.
Since 2012, Evidation Health has built the most diverse virtual pool of research participants through its proprietary app, Achievement. With more than 2 million individuals using the app, this cohort is unparalleled in clinical research today, and represents the U.S.’s largest virtual research site. Individuals have the opportunity to participate in research studies, enabling Evidation to drill down to the individual level to address new questions and generate new context as needed — to measure what treatments work best and under what conditions, and enable earlier diagnoses and better matched interventions.
About Evidation
Evidation Health is a new kind of health and measurement company that provides the world’s most innovative life sciences and health care companies the technology and expertise they need to understand how everyday behavior and health interact. The volume of behavior data generated from smartphones and connected sensors — including wearables and medical devices — has opened up new ways to analyze individuals’ behavior and health in real time, unlocking insights into what medicines and treatments work best and spotting significant changes in health earlier. The scale and utility of everyday behavior data has the potential to be one of the most transformative forces in medicine, and Evidation Health is leading the way. Over the years, Evidation has built the largest, most diverse virtual pool of research participants through its proprietary and popular app, Achievement. With a direct and trusted relationship with more than 2 million individuals, its deep research expertise, and its data platform, Evidation Health can undertake real world research for life sciences and health care companies — and, ultimately, transform how health is measured and how diseases are identified, treated, and monitored. Founded in 2012, Evidation Health is headquartered in San Mateo, Calif., with additional offices in San Francisco and Santa Barbara, Calif. To learn more, visit evidation.com, or follow us on Twitter @evidation.
About SV Health Investors
V Health Investors, formerly named SV Life Sciences, is a healthcare and life sciences venture capital and growth equity firm. SV targets early-stage opportunities in biotechnology; early-stage and revenue-stage opportunities in medical devices; and growth equity investments for later-stage businesses in healthcare services and digital health. Over the past 20 years, SV Health Investors has invested in more than 175 companies. The firm currently has over $2.5 billion of capital commitments under management. SV Health Investors has offices in Boston and London. For more information, please visit www.svhealthinvestors.com.
About B Capital Group
B Capital Group is a global venture capital firm that invests in pioneering healthcare, fintech, industrial logistics and consumer enablement companies that are primed to scale across the global stage. Founded in partnership with The Boston Consulting Group, B Capital Group delivers unique access to top corporations to match cutting-edge start-ups with the world's leading CEOs, platforms, and brands. Existing portfolio companies include AImotive, Atomwise, Bird, Bizongo, Bright.md, Capital Match, Carro, CXA, Evidation Health, Fishbrain, Hollar, Icertis, INTURN, Journera, Lanetix, Mswipe, Myia, Ninja Van and SilverCloud Health. For more information, visit www.bcapgroup.com.
CAMBRIDGE, Mass. and NEW YORK - July 23, 2018 - Click Therapeutics, Inc. (“Click”), a leader in Digital Therapeutics™ solutions as prescription medical treatments, announces a $17 million financing round led by Sanofi Ventures. Click will use this financing to continue advancing its proprietary platform and pipeline of prescription digital therapeutics to treat a wide range of diseases. Recent notes were converted to equity as part of the financing.
“We’ve evaluated many companies in this space, and we believe Click Therapeutics’ mobile patient engagement platform positions the company to be a leader in the field of prescription digital therapeutics,” said Bernard Davitian, SVP and Managing Director of Sanofi Ventures, who has joined Click’s Board in conjunction with the financing. “Click’s platform enables the company to target multiple indications efficiently and effectively, and we have invested with the intent of partnering across a variety of therapeutic areas. Sanofi Ventures is excited to join Click on this journey of building a new pillar of medicine.”
In addition to Clickotine®, Click’s commercial product for smoking cessation, the company is developing prescription digital therapeutics for the treatment of depression (CT-152), insomnia (CT-141), acute coronary syndrome (CT-111), and chronic pain (CT-130). Click will seek FDA clearance for these programs as class II medical devices with disease-specific treatment claims, to be prescribed by physicians and reimbursed by payers.
“The Click Therapeutics team is proud to partner with Sanofi Ventures to advance our pipeline and expand our product portfolio of prescription medical treatments,” remarked David Benshoof Klein, Co-founder and CEO of Click. “As we announced last summer, in 2017 we expanded our collaboration with Magellan Health, Inc. to pursue regulatory clearance from the FDA for indication-specific prescription digital therapies, leveraging the industry-leading suite of intellectual property and data from Magellan’s existing software as well as their vast coverage and reimbursement leadership. The addition of Sanofi as a strategic investor, and the closing of this financing, represent major steps forward for Click and for the field of software as prescription medical treatments. By connecting patients with cognitive and neurobehavioral interventions, our platform will bring clinically-validated digital therapeutic solutions into mainstream healthcare.”
About Sanofi Ventures
Sanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are rare diseases, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. For more information, visit www.sanofiventures.com.
About Magellan Health
Magellan Health, Inc., a Fortune 500 company, is a leader in managing the fastest growing, most complex areas of health, including special populations, complete pharmacy benefits and other specialty areas of healthcare. Magellan supports innovative ways of accessing better health through technology, while remaining focused on the critical personal relationships that are necessary to achieve a healthy, vibrant life. Magellan's customers include health plans and other managed care organizations, employers, labor unions, various military and governmental agencies and third-party administrators. For more information, visit MagellanHealth.com.
About Click Therapeutics
Click Therapeutics, Inc. develops and commercializes software as prescription medical treatments for people with unmet medical needs. Through cognitive and neurobehavioral mechanisms, Click’s Digital Therapeutics™ enable change within individuals, and are designed to be used independently or in conjunction with biomedical treatments. The Clickometrics® adaptive data science platform continuously personalizes user experience to optimize engagement and outcomes. Following a groundbreaking clinical trial, Click’s industry-leading smoking cessation program is available nationwide through a wide variety of payers, providers, and employers. Click’s lead prescription program is entering into a multi-center, randomized, controlled, parallel-group, phase III FDA registration trial for the treatment of Major Depressive Disorder in adults. For more information, visit ClickTherapeutics.com.
# # #
Company Contact
Sarah Jackson
Chief of Staff
sarah@clicktherapeutics.com
Media Contact
Stefanie Tuck
MacDougall Biomedical Communications
stuck@macbiocom.com
781-235-3060
Novel Enzyme Replacement Therapy in Development for Treatment of ENPP1 Deficiency
Boston, Mass., July 17, 2018 – Inozyme Pharma (Inozyme), a biopharmaceutical company dedicated to developing treatments for rare and debilitating diseases, today announced that both the Food and Drug Administration’s (FDA) Office of Orphan Products Development and the European Medicines Agency’s (EMA) Committee for Orphan Medical Products (COMP) granted Orphan Drug Designation to INZ-701 for the treatment of ENPP1 Deficiency. INZ-701, the Company’s lead therapeutic candidate, is in pre-clinical development for the treatment of patients with ENPP1 Deficiency, a serious and life-threatening calcification disorder that manifests as generalized arterial calcification of infancy (GACI) in infants and as autosomal recessive hypophosphatemic rickets type 2 (ARHR2) post-infancy.
“Orphan Drug Designation, both in the United States and the European Union, is an important regulatory milestone for Inozyme as we continue our quest to develop INZ-701 for patients with rare and life-threatening calcification disorders,” said Axel Bolte, co-founder and chief executive officer of Inozyme. “The dual designations from the FDA and EMA provide crucial momentum for INZ-701, putting us in an excellent position to rapidly advance the clinical development program for this novel enzyme replacement therapy.”
The FDA and EMA respectively grant Orphan Drug Designation to drugs intended for safe and effective treatment of rare, life-threatening or chronically debilitating conditions that affect fewer than 200,000 people in the United States or fewer than one in 2,000 individuals in Europe. By receiving Orphan Drug Designation, Inozyme qualifies for certain regulatory and financial incentives, including scientific assistance, fee reductions, tax credits and seven years of market exclusivity in the U.S., as well as 10 years of market exclusivity post-authorization in the European Union.
About ENPP1 Deficiency
The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/ phosphodiesterase 1 (ENPP1), which regulates inorganic pyrophosphate (PPi) levels in plasma. PPi is essential for preventing harmful soft tissue calcification and for regulating normal bone mineralization. ENPP1 Deficiency manifests as either generalized arterial calcification of infancy (GACI) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2). GACI type 1 is a devastating and often fatal disease affecting infants and is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about half of patients within the first six months of life. ARHR2 manifests in the post-infancy stage and causes rickets, weakened bones, repeated bone fractures, skeletal deformities, short stature, muscle weakness, fatigue, and bone pain.
About INZ-701
INZ-701 is an enzyme replacement therapy under development with the intention to be used for the treatment of calcification disorders of the circulatory system, bones, and kidneys. In pre-clinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys and normalizing bone.
Inozyme is developing INZ-701 for certain rare, life-threatening and devastating genetic disorders such as ENPP1 Deficiency (GACI and ARHR2) and pseudoxanthoma elasticum (PXE) in which PPi levels are below the normal physiological levels. For more information about INZ-701, please visit: http://www.inozyme.com/our-science/.
About Inozyme Pharma
Inozyme Pharma is a biotechnology company committed to developing novel medicines for the treatment of rare metabolic diseases of calcification. The company was founded in 2016 with technology licensed from Yale University. For more information, please visit: www.inozyme.com.
Contact:
Inozyme Pharma
Henric Bjarke, COO
(617) 299-8321
henric.bjarke@inozyme.com
SmithSolve
Alex Van Rees
(973) 442-1555 ext. 111
alex.vanrees@smithsolve.com
NV‐5138 is a specific and direct activator of mTORC1, a cellular pathway required for the efficacy of many rapid acting antidepressants
CAMBRIDGE, Mass., June 26, 2018 – Navitor Pharmaceuticals, Inc., a biopharmaceutical company targeting the mTORC1 pathway to develop novel therapeutics that help patients live longer and healthier lives, announced today the initiation of a Phase 1 clinical study with its lead pipeline candidate, NV‐5138, for treatment‐resistant depression (TRD). NV‐5138 is a novel small molecule that directly activates mTORC1, a master cellular regulator that has recently been shown to be a central signaling pathway required for the efficacy of several rapid acting antidepressants. NV‐5138 is initially being evaluated in TRD but may offer future potential for the treatment in the broader disease category of major depressive disorder (MDD.)
“We are enthusiastic about initiating clinical development with NV‐5138 for major depressive disorder, as we believe this novel activator of mTORC1 has the potential to offer a unique approach to meeting many of the unmet needs of this serious and chronic disease. Millions of patients with depression do not adequately respond to standard pharmacological therapies which can take weeks or months before patients experience their effects, if at all, “said George P. Vlasuk, PhD, President and Chief Executive Officer of Navitor. “We see the development of NV‐5138 in MDD/TRD as a pioneering advance toward realizing the therapeutic potential of modulating the mTORC1 signaling pathway to treat a wide range of chronic human diseases.”
The Phase 1, multicenter, two‐part, double‐blind, placebo‐controlled study will evaluate the safety, tolerability and pharmacokinetics of NV‐5138 in up to 88 subjects, including healthy volunteers and patients diagnosed with TRD. In Part A, the single‐ascending‐dose portion of the study, up to 48 healthy volunteers will be randomly assigned to double‐blind treatment in six dosage‐level cohorts. Within each cohort, six subjects will be randomized to receive NV‐5138 and two subjects will be randomized to receive placebo. In Part B of the study, approximately 40 subjects diagnosed with TRD will be randomly assigned to double‐blind treatment at a single dosage level that will be established based on data from Part A of the study. Other prespecified outcome measures to be evaluated in Part B include standard depression rating and symptomology scores such as the Montgomery‐Åsberg Depression Rating Scale (MADRS).
“Initiation of this clinical study is supported by preclinical studies demonstrating the potential of NV‐5138 as an oral treatment for depression through activation of mTORC1, a cellular pathway that appears to underlie the beneficial effects of several in a new class of rapidly acting antidepressants,” said Maurizio Fava, MD, Director of the Division of Clinical Research of the Massachusetts General Hospital (MGH) Research Institute and member of the Navitor Clinical Advisory Board.
Previously, Navitor has presented preclinical results on the efficacy of NV‐5138 in multiple models of depression‐like behavior, which demonstrated that NV‐5138 produced behavioral responses and concomitant increases in new synapses (synaptogenesis) consistent with a rapid‐acting antidepressant through transient, direct activation of the mTORC1 signaling pathway in the brain. Navitor leveraged multiple preclinical observations that have shown mTORC1 activation is required for the efficacy of many rapid‐acting antidepressant compounds including several modulators of the NMDA (N‐methyl‐D‐aspartic acid)‐mediated signaling pathway like ketamine, which is an active area of innovative drug development for depression.
About Treatment Resistant Depression and Treatment Options
Treatment‐resistant depression (TRD) is a subset of major depressive disorder (MDD) that refers to depressive episodes that are not adequately controlled by standard antidepressant therapy. Several studies including a postmortem analysis of healthy and severely depressed patients as well as multiple pre‐clinical settings have suggested an association between the activity of mTORC1 pathway signaling and depression. Standard antidepressant therapies, such as selective serotonin reuptake inhibitors (SSRIs) and serotonin and
norepinephrine reuptake inhibitors (SNRIs) are only modestly effective and have a very slow onset typically taking 6‐8 weeks to show efficacy. Newer drugs that antagonize or otherwise modulate the presynaptic glutamate N‐methyl‐D‐aspartic acid (NMDA) receptor, have demonstrated the potential for improved efficacy with a rapid onset of antidepressant effects (days as opposed to weeks) and today there are several NMDA modulators in clinical development for depression, including ketamine and related agents. Unfortunately, presynaptic NMDA receptor modulation can cause significant side effects including dissociation (hallucination) and has abuse potential.
About Rapid Acting Antidepressants and mTORC1 Activity
New antidepressant drugs that modulate the presynaptic glutamate N‐methyl‐D‐aspartic acid (NMDA) receptor, have demonstrated the potential for improved efficacy with a rapid onset of antidepressant effects (days as opposed to weeks) and today there are several NMDA modulators in clinical development for depression, including ketamine and related agents. Since the initial observations connecting NMDA receptor modulation and depression, scientists have demonstrated that these agents increase production of key synaptic signaling proteins resulting in synaptogenesis and have also elucidated the mechanism that underlies the therapeutic antidepressant benefit seen with these agents in specific pre‐clinical settings. This new research demonstrates that presynaptic NMDA receptor modulation transiently activates the postsynaptic mTORC1 signaling pathway and this activation is required to initiate the cellular processes like protein synthesis that lead to the synaptogenesis and antidepressant effects of these agents. Although the initial target engagement of these agents occurs within a short time frame of a few hours, this transient activation of mTORC1 results in sustained, long‐lasting synaptic and behavioral effects that persist for days to even weeks after a single treatment.
About NV‐5138
NV‐5138 is an orally bioavailable, small molecule that is designed to directly and transiently activate mTORC1 activity by binding to and modulating a newly discovered cellular sensor protein for the amino acid leucine, which is a potent natural activator of mTORC1. Unlike leucine, oral administration of NV‐5138 results in significant mTORC1 pathway activation in the brain since it is not broken down or incorporated into new proteins. These properties make NV‐5138 a unique agent with which to evaluate the role of mTORC1 in brain disorders, such as depression, where mTORC1 activity is often suppressed. Results from preclinical models demonstrate that NV‐5138 produces rapid upregulation of key synaptic proteins, synaptogenesis and sustained antidepressant behavioral responses via the transient and direct activation of the mTORC1 signaling pathway. Since NV‐5138 does not directly modulate the NMDA receptor pathway, it may not have the side effects and abuse potential observed with several NMDA receptor therapeutics currently in development. NV‐ 5138 is currently being clinically studied for the treatment of major depressive disorder (MDD) with an initial focus on treatment‐resistant depression (TRD).
About Navitor
Navitor Pharmaceuticals, Inc. is realizing the potential of modulating mTORC1, the master regulator of cellular function, to develop a pipeline of therapeutics that help patients live longer and healthier lives. Our industry leading team is unlocking the promise of recent discoveries in mTORC1 biology to address a broad range of chronic diseases. Our initial clinical application is a first‐in‐class drug to address unmet needs in depression. For more information, please visit www.navitorpharma.com.
- Duman, RS and Aghajanian, GK. Science. 2012 October 5; 338(6103): 68–72.
- Scheung, L, et al., Frontiers in Neuroscience. 2015 July 21; 9 (249).
Contact:
The Yates Network
Kathryn Morris, 914‐204‐6412
kathryn@theyatesnetwork.com
CAMBRIDGE, Mass., June 25, 2018 (GLOBE NEWSWIRE) -- Unum Therapeutics Inc. (Nasdaq:UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies based on its novel, universal Antibody-Coupled T cell Receptor (ACTR) technology platform, today announced the company’s addition to the Russell 3000 Index following the Russell US Indices’ annual reconstitution.
“We are pleased with our addition to the Russell 3000 Index, a leading US equity benchmark for institutional investors, which will further increase our exposure to these key investors,” said Christiana Stamoulis, President and Chief Financial Officer of Unum Therapeutics.
Membership in the Russell 3000 Index, which remains in place for one year, means automatic inclusion in the applicable growth and value style indexes. FTSE Russell determines membership for its Russell US Indexes primarily by objective, market capitalization rankings and style attributes. Russell US Indexes are widely used by investment managers and institutional investors as the basis for index funds and as benchmarks for active investment strategies. Approximately $9 trillion in assets are benchmarked against Russell US Indexes. Russell US Indexes are part of FTSE Russell, a leading global index provider.
About Unum Therapeutics
Unum Therapeutics is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel immunotherapy products designed to harness the power of a patient’s immune system to cure cancer. Unum’s novel proprietary technology, antibody-coupled T cell receptor (ACTR), is a universal, engineered cell therapy intended to be used in combination with a wide range of tumor-specific antibodies to target different tumor types. Unum is actively building a pipeline of product candidates composed of ACTR T cells co-administered with antibodies for use in both hematologic and solid tumor cancers. The Company is headquartered in Cambridge, MA.
Forward-looking Statements
This press release contains forward-looking statements. Statements in this press release about our future expectations, plans and prospects, including projections regarding future revenues and financing performance, our long-term growth, the anticipated timing of our clinical trials and regulatory filings, the development of our product candidates, including the four lead ACTR product candidates, as well as other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar expressions, constitute forward-looking statements within the meaning of the safe harbor provisions of The Private Securities Litigation Reform Act of 1995. We may not actually achieve the forecasts disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results could differ materially from the projections disclosed in the forward-looking statements we make as a result of a variety of risks and uncertainties, including risks related to the accuracy of our estimates regarding expenses, future revenues, capital requirements, and the need for additional financing, the success, cost and timing of our product development activities and clinical trials, our ability to obtain and maintain regulatory approval for our product candidates, and the other risks and uncertainties described in the "Risk Factors" sections of our public filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent our views as of the date hereof. We anticipate that subsequent events and developments may cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so. These forwardlooking statements should not be relied upon as representing our views as of any date subsequent to the date hereof.
Investor Contact:
Stern Investor Relations, Inc.
Mary T. Conway, 212-362-1200
mary@sternir.com
Media Contact:
Paul Kidwell, 617-680-1088
paul.kidwell@unumrx.com
- G100 directly targets and modifies TLR4 expressing malignant B cells making them more visible to the anti-tumor immune response-
SEATTLE and SOUTH SAN FRANCISCO, Calif., June 25, 2018 (GLOBE NEWSWIRE) -- Immune Design (Nasdaq:IMDZ), an immunotherapy company focused on novel therapies in oncology, today announced preclinical and translational data that support the mechanism of action of G100 in patients with indolent non-Hodgkin Follicular lymphomas (FL). These data were presented at the Inaugural AACR International Meeting Advances in Malignant Lymphoma: Maximizing the Basic-Translational Interface for Clinical Application 2018 in Boston.
The research presented was designed to understand why high TLR 4 expression in patient’s samples correlated with clinical responses to G100 treatment. By analyzing patient samples, cell lines and mouse lymphoma models the following was observed:
- Murine and human B-lymphoma cell lines express TLR4 and respond in vitro to G100 stimulation with upregulation of MHC-II and co-stimulatory markers CD40 and CD80, typical of the activation of antigen-presenting function of B-cells;
- In vivo murine tumors of lymphoma models respond to treatment with G100 in injected tumors as well as distal, untreated tumors showing local and abscopal tumor control, mediated by systemic T-cell response;
- Approximately 70% of follicular lymphoma patients in a Phase 1/2 study express TLR4 in >50% of tumor cells in baseline biopsies. TLR4 expression ranging from 10%-100% of tumor cells was also detected in biopsies of patients with marginal zone lymphoma, small lymphocytic lymphoma, diffuse large B-cell lymphoma and cutaneous T-cell lymphoma; and
- In an ongoing Phase 2 trial of G100 with low dose radiation and pembrolizumab, almost all patients with an objective tumor response (³50% tumor shrinkage) showed TLR4 expression in >50% of tumor cells.
“These data illustrate that in addition to the known activation by G100 of dendritic cells and macrophages in the tumor microenvironment, G100 can also act directly on malignant B cells expressing TLR4. G100 treated malignant B cells may become more visible to the anti-tumor immune response, which correlates with clinical responses following intratumoral therapy with G100.” said Jan ter Meulen, MD, PhD, Chief Scientific Officer at Immune Design. “In FL patients, a strong correlation was observed between expression of TLR4 in more than 50% of tumor cells and objective responses following G100 therapy. This discovery potentially allows for a TLR4 biomarker-targeted G100 therapy of other tumor types, independent of histology.”
The full poster presentation can be accessed from the publications page of the Immune Design website.
About G100
G100 is a product candidate from Immune Design's internal discovery platforms and contains a potent synthetic small molecule toll-like receptor-4 (TLR-4) agonist, Glucopyranosyl Lipid A (GLA). G100 leverages the activation of both innate and adaptive immunity in the tumor microenvironment to create an immune response against the tumor's preexisting diverse set of antigens. A growing set of clinical and preclinical data have demonstrated the ability of G100 to activate tumor-infiltrating lymphocytes, macrophages and dendritic cells, and promote antigen-presentation and the recruitment of T cells to the tumor. The ensuing induction of local and systemic immune responses has been shown to result in local and abscopal (shrinking of tumors outside the scope of the localized treatment) tumor control. G100 was evaluated in a Phase 1 study in Merkel cell carcinoma patients and produced a 50% overall response rate per protocol and a favorable safety profile. Currently, G100 is being evaluated as both a monotherapy (with local radiation) and in combination with Merck’s anti-PD-1 agent, pembrolizumab, pursuant to a clinical collaboration with Merck, in a randomized Phase 1/2 trial in patients with follicular non-Hodgkin lymphoma.
About Immune Design
Immune Design is a late-stage immunotherapy company employing next-generation in vivo approaches to enable the body's immune system to fight disease. The company's technologies are engineered to activate the immune system's natural ability to generate and/or expand antigen-specific cytotoxic immune cells to fight cancer and other chronic diseases. CMB305 and G100, the leading product candidates with broad potential in oncology, are based on the company’s two technology platforms that are potent stimulators of the immune system – ZVex ® and GLAAS® - the fundamental technologies of which were licensed from the California Institute of Technology and the Infectious Disease Research Institute (IDRI), respectively. Both ZVex and GLAAS also have potential applications in infectious disease and allergy indications, which are being developed through ongoing pharmaceutical collaborations. Immune Design has offices in Seattle and South San Francisco. For more information, please visit www.immunedesign.com.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Immune Design's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the timing of initiation, progress, scope and outcome of clinical trials for Immune Design's product candidates and the reporting of clinical data regarding Immune Design’s product candidates . Many factors may cause differences between current expectations and actual results including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of Immune Design's collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes. Other factors that may cause Immune Design's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Immune Design's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Immune Design assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
Media Contact
Julie Rathbun
julie.rathbun@immunedesign.com
206-769-9219
Investor Contact
Sylvia Wheeler
Sylvia.wheeler@immunedesign.com
Digital Therapeutics Pioneer Launches Comprehensive,
Personalized Solution for Obesity-Related Chronic Disease
San Francisco, CA (June 20, 2018) -- Building on the company’s pioneering approach to digital behavior change, Omada Health today announced the availability of new programs that will help individuals control type 2 diabetes and hypertension. Adding to Omada’s industry-leading diabetes prevention program, the company will now offer integrated condition management programs for enterprise and health plan customers, significantly expanding the populations Omada serves. Leveraging seven years of data-driven insights; integration experience with hundreds of health plan and employer customers; and a proven approach to sustainable lifestyle change, Omada will now provide personalized interventions for those at-risk for, or with, certain obesity-related chronic conditions.
“Since the early days of the company, we’ve developed Omada to be an adaptable program that delivers meaningful health outcomes for participants, and return on investment for customers,” said Omada Health CEO Sean Duffy. “Today’s announcement is the next step in that journey. Clients, partners, and participants want a holistic digital healthcare provider built on clinical evidence, best-in-class user experience, and validated results. As we deliver new programs and features, Omada will continue to help our customers manage the health of their populations, as well as their healthcare spending.”
In addition to the new condition management programs, Omada is leveraging industry-leading data science and expert coaching to support participants in new ways - adding features for medication adherence and integrating remote monitoring for glucose and blood pressure. The company will continue to contract as a digital healthcare provider, filing medical claims with billing tied to participation and outcomes. The Omada program will adapt to each participant based on his or her:
- Demographic profile;
- Clinical profile, including comorbidities and multiple conditions;
- Self-identified personal barriers to success;
- Individual actions once enrolled in the program; and
- Use of connected glucometer and blood pressure cuff
"Omada delivers evidence-based behavior change by combining digitally-enabled coaching and clinical fidelity with sophisticated data science." said Omada VP of Medical Affairs Carolyn Bradner Jasik, MD, "We will now bring that expertise to the challenging areas of medication adherence and remote monitoring through an extensible, flexible platform that treats the whole person -- not just a single piece of their diagnosis."
A recent industry report by Willis Towers Watson illustrated the employer need, as 76 percent surveyed planned to invest in specific clinical solutions to diabetes to improve member health and reduce costs in 2019. The same survey reported 84 percent of companies will seek to identify and manage population chronic conditions across their workforce. Recent estimates place the total cost of diabetes at $327 billion annually , and high blood pressure at $131 billion per year.
About Omada Health
Omada is a digital intensive behavioral counseling program focused on reducing costly chronic disease in employer and health plan populations. The company offers a scalable, adaptable intervention for employees or members at risk for, or with, obesity-related chronic conditions such as type 2 diabetes and hypertension. Omada is the largest CDC-recognized provider of the National Diabetes Prevention Program, with deep integration experience with leading health plans, outcomes-based pricing, and ten peer-reviewed studies demonstrating the company’s ability to deliver lasting, clinically-meaningful results. To learn more, visit www.omadahealth.com .
Contact Information:
Adam Brickman
(914) 548-3748
Press@Omadahealth.com
CAMBRIDGE, Mass., June 13, 2018 – Inozyme Pharma (Inozyme), a biopharmaceutical company dedicated to developing treatments for rare and debilitating diseases, today introduced a no-cost, third-party genetic testing program designed to improve detection and understanding of two rare calcification disorders. Offered globally and in partnership with PreventionGenetics, the Inozyme program tests eligible participants for mutations in the ENPP1 and ABCC6 genes. Both of these genes are implicated in rare, severe calcification disorders, known as ENPP1 deficiency and ABCC6 deficiency, respectively, (sometimes called generalized arterial calcification of infancy [GACI] and autosomal recessive hypophosphatemic rickets type 2 [ARHR2]).
“The genetic testing program introduced today will help to enhance our understanding of ENPP1 deficiency and ABCC6 deficiency, with the ultimate goal of improving diagnosis and developing effective treatments,” said Axel Bolte, co-founder and chief executive officer of Inozyme. “We urge people with a family history of calcification disorders and their physicians to seek more information about the genetic testing program. Over time and through ongoing research, we hope to alleviate the life-limiting and life-threatening impact of these severe diseases.”
Inozyme created the genetic testing program to increase disease awareness, reduce barriers to genetic testing, and help people and their healthcare providers make more informed decisions about these rare conditions.
“The Inozyme genetic testing program will provide an early diagnostic testing measure for ENPP1 and ABCC6 deficiencies, which are indicators of serious calcification disorders that sometimes take years to diagnose accurately,” said James Weber, president of PreventionGenetics. “Access to reliable genetic testing may help shorten the diagnostic journey for patients, potentially lifting a significant emotional burden and paving the way for more timely and effective intervention. We are excited to work with Inozyme as we make the test available to physicians and their patients.”
The no-cost program offered by Inozyme and PreventionGenetics provides genetic testing to those who qualify. Although genetic testing can confirm a suspected diagnosis of a calcification disorder linked to ENPP1 or ABCC6 gene mutations, the absence of a genetic alteration does not preclude diagnosis of such a disease. For more information about the genetic testing program, please visit: www.inozyme.com/genetic-testing/.
About ENPP1 Deficiency
The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/ phosphodiesterase 1 (ENPP1), which regulates inorganic pyrophosphate (PPi) levels in plasma. PPi is essential for preventing harmful soft tissue calcification and for regulating normal bone mineralization. ENPP1 deficiency manifests as either generalized arterial calcification of infancy (GACI) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2). GACI type 1 is a devastating and often fatal disease affecting infants and is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about half of patients within the first six months of life. ARHR2 usually manifests in the post-infancy stage, though it can occur in patients without prior GACI. ARHR2 causes rickets, weakened bones, repeated bone fractures, skeletal deformities, short stature, muscle weakness, fatigue and bone pain.
About ABCC6 Deficiency
Defects in the ABCC6 gene (ATP-binding cassette sub-family C member 6) lead to a decrease in plasma PPi and consequently to soft tissue calcification, and in rare circumstances cause GACI type 2 in infants. GACI type 2 is clinically similar to GACI type 1 and is also characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about half of patients within the first six months of life.
About Inozyme Pharma
Inozyme Pharma is a biotechnology company committed to developing novel medicines for the treatment of rare diseases characterized by mineral imbalances, which lead to over-calcification of soft tissues and under-mineralization of bone. The company was founded in 2016 with technology licensed from Yale University. For more information, please visit: www.inozyme.com.
About PreventionGenetics
PreventionGenetics is a CLIA and ISO 15189:2012 accredited clinical DNA testing laboratory founded in 2004 and located in Marshfield, Wisconsin. PreventionGenetics provides patients with sequencing and deletion/duplication tests for nearly all clinically relevant genes, including whole exome sequencing, PGxomeⓇ.
Contact:
Inozyme Pharma
Henric Bjarke, COO
(617) 299-8321 henric.bjarke@inozyme.com
SmithSolve
Alex Van Rees
973-442-1555 ext. 111 alex.vanrees@smithsolve.com
-- 1st global Phase 3 trial focused on synovial sarcoma patients
SYNOVATE is a randomized, global Phase 3 trial to evaluate CMB305 immuontherapy in synovial sarcoma patients. www.synovatestudy.com
SEATTLE and SOUTH SAN FRANCISCO, Calif., May 24, 2018 (GLOBE NEWSWIRE) -- Immune Design (Nasdaq:IMDZ), an immunotherapy company focused on novel therapies in oncology, today announced the launch of the patient and healthcare provider websites for the SYNOVATE study – a pivotal trial to evaluate CMB305 immunotherapy in synovial sarcoma patients. Clinical sites will soon be open for enrollment initially in the US, with information available at www.synovatestudy.com.
“Patients with advanced synovial sarcoma often have few systemic treatment options immediately after they complete first line chemotherapy. Clinically, we often give them a break from therapy, following closely with the hope that they will not progress in the relatively short time period that is unfortunately common in this disease,” said William D. Tap, M.D., Medical Oncology and Chief, Sarcoma Medical Oncology Service, Memorial Sloan Kettering Cancer Center. “It is our hope that CMB305 may offer a new option for patients to receive a potentially beneficial therapy that will defer the need for subsequent therapy.”
SYNOVATE is a randomized, global Phase 3 clinical trial that will evaluate CMB305 monotherapy versus placebo in 248 patients 12 years of age and older with NY-ESO-1 positive, unresectable, locally-advanced or metastatic synovial sarcoma. Patients who are responding to a first-line therapy can become eligible for SYNOVATE following completion of their chemotherapy. SYNOVATE will be opened and recruiting patients in cancer centers throughout the United States, Canada, Europe, and the Asia Pacific region.
“We are very pleased to be working with high quality clinical groups around the country and internationally to launch the SYNOVATE study to explore CMB305 immunotherapy in patients with synovial sarcoma,” said Sergey Yurasov, M.D., Ph.D., Chief Medical Officer, Immune Design. “SYNOVATE is the first randomized, global Phase 3 trial of an immunotherapy focused in synovial sarcoma, and we hope it results in a new, approved therapy for these patients.”
Patient and Health Care Provider Resouces for SYNOVATE Study
Information can be found on the SYNOVATE website at www.synovatestudy.com and on the NIH clinical trial registry www.clinicaltrials.gov (Identifier: NCT03520959).
About Synovial Sarcoma
Soft tissue sarcomas are malignancies that arise from the soft tissues of the body, such as tissues that connect, support and surround other body structures including muscle, fat, blood vessels, nerves, tendons and the lining of joints. Synovial sarcoma is a sub type of soft tissue sarcoma where 70% of diagnoses occur in patients under 40 years old, is associated with a high risk of recurrence, and has been shown to have high expression of the NY-ESO-1 tumor antigen. The primary treatment for patients with locally advanced, unresectable or metastatic synovial sarcoma typically consists of an anthracycline-based chemotherapy regimen administered alone or in combination with other agents. Following disease progression after first line systemic therapy, treatment options are limited and median overall survival rates have been reported to be approximately 12 months. In connection with the planned Phase 3 study for CMB305 monotherapy, the FDA has agreed with Immune Design that synovial sarcoma patients constitute an unmet medical need.
About CMB305
CMB305 is a prime-boost cancer vaccine targeting NY-ESO-1-expressing tumors. NY-ESO-1 is a cellular protein that is typically found only in certain cancer cells and is often frequently expressed in synovial sarcomas. CMB305 is administered to participants by injection and is designed to provide clinical benefit by generating an anti-NY-ESO-1 immune response by activating a participant’s antigen-presenting dendritic cells.
About Immune Design
Immune Design is a late-stage immunotherapy company employing next-generation in vivo approaches to enable the body's immune system to fight disease. The company's technologies are engineered to activate the immune system's natural ability to generate and/or expand antigen-specific cytotoxic immune cells to fight cancer and other chronic diseases. CMB305 and G100, the leading product candidates with broad potential in oncology, are based on the company’s two technology platforms that are potent stimulators of the immune system – ZVex® and GLAAS® - the fundamental technologies of which were licensed from the California Institute of Technology and the Infectious Disease Research Institute (IDRI), respectively. Both ZVex and GLAAS also have potential applications in infectious disease and allergy indications, which are being developed through ongoing pharmaceutical collaborations. Immune Design has offices in Seattle and South San Francisco. For more information, please visit www.immunedesign.com.
Cautionary Note on Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “expect,” “plan,” “anticipate,” “target,” “estimate,” “intend” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Immune Design’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause Immune Design’s clinical development programs, future results or performance to differ significantly from those expressed or implied by the forward-looking statements. Forwardlooking statements contained in this press release include, but are not limited to, statements about the progress, timing, scope and results of clinical trials and the timing and likelihood of obtaining regulatory approval of Immune Design’s product candidates. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, the uncertainties and timing of the regulatory approval process, and unexpected litigation or other disputes. Other factors that may cause Immune Design’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Immune Design’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” sections contained therein. Except as required by law, Immune Design assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
Media Contact
Julie Rathbun
julie.rathbun@immunedesign.com
206-769-9219
Investor Contact
Sylvia Wheeler
sylvia.wheeler@immunedesign.com
650-392-8318.
April 3, 2018
CAMBRIDGE, Mass., April 03, 2018 (GLOBE NEWSWIRE) -- Unum Therapeutics Inc. (“Unum Therapeutics”) (NASDAQ:UMRX), a clinical-stage biopharmaceutical company focused on the development of novel immunotherapy products designed to harness the power of a patient’s immune system to cure cancer, today announced the closing of its initial public offering of 5,770,000 shares of common stock at a public offering price of $12.00 per share. The gross proceeds from this offering, before deducting underwriting discounts and commissions and other offering expenses, are approximately $69.2 million. Unum Therapeutics’ common stock began trading on the Nasdaq Global Select Market under the ticker symbol "UMRX" on March 29, 2018.
Morgan Stanley and Cowen are acting as joint book-running managers for the initial public offering. SunTrust Robinson Humphrey and Wedbush PacGrow are acting as lead managers.
In addition to the shares sold in the initial public offering, Unum Therapeutics announced the concurrent sale of an additional 416,666 shares at the public offering price of $12.00, for gross proceeds of $5.0 million, in a private placement to Seattle Genetics, Inc., an existing shareholder of Unum Therapeutics. The sale of these shares of common stock is not registered under the Securities Act of 1933, as amended, and the shares are subject to a 180-day lock-up agreement.
A registration statement relating to the securities issued in the initial public offering was declared effective by the Securities and Exchange Commission (the “SEC”) on March 28, 2018. The offering will be made only by means of a prospectus. Copies of the final prospectus related to the offering may be obtained by visiting EDGAR on the SEC Web site at www.sec.gov or from Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014, or Cowen and Company, LLC, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, or by calling (631) 274-2806.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.
About Unum Therapeutics
Unum Therapeutics uses its proprietary antibody-coupled T cell receptor (ACTR) technology in combination with tumor-targeting antibodies to activate the body’s own immune system to fight cancer. Unum Therapeutics is actively building a pipeline of ACTR programs in combination with a wide range of proprietary, tumortargeting antibodies for use in both hematologic and solid tumor cancers. The Company is headquartered in Cambridge, MA.
Contact:
Unum Therapeutics Inc.
Christiana Stamoulis, +1-617-843-5352
christiana.stamoulis@unumrx.com
Source: Unum Therapeutics Inc.
– Names Kevin M. Forrest, Ph.D. President and CEO
– Proprietary technology based on pioneering work from the lab of Matthew D. Disney, Ph.D. of The Scripps Research Institute Florida
San Diego, January 3, 2018 -- Expansion Therapeutics, Inc., a new 5AM Ventures formed private company focused on the discovery and development of ribonucleic acid (RNA) targeted small molecule medicines, announced today the close of a $55.3 million Series A financing co-led by 5AM Ventures, Kleiner Perkins, Novartis Venture Fund, and Sanofi Ventures with participation from RA Capital Management and Alexandria Venture Investments. Proceeds will advance Expansion’s portfolio of small molecule drugs targeting key human disease-driving RNAs with an initial focus on expansion repeat disorders, a set of approximately 30 genetic diseases that currently have no satisfactory treatments.
“In a short period of time Expansion has assembled a leading team, the key scientific founder in Matt Disney of The Scripps Research Institute Florida, and a capital efficient plan to advance the emerging field of RNA targeted small molecule medicines,” said Scott M. Rocklage, Ph.D., Managing Partner of 5AM Ventures and founding investor and Chairman of the Board of Directors of Expansion Therapeutics. “We look forward to continuing to work with the company to develop medicines for patients with few treatment options.”
“The science underpinning Expansion’s RNA targeted medicines offers great promise in the treatment of a set of RNA triggered diseases, such as myotonic dystrophy, that currently have no viable therapies. We are excited to invest in a company that could make a real difference to patients suffering from these incurable diseases,” said Beth Seidenberg, M.D., General Partner of Kleiner Perkins.
Coincident with the close of the Series A, Expansion co-founder Kevin M. Forrest, Ph.D., was named president and chief executive officer. Dr. Forrest previously served as founding chief operating officer and chief financial officer for the San Diego-based anti-infectives company Cidara Therapeutics (Nasdaq: CDTX). Prior, he was a Principal at 5AM Ventures. Dr. Forrest holds a B.S. in biology from Boston College and a Ph.D. in molecular biology from Princeton University where he published on various RNA regulatory processes.
RNA Targeted Small Molecule Medicines
Incubated within 5AM Ventures’ 4:59 Initiative, and subsequently seed funded by 5AM and Sanofi Ventures, Expansion’s approach is based on key patent-protected platform technologies and enabling tools pioneered in the laboratory of Dr. Disney of The Scripps Research Institute, who is the leader in the field of small molecule targeting of RNA.
“I am gratified that our efforts over the past dozen years have culminated in this important opportunity,” said Dr. Disney. “It is clear that disease-related RNA is now an addressable target with small molecule medicines and we are now on the verge of developing treatments for patients with the most urgent medical needs. We will work tirelessly to fulfill this promise.”
Expansion repeat disorders include myotonic dystrophy type I (DM1), which is the leading cause of adult onset muscular dystrophy. “Expansion repeat disorders, in particular DM1, represent an attractive first application of our technology as it is well established that toxic RNA drives disease,” said Dr. Forrest. “Furthermore, our small molecule approach has the potential to address both peripheral and central symptoms that are debilitating for patients.”
Expansion Names Board of Directors and Forms Scientific Advisory Board
Following the close of the financing, the Expansion board of directors will include:
- Scott M. Rocklage, Ph.D., of 5AM Ventures and Chairman of the Expansion Board of Directors
- Matthew D. Disney, Ph.D., of The Scripps Research Institute
- Kevin M. Forrest, Ph.D., of Expansion Therapeutics
- Jason P. Hafler, Ph.D., of Sanofi Ventures
- Yujiro S. Hata, M.B.A., of Ideaya Biosciences
- Campbell Murray, M.D., M.B.A., M.P.P., of Novartis Venture Fund
- Beth Seidenberg, M.D., of Kleiner Perkins
- Andrew Levin, M.D., Ph.D., of RA Capital Management joins as a board observer
Expansion has also formed a scientific advisory board comprised of leaders in the field of RNA targeted small molecule chemistry and biology, RNA folding, and structural biology, including:
- Robert T. Batey, Ph.D., of the University Colorado, Boulder
- Dale L. Boger, Ph.D., of The Scripps Research Institute
- Ronald R. Breaker, Ph.D., of the Howard Hughes Medical Institute and Yale University
- M.G. Finn, Ph.D., of the Georgia Institute of Technology
- David H. Mathews, M.D., Ph.D., of the University of Rochester
- Michael Zuker, Ph.D., of the Rensselaer Polytechnic Institute
About RNA Targeted Small Molecule Medicines
Ribonucleic acid, or RNA, is a biomolecule that was once thought to be a simple messenger between DNA, or deoxyribonucleic acid, and protein. Recent advances in biology, however, have shown that RNA plays a much greater role than previously appreciated. This includes control of gene expression via long non-coding RNAs, RNA stability via small interfering RNAs, RNA translation via transfer RNAs and microRNAs, and even cellular communication (RNA exosomes). RNA can form higher order structures that form the basis of “druggable” surfaces and pockets that can be targeted by small molecule therapeutics. The best examples of RNA targeted small molecule medicines include several FDA approved classes of antibiotics that bind and inactivate key structures in bacterial RNAs.
About Myotonic Dystrophy Type I
Myotonic dystrophy type I (DM1) affects at least 1 in 8,000 people, or 40,000 individuals in the U.S. alone, and is the most frequent cause of adult onset muscular dystrophy. DM1 is caused by a toxic expansion in RNA, which leads to multi-systemic symptoms including muscular, cardiac, respiratory, gastrointestinal, endocrine, and central nervous system defects. This genetic disease often affects entire families, with progressively worsening disease across generations, and there are no effective treatment options available for DM1.
For more information about myotonic dystrophy type I, visit www.myotonic.org.
About Expansion Therapeutics, Inc.
Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases. Based on exclusive worldwide rights to groundbreaking research from the laboratory of Matthew D. Disney, Ph.D., at The Scripps Research Institute, Expansion has assembled the intellectual property, know-how, and proprietary enabling technologies and tools necessary to facilitate the creation of potent and specific small molecule binders of RNA. Through this unique platform, Expansion is building a portfolio of novel RNA-targeted drug candidates with activity across a broad number of disease indications. The company’s initial development focus is on therapies for patients with expansion repeat diseases who currently have limited and unsatisfactory treatment options. Expansion is based in San Diego, California and Jupiter, Florida.
For more information, visit www.expansionrx.com.
Media Contact:
Christy Curran
Sam Brown Inc.
christycurran@sambrown.com
615.414.8668
NEW YORK, Dec. 19, 2017 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ:OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OV101 for the treatment of Angelman syndrome.
“This designation is an important milestone for both Ovid and the Angelman community as it enables increased dialog with the FDA, speeding our ability to bring this potential therapeutic option to people living with Angelman syndrome. We believe that OV101, with its novel mechanism of action, has the potential to be an innovative and impactful therapy,” said Amit Rakhit, M.D., MBA, chief medical and portfolio management officer of Ovid Therapeutics. “In addition to the regulatory milestones of orphan drug and Fast Track designations for Angelman syndrome, we achieved significant clinical progress with our OV101 program. As a result of positive Phase 1 data, we were recently able to expand our ongoing Phase 2 STARS clinical trial to include both adults and adolescents with Angelman syndrome. We look forward to data from the STARS trial in the second half of 2018.”
OV101 is a delta (δ)-selective GABAA receptor agonist that targets the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of Angelman syndrome and other neurodevelopmental disorders. Ovid is currently studying OV101 in its Phase 2 STARS clinical trial, a randomized, double-blind, placebo-controlled study investigating the safety and efficacy of OV101 in patients with Angelman syndrome. Upon successful completion of a Phase 1 pharmacokinetic (PK) and safety study showing that OV101 has a similar PK profile in adolescents as in adults, Ovid recently amended the STARS protocol to include patients aged 13 years and older.
The FDA’s Fast Track process is designed to expedite the development and review of drugs used to treat serious conditions and fill an unmet medical need. Fast Track designation enables the company to have early and frequent communication with the FDA throughout the drug development and review process, often leading to faster drug approval and patient access.
About OV101
OV101 (gaboxadol) is believed to be the only delta (δ)-selective GABAA receptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has been demonstrated in laboratory studies and animal models to selectively activate the δ-subunit of GABAA receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through tonic inhibition.
Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and relieve several of the symptoms of these disorders. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. Gaboxadol has previously been tested in over 4,000 patients (approximately 950 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles.
The FDA has granted orphan drug and Fast Track designations for OV101 for the treatment of Angelman syndrome and orphan drug designation for the treatment of Fragile X syndrome. The U.S. Patent and Trademark Office has granted Ovid patents directed to methods of treating Angelman syndrome using OV101. The issued patents expire in 2035 for Angelman syndrome.
About Angelman Syndrome
Angelman syndrome is a genetic disorder that is characterized by a variety of signs and symptoms. Characteristic features of this disorder include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the disruption of a gene that codes for ubiquitin protein ligase E3A (UBE3A). Angelman syndrome affects approximately 1 in 12,000 to 20,000 people in the U.S. There are currently no U.S. Food and Drug Administration (FDA)-approved therapies for the treatment of Angelman syndrome.
Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta (δ)-selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information.
About Ovid Therapeutics
Ovid Therapeutics (NASDAQ:OVID) is a New York-based biopharmaceutical company using its BoldMedicine™ approach to develop therapies that transform the lives of patients with rare neurological disorders. Ovid’s drug candidate, OV101, is currently in development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid initiated the Phase 2 STARS trial of OV101 in people with Angelman syndrome in 2017 and completed a Phase 1 trial in adolescents with Angelman syndrome or Fragile X syndrome. Ovid is also developing OV935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of epileptic encephalopathies and in August 2017 initiated a Phase 1b/2a trial of OV935.
For more information on Ovid, please visit http://www.ovidrx.com/.
Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding progress, timing, scope and results of clinical trials for Ovid’s product candidates, the timing of clinical data, the development of therapies for younger patients, the provision of access to effective therapies, and the FDA Fast Track process leading to faster drug approval and patient access. You can identify forward-looking statements because they contain words such as “will,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended June 30, 2017, under the caption “Risk Factors.” Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
Contacts
Investors:
Burns McClellan
Steve Klass, 212-213-0006
Sklass@burnsmc.com
Media:
Pure Communications, Inc.
Katie Engleman, 910-509-3977
katie@purecommunicationsinc.com
Industry Experts Join Curisium’s Inaugural Advisory Board
Manhattan Beach, CA - December 14, 2017 - Curisium, a healthcare technology and services company that enables innovative contracting at scale via its blockchain-based platform, announced today it has raised $3.5M in seed financing from Flare Capital Partners, New Enterprise Associates (NEA), Shuttle Fund, Sanofi Ventures, and Green Bay Ventures. In conjunction with the financing, Bill Geary of Flare Capital and Mohamad Makhzoumi of NEA have joined the Curisium Board of Directors, and Ruchita Sinha of Sanofi Ventures has been appointed a Board Observer.
The Curisium platform uses cutting-edge blockchain and secure computation technologies to allow payers, providers, and life science companies to efficiently and securely engage in innovative, patient-centric value-based contracts. Nearly a third of the payments in the $3T US healthcare market are already tied to some form of innovative payment model; Curisium aims to transform healthcare by disrupting existing frameworks and enabling scalable value-based care contracting at the individual patient level.
“Payers, providers, and life science companies are increasingly entering into various forms of innovative contracts,” said Peter Kim, co-founder and CEO of Curisium. “However, effective implementation today is hampered by costly logistics, lack of trust, and difficulty verifying patient-level outcomes.”
“Curisium’s platform, by enabling outcome verification at the patient level, while automating the payment side, has the potential to rapidly accelerate the breadth and depth of innovative contracting arrangements,” said Bill Geary, co-founder and Partner at Flare Capital. “We’re thrilled to be an investor partner with the Curisium founding team having successfully backed them before, and are impressed with their deep industry and technology insights and capabilities.”
“There is no mechanism today to effectively define or share patient health states,” added Milind Kamkolkar, Chief Data Officer at Sanofi. “With the Curisium platform, we now have a trusted version of health records that will get better over time, and the industry can finally start to discover, maintain and improve patient health.”
Greg Papadopoulos, Venture Partner at NEA and former CTO of Sun Microsystems, noted “Healthcare data are siloed, and there is a reluctance to share due to fundamental distrust among its custodians. Curisium’s platform tackles this logjam with unique cryptographic guarantees around its data access.”
Both Kamkolkar and Papadopoulos have joined Curisium’s inaugural Industry Advisory Board.
“Blockchain technology has tremendous potential and there is a huge opportunity to implement this application within the healthcare industry,” stated Mohamad Makhzoumi, General Partner at NEA and Head of Healthcare Services. “We are thrilled to partner with the Curisium team as they pioneer blockchain for healthcare in a pragmatic, market-driven way, that addresses pain points across trust, security, and payment logistics.”
To learn more about Curisium, please visit www.curisium.com.
About Curisium
Curisium is a healthcare technology and services company based in Manhattan Beach, CA that provides scalable innovative contracting solutions with its blockchain-based platform. Founded by seasoned healthcare technology executives, Curisium provides tailored solutions for payers, providers, and life science companies to enter into patient-centric, secure and efficient innovative contracting arrangements. For more information, please visit www.curisium.com.
About Flare Capital Partners
Flare Capital is a team of proven healthcare technology venture capital investors known for their strategic industry resources, insight and total commitment to the success of its entrepreneurs. Flare Capital raised one of the healthcare industry’s largest dedicated venture capital funds focused exclusively on early stage and emerging growth investments in healthcare technology innovation and is privileged to closely partner with founders and management. Investments include Aetion, Bright Health, Circulation, ClearDATA, Curisium, Evolent Health, HealthReveal, HealthVerity, Iora Health, VisitPay and Welltok. Learn more at www.flarecapital.com.
About NEA
New Enterprise Associates, Inc. (NEA) is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors and geographies. With more than $20 billion in cumulative committed capital since the firm’s founding in 1977, NEA invests in technology and healthcare companies at all stages in a company’s lifecycle, from seed stage through IPO. The firm's long track record of successful investing includes more than 210 portfolio company IPOs and more than 360 acquisitions. For additional information, visit www.nea.com.
About Shuttle Fund
Shuttle is a global investment fund focused on disruptive blockchain-based technologies. Decentralized blockchain technologies are redefining the way people and business interact, and Shuttle has a successful track record of investing into and advising fundamentally disruptive blockchain-based platforms across multiple domains. For more information, visit www.shuttlefund.com.
About Sanofi Ventures
Sanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies that are of strategic interest to Sanofi. Among these areas are rare diseases, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. For more information, visit www.sanofiventures.com.
About Green Bay Ventures
Green Bay Ventures (GBV) is a San Francisco based venture capital firm investing at the intersection of technology and large markets including manufacturing, energy, transportation, logistics, real estate and telecommunications. GBV focuses on a small number of high conviction opportunities and works side-by-side with a select group of visionary entrepreneurs to create breakthrough technologies and transform industries. For more information, visit www.greenbayventures.com.
NEW YORK, Dec. 05, 2017 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ:OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TAK-935/OV935 for the treatment of Dravet syndrome, a severe and rare form of childhood epilepsy that typically presents during the first year of life. Takeda Pharmaceutical Company Limited and Ovid formed a global collaboration to develop and commercialize TAK-935/OV935 for the treatment of developmental and epileptic encephalopathies in January 2017.
Dravet syndrome is classified as a developmental and epileptic encephalopathy, a group of rare epilepsies that cause significant morbidities and can worsen over time. Children with Dravet syndrome experience frequent seizures, loss of muscle control, cognitive deficits and, in approximately 10 percent of cases, death before the age of 12 years. Moreover, in those who survive into adulthood, their long-term intellectual development and seizure outcomes are typically extremely poor.
“We are pleased by the FDA’s decision to grant orphan drug designation to TAK-935/OV935 for the treatment of Dravet syndrome, a severe and debilitating disease,” said Dr. Emiliangelo Ratti, head of Takeda’s Neuroscience Therapeutic Area Unit. “This designation is a significant step forward in researching a potential treatment option for people living with Dravet syndrome for whom therapeutic options are severely limited, and an important milestone for this investigational molecule.”
TAK-935/OV935 is a potent, highly-selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H). It is believed that CH24H is involved in over-activation of the glutamatergic pathway, which has been shown to play a role in the initiation and spread of seizure activity. To Ovid and Takeda’s knowledge, TAK-935/OV935 is the only molecule with this mechanism of action in clinical development.
“We believe that TAK-935/OV935, with its novel mechanism of action, has the potential to be an innovative treatment for people with rare epilepsies, such as Dravet syndrome,” said Matthew During M.D., DSc, FACP, FRACP, president and chief scientific officer of Ovid Therapeutics. “We have rapidly advanced this program into a Phase 1b/2a clinical trial and anticipate data in 2018. We look forward to continuing our work with Takeda to bring this potentially transformative therapy to patients.”
Orphan drug designation is intended to facilitate and expedite drug development for rare diseases for which there are no current treatments available. It also provides substantial benefits to the sponsor, including the potential for tax credits for clinical development costs, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.
About Dravet Syndrome
Dravet syndrome is a severe form of childhood epilepsy that typically presents during the first year of life. It is believed to be largely caused by mutations in the SCN1A gene. Children experience frequent seizures, loss of muscle control, cognitive deficits and, in approximately 10 percent of cases, death before the age of 12 years. While some patients may survive into adulthood, their long-term intellectual development and seizure outcomes are typically extremely poor. The incidence of Dravet syndrome in the United States ranges from 1 in 15,700 to 1 in 20,900 births. Patients are frequently treated with combinations of classic anti-epileptic drugs, none of which are particularly effective. No drugs have been approved specifically for the treatment of Dravet syndrome in the United States and only one drug, the anticonvulsant stiripentol, has been approved in Europe.
Dravet syndrome is one of several disorders which together are designated as developmental and epileptic encephalopathies. This group includes epilepsy syndromes associated with severe cognitive and behavioral disturbances. The International League Against Epilepsy (ILAE) defines an epileptic encephalopathy as a condition in which “the epileptiform EEG abnormalities themselves are believed to contribute to a progressive disturbance in cerebral function.”
These epilepsies cause significant morbidities for patients beyond what might be expected from the known underlying pathology alone and can worsen over time. Developmental and epileptic encephalopathies typically present early in life and are often associated with severe cognitive and developmental impairment in addition to frequent treatment-resistant seizures throughout the person’s lifetime. These disorders vary in age of onset, developmental outcomes, etiologies, neuropsychological deficits, electroencephalographic (EEG) patterns, seizure types and prognosis.
About TAK-935/OV935
TAK-935/OV935, which is being studied in developmental and epileptic encephalopathies, is a potent, highly-selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H). CH24H is predominantly expressed in the brain, where it plays a central role in cholesterol homeostasis. CH24H converts cholesterol to 24S-hydroxycholesterol (24HC), which then exits the brain into the blood plasma circulation. Glutamate is one of the main neurotransmitters in the brain and has been shown to play a role in the initiation and spread of seizure activity. Recent literature indicates 24HC is involved in over-activation of the glutamatergic pathway through modulation of the NMDA channel, implying its potential role in central nervous system diseases such as epilepsy. To Ovid and Takeda’s knowledge, TAK-935/OV935 is the only molecule with this mechanism of action in clinical development.
TAK-935/OV935 has been tested in preclinical models to provide data to support the advancement of the drug into human clinical studies in patients suffering from rare epilepsy syndromes. A novel proprietary PET ligand, developed by Takeda and Molecular Neuroimaging, LLC (MNI), has been used to determine target enzyme occupancy of TAK-935/OV935 in the brain. In addition, the effect of TAK-935/OV935 on CH24H enzyme activity in the brain has been assessed by following measurable reductions in the plasma concentration of 24HC.
TAK-935/OV935 has completed four Phase 1 clinical studies, which have assessed tolerability and target engagement at doses believed to be therapeutically relevant. TAK-935/OV935 is being co-developed by Ovid and Takeda Pharmaceutical Company Limited.
About Ovid Therapeutics
Ovid Therapeutics (NASDAQ:OVID) is a New York-based biopharmaceutical company using its BoldMedicine™ approach to develop therapies that transform the lives of patients with rare neurological disorders. Ovid’s drug candidate, OV101, is currently in development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid initiated the Phase 2 STARS trial of OV101 in people with Angelman syndrome in 2017 and completed a Phase 1 trial in adolescents with Angelman syndrome or Fragile X syndrome. Ovid is also developing OV935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of epileptic encephalopathies and in August 2017 initiated a Phase 1b/2a trial of OV935.
For more information on Ovid, please visit http://www.ovidrx.com/.
Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding the progress, timing, scope and results of clinical trials for Ovid’s product candidates, the reporting of clinical data regarding Ovid’s product candidates, and the potential use of TAK-935/OV935 to treat rare epilepsies. You can identify forward-looking statements because they contain words such as “will,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2017, under the caption “Risk Factors.” Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
Contacts
Investors:
Burns McClellan
Steve Klass, 212-213-0006
Sklass@burnsmc.com
Media:
Pure Communications, Inc.
Katie Engleman, 910-509-3977
katie@purecommunicationsinc.com
Company bolsters leadership team with several executive appointments
Cambridge, Mass., November 15, 2017 - Inozyme Pharma, Inc., a biotechnology company developing novel medicines to treat rare diseases of calcification, affecting soft tissues and bone, today announced it has raised a $49 million Series A financing. The financing was led by Longitude Capital, and included participation from New Enterprise Associates (NEA), Novo Ventures and Sanofi Ventures.
“Our mission is to develop potentially disease-modifying therapies to help children who are affected with rare, but severe and debilitating disorders of metabolism. These patients have very poor treatment options,” said Axel Bolte, chief executive officer and co-founder of Inozyme Pharma. “We have attracted a premier syndicate of healthcare investors who are committed to helping us achieve our goal, and this funding positions us well to advance our therapeutic approach.”
Inozyme Pharma was founded in 2016 with technology developed in the laboratory of Demetrios Braddock, M.D., Ph.D., and licensed from Yale University. The company will use the proceeds from this financing to advance its lead enzyme replacement therapy for the treatment of Generalized Arterial Calcification of Infancy (GACI) and Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2) into the clinic. These disorders are characterized by mineral imbalances that lead to over calcification of soft tissues and under mineralization of bone.
“Inozyme Pharma’s deep understanding of the biology of calcification will be used to develop new medicines that have the potential to drastically improve the standard-of-care,” said Reinaldo Diaz, venture partner at Longitude Capital, and a member of Inozyme Pharma’s board of directors. “With strong foundational intellectual property and a multidisciplinary team of experts, the company is well-positioned to advance new therapies for underserved populations.”
In addition to the Series A financing, Inozyme Pharma has also expanded its experienced leadership team of industry veterans, including:
- Axel Bolte, president, chief executive officer, and co-founder Axel Bolte brings 20 years of experience in biotech and healthcare venture capital, with considerable background in rare diseases, to Inozyme Pharma. Mr. Bolte has served as a board member of various private and public companies.
- Henric Bjarke, senior vice president and chief operating officer Henric Bjarke’s 20 years in pharma and biotech bring a wealth of experience in metabolic and rare diseases to Inozyme Pharma. Having previously served as vice president and therapeutic area head for the metabolic business unit at Alexion Pharmaceuticals, Mr. Bjarke was responsible for asfotase-alfa for the treatment hypophosphatasia and other development programs.
- Steven Jungles, senior vice president and chief technical operations officer Steven Jungles offers 20 years of industry experience to the Inozyme Pharma leadership team. Before joining Inozyme Pharma, Mr. Jungles served as senior vice president, technical operations at Ultragenyx Pharmaceutical, vice president of contract manufacturing and supply chain at BioMarin Pharmaceuticals and worked at the Harvard Gene Therapy Initiative.
- Eric Yuen, senior vice president and chief medical officer Erin Yuen brings 24 years of experience in academia and the biopharmaceutical industry. Dr. Yuen has developed biologics and small molecules for a variety of diseases, including rare genetic, neurology, psychiatry, pain, and oncology disorders. Dr. Yuen has held several senior positions at Merck, Johnson & Johnson, Ultragenyx and BioClin.
- Ruhi Ahmed, vice president of regulatory and government affairs ; Ruhi Ahmed’s 15 years of experience in regulatory affairs include drug development and life cycle management of programs from the preclinical to the commercial stage. Prior to Inozyme Pharma, Dr. Ahmed held positions at Ultragenyx and BioMarin Pharmaceuticals where she worked on the MPS, XLH and PKU programs.
- Stephen Basso, vice president of finance Stephen Basso has 25 years of experience in life sciences and financial services that he brings to Inozyme Pharma. Prior to joining the company, Mr. Basso was vice president of corporate finance at Alexion, where he supported the development and commercialization of Soliris, Strensiq and Kanuma.
In addition, Inozyme has appointed the following seasoned directors to its board:
- Joseph Schlessinger, chairman and co-founder, Yale University • Reinaldo Diaz, Longitude Capital
- Ed Mathers, New Enterprise Associates
- Martin Edwards, Novo Holdings A/S
- Axel Bolte, president, chief executive officer and co-founder
About GACI and ARHR2
GACI is an ultra-rare, autosomal recessive orphan disease affecting infants. It is caused by loss-of-function mutations in either the ENPP1 or ABCC6 gene, and results in low circulating levels of pyrophosphate (PPi) and calcification of medium and large arteries and heart. GACI presents as a crisis within the first week of life and is associated with high mortality rates. The majority of patients are likely to die within the first year of life. If affected children survive past six months, they are likely to continue living but develop ARHR2, a rare skeletal disorder characterized by low levels of serum PPi, which can result in rickets, repeated fractures of the long bones, rachitic skeletal deformities, and impaired growth and development.
About Inozyme Pharma, Inc.
Inozyme Pharma is a biotechnology company committed to developing novel medicines for the treatment of rare diseases characterized by mineral imbalances, which lead to over calcification of soft tissues and under mineralization of bone. The company was founded in 2016 with technology licensed from Yale University.
MEDIA CONTACT:
Lauren Barbiero
W2Opure
lbarbiero@w2ogroup.com
+1 (646) 564-2156
SEATTLE and SOUTH SAN FRANCISCO, Calif., Oct. 19, 2017 (GLOBE NEWSWIRE) -- Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation for G100, Immune Design’s investigational intratumoral therapy, for the treatment of follicular non-Hodgkin’s lymphoma.
The EMA orphan drug designation is assigned to products targeting the treatment of rare diseases, which are defined as having a prevalence of not more than 5 in 10,000 people in the European Union (EU). This designation provides the sponsor with certain benefits, including protocol assistance, reduced fees for regulatory activities and up to 10 years of market exclusivity in the EU upon marketing approval for the designated indication.
G100 has also been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of follicular non-Hodgkin's lymphoma.
G100 is a product candidate from Immune Design's GLAAS® discovery platform. It contains a potent synthetic small molecule toll-like receptor-4 (TLR-4) agonist, Glucopyranosyl Lipid A (GLA), and is the lead product candidate in Immune Design’s Antigen Agnostic approach. G100 activates innate and adaptive immunity in the tumor microenvironment to generate an immune response against the tumor's preexisting diverse set of antigens. A growing set of clinical and preclinical data have demonstrated the ability of G100 to activate tumor-infiltrating lymphocytes, macrophages and dendritic cells, and promote antigen-presentation and the recruitment of T cells to the tumor. The induction of local and systemic immune responses has been shown in preclinical studies to result in local and abscopal (shrinking of tumors outside the scope of the localized treatment) tumor control. Currently, G100 is being evaluated as both a monotherapy (with local radiation) and in combination with Merck’s anti-PD-1 agent, pembrolizumab, pursuant to a clinical collaboration with Merck, in a randomized Phase 1/2 clinical trial in patients with follicular non-Hodgkin’s lymphoma.
About Immune Design
Immune Design is a clinical-stage immunotherapy company employing next-generation in vivo approaches to enable the body's immune system to fight disease. The company's technologies are engineered to activate the immune system's natural ability to generate and/or expand antigen-specific cytotoxic T cells, while also enhancing other immune effectors, to fight cancer and other chronic diseases. CMB305 and G100, the two leading product candidates focused in cancer immunotherapy, are the first products from Immune Design’s two separate discovery platforms targeting dendritic cells in vivo, ZVex® and GLAAS®. Both ZVex and GLAAS also have potential
applications in infectious disease and allergy as demonstrated by ongoing pharmaceutical collaborations. Immune Design has offices in Seattle and South San Francisco. For more information, please visit www.immunedesign.com.
Media Contact
Julie Rathbun
Rathbun Communications
julie@rathbuncomm.com
206-769-9219
Investor Contact
Shari Annes
Annes Associates
sannes@annesassociates.com
650-888-0902
NEW YORK, Oct. 16, 2017 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ:OVID), a biopharmaceutical company committed to developing medicines for patients with rare neurological diseases, today announced new positive preclinical data on OV101 that shows normalization of behavioral abnormalities that resemble those seen in people with Fragile X syndrome. The improvements seen with OV101, a novel agonist of the extrasynaptic GABA receptor, were consistent across multiple behavioral measures in a model of Fragile X syndrome. The data were presented at the 18th International Fragile X and Related Neurodevelopmental Disorders Workshop.
“Treatment with OV101 results in significant behavioral improvements that are consistent across all behavioral endpoints tested,” said Matthew During, M.D., DSc, FACP, FRACP, president and chief scientific officer of Ovid Therapeutics. “We designed this study to deepen our understanding of the mechanisms of action and potential benefit of OV101 to treat patients with Fragile X syndrome.”
In the study, researchers demonstrated that acute administration of 0.5 mg/kg of OV101 to Fmr1 knockout mice fully normalized behavioral abnormalities relevant to Fragile X syndrome (hyperactivity, anxiety, irritability and aggression, and restricted and repetitive behaviors). All effects were highly statistically significant (p < 0.001). It is believed that symptoms of Fragile X syndrome are a result of disrupted tonic inhibition, the key to the brain's ability to discriminate signal from noise. The results presented indicate that by specifically targeting the delta (δ)-subset of GABA receptors, OV101 may be able to alleviate symptoms of Fragile X syndrome by modulating the GABA pathway and restoring tonic inhibition.
“We have built a strong foundation in scientific analysis, translational medicine, drug development and regulatory capabilities. This study builds upon this foundation and underscores the potential role of OV101 in modulating tonic inhibition, an important underlying mechanism in certain neurodevelopmental disorders,” said Amit Rakhit M.D., MBA, chief medical and portfolio officer of Ovid Therapeutics. “Together with the recent FDA orphan drug designation for OV101 for the treatment of Fragile X syndrome, this data is another important step in our disciplined strategy to develop OV101 as a potential first in class compound for Fragile X syndrome.”
About OV101
OV101 (gaboxadol) is believed to be the only delta (δ)-selective GABAA receptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has been demonstrated in laboratory studies and animal models to selectively activate the δ-subunit of GABAA receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through tonic inhibition.
Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and relieve several of the symptoms of these disorders. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. To date, gaboxadol has been tested in over 4,000 patients (approximately 950 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles.
The FDA granted orphan drug designation for OV101 for the treatment of both Angelman syndrome and Fragile X syndrome. The United States Patent and Trademark Office has granted Ovid two patents directed to methods of treating Angelman syndrome using OV101. The issued patents expire in 2035, without regulatory extensions.
About Fragile X Syndrome
Fragile X syndrome is the most common inherited form of intellectual disability and autism, with a prevalence of 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females in the United States. Individuals with Fragile X syndrome often have a range of behavioral challenges, such as cognitive impairment, anxiety, mood swings, hyperactivity, attention deficit, poor sleep, self-injury and heightened sensitivity to various stimuli, such as sound. Additionally, individuals with Fragile X syndrome are prone to comorbid medical issues including seizures and sleep disturbance. Fragile X syndrome results from mutations in the FMR1 gene, which blocks expression of the Fragile X Mental Retardation Protein (FMRP), an important protein in GABA synthesis. There are no FDA-approved therapies for Fragile X syndrome, and treatment primarily consists of behavioral interventions and pharmacologic management of symptoms.
In studies of individuals with Fragile X syndrome and in experimental models, extrasynaptic GABA levels are abnormally reduced, and there is also dysregulation of GABA receptors. This ultimately contributes to a decrease in tonic inhibition, causing the brain to become inundated with signals and lose the ability to separate background noise from critical information.
About Ovid Therapeutics
Ovid Therapeutics (NASDAQ:OVID) is a New York-based biopharmaceutical company using its BoldMedicine™ approach to develop therapies that transform the lives of patients with rare neurological disorders. Ovid’s drug candidate, OV101, is currently in development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid has initiated the Phase 2 STARS trial of OV101 in adults with Angelman syndrome and a Phase 1 trial in adolescents with Angelman syndrome or Fragile X syndrome. Ovid is also developing OV935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of rare epileptic encephalopathies and has initiated a Phase 1b/2a trial of OV935.
For more information on Ovid, please visit http://www.ovidrx.com/.
Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding progress, timing, scope and results of clinical trials for Ovid’s product candidates, the reporting of clinical data regarding Ovid’s product candidates, the development of OV101 as a potential first in class compound for Fragile X syndrome and the potential use of TAK-935/OV935 to treat rare epilepsies. You can identify forward-looking statements because they contain words such as “will,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended June 30, 2017, under the caption “Risk Factors.” Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
Contacts
Investors:
Burns McClellan
Steve Klass, 212-213-0006
Sklass@burnsmc.com
Media:
Pure Communications, Inc.
Katie Engleman, 910-509-3977
katie@purecommunicationsinc.com
- CMB305 Monotherapy vs. Placebo in the Maintenance Setting
- PFS Analysis for Potential Full Approval as Early as 24 Months After Study Start
- Conference Call and Webcast Tomorrow at 5:30am PT/8:30am ET
SEATTLE and SOUTH SAN FRANCISCO, Calif., Oct. 16, 2017 (GLOBE NEWSWIRE) -- Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, today announced that based on productive discussions with the U.S. Food and Drug Administration (FDA), it plans to initiate a pivotal Phase 3 trial to support a Biologics License Application (BLA) for CMB305, a novel cancer vaccine, in patients with synovial sarcoma.
The randomized Phase 3 trial will evaluate CMB305 monotherapy vs. placebo in patients with NY-ESO-1+ locally advanced unresectable or metastatic synovial sarcoma, a sub type of soft tissue sarcoma, who have no evidence of progression after first-line chemotherapy. Immune Design intends to start the study in mid-2018 and enroll 248 patients aged twelve and older. Patients will be randomized 1:1 to receive either CMB305 monotherapy or placebo. The trial will have progression free survival (PFS) followed by overall survival (OS) as co-primary endpoints. If the PFS endpoint is successful, the FDA offered that it may support full approval of CMB305. Depending on the rate of events, final PFS analysis may occur as early as 24 months from the first patient dosed.
About Synovial Sarcoma
Soft tissue sarcomas are malignancies that arise from the soft tissues of the body, such as tissues that connect, support and surround other body structures including muscle, fat, blood vessels, nerves, tendons and the lining of joints. Synovial sarcoma is a sub type of soft tissue sarcoma where 70% of diagnoses occur in patients under 40 years old, is associated with a high risk of recurrence, and has been shown to have high expression of the NY-ESO-1 tumor antigen. The primary treatment for patients with locally advanced, unresectable or metastatic synovial sarcoma typically consists of an anthracycline-based chemotherapy regimen administered alone or in combination with other agents. Following disease progression after first line systemic therapy, treatment options are limited and median overall survival rates have been reported to be approximately 12 months. In connection with the planned Phase 3 study for CMB305 monotherapy, the FDA has agreed with Immune Design that synovial sarcoma patients constitute an unmet medical need.
About CMB305
CMB305 is an investigational prime-boost vaccine approach against NY-ESO-1-expressing tumors, designed to generate an integrated, anti-NY-ESO-1 immune response in vivo via a targeted, specific interaction with dendritic cells, a mechanism of action Immune Design believes differs from traditional cancer vaccines. CMB305 is being evaluated in soft tissue sarcoma patients in ongoing Phase 1 monotherapy and Phase 2 combination studies. Immune Design has received Orphan Drug Designation for CMB305 from the FDA for the treatment of soft tissue sarcoma, as well as from the FDA and European Commission for each of the components of CMB305 for the treatment of soft tissue sarcoma.
Conference Call Information
Immune Design will host a conference call and live audio webcast tomorrow, October 17, at 5:30 a.m. Pacific time / 8:30 a.m. Eastern time to discuss the CMB305 development strategy and pivotal trial design.
To participate in the conference call, please dial 844-266-9538 for domestic callers and 216-562-0391 for international callers and provide the conference ID 9299539, or access the listen-only live webcast by visiting the investor relations section of the company website at http://ir.immunedesign.com/events.cfm.
A telephone replay of the call will be available for five days by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference code: 9299539. An archived copy of the webcast will be available on Immune Design's website beginning approximately two hours after the conference call and will be available for at least 30 days after the conference call.
About Immune Design
Immune Design is a clinical-stage immunotherapy company employing next-generation in vivo approaches to enable the body's immune system to fight disease. The company's technologies are engineered to activate the immune system's natural ability to generate and/or expand antigen-specific cytotoxic T cells, while also enhancing other immune effectors, to fight cancer and other chronic diseases. CMB305 and G100, the two leading product candidates focused in cancer immunotherapy, are the first products from Immune Design’s two separate discovery platforms targeting dendritic cells in vivo, ZVex® and GLAAS®. Both ZVex and GLAAS also have potential applications in infectious disease and allergy as demonstrated by ongoing pharmaceutical collaborations. Immune Design has offices in Seattle and South San Francisco. For more information, please visit www.immunedesign.com.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Immune Design's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause our clinical development programs, future results or performance to differ significantly from those expressed or implied by the forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the design, progress, timing, scope and potential results of the pivotal Phase 3 clinical trial of CMB305 in synovial sarcoma patients, the possibility that PFS data will be sufficient to support regulatory approval and the timing of the PFS analysis. Many factors may cause differences between current expectations and actual results including unexpected safety or efficacy data, clinical trial site activation or enrollment rates that are slower than expected, changes in expected or existing competition, changes in the regulatory environment, the uncertainties and timing of the regulatory approval process and unexpected litigation or other disputes. Other factors that may cause Immune Design's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Immune Design's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Immune Design assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
Media Contact
Julie Rathbun
Rathbun Communications
julie@rathbuncomm.com
206-769-9219
Investor Contact
Shari Annes
Annes Associates
sannes@annesassociates.com
650-888-0902
- Phase 2 Interim Analysis Data Presented at ESMO 2017 Congress
SEATTLE and SOUTH SAN FRANCISCO, Calif., Sept. 08, 2017 (GLOBE NEWSWIRE) -- Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, today announced that an interim analysis of its ongoing, randomized Phase 2 trial showed that NY-ESO-1+ soft tissue sarcoma (STS) patients receiving the combination of CMB305 and Genentech’s checkpoint inhibitor, atezolizumab (TECENTRIQ®), experienced greater clinical benefit and immune response than those receiving atezolizumab alone. The data will be presented in a poster discussion session at the European Society of Medical Oncology (ESMO) 2017 Congress, September 8-12, 2017 in Madrid, Spain.
This fully enrolled trial is evaluating the safety, immunogenicity and efficacy of CMB305 in combination with atezolizumab (C+A, n=45), or atezolizumab alone (A, n=43), in a total of 88 patients with locally advanced, relapsed, or metastatic NY-ESO-1+ synovial sarcoma or myxoid/round-cell liposarcoma. Data to be presented at ESMO summarize patients evaluated in an interim analysis in a data cut as of July 21, 2017, with analysis divided into two groups: pre-specified interim analysis (n=36) and full study population (n=88).
Patient Characteristics:
Patients receiving CMB305 plus atezolizumab have more advanced disease than those receiving atezolizumab alone, including:
- Metastatic Disease: 98% (C+A) vs. 79% (A)
- ≥2 prior lines of systemic anti-cancer therapy: 61% (C+A) vs. 49% (A)
- ≥2 lesions at time of study entry: 96% (C+A) vs. 84% (A)
- Grade 3 disease at diagnosis: 47% (C+A) vs. 33% (A)
Greater Clinical Benefit with Combination of CMB305 and Atezolizumab:
Interim analysis data (n=36) show that patients receiving CMB305 plus atezolizumab experienced greater clinical benefit than those receiving atezolizumab alone.
- Disease Control Rate (Partial Responses (PR) + Stable Disease (SD)): 61%, including 1 PR (C+A) vs. 28% with no PRs (A)
- Median Progression Free Survival (PFS): 2.6 months (C+A) vs. 1.4 months (A)
- Time to Next Treatment (TTNT): 9 months (C+A) vs. 6.3 months (A)
- Overall survival: as of the collection date, overall survival data are immature (median duration of observation is less than six months); Immune Design intends to present survival data in 2018 once all patients approach at least one year of follow up.
In the full study population (n=88), the trend of greater clinical benefit on the combination arm remains consistent for the entire patient population:
- Disease Control Rate: 57% (3 PRs total, 1 unconfirmed) (C+A) vs. 38% (0 responses) (A)
More Robust Immune Response with Combination of CMB305 and Atezolizumab:
Patients in the full study population (n=88) who received the combination of CMB305 and atezolizumab demonstrated stronger anti-NY-ESO-1 immune responses compared to those receiving atezolizumab alone (samples evaluable from (n=60/88)), including:
- Induced anti-NY-ESO-1 T cells: 52% (C+A) vs. 17% (A)
- Induced anti-NY-ESO-1 antibodies: 52% (C+A) vs. 0% (A)
- Induced antigen spreading: 19% (C+A) vs. 0% (A)
Biomarker Analysis Shows Continued Link Between Induced Immune Response and Clinical Benefit:
In an exploratory analysis, a trend towards improved overall survival was observed in patients with an induced immune response (T cells or antibodies) who received CMB305 plus atezolizumab.
- Induced anti-NY-ESO-1 T cells: 78% reduction in mortality rate, as compared to patients without induced T cells [HR=0.22, log-rank p value=0.043]
- Induced anti-NY-ESO-1 antibodies: 87% reduction in mortality rate, as compared to patients without induced antibodies [HR=0.13, log-rank p value=0.025]
- This trend of improved overall survival in patients with induced immune response was not observed in the atezolizumab-only arm.
In addition, pretreatment tumor biopsies available from 70 patients show both an absent or very low level of PD-L1 expression and CD8 T cell infiltration, further supporting that these subtypes of STS are "cold" tumors.
Positive Safety Profile with Combination of CMB305 and Atezolizumab:
This combination was observed to be well tolerated, and there were no new safety signals in either arm.
“We believe the greater clinical benefit and more robust immune response shown in the combination study arm supports the hypothesis that the combination of an appropriately-designed, next-generation cancer vaccine such as CMB305 with a checkpoint inhibitor is important to produce clinical benefit in a cold tumor such as STS, where checkpoint inhibitors otherwise may have limited efficacy,” said Carlos Paya, M.D., Ph.D., President and Chief Executive Officer of Immune Design. “In addition, we are pleased to observe the trend towards longer overall survival in patients with an induced anti-NY-ESO-1 immune response, further supporting the positive CMB305 monotherapy data we presented at ASCO in June.”
Atezolizumab is a monoclonal antibody being developed by Genentech, a member of the Roche Group, and is designed to target and bind to a protein called PD-L1 (programmed death ligand-1). The trial is being conducted pursuant to a clinical collaboration with Genentech. TECENTRIQ® is a registered trademark of Genentech.
The ESMO Poster Discussion session presentation details are as follows:
A Phase 2 Study of CMB305 and Atezolizumab in NY-ESO-1+ Soft Tissue Sarcoma: Interim Analysis of Immunogenicity, Tumor Control and Survival
Abstract # 1480PD
Session Title: Sarcoma Poster Discussion Session
Date: Monday, Sept. 11, 2017
Time: 11 a.m. – 12:30 p.m.
Location: Bilbao Auditorium
Poster Presenter: Dr. Sant Chawla
Poster Discussant: Dr. Robert Maki
About CMB305
CMB305 is a prime-boost vaccine approach against NY-ESO-1-expressing tumors, designed to generate an integrated, anti-NY-ESO-1 immune response in vivo via a targeted, specific interaction with dendritic cells, a mechanism of action Immune Design believes differs from traditional cancer vaccines. CMB305 is being evaluated in soft tissue sarcoma patients in ongoing Phase 1 monotherapy and Phase 2 combination studies. Immune Design has received Orphan Drug Designation for CMB305 from the U.S. Food and Drug Administration (FDA) for the treatment of soft tissue sarcoma, as well as from the FDA and European Medicines Agency for each of the components of CMB305 for the treatment of soft tissue sarcoma.
About Immune Design
Immune Design is a clinical-stage immunotherapy company employing next-generation in vivo approaches to enable the body's immune system to fight disease. The company's technologies are engineered to activate the immune system's natural ability to generate and/or expand antigen-specific cytotoxic T cells, while also enhancing other immune effectors, to fight cancer and other chronic diseases. CMB305 and G100, the two leading product candidates focused in cancer immunotherapy, are the first products from Immune Design’s two separate discovery platforms targeting dendritic cells in vivo, ZVex® and GLAAS®. Both ZVex and GLAAS also have potential applications in infectious disease and allergy as demonstrated by ongoing pharmaceutical collaborations. Immune Design has offices in Seattle and South San Francisco. For more information, please visit www.immunedesign.com.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Immune Design's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause our clinical development programs, future results or performance to differ significantly from those expressed or implied by the forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, scope and results of clinical trials, the association of data with treatment outcomes, and the timing and likelihood of obtaining regulatory approval of Immune Design's product candidates. Many factors may cause differences between current expectations and actual results including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, the uncertainties and timing of the regulatory approval