Strategic Wins
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
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
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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.”
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.”
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.”
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.
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.
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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
- 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
- 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
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
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- 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.
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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.
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
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.”
CAMBRIDGE, Massachusetts, November 20, 2015 – Proteostasis Therapeutics, Inc. (PTI), acompany developing small molecule therapeutics to treat diseases caused by defects in protein processing, announced today the expansion of its cystic fibrosis (CF) drug candidate pipeline to include the addition of a novel triple combination therapy of PTI’s own CFTR amplifiers, correctors and potentiators. PTI’s testing has shown that a triple combination comprising a proprietary corrector and potentiator, currently in late lead optimization stage, and one of PTI’s CFTR amplifiers, can restore the activity of mutant F508del CFTR protein to 80% of normal activity in Ussing chamber assays.
The components of the triple combination were developed internally using the Company’s proprietary Disease-Relevant Translation, or DRT™, platform. The screening assay was optimized to identify novel CFTR modulators that show functional synergy with the Company’s lead drug candidate, PTI-428, which belongs to a novel class of CFTR modulators the Company refers to as “amplifiers”, while restoring chloride currents above levels achieved by existing commercially available products. In Ussing chamber assays, one PTI potentiator, PTI-P271, has shown comparable efficacy with Vertex Pharmaceuticals’ (Vertex) potentiator, ivacaftor, and did not cause F508del CFTR protein destabilization under chronic administration conditions when combined with a PTI corrector. Also in Ussing chamber assays, one PTI corrector, PTI-C1811, has been shown to restore at least 140% of CFTR functional levels relative to Vertex’s corrector lumacaftor, which is currently marketed together with ivacaftor as Orkambi™.
Based on the data generated in the human bronchial epithelial (hBE) cells homozygous for the F508del mutation, the Company believes that the combined use of the three molecules has the potential to restore mutant CFTR function in CF patients homozygous for the F508del mutation to approximately 80% of normal activity. Further, PTI-C1811 and PTI-P271 combined have demonstrated higher levels of CFTR function in vitro than Orkambi™.
“The unique screening set-up allowed us to identify novel chemical moieties with corrector and potentiator properties that act synergistically with our CFTR amplifiers across several CFTR mutation classes” said Meenu Chhabra, President and Chief Executive Officer of PTI. “We are very pleased with the rapid advancement of all of our CF programs, and are confident that we will continue to build on our promising amplifier program to expand the range of treatment options for most CF patients.”
PTI is advancing its CFTR amplifier PTI-428 as its lead clinical development candidate for the treatment of CF and expects to file an IND with the FDA by the end of 2015. The PTI correctors and potentiators are expected to enter clinical development by the middle of 2017.
About Cystic Fibrosis
Cystic fibrosis is a genetic disorder affecting approximately 70,000 to 100,000 people worldwide.
Improvement in disease management protocols and approval of new drugs to treat the symptoms have extended the life expectancy for CF patients. However, CF remains an incurable disease that leads to death.
About Proteostasis Therapeutics
Proteostasis Therapeutics, Inc. (PTI) is developing disease-modifying therapeutics for diseases of protein processing. By combining the DRT™ platform, a phenotypic screening approach based on the use of functionally pertinent cellular assays, with state of the art medicinal chemistry tools, PTI generates highly selective drug candidates that modulate the proteostasis imbalance in the cell. In addition to its multiple wholly-owned programs in CF, PTI has formed collaborations with Biogen Inc. to research and identify therapeutic candidates for neurodegenerative disease and with Astellas Pharma Inc. to research and identify therapies targeting the Unfolded Protein Response (UPR) pathway.
Investor Contact:
Janet Smart
Proteostasis Therapeutics, Inc.
617-225-0096 Ext. 2128
Janet.Smart@proteostasis.com
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
DUBLIN and SAN DIEGO, May 12, 2014 /PRNewswire/ --
- Acquisition of Lumena Pharmaceuticals, a biopharmaceutical company with late stage rare disease pipeline assets
- Adds to Shire's rare diseases portfolio and leverages this expertise, and is a perfect combination with Shire's already strong Gastrointestinal (GI) presence
- Adds LUM001 in Phase 2 development for four rare and devastating hepatic diseases, two pediatric and two adult with a potential 2016 approval and LUM002 a Phase 2-ready candidate for the treatment of non-alcoholic steatohepatitis (NASH)
- Very attractive opportunity to develop treatments for significant unmet need in rare cholestatic liver diseases as well as a treatment for non-alcoholic steatohepatitis (NASH)
- Shire will acquire Lumena Pharmaceuticals for an upfront payment of $260 million in cash, plus a payment for net cash at closing, and near-term contingent milestone payments related to ongoing clinical trials
- These two compounds, and Shire's full portfolio, will be discussed in more detail at an Investor Day later in 2014.
Shire plc (LSE: SHP,NASDAQ: SHPG) and Lumena Pharmaceuticals, Inc., a biopharmaceutical company with rare disease pipeline assets, announce the acquisition of Lumena Pharmaceuticals by Shire.
Chief Executive of Shire, Flemming Ornskov, MD comments:
"Our pipeline and strategic focus on rare diseases is even further strengthened with the acquisition of Lumena Pharmaceuticals, which also complements our strong GI presence. These attractive potential treatments may offer new hope to patients with rare cholestatic liver disease and further contribute to Shire's future growth. We are excited by the possibilities of these new assets in liver disease. We have the resources, the infrastructure and the operating capacity to invest in these new potential growth drivers which add further value to Shire's innovative pipeline."
President and CEO of Lumena Pharmaceuticals, Mike Grey comments:
"I believe that this transaction is a significant win for all parties involved, especially the patients, and the future of LUM001 as a treatment for rare cholestatic liver diseases looks brighter than ever. Shire has deep rare disease experience, a global infrastructure, and the commercial expertise to deliver LUM001 to patients around the world. The Lumena team will work closely with Shire to finish the ongoing Phase 2 clinical programs as part of our commitment to the patient populations we have championed since we formed Lumena Pharmaceuticals."
Strategic rationale and background on Lumena Pharmaceuticals
The acquisition of Lumena strengthens Shire's already valuable and robust pipeline. It complements Shire's strategic focus on Rare Diseases and provides a future growth path for Shire's Gastrointestinal business, which generated revenues of over $800 million in 2013. In acquiring Lumena, Shire is gaining experience in liver disease with the opportunity to leverage its existing GI commercial infrastructure. In addition, there is a good fit with Shire's recent acquisition of Fibrotech, which has brought pipeline programs to address unmet patient need in other fibrotic conditions including renal impairment.
Lumena Pharmaceuticals brings to Shire two new oral therapeutic compounds; LUM001, in Phase 2 with four potential orphan indications and LUM002, ready to enter Phase 2 later in 2014.
LUM001 and LUM002 are both inhibitors of the apical sodium-dependent bile acid transporter (ASBT), which is primarily responsible for recycling bile acids from the intestine to the liver. Blocking bile acid transport with ASBT inhibitors reduces bile acid absorption and has the potential to improve liver function and relieve disease symptoms (such as extreme itching associated with cholestatic liver diseases), and may slow disease progression.
These rare cholestatic liver diseases are primarily treated by hepatologists and gastroenterologists and can be covered by a small, specialty sales force consistent with Shire's model.
Shire does not expect the acquisition of Lumena to result in a change to its previously stated earnings guidance for 2014.
About LUM001
LUM001 is a novel, once-daily, orally-administered, potent and selective ASBT inhibitor that works by preventing recycling of bile acids back to the liver and is thought to reduce bile acid accumulation, improve liver function and potentially relieve the extreme itching associated with cholestatic liver disease.
LUM001 is currently in Phase 2 clinical development for four rare cholestatic liver disease indications; two pediatric and two adult with a potential 2016 launch. These potential indications are: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), primary biliary cirrhosis (PBC) and primary sclerosing cholangitis (PSC).
Some of the key characteristics of cholestatic liver diseases are elevated bile acids, leading to progressive liver damage that can cause liver failure, and pruritus, or severe itching. Pruritus is generally the most debilitating symptom afflicting children and adults with these diseases.
Surgical intervention, which lowers bile acid levels, has been shown to relieve symptoms and slow disease progression in patients with ALGS and PFIC - this is currently the only treatment option for these patients. Patients with cholestatic liver diseases may ultimately require liver transplants.
By reducing serum bile acids, LUM001 may offer a novel therapeutic approach for alleviating the pruritus and progressive liver damage associated with cholestatic liver diseases.
The prevalence of each of the four diseases is as follows:
| Indication US | Indication US prevalence 3 per 100,000 |
EU prevalence |
| ALGS | 9476 individuals <1 per 100,000 |
3 per 100,000 |
| PFIC | 3159 individuals 40 per 100,000 |
1 per 100,000 |
| PBC | 126,979 individuals 14 per 100,000 |
30 per 100,000 |
| PSC | 44,222 individuals 3 per 100,000 |
3 per 100,000 |
(Source: Lumena Pharmaceuticals Applications for Orphan Designation):
LUM001 has received orphan drug designation for all four potential indications in both the United States and the European Union.
About LUM002
LUM002 is a novel, once daily, orally-administered, highly potent and selective inhibitor of ASBT, in development for the treatment of nonalcoholic steatohepatitis (NASH), a common and often "silent" liver disease characterized by fat deposits in the liver and inflammation which can progress to significant fibrosis.
While the underlying cause of liver injury in NASH is not fully known, it is strongly associated with obesity, Type 2 diabetes, high cholesterol and triglycerides, and other metabolic disorders. Approximately 6 million individuals in the US are estimated to have progressed to NASH and some 600,000 to NASH-related cirrhosis (Source: World Gastroenterology Organisation Global Guidelines June 2012).
By blocking bile acid reabsorption, LUM002 is thought to modulate colonic bile acid concentrations and receptor signaling on cells in the lower portion of the GI tract. This signaling is believed to result in the secretion of peptides that regulate insulin release from the pancreas, glucose metabolism and the synthesis of cholesterol and fatty acids.
Therapeutic strategies aimed at modulating insulin resistance and normalizing lipoprotein metabolism have significant potential to benefit patients with NASH.
Phase 1 safety trials in healthy volunteers and a Phase 1b trial in patients with metabolic disease have been completed. The next step is to initiate a Phase 2 clinical trial in patients with NASH - anticipated to begin in the second half of 2014.
NOTES TO EDITORS
About Lumena Pharmaceuticals
Lumena Pharmaceuticals is a privately held, San Diego-based, biopharmaceutical company founded in 2011 by Pappas Ventures. Other investors include Alta Partners, RiverVest Venture Partners, New Enterprise Associates, Adage Capital Management and RA Capital Management.
Citi acted as the exclusive financial advisor to Lumena.
About Shire plc
Shire enables people with life-altering conditions to lead better lives.
Our strategy is to focus on developing and marketing innovative specialty medicines to meet significant unmet patient needs.
We provide treatments in Neuroscience, Rare Diseases, Gastrointestinal and Internal Medicine and we are developing treatments for symptomatic conditions treated by specialist physicians in other targeted therapeutic areas.
FORWARD - LOOKING STATEMENTS - "SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995
Statements included in this announcement that are not historical facts are forward-looking statements. Forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire's results could be materially adversely affected. The risks and uncertainties include, but are not limited to, that:
- Shire's products may not be a commercial success;
- revenues from ADDERALL XR are subject to generic erosion and revenues from INTUNIV will become subject to generic competition starting in December 2014;
- the failure to obtain and maintain reimbursement, or an adequate level of reimbursement, by third-party payors in a timely manner for Shire's products may impact future revenues, financial condition and results of operations;
- Shire conducts its own manufacturing operations for certain of its Rare Diseases products and is reliant on third party contractors to manufacture other products and to provide goods and services. Some of Shire's products or ingredients are only available from a single approved source for manufacture. Any disruption to the supply chain for any of Shire's products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time.
- the development, approval and manufacturing of Shire's products is subject to extensive oversight by various regulatory agencies and regulatory approvals or interventions associated with changes to manufacturing sites, ingredients or manufacturing processes could lead to significant delays, increase in operating costs, lost product sales, an interruption of research activities or the delay of new product launches;
- the actions of certain customers could affect Shire's ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely impact Shire's revenues, financial conditions or results of operations;
- investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire's activities in the highly regulated markets in which it operates may result in the distraction of senior management, significant legal costs and the payment of substantial compensation or fines;
- adverse outcomes in legal matters and other disputes, including Shire's ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on Shire's revenues, financial condition or results of operations;
- Shire faces intense competition for highly qualified personnel from other companies, academic institutions, government entities and other organizations. Shire is undergoing a corporate reorganization and the consequent uncertainty could adversely impact Shire's ability to attract and/or retain the highly skilled personnel needed for Shire to meet its strategic objectives;
- failure to achieve Shire's strategic objectives with respect to the acquisition of ViroPharma Incorporated may adversely affect Shire's financial condition and results of operations; and other risks and uncertainties detailed from time to time in Shire's filings with the U.S. Securities and Exchange Commission, including its most recent Annual Report on Form 10-K.
For further information please contact:
Shire
Investor Relations:
Jeff Poulton
jpoulton@shire.com
+1-781-482 0945
Sarah Elton-Farr
seltonfarr@shire.com
+44-1256-894157
Media:
Jessica Mann
jmann@shire.com
+44-1256-894-280
Gwen Fisher
gfisher@shire.com
+1-484-595-9836
Lumena Media contact:
Heidi Chokeir, Ph.D.
Canale Communications
Heidi@canalecomm.com
+1-619-203-5391
SOURCE Shire plc and Lumena Pharmaceuticals