News

-Clinical-stage company targeting fundamental drivers of inflammation, autoimmunity and fibrosis, founded by Versant Ventures and Novartis Venture Fund-

-Series B financing led by Forbion and Sanofi Ventures-

-Antibody programs developed in collaboration with Versant’s Ridgeline Discovery Engine in Basel-

Basel, Switzerland and San Francisco, Calif., April 24, 2025 - Granite Bio AG, a clinical-stage immunology company, has emerged from stealth with $100 million in funding. This includes a $30 million Series A led by founding investors Versant Ventures and Novartis Venture Fund, and a $70 million Series B led by Forbion and Sanofi Ventures.

Granite’s pipeline features two first-in-class antibodies targeting multiple autoimmune diseases that address large market opportunities.

GRT-001 depletes pro-inflammatory monocytes, key drivers of autoimmunity and inflammation. In non-human primate studies, GRT-001 efficiently and dose-dependently depleted pro-inflammatory monocytes. Treatment was well tolerated and spared tissue-resident macrophages, which are important for tissue homeostasis. GRT-001 is currently in Phase 1a testing in healthy volunteers and is expected to enter a Phase 1b trial in patients with inflammatory bowel disease later this year.

GRT-002 blocks interleukin-3, a key player in autoimmune inflammation and type II inflammation, offering a new approach to treating itch and allergy. The molecule is in preclinical development and is expected to enter clinical trials in 2026.

Both of Granite’s lead molecules were developed in collaboration with Versant’s Ridgeline Discovery Engine in Basel, Switzerland, and originated from the laboratories of Professor and Scientific Co-Founder Matthias Mack at University of Regensburg.

"Granite is pioneering a new approach to tackling inflammation, autoimmunity, and fibrosis by addressing fundamental disease drivers at their source,” said Patrick Loustau, president and CEO. “With the support of an exceptional investor syndicate and a world-class team, we are advancing a pipeline of first-in-class therapies with the potential to transform patient outcomes. I look foward to working with the management team to deliver groundbreaking treatments that address the urgent unmet needs in immunology and beyond."

“Despite multiple immunology-based therapeutic approaches currently commercialized, patients with inflammatory disorders continue to experience a lack of symptom control and relapse,” said Nigel Sheail, partner at Versant and Granite board member. “Granite has the potential to enable a real step change for patients with its innovative programs that target key fundamental disease pathways.”

“I am proud to support Granite as it advances its lead assets using a truly differentiated approach to tackling inflammation at its root,” said Rogier Rooswinkel, Ph.D., General Partner at Forbion and Granite board member. “With the strength of this investor syndicate and an exceptional leadership team, Granite is well-positioned to drive its pipeline forward and deliver meaningful new therapies for patients.”

Leadership team

Granite is led by an experienced team including:

• Patrick Loustau, president and CEO, brings broad and successful experience in building and developing organizations in biotech and pharmaceutical companies. Prior to joining Granite Bio, he was the CBO of Amolyt Pharma until its acquisition by Alexion (AstraZeneca Rare Disease) for up to $1.05B in July 2024. Before Amolyt, he was CEO of Zumbro Discovery Inc. and president of Zafgen Inc. Earlier in his career, he led global organizations at Bristol Myers-Squibb and Novo Nordisk.
• Dominik Hartl, M.D., CMO has extensive background in immunology and fibrosis drug development. He previously served as CMO for Quell Therapeutics, a company specialized in immune-mediated diseases. Before that, he held leadership positions in Immunology Drug Development/Translational Medicine at Novartis (NIBR/Immunology), Roche (pRED/I2O) and Galapagos Pharma. Dominik is a board-certified M.D. and holds an adjunct professorship in Pediatric Immunology at University of Tübingen.
• Gijs van den Brink, M.D., Ph.D., CSO, possesses deep academic and industry experience in immunology and IBD. Currently also an Operating Partner at Forbion, he previously served as SVP and Global Head of Immunology, CVM, ID, and Ophthalmology Discovery and Early Development at Roche. Before that, Dr. van den Brink was head of Immunology discovery and early and late-stage clinical development at GlaxoSmithKline plc. Gijs previously was a gastroenterologist and professor of experimental gastroenterology and co-authored over 150 peer-reviewed scientific publications.
• Eliot Forster, chairperson of the board, has more than 30 years of extensive biotech experience from both executive and non-executive roles. He was CEO of F-star Therapeutics until its acquisition by inovX Pharma LTD in March 2023, and formerly CEO of Immunocore. Eliot is currently CEO of Levicept LTD, non-executive chair of Tessellate BIO and a director at Immatics.

About Granite Bio

Granite Bio is a biotechnology company developing first-in-class antibodies that target the root causes of a variety of inflammatory, autoimmune and fibrotic conditions. Granite is backed by leading healthcare venture firms including Versant Ventures, Novartis Venture Fund, Forbion and Sanofi Ventures. For more information, please visit www.GRANITEBIO.com.

About Versant Ventures

Versant Ventures is a leading healthcare venture capital firm committed to helping exceptional entrepreneurs build the next generation of great companies. The firm’s emphasis is on biotechnology companies that are discovering and developing novel therapeutics. With $5.3 billion under management and offices in the U.S., Canada and Europe, Versant has built a team with deep investment, operating and R&D expertise that enables a hands-on approach to company building. Since the firm’s founding in 1999, more than 95 Versant companies have achieved successful acquisitions or IPOs. For more information, please visit www.versantventures.com.

About NVF

Novartis Venture Fund is a financially driven corporate life science venture fund whose purpose is to foster innovation, drive significant patient benefit and generate superior returns by creating and investing in innovative life science companies at various stages of their development. For more information, go to www.nvfund.com.

About Forbion

Forbion is a leading global venture capital firm with deep expertise in Europe and offices in Naarden, The Netherlands, Munich, Germany and Boston, USA. Forbion invests in innovative biotech companies, managing approximately €5 billion across multiple fund strategies that cover all stages of (bio-) pharmaceutical drug development. In addition, Forbion leverages its biotech expertise beyond human health to address ‘planetary health’ challenges through its BioEconomy fund strategy, which invests in companies developing sustainable solutions in food, agriculture, materials, and environmental technologies. Forbion’s team consists of over 30 investment professionals that have built an impressive performance track record since the late nineties with 128 investments across 11 funds. Forbion’s record of sourcing, building and guiding life sciences companies has resulted in many approved breakthrough therapies and valuable exits. Forbion typically selects impactful investments that will positively affect the health and well-being of people and the planet, as well as meet its financial return objectives. The firm is a signatory to the United Nations Principles for Responsible Investment. Forbion operates a joint venture with BGV, the manager of seed and early-stage funds, especially focused on Benelux and Germany.

About Sanofi Ventures

Sanofi Ventures is the corporate venture capital arm of Sanofi, focused on investing in promising early-stage healthcare companies. The firm supports pioneering innovations in biotechnology, digital health, and life sciences aligning with Sanofi’s mission to bring life-changing treatments to patients worldwide. For more information, please visit www.sanofiventures.com.

Granite Bio contact:

Steve Edelson

sedelson@versantventures.com

415-801-8088

• GLM101 is the first disease-modifying therapeutic in development to treat PMM2-CDG, the most common congenital disorder of glycosylation  
• Data from the ongoing Phase 2 open-label study of GLM101 provide clinical proof of concept, showing improvement in ataxia, a key burden of disease in PMM2-CDG 
• Funding will support advancement of GLM101 into a randomized, placebo-controlled Phase 2b safety and efficacy study 

SAN CARLOS, Calif., April 16, 2025 – Glycomine, Inc., a biotechnology company focused on developing transformative new therapies for orphan diseases, today announced a $115 million Series C financing to advance its lead candidate, GLM101, into a Phase 2b clinical trial. The financing was led by CTI Life Sciences Fund, funds managed by abrdn Inc., and Advent Life Sciences, alongside continued investment from existing investors, Novo Holdings, Sanofi Ventures, Abingworth, RiverVest Venture Partners, Sanderling Ventures, Chiesi Ventures, Remiges Ventures, and Asahi Kasei Ventures.  

“We are excited to partner with our new investors who have strong track records in rare diseases and for the continued support from our existing investors,” said Steve Axon, Glycomine’s CEO. “This financing will enable us to advance GLM101 into a randomized, placebo-controlled trial later this year—an important step toward bringing the first disease-modifying therapeutic to patients with PMM2-CDG.” 

GLM101, a first-in-class mannose-1-phosphate replacement therapy, is in development for phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG), a rare and life-threatening genetic disorder with no approved treatments. Glycomine has enrolled more than 20 patients across Europe and the U.S. in its ongoing Phase 2 study and recently initiated dosing in pediatric patients. 

Data from Glycomine’s ongoing Phase 2 open-label study have demonstrated promising improvements in ataxia, a hallmark debilitating manifestation of PMM2-CDG. Among nine adult and adolescent patients, treatment with GLM101 led to an average 11.9-point improvement on the ICARS (International Cooperative Ataxia Rating Scale) over 24 weeks. 

“We are impressed by the therapeutic approach and strong progress of the Glycomine team,” said Youssef Bennani, Ph.D., Managing Partner with CTI Ventures. “We are excited to be part of such a strong investor syndicate and look forward to the potential of making a positive impact in the lives of patients with PMM2-CDG.” 

Dominic Schmidt, Ph.D., General Partner with Advent Life Sciences added, “We are highly encouraged by the clinical signal observed in the ongoing Phase 2 study. Most notably, the data show strong potential for clinically meaningful improvement in ataxia, a key driver of disease burden for PMM2-CDG patients.” 

In connection with the financing, Drs. Bennani and Schmidt have been appointed to Glycomine’s Board of Directors. 

About PMM2-CDG 

Phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG-1a, is the most prevalent congenital disease of glycosylation. PMM2-CDG is caused by a genetic mutation in phosphomannomutase 2 (PMM2), which results in the protein having reduced activity. PMM2 is an enzyme that converts mannose-6-phosphate to mannose-1-phosphate, which is required to insert the mannose sugar building block into developing glycans that are crucial for proper protein structure and function. The deficiency of mannose-1-phosphate disrupts the process of N-glycosylation and causes a wide array of clinical symptoms and, in many cases, can be life-threatening.  

About Glycomine, Inc. 

Glycomine is a clinical-stage biotechnology company that is advancing treatments for serious rare diseases for which no other therapeutic options exist. The Company’s lead investigational drug candidate GLM101 is a mannose-1-phosphate replacement therapy in development to treat PMM2-CDG. GLM101 is designed to deliver mannose-1-phosphate into cells and thereby bypass disease-causing PMM2 mutations to restore pathway function. GLM101 has received Orphan Drug Designation in the U.S. and E.U. and Rare Pediatric Disease Designation and Fast Track Designation in the U.S. The company is based in San Carlos, California, and supported by leading international life sciences investors. For more info visit www.glycomine.com.  

Corporate Contact:
Peter McWilliams, Ph.D.
info@glycomine.com  

Media Contact:
Jessica Yingling, Ph.D.
Little Dog Communications Inc.
jessica@litldog.com  

Series C round led by Deep Track Capital with participation fromadditional new and existing investors

Financing will fund early-to-mid-stage clinical studies for ATTO-1310 inchronic pruritus and ATTO-3712 in atopic dermatitis, and multiple earlier-stage multi-specific programs

SAN CARLOS, Calif., April 15, 2025 -- AttoviaTherapeutics, a privately-held, clinical-stage biopharma companydeveloping treatments for immune-mediated diseases with high unmetpatient need, today announced the close of a $90 million Series Cfinancing. The funding round was led by Deep Track Capital withparticipation from new investors including Vida Ventures, Sanofi Ventures,and Mirae Asset Capital Life Science, and ongoing support from existinginvestors including Frazier Life Sciences, venBio, Goldman SachsAlternatives, Nextech Ventures, Cormorant Asset Management, EcoR1Capital, Marshall Wace, and Illumina Ventures. In conjunction with thefinancing, Rebecca Luse, Managing Director at Deep Track Capital, will jointhe Company's Board of Directors.

Proceeds from the financing, along with the Company’s existing cash andinvestments, will be used to advance Attovia’s lead assets, ATTO-1310 andATTO-3712, through clinical proof-of-concept, with the aim of achievingbest-in-disease efficacy for the treatment of chronic pruritus and atopicdermatitis. The funds will also enable focused expansion of Attovia’s  pipeline of multi-specific therapeutic candidates utilizing the company’sproprietary ATTOBODY technology to develop breakthrough treatmentoptions for patients suffering from immune-mediated disorders, includinginflammatory bowel disease (IBD) and others.

“Completing what was an oversubscribed round of financing during thistime speaks to the standout potential of our platform, programs, andexcellent team,” said Tao Fu, Attovia’s founder and CEO. “We deeplyappreciate the support of our exceptional investor base and the sharedcommitment to transforming the lives of patients living with immune-mediated diseases. We now have a multi-year runway to advance ourprograms expeditiously, expand our pipeline judiciously, and pursuepotential partnerships, all of which should give us greater optionality forthe future.”

The financing allows Attovia to continue to advance its first ATTOBODY-based product candidate, ATTO-1310, a first-in-class, half-life extended anti-IL31 biologic, currently in Phase 1 studies, to provide a highly efficacious,fast acting, and convenient treatment option for chronic pruritus ofunknown origin (CPUO)—a disease affecting millions of patients in the U.S.and other developed countries with currently no approved treatments—aswell as additional pruritic conditions with urgent unmet need.

Attovia is also advancing ATTO-3712, a first-in-class anti-IL13 x IL31, half-lifeextended bispecific designed to provide breakthrough efficacy andconvenient dosing in atopic dermatitis and potentially chronicspontaneous urticaria, prurigo nodularis, and other inflammatory skinconditions. The company plans to start Phase 1 clinical studies with ATTO-3712 in the second half of 2025.

Attovia’s earlier-stage pipeline includes ATTO-004, a multi-specific drugcandidate targeting IBD, and two other multi-specific discovery-stageprograms.

“Attovia has created a transformative pipeline leveraging its revolutionaryATTOBODY platform,” said Rebecca Luse, Managing Director at Deep TrackCapital. “We are highly impressed with the team's speed and quality ofdisciplined execution, and we are looking forward to supporting theirgrowth and success as they work to realize the full potential of theirplatform and pipeline programs.”

About Attovia

Attovia is creating a pipeline of biotherapeutics for the treatment ofimmune-mediated diseases. We leverage ATTOBODY™, our proprietarybiologics platform, to generate potentially first-in-class and best-in-classmulti-specifics.

ATTOBODIES utilize spatial positioning technology to achieve biparatopictarget engagement. The biparatopic binding mode of ATTOBODIES resultsin ultra-high affinity, which translates to best-in-class potency in biologicactivity. ATTOBODIES offer unconstrained engineering, making them anideal modality for the development of multi-specific biologics, with tunablehalf-life translating to the potential for quarterly or less frequent dosing inpatients. The high-throughput, evolution-based ATTOBODY discoveryplatform delivers a high degree of diversity at industry-leading speed,accelerating and de-risking therapeutics development, offering thepotential to become the next-generation modality of choice. Learn more atwww.attovia.com and follow us on LinkedIn.

Press Contact

Attovia Therapeutics:

info@attovia.com

Results demonstrated statistically significant effects on biomarkers that predict ALS disease progression and severity

Evidence of brain penetration, target engagement, and potential anti-seizure effects demonstrated through biomarkers for TMS-EMG and EEG

Safety and tolerability profile consistent with previously reported results for QRL-101

QurAlis intends to advance QRL-101 into proof-of-concept studies as a potentially best-in-class treatment for both ALS and epilepsy

CAMBRIDGE, Mass., March 12, 2025 – QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of neurodegenerative and neurological diseases, today announced positive topline data from QurAlis' Phase 1 proof-of-mechanism (PoM) clinical trial of QRL-101 in healthy volunteers evaluating biomarkers related to amyotrophic lateral sclerosis (ALS) and epilepsy.

Topline results demonstrated a dose-dependent, statistically significant decrease in motor nerve excitability threshold tracking (mNETT) strength-duration time constant (SDTC), the co-primary endpoint of the trial related to ALS. SDTC is a well-established electrophysiological biomarker of peripheral motor excitability related to ALS, where elevation of SDTC has been clinically shown to predict faster disease progression and mortality. The decrease in SDTC observed with QRL-101 in this study was approximately 50% greater than previously reported for the Kv7 potassium channel opener ezogabine in a single-dose study in people with ALS. Additional observations for multiple secondary and exploratory endpoints related to motor nerve excitability were also significantly impacted.

The clinical trial demonstrated a statistically significant impact on multiple secondary and exploratory endpoints related to epilepsy. These included transcranial magnetic stimulation electromyography (TMSEMG) intracortical facilitation which indicates effective inhibition of cortical excitability, as well as significant increases in electroencephalography (EEG) spectral power in beta and gamma spectral bands further demonstrating cortical target engagement and brain penetration. There was no statistically significant impact on the slower delta and theta EEG bands, which suggests a low potential for GABA-A receptor activation and sedation. The study did not reach statistical significance on the co-primary endpoint of TMS-EMG motor evoked potential (MEP) amplitude, another measure of corticospinal excitability.

Results also demonstrated that the safety and tolerability profile of QRL-101 was consistent with previously reported study results evaluating QRL-101 to date. There were no serious adverse events or discontinuations due to adverse events reported observed in the study.

“We are excited by these topline data from our biomarker study in healthy participants which suggest that QRL-101 has the potential to provide a therapeutic effect for both ALS and epilepsy,” said Kasper Roet, Ph.D., CEO and co-founder of QurAlis. “Loss of Kv7.2/7.3 function from the mis-splicing of the KCNQ2 gene leading to hyperexcitability-induced neurodegeneration was one of the first genetic breakthroughs from human ALS motor neuron stem cell models made by our founders at Harvard. QurAlis was started  to develop a best-in-class Kv7 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS, which occurs in about half of all people living with ALS. Elevated SDTC has been clinically linked to faster disease progression and mortality, underscoring the need for an effective treatment for this population.”

Dr. Roet continued, “The decrease in SDTC observed with QRL-101 in this study was approximately 50% greater than previously reported for the Kv7 opener ezogabine in a single-dose study in people with ALS. QRL-101 has the potential to be a promising therapeutic, especially considering the encouraging results from a previous study with ezogabine in ALS patients showing positive effects on both SDTC and the disease progression biomarker compound muscle action potential (CMAP). Further, based on the strong clinical validation of Kv7.2/7.3 ion channel openers in seizure reduction, the TMS-EMG intracortical facilitation together with the EEG data from this study suggest QRL-101 may have the potential to reduce both frequency and severity of seizures in people living with epilepsy without causing sedation. We are encouraged by these results and look forward to advancing the clinical program for QRL-101 in both ALS and epilepsy so that we can fulfill our mission of bringing much-needed precision medicine options to patients.”

Kv7.2/7.3 is a voltage-gated potassium channel whose role is crucial for the regulation of both neuronal excitability and membrane potential. QRL-101 is a potentially best-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS, which occurs in both sporadic and genetic forms of ALS, with the majority caused by the mis-splicing of the KCNQ2 gene in the pre-mRNA. Kv7 is also a clinically validated target to regulate the hyperexcitable state in epilepsy. In vivo and in vitro studies have demonstrated that QRL-101 is more potent in threshold track studies and exhibits the potential for fewer clinical adverse events that ezogabine, a less selective, first-generation, Kv7.2/7.3 channel opener.

The Phase 1 PoM study (QRL-101-05, NCT06681441) was a randomized, double-blind, placebo-controlled, three-way crossover trial. The study was conducted at the Centre for Human Drug Research, an earlyphase Contract Research Organization based in Leiden, The Netherlands. The study evaluated two dose levels of QRL-101 versus placebo in healthy volunteers, assessing:

• Co-primary endpoints:
  • Motor nerve excitability (mNETT SDTC; ALS biomarker)
  • Cortical excitability (TMS-EMG MEP amplitude; epilepsy biomarker)
• Secondary endpoints:
  • Additional TMS-EMG cortical excitability measures
  • Resting-state pharmaco-EEG
  • Safety, tolerability, and pharmacokinetics (PK)

Additional QRL-101 clinical trials include:

• QRL-101-01 (NCT05667779) was a first-in-human, single-ascending dose clinical trial in 88 healthy participants. In this clinical trial, QRL-101 was shown to be well tolerated. The study completed in late 2023 and results from QRL-101-01 supported a tolerable dose range for subsequent studies.
• QRL-101-03 (NCT06532396) is a randomized, double-blind, placebo-controlled, multiple-ascending dose clinical trial evaluating the safety, tolerability, and PK of QRL-101 in up to 60 healthy participants.
• QRL-101-04 (NCT06714396) is a Phase 1 PoM single-dose, placebo-controlled clinical trial designed to evaluate the safety and tolerability of QRL-101 in people living with ALS. QRL-101-04 is expected to enroll approximately 12 participants with ALS and will evaluate the impact of QRL-101 on mNETT.
• QRL-101-06 is a Phase 1 randomized, open-label, single dose, cross-over study to evaluate the PK of three formulations of QRL-101 in a fasted condition or in the presence of a high fat meal in healthy participants.

More information about the QRL-101 clinical trials can be found at www.clinicaltrials.gov.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure, boldly seeking to translate scientific breakthroughs into powerful precision medicines. We work collaboratively with a relentless pursuit of knowledge, precise attention to craft, and compassion to discover and develop medicines that have the potential to transform the lives of people living with neurodegenerative and neurological diseases. QurAlis is the leader in development of precision therapies for amyotrophic lateral sclerosis (ALS). In addition to ALS, QurAlis is advancing a robust precision medicine pipeline to bring effective disease-modifying therapeutics to patients suffering from severe diseases defined by genetics and clinical biomarkers. For more information, please visit www.quralis.com or follow us on X @QurAlisCo or LinkedIn.

Contact:

Kathy Vincent

kathy.vincent@quralis.com

310-403-8951

• Financing led by funds managed by Blue Owl Capital; Senior Managing Director Kevin Raidy joins Latigo’s board
• Proceeds support advancement of company’s Nav1.8 inhibitor clinical programs and development of broader pipeline

THOUSAND OAKS, Calif. – March 17, 2025 – Latigo Biotherapeutics (“Latigo”), a clinical-stage biotechnology company developing best-in-class non-opioid pain treatments that target pain at its source, today announced it has closed $150 million in a Series B financing. Proceeds from the financing will support the advancement of the company’s highly selective Nav1.8 inhibitors currently in clinical development for the non-opioid treatment of pain, as well as the development of Latigo’s broader pipeline.

The financing was led by funds managed by Blue Owl Capital, with participation from Deep Track Capital, Access Biotechnology, Qatar Investment Authority, Cormorant Asset Management, Sanofi Ventures, Rock Springs Capital, UPMC Enterprises, and Kern Capital. Existing investors Westlake Village BioPartners, Foresite Capital, 5AM Ventures, and Alexandria Venture Investments also participated in the financing round, reaffirming their commitment to Latigo’s mission to develop safer, more effective pain treatments.

“The need for non-opioid pain treatments has never been more urgent, and this financing allows us to accelerate the development of our robust portfolio of pain medicines that have the potential to transform the treatment landscape,” said Nima Farzan, chief executive officer of Latigo Biotherapeutics. “We appreciate the support of our new and existing investors as we work to bring best-in-class, non-addictive pain treatments to patients.”

As part of the financing, Kevin Raidy, senior managing director at Blue Owl Capital, has joined Latigo’s board of directors. “The field of pain management is long overdue for innovation beyond opioids, and we believe Latigo is well-positioned to advance novel, non-addictive treatments that could make a real difference for patients,” Raidy said. “We are pleased to welcome Kevin to the board. His experience in life sciences investing and strategic growth will be invaluable as Latigo continues to scale,” said Timothy P. Walbert, chair of Latigo’s board. “With this financing and the continued support of world-class investors, we will be able to rapidly advance our pipeline of non-opioid treatments for chronic and acute pain.”

About Latigo Biotherapeutics’ Clinical Programs

Latigo recently reported positive Phase 1 results for LTG-001, its lead potential best-in-class non-opioid pain medicine candidate. LTG-001 is an oral, selective Nav1.8 inhibitor in development to treat acute pain at its source. In the Phase 1 first-in-human clinical trial, data showed that LTG-001 was well tolerated with rapid absorption.

LTG-305, oral, selective Nav1.8 inhibitor, currently in Phase 1 clinical trials, is a potential best-in- class non-opioid therapeutic candidate for the treatment of chronic pain. The Phase 1 trial is designed to evaluate the safety, tolerability, and pharmacokinetics of LTG-305 in healthy volunteers through single-ascending dose and multiple-ascending dose cohorts.

About Latigo Biotherapeutics

Latigo Biotherapeutics is a private clinical-stage biotechnology company developing innovative non-opioid pain medicines with potential best-in-class profiles that directly target the source of pain. Latigo’s goal is to provide effective, rapid-acting pain relief without the risk of addiction. For more information, please visit www.latigobio.com or follow us on LinkedIn.

Media Contact:

Kathy Vincent

Greig Communications Inc.

kathy@greigcommunications.com

• Amezosvatein has shown excellent immunogenicity and improved tolerability in Phase 2 study
• Medicxi leads round, joined by new investors OrbiMed, HBM Healthcare Investments, and Sanofi Ventures
• Former Chair of GSK’s vaccine business and Chief Scientific Advisor to Operation Warp Speed, Moncef Slaoui, to join Curevo as Board Chair

Seattle, WA – March 17, 2025 – Curevo Vaccine (Curevo), a privately-held clinical-stage biotechnology company dedicated to developing varicella zoster virus (VZV) vaccines with improved tolerability, today announces the closing of a $110 million Series B round to advance development of amezosvatein, its vaccine for shingles.

Leading the round is new investor Medicxi, a European biotech-focused investment firm with significant experience investing in vaccine companies such as Vaxcyte and ViceBio. Joining Medicxi is an international group of investment funds including OrbiMed, HBM Healthcare Investments, and Sanofi Ventures plus existing investors RA Capital Management, Janus Henderson Investors, Adjuvant Capital, and founding investor GC Biopharma.

“This Series B round will fund the extension of our successful Phase 2 program into an additional 640 participants, including the key population of adults over age 70, to finalize dose selection ahead of the Phase 3 program,” said Curevo’s CEO, George Simeon, MBA/MPH.

“Designed based upon feedback from regulators and other stakeholders, this short extension trial will begin mid-2025 and serve to set the company for clinical, strategic, and regulatory success.”

Concurrent with the round, Moncef Slaoui, PhD, will join Curevo as Board Chair. Dr. Slaoui was most recently the Chief Scientific Advisor to Operation Warp Speed during the COVID-19 pandemic, helping deliver vaccines against the SARS-CoV-2 virus. He spent nearly 30 years at GlaxoSmithKline, during which he helped shape its vaccine business by contributing to the creation of numerous new vaccines including Shingrix^® (shingles), Cervarix^® (HPV-induced cervical cancer), Mosquirix^® (malaria), Rotarix^® (rotavirus gastroenteritis), and Synflorix^® (pneumococcal disease).

“I have been collaborating with the Curevo team for several weeks now,” stated Dr. Slaoui. “I’m very excited to work with them to help perfect shingles vaccination, adding good tolerability to the exceptional efficacy achieved by the current vaccine. The data so far show Curevo’s adjuvant technology has the attributes to succeed in this endeavor.”

Also joining Curevo’s Board of Directors will be Giovanni Mariggi, PhD, co-founder and Partner at Medicxi, an experienced infectious disease investor and board member. “Patients, doctors, and payors are very clear a new shingles vaccine like amezosvatein would be welcome in the global marketplace. Amezosvatein’s activity and improved tolerability profile could allow it to be a significant product in the shingles vaccines market. We are excited to support Curevo’s plan with a streamlined path to approval,” stated Dr. Mariggi.

Tal Zaks, MD/PhD, will be joining Curevo’s Board of Directors on behalf of OrbiMed. Dr. Zaks is the former Chief Medical Officer of Moderna, where he led development of their mRNA vaccine against the SARS-CoV-2 virus. He previously held senior leadership positions in drug

development at GSK and Sanofi. “OrbiMed has a long history of investing in biotech companies like Curevo seeking to improve healthcare outcomes while creating long-term shareholder value,” said Dr. Zaks. “The fact amezosvatein contains an optimized version of the TLR4 agonist with a known mechanism of action reduces biological risk while providing the competitive advantage of potentially improved tolerability, which should position it as the first choice for people who want to be protected from shingles.”

About amezosvatein

‘Amezosvatein’ is the assigned non-proprietary name for CRV-101, a non-mRNA adjuvanted subunit vaccine under investigation by Curevo. Like Shingrix, amezosvatein uses a subunit protein antigen called glycoprotein ‘E’ (gE). Targeting the gE antigen is proven to elicit a long-term, protective immune response to prevent shingles. Amezosvatein’s adjuvant contains an optimized version of the TLR4 agonist proven by Shingrix to be biologically active in shingles vaccination. Amezosvatein was engineered to maintain exceptional efficacy and have a best-in-class tolerability profile. The SLA-SE adjuvant formulation was developed at Seattle-based Access to Advanced Health Institute (AAHI) and amezosvatein was licensed from the Mogam Institute for Biomedical Research, a research institute funded by South Korea’s GC Biopharma.

About Curevo

Curevo is a privately held, clinical-stage biotechnology company based near Seattle dedicated to reducing the burden of infectious disease by developing vaccines with improved tolerability and accessibility. Curevo’s lead product is amezosvatein, a non-mRNA adjuvanted sub-unit vaccine to prevent shingles, a serious medical condition involving a painful, blistering skin rash where 10-18% of people also develop serious, long-lasting nerve pain. The current $4+ billion shingles vaccine market is characterized by accessibility issues and vaccine hesitancy/dose avoidance related to vaccine tolerability. For more information visit https://curevovaccine.com/.

About Medicxi

Medicxi is a healthcare-focused investment firm with the mission to create and invest in companies across the full drug development continuum. Leveraging deep expertise in drug development and company creation spanning over two decades, Medicxi invests in early and late-stage therapeutics with a product vision that can fulfill a clear unmet medical need. For more information, please visit: https://www.medicxi.com.

About OrbiMed

OrbiMed is a leading global healthcare investment firm with approximately $17 billion in assets under management. OrbiMed invests globally across the healthcare industry, from start-ups to

large multinational corporations, via private equity funds, public equity funds, and royalty/credit funds. OrbiMed seeks to be a capital provider of choice, providing tailored financing solutions and extensive global team resources to help build world-class healthcare companies. OrbiMed’s team of over 100 professionals is based in New York City, London, San Francisco, Shanghai, Hong Kong, Mumbai, Herzliya, and other key global markets. For more information, please visit www.orbimed.com.

About HBM Healthcare Investments

Switzerland-based HBM Healthcare Investments focuses on the healthcare sector, holding and managing an international portfolio of promising companies in the human medicine, biotechnology, medical technology, diagnostics, and related areas. Many of these companies have their lead products already available on the market or are at an advanced stage of development. The portfolio companies are closely tracked and actively guided in their strategic direction. This is what makes HBM Healthcare Investments an interesting alternative to investments in big pharma and biotechnology companies. HBM Healthcare Investments has an international shareholder base and is listed on SIX Swiss Exchange (SIX:HBMN).

About Sanofi Ventures

Sanofi Ventures is the corporate venture capital arm of Sanofi, focused on investing in promising early-stage healthcare companies. The firm supports pioneering innovations in biotechnology, digital health, and life sciences aligning with Sanofi’s mission to bring life-changing treatments to patients worldwide. For more information, please visit www.sanofiventures.com.

About RA Capital

Founded in 2004, RA Capital Management is a multi-stage investment manager dedicated to evidence-based investing in public and private healthcare, life sciences, and planetary health companies. RA Capital creates and funds innovative companies, from private seed rounds to public follow-on financings, allowing management teams to drive value creation from inception through commercialization and beyond. RA Capital's knowledge engine is guided by its TechAtlas internal research division, and Raven, RA Capital's healthcare incubator, offers entrepreneurs and innovators a collaborative and comprehensive platform to explore the novel and the re-imagined. RA Capital has more than 150 employees and over $10 billion in assets under management.

About Janus Henderson Investors

Janus Henderson Investors exists to help clients achieve their long-term financial goals. Its active management offers clients the opportunity to outperform passive portfolios over the course of market cycles. With more than 360 investment professionals, Janus Henderson Investors provides access to some of the industry’s most talented and innovative thinkers, spanning equities, fixed income, multi-asset, and alternatives, globally. Its investment teams blend insight, originality, and precision with rigorous analysis, structured processes, and robust risk management. Janus Henderson Investors builds client partnerships on openness and trust, channeling expertise from across the business and communicating the views of its experts in a timely and relevant way. https://www.janushenderson.com/

About Adjuvant Capital

Headquartered in New York, with offices in Zürich, Adjuvant is a global life science investment fund built to accelerate the development of new technologies for the world's most pressing public health challenges. Backed by prominent healthcare and emerging market investors, Adjuvant draws upon its network of scientists, public health experts, biopharmaceutical industry veterans, and development finance professionals to identify new investment opportunities. Adjuvant invests in companies developing promising new vaccines, therapeutics, diagnostics, and medical devices targeting high-burden infectious diseases, maternal and child health, antimicrobial resistance, and malnutrition, with a commitment to make these interventions accessible globally. For more information, visit http://www.adjuvantcapital.com

About GC Biopharma

Founding investor GC Biopharma (formerly known as Green Cross Corporation) is a biopharmaceutical company headquartered in Yongin, South Korea. The company has over half a century of experience in the development and manufacturing of plasma derivatives and vaccines and expanded its global presence with the successful USA market entry of ALYGLO™ (intravenous immunoglobulin G) in 2024. In line with its mission to meet the demands of future healthcare, GC Biopharma continues to drive innovation by leveraging its core R&D capabilities in engineering of proteins, mRNAs, and lipid nanoparticle (LNP) drug delivery platforms to develop therapeutics for the field of rare disease as well as I&I (Immunology & Inflammation). To learn more about the company, visit https://www.gcbiopharma.com/eng/

Shingrix^®, Cervarix^®, Mosquirix^®, Rotarix^®, and Synflorix^® are registered trademarks of GlaxoSmithKline, PLC.

Contacts

David Miller

Sr. Director of Strategic Communications

pr@curevovaccine.com

Professor Allan Bradley to transition to Chief Scientific Officer, and will continue to drive the Company’s scientific direction

10 March 2025; Cambridge, England – T-Therapeutics, a biotechnology company developing next-generation soluble T cell receptor (TCR) therapeutics targeting cancer and autoimmune indications, announces the appointment of Theodora Harold as its new Chief Executive Officer (CEO). Founder and current CEO of T-Therapeutics Professor Allan Bradley will transition to the role of Chief Scientific Officer.

Theodora has over 25 years’ experience in the biotech sector, with a proven track record of dynamic leadership. She has raised significant capital for multiple companies as well as originating and driving numerous, high-profile, business development deals. She brings strong prior experience at the Board level and has held executive leadership positions in development and clinical stage oncology-focused companies, most recently as CEO of Crescendo Biologics. Theodora qualified as a Chartered Accountant with PricewaterhouseCoopers and graduated from the University of Cambridge.

Since its $60 million Series A fundraise at the end of 2023, T-Therapeutics has launched its OpTiMus® platform which is able to address previously intractable TCR targets with fully human, high affinity binders. The OptTiMus platform has now been used to generate a pipeline of first-in-class TCR therapeutics for cancer indications as well as inflammatory disorders. Theodora’s appointment as CEO is designed to support the Company’s transition into the development phase of its highly differentiated assets.

Professor Allan Bradley, Chief Scientific Officer of T-Therapeutics, said: “I am incredibly proud of what our team has already achieved - from developing a best-in-class transgenic mouse platform to securing the support of high-quality investors, and recently demonstrating ground-breaking preclinical activity with our T cell engaging molecules. Now is the right time to hand on the baton and I’m convinced that Theodora’s energetic leadership, capital markets expertise and deep understanding of the T cell landscape will take us further and faster towards our goal of bringing our next-generation TCR medicines to patients. As CSO, I look forward to working closely with her in the months and years ahead.”

Theodora Harold, newly-appointed Chief Executive Officer of T-Therapeutics, said: “I have been very impressed by T-Therapeutics’ world-leading science, the passion of the team and the first-in-class pipeline. The OpTiMus platform is uniquely able to address certain novel TCR targets and thus has the potential to transform the clinical landscape in both cancer and autoimmune diseases. I look forward to working with the Board of Directors, Allan and the whole team as we take the business into the next phase of its development.”

Dr David Hung, Chairman of T-Therapeutics, added: “Theodora’s appointment as our new CEO is a natural next step for T-Therapeutics and I’m delighted we have secured someone of such calibre to the team, who brings a wealth of complementary knowledge and experience. The team will continue to benefit from Allan’s expertise and leadership in his new role as CSO, overseeing the scientific development of our product candidates.”

Contact Us

T-Therapeutics

Theodora Harold

info@t-therapeutics.com

ICR Healthcare

Amber Fennell, Lucy Featherstone

t-therapeutics@icrhealthcare.com

About T-Therapeutics

T-Therapeutics is a next-generation T cell receptor (TCR) company spun out from the University of Cambridge. The company was created to harness the power of T cell biology, evolved over millions of years, to create safe and effective treatments for many cancers and autoimmune diseases. T-Therapeutics combines world-leading expertise in mouse genome engineering, deep knowledge and experience in biopharmaceutical drug development, single cell genomics, machine-learning and structural biology, anchored in a culture of creativity and collaboration. T-Therapeutics is developing ‘optimal’ TCR therapeutics using a proprietary OpTiMus® platform, based on a fully humanized TCR mouse that provides an almost unlimited source of unique, antigen-specific human TCRs. The company is developing a pipeline of first-in-class drugs that are intended to become transformative medicines, reshaping the clinical landscape for patients with cancer or autoimmune diseases.

The company is backed by blue-chip investors, including Sofinnova Partners, F-Prime, Digitalis Ventures, Cambridge Innovation Capital and Sanofi Ventures.

Oxford, United Kingdom – 26 February 2025 – OMass Therapeutics (‘OMass’ or ‘the Company’), a biotechnology company identifying medicines against highly validated target ecosystems such as membrane proteins or intracellular complexes, today announces that Birgitte Volck MD, PhD has joined its Board as a non-executive director.

Dr Volck has over 25 years of industry experience spanning R&D leadership, platform innovation, and commercialization, with a strong focus on rare diseases and precision medicine. She currently serves on the Board of Soleno Therapeutics and has held Board roles at many leading biotech companies including Nykode Therapeutics, Wilson Therapeutics, and Ascendis Pharma.

Her prior executive roles include Executive Vice President and Interim Chief Medical Officer at Ascendis Pharma, where she led clinical development, regulatory affairs, and medical strategy for the company’s rare disease portfolio and President of R&D at Avrobio Inc, where she oversaw the development of the company’s gene therapy programs. She also served as Senior Vice President, Head of R&D, Rare Diseases at GSK, where she helped shape the company’s rare disease portfolio leveraging internal and external partnerships. Other prior executive leadership roles include developing and launching innovative therapies for specialty care and rare disease indications at Sobi, Amgen, and Genzyme. Furthermore, she has contributed to the rare disease and precision medicine field as speaker and external lecturer. Birgitte earned her MD and PhD in Biomarkers for Arthritis from Copenhagen University.

Dr Volck, non-executive Director at OMass Therapeutics commented, “I have been following the OMass story for a number of years and have been impressed by its innovative approach to drug discovery and the study of protein interactions in their native ecosystem, as well as the Company’s leadership and strategic focus. I am passionate about developing new medicines and look forward to using my experience and network to help guide the team as they prepare to enter the clinic with their potentially best-in-class MC2 antagonist for congenital adrenal hyperplasia and ACTHdependent Cushing’s.”

Jim Geraghty, Chairman of OMass Therapeutics’ Board of directors added, “Birgitte is very well known in the rare diseases community and has a wealth of experience in developing drugs in numerous indications. Ros, the Board and I look forward to capitalising on this deep experience as we approach the next step in our journey as a clinical stage company.”

OMass Therapeutics

Rosamond Deegan, Chief Executive Officer

Phone: +44 (0) 1235 527589

Email: ros.deegan@omass.com

ICR Healthcare

Sue Charles / Ashley Tapp

Phone: +44 (0)20 3709 5700

Email: omass@icrhealthcare.com

About OMass Therapeutics

OMass Therapeutics is a biotechnology company discovering medicines against highly-validated target ecosystems, such as membrane proteins or intracellular complexes.

OdyssION™, OMass’ unique drug discovery platform, comprises next-generation native mass spectrometry with novel biochemistry techniques and custom chemistry to interrogate not just a drug target, but also the interaction of the target with its native ecosystem, separate from the confounding complexity of the cell. This unique approach results in cell-system fidelity with cell-free precision.

OMass is advancing a pipeline of small molecule therapeutics in rare diseases and immunological conditions. Its lead programme is a best-in-class MC2 (melanocortin-2) receptor antagonist for the treatment of Congenital Adrenal Hyperplasia (CAH) and Cushing’s Syndrome. The focus of the program has been to increase receptor residency time to make OMass’ antagonists resistant to competition by the endogenous ligand, thereby avoiding loss of efficacy in the face of rising adrenocorticotropic hormone (ACTH) levels due to reductions in glucocorticoid supplementation for CAH or progression of Cushing’s Syndrome.

Headquartered in Oxford, UK, OMass has raised over $160M (£129M) from a top-tier international investor syndicate including Syncona, Oxford Science Enterprises, GV, Northpond Ventures, Sanofi Ventures and British Patient Capital.

To learn more, please visit www.omass.com. Follow us on LinkedIn.

CAMBRIDGE, Mass., February 24, 2025 – QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases, today announced that company management will participate in the following investor conferences in March. 

T.D. Cowen 45th Annual Health Care Conference (March 3-5, 2025) 

Format:                One-on-one investor meetings

Location:              Boston, MA

Stifel 2025 Virtual CNS Forum (March 18-19, 2025)

Format:                Corporate presentation

Date:                    Wednesday, March 19, 2025

Time:                    3:30 PM ET

Location:              Virtual

The QurAlis corporate presentation can be accessed by visiting the presentations section of the Company’s website at www.quralis.com.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a robust precision medicine pipeline with therapeutic candidates aimed at modifying severe disease pathology in defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on X @QurAlisCo or LinkedIn.

Contact: Kathy Vincent kathy.vincent@quralis.com 310-403-8951

• Series C financing led by NEA, joined by new investor Foresite Capital and Abcuro’s existing investors
• Proceeds to support completion of the registrational Phase 2/3 MUSCLE trial of ulviprubart in inclusion body myositis, BLA filing, and commercial launch preparation

Newton, Massachusetts, February 12, 2025— Abcuro, Inc., a clinical-stage biotechnology company developing therapies for the treatment of autoimmune diseases and cancer through precise modulation of cytotoxic T cells, today announced the closing of a $200 million Series C financing led by New Enterprise Associates (NEA) with Foresite Capital joining the round and participation of existing investors including RA Capital Management, Bain Capital Life Sciences, Redmile Group, Samsara BioCapital, Sanofi Ventures, Pontifax, Mass General Brigham Ventures, New Leaf Ventures, funds managed by abrdn Inc., funds and accounts managed by BlackRock, Eurofarma Ventures, and Soleus Capital.

Proceeds from the Series C financing will be used to complete the registrational Phase 2/3 MUSCLE clinical trial evaluating ulviprubart (ABC008), a first-in-class monoclonal antibody targeting killer cell lectin like receptor G1 (KLRG1), for the treatment of inclusion body myositis (IBM). Assuming positive results from the MUSCLE clinical trial, Abcuro plans to file a BLA and will use a portion of the proceeds to support commercial launch preparation.

“Continued support from all of our investors in this latest financing round validates our vision for the potential that ulviprubart may have as a novel treatment for progressive and devastating diseases mediated by highly cytotoxic T cells, including Inclusion Body Myositis” said Alex Martin, Chief Executive Officer of Abcuro. “We are in a strong position to execute on our clinical development plan, including completing our ongoing, registrational Phase 2/3 MUSCLE clinical trial of ulviprubart in IBM, and expect to report initial data in the first half of 2026. We will also look to fund the expansion of manufacturing capabilities and other pre-commercial activities this year.”

“Abcuro represents an exciting opportunity with its lead candidate, ulviprubart, a potential first-in-class therapy that could make a big impact to the treatment paradigm of IBM, an indication with a significant unmet clinical need,” said Michele Park, PhD, Partner at NEA. “Ulviprubart targets a unique mechanism that can selectively deplete cytotoxic T cells, backed by encouraging clinical and preclinical data that have been presented to date.”

About Ulviprubart

Ulviprubart (ABC008) is a first-in-class anti-KLRG1 antibody product candidate capable of selectively depleting highly cytotoxic T cells, while sparing naïve, regulatory and central memory T cells. Ulviprubart is designed to treat diseases mediated by highly cytotoxic T cells, including the autoimmune muscle disease inclusion body myositis (IBM) and T cell large granular lymphocytic leukemia (T-LGLL). The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have each granted orphan drug designation to ulviprubart for the treatment of IBM.

About Inclusion Body Myositis (IBM)

IBM is an autoimmune disease in which highly cytotoxic T cells chronically attack muscle tissue leading to progressive weakness and limb muscle atrophy. Patients progressively lose muscle function, including loss of grip, dexterity and mobility. There are currently no available disease-modifying treatment options for IBM. Based on published epidemiology literature, it is estimated that there are more than 50,000 people with IBM across the US and Europe.

About Abcuro

Abcuro is a clinical stage biotechnology company developing first-in-class immunotherapies for the treatment of autoimmune diseases and cancer through precise modulation of highly cytotoxic T cells. The company’s lead program is ulviprubart (ABC008) and is currently in clinical trials for inclusion body myositis (IBM) and T cell large granular lymphocytic leukemia.

For more information, visit us on LinkedIn and at abcuro.com.

Contact:

Matthew DeYoung
Investor Relations and Media
Argot Partners
abcuro@argotpartners.com

Brisbane, Australia and Indianapolis, USA – February 10, 2025 – AdvanCell, a clinical-stage radiopharmaceutical company specializing in targeted alpha therapies, today announced an expansion to the scope and breadth of its strategic collaboration with Eli Lilly and Company to research and develop innovative treatments for various cancers.

Under this new agreement, the parties will leverage AdvanCell’s proprietary Pb-212 production technology and radionuclide development infrastructure and Lilly’s drug candidate programs and extensive expertise in drug development to facilitate the development and accelerate the clinical advancement of an expanded portfolio of targeted alpha therapies.

AdvanCell’s competitive advantage in technology development and the infrastructure it has built to accelerate early-stage clinical trials in Australia enables AdvanCell to rapidly develop and progress novel Pb-212-containing radiotherapeutics from discovery into clinical trials.

“This collaboration with Lilly represents a significant milestone for AdvanCell, recognizing our company as one of the leaders in the Pb-212 targeted alpha therapy space,” said Andrew Adamovich, CEO of AdvanCell. “By combining our groundbreaking isotope production capabilities, our team’s expertise and infrastructure with Lilly’s pharmaceutical and oncology expertise and global scale, we aim to bring transformative treatments to patients with hard-to-treat cancers. It is especially pleasing to continue and expand our existing relationship.”

Jacob Van Naarden, President of Lilly Oncology, added, “Partnering with AdvanCell aligns with our commitment to advancing innovative radiopharmaceuticals. We are excited to explore the potential of Pb-212-based alpha therapies as we work to bring meaningful new treatments for patients.”

Financial terms of the agreement were not disclosed.

About AdvanCell
AdvanCell is dedicated to developing innovative cancer therapies that harness the power of targeted alpha-emitting radionuclides. By combining advanced manufacturing capabilities with cutting-edge science and clinical development capabilities, AdvanCell aims to deliver novel treatments that improve outcomes for cancer patients globally. For more information, visit www.advancell.com.au.

Patient-Centered PRIZM Study Measures Impact of Once-Daily Oral Zagociguat on Fatigue and Cognitive Impairment, Hallmark Features of this Rare Mitochondrial Disease

CAMBRIDGE, Mass., January 27, 2025 – Tisento Therapeutics today announced that the first patient has been dosed in its global Phase 2b PRIZM study. The study is investigating the impact of once-daily oral
zagociguat treatment on fatigue, cognitive impairment, and other key aspects of the rare mitochondrial disease MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes).

The PRIZM study is centered in multiple ways on what matters to people living with MELAS. The study incorporates features that maximize convenience for participants, including at-home assessments, the potential for at-home study visits, and door-to-door travel support. In addition, all participants will receive zagociguat during one period of this crossover study, and participants who complete the study may be eligible for continued access via an open-label extension study. Importantly, the clinical outcome
assessments and endpoint strategy for the PRIZM study were informed by Tisento’s interview study in which individuals living with MELAS described the symptoms and impacts of the disease that are most important to them. PRIZM is a global study now enrolling in North America, Europe, and Australia. Patients and families interested in learning more can visit Tisento’s PRIZM page, ClinicalTrials.gov (NCT06402123), or discuss with their physician.

“Since Tisento’s founding, we have been focused on initiating a thoughtful, well-designed clinical study to evaluate zagociguat for the treatment of MELAS, and we are humbled by the enthusiastic response we have received from physicians and the mitochondrial disease community,” said Peter Hecht, PhD, chief executive officer of Tisento. “We are pleased that the first participants are enrolling in the global PRIZM study, which was designed with patient perspectives at the forefront. We will continue to put patients first every step of the way as we work to develop creative solutions to address what matters most for people with serious diseases.”

“MELAS is the most common condition we see in our mitochondrial clinic, with profound effects on patients' daily lives and wellbeing. Right now, we have no approved treatments, making the search for effective therapies absolutely critical,” said Dr. Austin Larson, associate professor of pediatric clinical genetics and metabolism at the University of Colorado School of Medicine and PRIZM study investigator. “I am excited about zagociguat's potential to address both the severe fatigue and cognitive challenges that so significantly impact our MELAS patients.”

About the PRIZM Study
PRIZM – a Phase 2b Randomized, Placebo-Controlled Trial Investigating Zagociguat in MELAS – is evaluating the efficacy and safety of oral zagociguat 15 mg or 30 mg compared to placebo when administered once-daily for 12 weeks in participants with genetically and phenotypically defined MELAS. The PRIZM study has a crossover design, with two 12-week treatment periods separated by a 4-week washout period. All participants will receive zagociguat during one of the 12-week periods and placebo during the other. Participants who complete the study may be eligible for an open-label extension
study.

PRIZM is a global study that will enroll approximately 44 participants at mitochondrial disease centers of excellence in the U.S., Italy, Germany, United Kingdom, Australia, and Canada. For more information, please visit www.tisentotx.com/prizm or ClinicalTrials.gov (NCT06402123) for more information. Interested individuals can also reach out to their physicians for participation details.

About Zagociguat
Zagociguat is a once-daily, oral, clinical-stage investigational medicine with potential to positively impact both peripheral and central nervous system manifestations of mitochondrial diseases. Zagociguat stimulates soluble guanylate cyclase (sGC), an enzyme that is found in virtually every cell in every tissue of the body and is part of a system of cellular mechanisms that control critical physiological functions including neuronal function and blood flow.

A first-in-class, brain-penetrant sGC stimulator, zagociguat is hypothesized to rebalance dysregulated cellular pathways in MELAS. By restoring cellular functions that support mitochondria, zagociguat may help restore mitochondrial energy production and physiological function.

In a Phase 2a study in patients with MELAS, zagociguat exhibited a favorable safety profile, exposure throughout the body including in the central nervous system, and improvements in neuronal function, mitochondrial function, and blood flow in the brain. Zagociguat is currently being evaluated as a treatment for MELAS in the Phase 2b PRIZM study.

For more information, visit www.tisentotx.com/our-science.

About Tisento Therapeutics
Tisento Therapeutics, a privately held biotech company, is developing novel medicines to treat diseases with significant unmet need, beginning with MELAS and other genetic mitochondrial diseases. Tisento means “I hear you” in Italian; our approach to innovation begins with listening to patients and then channeling what we learn into decisive actions that shape our research and clinical programs.

Tisento is guided by a high-caliber internal team of biopharma veterans and an extensive external network of expert physicians, patient advocacy groups, researchers, industry-leading vendors, and other close collaborators who are partners in our mission to develop meaningful treatments for mitochondrial diseases.

Learn more at our website, www.tisentotx.com, or connect with us on LinkedIn (Tisento Therapeutics) or X (@tisentotx).

Contact

Tisento Media Relations
Jessi Rennekamp, Astrior Communications
Email: jessi@astriorcomms.com

Sydney, Australia – 3 February 2025 – AdvanCell, a clinical-stage radiopharmaceutical company developing innovative cancer therapeutics, today announced the successful completion of an oversubscribed US$112 million Series C financing. This milestone funding round was co-led by SV Health Investors, Sanofi Ventures, Abingworth, and SymBiosis. Additional support came from existing investor Morningside, alongside new investors Tenmile, Brandon Capital, and others.

Since its founding in June 2019, AdvanCell has grown from a belief in the potential of Targeted Alpha Therapy into a global organisation with 60 passionate team members, a 40,000-square- foot manufacturing facility, world-class pre-clinical infrastructure, a potentially best-in-class drug for prostate cancer that is in trials, and a deep and developing pipeline of assets.

This investment will fuel AdvanCell’s ongoing efforts to expand its manufacturing capacity, accelerate the clinical development of its pipeline of radionuclide therapies, and advance its mission of delivering life-changing treatments to cancer patients worldwide.

AdvanCell is currently enrolling patients for the highest dose cohort of its multi-center TheraPb Ph I/II dose escalation clinical trial of ADVC001 for metastatic prostate cancer, a potentially best-in-class Targeted Alpha Therapy. The trial aims to demonstrate the safety and efficacy of Pb-212-based radionuclide treatment.

As part of the financing, Jamil M. Beg from SV Health Investors, Christopher Gagliardi from Sanofi Ventures, and Bali Muralidhar from Abingworth have joined the AdvanCell Board of Directors. They bring extensive industry expertise to support the company’s growth and join existing directors Bill Ferris AC, Anthony Aiudi, Kevin Cameron, and Andrew Adamovich.

Jamil M. Beg, Partner at SV Health Investors, commented:

“We are delighted to support AdvanCell’s growth and play a role in its remarkable journey through leading this oversubscribed Series C. We have looked long and deep at the field of radionuclide therapies and are confident that AdvanCell stands out with a first-in-class Pb-212- PSMA program and a best-in-class manufacturing platform. The company’s exceptional team, technologies, robust infrastructure, collaborations with some of the world’s largest pharma companies and ability to consistently execute, position it to truly change outcomes for patients. We are excited to help AdvanCell realize its potential in transforming cancer care globally.”

AdvanCell was founded on the belief that Targeted Alpha Therapies could change the course of cancer treatment and that the scalable supply of isotopes would enable the development of multiple practice-changing drugs. Radionuclide therapies for prostate cancer and gastroenteropancreatic neuroendocrine tumours have transformed patient care. Pb-212 Targeted Alpha Therapy has the potential to further advance this progress by leveraging the radiobiological and physical attributes of Pb-212 to make life-changing treatments for patients.

“This successful Series C round demonstrates strong confidence in our vision and capabilities,” said Andrew Adamovich, CEO of AdvanCell. “We are grateful for the continued support from our existing investors, particularly the long-term support from Morningside and are excited to welcome new partners who share our commitment to transforming cancer care. With this funding, AdvanCell is well-positioned to scale our manufacturing operations and progress our cutting-edge therapies towards commercialization.”

The Series C funding represents a significant step in AdvanCell’s journey to become a global leader in radionuclide-based cancer therapeutics.

For media inquiries, please contact:
Andrew Adamovich CEO
Email: contact@advancell.com.au
Phone: +612 8000 4199

About AdvanCell
AdvanCell is dedicated to developing innovative cancer therapies that harness the power of targeted alpha-emitting radionuclides. By combining advanced manufacturing capabilities with cutting-edge science and clinical development capabilities, AdvanCell aims to deliver novel treatments that improve outcomes for cancer patients globally. For more information, visit www.advancell.com.au.

About SV Health Investors
SV Health Investors is a leading healthcare fund manager committed to investing in tomorrow’s healthcare breakthroughs. The SV funds invest across stages, geographic regions, and sectors, with expertise spanning biotechnology, dementia, medical devices, healthcare growth and healthcare technology. With approximately $2bn in assets under management and a truly transatlantic presence with offices in Boston and London, SV has built an extensive network of talented investment professionals and experienced industry veterans. Since its founding in 1993, SV has invested in, created and built more than 200 companies attracting global talent, entrepreneurs and pharma partners. To date, these investments have resulted in the licensing of 26 novel drugs and six new drug classes able to treat indications with unmet medical needs and deliver positive impact to patients. For more information, please visit www.svhealthinvestors.com.

About Sanofi Ventures
Sanofi Ventures is the corporate venture capital arm of Sanofi, focused on investing in promising early-stage healthcare companies. The firm supports pioneering innovations in biotechnology, digital health, and life sciences that align with Sanofi’s mission to bring life- changing treatments to patients worldwide. For more information, please visit www.sanofiventures.com.

About Abingworth
Abingworth is a leading transatlantic life sciences investment firm and part of the global investment firm Carlyle (NASDAQ: CG). Abingworth helps transform cutting-edge science into novel medicines by providing capital and expertise to top calibre management teams building world-class companies. Since 1973, Abingworth has invested in over 185 life science companies, leading to 50+ M&As and more than 75 IPOs. Our therapeutic focused investments fall into three categories: seed and early-stage, development stage, and clinical co- development. Abingworth supports its portfolio companies with a team of experienced professionals at offices in London, Menlo Park (California), and Boston. For more information, please visit www.abingworth.com.

About SymBiosis Capital Management
SymBiosis is an investment firm focused on advancing biotherapeutics innovations for serious and life-threatening diseases. The firm invests in groundbreaking medicines across disease areas, financing stages, and geography, with a focus on programs in, or about to enter, human trials. SymBiosis currently manages a portfolio of more than 30 investments and has significant, long-term capital commitments to fund future investments. For more information, please visit www.symbiosis.vc or follow us on LinkedIn.

– IND submissions planned in 2025 for first two programs within leading portfolio of di- siRNA therapies –

– Series B funds will support progressing programs for KCNT1-related epilepsy and Huntington’s disease through clinical proof-of-concept –

BOSTON, January 28, 2025 – Atalanta Therapeutics, a biotechnology company pioneering RNA interference (RNAi) for the treatment of neurological diseases, today announced the completion of a $97 million Series B financing to support Phase 1 clinical trials of the company’s investigational RNAi therapies for KCNT1-related epilepsy and Huntington’s disease. The financing was co-led by EQT Life Sciences and Sanofi Ventures, with participation from other new investors RiverVest Venture Partners, funds managed by abrdn Inc, Novartis Venture Fund, Pictet Alternative Advisors, Mirae Asset Financial Group, and GHR Foundation alongside existing investor F-Prime Capital.

“This financing validates the truly transformative potential of Atalanta’s best-in-class di-siRNA platform for delivering oligonucleotide therapies to the central nervous system and the exciting promise of our expansive wholly-owned pipeline,” said Alicia Secor, Atalanta’s president and chief executive oXicer. “Importantly, this Series B will support a path to the clinic for two programs for serious neurological diseases that today lack disease-modifying therapies — KCNT1-related epilepsy and Huntington’s disease — and will anchor our growing franchise of investigational medicines for Huntington’s disease. We are diligently progressing these programs toward IND submissions this year so that we can start our Phase 1 trials and reach patients who are waiting.”

In conjunction with this financing, the company announced the appointments of Arno de Wilde, M.D., Ph.D., MBA, managing director at EQT Life Sciences; Jason Hafler, Ph.D., managing director of Sanofi Ventures; and Niall O’Donnell, Ph.D., managing director of RiverVest Venture Partners to its Board of Directors.

“Atalanta’s di-siRNA technology has shown promising ability to durably silence disease-promoting genes throughout previously inaccessible regions of the brain and spinal cord — opening a wide range of treatment possibilities for devastating neurological diseases,” said Dr. de Wilde. “EQT Life Sciences is proud to co-lead this investment in Atalanta’s future as part of such a high-quality investor syndicate, and we look forward to partnering with Atalanta’s leadership to support their continued success.”

“We are excited to partner with Atalanta as they enter their next chapter as a clinical-stage company,” said Dr. Hafler. “Their success to-date is a strong validation of their ability to create meaningful new RNAi therapies, and Sanofi Ventures is glad to support Atalanta as they advance their pipeline.”

ATL-201 is Atalanta’s investigational therapy for KCNT1-related epilepsy, an early-onset seizure disorder and encephalopathy driven by gain-of-function variants in the KCNT1 gene. Infants and children with KCNT1-related epilepsy have severe, frequent seizures that are unable to be controlled with anti-seizure medications, and they often experience developmental delays and intellectual disability. ATL-201 is designed to reduce KCNT1 levels and to normalize neuronal excitability. Preclinical studies have shown that ATL-201 produces a significant reduction of seizures and improvement in behavior with impressive durability and tolerability.

The company’s second development candidate, ATL-101, is a di-siRNA designed to silence the HTT gene for the treatment of Huntington’s disease. Huntington’s disease is a progressive neurodegenerative disease caused by an expansion of the HTT gene, which leads to deterioration in a person’s physical, cognitive, and psychiatric abilities. Preclinical studies have shown that a single dose of ATL-101 produces a potent and strong reduction in HTT expression, including in deep brain regions, with six months of durability and excellent tolerability.

A full overview of the company’s pipeline, disclosed today, is available here.

About Atalanta Therapeutics
Atalanta Therapeutics is a biotechnology company developing treatments for intractable diseases of the central nervous system using RNA interference. Atalanta’s unique platform of divalent small interfering RNA (di-siRNA) enables durable, selective gene silencing throughout the brain and spinal cord. Atalanta is advancing a wholly owned pipeline of disease-modifying programs for Huntington’s disease, genetic epilepsy, severe chronic pain, and other neurological diseases in addition to partnered programs as part of a strategic collaboration with Genentech. Atalanta is headquartered in Boston, Mass. For more information, visit www.atalantatx.com.

Media Contact:
Michael Galfetti
Ten Bridge Communications
mgalfetti@tenbridgecommunications.com

CAMBRIDGE, Mass., January 7, 2025 – QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases, today announced that it has been invited to present at the 43rd Annual J.P. Morgan Healthcare Conference to be held January 13-16, 2025, in San Francisco, CA.

Kasper Roet, Ph.D., QurAlis’ chief executive officer and co-founder, will present a corporate overview on Monday, January 13, 2025 at 5:30PM PT at the Westin St. Francis in the Golden Gate room.

The QurAlis corporate presentation can be accessed by visiting the presentations section of the Company’s website at www.quralis.com.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a robust precision medicine pipeline with therapeutic candidates aimed at modifying severe disease pathology in defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on

X @QurAlisCo or LinkedIn.

Contact:

Kathy Vincent

kathy.vincent@quralis.com

310-403-8951

Financing co-led by Samsara Biocapital and Enavate Sciences, with participation from Pfizer Ventures and Regeneron Ventures in addition to other new and existing investors

Normunity’s lead program is a novel T cell engager against a tumor-specific target that is relevant in multiple solid tumors and has the potential to be leveraged for a range of biologic modalities

Boston, Mass., and West Haven, Conn. – January 13, 2024 – Normunity, a biotechnology company creating novel anti-cancer therapies, today announced that it has closed a Series B financing for $75 million. The financing was co-led by Samsara BioCapital and Enavate Sciences, alongside other new investors Regeneron Ventures, Pfizer Ventures and YK Bioventures, as well as existing investors Canaan Partners, Sanofi Ventures, Taiho Ventures, Osage Venture Partners, HongShan Capital Group, and Connecticut Innovations. The Board of Directors will be expanded to include David Parry, PhD (Samsara), Sara Nayeem, MD (Enavate) and Marie-Clare Peakman, MD (Pfizer).

“We are delighted to secure this financing from such an experienced and well-respected syndicate of life sciences investors. Their support will enable Normunity to continue our strong momentum and advance our lead drug program NRM-823 into the clinic this year and explore other modalities with this exciting tumor specific target,” said Rachel Humphrey, MD, founding Chief Executive Officer of Normunity. “With our proprietary target discovery process, we will continue to build our pipeline around novel targets, with new biological insights that could translate into life-changing medicines for cancer patients.”

Proceeds from the financing will be used to advance Normunity’s lead program, NRM‑823, a first-in-class T cell engager that binds a novel, highly specific tumor target expressed on multiple types of solid tumors. The company plans to initiate the Phase 1 clinical trial of NRM-823 in 2H 2025 and leverage its prior work by advancing other modalities against this tumor-specific target, including antibody‑drug conjugates and radiotheranostics. The funding will also be used to advance Normunity’s pipeline of programs that address novel targets responsible for tumor-specific immune suppression. These pipeline candidates derive from Normunity’s proprietary target discovery process, conducted in collaboration with the lab of Professor Lieping Chen, MD PhD, at the Yale University School of Medicine, and built on insights into untapped biological mechanisms that occur from the complex interactions of the immune system and cancer.

“Normunity has an outstanding team that has made impressive progress in discovering and advancing an exciting cancer drug program in NRM-823, which has the potential to address a previously unrecognized target that plays a role in supporting cancer survival across a range of solid tumors,” said David Parry, PhD, Venture Partner at Samsara BioCapital. “We look forward to working with the Normunity team as they continue to advance NRM-823 and other novel agents in the pipeline.”

“The T cell engager space has seen multiple recent successes in solid tumors, and we believe this modality is poised to transform the treatment of numerous cancers in the coming years,” said Sara Nayeem, MD, EVP, Investments at Enavate Sciences.  “We are excited to support the development of NRM-823, which has many characteristics of an ideal T cell engager and has demonstrated compelling preclinical efficacy and safety.”

About Normunity

Normunity, is a biotechnology company creating novel anti-cancer therapies that address untapped biology at the interface of the immune system and the tumor to target mechanisms that impact tumor growth and circumvent immune surveillance and tumor clearance. The company is using these novel targets to build a pipeline of anti-cancer medicines, including therapeutic antibodies, bispecific antibodies, and payload-carrying biologics. The company’s lead program, NRM‑823, is a T cell engager with tumor-specific targeting for multiple solid tumors and is expected to enter the clinic in 2025. Normunity is located in Boston, MA, and New Haven, CT. For more information, please visit www.normunity.com and follow us on LinkedIn.

Media Contact

Kathryn Morris, The Yates Network LLC

914-204-6412

kathryn@theyatesnetwork.com

• Acquisition adds potential best-in-class disease-modifying therapy for Alzheimer's disease, ALIA-1758, and novel blood-brain barrier (BBB)-crossing technology to strengthen neuroscience pipeline and R&D capabilities 

NORTH CHICAGO, Ill., Dec. 11, 2024 /PRNewswire/ -- AbbVie (NYSE: ABBV) announced today that it has completed its acquisition of Aliada Therapeutics. With the completion of the acquisition, Aliada is now a part of AbbVie. 

Aliada's lead investigational asset is ALIA-1758, an anti-pyroglutamate amyloid beta (3pE-Aβ) antibody, which is in development for the treatment of patients with Alzheimer's disease and is currently in a Phase 1 clinical trial. ALIA-1758 utilizes a novel blood-brain barrier (BBB)-crossing technology that enhances delivery of targeted drugs into the central nervous system (CNS). 

"Alzheimer's disease poses a significant public health challenge, impacting millions worldwide and is becoming more prevalent as populations age," said Dawn Carlson, M.D., M.P.H., vice president, neuroscience development at AbbVie. "With the acquisition now complete, we look forward to advancing potentially disease-modifying therapies such as ALIA-1758 for Alzheimer's disease and bolstering our neuroscience discovery and development efforts by leveraging Aliada's novel CNS drug delivery platform." 

For additional background on the acquisition, please read the announcement press release here. 

About AbbVie 

AbbVie's mission is to discover and deliver innovative medicines and solutions that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas – immunology, oncology, neuroscience, and eye care – and products and services in our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on LinkedIn, Facebook, Instagram, X (formerly Twitter), and YouTube. 

Forward-Looking Statements  

Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2023 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law.  

For further information: Media: Elizabeth Tang, Ph.D., liz.tang@abbvie.com; Investors: Liz Shea, liz.shea@abbvie.com 

Menlo Park, Calif., December 5, 2024 – Nuvig Therapeutics, Inc., (“Nuvig”) a privately held biotechnology company developing novel immunomodulatory therapeutics for patients with inflammatory autoimmune diseases, announced today the closing of a $161 million Series B financing. The financing was co-led by Sanofi Ventures, Blue Owl Healthcare Opportunities (formerly Cowen Healthcare Investments), and Norwest Venture Partners, with participation from new investors, B Capital, Leaps by Bayer, Global BioAccess Fund, LOTTE Holdings, Alexandria Venture Investments, and funds managed by abrdn Inc., and existing shareholders, Novo Holdings A/S, Platanus, Bristol Myers Squibb, Digitalis Ventures, and Mission BioCapital.  

“We are delighted to partner with additional high-caliber investors. Their scientific and strategic perspectives will support our efforts to diversify our pipeline and bring potentially transformative medicines to patients,” said Pamela Conley, Ph.D., Cofounder, Chief Scientific Officer, and founding Chief Executive Officer of Nuvig Therapeutics.  

Nuvig aims to deliver novel methods for reducing autoimmune dysregulation without immunosuppression. Lead candidate NVG-2089, a first-in-class, recombinant, Fc fragment immunomodulator, is engineered to bind type II Fc receptors and engage an endogenous regulatory mechanism that improves autoimmune dysregulation. The proceeds from the Series B financing will support clinical proof-of-concept studies for NVG-2089 and advance Nuvig’s preclinical pipeline. The company is progressing NVG-2089 to Phase 2 clinical development in chronic inflammatory demyelinating polyneuropathy (CIDP) and other undisclosed indications for which there is high unmet need for non-immunosuppressive, efficacious new therapies.  

Dosing in the Phase 1 study for NVG-2089 has been successfully completed. In Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) studies, NVG-2089 was safe and well-tolerated, the pharmacokinetic data were dose proportional, and pharmacodynamic data demonstrated target engagement. The positive results from Phase 1 support the progression of NVG-2089 to Phase 2 studies across multiple indications. 

In conjunction with the financing, Paulina Hill, Ph.D., Partner at Sanofi Ventures, Tim Anderson, Managing Director at Blue Owl Capital, and Tiba Aynechi, Ph.D., General Partner at Norwest Venture Partners, joined Nuvig’s Board of Directors.  

Dr. Hill said, “Nuvig’s innovative and immunomodulatory approach to inflammatory and autoimmune diseases is well aligned with Sanofi Ventures’ development and investment philosophy. We are impressed by the detailed progress performed by the accomplished team of scientists and executives at Nuvig. They have not only demonstrated powerful anti-inflammatory effects of their lead candidate NVG-2089 without immunosuppression but have also created a reproducible and scalable recombinant method of recapitulating the effects of IVIg without the downsides and supply limitations of IVIg.” 

Mr. Anderson added, “We are happy to be a partner to Nuvig on the Series B financing and are very pleased with the recent advancements at Nuvig. In particular, we are excited about Nuvig’s ability to demonstrate that the specific biology of NVG-2089 is engaging the relevant mechanistic pathways in early clinical studies and look forward to further advancing NVG-2089 in the clinic in indications where Nuvig can offer significant advances in safety and efficacy.” 

Dr. Aynechi concluded, “We are pleased to support Nuvig’s continued progress towards delivering powerful and safe immune-modulating therapies in autoimmune therapeutic areas where there is significant unmet need. We believe that NVG-2089 has life-changing potential for patients who otherwise have limited treatment options, and Nuvig’s platform programs show promise for applications across a wide range of autoimmune diseases.”  

In addition, Nuvig also appointed as independent members of the Nuvig Board of Directors: Ciara Kennedy, Ph.D., founder, President, and CEO of Sorriso Pharmaceuticals, and James Mackay, Ph.D., who most recently was the founder, President, and CEO of Aristea Therapeutics and currently serves as the founder and CEO of Kateran Consulting.  

About Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) 

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a neurological autoimmune disorder characterized by progressive weakness and impaired sensory function in the legs and arms. Symptoms typically include symmetrical muscle weakness leading to difficulties with walking, climbing stairs, and performing fine motor tasks. Sensory disturbances such as numbness, tingling, or burning sensations. Additionally, diminished or absent reflexes are a common clinical finding. Fatigue is frequent, significantly impacting the patient's quality of life. CIDP symptoms can vary in severity and may progress over several months. Diagnosis is typically confirmed through nerve conduction studies, which reveal demyelination, and cerebrospinal fluid analysis showing elevated protein levels.  

About Nuvig Therapeutics 

Nuvig Therapeutics is a clinical-stage biotechnology company that is advancing an innovative and transformational pipeline of novel immune therapeutics for chronic inflammatory and autoimmune diseases. The Company’s lead investigational drug candidate, NVG-2089, is an engineered Fc fragment designed to precisely target type II Fc receptors. When NVG-2089 binds to its target, it upregulates the expression of FcgRIIb and causes the expansion of T regulatory cells and the downregulation of numerous inflammatory pathways. Nuvig is based in Menlo Park, California. For more information, please visit www.nuvigtherapeutics.com. 

Contacts 

Corporate:
info@nuvigtx.com 

Media:
Jessica Yingling, Ph.D.,
Little Dog Communications Inc.,
jessica@litldog.com
+1.858.344.8091 

Multi-year effort leverages Muna's all-in-human MiND-MAP spatial multi-omics approach to identify and validate new drug targets and treatment pathways for Alzheimer’s disease

GSK secures option to multiple high-value, validated Alzheimer’s-relevant targets for drug discovery, development, and commercialization

Copenhagen, Denmark, December 5, 2024 –Muna Therapeutics (Muna), a biotechnology company focused on developing innovative therapeutics for neurodegenerative diseases, today announced a research alliance with GSK to identify and validate novel drug targets for the treatment of Alzheimer’s disease. The companies will explore insights from Muna’s MiND-MAP platform, which applies spatial transcriptomics to brain samples from Alzheimer’s disease patients, cognitively resilient individuals, healthy controls, and centenarians with and without cognitive impairment. This unique dataset of exceptional breadth and resolution will fuel the discovery and development of innovative medicines for Alzheimer's disease.

Together, Muna and GSK will assess postmortem human brain samples with spatial transcriptomics and other approaches to identify and validate potential new drug targets. The collaboration leverages Muna’s deep expertise in mapping the brain’s response to pathological protein aggregates and its all-in-human platform to identify cellular mechanisms, gene networks, and molecular interactions that underlie brain resilience. Candidate drug targets will be validated using Muna’s suite of humanized cell and animal models, supported by insights from patient tissue and biofluid samples.

“Our agreement marks a pivotal moment in Muna’s evolution and in the broader Alzheimer's research landscape,” said Rita Balice-Gordon, Ph.D., Muna’s Chief Executive Officer. "By combining GSK's commitment to breakthrough science with our MiND-MAP platform's ability to deliver novel insights into brain resilience, we aim to transform the landscape of drug discovery for neurodegenerative diseases and bring new hope to millions of patients worldwide."

Under the terms of the agreement, Muna will receive an upfront payment from GSK of €33.5 million. In addition, Muna will be eligible to receive up to €140 million per target in milestone payments, as well as tiered royalties on net sales of products. Muna will expand and enhance its existing MiND-MAP dataset and will lead the identification and validation of new Alzheimer’s disease targets. GSK will lead drug development and be responsible for preclinical activities, clinical development, manufacturing, and commercialization resulting from work on targets discovered and validated in the collaboration.

“By applying spatial multi-omics to unique patient phenotypes, Muna's MiND-MAP platform is able to determine the genetic and cellular basis of progression and resilience in neurodegenerative diseases,” said Kaivan Khavandi M.D., Ph.D., SVP & Global Head of Respiratory/Immunology R&D at GSK. “The alliance exemplifies our discovery ethos, to utilize advanced data and platform tech to identify high-confidence, human-data-derived, causal targets, which we can support with GSK’s scale and expertise in clinical development and commercialization, to bring desperately needed new therapeutic solutions in Alzheimer’s disease.”

About Muna Therapeutics

Muna Therapeutics discovers and develops therapies that slow or stop devastating neurodegenerative diseases including Alzheimer’s and Parkinson’s disease. These disorders impact memory, movement, language, behavior and personality, resulting in disability and death of millions of patients around the globe. Muna focuses its groundbreaking science on identifying new medicines to preserve cognition and other brain functions, enhance resilience to disease pathology, and slow or stop the progression of neurodegenerative diseases. Its name reflects this focus: Muna means ‘to remember’ in Old Norse. For more information, visit www.munatherapeutics.com. Follow Muna on Linkedin.

###

Media Contact:

Lia Dangelico

Deerfield Group

Email: lia.dangelico@deerfieldgroup.com

QRL-101 is the only Kv7 ion channel opener being actively studied for the treatment of hyperexcitability-induced disease progression in ALS, which occurs in nearly 50 percent of patients

Kv7 is a clinically validated target to regulate hyperexcitable state in epilepsy

QurAlis’ Phase 1 study in healthy volunteers of QRL-101 to evaluate biomarkers of both ALS and epilepsy also underway

CAMBRIDGE, Mass., December 4, 2024 – QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases, today announced that the first patient with ALS has been dosed in a Phase 1 clinical trial evaluating QRL-101 in people living with ALS (QRL-101-04). QRL-101 is a first-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS, which is present in approximately 50 percent of ALS patients. 

Kv7 hyperexcitability occurs in both sporadic and genetic forms of ALS, with the majority caused by the mis-splicing of the KCNQ2 gene pre-mRNA. Kv7 is also a clinically validated target to regulate the hyperexcitable state in epilepsy. QurAlis recently announced the company has expanded the development program for QRL-101 to include epilepsy.

“QRL-101 is the only Kv7 ion channel opener being actively studied for the treatment of hyperexcitabilityinduced disease progression in ALS. The dosing of the first patient with ALS in the clinical development program of QRL-101 is a significant milestone,” said Kasper Roet, Ph.D., CEO and co-founder of QurAlis. “Kv7 is implicated in ALS as well as epilepsy. We believe that the data from this study, along with the data from our Phase 1 study evaluating biomarkers of ALS and epilepsy in healthy volunteers, will be valuable as we advance the clinical program for QRL-101 in ALS so that we can bring a much-needed therapeutic option to patients rapidly.”

“Preclinical models of QRL-101 show its strong potential to control motor neuron hyperexcitabilityinduced neurodegeneration with an attractive side effect profile,” said Leonard H. van den Berg, M.D., Ph.D., professor of neurology and chair, TRICALS. “ALS is a devastating, fatal neurodegenerative disease and there are currently no therapies that can significantly extend patients’ lives. We look forward to results from this Phase 1 study in ALS patients.” 

QRL-101-04 (NCT06714396) is a Phase 1 proof-of-mechanism (PoM) single-dose, placebo-controlled clinical trial designed to evaluate the safety and tolerability of QRL-101 in people living with ALS. The study is expected to enroll approximately 12 participants with ALS and will evaluate the impact of QRL-101 on excitability biomarkers including on the strength-duration time constant (SDTC), a known predictor of survival in ALS patients. QurAlis is also conducting a Phase 1 PoM biomarker clinical trial (QRL-101-05; NCT06681441) to evaluate biomarkers of ALS and epilepsy of QRL-101 in healthy volunteers. These PoM studies together will inform QurAlis’ future development program, including dose levels for proof-of-concept studies.

QurAlis anticipates reporting topline data from the QRL-101-04 Phase 1 clinical trial in the first half of 2025.

More information about the QRL-101 Phase 1 clinical trials can be found at www.clinicaltrials.gov.

About Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease impacting nerve cells in the brain and spinal cord, reducing muscle function and control. ALS can be traced to mutations in more than 25 different genes and is often caused by a combination of multiple sub-forms of the condition. Cases usually cannot be predicted, although a small percentage are inherited. ALS has a devastating impact on patients and families. ALS patients’ average life expectancy is three years; after diagnosis patients only have two years to live. There is currently no cure for the disease.

About Kv7

Kv7.2/7.3 is a voltage-gated potassium channel whose role is crucial for the regulation of neuronal excitability and membrane potential. Kv7.2 is mis-spliced in sporadic amyotrophic lateral sclerosis (ALS) leading to loss of function and abnormal electrical activity in the spinal cord and brain. The activation of this channel shows the potential to decrease spinal and cortical/motor neuron excitability and to positively affect CMAP (compound muscle action potential), a disease progression biomarker for ALS. This suggests that this may be an effective therapeutic approach for ALS patients suffering from hyperexcitability-induced motor neuron degeneration.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a robust precision medicine pipeline with therapeutic candidates aimed at modifying severe disease pathology in defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on X @QurAlisCo or LinkedIn.

Contact:

Kathy Vincent

kathy.vincent@quralis.com

310-403-8951

• SUDO-550 is a potential best-in-class, orally administered, brain-penetrant TYK2 inhibitorbeing developed for the treatment of neuroinflammatory diseases.
• Phase 1 clinical trial supported by preclinical data demonstrating high potency, selectivity,and efficacy in preclinical models, along with the ability to cross the blood-brain barrier.

CARMEL, Ind.--(BUSINESS WIRE)-- Sudo Biosciences (“Sudo”), a biopharmaceutical company committed to designing and developing best-in-class precision TYK2 (tyrosine kinase 2) inhibitors, announced today that the first participants have been dosed in a Phase 1 clinical trial evaluating SUDO-550, a novel brain-penetrant allosteric TYK2 inhibitor for the treatment of neuroinflammatory diseases.

The Phase 1 clinical trial is designed to evaluate the safety, tolerability, and pharmacokinetics of single and multiple- ascending doses of SUDO-550 in healthy volunteers, including confirmation that the compound effectively crosses the blood-brain barrier.

“Entering the clinic is a critical step in the development of SUDO-550 and establishing it as a best-in-class brain-penetrant TYK2 inhibitor. This is a therapy that could significantly advance the treatment of diseases such as multiple sclerosis, ALS and Alzheimer’s,” said Ian Mills, Chief Medical Officer, Sudo Biosciences. “This is the second allosteric TYK2 inhibitor we have advanced into the clinic this year, after SUDO-286 which is progressing in two Phase 1 trials as a topical treatment for psoriasis.”

The company is developing multiple highly potent and selective small molecule TYK2 pseudokinase inhibitors designed to provide targeted treatments across a broad range of autoimmune and neurologic conditions.

About SUDO-550

SUDO-550 is an orally administered, allosteric TYK2 inhibitor that demonstrates high selectivity and potency for TYK2, minimizing off-target effects. Non-clinical studies have demonstrated excellent blood-brain barrier penetration with the compound, enabling therapeutic potential for CNS diseases characterized by compartmentalized neuroinflammation. These results support its potential as a best-in-class treatment for multiple neuroinflammatory diseases.

About SUDO-286

SUDO-286 is a highly potent, selective and potential first and best-in-class topical TYK2 inhibitor for psoriasis and other immune-mediated dermatologic diseases. The program entered the clinic earlier this year and is currently being evaluated in two Phase 1 studies in healthy volunteers and patients.

About Sudo Biosciences

Sudo Biosciences is a biopharmaceutical company committed to designing and developing novel medicines to transform patients’ lives. The company’s programs target the tyrosine kinase 2 (TYK2) pseudokinase domain. TYK2 is a key mediator in cytokine signaling pathways that have been linked to a broad range of immune-mediated inflammatory conditions. The company’s pipeline of next generation TYK2 inhibitors includes a potential first- and best-in-class brain- penetrant candidate for the treatment of multiple sclerosis and neurodegenerative diseases with underlying neuroinflammation and a potential first- and best-in-class topical candidate for immune-mediated dermatologic diseases. Sudo Biosciences is based in Carmel, IN, with operations across the US and UK. For more information, visit www.sudobio.com.

Contacts Media

Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com

Source: Sudo Biosciences

• PK data analysis in dose-escalation phase indicated exposures of QRL-201 met or exceeded the targeted
• therapeutic range prompting advancement to the DRF phase
• ANQUR protocol amended to include additional biomarkers and cohort of participants with C9orf72-related ALS; Company to present update at 35th International Symposium on ALS/MND

CAMBRIDGE, Mass., Nov. 19, 2024 /PRNewswire/ -- QurAlis Corporation, a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases, today announced that the Phase 1 ANQUR clinical trial evaluating QRL-201 for the treatment of ALS has successfully completed the dose-escalation phase, based on pharmacokinetic (PK) data analysis from Cohorts 1 and 2, indicating cerebrospinal fluid (CSF) exposure levels of QRL-201 met or exceeded the targeted therapeutic range. QRL-201 is a first-in-class precision therapeutic product candidate that has the potential to restore STATHMIN-2 (STMN2) expression in ALS patients with the aim to modify disease progression and improve outcomes.

The ANQUR clinical trial (QRL-201-01; NCT05633459) has advanced to the dose range-finding (DRF) phase which will evaluate two doses of QRL-201 and include additional biomarkers. The study design will now also include a cohort of participants with C9orf72-related ALS in addition to participants with sporadic ALS. Based on previous expression analysis, patients with C9orf72-related ALS show consistent mis-splicing of STMN2. The ANQUR clinical trial is currently active in Canada, the United Kingdom (UK), and the European Union (EU). Cohorts 1 and 2 have successfully completed dosing and an amendment to the DRF study design has been approved in Canada and the UK, with anticipated approval from the EU in the near future. The first participant in the DRF phase has been dosed in Canada.

"ALS is a devastating, fatal neurodegenerative disease with a large unmet medical need. Our mission is to make a meaningful difference in patients' lives, and we believe QRL-201 could potentially modify disease progression and improve outcomes in ALS patients who have a loss of STMN2 due to TDP-43 pathology," said Kasper Roet, Ph.D., CEO and co-founder of QurAlis. "The PK data analysis from the first two completed cohorts of ANQUR indicated CSF exposures of QRL-201 met or exceeded the targeted therapeutic range. These findings further support our confidence in the potential therapeutic effect of QRL-201 in the treatment of sporadic ALS."

STMN2 is a well-validated protein important for neural repair, axonal stability, and muscle innervation and is the most significantly regulated gene by TDP-43 exclusively in humans. STMN2 is the most consistently decreased gene across multiple ALS RNA expression studies. Loss of nuclear TDP-43 leads to mis-splicing of the pre-mRNA of STMN2, resulting in loss of full-length transcript and protein. QRL-201 rescues STMN2 loss of function in QurAlis' ALS patient-derived motor neuron disease models in the presence of TDP-43 pathology. TDP-43 pathology is associated with nearly all ALS patients, approximately 50 percent of patients with FTD, the second most common form of dementia, and with about a third of Alzheimer's Disease patients. There are currently no cures for ALS or FTD, and there are limited therapeutic options available for ALS and FTD patients who are in desperate need of effective therapies.

"We are pleased that the first participant in Canada has been dosed in the dose range-finding stage of the ANQUR clinical trial," said Doug Williamson, M.D., QurAlis' chief medical officer. "This represents a significant milestone in the QRL-201 program to evaluate a potential transformative breakthrough precision medicine for patients with sporadic ALS."

QurAlis will present an update on the ANQUR clinical trial in a poster presentation at the 35th International Symposium on ALS/MND being held December 6-8, 2024, in Montreal, Canada and virtually. Details of the presentation are as follows:

Title: QRL-201-01 (ANQUR): A multicenter, randomized, double-blind, placebo-controlled multiple ascending dose study to evaluate the safety and tolerability of QRL-201 in amyotrophic lateral sclerosis

Date/Time: Saturday, December 7, 2024 5:00-7:00PM ET

Theme: Clinical Trials and Trial Design

Abstract Number: CLT-11

Session: Poster Session B

Presenter: Emma Bowden, Ph.D., senior vice president, head of clinical development, QurAlis

About the ANQUR Clinical Trial

ANQUR (QRL-201-01; NCT05633459) is the first-ever clinical trial to evaluate a potential therapy to rescue STATHMIN-2 (STMN2) expression in people with amyotrophic lateral sclerosis (ALS). ANQUR is a global, multi-center, randomized, double-blind, placebo-controlled multiple-ascending dose Phase 1 clinical trial designed to evaluate the safety and tolerability of QRL-201 versus placebo in participants with ALS. The primary objective and endpoint of the study is to determine the safety and tolerability of multiple doses of QRL-201. The secondary objective and endpoint is the plasma pharmacokinetic (PK) profile of QRL-201 after multiple doses. ANQUR also intends to evaluate multiple exploratory endpoints including biomarkers of neuronal loss and STMN2 biology (neurofilament levels, STMN2 levels, Chitinase-3-like protein 1, and miRNA profiles), clinical outcome measures (ALSFRS-R, ALSAQ-5, ROADS, King's Staging, SVC, muscle strength, electrophysiology markers of denervation [maximum compound muscle action potential/CMAP and repetitive nerve stimulation/RNS]), and cerebrospinal fluid (CSF) PK profile.

The ANQUR clinical trial is expected to include 64 study participants with ALS across sites in Canada, the European Union (EU), and United Kingdom (UK). Sites participating in the ANQUR clinical trial: Canada – University of Alberta (Edmonton, Alberta) University of Calgary (Calgary, Alberta), Montréal Neurological Institute-Hospital (Montreal, Quebec), and CHUM-Hopital Notre-Dame (Montréal, Quebec); EU – Universitaire Ziekenhuizen Leuven (Leuven, Belgium), Charité Research Organisation (Berlin, Germany), University Hospital Schleswig-Holstein Campus Lübeck (Lübeck, Germany), Universitätsklinikum Ulm (Ulm, Germany), Universitair Medisch Centrum Utrecht (Utrecht, The Netherlands); and UK – St. James Hospital (Dublin, Ireland), Kings College Hospital NHS Foundation Trust (London, England), and The University of Sheffield, Royal Hallamshire Hospital (Sheffield, England).

The ANQUR clinical trial successfully completed the dose-escalation phase, based on PK data analysis from Cohorts 1 and 2, indicating CSF exposure levels of QRL-201 met or exceeded the targeted therapeutic range. The dose range-finding phase will evaluate two doses of QRL-201 and will enroll an additional 48 participants: 32 participants with sporadic ALS and 16 participants with C9orf72-related ALS.

Visit www.clinicaltrials.gov for more information about the ANQUR study.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a robust precision medicine pipeline with therapeutic candidates aimed at modifying severe disease pathology in defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on X @QurAlisCo or LinkedIn.

Media contact:

Kathy Vincent

kathy@kathyvincent.com

310-403-8951

• Aliada's lead compound, ALIA-1758, an anti-pyroglutamate amyloid beta (3pE-Aβ) antibody, is a potential best-in-class therapy for Alzheimer's disease
• Acquisition also allows AbbVie to utilize Aliada's novel blood-brain barrier (BBB)-crossing technology to enhance discovery and development efforts across neuroscience

NORTH CHICAGO, Ill. and BOSTON, Oct. 28, 2024 /PRNewswire/ -- AbbVie (NYSE: ABBV) and Aliada Therapeutics today announced a definitive agreement under which AbbVie will acquire Aliada, a biotechnology company advancing therapies using a novel blood-brain barrier (BBB)-crossing technology to address challenging central nervous system (CNS) diseases. Aliada's lead investigational asset utilizing this delivery technology, ALIA-1758, is an anti-pyroglutamate amyloid beta (3pE-Aβ) antibody in development for the treatment of Alzheimer's disease.

"Neuroscience is one of our key growth areas and we are committed to driving innovation in this field to address critical unmet needs for patients living with seriously debilitating neurological diseases such as Alzheimer's disease," said Roopal Thakkar, M.D., executive vice president, research and development and chief scientific officer, AbbVie. "This acquisition immediately positions us to advance ALIA-1758, a potentially best-in-class disease-modifying therapy for Alzheimer's disease. In addition, Aliada's novel BBB-crossing technology strengthens our R&D capabilities to accelerate the development of next-generation therapies for neurological disorders and other diseases where enhanced delivery of therapeutics into the CNS is beneficial."

"We are pleased to announce the acquisition of Aliada by AbbVie and are excited about AbbVie's commitment to bringing ALIA-1758 to patients with Alzheimer's disease. Our proprietary MODEL™ platform has enabled the development of ALIA-1758, a promising step forward in brain delivery of an anti-amyloid antibody therapy," said Michael Ryan, M.D., chief medical officer at Aliada Therapeutics. "Many promising CNS-targeted therapies fail to reach late-stage trials due to their inability to cross the blood-brain barrier. Our MODEL™ platform addresses this challenge directly, efficiently delivering targeted drugs and potentially transforming how we treat neurological diseases." 

Aliada is advancing therapeutic candidates using its Modular Delivery (MODEL™) platform, engineered for high-precision CNS drug delivery. The novel BBB-crossing technology targets transferrin and CD98 receptors (TfR and CD98) which are highly expressed in brain endothelial cells. By engineering highly optimized TfR or CD98 binders, this platform is designed to deliver different types of biological cargoes into the brain, including therapeutic antibodies and genetic medicines such as siRNA.

ALIA-1758 utilizes TfR to transport a 3pE-Aβ antibody across the BBB to enable degradation and elimination of amyloid beta plaques, a pathological hallmark of Alzheimer's disease. This investigational candidate is currently in a Phase 1 clinical trial to assess its safety and tolerability in healthy participants (NCT06406348).

Under the terms of the agreement, AbbVie will acquire all outstanding Aliada equity for $1.4 billion in cash, subject to certain customary adjustments. This transaction is expected to close in 4Q2024, subject to regulatory approvals and other customary closing conditions. 

Advisors

AbbVie's legal advisor was Covington & Burling LLP. Aliada's exclusive financial advisor was Centerview Partners LLC and Fenwick & West LLP served as legal advisor.

About AbbVie

AbbVie's mission is to discover and deliver innovative medicines and solutions that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas – immunology, oncology, neuroscience, and eye care – and products and services in our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on LinkedIn, Facebook, Instagram, X (formerly Twitter), and YouTube.

About Aliada Therapeutics

Aliada Therapeutics is a biotechnology company focused on addressing delivery challenges in CNS drug development. Aliada is developing next-generation CNS therapeutics with its novel BBB-crossing Modular Delivery (MODEL™) platform technology, which has been shown to efficiently transport diverse therapeutic cargoes into the brain, enhancing effectiveness and addressing the critical need for efficient and versatile large molecule and oligonucleotide delivery. Johnson & Johnson (through its venture capital arm, Johnson & Johnson Innovation – JJDC, Inc.), RA Capital Management, and Raven (RA Capital Management's incubator) co-founded Aliada and co-led the series seed financing in 2021 to advance the MODEL™ platform created by Johnson & Johnson scientists that was licensed to Aliada at its inception. Further investment was made by OrbiMed and Sanofi Ventures. 

For more information visit www.aliadatx.com and follow us on LinkedIn and X. 

Forward-Looking Statements 

Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2023 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law. 

• Dr. Gaspar brings invaluable expertise to gene-editing pioneering company Eligo as it prepares for clinical trials with world’s first topical CRISPR treatment
• Eligo also strengthens its investor network with new strategic investors, including VIVES Partners and CRISPR pioneer Rodolphe Barrangou

Paris, France, October 1, 2024 – Eligo Bioscience, a biotech company expanding the scope of gene-editing targets by focusing on the delivery of genetic medicines to the microbiome, today announces the appointment of Dr. Bobby Gaspar, CEO of Orchard Therapeutics, as chair of its board of directors. This announcement follows the publication in the prestigious Nature journal, highlighting its novel approach to using gene editing to target the microbiome.

“I am thrilled to welcome Bobby as chair of Eligo. His exceptional ability to translate breakthrough innovations from concept to commercialization at Orchard Therapeutics is impressive. As a co-founder and CEO of a pioneering genetic medicines company, his expertise will be invaluable as Eligo advances toward clinical trials and expands its therapeutic pipeline,” said Xavier Duportet, CEO and co-founder of Eligo.

With three decades of experience spearheading the development of cutting-edge gene therapies, Dr. Bobby Gaspar is a recognized pioneer in genetic medicine. As the co-founder and CEO of Orchard Therapeutics, his leadership guided the company from early development stages through regulatory approvals and commercialization in Europe and the US, as well as a notable $477M acquisition in 2023. An honorary professor of pediatrics and immunology at University College London, Dr. Gaspar was named on the inaugural TIME100 Health List, recognizing him as one of the world’s most influential individuals in health. His extensive expertise in both private and public fundraising, along with a proven track record of taking therapies from lab to market, will greatly benefit Eligo as it enters this critical next phase.

“In my early career as a pediatrician caring for children with devastating genetic diseases, I developed a passion and commitment for transforming the way medicine is practiced through the delivery of paradigm-shifting therapies,” said Dr. Gaspar. “At Eligo, there is an unprecedented opportunity to utilize gene editing technology to make specific and targeted genetic changes to patients’ microbiome and address a wide range of severe diseases. What the company has achieved since its inception is impressive, and I look forward to partnering with Xavier and the broader team to help translate this powerful platform into new scientific breakthroughs and innovative treatments.”

Eligo Expands Investor Syndicate with Key Strategic Additions

Eligo is pleased to welcome new investors, VIVES Partners, which is supported by the European Union under the InvestEU Fund, and Prof. Rodolphe Barrangou, a CRISPR pioneer and co-founder of Intellia Therapeutics. The continued interest from these strategic investors, following the company’s successful $30M fundraising round last year, underscores the strong potential of Eligo’s first-in-class gene-editing platform.

“This is a very exciting time for translational pursuits of CRISPR-based effectors into the clinic and I am delighted to support Eligo’s journey as it gets to the clinical stage of in vivo editing of the microbiome and disrupts the field with a radically game-changing and innovative approach,” said Rodolphe Barrangou.

Leveraging its proprietary platform, Eligo is developing first-in-class modalities that deliver CRISPR and therapeutic genetic circuits directly to the microbiome, enabling precise editing of both its genetic composition and function. Eligo is progressing toward a key milestone: advancing its lead program into clinical trials for moderate to severe acne with the world’s first topical CRISPR treatment. 

Moderate to severe acne affects approximately 120 million patients globally, with a quality-of-life impact comparable to conditions like asthma, diabetes and epilepsy. Despite the significant unmet need, innovation in acne treatment has been almost stagnant for the past three decades. Eligo’s novel mechanism of action, targeting microbiome dysbiosis through the CRISPR-based precise elimination of pro-inflammatory C. acnes strains within hair follicles, offers a groundbreaking and targeted solution. This represents a significant opportunity for safe, effective treatment in a $10 billion market ripe for disruption.

“In addressing the pressing medical need for effective solutions to severe acne, we recognize that current treatments fall short, often accompanied by significant adverse effects and limited efficacy. This large unmet medical need profoundly impacts a young population, struggling with not only physical skin defects but also psychological challenges. That’s why we are committed to investing in Eligo and collaborating with a strong consortium of investors, as we believe its pioneering approach holds the potential to truly tackle this critical issue,” said Philippe Durieux, CEO of VIVES Partners.

About Eligo Bioscience

Eligo Bioscience is the world leader in microbiome in vivo gene editing and is advancing a highly differentiated pipeline of precision medicines to address unmet medical needs in immunoinflammation, oncology and infectious diseases driven by the expression of deleterious bacterial genes.

Eligo was founded by Luciano Marraffini (Professor at The Rockefeller University and co-founder of Intellia Therapeutics), Timothy Lu (Professor at MIT, and CEO at Senti Biosciences), Dr. David Bikard (Professor at Institut Pasteur) and Xavier Duportet (MIT TR35, Young Global Leader, and Termeer Fellow).

Eligo was named a Technology Pioneer by the World Economic Forum and received venture capital funding from Sanofi Ventures, Khosla Ventures, Bpifrance, Seventure Partners and VIVES Partners.

www.eligo.bio

Media and analyst contact

Andrew Lloyd & Associates

Juliette Schmitt & Saffiyah Khalique

juliette@ala.associates - saffiyah@ala.associates

UK/US: +44 1273 952 481

Oxford, United Kingdom – 30 September 2024 – OMass Therapeutics (‘OMass’ or ‘the Company’), a biotechnology company identifying medicines against highly validated target ecosystems such as membrane proteins or intracellular complexes, today announces the candidate nomination of its lead program targeting the melanocortin-2 (MC2) receptor and key appointments to support its progress towards becoming a clinical-stage company.

Over the last year, OMass has made significant progress in advancing its pipeline and has selected its first clinical candidate targeting the MC2 receptor, a GPCR for the adrenocorticotropic hormone (ACTH). The focus of the program has been to increase the receptor residency time to make OMass’ antagonists resistant to competition by the endogenous ligand. This can allow all patients suffering from conditions related to ACTH excess, including congenital adrenal hyperplasia and Cushing’s syndrome, to be treated. The long residence time is expected to avoid loss of efficacy in the face of rising ACTH levels due to reductions in glucocorticoid supplementation for CAH or progression of Cushing’s Syndrome.

To support the advancement of OMass’ pipeline, OMass has expanded its development team. Based in the USA, Dr Steve Griffen joins as Vice President of Clinical Development and Angela Hecyk joins as Director of Clinical Operations. Based in the UK, Stuart Hadley has been appointed as Senior Director of Chemistry Manufacture and Controls (CMC). The new appointments add critical expertise in clinical development and manufacturing:

• Steve is an endocrinologist with more than 25 years’ experience in basic and clinical research and drug development. He has previously served as Vice President, Clinical Development at MBX Biosciences following his role as the Medical Lead for Integrated Care for Sanofi developing devices and software to assist people managing their diabetes. Prior to that, he served as Senior Vice President, Research for the non-profit organization JDRF and Type 1 Diabetes Full Development Team leader for dapagliflozin at Bristol-Myers Squibb, after having served as Exploratory Development Team leader for metabolic assets taking multiple assets into first-in-human studies. Steve holds an AB in Physiology and an MA in Endocrinology from the University of California, Berkeley and completed his medical doctorate at the Medical College of Wisconsin.

• Angela is a global clinical operations professional with over 16 years of experience in clinical research across all stages of clinical development to post-approval, including significant contributions to programs in rare pediatric diseases and four FDA-approved therapies. She joins OMass after holding positions at several growing small to mid-sized biotech and pharmaceutical companies, including MBX Biosciences, Taysha Gene Therapies, Ayala Pharma, Phathom Pharmaceuticals, and Astellas Pharma. Prior to this, she oversaw clinical trials in islet cell transplantation at Northwestern University and was a study monitor for ICON. Angela holds a BS in Natural Sciences, Biology from the University of Wisconsin-Madison. 

• Stuart has over 25 years' experience in drug development and operations working across all disciplines of CMC and supply chain management. He joins OMass from F2G Ltd, where he was Senior Director CMC, leading the CMC program for Olorofim to launch readiness. Prior to that he held multiple CMC and supply chain roles over a 20-year period with AstraZeneca, starting his career there as a graduate analytical chemist, having completed his BSc (Hons) in Chemistry with Pharmaceutical and Forensic Science at the University of Bradford.

Ros Deegan, Chief Executive Officer at OMass Therapeutics, commented: “Nomination of our first clinical candidate represents a key milestone for the company. I am delighted to welcome Steve, Angela and Stuart to the OMass team. They bring extensive experience, and I have no doubt that they will prove to be invaluable additions as we progress our MC2 program to the clinic.”

Steve Griffen, Vice President of Clinical Development at OMass Therapeutics, added: “I’m very excited by the opportunity to join OMass at such a pivotal time for the company. I look forward to working closely with Ros, Stuart, Angela and the rest of the OMass team in advancing our pipeline of small molecule drug candidates.”

-ENDS-

For further information, please contact:

About OMass Therapeutics

OMass Therapeutics is a biotechnology company discovering medicines against highly-validated target ecosystems, such as membrane proteins or intracellular complexes.

OdyssION™, OMass’ unique drug discovery platform, comprises next-generation native mass spectrometry with novel biochemistry techniques and custom chemistry to interrogate not just a drug target, but also the interaction of the target with its native ecosystem, separate from the confounding complexity of the cell. This unique approach results in cell-system fidelity with cell-free precision.

OMass is advancing a pipeline of small molecule therapeutics in rare diseases and immunological conditions. Its lead programme is a best-in-class MC2 (melanocortin-2) receptor antagonist for the treatment of Congenital Adrenal Hyperplasia (CAH) and Cushing’s Syndrome. The focus of the program has been to increase receptor residency time to make OMass’ antagonists resistant to competition by the endogenous ligand, thereby avoiding loss of efficacy in the face of rising adrenocorticotropic hormone (ACTH) levels due to reductions in glucocorticoid supplementation for CAH or progression of Cushing’s Syndrome.

Headquartered in Oxford, UK, OMass has raised over $160M (£129M) from a top-tier international investor syndicate including Syncona, Oxford Science Enterprises, GV, Northpond Ventures, Sanofi Ventures and British Patient Capital.

To learn more, please visit www.omass.com. Follow us on LinkedIn and X.

• QRL-101 is the only Kv7.2/7.3 ion channel opener being actively studied for the treatment of hyperexcitability-induced disease progression in ALS 
• Kv7.2/7.3 is a clinically validated target to regulate the hyperexcitable state in epilepsy 
• Company initiates exploratory Phase 1 proof-of-mechanism study in healthy volunteers to characterize the potential anti-seizure effects of QRL-101 

CAMBRIDGE, Mass., September 19, 2024 – QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases, today announced that the company is expanding its Kv7 development program to include epilepsy. The Company’s lead investigational candidate, QRL-101 is the only Kv7.2/7.3 ion channel opener being actively studied for the treatment of hyperexcitability-induced disease progression in ALS, which is present in approximately 50 percent of ALS patients. Kv7 is a clinically validated target to regulate the hyperexcitable state in epilepsy. 

QurAlis also announced the initiation of an exploratory Phase 1 proof-of-mechanism electroencephalogram biomarker study in healthy volunteers of QRL-101 to characterize the potential anti-seizure effects of QRL-101. 

“Epilepsy, one of the most common and most disabling neurological seizure disorders, is characterized by spontaneous recurrent seizures, which disrupt normal brain functions, lead to neuronal loss, and result in cognitive and emotional deficits. In about one-third of people living with epilepsy, the seizures are resistant to current treatments; so more effective treatments are urgently needed,” said Kasper Roet, Ph.D., chief executive officer and co-founder of QurAlis. “QRL-101, is a highly selective Kv7.2/7.3 ion channel opener, which in preclinical models shows a strong potential to control motor neuron hyperexcitability-induced neurodegeneration with an attractive side effect profile. Since Kv7 is a clinically validated target in controlling hyperexcitability in epilepsy, we are excited to expand our scope of QRL-101 into a new therapeutic area and explore the potential of QRL-101 in epilepsy so that QurAlis can continue the goal of making a real difference in patients’ lives.” 

About Kv7 

Kv7.2/7.3 is a voltage-gated potassium channel whose role is crucial for the regulation of neuronal excitability and membrane potential. The activation of this channel shows the potential to decrease spinal and cortical motor neuron excitability and to positively affect several electrophysiological biomarkers. This suggests that this may be an effective therapeutic approach in several neurodegenerative and neurological diseases including ALS and epilepsy. 

About Epilepsy 

Epilepsy is one of the most common chronic neurological diseases and, according to the Centers for Disease Control, affects more than 65 million people around the world of which 3.4 million are in the U.S. Epilepsy is characterized by unpredictable, recurrent seizures, which are brief episodes of involuntary movement that may involve a part of the body (partial) or the entire body (generalized). Seizure episodes 

are a result of excessive electrical discharges in a group of brain cells. According to the World Health Organization, recurrent seizures disrupt normal brain functions, lead to neuronal loss, and result in cognitive and emotional deficits. Patients suffer from stigmatization, social isolation, combined with disability, educational underachievement, and poor employment outcomes. The Epilepsy Foundation estimates that one-third of people with epilepsy live with uncontrollable seizures because no available treatments are effective. 

About QurAlis Corporation 

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a robust precision medicine pipeline with therapeutic candidates aimed at modifying severe disease pathology in defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on X @QurAlisCo or LinkedIn. 

Contact: Kathy Vincent kathy.vincent@quralis.com 310-403-8951 

• Proceeds will support Nura Bio’s pipeline of neuroprotective medicines
• Company announces successful completion of Phase 1 study of NB-4746, Nura Bio’sbrain-penetrant SARM1 inhibitor
• Shilpa Sambashivan, Ph.D., appointed CEO and a Director of the company

South San Francisco, Calif., September 17, 2024--(BUSINESS WIRE)- Nura Bio Inc. (Nura Bio), a clinical- stage, biopharmaceutical company developing neuroprotective, small molecule therapies for the treatment of debilitating neurological diseases, announced today the closing of more than $140 million in Series A financing. This includes the addition of $68 million to the initial Series A round of $73 million which was announced in 2020. The round was led by founding investor The Column Group, with participation from continuing investors Samsara Bio Capital and Euclidean Capital, and new investor Sanofi Ventures.

The company also announced the appointment of Shilpa Sambashivan, Ph.D., as Chief Executive Officer (CEO) and a company Director. As a member of the founding team at Nura Bio and Chief Scientific Officer (CSO), Dr. Sambashivan has been the driving force behind Nura Bio’s bespoke research engine and differentiated R&D pipeline, with the company’s first clinical candidate, NB-4746, recently completing Phase 1 studies in healthy volunteers.

“Under Shilpa’s leadership, Nura Bio has successfully transitioned to a clinical-stage organization, making remarkable progress in identifying ways to translate complex biology into potential therapies,” said Tim Kutzkey, Ph.D., Managing Partner, The Column Group and Nura Bio’s founding chairman. “We are excited to continue to support the company through this next phase of growth and clinical development. Shilpa’s leadership, combined with her deep scientific expertise, will be key to maximizing Nura Bio’s broad therapeutic potential in areas of large unmet need.”

Nura Bio’s financing close comes at a pivotal point with the Phase 1 success of NB-4746, a brain- penetrant SARM1 inhibitor that has been shown to prevent axon degeneration and provide neuroprotection in multiple preclinical models of nerve injury and disease. Nura Bio plans to initiate a Phase 1b/2 trial in a patient population in 2025.

“At Nura Bio, we have been laser-focused on our mission of delivering novel neuroprotective therapies to patients by leveraging our deep scientific understanding of underlying disease mechanisms including axon degeneration and neuroinflammation. The strong support demonstrated by our investors through this financing reflects the tremendous potential of our R&D pipeline,” said Dr. Sambashivan. “I am proud of the results our team has delivered. I look forward to leading the company through this next phase as we prepare to test the SARM1 hypothesis in a patient population in 2025 with our lead candidate NB-4746 while continuing to advance our promising preclinical pipeline.”

Results from the recently completed Phase 1 study of NB-4746 in healthy volunteers show it was well-tolerated in the single ascending and multiple ascending dose arms of the study. In this Phase 1 study, NB-4746 achieved targeted plasma exposure levels that the company believes are required for efficacy with no associated serious treatment-emergent adverse events. Cerebrospinal fluid levels of NB-4746 confirm brain penetration and support this molecule's advancement in diseases that impact both the peripheral and central nervous systems.

About NB-4746

NB-4746 is the lead asset in Nura Bio’s small molecule pipeline. NB-4746 targets SARM1, a neuronally enriched nicotinamide adenine dinucleotide (NAD) hydrolase that has emerged as an important axon- intrinsic metabolic sensor and central driver of axon degeneration. Axon degeneration is an early hallmark of several neurological diseases. Halting axon degeneration early can confer significant structural and functional neuroprotection and has tremendous potential in the treatment of several neurological diseases. Preclinical studies support the potential of NB-4746 to provide broad axonal protection and functional improvement across diseases of the central, peripheral, and ocular nervous systems.

About Nura Bio

Nura Bio, Inc. (Nura Bio) is a clinical-stage biopharmaceutical company developing neuroprotective therapies for the treatment of a broad range of neurological diseases. Nura Bio’s research and early development small molecule pipeline is focused on developing therapies that halt axon degeneration and/or modulate microglial responses to degeneration and injury, with the goal of conferring neuroprotection, across diseases of the central, peripheral, and ocular nervous systems.

• MinervaX scaling up supply of novel GBS vaccine, ahead of phase III studies
• Wacker Biotech to manufacture active vaccine protein ingredients and prepare for commercial supply after regulatory approval
• Vaccine to address the unmet medical burden of GBS, both by maternal vaccination to prevent adverse pregnancy outcomes and life-threatening infections in infants and by vaccination of older/at risk adults

Copenhagen/Munich, September 17, 2024 – MinervaX ApS, a privately held Danish biotechnology company developing a novel, prophylactic vaccine against Group B Streptococcus (GBS) and Wacker Biotech, a contract development and manufacturing organization (CDMO), wholly owned subsidiary of Wacker Chemie AG, today announce a collaboration to manufacture MinervaX’s active protein ingredients of its GBS vaccine.

GBS is responsible for nearly 50 percent of all life-threatening infections in newborns. At any given time, some 15-25 percent of the population, including pregnant people are spontaneously colonized with GBS, and during pregnancy they run the risk of transmitting the bacteria to their child in the womb, during birth and/or during the first months of life. GBS colonization may lead to late abortions, premature delivery, or stillbirth and in the newborn child, may result in sepsis, pneumonia or meningitis, all of which carry a significant risk of severe morbidity, long- term disability or death. With no general implemented and fully protective preventative treatment available for GBS, this underlines the unmet medical need of developing and providing a vaccine to prevent adverse pregnancy outcomes and life- threatening infections in infants caused by GBS. Furthermore, older adults and adults with certain co-morbidities such as diabetes or obesity are also at an increased risk of severe GBS infections and form a second population which would benefit from a prophylactic vaccine against this potentially fatal disease.

MinervaX’s lead vaccine candidate, is a novel protein-only vaccine, based on fusions of highly immunogenic and proactive protein domains from selected surface proteins of GBS. The company is strongly committed and dedicated to advancing its novel vaccine and has successfully completed two Phase II clinical trials of its maternal vaccine against GBS and is in preparation to commence Phase III clinical trials in this indication. Data from MinervaX’s GBS vaccine is very positive, demonstrating an acceptable safety profile in pregnant people and their infants, with high immunogenicity, leading to functionally active antibodies, with the potential for broad coverage and protection, alleviating the need for excessive use of antibiotics.

Wacker Biotech will manufacture the active ingredients of MinervaX’s novel vaccine candidate, alongside performing the technology transfer, process validation and process characterization activities for later commercial manufacturing. Subsequently, Wacker Biotech will perform all key functions critical to ensure stable commercial supply at its site in Amsterdam, The Netherlands.

Per Fischer, CEO of MinervaX, said: “GBS can be life-threatening for newborn babies and is linked to over half a million preterm births annually. Following the EUR 54 million financing last year, our team is advancing the development of our novel prophylactic vaccine against GBS for the benefit of all populations at risk, worldwide. Wacker Biotech is a robust manufacturing partner with a strong track record in late clinical and commercial supply and we look forward to collaborating with the team ahead of commencing Phase III studies.”

Ronald Eulenberger, Managing Director of Wacker Biotech B.V. in Amsterdam, stated: “With our strong background in E. coli processes, process characterization, and process validation experiences, we at Wacker Biotech are perfectly suited to support MinervaX with its ongoing program for the prevention of invasive GBS disease.”

Details  of MinervaX’s  completed Phase  II  clinical  trials  in pregnant  people  can  be  found at https://clinicaltrials.gov/ under the identifiers NCT04596878 and NCT05154578. In addition to pregnant people, MinervaX is also pursuing Phase I development of its novel GBS vaccine in older adults, under identifier NCT05782179.

ENDS

For further information please contact:

MinervaX

Per Fischer | Chief Executive Officer
Email: info@minervax.com

Optimum Strategic Communications

Mary Clark / Zoe Bolt / Vareen Outhonesack
Email: minervax@optimumcomms.com
Tel: +44 (0) 203 882 9621

Wacker Chemie AG

Dr. Karsten Werth
Email: karsten.werth@wacker.com
Tel: +49 89 (0) 6279-1573

Notes to Editors:

About MinervaX

MinervaX is a Danish biotechnology company, established in 2010 to develop a prophylactic vaccine against Group B Streptococcus (GBS), based on research from Lund University. MinervaX is developing a GBS vaccine for maternal immunization, and also for vaccination of older/at risk adults. Phase II clinical data from its maternal vaccination program suggest a high efficacy, based on the preliminary Correlate of Protection data from a natural history study. MinervaX’s GBS vaccine is a protein-only vaccine based on fusions of highly immunogenic and protective protein domains from selected surface proteins of GBS, the Alpha-like protein family (AIpN). Given the broad distribution of proteins contained in the vaccine on GBS strains globally, it is expected that MinervaX’s GBS vaccine will confer protection against virtually 100% of all GBS isolates. www.minervax.com

About Wacker Biotech

Wacker Biotech is a full-service contract manufacturer of therapeutic proteins, live biopharmaceutical products (LBPs), plasmid DNA (pDNA), messenger ribonucleic acid (mRNA) and vaccines based on microbial systems. Wacker Biotech’s portfolio extends from strain/process development and analytical testing through to production for clinical and commercial applications. Wacker Biotech operates three GMP-compliant, FDA- and EMA-certified production plants at its Jena and Halle sites in Germany and in Amsterdam in the Netherlands. In addition, Wacker Biotech has had a plant in San Diego (Wacker Biotech US Inc.) since February 2021. Wacker Biotech GmbH, Wacker Biotech B.V. and Wacker Biotech US Inc. are wholly owned subsidiaries of Munich-based Wacker Chemie AG.

MinervaX ApS, Nordre Fasanvej 215, DK-2000 Frederiksberg, Denmark VAT no. DK32673287

• QRL-101 aims to reduce hyperexcitability-induced neurodegeneration, which is present in approximately 50 percent of all ALS patients
• Completed Phase 1 single-ascending dose (SAD) clinical trial of QRL-101 enrolled 88 participants; no reported significant safety concerns or serious adverse events
• MAD data expected to be reported in first half of 2025; results will support larger global studies in people living with ALS

CAMBRIDGE, Mass., September 10, 2024 – QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases, today announced that the company recently completed dosing of the first participant cohort in the Phase 1 multiple-ascending dose (MAD) clinical trial evaluating QRL-101 (QRL-101-03; NCT06532396). QRL-101 is a first-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS. Kv7.2 is a mis-spliced protein in sporadic ALS patients.

QRL-101-03 is a randomized, double-blind, placebo-controlled, single-site Phase 1 clinical trial designed to evaluate the safety, tolerability, and pharmacokinetics of multiple-ascending doses of QRL-101 in adult healthy volunteers. The study is expected to enroll approximately 60 participants, who will be randomized in a 9:3 ratio of QRL-101 to placebo into five planned cohorts. The dose range of QRL-101 for this MAD study was determined by results from QurAlis’ Phase 1 single-ascending dose (SAD) clinical trial (QRL-101- 01; NCT05667779). Of the 88 healthy participants in the SAD clinical trial, no significant safety concerns or serious adverse events have been reported for QRL-101.

“We are excited to complete dosing of our first participant cohort in our Phase 1 MAD clinical trial of QRL-101. In the SAD study, QRL-101 was shown to be well tolerated, with no significant safety concerns or serious adverse events,” said Doug Williamson, M.D., chief medical officer of QurAlis. “ALS is a devastating, fatal neurodegenerative disease and there are currently no therapies that can significantly extend patients’ lives. QRL-101 has the potential to be a first-in-class effective therapy for ALS patients suffering from hyperexcitability-induced motor neuron degeneration. We look forward to advancing the clinical program for QRL-101 so QurAlis can bring much-needed therapies to people living with ALS.”

“Motor system hyperexcitability occurs in approximately 50 percent of all ALS patients and is linked to potassium channel dysfunction,” said Leonard H. van den Berg, M.D., Ph.D., professor of neurology and chair, TRICALS. “QRL-101, is a highly selective Kv7.2/7.3 ion channel opener, which in preclinical models shows a strong potential to control motor neuron hyperexcitability-induced neurodegeneration with an attractive side effect profile. We are encouraged by the findings from the SAD study of QRL-101 and look forward to results from the MAD study.”

QurAlis anticipates reporting topline data from the Phase 1 MAD clinical trial of QRL-101 in the first half of 2025.

More information about the QRL-101 Phase 1 clinical trials can be found at www.clinicaltrials.gov.

About Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic  lateral  sclerosis  (ALS),  also  known  as  Lou  Gehrig’s  disease,  is  a  progressive neurodegenerative disease impacting nerve cells in the brain and spinal cord, reducing muscle function and control. ALS can be traced to mutations in more than 25 different genes and is often caused by a combination of multiple sub-forms of the condition. Cases usually cannot be predicted, although a small percentage are inherited. ALS has a devastating impact on patients and families. ALS patients’ average life expectancy is three years; after diagnosis patients only have two years to live. There is currently no cure for the disease.

About Kv7

Kv7.2/7.3 is a voltage-gated potassium channel whose role is crucial for the regulation of neuronal excitability and membrane potential. Kv7.2 is mis-spliced in sporadic ALS leading to loss of function and abnormal electrical activity in the spinal cord and brain. The activation of this channel shows the potential to decrease spinal and cortical/motor neuron excitability and to positively affect CMAP (compound muscle action potential). This suggests that this may be an effective therapeutic approach for ALS patients suffering from hyperexcitability-induced motor neuron degeneration.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a robust precision medicine pipeline with therapeutic candidates aimed at modifying severe disease pathology in defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on X @QurAlisCo or LinkedIn.

Contact:

Kathy Vincent
kathy.vincent@quralis.com
310-403-8951

• CD3 bispecific antibody developed to redirect T cell-mediated immunity toward B7-H7 expressing tumors, expanding monotherapy treatment possibility for a new patient population
• Company’s precision immunotherapy approach enables prospective identification of patients most likely to benefit from NPX372 with a defined clinical biomarker

Cambridge, MA –September 5, 2024 – NextPoint Therapeutics, a clinical-stage biotechnology company developing a new class of precision immuno-oncology and tumor-directed therapeutics targeting the novel B7-H7 axis, today has unveiled NPX372, a novel T cell engager. NPX372 further expands NextPoint’s multi-modal focus on the emerging B7-H7 axis in cancer therapy.

B7-H7, also known as HHLA2, is an emerging immunomodulatory receptor upregulated in various solid tumor types, including colorectal carcinoma, non-small cell lung cancer, renal cell carcinoma, prostate cancer, and many others. Notably, this receptor is induced independently of PD-L1 or other B7 family members. Unlike other B7 family proteins that are expressed across a wide range of cell types, B7-H7 is primarily found on the epithelial cells of tumors, making it a unique and potentially specific target for tumor-directed therapies.

“T cell engagers have shown immense potential, but to date their application in solid tumors has remained a formidable challenge. NPX372 represents a significant advancement, aiming to redirect T cell-mediated immunity with high specificity by modulating key biological components, potentially offering more effective monotherapy treatment options for a range of solid tumors,” said Tatiana Novobrantseva, PhD, Chief Scientific Officer of NextPoint Therapeutics.

NPX372 is a CD3 bispecific antibody with unique capabilities to redirect T cell-mediated cytotoxicity toward B7-H7-positive tumors. In addition to CD3 engagement, this antibody interacts with the B7-H7 immune axis to achieve added potency. Preclinical data highlight NPX372’s potent anti-tumor responses and a favorable safety profile at clinically relevant doses with no indication of cytokine release syndrome. This asset is part of NextPoint's diverse portfolio of immunotherapies designed to target various tumor types. NextPoint is rapidly advancing the Investigational New Drug (IND) application for NPX372.

“NPX372 represents a significant advancement in our pursuit of precision immunotherapy," said Ivan Cheung, CEO of NextPoint Therapeutics. “As part of our ongoing immune checkpoint clinical programs, NPX267 and NPX887, we have developed a clinical biomarker for B7-H7 expression, which allows us to selectively target patients across various tumor types who may benefit from a potent T cell engager such as NPX372. This precision medicine approach allows us to potentially address solid tumors expressing B7-H7, tailoring treatments to those who will respond best. Our deep knowledge of B7-H7 biology drives our leadership in advancing innovative, transformative treatments that can make a meaningful difference in the lives of cancer patients.”

About NextPoint Therapeutics

NextPoint is launching a new world of precision immuno-oncology and tumor-targeting therapeutics through its leading scientific work on the novel B7-H7/HHLA2 axis. Our innovative approach integrates foundational science with a defined clinical biomarker to identify the right patient population for each B7-H7-directed therapy, so that we can deliver a new class of monotherapies for patients. Our team of proven drug developers is simultaneously advancing therapeutic approaches blocking the B7-H7 immune signaling pathway and utilizing the unique upregulation of B7-H7 in cancer as an anchor for tumor-targeting treatment modalities. To learn more, visit nextpointtx.com.

Contacts

Media Contact
Lauren Arnold
LA Communications
Lauren@lacommunications.net

NodThera Named a ‘Fierce 15’ Company by Fierce Biotech Philadelphia, PA, August 5, 2024 - NodThera, a leading clinical-stage biotech delivering a paradigm shift in the treatment of chronic inflammatory diseases through selective modulation of the NLRP3 inflammasome, today announces that it has been named by Fierce Biotech as one of 2024’s ‘Fierce 15’ biotechnology companies, designating it as one of the most innovative and promising biotechnology companies in the industry.

Daniel Swisher, Chief Executive Officer of NodThera, commented: “Over the past year, NodThera has made significant progress towards realizing the full potential of targeting the NLRP3 inflammasome, supported by a proven leadership team, best-in-class molecules and compelling pre-clinical and clinical data. We are incredibly honored to be profiled by Fierce Biotech among the industry’s most exciting and innovative companies. With preparations underway for our Phase II clinical development program in cardiovascular disease, obesity, and neuro-inflammation among other value inflection points, this achievement underscores the strength of our science and commitment to leading a paradigm shift in the treatment of chronic inflammatory diseases.”

Ayla Ellison, Editor-in-Chief, Fierce Life Sciences and Healthcare, said: “For the past 22 years, we have evaluated hundreds of companies for inclusion in the ‘Fierce 15’ special report. Our selection process considers various factors, including technological robustness, strategic partnerships, venture support and market positioning. This report highlights innovation and creativity amid intense competition.”

This year’s full list of winners can be viewed here: https://www.fiercebiotech.com/special- reports/introducing-fierce-biotechs-2024-fierce-15

For more information about NodThera please contact:

NodThera
Tel: +44 (0) 1223 608130
Email: info@nodthera.com

ICR Consilium
Amber Fennell, David Daley, Sukaina Virji
Tel: +44 (0)20 3709 5700
Email: nodthera@consilium-comms.com

About NodThera

NodThera is a leading clinical-stage biotech developing brain-penetrant NLRP3 inflammasome inhibitors to treat chronic inflammatory diseases. Led by an experienced management team, NodThera is combining a deep understanding of NLRP3 inhibition, pharmaceutical neuroscience expertise and precision chemistry. Its two lead clinical candidates are oral, small molecule NLRP3 inflammasome inhibitors, which have demonstrated differentiated, potentially best-in-class clinical profiles with significant anti-inflammatory effects and high brain penetration, offering distinct opportunities to treat multiple indications. The Company is backed by top-tier investors including 5AM Ventures, Blue Owl Capital, Epidarex Capital, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera is headquartered in Philadelphia, Pennsylvania, with additional operations in Cambridge, UK and Seattle, WA. Learn more at www.nodthera.com or follow the Company on LinkedIn.

About Fierce Biotech

Fierce Biotech is the biotech industry’s daily monitor, providing the latest news, articles, and resources related to clinical trials, drug discovery, FDA approval, FDA regulation, patent news, pharma news, biotech company news and more. More than 300,000 top biotech professionals rely on Fierce Biotech for an insider briefing on the day’s top stories.

Greg brings over 25 years of public and private company leadership experience

Philadelphia, PA, August 1, 2024 - NodThera, a leading clinical-stage biotech delivering a paradigm shift in the treatment of chronic inflammatory diseases through selective modulation of the NLRP3 inflammasome, today announces the appointment of Greg Chow as Chief Financial and Business Officer (CFBO), effective immediately.

Greg is an experienced executive with over 25 years of corporate finance, capital markets, investment banking, financial accounting, drug development operations, and business development experience. Over the course of his career, he has led major financing rounds for both public and private companies, as well as supported operations and administrative functions.

Greg joins NodThera from Freenome Holdings, where he served as Chief Financial Officer (CFO) and helped successfully execute the company’s recent $254 million Series F financing round. Prior to Freenome, Greg was CFO at Frontier Medicines, where he led its Series B and C fundraising rounds and headed up Alliance Management for its global, multi-year collaboration with Abbvie.  His leadership across Frontier’s business operations was instrumental in helping the company transition from a discovery-stage to clinical-stage organization. Greg gained significant public company experience as Executive Vice President and CFO at Aptose Biosciences (Nasdaq: APTO), overseeing the company’s dual listing on the Nasdaq and Toronto stock exchanges in addition to successfully raising over $225 million and attracting multiple new shareholders.

Prior to his time as an industry executive, Greg spent 14 years in investment banking as Managing Director and Director of Private Placements at Wedbush Securities and in senior roles at RBC Capital Markets and Wells Fargo Securities. Greg holds an MBA in Finance from the Wharton School at the University of Pennsylvania and a BA in Business Economics from the University of California, Santa Barbara. He is a Certified Public Accountant (inactive) in the state of California.

Daniel Swisher, Chief Executive Officer of NodThera, said: “Greg’s financial experience and extensive track record of long-term value creation for both private and public biotechs will be invaluable as NodThera continues to execute its strategy and evolves into a mature, high-value, clinical-stage company. I look forward to working closely with him as part of our executive team. Greg will be a key strategic partner as we continue to unlock the broad potential and maximum value of our highly differentiated brain-penetrant NLRP3 inflammasome inhibitors in the treatment of chronic inflammatory diseases. On behalf of the whole team, I would like to wish him a very warm welcome to NodThera.”

Greg Chow, Chief Financial Officer of NodThera, added: “I am incredibly excited to join NodThera at such a pivotal time in its development. The potential to modulate inflammation for the treatment of chronic diseases presents a significant opportunity to impact patients’ lives in a meaningful way. I look forward to being immersed in one of the most exciting and rapidly evolving therapeutic categories in biotech today. With a highly accomplished team, positive progression of lead candidate NT-0796 and upcoming Phase II studies in obesity, cardiovascular and Parkinson’s disease, NodThera is well positioned in its current trajectory with some important inflection points on the near-term horizon. I

am eager to bring my capital markets, operations, and business development experience to NodThera and look forward to partnering with Dan, the Board of Directors, and the wider team.”

For more information about NodThera please contact:

NodThera
Tel: +44 (0) 1223 608130
Email: info@nodthera.com

ICR Consilium
Amber Fennell, David Daley, Sukaina Virji
Tel: +44 (0)20 3709 5700
Email: nodthera@consilium-comms.com

About NodThera

NodThera is a leading clinical-stage biotech developing brain-penetrant NLRP3 inflammasome inhibitors to treat chronic inflammatory diseases. Led by an experienced management team, NodThera is combining a deep understanding of NLRP3 inhibition, pharmaceutical neuroscience expertise and precision molecular chemistry. Its two lead clinical candidates are oral, small molecule NLRP3 inflammasome inhibitors, which have demonstrated differentiated, potentially best-in-class clinical profiles with significant anti-inflammatory effects and the ability to penetrate different areas of the brain, offering distinct opportunities to treat multiple indications. The Company is backed by top-tier investors including 5AM Ventures, Blue Owl Capital, Epidarex Capital, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera is headquartered in Philadelphia, Pennsylvania, with additional operations in Cambridge, UK and Seattle, WA. Learn more at www.nodthera.com or follow the Company on LinkedIn.

Paris, July 10, 2024 — In a groundbreaking development, Eligo Bioscience has made in vivo bacterial genome editing a reality. In work published in the journal Nature, Eligo demonstrates, for the first time, the ability to precisely and efficiently edit the genome of bacteria directly in the gut. Achieving gene editing with nearly 100% efficiency and without disturbing the surrounding microbial communities is a major milestone in microbial gene therapy, paving the way for the development of new treatments for microbiome-associated diseases.

A novel chapter for gene editing: from human genes to bacterial genes

Since the advent of gene therapies in the 1970s, culminating in the recent approval of the first CRISPR-based drugs, researchers have made significant strides in delivering DNA to various cell types and organs to correct genetic defects. Until now, however, a large proportion of the genes carried by humans remained completely out of reach: the microbiome.

The microbiome, composed of billions of commensal bacteria, is essential to our health and the proper functioning of our immune system. However, we are discovering a growing number of bacterial genes implicated in chronic and serious diseases: from bacterial peptides that can trigger autoimmune diseases to bacterial virulence factors that contribute to inflammatory diseases, tumor formation, neurodegenerative diseases. As it becomes clearer that conventional therapeutics affecting microbiome composition can cause serious adverse events and side effects, it is critical to develop approaches to edit the microbiome in a targeted way.

"What Eligo Bioscience has achieved shows that it’s now possible to make specific changes to the DNA of bacteria in the gut, similar to how scientists have been editing human genes to investigate or treat genetic disorders," said David Bikard, co-founder of Eligo Bioscience and researcher at the Institut Pasteur in Paris.

A precise and efficient method

Achieving efficient in vivo gene editing of bacteria requires both an effective delivery method and a robust editing system.

In a ‘tour de force,’ Eligo demonstrated the ability to engineer, produce, and purify a bacteriophage-derived capsid containing a synthetic DNA payload encoding a base-editor system. After oral administration to mice, the capsid delivered the payload with extreme efficiency and precision to target bacterial populations residing among the hundreds of bacterial species in the mouse gut. Remarkably, the system could precisely inactivate antibiotic resistance genes or virulence factors by creating single-base pair mutations in the corresponding genes. When targeting E. coli strains colonizing the mouse gut, the technology modified the target gene in over 90% of the bacteria, reaching up to 99.7% in some cases.

These modifications remained stable for at least 42 days.

Jesus Fernandez, Eligo VP of Technology and a senior author of the study, highlights how "this achievement is the culmination of eight years of work by the team at Eligo Bioscience and represents a paradigm shift in microbiome research. This leap forward provides Eligo with a unique edge to develop microbial genetic medicines, and also to find novel therapeutic targets with a novel tool to interrogate the role and function of bacterial genes in health and disease."

Looking Ahead

Eligo Bioscience is already using this novel and patented approach to create new treatments for various conditions that affect millions of people worldwide. "This technology enhances Eligo's pioneering arsenal of in-vivo editing tools, and can be applied to various bacteria and genes, opening the door to treatments for a wide range of health issues", said Xavier Duportet, CEO and co-founder of Eligo. ‘It therefore broadens the landscape of addressable therapeutic targets in the gene editing field’.

Read the paper in Nature here.

–

About Eligo Bioscience

Eligo Bioscience is the world leader in microbiome in-vivo gene editing and is advancing a highly differentiated pipeline of precision medicines to address unmet medical needs driven by the expression of deleterious bacterial genes in immuno-inflammation, oncology, and infectious diseases.

Eligo’s first drug candidates advancing to the clinic are based on the delivery of CRISPR-Cas systems to kill bacteria based on their genetic signature. One of these programs targets

pro-inflammatory genes in skin bacteria driving moderate-to-severe acne pathology in dozens of millions of patients around the world. This lead program is expected to enter clinical trials in patients in 2025.

Eligo was named a Technology Pioneer by the World Economic Forum and has raised over €50M from top-tier investors including Sanofi Ventures, Khosla Ventures, BPIFrance, and Seventure Partners.

Eligo was founded by Luciano Marraffini (Professor at The Rockefeller University and cofounder of Intellia Therapeutics, Timothy Lu (Professor at MIT, and CEO at Senti Biosciences), Dr. David Bikard (Professor at Institut Pasteur) and Dr. Xavier Duportet (MIT TR35, Young Global Leader, and Termeer Fellow).

For more information about Eligo Bioscience and our latest research, please visit Eligo’s website: https://eligo.bio/

Contact:
xavier.duportet@eligo-bioscience.com

• Company initiates partnership and collaboration with UMass Chan Medical School to characterize relevant targets that enable ASO-mediated correction for FXS
  the leading inherited form of intellectual disability and a known cause of autism
• Fragile X syndrome is a rare, genetic neurological disease that is

CAMBRIDGE, Mass., June 25, 2024 – QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases, today announced it is expanding its industry-leading precision medicine antisense oligonucleotide (ASO) splicing expertise beyond neurodegeneration into Fragile X syndrome (FXS).

As part of this strategy, QurAlis has initiated a partnership and collaboration with UMass Chan Medical School to explore the biology of FXS to determine and confirm relevant targets that could enable ASO- mediated correction for FXS. QurAlis can leverage its deep understanding, knowledge and expertise in developing ASOs including its proprietary FlexASO™ Splice Modulator Platform as part of the collaboration.

“QurAlis’ deep knowledge and expertise of ASO splicing targets combined with our FlexASO™ Splice Modulator Platform gives us the latitude to go beyond neurodegeneration to explore the potential of bringing much-needed precision medicines for other serious neurological diseases like FXS,” said Kasper Roet, Ph.D., chief executive officer and co-founder of QurAlis. “Our partnership and collaboration with UMass Chan in FXS underscores the enormous value of partnerships between academia and industry to drive innovation forward to help fulfill unmet medical needs for patients. We are excited to expand our scope into a new therapeutic area and to make use of our expertise in splicing targets so that QurAlis can continue the goal of making a real difference in patients’ lives.”

“We are excited to be partnering with QurAlis in the next step of this years-long research,” said Joel Richter, Ph.D., the Arthur F. Koskinas Chair in Neuroscience and professor of molecular medicine at UMass Chan Medical School in Worcester, MA. “QurAlis’ translational knowledge and clinical trial experience in ASO splicing for neurodegenerative disorders is an important component to bridging the gap between biomedical discoveries made in the laboratory and delivering therapies to patients in the clinic. With their help, we are hopeful that one day we may be able to offer patients with FXS meaningful treatment options.”

About Fragile X Syndrome (FXS)

Fragile X syndrome is the leading inherited form of intellectual disability and a known cause of autism. It is a genetic condition caused by a mutation of a single gene – FMR1 – on the X chromosome. This mutation of FMR1 causes a range of developmental problems including learning disabilities, behavioral challenges, and cognitive impairment.

An orphan disease, FXS affects approximately 87,000 individuals in the U.S. alone – one in 4,000 men and one in 6,000 women. Though FXS occurs in both genders, males are more frequently affected than females, and generally with greater severity. In addition to intellectual disability, FXS patients endure a wide range of disabling symptoms including severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression, developmental seizures, and others. There are no disease- modifying therapies currently available for FXS.

About QurAlis’ FlexASO^TM Splice Modulator Platform

Antisense oligonucleotides (ASOs) are short, engineered single-stranded DNA/RNA molecules that can selectively bind RNA to regulate its expression in the cell. ASO technology has been leading in the field of protein regulation and has since allowed us to develop treatments for neurodegenerative disease by changing the expression of genes connected to the disease.

QurAlis’ FlexASO^TM Splice Modulator Platform was developed to generate splice-switching ASOs with improved potency, increased therapeutic index and improved bio-distribution. This bespoke platform has the potential to tackle the spectrum of neurodegenerative and neurological diseases.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that have the potential to alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative and neurological diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a robust precision medicine pipeline with therapeutic candidates aimed at modifying severe disease pathology in defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on X @QurAlisCo or LinkedIn.

Contact:

Kathy Vincent
kathy.vincent@quralis.com
310-403-8951

Industry veteran, founder of Medivation and serial entrepreneur joins T-Therapeutics to support development of the company’s pipeline of TCR-based immuno-oncology drugs

12 June 2024; Cambridge, England – T-Therapeutics, a biotechnology company developing next- generation soluble TCR therapeutics targeting cancer and autoimmune indications, today announces the appointment of biopharma industry veteran Dr David Hung as Chairman of its Board of Directors.

Dr Hung is a hugely successful serial entrepreneur in the oncology space. He founded Medivation in 2003 which was sold to Pfizer for $14.3 billion in 2016. At Medivation, Dr Hung identified, in-licensed and led bench-to-bedside development of enzalutamide (XTANDI®) for advanced prostate cancer, taking it from first in vitro laboratory experiment to FDA approval in seven years, one of the fastest development timelines in pharmaceutical history. XTANDI, approved in more than 60 countries is one of the top cancer drugs by revenue, reaching blockbuster drug status and exceeding $6 billion in global annual sales in 2023.

Prior to founding Medivation, Dr Hung served as President and CEO of ProDuct Health, a venture- backed startup medical device company founded in 1998 that developed, manufactured and commercialized a breast microcatheter – invented by Dr Hung – for breast cancer risk assessment. ProDuct Health was acquired in 2001 for $168 million by Cytyc Corporation.

Dr Hung is currently President and CEO of Nuvation Bio, which he founded in 2018, a late-stage biopharma developing differentiated and novel therapeutic candidates to tackle some of the greatest unmet needs in oncology.

Dr Hung received an A.B. summa cum laude in biology from Harvard College and an M.D. Alpha Omega Alpha from the University of California, San Francisco, School of Medicine. He completed simultaneous clinical fellowships in hematology, oncology and transfusion medicine as well as two basic science research fellowships in molecular biology at the University of California, San Francisco, School of Medicine.

Professor Allan Bradley, CEO of T-Therapeutics, commented: “Having followed David’s career for many years, I’m delighted that he is joining T-Therapeutics’ Board as Chair. David brings a wealth of scientific, clinical and company building experience to our Board. I look forward to drawing on his deep understanding of drug development in the oncology space, as well as his substantial experience in building biotechnology companies as we work to develop a pipeline of drugs designed to reshape the clinical landscape for cancer patients.”

Dr David Hung, Chairman of T-Therapeutics, also commented: “I’m excited to work with Allan and his very experienced team to help realise their vision. T-Therapeutics has an unusually strong and unique scientific foundation that is being leveraged to build a pipeline of transformative medicines. The team have an excellent track record of successful development of drugs that leverage immune cell biology. I believe their unique TCR platform has the potential to deliver a pipeline of transformative medicines.”

In November 2023 T-Therapeutics announced a £48m series A fundraise for the development of next-generation TCR therapeutics to transform cancer treatment, led by Sofinnova Partners, F-Prime Capital, Digitalis Ventures and Cambridge Innovation Capital with participation from Sanofi Ventures and the University of Cambridge Venture Fund.

– ENDS –

Contact Us

T-Therapeutics
Allan Bradley
info@t-therapeutics.com

ICR Consilium
Amber Fennell, Sukaina Virji
t-therapeutics@consilium-comms.com

About T-Therapeutics

T-Therapeutics is a next-generation T cell receptor (TCR) company spun off from the University of Cambridge. The company was created to harness the power of T cell biology, evolved over millions of years, to create safe and effective treatments for many cancers and autoimmune diseases. T- Therapeutics combines world-leading expertise in mouse genome engineering, deep knowledge and experience in biopharmaceutical drug development, single cell genomics, machine-learning and structural biology, anchored in a culture of creativity and collaboration. T-Therapeutics is developing ‘optimal’ TCR based therapeutics using a proprietary OpTiMus® platform, based on a fully humanized TCR mouse that provides an almost unlimited source of unique, antigen-specific human TCRs. These TCRs are directed at multiple target classes, many of which have never been worked on before. The company is developing a pipeline of first-in-class drugs that are intended to become transformative medicines, reshaping the clinical landscape for patients with cancer or autoimmune diseases.

• Brain-penetrant NLRP3 inflammasome inhibitor demonstrates highly significant and rapid reduction of key inflammatory marker CRP compared to placebo
• Reductions in multiple markers of cardiometabolic risk confirm profound antiinflammatory effect of NT-0796
• Most pronounced weight loss seen among high-risk subgroups
• Planning is underway for Phase II studies in obesity, Parkinson’s disease and other cardiometabolic indications

BOSTON, MA, June 12, 2024 - NodThera, a leading clinical-stage biotech developing brain-penetrant NLRP3 inflammasome inhibitors to treat chronic inflammatory diseases, today announces positive data from its Phase Ib/IIa cardiovascular risk study in inflamed obese subjects, evaluating the effects of its oral, brain-penetrant NLRP3 inflammasome inhibitor NT-0796, on inflammatory, cardiovascular and metabolic risk parameters.

Obese subjects with elevated baseline C-reactive protein (CRP), measured using an hsCRP assay, were recruited. Elevated CRP is a key marker of chronic inflammatory diseases such as coronary artery disease, and one or more cardiovascular risk factors (e.g. metabolic syndrome, prediabetes, diabetes, hyperlipidemia or hypertension). The study was conducted in-clinic for 28 days and energy intake was limited to 2000 kCal per day. At study end, a highly statistically significant and rapid CRP reduction in NT-0796 dosed subjects was observed compared to placebo, meeting the primary endpoint of the study. Furthermore, more than 75% of NT-0796 dosed subjects achieved a CRP reduction at day 28 to ≤2mg/L compared to less than 25% among the placebo group, a value generally regarded as a threshold for reducing cardiovascular risk.

NodThera’s study also demonstrated reductions in additional pro-inflammatory and cardiometabolic biomarkers, secondary endpoints that build on the previously reported diet-induced obesity preclinical study and Phase Ib/IIa Parkinson’s disease clinical study. Key reductions in cardiovascular and metabolic biomarkers were found to be highly translatable and consistent with the importance of targeting both brain and peripheral NLRP3. While all subjects lost weight due to calorie restriction in both the active and placebo groups, the most pronounced placebo-adjusted reductions in body weight were among high-risk subgroups of NT-0796 dosed subjects, and, with the Company’s earlier preclinical data, support the anti-obesity potential of the molecule.

Preparations are underway for a Phase II study in obesity and other Phase II studies including additional cardiometabolic diseases and Parkinson’s disease.

Alan Watt, President & Chief Scientific Officer of NodThera, said: “This is an impressive data set, made all the more remarkable by being achieved within 28 days. In addition to the profound anti- neuroinflammatory effects in Parkinson’s disease patients demonstrated previously, the data generated in this study of NT-0796 show improvements in inflammatory and metabolic biomarkers of CV risk that go beyond that seen with antibody and peptide therapies. With additional reductions in body weight that should be enhanced with longer-duration dosing, NT-0796 is confirmed as a highly promising therapeutic agent for chronic inflammatory indications.”

As before, NT-0796 was generally safe and well tolerated; adverse events (AEs) were mainly mild and transient, and no serious adverse events (SAEs) were observed.

Taken together, the findings demonstrate that NT-0796 successfully delivered anti-inflammatory changes in an obese inflamed population, amelioration of cardiometabolic risk factors and reductions in body weight within 28 days, indicating excellent potential for long-term, oral dosing in obese subjects.

For more information about NodThera please contact:

NodThera
Tel: +44 (0) 1223 608130
Email: info@nodthera.com

ICR Consilium
Amber Fennell, David Daley, Sukaina Virji
Tel: +44 (0)20 3709 5700
Email: nodthera@consilium-comms.com

About NodThera

NodThera is a leading clinical-stage biotech developing brain-penetrant NLRP3 inflammasome inhibitors to treat chronic inflammatory diseases. Led by an experienced management team, NodThera is combining a deep understanding of NLRP3 inhibition, pharmaceutical neuroscience expertise and precision molecular chemistry. Its two lead clinical candidates are oral, small molecule NLRP3 inflammasome inhibitors, which have demonstrated differentiated, potentially best-in-class clinical profiles with significant anti-inflammatory effects and the ability to penetrate different areas of the brain, offering distinct opportunities to treat multiple indications. The Company is backed by top-tier investors including 5AM Ventures, Blue Owl Capital, Epidarex Capital, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera is headquartered in Boston, MA, with additional operations in Cambridge, UK and Seattle, WA. Learn more at www.nodthera.com or follow the Company on LinkedIn.

• Industry veteran Williamson brings nearly three decades’ experience as a leader in neuroscience R&D at organizations including Eli Lilly and Company, Lundbeck, Parexel, and Acadi Pharmaceuticals
• Former Karuna CFO Brown brings more than 15 years of corporate finance, strategy, business development, M&A, and company-building experience in the biopharmaceutical industry
• Highly respected biotech executive Virani brings deep experience in corporate development, life science R&D, and building successful companies

CAMBRIDGE, Mass., June 11, 2024 – QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that will alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, today announced that it has appointed Douglas (Doug) Williamson, M.D., as chief medical officer (CMO) and Jason Brown, MBA, as chief financial officer (CFO). Dr. Williamson succeeds Angela Genge, M.D., who continues in a consulting role for QurAlis. The company also announced the appointment of Shafique Virani, M.D., to its board of directors.

“Doug is an expert drug developer for neurological disorders, talented pharmaceutical executive, and dedicated physician who brings a breadth of successful experience across all phases of clinical development – from the interface of discovery with first-in-human trials, through Phase 2 dose-range finding studies, Phase 3 pivotal trials, and post-market lifecycle optimization,” said Kasper Roet, Ph.D., chief executive officer (CEO) and co-founder of QurAlis.

Dr. Roet continued, “Jason’s experience and track record in raising capital, preparing companies to go public, scaling companies, and executing partnership and M&A deals, will be instrumental to QurAlis’ next phase of growth. Jason’s outstanding profile in finance and operational growth combined with Doug’s extensive global R&D experience, will greatly complement our team as we execute on our strategy to bring promising new precision medicines to patients with ALS, FTD, and other neurodegenerative and serious neurological diseases rapidly.”

“Neurodegenerative diseases like ALS and FTD are areas of huge unmet medical need, and QurAlis is a company that is changing the future of how we treat these diseases by pioneering the development of powerful precision medicines,” said Dr. Williamson. “QurAlis’ cutting-edge science and Kasper’s leadership is a powerful combination. I am thrilled to join such a dedicated team and apply my clinical and drug development experience toward building this innovative biopharma company to bring new, life- changing treatment options to patients with serious diseases and making a real difference in patients’ lives.”

“QurAlis is a mission-driven organization dedicated to bringing transformative therapies to patients with ALS and other serious neurodegenerative diseases,” said Mr. Brown. “This is an exciting time for QurAlis and I am thrilled to join QurAlis and work closely with Kasper, our board, and the broader team to advance our corporate strategy and lead the company’s financial activities as we further scale our organization and rapidly advance clinical development to bring much-needed medicines to patients.”

Dr. Williamson has almost thirty years of leadership experience in all phases of neuroscience clinical drug development and has led several successful investigational new drug (IND) applications, global new drug applications (NDAs), and commercial launches in a broad range of psychiatry and neurology indications. He spent many years in senior leadership positions at large biopharma organizations including Eli Lilly and Company, Parexel International, and Lundbeck, where he led research and development (R&D) in the U.S. More recently, Dr. Williamson has gained experience at smaller biotechnology companies including as CMO at Avadel Pharmaceuticals, and head of R&D at Acadia Pharmaceuticals. He has consulted for numerous companies on neuroscience clinical development strategy. He graduated in medicine from Edinburgh University in Scotland and held a full-time academic appointment at Oxford University prior to joining the industry. Dr. Williamson is a member of the Royal College of Psychiatrists and the National Academy of Medicine’s Neuroscience Forum.

Before joining QurAlis, Mr. Brown was the CFO of Karuna Therapeutics. He joined Karuna in 2018 as vice president (VP) of corporate finance and its first finance employee. Throughout his tenure at Karuna, Mr. Brown drove the growth of both the finance department and broader organization, which included supporting multiple equity financings, business development transactions, and ultimately the acquisition by Bristol-Myers Squibb for $14 billion in 2024. Previously, Mr. Brown worked at PureTech Health as VP of corporate finance where he was responsible for the financing and operational growth of the company. He also spent five years at Novartis in roles of increasing responsibility within the finance department. He holds an MBA from Boston College and BA from Hamilton College.

Dr. Roet continued, “On behalf of the team, I would like to thank Angela Genge for her leadership and many contributions to QurAlis and we are delighted that she continues to be an integral member of our team.”

Biopharma leader Shafique Virani, M.D., joins QurAlis’ board of directors

QurAlis also announced the appointment of Shafique (Shaf) Virani, M.D., to its board of directors.

Dr. Roet said, “I am delighted to welcome Shaf to our board of directors. His success as a biopharma leader has given hope to patients around the world, and his perspectives will help QurAlis build our capabilities, drive growth for our investors, and realize our vision of accelerating the development of life-changing medicines to help patients with serious neurodegenerative and neurological diseases.”

“Having spent most of my career in neuroscience, I am excited about QurAlis’ groundbreaking science and its robust pipeline and platforms,” said Dr. Virani. “QurAlis’ pioneering approach could revolutionize how we treat serious neurodegenerative diseases and offers tremendous hope to patients, particularly those with intractable diseases like ALS for which there remains significant unmet medical need. I am impressed by QurAlis’ leadership and look forward to working with Kasper and the leadership team and my fellow board members to grow the business and shareholder value and pursue QurAlis’ mission to improve patients’ lives.”

Dr. Virani currently serves as chief business officer at Noetik, Inc., an AI-native biotechnology company focused on discovering and developing cancer therapies. Before joining Noetik in March 2024, he served as Recursion’s chief business officer for four years, as well as interim chief medical officer for half that time. At Recursion, Dr. Virani was instrumental in forging transformative partnerships with Bayer and Roche/Genentech, the latter being one of the largest drug discovery partnerships ever consummated in industry. Previously, Dr. Virani was chief executive officer (CEO) of Navire Pharma and CoA Therapeutics, each a subsidiary of BridgeBio Pharma, Inc., where he also served as CEO-in-residence.

Prior to BridgeBio, Dr. Virani assumed a 13-year-long tenure at Genentech/Roche in several senior roles, most recently as vice president, business development, licensing M&A, and global head of neuroscience, rare disease, and ophthalmology partnering. There, he helped build a portfolio of medicines including Evrysdi® (risdiplam) for spinal muscular atrophy, Enspryng® (satralizumab-mwge) for neuromyelitis optica spectrum disorder, and several therapeutics in the mid-to-late-stage clinical pipeline via licensing and acquisitions. Dr. Virani trained as a neurosurgeon in Cambridge, UK and Boston and received his M.D. from the University of Nottingham, UK.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that will alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a pipeline with therapeutic candidates that target specific components of ALS and FTD pathology and defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on X @QurAlisCo or LinkedIn.

Contact:

Kathy Vincent
kathy.vincent@quralis.com
310-403-8951

• QRL-204 is a splice-switching ASO generated through QurAlis’ FlexASO™ Platform; represents Lilly’s first program targeting UNC13A in ALS and FTD
• Parties to also collaborate to leverage QurAlis’ ALS and ASO development expertise to advance QRL-204 and next-generation compounds
• UNC13A is an essential regulator of neurotransmitter release at synapses; mis-splicing is a critical RNA alteration occurring in up to 63 percent of all ALS patients and up to one-third of all FTD cases

CAMBRIDGE, Mass., June 3, 2024 – QurAlis Corporation (“QurAlis”) today announced that it has entered into an exclusive license agreement with Eli Lilly and Company (“Lilly”) in which QurAlis is granting Lilly global rights to develop and commercialize QRL-204, a potentially best-in-class splice-switching antisense oligonucleotide (ASO) designed to restore UNC13A function in amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases.

Under the terms of the agreement, QurAlis granted Lilly an exclusive, worldwide license to develop and commercialize QRL-204 and other UNC13A-targeting compounds in exchange for an upfront payment of $45 million to QurAlis, plus an additional equity investment. QurAlis is also eligible for future milestone payments of up to $577 million and tiered royalties on net sales.

The agreement includes a research and development (R&D) collaboration to identify and develop additional candidates targeting UNC13A, leveraging QurAlis’ proprietary FlexASO™ Splice Modulator Platform. The QurAlis FlexASO Splice Modulator Platform was developed to generate splice-switching ASOs with improved potency and increased therapeutic index. QurAlis’ ASOs correct UNC13A mis-splicing, restore UNC13A protein production and reduce cryptic exons that may contribute to disease progression.

“We are determined at Lilly to uncover the next great idea, the next collaboration, the next advancement in technology that will drive us toward meaningful treatments for ALS and FTD patients. It’s all for one reason – to make life better for even more people,” said Andrew Adams, senior vice president, neurodegeneration research, and director, Lilly Institute for Genetic Medicine. “Genetic precision medicines like QRL-204 that target specific causal components of disease pathology hold great promise for delivering meaningful advances against a range of neurodegenerative diseases like ALS and FTD. We look forward to collaborating with QurAlis to identify and develop additional next-generation candidates targeting UNC13A.”

“On behalf of the entire team at QurAlis, we are extremely pleased to partner with Lilly, an innovation- led company advancing transformative medicines to help make life better for people around the world,” said Kasper Roet, chief executive officer and co-founder of QurAlis. “At QurAlis, we are driven to explore the deepest aspects of human neurology to find the treatments patients urgently need. This partnership enables QRL-204 to rapidly move toward the clinic, while we continue to advance our other lead programs. We look forward to combining our complementary strengths to uncover additional candidates that target UNC13A that have the potential to transform the treatment of neurodegenerative diseases like ALS and FTD and beyond.”

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease characterized by the loss of neurons in the spinal cord, brainstem, and brain. A defining feature of both sporadic and familial disease is the cytoplasmic mis-localization of TAR DNA Binding Protein-43 (TDP-43). TDP-43 pathology is implicated in 90 percent of ALS cases and approximately 50 percent of FTD cases.

UNC13A is an essential regulator of neurotransmitter release at synapses and is one of several pre-mRNAs that becomes mis-spliced due to loss of nuclear TDP-43 in disease. Up to 63 percent of ALS patients and up to one-third of FTD patients carry a single nucleotide polymorphism in the UNC13A gene or show TDP- 43 pathology, which greatly exacerbates UNC13A mis-splicing leading to loss of function of the UNC13A protein.

Preclinical data recently presented at the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders showed QurAlis’ UNC13A splice-switching ASOs modulate UNC13A splicing and restore normal synaptic activities in ALS and FTD. QurAlis’ ASOs prevented cryptic exon inclusion in UNC13A transcripts, increased UNC13A protein levels, and normalized localization of UNC13A protein at the synapse.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that will alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a pipeline with therapeutic candidates that target specific components of ALS and FTD pathology and defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on X @QurAlisCo or LinkedIn.

Contact:

Kathy Vincent
kathy.vincent@quralis.com
310-403-8951

• Dan joins NodThera with over 30 years of pharmaceutical industry leadership experience, including as President and COO of Jazz Pharmaceuticals and CEO of Sunesis Pharmaceuticals
• Interim CEO Alan Watt becomes President and CSO including leadership of R&D to further build out NodThera’s pioneering work in CNS modulation of chronic inflammatory diseases

BOSTON, MA, May 28, 2024 - NodThera, a leading clinical-stage biotech delivering a paradigm shift in the treatment of chronic inflammatory diseases through selective modulation of the NLRP3 inflammasome, today announces the appointment of Daniel Swisher as Chief Executive Officer (CEO), to strengthen the Company’s commercial capabilities, effective immediately. Alan Watt, the interim CEO moves to an expanded role as President and Chief Scientific Officer (CSO).

Dan joins NodThera with significant leadership expertise of over 30 years in the life sciences industry. During his career, he has led the advancement and commercialization of novel therapeutics across geographic regions and in multiple therapeutic areas, including neuroscience, oncology and rare diseases. He brings to NodThera significant private and public company experience driving cross functional, high-performing teams and building relationships with investors, analysts and corporate partners.

Dan joins NodThera from Jazz Pharmaceuticals (NASDAQ: JAZZ), where he recently served as President and Chief Operating Officer. At Jazz, he was instrumental in the geographic and therapeutic expansion of the company’s commercial and R&D pipeline, as well as playing a critical role in many key corporate development transactions. Prior to his time at Jazz, Dan was CEO and President of Sunesis Pharmaceuticals, guiding the evolution of its early-stage chemistry platform into a multi-asset development platform. During his CEO tenure at Sunesis, he oversaw equity capital market fundraises totaling over $400 million and led the Group to an IPO on the Nasdaq stock exchange.

Earlier in his career, Dan served as Senior Vice President of Sales and Marketing at ALZA Corporation, where he was responsible for its flagship pharmaceutical businesses based in the United States. He also played a pivotal role in the company’s growth into a fully integrated specialty pharmaceutical company and eventual $10.5 billion acquisition by Johnson & Johnson.

Alan Watt, who as the Company’s founder has also served as NodThera’s interim CEO, becomes President and CSO. He will be responsible for the growing R&D organization and focused on strengthening NodThera’s world-leading position in NLRP3 inflammasome inhibition research, with a focus on modulation of neuroinflammation for the treatment of obesity, cardiometabolic disease and neurodegeneration.

Don Nicholson, Executive Chair of the Board of Directors of NodThera, said: “Dan is an accomplished, results-driven leader with a proven track record in developing and commercializing novel therapeutics and building strong, successful businesses. We look forward to drawing on his deep understanding of the biopharmaceutical and financial markets, as well as his substantial experience in advancing clinical-stage assets towards commercialization, as we progress our broad pipeline of NLRP3 inflammasome inhibitors to deliver a paradigm shift in the treatment of chronic inflammatory diseases.

“We are extremely grateful for Alan’s strategic and scientific leadership during his tenure as interim CEO. His creativity, determination and integrity have been invaluable to NodThera in shaping the Company’s strategy and we are delighted that NodThera will continue to benefit from his expertise in his new role.”

Daniel Swisher, newly appointed Chief Executive Officer of NodThera, said: “NodThera is leading the NLRP3 inflammasome space with its best-in-class molecules and a wealth of pre-clinical and clinical data that is setting the tone for the field. NodThera has the potential to drive a positive and meaningful difference to the lives of patients suffering with obesity, cardiometabolic and neurodegenerative diseases and I am delighted to be leading the team at such an exciting point in the Company’s development, ahead of the readout from our ongoing cardiovascular risk trial in an inflamed obese population. I look forward to working alongside Alan, the Board and the wider team to achieve NodThera’s full potential.”

Alan Watt, President of Clinical & Preclinical R&D of NodThera, said: “I would like to extend the warmest welcome to Dan, who joins us at an exciting and pivotal moment. Our sector leading research in the field of NLRP3 inflammasome inhibition, our pioneering and demonstrated approach to reversal of neuroinflammation in humans and the strength of the NodThera team, provide an opportunity to bring about profound change in the management of obesity and multiple cardiometabolic and neurodegenerative diseases. I have the utmost confidence that, together, we will deliver on this opportunity.”

For more information about NodThera please contact:

NodThera
Tel: +44 (0) 1223 608130
Email: info@nodthera.com

ICR Consilium
Amber Fennell, David Daley, Sukaina Virji
Tel: +44 (0)20 3709 5700
Email: nodthera@consilium-comms.com

About NodThera

NodThera is a leading clinical-stage biotech developing brain-penetrant NLRP3 inflammasome inhibitors to treat chronic inflammatory diseases. Led by an experienced management team, NodThera is combining a deep understanding of NLRP3 inhibition, pharmaceutical neuroscience expertise and precision molecular chemistry. Its two lead clinical candidates are oral, small molecule NLRP3 inflammasome inhibitors, which have demonstrated differentiated, potentially best-in-class clinical profiles with significant anti-inflammatory effects and the ability to penetrate different areas of the brain, offering distinct opportunities to treat multiple indications. The Company is backed by top-tier investors including 5AM Ventures, Cowen Healthcare Investments, Epidarex Capital, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera is headquartered in Boston, MA, with additional operations in Cambridge, UK and Seattle, WA. Learn more at www.nodthera.com or follow the Company on LinkedIn.

• Sanofi receives exclusive rights to commercialize losmapimod in all territories outside the U.S.; Fulcrum retains full U.S. commercialization rights
• Fulcrum will receive an upfront payment of $80.0 million, and is eligible to receive $975.0 million in potential milestones, plus royalties on ex-U.S. product sales; parties will share future global development costs 50:50
• Conference call and webcast scheduled for 8:00 a.m. ET today to discuss the collaboration and other recent corporate developments, in conjunction with the first quarter 2024 financial results

CAMBRIDGE, Mass., May 13, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.^® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced that it has entered into a collaboration and license agreement with Sanofi (Nasdaq: SNY) for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Under the collaboration and license agreement, Sanofi obtains exclusive commercialization rights for losmapimod outside of the U.S.

The collaboration and license agreement combines Fulcrum’s expertise in FSHD with Sanofi’s global reach and unparalleled commitment to treating patients with rare diseases. Losmapimod is currently being evaluated in a global Phase 3 clinical trial for the treatment of FSHD, a chronic and progressive genetic muscular disorder that is characterized by significant muscle cell death and fat infiltration into muscle tissue. Results from ReDUX4, the Phase 2 clinical trial evaluating losmapimod for the treatment of FSHD, demonstrated a slowing of disease progression and improved muscle health. Fulcrum expects to report topline data from REACH, the global Phase 3 clinical trial, in the fourth quarter of 2024. Following positive data from the Phase 3 trial, Fulcrum and Sanofi plan to submit marketing applications in the U.S., Europe, Japan, and other geographies.

“Sanofi is a proven leader in developing therapeutics for rare neuromuscular diseases and is the ideal partner to maximize the opportunity and reach of losmapimod outside the U.S.,” said Alex C. Sapir, Fulcrum’s president and chief executive officer. “This deal aligns with our core strategy, allowing Fulcrum to remain focused on preparations for commercialization of losmapimod in the U.S., while leveraging Sanofi’s exceptional global commercial capabilities and established infrastructure in key markets around the world. We are excited about the potential to provide the first approved treatment for FSHD patients, and we look forward to working with Sanofi to bring losmapimod to patients globally.”

“This partnership provides an exciting opportunity to expand Sanofi’s rare disease franchise and deliver the first approved FSHD treatment to patients with the strength and reach of our commercial organization,” said Burcu Eryilmaz, Sanofi’s Global Head of Rare Diseases. “Losmapimod has shown meaningful clinical benefits that underscore the disease-modifying potential and opportunity to address the high unmet need for a safe and effective drug that slows disease progression. With a deep commitment to bringing hope and new treatment options to patients, we look forward to working closely with Fulcrum as losmapimod advances through late-stage development.”

Per the terms of the agreement, Fulcrum will receive an upfront payment of $80.0 million and is eligible to receive up to an additional $975.0 million in specified regulatory and sales-based milestones, along with tiered escalating royalties starting in the low-teens on annual net sales of losmapimod outside the U.S. In addition, Fulcrum and Sanofi will equally share future global development costs.

Conference Call and Webcast
Individuals may register for the conference call by clicking the link here. Once registered, participants will receive dial-in details and unique PIN which will allow them to access the call. An audio webcast will be accessible through the Investor Relations section of Fulcrum’s website at www.fulcrumtx.com or by clicking here. Following the live webcast, an archived replay will also be available.

About Losmapimod
Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively in-licensed losmapimod from GSK following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Results reported from the Phase 2 ReDUX4 trial demonstrated a slowing of disease progression and improved function, including positive impacts on upper extremity strength and functional measures supporting losmapimod’s potential to be a transformative therapy for the treatment of FSHD. Although losmapimod had never previously been explored in muscular dystrophies, it had been evaluated in more than 3,600 subjects in clinical trials across multiple other indications, with no safety signals attributed to losmapimod. Losmapimod has been granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of FSHD. Losmapimod is currently being evaluated in a Phase 3 multi-center randomized, double-blind, placebo-controlled, 48-week parallel-group study in people with FSHD (NCT05397470).

About FSHD
FSHD is a serious, rare, progressive and debilitating disease for which there are no approved treatments. It is characterized by fat infiltration of skeletal muscle leading to muscular atrophy involving primarily the face, scapula and shoulders, upper arms, and abdomen. Impact on patients includes profound decreases in the ability to perform activities of daily living, loss of upper limb function, loss of mobility and independence and chronic pain. FSHD is one of the most common forms of muscular dystrophy and has an estimated patient population of 30,000 in the United States alone.

About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s two lead programs in clinical development are losmapimod, a small molecule in development for the treatment of facioscapulohumeral muscular dystrophy (FSHD), and pociredir (formerly known as FTX-6058), a small molecule designed to increase expression of fetal hemoglobin and in development for the treatment of sickle cell disease (SCD). Fulcrum uses proprietary technology to identify drug targets that can modulate gene expression to treat the known root cause of gene mis-expression. For more information, visit www.fulcrumtx.com and follow us on Twitter/X (@FulcrumTx) and LinkedIn.

Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release are forward-looking statements, including express or implied statements regarding Fulcrum’s collaboration and license agreement with Sanofi and receipt of the upfront payment thereunder; its ability to receive the milestone and royalty payments thereunder and achieve benefits therefrom; timing of data from REACH and its ability to support submission of marketing applications for losmapimod; and Fulcrum’s ability to deliver an FDA-approved therapy for FSHD patients; among others. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum’s ability to continue to advance its product candidates in clinical trials; initiating and enrolling clinical trials on the timeline expected or at all; obtaining and maintaining necessary approvals from the FDA and other regulatory authorities; replicating in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod, pociredir and any other product candidates; obtaining, maintaining or protecting intellectual property rights related to its product candidates; managing expenses; managing executive and employee turnover, including integrating a new CMO; and raising the substantial additional capital needed to achieve its business objectives, among others. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Fulcrum’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties, and other important factors, in Fulcrum’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Fulcrum’s views as of the date hereof and should not be relied upon as representing Fulcrum’s views as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will cause Fulcrum’s views to change. However, while Fulcrum may elect to update these forward-looking statements at some point in the future, Fulcrum specifically disclaims any obligation to do so.

Contact:
Chris Calabrese
LifeSci Advisors, LLC
ccalabrese@lifesciadvisors.com
917-680-5608

Inaugural list recognizes Cheung as one of the year’s most influential individuals in global health

Cambridge, MA – May 6, 2024 – NextPoint Therapeutics, a clinical-stage biotechnology company developing a new class of precision immuno-oncology and tumor-directed therapeutics targeting the novel B7-H7/HHLA2 axis, announced today that Chief Executive Officer Ivan Cheung has been named to the 2024 TIME100 Health List.

The inaugural TIME100 Health list recognizes the 100 most influential individuals in health and medicine who have made significant contributions to improving global health outcomes, advancing medical research and driving innovation in healthcare delivery.

“Ivan has demonstrated a relentless commitment to translating scientific breakthroughs into tangible solutions that benefit patients worldwide. The well-deserved recognition by TIME underscores his outstanding contributions to public health and exemplifies his visionary leadership and unwavering dedication to improving the lives of patients,” commented Detlev Biniszkiewicz, PhD, Managing Director at MPM BioImpact and Chairman of NextPoint’s Board of Directors. “Under Ivan’s leadership, NextPoint is advancing a novel class of therapeutics targeting the B7-H7 pathway to improve treatment options for cancer patients that do not respond to PD-1/L1 therapy.”

With over 25 years of experience leading drug development, approvals and launches, Mr. Cheung joined NextPoint as CEO in January 2024 aiming to deliver breakthrough clinical outcomes from a new world of precision immuno-oncology and tumor-targeting therapeutics. He previously served as Eisai’s U.S. CEO, where he guided the launch of the anti-cancer therapy lenvatinib in a range of tumor types. Importantly, Mr. Cheung led the first-ever full U.S. approval for lecanemab, a monoclonal antibody therapeutic to treat the underlying pathophysiology of early-stage Alzheimer’s disease and helped steward the reimbursement access from the Centers for Medicare & Medicaid Services for this class of medicines.

As a member of the TIME100 Health list, Mr. Cheung joins a distinguished group of individuals who are shaping the future of healthcare and driving positive change on a global scale. The full list and related tributes appear in the May 13, 2024 issue of TIME and at time.com/time100health.

About NextPoint Therapeutics

NextPoint is launching a new world of precision immuno-oncology and tumor-targeting therapeutics through its leading scientific work on the novel B7-H7/HHLA2 axis. Our innovative approach integrates foundational science with a defined clinical biomarker to identify the right patient population for each B7-H7-directed therapy, so that we can deliver a new class of monotherapies for patients including those who do not benefit from PD-1/L1 inhibitors. Our team of proven drug developers is simultaneously advancing therapeutic approaches blocking the B7-H7 immune signaling pathway and utilizing the unique upregulation of B7-H7 in cancer as an anchor for tumor-targeting treatment modalities. To learn more, visit nextpointtx.com.

Contacts

Media Contact

Chelsea Rule
MacDougall Advisors
1(781)235-3060

crule@macdougall.bio

WILMINGTON, Del. & SAN DIEGO--(BUSINESS WIRE)--Apr. 23, 2024-- Incyte (Nasdaq:INCY) and Escient Pharmaceuticals, a clinical-stage drug discovery and development company advancing novel small molecule therapeutics for systemic immune and neuro-immune disorders, have entered into a definitive agreement under which Incyte has agreed to acquire Escient, including EP262, a first-in-class, potent, highly selective, once-daily small molecule antagonist of Mas-related G protein-coupled receptor X2 (MRGPRX2) and EP547, a first-in-class oral MRGPRX4 antagonist.

“As a company dedicated to innovation and the discovery of transformative medicines, we are excited to add EP262 and EP547 to our portfolio. This acquisition builds on our strategy to develop differentiated and first-in-class medicines with high potential,” said Hervé Hoppenot, Chief Executive Officer, Incyte. “EP262 and EP547 are complementary additions to our portfolio, providing an opportunity to leverage our expertise, address the needs of patients with inflammatory diseases and additional potential launch opportunities starting in 2029.”

By blocking MRGPRX2 and degranulation of mast cells, EP262 has the potential to effectively treat multiple mast cell-mediated diseases including atopic dermatitis (AD), chronic inducible urticaria (CIndU) and chronic spontaneous urticaria (CSU). Preclinical studies presented at the American Academy of Dermatology annual meeting in March 2023 showed that EP262 improved AD-like skin lesions and markers of type 2 inflammation. Additionally, in a Phase 1 study of 64 healthy volunteers, EP262 was safe and well tolerated at all doses tested, with no serious or severe adverse events, no adverse events leading to discontinuation and no clinically meaningful adverse changes in safety laboratory parameters, vital signs or ECG parameters. Treatment-emergent adverse events for EP262 were mild, with an incidence that was lower than placebo (33.3% vs. 62.5%) and did not increase with dose.

“These drug candidates are the result of the highly innovative research performed by Escient’s employees and scientific collaborators,” said Joshua A. Grass, President and Chief Executive Officer, Escient. “With its experienced development and commercial teams in Inflammation and Autoimmunity and portfolio of commercial and development stage products, Incyte is well positioned to translate this new science into valuable medicines for patients.”

Under the terms of the agreement, Incyte will acquire Escient and its assets for $750 million plus Escient’s net cash remaining at the close of the transaction, subject to customary adjustments. The acquisition is subject to clearance under the Hart-Scott-Rodino Act, among other customary conditions, and will become effective promptly following the satisfaction or waiver of these conditions which is currently anticipated to be by the third quarter of 2024.

Centerview Partners LLC and Goldman Sachs & Co. LLC advised Escient on the transaction and Fenwick & West LLP acted as legal counsel for Escient. Covington & Burling LLP acted as legal counsel for Incyte.

Incyte Conference Call and Webcast

Incyte will hold a conference call and webcast this morning at 8:00 a.m. ET. To access the conference call, please dial 877-407-3042 for domestic callers or +1 201-389-0864 for international callers. When prompted, provide the conference identification number: 13746287. If you are unable to participate, a replay of the conference call will be available for 90 days. The replay dial-in number for the United States is 877-660-6853 and the dial-in number for international callers is +1 201-612-7415. To access the replay, you will need the conference identification number: 13746287.

The conference call will also be webcast live and can be accessed at investor.incyte.com.

About EP262

EP262 is a potent, highly selective once-daily small molecule antagonist of MRGPRX2, a receptor expressed on mast cells that is activated by numerous ligands, including many peptides released from sensory neurons as well as other cell types. In response to MRGPRX2 activation, mast cells release histamine, tryptase, chymase, chemokines and cytokines, which can cause itchy hives, angioedema, type 2 inflammation (through engagement of the adaptive immune system) and chronic pruritus and pain. Preclinical data demonstrate that, by blocking activation of MRGPRX2, EP262 has the potential to effectively treat a broad range of mast cell-mediated conditions, with an initial focus on chronic urticarias and atopic dermatitis.

About EP547

EP547 is a potent, highly selective antagonist that blocks the activation of MRGPRX4 by various bile acids, bilirubin and urobilin. By virtue of this disease-specific mechanism of action, EP547 has the potential to be a highly targeted and efficacious treatment for cholestatic and uremic pruritus.

About Chronic Urticaria

Chronic urticaria, defined as urticaria persisting for more than 6 weeks, manifests with very itchy hives that may vary in size and can significantly impact a patient’s quality of life by interfering with sleep and daily activities. Some patients with chronic urticaria may also develop swelling deeper under the skin or in other tissues (angioedema). There are two main forms of chronic urticaria. In chronic spontaneous urticaria (CSU), hives occur spontaneously, without known triggers. In chronic inducible urticaria (CIndU), hives are induced by specific triggers, such as cold exposure (cold urticaria) or touch (symptomatic dermographism), among others.

About Incyte

A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia.

For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube.

About Escient Pharmaceuticals

Escient Pharmaceuticals is a clinical-stage company focused on developing novel therapeutics to address a broad range of neurosensory-inflammatory disorders. The company’s pipeline includes two first-in-class small molecule antagonists targeting MRGPRX2 for the treatment of various mast cell mediated disorders and MRGPRX4 for cholestatic pruritus. Based in San Diego, California, Escient is led by an experienced management and scientific team and funded by top-tier life science investors.

Incyte Forward-looking Statements

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the opportunities presented by this transaction; whether and when EP262 or EP547 will be approved for use; whether and when Incyte will bring EP262 or EP547 to market; the potential of EP262 or EP547 to treat patients with atopic dermatitis (AD), chronic inducible urticaria (CIndU) and chronic urticaria (CSU) or for any other indication; and the potential for Incyte to broaden its ability to bring new medicines to patients, contain predictions, estimates and other forward-looking statements.

These forward-looking statements are based on the Company's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; the Company’s dependence on its relationships with its collaboration partners; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission, including its annual report filed on Form 10-K for the year ended December 31, 2023. The Company disclaims any intent or obligation to update these forward-looking statements.

Incyte Contacts:
Media
media@incyte.com

Investors
ir@incyte.com

Escient Contact:
ir@escientpharma.com

• The Extension Funds Nucleai to Advance Its First-in-Class Spatial AI Biomarker in Active Clinical Enrollment
• Investment Expands Deployments of AI-Powered Spatial Biomarker Technology, Strengthening Companion Diagnostics for ADCs, Bi-specifics, and Immunotherapies
• M Ventures is the corporate strategic venture arm of Merck KGaA, Darmstadt, Germany

CHICAGO--(BUSINESS WIRE)--Nucleai, a spatial AI biomarker company that deciphers cellular conversations and maps cellular interactions within tissue samples to predict therapeutic outcomes, has secured a $14 million investment led by M Ventures, the corporate venture capital arm of Merck KGaA, Darmstadt, Germany, and supported by existing investors, bringing the total funding to $60 million. The investment enables Nucleai to further deploy its AI algorithms for the prospective enrollment of patients in clinical trials – a first in the field and a significant advancement in personalized solutions tailored to the distinct needs of patients.

The funding exemplifies Nucleai’s growing momentum in the life sciences industry, evidenced by collaborations with over 60 percent of the top 20 biopharma companies, venture backing from renowned investors like Section 32 and Sanofi Ventures, and now with M Ventures. The funding will accelerate the deployment of Nucleai’s spatial analysis technology that transforms a static biopsy slide into a dynamic AI-guided action plan, empowering pathologists with the intelligence to anticipate and navigate complex diseases, like cancer, with unmatched precision.

By integrating AI algorithms for prospective patient enrollment in clinical trials, Nucleai is spearheading a first-in-field approach to personalized medicine. This process is crucial for enhancing the accuracy of treatment targeting and activation within the body, particularly for advanced therapeutics such as antibody-drug conjugates (ADCs) and bi-specifics. Nucleai's platform tailors therapy strategies to the intricate cellular landscape of each patient, optimizing clinical trial participant selection. This personalized approach not only improves the likelihood of successful clinical outcomes but also significantly contributes to the speed and efficiency of bringing new therapies to market.

Leveraging AI and machine learning (ML), Nucleai analyzes pathology images and spatial data at the cellular and tissue levels. The proprietary technology extracts detailed patterns and features from medical images, offering profound insights into the tumor microenvironment, cellular morphology, and spatial relationships between different cell types. These capabilities advance drug development, refine biomarker discovery, and improve the precision of therapeutic targeting, ultimately leading to more effective and personalized treatment options for patients.

Avi Veidman, CEO and Co-Founder of Nucleai, said, “M Ventures' investment boosts our ability to scale and deploy our spatial AI technology for patient enrollment in clinical trials and supports our work in the rapidly emerging areas of immunotherapies, antibody-drug conjugates, and bi-specifics. Our vision is that every next-generation therapeutic is accompanied with an AI-enabled companion diagnostic, ensuring that each patient's treatment pathway is informed and efficacious. This funding positions us to scale spatial AI, not just to intercept but anticipate the complex behavior of diseases.”

Noga Yerushalmi, Investment Director at M Ventures, who is appointed to the board of Nucleai, stated, “Nucleai’s technology stands out by being the first spatial AI tool used by pathologists for clinical trial patient selection that is directly connected to a drug development program, setting them apart from other companies. Nucleai’s biology-driven mindset and their multimodal approach, which combines traditional pathology data with spatial biology data, allows for more accurate predictions of treatment responses, aligning perfectly with our commitment to optimise speed of new therapies to reach patients."

By moving beyond traditional pathology's analog analysis, Nucleai leverages AI to create dynamic, digital maps from biopsy samples, uncovering the complex interactions at play within tissue. This innovation marks a shift in disease diagnosis and treatment, offering an in-depth contextual view that underpins the development of novel therapeutics.

About Nucleai

Nucleai is the leading AI-powered spatial biomarker company, driving innovation at the intersection of technology and healthcare. Leveraging military intelligence-grade geospatial analytical methods, it intercepts, interprets, and analyzes complex cellular conversations and spatially oriented interactions within tissue samples, translating them into actionable insights. Nucleai’s platform empowers pathologists and researchers with an AI-powered data-rich action plan, paving the way for more informed decisions in the development of bi-specifics, antibody-drug conjugates (ADCs), and immunotherapy. Nucleai's investors include Section 32, Sanofi, Vertex Ventures, M Ventures, and Debiopharm Innovation Fund. It is headquartered in Israel and Chicago. For more information, please visit www.nucleai.ai.

About M Ventures

M Ventures is the strategic, corporate venture capital arm of Merck KGaA, Darmstadt, Germany. From its headquarters in the Netherlands and offices in Germany, USA and Israel, M Ventures invests globally in transformational ideas driven by innovative entrepreneurs. Taking an active role in its portfolio companies, M Ventures teams up with management teams and co-investors to translate scientific discoveries into commercial success. M Ventures focuses on identifying and financing novel solutions to some of the most difficult challenges, through company creation and equity investments in fields that will impact the vitality and sustainability of Merck KGaA, Darmstadt, Germany's current and future businesses.

http://www.m-ventures.com/

Contacts

Consort Partners for Nucleai
nucleai@consortpartners.com

Today is a monumental day for Click, and I could not be more thrilled to share this news with you. Rejoyn™, originally developed as CT-152, is now THE FIRST prescription digital therapeutic to receive FDA clearance for the treatment of Major Depressive Disorder (MDD) symptoms. Rejoyn™ is indicated as a prescription digital therapeutic for the treatment of Major Depressive Disorder (MDD) symptoms as an adjunct to clinician-managed outpatient care for adult patients with MDD age 22 years and older who are on antidepressant medication. It is intended to reduce MDD symptoms. As first in its device type, Rejoyn™ was also assigned a new product code by FDA: “SAP - computerized behavioral therapy to treat major depressive disorder."
 
This FDA clearance represents an example of how digital therapeutics are positioned to help define the future of clinical care. The MIRAI study validated Rejoyn’s™ novel treatment approach and was central to the FDA filing. It is one of the largest studies completed to date on a digital therapeutic and one of the very few to evaluate a digital therapeutic’s effectiveness in a blinded comparison to a sham app that was designed to match the treatment in time, attention, and participant expectation of therapeutic effect.
 
The robust randomized controlled trial provided consistent evidence of effectiveness from both clinician and patient perspectives giving confidence in the benefit of Rejoyn™ as an adjunctive option for the treatment of MDD symptoms. The effectiveness was supported by a demonstrated safety profile with no treatment-emergent adverse events assessed as related to Rejoyn™ in the trial. Together the clinical trial data demonstrate that Rejoyn™ offers a novel adjunctive therapeutic option to treat MDD symptoms for adult patients who are experiencing an inadequate response to their antidepressant medication. And importantly, the Rejoyn™ data show that a prescription digital therapeutic can demonstrate a benefit beyond what patients were able to achieve with their existing antidepressant medication.
 
Quite simply, Rejoyn™ has the potential to help shift the course of mental health treatment by being the first to introduce the concept of a prescription digital therapeutic for the adjunctive treatment of MDD symptoms into the clinical mainstream at scale. We believe this product could mark a turning point toward a future in which clinically validated prescription software is a routine part of the standard of care.
 
I’m incredibly proud of the teams at Click, Otsuka and Mount Sinai who worked together to achieve today’s news, which is the culmination of years of effort. Congratulations to all involved! 
 
This news comes at a very exciting time for our industry given the FDA Draft Guidance released last September on Prescription Drug Use-Related Software (PDURS) formally clarified that any added clinically meaningful benefit from the use of software together with a drug can be added directly to the drug label, right in the package insert. Soon, combination products developed using PDURS will unlock drug-like reimbursement via existing access pathways and extend the benefits of digital therapies to any therapeutic area with unmet behavioral, neurological, or cognitive needs where the drug alone may not be enough.
 
The future ahead is an exciting one, that if approached right, will enable a vibrant and evolving ecosystem of digital treatment options for patients. We are passionate about this vision and could not be more excited to make this future a reality. We are grateful for our collaborators, dedicated investors, and many exceedingly talented colleagues who have made today’s news possible. And if you haven’t already, we invite you to please join us – the opportunity to improve patient care with digital therapeutics is just beginning.
 
You can read more about the clinical data for Rejoyn™ in the Clinician Brief Summary; a link is provided below.

https://rejoynhcp.com/Clinician-Brief-Summary.pdf

You can read more about the FDA Clearance in the press release; a link is provided below.

https://www.otsuka-us.com/news/rejoyn-fda-authorized

With warm regards,

David Benshoof Klein
CEO, Click Therapeutics

INDICATION and SAFETY INFORMATION for Rejoyn™
 
INDICATION:
Rejoyn™ is a prescription digital therapeutic for the treatment of Major Depressive Disorder (MDD) symptoms as an adjunct to clinician-managed outpatient care for adult patients with MDD age 22 years and older who are on antidepressant medication. It is intended to reduce MDD symptoms.
 
SAFETY INFORMATION:
Rejoyn™ is not intended to be used as a standalone therapy or a substitute for medication. Patients should continue their current treatment as directed. Rejoyn™ does not monitor the patient’s symptoms or clinical status and cannot send or receive alerts or warnings to the prescriber.
 
Patients should be clearly instructed that if they believe their depression is worsening or if they have feelings or thoughts of harming themselves or others, to contact a healthcare professional, dial 911, or go to the nearest emergency room immediately.

• Rapidly advancing multiple therapeutic programs leveraging Mirador360 TM precision development engine incorporating advances in human genetics and cutting-edge data science
• Company led by several executives of Prometheus Biosciences and founded by chairman and CEO Mark C. McKenna
• Over $400 million in financing led by ARCH Venture Partners with early investments from OrbiMed and Fairmount and participation from other premier life sciences investors

SAN DIEGO, March 20, 2024 -- Mirador Therapeutics, Inc. (Mirador), announced its launch today. Founded by Mark C. McKenna and led by several former executives of Prometheus Biosciences (acquired in 2023 by Merck for $10.8 billion), Mirador aims to revolutionize precision medicine for immune-mediated inflammatory and fibrotic diseases by leveraging its proprietary Mirador360^TM development engine to rapidly advance multiple programs. Mirador has raised more than $400 million in financing led by ARCH Venture Partners, with early investments from OrbiMed and Fairmount. Other premier life sciences investors also participated, including Fidelity Management & Research Company, Point72, Farallon Capital Management, Boxer Capital, TCGX, Invus, Logos Capital, Moore Strategic Ventures, Blue Owl Healthcare Opportunities, Sanofi Ventures, Woodline Partners LP, Venrock Healthcare Capital Partners, RTW Investments and Alexandria Venture Investments.

“At Mirador, we envision a bold new era of precision medicine for immune-mediated inflammatory and fibrotic diseases driven by speed and superior development accuracy,” said Mark C. McKenna, chairman and CEO of Mirador. “The industry has only scratched the surface of utilizing advances in human genetics – coupled with exponential progress in machine learning – to accelerate the development of precision therapies for patients who need them the most. With a proven team, distinguished board of directors, leading healthcare investors and proprietary data-driven approach, we aim to create a leading precision medicine company at scale to provide important new treatment options for patients.”

“The I&I field is in need of better, novel therapeutics as well as new R&D approaches that target enriched patient populations for improved probability of success in the clinic,” said Kristina Burow, Mirador board member and managing director of ARCH Venture Partners. “The Mirador team has an outstanding track record of success in precision immunology, and we are well on our way to building a company that will make a lasting impact on the lives of millions of patients suffering from a broad range of immune-mediated inflammatory and fibrotic diseases.”

The Next Wave of Precision Medicine in Immunology and Inflammation (I&I)

Mirador’s focus is on developing first-in-class or best-in-class precision medicines. To accelerate development, Mirador utilizes Mirador360, its proprietary precision development engine that leverages recent breakthroughs in human genetics and cutting-edge data science. Mirador360 is purpose-built to harmonize millions of patient samples to discover and validate genetic associations to immuno-fibrotic diseases, identify novel therapeutic targets and elucidate target-target interactions as well as optimal target-target pairs for potential combination therapies. Mirador360 also allows Mirador to develop diagnostics and stratify heterogeneous patient populations for precise clinical development.

About Mirador Therapeutics

Mirador is a next-generation precision medicine company focused on immunology and inflammation. The company’s Mirador360 ^TM precision development engine leverages the latest advances in human genetics and cutting-edge data science to rapidly advance new precision medicines for patients living with chronic immune-mediated inflammatory and fibrotic diseases. Launched in 2024, Mirador has raised over $400 million from leading life sciences investors and is based in San Diego, CA. For more information, please visit us at www.miradortx.com and follow us on Linkedin.

Contacts:

Media:

Dan Budwick
1AB
dan@1abmedia.com

Investors:

Steve Klass
1AB
steve@1abmedia.com

• Brain-penetrant NLRP3 inflammasome inhibitor NT-0796 reduced key neuroinflammatory and inflammatory biomarkers in Parkinson’s disease patients to the levels found in healthy elderly controls over 28 days
• Demonstrates potential to change the treatment paradigm and halt disease progression with a disease-modifying approach
• Trial results will be presented at AD/PDTM 2024 on Friday March 8 in Lisbon, Portugal
• Preparations for a Phase IIa/IIb study in Parkinson’s disease are ongoing

BOSTON, MA, March 7, 2024 - NodThera, a leading clinical-stage biotech developing brain-penetrant NLRP3 inflammasome inhibitors to treat chronic inflammatory diseases, today announces positive data from its Phase Ib/IIa study in Parkinson’s disease patients, evaluating the effects of its oral, brain-penetrant NLRP3 inflammasome inhibitor NT-0796, on inflammatory and disease-specific biomarkers in the blood and cerebrospinal fluid (CSF).

NodThera’s study demonstrated mean reductions of key pro-inflammatory biomarkers in CSF (e.g. IL-1β, IL-6, CCL2, CXCL1 and CXCL8) over 28 days compared to baseline to levels approximating those of healthy elderly controls, demonstrating reversal of NLRP3-mediated neuroinflammation. In addition, reductions in neurodegenerative markers were also observed following oral dosing of NT-0796, including NfL and soluble TREM (sTREM2). In subjects with elevated acute phase biomarkers CRP and fibrinogen, levels were reduced significantly, consistent with the peripheral anti-inflammatory effects of NT-0796 seen in elderly healthy volunteers studied in an earlier stage of the trial.

Alan Watt, Chief Executive Officer of NodThera, said: “Our new findings in Parkinson’s disease, a condition with substantial unmet medical needs, are profound and highly encouraging. They bolster our confidence in the ongoing program, with Phase II studies now in advanced planning stages. The correlation between Parkinson’s disease and neuroinflammation is well-documented, with alpha-synuclein fibrils triggering microglial NLRP3 activation, leading to neuroinflammation and subsequent neurodegeneration. This is the inaugural demonstration of an NLRP3 inhibitor’s potential to not only address Parkinson’s disease but also offer a broader impact on neurodegenerative diseases. Given that existing Parkinson’s treatments primarily manage symptoms, our innovative, disease-modifying strategy presents a significant shift, aiming to stop the disease progression. NT-0796’s demonstrated efficacy in reducing neuroinflammation in patients heralds a substantial advancement towards halting this devastating disease.”

NT-0796 was safe and well tolerated; adverse events (AEs) were mainly mild and transient, and no serious adverse events (SAEs) were observed. Pharmacokinetics indicated the drug candidate’s potential for once-daily dosing and levels of the drug candidate in the brain, determined by measuring the concentration in CSF, were found to be maintained over 24 hours.

Taken together, the findings demonstrate that NT-0796 successfully delivered anti-neuroinflammatory and anti-inflammatory changes within 7 days and sustained for 28 days, indicating excellent potential for long-term, oral dosing in Parkinson’s disease patients.

Prof. Thomas Foltynie BSc, MBBS, MRCP, PhD, Professor of Neurology in the Department of Clinical and Movement Neurosciences, UCL Institute of Neurology and Consultant Neurologist at the National Hospital for Neurology and Neurosurgery, Queen Square, London, said: “These results are particularly promising for the future of Parkinson’s disease treatment, providing a compelling approach to the modulation of inflammation in the brain, a key driver in the development of this disease. Such an approach has the potential to change the face of treatment – actually stopping the disease in its tracks, that would be of enormous value to those living with Parkinson’s disease.”

The findings from NodThera’s study will be presented on Friday March 8 at AD/PDTM 2024, the international conference on Alzheimer’s and Parkinson’s diseases and related neurological disorders, taking place in Lisbon, Portugal.

Additionally, following the recently published preclinical data demonstrating NodThera’s NLRP3 inflammasome inhibitors reversed diet-induced obesity (DIO) and inflammation in mice the Company’s pioneering Phase Ib/IIa clinical study of NT-0796 in obese subjects with cardiovascular risk is in progress with results expected by end of 2Q24. This biomarker-rich study is measuring the change in baseline to Day 28 of CRP levels, a key peripheral inflammatory marker and known predictor of risk of developing atherosclerotic cardiovascular (CV) disease as well as the potential for modification of body weight over 28 days.

For more information about NodThera please contact:

NodThera
Tel: +44 (0) 1223 608130
Email: info@nodthera.com

ICR Consilium
Amber Fennell, David Daley, Sukaina Virji
Tel: +44 (0)20 3709 5700
Email: nodthera@consilium-comms.com

About NodThera
NodThera is a leading clinical-stage biotech developing brain-penetrant NLRP3 inflammasome inhibitors to treat chronic inflammatory diseases. Led by an experienced management team, NodThera is combining a deep understanding of NLRP3 inhibition, pharmaceutical neuroscience expertise and precision molecular chemistry. Its two lead clinical candidates are oral, small molecule NLRP3 inflammasome inhibitors, which have demonstrated differentiated, potentially best-in-class clinical profiles with significant anti-inflammatory effects and the ability to penetrate different areas of the brain, offering distinct opportunities to treat multiple indications. The Company is backed by top-tier investors including 5AM Ventures, Blue Owl Capital, Epidarex Capital, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera is headquartered in Boston, MA, with additional operations in Cambridge, UK and Seattle, WA. Learn more at www.nodthera.com or follow the Company on LinkedIn.

UNC13A is an essential regulator of neurotransmitter release at synapses; mis-splicing is a critical RNA alteration occurring in up to 63 percent of all ALS patients and up to one-third of all FTD cases

Preclinical data to be featured in a poster presentation at AD/PD™ 2024

CAMBRIDGE, Mass., March 4, 2024 – QurAlis Corporation, a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that will alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, today announced preclinical data that showed the company’s UNC13A splice-switching antisense oligonucleotides (ASOs) modulate UNC13A splicing and restore normal synaptic activities in ALS and FTD. QurAlis’ ASOs prevented cryptic exon inclusion in UNC13A transcripts, increased UNC13A protein levels, and normalized localization of UNC13A protein at the synapse.

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease characterized by the loss of neurons in the spinal cord, brainstem, and brain. A defining feature of both sporadic and familial disease is the cytoplasmic mis-localization of TAR DNA Binding Protein-43 (TDP-43). TDP- 43 pathology is implicated in 95 percent of ALS cases and 50 percent of FTD cases.

UNC13A is an essential regulator of neurotransmitter release at synapses and is one of a number of pre-mRNAs that becomes mis-spliced due to loss of nuclear TDP-43 in disease. Up to 63 percent of ALS patients and up to one-third of FTD patients carry a single nucleotide polymorphism in the UNC13A gene or show TDP-43 pathology which greatly exacerbates UNC13A mis-splicing leading to loss of function of the UNC13A protein.

“There are currently no cures for ALS or FTD and limited therapeutic options are available for ALS and FTD patients who are in desperate need for effective therapies,” said Dan Elbaum, Ph.D., chief scientific officer of QurAlis. “QurAlis has identified ASOs that modulate UNC13A splicing and restore normal synaptic activities. We believe that correction of UNC13A splicing can provide therapeutic benefit in relevant patient populations.”

These data will be featured in a poster presentation at the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders being held March 5-9, 2024 in Lisbon, Portugal. Details of the presentation are as follows:

Title: UNC13A Targeting Splice Switching ASOs Ameliorate TDP-43 Dependent Mis-splicing Phenotypes in FTD and ALS
Date(s)/Time: Thursday, March 7, 2024, and Friday, March 8, 2024 at 1:50-3:50PM CET
Poster and Abstract Number: P1214 / 2851
Poster Topic: FTD, ALS: TDP43, C9orf72, and TMEM106B 1 Session: D02 Therapeutic Targets, Mechanisms for Treatment Location: Auditorium VIII
Presenter: Marisa Kamelgarn, Ph.D., senior scientist, QurAlis

Splice-switching oligonucleotides targeting UNC13A were screened via quantitative polymerase chain reaction (qPCR) and a small subset demonstrated correction of splicing and rescue of protein. From those rescue experiments, ASOs were used for functional assay testing.

Using in-house models of induced pluripotent stem cell (iPSC) motor and cortical neurons, QurAlis established a phenotype to demonstrate the cellular consequences of UNC13A mis- splicing due to TDP-43 loss of function.

TDP-43 mediated UNC13A loss partially disabled SNARE complex assembly and evoked synaptic vesicle release in human motor neurons. QurAlis’ ASOs restored all or some aspects of SNARE complex assembly and synaptic vesicle fusion and release.

About QurAlis’ FlexASO™ Splice Modulator Platform

Incorporating its proprietary FlexASO™ Splice Modulator Platform, QurAlis’ ASOs correct UNC13A mis-splicing, restore UNC13A protein production, and reduce cryptic exons that may contribute to disease progression. The QurAlis FlexASO Splice Modulator Platform was developed to generate splice-switching ASOs with improved potency and an increased therapeutic index. In addition to UNC13A, QurAlis is currently exploring this ASO technology for multiple other disease targets.

About QurAlis Corporation

At QurAlis, we are neuro pioneers on a quest to cure. We work with a relentless pursuit of knowledge, a precise attention to craft, and an optimistic mindset to discover and develop effective precision medicines that will alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a pipeline with therapeutic candidates that target specific components of ALS and FTD pathology and defined patient populations based on both disease-causing genetic mutation(s) and clinical biomarkers. For more information, please visit www.quralis.com or follow us on Twitter @QurAlisCo.

Media contact:

Kathy Vincent
kathy@kathyv ncent.com
310-403-8951

EpiMonitor is an all-in-one epilepsy monitoring solution that uses the EmbracePlus wearable and a mobile app to detect and alert for seizures, while providing patients and physicians with continuous, accurate health insights

BOSTON--(BUSINESS WIRE)--Empatica, a leader in remote health monitoring powered by AI, is proud to announce the official launch of EpiMonitor, the latest advancement in wearable epilepsy monitoring.

EpiMonitor has been FDA-cleared for use in adult and children populations aged 6 and up, and is the only wearable solution with regulatory approval for seizure detection available to purchase in the US. It is the successor to Embrace2, which enjoyed global acclaim as the leading wearable for seizure detection for years, and the first ever to be available to patients.

EpiMonitor represents a significant leap forward in epilepsy monitoring technologies, thanks to enhanced capabilities including new seizure alert features, up to a week of battery life, and advanced health insights. It utilizes Empatica’s next-generation medical watch, EmbracePlus, which has already been successfully used to monitor the health of thousands of participants in hundreds of clinical trials. Together with a companion smartphone app and smart algorithms, EpiMonitor is a complete system that continuously monitors its wearer’s health data, detecting possible generalized tonic-clonic seizures, alerting caregivers, and providing valuable health information to help users better manage their condition.

"We are thrilled to introduce EpiMonitor to people with epilepsy in the US,” said Empatica’s Chief Scientist and Co-founder, Dr. Rosalind Picard. “Empatica has always provided the only smartwatch seizure monitors validated by the FDA. EpiMonitor takes this to a new level: giving patients up to 7 days of battery life, and new features that give them greater control over seizure alerts. Also, at the touch of a button, patients can now see comprehensive health insights – including seizures, sleep, and activity reports – in a form easy to give their clinician. EpiMonitor is the device we've long dreamed of to open a new era in giving epilepsy patients greater control over their lives.”

Key features of EpiMonitor include:

• FDA-cleared for adults and children ages 6 and up
• Automatic seizure detection through a smart algorithm (98% accuracy; low False Alarm Rate) and alerts to caregivers via a call and SMS with the user’s location
• Up to 7 days of battery life
• Advanced sensor technology for accurate data collection
• Option to send self-triggered alerts to caregivers
• A user-friendly mobile app with a seizure diary
• Sleep and activity tracking through Empatica’s algorithms
• Seizure, sleep and activity reports that can be exported and shared with a physician
• iOS and Android compatibility

The EpiMonitor FDA clearance also represents a significant milestone for Empatica as it demonstrates the successful integration of a new algorithm with the existing technology stack of the Empatica Health Monitoring Platform, highlighting its potential to host future SaMD products.

Seizure forecasting

In December 2023, Empatica also announced plans to conduct a groundbreaking study to develop a seizure forecasting algorithm, based on the world’s largest real-world data set in epilepsy patients. Recruitment for the study is available to all US-based EpiMonitor users.

“Our FORESIGHT clinical study is an incredibly exciting opportunity to advance personalized seizure forecasting,” said Dr. Marisa Cruz, Empatica’s Chief Medical Officer. “This research is a critical step in our ongoing development of innovative and effective seizure management tools that improve clinical outcomes and quality of life for patients with epilepsy.”

By enrolling in the study, users can contribute valuable data to ongoing research efforts, and participate in a large-scale scientific effort to create a better tomorrow for people living with epilepsy. Large datasets are extremely important to train an algorithm to achieve a prediction of when someone will have a seizure, but they are also extremely difficult to gather. With this study, Empatica seeks to overcome this challenge with the participation of its community.

Seizure forecasting can have multiple benefits, including early intervention in the form of tailoring therapies to the start of seizure onset¹, better seizure management and treatment², and useful information that can be used to modify daily activities^2,3.

EpiMonitor is available for purchase in the US from the Empatica website. To learn more visit empatica.com/epimonitor.

Empatica Inc is a pioneer in continuous, unobtrusive remote health monitoring driven by AI. Empatica's platform and technology are used by thousands of institutional partners for research purposes, in studies examining stress, sleep, epilepsy, migraine, depression, addiction, and other conditions. In 2018, Empatica’s Embrace wearable received FDA clearance for seizure monitoring, making it the world's first epilepsy watch to be cleared by the FDA. Its latest flagship medical wearable, EmbracePlus, has been developed with key partners including HHS, USAMRDC, and NASA.

¹ Epilepsy Foundation (https://www.epilepsy.com/research-funding/epilepsy-innovation-institute/seizure-gauge-challenge)
² https://www.nature.com/articles/s41598-021-01449-2
³ https://pubmed.ncbi.nlm.nih.gov/33040327/

Contacts

Marianna Xenophontos
mx@empatica.com

• Data published in Journal of Pharmacology and Experimental Therapeutics
• NodThera’s brain-penetrant NLRP3 inhibitors matched weight loss driven by GLP-1 receptor agonist semaglutide (Wegovy®) or calorie restriction, while also providing enhanced improvements in disease-relevant biomarkers of cardiovascular inflammation and lipid metabolism
• Findings indicate NLRP3 activation in the brain is implicated in driving obesity which can be reversed with brain-penetrant NLRP3 inhibitors
• Additionally, combinations of an NLRP3 inhibitor and a GLP-1 receptor agonist were shown to be additive on weight loss in as-yet unpublished data
• NodThera’s lead candidate NT-0796 is currently in Phase Ib/IIa development in cardiometabolic disease and Parkinson’s disease

BOSTON, MA, February 19, 2024 – NodThera, a leading clinical-stage biotech developing brain-penetrant NLRP3 inhibitors to treat chronic inflammatory diseases, today announces the publication of preclinical data demonstrating its clinical-stage investigational compounds reversed diet-induced obesity (DIO) and inflammation in an animal model of disease.

The data are published in the Journal of Pharmacology and Experimental Therapeutics in a paper titled ‘Reversal of high fat diet-induced obesity, systemic inflammation and astrogliosis by the NLRP3 inflammasome inhibitors NT-0249 and NT-0796’¹.

The NLRP3 inflammasome is a highly validated anti-inflammatory drug target, and these findings demonstrate that NLRP3 plays a key role in controlling obesity and obesity-associated inflammation through the modulation of hypothalamic gliosis. Both NT-0796 and NT-0249, two structurally distinct NLRP3 inhibitors in clinical development by NodThera, have generated a wealth of preclinical and clinical data demonstrating brain-penetration and broad anti-inflammatory effects, with NT-0796 being the first NLRP3 inhibitor to show reduced neuroinflammation in the clinic.

In this latest publication, NodThera’s researchers show for the first time the ability of NT-0796 and NT-0249 to reverse DIO in a murine model, providing comparisons against the effects of the GLP-1 receptor agonist (GLP-1RA) semaglutide (Wegovy®) and calorie restriction. While all three therapeutic approaches led to statistically significant reductions in body fat in DIO mice, only the NLRP3 inhibitors reduced disease-relevant cardiovascular inflammatory biomarkers such as fibrinogen, sVCAM-1, suPAR, and PCSK9, suggesting their potential to further reduce cardiovascular risk in obese populations. NodThera has additionally explored alternative treatment scenarios where NLRP3 inhibitors can be combined with a GLP1-RA or used as a follow-on therapy for patients who do not tolerate GLP-1RA drugs. Yet-to-be-published preclinical findings have demonstrated an additive weight loss effect when combining the brain-penetrant NLRP3 inhibitors with low dose GLP-1RAs, and stable weight maintenance following cessation of GLP-1RA therapy by dosing of a brain-penetrant NLPR3 inhibitor, thereby preventing body mass regain.

Obesity is a global health concern that predisposes individuals to chronic disease such as diabetes and cardiovascular disease at least in part by promoting systemic inflammation.

Alan Watt, Chief Executive Officer of NodThera, said: “These remarkable findings – the first in the field of NLRP3 inflammasome research – suggest that in obese mice consuming a high-fat diet, brain- penetrant NLRP3 inhibition and the resulting anti-inflammatory effect confers not only reversal of obesity but metabolic benefits that extend well beyond this. While GLP-1 receptor agonists have undoubtedly delivered significant achievements in the management of obesity, their adverse event profile is well established, presenting difficulties for some patients. Our brain-penetrant NLRP3 inhibitors deliver robust weight loss and broad cardiometabolic benefits by targeting a novel molecular mechanism with the convenience of oral dosing and an exceptional safety profile. Our ongoing Phase IIa study in obese individuals at cardiovascular risk will further validate these pre- clinical findings.”

References:

1.  Reversal of High Fat Diet-Induced Obesity, Systemic Inflammation, and Astrogliosis by the NLRP3 Inflammasome Inhibitors NT-0249 and NT-0796. Peter Thornton, Valérie Reader, Zsofia Digby, Pamela Smolak, Nicola Lindsay, David Harrison, Nick Clarke and Alan P. Watt. Journal of Pharmacology and Experimental Therapeutics March 2024, 388 (3) 813-826; DOI: https://doi.org/10.1124/jpet.123.002013

For more information about NodThera please contact:

NodThera
Tel: +44 (0) 1223 608130
Email: info@nodthera.com

ICR Consilium
Amber Fennell, David Daley, Sukaina Virji
Tel: +44 (0)20 3709 5700
Email: nodthera@consilium-comms.com

About NodThera

NodThera is a leading clinical-stage biotech developing brain-penetrant NLRP3 inflammasome inhibitors to treat chronic inflammatory diseases. Led by an experienced management team, NodThera is combining a deep understanding of NLRP3 inhibition, pharmaceutical neuroscience expertise and precision molecular chemistry. Its two lead clinical candidates are oral, small molecule NLRP3 inflammasome inhibitors, which have demonstrated differentiated, potentially best-in-class clinical profiles with significant anti-inflammatory effects and the ability to penetrate different areas of the brain, offering distinct opportunities to treat multiple indications. The Company is backed by top-tier investors including 5AM Ventures, Blue Owl Capital, Epidarex Capital, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera is headquartered in Boston, MA, with additional operations in Cambridge, UK and Seattle, WA. Learn more at www.nodthera.com or follow the Company on LinkedIn.

Collaboration combines Intellia’s leading CRISPR-based platform, including its DNA writing technology, with ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) to extend the reach of gene editing to disease-causing targets in the lung

CAMBRIDGE, Mass. and MENLO PARK, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF). CF is a genetic disease caused by mutations in the CFTR gene, leading to the accumulation of thick mucus in the lungs, digestive systems and other organs. CF can result in life-threatening infections, respiratory failure and other serious complications.

The collaboration will leverage Intellia’s proprietary CRISPR-based gene editing platform, including its DNA writing technology, and ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform to precisely correct one or more CF disease-causing gene mutations. As part of the agreement, the companies will focus initial research efforts on therapeutic approaches that address CF for patients who have limited or no treatment options available, with the opportunity to expand the scope of the collaboration in later phases. Intellia will be responsible for the design of the editing strategy and research- grade components for the investigational therapies. ReCode will lead the subsequent preclinical and clinical development. ReCode will also lead worldwide commercialization for certain programs arising from the collaboration. Intellia will be eligible to receive pre-specified development and commercial milestone payments, as well as royalties on potential sales. Intellia may also exercise an option to lead commercialization in the U.S. for certain programs.

“Intellia’s vision to realize the full promise of gene editing includes extending the reach of our industry-leading CRISPR-based platform to targets outside the liver. This collaboration with ReCode is aimed at achieving that goal as we work together to accelerate the development of potentially life-changing therapies for people with cystic fibrosis,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Building on our CRISPR/Cas9 capabilities, we have made important progress advancing our proprietary DNA writing technology to enable a range of precise editing strategies. We are excited to combine our gene editing expertise and platform with ReCode’s novel lung-directed LNP delivery platform.”

"We are excited to partner with Intellia, a clear leader in the gene editing space, with the ultimate goal of bringing life-altering therapies to CF patients,” said ReCode Chief Executive Officer Shehnaaz Suliman, M.D. (MB ChB), M.B.A., M.Phil. “This collaboration provides further validation of ReCode's SORT LNP platform to deliver diverse gene editing modalities to specific cells and tissues. By combining our highly synergistic technologies and capabilities, we are excited about the potential to enable a faster path for next-generation gene editing therapeutics to CF patients.”

About Intellia Therapeutics

Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases.

Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at intelliatx.com and follow us @intelliatx.

About ReCode Therapeutics

ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues and cells implicated in disease, enabling improved efficacy and potency. ReCode’s lead programs include RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene, and RCT2100 for the treatment of the 10-13% of cystic fibrosis patients who have Class I mutations in the CFTR gene and do not respond to currently approved CFTR modulators. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated using the SORT LNP delivery platform. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases, including musculoskeletal, central nervous system, liver and infectious disease indications.

ReCode has been recognized by the San Francisco Business Times and Silicon Valley Business Journal as a Best Place to Work. For more information, visit www.recodetx.com and follow us on LinkedIn.

Intellia Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding Intellia’s beliefs and expectations regarding: its strategy, business plans and focus; its ability to leverage its proprietary CRISPR-based gene editing platform, including its DNA writing technology, and to combine its platform with the proprietary lipid nanoparticle (“LNP”) delivery platform developed by ReCode Therapeutics, Inc. (“ReCode”) to precisely correct one or more cystic fibrosis (“CF”) disease-causing gene mutations; its ability to develop therapeutic approaches that address CF for patients who have limited or no treatment options available, and to expand the scope of the collaboration in later phases; its ability to commercialize in the U.S. the products that may result from its collaboration with ReCode; and the expected strategic benefits of any current or future collaborations, including its collaboration with ReCode.

Any forward-looking statements in this press release are based on management's current expectations and beliefs of future events, and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks related to Intellia’s ability to protect and maintain its intellectual property portfolio; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of Intellia’s licensors to protect and maintain their intellectual property position; uncertainties related to the development of the company’s product candidates, including product candidates to be developed in its collaboration with ReCode, and the authorization, initiation and conduct of studies and other development requirements for such product candidates; the risk that any one or more of Intellia’s or its collaborators’ product candidates (including the product candidates to be developed with ReCode) will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellia’s collaboration with ReCode or its other collaborations will not continue or will not be successful. These and other risks and uncertainties are described in greater detail in Intellia’s other filings with the Securities and Exchange Commission, including the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K and quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in other filings. Any forward-looking statements contained in this press release represent Intellia’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Intellia explicitly disclaims any obligation to update any forward-looking statements, except as required by law.

Intellia Contacts:

Investors:

Ian Karp
Senior Vice President, Investor Relations and Corporate Communications
ian.karp@intelliatx.com

Lina Li
Senior Director, Investor Relations and Corporate Communications
lina.li@intelliatx.com

Media:

Matt Crenson
Ten Bridge Communications
media@intelliatx.com
BCIntellia@tenbridgecommunications.com

ReCode Contacts:

Investors:

Anne Marie
Fields Stern IR
AnneMarie.Fields@sternir.com
IR@recodetx.com

Media:

Erica Jefferson
Senior Vice President, Corporate Affairs
ejefferson@recodetx.com