This Week in Biotech #20
Catch up on the latest biotech breakthroughs and upcoming trends (Oct 16-17).
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on of This Week in Biotech by Biotech Blueprint!
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MARKET UPDATES
🔹 On October 17, Kezar Life Sciences (KZR) announced that its board unanimously rejected an unsolicited acquisition proposal from Concentra Biosciences, which had offered $1.10 per share plus a contingent value right. Kezar’s board deemed the offer significantly undervalued both the company and its lead therapeutic candidate, zetomipzomib. In response, the company adopted a limited-duration stockholder rights plan. On the same day, Kezar revealed the discontinuation of its phase 2b trial of zetomipzomib for lupus nephritis due to multiple serious adverse events, including four fatalities. Moving forward, Kezar will focus on developing zetomipzomib for autoimmune hepatitis (AIH), a condition with substantial unmet medical need. The company also shared positive safety results from its phase 2a trial for AIH. Following these developments, Kezar’s stock dropped by more than 9%.
🔹 On Oct. 17, Tonix Pharmaceuticals (TNXP) announced that it has received its first payment from the U.S. Department of Defense’s Defense Threat Reduction Agency (DTRA) under a contract worth up to $34M over five years. This funding will accelerate the development of Tonix’s broad-spectrum antiviral agents aimed at protecting military personnel from biological threats. CEO Seth Lederman highlighted the importance of this non-dilutive funding for advancing their antiviral research. Tonix’s stock had surged more than 16% following the announcement.
🔹 Following the FDA’s decision to place a clinical hold on Novavax’s COVID-19-Influenza Combination (CIC) and stand-alone influenza vaccine candidates, Novavax’s stock (NVAX) plunged by approximately 30% on Oct. 16. The hold was due to a reported case of motor neuropathy in a single participant from a phase 2 CIC trial. Novavax said it is working closely with the FDA to resolve the issue, emphasizing that previous trials showed no signs of motor neuropathy.
BIOTECH NEWS
🔹 On Oct. 17, Merck announced positive results from its phase 2b/3 trial of clesrovimab (MK-1654), an investigational monoclonal antibody for preventing respiratory syncytial virus (RSV) in infants. A single dose of clesrovimab reduced RSV-associated hospitalizations by over 84% and lower respiratory infection hospitalizations by over 90%, with consistent safety results across both preterm and full-term infants. Clesrovimab could become the first approved immunization providing the same dose for all infants, regardless of weight. Merck aims for regulatory approval by the 2025-26 RSV season to help address the significant burden of RSV on infant health.
🔹 BenevolentAI has appointed its founder, Kenneth Mulvany, as Executive Chairman, replacing Joerg Moeller, who has stepped down as CEO after less than a year in the role. Mulvany, who originally founded the AI-driven drug discovery company in 2013, returns to leadership to capitalize on the growing adoption of AI in the biopharma sector.
🔹 On Oct. 17, Sanofi and Orano Med announced a partnership to accelerate the development of next-generation radioligand therapies for rare cancers. Sanofi will invest €300M for a 16% equity stake in a new entity focused on advancing targeted alpha therapies using lead-212 (212Pb) isotopes, which combine cancer-targeting vectors with powerful, short-range radiation to kill cancer cells while minimizing damage to healthy tissues.
🔹 On Oct. 17, Sage Therapeutics announced a strategic reorganization, including a 33% reduction in its workforce, impacting over 165 employees, including senior leadership roles. This move is intended to extend the company’s cash runway and support the ongoing launch of Zurzuvae for postpartum depression. The restructuring follows a series of clinical setbacks, including the recent failure of Sage’s phase 2 LIGHTWAVE study for Alzheimer’s disease, marking its third failed trial in six months. Sage will now shift focus to prioritizing its pipeline, including an upcoming clinical readout for dalzanemdor in Huntington’s disease.
🔹 On Oct. 17, the FDA approved AbbVie’s Vyalev (foscarbidopa and foslevodopa) as the first 24h continuous subcutaneous infusion treatment for motor fluctuations in advanced Parkinson’s disease. Vyalev offers personalized dosing and provides superior “on” time (periods of optimal motor symptom control) without troublesome dyskinesia compared to standard oral therapies. It allows for continuous symptom management day and night, helping patients maintain better control over Parkinson’s symptoms. The approval is based on a successful phase 3 trial, and the treatment is expected to be covered by Medicare starting in late 2025.
🔹 On Oct. 16, Myriad Genetics and jscreen™ announced a strategic partnership to expand access to genetic testing, particularly for high-risk populations. Myriad’s hereditary cancer and reproductive genetics products will be combined with jscreen’s education and care navigation services. The partnership aims to reach hundreds of thousands of individuals across the U.S., focusing on outreach, education, and in-person genetic screenings, with a special emphasis on individuals of Jewish descent, who are at higher risk for certain genetic conditions.
🔹 On Oct. 16, Tonix Pharmaceuticals submitted a new drug application to the FDA for TNX-102 SL, a non-opioid analgesic designed to treat fibromyalgia. The submission is based on two phase 3 studies showing significant reductions in widespread pain and good tolerability. If approved, TNX-102 SL would be the first new fibromyalgia drug in over 15 years and the first in a new class of medications. The drug, which has Fast Track designation, is taken under the tongue at bedtime and aims to improve non-restorative sleep, a key feature of fibromyalgia. A decision from the FDA is expected in 2025.
🔹 On Oct. 16, Exscientia announced it has reached milestones in two drug discovery programs within its collaboration with Sanofi, earning $15M in payments. Both programs, which aim to create highly differentiated, potentially best-in-class molecules, have advanced to the lead optimization phase. Exscientia could receive over $600M in additional milestone payments and royalties if these programs continue to succeed. The collaboration focuses on developing innovative treatments in areas like oncology and inflammation.
CLINICAL TRIAL UPDATES
🔹 On Oct. 17, Amylyx Pharmaceuticals announced positive results from its phase 2 HELIOS clinical trial for AMX0035 in treating Wolfram syndrome, a rare and progressive neurodegenerative disease. After 24 weeks, participants showed improved pancreatic function, as measured by C-peptide response, along with stabilization in glycemic control, vision, and overall disease burden. These improvements were sustained for participants who completed 36 and 48 weeks of treatment. AMX0035 was well-tolerated with no serious side effects. Amylyx plans to consult with the FDA to design a phase 3 trial and aims to provide updates in 2025.
SCIENCE SPOTLIGHT
🔹 A study published on Oct. 16 in the journal Addiction investigated the association between prescriptions of glucose-dependent insulinotropic polypeptide (GIP) and/or glucagon-like peptide-1 receptor agonists (GLP-1 RA) and substance-related outcomes in patients with opioid use disorder (OUD) and alcohol use disorder (AUD). The research utilized a retrospective cohort design, analyzing de-identified electronic health record data from 136 U.S. health systems, covering over 100 million patients from January 2014 to September 2022. The study included 503,747 patients with OUD and 817,309 patients with AUD, aged 18 years or older.
The primary outcomes were the incidence rates of opioid overdose in the OUD cohort and alcohol intoxication in the AUD cohort. The exposure was defined as having one or more GIP/GLP-1 RA prescriptions. The analysis accounted for various potential confounders, including demographic factors, comorbidities, and medication histories. The study also stratified results based on comorbid conditions of Type 2 diabetes and obesity.
Key findings revealed that patients with GIP/GLP-1 RA prescriptions demonstrated significantly lower rates of opioid overdose and alcohol intoxication compared to those without such prescriptions. When stratified by comorbid conditions, the protective effect of GIP/GLP-1 RA prescriptions remained consistent among patients with OUD and AUD.
These results suggest that prescriptions of GIP/GLP-1 RA medications are associated with lower rates of opioid overdose and alcohol intoxication in patients with OUD and AUD. The protective effects appear consistent across various subgroups, including patients with comorbid type 2 diabetes and obesity. This study provides large-scale, real-world evidence supporting the potential role of GIP/GLP-1 RA medications in addressing substance use disorders.
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DISCLAIMER: This content is for informational purposes only. It should not be taken as legal, tax, investment, financial, or other advice. The views expressed here are my own and do not reflect the opinions of any company or institution.
DISCLOSURE: I have no business relationships with any company mentioned in this article.