This Week in Biotech #19
Catch up on the latest biotech breakthroughs and upcoming trends (Oct 11-15).
Welcome to the Wednesday edition of This Week in Biotech by Biotech Blueprint!
🎙️ PODCAST
MARKET UPDATES
🔹 On Oct. 15, MeiraGTx (MGTX) announced positive results from its study of AAV-GAD for Parkinson’s disease, showing significant improvements in both safety and efficacy. The primary goal of safety and tolerability was met, and the high-dose group demonstrated a remarkable improvement on the Parkinson’s rating scale and quality of life enhancements. The study’s promising results led to a 15% surge in MeiraGTx’s stock. The company aims to initiate phase 3 trials.
🔹 Following the FDA’s clearance for Annovis Bio (ANVS) to proceed with pivotal phase 3 clinical trials for its Alzheimer’s disease treatment, buntanetap, the company’s stock saw a significant boost on Oct. 15, rising over 6%. The phase 3 program will include two trials: a 6 month study to confirm symptomatic benefits and an 18 month study to assess potential disease modifying effects. A well-executed 6 month trial could lead to a new drug application filing within a year. The FDA raised no concerns regarding buntanetap’s safety or pharmacokinetics.
🔹 On Oct. 14, Amgen’s (AMGN) stock declined after Truist Securities downgraded the company from a Buy to a Hold rating, despite raising the price target from $320 to $333. The downgrade was based on the belief that the $5B market potential of Amgen’s obesity drug, MariTide, is already factored into the current stock price. MariTide is in phase 2 trials, showing promising weight loss results by targeting GLP-1 and GIP receptors. However, the competitive obesity drug landscape, with rivals like Lilly’s Zepbound and other emerging treatments, poses challenges. If MariTide’s effectiveness and safety prove superior, peak sales could reach $10B.
🔹 On Oct. 14, Jasper Therapeutics (JSPR) reported positive data from its SPOTLIGHT phase 1b/2a study on briquilimab for chronic inducible urticaria, with 93% of participants achieving a clinical response and 83% of those in the 120mg cohort experiencing a complete response. The drug was well tolerated, with no serious adverse events reported. Following the news, Jasper’s stock rose over 10%. The company also announced it will present initial data from its BEACON study on chronic spontaneous urticaria in January 2025, with full SPOTLIGHT study results expected in the first half of 2025.
BIOTECH NEWS
🔹 GSK has filed lawsuits against Moderna in U.S. federal court, accusing it of infringing on GSK’s mRNA technology patents through Moderna’s COVID-19 vaccine Spikevax and RSV vaccine mResvia. GSK claims Moderna’s use of lipid nanoparticles to deliver mRNA into the body violates several of its patents. GSK is seeking unspecified monetary damages but is open to licensing the patents under “commercially reasonable terms.” This lawsuit adds to ongoing legal battles over patent royalties involving Pfizer, BioNTech, and Moderna for their COVID vaccines. Moderna, which earned $6.7B from Spikevax in 2023, plans to defend against GSK’s claims. GSK’s patents stem from innovations developed in 2008, which it acquired through Novartis in 2015.
🔹 On Oct. 15, Triana Biomedicines entered into a strategic collaboration with Pfizer to discover novel molecular glue degraders targeting multiple disease areas, including oncology. As part of the agreement, Triana will receive an upfront payment of $49M and is eligible for milestone payments exceeding $1.5B, as well as royalties. Triana will leverage its molecular glue discovery platform to identify candidates for Pfizer, which will have the option to license and develop them further.
🔹 On Oct. 14, Lundbeck announced its plan to acquire Longboard Pharmaceuticals in a strategic deal valued at approximately $2.6B. This acquisition is aimed at bolstering Lundbeck’s neuroscience pipeline, particularly in the area of developmental and epileptic encephalopathies. The key asset in the acquisition is bexicaserin, a drug in late-stage development that targets 5-HT2C receptors and has shown promising results in reducing seizures for conditions like Dravet syndrome and Lennox-Gastaut syndrome. Currently undergoing a phase 3 clinical trial, bexicaserin holds significant potential, with estimated peak global sales of up to $2B.
🔹 On Oct. 14, GSK announced positive phase 3 results from the ANCHOR-1 and ANCHOR-2 trials, which evaluated depemokimab for treating chronic rhinosinusitis with nasal polyps. The trials met primary endpoints, showing significant reductions in nasal polyp size and nasal obstruction over 52 weeks compared to a placebo. Depemokimab, an ultra-long-acting biologic targeting IL-5, is administered every six months. This condition affects up to 4% of the population and often requires surgery and corticosteroid use. GSK is preparing for global regulatory filings.
🔹 On Oct. 14, Exelixis and Merck entered into a clinical development collaboration to evaluate Exelixis’ investigational drug zanzalintinib in combination with two of Merck’s therapies. The partnership will test zanzalintinib with Keytruda (pembrolizumab) in a phase 3 trial for head and neck squamous cell carcinoma (HNSCC) and with Welireg (belzutifan), Merck’s HIF-2α inhibitor, in both phase 1/2 and phase 3 trials for renal cell carcinoma. Merck will provide the drugs for these trials, and both companies will share funding responsibilities. Exelixis retains the global commercial rights to zanzalintinib.
🔹 Last week, I mentioned how a compounding industry group sued the FDA for removing Eli Lilly’s tirzepatide from its shortage list. The lawsuit, filed in Texas, claims the drug is still in short supply and argues the FDA’s decision was arbitrary, without public input. On Oct. 11, the FDA agreed to reconsider its decision to prevent compounded versions of Lilly’s tirzepatide from being sold. The FDA’s reconsideration allows compounding pharmacies to continue providing these cheaper alternatives while the regulatory agency assesses the drug’s availability. In the meantime, Novo Nordisk has initiated legal action against yet another semaglutide compounder, bringing the total number of lawsuits it has filed to 50.
🔹 On Oct. 11, Denali Therapeutics and its partner Sanofi have halted a mid-stage clinical trial for their experimental multiple sclerosis drug, oditrasertib (DNL788), after it failed to meet the primary and secondary goals. This is the second setback for oditrasertib, as it also failed in a separate study for treating amyotrophic lateral sclerosis (ALS). The partnership between Sanofi and Denali, established in 2018, focuses on developing treatments for neurological and inflammatory diseases.
CLINICAL TRIAL UPDATES
🔹 On Oct. 15, Jazz Pharmaceuticals announced positive results from a phase 3 trial of Zepzelca (lurbinectedin) combined with Tecentriq (atezolizumab) in first-line maintenance therapy for extensive-stage small cell lung cancer. The combination therapy showed statistically significant improvements in overall survival and progression-free survival compared to atezolizumab alone. Based on these results, Jazz plans to submit a supplemental new drug application in early 2025.
🔹 On Oct. 11, Perspective Therapeutics announced progress in its phase 1/2a trial of VMT01, a radiopharmaceutical therapy for melanoma. The drug was safe, with no dose-limiting toxicities. In Cohort 1, only 1/3 patients showed a measurable response following treatment, while the remaining 2 maintained stable disease for 9 and 11 months. In Cohort 2, among 7 patients, 3 experienced disease progression after the first treatment cycle, and the other 4 progressed after the second cycle. Following the news, the company’s stock dipped but has since partially recovered.
🔹 On Oct. 11, Sagimet Biosciences announced positive results from its phase 2b trial of denifanstat in patients with metabolic-dysfunction associated steatohepatitis (MASH) and fibrosis (F2/F3). The study, published in The Lancet Gastroenterology & Hepatology, showed significant improvements in MASH resolution and fibrosis, with 49% of advanced fibrosis patients (F3) improving without worsening MASH. The drug was well tolerated with no severe side effects. These findings support advancing denifanstat to phase 3 trials in 2024.
🔹 On Oct. 11, Capricor Therapeutics announced positive long-term results from its HOPE-2 open label extension study at the 2024 World Muscle Society Congress, highlighting the efficacy and safety of its lead therapy, deramiocel, in treating Duchenne muscular dystrophy. The 3 year data showed significant and sustained improvements in both cardiac and skeletal muscle function particularly in patients with cardiomyopathy, which is a leading cause of death in DMD.
🔹 Allakos announced positive results from its phase 1 trial of AK006, a subcutaneous treatment targeting mast cell-driven diseases. The drug showed about 77% bioavailability and extended receptor occupancy on mast cells, with a favorable safety profile and no serious adverse events. Tested at doses of 150 mg and 720 mg, the higher dose showed 98% receptor occupancy at day 113, indicating potential for infrequent dosing. AK006 is being developed to treat conditions such as chronic spontaneous urticaria.
Happy Wednesday and thanks for reading Biotech Blueprint!
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.