This Week in Biotech #25
Catch up on the latest biotech breakthroughs and upcoming trends (Nov 1-5).
Welcome to the Wednesday edition of This Week in Biotech by Biotech Blueprint and Happy November 🍂!
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MARKET UPDATES
🔹 On Nov. 4, Sana Biotechnology announced will suspend its oncology development of SC291 and the glial progenitor cell program SC379 while seeking partnerships for these projects, aiming to extend its cash runway into 2026 and enable multiple clinical data readouts in 2024 and 2025. Key initiatives include investigating HIP-modified pancreatic islet cells (UP421) to overcome transplant rejection in type 1 diabetes, with proof of concept data expected soon, and advancing SC291, a CD19-directed CAR T cell therapy for autoimmune diseases, with clinical data anticipated in 2024. On Nov. 5, the company’s stock fell about 12%. JMP has also downgraded Sana to market perform from market outperform.
🔹❗ On Nov. 4, Viking Therapeutics (VKTX) saw an initial 9% surge in stock price after it presented new data on its obesity drug VK2735 at ObesityWeek 2024 in San Antonio, Texas. The phase 1 trial results indicated that VK2735, an oral dual GLP-1/GIP receptor agonist similar to Eli Lilly’s Zepbound, helped patients on a 100 mg dose lose an average of 8.2% of their body weight in 28 days, with minimal adverse effects and no serious incidents. However, after the initial rise, VKTX shares declined sharply, dropping by 14% to $63 per share by market open. The reversal came as investors expressed concerns over Viking’s ability to mass produce VK2735, despite its promising trial data. Viking is committed to advancing VK2735 to phase 2 trials, with results anticipated in the first half of 2024.
🔹 Myriad Genetics’ (MYGN) stock dropped 18% after UnitedHealthcare announced it would stop covering GeneSight, Myriad’s genetic test for matching mental health medications, for its commercial and individual exchange benefit plans starting Jan. 2025. UnitedHealthcare cited insufficient evidence of efficacy for multi-gene panel pharmacogenetic tests, including GeneSight, a decision Myriad strongly opposes. Myriad CEO Paul Diaz voiced disappointment, emphasizing GeneSight’s proven clinical utility, especially as an essential tool for primary care providers treating patients with depression and anxiety.
🔹 As mentioned last week, Argenx (ARGX) recently reported robust Q3 2024 financial results, with $573M in global net product sales, largely driven by Vyvgart, as the company expands in the CIDP market with pending regulatory approvals in several countries. On Nov. 1, Baird downgraded Argenx to Neutral from Outperform, raising its price target to $650, noting that the stock, up 65% over the past six months, now reflects high growth expectations for Vyvgart and pipeline advancements.
🔹 Lexicon Pharmaceuticals (LXRX) announced that the FDA Advisory Committee voted 11-3 against recommending Zynquista (sotagliflozin) as an adjunct to insulin therapy for adults with type 1 diabetes and chronic kidney disease, due to concerns about its risk-benefit balance in this population. Despite the outcome, Lexicon’s CEO, Dr. Mike Exton, highlighted the support from the diabetes community and the need for FDA-approved adjunct therapies to help T1D patients improve glycemic control. The FDA is set to make a final decision by Dec. 20, 2024. Following the advisory committee’s vote, Lexicon’s stock dropped by 35% on Nov. 1.
🔹 ESSA Pharma (EPIX) announced it is terminating its phase 2 trial of masofaniten combined with enzalutamide for treating prostate cancer after an interim analysis showed no added efficacy over enzalutamide alone, the standard treatment. The decision extends to all remaining masofaniten trials, with ESSA planning to withdraw related regulatory applications globally. The said it will focus on strategic options to maximize shareholder value, supported by cash reserves of $126.8M. Following the announcement, ESSA’s stock plummeted by over 70% on Nov. 1.
BIOTECH NEWS
🔹 Beam Therapeutics announced its upcoming presentations at the American Society of Hematology conference in Dec. 2024. Highlights include initial phase 1/2 results for BEAM-101, a base-edited stem cell therapy for sickle cell disease, which showed efficacy with significant fetal hemoglobin induction and reduced sickle hemoglobin in six patients, though one patient died due to complications unrelated to the treatment. Additionally, early data from the BEAM-201 trial for T-cell leukemia and lymphoma indicated a complete response in two of three patients.
🔹 J&J’s Darzalex (daratumumab) received expanded European approval to treat newly diagnosed multiple myeloma patients eligible for autologous stem cell transplant. This decision is based on the phase 3 PERSEUS trial, which showed that the Darzalex-VRd combination significantly reduced disease progression or death risk by 58% compared to VRd alone. Darzalex, approved initially in 2016 for multiple myeloma, is the only subcutaneous CD38-targeting therapy. The drug’s revenue is projected to reach $20B annually by 2030.
🔹 On Nov. 5, Merus announced that the FDA has extended the PDUFA goal date for the biologics license application of its bispecific antibody therapy, zenocutuzumab (Zeno), to Feb. 4, 2025. No further clinical data were requested. Merus is also exploring a commercialization partnership to potentially support the launch of Zeno for NRG1+ cancer if FDA approval is secured.
🔹 On Nov. 5, Arcus Biosciences announced that its drug combination of domvanalimab and zimberelimab significantly improved overall survival in patients with PD-L1-high non-small cell lung cancer (NSCLC) compared to zimberelimab alone or chemotherapy. In the ARC-10 trial, this combination achieved a 36% reduction in the risk of death and had not yet reached median overall survival, while zimberelimab alone reached a median survival of two years. Additionally, the adverse events leading to treatment discontinuation were lower for the domvanalimab and zimberelimab combination (10.5%) compared to chemotherapy (23.5%). The trial included 98 patients with advanced NSCLC. The study, conducted in partnership with Gilead Sciences, aims to assess the benefits of dual inhibition of the TIGIT and PD-1 pathways in improving the immune response against cancer.
🔹 Syndax Pharmaceuticals has announced a $350M synthetic royalty funding agreement with Royalty Pharma for its drug Niktimvo (axatilimab-csfr), which is set to support the drug’s upcoming launch and ongoing development, as well as the development of revumenib. This agreement positions Syndax toward profitability, with projected cash reserves nearing $800M as of Jun. 30. Under the terms, Syndax will receive an upfront payment in exchange for a 13.8% royalty on U.S. net sales of Niktimvo, which targets chronic graft-versus-host disease after prior treatment failures. The drug is expected to be launched in the U.S. in early 2025, co-commercialized with Incyte.
🔹 On Nov. 4, Ascendis Pharma announced a collaboration with Novo Nordisk to develop and commercialize products based on its TransCon technology for metabolic and cardiovascular diseases. Under this agreement, Novo Nordisk has secured an exclusive worldwide license to the TransCon platform for creating treatments for conditions such as obesity and type 2 diabetes, with a focus on a once-monthly GLP-1 receptor agonist as the lead candidate. Ascendis is set to receive up to $285M in payments for the lead program, alongside potential additional payments for further product candidates. Ascendis will handle early development of the TransCon products, while Novo Nordisk will manage clinical development, manufacturing, and commercialization.
🔹 On Nov. 4, BioNTech reported its Q3 2024 financial results, achieving €1.2B in revenues and a net profit of €198.1M. The company advanced its clinical pipeline, presenting data on the bispecific antibody candidate BNT327/PM8002 and the mRNA cancer vaccine BNT113. They launched two phase 2 trials for BNT327/PM8002 in small-cell lung cancer and triple-negative breast cancer and are progressing with a phase 2 trial for the individualized cancer vaccine autogene cevumeran (BNT122). BioNTech introduced variant-adapted COVID-19 vaccines for the 2024/2025 season but anticipates revenues for the year will be at the low end of its guidance range of €2.5 to €3.1B.
🔹❗ On Nov. 1, Novo Nordisk announced the results of part 1 of the ESSENCE trial, which evaluated semaglutide 2.4 mg in 1,200 adults with metabolic dysfunction-associated steatohepatitis (MASH) and moderate to advanced liver fibrosis. The trial demonstrated that semaglutide significantly improved liver fibrosis and led to the resolution of steatohepatitis compared to a placebo at 72 weeks. The treatment was well tolerated. Novo Nordisk plans to seek regulatory approval for semaglutide in the US and EU in the first half of 2025, with full results to be presented at a scientific conference in 2024. Part 2 of the trial is ongoing, aiming to assess long-term clinical outcomes over 240 weeks. MASH is a serious liver disease that affects over 250 million people worldwide, with the number of advanced cases expected to double by 2030.
CLINICAL TRIAL UPDATES
🔹 On Nov. 5, Arcellx announced positive clinical results for its multiple myeloma therapy, anito-cel, to be presented at the upcoming ASH Annual Meeting. Key findings include a 95% overall response rate and 62% complete response in the phase 2 iMMagine-1 study, with no major neurotoxicities observed. The phase 1 trial showed a median progression-free survival of 30.2 months, and the ongoing iMMagine-3 phase 3 study recently began dosing its first patient.
🔹 On Nov. 3, 2024, CinFina Pharma announced positive phase 1 results for its obesity treatments CIN-110 and CIN-109 at ObesityWeek 2024. CIN-110, a long-acting PYY3-36 analog, showed significant reductions in caloric intake (up to 28%) and body weight (up to 1.8%) after a single dose in a SAD study. It was well tolerated, with mild side effects and no withdrawals due to adverse events. CIN-109, a first-in-class GDF-15 analog, produced meaningful weight loss (up to 3.7%) and reduced food intake (up to 50%) in a MAD study. Participants tolerated the treatment well, particularly with the every-other-week dosing schedule. CinFina’s CEO emphasized the potential of these next-generation therapies to meet the growing demand for effective obesity medications. Both candidates are set for further clinical development.
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