Edgewise Therapeutics’ stock soared after positive Phase 2 trial results for its Becker muscular dystrophy treatment. Other companies saw significant pre-market price changes, with some gaining substantially while others experienced sharp declines, reflecting the volatility of the market. The biotech sector and clinical trial results remain major market drivers.
Outlook Therapeutics announced that its ONS-5010 drug for wet age-related macular degeneration (AMD) did not meet the primary endpoint in the NORSE EIGHT clinical trial. Despite this setback, the company plans to resubmit its marketing application to the FDA in Q1 2025 based on ongoing data analysis and the drug’s favorable safety profile. The stock price has fallen significantly following the announcement.
Interim data from IGC Pharma’s Phase 2 trial reveals IGC-AD1, a novel drug targeting neuro-inflammation and CB1 receptor dysfunction, demonstrates significant cognitive improvement in Alzheimer’s patients alongside previously reported agitation reduction. This offers potential for a new treatment addressing both symptoms and underlying disease progression.
French biotech company Aelis Farma announced positive results from a Phase 1/2 trial of its drug candidate AEF0217 for young adults with Down syndrome. The drug showed significant improvements in adaptive behavior and cognition, paving the way for potential treatments of other cognitive disorders.
Syros Pharmaceuticals’ tamibarotene, in combination with azacitidine, failed to meet its primary endpoint in a Phase 3 trial for higher-risk myelodysplastic syndrome (HR-MDS). The trial, SELECT-MDS-1, evaluated the treatment’s effectiveness in patients with RARA gene overexpression. The company plans to stop the study and assess the next steps, while its stock has plummeted over 86% following the news.
Rapt Therapeutics, Inc. (RAPT) saw its stock plummet by over 46% on Monday after the company announced the termination of its zelnecirnon (RPT193) program due to a serious adverse event (SAE) of liver injury. The program was being investigated in two Phase 2 clinical trials for asthma and atopic dermatitis, but both trials were placed on FDA clinical hold in February 2024. This decision followed a liver injury requiring a transplant in a patient participating in the atopic dermatitis trial. The company subsequently closed both studies and discontinued the zelnecirnon program, highlighting the challenges faced in drug development due to safety concerns. Rapt Therapeutics is now focusing on developing next-generation CCR4 compounds with improved safety profiles.
ESSA Pharma Inc. has announced the termination of its Phase 2 trial for masofaniten in combination with enzalutamide for metastatic castration-resistant prostate cancer (mCRPC). The decision was based on interim data showing a lower PSA90 response rate compared to enzalutamide alone, and a lack of clear efficacy benefit with the combination. ESSA is also halting other masofaniten studies and will focus on its remaining resources.
The Sabin Vaccine Institute has expanded its clinical trial for an investigational Marburg virus vaccine in Rwanda, increasing the number of participants to include mine workers at risk of exposure. The expanded trial aims to assess the vaccine’s safety, immunogenicity, and efficacy. This initiative comes amidst a large-scale Marburg outbreak in Rwanda, where over 1,500 frontline workers have already been vaccinated with the Sabin vaccine.
Boston Scientific’s ACURATE neo2 Aortic Valve System failed to meet its primary endpoint in the ACURATE IDE trial, resulting in a decline in the company’s stock price. The trial compared the ACURATE neo2 valve to competitor valves from Edwards Lifesciences and Medtronic, but the ACURATE neo2 failed to demonstrate non-inferiority in terms of mortality, stroke, or rehospitalization at one year. Despite this setback, the company highlighted positive results from a post-hoc analysis, and it continues to work with the FDA for U.S. approval.
Tharimmune, Inc. (THAR) stock skyrocketed on Wednesday after the company received positive feedback from the European Medicines Agency (EMA) regarding its TH104 clinical program for treating pruritus in primary biliary cholangitis. This positive news, combined with the company’s commitment to advancing innovative solutions in inflammation and immunology, has investors excited about the future of TH104.
