Ascendis Pharma reported positive two-year data for achondroplasia treatment. Data from the ApproaCH trial, announced on March 16, showed consistent growth improvement in children with achondroplasia using TransCon CNP. Further improvement in body proportionality was observed during the second year of weekly treatment.
Agios Pharmaceuticals (Nasdaq: AGIO) announced its intention to pursue accelerated approval from the U.S. Food and Drug Administration (FDA) for its drug mitapivat. The treatment is designed for patients suffering from sickle cell disease, a chronic and debilitating blood disorder. This strategic decision follows a…
Read the full storyPepGen Inc. (Nasdaq: PEPG) has announced positive topline results from the 5 mg/kg dose cohort of its Phase 2 FREEDOM2-DM1 clinical trial for Myotonic Dystrophy Type 1 (DM1). The therapy demonstrated a favorable safety profile and promising results in genetic splicing, indicating biological activity even at the lowest…
Alnylam Pharmaceuticals (Nasdaq: ALNY) presented new clinical and real-world data at the American College of Cardiology's Annual Scientific Session (ACC.26). The findings reinforce the therapeutic potential of the company's RNAi portfolio, specifically highlighting the efficacy of Vutrisiran in treating transthyretin…
Bristol Myers Squibb (BMS) has announced positive results from its Phase 3 SCOUT-HCM trial, evaluating Camzyos in adolescents with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The data, presented at the American College of Cardiology (ACC) conference, confirms the drug's efficacy and safety in a younger…
MoonLake Immunotherapeutics has released long-term Week 40 data from its global Phase 3 registrational program for Sonelokimab. The results stem from the VELA-1 and VELA-2 trials, focusing on patients with moderate-to-severe Hidradenitis Suppurativa (HS). This clinical data was presented at the 2026 American Academy…
Priovant Therapeutics has announced the formal publication of positive Phase 3 VALOR trial results for brepocitinib in the New England Journal of Medicine (NEJM). The study evaluated the efficacy and safety of the drug in adults suffering from dermatomyositis (DM), a rare and debilitating autoimmune disease. The…
Corcept Therapeutics (NASDAQ: CORT) recently presented late-breaking clinical data from its MOMENTUM trial during the American College of Cardiology (ACC) Annual Scientific Session. The study focused on investigating the prevalence of endogenous hypercortisolism among patients suffering from resistant hypertension.…
Disc Medicine, Inc. (NASDAQ: IRON) has announced the successful completion of participant enrollment for its pivotal Phase 3 APOLLO clinical trial. The trial evaluates bitopertin as a potential treatment for Erythropoietic Protoporphyria (EPP), a serious and rare hematologic disorder. Due to significant interest from…
Seaport Therapeutics, a biotech firm founded by PureTech Health, has announced the publication of peer-reviewed research in the prestigious journal Science Translational Medicine regarding its SPT-300 candidate. The study identifies SPT-300 as the first triglyceride-mimetic prodrug to successfully achieve…
Denali Therapeutics Inc. (Nasdaq: DNLI) has secured accelerated approval from the U.S. Food and Drug Administration (FDA) for its drug AVLAYAH. The medication is indicated for the treatment of Hunter syndrome (MPS II) in pediatric patients weighing at least 5 kg. Notably, AVLAYAH is the first FDA-approved biologic…
Vaxcyte (PCVX) announced that the prestigious medical journal The Lancet Infectious Diseases has published positive results from its Phase 1/2 study of VAX-31. VAX-31 is a 31-valent pneumococcal conjugate vaccine candidate designed to provide broader protection against bacterial infections. Following these successful…