Dyne Therapeutics Shares Rise After Announcing Positive Topline Results for z-rostudirsen in Phase 1/2 DMD Trial

Core Insights - Dyne Therapeutics Inc. has announced positive topline results from its Phase 1/2 DELIVER trial for z-rostudirsen, a treatment for Duchenne muscular dystrophy (DMD) [1][3] - The trial demonstrated a statistically significant increase in muscle content-adjusted dystrophin expression to 5.46% of normal at 6 months, meeting its primary endpoint [3] - Dyne Therapeutics is preparing to submit a Biologics License Application for US Accelerated Approval in Q2 2026 [3] Company Overview - Dyne Therapeutics is a clinical-stage company focused on developing therapeutics for neuromuscular diseases in the US [4] - The investigational drug z-rostudirsen utilizes Dyne's FORCE platform to produce near full-length dystrophin [2] Regulatory Designations - Z-rostudirsen has received multiple designations from the US FDA, including Breakthrough Therapy, Fast Track, and Rare Pediatric Disease, as well as Orphan Drug designations from the FDA, EMA, and MHLW in Japan [3] Clinical Trial Results - The DELIVER trial also provided positive long-term clinical data from its ongoing open-label extension and long-term extension portions [2] - Functional improvements were observed across all six prespecified clinical endpoints at 6 months compared to baseline, with declines noted in the pooled placebo group [3]