Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne Muscular Dystrophy (DMD)

Core Insights - Dyne Therapeutics announced positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1/2 DELIVER trial for zeleciment rostudirsen (z-rostudirsen) in Duchenne muscular dystrophy (DMD), showing a statistically significant increase in dystrophin expression and functional improvements across multiple clinical endpoints [2][3][4] Clinical Trial Results - The REC met its primary endpoint with a muscle content-adjusted dystrophin expression of 5.46% of normal at six months (p<0.0001), replicating a previously observed 7-fold increase from baseline [3][6] - Functional improvements were noted across all six prespecified clinical endpoints, including Time to Rise (TTR) Velocity and 10-Meter Walk/Run (10MWR) Velocity, both showing nominal p<0.05 compared to placebo [4][10] - Lung function, measured by Forced Vital Capacity Percent Predicted (FVC%p), was preserved at six months, contrasting with a decline in the placebo group [4][8] Long-term Efficacy and Safety - New long-term results from the DELIVER trial indicated sustained functional improvement across all assessed endpoints through 24 months [12] - The safety profile of z-rostudirsen remained favorable, with most treatment-emergent adverse events being mild or moderate, and no serious adverse events reported in the REC [9][11] Regulatory and Market Outlook - The company is on track to submit for U.S. Accelerated Approval in Q2 2026, with a potential market launch in Q1 2027, targeting approximately 1,600 individuals with significant unmet needs [5][16] - Dyne Therapeutics is also pursuing approval pathways outside the U.S. for z-rostudirsen [16][17] Company Background - Dyne Therapeutics focuses on developing therapeutics for genetically driven neuromuscular diseases, with ongoing clinical programs for DMD and other conditions [19]