Dyne Therapeutics announced positive long-term clinical data from its Phase 1/2 DELIVER trial of DYNE-251 for Duchenne muscular dystrophy (DMD). The data shows sustained functional improvement in patients amenable to exon 51 skipping at the 20 mg/kg dose. The company plans to submit a Biologics License Application for accelerated approval in the U.S. in early 2026.
This data is potentially game-changing for DMD patients as current therapies offer limited benefits. The sustained functional improvements observed with DYNE-251, particularly exceeding the minimal clinically important difference in Stride Velocity 95th Centile, suggest a potential for a significant impact on patients’ quality of life. The possibility of an accelerated approval pathway further underscores the urgent need and potential for this therapy to quickly reach those who could benefit.
The DELIVER trial data revealed sustained improvements in multiple functional endpoints, including Stride Velocity 95th Centile, North Star Ambulatory Assessment, 10-Meter Walk/Run Time, and Time to Rise from Floor, at both 10 mg/kg and 20 mg/kg doses. Unprecedented near-full length dystrophin expression was observed. The safety profile of DYNE-251 remains favorable after 970 administered doses and over 77 patient-years of follow-up. The registrational expansion cohort of the DELIVER trial is fully enrolled, with data expected in late 2025.
The positive data and planned BLA submission for DYNE-251 positions Dyne Therapeutics at the forefront of DMD treatment. The potential for accelerated approval could lead to rapid adoption of the therapy, offering new hope to DMD patients and potentially reshaping the treatment landscape for this debilitating disease.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
