Dyne Therapeutics announced positive topline results from the Registrational Expansion Cohort of the Phase 1/2 DELIVER trial for Duchenne Muscular Dystrophy.
The trial demonstrated a significant increase in dystrophin expression, functional improvement, and sustained positive long-term results.
Results showed continued favorable safety and tolerability profile, with a submission for U.S. Accelerated Approval on track for Q2 2026.
Primary Endpoint Met
The REC met its primary endpoint with a statistically significant increase in dystrophin expression to 5.46% at 6 months.
Functional Improvement
Functional improvement was observed across multiple clinical endpoints, including preserved lung function at 6 months.
Long-Term Results
New positive long-term data from the DELIVER trial showed sustained functional improvement through 24 months.
Accelerated Approval
Dyne is on track for U.S. Accelerated Approval submission in Q2 2026, with a potential launch in Q1 2027 assuming Priority Review.
Clinical Potential
The high level of dystrophin expression and favorable safety profile of z-rostudirsen position it as a transformative therapy for DMD patients.
- The results demonstrate the potential for z-rostudirsen to transform care for DMD patients.
- The data supports a submission for U.S. Accelerated Approval, with positive implications for the DMD community and future therapies.
With unprecedented clinical data and promising results, Dyne Therapeutics is poised for potential transformation in DMD care and future therapies.