Dyne Therapeutics Announces New Long-Term Clinical Data
Dyne Therapeutics Announces New Long-Term Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented and Sustained Functional Improvement Through 18 Months
- Continued favorable safety profile for DYNE-251 -
- DELIVER Registrational Expansion Cohort is fully enrolled; data from this cohort planned for late 2025 -
- Potential for Biologics License Application submission for U.S. accelerated approval in early 2026 -
WALTHAM, Mass., March 16, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced new long-term clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 demonstrating unprecedented and sustained functional improvement at the selected registrational dose of 20 mg/kg Q4W (approximate PMO dose). The DELIVER trial is evaluating DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, and updated results from the trial are being presented this week at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
“With Dyne-251, we have the opportunity to deliver a durable and redosable therapy demonstrating clinically meaningful and sustained functional improvement in DMD. The consistency of these new data across multiple endpoints and timepoints underscores the potential of DYNE-251 to meaningfully address the significant unmet need in Duchenne despite available therapies,” said John Cox, president and chief executive officer of Dyne. “We are rapidly advancing DYNE-251 toward a readout later this year with the potential to submit for U.S. accelerated approval in early 2026 based on a well-established regulatory pathway leveraging dystrophin expression as a surrogate endpoint. If approved, we believe there is an opportunity for rapid adoption by physicians and currently treated patients, as well as those naïve to therapy.”