Dyne Therapeutics Reports Positive Data From Phase 1/2 DELIVER Trial Of DYNE-251 In DMD

(RTTNews) - Tuesday, Dyne Therapeutics, Inc. (DYN) revealed new clinical findings from its Phase 1/2 DELIVER trial of DYNE-251 for patients with Duchenne muscular dystrophy also known as DMD. The results indicated remarkable dystrophin expression and functional enhancements across various cohorts.

In the trial's 20 mg and 10 mg/kg cohorts, key findings included a mean absolute dystrophin expression of 3.71% in patients receiving the 20 mg/kg dose, with an adjusted value of 8.72% for muscle content. DYNE-251 also showed a favorable safety profile, with most treatment-related side effects being mild or moderate. Significant improvements were recorded in several functional measures for both the 20 mg/kg and 10 mg/kg cohorts, such as the North Star Ambulatory Assessment, Stride Velocity 95th Centile, 10-Meter Walk/Run Time, and Time to Rise from Floor.

The company announced its intention to commence registrational cohorts within the DELIVER trial and aims to provide an update regarding the registration process by the end of 2024.

Additionally, Dyne Therapeutics is advancing its ongoing Phase 1/2 ACHIEVE trial for DYNE-101, targeting myotonic dystrophy type 1.