On Tuesday, Dyne Therapeutics, Inc. (DYN) disclosed new clinical findings from its Phase 1/2 DELIVER trial of DYNE-251 for patients with Duchenne muscular dystrophy (DMD). The data demonstrated notable dystrophin expression and functional improvements across various patient groups.
Key results from the 20 mg/kg and 10 mg/kg cohorts included an average 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 exhibited a favorable safety profile, with the majority of treatment-related side effects being mild or moderate. Significant advancements were observed in several functional measures for both the 20 mg/kg and 10 mg/kg groups, including the North Star Ambulatory Assessment, Stride Velocity 95th Centile, 10-Meter Walk/Run Time, and Time to Rise from Floor.
The company announced plans to initiate registrational cohorts within the DELIVER trial and aims to provide an update on the registration process by the end of 2024.
Moreover, Dyne Therapeutics is progressing with its ongoing Phase 1/2 ACHIEVE trial for DYNE-101, aimed at treating myotonic dystrophy type 1.
