WASHINGTON (dpa-AFX) - Dyne Therapeutics Inc. (DYN) announced new long-term clinical data from its 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 trial is evaluating DYNE-251 in individuals with Duchenne muscular dystrophy or DMD who are amenable to exon 51 skipping.
The company will present updated results from the trial this week at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
The updated assessment of the DELIVER trial evaluating DYNE-251 includes new functional data out to 12 months from 6 patients enrolled in the 20 mg/kg Q4W cohort, and 18-month functional data from 6 patients in the 10 mg/kg Q4W cohort. In addition, updated safety data as of February 7, 2025, continue to demonstrate a favorable safety profile for DYNE-251.
According to the company, Meaningful and sustained improvements from baseline in multiple functional endpoints were observed in both the 20 mg/kg (selected registrational dose) and 10 mg/kg1 DYNE-251 Q4W cohorts, through 12 and 18 months, respectively. Functional assessments included Stride Velocity 95th Centile (SV95C), North Star Ambulatory Assessment (NSAA), 10-Meter Walk/Run Time (10-MWR), and Time to Rise from Floor.
As previously reported, DYNE-251 demonstrated unprecedented near-full length dystrophin expression as measured by Western blot for patients with DMD who are amenable to exon 51 skipping. At the 6-month time point, patients treated with 20 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin expression of 8.72% of normal, the company said.
DYNE-251 has demonstrated a favorable safety profile based on 54 participants enrolled in the DELIVER trial. Since the prior update provided as of November 21, 2024, the safety profile remains unchanged, and no new treatment-related serious adverse events have been observed.
Dyne said it continues to pursue expedited approval pathways globally for DYNE-251 in patients with DMD who are amenable to exon 51 skipping.
Dyne has fully enrolled the Registrational Expansion Cohort of 32 patients as part of the DELIVER trial. Data from this cohort are planned for late 2025.
Dyne anticipates a potential Biologics License Application (BLA) submission for U.S. accelerated approval in early 2026.
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