Dyne Therapeutics reported that the Registrational Expansion Cohort of its Phase 1/2 DELIVER trial met its primary endpoint, achieving a statistically significant increase in muscle‑content‑adjusted dystrophin expression to 5.46 % of normal at six months (p < 0.0001). This level of dystrophin restoration exceeds the 2–3 % range typically seen in earlier exon‑skipping studies and represents a meaningful therapeutic advance for patients with exon 51‑skipping mutations.
The study also documented functional gains across multiple prespecified endpoints. Time to Rise Velocity improved by 12 %, 10‑Meter Walk/Run Velocity increased by 9 %, and Forced Vital Capacity Percent Predicted was preserved, with no decline observed over the 24‑month follow‑up. These sustained benefits suggest that the therapeutic effect is durable and clinically relevant for the 1,600‑patient U.S. population eligible for exon 51 skipping.
Dyne’s data set the stage for a U.S. Accelerated Approval submission in the second quarter of 2026. The company’s quarterly‑dosed regimen would be the first of its kind for Duchenne muscular dystrophy, potentially shortening the treatment interval compared with existing monthly therapies and improving patient adherence. A successful approval would open a market with a clear unmet need and a defined patient cohort.
The trial success validates Dyne’s proprietary FORCE™ platform, which is engineered for targeted delivery to skeletal muscle and the central nervous system. The platform is already being leveraged in other pipeline programs, including z‑basivarsen for myotonic dystrophy type 1 and preclinical work in facioscapulohumeral muscular dystrophy and Pompe disease. Although Dyne is a clinical‑stage company without product sales, its balance sheet remains robust, with high liquidity and low debt, positioning it to fund continued development and potential commercialization of its pipeline.
CEO John Cox emphasized the clinical significance, stating, “With its high level of dystrophin expression, favorable safety profile, and convenient monthly dosing, z‑rostudirsen has the potential to transform the care of those living with DMD amenable to exon 51 skipping.” Chief Medical Officer Doug Kerr added, “The data from all 86 participants will form the basis for our planned U.S. Accelerated Approval submission.”
In the broader therapeutic landscape, Dyne’s dystrophin expression level appears higher than that reported for Sarepta’s Exondys 51, though cross‑trial comparisons are limited. The company’s quarterly dosing schedule and robust safety profile could provide a competitive edge, while the strong regulatory designations—Fast Track, Orphan Drug, and Rare Pediatric Disease—further support a streamlined approval pathway.
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