Dyne Therapeutics disclosed that 24‑month analyses from its Phase 1/2 DELIVER trial of z‑rostudirsen (DYNE‑251) show measurable gains in heart and lung function for patients with exon 51‑skipping Duchenne muscular dystrophy. The data were presented in a poster at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference held March 8‑11, 2026.
The cardiopulmonary metrics that improved include forced vital capacity percent predicted (FVC%p), circumferential strain, and left ventricular ejection fraction. These gains were observed relative to the expected natural‑history decline in the patient population, underscoring the therapeutic’s impact on the two leading causes of morbidity and mortality in DMD.
The results reinforce the validity of Dyne’s FORCE platform, which is engineered to deliver therapeutics to skeletal, cardiac, and respiratory muscle tissues. The data support the company’s plan to seek accelerated regulatory approval and to launch a quarterly‑dosed therapy that targets both skeletal and cardiac muscle. Z‑rostudirsen already holds Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations from the FDA, EMA, and Japan’s Ministry of Health, Labour and Welfare.
Chief Medical Officer Doug Kerr said, “This week we are presenting additional analyses of 24‑month data from the DELIVER trial showing the breadth of potential benefits z‑rostudirsen may bring to individuals with exon 51 skip amenable DMD beyond the previously reported unprecedented improvements in muscle function.” He added that the cardiopulmonary benefits “are a key area of concern in DMD, and we are particularly encouraged by new analyses showing improvement in both heart and lung function out to 24 months.”
Dyne plans to initiate a global Phase 3 study to support worldwide approvals and has outlined a potential U.S. launch of z‑rostudirsen in Q1 2027, contingent on FDA priority review. The company’s strategy to combine a quarterly dosing regimen with a platform that reaches multiple muscle groups positions it to address unmet needs in a disease with limited treatment options.
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