Dyne Therapeutics Announces Initiation of Phase 3 FORZETTO Trial of Z-Rostudirsen in Duchenne Muscular Dystrophy (DMD) Ahead of Planned BLA Submission for U.S. Accelerated Approval

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[{"type":"text","content":"- 72-week trial will enroll approximately 90 participants; first site now open for enrollment -- Primary endpoint is rise from floor (RFF) velocity with multiple secondary endpoints to assess muscle and pulmonary function -- FORZETTO trial design and protocol aligned with FDA; trial intended to serve as confirmatory trial for traditional approval in the U.S. and support ex-U.S. marketing applications - WALTHAM, Mass., May 20, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced the initiation of the Phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The design of the FORZETTO trial will be presented at the 19th International Congress on Neuromuscular Diseases (ICNMD) being held July 7-11, 2026, in Florence, Italy. “As we approach the planned submission of our application for Accelerated Approval in the U.S. later this quarter based on the unprecedented results seen in the DELIVER trial, we are pleased to have initiated a Phase 3 trial designed to further demonstrate the potential of z-rostudirsen to enable functional improvement for people with DMD amenable to exon 51 skipping,” said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. “Z-rostudirsen is designed to enable the production of near-full length dystrophin across muscle tissues and the central nervous system in order to improve how DMD patients feel and function. By assessing multiple measures of mobility, lung health and patient-reported outcomes, we aim to demonstrate the breadth of potential benefits of z-rostudirsen.” FORZETTO is a global, randomized, placebo-controlled, double-blind, confirmatory Phase 3 trial designed to assess the efficacy, safety, and tolerability of z-rostudirsen administered intravenously to ambulatory male participants with DMD amenable to exon 51 skipping. The trial will enroll approximately 90 participants 4 to 18 years of age who will be randomized 1:1 to receive 20 mg/kg of z-rostudirsen or placebo every four weeks (Q4W). The first trial site is activated and open to enrollment. The primary endpoint is the change from baseline in rise from floor (RFF) ve...

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