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Dyne Therapeutics Announces Initiation of Phase 3 HARMONIA Trial of Z-Basivarsen in Myotonic Dystrophy Type 1 (DM1)
- HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 - - 48-week trial will enroll approximately 150
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 - - 48-week trial will enroll approximately 150 individuals, and first sites are now open for enrollment - - Primary endpoint is the five times sit to stand (5xSTS) test; secondary and exploratory endpoints will assess muscle function, CNS manifestations, and patient- and clinician-reported outcomes - - HARMONIA 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., March 08, 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 HARMONIA trial of zeleciment basivarsen (z-basivarsen, also known as DYNE-101), in individuals with myotonic dystrophy type 1 (DM1). The design of the HARMONIA trial is being presented at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference on Wednesday, March 11, 2026 at 9:30 a.m. ET. A corresponding poster is also available in the Scientific Publications & Presentations section of Dyne’s website. “We are proud to be contributing to key advances in myotonic dystrophy clinical research with the initiation of a field-defining Phase 3 study designed to demonstrate the broad potential benefits of z-basivarsen,” said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. “Building on the ongoing registrational expansion cohort of the Phase 1/2 ACHIEVE trial, which is utilizing myotonia, as measured by video hand opening time, as an early indicator of clinical benefit for potential U.S. Accelerated Approval, HARMONIA is a larger and longer-term study utilizing a clinically meaningful functional measure as the primary endpoint. HARMONIA was designed to reinforce the best-in-class potential of z-basivarsen based on the differentiated capabilities of our FORCE platform to deliver therapeutics to a broad range of muscle systems as well as the CNS.” HARMONIA is a global, randomized, placebo-controlled, double-blind, confirmatory Phase 3 trial designed to assess the multi-system efficacy, safety, and tolerability of z-basivarsen administered intravenously to individuals wit...