Dyne Therapeutics, Inc. (DYN), a clinical-stage company, is advancing its lead drug candidate, Zeleciment rostudirsen, toward a major regulatory milestone next year.
Zeleciment rostudirsen, DYNE-251, being developed for individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amenable to exon 51 skipping, has been successfully tested in a Phase 1/2 trial, dubbed DELIVER.
Duchenne muscular dystrophy (DMD) is a rare X-linked progressive neuromuscular disorder caused by mutations in the DMD gene. These mutations result in a complete or near-complete absence of dystrophin, a protein critical for maintaining muscle structure and function. Zeleciment rostudirsen is designed to enable the production of near full-length dystrophin in muscle and the central nervous system (CNS).
In the Registrational Expansion Cohort (REC) of the phase 1/2 DELIVER trial, the results of which were announced yesterday, Zeleciment rostudirsen demonstrated a statistically significant increase in dystrophin levels, reaching 5.46% at six months. Functional improvements were observed across multiple clinical endpoints, and lung function was maintained during this period.
The DELIVER trial further demonstrated that these functional gains were sustained across all assessed endpoints through 24 months, with a continued favourable safety and tolerability profile.
Based on the encouraging DELIVER trial results, Dyne plans to submit a BLA (Biologics License Application) for U.S. Accelerated Approval of Zeleciment rostudirsen in DMD in the second quarter of 2026. The company plans to initiate a global phase 3 clinical trial of Zeleciment rostudirsen during the same time frame to support global approvals.
Assuming FDA grants Priority Review, the company expects Zeleciment rostudirsen to hit pharmacy shelves in Q1 2027.
Another clinical drug candidate of the company that merits attention is Zeleciment basivarsen, also known as DYNE-101, being evaluated in a phase I/II global clinical trial in Myotonic Dystrophy Type 1, dubbed ACHIEVE.
A Registrational Expansion Cohort of the ACHIEVE trial with video hand opening time (vHOT) as the primary endpoint, to serve as an intermediate clinical endpoint, is enrolling patients, with enrolment expected to be completed in early Q2, 2026. If all goes well as planned, a potential BLA (Biologics License Application) submission for U.S. Accelerated Approval of Zeleciment basivarsen in Myotonic Dystrophy Type 1 could occur in early Q3 2027.
The company ended Sep.30, 2025, with cash, cash equivalents and marketable securities of $791.9 million. Yesterday, it commenced an underwritten public offering of $300 million of shares of its common stock to further strengthen its balance sheet and pipeline development.
When we alerted readers to DYN on July 29, 2025, it was trading around $9.50. The stock closed yesterday's trading at $22.20, up 9.47%.
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