Two Clinical Wins Ahead: Dyne's Path to DMD and Myotonic Dystrophy Approvals Looking Stronger

Dyne Therapeutics is making serious progress on its pipeline, with two major programs moving toward pivotal moments. The company’s lead candidate, Zeleciment rostudirsen (DYNE-251), just showed what could be a game-changer in Duchenne muscular dystrophy (DMD).

DELIVER Trial Results: Dystrophin Production at Scale

In the Phase 1/2 DELIVER trial, Zeleciment rostudirsen—designed for DMD patients with exon 51-skipping mutations—demonstrated measurable dystrophin production at 5.46% after six months in the Registrational Expansion Cohort. What’s more important: functional improvements held up across multiple clinical endpoints, and crucially, lung function remained stable through 24 months of follow-up. The safety profile stayed consistent throughout.

Here’s what this means commercially: Dyne plans to file a Biologics License Application (BLA) for U.S. Accelerated Approval in Q2 2026. If the FDA grants Priority Review status (as hoped), the drug could reach patients by Q1 2027. That’s a realistic regulatory timeline assuming no major setbacks.

Simultaneously, the company is prepping a global Phase 3 trial to support international approvals, keeping momentum on the DMD front.

ACHIEVE Trial Advancing Myotonic Dystrophy Program

While DELIVER dominates headlines, Dyne’s second program deserves equal attention. Zeleciment basivarsen (DYNE-101) is in a Phase I/II global trial for myotonic dystrophy type 1, branded ACHIEVE. The trial’s Registrational Expansion Cohort is currently enrolling, with patient recruitment expected to wrap up in early Q2 2026.

The primary endpoint—video hand opening time (vHOT)—will serve as an intermediate clinical measure. If results support efficacy, a BLA for myotonic dystrophy type 1 could follow in early Q3 2027, creating a potential dual-approval narrative for the company over the next 18 months.

Balance Sheet Muscle

As of September 30, 2025, Dyne held $791.9 million in cash and marketable securities. Yesterday, the company kicked off a $300 million underwritten stock offering to fortify its runway and accelerate pipeline development—a move that signals confidence in near-term catalysts.

The stock reflects this momentum: since July 29, 2025, when it traded around $9.50, DYN closed yesterday at $22.20, up 9.47%. Whether that can sustain depends entirely on clinical and regulatory execution in 2026-2027.