DELIVER Trial Shows Significant Dystrophin Gains in DMD Patients
Dyne Therapeutics (NASDAQ:DYN) has unveiled new topline results from its Phase 1/2 DELIVER trial evaluating z-rostudirsen in Duchenne muscular dystrophy (DMD) patients, offering hope for a meaningful shift in disease management. The registrational expansion cohort (REC) met its primary endpoint, posting a 5.46% mean absolute dystrophin expression (muscle content-adjusted) at six months—statistically significant with p<0.0001. This represents a 7-fold change from baseline, aligning with prior data from earlier trial phases and showing far greater improvement than existing standards.
Functional Improvement Observed Across Multiple Clinical Endpoints
Beyond the primary biomarker, patients treated with z-rostudirsen displayed improvement across all six prespecified clinical measures. Notably, Time to Rise (TTR) Velocity and 10-Meter Walk/Run (10MWR) Velocity improved versus placebo at six months, achieving nominal p<0.05. Additionally, lung function (FVC%p), a crucial indicator in DMD, was preserved in the treatment group but declined among placebo participants.
| Key Measure | Z-Rostudirsen (6 months) | Placebo (6 months) | Significance |
|---|---|---|---|
| Dystrophin Expression (% of Normal, Adjusted) | 5.46% | N/A | p<0.0001 |
| Dystrophin Expression (Unadjusted) | 2.87% | N/A | p<0.0001 |
| TTR Velocity | Improved | Declined | p<0.05* |
| 10MWR Velocity | Improved | Declined | p<0.05* |
| Lung Function (FVC%p) | Preserved | Declined | - |
*Nominal significance; the trial was not powered for formal statistical comparison on functional endpoints.
Sustained Improvement Evident Out to 24 Months
Newly reported data from the open-label and long-term extension portions show sustained gains across all key endpoints out to 24 months. Functional improvements—measured by TTR Velocity, 10MWR Velocity, NSAA, SV95C, PUL2.0, and FVC%p—remained above baseline and relative to historical decline, strengthening the long-term value case for z-rostudirsen in DMD management.
Safety Remains Favorable Over Long-Term Follow-up
The DELIVER trial included 86 participants followed up to 36 months, with over 1,441 administered doses and 113 patient-years of exposure. Most treatment-related adverse events were mild or moderate, with no serious related adverse events observed in the REC cohort. Importantly, two long-term extension cases of fever or malaise fully resolved without interrupting treatment.
Upcoming Regulatory Milestones May Change the DMD Landscape
Dyne expects to submit a Biologics License Application (BLA) for U.S. Accelerated Approval in Q2 2026, targeting a possible launch as early as Q1 2027 if granted Priority Review. The total U.S. market for this specific genetic segment is estimated at about 1,600 patients, and Dyne is pursuing global regulatory strategies for additional reach.
| Milestone | Timeline |
|---|---|
| Accelerated Approval BLA Submission | Q2 2026 |
| Potential U.S. Launch | Q1 2027 (with Priority Review) |
| Phase 3 Global Trial Initiation | Q2 2026 |
Takeaway: Dyne Advances Toward Regulatory Filing with Robust Efficacy and Safety Profile
Dyne's latest DELIVER results put the company on a strong footing for regulatory engagement, highlighting a marked and durable improvement in both biomarker and function for DMD patients with exon 51 skipping mutations. Investors and clinicians will be closely monitoring the 2026 filing and subsequent commercial plans. For those tracking breakthroughs in rare neuromuscular disease, Dyne’s data and the planned global expansion signal a meaningful step forward in the quest for improved patient outcomes.
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