Overview
- The REC achieved a statistically significant increase in muscle content–adjusted dystrophin to 5.46% of normal at six months versus baseline.
- Patients improved versus placebo across all six prespecified functional measures, with Time to Rise velocity and 10‑Meter Walk/Run velocity reaching nominal p<0.05 at six months, and FVC%p was preserved compared with a decline on placebo.
- Open‑label extension and long‑term extension data showed a favorable safety profile, with most related adverse events mild or moderate and pyrexia and headache most common.
- Findings replicate earlier multiple‑ascending dose results, and Dyne plans a global Phase 3 start in Q2 2026 and a U.S. accelerated approval BLA submission in Q2 2026, targeting a potential Q1 2027 launch.
- On an unadjusted basis, mean dystrophin reached 2.87% of normal with monthly dosing, which Benzinga noted is roughly tenfold the level reported for eteplirsen in a referenced trial, as Dyne shares rose about 6.8% on the announcement.