Overview
- In Part 1 of the Phase 3 EMBARK trial, Elevidys produced statistically significant, clinically meaningful, durable gains across NSAA, Time to Rise, and 10‑meter walk/run versus a propensity‑weighted external control.
- At Year 3, mean NSAA in Elevidys‑treated boys (n=52) remained above baseline while the control cohort (n=73) declined below baseline.
- Disease progression slowed 73% by Time to Rise and 70% by 10‑meter walk/run compared with the control group, and no new treatment‑related safety signals were seen.
- The company said analyses continue, including outcomes two years after crossover dosing, and noted Elevidys has been given to more than 1,200 patients in clinical and real‑world use.
- Elevidys remains available only to ambulatory patients under an FDA label that followed two deaths under investigation, and Sarepta shares rose about 5% premarket as the 8:30 am ET webcast began.