Sarepta announced 3-year topline functional results from patients treated in Part One of EMBARK (Study 9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment.
Sarepta were not disclosed creatine kinase levels, the most major biomarker of Duchenne muscular dystrophy, among the 3-year data.
- At a mean age of 9 years old, ELEVIDYS-treated patients achieved mean North Star Ambulatory Assessment (NSAA) scores above baseline three years after treatment (n=52)
- ELEVIDYS gene therapy demonstrated 70% or greater reduction in the rate of decline relative to the propensity-weighted external control group, as measured by “time to rise” (TTR) and 10-meter walk/run (10MWR). ELEVIDYS-treated patients showed an increasing treatment effect over time, with the functional gap versus the external control group significantly widening between Year 2 and Year 3
- No new treatment-related safety signals were observed, consistent with the manageable safety profile observed with ELEVIDYS in ambulatory patients to-date
Read More: North Star Ambulatory Assessment (NSAA) in Duchenne
| Functional Outcomes | LSM Change Difference vs EC | p-Value |
| NSAA | +4.39 points (improvement) | p=0.0002 |
| TTR | -6.05 seconds (improvement) | p<0.0001 |
| 10MWR | -2.70 seconds (improvement) | p=0.0039 |
On January 26, 2026, Sarepta shares began trading at 22 American dolars, then rose to 24.68 American dolars following the release of three years’ worth of data on its Elevidys gene therapy. However, hours later, they fell, trading at 22.52 American dolars as of 1:04 PM Eastern Time. This decline was interpreted as a sign that investors were not satisfied with the three-year data on Elevidys gene therapy.
Learn More: Creatine Kinase (CK), Dystrophin Level and NSAA Score in Elevidys Gene Therapy
