Key insights
- Sarepta Therapeutics presented positive data for its gene therapy Elevidys in older Duchenne muscular dystrophy patients (ages 8-12) at the World Muscle Society Congress. Treated patients showed functional improvements compared to controls. Data also indicated promising dystrophin expression levels in younger patients. The safety profile remained consistent. This data supports Elevidys' potential in a broader Duchenne patient population.
CAMBRIDGE, Mass. - Sarepta Therapeutics Inc. (NASDAQ:SRPT) presented data on Wednesday showing functional outcomes for its gene therapy Elevidys in ambulatory Duchenne muscular dystrophy patients aged 8 to 12 years at the World Muscle Society Congress.
The analysis compared 25 treated patients from the EMBARK and ENDEAVOR studies with 99 patients from external control datasets. At two years, treated patients showed a 3.32-point difference in North Star Ambulatory Assessment scores compared to the control group. The company reported improvements in time to rise from floor velocity of 0.041 rise per second and 10-meter walk run velocity of 0.195 meters per second versus controls.
The data is based on a press release statement from the company.
Sarepta also presented findings on patients treated between ages 2 and 3, showing dystrophin expression levels at 12 weeks post-infusion. Six patients in ENDEAVOR Cohort 6 showed mean vector genome copies of 4.5 per nucleus and mean Western blot results of 93.9% of control. Three patients in ENVOL Cohort B showed mean vector genome copies of 5.3 per nucleus and mean Western blot results of 72.8% of control.
The company stated the safety profile remained consistent with previous observations, with nausea and vomiting being the most common treatment-related adverse events in the older patient group.
Elevidys carries a boxed warning for acute serious liver injury and acute liver failure. The therapy received U.S. approval for ambulatory Duchenne patients aged 4 years and older with confirmed DMD gene mutations. Use in patients under age 4 remains investigational.
The EMBARK study was a Phase 3 randomized, placebo-controlled trial that enrolled 125 participants aged 4 to 7 years. ENDEAVOR is an open-label Phase 1b study assessing safety and expression across multiple patient cohorts.
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