Sarepta Therapeutics Inc (NASDAQ:SRPT) shares moved higher in early trade on Monday as it reported positive topline three-year results from its Phase 3 EMBARK study evaluating its gene therapy ELEVIDYS in ambulatory patients with Duchenne muscular dystrophy.
Sarepta said patients treated with ELEVIDYS demonstrated statistically significant and clinically meaningful slowing of disease progression across key functional measures compared with an untreated external control group.
The EMBARK trial is a global, randomized, placebo-controlled Phase 3 study of ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory Duchenne patients who were aged four to seven at the time of treatment. At the time of the three-year assessment, patients were on average just over nine years old.
According to the company, patients who received ELEVIDYS maintained mean North Star Ambulatory Assessment (NSAA) scores above baseline three years after treatment. In comparison, patients in the propensity-weighted untreated external control group showed the expected age-related decline below baseline over the same period.
Sarepta reported that ELEVIDYS reduced the rate of disease progression by approximately 73% as measured by Time to Rise (TTR) and by about 70% as measured by the 10-meter walk/run (10MWR), relative to the external control group.
The company added that the treatment effect increased over time, with the functional gap between treated patients and the control group widening between year two and year three.
The three-year analysis included 52 ELEVIDYS-treated patients and 73 patients in the external control group.
Sarepta said no new treatment-related safety signals were observed, and that the safety profile remained consistent with what has previously been reported for ELEVIDYS in ambulatory patients.
“ELEVIDYS is the first gene therapy for Duchenne to show a dramatic shift in disease trajectory out to three years consistent with earlier long-term data extending up to five years,” said Dr Louise Rodino-Klapac, president of research & development and technical operations.
Jefferies analyst repeated their ‘Buy’ rating on Sarepta following the update. They have a $30 price target on the stock, which was up more than 12% at about $23 on Monday morning.
“The data could help drive real-world demand, allowing sales to rebound from $100 million in Q4 2025,” they believe.
They pointed to other 2026 stock drivers, including new data in myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy expected in the first quarter, data from an ELEVIDYS plus sirolimus combination study in non-ambulatory Duchenne patients by year-end, and a US Food and Drug Administration meeting in the first quarter regarding the company’s exon-skipping therapies Vyondys 53 and Amondys 45.