Sarepta Therapeutics Shares Rise Following Promising Three-Year Data for Elevidys in Duchenne Muscular Dystrophy

Sarepta Therapeutics Inc. (NASDAQ: SRPT) disclosed topline results on Monday from the Part 1 cohort of its EMBARK (Study SRP-9001-301) Phase 3 clinical trial, assessing the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in ambulatory individuals diagnosed with Duchenne muscular dystrophy (DMD). The data, presenting three-year follow-up outcomes, demonstrated persistent, statistically significant, and clinically meaningful efficacy across key measures of motor function. The trial compared Elevidys-treated patients to a pre-specified, propensity-weighted external control group of untreated individuals, highlighting durable benefits in motor abilities. Measures included the North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and the 10-meter walk/run (10MWR). At the three-year milestone, patients receiving Elevidys (n=52) maintained mean NSAA scores above their baseline level, contrasting with an expected decline observed in the external control group (n=73). Additionally, progression of disease-related motor decline slowed considerably in the Elevidys cohort. Specifically, the therapy reduced deterioration by 73% as measured by TTR and 70% by the 10MWR metric relative to untreated controls. The sustained higher motor function levels in the Elevidys group at three years underscore the gene therapy's ability to significantly alter the typical course of DMD. Importantly, no new safety concerns related to treatment were identified during this interval. Ongoing analyses include a subset of patients who received treatment in a crossover design, with functional data being evaluated two years post-treatment. Elevidys remains the only regulatory-approved gene therapy for Duchenne muscular dystrophy and, to date, has been administered to more than 1,200 patients worldwide across clinical trials and real-world usage. Following a label update by the U.S. Food and Drug Administration (FDA) in November 2025, Elevidys is indicated for appropriate use in ambulatory patients aged four years and older. Strategic partnerships enhance the therapy's development and accessibility. Sarepta collaborates with Roche Holdings AG under an agreement established in 2019 aiming to advance treatment options for the Duchenne community by preserving muscle function. Under this partnership, Sarepta manages Elevidys' U.S. regulatory approvals, manufacturing, and commercialization, while Roche oversees regulatory processes and distribution outside the United States. In Japan, Chugai Pharmaceuticals leads commercialization efforts. Most recently, in November 2025, the FDA authorized dosing for Cohort 8 of Sarepta’s ENDEAVOR study (Study 9001-103). This cohort explores an enhanced immunosuppressive regimen alongside Elevidys administration in non-ambulatory patients, targeting those with more advanced disease stages who have lost the ability to walk. From a financial perspective, Sarepta reported preliminary product revenues of $369.6 million for the fourth quarter and $1.86 billion for the entire year of 2025. Elevidys contributed $110.4 million in net product revenue during the fourth quarter and $898.7 million for the full year, reflecting its growing market uptake and commercial success. The positive trial findings positively influenced market sentiment. SRPT shares rose 5.06%, reaching $22.20 during Monday’s premarket trading session according to Benzinga Pro data, signaling investor confidence following the announced data. As functional outcome data accrues and expanded patient cohorts are evaluated, Sarepta continues to advance Elevidys' role in modifying the disease trajectory of Duchenne muscular dystrophy, aiming to improve quality of life and clinical outcomes for affected individuals.