Sarepta Therapeutics Inc. (SRPT) is scheduled to report 3-year topline functional results from patients treated in Part 1 of the EMBARK Phase 3 study on Monday, January 26, 2026, at 8:30 am Eastern Time.
EMBARK is a randomized placebo-controlled Phase 3 study evaluating ELEVIDYS in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at the time of treatment.
Duchenne muscular dystrophy is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body's muscles. The disease occurs due to a defective gene that results in absence of dystrophin, a protein that helps keep the body's muscle cells intact.
EMBARK is a 2-part, placebo-controlled trial of 125 ambulatory people aged 4 to 7 years. In Part 1, which lasted 52 weeks, participants were randomly assigned to 2 groups: ELEVIDYS and placebo. Part 2 of the trial, which also lasted 52 weeks, reversed the groups, so those who had received ELEVIDYS in Part 1 now received a placebo, and vice versa.
On June 22, 2023, the FDA approved ELEVIDYS as the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.
Part 1 of EMBARK (Phase 3) was the confirmatory trial for the already accelerated-approved Elevidys, using the same gene therapy material for the patient population, including ambulatory boys 4-7 with DMD, but focused on proving efficacy, including the NSAA primary endpoint for full approval.
The FDA approval was then supported by results from the phase 3 EMBARK trial, in which Elevidys showed an increase in the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, but did not meet the primary endpoint. However, FDA approval was supported by secondary endpoint improvements and a consistent safety profile.
Elevidys is given as a one-time intravenous infusion, meaning patients receive a single dose through a vein, which aids the body in producing a protein called micro-dystrophin, which may strengthen muscles and improve motor function.
Elevidys should not be used if you have elevated levels of antibodies to the Elevidys vector (AAVrh74) or if you have any deletion in exon 8 and/or exon 9 in the DMD gene.
Preliminary results indicate ELEVIDYS net product revenue of $110.4 million for the fourth quarter of 2025 and $898.7 million for full-year 2025. This compares with net product revenue of $384.2 million in Q4, 2024, and $820.8 million in the full year of 2024.
Over the year, SRPT traded in a range of $10.42 and $120.05.
SRPT closed Friday's trade at $21.13, down 3.30%
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