| | | Sarepta Therapeutics Shares New Protein Expression and Safety Results from ENDEAVOR in Participants 2 Years Old at Time of Treatment
BUSINESS WIRE
May-16-2025 9:01 a.m. ET
- Treatment with ELEVIDYS for Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6)
- Safety profile consistent with prior studies of ELEVIDYS and real-world experience
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (SRPT) , the leader in precision genetic medicine for rare diseases, today reported new results from Study 9001-103. Also known as ENDEAVOR, Study 9001-103 is a multi-cohort study of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of Duchenne muscular dystrophy.
Treatment with ELEVIDYS in the ENDEAVOR participants in cohort 6 who were 2 years old at the time of treatment (n=6), demonstrated mean expression of 93.87% of normal, as measured by western blot, and 79.9 percent dystrophin positive fibers (PDPF), as measured by immunofluorescence. The results were seen in biopsies taken 12 weeks after treatment. Safety in cohort 6 was consistent with clinical and real-world experience with ELEVIDYS. The most common adverse events were nausea and vomiting. Elevated liver enzymes were seen in two patients and resolved with steroid administration.
“The strength of the biomarker results that we are seeing in younger patients is extremely encouraging and we have a meeting with U.S. FDA next month to discuss expanding the ELEVIDYS label to include younger patients,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics (SRPT). “In addition to positive expression results, the safety profile in these patients is consistent with what we’ve seen in prior studies and in patients who have been prescribed treatment.”
Sarepta had previously shared expression and safety from Cohort 4 of ENDEAVOR, in which participants were 3 years old at the time of treatment. In biopsies taken 12 weeks after treatment with ELEVIDYS, participants in Cohort 4 (n=7) demonstrated mean protein levels of 99.64 percent, as measured by western blot. More than 25 patients under the age of 4 have now been treated in our clinical studies. |
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