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CAMBRIDGE, Mass. - Sarepta Therapeutics (NASDAQ:SRPT) announced Tuesday that the U.S. Food and Drug Administration has approved dosing in Cohort 8 of the ENDEAVOR study to evaluate an enhanced immunosuppressive regimen for non-ambulatory Duchenne muscular dystrophy patients receiving ELEVIDYS gene therapy. The announcement comes as the company’s stock has seen a significant 8.43% return over the past week, despite falling more than 83% over the past year.
The new cohort will enroll approximately 25 non-ambulatory participants and incorporate sirolimus into the treatment protocol. The enhanced regimen aims to reduce the risk of acute liver injury (ALI), a known complication associated with AAV gene therapy that has been particularly concerning in non-ambulatory patients. According to InvestingPro data, Sarepta maintains a strong liquidity position with a current ratio of 2.95, indicating its ability to fund important clinical studies like ENDEAVOR while meeting short-term obligations.
The immunosuppression approach will include 14 days of sirolimus before ELEVIDYS administration and continue for 12 weeks afterward. Primary endpoints include the incidence of ALI and ELEVIDYS-dystrophin expression at 12 weeks.
"We remain deeply committed to serving all individuals living with Duchenne, including those who have lost the ability to walk," said Louise Rodino-Klapac, president of research & development at Sarepta, in the press release.
The company plans to initiate the cohort by the end of 2025 with primary endpoint data collection expected in the second half of 2026. Decisions regarding resuming commercial dosing for non-ambulatory patients will be made in collaboration with the FDA after reviewing study data.
ELEVIDYS, the only approved gene therapy for Duchenne, has been administered to over 1,100 patients globally. The therapy remains available to ambulatory patients ages 4 and older following a recent label update.
The FDA had previously identified serious liver complications in non-ambulatory patients, including fatal cases of acute liver failure in the post-marketing setting, leading to restrictions on the therapy’s use in this population.
In other recent news, Sarepta Therapeutics announced significant updates regarding its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys. The U.S. Food and Drug Administration (FDA) approved new labeling for Elevidys that includes a Boxed Warning for the risk of acute serious liver injury and acute liver failure. The updated label also limits the therapy’s use to ambulatory patients aged four years and older. Despite these safety concerns, the FDA maintained its approval for Elevidys, which seemed to reassure investors. In related developments, Bernstein raised its price target for Sarepta Therapeutics to $20, citing higher estimates for Elevidys after solid third-quarter results and the recent label update. Additionally, Mizuho reaffirmed an Outperform rating on Sarepta Therapeutics with a price target of $26, despite recent investor concerns over safety warnings. Sarepta is also advancing its Phase 1/2 study of SRP-1003, a treatment for type 1 myotonic dystrophy (DM1), having completed the first two dosing cohorts and fully enrolled the third. The study will continue with higher doses following a positive safety review.
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