- Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the ENDEAVOR study

- The enhanced immunosuppressive regimen is designed to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF) associated with AAV gene therapy in non-ambulatory patients

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced screening and enrollment are underway in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to assess prophylactic sirolimus treatment as part of an enhanced safety protocol during treatment with ELEVIDYS (delandistrogene moxeparvovec-rokl) in non-ambulant individuals with Duchenne muscular dystrophy.

Data from Cohort 8 will be used to determine whether administering sirolimus prior to and after ELEVIDYS infusion can help reduce acute liver injury (ALI), a known risk associated with AAV gene therapy. The cohort is enrolling approximately 25 participants in the U.S. who are non-ambulatory. The immunosuppression regimen will include 14 days of peri-infusion sirolimus dosing (prior to ELEVIDYS administration) and will continue for 12 weeks after ELEVIDYS administration. Primary endpoints include incidence of ALI and ELEVIDYS-dystrophin expression at 12 weeks. The approach is based on preclinical data and shaped by real-world clinical experience, including guidance from independent specialists in Duchenne and liver health.