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.

“We are proud to announce that clinical trial sites are now open and actively recruiting non-ambulatory individuals with Duchenne to participate in ENDEAVOR Cohort 8,” said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta. “Individuals with non-ambulatory Duchenne face profound unmet need and fewer treatment options. Cohort 8 of ENDEAVOR is expected to build on the dystrophin expression data generated with ELEVIDYS to-date while deepening our understanding of its safety profile in older patients with more advanced disease so we can urgently advance this treatment option for them.”

Additional information can be found on clinicaltrials.gov (NCT04626674). ELEVIDYS is the only approved gene therapy for Duchenne. To date, ELEVIDYS has been administered to over 1,200 patients globally in clinical and real-world settings.