Sarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne

Core Viewpoint - Sarepta Therapeutics is advancing its ENDEAVOR study by initiating screening and enrollment for Cohort 8, which aims to evaluate an enhanced immunosuppression regimen using sirolimus in conjunction with the ELEVIDYS gene therapy for non-ambulant individuals with Duchenne muscular dystrophy [1][3]. Group 1: Study Details - Cohort 8 will enroll approximately 25 non-ambulatory participants in the U.S. to assess the effectiveness of sirolimus in reducing the risk of acute liver injury (ALI) associated with AAV gene therapy [1][2]. - The immunosuppression regimen includes 14 days of sirolimus dosing prior to the ELEVIDYS infusion and continues for 12 weeks post-infusion [2]. - Primary endpoints for the study include the incidence of ALI and the expression of ELEVIDYS-dystrophin at 12 weeks [2][4]. Group 2: Background on ELEVIDYS - ELEVIDYS (delandistrogene moxeparvovec-rokl) is the only approved gene therapy for Duchenne muscular dystrophy, targeting the underlying genetic cause by delivering a transgene for micro-dystrophin production in skeletal muscle [5][6]. - To date, ELEVIDYS has been administered to over 1,200 patients globally in both clinical and real-world settings [3]. Group 3: Safety and Efficacy Considerations - The study is informed by preclinical data and real-world clinical experiences, with input from independent specialists in Duchenne and liver health [2]. - The primary endpoint in the ENDEAVOR study is the change from baseline in the quantity of ELEVIDYS micro-dystrophin protein expression measured by western blot at 12 weeks [4][5].