Sarepta Issues Update From Clinical Programs Focused On LGMD Subtypes 2C/R5, 2D/R3, And 2E/R4; FDA Confirmed That Screening, Dosing May Proceed In Study SRP-9005-101 For Lgmd2C/R5

–      U.S. FDA has confirmed that screening and dosing may proceed in Study SRP-9005-101 for LGMD2C/R5

–      Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3

–      Data expected for SRP-9003 for LGMD2E/R4 by mid-2025

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared updates from its clinical programs focused on limb-girdle muscular dystrophy (LGMD) subtypes 2C/R5, 2D/R3, and 2E/R4.

• SRP-9005 for LGMD type 2C/R5: Following input from the U.S. Food and Drug Administration (FDA), Office of Therapeutic Products (OTP), Sarepta is cleared to proceed with dosing in Study SRP-9005-101 (COMPASS) in the U.S. COMPASS is a first-in-human clinical study of SRP-9005, an investigational gene therapy for LGMD type 2C/R5, or gamma-sarcoglycanopathy.
• SRP-9004 for LGMD type 2D/R3: Enrollment and dosing is complete in Study SRP-9004-102 (DISCOVERY).DISCOVERY is a phase 1, proof-of-concept study evaluating safety and expression of the alpha-sarcoglycan protein after treatment with SRP-9004, an investigational gene therapy for the treatment of LGMD type 2D/R3, or alpha-sarcoglycanopathy.
• SRP-9003 for LGMD type 2E/R4: Enrollment and dosing is complete in Study SRP-9003-301 (EMERGENE). EMERGENE is a phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of LGMD type 2E/R4, or beta-sarcoglycanopathy. EMERGENE is a global study and the primary endpoint is the biomarker expression of beta-sarcoglycan protein. A pre-Biologics License Application (BLA) meeting has occurred and the OTP has confirmed eligibility for the accelerated approval pathway for the program. Sarepta remains on track to submit a BLA to the U.S. FDA in the second half of 2025.