Sarepta Therapeutics Accelerates LGMD Gene Therapy Pipeline Following FDA Green Light

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Apr. 15, 2025– Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, today announced significant advancements in its clinical programs targeting limb-girdle muscular dystrophy (LGMD) subtypes 2C/R5, 2D/R3, and 2E/R4, marking a crucial step forward in addressing this debilitating condition.

SRP-9005 for LGMD type 2C/R5 (gamma-sarcoglycanopathy):

• Following productive discussions with the U.S. Food and Drug Administration (FDA) Office of Therapeutic Products (OTP), Sarepta has received clearance to initiate dosing in Study SRP-9005-101 (COMPASS) within the United States.
• COMPASS represents a first-in-human clinical study evaluating the safety and efficacy of SRP-9005, an investigational gene therapy designed to deliver a functional gamma-sarcoglycan transgene.

SRP-9004 for LGMD type 2D/R3 (alpha-sarcoglycanopathy):

• Enrollment and dosing procedures have been successfully completed for Study SRP-9004-102 (DISCOVERY), a Phase 1 proof-of-concept study.
• This study is focused on assessing the safety profile and the expression of the alpha-sarcoglycan protein following treatment with SRP-9004, another investigational gene therapy.

SRP-9003 for LGMD type 2E/R4 (beta-sarcoglycanopathy):

• The Phase 3 clinical trial, Study SRP-9003-301 (EMERGENE), has concluded enrollment and dosing.
• EMERGENE, a global study, is evaluating SRP-9003 (bidridistrogene xeboparvovec), with the primary endpoint being the measurement of beta-sarcoglycan protein expression.
• A pre-Biologics License Application (BLA) meeting has been conducted, and the OTP has confirmed the program’s eligibility for the accelerated approval pathway. Sarepta remains on schedule to submit a BLA to the U.S. FDA in the second half of 2025.

“With no disease-modifying treatments currently available for LGMD, these advancements represent significant hope for patients and their families,” said Louise Rodino-Klapac, Ph.D., Executive Vice President, Chief Scientific Officer, and Head of Research and Development at Sarepta Therapeutics. “We are particularly pleased to advance SRP-9005 into its first clinical study, and we eagerly anticipate sharing data from the EMERGENE trial in the coming months. Our confidence in the transformative potential of gene therapy remains strong.”

About Limb-Girdle Muscular Dystrophy (LGMD):

LGMD encompasses a group of genetic disorders characterized by progressive muscle weakness and wasting, initially affecting muscles around the hips and shoulders before extending to the arms and legs. With over 30 identified subtypes, each presenting unique genetic mutations and clinical manifestations, Sarepta is committed to developing targeted therapies. The company’s LGMD pipeline, including programs for LGMD 2B/R2, 2E/R4, 2D/R3, 2C/R5, and 2A/R1, addresses more than 70 percent of known LGMD cases. Sarepta also sponsors the JOURNEY natural history study, a global initiative to better understand disease progression.

About Sarepta’s Gene Therapies:

SRP-9003 (bidridistrogene xeboparvovec):

• Employs the AAVrh74 vector for systemic and robust delivery to skeletal, diaphragm, and cardiac muscle.
• Delivers a full-length beta-sarcoglycan transgene driven by the MHCK7 promoter.

SRP-9004 (patidistrogene bexoparvovec):

• Utilizes the AAVrh74 vector for broad muscle delivery.
• Delivers a full-length alpha-sarcoglycan transgene under the tMCK promoter.

SRP-9005:

• Utilizes the AAVrh74 vector.
• Delivers a full-length gamma-sarcoglycan transgene under the MHCK7 promoter.