Sarepta: Sarepta Therapeutics Announces Pipeline Progress for Multiple Limb-Girdle Muscular Dystrophy Programs

- U.S. FDA has confirmed that screening and 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 - 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 - Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3 - Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3 - Data expected for SRP-9003 for LGMD2E/R4 by mid-2025 - Data expected for SRP-9003 for LGMD2E/R4 by mid-2025 - Data expected for SRP-9003 for LGMD2E/R4 by mid-2025 CAMBRIDGE, Mass. --(BUSINESS WIRE) CAMBRIDGE, Mass. --(BUSINESS WIRE) CAMBRIDGE, Mass. --(BUSINESS WIRE) 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. 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. 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. 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. 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. "There are no disease-modifying treatments approved for patients with any subtype of limb-girdle muscular dystrophy, and the unmet medical need is significant. Following feedback from U.S. FDA, we are pleased to announce that screening can proceed in Sarepta's first clinical study for a gene therapy for individuals with LGMD type 2C - the fourth LGMD program that Sarepta has advanced into the clinic," said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics. "In addition to our progress with SRP-9004 and SRP-9005, we remain on track to share data in the first half of this year from the EMERGENE study with SRP-9003. Our confidence in the potential for gene therapy to bring meaningful treatments to patients with rare, genetic based diseases remains high and the rapid progress across our LGMD pipeline is encouraging." "There are no disease-modifying treatments approved for patients with any subtype of limb-girdle muscular dystrophy, and the unmet medical need is significant. Following feedback from U.S. FDA, we are pleased to announce that screening can proceed in Sarepta's first clinical study for a gene therapy for individuals with LGMD type 2C - the fourth LGMD program that Sarepta has advanced into the clinic," said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics. "In addition to our progress with SRP-9004 and SRP-9005, we remain on track to share data in the first half of this year from the EMERGENE study with SRP-9003. Our confidence in the potential for gene therapy to bring meaningful treatments to patients with rare, genetic based diseases remains high and the rapid progress across our LGMD pipeline is encouraging." "There are no disease-modifying treatments approved for patients with any subtype of limb-girdle muscular dystrophy, and the unmet medical need is significant. Following feedback from U.S. FDA, we are pleased to announce that screening can proceed in Sarepta's first clinical study for a gene therapy for individuals with LGMD type 2C - the fourth LGMD program that Sarepta has advanced into the clinic," said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics. "In addition to our progress with SRP-9004 and SRP-9005, we remain on track to share data in the first half of this year from the EMERGENE study with SRP-9003. Our confidence in the potential for gene therapy to bring meaningful treatments to patients with rare, genetic based diseases remains high and the rapid progress across our LGMD pipeline is encouraging." About Limb-Girdle Muscular Dystrophy About Limb-Girdle Muscular Dystrophy About Limb-Girdle Muscular Dystrophy Limb-girdle muscular dystrophies (LGMD) are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. There are more than 30 distinct subtypes of LGMD, each with a unique genetic mutation and distinct symptoms, progression, and treatment approaches. Limb-girdle muscular dystrophies (LGMD) are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. There are more than 30 distinct subtypes of LGMD, each with a unique genetic mutation and distinct symptoms, progression, and treatment approaches. Limb-girdle muscular dystrophies (LGMD) are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. There are more than 30 distinct subtypes of LGMD, each with a unique genetic mutation and distinct symptoms, progression, and treatment approaches. Sarepta's leading LGMD pipeline currently has gene therapy programs in different stages of development for LGMD 2B/R2, LGMD 2E/R4, LGMD 2D/R3, LGMD 2C/R5, and LGMD 2A/R1 which together represent more than 70 percent of known LGMD cases. Sarepta is also the sponsor of JOURNEY, a natural history study evaluating disease progression for four LGMD subtypes: 2C/R5, 2D/R3, 2E/R4 and 2A/R1. JOURNEY is actively enrolling globally. Sarepta's leading LGMD pipeline currently has gene therapy programs in different stages of development for LGMD 2B/R2, LGMD 2E/R4, LGMD 2D/R3, LGMD 2C/R5, and LGMD 2A/R1 which together represent more than 70 percent of known LGMD cases. Sarepta is also the s