Sarepta Therapeutics Announces Pipeline Progress for Multiple Limb-Girdle Muscular Dystrophy Programs
Sarepta Therapeutics (NASDAQ:SRPT) announced significant progress in its limb-girdle muscular dystrophy (LGMD) pipeline programs. The FDA has cleared the company to proceed with dosing in Study SRP-9005-101 (COMPASS) for LGMD2C/R5, marking their fourth LGMD program to enter clinical trials.
The company has completed enrollment and dosing in Study SRP-9004-102 (DISCOVERY) for LGMD2D/R3. Additionally, enrollment and dosing are complete in the phase 3 EMERGENE trial of SRP-9003 for LGMD2E/R4, with data expected by mid-2025. The FDA has confirmed eligibility for accelerated approval, and Sarepta plans to submit a Biologics License Application in the second half of 2025.
These developments are significant as there are currently no disease-modifying treatments approved for any LGMD subtype. Sarepta's LGMD pipeline covers subtypes that represent over 70% of known LGMD cases.
Sarepta Therapeutics (NASDAQ:SRPT) ha annunciato progressi significativi nei suoi programmi di sviluppo per la distrofia muscolare dei cingoli (LGMD). La FDA ha autorizzato l'azienda a procedere con la somministrazione nello studio SRP-9005-101 (COMPASS) per LGMD2C/R5, segnando il quarto programma LGMD ad entrare in fase clinica.
L'azienda ha completato l'arruolamento e la somministrazione nello studio SRP-9004-102 (DISCOVERY) per LGMD2D/R3. Inoltre, l'arruolamento e la somministrazione sono terminati nella fase 3 dello studio EMERGENE su SRP-9003 per LGMD2E/R4, con dati attesi entro metà 2025. La FDA ha confermato l'idoneità per l'approvazione accelerata e Sarepta prevede di presentare una domanda di autorizzazione biologica nella seconda metà del 2025.
Questi sviluppi sono rilevanti poiché attualmente non esistono trattamenti modificanti la malattia approvati per nessun sottotipo di LGMD. La pipeline di Sarepta copre sottotipi che rappresentano oltre il 70% dei casi noti di LGMD.
Sarepta Therapeutics (NASDAQ:SRPT) anunció avances significativos en sus programas para la distrofia muscular de cinturas (LGMD). La FDA autorizó a la compañía a proceder con la dosificación en el estudio SRP-9005-101 (COMPASS) para LGMD2C/R5, marcando su cuarto programa de LGMD en entrar en ensayos clínicos.
La compañía ha completado la inscripción y dosificación en el estudio SRP-9004-102 (DISCOVERY) para LGMD2D/R3. Además, la inscripción y dosificación están completas en el ensayo de fase 3 EMERGENE de SRP-9003 para LGMD2E/R4, con datos esperados para mediados de 2025. La FDA ha confirmado la elegibilidad para una aprobación acelerada, y Sarepta planea presentar una solicitud de licencia biológica en la segunda mitad de 2025.
Estos avances son importantes ya que actualmente no existen tratamientos modificadores de la enfermedad aprobados para ningún subtipo de LGMD. La cartera de LGMD de Sarepta abarca subtipos que representan más del 70% de los casos conocidos de LGMD.
Sarepta Therapeutics (NASDAQ:SRPT)가 팔다리 근위축성 근이영양증(LGMD) 파이프라인 프로그램에서 중요한 진전을 발표했습니다. FDA는 LGMD2C/R5를 위한 SRP-9005-101 연구(COMPASS)의 투여 진행을 승인했으며, 이는 네 번째 LGMD 프로그램이 임상시험에 진입했음을 의미합니다.
회사는 LGMD2D/R3를 위한 SRP-9004-102 연구(DISCOVERY)의 등록 및 투여를 완료했습니다. 또한, LGMD2E/R4를 위한 SRP-9003의 3상 EMERGENE 시험의 등록 및 투여도 완료되었으며, 데이터는 2025년 중반에 발표될 예정입니다. FDA는 가속 승인 자격을 확인했으며, Sarepta는 2025년 하반기에 생물의약품 허가 신청을 계획하고 있습니다.
이러한 발전은 현재 어떤 LGMD 아형에 대해서도 질병을 변화시키는 치료제가 승인되지 않은 상황에서 매우 중요합니다. Sarepta의 LGMD 파이프라인은 알려진 LGMD 사례의 70% 이상을 차지하는 아형을 포함하고 있습니다.
Sarepta Therapeutics (NASDAQ:SRPT) a annoncé des progrès significatifs dans ses programmes pour la dystrophie musculaire des ceintures (LGMD). La FDA a autorisé la société à poursuivre l'administration dans l'étude SRP-9005-101 (COMPASS) pour LGMD2C/R5, marquant ainsi leur quatrième programme LGMD entrant en essais cliniques.
La société a terminé le recrutement et l'administration dans l'étude SRP-9004-102 (DISCOVERY) pour LGMD2D/R3. De plus, le recrutement et l'administration sont terminés dans l'essai de phase 3 EMERGENE de SRP-9003 pour LGMD2E/R4, avec des données attendues d'ici mi-2025. La FDA a confirmé l'éligibilité à une approbation accélérée, et Sarepta prévoit de soumettre une demande d'autorisation de mise sur le marché biologique au second semestre 2025.
Ces avancées sont importantes car il n'existe actuellement aucun traitement modificateur de la maladie approuvé pour aucun sous-type de LGMD. Le pipeline LGMD de Sarepta couvre des sous-types représentant plus de 70 % des cas connus de LGMD.
Sarepta Therapeutics (NASDAQ:SRPT) gab bedeutende Fortschritte in seinen Programmen zur Gliedergürtelmuskeldystrophie (LGMD) bekannt. Die FDA hat dem Unternehmen die Fortsetzung der Dosierung in der Studie SRP-9005-101 (COMPASS) für LGMD2C/R5 genehmigt, was das vierte LGMD-Programm markiert, das in klinische Studien eintritt.
Das Unternehmen hat die Einschreibung und Dosierung in der Studie SRP-9004-102 (DISCOVERY) für LGMD2D/R3 abgeschlossen. Außerdem sind die Einschreibung und Dosierung in der Phase-3-Studie EMERGENE von SRP-9003 für LGMD2E/R4 abgeschlossen, mit Daten, die Mitte 2025 erwartet werden. Die FDA hat die Eignung für eine beschleunigte Zulassung bestätigt, und Sarepta plant, im zweiten Halbjahr 2025 einen Antrag auf Biologika-Zulassung einzureichen.
Diese Entwicklungen sind bedeutsam, da derzeit keine krankheitsmodifizierenden Behandlungen für irgendeinen LGMD-Subtyp zugelassen sind. Sareptas LGMD-Pipeline umfasst Subtypen, die über 70 % der bekannten LGMD-Fälle ausmachen.
- FDA clearance to proceed with new LGMD2C/R5 clinical trial
- Completion of enrollment in LGMD2D/R3 study
- FDA confirmation of accelerated approval pathway eligibility for SRP-9003
- Pipeline covers 70% of known LGMD cases
- No approved disease-modifying treatments yet for any LGMD subtype
- BLA submission for SRP-9003 not expected until second half of 2025
Insights
Sarepta's announcement reflects significant regulatory and clinical progression across multiple limb-girdle muscular dystrophy (LGMD) programs, representing material positive developments for its gene therapy pipeline.
The FDA clearance to begin dosing in the SRP-9005-101 study for LGMD2C/R5 adds a fourth LGMD program to Sarepta's clinical-stage portfolio. More importantly, the regulatory confirmation that SRP-9003 (for LGMD2E/R4) qualifies for the accelerated approval pathway substantially derisks this program's timeline, potentially enabling faster commercialization through the planned BLA submission in H2 2025.
The completed enrollment and dosing in the SRP-9004-102 study for LGMD2D/R3 is similarly positive, demonstrating consistent execution across the LGMD franchise. Collectively, these programs target subtypes representing over 70 percent of known LGMD cases, positioning Sarepta to potentially dominate this therapeutic space.
This portfolio approach spanning multiple LGMD subtypes creates pipeline diversification that reduces the impact of potential clinical setbacks in any single program. With no currently approved disease-modifying treatments for LGMD, successful development would address significant unmet medical needs while establishing first-mover advantages in these rare disease markets.
While these developments don't generate immediate revenue, they enhance Sarepta's medium-term commercial prospects, particularly for SRP-9003, which appears positioned to be the company's next potential commercial product following its accelerated pathway designation.
Sarepta's LGMD gene therapy pipeline represents a comprehensive approach to these devastating rare diseases. The advancements across three distinct programs underscore the technical viability of their gene delivery platform while addressing conditions with zero approved disease-modifying options.
The FDA's confirmation of accelerated approval eligibility for SRP-9003 is particularly significant from a clinical perspective. This regulatory designation suggests the agency recognizes both the severity of beta-sarcoglycanopathy and the strength of Sarepta's preliminary data supporting beta-sarcoglycan protein expression as a valid surrogate endpoint.
Mechanistically, these therapies share a common scientific approach—utilizing AAVrh74 vectors with muscle-specific promoters (MHCK7 or tMCK) to deliver functional copies of disease-associated genes. The targeted emphasis on cardiac expression for SRP-9003 and SRP-9005 reflects understanding of the life-threatening cardiopulmonary complications in these LGMD subtypes.
Moving a fourth LGMD program (SRP-9005) into clinical testing demonstrates Sarepta's platform scalability across multiple genetic targets. Each successful program validation strengthens confidence in the company's vector delivery technology.
The natural history study JOURNEY will provide critical context for interpreting intervention results and potentially support label expansions. Completion of enrollment in the SRP-9004-102 study advances a proof-of-concept program that could validate Sarepta's approach for alpha-sarcoglycanopathy, while data expected by mid-2025 for SRP-9003 could establish the company's leadership position in LGMD therapies.
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– Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3
– Data expected for SRP-9003 for LGMD2E/R4 by mid-2025
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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 theU.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.
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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
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.
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.
About SRP-9003 (bidridistrogene xeboparvovec)
SRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy that uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat neuromuscular diseases. SRP-9003 is intended to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to robustly express in the heart1,2,3 which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.
About SRP-9004 (patidistrogene bexoparvovec)
SRP-9004 (patidistrogene bexoparvovec) is an investigational gene therapy for limb-girdle muscular dystrophy Type 2D (LGMD2D/R3). LGMD2D/R3 causes muscle weakness and primarily affects the muscles around the hips, shoulders, and thighs. SRP-9004 is engineered using the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat neuromuscular diseases. Intended to deliver a full-length alpha-sarcoglycan transgene, SRP-9004 uses the tMCK promoter, chosen for its ability to selectively express in skeletal muscle which is critically important for patients with LGMD2D/R3.
About SRP-9005
SRP-9005 is an investigational gene therapy for patients with limb-girdle muscular dystrophy Type 2C (LGMD2C/R5), also known as gamma-sarcoglycanopathy. SRP-9005 is intended to deliver a full-length gamma-sarcoglycan transgene using the AAVrh74 vector. SRP-9005 uses the MHCK7 promoter, chosen for its ability to robustly express in the heart, which is critically important for those with LGMD2C, many of whom die from pulmonary or cardiac complications.
About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.
Internet Posting of Information
We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
Forward-Looking Statements
In order to provide Sarepta’s investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,” “looks,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our future operations, business plans, market opportunities, priorities and research and development programs and technologies; the potential benefits of our technologies, scientific approaches and strategic partnerships; and expected milestones and plans, including beginning dosing in Study SRP-9005-101, sharing data from our EMERGENE study in the first half of the year, and our expectation to file a BLA for SRP-9003 in the second half of 2025.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; the expected benefits and opportunities related to our agreements with strategic partners may not be realized or may take longer to realize than expected due to a variety of reasons, including any inability of the parties to perform their commitments and obligations, challenges and uncertainties inherent in product research and development and manufacturing limitations; if the actual number of patients suffering from the diseases we aim to treat is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.
References
- Pozsgai ER, et al. Systemic AAV-Mediated b-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice. Mol. Ther. 2017 Apr 5;25(4):855-869.
- Mendell JR, et al. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;77(9):1-10.
- Salva MZ, et al. Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Mol Ther. 2007;15(2):320-329.
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Investor Contact:
Ian Estepan
617-274-4052
iestepan@sarepta.com
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Source: Sarepta Therapeutics, Inc.
FAQ
When will Sarepta (SRPT) release data from the EMERGENE study for LGMD2E/R4?
What is the timeline for Sarepta's (SRPT) BLA submission for SRP-9003?
How many LGMD subtypes does Sarepta's (SRPT) pipeline cover?
What is the status of Sarepta's (SRPT) COMPASS study for LGMD2C/R5?
