Sarepta Therapeutics to Present New Data from its Neuromuscular Portfolio at 2024 World Muscle Society Congress
Sarepta Therapeutics (NASDAQ:SRPT) will present new data from its neuromuscular portfolio at the 29th Annual Congress of the World Muscle Society 2024 (WMS 2024) in Prague, Czechia, from Oct. 8-12, 2024.
Highlights include new safety and efficacy results from several studies in the delandistrogene moxeparvovec clinical development program, including Studies 9001-101, 9001-103 (ENDEAVOR), and 9001-301 (EMBARK). Notable presentations are:
- Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy (DMD) treated with delandistrogene moxeparvovec from the EMBARK study on Oct. 12, 2024.
- Long-term safety and tolerability data from phase 1 to phase 3 clinical trials of delandistrogene moxeparvovec on Oct. 11, 2024.
Additional poster presentations cover various aspects of delandistrogene moxeparvovec's safety, efficacy, and functional outcomes over five years. The full WMS 2024 program is available at the event's website.
Sarepta Therapeutics (NASDAQ:SRPT) presenterà nuovi dati dal suo portafoglio neuromuscolare al 29° Congresso Annuale della World Muscle Society 2024 (WMS 2024) che si terrà a Praga, Repubblica Ceca, dal 8 al 12 ottobre 2024.
Tra i punti salienti ci sono nuovi risultati di sicurezza ed efficacia provenienti da diversi studi nel programma di sviluppo clinico del delandistrogene moxeparvovec, inclusi gli studi 9001-101, 9001-103 (ENDEAVOR) e 9001-301 (EMBARK). Presentazioni notevoli includono:
- Risultati della risonanza magnetica muscolare in pazienti con Distrofia Muscolare di Duchenne (DMD) trattati con delandistrogene moxeparvovec dallo studio EMBARK il 12 ottobre 2024.
- Dati di sicurezza a lungo termine e tollerabilità dagli studi clinici di fase 1 a fase 3 di delandistrogene moxeparvovec l'11 ottobre 2024.
Ulteriori presentazioni di poster coprono vari aspetti della sicurezza, dell'efficacia e degli esiti funzionali del delandistrogene moxeparvovec nell'arco di cinque anni. Il programma completo del WMS 2024 è disponibile sul sito web dell'evento.
Sarepta Therapeutics (NASDAQ:SRPT) presentará nuevos datos de su cartera neuromuscular en el 29° Congreso Anual de la World Muscle Society 2024 (WMS 2024) en Praga, República Checa, del 8 al 12 de octubre de 2024.
Los aspectos destacados incluyen nuevos resultados de seguridad y eficacia de varios estudios en el programa de desarrollo clínico de delandistrogene moxeparvovec, incluidos los estudios 9001-101, 9001-103 (ENDEAVOR) y 9001-301 (EMBARK). Las presentaciones notables son:
- Resultados de MRI muscular en pacientes con Distrofia Muscular de Duchenne (DMD) tratados con delandistrogene moxeparvovec del estudio EMBARK el 12 de octubre de 2024.
- Datos de seguridad a largo plazo y tolerabilidad de los ensayos clínicos de fase 1 a fase 3 de delandistrogene moxeparvovec el 11 de octubre de 2024.
Presentaciones adicionales en formato póster abordan varios aspectos de la seguridad, eficacia y resultados funcionales del delandistrogene moxeparvovec a lo largo de cinco años. El programa completo de WMS 2024 está disponible en el sitio web del evento.
Sarepta Therapeutics (NASDAQ:SRPT)는 2024년 10월 8일부터 12일까지 체코 프라하에서 열리는 World Muscle Society 2024 (WMS 2024) 제29회 연례 회의에서 신경근육 포트폴리오의 새로운 데이터를 발표할 예정입니다.
주요 내용으로는 delandistrogene moxeparvovec 임상 개발 프로그램의 여러 연구에서의 새로운 안전성 및 효능 결과가 포함되며, 9001-101, 9001-103 (ENDEAVOR), 9001-301 (EMBARK) 연구가 포함됩니다. 주목할 만한 발표는 다음과 같습니다:
- 두센 근이영양증(DMD) 환자에서 delandistrogene moxeparvovec로 치료된 근육 MRI 결과가 EMBARK 연구에서 2024년 10월 12일에 발표됩니다.
- 2024년 10월 11일에 delandistrogene moxeparvovec의 1상에서 3상 임상 시험까지의 장기 안전성 및 내약성 데이터가 발표됩니다.
추가적인 포스터 발표에서는 delandistrogene moxeparvovec의 안전성, 효능 및 기능적 결과에 대한 다양한 측면이 5년에 걸쳐 다루어집니다. WMS 2024의 전체 프로그램은 행사 웹사이트에서 확인할 수 있습니다.
Sarepta Therapeutics (NASDAQ:SRPT) présentera de nouvelles données de son portefeuille neuromusculaire lors du 29e Congrès Annuel de la World Muscle Society 2024 (WMS 2024) à Prague, République tchèque, du 8 au 12 octobre 2024.
Parmi les points forts, on trouve de nouveaux résultats de sécurité et d’efficacité provenant de plusieurs études dans le programme de développement clinique du delandistrogene moxeparvovec, y compris les études 9001-101, 9001-103 (ENDEAVOR) et 9001-301 (EMBARK). Les présentations notables incluent :
- Résultats de l'IRM musculaire chez des patients atteints de dystrophie musculaire de Duchenne (DMD) traités avec delandistrogene moxeparvovec de l'étude EMBARK le 12 octobre 2024.
- Données de sécurité à long terme et de tolérance des essais cliniques de phase 1 à phase 3 du delandistrogene moxeparvovec le 11 octobre 2024.
Des présentations de posters supplémentaires couvrent divers aspects de la sécurité, de l’efficacité et des résultats fonctionnels du delandistrogene moxeparvovec sur cinq ans. Le programme complet de WMS 2024 est disponible sur le site web de l'événement.
Sarepta Therapeutics (NASDAQ:SRPT) wird vom 8. bis 12. Oktober 2024 neue Daten aus seinem neuromuskulären Portfolio beim 29. Annual Congress der World Muscle Society 2024 (WMS 2024) in Prag, Tschechische Republik, präsentieren.
Highlights sind neue Sicherheits- und Wirksamkeitsresultate aus mehreren Studien im klinischen Entwicklungsprogramm von delandistrogene moxeparvovec, einschließlich der Studien 9001-101, 9001-103 (ENDEAVOR) und 9001-301 (EMBARK). Bemerkenswerte Präsentationen sind:
- Muskel-MRT-Ergebnisse bei Patienten mit Duchenne-Muskeldystrophie (DMD), die im EMBARK-Studie mit delandistrogene moxeparvovec behandelt wurden, am 12. Oktober 2024.
- Daten zur Langzeit-Sicherheit und Verträglichkeit aus klinischen Studien der Phasen 1 bis 3 von delandistrogene moxeparvovec am 11. Oktober 2024.
Zusätzliche Posterpräsentationen behandeln verschiedene Aspekte der Sicherheit, Wirksamkeit und funktionalen Ergebnisse von delandistrogene moxeparvovec über fünf Jahre. Das vollständige WMS 2024 Programm ist auf der Website der Veranstaltung verfügbar.
- None.
- None.
Among the data to be presented are new safety and efficacy results from several studies in the delandistrogene moxeparvovec clinical development program, including data from Studies 9001-101, 9001-103 (ENDEAVOR) and 9001-301 (EMBARK).
“New data to be presented at WMS from clinical studies of delandistrogene moxeparvovec include the first look at skeletal muscle and cardiac MRI outcomes from the EMBARK study, a late-breaking analysis of safety data from across all delandistrogene moxeparvovec clinical studies, and five-year functional results from Study SRP-9001-101, the longest-term data to date for a gene therapy in Duchenne,” said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics. “The results add to the substantial and growing body of data for delandistrogene moxeparvovec.”
Podium Presentation:
19O: Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 |
Oct. 12, 2024 1:45-2:00 a.m. EDT (07:45-08:00 CEST) |
Late Breaking Poster:
726LBP: Long-term safety and tolerability of delandistrogene moxeparvovec in Duchenne muscular dystrophy: phase 1 to phase 3 clinical trials |
Oct. 11, 2024 9:45-10:45 a.m. EDT (15:45-16:45 CEST) |
Poster Presentations (*Denotes encore presentation)
391P: Agreement and accuracy of ambulatory definitions in Duchenne muscular dystrophy (DMD): a cross-sectional analysis* |
Oct. 9, 2024 8:30-9:30 a.m. EDT (14:30-15:30 CEST) |
94P: JOURNEY: a natural history study of Limb Girdle muscular dystrophies R3-R5: baseline characteristics of study cohort |
Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
423P: Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy: Phase 3 EMBARK primary results* |
Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
424P: Micro-dystrophin expression and safety with delandistrogene moxeparvovec gene therapy for DMD in a broad population: Phase 1B trial (ENDEAVOR) |
Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
425P: Five-year outcomes with delandistrogene moxeparvovec in patients with Duchenne Muscular dystrophy (DMD): a phase 1/2a study |
Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
428P: Cardiac MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 |
Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
The full WMS 2024 program is available at https://www.wms2024.com/page/programme.
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 (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on LinkedIn, X (formerly Twitter), 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
This press release contains forward-looking statements. 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," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements related to our research and development programs, technologies, scientific approaches, and product and product candidates, including potential benefits related to safety and efficacy, and our mission.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: Sarepta may not be able to execute on its business plans, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its products to markets for various reasons 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 Sarepta’s product candidates; 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; different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2023, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.
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Investor Contact:
Ian Estepan, 617-274-4052
iestepan@sarepta.com
Media Contact:
Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com
Source: Sarepta Therapeutics, Inc.
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