Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
Capricor Therapeutics (NASDAQ: CAPR) received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Deramiocel, its cell therapy candidate for treating cardiomyopathy in Duchenne muscular dystrophy (DMD).
The FDA stated it cannot approve the BLA in its current form, citing insufficient evidence of effectiveness and the need for additional clinical data. Capricor plans to resubmit the BLA with data from its ongoing Phase 3 HOPE-3 trial, which includes 104 patients, with topline results expected in Q3 2025.
The company will request a Type A meeting with the FDA to discuss next steps. Deramiocel has received multiple designations including Orphan Drug, RMAT, and Rare Pediatric Disease Designation, potentially qualifying for a Priority Review Voucher upon approval.
Capricor Therapeutics (NASDAQ: CAPR) ha ricevuto una Lettera di Risposta Completa (CRL) dalla FDA riguardo alla sua Domanda di Licenza Biologica (BLA) per Deramiocel, il suo candidato per la terapia cellulare destinata al trattamento della cardiomiopatia nella distrofia muscolare di Duchenne (DMD).
La FDA ha dichiarato di non poter approvare la BLA nella sua forma attuale, citando prove insufficienti di efficacia e la necessità di ulteriori dati clinici. Capricor prevede di ripresentare la BLA con i dati del suo attuale studio HOPE-3 di Fase 3, che coinvolge 104 pazienti, con risultati preliminari attesi nel terzo trimestre del 2025.
L'azienda richiederà un incontro di tipo A con la FDA per discutere i prossimi passi. Deramiocel ha ottenuto diverse designazioni, tra cui Orphan Drug, RMAT e Rare Pediatric Disease Designation, potenzialmente qualificandosi per un Voucher di Revisione Prioritaria in caso di approvazione.
Capricor Therapeutics (NASDAQ: CAPR) recibió una Carta de Respuesta Completa (CRL) de la FDA respecto a su Solicitud de Licencia Biológica (BLA) para Deramiocel, su candidato en terapia celular para tratar la miocardiopatía en la distrofia muscular de Duchenne (DMD).
La FDA indicó que no puede aprobar la BLA en su forma actual, citando evidencia insuficiente de efectividad y la necesidad de datos clínicos adicionales. Capricor planea reenviar la BLA con datos de su ensayo Fase 3 HOPE-3 en curso, que incluye 104 pacientes, con resultados preliminares esperados para el tercer trimestre de 2025.
La compañía solicitará una reunión Tipo A con la FDA para discutir los próximos pasos. Deramiocel ha recibido varias designaciones, incluyendo Fármaco Huérfano, RMAT y Designación de Enfermedad Pediátrica Rara, lo que podría calificar para un Vale de Revisión Prioritaria tras su aprobación.
Capricor Therapeutics (NASDAQ: CAPR)는 듀센 근이영양증(DMD) 심근병증 치료를 위한 세포 치료 후보제 Deramiocel에 대한 생물학적 제제 허가 신청서(BLA)에 대해 FDA로부터 완전 반응 서한(CRL)을 받았습니다.
FDA는 현재 형태의 BLA를 승인할 수 없으며, 효과에 대한 증거가 부족하고 추가 임상 데이터가 필요하다고 밝혔습니다. Capricor는 104명의 환자가 포함된 진행 중인 3상 HOPE-3 시험 데이터를 포함하여 BLA를 재제출할 계획이며, 주요 결과는 2025년 3분기에 발표될 예정입니다.
회사는 다음 단계를 논의하기 위해 FDA와 Type A 미팅을 요청할 예정입니다. Deramiocel은 희귀의약품, RMAT, 희귀 소아 질환 지정 등 여러 지정을 받았으며, 승인 시 우선심사 바우처를 받을 자격이 있을 수 있습니다.
Capricor Therapeutics (NASDAQ : CAPR) a reçu une lettre de réponse complète (CRL) de la FDA concernant sa demande d'autorisation de mise sur le marché biologique (BLA) pour Deramiocel, son candidat en thérapie cellulaire destiné au traitement de la cardiomyopathie dans la dystrophie musculaire de Duchenne (DMD).
La FDA a indiqué qu'elle ne peut pas approuver la BLA sous sa forme actuelle, invoquant des preuves insuffisantes d'efficacité et la nécessité de données cliniques supplémentaires. Capricor prévoit de soumettre à nouveau la BLA avec les données de son essai de phase 3 HOPE-3 en cours, qui inclut 104 patients, avec des résultats principaux attendus au troisième trimestre 2025.
L'entreprise demandera une réunion de type A avec la FDA pour discuter des prochaines étapes. Deramiocel a reçu plusieurs désignations, notamment médicament orphelin, RMAT et désignation de maladie pédiatrique rare, ce qui pourrait lui permettre d'obtenir un bon de révision prioritaire en cas d'approbation.
Capricor Therapeutics (NASDAQ: CAPR) erhielt von der FDA ein vollständiges Antwortschreiben (CRL) bezüglich seines Biologika-Zulassungsantrags (BLA) für Deramiocel, seinen Zelltherapie-Kandidaten zur Behandlung der Kardiomyopathie bei Duchenne-Muskeldystrophie (DMD).
Die FDA erklärte, dass sie den BLA in der aktuellen Form nicht genehmigen kann, da die Wirksamkeitsnachweise unzureichend sind und zusätzliche klinische Daten erforderlich sind. Capricor plant, den BLA mit Daten aus der laufenden Phase-3-HOPE-3-Studie mit 104 Patienten erneut einzureichen, wobei die wichtigsten Ergebnisse im dritten Quartal 2025 erwartet werden.
Das Unternehmen wird ein Type-A-Meeting mit der FDA beantragen, um die nächsten Schritte zu besprechen. Deramiocel hat mehrere Designationen erhalten, darunter Orphan Drug, RMAT und Rare Pediatric Disease Designation, und könnte bei Zulassung für einen Priority Review Voucher in Frage kommen.
- Ongoing Phase 3 HOPE-3 trial with 104 patients could provide additional effectiveness data in Q3 2025
- Multiple regulatory designations received including Orphan Drug, RMAT, and Rare Pediatric Disease Designation
- Successful pre-licensure inspection completed before CRL
- Exclusive commercialization agreement with Nippon Shinyaku for U.S. and Japan markets
- FDA rejected BLA for Deramiocel citing insufficient evidence of effectiveness
- Additional clinical data required for approval
- Outstanding Chemistry, Manufacturing, and Controls (CMC) issues identified
- Unexpected regulatory setback delays potential market entry
Insights
FDA rejection of Capricor's DMD therapy requires additional clinical data, delaying potential approval until after Q3 2025 Phase 3 results.
The FDA has issued a Complete Response Letter (CRL) for Capricor's Biologics License Application (BLA) for Deramiocel, their lead cell therapy for treating cardiomyopathy in Duchenne muscular dystrophy (DMD). This represents a significant regulatory setback for Capricor, as the FDA determined the application doesn't meet the statutory requirement for substantial evidence of effectiveness and needs additional clinical data.
This decision is particularly concerning because Capricor's application had previously been granted Priority Review in March 2025 and had progressed through pre-licensure inspection and mid-cycle review without major issues. The company's CEO expressed surprise at the decision, stating they had followed FDA guidance throughout the development process.
Capricor's strategy to address this rejection involves submitting data from their ongoing Phase 3 HOPE-3 trial, which is expected to report topline results in Q3 2025. This randomized, double-blind, placebo-controlled trial involves 104 patients and, if positive, could provide the additional effectiveness evidence the FDA requires.
The CRL also cited Chemistry, Manufacturing, and Controls (CMC) issues, though Capricor believes they've already addressed most of these concerns in prior communications. The FDA has offered Capricor the opportunity to request a Type A meeting to discuss next steps and confirmed they will restart the review clock upon resubmission.
This regulatory setback creates a minimum delay of several months in Capricor's commercialization timeline for Deramiocel. For a company focused primarily on this lead candidate, the delay in potential revenue generation and the uncertainty around eventual approval creates significant near-term challenges, despite their continued commitment to advancing the therapy for the DMD community.
- FDA issued Complete Response Letter
- Capricor plans to resubmit its BLA to include data from the ongoing Phase 3 HOPE-3 trial in Q3 2025 to continue pursuing the indication for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy
- FDA advised Capricor to request a meeting to determine next steps toward potential approval
- Conference call and webcast scheduled for today at 8:30 a.m. ET
SAN DIEGO, July 11, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the Company’s lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
In the CRL, the FDA stated that it had completed its review of the application but is unable to approve the BLA in its current form, specifically citing that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data. The CRL also referenced certain outstanding items in the Chemistry, Manufacturing, and Controls (CMC) section of the application, most of which Capricor believes it has addressed in prior communications to the FDA. However, these materials were not reviewed by the FDA due to the timing of the CRL issuance. The FDA confirmed that it will restart the review clock upon resubmission. In addition, the agency offered the company the opportunity to request a Type A meeting to discuss the path forward. Capricor plans to engage further with the FDA to determine the appropriate next steps.
Capricor’s BLA for Deramiocel was granted Priority Review in March 2025 and was supported by data from the HOPE-2 trial, its open-label extension (OLE), and natural history comparisons from FDA-funded datasets.
“We are surprised by this decision by the FDA. We have followed their guidance throughout the process. Prior to the CRL, the review had advanced without major issues, including a successful pre-licensure inspection and completion of the mid-cycle review,” said Linda Marbán, Ph.D., CEO of Capricor. “Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness from an adequate and well-controlled study. The HOPE-3 trial is a randomized, double-blind, placebo-controlled clinical trial of 104 patients, with topline results expected in the third quarter of 2025. We believe these data, if positive, along with our existing long-term clinical results showing cardiac stabilization, preservation of skeletal muscle function, and a consistent safety profile, could support efforts to resolve the questions raised by the FDA for the treatment of cardiomyopathy associated with DMD. While this was an unexpected decision by the FDA, we remain committed to the DMD community to get Deramiocel through the approval process.”
Conference Call and Webcast
To participate in the conference call, please dial 1-800-717-1738 (Domestic) or 1-646-307-1865 (International) and reference the conference ID: 30613. Participants may dial in using the numbers above to speak with an operator or click the Call me™ link for instant access. To participate via webcast, click here to access the live stream. A replay of the webcast will be available following the conclusion of the live broadcast and will be accessible on the Company’s website.
About the HOPE-3, Phase 3 Trial
HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial consisting of two cohorts evaluating the safety and efficacy of Deramiocel in participants with DMD. Non-ambulatory and ambulatory boys who meet eligibility criteria are randomly assigned to receive either Deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months of the study. Approximately 104 eligible study subjects have been enrolled in the dual-cohort study. For more information on this study, please visit (NCT05126758).
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited.
About Deramiocel
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in dystrophiopathies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing, rather than a pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials.
Deramiocel has received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
About Capricor Therapeutics
Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, Deramiocel, an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown Deramiocel to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. Deramiocel is currently in late-stage development for the treatment of Duchenne Muscular Dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the potential that required regulatory inspections may be delayed or not be successful which would delay or prevent product approval; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission on March 26, 2025, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, as filed with the Securities and Exchange Commission on May 14, 2025. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.
Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug (IND) and is not yet approved for any indications. Neither BMD nor any of Capricor’s exosome-based candidates have been approved for clinical use.
For more information, please contact:
Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
rcona@kcsa.com
212.896.1204
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
FAQ
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