Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy
Capricor Therapeutics (NASDAQ: CAPR) has completed its Biologics License Application (BLA) submission to the FDA for deramiocel, seeking full approval for treating Duchenne muscular dystrophy (DMD) cardiomyopathy. The submission triggers a $10 million milestone payment from Nippon Shinyaku.
The BLA is supported by cardiac data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, compared with FDA-funded natural history data. Capricor has requested priority review, which could reduce the review timeline from 10 to 6 months.
Deramiocel has received Orphan Drug Designation from both FDA and EMA, RMAT Designation in the U.S., and ATMP Designation in Europe. If approved, it would be the first therapy for DMD cardiomyopathy, and Capricor would be eligible for a Priority Review Voucher based on its rare pediatric disease designation.
Capricor Therapeutics (NASDAQ: CAPR) ha completato la sua domanda di Licenza Biologica (BLA) all'FDA per deramiocel, richiedendo l'approvazione completa per il trattamento della cardiomiopatia da distrofia muscolare di Duchenne (DMD). La sottomissione attiva un pagamento di milestone di 10 milioni di dollari da Nippon Shinyaku.
La BLA è supportata da dati cardiaci degli studi di Fase 2 HOPE-2 e HOPE-2 Open Label Extension, confrontati con i dati di storia naturale finanziati dall'FDA. Capricor ha richiesto una revisione prioritaria, che potrebbe ridurre il tempo di revisione da 10 a 6 mesi.
Deramiocel ha ricevuto la Designazione di Farmaco Orfano sia dall'FDA che dall'EMA, la Designazione RMAT negli Stati Uniti e la Designazione ATMP in Europa. Se approvato, sarebbe la prima terapia per la cardiomiopatia DMD, e Capricor sarebbe idonea per un Voucher di Revisione Prioritaria basato sulla sua designazione come malattia pediatrica rara.
Capricor Therapeutics (NASDAQ: CAPR) ha completado su Solicitud de Licencia Biológica (BLA) a la FDA para deramiocel, buscando la aprobación completa para el tratamiento de la cardiomiopatía asociada a la distrofia muscular de Duchenne (DMD). La presentación activa un pago por hitos de 10 millones de dólares de Nippon Shinyaku.
La BLA está respaldada por datos cardíacos de los ensayos de Fase 2 HOPE-2 y HOPE-2 Open Label Extension, en comparación con los datos de historia natural financiados por la FDA. Capricor ha solicitado una revisión prioritaria, lo que podría reducir el tiempo de revisión de 10 a 6 meses.
Deramiocel ha recibido la Designación de Medicamento Huérfano tanto de la FDA como de la EMA, la Designación RMAT en EE.UU. y la Designación ATMP en Europa. Si se aprueba, sería la primera terapia para la cardiomiopatía DMD, y Capricor sería elegible para un Vale de Revisión Prioritaria basado en su designación de enfermedad pediátrica rara.
Capricor Therapeutics (NASDAQ: CAPR)는 deramiocel에 대한 생물학적 라이센스 신청서(BLA)를 FDA에 제출하였으며, 뒤셴 근육 위축증(DMD) 심근병증 치료의 전체 승인을 요청하고 있습니다. 이 제출은 Nippon Shinyaku로부터 1천만 달러의 이정표 지급을 촉발합니다.
BLA는 FDA의 자연 역사 데이터와 비교된 2단계 HOPE-2 및 HOPE-2 공개 라벨 확장 시험의 심장 데이터를 기반으로 합니다. Capricor는 심사 기간을 10개월에서 6개월로 단축할 수 있는 우선 심사를 요청했습니다.
Deramiocel은 FDA와 EMA로부터 모두 고아약 지정, 미국에서 RMAT 지정, 유럽에서 ATMP 지정을 받았습니다. 승인될 경우 DMD 심근병증을 치료하는 최초의 요법이 될 것이며, Capricor는 희귀 소아 질병으로 인한 우선 심사 바우처를 받을 수 있는 자격이 있습니다.
Capricor Therapeutics (NASDAQ: CAPR) a complété sa soumission de Demande de Licence Biologique (BLA) auprès de la FDA pour deramiocel, sollicitant l'approbation complète pour le traitement de la cardiomyopathie associée à la dystrophie musculaire de Duchenne (DMD). Cette soumission déclenche un paiement d'étape de 10 millions de dollars de Nippon Shinyaku.
La BLA est soutenue par des données cardiaques provenant des essais de Phase 2 HOPE-2 et de l'extension ouverte HOPE-2, comparées aux données historiques naturelles financées par la FDA. Capricor a demandé un examen prioritaire, ce qui pourrait réduire le délai d'examen de 10 à 6 mois.
Deramiocel a reçu la désignation de médicament orphelin à la fois de la FDA et de l'EMA, la désignation RMAT aux États-Unis et la désignation ATMP en Europe. S'il est approuvé, ce serait la première thérapie pour la cardiomyopathie DMD, et Capricor serait éligible à un bon de révision prioritaire basé sur sa désignation de maladie pédiatrique rare.
Capricor Therapeutics (NASDAQ: CAPR) hat seinen Antrag auf biologische Lizenz (BLA) bei der FDA für deramiocel eingereicht, um die vollständige Genehmigung zur Behandlung der Duchenne-Muskeldystrophie (DMD) Kardiomyopathie zu beantragen. Die Einreichung löst eine Meilensteinzahlung von 10 Millionen Dollar von Nippon Shinyaku aus.
Die BLA wird durch kardiale Daten aus den Phase-2-Studien HOPE-2 und HOPE-2 Open Label Extension gestützt, die mit von der FDA finanzierten Daten zur natürlichen Geschichte verglichen wurden. Capricor hat eine vorrangige Prüfung beantragt, die den Prüfungszeitraum von 10 auf 6 Monate verkürzen könnte.
Deramiocel hat sowohl von der FDA als auch von der EMA die Orphan Drug Designation, die RMAT-Designation in den USA und die ATMP-Designation in Europa erhalten. Wenn es genehmigt wird, wäre es die erste Therapie für DMD-Kardiomyopathie, und Capricor wäre aufgrund seiner seltenen pädiatrischen Erkrankung für einen Priority Review Voucher berechtigt.
- Completion of BLA submission for deramiocel, potentially first therapy for DMD cardiomyopathy
- Immediate $10 million milestone payment from Nippon Shinyaku
- Possibility of expedited 6-month review if priority review is granted
- Potential to receive valuable Priority Review Voucher upon approval
- FDA approval not guaranteed
- Timeline for review and potential approval remains uncertain
Insights
The BLA submission for deramiocel represents a critical regulatory milestone in the DMD treatment landscape. The request for priority review, coupled with RMAT designation, positions this application for potentially expedited assessment. The supporting data from Phase 2 HOPE-2 trials compared against FDA-funded natural history data strengthens the application's scientific merit. The
The dual designations (Orphan Drug and RMAT/ATMP) provide enhanced regulatory support and market exclusivity potential. This comprehensive regulatory package, targeting DMD cardiomyopathy specifically, addresses an unmet medical need in a rare disease space with treatment options.
The focus on DMD cardiomyopathy is particularly significant as cardiac complications represent a leading cause of mortality in DMD patients. Deramiocel's development targets a critical unmet need - there are currently no FDA-approved therapies specifically for DMD-related heart problems. The multiple clinical trials demonstrating cardiac benefit suggest potential disease-modifying effects, which is rare in DMD therapeutics.
For investors unfamiliar with DMD: Think of it as a progressive muscle-wasting disease that affects about 1 in 3,500 male births. While most current treatments focus on skeletal muscle, heart failure is a major cause of death. A therapy that addresses cardiac complications could significantly impact patient survival and quality of life.
This BLA submission catalyzes multiple value-driving events for CAPR. Beyond the immediate
With a market cap of
-If approved, deramiocel would be first approved therapy for Duchenne muscular dystrophy cardiomyopathy-
-BLA submission triggers
SAN DIEGO, Jan. 02, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel, an investigational cell therapy, to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy.
“The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD. This BLA is the culmination of a body of work that has been focused on bringing this potentially transformational therapy to those patients in need,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “We believe that the strength of this application is that deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. We look forward to working with the FDA throughout the review process to support this potential approval.”
The full submission of the rolling BLA was completed as the Company had previously guided in late December 2024 and is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data from an FDA funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. Capricor has requested a priority review, which, if granted, would reduce the review timeline from the standard 10-month to a priority 6-month review from the date the submission is accepted by the FDA.
In conjunction with this achievement, Capricor will receive a milestone payment of
Deramiocel for the treatment of DMD, has received Orphan Drug Designation from the FDA and European Medicines Agency (EMA). The regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and the Advanced Therapy Medicinal Product (ATMP) Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.
About Deramiocel
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. Treatment options are limited and there is no cure.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (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 (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. 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 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, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as filed with the Securities and Exchange Commission on November 14, 2024. 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 (CAP-1002) 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 and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.
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
This press release was published by a CLEAR® Verified individual.
FAQ
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