Capricor Therapeutics Announces Intent to File Biologics License Application for Full Approval of Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy
Capricor Therapeutics (NASDAQ: CAPR) has announced its intent to file a Biologics License Application (BLA) for deramiocel, a potential treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy. Key points include:
1. The BLA filing is planned to commence in October 2024, with full submission expected by year-end 2024.
2. The application will be based on existing cardiac data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, compared to natural history data.
3. The initial label would include all patients with cardiomyopathy associated with DMD.
4. Capricor plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial for potential label expansion to treat DMD skeletal muscle myopathy.
5. Deramiocel has received FDA Orphan Drug Designation and RMAT designation.
Capricor Therapeutics (NASDAQ: CAPR) ha annunciato la sua intenzione di presentare una domanda di licenza biologica (BLA) per deramiocel, un potenziale trattamento per la cardiomiopatia associata alla distrofia muscolare di Duchenne (DMD). I punti chiave includono:
1. La presentazione della BLA è prevista per ottobre 2024, con la sottomissione completa attesa entro la fine dell'anno 2024.
2. L'applicazione si baserà sui dati cardiaci esistenti degli studi di Fase 2 HOPE-2 e HOPE-2 Open Label Extension, confrontati con i dati della storia naturale.
3. L'etichetta iniziale includerebbe tutti i pazienti con cardiomiopatia associata a DMD.
4. Capricor prevede di combinare i Coorti A e B dello studio clinico di Fase 3 HOPE-3 per potenziali espansioni dell'etichetta per trattare la miopatia del muscolo scheletrico DMD.
5. Deramiocel ha ricevuto la designazione FDA di farmaco orfano e la designazione RMAT.
Capricor Therapeutics (NASDAQ: CAPR) ha anunciado su intención de presentar una Solicitud de Licencia Biológica (BLA) para deramiocel, un posible tratamiento para la cardiomiopatía asociada a la distrofia muscular de Duchenne (DMD). Los puntos clave incluyen:
1. La presentación de la BLA está prevista para comenzar en octubre de 2024, con la presentación completa esperada para fin de año 2024.
2. La solicitud se basará en datos cardíacos existentes de los ensayos de Fase 2 HOPE-2 y HOPE-2 Open Label Extension, en comparación con los datos de la historia natural.
3. La etiqueta inicial incluirá a todos los pacientes con cardiomiopatía asociada a DMD.
4. Capricor planea combinar las Cohortes A y B del ensayo clínico de Fase 3 HOPE-3 para una posible expansión de la etiqueta para tratar la miopatía del músculo esquelético DMD.
5. Deramiocel ha recibido la designación de Medicamento Huérfano de la FDA y la designación de RMAT.
Capricor Therapeutics (NASDAQ: CAPR)는 deramiocel에 대한 생물학적 제품 허가 신청(BLA)을 제출할 계획이라고 발표했습니다. 이는 뒤셴 근육형성이상(DMD)에 관련된 심근병증의 잠재적 치료법입니다. 주요 사항은 다음과 같습니다:
1. BLA 제출은 2024년 10월에 시작될 예정이며, 전체 제출은 2024년 연말까지 예상됩니다.
2. 이 신청서는 기존의 2상 HOPE-2 및 HOPE-2 개방형 라벨 연장 시험의 심장 데이터를 기반으로 하며, 자연 발생 데이터와 비교할 것입니다.
3. 초기 레이블은 DMD와 관련된 심근병증이 있는 모든 환자를 포함할 것입니다.
4. Capricor는 3상 HOPE-3 임상 시험의 A군과 B군을 결합하여 DMD에 의한 골격근 미세병을 치료하는 잠재적 레이블 확장을 계획하고 있습니다.
5. Deramiocel은 FDA의 고아약 및 RMAT 지정을 받았습니다.
Capricor Therapeutics (NASDAQ: CAPR) a annoncé son intention de soumettre une Demande de Licence Biologique (BLA) pour deramiocel, un traitement potentiel pour la cardiomyopathie liée à la dystrophie musculaire de Duchenne (DMD). Les points clés comprennent :
1. Le dépôt de la BLA est prévu pour octobre 2024, avec une soumission complète attendue d'ici la fin de l'année 2024.
2. La demande sera basée sur des données cardiaques existantes des essais de phase 2 HOPE-2 et HOPE-2 Open Label Extension, comparées aux données de l'histoire naturelle.
3. L'étiquette initiale inclura tous les patients présentant une cardiomyopathie associée à la DMD.
4. Capricor prévoit de combiner les Cohortes A et B de l'essai clinique de phase 3 HOPE-3 pour une éventuelle extension de l'étiquette pour traiter la myopathie du muscle squelettique DMD.
5. Deramiocel a reçu la désignation de Médicament Orphelin et la désignation RMAT de la FDA.
Capricor Therapeutics (NASDAQ: CAPR) hat bekannt gegeben, dass sie eine Antrag auf eine Biologics License Application (BLA) für deramiocel einreichen möchte, ein potenzielles Behandlungmittel für Herzmuskelentzündung bei Duchenne-Muskeldystrophie (DMD). Wichtigste Punkte sind:
1. Die Einreichung der BLA ist für Oktober 2024 geplant, wobei die vollständige Einreichung bis Ende 2024 erwartet wird.
2. Der Antrag wird auf bestehenden kardialen Daten aus den Phase-2-Studien HOPE-2 und HOPE-2 Open Label Extension basieren, verglichen mit Daten zur natürlichen Krankheitsgeschichte.
3. Das anfängliche Label würde alle Patienten mit der DMD-assoziierten Herzmuskelentzündung umfassen.
4. Capricor plant, die Kohorten A und B der Phase-3-Studie HOPE-3 zu kombinieren, um eine potenzielle Erweiterung des Labels zur Behandlung von DMD-Skelettmuskeldystrophie zu erreichen.
5. Deramiocel hat die FDA-Orphan-Drug-Designation und RMAT-Designation erhalten.
- Intent to file BLA for deramiocel to treat DMD cardiomyopathy
- Potential to be the first approved therapy for DMD cardiomyopathy
- BLA filing based on existing cardiac data from Phase 2 trials
- FDA Orphan Drug Designation and RMAT designation received
- Eligibility for Priority Review Voucher upon FDA marketing approval
- Internal GMP manufacturing established to support BLA and commercialization
- BLA submission not expected until October 2024
- Full approval still pending FDA review
- Potential label expansion for skeletal muscle myopathy requires additional studies
Insights
This announcement marks a significant milestone for Capricor Therapeutics. The company's intent to file a BLA for deramiocel to treat DMD cardiomyopathy is a major step towards potential commercialization. Key points to consider:
- The BLA will be based on existing cardiac data from Phase 2 trials and natural history data, targeting an initial label for all DMD cardiomyopathy patients.
- Rolling submission planned for October 2024, with full submission expected by year-end.
- No currently approved therapies for DMD cardiomyopathy, positioning deramiocel as a potential first-in-class treatment.
- Regulatory advantages include Orphan Drug Designation, RMAT designation and potential eligibility for a Priority Review Voucher.
This approach could accelerate the path to market, potentially bringing a novel treatment to patients sooner. However, investors should note that regulatory approval is not guaranteed and the company will need to demonstrate compelling efficacy and safety data to secure FDA approval.
The focus on DMD cardiomyopathy is crucial, as it's the leading cause of death in Duchenne patients. Deramiocel's potential in this area is significant:
- Multiple clinical trials have shown attenuation of cardiac implications in DMD.
- The use of natural history data for comparison strengthens the case for efficacy.
- Targeting cardiomyopathy first, with plans to expand to skeletal muscle myopathy later, is a strategic approach.
The lack of current treatments for DMD cardiomyopathy underscores the unmet medical need. If approved, deramiocel could significantly impact patient care and survival rates. However, the true long-term efficacy and safety profile will need to be closely monitored post-approval.
This development could have substantial financial implications for Capricor:
- Potential first-mover advantage in the DMD cardiomyopathy market.
- Eligibility for a Priority Review Voucher, which can be sold for
$100-$200 million or used to expedite future drug approvals. - Internal GMP manufacturing established, potentially reducing costs and improving margins.
- Market opportunity: DMD affects approximately 1 in 3,500 male births worldwide.
While the news is positive, investors should consider the costs associated with BLA filing and potential commercialization. The company's cash position and burn rate will be critical factors to watch. Additionally, the timeline for potential revenue generation extends into 2025 and beyond, assuming FDA approval.
-BLA to be Supported by Existing and Natural History Cardiac Data as Discussed with the FDA-
-Initial Label Would Include All Patients with Cardiomyopathy Associated with Duchenne Muscular Dystrophy-
-Rolling Submission Planned to Commence in October 2024-
-Internal GMP Manufacturing Established to Support BLA and Commercialization-
-Investor Webcast Today at 8:30 a.m. ET-
SAN DIEGO, Sept. 24, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, announced today, following recent meetings with the U.S. Food and Drug Administration (FDA), its intent to file a Biologics License Application (BLA) based on existing cardiac and natural history data for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy.
Following the FDA meetings:
- Capricor plans to commence the filing of a BLA in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected by year-end 2024.
- The BLA filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center.
- In order to support potential label expansion to treat DMD skeletal muscle myopathy, Capricor plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024.
“There are currently no approved therapies for DMD cardiomyopathy, which is the leading cause of death in those with Duchenne. Based on the strength of our cardiac data, combined with the FDA’s commitment to advancing therapeutics for the treatment of rare diseases, we are seeking approval for the cardiomyopathy associated with DMD and will look to expand the label for skeletal muscle myopathy post-approval,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “This approach is the result of multiple in-depth meetings with FDA where we showed robust and positive cardiac data from our HOPE-2 and HOPE-2 OLE studies compared to natural history data from a large cohort of patients.”
Dr. Marbán continued, “Deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. Based on the totality of evidence of the safety and efficacy data deramiocel has shown, we believe this is the best path forward to potential approval, allowing us to bring this novel, first-in-class treatment to patients in need in the most expeditious manner. We want to extend our appreciation to the patients, their families and advocates who continue to work with us and to the FDA for their commitment to accelerating treatments for DMD.”
Deramiocel for the treatment of DMD, has received FDA Orphan Drug Designation and the regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for deramiocel for 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.
Webcast Details
Capricor will host a conference call and webcast at 8:30 a.m. ET today to discuss these updates. To participate in the conference call, please dial 1-800-717-1738 (domestic/toll-free) or 1-646-307-1865 (international) and reference the conference ID: 62574. Participants can use guest dial-in numbers above and be answered by an operator or click here for instant telephone access. To participate via webcast, please click here to view the slides. 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 Deramiocel (CAP-1002)
Deramiocel 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. 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 advancing through Phase 3 clinical 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 June 30, 2024, as filed with the Securities and Exchange Commission on August 8, 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
FAQ
When does Capricor Therapeutics (CAPR) plan to file the BLA for deramiocel?
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