Capricor Therapeutics Provides Regulatory Update on Deramiocel Program for Duchenne Muscular Dystrophy Following Type A Meeting
Capricor Therapeutics (NASDAQ: CAPR) provided a regulatory update following a Type A meeting with the FDA regarding its Biologics License Application (BLA) for Deramiocel, a cell therapy treatment for Duchenne Muscular Dystrophy (DMD). The FDA agreed that the completed HOPE-3 pivotal trial can serve as the additional study requested in the July 2025 Complete Response Letter (CRL).
Key developments include: The FDA will maintain PUL v2.0 as primary efficacy endpoint and consider left ventricular ejection fraction (LVEF) as key secondary endpoint. Topline data from HOPE-3, which enrolled 105 subjects, is expected in mid-Q4 2025. The company plans to submit this data within the current BLA, aiming for a label covering both cardiac and skeletal muscle function in DMD.
Prior to the CRL, most of the BLA had undergone review with no significant deficiencies identified, and all CMC items have been addressed. The FDA expressed commitment to regulatory flexibility in reviewing HOPE-3 trial data.
Capricor Therapeutics (NASDAQ: CAPR) ha fornito un aggiornamento regolatorio a seguito di un incontro di Tipo A con la FDA riguardo la sua Biologics License Application (BLA) per Deramiocel, una terapia cellulare per la Distrofia Muscolare di Duchenne (DMD). La FDA ha concordato che lo studio chiuso HOPE-3, pilota, può servire come lo studio aggiuntivo richiesto nella Lettera di Risposta Completa (CRL) di luglio 2025.
I principali sviluppi includono: la FDA manterrà PUL v2.0 come endpoint di efficacia primario e considererà la frazione di eiezione del ventricolo sinistro (LVEF) come principale endpoint secondario. I dati principali di HOPE-3, che ha reclutato 105 soggetti, sono attesi a metà del IV trimestre 2025. L’azienda intende presentare tali dati all’interno della BLA attuale, con l’obiettivo di ottenere un’etichetta che copra sia la funzione cardiaca che quella dei muscoli scheletrici nella DMD.
Prima della CRL, la maggior parte della BLA era stata esaminata senza deficienze significative, e tutti gli elementi CMC sono stati affrontati. La FDA ha espresso impegno per una flessibilità regolatoria nella revisione dei dati del trial HOPE-3.
Capricor Therapeutics (NASDAQ: CAPR) proporcionó una actualización regulatoria tras una reunión de Tipo A con la FDA respecto a su Biologics License Application (BLA) para Deramiocel, una terapia celular para la Distrofia Muscular de Duchenne (DMD). La FDA acordó que el ensayo pivotal HOPE-3 completado puede servir como el estudio adicional solicitado en la Carta de Respuesta Completa (CRL) de julio de 2025.
Los desarrollos clave incluyen: la FDA mantendrá PUL v2.0 como endpoint de eficacia primario y considerará la fracción de eyección del ventrículo izquierdo (LVEF) como endpoint secundario clave. Los datos iniciales de HOPE-3, que inscribió a 105 sujetos, se esperan a mediados del cuarto trimestre de 2025. La empresa planea presentar estos datos dentro de la BLA actual, con el objetivo de una etiqueta que cubra tanto la función cardíaca como la muscular esquelética en DMD.
Antes de la CRL, la mayor parte de la BLA había sido revisada sin deficiencias significativas, y todos los elementos CMC han sido abordados. La FDA expresó compromiso con la flexibilidad regulatoria al revisar los datos del ensayo HOPE-3.
Capricor Therapeutics (NASDAQ: CAPR)는 FDA와의 Type A 회의에 따른 규제 업데이트를 제공했습니다. 이는 Duchenne 근이영양증(DMD)을 위한 세포 치료제 Deramiocel의 생물학적 제제 라이선스 신청(BLA)에 관한 것입니다. FDA는 완료된 HOPE-3 결정적 시험이 2025년 7월 CRL에서 요청된 추가 연구로서 활용될 수 있다고 동의했습니다.
주요 개발 내용은 다음과 같습니다: FDA는 PUL v2.0을 1차 유효성 평가 지표로 유지하고 좌심실 박출분율(LVEF)을 주요 2차 지표로 간주합니다. HOPE-3의 상위 데이터는 105명의 피험자를 모집했으며 2025년 4분기 중반에 발표될 것으로 예상됩니다. 회사는 이 데이터를 현재 BLA에 제출하여 DMD에서 심장 및 골격근 기능을 모두 포함하는 라벨을 목표로 합니다.
CRL 이전에 BLA의 대부분은 검토되었으며 주요 결함은 확인되지 않았고, 모든 CMC 항목이 해결되었습니다. FDA는 HOPE-3 시험 데이터를 검토하는 데 있어 규제적 융통성에 대한 약속을 표명했습니다.
Capricor Therapeutics (NASDAQ: CAPR) a fourni une mise à jour réglementaire à la suite d’une réunion de type A avec la FDA concernant sa Biologics License Application (BLA) pour Deramiocel, une thérapie cellulaire pour la dystrophie musculaire de Duchenne (DMD). La FDA a convenu que l’essai pivot HOPE-3 complété peut servir d’étude additionnelle demandée dans la lettre de réponse complète (CRL) de juillet 2025.
Les développements clés incluent : la FDA maintiendra PUL v2.0 comme endpoint d’efficacité primaire et considèrera la fraction d’éjection du ventricule gauche (LVEF) comme endpoint secondaire clé. Les données préliminaires de HOPE-3, qui a recruté 105 sujets, devraient être publiées au milieu du quatrième trimestre 2025. L’entreprise prévoit de soumettre ces données dans le cadre de la BLA actuelle, dans le but d’obtenir un étiquetage couvrant à la fois la fonction cardiaque et les muscles squelettiques dans la DMD.
Avant la CRL, la majorité de la BLA avait été examinée sans déficiences significatives, et tous les éléments CMC ont été traités. La FDA a exprimé son engagement envers une flexibilité réglementaire lors de l’examen des données de l’essai HOPE-3.
Capricor Therapeutics (NASDAQ: CAPR) hat nach einem Type-A-Meeting mit der FDA ein Regulierung-Update bezüglich seines Biologics License Application (BLA) für Deramiocel, eine Zelltherapie zur Duchenne-Muskeldystrophie (DMD), gegeben. Die FDA stimmte zu, dass die abgeschlossene HOPE-3-Pivotal-Studie als die in der Juli-2025-CRL geforderte zusätzliche Studie dienen kann.
Zu den wichtigsten Entwicklungen gehört: Die FDA wird PUL v2.0 als primären Wirksamkeitsendpunkt beibehalten und die linke Ventricular Ejection Fraction (LVEF) als Schlüssel-Sekundärendpunkt in Betracht ziehen. Die Topline-Daten von HOPE-3, an der 105 Probanden beteiligt waren, werden voraussichtlich Mitte Q4 2025 veröffentlicht. Das Unternehmen plant, diese Daten innerhalb der aktuellen BLA einzureichen, mit dem Ziel einer Etikettierung, die sowohl Kardio- als auch Skelettmuskel-Funktion bei DMD abdeckt.
Vor der CRL war der Großteil der BLA geprüft worden, ohne nennenswerte Mängel, und alle CMC-Positionen wurden adressiert. Die FDA äußerte sich zu regulatorischer Flexibilität bei der Prüfung der HOPE-3-Daten.
Capricor Therapeutics (NASDAQ: CAPR) قدمت تحديثاً تنظيمياً عقب اجتماع من النوع A مع هيئة الغذاء والدواء الأمريكية بخصوص طلب ترخيص بيولوجي (BLA) لـ Deramiocel، علاجي بالخلايا لـمرض الضمور العضلي الدوشيني (DMD). وافقت FDA على أن التجربة المحورية HOPE-3 المكتملة يمكن أن تكون الدراسة الإضافية المطلوبة في رسالة الاستجابة الشاملة (CRL) المؤرخة يوليو/تموز 2025.
التطورات الرئيسية تشمل: ستحتفظ FDA بنقطة النهاية الفعّالية الأساسية PUL v2.0 وستعتبر نسبة إخراج البطين الأيسر (LVEF) كنقطة نهاية ثانوية رئيسية. من المتوقع أن تكون البيانات الأولية من HOPE-3، التي شارك فيها 105 مشاركاً، متاحة في منتصف الربع الرابع من 2025. تخطط الشركة لتقديم هذه البيانات ضمن BLA الحالي، بهدف تسمية تغطي وظيفة القلب والعضلات الهيكلية في DMD.
قبل CRL، كان معظم BLA قد خضع للمراجعة دون عيوب كبيرة، وتم معالجة جميع عناصر CMC. عبرت FDA عن التزامها بالمرونة التنظيمية عند مراجعة بيانات تجربة HOPE-3.
Capricor Therapeutics（股票代码：CAPR） 在与FDA举行的Type A会议后，就其用于Deramiocel的生物制品许可申请（BLA）提供了监管更新——这是一种用于杜氏肌营养不良症（DMD）的细胞治疗。FDA 同意完成的 HOPE-3 关键试验可以作为2025年7月的 CRL（完整回复信）中所要求的额外研究。
主要进展包括：FDA 将把 PUL v2.0 维持为主要有效性终点，并将 左心室射血分数（LVEF） 视为关键的次要终点。招募了 105 名受试者 的 HOPE-3 的初步数据预计在 2025 年第四季度中期公布。公司计划在当前的 BLA 内提交这些数据，目标是在标签中覆盖 DMD 的心脏和骨骼肌功能。
在 CRL 之前，大部分 BLA 已经过审查，未发现重大缺陷，所有 CMC 项目均已解决。FDA 表示在审查 HOPE-3 试验数据时，将保持监管灵活性。
- FDA agreement to accept HOPE-3 data within current BLA submission
- FDA's commitment to regulatory flexibility in reviewing HOPE-3 trial data
- Previous BLA review showed no significant deficiencies during mid-cycle review
- All CMC items from CRL have been addressed
- Strong financial position maintained for potential product launch
- Complete Response Letter (CRL) received in July 2025 requiring additional study
- Final approval contingent on positive HOPE-3 trial results
- Regulatory timeline extended due to need for additional data submission
Insights
FDA alignment on HOPE-3 trial endpoints positions Capricor's Deramiocel for potential approval despite prior setback.
The FDA's constructive stance following Capricor's Type A meeting represents a significant regulatory milestone for Deramiocel in DMD treatment. The agency's agreement that the HOPE-3 trial can serve as the "additional study" requested in July's Complete Response Letter (CRL) is particularly noteworthy, as it allows Capricor to maintain its current BLA rather than requiring a completely new submission – potentially saving months in regulatory review time.
The alignment on endpoints is strategically important: PUL v2.0 (Performance of Upper Limb) remains the primary efficacy endpoint while LVEF (left ventricular ejection fraction) is positioned as a key secondary endpoint for labeling consideration. This dual focus could enable a differentiated label addressing both skeletal and cardiac muscle function – crucial for DMD where cardiomyopathy is the leading cause of death.
The FDA's stated commitment to "exercise further regulatory flexibility" signals unusual regulatory accommodation, suggesting the agency recognizes both the unmet need in DMD and the potential benefit of Deramiocel. Most critically, the company reports that the majority of the BLA had already undergone review with "no significant deficiencies" identified during mid-cycle review or pre-licensing inspections, and that all Chemistry, Manufacturing and Controls (CMC) issues from the CRL have been addressed – removing major potential approval barriers.
With HOPE-3 already completed and data expected in Q4 2025, Capricor appears well-positioned for a potential approval, assuming positive trial results.
HOPE-3 trial completion marks critical advancement for Deramiocel's dual-action DMD therapy with near-term data readout.
The completed enrollment of 105 subjects in the HOPE-3 pivotal trial represents a crucial inflection point in Deramiocel's development program. This dual-cohort study design, which includes both ambulatory and non-ambulatory DMD patients, addresses the full disease spectrum – an important consideration for a progressive disorder like DMD where therapeutic options drastically diminish as patients lose ambulation.
The trial's 3-month dosing interval (for a total of four doses over 12 months) aligns with previous HOPE-2 studies that demonstrated clinically meaningful and statistically significant benefits in both cardiac and skeletal muscle function. This dosing regimen strikes a balance between treatment burden and sustained therapeutic effect.
Mechanistically, Deramiocel's approach is distinct from other DMD therapies. Rather than attempting to restore dystrophin (like exon-skipping therapies) or mitigate inflammation (like corticosteroids), these cardiosphere-derived cells (CDCs) act through immunomodulatory and anti-fibrotic mechanisms via secreted exosomes. This approach directly targets macrophages to promote a healing phenotype rather than pro-inflammatory activity – addressing core disease pathophysiology.
The use of PUL v2.0 as the primary endpoint is appropriate for a population that includes non-ambulatory patients, while the inclusion of LVEF as a key secondary endpoint addresses the critical cardiac component of DMD that ultimately determines mortality. With data expected in Q4 2025, this readout will be definitive for Deramiocel's future as a potentially first-in-class cell therapy for DMD.
- FDA and Capricor aligned on endpoints for HOPE-3 pivotal trial
- HOPE-3 pivotal trial completed; topline data expected mid-Q4 2025 to support BLA resubmission
- Company preparing to resubmit CRL response under the current BLA
- Conference call and webcast scheduled for today at 8:30 a.m. ET
SAN DIEGO, Sept. 25, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced a regulatory update for its Biologics License Application (BLA) for Deramiocel, the Company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD). This update follows a recent Type A meeting with the U.S. Food and Drug Administration (FDA) after the receipt of a Complete Response Letter (CRL) in July 2025.
The goal of the Type A meeting was to establish a path toward potential approval of Deramiocel for the treatment of DMD. Key outcomes included:
- The HOPE-3 clinical trial should serve as the “additional study” requested in the CRL.
- The HOPE-3 data can be submitted within the current BLA, maintaining PUL v2.0 as the primary efficacy endpoint and suggesting left ventricular ejection fraction (LVEF) as a key secondary endpoint, which Capricor intends to request for labeling consideration.
- Capricor plans to submit HOPE-3 data with its complete response to the CRL, with the goal of securing a label encompassing both cardiac and skeletal muscle function in DMD. In its meeting minutes, the FDA further emphasized its commitment, stating: “The FDA remains committed to collaborating with the applicant and will exercise further regulatory flexibility by reviewing data from the HOPE-3 trial.”
“We are encouraged by the outcome of our discussions with the FDA, which provided clarity on our regulatory strategy and reinforced the opportunity to deliver HOPE-3 data as the basis for approval, should it meet regulatory requirements,” said Linda Marbán, Ph.D., Capricor’s Chief Executive Officer. “The results from the HOPE-2 and HOPE-2-OLE studies have already demonstrated clinically meaningful and statistically significant benefits in both cardiac and skeletal muscle function, and HOPE-3 is designed to further validate these findings in an adequate and well-controlled study. With HOPE-3 completed and data expected later this year, we remain confident in our ability to advance Deramiocel toward potential approval. Above all, our mission remains unchanged: to bring this therapy to patients and families living with Duchenne as quickly as possible.”
Importantly, prior to issuance of the CRL, the majority of the BLA had undergone rigorous review with no significant deficiencies identified by the FDA during the mid-cycle review or pre-licensing inspections. All CMC items identified in the CRL have been addressed and communicated to the FDA. Capricor believes that the addition of HOPE-3 data will further strengthen the clinical package and support the broad potential of Deramiocel as a treatment for DMD.
The Company also maintains a strong financial position to support the advancement of Deramiocel through regulatory review and toward potential launch.
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: 96317. 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 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, primarily 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 muscular dystrophies 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 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 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 four doses during the first 12 months of the trial. A total of 105 eligible subjects have been enrolled in the dual-cohort trial. For more information, please visit ClinicalTrials.gov (NCT05126758).
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 with potential to treat and prevent a diverse array of diseases. At Capricor, we are 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 clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including future interactions with regulatory authorities and the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; 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; 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 June 30, 2025, as filed with the Securities and Exchange Commission on August 11, 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 and the StealthX™ vaccine are investigational candidates and have not been approved for commercial use in any indication.
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
What was the outcome of Capricor's (CAPR) Type A meeting with the FDA for Deramiocel?
When will Capricor (CAPR) release HOPE-3 trial results for Deramiocel?
How many patients were enrolled in Capricor's HOPE-3 trial for DMD?
What regulatory designations has Deramiocel received for DMD treatment?
What is the market potential for Deramiocel in treating DMD?
