SAN DIEGO, Calif. — 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.
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.
Capricor Media Contact
Raquel Cona
KCSA Strategic Communications
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212.896.1204
Capricor Company Contact
AJ Bergmann, Chief Financial Officer
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858.727.1755