Capricor Therapeutics Provides Regulatory Update on Deramiocel Program for Duchenne Muscular Dystrophy Following Type A Meeting
Rhea-AI Summary
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.
Positive
- 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
Negative
- 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
News Market Reaction
On the day this news was published, CAPR gained 5.33%, reflecting a notable positive market reaction. Argus tracked a peak move of +26.3% during that session. Our momentum scanner triggered 31 alerts that day, indicating elevated trading interest and price volatility. This price movement added approximately $17M to the company's valuation, bringing the market cap to $334M at that time. Trading volume was elevated at 2.0x the daily average, suggesting notable buying interest.
Data tracked by StockTitan Argus on the day of publication.
- 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?
