Capricor Therapeutics Responds to FDA Posting of Complete Response Letter (CRL) for Deramiocel

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) has responded to the FDA's public posting of a Complete Response Letter (CRL) regarding their Biologics License Application (BLA) for Deramiocel, their cell therapy treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy. The company was not pre-notified of the CRL posting from July 2025 and notes that their comprehensive preliminary response was not included in the FDA's public disclosure.

CEO Linda Marbán emphasized their commitment to transparency and continued collaboration with the FDA. The company awaits Type A meeting minutes later this quarter to determine next steps. Importantly, topline HOPE-3 data is expected in Q4 2025, which could influence the timing of their BLA resubmission.

Deramiocel (CAP-1002) has received multiple designations including Orphan Drug, RMAT, ATMP, and Rare Pediatric Disease Designation. The therapy has been tested in over 250 human subjects and documented in numerous peer-reviewed publications.

Positive

• Deramiocel has multiple important regulatory designations (Orphan Drug, RMAT, ATMP, Rare Pediatric Disease)
• Extensive clinical evidence with over 250 human subjects tested
• Exclusive commercialization agreement secured with Nippon Shinyaku for US and Japan markets
• Potential to receive Priority Review Voucher upon approval

Negative

• FDA issued Complete Response Letter (CRL) rejecting initial BLA application
• Regulatory pathway remains uncertain pending Type A meeting minutes
• Additional data from HOPE-3 trial required before BLA resubmission
• Significant delay in potential market approval timeline

Insights

Biotech Regulatory Analyst

FDA's rejection of Deramiocel's BLA is a significant setback for Capricor, though the company is working to address regulatory concerns.

The FDA's public posting of a Complete Response Letter (CRL) for Capricor's Deramiocel represents a significant regulatory setback in the company's pursuit of approval for its Duchenne muscular dystrophy (DMD) therapy. A CRL is effectively the FDA's formal rejection of a Biologics License Application (BLA), indicating the therapy cannot be approved in its current form.

What's notable here is the unusual sequence of events - Capricor received the CRL in July 2025, subsequently submitted a preliminary response, but was then surprised by the FDA's unannounced public posting of only the CRL without their response. This has prompted Capricor to take the uncommon step of publishing their response document independently to ensure complete transparency.

The company is now in a critical waiting period for the minutes from their Type A meeting with the FDA. Type A meetings are specifically designated for drugs that have received a CRL and are used to discuss pathways to address deficiencies. The outcome of this meeting, expected later this quarter, will be crucial in determining the timeline and requirements for BLA resubmission.

Capricor appears to be pinning significant hopes on their upcoming HOPE-3 clinical trial data, expected in Q4 2025, suggesting that these results may address some of the FDA's concerns. For DMD patients - approximately 15,000 in the US alone - this regulatory delay is particularly significant as Deramiocel represents a potential treatment option for cardiomyopathy, which is the leading cause of death in this devastating disease.

The multiple regulatory designations Deramiocel has received (Orphan Drug, RMAT, ATMP, Rare Pediatric Disease) indicate the recognized medical need, but do not guarantee approval without satisfying the FDA's safety and efficacy requirements.

SAN DIEGO, Sept. 09, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today issued a statement regarding the U.S. Food and Drug Administration’s (FDA) public posting of its Complete Response Letter (CRL) for the Biologics License Application (BLA) for Deramiocel, the Company’s investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

The Company was not notified in advance that the CRL would be posted, but acknowledges the FDA’s decision to publish the letter, originally received in July 2025. However, the FDA did not release the comprehensive preliminary response that Capricor submitted shortly after receipt of the CRL. This written response provided clarifications to the Agency’s feedback and outlined the Company’s proposed plan to address the outstanding issues. To ensure transparency, Capricor will make its preliminary response available on the investor section of its website for patients, families, and other stakeholders to review.

“Transparency is vital in regulatory communications, especially when patients are waiting for therapies with the potential to alter the course of devastating diseases such as Duchenne muscular dystrophy,” said Linda Marbán, Ph.D., Chief Executive Officer. “Our focus remains on working closely with the FDA to resolve the outstanding issues and to advance Deramiocel toward approval. While we respect the FDA’s process, we believe it is important that the public has visibility into both the CRL and our detailed written response submitted to the Agency. We are now awaiting the official minutes from our recent Type A meeting with the FDA review team, expected to be issued later this quarter, which will help define the next steps in our regulatory pathway. Looking ahead, we expect topline HOPE-3 data in the fourth quarter of 2025, and our discussions with the FDA have centered on how these data will inform and support the timing of our BLA resubmission.”

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 afflicts approximately 15,000 individuals in the United States and primarily impacts 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 dystrophiopathies 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 a 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 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 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 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 FDA's decision on Capricor's Deramiocel BLA application?

The FDA issued a Complete Response Letter (CRL) in July 2025, effectively rejecting the initial Biologics License Application for Deramiocel in treating DMD cardiomyopathy.

When will Capricor (CAPR) release the HOPE-3 trial results for Deramiocel?

Capricor expects to release topline data from the HOPE-3 trial in the fourth quarter of 2025.

What regulatory designations has Deramiocel received from the FDA?

Deramiocel has received Orphan Drug Designation, Regenerative Medicine Advanced Therapy (RMAT) designation, and Rare Pediatric Disease Designation from the FDA.

How many patients have been treated with Deramiocel in clinical trials?

Deramiocel has been administered to over 250 human subjects across multiple clinical trials and has been investigated in more than 250 peer-reviewed scientific publications.

Who will commercialize Deramiocel in the US and Japan markets?

Capricor has entered an exclusive commercialization and distribution agreement with Nippon Shinyaku Co., Ltd. (US subsidiary: NS Pharma, Inc.) for Deramiocel in the United States and Japan, subject to regulatory approval.