Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) has received positive regulatory updates for Deramiocel, its cell therapy candidate for treating Duchenne Muscular Dystrophy (DMD)-associated cardiomyopathy. The FDA has indicated that an Advisory Committee meeting is not required, and the Biologics License Application (BLA) continues under Priority Review with a PDUFA target date of August 31, 2025.

Key developments include a successful pre-license inspection, mid-cycle review with no major issues, and an upcoming late-cycle review meeting in mid-July. Additionally, four-year data from the HOPE-2 Open-Label Extension study presented at PPMD 2025 Conference showed sustained cardiac function and clinical benefits from long-term Deramiocel treatment.

Positive

• FDA Advisory Committee meeting not required, suggesting positive review progress
• BLA maintains Priority Review status with August 31, 2025 PDUFA date
• Successful pre-license inspection and mid-cycle review completed with no major issues
• Four-year data demonstrates sustained cardiac function and clinical benefits
• Exclusive commercialization agreement secured with Nippon Shinyaku for U.S. and Japan markets

Negative

• Final FDA approval still pending and not guaranteed
• Product remains investigational and not yet approved for any indications

Insights

Biopharmaceutical Regulatory Expert

Positive FDA review progress for Deramiocel BLA suggests increasing approval likelihood for Capricor's DMD treatment.

The FDA's decision not to require an Advisory Committee meeting for Deramiocel represents a significant positive signal in the regulatory review process. This typically indicates the agency hasn't identified major concerns requiring external expert input, substantially increasing approval probability. The continued Priority Review status with the August 31, 2025 PDUFA date remaining intact further reinforces this positive trajectory.

Several additional regulatory milestones reinforce approval likelihood: the successful completion of the pre-license inspection, a mid-cycle review without major issues identified, and the upcoming late-cycle review meeting in July. This regulatory pathway is progressing precisely as expected for a successful application.

The presentation of four-year HOPE-2 Open-Label Extension data demonstrating sustained cardiac function preservation provides compelling evidence supporting Deramiocel's long-term efficacy. This represents one of the longest continuous treatment datasets in DMD, addressing the leading cause of mortality in these patients – cardiomyopathy.

Deramiocel's multiple special designations (Orphan Drug, RMAT, ATMP, Rare Pediatric Disease) provide regulatory advantages and reflect the FDA's recognition of the therapy's potential significance. The Rare Pediatric Disease designation is particularly valuable as it potentially qualifies Capricor for a Priority Review Voucher upon approval – an asset that can be worth $100+ million if sold.

The partnership with Nippon Shinyaku for commercialization in the US and Japan provides validation from an established pharmaceutical company with existing rare disease infrastructure, significantly derisking the commercialization process if approval is granted.

• FDA has indicated that an Advisory Committee meeting is not required at this time
• In-person late-cycle review meeting scheduled for mid-July
• Biologics License Application (BLA) remains under Priority Review with PDUFA target action date of August 31, 2025
• Four-year data presented at PPMD 2025 Conference demonstrate sustained cardiac function

SAN DIEGO, June 24, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the Company’s lead cell therapy candidate for the treatment of Duchenne Muscular Dystrophy (DMD)—associated cardiomyopathy.

As part of the FDA’s ongoing review, the Company has been informed that an Advisory Committee meeting is not indicated at this time. The BLA remains under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025.

“We remain confident in the strength of our submission and continue to advance toward potential approval, with our next major step being the upcoming late-cycle review meeting,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “To date, all regulatory milestones have proceeded as expected, including a successful pre-license inspection and a mid-cycle review with no major issues. Our application remains under Priority Review, and we believe we are well positioned as we move toward our PDUFA date. We continue to work closely with the FDA and remain encouraged by the progress of our review.”

Capricor recently presented four-year data from its HOPE-2 Open-Label Extension (OLE) study at the 2025 Parent Project Muscular Dystrophy (PPMD) Conference, representing one of the longest-running treatment datasets in DMD, including measures of both cardiac and skeletal muscle function. The data demonstrated continued preservation of cardiac function, along with sustained clinical benefit from long-term Deramiocel treatment.

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 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. For Becker Muscular Dystrophy (BMD), Deramiocel has also received Orphan Drug Designation from the FDA.

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 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 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; 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, 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 March 31, 2025, as filed with the Securities and Exchange Commission on May 14, 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 is an Investigational New Drug (IND) and is not yet approved for any indications. Neither BMD nor any of Capricor’s exosome-based candidates have been approved for clinical use.

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 is the PDUFA date for Capricor's (CAPR) Deramiocel BLA?

The PDUFA target action date is August 31, 2025, with the application under Priority Review.

What are the latest regulatory updates for CAPR's Deramiocel?

The FDA has indicated that an Advisory Committee meeting is not required, and a late-cycle review meeting is scheduled for mid-July 2025. The pre-license inspection and mid-cycle review were completed successfully with no major issues.

What did Capricor's 4-year HOPE-2 study data show for Deramiocel?

The four-year data demonstrated continued preservation of cardiac function and sustained clinical benefit from long-term Deramiocel treatment in DMD patients.

What is Deramiocel's target market and commercial potential?

Deramiocel targets Duchenne Muscular Dystrophy (DMD), affecting approximately 15,000 individuals in the United States. Capricor has secured exclusive commercialization rights with Nippon Shinyaku for the U.S. and Japan markets.

What regulatory designations has CAPR's Deramiocel received?

Deramiocel has received Orphan Drug Designation from both FDA and EMA, RMAT designation in the U.S., ATMP designation in Europe, and Rare Pediatric Disease Designation which may qualify for a Priority Review Voucher.