Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy
- FDA found no significant deficiencies in the BLA review
- Company remains on track for PDUFA date of August 31, 2025
- Deramiocel has received multiple important designations (Orphan Drug, RMAT, ATMP)
- Company would receive Priority Review Voucher upon approval
- Exclusive commercialization agreement already secured with Nippon Shinyaku for US and Japan markets
- Advisory committee meeting date not yet set
- Efficacy data from ongoing HOPE-3 study not included in BLA package
- Product still requires regulatory approval with no guarantee of success
Insights
Capricor's mid-cycle FDA review without deficiencies significantly increases approval probability for its DMD cardiomyopathy therapy, with advisory committee evaluation pending.
The completed FDA mid-cycle review for deramiocel represents a meaningful milestone in the regulatory pathway. The absence of "significant deficiencies" at this critical halfway point substantially reduces regulatory risk, as this meeting typically reveals major concerns that could prevent approval. While not guaranteeing success, this positive signal increases the probability of a favorable outcome at the August 31, 2025 PDUFA date.
The scheduled advisory committee meeting is standard protocol for novel cellular therapies and first-in-class treatments like deramiocel. These committees evaluate benefit-risk profiles with recommendations that carry substantial weight with the FDA. Capricor's proactive preparation for this scrutiny is strategically sound, as these meetings often focus intensely on efficacy evidence and safety considerations.
Particularly noteworthy is the BLA's reliance on Phase 2 HOPE-2 trial data compared against an FDA-funded natural history dataset rather than a traditional randomized control arm. This approach has gained increasing acceptance for rare diseases like DMD where placebo-controlled trials present ethical challenges due to the progressive and fatal nature of the disease.
The multiple regulatory designations secured (Orphan Drug, RMAT in US, ATMP in Europe) provide advantages including enhanced agency interactions and potentially expedited review. The eligibility for a Priority Review Voucher upon approval represents a valuable additional asset independent of the therapy's market potential.
Deramiocel targets DMD cardiomyopathy—now the leading cause of mortality—with a novel immunomodulatory approach that could address a critical treatment gap.
Duchenne muscular dystrophy cardiomyopathy represents a critical unmet need within this devastating rare disease. As respiratory management has improved DMD patient longevity over recent decades, cardiac complications have emerged as the predominant cause of mortality. The median survival age of approximately 30 years mentioned reflects this shift, with heart failure now being the leading cause of death among DMD patients.
Current DMD treatments primarily target skeletal muscle manifestations, with few options specifically addressing cardiac involvement. This creates a significant treatment gap that deramiocel aims to fill. The patient population of 15,000-20,000 in the U.S. represents a concentrated group with limited therapeutic options.
Deramiocel's mechanism using allogeneic cardiosphere-derived cells (CDCs) represents a novel approach distinct from other DMD therapies under development. Rather than attempting to restore dystrophin expression, these cells secrete exosomes that modify macrophage phenotype from pro-inflammatory to reparative, potentially slowing fibrotic progression in cardiac tissue.
The extensive research foundation (200+ peer-reviewed publications) and clinical experience (250+ human subjects) provides substantial scientific validation. For the DMD community, a therapy specifically targeting cardiomyopathy could significantly impact disease management and potentially extend meaningful survival. Capricor's partnership with Nippon Shinyaku/NS Pharma establishes a commercialization pathway in key markets should approval be secured.
-Company remains on track for PDUFA target action date of August 31, 2025-
-Advisory committee meeting to be held in advance of target action date-
SAN DIEGO, May 05, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) for the Company’s Biologics License Application (BLA) seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. During the meeting, FDA stated that no significant deficiencies have been identified by the Review Committee and that the package is on track for a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025. The FDA has also confirmed its intent to hold an advisory committee meeting, although an official date has not yet been set.
“The successful completion of our mid-cycle review meeting along with the upcoming advisory committee meeting represents major milestones on the path towards approval of deramiocel,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “Deramiocel is a first-in-class cellular therapy with the potential to halt or slow the progression of DMD-cardiomyopathy, and we are pleased to have the opportunity to present the efficacy and safety data to the advisory committee. We have been actively preparing for an advisory committee meeting, and we look forward to providing the physician and patient perspectives to highlight the weight of evidence supporting the transformative potential of deramiocel in treating DMD-cardiomyopathy.”
The BLA submission is supported by Capricor’s cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to patient level data from an FDA-funded and published dataset on the natural history of DMD-cardiomyopathy and potential biomarkers of disease progression. Efficacy from the ongoing HOPE-3 study is not part of this BLA package submission.
About Duchenne Muscular Dystrophy
DMD is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. Treatment options are limited and there is no cure.
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 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 the subject of over 200 peer-reviewed scientific publications and have been administered to over 250 human subjects across several clinical trials.
Deramiocel for the treatment of DMD, has received Orphan Drug Designation from the FDA and European Medicines Agency (EMA). The regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and the Advanced Therapy Medicinal Product (ATMP) Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.
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 preservation of cardiac and skeletal muscle function in dystrophiopathies 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. 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. None of Capricor’s exosome-based candidates have been approved for clinical investigation.
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
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