Deramiocel

Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy will face an FDA advisory committee ahead of an approval decision that’s expected Aug. 31, the company disclosed on Monday. The San Diego-based company, which saw its stock price $CAPR fall more than 10% on Monday, has been looking to bring its lead program across the finish line since scoring an up to $730 million US deal with Nippon Shinyaku in 2022 for its allogeneic cell therapy known as deramiocel. A date for the adcomm meeting has not yet been set. This will be the first new adcomm scheduled under FDA Commissioner Marty Makary’s tenure, and shows the extent to which the agency will look to use adcomms when questions remain regarding an experimental therapy. The FDA’s adcomm schedule has yet to be updated since Makary took over. Capricor’s submission to the FDA for deramiocel consists of mixed data from a small Phase 2 trial, known as HOPE-2, from 2020, which showed a statistically meaningful improvement versus placebo on a measure that evaluates shoulder, arm and hand strength in patients who are generally non-ambulant. However, on another measure of upper limb function, the trend was in favor of the Capricor drug, but it did not hit statistical significance. “The FDA has acknowledged the clinical data are strong and there aren’t any new issues with manufacturing,” Capricor CEO Linda Marbán told Endpoints News in an interview Monday. “I think it’s the newness of the indication,” she said, explaining why the agency may have scheduled the adcomm. DMD is a rare muscle-wasting disease caused by the absence of dystrophin, a protein that helps keep muscle cells intact. It disproportionately affects boys — and affects roughly 6,000 in the US. The FDA controversially approved Sarepta Therapeutics’ first DMD treatment eteplirsen in 2016, with leadership overruling the agency’s reviewers. Marbán said she didn’t think there would be any “overhang from Sarepta,” and she acknowledged that this is still a patient population without a standard of care. The company is also still waiting on ongoing Phase 3 data, but that’s likely to support a label expansion to treat DMD skeletal muscle myopathy, Marbán said, acknowledging that would only be a small addition to the initial approval.

-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 ConaKCSA Strategic Communications rcona@kcsa.com212.896.1204 Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com858.727.1755