FDA decision on DMD cell therapy pushed to November. August 25, 2026 The U.S. Food and Drug Administration (FDA) is extending its review of deramiocel, an experimental cell therapy for Duchenne muscular dystrophy (DMD) as it reviews additional data. Capricor Therapeutics, the company developing deramiocel, said it amended its application seeking FDA approval to include new long-term data from the Phase 3 HOPE-3 clinical trial (NCT05126758) and additional statistical analyses. Capricor also said it refined the proposed indication for deramiocel, focusing on the therapy's impact on upper arm function. The FDA set a new decision date of Nov. 22; a decision was originally expected by Aug. 22. "We appreciate the FDA's continued engagement and look forward to working constructively with the agency as it completes its review," Linda Marbán, PhD, CEO of Capricor, said in a company press release. DMD is a genetic disease in which muscles accumulate damage over time. Deramiocel uses immature heart cells called cardiosphere-derived cells, which are thought to dampen inflammation and scarring via the release of signaling molecules. "In a progressive disease where function, once lost, cannot be recovered, we believe preservation of upper limb function has the potential to translate into meaningful differences in patients' independence and daily lives," Marbán said. "That impact reinforces the urgency of our work and our commitment to bringing Deramiocel to the Duchenne community as soon as possible." Recommended Reading July 31, 2026 A virtual FDA meeting gave me the opportunity to advocate for my sons. New studies, new data. Capricor has been developing deramiocel to improve mobility and heart function in people with DMD. The company initially applied for FDA approval of deramiocel based on data from the Phase 2 HOPE-2 clinical trial (NCT03406780) and its open-label extension (NCT04428476). Results from these early trials indicated that deramiocel improved measures of arm and heart function, but the studies included fewer than two dozen people with DMD, and in 2025 the FDA declined the application saying more data were needed. Capricor resubmitted its application based on data from the larger Phase 3 trial HOPE-3, which involved more than 100 people with DMD who either were predicted to be unable to walk in two years or were already unable to walk. The trial's main goal was to show that deramiocel led to better outcomes than placebo on the Performance of the Upper Limb test, version 2 (PUL 2.0), a standardized measure of arm and hand function. According to Capricor, HOPE-3 met its goal, with deramiocel leading to significantly better PUL 2.0 scores than the placebo. Published results also showed that the therapy met a key second goal of improving left ventricular ejection fraction (LVEF), a measure of how well the heart pumps blood to the body. The FDA disputed these claims, saying Capricor altered its statistical analysis plan and that, under the original plan, neither PUL 2.0 nor LVEF showed significant improvements with deramiocel relative to the placebo. Capricor has maintained that the study met its main goal. "HOPE-3 met its primary endpoint, demonstrating a statistically significant benefit in upper limb function," Marbán said. The FDA convened an advisory committee meeting in which independent experts were asked to vote on whether the available evidence supports deramiocel as an effective treatment for heart disease in DMD. A few weeks ago, the council voted no. As part of ongoing discussions with the FDA following that meeting, Capricor submitted an amendment that includes two years of data from the HOPE-3 open-label extension, in which all patients were treated with deramiocel and monitored for long-term outcomes. Since the FDA has classified these new data as a major amendment, the agency extended its review to allow time to analyze the new information. "With an additional year of follow-up from HOPE-3, we now have one of the most extensive clinical datasets evaluating upper limb function in Duchenne," Marbán said. "We believe the additional open-label data and further analyses included in the amendment strengthen the evidence supporting a refined proposed indication."

Capricor Therapeutics reported a net loss of $40.7 million for Q2 2026, with $237.9 million in cash and marketable securities. The company notably omitted the analyst Q&A session from its earnings call, citing ongoing FDA discussions about its regulatory path. Capricor faces a securities fraud class action lawsuit covering the period from 17 December 2025 to 26 July 2026. The lawsuit alleges the company made misleading statements about clinical trial data for Deramiocel, its Duchenne muscular dystrophy treatment candidate. In December 2025, Capricor announced positive results from its HOPE-3 trial, causing shares to surge 370%. The company subsequently raised capital through a share offering at $25 per share. However, FDA briefing documents published on 27 July 2026 revealed unauthorised modifications to the trial's statistical analysis plan. The FDA stated the trial did not meet its efficacy endpoints. Capricor shares fell 64% to $7.00. The lead plaintiff deadline is 28 September 2026.

Capricor Therapeutics announces extension of PDUFA target action date as FDA continues review of Deramiocel BLA. - New PDUFA target action date of November 22, 2026 follows submission of additional Phase 3 HOPE-3 data and analyses supporting a refined proposed indication - SAN DIEGO, Aug. 24, 2026 (GLOBE NEWSWIRE) - Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) target action date for its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22, 2026 to November 22, 2026. As part of its ongoing discussions with the FDA following the July 2026 Advisory Committee meeting, Capricor submitted an amendment to the BLA that includes 24-month open-label extension data from its pivotal Phase 3 HOPE-3 study and additional robustness analyses, with a request that the FDA review the existing and new data in support of a refined proposed indication focused on upper limb function, the primary endpoint of HOPE-3. The FDA's Center for Biologics Evaluation and Research (CBER) accepted the amendment for review, citing the significant unmet medical need in DMD. The FDA has classified the submission as a major amendment and extended the PDUFA target action date by three months to allow additional time to review the information. "With an additional year of follow-up from HOPE-3, we now have one of the most extensive clinical datasets evaluating upper limb function in Duchenne," said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. "HOPE-3 met its primary endpoint, demonstrating a statistically significant benefit in upper limb function, and we believe the additional open-label data and further analyses included in the amendment strengthen the evidence supporting a refined proposed indication. We appreciate the FDA's continued engagement and look forward to working constructively with the agency as it completes its review." Marbán continued, "The powerful testimony shared by patients, families and clinicians at the July Advisory Committee meeting underscored the importance of preserving upper limb function and the independence it provides for people living with Duchenne. In a progressive disease where function, once lost, cannot be recovered, we believe preservation of upper limb function has the potential to translate into meaningful differences in patients' independence and daily lives. That impact reinforces the urgency of our work and our commitment to bringing Deramiocel to the Duchenne community as soon as possible." 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 immunomodulatory and anti-fibrotic actions in the preservation of skeletal and cardiac 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. For the treatment of DMD, Deramiocel holds Orphan Drug, RMAT and Rare Pediatric Disease designations in the U.S., and Orphan Drug and ATMP designations in Europe. The Rare Pediatric Disease Designation may qualify Capricor for a Priority Review Voucher upon approval. About Capricor Therapeutics Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing cell and exosome-based therapeutics for the treatment of rare diseases. Our lead product candidate, Deramiocel, is an allogeneic cardiac-derived cell therapy in late-stage development for Duchenne muscular dystrophy (DMD), evaluated in clinical studies for its potential to preserve skeletal and cardiac muscle function. Capricor is also advancing its proprietary StealthX(TM) exosome platform for the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics across a range of diseases. At Capricor, we are committed to delivering new therapies for patients with rare diseases. For more information, visit capricor.com and follow Capricor on Facebook, Instagram and X. 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, revenue and reimbursement estimates, projected terms of definitive agreements, our financial position, our possible uses of existing cash and investment resources; results of securities litigation; and statements regarding our litigation with Nippon Shinyaku Co., Ltd. and NS Pharma, Inc., including the nature of the dispute, our expectations regarding any legal proceedings, and our ability to commercialize Deramiocel independent of our existing distribution agreement 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, 2025, as filed with the Securities and Exchange Commission on March 17, 2026 and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as filed with the Securities and Exchange Commission on August 14, 2026. 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. Deramiocel and the StealthX(TM) vaccine are investigational candidates and have not been approved for commercial use in any indication. Capricor Media Contact: Caitlin Kasunich / Raquel Cona KCSA Strategic Communications [email protected] / [email protected] 212.896.1241 / 516.779.2630 Capricor Company Contact: AJ Bergmann, Chief Financial Officer [email protected] 858.727.1755