SAN DIEGO, Nov. 09, 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 abstract presenting cardiac results from the Company’s HOPE-3 Phase 3 trial of Deramiocel in Duchenne muscular dystrophy (DMD) received the Outstanding Research Award in Pediatric Cardiology at the American Heart Association (AHA) Scientific Sessions 2026 on Saturday, November 7, in Chicago. The presentation included new cardiac MRI data on myocardial fibrosis, measured by late gadolinium enhancement (LGE), and a comparison with matched patients not treated with Deramiocel from a natural history dataset.

The HOPE-3 trial randomized 106 patients with DMD to Deramiocel or placebo for 12 months. The trial met its primary endpoint of upper limb function. The key secondary endpoint was change in left ventricular ejection fraction (LVEF) in the intent-to-treat population. LVEF, the percentage of blood the heart pumps out with each beat, is a standard measure of cardiac function.

Key results include:

A smaller increase in scarred segments means slower progression of scarring. LVEF: left ventricular ejection fraction. LGE: late gadolinium enhancement, cardiac MRI scar imaging. The natural history comparison, using data from the Duchenne Muscular Dystrophy Cardiac Care Consortium (DMDCCC), is post hoc and non-randomized. The key secondary endpoint (LVEF, all patients) did not reach statistical significance (p=0.09). P-values for the cardiomyopathy subgroup, LGE and the natural history comparison are nominal (not adjusted for multiple comparisons).Number of patients (Deramiocel vs. comparator) — placebo comparison: LVEF, all patients, 39 vs. 44; LVEF, cardiomyopathy subgroup, 32 vs. 32; LGE, 9 vs. 13. Natural history comparison: LVEF, all patients, 39 vs. 75; LVEF, cardiomyopathy subgroup, 32 vs. 60; LGE, 9 vs. 62.

“Cardiomyopathy is the leading cause of death in patients with Duchenne muscular dystrophy. There is no therapy approved specifically to treat DMD cardiomyopathy; therefore, development of novel therapeutics that can slow or stop its progression is of the utmost importance,” said Jonathan Soslow, M.D., MSCI, Professor of Pediatrics (Cardiology) at Vanderbilt University Medical Center and lead author of the abstract. “While the study suggests benefit across all treated patients, the most pronounced improvements in ventricular function were seen in the cardiomyopathy subgroup. Patients on therapy also showed slower progression of scar aggregation in their hearts. As cardiologists, we watch the scar increase and the heart function decrease year after year in patients with Duchenne. Seeing signals of stabilization for both is encouraging, and it is the combination we would want from a cardiac therapy in Duchenne.”

“The one-year HOPE-3 results were published in The Lancet, and these cardiac findings have now received AHA’s Outstanding Research Award in Pediatric Cardiology – independent recognition of the strength of these data. Protecting the heart remains one of the most urgent unmet needs in Duchenne, and we believe Deramiocel could be an important tool in addressing it. We congratulate Dr. Soslow on this recognition and thank him for sharing these results with the cardiology community,” added Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “Our Biologics License Application remains under FDA review, with a PDUFA target action date of November 22, 2026. We look forward to the completion of that review and to the FDA’s continued focus on rare diseases such as Duchenne.”

Presentation Details

Title: Cardiac Effects of Deramiocel in Duchenne Muscular Dystrophy: Randomized Placebo-Controlled Phase-3 Trial Results and Natural History Comparison
Lead Author: Jonathan Soslow, M.D., MSCI, Vanderbilt University Medical Center
Session: Outstanding Research Award in Pediatric Cardiology
Date: November 7, 2026

AHA Scientific Sessions is the American Heart Association’s annual meeting covering the full spectrum of cardiovascular science. Meeting details, including the full program, are available on the AHA Scientific Sessions 2026 website. The presentation slides will be made available in the publications section of the Capricor website.

About the HOPE-3 Study

HOPE-3 is a Phase 3, randomized, double-blind, placebo-controlled trial evaluating Deramiocel in patients with Duchenne muscular dystrophy. The study enrolled 106 patients, randomized to receive Deramiocel or placebo administered intravenously every three months over a 12-month treatment period. One-year results were published in The Lancet in July 2026. Patients who completed the randomized portion of the study were eligible to continue receiving Deramiocel in an open-label extension (OLE).

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, 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 the treatment of 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™ 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 Capricor’s StealthX™ exosome therapeutics are investigational and have not been approved for commercial use in any indication.

For more information, please contact:

Capricor Media Contact:
Caitlin Kasunich / Raquel Cona
KCSA Strategic Communications
ckasunich@kcsa.com / rcona@kcsa.com
212.896.1241 / 516.779.2630

Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755