Capricor Therapeutics to Present Late-Breaking Findings at 2026 MDA Clinical Conference

Capricor Therapeutics, a pioneering biotechnology company specializing in innovative cell and exosome-based therapeutics for rare diseases, recently unveiled significant results from its Phase 3 HOPE-3 clinical study pertaining to Deramiocel in Duchenne muscular dystrophy (DMD). The Phase 3 clinical study findings will be the focal point of a late-breaking oral presentation at the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference scheduled for March 8-11, 2026, in Orlando, Florida.

Linda Marbán, Ph.D., Chief Executive Officer of Capricor, expressed enthusiasm about the selection of HOPE-3 for a late-breaking presentation at the eminent MDA Conference. Dr. Marbán emphasized the increasing clinical evidence substantiating Deramiocel and its potential life-changing implications for individuals living with Duchenne. She added that sharing these pivotal Phase 3 results with the DMD community is a significant step forward as the company continues its regulatory endeavors. Part of these efforts included the recent submission of the HOPE-3 clinical study report to the U.S. Food and Drug Administration (FDA) in line with the ongoing Biologics License Application (BLA) review process.

The Phase 3 HOPE-3 study’s oral presentation at the MDA conference will delve into the confirmed benefits of Deramiocel in enhancing musculoskeletal and cardiac health in individuals with DMD. Renowned faces like Craig McDonald, M.D., who serves as a Professor of Physical Medicine & Rehabilitation and Pediatrics at UC Davis Health and is the National Principal Investigator of the HOPE-3 trial, will be spearheading the presentation scheduled for March 11, 2026, at 2:45 p.m. ET in Florida 4 at the event venue.

The FDA had previously requested the submission of the Clinical Study Report (CSR) to address points highlighted in the Complete Response Letter (CRL) and bolster the comprehensive review of Capricor’s BLA for Deramiocel in the realm of Duchenne muscular dystrophy. This CSR submission paves the way for potential next steps in the regulatory process, with specific emphasis on the Prescription Drug User Fee Act (PDUFA) target action date.

Duchenne Muscular Dystrophy, a debilitating X-linked genetic disorder, is marked by progressive muscle deterioration that affects key muscle groups, encompassing skeletal, respiratory, and cardiac muscles. This condition stems from the absence of functional dystrophin, a critical protein in muscle cells. DMD predominantly impacts boys and, unfortunately, leads to heart muscle deterioration over time, contributing to cardiomyopathy and eventual heart failure – a primary cause of fatality in DMD cases. Presently, there is no definitive cure for DMD, and available treatment options are limited.

Deramiocel, also known as CAP-1002, incorporates allogeneic cardiosphere-derived cells (CDCs) that exhibit potent immunomodulatory and anti-fibrotic properties. Through the secretion of extracellular vesicles like exosomes, CDCs target macrophages, prompting them to transition from a pro-inflammatory state to a healing-oriented phenotype. The therapeutic potential of CDCs has been substantiated across more than 250 scientific publications and has been administered to over 250 human subjects in various clinical trials. Notably, Deramiocel has secured Orphan Drug Designation in the U.S. and Europe for DMD treatment, in addition to garnering several other noteworthy designations like Regenerative Medicine Advanced Therapy (RMAT) and Rare Pediatric Disease Designation from the FDA.

The HOPE-3 Phase 3 Trial, a pivotal study on Deramiocel, is designed as a multicenter, randomized, double-blind, placebo-controlled endeavor involving non-ambulatory and ambulatory boys who qualify as per defined eligibility criteria. The trial aims to evaluate the safety and efficacy of Deramiocel in adolescents with DMD, signifying a crucial step towards advancing therapeutic interventions in a condition where treatment options are regrettably scarce.