Capricor Publishes Peer-Reviewed Study Validating Deramiocel's Anti-Fibrotic Mechanism for Duchenne Muscular Dystrophy
Key Insights
Capricor Therapeutics published a peer-reviewed study in Biomedicines describing a novel in-vitro potency assay that characterizes the anti-fibrotic mechanism of action of Deramiocel for Duchenne muscular dystrophy (search) treatment.
The study demonstrated that cardiosphere-derived cells (search) suppress collagen I (search) and III gene expression in primary human fibroblasts through secreted exosomes and soluble factors, with consistent results across over 100 manufacturing lots.
The validated assay enhances quality control and product consistency for Deramiocel as it advances through late-stage development, with Phase 3 HOPE-3 trial topline data expected in mid-fourth quarter 2025.
Capricor Therapeutics has published a peer-reviewed study in the international journal Biomedicines that validates the anti-fibrotic mechanism of action of its investigational cell therapy Deramiocel, currently in late-stage development for Duchenne muscular dystrophy (search) (DMD (search)). The publication provides critical scientific evidence supporting the therapeutic approach as the company prepares for Phase 3 trial results.
Novel Potency Assay Demonstrates Anti-Fibrotic Activity
The study, titled "A Novel In Vitro Potency Assay Demonstrating the Anti-Fibrotic Mechanism of Action of CDCs (search) in Deramiocel," elucidates the biological activity of cardiosphere-derived cells (search) (CDCs), the active component of Deramiocel. The research found that CDCs suppress collagen I (search) and III gene expression in primary human fibroblasts in vitro through their secreted exosomes and soluble factors, supporting their potential to modulate fibrotic processes associated with disease progression.
The findings demonstrated remarkable consistency across more than one hundred Deramiocel manufacturing lots, establishing the anti-fibrotic mechanism of action that aligns with in vivo pharmacodynamic studies and more than four years of clinical data showing stabilization of cardiac function in DMD (search) patients.
Quality Control Enhancement for Late-Stage Development
The validated assay serves as a critical tool for assessing Deramiocel's lot-to-lot bioactivity and potency under quality-controlled manufacturing conditions. This development enhances Capricor's ability to ensure product consistency and quality as the therapy advances through clinical development.
"This publication contributes to the growing body of evidence describing how cardiosphere-derived cells (search) may modulate fibrotic and inflammatory pathways that underlie the progression of Duchenne muscular dystrophy (search)," said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. "By establishing a reproducible and quantitative assay, we have strengthened our understanding of Deramiocel's biological profile and enhanced our ability to ensure product consistency as our Duchenne program advances."
Mechanism of Action and Clinical Development
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (search), a rare population of cardiac cells that have demonstrated potent immunomodulatory and anti-fibrotic actions in preserving cardiac and skeletal muscle function in muscular dystrophies. CDCs (search) act by secreting extracellular vesicles known as exosomes, which target macrophages (search) and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype.
The therapy has been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. CDCs (search) have shown extensive preclinical and clinical data demonstrating their effects in helping to preserve cardiac and skeletal muscle function in DMD (search).
Phase 3 Trial and Regulatory Status
Deramiocel is currently being evaluated in the Phase 3 HOPE-3 trial for DMD (search) treatment, with topline data expected in the mid-fourth quarter of 2025. Following receipt of these data, Capricor expects to resubmit its Biologics License Application (BLA) with its response to the Complete Response Letter (CRL).
The HOPE-3 trial is a multi-center, randomized, double-blind, placebo-controlled clinical trial consisting of two cohorts evaluating the safety and efficacy of Deramiocel in participants with DMD (search). Non-ambulatory and ambulatory boys who meet eligibility criteria are randomly assigned to receive either Deramiocel or placebo every 3 months for a total of four doses during the first 12 months of the trial. A total of 105 eligible subjects have been enrolled in the dual-cohort trial.
Regulatory Designations and Commercial Partnership
Deramiocel has received Orphan Drug Designation for DMD (search) treatment from both the U.S. FDA and the European Medicines Agency (EMA). Additionally, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD (search) in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval.