Capricor Therapeutics announced the publication in Biomedicines, an international peer-reviewed journal, describing a novel in-vitro potency assay designed to characterize the anti-fibrotic mechanism of action of the Capricor’s lead investigational product, Deramiocel, which is currently in late-stage development for the treatment of Duchenne muscular dystrophy (DMD). >>> What is Deramiocel? What Does CAP-1002 Do?
The publication, titled “A Novel In Vitro Potency Assay Demonstrating the Anti-Fibrotic Mechanism of Action of CDCs in Deramiocel,” showed results that further elucidate the biological activity of cardiosphere-derived cells (CDCs), the active component of Deramiocel. The study found that CDCs, through their secreted exosomes and soluble factors, suppressed collagen I and III gene expression in primary human fibroblasts in vitro, supporting their potential to modulate fibrotic processes associated with disease progression.
Findings were consistent across more than one hundred Deramiocel manufacturing lots, demonstrating the anti-fibrotic mechanism of action, which aligns with in vivo pharmacodynamic studies and more than four years of clinical data demonstrating stabilization of cardiac function in DMD patients. The assay was developed and validated as a tool to help assess Deramiocel’s lot-to-lot bioactivity and potency under quality-controlled manufacturing conditions, enhancing Capricor’s ability to ensure product consistency and quality as development progresses.
“This publication contributes to the growing body of evidence describing how cardiosphere-derived cells may modulate fibrotic and inflammatory pathways that underlie the progression of Duchenne muscular dystrophy,” 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.”
Deramiocel is currently being evaluated in the Phase 3 HOPE-3 trial for the treatment of DMD with topline data expected to be available 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 CRL. >>> What is Biologics License Application (BLA)?
To complement this publication, Capricor has released a short educational video describing the mechanism of action of Deramiocel: Watch Here.
Read More: Clinical Trials for Duchenne (List of All Researches)
