Capricor Therapeutics(US:CAPR) Globenewswire·2025-11-03 14:15Core Insights - Capricor Therapeutics has published a study on Deramiocel's anti-fibrotic activity, highlighting a validated potency assay that supports quality control in late-stage development for Duchenne muscular dystrophy (DMD) [1][4] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapies for rare diseases, with Deramiocel as its lead investigational product [1][10] - The company has received multiple designations for Deramiocel, including Orphan Drug Designation from the U.S. FDA and EMA, and Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S. [7] Research Findings - The publication details a novel in-vitro potency assay that characterizes the anti-fibrotic mechanism of action of cardiosphere-derived cells (CDCs) in Deramiocel, showing that CDCs can suppress collagen I and III gene expression in human fibroblasts [2][3] - Consistent findings across over one hundred manufacturing lots of Deramiocel demonstrate its anti-fibrotic mechanism, aligning with in vivo studies and clinical data showing stabilization of cardiac function in DMD patients [3] Clinical Development - Deramiocel is currently being evaluated in the Phase 3 HOPE-3 trial, with topline data expected in mid-fourth quarter of 2025 [4][8] - The HOPE-3 trial is a multi-center, randomized, double-blind, placebo-controlled study involving 105 eligible subjects [8][9] Mechanism of Action - Deramiocel consists of allogeneic CDCs that exert immunomodulatory and anti-fibrotic actions, preserving cardiac and skeletal muscle function in muscular dystrophies [6][10] - CDCs secrete exosomes that target macrophages, promoting a healing phenotype rather than a pro-inflammatory one [6] Educational Initiatives - To complement the publication, Capricor has released an educational video explaining the mechanism of action of Deramiocel [5]