Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA Following FDA Review of HOPE-3 Topline Data

Core Viewpoint - Capricor Therapeutics is advancing its Biologics License Application (BLA) for Deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD), following a request from the FDA for the full clinical study report to address a Complete Response Letter [1][2][3][4] Company Overview - Capricor Therapeutics (NASDAQ: CAPR) focuses on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel as its lead product candidate for DMD [1][11] - The company has received multiple designations for Deramiocel, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA [7] Clinical Development - The Phase 3 HOPE-3 trial evaluated the safety and efficacy of Deramiocel in 106 eligible subjects, with results showing significant improvements in skeletal muscle and cardiac function [4][10] - The company plans to submit the requested clinical study report to the FDA in February 2026, which is expected to support the continued review of the BLA [3][9] Disease Context - Duchenne muscular dystrophy (DMD) is a severe genetic disorder affecting approximately 15,000 individuals in the U.S., primarily boys, leading to progressive muscle degeneration and limited treatment options [5] - The absence of functional dystrophin in muscle cells causes deterioration of skeletal and cardiac muscles, with heart failure being the leading cause of death in DMD patients [5] Mechanism of Action - Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory and anti-fibrotic effects, preserving muscle function in DMD [6] - CDCs secrete exosomes that target macrophages, promoting a healing response rather than a pro-inflammatory one [6]