Capricor Therapeutics Provides Regulatory Update on Deramiocel Program for Duchenne Muscular Dystrophy Following Type A Meeting

Core Insights - Capricor Therapeutics announced a regulatory update regarding its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), following a Type A meeting with the FDA after receiving a Complete Response Letter (CRL) in July 2025 [1][2][3] Regulatory Update - The Type A meeting aimed to establish a path toward potential approval of Deramiocel, with key outcomes providing clarity on the regulatory strategy and the opportunity to use HOPE-3 data for approval if it meets regulatory requirements [2][3] - The FDA has aligned with Capricor on endpoints for the HOPE-3 pivotal trial, which is expected to support the BLA resubmission [7][8] Clinical Trials - The HOPE-3 trial is designed to validate findings from previous studies (HOPE-2 and HOPE-2-OLE) that demonstrated significant benefits in cardiac and skeletal muscle function [3][8] - The trial consists of two cohorts evaluating the safety and efficacy of Deramiocel in DMD participants, with 105 subjects enrolled [11] Financial Position - Capricor maintains a strong financial position to support the advancement of Deramiocel through regulatory review and potential launch [4] Product Information - Deramiocel (CAP-1002) is composed of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory and anti-fibrotic actions in preserving muscle function in muscular dystrophies [9][12] - The product has received multiple designations, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation, which may qualify Capricor for a Priority Review Voucher upon approval [10]