--Findings support the versatility of Capricor’s exosome technology, enabling efficient and scalable loading of siRNAs and PMOs to support future clinical development-- SAN DIEGO, Nov. 24, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc., (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics, today announced new data describing a scalable framework for loading therapeutic small interfering RNAs (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into ...

Core Insights - Capricor Therapeutics will release its financial results for Q3 2025 on November 10, 2025, followed by a conference call at 4:30 p.m. ET [1] - The company is focused on developing cell and exosome-based therapeutics for rare diseases, with its lead product candidate being Deramiocel for Duchenne muscular dystrophy (DMD) [2] Company Overview - Capricor Therapeutics is dedicated to advancing transformative therapies, particularly through its lead candidate Deramiocel, which is in late-stage clinical development for DMD [2] - Deramiocel has shown potent immunomodulatory and anti-fibrotic effects in preserving cardiac and skeletal muscle function in DMD patients [2] - The company is also utilizing its proprietary StealthX™ platform for the targeted delivery of therapeutics, with applications in vaccinology and treatment of various diseases [2] Recent Developments - Capricor has entered into an exclusive commercialization agreement for Deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [5]

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]

Core Insights - Capricor Therapeutics has initiated a Phase 1 clinical trial for its StealthX™ exosome-based vaccine, marking a significant milestone in vaccine development [1][2] - The trial is funded by the National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID) and follows FDA clearance of the Investigational New Drug (IND) application [1][2] - The StealthX™ platform aims to provide an alternative to mRNA vaccines, utilizing a more natural delivery system without adjuvants [2] Company Overview - Capricor Therapeutics is focused on developing transformative cell and exosome-based therapeutics for rare diseases, with its lead product candidate being Deramiocel, a therapy for Duchenne Muscular Dystrophy (DMD) [5][8] - The company is leveraging its proprietary StealthX™ platform for vaccine development and targeted delivery of therapeutics [5] Clinical Trial Details - The Phase 1 trial includes four dosing arms, initially targeting the spike protein of SARS-CoV-2, with plans for an additional arm focusing on the nucleocapsid protein pending FDA clearance [2] - Initial data from the trial is expected in the first quarter of 2026 [2] Strategic Collaborations - Capricor is collaborating with the NIH on the trial and has held discussions with the FDA regarding its Deramiocel program for DMD [2][8] - The company has entered into an agreement with Nippon Shinyaku Co., Ltd. for the exclusive commercialization and distribution of Deramiocel in the U.S. and Japan, subject to regulatory approval [8]

Core Viewpoint - Capricor Therapeutics is advancing its lead cell therapy candidate, Deramiocel, for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD) and has scheduled a Type A meeting with the FDA to discuss its regulatory path for the Biologics License Application (BLA) [1][4][7] Financial Results and Corporate Update - The release of Capricor's financial results for the second quarter ended June 30, 2025, has been rescheduled to August 11, 2025, after market close [2][3] - A conference call and webcast will be held on the same day at 4:30 p.m. ET to review the financial results and provide a corporate update [2][3] Company Overview - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel being a key product candidate showing immunomodulatory and anti-fibrotic effects in muscular dystrophies [4] - The company is also utilizing its proprietary StealthX™ platform for preclinical development in areas such as vaccinology and targeted delivery of therapeutics [4] Commercialization Agreement - Capricor has entered into an exclusive agreement with Nippon Shinyaku Co., Ltd. for the commercialization and distribution of Deramiocel for DMD in the United States and Japan, pending regulatory approval [7]