– Webinar to be held Wednesday, December 17, 2025, at 1:00 p.m. ET – SAN DIEGO, Dec. 16, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics, today announced that Parent Project Muscular Dystrophy (PPMD) will host a community webinar to share and discuss positive topline results from Capricor’s Phase 3 HOPE-3 trial evaluating Deramiocel, the Company’s investigational cell therapy for the treatment of Duche ...

Core Viewpoint - Capricor Therapeutics, Inc. has announced a public offering of 6,000,000 shares of common stock priced at $25.00 per share, aiming to raise approximately $150 million in gross proceeds to support its product development and general corporate purposes [1][2][3]. Group 1: Offering Details - The public offering consists of 6,000,000 shares at a price of $25.00 per share, with an additional 900,000 shares available for underwriters to purchase within 30 days [1] - The offering is expected to close around December 8, 2025, pending customary closing conditions [1] - Piper Sandler and Oppenheimer & Co. are the joint book-running managers, while H.C. Wainwright & Co. serves as the co-manager for the offering [2] Group 2: Use of Proceeds - The net proceeds from the offering will be utilized for the continued development and manufacturing of product candidates, working capital, and general corporate purposes [3] Group 3: Company Overview - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics, with its lead product candidate, Deramiocel, in late-stage clinical development for Duchenne muscular dystrophy (DMD) [6] - Deramiocel has shown significant immunomodulatory and anti-fibrotic effects in preserving cardiac and skeletal muscle function in DMD patients [6] - The company is also advancing its proprietary StealthX™ platform for targeted delivery of therapeutics, with applications in vaccinology and treatment of various diseases [6] Group 4: Regulatory and Commercialization - The offering is conducted under an effective shelf registration statement filed with the SEC, ensuring compliance with regulatory requirements [4] - Capricor has an exclusive agreement with Nippon Shinyaku Co., Ltd. for the commercialization and distribution of Deramiocel in the U.S. and Japan, pending regulatory approval [9]

Core Insights - Capricor Therapeutics announced positive topline results from the Phase 3 HOPE-3 trial for Deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) [1][2] - The trial demonstrated statistically significant improvements in both skeletal and cardiac function, reinforcing previous findings from the HOPE-2 trial [2][5] - The results are expected to support regulatory approval following a Complete Response Letter from the FDA earlier this year [2][5] Study Details - HOPE-3 was a randomized, double-blind, placebo-controlled trial involving 106 participants across 20 U.S. clinical sites, with an average age of approximately 15 years [2][11] - Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for 12 months [2][12] - The study maintained a favorable safety and tolerability profile consistent with prior clinical experience [2][5] Efficacy Results - The primary endpoint, Performance of Upper Limb (PUL v2.0), showed a 54% slowing of progression compared to placebo (p=0.029) [3][5] - The key secondary endpoint, Left Ventricular Ejection Fraction (LVEF), demonstrated a 91% improvement (p=0.041) [3][5] - Statistically significant results were achieved across all controlled secondary endpoints [5] Clinical Implications - The findings indicate meaningful treatment effects on both upper limb function and cardiomyopathy, addressing critical aspects of DMD [5][6] - The preservation of cardiac function is particularly significant, as cardiomyopathy is the leading cause of mortality in DMD [6][8] - The results provide renewed confidence for families seeking therapies that maintain functional ability and protect heart health [6][10] Company Background - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel as its lead candidate for DMD [13] - Deramiocel has received Orphan Drug Designation from the FDA and EMA, along with other designations that may facilitate regulatory approval [10][15] - The company is committed to advancing innovative therapies and has a proprietary platform for targeted delivery of therapeutics [13]

Core Insights - Capricor Therapeutics has announced new data on a scalable framework for loading therapeutic siRNAs and PMOs into exosomes, which is crucial for future clinical development [1][2][4] Group 1: Exosome Technology - The poster presented at the AAEV 2025 highlighted Capricor's proprietary exosome-based technology, showcasing strategies for scale-up and scale-out electroporation to achieve larger yields of engineered exosomes [2][4] - Key findings indicate that engineered exosomes derived from 293F cells can be successfully loaded with therapeutic siRNA and PMO using optimized electroporation conditions, with comparable loading efficiencies to standard methods [7] Group 2: Clinical Development - Establishing a scalable and reproducible loading process is essential for advancing Capricor's exosome pipeline for therapeutic development [4] - The framework developed provides a feasible pathway for producing clinically relevant quantities of loaded exosomes to support future clinical development [7] Group 3: Company Overview - Capricor Therapeutics is focused on advancing cell and exosome-based therapeutics, with its lead product candidate, Deramiocel, in late-stage clinical development for Duchenne muscular dystrophy (DMD) [5][9] - The company is leveraging its proprietary StealthX™ platform for preclinical development aimed at targeted delivery of therapeutics, with potential applications across a wide range of diseases [5]

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]