SAN DIEGO, Dec. 05, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics, today announced the pricing of its underwritten public offering of 6,000,000 shares of common stock at a public offering price of $25.00 per share. Capricor also granted the underwriters a 30-day option to purchase up to an additional 900,000 shares of its common stock at the public offering price, less the underwriting discounts and ...

Core Insights - Capricor Therapeutics is approaching a critical phase in its development with the upcoming topline results from the HOPE-3 Phase 3 trial of Deramiocel for Duchenne muscular dystrophy (DMD) [2][4][5] - The company has prepared for a potential commercial launch of Deramiocel in 2026, contingent on the trial results and regulatory approval [4][5] - Financial results indicate a significant increase in operating expenses and a net loss for the third quarter of 2025 compared to the same period in 2024 [9][10] Company Updates - The topline results from the HOPE-3 Phase 3 study, which includes 105 participants, are expected in Q4 2025 [4][5] - Following a recent Type A meeting with the FDA, Capricor plans to resubmit its Biologics License Application (BLA) using the HOPE-3 results to address previous concerns raised in a Complete Response Letter [5] - The company has completed its FDA Pre-License Inspection for its commercial manufacturing facility, which is now operational and ready for initial product launch [5] Financial Performance - As of September 30, 2025, Capricor reported cash, cash equivalents, and marketable securities totaling approximately $98.6 million, down from $151.5 million at the end of 2024 [7][25] - Revenues for Q3 2025 were $0, a decrease from approximately $2.3 million in Q3 2024, with total revenues for the first nine months of 2025 also at $0 compared to approximately $11.1 million in the same period of 2024 [8] - Total operating expenses for Q3 2025 were approximately $26.3 million, up from approximately $15.3 million in Q3 2024, leading to a net loss of approximately $24.6 million for Q3 2025 compared to a net loss of approximately $12.6 million in Q3 2024 [9][10] Upcoming Developments - Capricor is preparing for a commercial launch of Deramiocel in 2026, pending the results of the HOPE-3 trial [4][5] - The company is also advancing its StealthX™ exosome-based vaccine platform, with initial data from a Phase 1 clinical trial expected in Q1 2026 [4][6]

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 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]

Core Insights - Capricor Therapeutics is hosting a webinar on July 29, 2025, to discuss the status of its Biologics License Application (BLA) for Deramiocel and provide information on cardiomyopathy in Duchenne Muscular Dystrophy (DMD) [1][2] - Deramiocel is an allogeneic cardiac-derived cell therapy currently in late-stage development for DMD, demonstrating potent immunomodulatory and anti-fibrotic actions [3][5] - Capricor has entered an exclusive commercialization agreement for Deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [5] Company Overview - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with a commitment to innovative treatments [3] - The company utilizes its proprietary StealthX™ platform for preclinical development in various therapeutic areas, including vaccinology and targeted delivery [3] Webinar Details - The webinar will include a review of the BLA status for Deramiocel and a Q&A session with the community [1] - A replay of the webinar will be available on the Parent Project Muscular Dystrophy (PPMD) website [2]

Core Insights - Capricor Therapeutics is advancing its lead cell therapy candidate, Deramiocel, for the treatment of Duchenne Muscular Dystrophy (DMD) associated cardiomyopathy, with a Biologics License Application (BLA) under Priority Review by the FDA, targeting an action date of August 31, 2025 [1][2][3] Regulatory Updates - The FDA has indicated that an Advisory Committee meeting is not required at this time, and the BLA remains under Priority Review [2][7] - A late-cycle review meeting is scheduled for mid-July 2025 [7] Clinical Data - Capricor presented four-year data from its HOPE-2 Open-Label Extension study at the 2025 Parent Project Muscular Dystrophy Conference, showing sustained cardiac function and clinical benefits from long-term Deramiocel treatment [3][7] Product Information - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have demonstrated immunomodulatory and anti-fibrotic actions in preserving cardiac and skeletal muscle function in DMD [5][8] - The product has received multiple designations, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy designation from the FDA [6][10] Market and Collaboration - Capricor has entered into an exclusive commercialization agreement for Deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [10]

Core Insights - Capricor Therapeutics is making significant progress towards delivering the first approved therapy for Duchenne cardiomyopathy, with an FDA target action date of August 31, 2025 for their Biologics License Application (BLA) [2][4][5] Financial Results - As of March 31, 2025, Capricor reported a cash position of approximately $145 million, down from $151.5 million at the end of 2024 [6][9] - Revenues for the first quarter of 2025 were $0, a decrease from $4.9 million in the same quarter of 2024, primarily due to the recognition of previous milestone payments [7] - Total operating expenses for Q1 2025 were approximately $25 million, compared to $15.2 million in Q1 2024, leading to a net loss of approximately $24.4 million, or $0.53 per share, compared to a net loss of $9.8 million, or $0.31 per share, in Q1 2024 [8][20] Corporate Developments - The FDA has accepted Capricor's BLA for deramiocel, which is under priority review, with no significant deficiencies identified in the recent mid-cycle review [4][5] - Capricor appointed Dr. Michael Binks as Chief Medical Officer, who brings over 25 years of experience in clinical development [5] - The National Institute of Allergy and Infectious Diseases (NIAID) plans to initiate a Phase 1 clinical trial of Capricor's StealthX™ exosome vaccine in Q3 2025, pending regulatory approval [5][12] Upcoming Milestones - Capricor is preparing for an FDA advisory committee meeting and a pre-approval inspection as part of the BLA process [2][4] - The company expects its cash reserves to support operations into 2027, excluding additional potential milestone payments [9]