– 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 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 is focused on developing therapies for Duchenne cardiomyopathy and has faced regulatory challenges but is working on a clear path forward for approval [2][4] - The company has made significant progress with its exosome platform, StealthX™, which has received FDA clearance for clinical trials [4][6] - Financial results indicate a net loss for the second quarter of 2025, with revenues dropping to zero compared to the previous year [8][10] Corporate Update - Capricor aims to resubmit its Biologics License Application (BLA) for Deramiocel based on existing data, with additional data from the HOPE-3 trial expected in Q4 2025 [4][5] - The FDA has accepted all observations from the Pre-License Inspection, marking a milestone in regulatory progress [4][5] - The company is preparing for a Type A meeting with the FDA to discuss the approval pathway [4][5] Financial Performance - Cash position as of June 30, 2025, was approximately $122.8 million, down from $151.5 million at the end of 2024 [7][26] - Revenues for Q2 2025 were $0, a decrease from approximately $4.0 million in Q2 2024, and for the first half of 2025, revenues were also $0 compared to approximately $8.9 million in the first half of 2024 [8][10] - Total operating expenses for Q2 2025 were approximately $27.7 million, up from $15.6 million in Q2 2024 [9][10] Clinical Development - The HOPE-3 Phase 3 clinical trial is ongoing, with topline data expected in Q4 2025 [5][6] - The FDA has cleared the IND for the StealthX™ exosome-based vaccine, marking its first clinical entry [4][6] - Orphan Drug Designation has been granted for Deramiocel in Becker muscular dystrophy, expanding its therapeutic strategy [5][6]

Core Insights - Capricor Therapeutics announced positive four-year safety and efficacy results for Deramiocel, its lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD) [1][3] - The findings will be presented at the PPMD 2025 Annual Conference, highlighting the importance of addressing both cardiac and skeletal muscle functions in DMD treatment [1][4] Efficacy Results - After four years of treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline, indicating clinical benefit, especially in patients with baseline LVEF >45% [2] - The treatment also slowed skeletal muscle disease progression, with a smaller average decline in Performance of the Upper Limb (PUL v2.0) in the fourth year (0.6 points) compared to the first year (1.8 points) [3][7] Safety Profile - Deramiocel maintained a favorable safety profile throughout the study, reinforcing its potential as a therapeutic option for DMD [3][7] Regulatory Progress - Capricor is in the process of obtaining regulatory approval for Deramiocel, with its Biologics License Application (BLA) under priority review and no evidence of delays in discussions with the FDA [4][10] Study Background - The HOPE-2 study was a randomized, double-blind, placebo-controlled Phase 2 trial, with patients receiving intravenous infusions of Deramiocel (150 million cells) every three months [5] - Following the study, all patients entered a treatment gap phase before enrolling in the HOPE-2 Open-Label Extension (OLE) study, which continues to monitor safety and efficacy [5] About Duchenne Muscular Dystrophy - DMD is a severe genetic disorder affecting approximately 15,000 individuals in the U.S., characterized by progressive muscle degeneration and leading to cardiomyopathy, which is a major cause of mortality [6][9] About Deramiocel - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) known for their immunomodulatory and anti-fibrotic properties, which may help preserve cardiac and skeletal muscle function in DMD [8][10] - The therapy has received multiple designations from regulatory agencies, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation [9]

Core Insights - Capricor Therapeutics is progressing towards the FDA's PDUFA target action date of August 31, 2025, for its Biologics License Application (BLA) for deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy [1][2] - The FDA's mid-cycle review meeting indicated no significant deficiencies in the BLA submission, and an advisory committee meeting will be scheduled [1][2] - DMD is a severe genetic disorder affecting approximately 15,000-20,000 patients in the U.S., characterized by progressive muscle weakness and heart failure [3] Company Overview - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with deramiocel as its lead product candidate [7] - Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory and anti-fibrotic effects in clinical studies [4][7] - The company has received Orphan Drug Designation from the FDA and EMA for deramiocel, along with RMAT and ATMP designations to support its regulatory pathway [5] Clinical Development - The BLA submission for deramiocel is backed by data from Phase 2 trials (HOPE-2 and HOPE-2 OLE) and comparisons to FDA-funded datasets on DMD-cardiomyopathy [2] - Efficacy data from the ongoing HOPE-3 study is not included in the current BLA submission [2] - Capricor has entered an agreement with Nippon Shinyaku Co., Ltd. for the exclusive commercialization of deramiocel in the U.S. and Japan, pending regulatory approval [9]