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]

-Company remains on track for PDUFA target action date of August 31, 2025- Deramiocel for the treatment of DMD, has received Orphan Drug Designation from the FDA and European Medicines Agency (EMA). The regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and the Advanced Therapy Medicinal Product (ATMP) Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment ...