Capricor Therapeutics announced a positive outcome of the interim futility analysis for HOPE-3, the pivotal Phase 3 trial evaluating CAP-1002 in patients with Duchenne muscular dystrophy (DMD). The results of the interim futility analysis, reviewed by the Data Safety Monitoring Board (DSMB), resulted in a favorable recommendation to continue the HOPE-3 trial as planned. The company believe CAP-1002 may address the high unmet medical need for these patients and remain committed to its expeditious advancement towards approval.

Based on this important milestone, the company will be requesting a meeting with the U.S. Food and Drug Administration (FDA) to further discuss options for expedited review and approval. In addition, the company believe CAP-1002 will be requesting a Meeting with the U.S. food and Drug Administration (FDA). In addition, the company believe the company are well positioned to execute on additional value-driving clinical and regulatory milestones, including reporting topline data from HOPE-3 (Cohort A) in the fourth quarter of 2024.

HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial comprised of two cohorts evaluating the safety and efficacy of CAP-1002 in participants with DMD and impaired skeletal muscle function. Non-ambulatory and ambulatory boys who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. Approximately 102 eligible study subjects will participate in this dual-cohort study.

Enrollment has been completed for Cohort A which is designed to enroll approximately 58 subjects randomized to either CAP-1002 or trial in a 1:1 ratio and is intended to support a Biologics License Application submission. Enrollment has commenced for Cohort B which is designed to enroll approximately 44 participants randomized to either CAP-100 2 or placebo in a 1:1 ratio. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype.

CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials. CAP-1002 for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for CAP-1002 is supported RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for CAP-1002 for thetreatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.