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Capricor Therapeutics: Continuation of Phase 3 HOPE-3 Trial of CAP-1002

December 14, 2023

Capricor Therapeutics has announced a positive outcome of the interim futility analysis for HOPE-3, the 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.

“Based on this important milestone, we 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, we believe we 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.”

Linda Marbán, Ph.D., Capricor’s chief executive officer

About the HOPE-3, Phase 3 Trial

HOPE-3 is a Phase 3, multicenter, 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 a placebo every three 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 placebo 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-1002 or placebo in a 1:1 ratio. The aim of Cohort B is to support the inclusion of our San Diego site following initial registration.

Currently, the trials are only being conducted in the United States; however, Defeat Duchenne Canada will continue to work with Capricor Therapeutics to bring this trial to Canadian patients.

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