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Capricor Therapeutics Announces Continuation of Phase 3 HOPE-3 Trial of CAP-1002 in Duchenne Muscular Dystrophy Based on Completion of Interim Futility Analysis
--Favorable Interim Futility Analysis Results-- --Successful Completion Triggers First Milestone Payment Under U.S. Agreement with Nippon Shinyaku-- --HOPE-3
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"--Favorable Interim Futility Analysis Results-- --Successful Completion Triggers First Milestone Payment Under U.S. Agreement with Nippon Shinyaku-- --HOPE-3 (Cohort A) Enrollment Complete; Topline Data Expected in the Fourth Quarter of 2024; Cohort B Enrollment Initiated-- --Company Plans to Request a Meeting with FDA in the First Quarter of 2024 to Further Discuss Opportunities for Expedited Approval Pathways-- SAN DIEGO, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, today 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. This interim futility analysis triggers the first milestone payment under the Company’s U.S. Commercialization and Distribution Agreement with Nippon Shinyaku Co., Ltd. “We are pleased with the positive outcome of the DSMB review which supports the continued advancement of our HOPE-3 trial towards potential approval of CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “We believe CAP-1002 may address the high unmet medical need for these patients and we remain committed to its expeditious advancement towards approval. 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.” About the HOPE-3, Phase 3 Trial 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-...