Capricor Therapeutics' HOPE-3 clinical trial, investigating the cell therapy CAP-1002 for Duchenne muscular dystrophy (DMD), will proceed as planned following a positive interim futility analysis. The independent Data Safety Monitoring Board (DSMB) reviewed the trial's interim results and recommended its continuation, indicating the trial has a reasonable chance of achieving its primary goal.
Potential Expedited Approval
Capricor intends to request a meeting with the U.S. Food and Drug Administration (FDA) in early 2025 to discuss the potential for expedited approval of CAP-1002. The FDA previously confirmed that positive data from the Phase 3 HOPE-3 trial would be sufficient for a Biologics License Application (BLA) seeking the therapy's approval.
"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, PhD, Capricor’s CEO.
Trial Design and Endpoints
The HOPE-3 trial (NCT05126758) is enrolling 102 DMD patients, ages 10 and older, with impaired muscle function. Participants are randomized to receive either CAP-1002 or a placebo every three months for one year. The primary endpoint is the change in arm and hand function, as measured by the Performance of the Upper Limb test, version 2 (PUL 2.0), after one year. Secondary endpoints include assessments of cardiac function and other functional evaluations.
Patients are divided into two groups based on the manufacturing site of CAP-1002. Group A (approximately 58 patients) receives therapy manufactured at Capricor’s Los Angeles facility, while Group B (approximately 44 patients) receives therapy from the San Diego facility. Enrollment for Group A is complete, and enrollment for Group B is ongoing. Capricor plans to use results from Group A to support a BLA.
CAP-1002: Mechanism of Action
CAP-1002 consists of cardiosphere-derived cells, which are progenitor heart cells that release signaling molecules to promote heart health. These cells are derived from the heart tissue of healthy donors.
Prior Clinical Data
HOPE-3 builds upon promising data from the HOPE-2 trial (NCT03406780) and its open-label extension study. HOPE-2 demonstrated improvements in arm and heart function with CAP-1002 treatment. These improvements in arm function were sustained in the extension study after a treatment break of approximately one year.
Regulatory Designations and Commercialization
CAP-1002 has been granted orphan drug, regenerative medicine advanced therapy, and rare pediatric disease designations in the U.S.
Capricor has partnered with Nippon Shinyaku for the exclusive commercialization and distribution of CAP-1002 in the U.S. and Japan. The successful completion of the interim futility analysis triggered the first milestone-based payment under this partnership.
Top-line data from the HOPE-3 trial are expected in late 2024.
