HOPE-3 trial of CAP-1002 for DMD to continue as planned post-analysis
Capricor planning to request FDA meeting on potential expedited approval
That’s the result of an interim futility analysis, which evaluates whether it is mathematically possible for a trial to achieve its goal — if not, the trial could be stopped to save resources and minimize participant burden.
The trial’s interim results were reviewed by the study’s independent Data Safety Monitoring Board (DSMB), which recommended its continuation.
Now, Capricor said it plans to request a meeting with the U.S. Food and Drug Administration (FDA) early next year to discuss the potential of expedited approval for CAP-1002. In a prior meeting, the FDA confirmed that the data from HOPE-3, a Phase 3 trial, would be sufficient, if positive, for an application 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,” Linda Marbán, PhD, Capricor’s CEO, said in a company press release.
Recruiting for Phase 3 trial ongoing at several US sites
Marbán said the company believes CAP-1002 “may address the high unmet medical need” for individuals with DMD and, as such, its team “remain committed to its expeditious advancement towards approval.”
Based on the positive DSMB review, Capricor will seek an FDA meeting “to further discuss options for expedited review and approval,” Marbán said.
Top-line data from the trial are expected in late 2024.
To develop CAP-1002, Capricor has partnered with Nippon Shinyaku regarding the treatment’s exclusive commercialization and distribution in the U.S. and Japan. As part of the partnership, the successful completion of the interim futility analysis led to the first milestone-based payment.
“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,” Marbán said.
CAP-1002 is made of cardiosphere-derived cells — progenitor or precursor heart cells that are able to release signaling molecules to promote heart health. These therapeutic cells are themselves derived from the heart tissue of a healthy donor.
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.
HOPE-3 (NCT05126758) is recruiting a total of 102 DMD patients, ages 10 and older, with impaired muscle function. All participants are expected to be randomly assigned to either CAP-1002 or a placebo, given every three months for the course of one year. The study is open to patients who can or cannot walk independently.
Patients assigned to CAP-1002 will first receive the therapy manufactured at Capricor’s facility in Los Angeles. These participants, dubbed group A, will total about 58 patients. A second group — about 44 participants and known as group B — will receive CAP-1002 manufactured at the company’s facility in San Diego.
Recruitment is ongoing at several U.S. sites; enrollment for group A has been completed, but that for group B is underway.
According to the company, results from group A are intended to support a Biologics License Application to obtain regulatory approval.
The study’s primary goal is to assess how the treatment affects arm and hand function, as measured by the Performance of the Upper Limb test, version 2 (PUL 2.0), after a year. Secondary goals include cardiac function assessments, as well as other functional evaluations.
HOPE-3 comes on the heels of promising data from HOPE-2 (NCT03406780) and its open-label extension study. In HOPE 2, the therapy was shown to improve arm and heart function. The improvements in arm function continued to be seen in the extension study, in which patients resumed treatment after a break of about one year.
CAP-1002 has been granted orphan drug, regenerative medicine advanced therapy, and rare pediatric disease designations in the U.S., which are all intended to encourage treatment development.