Capricor Therapeutics announces outcome of FDA meeting for CAP-1002
Capricor Therapeutics announced that it had "positive" outcomes in its comprehensive multidisciplinary meeting with the FDA regarding development of the company's novel cell therapy candidate, CAP-1002, to treat Duchenne muscular dystrophy. Capricor met with the FDA in December 2018, as part of the expedited review afforded under the regenerative medicine advanced therapy, or RMAT, designation which the FDA granted to CAP-1002 in February 2018. During the meeting discussion and in subsequent meeting minutes, Capricor asked whether the FDA would agree to its current clinical trial, HOPE-2, serving as the registration study, if HOPE-2 provides evidence that CAP-1002 is safe and effective in treating Duchenne muscular dystrophy. The FDA advised Capricor to request an end of phase meeting after completion of the trial to determine whether HOPE-2 could serve as the registration study. The FDA also reiterated support for the use of the performance of the upper limb, or PUL, 2.0 mid-level test, which measures the ability to use arm and hand muscles, as the primary efficacy endpoint for HOPE-2. In addition, the agency stated that the trial would need to provide evidence of clinically meaningful changes in the PUL, as well as other evidence supportive of CAP-1002 efficacy for patients with advanced Duchenne muscular dystrophy, in order to serve as a registration trial.