PPMD is pleased to share that Capricor Therapeutics announced today positive final data from the HOPE-2 randomized, double-blind, placebo controlled clinical trial using CAP-1002 to treat patients with Duchenne. The HOPE-2 clinical trial met its primary efficacy endpoint of mid-PUL v1.2 (Performance of Upper Limb), as well as various skeletal and cardiac endpoints suggesting clinically relevant slowing of disease progression. CAP-1002 is Capricor’s cell-based therapeutic candidate whose mechanism of action is immunomodulatory and anti-fibrotic.

 

Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point in the intent to treat (ITT) population. Approximately 80% of the patients were non-ambulant and all patients were on a stable regimen of steroids. Demographic and baseline characteristics were similar between the two treatment groups. Final data analysis demonstrated that young men in the advanced stages of DMD experienced improvements in skeletal and cardiac measurements after receiving four doses of CAP-1002 over the course of 1 year.

 

CAP-1002 was generally safe and well tolerated throughout the study. With the exception of two hypersensitivity reactions early in the clinical trial, which were mitigated with a common pre-medication regimen, there were no serious safety signals identified by the HOPE-2 Data and Safety Monitoring Board (DSMB).

 

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