Capricor Therapeutics announces 24-month results from HOPE-2 study

Capricor Therapeutics announced 24-month safety and efficacy results from its ongoing HOPE-2 open label extension study with its lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy. Data from the OLE study demonstrated that the majority of patients had an improvement in left ventricular ejection fraction, after two years of CAP-1002 treatment, which suggests preservation of cardiac function. Additionally, patients continue to show statistically significant benefit after two years of treatment in the Performance of the Upper Limb scale when compared to the original rate of decline of the placebo group from HOPE-2 after one year. Furthermore, the OLE study continues to show a favorable safety profile for long-term treatment of CAP-1002. These data will be featured in an oral presentation being webcast today at this year’s Parent Project Muscular Dystrophy Annual Conference. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 scale. At the 24-month timepoint, data showed statistically significant differences in the PUL v2.0 in the OLE treatment group when compared to the original rate of decline of the placebo group from HOPE-2 after one-year. LVEF was measured using cardiac magnetic resonance imaging and six of nine patients showed improvements in heart function with CAP-1002 treatment compared to their final assessment at the end of the HOPE-2 study. Over time, there was an increasing correlation with PUL v2.0 and ejection fraction results. CAP-1002 treatment during the HOPE-2-OLE study continues to yield a consistent safety profile and has been well-tolerated throughout the study. The HOPE-2-OLE study remains ongoing, and participants continue to be monitored for safety and functional performance.