Capricor Therapeutics announced positive 24-month safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD). Data from the OLE study demonstrated that the majority of patients had an improvement in left ventricular ejection fraction (LVEF), after two years of CAP-1002 treatment, which suggests preservation of cardiac function. Additionally, patients continue to show statistically significant benefit (p=0.021) after two years of treatment in the Performance of the Upper Limb (PUL v2.0) 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. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 scale (p=0.02). 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 (p=0.021).

LVEF was measured using cardiac magnetic resonance imaging (cMRI) 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 (24-month OLE results r=0.75, p=0.02). HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD.

Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase.

Then all eligible patients who wished to remain on treatment re-entered the OLE study where they received CAP-1002 (150 million cells per infusion) every three months over the course of 24 months. Patients continued through the gap phase (off treatment for both groups) and the OLE phase (on treatment for both groups).