Capricor announces 18-month results from ongoing HOPE-2 OLE study of CAP-1002

Capricor Therapeutics announced positive 18-month results from its ongoing HOPE-2 open label extension, OLE, study in patients with later-stage Duchenne muscular dystrophy, DMD. Data from the OLE study continues to show evidence for disease modification with statistically significant differences in the Performance of the Upper Limb scale in the CAP-1002 original treatment group when compared to the original placebo group from HOPE-2. In addition, disease progression is attenuated equally in both groups once patients begin treatment in the OLE. The study met its primary endpoint at the one-year timepoint and continues to show statistically significant improvements on the Performance of the Upper Limb scale for patients treated with CAP-1002 at 18-months. In the study, CAP-1002 was made available to the original 20 patients enrolled in the HOPE-2 study. Of those, 13 entered and 12 completed 18-months of study follow-up. The breakdown of patients included seven from the original placebo group and six from the original CAP-1002 treatment group. All patients were off CAP-1002 or placebo for a mean of approximately one-year before resumption or initiation of therapy under the HOPE-2 OLE program. As in HOPE-2, CAP-1002 was administered quarterly and current results are from the 18-month analysis. Linda Marban, Ph.D., CEO of Capricor, commented, "In the OLE phase of the HOPE-2 study, we observed statistically significant slowing of the progression of disease across patients treated with CAP-1002. We are extremely pleased by the robust and consistent results observed to date, which, together with the favorable safety/tolerability profile, suggest that CAP-1002 holds promise as a potential anchor therapy for DMD patients. Further, the long-term efficacy and potential disease modification effect will augment our clinical package as we continue on our regulatory pathway towards potential approval of CAP-1002 for treatment of patients with DMD. We thank the patients, their families, caregivers, and the broader Duchenne community for working with us on this promising therapy."