Capricor Therapeutics, Inc. 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 (PUL version 2.0) scale in the CAP-1002 original treatment group when compared to the original placebo group from HOPE-2 (p=0.02). 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 (p=0.02) 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.

CAP-1002 treatment during the open-label portion of the study continues to yield a consistent safety profile and has been well-tolerated throughout the study. The HOPE-2 OLE study is ongoing, and all participants continue to be monitored for safety and functional performance. The company is currently conducting a pivotal Phase 3 trial, HOPE-3, designed as a randomized, double-blind, placebo-controlled study with approximately 70 patients and enrollment criteria similar to HOPE-2. The Phase 3 study is currently enrolling subjects (NCT05126758).