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Capricor Reports Positive 18-Month Results from Ongoing DMD Study

January 25, 2023

Capricor Therapeutics said 18-month results from its ongoing HOPE-2 open label extension study in patients with later-stage Duchenne muscular dystrophy continues to show evidence for disease modification with statistically significant differences in a measure of upper limb performance in patients treated with the company’s experimental therapy.

Photo: Linda Marbán, CEO of Capricor

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. Treatment options are limited, and there is no cure.

CAP-1002 consists of off-the-shelf cardiosphere-derived cells, or CDCs, a type of cardiac cell therapy that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity. It is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. The cells function by releasing exosomes that are taken up largely by macrophages and T-cells and begin a cycle of repair. Capricor entered into a partnership for the exclusive commercialization and distribution of CAP-1002 for DMD in the United States with Nippon Shinyaku’s U.S. subsidiary NS Pharma, subject to regulatory approval.

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 open label extension program. As in HOPE-2, CAP-1002 was administered quarterly and current results are from the 18-month analysis.

The company said the CAP-1002 treatment during the open-label portion of the study continues to demonstrate 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.

Capricor 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.

“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,” Linda Marbán, CEO of Capricor, said. “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.”

Author: Rare Daily Staff

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