RARE Daily

Capricor Reports Positive Three-Year Results of CAP-1002 in DMD

June 4, 2024

Rare Daily Staff

Capricor Therapeutics said people with Duchenne muscular dystrophy treated with its experimental therapy CAP-1002 continue to show positive benefits after three years of treatment in key measures of upper limb function and heart function when compared to an external comparator dataset of similar DMD patients.

The company said CAP-1002 treatment during the HOPE-2-OLE study continues to have a consistently well-tolerated safety profile throughout the study. The Company plans to present these results in more detail at the upcoming Parent Project Muscular Dystrophy 30th Annual Conference June 27-29, 2024.

Duchenne muscular dystrophy (DMD) is a devastating 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. It occurs in approximately one in every 3,600 live male births across all races, cultures, and countries. DMD afflicts approximately 200,000 boys and young men around the world. Treatment options are limited and there is no cure.

CAP-1002 consists of allogeneic cardiosphere-derived cells (CDCs), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to more than 200 human subjects across several clinical trials.

“Importantly, the external comparator of the disease shows a steady state of decline in both skeletal and cardiac function,” said Linda Marbán, CEO of Capricor. “However, over the course of three-years, we observed a statistically significant reduction in PUL [performance upper limb] decline as well as stabilization in LVEF [left ventricular ejection fraction] in CAP-1002 treated patients suggesting sustained disease attenuation.”

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, which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment entered the HOPE-2-OLE study where they receive CAP-1002 (150 million cells per infusion) every three months.

The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL 2.0. The HOPE-2-OLE study remains ongoing and into its fourth year and participants continue to be monitored for safety and functional performance.

Capricor has entered into a partnership for the exclusive commercialization and distribution of CAP-1002 for DMD in the United States and Japan with Nippon Shinyaku (U.S. subsidiary: NS Pharma), subject to regulatory approval.

Photo: Linda Marbán, CEO of Capricor

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