Capricor Reports Positive 24-Month Results from Ongoing HOPE-2 OLE Study of CAP-1002 in Duchenne

Rare Daily Staff

Capricor Therapeutics reported positive 24-month safety and efficacy results from its ongoing HOPE-2 open label extension study with its lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy.

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 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. The OLE study also continues to show a favorable safety profile for long-term treatment of CAP-1002. These data were featured in an oral presentation that was webcast at this year’s Parent Project Muscular Dystrophy Annual Conference.

“The results from this two-year open label study are tremendously impactful for DMD patients showing cardiac and skeletal functional benefits, which underscores the potential long-term benefits of CAP-1002 treatment in DMD,” said Linda Marbán, CEO of Capricor. “Importantly, the natural history of DMD cardiomyopathy suggests a steady decline in cardiac function as measured by ejection fraction, however, in HOPE-2-OLE, we observed improvements in heart function in six of nine patients. Furthermore, as the HOPE-2-OLE data highlights the disease modifying potential of CAP-1002, we believe it is imperative to start treatment as early as possible to prevent the irreversible loss of muscle. Taken together with the favorable safety/tolerability profile, these data position CAP-1002 as a potential anchor therapy for DMD patients. We thank the patients, their families, caregivers, and the broader Duchenne community for continuing to work with us on this promising therapy.”

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.

The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 scale. 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. 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.

CAP-1002 treatment during the HOPE-2-OLE study continues to yield a consistent safety profile and has been well-tolerated throughout the study. The HOPE-2-OLE study remains ongoing, and participants continue to be monitored for safety and functional performance.

Capricor plans to discuss these new results as well as the key features of the ongoing phase 3 HOPE-3 trial and outline its proposed path towards submission of a potential Biologics License Application during an upcoming meeting with the FDA. In parallel, HOPE-3 continues to enroll patients and remains on track to complete enrollment in the second half of 2023 and report the interim analysis in the fourth quarter of 2023.

Photo: Linda Marbán, CEO of Capricor Therapeutics