Capricor Therapeutics Announces Positive 24-Month Results from Ongoing HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy

-Improvements in Left Ventricular Ejection Fraction (LVEF) for the M

ajority of Patients Suggest Preservation of Cardiac Function-

-Results in Performance of the Upper Limb PUL v2.0 Continue to Show Long-Term Benefit in Skeletal Muscle Function (p=0.02)-

-Safety and Efficacy Results Continue to Suggest Potential Disease Attenuation in Duchenne Muscular Dystrophy (DMD)

Excerpt from the Press Release:

SAN DIEGO, June 30, 2023 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, today announced positive 24-month safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD). 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 (p=0.021) 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. Furthermore, the OLE study continues to show a favorable safety profile for long-term treatment of CAP-1002. These data will be featured in an oral presentation being webcast today at this year’s Parent Project Muscular Dystrophy (PPMD) 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, Ph.D., Capricor’s chief executive officer. “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.”

The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 scale (p=0.02). 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 (p=0.021).

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