Capricor Therapeutics (NASDAQ: CAPR) announced the completion of its Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for deramiocel, an investigational cell therapy for patients with Duchenne muscular dystrophy (DMD) cardiomyopathy. If approved, deramiocel would be the first therapy specifically for DMD-related heart disease.
The BLA submission, completed in late December 2024, is based on cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, compared to natural history data. Capricor has requested priority review, potentially shortening the review timeline to six months.
Clinical Data and Outcomes
Data from the HOPE-2 OLE study demonstrated improvements in cardiac function, including left ventricular ejection fraction (LVEF). In June 2024, the company announced favorable three-year results from the HOPE-2 OLE study of deramiocel for the DMD indication. The data showed a statistically significant reduction in the decline of upper limb function (PUL 2.0) and stabilization of left ventricular ejection fraction among deramiocel-treated patients over three years. This contrasts with the expected decline in untreated DMD patients, suggesting deramiocel can potentially slow disease progression and improve quality of life.
In the HOPE-2 trial, deramiocel was well-tolerated, with manageable hypersensitivity reactions addressed through pretreatment procedures. Patients on deramiocel showed a marked decrease in creatine kinase (CK)-MB as a proportion of total CK, indicative of reduced cardiac muscle damage (p=0.025).
Regulatory Designations and Financial Implications
Deramiocel has received Orphan Drug Designation from the FDA and EMA, along with Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S. and Advanced Therapy Medicinal Product (ATMP) designation in Europe. The BLA submission triggers a $10 million milestone payment from Nippon Shinyaku, Capricor's distribution partner.
About Duchenne Muscular Dystrophy
DMD is a devastating genetic disorder affecting approximately 1 in every 3,500 male births, characterized by progressive muscle weakness and chronic inflammation of skeletal, heart, and respiratory muscles. Mortality typically occurs around age 30, with heart failure being the leading cause of death. Current treatment options are limited, and there is no cure.
Company Statements
"The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD," said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. "We believe that the strength of this application is that deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. We look forward to working with the FDA throughout the review process to support this potential approval."
