Capricor Completes BLA for Deramiocel for Duchenne Cardiomyopathy

Capricor Therapeutics has completed its submission of the biologics license application (BLA) seeking full approval for the cell therapy deramiocel to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy.

Duchenne muscular dystrophy is a rare and fatal genetic disorder that causes progressive muscle weakness, mostly in boys. The disorder results from impaired production of functional dystrophin, which normally functions as a structural protein in muscle. In the heart, muscle cells progressively die and are replaced with scar tissue, which eventually leads to heart failure.

If approved, deramiocel would be first therapy specifically approved for Duchenne muscular dystrophy cardiomyopathy. Company officials have said that deramiocel will likely be a lifelong treatment, with an infusion delivered quarterly.

Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a population of stromal cells, which provide nutrients to tissue and organs and remove waste. Cardiosphere-derived cells secrete extracellular vesicles known as exosomes, which target macrophages and help in healing. These cells have been shown to have regenerative properties.

The BLA, which was a rolling that began in October 2024, is supported cardiac data from the phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared with natural history data from an FDA funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. Capricor has requested a priority review, which, if granted, would reduce the review timeline from the standard 10-month to a priority 6-month review from the date the submission is accepted by the FDA.

Data from the HOPE-2 OLE were presented at a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society in October 2024. The three-year data from HOPE-2 OLE demonstrated improvements in multiple measures of cardiac function, including left ventricular ejection fraction, as well as indexed volumes, which are considered relevant in terms of predicting long-term cardiac outcomes.

Patients also demonstrated a statistically and clinically relevant benefit in the Performance of Upper Limb (PUL) v2.0 total score when compared with an external comparator dataset of similar DMD patients. The Performance of Upper Limb measurement is a 22-item scale that physicians use to grade performance of various functional skills.

Linda Marbán, Ph.D.

"We have been working closely with FDA to move deramiocel towards potential approval as quickly as possible because once heart function is lost, it is unlikely to be restored,” Linda Marbán, Ph.D., Capricor’s CEO said in a news release in October 2024. “Furthermore, as more therapies become available that could impact the trajectory of skeletal muscle loss in DMD, preservation of cardiac function will be even more important.”

Capricor has partnered Nippon Shinyaku Co. for U.S. commercialization and distribution.