Capricor Therapeutics Says Duchenne Muscular Dystrophy Candidate Shows Sustained Cardiac, Skeletal Benefits After 3 Years

On Friday, Capricor Therapeutics CAPR announced 3-year safety and efficacy results from its ongoing HOPE-2 open-label extension (OLE) study for its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD).

DMD is a genetic disease that causes progressive muscle weakness and degeneration. 

Cardiac and orthopedic complications, including scoliosis and contractures (tightened muscles) 

The data was highlighted at the Annual Congress of the World Muscle Society.

Also Read: EXCLUSIVE: Capricor Therapeutics Inks European Expansion, Commercialization Pact For Lead Asset With Japanese Pharma Firm.

The 3-year data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac function measures, including left ventricular ejection fraction (LVEF%) and indexed volumes, which are considered highly relevant in terms of predicting long-term cardiac outcomes.

In addition, the treatment effect was clearly bifurcated in those with ejection fractions greater than 45% at the end of HOPE-2, suggesting that early and sustained intervention will be key in attenuating the impacts of DMD cardiomyopathy.

To evaluate the relevance of the data to disease progression and the chronic and progressive nature of DMD, where cardiac function can decline year over year, a natural history data set was used to compare the trajectory of those treated with deramiocel to the standard of care.

In addition to the cardiac data, patients demonstrated a statistically and clinically relevant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score compared to an external comparator dataset of similar DMD patients.

The HOPE-2 OLE study continues to show a favorable safety profile for long-term treatment of deramiocel.

Price Action: CAPR stock is up 8.05% at $18.52 at the last check Friday.

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