Capricor Therapeutics announces long-term data from HOPE-2 OLE study

Capricor Therapeutics announced positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension study for its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy. The data was highlighted in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society October 8-12, 2024 in Prague, Czechia. “These findings are crucial as they demonstrate consistent long-term benefit for cardiac function in DMD. There are no approved therapies for cardiomyopathy in DMD, which is the leading cause of mortality in DMD,” said Dr. Craig McDonald, National PI, University of California, Davis. “In addition, the data demonstrated sustained improvements in skeletal muscle function, as measured by PUL v2.0, over a three-year period. To our knowledge, no other therapeutic has exhibited such a favorable safety and efficacy profile in DMD.” The 3-year data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures of cardiac function, including left ventricular ejection fraction, as well as indexed volumes, which are considered highly relevant in terms of predicting long-term cardiac outcomes. In addition, there was clear bifurcation in the treatment effect seen in those that had ejection fractions greater than 45% at the end of HOPE-2 which suggests that early and sustained intervention will be key in attenuating the impacts of DMD cardiomyopathy. In order to evaluate the relevance of the data to disease progression as well as 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 standard of care. In addition to the cardiac data, patients demonstrated a statistically and clinically relevant benefit in the PUL v2.0 total score when 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. In conclusion, the results of this study showed sustained cardiac and skeletal benefits after 3 years of continuous treatment with deramiocel. This data was previously highlighted at the PPMD Annual Meeting in June 2024.