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Capricor Therapeutics Announces Positive Long-Term Data from HOPE-2 OLE Study in Duchenne Muscular Dystrophy at 2024 World Muscle Society Congress
-Data Supports Deramiocel’s Sustained Efficacy and Safety in Treating DMD- -Improvements Seen in Multiple Cardiac and Skeletal Endpoints Demonstrating
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"-Data Supports Deramiocel’s Sustained Efficacy and Safety in Treating DMD- -Improvements Seen in Multiple Cardiac and Skeletal Endpoints Demonstrating Stabilization of Cardiac and Skeletal Muscle Function Over 3 Years of Treatment- -Company on Track to Fully Submit Biologics License Application (BLA) by End of 2024- SAN DIEGO, Oct. 11, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced positive 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). The data was highlighted in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society (WMS), 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.” “These results are extremely impactful for patients living with DMD as they showed sustained cardiac and skeletal muscle benefits after 3 years of continuous treatment with deramiocel, which underscores the potential long-term efficacy this therapy can offer. As we previously announced, this dataset will be one of the key elements of our BLA submission to the U.S. Food and Drug Administration (FDA), for approval of deramiocel to treat patients with DMD cardiomyopathy,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “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. 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. We expect deramiocel to be a lifelong treatment, with an i...