Capricor Therapeutics is developing deramiocel, an investigational drug to treat cardiomyopathy in people with Duchenne muscular dystrophy (DMD). Cardiomyopathy or heart disease is a main cause of death (also called mortality) for people with DMD. Cardiomyopathy can involve different areas of the heart. Deramiocel is a cell-based product developed to reduce heart inflammation and fibrosis associated with DMD. It is also pro-regenerative, which mean it encourages regeneration of cells.
All people in the study who were treated with deramiocel had improvements in heart symptoms. People with DMD heart had less severe symptoms at the beginning of the study had even more improvement than the overall treated group. People with DMD in an external comparator (EC) group (in this case, a group of people with DMD who were previously studied) who were NOT treated with deramiocel had worsening cardiac function based on all assessments. This suggests that long-term treatment with deramiocel not only improves heart function for all people with DMD, but especially for those who are treated before the symptoms get severe.
“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.”
Linda Marbán, Ph.D., Capricor’s Chief Executive Officer
Please visit the links below to access the full Capricor Press Release and view the full poster presentation.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: