Capricor Therapeutics Reports Positive Long-Term HOPE-2 OLE Data in DMD at 2024 World Muscle Congress

Capricor Therapeutics, a biotechnology firm focusing on innovative cell and exosome-based treatments for rare diseases, released encouraging three-year safety and efficacy data from its ongoing HOPE-2 open label extension (OLE) study. This study evaluates deramiocel, their leading therapy for Duchenne muscular dystrophy (DMD). This data was featured at the 29th Annual Congress of the World Muscle Society held in Prague, Czechia, from October 8-12, 2024.

Dr. Craig McDonald, the National Principal Investigator at the University of California, Davis, commented on the significance of these findings, highlighting the sustained long-term benefits for cardiac function in DMD patients. Cardiomyopathy, the primary cause of death in DMD, currently has no approved treatments. The data also demonstrated lasting improvements in skeletal muscle function over a three-year period, as measured by the PUL v2.0 scale. According to Dr. McDonald, no other treatment for DMD has shown such promising safety and efficacy data over a similar timeframe.

Linda Marbán, Ph.D., Capricor’s CEO, emphasized the potential impact of these results on DMD patients. The data indicates prolonged cardiac and skeletal muscle benefits from continuous deramiocel treatment over three years, underscoring its potential long-term efficacy. This dataset will be a key part of Capricor’s Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for deramiocel’s approval to treat DMD cardiomyopathy. Capricor has been collaborating closely with the FDA to expedite deramiocel’s approval, recognizing the urgency in preserving heart function, which, once lost, is difficult to restore. As more therapies emerge that might influence the progression of skeletal muscle loss in DMD, maintaining cardiac function becomes increasingly critical. Deramiocel is anticipated to be a lifelong treatment, administered quarterly, and it has significant potential for widespread use in the DMD cardiomyopathy treatment landscape.

The three-year HOPE-2 OLE study data showed improvements in multiple cardiac function measures, including left ventricular ejection fraction (LVEF%) and indexed volumes, which are important indicators of long-term cardiac health. A clear distinction in treatment outcomes was noted in patients with ejection fractions above 45% at the study’s end, suggesting that early and sustained intervention is vital in mitigating DMD cardiomyopathy's impacts. To assess the data’s relevance to disease progression, a natural history dataset was used to compare patients treated with deramiocel to those receiving standard care. Additionally, patients treated with deramiocel showed a statistically and clinically significant benefit in the PUL v2.0 total score compared to an external comparator dataset of similar DMD patients. The HOPE-2 OLE study continues to demonstrate deramiocel’s favorable safety profile over long-term treatment.

In summary, the study’s results indicate sustained cardiac and skeletal muscle benefits after three years of continuous deramiocel treatment. This data, previously highlighted at the PPMD Annual Meeting in June 2024, reinforces deramiocel's potential as an effective long-term treatment for DMD.

Deramiocel (CAP-1002) is composed of allogeneic cardiosphere-derived cells (CDCs), a type of stromal cell shown in studies to have strong immunomodulatory, antifibrotic, and regenerative effects in dystrophinopathy and heart failure. CDCs release exosomes which target macrophages, prompting them to adopt a healing rather than a pro-inflammatory role. CDCs have been the focus of over 100 peer-reviewed publications and have been tested in over 200 human subjects across several clinical trials.

Deramiocel for DMD has received Orphan Drug Designation, and its regulatory pathway is supported by RMAT (Regenerative Medicine Advanced Therapy Designation). If Capricor obtains FDA approval for deramiocel in treating DMD, the company may also receive a Priority Review Voucher (PRV) due to its prior receipt of a rare pediatric disease designation.

Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle weakness and chronic inflammation, affecting skeletal, heart, and respiratory muscles, with a median mortality age of around 30 years. DMD occurs in approximately one in every 3,500 male births, with an estimated patient population of 15,000-20,000 in the United States. The disease is caused by a lack of functional dystrophin, a protein crucial for muscle structure, leading to muscle cell damage and death. Current treatment options are limited, and there is no cure.

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is at the forefront of developing cell and exosome-based therapies for rare diseases. Their leading product candidate, deramiocel, has shown significant promise in treating dystrophinopathies and heart disease, and is currently in Phase 3 clinical development for DMD. Capricor is also exploring its exosome technology for various therapeutic applications using its proprietary StealthX™ platform.