Capricor Reports Positive 3-Year Results from HOPE-2 Study of CAP-1002 in Duchenne Muscular Dystrophy

Capricor Therapeutics, a biotechnology company listed on NASDAQ as CAPR, has announced promising three-year results from its ongoing HOPE-2 open-label extension (OLE) study. This study focuses on the use of CAP-1002 for treating Duchenne muscular dystrophy (DMD). The data shows sustained benefits in skeletal muscle function, measured by the Performance of Upper Limb (PUL 2.0), and stabilization in cardiac function, measured by Left Ventricular Ejection Fraction (LVEF).

The HOPE-2 OLE study, an open-label extension of a prior double-blind, placebo-controlled Phase 2 study, reports that patients treated with CAP-1002 exhibited a lesser decline in PUL 2.0 scores compared to an external comparator group from Cincinnati Children’s Hospital Medical Center. Specifically, CAP-1002 patients had a decline of 4.1 points versus the 7.8 points in the comparator group, marking a statistically significant difference (p < 0.001). Additionally, the stabilization of LVEF in CAP-1002 patients indicates a potential preservation of cardiac function, contrasting the expected decline seen in untreated DMD patients.

Dr. Linda Marbán, CEO of Capricor, expressed optimism about these findings. She emphasized that the results reinforce the long-term impact of CAP-1002 on both skeletal and cardiac functions in DMD patients. Marbán noted that these positive outcomes were shared with the U.S. Food and Drug Administration (FDA) during a Type-B meeting in May 2024. The company anticipates releasing further details of this meeting later in the month. Capricor is also preparing to announce topline results from their Phase 3 HOPE-3 pivotal trial in the fourth quarter of 2024.

The HOPE-2 study originally included boys and young men with DMD, who received either CAP-1002 or a placebo via intravenous infusion every three months. Following the completion of the initial study, there was a gap phase of approximately 392 days on average, after which eligible patients entered the HOPE-2-OLE study. Here, they continued receiving CAP-1002 every three months, with the study already having met its primary endpoint at the one-year mark.

Duchenne muscular dystrophy is a severe genetic disorder that causes progressive muscle weakness and inflammation of skeletal, heart, and respiratory muscles, often leading to mortality around the age of 30. It affects approximately one in every 3,500 male births in the U.S., amounting to an estimated 15,000-20,000 patients. The disease is caused by mutations that impair the production of dystrophin, a protein essential for muscle function. The lack of functional dystrophin leads to muscle cell damage and death, and eventually fibrotic replacement.

Capricor Therapeutics is committed to developing innovative cell and exosome-based treatments for rare diseases. Their leading product, CAP-1002, is an allogeneic cardiac-derived cell therapy currently in Phase 3 clinical development for DMD. CAP-1002 has demonstrated immunomodulatory, antifibrotic, and regenerative properties in preclinical and clinical studies. Additionally, Capricor is advancing its exosome technology through the StealthX™ platform, aimed at delivering treatments for various diseases by targeting oligonucleotides, proteins, and small molecules.

The promising results from the HOPE-2 OLE study underscore the potential of CAP-1002 as a transformative therapy for DMD, providing hope for improved treatment options for those affected by this devastating disease. The company remains dedicated to advancing their clinical programs and expects significant developments in the near future.