Capricor Therapeutics Reports Long-Term Benefits of CAP-1002 in Duchenne Muscular Dystrophy Study

Capricor Therapeutics, a biotechnology company specializing in cell and exosome-based therapies, has reported promising results from its ongoing HOPE-2 open-label extension (OLE) study. The study focuses on their lead product, deramiocel (CAP-1002), intended for the treatment of Duchenne muscular dystrophy (DMD).

The latest data from the HOPE-2 OLE study revealed significant improvements in several cardiac measures, including left ventricular ejection fraction (LVEF), left ventricular end systolic volume (LVESV), and left ventricular end diastolic volume (LVEDV). These indicators are crucial for assessing cardiac function and predicting long-term patient outcomes. Notably, patients with higher ejection fractions (greater than 45%) at the start of the HOPE-2 randomized trial exhibited more pronounced improvements. This finding emphasizes the importance of early intervention in preserving cardiac function and slowing down cardiomyopathy, a leading cause of mortality in DMD patients. Currently, there is no approved treatment specifically for DMD cardiomyopathy, highlighting the urgent need for new therapies.

Additionally, as previously reported, patients in the study experienced a statistically significant improvement of 3.7 points in the Performance of the Upper Limb (PUL v2.0) score compared to an external comparator dataset of similar DMD patients. The continued favorable safety profile of deramiocel over the long term further underscores its potential as a valuable treatment for DMD.

These results will be presented at the Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference in Orlando, Florida, on June 29, 2024.

Key outcomes from the 3-year HOPE-2 OLE study include:

- Primary Endpoint (Skeletal Muscle Function):
- Performance of Upper Limb (PUL v2.0) showed a 3.7-point improvement compared to the external comparator (p<0.001).

- Secondary Endpoints (Cardiac Function):
- LVEF saw a positive change of 1.2% overall and 3.0% in patients with over 45% LVEF at the end of HOPE-2.
- LVESV and LVEDV showed reductions of 2.4 mL/m² and 5.7 mL/m², respectively, with greater improvements in patients with higher LVEF.

These cardiac outcomes were measured using magnetic resonance imaging (cMRI).

Dr. Linda Marbán, CEO of Capricor, highlighted the significance of these findings, noting the sustained skeletal and cardiac benefits after three years of continuous deramiocel treatment. She emphasized the potential long-term advantages this therapy could offer to DMD patients. Based on the HOPE-2 OLE data and previous clinical results, Capricor plans to discuss options with the FDA to expedite their Biologics License Application (BLA) filing. The company aims to bring deramiocel to patients as swiftly as possible and will continue to update the community on their progress.

The PPMD Annual Conference is the largest international event dedicated to the latest research, clinical trials, and care initiatives for DMD. The meeting attracts researchers, caregivers, and patients united in their efforts to combat DMD.

About the HOPE-2 Open Label Extension (OLE) Study:
The HOPE-2 study was a randomized, double-blind, placebo-controlled Phase 2 clinical trial of deramiocel in boys and young men with DMD. Patients received either deramiocel or placebo intravenously every three months. Data from 20 patients (12 placebo, 8 treated) were analyzed at the 12-month point and published in The Lancet. Following a gap phase, eligible patients entered the HOPE-2 OLE study, receiving deramiocel infusions every three months. The study previously met its primary endpoint at the one-year mark and continues into its fourth year, with ongoing monitoring of safety, cardiac, and functional performance.

About Duchenne Muscular Dystrophy (DMD):
DMD is a severe genetic disorder characterized by progressive muscle weakness and chronic inflammation affecting skeletal, heart, and respiratory muscles. It typically results in mortality around the age of 30. DMD affects approximately one in every 3,500 male births, with an estimated 15,000-20,000 patients in the U.S. The disorder stems from impaired production of dystrophin, a structural protein in muscle cells, leading to significant cell damage and muscle cell death. Current treatment options are limited, and there is no cure.

About Capricor Therapeutics:
Capricor Therapeutics (NASDAQ: CAPR) is dedicated to developing cell and exosome-based therapies for rare diseases. Their lead product, deramiocel (CAP-1002), is in Phase 3 clinical development for DMD. Capricor is also exploring exosome technology for various medical applications. The company is committed to advancing transformative treatments for patients in need.

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