Capricor Therapeutics announces promising long-term results for deramiocel deramiocel in Duchenne muscular dystrophy

Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company at the forefront of developing cell and exosome-based therapies, has shared encouraging three-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study. The study investigates the effects of deramiocel (CAP-1002), Capricor’s leading therapeutic candidate, on Duchenne muscular dystrophy (DMD), particularly focusing on cardiac aspects of the disease.

The data highlighted significant improvements in key cardiac function measures such as left ventricular ejection fraction (LVEF), and volumes (left ventricular end systolic volume [LVESV] and left ventricular end diastolic volume [LVEDV]). These results are crucial as they are indicative of long-term health outcomes for DMD patients, a group currently lacking approved treatments for the cardiomyopathy associated with their condition. Remarkably, the greatest improvements were noted in patients who had higher ejection fractions at the start of the study, underscoring the potential benefits of early intervention.

Capricor’s CEO, Dr. Linda Marbán, emphasized the significance of these findings. “The results of the open label study are tremendously important for DMD patients, as they showed sustained skeletal and cardiac benefits after 3 years of continuous treatment with deramiocel,” she stated. This ongoing success has paved the way for discussions with the U.S. Food and Drug Administration (FDA) about expediting the Biologics License Application (BLA) for deramiocel, with the aim of making this promising therapy available to patients sooner.

In addition to cardiac improvements, the HOPE-2 OLE study also demonstrated a statistically significant benefit in overall patient functionality. Patients treated with deramiocel showed a significant improvement (+3.7 points, p < 0.001) in the PUL v2.0 total score compared to a similar external dataset of DMD patients.

The findings will be presented at the upcoming PPMD Annual Conference, the largest international event focused on DMD research, clinical trials, and care initiatives, now in its 30th year. This platform will allow Capricor to engage directly with the broader Duchenne community, including researchers, caregivers, and patients, to discuss these promising results and the next steps in the therapy’s development.

Originally a randomized, double-blind, placebo-controlled Phase 2 clinical trial, HOPE-2 tested deramiocel in boys and young men with DMD, treating patients via intravenous delivery either with the investigational therapy or a placebo every three months. Following a gap phase where treatment was paused, patients who chose to continue were enrolled in the HOPE-2 OLE study to receive ongoing treatment. This extension study has not only met its primary endpoint at the one-year mark but continues to show a favorable safety profile and sustained benefits into its fourth year.

As the HOPE-2 OLE study progresses into its fourth year, Capricor remains committed to monitoring participants for safety and ongoing performance in cardiac and functional measures. The continued collaboration with the FDA and the DMD community is a critical component of bringing this innovative treatment to the forefront of DMD care.