Capricor Therapeutics Reports Positive Phase 3 Results for Duchenne Treatment Study

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Capricor Therapeutics Reports Positive Phase 3 Results for Duchenne Treatment Study

Capricor Therapeutics has announced favorable results from its Phase 3 HOPE-3 trial involving Deramiocel, a promising treatment for Duchenne muscular dystrophy (DMD). The randomized, double-blind, placebo-controlled trial consisted of 106 participants enrolled across 20 clinical sites in the United States.

Key Trial Outcomes

The HOPE-3 trial met its primary endpoint, demonstrating a significant slowing in disease progression. The Performance of Upper Limb (PUL v2.0) score showed a 54% improvement compared to placebo (p=0.03). In addition, the left ventricular ejection fraction (LVEF), a key secondary endpoint, also showed significant results with a 91% improvement (p=0.04).

Significance of Results

These outcomes suggest that Deramiocel offers both skeletal and cardiac benefits, marking it as a potential first-in-class therapy for Duchenne cardiomyopathy, a major cause of mortality in DMD patients. Linda Marbán, Chief Executive Officer of Capricor, emphasized that the trial results provide robust evidence of Deramiocel’s efficacy in improving patient outcomes for those with DMD.

Trial Design and Participant Details

Participant Age: The average age of the 106 participants was approximately 15 years.
Regimen: Participants received 150 million cells of Deramiocel or a placebo every three months over 12 months.
Cardiac Treatment: About 90% of participants were on cardiac medications, with over 75% diagnosed with cardiomyopathy.

Safety Profile

Throughout the trial, Deramiocel maintained a favorable safety and tolerability profile, consistent with previous clinical experiences. This positive outcome is crucial for potential regulatory approval, pending a comprehensive response to a prior Complete Response Letter from the FDA.

Clinical Implications

The findings are monumental as they represent the first successful Phase 3 trial in a largely non-ambulatory population with DMD. The preservation of skeletal muscle function and improvements in cardiac health could translate into better long-term survival and quality of life for patients.

Next Steps

Capricor plans to submit detailed results from the HOPE-3 trial for peer-reviewed publication and presentation at a future scientific meeting. This will further solidify the data supporting Deramiocel as a viable treatment option for Duchenne muscular dystrophy.

For those interested, a conference call regarding these findings is set for today at 8:00 a.m. ET, allowing further discussion on the implications of Deramiocel for DMD therapies.