Capricor Therapeutics Inc. (NASDAQ:CAPR) has announced promising topline results from its pivotal Phase 3 HOPE-3 trial, evaluating the efficacy of Deramiocel, an investigational cell therapy aimed at treating Duchenne muscular dystrophy (DMD). The results have led to a significant rally in the company’s stock, with shares rising approximately 8.96% to $6.93 in premarket trading.
Duchenne muscular dystrophy is a severe genetic disorder that leads to rapid muscle degeneration, primarily affecting boys. The HOPE-3 trial was conducted with a randomized group of 106 participants who received either intravenous Deramiocel at 150 million cells per infusion or a placebo every three months for a duration of 12 months. The average age of participants was around 15 years.
The trial results demonstrated that nearly 54% of the patients experienced a slowdown in skeletal muscle disease progression. Furthermore, about 91% of participants showed improvement in their heart function, specifically regarding cardiomyopathy.
Significant Findings from HOPE-3 Trial
Craig McDonald, an investigator involved in the HOPE-3 trial, expressed his enthusiasm, stating, “The HOPE-3 study is the first-ever Phase 3 trial in a largely non-ambulatory population with DMD to successfully meet its primary endpoint.” He emphasized the importance of the trial’s findings in supporting the development of innovative therapies for DMD.
Jonathan Soslow, a Professor of Pediatrics (Cardiology) at Vanderbilt University Medical Center, highlighted the significance of preserving left ventricular ejection fraction among patients treated with Deramiocel. “The statistically and clinically significant preservation of left ventricular ejection fraction in patients treated with Deramiocel underscores the potential of Deramiocel to address one of the most critical aspects of the disease,” he noted.
Regulatory Context and Future Prospects
In July, Capricor Therapeutics received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA)
