Capricor Therapeutics, Inc. announced additional positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, deramiocel (CAP-1002), for the treatment of Duchenne muscular dystrophy. Data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures, including left ventricular ejection fraction (LVEF), as well as indexed volumes (left ventricular end systolic volume (LVESV) and left ventricular end diastolic volume (LVEDV)). These are measures of cardiac function and are considered highly relevant in terms of predicting long-term outcomes.

In addition, greater improvements in cardiac function were observed in those patients that had higher ejection fractions (> 45%) at the beginning of the HOPE-2 randomized trial. Published data supports the need for early intervention in order to maintain function and potentially slow the progression of the cardiomyopathy, one of the leading causes of death in patients with DMD. Currently, there is no approved treatment specifically for DMD cardiomyopathy, which underscores the need for additional therapies to treat DMD.

Additionally, as previously reported, patients showed a statistically significant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. The HOPE-2 OLE study continues to show a favorable safety profile for long-term treatment of deramiocel. HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor?s lead investigational therapy, deramiocel, in boys and young men who have DMD.

Study patients were treated via intravenous delivery with either deramiocel (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase.

Then all eligible patients who wished to remain on treatment entered the HOPE-2-OLE study where they receive deramiocel (150 million cells per infusion) every three months. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 (p=0.02). The HOPE-2-OLE study remains ongoing and into its fourth year and participants continue to be monitored for safety, cardiac and functional performance.

Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle.

The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure.