Dermamiocel appears to effectively prevent cardiac scarring (fibrosis) in patients with Duchenne muscular dystrophy (DMD), according to a recently published press release.
Despite the emergence of new therapies, cardiac disease (cardiomyopathy) remains a leading cause of mortality for individuals with DMD. There is a critical unmet medical need for treatments that can effectively address the cardiac manifestations of the disease while also preserving skeletal muscle function.
A current research focus is on transformative cell-based therapeutics that aim to modulate the immune response and promote tissue repair.
Read more about therapies for DMD
To evaluate these potential benefits, researchers conducted additional analyses of the Phase 3 HOPE-3 clinical trial, a randomized, double-blind, placebo-controlled study investigating Deramiocel (CAP-1002). The trial assessed treatment efficacy using markers of myocardial fibrosis, cardiac structure and real-world physical performance to determine the drug’s impact on both the heart and skeletal muscle systems in patients.
The results of the cardiac magnetic resonance imaging (MRI) analyses demonstrated that Deramiocel significantly impacted cardiac structure by reducing the progression of myocardial fibrosis.
Furthermore, patients with baseline cardiomyopathy taking the drug experienced a significant improvement in their capacity to pump blood out of the heart, known as left ventricular ejection fraction (LVEF), compared to those receiving a placebo.
These findings suggest that the therapy may directly stabilize or improve heart function in patients already exhibiting signs of heart disease.
“These newly released data provide additional clinically meaningful evidence supporting the potential impact of Deramiocel on the daily lives and long-term outcomes of patients living with Duchenne,” said Linda Marbán, chief executive officer of Capricor. “With our Biologics License Application currently under FDA review and a PDUFA target action date of August 22, 2026, we remain focused on working with the FDA toward potential approval and bringing this therapy to patients as quickly as possible.”
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