Duchenne Muscular Dystrophy Overview

Understanding the Quality of Life for DMD Patients

Duchenne muscular dystrophy (DMD) is a rare form of muscular dystrophy which results in muscle degeneration and premature death. DMD affects approximately 1 in 3,600 male infants worldwide, and it is estimated that approximately 15,000 to 20,000 boys and young men are living with the disease in the United States and approximately 200,000 worldwide.1 DMD results from the lack of functional dystrophin protein caused by a gene mutation. The lack of dystrophin, an essential structural component of muscle cells, causes them to have increased susceptibility to damage and to die progressively. Additionally, the absence of dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD.

Patients with DMD experience progressive muscle weakness and degeneration starting at an early age. Generally, a loss of ambulation occurs after the first decade of life and, eventually, the patients suffer respiratory and cardiac failure. Their lifespan is abbreviated and averages less than three decades. Very few therapeutic options exist and the high annual cost of care for patients with DMD increases with disease progression. We therefore believe that DMD represents a significant market opportunity for our lead product candidate, CAP-1002.

The regulatory pathway for CAP-1002 is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) and Orphan Drug Designation. If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher based on its designation as a rare pediatric disease.

1 DMD prevalence estimate

Duchenne Muscular Dystrophy Trajectory