Capricor Therapeutics Inc. (NASDAQ: CAPR) is a biotechnology firm focused on developing cell and exosome-based treatments for rare diseases like Duchenne muscular dystrophy (DMD). Capricor's stock surged more than 100% after announcing it will pursue full FDA approval for its primary drug deramiocel (CAP-1002) in this year. The medical sector company plans to file the biologics license application (BLA) in October 2024, seeking full FDA approval to treat all patients with cardiomyopathy associated with DMD.
This rare genetic neuromuscular disorder causes progressive muscle degeneration and weakness. Cardiomyopathy is the leading cause of death among patients with DMD.
Around 30% to 60% of DMD patients suffer from cardiomyopathy. DMD has roughly 300,000 known sufferers worldwide, as 1 in 3,500 boys are born with it. The total addressable market (TAM) size is expected to be around $5.5 billion by 2032.
The company operates in the medical sector, competing with the incumbent leader in DMD treatments, Sarepta Therapeutics Inc. (NASDAQ: SRPT) biotech PepGen Inc. (NASDAQ: PEPG).
DMD Has Many Complications and Symptoms
Duchenne muscular dystrophy causes muscles to degenerate and weaken due to a mutation in the gene that produces a crucial protein that maintains the structural integrity of all muscle cells called dystrophin. DMD complications range from muscle degeneration and weakness resulting in difficulty walking, paralysis and frequent falls to cardiomyopathy, which is a weakening of the heart muscle, which can result in shortness of breath, fatigue, and chest pain, to an increased risk of arrhythmias, lung and heart failure.
Respiratory problems arise from weakening respiratory muscles, resulting in breathing difficulty and ventilator support. Skeletal ...
