Capricor Therapeutics Inc (NASDAQ: CAPR) announced that its HOPE-2 Phase II trial had received positive results from The Lancet. The latter has published results for the study of CAP-1002. This therapy is for treating Duchenne muscular dystrophy in its advanced stages.
Capricor’s study found that the therapy could improve the functioning of cardiac and skeletal muscles in young men after the researchers administered four doses in a year.
According to the lead study author, chair, and professor of Capricor’s Physical Medicine and Rehabilitation Department, UC Davis, past trials for the disease have not looked into therapies for non-ambulatory and severely affected patients even though they bear the most significant burden of Duchenne. For this reason, the company intends to find medication to help them.
The approval of the drug could be ground-breaking
Moreover, the company believes that having The Lancet, a highly respected publication, give positive results for CAP-1002 is an accomplishment. The publication from The Lancet also shows the efficacy of the treatment for different disease stages.
The CEO of Capricor, Linda Marbán, adds that the company’s researchers have found CAP-1002 to be safe and efficient for treating Duchenne. They have also found positive results for the drug from two different trials. Marbán adds that CAP-1002 could be a life-altering drug and vastly improve patients’ lives.
Marbán states that the company will leverage its new partnership with Nippon Shinyaku to market and distribute the product in the U.S. Furthermore, Nippon Shinyanku has experience in rare diseases, especially Duchenne. The company is now beginning its HOPE-3 Phase III clinical trial of CAP-1002.
How the company carried out the study
The HOPE-2 Phase II clinical trial was a placebo-controlled, double-blind, randomized study. The trial tested CAP-1002 in young men and boys with advanced stages Duchenne, accounting for 50% of real-world patients.
Duchesne, which is genetic, is characterized by an increase in chronic inflammation and weakness of the respiratory, cardia, and skeletal muscles. These patients are usually paralyzed as teenagers. They could also die due to cardiac and respiratory problems before they reach 30.
The team noted positive results in young men after a year of therapy. They monitored their progress by evaluating the functioning of their wrist and hand (distal), elbow (mid, and shoulder (high) in the upper limbs.
