After the promising results of Catabasis’ Phase 1/2 MoveDMD trial (NCT02439216) and its open-label extension, edasalonexent is now being tested as a potential candidate for treatment of Duchenne in the randomized, double-blind, placebo-controlled Phase 3 PolarisDMD trial (NCT03703882). The company is recruiting boys with Duchenne ages 4-7 in the U.S.
The company is expecting to enroll 125 boys who will receive edasalonexent (a capsule of 100 mg/kg, three times a day) or a placebo for 52 weeks. The first results of PolarisDMD should be available in early 2020.
Muscular dystrophies are a group of genetic disorders characterized by gradual weakening and loss of muscle control.
Besides skeletal muscle wasting, patients with muscular dystrophies may also experience heart muscle weakening that can lead to irregular heartbeat and heart failure. Heart disease, also known as cardiomyopathy, is the major cause of death in patients with Duchenne and Becker muscular dystrophies.
The one-year collaboration between Catabasis and UT Southwestern builds upon previous findings indicating potential therapeutic benefits of edasalonexent on heart function in both patients and animal models of disease.
Edasalonexent is an experimental compound that specifically blocks the function of NF-kB, interfering with a signaling cascade that is involved in muscle breakdown and failure to repair damaged muscular tissue.
“Inhibiting NF-kB with edasalonexent offers a unique mechanism with the advantage of potentially impacting both skeletal and cardiac muscle disease in those living with Duchenne and Becker muscular dystrophies,” Andrew Nichols, chief scientific officer at Catabasis Pharmaceuticals, said in a press release.
The collaboration will focus on assessing the effects of edasalonexent treatment in a mouse model of Duchenne that also displays clear signs of heart disease.
Researchers will monitor heart activity through functional, histological, biochemical and molecular assays to examine possible clinical benefits of edasalonexent treatment. The company expects the first results of the study to be available in late 2019.
“There is significant unmet medical need for therapies that could treat both the skeletal and cardiac muscle disease in Duchenne and Becker muscular dystrophies,” said Pradeep Mammen, MD, medical director of the neuromuscular cardiomyopathy clinic at the University of Texas Southwestern. “I have dedicated my career to improving the lives of patients with heart failure, and I look forward to helping advance the understanding of edasalonexent and how it could benefit patients in the future.”