Catabasis Pharmaceuticals, a company focused on clinical-stage drug development that is supported by a pharmacology technology platform pathway, recently announced that Joanne Donovan, Chief Medical Officer, was invited by Parent Project Muscular Dystrophy (PPMD) to present the latest updates on the upcoming MoveDMD trial.
MoveDMD is a Phase ½ clinical trial for CAT-1004 to address treatment for boys suffering from Duchenne Muscular Dystrophy (DMD) with the dystrophin mutation. The presentation will be held Wednesday, May 13, between 1pm and 2pm ET. This webinar is designed for parents of DMD boys in order to describe the MoveDMD trial design and to provide enrollment guidance. It can be accessed through the www.readytalk.com through the 9449985 code. The audio dial-in can be accessed by dialing 1-866-740-1260 and by providing the same code.
Duchenne muscular dystrophy is an inherited disorder caused by a defective gene called dystrophin. The disorder is characterized by a progressive skeletal muscle weakness that leads to chronic inflammation and the loss of muscle cells and tissue, compromising locomotion. Duchenne muscular dystrophy has a rapid progression and affects mainly boys.
CAT-1004 works through inhibiting the activated NF-kB protein responsible for the coordination to cellular response to stress, muscular damage, and inflammation and it is also crucial in muscle health. It is an investigational drug that drives muscle regeneration. In DMD animal models, researchers observed that CAT-1004 inhibited activated NF-kB, managed to reduce muscle degeneration and inflammation, and increased muscle regeneration. In Phase 1 adult clinical trials, it was observed that CAT-1004 inhibited NF-kB, was well tolerated and no safety concerns were observed.
In other recent DMD news, a chain of convenience stores has joined forces with the Muscular Dystrophy Association (MDA) to help improve the quality of life of pediatric patients suffering from muscular dystrophy. Over 260 Western Refining convenience stores have joined the campaign to raise funding to allow more children to participate in the MDA Summer Camp mobile program.