CAT-1004, a candidate treatment for Duchenne muscular dystrophy (DMD) being developed by Catabasis Pharmaceuticals, will be featured in a poster presentation at the International Congress of the World Muscle Society, taking place in Berlin, Germany between October 7 and 11. Catabasis Pharmaceuticals is a drug developer currently focused on the development of clinical stage drugs, building on a pathway pharmacology technology platform.
The session featuring CAT-1004, entitled “DMD 2 Therapeutic Evaluations and Approaches” will be presented by the chief medical officer of Catabasis, Joanne Donovan, M.D., Ph.D., and will be held on Wednesday, October 8 between 4 and 5:30 p.m CET. During the session, Dr. Donovan will address questions about the novel oral therapy that is being developed for the treatment of DMD, which works by inhibiting a key mediator of cellular injury, NF-kB.
The studies are based on the knowledge that NF-kB contributes to the development of the disease. Catabasis Pharmaceuticals is using preclinical models of DMD in order to demonstrate that CAT-1004 is able to reduce inflammation in the muscles and its consequent degeneration, as well as increased regeneration of the muscle cells. The company already concluded its phase 1 studies in adult patients that revealed the safety and tolerability of the oral therapy, as well as its effectiveness in inhibiting NF-kB activation.
Duchenne muscular dystrophy is a deadly hereditary disease that attacks one in each 3,600 men at early ages. The disease causes weakness of the muscles and loss of the muscle mass, which is eventually replaced by fat and fibrotic tissue. Common symptoms include difficulties in standing and walking. One of the main non-profit organization in the United States dedicated to support DMD is the Parent Project Muscular Dystrophy (PPMD), which has joined forces with the American Football Coaches Association in order to develop The Coach to Cure MD initiative and support young patients who are not able to play sports due to the disease.