BioCentury
ARTICLE | Clinical News

Catabasis' DMD candidate slows disease progression in Phase I/II

October 6, 2017 7:09 PM UTC

Catabasis Pharmaceuticals Inc. (NASDAQ:CATB) reported data from the open-label extension of the Phase I/II MoveDMD trial showing that edasalonexent (CAT-1004) slowed disease progression in boys ages 4-7 with Duchenne muscular dystrophy (DMD). The company plans to start a registrational Phase III trial in 1H18 in pediatric patients with DMD regardless of mutation type.

Edasalonexent is a conjugate of salicylate and docosahexaenoic acid (DHA) that inhibits activated nuclear factor of kappa light polypeptide gene enhancer in B cells 1 (NF-kB; NFKB1; p105; p50). Catabasis said the therapy significantly improved muscle inflammation at 12 weeks and at last observation compared to a mean 39-week control period prior to treatment, as measured by MRI of lower leg muscles (p<0.05). Edasalonexent also significantly reduced levels of 4 muscle enzymes -- creatine kinase, alanine aminotransferase (ALT), aspartate aminotransferase (AST) and lactate dehydrogenase (LDH) -- compared to baseline at 12 weeks (p<0.05)...