Catabasis Pharmaceuticals, Inc. announced the publication of preclinical data on the edasalonexent program, a potential disease-modifying therapy for Duchenne muscular dystrophy (DMD). The preclinical data demonstrate that edasalonexent (CAT-1004) and an analog, CAT-1041, oral inhibitors of NF-kB, are effective. Edasalonexent (CAT-1004) and CAT-1041, which represent a novel class of NF-kB inhibitors, were evaluated in both mdx mouse and golden retriever muscular dystrophy (GRMD) dog models of DMD. Initial studies with edasalonexent and CAT-1041 in mdx mice demonstrated nearly identical in vitro and in vivo efficacy, and CAT-1041 was selected for further evaluation in the treatment of dystrophic muscle. In vivo, CAT-1041 effectively improved the phenotype of mdxmice undergoing voluntary wheel running, in terms of activity, muscle mass and function, damage, inflammation, fibrosis and cardiac pathology. The researchers identified significant increases in dysferlin as a possible contributor to the protective effect of CAT-1041 against sarcolemmal damage. Furthermore, CAT-1041 improved the more severe GRMD phenotype in a canine case study, where muscle mass and diaphragm function were maintained in a treated GRMD dog. Edasalonexent (CAT-1004) is an oral small molecule that has the potential to be a disease-modifying therapy for all patients affected by Duchenne muscular dystrophy (DMD or Duchenne), regardless of their underlying mutation. Edasalonexent inhibits NF-kB, a protein that is activated in Duchenne and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration. In animal models of DMD, edasalonexent produced beneficial effects in skeletal, diaphragm and cardiac muscle and improved function. The FDA has granted orphan drug, fast track and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. As previously reported safety, tolerability and reduction in NF-kB activity in Phase 1 trials in adults. From Part A of the MoveDMD trial, it is reported that edasalonexent was generally well tolerated with no safety signals observed and NF-kB target engagement. Pharmacokinetic results demonstrated edasalonexent plasma exposure levels consistent with those previously observed in adults, at which inhibition of NF-kB was observed.