losmapimod

Losmapimod is an orally bioavailable small molecule inhibitor of the alpha and beta isoforms of p38 mitogen-activated protein kinase (MAPK). It was originally developed by GSK for cardiovascular diseases, but did not demonstrate efficacy in that indication. The drug is now being repurposed by Fulcrum Therapeutics for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a progressive muscle-wasting disorder. Losmapimod works by selectively inhibiting p38α/β MAPK, which reduces DUX4 gene activity and subsequent toxic protein production implicated in FSHD pathogenesis. Its mechanism also results in immunomodulating and anti-inflammatory effects through inhibition of proinflammatory cytokine production such as TNF-alpha, IL-1, IL-6, and IL-8[1][4][5][6].

Other names

585543-15-3