Therapeutics: Farnesoid X receptor (FXR; NR1H4)

Mouse studies suggest FXR agonists could help treat MS. In the experimental autoimmune encephalomyelitis (EAE) mouse model of MS, the endogenous FXR ligand chenodeoxycholic acid or the FXR agonist obeticholic acid decreased disease severity, splenocyte levels of proinflammatory cytokines and T cell proliferation compared with vehicle. Next steps include testing FXR agonists in models of progressive MS and neuromyelitis optica.

Intercept Pharmaceuticals Inc. and Sumitomo Dainippon Pharma Co. Ltd. have obeticholic acid (OCA; DSP-1747) in registration to treat primary biliary cirrhosis, Phase III testing to treat non-alcoholic steatohepatitis (NASH) and Phase II testing to treat biliary atresia and primary sclerosing cholangitis...