Discovery of Tropifexor (LJN452), a Highly Potent Non-bile Acid FXR Agonist for the Treatment of Cholestatic Liver Diseases and Nonalcoholic Steatohepatitis (NASH)

J Med Chem. 2017 Dec 28;60(24):9960-9973. doi: 10.1021/acs.jmedchem.7b00907. Epub 2017 Dec 8.

Abstract

The farnesoid X receptor (FXR) is a nuclear receptor that acts as a master regulator of bile acid metabolism and signaling. Activation of FXR inhibits bile acid synthesis and increases bile acid conjugation, transport, and excretion, thereby protecting the liver from the harmful effects of bile accumulation, leading to considerable interest in FXR as a therapeutic target for the treatment of cholestasis and nonalcoholic steatohepatitis. We identified a novel series of highly potent non-bile acid FXR agonists that introduce a bicyclic nortropine-substituted benzothiazole carboxylic acid moiety onto a trisubstituted isoxazole scaffold. Herein, we report the discovery of 1 (tropifexor, LJN452), a novel and highly potent agonist of FXR. Potent in vivo activity was demonstrated in rodent PD models by measuring the induction of FXR target genes in various tissues. Tropifexor has advanced into phase 2 human clinical trials in patients with NASH and PBC.

MeSH terms

  • Administration, Oral
  • Animals
  • Benzothiazoles / pharmacology*
  • Benzothiazoles / therapeutic use
  • Biological Availability
  • Cholestasis / drug therapy*
  • Dogs
  • Drug Evaluation, Preclinical / methods
  • Fibroblast Growth Factors / genetics
  • Gene Expression Regulation / drug effects
  • Humans
  • Isoxazoles / pharmacology*
  • Isoxazoles / therapeutic use
  • Male
  • Microsomes, Liver / drug effects
  • Non-alcoholic Fatty Liver Disease / drug therapy*
  • Piperidines / chemistry
  • Rats, Sprague-Dawley
  • Receptors, Cytoplasmic and Nuclear / agonists*
  • Receptors, Cytoplasmic and Nuclear / genetics
  • Receptors, Cytoplasmic and Nuclear / metabolism
  • Structure-Activity Relationship
  • Triglycerides / blood

Substances

  • Benzothiazoles
  • FGF19 protein, rat
  • Isoxazoles
  • Piperidines
  • Receptors, Cytoplasmic and Nuclear
  • Triglycerides
  • farnesoid X-activated receptor
  • Fibroblast Growth Factors
  • piperidine
  • tropifexor