Design and Structural Optimization of Dual FXR/PPARδ Activators

J Med Chem. 2020 Aug 13;63(15):8369-8379. doi: 10.1021/acs.jmedchem.0c00618. Epub 2020 Jul 20.

Abstract

Nonalcoholic steatohepatitis (NASH) is considered as severe hepatic manifestation of the metabolic syndrome and has alarming global prevalence. The ligand-activated transcription factors farnesoid X receptor (FXR) and peroxisome proliferator-activated receptor (PPAR) δ have been validated as molecular targets to counter NASH. To achieve robust therapeutic efficacy in this multifactorial pathology, combined peripheral PPARδ-mediated activity and hepatic effects of FXR activation appear as a promising multitarget approach. We have designed a minimal dual FXR/PPARδ activator scaffold by rational fusion of pharmacophores derived from selective agonists. Our dual agonist lead compound exhibited weak agonism on FXR and PPARδ and was structurally refined to a potent and balanced FXR/PPARδ activator in a computer-aided fashion. The resulting dual FXR/PPARδ modulator comprises high selectivity over related nuclear receptors and activates the two target transcription factors in native cellular settings.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Drug Design
  • Drug Discovery
  • Humans
  • Ligands
  • Molecular Docking Simulation
  • Non-alcoholic Fatty Liver Disease / drug therapy
  • Non-alcoholic Fatty Liver Disease / metabolism
  • PPAR delta / agonists*
  • PPAR delta / metabolism
  • Receptors, Cytoplasmic and Nuclear / agonists*
  • Receptors, Cytoplasmic and Nuclear / metabolism
  • Structure-Activity Relationship

Substances

  • Ligands
  • PPAR delta
  • Receptors, Cytoplasmic and Nuclear
  • farnesoid X-activated receptor