Design, synthesis, and biological evaluation of potent dual agonists of nuclear and membrane bile acid receptors

J Med Chem. 2014 Feb 13;57(3):937-54. doi: 10.1021/jm401873d. Epub 2014 Jan 17.

Abstract

Bile acids exert genomic and nongenomic effects by interacting with membrane G-protein-coupled receptors, including the bile acid receptor GP-BAR1, and nuclear receptors, such as the farnesoid X receptor (FXR). These receptors regulate overlapping metabolic functions; thus, GP-BAR1/FXR dual agonists, by enhancing the biological response, represent an innovative strategy for the treatment of enteroendocrine disorders. Here, we report the design, total synthesis, and in vitro/in vivo pharmacological evaluation of a new generation of dual bile acid receptor agonists, with the most potent compound, 19, showing promising pharmacological profiles. We show that compound 19 activates GP-BAR1, FXR, and FXR regulated genes in the liver, increases the intracellular concentration of cAMP, and stimulates the release of the potent insulinotropic hormone GLP-1, resulting in a promising drug candidate for the treatment of metabolic disorders. We also elucidate the binding mode of the most potent dual agonists in the two receptors through a series of computations providing the molecular basis for dual GP-BAR1/FXR agonism.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cholanes / chemical synthesis*
  • Cholanes / chemistry
  • Cholanes / pharmacology
  • Drug Design
  • HEK293 Cells
  • Humans
  • Hypoglycemic Agents / chemical synthesis*
  • Hypoglycemic Agents / chemistry
  • Hypoglycemic Agents / pharmacology
  • Molecular Docking Simulation
  • Protein Binding
  • Receptors, Cytoplasmic and Nuclear / agonists*
  • Receptors, Cytoplasmic and Nuclear / chemistry
  • Receptors, G-Protein-Coupled / agonists*
  • Receptors, G-Protein-Coupled / chemistry
  • Stereoisomerism
  • Structure-Activity Relationship
  • Transcriptional Activation

Substances

  • Cholanes
  • GPBAR1 protein, human
  • Hypoglycemic Agents
  • Receptors, Cytoplasmic and Nuclear
  • Receptors, G-Protein-Coupled
  • farnesoid X-activated receptor