Design and Discovery of a Potent and Selective Inhibitor of Integrin αvβ1

J Med Chem. 2024 Jun 27;67(12):10306-10320. doi: 10.1021/acs.jmedchem.4c00743. Epub 2024 Jun 13.

Abstract

Selective inhibition of the RGD (Arg-Gly-Asp) integrin αvβ1 has been recently identified as an attractive therapeutic approach for the treatment of liver fibrosis given its function, target expression, and safety profile. Our identification of a non-RGD small molecule lead followed by focused, systematic changes to the core structure utilizing a crystal structure, in silico modeling, and a tractable synthetic approach resulted in the identification of a potent small molecule exhibiting a remarkable affinity for αvβ1 relative to several other integrin isoforms measured. Azabenzimidazolone 25 demonstrated antifibrotic efficacy in an in vivo rat liver fibrosis model and represents a tool compound capable of further exploring the biological consequences of selective αvβ1 inhibition.

MeSH terms

  • Animals
  • Benzimidazoles / chemical synthesis
  • Benzimidazoles / chemistry
  • Benzimidazoles / pharmacology
  • Crystallography, X-Ray
  • Drug Design*
  • Drug Discovery
  • Humans
  • Liver Cirrhosis / drug therapy
  • Male
  • Models, Molecular
  • Rats
  • Rats, Sprague-Dawley
  • Receptors, Vitronectin* / antagonists & inhibitors
  • Receptors, Vitronectin* / metabolism
  • Structure-Activity Relationship

Substances

  • integrin alphavbeta1
  • Receptors, Vitronectin
  • Benzimidazoles