Pocket-to-Lead: Structure-Based De Novo Design of Novel Non-peptidic HIV-1 Protease Inhibitors Using the Ligand Binding Pocket as a Template

J Med Chem. 2022 Apr 28;65(8):6157-6170. doi: 10.1021/acs.jmedchem.1c02217. Epub 2022 Apr 13.

Abstract

A novel strategy for lead identification that we have dubbed the "Pocket-to-Lead" strategy is demonstrated using HIV-1 protease as a model target. Sometimes, it is difficult to obtain hit compounds because of the difficulties in satisfying the complex pharmacophoric features. In this study, a virtual fragment hit which does not match all of the pharmacophore features but has key interactions and vectors that could grow into remaining pharmacophore features was optimized in silico. The designed compound 9 demonstrated weak but evident inhibitory activity (IC50 = 54 μM), and the design concept was proven by the co-crystal structure. Then, structure-based drug design promptly gave compound 14 (IC50 = 0.0071 μM, EC50 = 0.86 μM), an almost 10,000-fold improvement in activity from 9. The structure of the designed molecules proved to be novel with high synthetic feasibility, indicating the usefulness of this strategy to tackle tough targets with complex pharmacophore.

MeSH terms

  • Drug Design
  • HIV Protease / metabolism
  • HIV Protease Inhibitors* / chemistry
  • HIV Protease Inhibitors* / pharmacology
  • HIV-1* / metabolism
  • Ligands
  • Molecular Docking Simulation
  • Protease Inhibitors

Substances

  • HIV Protease Inhibitors
  • Ligands
  • Protease Inhibitors
  • HIV Protease
  • p16 protease, Human immunodeficiency virus 1