"Pseudosubstrate Envelope"/Free Energy Perturbation-Guided Design and Mechanistic Investigations of Benzothiazole HIV Capsid Modulators with High Ligand Efficiency

J Med Chem. 2024 Nov 14;67(21):19057-19076. doi: 10.1021/acs.jmedchem.4c01544. Epub 2024 Oct 17.

Abstract

Based on our proposed "pseudosubstrate envelope" concept, 25 benzothiazole-bearing HIV capsid protein (CA) modulators were designed and synthesized under the guidance of free energy perturbation technology. The most potent compound, IC-1k, exhibited an EC50 of 2.69 nM against HIV-1, being 393 times more potent than the positive control PF74. Notably, IC-1k emerged as the highest ligand efficiency (LE = 0.32) HIV CA modulator, surpassing that of the approved drug lenacapavir (LE = 0.21). Surface plasmon resonance assay and crystallographic analysis confirmed that IC-1k targeted HIV-1 CA within the chemical space of the "pseudosubstrate envelope". Further mechanistic studies revealed a dual-stage inhibition profile: IC-1k disrupted early-stage capsid-host-factor interactions and promoted late-stage capsid misassembly. Preliminary pharmacokinetic evaluations demonstrated significantly improved metabolic stability in human liver microsomes for IC-1k (T1/2 = 91.3 min) compared to PF74 (T1/2 = 0.7 min), alongside a favorable safety profile. Overall, IC-1k presents a promising lead compound for further optimization.

MeSH terms

  • Anti-HIV Agents* / chemical synthesis
  • Anti-HIV Agents* / chemistry
  • Anti-HIV Agents* / pharmacokinetics
  • Anti-HIV Agents* / pharmacology
  • Benzothiazoles* / chemical synthesis
  • Benzothiazoles* / chemistry
  • Benzothiazoles* / pharmacokinetics
  • Benzothiazoles* / pharmacology
  • Capsid Proteins* / chemistry
  • Capsid Proteins* / metabolism
  • Drug Design*
  • HIV-1* / drug effects
  • Humans
  • Ligands
  • Microsomes, Liver / metabolism
  • Structure-Activity Relationship
  • Thermodynamics

Substances

  • Ligands
  • Benzothiazoles
  • Anti-HIV Agents
  • Capsid Proteins
  • benzothiazole