Discovery of Triazolopyrimidine Derivatives as Selective P2X3 Receptor Antagonists Binding to an Unprecedented Allosteric Site as Evidenced by Cryo-Electron Microscopy

J Med Chem. 2024 Aug 22;67(16):14443-14465. doi: 10.1021/acs.jmedchem.4c01214. Epub 2024 Aug 5.

Abstract

The P2X3 receptor (P2X3R), an ATP-gated cation channel predominantly expressed in C- and Aδ-primary afferent neurons, has been proposed as a drug target for neurological inflammatory diseases, e.g., neuropathic pain, and chronic cough. Aiming to develop novel, selective P2X3R antagonists, tetrazolopyrimidine-based hit compound 9 was optimized through structure-activity relationship studies by modifying the tetrazole core as well as side chain substituents. The optimized antagonist 26a, featuring a cyclopropane-substituted triazolopyrimidine core, displayed potent P2X3R-antagonistic activity (IC50 = 54.9 nM), 20-fold selectivity versus the heteromeric P2X2/3R, and high selectivity versus other P2XR subtypes. Noncompetitive P2X3R blockade was experimentally confirmed by calcium influx assays. Cryo-electron microscopy revealed that 26a stabilizes the P2X3R in its desensitized state, acting as a molecular barrier to prevent ions from accessing the central pore. In vivo studies in a rat neuropathic pain model (spinal nerve ligation) showed dose-dependent antiallodynic effects of 26a, thus presenting a novel, promising lead structure.

MeSH terms

  • Allosteric Site
  • Animals
  • Cryoelectron Microscopy*
  • Drug Discovery
  • Humans
  • Male
  • Neuralgia / drug therapy
  • Purinergic P2X Receptor Antagonists* / chemical synthesis
  • Purinergic P2X Receptor Antagonists* / chemistry
  • Purinergic P2X Receptor Antagonists* / pharmacology
  • Pyrimidines* / chemical synthesis
  • Pyrimidines* / chemistry
  • Pyrimidines* / pharmacology
  • Rats
  • Rats, Sprague-Dawley
  • Receptors, Purinergic P2X3* / metabolism
  • Structure-Activity Relationship
  • Triazoles* / chemical synthesis
  • Triazoles* / chemistry
  • Triazoles* / pharmacology

Substances

  • Purinergic P2X Receptor Antagonists
  • Pyrimidines
  • Receptors, Purinergic P2X3
  • Triazoles