Discovery of a Biased Allosteric Modulator for Cannabinoid 1 Receptor: Preclinical Anti-Glaucoma Efficacy

J Med Chem. 2021 Jun 24;64(12):8104-8126. doi: 10.1021/acs.jmedchem.1c00040. Epub 2021 Apr 7.

Abstract

We apply the magic methyl effect to improve the potency/efficacy of GAT211, the prototypic 2-phenylindole-based cannabinoid type-1 receptor (CB1R) agonist-positive allosteric modulator (ago-PAM). Introducing a methyl group at the α-position of nitro group generated two diastereomers, the greater potency and efficacy of erythro, (±)-9 vs threo, (±)-10 constitutes the first demonstration of diastereoselective CB1R-allosteric modulator interaction. Of the (±)-9 enantiomers, (-)-(S,R)-13 evidenced improved potency over GAT211 as a CB1R ago-PAM, whereas (+)-(R,S)-14 was a CB1R allosteric agonist biased toward G protein- vs β-arrestin1/2-dependent signaling. (-)-(S,R)-13 and (+)-(R,S)-14 were devoid of undesirable side effects (triad test), and (+)-(R,S)-14 reduced intraocular pressure with an unprecedentedly long duration of action in a murine glaucoma model. (-)-(S,R)-13 docked into both a CB1R extracellular PAM and intracellular allosteric-agonist site(s), whereas (+)-(R,S)-14 preferentially engaged only the latter. Exploiting G-protein biased CB1R-allosteric modulation can offer safer therapeutic candidates for glaucoma and, potentially, other diseases.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Allosteric Site
  • Animals
  • CHO Cells
  • Cannabinoid Receptor Agonists / chemical synthesis
  • Cannabinoid Receptor Agonists / metabolism
  • Cannabinoid Receptor Agonists / therapeutic use*
  • Cricetulus
  • Glaucoma / drug therapy*
  • HEK293 Cells
  • Hippocampus / cytology
  • Humans
  • Indoles / chemical synthesis
  • Indoles / metabolism
  • Indoles / therapeutic use*
  • Intraocular Pressure / drug effects
  • Ligands
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Molecular Conformation
  • Molecular Docking Simulation
  • Molecular Dynamics Simulation
  • Neurons / drug effects
  • Receptor, Cannabinoid, CB1 / agonists*
  • Receptor, Cannabinoid, CB1 / chemistry
  • Receptor, Cannabinoid, CB1 / metabolism
  • Stereoisomerism
  • Structure-Activity Relationship

Substances

  • 3-(2-nitro-1-phenylethyl)-2-phenyl-1H-indole
  • Cannabinoid Receptor Agonists
  • Indoles
  • Ligands
  • Receptor, Cannabinoid, CB1