Identification of ABX-1431, a Selective Inhibitor of Monoacylglycerol Lipase and Clinical Candidate for Treatment of Neurological Disorders

J Med Chem. 2018 Oct 25;61(20):9062-9084. doi: 10.1021/acs.jmedchem.8b00951. Epub 2018 Aug 23.

Abstract

The serine hydrolase monoacylglycerol lipase (MGLL) converts the endogenous cannabinoid receptor agonist 2-arachidonoylglycerol (2-AG) and other monoacylglycerols into fatty acids and glycerol. Genetic or pharmacological inactivation of MGLL leads to elevation in 2-AG in the central nervous system and corresponding reductions in arachidonic acid and eicosanoids, producing antinociceptive, anxiolytic, and antineuroinflammatory effects without inducing the full spectrum of psychoactive effects of direct cannabinoid receptor agonists. Here, we report the optimization of hexafluoroisopropyl carbamate-based irreversible inhibitors of MGLL, culminating in a highly potent, selective, and orally available, CNS-penetrant MGLL inhibitor, 28 (ABX-1431). Activity-based protein profiling experiments verify the exquisite selectivity of 28 for MGLL versus other members of the serine hydrolase class. In vivo, 28 inhibits MGLL activity in rodent brain (ED50 = 0.5-1.4 mg/kg), increases brain 2-AG concentrations, and suppresses pain behavior in the rat formalin pain model. ABX-1431 (28) is currently under evaluation in human clinical trials.

MeSH terms

  • Animals
  • Dogs
  • Dose-Response Relationship, Drug
  • Drug Discovery*
  • Humans
  • Male
  • Mice
  • Molecular Targeted Therapy
  • Monoacylglycerol Lipases / antagonists & inhibitors*
  • Nervous System Diseases / drug therapy*
  • Nervous System Diseases / enzymology*
  • Pain / drug therapy
  • Pain / enzymology
  • Piperazine / pharmacokinetics
  • Piperazine / pharmacology*
  • Piperazine / therapeutic use
  • Piperazines / pharmacokinetics
  • Piperazines / pharmacology*
  • Piperazines / therapeutic use
  • Pyrrolidines / pharmacokinetics
  • Pyrrolidines / pharmacology*
  • Pyrrolidines / therapeutic use
  • Rats
  • Tissue Distribution

Substances

  • ABX-1431
  • Piperazines
  • Pyrrolidines
  • Piperazine
  • Monoacylglycerol Lipases