Discovery and Optimization of Potent and CNS Penetrant M5-Preferring Positive Allosteric Modulators Derived from a Novel, Chiral N-(Indanyl)piperidine Amide Scaffold

ACS Chem Neurosci. 2018 Jul 18;9(7):1572-1581. doi: 10.1021/acschemneuro.8b00126. Epub 2018 Apr 26.

Abstract

The pharmacology of the M5 muscarinic acetylcholine receptor (mAChR) is the least understood of the five mAChR subtypes due to a historic lack of selective small molecule tools. To address this shortcoming, we have continued the optimization effort around the prototypical M5 positive allosteric modulator (PAM) ML380 and have discovered and optimized a new series of M5 PAMs based on a chiral N-(indanyl)piperidine amide core with robust SAR, human and rat M5 PAM EC50 values <100 nM and rat brain/plasma Kp values of ∼0.40. Interestingly, unlike M1 and M4 PAMs with unprecedented mAChR subtype selectivity, this series of M5 PAMs displayed varying degrees of PAM activity at the other two natively Gq-coupled mAChRs, M1 and M3, yet were inactive at M2 and M4.

Keywords: M5; SAR; muscarinic acetylcholine receptor; positive allosteric modulator (PAM); selectivity.

Publication types

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

MeSH terms

  • Allosteric Regulation
  • Amides / chemistry
  • Animals
  • Brain / drug effects
  • Brain / metabolism
  • Cholinergic Agents / chemical synthesis
  • Cholinergic Agents / chemistry
  • Cholinergic Agents / pharmacokinetics
  • Cholinergic Agents / pharmacology*
  • Drug Discovery
  • Humans
  • Male
  • Microsomes, Liver / drug effects
  • Microsomes, Liver / metabolism
  • Molecular Structure
  • Piperidines / chemistry
  • Rats, Sprague-Dawley
  • Receptors, Muscarinic / metabolism
  • Structure-Activity Relationship

Substances

  • Amides
  • Cholinergic Agents
  • Piperidines
  • Receptors, Muscarinic