Kinetics-Driven Drug Design Strategy for Next-Generation Acetylcholinesterase Inhibitors to Clinical Candidate

J Med Chem. 2021 Feb 25;64(4):1844-1855. doi: 10.1021/acs.jmedchem.0c01863. Epub 2021 Feb 11.

Abstract

The acetylcholinesterase (AChE) inhibitors remain key therapeutic drugs for the treatment of Alzheimer's disease (AD). However, the low-safety window limits their maximum therapeutic benefits. Here, a novel kinetics-driven drug design strategy was employed to discover new-generation AChE inhibitors that possess a longer drug-target residence time and exhibit a larger safety window. After detailed investigations, compound 12 was identified as a highly potent, highly selective, orally bioavailable, and brain preferentially distributed AChE inhibitor. Moreover, it significantly ameliorated cognitive impairments in different mouse models with a lower effective dose than donepezil. The X-ray structure of the cocrystal complex provided a precise binding mode between 12 and AChE. Besides, the data from the phase I trials demonstrated that 12 had good safety, tolerance, and pharmacokinetic profiles at all preset doses in healthy volunteers, providing a solid basis for its further investigation in phase II trials for the treatment of AD.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetylcholinesterase / metabolism*
  • Alzheimer Disease / chemically induced
  • Alzheimer Disease / drug therapy
  • Animals
  • Cholinesterase Inhibitors / chemical synthesis
  • Cholinesterase Inhibitors / metabolism
  • Cholinesterase Inhibitors / therapeutic use*
  • Crystallography, X-Ray
  • Dogs
  • Drug Design
  • Female
  • Humans
  • Indans / chemical synthesis
  • Indans / metabolism
  • Indans / therapeutic use*
  • Kinetics
  • Male
  • Mice
  • Mice, Inbred ICR
  • Molecular Structure
  • Nootropic Agents / chemical synthesis
  • Nootropic Agents / metabolism
  • Nootropic Agents / therapeutic use*
  • Piperidines / chemical synthesis
  • Piperidines / metabolism
  • Piperidines / therapeutic use*
  • Protein Binding
  • Rats
  • Rats, Sprague-Dawley
  • Scopolamine
  • Structure-Activity Relationship

Substances

  • Cholinesterase Inhibitors
  • Indans
  • Nootropic Agents
  • Piperidines
  • Scopolamine
  • Acetylcholinesterase