Discovery of biphenylacetamide-derived inhibitors of BACE1 using de novo structure-based molecular design

J Med Chem. 2013 Mar 14;56(5):1843-52. doi: 10.1021/jm301127x. Epub 2013 Feb 22.

Abstract

β-Secretase (BACE1), the enzyme responsible for the first and rate-limiting step in the production of amyloid-β peptides, is an attractive target for the treatment of Alzheimer's disease. In this study, we report the application of the de novo fragment-based molecular design program SPROUT to the discovery of a series of nonpeptide BACE1 inhibitors based upon a biphenylacetamide scaffold. The binding affinity of molecules based upon this designed molecular scaffold was increased from an initial BACE1 IC50 of 323 μM to 27 μM following the synthesis of a library of optimized ligands whose structures were refined using the recently developed SPROUT-HitOpt software. Although a number of inhibitors were found to exhibit cellular toxicity, one compound in the series was found to have useful BACE1 inhibitory activity in a cellular assay with minimal cellular toxicity. This work demonstrates the power of an in silico fragment-based molecular design approach in the discovery of novel BACE1 inhibitors.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alzheimer Disease / enzymology
  • Amyloid Precursor Protein Secretases / antagonists & inhibitors*
  • Aspartic Acid Endopeptidases / antagonists & inhibitors*
  • Benzeneacetamides / chemical synthesis*
  • Benzeneacetamides / pharmacology
  • Cell Survival / drug effects
  • Drug Design
  • Humans
  • Phenylacetates / chemical synthesis*
  • Phenylacetates / pharmacology
  • Protease Inhibitors / chemical synthesis*
  • Protease Inhibitors / pharmacology
  • Software
  • Structure-Activity Relationship

Substances

  • Benzeneacetamides
  • Phenylacetates
  • Protease Inhibitors
  • biphenylylacetic acid
  • Amyloid Precursor Protein Secretases
  • Aspartic Acid Endopeptidases
  • BACE2 protein, human
  • BACE1 protein, human