Acyl guanidine inhibitors of β-secretase (BACE-1): optimization of a micromolar hit to a nanomolar lead via iterative solid- and solution-phase library synthesis

J Med Chem. 2012 Nov 8;55(21):9208-23. doi: 10.1021/jm300931y. Epub 2012 Oct 2.

Abstract

This report describes the discovery and optimization of a BACE-1 inhibitor series containing an unusual acyl guanidine chemotype that was originally synthesized as part of a 6041-membered solid-phase library. The synthesis of multiple follow-up solid- and solution-phase libraries facilitated the optimization of the original micromolar hit into a single-digit nanomolar BACE-1 inhibitor in both radioligand binding and cell-based functional assay formats. The X-ray structure of representative inhibitors bound to BACE-1 revealed a number of key ligand:protein interactions, including a hydrogen bond between the side chain amide of flap residue Gln73 and the acyl guanidine carbonyl group, and a cation-π interaction between Arg235 and the isothiazole 4-methoxyphenyl substituent. Following subcutaneous administration in rats, an acyl guanidine inhibitor with single-digit nanomolar activity in cells afforded good plasma exposures and a dose-dependent reduction in plasma Aβ levels, but poor brain exposure was observed (likely due to Pgp-mediated efflux), and significant reductions in brain Aβ levels were not obtained.

MeSH terms

  • Amyloid Precursor Protein Secretases / antagonists & inhibitors*
  • Amyloid Precursor Protein Secretases / chemistry
  • Amyloid beta-Peptides / metabolism
  • Amyloid beta-Protein Precursor / genetics
  • Animals
  • Aspartic Acid Endopeptidases / antagonists & inhibitors*
  • Aspartic Acid Endopeptidases / chemistry
  • Brain / metabolism
  • Cell Line
  • Crystallography, X-Ray
  • Guanidines / chemical synthesis*
  • Guanidines / pharmacokinetics
  • Guanidines / pharmacology
  • Humans
  • Isoxazoles / chemical synthesis
  • Isoxazoles / pharmacokinetics
  • Isoxazoles / pharmacology
  • Models, Molecular
  • Molecular Structure
  • Mutation
  • Peptide Fragments / metabolism
  • Protein Binding
  • Radioligand Assay
  • Rats
  • Small Molecule Libraries*
  • Solid-Phase Synthesis Techniques
  • Solutions
  • Structure-Activity Relationship

Substances

  • Amyloid beta-Peptides
  • Amyloid beta-Protein Precursor
  • Guanidines
  • Isoxazoles
  • Peptide Fragments
  • Small Molecule Libraries
  • Solutions
  • amyloid beta-protein (1-40)
  • Amyloid Precursor Protein Secretases
  • Aspartic Acid Endopeptidases
  • BACE1 protein, human

Associated data

  • PDB/4FSL
  • PDB/4SFE