Discovery of novel triazolobenzazepinones as γ-secretase modulators with central Aβ42 lowering in rodents and rhesus monkeys

Bioorg Med Chem Lett. 2015 Sep 1;25(17):3488-94. doi: 10.1016/j.bmcl.2015.07.003. Epub 2015 Jul 9.

Abstract

Synthesis and SAR studies of novel triazolobenzazepinones as gamma secretase modulators (GSMs) are presented in this communication. Starting from our azepinone leads, optimization studies toward improving central lowering of Aβ42 led to the discovery of novel benzo-fused azepinones. Several benzazepinones were profiled in vivo and found to lower brain Aβ42 levels in Sprague Dawley rats and transgenic APP-YAC mice in a dose-dependent manner after a single oral dose. Compound 34 was further progressed into a pilot study in our cisterna-magna-ported rhesus monkey model, where we observed robust lowering of CSF Aβ42 levels.

Keywords: Alzheimer’s; CSF Aβ lowering; GSM; Rhesus PK/PD; γ-Secretase.

MeSH terms

  • Alzheimer Disease / genetics*
  • Alzheimer Disease / metabolism*
  • Amyloid Precursor Protein Secretases / metabolism*
  • Animals
  • Drug Discovery
  • Macaca mulatta
  • Mice
  • Mice, Transgenic
  • Rats
  • Rats, Sprague-Dawley

Substances

  • Amyloid Precursor Protein Secretases