Discovery of a Novel, Potent Spirocyclic Series of γ-Secretase Inhibitors

J Med Chem. 2015 Nov 25;58(22):8806-17. doi: 10.1021/acs.jmedchem.5b00774. Epub 2015 Nov 6.

Abstract

In the present paper, we described the design, synthesis, SAR, and biological profile of a novel spirocyclic sulfone series of γ-secretase inhibitors (GSIs) related to MRK-560. We utilized an additional spirocyclic ring system to stabilize the active chair conformation of the parent γ-secretase inhibitors. The resulting series is devoid of the CYP2C9 inhibition liability of MRK-560. A few representative analogs were assessed in a nontransgenic animal model of Alzheimer's disease (AD), demonstrating reduction of amyloid-β (Aβ) in the CNS after acute oral dosing. A spirocyclic phosphonate was identified as the optimal ring system for both potency and pharmacokinetics. Compared to GSIs studied in the clinic, representative spirocyclic phosphonate 18a(-) features improved selectivity for the inhibition of the PS-1 isoform of γ-secretase (33-fold vs PS-2), which may alleviate the adverse effect profile of the clinical GSIs.

MeSH terms

  • Alzheimer Disease / drug therapy
  • Amyloid Precursor Protein Secretases / antagonists & inhibitors*
  • Amyloid beta-Peptides / antagonists & inhibitors
  • Amyloid beta-Peptides / metabolism
  • Animals
  • Biological Availability
  • Central Nervous System / drug effects
  • Central Nervous System / metabolism
  • Cytochrome P-450 CYP2C9 / metabolism
  • Cytochrome P-450 Enzyme Inhibitors / pharmacology
  • Drug Discovery / methods*
  • Enzyme Inhibitors / chemical synthesis*
  • Enzyme Inhibitors / pharmacokinetics
  • Enzyme Inhibitors / pharmacology*
  • HEK293 Cells
  • Humans
  • Molecular Conformation
  • Rats
  • Rats, Sprague-Dawley
  • Structure-Activity Relationship
  • Sulfonamides / pharmacology

Substances

  • Amyloid beta-Peptides
  • Cytochrome P-450 Enzyme Inhibitors
  • Enzyme Inhibitors
  • MRK 560
  • Sulfonamides
  • Cytochrome P-450 CYP2C9
  • Amyloid Precursor Protein Secretases