Identification and in Vivo Evaluation of Liver X Receptor β-Selective Agonists for the Potential Treatment of Alzheimer's Disease

J Med Chem. 2016 Apr 14;59(7):3489-98. doi: 10.1021/acs.jmedchem.6b00176. Epub 2016 Apr 1.

Abstract

Herein, we describe the development of a functionally selective liver X receptor β (LXRβ) agonist series optimized for Emax selectivity, solubility, and physical properties to allow efficacy and safety studies in vivo. Compound 9 showed central pharmacodynamic effects in rodent models, evidenced by statistically significant increases in apolipoprotein E (apoE) and ATP-binding cassette transporter levels in the brain, along with a greatly improved peripheral lipid safety profile when compared to those of full dual agonists. These findings were replicated by subchronic dosing studies in non-human primates, where cerebrospinal fluid levels of apoE and amyloid-β peptides were increased concomitantly with an improved peripheral lipid profile relative to that of nonselective compounds. These results suggest that optimization of LXR agonists for Emax selectivity may have the potential to circumvent the adverse lipid-related effects of hepatic LXR activity.

Publication types

  • Evaluation Study

MeSH terms

  • ATP-Binding Cassette Transporters / metabolism*
  • Alzheimer Disease / drug therapy*
  • Amyloid beta-Peptides / cerebrospinal fluid*
  • Animals
  • Apolipoproteins E / cerebrospinal fluid*
  • Benzamides / chemistry*
  • Benzamides / pharmacology*
  • Brain / drug effects
  • Brain / metabolism
  • Dogs
  • Hep G2 Cells
  • Humans
  • Lipids / analysis
  • Liver / drug effects
  • Liver / metabolism
  • Liver X Receptors
  • Locomotion / drug effects
  • Macaca mulatta
  • Madin Darby Canine Kidney Cells
  • Mice
  • Mice, Transgenic
  • Orphan Nuclear Receptors / agonists*
  • Piperidines / chemistry*
  • Piperidines / pharmacology*

Substances

  • 2-chloro-4-(1'-(2-hydroxy-3,3-dimethyl-2-(trifluoromethyl)butanoyl)-(4,4'-bipiperidin)-1-yl)-N,N-dimethylbenzamide
  • ATP-Binding Cassette Transporters
  • Amyloid beta-Peptides
  • Apolipoproteins E
  • Benzamides
  • Lipids
  • Liver X Receptors
  • Orphan Nuclear Receptors
  • Piperidines