LXR ligand lowers LDL cholesterol in primates, is lipid neutral in hamster, and reduces atherosclerosis in mouse

J Lipid Res. 2009 Dec;50(12):2358-70. doi: 10.1194/jlr.M900037-JLR200. Epub 2009 Mar 24.

Abstract

Liver X receptors (LXRs) are ligand-activated transcription factors that coordinate regulation of gene expression involved in several cellular functions but most notably cholesterol homeostasis encompassing cholesterol transport, catabolism, and absorption. WAY-252623 (LXR-623) is a highly selective and orally bioavailable synthetic modulator of LXR, which demonstrated efficacy for reducing lesion progression in the murine LDLR(-/-) atherosclerosis model with no associated increase in hepatic lipogenesis either in this model or Syrian hamsters. In nonhuman primates with normal lipid levels, WAY-252623 significantly reduced total (50-55%) and LDL-cholesterol (LDLc) (70-77%) in a time- and dose-dependent manner as well as increased expression of the target genes ABCA1/G1 in peripheral blood cells. Statistically significant decreases in LDLc were noted as early as day 7, reached a maximum by day 28, and exceeded reductions observed for simvastatin alone (20 mg/kg). Transient increases in circulating triglycerides and liver enzymes reverted to baseline levels over the course of the study. Complementary microarray analysis of duodenum and liver gene expression revealed differential activation of LXR target genes and suggested no direct activation of hepatic lipogenesis. WAY-252623 displays a unique and favorable pharmacological profile suggesting synthetic LXR ligands with these characteristics may be suitable for evaluation in patients with atherosclerotic dyslipidemia.

MeSH terms

  • Animals
  • Atherosclerosis / drug therapy*
  • Atherosclerosis / metabolism
  • Caco-2 Cells
  • Cholesterol, LDL / drug effects*
  • Cholesterol, LDL / metabolism*
  • Cricetinae
  • Disease Models, Animal
  • Humans
  • Indazoles / blood
  • Indazoles / chemistry
  • Indazoles / pharmacology*
  • Ligands
  • Lipid Metabolism / drug effects*
  • Liver / enzymology
  • Liver / metabolism
  • Liver X Receptors
  • Macaca fascicularis / metabolism*
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Orphan Nuclear Receptors / agonists*
  • Orphan Nuclear Receptors / metabolism

Substances

  • 2-(2-chloro-4-fluorobenzyl)-3-(4-fluorophenyl)-7-(trifluoromethyl)-2H-indazole
  • Cholesterol, LDL
  • Indazoles
  • Ligands
  • Liver X Receptors
  • Orphan Nuclear Receptors