Discovery of a Novel, Orally Efficacious Liver X Receptor (LXR) β Agonist

J Med Chem. 2016 Apr 14;59(7):3264-71. doi: 10.1021/acs.jmedchem.5b02029. Epub 2016 Mar 29.

Abstract

This article describes the application of Contour to the design and discovery of a novel, potent, orally efficacious liver X receptor β (LXRβ) agonist (17). Contour technology is a structure-based drug design platform that generates molecules using a context perceptive growth algorithm guided by a contact sensitive scoring function. The growth engine uses binding site perception and programmable growth capability to create drug-like molecules by assembling fragments that naturally complement hydrophilic and hydrophobic features of the protein binding site. Starting with a crystal structure of LXRβ and a docked 2-(methylsulfonyl)benzyl alcohol fragment (6), Contour was used to design agonists containing a piperazine core. Compound 17 binds to LXRβ with high affinity and to LXRα to a lesser extent, and induces the expression of LXR target genes in vitro and in vivo. This molecule served as a starting point for further optimization and generation of a candidate which is currently in human clinical trials for treating atopic dermatitis.

MeSH terms

  • Benzylamines / chemistry*
  • Binding Sites
  • Crystallography, X-Ray
  • Drug Design*
  • Drug Discovery*
  • Humans
  • Liver X Receptors
  • Orphan Nuclear Receptors / agonists*
  • Piperazines / chemistry*
  • Pyrimidines / chemistry*
  • Pyrimidines / metabolism*
  • Structure-Activity Relationship
  • Sulfones / chemistry*
  • Sulfones / metabolism*

Substances

  • (2-(4-(4-(hydroxymethyl)-3-(methylsulfonyl)phenyl)-2-isopropylpiperazin-1-yl)-4-(trifluoromethyl)pyrimidin-5-yl)methanol
  • Benzylamines
  • Liver X Receptors
  • NR1H3 protein, human
  • Orphan Nuclear Receptors
  • Piperazines
  • Pyrimidines
  • Sulfones