Potent and Orally Bioavailable Inverse Agonists of RORγt Resulting from Structure-Based Design

J Med Chem. 2018 Sep 13;61(17):7796-7813. doi: 10.1021/acs.jmedchem.8b00783. Epub 2018 Aug 27.

Abstract

Retinoic acid receptor related orphan receptor γt (RORγt), has been identified as the master regulator of TH17-cell function and development, making it an attractive target for the treatment of autoimmune diseases by a small-molecule approach. Herein, we describe our investigations on a series of 4-aryl-thienyl acetamides, which were guided by insights from X-ray cocrystal structures. Efforts in targeting the cofactor-recruitment site from the 4-aryl group on the thiophene led to a series of potent binders with nanomolar activity in a primary human-TH17-cell assay. The observation of a DMSO molecule binding in a subpocket outside the LBD inspired the introduction of an acetamide into the benzylic position of these compounds. Hereby, a hydrogen-bond interaction of the introduced acetamide oxygen with the backbone amide of Glu379 was established. This greatly enhanced the cellular activity of previously weakly cell-active compounds. The best compounds combined potent inhibition of IL-17 release with favorable PK in rodents, with compound 32 representing a promising starting point for future investigations.

MeSH terms

  • Acetamides / administration & dosage
  • Acetamides / chemistry
  • Acetamides / pharmacokinetics
  • Acetamides / pharmacology*
  • Administration, Oral
  • Animals
  • Binding Sites
  • Biological Availability
  • Cells, Cultured
  • Crystallography, X-Ray
  • Drug Design*
  • Drug Inverse Agonism*
  • Humans
  • Interleukin-17 / metabolism
  • Models, Molecular
  • Molecular Structure
  • Nuclear Receptor Subfamily 1, Group F, Member 3 / agonists*
  • Nuclear Receptor Subfamily 1, Group F, Member 3 / chemistry
  • Nuclear Receptor Subfamily 1, Group F, Member 3 / metabolism
  • Protein Binding
  • Protein Conformation*
  • Rodentia
  • Structure-Activity Relationship
  • Th17 Cells / drug effects*
  • Th17 Cells / immunology
  • Th17 Cells / metabolism*
  • Tissue Distribution

Substances

  • Acetamides
  • IL17A protein, human
  • Interleukin-17
  • Nuclear Receptor Subfamily 1, Group F, Member 3