Development, Optimization, and Structure-Activity Relationships of Covalent-Reversible JAK3 Inhibitors Based on a Tricyclic Imidazo[5,4- d]pyrrolo[2,3- b]pyridine Scaffold

J Med Chem. 2018 Jun 28;61(12):5350-5366. doi: 10.1021/acs.jmedchem.8b00571. Epub 2018 Jun 13.

Abstract

Janus kinases are major drivers of immune signaling and have been the focus of anti-inflammatory drug discovery for more than a decade. Because of the invariable colocalization of JAK1 and JAK3 at cytokine receptors, the question if selective JAK3 inhibition is sufficient to effectively block downstream signaling has been highly controversial. Recently, we discovered the covalent-reversible JAK3 inhibitor FM-381 (23) featuring high isoform and kinome selectivity. Crystallography revealed that this inhibitor induces an unprecedented binding pocket by interactions of a nitrile substituent with arginine residues in JAK3. Herein, we describe detailed structure-activity relationships necessary for induction of the arginine pocket and the impact of this structural change on potency, isoform selectivity, and efficacy in cellular models. Furthermore, we evaluated the stability of this novel inhibitor class in in vitro metabolic assays and were able to demonstrate an adequate stability of key compound 23 for in vivo use.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenosine Triphosphate / metabolism
  • Animals
  • Binding Sites
  • Cells, Cultured
  • Crystallography, X-Ray
  • Drug Evaluation, Preclinical / methods
  • Drug Stability
  • Humans
  • Janus Kinase 3 / antagonists & inhibitors*
  • Janus Kinase 3 / chemistry
  • Janus Kinase 3 / metabolism
  • Luminescent Measurements / methods
  • Mice
  • Phosphorylation / drug effects
  • Protein Kinase Inhibitors / chemistry*
  • Protein Kinase Inhibitors / pharmacology*
  • Pyridines / chemistry
  • STAT5 Transcription Factor / metabolism
  • Structure-Activity Relationship*
  • T-Lymphocytes / drug effects

Substances

  • Protein Kinase Inhibitors
  • Pyridines
  • STAT5 Transcription Factor
  • Adenosine Triphosphate
  • JAK3 protein, human
  • Janus Kinase 3