A Novel Triazolopyridine-Based Spleen Tyrosine Kinase Inhibitor That Arrests Joint Inflammation

PLoS One. 2016 Jan 12;11(1):e0145705. doi: 10.1371/journal.pone.0145705. eCollection 2016.

Abstract

Autoantibodies and the immunoreceptors to which they bind can contribute to the pathogenesis of autoimmune diseases such as rheumatoid arthritis (RA). Spleen Tyrosine Kinase (Syk) is a non-receptor tyrosine kinase with a central role in immunoreceptor (FcR) signaling and immune cell functionality. Syk kinase inhibitors have activity in antibody-dependent immune cell activation assays, in preclinical models of arthritis, and have progressed into clinical trials for RA and other autoimmune diseases. Here we describe the characterization of a novel triazolopyridine-based Syk kinase inhibitor, CC-509. This compound is a potent inhibitor of purified Syk enzyme, FcR-dependent and FcR-independent signaling in primary immune cells, and basophil activation in human whole blood. CC-509 is moderately selective across the kinome and against other non-kinase enzymes or receptors. Importantly, CC-509 was optimized away from and has modest activity against cellular KDR and Jak2, kinases that when inhibited in a preclinical and clinical setting may promote hypertension and neutropenia, respectively. In addition, CC-509 is orally bioavailable and displays dose-dependent efficacy in two rodent models of immune-inflammatory disease. In passive cutaneous anaphylaxis (PCA), CC-509 significantly inhibited skin edema. Moreover, CC-509 significantly reduced paw swelling and the tissue levels of pro-inflammatory cytokines RANTES and MIP-1α in the collagen-induced arthritis (CIA) model. In summary, CC-509 is a potent, moderately selective, and efficacious inhibitor of Syk that has a differentiated profile when compared to other Syk compounds that have progressed into the clinic for RA.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Arthritis, Experimental / drug therapy
  • Arthritis, Experimental / physiopathology
  • Basophils / cytology
  • Cell Line
  • Collagen / chemistry
  • Crystallography, X-Ray
  • Dose-Response Relationship, Drug
  • Edema / pathology
  • Eosinophils / cytology
  • Female
  • HEK293 Cells
  • Humans
  • Hypertension / drug therapy
  • Indazoles / chemistry*
  • Inflammation / drug therapy*
  • Inflammation / physiopathology
  • Inhibitory Concentration 50
  • Intracellular Signaling Peptides and Proteins / antagonists & inhibitors*
  • Janus Kinase 2 / antagonists & inhibitors
  • Male
  • Neutropenia / drug therapy
  • Neutrophils / cytology
  • Protein-Tyrosine Kinases / antagonists & inhibitors*
  • Pyridines / chemistry*
  • Rats
  • Rats, Inbred Lew
  • Rats, Sprague-Dawley
  • Receptors, Fc / chemistry
  • Skin / pathology
  • Syk Kinase
  • Triazoles / chemistry*
  • Vascular Endothelial Growth Factor Receptor-2 / antagonists & inhibitors

Substances

  • CC-509
  • Indazoles
  • Intracellular Signaling Peptides and Proteins
  • Pyridines
  • Receptors, Fc
  • Triazoles
  • Collagen
  • Protein-Tyrosine Kinases
  • Vascular Endothelial Growth Factor Receptor-2
  • JAK2 protein, human
  • Janus Kinase 2
  • SYK protein, human
  • Syk Kinase
  • Syk protein, rat

Grants and funding

The authors received no specific funding for this work.