Identification of TUL01101: A Novel Potent and Selective JAK1 Inhibitor for the Treatment of Rheumatoid Arthritis

J Med Chem. 2022 Dec 22;65(24):16716-16740. doi: 10.1021/acs.jmedchem.2c01550. Epub 2022 Dec 13.

Abstract

Janus kinase 1 (JAK1) is a potential target for the treatment of rheumatoid arthritis (RA). In this study, the introduction of a spiro ring with a difluoro-substituted cyclopropionamide resulted in the identification of TUL01101 (compound 36) based on a triazolo[1,5-a]pyridine core of filgotinib. It showed excellent potency on JAK1 with an IC50 value of 3 nM and exhibited more than 12-fold selectivity for JAK2 and TYK2. Whole blood assay also demonstrated the high activity and selectivity (37-fold for JAK2). At the same time, TUL01101 also demonstrated excellent metabolic stability and pharmacokinetics (PK) profiles were assayed in three species (mouse, rat, and dog). Moreover, it has been validated for effective activity in the treatment of RA both in collagen-induced arthritis (CIA) and adjuvant-induced arthritis (AIA) models, with low dose and low toxicity. Now, TUL01101 has progressed into phase I clinical trials.

MeSH terms

  • Animals
  • Arthritis, Experimental* / drug therapy
  • Arthritis, Rheumatoid* / drug therapy
  • Biological Assay
  • Dogs
  • Janus Kinase 1* / antagonists & inhibitors
  • Janus Kinase Inhibitors* / pharmacokinetics
  • Janus Kinase Inhibitors* / pharmacology
  • Janus Kinase Inhibitors* / therapeutic use
  • Mice
  • Rats

Substances

  • Janus Kinase 1
  • Janus Kinase Inhibitors