Discovery of Branebrutinib (BMS-986195): A Strategy for Identifying a Highly Potent and Selective Covalent Inhibitor Providing Rapid in Vivo Inactivation of Bruton's Tyrosine Kinase (BTK)

J Med Chem. 2019 Apr 11;62(7):3228-3250. doi: 10.1021/acs.jmedchem.9b00167. Epub 2019 Mar 29.

Abstract

Bruton's tyrosine kinase (BTK), a non-receptor tyrosine kinase, is a member of the Tec family of kinases and is essential for B cell receptor (BCR) mediated signaling. BTK also plays a critical role in the downstream signaling pathways for the Fcγ receptor in monocytes, the Fcε receptor in granulocytes, and the RANK receptor in osteoclasts. As a result, pharmacological inhibition of BTK is anticipated to provide an effective strategy for the clinical treatment of autoimmune diseases such as rheumatoid arthritis and lupus. This article will outline the evolution of our strategy to identify a covalent, irreversible inhibitor of BTK that has the intrinsic potency, selectivity, and pharmacokinetic properties necessary to provide a rapid rate of inactivation systemically following a very low dose. With excellent in vivo efficacy and a very desirable tolerability profile, 5a (branebrutinib, BMS-986195) has advanced into clinical studies.

MeSH terms

  • Agammaglobulinaemia Tyrosine Kinase / antagonists & inhibitors*
  • Animals
  • Arthritis, Rheumatoid / drug therapy
  • Dose-Response Relationship, Drug
  • Drug Discovery*
  • Humans
  • Indoles / pharmacokinetics
  • Indoles / pharmacology*
  • Indoles / therapeutic use
  • Inhibitory Concentration 50
  • Lupus Erythematosus, Systemic / drug therapy
  • Macaca fascicularis
  • Mice
  • Piperidines / pharmacokinetics
  • Piperidines / pharmacology*
  • Piperidines / therapeutic use
  • Protein Kinase Inhibitors / pharmacokinetics
  • Protein Kinase Inhibitors / pharmacology*
  • Protein Kinase Inhibitors / therapeutic use

Substances

  • Indoles
  • Piperidines
  • Protein Kinase Inhibitors
  • branebrutinib
  • Agammaglobulinaemia Tyrosine Kinase
  • BTK protein, human