Discovery of GDC-0853: A Potent, Selective, and Noncovalent Bruton's Tyrosine Kinase Inhibitor in Early Clinical Development

J Med Chem. 2018 Mar 22;61(6):2227-2245. doi: 10.1021/acs.jmedchem.7b01712. Epub 2018 Feb 23.

Abstract

Bruton's tyrosine kinase (Btk) is a nonreceptor cytoplasmic tyrosine kinase involved in B-cell and myeloid cell activation, downstream of B-cell and Fcγ receptors, respectively. Preclinical studies have indicated that inhibition of Btk activity might offer a potential therapy in autoimmune diseases such as rheumatoid arthritis and systemic lupus erythematosus. Here we disclose the discovery and preclinical characterization of a potent, selective, and noncovalent Btk inhibitor currently in clinical development. GDC-0853 (29) suppresses B cell- and myeloid cell-mediated components of disease and demonstrates dose-dependent activity in an in vivo rat model of inflammatory arthritis. It demonstrates highly favorable safety, pharmacokinetic (PK), and pharmacodynamic (PD) profiles in preclinical and Phase 2 studies ongoing in patients with rheumatoid arthritis, lupus, and chronic spontaneous urticaria. On the basis of its potency, selectivity, long target residence time, and noncovalent mode of inhibition, 29 has the potential to be a best-in-class Btk inhibitor for a wide range of immunological indications.

MeSH terms

  • Agammaglobulinaemia Tyrosine Kinase / antagonists & inhibitors*
  • Agammaglobulinaemia Tyrosine Kinase / drug effects
  • Agammaglobulinaemia Tyrosine Kinase / genetics
  • Animals
  • Anti-Inflammatory Agents / pharmacokinetics
  • Anti-Inflammatory Agents / pharmacology*
  • Anti-Inflammatory Agents / toxicity
  • Arthritis, Experimental / drug therapy
  • Arthritis, Rheumatoid / drug therapy
  • Dogs
  • Drug Discovery
  • Humans
  • Lupus Erythematosus, Systemic / drug therapy
  • Madin Darby Canine Kidney Cells
  • Models, Molecular
  • Molecular Structure
  • Piperazines / pharmacokinetics
  • Piperazines / pharmacology*
  • Piperazines / toxicity
  • Protein Kinase Inhibitors / pharmacokinetics
  • Protein Kinase Inhibitors / pharmacology*
  • Protein Kinase Inhibitors / toxicity
  • Pyridones / pharmacokinetics
  • Pyridones / pharmacology*
  • Pyridones / toxicity
  • Rats
  • Rats, Inbred Lew
  • Rats, Sprague-Dawley

Substances

  • Anti-Inflammatory Agents
  • Piperazines
  • Protein Kinase Inhibitors
  • Pyridones
  • fenebrutinib
  • Agammaglobulinaemia Tyrosine Kinase
  • BTK protein, human