Potent and Selective Inhibitors of the Epidermal Growth Factor Receptor to Overcome C797S-Mediated Resistance

J Med Chem. 2021 Sep 23;64(18):13704-13718. doi: 10.1021/acs.jmedchem.1c01055. Epub 2021 Sep 7.

Abstract

The epidermal growth factor receptor (EGFR) harboring activating mutations is a clinically validated target in non-small-cell lung cancer, and a number of inhibitors of the EGFR tyrosine kinase domain, including osimertinib, have been approved for clinical use. Resistance to these therapies has emerged due to a variety of molecular events including the C797S mutation which renders third-generation C797-targeting covalent EGFR inhibitors considerably less potent against the target due to the loss of the key covalent-bond-forming residue. We describe the medicinal chemistry optimization of a biochemically potent but modestly cell-active, reversible EGFR inhibitor starting point with sub-optimal physicochemical properties. These studies culminated in the identification of compound 12 that showed improved cell potency, oral exposure, and in vivo activity in clinically relevant EGFR-mutant-driven disease models, including an Exon19 deletion/T790M/C797S triple-mutant mouse xenograft model.

MeSH terms

  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / metabolism
  • Antineoplastic Agents / therapeutic use*
  • Cell Line, Tumor
  • Drug Resistance, Neoplasm / drug effects
  • ErbB Receptors / antagonists & inhibitors*
  • ErbB Receptors / genetics
  • ErbB Receptors / metabolism
  • Female
  • Humans
  • Mice
  • Mice, Nude
  • Mice, SCID
  • Mutation
  • Neoplasms / drug therapy*
  • Organophosphorus Compounds / chemical synthesis
  • Organophosphorus Compounds / metabolism
  • Organophosphorus Compounds / therapeutic use*
  • Protein Kinase Inhibitors / chemical synthesis
  • Protein Kinase Inhibitors / metabolism
  • Protein Kinase Inhibitors / therapeutic use*
  • Pyrimidines / chemical synthesis
  • Pyrimidines / metabolism
  • Pyrimidines / therapeutic use*
  • Rats
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Organophosphorus Compounds
  • Protein Kinase Inhibitors
  • Pyrimidines
  • ErbB Receptors