Discovery of Potent and Selective Epidermal Growth Factor Receptor (EGFR) Bifunctional Small-Molecule Degraders

J Med Chem. 2020 Feb 13;63(3):1216-1232. doi: 10.1021/acs.jmedchem.9b01566. Epub 2020 Jan 14.

Abstract

Several epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors have been developed and approved by Food and Drug Administration for the treatment of non-small-cell lung cancers, but their efficacy can be compromised by acquired drug resistance conferred by EGFR-mutant variants. Here, we described the discovery of a novel E3 ligase von Hippel-Lindau-recruiting EGFR degrader, MS39 (compound 6), and a first-in-class E3 ligase cereblon-recruiting EGFR degrader, MS154 (compound 10), using the proteolysis targeting chimera technology. These compounds potently induced the degradation of mutant but not wild-type EGFR in an E3 ligase-dependent manner in cancer cell lines and effectively suppressed the growth of lung cancer cells compared with the corresponding negative controls. The global proteomic analyses revealed that the compounds were highly selective for EGFR. Furthermore, both compounds were bioavailable in mouse pharmacokinetic studies, and compound 6 is the first EGFR degrader suitable for in vivo efficacy studies. Overall, we provide a set of well-characterized chemical tools to the research community.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adaptor Proteins, Signal Transducing / metabolism
  • Animals
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Drug Design
  • Drug Discovery
  • ErbB Receptors / antagonists & inhibitors
  • ErbB Receptors / genetics
  • ErbB Receptors / metabolism
  • Gefitinib / analogs & derivatives*
  • Gefitinib / metabolism
  • Gefitinib / pharmacokinetics
  • Gefitinib / pharmacology*
  • Humans
  • Male
  • Mice
  • Mutation
  • Protein Kinase Inhibitors / chemical synthesis
  • Protein Kinase Inhibitors / metabolism
  • Protein Kinase Inhibitors / pharmacokinetics
  • Protein Kinase Inhibitors / pharmacology*
  • Proteolysis
  • Signal Transduction / drug effects
  • Ubiquitin-Protein Ligases
  • Von Hippel-Lindau Tumor Suppressor Protein / metabolism

Substances

  • Adaptor Proteins, Signal Transducing
  • CRBN protein, human
  • Protein Kinase Inhibitors
  • Ubiquitin-Protein Ligases
  • Von Hippel-Lindau Tumor Suppressor Protein
  • EGFR protein, human
  • ErbB Receptors
  • VHL protein, human
  • Gefitinib