Design and synthesis of pyrrolo[3,2-d]pyrimidine human epidermal growth factor receptor 2 (HER2)/epidermal growth factor receptor (EGFR) dual inhibitors: exploration of novel back-pocket binders

J Med Chem. 2012 Apr 26;55(8):3975-91. doi: 10.1021/jm300185p. Epub 2012 Apr 10.

Abstract

To develop novel human epidermal growth factor receptor 2 (HER2)/epidermal growth factor receptor (EGFR) kinase inhibitors, we explored pyrrolo[3,2-d]pyrimidine derivatives bearing bicyclic fused rings designed to fit the back pocket of the HER2/EGFR proteins. Among them, the 1,2-benzisothiazole (42m) ring was selected as a suitable back pocket binder because of its potent HER2/EGFR binding and cell growth inhibitory (GI) activities and pseudoirreversibility (PI) profile as well as good bioavailability (BA). Ultimately, we arrived at our preclinical candidate 51m by optimization of the N-5 side chain to improve CYP inhibition and metabolic stability profiles without a loss of potency (HER2/EGFR inhibitory activity, IC(50), 0.98/2.5 nM; and GI activity BT-474 cells, GI(50), 2.0 nM). Reflecting the strong in vitro activities, 51m exhibited potent tumor regressive efficacy against both HER2- and EGFR-overexpressing tumor (4-1ST and CAL27) xenograft models in mice at oral doses of 50 mg/kg and 100 mg/kg.

MeSH terms

  • Animals
  • Biological Availability
  • Bridged Bicyclo Compounds, Heterocyclic / chemical synthesis
  • Bridged Bicyclo Compounds, Heterocyclic / pharmacology*
  • Drug Design
  • Enzyme Inhibitors / chemical synthesis
  • ErbB Receptors / antagonists & inhibitors*
  • Female
  • Humans
  • Hydroxybutyrates / chemical synthesis
  • Hydroxybutyrates / pharmacology*
  • Mice
  • Protein-Tyrosine Kinases / antagonists & inhibitors*
  • Pyrimidines / chemical synthesis*
  • Pyrimidines / pharmacology
  • Pyrroles / chemical synthesis*
  • Pyrroles / pharmacology
  • Receptor, ErbB-2 / antagonists & inhibitors*
  • Structure-Activity Relationship
  • Xenograft Model Antitumor Assays

Substances

  • Bridged Bicyclo Compounds, Heterocyclic
  • Enzyme Inhibitors
  • Hydroxybutyrates
  • Pyrimidines
  • Pyrroles
  • N-(2-(4-((3-chloro-4-(3-(trifluoromethyl)phenoxy)phenyl)amino)-5H-pyrrolo(3,2-d)pyrimidin-5-yl)ethyl)-3-hydroxy-3-methylbutanamide
  • ErbB Receptors
  • Protein-Tyrosine Kinases
  • Receptor, ErbB-2