Macrocyclic Inhibitors of HGF-Activating Serine Proteases Overcome Resistance to Receptor Tyrosine Kinase Inhibitors and Block Lung Cancer Progression

J Med Chem. 2021 Dec 23;64(24):18158-18174. doi: 10.1021/acs.jmedchem.1c01671. Epub 2021 Dec 13.

Abstract

Hepatocyte growth factor (HGF), the ligand for the MET receptor tyrosine kinase, is a tumor-promoting factor that is abundant in the tumor microenvironment. Proteolytic activation of inactive pro-HGF by one or more of the serine endopeptidases matriptase, hepsin, and HGF activator is the rate-limiting step in HGF/MET signaling. Herein, we have rationally designed a novel class of side chain cyclized macrocyclic peptide inhibitors. The new series of cyclic tripeptides has superior metabolic stability and significantly improved pharmacokinetics in mice relative to the corresponding linear peptides. We identified the lead compound VD2173 that potently inhibits matriptase and hepsin, which was tested in parallel alongside the acyclic inhibitor ZFH7116 using both in vitro and in vivo models of lung cancer. We demonstrated that both compounds block pro-HGF activation, abrogate HGF-mediated wound healing, and overcome resistance to EGFR- and MET-targeted therapy in lung cancer models. Furthermore, VD2173 inhibited HGF-dependent growth of lung cancer tumors in mice.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Carcinoma, Non-Small-Cell Lung / drug therapy
  • Carcinoma, Non-Small-Cell Lung / pathology*
  • Disease Progression
  • Drug Discovery
  • Hepatocyte Growth Factor / metabolism*
  • Humans
  • Lung Neoplasms / drug therapy
  • Lung Neoplasms / pathology*
  • Macrocyclic Compounds / blood
  • Macrocyclic Compounds / chemistry
  • Macrocyclic Compounds / pharmacology*
  • Macrocyclic Compounds / therapeutic use
  • Mice
  • Receptor Protein-Tyrosine Kinases / metabolism*
  • Serine Proteinase Inhibitors / blood
  • Serine Proteinase Inhibitors / chemistry
  • Serine Proteinase Inhibitors / pharmacology*
  • Serine Proteinase Inhibitors / therapeutic use
  • Structure-Activity Relationship

Substances

  • Macrocyclic Compounds
  • Serine Proteinase Inhibitors
  • Hepatocyte Growth Factor
  • Receptor Protein-Tyrosine Kinases