Design, Synthesis, and Biological Evaluation of 5-Formyl-pyrrolo[3,2- b]pyridine-3-carboxamides as New Selective, Potent, and Reversible-Covalent FGFR4 Inhibitors

J Med Chem. 2022 Nov 10;65(21):14809-14831. doi: 10.1021/acs.jmedchem.2c01319. Epub 2022 Oct 24.

Abstract

The FGF19-FGFR4 signaling pathway has been extensively studied as a promising target for the treatment of hepatocellular carcinoma (HCC). Several FGFR4-selective inhibitors have been developed, but none of them receives approval. Additionally, acquired resistance caused by FGFR4 gatekeeper mutations is emerging as a serious limitation for these targeted therapies. Herein, we report a novel series of 5-formyl-pyrrolo[3,2-b]pyridine derivatives as new reversible-covalent inhibitors targeting wild-type and gatekeeper mutant variants of FGFR4 kinase. The representative compound 10z exhibited single-digit nanomolar activity against wild-type FGFR4 and the FGFR4V550L/M mutant variants in biochemical and Ba/F3 cellular assays, while sparing FGFR1/2/3. Furthermore, 10z showed significant antiproliferative activity against Hep3B, JHH-7, and HuH-7 HCC cells with IC50 values of 37, 32, and 94 nM, respectively. MALDI-TOF-MS and X-ray protein crystallography studies were consistent with 10z acting as a reversible-covalent inhibitor of FGFR4, serving as a promising lead compound for further anticancer drug development.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents* / chemistry
  • Carcinoma, Hepatocellular* / drug therapy
  • Cell Line, Tumor
  • Humans
  • Liver Neoplasms* / drug therapy
  • Protein Kinase Inhibitors / chemistry
  • Pyridines / pharmacology
  • Pyridines / therapeutic use
  • Receptor, Fibroblast Growth Factor, Type 4
  • Signal Transduction

Substances

  • Antineoplastic Agents
  • FGFR4 protein, human
  • Protein Kinase Inhibitors
  • Pyridines
  • Receptor, Fibroblast Growth Factor, Type 4
  • 1,3-bis(2,6-diisopropylphenyl)imidazol-2-ylidene