Non-rigid Diarylmethyl Analogs of Baloxavir as Cap-Dependent Endonuclease Inhibitors of Influenza Viruses

J Med Chem. 2020 Sep 10;63(17):9403-9420. doi: 10.1021/acs.jmedchem.0c00565. Epub 2020 Aug 17.

Abstract

4-Substituted 2,4-dioxobutanoic acids inhibit influenza virus cap-dependent endonuclease (CEN) activity. Baloxavir marboxil, 4, is approved for treating influenza virus infections. We describe here the synthesis and biological evaluation of active compounds, 5a-5g, and their precursors (6a, 6b, 6d, and 6e) with flexible bulky hydrophobic groups instead of the rigid polyheterocyclic moieties. In silico docking confirmed the ability of 5a-5g to bind to the active site of influenza A CEN (PDB code: 6FS6) like baloxavir acid, 3. These novel compounds inhibited polymerase complex activity, inhibited virus replication in cells, prevented death in a lethal influenza A virus mouse challenge model, and dramatically lowered viral lung titers. 5a and 5e potently inhibited different influenza genera in vitro. Precursors 6a and 6d demonstrated impressive mouse oral bioavailability with 6a, providing effective in vivo protection. Thus, these novel compounds are potent CEN inhibitors with in vitro and in vivo activity comparable to baloxavir.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Dibenzothiepins / adverse effects
  • Dibenzothiepins / chemistry*
  • Dibenzothiepins / pharmacokinetics
  • Dibenzothiepins / pharmacology*
  • Endonucleases / antagonists & inhibitors*
  • Endonucleases / chemistry
  • Enzyme Inhibitors / adverse effects
  • Enzyme Inhibitors / chemistry*
  • Enzyme Inhibitors / pharmacokinetics
  • Enzyme Inhibitors / pharmacology*
  • Female
  • HEK293 Cells
  • Humans
  • Influenza A Virus, H1N1 Subtype / drug effects
  • Influenza A Virus, H1N1 Subtype / enzymology*
  • Mice
  • Models, Molecular
  • Morpholines / adverse effects
  • Morpholines / chemistry*
  • Morpholines / pharmacokinetics
  • Morpholines / pharmacology*
  • Protein Conformation
  • Pyridones / adverse effects
  • Pyridones / chemistry*
  • Pyridones / pharmacokinetics
  • Pyridones / pharmacology*
  • Tissue Distribution
  • Triazines / adverse effects
  • Triazines / chemistry*
  • Triazines / pharmacokinetics
  • Triazines / pharmacology*

Substances

  • Dibenzothiepins
  • Enzyme Inhibitors
  • Morpholines
  • Pyridones
  • Triazines
  • baloxavir
  • Endonucleases