Discovery of a novel, first-in-class, orally bioavailable azaindole inhibitor (VX-787) of influenza PB2

J Med Chem. 2014 Aug 14;57(15):6668-78. doi: 10.1021/jm5007275. Epub 2014 Jul 24.

Abstract

In our effort to develop agents for the treatment of influenza, a phenotypic screening approach utilizing a cell protection assay identified a series of azaindole based inhibitors of the cap-snatching function of the PB2 subunit of the influenza A viral polymerase complex. Using a bDNA viral replication assay (Wagaman, P. C., Leong, M. A., and Simmen, K. A. Development of a novel influenza A antiviral assay. J. Virol. Methods 2002, 105, 105-114) in cells as a direct measure of antiviral activity, we discovered a set of cyclohexyl carboxylic acid analogues, highlighted by VX-787 (2). Compound 2 shows strong potency versus multiple influenza A strains, including pandemic 2009 H1N1 and avian H5N1 flu strains, and shows an efficacy profile in a mouse influenza model even when treatment was administered 48 h after infection. Compound 2 represents a first-in-class, orally bioavailable, novel compound that offers potential for the treatment of both pandemic and seasonal influenza and has a distinct advantage over the current standard of care treatments including potency, efficacy, and extended treatment window.

MeSH terms

  • Administration, Oral
  • Animals
  • Antiviral Agents / chemical synthesis
  • Antiviral Agents / chemistry*
  • Antiviral Agents / pharmacology
  • Aza Compounds / chemical synthesis
  • Aza Compounds / chemistry*
  • Aza Compounds / pharmacology
  • Biological Availability
  • Dogs
  • Drug Resistance, Viral
  • Indoles / chemical synthesis
  • Indoles / chemistry*
  • Indoles / pharmacology
  • Influenza A virus / drug effects
  • Influenza A virus / physiology
  • Madin Darby Canine Kidney Cells
  • Male
  • Mice, Inbred BALB C
  • Models, Molecular
  • Molecular Structure
  • Orthomyxoviridae Infections / drug therapy
  • RNA-Dependent RNA Polymerase / antagonists & inhibitors*
  • Rats
  • Species Specificity
  • Stereoisomerism
  • Structure-Activity Relationship
  • Viral Proteins / antagonists & inhibitors*
  • Virus Replication / drug effects

Substances

  • Antiviral Agents
  • Aza Compounds
  • Indoles
  • PB2 protein, Influenzavirus A
  • Viral Proteins
  • RNA-Dependent RNA Polymerase