A 7-deaza-adenosine analog is a potent and selective inhibitor of hepatitis C virus replication with excellent pharmacokinetic properties

Antimicrob Agents Chemother. 2004 Oct;48(10):3944-53. doi: 10.1128/AAC.48.10.3944-3953.2004.

Abstract

Improved treatments for chronic hepatitis C virus (HCV) infection are needed due to the suboptimal response rates and deleterious side effects associated with current treatment options. The triphosphates of 2'-C-methyl-adenosine and 2'-C-methyl-guanosine were previously shown to be potent inhibitors of the HCV RNA-dependent RNA polymerase (RdRp) that is responsible for the replication of viral RNA in cells. Here we demonstrate that the inclusion of a 7-deaza modification in a series of purine nucleoside triphosphates results in an increase in inhibitory potency against the HCV RdRp and improved pharmacokinetic properties. Notably, incorporation of the 7-deaza modification into 2'-C-methyl-adenosine results in an inhibitor with a 20-fold-increased potency as the 5'-triphosphate in HCV RdRp assays while maintaining the inhibitory potency of the nucleoside in the bicistronic HCV replicon and with reduced cellular toxicity. In contrast, while 7-deaza-2'-C-methyl-GTP also displays enhanced inhibitory potency in enzyme assays, due to poor cellular penetration and/or metabolism, the nucleoside does not inhibit replication of a bicistronic HCV replicon in cell culture. 7-Deaza-2'-C-methyl-adenosine displays promising in vivo pharmacokinetics in three animal species, as well as an acute oral lethal dose in excess of 2,000 mg/kg of body weight in mice. Taken together, these data demonstrate that 7-deaza-2'-C-methyl-adenosine is an attractive candidate for further investigation as a potential treatment for HCV infection.

MeSH terms

  • Animals
  • Antiviral Agents*
  • Culture Techniques
  • Drug Resistance, Viral
  • Female
  • Genotype
  • Hepacivirus / drug effects*
  • Hepacivirus / enzymology
  • Hepatitis C / drug therapy*
  • Hepatitis C / enzymology
  • Hepatitis C / metabolism*
  • Humans
  • Jurkat Cells
  • Lethal Dose 50
  • Mice
  • Polynucleotide Adenylyltransferase / metabolism
  • RNA / biosynthesis
  • RNA Polymerase II / metabolism
  • RNA-Dependent RNA Polymerase / metabolism
  • Thymidine / pharmacology
  • Tubercidin / pharmacokinetics*
  • Tubercidin / pharmacology*
  • Virus Replication / drug effects

Substances

  • Antiviral Agents
  • RNA
  • RNA Polymerase II
  • Polynucleotide Adenylyltransferase
  • RNA-Dependent RNA Polymerase
  • Tubercidin
  • Thymidine