Preclinical characterization of the novel hepatitis C virus NS3 protease inhibitor GS-9451

Antimicrob Agents Chemother. 2014;58(2):647-53. doi: 10.1128/AAC.00487-13. Epub 2013 Aug 12.

Abstract

GS-9451 is a selective hepatitis C virus (HCV) NS3 protease inhibitor in development for the treatment of genotype 1 (GT1) HCV infection. Key preclinical properties of GS-9451, including in vitro antiviral activity, selectivity, cross-resistance, and combination activity, as well as pharmacokinetic properties, were determined. In multiple GT1a and GT1b replicon cell lines, GS-9451 had mean 50% effective concentrations (EC50s) of 13 and 5.4 nM, respectively, with minimal cytotoxicity; similar potency was observed in chimeric replicons encoding the NS3 protease gene of GT1 clinical isolates. GS-9451 was less active in GT2a replicon cells (EC50 = 316 nM). Additive to synergistic in vitro antiviral activity was observed when GS-9451 was combined with other agents, including alpha interferon, ribavirin, and the polymerase inhibitors GS-6620 and tegobuvir (GS-9190), as well as the NS5A inhibitor ledipasvir (GS-5885). GS-9451 retained wild-type activity against multiple classes of NS5B and NS5A inhibitor resistance mutations. GS-9451 was stable in hepatic microsomes and hepatocytes from human and three other tested species. Systemic clearance was low in dogs and monkeys but high in rats. GS-9451 showed good oral bioavailability in all three species tested. In rats, GS-9451 levels were ∼40-fold higher in liver than plasma after intravenous dosing, and elimination of GS-9451 was primarily through biliary excretion. Together, these results are consistent with the antiviral activity observed in a recent phase 1b study. The results of in vitro cross-resistance and combination antiviral assays support the ongoing development of GS-9451 in combination with other agents for the treatment of chronic HCV infection.

MeSH terms

  • Animals
  • Antiviral Agents / pharmacokinetics
  • Antiviral Agents / pharmacology*
  • Benzimidazoles / pharmacology
  • Dogs
  • Drug Evaluation, Preclinical
  • Drug Resistance, Viral / drug effects
  • Drug Resistance, Viral / genetics
  • Drug Synergism
  • Drug Therapy, Combination
  • Fluorenes / pharmacology
  • Haplorhini
  • Hepacivirus / drug effects*
  • Hepacivirus / physiology
  • Hepatitis C, Chronic / drug therapy*
  • Hepatitis C, Chronic / pathology
  • Hepatitis C, Chronic / virology
  • Hepatocytes / drug effects
  • Hepatocytes / virology
  • Humans
  • Inhibitory Concentration 50
  • Interferon-alpha / pharmacology
  • Protease Inhibitors / pharmacokinetics
  • Protease Inhibitors / pharmacology*
  • Purines / pharmacology
  • Pyridazines / pharmacology
  • Quinolines / pharmacokinetics
  • Quinolines / pharmacology*
  • Rats
  • Replicon / drug effects
  • Ribavirin / pharmacology
  • Viral Nonstructural Proteins / antagonists & inhibitors*
  • Viral Nonstructural Proteins / metabolism

Substances

  • Antiviral Agents
  • Benzimidazoles
  • Fluorenes
  • GS-9451
  • Interferon-alpha
  • NS3 protein, hepatitis C virus
  • Protease Inhibitors
  • Purines
  • Pyridazines
  • Quinolines
  • Viral Nonstructural Proteins
  • ledipasvir
  • Ribavirin
  • tegobuvir