Discovery of 1-((2R,4aR,6R,7R,7aR)-2-Isopropoxy-2-oxidodihydro-4H,6H-spiro[furo[3,2-d][1,3,2]dioxaphosphinine-7,2'-oxetan]-6-yl)pyrimidine-2,4(1H,3H)-dione (JNJ-54257099), a 3'-5'-Cyclic Phosphate Ester Prodrug of 2'-Deoxy-2'-Spirooxetane Uridine Triphosphate Useful for HCV Inhibition

J Med Chem. 2016 Jun 23;59(12):5790-8. doi: 10.1021/acs.jmedchem.6b00382. Epub 2016 Jun 3.

Abstract

JNJ-54257099 (9) is a novel cyclic phosphate ester derivative that belongs to the class of 2'-deoxy-2'-spirooxetane uridine nucleotide prodrugs which are known as inhibitors of the HCV NS5B RNA-dependent RNA polymerase (RdRp). In the Huh-7 HCV genotype (GT) 1b replicon-containing cell line 9 is devoid of any anti-HCV activity, an observation attributable to inefficient prodrug metabolism which was found to be CYP3A4-dependent. In contrast, in vitro incubation of 9 in primary human hepatocytes as well as pharmacokinetic evaluation thereof in different preclinical species reveals the formation of substantial levels of 2'-deoxy-2'-spirooxetane uridine triphosphate (8), a potent inhibitor of the HCV NS5B polymerase. Overall, it was found that 9 displays a superior profile compared to its phosphoramidate prodrug analogues (e.g., 4) described previously. Of particular interest is the in vivo dose dependent reduction of HCV RNA observed in HCV infected (GT1a and GT3a) human hepatocyte chimeric mice after 7 days of oral administration of 9.

MeSH terms

  • Administration, Oral
  • Animals
  • Antiviral Agents / administration & dosage
  • Antiviral Agents / chemistry
  • Antiviral Agents / pharmacology*
  • Dose-Response Relationship, Drug
  • Drug Discovery*
  • HIV Infections / drug therapy*
  • HIV Infections / virology
  • Hepacivirus / drug effects*
  • Hepatocytes / drug effects*
  • Hepatocytes / virology
  • Humans
  • Mice
  • Microbial Sensitivity Tests
  • Prodrugs / administration & dosage
  • Prodrugs / chemistry
  • Prodrugs / pharmacology*
  • Pyrimidinones / administration & dosage
  • Pyrimidinones / chemistry
  • Pyrimidinones / pharmacology*
  • Spiro Compounds / administration & dosage
  • Spiro Compounds / chemistry
  • Spiro Compounds / pharmacology*
  • Structure-Activity Relationship
  • Viral Nonstructural Proteins / antagonists & inhibitors
  • Virus Replication / drug effects

Substances

  • 1-2-isopropoxy-2-oxidodihydro-4H,6H-spiro(furo(3,2-d)(1,3,2)dioxaphosphinine-7,2'-oxetan)-6-yl)pyrimidine-2,4(1H,3H)-dione
  • Antiviral Agents
  • Prodrugs
  • Pyrimidinones
  • Spiro Compounds
  • Viral Nonstructural Proteins
  • NS-5 protein, hepatitis C virus