Prodrugs of a 1'-CN-4-Aza-7,9-dideazaadenosine C-Nucleoside Leading to the Discovery of Remdesivir (GS-5734) as a Potent Inhibitor of Respiratory Syncytial Virus with Efficacy in the African Green Monkey Model of RSV

J Med Chem. 2021 Apr 22;64(8):5001-5017. doi: 10.1021/acs.jmedchem.1c00071. Epub 2021 Apr 9.

Abstract

A discovery program targeting respiratory syncytial virus (RSV) identified C-nucleoside 4 (RSV A2 EC50 = 530 nM) as a phenotypic screening lead targeting the RSV RNA-dependent RNA polymerase (RdRp). Prodrug exploration resulted in the discovery of remdesivir (1, GS-5734) that is >30-fold more potent than 4 against RSV in HEp-2 and NHBE cells. Metabolism studies in vitro confirmed the rapid formation of the active triphosphate metabolite, 1-NTP, and in vivo studies in cynomolgus and African Green monkeys demonstrated a >10-fold higher lung tissue concentration of 1-NTP following molar normalized IV dosing of 1 compared to that of 4. A once daily 10 mg/kg IV administration of 1 in an African Green monkey RSV model demonstrated a >2-log10 reduction in the peak lung viral load. These early data following the discovery of 1 supported its potential as a novel treatment for RSV prior to its development for Ebola and approval for COVID-19 treatment.

MeSH terms

  • Adenosine Monophosphate / analogs & derivatives*
  • Adenosine Monophosphate / pharmacology
  • Alanine / analogs & derivatives*
  • Alanine / pharmacology
  • Animals
  • Antiviral Agents / chemistry
  • Antiviral Agents / pharmacokinetics
  • Antiviral Agents / pharmacology*
  • Caco-2 Cells
  • Cells, Cultured
  • Chlorocebus aethiops
  • Disease Models, Animal
  • Dogs
  • Drug Evaluation, Preclinical / methods
  • Epithelial Cells / virology
  • Humans
  • Macaca fascicularis
  • Male
  • Prodrugs / chemistry
  • Prodrugs / pharmacokinetics
  • Prodrugs / pharmacology*
  • Rats
  • Rats, Sprague-Dawley
  • Respiratory Syncytial Virus Infections / drug therapy*
  • Respiratory Syncytial Virus Infections / virology
  • Respiratory Syncytial Virus, Human / drug effects*
  • Structure-Activity Relationship
  • Tissue Distribution
  • Tubercidin / analogs & derivatives
  • Tubercidin / chemistry
  • Viral Load

Substances

  • Antiviral Agents
  • Prodrugs
  • 1,3-dideazaadenosine
  • remdesivir
  • Adenosine Monophosphate
  • Tubercidin
  • Alanine