Discovery of GS-9688 (Selgantolimod) as a Potent and Selective Oral Toll-Like Receptor 8 Agonist for the Treatment of Chronic Hepatitis B

J Med Chem. 2020 Sep 24;63(18):10188-10203. doi: 10.1021/acs.jmedchem.0c00100. Epub 2020 Jun 3.

Abstract

Toll-like receptor 8 (TLR8) recognizes pathogen-derived single-stranded RNA fragments to trigger innate and adaptive immune responses. Chronic hepatitis B (CHB) is associated with a dysfunctional immune response, and therefore a selective TLR8 agonist may be an effective treatment option. Structure-based optimization of a dual TLR7/8 agonist led to the identification of the selective TLR8 clinical candidate (R)-2-((2-amino-7-fluoropyrido[3,2-d]pyrimidin-4-yl)amino)-2-methylhexan-1-ol (GS-9688, (R)-7). Potent TLR8 agonism (IL-12p40 EC50 = 220 nM) and >100-fold TLR7 selectivity (IFN-α EC50 > 50 μM) was observed in human peripheral blood mononuclear cells (PBMCs). The TLR8-ectodomain:(R)-7 complex confirmed TLR8 binding and a direct ligand interaction with TLR8 residue Asp545. Oral (R)-7 had good absorption and high first pass clearance in preclinical species. A reduction in viral markers was observed in HBV-infected primary human hepatocytes treated with media from PBMCs stimulated with (R)-7, supporting the clinical development of (R)-7 for the treatment of CHB.

MeSH terms

  • Administration, Oral
  • Animals
  • Antiviral Agents / administration & dosage
  • Antiviral Agents / chemical synthesis
  • Antiviral Agents / metabolism
  • Antiviral Agents / pharmacology*
  • Crystallography, X-Ray
  • Dogs
  • Drug Discovery
  • Hepatitis B virus / drug effects
  • Hepatitis B, Chronic / drug therapy*
  • Hexanols / administration & dosage
  • Hexanols / chemical synthesis
  • Hexanols / metabolism
  • Hexanols / pharmacology*
  • Humans
  • Macaca fascicularis
  • Molecular Structure
  • Protein Domains
  • Pyridines / administration & dosage
  • Pyridines / chemical synthesis
  • Pyridines / metabolism
  • Pyridines / pharmacology*
  • Pyrimidines / administration & dosage
  • Pyrimidines / chemical synthesis
  • Pyrimidines / metabolism
  • Pyrimidines / pharmacology*
  • Rats
  • Structure-Activity Relationship
  • Toll-Like Receptor 8 / agonists*
  • Toll-Like Receptor 8 / metabolism

Substances

  • Antiviral Agents
  • Hexanols
  • Pyridines
  • Pyrimidines
  • TLR8 protein, human
  • Toll-Like Receptor 8
  • selgantolimod