A Chemical Switch for Transforming a Purine Agonist for Toll-like Receptor 7 to a Clinically Relevant Antagonist

J Med Chem. 2020 May 14;63(9):4776-4789. doi: 10.1021/acs.jmedchem.0c00011. Epub 2020 May 4.

Abstract

Toll-like receptor 7 (TLR7) is an established therapeutic target in myriad autoimmune disorders, but no TLR7 antagonist is available for clinical use to date. Herein, we report a purine scaffold TLR7 antagonist, first-of-its-kind to our knowledge, which was developed by rationally dissecting the structural requirements for TLR7-targeted activity for a purine scaffold. Specifically, we identified a singular chemical switch at C-2 that could make a potent purine scaffold TLR7 agonist to lose agonism and acquire antagonist activity, which could further be potentiated by the introduction of an additional basic center at C-6. We ended up developing a clinically relevant TLR7 antagonist with favorable pharmacokinetics and 70.8% oral bioavailability in mice. Moreover, the TLR7 antagonists depicted excellent selectivity against TLR8. To further validate the in vivo applicability of this novel TLR7 antagonist, we demonstrated its excellent efficacy in preventing TLR7-induced pathology in a preclinical murine model of psoriasis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Binding Sites
  • Caco-2 Cells
  • Dermatologic Agents / chemical synthesis
  • Dermatologic Agents / metabolism
  • Dermatologic Agents / pharmacokinetics
  • Dermatologic Agents / therapeutic use*
  • HEK293 Cells
  • Humans
  • Male
  • Mice, Inbred C57BL
  • Molecular Docking Simulation
  • Molecular Structure
  • Psoriasis / drug therapy
  • Psoriasis / pathology
  • Purines / chemical synthesis
  • Purines / metabolism
  • Purines / pharmacokinetics
  • Purines / therapeutic use*
  • Skin / pathology
  • Structure-Activity Relationship
  • Toll-Like Receptor 7 / agonists*
  • Toll-Like Receptor 7 / antagonists & inhibitors*
  • Toll-Like Receptor 7 / metabolism

Substances

  • Dermatologic Agents
  • Purines
  • TLR7 protein, human
  • Toll-Like Receptor 7