Design of Potent Small-Molecule Stimulator of Interferon Gene Inhibitor and Stimulator of Interferon Gene Mutant-Specific Degrader

J Med Chem. 2025 Jun 12;68(11):11100-11126. doi: 10.1021/acs.jmedchem.5c00123. Epub 2025 May 19.

Abstract

Stimulator of interferon genes (STING) is involved in various autoimmune diseases. However, it is challenging to develop small-molecule STING inhibitors with potent activity. Herein, we designed a small-molecule STING inhibitor and STING mutant-specific degrader by binding two coupled pockets of a STING dimer. Structure optimization selected SI-24, SI-42, and SI-43 with low nanomolar activity to inhibit 2'3'-cyclic GMP-AMP (cGAMP)-induced STING activation and release of IFN-β and CXCL-10, which were far more potent than reported STING inhibitors. Moreover, the three lead compounds suppressed cGAMP-induced oligomerization of STING and phosphorylation of interferon regulatory factor 3 (IRF3) and STING. Surprisingly, SI-43 promoted mutant-specific and proteasome-independent degradation of STINGS154 and STINGM155. Subcutaneous or oral administration of SI-24, SI-42, and SI-43 reduced serum IFN-β and CXCL-10 in the cGAMP-induced autoimmune disease mouse model. Our dual-functional compounds provide a new strategy to investigate STING function through both inhibition and mutant-specific degradation in autoimmune diseases.

MeSH terms

  • Animals
  • Autoimmune Diseases / drug therapy
  • Chemokine CXCL10 / blood
  • Chemokine CXCL10 / metabolism
  • Drug Design*
  • HEK293 Cells
  • Humans
  • Interferon Regulatory Factor-3 / metabolism
  • Interferon-beta / blood
  • Interferon-beta / metabolism
  • Membrane Proteins* / antagonists & inhibitors
  • Membrane Proteins* / genetics
  • Membrane Proteins* / metabolism
  • Mice
  • Mice, Inbred C57BL
  • Mutation
  • Nucleotides, Cyclic
  • Small Molecule Libraries* / chemistry
  • Small Molecule Libraries* / pharmacology
  • Structure-Activity Relationship

Substances

  • Membrane Proteins
  • STING1 protein, human
  • Interferon-beta
  • Interferon Regulatory Factor-3
  • Chemokine CXCL10
  • Small Molecule Libraries
  • Nucleotides, Cyclic