Design, Synthesis, and Bioevaluation of Novel NLRP3 Inhibitor with IBD Immunotherapy from the Virtual Screen

J Med Chem. 2024 Sep 26;67(18):16612-16634. doi: 10.1021/acs.jmedchem.4c01445. Epub 2024 Sep 13.

Abstract

NLRP3, a crucial member of the NLRP family, plays a pivotal role in immune regulation and inflammatory modulation. Here, we report a potent and specific NLRP3 inhibitor Z48 obtained though docking-based virtual screening and structure-activity relationship studies with an IC50 of 0.26 μM in THP-1 cells and 0.21 μM in mouse bone marrow-derived macrophages. Mechanistic studies indicated that Z48 could bind directly to the NLRP3 protein (KD = 1.05 μM), effectively blocking the assembly and activation of the NLRP3 inflammasome, consequently manifesting anti-inflammatory properties. Crucially, with acceptable mouse pharmacokinetic profiles, Z48 demonstrated notable therapeutic efficacy in a mouse model of DSS-induced ulcerative colitis, while displaying no significant therapeutic impact on NLRP3KO mice. In conclusion, this study provided a promising NLRP3 inflammasome inhibitor with novel molecular scaffold, poised for further development as a therapeutic candidate in the treatment of inflammatory bowel disease.

MeSH terms

  • Animals
  • Colitis, Ulcerative / drug therapy
  • Drug Design*
  • Humans
  • Immunotherapy / methods
  • Inflammasomes / antagonists & inhibitors
  • Inflammasomes / metabolism
  • Inflammatory Bowel Diseases / drug therapy
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Molecular Docking Simulation
  • NLR Family, Pyrin Domain-Containing 3 Protein* / antagonists & inhibitors
  • NLR Family, Pyrin Domain-Containing 3 Protein* / metabolism
  • Structure-Activity Relationship
  • THP-1 Cells

Substances

  • NLR Family, Pyrin Domain-Containing 3 Protein
  • Inflammasomes
  • Nlrp3 protein, mouse
  • NLRP3 protein, human