Discovery of Selective Inhibitors of Endoplasmic Reticulum Aminopeptidase 1

J Med Chem. 2020 Jan 9;63(1):103-121. doi: 10.1021/acs.jmedchem.9b00293. Epub 2019 Dec 30.

Abstract

ERAP1 is an endoplasmic reticulum-resident zinc aminopeptidase that plays an important role in the immune system by trimming peptides for loading onto major histocompatibility complex proteins. Here, we report discovery of the first inhibitors selective for ERAP1 over its paralogues ERAP2 and IRAP. Compound 1 (N-(N-(2-(1H-indol-3-yl)ethyl)carbamimidoyl)-2,5-difluorobenzenesulfonamide) and compound 2 (1-(1-(4-acetylpiperazine-1-carbonyl)cyclohexyl)-3-(p-tolyl)urea) are competitive inhibitors of ERAP1 aminopeptidase activity. Compound 3 (4-methoxy-3-(N-(2-(piperidin-1-yl)-5-(trifluoromethyl)phenyl)sulfamoyl)benzoic acid) allosterically activates ERAP1's hydrolysis of fluorogenic and chromogenic amino acid substrates but competitively inhibits its activity toward a nonamer peptide representative of physiological substrates. Compounds 2 and 3 inhibit antigen presentation in a cellular assay. Compound 3 displays higher potency for an ERAP1 variant associated with increased risk of autoimmune disease. These inhibitors provide mechanistic insights into the determinants of specificity for ERAP1, ERAP2, and IRAP and offer a new therapeutic approach of specifically inhibiting ERAP1 activity in vivo.

MeSH terms

  • Aminopeptidases / antagonists & inhibitors*
  • Aminopeptidases / genetics
  • Aminopeptidases / metabolism
  • Catalytic Domain / genetics
  • Drug Discovery
  • HeLa Cells
  • Humans
  • Minor Histocompatibility Antigens / genetics
  • Minor Histocompatibility Antigens / metabolism
  • Molecular Docking Simulation
  • Molecular Structure
  • Phenylurea Compounds / chemical synthesis
  • Phenylurea Compounds / metabolism
  • Phenylurea Compounds / pharmacology*
  • Polymorphism, Single Nucleotide
  • Protease Inhibitors / chemical synthesis
  • Protease Inhibitors / metabolism
  • Protease Inhibitors / pharmacology*
  • Protein Binding
  • Structure-Activity Relationship
  • Sulfonamides / chemical synthesis
  • Sulfonamides / metabolism
  • Sulfonamides / pharmacology*
  • Tryptamines / chemical synthesis
  • Tryptamines / metabolism
  • Tryptamines / pharmacology*

Substances

  • Minor Histocompatibility Antigens
  • Phenylurea Compounds
  • Protease Inhibitors
  • Sulfonamides
  • Tryptamines
  • Aminopeptidases
  • ERAP1 protein, human