Discovery of highly potent and selective small molecule ADAMTS-5 inhibitors that inhibit human cartilage degradation via encoded library technology (ELT)

J Med Chem. 2012 Aug 23;55(16):7061-79. doi: 10.1021/jm300449x. Epub 2012 Aug 14.

Abstract

The metalloprotease ADAMTS-5 is considered a potential target for the treatment of osteoarthritis. To identify selective inhibitors of ADAMTS-5, we employed encoded library technology (ELT), which enables affinity selection of small molecule binders from complex mixtures by DNA tagging. Selection of ADAMTS-5 against a four-billion member ELT library led to a novel inhibitor scaffold not containing a classical zinc-binding functionality. One exemplar, (R)-N-((1-(4-(but-3-en-1-ylamino)-6-(((2-(thiophen-2-yl)thiazol-4-yl)methyl)amino)-1,3,5-triazin-2-yl)pyrrolidin-2-yl)methyl)-4-propylbenzenesulfonamide (8), inhibited ADAMTS-5 with IC(50) = 30 nM, showing >50-fold selectivity against ADAMTS-4 and >1000-fold selectivity against ADAMTS-1, ADAMTS-13, MMP-13, and TACE. Extensive SAR studies showed that potency and physicochemical properties of the scaffold could be further improved. Furthermore, in a human osteoarthritis cartilage explant study, compounds 8 and 15f inhibited aggrecanase-mediated (374)ARGS neoepitope release from aggrecan and glycosaminoglycan in response to IL-1β/OSM stimulation. This study provides the first small molecule evidence for the critical role of ADAMTS-5 in human cartilage degradation.

MeSH terms

  • ADAM Proteins / antagonists & inhibitors*
  • ADAMTS5 Protein
  • Aggrecans / metabolism
  • Animals
  • Cartilage, Articular / drug effects*
  • Cartilage, Articular / metabolism
  • Cartilage, Articular / pathology
  • Databases, Chemical*
  • Endopeptidases / metabolism
  • Epitopes
  • Glycosaminoglycans / metabolism
  • Humans
  • In Vitro Techniques
  • Male
  • Middle Aged
  • Osteoarthritis / drug therapy
  • Osteoarthritis / pathology*
  • Rats
  • Rats, Sprague-Dawley
  • Small Molecule Libraries
  • Stereoisomerism
  • Structure-Activity Relationship
  • Sulfonamides / chemical synthesis*
  • Sulfonamides / pharmacokinetics
  • Sulfonamides / pharmacology
  • Triazines / chemical synthesis*
  • Triazines / pharmacokinetics
  • Triazines / pharmacology

Substances

  • Aggrecans
  • Epitopes
  • Glycosaminoglycans
  • N-((1-(4-(but-3-en-1-ylamino)-6-(((2-(thiophen-2-yl)thiazol-4-yl)methyl)amino)-1,3,5-triazin-2-yl)pyrrolidin-2-yl)methyl)-4-propylbenzenesulfonamide
  • Small Molecule Libraries
  • Sulfonamides
  • Triazines
  • Endopeptidases
  • ADAM Proteins
  • ADAMTS5 Protein
  • ADAMTS5 protein, human
  • aggrecanase