Application of fragment screening and fragment linking to the discovery of novel thrombin inhibitors

J Med Chem. 2006 Feb 23;49(4):1346-55. doi: 10.1021/jm050850v.

Abstract

The screening of fragments is an alternative approach to high-throughput screening for the identification of leads for therapeutic targets. Fragment hits have been discovered using X-ray crystallographic screening of protein crystals of the serine protease enzyme thrombin. The fragment library was designed to avoid any well-precedented, strongly basic functionality. Screening hits included a novel ligand (3), which binds exclusively to the S2-S4 pocket, in addition to smaller fragments which bind to the S1 pocket. The structure of these protein-ligand complexes are presented. A chemistry strategy to link two such fragments together and to synthesize larger drug-sized compounds resulted in the efficient identification of hybrid inhibitors with nanomolar potency (e.g., 7, IC50 = 3.7 nM). These potent ligands occupy the same area of the active site as previously described peptidic inhibitors, while having very different chemical architecture.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Carbamates / chemical synthesis
  • Carbamates / chemistry
  • Crystallography, X-Ray
  • Databases, Factual
  • Humans
  • Models, Molecular*
  • Protein Conformation
  • Stereoisomerism
  • Structure-Activity Relationship
  • Sulfonamides / chemical synthesis
  • Sulfonamides / chemistry
  • Tetrazoles / chemical synthesis
  • Tetrazoles / chemistry
  • Thrombin / antagonists & inhibitors*
  • Thrombin / chemistry*

Substances

  • Carbamates
  • Sulfonamides
  • Tetrazoles
  • Thrombin