Design, synthesis, and biological evaluation of peptidomimetic inhibitors of factor XIa as novel anticoagulants

J Med Chem. 2006 Dec 28;49(26):7781-91. doi: 10.1021/jm060978s.

Abstract

Human coagulation factor XIa (FXIa), a serine protease activated by site-specific cleavage of factor XI by thrombin, FXIIa, or autoactivation, is a critical enzyme in the amplification phase of the coagulation cascade. To investigate the potential of FXIa inhibitors as safe anticoagulants, a series of potent, selective peptidomimetic inhibitors of FXIa were designed and synthesized. Some of these inhibitors showed low nanomolar FXIa inhibitory activity with >1000-fold FXa selectivity and >100-fold thrombin selectivity. The X-ray structure of one of these inhibitors, 36, demonstrates its unique binding interactions with FXIa. Compound 32 caused a doubling of the activated partial thromboplastin time in human plasma at 2.4 microM and was efficacious in a rat model of venous thrombosis. These data suggest that factor XIa plays a significant role in venous thrombosis and may be a suitable target for the development of antithrombotic therapy.

Publication types

  • Evaluation Study

MeSH terms

  • Animals
  • Anticoagulants / chemical synthesis
  • Anticoagulants / chemistry
  • Anticoagulants / pharmacology*
  • Binding Sites
  • Crystallography, X-Ray
  • Drug Design*
  • Factor XIa / antagonists & inhibitors*
  • Factor Xa Inhibitors*
  • Humans
  • Inhibitory Concentration 50
  • Male
  • Molecular Structure
  • Partial Thromboplastin Time
  • Peptide Fragments / chemical synthesis*
  • Peptide Fragments / chemistry
  • Peptide Fragments / pharmacology
  • Protein Binding
  • Protein Conformation
  • Rats
  • Rats, Sprague-Dawley
  • Serine Proteinase Inhibitors / chemical synthesis
  • Serine Proteinase Inhibitors / chemistry
  • Serine Proteinase Inhibitors / pharmacology*
  • Structure-Activity Relationship
  • Thrombin / antagonists & inhibitors
  • Venous Thrombosis / drug therapy

Substances

  • Anticoagulants
  • Factor Xa Inhibitors
  • Peptide Fragments
  • Serine Proteinase Inhibitors
  • Factor XIa
  • Thrombin