Potent fibrinolysis inhibitor discovered by shape and electrostatic complementarity to the drug tranexamic acid

J Med Chem. 2013 Apr 25;56(8):3273-80. doi: 10.1021/jm301818g. Epub 2013 Apr 10.

Abstract

Protein-protein interfaces provide an important class of drug targets currently receiving increased attention. The typical design strategy to inhibit protein-protein interactions usually involves large molecules such as peptides and macrocycles. One exception is tranexamic acid (TXA), which, as a lysine mimetic, inhibits binding of plasminogen to fibrin. However, the daily dose of TXA is high due to its modest potency and pharmacokinetic properties. In this study, we report a computational approach, where the focus was on finding electrostatic potential similarities to TXA. Coupling this computational technique with a high-quality low-throughput screen identified 5-(4-piperidyl)-3-isoxazolol (4-PIOL) as a potent plasminogen binding inhibitor with the potential for the treatment of various bleeding disorders. Remarkably, 4-PIOL was found to be more than four times as potent as the drug TXA.

MeSH terms

  • Antifibrinolytic Agents / chemistry
  • Antifibrinolytic Agents / pharmacology*
  • Computational Biology
  • Drug Evaluation, Preclinical
  • Fibrinolysis / drug effects*
  • Humans
  • Isoxazoles / chemistry
  • Isoxazoles / pharmacology*
  • Models, Molecular
  • Piperidines / chemistry
  • Piperidines / pharmacology*
  • Plasminogen / metabolism
  • Protein Binding
  • Static Electricity
  • Tranexamic Acid / pharmacology

Substances

  • Antifibrinolytic Agents
  • Isoxazoles
  • Piperidines
  • 5-(4-piperidyl)isoxazol-3-ol
  • Tranexamic Acid
  • Plasminogen