Discovery of a Highly Potent, Selective, and Orally Bioavailable Macrocyclic Inhibitor of Blood Coagulation Factor VIIa-Tissue Factor Complex

J Med Chem. 2016 Aug 11;59(15):7125-37. doi: 10.1021/acs.jmedchem.6b00469. Epub 2016 Aug 2.

Abstract

Inhibitors of the tissue factor (TF)/factor VIIa complex (TF-FVIIa) are promising novel anticoagulants which show excellent efficacy and minimal bleeding in preclinical models. Starting with an aminoisoquinoline P1-based macrocyclic inhibitor, optimization of the P' groups led to a series of highly potent and selective TF-FVIIa inhibitors which displayed poor permeability. Fluorination of the aminoisoquinoline reduced the basicity of the P1 group and significantly improved permeability. The resulting lead compound was highly potent, selective, and achieved good pharmacokinetics in dogs with oral dosing. Moreover, it demonstrated robust antithrombotic activity in a rabbit model of arterial thrombosis.

MeSH terms

  • Administration, Oral
  • Animals
  • Anticoagulants / administration & dosage
  • Anticoagulants / chemistry
  • Anticoagulants / pharmacology*
  • Biological Availability
  • Dogs
  • Dose-Response Relationship, Drug
  • Drug Discovery*
  • Factor VIIa / antagonists & inhibitors*
  • Factor VIIa / metabolism
  • Healthy Volunteers
  • Humans
  • Macrocyclic Compounds / administration & dosage
  • Macrocyclic Compounds / chemistry
  • Macrocyclic Compounds / pharmacology*
  • Male
  • Models, Molecular
  • Molecular Structure
  • Rabbits
  • Structure-Activity Relationship
  • Thromboplastin / antagonists & inhibitors*
  • Thromboplastin / metabolism

Substances

  • Anticoagulants
  • Macrocyclic Compounds
  • Thromboplastin
  • Factor VIIa