Novel antiplatelet agents: ALX-0081, a Nanobody directed towards von Willebrand factor

J Cardiovasc Transl Res. 2013 Jun;6(3):355-63. doi: 10.1007/s12265-012-9435-y. Epub 2013 Jan 11.

Abstract

This manuscript reviews the studies performed with ALX-0081 (INN: caplacizumab), a Nanobody targeting von Willebrand factor, in the context of current antithrombotic therapy in coronary artery disease. ALX-0081 specifically inhibits platelet adhesion to the vessel wall, and may control platelet aggregation and subsequent clot formation without increasing bleeding risk. A substantial number of antithrombotics are aimed at this cascade; however, their generally indiscriminative mode of action can result in a narrow therapeutic window, defined by the risk for bleeding complications, and thrombotic events. Nonclinically, ALX-0081 compared favorably to several antithrombotics. In Phase I studies in healthy subjects and stable angina patients undergoing percutaneous coronary intervention (PCI), ALX-0081 was well tolerated, and effectively inhibited pharmacodynamic markers. Following these results, a phase II study was initiated in high-risk acute coronary syndrome patients undergoing PCI. Based on its mechanism of action, ALX-0081 is also being developed for acquired thrombotic thrombocytopenic purpura.

Publication types

  • Review

MeSH terms

  • Animals
  • Blood Platelets / drug effects*
  • Blood Platelets / immunology
  • Blood Platelets / metabolism
  • Coronary Artery Disease / blood
  • Coronary Artery Disease / immunology
  • Coronary Artery Disease / therapy*
  • Coronary Thrombosis / blood
  • Coronary Thrombosis / immunology
  • Coronary Thrombosis / prevention & control*
  • Fibrinolytic Agents / adverse effects
  • Fibrinolytic Agents / therapeutic use*
  • Hemorrhage / chemically induced
  • Humans
  • Percutaneous Coronary Intervention* / adverse effects
  • Platelet Aggregation Inhibitors / adverse effects
  • Platelet Aggregation Inhibitors / therapeutic use*
  • Single-Domain Antibodies / adverse effects
  • Single-Domain Antibodies / therapeutic use*
  • Treatment Outcome
  • von Willebrand Factor / antagonists & inhibitors*
  • von Willebrand Factor / immunology
  • von Willebrand Factor / metabolism

Substances

  • Fibrinolytic Agents
  • Platelet Aggregation Inhibitors
  • Single-Domain Antibodies
  • von Willebrand Factor