ABLYNX'S ANTI-vWF NANOBODY, CAPLACIZUMAB, ACHIEVES CLINICAL PROOF-OF-CONCEPT IN PHASE II TITAN STUDY - Seite 3

Dr Flora Peyvandi, Principal Investigator for the TITAN study at IRCCS Maggiore Hospital Foundation, University of Milan, Italy, commented: "The outcome of this study makes us believe that caplacizumab has the potential to become an important pillar in the management of acquired TTP. The results show that caplacizumab is able to control the acute phase of the disease and buys time for the later onset of the immunosuppressive treatment which is often needed to fully resolve a TTP episode."

Dr Dominique Tersago, Chief Medical Officer of Ablynx said: "The potential of caplacizumab to act as a protective agent for patients with acquired TTP has been demonstrated in this trial. Further and more detailed analyses of the study results are still ongoing and we look forward to working closely with clinical experts and regulatory authorities to ensure the design of an optimal Phase III trial which we plan to start in 2015."

Dr Edwin Moses, Chief Executive Officer of Ablynx added: "We are very pleased with the results of the study and the potential of our first-in-class anti-vWF Nanobody. It is our third Nanobody programme to achieve clinical proof-of-concept in the space of just three years, which we believe demonstrates the exceptional ability of our platform to deliver clinically meaningful assets. We will proceed with preparations for the start of a Phase III study in 2015 and in parallel we will be talking to potential partners and evaluating all of our strategic options for the further development and commercialisation of caplacizumab."

About caplacizumab

Caplacizumab is a bivalent anti-vWF Nanobody which is highly potent and selective. It received Orphan Drug Designation in the US and EU in 2009 and could be the first drug specifically approved for the treatment of acquired TTP as an adjunct to plasma exchange.

Von Willebrand factor (vWF) is a blood glycoprotein involved in haemostasis, a complex process that causes the bleeding process to stop. vWF's primary function is to bind to other proteins, including glycoprotein Ib in the initiation of platelet adhesion. vWF is implicated in TTP where ultra-large, multimeric precursors of vWF (UL-vWF) are present in the blood of patients leading to unwanted blood clot formation. UL-vWF can readily bind platelets leading to the formation of characteristic string-like clots in small blood vessels.