• Proof-of-concept in the Phase II TITAN study was achieved with significant reductions in time to platelet count normalisation and recurrences while on treatment with caplacizumab
• Based on these results, Ablynx is on track to file for conditional approval of caplacizumab in Europe in the first half of 2017
• A confirmatory international Phase III study in patients with acquired TTP is ongoing and will be used to support a BLA submission in 2018 in the USA
• Ablynx intends to lead the commercialisation of caplacizumab in Europe and the USA
Ghent, Belgium, 11 February 2016 - Ablynx [Euronext Brussels: ABLX; OTC: ABYLY] today announced that the results of the Company’s worldwide Phase II TITAN study1 with caplacizumab for patients with acquired thrombotic thrombocytopenic purpura (aTTP) have been published in today’s issue of the New England Journal of Medicine (NEJM). “Caplacizumab has the potential to become an important new component in the standard of care for patients with acquired TTP” said Professor Flora Peyvandi, Principal Investigator for the TITAN study at IRCCS Maggiore Hospital Foundation, University of Milan, Italy, and lead author of the NEJM paper. “The results from the Phase II TITAN study showed that caplacizumab acts quickly to control the critical acute phase of the disease and protects patients until immunosuppressive treatments take effect.”
Dr Robert K. Zeldin, Chief Medical Officer of Ablynx, commented: “The publication of the TITAN data in this high-impact clinical journal2 is a further validation of the potential of caplacizumab in the treatment of acquired TTP. This publication is the culmination of over a decade of work by Ablynx and its external collaborators. We are on track to file for conditional approval of caplacizumab in Europe in 2017 and to complete enrolment of the confirmatory Phase III study before the end of 2017. We look forward to making caplacizumab available for patients with this devastating disease.”
About caplacizumab and the TITAN study results
Caplacizumab is a highly potent and selective bivalent anti-von Willebrand Factor (vWF) Nanobody® that received Orphan Drug Designation in the USA and EU in 2009. Caplacizumab inhibits the interaction between ultra-large vWF and platelets by targeting the A1 domain of vWF. It thereby prevents platelet aggregation and the formation of micro-clots during the acute, critical phase of acquired TTP.
Caplacizumab’s clinical effect was demonstrated in the Phase II TITAN study in 75 patients with aTTP:
• As indicated by a nearly 40% reduction in median time to platelet count normalisation (p = 0.005). Treatment with caplacizumab reduced the use of daily plasma exchange (PEX) and prevented further consumption of platelets in microthrombi and small blood vessel occlusion.
• As shown by the low number of recurrences requiring re-initiation of daily plasma exchange during treatment with caplacizumab (N=3) vs. placebo (N=11).
These results will serve as the basis for filing for conditional approval in Europe in H1 2017. Caplacizumab could be the first drug specifically approved for the treatment of acquired TTP.
More information on caplacizumab, including the NEJM paper, can be found on Ablynx’s website.
1Top line data from the TITAN study were communicated in June 2014 and were subsequently presented at ASH 2014 and ISTH 2015 (results from post hoc analysis and ADAMTS13 activity to guide treatment duration)
2The most recent (2014) impact factor for NEJM is 55.873, the highest among general medical journals
