GHENT, Belgium, June 17, 2015 (GLOBE NEWSWIRE) -- Ablynx [Euronext Brussels: ABLX; OTC: ABYLY] today announced that additional data from the post-hoc analysis of the worldwide Phase II TITAN study with its wholly-owned anti-von Willebrand Factor (vWF) Nanobody®, caplacizumab, in patients with acquired thrombotic thrombocytopenic purpura (TTP), will be presented at the 2015 Annual Meeting of the International Society on Thrombosis and Haemostasis (ISTH), being held from 20-25 June 2015 in Toronto, Canada.
The efficacy and safety of caplacizumab in conjunction with the standard of care were evaluated in the TITAN study and the primary endpoint of a reduction in time to platelet normalization was achieved. Additional post-hoc analyses were performed on plasma exchange (PE) parameters and biomarkers of organ damage.
The following abstract will be presented in a late-breaking oral session by Professor Dr Flora Peyvandi (Principal Investigator for the TITAN study at IRCCS Maggiore Hospital Foundation, University of Milan, Italy) on Wednesday 24 June 2015 at 8.00am EDT (14.00h CET) (Plenary Hall F&G):
- Abstract LB006: "Additional data from the TITAN trial with the anti-vWF Nanobody, caplacizumab, in the treatment of acquired TTP", available on the ISTH website at http://www.isth.org/page/2015AbstractOnline.
In the majority of patients with acquired TTP, the activity of the ADAMTS13 enzyme is impaired due to the formation of auto-antibodies against the enzyme. The potential of ADAMTS13 levels as a predictive marker of underlying disease activity and subsequent exacerbations and relapses was evaluated, using data from the TITAN study.
The following abstract will be presented in an oral session by Filip Callewaert (Clinical Scientist, Department of Clinical Development, Ablynx) on Wednesday 24 June 2015 at 2.30pm EDT (20.30h CET) (Room 705):
- Abstract OR363: "The predictive value of ADAMTS13 activity for treatment monitoring of patients with acquired TTP; data from the Phase II TITAN trial with caplacizumab", available on the ISTH website at http://www.isth.org/page/2015AbstractOnline.
The key take-aways from the Phase II TITAN study with caplacizumab will also be presented during the session 'Highlights of ISTH' on 25 June 2015 at 12:00am ETD (18:00h CET) which provides a synopsis of the top thrombosis and haemostasis research presented at the congress.
Shortly after the conference, both oral presentations will be made available on the Ablynx website under the R&D portfolio section.
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 the formation of unwanted characteristic string-like clots in small blood vessels. In healthy subjects, UL-vWF are immediately cleaved into smaller, regular-sized multimers by the ADAMTS13 enzyme. In patients with acquired TTP, activity of the ADAMTS13 enzyme is impaired (<10%) due to the formation of auto-antibodies against the enzyme. In some cases, ADAMTS13 activity is normal (>10%) but UL-vWF are present as a result of massive endothelial stimulation with consequent release of UL-vWF multimers in amounts exceeding the system's ability to degrade them.
Caplacizumab inhibits platelet binding to UL-vWF and thus has the potential to prevent the formation of these string-like clots in the blood of patients with acquired TTP. Importantly, caplacizumab selectively prevents thrombus formation in high-shear blood vessels and is expected not to interact with haemostasis in normal, healthy blood vessels.
Ablynx expects to start the Phase III registration study for caplacizumab in Europe, the US and Canada, in H2 2015. In parallel, the Company will make further preparations to file for conditional approval for caplacizumab in Europe in 2017. At the same time, Ablynx is exploring the optimal value-creating commercial strategy for this asset and expects to announce its conclusions later this year.
About Phase II TITAN trial
The worldwide Phase II TITAN clinical trial was a single-blinded, randomised, placebo-controlled study which recruited from January 2011 to January 2014. In total, 75 patients were randomized on a 1:1 basis with one active drug treatment arm and one placebo arm. All patients received the current standard of care which is primarily multiple plasma exchanges. The protocol for the study was adapted in September 2013, to also allow one day of plasma exchange prior to study enrolment. Those patients in the active drug treatment arm immediately received an intravenous bolus dose of 10 mg caplacizumab and then a 10 mg subcutaneous dose of the drug daily until 30 days had elapsed after the final plasma exchange. Patients in the control arm received placebo at the same time points.
TTP is a rare disorder of the blood coagulation system that causes extensive microscopic thromboses in small blood vessels throughout the body. It is a potentially life-threatening disorder characterised by thrombocytopenia, haemolytic anaemia and microvascular thrombosis causing variable degrees of tissue ischemia and infarction. TTP exists in two forms: a congenital and an acquired form, with the latter accounting for >90% of the patients. There are currently no drugs specifically approved for the treatment of TTP. The standard of care for the acquired form of TTP is multiple daily plasma exchanges (PE) until confirmed platelet normalisation which occurs when the patient's platelet count returns to normal, as well as immunosuppressant treatment. Daily PE requires lengthy hospital stays and may be associated with clinical complications. Additionally, a significant number of patients will subsequently suffer a relapse after recovering from a first TTP episode. There are believed to be approximately 10,000 TTP-related events in the US and top 15 European markets per year.
Ablynx is a biopharmaceutical company engaged in the development of Nanobodies®, proprietary therapeutic proteins based on single-domain antibody fragments, which combine the advantages of conventional antibody drugs with some of the features of small-molecule drugs. Ablynx is dedicated to creating new medicines which will make a real difference to society. Today, the Company has more than 30 proprietary and partnered programmes in development in various therapeutic areas including inflammation, haematology, immuno-oncology, oncology and respiratory disease. The Company has collaborations with multiple pharmaceutical companies including AbbVie, Boehringer Ingelheim, Eddingpharm, Genzyme, Merck & Co, Inc., Merck Serono and Novartis. The Company is headquartered in Ghent, Belgium. More information can be found on www.ablynx.com.
For more information, please contact Ablynx:
Dr Edwin Moses
t: +32 (0)9 262 00 07
m: +32 (0)473 39 50 68
Associate Director Investor Relations
t: +32 (0)9 262 00 82
m: +32 (0)479 49 06 03
Ablynx media relations Consilium Strategic Communications:
Mary-Jane Elliott, Jonathan Birt, Chris Welsh, Lindsey Neville
t: +44 203 709 5700
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