Date: 2014-11-26
Type of
information: Results
phase: 1
Announcement: results
Company: Ablynx (Belgium)
Product: caplacizumab
Action
mechanism:
- nanobody. 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 and immunosuppressants. Efficacy and safety were demonstrated for caplacizumab in a worldwide Phase II study in patients with acquired TTP, demonstrating that it could act as a protective agent in the acute phase of the disease, preventing microthrombi and having the potential to reduce morbidity due to organ damage as well as preventing exacerbations. In addition, in this Phase II study, caplacizumab was generally well-tolerated and had a manageable bleeding side effect profile.
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. 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.
Disease: acquired thrombotic thrombocytopenic purpura (TTP)
Therapeutic
area: Autoimmune diseases - Rare diseases
Country:
Trial
details:
Latest
news:
- • On November 2014, Ablynx announced positive results from the Phase I trial to demonstrate bioequivalence between the liquid and lyophilised formulations of caplacizumab, Ablynx's anti-von Willebrand factor Nanobody developed for the treatment of acquired thrombotic thrombocytopenic purpura (TTP). The liquid formulation has been used in clinical trials to date but the lyophilised form is more stable and can be stored and transported at 5°C, which is much more convenient than the liquid formulation, which has to be stored and shipped frozen. The lyophilised form will now be used in the forthcoming Phase III trial and would be the formulation of choice for commercialisation.
- The Phase I trial involved a single-centre, open-label, randomised, single dose cross-over study in 24 healthy male subjects to evaluate the bioequivalence and tolerability of liquid and reconstituted lyophilised formulations of caplacizumab administered subcutaneously. The bioequivalence criteria were based on the evaluation of the "area under the plasma concentration versus time curve (AUC)" and the "maximum observed plasma concentration (Cmax)". The results from this study demonstrate that the lyophilised and liquid formulations of caplacizumab administered subcutaneously are bioequivalent based on the pharmacokinetic parameters as described above. In addition, both formulations were well tolerated and no treatment emergent serious adverse events were reported.
Is
general: Yes
