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news.Biopharmaceutical firm Ablynx has reported positive results from the Phase I trial of its anti-von Willebrand factor Nanobody caplacizumab to treat acquired thrombotic thrombocytopenic purpura (TTP), a rare disorder of the blood coagulation system.
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The trial showed bioequivalence between the liquid and lyophilised formulations of caplacizumab.
To date, the liquid formulation has been used in clinical trials but the lyophilised form is more stable and can be stored and transported at 5*C. The company said that the liquid formulation must be stored and shipped frozen.
Ablynx CEO Dr Edwin Moses said: "We believe that this lyophilised form has a number of advantages and will allow for convenient self-administration in the home setting. We are on track to starting the Phase III study with this new lyophilised formulation in patients with acquired TTP in mid-2015."
The lyophilised form would be the formulation of choice for commercialization.
A total of 24 healthy male subjects were part of the single-centre, open-label, randomised, single dose cross-over Phase I trial, which evaluated the bioequivalence and tolerability of liquid and reconstituted lyophilised formulations of caplacizumab administered subcutaneously.
The bioequivalence criteria were based on the assessment of the area under the plasma concentration versus time curve (AUC) as well as the maximum observed plasma concentration (Cmax).
Based on the pharmacokinetic parameters, the results from the trial show that the lyophilised and liquid formulations of caplacizumab administered subcutaneously are bioequivalent.
Additionally, both formulations were well tolerated and no treatment emergent serious adverse events were reported.