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).

The trial showed bioequivalence between the liquid and lyophilised formulations of caplacizumab.

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The liquid formulation has been used in clinical trials so far, 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.

"We believe that this lyophilised form has a number of advantages and will allow for convenient self-administration in the home setting."

The lyophilised form will now be used in the upcoming Phase III trial and would be the formulation of choice for commercialisation.

A total of 24 healthy male subjects were part of the single-centre, open-label, randomised, single dose cross-over Phase I trial, which assessed 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) 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.

The results showed that both formulations were well tolerated and no treatment emergent serious adverse events were reported.

Ablynx CEO Dr Edwin Moses said: "We are very pleased to show bioequivalence between the liquid and lyophilised formulations of caplacizumab, which is a very important milestone in the development of the product.

"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."

In 2009, caplacizumab received orphan drug designation in the US and EU, and could be the first drug specifically approved to treat acquired TTP as an adjunct to plasma exchange and immunosuppressants.