Ablynx, a pioneer in the discovery and development of Nanobodies, a novel class of antibody-derived therapeutic proteins, received US Food and Drug Administration (FDA) and the European Commission (EC) orphan drug status for its anti-thrombotic Nanobody programme targeting von Willebrand Factor (vWF) for the treatment of thrombotic thrombocytopenic purpura (TTP), a rare life-threatening blood disorder.
During the first quarter, Ablynx announced positive detailed results from the ALX-0081 phase-Ib study. The primary endpoint of this study, demonstrating the complete inhibition of the target protein as measured by a specific biomarker, was reached in December 2008. These positive data support the progression of ALX-0081 into phase-II testing, still on track to commence in Q3 2009. ALX-0681 is a subcutaneous form of Ablynx's lead anti-thrombotic programme, ALX-0081, and is designed to selectively prevent unwanted thrombus formation in blood vessels. ALX-0681 is being developed for TTP and, if it reaches market, it will benefit from a market exclusivity period of 7 years in the USA and 10 years in Europe, based on the granted orphan drug designation. Also during the first quarter, both Novartis and Wyeth Pharmaceuticals extended their research collaborations with Ablynx for a further 12 months.
"At present, there are no specific drug therapies available to patients with TTP. We are therefore extremely pleased that our vWF programme has been granted orphan drug designation both in the USA and Europe. This product has the potential to significantly improve the quality of life for patients with this life-threatening disease," commented Dr Edwin Moses, chief executive officer and chairman. "I am also delighted with the rapid and positive progress made with ALX-0081 and the fact that we are on track to have four Nanobodies in the clinic by the end of 2009 including the TNF-alpha Nanobody we licensed to Wyeth," added Dr Moses.
The company anticipates obtaining additional data from the open label extension of the ALX- 0081 phase-Ib study by the end of the second quarter and is on track to initiate phase-II development of ALX-0081 in the third quarter of this year. ALX-0681, the subcutaneous delivery form of ALX-0081, is progressing in a phase-I volunteer study and this study is expected to conclude later this year.
It is anticipated that Wyeth Pharmaceuticals will complete the initial phase-I study for the lead anti-TNF-alpha Nanobody in 2009. During 2009, Ablynx will further develop its own product pipeline and expects to advance two other Nanobodies into pre-clinical studies.
Additionally, ALX-0141, which targets Rankl and could be important in osteoporosis and some other bone related disorders, is on track to enter a phase-I study before the end of 2009. By the end of 2009, it is anticipated there will be four Nanobodies in clinical trials. The company is further developing its own technology platform and exploiting the key advantages of Nanobodies in areas such as alternative routes of administration where additional data should be generated over the next six months. Ablynx also expects to obtain additional milestone payments from existing collaborations before the end of the year.
ALX-0681 and ALX-0081 are novel 'first-in-class' therapeutic Nanobodies targeting vWF, a protein found in the blood that acts at a very early stage in the coagulation cascade, namely platelet adhesion, in contrast to currently available anti-platelet drugs which act only in the late stage of platelet aggregation. ALX-0081 is administered intravenously while ALX-0681 is administered subcutaneously.