The News

Ablynx inititates Phase II clinical trial for TTP

30/09/2010

GHENT, Belgium, 30 September 2010 - Ablynx [Euronext Brussels: ABLX] today announced that it has opened recruitment in its first clinical trial centre for an open-label, randomised Phase II study for patients with acquired thrombotic thrombocytopenic purpura (TTP). This Phase II study will be conducted in Europe and North America and it is planned to enroll over 100 patients. Ablynx’s anti-von Willebrand factor (vWF) Nanobody® was granted orphan drug designation for the treatment of TTP in May 2009, by both the U.S. Food and Drug Administration and the European Commission.

During the Phase II study, the Nanobody will be administered to TTP patients as an adjunct to standard plasma exchange therapy. The primary endpoint of the study will be the time to response, defined by the normalisation of platelet count and the blood marker lactate dehydrogenase (LDH). The study goal is to see a significant reduction in the time to response for the anti-vWF treated population compared with placebo treatment. It is anticipated that this could translate into a shorter duration of life-threatening episode, a reduction of the number of plasma exchanges required and the amount of plasma product administered.

Dr Edwin Moses, CEO and Chairman of Ablynx commented: “There is currently no drug treatment approved specifically for TTP. Patients are often hospitalised for up to two weeks and have to receive frequent plasma exchanges which are costly and can lead to serious side effects such as allergic reactions or infections. Despite the plasma exchange treatment, the condition is very serious with up to 20% mortality. We believe that our anti-vWF Nanobody may reduce the number of days a TTP patient is hospitalised for plasma exchange thereby reducing the overall number of plasma transfusions and increasing the quality of life for these patients.” He added: “This is the third Nanobody programme entering Phase II clinical trials.”

Coordinating investigator Professor Peyvandi from the IRCCS Maggiore Hospital Foundation in Milan added: “We are very excited that the centre of Dr Dierickx, at the University Hospitals Leuven in Belgium, is the first to be activated for recruitment in this study. This TTP study investigates a novel treatment approach that could have an important impact on the future treatment of our TTP patients.”

About ALX-0081 and ALX-0681
ALX-0081 and ALX-0681 are novel “first-in-class” therapeutic Nanobodies targeting von Willebrand factor ("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. ALX-0081 is a bivalent Nanobody with a molecular weight of 28,000 daltons, designed to selectively prevent unwanted thrombus formation in vessels under high shear conditions without interfering with desirable haemostasis and, as such, to minimise bleeding complications. The Phase II study in TTP patients is an open-label, randomised study in patients with acquired TTP. In addition to plasma exchange the patients will receive one intravenous bolus injection (ALX-0081)
followed by daily subcutaneous injections (ALX-0681), or plasma exchange plus placebo. The anti-vWF Nanobody was assigned orphan drug designation in May 2009 by EMEA and FDA and is currently being developed for TTP.

About Thrombotic Thrombocytopenic Purpura (TTP)
TTP is a rare disorder and it can lead to life threatening disease episodes, characterised by the extensive formation of small platelet clots. This leads to occlusion of small vessels throughout the body, particularly within blood vessels supplying the brain and the kidneys. Other key characteristics of the disease are fragmentation of red blood cells (haemolysis) and an abnormal reduction of the platelets (thrombocytopenia). The latter is caused by the consumption of these blood cells via platelet clot formation on the activated vWF clusters or strings. Approximately four cases of TTP per million inhabitants are diagnosed per year in Europe and the United States. There is currently no approved drug therapy for TTP and plasma exchange is the only available treatment for these patients today. Plasma exchange involves the removal of the patient’s plasma (the non-cellular component of blood) and its replacement by donor plasma. TTP remains a condition with extremely high morbidity and mortality, even with timely plasma exchange, and so there is still a significant unmet medical need for this disease.
 

About Ablynx [Euronext Brussels: ABLX]
Founded in 2001 in Ghent, Belgium, Ablynx is a biopharmaceutical company focused on the discovery and development of Nanobodies, a novel class of therapeutic proteins based on single-domain antibody fragments, for a range of serious and life-threatening human diseases. The Company currently has over 250 employees. Ablynx completed a successful IPO on Euronext Brussels [ABLX] on 7 November 2007 and raised €50 million through an SPO in March 2010.
Ablynx is developing a portfolio of Nanobody-based therapeutics in a number of major disease areas, including inflammation, thrombosis, oncology and Alzheimer’s disease. Ablynx now has over 25 programmes in its therapeutic pipeline including four Nanobodies in clinical development. So far, Nanobodies have been successfully generated against more than 220 different protein targets including several complex targets such as chemokines, GPCRs, ion channels and viruses, which are typically very difficult to address with conventional monoclonal antibodies. Efficacy data have been obtained in 35 in vivo models for Nanobodies against a range of different targets.
Ablynx has an extensive patent position in the field of Nanobodies for healthcare applications. It has exclusive and worldwide rights to more than 130 families of granted patents and pending patent applications, including the Hamers patents covering the basic structure, composition, preparation and uses of Nanobodies.
Ablynx has ongoing research collaborations and significant partnerships with several major pharmaceutical companies, including Boehringer Ingelheim, Merck Serono and Pfizer. Ablynx is building a diverse and broad portfolio of therapeutic Nanobodies through these collaborations as well as through its own internal discovery programmes.
The Company’s lead programme ALX-0081, an intravenously administered novel anti-thrombotic, entered a Phase II study in patients undergoing percutaneous coronary intervention (PCI) in September 2009. Ablynx demonstrated proof-of-concept by biomarker for ALX-0081 in December 2009. ALX-0681, a subcutaneous administration of the anti-von Willebrand factor (vWF) Nanobody and ALX-0081 entered into a Phase II study in TTP patients in September 2010.
In September 2009, Ablynx’s partner Pfizer entered a Phase II study in RA patients, with an anti-TNF-alpha Nanobody, ATN-103. ATN-103 is administered via subcutaneous injection every 4 and 8 weeks.
ALX-0061, an anti-IL6R Nanobody is in preclinical development for the treatment of autoimmune and inflammatory diseases. Ablynx is on track to initiate a Phase I/II single and multiple dose study in patients with active RA during the first quarter of 2011.
In December 2009, Ablynx initiated a double-blind, randomised, placebo-controlled Phase I study with ALX-0141, a Nanobody targeting Receptor Activator of Nuclear Factor kappa B Ligand (RANKL), in healthy post-menopausal women for which initial positive Phase I data were reported in September 2010. In February 2010, Ablynx
announced that it had reached its criteria for initiating the preclinical development of ALX-0651, a Nanobody against CXCR4, and will progress this programme towards the clinic. CXCR4 plays an important role in cell mobility, tumor growth and metastasis. In March 2010, Ablynx advanced an anti-RSV Nanobody, ALX-0171, into preclinical development. ALX-0171 will be developed for the treatment of respiratory syncytial virus (RSV) infections, delivered through inhalation and has the potential to be effective both in the prevention of infection as well as in treatment once infection has occurred.

Nanobody® is a registered trademark of Ablynx NV.

For more information, please contact Ablynx:
Dr Edwin Moses
Chairman and CEO
t: +32 (0)9 262 00 07
m: +44 (0)7771 954 193 / +32 (0)473 39 50 68
e: edwin.moses@ablynx.com

Eva-Lotta Allan
Chief Business Officer
t: +32 (0)9 262 00 75
m: +32 (0)475 78 36 21 / +44 (0)7990 570 900
e: eva-lotta.allan@ablynx.com

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