Ablynx expands its anti-thrombotic portfolio and announces initiation of a phase I study for ALX-0681


Ablynx [Euronext Brussels: ABLX], a pioneer in the discovery and development of Nanobodies®, a novel class of antibody-derived therapeutic proteins, announced today that it has initiated a double-blind, randomized, placebo-controlled Phase I study in healthy volunteers with ALX-0681. The subjects will receive a subcutaneous injection of the novel anti-thrombotic Nanobody® that selectively targets von Willebrand (vWF) factor.

The Phase I study will investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and repeated subcutaneous administrations of ALX-0681. Ablynx will recruit up to 36 healthy volunteers for this study.

It is anticipated that subcutaneous administration of ALX-0681 will provide access to additional patient populations suffering from unwanted blood-clot formation, such as acute coronary syndrome (ACS), that are not currently addressed by the intravenous administration of ALX-0081. In addition, ALX-0681 is also being developed for the treatment of patients with thrombotic thrombocytopenic purpura (TTP).

“Ablynx is committed to developing its own internal pipeline of Nanobody®-based drugs and, with the positive progress to date of our lead product, ALX-0081, which also selectively targets von Willebrand factor, we are delighted to have now advanced a second product from our platform, ALX-0681, into a healthy volunteer Phase I study,” commented Dr Edwin Moses, Chairman and Chief Executive Officer.

About ALX-0681 and ALX-0081
ALX-0681 and ALX-0081 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-0681, a new potential anti-thrombotic agent, 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 minimize bleeding complications.

About the Thrombosis Market
Ablynx believes that ALX-0681 and ALX-0081 target a key opportunity in the anti-thrombotic market as they may provide a solution to the cardiologist’s current dilemma in acute coronary syndrome (ACS) which typically involves achieving a balance between the prevention of unwanted blood clots and potentially life-threatening bleeding complications. ALX-0081 and ALX-0681 could potentially prevent arterial thrombosis following angioplasty, which is a serious clinical problem. Other potential indications for ALX-0081 and ALX-0681 include thrombotic thrombocytopenic purpura (TTP), myocardial infarction (MI) and stroke.

About Acute Coronary Syndrome (ACS)
ACS is expected to afflict approximately 2.9 million people in the Unites States, Japan and certain European countries in 2009 according to Datamonitor’s Pipeline Insight: Antithrombotics, Reaching the untreated prophylaxis market report, DMHC2284 March 2007, and is the leading cause of mortality in the area of cardiovascular disease. Experts believe that the prevalence and incidence of acute infarcts due to arteriosclerosis will increase further, due to the ageing population. Peripheral artery occlusive disease (PAOD) will affect an estimated 22.1 million individuals in the US, Japan and certain European countries in 2009 and is associated with significant morbidity and mortality.

About Percutaneous Coronary Intervention (PCI)
The term percutaneous coronary intervention (sometimes called PTCA, angioplasty or stenting) describes a range of procedures that treat narrowing or blockages in coronary arteries supplying blood to the heart. Many patients undergoing this procedure will have previously had cardiac catheterisation (sometimes called coronary angiography) to examine the condition of the coronary vessels. Alternatively, percutaneous coronary intervention may be undertaken immediately after the diagnostic angiogram. Most patients with angina can be helped substantially by coronary stenting. For some patients with very mild disease stents are not required and medication is sufficient. For a small number of people bypass surgery is necessary. Almost all stent procedures are successful and completed in < 2 hours. Inevitably however there are risks and it is important that patients understand these risks before accepting treatment. Source: http://www.thecardiologist.co.uk/coronary.htm

About Thrombotic Thrombocytopenic Purpura (TTP)
TTP is a disease related to the formation of white clots. The underlying abnormality in TTP is the formation of small platelet clots, which leads to occlusions of small vessels throughout the body particularly within blood vessels supplying the brain and the kidneys. It has been shown that these small platelet clots are caused by the presence of large clusters or strings of activated vWF. Approximately four cases of TTP per million inhabitants are diagnosed per year in Europe and the United States. This incidence estimate suggests that orphan drug designation should be achievable for this indication, which would enable an accelerated development and approval timetable. There is currently no approved drug therapy for TPP 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] - http://www.ablynx.com
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 200 employees. Ablynx completed a successful IPO on Euronext Brussels [ABLX] on 7 November 2007.

Ablynx is developing a portfolio of Nanobody®-based therapeutic programmes in a number of major disease areas, including inflammation, thrombosis, oncology and Alzheimer’s disease. Nanobodies® have been generated against more than 100 different disease targets. Efficacy data has been obtained in over 25 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 50 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, Novartis and Wyeth Pharmaceuticals. 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 is in a multi-dose Phase Ib study in patients undergoing PCI and ALX-0681, also an anti-thrombotic but with a subcutaneous route of administration has started Phase I in healthy volunteers. Ablynx has progressed ALX-0141, an anti-RANKL Nanobody® for bone disorders into preclinical development. In addition, it is anticipated that an anti-TNF alpha Nanobody® partnered with Wyeth Pharmaceuticals will progress into the clinic during 2009.

Nanobody® is a registered trademark of Ablynx NV.

For more information, please contact:
College Hill Life Sciences – for UK/International media enquiries:
Sue Charles, Justine Lamond, John McIntyre
t: +44 (0)20 7866 7857
f: +44 (0)20 7866 7900
e: ablynx@collegehill.com

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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