Ablynx Reports Positive Phase Ib Results for its Anti-Thrombotic Nanobody ALX-0081
GHENT, Belgium, December 12, 2008 - Ablynx [Euronext Brussels: ABLX], a pioneer in the discovery and development of Nanobodies®, a novel class of antibody-derived therapeutic proteins, announced today that its anti-thrombotic ALX-0081, has reached the primary endpoint of the current Phase Ib study, demonstrating the desired pharmacological effect.
The objectives of the double-blind, randomized, placebo-controlled multiple dose Phase Ib study were to determine safety and tolerance when adding ALX-0081 via intravenous administration to a standard anti-thrombotic regimen in patients undergoing percutaneous coronary intervention (PCI) and to determine biological efficacy using a biomarker. The study involved a total of 25 patients and ALX-0081 administration was shown to be safe and well tolerated.
ALX-0081 is a novel “first-in-class” therapeutic Nanobody® targeting von Willebrand Factor. It is being developed to reduce the risk of thrombosis in patients with acute coronary syndrome (ACS). These positive Phase Ib results will now support the progress into Phase II clinical development in 2009.
Edwin Moses, CEO and Chairman said: “We are extremely pleased with these positive results in patients and the first demonstration of the clinical potential of ALX-0081. Impressively, ALX-0081 has been progressed from discovery to completion of this Phase Ib study in only four years, illustrating the speed at which our discovery platform can generate a therapeutic product. Based on these positive data, we look forward to initiating our discussions with the regulatory authorities and embarking on a Phase II study in 2009.”
Earlier this week Ablynx announced the initiation of Phase I trials for two additional Nanobodies®. Firstly, ALX-0681, which also targets von Willebrand Factor and is administered subcutaneously, entered a healthy volunteer 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 Ablynx’s anti-thrombotic ALX-0081. In addition, ALX-0681 is also being developed for the treatment of patients with thrombotic thrombocytopenic purpura (TTP).
The second healthy volunteer study initiated this week involved a Nanobody® targeting tumour necrosis factor alpha, partnered with Wyeth Pharmaceuticals.
“We are delighted to see three Nanobodies® in the clinic and look forward to further progress in clinical development,” added Dr Moses.
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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-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 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.
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]
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 has reached its primary endpoint 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, Ablynx’s partner Wyeth Pharmaceuticals has initiated a Phase I study in December 2008 for an anti-TNF alpha Nanobody®.
Nanobody® is a registered trademark of Ablynx NV.
Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company’s or, as appropriate, the Company’s directors’ current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its or their parent or subsidiary undertakings or any such person’s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in thispress release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release
Posted: December 2008