The results of the double-blind, placebo controlled study in 40 healthy male volunteers show that ALX-0081 was safe and well tolerated at all doses tested, with no dose limiting toxicities or serious adverse events
GHENT, Belgium | December 17, 2007 | Ablynx [Euronext Brussels: ABLX], a pioneer in the discovery and development of Nanobodies®, a novel class of antibody-derived therapeutic proteins, today reported the final, positive results from a Phase I study of its lead development programme, ALX-0081. The results of the double-blind, placebo controlled study in 40 healthy male volunteers show that ALX-0081, an anti-thrombotic therapeutic, was safe and well tolerated at all doses tested, with no dose limiting toxicities or serious adverse events.
ALX-0081, generated through Ablynx’s in-house discovery platform, is a novel “first-in-class” therapeutic Nanobody® targeting von Willebrand Factor (anti-vWF). It is being developed to reduce the risk of thrombosis in patients with acute coronary syndrome (ACS) and thrombotic thrombocytopenic purpura (TTP). Following these positive Phase I results, Ablynx will now progress ALX-0081 to a multi-dose study in 2008.
In the study, treatment with the Nanobody® did not result in detectable immunogenicity. The study suggests that ALX-0081 adopts at least the plasma half-life of the target, von Willebrand Factor. The expected anti-thrombotic activity was shown with a biomarker in all volunteers receiving at least 2 mg of ALX-0081, indicating the high potency of the drug. ALX-0081’s pharmacological activity, based on a single injection, started at the lowest dose of 2 mg and reached a maximum duration of 12 hours at a dose of 12 mg.
Edwin Moses, CEO and Chairman said: “We are extremely pleased with these positive safety results and demonstration of the high potential potency of ALX-0081, our first Nanobody® in clinical development. In addition, ALX-0081 has been progressed from discovery to completion of Phase I in just over three years, demonstrating the speed at which our discovery platform can generate a novel therapeutic. Based on these positive data, we are looking forward to initiating our discussions with the regulatory authorities this year and embarking on our next clinical study in 2008 in order to progress programmes in acute coronary syndrome and TTP.”
About ALX-0081
ALX-0081 is a Nanobody® targeted against 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 work only in the late stage of platelet aggregation. The addition of ALX-0081 to the range of tools currently employed in an anti-thrombotic regimen may, therefore, have the potential to improve the overall efficacy of anti-platelet aggregation therapy and lead to an increased use of anti-thrombotic treatment in clinical indications like ACS where there is a high need for immediate and safe therapy.
Through its novel, highly selective mode of action, ALX-0081 is intended to prevent arterial thrombosis, without interfering with the desired haemostatis (wound healing) in the patient which results in less bleeding complications. Pre-clinical in vivo studies confirmed Ablynx’s belief that ALX-0081 has unique potential to set a new standard in anti-thrombotic therapy based on its immediate onset of action, its high potency and significantly improved safety compared to the currently marketed therapies in the form of significantly reduced bleeding complications.
About ACS and TTP
Acute coronary syndrome (ACS) is expected to afflict approximately 2.8 million people in the Unites States, Japan and certain European countries in 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 further increase, due to the ageing population. Peripheral arterial occlusive disease (PAOD) will affect an estimated 21.7 million individuals in the US, Japan and certain European countries in 2007 and is associated with significant morbidity and mortality.
Another disease related to the formation of white clots is thrombotic thrombocytopenic purpura (TTP). 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 clumps of von Willebrand Factor (vWF). 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 TPP and plasma exchange is the only available treatment for these patients today. Plasma exchange involves the removal of the patient’s plasma 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.
Besides ACS and TTP, another relevant indication for ALX-0081 is ischemic stroke. After heart disease and cancer, stroke is the third most frequent cause of death in the Western world. It is estimated that over two million people suffer a stroke annually. Mortality is high with 20% of patients dying, whilst the majority of the remainder is left permanently disabled.
About Ablynx
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 140 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 16 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 fifty 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, Wyeth Pharmaceuticals, Novartis, and P&G Pharma. Ablynx is building a diverse and broad portfolio of therapeutic Nanobodies® through these collaborations as well as through its own internal discovery programmes.
Ablynx announced final Phase I data from its first programme, an anti-thrombotic (ALX-0081) in December 2007 and another programme, which is partnered, is in advanced preclinical development.
Nanobody® is a registered trademark of Ablynx NV.
SOURCE: ABLYNX
Post Views: 140
The results of the double-blind, placebo controlled study in 40 healthy male volunteers show that ALX-0081 was safe and well tolerated at all doses tested, with no dose limiting toxicities or serious adverse events
GHENT, Belgium | December 17, 2007 | Ablynx [Euronext Brussels: ABLX], a pioneer in the discovery and development of Nanobodies®, a novel class of antibody-derived therapeutic proteins, today reported the final, positive results from a Phase I study of its lead development programme, ALX-0081. The results of the double-blind, placebo controlled study in 40 healthy male volunteers show that ALX-0081, an anti-thrombotic therapeutic, was safe and well tolerated at all doses tested, with no dose limiting toxicities or serious adverse events.
ALX-0081, generated through Ablynx’s in-house discovery platform, is a novel “first-in-class” therapeutic Nanobody® targeting von Willebrand Factor (anti-vWF). It is being developed to reduce the risk of thrombosis in patients with acute coronary syndrome (ACS) and thrombotic thrombocytopenic purpura (TTP). Following these positive Phase I results, Ablynx will now progress ALX-0081 to a multi-dose study in 2008.
In the study, treatment with the Nanobody® did not result in detectable immunogenicity. The study suggests that ALX-0081 adopts at least the plasma half-life of the target, von Willebrand Factor. The expected anti-thrombotic activity was shown with a biomarker in all volunteers receiving at least 2 mg of ALX-0081, indicating the high potency of the drug. ALX-0081’s pharmacological activity, based on a single injection, started at the lowest dose of 2 mg and reached a maximum duration of 12 hours at a dose of 12 mg.
Edwin Moses, CEO and Chairman said: “We are extremely pleased with these positive safety results and demonstration of the high potential potency of ALX-0081, our first Nanobody® in clinical development. In addition, ALX-0081 has been progressed from discovery to completion of Phase I in just over three years, demonstrating the speed at which our discovery platform can generate a novel therapeutic. Based on these positive data, we are looking forward to initiating our discussions with the regulatory authorities this year and embarking on our next clinical study in 2008 in order to progress programmes in acute coronary syndrome and TTP.”
About ALX-0081
ALX-0081 is a Nanobody® targeted against 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 work only in the late stage of platelet aggregation. The addition of ALX-0081 to the range of tools currently employed in an anti-thrombotic regimen may, therefore, have the potential to improve the overall efficacy of anti-platelet aggregation therapy and lead to an increased use of anti-thrombotic treatment in clinical indications like ACS where there is a high need for immediate and safe therapy.
Through its novel, highly selective mode of action, ALX-0081 is intended to prevent arterial thrombosis, without interfering with the desired haemostatis (wound healing) in the patient which results in less bleeding complications. Pre-clinical in vivo studies confirmed Ablynx’s belief that ALX-0081 has unique potential to set a new standard in anti-thrombotic therapy based on its immediate onset of action, its high potency and significantly improved safety compared to the currently marketed therapies in the form of significantly reduced bleeding complications.
About ACS and TTP
Acute coronary syndrome (ACS) is expected to afflict approximately 2.8 million people in the Unites States, Japan and certain European countries in 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 further increase, due to the ageing population. Peripheral arterial occlusive disease (PAOD) will affect an estimated 21.7 million individuals in the US, Japan and certain European countries in 2007 and is associated with significant morbidity and mortality.
Another disease related to the formation of white clots is thrombotic thrombocytopenic purpura (TTP). 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 clumps of von Willebrand Factor (vWF). 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 TPP and plasma exchange is the only available treatment for these patients today. Plasma exchange involves the removal of the patient’s plasma 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.
Besides ACS and TTP, another relevant indication for ALX-0081 is ischemic stroke. After heart disease and cancer, stroke is the third most frequent cause of death in the Western world. It is estimated that over two million people suffer a stroke annually. Mortality is high with 20% of patients dying, whilst the majority of the remainder is left permanently disabled.
About Ablynx
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 140 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 16 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 fifty 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, Wyeth Pharmaceuticals, Novartis, and P&G Pharma. Ablynx is building a diverse and broad portfolio of therapeutic Nanobodies® through these collaborations as well as through its own internal discovery programmes.
Ablynx announced final Phase I data from its first programme, an anti-thrombotic (ALX-0081) in December 2007 and another programme, which is partnered, is in advanced preclinical development.
Nanobody® is a registered trademark of Ablynx NV.
SOURCE: ABLYNX
Post Views: 140
