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Ablynx Announces That its vWF Nanobody® Programme Has Been Granted Orphan
Drug Designation and Provides a Business Update for the First Quarter 2009
REGULATED INFORMATION
GHENT, Belgium, 7 May 2009 - 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
Nanobody® programme targeting von Willebrand Factor (vWF), has been granted
orphan drug designation by both the U.S. Food and Drug Administration and
the European Commission for the treatment of thrombotic thrombocytopenic
purpura (TTP), a rare life-threatening blood disorder. Ablynx also provided
a business update, in compliance with the EU transparency directive,
summarising material events and Ablynx’s financial position for the first
quarter of 2009.
Q1 Highlights
· 127% increase in revenues
· Cash, cash equivalents and available-for-sale financial assets at €111.8
million
· Positive Phase Ib data supporting progression of ALX-0081 (vWF) into Phase
II
· vWF Nanobody® programme granted orphan drug designation
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.
Business update
Revenues increased for the first quarter by 127% to €7.5 million (2008: €3.3
million). Expenses increased by 46% to €11.5 million (2008: €7.9 million).
This resulted in a net loss for the first quarter of €3.0 million (2008:
€3.3 million). The Company’s cash, cash equivalents and available-for-sale
financial assets were €111.8 million (2008: €121.2 million) at 31 March
2009.
Prospects for 2009
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 6 months. Ablynx also
expects to obtain additional milestone payments from existing collaborations
before the end of the year.
- ends -
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 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 ALX-0141
ALX-0141, is a fully humanized Nanobody® that targets the Receptor Activator
for Nuclear Factor kappa B Ligand (RANKL), a molecule important in bone
metabolism. It is currently in preclinical development and Ablynx aims to
develop ALX-0141 for a range of indications including the prevention of
osteoporosis. Other indications include bone metastases in cancer and bone
erosion associated with rheumatoid arthritis.
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. Importantly the Nanobodies® which
naturally exist in llamas have a very high homology with humans. 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.
For more information, please contact:
For international media enquiries:
College Hill Life Sciences
Sue Charles, Justine Lamond,
Dr John McIntyre
t: +44 (0)20 7866 7857
e: ablynx@collegehill.com
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
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 this press 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.
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