Amsterdam Molecular Therapeutics’ Glybera(R) Significantly Reduces the Risk of Pancreatitis in LPLD Patients

By Amsterdam Molecular Therapeutics B.v, PRNE
Tuesday, June 28, 2011

AMSTERDAM, June 29, 2011 -

Data Presented at the
thEuropean Atherosclerosis
Society Meeting

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in
the field of human gene therapy, today released data showing that
its gene therapy Glybera® (alipogene tiparvovec) significantly
reduces the risk of pancreatitis in patients with Lipoprotein
Lipase Deficiency (LPLD). Pancreatitis, or inflammation of the
pancreas, is a major clinical symptom of LPLD. It causes severe
abdominal pain and often leads to hospitalization of patients as
well as other complications such as diabetes and early
atherosclerosis. Data were presented at the 79th
European Atherosclerosis Society Meeting (26-29 June 2011,
Gothenburg, Sweden).

“These data demonstrate the dramatic impact Glybera can have in
the lives of LPLD patients. Pancreatitis is a debilitating and
extremely painful condition. Many patients end up in the hospital
emergency room and try to control the pain by any means,” explained
Janneke de Wal, Director of Global Sales & Marketing at AMT.
“By reducing the incidence of pancreatitis episodes, Glybera has
the potential to help “normalize” the day to day lives of patients
affected by this disease and prevent the often frequent trips to

Study Details

Historical data on hospital presentations due to abdominal pain
from 17 patients already treated with Glybera were collected in
this case control study. Blinded event assessment was done by an
expert adjudication committee using a modified Atlanta Diagnostic
Criteria for acute pancreatitis to allow for incomplete historical
data. A statistically significant (p=0.0434) reduction in the risk
of acute pancreatitis was seen when the period from the first
pancreatitis event to administration of Glybera was compared with
the post-therapy period (median = 2.9 years). The hazard ratio, a
measure of how often an event occurs over time, indicated a 63%
reduction in risk of acute pancreatitis after administration of
Glybera (95% CI 0.142-0.971).

About Glybera®

AMT has developed Glybera as a treatment for patients with the
genetic disorder lipoprotein lipase deficiency. The dossier for
Glybera that was submitted to the European Medicines Agency is in
the process of being submitted for re-evaluation. LPLD is an orphan
disease for which no treatment exists today. The disease is caused
by mutations in the LPL gene, resulting in highly decreased or
absent activity of LPL protein in patients. This protein is needed
in order to break down large fat-carrying particles that circulate
in the blood after each meal. When such particles, called
chylomicrons, accumulate in the blood, they may obstruct small
blood vessels. Excess chylomicrons result in recurrent and severe
acute inflammation of the pancreas, called pancreatitis, the most
debilitating complication of LPLD. Glybera® has orphan drug status
in the EU and US.

About Amsterdam Molecular

AMT is a world leader in the development of human gene
based therapies. In addition to Glybera, AMT has a product
pipeline of several gene therapy products in development for
hemophilia B, Duchenne muscular dystrophy, acute intermittent
porphyria, Parkinson’s disease and SanfilippoB. Using
adeno-associated viral (AAV) derived vectors as the delivery
vehicle of choice for therapeutic genes, the company has been able
to design and validate probably the world’s first stable and
scalable AAV manufacturing platform. This proprietary platform
can be applied to a large number of rare (orphan) diseases
caused by one faulty gene and allows AMT to pursue its strategy of
focusing on this sector of the industry.  AMT was founded in
1998 and is based in Amsterdam. Further information can be found at

Certain statements in this press
release are “forward-looking statements” including those that refer
to management’s plans and expectations for future operations,
prospects and financial condition. Words such as “strategy,”
“expects,” “plans,” “anticipates,” “believes,” “will,” “continues,”
“estimates,” “intends,” “projects,” “goals,” “targets” and other
words of similar meaning are intended to identify such
forward-looking statements. Such statements are based on the
current expectations of the management of AMT only. Undue reliance
should not be placed on these statements because, by their nature,
they are subject to known and unknown risks and can be affected by
factors that are beyond the control of AMT. Actual results could
differ materially from current expectations due to a number of
factors and uncertainties affecting AMT’s business. AMT expressly
disclaims any intent or obligation to update any forward-looking
statements herein except as required by law. 


Further enquiries: Jörn Aldag, CEO, AMT, Tel : +31-20-566-7394, j.aldag at; Mike Sinclair, Partner, Halsin Partners, Tel : +44-20-7318-2955, msinclair at

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