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

June 29, 2011

AMSTERDAM, June 29, 2011 /PRNewswire/ –

              Data Presented at the 79th European Atherosclerosis Society

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field
of human gene therapy, today released data showing that its gene therapy
Glybera(R) (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(R)

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(R) has orphan drug status in the EU and US.

About Amsterdam Molecular Therapeutics

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.


SOURCE Amsterdam Molecular Therapeutics B.V

Source: newswire

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