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Amsterdam Molecular Therapeutics Provides Status Update on Glybera(R)

May 10, 2011

AMSTERDAM, May 10, 2011 /PRNewswire-FirstCall/ — Amsterdam Molecular
Therapeutics (Euronext: AMT), a leader in the field of human gene therapy,
provided today a status update on Glybera (alipogene tiparvovec), its gene
therapy product under development for the treatment of lipoprotein lipase
deficiency (LPLD).

Following the submission of the Day 180 questions at the end of the first
quarter to the European Medicines Agency (EMA) as part of Glybera’s Market
Authorisation Application (MAA), AMT continues to work diligently with the
EMA and is confident that a decision on the approval of Glybera remains on
track for mid-2011 as previously guided.

“Our interactions with the EMA continue to be positive and we are
encouraged as we hit each milestone in the path towards a regulatory
decision. If approved, Glybera would be the first gene therapy product to
reach the market in Europe. We will continue to work with the EMA so that the
review of our data remains on track as much as possible from our side and
look forward to a decision in mid-2011,” stated Jorn Aldag, CEO of AMT. “Our
data are key to supporting our MAA. Later this month we will present
additional data at ASGCT regarding improved chylomicron handling, which we
believe could be used in the future by physicians as a biomarker for Glybera
in LPLD patients.”

AMT scientists and collaborators will present data from Glybera and
several other gene therapy programs in a total of nine posters at the
American Society of Gene and Cell Therapy (ASGCT) 14th Annual Meeting to be
held in Seattle, USA, May 18-21. In particular, the company will provide an
update on previously announced initial results from a long-term clinical
study of Glybera that showed improved chylomicron metabolism could show
utility as a biomarker in LPLD patients.

Several posters will report advances made by AMT in the field of RNAi
delivery including additional data relating to Apoliprotein B silencing in
hypercholesterolemia and delivery of miRNA molecules into the brain for
development in Huntington’s disease.

Details and times of the poster presentations can be found below:

[123] Absence of Integration Hotspots in Mice after Intramuscular
Injection of AAV1-LPLS447X Authors: C. Kaeppel, S. Beattie, A. Nowrouzi, U.
Appelt, R. Kirsten, H. Glimm, J. Snapper, C. von Kalle, H. Petry, M. Schmidt
Date/Time: Thursday, May 19, 20114:40 pm

[133] Construction of (Multi) Monomeric Duplex Adeno-Associated Vectors
for Human Gene Therapy Authors: Jacek Lubelski, Yvet Noordman, Bas Bosma,

Larbi Afia, Harald Petry, Andrew Bakker Date/Time: Thursday, May 19, 2011 -
4:40 pm

[183] Improved Chylomicron Clearance as a Marker for Long-Term Efficacy
of Alipogene Tiparvovec (AAV1-LPLS447X Gene Therapy) in LPLD Patients
Authors: Jaap Twisk, Frederique Frisch, Stephen Greentree, Harald Petry,

Janneke de Wal, Diane Brisson, Claude Gagne, Andre C. Carpentier, Daniel
Gaudet Date/Time: Thursday, May 19, 20114:40 pm

[551] Evaluation of the X-Protein Truncate Expression Potential from the
WPRE Element in Alipogene Tiparvovec, an AAV-Based Gene Therapy Vector
Authors: Jacek Lubelski, Florence Salmon, Betty Au, Larbi Afia, Andrew
Bakker
, Harald Petry Date/Time: Friday, May 20, 20114:40 pm

[562] In Vivo Comparison of the Long-Term Efficacy of AAV Expressed ShRNA
and MiRNA Targeting Apolipoprotein B100 Authors: Piotr Maczuga, Annemart
Koornneef, Richard van Logtenstein, Florie Borel, Harald Petry, Sander van
Deventer
, Pavlina Konstantinova Date/Time: Friday, May 20, 20114:40 pm

[564] Apolipoprotein B Knockdown by AAV-Delivered ShRNA Lowers Plasma
Cholesterol in Mice Authors: Annemart Koornneef, Piotr Maczuga, Richard van
Logtenstein, Florie Borel, Bas Blits, Tita Ritsema, Sander van Deventer,

Harald Petry, Pavlina Konstantinova Date/Time: Friday, May 20, 20114:40 pm

[566] Multidrug Resistance Transporters and miRNAs Expression: Changes in
Hepatocellular Carcinoma Authors: Florie Borel, Harald Petry, Peter Jansen,

Sander van Deventer, Pavlina Konstan-tinova Date/Time: Friday, May 20, 2011 -
4:40 pm

[569] RNAi-Mediated Gene Therapy of Human Diseases Authors: Annemart
Koornneef, Piotr Maczuga, Florie Borel, Angelina Huseinovic, Peter Jansen,

Harald Petry, Pavlina Konstantinova Date/Time: Friday, May 20, 20114:40 pm

[847] Apolipoprotein B Knock-Down by AAV-Delivered sh/miRNA Lowers Plasma
Cholesterol in Mice Authors: Piotr Maczuga, Annemart Koornneef, Richard van
Logtenstein, Florie Borel, Bas Blits, Sander van Deventer, Harald Petry,
Pavlina Konstantinova Date/Time: Saturday, May 21, 20116:15 pm

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based therapies.
The company’s lead product Glybera(R), a gene therapy for lipoprotein lipase
deficiency (LPLD), is currently under review by the European Medicines Agency
(EMA). If approved, Glybera will be the first gene therapy product to be
marketed in Europe. AMT also has a product pipeline of several gene therapy
products in development for hemophilia B, Duchenne muscular dystrophy, acute
intermittent porphyria, and Parkinson’s disease. 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 http://www.amtbiopharma.com.

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