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Amsterdam Molecular Therapeutics Provides Status Update on Glybera(R)

Tuesday, May 10, 2011 Drug News J E 4
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Company to Present Updated Glybera Biomarker Data at American Society of Gene and Cell Therapy Annual Meeting

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, 2011 - 4:40 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, 2011 - 4:40 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, 2011 - 4:40 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, 2011 - 4:40 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, 2011 - 4:40 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, 2011 - 4:40 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, 2011 - 6:15 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

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