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AMT's Lead Product Poised to Address Major Liver Disease
Date:1/27/2008

AMSTERDAM, The Netherlands, January 28 /PRNewswire-FirstCall/ --

- European Patent Office Grants Patent for Treatment of Non-Alcoholic Steatotic Hepatitis

AMT (Euronext: AMT), a leader in the field of human gene therapy, today announced that the European Patent Office (EPO) has granted the company an important patent for the treatment of Non-Alcoholic Steatotic Hepatitis (NASH) with its lead product AMT-11. AMT has a similar patent pending with the U.S. Patent and Trademark Office.

The most common liver disorder in the Western world, non-alcoholic fatty liver disease (NAFLD), affects 20 to 40 percent of the general population. At least 11 percent of NAFLD patients will develop NASH, a very serious inflammatory liver disease for which there is no therapy and affects many millions in the developed world.

AMT-011, NAFLD, NASH and the metabolic syndrome

AMT-011 is currently in its last phase of clinical development for the treatment of lipoprotein lipase (LPL) deficiency that is associated with very high circulating triglyceride (fat) levels. The product consists of an AAV-based vector that delivers the LPL gene to the muscle, leading to long-term expression of the therapeutic protein, lipoprotein lipase.

The patented invention is related to the observation that expression of AMT-011 in muscle causes a reduction of the liver triglyceride content by redistributing triglycerides from the liver to the peripheral muscles where it is metabolized. NAFLD and NASH are closely related to the pathogenesis of the "metabolic syndrome". This latter condition is characterized by central obesity, increase of serum triglycerides and insulin resistance, and is a major cause of diabetes and coronary vascular disease in the Western world. Recent epidemiological studies published in the journals Gastroenterology and Annals of Hepatology show that the prevalence of NAFLD in the general population is extremely high. Over 60 million adult Americans and an unknown proportion of children are believed to suffer from NAFLD. At the lowest range of the estimates, 11 percent of these patients develop NASH.

Ronald Lorijn, CEO of AMT, said, "We are very pleased that EPO granted this important patent to AMT. This substantially widens the potential use of our lead product AMT-011 beyond the current orphan indications to the large patient populations who suffer from fatty liver diseases, hypertriglyceridemia and insulin resistance. In the course of 2008, it is our intention to initiate a development track for AMT-011 in NASH to establish proof of concept in man."

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV GMP production platform. As such, AMT's proprietary platform holds tremendous promise for thousands of (rare) diseases that are caused by one faulty gene. AMT currently has a product pipeline with six products at different stages of development.

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 Amsterdam Molecular Therapeutics 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, including, but not limited to, the timely commencement and success of AMT's clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health Canada), market acceptance of AMT's products, effectiveness of AMT's marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT's initial public offering on June 20, 2007, and AMT's public announcements made from time to time.


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SOURCE Amsterdam Molecular Therapeutics B.V
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