AMT prepares for submission of Marketing Authorization Dossier for AMT-011 (Glybera®)
Amsterdam, The Netherlands – Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that it has concluded enrollment and treated the last patient in the pivotal trial for it’s lead product AMT-011 (Glybera®) for Hyperlipoproteinemia, a seriously debilitating and potentially lethal disease. After recent preparatory meetings with the rapporteur countries Germany and the United Kingdom of the European Medicines Agency (EMEA), AMT expects to file the Marketing Authorization Dossier with EMEA not later than the fourth quarter of 2008.
AMT has developed AMT-011 as a cure for patients with the rare genetic disorder Hyperlipoproteinemia (HPL) type I, also known as LPL-deficiency. These patients have extremely high fat levels in their blood resulting in recurrent and potentially lethal pancreatitis as well as an increased risk of cardiovascular complications and diabetes. Because of a defective gene HPL-patients do not produce an enzyme that normally breaks down fats in the blood. Currently, there is no effective treatment or cure for this seriously debilitating and potentially lethal disease.
AMT-011 is a gene therapy product designed to insert a healthy gene into the patient’s body to replace the defective gene. The product is administered by a series of injections into muscle tissue after which the body starts to make the missing enzyme again. The advantage of this therapy is its potential to cure a disease instead of just treating the symptoms. AMT expects to finish the ongoing clinical trial with AMT-011 according to plan.
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 production platform. As such, AMT’s proprietary platform holds tremendous promise for thousands of rare (orphan) diseases that are caused by one faulty gene. AMT currently has a product pipeline with seven products at different stages of development.
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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.