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AMT has had two meetings with the Committee for Advanced TherapyMedicinal Products (CAT) at the EMA for clarification about the Day 120questions. These meetings have enabled AMT to finalise its strategy forresponding to these questions in a timely and effective manner.
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The outcome of the meetings suggests that AMT will not be required toconduct more clinical trials with additional new patients to be treated atthis time. The responses to the questions will be based in part on additionaldata and analyses from patients previously treated with Glybera(R). This willinclude new data available from the last clinical trial (CT-AMT-011-02) andits one year extension.
"We have developed a clear response strategy which, if executed with nounforeseen adverse events or delays, should allow us to remain on track foran EMA decision in the middle of 2011," noted Jorn Aldag, CEO of AmsterdamMolecular Therapeutics. "The route to registration for an innovative productsuch as Glybera(R) is not only very important for AMT, but it also gives hopeto thousands of patients suffering from rare diseases. Gene therapy carriesthe promise to be able to cure a range of diseases thought to be caused by asingle gene. AMT will exert every effort to succeed and reach the market withthis unique product."
About the Disease
LPLD is a seriously debilitating orphan disease for which no treatmentexists today. The disease is caused by mutations in the LPL gene, resultingin highly decreased or absent activity of LPL protein in patients. Thisprotein is needed in order to break down large fat-carrying particles thatcirculate in the blood after each meal. When such particles, calledchylomicrons, accumulate in the blood, they may obstruct small blood vessels.This can result not only in potentially lethal pancreatitis, but also indifficult-to-treat diabetes, and is associated with significant morbidity andmortality.
About Amsterdam Molecular Therapeutics
AMT is a leader in the development of human gene based therapies. Usingadeno-associated viral (AAV) derived vectors as the delivery vehicle ofchoice for therapeutic genes, the company has been able to design andvalidate what is probably the first stable and scalable AAV productionplatform. This proprietary platform can be applied to a large number of rare(orphan) diseases that are caused by one faulty gene. Currently, AMT has aproduct pipeline with several AAV-based gene therapy products in LPLD,Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria, and,Parkinson's Disease at different stages of research or development. AMT wasfounded in 1998 and is based in Amsterdam.
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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 futureoperations, prospects and financial condition. Words such as "strategy,""expects," "plans," "anticipates," "believes," "will," "continues,""estimates," "intends," "projects," "goals," "targets" and other words ofsimilar meaning are intended to identify such forward-looking statements.Such statements are based on the current expectations of the management ofAmsterdam Molecular Therapeutics only. Undue reliance should not be placed onthese statements because, by their nature, they are subject to known andunknown risks and can be affected by factors that are beyond the control ofAMT. Actual results could differ materially from current expectations due toa number of factors and uncertainties affecting AMT's business, including,but not limited to, the timely commencement and success of AMT's clinicaltrials and research endeavors, delays in receiving U.S. Food and DrugAdministration or other regulatory approvals (i.e. EMA, Health Canada),market acceptance of AMT's products, effectiveness of AMT's marketing andsales efforts, development of competing therapies and/or technologies, theterms of any future strategic alliances, the need for additional capital, theinability to obtain, or meet, conditions imposed for required governmentaland regulatory approvals and consents. AMT expressly disclaims any intent orobligation to update these forward-looking statements except as required bylaw. For a more detailed description of the risk factors and uncertaintiesaffecting AMT, refer to the prospectus of AMT's initial public offering onJune 20, 2007, and AMT's public announcements made from time to time.
SOURCE Amsterdam Molecular Therapeutics B.V
