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AMT has concluded two clinical studies for LPLD, in Europe and Canada,and long term follow-up from both of these is ongoing, as is a third clinicalstudy in Canada. In these three studies Glybera(R) has shown a sizeabledecrease in the incidence of pancreatitis, or acute inflammation of thepancreas, the most debilitating complication of LPLD. In addition, thesestudies indicate that Glybera(R) has an excellent safety profile.
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"The acceptance of the Glybera(R) dossier by EMA is a significant steptowards marketing approval for Glybera(R). Moreover, it demonstrates AMT'sdevelopment capability. A future approval of the MAA for Glybera would fullyvalidate our gene therapy approach and our adeno-associated viral (AAV)vector delivery platform. We believe this step offers hope to many patients,as gene therapy may become the therapeutic approach of choice for inheriteddisorders" said Jorn Aldag, Chief Executive Officer of AMT.
The EMA formal review will be conducted via the centralised procedure,which is the standard route for all advanced therapies. During 2010, AMTexpects to provide further updates on the results from its follow-up andongoing studies, in accordance with reporting regulations.
About the Disease
LPLD is a seriously debilitating, and potentially lethal, orphan diseasefor which no treatment exists today. The disease is caused by mutations inthe LPL gene, resulting in highly decreased or absent activity of LPL proteinin patients. This protein is needed in order to break down large fat-carryingparticles that circulate in the blood after each meal. When such particles,called chylomicrons, accumulate in the blood, they may obstruct small bloodvessels. This can result not only in pancreatitis, but also indifficult-to-treat diabetes, and is associated with significant morbidity andmortality.
About Amsterdam Molecular Therapeutics
AMT, founded in 1998 and based in Amsterdam, is a leader in thedevelopment of human gene based therapies. Using AAV as the delivery vehicleof choice for therapeutic genes, the company has been able to design andvalidate what is probably the first stable and scalable AAV productionplatform. This safe and efficacious proprietary platform offers a uniquemanufacturing capability which 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 LPLDeficiency, Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson'sDisease at different stages of research or development.
For information
AMT will be presenting at the BioCEO & Investor Conference,Waldorf-Astoria, New York City, at 9:30 am (EST) on Tuesday, February 9, 2010.
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. EMEA, 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
