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"We are delighted to receive this credit for DMD, as these awards areonly made to innovative projects with strong commercial prospects," said JornAldag, Chief Executive Officer of AMT. "This credit will allow us toprioritize the development of our gene therapy for this progressive anddevastating disease."
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Recently, AMT has reported it has successfully treated DMD in apreclinical model of the disease with its proprietary gene therapy productAMT-080. These proof of concept studies demonstrated that AMT's technologyresulted in functional dystrophin synthesis in both the heart and skeletalmuscles, which prevents muscular dystrophy . These data reinforce a previousstudy in which this gene therapy approach was shown to successfully restoredystrophin in diseased human muscle cells obtained from biopsies of DMDpatients. Together, these results establish a robust basis for AMT'stherapeutic approach to DMD.
On October 8, 2009 the European Medicines Evaluation Agency (EMEA)granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for thetreatment of DMD. This entitles AMT to ten years of market exclusivity totreat DMD in Europe following marketing approval for AMT-080, provided thatthis product candidate is the first such approved new drug with a majormedical benefit.
The credit, together with accrued interest, will become repayable inmid-2013, subject to the commercial success of the project. The credit ispayable in tranches linked to the achievement of specific milestones, andwill fund 35% of the total anticipated costs of the project during thisperiod.
About Duchenne Muscular Dystrophy
DMD is a severe disease characterized by progressive muscle degeneration.It affects young children, almost exclusively boys, and leads to progressiveparalysis and death in young adulthood. The disease is caused by mutations inthe dystrophin gene, as a result of which the production of functionaldystrophin protein, an important structural component within muscle tissue,is blocked. Currently, there is no treatment to prevent the fatal outcome ofthis disease. DMD affects one in 3,500 males, making it one of the mostprevalent of muscular dystrophies.
AMT is developing a gene therapy product for DMD based on 'exon skipping'technology which results in bypassing the genetic defect such that thefunctional protein can be formed again. Positive long-term therapeuticeffects of this approach have been demonstrated in animals.
About Amsterdam Molecular Therapeutics
AMT, founded in 1998 and based in Amsterdam, is a leader in thedevelopment of human gene based therapies. Using adeno-associated viral (AAV)vectors as the delivery vehicle of choice for therapeutic genes, the companyhas been able to design and validate what is probably the first stable andscalable AAV production platform. This safe and efficacious proprietaryplatform offers a unique manufacturing capability which can be applied to alarge number of rare (orphan) diseases that are caused by one faulty gene.Currently, AMT has a product pipeline with several AAV-based gene therapyproducts in LPL Deficiency, Hemophilia B, DMD, Acute Intermittent Porphyriaand Parkinson's Disease at different stages of research or development.
About SenterNovem and the Innovation Credit program (http://www.senternovem.nl/innovatiekrediet)
The Innovation Credit is a risk-carrying credit aimed at fundingdevelopment projects that have a strong commercial potential but also a hightechnical risk. The projects have to be focused at the development of newproducts, processes or services. The purpose of the Innovation Credit is toreduce the financial risk for entrepreneurs. The Innovation Credit does notneed to be repaid if the project fails. The Innovation Credit has separatebudgets for technical development projects and for clinical developmentprojects.
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
