Amsterdam Molecular Therapeutics receives € 4 million Innovation Credit - Gilde Healthcare

Amsterdam Molecular Therapeutics receives € 4 million Innovation Credit

January 6, 2010

Amsterdam, The Netherlands – Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that it will receive an Innovation Credit of up to € 4 million from the Dutch government to support the development of AMT’s gene therapy treatment for Duchenne Muscular Dystrophy (DMD). The credit is granted by SenterNovem, an agency of the Dutch Ministry of Economic affairs.

“We are delighted to receive this credit for DMD, as these awards are only made to innovative projects with strong commercial prospects,”

said Jörn Aldag, Chief Executive Officer of AMT.

“This credit will allow us to prioritize the development of our gene therapy for this progressive and devastating disease.”

Recently, AMT has reported it has successfully treated DMD in a preclinical model of the disease with its proprietary gene therapy product AMT-080. These proof of concept studies demonstrated that AMT’s technology resulted in functional dystrophin synthesis in both the heart and skeletal muscles, which prevents muscular dystrophy . These data reinforce a previous study in which this gene therapy approach was shown to successfully restore dystrophin in diseased human muscle cells obtained from biopsies of DMD patients. Together, these results establish a robust basis for AMT’s therapeutic 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 the treatment of DMD. This entitles AMT to ten years of market exclusivity to treat DMD in Europe following marketing approval for AMT-080, provided that this product candidate is the first such approved new drug with a major medical benefit.

The credit, together with accrued interest, will become repayable in mid-2013, subject to the commercial success of the project. The credit is payable in tranches linked to the achievement of specific milestones, and will fund 35% of the total anticipated costs of the project during this period.

About Duchenne Muscular Dystrophy
DMD is a severe disease characterized by progressive muscle degeneration. It affects young children, almost exclusively boys, and leads to progressive paralysis and death in young adulthood. The disease is caused by mutations in the dystrophin gene, as a result of which the production of functional dystrophin protein, an important structural component within muscle tissue, is blocked. Currently, there is no treatment to prevent the fatal outcome of this disease. DMD affects one in 3,500 males, making it one of the most prevalent 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 the functional protein can be formed again. Positive long-term therapeutic effects of this approach have been demonstrated in animals.

About Amsterdam Molecular Therapeutics
AMT, founded in 1998 and based in Amsterdam, is a leader in the development of human gene based therapies. 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. This safe and efficacious proprietary platform offers a unique manufacturing capability which can be applied to a large number of rare (orphan) diseases that are caused by one faulty gene. Currently, AMT has a product pipeline with several AAV-based gene therapy products in LPL Deficiency, Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson’s Disease at different stages of research or development.

About SenterNovem and the Innovation Credit program (
The Innovation Credit is a risk-carrying credit aimed at funding development projects that have a strong commercial potential but also a high technical risk. The projects have to be focused at the development of new products, processes or services. The purpose of the Innovation Credit is to reduce the financial risk for entrepreneurs. The Innovation Credit does not need to be repaid if the project fails. The Innovation Credit has separate budgets for technical development projects and for clinical development projects.

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