"We are delighted to receive this credit for DMD, as these awards are
only made to innovative projects with strong commercial prospects," said
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.
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
About SenterNovem and the Innovation Credit program ( http://www.senternovem.nl/innovatiekrediet)
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.
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
SOURCE Amsterdam Molecular Therapeutics B.V