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Last updated on April 18, 2014 at 1:21 EDT

AMT Receives Innovation Credit From Dutch Government for Duchenne Muscular Dystrophy

January 6, 2010

AMSTERDAM, January 6 /PRNewswire-FirstCall/ — 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 EUR 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 Jorn
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 (
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
June 20, 2007, and AMT’s public announcements made from time to time.

SOURCE Amsterdam Molecular Therapeutics B.V


Source: newswire