Amsterdam Molecular Therapeutics Amends Amgen GDNF Gene License Agreement

December 2, 2010

AMSTERDAM, December 2, 2010 /PRNewswire-FirstCall/ — Amsterdam Molecular
Therapeutics (AMT) Holding N.V. (Euronext: AMT), a leader in the development
of gene-based therapies, today announced that it has amended and restated its
licensing agreement with Amgen (Nasdaq: AMGN) for gene therapy applications
incorporating the GDNF (glial cell derived neurotrophic factor) gene, to
which Amgen holds rights. Financial terms were not disclosed.

The GDNF gene contains the information to produce a protein necessary for
the development and survival of nerve cells. The positive effect of GDNF on
nerve cells has already been demonstrated in early research. Studies with a
GDNF gene therapy, AMT-090, in a Parkinson’s disease model are being
conducted by AMT in collaboration with the University of Lund, Sweden. AMT
also plans to combine the GDNF gene with its proprietary adeno-associated
virus (AAV) technology to develop gene therapies for a range of CNS
applications, such as Huntington’s disease and amyotrophic lateral sclerosis
(ALS), with an aim to protect and enhance the function of the affected nerve

“Based on the promising early results of our GDNF gene therapy product in
Parkinson’s disease models, we believe there is an opportunity for a similar
approach in other debilitating CNS disorders. For many of these disorders,
current therapies are limited and tend only to treat symptoms. Treatment with
our gene therapies has the potential to halt or reverse disease progress,”
said Jorn Aldag, CEO of AMT. “This agreement will allow us to progress the
program for Parkinson’s Disease forward and at the same time find a partner
who will support the funding of our GDNF programs in alternative indications.
Expanding the partnering opportunities could mean even greater interest as
the widened therapeutic applications offer more chances of success,
potentially less complex product development paths and in many cases fewer
patients to enroll in clinical trials.”

About Amsterdam Molecular Therapeutics

AMT is a leader in the development of human gene based therapies. Using
adeno-associated viral (AAV) derived 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 proprietary platform 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 LPLD,
Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria, and
Parkinson’s Disease at different stages of research or development. AMT was
founded in 1998 and is based in Amsterdam.

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
AMT 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. AMT expressly disclaims any intent or
obligation to update any forward-looking statements herein except as required
by law.

SOURCE Amsterdam Molecular Therapeutics B.V

Source: newswire

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