Amsterdam Molecular Therapeutics Amends Amgen GDNF Gene License Agreement - Gilde Healthcare

Amsterdam Molecular Therapeutics Amends Amgen GDNF Gene License Agreement

December 2, 2010

Amsterdam, The Netherlands – 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 cells. 

“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 Jörn 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.

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