AMT, Amgen amend GDNF gene license agreement

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 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."