AMT receives funding from Dutch government for development of Duchenne Muscular Dystrophy therapy

Amsterdam Molecular Therapeutics, a human gene therapy specialist, will receive an Innovation Credit of up to Euro 4m from the Dutch government to support the development of its gene therapy treatment for Duchenne Muscular Dystrophy (DMD).

"This credit will allow us to prioritise the development of our gene therapy for this progressive and devastating disease," said Joern Aldag, chief executive of AMT.

AMT claims to have successfully treated DMD in a preclinical model 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 approach was shown to restore dystrophin in diseased human muscle cells obtained from biopsies of DMD patients.

The European Medicines Evaluation Agency (EMEA) granted orphan drug status to AMT-080 for the treatment of DMD on October 8, 2009. This entitles AMT to 10 years of market exclusivity to treat DMD in Europe following marketing approval, provided that this product candidate is the first such approved new drug with a major medical benefit.

The Innovation Credit, together with accrued interest, will become repayable in mid-2013, subject to the commercial success of the project. The credit is payable in stages linked to the achievement of milestones, and will fund 35% of the total anticipated costs of the project during this period.

The Innovation Credit funds development projects that have a strong commercial potential, but also a high technical risk. The projects have to be focused on the development of new products, processes or services. The purpose of the Innovation Credit is to reduce the financial risk for entrepreneurs.