AMT gets €4M innovation credit for muscular dystrophy trial

Funding

Amsterdam Molecular Therapeutics has said that it will receive an Innovation Credit of up to €4 million from the Dutch government to support the development of its 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 Jörn Aldag, Chief Executive Officer of AMT.  “This credit will allow us to prioritise the development of our gene therapy for this progressive and devastating disease.”

AMT recently reported it has successfully treated DMD in a preclinical model of the disease with its product AMT-080.  The proof of concept studies demonstrated the technology resulted in synthesis of a functional version of the muscle protein dystrophin in both the heart and skeletal muscles.  The 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.  

On October 8, 2009 the European Medicines Evaluation Agency granted Orphan Drug Designation to AMT-080 for the treatment of DMD.  

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 tranches linked to the achievement of specific milestones, and will fund 35 per cent of the total anticipated costs of the project during this period.

DMD is a severe disease characterised 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.  

AMT-080 is based on exon-skipping technology which results in the defective region of the gene being bypassed during expression of the protein, such that an incomplete but functional of dystrophin is formed.  Positive long-term therapeutic effects of this approach have been demonstrated in animals.

The Dutch Innovation Credit is a risk-carrying credit for 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 and it does not need to be repaid if the project fails. The programme has separate budgets for technical development projects and for clinical development projects.