( e). The study wasstarted in August 200...)AMT Presents Positive Results of Phase I/II Study with Lead Product,at Annual Meeting of American Society of Gene Therapy,Health

Efficacy

The primary endpoints of the study were either median fasting plasma triglyceride (TG) levels after administration of AMT-010 equal to or less than 10 mmol/L, or a 40% reduction in median fasting plasma TG after administration on top of a fat-free diet. For all subjects a substantial reduction of median TG levels was observed. Three patients (one from the low dose and 2 from the mid dose group) hit the primary endpoint, with TG levels below the target level of 10 mmol/L or a 40% reduction in TG levels. In two patients of the mid dose group the reduction of TG coincided with expression of active LPL in the injected muscle at 26-32 weeks after vector administration, which clearly demonstrates long-term expression of the therapeutic gene.

Safety

Furthermore, the study shows that AMT-010 was well-tolerated; there were no drug-related severe adverse events, and no dose-limiting toxicity. There was no measurable sign of local or systemic inflammation and no increase in creatinine phosphokinase concentrations, which implies that the impact on tissue function is limited and not harmful to humans.

About AMT

Amsterdam Molecular Therapeutics BV (AMT) is a gene therapy company founded by scientists of the University of Amsterdam Medical Center (AMC) in 1998. AMT focuses on the development of gene-based therapies for orphan diseases. AMT's technology allows long-term gene expression and the specific delivery of therapeutic genes to target organs or tissues. AMT has optimized and validated the production of AAV-based gene therapy vectors. The result is a unique, stable, and scalable GMP production platform. Its lead product, AMT-011, is in pre-registration trials for the first indication: treatment of comp
'"/>