AMT sees positive data from Ph I/II AMT-011 study

Dutch gene therapy firm Amsterdam Molecular Therapeutics BV has reported positive results from a Phase I/II clinical study of its lead product, AMT-011 (formerly referred to as AMT-010). The drug is being assessed as a treatment for patients with lipoprotein lipase deficiency.

The study results, which were announced at the annual meeting of the America Society of Gene Therapy in Seattle, showed that AMT-011 brought about a substantial reduction in fasting plasma triglyceride levels in conjunction with a fat-free diet. The primary endpoint of TG levels of less than 10 mmol/l, or a 40% reduction, was achieved by three patients, one from the low-dose group and two from the mid-dose arm.

Additionally, in two members of the mid-dose cohort, the drop in TG levels was accompanied by the expression of active LPL between 26 and 32 weeks after administration of initial treatment. The firm said this indicates that long-term benefits can be achieved via gene therapy.