AMT presents long-term efficacy study of Glybera in treatment of lipoprotein lipase deficiency

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today results from a long-term efficacy study of Glybera (alipogene tiparvovec) that showed improved chylomicron metabolism could be used as a biomarker for increased lipoprotein lipase (LPL) activity in those patients missing the gene that produces this protein. Glybera is a gene therapy product under development for the treatment of lipoprotein lipase deficiency (LPLD) that is currently under review for marketing approval with the European Medicines Agency (EMA).

In patients with mutations in the LPL gene, dietary fat (triglyceride molecules) cannot be broken down and so cause chylomicrons, which carry triglycerides around the body, to accumulate in the blood. This may result in recurrent extremely painful and life-threatening episodes of pancreatitis. Data presented last night at the American Society of Gene and Cell Therapy 14th Annual Meeting in Seattle, USA, showed that breakdown of chylomicrons produced after meals was greatly and significantly improved at both 14 and 52 weeks following one-time Glybera administration.

"Lipoprotein lipase plays a central role in chylomicron metabolism so it follows that evidence of improved long-term clearance following Glybera administration could be taken as a measure of efficacy. It also helps to explain the decrease in pancreatitis episodes, the most debilitating portrayal of LPLD, seen in our clinical trials," explained Dr Harald Petry, Head of Research at AMT.

"These data are important as they support the biological activity of Glybera and provide a plausible mechanism of action as well as a measurable marker of effect. We have submitted this data on chylomicron handling to the EMA as part of our Marketing Authorisation Application. We believe a decision by the EMA will be made by mid-2011 as previously guided," noted Jorn Aldag, CEO of AMT.