Amsterdam Molecular Therapeutics (AMT) has announced the positive data from a Glybera 14 week study, demonstrating that one-time administration of the gene therapy Glybera (alipogene tiparvovec) is able to improve chylomicron metabolism following consumption of a low fat meal.
Glybera introduces a normal, healthy LPL gene into the body so that it can make functional lipoprotein lipase (LPL) protein.
In an open label CT-AMT-011-02 clinical study, five lipoprotein lipase deficiency (LPLD) subjects in Quebec, Canada, were administered alipogene tiparvovec at a dose of 1 x 1012 genome copies per kg.
Following ingestion of a low fat meal, chylomicron metabolism, plasma palmitate and glycerol appearance rates were determined at two weeks before and 14 weeks after administration of alipogene tiparvovec.
The study showed that following administration of alipogene tiparvovec, the triglyceride (TG) content of the chylomicron fraction and the chylomicron-triglyceride (TG)/total plasma TG ratio were reduced throughout the postprandial period.
AMT chief medical officer Carlos Camozzi said the study provides additional, independent support on the ability of Glybera to restore chylomicron metabolism in LPLD patients.
"We believe by restoring the body's ability to metabolise these particles in LPLD patients, Glybera treatment results in fewer pancreatitis attacks," Camozzi added.
Glybera has been evaluated in three interventional clinical studies which demonstrated that fat concentrations in blood were reduced after therapy in nearly all patients between three and 12 weeks after injection of Glybera.
Dr. André Carpentier, Division of Endocrinology at the Université de Sherbrooke, Quebec, Canada, said the data showed that Glybera has an impact on chylomicron metabolism 14 weeks after a single administration.
"Although the patient cohort is small, due to the rare nature of LPLD, these results are very encouraging. LPLD patients often suffer from extremely painful bouts of pancreatitis, which is believed to be caused by the accumulation of chylomicron particles in the blood," Carpentier added.