AMSTERDAM, The Netherlands, March 28, 2012 /PRNewswire/ --

Amsterdam Molecular Therapeutics (Euronext: AMT - News), a leader in the field of human gene therapy, announced today data demonstrating that one-time administration of the gene therapy Glybera (alipogene tiparvovec) is able to markedly improve chylomicron (fat particles in the blood) metabolism following consumption of a low fat meal. This results in a much reduced level of newly-formed chylomicrons in the bloodstream, which are considered to be the cause of the acute and recurring bouts of pancreatitis seen in lipoprotein lipase deficiency (LPLD) subjects. LPLD is a very rare inherited condition that is associated with increased levels of chylomicrons. These particles carry certain types of fat in the blood, which because they are not removed from the body can cause recurrent pancreatitis. Data were published online in the Journal of Clinical Endocrinology & Metabolism (JCEM, Mar 2012).

"These data show that Glybera has a profound 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," explained Dr. Andr Carpentier, Division of Endocrinology at the Universit de Sherbrooke, Quebec, Candada. "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."

"This publication 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 metabolize these particles in LPLD patients, Glybera treatment results in fewer pancreatitis attacks," stated Carlos Camozzi, Chief Medical Officer at AMT. "LPLD patients are under constant risk of these attacks and the associated excruciating pain."

Study Details

In an open label clinical trial (CT-AMT-011-02), 5 LPLD subjects in Quebec, Canada, were administered alipogene tiparvovec at a dose of 1 x 1012 genome copies per kg. Two weeks before and 14 weeks after administration, chylomicron metabolism, and plasma palmitate (fatty acid) and glycerol appearance rates were determined following ingestion of a low fat meal. 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. The postprandial peak chylomicron level and chylomicron AUC were greatly reduced (by 79% and 93%, 6- and 24 hours after the test meal, respectively). There were no significant changes in plasma fatty acid and glycerol appearance rates. Plasma glucose, insulin and C-peptide also did not change. The data was obtained from AMT's study in patients treated with Glybera in 2009.

About Glybera

AMT has developed Glybera as a treatment for patients with the genetic disorder lipoprotein lipase deficiency.

LPLD is an orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating and life threatening clinical complication of LPLD. Glybera has orphan drug status in the EU and US.

About Amsterdam Molecular Therapeutics

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Amsterdam Molecular Therapeutics Publishes Positive Data from Glybera® 14 Week Study

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