AMT Addresses Major Liver Disease

Armen Hareyan's picture
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Liver Disease

European Patent Office has granted AMT an important patent for the treatment of Non-Alcoholic Steatotic Hepatitis (NASH) with its lead product AMT-11. AMT has a similar patent pending with the U.S. Patent and Trademark Office.

The most common liver disorder in the Western world, non-alcoholic fatty liver disease (NAFLD), affects 20 to 40 percent of the general population. At least 11 percent of NAFLD patients will develop NASH, a very serious inflammatory liver disease for which there is no therapy and affects many millions in the developed world.

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AMT-011, NAFLD, NASH and the metabolic syndrome

AMT-011 is currently in its last phase of clinical development for the treatment of lipoprotein lipase (LPL) deficiency that is associated with very high circulating triglyceride (fat) levels. The product consists of an AAV-based vector that delivers the LPL gene to the muscle, leading to long-term expression of the therapeutic protein, lipoprotein lipase.

The patented invention is related to the observation that expression of AMT-011 in muscle causes a reduction of the liver triglyceride content by redistributing triglycerides from the liver to the peripheral muscles where it is metabolized. NAFLD and NASH are closely related to the pathogenesis of the "metabolic syndrome". This latter condition is characterized by central obesity, increase of serum triglycerides and insulin resistance, and is a major cause of diabetes and coronary vascular disease in the Western world. Recent epidemiological studies published in the journals Gastroenterology and Annals of Hepatology show that the prevalence of NAFLD in the general population is extremely high. Over 60 million adult Americans and an unknown proportion of children are believed to suffer from NAFLD. At the lowest range of the estimates, 11 percent of these patients develop NASH.

Ronald Lorijn, CEO of AMT, said, "We are very pleased that EPO granted this important patent to AMT. This substantially widens the potential use of our lead product AMT-011 beyond the current orphan indications to the large patient populations who suffer from fatty liver diseases, hypertriglyceridemia and insulin resistance. In the course of 2008, it is our intention to initiate a development track for AMT-011 in NASH to establish proof of concept in man."

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