Amsterdam, The Netherlands – Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that positive interim data from its pivotal clinical trial with AMT’s lead product Glybera® (AMT-011) were presented by the Principal Investigator, professor Daniel Gaudet, at the American Society of Gene Therapy Annual Meeting in Boston on May 29th. These data confirm the outcome of a previous study conducted in The Netherlands, demonstrating safety and efficacy of Glybera® for lipoprotein-lipase deficiency, a disease also known as Hyperlipoproteinemia (HPL) type I. Currently, there is no effective treatment or cure for this seriously debilitating and potentially lethal disease.
Glybera® is a gene therapy product that corrects the genetic defect in lipoprotein-lipase (LPL) deficient patients. LPL is normally produced in muscle tissue and is needed to break down the large fat-carrying particles that circulate in the blood after each meal. As a result, LPL-deficient patients have very high fat (triglyceride) levels in their blood which may lead to recurrent episodes of pancreatitis, insulin resistance, and fat accumulations in skin as well as liver and retina.
In the pivotal study conducted in Quebec (Canada), patients with LPL-deficiency were first enrolled in an observation study and subsequently treated in a study in which there are two different dose groups. All patients showed very high fat levels in the observation study and had experienced (recurrent) pancreatitis episodes prior to treatment. To date 6 patients have been treated with the lower dose and 7 with the higher dose.
Professor Gaudet presented data on the first 10 patients, of which 6 were in the lower dose group and 4 were in the higher dose group. The data showed that treatment was well tolerated. Fat concentrations were reduced after treatment in all patients, with one exception. In all patients with fat accumulations in skin or retina, these accumulations disappeared or were reduced. All patients reported gain of energy. In the two patients that had diabetes, a reduction of insulin resistance was observed, leading to a reduction of their diabetic medication. One patient experienced a pancreatitis episode immediately after injection, when fat levels in the blood were still high. But to date no other episodes of pancreatitis have been observed in patients treated with Glybera® in this study.
Upon completion of the study the results will be part of the filing dossier for the marketing approval of Glybera®.
About Amsterdam Molecular Therapeutics
AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT’s proprietary platform holds tremendous promise for thousands of rare (orphan) diseases that are caused by one faulty gene. AMT currently has a product pipeline with six products at different stages of development.
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