Amsterdam, The Netherlands – Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today during the Phacilitate Cell & Gene Therapy Forum new data showing that one treatment of its lead product Glybera® results in a long term significant and clinically important lowering of the incidence of acute pancreatitis in Lipoprotein Lipase Deficient patients. Recurrent acute pancreatitis is the most debilitating complication of Lipoprotein Lipase Deficiency (LPLD) and is associated with significant morbidity and mortality.
To date only one patient from the two clinical trials had an acute pancreatitis during the long-term follow-up, while seven episodes of pancreatitis would have been expected based on the observational studies in the same patients. This equals a lowering of the incidence of pancreatitis from 0.33 per patient per year to 0.05 for both trials. The data were obtained from two observational and two interventional clinical studies with 22 patients and follow up data from 1 to 3.5 years after the treatment. The studies result in a total of more than 38 patient years worth of data.
Other positive effects of Glybera are the disappearance or reduction of fat accumulations in the skin or retina. The data also confirm that the treatment is well-tolerated and safe.
AMT has developed Glybera as an effective treatment for patients with the rare genetic disorder LPLD. Because of a defective gene, LPLD patients do not produce an enzyme that normally breaks down a certain type of fat carrying particles in the blood. Glybera is a gene therapy, which restores the enzyme (lipoprotein lipase) activity required to process the fat carrying particles. LPLD patients have extremely high fat levels in their blood, resulting in recurrent and potentially lethal pancreatitis as well as an increased risk of cardiovascular complications and diabetes. Currently, there exists no effective treatment or cure for this serious disease.
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 seven products at different stages of development.
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