AMSTERDAM, The Netherlands — Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today new data showing that a one-time administration of its lead product GlyberaTM results in significant long-term health benefits.
Long-term follow-up data from two clinical trials show that one administration with GlyberaTM brings significant and clinically important reduction in 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. These data were presented at the International Symposium on Atherosclerosis in Boston, one on the most prestigious conferences on arterial disease well-attended by expert physicians.
The three year follow-up data for the eight patients from the first clinical trial show a statistically significant, tenfold decrease in the incidence of acute pancreatitis. From the second trial in fourteen patients one year data are available showing similar results. The data from both trials also confirm that the treatment is well-tolerated and safe.
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 complication of LPLD. In the second trial it was observed that the white colouring of blood vessels in het eyes of patients (lipemia retinalis) disappeared after administration of GlyberaTM showing that this gene therapy lowers the chylomicron levels in the blood. The disease can result in difficult-to-treat diabetes and is associated with significant morbidity and mortality.
About Amsterdam Molecular Therapeutics
AMT has a unique production platform that circumvents the obstacles that have hindered development of gene therapy technologies into drugs. Using adeno-associated viral vectors as the delivery vehicle of choice for therapeutic genes, the company has designed and validated a stable, scalable, commercially attractive and GMP-compliant AAV manufacturing 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 nine products at different stages of development.
Certain statements in this press release are “forward-looking statements” including those that refer to management’s plans and expectations for future operations, prospects and financial condition. Words such as “strategy,” “expects,” “plans,” “anticipates,” “believes,” “will,” “continues,” “estimates,” “intends,” “projects,” “goals,” “targets” and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of Amsterdam Molecular Therapeutics only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT’s business, including, but not limited to, the timely commencement and success of AMT’s clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health Canada), market acceptance of AMT’s products, effectiveness of AMT’s marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT’s initial public offering on June 20, 2007, and AMT’s public announcements made from time to time.