uniQure Announces Analysis of Six-Year Follow-up Data for Glybera(R)

--50% Fewer Pancreatitis Events, No Severe Pancreatitis Post One-time Treatment--

AMSTERDAM, The Netherlands, June 3, 2014 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq:QURE), a leader in human gene therapy, today announced the full analysis of the six-year follow-up data for patients treated once with Glybera® (alipogene tiparvovec), uniQure's gene therapy product for the treatment of lipoprotein lipase deficiency (LPLD). Dr. Daniel Gaudet, the leading investigator during the majority of the clinical studies performed with Glybera, presented the results at the 82nd Annual Congress of the European Atherosclerosis Society (EAS) held in Madrid, Spain. Glybera is approved in the EU as a treatment for LPLD, a potentially life-threatening, orphan metabolic disease that results in patients' inability to process fat in the blood after a meal. LPLD patients eligible for Glybera treatment suffer from reoccurring and often severe pancreatitis events leading to recurrent admissions to hospitals and intensive care units (ICUs).

The retrospective analysis of hospital records for 19 patients with LPLD compares an equal time period of up to six years before and after treatment with Glybera. The analysis demonstrates that after a single administration of Glybera, patients have experienced reductions in both the frequency and severity of pancreatitis, long-term clinical benefits that reduce the burden on healthcare resources.

Pre-treatment (up to 6 years) Post-treatment (up to 6 years)
n =19 Rate
Events Rate (events/year) Total hosp. days Events Total hosp. days
Documented pancreatitis 19 0.21 143 10 0.11 75
Abdominal pain consistent with pancreatitis 18 0.20 116 11 0.12 107

Specifically, the data show that these patients have:

  • a range of approximately 40-50% lower post-treatment documented pancreatitis events and abdominal pain events consistent with pancreatitis;
  • no severe pancreatitis up to six years post-treatment; and
  • an approximately 50% lower post-treatment hospitalization rate and number of days spent in the hospital for documented pancreatitis, including only one ICU stay that occurred following treatment.

Glybera was generally well tolerated, with most adverse events such as myalgia, muscular stiffness, and burning sensation from injection site reactions occurring around the time of administration. No long-term safety concerns have been identified.

"It is encouraging to see that Glybera has been associated with reduced incidence of severe pancreatitis in a significant portion of treated patients and with less time spent in intensive care units," said Daniel Gaudet, MD, Ph.D., Associate Professor of Medicine, Université de Montréal and primary investigator. "Glybera is the first approved gene therapy in the western world and the way that this technology will be transferred to healthcare systems might influence the future of gene therapy for other diseases and have an important impact on the future of LPLD management."

The aim of the analysis was to measure incidence and severity of acute pancreatitis episodes and utilization of health resources (hospitalizations, ICU stays, days-in-hospital) over an extended period up to six years post-treatment. An independent adjudication board of three experts blinded to patient identification and pre- or post-treatment period information performed a retrospective review of individual hospital records and categorized each abdominal pain event that lead to hospitalization before and after treatment with Glybera. This six-year analysis represents an extension of the three-year retrospective review that formed a part of the data package upon which the European Commission approved Glybera in October 2012 under exceptional circumstances for the treatment of a subset of patients with LPLD. The data confirm the trend exhibited in the first case note review.

Glybera is indicated for the treatment of adult patients diagnosed with familial LPLD confirmed by genetic testing and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. LPLD results in hyper-chylomicronemia, or dramatic and potentially life-threatening increases in the level of large fat-carrying particles, called chylomicrons, in the blood after eating. In many cases, LPLD and the associated elevated levels of chylomicrons can cause acute and potentially life-threatening inflammation of the pancreas, known as pancreatitis, thus leading to frequent hospitalizations. Recurrent pancreatitis can lead to chronic abdominal pain, pancreatic insufficiency, which is an inability to properly digest food due to a lack of digestive enzymes made by the pancreas, and diabetes. There is no other approved treatment for LPLD.

About uniQure

uniQure is delivering on the promise of gene therapy through single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease-modifying therapies to patients with severe disorders. We are engaged in multiple partnerships and have obtained regulatory approval of our lead product, Glybera, in the European Union for a subset of patients with LPLD. www.uniQure.com


This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding the risk of cessation or delay of any of the ongoing or planned clinical studies and/or development of our product candidates, the risk of delay or failure to successfully commercialize or obtain further regulatory approval of Glybera, and the risk that our collaborations with Chiesi or our other collaboration partners will not continue or will not be successful. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's Form 20-F and its prospectus dated February 5, 2014, both filed with the Securities and Exchange Commission. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

Aicha Diba
Investor Relations
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Source: uniQure