uniQure and Chiesi Announce Six Year Follow-up Data from Glybera(r) Treated Patients

AMSTERDAM, Netherlands, March 17, 2014 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leader in human gene therapy, and Chiesi Farmaceutici S.p.A. ("Chiesi"), a leading international pharmaceutical company, today announced initial analysis of retrospectively collected individual patient data from LPLD patients treated with Glybera®. The analysis covers follow-up data for up to six years post treatment for 13 patients, all of whom met indication requirements for the current labeling of Glybera in the EU. This analysis represents an extension of the case note review which 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 lipoprotein lipase deficiency, or LPLD, a potentially life-threatening, orphan metabolic disease. Glybera currently is not approved for use outside of the European Union.

An external adjudication board of independent experts performed the review of the individual patient profiles. In the analysis equal time periods of up to six years before and after Glybera treatment were compared to evaluate the number and severity of attacks of pancreatitis in each LPLD patient with a history of severe or repeated pancreatitis. The review suggests that treatment with Glybera provides long-term beneficial effects with regard to the risk of encountering new pancreatitis events, including the occurrence of severe pancreatitis events. These results are in line with the trend exhibited in the first case note review performed up to 3 years after treatment with Glybera.

"Despite the small number of patients in the study, it is encouraging to note that this new single treatment paradigm continues to indicate long term and relevant clinical benefit in a patient population that has no other treatment option," said Daniel Gaudet, MD, Ph.D., Associate Professor of Medicine, Université de Montreal, who was the leading investigator during the majority of the clinical studies performed with Glybera.

John Kastelein, MD, Ph.D., Professor of Medicine and Chairman of the Department of Vascular Medicine at the Academic Medical Center of the University of Amsterdam, who was among the inventors of Glybera, highlighted how the trend of long term benefit combined with a very encouraging safety profile of Glybera differentiates the gene therapy approach: "I hope that this approach of treating patients with severe lipid disorders will lead to additional gene therapy initiatives in our field."

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 hyperchylomicronemia, 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.

About Chiesi Farmaceutici
Chiesi Farmaceutici is a research-focused international group, with more than 75 years of experience headquartered in Parma (Italy). Chiesi researches, develops, and commercializes innovative pharmaceutical solutions in the respiratory therapeutics and specialist medicine areas. In 2013, Chiesi achieved sales of over 1.2 billion Euros, constituting double digit growth over 2012. Its R&D centers in Parma (Italy), Paris (France), Rockville (USA), Chippenham (UK), and the R&D team of the newly-acquired Danish company Zymenex, integrate their efforts to advance Chiesi's pre-clinical, clinical, and registration programs. Chiesi Group employs approximately 3900 people, 480 of which are dedicated to R&D activities. For more information, please visit www.chiesi.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 statements regarding the final analysis of the data discussed and the potential longer-term effects of Glybera. 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 prospectus filed with the Securities and Exchange Commission dated February 5, 2014. 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.

uniQure: Media inquiries:
Aicha Diba Gretchen Schweitzer
Investor Relations MacDougall Biomedical Communications
Direct : +31 20 566 8014 Direct: +49 172 861 8540
Main: +31 20 566 7394 Main: +1 781 235 3060
a.diba@uniQure.com gschweitzer@macbiocom.com

Chiesi Farmaceutici:
Valentina Biagini
Group Communication Manager
Mobile: +39 348 7693 623


Source: uniQure