uniQure Announces Dosing of First Patient in High-Dose Cohort in Phase I/II Hemophilia B Clinical Trial

AMSTERDAM, the Netherlands, March 14, 2016 -- uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced that the Company has treated the first patient in the second cohort of its ongoing AMT-060-01 Phase I/II trial in hemophilia B patients. The primary objective of the second cohort is to assess the safety of the systemic administration of a higher dose of uniQure's AMT-060 gene therapy in hemophilia B patients that present with a severe or moderately-severe disease phenotype. Secondary objectives of the trial include evaluation of Factor IX (FIX) activity levels as well as evaluation of annualized bleeding rates and recombinant FIX usage.

The Company announced preliminary top-line results of the low-dose cohort on January 7^th, 2016, showing that AMT-060 was generally well tolerated. Additionally, the first two patients that had completed at least 12 weeks of follow-up showed promising increases in FIX expression levels of 5.5% and 4.5% of normal as of the December 16, 2015 cutoff date, and in four of the five patients dosed the need for continuous, prophylactic rFIX treatment was eliminated as of January 6^th, 2016. Including the patients screened and treated in the low-dose cohort, a total of eight patients screened to date for the trial have tested negative for pre-existing anti-AAV5 antibodies. uniQure intends to present  an updated  analysis of all patients in the low-dose cohort at a scientific conference in the second quarter of 2016.

"Our goal for the second cohort is to evaluate AMT-060's safety profile at a higher dose and to test our hypothesis that with this dose we could achieve further increases in FIX expression levels," said Deya Corzo, Senior Vice President and Therapeutic Area Head, Liver/Metabolism. "The early data we announced in January indicate that our proprietary AAV5 viral vector can achieve a clinically relevant level of expression of the FIX transgene. Our data thereby reproduced previously published long-term FIX expression levels seen with a dose comparable to that used in our low-dose cohort with the same gene cassette but using a different vector." 

"The successful dosing of the first patient in the high-dose cohort marks another significant step towards bringing this gene therapy to patients," said Dan Soland, CEO of uniQure. "With the greenlight from the Data Monitoring Committee after the first cohort, we are excited to be able to move the program forward. We strongly believe that the product profile of AMT-060 has the potential to improve the quality of life of hemophilia B patients."

The AMT-060-01 Phase I/II study is a 5-year, open-label, uncontrolled, dose-ascending trial that includes two cohorts, with the low-dose cohort using a treatment of 5x10¹² gc/kg and the high-dose cohort using 2x10¹³ gc/kg. AMT-060 consists of a codon-optimized wild type FIX gene and the LP1 liver promoter together with the AAV5 viral vector, manufactured using uniQure's proprietary insect cell based manufacturing technology. It is administered, without immunosuppressant therapy, through the peripheral vein in a single treatment session for approximately 30 minutes. All patients are screened for pre-existing AAV5 antibodies before treatment and Data Monitoring Committee (DMC) reviews are conducted  after each of the first 2 patients in the second cohort.

About Hemophilia B

Hemophilia B is a serious and rare inherited disease in males characterized by insufficient blood clotting.  The condition can lead to repeated and sometimes life-threatening episodes of external and internal bleeding following accidental trauma or medical interventions.  The episodes can cause long-term damage, for example to the joints, and can be fatal if they occur in the brain.  The deficient blood clotting results from the lack of functional human Factor IX, or hFIX. Treatment of hemophilia B today consists of prophylactic or on-demand protein replacement therapy, in which frequent intravenous administrations of plasma-derived or recombinant hFIX are required to stop or prevent bleeding.  Hemophilia B occurs in approximately 1 out of 30,000 live births.

About uniQure

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with CNS, liver/metabolic and cardiovascular diseases. www.uniQure.com

uniQure Forward-Looking Statement

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 course of development of our program hemophilia B. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with collaboration arrangements, our and our collaborators' clinical development activities, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's 2014 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 7, 2015. 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.

CONTACT: uniQure:
         Eva Mulder
         Direct: +31 20 240 6103
         Main: +31 20 240 6000
         [email protected]
         
         Media inquiries:
         Gretchen Schweitzer
         MacDougall Biomedical Communications
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