uniQure Announces Financial Results For Second Quarter 2016 and Update on Company Progress

~ Progress Across the Pipeline
~ Additional data on AMT-060 in Hemophilia B Expected Later this Year
~Data from Sanfilippo B and Parkinson's Disease Studies Expected in Q1 2017
~Strong Balance Sheet to Support Advancement of Programs

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Aug. 25, 2016 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced its financial results for the second quarter, ended June 30, 2016, and provided an update on corporate developments.

"We continue to make solid progress on our pipeline programs and are pleased to maintain a strong cash position that will enable us to advance our product candidates well into the future," stated Daniel Soland, chief executive officer of uniQure. "We presented promising updated clinical data from the low-dose cohort of our ongoing Phase I/II study of AMT-060, which demonstrated durable, therapeutically relevant Factor IX activity in older patients with severe hemophilia B and advanced joint disease.  Importantly, we expect to present up to one year of follow-up data from our low dose cohort and up to 6 months of follow-up data from our second dose cohort of AMT-060 later this year.  Additionally, we look forward to clinical data presentations from studies in Sanfilippo B and Parkinson's disease in the first quarter of 2017."

Recent Corporate Highlights

Pipeline and Business Updates

Liver/Metabolic Diseases

• Advancing AMT-060 in Hemophilia B - uniQure presented updated results from its ongoing Phase I/II clinical trial of AMT-060 in patients with severe hemophilia B and advanced joint disease at the World Federation of Hemophilia Congress. The clinical data from the low-dose cohort showed sustained Factor IX (FIX) activity and a substantial reduction in FIX usage. All patients had improvements in their disease phenotype and continued to maintain durable levels of FIX activity for up to 9 months post treatment.  Four out of five patients remained free of prophylactic FIX usage with mean steady-state FIX activity of 5.4% of normal and an 82% reduction in total FIX usage through up to 9 months.  Of note, AMT-060 continues to demonstrate a very low screening failure rate, with all patients screened in the study testing negative for pre-existing anti-AAV5 antibodies. All five patients in the second-dose cohort have now been treated, with 6-month data expected to be available for presentation later this year.    

Central Nervous System (CNS) Diseases

• Ongoing Follow-up of AMT-110 in Sanfilippo B - uniQure anticipates that all four patients in this Phase I/II extension study will have completed their 30-month evaluation late this year and that clinical data will be presented in the first quarter of 2017.  uniQure assumed sponsorship of the Phase I/II study extension protocol earlier this year.
• Advancing Preclinical Research of AMT-130 in Huntington's Disease - uniQure is conducting ongoing IND-enabling studies of AMT-130 in non-human primates.  Earlier this year, uniQure published and presented data providing preclinical proof-of-concept and demonstrating the potential of a one-time administration of AAV5-delivered gene therapy to successfully silence the Huntingtin gene.   
• Continued Progress on Phase I/IIa Study of AMT-140 in Parkinson's Disease - Two of the four, six-patient cohorts have now been fully enrolled since June 2016 in an ongoing Phase I/IIa clinical study of AMT-140, an AAV2-GDNF gene therapy for the treatment of Parkinson's disease.  Top-line data, including safety, tolerability and preliminary efficacy results, are expected to be available for presentation in the first quarter of 2017.  The study is being conducted under the direction of Krystof Bankiewicz at the University of California, San Francisco (UCSF) and John Heiss at the National Institutes of Health.  uniQure has exclusive rights to GDNF in gene therapy and has the option to acquire the program from UCSF. 

Cardiovascular Diseases

• Increasing Activity in Support of BMS Collaboration - uniQure and its collaboration partner, Bristol Myers Squibb (BMS), continue to conduct IND-enabling studies for S100A1, a proprietary gene therapy for chronic heart failure.  In addition to S100A1, uniQure and BMS have initiated work on 3 additional targets designated under their collaboration. 

Leadership Team and Board of Directors

• Added Key Talent - In the second quarter, uniQure significantly strengthened its senior management team with the appointment of Paul Firuta as Chief Commercial Officer, Hans Janssen as Senior Vice President, Human Resources, Maria E. Cantor as Senior Vice President, Investor Relations & Communications, and Jonathan Garen as Chief Business Officer.  
• Completed Corporate Governance Changes - Following its Annual General Meeting in June, the Company transitioned from a two-tier Supervisory Board and Management Board to a single Board of Directors structure with executive and non-executive members. This new structure reflects corporate governance standards most familiar to the majority of the Company's shareholder base.
  
  In addition, the Company appointed Jack Kaye, a seasoned financial executive with more than 40 years of diversified experience, to its Board of Directors.  Company shareholders also approved the appointments of Daniel Soland, chief executive officer, and Matthew Kapusta, chief financial officer, as executive directors of the Board.  Finally, as part of the governance transition, Mr. Ferdinand Verdonck and Mr. Joseph Fezcko retired from the board.

Business Development

• In the second quarter, uniQure revised its existing license contract with Protein Sciences Corporation for the use of its expresSF+ (SF+) insect cell line to provide the Company with a royalty free, perpetual right and license to the licensed technology in the field of AAV-based gene therapy.

Financial Highlights for Q2 2016 

As of June 30, 2016, the Company held cash and cash equivalents of €165.6 million, compared with of €203.5 million as of December 31, 2015, a decrease of €37.9 million. The decrease includes €36.4 million of cash used in operating, investing and financing activities during the six months ended June 30, 2016, in addition to a €1.5 million loss related to foreign currency effects on U.S. dollar-denominated deposits. The Company maintains its expectation that existing cash resources will be sufficient to fund operations into the second half of 2018.   

Licensing and collaboration revenues for the three months ended June 30, 2016 were €3.9 million, compared with €1.6 million for the comparable period in 2015. The increase relates to increased research activity associated with S100A1 for heart failure, which are fully reimbursed by BMS in accordance with the Company's collaboration agreement.

Research and development expenses were €17.3 million for the three months ended June 30, 2016, compared with €10.6 million for the comparable period in 2015. The increase is mainly due to increased activity in support of our collaboration agreement with BMS, validation and scale-up activities related to AMT-060 in the Company's Lexington facility, increased facility costs in Amsterdam and the continued progression of our gene therapy product candidates targeting Sanfillippo B, Huntington's disease and hemophilia A.           

Selling, general and administrative expenses were €5.5 million for the three months ended June 30, 2016, compared with €4.5 million for the comparable period in 2015. The change is primarily related to an increase in personnel expense, as well as an increase in expenses associated with the Glybera global registry and Phase IV study, which are currently classified as selling, general and administrative expenses, but were previously capitalized in the same period in 2015 prior to the commencement of Glybera commercialization.

The net loss for the second quarter 2016 was €19.3 million, or €0.77 per share, compared with €18.9 million, or €0.87 per share, for the second quarter of 2015. The Company had 25,137,009 ordinary shares outstanding as of June 30, 2016.

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 liver/metabolic, central nervous system and cardiovascular diseases. www.uniQure.com

uniQure Forward-Looking Statements
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 development of our gene therapy product candidates, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates. 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 2015 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2016. 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 Contacts:

Maria E. Cantor
Direct: 339-970-7536
Mobile:  617-680-9452
m.cantor@uniQure.com

Eva Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com

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