uniQure Announces Financial Results For Third Quarter 2016 And Provides Update On Company Progress

Published: Nov 22, 2016

~ Strategic Review Completed to Refocus Pipeline, Reduce Operating Costs and Deliver Long-Term Shareholder Value

~ Updated Clinical Data on AMT-060 in Hemophilia B at Upcoming ASH Annual Meeting

Lexington, MA and Amsterdam, the Netherlands, November 22, 2016 - uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced its financial results for the third quarter ended September 30, 2016 and provided an update on corporate developments.

"With the completion of our company-wide strategic review, we have focused our pipeline, streamlined operations and improved our financial position," stated Matthew Kapusta, interim chief executive officer of uniQure. "Our near-term priorities are to aggressively prepare for a pivotal study in hemophilia B and to complete IND-enabling studies for our programs in Huntington's disease and congestive heart failure. We look forward to the presentation of updated Phase I/II data on AMT-060 in a couple of weeks at ASH, and discussing our late-stage development plans."

Recent Highlights

• Completed strategic review to refocus pipeline, simplify organization and reduce operating expenses - On November 15, uniQure announced the completion of a company-wide strategic review aimed at focusing its pipeline, consolidating its manufacturing and enhancing overall execution to drive shareholder value. As a result of this initiative, the Company will prioritize programs in hemophilia B, Huntington's disease and those associated with its landmark collaboration with Bristol-Myers Squib (BMS) in cardiovascular disease. Regarding the Company's clinical product candidates in Sanfilippo B and Parkinsons' disease, the Company has initiated discussions with its collaborators to explore options for these programs, including their potential transfer or partnership. Additionally, the Company will restructure its research and development organization in the Netherlands and consolidate manufacturing in the United States.

• Updated Phase I/II data on AMT-060 at ASH; Preparations for pivotal study underway - Professor Frank W.G. Leebeek, a clinical investigator at the Erasmus Medical Center in Rotterdam, the Netherlands, will be presenting new and updated data from uniQure's ongoing Phase I/II clinical trial of AMT-060 at the 58th American Society of Hematology (ASH) Annual Meeting, on Saturday, December 3, 2016. The data will include up to 52 weeks of follow-up on the first patient cohort receiving 5x1012 gc/kg and up to 26 weeks of follow-up on the second patient cohort receiving a higher dose of 2x1013 gc/kg. In early 2017, uniQure expects to initiate interactions with regulatory authorities regarding a potential pivotal study of AMT-060 and will commence scaled-up production of clinical material in its Lexington, MA manufacturing facility.

• Progressing IND-enabling studies of AMT-130 - uniQure is conducting ongoing preclinical studies of AMT-130, its wholly owned AAV5 gene therapy product candidate for Huntington's disease. AMT-130 has demonstrated in preclinical studies up to 80 percent knockdown of the Huntingtin gene in the cortex and putamen in rat and humanized mouse models. Additional preclinical data, presentations and publications are expected in 2017 that will feature ongoing progress in moving toward filing an investigational new drug (IND) application to begin clinical studies.

• Advancing S100A1 through multiple preclinical studies - Significant progress has been made over the past year in transferring S100A1 from a mammalian system to an insect cell preparation, as well as conducting preclinical dose-ranging analyses and comparability studies. Various administration techniques have been analyzed and further animal studies are expected in 2017 to support an IND filing in 2018. uniQure also has begun early work on two additional cardiovascular targets, including vector development and other preclinical activities.

• Maintaining strong cash position sufficient to fund operations into 2019 - As of September 30, 2016, the Company held cash and cash equivalents of €140.3 million. uniQure expects to realize significant cost savings as a result of the activities associated with its strategic restructuring. Specifically, uniQure expects to realize €5 million to €6 million of annualized cost savings in personnel and other related operating expenses as a result of the elimination of approximately 50 to 60 positions, or 20% to 25% of global headcount, by the end of 2017. Additionally, the Company expects to further reduce planned operating expenses by €11 million to €15 million over the next two years through the focusing of its pipeline. Based on its strong cash position and these cost savings, uniQure believes its existing cash resources will be sufficient to fund operations into 2019.

Financial Highlights for Q3 2016

Revenues for the three months ended September 30, 2016 were €6.5 million, compared with €3.1 million for the comparable period in 2015. The increase relates to increased research activity associated with S100A1 for heart failure, the expenses of which are fully reimbursed by BMS in accordance with the Company's collaboration agreement.

Research and development expenses were €15.3 million for the three months ended September 30, 2016, compared with €11.9 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 clinical program for AMT-060 in hemophilia B and preclinical candidate AMT-130 in Huntington's disease.

Selling, general and administrative expenses were €4.1 million for the three months ended September 30, 2016, compared with €4.8 million for the comparable period in 2015. The decrease results from a reduction in professional fees and a reversal of share based payment expenses for unvested equity grants to our former CEO upon his resignation in September 2016, offset in part by expenses associated with the Glybera Phase IV study and global registry, which in prior years were capitalized.

Losses related to foreign currency effects on U.S. dollar-denominated deposits were €0.6 million for the three months ended September 30, 2016, compared to €1.6 million for the comparable period in 2015. Net finance costs were €0.2 million for the three months ended September 30, 2016, compared to a gain of €1.2 million for the comparable period in 2015.

The net loss for the third quarter 2016 was €13.5 million, or €0.54 per share, compared with €25.8 million, or €1.08 per share, for the third quarter of 2015.

As of September 30, 2016, the Company held cash and cash equivalents of €140.3 million and had 25,143,760 ordinary shares outstanding. Cash used in operating and investing activities in the third quarter of 2016 includes approximately €6.6 million of one-time capital expenditures related to the refurbishment of a new R&D facility in Amsterdam, the Netherlands. Excluding these one-time expenditures related to the new facility, cash used in operating and investing activities in the third quarter of 2016 would have been €18.0 million.

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

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