Exhibit 99.1

 

 

uniQure Announces Second Quarter 2019 Results and Highlights Recent Company Progress

 

~ Presented 36 Weeks of Follow-up Data from Phase IIb Study of AMT-061 Demonstrating Sustained Increases in FIX Activity at up to 54% of Normal ~

 

~ Continuing Patient Recruitment in HOPE-B Pivotal Study of AMT-061, With Completion of Enrollment Expected This Year ~

 

~ On Track to Initiate Patient Dosing of Phase I/II Study in Huntington’s Disease This Year ~

 

Lexington, MA and Amsterdam, the Netherlands, July 29, 2019 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the second quarter of 2019 and highlighted recent progress across its business.

 

“In the second quarter of 2019, we continued to make substantial progress across our pipeline.  Patient enrollment in our ongoing Phase III HOPE-B pivotal study of AMT-061 continues to advance, and we remain on track to complete patient enrollment by the end of this year,” stated Matt Kapusta, chief executive of uniQure. “We are particularly encouraged by the Phase IIb data on AMT-061 and the long-term Phase I/II data on our first-generation AMT-060 presented earlier this month, which we believe demonstrate the potential of AMT-061 to provide durable increases of FIX activity into the normal range for people living with hemophilia B. In addition, significant efforts and progress have been made to initiate our Phase I/II study of AMT-130 in Huntington’s disease.  There is considerable patient and investigator enthusiasm for a potential one-time administered treatment of this devastating disease, and we look forward to our planned initiation of patient dosing in this landmark study this year.”

 

Recent Company Progress

 

·                 Advancing late-stage development of AMT-061 for the treatment of hemophilia B

 

·                  Earlier this month, the Company presented updated clinical data at the 2019 ISTH conference demonstrating that a single administration of AMT-061 resulted in sustained increases in Factor IX (FIX) levels up to 54% of normal, with a mean for the three patients in the Phase IIb study of 45% of normal at 36 weeks post administration. No patient experienced a material loss of FIX activity, reported any bleeding events or required any infusion of FIX replacement therapy for bleeds.

 

·                  Patient recruitment and additional site activations continue to advance for the ongoing HOPE-B pivotal trial of AMT-061. The Company currently has 38 sites activated across 9 countries and the study is expected to complete enrollment this year.

 

·                 Advancing AMT-130 into clinical development for the treatment of Huntington’s disease

 

·                  The Company continues to make significant progress in the preparation for its dose-escalating, randomized and controlled Phase I/II clinical study to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington’s disease. cGMP clinical material has been manufactured at the Company’s state-of-the-art Lexington facility and released for shipment.

 


The following information was filed by Uniqure N.V. (QURE) on Monday, July 29, 2019 as an 8K 2.02 statement, which is an earnings press release pertaining to results of operations and financial condition. It may be helpful to assess the quality of management by comparing the information in the press release to the information in the accompanying 10-Q Quarterly Report statement of earnings and operation as management may choose to highlight particular information in the press release.

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