Uniqure N.V. (QURE) SEC Filing 10-Q Quarterly report for the period ending Tuesday, March 31, 2020

Earnings Press Release
10-Q Quarterly Report
10-Q QoQ Changes
Uniqure N.V.
Ticker: QURE CIK: 1590560

Exhibit 99.1

uniQure Announces 2019 Financial Results and Highlights Recent Company Progress

~ Patient Dosing Nearly Completed in Phase III HOPE-B Trial of Etranacogene Dezaparvovec
in Hemophilia B ~

~ First Patient Procedure in Phase I/II Clinical Trial of AMT-130 in Huntington’s Disease
Expected to Occur Around the End of the First Quarter of 2020 ~

~ Strong Balance Sheet Expected to Fund Operations into 2022 ~

Lexington, MA and Amsterdam, the Netherlands, March 2, 2020 —

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 2019 and highlighted recent progress across its business.

“The past year was marked by strong execution in our pivotal, Phase III HOPE-B study of etranacogene dezaparvovec in hemophilia B for which we achieved full enrollment and expect to announce top-line data before the end of this year,” stated Matt Kapusta, chief executive officer of uniQure. “We believe etranacogene dezaparvovec has a potential best-in-class profile as a one-time treatment for patients with hemophilia B, and our primary goal is to advance this program for regulatory review and potential approval.”

“We have also successfully screened patients in the randomized Phase I/II clinical trial of AMT-130 in Huntington’s disease and expect the first procedure to occur around the end of this first quarter of 2020,” he added. “In addition, we have continued to advance our research pipeline of innovative AAV gene therapy candidates and further build on our proprietary, commercial-scale manufacturing capabilities. We expect this year to be a transformational one for uniQure in which we announce new clinical data and advance our internally discovered investigational gene therapies.”

Recent Company Progress

Advancing late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B

Nearly all of the eligible patients in the pivotal, Phase III HOPE-B study of etranacogene dezaparvovec have received their one-time administration of etranacogene dezaparvovec. Approximately 60 patients were enrolled in the study, which is being conducted in approximately 40 sites in the United States and Europe. The Company is on track to provide top-line data on all patients in the HOPE-B study before the end of this year, and to submit a Biologics License Application (BLA) for marketing authorization in 2021.

Late last year at the annual meeting of the American Society of Hematology, the Company presented one-year follow-up data on the three patients in the Phase IIb study of etranacogene dezaparvovec. Mean FIX activity for the three patients at 52 weeks after administration was 41% of normal. No patient in the study had reported any bleeding events, and all patients in the study have remained free of prophylaxis after receiving etranacogene dezaparvovec.

The following information was filed by Uniqure N.V. (QURE) on Tuesday, March 3, 2020 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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