Exhibit 99.1

 

 

 

uniQure Announces First Quarter 2020 Financial Results

and Highlights Recent Company Progress

 

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

in Hemophilia B ~

 

~ Announced 22 Presentations at Upcoming ASGCT Virtual Annual Meeting ~

 

Lexington, MA and Amsterdam, the Netherlands, April 29, 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 the first quarter of 2020 and highlighted recent progress across its business.

 

“Despite the ongoing global crisis caused by the coronavirus pandemic, we ended the first quarter of 2020 in a position of strength and remain well capitalized,” stated Matt Kapusta, chief executive officer of uniQure. “I am very proud of the continued dedication of our employees in both the United States and the Netherlands, and thank them for the outstanding work they continue to do in driving forward our clinical programs, research pipeline, and manufacturing and technology operations. We are well positioned for an important second half of the year when we expect to announce new clinical data and advancements from across the business.”

 

Recent Company Progress

 

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

 

·In the first quarter, the Company announced the achievement of targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. A total of 54 patients now have received the one-time dose of etranacogene dezaparvovec.

 

·The Company remains on track to provide 26-weeks of Factor IX data for all 54 patients in the HOPE-B trial before the end of this year and to file an application for marketing authorization in 2021.

 

·The manufacturing process validation for etranacogene dezaparvovec is ongoing, and the Company believes it is able to produce material at commercial scale.

 

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

 

·In March 2020, the Company announced that the first two patients in the Phase I/II clinical trial of AMT-130 in Huntington’s disease have been enrolled after successfully meeting all screening and eligibility criteria. Due to the impact of the coronavirus pandemic, the two procedures that were scheduled to occur in late March have been temporarily postponed.

 

·The Company continues its work to resume treatment in the Phase I/II trial as soon as it is clinically appropriate and will provide further updates on the program at that time.

 


The following information was filed by Uniqure N.V. (QURE) on Wednesday, April 29, 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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