Exhibit 99.1

 

 

uniQure Announces 2018 Financial Results and Highlights Recent Company Progress

 

~ Initiated Dosing Phase of HOPE-B Pivotal Study of AMT-061 in Hemophilia B ~

 

~ Reported Updated Clinical Data from Phase IIb Study of AMT-061 Demonstrating Sustained FIX Activity at up to 51% of Normal, with Mean FIX of 38% of Normal at Twelve Weeks After Administration ~

 

~ Achieved Clearance of Investigational New Drug Application for AMT-130 in Huntington’s Disease Providing for Start of Phase I/II Clinical Trial in 2H 2019 ~

 

~ Announced Expanded Research Pipeline with New AAV Gene Therapy Approaches to Hemophilia A, Fabry Disease and Spinocerebellar Ataxia Type 3 ~

 

Lexington, MA and Amsterdam, the Netherlands, February 28, 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 2018 and highlighted recent progress across its business.

 

“2018 was a year marked by tremendous progress across all of our programs and gene therapy platform,” stated Matt Kapusta, chief executive officer of uniQure. “We’re also off to a very strong start in 2019 having achieved a number of value-driving milestones across our programs, including the initiation of the dosing phase of our global HOPE-B pivotal trial of AMT-061 in hemophilia B and the clearance of our IND for AMT-130 in Huntington’s disease. Looking ahead in the year, we expect to complete enrollment in the HOPE-B pivotal trial and report longer-term follow up from the ongoing Phase IIb study.  We also expect to initiate dosing of a Phase I/II study of AMT-130 and advance our pipeline programs in hemophilia A, Fabry disease and Spinocerebellar Ataxia Type 3.  We believe achieving these goals will move us closer to providing potentially transformative therapies to patients and further demonstrate the importance of our industry leading technology platform and AAV manufacturing capabilities.”

 

Recent Company Progress

 

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

 

·                  The Company announced earlier this month that the first patient has been treated in the global Phase III HOPE-B pivotal study of AMT-061 in hemophilia B. The Company expects to complete the enrollment of approximately 50 patients in the trial by year-end.

 

·                  Also this month, the Company presented updated clinical data on AMT-061 demonstrating increasing and sustained FIX levels of up to 51% of normal after the one-time administration of AMT-061 in the ongoing Phase IIb study. Mean Factor IX (FIX) activity for the three patients at 12 weeks increased to 38% of normal, exceeding threshold FIX levels generally considered sufficient to eliminate or significantly reduce the risk of bleeding events.  None of the patients received Factor infusions, reported bleeding events or required immunosuppression over a combined 42 weeks of observation.  The second and third patients had previously screen-failed and were excluded from another gene therapy study due to pre-existing neutralizing antibodies to a different AAV vector.

 


The following information was filed by Uniqure N.V. (QURE) on Thursday, February 28, 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-K Annual Report statement of earnings and operation as management may choose to highlight particular information in the press release.

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