Exhibit 99.1

 

 

uniQure Announces First Quarter 2019 Results and Highlights Recent Company Progress

 

~ Presented Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B Demonstrating Increases in FIX Activity Sustained at up to 51% of Normal at 12 Weeks

 

~ Achieved IND Clearance and Fast Track Designation for AMT-130 in Huntington’s disease

 

~ Announced 6 Presentations at Upcoming ASGCT Meeting, Including Preclinical Data on Research Pipeline

 

Lexington, MA and Amsterdam, the Netherlands, April 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 first quarter of 2019 and highlighted recent progress across its business.

 

“During the first quarter of 2019, we continued to make excellent progress across our portfolio of gene therapy product candidates,” stated Matt Kapusta, chief executive of uniQure. “We remain highly encouraged by the continued follow-up from our Phase IIb study of AMT-061, which shows the potential to normalize FIX activity in patients with hemophilia B. We are now focused on advancing our ongoing Phase III HOPE-B pivotal study of AMT-061 and have made significant progress activating sites and enrolling patients, with a goal of completing patient enrollment by the end of this year. We are also pleased to announce that AMT-061 received Orphan Drug Designation by the U.S. Food and Drug Administration, which, combined with our FIX-Padua intellectual property position and AAV5’s potentially favorable immunogenicity profile, may provide a meaningful first-mover advantage.”

 

“With the clearance of our Investigational New Drug application for AMT-130 earlier this year, we are making headway in the preparations for our Phase I/II study of AMT-130 in Huntington’s disease.  AMT-130, which recently received Fast Track Designation, is the world’s first one-time administered therapy for Huntington’s disease to enter clinical testing, and we continue to expect patient dosing in this landmark study to begin in the second half of the year.”

 

Recent Company Progress

 

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

 

·                  Enrollment of patients in the global HOPE-B pivotal trial in hemophilia B is advancing and the Company currently is on track to complete enrollment by the end of 2019. In February, the Company presented updated clinical data on AMT-061 demonstrating sustained increases in Factor IX activity (FIX) up to 51% of normal and mean FIX activity for the three patients of 38% of normal at 12 weeks. None of the patients received Factor infusions, reported bleeding events or required immunosuppression over a combined 42 weeks of observation.

 

·                  On April 17, 2019 the U.S. Food and Drug Administration (FDA) granted AMT-061 Orphan Drug Designation (ODD). ODD in the U.S. provides special status for investigational drugs being developed for rare diseases considered to affect only up to 200,000 people in the U.S. The ODD program offers product market exclusivity for up to seven years in the U.S. following regulatory approval, along with tax and financial incentives for companies developing medicines for such orphan indications.

 


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