Exhibit 99.1

 

 

uniQure Announces 2017 Financial Results and Recent Company Progress

 

~ On Track to Begin Pivotal Study of AMT-061 in Hemophilia B in Q3 2018

 

~ Submitted IND Amendment for AMT-061 Dose Confirmation Study, with Topline Data Expected by End of 2018

 

~ Expects IND Submission for AMT-130 in Huntington’s Disease in the Second Half of 2018

 

~ $159 million of cash and cash equivalents as of December 31, 2017

 

Lexington, MA and Amsterdam, the Netherlands, March 14, 2018 — 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 2017 and highlighted recent progress across its business.

 

“We ended 2017 with significant momentum across all of our gene therapy programs, and a strong cash position to fund our business into 2020,” stated Matthew Kapusta, chief executive officer of uniQure.  “We remain highly focused on initiating our pivotal study for AMT-061 in hemophilia B and advancing AMT-130 into a Phase I/II study in Huntington’s disease.  We look forward to providing updates on our progress throughout the year, including the presentation of top-line clinical data on AMT-061 by year-end.”

 

Recent Company Progress:

 

·                 Advancing AMT-061 for the treatment of hemophilia B into a pivotal trial

 

·                  In the first quarter of 2018, the Company submitted an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) supporting its planned AMT-061 dose-confirmation study.  Extensive data including comparability, manufacturing capability and non-clinical safety and bioequivalence were included in the submission.  The Company anticipates initiating the treatment of patients early in the third quarter of 2018 at a single dose of 2 x 10(13) gc/kg. Top line data from these patients are expected to be presented by the end of the year.

 

·                  The process of site selection for the pivotal phase III study has been initiated and reviews by institutional review boards (IRB) are underway. The Company expects to begin patient enrollment in the lead-in phase of the pivotal study in the third quarter of 2018.

 

·                  In the first quarter of 2018, the Company completed the full comparability analysis for AMT-061, which the Company believes continues to support the comparability between AMT-060 and AMT-061.

 

·                  The Company has completed the manufacturing and full quality release of product for use in the dose confirmation study. The product was produced in the Company’s state-of-the-art manufacturing facility in Lexington, Massachusetts.

 

·                  The European Medicines Agency (EMA) issued a positive opinion on Orphan Drug Designation for AMT-061 in hemophilia B.

 

·                 Advancing AMT-130 for the treatment of Huntington’s disease into a Phase I/II study

 

·                  Preclinical data presented at the 13th Annual Huntington’s Disease Therapeutics Conference in Palm Springs, CA demonstrated that knocking down human mutant huntingtin (mHTT) by AMT-130 was safe and well-tolerated in both the deep structure of the striatum and the cortex when

 


The following information was filed by Uniqure N.V. (QURE) on Wednesday, March 14, 2018 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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