Please wait while we load the requested 10-K report or click the link below:
uniQure Announces 2016 Financial Results and Provides Update on Company Progress
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, March 15, 2017 (GLOBE NEWSWIRE) uniQure N.V. (NASDAQ:QURE), a leading gene therapy company developing transformative therapies for patients with severe medical needs, today announces its financial results for the year 2016 and provides an update on company progress.
In 2016, we made significant progress across all of our core programs and implemented strategic changes that have strengthened our organization and enhanced focus on our key priorities, stated Matthew Kapusta, chief executive officer of uniQure. We enter 2017 with a solid financial position and strong clinical data to advance our hemophilia B program into a Phase III study, as well as to progress our gene therapy candidates in Huntingtons disease and congestive heart failure towards IND filings. We believe the achievement of these objectives will deliver meaningful value to shareholders.
Recent Highlights from 2016
· Promising Updated Phase I/II Data Presented on AMT-060 at ASH
· Updated results on AMT-060, including up to 52 weeks of follow-up on the first patient cohort receiving 5x1012 gc/kg and up to 26 weeks of follow-up on the second patient cohort receiving a higher dose of 2x1013 gc/kg, were presented at the 58th American Society of Hemophilia Annual Meeting.
· Patients in the first dose cohort that discontinued prophylactic FIX infusions experienced an 85 percent reduction in total FIX usage and a marked reduction in frequency of spontaneous bleeding after treatment with AMT-060, including a complete cessation of spontaneous bleeding during the last 14 weeks of observation. The five patients in the second dose cohort experienced a near cessation of spontaneous bleeds with only one spontaneous bleed reported in 96 weeks of cumulative observation.
· At both doses, AMT-060 appears to be safe and well-tolerated with no loss of FIX activity, no activation of T-cell response and no development of inhibitors for any of the 10 patients in the study. None of the 10 patients in the study tested positive for anti-AAV5 antibodies.
· FDA Breakthrough Therapy Designation Granted for AMT-060 in Hemophilia B
· In January 2017, the U.S. Food and Drug Administration (FDA), granted Breakthrough Therapy Designation for AMT-060 in Hemophilia B patients. The designation is based on results from the ongoing Phase I/II study in patients with severe disease at up to 12 months follow-up. According to FDA data for its fiscal year 2016, the Center for Biologics Research and Review received a total of 23 requests for Breakthrough Therapy designation with only four designations granted, or 17% of all requests.
The following information was filed by Uniqure N.V. (QURE) on Wednesday, March 15, 2017 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.
View differences made from one year to another to evaluate Uniqure N.V.'s financial trajectory
Compare this 10-K Annual Report to its predecessor by reading our highlights to see what text and tables were
removed , and by Uniqure N.V..