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uniQure Announces Second Quarter 2018 Financial Results and Highlights Company Progress
~ Patient Enrollment Underway in Global Phase III HOPE-B Pivotal Study of AMT-061
~ First Patient Successfully Screened into Phase IIb Dose-Confirmation Trial of AMT-061
~ Robert Gut, M.D., Ph.D., Named Chief Medical Officer and Other Appointments to Clinical Development Leadership
~ Ended Second Quarter with $259 Million in Cash and Cash Equivalents; Expected to Fund Operations into 2021
Lexington, MA and Amsterdam, the Netherlands, August 8, 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 the second quarter of 2018 and highlighted recent progress across its business.
We have made tremendous progress over the past several months advancing our gene therapy programs in hemophilia B and Huntingtons disease, as well as in expanding our intellectual property portfolio and executing on our manufacturing plan, said Matt Kapusta, chief executive officer of uniQure. Our Phase III pivotal trial of AMT-061 is actively enrolling patients, and we have consented and successfully screened the first of three patients in our Phase IIb dose-confirmation study.
The initiation of our registrational study provides the opportunity for uniQure to be first to market with a one-time treatment that has the potential to transform the standard of care for patients with hemophilia B, Mr. Kapusta added. We continue to work toward what we expect will be an equally productive second half of 2018 with the achievement of multiple value-creating catalysts across the company, including top-line data from our dose-confirmation study, the submission of an Investigational New Drug application for AMT-130 in Huntingtons disease, and hosting a Research and Development Day that will feature expansion of our early-stage pipeline.
Recent Company Progress
· Advancing AMT-061 into late-stage clinical development for the treatment of hemophilia B
· The first patient in the Phase IIb dose-confirmation study of AMT-061 in hemophilia B has consented and successfully screened into the study and is expected to be treated shortly. The Phase IIb study is an open-label, single-arm, single-dose trial being conducted in multiple centers in the U.S. Three patients will receive a single intravenous (IV) infusion of 2x1013 vc/kg and be evaluated for a period of approximately six to eight weeks to assess Factor IX (FIX) activity and confirm the dose for the global Phase III HOPE-B study. Top-line data are expected to be made available by the end of this year.
· Patient enrollment has commenced for the global Phase III HOPE-B pivotal study. This registrational study includes a six-month lead-in phase to collect baseline data as patients will serve as their own control. The Company expects to begin dosing patients with AMT-061 in the first quarter of 2019.
The following information was filed by Uniqure N.V. (QURE) on Wednesday, August 8, 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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