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First Patient Treated in uniQure’s in Dose-Confirmation Study of AMT-061

uniQure has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B.  AMT-061 has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.


“The initiation of the AMT-061 dose-confirmation study is an important step toward our goal of advancing a potentially life-changing treatment for patients with hemophilia B,” said Robert Gut, M.D., Ph.D., chief medical officer of uniQure.  “I am extremely proud of the efforts made by the uniQure team to initiate this study, the objective of which is to demonstrate meaningful increases in FIX activity using the Padua variant and confirm dosing for the HOPE-B pivotal trial initiated this past June. We look forward to completing patient enrollment shortly and providing top-line data before the end of the year.”


“As a one-time administered therapy, AMT-061 has the potential to transform the treatment paradigm for hemophilia B patients,” said Annette von Drygalski, M.D., associate clinical professor at the University of California San Diego and director of its hemophilia and thrombosis treatment center. “By incorporating both AAV5 and the FIX-Padua variant, AMT-061 has the potential to deliver clinically relevant increases in FIX activity with low risk of cellular immune responses, which could expand patient eligibility for treatment with gene therapy. I greatly appreciate the opportunity to participate in the AMT-061 clinical program and view the initiation of the Phase IIb and Phase III studies as important milestones in the development of this potentially important therapy for patients with hemophilia B.”


The Phase IIb dose-confirmation study is an open-label, single-arm, single-dose trial being conducted in the United States. Approximately three patients are expected to receive a single intravenous (IV) infusion of 2×1013 vc/kg and be evaluated for a period of approximately six to eight weeks to assess Factor IX (FIX) activity.


Patient enrollment is also underway in the global Phase III HOPE-B clinical trial to evaluate the safety and efficacy of AMT-061.  Approximately 50 adult hemophilia B patients classified as severe and moderately-severe will be enrolled in a six-month observational period during which time they will continue to use their current standard of care to establish a baseline control.  After the six-month lead-in period, patients will go onto receive a single intravenous administration of AMT-061.  Dosing of patients in the HOPE-B pivotal trial is expected to start early in the first quarter of 2019.

Source: uniQure