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uniQure announces first patient treated in HOPE-B trial of AMT-061.

uniQure announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the patent-protected 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 FDA and access to Priority Medicines regulatory initiative by the European Medicines Agency. The global HOPE-B Phase III clinical trial will evaluate the efficacy and safety of AMT-061. Approximately 50 adult patients with hemophilia B classified as severe or 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 prospective comparator. After the six-month lead-in period, patients will receive a single intravenous administration of AMT-061. The primary outcome measure is the assessment of Factor IX activity 26 weeks after AMT-061 dosing. Secondary outcome measures include annualized bleeding rate and usage of Factor IX replacement therapy over a 52-week time frame, as well as other efficacy and safety aspects. Post-treatment, patients will be followed for 5 years.

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Publication:The Fly
Date:Feb 4, 2019
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