=uniQure (QURE) : Hemophilia B gene therapy program; strengthens intellectual property portfolio

uniQure acquires a patent family, with claims issued in the U.S., that broadly covers a hyperactive variant of Factor IX carrying an R338L mutation and its use in gene therapy for the treatment of coagulopathies, including hemophilia B; terms not disclosed

uniQure announces, following multi-disciplinary meetings w/ the FDA and EMA, plans to expeditiously advance AMT-061 into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B

• Co plans to expeditiously advance AMT-061, which combines an AAV5 vector with the FIX- Padua mutant, into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B.
• The FDA has agreed that AMT-061 will be included under the existing Breakthrough Therapy designation and Investigational New Drug (IND) for AMT- 060. The EMA also has agreed that AMT-061 will be included under the current PRIME designation.
• The Company achieved general agreement with the FDA and EMA on the proposed pivotal trial plan for AMT-061. The study is expected to be an open-label, single-dose, multi-center, multi-national trial investigating the efficacy and safety of AMT-061 administered to adult patients with severe or moderately severe hemophilia B. The primary objective of the trial is to evaluate AMT-061 for prevention of bleedings. Secondary objectives include additional efficacy and safety aspects.