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uniQure Provides Update on Dose-Confirmation Study of AMT-061 in Patients with Hemophilia B

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Third patient treated in Phase IIb study

Topline data expected to be available before the end of 2018

uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that the third patient has been treated in the Company’s 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 Medicine (PRIME) regulatory initiative by the European Medicines Agency.

The Phase IIb dose-confirmation study is an open-label, single-arm, single-dose trial being conducted in the United States to confirm the dose of AMT-061 prior to patient treatment in the ongoing Phase III HOPE-B pivotal trial. Three patients received a single intravenous (IV) infusion of 2x1013 vc/kg over the past month and are currently under evaluation to assess safety and Factor IX (FIX) activity.

“We are very pleased with the progress we have made in the clinical development of AMT-061, our lead gene therapy product candidate for hemophilia B,” stated Matthew Kapusta, chief executive officer of uniQure. “The treatment of the third patient puts us in the position to present topline data and confirm dosing for our Phase III HOPE-B pivotal study by the end of the year.”

“We also have made advancements in the execution of the HOPE-B pivotal study, which now includes multiple recruiting sites and enrolled patients,” he added. “We believe that AMT-061 has the potential to be a leading gene therapy treatment that provides durable, clinically relevant increases in FIX activity with a favorable immunogenicity profile which could expand patient eligibility for treatment with our gene therapy.”

Phase III HOPE-B Pivotal Trial

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 comparator. After the six-month lead-in period, patients will go on to 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.