uniQure Has Follow-Up Data from Phase 2b Hemophilia B Study

~ FIX Activity Up to 54% of Normal, with Mean of 45% of Normal, at 36 WeeksAfter Administration of AMT-061 in Phase IIb Study ~

~ Clinical Benefit and Tolerability of AMT-060 Maintained in All Patients Through up to 3.5 Years of Follow-up ~

uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated clinical data on the three patients treated in the Company’s ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. In addition, the Company presented up to 3.5 years of follow-up data on the 10 patients in the Phase I/II trial of AMT-060, its first-generation gene therapy for the treatment of hemophilia B. These clinical data were presented on Saturday, July 6 in oral presentations at the 27th Congress of the International Society on Thrombosis and Haemostasis (ISTH), taking place in Melbourne, Australia.

Featured in an oral presentation at ISTH, the 36 weeks of follow-up data show that all three patients have sustained increases in FIX levels after the one-time administration of AMT-061, with two of the three patients maintaining FIX activity in the normal range. Mean FIX activity for the three patients at 36 weeks after administration was 45% of normal, with the first patient achieving FIX activity of 54% of normal, the second patient achieving FIX activity of 30% of normal and the third patient achieving FIX activity of 51% of normal. The second and third patients had previously screen-failed and were excluded from another gene therapy study due to pre-existing neutralizing antibodies to a different AAV vector. Reported FIX activity was measured using an activated partial thromboplastin time (aPTT) assay performed at a central laboratory.

No patient in the study has experienced a material loss of FIX activity, reported any bleeding events or required any infusions of FIX replacement therapy for bleeds. One patient underwent hip surgery due to a pre-existing condition and was treated perioperatively with short-acting factor replacement. This was reported by the investigator as a serious adverse event unrelated to AMT-061.

“These updated data continue to show that a single administration of AMT-061 is well-tolerated and has the potential to increase FIX activity into the normal range for people living with hemophilia B,” stated Matt Kapusta, chief executive officer of uniQure. “We continue to be very pleased with these results, which as of this data report show durable increases in FIX activity with no bleeds and no requirement for infusions of FIX replacement therapy or immunosuppression. We believe AMT-061 has the potential to be a first- and best-in-class gene therapy for patients with hemophilia B, and we remain focused on our goal of completing enrollment in our ongoing HOPE-B pivotal trial by the end of this year.”

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