UniQure’s gene therapy potential in haemophilia B

uniQure, a leading gene therapy company announced updated clinical data on the three patients treated in uniQure’s ongoing Phase IIb study of etranacogene dezaparvovec, 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, uniQure presented up to 4 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 are being presented this weekend in poster presentations at the 61^st Annual Meeting of the American Society of Hematology (ASH), taking place in Orlando, Florida.

Key Outcomes

• Sustained FIX Activity at Therapeutic Levels Up to 50% of Normal at One Year After Administration of Etranacogene Dezaparvovec in Phase IIb Study

• Zero Bleeds and No Requirement for Immunosuppression in One Year Following Dosing with Etranacogene Dezaparvovec

• Long-term Clinical Benefit and Tolerability of AMT-060 Maintained in All Patients Through up to 4 Years of Follow-Up

“We are very pleased with these latest results, and continue to believe that etranacogene dezaparvovec has the potential to be the first- and best-in-class gene therapy for patients with hemophilia B,” stated Robert Gut, M.D., Ph.D., chief medical officer of uniQure. “We remain focused on dosing all patients in our ongoing, fully-enrolled HOPE-B pivotal trial, and expect to announce top-line data on our primary endpoint of Factor IX activity by the end of 2020.”

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