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uniQure Announces Two-Year Follow-Up Data from the Phase IIb Study of Etranacogene Dezaparvovec and Long-Term Follow-Up Data for AMT-060 in Patients with Hemophilia B
~ Sustained FIX Activity at Therapeutic Levels1 with a Mean of 44% of Normal at Two Years After Administration of Etranacogene Dezaparvovec in Phase IIb Study
About this update from Uniqure N.v.
[{"type":"text","content":"~ Sustained FIX Activity at Therapeutic Levels1 with a Mean of 44% of Normal at Two Years After Administration of Etranacogene Dezaparvovec in Phase IIb Study ~ ~ Long-term Clinical Benefit and Tolerability Maintained in All Patients Through up to Five Years of Follow-Up in Phase I/II Study of AMT-060 ~ LEXINGTON, Mass. and AMSTERDAM, Dec. 07, 2020 (GLOBE NEWSWIRE) -- 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 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. These clinical data were presented today in an oral presentation at the virtual 62nd Annual Meeting of the American Society of Hematology (ASH). In addition, uniQure presented up to 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, in a poster presentation at the meeting. The oral presentation slides and poster are available in the Investor Relations section of uniQure’s website at www.uniQure.com under Investor Downloads. At Least Two Years of Stable, Therapeutic Levels of FIX Activity in Patients Treated with Etranacogene Dezaparvovec The Phase IIb study of etranacogene dezaparvovec is an open-label, single-dose, single-arm, multi-center trial being conducted in the United States. Three patients with severe hemophilia (endogenous Factor IX (FIX) activity less than or equal to one percent) were enrolled in the study and received a single intravenous infusion of 2x1013 gc/kg. Prior to the administration of etranacogene dezaparvovec, all three patients showed low levels of pre-existing neutralizing antibodies to AAV5 but were not excluded from the trial on that basis. The patients in the Phase IIb study have now been followed for two years to assess FIX activity, bleeding rates and usage of FIX replacement therapy, and will be monitored for five years to evaluate the safety of etranacogene dezaparvovec. In today’s oral presentation at ASH, two-year follow-up data show that all three patients have sustained FIX activity at therapeutic lev...