uniQure reports 1-year follow-up data from etranacogene dezaparvovec study
uniQure 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 61st Annual Meeting of the American Society of Hematology, or ASH. "These updated data show that a single administration of etranacogene dezaparvovec has been well-tolerated now out 52 weeks and has increased FIX activity into the therapeutic range for people living with hemophilia B," stated Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan and a principal investigator in the HOPE-B clinical trial. "These data show a full year of meaningful clinical benefit for all three patients in the study, including durable levels of FIX activity with no bleeds, no requirement for infusions of FIX replacement therapy outside of surgery, and no need for immunosuppression." Robert Gut, chief medical officer of uniQure, added: "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. 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."