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uniQure Announces Long-Term Clinical Data from Ongoing Phases and Confirms Dose for Hemophilia B Study

Industry News
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.

uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated results from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company’s next-generation gene therapy candidate currently in late-stage clinical development for patients with hemophilia B. AMT-060 is a first-generation gene therapy consisting of an AAV5 vector carrying a gene cassette with the wild-type FIX gene. The data on AMT-060 includes up to two and a half years of follow-up from the low-dose cohort and up to two years of follow-up from the second, higher-dose cohort.  These clinical data were presented on Sunday, December 2 in a poster presentation at the 60th American Society of Hematology (ASH) Annual Meeting taking place in San Diego, Calif.
AMT-060 continues to be safe and well-tolerated, with no new serious adverse events and no development of inhibitors. All 10 patients sustained increases in FIX activity and improvements in their disease state as measured by reduced usage of FIX replacement therapy and decreased bleeding frequency.
All five patients in the second dose cohort of 2×1013 gc/kg continue to be free of routine prophylaxis at up to two years after treatment.  During the last 12 months of observation, the mean annualized bleeding rate was 0.5 bleeds, representing an 88% reduction compared to the year prior to treatment.  During the same period, the usage of FIX replacement therapy declined 93% compared to the year prior to treatment.  Mean FIX activity increased from 7.1% in the first year after treatment to 8.3% in the second year and was 8.9% of normal at the last measurement.
“With up to two and a half years of follow-up, patients in the study continue to show evidence of durable clinical benefits, including sustained FIX activity, substantial reductions in the usage of replacement therapy and a near cessation of spontaneous bleeds,” stated Professor Frank W.G. Leebeek, M.D. Ph.D. of the Erasmus University Medical Center in Rotterdam, the Netherlands. “Most importantly, since the last data update at ASH 2017, the study continues to demonstrate the long-term safety and tolerability of AAV5-based gene therapies, with no new treatment-related adverse events, no toxic cellular immune responses and no patients losing FIX activity.”
Advancing AMT-061 in Late-Stage Clinical Development
The Company recently announced initial clinical data in patients treated in a Phase IIb dose-confirmation study of AMT-061, a next-generation, AAV5-based gene therapy containing a patent protected FIX-Padua variant for the treatment of hemophilia B.  Six weeks after administration, mean FIX activity for the three patients in the study was 31% of normal, and FIX levels continue to increase beyond the initial six to ten weeks of follow up.  Based on the data obtained to date, no patient has required any infusions of FIX replacement therapy, reported any bleeding events or required any immunosuppression therapy.
Read the press release in its entirety here. 

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