uniQure announces long-term follow-up data for AMT 060 in patients with hemophilia B

uniQure N.V. has 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.

In the ongoing Phase I/II study of AMT 060, all 10 patients continue to show long-term meaningful clinical impact, including sustained increases in FIX activity and improvements in their disease state as measured by reduced usage of FIX replacement therapy and decreased bleeding frequency. At up to 3.5 years of follow-up, AMT-060 continues to be safe and well-tolerated, with no new serious adverse events and no development of inhibitors since the last reported data. All five patients in the second dose cohort of 2x1013 gc/kg continue to be free of routine FIX replacement therapy at up to 3 years after treatment.

During the last 12 months of observation, the mean annualized bleeding rate was 0.7 bleeds, representing an 83% reduction to the year prior to treatment. During this same period, the usage of FIX replacement therapy declined 96% compared to the year prior to treatment. Steady-state mean yearly FIX activity at 3 years was 7.9%, as compared to 7.1% in the first year and 8.4% in the second year. AMT-060 is uniQure’s first-generation gene therapy, consisting of an AAV5 vector carrying a gene cassette with the wild-type FIX gene. Data from this Phase I/II trial of AMT 060 will be part of the regulatory submission for marketing approval of AMT 061.

" The Phase I/II study of AMT 060 continues to demonstrate the impressive long-term safety and tolerability of uniQure’s gene therapy platform in hemophilia B,” stated Professor Frank W.G. Leebeek, M.D., Ph.D. of the Erasmus University Medical Center in Rotterdam, the Netherlands. “We have now demonstrated evidence of durable clinical benefits, including sustained FIX activity, improved disease phenotype and substantial reductions in spontaneous bleeds for up to 3.5 years after administration. These data are very encouraging for uniQure’s hemophilia B program.”.