uniQure announces updated AMT-060 results, AMT-061 update at ASH

uniQure 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 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 in a poster presentation at the American Society of Hematology, or ASH. 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.

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