Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here.
uniQure N.V.聽a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced on Dec. 11, 2017, updated results聽from its ongoing, dose-ranging Phase I/II trial of AMT-060, its investigational gene therapy聽in patients with severe hemophilia B. The data includes up to two years of follow-up from the low-dose cohort and up to 18 months of follow-up from the second, higher-dose cohort.
The AAV5-based AMT-060 remains safe and well-tolerated with up to two years of follow-up, with no new serious adverse events and no development of inhibitors. No patient in the study has had any loss of Factor IX (FIX) activity or capsid-specific, T-cell-mediated immune response.
Eighteen-month follow-up data from the second-dose cohort continue to show stable FIX activity with substantial improvement in disease state in all five patients, including the discontinuation of routine prophylactic FIX infusions in all patients that previously required chronic replacement therapy. The annualized spontaneous bleeding rate for the second dose cohort declined 89 percent to a mean of 0.3 bleeds after gene transfer. In the last year of follow-up, no patient in the second cohort has reported any spontaneous bleeds.
These clinical data were presented in an oral presentation at the 58th American Society of Hematology (ASH) Annual Meeting.