QURE provided updated results on 2/8/19 from its upgraded hemophilia B gene therapy with the Padua Factor IX variant (AMT-061). The data continues to be impressive with these 3 patients in this dose confirmation study that has led to the start of the Ph3 study at this dose. Factor IX levels were on average 38% of normal at 12 weeks, which is above the level needed to correct severe bleeding in these patients. With the prior version of their therapy (AMT-060), FIX levels of 7% of normal were achieved at similar times and were effective to stop the need for FIX blood infusions in 8 of 9 patients. In the current study with the AMT-061 product, one patient achieved FIX levels of 51% of normal at twelve weeks. All 3 patients had been excluded from competitive trials because of pre-existing antibodies against AAV. The QURE AAV vector continues to show best in class results with respect to not eliciting an immune response even when pre-existing AAV antibodies are present. Furthermore, FIX levels are now similar to those obtained with the best competitive product of Spark/Pfizer.
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