uniQure reports new Phase 2b data for hemophilia B gene therapy candidate


uniQure (NASDAQ:QURE) reported new data from its Phase 2b trial of hemophilia B candidate, AMT-061, which demonstrated sustained activity six months after administration in three patients.

AMT-061 is a therapy that delivers the gene for Padua variant factor IX (FIX), a blood clotting factor that is not made in sufficient quantities in patients with hemophilia B. AMT-061 has received FDA breakthrough therapy designation and access to the EMA’s priority medicine regulatory initiative.

AMT-061 demonstrated increasing and sustained FIX levels after the one-time IV administration. Two of the three patients achieved normal FIX activity, with mean FIX activity for all three patients increasing to 47% of normal at six months post-administration.

“Our goal with AMT-061 is to give all people living with hemophilia B access to a one-time treatment capable of normalizing FIX activity and eliminating the need for replacement therapy, without the risk of immune responses that require immunosuppression or may lead to a loss of efficacy,” Matt Kapusta, uniQure’s CEO, said in a statement.

UniQure is currently dosing hemophilia B patients in a pivotal Phase 3 trial of AMT-061 and hopes to complete enrollment of some 50 patients by the end of 2019.

Sydney Stewart