July 27, 2016 4:10pm

Patients in the low-dose cohort have sustained improvements in their disease phenotype and continue to maintain durable levels of Factor IX (FIX) gene activity for up to 39 weeks post treatment

 

 

uniQure N.V. (QURE), a leader in human gene therapy, today announced updated clinical data from its ongoing P1/2 trial of AMT-060, its proprietary, investigational gene therapy, in patients with severe hemophilia B.

 

The P1/2, open-label, multi-center study includes 10 patients each receiving a one-time, 30-minute, intravenous administration of AMT-060, without the use of corticosteroids. The study includes two dose cohorts of five patients each, with the first cohort receiving 5x1012 gc/kg and the second cohort receiving 2x1013 gc/kg. All patients in the trial had documented severe or moderately-severe hemophilia, including documented FIX levels less than 1-2% of normal, and required chronic infusions of prophylactic or on-demand FIX therapy at the time of enrollment.

  • There were no screen failures due to pre-existing anti-AAV5 neutralizing antibodies (NABs) in the study.

The updated data show that all patients in the low-dose cohort have sustained improvements in their disease phenotype and continue to maintain durable levels of Factor IX (FIX) gene activity for up to 39 weeks post treatment.

  • Four out of the five patients are over 50 years of age and had advanced joint disease at study entry. These updated data are being presented today in an oral session of the World Federation of Hemophilia (WFH) 2016 World Congress taking place in Orlando, Florida;
  • The majority of patients in this low-dose cohort of AMT-060 are showing FIX activity in the range of 5% of normal and clinical experience has shown that patients in this range generally do not require prophylactic factor replacement and have a very low frequency of spontaneous joint bleeding episodes," Professor Miesbach added. "Furthermore, at up to 9 months of follow up, AMT-060 continues to be well-tolerated and show durable, clinically relevant levels of FIX activity, which is the key goal of an effective gene therapy approach."

 

The Bottom Line: Data from low-dose cohort of AMT-060 clinical trial continues to show durability and therapeutically relevant Factor IX (FIX) activity in severe, older patients with advanced joint disease and frequent bleeding episodes demonstrated up to 9 months of sustained levels of Factor IX Activity and therapeutic effect as four patients remain free of prophylactic FIX infusions, achieving 82% reduction in total FIX usage.

This effect is particularly important because it is seen in severe patients with established joint disease who experienced a high frequency of joint bleeds despite intense use of prophylactic FIX prior to study entry.

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