uniQure's hemophilia B gene therapy candidates show long-term benefit

 

• uniQure (NASDAQ:QURE) announces updated clinical data on the three patients treated in its ongoing Phase 2b study evaluating its gene therapy candidate etranacogene dezaparvovec, for the treatment of hemophilia B. Data were presented at the American Society of Hematology.
• Two-year follow-up data show that all three patients have sustained FIX activity (a protein that helps blood form clots to stop bleeding) after the one-time administration of etranacogene dezaparvovec. Mean FIX activity was 44.2% of normal (compared to 41% of normal at 52 weeks).
• At two years after dosing, two of the three participants remain free from bleeds and use of FIX replacement therapy. A single bleed has been reported in one participant, who has used a total of two FIX infusions (excluding surgery). All patients have remained free of prophylaxis in the two years since receiving etranacogene dezaparvovec.
• Additionally, the company announced presentation of up to 5 years of follow-up data on the 10 patients in the Phase 1/2 trial of AMT-060, its gene therapy for the treatment of hemophilia B. All 10 patients continue to show long-term clinical benefit, including sustained increases in FIX activity, reduced usage of FIX replacement therapy, and decreased bleeding frequency. At up to 5 years of follow-up, AMT-060 continues to be well-tolerated, with no new treatment-related adverse events.
• https://seekingalpha.com/news/3642295-uniqures-hemophilia-b-gene-therapy-candidates-show-long-term-benefit