One Year Following Single Administration, UniQure's Hemophilia B Gene Therapy Candidate Shows Sustained Increase In Factor IX Levels

UniQure N.V. QURE has announced 52-week data from its Phase 3 HOPE-B gene therapy trial of etranacogene dezaparvovec to treat hemophilia B.
Data demonstrated durable, sustained increases in Factor IX (FIX) activity at 52-weeks post-infusion with a mean FIX activity of 41.5% of normal, compared to a mean FIX activity of 39% of normal, at 26-weeks of follow-up.
FIX is a protein produced naturally in the body that helps the blood form clots to stop bleeding.
During the 52 weeks, a single dose of etranacogene dezaparvovec significantly reduced the annualized rate of bleeding requiring treatment by 80% from 3.39 at baseline to 0.68 bleeding episodes per year.
The annualized rate of spontaneous bleeding requiring treatment was also significantly reduced by 85%, from 1.16 at baseline to 0.18 bleeds per year.
In all patients, usage of FIX replacement therapy declined 96%, with 52 of 54 patients successfully discontinuing their prophylactic infusions.
Etranacogene dezaparvovec continues to be generally well-tolerated with no treatment-related serious adverse events.
UniQure will conduct as the sole primary endpoint a non-inferiority analysis of annualized bleeding rates (ABR) at 78 weeks after the administration (approximately 52-weeks after steady-state is achieved).
The Company expects all patients to complete their 78-week follow-up visits by the end of Q3 of 2021.
The marketing application is expected to be submitted in Q1 of 2022.
In April, the FDA removed the clinical hold on the hemophilia B gene therapy program after QURE addressed all identified issues related to a single patient diagnosed with hepatocellular carcinoma in the HOPE-B pivotal trial.
Price Action: QURE shares are down 8.71% at $29.98 during the market session on the last check Tuesday.

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