- 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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