Press "Enter" to skip to content

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

  • UniQure N.V. (NASDAQ: QUREhas 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.

This post was originally published on this site

Be First to Comment

Leave a Reply

Your email address will not be published. Required fields are marked *