CSL Behring, UniQure's Hemophilia B Gene Therapy Hits Primary Endpoint

CSL Behring and uniQure N.V.'s QURE etranacogene dezaparvovec, a hemophilia B gene therapy, achieved the pre-specified primary endpoint of non-inferiority in annualized bleeding rate (ABR) compared to baseline Factor IX (FIX) prophylactic treatment.

• The study also achieved a secondary endpoint demonstrating statistical superiority in reducing ABR compared to baseline FIX prophylactic therapy.
• CSL Behring plans to submit regulatory applications for marketing approval of etranacogene dezaparvovec in the U.S. and Europe in 1H of 2022.
• ABR for all bleeds after stable FIX expression, assessed at 18 months, was 1.51 compared with the ABR of 4.19 for at least six months for the lead-in period.
• ABR for investigator-adjudicated FIX-treated bleeds was 0.83 compared with lead-in ABR of 3.65.
• Stable and durable FIX levels with mean FIX activity of 36.9% of normal in the full study population at 18-months, compared to a mean of 39% of normal at six months.
• Etranacogene dezaparvovec was generally well-tolerated, with over 80% of adverse events considered mild. 
• Related: One Year Following Single Administration, UniQure's Hemophilia B Gene Therapy Candidate Shows Sustained Increase In Factor IX Levels.
• Price Action: QURE shares are down 3.24% at $28.40 during the market session on the last check Thursday.