Benzinga | Dec 9, 2021 09:45PM EST

uniQure And CSL Behring Announce Primary Endpoint Achieved In HOPE-B Pivotal Trial Of Etranacogene Dezaparvovec Gene Therapy In Patients With Hemophilia B

~ Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec ~



~ Etranacogene dezaparvovec also achieved secondary endpoint demonstrating statistical superiority in reduction of annualized bleeding rate compared to baseline FIX prophylactic therapy ~

~ Stable and durable FIX levels with mean FIX activity of 36.9 percent of normal in full study population at 18-months, compared to a mean of 39.0 percent of normal at 6 months ~

~ Manufacturing operations supporting process validation of etranacogene dezaparvovec successfully completed by uniQure ~