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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,
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[{"type":"text","content":"~ 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 ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands and KING OF PRUSSIA, Pa., Dec. 09, 2021 (GLOBE NEWSWIRE) -- CSL Behring, a global biotherapeutics leader, and uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe to moderately severe hemophilia B, achieved the pre-specified primary endpoint of non-inferiority in annualized bleeding rate (ABR) 18-months following administration compared to baseline Factor IX (FIX) prophylactic therapy in the pivotal Phase III HOPE-B gene therapy trial. The study also successfully achieved a secondary endpoint demonstrating statistical superiority in reduction of ABR compared to baseline FIX prophylactic therapy. The primary endpoint in the pivotal study was 52-week ABR after achievement of stable FIX expression compared with the six-month lead-in period, considering all bleeds regardless of investigator adjudication as true bleeds. For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented likely steady-state FIX transgene expression. Secondary endpoints included assessment of FIX activity and statistical superiority of ABR after dosing. “We are very pleased with these top-line results from what is the largest and first pivotal trial of a gene therapy for patients with hemophilia B,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “The HOPE-B data ...