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uniQure Announces Positive Top-Line Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B
~ Phase III study in 54 patients met primary endpoint with mean Factor IX activity of 37% of normal at 26 weeks ~ ~ Patients achieved significant increases in
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[{"type":"text","content":"~ Phase III study in 54 patients met primary endpoint with mean Factor IX activity of 37% of normal at 26 weeks ~\n ~ Patients achieved significant increases in Factor IX activity irrespective of pre-existing neutralizing antibodies, potentially supporting broad patient access ~ ~ Increases in Factor IX activity were sustained for up to 18 months with near elimination of bleeding ~ ~ Mean annualized usage of FIX replacement therapy declined by 96 percent after dosing compared to the observational lead-in period ~ ~ Etranacogene dezaparvovec was well-tolerated with no treatment-related serious adverse events ~ ~ Data selected for late-breaker oral presentation on December 8, 2020 at the Annual Meeting of the American Society of Hematology (ASH) ~ LEXINGTON, Mass. and AMSTERDAM, Nov. 19, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive top-line data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B. This is the first data set to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of patients receiving a single gene therapy investigational product to be reported to date. These clinical data were published today as a late-breaking abstract, one of only six accepted for presentation at the 62nd Annual Meeting of the American Society of Hematology (ASH) and will be featured as an oral presentation in the conference on December 8, 2020. The abstract is available here. “We are extremely pleased that these top-line pivotal data show that a single administration of etranacogene dezaparvovec gene therapy led to sustained increases of Factor IX (FIX) to functionally-curative levels capable of eliminating the need for regular infusions to control and prevent bleeding episodes,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “Most impressively, these data also demonstrate the potential to achieve clinical benefit in patients with a range of pre-existing neutralizing antibodies representative of the general population. The ability to dose a gen...