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uniQure Presents Late-Breaking Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B at the 62nd Annual Meeting of the American Society of Hematology
~ First primary endpoint in study met with mean Factor IX activity of 37% of normal at 26 weeks ~ ~ Data show 91% reduction in reported bleeds requiring
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[{"type":"text","content":"~ First primary endpoint in study met with mean Factor IX activity of 37% of normal at 26 weeks ~ ~ Data show 91% reduction in reported bleeds requiring treatment, with 87% of patients reporting no such bleeds during the 26 weeks after dosing ~ ~ Durable increases in Factor IX activity maintained for up to 18 months irrespective of pre-existing neutralizing antibodies ~ ~ Etranacogene dezaparvovec was well-tolerated with no treatment-related serious adverse events ~ ~ uniQure to host investor webcast today at 5:00 p.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Dec. 08, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the late-breaking presentation of initial 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 will be presented today in an oral session at the virtual 62nd Annual Meeting of the American Society of Hematology (ASH). The oral presentation slides are available in the Investors section of uniQure’s website under Investor Downloads. “These initial data on etranacogene dezaparvovec are encouraging, because thus far in these patients, severe hemophilia B appears to have been transformed into a functionally curative state following administration of this one-time gene therapy, providing cessation in bleeding for a majority of patients and no need for ongoing, chronic replacement therapy,” stated Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan, and the principal investigator of the HOPE-B pivotal trial. “Importantly, these data also show that those patients in the trial who may not have been eligible for other gene therapies because they had pre-existing neutralizing antibodies (NAbs) have achieved results with etranacogene dezaparvovec that ...