Business
uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-existing Antibodies to AAV5 Vector
~ Data Show No Clinically Significant Correlation Between Pre-Existing NAbs and FIX Activity ~ ~ Further Supports Potential for AAV5 Gene Therapies to be
About this update from Uniqure N.v.
[{"type":"text","content":"~ Data Show No Clinically Significant Correlation Between Pre-Existing NAbs and FIX Activity ~ ~ Further Supports Potential for AAV5 Gene Therapies to be Viable Treatments in Nearly All Patients -- ~ Data Presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting ~ LEXINGTON, Mass. and AMSTERDAM, May 13, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, yesterday presented 26-week clinical data from the pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec showing clinical benefit in hemophilia B patients with pre-existing neutralizing antibodies (NAbs) to AAV5, and no relationship between AAV5 NAbs and the tolerability of the therapy. The data were featured on Wednesday, May 12, 2021 at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in an oral presentation by Michael Recht, M.D., Ph.D., M.B.A., professor of pediatrics, division of hematology and oncology at the Oregon Health & Science University School of Medicine. These data were previously presented at a medical meeting late last year. “The ASGCT presentation highlights these initial HOPE-B data that demonstrate the successful treatment with an AAV5 gene therapy of patients with pre-existing NAbs,” stated Ricardo Dolmetsch, Ph.D., president of Research and Development at uniQure. “Patients in the trial who may not have been eligible for other gene therapies because of pre-existing neutralizing antibodies have achieved similar results with etranacogene dezaparvovec compared to those who did not have pre-existing NAbs. We believe this distinguishes etranacogene dezaparvovec as the only hemophilia gene therapy shown in a clinical trial to have the potential to treat nearly all patients, regardless of NAb levels in the generally prevalent range.” Patients in the Phase III HOPE-B clinical study were initially enrolled into a prospective, observational lead-in period of at least six months during which bleeding events and FIX replacement therapy usage were monitored. All patients required prophylactic routine FIX replacement prior to entering the clinical trial, and patients were not excluded from the trial based on pre-existing NAbs to AAV5. Fifty-four participants were dosed and completed 26 weeks of follow-up...