HOPE-B clinical data shows clinical benefit of uniQure drug in hemophilia B patients

Netherlands-headquartered uniQure (Nasdaq: QURE) 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 Dr Michael Recht, 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, president of R&D 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.”