Assessing EtranaDez for Hemophilia B in HOPE-B: Wolfgang Miesbach, MD, PhD

“The HOPE-B trial was the first phase 3 study in hemophilia B and has the largest cohort of gene therapy for hemophilia B to report to date. It is also the only trial to include participants with neutralizing antibodies to AAV and this is really unique.”

Etranacogene dezaparvovec (EtranaDez, AMT-061; CSL Behring, UniQure) is superior to Factor IX (FIX) prophylactic therapy in reducing annualized bleeding rate (ABR) in patients with hemophilia B, according to results from the final analysis of the phase 3 HOPE-B study (NCT03569891).

Data from the analysis were presented at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting, February 2-4, 2022, by investigator Wolfgang Miesbach, MD, PhD, professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany. Miesbach and colleagues found that, after the 6-month lead-in period, adjusted ABR was reduced by 64% (P = .0002) and FIX-treated bleeds reduced by 77% (P <.0001) over months 7 to 18. Almost all participants (98%) treated with the full dose of EtranaDez were able to discontinue prophylaxis.

GeneTherapyLive spoke with Miesbachto learn more about the HOPE-B study of EtranaDez. He gave an overview of the study, discussing the positive data and outcomes.

REFERENCE

Final analysis of pivotal HOPE-B study demonstrates durable and sustained therapeutic effect of etranacogenedezaparvovec gene therapy in hemophilia B – Data presented at EAHAD 2022. News release. CSL Behring. February 4, 2022. https://www.cslbehring.com/newsroom/2022/hope-b-data-presented-at-eahad2022