EHA 2021 | HOPE-B: etranacogene dezaparvovec in adult hemophilia B

Wolfgang Miesbach, MD, PhD, University Hospital Frankfurt, Frankfurt, Germany, gives an update on the Phase III HOPE-B trial (NCT03569891) investigating the efficacy and safety of etranacogene dezaparvovec in adults with hemophilia B. Etranacogene dezaparvovec consists of an adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized Padua variant human factor IX (FIX) gene with a liver-specific promoter. Prof. Miesbach highlights that this study has not excluded patients based on pre-existing neutralizing AAV5. Prof. Miesbach discusses the results of the trial at 26 weeks after a single dosage of etranacogene dezaparvovec, at which point 54 patients had been treated. 42.6% of patients had neutralizing antibodies to AAV5 at baseline. Following treatment, FIX activity increased to a mean of 37.2%. At 26 weeks, 96.3% of patients had successfully discontinued routine prophylaxis with 72.2% of patients reporting no bleeds in the first 26 weeks post-treatment. No correlation between pre-existing neutralizing antibodies and FIX activity was observed. No deaths and no treatment-related serious adverse events were reported. This interview took place at the virtual European Hematology Association (EHA) Congress 2021.