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.
Wolfgang Miesbach, MD, PhD, discloses links to Bayer, Biomarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Takeda/Shire, uniQure.