Highlights From the ASGCT 25th Annual Meeting
Stable Hemostatic Correction and Improved Hemophilia-Related Quality of Life: Final Analysis from the Pivotal Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec
Presented by: Steven W. Pipe, MD, University of Michigan, Ann Arbor, Michigan, United States
Steven W. Pipe1, Frank W. G. Leebeek2, Michael Recht3, Nigel S. Key4, Giancarlo Castaman5, David Cooper6, Robert Gut6, Ricardo Dolmetsch6, Yanyan Li7, Paul E. Monahan7, Wolfgang Miesbach8
1University of Michigan, Ann Arbor, MI
2Erasmus MC, University Medical Center, Rotterdam, Netherlands
3Oregon Health & Science University, Portland, OR
4University of North Carolina, Chapel Hill, NC
5Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy
6uniQure Inc., Lexington, MA
7CSL Behring, King of Prussia, PA
8University Hospital Frankfurt, Frankfurt, Germany
Key Data Points
One-stage FIX activity for moderately severe (N=10) and severe (N=44) patients infused with etranacogene dezaparvovec (AAV5-Padua hFIX) at a dose of 2x1013 gc/kg in the phase 3 HOPE-B trial. This analysis included patients with and without AAV5 neutralizing antibodies (NAbs).
One-state FIX activity at baseline and at 18 months post-infusion for patients with and without pre-existing AAV5 NAbs. With antibodies was defined as having a titer of greater than the limit of detection (LOD). Without antibodies was defined as having a titer of less than or equal to LOD.
The safety profile for etranacogene dezaparvovec at 18 months was consistent with previously presented data. The most frequent treatment-related adverse events (TRAE) occurring in more than 10% of patients were ALT increase (17%), headache (15%), influenza-like illness (13%), and infusion-related reactions (13%).
Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Johnny Mahlangu, BSc, MBBCh, MMed, FCPath
Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Wolfgang A. Miesbach, MD, PhD
Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Declan Noone
Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Maria Elisa Mancuso, MD, PhD