Stable Hemostatic Correction and Improved Hemophilia-Related Quality of Life: Final Analysis from the Pivotal Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec
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

Sustained FIX Activity for Up to 18 Months

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).

Similar FIX Activity (%) for Subjects With and Without Pre-existing NAbs to AAV5

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.

Consistent Safety Profile

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%).

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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