Steven W. Pipe, MD
Highlights From the ISTH 2021 Congress

52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: Data From the Phase 3 HOPE-B Gene Therapy Trial

S.W Pipe1, F.W. Leebeek2, M. Recht3, N.S Key4, S. Lattimore3, G. Castaman5, E.K Sawyer6,7, S. Verweij6,7, V. Colletta6,7, D. Cooper6,7, R. Dolmetsch6,7, W. Miesbach8, HOPE-B Investigators

1University of Michigan, Ann Arbor, United States

2Erasmus University Medical Center, Rotterdam, Netherlands

3Oregon Health & Science University, Portland, United States

4University of North Carolina, Chapel Hill, United States

5Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy

6uniQure Inc, Lexington, United States

7uniQure BV, Amsterdam, Netherlands

8University Hospital Frankfurt, Frankfurt, Germany

Key Data Points

FIX Activity Over 52 Weeks Post-infusion

Mean and median FIX activity for all participants in the HOPE-B trial for 52 weeks after dosing with 2×1013 gc/kg (N = 54). Following treatment, FIX activity increased rapidly to a mean of 39.0 IU/dL at Week 26 and 41.5 IU/dL at Week 52.

Treatment-related Adverse Events

Most common treatment related-adverse events (possibly related or related) with an incidence of ≥ 5% in the safety population during the post-treatment period. There were 408 adverse events among 53 participants, of which 91 (among 39 participants) were considered treatment-related adverse events (TRAEs).

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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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