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.

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%).