Long-term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption, and Safety of Hemophilia B Gene Therapy
Highlights From the 65th ASH Annual Meeting and Exposition

Long-term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption, and Safety of Hemophilia B Gene Therapy: Results From the HOPE-B Trial 3 Years After Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients With Severe or Moderately Severe Hemophilia B

Presented by: Steven W. Pipe, MD, University of Michigan, Ann Arbor, Michigan, United States

Steven Pipe, MD1, Paul van der Valk, MD2, Peter Verhamme3, Peter Kampmann4, Frank W.G. Leebeek, MD, PhD5, Michiel Coppens, MD6,7, Karina Meijer, MD, PhD8, Priyanka Raheja9, Nigel S. Key, MD10, Nathan Visweshwar, MD11, Guy Young, MD12, Richard S. Lemons, MD, PhD13, Robert Klamroth, MD14, Wolfgang Miesbach, MD15, Jan Astermark16,17, Niamh O'Connell18, Rashid Saeed Kazmi, MBBS, FRCP, FRCPath19, Nicholas Galante, PhD20, Sandra LeQuellec, PhD20, Paul Monahan20 and Cedric R. Hermans, MD, PhD21

1 University of Michigan, Ann Arbor, MI
2 Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands
3 University Hospitals Leuven, Belgium, Leuven, Belgium
4 Rigshospitalet, Copenhagen, Denmark
5 Department of Haematology, Erasmus University Medical Center-Erasmus MC, Rotterdam, Rotterdam, Netherlands
6 Amsterdam University Medical Centers, Department of Vascular Medicine, University of Amsterdam, Amsterdam, Netherlands
7 Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
8 Department of Hematology, University Medical Center Groningen, Groningen, Groningen, Netherlands
9 Royal London Haemophilia Centre, Bart Health NHS trust, London, United Kingdom
10 UNC Blood Research Center, Department of Medicine, University of North Carolina, Chapel Hill, NC
11 University of South Florida, Tampa, FL
12 Keck School of Medicine, University of Southern California, Los Angeles, CA
13 University of Utah, Salt Lake City, UT
14 Internal Medicine, Vascular Medicine and Coagulation Disorders, Vivantes Clinic Friedrichshain, Berlin, Germany
15 Goethe University Hospital, Frankfurt, Germany
16 Department of Hematology, Oncology and Radiation Physics, Skane University Hospital, Malmo, Sweden
17 Department of Translational Medicine, Lund University, Malmö, Sweden
18 National Coagulation Centre, St James Hospital, Dublin, Dublin, Ireland
19 University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom
20 CSL Behring, King of Prussia, PA
21 Cliniques Universitaires Saint-Luc, Brussels, Belgium

Data Points

Graphic shows the analysis of annualized bleeding rates in the Phase 3 HOPE-B study in patients at lead in and 7 to 36 months following infusion with etranacogene dezaparvovec. There was a 64% reduction for all bleeding events, with significant reduction in FIX-treated bleeds, spontaneous bleeds, traumatic bleeds, and joint bleeds relative to lead-in.

Graphic shows the analysis of annualized bleeding rates in the Phase 3 HOPE-B study in patients at lead in and 7 to 36 months following infusion with etranacogene dezaparvovec. There was a 64% reduction for all bleeding events, with significant reduction in FIX-treated bleeds, spontaneous bleeds, traumatic bleeds, and joint bleeds relative to lead-in.

Graphic shows the analysis of endogenous FIX activity levels over 3 years in patients treated with etranacogene dezaparvovec. Over 3 years following infusion patients demonstrated stable FIX activity levels at each timepoint.

Graphic shows the analysis of endogenous FIX activity levels over 3 years in patients treated with etranacogene dezaparvovec. Over 3 years following infusion patients demonstrated stable FIX activity levels at each timepoint.

Graphic shows the analysis of treated bleeding events in the Phase 3 HOPE-B study over 36 months in patients treated with etranacogene dezaparvovec. The analysis on the left reveals a significant, 96% reduction in mean Factor IX consumption from lead-in to 3 years post-infusion. On the right, the analysis indicates that 94% of treated patients were free from continuous prophylactic FIX infusions through 3 years post-administration.

Graphic shows the analysis of treated bleeding events in the Phase 3 HOPE-B study over 36 months in patients treated with etranacogene dezaparvovec. The analysis on the left reveals a significant 96% reduction in mean Factor IX consumption from lead-in to 3 years post-infusion. On the right, the analysis indicates that 94% of treated patients were free from continuous prophylactic FIX infusions through 3 years post-administration.

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