Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: First Data From the Phase 3 Hope-B Gene Therapy Trial
Highlights From the 14th Annual Congress of EAHAD

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

Steven W Pipe, MD1, Michael Recht, MD, PhD2, Nigel S Key, MBChB3, Frank WG Leebeek, MD4, Giancarlo Castaman, MD5, Susan U Lattimore, RN6, Paul van der Valk, MD6, Kathelijne Peerlinck, MD, PhD7, Michiel Coppens, MD, PhD8, Niamh O'Connell, MB PhD9, K. John Pasi, MBChB, PhD10, Peter Kampmann, MD11, Karina Meijer, MD, PhD.12, Annette von Drygalski, MD, PharmD13, Guy Young, MD14, Cedric Hermans, MD15, Jan Astermark, M.D., PhD16, Robert Klamroth, MD, PhD17, Richard S Lemons, MD, PhD18, Nathan Visweshwar, MD19, Shelley Crary, MD20, Rashid Kazmi, MBBS21, Emily Symington, MBChB22, Miguel A Escobar, MD23, Esteban Gomez, MD24, Rebecca Kruse-Jarres, MD, MPH25, Adam Kotowski, MD26, Doris Quon, MD, PhD27, Michael Wang, MD28, Allison P Wheeler, MD29, Eileen K Sawyer, PhD30, Stephanie Verweij30, Valerie Colletta, MSc30, Naghmana Bajwa, MD, Robert Gut, MD, P.D30, Wolfgang Miesbach, MD, PhD31

1University of Michigan, Ann Arbor, MI, USA

2Oregon Health & Science University, Portland, OR, USA

3University of North Carolina, Chapel Hill, NC, USA

4Erasmus MC, University Medical Center, Rotterdam, The Netherlands

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

6Universitair Medisch Centrum Utrecht, Netherlands

7Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium

8Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands

9The National Coagulation Centre, St James’s Hospital, Dublin, Ireland

10Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, UK

11Rigshospitalet, Copenhagen, Denmark

12University Medical Center Groningen, Groningen, Netherlands

13University of California San Diego, La Jolla, CA, USA

14Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA

15Cliniques Universitaires Saint‐Luc, Université Catholique de Louvain, Brussels, Belgium

16Skåne University Hospital, Malmö

17Vivantes Klinikum im Friedrichshain, Berlin, Germany

18University of Utah, Salt Lake City, UT, USA

19University of South Florida, Tampa, FL, USA

20University of Arkansas for Medical Sciences, AR, USA

21University Hospital Southampton NHS Foundation Trust; Southampton, UK

22Cambridge University - Addenbrooke’s Hospital, Cambridge, UK

23University of Texas Health Science Center at Houston, Houston, TX, USA

24Phoenix Children’s Hospital, Phoenix USA

25Bloodworks Northwest, Seattle, WA

26WNY BloodCare, NY, USA

27Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center, Los Angeles, CA, USA

28University of Colorado Hemophilia and Thrombosis Center, Anschutz Medical Campus, Aurora, CO

29Vanderbilt University Medical Center, Nashville, TN, USA

30uniQure BV, Amsterdam, The Netherlands/ uniQure Inc. Lexington, MA, USA

31University Hospital Frankfurt, Frankfurt, Germany

Key Data Points

FIX Expression

This figure shows 1 of the 3 co-primary endpoints of the study, mean and median FIX activity for all participants at 26 weeks after dosing with 2×1013 gc/kg (N = 54). FIX expression was robust by 3 weeks post-dosing and stable by week 26, with a mean of 37.2%.


Because this study did not exclude participants with neutralizing antibodies (NAbs) to AAV, it was possible to evaluate results for correlation between NAbs and FIX activity. The vertical axis is the observed week 26 FIX activity of those who received a full dose of vector. Patients with undetectable NAbs were plotted at 7 on the horizontal axis (which was the threshold of the assay). The horizontal axis shows the titers of the other participants over a range up to 678. There was one nonresponding participant who had a titer > 3200, which is not shown on this graphic scale. There was no observed correlation of pre-existing NAbs with FIX activity up to a titer of 678.



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Supported by educational grants from BioMarin, CSL Behring, Pfizer and Spark Therapeutics

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