Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial
Highlights From the 62nd ASH Annual Meeting and Exposition

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial

Annette von Drygalski, MD, Pharm D1, Adam Giermasz, MD, PhD2, Giancarlo Castaman, MD3, Nigel S. Key, MD4,5, Susan U. Lattimore, R.N.6, Frank W.G. Leebeek, MD, PhD7, Wolfgang A. Miesbach, MD8, Michael Recht, MD, PhD9, Esteban Gomez, MD10, Robert Gut, MD, PhD11, and Steven W. Pipe, MD12

1Department of Medicine, Division of Hematology/Oncology, UC San Diego Health, San Diego, CA

2University of California Davis, Sacramento, CA

3Azienda Ospedaliera Universitaria Careggi, Florence, Italy

4University of North Carolina at Chapel Hill, Chapel Hill, NC

5Division of Hematology, University of North Carolina at Chapel Hill, Chapel Hill, NC

6Oregon Health & Science University, Portland, OR

7Erasmus University Medical Center, Rotterdam, Netherlands

8Hemophilia Centre, University Hospital Frankfurt, Frankfurt, Germany

9Oregon Health and Science University, Portland, OR

10Phoenix Children's Hospital, Phoenix, AZ

11uniQure Inc, Lexington, MA

12University of Michigan, Ann Arbor, MI

Key Data Points

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The phase 2B clinical trial for AMT-061 (etranacogene dezaparvovec) was designed as an open-label, single arm study. A single dose of AAV5-Padua hFIX was infused in 3 participants at a dose of 2x1013 gc/kg. The primary endpoint was FIX activity at 6 weeks, and duration of follow-up was 5 years. Pre-existing neutralizing antibodies to AAV5 was not an exclusion criterion.

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The primary endpoint of FIX activity ≥ 5% at 6 weeks post-infusion was achieved in all 3 participants. The mean FIX activity at 2 years was 44.2%, and FIX activity was stable from year 1 to year 2. Over 2 years, 2 or 3 participants had no bleeds or use of FIX concentrate. The third participant had 2 infusions of FIX replacement therapy for 1 suspected and 1 confirmed bleed.

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