Updates on Three Ongoing Clinical Trials on Adults with Severe Hemophilia B
Highlights From the 64th ASH Annual Meeting and Exposition

Updates on Three Ongoing Clinical Trials on Adults with Severe Hemophilia B

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

Use the tabs below to review additional information about the abstracts discussed in this video.

Abstracts:

  1. Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals With and Without Adeno-associated Virus Serotype 5 Neutralizing Antibodies (Titers < 1:700) With Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B
  2. Adults With Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection After 24 Months’ Follow-up
  3. Durability of Factor IX Activity and Bleeding Rate in People With Severe or Moderately Severe Hemophilia B After 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and After 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)

Abstracts Discussed in this Video

Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals With and Without Adeno-associated Virus Serotype 5 Neutralizing Antibodies (Titers < 1:700) With Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B
Steven W. Pipe, MD1, Frank W.G. Leebeek2, Michael Recht, MD, PhD, MBA3, Nigel S. Key4, Susan Lattimore3, Giancarlo Castaman5, David Cooper6,7, Stephanie Verweij6,7, Ricardo Dolmetsch6,7, Jacqueline Tarrant8, Yanyan Li8, Paul E. Monahan8 and Wolfgang A. Miesbach9
1University of Michigan, Ann Arbor, MI
2Erasmus Medical Center, 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 BV, Amsterdam, Netherlands
7uniQure Inc., Lexington, MA
8CSL Behring, King of Prussia, PA
9University Hospital Frankfurt, Frankfurt, Germany
Summary of FIX activity<sup>a</sup> (%) by baseline NAb status (FAS)
Values for FIX activity in patients from HOPE-B, the phase 3 trial for etranacogene dezaparvovec, based on AAV antibody (NAb) titer. Of the 54 participants, 33 were NAb- (NAb < 1:7) and 21 were NAb+. One NAb+ patient had a titer of 3,212 and 20/21 had titers < 1:700 (median 56.9). At 24 months, mean FIX activity was similar for the NAb+ cohort (32.98 IU/dL) and the NAb- cohort (38.55 IU/dL).
Adults With Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection After 24 Months’ Follow-up

Steven W. Pipe, MD1, Frank W.G. Leebeek2, Michael Recht, MD, PhD, MBA3, Nigel S. Key4, Susan Lattimore3, Giancarlo Castaman5, Michiel Coppens6,7, David Cooper8,9, Robert Gut8,9, Sergio Slawka8,9, Stephanie Verweij8,9, Ricardo Dolmetsch8,9, Yanyan Li10, Paul E. Monahan10 and Wolfgang A. Miesbach11

1University of Michigan, Ann Arbor, MI
2Erasmus Medical Center, 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
6Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
7Amsterdam UMC location, University of Amsterdam, Vascular Medicine, Amsterdam, Netherlands
8uniQure BV, Amsterdam, Netherlands
9uniQure Inc., Lexington, MA
10CSL Behring, King of Prussia, PA
11University Hospital Frankfurt, Frankfurt, Germany
FIX Activity After Infusion of Etranacogene Dezaparvovec

Mean and median FIX levels out to 24 months for the phase 3 HOPE-B trial for etranacogene dezaparvovec. The mean value was 39.0 IU/dL (SD ±18.7; min-max 8.2-9.7) at month 6 and 36.7 IU/dL (±19.0; 4.7-99.2) at month 24.

Durability of Factor IX Activity and Bleeding Rate in People With Severe or Moderately Severe Hemophilia B After 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and After 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)

Wolfgang A. Miesbach1, Michael Recht, MD, PhD, MBA2, Nigel S. Key3, Krupa Sivamurthy4, Paul E. Monahan4* and Steven W. Pipe, MD5

1University Hospital Frankfurt, Frankfurt, Germany
2Oregon Health & Science University, Portland, OR
3University of North Carolina, Chapel Hill, NC
4CSL Behring, King of Prussia, PA
5University of Michigan, Ann Arbor, MI
Mean uncontaminated FIX activity levels and ABR after etranacogene dezaparvovec

aPhase 2b had no lead-in period; phase 3 had a ≥6 month lead-in period, during which participants received FIX prophylaxis.

Phase 2b and 3 mean FIX and mean ABR efficacy outcomes up to 24 months post-infusion for etranacogene dezaparvovec.

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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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