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:

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
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
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

REVIEW ABSTRACT

Steven W. Pipe, MD¹, Frank W.G. Leebeek², Michael Recht, MD, PhD, MBA³, Nigel S. Key⁴, Susan Lattimore³, Giancarlo Castaman⁵, David Cooper^6,7, Stephanie Verweij^6,7, Ricardo Dolmetsch^6,7, 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

REVIEW ABSTRACT

Steven W. Pipe, MD¹, Frank W.G. Leebeek², Michael Recht, MD, PhD, MBA³, Nigel S. Key⁴, Susan Lattimore³, Giancarlo Castaman⁵, Michiel Coppens^6,7, David Cooper^8,9, Robert Gut^8,9, Sergio Slawka^8,9, Stephanie Verweij^8,9, Ricardo Dolmetsch^8,9, Yanyan Li¹⁰, Paul E. Monahan¹⁰ and Wolfgang A. Miesbach¹¹

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)

REVIEW ABSTRACT

Wolfgang A. Miesbach¹, Michael Recht, MD, PhD, MBA², Nigel S. Key³, Krupa Sivamurthy⁴, Paul E. Monahan^4* 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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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

Abstracts Discussed in this Video

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