Steven W. Pipe, MD
Highlights From the ISTH 2021 Congress

Evolution of AAV Vector Gene Therapy Is Ongoing in Hemophilia. Will the Unique Features of BAY 2599023 Address the Outstanding Needs?

S.W. Pipe1, C. Hay2, J. Sheehan3, T. Lissitchkov4, M. Coppens5, H. Eichler6, S. Weigmann7, F. Ferrante8

1University of Michigan, Ann Arbor, United States

2Manchester University Dept. of Haematology, Manchester, United Kingdom

3University of Wisconsin–Madison, Madison, United States

4National Specialized Hospital for Active Treatment of Haematologic Diseases, Sofia, Bulgaria

5Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands

6Institute of Clinical Haemostaseology and Transfusion Medicine, Saarland University, Homburg, Germany

7Bayer, Wuppertal, Germany

8Bayer, Basel, Switzerland

Key Data Points

Phase 1/2 Study Design

Inclusion criteria, cohort dosing, and study endpoints for the ongoing phase 1/2 BAY2599023 dose-finding study. Males with severe hemophilia A, no known FVIII inhibitor history, an AAVhu37 neutralizing antibody titer ≤ 5, and > 150 exposure days to FVIII products were enrolled sequentially into three dose cohorts to receive a single intravenous infusion of BAY2599023, with at least two patients per dose level (patients to be enrolled in Cohort 4 will receive 4 × 1013 gene copies/kg).

Factor FVIII Expression Following Infusion of BAY2599023

FVIII expression levels following transgene infusion with the first 3 doses in a total of 8 patients. At the data cut off, May 21, 2021, chromogenic B-domain-deleted FVIII levels were available for a period ranging from 12 weeks for Patient 8 to 100 weeks for Patient 2. A dose response was observed from cohort 1 to cohort 3, with sustained FVIII levels achieved over a time of up to > 23 months. None of the patients reported spontaneous bleeds or other bleeds requiring treatment, once protective FVIII levels of > 11 IU/dL were achieved.

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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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