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    Poster Networking

    Hemophilia and Rare Bleeding Disorders

    Poster Networking Session

    PB0186 - Results from BeneGene-1 Study: Prospective collection of bleeding rate in hemophilia B patients prior to phase 3 study (BeneGene-2) of fidanacogene elaparvovec

    Sunday, June 25, 2023
    18:30 – 19:30 ET
    Room: Exhibition

      Presenting Author(s)

    • Davide Matino, MD (he/him/his)

      Assistant Professor
      Department of Medicine, Division of Hematology, McMaster University, Hamilton, ON
      Hamilton, Ontario, Canada

      • Co-Author(s)

    • HA

      Hazzaa Alzahrani

      Consultant
      Adult Haematology, King Faisal Specialist Hospital and Research Centre Riyadh, Saudi Arabia
      Riyadh, Ar Riyad, Saudi Arabia

    • Adam Cuker, MD, MS (he/him/his)

      Associate Professor
      Department of Medicine and Department of Pathology & Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
      Philadelphia, Pennsylvania, United States

    • JA

      Jan Astermark

      Professor, Senior Consultant
      Department of Hematology Oncology and Radiation Physics, Skane University Hospital, and Institution of Translational Medicine, Lund University, Malmö, Sweden
      Malmö, Skane Lan, Sweden

    • LF

      Laurent Frenzel

      Medical Doctor
      Department of Haematology, Institut Necker
      Paris, Ile-de-France, France

    • OK

      Olga Katsarou

      Director
      Blood Transfusion Centre, National Reference Centre for Congenital Bleeding Disorders, Laiko General Hospital
      Athens, Attiki, Greece

    • KK

      Kaan Kavakli

      Medical Doctor
      Department of Hematology, Ege University Children’s Hospital, Izmir, Turkey
      Ege, Izmir, Turkey

    • Robert Klamroth, MD, PhD (he/him/his)

      Priv. Doz. Dr. med.
      Vivantes Klinikum im Friedrichshain, Clinic for Internal Medicine Angiology and Haemostaseology
      Berlin, Berlin, Germany

    • AS

      Amy Shapiro

      Medical Director
      Indiana Hemophilia & Thrombosis Center, Indianapolis, IN, USA
      Indianapolis, Indiana, United States

    • JW

      Jiaan-Der Wang

      Medical Doctor
      Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital
      Taichung, Taichung, Taiwan (Republic of China)

    • JF

      Joanne Fuiman

      Director, Study Clinician
      Pfizer Inc.
      Collegeville, Pennsylvania, United States

    • JM

      John McKay

      Senior Director
      Pfizer Inc.
      Groton, Connecticut, United States

    • PS

      Pengling Sun

      Statistician
      Pfizer Inc.
      Cambridge, Massachusetts, United States

    • Jeremy Rupon, MD,PhD

      Hematology Clinical Program Lead
      Pfizer Inc.
      Collegeville, Pennsylvania, United States

    Background: There has been great advancement in hemophilia B adeno-associated virus (AAV) mediated gene transfer. Fidanacogene elaparvovec is an AAV-based gene therapy for the treatment of hemophilia B that is currently under investigation in a Phase 3 dosing study. The primary endpoint of this study is to demonstrate non-inferiority to regular prophylaxis in total (treated and untreated) annualized bleeding rate (ABR).

    Aims: To gather baseline ABR data while on prophylaxis as a part of routine care, a prospective non-interventional lead-in study was initiated.

    Methods: Patients with hemophilia B (FIX<=2%) on regular prophylaxis who tested negative for neutralizing antibodies (nAb) to the rh74var-capsid were eligible to enroll in the study. Subjects recorded infusions and bleeding events in an electronic diary for at least 6 months prior to entry in the phase 3 study.

    Results: 316 hemophilia B patients were screened and 102 were enrolled and entered the study. nAb was positive in 193 participants and in part the cause of over 90% of screen failures. At the time of data cut, 59 had completed at least 6 months of follow-up, 40 had less than 6 months, and 3 patients had withdrawn. The mean ABRTotal was 4.41 and the annualized infusion rate (AIR) was 61.63 Additional efficacy data in Table 1. No safety signals emerged.

    Conclusion(s): The ABR and AIR in this globally diverse study are similar to values previously reported in clinical studies of FIX prophylaxis. While factor prophylaxis was well tolerated with no emerging safety signals, an ABR of 4.41 illustrates the limitations of current standard of care prophylaxis. The ABR in the subset of this population that enrolled in the Phase 3 dosing study of fidanacogene elaparvovec will be used as the comparator for the primary endpoint of non-inferiority in total ABR post-gene therapy infusion.