PB0323 - Preliminary Analysis of ATHN 16: Real-World Safety of Eptacog Beta

Sunday, June 25, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Tammuella E. Singleton, MD

Chief Science Officer
American Thrombosis and Hemostasis Network
New Orleans, Louisiana, United States

Co-Author(s)

Suchitra Acharya, MD

Director Hemostasis and Thrombosis Center
Cohen Children’s Medical Center, Northwell Health
New Hyde Park, New York, United States
SA

SANJAY AHUJA

Director - Hemophilia Program
University Hospitals Health System, Cleveland, OH
Cleveland, Ohio, United States
LA

Lauren Amos

Associate Professor of Pediatrics
Children's Mercy Hospital, Kansas City, MO, USA
Kansas City, Missouri, United States
DB

DANIEL BONZO

VP and Global Head of Biometry
LFB
Frammingham, Massachusetts, United States
AE

Ashley Eason

Pediatric Hematology/Oncology
Willett Children's Hemophilia Treatment Center at Memorial Health
Savannah, Georgia, United States
Miguel Escobar, MD

Professor of Medicine and Pediatrics
University of Texas Health Science Center of Houston, McGovern Medical School, Gulf States Hemophilia and Thrombophilia Center, Houston, Texas
Houston, Texas, United States
CK

Christine Knoll

Director Hemophilia Treatment Center
Phoenix Childrens
Phoenix, Arizona, United States
PK

Philip Kuriakose

HTC Director
Henry Ford Health System Bleeding and Thrombosis Treatment Center
Detroit, Michigan, United States
EL

Emmanuelle Lagrue

Clinical Development and Medical Affairs Lead
LFB
Courtabeouf, Champagne-Ardenne, France
Michael Recht, MD, PhD, MBA (he/him/his)

Chief Medical and Scientific Officer
National Hemophilia Foundation
New York, New York, United States
SS

Spencer Sullivan

Chief Executive Officer, Hematology
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States
Doris V. Quon, MD, PhD (she/her/hers)

Medical Director, Orthopaedic Hemophilia Treatment Center
Luskin Orthopaedic Institute for Children
Los Angeles, California, United States
MR

Mark T. Reding

Professor and Director
Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, MN, USA
Minneapolis, Minnesota, United States

Background: Eptacog beta (rFVIIa-jncw) received regulatory approval in the U.S. in 2020 for the treatment of bleeding events (BEs) in persons with hemophilia A/B with inhibitors (PwHABI), 12 years of age and older. The ATHN 16 study was designed by the American Thrombosis and Hemostasis Network (ATHN) and the investigators to collect real-world evidence on the safety of eptacog beta.

Aims: To evaluate the safety of eptacog beta when used to treat BEs in participants with HAB with inhibitors with or without prophylactic treatment.

Methods: ATHN 16 (NCT04647227) is a phase IV, multi-center, open-label safety study enrolling PwHABI, who are either on long-term prophylaxis (e.g., emicizumab) or episodic treatment. ATHN 16 received central IRB approval.

After informed consent is obtained, each participant is provided nine (9) 75 µg/kg doses of eptacog beta to be administered as needed for bleed treatment. BEs are treated by the participant or by study staff at the time of an event. Actual dose administered (75 mcg/kg or 225 mcg/kg) is determined at the discretion of the treating physician.
The safety of eptacog beta is evaluated based on events included in the European Haemophilia Safety Surveillance (EUHASS) protocol. There are no pre-specified efficacy endpoints.

Results: Between August 2021 and October 2022, eleven subjects have been enrolled, ten of whom are on prophylactic emicizumab. Twenty-eight bleeding events have been treated (57% spontaneous, 36% traumatic, 8% other (surgery, activity/exercise) in four of the eleven enrolled participants. One adverse event (AE), unrelated to study product, was reported and resolved. One serious adverse event (SAE), unrelated to study product was reported and resolved.

Conclusion(s): To date, the safety profile of eptacog beta, in the ATHN 16 participants treated with eptacog beta, is favorable and consistent with previous reports.