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Ernest Beutler Lecture and Prize

Program: General Sessions
Monday, December 11, 2023: 1:30 PM-2:30 PM
Hall A (San Diego Convention Center)

Lecture Title:
Non-Replacement Therapy in Hemophilia Management: The Evolution of FVIII Mimetics from Basic Science to Clinical Translation

Robert A. Brodsky, MD, Johns Hopkins University
Brodsky: Alexion, AstraZeneca Rare Disease: Research Funding.
The development of novel therapies in the management of inherited bleeding disorders in the last few decades has been remarkable and unprecedented. Consequently, the standard of care in hemophilia A management is switching rapidly from clotting factor replacement therapies to non-replacement therapies. The shift started with the invention and subsequent clinical development of the subcutaneously administered FVIII mimetic, emicizumab. In this lecture, Dr. Kitazawa and Dr. Mahlangu will share the remarkable story of the development of emicizumab, a FVIII mimetic, from bench to bedside.

Dr. Kitazawa will describe the conception of the emicizumab molecule, its preclinical proof-of-concept validation, and its proposed mechanism of action. He will summarize the results of the early clinical development program. In his reflections, Dr. Kitazawa will share with us the trials and tribulations encountered during the development of emicizumab and how these obstacles were addressed.

In continuing the remarkable story of emicizumab development, Dr. Mahlangu will remind us of the unmet needs of patients with hemophilia A who are managed with replacement therapies. He will then take us through the safety and efficacy data of the multiple Phase 3 studies of emicizumab prophylaxis in non-inhibitor and inhibitor hemophilia A patients across all ages. He will summarize the real-world data from observational studies and registries and end his presentation with a personal perspective on the role of non-replacement therapies in the future management of patients with hemophilia A.

Takehisa Kitazawa, PhD, DVM

Chugai Pharmaceutical Co., Ltd., Yokohama, Kanagawa, Japan; Chugai Pharmaceutical, Totsuka-Ku, SHZ, Japan

Johnny Mahlangu, MBBCh, MMed

University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa

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