Three Chugai Researchers Behind Hemlibra Discovery Receive Lasker-DeBakey Clinical Medical Research Award
核心洞察
Three Chugai scientists who led the creation of Hemlibra (emicizumab) for hemophilia A (搜索) have received the Lasker-DeBakey Clinical Medical Research Award, one of the world's most prestigious scientific honors.
The award recognizes the unconventional concept of replacing coagulation Factor VIII (搜索)'s function with a bispecific antibody, enabling subcutaneous administration and sustained bleed prevention regardless of inhibitor status.
An integrated analysis of phase III HAVEN 1–4 studies showed an annualized bleeding rate of 1.4 events per year, with 82.4% of participants experiencing zero treated bleeds by Weeks 121–144.
Chugai Pharmaceutical Co., Ltd. announced on September 9, 2026 that Dr. Kunihiro Hattori (former Senior Fellow), Dr. Takehisa Kitazawa (Deputy Head of Research Division), and Dr. Tomoyuki Igawa (Head of Research Division), who led the creation of Hemlibra (emicizumab) for hemophilia A (搜索), have been awarded the Lasker-DeBakey Clinical Medical Research Award. The honor recognizes the unconventional concept of replacing the function of coagulation Factor VIII (搜索) with a bispecific antibody, an approach that transformed the treatment paradigm for hemophilia A.
The award marks the second time Japanese researchers have received the honor in the clinical medical research category since the Lasker Awards were established in 1945, and the first time three Japanese researchers have been recognized simultaneously in this category.
A Novel "Additive" Approach to Antibody Therapeutics
Hemophilia A (搜索) is caused by a deficiency of coagulation Factor VIII (搜索), one of the proteins required for normal blood clotting. When activated, Factor VIII serves as a bridge that facilitates the activation of Factor X by activated Factor IX on the surface of activated platelets. Rather than replacing Factor VIII itself, Hattori and colleagues pursued a novel concept of substituting its function with an antibody, developing a bispecific antibody that bridges activated Factor IX and Factor X at the appropriate orientation and spatial position on the surface of activated platelets.
Conventional antibody therapeutics typically exert their effects through a "subtractive" approach, inhibiting the activity of disease-related molecules or cells, or eliminating target cells through immune-mediated mechanisms. In contrast, emicizumab became the world's first antibody medicine to embody an "additive" approach, restoring a missing biological function by endowing the antibody itself with a function normally performed by a different protein. It is also the world's first recombinant full-length IgG bispecific antibody therapeutic designed to bind two different targets.
During its creation, Chugai developed and applied its proprietary antibody engineering technology, ART-Ig, which enabled commercial-scale manufacturing. The collaboration with the Department of Pediatrics at Nara Medical University, which has conducted collaborative research with Chugai since 2003, generated achievements including the establishment of coagulation assay systems for evaluating emicizumab and significant contributions to early clinical studies in Japan.
Clinical Evidence and Global Reach
Emicizumab was licensed out to Roche, and Chugai worked together with Roche and Genentech to advance global clinical development and regulatory submissions. Following its approval in the United States in 2017 and in Japan and Europe in 2018, Hemlibra is now approved in more than 120 countries and regions worldwide.
An integrated analysis of the phase III HAVEN 1–4 studies demonstrated that, regardless of the presence or absence of factor VIII (搜索) inhibitors, disease severity, or age, the primary endpoint of the annualized bleeding rate (ABR) for treated bleeds over 24-week intervals throughout the study was 1.4 events per year. Moreover, 70.8% of participants (277/391) experienced zero treated bleeds during Weeks 1–24, and this proportion increased over time to 82.4% (140/170) during Weeks 121–144. No new safety signals were identified, and the most common adverse event was injection-site reactions (27.8%).
In addition to providing sustained bleed prevention regardless of the presence or absence of factor VIII (搜索) inhibitors, emicizumab offers a new treatment option by not inducing new factor VIII inhibitors and improving convenience through subcutaneous administration and extended dosing intervals. To date, it has been used by more than 30,000 people with hemophilia A (搜索) worldwide (cumulative global total as of June 30, 2026).
Addressing an Unmet Medical Need
Congenital hemophilia A (搜索) predominantly affects males and occurs in approximately one in 5,000 male births, with occurrence in females extremely rare because it is an X-linked recessive disorder. Since around 2000, prophylactic treatment with regular replacement of the missing factor VIII (搜索) has become widely adopted, but this approach requires intravenous infusions as frequently as once to several times per week. People with hemophilia A who developed factor VIII inhibitors faced significant treatment challenges due to the limited treatment options available.
Dr. Osamu Okuda, Chugai's President and CEO, commented, "I am truly delighted that this original concept, inspired by our desire to reduce the burden on people with hemophilia A (搜索) and their families, together with the innovative drug discovery technologies that brought it to life, has been recognized through this prestigious award." He added that emicizumab has reduced treatment burden through subcutaneous administration once every one to four weeks, "thereby bringing a new everyday life to people with hemophilia A and their families."
Dr. Hattori, who originated the concept of a bispecific antibody based on expertise in blood coagulation and antibodies, stated, "I believe our continuing mission is to ensure that this treatment reaches patients around the world who can benefit from it." Dr. Kitazawa, who led pharmacology and biology research and demonstrated factor VIII (搜索)-mimetic activity, expressed gratitude to "the people with hemophilia A (搜索) and their families who participated in the clinical studies." Dr. Igawa, who led the design of emicizumab and established technologies for efficient manufacturing of bispecific antibodies, said the desire to lessen patient burden "has motivated us to overcome many scientific challenges."