Chugai Pharmaceutical Co., Ltd. announced that latest data from an ongoing Japanese phase I/II study (ACE002JP) of emicizumab was presented at the World Federation of Hemophilia 2016 (WFH 2016) World Congress in Orlando, Florida, United States. Emicizumab is a bispecific antibody for subcutaneous injection under development for hemophilia A.
ACE002JP is an extension study of the patient part of a phase I study (ACE001JP), which was conducted to investigate safety and exploratory prophylactic efficacy profiles of emicizumab in Japanese hemophilia A patients both with and without FVIII inhibitors. The latest data analysis continued to show a promising profile of once-weekly subcutaneous injection of emicizumab in terms of safety and prophylactic efficacy, regardless of the presence of factor VIII inhibitors. The mean follow-up period were 32.6, 27.0 and 21.4 months in the 0.3, 1 and 3 mg/kg groups respectively.
“This new long-term follow-up data supports emicizumab’s continued prophylactic efficacy in hemophilia A patients both with and without inhibitors,” said Chugai’s vice president, head of project & lifecycle management unit, Dr. Yasushi Ito. “We anticipate that emicizumab will bring innovation to the current treatment for hemophilia A and deliver significant value to patients and their families.”
Emicizumab was designated as a Breakthrough Therapy by the US Food and Drug Administration in September 2015. Currently, two phase III global studies in adolescents/adults and children with hemophilia A who acquired FVIII inhibitors are ongoing respectively in collaboration with Roche, Chugai’s strategic alliance partner. A phase III global study in patients without FVIII inhibitors is also planned to start later this year. Furthermore, a phase III global study investigating administration once every four weeks is also planned.
The data of the patient part of ACE001JP was published in The New England Journal of Medicine in May 2016.
Chugai Pharmaceutical is one of Japan’s leading research-based pharmaceutical companies with strengths in biotechnology products.