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.
