Date: 2016-07-28
Type of
information: Presentation of results at a congress
phase: 1-2
Announcement: presentation of results at the World Federation of Hemophilia 2016 World Congress in Orlando
Company: Chugai Pharmaceutical (Japan)
Product: emicizumab (ACE910 - anti-factor IXa x anti-factor X humanized bispecific antibody)
Action
mechanism:
- bispecific antibody. Emicizumab is a bispecific antibody that mimics coagulation factor VIII. It is currently investigated as a therapy for people with hemophilia A. Emicizumab was designated as a Breakthrough Therapy by the FDA in September 2015.
Disease: hemophilia A
Therapeutic
area: Rare diseases - Genetic diseases - Hematological diseases
Country: Japan
Trial
details:
Latest
news:
- • On July 28, 2016, Chugai Pharmaceutical announced that latest data from an ongoing Japanese Phase I/II study (ACE002JP) of emicizumab was presented at the World Federation of Hemophilia 2016 World Congress in Orlando, Florida, United States. 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. The data of the patient part of ACE001JP was published in The New England Journal of Medicine in May 2016. 18 patients all experienced adverse events. Those adverse events were of mild or moderate intensity, except for 2 severe cases (appendicitis and mesenteric hematoma). No thromboembolic adverse events or clinically significant abnormality of coagulation tests were observed. 7 of 18 patients experienced local injection site reactions which were manageable. No neutralizing ADA (anti-drug antibodies) were observed. ABR (Annualized Bleeding Rate) remained low and 8 of 18 patients had experienced zero bleeds. Breakthrough bleeds were successfully treated with standard episodic treatment, FVIII products or bypassing agents.
Is
general: Yes
