information: Presentation of results at a congress
Announcement: presentation of results at the 26th International Society on Thrombosis and Haemostasis (ISTH) Meeting
Company: Roche (Switzerland)
Product: emicizumab (ACE910 - anti-factor IXa x anti-factor X humanized bispecific antibody)
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
area: Rare diseases - Genetic diseases - Hematological diseases
Country: Costa Rica, Germany, Italy, Japan, South Africa, Spain, Turkey, UK, USA
details: HAVEN 2 is a single-arm study that will enroll children (less than 12 years of age) and adolescents (12 to 17 years of age) with hemophilia A and FVIII inhibitors who are currently receiving treatment with bypassing agents. Participants will receive weekly subcutaneous doses of emicizumab for a designated period of 52 weeks. All participants will continue to receive standard of care/background treatment with their usual episodic bypassing agent therapy to treat breakthrough bleeds as needed. (NCT02795767)
- • On June 26, 2017, Chugai Pharmaceutical and Genentech announced results from the interim analysis of HAVEN 2 study. The data will be presented on July 10 at the upcoming 26th International Society on Thrombosis and Haemostasis (ISTH) Meeting in Berlin, Germany (July 8 to 13). The interim analysis was conducted with 19 children younger than 12 years of age with haemophilia A with inhibitors who require treatment with BPAs. Interim results are consistent with the positive results from the HAVEN 1 study. After a median observation time of 12 weeks, the study showed that only one of 19 children receiving emicizumab reported a treated bleed. There were no reported joint or muscle bleeds. An intra-patient comparison (n=8) in patients who were previously enrolled in the NIS showed that all patients experienced a 100 percent reduction in treated bleeds following treatment with emicizumab (previous ABR ranged from 0 to 34.24); this included seven children who had received prior BPA prophylaxis and one who had received prior on-demand BPA. The data also indicate that the same dose of emicizumab is appropriate for children as for adults and adolescents, based on the levels of emicizumab in the blood (pharmacokinetics) of the children compared with the level of emicizumab in the blood of adults and adolescents. The most common AEs with emicizumab in the HAVEN 2 study were mild injection site reactions and common cold symptoms (nasopharyngitis).
- • On April 17, 2017, Roche announced interim results from the phase III HAVEN 2 study evaluating emicizumab prophylaxis in children less than 12 years of age with haemophilia A and inhibitors to factor VIII. At this interim analysis after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time. These findings are consistent with results from the phase III HAVEN 1 study in adults and adolescents (12 years of age or older) with haemophilia A and inhibitors to factor VIII, in which emicizumab prophylaxis showed a statistically significant and clinically meaningful reduction in the number of bleeds over time compared to no prophylaxis, as well as compared to prior prophylaxis with bypassing agents. The most common adverse events with emicizumab in the HAVEN 2 study were injection site reactions and nasopharyngitis.
- HAVEN 2 is the second phase III study in the emicizumab clinical development programme to report results. Data from both HAVEN 1 and the interim data from HAVEN 2 studies will be presented at an upcoming medical meeting and submitted to health authorities for approval consideration.
- Two additional phase III studies of emicizumab are ongoing:
- • HAVEN 3, evaluating emicizumab prophylaxis dosed once weekly or once every other week in people 12 years of age or older with haemophilia A without inhibitors to factor VIII.
- • HAVEN 4, evaluating emicizumab prophylaxis dosed every four weeks in people 12 years of age or older with haemophilia A with or without inhibitors to factor VIII.
- • On June 7, 2016, a Phase 3 trial sponsored by Roche was published on the NIH website ClinicalTrials.gov for emicizumab and is currently recruiting participants.