information: Interim results
Announcement: interim results
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 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.