Results of two phase III trials of Hemlibra in haemophilia A show effectiveness of therapy without inhibitors.- Chugai Pharma.

Chugai Pharmaceutical announced that full results from HAVEN 3 study (NCT02847637) and HAVEN 4 study (NCT03020160) evaluating Hemlibra (emicizumab) are being presented. HAVEN 3 study is conducted in people with hemophilia A without inhibitors, and HAVEN 4 study is conducted in people with hemophilia A with or without inhibitors.

The results from HAVEN 3 study demonstrate that Hemlibra can reduce the bleeding risk in people with hemophilia A without inhibitors. The intra-patient comparison in this study also shows a statistically significant reduction in bleeding by Hemlibra prophylaxis compared to factor VIII therapy. Hemlibra achieved a statistically significant reduction in treated bleeds by 96% (Wald test) with once-weekly prophylaxis of Hemlibra (Arm A) and 97% with once every two weeks prophylaxis respectively, compared with no prophylaxis control arm (Arm C). In addition, 55.6% of patients receiving once-weekly prophylaxis of Hemlibra (Arm A) experienced zero treated bleeds and 60% of patients receiving once every two weeks prophylaxis of Hemlibra (Arm B) compared to 0% of those not receiving prophylaxis treatment (Arm C).

In addition, HAVEN 4 data indicates that regardless of the presence of inhibitors, Hemlibra administration once every four weeks can reduce the risk of bleeding in people with hemophilia A compared with existing treatments. Patients receiving Hemlibra prophylaxis in Expansion cohort had a median annualized bleeding rate (ABR) for treated bleeds of 0.0 (IQR: 0.0; 2.1). And 56.1% of patients in Expansion cohort achieved zero treated bleeds and 90.2% of patients experienced three or fewer treated bleeds. Data are being presented at the World Federation of Hemophilia 2018 World Congress.