Data from Phase III HAVEN I study of subcutaneous emicizumab prophylaxis (preventative) treatment in adults and adolescents with hemophilia A with inhibitors is published in NEJM.- Genentech

Genentech announced that data from HAVEN 1, a Phase III study evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) in adults and adolescents with hemophilia A with inhibitors, were published in The New England Journal of Medicine (NEJM). The primary endpoint showed a clinically meaningful and statistically significant reduction in treated bleeds of 87 percent (risk rate [RR]=0.13, p<0.0001) with emicizumab prophylaxis compared with on-demand no prophylaxis episodic use only bypassing agents bpas. all 12 secondary endpoints were positive including a statistically significant reduction of 79 percent rr="0.21," p="0.0003)" in treated bleeds in a first-of-its-kind intra-patient analysis in a subset of patients comparing two prophylaxis regimens emicizumab and bpas. data from haven 1 and the interim analysis of the phase iii haven 2 study of emicizumab in children are being presented at the 26th international society on thrombosis and haemostasis isth congress .>

Further data from HAVEN 1 showed that, after a median observation time of 31 weeks, substantially more patients experienced zero bleeds with emicizumab prophylaxis than with on-demand BPAs across all bleed measurements, including zero treated bleeds (62.9 percent vs. 5.6 percent), zero treated spontaneous bleeds (68.6 percent vs. 11.1 percent), zero treated joint bleeds (85.7 percent vs. 50.0 percent), zero treated target joint bleeds (94.3 percent vs. 50.0 percent) and zero bleeds overall, which includes all treated and non-treated bleeds (37.1 percent vs. 5.6 percent). A clinically meaningful and statistically significant improvement in health-related quality of life (HRQoL) measured at 25 weeks, using two validated instruments (Haem-A-QoL and EQ-5D-5L), was also observed.

In an additional study arm (Arm C, n=49), patients who had previously received BPA prophylaxis were treated with emicizumab prophylaxis. A subset of patients in this arm (n=24) had previously participated in a non-interventional study (NIS), allowing for a first-of-its-kind intra-patient analysis comparing two prophylaxis regimens. This analysis showed a 79 percent (RR=0.21, p=0.0003) reduction in treated bleeds in patients receiving emicizumab compared with their prior BPA prophylaxis during the NIS. Data also showed that 70.8 percent of patients in this subset experienced zero treated bleeds with emicizumab prophylaxis, whereas only 12.5 percent of these patients had experienced zero bleeds with their prior BPA prophylaxis during the NIS.

See- "Emicizumab Prophylaxis in Hemophilia A with Inhibitors"-Johannes Oldenburg, M.D., Ph.D., Johnny N. Mahlangu, M.D., Benjamin Kim, M.D., Christophe Schmitt, Pharm.D., et al., 10 July 10, 2017DOI: 10.1056/NEJMoa1703068.

Comment: Nearly one in three people with hemophilia A develop inhibitors to standard factor VIII therapy, leaving them at greater risk of life-threatening bleeds and long-term joint damage.