More positive Phase III results for emicizumab in hemophilia A

Data from HAVEN 1, a Phase III study evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) in adults and adolescents with hemophilia A with inhibitors, have been published in The New England Journal of Medicine (NEJM).

The drug is under development by Swiss pharma giant Roche (ROG: SIX) and its majority-owned Japanese affiliate Chugai Pharmaceutical (TYO: 4519).

The primary endpoint showed a clinically-meaningful and statistically-significant reduction in treated bleeds of 87% (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% (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 as well as 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 Hemostasis (ISTH) Congress today.

“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,” said Sandra Horning, Roche’s chief medical officer and head of global product development, at Roche. “Based on the bleed reduction shown in the HAVEN 1 and HAVEN 2 studies, we believe emicizumab has the potential to make a meaningful difference for people with haemophilia A with inhibitors, while also reducing the burden of managing the condition with a subcutaneous, once-weekly administration,” Dr Horning noted.