Roche and UK-based Chugai Pharmaceutical have reported positive data from the Phase III HAVEN 1 clinical trial of emicizumab in patients suffering from haemophilia A.
Emicizumab is a bispecific monoclonal antibody being developed to combine IXa and X factors that are needed for natural coagulation cascade activation and restoration of blood clotting.
Created by Chugai, the investigational candidate is being co-developed by the firm with Roche and Genentech.
The HAVEN 1 trial met the primary endpoint with a clinically meaningful and statistically significant decrease of 87% in bleed rate compared to on-demand bypassing agents (BPAs).
The randomised, multi-centre, open-label HAVEN 1 trial evaluated the efficacy, safety, and pharmacokinetics of once-weekly subcutaneous emicizumab prophylaxis in 109 adult and adolescent subjects.
Based on the results, it was found that all the 12 secondary endpoints of the trial were positive with a 79% reduced bleed rate during an intra-patient analysis.
Following a median observation time of 31 weeks, results further showed no bleeds with emicizumab in a larger proportion of patients.
Roche head of global product development and chief medical officer Sandra Horning said: “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.”
In addition, the firms reported the Phase III HAVEN 2 trial’s interim results were found to be consistent with the results from HAVEN 1.
HAVEN 2 evaluated the efficacy, safety, and pharmacokinetics of emicizumab in 19 patients under 12 years old.
During the single-arm, multi-centre, open-label trial, an intra-patient comparison revealed a 100% decrease in treated bleeds with once-weekly subcutaneous emicizumab.
Roche has submitted both HAVEN 1 and HAVEN 2 findings to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for approval.