Roche’s Hemlibra has hit key targets in a Phase III trial assessing its effectiveness in preventing bleeds in adults and adolescents with haemophilia A without inhibitors to factor VIII.

Hemlibra is a bispecific factor IXa- and factor X-directed antibody, designed to bring together proteins required to activate the natural coagulation cascade and restore the blood clotting process for haemophilia A patients. The therapy is used prophylactically, and can be administered subcutaneously.

Data from the Phase III HAVEN 3 study showed a statistically significant and clinically meaningful reduction in the number of treated bleeds over time in people receiving Hemlibra (emicizumab) prophylaxis every week compared to those receiving no prophylaxis.

The study also met key secondary endpoints, including a statistically significant and clinically meaningful reduction in the number of treated bleeds over time with Hemlibra prophylaxis dosed every two weeks compared to no prophylaxis.

Also of note, once-weekly Hemlibra prophylaxis was found to be superior to factor VIII prophylaxis, Roche noted.

“Hemlibra is the first product to show superior efficacy to factor VIII prophylaxis. These results in people with haemophilia A without inhibitors represent the next step forward in our clinical trial programme, which includes the positive HAVEN 1 and interim HAVEN 2 data in people with inhibitors,” said Sandra Horning, MD, Roche’s chief medical officer and head of Global Product Development.

“It is well established that prophylaxis is the preferred approach for treatment of haemophilia A, but this can require frequent intravenous infusions, and some patients on prophylaxis can still experience bleeds, while others prefer on-demand treatment,” said Johnny Mahlangu, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa.

“Given its potential to be dosed through subcutaneous injection only once weekly or every other week, Hemlibra may provide a further effective prophylactic treatment option for more people with haemophilia A and help alleviate some of the administration burden associated with current treatment.”

Hemlibra was approved in the US last week.