Genentech has reported positive results from its HAVEN 3 study after meeting the trial’s primary endpoint and key secondary endpoints.
HAVEN 3 is a Phase lll trial investigating efficacy, safety and pharmacokinetics of Hemlibra (emicizumab-kxwh) prophylaxis against no prophylaxis (episodic/on-demand factor VIII treatment) to treat people with haemophilia A without factor VIII inhibitors.
The randomised, multicentre, open-label trial enrolled 152 patients with haemophilia A aged 12 or older, who were previously treated with factor VIII therapy either on-demand or for prophylaxis.
The patients were randomised in a 2:2:1 ratio to receive a subcutaneous injection of Hemlibra every week or every two weeks for up to 24 weeks.
The primary endpoint of the trial was the number of bleeds from baseline to up to 24 weeks.
The trial’s secondary endpoints featured a reduction in the number of bleeds, number of joint bleeds, and number of target joint bleeds, as well as quality of life, adverse events (AEs), among others.
The latest data from the HAVEN 3 study revealed that Hemlibra prophylaxis given subcutaneously every week or every two weeks significantly reduced treated bleeds by 96% and 97%, respectively, compared to no prophylaxis.
The data also demonstrated that 55.6% of people treated with Hemlibra every week and 60% of people treated with Hemlibra every two weeks experienced zero treated bleeds, compared to 0% of people who received no prophylaxis.
No unexpected or serious AEs associated with Hemlibra were reported in the HAVEN 3 study.
The most common AEs were similar to the ones found in previous studies.
Genentech Global Product Development head and chief medical officer Sandra Horning said: “Given the challenges many people face managing their haemophilia, we believe Hemlibra could make a meaningful difference, and we are working with health authorities to hopefully make this treatment available to people with haemophilia A without factor VIII inhibitors as soon as possible.”
Hemlibra has been formulated by Chugai Pharmaceutical and is being co-developed globally by a partnership of Chugai, Roche and Genentech.