Roche details full data from late-stage studies of haemophilia A therapy Hemlibra

Roche on Monday reported full results from two Phase III studies of Hemlibra (emicizumab), with data from the HAVEN 3 trial showing that people with haemophilia A without factor VIII inhibitors who received prophylaxis with the therapy every week or every two weeks had a 96 percent and 97 percent reduction in treated bleeds, respectively, compared to those who received no prophylaxis. The results were presented at the World Federation of Hemophilia (WFH) world congress.

In November, Roche and partner Chugai Pharmaceutical announced that the HAVEN 3 study met its primary endpoint, with Hemlibra showing a significant and clinically meaningful reduction in the number of treated bleeds over time compared to those receiving no prophylaxis. "These new pivotal data show that Hemlibra controlled bleeds in people with haemophilia A, while offering the flexibility of less frequent subcutaneous dosing options," remarked Sandra Horning, Roche's head of global product development.

The HAVEN 3 trial included 152 patients with haemophilia A who were previously treated with factor VIII therapy either on-demand or for prophylaxis. Results from the study also showed that in a subset of patients who previously received factor VIII prophylaxis, Hemlibra reduced bleeds by 68 percent compared to prior therapy. In addition, 55.6 percent of people treated with Hemlibra every week and 60 percent treated every two weeks experienced zero treated bleeds, compared to 0 percent of people treated with no prophylaxis.

Roche also presented full data at the WFH world congress from the Phase III HAVEN 4 study, with results showing that people with haemophilia A with or without factor VIII inhibitors who received Hemlibra prophylaxis every four weeks had a median annualised bleeding rate for treated bleeds of 0.0, with 56.1 percent experiencing zero treated bleeds and 90.2 percent experiencing three or fewer treated bleeds.

The trial included 48 patients with haemophilia A with or without factor VIII inhibitors who were previously treated with either factor VIII or bypassing agents, on-demand or as prophylaxis. The company said the HAVEN 4 "results demonstrate that Hemlibra administration every four weeks can provide clinically meaningful control of bleeding in people with haemophilia A with or without factor VIII inhibitors."

Meanwhile, commenting on the HAVEN 4 results, Jefferies analysts said the study showed there was a potential to treat both inhibitor and non-inhibitor patients on a once-monthly basis, rather than the current once-weekly injection. "This sets Hemlibra up to become the new standard of care for haemophilia A, which we view as a $5 billion peak sales opportunity," the analysts added.

Hemlibra gained FDA approval last year for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with haemophilia A with factor VIII inhibitors. Last month, the agency granted the drug breakthrough therapy designation for people with haemophilia A without factor VIII inhibitors, based on data from the HAVEN 3 study. Hemlibra has also been authorised in other countries, including Europe and Japan, for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors.

Earlier this year, Roche's Genentech unit confirmed that five deaths have occurred in patients receiving Hemlibra, with two of the deaths this year. The company noted that despite having "limited information" surrounding the circumstances, the deaths have all been deemed unrelated to Hemlibra by the investigator or treating physician. For related analysis, see ViewPoints: Investors willing to overlook Hemlibra deaths – for now.

For more insights, read ViewPoints: Roche’s Hemlibra set to test ‘stickiness’ of haemophilia market.

To read more Top Story articles, click here.