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Genentech Announces Positive HEMLIBRA Trial Results

May 23, 2018

Genentech recently announced results from a pair of phase III studies for HEMLIBRA®, a subcutaneously administered therapy currently being investigated for the treatment of patients with hemophilia A, without factor VIII (FVIII) inhibitors.

HEMLIBRA, which is co-developed by Genentech, Chugai and Roche, is already approved by the U.S. Food and Drug Administration for routine once-weekly prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors. The phase III HAVEN 3 and phase III HAVEN 4 studies are designed to evaluate the efficacy, safety and pharmacokinetics of the therapy when used prophylactically in adults and adolescents aged 12 years or older without FVIII inhibitors.

In the Phase III HAVEN 3 study, patients who received HEMLIBRA prophylaxis every week or every two weeks experienced a 96% and 97% reduction in treated bleeds, respectively, compared to those who received no prophylaxis. In addition, 55.6% of individuals treated with HEMLIBRA every week and 60% of those treated with HEMLIBRA every two weeks experienced zero treated bleeds, compared to 0% in patients not treated prophylactically. No unexpected or serious adverse events (AEs) were reported, with the most common AEs being injection site reactions, joint pain, common cold symptoms, headache, upper respiratory tract infection and influenza.

In the Phase III HAVEN 4 study, patients receiving HEMLIBRA prophylaxis every four weeks had a median annualized bleeding rate for treated bleeds of 0.0, with 56.1% of people experiencing zero treated bleeds and 90.2% experiencing three or fewer treated bleeds. According to a Genentech press release, the results show that HEMLIBRA administration every four weeks can provide a “clinically meaningful control of bleeding” in individuals with hemophilia A with or without FVIII inhibitors. In addition, no serious AEs were reported in HAVEN 4, with the most common being injection site reactions.

“These new pivotal data show that HEMLIBRA controlled bleeds in people with hemophilia A, while offering the flexibility of less frequent subcutaneous dosing options,” said Sandra Horning, MD, chief medical officer and head of Global Product Development. “With this data, we now have positive results from all four of our Phase III trials that reinforce the overall efficacy and safety of HEMLIBRA and its potential to improve care for all people with hemophilia A.”

Data from these studies were presented as late-breaking abstracts at the World Federation of Hemophilia 2018 World Congress, held May 20-24 in Glasgow, Scotland.

Source: Genentech press release dated May 23, 2018