– Emicizumab prophylaxis reduced the number of bleeds in children with hemophilia A and inhibitors to factor VIII –
– Results build upon data for emicizumab in adults and adolescents with hemophilia A and inhibitors to factor VIII –
“Managing hemophilia A with inhibitors to factor VIII is especially challenging for children and their caregivers, because bleeding is difficult to control and current treatments require frequent intravenous infusions,” said
HAVEN 2 is the second Phase III study in the emicizumab clinical development program to report results. Data from both HAVEN 1 and the interim data from HAVEN 2 studies will be presented at an upcoming medical meeting and submitted to health authorities for approval consideration.
Two additional Phase III studies of emicizumab are ongoing:
- HAVEN 3, evaluating emicizumab prophylaxis dosed once weekly or once every other week in people 12 years of age or older with hemophilia A without inhibitors to factor VIII.
- HAVEN 4, evaluating emicizumab prophylaxis dosed every four weeks in people 12 years of age or older with hemophilia A with or without inhibitors to factor VIII.
The development program for emicizumab reflects Genentech’s commitment to help address clinical unmet needs in the treatment of hemophilia A. As part of this commitment,
About the HAVEN 2 study
HAVEN 2 (NCT02795767) is a single-arm, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of once weekly subcutaneous administration of emicizumab. The interim analysis after a median of 12 weeks of treatment included 19 children less than 12 years of age with hemophilia A and inhibitors to factor VIII, who require treatment with bypassing agents. The objectives of the study are to evaluate the number of bleeds over time with emicizumab prophylaxis, safety, pharmacokinetics, health-related quality of life (HRQoL) and proxy HRQoL with aspects of caregiver burden. The study will enroll a total of 60 children for its final analysis planned after 52 weeks of treatment with emicizumab.
About the HAVEN 1 study
HAVEN 1 (NCT02622321) is a randomized, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of emicizumab prophylaxis versus no prophylaxis in people with hemophilia A and inhibitors to factor VIII. The study included 109 patients with hemophilia A (12 years of age or older) with inhibitors to factor VIII, who were previously treated with episodic or prophylactic bypassing agents. Patients previously treated with episodic bypassing agents were randomized in a 2:1 fashion to receive emicizumab prophylaxis (Arm A) or no prophylaxis (Arm B). Patients previously treated prophylactically with bypassing agents received emicizumab prophylaxis (Arm C).
Episodic treatment of breakthrough bleeds with bypassing agents was allowed per protocol. The primary endpoint of the study is the number of bleeds over time with emicizumab prophylaxis (Arm A) versus no prophylaxis (Arm B). Secondary endpoints include all bleed rate, joint bleed rate, spontaneous bleed rate, target joint bleed rate, HRQoL/ health status, intra-patient comparison to bleed rate on their prior prophylaxis regimen with bypassing agents (Arm C) and safety. As previously reported, the study showed a statistically significant reduction in the number of bleeds over time in people treated with emicizumab prophylaxis compared to those receiving no prophylactic treatment. The study also met all secondary endpoints, including a statistically significant reduction in the number of bleeds over time with emicizumab prophylaxis treatment in an intra-patient comparison in people who had received prior bypassing agent prophylaxis treatment. The most common adverse event with emicizumab was injection site reactions, consistent with prior studies.
About emicizumab (ACE910)
Emicizumab is an investigational bispecific monoclonal antibody designed to bring together factors IXa and X, proteins required to activate the natural coagulation cascade and restore the blood clotting process. Emicizumab can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once weekly. Emicizumab is being evaluated in pivotal Phase III studies in people 12 years of age and older, both with and without inhibitors to factor VIII, and in children under 12 years of age with factor VIII inhibitors. Additional trials are exploring less frequent dosing schedules. The clinical development program is assessing the safety and efficacy of emicizumab and its potential to help overcome current clinical challenges: the short-lasting effects of existing treatments, the development of factor VIII inhibitors and the need for frequent venous access. Emicizumab was created by
About hemophilia A
Hemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Hemophilia affects around 20,000 people in
These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damage. In addition to impacting a person’s quality of life, these bleeds can be life threatening if they go into vital organs, such as the brain. A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies. Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII, making it difficult, if not impossible to obtain a level of factor VIII sufficient to control bleeding.
Founded 40 years ago,