Roche announced that phase III results for Hemlibra (emicizumab) will be presented for the first time during the World Federation of Hemophilia (WFH) 2018 World Congress from 20-24 May in Glasgow, Scotland. The late-breaking presentations include positive results from the pivotal HAVEN 3 study of Hemlibra dosed every week or every two weeks in people with haemophilia A without factor VIII inhibitors and the pivotal HAVEN 4 study of Hemlibra dosed every four weeks in people with haemophilia A with or without factor VIII inhibitors. These data support the promising potential of Hemlibra for all people with haemophilia A.

Data include results from HAVEN 3 study in people with haemophilia A without factor VIII inhibitors and HAVEN 4 study in people with haemophilia A with or without factor VIII inhibitors

Ongoing Hemlibra clinical development programme demonstrates commitment to advancing care for all people with haemophilia A

“We look forward to sharing these new results from the HAVEN 3 and HAVEN 4 studies, which demonstrate the potential of Hemlibra to redefine treatment expectations for people with haemophilia A with and without inhibitors to factor VIII,” said Sandra Horning, MD, Roche’s chief medical officer and head of global product development. “We will also be sharing real-world data that provides new insight into the impact of haemophilia A treatment on daily life, as part of our ongoing commitment to advancing management and care for the global haemophilia community.”

Data from the HAVEN 3 and HAVEN 4 studies will be presented for the first time in late-breaking oral presentations on Monday 21 May. The HAVEN 3 presentation will highlight new data on Hemlibra prophylaxis administered every week or every two weeks in people 12 years of age or older with haemophilia A without factor VIII inhibitors compared to no prophylaxis. The presentation will also include results from an intra-patient analysis comparing Hemlibra prophylaxis to prior treatment with factor VIII prophylaxis.

The US Food and Drug Administration (FDA) recently granted Breakthrough Therapy Designation for Hemlibra in people with haemophilia A without factor VIII inhibitors based on data from this study. The HAVEN 4 presentation will highlight primary data in people 12 years of age or older with haemophilia A with or without factor VIII inhibitors receiving Hemlibra prophylaxis every four weeks.

These presentations at WFH follow the announcement of positive top-line results from the HAVEN 3 study in November 2017 and positive top-line interim results from the HAVEN 4 study in December 2017. Data from both studies are being submitted to health authorities around the world for approval consideration.

Roche will also present real-world data from a non-interventional study in adults with haemophilia A without factor VIII inhibitors and children with haemophilia A with factor VIII inhibitors. These data on health-related quality of life and health status will provide insights into challenges of living with and managing haemophilia A for patients and caregivers.

HAVEN 3 is a randomised, multicentre, open-label, phase III study evaluating the efficacy, safety and pharmacokinetics of Hemlibra prophylaxis versus no prophylaxis (episodic/on-demand factor VIII treatment) in people with haemophilia A without factor VIII inhibitors. The study included 152 patients with haemophilia A (12 years of age or older) who were previously treated with factor VIII therapy either on-demand or for prophylaxis.

HAVEN 4 is a single-arm, multicentre, open-label, phase III study evaluating the efficacy, safety and pharmacokinetics (PK) of subcutaneous administration of Hemlibra dosed every four weeks. The study included 48 patients (12 years of age or older) 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.

Hemlibra (emicizumab) is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for people with haemophilia A. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once-weekly.

Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Haemophilia A affects around 320,000 people worldwide, approximately 50-60% of whom have a severe form of the disorder. People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their disorder, people with haemophilia A can bleed frequently, especially into their joints or muscles.

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. 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.