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Genentech’s HEMLIBRA (emicizumab-kxwh) Every Four Weeks Controlled Bleeds in Phase III Study in Hemophilia A

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Thursday, December 07, 2017

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SOUTH SAN FRANCISCO, CA

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced positive interim results from the Phase III HAVEN 4 study evaluating HEMLIBRA® (emicizumab-kxwh) prophylaxis dosed once every four weeks in adults and adolescents (12 years of age or older) with hemophilia A with and without inhibitors to factor VIII. At this interim analysis after a median of 17 weeks of treatment, HEMLIBRA prophylaxis showed a clinically meaningful control of bleeding. These results are consistent with previous studies of HEMLIBRA dosed once weekly or every two weeks, including the pivotal studies in hemophilia A with inhibitors, HAVEN 1 in adults and adolescents and HAVEN 2 in children, as well as the Phase III HAVEN 3 study in adults and adolescents with hemophilia A without inhibitors. The most common adverse events with HEMLIBRA were injection site reactions, with no new safety signals observed. No thrombotic microangiopathy or thrombotic events occurred in this study.

"Current treatment regimens for hemophilia A can require frequent intravenous infusions. We are encouraged that HEMLIBRA prophylaxis administered by injection under the skin once every four weeks showed clinically meaningful bleed control in people with hemophilia A," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "Together with the findings from other Phase III studies, these interim results support the potential for HEMLIBRA to be dosed at different schedules, allowing patients to choose the option that's right for them."

Data from the HAVEN 4 study will be presented at an upcoming medical meeting and submitted to health authorities around the world for approval consideration. These results add to the growing body of evidence supporting that HEMLIBRA may benefit all people with hemophilia A regardless of inhibitor status, while providing flexible and less burdensome administration options.

About HAVEN 4 (NCT03020160)

HAVEN 4 is a single-arm, multicenter, 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 hemophilia A with or without inhibitors to factor VIII who were previously treated with either factor VIII or bypassing agents, on-demand or as prophylaxis. The study was conducted in two parts: a PK run-in; and an expansion cohort. All patients in the PK run-in (n=7) were previously treated on-demand, and received subcutaneous HEMLIBRA at 6 mg/kg to fully characterize the PK profile after a single dose during four weeks, followed by 6 mg/kg every four weeks for at least 24 weeks. Patients in the expansion cohort (n=41) received subcutaneous HEMLIBRA prophylaxis at 3 mg/kg/wk for four weeks, followed by 6 mg/kg every four weeks for at least 24 weeks. Episodic treatment of breakthrough bleeds with factor VIII therapy or bypassing agents, depending on a patient's inhibitor status, was allowed per study protocol.

About HEMLIBRA

HEMLIBRA 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 hemophilia A patients. 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. HEMLIBRA was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed by Chugai, Roche and Genentech.

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 the United States, with hemophilia A being the most common form and approximately 50-60 percent of people living with a severe form of the disorder.

People with hemophilia 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 hemophilia 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.

About Genentech in hemophilia

In 1984, Genentech scientists were the first to clone recombinant factor VIII in response to the contaminated hemophilia blood supply crisis of the early 1980s. For more than 20 years, Genentech has been developing medicines to bring innovative treatment options to people with diseases of the blood within oncology, and in hemophilia A. Genentech is committed to improving treatment and care in the hemophilia community by delivering meaningful science and clinical expertise. For more information visit http://www.gene.com/hemophilia .

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

Contact:

Genentech
1 DNA Way
South San Francisco, CA 94080

Phone: (650) 467-6800

Source: Genentech, Inc.
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