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Genentech’s Hemophilia Drug Demonstrates Positive Interim Results in Late-Stage Study in Children

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced interim results from the Phase III HAVEN 2 study evaluating emicizumab prophylaxis in children less than 12 years of age with hemophilia A and inhibitors to factor VIII. At this interim analysis after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time. These findings are consistent with results from the Phase III HAVEN 1 study in adults and adolescents (12 years of age or older) with hemophilia A and inhibitors to factor VIII, in which emicizumab prophylaxis showed a statistically significant and clinically meaningful reduction in the number of bleeds over time compared to no prophylaxis, as well as compared to prior prophylaxis with bypassing agents. The most common adverse events with emicizumab in the HAVEN 2 study were injection site reactions and nasopharyngitis (common cold symptoms).

“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 Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We are encouraged that once-weekly subcutaneous emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time in children and are pleased to share these results with the community as we join in celebrating World Hemophilia Day.”

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, Roche and Genentech are proud to support the World Federation of Hemophilia and the global bleeding disorders community as sponsors of World Hemophilia Day. To learn more about World Hemophilia Day and the World Federation of Hemophilia visit http://www.wfh.org/en/whd.

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 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. 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.

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 now in hemophilia A with our investigational medicine emicizumab. 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 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.

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