Genentech Announces Positive Interim Results for Emicizumab in Phase III Study of Children with Hemophilia A

Monday, April 17, 2017

Genentech, a member of the Roche Group, 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.

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