Investor Update

Basel, 28 July 2016

Roche’s emicizumab continued to show promising safety and efficacy profile in long-term study in people with severe haemophilia A

  • Results from Phase I/II follow-up study presented at the World Federation of Hemophilia 2016 World Congress

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced yesterday new data for investigational haemophilia medicine emicizumab (ACE910) at the World Federation of Hemophilia 2016 World Congress. The long-term follow-up data from a Phase I/II study showed that emicizumab given once-weekly by injection under the skin (subcutaneously), resulted in promising safety and prophylactic efficacy for people with severe haemophilia A, regardless of the presence of factor VIII inhibitors. Results showed that, after a median follow-up of 32.6, 27.0 and 21.4 months for the patients treated with emicizumab at 0.3, 1 and 3 mg/kg/week respectively, patients experienced a continued decrease in the annualised bleeding rate (ABR), irrespective of their inhibitor status and previous treatment regimen.

“We’re excited to see that with longer follow-up data, the safety and efficacy profile of emicizumab has been maintained,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “These results build on previous findings that were recently published in the New England Journal of Medicine, and support the potential benefit that emicizumab may offer to people with haemophilia A, regardless of inhibitor status.”

Emicizumab treatment continued to demonstrate a reduction in median ABR, resulting in patients with severe haemophilia A achieving an ABR of 1.4, 0.2 and 0 for the emicizumab 0.3, 1 and 3 mg/kg/week treatment groups respectively. This equated to a reduction in ABR of greater than 95% in each group. The safety profile of emicizumab was consistent with that previously observed in the Phase I study. The results of this longer follow-up confirm data previously presented at the International Society on Thrombosis and Haemostasis Congress in June 2015. Data from the first 12 week period of treatment, originally presented during the annual meeting of the American Society of Hematology in 2014, were recently published in the New England Journal of Medicine1.

About the emicizumab Phase I /II trial


About haemophilia A

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 worldwide2,3, approximately 50-60% of whom have a severe form of the disorder4. 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.

Currently available treatments for haemophilia A can be time-consuming due to the need for frequent intravenous administrations to prevent bleeding episodes2. A further complication of treatment is the development of ‘inhibitors’ to factor VIII replacement therapies5. Inhibitors are antibodies developed by the body’s immune system that attack and destroy the replacement factor VIII6. The development of inhibitors is a serious complication of current haemophilia A treatment7 and further options are needed for those who develop them.

About emicizumab (ACE910)

Emicizumab is a bispecific antibody engineered to bind both factors IXa and X, replacing the function of the missing factor VIII to improve clotting function and prevent spontaneous bleeding. Emicizumab can be administered by an injection of a ready-to-use solution under the skin (subcutaneous) once a week. Future trials will seek to explore less frequent dosing schedules.

The development programme for emicizumab is assessing its potential to help overcome some of the clinical challenges faced in haemophilia care, such as 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 Roche.

About Roche in haematology

For more than 20 years, Roche has been developing medicines that redefine treatment in haematology. Today, we are investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. In addition to approved medicines MabThera®/Rituxan® (rituximab), Gazyva®/Gazyvaro® (obinutuzumab) and in collaboration with AbbVie, Venclexta® (venetoclax), Roche’s pipeline of investigational haematology medicines includes Tecentriq® (atezolizumab), an anti-CD79b antibody drug conjugate (polatuzumab vedotin/RG7596) and a small molecule antagonist of MDM2 (idasanutlin/RG7388). Roche is a pioneer in the development of monoclonal antibodies (mAb) and has the largest portfolio of approved mAb medicines for cancer. Roche’s dedication to developing novel molecules in haematology expands beyond oncology, bringing our significant mAb expertise to the treatment of people with haemophilia A with the development of the investigational medicine emicizumab (ACE910).

About Roche

Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. Twenty-nine medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry seven years in a row by the Dow Jones Sustainability Indices.

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2015 employed more than 91,700 people worldwide. In 2015, Roche invested CHF 9.3 billion in R&D and posted sales of CHF 48.1 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit

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1 Shima M, et al. Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med 2016; 374:2044-2053.

2 WFH. Guidelines for the management of hemophilia. 2012. Last accessed 21 October 2015:

3 Berntorp E, Shapiro AD. Modern haemophilia care. The Lancet 2012; 370:1447-1456.

4 Marder VJ, et al. Hemostasis and Thrombosis. Basic Principles and Clinical Practice. 6th Edition, 2013. Milwakee, Wisconsin. Lippincott Williams and Wilkin.

5 Gomez K, et al. Key issues in inhibitor management in patients with haemophilia. Blood Transfus. 2014; 12:s319–s329.

6 Whelan SF, et al. Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of hemophilia A patients. Blood 2013; 121:1039–48.

7 Astermark J. Overview of Inhibitors. Semin Hematol 2006; 43(suppl 4):S3-S7.