New data for Roche’s Hemlibra reinforce safety profile in people with haemophilia A

• Final analysis from the phase IIIb STASEY study, including data from 193 people with haemophilia A, further support the benefit/risk profile of Hemlibra, with no new safety signals identified¹
• STASEY is one of the largest open-label studies primarily assessing safety and tolerability of a medicine for people with haemophilia A with factor VIII inhibitors
• Hemlibra also continued to demonstrate effective bleed control with a high proportion of participants (82.6%) achieving zero treated bleeds¹
  

Basel, 19 July 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from the final analysis of the phase IIIb STASEY study, which confirm the favourable safety profile of Hemlibra® (emicizumab), consistent with the phase III HAVEN clinical programme.^1,2,3,4 In the analysis, no new safety signals were identified with longer-term Hemlibra treatment in adults and adolescents with haemophilia A with inhibitors to factor VIII, the clotting protein that is missing or defective in people with haemophilia A. The data were presented at the virtual International Society on Thrombosis and Haemostasis (ISTH) 2021 Congress, 17-21 July 2021.

“As the treatment landscape evolves, determining the long-term benefit/risk profile of medicines for people living with haemophilia A remains a top priority for the community,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “These results provide further confidence in Hemlibra’s favourable safety profile in people with haemophilia A with factor VIII inhibitors, who have historically faced significant treatment challenges.”

Nearly one in three people with haemophilia A develop factor VIII inhibitors, antibodies that bind to and block the efficacy of replacement factor VIII.⁵ People with haemophilia A with inhibitors are at greater risk of more frequent bleeding, including life-threatening bleeds, and may face greater challenges in their day-to-day lives than people with haemophilia A who do not have inhibitors.⁶ Hemlibra has been approved in more than 100 countries worldwide for the treatment of people with haemophilia A with factor VIII inhibitors.

The final analysis of the STASEY study included data from 193 people with haemophilia A with factor VIII inhibitors, who received Hemlibra prophylaxis once-weekly for up to two years (median treatment duration of 103.1 weeks).¹ The analysis did not show any new cases of thrombotic microangiopathy or serious thrombotic events (adverse events [AEs] that have been observed in people with bleeding disorders) related to Hemlibra.¹ The most common AEs occurring in 10% or more of people in the STASEY study were joint pain (arthralgia; 17.1%), common cold symptoms (nasopharyngitis; 15.5%), headache (15.0%), injection site reaction (ISR; 11.4%) and fever (pyrexia; 10.9%). Thirty-five (18.1%) people reported a Hemlibra-related AE, with ISRs being the most common (9.8%).¹

In addition, the STASEY study reinforced that Hemlibra is associated with a low incidence of anti-drug antibody (ADA) development. Ten participants (5.2%) tested positive for ADAs, five (2.6%) of whom were classified as having ADAs that were neutralising in vitro.¹ In all ten participants, ADA development did not affect the efficacy or safety of Hemlibra; none of the participants had ADAs that resulted in a decrease in Hemlibra plasma concentration, and none of the ADA-positive participants experienced a treated bleed. In addition, the ADAs disappeared over time, as all study participants tested negative for ADAs at their last visit.¹

Hemlibra also continued to demonstrate effective bleed control in the STASEY study, with 82.6% of participants experiencing no bleeding episodes that required treatment. Annualised bleeding rates were consistent with previously reported observations from the pivotal HAVEN studies.^1,2,3,4

Hemlibra is approved to treat people with haemophilia A with factor VIII inhibitors in more than 100 countries worldwide and people with haemophilia A without factor VIII inhibitors in more than 80 countries worldwide, including the US, EU and Japan. Hemlibra has been studied in one of the largest clinical trial programmes in haemophilia A with and without factor VIII inhibitors, including eight phase III studies.

About Hemlibra® (emicizumab)
Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins involved in 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, every two weeks or every four weeks (after an initial once-weekly dose for the first four weeks). Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech. It is marketed in the United States by Genentech as Hemlibra (emicizumab-kxwh), with kxwh as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration.

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 900,000 people worldwide,^5,7 approximately 35-39% of whom have a severe form of the disorder.⁷ 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.⁵ 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.⁸ A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies.⁹ Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII,¹⁰ making it difficult, if not impossible, to obtain a level of factor VIII sufficient to control bleeding.

About Roche in haematology
Roche has been developing medicines for people with malignant and non-malignant blood diseases for over 20 years; our experience and knowledge in this therapeutic area runs deep. Today, we are investing more than ever in our effort to bring innovative treatment options to patients across a wide range of haematologic diseases. Our approved medicines include MabThera®/Rituxan® (rituximab), Gazyva®/Gazyvaro® (obinutuzumab), Polivy® (polatuzumab vedotin), Venclexta®/Venclyxto® (venetoclax) in collaboration with AbbVie, and Hemlibra® (emicizumab). Our pipeline of investigational haematology medicines includes T-cell engaging bispecific antibodies, glofitamab and mosunetuzumab, targeting both CD20 and CD3, and cevostamab, targeting FcRH5 and CD3; Tecentriq® (atezolizumab), a monoclonal antibody designed to bind with PD-L1; and crovalimab, an anti-C5 antibody engineered to optimise complement inhibition. Our scientific expertise, combined with the breadth of our portfolio and pipeline, also provides a unique opportunity to develop combination regimens that aim to improve the lives of patients even further.

About Roche
Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics, as well as growing capabilities in the area of data-driven medical insights help Roche deliver truly personalised healthcare. Roche is working with partners across the healthcare sector to provide the best care for each person.

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. In recent years, Roche has invested in genomic profiling and real-world data partnerships and has become an industry-leading partner for medical insights.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty 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. Moreover, for the twelfth consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 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 www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References
[1] Jiménez-Yuste J, et al. Final Analysis of the STASEY Trial: A Single-arm, Multicenter, Open-label, Phase III Clinical Trial Evaluating the Safety and Tolerability of Emicizumab Prophylaxis in Persons with Hemophilia A (PwHA) with factor (F)VIII Inhibitors. Presented at: International Society on Thrombosis and Haemostasis (ISTH) Congress; 2021 Jul 17-21; Philadelphia, PA, USA. Abstract PB0521.
[2] Oldenburg J, et al. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017; 377:809-818.
[3] Young G, et al. Emicizumab prophylaxis provides flexible and effective bleed control in children with hemophilia A with inhibitors: results from the HAVEN 2 study. Blood. 2018; 132 (Supplement 1): 632.
[4] Pipe S, et al. Emicizumab subcutaneous dosing every 4 weeks is safe and efficacious in the control of bleeding in persons with haemophilia A with and without inhibitors: Results from the phase 3 HAVEN 4 study. Presented at: WFH World Congress; 2018 May 20-24; Glasgow, Scotland, UK. Abstract M-LBMED01-005 (854).
[5] Srivastava, A, Santagostino, E, Dougall, A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020: 26 (Suppl 6): 1‐158.
[6] Carcao M, Goudemand J., Inhibitors in hemophilia: a primer. 5th ed. Montreal: World Federation of Hemophilia; 2018
[7] Iorio A et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males. Ann Intern Med. 2019 Oct 15;171(8):540-546.
[8] Franchini M, et al. Haemophilia A in the third millennium. Blood Rev. 2013; 179-84.
[9] Gomez K, et al. Key issues in inhibitor management in patients with haemophilia. Blood Transfus. 2014; 12:s319-s329.
[10] Whelan SF, et al. Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of haemophilia A patients. Blood. 2013; 121:1039-48.

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