New follow-up phase III data reinforce the long-term benefit of Roche’s Hemlibra for people with haemophilia A

• With nearly three years of follow-up, Hemlibra maintained low treated bleed rates and was well tolerated in people with haemophilia A of all ages, with and without factor VIII inhibitors
• The proportion of participants who experienced zero treated bleeds increased over the course of the study period
• These data, presented at ASH 2020, build on results previously observed in the HAVEN studies, one of the largest pivotal clinical trial programmes in haemophilia A
  
  

Basel, 7 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from a new analysis of pooled, three-year follow-up data of 401 people with haemophilia A from the pivotal HAVEN 1-4 studies, which reinforce the long-term efficacy and safety profile of Hemlibra® (emicizumab).^[1] These data, from adults, adolescents and children with haemophilia A with and without factor VIII inhibitors, were presented at the all-virtual 62^nd American Society of Hematology (ASH) Annual Meeting and Exposition, 5-8 December 2020.

“The long-term benefit of Hemlibra, with a consistent safety profile and durably effective control of bleeding, underscores its potential to redefine the standard of care for people living with haemophilia A,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “As the first new class of medicine in nearly 20 years, Hemlibra continues to show a positive impact on disease burden and quality of life for people with haemophilia A, regardless of their inhibitor status or age.”

The analysis included pooled data from 401 people with haemophilia A with and without factor VIII inhibitors, from across the four pivotal HAVEN studies (HAVEN 1, n=113; HAVEN 2, n=88; HAVEN 3, n=152; HAVEN 4, n=48) with a median duration efficacy period of 120.4 weeks.^[1] Hemlibra maintained low treated bleed rates across the study period, with model based annualised bleed rates (ABR) remaining low throughout the evaluation period at 1.4 (95% CI: 1.1-1.7). Further, the proportion of participants who experienced zero treated bleeds (70.8-83.7%) increased with each consecutive 24-week period.^[1] In addition, with Hemlibra prophylaxis, 95.1% of target joints were resolved.^[1] Results showed that Hemlibra’s safety profile was consistent with previous observations and no new safety signals were observed after the longer follow-up.

Furthermore, the first interim analysis of the European Haemophilia Safety Surveillance (EUHASS) Database, also presented at the ASH Virtual Congress, suggests the safety profile of Hemlibra in the real-world setting is consistent with that seen in clinical trials, with no new or emerging safety signals. EUHASS is a large pharmacovigilance programme that monitors the safety of treatments for inherited bleeding disorders.^[2]

Data from the HAVEN studies supported Hemlibra approvals to treat people with haemophilia A with factor VIII inhibitors in more than 90 countries worldwide and for people without factor VIII inhibitors in more than 80 countries worldwide, including the US, EU and Japan. Since its initial approval more than three years ago, 8,200 people have received Hemlibra globally, and in the US, Hemlibra is now the most prescribed preventative (prophylactic) treatment for haemophilia A.

About the Hemlibra clinical development programme
Hemlibra has been studied in one of the largest pivotal clinical trial programmes in haemophilia A with and without factor VIII inhibitors, including eight phase III studies. The programme assesses the efficacy and safety of Hemlibra and its potential to help overcome current clinical challenges in the management of haemophilia A, including the short-lasting effects of existing treatments, the development of factor VIII inhibitors and the need for frequent venous access. Results from the HAVEN 1 and HAVEN 2 studies supported approvals of Hemlibra for people with haemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors. These data, as well as results from the HAVEN 3 and HAVEN 4 studies supported approvals of Hemlibra for people with haemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors. Additional studies, HAVEN 5, HAVEN 6, HAVEN 7 and STASEY, reflect Roche’s ongoing commitment to continuing to characterise the safety and efficacy of Hemlibra and to addressing unmet needs for people living with haemophilia A.

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 US 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 320,000 people worldwide,^[3,4] approximately 50-60% of whom have a severe form of the disorder.^[5] 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.^[3] 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.^[6] A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies.^[7] Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII,^[8] 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 both 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.

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

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 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.

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.

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 2019 employed about 98,000 people worldwide. In 2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF 61.5 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] Michael C, et al. Safety and Efficacy of Emicizumab in Persons with Hemophilia A With or Without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4) [Abstract #1800] Poster presented at: American Society Of Hematology (ASH) Annual Meeting; 5 – 8 December, 2020.
[2] Shang A, et al. Real-World Safety of Emicizumab: The First Interim Analysis of the European Haemophilia Safety Surveillance (EUHASS) Database [Abstract #2685] Poster presented at: American Society Of Hematology (ASH) Annual Meeting; 5 – 8 December, 2020.
[3]WFH. Guidelines for the management of haemophilia. 2012 [Internet; cited December 2020]. Available from: http://www1.wfh.org/publications/files/pdf-1472.pdf.
[4] Berntorp E, et al. Modern haemophilia care. The Lancet. 2012; 370:1447-1456.
[5] Marder VJ, et al. Hemostasis And Thrombosis. 6th ed. Wisconsin. Lippincott Williams & Wilkins; 2013.
[6]Franchini M, et al. Haemophilia A in the third millennium. Blood Rev. 2013; 179-84.
[7] Gomez K, et al. Key issues in inhibitor management in patients with haemophilia. Blood Transfus. 2014; 12:s319-s329.
[8]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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