Roche Reports Zero Treatment-Requiring Bleeds with NXT007 in Phase I/II Haemophilia A Study

Roche Reports Zero Treatment-Requiring Bleeds with NXT007 in Phase I/II Haemophilia A Study

(IN BRIEF) Roche presented positive phase I/II data for NXT007, its Chugai-engineered bispecific antibody for haemophilia A, at the 2025 ISTH Congress. In the NXTAGE study’s highest-dose cohorts (B-3 and B-4), none of the 30 participants experienced bleeds requiring treatment, and no thromboembolic events were observed. NXT007 is administered subcutaneously every two to four weeks after loading doses, aiming to mimic factor VIII activity by bridging factor IXa and factor X. Further phase II results are due later this year, with three phase III trials planned in 2026—including a head-to-head with Hemlibra®—to evaluate its efficacy, safety and dosing flexibility. This investigational therapy could offer people with haemophilia A sustained haemostatic normalization and a reduced treatment burden.

(PRESS RELEASE) BASEL, 24-Jun-2025 — /EuropaWire/ — Roche announced compelling phase I/II results for NXT007, its next-generation bispecific antibody for haemophilia A, at the 2025 ISTH Congress in Washington, D.C. The data from the NXTAGE study demonstrate that, in the highest dose cohorts, participants experienced zero bleeds requiring treatment. Engineered by Chugai, a member of the Roche Group, NXT007 is designed to restore normal haemostasis while reducing the burden of frequent injections.

During Part B of NXTAGE—conducted across Japan, Taiwan and South Korea—thirty previously untreated adults (ages 12–65) received ascending subcutaneous doses of NXT007 every two to four weeks, following an initial loading phase. After typical 9–10-hour activity periods, the highest-dose groups (B-3 and B-4) reported no breakthrough bleeds, and no thromboembolic events have occurred to date. Further phase II results are expected later this year, paving the way for three phase III trials slated for 2026, including a head-to-head comparison against Hemlibra® (emicizumab) with flexible dosing schedules.

Levi Garraway, MD, PhD, Roche’s Chief Medical Officer, remarked, “These encouraging NXT007 findings reinforce our commitment to delivering transformative therapies that let people with haemophilia A live unencumbered by their condition.” By combining factor IXa and factor X into a single antibody scaffold, NXT007 seeks to elevate bleed protection to levels seen in individuals without haemophilia, all while offering extended half-life and factor-independent dosing.

Haemophilia A affects an estimated 900,000 people worldwide and, without sufficient factor VIII, patients face frequent, often spontaneous bleeding that can lead to joint damage and chronic pain. The approval of subcutaneous Hemlibra revolutionized care; NXT007 aims to build on that legacy by further optimizing potency, convenience and durability. Roche—backed by over 25 years of expertise in haematology—will continue to advance NXT007 through its global development programme as part of its broader mission to innovate in blood disorders.

About NXT007
NXT007 is a next-generation investigational bispecific antibody, being investigated as a prophylactic (preventive) treatment option for people with haemophilia A.1,2,3

NXT007 was engineered by Chugai – a member of the Roche Group – built on Hemlibra® (emicizumab)’s framework, with the aim of optimising factor VIII-mimetic activity and half-life, to further enhance potency, efficacy, dosing and administration convenience. NXT007 brings together factor IXa and factor X, proteins required to activate the natural coagulation cascade.1,2,3 NXT007 is being studied in a robust clinical development programme exploring its potential to achieve sustained elevated bleed protection equivalent to people who do not have haemophilia A (sustained haemostatic normalisation), and reduced treatment burden with factor independence, offering people living with haemophilia A greater therapeutic choice.1,2,3

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.6,7 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 symptoms, people with haemophilia A can bleed frequently, especially into their joints or muscles.8 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.9 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.6

About Roche in haematology
Roche has been developing medicines for people with malignant and non-malignant blood diseases for more than 25 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, Hemlibra® (emicizumab), PiaSky® (crovalimab), Lunsumio® (mosunetuzumab) and Columvi® (glofitamab). Our pipeline of investigational haematology medicines includes T-cell engaging bispecific antibody cevostamab, targeting both FcRH5 and CD3 and Tecentriq® (atezolizumab). 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
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.

For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045.

Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.

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References
[1] 1. Shima M, et al. NXT007 Prophylaxis in Emicizumab-Naive Persons with Hemophilia A without Inhibitor: Phase I/II Study (NXTAGE) Presented at International Society on Thrombosis and Haemostasis (ISTH) congress; 2025 June. Abstract OC.20.3.
[2] Shima M, et al. Safety, Pharmacokinetics, and Pharmacodynamics of Single Subcutaneous Injection of NXT007, an Emicizumab-Based Next-Generation Bispecific Antibody, in Healthy Volunteers (NXTAGE Study). Presented at: International Society on Thrombosis and Haemostasis (ISTH) Congress; 2023 July 28. Abstract OC 69.4.
[3] Teranishi-Ikawa Y., et al. A bispecific antibody NXT007 exerts a hemostatic activity in hemophilia A monkeys enough to keep a non-hemophiliac state. Journal of Thrombosis and Haemostasis. 2023; doi: 10.1016/j.jtha.2023.09.034
[4] Hemlibra SmPC [Internet; cited 2025 June] Available from: https://www.medicines.org.uk/emc/product/9043/smpc
[5] FDA Prescribing Information [Internet; cited 2025 June]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761083s002s004lbl.pdf.
[6] Srivastava A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26 (Suppl 6): 1-158.
[7] Iorio A, et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males. Ann Intern Med. 2019;171(8):540-546.
[8] NHS. Symptoms of haemophilia [Internet; cited 2025 June]. Available from: https://www.nhs.uk/conditions/haemophilia/symptoms/.
[9] Franchini M, et al. Haemophilia A in the third millennium. Blood Rev. 2013; 179-84.

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SOURCE: Roche