Periop Pembrolizumab's Results for HNSCC Raise Questions
Adding pembrolizumab to standard-of-care treatment improved event-free survival (EFS) in locally advanced head and neck squamous cell carcinoma (HNSCC), according to new results of the KEYNOTE-689 trial.
'This new information supports changing the current standard of care to now include neoadjuvant and adjuvant pembrolizumab. For the first time in more than 20 years, patients with this challenging disease have a new therapeutic approach,' said lead investigator Ravindra Uppaluri, MD, PhD, head and neck surgical oncologist, Dana-Farber Cancer Institute, Boston, at the American Association for Cancer Research (AACR) Annual Meeting 2025.
Pembrolizumab — which already carries indications for unresectable/metastatic HNSCC — is currently under review by the US Food and Drug Administration (FDA) for a perioperative HNSCC indication based on the trial. A decision is expected by June 23, 2025.
Methods and Results
KEYNOTE-689 trial randomized 714 patients with stage III-IVa HNSCC; almost all patients were human papillomavirus–negative, about equally to either standard-of-care treatment — surgery followed by radiation plus chemotherapy for high-risk features on pathology — or standard of care plus perioperative pembrolizumab.
The perioperative regimen included two 3-week cycles of 200 mg neoadjuvant pembrolizumab followed by surgery, then three cycles of pembrolizumab concurrent with radiation plus/minus cisplatin, with 12 cycles of maintenance pembrolizumab afterward.
At a median follow-up of 38.3 months, EFS — defined as no recurrence or death — was a median of 59.7 months in the pembrolizumab arm, compared with 26.9 months in those receiving standard of care alone arm among the 465 patients with a programmed death ligand 1 combined positive score combined positive score (CPS) of ≥ 10. Similar results were seen among patients with a CPS of ≥ 1, with EFS of 59.7 months vs 29.6 months for the pembrolizumab vs standard of care alone groups, respectively. Across all patients, including the less than 5% of those patients with a CPS < 1, EFS was 51.8 months with perioperative pembrolizumab vs 30.4 months without it.
Neoadjuvant pembrolizumab also seemed to downstage tumors; a third of pembrolizumab patients had high-risk features — for instance, positive margins or extranodal involvement — on surgical pathology vs 44.4% in the standard of care arm. Subsequently, the use of adjuvant chemotherapy in the pembrolizumab group was lower at 29.6% vs 44.1%.
With pembrolizumab, 13.7% of patients with a CPS of ≥ 10, 9.8% of those with a CPS of ≥ 1, and 9.3% of all patients had a major pathologic response, defined as a tumor reduction of 90% or more.
Median overall survival has not been reached in the pembrolizumab add-on group, but it was 61.8 months in the standard of care arm. Follow-up for overall survival is ongoing.
Discussion of Results
'I think this trial does constitute a leap forward and a new standard of care,' said study discussant Robert Ferris, MD, PhD, a head and neck surgical oncologist at UNC Lineberger Comprehensive Cancer Center in Chapel Hill, North Carolina.
However, 'we have a great deal of new things we need to do' to get the most from the approach, he said.
For one, the hazard ratio for EFS benefit among 528 stage IVa patients was not statistically significant, so further study is necessary to identify stage IVa patients who do benefit, he said. Further work is also needed to identify the optimal treatment after progression, the risk for hyperprogression during programmed cell death 1 monotherapy, and the possibility of deintensifying surgery if patients who respond strongly to neoadjuvant treatment can be identified. It's also unknown if adjuvant pembrolizumab is best given with chemoradiation or afterward, Ferris said. Some studies have shown benefit by separating them, he added.
There might be a role for combining neoadjuvant pembrolizumab with chemotherapy or another immunotherapy, but further research is needed, noted Uppaluri. He added that it's unclear how long the maintenance phase of pembrolizumab should be, and the study did not address whether patients need pembrolizumab both before and after surgery, or if one or the other will do.
The FDA has also raised the question about the need for treatment on both sides of surgery in other perioperative immunotherapy trials. Ferris and Uppaluri said they both look forward to future related investigations for clarity.
The grade 3 or higher treatment-related adverse event rate was 44.6% with pembrolizumab add-on vs 42.9% without it. Immune-mediated adverse events occurred in 43.2% of pembrolizumab patients, most commonly hypothyroidism in 24.7%; grade 3 or higher immune-mediated adverse events occurred in 10%, including one fatal case of pneumonitis. There were four fatal adverse events with the pembrolizumab add-on and one in the standard-of-care group.
The work was funded by Merck, manufacturer of pembrolizumab, and KEYNOTE-689 investigators included Merck employees. Uppaluri reported receiving research funding and consulting fees from the company. Ferris is a Merck advisor.
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Inflammation Link Allergy, Asthma, and Depression?
The prevalence of depression is consistently higher in persons with asthma than in persons without it, according to experts, and a review of 24 studies also found that allergic rhinitis was associated with higher odds of depression or anxiety. Over the past two decades, multiple investigators have looked beyond emotional or lifestyle triggers of depression in chronic airway or allergic conditions, focusing instead on eco-biological ones. This has led to inflammation being identified as the common denominator in all three — allergies, asthma, and depression. As the understanding of how inflammation, as measured by an increase in immune cells, cytokines, and other biomarkers, affects neural signaling continues to improve; potential implications for clinical practice are beginning to emerge, experts say. Evolutionary 'Mismatch' One line of inquiry that implicates inflammation in depression, chronic airway trouble, and allergies, involves the microbiome and how modern living has altered it. 'There's essentially an evolutionary mismatch in the modern world,' Charles Raison, MD, professor of psychiatry and human ecology at the University of Wisconsin - Madison, told Medscape Medical News in an interview. 'We've made the world so clean we deprive ourselves — especially in early life when allergies and asthma tend to develop — of contact with microorganisms that train the immune system not to fire off at things they don't need to fire off about.' In a review of literature published in 2013 on immune function titled, Inflammation, Sanitation, and Consternation , Raison and his coauthors concluded that measured exposure to certain microorganisms and their antigens could offer both prevention and intervention for depression. 'Our current challenge is to determine who among those with allergies is at highest risk of developing depression, and why,' Christopher Lowry, PhD, a coauthor on the paper with Raison, told Medscape Medical News . Lowry is a professor of integrative physiology and associate chair of faculty affairs at the University of Colorado Boulder. 'Old Friends' Raison and Lowry's collaboration follows on from the 'Old Friends' work of Graham Rook, a coauthor of their review study, and an emeritus professor of medical microbiology at the University College London in London, England. 'The association between allergies and depression is embedded in the 'Old Friends' or Biodiversity Hypothesis which posits that reduced exposures to diverse microbial environments, which is typical of modern urban lifestyles, increases risk of allergies, anxiety disorders, and depression,' Lowry told Medscape Medical News . Because the commensal microorganisms in the gut are one of the major types of microbial 'Old Friends' that can induce anti-inflammatory and immunoregulatory responses, Lowry said, 'Those who lack diverse microbial exposures, including diverse microbial exposures in the gut microbiome, are thought to be at higher risk of inflammatory disease and inflammation-associated conditions.' Similarly, 'we have shown that this response is greater in persons who grow up in an urban environment, without daily exposure to pets, relative to persons who grow up on a farm, with daily exposure to farm animals,' said Lowry, whose research on the immune effects of exposure to animals was published in the Proceedings of the National Academies of Science . Other categories of microbial 'Old Friends,' according to Lowry, are environmental saprophytes found in soil, mud, and unpurified water, and so-called 'Old Infections' such as the hepatitis A virus or Salmonella . Rook has gone so far as to say that these microbial inputs, 'are absolutely crucial in early life, but continue to be important in adulthood and old age.' Raison added, 'These microbes don't cause illnesses, they are the teachers of tolerance, is how we used to say it. When we began to institute a bunch of modern sanitation and medical practices, on the plus side people stopped dying in youth. Huge win. But we didn't see we were tossing out the good with the bad, and we've lost our exposure to these environmental immunoregulatory organisms that suppress inflammation.' In the past 40 years, incident rates for asthma in the US have doubled according to federal data. A third of persons in the US now have an allergy of some kind, these data also showed. Rates of depression are also at their highest, according to a probability-based Gallup survey of 100,000 adults in the US. In 2023, the survey found that 29% of US adults had been given a diagnosis of depression in their lifetime. Microbial Population Health Initiative The 'Old Friends' theory was tested in Finland for the decade between 2008 and 2018, when a population health initiative to increase exposures to diverse microbial environments led to a reduction in hospitalizations for asthma by half nationally and a 50% drop in the number of food allergies reported in Finnish schools and daycares. The results were published in 2021 in The Journal of Allergy and Clinical Immunology . Depression rates for this time period in Finland are not currently available. 'This was hot, sexy stuff back 20 years ago, but nobody figured out the magic bullet to end all allergies and asthma, so like a lot of things in mental health, it didn't come too much,' Raison said. 'It's gone through fads, like the probiotic fad. But another question would be, what if you were to re-expose kids to these kinds of microbes that train the immune system not to be so agitated, would you lower rates of depression and suicide in adults? Those studies were never done.' Lowry, however, now studies whether the microorganism, Mycobacterium vaccae ATCC 15483, which has been identified as having anti-inflammatory, immunoregulatory, and stress-resilience properties, can be leveraged to protect humans from the effects of inflammation. In a study that was published earlier this year in Brain, Behavior and Immunity , Lowry and his colleagues found M vaccae reduced biomarkers of neuroinflammation and anxiety-like behavior in animals. It also reduced several indicators of obesity such as plasma leptin concentrations and adipose tissue. 'We hope to conduct clinical trials in the next 2-5 years to determine if treatment with mycobacteria can reduce inflammation and promote stress resilience in humans,' Lowry said. 'Future studies should evaluate if mycobacteria or other 'Old Friends' can prevent or treat anxiety and depression.' Inflammation Is Bidirectional If depression in asthma and allergies is at least partially biological, then there are implications for clinical practice, according to one expert. 'The default for physicians is usually that it's hard to have a chronic disease, and that's why their patients are depressed,' Melissa Rosenkranz, PhD, told Medscape Medical News . 'But if they understand that there is a biology that underlies it, that the immune dysregulation that's giving rise to the disease is also contributing to the depression, and that they can do something about that — if we can target those signaling pathways, then we can be treating both simultaneously.' Rosenkranz is an associate professor of psychiatry at the University of Wisconsin - Madison and is the distinguished chair of contemplative neuroscience at the Center for Healthy Minds. Her field is psycho-neuroimmunology, which focuses on interactions between the mind, brain, and immune system. Whether inflammation is the mechanism of action or a symptom in both asthma and depression is something she and her colleagues have studied. 'It goes in both directions,' Rosenkranz said. 'We've done that research.' Alterations in Neural Signaling Their first and second studies involved scanning study patients' brains before and after their airways were challenged with an inflammatory antigen and comparing those results to the before and after scans of the brains of participants exposed to methacholine, which does not irritate, but does constrict, the bronchial passages, making it difficult to breathe. A third study introduced an inflammatory agent to a micro region of the bronchial passages. 'All three studies showed what was happening in the brain was different in the inflammatory context,' Rosenkranz said in the interview. Specifically, Rosenkranz and her colleagues discovered that the brain's salience network — the part that prioritizes which stimuli, both internal and external to the body, require the brain's attention most — responded differently to emotional information when the immune system had been activated. They found that the more the salience network was engaged during psychological stress, the more the inflammatory response in the airway was activated, even without exposure to allergy triggers. 'Our results identify a specific inflammatory pathway linking asthma-related airway inflammation and emotion-related neural function,' Rosenkranz and her coauthors wrote in their study, published in The Journal of Allergy and Clinical Immunology . An immune pathway common to both depression and the airway inflammatory response in asthma is the T helper cell 17 (TH17) response, according to Rosenkranz, whose work has shown that the TH17 response is amplified in the lung during stress. Activity in the TH17 pathway is also elevated in people with depression, according to Rosenkranz, and TH17 is also less responsive to common asthma medications and is associated with treatment-resistant depression. 'There are probably multiple pathways through which [inflammation] happens, so I don't want to claim that this is the only one, but I think it's one among a few that may provoke inflammation in the brain,' Rosenkranz said. 'And inflammation has been shown to be part of the pathophysiology of depression. It's also part of the cascade that gives rise to Alzheimer's disease and other forms of dementia.' Rosenkranz's latest study is on how an asthma exacerbation can lead to inflammation in the brain, and whether those changes might set the stage for dementia. 'It's possible that depression is along the same trajectory as dementia,' Rosenkranz said. 'As you get on later in life, you see the cognitive decline. With nearly 10% of the population having asthma, this is a really important public health question.' Rosenkranz said she doesn't want to be an alarmist. 'I'm not suggesting that everybody who has depression has inflammation in their brain,' she said. As the field searches for an immune-signaling pathway that, if targeted with drugs, could improve asthma, allergies, prevent asthma-related depression, and possibly protect the brain, Rosenkranz said that for now, 'Physicians should evaluate the emotional well-being of their patients and work closely with mental health professionals to address mental and physical symptoms in tandem, so that they can best serve their patients.' They need to understand that depression in patients with asthma or allergy could be a symptom of, not a reaction to their diagnosis, she said.
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Early data suggest Roche's NXT007 may have the potential to provide haemostatic normalisation in people with haemophilia A
Positive phase I/II data presented at the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress show NXT007 achieved no bleeds requiring treatment in the highest dose groups in people with haemophilia A1 The NXT007 clinical development programme aims to normalise haemostasis and minimise treatment burden2,3 Three phase III clinical studies on NXT007, a next-generation bispecific antibody, set to begin in 20261 Basel, 23 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today positive phase I/II data on NXT007 in people with haemophilia A, supporting its progression into phase III clinical development. NXT007 is a next-generation investigational bispecific antibody, engineered by Chugai, a member of the Roche Group. Early data from the NXTAGE study suggest that NXT007 may have the potential to provide haemostatic normalisation in people with haemophilia A (without factor VIII inhibitors). NXT007 showed a tolerable safety profile with no thromboembolic events reported so far.1 These results were featured as an oral presentation at the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress, 21-25 June, Washington D.C., United States.1 'These NXT007 data are promising for people with haemophilia A and underscore our ongoing commitment to advancing care and addressing the real-world challenges faced by this community,' said Levi Garraway, MD, PhD, Roche's Chief Medical Officer and Head of Global Product Development. 'Hemlibra established a new standard of care, and our focus is to continue to deliver breakthrough innovation that might ultimately help people with haemophilia to live their lives in a manner unaffected by this condition.' NXT007 leverages the Roche Group's expertise in haemophilia A and bispecific antibody development. Our goal is to bring a next generation prophylactic to our portfolio, offering greater therapeutic choice, sustained, elevated bleed protection and reduced treatment burden with factor independence - to allow patients to experience freedom from constant vigilance and have confidence in bleed protection. NXT007 will be further evaluated in a robust clinical development programme, including ongoing phase I/II clinical trials, with additional phase II data expected later this year. There are also three phase III studies currently planned for 2026, including a phase III head-to-head study with Roche's Hemlibra, the first available prophylactic treatment that can be administered subcutaneously and with flexible dosing options (every week, two weeks or four weeks).4,5 Part B of the phase I/II NXTAGE study, conducted by Chugai, in Japan, Taiwan and South Korea, is evaluating the safety, pharmacokinetics, pharmacodynamics and efficacy of prophylaxis with NXT007 in people with haemophilia A without factor VIII inhibitors who had not been previously treated with Hemlibra® (emicizumab).1 Thirty participants (from 12 to 65 years of age) were enrolled in four cohorts (B-1 to B-4) to receive ascending doses of subcutaneous NXT007 every two-to-four weeks during the maintenance period (following four-to-six weeks of loading doses). In presented data from the primary analysis, no treated bleeds were observed with NXT007 in the highest dose cohorts (B-3 and B-4). NXT007 was well tolerated, with no thromboembolic events observed so far.1 About NXT007NXT007 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 AHaemophilia 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 haematologyRoche 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. For more information, please visit All trademarks used or mentioned in this release are protected by law. 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/ Hemlibra SmPC [Internet; cited 2025 June] Available from: [5] FDA Prescribing Information [Internet; cited 2025 June]. Available from: [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: [9] Franchini M, et al. Haemophilia A in the third millennium. Blood Rev. 2013; 179-84. Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: Hans Trees, PhDPhone: +41 79 407 72 58 Sileia UrechPhone: +41 79 935 81 48 Nathalie AltermattPhone: +41 79 771 05 25 Lorena CorfasPhone: +41 79 568 24 95 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Nina MählitzPhone: +41 79 327 54 74 Kirti PandeyPhone: +49 172 6367262 Yvette PetillonPhone: +41 79 961 92 50 Dr Rebekka SchnellPhone: +41 79 205 27 03 Roche Investor Relations Dr Bruno EschliPhone: +41 61 68-75284e-mail: Dr Sabine BorngräberPhone: +41 61 68-88027e-mail: Dr Birgit MasjostPhone: +41 61 68-84814e-mail: Investor Relations North America Loren KalmPhone: +1 650 225 3217e-mail: Attachment Media Investor Release phase I_II data on NXT007 EnglishError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
an hour ago
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Early data suggest Roche's NXT007 may have the potential to provide haemostatic normalisation in people with haemophilia A
Positive phase I/II data presented at the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress show NXT007 achieved no bleeds requiring treatment in the highest dose groups in people with haemophilia A1 The NXT007 clinical development programme aims to normalise haemostasis and minimise treatment burden2,3 Three phase III clinical studies on NXT007, a next-generation bispecific antibody, set to begin in 20261 Basel, 23 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today positive phase I/II data on NXT007 in people with haemophilia A, supporting its progression into phase III clinical development. NXT007 is a next-generation investigational bispecific antibody, engineered by Chugai, a member of the Roche Group. Early data from the NXTAGE study suggest that NXT007 may have the potential to provide haemostatic normalisation in people with haemophilia A (without factor VIII inhibitors). NXT007 showed a tolerable safety profile with no thromboembolic events reported so far.1 These results were featured as an oral presentation at the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress, 21-25 June, Washington D.C., United States.1 'These NXT007 data are promising for people with haemophilia A and underscore our ongoing commitment to advancing care and addressing the real-world challenges faced by this community,' said Levi Garraway, MD, PhD, Roche's Chief Medical Officer and Head of Global Product Development. 'Hemlibra established a new standard of care, and our focus is to continue to deliver breakthrough innovation that might ultimately help people with haemophilia to live their lives in a manner unaffected by this condition.' NXT007 leverages the Roche Group's expertise in haemophilia A and bispecific antibody development. Our goal is to bring a next generation prophylactic to our portfolio, offering greater therapeutic choice, sustained, elevated bleed protection and reduced treatment burden with factor independence - to allow patients to experience freedom from constant vigilance and have confidence in bleed protection. NXT007 will be further evaluated in a robust clinical development programme, including ongoing phase I/II clinical trials, with additional phase II data expected later this year. There are also three phase III studies currently planned for 2026, including a phase III head-to-head study with Roche's Hemlibra, the first available prophylactic treatment that can be administered subcutaneously and with flexible dosing options (every week, two weeks or four weeks).4,5 Part B of the phase I/II NXTAGE study, conducted by Chugai, in Japan, Taiwan and South Korea, is evaluating the safety, pharmacokinetics, pharmacodynamics and efficacy of prophylaxis with NXT007 in people with haemophilia A without factor VIII inhibitors who had not been previously treated with Hemlibra® (emicizumab).1 Thirty participants (from 12 to 65 years of age) were enrolled in four cohorts (B-1 to B-4) to receive ascending doses of subcutaneous NXT007 every two-to-four weeks during the maintenance period (following four-to-six weeks of loading doses). In presented data from the primary analysis, no treated bleeds were observed with NXT007 in the highest dose cohorts (B-3 and B-4). NXT007 was well tolerated, with no thromboembolic events observed so far.1 About NXT007NXT007 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 AHaemophilia 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 haematologyRoche 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. For more information, please visit All trademarks used or mentioned in this release are protected by law. 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/ Hemlibra SmPC [Internet; cited 2025 June] Available from: [5] FDA Prescribing Information [Internet; cited 2025 June]. Available from: [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: [9] Franchini M, et al. Haemophilia A in the third millennium. Blood Rev. 2013; 179-84. 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