Latest news with #CRSwNP
Yahoo
4 days ago
- Business
- Yahoo
GSK makes new drug submission for depemokimab in Canada
GSK has made a new drug submission (NDS) to Health Canada for the monoclonal antibody, depemokimab, targeting two specific conditions. The first proposed indication is for the use as an add-on maintenance treatment for asthma in individuals aged 12 years and above with type 2 inflammation marked by an eosinophilic phenotype. The second is for treating adults with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP). Depemokimab targets interleukin-5 (IL-5), which plays a crucial role in type 2 inflammation. GSK's submission is supported by the positive Phase III SWIFT and ANCHOR trial data. These trials have demonstrated that the antibody could provide sustained inhibition of a key disease driver and assist in achieving clinical outcomes with two injections annually. The extended half-life and binding affinity of the antibody support a dosing regimen of a single injection every six months. Patients with CRSwNP often suffer from symptoms such as loss of smell, facial pain, nasal obstruction, sleep disturbance, nasal discharge and infections. The effectiveness and safety of the antibody are still being investigated, and it has neither been granted any authorisation nor approved for use in any nation at present. GSK Canada country medical director Michelle Horn stated: 'The combined submission for asthma and CRSwNP marks a significant step toward addressing the unmet needs of patients. 'Backed by strong clinical evidence, depemokimab has the potential to become the first ultra-long-acting biologic offering patients sustained inhibition of IL-5, a key driver of their disease with twice-yearly dosing, and represents a promising advancement for patients and physicians alike.' In November 2024, GSK secured Health Canada's approval for Ojjaara (momelotinib) to treat myelofibrosis in adults with moderate-to-severe anaemia. "GSK makes new drug submission for depemokimab in Canada" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Cision Canada
5 days ago
- Health
- Cision Canada
GSK's depemokimab accepted for review by Health Canada for the treatment of asthma with type 2 inflammation and for chronic rhinosinusitis with nasal polyps
SWIFT-1 and -2 trials showed depemokimab reduced exacerbation and hospitalization rates as an add-on therapy for patients with asthma with type 2 inflammation versus placebo ANCHOR-1 and -2 trials showed early and sustained reductions in nasal polyp size and nasal obstruction versus placebo for patients with chronic rhinosinusitis with nasal polyps Across the SWIFT and ANCHOR clinical trials, the overall incidence and severity of treatment-emergent adverse events were similar in patients treated with either depemokimab or placebo If approved, depemokimab will be the first ultra-long-acting biologic with two doses per year (6-month dosing) MISSISSAUGA, ON, May 26, 2025 /CNW/ - GSK has submitted a New Drug Submission (NDS) to Health Canada for depemokimab for two proposed indications: The first indication is as an add-on maintenance treatment of asthma in adult and adolescent patients aged 12 years and older with type 2 inflammation characterized by an eosinophilic phenotype on medium- to high-dose inhaled corticosteroids (ICS) plus another asthma controller. The second indication is as an add-on maintenance treatment of adult patients with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP). The NDS is based on data from the positive SWIFT and ANCHOR trials. Michelle Horn, Country Medical Director, GSK Canada, said: "The combined submission for asthma and CRSwNP marks a significant step toward addressing the unmet needs of patients. Backed by strong clinical evidence, depemokimab has the potential to become the first ultra-long-acting biologic offering patients sustained inhibition of IL-5, a key driver of their disease with twice-yearly dosing, and represents a promising advancement for patients and physicians alike." Depemokimab, a monoclonal antibody that targets IL-5, is the first ultra-long-acting biologic to be evaluated in phase III trials. 1, 2, 3 IL-5 is a key cytokine (protein) in type 2 inflammation. 1, 5, 6 Type 2 inflammation is typically identified by blood eosinophil count and is an underlying driver in many diseases. This type of inflammation is present in the majority of patients with difficult to treat asthma and can lead to exacerbations and hospitalization. 5, 6, 7 Type 2 inflammation is also present in up to 85% of people with CRSwNP and is associated with more severe disease and symptoms. 8, 9, 10, 11, 12 With IL-5 inhibition, eosinophils are significantly reduced and there is evidence to show IL-5 has broader effects on other structural and immune cell types beyond eosinophils. 5, 6, 22, 23, 24, 25, 26 In patients with asthma with type 2 inflammation and patients with CRSwNP, the SWIFT and ANCHOR trials, respectively, met their primary endpoints, showing that depemokimab could offer sustained inhibition of an important driver of their disease, and help achieve key clinical outcomes with a dosing schedule of just two injections per year. 1, 2, 3 Depemokimab's extended half-life, high-binding affinity and potency, support a dosing regimen of one injection every six months (26 weeks).. 1, 2, 3 As demonstrated in studies of other diseases, longer intervals between doses have been shown to overcome barriers to optimal care, such as patient adherence, and can reduce the burden of disease for patients. 4 In Canada, more than 4.7 million people are currently affected by asthma, a chronic and sometimes debilitating condition. 27 Many Canadian asthmatics continue to experience symptoms such as difficulty breathing and chest tightness, despite treatment with high-dose inhaled corticosteroids plus a second controller (and/or systemic corticosteroids). 5,20 People with CRSwNP experience symptoms such as nasal obstruction, loss of smell, facial pain, sleep disturbance, infections and nasal discharge that can significantly affect their emotional and physical well-being. 8, 9, 10, 11 Such symptoms mean the impact of CRSwNP on overall quality of life has been reported to be comparable with other chronic diseases such as COPD, asthma, and diabetes. 9 The safety and effectiveness of depemokimab are still under investigation and authorization has not yet been granted. Depemokimab is currently not approved for use in any country. About SWIFT-1 and SWIFT-2 SWIFT-1 and SWIFT-2 were replicate 52-week, randomised (2:1), double-blind, placebo-controlled, parallel-group, multi-centre Phase III clinical trials. 1 The trials assessed the efficacy and safety of depemokimab as adjunctive therapy in 382 and 380 participants with severe asthma with type 2 inflammation characterised by blood eosinophil count, including adult and adolescent patients, who were randomised to receive depemokimab or a placebo respectively, in addition to their standard of care treatment with medium to high-dose inhaled corticosteroids plus at least one additional controller. 1 In each trial, the rate of asthma exacerbations was significantly lower in the depemokimab group than in the placebo group. 1 These results have been reported and published in the New England Journal of Medicin e. 1 About ANCHOR-1 and ANCHOR-2 ANCHOR-1 and ANCHOR-2 were replicate 52-week, randomised (1:1), double-blind, placebo-controlled, parallel-group, multi-centre Phase III clinical trials. 2, 3 The trials assessed the efficacy and safety of depemokimab as add-on therapy to standard of care in 271 and 257 adult patients with CRSwNP inadequately controlled on intranasal corticosteroids. 2, 3 The co-primary endpoints were met with statistically significant reductions in nasal polyp size and nasal obstruction in patients receiving depemokimab versus placebo, at 52 weeks. 2, 3 Full results of ANCHOR-1 and ANCHOR-2 have been reported and published in The Lancet. 19 About GSK in respiratory GSK is redefining the future of respiratory medicine as it builds on decades of pioneering work to deliver more ambitious treatment goals and develop the next-generation standard of care, for hundreds of millions of people with respiratory diseases. With an industry-leading respiratory portfolio and pipeline of vaccines, targeted biologics, and inhaled medicines, we are focused on improving outcomes and the lives of people living with all types of asthma and COPD along with less understood diseases like refractory chronic cough or rarer conditions like systemic sclerosis with interstitial lung disease. GSK is harnessing the latest science and technology with the aim to modify underlying disease dysfunction and prevent disease progression. About GSK GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at Cautionary statement regarding forward-looking statements GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described in the "Risk Factors" section in GSK's Annual Report on Form 20-F for 2024, and GSK's Q1 Results for 2025. 1. Jackson DJ, et al. Six Monthly Depemokimab in Severe Asthma With an Eosinophilic Phenotype. NEJM. Published on September 9 at 2. Efficacy and Safety of Depemokimab (GSK3511294) in Participants With Chronic Rhinosinusitis With Nasal Polyps (ANCHOR-1) Available at: Accessed February 2025 3. Efficacy and Safety of Depemokimab (GSK3511294) in Participants With Chronic Rhinosinusitis With Nasal Polyps (ANCHOR-2) Available at: Accessed February 2025 4. Scarsi KK, Swindells S. The Promise of Improved Adherence With Long-Acting Antiretroviral Therapy: What Are the Data? Journal of the International Association of Providers of AIDS Care (JIAPAC). 2021;20. 5. Global Initiative for Asthma. Global Strategy for Asthma Management and Prevention,2024. Updated May 2024. Available at: Accessed February 2025. 6. Heaney L, et al. Eosinophilic and Noneosinophilic Asthma: An Expert Consensus Framework to Characterize Phenotypes in a Global Real-Life Severe Asthma Cohort. Chest. 2021;160(3):814-830. 7. Principe S, et al. Severe asthma: Targeting the IL-5 pathway. Clin Exp Allergy. 2021 Aug;51(8):992-1005 8. Laidlaw TM, et al. Chronic Rhinosinusitis with Nasal Polyps and Asthma. J. Allergy Clin. Immunol. 2001;9(3):1133-1141. 9. Bachert C, et al. Burden of Disease in Chronic Rhinosinusitis with Nasal Polyps. J Asthma Allergy. 2021;b 11;14:127-134. 10. De Corso E, et al. How to manage recurrences after surgery in CRSwNP patients in the biologic era: a narrative review. Acta Otorhinolaryngol Ital. 2023;43(Suppl. 1):S3-S13. 11. Chen S, et al. Systematic literature review of the epidemiology and clinical burden of chronic rhinosinusitis with nasal polyposis. Curr Med Res Opin. 2020;36(11):1897-1911. 12. Bachert C, et al. EUFOREA expert board meeting on uncontrolled severe chronic rhinosinusitis with nasal polyps (CRSwNP) and biologics: Definitions and management. J Allergy Clin Immunol. 2021;147(1):29-36. 13. American Lung Association. Severe Asthma. Available at: Accessed February 2025. 14. American Lung Association. Asthma Trends and Burden. Available at: Accessed February 2025. 15. An Open-Label Extension Study of GSK3511294 (Depemokimab) in Participants Who Were Previously Enrolled in 206713 (NCT04719832) or 213744 (NCT04718103) (AGILE). Available at: Accessed February 2025. 16. A Study of GSK3511294 (Depemokimab) Compared With Mepolizumab or Benralizumab in Participants With Severe Asthma With an Eosinophilic Phenotype (NIMBLE). Available at: Accessed February 2025. 17. Efficacy and Safety of Depemokimab Compared With Mepolizumab in Adults With Relapsing or Refractory Eosinophilic Granulomatosis With Polyangiitis (EGPA) Available at: Accessed February 2025. 18. Depemokimab in Participants With Hypereosinophilic Syndrome, Efficacy, and Safety Trial (DESTINY) Available at: Accessed February 2025. 19. Gevaert P, et al. Efficacy and safety of twice per year depemokimab in chronic rhinosinusitis with nasal polyps (ANCHOR-1 and ANCHOR-2): phase III, randomised, double-blind, parallel trials. The Lancet. Published on February 28 at 20. World Health Organisation. Asthma Key Facts. Available at: Accessed February 2025 21. Israel, E, et al. Severe and Difficult-to-Treat Asthma in Adults. N Engl J Med 2017;377:965-76. 22. Buchheit KM, et al. Mepolizumab targets multiple immune cells in aspirin-exacerbated respiratory disease. J Allergy Clin Immunol. 2021;148(2):574-584. 23. Barretto KT, et al. Human airway epithelial cells express a functional IL-5 receptor. Allergy. 2020;75(8):2127-2130. 24. Bajbouj K, et al. IL-5 receptor expression in lung fibroblasts: Potential role in airway remodelling in asthma. Allergy. 2023;78(3):882-885. 25. Siddiqui S, et al. Eosinophils and tissue remodeling: Relevance to airway disease. J Allergy Clin Immunol. 2023;152(4):841-857. 26. Bergantini L, et al. Regulatory T cell monitoring in severe eosinophilic asthma patients treated with mepolizumab. Scand J Immunol. 2021;94(1):e13031. SOURCE GlaxoSmithKline Inc.
Yahoo
5 days ago
- Health
- Yahoo
GSK's depemokimab accepted for review by Health Canada for the treatment of asthma with type 2 inflammation and for chronic rhinosinusitis with nasal polyps
SWIFT-1 and -2 trials showed depemokimab reduced exacerbation and hospitalization rates as an add-on therapy for patients with asthma with type 2 inflammation versus placebo ANCHOR-1 and -2 trials showed early and sustained reductions in nasal polyp size and nasal obstruction versus placebo for patients with chronic rhinosinusitis with nasal polyps Across the SWIFT and ANCHOR clinical trials, the overall incidence and severity of treatment-emergent adverse events were similar in patients treated with either depemokimab or placebo If approved, depemokimab will be the first ultra-long-acting biologic with two doses per year (6-month dosing) MISSISSAUGA, ON, May 26, 2025 /CNW/ - GSK has submitted a New Drug Submission (NDS) to Health Canada for depemokimab for two proposed indications: The first indication is as an add-on maintenance treatment of asthma in adult and adolescent patients aged 12 years and older with type 2 inflammation characterized by an eosinophilic phenotype on medium- to high-dose inhaled corticosteroids (ICS) plus another asthma controller. The second indication is as an add-on maintenance treatment of adult patients with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP). The NDS is based on data from the positive SWIFT and ANCHOR trials. Michelle Horn, Country Medical Director, GSK Canada, said: "The combined submission for asthma and CRSwNP marks a significant step toward addressing the unmet needs of patients. Backed by strong clinical evidence, depemokimab has the potential to become the first ultra-long-acting biologic offering patients sustained inhibition of IL-5, a key driver of their disease with twice-yearly dosing, and represents a promising advancement for patients and physicians alike." Depemokimab, a monoclonal antibody that targets IL-5, is the first ultra-long-acting biologic to be evaluated in phase III trials.1,2,3 IL-5 is a key cytokine (protein) in type 2 inflammation.1,5,6 Type 2 inflammation is typically identified by blood eosinophil count and is an underlying driver in many diseases. This type of inflammation is present in the majority of patients with difficult to treat asthma and can lead to exacerbations and hospitalization.5,6,7 Type 2 inflammation is also present in up to 85% of people with CRSwNP and is associated with more severe disease and symptoms.8,9,10,11,12 With IL-5 inhibition, eosinophils are significantly reduced and there is evidence to show IL-5 has broader effects on other structural and immune cell types beyond eosinophils.5,6,22,23,24,25,26 In patients with asthma with type 2 inflammation and patients with CRSwNP, the SWIFT and ANCHOR trials, respectively, met their primary endpoints, showing that depemokimab could offer sustained inhibition of an important driver of their disease, and help achieve key clinical outcomes with a dosing schedule of just two injections per year.1,2,3 Depemokimab's extended half-life, high-binding affinity and potency, support a dosing regimen of one injection every six months (26 weeks)..1,2,3 As demonstrated in studies of other diseases, longer intervals between doses have been shown to overcome barriers to optimal care, such as patient adherence, and can reduce the burden of disease for patients.4 In Canada, more than 4.7 million people are currently affected by asthma, a chronic and sometimes debilitating condition.27 Many Canadian asthmatics continue to experience symptoms such as difficulty breathing and chest tightness, despite treatment with high-dose inhaled corticosteroids plus a second controller (and/or systemic corticosteroids). 5,20 People with CRSwNP experience symptoms such as nasal obstruction, loss of smell, facial pain, sleep disturbance, infections and nasal discharge that can significantly affect their emotional and physical well-being.8,9,10,11 Such symptoms mean the impact of CRSwNP on overall quality of life has been reported to be comparable with other chronic diseases such as COPD, asthma, and diabetes.9 The safety and effectiveness of depemokimab are still under investigation and authorization has not yet been granted. Depemokimab is currently not approved for use in any country. About SWIFT-1 and SWIFT-2SWIFT-1 and SWIFT-2 were replicate 52-week, randomised (2:1), double-blind, placebo-controlled, parallel-group, multi-centre Phase III clinical trials.1 The trials assessed the efficacy and safety of depemokimab as adjunctive therapy in 382 and 380 participants with severe asthma with type 2 inflammation characterised by blood eosinophil count, including adult and adolescent patients, who were randomised to receive depemokimab or a placebo respectively, in addition to their standard of care treatment with medium to high-dose inhaled corticosteroids plus at least one additional controller.1 In each trial, the rate of asthma exacerbations was significantly lower in the depemokimab group than in the placebo group. 1 These results have been reported and published in the New England Journal of Medicine.1 About ANCHOR-1 and ANCHOR-2ANCHOR-1 and ANCHOR-2 were replicate 52-week, randomised (1:1), double-blind, placebo-controlled, parallel-group, multi-centre Phase III clinical trials. 2,3 The trials assessed the efficacy and safety of depemokimab as add-on therapy to standard of care in 271 and 257 adult patients with CRSwNP inadequately controlled on intranasal corticosteroids.2,3 The co-primary endpoints were met with statistically significant reductions in nasal polyp size and nasal obstruction in patients receiving depemokimab versus placebo, at 52 weeks. 2,3 Full results of ANCHOR-1 and ANCHOR-2 have been reported and published in The Lancet.19 About GSK in respiratoryGSK is redefining the future of respiratory medicine as it builds on decades of pioneering work to deliver more ambitious treatment goals and develop the next-generation standard of care, for hundreds of millions of people with respiratory diseases. With an industry-leading respiratory portfolio and pipeline of vaccines, targeted biologics, and inhaled medicines, we are focused on improving outcomes and the lives of people living with all types of asthma and COPD along with less understood diseases like refractory chronic cough or rarer conditions like systemic sclerosis with interstitial lung disease. GSK is harnessing the latest science and technology with the aim to modify underlying disease dysfunction and prevent disease progression. About GSKGSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at Cautionary statement regarding forward-looking statementsGSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described in the "Risk Factors" section in GSK's Annual Report on Form 20-F for 2024, and GSK's Q1 Results for 2025. References 1. Jackson DJ, et al. Six Monthly Depemokimab in Severe Asthma With an Eosinophilic Phenotype. NEJM. Published on September 9 at 2. Efficacy and Safety of Depemokimab (GSK3511294) in Participants With Chronic Rhinosinusitis With Nasal Polyps (ANCHOR-1) Available at: Accessed February 2025 3. Efficacy and Safety of Depemokimab (GSK3511294) in Participants With Chronic Rhinosinusitis With Nasal Polyps (ANCHOR-2) Available at: Accessed February 2025 4. Scarsi KK, Swindells S. The Promise of Improved Adherence With Long-Acting Antiretroviral Therapy: What Are the Data? Journal of the International Association of Providers of AIDS Care (JIAPAC). 2021;20. 5. Global Initiative for Asthma. Global Strategy for Asthma Management and Prevention,2024. Updated May 2024. Available at: Accessed February 2025. 6. Heaney L, et al. Eosinophilic and Noneosinophilic Asthma: An Expert Consensus Framework to Characterize Phenotypes in a Global Real-Life Severe Asthma Cohort. Chest. 2021;160(3):814-830. 7. Principe S, et al. Severe asthma: Targeting the IL-5 pathway. Clin Exp Allergy. 2021 Aug;51(8):992-1005 8. Laidlaw TM, et al. Chronic Rhinosinusitis with Nasal Polyps and Asthma. J. Allergy Clin. Immunol. 2001;9(3):1133-1141. 9. Bachert C, et al. Burden of Disease in Chronic Rhinosinusitis with Nasal Polyps. J Asthma Allergy. 2021;b 11;14:127-134. 10. De Corso E, et al. How to manage recurrences after surgery in CRSwNP patients in the biologic era: a narrative review. Acta Otorhinolaryngol Ital. 2023;43(Suppl. 1):S3-S13. 11. Chen S, et al. Systematic literature review of the epidemiology and clinical burden of chronic rhinosinusitis with nasal polyposis. Curr Med Res Opin. 2020;36(11):1897-1911. 12. Bachert C, et al. EUFOREA expert board meeting on uncontrolled severe chronic rhinosinusitis with nasal polyps (CRSwNP) and biologics: Definitions and management. J Allergy Clin Immunol. 2021;147(1):29-36. 13. American Lung Association. Severe Asthma. Available at: Accessed February 2025. 14. American Lung Association. Asthma Trends and Burden. Available at: Accessed February 2025. 15. An Open-Label Extension Study of GSK3511294 (Depemokimab) in Participants Who Were Previously Enrolled in 206713 (NCT04719832) or 213744 (NCT04718103) (AGILE). Available at: Accessed February 2025. 16. A Study of GSK3511294 (Depemokimab) Compared With Mepolizumab or Benralizumab in Participants With Severe Asthma With an Eosinophilic Phenotype (NIMBLE). Available at: Accessed February 2025. 17. Efficacy and Safety of Depemokimab Compared With Mepolizumab in Adults With Relapsing or Refractory Eosinophilic Granulomatosis With Polyangiitis (EGPA) Available at: Accessed February 2025. 18. Depemokimab in Participants With Hypereosinophilic Syndrome, Efficacy, and Safety Trial (DESTINY) Available at: Accessed February 2025. 19. Gevaert P, et al. Efficacy and safety of twice per year depemokimab in chronic rhinosinusitis with nasal polyps (ANCHOR-1 and ANCHOR-2): phase III, randomised, double-blind, parallel trials. The Lancet. Published on February 28 at 20. World Health Organisation. Asthma Key Facts. Available at: Accessed February 2025 21. Israel, E, et al. Severe and Difficult-to-Treat Asthma in Adults. N Engl J Med 2017;377:965-76. 22. Buchheit KM, et al. Mepolizumab targets multiple immune cells in aspirin-exacerbated respiratory disease. J Allergy Clin Immunol. 2021;148(2):574-584. 23. Barretto KT, et al. Human airway epithelial cells express a functional IL-5 receptor. Allergy. 2020;75(8):2127-2130. 24. Bajbouj K, et al. IL-5 receptor expression in lung fibroblasts: Potential role in airway remodelling in asthma. Allergy. 2023;78(3):882-885. 25. Siddiqui S, et al. Eosinophils and tissue remodeling: Relevance to airway disease. J Allergy Clin Immunol. 2023;152(4):841-857. 26. Bergantini L, et al. Regulatory T cell monitoring in severe eosinophilic asthma patients treated with mepolizumab. Scand J Immunol. 2021;94(1):e13031. 27. Public Health Agency of Canada. (2024). Canadian Chronic Disease Surveillance System (CCDSS) Data Tool. Retrieved from SOURCE GlaxoSmithKline Inc. View original content to download multimedia: Error 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
Medscape
15-05-2025
- Health
- Medscape
Tezepelumab Slashes Surgery Risk in Severe Nasal Polyposis
Tezepelumab (Tezspire) has shown significant clinical benefits in adults with severe chronic rhinosinusitis with nasal polyps (CRSwNP), including marked reductions in nasal congestion and polyp size and a near elimination of the need for surgical intervention. Findings from the phase 3 WAYPOINT trial were published in The New England Journal of Medicine and presented as a late-breaking oral presentation at the 2025 American Academy of Allergy, Asthma & Immunology/World Allergy Organization annual meeting. Chronic rhinosinusitis with NP is a chronic inflammatory disorder characterized by persistent inflammation of the nasal mucosa and the presence of benign growths (NP). These polyps can obstruct nasal airflow and lead to symptoms such as nasal congestion, loss of smell, rhinorrhea, facial pressure or pain, sleep disturbances, and a substantial reduction in quality of life. Standard therapies include intranasal or systemic corticosteroids, surgical resection, and more recently, biologic agents. Adults With Severe Nasal Polyposis Tezepelumab, developed by AstraZeneca in collaboration with Amgen, is a first-in-class human monoclonal antibody that targets thymic stromal lymphopoietin (TSLP), an upstream epithelial cytokine known to initiate and amplify various inflammatory pathways, including those involved in allergic and eosinophilic airway diseases. Preclinical and clinical data suggest that inhibiting TSLP could be an effective strategy for modulating inflammation in both upper and lower airway diseases. The WAYPOINT study was a randomized, double-blind, placebo-controlled, multicenter, parallel-group trial designed to assess the efficacy and safety of subcutaneous tezepelumab in adults with severe CRSwNP. Participants received either tezepelumab or placebo for a 52-week treatment period, followed by a posttreatment follow-up phase lasting 12-24 weeks. Reduction in Polyp Severity Treatment with tezepelumab resulted in a significant reduction in NP severity, as demonstrated by the co-primary endpoints in the phase 3 WAYPOINT trial. The NP score improved by −2.065 ( P < .0001), and use of systemic corticosteroids was reduced by 88% ( P < .0001) compared with placebo. Improvements in NP score were observed as early as week 4, while improvements in nasal congestion score were noted by week 2, the first posttreatment assessment. These effects were sustained through week 52. Significant and clinically meaningful improvements were also observed across all key secondary endpoints in the overall trial population. Tezepelumab was associated with a 98% reduction in the need for NP surgery ( P < .0001) and again an 88% reduction in systemic corticosteroid use ( P < .0001) compared with placebo. 'The WAYPOINT study confirms the efficacy of tezepelumab in reducing the need for further surgery, systemic corticosteroid use, improving SNOT-22 scores, and restoring olfactory function,' said Geoffrey Mortuaire, MD, PhD, head of the Department of ENT and Head and Neck Surgery at Lille University Hospital, Lille, France. 'The rapid and consistent treatment response observed in patients supports the potential for making tezepelumab more broadly available. We hope to see its approval for this indication in France soon.' Favorable Safety Profile Tezepelumab was generally well tolerated in patients with severe nasal polyposis and demonstrated a safety profile consistent with its current indication for severe asthma. The most frequently reported adverse events in the WAYPOINT study were COVID-19, nasopharyngitis, and upper respiratory tract infections. There were no clinically meaningful differences in safety outcomes between the tezepelumab and placebo groups. Tezepelumab is currently approved as add-on maintenance therapy for severe asthma in adults and adolescents aged 12 years or older who remain uncontrolled despite high-dose inhaled corticosteroids and additional maintenance therapy. It is approved in the United States, Europe, Japan, and nearly 60 countries worldwide. Regulatory applications for tezepelumab in severe nasal polyposis are currently under review by health authorities in several regions, according to AstraZeneca.
Business Upturn
06-05-2025
- Business
- Business Upturn
Upstream Bio Reports First Quarter 2025 Financial Results and Accelerates Guidance on All Clinical Programs
By GlobeNewswire Published on May 6, 2025, 16:00 IST – Top-line data from Phase 2 clinical trial of verekitug in patients with chronic rhinosinusitis with nasal polyps expected in the third quarter of 2025 – – Top-line data from Phase 2 clinical trial of verekitug in patients with severe asthma now expected in the first half of 2026 – – First patient in Phase 2 clinical trial of verekitug in patients with chronic obstructive pulmonary disease to be dosed in mid-2025 – WALTHAM, Mass., May 06, 2025 (GLOBE NEWSWIRE) — Upstream Bio, Inc. (Nasdaq: UPB), a clinical-stage company developing treatments for inflammatory diseases, with an initial focus on severe respiratory disorders, today reported financial results for the first quarter ended March 31, 2025, and provided a summary of recent business highlights. The Company is developing verekitug, the only monoclonal antibody currently in clinical development that targets and inhibits the thymic stromal lymphopoietin (TSLP) receptor, in multiple severe respiratory diseases including chronic rhinosinusitis with nasal polyps (CRSwNP), severe asthma and chronic obstructive pulmonary disease (COPD). 'This quarter we made excellent progress in our development of verekitug, positioning us well to deliver on our upcoming clinical milestones. We are pleased to accelerate our guidance on several near-term events, including the top-line data readout from our Phase 2 clinical trial of verekitug in patients with CRSwNP, expected in the third quarter of this year,' said Rand Sutherland, MD, Chief Executive Officer of Upstream Bio. 'In addition, we now anticipate reporting top-line data from our Phase 2 clinical trial in severe asthma in the first half of 2026. We also now expect to dose the first patient in our Phase 2 clinical trial in COPD in mid-2025. We look forward to sharing further updates as we reach these key milestones.' Dr. Sutherland continued, 'Verekitug is the only known molecule currently in clinical development targeting the TSLP receptor. Early clinical data suggest that this unique mechanism of action has the potential to meaningfully impact disease activity in patients with these severe respiratory diseases through both differentiated efficacy and an extended dosing interval, and we are testing the therapeutic implications of these observations across our development programs.' First Quarter 2025 and Recent Business Highlights Top-line data from Phase 2 clinical trial in patients with CRSwNP expected in the third quarter of 2025: In January 2025, Upstream Bio completed patient enrollment in its Phase 2 multicenter, randomized, placebo-controlled, parallel group clinical trial designed to assess the efficacy and safety of verekitug in participants with CRSwNP. Top-line data from this clinical trial is expected to be reported in the third quarter of 2025. The Company has designed this trial using endpoints that, pending interactions with regulatory authorities, could produce data to support submissions for product approval. Patients were randomized to receive either 100 mg of verekitug or placebo administered subcutaneously every 12 weeks over a 24-week treatment period. The primary endpoint is change from baseline in nasal polyp score (NPS) at week 24, a primary endpoint that has been used in several registrational trials for other biologic treatments for CRSwNP. Secondary endpoints include: nasal congestion score, sinus opacification, difficulty with sense of smell, nasal symptoms, percentage of participants requiring systemic corticosteroids or nasal polyp surgery, time to nasal polyp surgery and/or time to systemic corticosteroids for nasal polyps, total symptom score, and characterization of safety. Top-line data from Phase 2 clinical trial in patients with severe asthma now expected in the first half of 2026: The Company has designed this trial using endpoints that, pending interactions with regulatory authorities, could produce data to support submissions for product approval. Upstream Bio also plans to initiate a long-term safety and efficacy extension study (Phase 2 LTE) in certain adult patients with severe asthma following completion of its Phase 2 severe asthma trial with the first patient expected to transition to the LTE study in the second quarter of 2025. First patient dosing in Phase 2 clinical trial in COPD expected in mid-2025: Upstream Bio is initiating development of verekitug in a Phase 2 clinical trial in patients with moderate-to-severe COPD and now expects to dose the first patient in mid-2025. The Company has designed this trial using endpoints that, pending interactions with regulatory authorities, could produce data to support submissions for product approval. First Quarter 2025 Financial Results As of March 31, 2025, Upstream Bio had cash, cash equivalents and short-term investments of $431.4 million, which is expected to fund planned operations through 2027. Research and development expenses were $25.8 million for the quarter ended March 31, 2025, compared to $11.7 million for the same period in 2024. The increase of $14.1 million was primarily driven by an increase in clinical and manufacturing expenses related to the Company's verekitug program. General and administrative expenses were $6.8 million for the quarter ended March 31, 2025, compared to $4.0 million for the same period in 2024. The increase of $2.8 million was primarily driven by an increase in personnel-related expenses, including share-based compensation, and professional service fees. Net loss was $27.3 million for the quarter ended March 31, 2025, compared to a net loss of $10.9 million for the same period in 2024. The increase of $16.4 million was largely due to increased research and development and general and administrative expenses, partially offset by increased interest income. Upcoming Events Upstream Bio expects to participate in the following investor conferences and medical congresses: Goldman Sachs 46 th Annual Global Healthcare Conference 2025, Miami, FL, Upstream Bio presentation on June 11, 2025, at 9:20 a.m. ET Annual Global Healthcare Conference 2025, Miami, FL, Upstream Bio presentation on June 11, 2025, at 9:20 a.m. ET European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, Glasgow, United Kingdom, June 13-16, 2025 About Upstream Bio Upstream Bio is a clinical-stage biotechnology company developing treatments for inflammatory diseases, with an initial focus on severe respiratory disorders. Upstream Bio is developing verekitug, the only known antagonist currently in clinical development that targets the receptor for thymic stromal lymphopoietin, a cytokine which is a clinically validated driver of inflammatory response positioned upstream of multiple signaling cascades that affect a variety of immune mediated diseases. Upstream Bio has advanced this highly potent monoclonal antibody into separate Phase 2 trials for the treatment of severe asthma and chronic rhinosinusitis with nasal polyps and is initiating development in chronic obstructive pulmonary disease. Upstream Bio's team is committed to maximizing verekitug's unique attributes to address the substantial unmet needs for patients underserved by today's standard of care. To learn more, please visit . Upstream Bio intends to use the investor relations page on its website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor its website in addition to following press releases, filings with the Securities and Exchange Commission (SEC), public conference calls, presentations and webcasts. Forward-Looking Statements This press release contains 'forward-looking statements' within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. These statements may be identified by words such as 'aims,' 'anticipates,' 'believes,' 'continue,' 'could,' 'estimates,' 'expects,' 'forecasts,' 'goal,' 'intends,' 'may,' 'plans,' 'possible,' 'potential,' 'predict,' 'project,' 'seeks,' 'should,' 'target,' 'will' and variations of these words or similar expressions. Any statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, express or implied statements regarding: the clinical development of verekitug for the treatment of severe asthma, CRSwNP and COPD, including the initiation, timing, progress and results of ongoing and planned clinical trials; expectations for future discussions with regulatory authorities and the potential of the endpoints of the Company's clinical trials to produce data that could support submissions for product approval; expectations regarding the safety, efficacy or tolerability of verekitug; Upstream Bio's expected operating expenses and capital expenditure requirements, including its cash runway through 2027; and participation at upcoming investor conferences and medical congresses. Any forward-looking statements in this press release are based on the Company's current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Readers are cautioned that actual results, levels of activity, safety, efficacy, performance or events and circumstances could differ materially from those expressed or implied in the Company's forward-looking statements due to a variety of risks and uncertainties, which include, without limitation, risks and uncertainties related to: Upstream Bio's ability to advance verekitug through clinical development, and to obtain regulatory approval of and ultimately commercialize verekitug on the expected timeline, if at all; the initiation, timing, progress and results of clinical trials; Upstream Bio's ability to fund its development activities and achieve development goals; Upstream Bio's dependence on third parties to conduct clinical trials and manufacture verekitug, and commercialize verekitug, if approved; Upstream Bio's ability to attract, hire and retain key personnel, and protect its intellectual property; Upstream Bio's financial condition and need for substantial additional funds in order to complete development activities and commercialize verekitug, if approved; regulatory developments and approval processes of the U.S. Food and Drug Administration and comparable foreign regulatory authorities; Upstream Bio's competitors and industry; and other risks and uncertainties described in greater detail under the caption 'Risk Factors' in Upstream Bio's most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, as well as any subsequent filings with the SEC. Any forward-looking statements represent Upstream Bio's views only as of today and should not be relied upon as representing its views as of any subsequent date. Upstream Bio explicitly disclaims any obligation or undertaking to update any forward-looking statements contained herein to reflect any change in its expectations or any changes in events, conditions or circumstances on which any such statement is based except to the extent required by law, and claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. UPSTREAM BIO, INC. CONDENSED CONSOLIDATED BALANCE SHEET (IN THOUSANDS) (UNAUDITED) March 31, December 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 71,312 $ 325,892 Short-term investments 360,068 144,559 Accounts receivable 566 613 Prepaid expenses and other current assets 21,841 8,096 Total current assets 453,787 479,160 Property and equipment, net 539 582 Operating lease right-of-use assets 1,649 1,783 Restricted cash 194 194 Total assets $ 456,169 $ 481,719 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $ 4,718 $ 4,041 Accrued expenses and other current liabilities 4,141 5,992 Operating lease liabilities, current portion 708 704 Total current liabilities 9,567 10,737 Operating lease liabilities, net of current portion 992 1,130 Total liabilities 10,559 11,867 Stockholders' equity: Common stock 53 53 Additional paid-in capital 663,239 660,604 Accumulated other comprehensive income (loss) 368 (25 ) Accumulated deficit (218,050 ) (190,780 ) Total stockholders' equity 445,610 469,852 Total liabilities and stockholders' equity $ 456,169 $ 481,719 UPSTREAM BIO, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (IN THOUSANDS) (UNAUDITED) Three Months Ended March 31, 2025 2024 Collaboration revenue $ 566 $ 640 Operating expenses: Research and development 25,797 11,691 General and administrative 6,782 3,962 Total operating expenses 32,579 15,653 Loss from operations (32,013 ) (15,013 ) Other income (expense): Change in fair value of preferred stock tranche right liability — 2,859 Interest income 4,743 1,266 Other expense, net — (6 ) Total other income, net 4,743 4,119 Net loss $ (27,270 ) $ (10,894 ) Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. GlobeNewswire provides press release distribution services globally, with substantial operations in North America and Europe.
