Latest news with #SGRQ
Yahoo
19-05-2025
- Health
- Yahoo
ATS 2025: Tezepelumab reduces COPD exacerbations in eosinophilic patients, despite CB
On 18 May 2025, at the American Thoracic Society International Conference, held in San Francisco, California, post hoc findings from the Phase IIa COURSE trial (NCT04039113) shed light on the potential for tezepelumab to reduce exacerbation rates in chronic obstructive pulmonary disease (COPD) patients with elevated blood eosinophil counts (BECs), regardless of chronic bronchitis (CB) status. Tezepelumab, a thymic stromal lymphopoietin (TSLP) inhibitor developed by AstraZeneca and Amgen, demonstrated clinically relevant benefits in specific inflammatory subtypes of COPD, re-inforcing the commercial opportunity for precision biologics in this historically underserved market. The double-blind, placebo-controlled COURSE study enrolled 337 patients aged 40 to 80 with moderate to very severe COPD and two or more exacerbations in the previous year, all on optimised triple inhaled therapy. Patients were randomised 1:1 to receive tezepelumab 420mg or placebo subcutaneously every four weeks for 52 weeks. This post hoc analysis evaluated treatment outcomes stratified by the presence of St George's Respiratory Questionnaire (SGRQ)-defined chronic bronchitis and baseline BEC (<150 vs. ≥150cells/μL). In patients with CB, tezepelumab reduced the annualised rate of moderate or severe exacerbations by 26% versus placebo. The effect was most pronounced in those with baseline BEC 150cells/μL or higher, where a 28% reduction was observed. Among patients without CB, no effect was seen in those with low BECs, but a 56% reduction was observed in the elevated eosinophil subgroup. These findings underscore the role of type 2 inflammation, particularly in the context of chronic bronchitis, in modulating biologic response. Additionally, improvements in pre-bronchodilator FEV₁ were observed in tezepelumab-treated patients with CB and BEC 150cells/μL or higher. Health-related quality of life, as measured by SGRQ total score, also improved more significantly in these groups — aligning with the drug's potential to target upstream inflammatory pathways beyond eosinophils alone. These findings re-inforce the growing clinical narrative that eosinophilic inflammation is a key modulator of response to biologic therapies in COPD, echoing similar subgroup-driven efficacy patterns seen with anti–IL-5 therapies such as GSK's Nucala (mepolizumab) and AstraZeneca's Fasenra (benralizumab). While tezepelumab missed its primary endpoint in the overall COURSE population, this subgroup data supports further targeted development and suggests potential for regulatory paths through biomarker enrichment strategies. From a commercial standpoint, this data has important implications. The global COPD market remains heavily dominated by inhaled therapies, with limited penetration of biologics due to historically poor trial outcomes in unstratified populations. However, the increasing focus on eosinophilic COPD opens the door for biologic differentiation, particularly in high-risk, high-exacerbation patients who fail standard inhaled triple therapy. If further validated in Phase III trials, tezepelumab could secure a foothold as a biomarker-driven add-on therapy, potentially capturing market share from other biologics with overlapping mechanisms. Moreover, AstraZeneca's existing respiratory portfolio, including Symbicort and Fasenra, positions it well to integrate tezepelumab into commercial pathways leveraging established prescriber networks. Still, payers will likely demand compelling value propositions and stratification criteria to justify premium pricing, especially as biologic competition intensifies. The trajectory of tezepelumab will be shaped by its ability to demonstrate differentiated, biomarker-driven efficacy in a clearly defined eosinophilic population. The data from COURSE strengthens the rationale for a precision medicine strategy, but meaningful commercial success will depend on validating these subgroup effects in a pivotal Phase III programme. In a competitive landscape that now includes Sanofi and Regeneron's Dupixent's (dupilumab) first-in-class approval and emerging IL-5 agents with broader datasets, tezepelumab must establish a compelling value proposition, both clinically and economically. Key opinion leaders interviewed by GlobalData have noted that while tezepelumab's upstream mechanism offers theoretical advantages across inflammatory endotypes, its success will ultimately hinge on clear evidence of clinical superiority or meaningful label differentiation. Without this, market access may remain limited. Experts emphasised that strategic deployment will require precise positioning, alignment with companion diagnostics, and competitive pricing to secure payer reimbursement and prescriber uptake in an increasingly crowded and cost-sensitive biologics landscape. Overall, tezepelumab's emerging COPD profile may represent a pivot point for the biologics market in respiratory disease, provided future trials continue to emphasise precision targeting over broad population efficacy. "ATS 2025: Tezepelumab reduces COPD exacerbations in eosinophilic patients, despite CB" 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. 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
Business Wire
18-05-2025
- Business
- Business Wire
Savara Presented New Data From Pivotal Phase 3 IMPALA-2 Trial of Molgramostim Inhalation Solution (Molgramostim) in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) at American Thoracic Society Conference (ATS) 2025
LANGHORNE, Pa.--(BUSINESS WIRE)-- Savara Inc. (Nasdaq: SVRA) (the Company), a clinical-stage biopharmaceutical company focused on rare respiratory diseases, today announced new data in two poster presentations at the ATS International Conference 2025. Data presented were from the Phase 3 IMPALA-2 clinical trial of molgramostim in aPAP and demonstrated that molgramostim reduces surfactant burden and improves health-related quality of life outcomes in patients with aPAP. Poster Title: Molgramostim Reduces Surfactant Burden and Number of Whole Lung Lavage Procedures in Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP): Results From the IMPALA-2 Phase 3 Clinical Trial Presenter: Tisha S. Wang, M.D., Professor of Clinical Medicine, Senior Executive Clinical Vice Chair, University of California Los Angeles Department of Medicine Poster Number: 918 Poster Location: PD05, Room 3009/3011 West Building, Level 3 Summary: Molgramostim reduced surfactant burden as measured by ground-glass opacification (GGO) scores, a radiological measure of surfactant burden. The mean reduction in GGO score from baseline to Week 24 was greater in the molgramostim group (n=78) than in the placebo group (n=79) (-2.1 vs. -1.1; P=0.0004) Fewer patients in the molgramostim group required rescue whole lung lavages (WLL) compared with placebo. During the 48-week double-blind treatment period, 6 patients (7.4%) in the molgramostim group underwent a total of 15 WLLs and 11 patients (13.3%) in the placebo group underwent a total of 24 WLLs Molgramostim reduces pulmonary surfactant burden, which drives the clinical manifestations of aPAP, and provides support for the potential beneficial treatment effect of molgramostim Poster Title: The Effects of Molgramostim on Respiratory Health-related Quality of Life and Patient-reported Outcomes in Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Presenter: Ali Ataya, M.D., Associate Professor of Medicine, University of Florida, Division of Pulmonary and Critical Care Medicine Poster number: P31 Poster Location: TP22, Area A, Hall F (North Building, Exhibition Level) Summary: Molgramostim showed benefit on measures of health-related quality of life (HRQoL) and patients' assessment of breathing problems and physical activity, including changes from baseline in St. George's Respiratory Questionnaire (SGRQ) Impact and Symptom scores, the EuroQol 5 Dimensions, 5 Levels (EQ-5D-5L), Patient Global Impression of Severity (PGIS), and Patient Global Impression of Change (PGIC) at Weeks 24 and 48, which were included as exploratory endpoints in the trial Molgramostim improved respiratory HRQoL as measured by changes from baseline to Week 24 in SGRQ Impact (P=0.0084) and Symptom scores compared with placebo, and the EQ-5D-5L, a generic HRQoL instrument comprised of a short descriptive system questionnaire that allows patients to rate their health across 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Odds ratios of responses on the EQ-5D-5L numerically favored the molgramostim group on 3 of the 5 dimensions (mobility, self-care, and usual activities) at Weeks 24 and 48 Molgramostim reduced the severity of breathing problems, as assessed by PGIS, at both Weeks 24 (P=0.0305) and 48 (P=0.0049). Additionally, more molgramostim patients reported improvements in overall change in daily physical activity level, as measured by more patients assessing themselves as 'Much better' or 'A little better' compared with placebo at Week 24 (P=0.0368) and Week 48 (P=0.0193) The abstracts were published in a supplement of the American Journal of Respiratory and Critical Care Medicine. For more details about the ATS International Conference please visit their website. The posters are available on the Congresses & Publications page of the Company's corporate website. About the IMPALA-2 Trial IMPALA-2 is a global, pivotal, Phase 3, 48-week, randomized, double-blind, placebo-controlled clinical trial designed to compare the efficacy and safety of molgramostim 300 mcg self-administered once daily by inhalation with matching placebo in patients with aPAP. The trial is being conducted at 43 clinical trial sites across 16 countries, including the U.S., Canada, Japan, South Korea, Australia, and countries in Europe, including Turkey. The primary efficacy assessment was diffusing capacity of the lungs for carbon monoxide (DLCO), a gas exchange measure, and the primary endpoint was change from baseline to Week 24 in percent predicted DLCO, with a secondary endpoint of change from baseline to Week 48 in percent predicted DLCO. Three additional secondary efficacy variables evaluated clinical measures of direct patient benefit: St. George's Respiratory Questionnaire (SGRQ) Total score, SGRQ Activity score, and exercise capacity using a treadmill test, with each endpoint measured at Weeks 24 and 48. The primary time point for efficacy assessments was at Week 24; however, efficacy was assessed through Week 48 to evaluate durability of effect. Safety was assessed through Week 48. All patients who completed the 48-week double-blind treatment period continued into a 96-week open-label period during which they are receiving molgramostim 300 mcg administered once daily. About Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages. Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in aPAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas exchange, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary lung infection develops. In the long term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant. About Savara Savara is a clinical-stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim inhalation solution (molgramostim), is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim is delivered via an investigational eFlow ® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of a large molecule. Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at and LinkedIn.
Yahoo
16-05-2025
- Health
- Yahoo
ArkBio Announces Positive Phase II Results of AK3280 for Treatment of Idiopathic Pulmonary Fibrosis (IPF)
SHANGHAI, May 16, 2025 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") today announced positive top-line Phase II study results for its novel anti-fibrotic drug AK3280 in treatment of idiopathic pulmonary fibrosis (IPF). The study, led by Professor Huaping Dai of the Department of Pulmonary and Critical Care Medicine at China-Japan Friendship Hospital, Beijing, was conducted across 31 clinical sites in China. Global Clinical Challenge: Unresolved Treatment Needs in IPF Idiopathic Pulmonary Fibrosis (IPF) is a progressive, irreversible, and fatal interstitial lung disease characterized by fibrotic remodeling of lung tissue, ultimately leading to respiratory failure, with a median survival period of only 2–5 years after diagnosis. Current therapeutics including pirfenidone and nintedanib have been available for years, however, with limited efficacy, significant adverse effects and poor long-term tolerability, highlighting the urgent need for novel and more effective therapeutics. Phase 2 Proof-of-Concept Study of AK3280, a New Generation Broadly Active Antifibrotic Drug AK3280 is a new generation broadly active antifibrotic drug that is optimized for pharmacological and pharmacokinetic properties based on current IPF drugs. Preclinical data indicate that it exhibits enhanced anti-fibrotic activity and improved pharmacokinetic properties, without the gastrointestinal tolerability issues and other toxicities associated with current therapeutics. Previous Phase I studies have demonstrated its favorable safety, tolerability, and human pharmacokinetic profile. The completed randomized, double-blind, placebo-controlled Phase II proof-of-concept study evaluated the safety, tolerability and clinical efficacy of AK3280 in China IPF patients. Participants were randomized to receive AK3280 (100/200/300/400 mg BID) or placebo for 24 weeks. Clinical efficacy endpoints included forced vital capacity of lung (FVC and %pFVC), diffusing capacity of lung for carbon monoxide (DLco), the 6-minute walk test (6MWT) and patient reported St. George's Respiratory Questionnaire (SGRQ) scores. The high dose groups demonstrated FVC improvement from baseline, especially the 400 mg group which had the absolute FVC increased by 209.4 mL and a 6.4% adjusted %pFVC improvement from baseline, which is statistically significant compared to placebo (p=0.002 and 0.004, respectively). Other lung and respiratory functions have also got better. The drug exhibited a good safety and tolerability profile without the gastrointestinal intolerability issues associated with current IPF therapeutics. Implications for Future IPF Therapeutics This study with a rigorous multicenter, randomized, double-blind study design for 24 weeks followed by another 24 week's open-label study represents the first phase 2 proof-of-concept study of AK3280 in fibrotic patient populations. These findings not only demonstrate AK3280's potential to improve both lung function and respiratory outcome but also highlight its unique safety profile that may support long-term use, positioning it as a potential future standard-of-care therapy for IPF treatment. Dr. Huaping Dai, principal investigator of the phase 2 study and professor at China-Japan Friendship Hospital, commented, "In the therapeutic landscape of IPF, the development of safer and more effective anti-fibrotic agents remains urgent. The most encouraging aspect of this Phase II study is AK3280's positive signal in pulmonary function improvement. Unlike existing therapies that merely slow FVC decline, the high dosing groups of the phase 2 study achieved an impressive absolute increase in FVC over 24 weeks. In addition, we observed other respiratory and lung function improvements, indicating the substantial symptom relief and benefits to quality-of-life in the IPF patients. Notably, AK3280's favorable tolerability across all dose levels is essential for the long-term management of IPF patients. This study provides novel scientific and medical perspectives, and we look forward to seeing this innovation to become a global standard treatment option in the near future." About AK3280 AK3280 is a potential next-generation broad-spectrum anti-fibrotic molecule optimized from the marketed drug. It modulates multiple pathways and biomarkers closely associated with the fibrotic process, including the expression of fibrosis-related genes and proteins induced by transforming growth factor-beta (TGF-β) and lysophosphatidic acid (LPA). AK3280 works by reducing fibroblast cellular proliferation and inhibiting the synthesis and accumulation of extracellular matrix. Compared to current therapies, AK3280 offers advantages in safety and tolerability, with potentially much improved clinical efficacy. The Phase II randomized, double-blind, placebo-controlled confirmatory clinical study has been completed, and preparations are underway to initiate the pivotal Phase III clinical study. About ArkBio ArkBio is a global biotech company focused on developing innovative therapies for respiratory, infectious, and pediatric diseases. Founded in 2014, it has built core technology platforms and a differentiated R&D pipeline through in-house R&D efforts and external collaboration. Key drug assets include ziresovir, the first direct-acting RSV antiviral with positive pivotal phase III results, and AK0901, an FDA-approved pediatric ADHD therapeutic drug. ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology of Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture capital institutions. For more information about the company, please visit our website: Investor Inquiries: IR@ View original content to download multimedia: SOURCE Arkbio 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
