Latest news with #Sjogren
Business Insider
2 days ago
- Business
- Business Insider
Argenx presents new Efgartigimod data at EULAR 2025
argenx (ARGX) SE 'announced the presentation of positive results from Phase 2 studies evaluating VYVGART in Sjogren's disease and idiopathic inflammatory myopathies at the European Congress of Rheumatology, EULAR 2025, from June 11 – 14 in Barcelona, Spain. argenx also announced that the FDA has granted efgartigimod Fast Track designation for the treatment of primary Sjogren's disease.' Confident Investing Starts Here: Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
Yahoo
3 days ago
- Business
- Yahoo
argenx Presents New Efgartigimod Data at EULAR 2025 Highlighting Positive Phase 2 Proof-of-Concept Results in Myositis and Sjogren's Disease
ALKIVIA data demonstrate significant improvement in muscle strength and physical function in myositis patients treated with efgartigimod RHO data show efgartigimod achieved sustained reduction in autoantibodies and improved functional outcomes in patients with Sjogren's disease; program granted U.S. FDA Fast Track designation argenx committed to new therapeutic areas in rheumatology with ongoing Phase 3 studies in myositis (ALKIVIA) and Sjogren's disease (UNITY) June 11, 2025, 12:01 AM CET Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced the presentation of positive results from Phase 2 studies evaluating VYVGART® (IV: efgartigimod alfa-fcab and SC or Hytrulo: efgartigimod alfa and hyaluronidase-qvfc) in Sjogren's disease (SjD) and idiopathic inflammatory myopathies (IIM or myositis) at the European Congress of Rheumatology, EULAR 2025, from June 11 – 14 in Barcelona, Spain. argenx also announced that the U.S. Food and Drug Administration (FDA) has granted efgartigimod Fast Track designation (FTD) for the treatment of primary Sjogren's disease. 'Our innovation model prioritizes strong biologic rationale and efficient clinical program design, which enables us to rapidly advance development in rheumatic diseases,' said Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. 'The accumulating body of evidence about the role of IgG autoantibodies reinforces the therapeutic potential of efgartigimod as a new approach for treating several rheumatic diseases – aiming to go beyond symptom management by targeting the underlying disease. The data presented at EULAR highlight efgartigimod's potential as a precision therapy for patients living with myositis and Sjogren's disease, and we are hopeful this novel treatment will offer a new therapeutic option and lead to improved outcomes for patients.' ALKIVIA Data Show Efgartigimod Provides Functional Improvement in Patients with Myositis Consistent and statistically significant treatment effect: In the ongoing, seamless ALKIVIA Phase 2/3 study evaluating three myositis subtypes (IMNM, ASyS, DM), data from Phase 2 show that patients demonstrated significant improvement in muscle strength and physical function when treated with efgartigimod. The study's primary endpoint, mean Total Improvement Score (TIS) at 24 weeks, is a composite of six core measures of disease activity and muscle function. TIS improvement was observed in a majority of efgartigimod-treated patients across all six core measures, and the primary endpoint was met. Efgartigimod patients showed a significantly higher mean TIS of 50.45 compared to 35.65 in the placebo arm (2-sided P=0.0004). In addition, for patients treated with efgartigimod, 79% achieved a moderate improvement (TIS ≥40) and 34% achieved a major improvement (TIS ≥60), compared to 47% and 9.5% respectively of patients receiving placebo. Favorable Time to TIS and Safety Profile: Among the study's secondary endpoints, patients receiving efgartigimod improved significantly faster than patients receiving placebo, leading to a median time to minimal improvement (TIS ≥20) of 30 days and time to moderate improvement (TIS ≥40) of 16 weeks. Comparatively, patients in the placebo arm reached minimal improvement (TIS ≥20) in 72 days, while there was no majority of placebo patients reaching moderate improvement (TIS ≥40) at any point in the 24-week study. Efgartigimod was well-tolerated and the proportion of patients experiencing at least one treatment-emergent adverse event (TEAE) was similar in the efgartigimod and placebo arms. Evaluation of efgartigimod in myositis is ongoing in the Phase 3 portion of the ALKIVIA study. 'Myositis is a debilitating disease that can cause muscle weakness, affect multiple organs, and have a severe impact on patients' quality of life. Physicians struggle to treat it because current options are limited and have significant side effects,' said Hector Chinoy, Ph.D., ALKIVIA study investigator and Professor of Rheumatology and Neuromuscular Disease at The University of Manchester. 'Results from this study, the first of an FcRn inhibitor in myositis, demonstrate the potential of a transformative targeted treatment approach. Efgartigimod was well-tolerated and led to significant improvements compared to placebo, offering new hope for a treatment that targets autoantibodies as one of the potential key drivers of disease.' RHO Data Show Efgartigimod's Clinical Effect Across Endpoints in Sjogren's Disease Improved systemic disease activity and reduction in symptoms: In the Phase 2 proof-of-concept RHO study, efgartigimod showed significant improvement in systemic disease activity and patient symptoms. 45.5% of patients receiving efgartigimod achieved improved outcomes on the CRESS composite primary endpoint at Week 24 – including systemic disease activity, salivary and tear gland function – compared to 11.1% among patients treated with placebo. Improvements among patients treated with efgartigimod were achieved in 4 out of 5 CRESS measures. In addition, disease activity among patients treated with efgartigimod showed a median change in clinESSDAI total score of -7.0 versus -4.0 in the placebo arm. A key secondary endpoint is the cSTAR composite of five disease measures, which showed patients treated with efgartigimod achieved a 54.5% response versus 33.3% in the placebo arm. Potential for disease biology modulation: Biomarker response in RHO study also demonstrated rapid and sustained reduction of IgG with a ~60% reduction from Week 4 onwards. The efgartigimod group showed notable decreases in the disease-associated antibodies anti-Ro52 (-57% vs +13%) and Rheumatoid Factor (-26.6% vs -5.3%), as well as reduction in C1Q immune complexes (-4.5 vs -0.06 mc eq/mL) compared to placebo. Efgartigimod demonstrated a favorable safety profile among patients with Sjogren's disease. The observed safety and tolerability was consistent with other clinical trials, with no new safety signals observed. The Phase 3 UNITY trial is currently ongoing to assess efficacy and safety of efgartigimod in patients with moderate to severe Sjogren's disease. 'These data suggest that targeting FcRn and reducing IgGs has a meaningful impact on Sjogren's disease,' said Isabelle Peene M.D., Ph.D., study investigator, Department of Rheumatology, Ghent University Hospital. 'The clinical and biomarker findings add to our growing understanding of IgG autoantibodies in Sjogren's disease and could inform future treatment strategies for this complex, progressive and underserved condition.' More information on the data presented at the EULAR 2025 meeting can be found for presentations are as follows: Title Presenter Presentation Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants with Active Idiopathic Inflammatory Myopathy: Phase 2 Results from the ALKIVIA Study Hector Chinoy Oral Presentation #OP0002Session: Abstract PlenaryWednesday, June 1116:40-16:50 CEST Treatment of Sjögren's disease by blocking FcRn: clinical and translational data from Rho, a phase 2 randomized, placebo controlled, double-blind, proof-of-concept study with efgartigimod Isabelle Peene Oral Presentation #OP0041Session: Clinical AbstractWednesday, June 1116:30-16:40 CEST Efficacy and safety of efgartigimod PH20 subcutaneous by prefilled syringe in adults with Sjögren's disease: A Phase 3, randomized, double-blind, placebo-controlled, multicenter trial with open-label extension (UNITY) Simon Bowman Poster #POS0844Poster View IIIThursday, June 1212:00-13:30 CEST Safety, tolerability, and efficacy of empasiprubart in adults with dermatomyositis (EMPACIFIC): A Phase 2, randomized, double-blind, placebo-controlled, multicenter study Tetyana Storie Poster #POS1049Poster View VIFriday, June 1312:00-13:30 CEST ALKIVIA Study DesignThe ALKIVIA study is a randomized, double-blind, placebo-controlled, multicenter, operationally seamless Phase 2/3 study of efgartigimod SC for the treatment of idiopathic inflammatory myopathies (IIM or myositis) across three subtypes, including immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and dermatomyositis (DM). The ALKIVIA study enrolled 240 patients in total and is being conducted in two phases, with an analysis of the Phase 2 portion of the clinical trial after the first 90 patients completed the study, followed by a Phase 3 portion if a signal is observed in the Phase 2 portion. The primary endpoint is the mean total improvement score (TIS) at the end of the treatment period (24 weeks in Phase 2 and 52 weeks in Phase 3) of all treated patients (IMNM, ASyS, DM) compared to placebo. Key secondary endpoints include response rates at the end of treatment, time to response, and duration of response in TIS, as well as change from baseline in individual TIS components. Other secondary endpoints include quality of life and other functional scores. About Idiopathic Inflammatory MyopathiesIdiopathic inflammatory myopathies (myositis) are a rare group of autoimmune diseases that can be muscle specific or affect multiple organs including the skin, joints, lungs, gastrointestinal tract and heart. Myositis can be very severe and disabling and have a material impact on quality of life. Initially, myositis was classified as either DM or polymyositis, but as the underlying pathophysiology of myositis has become better understood, including through the identification of characteristic autoantibodies, new polymyositis subtypes have emerged. Two of these subtypes are IMNM and ASyS. Proximal muscle weakness is a unifying feature of each subtype. IMNM is characterized by skeletal muscle weakness due to muscle cell necrosis. ASyS is characterized by muscle inflammation, inflammatory arthritis, interstitial lung disease, thickening and cracking of the hands ('mechanic's hands') and Raynaud's phenomenon. DM is characterized by muscle inflammation and degeneration and skin abnormalities, including heliotrope rash, Gottron's papules, erythematous, calcinosis and edema. RHO Study DesignThe Phase 2 RHO study was a randomized, double-blinded, placebo-controlled multicenter proof of concept study to evaluate the safety and efficacy of efgartigimod in adults with Sjogren's Disease. In order to enter the study, patients needed to test positive for anti-Ro autoantibodies and maintain residual salivary flow. Thirty four patients were randomized 2:1 to receive either efgartigimod or placebo for up to 24 weeks. Multiple endpoints and biomarkers were evaluated in the signal-finding study, including the primary endpoint of CRESS (Composite of Relevant Endpoints for Sjogren's Syndrome). Within CRESS there are five components spanning: systemic disease activity as measured by the ESSDAI (EULAR Sjogren's Syndrome Activity Index), patient reported outcomes as measured by the ESSPRI (EULAR Sjogren's Syndrome Patient Reported Index), tear and salivary gland function and serology. To be a CRESS responder, patients needed to demonstrate a clinically meaningful benefit in at least 3 of the 5 composite items. Additional datapoints were gathered including the clinESSDAI, STAR (Sjogren's Tool for Assessing Response), biomarker data, and the change in lymphocytic infiltrate levels through parotid biopsies. About Sjogren's DiseaseSjogren's Disease (SjD) is a chronic, slowly progressive inflammatory systemic autoimmune disease characterized by immune-mediated destruction of exocrine glands. SjD can be severely debilitating and have a negative impact on patient quality of life, with common symptoms reported as dry eyes and mouth, fatigue, and joint point. In addition, a substantial subset of patients suffer from extraglandular systemic disease. While the presence of anti-Ro and anti-La IgG autoantibodies are considered a hallmark of disease, the underlying cause of SjD is believed to be multi-factorial, triggered by environmental factors, leading to autoimmunity and chronic inflammation. SjD predominantly impacts women with a 9:1 female:male incidence ratio. Given the heterogeneous nature of the disease, the treatment journey can be challenging with long delays and high rates of misdiagnosis. There are no FDA- approved treatments targeting the disease itself, leaving current treatments to focus primarily on individual symptom management. About EfgartigimodEfgartigimod (efgartigimod alfa and hyaluronidase-qvfc) is a human IgG1 antibody fragment designed to reduce pathogenic immunoglobulin G (IgG) antibodies by binding to the neonatal Fc receptor (FcRn) and blocking the IgG recycling process. Efgartigimod is the first-approved FcRn blocker globally and is marketed as VYVGART® and VYVGART® Hytrulo in the United States and China for the treatment of generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP), and as VYVDURA (Japan) or VYVGART SC for gMG in other regions globally. Efgartigimod is currently being evaluated in more than 15 severe autoimmune diseases where pathogenic IgGs are believed to be mediators of disease. About argenxargenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker and is evaluating its broad potential in multiple serious autoimmune diseases while advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit and follow us on LinkedIn, Instagram, Facebook, and YouTube. For further information, please contact: Media: Colin McBeancmcbean@ Investors: Alexandra Royaroy@ Forward-looking Statements The contents of this announcement include statements that are, or may be deemed to be, 'forward-looking statements.' These forward-looking statements can be identified by the use of forward-looking terminology, including the terms 'aim,' 'are,' 'believe,' 'can,' 'commit,' and 'will' and include statements argenx makes concerning its commitment to improving the lives of people suffering from severe autoimmune diseases and to new therapeutic areas in rheumatology; the discussion of its Phase 2 proof-of-concept results as well as the ongoing Phase 3 studies for efgartigimod in myositis and Sjogren's disease at EULAR 2025, including the planned agenda of such congress; its ability to rapidly advance development in rheumatic diseases; its goal to have efgartigimod not just manage symptoms but target the underlying disease and its potential as a precision therapy for myositis and Sjogren's patients; the potential for efgartigimod to be a transformative targeted treatment approach; and its hope that efgartigimod leads to improved outcomes and offer a new therapeutic options for such patients. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx's actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including but not limited to, the results of argenx's clinical trials; expectations regarding the inherent uncertainties associated with the development of novel drug therapies; preclinical and clinical trial and product development activities and regulatory approval requirements; the acceptance of its products and product candidates by its patients as safe, effective and cost-effective; the impact of governmental laws and regulations, including tariffs, export controls, sanctions and other regulations on its business; its reliance on third-party suppliers, service providers and manufacturers; inflation and deflation and the corresponding fluctuations in interest rates; and regional instability and conflicts. A further list and description of these risks, uncertainties and other risks can be found in argenx's U.S. Securities and Exchange Commission (SEC) filings and reports, including in argenx's most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by 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
28-04-2025
- Health
- Yahoo
NHS letting patients ‘fall through the cracks'
The health service in the UK is 'not working' for people with rare diseases and they are being left to 'fall through the cracks', according to a new report. Almost three in 10 (30%) people with certain uncommon conditions say they waited for five years after their symptoms started before they were diagnosed with their condition. Even after diagnosis, many patients are left facing 'poor care co-ordination' and face difficulties accessing information and support, the Rare Autoimmune Rheumatic Disease Alliance (RAIRDA) said. And people with rare diseases see drastic changes in the way they are cared for based on where they live, according to a new report from the Alliance. The report claims that the UK health system has had an 'increasing focus' on major and common conditions, but people with these rare conditions are being left to 'fall through the cracks'. Rare autoimmune rheumatic diseases are a group of conditions, including: lupus, Sjogren's, vasculitis, scleroderma and Raynaud's, where the immune system becomes overactive and attacks the body's healthy tissues. As part of the report, Ipsos surveyed 1,300 people with these rare conditions in the UK and found: – On average, patients waited for 31 months from symptoms to diagnosis, but this varied considerably between conditions. Many people said that they were initially misdiagnosed with other conditions before they received their diagnosis. – Almost one in 10 (9%) said that after diagnosis, they waited for a year before seeing a specialist. The average wait to see a specialist was five months. The Alliance said treatment needs to 'begin rapidly to prevent unnecessary disease progression'. – After diagnosis, 5% of people said they were responsible for co-ordinating their care, while 9% said they did not know who was in charge. – Only a quarter (26%) said they felt their GP understood their condition. RAIRDA said that the Government's upcoming 10-year plan for health represents a 'genuine opportunity' to make improvements in care to make sure that people living with rare diseases are not left feeling 'totally alone with their disease'. The report makes a series of recommendations, including: the expansion of specialised networks; the reduction in waiting times for diagnosis; giving greater access to support; and improving people's experiences of how they are cared for. Sue Farrington, co-chairwoman of RAIRDA, said: 'As this report shows, across the UK, people with RAIRDs are not getting the care they need. 'The evidence echoes the stories we hear every day from our patient communities – the UK's health system is not working for people living with RAIRDs, and they are falling through the cracks. 'These findings are perhaps not unsurprising, in a system where there has been an increasing focus on major and common conditions. 'The UK Rare Diseases Framework and subsequent action plans have enabled a significant step for rare conditions, but more is needed.' A Department of Health and Social Care spokesperson said: 'We know that those living with rare diseases and their families face immense everyday challenges. 'Improving co-ordination of care for people with rare diseases is a priority, and in our recent Rare Diseases Action Plan we set out plans to achieve this. 'More widely, our Plan for Change will transform the NHS by driving down waiting lists and investing in quality facilities to ensure all patients – including those with rare diseases – receive the care and treatment they deserve.'
South China Morning Post
24-04-2025
- Health
- South China Morning Post
How wellness retreat helps Filipino influencer cope with Sjogren's syndrome
Popular Filipino influencer Pinky Tobiano recognised that, after years of struggling with Sjogren's syndrome, she needed help to reset her mind and body. Advertisement At the age of 40, she was diagnosed with thyroid cancer. She beat the disease, but the prolonged radiation therapy to treat the cancer led to her developing Sjogren's, a little-known chronic autoimmune condition in which the body's immune system attacks the moisture-producing glands, primarily in the eyes and mouth. 'Living with Sjogren's syndrome is difficult,' shares Tobiano, now 55 and in Manila in the Philippines. She is a chemist by training and an entrepreneur with more than 4.3 million followers on her Instagram account, @pinkytobiano. 'I have no saliva or tears, so I have trouble swallowing food,' she says. 'Just eating is painful and exhausting.' Pinky Tobiano has more than 4.3 million followers on her Instagram account. Photo: Instagram/pinkytobiano Other symptoms have worsened over time.
Gulf Business
09-04-2025
- Health
- Gulf Business
Using AI for medical diagnosis? What you should know about its safety
Image credit: Getty Images Artificial intelligence models may recommend different treatments for the same medical condition based solely on a patient's socioeconomic and demographic characteristics, researchers warn. The researchers invented nearly three dozen different patients and asked nine healthcare large language AI models how each one should be managed, in a thousand different emergency room situations. How does AI alter decisions? Despite identical clinical details, the AI models occasionally altered decisions based on patients' personal characteristics, affecting priority for care, diagnostic testing, treatment approach, and mental health evaluation, the researchers reported in Nature Medicine. Read- For example, advanced diagnostic tests such as CT scans or MRI were more often recommended for high-income patients, while low-income patients were more frequently advised to undergo no further testing, somewhat mimicking real-world healthcare inequities. The problems were seen in both proprietary and open-source AI models, the researchers found. AI in healthcare: Responsibility that comes with its usage 'AI has the power to revolutionise healthcare, but only if it's developed and used responsibly,' study co-leader Dr Girish Nadkarni of the 'By identifying where these models may introduce bias, we can work to refine their design, strengthen oversight, and build systems that ensure patients remain at the heart of safe, effective care,' added coauthor Dr Eyal Klang, also of the Icahn School. Potential fixes for Sjogren's saliva and tears symptoms Researchers are closer to being able to fix the life-altering dryness of the mouth and eyes that afflicts patients with Sjogren's syndrome, based on success of two approaches tested in mice. The symptoms of the autoimmune disorder can make it hard to speak, eat and sleep. But exactly how the disease shuts down the body's production of tears and saliva has been a mystery until now, researchers reported in the International Journal of Oral Science. Their new study found that early in the progression of Sjogren's syndrome, a protein called tricellulin, which clasps together the cells of the glands that produce tears and saliva, is destroyed. Loss of the tight cellular junctions results in inadequate saliva secretion, the researchers found. Two possible interventions – an investigational drug (AT1001) and an experimental molecule – each restored saliva secretion in the mice, one by repairing the cell junctions and the other by stopping the breakdown of the junctions before it began. Both restored normal gland function, offering a potential blueprint for human treatment, the researchers said. 'This changes how we think about treating Sjogren's syndrome,' study leader Dr Xin Cong of Peking University said in a statement. 'We're moving beyond simply calming inflammation. Now we can fix the actual structural damage in the glands,' Xin said. 'What's even more encouraging is that both approaches worked, which gives us real confidence in developing patient-ready therapies.' Experimental drug shows promise for one type of MS An experimental drug originally developed to treat lymphomas is the first-ever to show an effect against a form of multiple sclerosis for which no approved treatments are available, researchers reported at the American Academy of Neurology meeting in San Diego. Sanofi's tolebrutinib, an investigational oral Bruton's tyrosine kinase inhibitor, demonstrated a 31 per cent delay in the onset of six-month confirmed disability progression in patients with non-active, non-relapsing secondary progressive multiple sclerosis, in a clinical trial. 'This is the first clinical trial showing a positive effect in delaying disability progression in non-relapsing SPMS, a later form of the disease where neurological function gradually worsens over time and disability increases relentlessly,' study leader Dr Robert Fox of the Cleveland Clinic said in a statement. With 1,131 patients enrolled in the trial, the rate of confirmed disability progression at six months was 22.6 per cent in the tolebrutinib group versus 30.7 per cent in the placebo group, according to a report of the study published in The New England Journal of Medicine. More patients receiving tolebrutinib achieved improvement in disability, with a six-month confirmed disability improvement rate of 8.6 per cent versus 4.5 per cent with placebo, the researchers also reported. Markers of disease activity, including inflammation and tissue damage, also were reduced with tolebrutinib compared with placebo. Serious adverse events, particularly liver complications, were more frequent with tolebrutinib, which is currently under review for potential US approval. 'It appears that about one in 200 patients will have severe elevation of liver enzymes during the first three months of use, so careful monitoring is important, and the drug should be stopped immediately in those with liver enzyme elevations,' Fox said. Separately, in two studies of patients with relapsing multiple sclerosis, tolebrutinib was not superior to Sanofi's Aubagio (teriflunomide) in decreasing annualised relapse rates, according to a second report in the same journal.
