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Yahoo
30-06-2025
- Business
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argenx Advances Clinical Development of ARGX-119 in Congenital Myasthenic Syndromes
Phase 1b study supports proof-of-concept in DOK7 congenital myasthenic syndromes Decision informed by favorable safety profile and consistent functional improvement over time across multiple efficacy measures Advancing ARGX-119 further validates strong track record of Immunology Innovation Program (IIP), argenx's collaborative discovery model June 30, 2025, 7:00 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 its plan to advance the clinical development of ARGX-119, a first-in-class agonist antibody to muscle-specific kinase (MuSK), to a registrational study in patients with congenital myasthenic syndromes (CMS) following the analysis of topline data from the Phase 1b study. Detailed results will be presented at a future medical meeting. 'The results of our Phase 1b ARGX-119 study in congenital myasthenic syndromes, an ultra-rare disorder that affects patients from birth, builds on our experience and understanding of myasthenic disorders and aligns with our aspiration to serve even more patients living with these debilitating diseases,' said Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx. 'ARGX-119 is the sixth molecule developed through our Immunology Innovation Program to show proof-of-concept, reflecting the strength of our innovation model where our deep knowledge of the biology and expertise in antibody engineering come together to push the boundaries of what's possible. argenx remains focused on uncovering new biological insights into misunderstood diseases to meaningfully change the lives of patients who have long-been underserved,' said Peter Ulrichts, Ph.D., Chief Scientific Officer of argenx. The decision to advance the development of ARGX-119 in CMS is supported by the results of the Phase 1b study. ARGX-119 demonstrated a favorable safety and tolerability profile, which was the primary endpoint. Efficacy was evaluated across multiple secondary and exploratory endpoints, including Six-Minute Walk Test (6MWT), Quantitative Myasthenia Gravis (QMG) score and Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Consistent improvements were observed in treated DOK7-CMS patients through the 12-week study across multiple efficacy scores. Phase 1b CMS Study Design The Phase 1b, multicenter, randomized, double-blinded, placebo-controlled clinical trial assessed safety, tolerability, PK, immunogenicity, and preliminary efficacy of ARGX-119 in participants with DOK7-CMS. The study was also designed to demonstrate proof-of-biology through preliminary efficacy assessments, including muscle weakness, fatigability, daily activities and patient-reported global health outcomes. The clinical trial spanned approximately 11 months across a screening period (up to 28 days), a 12-week treatment period and a follow-up period of nearly seven months. At baseline, eligible participants were randomized 4:1 to receive intravenous ARGX-119 or placebo. The primary objective was to evaluate safety and tolerability; secondary objectives included PK, immunogenicity and preliminary efficacy of ARGX-119 in participants with DOK7-CMS. All but one of the patients enrolled in the Phase 1b study also participated in an observational natural history study initiated by argenx in 2024 to better understand the CMS patient journey and disease burden, helping to inform future development plans. About Congenital Myasthenic Syndromes Congenital Myasthenic Syndromes (CMS) are an ultra-rare and heterogenous group of congenital neuromuscular disorders caused by genetic defects that are essential for the integrity of the neuromuscular junction. Early age of onset and fatigable muscle weakness are considered clinical hallmarks of CMS. Muscle weakness can be debilitating and life-threatening causing difficulties in speaking or swallowing, impaired or absent mobility, proximal arm and leg weakness, and respiratory insufficiency. DOK7 variations are one of the more frequent and severe causes of CMS, accounting for approximately 24% of CMS cases. There are no approved treatments. The prevalence of CMS is estimated to be 5 per 1M (DOK7-CMS estimated to be 1.2 per 1M). About ARGX-119 ARGX-119 is a first-in-class humanized agonist monoclonal antibody (mAb) that specifically targets and activates muscle-specific tyrosine kinase (MuSK) to promote maturation and stabilization of the neuromuscular junction (NMJ). It is a mAb derived from llamas and discovered using the argenx SIMPLE Antibody™ platform technology. ARGX-119 is being developed for patients with neuromuscular disease, including congenital myasthenic syndromes (CMS), amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). ARGX-119 was developed through argenx's IIP program in collaboration with the world's leading key opinion leaders on MuSK and the NMJ, Professor Steven J. Burden from MGH, Professor Shohei Koide from NYU and Professor Jan Verschuuren and Associate Professor Maartje Huijbers from LUMC. About argenx argenx 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. Media: Colin McBeancmcbean@ Investors: Alexandra Roy aroy@ 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 'aims,' 'committed,' or 'plan' and include statements argenx makes concerning its commitment to improving the lives of people suffering from severe autoimmune diseases; its plan to advance the clinical development of ARGX-119 to a registrational study in CMS patients; and its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. 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
10-06-2025
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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
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28-04-2025
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argenx Announces Positive CHMP Opinion for VYVGART (efgartigimod alfa) Subcutaneous Injection for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
VYVGART® is first-and-only targeted IgG Fc-antibody fragment for CIDP First novel mechanism of action for CIDP treatment in more than 30 years CHMP positive opinion based on ADHERE data, the largest ever CIDP clinical trial European Commission (EC) decision on marketing authorization application (MAA) expected within approximately two months April 28, 2025, 07:00 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 Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended European Commission (EC) approval of VYVGART® 1000mg (efgartigimod alfa) for subcutaneous (SC) injection as a monotherapy for the treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins. 'Our mission is to develop innovative, targeted treatments for patients with rare and severe autoimmune diseases, who continue to face significant unmet needs. The positive CHMP opinion for VYVGART in CIDP brings us one step closer to providing patients across Europe with a transformational new treatment option that provides meaningful functional improvement,' said Luc Truyen M.D., Ph.D., Chief Medical Officer, argenx. 'VYVGART is the first and only targeted IgG Fc-antibody fragment for CIDP and if approved, would mark the first treatment in Europe with a novel, precision mechanism of action for CIDP patients in 30 years.' VYVGART for subcutaneous injection is available as a vial or prefilled syringe and can be administered by a patient, caregiver, or healthcare professional. Treatment is initiated with a weekly dose regimen and may be adjusted to every other week based on clinical evaluation. The CHMP recommendation is based on positive results from the ADHERE clinical trial, the largest study of CIDP patients to date. In the ADHERE study, 66.5% (214/322) of patients treated with VYVGART, regardless of prior treatment, demonstrated evidence of clinical improvement, including improvements in mobility, function and strength. ADHERE met its primary endpoint (p<0.0001) demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse versus placebo. The study also demonstrated functional improvements across the Inflammatory Neuropathy Cause and Treatment (INCAT) disability scores (>1-point), grip strength (>17 kPa) and I-RODS scale (>8 points) at week 36 compared to baseline at entry to standard of care withdrawal phase. Ninety-nine percent of trial participants elected to participate in the ADHERE open-label extension. The safety results were generally consistent with the known safety profile of VYVGART in previous clinical studies. "For the patient population represented by the European Patient Organisation for Dysimmune and Inflammatory Neuropathies (EPODIN) and for those affected by CIDP, this is excellent news," said Jean-Philippe Plançon, President of EPODIN. "There are still considerable unmet medical needs in the management of CIDP, and the CHMP's recommendation brings renewed hope for improved treatment options and quality of life." The positive CHMP opinion is a scientific recommendation for marketing authorization, serving as a basis for the EC's final decision on argenx's CIDP application for subcutaneous VYVGART. The EC is expected to make a decision following CHMP recommendation and the decision will apply to all 27 European Union Member States, and also to Iceland, Norway and Liechtenstein. Currently, VYVGART is indicated as an add-on to standard therapy for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. About ADHERE The ADHERE trial was a multicenter, randomized, double-blind, placebo-controlled trial evaluating SC efgartigimod alfa for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP). ADHERE enrolled 322 adult patients with CIDP, 130 of whom were based in Europe, who were off treatment (not on active treatment within the past six months or newly diagnosed) or being treated with immunoglobulin therapy or corticosteroids. The trial consisted of an open-label Stage A followed by a randomized, placebo-controlled Stage B. In order to be eligible for the trial, the diagnosis of CIDP was confirmed by an independent panel of experts. Patients entered a run-in stage, where any ongoing CIDP treatment was stopped and in order to be eligible for Stage A had to demonstrate active disease, with clinically meaningful worsening on at least one CIDP clinical assessment tool, including INCAT, I-RODS, or mean grip strength. Treatment naïve patients were able to skip the run-in period with proof of recent worsening. To advance to Stage B, patients needed to demonstrate evidence of clinical improvement (ECI) with SC efgartigimod alfa. ECI was achieved through improvement of the INCAT score, or improvement on I- RODS or mean grip strength if those scales had demonstrated worsening during the run-in period. In Stage B, patients were randomized to either SC efgartigimod alfa or placebo for up to 48 weeks. The primary endpoint was measured once 88 total relapses or events were achieved in Stage B and was based on the hazard ratio for the time to first adjusted INCAT deterioration (i.e. relapse). After Stage B, all patients had the option to roll-over to an open-label extension study to receive SC efgartigimod alfa. About Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. There is increasing evidence that IgG antibodies play a key role in the damage to the peripheral nerves. People with CIDP experience fatigue, muscle weakness and a loss of feeling in their arms and legs that can worsen over time or may come and go. These symptoms can significantly impair a person's ability to function in their daily lives. Without treatment, one-third of people living with CIDP will need a wheelchair. There are an estimated 31,413 people living with CIDP in the European Union. About EfgartigimodEfgartigimod is an antibody fragment designed to reduce pathogenic immunoglobulin G (IgG) antibodies by binding to the neonatal Fc receptor and blocking the IgG recycling process. Efgartigimod is being investigated in several autoimmune diseases known to be mediated by disease-causing IgG antibodies, including neuromuscular disorders, blood disorders, and skin blistering diseases, in both an IV and SC formulation. SC efgartigimod is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE® drug delivery technology. In August 2022, efgartigimod received approval from the EC for IV administration as an add on to standard therapy for the treatment of adult patients with gMG who are AChR antibody positive. About argenx argenx 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. Contacts Media:Kate Dion kdion@ 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,' 'continue,' 'expect,' 'may,' and 'will' and include statements argenx makes concerning the expected timing and decision of the EC regarding VYVGART for SC injection for CIDP treatment and the application of such decision; the potential for improved treatment options and quality of life; and its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. 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 to access your portfolio
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08-04-2025
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argenx Highlights VYVGART Data at AAN 2025 Setting New Standard in Sustained Efficacy and Improved Quality of Life Measures for Patients Living with gMG and CIDP
ADAPT-NXT data demonstrate consistent, sustained disease control across dosing schedules, further supporting individualized VYVGART dosing for gMG patients ADHERE+ oral presentation features long-term CIDP data demonstrating VYVGART Hytrulo's durable efficacy, sustained functional improvements and favorable safety profile argenx continues to advance a robust neuromuscular pipeline of clinical candidates; first-in-human data of ARGX-119 (MuSK agonist) support pipeline-in-a-product development plan April 8, 2025, 7:00 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 15 abstracts, including an oral presentation, at the 2025 American Academy of Neurology (AAN) Annual Meeting from April 5 – 9, 2025 in San Diego, CA. The presentations showcase long-term data of VYVGART® (IV: efgartigimod alfa-fcab and SC or Hytrulo: efgartigimod alfa and hyaluronidase-qvfc) demonstrating sustained disease control of generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP) with a favorable safety profile. argenx also highlighted its commitment to reach the broader MG patient community with two ongoing label expansion studies in ocular myasthenia gravis (oMG) and seronegative MG (snMG). In addition, first-in-human data were presented for the company's third clinical candidate, ARGX-119 (MuSK agonist), which is being evaluated in disorders of the neuromuscular junction (NMJ), including congenital myasthenic syndromes (CMS). 'The data presented at AAN underscore VYVGART and VYVGART Hytrulo's differentiated efficacy and safety profile, connecting data from our long-term studies to what matters most to gMG and CIDP patients, which is durable, significant quality of life improvements,' said Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. 'The extensive data from ADAPT-NXT reinforce the sustained efficacy in patients living with gMG and showcase the opportunity of individualized VYVGART treatment across fixed cycles or every two- or three-week dosing. Also, our long term ADHERE+ data highlight the strength of VYVGART Hytrulo to meaningfully impact motor function and muscle strength for patients with CIDP. Overall, the data we are sharing at AAN reinforce our commitment to the neuromuscular community and further solidify VYVGART as a leading biologic to redefine patient outcomes.' VYVGART Sets New Benchmark of Sustained Efficacy and Safety for Patients with gMG Sustained disease control achieved across multiple dosing approaches: ADAPT-NXT Part B data demonstrate clinically meaningful improvements as early as Week 1 with both bi-weekly and every three-week dosing schedules of VYVGART. Over the course of the study (126 weeks), 75% of patients showed sustained efficacy, achieving 2-points or more of improvement in MG activities of daily living (MG-ADL score) during more than 75% of study visits. In addition, more than half (56.5%) of participants achieved minimal symptom expression (MSE) during the study. ADAPT NXT data support multiple options to individualize treatment for patients living with gMG. (Poster P1.004) Consistent efficacy and safety results over nine treatment cycles: Interim results of ADAPT-SC+ demonstrate consistent and repeatable improvements in MG-ADL and MG Quality of Life (MG-QoL) scores in gMG patients treated with VYVGART Hytrulo. There was no observed increase in infections or injection-site reactions over nine cycles of treatment. Also, the proportion of patients able to achieve MSE was consistent across multiple cycles. (Poster P1.005) VYVGART Hytrulo Delivers Long-term Functional Improvements and Favorable Safety Profile for Patients with CIDP Significant functional improvements and rapid stabilization: ADHERE+ data demonstrate VYVGART Hytrulo delivers long-term clinical efficacy. Study results report functional improvements across aINCAT disability scores (>1-point), grip strength (>17 kPa) and I-RODS scale (>8 points) at week 36 compared to baseline at entry to standard of care withdrawal phase. In addition, the majority of ADHERE patients who relapsed during randomized treatment withdrawal stage, restabilized on VYVGART – 50% as early as week 4. Treatment-emergent adverse events (TEAEs) were consistent with label and no new events, nor increased rate or severity of TEAEs were reported with longer treatment with VYVGART Hytrulo. (Oral Presentation S16.002) Real-world insights on transitioning from IVIg to VYVGART Hytrulo: The ADHERE Phase 4 switch open-label study will build upon the ADHERE registrational trial with new data evaluating the transition of patients from a stable dose of IVIg to VYVGART Hytrulo in a one-week transition period (Poster P10.026). Currently, real-world data of 1,316 CIDP patients (as of Jan. 31, 2025) treated with VYVGART Hytrulo show that 3.3% of patients reported any general CIDP worsening. (Symposium: ITU From Discovery to Practice: FcRn Blockade and its Role in CIDP and gMG) Pipeline Targets Unmet Needs in Underserved Patient Communities First-in-human Phase 1 study supports continued investigation of ARGX-119: Across multiple and single dosing regimens, data from ARGX-119 show a favorable safety profile with no new safety signals observed, supporting further development as a treatment for patients with disorders of the NMJ. (Poster P10.007) Expansion to Seronegative and Ocular MG: argenx is pursuing label extension for VYVGART to broaden its impact with the MG community, including through two Phase 3 studies for seronegative gMG (ADAPT-SERON) and ocular MG (ADAPT-OCULUS). The ADAPT-SERON study is supported by data from seronegative patients in prior VYVGART studies showing consistent and clinically meaningful MG-ADL improvements, including patients achieving MSE. (Poster P1.029) Full study details can be found at 2025 American Academy of Neurology Abstract Website See FDA-approved Important Safety Information below, full Prescribing Information for VYVGART, and full Prescribing Information for VYVGART Hytrulo for additional information. Important Safety Information What is VYVGART® (efgartigimod alfa-fcab)?VYVGART is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive). IMPORTANT SAFETY INFORMATIONDo not use VYVGART if you have a serious allergy to efgartigimod alfa or any of the other ingredients in VYVGART. VYVGART can cause serious allergic reactions and a decrease in blood pressure leading to fainting. VYVGART may cause serious side effects, including: Infection. VYVGART may increase the risk of infection. The most common infections were urinary tract and respiratory tract infections. Signs or symptoms of an infection may include fever, chills, frequent and/or painful urination, cough, pain and blockage of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain. Allergic Reactions (hypersensitivity reactions). VYVGART can cause allergic reactions such as rashes, swelling under the skin, and shortness of breath. Serious allergic reactions, such as trouble breathing and decrease in blood pressure leading to fainting have been reported with VYVGART. Infusion-Related Reactions. VYVGART can cause infusion-related reactions. The most frequent symptoms and signs reported with VYVGART were high blood pressure, chills, shivering, and chest, abdominal, and back pain. Tell your doctor if you have signs or symptoms of an infection, allergic reaction, or infusion-related reaction. These can happen while you are receiving your VYVGART treatment or afterward. Your doctor may need to pause or stop your treatment. Contact your doctor immediately if you have signs or symptoms of a serious allergic reaction. Before taking VYVGART, tell your doctor if you: take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines, have received or are scheduled to receive a vaccine (immunization), or have any allergies or medical conditions, including if you are pregnant or planning to become pregnant, or are breastfeeding. What are the common side effects of VYVGART?The most common side effects of VYVGART are respiratory tract infection, headache, and urinary tract infection. These are not all the possible side effects of VYVGART. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088. Please see the full Prescribing Information for VYVGART and talk to your doctor. What is VYVGART® HYTRULO (efgartigimod alfa and hyaluronidase-qvfc)?VYVGART HYTRULO is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive).VYVGART HYTRULO is a prescription medicine used for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) IMPORTANT SAFETY INFORMATIONDo not use VYVGART HYTRULO if you have a serious allergy to efgartigimod alfa, hyaluronidase, or any of the other ingredients in VYVGART HYTRULO. VYVGART HYTRULO can cause serious allergic reactions and a decrease in blood pressure leading to fainting. VYVGART HYTRULO may cause serious side effects, including: Infection. VYVGART HYTRULO may increase the risk of infection. The most common infections for efgartigimod alfa-fcab-treated patients were urinary tract and respiratory tract infections. Signs or symptoms of an infection may include fever, chills, frequent and/or painful urination, cough, pain and blockage of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain. Allergic Reactions (hypersensitivity reactions). VYVGART HYTRULO can cause allergic reactions such as rashes, swelling under the skin, and shortness of breath. Hives were also observed in patients treated with VYVGART HYTRULO. Serious allergic reactions, such as trouble breathing and decrease in blood pressure leading to fainting have been reported with efgartigimod alfa-fcab. Infusion-Related Reactions. VYVGART HYTRULO can cause infusion-related reactions. The most frequent symptoms and signs reported with efgartigimod alfa-fcab were high blood pressure, chills, shivering, and chest, abdominal, and back pain. Tell your doctor if you have signs or symptoms of an infection, allergic reaction, or infusion-related reaction. These can happen while you are receiving your VYVGART HYTRULO treatment or afterward. Your doctor may need to pause or stop your treatment. Contact your doctor immediately if you have signs or symptoms of a serious allergic reaction. Before taking VYVGART HYTRULO, tell your doctor if you: take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines, have received or are scheduled to receive a vaccine (immunization), or have any allergies or medical conditions, including if you are pregnant or planning to become pregnant, or are breastfeeding. What are the common side effects of VYVGART HYTRULO?The most common side effects in efgartigimod-alfa-fcab-treated patients were respiratory tract infection, headache, and urinary tract infection. Additional common side effects with VYVGART HYTRULO are injection site reactions, including rash, redness of the skin, itching sensation, bruising, pain, and hives. These are not all the possible side effects of VYVGART HYTRULO. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088. Please see the full Prescribing Information for VYVGART HYTRULO and talk to your doctor. About VYVGART and VYVGART HytruloVYVGART® (efgartigimod alfa fcab) is a first-in-class human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG autoantibodies. VYVGART® Hytrulo is a subcutaneous combination of efgartigimod alfa (VYVGART) and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE® drug delivery technology, which facilitates subcutaneous injection delivery of biologics. VYVGART is approved for generalized myasthenia gravis (gMG) and immune thrombocytopenia (Japan only). VYVGART Hytrulo is approved for gMG and chronic inflammatory demyelinating polyneuropathy (CIDP). VYVGART Hytrulo may be marketed under different proprietary names in other regions. About ARGX-119 ARGX-119 is a humanized agonistic monoclonal antibody (mAb) that targets and activates muscle-specific kinase (MuSK) to promote maturation and stabilization of the neuromuscular junction (NMJ). MuSK is a receptor kinase that has a critical role in the structure and function of the NMJ. ARGX-119 is being developed as a potential therapy for patients with neuromuscular disease. About Generalized Myasthenia Gravis (gMG)Generalized myasthenia gravis (gMG) is a rare and chronic autoimmune disease where IgG autoantibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. Approximately 85% of people with MG progress to gMG within 24 months¹, where muscles throughout the body may be affected. Patients with confirmed AChR antibodies account for approximately 85% of the total gMG population. About Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. Although confirmation of disease pathophysiology is still emerging, there is increasing evidence that IgG antibodies play a key role in the damage to the peripheral nerves. People with CIDP experience fatigue, muscle weakness and a loss of feeling in their arms and legs that can get worse over time or may come and go. These symptoms can significantly impair a person's ability to function in their daily lives. Without treatment, one-third of people living with CIDP will need a wheelchair. 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, X/Twitter, Instagram, Facebook, and YouTube. References1 Behin et al. New Pathways and Therapeutics Targets in Autoimmune Myasthenia Gravis. J Neuromusc Dis 5. 2018. 265-277 For further information, please contact: Media: Colin McBeancmcbean@ Investors: Alexandra Roy (US) aroy@ Lynn Elton (EU) lelton@ 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,' 'continue,' 'expect,' 'may,' and 'will' and include statements argenx makes concerning the potential impact of VYVGART, VYVGART Hytrulo and ARGX-119 for patients; the data for VYVGART, VYVGART Hytrulo and ARGX-119 as well as clinical studies, including ADAPT-NXT and ADHERE+; its commitment to reach the broader MG patient community with two ongoing label-expansion in oMG and snMG; its commitment to improve the lives of people suffering from severe autoimmune diseases; its goal to continue to advance a robust neuromuscular pipeline of clinical candidates; its view that first-in-human data of ARGX-119 support pipeline-in a-product development plan; the ability for ADAPT-NEXT to reinforce the sustained efficacy in patients living with gMG and showcase the opportunity of individualized VYVGART treatment; the ability of VYVGART Hytrulo to meaningfully impact motor function and muscle strength for patients with CIDP; its commitment to the neuromuscular community; its commitment to further solidify VYVGART as a leading biologic to redefine patient outcomes; its expectations regarding the ADHERE Phase 4 switch open-label study; its aim to target unmet needs in underserved patient communities; and its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. 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 to access your portfolio
Associated Press
07-03-2025
- Health
- Associated Press
argenx Highlights FcRn Leadership with Long-term Data and Transformational Patient Outcomes at the American Academy of Neurology 2025 Annual Meeting
Largest safety data set on FcRn blocking demonstrates consistent, favorable safety profile of VYVGART and VYVGART Hytrulo gMG patients on VYVGART achieve rapid, substantial, and sustained efficacy across multiple dosing regimens, supporting individualized treatment approach ADHERE+ oral presentation builds upon evidence of VYVGART Hytrulo driving improved functional ability in CIDP Amsterdam, the Netherlands – March 7, 2025 – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced clinical trial and real-world data for VYVGART® (efgartigimod alfa-fcab) and VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) will be presented at the American Academy of Neurology (AAN) Annual Meeting, taking place in San Diego, CA from April 5-9, 2025. 'Our goal is to help people living with rare autoimmune diseases feel and function the way they did before experiencing life with a debilitating condition. This year at AAN, we are sharing more evidence demonstrating the long-term benefits of VYVGART for patients living with gMG and CIDP,' said Luc Truyen, M.D., Ph.D., Chief Medical Officer at argenx. 'Our breadth of data continues to support VYVGART as a leading biologic. It has a proven ability to achieve minimal symptom expression for gMG patients and reduce CIDP symptoms quickly while providing improved functional ability, all with a favorable safety profile. We look forward to engaging in the latest science at AAN to continue pushing the boundaries of helping patients live better.' Abstracts at AAN will highlight real-world and clinical data demonstrating VYVGART's sustained clinical improvements, including consistent functional improvement and a favorable safety profile. In addition, presentations support an individualized treatment approach and the ambition for VYVGART to reach patients earlier in the treatment paradigm. Additional dosing approaches achieve clinical improvements in gMG through 126 weeks: New data from ADAPT-NXT, investigating biweekly or every three-week dosing of VYVGART, demonstrated sustained clinical improvements, including minimal symptom expression (MSE), and consistent long-term safety through 126 weeks. Largest long-term data set of any FcRn blocker in gMG shows sustained safety and efficacy: ADAPT-SC+ analyses of VYVGART Hytrulo demonstrate consistent safety results and sustained efficacy through nine cycles of treatment. Favorable benefit-risk profile in gMG: A comparative effectiveness study of emerging immunomodulatory therapies for patients with gMG shows that Fc receptor blockers, particularly VYVGART, show a more favorable benefit-risk profile. Long-term effectiveness in CIDP: Interim results from the open-label extension ADHERE+ further build upon the largest clinical data set supporting long-term efficacy, including functional improvement and safety of VYVGART Hytrulo in CIDP. Switch from IVIg to efgartigimod in CIDP: The Phase 4 open-label trial is investigating effective and safe transition from stable IVIg doses to VYVGART Hytrulo within one week after last IVIg dose. Details for oral and poster presentations at AAN are as follows: Title Lead Author Presentation Long-term Efficacy of Efgartigimod PH20 SC in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: Interim Results From The ADHERE+ Study Jeffrey Allen Oral Presentation #002 S:16 Updates on Nerve and Muscle Disorders Monday, April 7 1:12 PM Design of a Phase 3 Randomized, Double-Blinded, Placebo-Controlled Study Evaluating the Efficacy and Safety of Subcutaneous Efgartigimod PH20 Administered by Prefilled Syringe in Adults with Ocular Myasthenia Gravis Carolina Barnett-Tapia Poster #003 Neighborhood 11 Saturday, April 5 11:45 - 12:45 PM Long-Term Safety and Efficacy of Subcutaneous Efgartigimod PH20 in Adult Participants with Generalized Myasthenia Gravis: Interim Results of the ADAPT-SC+ Study Tuan Vu Poster #005 Neighborhood 11 Saturday, April 5 11:45 - 12:45 PM Fixed Cycle and Every-Other-Week Dosing of Intravenous Efgartigimod for Generalized Myasthenia Gravis: Part B of ADAPT NXT Kelly Gwathmey Poster #004 Neighborhood 11 Saturday, April 5 11:45 - 12:45 PM Hospitalization Outcomes After Efgartigimod Initiation In Patients with Myasthenia Gravis A. Gordon Smith Poster #011 Neighborhood 11 Saturday, April 5 11:45 – 12:45 PM A Retrospective Claims Study to Investigate Safety Risks Associated with Chronic Inflammatory Demyelinating Polyneuropathy and the Mediating Effects of Immunoglobulin Treatments Jana Podhorna Poster #011 Neighborhood 2 Saturday, April 5 11:45 AM – 12:45 PM Changes In Nonsteroidal Immunosuppressive Treatment Usage Before and After Efgartigimod Initiation in Patients with Myasthenia Gravis Pushpa Narayanaswami Poster #015 Neighborhood 11 Saturday, April 5 11:45 – 12:45 PM Combined Analyses of Participants with Anti-Acetylcholine Receptor Seronegative Generalized Myasthenia Gravis Treated with Efgartigimod Across Clinical Studies Vera Bril Poster #029 Neighborhood 11 Saturday, April 5 11:45 - 12:45 PM Evaluating the Comparative Effectiveness of Emerging Immunomodulatory Therapies for Patients with Generalized Myasthenia Gravis A. Gordon Smith Poster #033 Neighborhood 11 Saturday, April 5 11:45 – 12:45 PM Study Design of Subcutaneous Efgartigimod PH20 in Juvenile Generalized Myasthenia Gravis Abigail Schwaede Poster #009 Neighborhood 6 Monday, April 7 5:00 - 6:00 PM Phase 3 Trial Investigating Impact of Intravenous Efgartigimod in Anti-Acetylcholine Receptor Antibody Negative Generalized Myasthenia Gravis James F. Howard Jr Poster #032 Neighborhood 11 Monday, April 7 5:00 - 6:00 PM First-in-Human Dose Selection and Pharmacokinetics, Safety, Tolerability, and Immunogenicity of ARGX-119, an Agonist Antibody for Human Muscle-Specific Kinase Tonke van Bragt Poster #007 Neighborhood 2 Tuesday, April 8 5:00-6:00 PM Treatment Impact of Efgartigimod PH20 SC in I-RODS Daily Activity Assessment in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: Post hoc Analysis of the Registrational ADHERE Study Richard Lewis Poster #025 Neighborhood 11 Tuesday, April 8 5:00 PM – 6:00 PM Investigating the Pharmacodynamics, Injection Speed, and Usability of Subcutaneous Efgartigimod PH20 Administration Using a Prefilled Syringe Tiffany Hargraves Poster #026 Neighborhood 11 Tuesday, April 8 11:45 - 12:45 PM Transition From Intravenous Immunoglobulin to Efgartigimod PH20 SC in Participants with Chronic Inflammatory Demyelinating Polyneuropathy: A Phase 4 Study in Progress Yessar Hussain Poster #026 Neighborhood 11 Tuesday, April 8 5:00 PM – 6:00 PM COVID-19 Vaccination Response in Participants Across Clinical Trials Investigating Efgartigimod PH20 SC Ali A. Habib Poster #029 Neighborhood 11 Tuesday, April 8 11:45 - 12:45 PM More information on the program is available at See FDA-approved Important Safety Information below, full Prescribing Information for VYVGART, and full Prescribing Information for VYVGART Hytrulo for additional information. Important Safety Information What is VYVGART® (efgartigimod alfa-fcab)? VYVGART is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive). IMPORTANT SAFETY INFORMATION Do not use VYVGART if you have a serious allergy to efgartigimod alfa or any of the other ingredients in VYVGART. VYVGART can cause serious allergic reactions and a decrease in blood pressure leading to fainting. VYVGART may cause serious side effects, including: Infection. VYVGART may increase the risk of infection. The most common infections were urinary tract and respiratory tract infections. Signs or symptoms of an infection may include fever, chills, frequent and/or painful urination, cough, pain and blockage of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain. Allergic Reactions (hypersensitivity reactions). VYVGART can cause allergic reactions such as rashes, swelling under the skin, and shortness of breath. Serious allergic reactions, such as trouble breathing and decrease in blood pressure leading to fainting have been reported with VYVGART. Infusion-Related Reactions. VYVGART can cause infusion-related reactions. The most frequent symptoms and signs reported with VYVGART were high blood pressure, chills, shivering, and chest, abdominal, and back pain. Tell your doctor if you have signs or symptoms of an infection, allergic reaction, or infusion-related reaction. These can happen while you are receiving your VYVGART treatment or afterward. Your doctor may need to pause or stop your treatment. Contact your doctor immediately if you have signs or symptoms of a serious allergic reaction. Before taking VYVGART, tell your doctor if you: take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines, have received or are scheduled to receive a vaccine (immunization), or have any allergies or medical conditions, including if you are pregnant or planning to become pregnant, or are breastfeeding. What are the common side effects of VYVGART? The most common side effects of VYVGART are respiratory tract infection, headache, and urinary tract infection. These are not all the possible side effects of VYVGART. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088. Please see the full Prescribing Information for VYVGART and talk to your doctor. What is VYVGART® HYTRULO (efgartigimod alfa and hyaluronidase-qvfc)? VYVGART HYTRULO is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive). VYVGART HYTRULO is a prescription medicine used for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) IMPORTANT SAFETY INFORMATION Do not use VYVGART HYTRULO if you have a serious allergy to efgartigimod alfa, hyaluronidase, or any of the other ingredients in VYVGART HYTRULO. VYVGART HYTRULO can cause serious allergic reactions and a decrease in blood pressure leading to fainting. VYVGART HYTRULO may cause serious side effects, including: Infection. VYVGART HYTRULO may increase the risk of infection. The most common infections for efgartigimod alfa-fcab-treated patients were urinary tract and respiratory tract infections. Signs or symptoms of an infection may include fever, chills, frequent and/or painful urination, cough, pain and blockage of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain. Allergic Reactions (hypersensitivity reactions). VYVGART HYTRULO can cause allergic reactions such as rashes, swelling under the skin, and shortness of breath. Hives were also observed in patients treated with VYVGART HYTRULO. Serious allergic reactions, such as trouble breathing and decrease in blood pressure leading to fainting have been reported with efgartigimod alfa-fcab. Infusion-Related Reactions. VYVGART HYTRULO can cause infusion-related reactions. The most frequent symptoms and signs reported with efgartigimod alfa-fcab were high blood pressure, chills, shivering, and chest, abdominal, and back pain. Tell your doctor if you have signs or symptoms of an infection, allergic reaction, or infusion-related reaction. These can happen while you are receiving your VYVGART HYTRULO treatment or afterward. Your doctor may need to pause or stop your treatment. Contact your doctor immediately if you have signs or symptoms of a serious allergic reaction. Before taking VYVGART HYTRULO, tell your doctor if you: take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines, have received or are scheduled to receive a vaccine (immunization), or have any allergies or medical conditions, including if you are pregnant or planning to become pregnant, or are breastfeeding. What are the common side effects of VYVGART HYTRULO? The most common side effects in efgartigimod-alfa-fcab-treated patients were respiratory tract infection, headache, and urinary tract infection. Additional common side effects with VYVGART HYTRULO are injection site reactions, including rash, redness of the skin, itching sensation, bruising, pain, and hives. These are not all the possible side effects of VYVGART HYTRULO. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088. Please see the full Prescribing Information for VYVGART HYTRULO and talk to your doctor. About VYVGART VYVGART is a human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG autoantibodies. It is the first approved FcRn blocker in the United States, EU, China and Canada for the treatment of adults with generalized myasthenia gravis (gMG) who are anti- acetylcholine receptor (AChR) antibody positive and in Japan for the treatment of adults with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs). About VYVGART Hytrulo VYVGART Hytrulo is a subcutaneous combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. In binding to the neonatal Fc receptor (FcRn), VYVGART Hytrulo results in the reduction of circulating IgG. It is the first-approved FcRn blocker administered by subcutaneous injection. VYVGART Hytrulo is the proprietary name in the U.S. for subcutaneous efgartigimod alfa and recombinant human hyaluronidase PH20. It may be marketed under different proprietary names following approval in other regions. About Generalized Myasthenia Gravis Generalized myasthenia gravis (gMG) is a rare and chronic autoimmune disease where IgG autoantibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. Approximately 85% of people with MG progress to gMG within 24 months1, where muscles throughout the body may be affected. Patients with confirmed AChR antibodies account for approximately 85% of the total gMG population1. About Chronic Inflammatory Demyelinating Polyneuropathy Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. Although confirmation of disease pathophysiology is still emerging, there is increasing evidence that IgG antibodies play a key role in the damage to the peripheral nerves. People with CIDP experience fatigue, muscle weakness and a loss of feeling in their arms and legs that can get worse over time or may come and go. These symptoms can significantly impair a person's ability to function in their daily lives. Without treatment, one-third of people living with CIDP will need a wheelchair. About ARGX-119 ARGX-119 is a humanized agonistic monoclonal antibody (mAb) that targets and activates muscle-specific kinase (MuSK) to promote maturation and stabilization of the neuromuscular junction (NMJ). MuSK is a receptor kinase that has a critical role in the structure and function of the NMJ. ARGX-119 is being developed as a potential therapy for patients with neuromuscular disease. About argenx argenx 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 LinkedIn, X/Twitter, Instagram, Facebook, and YouTube. References 1 Behin et al. New Pathways and Therapeutics Targets in Autoimmune Myasthenia Gravis. J Neuromusc Dis 5. 2018. 265-277 For further information, please contact: Media: Ben Petok [email protected] Investors: Alexandra Roy (US) Lynn Elton (EU) 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,' 'continue,' 'engage,' 'may,' and 'will' and include statements argenx makes concerning the potential impact of VYVGART and VYVGART Hytrulo for patients; the data for VYVGART and VYVGART Hytrulo that will be presented at the upcoming AAN Annual Meeting; its goal of pushing the boundaries of helping patients live better; the planned agenda for the AAN Annual Meeting; its data showing VYVGART and VYVGART Hytrulo as one of the leading biologics for gMG and CIDP; and its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. 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 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 law.
