Core Insights - Argenx SE presented new data on the efficacy and safety of VYVGART for generalized myasthenia gravis (gMG) at the 2025 AANEM and MGFA meetings, highlighting its potential to improve patient outcomes across various gMG populations [1][2][3] Efficacy and Safety Data - The pivotal Phase 3 ADAPT SERON trial demonstrated that AChR-Ab seronegative gMG patients treated with VYVGART achieved a statistically significant improvement in MG-ADL total score compared to placebo, with a mean change of 3.35 points at week 4 [6] - Approximately 60% of VYVGART-treated gMG patients achieved minimal symptom expression (MSE), with 88% sustaining MSE for at least 4 weeks [5][6] - Real-world data indicated that over 70% of patients treated with VYVGART significantly reduced glucocorticoid use while maintaining clinical benefits [5][15] Treatment Impact - VYVGART is positioned as a targeted, effective, and safe treatment for gMG patients, regardless of autoantibody status, with ongoing studies contributing to redefining treatment standards [4][7] - The ADAPT Jr study showed that VYVGART improved outcomes in adolescents with gMG, addressing a high unmet need in this population [8] Regulatory Plans - Argenx plans to share the ADAPT SERON trial results with the U.S. FDA to seek an expansion of the VYVGART label to include adult AChR-Ab seronegative gMG patients across all subtypes [7] Long-term Outcomes - Long-term treatment with VYVGART was associated with substantial reductions in oral glucocorticoid use, with 72.5% of patients achieving meaningful tapering and an average daily dose reduction of over 50% after 18 months [15][26]

Accessibility StatementSkip Navigation The EPIC study will evaluate treatment with IMAAVYâ"¢ versus efgartigimod in adults with gMG and will include a treatment-switch arm The Company also announced new data from the pivotal Vibrance-MG study in which pediatric patients receiving treatment with IMAAVYâ"¢ demonstrated 72 weeks of sustained reduction in immunoglobulin G (IgG), sustained disease control and no new safety concerns IMAAVYâ"¢ is the only FcRn blocker for anti-AChR and anti-MuSK antibody positive ...

Core Insights - argenx SE announced positive topline data from the pivotal ADAPT SERON study of VYVGART, demonstrating significant improvement in AChR-Ab seronegative gMG patients compared to placebo with a p-value of 0.0068 [1][7] - The company plans to submit a supplemental Biologics License Application (sBLA) to the U.S. FDA to expand VYVGART's label to include adult AChR-Ab seronegative gMG patients across all three subtypes [2][7] - VYVGART was well tolerated and safe, with no new safety concerns identified, consistent with its established safety profile [3] Study Design and Results - The Phase 3 ADAPT SERON study was a randomized, double-blind, placebo-controlled trial involving 119 participants across North America, Europe, China, and the Middle East [5] - The primary endpoint was the change in MG-ADL total score from baseline to day 29, with participants receiving four once-weekly infusions of efgartigimod or placebo [5] - The study confirmed that VYVGART has the potential to be an effective treatment for gMG patients, regardless of autoantibody status [4] Implications for Treatment - The ADAPT SERON study is the largest to date for AChR-Ab seronegative gMG, indicating a critical advancement in treatment options for patients with limited alternatives [4][8] - Approximately 20% of gMG patients are AChR-Ab seronegative, and current treatments are lacking for those with anti-LRP4 antibodies or triple seronegative status [8] - The positive results from the study highlight VYVGART's ability to provide meaningful benefits across all AChR-Ab seronegative gMG subtypes [4][8] Company Overview - argenx is a global immunology company focused on improving the lives of individuals with severe autoimmune diseases, developing novel antibody-based medicines [18] - The company has developed VYVGART, the first approved neonatal Fc receptor blocker for treating generalized myasthenia gravis [18]

Core Insights - Johnson & Johnson presented new data from the 24-week pivotal Vivacity-MG3 study, demonstrating sustained disease control through treatment with nipocalimab in antibody-positive adult patients with generalized myasthenia gravis (gMG) [1][2] - The company filed a Biologics License Application (BLA) for nipocalimab in August 2024, which has been granted U.S. FDA Priority Review for the treatment of gMG [1][2] - Real-world studies indicate a significant unmet need for effective treatments for patients living with gMG, particularly among pregnant women and those receiving steroids [1][3] Company Developments - Johnson & Johnson announced that 12 abstracts, including two oral presentations, will be presented at the 2025 American Academy of Neurology (AAN) Annual Meeting, highlighting the potential of nipocalimab in treating gMG [1][4] - The ongoing open-label extension (OLE) study of nipocalimab shows long-term efficacy and safety, indicating sustained disease control in a broad population of antibody-positive gMG adult patients [6][11] - The company emphasizes its commitment to addressing critical unmet needs in the autoantibody disease space through innovative research and development [4][14] Industry Context - Myasthenia gravis (MG) is an autoantibody disease affecting approximately 700,000 people worldwide, with a significant portion being women of child-bearing potential [10][11] - The Phase 3 Vivacity-MG3 study was designed to measure sustained efficacy and safety in gMG patients with insufficient response to standard of care (SOC) therapy [11][12] - There is a growing recognition of the need for additional approved immunoselective therapies that are effective and have demonstrated safety profiles for individuals living with gMG [3][6]

Core Insights - Amgen announced new data from the Phase 3 MINT trial showing the efficacy and safety of UPLIZNA in adults with generalized myasthenia gravis (gMG), indicating durable symptom relief with a simplified treatment regimen of two doses per year after an initial loading dose [1][3] Group 1: Trial Results - The MINT trial demonstrated significant improvement in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score for UPLIZNA compared to placebo, with an adjusted difference of -2.8 (95% CI, -3.9 to -1.7) in the AChR+ subpopulation at week 52 [2] - Among AChR+ patients treated with UPLIZNA, 72.3% experienced a ≥3 point improvement in the MG-ADL score, compared to 45.2% in the placebo group [2] - The trial also showed a greater change from baseline in the Quantitative Myasthenia Gravis (QMG) score for UPLIZNA patients at week 52, with an adjusted difference of -4.3 (95% CI, -5.9 to -2.8) [3] Group 2: Treatment Protocol - The MINT trial was notable for incorporating a corticosteroid tapering protocol, where patients on corticosteroids were reduced to prednisone 5 mg per day by week 24 [3][10] - UPLIZNA is administered as an initial loading dose followed by maintenance doses every six months, which simplifies the treatment regimen for patients [12] Group 3: Regulatory Status - UPLIZNA is currently approved for treating adult patients with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD) and is under priority FDA review for Immunoglobulin G4-related disease (IgG4-RD) with a PDUFA date of April 3, 2025 [5] - The FDA has granted UPLIZNA Orphan Drug Designation for the treatment of gMG, with regulatory filing activities expected to be completed in the first half of 2025 [5] Group 4: Safety Profile - No new safety signals were identified during the MINT trial, and the overall treatment-emergent adverse event (TEAE) profile was consistent with the known safety profile for UPLIZNA [4] - The most common adverse events reported included infusion-related reactions, nasopharyngitis, and urinary tract infections [4][15]