A Phase 4, Open-Label, Single-Group, Multicenter Study in Adult Participants With Chronic Inflammatory Demyelinating Polyneuropathy Who Transition From Treatment With Intravenous Immunoglobulin to Efgartigimod PH20 SC

This study will measure how adults with CIDP receiving IVIg treatment adjust to efgartigimod PH20 SC. The study duration for each participant will be approximately 17 to 19 weeks.

开始日期2024-12-10

申办/合作机构

argenx SE

CTR20243345

/ Active, not recruiting临床2/3期

一项在18岁及以上活动性特发性炎性肌病受试者中评价efgartigimod PH20 SC有效性、安全性、耐受性、药效学、药代动力学和免疫原性的II/III期、随机、双盲、安慰剂对照、平行组、双臂、多中心、操作无缝研究

在IIM受试者中评价efgartigimod PH20 SC与安慰剂相比的治疗效果

开始日期2024-11-21

申办/合作机构

再鼎医药（上海）有限公司

[+2]

100 项与 Efgartigimod/Hyaluronidase 相关的临床结果

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100 项与 Efgartigimod/Hyaluronidase 相关的转化医学

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100 项与 Efgartigimod/Hyaluronidase 相关的专利（医药）

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项与 Efgartigimod/Hyaluronidase 相关的文献（医药）

2024-12-01·DRUGS IN R&D

Pharmacokinetics, Pharmacodynamics, and Safety of Intravenous Efgartigimod and Subcutaneous Efgartigimod PH20 in Healthy Chinese Participants

Article

作者: Reinhart, Harald ; Chen, Jiajia ; Van Bragt, Tonke ; Gu, Xiaowen ; Noukens, Jan ; Wang, Lina ; Jing, Shan ; Pu, Xia ; Liu, Jing ; Zhang, Yu ; Lin, Yang ; Guglietta, Antonio

BACKGROUND:

Efgartigimod, a human immunoglobulin G (IgG)1-derived Fc fragment targeting the neonatal Fc receptor, has been developed into intravenous (IV) and subcutaneous (SC) formulations for treating generalized myasthenia gravis (gMG) and other autoimmune diseases. Data in the Chinese population were not available to date, and while both formulations have been approved in the USA, the EU, Japan and China for the treatment of gMG.

OBJECTIVE:

We present the pharmacokinetic, pharmacodynamic, and safety of IV and SC PH20 efgartigimod in healthy Chinese participants.

METHODS:

In two independent, double-blinded, placebo-controlled, phase I studies of the IV and SC formulations of efgartigimod, healthy Chinese adults were randomized 3:1 to receive active treatment or matching placebo once every 7 days for four doses. Primary endpoints were pharmacokinetic parameters.

RESULTS:

After the fourth IV infusion, a mean maximum observed concentration (C_max) of 194 µg/mL was reached at the end of the 1 h infusion; the mean area under concentration-time curve from time zero to 168 h (AUC_0-168h) was 5300 µg × h/mL. After the fourth SC injection, a mean C_max of 42.1 µg/mL was achieved with a median T_max of 47.74 h; the mean AUC_0-168h was 4790 µg × h/mL. Maximal mean reductions from baseline in total IgG levels were reached approximately 24 days after the first dose (60.7%, IV formulation; 66.4%, SC formulation). Treatment-related adverse events (TRAEs) were reported in seven (58.3%) participants receiving SC efgartigimod, mostly injection-site reactions. No TRAEs or AEs of special interest were reported in the IV study.

CONCLUSIONS:

The efgartigimod IV and SC pharmacokinetic, pharmacodynamic, and safety profiles in Chinese participants were similar to the known profiles in non-Chinese participants. Both formulations effectively reduced total IgG levels by a similar percentage.

CLINICAL TRIAL REGISTRATION:

CTR20211952 and CTR20211805.

2024-12-01·ADVANCES IN THERAPY

Risk–Benefit Analysis of Novel Treatments for Patients with Generalized Myasthenia Gravis

Article

作者: Qi, Cynthia Z. ; Brauer, Edward ; Smith, A. Gordon ; Yang, Hongbo ; Wolfe, Gil I ; Gelinas, Deborah ; Saccà, Francesco ; Smith, A Gordon ; Chen, Xin ; Phillips, Glenn ; Qi, Cynthia Z ; Habib, Ali A ; Wolfe, Gil I. ; Habib, Ali A. ; Du, Mandy

INTRODUCTION:

This study used network meta-analysis (NMA) to inform and compare the number needed to treat (NNT), number needed to harm (NNH), and cost per improved outcome (CPIO) associated with more recently approved treatments for anti-acetylcholine receptor antibody-positive (anti-AChR Ab+) generalized myasthenia gravis (gMG).

METHODS:

Clinical trials of neonatal Fc receptor (FcRn) inhibitors, efgartigimod intravenous (IV) and rozanolixizumab, and complement inhibitors, ravulizumab and zilucoplan, versus placebo (with background conventional treatment) were included in the primary NMA to compare efficacy and safety outcomes. The outputs from the NMAs were used to estimate NNT and NNH of each treatment versus placebo. CPIO (2024 USD) was estimated for a ≥ 3- or ≥ 5-point reduction from baseline in Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores. Sensitivity analyses were performed adding efgartigimod PH20 subcutaneous (SC) and eculizumab to the NMA.

RESULTS:

Efgartigimod IV had the lowest NNT versus placebo for achieving a ≥ 3- and ≥ 5-point reduction in QMG, as well as a ≥ 5-point reduction in MG-ADL, whereas rozanolixizumab had the lowest NNT for a ≥ 3-point reduction in MG-ADL. The NNH versus placebo was similar across comparator treatments. Efgartigimod IV had the lowest CPIO among all treatments for all assessed efficacy outcomes. Sensitivity analyses yielded results consistent with primary analysis and indicated that efgartigimod PH20 SC had comparable NNT and CPIO values to efgartigimod IV, whereas eculizumab had comparable NNT and higher CPIO values compared to other complement inhibitors.

CONCLUSIONS:

FcRn inhibitors and complement inhibitors assessed in this study all demonstrated clinical benefit in terms of NNT as well as an acceptable safety profile in terms of NNH. Within the limitations of this meta-analysis, efgartigimod was associated with a favorable benefit-risk profile as well as a better economic value compared to ravulizumab, rozanolixizumab, and zilucoplan as treatments for anti-AChR Ab+ gMG.

2024-11-01·DRUGS

Efgartigimod: A Review in Generalised Myasthenia Gravis

Review

作者: Blair, Hannah A. ; Blair, Hannah A

Efgartigimod (Vyvgart^®; Vyvgart^® Hytrulo) is a neonatal fragment crystallizable receptor (FcRn) antagonist indicated for the treatment of generalised myasthenia gravis (gMG) in adults who are acetylcholine receptor (AChR) antibody positive (Ab+). Efgartigimod is approved for both intravenous (IV) and subcutaneous (SC) use. In a pivotal phase III trial, IV efgartigimod was associated with significant and clinically meaningful improvements in myasthenia gravis symptoms and reductions in disease burden. The beneficial effects of IV efgartigimod were reproducible, durable and maintained over the long term. IV efgartigimod also improved health-related quality of life (HRQOL). In another phase III trial, SC efgartigimod PH20 was noninferior to IV efgartigimod in reducing total immunoglobulin G levels. Clinical improvement with SC efgartigimod PH20 was consistent with that of IV efgartigimod and was reproducible over the long term. Efgartigimod was generally well tolerated; the most common adverse events were headache and infections (with IV efgartigimod) and injection-site reactions (with SC efgartigimod PH20). Although further long-term data are required, IV and SC formulations of efgartigimod provide effective, generally well-tolerated and flexible treatment options for adults with AChR Ab+ gMG.

187

项与 Efgartigimod/Hyaluronidase 相关的新闻（医药）

2025-10-29

·GlobeNewswire-Health Care

argenx Presents New Data at AANEM and MGFA Highlighting the Strength and Broad Benefit of VYVGART for Myasthenia Gravis Patients

VYVGART demonstrated clinically meaningful benefit across all AChR-Ab seronegative gMG subtypes in ADAPT SERON, supporting broad role of pathogenic IgGs in disease activityFinal ADAPT SC+ results show ~60% of VYVGART gMG patients achieved minimal symptom expression (MSE), with 88% sustaining MSE for at least 4 weeks Real world data show >70% of patients treated with VYVGART meaningfully reduced glucocorticoid use while maintaining clinical benefit October 29, 2025, 8:00 AM PT 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 new data further highlighting the efficacy and safety of VYVGART® (IV: efgartigimod alfa-fcab and SC or Hytrulo: efgartigimod alfa and hyaluronidase-qvfc) across generalized myasthenia gravis (gMG) patient populations at the 2025 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting (AANEM) and Myasthenia Gravis Foundation of America (MGFA) Scientific Session in San Francisco from October 29 – November 1, 2025. The data presented feature pivotal Phase 3 results from the ADAPT SERON trial in acetylcholine receptor antibody (AChR-Ab) seronegative patients, interim findings from the ADAPT Jr study in adolescents, a real-world claims analysis showing significantly reduced steroid use among patients treated with VYVGART, long-term outcomes from VYVGART SC treatment with the majority of patients achieving minimal symptom expression (MSE), and a range of other important results. Collectively, these data provide strong evidence of VYVGART’s potential to improve outcomes across the full spectrum of gMG patient populations. “Our latest data reflect argenx’ ambition to deliver transformational impact for all people living with MG globally with rapid and significant functional improvements sustained over time, regardless of MG disease subtype,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. “Each new VYVGART study in MG is contributing to redefining treatment standards for patients seeking to regain quality of life and return to normal daily activities – a significant proportion of whom achieve this standard. And we’re not done yet.” First dedicated study to date of AChR-Ab seronegative gMG confirms VYVGART’s potential to be a targeted, effective, and safe treatment for patients living with gMG, regardless of autoantibody status (MGFA Oral Presentation #101) The Phase 3 ADAPT SERON study met its primary endpoint (p-value=0.0068), demonstrating that AChR-Ab seronegative gMG patients treated with VYVGART achieved a statistically significant improvement in MG-ADL (Myasthenia Gravis Activities of Daily Living) total score compared to placebo after four weeks.In the overall population, mean change from baseline in patients treated with VYVGART was a clinically meaningful 3.35 point improvement in MG-ADL total score at week 4. These positive results in MG-ADL score mean patients experienced significant improvements in one or a combination of their abilities in breathing, eating, eyesight and motor functions.Improvements in MG-ADL and QMG (Quantitative Myasthenia Gravis score) among patients treated with VYVGART were increasingly pronounced across subsequent treatment cycles in the overall population and in all patient subgroups – MuSK+, LRP4+, triple seronegative gMG. VYVGART was well tolerated across AChR-Ab seronegative subtypes and consistent with the established safety profile in patients with AChR-Ab seropositive gMG and other indications. No new safety concerns were identified.Results from the ongoing ADAPT SERON study indicate that pathogenic IgGs are an underlying driver of gMG across patient subtypes, regardless of autoantibody status. argenx plans to share these results with the U.S. FDA and seek expansion of the VYVGART label to include adult AChR-Ab seronegative gMG patients across all three subtypes. “The ADAPT SERON trial showed that efgartigimod generated tangible improvements in daily functioning, marking an important advancement for the field and for seronegative patients seeking better disease control. Patients with seronegative gMG, in particular, have historically lacked effective treatment options. Collectively, these findings highlight that efgartigimod has the potential to deliver meaningful and progressive benefits for patients regardless of antibody status, with its therapeutic impact strengthening through continued treatment,” said James F. Howard Jr., M.D., Professor of Neurology (Neuromuscular Disease), Medicine and Allied Health, Department of Neurology, The University of North Carolina at Chapel Hill School of Medicine and Principal Investigator for the ADAPT SERON trial. Addressing Unmet Need in Adolescent gMG Patients (MGFA Oral Presentation #113) Interim results from the Phase 2/3 ADAPT Jr study showed that VYVGART improved outcomes in adolescents (ages 12–17) with gMG, offering the potential to address a patient population with high unmet needs. Reduction in total IgG and AChR-Ab levels were similar to outcomes in adult gMG patients. VYVGART was well-tolerated and had a favorable safety profile. Reducing Steroid Use in Real-World Practice (MGFA Oral Presentation #100) A retrospective cohort study using real-world evidence showed that long-term VYVGART treatment was associated with substantial and progressive reductions in oral glucocorticoid (GC) use among adults with gMG. A majority of patients (72.5%) achieved meaningful steroid tapering – defined as at least a 1 mg per day reduction from baseline.At 18 months, the average daily GC dose for patients receiving VYVGART decreased by more than 50% (from 16.6 to 7.5 mg/day), with most patients (55%) receiving an average daily GC dose of 5 mg/day or less, and 30% of patients receiving no GC. Improvements in MG-ADL scores of ~5 points were observed among patients with available data, supporting the clinical benefit of steroid tapering alongside maintained disease control. Demonstrating Sustained MSE and Favorable Safety Profile in Long-Term Treatment (AANEM Poster Presentation #12) Final results from the ADAPT-SC+ open-label extension study demonstrated that VYVGART SC was well-tolerated during 459.4 participant-years of follow-up and up to 33 treatment cycles.Clinically meaningful improvements in mean MG-ADL total scores were observed in AChR-Ab+ participants as early as Week 1 and were sustained through Week 163. 59.2% of AChR-Ab+ participants achieved MSE (MG-ADL score of 0 or 1) at least once, while 88.1% of those sustained minimal symptom expression for at least 4 weeks. No new safety signals were identified with long-term use. More information on the data presented at the 2025 AANEM Annual Meeting can be found here. 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. 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 to facilitate 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. ADAPT SERON Study Design The Phase 3 ADAPT SERON study is a randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of efgartigimod in adults with AChR-Ab seronegative gMG (n=119) across North America, Europe, China, and the Middle East. Part A randomized participants (1:1) received 4 once-weekly infusions of efgartigimod IV or placebo, followed by a 5-week follow-up and primary analysis. Part B is an open-label extension: participants receive 2 fixed cycles of 4 once-weekly efgartigimod infusions (4-week interval between cycles); from cycle 3 onward, additional cycles could be started ≥1 week after the last administration of the previous cycle, based on clinical status. The primary endpoint is the MG-ADL total score change from baseline to day 29 in part A. Other scales of evaluation include QMG, MG-QoL 15r, MGC, and EQ-5D-5L VAS. Enrolled participants had a confirmed MG diagnosis by an independent panel of experts, and an MG-ADL total score of 5 or greater. Participants were on a stable dose of at least one gMG treatment prior to randomization, including acetylcholinesterase inhibitors, corticosteroids or nonsteroidal immunosuppressive drugs. Participants were eligible to enroll in ADAPT SERON if they were AChR-Ab seronegative, which included participants who are MuSK-Ab seropositive, LRP4-Ab seropositive, or triple seronegative. MG-ADL is a validated measure of disease activity in patients living with myasthenia gravis, which evaluates the functional impact of symptoms on daily activities such as speaking, chewing, swallowing, breathing, and limb strength. ADAPT Jr Study DesignADAPT Jr is an ongoing, open-label, multi-center clinical trial evaluating VYVGART in juvenile patients (ages 2 to <18 years) with anti-acetylcholine receptor (AChR) antibody positive generalized myasthenia gravis (gMG). The trial includes sites across the United States, Canada, and Europe. Key assessments include pharmacokinetics, immunogenicity, safety, tolerability, and clinical effect measured by MG-ADL, QMG, EQ-5D-Y, and pediatric fatigue scores. The primary objective is to confirm age-appropriate dosing; secondary endpoints include evaluating efgartigimod’s safety and activity in children and adolescents living with gMG. IQVIA Real-World Steroid Tapering Study DesignThe IQVIA steroid tapering study is a retrospective, real-world analysis evaluating changes in oral glucocorticoid (GC) use among adults with generalized myasthenia gravis (gMG) who initiated and continued VYVGART for at least 18 months. Patients were identified using the U.S. IQVIA Longitudinal Access and Adjudication Data (LAAD) medical and pharmacy claims database between April 2016 and January 2025. MG-ADL (Myasthenia Gravis Activities of Daily Living) data were integrated from the My VYVGART Path patient support program to provide additional clinical context. Glucocorticoid average daily dose (ADD) was evaluated at multiple time points, including baseline (90 days prior to therapy) and 18 months after starting VYVGART. The study included 168 adults (mean age: 59.7 years; 44% female) with chronic GC use prior to VYVGART initiation. Glucocorticoid average daily dose (ADD) was assessed at baseline (90 days pre-VYVGART) and at 18 months (days 515–545 post-initiation). This study provides real-world insights into steroid tapering practices and treatment outcomes among VYVGART-treated gMG patients in the United States. ADAPT-SC+ Study DesignADAPT-SC+ is a Phase 3, multicenter, open-label extension study evaluating the long-term safety, tolerability, and efficacy of VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in adult patients with generalized myasthenia gravis (gMG) who previously completed the ADAPT-SC or ADAPT+ studies. Participants received individualized treatment cycles of subcutaneous efgartigimod PH20, with flexible intervals based on clinical need, for up to 33 cycles and over 459.4 participant-years of follow-up. The study’s primary objectives were to assess the long-term safety and sustained clinical effectiveness of VYVGART Hytrulo in a real-world, antibody-positive gMG population. Efficacy was primarily evaluated using the Myasthenia Gravis Activities of Daily Living (MG-ADL) score, a validated measure of disease impact on everyday function. 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 AChR-Ab seronegative gMGgMG is a rare, chronic, neuromuscular autoimmune disease caused by pathogenic IgGs targeting the neuromuscular junction (NMJ), resulting in impaired neuromuscular transmission and debilitating and potentially life-threatening muscle weakness and chronic fatigue. Approximately 80% of patients with gMG have detectable antibodies against the AChR in sera, and these patients are diagnosed as AChR-Ab seropositive gMG. Approximately 20% of patients with gMG do not have detectable serum antibodies directed against AChR and are referred to as AChR-Ab seronegative gMG. These patients may have detectable autoantibodies targeting other NMJ proteins, such as muscle-specific tyrosine kinase (MuSK) and low-density lipoprotein receptor-related protein 4 (LRP4), or others. Anti-MuSK antibodies are detected in approximately 1-10% of patients with gMG, while anti-LRP4 antibodies are detected in approximately 1-5% of patients with gMG. About 10% of patients do not have any detectable autoantibodies against AChR, MuSK or LRP4. These triple seronegative patients have historically been excluded from studies and have a higher disease burden and unmet medical need compared to patients with detectable autoantibodies. Currently, there are no approved treatments available for patients with anti-LRP4 antibodies or for triple seronegative patients. 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 www.argenx.com and follow us on LinkedIn, Instagram, Facebook, and YouTube. Media: Colin McBeancmcbean@argenx.com Investors: Alexandra Roy aroy@argenx.com FORWARD LOOKING STATEMENTSThe 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 “advance,” “aim,” “ambition,” “can,” “committed,” “continue,” “deliver,” “goal,” “improve,” “may,” “potential,” and “will” and include statements argenx makes concerning VYVGART’s potential to improve outcomes across the full spectrum of gMG patient populations; its ambition to deliver transformational impact for all people living with MG globally with rapid and significant functional improvements sustained over time, regardless of specific MG disease subtype; VYVGART’s potential to be a targeted, effective, and safe treatment for patients living with gMG; efgartigimod’s potential to deliver meaningful and progressive benefits for patients regardless of antibody status, with its therapeutic impact strengthening through continued treatment; its plans to share its Phase 3 results from the ADAPT SERON trial results with the U.S. FDA and seek expansion of the VYVGART label to include adult AChR-Ab seronegative gMG patients across all three subtypes; its commitment to improve the lives of people suffering from severe autoimmune diseases; its aim to target unmet need in adolescent gMG patients; its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines; its commercialization of the first approved neonatal Fc receptor (FcRn) blocker and evaluation of its broad potential in multiple serious autoimmune diseases; and its advancement of several earlier stage experimental medicines within its therapeutic franchises. 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 law.

临床结果临床3期上市批准

2025-10-16

·再鼎医药

🤟2025 AANEM | 艾加莫德斩获3项口头报告，40余项研究成果将亮相

关键性ADAPT SERON研究结果和ADAPT Jr中期数据展现艾加莫德治疗多种类型重症肌无力患者的潜力真实世界证据与长期数据印证艾加莫德对患者结局的持续改善 10月15日，再鼎医药合作伙伴argenx宣布，艾加莫德（包括静脉输注和皮下注射剂型）的多项数据入选美国神经肌肉和电生理诊断医学协会年会（AANEM）及美国重症肌无力基金会（MGFA）科学会议。会议将于当地时间10月29日至11月1日在美国旧金山举行。艾加莫德有三项研究入选口头报告，40项研究入选壁报环节。口头报告详情如下：标题：艾加莫德静脉输注治疗抗乙酰胆碱受体（AChR）抗体阴性全身型重症肌无力的III期研究演讲人： James F. Howard 环节和时间： MGFA口头报告# 101 环节B：治疗和临床研究 10月29日10:40（太平洋时间）标题： ADAPT Jr研究结果：艾加莫德静脉输注治疗青少年全身型重症肌无力的有效性评估演讲人： Abigail N. Schwaede 环节和时间： MGFA口头报告# 113 环节B：治疗和临床研究 10月29日10:20（太平洋时间）标题：艾加莫德用药18个月后口服糖皮质激素用量减少：基于美国医疗理赔数据库的分析演讲人： Neelam Goyal 环节和时间： MGFA口头报告# 100 环节C：患者护理，热点聚焦，回顾性/事后分析研究 10月29日13:16（太平洋时间）其中，评估艾加莫德治疗AChR抗体阴性全身型重症肌无力成人患者的3期ADAPT SERON研究结果显示，所有三种亚型（三重血清阴性、MuSK抗体阳性及LRP4抗体阳性）患者的疾病活动度均表现出具有临床意义的改善。 ADAPT Jr中期结果评估艾加莫德在青少年全身型重症肌无力中的适龄给药方案、安全性及临床效果。关于艾加莫德卫伟迦®（艾加莫德静脉输注）是一款人IgG1抗体的Fc片段，可与新生儿Fc受体（FcRn）结合，旨在减少致病性免疫球蛋白G（IgG）抗体并阻断IgG循环。艾加莫德静脉输注是首个获批的FcRn拮抗剂，用于治疗乙酰胆碱受体抗体阳性的成人全身型重症肌无力患者。卫力迦®（艾加莫德皮下注射）是一款皮下注射剂型的生物制剂，艾加莫德皮下注射与重组人透明质酸酶PH20（rHuPH20）共同配制，使用Halozyme的ENHANZE®药物递送技术，从而促进生物制剂的皮下注射给药。艾加莫德皮下注射将在30-90秒内以单次皮下注射（固定剂量1,000mg）的方式给药，连续4周每周注射一次为一治疗周期。艾加莫德皮下注射已在美国（商品名为VYVGART® Hytrulo）、欧盟（商品名为VYVGART® SC）和日本（商品名为VYVDURA®）获批上市。艾加莫德皮下注射已获得中国国家药监局批准，用于治疗乙酰胆碱受体抗体阳性的成人全身型重症肌无力患者和成人慢性炎性脱髓鞘性多发性神经根神经病患者。艾加莫德有望治疗多种由致病性IgG抗体介导的严重的自身免疫性疾病，且正在多种自身免疫性疾病适应证中进行探索评估。再鼎医药与argenx达成独家授权合作，在大中华区（中国内地，香港、澳门和台湾地区）开发和商业化艾加莫德。关于再鼎医药再鼎医药（纳斯达克股票代码：ZLAB；香港联交所股份代号：9688）是一家以研发为基础、处于商业化阶段的创新型生物制药公司，总部位于中国和美国。我们致力于通过创新产品的发现、开发和商业化解决肿瘤、免疫、神经科学和感染性疾病领域未被满足的巨大医疗需求。我们的目标是利用我们的能力和资源努力促进中国及全世界人类的健康福祉。有关再鼎医药的更多信息，请访问www.zailaboratory.com或关注公司官微：再鼎医药。

临床3期临床结果上市批准临床成功

2025-10-15

·GlobeNewswire-Health Care

argenx to Highlight Key Data and Breadth of Immunology Innovation at 2025 AANEM Annual Meeting and MGFA Scientific Session

Pivotal ADAPT SERON results and interim ADAPT Jr data showcase VYVGART’s potential to treat broad set of myasthenia gravis patientsReal-world evidence and long-term data reinforce VYVGART’s sustained impact on patient outcomesMore than 40 abstracts across MG, CIDP, MMN, and IIM highlight depth of clinical evidence and ongoing commitment to rare neuromuscular disease communities October 15, 2025, 7:00 AM CEST Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, will present data for VYVGART® (IV: efgartigimod alfa-fcab and SC or Hytrulo: efgartigimod alfa and hyaluronidase-qvfc) and pipeline candidate empasiprubart at the 2025 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and Myasthenia Gravis Foundation of America (MGFA) Scientific Session in San Francisco from October 29-November 1, 2025. “We’re proud to have a robust presence at this year’s AANEM Annual Meeting and MGFA Scientific Session, where we are sharing pivotal data and meaningful evidence across a broad spectrum of serious neuromuscular diseases, including MG, CIDP, MMN and IIM,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. “The breadth of studies reflect the strength and momentum of our clinical development programs, from the continued expansion of VYVGART into new patient populations, to our fast-approaching growth opportunity with empasiprubart. These presentations also underscore our commitment to generating evidence that propels innovation and positively impacts people living with these debilitating diseases.” Abstracts at AANEM and MGFA will highlight real-world and clinical data demonstrating the potential of argenx’s innovative therapies to help more people living with autoimmune and neuromuscular diseases. Advancing VYVGART in Treating Additional gMG Patient Populations Results from the Phase 3 ADAPT SERON study evaluating VYVGART for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody negative (AChR-Ab seronegative) show clinically meaningful improvements in disease activity across all three subtypes (triple negative, MuSK, and LRP4-Ab seropositive).ADAPT Jr interim results assess age-appropriate dosing, safety, and clinical effect of VYVGART in juvenile gMG. Driving CIDP Innovation with VYVGART Hytrulo and Empasiprubart VYVGART Hytrulo CIDP data highlight functional improvement, long-term safety, and real-world characteristics of patients, underscoring the need for early treatment with effective and well-tolerated therapies.Two Phase 3 study designs for empasiprubart in CIDP – EMVIGORATE and EMNERGIZE – demonstrate argenx’s commitment to advancing innovative therapies and transforming outcomes for patients. Progressing Development of Empasiprubart in MMN Phase 2 ARDA study highlights clinical efficacy and safety data of empasiprubart in treated patients with multifocal motor neuropathy (MMN). Phase 3 study design – EMPASSION – will evaluate the efficacy and safety of empasiprubart versus IVIg in adult patients with MMN.Real-world data characterize MMN disease burden, management, and quality of life, reinforcing the need for therapeutic approaches that reduce the use of burdensome treatments and improve functional outcomes. Details for oral and poster presentations at AANEM and MGFA are as follows: Oral Presentations at MGFATitlePresentationGeneralized Myasthenia Gravis (gMG)Phase 3 Trial Investigating Impact of Intravenous Efgartigimod in Anti-Acetylcholine Receptor Antibody Negative Generalized Myasthenia GravisPresenter: James F. HowardMGFAOral Presentation #101Session B: Therapeutics and Clinical TrialsWednesday, October 29 10:40 a.m. PTResults From the ADAPT Jr Study Investigating Intravenous Efgartigimod in Juvenile Generalized Myasthenia GravisPresenter: Abigail N. SchwaedeMGFAOral Presentation#113Session B: Therapeutics and Clinical TrialsWednesday, October 29 10:20 a.m. PTReduction in Oral Glucocorticoid Use at 18 Months Following Efgartigimod Initiation Based on a United States Claims DatabasePresenter: Neelam GoyalMGFAOral Presentation#100Session C: Patient Care, Hot Topics, Retrospective/Post-hoc StudiesWednesday, October 291:16 p.m. PT Poster Presentations* at AANEM and MGFATitlePresentationGeneralized Myasthenia Gravis (gMG)Sustained Clinical Efficacy and Long-term Safety of Intravenous Efgartigimod for Generalized Myasthenia Gravis: Part B of Adapt NXTAANEMPoster #260Poster Sessions I & IIClinical Development Program for Efgartigimod in Juvenile Generalized Myasthenia GravisAANEMPoster #257Poster Sessions I & IICost and Use of Medical Devices in Incident Early-Onset Myasthenia Gravis Patients in FranceAANEMPoster #161Poster Sessions I & IIIDisability Progression and Associated Costs in Incident Early-onset Myasthenia Gravis Patients in France: A Longitudinal Cohort StudyAANEMPoster #162Poster Sessions I & IIILong-term Safety and Efficacy of Subcutaneous Efgartigimod PH20 in Adult Participants with Generalized Myasthenia Gravis: Final Results of the ADAPT-SC+ StudyAANEMPoster #12Poster Sessions I & IIIHigh Cardiovascular Disease Burden Among Patients with Myasthenia Gravis in USMGFAPoster #38Evaluation of Cardiovascular Comorbidity Burden in Patients with Generalized Myasthenia Gravis Treated with EfgartigimodMGFAPoster #68Impact of Long-Term Intravenous Efgartigimod on Quality of Life, Disease Severity, and Safety in Participants with Generalized Myasthenia Gravis during ADAPT NXTMGFAPoster #76 Design of a Phase 3 Randomized, Double Blinded, Placebo-Controlled Study Evaluating Subcutaneous Efgartigimod PH20 Administered by Prefilled Syringe in Adults with Ocular Myasthenia GravisMGFAPoster #14 Myasthenia Gravis Events in a Retrospective United States Claims Database StudyAANEMPoster #258Poster Sessions I & IIDesign of a Phase 4, Open-Label, Single-Group Study to Evaluate Clinical Outcomes of Efgartigimod PH20 Sc in Adult Participants with New-Onset Generalized Myasthenia GravisAANEMPoster #232Poster Sessions I & IIComparative Benefits of Immunomodulatory Therapies for Generalized Myasthenia GravisMGFAPoster #72Patient Characteristics, Dosing Patterns, and Outcomes Associated with Intravenous and Subcutaneous Efgartigimod Among Patients with Generalized Myasthenia Gravis in Clinical PracticeMGFAPoster #70Drivers of Mortality in Patients with Myasthenia Gravis in the United States National Veterans Affairs Health Care Network and Medicare DatabasesMGFAPoster #71Diagnosis Journey, Treatment, and Management of Patients with Ocular Myasthenia Gravis: Insights from a U.S. Patient PanelMGFAPoster #63Disease Burden, Impact on Daily Functioning, and Psychological Well-being in Patients with Ocular Myasthenia Gravis: Insights from a U.S. Patient PanelMGFAPoster #62Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Empasiprubart Versus Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: EMVIGORATE Phase 3 Study DesignAANEMPoster #249 Poster Sessions I & IIIEmpasiprubart Versus Placebo in Chronic Inflammatory Demyelinating Polyneuropathy: EMNERGIZE Phase 3 Study DesignAANEMPoster #251 Poster Sessions I & IIIImpact of Subcutaneous Efgartigimod PH20 on Autoimmune Biomarkers in the ADHERE Trial: Exploratory AnalysisAANEMPoster #325 Poster Sessions I & IIITreatment Impact of Efgartigimod PH20 SC on I-RODS Daily Activity Assessment in Patients With Chronic Inflammatory Demyelinating Polyneuropathy: Post Hoc Analysis of the Registrational ADHERE StudyAANEMPoster #246 Poster Sessions I & IIAutoantibody Signatures in Chronic Inflammatory Demyelinating Polyneuropathy: Insights on Glycolipid Reactivity From the ADHERE TrialAANEMPoster #248Poster Sessions I & IITransition From Intravenous Immunoglobulin to Efgartigimod PH20 SC in Participants with Chronic Inflammatory Demyelinating Polyneuropathy: A Phase 4 Study in ProgressAANEMPoster #250Poster Sessions I & IIADHERE+ Trial Interim Analysis: Long-Term Safety and Efficacy of Efgartigimod in Chronic Inflammatory Demyelinating PolyneuropathyAANEMPoster #217 Poster Sessions I & IIICharacteristics of Patients with Chronic Inflammatory Demyelinating Polyneuropathy Initiating Subcutaneous Efgartigimod in the United StatesAANEMPoster #141Poster Sessions I & IIIGlucocorticoid Exposure and the Risk of Serious Infections in CIDPAANEMPoster #160Poster Sessions I & IIPre-existing Conditions Among Adult Patients with Chronic Inflammatory Demyelinating Polyneuropathy in the United StatesAANEMPoster #150Poster Sessions I & IIThe Diagnostic Experience of Patients with Chronic Inflammatory Demyelinating Polyneuropathy: Results of a Real-World Survey in the United StatesAANEMPoster #255Poster Sessions I & IIIPhysician and Patient Satisfaction with Chronic Inflammatory Demyelinating Polyneuropathy Treatment: Results of a Real-World Survey in the United StatesAANEMPoster #139Poster Sessions I & IIIReal-World Characteristics of Patients Initiating Efgartigimod Subcutaneous in the United States: Insights From a Patient Support ProgramAANEMPoster #140Poster Sessions I & IIMultifocal Motor Neuropathy (MMN)Safety and Efficacy Data From the Phase 2 ARDA Study of Empasiprubart in Multifocal Motor NeuropathyAANEMPoster #215 Poster Sessions I & IIEmpasiprubart in Multifocal Motor Neuropathy: Exploratory Analyses of the Phase 2 ARDA StudyAANEMPoster #239 Poster Sessions I & IIIBurden of Disease in Multifocal Motor Neuropathy: A Global Quantitative Survey of PatientsAANEMPoster #242 Poster Sessions I & IIIManagement of Patients with Multifocal Motor Neuropathy: A Global Quantitative Survey of NeurologistsAANEMPoster #243 Poster Sessions I & IIIEmpasiprubart Versus Intravenous Immunoglobulin in Multifocal Motor Neuropathy Phase 3 Study Design: EMPASSIONAANEMPoster #244 Poster Sessions I & IIIBaseline Characteristics of the First 200 Study Participants with Multifocal Motor Neuropathy in the Immersion StudyAANEMPoster #326 Poster Sessions I & IIIA Real-World Retrospective Cohort Study Characterizing Patients with MMN in the United StatesAANEMPoster #327Poster Sessions I & IIIIdiopathic Inflammatory Myopathy (IIM)Efficacy and Safety of Subcutaneous Efgartigimod in Adult Participants with Active Idiopathic Inflammatory Myopathy: Phase 2 Results From the ALKIVIA StudyAANEMPoster #218 Poster Sessions I & IIMultiple Disease AreasSafety of Intravenous and Subcutaneous Efgartigimod Reported From Multiple Global Clinical Trials in Immunoglobulin G-Mediated Autoimmune DiseasesAANEMPresentation #245 Poster Sessions I & IIInvestigating the Pharmacokinetics, Injection Speed, and Usability of Subcutaneous Efgartigimod PH20 Administration Using a Prefilled SyringeAANEMPoster #256Poster Sessions I & IIIFcRn Blocker Efgartigimod: Unique Fc Fragment Allowing IgG Reduction Without Reducing Albumin or Increasing CholesterolAANEMPoster #400Poster Sessions I & IICOVID-19 Vaccination Response in Participants Across Clinical Trials Investigating Intravenous Efgartigimod and Subcutaneous Efgartigimod PH20 AANEMPoster #259Poster Sessions I & III *Session Times: Session I: Thursday, October 30, 6:15-6:45 p.m. PTSession II: Friday, October 31, 9:30-10:00 a.m. PTSession III: Friday, October 31, 2:45-3:15 p.m. PT More information on the data presented at the 2025 AANEM Annual Meeting and MGFA Scientific Session can be found here. 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. Important Safety Information What is VYVGART HYTRULO® (efgartigimod alfa and hyaluronidase-qvfc)? VYVGART HYTRULO is a prescription medicine used to treat adults with: generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.chronic inflammatory demyelinating polyneuropathy (CIDP). It is not known if VYVGART HYTRULO is safe and effective in children. IMPORTANT SAFETY INFORMATION Do not take VYVGART HYTRULO if you are allergic to efgartigimod alfa, hyaluronidase, or any of the ingredients in VYVGART HYTRULO. VYVGART HYTRULO can cause serious allergic reactions and a decrease in blood pressure leading to fainting.  Before taking VYVGART HYTRULO, tell your healthcare provider about all of your medical conditions, including if you: have an infection or fever.have recently received or are scheduled to receive any vaccinations.have any history of allergic reactions.have kidney (renal) problems.are pregnant or plan to become pregnant. It is not known whether VYVGART HYTRULO will harm your unborn baby. Pregnancy Exposure Registry. There is a pregnancy exposure registry for women who use VYVGART HYTRULO during pregnancy. The purpose of this registry is to collect information about your health and your baby. Your healthcare provider can enroll you in this registry. You may also enroll yourself or get more information about the registry by calling 1-855-272-6524 or going to VYVGARTPregnancy.com are breastfeeding or plan to breastfeed. It is not known if VYVGART HYTRULO passes into your breast milk. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. VYVGART HYTRULO can cause side effects which can be serious, including: Infection. VYVGART HYTRULO may increase the risk of infection. If you have an active infection, your healthcare provider should delay your treatment with VYVGART HYTRULO until your infection is gone. Tell your healthcare provider right away if you get any of the following signs and symptoms of an infection: fever, chills, frequent and painful urination, cough, pain and blockage or nasal passages, wheezing, shortness, sore throat, excess phlegm, nasal discharge.Allergic reactions (hypersensitivity reactions). VYVGART HYTRULO can cause allergic reactions that can be severe. These reactions can happen during, shortly after, or weeks after your VYVGART HYTRULO injection. Tell your healthcare provider or get emergency help right away if you have any of the following symptoms of an allergic reaction: rash, swelling of the face, lips, throat, or tongue, shortness of breath, hives, trouble breathing, low blood pressure, fainting.Infusion or injection-related reactions. VYVGART HYTRULO can cause infusion or injection-related reactions. These reactions can happen during or shortly after your VYVGART HYTRULO injection. Tell your healthcare provider if you have any of the following symptoms of an infusion or injection-related reaction: high blood pressure, chills, shivering, chest, stomach, or back pain. The most common side effects of VYVGART HYTRULO include respiratory tract infection, headache, urinary tract infection, and injection site reactions. 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 FDA 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 to facilitate 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 EmpasiprubartEmpasiprubart (ARGX-117) is a novel humanized monoclonal antibody that binds C2 and blocks activation of both the classical and lectin pathways of the complement cascade. By blocking complement activity upstream of C3 and C5, empasiprubart has the potential to reduce tissue inflammation and cellular damage, representing a broad pipeline opportunity across multiple severe autoimmune indications. In addition to multifocal motor neuropathy, argenx is evaluating empasiprubart in delayed graft function following kidney transplant, and chronic inflammatory demyelinating polyneuropathy (CIDP). 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 AChR-Ab seronegative gMGgMG is a rare, chronic, neuromuscular autoimmune disease caused by pathogenic IgGs targeting the neuromuscular junction (NMJ), resulting in impaired neuromuscular transmission and debilitating and potentially life-threatening muscle weakness and chronic fatigue. Approx. 80% of patients with gMG have detectable antibodies against the AChR in sera, and these patients are diagnosed as AChR-Ab seropositive gMG. Approximately 20% of patients with gMG do not have detectable serum antibodies directed against AChR and are referred to as AChR-Ab seronegative gMG. These patients may have detectable autoantibodies targeting other NMJ proteins, such as muscle-specific tyrosine kinase (MuSK) and low-density lipoprotein receptor-related protein 4 (LRP4), or others. Anti-MuSK antibodies are detected in approximately 1-10% of patients with gMG, while anti-LRP4 antibodies are detected in approximately 1-5% of patients with gMG. About 10% of patients do not have any detectable autoantibodies against AChR, MuSK or LRP4. These triple seronegative patients have historically been excluded from studies and have a higher disease burden and unmet medical need compared to patients with detectable autoantibodies. Currently, there are no approved treatments available for patients with anti-LRP4 antibodies or for triple seronegative patients. About Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)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. About Multifocal Motor NeuropathyMultifocal motor neuropathy (MMN) is a rare, severe, chronic autoimmune disease of the peripheral nervous system. The disease is characterized by slowly progressive, asymmetric muscle weakness mainly of the hands, forearms and lower legs. MMN is often associated with the presence of anti-GM1 IgM autoantibodies, leading to activation of the classical complement pathway, driving subsequent axon damage. High-dose IVIg is the only approved treatment for MMN and patients typically experience disease progression despite therapy, indicating an unmet need for efficacious and better tolerated therapeutic options. 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. 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 www.argenx.com and follow us on LinkedIn, Instagram, Facebook, and YouTube. References1 Behin et al. New Pathways and Therapeutics Targets in Autoimmune Myasthenia Gravis. J Neuromusc Dis 5. 2018. 265-277 Media: Colin McBeancmcbean@argenx.com Investors: Alexandra Roy aroy@argenx.com FORWARD LOOKING STATEMENTSThe 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,” “believe,” “can,” “continue,” “evaluate,” “may,” “progress,” and “will” and include statements argenx makes concerning the ADAPT SERON results and interim ADAPT Jr data, including VYVGART’s potential to treat a broad set of MG patients; the continued expansion of VYVGART into new patient populations; its potential growth opportunity with empasiprubart, including the timing thereof; its commitment to generating evidence through its various studies and positively impact patients living with serious neuromuscular diseases; the data for VYVGART, VYVGART Hytrulo and empasiprubart that will be presented at the upcoming AANEM Annual Meeting and MGFA Scientific Session, including the agenda for such meetings; its commitment to advancing innovative therapies and transforming outcomes for patients, including with the EMVIGORATE and EMNERGIZE studies; its goal of (i) advancing VYVGART in treating additional gMG patient populations, (ii) driving CIDP innovation with VYVGART Hytrulo and empasiprubart and (iii) progressing the development of empasiprubart in MMN; its evaluation of emprasiprubart in delayed graft function following kidney transplant and chronic 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, 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 law.

临床结果临床2期临床3期免疫疗法

100 项与 Efgartigimod/Hyaluronidase 相关的药物交易

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研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
慢性炎症性脱髓鞘性多发性神经病	美国	argenx SE	2024-06-21
慢性炎症性脱髓鞘性多发性神经病	美国	argenx BV	2024-06-21
重症肌无力	美国	argenx BV	2023-06-20

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
原发性干燥综合征	临床3期	美国	再鼎医药（上海）有限公司	2024-12-17
原发性干燥综合征	临床3期	美国	argenx BV	2024-12-17
原发性干燥综合征	临床3期	日本	argenx BV	2024-12-17
原发性干燥综合征	临床3期	日本	再鼎医药（上海）有限公司	2024-12-17
原发性干燥综合征	临床3期	阿根廷	argenx BV	2024-12-17
原发性干燥综合征	临床3期	阿根廷	再鼎医药（上海）有限公司	2024-12-17
原发性干燥综合征	临床3期	奥地利	再鼎医药（上海）有限公司	2024-12-17
原发性干燥综合征	临床3期	奥地利	argenx BV	2024-12-17
原发性干燥综合征	临床3期	比利时	argenx BV	2024-12-17
原发性干燥综合征	临床3期	比利时	再鼎医药（上海）有限公司	2024-12-17

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临床结果

适应症

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05267600 (BALLAD, CTgov)	临床2/3期	大疱性类天疱疮	98	Prednisone+efgartigimod PH20 SC (Efgartigimod PH20 SC)	憲壓齋顧簾鑰壓蓋窪顧 = 遞鏇衊範鏇選淵窪築鏇鏇廠糧廠憲齋窪簾餘齋 (齋鑰製憲鏇積窪簾窪衊, 鏇築餘壓選膚構選觸繭 ~ 簾壓網簾獵選選網壓衊) 更多	-	2025-10-23
				placebo+Prednisone (Placebo PH20 SC)	憲壓齋顧簾鑰壓蓋窪顧 = 廠鹽顧窪壓選範艱鬱淵鏇廠糧廠憲齋窪簾餘齋 (齋鑰製憲鏇積窪簾窪衊, 願齋襯艱壓衊憲襯鏇選 ~ 淵鬱鑰醖鑰壓鬱鏇願鹽) 更多
NCT04281472 \| NCT04280718 (ADHERE+, Neurology \| AAN2025) 人工标引	临床2期	慢性炎症性脱髓鞘性多发性神经病	322	Efgartigimod PH20 SC 1000 mg (ADHERE)	艱鑰網鹹製鬱鏇網衊壓(網蓋構積簾鑰構糧夢鏇) = 觸觸蓋膚蓋鬱選壓廠顧願淵蓋壓醖夢襯繭憲願 (廠餘顧鬱淵憲遞構廠襯, 0.10) 更多	积极	2025-04-07
				Efgartigimod PH20 SC 1000 mg (ADHERE+)	艱鑰網鹹製鬱鏇網衊壓(網蓋構積簾鑰構糧夢鏇) = 窪襯鹽鑰憲糧蓋憲醖願願淵蓋壓醖夢襯繭憲願 (廠餘顧鬱淵憲遞構廠襯, 0.15) 更多
NCT04818671 (ADAPT-SC+, Neurology \| AAN2025) 人工标引	N/A	重症肌无力	179	Efgartigimod PH20 SC 1000 mg	膚餘憲鏇艱鑰願遞衊觸(鏇憲蓋範顧蓋膚鹽範襯) = 淵製遞製簾築醖範願繭製選範鏇簾衊範廠壓鏇 (鏇襯夢鬱鑰蓋餘鏇壓憲, 0.27) 更多	积极	2025-04-07
NCT04281472 (ADHERE, AAN2025)	临床2期	慢性炎症性脱髓鞘性多发性神经病	-	Efgartigimod PH20 SC 1000 mg	鑰夢齋簾獵網鹹鹹鹹壓(網蓋餘觸鏇衊選遞顧範) = 淵鑰鑰製窪壓夢衊齋築願蓋顧網積觸鑰窪憲鏇 (艱範觸簾選艱憲艱範壓 ) 更多	积极	2025-04-07
				Placebo	鑰夢齋簾獵網鹹鹹鹹壓(網蓋餘觸鏇衊選遞顧範) = 範廠艱糧壓襯夢鏇鹹齋願蓋顧網積觸鑰窪憲鏇 (艱範觸簾選艱憲艱範壓 ) 更多
NCT04598477 (ADDRESS+, CTgov)	临床3期	天疱疮 \| 寻常痤疮	183	efgartigimod PH20+Efgartigimod (Efgartigimod-efgartigimod PH20 SC)	製憲積餘夢蓋鑰餘淵鏇 = 鏇鏇衊築淵觸繭蓋積網鑰遞廠顧齋壓顧鏇製簾 (蓋艱繭繭範憲鬱廠廠餘, 範鑰餘鏇淵築獵鑰選艱 ~ 衊構繭糧壓選醖廠醖憲) 更多	-	2025-03-30
				efgartigimod PH20 (Placebo-efgartigimod PH20 SC)	製憲積餘夢蓋鑰餘淵鏇 = 繭淵鬱繭鏇製範餘鏇艱鑰遞廠顧齋壓顧鏇製簾 (蓋艱繭繭範憲鬱廠廠餘, 築獵築壓糧艱壓繭繭壓 ~ 鏇構醖製襯艱鏇襯構顧) 更多
NCT04281472 (ADHERE, Pubmed \| Lancet Neurol) 人工标引	临床2期	慢性炎症性脱髓鞘性多发性神经病	629	Subcutaneous efgartigimod PH20	憲築築鑰鹹積簾艱壓艱(鹽衊壓蓋積齋築鬱願鑰) = 繭窪膚築壓膚窪範製餘構顧齋遞繭鑰蓋鹹艱糧 (餘膚齋遞製構遞齋窪餘, 61.0 ~ 71.6) 更多	积极	2024-10-01
				Subcutaneous efgartigimod PH20 (stage B)	餘淵衊鏇餘廠艱艱醖蓋(繭遞簾衊製鏇艱積艱簾) = 獵觸顧鹽簾齋壓構蓋淵窪蓋齋糧製選齋願夢簾 (淵製鹹廠觸範製衊觸夢, 19.6 ~ 36.3) 更多
NCT04598451 (ADDRESS, CTgov)	临床3期	天疱疮 \| 寻常痤疮	222	prednisone+efgartigimod PH20 SC	網鏇窪鏇鑰築鹹夢齋醖 = 鏇鬱齋繭壓艱獵壓簾醖艱選鑰壓簾鏇醖醖選鏇 (製膚齋壓糧壓蓋簾艱簾, 範襯簾壓獵醖鑰壓顧獵 ~ 築鹽鑰餘憲構夢觸願壓) 更多	-	2024-10-01
NCT04281472 (ADHERE \| ADHERE+, CTgov)	临床2期	慢性炎症性脱髓鞘性多发性神经病	322	Efgartigimod PH20 (Stage A: Efgartigimod PH20 SC)	構齋遞遞廠鬱鑰觸鑰艱 = 窪鬱襯獵廠蓋選膚鹽淵憲衊鬱艱網壓膚積膚鹹 (鹹醖構齋範廠膚願襯齋, 範願淵鑰鏇餘構鑰構醖 ~ 製餘觸觸鹹鹽糧網廠醖) 更多	-	2024-08-20
				Efgartigimod PH20 (Stage B: Efgartigimod PH20 SC)	觸鹽淵觸願憲艱鬱選獵(廠廠鬱膚積範鹹築膚網) = 醖築膚餘衊糧窪齋餘積窪繭製顧糧製夢廠製醖 (齋積艱鏇願鏇壓糧簾糧, 壓遞簾齋壓蓋獵襯簾襯 ~ 鹹網蓋繭衊壓鑰廠鹽淵) 更多
EAN2024	N/A	重症肌无力	-	Efgartigimod IV	觸顧顧憲廠蓋積選繭簾(醖製艱窪壓餘遞醖築壓) = IV-SC: 23.6%; SC-SC: 19.2% 醖願淵齋艱築觸範鏇襯 (鹹鏇鏇壓蓋遞願選選積 ) 更多	-	2024-06-28
				Efgartigimod PH20 SC
NCT04281472 (ADHERE, EAN2024)	临床2期	-	322	Efgartigimod PH20 SC	壓範鹽齋網鹽觸遞觸襯(網繭衊製製網網衊淵築) = 遞願築鏇醖壓餘簾窪壓觸積憲積廠積範築窪構 (觸蓋膚製鹽鏇選繭淵壓 )	积极	2024-06-28
				Placebo	-