The aims of this study are to assess the real-world effectiveness of efgartigimod in treating chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), describe the "treatment journey" of participants with CIDP, and assess the utilization of health care services among adult participants with CIDP who initiate treatment with efgartigimod.
As this is a noninterventional study, treatment choices and decisions will be left to the discretion of participants and their physicians, according to the standard of care.
Each participant will be prospectively followed for up to 2 years from the date of initial administration of efgartigimod.

Start Date11 Aug 2025

Sponsor / Collaborator

argenx SE

CTR20252164

/ RecruitingPhase 3

一项在成人原发性干燥综合征受试者中评价预充式注射器皮下给与Efgartigimod PH20 的有效性、安全性和耐受性的III 期、随机、双盲、安慰剂对照、多中心研究接开放标签扩展研究

[Translation] A Phase III, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy, safety, and tolerability of Efgartigimod PH20 administered subcutaneously via a prefilled syringe in adult subjects with primary Sjögren's syndrome followed by an open-label extension

通过clinESSDAI 比较Efgartigimod PH20 SC 与安慰剂的系统疾病活动度评价有效性

[Translation]

Efficacy of Efgartigimod PH20 SC versus placebo in systemic disease activity assessment by clinESSDAI

Start Date30 Jun 2025

Sponsor / Collaborator

Zai Lab (Shanghai) Co., Ltd.

[+2]

NCT06637072

/ Active, not recruitingPhase 4

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.

Start Date10 Dec 2024

Sponsor / Collaborator

argenx SE

100 Clinical Results associated with Efgartigimod/Hyaluronidase

100 Translational Medicine associated with Efgartigimod/Hyaluronidase

100 Patents (Medical) associated with Efgartigimod/Hyaluronidase

Literatures (Medical) associated with Efgartigimod/Hyaluronidase

01 Dec 2024·DRUGS IN R&D

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

Article

Author: Reinhart, Harald ; Chen, Jiajia ; Van Bragt, Tonke ; Gu, Xiaowen ; Noukens, Jan ; Jing, Shan ; Wang, Lina ; 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.

01 Dec 2024·ADVANCES IN THERAPY

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

Article

Author: 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.

01 Nov 2024·DRUGS

Efgartigimod: A Review in Generalised Myasthenia Gravis

Review

Author: 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.

115

News (Medical) associated with Efgartigimod/Hyaluronidase

12 Jan 2026

·www.globenewswire.com

argenx Highlights 2026 Strategic Priorities

Reported $4.15 billion (YoY growth of +90%) in preliminary* full-year 2025 global product net sales, inclusive of $1.29 billion in fourth quarter sales VYVGART impact continues with approximately 19,000 patients on treatment; and if approved, AChR-Ab seronegative gMG launch expected by end of 2026 Four registrational readouts expected in 2026, including first for empasiprubart, to advance toward next wave of 2027 commercial launches Successfully advanced four new pipeline molecules in 2025; three new molecules to enter Phase 1 in 2026, contributing to total of 10 clinical-stage molecules by year-end January 12, 2026, 7:00 a.m. CETAmsterdam, the Netherlands – argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today reported preliminary financial results for the full-year 2025, including global product net sales, and announced its strategic priorities for 2026. “argenx enters 2026 in a position of strength, delivering meaningful impact to approximately 19,000 patients globally while advancing a world-class pipeline toward Vision 2030,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “With VYVGART leading the growth of all biologics in MG and CIDP, we are proving the power of our approach: to redefine treatment paradigms through disciplined evidence generation and to redefine patient outcomes with medicines that are both more effective and more convenient. This same playbook will guide our future, as we aim to launch a portfolio of new medicines that could transform the lives of more than 50,000 patients across 10 indications.” “Looking at the year ahead, we will expand our FcRn franchise and report the first Phase 3 data for our next potential blockbuster medicine, with four registrational readouts across both efgartigimod and empasiprubart. In addition, we will continue to grow intentionally, by sourcing innovation where the best science emerges, nurturing an entrepreneurial culture, and scaling with discipline to deliver long-term, durable value for patients and shareholders,” added Mr. Van Hauwermeiren. 2026 Strategic Priorities argenx continues to advance its ‘Vision 2030’, anchored in the ambition to treat 50,000 patients globally with its medicines, secure 10 labeled indications across approved medicines, and progress five pipeline candidates into Phase 3 development by 2030. 2026 marks a defining year on the path to Vision 2030 with three strategic priorities: Impact more patients globally with VYVGART, driving broader adoption across current patient populations and unlocking new opportunities with potential label expansionsShape the long-term future of FcRn medicines, advancing future FcRn molecules, innovative delivery modalities and combination approaches designed to transform patient outcomesDeliver next wave of immunology innovation, accelerating empasiprubart and diversified pipeline of first-in-class molecules to drive sustainable value creation Impact more patients globally with VYVGART VYVGART® (IV: efgartigimod alfa-fcab and SC: efgartigimod alfa and hyaluronidase-qvfc) is a first-and-only IgG Fc-antibody fragment that targets the neonatal Fc receptor (FcRn). It is approved in three indications, including generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP) globally, and primary immune thrombocytopenia (ITP) in Japan. argenx aims to drive broad adoption across patients globally, reinforcing VYVGART’s position as the leading precision biologic in MG and CIDP and continuing to raise the bar for patient outcomes. The company is progressing toward multiple label expansions. This includes seeking the broadest label of any biologic in MG with the seronegative MG launch, if approved, and an ocular MG Phase 3 readout, and in ITP with the Phase 3 readout to support a U.S. launch. Submitted supplemental Biologics License Application (sBLA) for VYVGART IV for anti-acetylcholine receptor antibody negative gMG (MuSK+, LRP4+ and triple seronegative); if approved, launch expected by end of 2026Topline results expected for ocular MG (ADAPT OCULUS) in first quarter of 2026Topline results expected for primary ITP (ADVANCE-NEXT) in fourth quarter of 2026Registrational studies are ongoing in two rheumatology indications Topline results from ALKIVIA study evaluating autoimmune inflammatory myopathies (AIM or myositis) expected in third quarter of 2026Topline results from UNITY study (Sjogren’s disease) expected in second half of 2027 Registrational study in Graves’ disease (GD) expected to initiate in 2026, expanding development into thyroid-driven autoimmunityProof-of-concept studies ongoing in systemic sclerosis, antibody mediated rejection and autoimmune encephalitisExpanded global presence in Latin America with establishment of argenx Brazil in 2025 Shape the long-term future of FcRn medicines argenx is shaping the long-term future of FcRn medicines by advancing new pipeline candidates, innovative delivery modalities, and combination approaches to set new standards for patients. Two future FcRn molecules are progressing: ARGX-213, an FcRn-targeted antibody engineered for half-life extension and sustained IgG reduction, and ARGX-124, a first-in-class FcRn pipeline candidate. The ADAPT-Forward study is now underway, which is the first in a series of trials exploring efgartigimod-anchored combinations to potentially improve patient outcomes. VYVGART SC autoinjector expected to launch in 2027, reinforcing a commitment to continued flexibility and independence for patientsADAPT-Forward combination study ongoing to evaluate empasiprubart as an add‑on therapy to efgartigimod, exploring potential for even deeper efficacy in AChR‑positive gMG patientsARGX-213 is expected to enter patient studies in 2026ARGX-124 is expected to complete Phase 1 evaluation by end of 2026Innovation in FcRn continues through partnerships with Elektrofi (now part of Halozyme) and Unnatural Products to further enhance the patient experience Deliver next wave of immunology innovation By the end of 2026, the argenx pipeline will include four Phase 3 molecules and a total of 10 molecules in clinical development. Empasiprubart, a first-in-class antibody targeting C2, is in Phase 3 for MMN and CIDP, and adimanebart (ARGX-119), a first-in-class agonist antibody targeting muscle-specific kinase (MuSK), will enter Phase 3 for congenital myasthenic syndromes (CMS). Additional proof-of-concept studies are underway to further explore C2 and MuSK biology. In 2025, four new candidates emerged from the Immunology Innovation Program (IIP), argenx’s engine for sourcing novel biology and accelerating differentiated medicines. These include FcRn candidates ARGX-213 and ARGX-124, and ARGX-109 (targeting IL-6) and ARGX-121 (a first-in-class molecule targeting IgA). Three additional molecules from the IIP are expected to enter Phase 1 in 2026, supporting argenx’s goal of launching, on average, one new pipeline candidate each year. Empasiprubart Topline results from EMPASSION study (MMN) expected in fourth quarter of 2026Topline results from EMVIGORATE and EMNERGIZE studies (CIDP) expected in second half of 2027Decision for Phase 2 VARVARA study (DGF) now expected mid-year 2026 to complete 52-week efficacy analysis Adimanebart CMS registrational study on track to start in third quarter of 2026Proof-of-concept studies ongoing in amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA) Earlier-stage Programs Phase 2 study of ARGX-121 in IgA nephropathy (IgAN) expected to start in 2026Entered into a research collaboration with Tensegrity Pharma, including an option for future acquisition, to advance Tensegrity’s lead program TSP-101 in autoimmune disease and other indications.Three new molecules expected to enter Phase 1 studies in 2026, including ARGX-118, a first-in-class molecule targeting Galectin-10, ARGX-125, a first-in-class bispecific antibody, and TSP-101, targeting Fn14 Corporate Highlights argenx recently announced that Karen Massey, current Chief Operating Officer, will transition to Chief Executive Officer and Executive Director, and Tim Van Hauwermeiren, current Chief Executive Officer, will transition to non-Executive Director and Chairman of the Board of Directors. Tim will succeed Peter Verhaeghe, who is retiring from the Board of Directors. These changes are subject to shareholder approval at the Annual General Meeting on May 6, 2026. In addition, Sandrine Piret-Gérard has been appointed Chief Commercialization Officer. Sandrine brings extensive commercial and medical affairs experience, most recently leading the U.S. commercial organization at Gilead across virology and oncology. Preliminary* Key Fourth Quarter and Full-Year 2025 Financial Results Today, argenx also announced preliminary* global product net sales for the fourth quarter and full-year 2025 of approximately $1.29 billion and $4.15 billion, respectively. *The preliminary selected financial information is unaudited, subject to adjustment, and provided as an approximation in advance of the company’s announcement of complete financial results in February 2026. Refer to the Preliminary Financial Results note in this document. 44th Annual J.P. Morgan Healthcare Conference Presentation and Webcast CEO Tim Van Hauwermeiren will highlight these updates in a corporate presentation at the 44th Annual J.P. Morgan Healthcare Conference today, Monday, January 12, 2026, at 8:15 a.m. PT. The live webcast of the presentation may be accessed under the Investor section on the argenx website. A replay will be available for 30 days following the presentation. About VYVGART and VYVGART SC VYVGART® (efgartigimod alfa fcab) 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 for the treatment of generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP) globally, and for primary immune thrombocytopenia (ITP) in Japan. VYVGART SC is a subcutaneous combination of efgartigimod alfa and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. It is marketed as VYVGART® Hytrulo in the U.S., VYVGART SC in Europe, VYVDURA® in Japan, and may be marketed under different proprietary names following approval in other regions. 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. This press release contains inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation (Regulation 596/2014). Media:Ben Petokbpetok@argenx.com Investors:Alexandra Royaroy@argenx.com Preliminary Financial Results The financial information presented in this press release is preliminary, estimated, and unaudited. They are subject to the completion and finalization of argenx’s financial and accounting closing procedures. They reflect management’s estimates based solely upon information available to management as of the date of this press release. Further information learned during that completion and finalization may alter the final results. In addition, the preliminary estimates should not be viewed as a substitute for full quarterly and annual financial statements prepared in accordance with IFRS. There is a possibility that argenx’s financial results for the quarter ended December 31, 2025, and full year financial results for 2025 could vary materially from these preliminary estimates. In addition to the completion of the financial closing procedures, factors that could cause actual results to differ from those described above are set forth below. Accordingly, you should not place undue reliance upon this preliminary information. Additional information regarding the company’s fourth quarter 2025 financial results and full year financial results for 2025 will be available in the company’s annual report and Form 20-F, which will be filed with the Netherlands Authority for the Financial Markets and U.S. Securities and Exchange Commission (the “SEC”), respectively. 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,” “commit,” “continue,” “deepen,” “develop,” “expect,” “grow,” “potential,” “progress,” and “will,” and include statements argenx makes concerning its continued impact of VYVGART and if approved, the potential launch of AChR-Ab seronegative gMG launch by end of 2026; its four registrational readouts expected in 2026 to advance efgartigimod and empasiprubart towards next wave of 2027 commercial launches; the advancement of its pipeline, with three new molecules to enter Phase 1 in 2026, contributing to total of 10 clinical-stage molecules by year-end; its advancement of a world-class pipeline toward Vision 2030; its continued advancement of Vision 2030, anchored in the ambition to treat 50,000 patients globally with its medicines, secure 10 labeled indications across approved medicines, and progress five pipeline candidates into Phase 3 development by 2030; its intentional growth by sourcing innovation where the best science emerges, preserving its entrepreneurial culture, and scaling with discipline to deliver long-term, durable value for patients and shareholders; its belief that 2026 will be a defining year that will deepen its immunology leadership and broaden patient impact, including its goal to impact more patients globally with VYVGART by driving broader adoption across current patient populations while unlocking new opportunities through potential label expansions; the timing of a potential (1) launch in AChR- seronegative MG, and (2) Phase 3 readouts in ocular MG and ITP; the advancement of anticipated clinical development, data readouts and regulator milestones and plans, including: (1) the potential approval and launch by the end of 2026 of an sBLA for VYVGART IV for anti-acetylcholine receptor antibody negative gMG (MuSK+, LRP4+ and triple seronegative; (2) topline results for ocular MG (ADAPT OCULUS) in first quarter of 2026; (3) topline results for primary ITP (ADVANCE-NEXT) in fourth quarter of 2026 to support label expansion outside Japan; (4) ongoing registrational studies in two rheumatology indications, in Graves’ disease, and in multiple proof-of-concept indications, including: (a) potential topline results from ALKIVIA study evaluating autoimmune inflammatory myopathies (AIM or myositis) in third quarter of 2026; (b) potential topline results from UNITY study (Sjögren’s disease) in second half of 2027; (c) potential initiation of a registrational study in Graves’ disease (GD) in first half of 2026, expanding development into thyroid-driven autoimmunity; (5) broadened global presence in Latin America through establishment of argenx Brazil in 2025; the progression of ARGX-213 and ARGX-124; the progression of a series of trials exploring efgartigimod-anchored combinations to potentially improve patient outcomes, including: (1) the potential launch of VYVGART SC autoinjector in 2027; (2) the ongoing ADAPT-Forward combination study; (3) potential studies for ARGX-213 in 2026; (4) potential completion of ARGX-124’s Phase 1 evaluation by end of 2026; and (5) continued innovation in FcRn through partnerships with Elektrofi (now part of Halozyme) and Unnatural Products; its goal to have five Phase 3 molecules and a total of 10 molecules in clinical development by the end of 2026; the additional proof-of-concept studies underway to further explore the potential of C2 and MuSK biology, with: (1) potential topline results from EMPASSION study (MMN) for empasiprubart in fourth quarter of 2026; (2) potential topline results for empasiprubart from EMVIGORATE and EMNERGIZE studies (CIDP) in second half of 2027; (3) potential for decision for Phase 2 VARVARA study (DGF) for empasiprubart now mid-year 2026 to complete 52-week efficacy analysis; (4) potential for CMS registrational study for adimanebart to start in third quarter of 2026; and (5) proof-of-concept studies for adimanebart ongoing in amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA); its goal to launch at least one new pipeline candidate each year on a go-forward basis, with: (1) IgA nephropathy (IgAN) nominated as first Phase 2 indication to be explored with ARGX-121; (2) its research collaboration with Tensegrity Pharma; (3) the entry of three new molecules into its pipeline in 2026, including ARGX-118, ARGX-125, and TSP-101; its aim to shape the long-term future of FcRn medicines, and deliver the next wave of immunology innovation; its commitment to improve the lives of people suffering from severe autoimmune diseases; its belief that its priorities for VYVGART will reinforce its position as the leading precision biologic and continue to raise the bar on patient outcomes; its aim to translate 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; its anticipated leadership changes; its future financial and operating performance, including its anticipated global product net sales for Q4 2025 and FY 2025; 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.

Phase 2Drug ApprovalExecutive ChangePhase 3Phase 1

05 Jan 2026

·www.fiercepharma.com

As Vyvgart empire expands, argenx CEO passes reins to operating chief Karen Massey

Since the first approval of its flagship FcRn blocker, Vyvgart, in late 2021, argenx has ascended swiftly from biotech upstart to a commercial force in the rare disease space. After shepherding the company through its first approval and the successful launch of blockbuster autoimmune drug Vyvgart, argenx co-founder and chief executive Tim Van Hauwermeiren is passing the reins to the Dutch drugmaker’s operating chief.Karen Massey, who joined the Amsterdam-based immunology specialist as chief operating officer in early 2023, is now set to assume the role of CEO, the company announced on Monday. Hauwermeiren, who’s been with argenx since its inception in 2008, will take a step back to serve as a non-executive director and chairman of the company’s board.Van Hauwermeiren is himself succeeding Peter Verhaeghe as chairman of the board of directors, with the various leadership changes subject to shareholder approval at argenx’s upcoming annual meeting in early May, the company noted in its press release.“This leadership evolution comes at the right time and represents a natural step as we prepare for the next phase of growth at argenx,” Verhaeghe said in a statement Monday. “The board and I move forward with strong confidence in Tim as our incoming chairman, Karen as our trusted COO and incoming CEO, and the entire leadership team, to guide the organization towards Vision 2030 and beyond.”Since the first approval of its flagship FcRn blocker, Vyvgart (efgartigimod), in late 2021, argenx ascended swiftly from biotech upstart to a commercial force in the rare disease space. These days, the company can tout blockbuster sales of its drug across several indications and a market cap of around $50 billion.Argenx has yet to report full-year revenue for 2025, but in 2024, the company recorded $2.2 billion in product sales, buoyed exclusively by its efgartigimod franchise. In 2025’s third quarter, sales of Vyvgart and Vyvgart Hytrulo clocked in at $1.13 billion, growing 96% over the same period in 2024.Next on the docket is argenx’s Vision 2030 plan, unveiled at the company's R&D day back in 2024, under which the company has set the goal to push five new preclinical assets into phase 3 development and hold 10 approved indications across Vygart and other near-term potential launches by the start of the next decade. Argenx is also striving to reach 50,000 patients with its current and future medicines under that same 2030 deadline. “This is a special company with a bright future that is founded on the strength of our science, our entrepreneurial culture, and the deep accountability of our talented teams to transform the lives of our patients,” Massey said in argenx’s announcement Monday.“My commitment is to elevate the unique argenx DNA as we execute on Vision 2030 and beyond and to ensure we are making the most of this bold opportunity to build the biotech company of the future,” she continued.Much of argenx’s late-stage development work currently revolves around infused Vyvgart and its subcutaneous sibling Vyvgart Hytrulo. The company’s next most advanced asset, the C2-targeting antibody empasiprubart, is currently in phase 3 testing for multifocal motor neuropathy (MMN) and chronic inflammatory demyelinating polyneuropathy (CIDP).Argenx has largely prioritized the subcutaneous Vyvgart Hytrulo over its intravenous predecessor in its efforts. Vyvgart Hytrulo was initially cleared by the FDA in generalized myasthenia gravis (gMG) in June 2023 and subsequently picked up a U.S. green light in chronic inflammatory demyelinating polyneuropathy (CIDP) the following year.More recently, the FDA approved a prefilled syringe formulation of Vyvgart Hytrulo that patients can inject themselves at home. Prior to that FDA nod, Massey told Fierce Pharma in an interview last year that the prefilled syringe could help shift Vyvgart into earlier lines of therapy in gMG and build on the momentum of the drug’s 2024 launch in CIDP. Massey is set to become one of the few women CEOs spearheading a large commercial pharma company, especially as two prominent female leaders in the industry have recently telegraphed or made their exits.Back in September, Merck KGaA indicated that Belén Garijo, who helped steer the German healthcare and electronics conglomerate through the COVID-19 pandemic, is planning to end her five-year run at the company in April. Meanwhile, GSK’s Emma Walmsley officially stepped down from her post at the British drug giant on Jan. 1, handing over the CEO title to the company’s former chief commercial officer Luke Miels.

Executive ChangePhase 3Drug Approval

15 Dec 2025

·www.fiercepharma.com

Argenx permanently grounds studies of high-flying Vyvgart in thyroid eye disease

Argenx is discontinuing its late-stage UplighTED studies of subcutaneous efgartigimod in adults with moderate to severe thyroid eye disease. While argenx has been able to demonstrate the merit of its FcRn drug Vyvgart in multiple autoimmune indications, not every shot in the clinic is destined to land.Now, facing that fact in thyroid eye disease (TED), argenx is walking away from a set of trials.Argenx is discontinuing its late-stage UplighTED studies of subcutaneous efgartigimod in adults with moderate to severe TED, the Dutch biopharma announced Monday. Efgartigimod is the generic name for Vyvgart, which is approved both as an infusion and as an injectable under the brand name Vyvgart Hytrulo, with the latter being the format tested in argenx’s study.The drugmaker is calling it quits on the Vyvgart Hytrulo TED program following a recommendation from an independent data monitoring committee, which urged argenx to stop the trials for futility after reviewing data at a preset interim analysis.There was a small silver lining insofar as Vyvgart Hytrulo’s safety and tolerability looked favorable in the trials, with no new safety flags cropping up, argenx pointed out. The company’s stock was down more than 5% in morning trading Dec. 15.The futility evaluation came at the studies’ 24-week mark, argenx explained. The company says it plans to parse through the data, once available, so it can better understand the “studies’ outcome and uncover key biological insights that may inform future research in TED.”The company noted that it will share the results at an upcoming medical meeting, as well. TED, an autoimmune disease affecting the eye socket, can cause muscles that control the eye and fat behind the organ to grow abnormally, and it can also lead to orbital inflammation. This can in turn cause proptosis (when the eyes bulge from their natural position), diplopia (double vision) or, in severe cases, vision loss, argenx explained in its release.The company’s UplighTED studies specifically looked at TED patients with the autoimmune thyroid conditions Graves’ disease or Hashimoto’s thyroiditis. The primary endpoint of the studies—which pitted Vyvgart Hytrulo against placebo—concerned the percentage of patients classified as “proptosis responders” at Week 24, argenx said.The trials also looked at secondary metrics such as the change in proptosis measurement from baseline, change in total score on a quality-of-life measure known as Graves’ Orbitopathy Quality of Life, and the percentage of trial participants whose diplopia had resolved, all at 24 weeks.“We are disappointed the studies did not meet our desired outcome and we especially empathize with patients who are living with TED and seeking new therapies for this challenging disease,” Luc Truyen, M.D., Ph.D., argenx’s chief medical officer, said in a statement. “We had pre-planned this futility analysis as it provides a meaningful interim evaluation of observed patient outcomes and enables us to responsibly evaluate the study’s future likelihood of success.”Since the original approval of infused Vyvgart in generalized myasthenia gravis (gMG) in late 2021, argenx has largely made good on its thesis that the asset has potential in a range of autoimmune diseases. For its part, Vyvgart Hyrulo—the drug’s subcutaneous formulation—is broadly approved in gMG and chronic inflammatory demyelinating polyneuropathy around the world, and the drug also boasts a nod to treat primary immune thrombocytopenia in Japan. In the clinic, argenx is trialing efgartigimod in numerous autoimmune diseases, from ocular myasthenia gravis and myositis to Sjogren’s disease and systemic sclerosis, among other potential indications.In the nearer term, the Dutch drugmaker is aiming for an FDA nod for both subQ and infused Vyvgart in the subset of gMG patients who are seronegative, meaning their condition lacks detectable levels of certain antibodies that are often characteristic of the disease. With results from a positive phase 3 seronegative trial in hand, argenx has said it plans to file for an FDA review in the indication by the end of 2025.

Phase 3Drug ApprovalClinical Result

100 Deals associated with Efgartigimod/Hyaluronidase

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	United States	argenx SE	21 Jun 2024
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	United States	argenx BV	21 Jun 2024
Myasthenia Gravis	United States	argenx BV	20 Jun 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Primary Sjögren's syndrome	Phase 3	United States	argenx BV	17 Dec 2024
Primary Sjögren's syndrome	Phase 3	United States	Zai Lab (Shanghai) Co., Ltd.	17 Dec 2024
Primary Sjögren's syndrome	Phase 3	Japan	Zai Lab (Shanghai) Co., Ltd.	17 Dec 2024
Primary Sjögren's syndrome	Phase 3	Japan	argenx BV	17 Dec 2024
Primary Sjögren's syndrome	Phase 3	Argentina	Zai Lab (Shanghai) Co., Ltd.	17 Dec 2024
Primary Sjögren's syndrome	Phase 3	Argentina	argenx BV	17 Dec 2024
Primary Sjögren's syndrome	Phase 3	Austria	Zai Lab (Shanghai) Co., Ltd.	17 Dec 2024
Primary Sjögren's syndrome	Phase 3	Austria	argenx BV	17 Dec 2024
Primary Sjögren's syndrome	Phase 3	Belgium	Zai Lab (Shanghai) Co., Ltd.	17 Dec 2024
Primary Sjögren's syndrome	Phase 3	Belgium	argenx BV	17 Dec 2024

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04818671 (ADAPTSC+ \| ADAPT-SC+, CTgov)	Phase 3	Myasthenia Gravis	184	efgartigimod PH20 SC	esnxrrtgtx = nvqsqletyg vodavvcwtb (ofpwkuhcez, oaslwejwmz - zeyeanblqh) View more	-	22 Jan 2026
NCT05523167 (ALKIVIA, ACR2025)	Phase 2/3	Idiopathic Inflammatory Myopathies	89	Efgartigimod PH20 SC plus standard of care (SOC)	zkmvoyfbxy(ozkazhcrnp) = hjeipdizne ousmcewztk (dkllgueclg ) View more	Positive	24 Oct 2025
				Placebo PH20 (PBO) plus SOC	zkmvoyfbxy(ozkazhcrnp) = xymzwrattm ousmcewztk (dkllgueclg ) View more
NCT05267600 (BALLAD, CTgov)	Phase 2/3	Pemphigoid, Bullous	98	Prednisone+efgartigimod PH20 SC (Efgartigimod PH20 SC)	jpvemgfczs = ixojboptjt hmqgeesxjn (mewpqcbpei, mwejbxnrmw - hcwljqpfim) View more	-	23 Oct 2025
				placebo+Prednisone (Placebo PH20 SC)	jpvemgfczs = clqommdizr hmqgeesxjn (mewpqcbpei, shxvgtugyp - bhnbzpokej) View more
NCT04818671 (ADAPT-SC+, Neurology \| AAN2025) Manual	Not Applicable	Myasthenia Gravis	179	Efgartigimod PH20 SC 1000 mg	mxanmowikc(dxnahacezz) = opuyknxiid gltpgzfuvm (pnuhmtqilj, 0.27) View more	Positive	07 Apr 2025
NCT04281472 \| NCT04280718 (ADHERE+, Neurology \| AAN2025) Manual	Phase 2	Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	322	Efgartigimod PH20 SC 1000 mg (ADHERE)	ubtmnynnmr(umidgmdjtf) = rgillnyeub xsvriippud (ajftclnvdn, 0.10) View more	Positive	07 Apr 2025
				Efgartigimod PH20 SC 1000 mg (ADHERE+)	ubtmnynnmr(umidgmdjtf) = nmcytrtwle xsvriippud (ajftclnvdn, 0.15) View more
NCT04281472 (ADHERE, AAN2025)	Phase 2	Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	-	Efgartigimod PH20 SC 1000 mg	lavdjuaojv(tjhrfrekde) = clnvbbsyat ptblnuatin (vszrohhkiq ) View more	Positive	07 Apr 2025
				Placebo	lavdjuaojv(tjhrfrekde) = scoyhjhyfi ptblnuatin (vszrohhkiq ) View more
NCT04598477 (ADDRESS+, CTgov)	Phase 3	Pemphigus \| Acne Vulgaris	183	efgartigimod PH20+Efgartigimod (Efgartigimod-efgartigimod PH20 SC)	ubugxshmyr = djjjwzysmf jhnmihvlkq (qusxdeuwhr, gmffuftlmd - hzsgjfoouj) View more	-	30 Mar 2025
				Placebo+efgartigimod PH20 (Placebo-efgartigimod PH20 SC)	ubugxshmyr = wjjbrtmbed jhnmihvlkq (qusxdeuwhr, jrornftpwa - iookizuidw) View more
NCT04281472 (ADHERE, Pubmed \| Lancet Neurol) Manual	Phase 2	Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	629	Subcutaneous efgartigimod PH20	hrznyozfdw(oddlpppyhu) = dsjxxmpxca twkrozjdup (ujiupsyeoh, 61.0 - 71.6) View more	Positive	01 Oct 2024
				Subcutaneous efgartigimod PH20 (stage B)	edqysmxzky(ctvfiwypxn) = aksmdprupp pdieuwkvlz (pollmqyuji, 19.6 - 36.3) View more
NCT04598451 (ADDRESS, CTgov)	Phase 3	Pemphigus \| Acne Vulgaris	222	prednisone+efgartigimod PH20 SC	mbroxdqcnm = fkvuuqzuwl lmkirlqabj (zqvjisadgd, mzshyqkept - oxfhteticn) View more	-	01 Oct 2024
NCT04281472 (ADHERE \| ADHERE+, CTgov)	Phase 2	Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	322	Efgartigimod PH20 (Stage A: Efgartigimod PH20 SC)	vnvxnllcko = uoiyhfdbfu stjjhehtfg (exfixevwco, cwzgqecohl - uhbfzxsixr) View more	-	20 Aug 2024
				Efgartigimod PH20 (Stage B: Efgartigimod PH20 SC)	ihdvijnmlz(hmkxfgpolg) = geozknatxq sjxrjnpwhm (lrttyswymb, rtjgeiugpb - lybdwfzqjg) View more

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