Efgartigimod/Hyaluronidase

ALKIVIA+ data in myositis indicate efgartigimod provides sustained, clinically meaningful improvements and consistent safety RHO+ data in Sjogren’s disease indicate maintenance of response following switch to biweekly dosing Cross-indication analysis of efgartigimod data across rheumatology studies underscores consistent safety pro large patient population June 3, 2026, 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, today announced the presentation of new data evaluating VYVGART® (IV: efgartigimod alfa-fcab and SC or Hytrulo: efgartigimod alfa and hyaluronidase-qvfc) across autoimmune rheumatic diseases, including myositis and Sjogren’s disease, to be presented at the European Alliance of Associations for Rheumatology (EULAR) 2026 Congress from June 3-6, 2026, in London, UK. “Our presentations at EULAR further support the rationale for targeting FcRn in autoimmune rheumatic diseases and underscore its potential to address significant unmet patient need,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer at argenx. “We are encouraged by the consistency of the long-term data as well as data underscoring efgartigimod's favorable safety pro multiple autoimmune rheumatic diseases. We are looking forward to additional results evaluating efgartigimod in myositis later this year as well as in Sjogren’s disease next year.” Myositis and Sjogren’s disease are both chronic, progressive autoimmune diseases that are generally more common in women than men. Myositis can cause serious and irreversible damage to muscles and organs, leading to a loss of independence and a significant burden for patients. Sjogren’s disease can cause dry eyes and mouth, chronic fatigue, and joint pain. It can also affect multiple organ systems and lead to nervous system complications. Efgartigimod provides sustained functional improvement in patients with myositis ALKIVIA+ is an ongoing open-label extension study that enrolled patients who completed the 24-week, double-blind, placebo-controlled Phase 2 ALKIVIA trial, including those who continued efgartigimod treatment and those who transitioned from placebo to efgartigimod. Results presented at EULAR are an interim analysis through Week 52. A key metric in ALKIVIA+ is Total Improvement Score (TIS). TIS is a composite index based on six core set measures, including muscle strength, physician and patient global assessments of disease activity, physical function, enzyme levels, and extramuscular activity. Higher TIS values reflect greater overall clinical improvement. Moderate and major improvement are defined as TIS ≥40 and ≥60, respectively. Results showed: At 52 weeks, 37.5% of patients who had continuously received efgartigimod maintained their major TIS improvement from week 24. 33.3% of patients switching from placebo to efgartigimod achieved a similar major TIS improvement. Rates of moderate TIS improvement were also similar between the groups, at 75.0% and 66.7%, respectively. Sustained mean TIS benefit: Patients receiving continuous efgartigimod maintained clinically meaningful improvement through 52 weeks, with a mean TIS of 52.19. Patients who switched from placebo to efgartigimod achieved comparable improvement, with a mean TIS of 49.62 at week 52. The safety pro consistent over the course of the study, with no increase in adverse events with longer exposure. These results further support the mechanistic relevance of FcRn blockade in myositis, providing further evidence that pathogenic autoantibodies are the underlying driver of disease. “Patients with autoimmune myositis face the serious challenge that their immune system attacks their own body, causing irreversible muscle loss, weakness, pain and reduced quality of life, while the limited available treatments can carry significant side effects and be difficult to tolerate long term,” said Hector Chinoy, M.D., Ph.D., presenting study author and Professor of Rheumatology and Neuromuscular Disease at The University of Manchester. “Emerging results from the ALKIVIA+ study suggest that efgartigimod has the potential to deliver meaningful and sustained clinical benefit for patients with myositis, with a favorable tolerability profile.” Topline results from the Phase 3 ALKIVIA study are expected in the third quarter of 2026. Efgartigimod achieved maintenance of response in Sjogren’s disease RHO+, a 48-week open-label extension study, evaluated patients with Sjogren's disease who continued to receive efgartigimod, and those who transitioned from placebo to efgartigimod after completing the 24-week double-blind treatment period of the Phase 2 RHO study. Response to efgartigimod was assessed using the Composite of Relevant Endpoints for Sjogren’s Syndrome (CRESS), a composite endpoint designed to capture overall treatment benefit across multiple clinical domains, including disease activity as measured by ClinESSDAI. Patients in the efgartigimod arm were switched to biweekly dosing based on ClinESSDAI response. Results showed: Patients in the efgartigimod arm switching to biweekly dosing experienced maintenance of response on clinical measures. Patients in the placebo arm switching to efgartigimod experienced improvements in ClinESSDAI and increased CRESS response. At week 72, median ClinESSDAI scores were low in both groups: 2.5 in the efgartigimod arm and 2.0 in patients who transitioned from placebo to efgartigimod. Low disease activity is indicated by a ClinESSDAI score of <5. Efgartigimod was well tolerated, with no new safety signals identified following long-term use in participants with Sjogren’s disease. The Phase 3 UNITY trial is currently ongoing to assess efficacy and safety of efgartigimod in patients with moderate to severe Sjogren’s disease. Topline results are expected in the second half of 2027. Efgartigimod safety pro across autoantibody-driven autoimmune diseases A cross-indication safety analysis of efgartigimod spanning multiple global clinical studies, including autoimmune rheumatic diseases, indicated that efgartigimod is consistently well-tolerated across diseases and administration methods. Data showed a consistent safety pro 834 treated patients and more than 1,300 patient-years of follow-up. Efgartigimod was well tolerated in participants with autoimmune rheumatic diseases (myositis, Sjogren's disease, and lupus nephritis), consistent with findings from prior studies in other IgG-mediated autoimmune diseases and also consistent with what has been observed in approved indications globally. Adverse events were predominantly mild-to-moderate, and no increased event rates were seen in studies with a longer treatment duration. Deepening understanding of the underlying biology Additional argenx activities include: the presentation of the Phase 2 eSScape study design, evaluating the potential of efgartigimod in Systemic Sclerosis (SSc); an FcRn symposium exploring the role of autoantibodies in autoimmune rheumatic diseases and highlighting the potential of targeting FcRn; and the launch of a new myositis disease education campaign. argenx additional information on the data at EULAR 2026 can be found here . Details for argenx presentations are as follows: Title Lead Author Presentation Myositis The Long-Term Safety and Efficacy of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy: Results From the Phase 2 Randomized ALKIVIA and Open-Label Extension ALKIVIA+ Trials Rohit Aggarwal Poster #POS0261 Friday, June 5 13:36 BST Sjogren’s Disease Long-Term Efficacy and Safety of Efgartigimod in Sjogren’s Disease: Findings from the RHO+ Open-Label Extension Study Isabelle Peene Oral Presentation #130 Wednesday, June 3 17:30-17:40 BST Systemic Sclerosis Efgartigimod PH20 Subcutaneous in Adults With Systemic Sclerosis: Design of a Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study (eSScape) Dinesh Khanna Poster #POS0326 Friday, June 5 16:30 BST Multiple Disease Areas Safety Pro Efgartigimod Across Multiple Global Clinical Trials in Immunoglobulin G-Mediated Autoimmune Diseases With Emerging Data in Idiopathic Inflammatory Myopathy, Sjogren’s Disease, and Lupus Nephritis Hector Chinoy Poster #POS1200 Saturday, June 6 10:15 BST About Efgartigimod and Efgartigimod SC Efgartigimod (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. Efgartigimod 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. About Myositis Myositis is an autoimmune rheumatic disease that can be progressive and may lead to significant muscle and organ involvement, resulting in functional impairment and substantial burden for patients. It is characterized by skeletal muscle weakness with possible extramuscular manifestations involving the skin, joints, lungs, gastrointestinal tract, and heart. Major subtypes include immune-mediated necrotizing myopathy (IMNM), dermatomyositis (DM), polymyositis (PM), antisynthetase (ASyS), and inclusion body myositis, with muscle involvement being a common manifestation across most subtypes. About ALKIVIA and ALKIVIA+ Study Design The ALKIVIA study is a randomized, double-blind, placebo-controlled, multicenter, operationally seamless Phase 2/3 study of efgartigimod SC for the treatment of autoimmune myositis across IMNM, DM and PM. The ALKIVIA study enrolled 240 patients in total and is being conducted in two phases, with an analysis of the Phase 2 portion of the clinical trial after the first 90 patients completed the study, followed by a Phase 3 portion if a signal is observed in the Phase 2 portion. The primary endpoint is the mean total improvement score (TIS) at the end of the treatment period (24 weeks in Phase 2 and 52 weeks in Phase 3) of all treated patients (IMNM, DM and PM) compared to placebo. Key secondary endpoints include response rates at the end of treatment, time to response, and duration of response in TIS, as well as change from baseline in individual TIS components. Other secondary endpoints include quality of life and other functional scores. ALKIVIA+ is a 28-week open-label extension study that evaluated myositis patients who received continuous efgartigimod treatment and those who transitioned from placebo to efgartigimod after completing the 24-week double-blind, placebo-controlled Phase 2 ALKIVIA study. The primary endpoint of ALKIVIA+ is long-term safety and tolerability. About Sjogren’s Disease Sjogren’s disease is a chronic and progressive, systemic, autoimmune rheumatic disease associated with elevated autoantibodies and characterized by immune-mediated dysfunction of exocrine glands and extraglandular organ manifestations. Sjogren's disease can be severely debilitating and have a negative impact on patient quality of life, with common symptoms reported as dry eyes and mouth, fatigue, and joint pain. Sjogren's disease predominantly impacts women with a 9:1 female:male incidence ratio. Given the heterogeneous nature of the disease, the treatment journey can be challenging with long delays and high rates of misdiagnosis. There are no FDA-approved treatments targeting the disease itself, leaving current treatments to focus primarily on individual symptom management. About RHO and RHO+ Study Design The Phase 2 RHO study was a randomized, double-blinded, placebo-controlled multicenter proof-of-concept study to evaluate the safety and efficacy of efgartigimod in adults with Sjogren’s disease. In order to enter the study, patients needed to test positive for anti-Ro autoantibodies and maintain residual salivary flow. Thirty-four patients were randomized 2:1 to receive either efgartigimod or placebo for up to 24 weeks. Multiple endpoints and biomarkers were evaluated in the signal-finding study, including the primary endpoint of CRESS (Composite of Relevant Endpoints for Sjogren’s Syndrome). Within CRESS there are five components, spanning: systemic disease activity as measured by the ESSDAI (EULAR Sjogren’s Syndrome Activity Index), patient reported outcomes as measured by the ESSPRI (EULAR Sjogren’s Syndrome Patient Reported Index), tear and salivary gland function, and serology. To be a CRESS responder, patients needed to demonstrate a clinically meaningful benefit in at least 3 of the 5 composite items. Additional datapoints were gathered including the ClinESSDAI, STAR (Sjogren’s Tool for Assessing Response), biomarker data, and the change in lymphocytic infiltrate levels through parotid biopsies. RHO+ was a 48-week open-label extension study that evaluated the safety of efgartigimod in adult patients with Sjogren's disease who continued to receive efgartigimod IV, and those who transitioned from placebo to efgartigimod IV after completing the 24-week double-blind treatment period of the Phase 2 RHO study. The primary endpoint of RHO+ was incidence and severity of AEs and AESIs, incidence of SAEs, changes in laboratory test results, vital signs, and ECG results. About UNITY Study Design UNITY is an ongoing Phase 3 randomized, double-blinded, placebo-controlled multicenter study with open-label extension to evaluate the efficacy, safety, and tolerability of subcutaneously administered efgartigimod PH20, in adult participants with moderate-to-severe primary Sjogren’s disease. About argenx argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker and is evaluating its broad potential in multiple serious autoimmune diseases while advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit and follow us on LinkedIn , Instagram , Facebook , and YouTube . For further information, please contact: Media: Kate Dion kdion@argenx.com Colin McBean cmcbean@argenx.com Investors: Alexandra Roy aroy@argenx.com Forward-looking Statements The contents of this announcement include statements that are, or may be deemed to be, “forward-looking statements.” These forward-looking statements generally can be identified by the use of forward-looking words, such as “aim”, “anticipate”, “aspire”, “believe”, “can”, “continue”, “could”, “estimate”, “expect”, “entail”, “forecast”, “future”, “goals”, “hope”, “intend”, “is designed to”, “likely”, “may”, “might”, “objective”, “plan”, “possible”, “potential”, “pursue”, “project”, “predict”, “seek”, “should”, “strategy”, “target”, “will” and other words and terms of similar meaning and expression, including in connection with any discussion of future operating or financial performance. 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; uncertainties associated with the development of novel drug therapies; preclinical and clinical trial and product development risks and setbacks; interpretation of argenx’s clinical trial data by regulatory authorities and argenx’s ability to obtain regulatory approval; the risk that early stage clinical trials may not be predictive of results in later stage or large scale clinical trials; the occurrence of adverse safety events or participant dropouts; 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; the impact of healthcare regulations, including rules on reimbursement for argenx’s products; competition in drug discovery, development and commercialization efforts; its reliance on third-party suppliers, service providers and manufacturers; and instability and conflicts in the regions in which the company has suppliers or markets for its products. A further list and description of these and other risks, uncertainties, and factors that could cause actual results to differ materially from those referred to in the forward-looking statements 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 risks and uncertainties, the reader is advised not to place undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this press release. 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.

2026年5月22日，再鼎医药（09688）发布公告称，公司总裁兼首席运营官Josh Smiley先生将不再担任相关职务，该人事变动自2026年5月18日起正式生效，其最后任职日期为2026年5月22日。　　公告显示，Josh Smiley此前分管的相关工作职责，已由公司董事会主席、创始人兼首席执行官杜莹博士及其他管理团队成员承接。Smiley先生将在签署常规解除协议后，依据其现有雇佣协议，获得相应遣散补偿。 　　Josh Smiley入职再鼎医药已满四年，是一位拥有27年生物制药行业从业经验的资深高管，入职再鼎医药前，曾担任礼来高级副总裁兼首席财务官。　　值得注意的是，此次人事变动发生在公司2025年年度绩效评估完成后不久。2025年，Josh Smiley个人绩效系数为90%，当年公司管线研发、营收增长、盈利表现三项核心经营目标均未能完成。年度薪酬相应下调，后续其任职被终止。　　公司2025年营收4.57亿美元，同比增15%；整体尚未实现盈利，净亏损收窄至1.76亿美元。　　因绩效落差“被辞退”？　　美国证监会（SEC）官网披露信息显示，再鼎医药于2026年4月17日公示了2026年度股东投票代理声明（PRE 14A）。该声明详细披露了公司年度股东大会安排、财务业绩、董事会成员构成、高管薪酬体系及股东提案等多项核心信息。　　《财中社》梳理相关绩效薪酬评价信息后发现，Josh Smiley是公司五位高管中，唯一个人绩效评估结果未达100%、整体薪酬较上年小调的高管，其个人绩效表现的下滑与此次离职或存在潜在关联。　　再鼎医药的薪酬体系中，高管的激励奖励薪酬（Annual Incentive Award）发放与个人及公司绩效评估结果直接挂钩。其中，CEO杜莹的奖金完全依据公司整体绩效核算，不设置个人绩效权重；其余高管的奖金则按照80%公司绩效、20%个人绩效的比例进行核算分配。　　根据公司披露，公司绩效乘数（Corporate Performance Multiplier）从2024年的103%降至2025年的85%，这一变动直接影响全体高管的奖金发放，本年度高管奖金支付比例相应下调至85%-93%。　　在个人绩效评估维度，Josh Smiley的个人绩效乘数（Individual Performance Multiplier）为90%，为公司高管中最低水平，同时也是所有高管里唯一低于100%的人员。据薪酬委员会核定，2025绩效年度内，除CEO以外的其他高管（NEO/Named Excecutive Officer）可分配的个人绩效乘数区间为90%至125%。 （来源：公司2026 PRE 14A）　　根据披露，公司在2025年的绩效衡量标准包含三项核心关键目标，分别为管线推进和战略举措、收入增长、盈利能力，三项目标在公司绩效奖励中的权重占比分别为40%、30%、30%。2024年，公司设定了五项核心关键目标，整体均达标。　　而2025年，公司上述三大核心绩效目标均未完成。 　　在管线推进和战略举措方面，公司实际得分为37%，未达到40%的目标。2025年公司虽然推进了多项关键管线项目，但薪酬委员会严厉指出“部分产品、相关研究及项目里程碑被调低优先级、终止或延期，同时公司主动终止了部分临床试验与合作项目布局”。　　在收入增长方面，公司实际得分为24%，低于30%的设定目标。公司2025年实现营收4.57亿美元，较2024年的3.98亿美元同比增长15%，未及设定的预期目标。　　在盈利能力维度，公司实际得分为24%，同样低于30%的目标。薪酬委员会评估认为，2025年公司虽实现运营费用营收占比下降、净亏损同比缩减32%的改善，但整体盈利进展未达到委员会认定的盈利推进预期（“progress toward profitability”）。　　让股东失望的是，再鼎医药迟迟未能扭亏为盈。财报数据显示，公司净亏损从2024年的2.57亿美元亏损降至2025年的1.76亿美元。　　值得关注的是，2025年再鼎医药其余高管的整体薪酬均较上年上涨，唯有Josh Smiley薪酬显著缩水，充分体现公司对其年度履职绩效的负面评价。　　2025年，Josh Smiley总薪酬为470万美元，其中包含67.28万美元基本工资、34.71万美元非股权激励奖金及其他股权激励薪酬，整体薪酬规模较2024年降幅达33%。　　身为公司总裁兼首席运营官，Josh Smiley承担者营收增长与盈利能力建设，两大核心经营指标双双未达考核预期，便顺理成章成为其个人绩效评级下滑、薪酬大幅下调并最终“被遣散”的核心原因。 （来源：公司2026 PRE 14A）　　2025年整体绩效未达标，公司组织与人力成本的系统性优化同时也反映在公司总雇员人数和薪酬成本的微降上。　　据官方年报披露，2024年、2025年雇员人数为1844、1797；2024年、2025年公司总薪酬成本分别为2.81亿美元、2.53亿美元， （来源：公司2024年、2025年年报）　　商业化产品“青黄不接”　　长期以来，“License-in（授权引进）”模式是再鼎医药的核心标签。该模式通过外部引进成熟在研药物，能够有效压缩新药研发周期，规避药物早期研发的失败风险与不确定性，是药企发展前期快速实现产品商业化的策略。　　2016年9月，再鼎医药与Tesaro（现为GSK附属公司）达成合作，拿下尼拉帕利（则乐®）PARP抑制剂在中国大陆、香港、澳门地区的独家开发与商业化权益，成为国内药企License-in模式的经典案例。　　目前，再鼎医药已拥有八款商业化产品。2025年，公司核心产品则乐®、卫伟迦/卫力迦®营收增速疲软，两款产品分别实现收入1.89亿美元、9419.8万美元，同比增速仅为1%，合计占据公司整体营收略超六成。与之形成对比的是，公司另外两款产品鼎优乐®、奥凯乐®实现快速放量，同比增速分别达593%、409%。 （来源：公司2025年年报）　　现阶段，公司已形成“License-in+自研”的双轮驱动发展模式。除此之外，公司的对外合作模式持续升级，不再局限于单一的License-in授权引进，同时布局股权参与、联合开发、联合商业化等多元合作模式，合作对象涵盖GSK、BMS、辉瑞等大型跨国药企，以及argenx、Deciphera、宜联生物等专业生物技术公司。　　ZL-1310（Zocilurtatug Pelitecan）是再鼎医药研发管线中具备核心竞争力的重点品种，也是公司License-in管线的核心产品之一，为一款靶向DLL3的抗体药物偶联物（ADC），拥有极高的临床应用价值与商业化潜力。　　2023年4月，再鼎医药与宜联生物达成全球独家许可合作协议，买断ZL-1310项目的全球开发、生产及商业化全部权益。　　目前全球范围内获批上市的DLL3靶向药物仅有安进研发的Tarlatamab一款，该药物获批用于二线及以上小细胞肺癌的治疗。　　ZL-1310是当前全球唯一进入III期注册临床阶段的DLL3 ADC药物，赛道进度领先优势显著。国内同类竞品中，恒瑞医药联合IDEAYA Biosciences研发的同靶点药物SHR-4849目前仅处于中美I/II期临床研发阶段。　　2025年10月，ZL-1310正式启动全球III期临床试验，预计2028年11月完成整体试验。ZL-1310有望成为继Tarlatamab之后，小细胞肺癌治疗领域的新一代重磅产品。　　然而，需要清醒认识到的是，在现有核心产品增长乏力、下一代重磅产品尚未落地的“空窗期”，再鼎医药的经营发展正面临严峻考验。　　尽管再鼎医药公司整体绩效不及预期，公司并未收缩核心管线的临床研究开发推进，2025年其临床试验相关的CRO、CMO、研究者开支增加了6%。　　核心挑战在于，再鼎医药该如何依托成本管控与核心管线攻坚，平稳度过产品“空窗期”。（文章来源：财中社）