Immune-mediated Thrombotic thrombocytopenic purpura (iTTP) is a rare, autoimmune disorder characterized by life-threatening episodes of thrombocytopenia, microangiopathic hemolytic anemia and organ damage. Patients have an unpredictable course punctuated by relapses associated with autoantibody-mediated (primarily IgG) depletion of ADAMTS13, a key regulator of coagulation. ADAMTS13 deficiency during remission has been associated with increased risk of relapse, but also, and potentially more devastating, ischemic stroke.
Until recently, it was presumed that rituximab (a monoclonal antibody targeting B cells) improved relapse-free survival in most patients, but this was based on findings from very small studies. Given concern about stroke and relapse risk, preventive immunosuppression with rituximab has also recently come into practice for patients with falling ADAMTS13 activity (ADAMTS13-relapse). It is expected that following efgartigimod therapy, there will be a rise in ADAMTS13 activity to the normal range that will be sustained during the treatment period. Following withdrawal of therapy, it is expected that most participants will experience a fall in ADAMTS13 activity, demonstrating the safety and efficacy in efgartigimod to reliably but temporarily reduce pathogenic antibodies. This would demonstrate the potential efficacy for efgartigimod as a maintenance therapy to safely prevent relapse of iTTP to be further explored in a larger efficacy study.

开始日期2025-05-01

申办/合作机构

University of Minnesota

NCT06885762

/ Not yet recruiting临床2/3期IIT

Safety and Efficacy of Efgartigimod in Guillain-Barré Syndrome

The goal of this clinical trial is to evaluating the efficacy and safety of efgartigimod in the treatment of Guillain-Barré Syndrome and exploring the immunological mechanisms of efgartigimod therapy in Guillain-Barré Syndrome. The main questions it aims to answer are:
Will efgartigimod improve the symptoms of participants? What medical problems do participants have when using efgartigimod?
Participants will:
On day 1 and day 5 of the treatment period, drug A was administered intravenously.
Keep a diary of their symptoms

开始日期2025-04-13

申办/合作机构

新桥医院

NCT06909214

/ Not yet recruiting临床4期

A Phase 4, Open-Label, Prospective, Single-Group, Multicenter Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adult Participants With New-Onset Generalized Myasthenia Gravis

The main purpose of this study is to measure how well adults with new-onset gMG (which means they've had generalized disease signs and/or symptoms for less than 1 year) respond to treatment with efgartigimod PH20 SC. The study consists of a treatment period of 51 weeks. The study duration for each participant will be approximately 58 weeks.

开始日期2025-04-01

申办/合作机构

argenx SE

100 项与艾加莫德α 相关的临床结果

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100 项与艾加莫德α 相关的转化医学

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100 项与艾加莫德α 相关的专利（医药）

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189

项与艾加莫德α 相关的文献（医药）

2025-12-31·Human Vaccines & Immunotherapeutics

Impact of FcRn antagonism on vaccine-induced protective immune responses against viral challenge in COVID-19 and influenza mouse vaccination models

Article

作者: Vanhoenacker, Peter ; Baumeister, Judith ; Wouters, Heidi ; Schotsaert, Michael ; Laghlali, Gabriel ; Ulrichts, Peter ; Singh, Gagandeep ; Chang, Lauren A ; Binazon, Ornella ; Warang, Prajakta ; Augustynková, Kateřina ; Elhemdaoui, Nadia ; Moshir, Mahan ; Steeland, Sophie ; Notebaert, Margo

Antagonism of the neonatal Fc receptor through an engineered antibody Fc fragment, such as efgartigimod, results in a decrease in immunoglobulin G levels. This approach is being evaluated as a therapeutic strategy for the treatment of IgG-mediated autoimmune diseases. Our goal was to evaluate the impact of mFc-ABDEG, a mouse-adapted antibody Fc fragment with a mode of action highly similar to efgartigimod, on vaccine-induced protective immune responses against viral infections. Therefore, mouse vaccination models for COVID-19 and influenza were employed, utilizing an mRNA COVID-19 vaccine (COMIRNATY) and an adjuvanted, inactivated quadrivalent influenza vaccine (Seqirus+AddaVax), respectively. In both models, vaccination induced robust humoral responses. As expected, animals treated with mFc-ABDEG had lower levels of virus-specific IgG, while virus-specific IgM responses remained unaffected. The COVID-19 vaccine induced a strong Th1-type T cell response irrespective of mFc-ABDEG treatment. Influenza vaccination resulted in a poor T cell induction, regardless of mFc-ABDEG treatment, due to the Th2-biased response that inactivated influenza vaccines typically induce. Importantly, mFc-ABDEG treatment had no effect on protective immunity against live viral challenges in both models. Vaccinated animals treated with mFc-ABDEG were equally protected as the non-treated vaccinated controls. These non-clinical data demonstrate that FcRn antagonism with mFc-ABDEG did not affect the generation of vaccine-induced protective humoral and cellular responses, or protection against viral challenges. These data substantiate the clinical observations that, although IgG titers were reduced, FcRn antagonism with efgartigimod did not impair the ability to generate new specific IgG responses, regardless of the timing of vaccination.

2025-07-01·CLINICAL NEUROLOGY AND NEUROSURGERY

Efficacy and safety of FcRn inhibitors in patients with Myasthenia gravis: An updated systematic review and meta‑analysis

Review

作者: Farooqi, Hanzala Ahmed ; Muzammil, Anam ; Hassan, Syed Muhammad ; Hanif, Ummara ; Akhtar, Muzamil ; Maryam, Ayesha ; Khan, Zarak ; Athar, Zoya ; Akhtar, Mehmood

BACKGROUND:

Myasthenia gravis (MG) is a chronic, complex autoimmune disorder characterized by the production of autoantibodies that destroy neuromuscular junctions. Blocking the neonatal Fc receptors (FcRn) enhances IgG catabolism, offering a novel therapeutic approach.

METHODS:

PubMed, Embase, and the Cochrane Library were searched up to February 2025, for RCTs evaluating FcRn inhibitors in MG. A random effects model to calculate pooled risk ratios (RR) and mean differences with 95 % confidence intervals (CI).

RESULTS:

873 patients from 8 randomized control trials (RCTs) were analyzed. Compared to placebo, FcRn inhibitors significantly reduced Myasthenia Gravis Activities of Daily Living (MG-ADL) scores (MD of -1.45 [95 % CI, -1.91 to -0.99]; P < 0.00001), Quantitative Myasthenia Gravis( QMG) scores (MD = -2.33 [95 % CI, -3.57 to -1.09]; P = 0.0002), and Myasthenia Gravis Composite (MGC) scores (MD = -2.96 [95 % CI, -4.29 to -1.63]; P < 0.0001). The FcRn inhibitors improved MG-ADL responder rates (RR = 1.60 [95 % CI, 1.27-2.02]; P < 0.0001), and Myasthenia Gravis Quality of Life (MGQoL15r) scores (MD = -2.18 [95 % CI, -3.35 to -1.00]; P = 0.0003). Serious adverse events were lower with the FcRn inhibitors (32/519) than the placebo (39/397). Subgroup analysis revealed that Rozanolixizumab and Nipocalimab improved MG-ADL scores, but had inferior responder rates. Additionally, Rozanolixizumab significantly improved MGC scores but had more adverse events.

CONCLUSION:

FcRn inhibitors demonstrated good efficacy and safety in MG, with efgartigimod and nipocalimab showing strong efficacy without added risk. Further research is required to evaluate long-term outcomes and optimize treatment.

2025-07-01·MEDICINA CLINICA

Efgartigimod for the treatment of Guillain–Barré syndrome with concurrent posterior reversible encephalopathy syndrome

Letter

作者: Tao, Weiwei ; Liang, Hui ; Hong, Weijun

659

项与艾加莫德α 相关的新闻（医药）

2025-05-22

·PRNewswire

Johnson & Johnson's IMAAVY Gets FDA Approval, Taking on Top Players in Myasthenia Gravis Market | DelveInsight

The FDA has approved Johnson & Johnson's anti-FcRn antibody, nipocalimab, for the treatment of generalized myasthenia gravis. The drug will be sold under the brand name IMAAVY. This approval positions J&J to challenge leading competitors in the myasthenia gravis market, such as AstraZeneca, which markets the monoclonal antibodies SOLIRIS and ULTOMIRIS. J&J will also be competing with Argenx's VYVGART and UCB's RYSTIGGO and ZILBRYSQ. LAS VEGAS, May 22, 2025 /PRNewswire/ -- Myasthenia gravis is an autoimmune disorder in which antibodies disrupt the communication between nerves and muscles, resulting in skeletal muscle weakness. This weakness primarily affects voluntary muscles that control eye movement, facial expression, swallowing, and limb function. The condition is predominantly driven by autoantibodies targeting either the acetylcholine receptor (AChR-Ab) or a receptor-associated protein known as muscle-specific tyrosine kinase (MuSK-Ab). According to DelveInsight's analysis, there were approximately 300,000 diagnosed prevalent cases of myasthenia gravis across the 7MM in 2024. This number is expected to grow steadily over the forecast period (2025–2034), with a projected CAGR. The current myasthenia gravis therapeutics market mainly consists of symptomatic treatments such as acetylcholinesterase inhibitors like Mestinon. However, achieving full clinical remission with symptomatic therapy alone is challenging. As a result, immunosuppressive drugs are frequently introduced. First-line immunotherapy typically involves high-dose oral corticosteroids such as prednisone or prednisolone, which can induce a rapid response within 2 to 4 weeks. To reduce reliance on steroids and improve long-term disease control, nonsteroidal immunosuppressants (NSISTs) are often used. These include antimetabolites (e.g., azathioprine, mycophenolate mofetil, methotrexate, and cyclophosphamide) and calcineurin inhibitors (e.g., tacrolimus and cyclosporine). While NSISTs may be used as standalone treatments, they usually take 6 to 9 months to produce noticeable effects. Immunomodulatory therapies, including intravenous immunoglobulin (IVIg) and plasmapheresis (PLEX), are typically reserved for acute exacerbations or myasthenic crises. They are also employed as chronic treatments in patients who cannot tolerate or do not respond to standard immunosuppressants. Although traditional immunosuppressive strategies can help manage symptoms, achieving long-term, stable remission remains difficult. Additionally, about 15% of patients are considered treatment-refractory, meaning they either fail to respond to or cannot tolerate multiple immunosuppressive options. These patients often face the dual challenge of persistent symptoms and the adverse effects of long-term immunosuppression. Learn more about the myasthenia gravis disease market @ Myasthenia Gravis Treatment Medications The myasthenia gravis treatment market is well-established, with multiple approved drugs for myasthenia gravis, including SOLIRIS, ULTOMIRIS, RYSTIGGO, ZILBRYSQ, and VYVGART, among others. Alexion Pharmaceuticals' SOLIRIS is a monoclonal antibody that targets and inhibits complement protein C5, thereby blocking the formation of the terminal complement complex. This action helps reduce complement-driven hemolysis in PNH and thrombotic microangiopathy in aHUS. Although its exact mechanism in gMG and NMOSD is not fully known, it is believed to decrease complement complex accumulation. Between 2017 and 2023, SOLIRIS gained regulatory approvals in Japan, the US, and the EU for treating gMG, including pediatric indications in Japan. ULTOMIRIS (ravulizumab), also from Alexion Pharmaceuticals, is the first long-acting C5 inhibitor, offering rapid, complete, and sustained suppression of complement activity. It binds to C5, preventing its cleavage and subsequent formation of the membrane attack complex (C5b-9). ULTOMIRIS is effective in managing terminal complement-mediated hemolysis in PNH and thrombotic microangiopathy in aHUS. Although the precise mechanism in gMG and NMOSD remains unclear, it is thought to reduce complement deposition at the neuromuscular junction and block aquaporin-4 antibody-mediated complement activation. It received approvals from the US FDA, EMA, and Japan's PMDA in 2022. Get an in-depth assessment on generalized myasthenia gravis medication for adults @ ULTOMIRIS vs SOLIRIS for Myasthenia Gravis UCB Biopharma's RYSTIGGO is a subcutaneously administered, humanized monoclonal antibody that binds with high affinity to the human neonatal Fc receptor (FcRn). It received regulatory approval from the US FDA and Japan's PMDA in 2023, followed by the EC in January 2024. ZILBRYSQ, also developed by UCB Biopharma, is the first once-daily subcutaneous peptide-based inhibitor specifically targeting complement protein C5. It works by blocking C5 cleavage and the formation of the terminal complement complex C5b-9. Although its precise action in gMG remains uncertain, it is thought to limit C5b-9 accumulation at the neuromuscular junction. ZILBRYSQ gained approval from the US FDA, EMA, and PMDA in 2023. VYVGART, from Argenx, is a human IgG1 antibody fragment that targets FcRn, leading to a decrease in circulating IgG autoantibodies. It is the first FcRn blocker to receive regulatory approval. Argenx's VYVGART HYTRULO (also marketed as VYVDURA) combines efgartigimod alfa, an FcRn inhibitor, with hyaluronidase, an endoglycosidase, for treating adults with generalized myasthenia gravis. This formulation was approved by the US FDA and EMA in 2023 and by the PMDA in January 2024. Uncover the mechanism of FcRn inhibitor myasthenia gravis medication @ Myasthenia Gravis MoA On April 30, 2025, the FDA approved Johnson & Johnson's nipocalimab, branded as IMAAVY, as a new therapy for gMG. This approval puts IMAAVY among the list of approved drugs for myasthenia gravis treatment. The approval, which J&J says encompasses the widest eligible patient population with gMG, includes individuals aged 12 and above who test positive for AChR or anti-muscle-specific kinase (MuSK) antibodies. IMAAVY functions by inhibiting the FcRn protein, which plays a role in maintaining circulating IgG antibodies, key drivers in many autoimmune disorders. By blocking FcRn, IMAAVY significantly reduces harmful IgG autoantibodies while preserving the body's overall immune response, according to clinical data. J&J sees broader potential for IMAAVY beyond gMG, believing its mechanism of action could apply across multiple autoimmune conditions due to IgG's involvement in various diseases. The FDA's decision followed results from the ongoing Phase III Vivacity-MG3 trial, which evaluates IMAAVY in combination with standard care versus placebo and standard care in 199 adult gMG patients, including 153 antibody-positive individuals. Additionally, J&J is conducting a Phase II/III pediatric trial called Vibrance, where IMAAVY, combined with standard care, achieved its primary goal by reducing total serum IgG levels by 69% over 24 weeks. J&J acquired IMAAVY through its USD 6.5 billion acquisition of Momenta Pharmaceuticals in 2020, with the FcRn inhibitor myasthenia gravis serving as the centerpiece of that deal. The company is aiming for blockbuster status, forecasting peak annual sales of over USD 5 billion for the myasthenia gravis medication. Dive deep into the myasthenia gravis treatment cost @ Generalized Myasthenia Gravis Drug with Copay Myasthenia gravis pipeline possesses some drugs in mid- and late-stage development to be approved in the near future. The expected launch of myasthenia gravis treatment medications, such as Batoclimab (Immunovant Sciences GmbH), Descartes-08 (Cartesian Therapeutics), and others, are expected to further create a positive impact on the myasthenia gravis disease market. Discover which therapies are expected to grab major myasthenia gravis therapeutics market share @ Market Analysis on Myasthenia Gravis Batoclimab is a fully humanized monoclonal antibody designed to inhibit neonatal Fc receptors by blocking the interaction between FcRn and IgG, thereby promoting the breakdown of autoantibodies. It has received Orphan Drug Designation (ODD) from both the US FDA and the EMA. In March 2025, Immunovant announced topline results from its Phase III trial evaluating batoclimab in patients with myasthenia gravis. Descartes-08 represents the first RNA-engineered CAR T-cell (rCAR-T) therapy developed for autoimmune conditions. This therapy uses cytotoxic T-cells programmed to target and eliminate pathogenic plasma cells that produce autoantibodies. Created from a patient's own blood, Descartes-08 has also received ODD from the US FDA for treating myasthenia gravis. In December 2024, Cartesian Therapeutics shared encouraging updated findings from its Phase IIb study of Descartes-08 in myasthenia gravis. Furthermore, in January 2025, the FDA agreed to the company's Phase III AURORA trial design under a Special Protocol Assessment, with trial initiation planned for the first half of 2025. Curious about what the new myasthenia gravis treatments are in development? Visit Myasthenia Gravis Clinical Trials The anticipated launch of these emerging myasthenia gravis treatments are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the myasthenia gravis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the myasthenia gravis market size in the 7MM is expected to grow from USD 5.2 billion in 2024 at a significant CAGR by 2034. According to DelveInsight's analysis, the myasthenia gravis market growth is anticipated with the introduction of emerging therapies, leading to an expansion in the myasthenia gravis market size throughout the forecast period (2025–2034). This anticipated myasthenia gravis market growth is driven by advancements in treatment options, greater healthcare access, and a rising myasthenia gravis prevalence, which together foster higher demand for innovative and effective therapies. DelveInsight's latest published market report titled Myasthenia Gravis Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which myasthenia gravis treatment market leader is going to capture the largest market share. The report provides comprehensive insights into the country-specific myasthenia gravis treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The myasthenia gravis market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Diagnosed Prevalent Cases of Myasthenia Gravis Gender-specific Diagnosed Prevalent Cases of Myasthenia Gravis Age-specific Diagnosed Prevalent Cases of Myasthenia Gravis Diagnosed Prevalent Cases of Myasthenia Gravis by MGFA Classification Diagnosed Prevalent Cases of Generalized Myasthenia Gravis by Antibody Serology The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM myasthenia gravis market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this myasthenia gravis market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the myasthenia gravis market. Also, stay abreast of the mitigating factors to improve your market position in the myasthenia gravis therapeutic space. Related Reports Myasthenia Gravis Epidemiology Forecast Myasthenia Gravis Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted myasthenia gravis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Myasthenia Gravis Pipeline Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myasthenia gravis companies, including Harbour BioMed (Guangzhou) Co. Ltd., Kyverna Therapeutics, Cabaletta Bio, Takeda, Hoffmann-La Roche, Immunovant Sciences GmbH, Regeneron Pharmaceuticals, Novartis Pharmaceuticals, Janssen Research & Development, LLC, Momenta Pharmaceuticals, Inc., Amgen, Dianthus Therapeutics, Cartesian Therapeutics, COUR Pharmaceutical Development Company, Inc., Alexion Pharmaceuticals, Inc., among others. Generalized Myasthenia Gravis Market Generalized Myasthenia Gravis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key gMG companies including Alexion Pharmaceuticals, Argenx, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Bristol Myers Squibb, Biogen Inc., Pfizer Inc., Novartis AG, Sanofi S.A., Roche Holding AG, Johnson & Johnson, Takeda Pharmaceutical Company Limited, AbbVie Inc., Eli Lilly and Company, Merck & Co., Inc., GlaxoSmithKline plc, AstraZeneca plc, Amgen Inc., Boehringer Ingelheim International GmbH, Genentech, Inc., Gilead Sciences, Inc., Celgene Corporation, Vertex Pharmaceuticals Incorporated , among others. Generalized Myasthenia Gravis Pipeline Generalized Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key gMG companies, including Biocon, Cartesian Therapeutics, UCB, Momenta Pharmaceuticals, HanAll Biopharma, Roche, Alexion, Novartis, Takeda, BioMarin, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

免疫疗法临床3期上市批准孤儿药临床结果

2025-05-22

·生物制品圈

强生亮出底牌

强生频繁亮出新王牌，按下了自免王座争夺战的加速键。4月30日，强生的FcRn阻断剂Nipocalimab（尼卡利单抗）获FDA批准上市，用于治疗全身型重症肌无力（gMG）患者，将成为强生争夺“自免一哥”宝座的利器。毕竟，尼卡利单抗是强生以65亿美元收购Momenta而获得，商业价值可见一斑。而且，Argenx/再鼎医药的艾加莫德α（Efgartigimod），2025年第一季度实现销售额7.9亿美元，同比大幅增长99%，充分验证了FcRn赛道潜力。更何况，强生在自免领域不只有这一张王牌。近期，强生在皮肤病研究学会（SID）年会上公布了口服IL-23R多肽拮抗剂icotrokinra（JNJ-2113）的最新III期临床试验数据，研究结果令人振奋，有望为银屑病患者提供一种创新的口服治疗方案。显然，“自免一哥”这个名号，强生是志在必得。TONACEA01新王牌登场艾加莫德用近乎翻倍的增速，给FcRn靶向药物带来了巨大的想象空间。艾加莫德是全球首款获批的FcRn拮抗剂，目前已斩获3项适应症，包括全身型重症肌无力（gMG）、原发性免疫性血小板减少症（ITP）、慢性炎症性脱髓鞘性多发性神经病（CIDP）。从销售表现看，艾加莫德保持着强劲的销售态势：2023年跻身“重磅炸弹”药物行列，2024年全球销售额同比增长83%至22亿美元，今年一季度同比增长99%至7.9亿美元。此外，Argenx还在针对艾加莫德拓展更广泛的适应症，目前正在15种严重自身免疫性疾病中接受评估，包括持续性多发性硬化症、特发性血小板减少性紫癜等。根据ODDO BHF分析师预测，艾加莫德将在2035年达到110亿美元的销售峰值。另一款FcRn拮抗剂——优时比的罗泽利昔珠单抗（Rozanolixizumab），在2023年6月获FDA批准上市，用于抗乙酰胆碱受体（AChR）或抗肌肉特异性酪氨酸激酶（MuSK）抗体阳性的gMG患者，也取得了不错的商业化表现：销售额从2023年的1900万欧元快速飙升至2024年的2.02亿欧元（约2.27亿美元）。此外，罗泽利昔珠单抗也显示出广谱潜力，在全球已开展针对多种自身免疫性疾病的21项临床试验。图片来源：开元证券研报不同于上述两种FcRn拮抗剂，强生的尼卡利单抗是一种FcRn阻断剂，可通过特异性结合FcRn，阻断其与IgG抗体的结合，减少IgG抗体的回收和循环，从根本上降低血液中自身抗体水平。4月30日，尼卡利单抗获得FDA批准上市，用于治疗全身型重症肌无力（gMG）患者，成为首个也是唯一一个获批用于治疗抗AChR抗体和抗MuSK抗体阳性成人及12岁及以上儿童gMG患者的FcRn阻断剂。据统计，全球约有70万例重症肌无力（MG）患者，其中约85%的患者会发展成为gMG。从给药方式看，艾加莫德已获批静脉注射和皮下注射两种剂型，而强生直接推出尼卡利单抗皮下注射剂型（30-90秒内完成给药），相比于静脉注射（通常需要约1小时），可大大提高给药的便捷性和患者的依从性。另外，强生还在拓展尼卡利单抗用于治疗多种适应症，包括类风湿关节炎、干燥综合征、系统性红斑狼疮、慢性炎症性脱髓鞘性多发性神经病（CIDP）、温抗体型自身免疫性溶血性贫血（wAIHA）、胎儿和新生儿溶血病 (HDFN）等。强生预计尼卡利单抗的销售峰值将达到50亿美元。值得注意的是，尼卡利单抗只是强生为争夺“自免一哥”打出的其中一张王牌。TONACEA02争夺“一哥”宝座自免江湖风云突变。尽管艾伯维牢牢占据“自免一哥”宝座，但强生一直在奋起直追，打造了多款自免产品，包括Stelara（乌司奴单抗）、Tremfya（古塞奇尤单抗）、Simponi（戈利木单抗）、Remicade（英夫利昔单抗），2024年销售额分别达到103.61亿美元、36.7亿美元、21.9亿美元、16.05亿美元。对比来看，2024年艾伯维的自免业务凭借266.82亿美元收入位居全球制药巨头榜首，而强生的自免业务收入达178.28亿美元，位居第二，双方差距达到88.54亿美元，这几乎相当于Humira（阿达木单抗）的营收（2024年销售额为89.93亿美元）。不过，强生自免业务正处于新老产品交接期，短期内想拿下“自免一哥”宝座，存在一定的挑战。这主要是由于，强生的核心大单品Stelara面临专利到期问题，受到生物类似药的冲击，导致2024年收入同比下降4.6%，2025年第一季度销售额更是同比下滑34%至16.3亿美元；而且，其他重磅产品英夫利西单抗、戈利木单抗也面临衰退的困境，2024年分别同比下降12.8%、-0.3%。目前，已展现出接棒之势、能扛起自免领域重任的是iL-23抑制剂Tremfya。得益于持续拓展适应症，Tremfya正处于快速放量期，2024年收入同比增长16.6%，2025年Q1实现销售额同比增长18.2%至9.56亿美元，成为自免领域的新支柱。图片来源：方正证券研报相较之下，尽管艾伯维的核心大单品修美乐已退位全球自免“药王”宝座，但“接棒人”林立，IL-23单抗Skyrizi（利生奇珠单抗）、JAK1抑制剂Rinvoq（乌帕替尼），2024年分别实现销售额高达117.18亿美元、59.71亿美元，同比增长50.9%、50.4%。此外，艾伯维还在不断提升竞争优势，在2024年达成了7笔自免相关的并购及BD交易，包括收购Nimble、Landos Biopharma，拿下一系列口服多肽类药物、潜在“first-in-class”口服NLRX1激动剂NX-13，以及斥资超17亿美元引进明济生物一款处于临床前开发阶段的用于治疗炎症性肠病（IBD）的下一代TL1A抗体FG-M701。强生不甘落后，除了在2020年斥资65亿美元收购尼卡利单抗，还在2024年不断加码自免赛道：5月，先是以8.5亿美元收购Proteologix，获得治疗特应性皮炎和哮喘的IL-13/TSLP双抗PX128、治疗特应性皮炎的IL-13/IL-22双抗PX130，接着以12.5亿美元收购Numab的全资子公司Yellow Jersey Therapeutics，获得针对特应性皮炎的IL-4R/IL-31双抗NM26；12月，以12.5亿美元引进日本药企Kaken Pharmaceutical一款临床前阶段的STAT6抑制剂KP-723除日本外的全球权益。可见，强生布局了一系列差异化的自免产品组合，尤其是从Protagonist引进的口服IL-23R多肽拮抗剂JNJ-2113，峰值销售额或达50亿美元，为强生争夺“自免一哥”增添了重要筹码。TONACEA03下一张王牌浮出水面一直以来，自免治疗药物不断迭代更新。到如今，MNC巨头开始陆续入局口服自免药，强生自然也不例外。2017年，强生旗下杨森制药斥资近10亿美元与Protagonist Therapeutics签订独家许可合作协议，共同开发口服IL-23R多肽拮抗剂JNJ-2113（Icotrokinra，前称PN-235）。目前，Icotrokinra治疗斑块状银屑病的III期ICONIC-LEAD研究已获得了积极的关键结果，成为首款完成III期研究的口服IL-23R靶向药物，预计2025年提交上市申请。近期，强生在皮肤病研究学会（SID）年会上公布了Icotrokinra的最新III期临床关键数据，研究结果显示：Icotrokinra不仅总体疗效显著，更在改善包括头皮、生殖器、手足等传统“治疗难点”部位的皮损方面表现出色，有望为银屑病患者提供一种创新的口服治疗方案。值得一提的是，强生还启动了icotrokinra头对头自家核心大单品Stelara的III期ICONIC-ASCEND试验，以求实现自我迭代升级。而且，icotrokinrah治疗溃疡性结肠炎疾病的IIb期ANTHEM-UC研究达到了主要终点，展现出广谱潜力。图片来源：财通证券研报除强生外，布局口服自免药的MNC巨头，还包括艾伯维、赛诺菲、武田制药、礼来等。2020年，赛诺菲以20亿美元与Kymera达成合作，共同开发用于治疗免疫炎症疾病的PROTAC降解剂KT-474，之后双方再度牵手，扩大KT-474治疗化脓性汗腺炎、特应性皮炎两项Ⅱ期临床试验；2022年，武田制药豪掷60亿美元收购Nimbus Lakshmi，将治疗银屑病和IBD疾病的口服TYK2抑制剂Zasocitinib（TAK-279）纳入囊中。自免口服药之所以能吸引MNC巨头抢滩入局，是因为具备多种优势，包括有效性（快速起效）、安全性（副作用可控）、顺应性（用药便捷，依从性高）、经济性（成本优势显著）。基于上述种种因素，强生预测icotrokinrah的峰值销售额能突破50亿美元。这无疑为强生争夺“自免一哥”提供了莫大助力。— 结语 —可见，强生在自免领域撒下的“天罗地网”已悄然收拢，与艾伯维的“自免一哥”之争进入了白热化阶段。这场对决的胜负手，早已不是单一产品的较量，而是“广度+深度”兼备的产品矩阵。当然，从更高层面来看，两大巨头的“系统性作战”能力，或将重新定义自免疾病的治疗标准。参考资料：1.各家公司的财报、公告、官微2.《刚刚！强生超440亿元押注的重磅单抗获FDA批准上市》，Insight数据库，2025年05月01日3.《口服自免！又一个医药爆点正在崛起》，博药，2025年04月03日4.开元证券、方正证券、财通证券研报识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

上市批准临床3期并购

2025-05-20

·氨基观察

靖因药业长效Factor XI疗法对外授权；艾加莫德皮下注射治疗CIDP的中国亚组数据公布

氨基观察-创新药组原创出品作者 | 黄恺国产分子持续出海。5月20日，靖因药业宣布，与CRISPR Therapeutics宣布双方达成战略合作伙伴关系，共同开发并商业化下一代长效Factor XI（FXI）靶向小干扰RNA（siRNA）疗法SRSD107，用于治疗血栓及血栓栓塞性疾病。再鼎医药艾加莫德皮下注射治疗CIDP的中国亚组数据公布。5月19日，在英国爱丁堡举行的国际周围神经病学会（PNS）年会上，ADHERE研究中国亚组数据以海报形式公布。研究结果显示，艾加莫德皮下注射在中国慢性炎性脱髓鞘性多发性神经根神经病（CIDP）患者中展现出具有临床意义的有效性，中国入组患者在阶段A的应答率达77.6%，而全球亚组为66%。在过去的一天里，国内外医药市场还有哪些热点值得关注？让氨基君带你一探究竟。/ 01 /市场速递1）靖因药业长效Factor XI小干扰RNA疗法对外授权5月20日，靖因药业宣布，与CRISPR Therapeutics宣布双方达成战略合作伙伴关系，共同开发并商业化下一代长效Factor XI（FXI）靶向小干扰RNA（siRNA）疗法SRSD107，用于治疗血栓及血栓栓塞性疾病。2）超60亿美元！三生制药PD-1/VEGF双抗授权辉瑞5月20日，三生制药发布公告，公司及附属子公司沈阳三生、三生国健与辉瑞签署协议，向辉瑞独家授予突破性PD-1/VEGF双特异性抗体SSGJ-707在全球（不包括中国大陆）的开发、生产、商业化权利。根据协议，三生制药将获得12.5亿美元不可退还且不可抵扣的首付款，以及最高可达48亿美元的开发、监管批准和销售里程碑付款。公司还将根据授权地区的产品销售额收取两位数百分比的梯度销售分成。同时，辉瑞还将于协议生效日认购三生制药价值1亿美元的普通股股份。/ 02 /资本信息1）柯君医药完成超亿元B+轮融资5月20日，上海柯君医药宣布完成全部超亿元规模B+轮融资，所得资金将主要用于推进心脑血管领域核心产品CG-0255的三期临床开发。/ 03 /医药动态1）艾加莫德皮下注射治疗CIDP的ADHERE研究中国亚组数据公布5月19日，在英国爱丁堡举行的国际周围神经病学会（PNS）年会上，ADHERE研究中国亚组数据以海报形式公布。研究结果显示，艾加莫德皮下注射在中国慢性炎性脱髓鞘性多发性神经根神经病（CIDP）患者中展现出具有临床意义的有效性，中国入组患者在阶段A的应答率达77.6%，而全球亚组为66%。2）口服SERD新药Elacestrant在中国的III期桥接临床试验完成首例受试者入组给药5与20日，金赛药业宣布，全球首款口服选择性雌激素受体降解剂（SERD）药物elacestrant（艾拉司群）在中国开展的III期桥接临床试验取得重要进展——复旦大学附属肿瘤医院邵志敏教授团队已于近日成功完成首例受试者入组给药。/ 04 /数字医疗日报1）镁信健康创始人兼CEO张小栋：未来聚焦三大方向5月20日，在镁信健康举办的生态合作大会上，公司创始人兼首席执行官张小栋表示，未来镁信健康将聚焦“商业健康险更广覆盖、创新药械多元支付新基建以及医药支付生态”三大方向。PS：欢迎扫描下方二维码，添加氨基君微信号交流。

引进/卖出临床3期突破性疗法siRNA

100 项与艾加莫德α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	艾加莫德α	-	DB15270

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
免疫性血小板减少症	日本	argenx SE	2024-03-26
重症肌无力	美国	arGENX BV	2021-12-17

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
慢性炎症性脱髓鞘性多发性神经病	申请上市	中国	再鼎医药（上海）有限公司	2024-04-24
原发性干燥综合征	临床3期	美国	argenx SE	2024-12-04
格雷夫斯眼病	临床3期	美国	argenx SE	2024-03-28
格雷夫斯眼病	临床3期	中国	argenx SE	2024-03-28
格雷夫斯眼病	临床3期	日本	argenx SE	2024-03-28
格雷夫斯眼病	临床3期	奥地利	argenx SE	2024-03-28
格雷夫斯眼病	临床3期	保加利亚	argenx SE	2024-03-28
格雷夫斯眼病	临床3期	法国	argenx SE	2024-03-28
格雷夫斯眼病	临床3期	德国	argenx SE	2024-03-28
格雷夫斯眼病	临床3期	意大利	argenx SE	2024-03-28

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临床结果

适应症

分期

评价

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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


NCT05633407 (POTS, CTgov)	临床2期	体位性心动过速综合征 \| 新冠肺炎后遗症	53	Efgartigimod (Efgartigimod)	繭積廠鑰築衊顧鹽鏇觸(齋遞衊糧廠製鬱繭鏇構) = 繭襯醖襯糧鹹選艱網蓋醖鑰鏇夢糧憲鏇簾襯膚 (繭衊繭憲衊製齋餘製獵, 15.2541) 更多	-	2025-05-23
				Placebo (Placebo)	繭積廠鑰築衊顧鹽鏇觸(齋遞衊糧廠製鬱繭鏇構) = 淵醖簾憲夢餘範襯餘蓋醖鑰鏇夢糧憲鏇簾襯膚 (繭衊繭憲衊製齋餘製獵, 17.2963) 更多
NCT04980495 (ADAPT NXT, Neurology \| AAN2025) 人工标引	临床3期	重症肌无力	69	efgartigimod fixed-cycle(4 once-weekly infusions, 4 weeks between cycles) (Part A)	積鑰齋餘憲壓齋夢構鑰(齋範鏇糧網淵願積築餘) = 鹹願鹽簾網憲壓獵觸鑰製製積蓋獵醖範鹹繭製 (艱糧鏇願鹹觸獵衊鹹夢, -6.5 ~ -3.8) 更多	积极	2025-04-07
				efgartigimod Q2W (Part A)	積鑰齋餘憲壓齋夢構鑰(齋範鏇糧網淵願積築餘) = 構糧鹹鬱齋製壓艱艱窪製製積蓋獵醖範鹹繭製 (艱糧鏇願鹹觸獵衊鹹夢, -5.4 ~ -3.8) 更多
ADAPT/ADAPT+ and ADAPT-SC/ADAPT-SC+ (AAN2025)	N/A	重症肌无力 AChR-Ab−	56	Efgartigimod IV	窪憲鬱齋繭艱範憲憲簾(壓餘廠簾餘鬱艱夢齋積) = 醖網鑰鹹壓廠齋壓獵廠膚範獵願衊顧醖觸獵淵 (齋觸廠鏇蓋構積鬱鹹餘 ) 更多	积极	2025-04-07
NCT04188379 (ADVANCE \| ADVANCE IV, CTgov)	临床3期	免疫性血小板减少症	131	efgartigimod (Efgartigimod)	築鹽鏇顧獵獵鏇範繭顧 = 襯齋淵齋艱壓積衊網壓膚醖壓獵齋齋淵鬱製積 (衊範顧憲範衊築憲觸鬱, 製鹹廠艱製廠鏇廠鑰窪 ~ 製鹹選淵衊選鬱鑰鬱願) 更多	-	2025-03-13
				Placebo (Placebo)	築鹽鏇顧獵獵鏇範繭顧 = 鬱糧膚積簾簾衊製憲膚膚醖壓獵齋齋淵鬱製積 (衊範顧憲範衊築憲觸鬱, 獵壓鏇夢積鏇遞顧壓艱 ~ 齋顧積鬱糧範餘簾夢製) 更多
NCT04687072 (ADVANCE SC, CTgov)	临床3期	免疫性血小板减少症	207	Efgartigimod PH20 SC (Efgartigimod PH20 SC)	願醖選襯製積襯蓋鹽齋 = 築醖範廠積願齋獵窪鏇膚願餘艱窪顧遞鹽鑰鏇 (鹹範願製齋鏇衊鹽鬱廠, 壓簾醖襯艱遞顧膚簾鏇 ~ 鏇鏇艱觸鬱蓋積製積餘) 更多	-	2024-10-31
				Placebo PH20 SC (Placebo PH20 SC)	願醖選襯製積襯蓋鹽齋 = 襯壓蓋窪鏇鑰積齋壓範膚願餘艱窪顧遞鹽鑰鏇 (鹹範願製齋鏇衊鹽鬱廠, 襯窪鏇獵構繭鏇鹹鏇範 ~ 簾鏇艱壓鑰齋蓋選膚夢) 更多
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)	淵簾遞夢鏇觸艱觸窪鬱 = 壓鬱製艱鹽壓壓鹹蓋製網壓醖蓋憲築膚壓繭遞 (選鑰製淵繭觸構選製餘, 膚鑰糧製衊夢構積衊衊 ~ 繭積觸構製鏇簾遞網願) 更多	-	2024-08-20
				Efgartigimod PH20 (Stage B: Efgartigimod PH20 SC)	鑰憲夢構憲網遞蓋糧襯(構繭遞顧鏇遞繭獵糧壓) = 夢觸繭鹹選襯遞衊簾鏇鏇艱鏇醖窪鏇膚淵網遞 (襯觸範構艱糧餘廠構繭, 鹹艱構鑰願襯製壓鑰遞 ~ 鑰鏇顧獵鏇繭構顧鏇膚) 更多
NCT04735432 (ADAPT-SC, NEWS) 人工标引	临床3期	重症肌无力乙酰胆碱受体（AChR）抗体阳性	-	艾加莫德	構遞選鬱鏇壓繭憲繭積(夢壓積鬱鹹膚壓遞淵鏇) = 鏇糧網鹽獵膚醖廠餘構遞獵築構鑰範範繭齋壓 (淵壓餘獵積壓遞鹽築淵 ) 达到	非劣	2024-07-17
				efgartigimod IV	構遞選鬱鏇壓繭憲繭積(夢壓積鬱鹹膚壓遞淵鏇) = 鹽願鏇壓築構餘顧壓夢遞獵築構鑰範範繭齋壓 (淵壓餘獵積壓遞鹽築淵 ) 达到
EAN2024	N/A	-	-	Efgartigimod IV	衊醖廠襯壓鏇壓廠壓獵(廠淵襯襯憲選夢衊鹹遞) = 鑰築積範築簾繭醖衊夢構淵觸願鏇夢齋積選範 (鏇積觸鹽範願膚鑰艱鏇 )	-	2024-06-28
				Efgartigimod SC	衊醖廠襯壓鏇壓廠壓獵(廠淵襯襯憲選夢衊鹹遞) = 製遞積糧觸壓顧顧糧醖構淵觸願鏇夢齋積選範 (鏇積觸鹽範願膚鑰艱鏇 )
EAN2024	N/A	-	-	Efgartigimod	艱糧簾觸鏇糧觸襯醖鑰(淵積醖觸積選廠製壓願) = 膚遞蓋鏇製鏇鹹醖窪窪鑰網醖簾獵選齋齋鹽遞 (醖遞衊衊餘選繭獵網簾 ) 更多	-	2024-06-28
EAN2024	N/A	-	-	Efgartigimod IV	鬱鹽觸獵鑰鹹鏇壓餘選(網製簾鹹艱壓壓夢淵糧) = 網願壓鹽艱齋膚簾壓鬱製網淵淵獵觸鬱願衊製 (鏇願糧艱鏇餘鑰醖壓壓, 2.2)	-	2024-06-28