Empasiprubart

Long-term and real-world data of VYVGART® (efgartigimod alfa-fcab) and VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) demonstrate speed of onset, depth of response, and durability of response VYVGART demonstrates consistent, favorable safety profile from follow-up safety data that totals >8,000 patient years; no vaccinations required and no impact on human serum albumin levels Real-world data show more than 50 percent of gMG patients demonstrate substantial and sustained reduction in steroid use following VYVGART initiation argenx continues to expand its reach in neurology through pipeline programs, including empasiprubart advancing in MMN and ARGX-119 in ALS and CMS October 15, 2024 – 7:00am CET 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 clinical and real-world data across its growing immunology pipeline at the 2024 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and Myasthenia Gravis Foundation of America (MGFA) Scientific Sessions in Savannah, GA from October 15-18, 2024. “VYVGART continues to deliver impactful benefits to patients in terms of safety, speed of onset, depth of response, and durability of response,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. “The robust data we are showing at AANEM and MGFA continue to confirm VYVGART as the leading innovative biologic with an established ability to reduce steroid usage, drive minimal symptom expression for gMG patients, and reduce CIDP symptoms quickly. In addition to VYVGART, we are excited to highlight our growing neurology pipeline, including empasiprubart and ARGX-119, through which we can advance our mission of delivering transformative outcomes for even more patients.” VYVGART and VYVGART Hytrulo Demonstrate Rapid, Deep and Sustained Responses in gMG and CIDP The data presented at AANEM continue to demonstrate the significant impact of VYVGART (including VYVGART Hytrulo), the first-in-class neonatal Fc receptor (FcRn) blocker for people living with generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP). VYVGART is setting a new treatment standard in gMG and has shown rapid, deep and sustained responses, enabling a majority of patients to achieve minimal symptom expression (MSE) with a consistent and favorable safety profile and more than 8,000 patient years of safety data. Based on real-world data, more than half of patients can reduce steroid use by >5mg/day following VYVGART initiation. In CIDP, a majority of patients in the ADHERE trial responded to VYVGART Hytrulo and experienced reduced risk of relapse versus placebo and improvements in motor function and muscle strength, regardless of prior CIDP treatment. Highlights from VYVGART data presented at AANEM and MGFA: Early Line Use and Meaningful Steroid Reduction: VYVGART demonstrates consistent improvement across gMG patient subtypes, including those on mestinon alone, indicating its efficacy in early line use. Real-world gMG data show that at one-year post VYVGART initiation, 55% of patients reduced corticosteroid use by ≥5 mg/day and 42% of patients had achieved steroid doses of ≤5mg/day.Expansion to Seronegative and Ocular MG: argenx is honoring its long-term commitment to the broader MG community with two Phase 3 studies ongoing in additional MG patient populations, including seronegative (ADAPT-SERON) and ocular MG (ADAPT-OCULUS). Seronegative (AChR-) gMG patients evaluated in VYVGART clinical studies experienced consistent and clinically meaningful MG-ADL improvements, including patients achieving MSE.Sustained Functional Benefit in CIDP: VYVGART Hytrulo showed sustained functional benefit in motor function and muscle strength, regardless of prior treatment, which was maintained through ADHERE and the open-label extension study (through week 24).Consistent, Favorable Safety Profile: VYVGART’s consistent and favorable safety profile has been established across multiple autoimmune diseases with no increase in the incidence of adverse events with increased exposure. The unique safety profile of VYVGART is further supported by no black box warnings, no labs or immunoglobulin (Ig) monitoring, and no vaccination requirements. Advancing Immunology Pipeline Across Two First-in-Class Opportunities to Reach New Patients argenx will also highlight two additional pipeline candidates, including Phase 2 ARDA data of empasiprubart (anti-C2 inhibitor) for the treatment of multifocal motor neuropathy (MMN), and clinical trial designs of ARGX-119 (muscle-specific kinase (MuSK) agonist) for the treatment of congenital myasthenic syndromes (CMS) and amyotrophic lateral sclerosis (ALS). Cohort 1 data from the Phase 2 ARDA study will be presented in a poster, showing treatment with empasiprubart for MMN reduced the risk of IVIg retreatment by 91% (HR: 0.09 [95% CI: 0.02–0.44]) and demonstrated significant improvement in grip strength in both hands as compared to placebo. Data from a Patient Global Impression of Change scale show 94.4% of patients said they improved on empasiprubart from the start of the study, compared to 11.1% of placebo patients. As part of its commitment to the MMN community, argenx initiated the iMMersioN longitudinal study of ~150 patients to collect data on the impact of disease and burden of current treatment options on clinical outcomes and quality of life measures. A Phase 3 study of empasiprubart in MMN will start by the end of 2024. argenx posters included in MGFA Scientific Sessions All MGFA posters to be presented on Tuesday, October 15, 12:00 – 12:45pm ETPosters with an asterisk (*) will also be presented in AANEM scientific program Full TitlePresentation DetailsPatterns of Efgartigimod Dosing in Clinical Practice in the United StatesPoster # MG9Real-World Reduction in Oral Glucocorticoid Utilization at 1-Year Following Efgartigimod Initiation*Poster # MG31 / AANEM Poster # 262Efficacy, Safety, and Pharmacodynamics of Efgartigimod PH20 SC Across Bodyweight Quartiles: A Post hoc Analysis of the ADAPT-SC+ TrialPoster # MG32Fixed Cycle and Every-Other-Week Dosing of Intravenous Efgartigimod for Generalized Myasthenia Gravis: Part A of ADAPT NXT*Poster # MG33 /AANEM Poster # 182Design of a Phase 3 Randomized, Double-Blinded, Placebo-Controlled Study Evaluating the Efficacy and Safety of Subcutaneous Efgartigimod PH20 Administered by Prefilled Syringe in Adults with Ocular Myasthenia GravisPoster # MG38Phase 3 Trial Investigating Impact of Intravenous Efgartigimod in Anti-Acetylcholine Receptor Antibody Negative Generalized Myasthenia Gravis*Poster # MG39 / AANEM Poster # 178Observed Efficacy of Efgartigimod in Generalized Myasthenia Gravis Across Patient Subgroups in the ADAPT-SC+ StudyPoster # MG68Exploring the Impact of Non-Steroidal Immunosuppressive Drugs and Steroids on the Development of Comorbidities in Patients with Myasthenia Gravis in the National Veterans Affairs Health NetworkPoster # MG86Quality of Life of Patients with Symptomatic Ocular MG: Comparison with the General PopulationPoster # MG87Steroid Use, Toxicity, and Monitoring in Patients With Generalized Myasthenia Gravis: A Survey Of Neurologists In The United States*Poster # MG89 / AANEM Poster # 235Comparative Risk-Benefit Profiles of Immunomodulatory Therapies for Patients with Generalized Myasthenia GravisPoster # MG98 argenx posters included in AANEM Scientific Program * Session Times: Session I: Wednesday, October 16, 6:15 - 6:45pm ETSession II: Thursday, October 17, 9:30 - 10am ETSession III: Thursday, October 17, 2:45 - 3:15pm ET Full TitlePresentation Details*Long-Term Safety and Efficacy of Efgartigimod PH20 SC in Generalized Myasthenia Gravis: Interim Analysis of Anti-Acetylcholine Receptor Antibody Seronegative Participants in ADAPT-SC+Poster # 144Session I & Session IICombined Analyses of Participants With Anti-Acetylcholine Receptor Seronegative Generalized Myasthenia Gravis Treated With Efgartigimod Across Clinical StudiesPoster # 146Session I & Session IISafety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Participants With DOK7 Congenital Myasthenic Syndromes: Phase 1b Study in ProgressPoster # 165Session I & Session IIIEfficacy and Safety of Subcutaneous Efgartigimod PH20SC in Chronic Inflammatory Demyelinating Polyneuropathy: ADHERE Trial Subgroup AnalysisPoster # 176Session I & Session IIEfficacy and Safety of Subcutaneous Efgartigimod PH20 in Chronic Inflammatory Demyelinating Polyneuropathy: ADHERE/ADHERE+ TrialPoster # 177Session I & Session IIISafety Profile of Intravenous Efgartigimod From Clinical Trials in Immunoglobulin G–Mediated Autoimmune DiseasesPoster # 180Session I & Session IISafety Profile of Subcutaneous Efgartigimod PH20 From Clinical Trials in Immunoglobulin G–Mediated Autoimmune DiseasesPoster # 181Session I & Session IIIEmpasiprubart (ARGX-117) in Multifocal Motor Neuropathy: Initial Safety and Efficacy Data of the Phase 2 ARDA StudyPoster # 198Session I & Session IILong-Term Safety, Tolerability, and Efficacy of Subcutaneous Efgartigimod PH20 in Participants With Generalized Myasthenia Gravis: Interim Results of the ADAPT-SC+ StudyPoster # 212Session I & Session IIISafety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of ARGX-119 in Patients with Amyotrophic Lateral Sclerosis: A Phase 2a Study in ProgressPoster # 237Session I & Session IIRisk of Serious Infections and Malignancies in Adult Myasthenia Gravis Patients: A US Claims Database StudyPoster # 251Session I & Session IIChronic Steroid Toxicity in Adults With Myasthenia Gravis in the United States Based on Electronic Health RecordsPoster # 263Session I & Session IISubcutaneous Efgartigimod PH20 in Chronic Inflammatory Demyelinating Polyneuropathy: Key Secondary Outcomes from the ADHERE TrialPoster # 280Session I & Session IIIEmpasiprubart (ARGX-117) in Multifocal Motor Neuropathy: Baseline Characteristics and MMN Confirmation Committee Outcome of the Phase ARDA Study Cohort 1Poster # 293Session I & Session IICOVID-19 Vaccination Response in Participants Receiving Efgartigimod IV or Efgartigimod PH20 SC in ADAPT+ or ADAPT-SC+Poster # 298Session I & Session IIISteroid Use, Toxicity, and Monitoring in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: A Survey of Neurologists in The United StatesPoster # 306Session I & Session IIIClinical Outcomes, Disease Course, and Quality of Life in Patients With Multifocal Motor Neuropathy: iMMersioN, Study in ProgressPoster # 307Session I & Session II More information on the data presented at the 2024 AANEM Annual Meeting can be found here. See FDA-approved Important Safety Information below, full Prescribing Information for VYVGART and full Prescribing Information for VYVGART Hytrulo for additional information. Important Safety Information What is VYVGART® (efgartigimod alfa-fcab)?VYVGART is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive). IMPORTANT SAFETY INFORMATIONDo not use VYVGART if you have a serious allergy to efgartigimod alfa or any of the other ingredients in VYVGART. VYVGART can cause serious allergic reactions and a decrease in blood pressure leading to fainting. VYVGART may cause serious side effects, including: Infection. VYVGART may increase the risk of infection. The most common infections were urinary tract and respiratory tract infections. Signs or symptoms of an infection may include fever, chills, frequent and/or painful urination, cough, pain and blockage of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain.Allergic Reactions (hypersensitivity reactions). VYVGART can cause allergic reactions such as rashes, swelling under the skin, and shortness of breath. Serious allergic reactions, such as trouble breathing and decrease in blood pressure leading to fainting have been reported with VYVGART.Infusion-Related Reactions. VYVGART can cause infusion-related reactions. The most frequent symptoms and signs reported with VYVGART were high blood pressure, chills, shivering, and chest, abdominal, and back pain. Tell your doctor if you have signs or symptoms of an infection, allergic reaction, or infusion-related reaction. These can happen while you are receiving your VYVGART treatment or afterward. Your doctor may need to pause or stop your treatment. Contact your doctor immediately if you have signs or symptoms of a serious allergic reaction. Before taking VYVGART, tell your doctor if you: take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines,have received or are scheduled to receive a vaccine (immunization), orhave any allergies or medical conditions, including if you are pregnant or planning to become pregnant, or are breastfeeding. What are the common side effects of VYVGART?The most common side effects of VYVGART are respiratory tract infection, headache, and urinary tract infection. These are not all the possible side effects of VYVGART. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088. Please see the full Prescribing Information for VYVGART and talk to your doctor. What is VYVGART® HYTRULO (efgartigimod alfa and hyaluronidase-qvfc)?VYVGART HYTRULO is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive).VYVGART HYTRULO is a prescription medicine used for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) IMPORTANT SAFETY INFORMATIONDo not use VYVGART HYTRULO if you have a serious allergy to efgartigimod alfa, hyaluronidase, or any of the other ingredients in VYVGART HYTRULO. VYVGART HYTRULO can cause serious allergic reactions and a decrease in blood pressure leading to fainting. VYVGART HYTRULO may cause serious side effects, including: Infection. VYVGART HYTRULO may increase the risk of infection. The most common infections for efgartigimod alfa-fcab-treated patients were urinary tract and respiratory tract infections. Signs or symptoms of an infection may include fever, chills, frequent and/or painful urination, cough, pain and blockage of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain.Allergic Reactions (hypersensitivity reactions). VYVGART HYTRULO can cause allergic reactions such as rashes, swelling under the skin, and shortness of breath. Hives were also observed in patients treated with VYVGART HYTRULO. Serious allergic reactions, such as trouble breathing and decrease in blood pressure leading to fainting have been reported with efgartigimod alfa-fcab.Infusion-Related Reactions. VYVGART HYTRULO can cause infusion-related reactions. The most frequent symptoms and signs reported with efgartigimod alfa-fcab were high blood pressure, chills, shivering, and chest, abdominal, and back pain. Tell your doctor if you have signs or symptoms of an infection, allergic reaction, or infusion-related reaction. These can happen while you are receiving your VYVGART HYTRULO treatment or afterward. Your doctor may need to pause or stop your treatment. Contact your doctor immediately if you have signs or symptoms of a serious allergic reaction. Before taking VYVGART HYTRULO, tell your doctor if you: take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines,have received or are scheduled to receive a vaccine (immunization), orhave any allergies or medical conditions, including if you are pregnant or planning to become pregnant, or are breastfeeding. What are the common side effects of VYVGART HYTRULO?The most common side effects in efgartigimod-alfa-fcab-treated patients were respiratory tract infection, headache, and urinary tract infection. Additional common side effects with VYVGART HYTRULO are injection site reactions, including rash, redness of the skin, itching sensation, bruising, pain, and hives. These are not all the possible side effects of VYVGART HYTRULO. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088. Please see the full Prescribing Information for VYVGART HYTRULO and talk to your doctor. About VYVGARTVYVGART 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 in the United States, EU, China and Canada for the treatment of adults with generalized myasthenia gravis (gMG) who are anti- acetylcholine receptor (AChR) antibody positive and in Japan for the treatment of adults with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs). About VYVGART® HytruloVYVGART Hytrulo is a subcutaneous combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART®, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. In binding to the neonatal Fc receptor (FcRn), VYVGART Hytrulo results in the reduction of circulating IgG. It is the first-and-only approved FcRn blocker administered by subcutaneous injection. VYVGART Hytrulo is the proprietary name in the U.S. for subcutaneous efgartigimod alfa and recombinant human hyaluronidase PH20. It may be marketed under different proprietary names following approval in other regions. About Generalized Myasthenia Gravis (gMG)Generalized myasthenia gravis (gMG) is a rare and chronic autoimmune disease where IgG autoantibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. Approximately 85% of people with MG progress to gMG within 24 months,1 where muscles throughout the body may be affected. Patients with confirmed AChR antibodies account for approximately 85% of the total gMG population. About Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. Although confirmation of disease pathophysiology is still emerging, there is increasing evidence that IgG antibodies play a key role in the damage to the peripheral nerves. People with CIDP experience fatigue, muscle weakness and a loss of feeling in their arms and legs that can get worse over time or may come and go. These symptoms can significantly impair a person's ability to function in their daily lives. Without treatment, one-third of people living with CIDP will need a wheelchair. About EmpasiprubartEmpasiprubart (ARGX-117) is a first-in-class humanized monoclonal antibody that binds C2 and blocks activation of both the classical and lectin pathways of the complement cascade, leaving the alternative pathway intact for its antimicrobial properties. By blocking complement activity upstream of C3 and C5, empasiprubart has the potential to reduce tissue inflammation and cellular damage, representing a broad pipeline opportunity across multiple severe autoimmune indications. In addition to multifocal motor neuropathy, argenx is evaluating empasiprubart in delayed graft function following kidney transplant, dermatomyositis and chronic inflammatory demyelinating polyneuropathy. About Multifocal Motor NeuropathyMultifocal motor neuropathy (MMN) is a rare, severe, chronic autoimmune disease of the peripheral nervous system. The disease is characterized by slowly progressive, asymmetric muscle weakness mainly of the hands, forearms and lower legs. MMN is often associated with the presence of anti-GM1 IgM autoantibodies, leading to activation of the classical complement pathway, driving subsequent axon damage. High-dose IVIg is the only approved treatment for MMN and patients typically experience disease progression despite therapy, indicating an unmet need for efficacious and better tolerated therapeutic options. About ARGX-119ARGX-119 is a humanized agonistic monoclonal antibody (mAb) that targets and activates muscle-specific kinase (MuSK) to promote maturation and stabilization of the neuromuscular junction (NMJ). MuSK is a receptor kinase that has a critical role in the structure and function of the NMJ. ARGX-119 is being developed as a potential therapy for patients with neuromuscular disease. About Congenital Myasthenic Syndromes (CMS) Congenital Myasthenic Syndromes (CMS) are a heterogenous group of rare genetic disorders of the neuromuscular junction (NMJ) that lead to muscle weakness. CMS cases are classified into subtypes depending on the underlying genetic mutation. While clinical features vary widely across and within subtypes, the predominant manifestation of CMS is fatigable weakness. Age of onset varies widely; while many patients are diagnosed in infancy or early childhood, patients with milder phenotypes may not present or be diagnosed until adulthood. About Amyotrophic Lateral Sclerosis (ALS) ALS is a neurodegenerative disorder of the brain and spinal cord leading to deteriorating muscle function, weakness and atrophy. The multisystemic nature can have impacts throughout the body with specific signs and symptoms associated with lower motor neuron and upper motor neuron loss. Life expectancy is typically 2.5-5 years from diagnosis. About argenxargenx 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 in the U.S., Japan, Israel, the EU, the UK, Canada and China. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, X/Twitter, Instagram, Facebook, and YouTube. ContactsMedia:Ben Petokbpetok@argenx.com Investors:Alexandra Roy (US)aroy@argenx.com Lynn Elton (EU)lelton@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 can be identified by the use of forward-looking terminology, including the terms “aim,” and “will,” and include statements argenx makes regarding the anticipated timing of the initiation of the Phase 3 clinical trial for empasiprubart in MMN and its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’s actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including 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 in products and product candidates; the acceptance of argenx's products and product candidates by patients as safe, effective and cost-effective; the impact of governmental laws and regulations on our business; disruptions caused on our reliance of 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. ###

本文系基于公开资料撰写，仅作为信息交流之用，不构成任何投资建议。 再鼎医药终于找到营收第二增长点，凭借卫伟迦（艾加莫德）营收额的显著放量，公司净利润亏损大幅收窄。 一直以来，再鼎医药的最大问题正在于缺乏第二增长点，完全依靠则乐苦苦支撑。在则乐营收放缓的情况下，投资者迟迟看不到盈利希望。但这一切在卫伟迦上市后改变了。 2023年9月，卫伟迦正式在国内获批，并于今年1月纳入医保目录。虽然上市仅一年时间，但卫伟迦营收额却持续增长，二季度营收达2320万美元，环比暴增75.8%，而且再鼎医药还上调卫伟迦全年营收指引至8000万美元。 或许很多投资者对于再鼎医药的基本面反转颇感意外，但艾加莫德实际上是一款早已在全球市场证明自身价值的产品，其背后的Argenx公司更是凭借艾加莫德的成功，股价在7年间涨了30多倍，当下市值超越300亿美元，成为全球Biotech最渴望成为的标杆。 01 Biotech的范本 Argenx于2008年在荷兰成立，与大多数初创的生物技术公司一样，Argenx也是通过讲创新故事敲开了投资基金的大门。 当时，Argenx公司打造了一个名为“SIMPLE Antibody”的平台，它是全球唯一一个基于美洲驼IgG1抗体可变区进行治疗性抗体药物开发的平台。美洲驼是一种具有强大免疫系统的骆驼，它们产生的许多抗体与人类的抗体有许多相似之处，与人类治疗靶基因的同源性在60%—86%之间。当美洲驼的免疫系统与人类疾病靶点产生免疫反应时，就会生成高度多样化的抗体库。 借助SIMPLE Antibody平台，Argenx能够获取并探索广泛的治疗靶点，与使用传统方法相比，最大限度地减少发现候选抗体的时间。同时，该平台还具有延长抗体半衰期、增加组织渗透率、提高疾病靶标率等诸多潜在优势，基于这个独特的平台，Argenx开发出靶向新颖且靶标复杂的抗体疗法。 此外，Argenx还有一项长期战略——免疫学创新计划（IIP）。该计划旨在将外部科学和学术合作者对疾病和生物学机制的深刻理解与公司的抗体工程经验相结合，从而建立高度差异化的临床管线。Argenx几乎所有的候选产品都来自IIP计划，并计划每年从中孵化出至少一项新的管线资产，艾加莫德正诞生于IIP计划。 2013年，德克萨斯大学西南医学中心教授Sally Ward的一项研究发现，FcRn在IgG水平的维持和全身分布方面起关键作用。这一发现很快就吸引了Argenx公司的目光，于是就与Sally Ward教授团队合作获得了艾加莫德的独家专利，并于2014年启动项目研究。 三年之后，艾加莫德首个适应症重症肌无力MG临床II期成功，Argenx公司也借此在美国纳斯达克市场IPO上市，IPO首日总市值为6亿美元。 凭借艾加莫德优秀临床数据的不断披露，Argenx股价一路高歌猛进，连上数个台阶。 2020年，艾加莫德在临床III期的重症肌无力研究中取得成功；2021年，艾加莫德获得FDA批准上市，开启商业化之路；2022年，艾加莫德收获4.01亿美元的销售额。 图：Argenx股价走势，来源：雪球 截至8月20日，Argenx市值达到312.45亿美元，7年时间上涨超过30倍。 纵观Argenx的发展历程，其走过的路径堪称Biotech的范本。凭借独特的技术优势，获得初始创业资金，再开发出一系列研发管线，不断获得融资，其中一个创新药获批上市，成为爆款，得到更多资本的青睐。 现如今，Argenx已经有资本向产业巨头迈进，孵化更多的创新产品上市，这一切全因艾加莫德的成功。 02 神奇的艾加莫德 艾加莫德是一种人源化IgG1抗体Fc片段，通过阻断FcRn受体，可降低体内病理性自身抗体水平。2021年12月17日，艾加莫德正式获得美国FDA批准用于治疗抗乙酰胆碱受体（AChR）抗体阳性的成人全身型重症肌无力（gMG）。 重症肌无力隶属于自身免疫性疾病，主要影响神经肌肉接头。临床表现为骨骼肌波动性无力和不耐疲劳，活动后加重，休息后减轻。症状多分布于眼部、球部、四肢肌肉，严重者可能影响呼吸肌，造成呼吸衰竭。目前，约85%的gMG患者确认携带有乙酰胆碱受体（AChR）抗体。 虽然全球重症肌无力的年发病率并不高，但由于重症肌无力是一种慢性病，真实患者群体规模需累加计算，目前重症肌无力实际患病率大约为万分之一，全球合计患者人数至少超70万人。 在艾加莫德获批之前，重症肌无力并没有特别好的靶向疗法，仍以非靶向的糖皮质激素、免疫抑制剂等为主，虽然在一定程度上能改善患者的肌无力症状，但在疾病长期控制以及安全性等方面均存在不足。 随着重症肌无力创新药物上市，市场需求持续攀升，根据弗若斯特沙利文测算，全球重症肌无力治疗药物市场将从2020年的12.6亿美元增至2025年的30.48亿美元，复合增长率达19.3%。 艾加莫德是一种靶向FcRn的拮抗剂，FcRn能与体内的抗体结合，阻止抗体在溶酶体内的降解，在体内循环利用，参与疾病的发生和发展过程。艾加莫德可以组织抗体与FcRn结合，从而加快致病性抗体的溶解，其中就包括重症肌无力中存在的异常AChR抗体。 相较于现有的治疗方案，艾加莫德具有起效快、疗效相对持久、安全性优等特点，尤其是对于难治型患者和可能发展为危象的患者具有显著优势。 得益于在重症肌无力治疗领域的竞争力，艾加莫德仅凭这一个适应症就在上市首年（2022年）就斩获4.01亿美元的销售额，并在2023年轻松跨入“10亿美元俱乐部”，达到11.91亿美元。 2024年上半年，艾加莫德销量继续高歌猛进，销售收入8.75亿美元，同比增长79.7%，Argenx也凭此在二季度扭亏为盈，并有望实现2024年全年盈利。 03 Pipeline in a drug 尽管艾加莫德已经展现出超强竞争力，但其距离天花板却依然很远，因为其仍有极大的“Pipeline in a drug”价值。 所谓Pipeline in a drug，是一种药物开发策略，即一种药物用于多种适应症的情形。从修美乐到K药，新老两代药王都是属于这种研发策略的产物，前者获得的FDA针对不同适应症的批准12次，后者更是多达40次。 对于药企而言，这种产品就属于“自带管线的药物”，药企可以用更低的临床成本，最大化一个产品的商业化潜力，并实现极大的商业价值。 艾加莫德同样具有“Pipeline in a drug”的潜质。 由于很多自身免疫性疾病都是由IgG与自身抗原结合引起的，而FcRn延长了IgG在循环中的半衰期从而加速了自免疾病进程， FcRn成为治疗这些相关疾病的重要靶点。因此，靶向FcRn的艾加莫德理论上可治疗的适应症非常广泛。 除了全身型重症肌无力外，艾加莫德还分别于2024年3月和6月在日本与美国获批用于原发性免疫性血小板减少症（ITP）及慢性炎性脱髓鞘性多发性神经根神经病（CIDP）。 ITP主要是由于机体内免疫功能异常导致的血小板破坏增多和血小板生成减少所致，是一种常见的获得性自身免疫性出血性疾病。目前，ITP治疗主要以激素、TPO类药物、CD20单抗为主，仍存在巨大未被满足的需求，全球市场价值达到30亿美元左右。 CIDP是一种免疫介导的多发性神经病（临床上又称之为对称性肌无力）。目前，临床治疗CIDP主要以静脉注射免疫球蛋白（IVIG）、糖皮质激素、血浆置换等为主，但治疗效果十分有限。艾加莫德是30多年来首个获得批准且具有明确作用机制的CIDP新疗法。 除此以外，Argenx希望将艾加莫德的商业化或临床开发工作拓展到15项适应症，包括甲状腺眼病（TED）、大疱性类天疱疮（BP）、原发性干燥综合征（pSS）、新冠肺炎后体位性心动过速综合征（PC-POTS）、膜性肾病（MN）、狼疮性肾炎（LN）、寻常型天疱疮和落叶型天疱疮（PV和PF）、特发性炎症性肌病、ANCA相关性血管炎（AAV）、抗体介导的排斥反应（AMR）等。 图：Argenx研发管线，来源：Argenx官网 作为FcRn抑制剂的先行者，随着更多适应症的获批，艾加莫德的市场空间也将不断扩大。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。 除了艾加莫德，Argenx还有其他几款临床研发管线，包括ARGX-117、ARGX-119，以及数个早期候选产品，但这些管线距离获批上市还有很长的路要走，未来3-5年，Argenx仍需围绕艾加莫德进行不断拓展及迭代。 从研发策略看，Argenx避开了MNC激烈竞争的肿瘤、慢病等热门领域，选择源头创新，并布局有差异化的适应症，迅速成为冉冉升起的Biotech新星。Argenx的发展模式及研发策略，在同质化创新严重的中国生物医药领域，尤其值得借鉴。 Argenx的表现证明，艾加莫德具有足够商业价值，或许这一次再鼎医药真的找到了一座“金山”。 免责声明 “药渡Daily”公众号所转载该篇文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请及时告知，我们会在24小时内删除相关信息。