Antisynthetase Syndrome

Fundraise brings lead product, TriVerity™ Test, closer to commercialization following recent submission to FDA SUNNYVALE, Calif., Sept. 12, 2024 /PRNewswire/ -- Inflammatix, a pioneering molecular diagnostics company, announced today the closing of $57 million in Series E financing, led by Khosla Ventures and Think.Health. The funds will support regulatory filing and early commercialization of the company's lead product, the TriVerity™ Acute Infection and Sepsis Test (TriVerity). TriVerity is a blood test that is intended to fill a critical need in the emergency department (ED) setting. Each year, 20 million people arrive at the hospital suspected of having an acute infection or sepsis. Recent company-sponsored clinical studies1,2 validate previous research3-5 suggesting that physician assessment and vital-signs-based scoring underestimate severity in up to half of patients suspected of acute infection or sepsis. With an annual incidence of 2.7 million cases and an annual mortality rate of 350,0006, sepsis is a leading cause of death in U.S. hospitals and costs the Centers for Medicare and Medicaid Services (CMS) $53 billion each year, making it the most costly diagnosis among Medicare beneficiaries.7 "The newly raised funds will help us to expand our commercial team and plan clinical interventional and health economic studies as we await FDA clearance over the coming months," said Dr. Timothy Sweeney, CEO and co-founder of Inflammatix. "TriVerity is bringing the promise of machine learning and AI to infection and sepsis care. We hope to help hospitals improve their performance in terms of complying with sepsis detection and treatment protocols and optimizing patient throughput. We greatly appreciate the strong investor confidence in TriVerity and are excited to be very close to offering this novel test to physicians." In addition to the leading investors Khosla Ventures and Think.Health, the Inflammatix Series E funding round included participation from Northpond Ventures, D1 Capital Partners, Iberis Capital, Vesalius BioCapital, OSF Healthcare, RAW Ventures, and others. The funding round brings Inflammatix's total private capital raised to more than $200 million, in addition to more than $50 million in grants and contracts from various government agencies and foundations. Novel diagnostic approach to spur earlier detection of infection and sepsis TriVerity, a blood test performed on Inflammatix's novel Myrna™ Instrument, is uniquely designed to simultaneously determine whether an infection is present, and how likely a patient will need ICU-level interventions. Getting to an accurate diagnosis faster would not only save lives but would also dramatically improve hospital efficiency as well as health system resource allocation. Earlier and more accurate diagnoses may also help hospitals comply with the CMS SEP-1 Bundle, a value-based payments quality measure intended to ensure rapid sepsis detection and treatment. "Despite incremental improvements in patient outcomes, the death toll from sepsis remains disproportionately high among vulnerable populations," said Alex Morgan, partner at Khosla Ventures. "The challenge is that existing diagnostics are not able to detect sepsis early enough to trigger timely intervention, and by the time noticeable clinical symptoms appear, it's often too late. TriVerity takes a novel approach by detecting the RNA changes that occur in immune cells prior to the manifestation of disease, enabling clinicians to respond faster and sometimes before physiological symptoms are even present. This is a step-change in life-saving care that physicians have been wanting for decades." After receiving Breakthrough Device Designation from the U.S. Food and Drug Administration (FDA) in November 2023 and completing the SEPSIS-SHIELD study [NCT04094818], Inflammatix recently submitted a regulatory packet to the FDA for the TriVerity Test. The company hopes to receive FDA clearance later this year. About the TriVerity™ Acute Infection and Sepsis Test The TriVerity Acute Infection and Sepsis Test (TriVerity), Inflammatix's lead product, is performed on the Myrna™ Instrument, the company's proprietary, cartridge-based, benchtop analyzer. TriVerity is a blood test that measures 29 messenger RNAs (mRNAs) to rapidly "read" the body's immune response to infection using machine learning-derived algorithms. The test is designed to inform on the two "axes" of sepsis -- presence of infection and risk of progression to severe illness -- in adult patients with suspected acute infection or sepsis in the emergency department setting. TriVerity thus aims to help physicians to confidently make treatment decisions, including selection of antimicrobial therapy, administering additional diagnostic testing, and whether to admit or discharge the patient. The Myrna Instrument is capable of multiplex sample-to-answer quantitation of mRNAs in about 30 minutes. With its less than one-minute operator hands-on time and simple maintenance, the Myrna Instrument is designed for ease of use and low resource requirements. The TriVerity Acute Infection and Sepsis Test is not for sale. It is currently pending FDA clearance and has not received marketing approval or clearance from regulatory authorities in any jurisdiction. About Inflammatix Inflammatix, Inc., a pioneering molecular diagnostics company headquartered in Sunnyvale, California, USA, is developing novel diagnostics that rapidly read a patient's immune system to improve patient care and reduce major public health burdens. The Inflammatix tests will be developed to run on the company's sample-to-answer isothermal instrument platform, enabling the power of precision medicine at the point of care. The company's funders include Khosla Ventures, Northpond Ventures, Think.Health Ventures, D1 Capital, Iberis Capital, and Vesalius BioCapital. For more information, please visit and follow the company on LinkedIn and X (formerly Twitter) at @Inflammatix_Inc). Inflammatix product development has been funded in part with Federal funds from the Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority, under Contract Nos. 75A50119C00034 and 75A50119C00044. TriVerity, Myrna, and Inflammatix are trademarks of Inflammatix, Inc. in the U.S. and other countries and regions. References Whitfield N, Michelson EA, Steingrub J, et al. Host response severity score for ICU level care prediction in emergency patients with suspected sepsis. (2024), SAEM24 Abstracts. Acad Emerg Med, 31: 8-401. Data on file, Inflammatix, Inc. Askim A, Moser F, Gustad LT, et al. Poor performance of quick-SOFA (qSOFA) score in predicting severe sepsis and mortality – a prospective study of patients admitted with infection to the emergency department. Scand J Trauma Resusc Emerg Med. 2017;25(1):56. doi: 10.1186/s13049-017-0399-4. Tiwari AT, Jamshed N, Sahu AK, et al. Performance of qSOFA score as a screening tool for sepsis in the emergency department. J Emerg Trauma Shock. 2023;16(1):3-7. doi: 10.4103/jets.jets_99_22. Freund Y, Lemachatti N, Krastinova E, et al. Prognostic accuracy of Sepsis-3 criteria for in-hospital mortality among patients with suspected infection presenting to the emergency department. JAMA. 2017;317(3):301-308. doi: 10.1001/jama.2016.20329. Sepsis. U.S. Department of Health and Human Services, National Institutes of Health, National Institute of General Medical Sciences; 2024. . Frank CE, Buchman TG, Simpson SQ, et al. Sepsis among Medicare beneficiaries: 4. Precoronavirus Disease 2019 Update January 2012-February 2020. Crit Care Med. 2021;49(12):2058-2069. doi: 10.1097/CCM.0000000000005332. SOURCE Inflammatix WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

自身免疫性疾病是免疫系统对自身机体产生免疫反应，自身抗体或自身效应淋巴细胞攻击正常细胞，导致自身组织受损的一类疾病，如系统性红斑狼疮、1型糖尿病、重症肌无力等，多数无法治愈，需要终生治疗。理想的治疗方式是保留保护性免疫的同时消除病理性自身反应细胞。 B淋巴细胞/浆细胞是自身免疫性疾病最重要的效应细胞之一，既往嵌合抗原受体（CAR）T细胞疗法（后简称“CAR-T细胞疗法”）通过特异性杀伤B淋巴细胞在急性B淋巴细胞白血病（B-ALL）、B细胞非霍奇金淋巴瘤（B-NHL）、多发性骨髓瘤（MM）等疾病的治疗中获得了显著的疗效，提示CAR-T细胞疗法在自身免疫性疾病治疗中的巨大潜力。近期，《新英格兰医学杂志》（NEJM）发表一项具有里程碑意义的临床研究成果，表明“首批”接受CAR-T细胞疗法的15例严重自身免疫性疾病患者，在为期15个月的中位随访期内，均持续维持缓解或症状大幅减轻，并已停止使用所有免疫抑制与抗炎药物，NEJM发表的同期社论称这是项“标志性的发现”。 CAR-T细胞疗法能治愈自身免疫性疾病吗？CAR-T细胞疗法对所有自身免疫性疾病均有效吗？CAR-T细胞疗法的短期和长期疗效，以及安全性情况如何？《医学新视点》结合NEJM、《柳叶刀》（The Lancet）等医学顶刊近期发表的相关文献，与您共同了解CAR-T细胞疗法在自身免疫系统疾病中的应用情况，以飨读者。 自身免疫性系统疾病患者的免疫系统对自身抗原反应性增强，导致免疫耐受破坏，引起由T细胞和B细胞介导的自身免疫应答。T细胞可引起效应细胞毒性反应，B细胞可分化为浆细胞产生针对自身抗原的特异性抗体，效应细胞和抗体靶向表达自身组织，导致组织损伤。同时，自身蛋白经修饰也可诱导病理性自身抗体产生，诱发自身免疫性疾病，这种自身免疫循环一旦启动，就会造成自身免疫性疾病持续存在。常规治疗方式只是缓解了自身免疫循环，但并未从根源上打破这种循环。 根据上述免疫循环，自身免疫性疾病的潜在致病因素可能是：1）自身抗体的存在；2）与疾病相关的自身反应性淋巴细胞；3）介导免疫耐受的Treg细胞减少或功能缺失。 CAR细胞用于自身免疫系统疾病的治疗策略主要有3种，即CAR-T、嵌合自身抗体受体T细胞（CAAR-T）和嵌合抗原受体调节性T细胞（CAR-Treg）。本文重点阐述CAR-T细胞疗法在自身免疫性疾病中的进展。 系统性红斑狼疮患者B细胞对DNA和核抗原的病理反应先于临床症状，所以临床通常采用B细胞耗竭疗法，免疫抑制剂和激素类药物。 2021年，德国风湿免疫学家Georg Schett团队报道了首例重度系统性红斑狼疮患者接受靶向CD19的CAR-T细胞疗法数据，发现治疗后44天，患者疾病活动指数从16分下降到0分； 2022年他们再次报道了5例难治性系统性红斑狼疮患者的治疗效果，结果显示，所有患者病情均获得改善，狼疮性肾炎被"治愈"； 2024年该团队病例系列研究结果登顶NEJM，结果显示8例系统性红斑狼疮患者在治疗6个月后均达到狼疮低疾病活动状态（LLDAS）标准，并达到系统性红斑狼疮缓解定义（DORIS）缓解，长达29个月的随访显示，所有患者均无疾病活动，整个观察期间，抗DNA抗体消失并保持阴性，补体因子C3水平恢复正常，蛋白尿消失。 根据公开资料显示，已有多款用于治疗系统性红斑狼疮的CAR细胞疗法取得重要进展： 瑞基奥伦赛注射液（靶向CD19的CAR-T细胞疗法）用于治疗中重度难治性系统性红斑狼疮的新药临床试验申请获中国国家药品监督管理局（NMPA）默示许可； GC012F注射液（CD19/BCMA双靶点自体FasTCAR-T细胞疗法）在中国获批临床，拟开发治疗难治性系统性红斑狼疮； RJMty19注射液（CD19-CAR-DNT细胞药）在中国获得临床试验默示许可，拟开发治疗难治性系统性红斑狼疮。 …… 特发性炎性肌病是一组获得性免疫性肌病，以皮肤、肌肉、结缔组织和其他不同组织器官受累的炎性病变，不同类型的特发性炎性肌都有自己的特殊表现。 2024年，Georg Schett团队发表于NEJM的病例系列研究结果显示，3例特发性炎性肌病患者在治疗3个月后，均达到美国风湿病学会-欧洲风湿病防治联合会（ACR-EULAR）明显临床应答，肌酸激酶水平恢复正常，肌肉功能正常。抗合成酶抗体综合征（ASS）是特发性炎性肌病的一种特殊类型，多表现为肌炎、发热、关节炎、技工手等。ASS可激发肺间质病变，病情进展迅速，死亡率高，整体治疗依然比较困难。 Georg Schett团队在The Lancet发表了世界“首例”使用CAR-T细胞疗法“治愈”ASS的案例。这位41岁的男性患者，接受CAR-T细胞疗法6个月后，患者被“治愈”，肌肉病变完全好转，呼吸道症状改善，不再需要高浓度吸氧，肺炎问题也消失了。更令人感到惊喜的是，即使停用了所有免疫抑制药，患者的症状依然在持续好转。 无独有偶，德国图宾根大学医院Claudia Lengerke团队也带来了好消息，发表于JAMA的一篇病例报道显示，1位抗合成酶抗体综合征伴有进行性肌炎和难治性间质性肺部的患者接受靶向CD19的CAR-T细胞治疗后，临床症状快速得到改善，肌肉和肺功能好转。 视神经脊髓炎谱系障碍是免疫介导的中枢神经系统炎性脱髓鞘疾病，通常为急性或亚急性起病，进展迅速，80%~90%为复发病程，核心症状为视神经炎（单眼、双眼相继或同时视力减退）、急性脊髓炎等。B细胞及浆细胞会产生抗水通道蛋白4抗体（AQP4），损伤星形胶质细胞，进而诱导自身免疫相关的脱髓鞘改变。 CAR-T细胞疗法在清除B细胞肿瘤方面的能力，为治疗视神经脊髓炎带来了启示。2023年，华中科技大学同济医学院附属同济医院的王伟教授团队发现，使用BCMA特异性CAR-T细胞疗法治疗12例AQP4抗体相关的视神经脊髓炎谱系障碍患者，中位随访5.5个月后，11例患者疾病未复发。 据公开资料显示，伊基奥仑赛注射液（BCMA靶向CAR-T细胞疗法）拟用于治疗视神经脊髓炎谱系疾病适应证在中国获批临床。 重症肌无力是由自身抗体介导的获得性-肌肉接头传递障碍的自身免疫性疾病，B细胞在重症肌无力病理过程中发挥着重要作用。发表于The Lancet Neurology的MG-001试验显示，16例成人重症肌无力患者接受Descartes-08（靶向BCMA的rCAR-T细胞疗法）治疗12周后，MG-ADL评分、重症肌无力定量评分、综合评估和生活质量评分均显著好转，在长达9个月的随访中，重症肌无力严重程度量表出现有临床意义的降低。基于该研究令人惊喜的结果，有学者在The Lancet Neurology发表了相关病例报道，一位33岁的抗AchR阳性全身性重症肌无力女性，在接受自体CD19的CAR-T细胞疗法治疗2个月后，肌肉力量和疲劳感均得到改善，手臂水平伸直时间增加，在无任何支具的情况下，行走能力也有所改善。 据公开资料显示，已有多款用于治疗重症肌无力的CAR-T细胞疗法取得重要进展： 伊基奥仑赛注射液（BCMA靶向CAR-T细胞疗法）新适应证在中国获批临床，拟用于治疗难治性全身型重症肌无力； KYV-101（自体CD19靶向CAR-T细胞疗法）已获得美国FDA授予的快速通道资格，用于治疗重症肌无力和多发性硬化； …… CAR-T细胞疗法治疗自身免疫性疾病后，预期的无药物缓解时长仍有待进一步确认。部分系统性红斑狼疮患者无病进展观察期延长至2年多，从现有结果来看，这期间大部分患者处于B细胞完全重建状态。因此，部分自身免疫性疾病患者在接受CAR-T细胞疗法后有望获得痊愈。当然，这还需要有更长的随访时间来观察和佐证，因为需要明确新的B细胞产生和成熟的过程中，是否会伴随自身反应性B细胞克隆、自身抗体形式，并因此导致疾病复发。如果患者治疗后多年未复发，将有力推动CAR-T细胞疗法作为一种早期干预策略在自身免疫系统疾病中的临床应用。 NEJM发表的与CAR-T细胞疗法治疗自身免疫性疾病的相关社论也指出，CAR-T细胞疗法治疗自身免疫病的未来将受到疗效、安全性、可接受性等因素的影响，如果长期随访能够证实现有结果，那么对某些难治性疾病患者而言是有益的，如果CAR-T细胞疗法真的具有“耐受性”，那么就有理由将其用于早期和并不那么难治的疾病中。 参考资料 [1]Schett G, Mackensen A, Mougiakakos D. CAR T-cell therapy in autoimmune diseases. Lancet. 2023 Nov 25;402(10416):2034-2044. doi: 10.1016/S0140-6736(23)01126-1. Epub 2023 Sep 22. PMID: 37748491. [2]Müller F, Taubmann J, Bucci L, et al. CD19 CAR T-Cell Therapy in Autoimmune Disease - A Case Series with Follow-up. N Engl J Med. 2024 Feb 22;390(8):687-700. doi: 10.1056/NEJMoa2308917. PMID: 38381673. [3]罗秀娜,任军,贾凌云,等.自身免疫性疾病自身抗原的研究进展[J].中国免疫学杂志, 2023, 39(1):172-177. [4]abian Müller,Sebastian Boeltz,Johannes Knitza,et al. CD19-targeted CAR T cells in refractory antisynthetase syndrome. Published:February 15, 2023DOI:https://doi.org/10.1016/S0140-6736(23)00023-5 [5]Pecher AC, Hensen L, Klein R, et al. CD19-Targeting CAR T Cells for Myositis and Interstitial Lung Disease Associated With Antisynthetase Syndrome. JAMA. 2023 Jun 27;329(24):2154-2162. doi: 10.1001/jama.2023.8753. PMID: 37367976; PMCID: PMC10300719. [6]蒲传强. 特发性炎性肌病 [J] . 中华神经科杂志, 2019, 52(5) : 410-422. DOI: 10.3760/cma.j.issn.1006-7876.2019.05.009. [7]王维治, 王化冰. 视神经脊髓炎谱系疾病 [J] . 中华神经科杂志, 2022, 55(5) : 511-519. DOI: 10.3760/cma.j.cn113694-20220127-00062. [8]Qin C, Tian DS, Zhou LQ, et al. Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders: phase 1 trial interim results. Signal Transduct Target Ther. 2023 Jan 4;8(1):5. doi: 10.1038/s41392-022-01278-3. PMID: 36596762; PMCID: PMC9810610. [9]Granit V, Benatar M, Kurtoglu M, et al. Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study. Lancet Neurol. 2023 Jul;22(7):578-590. doi: 10.1016/S1474-4422(23)00194-1. Erratum in: Lancet Neurol. 2023 Sep;22(9):e10. Erratum in: Lancet Neurol. 2023 Sep;22(9):e10. PMID: 37353278; PMCID: PMC10416207. [10]Haghikia A, Hegelmaier T, Wolleschak D, et al. Anti-CD19 CAR T cells for refractory myasthenia gravis. Lancet Neurol. 2023 Dec;22(12):1104-1105. doi: 10.1016/S1474-4422(23)00375-7. PMID: 37977704. [11] Isaacs JD. CAR T Cells - A New Horizon for Autoimmunity? N Engl J Med. 2024 Feb 22;390(8):758-759. doi: 10.1056/NEJMe2400203. PMID: 38381679 内容来源于网络，如有侵权，请联系删除。

Reported $1.2 billion in preliminary* full-year 2023 global net product sales Submitted sBLA to FDA for VYVGART® Hytrulo for CIDP with priority review voucher (PRV); if approved, launch expected mid-2024 Reported positive data from Phase 2 ARDA study establishing proof-of-concept for empasiprubart in MMN Data from six Phase 2 proof-of-concept trials expected by end of 2024 Nominated four new pipeline candidates with IND filings expected by end of 2025 January 8, 2024, 7:00 AM 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 reported preliminary financial results for the full-year 2023, including global net product sales (inclusive of both VYVGART® and VYVGART Hytrulo), and announced its strategic priorities for 2024. “In 2023, we reached more than 6,000 patients globally, making VYVGART available to gMG patients around the world,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “We continued to invest in and demonstrate the sustainability of our business by successfully launching our subcutaneous VYVGART product, and are poised for continued expansion in gMG and beyond in 2024. argenx is delivering on its promise to transform how the world understands autoimmunity. It is with this commitment in mind that we submitted our sBLA for VYVGART Hytrulo in CIDP and, if approved, expect to launch in the U.S. in mid-2024. We will continue to be aggressive in advancing our pipeline this year and expect to report efgartigimod data from six Phase 2 studies in 2024, and to further develop empasiprubart in MMN. Through sustained investment in our IIP, we expect to see repeat value creation, and plan to submit four new INDs by the end of 2025.” “Two years ago, argenx’s key goal was to launch efgartigimod in the U.S. Today, we have built a formidable global commercial organization with product approvals in over 30 countries worldwide and a vibrant pipeline of promising new therapeutics to address immune-mediated diseases. We have forged important partnerships that support our mission to advance the human understanding of immunology to best benefit patients. argenx is well positioned for sustained growth throughout 2024 and well into the future.” 2024 Strategic Priorities argenx will focus on three strategic priorities in 2024 to drive sustainable long-term growth, including: Reach more patients with VYVGART by building upon its strong commercial foundation to address ongoing unmet patient need, broaden the MG opportunity, and expand into CIDPAdvance its extensive pipeline through new data readouts, creating multiple opportunities to demonstrate transformative clinical benefitLeverage its repeatable innovation engine, driving pipeline growth through its Immunology Innovation Program Reach More Patients with VYVGART VYVGART (efgartigimod alfa fcab) is a first-in-class antibody fragment targeting the neonatal Fc receptor (FcRn) and is now approved in more than 30 countries globally. VYVGART subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) is approved in the U.S. (as VYVGART Hytrulo) and Europe, making VYVGART the only gMG treatment available as both an IV and simple SC injection. argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts. argenx will work to reach patients earlier in the MG treatment paradigm and improve the lives of new MG patient populations through additional global regulatory approvals, and the expansion of uses to treat additional autoimmune indications. Regulatory approval decisions of VYVGART for gMG expected in Switzerland, Australia, Saudi Arabia and South Korea by end of 2024Through strategic collaboration with Zai Lab, VYVGART to be included on China’s 2023 National Reimbursement Drug List (NRDL), starting in January 2024Decision on approval of VYVGART SC for gMG in Japan expected in first quarter of 2024 and in China through Zai Lab by end of 2024Decision on approval of VYVGART for primary immune thrombocytopenia (ITP) in Japan expected in first quarter of 2024Supplemental Biologics License Application (sBLA) submitted to FDA for VYVGART Hytrulo for CIDP with priority review voucher (PRV); if approved, launch expected mid-2024Regulatory submissions of VYVGART SC for CIDP in Japan, Europe, China and Canada expected in 2024Registrational studies to expand VYVGART label into broader MG populations, including in seronegative patients, to start in 2024Update on pre-filled syringe development expected in first half of 2024; ongoing studies to support potential approval in gMG and CIDP in 2024 Advance Current Pipeline through Upcoming Data Readouts argenx continues to demonstrate breadth and depth within its immunology pipeline and is advancing multiple pipeline-in-a-product candidates. With efgartigimod, argenx is solidifying its leadership in FcRn and is on track to be approved or in development in 15 autoimmune indications by 2025. Beyond efgartigimod, argenx is advancing its earlier stage pipeline programs, including empasiprubart (C2 inhibitor) with Phase 2 studies ongoing in multifocal motor neuropathy (MMN), delayed graft function and dermatomyositis (DM). In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024. Today, argenx reported positive clinical data from the first cohort of the Phase 2 ARDA study of empasiprubart, establishing proof-of-concept in MMN. After confirming IVIg dependence, 27 patients were withdrawn from IVIg treatment and randomized 2:1 to either empasiprubart or placebo for 16 weeks. Patients were monitored for clinical deterioration that required IVIg retreatment, which was the main efficacy endpoint of the study. Empasiprubart demonstrated a 91% reduction in the need for IVIg rescue compared to placebo [HR: 0.09 95% CI (0.02; 0.044)]According to the Patient Global Impression of Change scale, 94% (17/18) of empasiprubart-treated patients rated their condition as improved since study start, including 55% (10/18) who were much or very much improved. Of placebo patients, 89% (8/9) worsened or had no change.Empasiprubart demonstrated improvement on all six efficacy measurements compared to baselineSafety and tolerability profile were consistent with Phase 1 resultsCohort 2 is ongoing to determine dose response ahead of a Phase 3 study start argenx is on track to report topline data from five additional proof-of-concept studies in 2024, including: Phase 2 RHO study evaluating efgartigimod in primary Sjogren’s syndrome expected in first half of 2024Phase 2 ALPHA study evaluating efgartigimod in post-COVID-19 postural orthostatic tachycardia syndrome (PC-POTS) expected in first half of 2024Seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM), antisynthetase syndrome (ASyS), and DM) expected in the second half of 2024 Leverage Repeatable Innovation Playbook to Drive Long-Term Pipeline Growth argenx continues to invest in its discovery engine, the Immunology Innovation Program (IIP), to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX-213 targeting FcRn and further solidifying argenx’s leadership in this new class of medicine; ARGX-121 and ARGX-220, which are first-in-class targets broadening argenx’s focus across the immune system; and ARGX-109, targeting IL-6, which plays an important role in inflammation. Preclinical work is ongoing in each candidate and argenx is on track to file four investigational new drug (IND) applications by the end of 2025. Preliminary* Fourth Quarter and Full-Year 2023 Financial Results Today, argenx also announced preliminary* global net VYVGART revenues for the fourth quarter and full-year 2023 of approximately $374 million and $1.2 billion, respectively. As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*. Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion. The projected 2024 cash burn will be approximately $500 million. argenx expects its existing cash, cash equivalents and current financial assets, together with anticipated future product revenues, to fund the Company to profitability. * - The preliminary selected financial results are unaudited, subject to adjustment, and provided as an approximation in advance of the Company’s announcement of complete financial results in February 2024. 42nd Annual J.P. Morgan Healthcare Conference Presentation and Webcast Mr. Van Hauwermeiren will highlight these updates in a corporate presentation at the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, 2024, at 9:00 a.m. PT. The live webcast of the presentation may be accessed under Investors on the argenx website. A replay will be available for 30 days following the presentation. Phase 2 ARDA Study Design The Phase 2 ARDA study is a randomized, double-blinded, placebo-controlled multicenter study to evaluate the safety and tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of two dose regimens of empasiprubart in adults with multifocal motor neuropathy (MMN). The study consists of an IVIg dependency and monitoring period and two 16-week treatment cohorts of 24 MMN patients receiving empasiprubart or placebo in a 2x1 randomization. The dosing for Cohort 2 was established after a planned interim analysis of the first nine patients to complete the 16-week treatment period from Cohort 1. The primary endpoint is safety and tolerability. Additional endpoints include time to IVIg retreatment, biomarker analyses of C2 levels, and changes in measurements on key functional scores (modified medical research council (mMRC)-10 sum score, grip strength, MMN-RODS) as well as several patient-reported quality of life outcome measures (fatigue severity score (FSS), chronic acquired polyneuropathy patient-reported index (CAP-PRI), and values of the patient global impression change (PGIC) scale). About Multifocal Motor Neuropathy Multifocal motor neuropathy (MMN) is a rare, 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 anti-GM1 IgM autoimmunity, 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 Empasiprubart Empasiprubart (ARGX-117) is a first-in-class humanized sweeping antibody that binds specifically to C2 thereby blocking both the classical and lectin pathways of the complement cascade. By blocking upstream complement activity, empasiprubart has the potential to reduce tissue inflammation representing a broad pipeline opportunity across multiple severe autoimmune indications. In addition to multifocal motor neuropathy, argenx is evaluating empasiprubart in delayed graft function following kidney transplant and dermatomyositis. About VYVGART and VYVGART SC VYVGART 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 globally 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). 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. and VYVGART SC in Europe, 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, globally in the U.S., Japan, Israel, the EU, the UK, China and Canada. 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, Twitter, and Instagram. Media: Ben Petokbpetok@argenx.com Investors: Alexandra Roy (US)aroy@argenx.com Lynn Elton (EU)lelton@argenx.com Preliminary Financial Results The financial results presented in this press release are 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, 2023, and full year financial results for 2023 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 2023 financial results and full year financial results for 2023 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 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 “plans,” “aims,” “believes,” “continues,” “hope,” “estimates,” “preliminary,” “anticipates,” “expects,” “intends,” “may,” “will,” “should,” or “commitment” and include statements argenx makes concerning its preliminary financial results for the full year 2023; its expansion efforts, including reaching more patients with VYVGART within the MG treatment paradigm, through geographic expansion and into new autoimmune indications, expanding into CIDP, and the anticipated development of empasiprubart and ARGX-119; the anticipated timing of its launch of SC efgartigimod for CIDP in the U.S.; the initiation, timing, progress and results of its anticipated clinical development, data readouts and regulatory milestones and plans; its strategic priorities, including the timing and outcome of regulatory filings and regulatory approvals; its expectations of future profitability; the potential of its innovative clinical programs; and the nomination of new development candidates. 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 argenx’s ability to successfully execute its business and growth strategies, the inherent uncertainties associated with development of novel drug therapies, preclinical and clinical trial and product development activities and regulatory approval requirements, the acceptance of our products and product candidates by our patients as safe, effective and cost-effective, volatile market conditions, and the impact of governmental laws and regulations on our business. 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.