argenx US, Inc.

Study Estimates Avoidable Costs Due to Delayed Diagnosis at Up To $517,000 Per Patient WASHINGTON--(BUSINESS WIRE)-- In one of the first studies of healthcare resource utilization and costs for patients with rare disease, the EveryLife Foundation for Rare Diseases reveals that timely diagnosis and screening can shorten and possibly eliminate the diagnostic odyssey while significantly reducing the cost impact of rare disease for individuals, families, and the healthcare system. The report, entitled, “The Cost of Delayed Diagnosis in Rare Disease,” provides an in-depth analysis of the avoidable costs associated with seven rare diseases: Adrenoleukodystrophy (ALD) Duchenne muscular dystrophy (DMD) Fragile X syndrome (FXS) Generalized myasthenia gravis (gMG) Pompe disease (PD) Severe combined immunodeficiency disorder (SCID) Wilson disease The study, which was presented at a September 14 briefing hosted by the Rare Disease Congressional Caucus, estimates the avoidable per patient medical costs and productivity losses attributable to delayed diagnosis of those seven rare diseases at between $86,000 and $517,000 per patient. The benefits of timely intervention are especially profound in diseases for which newborn screening has been implemented in some or all U.S. states, such as ALD, Pompe disease, and SCID. For these three rare diseases, timely diagnosis can eliminate the diagnostic odyssey and its associated medical costs and provide the opportunity for optimal intervention and improved health outcomes. “The results of this study demonstrate the urgent need for faster and improved diagnostic strategies to help decrease the financial and personal impact of rare diseases on families and the healthcare system,” commented Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement at the EveryLife Foundation. “Timely diagnosis, using tools such as newborn screening and next-generation, evidence-based neonatal sequencing, is especially important when there are disease-altering or life-saving treatments available that can prevent irreversible disease progression and change outcomes.” A recent white paper by the EveryLife Foundation entitled, "Pioneering the New Era of Newborn Screening” advocates for greater investment in these diagnostic resources that can yield significant economic benefits by reducing or eliminating unnecessary services or procedures while producing clinical and psychosocial benefits. This new study on the Cost of Delayed Diagnosis is a follow-up to the landmark 2021 “National Economic Burden of Rare Disease Study,” which estimated that the economic impact of 379 rare diseases in 2019 was nearly $1 trillion, with 60% of those costs being shouldered directly by families and society. The 2021 study also noted that from the first appearance of symptoms it can take more than six years and as many as 17 clinical encounters before a person receives a definitive rare disease diagnosis. That finding was a major impetus for the new study, which showed that individuals whose diagnoses were delayed by an average of five years were approximately four times as likely to see three or more specialists as those with shorter diagnostic odysseys. “Medical costs for rare diseases are inevitable, but avoidable costs from delayed diagnosis not only place financial strain on individuals and families but also divert crucial healthcare funds. These could be better used for treatments that enhance patient quality of life and boost workforce productivity,” stated Amy Brower, Ph.D., Director of Newborn Screening Translational Research Network at the American College of Medical Genetics and Genomics. “Timely diagnosis can lead to targeted therapy, surveillance for complications, and genetic counseling which can positively affect health outcomes, survival, and the overall healthcare system.” The financial effects of receiving a delayed diagnosis were particularly acute among patients with ALD and DMD, as measured by direct medical costs, a category that includes inpatient hospital or outpatient care, emergency department visits, physician visits, prescription medications, and Medicaid-covered caregiver services. For patients with delayed diagnosis of ALD and DMD, the mean annual direct medical costs per patient were roughly twice as high in the year of diagnosis compared with those who received a timely diagnosis. For individuals with late onset Pompe disease, delayed diagnosis increased mean annual direct medical costs in the year of diagnosis by approximately $50,000 per patient compared with timely diagnosis. The new report also documented the cost-saving benefits of early detection and newborn screening in diseases for which this life-saving intervention has been implemented (i.e., ALD, Pompe disease, and SCID). In the case of ALD, annualized per patient direct medical costs were nearly twice as low in the year of diagnosis and more than 10-fold lower per year post-diagnosis among those diagnosed in the first year of life compared with those diagnosed later. In contrast, in SCID, a disease with high mortality if not detected early, average annualized medical costs per patient in the year of diagnosis were more than two times higher among individuals diagnosed in the first year of life compared with those diagnosed at age 1 or older. “For this study we chose seven diseases for which there is an existing path to diagnosis, but for the vast majority of rare conditions the search for answers goes on and on,” said Kennedy. “Therefore, the costs described in the study are gross underestimates for those who never reach the end of their diagnostic odyssey.” The study also examined the impact of lost productivity related to time used for healthcare encounters on the seven rare diseases. In patients with FXS, for example, these cost categories were similar in the timely and delayed diagnosis groups. However, in FXS, caregiver days of missed work in the year of diagnosis (21.5) and post-diagnosis periods (21.4) were nearly twice as high in the delayed diagnosis group as in the timely diagnosis group (12.8 and 10.1, respectively). Study Methodology The “Cost Benefits of Early Diagnosis and Screening in Rare Disease” report researchers used detailed patient journey maps, based on comprehensive literature review and consultation with a clinical advisory group, to inform their analysis. The researchers conducted a comprehensive literature review to understand disease epidemiology, disease milestones and progression, testing and procedures that can be part of the diagnostic odyssey, and the economic burden of the seven rare diseases covered in the report. In collaboration with the EveryLife Foundation, the research team identified 27 experts representing the research, clinical, and patient advocacy communities to join a technical advisory panel composed of pediatricians, patients, family members, disease advocates, and researchers. The clinical advisory panel reviewed and provided input to refine and finalize the journey maps. In addition, over the course of the project, the research team conducted five panel discussions to spearhead the project direction and to review the interim output and study results. To estimate direct medical costs per patient, the researchers used claims data from two sources: the Transformed Medicaid Statistical Information System (TMSIS) and Optum deidentified Normative Health Information (dNHI), a large, geographically diverse medical and pharmacy claims database for the privately insured. The source population included patients diagnosed with any of the seven rare diseases of interest from 2016 to 2020 (except for individuals with DMD, who were diagnosed from 2018 through 2020). Medical costs were estimated for periods pre-diagnosis, the year of diagnosis, and post-diagnosis. To quantify the cost of the diagnostic journey and delayed diagnosis incurred by a rare disease patient and their caregiver, the researchers estimated the number of work hours missed due to medical appointments multiplied by the mean hourly wage in 2021 dollars from the Bureau of Labor Statistics, and estimated the cumulative productivity losses by multiplying the number of years diagnosis was delayed by the per patient per year productivity loss in the pre-diagnosis period. The researchers also analyzed data from the 2020 Rare Disease Impact Survey, respondents to which represented 1,300 people living with a rare disease, including 601 individuals diagnosed before age 19. Because the sample sizes for the seven diseases of interest were small, the researchers included all respondents with data sufficient to calculate a diagnostic odyssey. In addition, they used the survey data to summarize patient-reported counts of healthcare events before rare disease diagnosis, including number of primary care and specialty physicians seen for rare disease diagnosis, number of emergency department visits and hospital admissions related to rare disease before diagnosis, and number of out-of-state trips related to rare disease diagnosis. Study Contributors The EveryLife Foundation, the study’s sponsor, commissioned the Lewin Group, the research consulting business within Optum Serve, the federal health services business unit of Optum and UnitedHealth Group. The research team estimated the cost benefits of timely diagnosis of the seven rare diseases of interest and assembled the 27-member technical advisory panel. Funding support for the study was secured from Alexion Pharmaceuticals, Amicus Therapeutics, Argenx US, Inc., Chiesi Global Rare Diseases, the Danaher Foundation, Genentech, Invitae, PhRMA, Pfizer Inc., REGENXBIO Inc., Sanofi, and Ultragenyx. Study Limitations The study results are prone to limitations common to the use of administrative claims data, including the incompleteness of claims data. It is therefore possible that the researchers’ estimates of healthcare utilization for patients with conditions requiring certain services (e.g., physical therapy, occupational therapy, speech therapy, other early childhood services), such as FXS, underestimate the true utilization and costs. Perhaps most important for a study of autosomal recessive rare disease, such as Pompe and SCID, or an X-linked disorder such as FXS, DMD, or ALD, is the lack of complete information in the claims about siblings who may have been diagnosed or identified as disease carriers. Claims data typically do not include information about genetic testing and family history. Such information can eliminate the diagnostic journey altogether. Finally, despite access to two large databases, the study was limited by a small sample size. To preserve sample size, the researchers did not exclude outliers, which may have skewed some distributions of costs. However, these outliers represent the real-world experience of patients with rare diseases. For more information about “Cost Benefits of Early Diagnosis and Screening in Rare Disease” study, visit the study website at everylifefoundation.org/delayed-diagnosis-study. About EveryLife Foundation The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures. The Foundation provides training, education, resources and opportunities to make patient voices heard, help change public policy and save lives.

创新药BD高阶研讨会【上海站】！点击这里立刻报名！随着美联储加息预期逐渐走低，且今年几乎可以确定会停止加息，估值处于历史低位的中小市值医药股有望在今年迎来并购潮，结合2022年的并购情况（详见2022年生物医药行业并购盘点），我们认为Argenx、Apellis、Sarepta等十家公司有较大潜力被大药企并购。除了本文列出的十家药企之外，以下医药公司或技术领域值得关注：ADC领域：Mersana Therapeutics、ImmunoGen；CNS领域：Intra-cellular、Neurocrine、Sage Therapeutics;罕见病领域：Mirum、Krystal Biotech；其他领域：Halozyme、Prometheus、Viridian Therapeutics.1、Argenx(NASDAQ:ARGX)ARGX是一家以羊驼源抗体研发见长的荷兰药企，拥有SIMPLE Antibody platform、NHance、ABEDEG和POTELLIGENT三项与抗体Fc区改造相关的专利技术，具有技术平台属性。ARGX已经上市的治疗重症肌无力的药物VYVGART销售不错，仅在去年第三季度录得1.31亿美元的销售收入，预计全年收入达4.02亿美元，VYVGART的中国区权益也被再鼎医药拿下。2023年，ARGX还有一些重要的里程碑事件：公司已经递交了皮下注射版（SC）的efgartigimod，将在6月20日迎来上市审批，考虑到VYVGART已经上市且销售不错，皮下注射版的efgartigimod获批概率很大，销售收入更为可观；SC版的efgartigimod在二季度也将发布治疗慢性炎症性脱髓鞘性多发性神经病（CIDP）的三期数据；在研药物ARGX-113，将在下半年发布治疗原发性免疫性血小板减少症(ITP)和寻常性天疱疮（PV）的两个三期重磅数据。ARGX管线丰富，技术平台也得到了验证，销售收入上升迅速，加上今年众多重磅催化剂的加持，多家华尔街投行将ARGX列为今年看好的生物医药企业之一。2、Apellis(NASDAQ:APLS)APLS是一家补体技术的生物医药公司，主打药物是C3抑制剂APL-2，目前该药物已经获批了两个适应症：阵发性睡眠性血红蛋白尿症（PNH）与继发于年龄相关性黄斑变性的地理萎缩（GA），其中PNH适应症在2021年年中已经获批，去年仅在美国就销售了6510万美元（美国以外授权给了SOBI）。APL-2于近期获批用于治疗GA，拿到的标签比较广（包括foveal和extrafoveal），GA的市场较大，华尔街投行更是给出了50亿美元的巅峰销售额预测，同时投行进行的医生调查表明，医生和患者都对APL-2的期待很高，预计医生将在20-50%的患者中使用该药，估计有70-80%的EOM使用率。除了上述获批适应症外，公司在罕见病、眼科、神经系统、基因疗法等领域均有布局，其中以与SOBI合作的渐冻症（ALS）最为重要。对于想进入补体技术或GA领域的大药企而言，APLS是非常合适的选择。3、Alnylam(NASDAQ:ALNY)Alnylam成立于2002年，是RNAi疗法领域的龙头企业，在过去五年间，成功将5款RNAi药物推向市场，2022年实现销售收入8.92亿美元，同比增了35%。按产品分类来看，适应症相同的ONPATTRO 和 AMVUTTRA，在去年四季度分别录得1.22亿美元和6900万美元的收入，全年收入分别为5.58亿美元和9400万美元；GIVLAARI在去年四季度录得4700万美元的收入，全年实现1.73亿美元的收入；OXLUMO在去年四季度录得2400万美元的收入，全年实现7000万美元的收入。除了现有产品强劲的业绩表现之外，依托RNAi疗法技术平台，ALNY在研管线丰富，且有不少管线处于后期，短期内具有很大的商业化潜力：Patisiran治疗ATTR淀粉样变性心肌病的补充上市申请，审批时间为10月8日；今年还将发布Fitusiran治疗血友病的三期数据以及Vutrisiran治疗ATTR淀粉样变性心肌病的三期数据。此外， 公司一些早期管线的适应症市场极为广阔，例如治疗轻度和中度高血压Zilebesiran、治疗阿尔海默兹症的ALN-APP、治疗糖尿病和NASH的ALN-KHK等。无论是技术平台，还是销售业绩，以及未来潜力，ALNY都是大药企争相并购的标的。4、Axsome Therapeutics(NASDAQ:AXSM)AXSM是近些年中枢神经系统领域Biotech的典范，股价在过去4年上涨了30多倍，也让2018年从Pierre Fabre USA副总裁位置上投奔而来的CFO Nick Pizzie获得了财富自由，而当时AXSM的市值还不到1亿美元。股价上涨的背后，是AXSM接连取得的临床试验成功：AXS-05治疗重度抑郁症（MDD）和阿兹海默躁郁症的三期试验均获得成功，其中MDD适应症已经获批上市；AXS-07治疗偏头痛的三期试验成功，计划今年递交NDA；治疗嗜睡症的药物AXS-12也获得了FDA的突破性疗法认证，目前处于临床二期，今年将发布数据。除了自身研发做的有声有色之外，公司在商务拓展（BD）方面也是长袖善舞：2022年3月，AXSM从JAZZ手中以5300万美元买入了治疗嗜睡症的已上市药物Sunosi，而在今年2月，AXSM宣布将Sunosi的在欧洲以及中东北非的权益授予Pharmanovia，AXSM将获得6600万美元的预付款，以及高达1.01亿美元的里程碑款项。对于青睐商业化阶段的大药企而言，拥有2个CNS在售药物以及多个后期管线的AXSM无疑是最值得考虑的并购标的之一。5、Ascendis(NASDAQ:ASND)Ascendis Pharma是一家长效药物公司，拥有长效药物技术平台TransCon，目前主要是围绕各种罕见病，将用药频次降低，改善患者的依从性，降低副作用，进而提升治疗效果，随着多个药物三期临床试验的成功，ASND的技术平台也得到了验证。公司目前有三个在研药物TransCon hGH（长效生长激素）、TransCon PTH （治疗副甲狀腺功能低下症）和TransCon CNP（治疗软骨发育不良症）。2021年，公司治疗小儿生长激素缺乏症(GHD)的药物Skytrofa已经获批上市，今年4月30日，FDA将对公司用于治疗成年甲状旁腺功能减退症的药物TransCon PTH进行上市审批，年底前还将发布TransCon hGH治疗成人生长激素缺乏症的3期数据。此外，公司还在软骨发育不全症，肿瘤领域有管线推进，今年将提供相关临床试验的更新。ASND已上市的药物Skytrofa业绩增长不错，2022年实现了3570万欧元的收入，季度收入环比增速达39%。2018年，ASND还与Vivo Capital联合创办了维昇药业（VISEN Pharmaceuticals），维昇药业获得了ASND三个药物的大中华区权益。2022年11月，维昇药业向港交所递交了招股书，拟在香港主板挂牌上市，鉴于国内广阔的长效生长激素市场，长春高新、安科生物等公司已经布局，而维昇药业的到来将会加剧行业的竞争。鉴于公司的长效药物技术平台，以及拥有商业化和研发后期的管线，Ascendis Pharma也是大药企潜在的收购标的之一。6、Sarepta(NASDAQ:SRPT)Sarepta Therapeutics是治疗杜氏肌肉营养不良综合症（DMD）领域的领先企业，公司目前有已有3款获批的DMD药物：EXONDYS 51、AMONDYS 45和Vyondys 53，除了已上市的药物，公司围绕DMD，正在开发基因疗法、基因编辑等新的治疗药物，同时在肢带型肌营养不良症(LGMD)等适应症上亦有布局。2022年三季度，公司录得产品销售收入2.07亿美元，预计全年收入可达8.43亿美元，同比增长38%。除了强劲的销售业绩，公司的研发管线也同样值得期待。公司于去年3季度递交了与罗氏联合开发的治疗DMD的基因疗法药物SRP-9001的上市申请，FDA授予其优先审批的认定，审批时间为5月29日。SRP-9001的上市申请得到了多个临床试验数据的支持，获批的概率较大。对于想进入DMD领域的大药企而言，SRPT是最好的选择。不过SRP-9001的海外权益授权给了罗氏，该事件可能会降低并购者的预期。7、Karuna Therapeutics(NASDAQ:KRTX)KRTX是一家开发神经系统疾病治疗药物的公司，其主打药物KarXT是一款口服的M1/M4首选毒蕈碱激动剂，由毒蕈碱激动剂xanomeline和毒蕈碱拮抗剂trospium组成，旨在优先刺激中枢神经系统中的毒蕈碱受体，有潜力实现不依赖多巴胺能或血清素治疗严重精神疾病，包括精神分裂症和阿尔茨海默症中的精神症。2022年8月，公司发布了KarXT在治疗治疗精神分裂症的3期临床数据，不仅达到了试验终点，而且在治疗效果、安全性、患者覆盖面等方面均优于现有疗法，解决了现有疗法中依从性差、覆盖面少的问题，尤其是KarXT的临床P值<0.0001，在CNS领域更是极为少见。此数据已经发布，KarXT被瑞穗等投行视作精神分裂症领域的游戏规则改变者，预计到2028年预计将达到17亿美元，巅峰销售额将在50亿美元以上。公司计划在今年年中递交KarXT的上市申请，并在今年一季度发布另一项名为布EMERGENT-3三期试验结果，该实验和之前的EMERGENT1（Ph2b）和EMERGENT-2（Ph3）研究相似，成功概率很高，但最初该实验主要在乌克兰进行，现将大部分研究转移到美国，存在一定的不确定性。8、Revance(NASDAQ:RVNC)RVNC是一家致力于开发美容医学产品的药物公司，依托独有的TransMTS® 肽技术，开发了有差异化的注射用肉毒素DAXXIFY，目前已于去年三季度获批。与市场上现有的Dysport、Xeomin、Jeuveau等产品不同，DAXXIFY是一款真正意义上具备与肉毒素领域头号产品BOTOX差异化的新产品，在治疗眉间纹方面，DAXXIFY的疗效时间几乎是BOTOX的1.5到2倍，而后者年收入是146亿美元。由于效果显著，DAXXIFY瞄准的是现有医美用户中前三分之一的优质客户，投行预计DAXXIFY将占据美国30%的市场，年销售额将超过10亿美元，而在刚刚获批后的第一个季度（去年四季度），DAXXIFY体验用户的销售额就已经预计在1050万美元至1150万美元之间。除了DAXXIFY，公司还有比玻尿酸副作用更小，效果更好的RHA填充剂，上市后第一年销售收入7000多万美元，去年同比增速保持在40%+。与其他小药企不同的是，公司在商业化方面还有商务平台OPUL™，是一个集合了客户关系维护和金融支付功能的云平台，为各类医美商家提供服务，截至去年三季度的过去十二个月的平台GPV总计超过6.3亿美元。在加息环境下，面临重磅药物专利悬崖的大药企们，很可能会对销售收入潜力大，业绩见效快的RVNC产生并购兴趣。9、Krystal Biotech(NASDAQ:KRYS)KRYS是一家比较独特的基因疗法公司，没有采用常见的腺相关病毒或脂质纳米颗粒来输送遗传物质，而是将基因加载到单纯疱疹病毒（HSV-1）中，使其到达体内的目标。HSV-1比其他基因疗法技术具有更高的承载能力，不仅拓宽了KRYS可能治疗的潜在疾病范围，而且可以实现重复给药。KRYS在其基因疗法技术平台上，开发了一系列针对皮肤病和呼吸系统疾病的药物，其中最为重要的是治疗营养不良性大疱性表皮松解症（DEB）的B-VEC，目前已经处于等待PDUFA阶段。B-VEC已获得美国FDA授予的优先审评资格，因CMC的原因，将审批时间推迟到今年5月19日，获批概率较高。如果获得批准，这将是治疗DEB的首款获批基因疗法。虽然DEB的市场不是很大，但B-VEC一定程度上证明了公司技术平台的可行性，除了目前已经布局的皮肤病和呼吸系统疾病之外，公司还开启了治疗囊性纤维化的临床试验，此外，KRYS的子公司Jeune Aesthestics还在尝试进入医美领域。独特的技术平台价值以及多个有前景的在研管线，KRYS有望成为大药企的并购选项。10、Xenon(NASDAQ:XENE)XENE是一家专注于通过调节离子通道来治疗神经性疾病的公司，主要适应症是癫痫。目前管线中有四个在研药物XEN1101, XEN496, XEN901和XEN007，与CNS领域的知名企业Neurocrine和Alexion（被阿斯利康收购）均有合作。2021年10月，公司宣布主要管线XEN1101宣布治疗成人局部癫痫的临床二期成功，FDA要求公司再做一个三期即可申报上市申请。另外一个治疗婴儿癫痫的XEN496也处于临床三期，也要较大的成功概率。此外，公司药物XEN1101还处于治疗重度抑郁症（MDD）的临床二期，MDD比癫痫的市场更大，如果下半年发布的数据不错，则将增加公司被收购的概率。封面图来源：123rf推荐阅读辉瑞要收购Seagen？华尔街日报：正在谈判！问了CHATGPT亲兄弟「中国药企会面临哪些风险」虽然是车轱辘话，但好像很有道理……16个小时内，3起淘汰，制药江湖惊现达摩克里斯之剑！点击这里，报名创新药BD高阶研讨会【上海站】！

Despite early satisfaction with Vyvgart, half of surveyed neurologists & neuromuscular specialists report uncertainty regarding re-dosing after the first treatment cycle EXTON, Pa., Feb. 24, 2023 /PRNewswire/ -- Argenx's launch of Vyvgart (efgartigimod) has been a success, with global first-year revenue of ~$400 million USD. As the second biologic approved for generalized myasthenia gravis (gMG) and the first neonatal Fc receptor (FcRn) blocker, Vyvgart's novel and targeted mechanism of action has resonated with HCPs. Indeed, neurologists and neuromuscular specialists surveyed by Spherix Global Insights consider Vyvgart to address a substantial unmet in the treatment of this chronic, rare autoimmune condition. Despite these positive perceptions of argenx's brand, surveyed respondents (n=69) remain less confident in Vyvgart's dosing profile. While its infusion administration is familiar and the same as other approved biologics for gMG – Alexion's Soliris and Ultomiris – unlike these brands, it offers somewhat of a flexible dosing schedule. As per the label – and supported by data from its Phase 3 ADAPT trial – Vyvgart is administered as four (4) infusions one (1) week apart, followed by a treatment free period, with additional treatment cycles initiated as needed based on clinical evaluation. Interestingly, even with increased experience among neurologists, half continue to report uncertainty as to when to re-dose patients after the first Vyvgart treatment cycle. Indeed, when probed unaided on Vyvgart's biggest disadvantages, two in five offered its dosing profile as a leading disadvantage. Specifically, respondents reported: "Re-treatment time is left at discretion of reoccurrence of symptoms, which involves a subjective report from the patient." "I get some push back on the frequency of infusions. Not always sure how to know when the next infusion should be." "Uncertainty how dosed after first round." "Expensive, infusions are too frequent and unclear when to re-dose." This finding was further corroborated during qualitative interviews with Vyvgart prescribers, with one neurologist specifically recalling mixed feelings about Vyvgart's flexible dosing: "The hassle with Vyvgart, I mean, now, we're gaining experience, but the flexibility in dosing may be a good thing or a bad thing. I'm just accepting it for what it is. But in time, I may have other feelings about it. I may like it a lot, or I may hate it just because of the close monitoring you would need to do with patients." When put into context, the next question is whether upcoming launches could capitalize on questions around Vyvgart flexible dosing – questions that are likely to linger with the expected approval of argenx's subcutaneous efgartigimod in June 2023, given that it will likely have similar flexible dosing. Within the FcRn blocker class, UCB's rozanolixizumab is first up with a PDUFA date also in Q2 2023; other potential upcoming launches in the class, while further out, include Janssen's nipocalimab. Outside of this class, other contenders that could threaten argenx's gMG franchise include Genentech's Enspryng (satralizumab) and Horizon's Uplinza (inebilizumab), both of which are already approved for NMOSD and currently in late-phase development for gMG. Neurologists' early view on the pipeline reveals that Enspryng and Uplinza are already of high interest, largely due to their dosing frequencies, distinct mechanisms of action, and potential efficacy. Spherix will track the evolution of the gMG market through multiple services in 2023. With the expected launches of subcutaneous efgartigimod and rozanolixizumab, Spherix will track the impact of these brands in the US gMG market through its Launch Dynamix™ service. Additionally, insights on inline and pipeline assets, as well as remaining unmet needs in gMG, will be assessed in both the US and EU through research with general neurologists and specialists in the Market Dynamix™ service. The inaugural RealWorld Dynamix™ service will explore the gMG patient journey, including line of therapy, drivers of brand choice, potential next switches, and candidate patient profiles for emerging therapies. Spherix's 2023 gMG Publication Plan: Click here to view the 2023 gMG publication plan. About Launch Dynamix™ Launch Dynamix ™ is an independent service providing monthly benchmarking of newly launched products for the first eighteen months of commercial availability, augmented by a quarterly deep dive into promotional activity, barriers to uptake, and patient types gravitating to the launch brand. About Market Dynamix™ Market Dynamix ™ is an independent service providing analysis of markets anticipated to experience a paradigm shift within the next three to five years. Insights highlight market size, current treatment approaches, unmet needs, and expert opinions on the likely disruption introduced by pipeline agents. About RealWorld Dynamix™ RealWorld Dynamix ™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the "why" behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists' attitudinal & demographic data to highlight differences between stated and actual treatment patterns. About Spherix Global Insights Spherix Global Insights is a leading provider of market research, business intelligence and advisory services to the global life sciences industry. The company's unique service offerings are powered by deep therapeutic knowledge, the Spherix Network specialty physician panel, and commercially relevant analyses to enable strategic decision-making by our valued customers. A trusted advisor and industry thought leader, Spherix Global Insights provides specialized market expertise in six (6) focused therapeutic areas including: dermatology, gastroenterology, nephrology, neurology, rheumatology and ophthalmology. To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect through LinkedIn and Twitter. Spherix Global Insights Contacts Emma McFadden, Senior Insights Director, Neurology [email protected] Scott Upham, Corporate Communications [email protected] NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight's analysis and do not imply a relationship with or endorsement by any company, brand, or product aforementioned. SOURCE Spherix Global Insights