argenx SE (ARGX) Q1 2024 Earnings Call Transcript
argenx SE (ARGX) 2024 年第一季度收益电话会议实录

argenx SE (NASDAQ:ARGX) Q1 2024 Earnings Conference Call May 9, 2024 8:30 AM ET
argenx SE（纳斯达克股票代码：ARGX）2024 年第一季度收益电话会议 美国东部时间 2024 年 5 月 9 日上午 8:30

Company Participants 公司参与者

Beth DelGiacco - Vice President, Global Head of Corporate Communications and Investor Relations
贝丝-德尔贾科（Beth DelGiacco）- 副总裁兼企业传播与投资者关系全球负责人
Tim Van Hauwermeiren - Chief Executive Officer
Tim Van Hauwermeiren - 首席执行官
Karl Gubitz - Chief Financial Officer
卡尔-古比茨 - 首席财务官
Karen Massey - Chief Operating Officer
卡伦-梅西 - 首席运营官

Conference Call Participants
电话会议与会者

Tazeen Ahmad - Bank of America Securities
Tazeen Ahmad - 美银证券
Rajan Sharma - Goldman Sachs
Rajan Sharma - 高盛集团
Derek Archila - Wells Fargo
Derek Archila - 富国银行
James Gordon - JPMorgan
詹姆斯-戈登 - 摩根大通
Akash Tewari - Jefferies
Akash Tewari - 杰富瑞
Yaron Werber - TD Cowen
Brian Conley - Leerink Partners
Alex Thompson - Stifel
亚历克斯-汤普森 - Stifel
Xian Deng - UBS
Xian Deng - 瑞银
Vikram Purohit - Morgan Stanley
Vikram Purohit - 摩根士丹利
Danielle Brill - Raymond James
Samantha Semenkow - Citi
Samantha Semenkow - 花旗银行
Gavin Clark-Gartner - Evercore ISI
Yatin Suneja - Guggenheim
Yatin Suneja - 古根海姆博物馆
Victor Floch - BNP Paribas
Victor Floch - 法国巴黎银行
Joel Beatty - Baird
乔尔-比蒂 - 博德
Manos Mastorakis - Deutsche Bank
Manos Mastorakis - 德意志银行
Matt Phipps - William Blair
Douglas Tsao - H. C. Wainwright

Operator 操作员

Good morning. My name is Rob and I will be your conference operator today. I would like to welcome everyone to the call. At this time, all lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question-and-answer session. [Operator Instructions] Thank you.
早上好我叫罗布，是今天会议的接线员。欢迎大家参加电话会议。现在，所有线路都已静音，以防止任何背景噪音。发言人发言结束后，将进行问答环节。[谢谢。

I'd like to introduce Beth DelGiacco, Vice President, Global Head of Corporate Communications and Investor Relations. You may now begin your conference.
下面有请贝丝-德尔贾科（Beth DelGiacco），她是公司副总裁，企业传播和投资者关系全球负责人。现在可以开始会议了。

Beth DelGiacco 贝丝-德尔贾科

Thank you. A press release was issued earlier today with our first quarter financial results and recent business update. This can be found on our website along with the presentation for today's webcast.
谢谢。今天早些时候发布了一份新闻稿，介绍了我们第一季度的财务业绩和最近的业务更新。您可以在我们的网站上找到这份新闻稿以及今天网络直播的演示文稿。

Before we begin, I'd like to remind you on Slide 2 that forward-looking statements may be presented during this call. These may include statements about our future expectations, clinical development, regulatory time lines, the potential success of our product candidates, financial projections and upcoming milestones.
在开始之前，我想在幻灯片 2 中提醒大家，本次电话会议可能会出现前瞻性陈述。其中可能包括有关我们的未来预期、临床开发、监管时间表、候选产品的潜在成功、财务预测和即将到来的里程碑的陈述。

Actual results may differ materially from those indicated by these statements. Argenx is not under any obligation to update statements regarding the future or to conform those statements in relation to actual results unless required by law.
实际结果可能与这些声明所显示的结果存在实质性差异。除非法律要求，否则 Argenx 没有义务更新有关未来的声明或使这些声明与实际结果相符。

I'm joined on the call today by Tim Van Hauwermeiren, Chief Executive Officer; Karl Gubitz, Chief Financial Officer; and Karen Massey, Chief Operating Officer.
今天和我一起参加电话会议的还有首席执行官蒂姆-范豪韦梅伦（Tim Van Hauwermeiren）、首席财务官卡尔-古比茨（Karl Gubitz）和首席运营官凯伦-梅西（Karen Massey）。

I'll now turn the call to Tim.
现在请蒂姆发言。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Thank you, Beth, and welcome, everyone. At the beginning of the year, we shared an ambitious plan to maximize patient impact over time through our three innovation horizons. First, bringing VYVGART to more patients by employing a multi-dimensional launch strategy.
谢谢你，贝丝，欢迎大家。今年年初，我们分享了一项雄心勃勃的计划，即通过我们的三个创新领域，在一段时间内最大限度地提高对患者的影响。首先，通过采用多维度的发布战略，将 VYVGART 带给更多患者。

Second, advancing our clinical pipeline including empasiprubart and ARGX-119, which have potential across multiple indications with high unmet need. And third, leveraging our IIP to bring forward the next wave of novel targets to the clinic. Today, I'm pleased to share that our execution over the quarter puts us perfectly on track with this plan.
第二，推进我们的临床管线，包括empasiprubart和ARGX-119，它们在多个适应症上都有潜力，但需求尚未得到满足。第三，利用我们的 IIP，将下一波新靶点推向临床。今天，我很高兴地与大家分享，我们在本季度的执行情况使我们完全步入了这一计划的正轨。

Slide 4, let's dive into some of our recent accomplishments beginning with the team's success in delivering another quarter of solid revenue growth. We now have 7,500 patients on VYVGART and VYVGART subcu globally, which does not include China with over 2,700 patients started on therapy in the first quarter alone.
第 4 张幻灯片，让我们深入了解一下我们最近取得的一些成就，首先是我们的团队又成功实现了一个季度的收入稳健增长。目前，我们在全球有 7500 名患者在使用 VYVGART 和 VYVGART subcu，这还不包括中国，仅第一季度就有 2700 多名患者开始接受治疗。

This means we surpassed the 10,000 patient mark and we continue to reach new patients and prescribers each quarter, gaining market share among all gMG treatments. We have two key drivers of revenue growth in the first quarter.
这意味着我们的患者人数突破了 10,000 人大关，而且我们每个季度都在继续接触新的患者和处方者，在所有 gMG 治疗方法中不断扩大市场份额。我们在第一季度的收入增长有两个主要驱动因素。

First, we saw a 34% increase in patients on the VYVGART subcu in the US. The majority of these patients were naive to VYVGART, so the subcutaneous product is expanding the market in the way we planned. Second, we saw 46% growth in patients on treatment in Europe, driven by strong demand in Germany and new launches in Italy and Spain.
首先，在美国使用 VYVGART 皮下产品的患者增加了 34%。这些患者大多对 VYVGART 一无所知，因此皮下注射产品正在按照我们的计划扩大市场。其次，在德国的强劲需求以及意大利和西班牙新药上市的推动下，欧洲接受治疗的患者人数增长了 46%。

And while it's still early days in the ITP launch in Japan, we are pleased to see patients already start therapy in our second indication. Looking ahead to next month and the expected FDA decision for CIDP, we are making the right choices today with our gMG growth strategy that should serve us well for a CIDP launch in the scenario of an approval.
虽然 ITP 在日本的上市还为时尚早，但我们很高兴看到患者已经开始接受我们第二个适应症的治疗。展望下个月以及 FDA 对 CIDP 的预期决定，我们今天正在通过我们的 gMG 增长战略做出正确的选择，在 CIDP 获批上市的情况下，这将为我们提供良好的服务。

We shared at the beginning of this year that advancing our pre-filled syringe in both gMG and CIDP is a top priority for us and we have a positive update for you today. We have successfully collected all necessary data points from our bioequivalents and human factor studies and are on track to file with the FDA by the end of June.
我们在今年年初曾表示，推进预灌封注射器在 gMG 和 CIDP 领域的应用是我们的首要任务。我们已经成功地从生物等效性和人体因素研究中收集到了所有必要的数据点，并有望在 6 月底之前向 FDA 提交申请。

Our goal is to have the broadest product offering available to patients recognizing that different patients and prescribers have different treatment preferences. With the pre-filled syringe, we continue to innovate on the patient experience and will seek self-administration in the label, which we expect will help us reach patients earlier in the treatment paradigm.
我们的目标是为患者提供最广泛的产品，因为不同的患者和处方者有不同的治疗偏好。有了预灌封注射器，我们将继续在患者体验方面进行创新，并将在标签中标明自行给药，我们希望这将有助于我们在治疗模式中更早地接触到患者。

On the clinical front, we made important progress in advancing our next set of indications to Phase 3 and now have studies underway in powered eye disease and the anti-acetylcholine receptor antibody negative gMG population.
在临床方面，我们在推动下一组适应症进入 3 期方面取得了重要进展，目前正在进行动力眼病和抗乙酰胆碱受体抗体阴性 gMG 群体的研究。

The seronegative study is designed to enable a label expansion into 15% of the broader gMG population beyond those patients we can serve today. We are also advancing Phase 3 plans for efgartigimod in Sjogren's and empasiprubart in MMN based on Phase 2 results in both indications.
血清阴性研究的目的是将我们目前可以服务的患者扩大到 15%的更广泛的 gMG 患者。我们还在推进依加替莫德（efgartigimod）治疗 Sjogren 病和 empasiprubart（empasiprubart）治疗 MMN 的 3 期研究计划，这两个适应症的 2 期研究均取得了成果。

And we still have additional data readouts ahead this year in PC-POTS and three subtypes of myositis which all could jump start registrational studies depending on the outcome. The development of our earlier pipeline programs all remain on track including patient studies of ARGX-119 and our upcoming INDs across four molecules as we rapidly work to advance the next wave of novel targets.
今年，我们还将在 PC-POTS 和三种亚型肌炎方面获得更多数据，根据结果，这些项目都可能启动注册研究。在我们快速推进下一波新靶点的过程中，我们早期管线项目的开发仍在按部就班地进行，包括 ARGX-119 的患者研究和我们即将获得的四个分子的 IND。

Slide 5. Last month, we presented important data to the neurologist community during AAN, furthering our confidence in the opportunity we have with VYVGART. In the absence of a cure, the best we can achieve for patients is deep and sustained functional improvement, not just managing symptoms, but getting to the heart of the disease to deliver a better outcome than patients have with current treatments.
幻灯片 5。上个月，我们在 AAN 期间向神经科医生社区提交了重要数据，进一步增强了我们对 VYVGART 所带来机遇的信心。在无法治愈的情况下，我们能为患者实现的最好结果就是深度和持续的功能改善，而不仅仅是控制症状，而是直击疾病的核心，为患者带来比现有治疗更好的疗效。

In gMG, this is minimum symptom expression or MSE and we demonstrate that across studies and various dosing regimens, approximately 50% of patients are able to achieve MSE. This comes without compromising safety and in fact we show that patients can meaningfully take steroids post graft treatment, reducing treatment burden, and improving the overall experience.
在 gMG 中，这就是最低症状表现或 MSE，我们证明，在各项研究和各种给药方案中，约有 50% 的患者能够达到 MSE。事实上，我们的研究表明，患者可以在移植治疗后服用类固醇，从而减轻了治疗负担，改善了整体体验。

We also presented new ADHERE data at AAN specifically on functional improvement showing that some CIDP patients were able to improve more than three or four points on INCAT, which to put it into perspective can mean the difference for a patient between being wheelchair bound and walking without support.
我们还在 AAN 上展示了新的 ADHERE 数据，特别是关于功能改善的数据，显示一些 CIDP 患者在 INCAT 上的改善超过了 3 或 4 分。

Slide 6. During the first quarter, we announced our decision to advance efgartigimod to Phase 3 in Sjogren's disease following the outcome of the single finding RHO study. We are confident in moving forward based on the consistency of data across clinical and biomarker endpoints.
幻灯片 6.在第一季度，我们宣布了一项决定，即在RHO单一发现研究取得结果后，将依加替莫德推进到治疗Sjogren病的第三阶段。基于临床和生物标志物终点数据的一致性，我们有信心继续向前推进。

This was a relatively small trial, just 34 patients, but one of it could look patient by patient at how these endpoints moved together. We had two objectives with the RHO study. First, to gain confidence to invest in further development and second, to thoughtfully shape the Phase 3 study.
这是一项规模相对较小的试验，只有 34 名患者，但其中一项试验可以逐个观察患者的这些终点是如何一起移动的。我们进行 RHO 研究有两个目的。首先，为进一步开发投资赢得信心；其次，深思熟虑地制定 3 期研究计划。

We achieved both and see a clear opportunity ahead for VYVGART card in these disease where there is significant unmet need, specifically in those patients with moderate to severe systemic disease who can experience dry eyes and mouth, fatigue, joint pain, and even organ damage.
我们实现了这两项目标，并看到了 VYVGART 卡在这些疾病领域的巨大商机，因为这些疾病的需求尚未得到满足，特别是中重度全身性疾病患者，他们可能会出现眼干、口干、疲劳、关节疼痛甚至器官损伤。

Slide 7. We are leading this new field of medicine with FcRn, and at the end of last year, we made a commitment to apply key learnings from the ADDRESS and ADVANCE subcu trials to all ongoing and proposed indications.
幻灯片 7。去年年底，我们承诺将从 ADDRESS 和 ADVANCE subcu 试验中获得的关键经验应用于所有正在进行的和拟议的适应症。

The first trial in focus was the BALLAD study of efgartigimod in bullous pemphigoid. We stopped enrollment in the Phase 2 and are currently waiting for data to mature across all patients. We will be ready to communicate a path forward this year whether to change the study design and the normal Phase 2, advance to Phase 3 or stop development in BP altogether.
第一项重点试验是依加替莫德治疗大疱性丘疹性荨麻疹的 BALLAD 研究。我们停止了 2 期研究的招募，目前正在等待所有患者的数据成熟。我们将在今年就是否改变研究设计和正常的 2 期试验、进入 3 期试验或完全停止 BP 的开发进行沟通。

We also completed a thorough risk assessment of all of the programs across efgartigimod and Empa, recognizing the need to be disciplined in where we invest our capital and time. Based on our revaluation, we have decided to discontinue development in ANCA-associated vasculitis or AAV and to focus instead on a newly nominated indication, systemic scleroderma.
我们还对依加替莫德和 Empa 的所有项目进行了全面的风险评估，认识到我们需要严格控制资金和时间的投入。根据我们的重新评估，我们决定终止 ANCA 相关性血管炎或 AAV 的开发，转而专注于新提名的适应症--系统性硬皮病。

We determined the risk did not outweigh the benefit in AAV given the potentially unmanageable interference of background medication. All other indications are advancing forward with trials underway in MN, LN and AMR for efgartigimod and DGF and DM for empasiprubart. We have plans to nominate additional indications for both assets later this year.
考虑到背景药物可能会造成难以控制的干扰，我们认为 AAV 的风险并不比收益大。依加替莫德在MN、LN和AMR的试验，以及empasiprubart在DGF和DM的试验都在进行中。我们计划在今年晚些时候为这两项资产提名更多适应症。

Our opportunity to transform autoimmunity remains strong. The more data we generate in the clinic and translationally, the more informed we can get in our R&D investments and selecting indications where we can win.
我们改变自身免疫的机会仍然很大。我们在临床和转化方面获得的数据越多，我们在研发投资和选择适应症时就越有把握。

This is the best formula for long-term value creation and is a perfect transition to Karl to talk about our financials.
这是创造长期价值的最佳公式，也是卡尔谈论我们的财务状况的完美过渡。

Karl Gubitz 卡尔-古比茨

Thank you, Tim. Slide 8. The first quarter 2024 financial results are detailed in the press release of this morning. I will highlight the key points here.
谢谢你，蒂姆。幻灯片 8.2024 年第一季度的财务业绩详见今天上午的新闻稿。我将在此重点介绍其中的要点。

Total operating income in the first quarter totaled $413 million. This reflects $398 million in product net sales and $14 million in other income and collaboration revenue, including $2 million in royalty income from Zai Lab for VYVGART sales in China.
第一季度的总营业收入为 4.13 亿美元。这反映了 3.98 亿美元的产品净销售额和 1400 万美元的其他收入与合作收入，其中包括来自 Zai Lab 在中国销售 VYVGART 的 200 万美元特许权使用费收入。

Product net sales of $398 million represents 83% growth plus $180 million compared to the same period in 2023. Here is a regional breakdown along with key drivers. $347 million in the US with notable expansion of patients on VYVGART Hytrulo. $18 million in Japan indicating strong volume growth offset by a recent 8% price decrease.
产品净销售额为 3.98 亿美元，与 2023 年同期相比，增长了 83%，增加了 1.8 亿美元。以下是地区细分和主要驱动因素。美国 3.47 亿美元，使用 VYVGART Hytrulo 的患者显著增加。在日本，1,800 万美元的销售额显示出强劲的增长势头，但最近 8% 的降价幅度抵消了这一增长。

EMEA had an excellent quarter with net product revenues of $31 million. The majority of sales still come from Germany, where we had strong volume growth offset by higher accruals due to the planned reassessment of a German price, which will be finalized in 1Q 2025.
欧洲、中东和非洲地区本季度表现出色，产品净收入达到 3100 万美元。大部分销售额仍然来自德国，我们在德国的销售量增长强劲，但由于计划对德国价格进行重新评估（将于 2025 年第 1 季度最终确定），导致应计费用增加，从而抵消了销售额的增长。

We saw meaningful revenue contributions this quarter from Italy and Spain and these launches are just ramping up. We also saw our patient reach expand in Eastern European countries like Poland and ex-EU countries like Saudi Arabia and Switzerland through named patient sales.
本季度，我们看到意大利和西班牙为我们带来了可观的收入贡献，而且这些产品的投放正在加速。我们还通过指定患者销售，扩大了我们在波兰等东欧国家以及沙特阿拉伯和瑞士等欧盟以外国家的患者覆盖范围。

We expect these sales to be an important source of growth going forward as we finalize pricing and reimbursement discussions. We also had $2 million in product net sales to Zai Lab for the launch in China.
随着定价和报销讨论的最终确定，我们预计这些销售额将成为未来增长的重要来源。此外，我们还向 Zai Lab 提供了 200 万美元的产品净销售额，用于在中国上市。

Slide 9, operating expenses in Q1 were $506 million, a decrease of $51 million compared with Q4 2023. Excluding the $102 million impact of a priority review voucher in Q4 2023, operating expenses increased by $51 million. The increased expenses reflect our continued conviction in the long-term opportunity we have for value creation.
幻灯片 9，第一季度的运营支出为 5.06 亿美元，与 2023 年第四季度相比减少了 5100 万美元。剔除 2023 年第四季度优先审查券带来的 1.02 亿美元的影响，运营支出增加了 5100 万美元。支出的增加反映了我们对创造价值的长期机遇的持续信念。

SG&A expenses were $236 million in Q1, which is an increase of $27 million compared to Q4 2023 due to incremental investment in the commercial infrastructure, most notably expanding our customer facing organizations in the US to capitalize on the gMG opportunity and prepare for potential CIDP approval. R&D expenses for the first quarter were $225 million.
第一季度的 SG&A 费用为 2.36 亿美元，与 2023 年第四季度相比增加了 2700 万美元，原因是对商业基础设施的投资增加，其中最主要的是扩大了我们在美国的客户服务机构，以抓住 gMG 的机遇，并为潜在的 CIDP 批准做好准备。第一季度的研发费用为 2.25 亿美元。

Excluding the impact of the PRV from the fourth quarter, we had an increase in the underlying spend of $21 million. This increase reflects our continued investment in our pipeline and we currently have 48 clinical trials across 19 indications and 3 pipeline candidates.
如果不考虑第四季度 PRV 的影响，我们的基本支出增加了 2100 万美元。这一增长反映了我们对产品线的持续投资，我们目前有 48 项临床试验，涉及 19 个适应症和 3 个候选产品线。

Net cash burn for the first quarter was $75 million. We continue to have a strong balance sheet with $3.1 billion in cash, cash equivalents and current financial assets. Our finance guidance for 2024 remains unchanged.
第一季度的净现金消耗为 7500 万美元。我们的资产负债表依然强劲，现金、现金等价物和流动金融资产达 31 亿美元。我们 2024 年的融资指导保持不变。

I will now turn the call over to Karen, who will provide details on the commercial front.
现在我把电话交给凯伦，她将提供商业方面的详细情况。

Karen Massey 卡伦-梅西

Thank you, Karl. Slide 10. I'm proud of the continued momentum of the VYVGART launch, expanding our impact to give more gMG patients the opportunity to return to the activities they love and preparing to do the same to the CIDP patient community ahead of our June PDUFA date.
谢谢你，卡尔。幻灯片 10.我对 VYVGART 推出的持续势头感到自豪，我们扩大了影响，让更多的 gMG 患者有机会重返他们喜爱的活动，并准备在 6 月份的 PDUFA 日期之前为 CIDP 患者社区提供同样的服务。

Before I get into details on the quarter, I want to highlight two things. First, we are changing the gMG treatment paradigm with VYVGART. Our ambition is to enable patients to live without the constant reminder of their disease and we can achieve this with VYVGART in a majority of patients.
在详细介绍本季度的情况之前，我想强调两点。首先，我们正在通过 VYVGART 改变 gMG 治疗模式。我们的目标是让患者能够在没有疾病持续提醒的情况下生活，而通过 VYVGART，我们可以让大多数患者实现这一目标。

We now have extensive real world evidence and clinical trial data extending out beyond nine treatment cycles. That data consistently show approximately 50% of patients are able to achieve MSE or minimum symptom expression.
现在，我们已经掌握了大量的实际证据和临床试验数据，这些数据已经超过了九个治疗周期。这些数据一致显示，约 50% 的患者能够达到 MSE 或最低症状表达水平。

This translates into a very strong value proposition because patients report quality of life measures that are comparable to a healthy population. This is what paradigm changing means for patients, for physicians and for the societies in which we operate.
这可以转化为非常有力的价值主张，因为患者报告的生活质量可与健康人群媲美。这就是改变模式对患者、医生和我们所在社会的意义所在。

Second, we are making decisions today that will benefit us for the CIDP launch and beyond. We are planning for the long-term to sustain growth and we want to ensure that everything we do today can be leveraged to support that growth. We believe we have the right strategy in place, and now with our expanded customer facing team, we will be even better placed to reach new patients.
其次，我们今天做出的决定将有利于我们推出 CIDP 及其后的发展。我们正在进行长期规划，以保持增长，我们希望确保我们今天所做的一切都能为增长提供支持。我们相信我们已经制定了正确的战略，现在我们扩大了面向客户的团队，我们将能够更好地帮助新患者。

Slide 11. Our launch momentum continues with nine consecutive quarters of revenue growth. We have year-over-year revenue growth of 83% and we see consistent growth across every region with more than 10,000 patients on treatment globally. This is an incredible achievement and we are very happy with the momentum we continue to generate from our launch strategy.
幻灯片 11.我们的上市势头在继续，收入连续九个季度实现增长。我们的收入同比增长 83%，而且我们看到每个地区都在持续增长，全球接受治疗的患者超过 10,000 人。这是一个令人难以置信的成就，我们对我们的上市战略所带来的持续发展势头感到非常高兴。

Even with this sustained growth, we are still at the beginning of what we want to achieve and we are broadening our patient impact through our multidimensional expansion strategy. First, we want to reach patients earlier in the treatment paradigm by innovating on the patient experience with formats like the pre-filled syringe. Second, we wanted to expand our reach by seeking regulatory approval in new geographies. And third, we want to expand our label with new indications.
即使实现了持续增长，我们仍处于起步阶段，我们正通过多维扩张战略扩大对患者的影响。首先，我们希望通过预灌封注射器等形式的患者体验创新，在治疗模式中更早地接触患者。其次，我们希望通过在新的地区寻求监管批准来扩大我们的影响力。第三，我们希望通过新的适应症来扩大我们的标签。

Slide 12. In the US, VYVGART Hytrulo was a key driver of our growth this quarter. So let's start there because it's also an important strategy of how we will leverage momentum for the CIDP launch. As of January 1st, we had payor policies and a dedicated J-Code in place and we saw a strong uptake of Hytrulo over the quarter with 34% growth from Q4 in patients on our subcutaneous product.
幻灯片 12.在美国，VYVGART Hytrulo 是我们本季度增长的主要驱动力。因此，让我们从这里开始，因为这也是我们如何利用势头推出 CIDP 的重要战略。截至 1 月 1 日，我们已经制定了支付方政策和专用的 J 代码，本季度我们看到了对 Hytrulo 的强劲吸收，使用我们皮下注射产品的患者人数比第四季度增长了 34%。

The majority of these patients are brand new to the VYVGART franchise, which reflects the opportunity we have to expand within our gMG addressable market with VYVGART Hytrulo.
这些患者中的大多数都是 VYVGART 特许经营的新患者，这反映出我们有机会通过 VYVGART Hytrulo 来扩大我们的 gMG 可寻址市场。

With the backdrop of new innovations coming to market, our total market share across IV and subcutaneous increased over the quarter and we continue to expand the breadth of our prescriber base to now 2,700 neurologists using VYVGART or VYVGART Hytrulo in the US.
在新创新产品不断上市的背景下，我们在静脉注射和皮下注射领域的市场份额在本季度有所增长，而且我们还在继续扩大我们的处方客户群，目前在美国使用 VYVGART 或 VYVGART Hytrulo 的神经科医生已达 2700 名。

The first quarter of the year is notoriously challenging and we were not immune to the impact of recertification, holidays and weather. Taking the seasonality into consideration, I'm very pleased with our performance. The underlying fundamentals of our business is strong and have confidence that we are well positioned to maintain our growth momentum in gMG as we look ahead to the additional drivers this year.
每年的第一季度都充满了挑战，我们也不能幸免于重新认证、假期和天气的影响。考虑到季节性因素，我对我们的业绩表现非常满意。我们业务的基本面非常强劲，我们有信心，在展望今年的其他驱动因素时，我们完全有能力保持全球移动通信集团的增长势头。

Slide 13. The contribution from our ex-U.S markets was another key driver in the quarter, and we saw 46% quarter-over-quarter growth in patients on therapy in Europe specifically. We are encouraged to see this growth materialize. It's consistent with our expectations that Europe will represent an increasingly larger proportion of the opportunity over time.
幻灯片 13.美国以外市场的贡献是本季度的另一个主要驱动因素，我们看到欧洲接受治疗的患者人数较上季度增长了 46%。看到这一增长，我们深受鼓舞。这与我们的预期一致，即随着时间的推移，欧洲市场将占据越来越大的份额。

The volume growth can be attributed to meaningful uptake in Italy and Spain following pricing and reimbursement negotiations in those countries, but also the impact of the approval and launch of VYVGART subcutaneous. Whereas in the US, the strategy with subcutaneous is market expansion. In Europe, we see both the patient switch and the market expansion strategy. This is reflected in the growth this quarter.
销售量的增长可归因于意大利和西班牙在进行定价和报销谈判后出现了可观的增长，同时也受到 VYVGART 皮下注射剂获批和上市的影响。而在美国，皮下注射的战略是扩大市场。在欧洲，我们既看到了患者转换，也看到了市场扩张战略。这也反映在本季度的增长中。

Moving to Japan, it has been a very busy quarter. In addition to delivering continued growth in gMG and securing approval for VYVGART subcutaneous, we received the first global approval for our second indication ITP.
说到日本，这是一个非常繁忙的季度。除了 gMG 持续增长和 VYVGART 皮下注射获得批准外，我们的第二个适应症 ITP 也首次获得全球批准。

This is an important milestone for VYVGART and an important moment for ITP patients where there is a clear unmet need in the market. This was reflected in how quickly after approval ITP patients in Japan began treatment on VYVGART.
这是 VYVGART 的一个重要里程碑，也是 ITP 患者的一个重要时刻，因为市场上的需求显然还没有得到满足。日本的 ITP 患者在 VYVGART 获批后很快就开始接受治疗，就反映了这一点。

And finally in China, through our partner, Zai Lab, we reached an additional 2,700 patients in the first quarter alone, driven by VYVGART's inclusion on the NRDL. Demand has been very strong. You can see that our global launch efforts are steadily progressing. We are expanding our reach into new countries and new indications and expect to further accelerate growth as more opportunities come online.
最后，在中国，通过我们的合作伙伴--Zai Lab，在 VYVGART 被纳入 NRDL 的推动下，仅在第一季度，我们的患者人数就增加了 2,700 人。需求非常强劲。您可以看到，我们的全球上市工作正在稳步推进。我们正在将业务拓展到新的国家和新的适应症，随着更多机会的到来，我们有望进一步加快增长速度。

We are on track to receive VYVGART decisions on approval this year in Australia, Switzerland, Saudi Arabia and South Korea and with VYVGART subcutaneous in China. And we have filed our CIDP regulatory submissions in Japan and China with the EU to follow.
今年，我们有望在澳大利亚、瑞士、沙特阿拉伯和韩国获得 VYVGART 批准决定，并在中国获得 VYVGART 皮下注射批准。我们已经向日本和中国提交了 CIDP 的监管申请，随后将向欧盟提交申请。

Slide 14. Moving to indication expansion with CIDP. We expect a decision on approval in the US next month and preparations are well underway to expand our commercial engine to support this launch. CIDP patients continue to face a significant burden despite the availability of current treatment. The unmet need is high with patients failing to see meaningful innovation in the last 30 years.
幻灯片 14.扩大 CIDP 适应症。我们预计下个月美国就会做出批准决定，目前正在准备扩大我们的商业引擎，以支持这一药物的上市。尽管目前已有治疗手段，但 CIDP 患者仍然面临着沉重的负担。在过去的 30 年中，患者未能看到有意义的创新，因此未满足的需求很高。

Our data suggests that 88% of patients on current therapy still experience residual symptoms, and patients are constantly balancing the trade-off between efficacy and the treatment burden. A couple of weeks ago, the team had the chance to give firsthand accounts from patients suffering from CIDP. One story really struck me with the patient saying, what hurts the most is not having the treatments we deserve.
我们的数据表明，88%的患者在接受目前的治疗后仍会出现残余症状，患者需要在疗效和治疗负担之间不断权衡。几周前，团队有机会听取了 CIDP 患者的第一手资料。其中一个故事让我印象深刻，患者说："最痛苦的是我们得不到应有的治疗。

All I want is to get back to the things that make me feel alive. This patient is on a high dose of IVIG, which typically requires a two-day administration period. She cannot afford to be away from work for this time. So she consolidate the treatment into one day, which is not sufficient to address her needs and has led to disease progression.
我只想回到那些能让我感觉自己还活着的事情中去。这名患者正在接受大剂量的 IVIG 治疗，通常需要两天的用药时间。她无法在这段时间内离开工作岗位。因此，她将治疗时间合并为一天，但这并不能满足她的需求，反而导致病情恶化。

Our goal with VYVGART Hytrulo is to provide the convenience of a 30 to 92 second injection without compromising on safety or efficacy. And we believe this is possible from the ADHERE data, which we shared with the neurologist community at AAN.
我们对 VYVGART Hytrulo 的目标是在不影响安全性和疗效的前提下，提供 30 至 92 秒注射的便利性。我们在 AAN 会议上与神经科医生社区分享了 ADHERE 数据，我们相信这是可能实现的。

This is the largest trial in CIDP ever run, enrolling 322 patients. Data demonstrated a consistently strong response regardless of prior therapy and we also saw data that showcase the real world impact for CIDP patients.
这是迄今为止规模最大的 CIDP 试验，共招募了 322 名患者。数据显示，无论之前接受过哪种治疗，患者的反应都很强烈。

We successfully demonstrated efgartigimod's ability to drive a sustained improvement in functional strength across prior therapy groups, including almost 30% who improved three or more points on the INCAT scale.
我们成功证明了依加替莫德能够持续改善先前治疗组的功能强度，其中近 30% 的患者在 INCAT 量表上提高了三个或更多分。

As Tim mentioned, a three point improvement can signify the difference from using a wheelchair to walking. This is what a transformational outcome looks like in CIDP. Slide 15. We are well positioned to capture the CIDP opportunity in front of us.
正如蒂姆提到的，三点的改善就意味着从使用轮椅到可以行走。这就是 CIDP 的转化结果。幻灯片 15.我们完全有能力抓住眼前的 CIDP 机遇。

And as I said earlier, we are making decisions today to support our future success, leveraging our learnings and capabilities from the gMG launch and applying them to CIDP. We will take a consistent approach with each of our stakeholder groups to maximize its potential, early engagement with payors, disciplined execution to reach the right prescribers, and always putting patients at the center of our innovation mission.
正如我之前所说的，我们今天做出的决定将支持我们未来的成功，利用我们从 gMG 启动中学到的知识和能力，并将其应用于 CIDP。我们将对每一个利益相关群体采取一致的方法，以最大限度地发挥其潜力，尽早与支付方接触，严格执行以接触到正确的处方者，并始终将患者放在我们创新使命的中心位置。

We have already started our work on enabling broad access to patients. In MG, we partnered with payors so that they could see the value VYVGART creates for the health care system, and we will take the same approach in CIDP.
我们已经开始着手为患者提供广泛的服务。在 MG，我们与支付方合作，让他们看到 VYVGART 为医疗系统创造的价值，我们将在 CIDP 领域采取同样的方法。

We have a strong value proposition based on the ADHERE data and the open label extension study. But it takes about two quarters after approval for payor policies to kick in, which will have an effect on new patient starts during 2024.
基于 ADHERE 数据和开放标签扩展研究，我们有很强的价值主张。但支付方政策需要在批准后两个季度左右才能生效，这将对 2024 年的新患者启动产生影响。

With prescribers, we have expanded our customer facing team to maximize growth in MG while also delivering a successful launch in CIDP. CIDP is an improved indication for IVIG, so this will be a competitive market, but we are equipped to meet the challenge. And last, as we saw in MG, patients and their communities will play an incredibly important role.
在处方方面，我们扩大了面向客户的团队，以最大限度地提高 MG 的增长，同时成功推出 CIDP。CIDP 是 IVIG 的改进适应症，因此这将是一个竞争激烈的市场，但我们已做好准备迎接挑战。最后，正如我们在 MG 领域看到的那样，患者及其社区将发挥极其重要的作用。

In our market research, we've seen that it is a big step for CIDP patients to consider switching their treatment. Our goal is to raise awareness and to empower patients so that they can become advocates for their own care. It will take some time at first, but once the community starts to experience the impact of VYVGART, I'm confident it will happen.
在我们的市场调研中，我们发现 CIDP 患者考虑改变治疗方法是迈出的一大步。我们的目标是提高患者的意识，增强他们的能力，让他们成为自己治疗的倡导者。起初这需要一些时间，但一旦社区开始体验到 VYVGART 的影响，我相信这一定会实现。

I'll now turn the call back to Tim.
现在请蒂姆发言。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Thank you, Karen. Slide 16. I'm truly proud of the argenx team. Through relentless execution, we achieved an incredible milestone of treating over 10,000 patients globally.
谢谢 Karen。幻灯片 16.我为 argenx 团队感到自豪。通过坚持不懈的执行，我们实现了一个令人难以置信的里程碑，在全球治疗了超过 10,000 名患者。

Our commitment to innovation on all fronts has supported this phenomenal growth from generating new data in the clinic that strengthens the use case for VYVGART to our sales team moving deeper into the community setting to reach early life patients.
我们致力于全方位的创新，从在临床中生成新数据以加强 VYVGART 的应用案例，到我们的销售团队深入社区环境以接触早期患者，我们的承诺为这一惊人的增长提供了支持。

We will continue to diligently invest in and execute across our business to maximize the opportunity ahead of us. It is an exciting time for the company as we actively prepare to bring a game changing alternative to the CIDP community, while staying focused on advancing our pipeline.
我们将继续努力投资和执行我们的各项业务，最大限度地利用我们面前的机遇。这是一个令人兴奋的时刻，我们正积极准备为 CIDP 社区带来一个改变游戏规则的替代方案，同时继续专注于推进我们的产品线。

With notable size, we plan to uncover new opportunities to elevate treatment expectations and create value over the long run for autoimmune patients.
凭借显著的规模，我们计划发掘新的机会，提高治疗期望值，为自身免疫性疾病患者创造长期价值。

I would now like to open the floor for questions. Thank you.
现在请大家提问。谢谢。

Question-and-Answer Session
问答环节

Operator 操作员

[Operator Instructions] Your first question comes from the line of Tazeen Ahmad from Bank of America. Your line is open.
[接线员指示] 您的第一个问题来自美国银行的塔泽恩-艾哈迈德。您的电话已接通

Tazeen Ahmad 塔泽恩-艾哈迈德

Okay. Thanks. Hi, guys. Good morning. Thanks for taking my question. Tim, can you just give us a sense about how you're thinking about competitive landscape? Because that seems to be an increasing question that we're getting. Maybe just for gMG, you have talked about increased players in this space. In the time that those have been on the market, how can you talk about the market share that you've had? Have you seen any market share loss as a result of increased competition? And where would you think most of the competition is coming from? Thanks.
好的 谢谢 Okay.谢谢你们好Hi, guys. Good morning.谢谢你接受我的提问。蒂姆，你能告诉我们你是如何考虑竞争格局的吗？因为这个问题似乎越来越多。也许就 gMG 而言，你们已经谈到这个领域的参与者越来越多。在这些公司进入市场的这段时间里，你们如何看待自己的市场份额？你们是否看到竞争加剧导致市场份额下降？你认为竞争主要来自哪里？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Thank you, Tazeen. Thank you for joining us on the call today. And I think this is an excellent question for Karen to address. So I'm going to hand over to Karen straight away on the competition.
谢谢，塔泽恩。感谢你参加我们今天的电话会议。我认为这个问题非常适合凯伦来回答。我现在直接请凯伦回答关于竞争的问题。

Karen Massey 卡伦-梅西

Yes. Thank you, Tim. I guess, Tazeen, the first question you asked was around the competitive landscape, and what we're seeing and what we expected is increased competition and increased entrance into the market. And what we're seeing in our performance is really strong, underlying fundamentals despite those competitors coming to market. You asked specifically about market share. What we've seen quarter-over-quarter is our market share growing amongst biologics, and the majority of that growth is coming through Hytrulo, and it's coming from new patients, directly coming from orals to VYVGART. So at this point in time, we're not seeing significant impact on competition. I think because of the value proposition that we have with VYVGART and VYVGART Hytrulo. We've set the bar really high in terms of efficacy. We continue to show in the real world that our favorable safety profile, plays out. And, of course, with both VYVGART and Hytrulo we have that low treatment burden. So the whole package, as well as our team is competing really well. And, of course, what we see in MG, across the board is that we're just at the beginning of the opportunity. And I think the more innovation that comes to market, the more the biologic share overall is going to grow, and the more that we'll be able to lead within that biologic share.
好的，谢谢你，蒂姆。我想，塔泽恩，你问的第一个问题是关于竞争格局的，我们看到的和我们预期的是竞争加剧和市场进入增多。尽管有这些竞争对手进入市场，但我们在业绩中看到的是真正强劲的基本面。你特别问到了市场份额。我们看到，我们的市场份额在生物制剂中逐季增长，其中大部分增长来自于 Hytrulo，来自于新患者，直接从口服药物转向 VYVGART。因此，从目前来看，我们并没有看到对竞争产生重大影响。我认为这是因为 VYVGART 和 VYVGART Hytrulo 的价值主张。我们在疗效方面设定了很高的标准。我们将继续在现实世界中展示我们良好的安全性。当然，VYVGART 和 Hytrulo 的治疗负担都很低。因此，整套方案以及我们的团队都竞争得非常激烈。当然，我们在 MG 领域看到的是，我们的机会才刚刚开始。我认为，市场上的创新越多，生物制剂的整体份额就会增长得越多，我们就越能在生物制剂份额中处于领先地位。

Tazeen Ahmad 塔泽恩-艾哈迈德

Okay. Thanks so much, Karen.
好的非常感谢，凯伦。

Operator 操作员

Your next question comes from the line of Rajan Sharma from Goldman Sachs. Your line is open.
下一个问题来自高盛集团的拉詹-夏尔马。您的电话已接通

Rajan Sharma 拉詹-夏尔马

Hi. Thanks for taking the question. Just on the PFS filing and maybe related to that competition question, could you just sort of walk us through the steps to approval from here? Have you had any initial interactions with the agency or shared any data? And is there anything you can say about the potential regulatory review process as a faster market opportunity, for example? Thank you.
谢谢您接受我们的提问。关于 PFS 申报，也许与竞争问题有关，你能否向我们介绍一下审批的步骤？你们是否与监管机构进行过初步交流或分享过任何数据？关于潜在的监管审查程序，比如说，作为一个更快的市场机会，你们有什么可以说的吗？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Yes. Thank you for the question. The PFS, of course, is a centerpiece in our strategy for VYVGART overall. Remember we're patient centric and ultimately we think the prefilled syringe is going to be an important addition to the toolbox to serve patients in the most complete fashion possible. The meaningful update today and I'm delighted to give that update actually is that we did succeed in compiling stellar data from a bioequivalence point of view and of course the human factor study. Remember these were the two key data sets we needed to compile in order to submit the dossier. So we're on track to submit the dossier with the FDA before the end of June which is great. And then of course it's going to be in the hands of the FDA. The exact review time remains to be seen, but I'm confident in the quality of the data and the strength of the data which we have submitted. So I would say stay tuned and we will keep you informed on the progress we make with the prefilled syringe. Thanks for the question.
是的，谢谢您的提问。当然，PFS 是我们 VYVGART 整体战略的核心。请记住，我们是以患者为中心的，最终我们认为预灌封注射器将成为工具箱中的重要补充，以最完整的方式为患者提供服务。今天，我很高兴能提供有意义的最新进展，那就是我们确实成功地从生物等效性的角度编制了出色的数据，当然还有人为因素研究。请记住，这是我们提交申请材料所需的两组关键数据。因此，我们有望在 6 月底之前向 FDA 提交档案，这非常好。当然，之后它就会交到 FDA 的手中。具体的审查时间还有待观察，但我对我们提交的数据的质量和实力很有信心。所以我想说，请继续关注，我们会随时向您通报预灌封注射器的进展情况。感谢您的提问。

Operator 操作员

Your next question comes from the line of Derek Archila from Wells Fargo. Your line is open.
下一个问题来自富国银行的 Derek Archila。您的电话已接通

Derek Archila 德里克-阿奇拉

Hey, good morning, and thanks for taking the question. So this one is for Karen. You cited some market research on CID patients potentially switching to VYVGART. So we were wondering, I guess, from that research, what percent of patients said they would actually switch and what's the biggest reason behind the decision to switch? Thanks.
早上好，感谢您接受提问。这个问题是问凯伦的。您提到了一些关于 CID 患者可能转用 VYVGART 的市场调查。那么我们想知道，我想，从这项研究中，有百分之多少的患者表示他们确实会转用，决定转用的最大原因是什么？谢谢。

Karen Massey 卡伦-梅西

Yeah. Thanks for the question. And we've been learning a lot about the CIDP market as we, as we prepare for launch. You know, according to our market research, what we see is that about 88% of patients still experience residual symptoms despite their ongoing treatment. But as you, as you can imagine and as I've shared previously, the patient willingness to switch off their current therapy, it's a big step for them. It's a progressive disease. It's a serious disease. And so it takes a lot for a patient to take that step to switch to another therapy. What we're putting in place, I would say, is a comprehensive launch plan that focuses on educating the neurologists. So that they understand our data. So that they see, the advantage, of VYVGART as well as empowering patients so that they can advocate for themselves and for their health care and that they can really, really see the difference. What we see across the board both with health care providers as well as with patients is that the most meaningful parts of our dataset are, I mean, first of all, the fact that it is the first innovation to come to the market, in in many, many years. It's the biggest trial ever run in CIDP and what we what they find compelling is the broad response rate, that 70% response rate in Stage A as well as the efficacy that's seen in Stage B and Tim shared, during the call, in particular, the improvement on function. That is really compelling. And then, of course, the package is round out by the safety profile and the low treatment burden. So overall what both health care providers and patients like about VYVGART is the fact that they don't have to balance between that trade-off between efficacy and treatment burden. So we have strong conviction over the long-term that we'll be able to have a lot of patients on VYVGART.
好的谢谢你的提问。在我们准备上市的过程中，我们了解了很多关于 CIDP 市场的信息。你知道，根据我们的市场调研，我们发现约 88% 的患者尽管正在接受治疗，但仍有残留症状。但是，正如你所想象的那样，正如我之前所分享的那样，患者愿意放弃目前的治疗，这对他们来说是迈出的一大步。这是一种进展性疾病。这是一种严重的疾病。因此，病人要迈出这一步，换用另一种疗法，需要付出很大的努力。我想说的是，我们正在实施的是一项全面的启动计划，重点是教育神经科医生。让他们了解我们的数据。这样，他们就能看到 VYVGART 的优势，同时也能增强患者的能力，使他们能够为自己和自己的医疗保健进行宣传，让他们真正看到 VYVGART 的不同之处。我们在医疗服务提供者和患者中看到的总体情况是，我们的数据集最有意义的部分是，我是说，首先，它是多年来首次推向市场的创新。这是有史以来针对 CIDP 进行的最大规模试验，我们和他们认为最有说服力的是广泛的反应率，A 阶段 70% 的反应率以及 B 阶段的疗效，蒂姆在电话中特别分享了对功能的改善。这确实令人信服。当然，安全性能和低治疗负担也是整套方案的亮点。因此，总体而言，医疗服务提供者和患者都喜欢 VYVGART 的原因在于，他们不必在疗效和治疗负担之间权衡利弊。因此，我们坚信，从长远来看，VYVGART 会让更多的患者受益。

Operator 操作员

Your next question comes from the line of James Gordon from JPMorgan. Your line is open.
下一个问题来自摩根大通的詹姆斯-戈登。您的电话已接通

James Gordon 詹姆斯-戈登

Hello, James Gordon, JPMorgan. Thanks for taking the question. My question is about VYVGART and CIDP labeling. So we've seen the ADHERE data presented, but I don't think we've seen the open label extension data yet. So is your hope or your expectation that we get that data on the label in June? And is the thinking that that would allow for intermittent dosing as well based on the OLE, and that's how you'd address the potentially quite high price in CIDP? And also just on pricing in CIDP, am I right that if patients in MG use a lot more than the average patient that the pricing is capped. Are there mechanisms you would potentially be able to cap the price in CIDP? Because one of the questions I've had is how pricing could work in CIDP and whether that could be a barrier to use?
你好，我是摩根大通的詹姆斯-戈登。感谢您接受提问。我的问题是关于 VYVGART 和 CIDP 标签的。我们已经看到了ADHERE的数据，但我想我们还没有看到开放标签扩展数据。那么，您是否希望或期待我们能在 6 月份获得标签上的数据？您的想法是否是在开放标签扩展数据的基础上允许间歇给药，从而解决 CIDP 潜在的高昂价格问题？另外，就 CIDP 的定价而言，如果 MG 患者的用药量远高于普通患者，那么定价就会有上限，我的理解是否正确？你们是否有机制可以为 CIDP 的价格设定上限？因为我的一个问题是，在CIDP中如何定价，这是否会成为使用的障碍？

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Thank you, James for the question. Thank you for joining us on the call today. I will hand over in a second the pricing question to Karen. From a labeling point of view, James, of course, we need to wait and see how the conversation is going to go with the FDA. I cannot put myself in their shoes, but I think it's reasonable to assume that given the randomized controlled trial was done with weekly subcu dosing that the label would be reflecting that. And the data you referred to or you alluded to in the open label extension where we go to less frequent dosing that could actually have a meaningful impact in our peer conversation. And that's a nice segue Karen into the pricing question.
谢谢你，詹姆斯的问题。感谢您参加我们今天的电话会议。我马上把定价问题交给凯伦。从标签的角度来看，詹姆斯，当然，我们需要等待，看看与 FDA 的对话将如何进行。我无法设身处地地为他们着想，但我认为，鉴于随机对照试验是以每周一次的剂量进行的，标签上反映出这一点是合理的。而您提到的数据或您在开放标签扩展中暗指的数据，如果我们减少给药次数，实际上会对我们的同行对话产生有意义的影响。这也是凯伦进入定价问题的一个很好的切入点。

Karen Massey 卡伦-梅西

Yes. Happy to address this. It's an important one. So as you said, the price was set with MG, the price per vial, but at the time with an eye towards the CIDP launch and potential CIDP launch. So the approach that we'll take, setting price aside to ensuring access for patients is the same approach, that we took with MG. And you mentioned during that part of that approach was value based arrangements, through discussions with payors. And so we'll take a similar approach. Most importantly, when we have discussions with payors and what our commitment is as a company is that we want to create value for the health care system. I think we've demonstrated that we can do that, with MG, and that's reflected in the favorable payor policies we have whether you look in the US, those favorable payor policies. When you look in Canada, with the recent CADTH description that VYVGART is dominant over IVIG or even the progress in Europe with the reimbursement, that we're getting across the board. You can see that VYVGART is creating value for health care systems and our approach is creating value. And so we'll take the same approach with CIDP to make sure that we get broad access for patients. And I would say in terms of preparations for the launch, our discussions are on track, and our commitment to broad access to CIDP patients remains the same.
是的，很高兴能解决这个问题。这是一个重要问题。正如您所说，MG 的价格是按照每瓶的价格确定的，但当时考虑到了 CIDP 的上市和潜在的 CIDP 上市。因此，我们将采取与 MG 相同的方法，将价格放在一边，以确保患者能够获得治疗。你刚才提到，这种方法的一部分是通过与支付方讨论，做出基于价值的安排。因此，我们将采取类似的方法。最重要的是，当我们与支付方进行讨论时，作为一家公司，我们的承诺是希望为医疗系统创造价值。我认为，我们已经证明，我们可以做到这一点，与 MG 合作，这体现在我们拥有的有利的支付方政策上，无论你是在美国，还是在那些有利的支付方政策上。在加拿大，根据 CADTH 最近的描述，VYVGART 比 IVIG 占据主导地位，甚至在欧洲，我们的报销也取得了全面进展。你可以看到，VYVGART 正在为医疗保健系统创造价值，而我们的方法也在创造价值。因此，我们将对 CIDP 采取同样的方法，确保患者能够广泛获得治疗。我想说的是，就上市准备工作而言，我们的讨论已步入正轨，我们对让 CIDP 患者广泛获得治疗的承诺依然不变。

Operator 操作员

Your next question comes from the line of Akash Tewari from Jefferies. Your line is open.
下一个问题来自 Jefferies 的 Akash Tewari。您的电话已接通

Akash Tewari 阿卡什-特瓦里

Hey, thanks so much. So looking at the geographic breakdown, it looks like US VYVGART patient adds are starting to sequentially flatten out. That said, you mentioned 50% of your new patient ads are now from patients getting off of orals. As we think about 2025 and beyond, do you think that MG will remain a growth market for VYVGART? And have you seen any signs of uptake in the 70k patients upstream to the 17k that are currently not well controlled by steroids? Thanks so much.
非常感谢。从地域细分来看，美国 VYVGART 患者的新增数量似乎开始连续持平。尽管如此，您提到现在 50% 的新患者广告来自于停用口服药的患者。展望 2025 年及以后，您认为 MG 仍将是 VYVGART 的增长市场吗？您是否看到有任何迹象表明，在目前类固醇控制不佳的 7 万名上游患者中，有 1.7 万名患者在接受 VYVGART 治疗？非常感谢。

Karen Massey 卡伦-梅西

Yes. Thanks for the question. And, I would say, actually, I'm very confident that we're just at the beginning of the growth curve with MG. We actually see quite consistent new patient starts that are coming on board. As you said, they're generally coming, from the orals. Over 50% of those are coming from directly from the orals, and as part of that, we're advancing our market share among biologics. In particular, Hytrulo is really helping us to advance into those earlier lines of treatment and to expand the breadth of the our prescriber base. So we have about 2,700 prescribers now. The majority of that 34% growth in new patients for Hytrulo, they're naive to VYVGART. So those are new patients. So the way I see it, we're at the beginning of the curve. Overall, the biologics, in MG are a small percentage of the market. We're leading. That biologic share is growing and we have a long way to go ahead of us and a lot of opportunity.
是的，谢谢你的提问。我想说的是，实际上，我很有信心，我们只是处于 MG 增长曲线的起点。实际上，我们看到新患者的数量在持续增加。正如你所说，他们一般来自口服药。其中超过 50% 的患者直接来自口服药物，而作为其中的一部分，我们正在推进生物制剂的市场份额。特别是，Hytrulo 正在帮助我们进入早期治疗领域，并扩大我们的处方客户群。因此，我们现在拥有约 2,700 名处方者。Hytrulo的新患者增长了34%，其中大部分是VYVGART的新患者。所以这些都是新患者。因此，在我看来，我们正处于曲线的起点。总体而言，生物制剂在 MG 市场上只占很小的比例。我们处于领先地位。生物制剂的份额正在增长，我们还有很长的路要走，还有很多机会。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

And in addition, Akash, to what Karen just said, we also announced the start of the seronegative MG trial, which is going to be a label expanding trial right about 15% of MG patients are estimated to be seronegative. So that will also be a meaningful addition to the label in case this trial is successful.
此外，阿卡什，除了凯伦刚才所说的，我们还宣布启动血清阴性 MG 试验，这将是一项标签扩展试验，据估计约有 15%的 MG 患者血清阴性。因此，如果这项试验取得成功，这也将是一项有意义的新试验。

Operator 操作员

Your next question comes from the line of Yaron Werber from TD Cowen. Your line is open.
下一个问题来自 TD Cowen 的 Yaron Werber。您的电话已接通

Yaron Werber 亚伦-韦伯

Great. Thanks for taking my question. So maybe one that's related to the pipeline. You know, I think in the past, you've talked about potentially doing an R&D day kind of, at some point this year, maybe in the summer or in the fall. What would you want to cover, you know, if you were to do such an R&D day? And then secondly the decision to move to systemic scleroderma, it sounds like we've had ANCA-associated vasculitis. There's too much variability in background meds. What do we know about scleroderma? How homogeneous is it? What percentage of patients are autoantibody positive, if you can give us any sense? Thank you.
太好了。谢谢你回答我的问题。也许有一个问题与管道有关。你知道，我想在过去，你曾说过有可能在今年的某个时候，也许是夏天或秋天，举办一个研发日。如果要举办这样的研发日，你想涵盖哪些内容？其次，决定转向系统性硬皮病，听起来我们已经有了ANCA相关性血管炎。背景药物的可变性太大了。我们对硬皮病了解多少？它的同质性如何？自身抗体阳性的患者比例是多少？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Thank you for these questions, Yaron, and thank you for joining us. So look there's a tonne in this company to talk about when you organize an R&D Day. So we will have to make choices and be carefully listening to the customer of such an R&D Day. For sure a central piece will be in the full data presentation on MMN, which you know is the lead indication for Empa. But we also received strong feedback about the continuous need to be educated on some of these newer indications which we started to talk about. So stay tuned. I think Beth will be communicating in the not too distant future about the date and the agenda of the R&D Day. On systemic scleroderma, this is an indication which was always high on the list. You remember that we always start from a very strong biology rationale, but talking about a strong biology rationale in systemic scleroderma, we have a very good understanding of the autoantibodies of the IgG type and these autoantibodies actually work very well in passive transfer models, where antibodies from patients transferred into animal models actually cause the phenotype of the disease. We also know a number of the autoantigens. So if you immunize these animals with the autoantigens again they develop the typical symptoms of the disease. And then of course plasma exchange works, IVIG works, rituximab is approved in Japan. So there is a very solid body of evidence. This is an IgG driven disease. There are useful clinical endpoints with no real approved medication outside of rituximab in Japan. So we will have to interact of course with the regulators and calibrate expectations on endpoints. But in terms of biology rationale, unmet medical needs and feasibility of doing the clinical trials, this was an indication which made it higher on the list. And of course, we double clicked on it just like we did on all other indications in the portfolio review to really understand potential impact of background medication on autoantibody levels taking the learnings into account of the pemphigus trial. Thanks for the questions.
感谢您的提问，亚伦，感谢您参加我们的会议。在组织研发日活动时，公司有很多事情需要讨论。因此，我们必须做出选择，并认真听取客户对研发日的意见。大家都知道，MMN 是 Empa 的主要适应症。但是，我们也收到了强烈的反馈意见，认为我们需要继续了解一些新的适应症，我们已经开始讨论这些适应症了。敬请期待。我想贝丝将在不久的将来通报研发日的日期和议程。关于系统性硬皮病，这一直是我们优先考虑的适应症。我们对 IgG 型自身抗体有很好的了解，这些自身抗体在被动转移模型中的效果非常好，患者的抗体被转移到动物模型中实际上会导致疾病的表型。我们还知道一些自身抗原。因此，如果再次用自身抗原对这些动物进行免疫，它们就会出现该病的典型症状。当然，血浆置换也有效，IVIG 也有效，利妥昔单抗已在日本获得批准。因此，目前已有大量确凿证据。这是一种 IgG 驱动的疾病。在日本，除了利妥昔单抗外，还没有真正获批的药物能达到有用的临床终点。因此，我们必须与监管机构进行互动，调整对终点的预期。但从生物学原理、未满足的医疗需求和临床试验的可行性来看，这是一个在名单上比较靠前的适应症。当然，我们也对其进行了双击，就像我们对产品组合审查中的所有其他适应症所做的那样，以真正了解背景药物对自身抗体水平的潜在影响，并将丘疹性荨麻疹试验的经验教训考虑在内。感谢您的提问。

Operator 操作员

Your next question comes from the line of Thomas Smith from Leerink Partners. Your line is open.
下一个问题来自 Leerink Partners 的 Thomas Smith。您的电话已接通。

Brian Conley 布莱恩-康利

Hey, great. Thanks. This is Brian Conley on for Thomas Smith. Couple of questions on the TED program. So with respect to Phase 3 studies. Can you talk about how you see efgartigimod fitting into the competitive landscape there? Any specific differentiating aspects you would highlight from the way you've designed your Phase 3 active TED studies, competitors and are you contemplating pursuing broader development in chronic TED? Thanks.
嘿，太好了谢谢我是布莱恩-康利（Brian Conley），我是托马斯-史密斯（Thomas Smith）。我有几个关于TED项目的问题。关于三期研究。您能否谈谈您如何看待依加替莫德在该领域的竞争格局？从你们设计 3 期活性 TED 研究的方式、竞争对手以及你们是否考虑在慢性 TED 领域进行更广泛的开发等方面，你们是否有任何具体的差异化优势？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Yes. So as we announced the Phase 3 study campaign is open. We are enrolling whilst we speak. This will be a global trial also involving our partner Zai Lab in China. It's a sizable market opportunity and I think in the current treatment paradigm, it is becoming clear what the shortcomings are of the currently available medications. It's a relatively similar patient population which we are enrolling in our studies as compared to the [indiscernible] studies. Of course now also involving a capped number of patients which can have seen [indiscernible] in their lives. And that's where we're going to stick with the update today on TED and I think we will be ready to talk more about it when we're deeper into these studies. But the big news today is study is life and is also using by the way the PFS.
是的，正如我们宣布的那样，第三阶段研究活动已经开始。我们正在进行招募。这将是一项全球性试验，我们在中国的合作伙伴 Zai Lab 也将参与其中。这是一个相当大的市场机会，我认为在目前的治疗模式下，现有药物的不足之处已逐渐显现。与[难以辨认]的研究相比，我们的研究招募的患者群体相对相似。当然，现在也设定了患者人数上限，这些患者在他们的生命中可能已经出现过[难以辨认]的情况。这就是我们今天要介绍的 TED 的最新进展，我想当我们更深入地了解这些研究时，我们就可以谈论更多了。但今天的大新闻是研究是生命，也是顺便使用 PFS。

Operator 操作员

Your next question comes from the line of Alex Thompson from Stifel. Your line is open.
下一个问题来自 Stifel 的 Alex Thompson。您的电话已接通

Alex Thompson 亚历克斯-汤普森

Hey, great. Thanks for taking my question. I guess I wanted to ask about self-administration and for CIDP the expectation for potential self-administration at launch or whether the PFS is really the route to achieving self-administration and the label for both CIDP and MG. Thanks.
嘿，太好了。谢谢你接受我的提问。我想问一下自我管理的问题，对于 CIDP 而言，是期望在上市时实现潜在的自我管理，还是 PFS 真的是实现自我管理的途径，以及 CIDP 和 MG 的标签。谢谢。

Karen Massey 卡伦-梅西

Yes. Thanks for the question. We think self-administration is a really important step forward overall as we continue with our expansion strategy for VYVGART and VYVGART Hytrulo. So the label for if approved in CIDP will be for the current Hytrulo label. So that would be for HCP administration. But as we shared earlier, in the call, we're excited about the progress we're making with the prefilled syringe, and the path there, according to the plan that we've laid out is that we will have discussions around self-administration and we think that there's a good a positive path forward there for both MG and CIDP. But, of course, it's always, up to the FDA and a review issue.
是的，谢谢您的提问。我们认为，在我们继续实施 VYVGART 和 VYVGART Hytrulo 的扩张战略时，自我给药是向前迈出的非常重要的一步。因此，如果 CIDP 获批，其标签将沿用当前的 Hytrulo 标签。因此，这将用于 HCP 给药。但正如我们之前在电话中分享的那样，我们对预灌封注射器取得的进展感到兴奋，根据我们制定的计划，我们将围绕自我给药进行讨论，我们认为 MG 和 CIDP 都有很好的发展前景。当然，这始终取决于 FDA 和审查问题。

Operator 操作员

Your next question comes from the line of Xian Deng from UBS. Your line is open.
下一个问题来自瑞银集团的 Xian Deng。您的电话已接通

Xian Deng

Hi. Xian from UBS. Thank you for taking my question. Just a general question on Sjogren's please. So Sjogren's has traditionally been a challenging disease for mobile biologic. So just wondering, you know, what gives you the confidence in VYVGART in Phase 3? Do you think it's a mechanism, you know, FcRn is a target of some biomarker data you have collected, or it's a primary endpoint? We know you already, you know, use the composite endpoint, which is arguably more comprehensive than some others. But on the other hand, you know, Sjogren's, you probably also going to have quite some patients with a lot of prior medications. So just wondering, yeah, any thoughts on that that would be great. Thank you.
你好，我是瑞银的 Xian。感谢您接受我的提问。我只想问一个关于斯约亨氏病的一般性问题。对于移动生物制剂来说，Sjogren 病历来是一种具有挑战性的疾病。我只是想知道，是什么让你们对 VYVGART 的三期临床充满信心？你认为这是一种机制，你知道，FcRn 是你收集到的一些生物标志物数据的靶点，还是主要终点？我们知道你们已经使用了复合终点，这可以说比其他一些终点更全面。但另一方面，你知道，Sjogren's（斯约亨氏症）患者中可能也有不少人之前服用过很多药物。所以，我只是想知道，是的，如果您对此有任何想法，那就再好不过了。谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Yeah. Thanks for the question. Sjogren's, so the Phase 2 signal finding study served two big purposes. Purpose one was to establish confidence in the disease biology and our understanding of the biology. Objective two was to really learn about do's and don'ts for the Phase 3 clinical trial design. So let's start with objective number one. I think we see a very convincing biological signal here that by blocking FcRn you're effectively eliminating the circulating immune complexes which we think are the triggers of the disease and by clearing these circulating immune complexes you basically see a downstream effect in how the immune cell infiltration is going down in the glands and how systemic signs are actually improving and improving across multiple clinical scales hand-in-hand so consistently. Now that's exactly what we wanted to see in addition to a positive Phase 2 trial as announced for Nipocalimab by our colleagues from J&J. Secondly, we learned a great deal about potential impact of background medication and how to mitigate that, the imperfections of the currently used endpoints. So I think all-in-all we have conviction in the datasets and we are actually equipped to now go into our end of Phase 2 meeting with the FDA to discuss our proposal for Phase 3. Thanks for the question.
好的谢谢你的提问。Sjogren's（斯约亨氏症）第二阶段信号发现研究有两大目的。目的一是建立对疾病生物学的信心，以及我们对生物学的理解。目的二是真正了解 3 期临床试验设计的注意事项。让我们从目的一开始。我认为我们在这里看到了一个非常令人信服的生物学信号，即通过阻断 FcRn，你可以有效消除循环免疫复合物，我们认为这是疾病的诱因，而通过清除这些循环免疫复合物，你基本上可以看到下游效应，即免疫细胞在腺体中的浸润如何减少，以及全身症状如何得到改善，并在多个临床指标上得到持续改善。除了 J&J 公司的同事宣布尼泊卡利单抗的 2 期试验结果良好之外，这正是我们希望看到的。其次，我们学到了很多关于背景药物的潜在影响、如何减轻这种影响以及目前使用的终点指标的不完善之处。因此，我认为总的来说，我们对数据集深信不疑，而且我们现在已经具备了与 FDA 进行第二阶段末会议的条件，可以讨论我们的第三阶段提案。感谢您的提问。

Operator 操作员

Your next question comes from the line of Vikram Prahoejit from Morgan Stanley. Your line is open.
下一个问题来自摩根士丹利的 Vikram Prahoejit。您的电话已接通

Vikram Purohit 维克拉姆-普罗希特

Hi. Good morning. Thanks for taking our questions. So we just had two on the pipeline. First on empasiprubart for MMN which is curious if you could talk a bit about what that Phase 3 study could look like and just kind of how you are characterizing that commercial opportunity and then for pipeline readouts for this year, how are you thinking about the potential outcomes for the ALPHA and ALKIVIA datasets, and what would constitute strong outcomes there from your perspective? Thank you.
嗨，早上好。感谢您接受我们的提问。我们有两个问题是关于产品线的。首先是关于针对MMN的empasiprubart，我很想知道您能否谈谈该药的3期研究可能会是什么样子，以及您是如何描述这一商业机会的；然后是关于今年的管线读数，您是如何看待ALPHA和ALKIVIA数据集的潜在结果的，从您的角度来看，什么会构成强有力的结果？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Yes, thank you for all these questions. I think MMN is a sizable opportunity. Of course, it's a rare disease. It is a disease, which is I think under diagnosed and under treated. As we know the only available therapy out there is IVIG and similar to markets like for example MG, I would not be surprised to see this market grow substantially with real innovation coming in. I think from a trial design point of view, we are very well equipped to get ready for Phase 3 because the main objective of the Phase 2 clinical trial of course next to establishing proof-of-concept was to establish a dose response range where we can basically populate our PK/PD model and predict the Phase 3 dose and dosing regimen. I think we are very close to that point. We also had a very rich trial here in terms of clinical endpoints and we already said that all clinical endpoints really moved in sync with each other. So we will be able to have a real educated discussion with the FDA about which endpoint we would suggest and why we would be suggesting that endpoint out of the many ones which we tested. So I think we will be in a strong position to engage in that end of Phase 2 discussion with the FDA relatively soon. In terms of other outcomes this year, we are of course waiting now after the positive Phase 2 data for Sjogren's. We are waiting for the PC-POTS data, which should come in before the middle of the year and then of course the three myositis trials which will come in during the second half of the year. Similar to Sjogren's in PC-POTS, we will be really looking for proof-of-biology and whilst you should be thinking of the go, no-go decision point in the myositis studies in a similar fashion as we designed them for the CIDP study. So we will want to see a signal which is reasonably to be expected stronger than what a placebo signal could be in order to advance in one, two or three of these subsets of myositis and we will be communicating about these go, no-gos at the same time for all three indications. Thanks for the questions.
是的，感谢您提出这些问题。我认为 MMN 是一个相当大的机会。当然，这是一种罕见病。我认为这种疾病诊断不足、治疗不力。我们知道，目前唯一可用的疗法是 IVIG，与 MG 等市场类似，如果有真正的创新产品出现，我不会对这一市场的大幅增长感到惊讶。我认为，从试验设计的角度来看，我们已经为三期临床试验做好了充分准备，因为二期临床试验的主要目的是确定一个剂量反应范围，在这个范围内，我们基本上可以填充我们的 PK/PD 模型，并预测三期临床试验的剂量和给药方案。我认为我们已经非常接近这一点。在临床终点方面，我们也进行了非常丰富的试验，我们已经说过，所有临床终点都是同步进行的。因此，我们将能够与 FDA 进行真正有理有据的讨论，讨论我们会建议采用哪个终点，以及为什么我们会建议采用我们测试过的众多终点中的那个终点。因此，我认为我们很快就能与 FDA 进行第二阶段末期的讨论。至于今年的其他成果，我们现在当然在等待 Sjogren's 2 期的积极数据。我们正在等待 PC-POTS 数据，该数据应在今年年中之前公布，当然，三项肌炎试验也将在今年下半年公布。与PC-POTS中的Sjogren's试验类似，我们将真正寻求生物学证明，而肌炎研究中的 "去 "与 "不去 "的决定点也应与我们为CIDP研究设计的决定点类似。因此，我们希望看到比安慰剂信号更强的合理预期信号，以便在肌炎的一个、两个或三个亚组中取得进展。感谢您的提问。

Operator 操作员

Your next question comes from the line of Danielle Brill from Raymond James. Your line is open.
下一个问题来自 Raymond James 公司的 Danielle Brill。您的电话已接通

Danielle Brill 丹妮尔-布里尔

Hey, guys. Good morning. Thanks so much for the question. I want to circle back to the CIDP launch. Based on recent doc checks, it seems like many patients may have already been earmarked for therapy with VYVGART. I'm wondering what feedback you're encountering in your market research. And is there any reason at this point to think that the launch cadence won't be similar to what we saw in MG? Thank you.
嘿，伙计们早上好非常感谢你们的提问。我想回过头来谈谈 CIDP 的上市。根据最近的医生检查，似乎很多患者已经被指定接受 VYVGART 治疗。我想知道你们在市场调研中遇到了哪些反馈。目前是否有理由认为，VYVGART 的上市节奏不会与我们在 MG 上看到的相似？谢谢。

Karen Massey 卡伦-梅西

Well, thanks for the question, Danielle. And we're seeing something similar in market research in one way is in that there is excitement, amongst prescribers and patients about VYVGART, and the potential. We are not seeing that there's a bolus of patients that are waiting. And in particular, the prescribers have the same question that that many of us have, which is when will the payor policies come online. And we know, that payor policies generally take a couple of quarters to come online after an approval. So I think the uptake, combined with the payor policies coming online, that patient stickiness to IVIG that I talked about a little bit earlier, as well as the fact that IVIG is approved in this indication, and will be a strong competitor. I think that means that that we can expect to see maybe a little bit of a slower uptake versus MG. However, once we start to get the patient switches happening and once we start to shift the market, then I think that I'm confident that that over the long-term, this is a big opportunity, even if it won't be easy in the first days. Thanks for the question.
谢谢你的提问，丹妮尔。我们在市场调研中也看到了类似的情况，那就是处方者和患者对 VYVGART 及其潜力都感到兴奋。我们并没有看到大量患者在等待。特别是，处方者也有和我们很多人一样的问题，那就是支付方政策何时出台。我们知道，支付方政策一般需要几个季度才能在批准后上线。因此，我认为，随着支付方政策的出台，患者对 IVIG 的粘性（我在前面提到过），以及 IVIG 已获准用于该适应症，并将成为强有力的竞争者。我认为，这意味着与 MG 相比，IVIG 的使用可能会稍慢一些。但是，一旦我们开始让患者转换，一旦我们开始改变市场，我相信从长远来看，这是一个巨大的机会，即使在最初的日子里并不容易。感谢您的提问。

Operator 操作员

Your next question comes from the line of Suzanne van Voorthuizen from Van Lanschot Kempen. Your line is open.
下一个问题来自 Van Lanschot Kempen 公司的 Suzanne van Voorthuizen。您的电话已接通。

Unidentified Analyst 身份不明的分析员

Hi. It's Cara Monteironi. I'm on behalf of Suzanne. So I out of curiosity, again, on the drop of the ANCA program, which was replaced by SEC. I was wondering how those two indication compare in terms of effort, trial design, timelines or anything that you wish to highlight? Thank you.
嗨，我是卡拉-蒙泰罗尼我是代表苏珊娜来的出于好奇，我想再次问一下，ANCA项目被SEC取代后，ANCA项目的进展如何？我想知道这两项指标在工作强度、试验设计、时间安排或任何您想强调的方面相比如何？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Roughly speaking, I would park them in the same ballpark in terms of size of opportunity and size of investment. And the unmet medical need in AAV is substantial. The biology by the way is very strong. Actually the disease is called after these autoantibodies and we saw a recent case report published with spectacular data for VYVGART in the hands of physicians in the real world. The real issue for AAV I think is the confounding factor of the background medication. The mandated use of high dose of steroids is actually going to blur the effect of VYVGART. And we looked at it from multiple angles, but there is no credible way to go through that steroid barriers despite a high unmet medical needs. That is quite different in systemic scleroderma. So equally high unmet medical needs, equal number of patients, equally strong biological rationale, but I think a more straightforward path in clinical development. That's how I would call it for the time being.
粗略地说，就机会规模和投资规模而言，我会把它们放在同一起跑线上。而 AAV 未满足的医疗需求是巨大的。顺便说一句，其生物学特性非常强大。实际上，这种疾病是根据这些自身抗体而命名的，我们看到最近发表的一份病例报告中，医生在实际应用 VYVGART 时获得了惊人的数据。我认为，AAV 的真正问题在于背景药物的干扰因素。强制使用大剂量类固醇实际上会模糊 VYVGART 的效果。我们从多个角度研究了这一问题，但尽管存在大量未满足的医疗需求，却没有可信的方法来突破类固醇障碍。这与系统性硬皮病的情况截然不同。因此，同样高的未满足医疗需求，同样多的患者，同样强大的生物学原理，但我认为临床开发的路径更直接。这就是我目前的看法。

Operator 操作员

Your next question comes from the line of Samantha Simankow from Citi. Your line is open.
下一个问题来自花旗银行的 Samantha Simankow。您的电话已接通

Samantha Semenkow 萨曼莎-塞门科

Good morning and thank you for taking the question. My question is just on the uptake of Hytrulo. Is the growth that you highlighted in the prepared remarks, is that a recent uptake or has it been steadily climbing over the last several quarters? And then as you think about introducing PFS as an approved option formulation, would you expect a similar trajectory of growth for the PFS or would you expected to be a sharper uptake. Thank you.
早上好，感谢您接受提问。我的问题是关于 Hytrulo 的使用率。您在备注中强调的增长是最近才出现的，还是在过去几个季度中一直在稳步攀升？在您考虑引入 PFS 作为获批的可选制剂时，您是否预计 PFS 也会出现类似的增长轨迹，或者您是否预计 PFS 的吸收率会更高。谢谢。

Karen Massey 卡伦-梅西

Yes. Thanks for the question, Samantha. I'll take that. We actually did see an acceleration in the uptake of Hytrulo in Q1 and there were specific reasons for that, namely that the payor policies were in place and the J-Code was established in Q1. So we did see an acceleration. We expect that to continue, and it's important to note just talking about it with the CDC launch, it did take about two quarters for those payor policies to come into place, with Hytrulo. So we've seen that now. In moving forward, I think, we're very excited about the pre-filled syringe and being able to offer an even broader product presentation options to patients. I do think it's a significant advancement on the current Gen One of Hytrulo and I do think it will enable us to further move up to earlier lines of treatment with VYVGART and further advance or expand our prescriber base as well. So we do see that PFS will be another engine for growth, if you will, for VYVGART. Thanks for the question.
是的，谢谢你的问题，萨曼莎。我来回答。实际上，我们确实看到 Hytrulo 在第一季度加快了使用速度，这是有具体原因的，即支付方政策已经到位，J-Code 也在第一季度确立。因此，我们确实看到了加速度。我们希望这种情况能继续下去，值得注意的是，在谈及疾病预防控制中心的推出时，Hytrulo 的支付方政策确实花了两个季度的时间才到位。因此，我们现在已经看到了这一点。展望未来，我认为，我们对预灌封注射器以及能够为患者提供更广泛的产品展示选择感到非常兴奋。我认为这是对目前第一代 Hytrulo 的重大进步，而且我认为这将使我们能够进一步提高 VYVGART 的早期治疗水平，并进一步推进或扩大我们的处方客户群。因此，我们认为 PFS 将成为 VYVGART 的另一个增长引擎。谢谢您的提问。

Operator 操作员

Your next question comes from the line of Gavin Clark-Gartner from Evercore ISI. Your line is open.
下一个问题来自 Evercore ISI 的 Gavin Clark-Gartner。您的电话已接通。

Gavin Clark-Gartner 加文-克拉克-加特纳

Hey, thanks for taking the question. On Empa in the pipeline, I believe, you've noted there's been no cases of lupus in the ongoing MMN study. But I also wanted to ask about rates of ANA tighter elevations, specifically, any other markers such as dsDNA? Thank you.
谢谢您的提问。关于正在进行中的 Empa 研究，我相信您已经注意到，在正在进行的 MMN 研究中没有发现红斑狼疮病例。但我还想问一下 ANA 严格升高的比率，特别是其他标记物，如 dsDNA？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Yes, that's a great question and a potential differentiator actually for C2 versus the C1s.No, we did not see any increase of such status and at least from a theoretical point of view, we have always believed this could be one of the advantages of an anti-C2 antibody. So none of these signs and a great opportunity for me to remind everyone on the call that the Phase 1 data both for IV and Subcu came out with spic and span safety and tolerability data. So far so good. Thanks for the question.
是的，这是一个很好的问题，也是 C2 与 C1 的潜在区别所在。不，我们没有看到这种状态有任何增加，至少从理论上讲，我们一直认为这可能是抗 C2 抗体的优势之一。因此，这些迹象都不存在，我想借此机会提醒电话会议上的各位，IV 和 Subcu 的第一阶段数据已经出炉，安全性和耐受性数据都很好。到目前为止一切顺利。谢谢您的提问。

Operator 操作员

Your next question comes from the line of Yatin Suneja from Guggenheim. Your line is open.
下一个问题来自古根海姆的 Yatin Suneja。您的电话已接通

Yatin Suneja 雅廷-苏内贾

Hi. Quick one for me. On VYVGART could you comment on what you're seeing in terms of discontinuation there? Is it in that 20% range? And then anything you are able to say on number of cycles that you are able that patients are getting? Thanks.
你好，我有一个问题想问。关于 VYVGART，您能谈谈您看到的停产情况吗？是否在 20% 的范围内？然后，关于患者能够接受的治疗周期数，您有什么要说的吗？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Thank you for the question. On discontinuation, as you know, 20% of the patients from the DAP study did not respond. So you would expect this continuation to be 20% plus because there are also other reasons. And what we see in the real world is in line with our expectations and that has been consistent since launch. In terms of the number of cycles, the number of cycles for IV has also been consistent and we're about and it's around 5% as we previously said. So there's been no change. Thank you.
谢谢您的提问。关于停药，正如您所知，DAP 研究中有 20% 的患者没有反应。因此，您预计继续服用该药的比例会超过 20%，因为还有其他原因。我们在现实世界中看到的情况符合我们的预期，而且自上市以来一直如此。就周期数而言，静脉注射的周期数也是一致的，我们的周期数约为 5%，正如我们之前所说的那样。所以没有变化。谢谢。

Operator 操作员

Your next question comes from the line of Victor Floch from BNP Paribas. Your line is open.
下一个问题来自法国巴黎银行的 Victor Floch。您的电话已接通。

Victor Floch 维克多-弗洛奇

Hi. Thanks a lot for taking my question. Victor Floch from BNP Paribas Exane. So a couple of questions on my side. First, I was wondering if it's fair to say that the SG&A cost facing which in this quarter is reflecting a higher need to build that awareness, right, of launch in CDIP than what was needed for MG back in the day. So if you could just remind us the key challenges in CDIP compared to MG would be appreciated. And my second question is about ITP. And so I was just wondering if you could update us on your efforts to potentially expand the ITP opportunity beyond Japan. So I don't know what is in the balance to take any decision and if you could commit to any timing, but yeah any color on that would be very much appreciated. Thanks a lot.
你好，非常感谢你接受我的提问。我是来自法国巴黎银行 Exane 的 Victor Floch。我这边有几个问题。首先，我想知道，如果说本季度的 SG&A 成本反映了 CDIP 比 MG 更需要建立知名度，这是否公平。因此，如果您能提醒我们 CDIP 与 MG 相比所面临的主要挑战，我们将不胜感激。我的第二个问题是关于 ITP 的。我想知道您能否向我们介绍一下您在日本以外地区拓展 ITP 机会的最新情况。我不知道你们是否会做出任何决定，也不知道你们是否会承诺任何时间，但如果能提供任何相关信息，我将非常感激。非常感谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Thank you for the question. I will give the floor in a minute to Karl who is going to comment on the increase in SG&A expenses in preparation of the CIDP launch. Karl, you will be able to give some color there. Thank you for your question on ITP. We are of course delighted to see the PMD approval for ITP. We see the first patients coming on product very quickly underscoring I think the unmet medical needs which is present in ITP. And I think it's fair to say that throughout the interactions with the PMD, we also gained a number of insights we can use to actually pivot back to the FDA for a follow-up conversation and that's exactly what the team is preparing. I think we are equipped now to go back to the FDA for a dialogue to see whether we can make progress on the ITP front. So stay tuned. I certainly don't want to overpromise, but I think we're going to make an extra effort there which I think we owe to ITP patients. Karl, would you mind commenting on the SG&A question?
谢谢您的提问。我马上请卡尔发言，他将就为 CIDP 上市做准备而增加的 SG&A 费用发表评论。卡尔，你可以给我们提供一些信息。感谢您提出有关 ITP 的问题。我们当然很高兴看到ITP的PMD获批。我们看到首批患者很快就用上了产品，这凸显了我认为ITP尚未满足的医疗需求。可以说，在与 PMD 的互动过程中，我们也获得了许多真知灼见，我们可以利用这些真知灼见与 FDA 进行后续对话，而这正是我们团队正在准备的。我认为，我们现在有能力回到食品药物管理局进行对话，看看我们能否在ITP方面取得进展。敬请期待。我当然不想过度承诺，但我认为我们会在这方面做出额外的努力，这是我认为我们欠 ITP 患者的。卡尔，你能就 SG&A 问题发表一下意见吗？

Karl Gubitz 卡尔-古比茨

Okay. Yes. SG&A increased by around $27 million in Q1 '24 versus the end of last quarter in 2023. That increase is largely driven by the incremental infrastructure we put in place in the US versus customer facing colleagues, which will be promoting gMG today. And as we said previously, we believe that the gMG opportunity is bigger than what we originally thought and now we're expanding that footprint. That same footprint will also be used for the CIDP launch subject to approval later this year. Also part of that increase is the geographical expansion. You've heard us talk about certain markets like Italy and Spain and other European markets where we're starting to have sales. We've been very disciplined to guide any expenses in those markets until we have pricing and reimbursement in place. And as we start seeing sales in both markets, we are investing in both markets. So it is the US and the geographical expansion which is driving that increase. Thank you for the question.
好的，是的。与 2023 年上季度末相比，24 年第一季度的 SG&A 增加了约 2700 万美元。这一增长主要是由于我们在美国投入了更多的基础设施，以及今天将推广通用管理集团（gMG）的面向客户的同事。正如我们之前所说的那样，我们相信 gMG 的机会比我们最初想象的要大，现在我们正在扩大这一足迹。如果今年晚些时候获得批准，CIDP 的推出也将采用同样的覆盖范围。地域扩张也是增长的一部分。你们已经听到我们谈到了某些市场，如意大利、西班牙和其他欧洲市场，我们已经开始在这些市场销售。在定价和报销到位之前，我们一直严格控制在这些市场的任何支出。随着我们开始在这两个市场看到销售额，我们将在这两个市场进行投资。因此，是美国和地域扩张推动了这一增长。感谢您的提问。

Operator 操作员

Your next question comes from the line of Joel Beatty from Baird. Your line is now open.
下一个问题来自贝尔德公司的乔尔-比蒂。您的电话现在开通。

Joel Beatty 乔尔-比蒂

Thanks for taking the question. With Q1 typically being a challenging quarter, how did the trajectory of VYVGART sales change over the course of the quarter into early Q2?
感谢您接受提问。第一季度通常是一个充满挑战的季度，那么在本季度到第二季度初，VYVGART 的销售轨迹发生了怎样的变化？

Karen Massey 卡伦-梅西

Yes. Thanks for the question. As you say, Q1 is notoriously challenging in particular in the US with holidays and recertifications and weather, et cetera. We're really pleased to be with the growth, the quarter-on-quarter growth that we delivered in the US as well as the year-on-year growth. I mean, it was 76%, year-on-year growth, for the US. What we're seeing and what we feel is confidence that the underlying fundamentals of what we delivered in Q1 were very strong and I went over a few of those whether you look at new patient growth, where the patients are coming from, we're advancing market share, all of those factors, and we're seeing that continue into Q2. So we're confident, in the remainder of the year.
是的，谢谢您的提问。正如你所说，第一季度是出了名的挑战，尤其是在美国，假期、重新认证、天气等等。我们很高兴在美国实现了季度增长和同比增长。我的意思是，美国的同比增长率达到了 76%。我们所看到的和感受到的是，我们对第一季度业绩的基本面充满信心，无论是新病人的增长、病人来自哪里、我们正在推进市场份额，所有这些因素都非常强劲。因此，我们对今年剩余时间充满信心。

Operator 操作员

Your next question comes from the line of Manos Mastorakis from Deutsche Bank. Your line is open.
下一个问题来自德意志银行的马诺斯-马斯托拉基斯（Manos Mastorakis）。您的电话已接通

Manos Mastorakis 马诺斯-马斯托拉基斯

Hello. Thank you for taking my question. Just basically on the key learnings you took away from the exercise you did across indications and which led to the discontinuation of ANCA vasculitis and how do some of those learnings extend to empasiprubart? Thank you.
您好。感谢您接受我的提问。我只想问一下，你们从跨适应症的研究中获得了哪些重要启示，这些启示导致了 ANCA 血管炎的停药，其中一些启示是如何延伸到 empasiprubart 的？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Thank you. It's a great question. Actually when we did the portfolio review, we did not only limited to efgartigimod, we immediately included also the Empa indications. So we did a full review of all planned and ongoing indications and we gained confidence for all Empa indications which are currently on the rails, but also the ones which we're planning in addition that actually we can master the impact of the background medication. So I feel very strong about the big efforts all the teams did in such a short period of time following the pemphigus data. We've thoroughly double clicked on trial designs, understanding the impact of background medication and whether or not we are actually equipped to deal with those. So exercise has landed and actually most indications remain on track with the exception of AAV where we no longer feel it's an responsible allocation of capital to take such a risk from a background medication point of view. Thanks for the question.
谢谢。这是个很好的问题。事实上，我们在对药物组合进行审查时，不仅限于依加替莫德，还立即纳入了 Empa 适应症。因此，我们对所有计划中和正在进行中的适应症进行了全面审查，我们对目前正在进行中的所有 Empa 适应症以及我们正在计划中的其他适应症充满信心，相信我们能够掌握背景药物的影响。因此，在获得丘疹性荨麻疹数据后，我对所有团队在如此短的时间内做出的巨大努力感到非常欣慰。我们对试验设计、了解背景药物的影响以及我们是否有能力应对这些问题进行了彻底的双击。因此，这项工作已经着陆，实际上，除了 AAV 外，大多数适应症仍在按计划进行，从背景药物的角度来看，我们不再认为承担这样的风险是一种负责任的资金分配。谢谢您的提问。

Operator 操作员

Your next question comes from the line of Matt Phipps from William Blair. Your line is open.
下一个问题来自 William Blair 公司的 Matt Phipps。您的电话已接通

Matt Phipps 马特-菲普斯

Thanks for taking my question. Two quick ones. First, on the human factor studies, did you use CID patients in that study? I'm just wondering if somebody with reduced grip strength would be suitable for self-administrated PFS. And then, I guess, following the potential CID approval, given, what you said about the payor policies, would you plan to provide some kind of free drug program given the, you know, underlying patient interest and demand?
谢谢你回答我的问题。简单问两个。第一，关于人为因素的研究，你们在研究中使用了 CID 患者吗？我只是想知道，握力减弱的人是否适合自我管理 PFS。然后，我想，在潜在的 CID 批准之后，考虑到你所说的支付方政策，你是否计划提供某种免费药物计划，你知道，潜在的患者兴趣和需求？

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Thank you for the two questions. I will hand over the second question in terms of launch preparation to Karen, although I suspect we will not be showing too much of our [indiscernible]. From a human factor study point of view, you're absolutely right. I mean, we're targeting the pre-filled syringe for both gMG and CIDP patients. So you can imagine that in the human factor studies both patient groups were actively involved and we feel very strong about the data which we achieved and we feel very strong about the submission which is now in the works before end of June. Karen, question two please.
谢谢你的两个问题。我将把第二个关于发射准备的问题交给凯伦，尽管我猜想我们不会展示太多我们的[难以辨认]。从人为因素研究的角度来看，你说得非常对。我的意思是，我们的目标是为 gMG 和 CIDP 患者提供预灌封注射器。因此，你可以想象，在人为因素研究中，两个患者群体都积极参与其中，我们对我们取得的数据感到非常满意，我们对提交的文件也感到非常满意，该文件目前正在六月底之前的工作中。凯伦，请回答第二个问题。

Karen Massey 卡伦-梅西

Yes, happy to. Without going into too many details exactly on what our plans are. Maybe the best way to answer that is we're taking the same approach to the CIDP launch preparation as we did with MG. And the way that I would characterise that is that we're being really thinking very innovatively about how we want to go to market, how we want to empower patients, how we want to educate prescribers. And we're also be thinking in a very disciplined way, around our execution, and how do we make sure that we're making the right capital investments or the right use of our capital, and making the right investments. So we'll take that same approach for the CIDP launch that we did for MG.
是的，很乐意。我们的计划是什么，就不多说了。也许回答这个问题的最好方式是，我们将采取与 MG 一样的方式来准备 CIDP 的上市。我想说的是，我们正在以创新的方式思考如何进入市场、如何增强患者的能力、如何教育处方者。同时，我们也在以非常严谨的方式思考我们的执行，以及如何确保我们正在进行正确的资本投资或正确使用我们的资本，并进行正确的投资。因此，我们将采取与推出 MG 相同的方法来推出 CIDP。

Operator 操作员

And your final question comes from the line of Douglas Tsao from H. C. Wainwright. Your line is open.
最后一个问题来自 H. C. Wainwright 公司的 Douglas Tsao。您的电话已接通

Douglas Tsao

Hi. Good morning. Thanks for taking the questions. Just a quick one for me. I'm just curious, ultimately when we think about the different presentations of efgartigimod, I mean, where do you see the pre-filled syringe ultimately falling between the Subcu and the IV? I mean do you think that this could ultimately become the sort of dominant presentation that's utilized? Thank you.
嗨，早上好。感谢您接受提问。我只想问一个简单的问题。我只是很好奇，当我们考虑依加替莫德的不同表现形式时，我的意思是，你认为预灌封注射器最终会介于 Subcu 和 IV 之间吗？我的意思是，你认为这最终会成为一种主要的使用方式吗？谢谢。

Tim Van Hauwermeiren 蒂姆-范豪尔梅伦

Yes. Thank you for the question. So, we are a patient centric company and it's our ambition to serve the needs of the individual patient segments as completely as we can. We believe that especially in the US market the IV product will always be important because a subset of patients, but also actually physicians do prefer an IV infusion. The interesting thing about VYVGART Hytrulo today is that it's actually a very clean and easy execution which is still resolving under mainly I would say Medicare Part B whilst the PFS with the self-administration is mainly going to target I think or sit in the Medicare Part D channel. So if you think about the market leaders who is patient centric, I think, the PFS will be the closing piece in the totality of the product offering maximally serving I think different needs and preferences of different market segments. Thanks for the question.
是的，谢谢您的提问。我们是一家以患者为中心的公司，我们的目标是尽可能全面地满足不同患者的需求。我们相信，尤其是在美国市场，静脉注射产品将一直很重要，因为一部分患者和医生都更喜欢静脉注射。如今，VYVGART Hytrulo 的有趣之处在于，它实际上是一种非常简洁、易于执行的产品，目前仍主要在我认为的联邦医疗保险 B 部分下使用，而具有自我管理功能的 PFS 则主要针对我认为的联邦医疗保险 D 部分渠道。因此，如果你认为市场领导者是以患者为中心的，我认为，PFS 将是整个产品的收尾部分，最大限度地满足我认为不同细分市场的不同需求和偏好。感谢您的提问。

Operator 操作员

And we have now reached the end of our question-and-answer period. This concludes today's conference call. Thank you for your participation. You may now disconnect.
问答时间到此结束。今天的电话会议到此结束。感谢各位的参与。现在可以挂断电话。