argenx SE (ARGX) 2025 Q4 法說會逐字稿

Good morning. My name is Rob, and I will be your conference operator today. I would like to welcome everyone to the call. (Operator Instructions)

早安.我叫羅伯，今天我將擔任你們的會議接線生。歡迎各位參加本次通話。（操作說明）

I'd now like to introduce Beth DelGiacco, Vice President of Corporate Affairs. You may now begin your conference.

現在我來介紹一下公司事務副總裁貝絲·德爾賈科。現在您可以開始會議了。

Thank you. Two press releases were issued earlier today, one sharing the positive results from our Phase III ADAPT OCULUS study and the other, which outlines our fourth quarter and full year 2025 financial results and business update. These can be found on our website along with the presentation for today's webcast.

謝謝。今天早些時候發布了兩份新聞稿，一份分享了我們 III 期 ADAPT OCULUS 研究的積極結果，另一份概述了我們 2025 年第四季度和全年的財務業績和業務更新。這些內容以及今天網路直播的簡報都可以在我們的網站上找到。

Before we begin on slide 2, I'd like to remind you that forward-looking statements may be presented during this call. These may include statements about our future expectations, clinical developments, regulatory time lines, the potential success of our product candidates, financial projections and upcoming milestones.

在開始講解第二張投影片之前，我想提醒各位，本次電話會議中可能會出現前瞻性陳述。這些可能包括關於我們未來預期、臨床進展、監管時間表、我們候選產品的潛在成功、財務預測和即將到來的里程碑的聲明。

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.

實際結果可能與這些聲明中所述存在重大差異。除非法律另有規定，否則argenx沒有義務更新有關未來的聲明或使這些聲明與實際結果相符。

I'm joined on the call today by Karen Massey, Chief Operating Officer; Karl Gubitz, Chief Financial Officer; Luc Truyen, Chief Medical Officer; Sandrine Piret-Gerard, Chief Commercialization Officer; and Tim Van Hauwermeiren, Chief Executive Officer.

今天與我一起參加電話會議的有：營運長 Karen Massey；財務長 Karl Gubitz；醫療長 Luc Truyen；商業化長 Sandrine Piret-Gerard；以及執行長 Tim Van Hauwermeiren。

I'll now turn the call over to Karen.

現在我將把電話轉給凱倫。

Thank you, Beth, and welcome, everyone. I'll begin on slide 3. 2025 was an incredible year of execution for argenx. We reached 19,000 patients globally, driven in part by the successful launch of our prefilled syringe for self-injection. We also continue to advance our deep and differentiated immunology pipeline, including 4 new molecules from our IIP, positioning us for sustained long-term growth.

謝謝你，貝絲，也歡迎各位。我將從第 3 張投影片開始。 2025 年對 argenx 來說是執行力驚人的一年。我們在全球惠及了 19,000 名患者，部分原因是我們的預充式註射器（用於自我注射）成功上市。我們將繼續推進我們深度且差異化的免疫學產品線，包括來自我們 IIP 的 4 個新分子，這將使我們處於持續長期成長的地位。

This progress is grounded in our commitment to patients to innovate in ways that don't just improve care, but meaningfully change what patients can expect from their treatment. I'm speaking to you today from our US national team meeting, where hearing directly from patients is a powerful reminder of why our work matters.

這項進展源於我們對患者的承諾，透過創新不僅改善護理，而且從根本上改變患者對治療的期望。今天我正在美國國家隊會議上向大家講話，在這裡，直接聽取患者的心聲，讓我深刻地體會到我們工作的意義所在。

One moment in particular, stayed with me. We recently received a handwritten note from a patient, thanking the team for the impact VYVGART has had on her life. We later learned that before starting treatment, she had been living with very severe MG symptoms that significantly limited her day-to-day activities.

其中一個瞬間，我至今記憶猶新。我們最近收到一位患者的手寫便條，感謝團隊對 VYVGART 治療對她生活的影響。我們後來了解到，在開始治療之前，她一直飽受重症肌無力症狀的折磨，嚴重限制了她的日常生活。

Today, at the meeting, we saw a video of the patient about a year into treatment with VYVGART Hytrulo, sharing an update from a hype she was on. She is thriving. It's one individual story, but it reinforces the real-world difference VYVGART can make.

今天在會議上，我們看到了一段視頻，視頻中一位接受 VYVGART Hytrulo 治療約一年的患者分享了她最近接受治療的最新情況。她現在發展得很好。這只是一個個例，但它強調了 VYVGART 能在現實世界中帶來的改變。

Slide 4. At the start of the year, we outlined our strategic priorities for 2026 that will guide our next chapter of growth towards Vision 2030. We want to impact more patients globally with VYVGART through broader patient adoption and label expansion. We're shaping the future of FcRn medicines with next-generation molecules, delivery modalities and combination approaches and delivering the next wave of immunology innovation, supported by a strong late-stage portfolio and a goal of at least one new pipeline candidate per year.

幻燈片 4。今年年初，我們制定了 2026 年的策略重點，這將引導我們邁向 2030 年願景的下一個成長階段。我們希望透過擴大患者群體和擴大適應症範圍，使 VYVGART 在全球範圍內惠及更多患者。我們正在利用新一代分子、遞送方式和聯合療法塑造 FcRn 藥物的未來，並帶來下一波免疫學創新浪潮，這得益於我們強大的後期產品組合以及每年至少推出一個新候選藥物的目標。

Slide 5. VYVGART is leading the growth of biologics in both MG and CIDP, and we're confident that we have the right strategies and milestones ahead to sustain this momentum. Today marks an exciting moment for ocular MG patients with the positive ADAPT OCULUS results, which Luc will discuss shortly. Together with our progress in seronegative MG, we see a meaningful opportunity to broaden VYVGART's reach to patients who have historically had limited or no targeted treatment options.

幻燈片 5。VYVGART 在 MG 和 CIDP 的生物製劑領域處於領先地位，我們有信心，我們擁有正確的戰略和里程碑，可以保持這一發展勢頭。今天對於眼肌型重症肌無力患者來說是一個令人興奮的時刻，ADAPT OCULUS 檢測結果呈陽性，Luc 稍後將對此進行討論。結合我們在血清陰性重症肌無力方面取得的進展，我們看到了一個有意義的機會，將 VYVGART 的治療範圍擴大到那些歷史上缺乏或沒有標靶治療選擇的患者。

What's guided us here is a long-standing commitment to the MG community and to advancing our understanding of the underlying biology of the diseases we treat. Across MG populations, our data confirm that disease is driven by pathogenic IgGs regardless of antibody status.

一直以來，我們秉持著對 MG 患者群體的長期承諾，以及不斷加深對所治療疾病的潛在生物學機制的理解。我們的數據證實，在所有 MG 患者群體中，疾病是由致病性 IgG 驅動的，與抗體狀態無關。

In seronegative MG, we demonstrated a clinically meaningful improvement in MG-ADL in the overall population with responses becoming more pronounced with subsequent treatment cycles across all subtypes. In ocular MG, we're seeing that same biology extend to another patient population with VYVGART meeting its primary endpoint and driving clear improvements in ptosis and diplopia.

在血清陰性重症肌無力患者中，我們證實了整體族群的重症肌無力日常生活活動能力（MG-ADL）在臨床上得到了有意義的改善，並且隨著後續治療週期的進行，所有亞型的反應都變得更加明顯。在眼肌型重症肌無力中，我們看到同樣的生物學原理也延伸到了另一類患者群體，VYVGART 達到了其主要終點，並在眼瞼下垂和複視方面帶來了明顯的改善。

Our seronegative PDUFA date is May 10. And based on today's results, we see a clear path to expanding our label into ocular MG as well, positioning VYVGART to have the broadest MG label and to reach our target addressable population of approximately 60,000 patients in the US.

我們的血清陰性 PDUFA 日期是 5 月 10 日。根據今天的測試結果，我們看到了將我們的標籤擴展到眼部重症肌無力的明確途徑，這將使 VYVGART 擁有最廣泛的重症肌無力標籤，並涵蓋我們在美國約 60,000 名目標患者群體。

In CIDP, we're also having a meaningful impact on patients with clinical data showing functional improvement and these benefits increasingly reflected in real-world experience. VYVGART is driving a paradigm shift in CIDP. While there remains significant opportunity within the initial 12,000 addressable patient population, we're also beginning to see expansion beyond that population, a core focus as we build leadership in CIDP. Sandrine will speak more to this later in the call.

在 CIDP 領域，我們也對患者產生了有意義的影響，臨床數據顯示功能有所改善，這些益處也越來越多地體現在現實世界的經驗中。VYVGART正在推動CIDP領域的典範轉移。雖然在最初的 12,000 名目標患者群體中仍存在著巨大的機會，但我們也開始看到目標群體擴展到該群體之外，這是我們在 CIDP 領域建立領導地位的核心重點。Sandrine稍後會在通話中對此做更多說明。

Slide 6. Over the next 12- to 18 months, we have multiple avenues for expansion beyond MG and CIDP, including autoimmune myositis and Sjogren's disease, which broaden VYVGART's footprint into rheumatology.

幻燈片 6。在接下來的 12 到 18 個月裡，除了 MG 和 CIDP 之外，我們還有多種擴張途徑，包括自體免疫性肌肉炎和乾燥綜合徵，這將擴大 VYVGART 在風濕病領域的影響力。

In particular, our work in IMNM highlights a significant unmet need with an estimated 20,000 patients and no approved treatment options today. Meanwhile, our upcoming Q4 readout for empasiprubart in MMN marks an important milestone, positioning us to advance a second medicine to patients and extending our neurology footprint with a first-in-class C2 inhibitor. We have an opportunity to address a clear unmet need in MMN with IVIg as the only approved treatment and symptom progression in 60% of patients.

特別是，我們在 IMNM 領域的工作凸顯了一個重大的未滿足需求，估計有 20,000 名患者，但目前尚無核准的治療方案。同時，我們即將公佈的 empasiprubart 在 MMN 中的第四季度數據標誌著一個重要的里程碑，使我們能夠將第二種藥物推向患者，並透過首創的 C2 抑制劑擴大我們在神經病學領域的影響力。我們有機會解決 MMN 領域一個明顯的未滿足需求，目前 IVIg 是唯一核准的治療方法，但 60% 的患者會出現症狀進展。

Slide 7. Lastly, we continue to strengthen the pipeline that will shape our long-term future. VYVGART is just the beginning of FcRn leadership that we aim to establish for decades to come. As part of this, we're advancing two next-generation assets, ARGX-213 and ARGX-124. We're investing in efgartigimod anchored combination approaches and new delivery modalities like the auto-injector and oral peptide capabilities.

幻燈片 7。最後，我們將繼續加強對塑造我們長遠未來的人才儲備。VYVGART 只是 FcRn 未來幾十年領導地位的開端。作為其中的一部分，我們正在推動兩項下一代資產的開發，即 ARGX-213 和 ARGX-124。我們正在投資以 efgartigimod 為錨定劑的組合療法和新的給藥方式，如自動注射器和口服勝肽療法。

At the same time, we're seeing real momentum across our broader innovation platform, progressing first-in-class molecules like ARGX-121 targeting IgA and ARGX-118 targeting Galectin-10. We are deliberately source agnostic in how we identify new biology drawing from both leading academic research and opportunities emerging within biopharma.

同時，我們看到我們更廣泛的創新平台正在取得真正的進展，推進了針對 IgA 的首創分子 ARGX-121 和針對 Galectin-10 的 ARGX-118 等藥物的研發。我們在辨識新的生物學特性時，刻意不拘泥於特定來源，既藉鑒領先的學術研究，也關註生物製藥領域湧現的新機會。

In 2026, we expect to progress three Phase I programs, including a program from our Tensegrity collaboration, reinforcing our ability to bring forward high-quality science wherever it originates. We have an exciting year ahead of us with a strong foundation in place and exciting progress across the pipeline.

2026 年，我們預計將推進三個第一階段項目，其中包括我們與 Tensergrity 合作的項目，這將增強我們無論在何處都能推進高品質科學的能力。我們已經打下了堅實的基礎，各個專案都取得了令人振奮的進展，未來一年充滿機會。

Let's turn to the data that's shaping our next steps. Luc?

讓我們來看看影響我們下一步行動的數據。盧克？

Thank you, Karen. I'm excited to share the positive outcome of our Phase III ADAPT OCULUS study. Our second MG expansion milestone to hit just months after we shared positive seronegative data.

謝謝你，凱倫。我很高興地與大家分享我們 III 期 ADAPT OCULUS 研究的正面結果。在我們分享了積極的血清陰性數據幾個月後，我們實現了第二個 MG 擴張里程碑。

Let's turn to slide 8. Together with leading global experts, our team designed the first registrational study in ocular myasthenia gravis, filling a long-standing gap for the patient population that has historically been excluded from clinical trials. We leveraged the screening period to ensure patients had a confirmed diagnosis of ocular MG, defined as MGFA Class I, meaning patients had meaningful, measurable eye symptoms without evidence of generalized disease.

讓我們翻到第 8 張投影片。我們的團隊與全球領先的專家一起設計了首個眼肌型重症肌無力註冊研究，填補了長期以來被排除在臨床試驗之外的患者群體的空白。我們利用篩檢期來確保患者確診為眼肌型重症肌無力，定義為 MGFA I 級，這意味著患者有有意義的、可測量的眼部症狀，但沒有全身性疾病的證據。

Patients were also required to be on stable background therapy. 141 patients were randomized 1:1 to VYVGART Hytrulo versus placebo. And in Part A, they received four weekly injections. In Part B, participants received multiple cycles of VYVGART Hytrulo. The primary endpoint of the study was a change in MGII patient-reported ocular score from baseline to day 29, a measure focused on the key ocular symptoms of myasthenia gravis, diplopia and ptosis.

患者還需接受穩定的背景治療。 141 名患者以 1:1 的比例隨機分配至 VYVGART Hytrulo 組或安慰劑組。在 A 部分中，他們接受了四周的每週注射。在 B 部分，參與者接受了多個週期的 VYVGART Hytrulo 治療。研究的主要終點是 MGII 患者報告的眼部​​評分從基線到第 29 天的變化，該指標側重於重症肌無力的關鍵眼部症狀，即複視和眼瞼下垂。

Slide 9. The study met its primary endpoints. Treatment with VYVGART Hytrulo led to a statistically significant improvement in MGII patient-reported ocular score at week 4 compared to placebo with a p-value of 0.012. VYVGART-treated patients experienced a mean 4.04 point improvement compared to a 1.99 point improvement on placebo, including clear improvements on diplopia and ptosis. VYVGART was well tolerated, upholding its consistently strong safety profile with no new safety signals. We will present a broader data set at an upcoming medical meeting.

幻燈片 9。該研究達到了其主要終點。與安慰劑相比，VYVGART Hytrulo 治療組在第 4 週時 MGII 患者自述眼部評分有統計學意義上的顯著改善（p 值為 0.012）。 VYVGART 治療組患者的平均評分改善了 4.04 分，而安慰劑組患者的平均評分改善了 1.99 分，其中複視和眼瞼下垂症狀均有明顯改善。VYVGART 耐受性良好，保持了其一貫的良好安全性，沒有出現新的安全性訊號。我們將在即將召開的醫學會議上公佈更全面的數據集。

This is a big day for patients. Ocular MG strips people of independence. Many suffer from headaches and the persistent double vision and drooping eyelids don't just affect eyesight, they can take away the ability to drive, work and confidently engage in daily life, often with a heavy psychological burden and stigma. And today, too many patients are still relying on chronic steroids and symptomatic therapy, which comes with an unacceptable treatment burden over time.

對患者來說，今天是個重要的日子。眼部重症肌無力剝奪了患者的獨立性。許多人飽受頭痛之苦，持續的複視和眼瞼下垂不僅影響視力，還會剝奪駕駛、工作和自信地參與日常生活的能力，常常伴隨著沉重的心理負擔和恥辱感。如今，仍有太多患者依賴長期服用類固醇和症狀治療，這隨著時間的推移會帶來難以接受的治療負擔。

For the first time, we are bringing forward a therapy that specifically addresses the underlying pathological mechanism of ocular MG, and that is something we should all be excited about. Based on these results, we plan to file an sBLA with the FDA.

我們首次提出了一種專門針對眼肌型重症肌無力潛在病理機制的療法，值得我們所有人感到興奮。基於這些結果，我們計劃向FDA提交補充生物製品許可申請（sBLA）。

Now before I turn the call over to Karl, I want to sincerely thank the investigator site teams and most importantly, the patients and families who made this study possible. Karl?

在將電話交給卡爾之前，我衷心感謝研究團隊，更重要的是，感謝讓這項研究成為可能的患者及其家屬。卡爾？

Thank you, Luc. Slide 10. The fourth quarter and full year 2025 financial results are detailed in this morning's press release. Product net sales are consistent with our preannouncement in January at $1.3 billion for the fourth quarter and $4.2 billion for the full year, which represents a year-over-year growth of 90%.

謝謝你，盧克。第10張幻燈片。今天早上的新聞稿詳細介紹了2025年第四季和全年的財務表現。產品淨銷售額與我們 1 月的預告一致，第四季為 13 億美元，全年為 42 億美元，年增 90%。

Regional breakdown of product revenue in Q4 2025 reflects $1.1 billion in the US, $63 million in Japan, $110 million in the rest of the world and $26 million in product supplied to Zai Lab in China. The product net sales in the US grew by 68% from the fourth quarter of the prior year, reflecting solid patient demand and prescriber confidence driven by PFS. The gross to net adjustments and the net pricing in the US are in line with the prior quarter.

2025 年第四季產品收入的區域細分顯示，美國收入為 11 億美元，日本收入為 6,300 萬美元，世界其他地區收入為 1.1 億美元，向中國再鼎醫藥供應的產品收入為 2,600 萬美元。該產品在美國的淨銷售額比上年第四季成長了 68%，反映出患者需求強勁，處方醫生對 PFS 的信心也增強。美國毛利淨利調整及淨價與上一季一致。

Next slide, slide 11. Total operating expenses in the fourth quarter are $955 million, representing an increase of $149 million compared to the third quarter. Cost of sales for the quarter is $150 million as our year-to-date gross margin remains consistent at 11%. The combined R&D and SG&A expenses totaled $2.7 billion for the full year, which is in line with our financial guidance for 2025 discussed in our most recent earnings call.

下一張投影片，第 11 張投影片。第四季總營運支出為 9.55 億美元，比第三季增加了 1.49 億美元。本季銷售成本為 1.5 億美元，年初至今的毛利率維持在 11% 的穩定水準。全年研發和銷售、管理及行政費用合計為 27 億美元，這與我們最近一次財報電話會議上討論的 2025 年財務指導一致。

Looking ahead into 2026, operating expenses will continue to grow at a similar percentage as in prior years. SG&A growth will support the significant revenue growth in our current markets as well as expansion into new patient populations. R&D expenses will increase due to our continued commitment to execute on our pipeline.

展望 2026 年，營運費用將繼續以與往年類似的百分比成長。銷售、一般及行政費用的成長將支持我們在現有市場實現顯著的收入成長，並拓展到新的患者群體。由於我們持續致力於推動產品線的研發，研發費用將會增加。

Our operating profit for the quarter is $367 million and $1.1 billion for the year, which marks our first year of annual operating profitability. Tax for the quarter and full year reflects a net benefit. This is largely due to non-recurring tax items and favorable foreign exchange movements. Going forward, you should continue to expect an effective tax rate in the low to mid-teens. This brings us to the profit for the fourth quarter of $533 million and $1.3 billion for the full year, respectively.

本季營業利潤為 3.67 億美元，全年營業利潤為 11 億美元，這是我們首次實現年度營業盈利。本季和全年的稅收結果均反映出淨收益。這主要是由於非經常性稅收項目和有利的匯率變動所致。展望未來，您應該繼續預期實際稅率在十幾到十二幾個百分點之間。由此得出，第四季利潤為 5.33 億美元，全年利潤為 13 億美元。

Our cash balance represented by cash, cash equivalents and current financial assets is $4.4 billion at the end of the fourth quarter, which represents a more than $1 billion increase over the year. The strength of our balance sheet allows us to invest with confidence in growing our commercial business as well as our pipeline.

截至第四季末，我們的現金餘額（包括現金、現金等價物和流動金融資產）為 44 億美元，比去年同期增加了 10 多億美元。我們穩健的資產負債表使我們能夠充滿信心地投資於商業業務和產品線的成長。

I will now turn the call over to Sandrine, who will provide details on the commercial front.

現在我將把電話交給桑德琳，她將提供有關商業方面的詳細資訊。

Thank you, Karl. Slide 12. I'm thrilled to be joining argenx at such a pivotal moment. What excites me most is the combination of bold science and a deeply patient-driven mission, what I often describe as science with purpose.

謝謝你，卡爾。第12張投影片。我很高興能在這樣一個關鍵時刻加入 argenx。最令我興奮的是大膽的科學研究與以病人為中心的使命結合，我經常稱之為有目標的科學研究。

I've spent time in the field already, met clinicians and seen firsthand the real impact our science is having on patients' lives. With Vision 2030 as a road map, we have a clear path to meaningfully improve the lives of more than 50,000 patients.

我已經在實地考察過一段時間，與臨床醫生會面，親眼目睹了我們的科學對病人生活產生的真正影響。以「2030願景」為路線圖，我們有了明確的路徑，可以實際改善超過5萬名病患的生活。

Slide 13. Echoing Karen, we entered 2026 from a position of strength following a year of phenomenal execution. We closed 2025 with approximately 19,000 patients on treatment globally, reflecting consistent growth across all regions and all indications.

第13張幻燈片。與凱倫的觀點一致，經過一年出色的執行，我們以強大的實力進入了 2026 年。截至 2025 年底，全球約有 19,000 名患者接受治療，這反映出所有地區和所有適應症均實現了持續成長。

We successfully launched the prefilled syringe, which has proven to be a key driver in increased overall VYVGART demand. At the end of the fourth quarter, we had more than 4,700 prescribers, including dozen new prescribers since the PFS launch. This momentum underscores the execution strength of our field teams, the added convenience the PFS brings to patients and the growing confidence in VYVGART among clinicians.

我們成功推出了預充填注射器，事實證明，這是推動 VYVGART 整體需求成長的關鍵因素。截至第四季末，我們擁有超過 4700 名處方醫生，其中包括自 PFS 推出以來新增的十幾名處方醫生。這一勢頭凸顯了我們現場團隊的執行力，PFS 為患者帶來的額外便利，以及臨床醫生對 VYVGART 日益增長的信心。

As we highlighted at the start of the year, our next chapter is about applying a proven indication expansion playbook to reach even more patients. MG and CIDP remain the cornerstone of our commercial strength, and we are well positioned to build on that foundation as we scale.

正如我們在年初所強調的那樣，我們的下一個篇章是應用已被證明有效的適應症擴展策略，以惠及更多患者。MG 和 CIDP 仍然是我們商業實力的基石，隨著我們規模的擴大，我們有能力在此基礎上繼續發展。

Slide 14. We entered the MG market with strong biology and a first-in-class therapy. Since then, we have redefined what patients and clinicians can expect with the highest MSE and a favorable safety and tolerability profile. As a result, VYVGART is the fastest-growing and number one prescribed biologics in MG with continued momentum driven by earlier line adoption. 6 out of 10 MG patients starting on biologic start with VYVGART. 70% of VYVGART patients are already coming from orals, and we believe the PFS will continue to help drive near-term growth.

第14張幻燈片。我們憑藉著強大的生物學特性和一流的療法進入了MG市場。從那時起，我們重新定義了病人和臨床醫生可以期待的最高MSE以及良好的安全性和耐受性。因此，VYVGART 已成為重症肌無力 (MG) 領域成長最快、處方量最高的生物製劑，其持續成長勢頭得益於早期應用。十分之六的 MG 患者在開始接受生物製劑治療時首先使用 VYVGART。 70% 的 VYVGART 患者先前已接受口服藥物治療，我們相信其無惡化存活期 (PFS) 將繼續推動近期成長。

We are now on track to reach 18,000 additional patients through two label expanding opportunities, seronegative and ocular MG. Seronegative MG alone has the potential to move us towards the broadest possible MG label with our May PDUFA just around the corner. And ocular MG gives us a chance to be the first to market in a patient group that has had no precision treatment options.

我們現在正朝著透過兩個標籤擴展機會（血清陰性和眼肌型重症肌無力）為 18,000 名額外患者鋪平道路。僅憑血清陰性 MG 就有可能使我們獲得最廣泛的 MG 標籤，而我們的 5 月 PDUFA 即將到來。眼肌型重症肌無力讓我們有機會成為第一個將精準治療方案推向市場的患者族群。

What gives us confidence here is that these expansions build on strong relationships we have already established with neurologists, many of whom are confident in VYVGART through experience treating generalized MG.

讓我們充滿信心的是，這些擴張建立在我們與神經科醫生已經建立的牢固關係之上，其中許多神經科醫生透過治療全身性重症肌無力的經驗對 VYVGART 充滿信心。

Slide 15. We are earlier in the CIDP launch trajectory, but are delivering on the same disciplined approach that has led to our successful market expansion in MG. Significant opportunity remains within the 12 dozen patients who are not well managed on current treatment, and our focus today is on continued evidence generation, patient activation and new prescriber adoption.

第15頁。我們在 CIDP 的上市進程中還處於早期階段，但我們秉持著與 MG 市場成功擴張時相同的嚴謹方法。對於目前治療效果不佳的 12 多名患者而言，仍然存在著巨大的機會，我們今天的重點是繼續產生證據、動員患者和讓新的處方醫生接受治療。

Clinicians continue to respond to the meaningful functional benefit data and well-characterized safety shown in the ADHERE trial. The prefilled syringe is further driving uptake by reducing the administration burden and offering more flexibility to patients. Worth noting, we secured an important access win for PFS in Q4 with UnitedHealthcare, broadening our covered lives to over 90%.

臨床醫生繼續對 ADHERE 試驗中顯示的有意義的功能獲益數據和明確的安全性做出回應。預充式註射器透過減輕給藥負擔和為患者提供更大的靈活性，進一步推動了其普及。值得一提的是，我們在第四季度與 UnitedHealthcare 達成了一項重要的 PFS 准入協議，使我們的承保人群擴大到 90% 以上。

CIDP is a highly heterogeneous disease, and we are committed to advancing the science to expand our reach to broader set of patients. Our biomarker program is designed to better define responders and unlock earlier and broader use, and we are advancing empasiprubart in a head-to-head study against IVIg to further explore the bounds of efficacy. Together, these efforts position us to expand the CIDP population we can serve and continue shaping this market over the long term.

CIDP 是一種高度異質性的疾病，我們致力於推動科學發展，以擴大我們的服務範圍，並惠及更多患者。我們的生物標記計畫旨在更好地定義應答者，並解鎖更早、更廣泛的應用，我們正在推進 empasiprubart 與 IVIg 的頭對頭研究，以進一步探索療效的界限。這些努力共同使我們能夠擴大 CIDP 患者群體，並繼續在長期內塑造這個市場。

Slide 16. Our clinical pipeline continues to broaden and deepen, providing a multiyear runway for commercial growth. I'm excited to join the company at this pivotal moment to help scale the organization thoughtfully and translate this pipeline into even greater patient impact.

第16張幻燈片。我們的臨床研發管線不斷拓展和深化，為商業成長提供了多年的發展空間。我很高興在這個關鍵時刻加入公司，幫助公司穩定擴大規模，並將這些研發成果轉化為對病患更大的影響。

With that, I'll now turn the call over to Tim.

接下來，我會把電話交給提姆。

Thank you, Sandrine. Reflecting on where we stand, argenx has never been better positioned, and our leadership transition comes at the right moment as we enter our next phase of growth. Karen is the right leader to take this forward. She understands our innovation playbook, leads with patients at the center of every decision and brings the operational discipline needed to continue executing against Vision 2030 and beyond. I have complete confidence that she will nurture what has always made argenx special while driving the next chapter of growth for the company.

謝謝你，桑德琳。回顧我們所處的位置，argenx 從未像現在這樣處於有利地位，在我們進入下一個成長階段之際，領導階層的更迭也恰逢其時。凱倫是帶領這項工作向前邁進的合適人選。她了解我們的創新策略，以患者為中心做出每一個決定，並具備持續執行「2030 年願景」及未來發展所需的營運紀律。我完全相信她會保留 argenx 一直以來的獨特之處，同時推動公司進入下一個發展階段。

My dedication to argenx and to our mission remains as strong as ever. I look forward to supporting Karen and the entire leadership team as we continue to advance meaningful innovation and deliver for patients and shareholders alike.

我對 argenx 和我們使命的奉獻一如既往地堅定。我期待支持凱倫和整個領導團隊，我們將繼續推動有意義的創新，為患者和股東創造價值。

With that, operator, we will open the call up to questions.

接線員，接下來我們將開放問答環節。

(Operator Instructions)

（操作說明）

Tazeen Ahmad, Bank of America.

塔津·艾哈邁德，美國銀行。

First off, Karen, congratulations on the new role. We're looking forward to continuing to work with you. And Tim, what else can I say, but thank you. You've set the example for everyone to follow, and we wish you the best in your new upcoming role as well.

首先，凱倫，恭喜你榮升新職。我們期待繼續與您合作。提姆，我還能說什麼呢，除了謝謝你。你為大家樹立了榜樣，我們也祝福你在即將到來的新職位上一切順利。

So my first question is going to be on the addition of both seronegative as well as based on today's results, assuming ocular MG to the revenue stream for VYVGART. How should we think about, number one, what the average price would be for each of these sub-indications? And can you talk to us about what proportion -- you talked to us about how many patients there are. But have you done any market data research to indicate what proportion of those patients are more likely to seek this type of treatment?

所以我的第一個問題是關於將血清陰性患者以及根據今天的結果假設眼部 MG 患者納入 VYVGART 的收入來源。首先，我們該如何考慮每種子適應症的平均價格？能跟我們談談其中的比例嗎？您之前跟我們談過有多少病人。但是，您是否進行過任何市場數據調查，以顯示有多少比例的患者更有可能尋求這種類型的治療？

Well, thank you, Tazeen, for your comments and also for your question. It's a really exciting day for ocular MG patients and certainly for argenx, as you call out. It's important to think about the fact that we are now the first and only -- or VYVGART is the first and only to have positive data for patients with ocular MG. So a really exciting day for patients.

塔津，謝謝你的評論和提問。對於眼肌型重症肌無力患者來說，這真是令人興奮的一天，當然對 argenx 來說也是如此，正如你所說。重要的是要考慮到，我們現在是第一個也是唯一一個——或者說 VYVGART 是第一個也是唯一一個擁有眼肌型重症肌無力患者積極數據的機構。對患者來說，這真是令人振奮的一天。

And as you called out, that, combined with the seronegative data that just read out a few months ago, and we have the PDUFA date in May, really positions us well for continued sustained growth in MG and I think an expansion even further of our leadership position in MG. So we're very excited to share that data today.

正如您所指出的，再加上幾個月前公佈的血清陰性數據，以及 5 月份的 PDUFA 日期，這確實使我們在 MG 領域能夠繼續保持持續增長，我認為這將進一步擴大我們在 MG 領域的領先地位。所以，我們今天非常高興能與大家分享這些數據。

I'll let Karl talk to the price in a moment. But just on the second part of your question around the addressable market, we obviously have done quite a bit of market research, and we'll continue to do so to prepare for how best to go to market. But the best numbers to look at are those that we've provided with the seronegative expanding the addressable market by 11,000 patients and ocular by 7,000 patients that we've provided before.

稍後我會讓卡爾談談價格。但就您關於目標市場問題的第二部分而言，我們顯然已經做了大量的市場調查，並且我們將繼續這樣做，以便為如何最好地進入市場做好準備。但最值得關注的數字是我們先前提供的血清陰性患者群體擴大了目標市場 11,000 名患者，眼科患者群體擴大了 7,000 名患者。

That 7,000 patients in ocular MG, that's not the total ocular MG patient population. That's actually the portion that when we've done the research before, we thought would be eligible for VYVGART. So that's the number that I would stick with. And obviously, as we get closer to -- as we unpack the data more, as we get closer to submission and hopefully approval, we'll be able to provide more color on that.

那7,000位眼肌型重症肌無力患者，並不是眼肌型重症肌無力患者的總人數。實際上，在我們之前進行研究時，我們認為這部分符合 VYVGART 的條件。所以我會堅持使用這個數字。顯然，隨著我們越來越接近——隨著我們對數據進行更深入的分析，隨著我們越來越接近提交並有望獲得批准，我們將能夠提供更多細節。

And then maybe, Karl, you could comment on the price.

然後，卡爾，或許你可以對價格發表一下看法。

Thank you, Karen and Tazeen, thank you for the question. Yes, we still have to have the discussions with the players, of course. But I do want to mention that we have a strong capability and market access. It is an enabler of our launch, not a hurdle, and the value proposition of VYVGART is well understood and appreciated by all stakeholders.

謝謝Karen和Tazeen，謝謝你們的提問。當然，我們還需要和球員們進行討論。但我確實想提一下，我們擁有強大的能力和市場准入。它助力我們成功推出產品，而不是阻礙我們推出產品，VYVGART 的價值主張也得到了所有利害關係人的充分理解和讚賞。

At this stage, I will say that we would expect to have broad access also for seronegative and ocular, and we can assume a similar price as MG, i.e., $225,000 the net benefit or a net price to argenx. Thank you for the question.

現階段，我想說，我們預計血清陰性和眼部疾病患者也能廣泛獲得治療，我們可以假設價格與 MG 類似，即 225,000 美元的淨收益或 argenx 的淨價格。謝謝你的提問。

Danielle Brill, Truist Securities.

Danielle Brill，Truist Securities。

I think I'll ask a question on the CIDP opportunity. Karen, you mentioned in your prepared remarks that you're beginning to see expansion beyond the initial 12,000 patients that you were targeting. Can you elaborate a bit? Are you seeing a step-up in frontline use? And then I think you also mentioned that you secured additional coverage, broadening coverage for PFS to over 90% of covered lives. What impact do you expect this to have on adoption rates in the setting going forward?

我想問一個關於CIDP機會的問題。Karen，你在事先準備好的演講稿中提到，你開始看到患者數量超過了最初設定的 12,000 名患者。能詳細說說嗎？您是否觀察到第一線使用量增加？然後，我想您還提到您獲得了額外的保險覆蓋範圍，將 PFS 的覆蓋範圍擴大到 90% 以上的參保人。您認為這將對未來該領域的採用率產生什麼影響？

Thanks, Danielle, for the question and the interest in CIDP, we're really pleased with the continued growth in CIDP. So, yes, we laid out that the strategy was first to focus on that 12,000 patients that are treated -- that are already treated, but continue to have symptoms. And that is -- continues to be where we see the majority of our patients and the majority of our growth.

謝謝 Danielle 的提問以及對 CIDP 的關注，我們對 CIDP 的持續成長感到非常高興。所以，是的，我們制定的策略是先集中精力治療那 12,000 名已經接受治療但仍有症狀的患者。而且，這仍然是我們大多數患者所在地區，也是我們大部分成長所在地區。

But you'll recall that our label does allow us to be used in a broader patient population. And there are some payer policies actually that also allow that. So we are starting to see some use of VYVGART beyond just the switch from IVIg.

但您應該記得，我們​​的標籤確實允許我們在更廣泛的患者群體中使用。實際上，有些支付方的保單也允許這樣做。因此，我們開始看到 VYVGART 的應用不僅限於從 IVIg 切換到 VYVGART。

In general, it's still about at 85% of our patients are being switched from IVIg, but there are some that are coming directly. I think as prescribers and neurologists get more experience with VYVGART and see the impact in the real world, then over time, we'll start to see that expansion even more.

總的來說，仍有約 85% 的患者是從靜脈注射免疫球蛋白 (IVIg) 轉而接受治療，但也有一些患者直接接受了靜脈注射免疫球蛋白治療。我認為，隨著處方醫生和神經科醫生對 VYVGART 的使用經驗越來越豐富，並看到其在現實世界中的影響，隨著時間的推移，我們將開始看到這種療法得到更廣泛的應用。

And as you said, continuing to expand access with the recent UnitedHealthcare decision and having 90% coverage, that also helps to contribute to our growth. So I would say what to expect in CIDP is that continued steady momentum. We're still early in the launch. And so I think we still have some quite a bit of growth ahead of us.

正如您所說，隨著 UnitedHealthcare 最近的決定，我們繼續擴大了醫療服務覆蓋範圍，目前覆蓋率已達 90%，這也有助於我們的發展。所以我認為，CIDP 的發展趨勢是持續穩定的成長動能。我們目前還處於產品發布初期。所以我認為我們還有相當大的發展空間。

Derek Archila, Wells Fargo.

Derek Archila，富國銀行。

Congrats on the progress in the Phase III win today. So I had a question on, do you think approval in ocular MG will drive more utilization in the less advanced MG patients? And I guess, is there anything in the data set that you'll present in the future that could demonstrate prevention of progression to more generalized disease?

恭喜今天在第三階段試驗中取得進展並獲勝。所以我有個問題，您認為眼肌型重症肌無力的批准是否會促使病情較輕的重症肌無力患者更多地使用該藥物？那麼，您未來將要展示的資料集中是否有任何內容可以證明能夠預防疾病發展為更普遍的疾病呢？

Yes. Thanks for the question, Derek. I'll comment on the first and then maybe, Luc, I can hand it to you for the data. So certainly, I think that our hypothesis, I mean, we know that in MG, the majority of patients first do present with ocular symptoms and then the majority of those ocular MG patients do transition into gMG. So a big part of our strategy is expanding the use of biologics to earlier line uses of MG.

是的。謝謝你的提問，德里克。我會先評論第一個問題，然後，盧克，也許我可以把資料交給你。所以，我認為我們的假設是成立的，我的意思是，我們知道在重症肌無力中，大多數患者首先會出現眼部症狀，然後大多數眼部重症肌無力患者會發展為全身型重症肌無力。因此，我們策略的重要組成部分是將生物製劑的使用範圍擴大到重症肌無力的早期治療。

We are already seeing that. Biologic use is growing in generalized MG. We are driving -- we get 6 out of 10 of those patients that are first use biologics. So we're driving a lot of that earlier use and a lot of that growth. As you say, I think the ocular MG data will help us with that strategy and will provide a halo to that strategy. And then maybe, Luc, you could comment on the data and progression.

我們已經看到了這種情況。生物製劑在全身型重症肌無力的應用日益廣泛。我們正在努力－我們接診的10名患者中有6名是首次使用生物製劑的患者。因此，我們推動了早期使用和成長的很大一部分。正如你所說，我認為眼部 MG 數據將有助於我們實施該策略，並為該策略提供保障。然後，盧克，或許你可以對數據和進展發表一些評論。

Yes. Thanks, Karen, and thanks, Derek, for the question, which is close to my heart. So with the data in hand, we show that we can meaningfully impact the current symptomatology of ocular MG, which is not MG like. It's a significantly debilitating state to be in. But of course, the excitement of continuing to collect long-term data as we are planning to do and compare that to what is known with the natural progression, which, as Karen said, is a high percentage up to 80% will allow us to make some statements on do we delay progression to generalized MG. So I would say stay tuned.

是的。謝謝凱倫，也謝謝德里克，你們提出的問題我非常關心。因此，根據我們掌握的數據，我們證明我們可以對眼部重症肌無力的當前症狀產生有意義的影響，而眼部重症肌無力的症狀與典型的重症肌無力不同。這是一種極為令人痛苦的狀態。當然，繼續收集長期數據（正如我們計劃的那樣）並將其與已知的自然進展進行比較，這令人興奮。正如凱倫所說，自然進展的比例高達 80%，這將使我們能夠就我們是否能延緩發展為全身性重症肌無力發表一些聲明。所以我想說，敬請期待。

Yatin Suneja, Guggenheim.

Yatin Suneja，古根漢美術館。

Just with regard to the Q1 dynamics, could you point to us if there are any particular consideration that we should have for Q1 in particular?

就第一季的動態而言，您能否指出我們在第一季應該特別注意哪些方面？

Yes. Thanks for the question. And it's important as we're in Q1. So obviously, across the industry, we can see the pattern that there always are Q1 dynamics around reverifications and winter storms, of which we've had quite a few in the last couple of weeks. So argenx and VYVGART are, of course, privy to the same, those same seasonal dynamics. And we saw that last year as well. If you recall, we did have a slowdown in Q1. And then in the end of the year, we delivered 90% full year growth. So I think you can recognize the pattern and expect that. But maybe, Sandrine, you could comment on the underlying dynamics that we're seeing since you've joined.

是的。謝謝你的提問。而且這一點很重要，因為我們現在正處於第一季。顯然，在整個行業中，我們可以看到這樣的模式：第一季總是圍繞著重新核實和冬季風暴展開，而過去幾週我們已經經歷了不少這樣的風暴。所以，argenx 和 VYVGART 當然也受到同樣的季節性動態的影響。去年我們也看到了這種情況。如果你還記得的話，我們第一季確實出現了成長放緩的情況。年底，我們實現了全年 90% 的成長。所以我覺得你應該能識別出這種模式並預料到這種情況。不過，桑德琳，或許你可以談談你加入以來我們所看到的潛在動態。

Yes. Thank you, Karen. And this is something that I looked at before joining argenx, what is the growth we are seeing. And year-after-year, we have been delivering consistent growth, and this is a pattern you can expect this year, full year because the underlying dynamic are very healthy.

是的。謝謝你，凱倫。在加入 argenx 之前，我就一直在關注這個問題，那就是我們所看到的成長情況。我們年復一年地實現了持續成長，而且這種趨勢在今年全年都將持續，因為其基本面非常健康。

I mean when you look at the new patient starts, the provider and the prescriber expansion. When you look at our access, we just mentioned that, but also how strong we are and VYVGART is in leading the growth of the overall biologic market. These are all amazing underlying factors that will help us continue that growth.

我的意思是，當你觀察新病患的就診量、醫療服務提供者和處方醫生數量的增長情況時。當你審視我們的市場准入時，正如我們剛才提到的，也要看看我們以及 VYVGART 在引領整個生物製劑市場成長方面的實力。這些都是有助於我們持續成長的絕佳潛在因素。

And then you have the PFS that was launched less than a year ago that drove a lot of momentum last year, plus the expansion of the labels that we are expecting both for seronegative and later for ocular. So all are good underlying factors that will help us continue that growth, as Karen mentioned.

此外，不到一年前推出的 PFS 在去年推動了巨大的發展勢頭，再加上我們預計血清陰性和稍後眼部適應症的擴展適應症，也推動了 PFS 的發展。正如凱倫所提到的那樣，這些都是有助於我們繼續保持成長的良好潛在因素。

James Gordon, Barclays.

詹姆斯·戈登，巴克萊銀行。

James Gordon from Barclays. The question was on VYVGART for myositis. And my question was, what is the efficacy bar you're looking to exceed in the Phase III in myositis in Q3? What's a good result? Is there hope to be more efficacious than [brepo] or JAK/TYK and what they did in the VALOR trial? Or is it more -- a good result would be if you had a similar efficacy and you are better tolerated as well? So what's good and what's really good?

巴克萊銀行的詹姆斯‧戈登。問題是關於VYVGART治療肌炎的。我的問題是，在第三季肌炎的 III 期臨床試驗中，你們希望達到的療效標準​​是多少？什麼樣的結果才算好結果？是否有希望比 [brepo] 或 JAK/TYK 以及它們在 VALOR 試驗中的表現更有效？或者說，更好的結果是──療效相近，耐受性也更好？那麼，什麼是好的，什麼是非常好的呢？

And could I also just squeeze in a clarification, not a question, but just normally, there's an OpEx guide, but I didn't see a formal guide this year. Should we assume a similar pace of OpEx growth this year as last year, so '25 similar pace of '26 and maybe more R&D and less SG&A? How do we think about spend this year, please?

另外，我能否順便澄清一下，這不是問題，而是通常都會有一份營運支出指南，但我今年沒有看到正式的指南。我們是否應該假設今年的營運支出成長速度與去年類似，即 2025 年與 2026 年類似，且研發支出增加，銷售、一般及行政費用減少？我們打算如何度過今年呢？

Yes. Thanks for the questions there, James. And so I'll open. I'll hand over to Luc to provide some more color on myositis and then Karl on OpEx. But the first thing that I just wanted to frame is when you think about myositis, it's right out of the argenx playbook. I mean there is so little options available to patients here, really limited innovation in the market. And so what we're looking for is a statistical significant benefit coming out of this study.

是的。謝謝你提出的問題，詹姆斯。那我就開場吧。接下來，我會把麥克風交給 Luc，讓他更詳細地介紹肌肉炎，然後交給 Karl，讓他介紹 OpEx。但我首先想說明的是，當你想到肌肉炎時，你會發現它完全符合 Argenx 的治療模式。我的意思是，這裡患者可選擇的治療方案非常少，市場上的創新也很有限。因此，我們希望這項研究能夠得出具有統計意義的顯著結果。

In the DM, in IMNM, there are no approved therapies available. And you heard in the script that there are 20,000 patients with IMNM. So for them, any benefit, I think, is clinically meaningful. But maybe, Luc, you could talk about how we're thinking about the study.

在 DM 和 IMNM 中，目前尚無核准的治療方法。你在劇本裡也聽到了，有 20,000 名患有 IMNM 的患者。所以對他們來說，我認為任何好處都具有臨床意義。不過，盧克，或許你可以談談我們是如何看待這項研究的。

Yes. Thanks and also for laying it up that this is not a singular indication. So this is a constellation of indications that each have somewhat different pathological drivers. We continued our Phase III program based on the strength of a robust Phase II, which gave us the confidence that we could provide meaningful benefit across the three subsets. Ultimately, the data will speak once we complete Phase III.

是的。謝謝，也謝謝你指出這並非個案。所以這是一系列跡象，每個跡像都有一些不同的病理驅動因素。我們基於穩健的第二階段研究成果，繼續進行第三階段研究項目，這讓我們有信心能為三個子群體帶來有意義的益處。最終，第三階段完成後，數據會說明一切。

With respect to relative benefit compared to others, of course, studies are hard to compare. And the DM result of brepo certainly is encouraging for the DM patients. But we believe that in DM, multiple modes of actions could play a role. And therefore, we will go on the strength of our own data. In any event, positive data in these diseases is always good for the patient.

當然，就與其他研究相比的相對益處而言，各項研究很難進行比較。而brepo治療DM的結果對DM患者來說無疑是令人鼓舞的。但我們認為，在決策過程中，多種行動模式可以發揮作用。因此，我們將依據我們自己的數據做出決定。總之，對於這些疾病來說，正面的數據總是對患者有利的。

Thank you, Luc. And maybe, Karl, a comment on the OpEx.

謝謝你，盧克。卡爾，或許你對營運支出（OpEx）也有一些看法。

James, thank you for the question. Yes, in 2025, we spent around $2.7 billion on combined R&D and SG&A. That is around 30%, 3-0 percent increase over 2024. Looking ahead, you can expect the combined R&D and SG&A to grow at a similar rate with most of the growth in R&D because that is where we're going to invest to set the company up for the long run, investing in our very exciting pipeline. So thank you for the question, James.

詹姆斯，謝謝你的提問。是的，到 2025 年，我們在研發和銷售、管理及行政費用上的支出總計約為 27 億美元。這大約是 30%，比 2024 年增長 3-0%。展望未來，預計研發和銷售、管理及行政費用的合併成長率將與之相近，其中大部分成長將來自研發，因為我們將把資金投入研發領域，為公司的長遠發展奠定基礎，投資於我們非常令人興奮的研發管線。謝謝你的提問，詹姆斯。

Alex Thompson, Stifel.

Alex Thompson，Stifel。

Maybe on Graves, I was wondering if you could comment on where you're at from a regulatory discussion perspective on starting the pivotal and whether you think that a single pivotal could be sufficient for an sBLA or whether you might need two studies.

關於 Graves，我想請您從監管討論的角度談談您目前對啟動關鍵性研究的看法，以及您是否認為一項關鍵性研究足以獲得補充生物製品許可申請 (sBLA)，或者是否需要兩項研究。

Yes. Thanks for the question. We're excited about our Graves program, and it's well underway. Luc, do you want to comment on our strategy around the single study?

是的。謝謝你的提問。我們對格雷夫斯計劃感到非常興奮，而且該計劃進展順利。路克，你對我們圍繞單一研究的策略有什麼看法？

Well, I mean, the ability to run a single study has sufficient evidence of efficacy and be able to define a benefit risk is actually not new. That always existed, but it was at the discretion of the individual divisions as to how much they accepted that or not. This particular division has asked us at this moment for two trials, which we are executing on.

嗯，我的意思是，能夠進行一項研究，並有足夠的證據證明其有效性，同時能夠確定獲益風險，這實際上並不是什麼新鮮事。這種情況一直存在，但各部門可以自行決定接受或不接受的程度。該部門目前要求我們進行兩項試驗，我們正在執行。

Matt Phipps, William Blair.

馬特·菲普斯，威廉·布萊爾。

Congrats on the quarter and progress here. Just wondering if you might be able to give us any more details on the auto-injector, how you can position that versus the PFS and maybe if that can just continue the continued expansion you're seeing from that PFS launch across indications.

恭喜你本季的成績和進展。我想問一下，您能否提供更多關於自動注射器的細節，您如何看待它與 PFS 的對比，以及它是否能夠延續 PFS 上市以來在適應症方面持續擴張的勢頭。

Yes. Thanks for the question. We're excited about the auto-injector, and I think it just reinforces our innovation playbook, right, just continually bringing more and more innovation as we drive leadership in the MG market. So auto-injector is on track. We have planned for 2027. And the way we've talked about it is it won't be such a step forward in the way that PFS was because if you recall, the big step forward for prefilled syringe was that we moved from HCP administration to patient administration, and that was a meaningful and important step forward for many patients, giving them the freedom to self-inject.

是的。謝謝你的提問。我們對自動噴油嘴感到非常興奮，我認為這進一步鞏固了我們的創新策略，對吧，隨著我們在MG市場保持領先地位，我們將不斷推出更多創新。所以自動噴油嘴一切正常。我們已經制定了2027年的計畫。我們討論過，它不會像 PFS 那樣是一項重大進步，因為如果你還記得的話，預充式註射器的重大進步在於我們從醫護人員給藥轉變為患者給藥，這對許多患者來說是一個意義重大且重要的進步，使他們能夠自由地進行自我注射。

So auto-injector doesn't provide that step change, but it does provide an important step, for patients that provides a better experience for those patients and especially those that are needle phobic. Actually, we mentioned earlier, we're here at the US National Field Meeting. We had a patient just yesterday that was talking to our team, and she was sharing that she wanted to wait for auto-injector because the prefilled syringe needle was something that she was a little nervous about. So it does provide value to patients, but it's not such a step forward that you should think of it as an accelerator in the way that the prefilled syringe was.

所以自動注射器雖然不能帶來質的飛躍，但確實為患者帶來了重要的一步，為患者，特別是那些害怕針頭的患者，提供了更好的體驗。事實上，正如我們之前提到的，我們現在正在參加美國國家野外考察會議。昨天我們遇到一位病人，她跟我們的團隊談過，她說她想等自動注射器，因為她對預充式註射器的針頭有點緊張。所以它確實能為患者帶來價值，但它並不是像預充式註射器那樣的一大進步，因此你不應該把它看作是加速器。

Akash Tewari, Jefferies.

阿卡什‧特瓦里，傑富瑞集團。

This is Amy on for Akash. Maybe just a quick one on your two next-gen FcRns 124 and 213. Are you seeing an accelerated path to registrational study? And can you give us a sense of how you're thinking about the indication and then the size of these trials?

這裡是艾米，替阿卡什報道。或許可以簡單問一下關於你們的兩款下一代 FcRns 124 和 213 的問題。您是否看到註冊研究的加速推進路徑？您能否簡要介紹一下您對該適應症以及這些試驗規模的看法？

Yes. Thanks for the question and the interest in our future portfolio. Maybe I can start. The way that we think about our leadership of FcRn over the coming decades is that we know there is a lot of opportunity for FcRn, in fact, probably more than we can explore with VYVGART alone.

是的。感謝您的提問以及對我們未來投資組合的關注。或許我可以開始。我們對未來幾十年領導 FcRn 的看法是，我們知道 FcRn 有很多機會，事實上，可能比我們僅憑 VYVGART 所能探索的還要多。

And so we see the opportunity of having two next-generation molecules as opening up the opportunity to provide a better patient experience in some of the indications we're already in, but also start to push the biology even further and expand the indications that we can -- that an FcRn is making a difference to patients. So I think that's the strategy that we're planning. We think each of the next-generation molecules brings -- will bring that benefit to patients. Thanks for the question.

因此，我們認為擁有兩種下一代分子的機會，不僅能為我們已經涉足的一些適應症提供更好的患者體驗，還能進一步推動生物學發展，並擴大我們能夠涉足的適應症——FcRn 正在為患者帶來改變。所以我認為這就是我們正在計劃的策略。我們認為每一種新一代分子都將為患者帶來益處。謝謝你的提問。

Yaron Werber, TD Cowen.

Yaron Werber，TD Cowen。

Congrats on the ocular study. If you don't mind, maybe a couple of questions. For EMPASSION, you switched the primary endpoint to grip strength. In the previous study in ARDA, you gave us sort of the ranges of grip strength. So maybe help us understand how is it powered? Is it superiority sort of head-to-head? What's clinically meaningful?

恭喜你通過眼科檢查。如果您不介意的話，我想問幾個問題。對於 EMPASSION，您將主要終點指標改為握力。在先前的 ARDA 研究中，您為我們提供了握力範圍的大致數據。那麼，或許您可以幫我們了解它是如何供電的呢？這是實力上的較量嗎？什麼才具有臨床意義？

And then secondly, Karen, we have a huge confidence in you, and congrats on the role. Tim, we're just -- we continue to get questions on the timing. I know, obviously, Peter is retiring as Chair. So maybe give us a little bit of a sense what drove your decision to kind of step up the chair.

其次，凱倫，我們對你充滿信心，恭喜你獲得這個職位。提姆，我們只是——我們不斷收到關於時間安排的問題。我知道，很顯然，彼得即將卸任主席一職。那麼，您能否稍微透露一下是什麼促使您決定走上領導職位？

Thanks for the questions, Yaron. I'll hand it over to Luc first to talk about EMPASSION. And then, Tim, maybe you can take the follow-up question.

謝謝你的提問，亞倫。我先把麥克風交給盧克，讓他談談熱情。然後，提姆，也許你可以回答一下後續問題。

Yes. So in fact, this is a story of growing insights in data as they matured. As we looked at ARDA and ARDA plus, so the Phase IIs, the signal we saw in grip strength gave us increasing confidence that this is really a real and patient-relevant outcome with an increasing separation over time or improvement over time, which in these patients was not seen before.

是的。所以實際上，這是一個隨著數據成熟而不斷獲得新見解的故事。當我們觀察 ARDA 和 ARDA plus（即 II 期試驗）時，我們在握力方面看到的信號讓我們越來越相信，這確實是一個真實且與患者相關的結果，隨著時間的推移，這種差異或改善越來越明顯，而這在這些患者中以前從未見過。

And that's ultimately why in discussion with agency, we started utilizing this endpoint as the primary. You asked about superiority. The study is set up as a non-inferiority study, but with a prespecified option that if noninferiority is met, that we can formally test superiority. The data will ultimately drive that tree.

最終，在與代理商討論後，我們開始將此端點作為主要端點。你問的是優越性的問題。研究被設計為非劣效性研究，但預先設定了一個選項，即如果滿足非劣效性條件，我們可以正式檢驗優效性。數據最終將驅動這棵決策樹的建構。

Thank you, Luc. And Tim?

謝謝你，盧克。蒂姆呢？

Thank you for the question. I think we're doing this transition out of a position of strength. I think now is the time to do the transition because the business and the organization is in a very healthy and a very strong state. You have seen the pipeline. You know the profitability, which we achieved during the course of last year, very strong foundation of the business.

謝謝你的提問。我認為我們是在優勢地位的基礎上進行這種轉型的。我認為現在是進行過渡的時機，因為公司和組織目前處於非常健康、非常強大的狀態。你已經看到那條管道了。您知道，我們在去年取得了獲利，這為公司奠定了非常堅實的基礎。

Also from an internal candidate point of view, we are ready. Karen knows the innovation playbook. She's a very strong carrier of the culture of the company, and she's ready to help us scale into our future because she know new therapeutic areas will come on back relatively soon. So consider this as a proactive move based on a position of strength. Thanks for the question.

從內部候選人的角度來看，我們也做好了準備。凱倫深諳創新之道。她非常堅定地傳承著公司的文化，並且她已經準備好幫助我們擴大規模，迎接未來，因為她知道新的治療領域很快就會重新出現。所以，請將此視為基於自身優勢地位的積極舉措。謝謝你的提問。

Thomas Smith, Leerink Partners.

Thomas Smith，Leerink Partners。

I was just wondering if you could provide a bit more color on the Phase IIa results for adimanebart in ALS. Obviously, really difficult indication, very complex biology. But just wondering if there are any learnings from this study that could be applied to the Phase III CMS program or potentially other indications.

我想請您詳細介紹一下 adimanebart 在 ALS 治療中的 IIa 期研究結果。顯然，這確實是一個非常難以判斷的指標，生物學特性非常複雜。但我只是想知道這項研究是否有任何經驗可以應用於第三階段 CMS 專案或其他適應症。

Yes, I'll let Luc comment on the data.

是的，我會讓盧克對數據進行評論。

Yes. Thanks for that question. Evidently not an outcome we were hoping for. We felt we had the moral obligation to explore ALS as an indication given our mode of action, trying to -- in this disease where there's very, very limited treatment options to see if we could move the dial. From our POC, the data, unfortunately, don't supports progressing, but we learned a lot, not in the least about how novel endpoints could be used, and we hope to share that knowledge with the field.

是的。謝謝你的提問。顯然，這不是我們所希望的結果。鑑於我們的行動方式，我們覺得我們有道德上的責任去探索 ALS 是否是一種適應症，因為這種疾病的治療選擇非常非常有限，我們想看看我們是否能有所作為。從我們的 POC 數據來看，遺憾的是，數據不支持繼續推進，但我們學到了很多東西，尤其是關於如何使用新的終點指標，我們希望與該領域分享這些知識。

With respect to impact and learnings on CMS, the context of treatment is fundamentally different. And CMS is directly in the biology of this molecule with the DOK-7 and other mutations affecting the mask receptor. And so that's a much more direct application of this molecule. So we don't think there's a read-through.

就對 CMS 的影響和經驗而言，治療的背景從根本上來說是不同的。CMS 直接參與此分子的生物學過程，DOK-7 和其他突變會影響掩蔽受體。所以，這是該分子更直接的應用之一。所以我們認為不存在讀通現象。

And on our SMA program, likewise, there is a backdrop of approved treatments. The gene therapies are very well established. And we are going to evaluate whether we can have an add-on efficacy there, which is a different situation than ALS altogether.

同樣，我們的 SMA 計畫也擁有一系列核准的治療方案。基因療法已經非常成熟。我們將評估我們是否能在此方面取得附加療效，這與 ALS 的情況完全不同。

Rajan Sharma, Goldman Sachs.

Rajan Sharma，高盛集團。

I had just a question on VYVGART growth dynamics through 2026. So just thinking about kind of the underlying growth outside of potential new indications, how should we think about growth across the various formulations of the drug? And if you could maybe just comment on competitive dynamics. I realize there's been a recent new approval in myasthenia gravis. Could you just talk to your confidence in the VYVGART profile and to what extent you think you may be impacted by that emerging competition?

我有一個關於 VYVGART 到 2026 年的成長動態的問題。因此，除了潛在的新適應症之外，我們還要考慮藥物本身的潛在成長，那麼我們應該如何看待該藥物各種製劑的成長呢？如果您能談談競爭格局方面的話，那就太好了。我知道最近重症肌無力領域有一項新的藥物核准。您能否談談您對 VYVGART 職業前景的信心，以及您認為這種新興競爭會在多大程度上影響您？

Great. Thanks for the question. What -- I'll provide just one comment, which is one of the things that I think is incredible five years out from launch for VYVGART is that what we're seeing is continued growth across all indications, all geographies and all product presentations. And I think that's a sign that there's space for all of the different product presentations, and it's important that we're bringing those innovations. But Sandrine, maybe I can hand over to you to talk about the growth outlook for MG and CIDP.

偉大的。謝謝你的提問。我只想補充一點，VYVGART 上市五年後，我認為最令人難以置信的一點是，我們看到它在所有適應症、所有地區和所有產品展示方面都持續成長。我認為這顯示各種不同的產品展示方式都有其存在的空間，而我們引進這些創新是非常重要的。但桑德琳，或許我可以把麥克風交給你，讓你來談談 MG 和 CIDP 的成長前景。

Yes. Thank you, Karen. And I can maybe also help answer the question on the competition but that was a second question. So I think for MG, I mean, we have seen an amazing growth year-on-year. And we have, as I mentioned earlier, healthy fundamentals. I mean, our product, VYVGART is being used earlier and earlier. I mean, as I mentioned in the opening, 70% actually are coming from orals.

是的。謝謝你，凱倫。我或許還能幫忙回答關於比賽的問題，但那是第二個問題。所以我覺得對MG來說，我們已經看到了驚人的逐年成長。正如我之前提到的，我們擁有健康的基本條件。我的意思是，我們的產品 VYVGART 的使用時間越來越早了。我的意思是，正如我在開頭提到的，70% 的分數實際上來自口試。

And then on VYVGART, when you have 10 patients coming on biologics, 6 of them are actually starting with VYVGART. So this shows that this is the molecule that patients start on when they are starting on biologics. PFS is the one that has been driving and helping us drive strong growth last year. And as Karen just mentioned, we expect to continue to grow across all mode of administration, including PFS. And then the label expansion, of course, is going to help us this year, starting with seronegative.

然後，在 VYVGART 治療中，當有 10 名患者接受生物製劑治療時，其中 6 名患者實際上是從 VYVGART 開始的。所以這表明，這是患者開始接受生物製劑治療時首先使用的分子。PFS是去年推動並幫助我們實現強勁成長的關鍵因素。正如 Karen 剛才提到的，我們預計在所有管理模式（包括 PFS）中將繼續成長。當然，標籤的擴展今年肯定會對我們有所幫助，首先是血清陰性。

If you look at CIDP, I mean, we have -- we are still early in launch. So we have launched roughly 1.5 years ago, and we are still have a lot of room within the 12,000 patients that are not fully well managed. And beyond that, we are working very, very hard to lift any challenges either with payers or the inertia of prescribers to start earlier with VYVGART. So overall, very strong dynamics expected for this year like we had in the prior years.

如果你看看CIDP，我的意思是，我們——我們仍然處於早期階段。我們已經啟動這項服務大約一年半了，目前仍有 12,000 名患者尚未得到充分的管理，我們還有很多空間可以改進。除此之外，我們正在非常非常努力地克服支付方或處方醫生在儘早開始使用 VYVGART 方面遇到的任何挑戰。所以總的來說，預計今年的經濟狀況將非常強勁，就像前幾年一樣。

So now going to your question on the competition. Actually, we welcome competition. For me, this is -- and for us, this is a sign of progress, and this is a sign of innovation, and that's great for patients to have multiple options. This expands the overall market and VYVGART benefits from a market expansions. I just mentioned that 6 out of 10 patients starting on biologics go on VYVGART. So the more the market expands, the better it is for us.

現在來回答你關於比賽的問題。實際上，我們歡迎競爭。對我而言，這——對我們來說，這都是進步的標誌，也是創新的標誌，這對患者來說是一件好事，因為他們可以有多種選擇。這擴大了整體市場，VYVGART 也從市場擴張中受益。我剛剛提到，10位開始接受生物製劑治療的患者中有6位會接受VYVGART治療。所以市場規模越大，對我們就越有利。

And as we are a data-driven company, all the data we have generated support our confidence that VYVGART profile will help us continue leading that category and remain the number one prescribed biologics in MG from an efficacy viewpoint, and we are the only one that can really show the strong MSE, the robust safety that fosters earlier use, the ability to meaningfully reduce steroids and then as we mentioned, multiple flexibility on either IV subcutaneous or PFS. So when you take that all together, I mean, we believe that we have a very, very strong profile for continuing our leadership there.

作為一家數據驅動型公司，我們產生的所有數據都支持我們的信心，即 VYVGART 的特性將幫助我們繼續引領該類別，並從療效角度保持 MG 生物製劑處方量第一的地位。我們是唯一一家能夠真正展示強大的 MSE、促進早期使用的可靠安全性、顯著減少類固醇的能力，以及正如我們所提到的，靜脈注射、皮下注射或 PFS 等多種給藥方式的公司。所以綜合所有因素來看，我們認為我們擁有非常非常強大的實力，能夠繼續保持我們在該領域的領先地位。

Great. Thanks, Sandrine.

偉大的。謝謝你，桑德琳。

(Operator Instructions)

（操作說明）

Sean Laaman, Morgan Stanley.

肖恩拉曼，摩根士丹利。

This is Morgan on for Sean. Maybe one on the financials. So with VYVGART delivering $4.2 billion this year in net sales and 90% year-over-year growth resulting in the first year of operating profitability. How should we think about the sustainability of this growth profile as penetration deepens in MG and CIDP throughout this year and next year?

這裡是摩根替肖恩發言。或許可以寫一篇關於財務方面的文章。因此，VYVGART 今年的淨銷售額達到 42 億美元，年增 90%，實現了第一年的營運獲利。隨著MG和CIDP的滲透率在今年和明年不斷加深，我們該如何看待這種成長模式的可持續性？

Yes. Thanks for the question. Karl, maybe you want to talk about the growth profile?

是的。謝謝你的提問。卡爾，或許你想談談成長概況？

Yes. I think what we're building here is a long-term sustainable business, as Sandrine already mentioned, we see a lot of growth in MG and CIDP going forward. And the way we look at the financials long term is that revenue growth should exceed OpEx growth, which basically will return operating margins, which will increase over time. That in itself, of course, is not the objective of the company.

是的。我認為我們正在打造的是一個長期可持續發展的業務，正如 Sandrine 已經提到的那樣，我們看到 MG 和 CIDP 未來將會有很大的成長。從長遠來看，我們認為收入成長應該超過營運支出成長，這基本上會提高營運利潤率，而營運利潤率會隨著時間的推移而提高。當然，這本身並不是公司的目標。

We have very clear capital allocation priorities, and we're executing on those priorities. But what we should see is that we're going to build on our very strong balance sheet. We currently have $4.4 billion of cash in the bank. And going forward, that number should increase. So I think you can look forward to a long-term sustainable and profitable business. Thank you for your question, Morgan.

我們有非常明確的資本配置優先事項，並且正在執行這些優先事項。但我們應該看到的是，我們將鞏固我們非常強勁的資產負債表。我們目前銀行帳戶裡有44億美元的現金。展望未來，這個數字應該會增加。所以我認為你可以期待一個長期可持續且有利可圖的業務。謝謝你的提問，摩根。

Sophia Graeff Buhl Nielsen, JPMorgan.

索菲亞·格雷夫·布爾·尼爾森，摩根大通。

So just on the Phase III readout for VYVGART in myositis, could you clarify, would you have data to support approval by subgroup? Or will this largely be dependent on the overall data? I think you've been very clear on that the high unmet need within IMNM and the large patient population that could be addressed there and also the heterogeneity within DM. Given these dynamics, would you see these as relatively equally sized commercial opportunities despite the differences in addressable TAMs you've highlighted?

那麼，關於 VYVGART 治療肌炎的 III 期臨床試驗結果，您能否澄清一下，是否有數據支持按亞組進行審批？或者，這主要取決於整體數據？我認為您已經非常清楚地指出了IMNM領域存在著巨大的未滿足需求，以及可以解決的大量患者群體，以及DM領域的異質性。鑑於這些動態，儘管您強調了潛在市場規模的差異，您是否認為這些商業機會的規模相對相當？

Yes. Thanks for the question. Maybe, Luc, you can talk to the basket trial and our approach, and then I can comment on the commercial opportunity.

是的。謝謝你的提問。或許，盧克，你可以談談籃子試驗和我們的方法，然後我再對商業機會發表意見。

Yes. So the Phase III setup is indeed that we can make statements on all three subsets. And of course, the ultimate reflection of that in label will be connecting the data with the regulatory discussion. But in principle, all three could be in scope.

是的。因此，第三階段的設定確實是我們可以對所有三個子集發表聲明。當然，最終在標籤上體現這一點的，是將數據與監管討論聯繫起來。但原則上，這三者都可能在範圍內。

Thank you, Luc. And then in terms of the commercial opportunity, the way I think about it, I mean, the total myositis population that we're studying, we think about in terms of it being an MG-like opportunity. But obviously, there are other subtypes. And I like to think about the two bookends of the subtypes.

謝謝你，盧克。至於商業機會，我的想法是，我們正在研究的整個肌炎患者群體，我們認為這是一個類似重症肌無力的機會。但顯然，還有其他亞型。我喜歡思考這兩種子類型的兩端。

So we talked -- you mentioned IMNM. So IMNM, there are no other approved treatment options. There's about 20,000 IMNM patients. So what you can imagine there is that from a commercial perspective, you could imagine that we'll be able to gain a high portion of those patients because there are no other treatment options and the biology is so clear.

於是我們聊了一下——你提到了IMNM。因此，對於IMNM，目前沒有其他核准的治療方案。約有 20,000 名 IMNM 患者。所以你可以想像，從商業角度來看，我們可以想像我們將能夠獲得很大一部分患者，因為沒有其他治療選擇，而且生物學原理非常明確。

On the other end of the spectrum, you can think of DM. There are more patients in DM, but it's also more heterogeneous in dermatomyositis. There's also more innovation coming to the dermatomyositis space. So that will grow that patient population. Innovation is good for patients. And I think let's see the data, how it reads out, but I think we should have a good value proposition to be able to compete in that population if the data reads out. So overall, total population MG-like, but the subgroups provide quite different dynamics from a commercial perspective. Thanks for the question.

另一方面，你可以考慮 DM。DM 患者較多，但皮肌炎的異質性也較高。皮肌炎領域也將迎來更多創新。這樣一來，患者群體就會增加。創新對患者有益。我認為我們應該看看數據，看看結果如何，但如果數據顯示我們擁有良好的價值主張，我們就能夠在該群體中競爭。因此，整體而言，總人口類似於 MG，但從商業角度來看，各個亞群體呈現出截然不同的動態。謝謝你的提問。

Suzanne van Voorthuizen, Kempen.

蘇珊娜·範·沃特赫伊森，肯彭。

It's one on empa and MMN in particular. There was a change in the dosing regimen between the Phase II and III and the Phase III is also head-to-head. Could you elaborate on how you navigate the potential risks that these two changes introduce? What gives you comfort that the study can confirm empa's non-inferiority? And I'm also wondering if you can give some color on how you went about setting the non-inferiority margin in this progressive disease?

其中一篇是關於 empa 和 MMN 的。第二階段和第三階段的給藥方案有所不同，第三階段也是頭對頭試驗。您能否詳細說明您如何應對這兩項變化可能帶來的潛在風險？是什麼讓您確信這項研究能夠證實empa的非劣效性？我還想請教一下，您能否詳細說明一下，在這種進行性疾病中，您是如何設定非劣效性界值的？

Thank you. I think that's for you, Luc.

謝謝。我覺得那是給你的，路克。

Yes. Thanks for that question. And I can tell you a lot of thought went into that based on the data again from ARDA, where we tested multiple regimens, and we're able to model and look at an exposure response relationship, which ultimately made us choose the dose regimen we went for.

是的。謝謝你的提問。我可以告訴你，我們根據 ARDA 的數據對此進行了很多思考，我們測試了多種方案，並能夠建立模型並觀察暴露反應關係，這最終促使我們選擇了我們採用的劑量方案。

In terms of then choosing or choosing to go head-to-head, here, the thinking was if we were taking placebo-controlled study, we could have a lot of events because people on placebo in this progressive disease, as you say, will need rescue. And therefore, we said, well, why not just do them straight head-to-head? So that was that decision.

至於選擇還是進行正面交鋒，當時的想法是，如果我們進行安慰劑對照研究，可能會出現很多事件，因為正如你所說，在這種進行性疾病中，服用安慰劑的人需要救助。因此，我們說，為什麼不讓他們直接正面交鋒呢？這就是當時的決定。

The second one, how do you determine a non-inferiority margin? And this is actually also where the data on grip strength come in because the only available data on IVIg are on grip strength. So that's why we use that measure to determine the non-inferiority margin. Given the data we see from ARDA, one of the features that is different is that we continuously seem to improve grip strength, something which is not seen in the experience with IVIg. And that gives us confidence that we can at least meet but hopefully beat IVIg.

第二個問題，如何確定非劣效界值？實際上，握力數據也與此相關，因為目前唯一可用的靜脈注射免疫球蛋白數據就是握力數據。所以，這就是我們使用這種衡量標準來確定非劣效性界值的原因。根據我們從 ARDA 中看到的數據，其中一個不同的特徵是，我們的握力似乎一直在提高，這是在 IVIg 的經驗中沒有看到的。這讓我們有信心至少可以達到 IVIg 的水平，但希望能超越它。

That's great. Thanks, Luc. And when you zoom out, I think what you can see with your answer is the approach that we take for -- with our programs, strong biology rationale, derisking with a Phase II. And I think we're well positioned for success commercially with this head-to-head data that in the way that you've laid it out. So look forward to that data in Q4. Thanks, Luc.

那太棒了。謝謝你，盧克。當你從更宏觀的角度來看，我認為從你的回答中可以看出我們採取的方法——透過我們的項目，強有力的生物學原理，透過二期臨床試驗降低風險。我認為，根據你剛才列出的這些直接比較數據，我們在商業上已經處於非常有利的地位，有望取得成功。所以請期待第四季的數據。謝謝你，盧克。

Allison Bratzel, Piper Sandler.

艾莉森‧布拉澤爾，派珀‧桑德勒。

Just a follow-up on ocular MG and the potential for early treatment with VYVGART to prevent progression to generalized disease. Is that something you're able to capture in Part B of the oculus trial? Or just how long of a follow-up do you need to confidently be able to make that claim? Just any more color there would be appreciated.

本文旨在跟進眼肌型重症肌無力 (MG) 的病情，並探討早期使用 VYVGART 治療以防止病情發展為全身性疾病的可能性。這是你能在 Oculus 試用版 B 部分捕捉到的內容嗎？或者，你需要多長時間的後續跟進才能自信地做出這樣的聲明？如果能多一些色彩就更好了。

Yes. Thanks for that question to allow me again to go on one of my favorite topics, which is can we slow MG progression. So the open-label extension following Stage B, which we call Stage B, is going to give us over two years of data, which if you look at extent epidemiological data, et cetera, should give us enough window to capture these people progressing and whether or not it's to the rate that's there in the outside world.

是的。感謝您提出這個問題，讓我有機會再次探討我最喜歡的話題之一，那就是我們能否減緩重症肌無力的進展。因此，在 B 階段之後的開放標籤擴展研究（我們稱之為 B 階段）將為我們提供兩年多的數據，如果你查看流行病學數據等，應該能給我們足夠的時間來捕捉這些人的病情進展情況，以及他們的病情進展速度是否與外界的進展速度相同。

The caveat, of course, is this is noncontrolled data. So any expression of this delaying might have to be in a publication or if the real-world evidence is deemed strong enough in a discussion with the agency.

當然，需要注意的是，這些數據未經控制。因此，任何關於拖延的表態都可能需要發表在出版物上，或者在與相關機構的討論中，如果現實世界的證據被認為足夠有力，也可以進行表態。

Yes. I think that's what's exciting about this data, along with some of the other evidence generation that we're doing, a Phase IV study in early disease to be able to see that progression. But I think regardless, one thing that's important is with ocular MG is the symptom burden is significant and the opportunity to transform lives of patients suffering from ocular MG is significant even without the disease progression. So I think we can demonstrate that benefit in the short and the long term. Thanks, Luc.

是的。我認為，這些數據令人興奮之處在於，我們正在進行的其他一些證據產生工作，例如針對早期疾病的 IV 期研究，以便能夠看到疾病的進展。但我認為無論如何，對於眼肌型重症肌無力來說，有一點很重要，那就是症狀負擔很重，即使疾病沒有進展，改變眼肌型重症肌無力患者生活的機會也很大。所以我認為我們可以證明，無論從短期或長期來看，這都是有益的。謝謝你，盧克。

Luca Issi, RBC Capital.

Luca Issi，RBC Capital。

Congrats on the progress. Maybe, Luc, if I could circle back on ocular myasthenia gravis here. Again, I appreciate this is a fresh off the press, but how should we think about the kind of clinical significance of the data here, again, in the context of the p-value of 0.012.

恭喜你取得進展。或許，盧克，我可以再談談眼肌型重症肌無力嗎？我再次意識到這是最新發表的，但是我們應該如何看待這裡的數據在臨床上的意義呢？尤其是在 p 值為 0.012 的情況下。

And then maybe related to it, can you confirm the use of steroids or other therapy was relatively well balanced between the two arms, so we can kind of definitively say that the benefit here is coming from VYVGART and is not confounded by any other therapies?

另外，或許可以確認一下，兩組患者使用類固醇或其他療法的比例是否相對均衡，這樣我們才能明確地說，這種益處來自 VYVGART，而不是其他療法造成的混淆？

Yes. Thanks for that question. And of course, we don't share too many detailed data because we want to make sure that the representation in an external conference isn't impacted by doing so. But to come back to the -- yes, we have significant p-value, but that was driven by, in our mind, a very relevant treatment difference between active and placebo.

是的。謝謝你的提問。當然，我們不會分享太多詳細數據，因為我們希望確保這樣做不會影響我們在外部會議上的演講。但回到正題——是的，我們的 p 值很大，但我們認為這是由活性藥物和安慰劑之間非常相關的治療差異所驅動的。

Remember, these endpoints range is between 0 to 18 and to show an active 4-point difference for that individual patient is certainly a relevant outcome. So we feel that in totality, this is a meaningful signal that we have shown. And with respect to balancing on steroids, steroids were allowed but had to be stable. And we are confident that there's no imbalance in the outcome based on anything there.

請記住，這些終點的範圍是 0 到 18，對於該患者而言，4 分的積極差異無疑是一個相關的結果。因此，我們認為，總的來說，我們已經發出了一個有意義的信號。至於使用類固醇保持平衡的問題，類固醇是被允許的，但必須保持穩定。我們確信，基於那裡的任何因素，結果都不會出現不平衡。

And maybe just to add to your question on clinical significance. I mean, Luc mentioned in the script, what -- when you think about what the impact that ocular MG has, what patients will tell you is that it strips them of their independence. They lose -- because of the double vision, they lose the ability to drive, they lose the ability to work. And so it has a real impact on their quality of life.

或許我還可以補充一點關於臨床意義的問題。我的意思是，盧克在劇本中提到過，當你思考眼肌型重症肌無力的影響時，患者會告訴你，它剝奪了他們的獨立性。由於複視，他們會失去駕駛能力和工作能力。因此，這確實對他們的生活品質產生了影響。

So there's no other treatments available other than steroids. So any benefit that we can provide and certainly this a 4-point benefit that we've seen is demonstrable benefit for patients and I think clinically very meaningful.

所以除了類固醇之外，沒有其他治療方法。因此，我們能夠提供的任何益處，當然包括我們已經看到的這 4 點益處，對患者來說都是可證明的益處，我認為在臨床上非常有意義。

Justin Smith, Bernstein.

賈斯汀·史密斯，伯恩斯坦。

Just a very quick one, if you could talk about the commentary with regards to switching off subcutaneous Ig on to VYVGART and how that's changed over the last three months?

問個簡單的問題，您能否談談關於從皮下注射免疫球蛋白轉而使用 VYVGART 的說明，以及過去三個月來這方面的變化？

Yes, I'm happy to. Well, I think what you're asking about is there was an -- FDA looking into real-world evidence around switching and CIDP worsening? Actually, we had good news that we have completed that review and the label has been updated with some helpful guidance to HCPs around when switching from IVIg to VYVGART. So I think we're well positioned moving forward. That label update reinforces what we knew from ADHERE and reinforces the risk-benefit profile of VYVGART. Thanks for the question.

是的，我很樂意。我想你問的是，FDA是否正在調查有關轉換治療方案和CIDP病情惡化的真實世界證據？事實上，我們有好消息，我們已經完成了審查，標籤也已更新，其中包含一些對醫療保健專業人員在從 IVIg 切換到 VYVGART 時有用的指導。所以我認為我們向前邁進處於有利地位。此標籤更新強化了我們從 ADHERE 中了解到的訊息，並強化了 VYVGART 的風險效益概況。謝謝你的提問。

Samantha Semenkow, Citi.

Samantha Semenkow，花旗銀行。

Just one on the ocular MG opportunity. I'm wondering, can you speak to the mix of treating physicians that you're expecting for this patient population? Are they mainly managed by neurologists, ophthalmologists or even neuro-ophthalmologists? And I'm wondering how much education you think you need on the opportunity to drive VYVGART utilization in this segment?

眼部MG機會只有一次。我想請問，您能否談談您預計會有哪些類型的醫生來治療這部分患者群？這些疾病主要由神經科醫師、眼科醫師或神經眼科醫師治療？我想知道，您認為您需要接受多少教育才能有機會推動 VYVGART 在這個領域的應用？

Yes. Thanks for the question. Maybe, Sandrine, you can talk about that and also related to seronegative because we have the PDUFA date coming up in May. And just is there -- are there any changes for our field or the targeting strategy with this label -- with these potential label expansions?

是的。謝謝你的提問。桑德琳，或許你可以談談這個，也可以談談血清陰性，因為我們五月就要迎來 PDUFA 日期了。那麼，這些潛在的標籤擴展是否會對我們的領域或針對該標籤的目標策略產生任何影響？

That's a great question. Actually, we have a big overlap between the current prescribers and the target group we are visiting and the people that will be prescribing for MG in both seronegative and ocular. So it's mostly a neurologist-driven disease. So we don't expect to have to change our footprint.

這是一個很好的問題。實際上，我們目前的處方醫生和我們正在拜訪的目標群體，以及將要為血清陰性和眼部 MG 患者開處方的醫生之間存在很大的重疊。所以這主要是一種由神經科醫師主導的疾病。因此，我們預計無需改變我們的佔地面積。

And actually, we increased our footprint early 2024. If you remember, we doubled our footprint to be able to not only target academic medical centers, but then to also be able to visit the community neurologists where we feel the majority of the patients are being taken care of. So you won't see a change of our approach there. And with the big overlap, we're confident we can target the majority of the potential and the prescribers.

事實上，我們在 2024 年初就擴大了業務規模。如果你還記得的話，我們擴大了業務範圍，不僅是為了能夠服務學術醫療中心，也是為了能夠拜訪社區神經科醫生，因為我們認為大多數患者都是在那裡接受治療的。所以你不會看到我們在這方面的做法有所改變。由於重疊部分很大，我們有信心鎖定大多數潛在患者和處方醫生。

Victor Floch, BNP Paribas.

Victor Floch，法國巴黎銀行。

One question on ARGX-213. So I believe the last time you've updated us on time lines where you were pointing out Phase I results sometimes in H1 this year. So I was just wondering whether we should expect you to discuss those data or to a broader extent, your -- like the development program of that product later this year.

關於ARGX-213的一個問題。所以我相信，你上次向我們更新時間表的時候，你提到第一階段的結果有時會在今年上半年公佈。所以我想知道我們是否應該期待您在今年稍後討論這些數據，或者更廣泛地說，討論您的——比如該產品的開發計劃。

Yes. Thanks for the question. And again, the interest in our next-gen. We are excited. So we're moving forward with 213, and we've shared that update previously, and it is in the clinic. We're working on the indication strategy at the moment and our path forward, and we'll certainly share that when we have an update to share. Thanks for the question.

是的。謝謝你的提問。再次強調，我們對下一代產品很感興趣。我們很興奮。所以我們正在推進 213 號方案，之前我們已經分享過這個最新進展，目前正在臨床上進行測試。我們目前正在研究適應症策略和未來的發展方向，一旦有新的進展，我們一定會與大家分享。謝謝你的提問。

Douglas Tsao, H.C. Wainwright.

道格拉斯·曹，H.C. 溫賴特。

Just on oMG as a follow-up, we have heard from clinicians who have tried to use it in patients presenting with ocular symptoms, but they've had pushback from payers just given the fact it wasn't sort of on label. I'm just curious if you could provide some perspective on when you think it might start to become a contributor?

關於 oMG 的後續報導，我們聽到一些臨床醫生嘗試將其用於出現眼部症狀的患者，但由於它並非在說明書中明確列出，因此遭到了支付方的抵制。我只是好奇，您能否就它何時可能開始成為​​貢獻者提供一些看法？

Will it be sort of getting it added to the label? Or will there be a process where you need to also then talk to payers? Just sort of trying to understand the sequencing when we should think about this because it does seem to be a fairly meaningful commercial opportunity for you.

這會不會算是把它加到標籤上？或是否還需要與付款方溝通？我們只是想了解一下應該在什麼情況下考慮這件事，因為這似乎對你來說是一個相當有意義的商業機會。

Yes. Thank you. And I agree it is a meaningful opportunity and a meaningful benefit for patients. So what you can expect, I mean, we'll file as soon as we can based on this data, and I think we have a well-oiled machine. So we'll do that as soon as possible, and then we'll see when the PDUFA date is, assuming the submission is accepted by the FDA.

是的。謝謝。我同意這對患者來說是一個有意義的機會和有意義的益處。所以你們可以期待什麼呢？我的意思是，我們會根據這些數據盡快提交，而且我認為我們已經運作良好。所以我們會盡快完成這項工作，然後看看 PDUFA 日期是什麼時候，前提是 FDA 接受了我們的申請。

What we normally guide to is because we will need to have conversations with payers, and we will need to change those contracts. What we usually guide to is that it takes about two quarters after approval to get those payer policies in place and to really start to see the impact of the opportunity. So we'll take it step by step. And step number one will be preparing the filing as quickly as possible. Thank you.

我們通常的做法是，因為我們需要與付款方進行溝通，並且需要修改這些合約。我們通常建議，從批准到落實這些支付方政策，大約需要兩個季度的時間，才能真正開始看到這個機會的影響。所以我們會一步一步來。第一步就是盡快準備好文件。謝謝。

And this concludes today's conference call. We thank you for your participation. You may now disconnect.

今天的電話會議到此結束。感謝您的參與。您現在可以斷開連線了。