argenx SE (ARGX) 2024 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.我叫羅布，今天我將擔任您的會議主持人。我歡迎大家參加電話會議。（操作員指令）

Thank you. I'd now like to introduce Beth DelGiacco, Vice President, Corporate Communications and Investor Relations. You may begin your conference.

謝謝。現在我想介紹企業傳播與投資人關係副總裁 Beth DelGiacco。您可以開始您的會議了。

Thank you. A press release was issued earlier today with our fourth quarter and full year 2024 financial results and business update. This 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一份新聞稿，其中包含我們第四季度和 2024 年全年的財務業績和業務更新。您可在我們的網站上找到此內容以及今天的網路廣播的簡報。

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

在我們開始第二張投影片之前，我想提醒您，本次電話會議中可能會出現前瞻性陳述。這可能包括關於我們未來期望、臨床開發、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 these statements in relation to actual results unless required by law.

實際結果可能與這些陳述所示的結果有重大差異。除非法律要求，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 Tim Van Hauwermeiren, Chief Executive Officer; Karl Gubitz, Chief Financial Officer; and Karen Massey, Chief Operating Officer.

今天與我一起參加電話會議的還有執行長 Tim Van Hauwermeiren； Karl Gubitz，財務長；以及營運長 Karen Massey。

I will now turn the call over to Tim.

現在我將電話轉給提姆。

Thank you, Beth, and welcome, everyone. I'll begin on Slide number 3. 2024 was a phenomenal year for argenx. We expanded our reach to over 10,000 patients globally across three approved indications and delivered significant impacts to the CIDP community early into launch.

謝謝你，貝絲，歡迎大家。我將從幻燈片第 3 號開始。 2024 年對 argenx 來說是非凡的一年。我們將涵蓋範圍擴大到全球超過 10,000 名患者，涉及三個核准適應症，並在推出初期就對 CIDP 社群產生了重大影響。

We also achieved several key clinical milestones. Empasiprubart the ranks of efgartigimod as a Phase III asset following impressive MMN data, and we successfully advanced efgartigimod into larger registration studies following go-decisions in Sjogren’s disease and three subsets of myositis. Lastly, we continue to drive innovation, identifying additional level targets and nominating 4 pipeline molecules.

我們也實現了幾個關鍵的臨床里程碑。在獲得令人印象深刻的 MMN 數據後，我們確認 efgartigimod 為 III 期資產，並且在乾燥綜合徵和三種肌炎亞型的治療決策之後，我們成功地將 efgartigimod 推進到更大規模的註冊研究中。最後，我們繼續推動創新，確定額外的水平目標並提名 4 個管道分子。

These accomplishments have laid a robust foundation for our continued momentum into 2025. As we execute across the priorities highlighted on this slide towards our Vision 2030, we remain committed to maximizing the growth opportunities ahead of us in a data-driven way, always prioritizing transformational impact for patients.

這些成就為我們邁向2025年的持續發展奠定了堅實的基礎。當我們執行這張投影片中強調的優先事項以實現我們的 2030 願景時，我們仍然致力於以數據驅動的方式最大限度地利用我們未來的成長機會，始終優先考慮對患者的轉型影響。

Slide number 4. Let's begin with the commercial opportunity. VYVGART has meaningfully shaped the gMG treatment landscape by setting these standards for rapid, deep and sustained efficacy with favorable safety and without trade-offs on convenience.

投影片編號 4。讓我們從商業機會開始。VYVGART 透過制定這些標準，實現了快速、深度和持續的療效，同時具有良好的安全性，並且不會犧牲便利性，從而顯著改變了 gMG 治療格局。

We look forward to continuing the growth momentum with multiple regulatory milestones and continued data generation supporting earlier use across the treatment paradigm. The prefilled syringe, which opens the door for self-administration in the US, is expected to be a key driver of this growth in 2025 for both MG and CIDP.

我們期待透過多個監管里程碑和持續的數據生成來繼續保持成長勢頭，以支持整個治療模式的早期使用。預充式註射器為美國的自我給藥打開了大門，預計將成為 2025 年 MG 和 CIDP 成長的主要驅動力。

It is incredible to see the strong early adoption of VYVGART Hytrulo in CIDP patients, underpinning the real unmet need that still exists. In bringing the first major innovation in treatment to CIDP patients in over 30 years, we not only had a significant opportunity before us to set a new treatment standard, but also an important responsibility with the long-term commitment we made to the CIDP community.

令人難以置信的是，VYVGART Hytrulo 在 CIDP 患者中得到了廣泛的早期採用，這鞏固了仍然存在的真正未滿足的需求。在為 CIDP 患者帶來 30 多年來首次重大治療創新時，我們不僅有重大機會制定新的治療標準，而且也肩負著對 CIDP 社群做出長期承諾的重要責任。

We will work to continue delivering transformative impact with VYVGART by executing on our launch strategies while generating new evidence to support physician treatment decisions and advance our understanding of the underlying biology of this complex disease.

我們將繼續努力透過 VYVGART 實現變革性影響，執行我們的發布策略，同時產生新證據來支持醫生的治療決策並加深我們對這種複雜疾病的潛在生物學的理解。

This commitment informs our decision to run the IVIg switch study soon after launch to help inform treatment decisions, especially with recognition that over 80% of patients have IVIg experience. It was also the impetus to advance our first-in-class C2 inhibitors into a registration study in CIDP. We are confident that we have a unique opportunity to drive meaningful impact across two molecules.

這項承諾促使我們決定在推出後不久進行 IVIg 轉換研究，以幫助指導治療決策，特別是認識到超過 80% 的患者都有 IVIg 經驗。這也是推動我們首創的 C2 抑制劑進入 CIDP 註冊研究的動力。我們相信，我們擁有獨特的機會在兩個分子之間產生有意義的影響。

Slide 5. Our disciplined approach to scaling will be critical to navigating the full opportunity across our pipeline. This year, we look forward to advancing 10 Phase III studies and 10 proof-of-concept studies across efgartigimod, empasiprubart and ARGX-119.

幻燈片 5。我們嚴謹的擴展方法對於充分利用我們的整個通路機會至關重要。今年，我們期待推進 efgartigimod、empasiprubart 和 ARGX-119 的 10 項 III 期研究和 10 項概念驗證研究。

We are leading a new field of medicine in FcRn pushing the boundaries of its potential with the broadest development plan. Our registrational studies in myositis, [EED], Sjogren’s and ITP are built on established proof of concepts with additional proof-of-concept studies in systemic sclerosis, AMR and lupus nephritis running in parallel.

我們正在引領FcRn醫學的新領域，以最廣泛的發展計劃突破其潛力的界限。我們對肌炎、[EED]、乾燥症和 ITP 的註冊研究建立在已建立的概念驗證基礎之上，同時也在系統性硬化症、AMR 和狼瘡性腎炎方面開展了額外的概念驗證研究。

Each of these is grounded in solid biology with the potential to drive meaningful impact in high unmet need areas. Our first in Class 2 inhibitors, empasiprubart, is following the same innovation playbook as efgartigimod. The Phase II adult data in MMN were impressive with 94% of patients reporting improvements on empa compared to IVIg.

上述每一個都以紮實的生物學基礎為基礎，並有可能在尚未滿足的高需求領域產生有意義的影響。我們的第一個 2 類抑制劑 empasiprubart 遵循與 efgartigimod 相同的創新策略。MMN 第二階段成人數據令人印象深刻，94% 的患者報告 empa 與 IVIg 相比有所改善。

We see an opportunity to disrupt a blockbuster market bringing transformative benefits to patients with MMN and now CIDP as well, which is why we are exploring both indications in registrational head-to-head studies versus IVIg. And this is just scratching the surface of the broad potential opportunity ahead for empa to bring the same treatment option forward in diseases for the lectin and classical pathways at play.

我們看到了顛覆重磅藥物市場的機會，該市場將為 MMN 患者和現在的 CIDP 患者帶來變革性益處，這就是為什麼我們在註冊頭對頭研究中探索這兩種適應症而不是 IVIg。而這只是觸及了 empa 未來廣泛潛在機會的表面，該技術可以為凝集素和經典途徑相關的疾病帶來同樣的治療選擇。

Lastly, we look forward to the first proof-of-concept data from our third molecule, ARGX-119, in CMS by co-creating with the world experts in MuSK must biology. We have built a molecule that has the potential to alleviate diseases powered by [inter neuromuscular] synaptic function. The CMS data in the second half of the year is the first opportunity to assess whether ARGX-119 is doing what it is supposed to do clinically, which will further help us assess the opportunity ahead in SMA and ALS.

最後，我們期待與 MuSK 必需生物學領域的全球專家共同創造，在 CMS 中獲得第三個分子 ARGX-119 的第一個概念驗證數據。我們已經建構了一種分子，它有可能緩解由[神經肌肉間]突觸功能驅動的疾病。今年下半年的 CMS 數據是首次評估 ARGX-119 是否在臨床上發揮應有的作用，這將進一步幫助我們評估未來在 SMA 和 ALS 領域的機會。

Slide 6. We had a robust state of activity in our IIP, nominating four new molecules that we are now advancing into Phase I development. These molecules including a second FcRn inhibitor, ARGX-213, a sweeping IgA antibody and an anti-IL-6 all have significant therapeutic potential across new autoimmune indications.

幻燈片 6。我們的 IIP 活動非常活躍，我們提名了四種新分子，目前正在推進到 I 期開發。這些分子包括第二種 FcRn 抑制劑 ARGX-213、一種廣譜 IgA 抗體和一種抗 IL-6，在治療新的自體免疫疾病方面都具有巨大的治療潛力。

Our IIP is an incredible productive innovation engine, and we have nearly 20 active programs in the discovery stages across many relevant disease areas that will continue to feed our future pipeline.

我們的 IIP 是一個令人難以置信的高效創新引擎，我們在許多相關疾病領域擁有近 20 個處於發現階段的活躍項目，這些項目將繼續為我們未來的研發管線提供資訊。

2025 will be our first year as a profitable company, which is an important achievement and a reflection of our commercial success, our relentless execution and our commitment to scale innovation in a disciplined way. We have a long-term growth vision that is directly influenced by our ability to continue to innovate, to stay ahead of competition and to leverage our financial strength by investing in mobile targets and pipeline programs that will be the most impactful to patients. This is the future we are building with Vision 2030 and well beyond.

2025 年將是我們公司實現盈利的第一年，這是一項重要成就，也是我們商業成功、不懈執行以及以規範方式擴大創新規模的承諾的體現。我們有一個長期的成長願景，這個願景直接受到我們持續創新、保持競爭優勢以及透過投資對患者影響最大的移動目標和管道項目來利用我們的財務實力的能力的影響。這就是我們透過「2030願景」及更長遠的未來所建構的未來。

I will now turn the call over to Karl to discuss our financial position in greater detail.

現在我將把電話轉給卡爾，更詳細地討論我們的財務狀況。

Thank you, Tim. Slide 7. The fourth quarter and full year 2024 financial results are detailed in the press release of this morning.

謝謝你，提姆。幻燈片 7。2024 年第四季和全年財務業績已在今天上午的新聞稿中詳細說明。

Product net sales are consistent with our preannouncement in January at $737 million for Q4 and $2.2 billion for the full year. This brings total operating income in the fourth quarter to $761 million and $2.3 billion for the full year.

產品淨銷售額與我們 1 月的預告一致，第四季為 7.37 億美元，全年為 22 億美元。這使得第四季的總營業收入達到 7.61 億美元，全年總營業收入達到 23 億美元。

The product net sales represents 29% quarter-over-quarter growth and 98% growth compared with the same quarter from the prior year. The product revenue breaks down by region to $649 million in the US, $27 million in Japan, $49 million in the rest of the world and $12 million in product supplied to Zai Lab in China.

該產品淨銷售額較上季成長29%，較去年同期成長98%。按地區劃分，產品收入分別為美國 6.49 億美元、日本 2,700 萬美元、世界其他地區 4,900 萬美元以及向中國再鼎醫藥供應的產品收入 1,200 萬美元。

Gross to net in the US continues to be around 12%. In 2025, we expect the dynamics will change due to self-administration, resulting in an increase in gross to net, which will be offset by increased patient numbers.

美國毛利率與淨利率比率仍維持在 12% 左右。我們預計，到 2025 年，由於自我管理，動態情況將發生變化，導致總支出增加到淨支出，而這一增長將被患者數量的增加所抵消。

Next slide. Cost of sales were $73 million in Q4 and $227 million for the full year, representing a gross margin of 90%. This is consistent with prior quarters because supply chain efficiencies are offset by growth from VYVGART Hytrulo, which has a higher cost due to Halozyme royalties. The combined R&D and SG&A expenses totaled $2 billion for the full year. This is in line with our financial guidance for 2024.

下一張投影片。第四季銷售成本為 7,300 萬美元，全年銷售成本為 2.27 億美元，毛利率為 90%。這與前幾季一致，因為供應鏈效率被 VYVGART Hytrulo 的成長所抵消，而後者由於 Halozyme 的特許權使用費而成本更高。全年研發銷售、一般及行政開支總計 20 億美元。這符合我們 2024 年的財務指導。

Total operating expenses in Q4 were $658 million, an increase of $83 million compared to Q3 2024, the increase is primarily due to a $61 million increase in R&D, reflecting our capital allocation strategy of investing in innovation. This results in an operating profit for Q4 of $103 million and an operating loss for the full year of $22 million.

第四季的總營運費用為 6.58 億美元，較 2024 年第三季增加 8,300 萬美元，成長主要由於研發費用增加 6,100 萬美元，反映了我們投資於創新的資本配置策略。這導致第四季的營業利潤為 1.03 億美元，全年營業虧損為 2,200 萬美元。

The quarterly financial income is $39 million. And in the quarter, we incurred exchange losses of $55 million, mainly related to unrealized FX on our non-US denominated cash balances.

本季財務收入為3900萬美元。本季度，我們遭受了 5,500 萬美元的匯兌損失，主要與我們非美元計價現金餘額的未實現外匯有關。

Income tax is a benefit of $688 million in Q4 and a benefit of $748 million for the full year. This is due to the recognition of a deferred tax benefit of $802 million for the full year ended December 31, 2024, of which $725 million relates to a onetime nonrecurring recognition of previously unrecognized deferred tax assets existing as of December 31, 2023.

第四季所得稅收益為 6.88 億美元，全年所得稅收益為 7.48 億美元。這是因為確認了截至 2024 年 12 月 31 日的全年 8.02 億美元的遞延所得稅收益，其中 7.25 億美元與截至 2023 年 12 月 31 日存在的以前未確認的遞延所得稅資產的一次性非經常性確認有關。

We made the decision to recognize the deferred tax asset because of our assessment that it's probable that future taxable profits will be available. This results in profit in Q4 of $774 million and profit for the full year of $833 million.

我們決定確認遞延所得稅資產，因為我們評估未來很可能會獲得應稅利潤。這使得第四季利潤達到 7.74 億美元，全年利潤達到 8.33 億美元。

Our cash balance represented by cash, cash equivalents and current financial assets is $3.4 billion at year-end. The balance increased by $200 million in 2024. Our OpEx guidance for 2025 is approximately $2.5 billion of combined R&D and SG&A expenses.

我們的現金餘額（包括現金、現金等價物和流動金融資產）截至年底為 34 億美元。2024年餘額增加了2億美元。我們對 2025 年的營運支出預期為，研發和銷售、一般及行政開支總計約為 25 億美元。

This is a 25% increase year-over-year and reflects our commitment to invest in our R&D engine and pipeline growth. Zooming out, we are well positioned to prioritize innovation that will support our sustainable future with our strong balance sheet and transition to profitability in 2025.

這比去年同期成長了 25%，反映了我們對投資研發引擎和產品線成長的承諾。從長遠來看，我們已做好準備，優先考慮創新，以強勁的資產負債表和在 2025 年實現盈利，從而支持我們可持續的未來。

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

現在我將把電話轉給凱倫，她將提供有關商業方面的詳細資訊。

Thank you, Karl. Slide 9. I want to start by building on what both Tim and Karl have shared, which is the importance of innovation to Argenx. It's the driving force behind everything we do, and we all agree that innovation only matters if it reaches patients and provides meaningful benefit. In looking back at the fourth quarter and of all of last year, I'm pleased with the continued momentum we saw from the team in bringing meaningful innovation to patients.

謝謝你，卡爾。幻燈片 9。首先，我想基於 Tim 和 Karl 分享的內容，也就是創新對 Argenx 的重要性。這是我們所做的一切背後的驅動力，我們都同意，創新只有惠及患者並提供有意義的利益才有意義。回顧去年第四季及全年，我很高興看到團隊繼續保持強勁勢頭，為患者帶來有意義的創新。

Our growth was fueled by both MG and CIDP, and it's encouraging to see how VYVGART continues to transform outcomes as we broaden and deepen our reach in the MG community. And at just two quarters into the CIDP launch, we're already raising the bar on what patients can demand from their treatment. On today's call, I'll take a deeper dive into our performance, highlighting the drivers that will support our continued growth and patient impact.

我們的成長受到 MG 和 CIDP 的推動，隨著我們在 MG 社區的影響力不斷擴大和加深，VYVGART 不斷改變成果，這令人欣慰。CIDP 推出僅兩個季度，我們就已經提高了患者對治療的要求標準。在今天的電話會議上，我將更深入地探討我們的業績，重點介紹支持我們持續成長和對患者產生影響的驅動因素。

Next slide. We continue to deliver growth in the MG in the fourth quarter driven by consistent patient adds and new prescribers. VYVGART Hytrulo has played a key role in reaching patients earlier in the treatment paradigm and predominantly in patients brand new to VYVGART rather than switches from IV.

下一張投影片。在患者人數持續增加和新處方醫生不斷增加的推動下，我們第四季度的 MG 業務繼續實現成長。VYVGART Hytrulo 在早期接觸治療模式的患者方面發揮了關鍵作用，並且主要針對的是首次接受 VYVGART 治療的患者，而非從 IV 轉換過來的患者。

Some additional growth was supported by a halo effect from the CIDP launch, where we saw neurologists who first prescripts in CIDP now also prescribing in MG. To maintain our leadership as the number 1 prescribed branded biologic in MG, we are prioritizing the anticipated launch of the prefilled syringe and continuing to invest in generating new evidence, including through label extension studies.

CIDP 推出帶來的光環效應支持了一些額外的增長，我們看到最初在 CIDP 中開處方的神經科醫生現在也在 MG 中開處方。為了保持我們作為 MG 領域第一大處方品牌生物製劑的領先地位，我們優先考慮推出預充式註射器，並繼續投資於產生新證據，包括透過標籤擴展研究。

We have a long-term strategy to enable continued adoption of VYVGART in earlier treatment lines, and this remains our key focus. VYVGART's consistently strong safety and efficacy profile support this goal, particularly as physicians gain more experience with the treatment.

我們有一個長期策略，以使 VYVGART 能夠繼續在早期治療方法中得到應用，這仍然是我們的重點。VYVGART 一貫強大的安全性和有效性支持這一目標，特別是當醫生在治療方面獲得更多經驗時。

And we'll further build this confidence by investing in evidence generation. Real-world data efforts are underway to evaluate reduction in steroid use, safety in new patient populations and dosing through our ADAPT NEXT study, which we plan to share at upcoming medical conferences.

我們將透過投資證據生成來進一步增強這種信心。我們正在進行真實世界數據工作，以透過 ADAPT NEXT 研究評估類固醇使用的減少、新患者群體的安全性和劑量，我們計劃在即將召開的醫學會議上分享這項研究。

Innovation on route administration will also support our shift earlier in the treatment paradigm, and the prefilled syringe is a key step towards expanding our patient reach. The opportunity to self-inject at home is a significant innovation for patients and provides an added level of freedom in their treatment regime.

給藥途徑的創新也將支持我們更早轉變治療模式，而預充式註射器是我們擴大患者覆蓋範圍的關鍵一步。在家中自行注射的機會對於患者來說是一項重大創新，並為他們的治療方案提供了額外的自由。

We were thrilled to receive our first global approval of VYVGART prefilled syringe this month in the EU for gMG patients, and we're now looking ahead to the FDA PDUFA date in April for both MG and CIDP.

本月，我們很高興在歐盟獲得了 VYVGART 預充式註射器的全球首個批准，用於治療 gMG 患者，目前我們正期待 4 月份 FDA 對 MG 和 CIDP 的 PDUFA 批准日期。

Lastly, we see additional opportunity to address the unmet need in the MG community by expanding our label into CR-negative and ocular MG. ADAPT SERON is the first of our Phase III studies to read out this year, and we have ample real world and clinical data sets supporting our ability to drive responses in this population.

最後，透過將我們的標籤擴展到 CR 陰性和眼部 MG，我們看到了解決 MG 社區未滿足需求的更多機會。ADAPT SERON 是我們今年第一個完成的 III 期研究，我們擁有充足的真實世界和臨床數據集支持我們推動該族群回應的能力。

Ocular MG presents another exciting opportunity. And with 80% of patients progressing to generalized MG, we have the potential to introduce innovation earlier in the treatment paradigm.

Ocular MG 提供了另一個令人興奮的機會。由於 80% 的患者會發展為全身性 MG，我們有可能在治療模式中更早引入創新。

Next slide. We're very encouraged by the continued momentum of the VYVGART Hytrulo launch in CIDP, It's abundantly clear that our data are resonating across patients and physicians, with the initial demand highlighting the unmet need for safe and effective treatment alternatives.

下一張投影片。VYVGART Hytrulo 在 CIDP 中的持續推出讓我們深受鼓舞，很明顯，我們的數據在患者和醫生中引起了共鳴，最初的需求凸顯了對安全有效的治療替代方案的未滿足需求。

One patient shared that she have tried nearly all available CIDP treatments and they all failed. So she was left with a heavy treatment burden of plasmapheresis every 7 to 14 days. The physician was dedicated to improving this burden and suggested a switch to VYVGART Hytrulo.

一位患者說，她嘗試了幾乎所有可用的CIDP治療方法，但都失敗了。因此她每7至14天要承擔一次血漿置換的沉重治療負擔。醫生致力於改善這種負擔，並建議改用VYVGART Hytrulo。

She was initially very hesitant. But after seeing the improvements, she realized she had massively underestimated the disease burden she had still been facing. Even as functional as she was, it wasn't until VYVGART Hytrulo that she felt the enormous boulder lifted off her back.

她最初非常猶豫。但在看到改善後，她意識到自己嚴重低估了仍面臨的疾病負擔。儘管她已經恢復了功能，但直到 VYVGART Hytrulo 時她才感覺到巨大的巨石從她的背上移開了。

While this patient's improvement was dramatic, this is just one of the many inspiring stories from patients and their caregivers who are empowered to demand more from their treatment. I'm incredibly proud of the team for building a solid foundation to get us up to this strong start to ultimately reach our target addressable population of 12,000 patients. Our market access team has done a phenomenal job securing broad access to support patients getting on treatment quickly.

雖然這名患者的病情有了顯著改善，但這只是患者及其照護者眾多鼓舞人心的故事之一，他們有能力要求從治療中獲得更多。我為團隊感到無比自豪，他們奠定了堅實的基礎，使我們有了一個良好的開端，並最終達到了 12,000 名患者的目標人群。我們的市場准入團隊做出了卓越的工作，確保了廣泛的准入，支持患者快速接受治療。

The sales force expansion further enables us to successfully reach new prescribers deeper in the community setting and it paid off. 25% of prescribers over the last quarter were first time VYVGART users. As of year-end, we had approximately 1,000 patients on treatment with the majority of these falling within our initial addressable population, those who are not sufficiently controlled but all who experience side effects on IVIg or steroids. Most neurologists will treat patients for 12 weeks to assess this response to VYVGART, so we look forward to gaining more insight on response rates and utilization in the coming quarters.

銷售團隊的擴大使我們能夠在社區環境中更深入地成功接觸新的處方者，並且獲得了回報。上個季度有 25% 的處方者是首次使用 VYVGART。截至年底，我們約有 1,000 名患者接受治療，其中大多數屬於我們最初的目標族群，即那些沒有得到充分控制但在使用 IVIg 或類固醇時都會出現副作用的患者。大多數神經科醫生會對患者進行 12 週的治療，以評估對 VYVGART 的反應，因此我們期待在未來幾個季度獲得有關反應率和利用率的更多見解。

We're just at the beginning. Our priority this year will be to reach more patients and more prescribers and repeat the MG playbook to consistently generate data to build physician support of Hytrulo while also empowering patients to ask for more from the CIDP treatment.

我們才剛開始。我們今年的首要任務是接觸更多的患者和更多的處方者，並重複 MG 劇本，持續產生數據來建立醫生對 Hytrulo 的支持，同時也讓患者能夠從 CIDP 治療中獲得更多。

Next slide. We also look forward to additional expansion opportunities outside the US this year, particularly as we plan to launch the prefilled syringe and CIDP in multiple regions. In MG, we have launched in most of the major markets ex-US and expect consistent, steady growth over time as market access dynamics fall into place.

下一張投影片。我們也期待今年在美國以外有更多的擴張機會，特別是我們計劃在多個地區推出預充式註射器和 CIDP。在 MG，我們已在美國以外的大多數主要市場推出了產品，隨著市場准入動態的到位，我們預計會隨著時間的推移實現持續、穩定的成長。

We're now reimbursed in 13 countries in Europe, including four out of the five major markets. And we're pleased with the recent MG approval in South Korea and, one that is personally very close to my heart, Australia.

我們目前已在歐洲 13 個國家獲得報銷，其中包括五大主要市場中的四個。我們很高興看到 MG 最近在韓國和澳洲獲得批准，我個人也非常關心這個國家。

Earlier, I highlighted the positive CHMP opinion on PFS, enabling sales in the EU. And this is just the first of four regulatory decisions on approval this year. We are just at the beginning of our global CIDP launch with additional decisions on approval expected in China, Europe and Canada. Feedback from Japan, the first approval following the US, has been positive across patients and physicians in the CIDP community.

之前，我強調了 CHMP 對 PFS 的正面評價，認為其有利於在歐盟銷售。這只是今年批准的四個監管決定中的第一個。我們全球 CIDP 的啟動才剛開始，預計中國、歐洲和加拿大將做出更多批准決定。作為繼美國之後首個獲得批准的藥物，日本的回饋得到了 CIDP 社區患者和醫生的積極評價。

Next slide. Our expansive pipeline supports our next wave of growth, and we're committed to addressing the unique challenges and gaps in treatment for autoimmune patients across multiple inpatients, where there is often a lack of innovation and high barriers to access.

下一張投影片。我們廣泛的產品線支持著我們下一波的成長，我們致力於解決多名住院自體免疫患者在治療方面面臨的獨特挑戰和差距，這些患者往往缺乏創新且獲得治療的門檻較高。

We've built a robust network of relationships in the neurology community with over 3,500 VYVGART prescribers, which we'll leverage as we advance into our next launch wave with seronegative and ocular MG. The impressive data from the adult study in MMN for empasiprubart have already drawn attention from our prescriber base. where there is a lot of overlap with CIDP and MG.

我們在神經病學界已建立了強大的關係網絡，擁有超過 3,500 名 VYVGART 處方者，我們將利用這一網絡，推進下一波血清陰性和眼部 MG 的上市進程。在 MMN 成人研究中，empasirubart 的令人印象深刻的數據已經引起了我們處方人員的注意。其中與CIDP和MG有許多重疊。

I share Tim's excitement about empasiprubart and about MMN and our opportunity to transform the treatment paradigm. We see MMN as a nascent market ripe for innovation, similar to the initial dynamics we saw with MG.

我和Tim一樣對 empasiprubart 和 MMN 以及我們改變治療模式的機會感到興奮。我們認為 MMN 是一個適合創新的新興市場，與我們在 MG 中看到的初始動態相似。

Patients continue to be discouraged with the symptom burden and the lack of treatment options. So the Phase III data in will be a big moment for us to potentially introduce the first precision treatment to this community.

患者因症狀負擔和缺乏治療選擇而繼續感到沮喪。因此，第三階段的數據將成為我們向該族群推出首個精準治療方案的重要時刻。

Looking ahead, we see an opportunity with VYVGART in myositis to serve a bridge from neurology into rheumatology with subtypes across both therapeutic areas. We aim to leverage our neurology playbook as we expand into rheumatology, building on the traction we've already gained in the community, following our decision to advance Sjogren’ into a registrational study. All in all, this is an exciting time for Argenx across our entire business as we progress towards our Vision 2030. Tim?

展望未來，我們看到 VYVGART 在肌炎領域有機會充當從神經病學到風濕病學的橋樑，並在兩個治療領域提供亞型。在我們決定將乾燥綜合症推進到註冊研究之後，我們計劃利用我們的神經病學策略，在擴展到風濕病學的過程中，並利用我們在社區中已經獲得的支持。總而言之，對於 Argenx 的整個業務來說，這是一個令人興奮的時刻，因為我們正朝著 2030 願景邁進。提姆？

Thank you, Karen. Slide 14. I want to extend my sincere appreciation to the Argenx team, including our Board, for their outstanding work last year and the unwavering dedication to improving the lives of patients. We've deliberately set a very high bar, and as we move forward, it's critical that our continued innovation in the autoimmune space maintains its best-in-class standards.

謝謝你，凱倫。幻燈片 14。我要向 Argenx 團隊（包括我們的董事會）表示最誠摯的感謝，感謝他們去年的出色工作以及為改善患者生活所做的不懈奉獻。我們特意設定了一個非常高的標準，隨著我們不斷前進，我們在自體免疫領域的持續創新必須保持一流的標準。

2025 will be a year of significant growth for us, expanding our commercial reach, introducing new products, entering new markets and advancing our late-stage pipeline. Importantly, we never lose sight of our mission: harnessing innovation to develop transformative medicines for the patients we serve.

2025 年將是我們實現重大成長的一年，我們將擴大商業範圍、推出新產品、進入新市場並推動後期產品線。重要的是，我們從未忘記我們的使命：利用創新為我們服務的患者開發變革性藥物。

Thank you for your time today. I would now like to open the call to your questions.

感謝您今天抽出時間。現在我想開始回答你們的問題。

(Operator Instructions) Tazeen Ahmad, Bank of America.

(操作員指示) Tazeen Ahmad，美國銀行。

Mine is going to be on the PFS upcoming PDUFA. So maybe for Karen, can you talk about how you're seeing what pent-up demand there could be for PFS? Are there patients that have not gone on to therapy knowing that there potentially could be this more convenient option?

我即將參加 PFS 的 PDUFA。那麼對於 Karen，您能否談談您如何看待 PFS 可能存在的被壓抑的需求？有沒有患者知道可能有這種更方便的選擇而沒有接受治療？

And if you're looking for switches to recur, would that be gradual or would that be a bolus effect that we should see upon approval? And then I have a follow-up.

如果您希望轉換再次發生，那麼這將是漸進的還是我們在獲得批准後就應該看到的快速效應？然後我有一個後續問題。

Yeah. Thanks for the question, Tazeen, and for the interest. We're really excited for the upcoming PFS PDUFA date in April and potential approval. So here's what I would say, I don't think we're seeing pent-up demand. In fact, I'm quite pleased with the momentum we've seen on MG and CIDP in terms of new patient starts.

是的。謝謝 Tazeen 的提問與關注。我們對即將到來的 4 月 PFS PDUFA 日期和可能的批准感到非常興奮。所以我想說的是，我認為我們沒有看到被壓抑的需求。事實上，我對 ​​MG 和 CIDP 在新患者開始方面所看到的勢頭感到非常滿意。

It's pretty consistent. And -- but what we do expect is that prefilled syringe for self-injection will open up both the prescriber base and the patients that will consider they've got as an option for either MG or CIDP.

它相當一致。而且 — — 但我們確實期望用於自我注射的預充式註射器將擴大處方者基礎，也讓患者認為他們已經擁有了治療 MG 或 CIDP 的一種選擇。

So we think what it will enable us to do is maintain that consistent momentum and that consistent growth, which is pretty incredible as we get 13 quarters out from launch. And I think that's what the innovation allows us to do. It's not specifically a switch strategy that we're pursuing, to the second part of the question that you asked. Rather, we want to focus on expanding our reach to prescribers and to patients. Thanks.

因此，我們認為它將使我們能夠保持持續的勢頭和持續的成長，這非常不可思議，因為我們從發布之日起已經過去了 13 個季度。我認為這就是創新使我們能夠做到的。對於您提出的問題的第二部分，我們追求的並不是特定的轉換策略。相反，我們希望集中精力擴大我們對處方人員和患者的覆蓋範圍。謝謝。

I think you had a follow-up.

我認為你已經跟進了。

Yeah. Either for you or Tim, how should we be thinking about just generally pricing for PFS? Would it be similar to the current options available? Or should we expect any kind of difference?

是的。無論對於您還是 Tim，我們應該如何考慮 PFS 的一般定價？它會與目前可用的選項類似嗎？或者我們應該期待任何差異嗎？

Tazeen, it's Karl here. Thank you for your questions. Consistent with prior launches, we will provide more color on the price at the time of a launch. But let's provide some framework on how we think about it. we'll be bringing important innovation to patients, and our primary goal is to provide broad access and optionality to patients and physicians to choose on what's best for them.

塔澤恩，我是卡爾。感謝您的提問。與先前的發布一致，我們將在發佈時提供更多有關價格的資訊。但讓我們提供一些關於我們如何思考這個問題的框架。我們將為患者帶來重要的創新，我們的主要目標是為患者和醫生提供廣泛的獲取途徑和選擇權，以選擇最適合他們的治療方法。

And of course, we will aim to price in a responsible and sustainable way for Argenx. Thank you for your question.

當然，我們將致力於以負責任和永續的方式為 Argenx 定價。感謝您的提問。

Alexander Thompson, Stifel.

亞歷山大湯普森（Alexander Thompson），Stifel。

I guess as a follow-up to that question for Karl. You've talked about sort of net price impacts with the PFS. I guess, can you talk a little bit more about potential magnitude there and also the rate of some kind of -- of an impact like that would you expect to see upon the launch this year?

我想這是對卡爾這個問題的後續回答。您曾談到 PFS 對淨價格的影響。我想，您能否再多談談那裡的潛在規模以及您預計今年發射時會看到的某種影響的速度？

Thank you for the question, Alex. Yeah, the dynamics for self-administration, and this is based on FDA approval -- subject to FDA approval of self-administration, of course, will be different. And gross to net will increase. Currently, it's around 12% with a majority of patients currently in a medical benefit Part B.With growth -- with self-administration, the patients will transition to pharmacy benefit or Medicare Part D. You will then get the typical rebates for a pharmacy benefit, which will be higher than what we see today.

謝謝你的提問，亞歷克斯。是的，自我管理的動態，這是基於 FDA 的批准——當然，經 FDA 批准的自我管理會有所不同。毛利率與淨利率之比將會增加。目前，這一比例約為 12%，其中大多數患者目前享有醫療福利 B 部分。隨著自我管理的發展，患者將過渡到藥房福利或醫療保險 D 部分。然後您將獲得藥房福利的典型回扣，這將高於我們今天看到的水平。

And of course, we will be subject to IRA redesign, where the manufacturer, us, get 20% of cost post-catastrophic of Medicare Part D patients. All of that will result in a higher gross to net. We, of course, expect that increased patient volumes will offset the increase in gross to net. And over time, you will also see likely that the VBAs will be phasing out. Thank you for your question.

當然，我們將接受 IRA 重新設計，其中製造商（我們）將獲得 Medicare Part D 患者災難後成本的 20%。所有這些都將導致毛利潤與淨利潤的比率更高。當然，我們預計患者數量的增加將抵消總患者數量的增長。隨著時間的推移，您也可能會看到 VBA 逐漸被淘汰。感謝您的提問。

Derek Archila, Wells Fargo.

富國銀行的德里克‧阿奇拉 (Derek Archila)。

Just one from us. I guess how should we think about the quarter-over-quarter growth cadence in MG in 2025 relative to what we saw in '24? And then ultimately, in 1Q, should we expect some seasonal impacts?

我們只有一個。我想，我們該如何看待 2025 年 MG 相對於 24 年的季度環比成長節奏？那麼最終，在第一季度，我們是否應該預期會出現一些季節性影響？

Yeah. Thanks for the question, Derek. This is Karen. I'm happy to take it. So what -- how I think about it is what I was mentioning before in MG, continued momentum and continued strong uptake in line with the strategy that we have of moving earlier lines in the treatment paradigm and broadening the prescriber base.

是的。謝謝你的提問，德里克。這是凱倫。我很高興接受它。那麼 — — 我的想法就是我之前在 MG 中提到的，持續的勢頭和持續的強勁增長符合我們在治療模式中推進早期發展和擴大處方者基礎的戰略。

What I would expect that those underlying dynamics will continue. Specific to your question on Q1, look, Q1 seasonality is an effect that is seen across the industry. And don't forget, last year, we had our Q1 growth rate of 6%. So we are seeing the benefit reverification. That is an industry-wide phenomenon. So take that into consideration perhaps for Q1.

我預計這些潛在的動力將會持續下去。具體到您關於第一季的問題，第一季的季節性是整個產業都會看到的影響。別忘了，去年我們第一季的成長率是 6%。因此，我們看到了效益的重新驗證。這是一個全行業的現象。因此也許應該在第一季考慮到這一點。

But overall, what we're seeing is continued momentum and our underlying dynamics are good.

但總體而言，我們看到的是持續的勢頭，而且我們的潛在動力是良好的。

Yaron Werber, TD Cowen.

亞倫·韋伯（Yaron Werber），TD Cowen。

Great. Maybe a couple of questions. One for Karl. The -- when the VBAs expire because of Part D, how does that work? I assume access stays the same? Or do you need to renegotiate?

偉大的。也許有幾個問題。一份給卡爾。當 VBA 因 D 部分而到期時，該怎麼辦？我認為存取權限保持不變？還是你需要重新協商？

And then for Karen, for the first 12 weeks on CIDP, are physicians actually doing [in cat] and actually looking at -- and actually assessing formally whether patients are improving? Or I imagine they're just keeping them on therapy. And if they'll really not benefit, they'll take them off.

然後對於 Karen 來說，在 CIDP 的前 12 週，醫生是否真的在做 [in cat] 並且真正觀察 - 並且真正正式評估患者是否有所好轉？或者我想他們只是讓他們繼續接受治療。如果他們確實沒有受益，他們就會將其取消。

Yaron, thank you for your question. On VBAs, with PFS, of course, we will have to go back and the payer agreements will have to be established. We've done that now a couple of times, and we need to do it again. And as it is a pharmacy benefit, our expectation is that the VBAs will not be part of those contracts. And so therefore, it will phase out over time. Thank you for your question.

Yaron，謝謝你的提問。對於 VBA，當然，有了 PFS，我們必須回去，並且必須建立付款人協議。我們已經這樣做過幾次了，我們需要再做一次。由於這是一項藥房福利，我們預計 VBA 不會成為這些合約的一部分。因此，隨著時間的推移，它將逐漸被淘汰。感謝您的提問。

Thanks, Karl. And yeah, on the question of -- so we are seeing with CIDP that is generally doctors are giving more like a 12-week trial, as you mentioned. We're not hearing about a lot of use of in cat and those types of scales. They're more used in clinical trials that in clinical practice. So more generally, the doctors will have a conversation with their patient.

謝謝，卡爾。是的，關於這個問題——我們看到，正如您所說，CIDP 通常情況下，醫生會提供 12 週的試驗期。我們並沒有聽說貓和這類秤有很多用途。它們在臨床試驗中的應用比在臨床上有更多的應用。因此更普遍的情況是，醫生會與病人交談。

They're using more simple assessments to determine how their disease is being controlled by VYVGART and then making an assessment on response.

他們正在使用更簡單的評估來確定他們的疾病如何透過 VYVGART 控制，然後對反應進行評估。

Victor Floch, BNP Paribas.

法國巴黎銀行的 Victor Floch。

Victor Floch from BNP Paribas Exane. So my first question relates to your 2025 news flow, which is broadly seen, I guess, as lighter than what you are expecting for 2026. And I mean, it's fair to say that 2026 is going to be quite strong.

法國巴黎銀行的維克多‧弗洛赫 (Victor Floch)。所以我的第一個問題與您的 2025 年新聞流有關，我猜普遍認為它比您對 2026 年的預期要少。我的意思是，可以公平地說，2026 年將會相當強勁。

But in the meantime, would you say that investors are overlooking the potential for the seronegative Phase II trial to significantly expand MG opportunity as well as PFS ability to further drive earlier line penetration in both MG and CIDP? And my follow-up on the PFS is, I'm just wondering if you could help us understand how important self-administration has been for Hytrulo penetration ex-US.

但同時，您是否認為投資者忽略了血清陰性 II 期試驗的潛力，該試驗可以顯著擴大 MG 機會，以及 PFS 進一步推動 MG 和 CIDP 早期線路滲透的能力？我對 PFS 的後續問題是，我只是想知道您是否可以幫助我們了解自我管理對於 Hytrulo 在美國以外的滲透有多重要。

I know you don't report any sales breakdown and that there are structural differences between US markets and ex-US. But just trying to understand how self-administration on label in US could meaningfully drive sales.

我知道您沒有報告任何銷售細目，並且美國市場和美國以外市場之間存在結構性差異。但只是想了解美國標籤上的自我管理如何有效地推動銷售。

Thanks, and great to hear from you in the call. You're right to call out that we do have self-administration already for VYVGART Hytrulo outside of the United States. And I will let Karl and Karen comment on why that is important for physicians and patients.

謝謝，很高興在電話中接到您的來電。您說得對，我們在美國以外地區已經對 VYVGART Hytrulo 進行了自我管理。我會讓卡爾和卡倫評論為什麼這對醫生和病人很重要。

On new flow, this is an incredibly busy year from an execution point of view. I mean, pushing 10 Phase III clinical trials and 10 Phase II clinical trials forward at this speed, I think, is a very serious path. But in the first half of the year, I think PFS is important with hopefully self-administration, as Karen explained in a minute.

就新流程而言，從執行的角度來看，這是極其繁忙的一年。我的意思是，以這種速度推進 10 個 III 期臨床試驗和 10 個 II 期臨床試驗，我認為是一條非常嚴肅的道路。但在上半年，我認為 PFS 很重要，希望能夠自我管理，正如 Karen 稍後解釋的那樣。

And then you're correct to call out the significance of, hopefully, a positive readout in seronegative MG patients. There's a significant volume of patients either in high unmet medical needs. We have seen in Japan how important that patient population actually is and how successful VYVGART actually is in treating these patients.

那麼您正確地指出了血清陰性 MG 患者陽性讀數的重要性。有大量患者的醫療需求尚未被滿足。我們在日本看到了患者群體實際上有多重要，以及 VYVGART 在治療這些患者方面取得了多大的成功。

So that could be a very important force of driver in maintaining the momentum we were alluding to. And then I would also like to call out an important proof of concept study in lupus nephritis second half of this year.

因此，這可能是維持我們所提到的勢頭的一個非常重要的驅動力量。然後，我還想提一下今年下半年關於狼瘡性腎炎的一項重要概念驗證研究。

So Karen, you want to continue with the importance of self-administration?

那麼 Karen，你想繼續強調自我管理的重要性嗎？

Yeah, absolutely. And just to reinforce, we do have self-administration on label in both the EU as well as in Japan. And what we see in those markets is that when patients and physicians have a choice between the IV option and the self-administration subcutaneous option, not all patients move to self-administration. There are some patients that choose to stay with IV. That's what fits with their lifestyle. Perhaps they prefer not to do the self-administration because of needle phobia or something like that.

是的，絕對是如此。需要強調的是，我們在歐盟和日本都對標籤進行自我管理。我們在這些市場中看到的是，當患者和醫生可以在靜脈注射和自我給藥皮下注射之間做出選擇時，並不是所有患者都會選擇自我給藥。有些病人選擇繼續接受靜脈注射。這很符合他們的生活方式。或許他們因為針頭恐懼症或類似原因而不願意自行注射。

But a good percentage of patients do have the preference and it fits into their lifestyle to take the self-administration option. So what we see is that there's real benefit to both patients and prescribers to having multiple routes of administration so that the patient and the neurologist can really make a decision together about what makes most sense for that patient based on their disease and their lifestyle.

但相當一部分患者確實有這種偏好，選擇自我給藥也符合他們的生活方式。因此，我們看到，多種給藥途徑對於患者和處方者都有真正的好處，以便患者和神經科醫生可以根據患者的疾病和生活方式共同做出對患者最有意義的決定。

And so we think that, that same experience will come through in the US if we get approval for self-injection on April 10. Thanks for the question.

因此我們認為，如果我們在 4 月 10 日獲得自我注射的批准，同樣的經驗也會在美國實現。謝謝你的提問。

Akash Tewari, Jefferies.

Akash Tewari，傑富瑞（Jefferies）。

This is Amy on for Akash. Starting with the myositis trial, are you planning on making any changes to the trial design after your go-or-no-go decision? And how are you thinking about the probability of success across each of the subsets now? And then finally, wanted to get your thoughts on the (inaudible) data and how that reads across to your lupus nephritis trial.

我是 Amy，為 Akash 節目主持。從肌炎試驗開始，在做出進行或不進行的決定後，您是否計劃對試驗設計做出任何更改？您現在如何看待每個子集的成功機率？最後，想聽聽您對（聽不清楚）數據的看法，以及這些數據對您的狼瘡性腎炎試驗有何影響。

Just in myositis, we announced and we made the go-or-no-go decision to go forward in all three of the subtypes with no changes to the protocol. You are correct to call out that we have some degrees of flexibility of freedom to make these changes. But based on the Phase II data, we did not see any need to make changes. And you can assume that because we gave the go-ahead for all these subsets that people believe in all the subsets for success in Phase III.

僅在肌炎方面，我們就宣布了繼續進行這三種亞型的治療的決定，並且不改變治療方案。您說得對，我們在做出這些改變時具有一定的彈性。但根據第二階段的數據，我們認為沒有必要做出任何改變。並且你可以假設，因為我們對所有這些子集都給予了批准，所以人們相信所有子集都會在第三階段取得成功。

Look, in lupus nephritis, we see a number of metrics being tested. Whatever the data, which we see, I think there's ample of room for improvement. There is a significant unmet medical need. There's a need for a toolbox to treat these patients. And I don't think there's going to be one size fits all.

你看，在狼瘡性腎炎中，我們看到許多指標正在被測試。無論我們看到什麼數據，我認為都有足夠的改善空間。存在大量未滿足的醫療需求。需要一個工具箱來治療這些病人。我不認為會存在一種適合所有情況的統一方案。

So let's focus on our data. We have strong conviction in the biology, and we're testing that mechanism of action now in Phase II. So that's the data speed. But Amy, thank you for the questions.

因此讓我們集中關注我們的數據。我們對其生物學原理深信不疑，目前我們正在第二階段測試其作用機制。這就是數據速度。但是艾米，謝謝你的提問。

(Operator Instructions) Rajan Sharma, Goldman Sachs.

（操作員指示）高盛的拉詹夏爾馬 (Rajan Sharma)。

I'll keep it to just one. So just to understand some of the underlying dynamics in myasthenia gravis. So we know that back at launch of VYVGART, you said that the addressable market was about 17,000 patients and you think there's an additional 25,000 that will come from growth in the biologics market by 2030.

我將只保留一個。只是為了了解重症肌無力的一些潛在動態。所以我們知道，在 VYVGART 推出時，您說潛在市場約為 17,000 名患者，而您認為到 2030 年，生物製劑市場的成長將帶來另外 25,000 名患者。

So I'd just be interested, back in 2021, when you initially saw that 17,000 market, how do you think that biologics share has progressed now, i.e., how much through that additional 25,000 are we already?

所以我很感興趣，回到 2021 年，當您最初看到那個 17,000 個市場時，您認為現在生物製劑的份額如何發展，也就是說，我們已經通過了額外的 25,000 個市場多少？

Yeah. Thanks for the question around MG. And certainly, as we lay out our strategy for MG, we think that we are very much on the beginning still of the growth curve. And you talked about the market expansion that we're already seeing from the 17,000 since the launch of VYVGART. So we do think there has been market expansion.

是的。感謝您提出有關 MG 的問題。當然，當我們制定 MG 策略時，我們認為我們仍處於成長曲線的起步階段。您談到了市場擴張，自 VYVGART 推出以來，我們的市場規模已達到 17,000 家。因此我們確實認為市場已經擴張。

And the way that we see that is that when -- since launch, we're no longer just being used on advanced biologics are no longer just being used in the most refractory patients. Rather, the advanced biologics are being used earlier line. And VYVGART, let's say, leading the charge on that as the number 1 prescribed biologic.

我們看到的是，自推出以來，我們不再只是用於先進的生物製劑，也不再只是用於最難治的患者。相反，先進的生物製劑正在被更早使用。可以說，VYVGART 在該領域處於領先地位，成為排名第一的處方生物製劑。

We shared the statistic that 60% of patients are actually coming directly to VYVGART from orals. So that demonstrates that we're starting to penetrate that 25,000. But we believe that there's quite a long way to go and that we can maintain that consistent and steady growth.

我們分享了一項統計數據：60% 的患者實際上是透過口服藥物直接來到 VYVGART 的。這顯示我們開始突破那 25,000 個。但我們相信，還有很長的路要走，我們可以維持持續穩定的成長。

And certainly, prefilled syringe with self-injection will help us with that. Thanks for the question.

當然，具有自我注射功能的預充式註射器可以幫助我們實現這一點。謝謝你的提問。

Suzanne van Voorthuizen, Kempen.

蘇珊娜·範·沃特赫伊森，肯彭。

This is Suzanne from Kempen. I was wondering if you can elaborate a bit on your longer-term thinking of FcRn franchise. I guess you're getting to the point where investing in more Phase III trials for VYVGART may not always make sense, and you perhaps look towards the next generation FcRn, 213.

這是來自肯彭的蘇珊娜。我想知道您是否可以詳細說明您對 FcRn 特許經營的長期想法。我想你已經意識到，對 VYVGART 進行更多的 III 期試驗投資可能並不總是有意義的，你或許會著眼於下一代 FcRn，213。

So on 213's clinical development, do you see possibilities for shorter development time lines? What are elements that you can leverage from having developed VYVGART?

那麼，對於 213 的臨床開發，您是否認為有可能縮短開發時間？您可以利用開發 VYVGART 的哪些元素？

Yeah, Suzanne, I think you're right in calling out that [X7] is going to be a franchise. It's an incredible opportunity. I think the size of the opportunity exceeds the ability to serve that with one molecule given the patent life and the life of a molecule like VYVGART. So I think developing a molecule like ARGX-213 gives us optionality. And we said this is the first successful molecules.

是的，蘇珊娜，我認為你說[X7]將成為一個特許經營權是正確的。這是一個難得的機會。我認為，考慮到專利期限和 VYVGART 等分子的壽命，機會的規模超出了用一個分子提供服務的能力。因此我認為開發像 ARGX-213 這樣的分子為我們提供了選擇權。我們說這是第一個成功的分子。

It basically unfolds optionality. I mean, either you will go and try to roll up existing indications and then you're spot on. I mean, there's so much we know about MG and so much we know about CIDP, ITP and the other indications to come that actually you could leave us that know-how and leapfrog with molecules like 213, or you basically decide going to open up new opportunity where you have, for example, a whole new game plan from a positioning and pricing point of view.

它基本上展現了可選性。我的意思是，要嘛你去嘗試總結現有的跡象，然後你就成功了。我的意思是，我們對 MG、CIDP、ITP 以及未來其他跡象了解很多，實際上您可以將這些技術訣竅留給我們，然後利用 213 這樣的分子進行跨越式發展，或者基本上決定開闢新的機會，例如，從定位和定價的角度製定全新的遊戲計劃。

So that optionality is in front of us, and we will basically unpack it whilst we go based on data. We are really focused now on generating the Phase I data second half of this year, which will tell us a lot about the potential of this molecule going forward. Thanks for the question.

因此，這種可選性就在我們面前，我們將根據數據，在進行過程中逐步解開它。我們現在真正專注於在今年下半年產生第一階段的數據，這將告訴我們很多關於該分子未來潛力的資訊。謝謝你的提問。

Vikram Purohit, Morgan Stanley.

摩根士丹利的維克拉姆·普羅希特（Vikram Purohit）。

We'll keep our questions focused on the auto-injector. I believe your release mentioned that you'll be looking to move this forward in 2027. So we just wanted to see what the next update could be for the auto-injector and how you're thinking about how this form of VYVGART potentially could expand the opportunity beyond the IV and the subcu and the PFS?

我們的問題集中在自動注射器。我相信您的新聞稿中提到過，您將尋求在 2027 年推動這一目標的實現。所以我們只是想看看自動注射器的下一次更新是什麼，以及您如何看待這種形式的 VYVGART 如何潛在地將機會擴展到 IV、subcu 和 PFS 之外？

Yeah. Thank you, Vikram. The significance of the auto-injectors or the differentiation of the auto-injector from a prefilled syringe is actually having a device where the needle is invisible and does not need to be manipulated by the patient.

是的。謝謝你，維克拉姆。自動注射器的意義或自動注射器與預充式註射器的差別實際上在於它是一種針頭不可見且不需要病人操作的裝置。

So basically, now the only job left is to hold the device against your belly, you press the button, and then the device is manipulating the needle in a way that you don't see it. And that is significant for a subset of patients. So in an attempt to continue to innovate in our core markets, this is a logical next step building from the prefilled syringe.

所以基本上，現在唯一剩下的工作就是將設備靠在腹部，按下按鈕，然後設備以你看不見的方式操縱針頭。對於一部分患者來說，這具有重要意義。因此，為了繼續在我們的核心市場上進行創新，這是在預充式註射器的基礎上進行的合乎邏輯的下一步。

From the moment -- for this moment, actually, we've completely focused in our communication on the prefilled syringe. It is a very innovative product, thanks to the technologies we leverage in the formulation and the technologies we leverage for the containers. So a significant innovation and I think a significant driver for the business. Stay tuned on the auto-injector. We are working very hard on that in the background.

從此刻起——實際上，就此刻而言，我們的溝通重點已經完全集中在預充式註射器上。這是一個非常創新的產品，這要歸功於我們在配方中利用的技術和我們在容器中利用的技術。因此，這是一項重大的創新，我認為也是業務發展的重要推手。請繼續關注自動注射器。我們正在幕後努力實現這一目標。

But it's too early to commit to a next specific update, okay? So 2027 is the year we're all aiming for, and I will say stay-tuned. Thank you.

但現在承諾下一次具體更新還為時過早，好嗎？所以 2027 年是我們所有人都追求的一年，我想說的是，敬請期待。謝謝。

Andy Chen, Wolfe Research.

安迪陳（Andy Chen），沃爾夫研究公司。

This is Aman for Andy. A question on CIDP and maybe just some metrics you're seeing early on in its launch. It was mentioned majority of patients are IVIg-experienced. Is IVIg succeeded in early lines? Are there signs of early standard of care transformation, maybe docs preferring VYVGART over IViG? Just any insight would be great.

這是安迪的阿曼。關於 CIDP 的一個問題，也許只是您在其發布初期看到的一些指標。有人提到，大多數患者都有 IVIg 治療經驗。IVIg 在早期治療中成功了嗎？是否有早期護理標準轉變的跡象，也許醫生更喜歡 VYVGART 而不是 IViG？任何見解都很好。

Yeah. Thanks for the question about CIDP launch. As I shared, I'm really pleased with where we're at with the launch. We're clearly seeing early success. And there is a clear unmet need that you can see.

是的。感謝您提出有關 CIDP 啟動的問題。正如我所分享的，我對於我們發布的進展感到非常滿意。我們清楚地看到了早期的成功。並且您可以看到，存在明顯未滿足的需求。

And what we consistently hear, the feedback from the community, whether it be neurologists, patients, caregivers is that there is a higher treatment burden and a higher disease burden than they had realized. So bringing the first innovation in 30 years is making a big difference.

我們不斷聽到來自社區的回饋，無論是神經科醫生、患者還是護理人員，都表示治療負擔和疾病負擔比他們意識到的要高。因此，30 年來的第一次創新將帶來巨大的影響。

And I do think, to your question, over time, we will transform this market and reshape what is standard of care. Right now, we're very early in the launch. We're two quarters in. I think we're doing very well. 85% of our patients are switched from IVIg, and that's exactly where we thought we would be. So we're pleased with the momentum so far.

對於你的問題，我確實認為，隨著時間的推移，我們將改變這個市場並重塑護理標準。目前，我們正處於發布的早期階段。我們已經進行了兩個季度。我認為我們做得很好。我們有 85% 的患者從 IVIg 轉為使用 IVIg，這正是我們所期望的結果。我們對目前的勢頭感到滿意。

Yatin Suneja, Guggenheim.

古根漢的亞汀·蘇內賈（Yatin Suneja）。

This is [Selma] to for Yatin. So for seronegative and ocular NG trials, what's the dosing regimen in those studies? And based on the use of VYVGART in this population, would you anticipate that these patients will need a similar number of (inaudible) cycles as seropositive CMP or will they need more frequent dosing? And finally, how should we think about pricing in these indications?

這是 [賽爾瑪] 送給 Yatin 的。那麼對於血清陰性和眼部 NG 試驗，這些研究中的給藥方案是什麼？並且根據該族群對 VYVGART 的使用情況，您是否預期這些患者將需要與血清陽性 CMP 相似數量的（聽不清楚）週期數，或者他們是否需要更頻繁的給藥？最後，我們該如何考慮這些適應症的定價？

I think the answer to this question, thank you for the question, is very simple. We don't design and we don't anticipate any difference dosing in seronegative or ocular MG patients as compared to the general MG patients, which are (inaudible) antibody positive, which we have currently on label in the States.

我認為這個問題的答案（感謝您的提問）非常簡單。我們沒有設計，也不預期血清陰性或眼部 MG 患者的劑量與一般 MG 患者（（聽不清楚）抗體陽性，我們目前在美國的標籤上有此類藥物）相比有任何差異。

And we also don't anticipate any real pricing difference. I think that this is label expanding and is just broadening the offering to the MG community. So you can assume for the models exactly the same parameters. Thank you for the question.

我們也不預期會有任何實際的價格差異。我認為這是標籤的擴展，並且只是擴大了對 MG 社區的服務範圍。因此，您可以假設模型具有完全相同的參數。感謝您的提問。

-- Matt Phipps, William Blair.

——馬特·菲普斯、威廉·布萊爾。

I wanted to follow up on the myositis transition to Phase III. I'm just wondering if you're enrolling select -- a set number of patients for each of the 3 subsets so as to power each subset individually. Or will the primary be looked across all patients?

我想追蹤肌炎向第三階段的轉變。我只是想知道您是否正在為 3 個子集中的每個子集招募一定數量的患者，以便為每個子集單獨提供動力。或是否會對所有患者進行主要檢查？

Yeah, I think you're spot on, you will want to see a minimum representation of every subset in that Phase III registration trial in order to draw data based conclusions you can then also discuss with the FDA. So I think your underlying assumption is correct. Thank you.

是的，我認為你說得對，你會希望看到 III 期註冊試驗中每個子集的最小代表性，以便得出基於數據的結論，然後你也可以與 FDA 進行討論。所以我認為你的基本假設是正確的。謝謝。

Gavin Clark-Gartner, Evercore ISI.

加文·克拉克-加特納 (Gavin Clark-Gartner)，Evercore ISI。

For the prefilled syringe and the VBAs with the managed care organizations phasing out, I just wanted to clarify. In CIDP specifically, are you planning to not have the same cap on use in those contracts?

對於隨著管理式醫療組織逐步淘汰的預充式註射器和 VBA，我只是想澄清一下。具體來說，在 CIDP 中，您是否計劃在這些合約中不設定相同的使用上限？

So I think -- Gavin, it's Karl here. I think retail price negotiations would still need to take place for VBAs. But our expectation is that for a pharmacy benefit, that you would give incremental discounts because that's what you typically do.

所以我認為 —— 加文，這是卡爾。我認為 VBA 零售價格仍需要談判。但我們的期望是，對於藥房福利，您會給予增量折扣，因為這通常是您所做的。

But incremental-based rebates and that the VBAs would not be asked for or by the payers. So therefore, it will phase out. So therefore, those -- yeah, they won't be capped. Thank you for your question.

但是增量式回扣和 VBA 不會被付款人要求或提供。因此，它將逐步淘汰。因此，那些—是的，它們不會受到限制。感謝您的提問。

Samantha Semenkow, Citi.

Samantha Semenkow 的花旗銀行。

Perfect. I'm wondering, can you speak to your confidence in FDA granting the self-administration for the PFS? And perhaps you can just remind us on FDA's concerns surrounding self-administration for the fine needle process and how that's factored into your strategy as you look to secure self-administration for the PFS.

完美的。我想知道，您能否談談您對 FDA 授予 PFS 自我管理的信心？也許您可以提醒我們 FDA 對細針穿刺過程自我管理的擔憂，以及在您尋求確保 PFS 自我管理的策略中這是如何考慮到這一點的。

Yeah. So taking a step back, we feel we've submitted a very strong data set to the FDA with regards to the prefilled syringe, including, I think, a very solid human factor study, which is taking into account the typical questions a regulator may have on how patients can reliably and robustly manipulate the device. So we feel we're starting from a very strong data set. I think the review is on track based from (inaudible). We think we're on track for the PDUFA date of April 10.

是的。所以退一步來說，我們覺得我們已經向 FDA 提交了關於預充式註射器的一組非常強大的數據集，我認為其中包括一項非常可靠的人為因素研究，該研究考慮到了監管機構可能提出的典型問題，即患者如何可靠且穩健地操作該設備。因此，我們覺得我們是從一個非常強大的資料集開始的。我認為審查進展順利，因為（聽不清楚）。我們認為我們將按計劃於 4 月 10 日完成 PDUFA 程序。

And I think the type of questions which we're getting from the FDA signal that actually we are making good progress with the review of the file and that actually we are where we should be in light of the total review time line. So we are confident based on the data set and the currently ongoing process. Ultimately, of course, this is the final call to be made by the FDA, and we try to collaborate as much as we can. Thank you for the question.

我認為，我們從 FDA 得到的問題類型表明，我們在文件審查方面實際上正在取得良好進展，而且根據整體審查時間表，我們實際上已經達到了應有的水平。因此，根據數據集和目前正在進行的進程，我們很有信心。當然，這最終由 FDA 做出，我們會盡力合作。感謝您的提問。

Thomas Smith, Leerink Partners.

湯瑪斯‧史密斯 (Thomas Smith)，Leerink Partners。

Tim, you called out LN as an important proof-of-concept readout in the second half of this year. I know that's a study is being conducted by your partners in China, and they just recently completed enrollment. I was just wondering if you have a sense of sort of the baseline characteristics and disease severity of these patients and how they compare to other contemporary Western LN studies. And maybe you could just remind us how you're thinking about the bar for success with this readout.

提姆，您稱 LN 是今年下半年重要的概念驗證讀數。我知道這是你們在中國的合作夥伴正在進行的一項研究，他們最近才剛完成招募。我只是想知道您是否了解這些患者的基線特徵和疾病嚴重程度，以及與其他當代西方 LN 研究相比如何。也許您可以提醒我們您是如何看待這個讀數的成功標準的。

Yeah. Thomas, thank you for the question. First thing I want to do is applaud Zai as a reliable and high-quality development partner that's not only involved in this Phase II proof of concept studies but are also involved in a lot of the Phase III global registration trials with speed and high quality. So a very strong partnership.

是的。托馬斯，謝謝你的提問。我首先要讚揚再鼎醫藥，它是一家值得信賴的高品質開發合作夥伴，不僅參與了第二階段的概念驗證研究，還快速高品質地參與了大量第三階段的全球註冊試驗。因此這是一個非常牢固的夥伴關係。

And specifically to LN, the patient population which we are recruiting in China is, of course, perfectly in line with protocol and the inclusion/exclusion criteria, which is represented, I think, for that LN patient population with significant unmet medical needs. There are some variations in the type of treatment these patients on the go.

具體來說，對於狼瘡性腎炎 (LN) 患者來說，我們在中國招募的患者群體當然完全符合方案和納入/排除標準，我認為這代表了狼瘡性腎炎 (LN) 患者群體存在重大未滿足的醫療需求。這些患者接受的治療類型有一些差異。

But for us, this is Phase II, and it's a true Phase II, is a proof of concept. So the first question we need to answer in this Phase II trial is, are we spot on with this mechanism of action? When we dramatically reduce pathogenic IgGs, do we have the right to move the needle in this type of patients?

但對我們來說，這是第二階段，是真正的第二階段，也是一個概念驗證。因此，我們在第二階段試驗中需要回答的第一個問題是，我們是否正確了解這個作用機制？當我們大幅減少致病性 IgG 時，我們是否有權對這類患者採取治療措施？

That's a question we're going to answer. And once we have a positive answer to this question, we will flip it into a global Phase III registration trial, where we will take into account some of the global treatment regimens which are typically being used.

這就是我們要回答的問題。一旦我們對這個問題有了肯定的答案，我們就會將其轉變為全球第三階段註冊試驗，其中我們會考慮一些通常使用的全球治療方案。

So we take it step by step. And for the proof-of-concept question, I think Zai is perfectly equipped to help us address that question. So we're very much looking forward to the data. Thank you.

因此我們一步一步來。對於概念驗證問題，我認為 Zai 完全有能力幫助我們解決這個問題。因此我們非常期待這些數據。謝謝。

Leland Gershell, Oppenheimer.

利蘭‧格謝爾 (Leland Gershell)、奧本海默。

Just a question on 119. As we look forward to the proof-of-concept data in CMS later this year, just wondering if you could touch on what you are looking to see? And could this data impact the ongoing development of 119 in its other indications?

這只是關於 119 的一個問題。我們期待今年稍後 CMS 中的概念驗證數據，想知道您是否可以談談您希望看到什麼？這些數據是否會影響 119 在其他適應症上的持續發展？

Now I like the question about 119. Thank you for that. As an indication, congenital myasthenic syndrome is really in the bull's eye of the biology of this target. Remember, we have been publishing and presenting spectacular data in the DOK7 animal model, which is perfectly mimicking the DOK7 mutation in human beings, and that's exactly where we're testing for CMS. Think of a genetic form of MG which has overlapped with autoimmune energy.

現在我喜歡有關119的問題。謝謝你。作為一個跡象，先天性肌無力症候群確實是這一目標的生物學焦點。請記住，我們一直在發布和展示 DOK7 動物模型中的驚人數據，它完美地模擬了人類的 DOK7 突變，這正是我們測試 CMS 的地方。想像一下與自體免疫能量重疊的 MG 遺傳形式。

It's fatigable. It's typically limb-girdle which is being affected, but also the eyes. So we are basically looking for the strong signal that we move the needle in these patients. So I think it is a very important program from a proven biology point of view. But remember, we're running in parallel also a proof-of-concept study in ALS, and we are bringing life now to study in SMA.

它很容易疲勞。通常會影響肢帶，但眼睛也會受到影響。因此，我們基本上是在尋找能對這些患者產生影響的強烈訊號。因此我認為從已證實的生物學角度來看這是一個非常重要的項目。但請記住，我們也在同時進行 ALS 的概念驗證研究，現在我們正在將 SMA 的研究付諸實行。

I would say that all these three indications are very close to the mechanism of action of the molecule. But CMS, I think, is really close. So that is the significance of this first indication for ARGX-119. We're very much looking forward to the data, by the way. Thank you for the question.

我想說，這三種適應症都非常接近分子的作用機制。但我認為 CMS 已經非常接近了。這就是 ARGX-119 首次適應症的意義。順便說一句，我們非常期待這些數據。感謝您的提問。

Joon Lee, Truist Securities.

Joon Lee，Truist Securities。

Congrats on the quarter. This is (inaudible) on for Joon. A question related to your early pipeline assets. We appreciate more color on your plans for ARGX-109, anti-IL-6, based on the importance of the target but also given its interesting history of about 15 years and its 200 patients Phase II in RA in Brazil. And also, it's finally getting back to you. We appreciate the color there.

恭喜本季取得佳績。這是（聽不清楚）為 Joon 準備的。與您早期的管道資產相關的一個問題。我們很欣賞您對 ARGX-109（抗 IL-6）計劃的更多細節，不僅基於該目標的重要性，還考慮到其約 15 年的有趣歷史及其在巴西的 200 名 RA 患者 II 期研究。而且，它最終還是會回到你身邊。我們欣賞那裡的色彩。

I think you're calling out the strength of the molecule. I think this is a best-in-class IL-6 blocker with (inaudible) potency, a 60 to 80 days half-life and already proven safety profile in 2 Phase Is, actually one in Brazil and one in China. Happy to finally get this molecule back. And there's a whole new angle, which we could take in the meantime on the biology of IL-6 and how it is involved in some real interesting autoimmune diseases. So we decided not to talk too much about these indications yet.

我認為你正在彰顯分子的力量。我認為這是一流的 IL-6 阻斷劑，具有 (聽不清楚) 效力、60 至 80 天的半衰期，並且已經在 2 個 I 期試驗中證明了其安全性，實際上一個在巴西，一個在中國。很高興終於找回了這個分子。同時，我們可以從一個全新的角度來了解 IL-6 的生物學以及它如何參與一些真正有趣的自體免疫疾病。因此我們決定暫時不要過度談論這些跡象。

But I think we have a unique and novel angle to the biology, and we're progressing the molecule this year at high speed through Phase I. We're expecting the Phase I data in second half of this year. So take tuned. More to be told online soon. Thank you.

但我認為我們對生物學有一個獨特而新穎的角度，今年我們正在第一階段高速推進這項分子研究。我們預計第一階段的數據將在今年下半年公佈。因此請留意。更多內容將在網路上發布。謝謝。

Joel Beatty, Baird.

喬爾·比蒂，貝爾德。

This is Chris on for Joel. Just a quick one on PFS. If they're approved,in April, when -- what are your expectations for when they can reach patients?

這是克里斯代替喬爾。關於 PFS 簡單介紹一下。如果它們在四月獲得批准，您預計它們何時可以到達患者手中？

Yeah. Thanks for the question. So we're very excited obviously for the PFS -- for self-injection potential approval in April, as you said. We'll be ready to launch as so after the PDUFA date in the same way that we have for prior launches. So we expect to be within days that will be out there with PFS for self-injection.

是的。謝謝你的提問。因此，正如您所說，我們對於 PFS —— 4 月份自我注射潛力的批准感到非常興奮。我們將在 PDUFA 日期之後做好發布準備，方式與先前的發布相同。因此，我們預計幾天之內就會推出可供自我注射的 PFS。

Douglas Tsao, H.C. Wainwright.

曹國偉，H.C.溫賴特。

Maybe, Karen, I think it would be helpful to hear you talk a little bit about how you think potential approval in ocular MG might affect sort of the treatment paradigm for MG, and whether you sort of might see that push earlier, obviously, with some of the patients present with ocular symptoms first, but sort of really whether clinicians might view it a sort of disease-modifying therapy at that stage.

也許，凱倫，我想聽您談談您認為該藥物在眼部重症肌無力治療中的潛在批准將如何影響重症肌無力的治療模式，以及您是否可能更早地看到這種推動，顯然，一些患者首先出現眼部症狀，但實際上臨床醫生是否會在那個階段將其視為一種改善病情的療法。

Yeah. Thank you for the question. And I like how you frame it up as disease-modifying. Look, we'll have to let the data speak. But the way that I think about it is very much aligned with how you're thinking about it.

是的。感謝您的提問。我喜歡你將其描述為改變疾病的方式。瞧，我們必須讓數據說話。但我的想法和你的想法非常一致。

Our strategy is that we believe that treatment with VYVGART in earlier lines results in better outcomes for patients. And that's why we also wanted to move all the way into the MGFA classification I with ocular MG. And we will be the first and only that would have that indication.

我們的策略是，我們相信早期使用 VYVGART 治療會為患者帶來更好的結果。這就是為什麼我們希望將眼部 MG 完全轉入 MGFA 分類 I。我們將是第一個也是唯一一個有此跡象的人。

We know that 80% of patients with ocular MG generalize. And as you say, potentially over time, not with this first data readout, but maybe over time, we can imagine that you could see data in the real world where we're able to delay that generalization.

我們知道，80% 的眼部 MG 患者俱有全身性。正如您所說，可能隨著時間的推移，不是透過第一次讀取數據，而是隨著時間的推移，我們可以想像您可以在現實世界中看到數據，我們能夠延遲這種概括。

And I think that's really exciting for MG patients. And certainly, that's our vision for how we would transform the market. Let's see how the data plays out, but we're relatively confident and have really strong conviction in the strategy. Thanks for the question.

我認為這對 MG 患者來說真的令人興奮。當然，這就是我們改變市場的願景。讓我們看看數據如何發揮作用，但我們相對有信心，並且對該策略有著堅定的信念。謝謝你的提問。

Manos Mastorakis, Deutsche Bank.

馬諾斯‧馬斯托拉基斯，德意志銀行。

Manos Mastorakis from Deutsche Bank. So how do you think about the potential for competitors to further improve upon clinical outcomes in CIDP above efgartigimod outcomes. And do you believe complement inhibitors have a role to play in this space?

德意志銀行的馬諾斯·馬斯托拉基斯。那麼您如何看待競爭對手在 CIDP 臨床結果方面進一步改善以超越 efgartigimod 結果的潛力。您是否認為補體抑制劑在這個領域發揮作用？

Yeah, thank you for the question. I think in CIDP, we have written history because we have shown for the first time that actually this is an IgG-driven disease in the majority of patients. And I think we have shown a response rate which is the highest ever reported in this type of clinical trials.

是的，謝謝你的提問。我認為在 CIDP 中我們書寫了歷史，因為我們首次證明這實際上對大多數患者來說是一種由 IgG 驅動的疾病。我認為我們的反應率是此類臨床試驗中有史以來最高的。

So it is 70% response rate, a safety profile which is very much in line with the nonsafety profile, which we all recognize as pretty unique in this world. And then, of course, we're advancing fast with the dosing optionality, which we have been unpacking in this call.

因此，它的回應率為 70%，安全性概況與非安全性概況非常一致，我們都認為這在世界上是相當獨特的。然後，當然，我們正在快速推進劑量可選性問題，我們已經在這次電話會議中對此進行了探討。

So we've put the bar very high. We need to see data, of course, from competing molecules in the class that they take it. But we have certainly not seen that in myasthenia. We were also first to market.

因此我們的標準非常高。當然，我們需要了解同類競爭分子的數據。但我們在重症肌無力症中確實沒有見過這種情況。我們也是第一個進入市場的。

Is there a offer complement? I think there is. That's also why we're advancing empasiprubart in CIDP despite the fact that we printed the highest response rate average 70%. That is 30% of patients which have a medical need and were not served in the ADHERE trial, and we do know there are some clues from a biology point of view, for example, some patients in these pathogenic IgM autoantibodies, which do recruit a complement and which do not recycle through FcRn. So I think we're unraveling the answer to your question.

有沒有優惠補充？我認為有。這就是為什麼儘管我們的最高回應率平均為 70%，但我們仍要在 CIDP 中推進 empasiprubart。這意味著 30% 的患者有醫療需求，但未在 ADHERE 試驗中得到服務，我們確實知道從生物學的角度有一些線索，例如，一些患者體內的這些致病性 IgM 自身抗體確實會招募補體，但不會透過 FcRn 進行循環。所以我想我們正在解開你的問題的答案。

I think empasiprubart really deserves its goal, and we're very keen to start this study and look at complement data. So there's a lot to be unpacked and a lot to be developed in CIDP. Thank you.

我認為 empasiprubart 確實值得實現其目標，我們非常渴望啟動這項研究並查看補體數據。因此，CIDP 中還有很多內容有待解決，也有很多內容有待開發。謝謝。

And we have reached the end of our question-and-answer session. This concludes today's conference call. Thank you for your participation. You may now disconnect.

我們的問答環節已經結束。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。