argenx SE (ARGX) 2024 Q2 法說會逐字稿

Good morning, my name is Audra, and I will be your conference operator today. At this time, I would like to welcome everyone to the argenx second quarter 2024 financial results conference call. Today's conference is being recorded. (Operator Instructions) At this time, I would like to turn the conference over to investor relations, Beth DelGiacco. Please go ahead.

早安，我叫奧德拉，今天我將擔任你們的會議操作員。此刻，我歡迎大家參加 argenx 2024 年第二季財務業績電話會議。今天的會議正在錄製中。（操作員指示） 現在，我想將會議轉交給投資人關係部 Beth DelGiacco。請繼續。

A press release was issued earlier today with our half-year 2024 financial results and second quarter business update. This can be found on our website, along with a presentation for today's webcast. Before we begin, I would like to remind you that forward-looking statements may be presented during this call.

今天早些時候發布了一份新聞稿，其中介紹了我們的 2024 年半年財務業績和第二季度業務更新。您可以在我們的網站上找到該資訊以及今天網路廣播的簡報。在我們開始之前，我想提醒您，本次電話會議期間可能會提出前瞻性陳述。

These may include statements about our future expectations, clinical developments, regulatory timelines, the potential success of our product candidates, financial projections, and upcoming milestones. Actual results may differ materially from those indicated by these statements.

這些可能包括有關我們未來預期、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。實際結果可能與這些陳述所示的結果有重大差異。

Argenx is not under any obligation to update statements regarding the future or to confirm their statements in relation to actual results unless required by law. 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.

除非法律要求，Argenx 沒有義務更新有關未來的聲明或確認其與實際結果相關的聲明。今天，執行長 Tim Van Hauwermeiren 也加入了我的電話會議。卡爾‧古比茨，財務長；和首席營運長凱倫·梅西 (Karen Massey)。

I'll now turn the call over to Tim.

我現在將電話轉給蒂姆。

Thank you, Beth, and welcome, everyone. I'll begin on slide three. Last week, many of you joined us for our R&D Day, where we laid out an ambitious vision in 2030 and shared a comprehensive overview of the growth opportunities ahead in our goal to achieve this vision. We are in a very strong position today to generate substantial value across our business, investing in our internal innovation engine, executing on our differentiated multi-asset pipeline, and building on the commercial success we have achieved in our first 10 quarters of launch.

謝謝你，貝絲，歡迎大家。我將從第三張投影片開始。上週，你們中的許多人參加了我們的研發日，我們在會議上製定了 2030 年雄心勃勃的願景，並全面概述了實現這一願景的目標中未來的成長機會。今天，我們處於非常有利的地位，可以在整個業務中創造巨大的價值，投資我們的內部創新引擎，執行我們差異化的多資產管道，並在我們推出的前10 個季度取得的商業成功的基礎上再接再厲。

This is a team that delivers on our innovation mission time and time again all towards our goal to transform autoimmunity and reach 50,000 patients globally. Given the recency of this update, we will aim to keep our prepared remarks brief and focus primarily on the second quarter dynamics. But first, I'd like to highlight key themes that emerged from the R&D Day in the context of our 2030 vision.

這個團隊一次又一次地履行我們的創新使命，以實現我們改變自身免疫並惠及全球 50,000 名患者的目標。鑑於本次更新的時間較近，我們的目標是保持準備好的評論簡短，並主要關注第二季的動態。但首先，我想強調研發日在我們 2030 年願景背景下出現的關鍵主題。

Slide four. The first theme is around how we innovate, centered on a model of co-creation. This model has been core to our growth since our founding and still exists in the most obvious form today with our immunology innovation program.

幻燈片四。第一個主題是圍繞著我們如何創新，以共同創造的模式為中心。自我們成立以來，這種模式一直是我們發展的核心，並且在今天我們的免疫學創新計劃中仍然以最明顯的形式存在。

We have a strong track record of success in building out our differentiated pipeline, including eight out of 12 molecules that demonstrated human proof of concepts and nine that are first-in-class targets. We have no shortage of opportunity with identifying novel targets and believe our overall approach sets us up to drive continued pipeline expansion for years to come.

我們在建立差異化產品線方面擁有良好的成功記錄，其中包括 12 個分子中的 8 個分子，其中有 8 個分子展示了人類概念驗證，還有 9 個分子是一流的目標。我們不缺乏確定新目標的機會，並相信我們的整體方法將使我們能夠在未來幾年推動管道的持續擴張。

A second theme is the new standards we're setting with VYVGART in MG. We are currently de-leading advanced biologic in MG and have delivered 10 consistent quarters of growth since our initial launch. We are making excellent progress across all key indicators and are exactly in line with our internal long-term thinking of the MG marketplace.

第二個主題是我們在 MG 中為 VYVGART 設定的新標準。目前，我們正在 MG 領域的先進生物製劑領域取得領先地位，自首次推出以來，我們已經實現了 10 個季度的持續成長。我們在所有關鍵指標上都取得了出色的進展，這完全符合我們內部對 MG 市場的長期思考。

We think this is a big opportunity and we have a strategic plan to reach more patients as we move into earlier lines of treatment and broaden to new populations through label-enabling studies in seronegative and ocular MG patients. We will use the same playbook for CIDP and are happy with the early days of the launch. We got the best possible label which puts us in a strong position for continued growth as we work to get the excess piece in place.

我們認為這是一個巨大的機會，我們制定了一項戰略計劃，隨著我們進入早期治療線並通過對血清陰性和眼部 MG 患者進行標籤支持研究擴展到新人群，我們將覆蓋更多患者。我們將為 CIDP 使用相同的劇本，並對發布的早期階段感到滿意。我們獲得了最好的標籤，這使我們在努力將多餘的部分安裝到位時處於持續增長的有利地位。

Third, we laid out our next wave of indications and expect to initiate four new registration trials this year across VYVGART and Empasiprubart. We set the bar high with a medicine like VYVGART but we're thrilled to use the R&D day to put Empasiprubart more in the spotlight. This is a second pipeline in a product opportunity and we were able to show phenomenal patient data in MMN which we see as the first of many indications where we hope to drive transformational impact.

第三，我們列出了下一波適應症，並預計今年在 VYVGART 和 Empasiprubart 上啟動四項新的註冊試驗。我們對 VYVGART 這樣的藥物設定了很高的標準，但我們很高興能利用研發日讓 Empasiprubart 更加受到人們的關注。這是產品機會中的第二個管道，我們能夠在 MMN 中顯示驚人的患者數據，我們認為這是我們希望推動變革影響的眾多跡像中的第一個。

Slide five. The progress we have made across our business over the quarter moves us another step closer towards achieving our 2030 vision to bring five molecules to Phase 3, advance our late stage pipeline to 10 labeled indications across our molecules, and to ultimately have 50,000 patients on an argenx medicine by 2030.

幻燈片五。本季我們在業務上取得的進展使我們離實現2030 年願景又近了一步，即將5 種分子帶入3 期，將我們的後期產品線推進到分子中的10 種標記適應症，並最終讓50,000 名患者接受治療。

While ambitious, we believe we're well positioned to deliver on this goal with a continuum of innovation from discovery all the way to commercial. Underpinning our incredible success to date is the strong financial health of our business which will enable us to continue delivering on our innovation mission, ultimately driving value for our patients and our shareholders.

儘管雄心勃勃，但我們相信我們有能力透過從發現一直到商業化的持續創新來實現這一目標。迄今為止，我們業務強勁的財務狀況支撐著我們取得令人難以置信的成功，這將使我們能夠繼續履行我們的創新使命，最終為我們的患者和股東創造價值。

With that, I would like to turn over the call to Karl.

說到這裡，我想把電話轉給卡爾。

Slide six. Thank you, Tim. The second quarter 2024 financial results are detailed in the press release of this morning. Total operating income in the second quarter is $489 million. This reflects $478 million in product net sales and $12 million in other operating income. In the second quarter, the $478 million in product net sales is attributed solely to MG patients.

幻燈片六。謝謝你，提姆。今天早上的新聞稿詳細介紹了 2024 年第二季的財務表現。第二季總營業收入為4.89億美元。這反映了 4.78 億美元的產品淨銷售額和 1,200 萬美元的其他營業收入。第二季度，4.78 億美元的產品淨銷售額僅來自重症肌無力症患者。

We received the CIDP approval on June 21, and our teams were immediately in action the next day. It takes time at the start of a launch to get patients through the funnel from script to injection, so the first CIDP revenue will be a third quarter event. Product net sales of $478 million represents 20% growth quarter over quarter compared to Q1 '24, which we know with a biologic is typically a more challenging quarter due to insurance re-verifications and loss of shipping days with weather and holidays.

我們於 6 月 21 日收到 CIDP 批准，我們的團隊第二天就立即採取行動。在啟動之初，讓患者完成從腳本到注射的整個流程需要時間，因此第一個 CIDP 收入將是第三季的事件。產品淨銷售額為4.78 億美元，與24 年第一季相比，季度環比增長20%，我們知道，由於保險重新驗證以及天氣和假期造成的運輸天數損失，生物製劑通常是一個更具挑戰性的季度。

The product revenue breaks down by region to $407 million in the US, $20 million in Japan, $35 million in EMEA, and $14 million from product supply to Zai Lab in China. Operating expenses in Q2 are $535 million, an increase of $29 million compared with Q1 2024. SG&A expenses are $256 million in Q2, which is an increase of $20 million compared to Q1.

產品收入按地區劃分為：美國為 4.07 億美元，日本為 2,000 萬美元，歐洲、中東和非洲為 3,500 萬美元，中國再鼎醫藥的產品收入為 1,400 萬美元。第二季營運支出為 5.35 億美元，較 2024 年第一季增加 2,900 萬美元。第二季的 SG&A 費用為 2.56 億美元，比第一季增加了 2,000 萬美元。

The increase reflects a full quarter impact of the expansion of a customer-facing organization in the US, as well as incremental expenses to prepare for the CIDP launch. R&D expenses on the continued investment in your pipeline are $225 million in the second quarter, broadly in line with the previous quarter. The operating loss of a quarter of $45 million is offset by financial income of $30 million and the tax benefit of $44 million.

這一成長反映了美國一家面向客戶的組織擴張對整個季度的影響，以及為準備 CIDP 啟動而增加的費用。第二季持續投資管道的研發費用為 2.25 億美元，與上一季基本持平。四分之一的營運虧損 4,500 萬美元被 3,000 萬美元的財務收入和 4,400 萬美元的稅收優惠所抵銷。

The tax benefit is a result of a deferred tax asset recognized in your US legal entity due to intra-group inventory movements. It is a temporary timing difference and will reverse in future quarters. Overall, we saw a net profit for the quarter of $29 million and earnings per share of $0.49. It is an important distinction, but the net profit was primarily driven by the recognition of a deferred tax asset, and we still had an operating loss of a quarter of $45 million. We are on a clear path to profitability, but we are not there yet.

稅務優惠是由於集團內部庫存變動而在您的美國法人實體中確認的遞延稅資產的結果。這是暫時的時間差異，並將在未來幾季扭轉。總體而言，本季淨利潤為 2,900 萬美元，每股收益為 0.49 美元。這是一個重要的區別，但淨利潤主要是由遞延稅資產的確認所驅動的，而且我們仍然有四分之一的營運虧損 4500 萬美元。我們正走在一條明確的獲利之路上，但目前還沒有實現。

We continue to have a strong balance sheet with $3.1 billion in cash. Based on our year-to-date cash burn of $77 million, we are updating our 2024 cash burn guidance from approximately $500 million to less than $500 million. Our guidance on expenses remains unchanged. This puts us in a strong position to invest in innovation across our business, and we have many opportunities to do so.

我們的資產負債表依然強勁，現金達 31 億美元。根據我們今年迄今 7,700 萬美元的現金消耗，我們將 2024 年現金消耗指引從約 5 億美元更新至不到 5 億美元。我們對費用的指導保持不變。這使我們在整個業務的創新投資方面處於有利地位，而且我們有很多機會這樣做。

There's a sizable opportunity in our current markets, in new markets with our VYVGART franchise, with our current pipeline, and through our immunology innovation program to expand our pipeline. This is how we will set ourselves up for a sustainable future where we continue to generate significant value for our shareholders.

在我們目前的市場、透過我們的 VYVGART 特許經營權、我們現有的產品線以及透過我們的免疫學創新計劃來擴展我們的產品線的新市場中，存在著相當大的機會。這就是我們為永續發展的未來做好準備的方式，我們將繼續為股東創造巨大的價值。

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

我現在將把電話轉給凱倫，她將提供商業方面的詳細資訊。

Thank you, Karl. Slide eight. I'm very excited by our vision 2030, especially the ambition we have set for ourselves to impact the lives of 50,000 patients globally and their caregivers. To reach this goal, we will need to expand in a multi-dimensional way, getting more medicines approved across many indications, product presentations, and geographies.

謝謝你，卡爾。幻燈片八。我對我們的 2030 年願景感到非常興奮，尤其是我們為自己設定的影響全球 50,000 名患者及其護理人員生活的雄心。為了實現這一目標，我們需要以多維度的方式進行擴張，讓更多的藥物在多種適應症、產品展示和地理上獲得批准。

We have a strong commercial playbook in place that delivered in MG, and we'll be leveraging that same approach for CIDP, focusing on evidence generation that matters most to physicians and patients, empowering patients to demand more from their treatments, and last, the need to execute on our strategies with speed and urgency. We know that patients are waiting, and we also know that every day counts in building out our first-in-class leadership position.

我們在 MG 領域擁有強大的商業劇本，我們將在 CIDP 中利用同樣的方法，專注於對醫生和患者最重要的證據生成，使患者能夠從他們的治療中提出更多要求，最後，需要快速而緊迫地執行我們的策略。我們知道患者正在等待，我們也知道每一天對於建立我們一流的領導地位都很重要。

Slide nine. Starting with MG, I'm extremely proud of the team who worked really hard to expand our reach into new patients for our 10 consecutive quarter of revenue growth. We said this in Q1, and can reiterate it today, that all key indicators of growth are performing well, and the fundamentals to the commercial business are strong.

幻燈片九。從 MG 開始，我為我們的團隊感到非常自豪，他們非常努力地將我們的業務範圍擴大到新患者，從而實現了我們連續 10 個季度的收入成長。我們在第一季就說過這一點，今天可以重申，所有關鍵成長指標都表現良好，商業業務的基本面強勁。

Focusing on the key drivers of growth this quarter, we saw the seasonality impact in Q1, normalizing Q2, with the impressive 17% quarter-over-quarter growth. Within the US, Hytrulo continues to attract both new patients and prescribers to the VYVGART franchise. This is important for a few reasons.

關注本季成長的主要驅動因素，我們看到第一季的季節性影響，第二季正常化，季增 17%，令人印象深刻。在美國，Hytrulo 繼續吸引新患者和處方者使用 VYVGART 特許經營權。這很重要，原因有幾個。

First, over 50% of new Hytrulo patients are coming from Oral, and 60% are brand new to VYVGART, which is consistent with our goal to reach early aligned patients and to expand new patients with Hytrulo. Second, we continue to expand our prescriber base for MG, many of whom have the potential to be CIDP prescribers. This will serve us well in our early conversations with physicians who have grown accustomed to the favorable safety and efficacy profile of VYVGART Hytrulo.

首先，超過 50% 的新 Hytrulo 患者來自 Oral，60% 是 VYVGART 的新患者，這與我們接觸早期對齊患者並擴大 Hytrulo 新患者的目標是一致的。其次，我們繼續擴大 MG 處方者基礎，其中許多人有潛力成為 CIDP 處方者。這對於我們與已經習慣了 VYVGART Hytrulo 良好的安全性和有效性的醫生的早期對話很有幫助。

Slide 10. We continue to gain traction outside the US as well, where we are focused on securing broad access. We have strong momentum in EMEA, and VYVGART is now available in countries representing 82% of the gMG population in the region. We also continue to see strong growth in Japan, including positive early indicators on the launch of ITP.

幻燈片 10。我們在美國以外的地區也繼續受到關注，我們專注於確保廣泛的准入。我們在歐洲、中東和非洲地區發展勢頭強勁，VYVGART 現已在佔該地區 gMG 人口 82% 的國家推出。我們也持續看到日本的強勁成長，包括 ITP 推出的正面早期指標。

This launch rolled out rapidly, and 74% of HCPs were made aware of VYVGART in the weeks post-approval. It is clear that there is a need for innovation in ITP, which is why we are also launching an efficient label-enabling study with [VYVGART IV] in the US after discussions with the FDA. Lastly, in China, we continue to be very impressed by patient ads each quarter, and now Subcutaneous is also available, following the approval earlier this month.

此次發布迅速推出，7​​4% 的 HCP 在批准後幾週內就知道了 VYVGART。顯然，ITP 需要創新，這就是為什麼我們在與 FDA 討論後也在美國啟動了一項有效的標籤支持研究 [VYVGART IV]。最後，在中國，每季的病患廣告仍然給我們留下了深刻的印象，在本月初獲得批准後，現在皮下注射也可以使用。

Slide 11. Even with our strong performance to date, we believe that we're still at the front end of the opportunity in MG. We are seeing the MG market expand, a dynamic that we have seen play out in other rare disease markets when innovation enters the space. We believe that early use of advanced biologics will expand the addressable market significantly, and that we can reach broader populations of MG patients with our label expansion studies in seronegative and ocular MG, each of which represent 15% of the total MG population. Based on these evolving dynamics, we have updated our addressable market in MG to 60,000 patients.

幻燈片 11。儘管我們迄今為止表現強勁，但我們相信我們仍然處於 MG 機會的前端。我們看到 MG 市場正在擴張，當創新進入該領域時，我們也看到了其他罕見疾病市場的動態。我們相信，先進生物製劑的早期使用將顯著擴大潛在市場，並且我們可以透過血清陰性和眼部MG 的標籤擴展研究涵蓋更廣泛的MG 患者群體，這兩種類型分別佔MG 總人群的15% 。基於這些不斷變化的動態，我們已將 MG 的目標市場更新為 60,000 名患者。

Slide 12. Following our approval in June, we recognize that all eyes are on CIDP, and we share your excitement. We are thrilled with the broad label that we got, which will support use across the treatment paradigm. Now, we're leveraging our MG launch playbook to maximize our impact in CIDP, driving rapid adoption with neurologists based on the strength of our data, empowering patients, and working diligently with payers to put the necessary policies in place to secure access as quickly as possible.

幻燈片 12。繼 6 月獲得批准後，我們認識到所有人的目光都集中在 CIDP 上，我們也和您一樣興奮。我們對我們獲得的廣泛標籤感到興奮，這將支持在整個治療範式中的使用。現在，我們正在利用MG 發布手冊來最大限度地發揮我們在CIDP 中的影響力，根據我們的數據實力推動神經科醫生的快速採用，為患者提供支持，並與付款人努力合作，制定必要的政策以確保盡快獲得訪問盡可能。

Reception has been incredibly positive with physicians, particularly on the strength of the data and improvement in function. We've already reached 25% of our key targets within 14 days of launch, and 20% of the prescriptions we're receiving are from new prescribers to VYVGART.

醫生的反應非常積極，特別是在數據的強度和功能的改善方面。在推出後 14 天內，我們已經實現了 25% 的關鍵目標，並且我們收到的處方中有 20% 來自 VYVGART 的新處方者。

Many of the prescribers are starting multiple patients. Right now, our priority is to bring as many patients into the top of the funnel as possible. Once an enrollment comes in, payer approval at this stage takes a few weeks, after which patients can be scheduled for their first injection. This shows the importance of getting payer policies in place quickly. And as this happens, the time from script to injection gets shorter. Based on where we are today, we're making good progress with payers and we're tracking to plan.

許多處方醫生正在為多名患者開處方。現在，我們的首要任務是讓盡可能多的患者進入漏斗頂部。一旦登記開始，付款人在此階段的批准需要幾週時間，之後患者就可以安排第一次注射。這表明快速制定付款人政策的重要性。當這種情況發生時，從腳本到注入的時間就會變短。根據我們今天的情況，我們在付款人方面取得了良好進展，並且正在追蹤計劃。

Slide 13. Looking forward, we have an ambitious plan to successfully execute multiple launches across our pipeline in order to reach 50,000 patients in 2030. We're on track to start multiple Phase 3 studies before the end of the year. And each of these brings us a step closer to reaching new autoimmune indications where there is a high unmet need.

幻燈片 13。展望未來，我們有一個雄心勃勃的計劃，要在我們的產品線中成功進行多次上市，以便在 2030 年覆蓋 50,000 名患者。我們預計在今年年底前啟動多項三期研究。每一項都讓我們離實現新的自體免疫適應症又更近了一步，而這些適應症的需求尚未被滿足。

The TED registrational study has already started, and we see an opportunity here to provide a differentiated and targeted therapy with favorable safety. We look forward to initiating a Phase 3 study in Sjogren's before year end, following the positive signal we saw in our Phase 2 proof of concept study.

TED 註冊研究已經開始，我們在這裡看到了提供安全性良好的差異化、標靶治療的機會。繼我們在第二階段概念驗證研究中看到正面訊號之後，我們期待在年底前啟動 Sjogren 的第三階段研究。

There are no approved treatments for Sjogren's patients, and we know the disease goes well beyond sicker symptoms and can affect patients' ability to work and complete daily tasks, particularly the fatigue. This is a sizable opportunity, and while data will drive the specific target population, we know that there are 100,000 moderate to severe Sjogren's patients in the US. And last, we also look forward to decisions in myositis and BP on whether to advance to Phase 3 later this year.

目前還沒有針對乾燥症患者的核准治療方法，我們知道這種疾病遠遠超出了較嚴重的症狀，還會影響患者的工作和完成日常任務的能力，尤其是疲勞。這是一個相當大的機會，雖然數據將驅動特定的目標族群，但我們知道美國有 10 萬名中度至重度乾燥症患者。最後，我們也期待今年稍後肌炎和血壓方面的決定是否進入第三階段。

Finally, before year end, we will start a Phase 3 trial with our second molecule, empasiprubart, in MMN. This is an indication that fits perfectly into our neuromuscular focus, and it is another indication where the unmet need is high, and IVIG is the only approved treatment.

最後，在年底之前，我們將在 MMN 中開始使用我們的第二種分子 empasiprubart 進行 3 期試驗。這是一個非常適合我們神經肌肉重點的適應症，也是另一個未滿足需求的適應症，IVIG 是唯一被批准的治療方法。

The data we generated in Phase 2 were tremendous and very meaningful to patients. We demonstrated consistent improvement in grip strength, and in cohort one, 94% of patients felt better on empasiprubart than they did compared to their peak with IVIG. Similar to MG and CIDP, we believe this is an underdeveloped market, likely to grow over time as more innovation enters.

我們在第二階段產生的數據非常巨大，對患者非常有意義。我們證明了握力的持續改善，在隊列一中，94% 的患者在使用 empasiprubart 時感覺比使用 IVIG 時的峰值感覺更好。與 MG 和 CIDP 類似，我們認為這是一個不發達的市場，隨著更多創新的進入，可能會隨著時間的推移而成長。

Overall, this is an exciting time for the company. We've seen phenomenal growth to date, and we're eager to continue with this momentum as we enter into the second half of this year, applying innovation to every aspect of our business to reach even more patients.

總的來說，這對公司來說是一個令人興奮的時刻。到目前為止，我們已經看到了驚人的成長，我們渴望在進入今年下半年時繼續保持這種勢頭，將創新應用到我們業務的各個方面，以覆蓋更多的患者。

I'll turn it back to you, Tim.

我會把它還給你，提姆。

Thanks, Karen. Slide 14. I'm proud of the argenx's team and their tireless commitment to changing the lives of patients through our science. We are perfectly positioned with the right team and the right approach to execute on the broad opportunity ahead of us.

謝謝，凱倫。幻燈片 14。我為 argenx 的團隊以及他們透過我們的科學改變患者生活的不懈承諾感到自豪。我們處於完美的位置，擁有合適的團隊和正確的方法來執行我們面前的廣泛機會。

We will continue to lead with the strength of our data and deep knowledge to address the sizable MG opportunity ahead and will apply the same rigor with CIDP. And this is just the beginning. We are eager to raise the bar for how autoimmune diseases are treated as we continue to expand our horizons, embarking on new paths to create long-term value for patients and shareholders.

我們將繼續憑藉我們的數據優勢和深厚知識來應對未來巨大的 MG 機遇，並將與 CIDP 一樣嚴格。而這只是開始。隨著我們不斷拓展視野，開闢新道路，為患者和股東創造長期價值，我們渴望提高自體免疫疾病治療的標準。

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

感謝您今天抽出時間。我現在想打開電話詢問您的問題。

(Operator Instructions) James Gordon at JPMorgan.

（操作員指令）摩根大通的詹姆斯·戈登。

Hello. James Gordon, JPMorgan. Thanks for taking the question. Two questions, please. First question was on VYVGART performance in Q2 and the extent to which we can extrapolate that to Q3. Sales were up $79 million, which was a strong performance. But how much, if any, would you say was one-off phasing that we should take out if we're thinking about what the underlying performance would be?

你好。詹姆斯‧戈登，摩根大通。感謝您提出問題。請教兩個問題。第一個問題是關於 VYVGART 在第二季的表現以及我們可以在多大程度上推斷到第三季。銷售額成長了 7,900 萬美元，表現強勁。但是，如果我們考慮潛在的績效，您認為我們應該採取多少一次性分階段措施（如果有的話）？

And if we're thinking about doing our Q3 modelling, how careful do we need to be about any reversals or seasonality, or can we make quite a strong extrapolation from how well VYVGART has done today? That's the first question, please.

如果我們正在考慮進行第三季建模，我們需要對任何逆轉或季節性有多謹慎，或者我們可以根據 VYVGART 今天的表現做出相當有力的推斷？這是第一個問題，請。

The second question was Phase 3 initiation for ITP. And I saw a comment or heard a comment about this being an efficient trial. How might this trial differ from the previous two Phase 3 you've done in ITP? Can you expedite it if it's an efficient trial? And would it be going for a smaller patient population in some way or a similar size population, just a different design?

第二個問題是 ITP 第三階段的啟動。我看到或聽到評論說這是一次有效的試驗。此試驗與您在 ITP 中進行的前兩個第 3 階段有何不同？如果這是一次有效的審判，你能加快速度嗎？它會以某種方式針對較小的患者群體還是類似規模的人群，只是設計不同？

Thank you, James. Thank you for asking these two questions. I would like to hand over to Karen to comment on the underlying dynamics of the business and how to see the overall trends in our VYVGART MG business first. And then I will take the question on the ITP study in Phase 3.

謝謝你，詹姆斯。感謝您提出這兩個問題。我想先請 Karen 評論一下業務的基本動態以及如何看待我們 VYVGART MG 業務的整體趨勢。然後我會回答第三階段ITP研究的問題。

Karen, why don't you go ahead with question 1?

凱倫，你為什麼不繼續問題1？

Thanks, Tim. And yes, thanks for the question and for recognizing. I think -- like you, I'm really pleased with the results of the quarter -- this quarter. The way that I would think about it is that when you look at the first half of the year in totality, what you see is that we maintain that consistent momentum that we've had since launch.

謝謝，蒂姆。是的，感謝您的提問與認可。我想 - 和你一樣，我對本季的結果非常滿意 - 本季。我的想法是，當你整體審視今年上半年時，你會發現我們保持了自推出以來的一貫動能。

So we've had 10 quarters of growth -- pretty consistent growth. I would take that longer term view as I was looking to the future if I was you. And I'm confident that we will continue that consistent momentum. The underlying dynamics are strong. We continue to get early aligned patients.

所以我們已經實現了 10 個季度的成長——相當穩定的成長。如果我是你，我會採取更長遠的眼光，因為我會展望未來。我相信我們將繼續保持這種一貫的勢頭。潛在的動力強勁。我們繼續獲得早期對齊的患者。

The majority are coming directly from the orals. We're growing both VYVGART and Hytrulo. And we are broadening that prescriber base, which also serves as well for the CIDP launch. So I would say think about consistent momentum and growth as you've seen over the last 10 quarters.

大多數直接來自口頭。我們正在種植 VYVGART 和 Hytrulo。我們正在擴大處方者基礎，這也有助於 CIDP 的啟動。因此，我想說，請考慮過去 10 個季度所看到的持續勢頭和成長。

I'll hand it back to you, Tim.

我會把它還給你，提姆。

Thank you, Karen. And let me briefly comment on the ITP study. First of all, I would like to take a step back and remind the audience about the advanced IP study. But I think we had a very impressive efficacy with an [IVIG] score of more than 50% in a pretty effective patient population and a physician community which was excited also about the very clean safety profile.

謝謝你，凱倫。讓我簡單評論一下ITP研究。首先，我想退一步提醒觀眾有關智慧財產權的高級研究。但我認為我們的療效非常令人印象深刻，在相當有效的患者群體和醫生社群中，[IVIG] 評分超過 50%，他們也對非常乾淨的安全性感到興奮。

And we observed the patients in our open-label extension study. They continue to do actually very well on ITP. And also the Japan launch of ITP is actually going according to plan. So the task we gave to the team was sit down with the FDA go through all the data and propose a smaller confirmatory study in order to push the ITP indication over the finish line. And I think they succeeded in doing that.

我們在開放標籤擴展研究中觀察了患者。事實上，他們在 ITP 方面繼續做得非常好。ITP 在日本的推出實際上也按計劃進行。因此，我們交給團隊的任務是與 FDA 坐下來仔細檢查所有數據，並提出一項較小的驗證性研究，以推動 ITP 適應症越過終點線。我認為他們成功地做到了這一點。

So by leveraging all the insights and the know-how we have on the disease, by actually proposing an alternative primary endpoint, which allows for a smaller confirmatory study, we think we are on track to start a small ITP trial before the end of the year. But I think we will be able to push the product over the finish line. And in that refractory patient population, we have been studying all the way. Thanks for the questions.

因此，透過利用我們對該疾病的所有見解和專業知識，透過實際提出一個替代主要終點（這允許進行更小的驗證性研究），我們認為我們預計在 2019 年結束之前啟動一項小型 ITP 試驗。年。但我認為我們將能夠推動產品衝過終點線。而在那難治性患者族群中，我們一直在研究。感謝您的提問。

(Operator Instructions) Tazeen Ahmed at Bank of America.

（操作員指令）美國銀行的 Tazeen Ahmed。

Hi, good morning, guys. Here's my one question. I just was curious about the PFS filing. I know you've talked about already having -- you know, applied officially. But given that there's no PDUFA, we're just curious about how we should expect to think about communication from your team on what part of the application is in process and what back and forth there might be. And do you have an idea of when you would be able to get that full-on approval, given that doctors in particular seem to be excited about having that as an option for patients? Thanks.

嗨，早上好，夥計們。這是我的一個問題。我只是對 PFS 備案感到好奇。我知道你已經談過——你知道，正式申請。但考慮到沒有 PDUFA，我們只是好奇我們應該如何考慮您的團隊就申請的哪一部分正在處理中以及可能會發生什麼來回進行的溝通。鑑於醫生似乎對將其作為患者的一種選擇感到特別興奮，您是否知道什麼時候才能獲得全面批准？謝謝。

Thanks, Tazeen. And thanks for being with us this morning. Important questions, simple answer, the filing has been submitted, is accepted, and under review with the FDA. And I think we will have a better sense of approval timing when the process unfolds. So stay tuned. We will keep you updated on the progress we make. Thank you.

謝謝，塔津。感謝您今天早上和我們在一起。重要問題，簡單回答，備案已提交、被接受，並正在接受 FDA 審查。我認為，當流程展開時，我們會對批准時機有更好的感覺。所以請繼續關注。我們將隨時向您通報我們所取得的進展。謝謝。

Allison Bratzel, Piper Sandler.

艾莉森‧布拉澤爾，派珀‧桑德勒。

Hey, thank you for taking the question. Maybe just to follow up from the R&D day, could you help us understand just the scope of the data required by FDA, that'll be needed for filing in seronegative and ocular MG patients? You know, I think you described an ability to leverage existing trial data and real-world data, at least for seronegative patients. But I've just been getting a bunch of questions on this. So any more color there would be helpful. Thank you.

嘿，謝謝你提出問題。也許只是為了從研發日進行跟進，您能否幫助我們了解 FDA 所需的數據範圍，這些數據是在血清陰性和眼部 MG 患者中歸檔所需的？你知道，我認為你描述了利用現有試驗數據和現實世界數據的能力，至少對於血清陰性患者而言。但我剛剛收到了很多關於此的問題。所以更多的顏色會有幫助。謝謝。

Yeah, and thank you for the question, Allison. So remember that we printed strong results in seronegative MG patients. And as we already have an approval in Japan, but actually in the real world, the product is doing very well in seronegative patients. And we have impressive case reports out of Europe where people have been successfully using the drug in seronegative patients.

是的，謝謝你的提問，艾莉森。因此請記住，我們在血清陰性 MG 患者中取得了強有力的結果。由於我們已經在日本獲得批准，但實際上在現實世界中，該產品在血清陰性患者中表現良好。我們在歐洲有令人印象深刻的病例報告，人們已經成功地將這種藥物用於血清陰性患者。

So we made a commitment to the patient community, we would not give up. And we have been entertaining the dialogue with the FDA on seronegative patients in the background. I think where we could land with the FDA is, you know -- a pretty fast, elegant study in seronegative patients, which as Luc called it during the R&D day, is somewhat relaxed p-value.

所以我們向患者群體做出了承諾，我們不會放棄。我們一直在與 FDA 就血清陰性患者進行對話。我認為我們可以與 FDA 合作的地方是，你知道——對血清陰性患者進行一項相當快速、優雅的研究，正如 Luc 在研發期間所說的那樣，p 值有些寬鬆。

So I think the FDA is taking the existing evidence into account and is allowing us, you know, to run such a trial. So we think there's a high demand. This is an elegant study. And we will do everything we can to enroll that study as fast as we can. Thanks for the question.

所以我認為 FDA 正在考慮現有的證據，並允許我們進行這樣的試驗。所以我們認為需求很高。這是一項優雅的研究。我們將盡一切努力盡快註冊該研究。謝謝你的提問。

Rajan Sharma, Goldman Sachs.

拉詹·夏爾馬，高盛。

Hi, thanks for taking my question. So just wanted to discuss kind of competitive landscape in myasthenia gravis and how you see VYVGART kind of continuing to differentiate there. There's obviously a busy pipeline. We've got iptacopan in a Phase 3, a [Plinz] is also in a Phase 3.

您好，感謝您提出我的問題。所以我只是想討論一下重症肌無力的競爭格局，以及您如何看待 VYVGART 在那裡繼續保持差異化。顯然有一條繁忙的管道。我們在第 3 階段有 iptacopan，[Plinz] 也在第 3 階段。

So could you just kind of discuss how you think VYVGART differentiates relative to those mechanisms and then longer term, what gives you confidence that VYVGART and the FcRn class more broadly remains kind of the preferred treatment option in early line patients?

那麼您能否討論一下您認為 VYVGART 相對於這些機制有何不同？

Yeah, thank you for the question. And I will first hand over to Karen to comment on how they see, the overall dynamics of this market and how we're building it together. Maybe, Karen, you go first and then I will briefly comment on your differentiation question. Thank you.

是的，謝謝你的提問。我將首先請凱倫評論他們的看法、這個市場的整體動態以及我們如何共同建立它。也許，凱倫，你先說，然後我會簡單評論你的差異化問題。謝謝。

Sounds great. Yeah, thanks for the question. And look, I think building on what we shared at R&D Day last week, we believe that innovation coming to the MG market is good because it grows the impact that we can have for our patients, expands the market, expands the number of patients that are treated with advanced biologics earlier as well.

聽起來很棒。是的，謝謝你的提問。看，我認為基於我們上週在研發日分享的內容，我們相信 MG 市場的創新是好的，因為它增加了我們對患者的影響，擴大了市場，擴大了患者數量也更早地接受先進的生物製劑治療。

So we believe innovation is great for patients and I think we're seeing that. And we believe we're very well positioned to continue to lead in that growing market. And I think there's a few things that maybe you asked about the differentiation that I'll touch on.

因此，我們相信創新對患者來說是件好事，我想我們已經看到了這一點。我們相信，我們處於有利地位，可以繼續在這個不斷成長的市場中保持領先。我想您可能會問一些關於我將談到的差異化的事情。

One is around, you know, we're first in class FcRn and it's clear that FcRn and certainly VYVGART are being used early line. I said earlier, over 50% of our patients are coming directly from the oral. So that's where the growth is and we're positioned well there. I think we compete really well on our, we have rapid efficacy, but also deep and sustained efficacy. And we have it across cyclical and biweekly dosing regimens. So I think that's a really strong position to be competing from.

一個是，你知道，我們是 FcRn 類別中的第一名，很明顯 FcRn 和 VYVGART 正在早期使用。我之前說過，我們有50%以上的病人是直接從口腔來的。這就是成長所在，我們在那裡處於有利地位。我認為我們的競爭非常好，我們有快速的功效，但也有深度和持續的功效。我們有週期性和每兩週一次的給藥方案。所以我認為這是一個非常有利的競爭地位。

And don't forget about our real world safety, the length of that real world safety, the number of patients as the first mover advantage. And of course we have the treatment, low treatment burden. So we have both IV and subcutaneous, we have PFS plants. So overall, I think the dynamics in MG are positive. The market is growing and we're well positioned to maintain and even advance our leadership.

不要忘記我們現實世界的安全性、現實世界安全性的持續時間、作為先發優勢的患者數量。當然我們有治療，治療負擔低。所以我們有靜脈注射和皮下注射，我們有 PFS 植物。總的來說，我認為 MG 的動態是正面的。市場正在成長，我們有能力維持甚至提升我們的領先地位。

I'll hand it back to you, Tim.

我會把它還給你，提姆。

Yeah, thank you, Karen. I much appreciate it. If you just look at the biology of the disease, you know, it is crystal clear that MG is an IgG-mediated disease. Remember the three most effective of these pathogenic antibodies, complement recruitment just being one of them. And that nicely translates into the clinical data, right?

是的，謝謝你，凱倫。我非常感激。如果你只看一下這種疾病的生物學特性，你就會知道，很明顯 MG 是一種 IgG 介導的疾病。請記住這些致病性抗體中最有效的三種，補體募集只是其中之一。這很好地轉化為臨床數據，對嗎？

So let me remind you that 80% of VYVGART patients achieve an ADL lower than five, which is the threshold for entering a clinical trial. And that's 50% to 55% of VYVGART patients achieve minimum symptom expression. So there's always the need of the most effective line for alternative methods of action because the drug only works in 80% of patients, but it's difficult to go into more detail in absence of data. So let's look at the data to then further understand how that biology really plays. Thanks for the question.

因此，讓我提醒您，80% 的 VYVGART 患者的 ADL 低於 5，這是進入臨床試驗的門檻。也就是說，50% 到 55% 的 VYVGART 患者的症狀表現達到最低。因此，始終需要最有效的替代療法，因為該藥物僅對 80% 的患者有效，但在缺乏數據的情況下很難進行更詳細的研究。因此，讓我們來看看數據，然後進一步了解生物學的真正作用。謝謝你的提問。

Derek Archila, Wells Fargo.

德里克·阿奇拉，富國銀行。

Hey, good morning and congrats on the progress. Thanks for taking the question. So just -- can you discuss maybe how the time from payer approval to injection that you're currently seeing during the early part of the CIDP launch compares to what you saw in the early part of the MG launch? Is that similar or is it different and maybe a little color on why?

嘿，早上好，祝賀你取得的進展。感謝您提出問題。那麼，您能否討論一下您目前在 CIDP 啟動早期看到的從付款人批准到注入的時間與您在 MG 啟動早期看到的時間相比如何？這是相似的還是不同的，也許有一點顏色解釋為什麼？

Karen Massey, would you mind taking this question, please?

Karen Massey，你介意回答這個問題嗎？

Yeah, happy to take the question. Thanks for it. First of all, just to start with saying we're really pleased with the strong and positive response we're having in CIDP. And to answer your specific question, as enrollments come in, it does take a while for patients to get approved by their payers. And it's that period of time that can take a few weeks before they get injected. And that's standard for any launch.

是的，很高興回答這個問題。謝謝你。首先，首先要說的是，我們對 CIDP 得到的強烈而積極的回應感到非常高興。為了回答你的具體問題，隨著註冊人數的增加，患者確實需要一段時間才能獲得付款人的批准。這段時間可能需要幾週的時間才能注射。這是任何發布的標準。

It's the reason that we're so focused on getting payer policies in place because once the payer policy is in place, that process from script to injection can go more quickly. I would say we're exactly on track with where we thought we would be, both in terms of that process, as well as most importantly, the discussions that we're having with payers around getting those policies in place. We're really pleased with the progress and the team's acting with urgency. And I think we're right on track and pleased with where we're at.

這就是我們如此專注於讓付款人政策到位的原因，因為一旦付款人政策到位，從腳本到注入的過程就會更快。我想說，無論是在流程方面，還是最重要的是，我們與付款人圍繞落實這些政策進行的討論方面，我們都完全符合我們的預期。我們對進度和團隊的緊急行動感到非常滿意。我認為我們正走在正確的軌道上，並且對我們所處的位置感到滿意。

I'll hand it back to you, Tim.

我會把它還給你，提姆。

Yeah, thank you, Karen. We would like to ask a little bit of patience, Derek, because remember what we did for MG rights. I mean, it took us two quarters to install broad and favorable policies. That was our commitment to the patient community. And that is fast. Guys, I mean, two quarters is really outstanding, an outstanding job done by the team. And we will seek to replicate that for CIDP. Thank you.

是的，謝謝你，凱倫。德里克，我們想請您耐心等待，因為請記住我們為 MG 權利所做的一切。我的意思是，我們花了兩個季度的時間才制定了廣泛而有利的政策。這是我們對患者社群的承諾。而且速度很快。夥計們，我的意思是，兩個季度真的非常出色，團隊完成了出色的工作。我們將尋求為 CIDP 複製這一點。謝謝。

Akash Tewari, Jefferies.

阿卡什·特瓦里，杰弗里斯。

Hey, thanks so much. So kind of on that point, on CIDP, you mentioned in the past, clearly seeing strong demand from doctors, but you wanted to see how that actually translated to patient and payer demand, given the label, the overlap in the prescriber base and the amount of IVIG experienced patients here.

嘿，非常感謝。在這一點上，在 CIDP 上，您過去提到過，清楚地看到醫生的強烈需求，但考慮到標籤、處方者基礎的重疊和這裡有大量有 IVIG 經驗的患者。

Would it be fair to say, the CIDP launch should at least be half as good as gMG out to the first year? And maybe to that point, do you think we will see this kind of inflection after two quarters with CIDP, like we saw with gMG? I just wanted to double click on that point, like would say. Thanks so much.

公平地說，CIDP 的推出應該至少是第一年 gMG 的一半嗎？也許到那時，您認為 CIDP 兩個季度後我們會看到這種變化，就像我們在 gMG 看到的那樣？我只是想雙擊那個點，就像會說的。非常感謝。

Yeah, Akash, and thank you for the question. Thanks for being with us today. It is tempting, right, to draw analogies between an MG launch and a CIDP launch. But what we're trying to say and explain is that, you know, they're two distinctly different markets with their own dynamics, launch dynamics. And maybe, Karen, you want to dig a bit deeper into this question, right?

是的，阿卡什，謝謝你的提問。感謝您今天和我們在一起。人們很容易將 MG 發射和 CIDP 發射進行類比。但我們想說解釋的是，你知道，它們是兩個截然不同的市場，有自己的動態、發布動態。也許，凱倫，你想更深入地研究這個問題，對嗎？

Yeah, I think that's right. It's very hard to draw parallels. They each have their own dynamics. One thing that I would say around the inflection point that you mentioned after two quarters, I wouldn't think about an inflection point that the payer policies will come in sort of one by one over the two quarters. And what we like to think about is that sort of -- by the time you get to the end of two quarters, you might have sort of that critical mass where neurologists really can start to get confident that favorable payer policies are in place.

是的，我認為這是對的。很難進行類比。他們每個人都有自己的動力。關於您提到的兩個季度後的拐點，我要說的一件事是，我不會想到付款人政策將在兩個季度中一一出現的拐點。我們喜歡考慮的是，當你到兩個季度末時，你可能已經達到了臨界點，神經學家真的可以開始對有利的付款人政策到位充滿信心。

So we do believe that there will be uptake, the same as we saw in MG, and we think that it'll be consistent over the period of time. But the dynamics between the two launches are very different for the reasons that you pointed out.

因此，我們確實相信將會有吸收，就像我們在 MG 中看到的那樣，我們認為在一段時間內它會保持一致。但由於您指出的原因，兩次發布之間的動態非常不同。

Alex Thompson, Stiefel.

亞歷克斯湯普森，斯蒂菲爾。

Hey, great. Thanks for taking my question. I guess I wanted to ask about OpEx trajectory over the next couple of years, how you're thinking about that as it relates to expansion into the additional Phase 3 programs, as well as thinking about commercial expansion beyond neurology. Thanks.

嘿，太棒了。感謝您提出我的問題。我想我想問一下未來幾年的營運支出軌跡，您如何看待這個問題，因為它涉及擴展到其他第三階段項目，以及考慮神經病學以外的商業擴張。謝謝。

Thank you, Alex. I'll take that question. In terms of SG&A, our infrastructure is now largely in place. As you would remember, we put that expansion in the US in Q1. For the rest of the world, we have infrastructure in most of the markets. There's a few big markets still outstanding in Europe. I would expect SG&A from here on forward to grow, but if it grows, we'll be muted and you won't see that rapid expansion.

謝謝你，亞歷克斯。我來回答這個問題。就SG&A 而言，我們的基礎設施現已基本到位。正如您所記得的，我們將在美國的擴張放在第一季。對於世界其他地區，我們在大多數市場都擁有基礎設施。歐洲有幾個大市場仍然表現出色。我預計SG＆A從現在開始會成長，但如果它成長，我們將保持沉默，你不會看到快速擴張。

In terms of R&D, again, I think that will grow quarter-over-quarter as we invest in all the new science, which we talked about last week. I think we have a unique opportunity to invest in ourselves here to set us up for a long, sustainable future. That's what we're going to do. Thank you for your question, Alex.

在研發方面，我認為，隨著我們對所有新科學的投資，研發將逐季成長，我們上週談到了這一點。我認為我們在這裡有一個獨特的機會進行自我投資，為我們創造一個長期、可持續的未來。這就是我們要做的。謝謝你的提問，亞歷克斯。

Thomas Smith, Leerink Partners.

托馬斯史密斯，Leerink 合夥人。

Hey, guys. Good morning. Thanks for taking the questions and congrats on the strong results. For VYVGART in MG, can you just remind us on the data that's being generated that could support chronic dosing in addition to the current cycle-based dosing, whether you expect to get chronic dosing explicitly added into the label, or how important do you think it is for prescribers to have that chronic dosing flexibility in the label to facilitate access and reimbursement? Thanks.

嘿，夥計們。早安.感謝您提出問題並祝賀我們取得了優異的成績。對於重症肌無力的VYVGART，您能否提醒我們正在產生的數據，除了目前基於週期的給藥之外，還可以支持長期給藥，您是否希望將慢性給藥明確添加到標籤中，或者您認為有多重要處方者是否應該在標籤上具有長期劑量的彈性，以方便取得和報銷？謝謝。

Thank you for the question. The answer is simple. We have chronic dosing in the label. The label is basically describing the use of VYVGART for the treatment of statistically reseparated antibody positive gMG patients. So we have cyclical dosing, but that is, of course, chronic use. We're the only company which has already such a long timeline of chronic dosing of patients.

謝謝你的提問。答案很簡單。我們的標籤上有慢性劑量。該標籤基本上描述了 VYVGART 用於治療統計上重新分離的抗體陽性 gMG 患者的用途。所以我們有週期性給藥，但這當然是長期使用。我們是唯一一家擁有如此長的患者長期用藥時間表的公司。

I think it is important to call out that the safety profile of the drug maintains consistence, and that we see a consistent minimum symptom expression over multiple years now in 50% to 55% of the patients. What we did do is we ran an [ADEPT-Next] study, which was filling a data gap for those patients which, for example, are coming from chronic plasma exchange or chronic IVIG, patients which we really did not study in the ADEPT trial.

我認為重要的是要指出該藥物的安全性保持一致，而且多年來我們在 50% 至 55% 的患者中看到了一致的最低症狀表達。我們所做的是進行了一項 [ADEPT-Next] 研究，該研究填補了那些來自慢性血漿置換或慢性 IVIG 的患者的數據空白，而我們在 ADEPT 試驗中確實沒有研究過這些患者。

Because these patients cannot stand, of course, cyclical dosing, if you're on a weekly plasma exchange, you will need real intense chronic therapy. In the ADEPT- Next study, we have actually shown that every other week dosing with VYVGART is as powerful as the cyclical dosing, both from an efficacy and a safety point of view. In summary, I think we're the only company really with chronic dosing data, and we're the only company which can offer such a diverse set of dosing schedules. Thanks for the question.

當然，因為這些患者無法忍受週期性給藥，所以如果您每週進行一次血漿置換，您將需要真正的強化慢性治療。在 ADEPT-Next 研究中，我們實際上已經證明，從療效和安全性的角度來看，每隔一周服用 VYVGART 與週期性給藥一樣有效。總而言之，我認為我們是唯一一家真正擁有長期給藥數據的公司，也是唯一一家能夠提供如此多樣化的給藥方案的公司。謝謝你的提問。

Yaron Werber, TD Cowen.

亞龍·韋伯，TD·考恩。

Great. Thanks so much, and a really nice showing team. Quick question. Just in Europe and Japan, -- Europe is very tough these days, and Japan usually has got lumpiness, as you noted, and obviously changes in pricing. But just any sense kind of what should we expect there in terms of acceleration of sales? Thank you.

偉大的。非常感謝，還有一個非常好的表演團隊。快問。就在歐洲和日本，歐洲現在非常艱難，而日本通常會出現不穩定的情況，正如您所指出的，而且定價也發生了明顯的變化。但就銷售加速而言，我們該期待什麼？謝謝。

Yaron, that is a great question, and thank you for zooming out on the global aspirations of the company. This is a question I would like to hand over to Karen.

Yaron，這是一個很好的問題，感謝您關注公司的全球願景。這個問題我想交給Karen。

Yeah, happy to take this, and thanks for the question. So maybe I'll break them out and talk about Europe first and then Japan. I would say in Europe, we're on track. As you know, it takes a little longer, as you already called out, in Europe to get pricing and reimbursement in place across all of the different countries. We're pleased with the that we've made, and certainly in three of the big five markets, Germany, Spain, and Italy, we have good reimbursement, we have strong clinical advocacy, and we're really seeing that consistent uptake with what we're seeing in the US.

是的，很高興接受這個，謝謝你的提問。所以也許我會先討論歐洲，然後再討論日本。我想說，在歐洲，我們正步入正軌。如您所知，正如您已經指出的那樣，在歐洲，在所有不同國家/地區確定定價和報銷需要更長的時間。我們對我們所做的事情感到滿意，當然在五個大市場中的三個，德國、西班牙和意大利，我們有良好的報銷，我們有強有力的臨床宣傳，我們確實看到了一致的採用我們在美國看到的情況。

And what I mean by that is that we see a broader prescriber base, not just in the academic centers, broader potential, and really pushing towards early aligned use. So I think we're seeing consistent trends there, and as we open up new markets with pricing and reimbursement, then I think you'll start to see that consistent growth as well.

我的意思是，我們看到了更廣泛的處方者基礎，而不僅僅是在學術中心，更廣泛的潛力，並且真正推動早期一致使用。因此，我認為我們在那裡看到了一致的趨勢，當我們透過定價和報銷開闢新市場時，我認為您也會開始看到這種一致的成長。

So we're pleased where we are in Europe, we're on track, we take it step by step, it takes a little bit longer. In Japan, I just want to applaud the team, I think they've done a phenomenal job. I mean, if you look back quarter-over-quarter, it's just so incredibly consistent, the growth in Japan, and we see that again this quarter.

所以我們很高興我們在歐洲所處的位置，我們走上了正軌，我們一步一步地走下去，這需要更長的時間。在日本，我只想為團隊鼓掌，我認為他們做得非常出色。我的意思是，如果你回顧每個季度，日本的成長是如此令人難以置信的一致，我們在本季再次看到了這一點。

I would imagine -- you can imagine that outlook being consistent moving forward. We just recently launched Hytrulo, which is the name for Subcutaneous in Japan, and that, like in other markets, we're seeing that really expand the patient population that is open to VYVGART, with those, again, opening up those earlier lines of treatment. So again, I would say consistent growth, as you've seen for the last 10 quarters from Japan, that we can expect in the future. Thanks for the question.

我可以想像——你可以想像這種前景會持續向前發展。我們最近剛推出了 Hytrulo，它是日本皮下注射的名稱，與其他市場一樣，我們看到它確實擴大了接受 VYVGART 治療的患者群體，再次開放了早期的系列產品治療。因此，我想說，正如您在過去 10 個季度中看到的那樣，我們可以在未來看到日本的持續成長。謝謝你的提問。

Matt Phipps, William Blair.

馬特·菲普斯，威廉·布萊爾。

Hi, thanks for taking my questions, congrats on all the progress. Quickly, you mentioned a different endpoint in the next ITP trial, is that just looking at a different time frame for sustained plate response, or can you use something like IVIG responders as a primary endpoint?

您好，感謝您提出我的問題，恭喜所有的進展。很快，您在下一個 ITP 試驗中提到了一個不同的終點，只是觀察持續板反應的不同時間範圍，還是可以使用 IVIG 反應者等作為主要終點？

And maybe just quickly on the launch of ITP in Japan, any sense yet on where VYVGART is being used in the treatment paradigm for ITP patients? Is it after multiple thrombopoietin agonists, or can you slot earlier in that treatment paradigm? Thanks.

也許就在 ITP 在日本推出之際，VYVGART 在 ITP 患者治療模式中的應用有意義嗎？是在多種血小板生成素激動劑之後進行，還是可以在該治療模式中提前安排？謝謝。

Two great questions. Thank you for that. I will hand over question two to Karen. On the ITP trial, I think the reason why we can go with a significantly smaller study in a confirmatory mindset is A, because we can work with an endpoint, which is looking for extent of disease control. And B, we can really leave this to know how the expertise we have developed in running ITP clinical trials.

兩個很好的問題。謝謝你。我將把第二個問題交給凱倫。在 ITP 試驗中，我認為我們可以以驗證性心態進行規模小得多的研究的原因是 A，因為我們可以使用一個終點，即尋找疾病控制的程度。B，我們真的可以透過這個來了解我們在運行 ITP 臨床試驗中所累積的專業知識。

So I think this is going to be, roughly speaking, a trial, let them have the size of the global Phase 3 trials, which we have done so far. And we think it's a responsible investment to make for a significant patient and the need waiting on the other line.

所以我認為，粗略地說，這將是一次試驗，讓他們擁有我們迄今為止所做的全球第三階段試驗的規模。我們認為，對於重要的病人和在另一條線上等待的需要來說，這是一項負責任的投資。

Karen, would you mind discussing the Japan question, please?

凱倫，你介意討論一下日本問題嗎？

Yeah, happy to take that. I would start by saying the ITP launch in Japan is going really well. We've applied sort of the same launch label that we have in other launches, and we're seeing the same strong early performance. And what I would say is that it's clear that there's an unmet need in these ITP patients.

是的，很高興接受。首先我要說的是，ITP 在日本的推出進展非常順利。我們已經應用了與其他發布中相同的發布標籤，並且我們看到了同樣強勁的早期表現。我想說的是，很明顯，這些 ITP 患者的需求尚未被滿足。

So far, what we're seeing, to your specific question, is that the early experience is in later lines of therapy. And that's exactly what you would expect in any of these launches. The doctors want to try to get experience in those refractory patients, in those later line patients, get experience under their belt, and then start to move earlier lines.

到目前為止，對於你的具體問題，我們看到的是早期的經驗存在於後期的治療中。這正是您在這些發布中所期望的。醫生們希望嘗試在那些難治性患者、那些較晚接受治療的患者身上獲得經驗，然後開始轉移較早的治療線。

But what we're seeing that I'm really pleased with is that there's a strong belief in the MOA, in the mechanism of action of FcRN. The neurologists -- hematologists are responding that they believe in the MOA, and therefore that they believe in the reason to try this.

但令我真正滿意的是，我們看到人們對 MOA、對 FcRN 的作用機制抱持堅定的信念。神經學家和血液學家回應說，他們相信 MOA，因此他們相信嘗試這個的理由。

So I would say strong early results, exactly where we would expect them, and really important learnings that we're going to be able to take away from this Japan launch as we think about the US and potentially other launches with the second study that we're planning. Thanks for the question.

因此，我想說的是，早期結果非常強勁，正是我們所期望的，當我們考慮美國和其他可能的發射時，我們將能夠從日本的這次發射中獲得非常重要的經驗教訓。 。謝謝你的提問。

Suzanne van Voorthuizen, Kempen & Co.

Suzanne van Voorthuizen，肯彭公司

Hi, team. This is Suzanne from Kempen. Thanks for taking my question. I just have a small follow-up to last week's R&D day regarding ARGX-121, the IgA degrader that was revealed. Can you give some context to the indications where such molecule would fit well, how many indications you see, and also how you compare the opportunity in terms of size? Is there potential for this to be an opportunity of fifth-card size, or do you see more parallels to a drug like empa when looking at the commercial opportunity? Any thoughts or direction here would be appreciated. Thanks.

大家好。這是來自肯彭的蘇珊娜。感謝您提出我的問題。我只是對上週的研發日進行了一個關於 ARGX-121 的小型後續活動，ARGX-121 是已發布的 IgA 降解劑。您能否提供一些背景資訊來說明這種分子最適合的適應症，您看到了多少適應症，以及您如何比較機會的大小？這是否有可能成為第五張牌大小的機會，或者在考慮商業機會時，您是否發現與 empa 等藥物有更多相似之處？這裡的任何想法或方向將不勝感激。謝謝。

Thanks, Suzanne. Thank you for this question. You know that when we take a product forward in the pipeline that it has got the right to play across multiple indications, so it's not a single indication asset. I think what we said on the podium during the R&D day is that there's a growing understanding of the pathogenic role IgA autoantibodies play in autoimmune disease.

謝謝，蘇珊娜。謝謝你提出這個問題。您知道，當我們在管道中推進產品時，它有權跨多種適應症發揮作用，因此它不是單一適應症資產。我認為我們在研發日期間在講台上所說的是，人們對 IgA 自體抗體在自體免疫疾病中的致病作用有了越來越多的了解。

It's actually remarkable how little we know above and beyond pathogenic IgGs. So this is a field which is completely emerging. We're very pleased that we can go in and find it. That's the work we like to do. And then in terms of indications, we give you a conceptual list of indications where we know that IgA autoantibodies drive disease.

值得注意的是，我們對致病性 IgG 以外的知識知之甚少。所以這是一個完全新興的領域。我們很高興能夠進去找到它。這就是我們喜歡做的工作。然後就適應症而言，我們為您提供了一份概念性的適應症列表，我們知道 IgA 自體抗體會導致疾病。

Of course, everyone knows about IgA nephropathy. I think that is a field which is just being built. I think it's a large market opportunity which will support multiple generations of innovation and multiple innovative molecules. So that's an obvious one. We spoke about IgA vasculitis, but again from a biology point of view, that is in the bullseye of the disease.

當然，大家都知道IgA腎病。我認為這是一個剛剛建立的領域。我認為這是一個巨大的市場機會，將支持多代創新和多種創新分子。這是顯而易見的。我們談到了 IgA 血管炎，但同樣從生物學的角度來看，這是疾病的核心。

There are more indications. And at this moment, we will stay conceptual because we're still doing some background work on these indications. And the opportunity will unfold when we, bring this molecule online in all these different clinical trials. So stay tuned. We think it's a significant opportunity where I think we can follow the biology. So we're very excited about the molecule. Thank you

還有更多跡象。目前，我們將保持概念性，因為我們仍在對這些跡象進行一些背景工作。當我們將這種分子引入所有這些不同的臨床試驗時，機會就會出現。所以請繼續關注。我們認為這是一個重要的機會，我認為我們可以追蹤生物學。所以我們對這個分子非常興奮。謝謝

Yatin Suneja, Guggenheim Partners.

蘇內賈 (Yatin Suneja)，古根漢合夥人事務所。

Hey, guys. Congrats on the quarter. Two very quick ones for me. Could you just talk a little bit more about the subcu and IV dynamic? Like how is subcu, what sort of share subcu has right now that's sort of growing the market? And then just as we think about future with the launch of CIDP, any thoughts on establishing guidance for us? Thank you.

嘿，夥計們。恭喜本季。對我來說兩個非常快的。能多談談 subcu 和 IV 的動態嗎？例如 subcu 怎麼樣，subcu 目前擁有什麼樣的份額，可以推動市場的成長？然後，當我們隨著 CIDP 的啟動而思考未來時，有什麼想法可以為我們建立指導嗎？謝謝。

Thank you for both questions, Yatin. Let's start with question one, the subcu versus IV dynamic. Karen, would you mind commenting on the dynamic? I don't think we quantify, right? But maybe you can explain the dynamic.

謝謝你提出的兩個問題，Yatin。讓我們從第一個問題開始，subcu 與 IV 的動態。凱倫，你介意評論一下動態嗎？我認為我們沒有量化，對吧？但也許你可以解釋一下動態。

Yeah, happy to -- exactly. So we don't provide the specific breakdown. But what I would say that I think is helpful in terms of the dynamic, both VYVGART and Hytrulo are growing in terms of new patient starts and in terms of revenue. And what we see and what the real value of Hytrulo is, is that I would say it opens really up new prescribers that might not be, for whatever reason, interested or comfortable with an IV option for new urologists that are more comfortable with injections.

是的，很高興——確切地說。所以我們不提供具體的細分。但我想說的是，我認為在動態方面是有幫助的，VYVGART 和 Hytrulo 在新患者啟動和收入方面都在成長。我們所看到的以及Hytrulo 的真正價值在於，我想說它確實為新的處方者開闢了道路，這些處方者可能出於某種原因對靜脈注射選項不感興趣或不適應，而對於新的泌尿科醫生來說，他們更喜歡注射。

And that also opens up new patients that are seeing those urologists, or potentially there are patients that don't want to go have the IV. Maybe they think that their disease is not severe enough, that they need an IV option, but they're open to an injection option.

這也為那些正在看泌尿科醫生的新患者提供了機會，或者可能有一些患者不想接受靜脈注射。也許他們認為自己的疾病還不夠嚴重，需要靜脈注射，但他們願意接受注射。

So I would say by having subcutaneous, it aligns directly with our strategy of moving into early aligned treatment. And we see that very clearly. And it also aligns with our strategy of broadening the prescriber base for VYVGART, which obviously also helps us with CIDP.

所以我想說，透過皮下注射，它與我們進入早期聯合治療的策略直接一致。我們看得很清楚。這也符合我們擴大 VYVGART 處方者基礎的策略，這顯然也有助於我們的 CIDP。

The other important dynamic that I think I just want to highlight on Hytrulo is that our goal and our strategy is not a switch strategy from VYVGART. So around 60% of the Hytrulo patients that are starting on Hytrulo are actually new to the VYVGART franchise. And that just demonstrates how we're really expanding the market, both in terms of prescribers and patients with the subcutaneous option.

我想在 Hytrulo 上強調的另一個重要動態是，我們的目標和我們的策略不是 VYVGART 的切換策略。因此，大約 60% 開始使用 Hytrulo 的 Hytrulo 患者實際上是 VYVGART 特許經營的新手。這恰恰證明了我們如何真正擴大市場，無論是在處方者還是皮下注射患者方面。

And I'll hand it back to you, Tim.

我會把它還給你，提姆。

Actually, we had a cut in our line. If Yatin is still on, if you could just repeat his second question, that would be helpful.

事實上，我們的隊伍被削減了。如果亞廷還在的話，如果你能重複他的第二個問題，那會很有幫助。

Yeah, I'm online. So the second question was about guidance. How are you guys thinking about establishing guidance? And when should we expect that?

是的，我在線。所以第二個問題是關於指導。你們如何考慮建立指導？我們什麼時候該期待這一點？

Okay, that is good. Thank you, Yatin, for repeating it. This is a question for Karl, right?

好吧，那很好。謝謝亞廷的重複。這是卡爾的問題，對嗎？

Yeah, thank you. Thank you, Yatin. I mean, we didn't provide guidance this year, revenue guidance, due to all the unknowns out there, and particularly for the CIDP launch and the geographical expansion. So I think as a company matures, clearly we need to think about guidance. So we will listen to our stakeholders, including our investors and analysts. And this is something which we will consider. Maybe this is something we can do in January next year. But for now, we're going to focus on executing. And we have provided the expense guidance and we'll leave it at that for now.

是的，謝謝。謝謝你，亞汀。我的意思是，由於存在所有未知因素，特別是 CIDP 的啟動和地理擴張，我們今年沒有提供收入指導。所以我認為隨著公司的成熟，我們顯然需要考慮指導。因此，我們將聽取利害關係人的意見，包括投資者和分析師的意見。這是我們會考慮的事情。也許這是我們明年一月可以做的事情。但現在，我們將專注於執行。我們已經提供了費用指導，但現在就這樣吧。

Vikram Purohit, Morgan Stanley.

維克拉姆‧普羅希特，摩根士丹利。

Hi, good morning. Thank you for taking our question. We had one on the commercial opportunity in Ocular MG. So based on the neurologist feedback you received and just the experience you have in the space with this indication overall at this point, how distinctly do you think Ocular MG is managed and viewed and treated versus more generalized MG? And based on that, how drastically do you think the cadence of patient ads could change based on a potential label expansion into Ocular MG? Thanks.

嗨，早安。感謝您提出我們的問題。我們有一個關於 Oural MG 的商業機會。因此，根據您收到的神經科醫生的回饋以及您目前在該適應症領域的經驗，您認為眼部重症肌無力的管理、觀察和治療與更廣泛的重症肌無力有何不同？基於此，您認為患者廣告的節奏會因潛在的標籤擴展到 Oural MG 而發生多大的變化？謝謝。

Thanks for the question on Ocular MG. So by having been now in the MG space for some time, we have been hearing more about the unmet medical needs in Ocular MG. And it could be a wrong assumption that Ocular MG is a milder form of MG compared to the generalized form of myasthenia.

感謝您提出有關 Oural MG 的問題。因此，進入 MG 領域一段時間以來，我們已經聽到更多有關眼部 MG 未滿足的醫療需求的資訊。與全身性肌無力相比，眼部重症肌無力是一種較溫和的重症肌無力，這種假設可能是錯誤的。

So OcularMG patients are pretty debilitated and disabled because with double vision, there's not too much activities of daily living you can do in terms of working on screens, driving, and then I'm not even talking about the headaches these people experience.

因此，OuralMG 患者非常虛弱和殘疾，因為患有復視，您無法進行太多的日常生活活動，例如在螢幕上工作、駕駛，然後我什至不談論這些人經歷的頭痛。

Today, Ocular MG patients are basically only treated in steroids. And some of them actually do very well on steroids, but there's a subset of Ocular MG patients which badly need another tool in the toolbox. And after close consultation with the community and the experts, we crystallized -- I think, a pretty elegant Ocular MG study where we had a successful interaction with the FDA, agreeing on trial design and endpoints.

如今，眼部重症肌無力患者基本上只接受類固醇治療。其中一些實際上在使用類固醇後效果很好，但有一部分眼部重症肌無力患者迫切需要工具箱中的另一種工具。在與社區和專家密切協商後，我們具體化了——我認為，這是一項非常優雅的眼部 MG 研究，我們與 FDA 進行了成功的互動，就試驗設計和終點達成了一致。

And in the real world, some of these Ocular MG patients are actually seen by ophthalmologists or neuro-ophthalmologists. And then when the symptoms start to spread, they're being referred to a neuromuscular specialist. So I think this is a significant opportunity and a nice addition if we are successful to our presence in the MG space. Thanks for the question.

在現實世界中，一些眼部重症肌無力患者實際上是由眼科醫生或神經眼科醫生看診的。然後，當症狀開始蔓延時，他們就會被轉介給神經肌肉專家。因此，我認為，如果我們在 MG 領域取得成功，這是一個重要的機會，也是一個很好的補充。謝謝你的提問。

Charles Pitman-King, Barclays.

查爾斯·皮特曼-金，巴克萊銀行。

Hi, thank you very much for taking my questions. Two for me, just coming back to the potential risks from competitive readouts to, your kind of dominant position in the second half this year. I'm just wondering what metrics will you be looking at from these readouts to determine whether any of these really pose a risk to your increasing market dominance?

您好，非常感謝您回答我的問題。對我來說有兩點，只是回到今年下半年競爭性數據對你的主導地位的潛在風險。我只是想知道您將從這些讀數中查看哪些指標，以確定其中是否真的對您日益增長的市場主導地位構成風險？

And then just the second one on the kind of Chinese commercial opportunity. I mean, I understand Zai labs is not going to be a report until the August 20, but just in terms of how you are thinking about this from an organics perspective? How established are the treatments and what is the expected addressable market and pricing structure that you expect to benefit from going forward? Thanks.

然後是第二個關於中國商業機會的問題。我的意思是，我知道 Zai 實驗室要到 8 月 20 日才會發布報告，但您是如何從有機角度思考這個問題的？這些治療方法的成熟度如何？謝謝。

Thank you for both questions. Karen, I will give the second question to you. I think on the first question, we can be relatively brief. In absence of data, I think we would find ourselves in an area of speculation, which we do not really like to do as a science-based company. The only thing I can do is remind you of how high we have set the bar in the MG space.

謝謝你的兩個問題。凱倫，我將向你提出第二個問題。我認為對於第一個問題，我們可以說得比較簡短。如果沒有數據，我認為我們會發現自己處於一個猜測的領域，作為一家以科學為基礎的公司，我們並不喜歡這樣做。我唯一能做的就是提醒您我們在 MG 領域設定的標準有多高。

I think we have the fastest onset of action. We have the deepest action. We have a very nice durability now over many, many years in our MG patients. And importantly, I think we have a very clean safety profile. And then from a product presentation point of view, I think we have by far the broadest portfolio of product presentations, which we will continue to aggressively expand. So that is it for the data, but I think we are well equipped to compete.

我認為我們的行動是最快的。我們有最深入的行動。我們的重症肌無力患者在許多年裡都具有非常好的耐用性。重要的是，我認為我們擁有非常乾淨的安全狀況。然後從產品展示的角度來看，我認為我們擁有迄今為止最廣泛的產品展示組合，我們將繼續積極擴展。這就是數據，但我認為我們已經做好了競爭的準備。

And Karen, do you mind taking on question two?

凱倫，你介意回答第二個問題嗎？

No, happy to take that question, Tim. And look, I will not comment on Q2 results. Of course, we will let Zai lab comment on that. But what I will say is a few thoughts on -- first of all, starting with the fact that I think Zai are incredible partners, and they have done a great job with the launch of MG.

不，很高興回答這個問題，提姆。看，我不會對第二季的結果發表評論。當然，我們會讓Zai實驗室對此發表評論。但我要說的是一些想法——首先，我認為 Zai 是令人難以置信的合作夥伴，他們在 MG 的推出方面做得非常出色。

And we see -- with them a long-term partnership and I think a lot of opportunity in the future in China. Obviously, it is a big market. And what we have seen to date is a lot of volume coming in early on through Q1. You saw a big volume of new patients in Q1. And I think that reflects the market opportunity in China. I think we have done a great job of not just getting the approvals, but also the NRDL listing. That has certainly helped with uptake. We expect that to continue.

我們看到了與他們的長期合作關係，我認為未來在中國有很多機會。顯然，這是一個很大的市場。到目前為止，我們看到的是第一季初期的大量成交量。第一季出現了大量新患者。我認為這反映了中國的市場機會。我認為我們不僅在獲得批准方面做得很好，而且在國家醫療保健目錄上也做得很好。這無疑有助於提高採用率。我們預計這種情況會持續下去。

And more recently, we got the subcutaneous version approved in China. So I think overall, between the volume of patients with MG in China alongside the great partner we have in Zai lab, I think we are looking positively at the outlook for China.

最近，我們在中國獲得了皮下注射版本的批准。因此，我認為總體而言，鑑於中國的重症肌無力患者數量以及我們在再鼎實驗室的優秀合作夥伴，我認為我們對中國的前景持積極態度。

I will hand back to you, Tim.

我會把東西還給你，提姆。

Thanks, Karen. Thanks for the question.

謝謝，凱倫。謝謝你的提問。

Leland Gershell, Oppenheimer.

利蘭·格謝爾，奧本海默。

Great. Thanks for taking our questions. I just want to ask, Karen, you have been consistent in moderating expectations for securing payer agreements for CIDP. I just want to ask if that process has been going in line with your expectations internally. And I also want to ask if you have been facing any pushback from payers with respect to requiring a step-through from IVIG. Thank you.

偉大的。感謝您回答我們的問題。我只是想問，凱倫，您一直在降低對 CIDP 付款人協議的期望。我只是想問一下這個過程是否符合您內在的期望。我還想問一下，您在要求 IVIG 進行逐步處理方面是否面臨付款人的任何阻力。謝謝。

I think this is an excellent question.

我認為這是一個很好的問題。

I'm happy to take that. (multiple speakers) Yeah, sounds great. It's a great question. And we talked earlier about why it's so important to get these payer policies in place. I would say that -- as I said before, we're exactly on track with where we thought we would be. And I mean that in terms of both how the conversations with payers are progressing, which are progressing well, and also the conversations that we're having with payers in terms of what those policies should look like.

我很樂意接受。（多個揚聲器）是的，聽起來很棒。這是一個很好的問題。我們之前討論過為什麼制定這些付款人政策如此重要。我想說的是——正如我之前所說，我們完全按照我們的預期走上了正軌。我的意思是，與付款人的對話進展如何，進展順利，以及我們與付款人就這些政策應該是什麼樣子進行的對話。

In terms of IVIG, I think one thing that's important to remember about the CIDP market is that the majority of patients have been exposed to IVIG at some point. And we know from our clinical trial that we have equal right to respond, whether it's IVIG as background therapy coming off no treatment in the last six months or steroids.

就 IVIG 而言，我認為關於 CIDP 市場需要記住的一件重要事情是，大多數患者都曾在某個時候接觸過 IVIG。我們從臨床試驗中知道，無論是過去六個月未接受治療的 IVIG 作為背景治療還是類固醇，我們都有同等的反應權利。

So we think we're well-positioned with our data. And we think the payer discussions are going well to get policies in place that will set us up for a successful and a strong launch over the coming quarters. Anything to add, Tim? Thank you for the question. We're ready for the next one.

所以我們認為我們的數據處於有利地位。我們認為，付款人討論進展順利，政策到位，這將為我們在未來幾季的成功和強勁推出奠定基礎。有什麼要補充的嗎，提姆？謝謝你的提問。我們已經準備好迎接下一場了。

Samantha Semenkow, Citi.

薩曼莎·塞門科，花旗銀行。

Hi, good morning. And thanks for taking the question. Just one on the PFS for me. Now that you've filed and you're preparing for a potential launch of the PFS, I'm curious, how much more growth in gMG are you really thinking about that that formulation will drive? What patient segments do you expect to open or expand? Do you think this will be more of a switch market than what you've seen with Hytrulo? And just how should we think about the magnitude of impact on revenue, assuming you have that approval in the near future? Thank you.

嗨，早安。感謝您提出問題。就我而言，PFS 上只有一份。既然您已經提交了申請，並且正在為 PFS 的潛在推出做準備，我很好奇，您真正認為該配方將推動 gMG 的增長多少？您期望開放或擴大哪些患者細分市場？您認為這將是一個比您在 Hytrulo 上看到的更多的開關市場嗎？假設您在不久的將來獲得批准，我們應該如何考慮對收入的影響程度？謝謝。

Yeah, thank you. That's a great question on PFS. And Karen, I think, why don't you explain why this will just continue that steady, strong trajectory into the gMG population, please?

是的，謝謝。這是關於 PFS 的一個很好的問題。凱倫，我想，為什麼不解釋為什麼這會繼續以穩定、強勁的軌跡進入 gMG 人群呢？

Yes, I think that's exactly it, Tim, and what I was going to say. I would say this just continues to expand and reinforce the momentum as we continue. We started with IV, then we expanded into subcutaneous, and then certainly expanding into PFS. It opens up different prescribers as well as different patients. And I think that's reflected in terms of how we explain the addressable market expansion at R&D Day.

是的，我想這正是我要說的，提姆。我想說，隨著我們的繼續，這種勢頭只會繼續擴大和加強。我們從靜脈注射開始，然後擴展到皮下注射，最後當然擴展到 PFS。它為不同的處方者和不同的患者提供了機會。我認為這反映在我們如何在研發日解釋可尋址市場擴張。

Certainly what we see is that there will be a growth in biologic share of market, and that we will be well-positioned within that growth. And certainly PFS helps us to maintain our leadership, and certainly with the early-aligned patients and the broader prescriber base, given the fact that we will have IV and PFS.

當然，我們看到的是生物製品市場份額將會成長，並且我們將在這種成長中處於有利地位。當然，考慮到我們將有 IV 和 PFS，PFS 有助於我們保持領先地位，尤其是在早期患者和更廣泛的處方者基礎上。

So, I would think about it as continuing to maintain our momentum and continuing in line with our strategy of early-aligned and broader prescriber base. Thanks for the question.

因此，我認為這是繼續保持我們的勢頭，並繼續符合我們早期調整和更廣泛的處方者基礎的策略。謝謝你的提問。

Joon Lee, Truist Securities.

李俊，Truist 證券公司。

Hey, thanks for the update. Thanks for taking our questions. For the no-go decision on myositis by year end, are they all coming at once or one by one? And if Empasiprubart works in one but not in the other myositis subtypes, what would be some of the reasons for that? And what are you looking forward to learning from that to further enhance the choice of your future indications, and maybe even reprioritize your existing pipeline indications? Thank you.

嘿，謝謝你的更新。感謝您回答我們的問題。年底不去肌炎的決定，是一次全部來還是一一來？如果 Empasiprubart 對一種肌炎亞型有效，但對其他肌炎亞型無效，那麼原因是什麼？您希望從中學到什麼，以進一步增強您未來適應症的選擇，甚至可能重新確定現有管道適應症的優先順序？謝謝。

Yeah, thank you, Joon, for the question, and thanks for being with us. So, in this basket trial where we combine three subtypes of myositis to go no-go decision point will all come at the same time. So, we're synchronizing the first 30 patients across the three indications to make one decision.

是的，謝謝你，Joon，提出這個問題，也謝謝你和我們在一起。因此，在這個籃子試驗中，我們將肌炎的三種亞型結合起來，進行不進行的決策點將同時出現。因此，我們正在同步前 30 名患者的三種適應症，以做出決定。

Remember that this is an operationally seamless Phase 2, Phase 3 trial. So, whilst these data are maturing, we are actually already entering into the Phase 3 portion of the trial at risk. So, technically speaking, the only decision we can make is a stop decision.

請記住，這是一個運行無縫的第 2 階段、第 3 階段試驗。因此，雖然這些數據正在成熟，但我們實際上已經進入了試驗的第三階段部分，面臨風險。因此，從技術上講，我們唯一能做出的決定就是停止決定。

And, of course, the first 30 patients will be informative. So, it will allow us to make stop decisions in one, two, or three of these indications, but it will also allow us to adjust sample size. Look, all three indications come out of the same argenx mold.

當然，前 30 名患者將提供豐富的資訊。因此，它將允許我們針對其中一種、兩種或三種跡像做出停止決定，但它也將允許我們調整樣本量。瞧，這三種適應症都出自同一個 argenx 模具。

I mean, strong conviction in biology, I think a responsible, thoughtful clinical trial design, but not completely risk-free. So, I think we did everything we could to design it and risk mitigate it. So, I don't think there's any specific read-through of a myositis outcome onto any other indication which we have loaded into the pipeline because they all follow the same blueprint. Thanks for the question.

我的意思是，對生物學有堅定的信念，我認為這是一個負責任的、深思熟慮的臨床試驗設計，但並非完全沒有風險。因此，我認為我們已盡一切努力來設計它並降低風險。因此，我認為對於我們已加載到管道中的任何其他適應症，肌炎結果沒有任何具體的解讀，因為它們都遵循相同的藍圖。謝謝你的提問。

Gavin Clark-Gartner, Evercore ISI Group.

Gavin Clark-Gartner，Evercore ISI 集團。

Hey, thanks for taking the question. A quick one on thyroid eye disease. Just wanted to confirm you're doing weekly dosing in the Phase 3 and also just ask how enrollment is going now that you're about four months in. Thanks

嘿，謝謝你提出問題。關於甲狀腺眼疾的快速介紹。只是想確認您在第 3 階段每週進行一次給藥，並詢問現在大約四個月後的註冊進展如何。謝謝

Thanks for the question on TD. You're right that we're in weekly dosing. We're doing that with the pre-filled syringe already, and I think we're in a classical, typical trial execution mode. So, stay tuned on how the study is doing. We will keep you updated over the coming quarters, but we feel very strong about the opportunity and the traction we're getting with this global clinical trial.

感謝您對 TD 的提問。你說得對，我們每週給藥一次。我們已經用預填充注射器做到了這一點，我認為我們處於經典的、典型的試驗執行模式。因此，請繼續關注這項研究的進展。我們將在接下來的幾季向您通報最新情況，但我們對這項全球臨床試驗所帶來的機會和吸引力感到非常強烈。

Thank you.

謝謝。

Andy Chen, Wolfe Research.

安迪陳，沃爾夫研究中心。

Good morning. Thank you for taking the question. So, I want to share this $450,000 pricing. I'm just curious if you can talk about how robustly this pricing can stay at $450,000, because as you have payer contracts, there's a natural pressure for ASP erosion. So, just curious if you can comment on the dynamics here. Do you see room for this number to go up over time in the next few years, or do you think this is going to stay the same at $450,000, or do you think this is going to go down? Thank you.

早安.感謝您提出問題。所以，我想分享一下這個 45 萬美元的定價。我只是很好奇您能否談談這個定價能在多大程度上保持在 450,000 美元，因為當您有付款人合約時，平均售價自然會受到侵蝕。所以，只是好奇你是否可以評論這裡的動態。您認為這個數字在未來幾年內是否有上升的空間，或者您認為這個數字會保持在 450,000 美元不變，還是您認為這個數字會下降？謝謝。

Yeah. So, Andy, thank you for the question, which I'm happy to take. So, let me first briefly comment on the $450,000 number. That number, of course, did not come out of the blue sky. I think it is the result of a thorough understanding of, I think, the value proposition which we have to offer.

是的。安迪，謝謝你提出這個問題，我很高興接受這個問題。那麼，讓我先簡單評論一下45萬美元這個數字。當然，這個數字並非憑空而來。我認為這是對我們必須提供的價值主張的透徹理解的結果。

Careful calibration, of course, with payers, where we socialize the Phase 3 data with them. And the data, actually, which resonates very well with payers is the regain of function data Karen was already alluding to, and especially, I think -- the fact that the majority of the people who entered the trial wheelchair-bound were able to leave the trial outside of the wheelchair.

當然，與付款人進行仔細校準，我們與他們交流第三階段的數據。實際上，與付款人產生很好共鳴的數據是凱倫已經提到的功能數據的恢復，尤其是，我認為，大多數坐輪椅進入試驗的人都能夠離開輪椅外的試驗。

So, these are pretty meaningful data, which represents significant value. We are now finalizing the data agreements, so stay tuned. And we also discussed during the call -- look, I cannot predict the future. I think it is fair to assume that with more competition and more indications, there may be some pressure on your price. But I think, as a company, we're pricing transparently and responsibly, and I think we're very well equipped with our market access strategies to go into our future. Thank you for the question.

所以，這些都是非常有意義的數據，代表著重要的價值。我們現在正在敲定數據協議，敬請期待。我們在電話會議中還討論了——看，我無法預測未來。我認為可以公平地假設，隨著更多的競爭和更多的跡象，您的價格可能會受到一些壓力。但我認為，作為一家公司，我們的定價是透明且負責任的，而且我認為我們已經做好了進入未來的市場准入策略的準備。謝謝你的提問。

Joel Beatty, RW Baird.

喬爾·比蒂，RW·貝爾德。

Thanks, and congrats on the strong growth from Q1. Looking back, what led to the weaker Q1, and how much of that was seasonal in nature and would be expected to repeat versus any dynamics that might have been one time in nature?

謝謝，並祝賀第一季的強勁增長。回顧過去，是什麼導致了第一季的疲軟，其中有多少是季節性的，並且與任何可能一次性發生的動態相比，預計會重複出現？

Yes, thank you for asking the question. I think we alluded to that in the prepared remarks, but maybe, Karen, do you want to comment? When we look through the optics of Q1 and Q2 for the real underlying strength of the businesses?

是的，謝謝您提出問題。我想我們在準備好的發言中提到了這一點，但也許，凱倫，你想發表評論嗎？當我們透過第一季和第二季的數據來了解企業真正的潛在實力？

Yeah, absolutely. When we look at the business, what we look at is those underlying drivers of growth. So are we adding new patients consistently? Are we moving earlier line? Are we broadening our prescriber base? And we see those pretty consistently through Q1 and Q2.

是的，絕對是。當我們審視業務時，我們關注的是那些成長的潛在驅動力。那我們是否持續增加新患者？我們要提前排隊嗎？我們是否正在擴大我們的處方者基礎？我們在第一季和第二季看到這些情況非常一致。

I would say those Q1 dynamics that you see across the industry, not just with VYVGART, around re-verifications, weather, -- I'd say they're always going to be there. Whether they were worse in Q1 because the weather was worse in some parts of the US or whatever you may have, I mean, I think we can all look back at that. But those Q1 dynamics are more, I would say, across the industry. The VYVGART performance and the KPIs, I would say it's pretty consistent throughout actually the 10 quarters of growth that we've had, and we would expect that to continue. Thanks for the question.

我想說的是，您在整個行業中看到的第一季動態，而不僅僅是 VYVGART，圍繞著重新驗證、天氣——我想說它們將永遠存在。無論第一季的情況是否更糟，是因為美國某些地區的天氣更糟，還是其他什麼原因，我的意思是，我認為我們都可以回顧一下這一點。但我想說，第一季的動態更多的是整個產業。我想說，VYVGART 的表現和 KPI 在我們過去 10 個季度的成長中非常一致，我們預計這種情況會持續下去。謝謝你的提問。

Douglas Tsao, H. C. Wainwright.

道格拉斯·曹（Douglas Tsao），H.C.溫賴特（H.C.Wainwright）。

Hi, good morning, and thanks for taking the questions. Just a quick follow-up on ITP. I understand you think you have, from a regulatory standpoint, an efficient way to move forward.

你好，早上好，感謝您提出問題。只是對 ITP 的快速跟進。我理解您認為從監管的角度來看，您擁有一種有效的前進方式。

I'm just curious what feedback you may have gotten from KOLs as well as how you're thinking about the commercial positioning, just given the finding from the prior Phase 3 study in terms of the effect of low-dose steroids. Thank you.

我只是好奇您可能從 KOL 那裡得到了哪些反饋，以及您如何考慮商業定位，只是考慮到之前的第 3 階段研究關於低劑量類固醇效果的發現。謝謝。

I'm not sure we understood the question completely because the line was breaking up. Would you mind briefly recapping the question, please?

我不確定我們是否完全理解了這個問題，因為線路正在中斷。您介意簡單地重述一下這個問題嗎？

Yeah, sorry. Can you hear me now, Tim?

是的，抱歉。提姆，你現在聽得到我說話嗎？

Yes, we can.

是的，我們可以。

Okay. So I was just asking, so I understand you believe in ITP that you have an efficient path forward from a regulatory standpoint. I'm just curious in terms of how you're thinking about the product's positioning, if it has changed prior to the prior Phase 3 results, and what feedback you may have gotten from KOLs, especially in terms of the efficacy or the apparent efficacy of low-dose steroids. Thank you.

好的。我只是想問一下，所以我理解您相信 ITP，從監管的角度來看，您有一條有效的前進道路。我只是好奇您如何看待該產品的定位，在之前的第三階段結果之前它是否發生了變化，以及您可能從 KOL 那裡得到了哪些反饋，特別是在功效或表觀功效方面低劑量類固醇。謝謝。

We got it. So there is quite some excitement in the community about the data which we generated in the clinical trial. In this refractory patient population which we are targeting and in which we will be positioning third-line drugs after steroids and TPOs, in that patient population, not much is really working.

我們明白了。因此，社區對我們在臨床試驗中產生的數據感到非常興奮。在我們瞄準的難治性患者群體中，我們將在類固醇和 TPO 之後定位三線藥物，但在這些患者群體中，真正起作用的並不多。

And we showed a very high response rate and an unprecedented safety profile. And that is really important for the KOLs. They also continued to call us about how well their patients were doing in the study, their ongoing open-label extension.

我們表現出了非常高的回應率和前所未有的安全狀況。這對 KOL 來說非常重要。他們也繼續打電話給我們，詢問他們的患者在這項研究中的表現，以及他們正在進行的開放標籤擴展。

So there was quite some pull from the marketplace, I would say, for this product. And I think they were also very collaborative in helping us to think through the proposal we would make at least at the FDA. So positioning has not changed. KOL feedback is very positive, and that strengthens our determination to go forward. Thanks for the question.

所以我想說，這個產品有相當大的市場吸引力。我認為他們在幫助我們思考至少在 FDA 提出的提案方面也非常合作。所以定位沒有改變。KOL 的回饋非常積極，這增強了我們前進的決心。謝謝你的提問。

This concludes today's question-and-answer session and today's conference call. Thank you for your participation. You may now disconnect.

今天的問答環節和電話會議到此結束。感謝您的參與。您現在可以斷開連線。