argenx SE (ARGX) 2024 Q3 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.

早安.我叫羅布，今天我將擔任你們的會議操作員。我謹歡迎大家致電。

(Operator Instructions)

（操作員說明）

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

謝謝。現在我想介紹一下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 third-quarter 2024 financial results and business update. This can be found on our website along with the presentation for today's webcast. Before we begin, I'd like to remind you that forward-looking statements may be presented during this call. These may include statements about our future expectations, clinical developments, regulatory timelines, the potential success of our product candidates, financial projections and upcoming milestones. Actual results may differ materially from those indicated by these statements.

謝謝。今天稍早發布了一份新聞稿，介紹了我們 2024 年第三季的財務表現和業務更新。您可以在我們的網站上找到該內容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。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 these 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. I will now turn the call over to Tim.

除非法律要求，Argenx 沒有義務更新有關未來的聲明或確認這些與實際結果相關的聲明。執行長 Tim Van Hauwermeiren 也參加了今天的電話會議。卡爾‧古比茨，財務長；和首席營運長凱倫·梅西 (Karen Massey)。我現在將把電話轉給蒂姆。

Thank you, Beth, and welcome, everyone. I'll begin on slide number 3. As we approach the end of 2024, it is incredible to reflect on all that we have accomplished. Having achieved the goal set out in our 2025 vision ahead of plan, we moved the goalpost again in July, raising our ambition with Vision 2030.

謝謝你，貝絲，歡迎大家。我將從第 3 張投影片開始。隨著 2024 年底的臨近，回顧我們所取得的一切真是令人難以置信。在提前實現 2025 年願景中設定的目標後，我們在 7 月再次移動了目標，並提高了 2030 年願景的雄心。

Building on the momentum from R&D day, we are now executing across the business to advance this vision beginning with our commercial expansion. This is our 11th consecutive quarter of growth, and I continue to be impressed with the team's efforts to reach more gMG patients and prescribers, and build our wall of data in support of VYVGART as the leading branded biologic in MG.

憑藉研發當天的勢頭，我們現在正在整個業務範圍內執行，以從商業擴張開始推進這一願景。這是我們連續第 11 個季度實現成長，我對團隊為接觸更多 gMG 患者和處方者以及建立數據牆以支持 VYVGART 作為 MG 領先品牌生物製劑所做的努力印象深刻。

The CIDP launch has started strong as we leverage the powerful data from ADHERE and our established relationships with payers and prescribers to support a quarter that surpassed our expectations. Overall, we are thrilled with our initial progress, bringing innovation to CIDP patients. Karen will provide metrics that highlight the positive reception of the VYVGART Hytrulo across key stakeholders, but overall, we're very happy with our strong start.

CIDP 的推出已經開始強勁，因為我們利用 ADHERE 的強大數據以及我們與付款人和處方者建立的關係來支持超出我們預期的季度。總的來說，我們對我們的初步進展感到非常興奮，為 CIDP 患者帶來了創新。Karen 將提供一些指標來強調 VYVGART Hytrulo 在主要利害關係人中的積極接受，但總的來說，我們對我們的良好開端感到非常滿意。

We are navigating the unique market dynamics of CIDP well, and we're able to deliver our innovation to more than 300 CIDP patients last quarter, which correlates to revenue that is approaching what we saw in the first quarter of our MG launch.

我們正在很好地掌握 CIDP 獨特的市場動態，上季度我們能夠向 300 多名 CIDP 患者提供我們的創新，這與我們在 MG 推出第一季所看到的收入相關。

Slide 4. We now have the benefit of seeing the ADHERE data drive real-world outcomes with some patients already experiencing regain of function early into their treatment journey. My personal favorite story comes from the first CIDP patient dosed with the VYVGART Hytrulo.

幻燈片 4。我們現在受益於看到 ADHERE 數據推動了現實世界的結果，而一些患者在治療過程的早期就已經恢復了功能。我個人最喜歡的故事來自第一位接受 VYVGART Hytrulo 治療的 CIDP 患者。

The patient's wife shared that prior to treatment, her husband struggled with mobility. He recently checked-in, and the patient is now more active. He and his wife have enjoyed traveling again and were able to attend a friend's wedding. He has now been able to take care of himself more independently and is eager to return to activities he loves, like golfing and going to the gym. The wife of the patient expressed her gratitude for this renewed lease on life. This is the type of story that makes this industry so rewarding.

患者的妻子表示，在接受治療之前，她的丈夫行動不便。他最近來院檢查，病人現在更活躍了。他和妻子再次享受旅行的樂趣，並能夠參加朋友的婚禮。他現在已經能夠更獨立地照顧自己，並渴望重返他喜歡的活動，例如打高爾夫球和去健身房。患者的妻子對這次重獲新生表示感謝。正是這種故事讓這個產業變得如此有價值。

Slide 5. Earlier this month, we had a strong presence at AANEM, where we were able to present new clinical trial and real-world data demonstrating our long-term commitment to the neurology community and driving transformational outcomes for patients.

幻燈片 5。本月早些時候，我們在 AANEM 上表現強勁，在那裡我們能夠展示新的臨床試驗和真實世界數據，證明我們對神經病學界的長期承諾並推動患者的轉型結果。

We continue to set a high bar with VYVGART, driving rapid deep and sustained responses, and now we have multiyear data demonstrating durable efficacy, supporting maintenance use. Over 50% of gMG patients on VYVGART achieve MSE, which physicians agree is an important goal of treatment for their patients and we hear the speed of onset is a clear advantage.

我們繼續為 VYVGART 設定高標準，推動快速、深入和持續的反應，現在我們擁有證明持久功效的多年數據，支援維護使用。超過 50% 的接受 VYVGART 治療的 gMG 患者達到 MSE，醫生認為這是患者治療的一個重要目標，我們聽說起效速度是一個明顯的優勢。

We now have more than 8,000 patient years of safety data which demonstrates consistent, favorable safety, no black box warning, no impact on albumin levels, no REMS program, no monitoring requirements, and no need to vaccinate.

我們現在擁有超過 8,000 個患者年的安全數據，這些數據證明了一致、良好的安全性、無黑框警告、對白蛋白水平沒有影響、沒有 REMS 計劃、沒有監測要求、無需接種疫苗。

Consistent with our goal to bring VYVGART into early lung treatment settings, we also presented new visual data that patients can meaningfully reduce steroid use within six months of treatment. This effect was maintained and improved to 12 months, with one out of four patients tapering to 0 mg of steroids, and 42% of patients tapering to 5 mg or less.

與我們將 VYVGART 納入早期肺部治療環境的目標一致，我們還提供了新的視覺數據，表明患者可以在治療後六個月內有意義地減少類固醇的使用。這種效果維持並改善至 12 個月，四分之一的患者逐漸減少到 0 毫克類固醇，42% 的患者逐漸減少到 5 毫克或更少。

AANEM was notably the first congress where we showcased three clinical programs from our pipeline including advancing empasiprubart and ARGX-119. Through these programs, we continue to solidify our leadership in neuromuscular medicine, advancing clinical development across multiple indications, including MG, CIDP, MMN, CMS, and ALS, and generating early excitement as we address unmet needs with our precision medicines.

值得注意的是，AANEM 是我們首次在大會上展示了我們管道中的三個臨床項目，包括推進 empasiprubart 和 ARGX-119。透過這些項目，我們繼續鞏固我們在神經肌肉醫學領域的領導地位，推進包括MG、CIDP、MMN、CMS 和ALS 在內的多種適應症的臨床開發，並在我們用精準藥物解決未滿足的需求時引起早期的興奮。

Slide 6. Before turning the call over to Karl, I would like to highlight some of the opportunities ahead to advance innovation, as we approach the yearend. Remember, we like novel targets with pipeline in a product potential and seek out indications where we believe we can drive the most impact for patients. Indications continue to mature across our three pipeline programs in the clinic, efgartigimod, empasiprubart, and ARGX-119.

幻燈片 6。在將電話轉交給卡爾之前，我想強調一下，隨著年底的臨近，未來推進創新的一些機會。請記住，我們喜歡具有產品潛力的新目標，並尋找我們認為可以為患者帶來最大影響的適應症。我們臨床上的三個管道項目（efgartigimod、empasiprubart 和 ARGX-119）的適應症不斷成熟。

Next step is our Myositis Go/No Go decision before year end. As you may recall, we thoughtfully designed ALKIVIA as our first basket study that would allow us to simultaneously evaluate efgartigimod in three subsets of myositis, necrotizing, antisynthetase, and dermatomyositis.

下一步是我們在年底前決定肌炎是否繼續。您可能還記得，我們​​精心設計了ALKIVIA 作為我們的第一個籃子研究，使我們能夠同時評估efgartigimod 在肌炎、壞死性肌炎、抗合成酶肌炎和皮肌炎三個亞類中的作用。

The primary endpoint of the study is the same across all subsets and is based on the total improvement score with the seamless Phase 2/3 design. We started enrolling in the Phase 3 as soon as we completed enrollment in the Phase 2, which means that the decision we will make in the coming weeks is whether we will continue recruitment in one or more subsets based on the signal we see in the first 90 patients from the Phase 2.

研究的主要終點在所有子集中都是相同的，並且基於無縫 2/3 期設計的總改善分數。我們在完成第2 階段的註冊後立即開始註冊第3 階段，這意味著我們將在未來幾週內做出的決定是，我們是否將根據我們在第一個階段看到的信號繼續在一個或多個子集中進行招募第二階段的 90 名患者。

Lastly, as you saw in today's release, we made a decision to discontinue the development of efgartigimod in MN. As a part of our strategic alliance with Zai Lab, we are leveraging Zai's clinical development in MN and LN, two kidney indications prevalent in China. We did not see a sufficient efficacy signal from the interim data to warrant further investment in MN. Importantly, no safety signals were detected, and now we look forward to the Phase 2 data in LN.

最後，正如您在今天的發布中看到的，我們決定停止在明尼蘇達州開發 efgartigimod。作為我們與再鼎醫藥策略聯盟的一部分，我們正在利用再鼎醫藥在 MN 和 LN（中國常見的兩種腎臟適應症）的臨床發展。我們沒有從中期數據中看到足夠的有效性訊號來保證對 MN 的進一步投資。重要的是，沒有偵測到安全訊號，現在我們期待 LN 的第二階段資料。

It is part of our mission to privatize programs that have the potential to drive transformational outcomes in patients, and we do not see this opportunity with efgartigimod in MN. To close, we are well positioned to continue investing in our innovation, advancing high-value novel treatments that can improve outcomes for patients and maximize value for shareholders.

我們使命的一部分是將有潛力推動患者轉化結果的項目私有化，但我們在 MN 中沒有看到 efgartigimod 帶來的機會。最後，我們處於有利位置，可以繼續投資我們的創新，推進高價值的新型治療方法，從而改善患者的治療結果並為股東實現價值最大化。

I will now turn the call over to Karl.

我現在將把電話轉給卡爾。

Thank you, Tim. Slide 7. The third quarter 2024 financial results are detailed in the press release of this morning. Total operating income in the third quarter totals $589 million. This reflects $573 million in product net sales and $16 million in other operating income.

謝謝你，提姆。幻燈片 7。今天上午的新聞稿詳細介紹了 2024 年第三季財務業績。第三季營業總收入總計 5.89 億美元。這反映了 5.73 億美元的產品淨銷售額和 1,600 萬美元的其他營業收入。

The product net sales of $573 million represent 20% quarter-over-quarter growth and 74% growth compared with a corresponding prior year quarter. The product revenue breaks down by region to $492 million in the US, $24 million in Japan, $46 million in the rest of the world, and $11 million products supplied to Zai Lab in China. The net sales in the US is inclusive of both CIDP and MG revenue. Karen will provide additional color in her section on the initial launch performance.

該產品淨銷售額為 5.73 億美元，較上季成長 20%，與去年同期相比成長 74%。產品收入按地區劃分為：美國為 4.92 億美元，日本為 2,400 萬美元，世界其他地區為 4,600 萬美元，向中國再鼎醫藥供應的產品收入為 1,100 萬美元。美國的淨銷售額包括 CIDP 和 MG 收入。凱倫（Karen）將在她的初次發布性能部分提供更多的色彩。

Slide 8. Total operating expense in Q3 are $575 million, an increase of $40 million compared with Q2 2024. The increase is due to a $22 million increase in SG&A reflecting incremental expenses on the CIDP loans in the US, and $11 million increase in R&D, and an increase of $7 million in cost of sales. Cost of sales is $59 million in Q3. This reflects a gross margin of 90%, which is in line with previous quarters.

幻燈片 8。第三季的總營運費用為 5.75 億美元，比 2024 年第二季增加了 4,000 萬美元。這一增長是由於 SG&A 增加了 2,200 萬美元，反映了美國 CIDP 貸款的增量費用，研發費用增加了 1,100 萬美元，銷售成本增加了 700 萬美元。第三季的銷售成本為 5,900 萬美元。這反映出 90% 的毛利率，與前幾季持平。

In Q3, SG&A expenses are $278 million and R&D expenses are $236 million. This results in operating profit for Q3 of $14 million. On a year-to-date basis, operating loss is $125 million.

第三季度，SG&A 費用為 2.78 億美元，研發費用為 2.36 億美元。這使得第三季的營業利潤達到 1,400 萬美元。今年迄今，營運虧損為 1.25 億美元。

The quarterly net financial income is $41 million, and it also benefits from unrealized exchange gains of $34 million on our euro-denominated cash balances. After tax, the profit for the quarter is $91 million and the year-to-date profit after tax is $59 million.

季度淨財務收入為 4,100 萬美元，也受惠於我們以歐元計價的現金餘額中 3,400 萬美元的未實現匯兌收益。該季度稅後利潤為 9,100 萬美元，年初至今稅後利潤為 5,900 萬美元。

Our cash balance, represented by cash, cash equivalents, and current financial assets is $3.4 billion at quarter end. The balance increased by $272 million in the quarter, and our cash guidance for 2024 no longer applies. The financial guidance on the combined SG&A and R&D spend of $2 billion remains unchanged.

截至季末，我們的現金餘額（以現金、現金等價物和流動金融資產為代表）為 34 億美元。本季餘額增加了 2.72 億美元，我們對 2024 年的現金指引不再適用。SG&A 和研發支出合計 20 億美元的財務指引維持不變。

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 9. It's an exciting time for argenx. I want to first give a huge thank you to the entire team who continue to execute on all fronts to make this possible.

謝謝你，卡爾。幻燈片 9。對於 argenx 來說，這是一個令人興奮的時刻。我首先要向整個團隊表達衷心的感謝，他們在各方面不斷努力，使這一切成為可能。

Patients are at the center of our innovation mission, and I'm pleased that our clear patient centric strategy and disciplined approach has delivered continuous growth in gMG while at the same time contributing to a strong first quarter of launch in CIDP in the US, and ITP in Japan.

患者是我們創新使命的中心，我很高興我們明確的以患者為中心的策略和嚴格的方法實現了 gMG 的持續增長，同時為 CIDP 在美國第一季的強勁推出做出了貢獻，日本的ITP 。

I'll begin with the details of our CIDP launch before zooming out to the overall performance for the quarter, highlighting the dynamics driving our gMG performance and sharing our outlook for sustained growth.

我將首先介紹 CIDP 發布的詳細信息，然後介紹本季度的整體業績，強調推動我們 gMG 業績的動力，並分享我們對持續增長的展望。

Slide 10. The team has successfully advanced each of the core strategies that we outlined at the CIDP approval to reach our key stakeholders, patients, physicians and payers.

幻燈片 10。該團隊已成功推進了我們在 CIDP 批准時概述的每項核心策略，以惠及我們的主要利害關係人、患者、醫生和付款人。

Let's begin with payers, where enabling broad patient access has been an area of core focus. Since CIDP approval, it has been our priority to secure access as quickly as possible, leveraging the credibility and the strong partnerships we establish with payers through our MG experience.

讓我們從付款人開始，讓廣泛的患者獲得服務一直是核心關注領域。自 CIDP 獲得批准以來，我們的首要任務是利用我們透過 MG 經驗與付款人建立的信譽和牢固的合作夥伴關係，盡快確保訪問。

Today, we are pleased to have broadly favorable policies in place that cover approximately 54% of US commercial lives. Most but not all policies require some prior IG utilization. This is not surprising to us and supports our expected growth trajectory because most CIDP patients have prior IVIg experience, and it's commonly part of diagnosis.

今天，我們很高興能夠制定覆蓋美國約 54% 商業生活的廣泛優惠政策。大多數（但並非全部）保單都需要事先利用 IG。這對我們來說並不奇怪，並且支持我們預期的成長軌跡，因為大多數 CIDP 患者都有先前的 IVIg 經驗，而且它通常是診斷的一部分。

Important to note is that for those policies that require prior IVIg use, it's usually exposure of three months or less at any time with the patient's history which is favorable for VYVGART Hytrulo. It typically takes two quarters to get most policies in place after any launch, and we're on track with this timeline, with the remaining policies expected to come into place over the next few months. Overall, payers recognize the value that Hytrulo can bring to patients.

需要注意的是，對於那些需要事先使用 IVIg 的政策，根據患者的病史，通常在任何時間暴露三個月或更短時間，這對 VYVGART Hytrulo 是有利的。任何政策推出後，通常需要兩個季度的時間才能落實到位，我們正在按照這個時間表實施，其餘政策預計將在未來幾個月內落實到位。總體而言，付款人認識到 Hytrulo 可以為患者帶來的價值。

Moving on to prescribers, the strength of our data, and the clear value proposition of VYVGART Hytrulo to patients is also driving initial uptake with physicians. Neurologists are excited to gain experience with the first CIDP innovation in 30 years, a safe and effective treatment that has the power to drive functional benefit in patients with a 30- to 90-second simple injection.

對於處方者來說，我們的數據優勢以及 VYVGART Hytrulo 對患者的明確價值主張也推動了醫生的初步接受。神經科醫生很高興能獲得 30 年來首個 CIDP 創新的經驗，這是一種安全有效的治療方法，只需 30 至 90 秒的簡單注射即可使患者功能受益。

At the time of launch, we shared that we have an estimated 10,000 total neurologists banning CIDP and MG with approximately 72% overlap in physicians who treat both. We expected that physicians with prior VYVGART experience would most likely be the first CIDP prescribers. But our goal was also to broaden the VYVGART prescriber base.

在發佈時，我們表示，估計有 10,000 名神經科醫生禁止 CIDP 和 MG，其中治療這兩種藥物的醫生大約有 72% 的重疊。我們預計具有 VYVGART 經驗的醫生很可能是第一批 CIDP 處方者。但我們的目標也包括擴大 VYVGART 處方者基礎。

I'm pleased to share that 25% of CIDP prescribers to date are first time VYVGART prescribers, and some who started with CIDP have now also prescribed the gMG. We are seeing initial breadth through our prescriber base as well as depth. Many prescribers have already started multiple patients on treatment.

我很高興地告訴大家，到目前為止，25% 的 CIDP 處方者是第一次使用 VYVGART 處方，一些開始使用 CIDP 的處方者現在也開了 gMG。我們看到了我們的處方者基礎的初步廣度和深度。許多處方醫生已經開始對多名患者進行治療。

Lastly, and most importantly, we are thrilled to see innovation deliver tangible impact as VYVGART Hytrulo reaches CIDP patients in need. Expanding on Tim's earlier story, we're inspired by the feedback we've received so far. Many patients are able to enjoy more moments with their loved ones and regain the independence to do the activities that you and I often take for granted.

最後也是最重要的是，我們很高興看到隨著 VYVGART Hytrulo 惠及有需要的 CIDP 患者，創新帶來了切實的影響。擴展蒂姆之前的故事，我們受到迄今為止收到的反饋的啟發。許多患者能夠與親人一起享受更多的時光，並重新獲得獨立性來進行你我經常認為理所當然的活動。

We are pleased to share that as of the end of the quarter, there were over 300 patients on VYVGART Hytrulo, delivering a strong quarter of CIDP revenue which approached that of our first MG quarter. Of these patients, 85% to 90% are coming from IVIg, which supports our position that this would largely be a switch market.

我們很高興與大家分享，截至本季末，VYVGART Hytrulo 上有超過 300 名患者，CIDP 營收季度表現強勁，接近我們第一個 MG 季度的收入。在這些患者中，85% 至 90% 來自 IVIg，這支持了我們的立場，即這將在很大程度上是一個轉換市場。

Overall, the team has done an incredible job getting patients on treatment smoothly and we will continue to monitor how the launch trajectory progresses. We will specifically be looking at response rates, patients getting on, and staying on treatment, and how utilization trends over time. In particular, in light of the OLE data that we will share next year with bi-weekly or every three-week dosing.

總體而言，團隊在讓患者順利接受治療方面做了令人難以置信的工作，我們將繼續監測啟動軌蹟的進展。我們將特別關注緩解率、患者的繼續治療和持續治療，以及隨時間推移的使用率趨勢。特別是，根據我們明年將分享的每兩週或每三週給藥一次的 OLE 數據。

Now let's turn our attention to MG, slide 11. MG drove the majority of growth over the quarter with continued momentum and steady growth across VYVGART and VYVGART Hytrulo. In the US, Hytrulo continues to attract both new patients and new prescribers. This is important for a couple of reasons. First, over 60% of new VYVGART and Hytrulo patients are coming from oral, which is consistent with our goal to reach earlier line patients.

現在讓我們把注意力轉向 MG，投影片 11。MG 憑藉 VYVGART 和 VYVGART Hytrulo 的持續勢頭和穩定增長推動了本季度的大部分成長。在美國，Hytrulo 繼續吸引新患者和新處方者。這很重要，原因有幾個。首先，超過 60% 的新 VYVGART 和 Hytrulo 患者來自口服，這與我們接觸早期第一線患者的目標是一致的。

Second, we continue to expand our prescriber base for MG with Hytrulo, and 12% of prescribers were brand new to VYVGART. Our goal is for patients not to have the constant reminder of their disease, and we hope to achieve this with our efficacy results delivering MSE in over half of our patients and our demonstrated safety.

其次，我們繼續透過 Hytrulo 擴大 MG 處方者基礎，12% 的處方者是 VYVGART 的全新處方者。我們的目標是讓患者不再不斷想起自己的疾病，我們希望透過我們為超過一半的患者提供 MSE 的療效結果以及我們所證明的安全性來實現這一目標。

We're generating real world evidence that shows that more consistent cycles lead to sustained efficacy for patients, and that over half of patients can meaningfully taper off steroids after VYVGART initiation.

我們正在產生真實世界的證據，表明更一致的週期可以為患者帶來持續的療效，超過一半的患者在 VYVGART 開始後可以有意義地逐漸減少類固醇。

We also aim to enhance the patient experience by offering both IV and a simple subcutaneous option. VYVGART Hytrulo was an important step forward with this strategy and advancing our prefilled syringe is next. A significant amount of innovation went into this regulatory submission and we're looking forward to the PFS PDUFA date in April 2025.

我們還旨在透過提供靜脈注射和簡單的皮下注射選項來增強患者體驗。VYVGART Hytrulo 是這項策略的重要一步，下一步是改進我們的預充式註射器。此次監管提交包含大量創新，我們期待 2025 年 4 月的 PFS PDUFA 日期。

Slide 12. We also had strong momentum in MG sales globally and are proud that VYVGART is now available to over 80% of the gMG population in the EU. The ITP launch in Japan is showing early success and the data from advance are playing out in the real world with 50% of patients responding. And last, we are working to bring our innovation to CIDP patients worldwide. Regulatory reviews are ongoing in China, Japan, and Europe, with approval decisions on track for 2025.

幻燈片 12。我們在全球 MG 銷售方面也表現強勁，令我們感到自豪的是，歐盟 80% 以上的 gMG 人群現在都可以使用 VYVGART。ITP 在日本的推出顯示出早期的成功，且前期數據正在現實世界中發揮作用，50% 的患者做出了反應。最後，我們正在努力將我們的創新帶給全球 CIDP 患者。中國、日本和歐洲正在進行監管審查，預計 2025 年做出批准決定。

Slide 13. We have a lot to look forward to. There is significant opportunity in the pipeline as we expand into new diseases that all have high unmet need for innovation, and the commercial organization is laser focused on executing our plan to maintain momentum and growth in MG, CIDP, and ITP. All to fuel our differentiated pipeline and broaden our patient impact globally. I will now turn the call back to Tim.

幻燈片 13。我們有很多值得期待的事。隨著我們擴展到新疾病，這些疾病對創新的需求都未得到滿足，商業組織正在專注於執行我們的計劃，以保持 MG、CIDP 和 ITP 的勢頭和增長，因此存在重大機會。所有這些都是為了推動我們的差異化產品線並擴大我們在全球範圍內對患者的影響。我現在將把電話轉回給蒂姆。

Thanks, Karen. I'd like to thank the argenx team for their ongoing commitment to changing the lives of patients through our science. As we approach the end of the year, we are part of all that we have accomplished, gaining invaluable insights along the way to solidify our deep understanding of immunology.

謝謝，凱倫。我要感謝 argenx 團隊持續致力於透過我們的科學改變患者的生活。隨著年底的臨近，我們成為我們所取得的一切成就的一部分，一路上獲得了寶貴的見解，以鞏固我們對免疫學的深刻理解。

We are energized by the opportunity ahead to amplify our impact, recognizing we're still early into the CIDP launch, and have the potential to reach new patients across multiple indications in the clinic. With yearend in sight, our best-in-class innovation shows no signs of slowing down as we continue to build out long-term value with VYVGART, and move swiftly to advance our promising clinical candidates.

我們對未來擴大影響力的機會感到充滿活力，認識到我們仍處於 CIDP 啟動的早期階段，並且有潛力接觸臨床中多種適應症的新患者。臨近年底，我們一流的創新絲毫沒有放緩的跡象，我們繼續透過 VYVGART 創造長期價值，並迅速採取行動推進我們有前途的臨床候選藥物。

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。我現在想開始回答你們的問題。

Thank you. (Operator Instructions)

謝謝。（操作員說明）

Derek Archila, Wells Fargo.

德里克·阿奇拉，富國銀行。

Hey, good morning. Thanks for taking the questions and congrats on the strong performance here. Maybe just first off, I was hoping you could talk about the trajectory of the CIDP launch since the two patients, Karen talked about the R&D Day to now be over 300 at the end of the third quarter. Has that been fairly linear or was there a recent bolus?

嘿，早安。感謝您提出問題，並祝賀您在這裡的出色表現。也許首先，我希望您能談談 CIDP 啟動的軌跡，因為凱倫兩位患者談到了研發日，截至第三季末，研發日已超過 300 個。這是相當線性的還是最近有推注？

And then just to follow up to that, I guess, how do you think about some of the variables in the CIDP launch, and how does that figure into your thinking on potentially issuing guidance in 2025? Thanks.

接下來，我想，您如何看待 CIDP 啟動中的一些變量，以及您對 2025 年可能發布指南的想法有何影響？謝謝。

Thank you, Derek. Thanks for being with us today and thank you for the compliments on a very strong quarter. I think this is a question for you, right, Karen?

謝謝你，德里克。感謝您今天與我們在一起，並感謝您對一個非常強勁的季度的讚揚。我想這是你的問題，對吧，凱倫？

Yeah. Thanks for the question, and first of all, thanks for recognizing the strong quarter, I think we're really proud of the team, and the disciplined execution, the focus that they've delivered a strong quarter.

是的。感謝您提出問題，首先，感謝您對這一強勁季度的認可，我認為我們真的為我們的團隊、紀律嚴明的執行力以及他們交付強勁季度的專注感到自豪。

In terms of your first question, we have not seen a bolus in the CIDP launch and to use your phrase, I would say, the patient starts have been relatively linear through the quarter. As with any launch, it takes time for from when the prescription is written to when the patient gets on therapy, as payer policies have come on board through the quarter. So the majority of our patient starts were in September, but there has not been a bolus that we've seen.

關於你的第一個問題，我們沒有看到 CIDP 啟動中的推注，用你的話來說，我想說的是，整個季度患者的啟動相對線性。與任何產品的推出一樣，從開立處方到患者接受治療都需要一段時間，因為支付保險政策在整個季度都已生效。因此，我們的大多數患者都是在 9 月開始治療的，但我們還沒有看到推注。

On your second question related to what we're seeing in terms of the CIDP launch dynamics, I would say we're exactly where we thought we would be. And we're really proud of the strong start that we have. In terms of how we laid out the strategy, the payer policies, we said they would take two quarters to come in to get the payer policies in place, and we're right on track with that.

關於你的第二個問題，即我們所看到的 CIDP 啟動動態，我想說的是，我們正處於我們預期的位置。我們為我們擁有的強勁開局感到非常自豪。就我們如何制定策略、付款人政策而言，我們表示他們需要兩個季度的時間才能落實付款人政策，而我們正在按計劃進行。

We shared that we have about 54% of commercial lives covered, and we expect that the payer policies will continue to come in over the next quarter. We've certainly seen strong uptake amongst physicians. We shared that 75% of the CIDP prescribers have had prior VYVGART use, but we've also seen a broadening in the prescriber base.

我們表示，我們涵蓋了大約 54% 的商業生活，我們預計付款人保單將在下個季度繼續實施。我們確實看到了醫生的強烈接納。我們分享說，75% 的 CIDP 處方者之前曾使用過 VYVGART，但我們也看到處方者基礎有所擴大。

So 25% of prescribers for CIDP are new to the VYVGART franchise. And in fact, some of those that wrote for CIDP first have also now started a patient on MG. So that's also a really good signal. The reflection from the neurologist is that the regain of function data is resonating very well with them and with their patients. So I would say we're off to a strong start. The dynamics are exactly as we expected.

因此，25% 的 CIDP 處方者是 VYVGART 特許經營的新手。事實上，一些最初為 CIDP 撰稿的人現在也開始對 MG 患者進行治療。所以這也是一個非常好的訊號。神經科醫生的反映是，功能數據的恢復與他們及其患者產生了很好的共鳴。所以我想說我們有了一個好的開始。動態完全符合我們的預期。

Moving forward, what we plan to provide in terms of launch metrics is not guidance on revenue or number of patients, but rather we'll continue to provide those metrics or those KPIs that are most relevant to how the launch is progressing. Thanks for the question.

展望未來，我們計劃在發布指標方面提供的不是收入或患者數量的指導，而是我們將繼續提供與發布進度最相關的指標或 KPI。謝謝你的提問。

Tazeen Ahmad, Bank of America.

塔津·艾哈邁德，美國銀行。

Hi, guys. Good morning. Thanks for taking my question. Maybe this is also for Karen. I think at the beginning of the launch, you talked about the time it might take to get onto formulary and the time it might take for a patient to receive treatment for CIDP from the time the script is written.

嗨，大家好。早安.感謝您提出我的問題。或許這也是為了凱倫。我想在發布之初，您談到了進入處方集可能需要的時間，以及從腳本編寫開始患者接受 CIDP 治療可能需要的時間。

Can you give us an update on both of those metrics? Maybe a little bit more focused on the latter because I think people are still wondering if your initial cautious guidance about the time it would take to receive treatment is still true or is that trending above expectations? Thanks.

能為我們介紹一下這兩個指標的最新情況嗎？也許更關注後者，因為我認為人們仍然想知道您最初關於接受治療所需時間的謹慎指導是否仍然正確，或者趨勢是否超出預期？謝謝。

Yeah. Thanks for the question. Happy to address this. So with any launch, actually, with any patient start, there is a delay between when the prescription is written, and the patient actually receives the treatment. And at launch, this is often longer than once you're in steady state because payer policies are coming online during that time. What we've seen for CIDP is in line with what we saw for MG, and in line with what our expectations are.

是的。謝謝你的提問。很高興解決這個問題。因此，實際上，無論是任何啟動，還是任何患者啟動，從開立處方到患者實際接受治療之間都會存在延遲。在啟動時，這通常比處於穩定狀態時要長，因為付款人保單會在這段時間內上線。我們對 CIDP 的觀察與對 MG 的觀察一致，也符合我們的期望。

So patients are moving relatively smoothly from prescription to patients -- to getting on therapy when there are payer policies in place. And I really do want to recognize the team for their efforts, not just for getting payer policies in place, but also for the team that does support getting patients through that process and getting them onto treatment.

因此，在支付政策到位的情況下，患者從處方到患者的轉變相對順利，再到接受治療。我真的很想認可這個團隊所做的努力，不僅是為了讓付款人政策到位，而且是為了支持患者完成這一過程並讓他們接受治療的團隊。

James Gordon, JP Morgan.

詹姆斯‧戈登，摩根大通。

Hello. James Gordon, JP Morgan. Thanks for taking the question.

你好。詹姆斯‧戈登，摩根大通。感謝您提出問題。

The question was just, if we try and break out how much of sales came from CIDP or MG during the quarter, would it be fair that most of the [odds] were still MG? And it was something like a $20 million odd in terms of CIDP? And if that is right, is that pretty much all in the last month based on that when the patients came on to therapy? So is that then a figure that we could annualize, or would we need to be a bit careful extrapolating what you did in the final month of the quarter? And what that would equate to on a yearly basis?

問題只是，如果我們嘗試統計本季有多少銷售額來自 CIDP 或 MG，那麼大多數 [賠率] 仍然是 MG 是否公平？就 CIDP 而言，這大約是 2000 萬美元？如果這是正確的，那麼這幾乎都是上個月基於患者接受治療時的情況嗎？那麼，我們是否可以將這個數字按年化，或者我們是否需要謹慎地推斷您在本季度最後一個月所做的事情？每年這相當於什麼？

Thank you, James, for the question. These are actually two questions in one. I think, Karl, why don't you comment on question number one, and then we can handle question number two, right, Karen?

謝謝詹姆斯提出的問題。這其實是兩個問題合而為一。我想，卡爾，為什麼不對第一個問題發表評論，然後我們可以處理第二個問題，對吧，凱倫？

Yeah. Thank you, James. I mean, I think that actually your numbers are correct in terms of we have 300 -- more than 300 patients on treatment for CIDP at the end of the quarter, that compares to 380 which we had for MG, and during the first quarter. For MG, we had $21 million of sales. So your extrapolation to CIDP makes sense because most of those patients came on board or initiated their treatment, I should say, at the end of a quarter in September. So I think that makes perfect sense.

是的。謝謝你，詹姆斯。我的意思是，我認為實際上你的數字是正確的，因為到本季末我們有300 名——超過300 名患者正在接受CIDP 治療，而第一季我們接受MG 治療的患者人數為380 名。MG 的銷售額為 2100 萬美元。因此，您對 CIDP 的推論是有道理的，因為我應該說，大多數患者在 9 月的一個季度末就開始接受治療或開始治療。所以我認為這是完全合理的。

And then on your second question, James, a quick toss here from Karen.

然後，關於你的第二個問題，詹姆斯，凱倫快速拋出。

Yes. In terms of extrapolating, moving forward. Look, I'd say still early days, I wouldn't try to get ahead of ourselves just yet. We still have a few dynamics that we need to see play out in the CIDP launch. Just a few of those really quick. Don't forget that with the ADHERE data, there was a 67% response rate. So we need to see how that plays out in the real world.

是的。就推斷而言，向前推進。聽著，我想說還為時過早，我還不會試圖超越自己。我們仍然需要看到一些動態在 CIDP 啟動中發揮作用。只是其中的一些，速度非常快。不要忘記，根據 ADHERE 數據，回應率為 67%。所以我們需要看看這在現實世界中是如何發生的。

We also have open label extension data that we'll be sharing next year on biweekly and every three-week dosing. So we need to see what real utilization looks like. And of course, there is competition in this market as well that we'll have to be watching. So I'd say it's still early days.

我們還有開放標籤擴展數據，我們將在明年分享每兩週一次和每三週一次的給藥劑量。所以我們需要看看真正的利用率是什麼樣的。當然，這個市場也存在著我們必須關注的競爭。所以我想說現在還為時過早。

Allison Bratzel, Piper Sandler.

艾莉森‧布拉澤爾，派珀‧桑德勒。

Hey, good morning, and congrats on the quarter. Thanks for taking the question. I think you indicated that around 85% or 90% of CIDP patients have switched from IVIg. Could you just talk to those who aren't IVIg switchers? Were any of those newly diagnosed patients or were they coming from a different therapy? Any color there would be helpful. Thank you.

嘿，早上好，恭喜本季。感謝您提出問題。我認為您指出大約 85% 或 90% 的 CIDP 患者已不再使用 IVIg。您能和那些不是 IVIg 轉換者的人談談嗎？這些患者是新診斷的還是接受了不同的治療？任何顏色都會有幫助。謝謝。

Thanks, Alison for being with us today. So it is absolutely right that about 85% of patients are actually on IVIg when they come on to Hytrulo. Remember that's perfectly in line with expectations. The line share of the CIDP patients are on IVIg, that's also used as a diagnostic tool in the diagnostic journey. So no surprise.

謝謝艾莉森今天和我們在一起。因此，大約 85% 的患者在接受 Hytrulo 治療時實際上正在接受 IVIg，這是絕對正確的。請記住，這完全符合預期。CIDP 患者的線路份額使用 IVIg，這也被用作診斷過程中的診斷工具。所以這並不奇怪。

The other 15% or so of patients think of them as mainly coming from steroids or other immunosuppressants. Thank you for the question.

另外 15% 左右的患者認為它們主要來自類固醇或其他免疫抑制劑。謝謝你的提問。

Myles Minter, William Blair.

邁爾斯·明特，威廉·布萊爾。

Thanks for taking the question. Can you just give us a little bit more color on exactly what the prior step through therapy on immunoglobulin looks like and that the payer formulary coverage for CIDP. Like you said, you have to respond and then go on to SCIg maintenance and then relapse and that's a patient that would be considered successful in a prior auth setting or is it really up to the clinician to see how their patients are responding to IVIg before they can kind of tick that box and put them on VYVGART Hytrulo. Thanks very much.

感謝您提出問題。您能否為我們提供更多信息，說明免疫球蛋白治療的前一步到底是什麼樣子，以及付款人的 CIDP 處方承保範圍。就像你說的，你必須做出反應，然後繼續SCIg 維持，然後復發，這是一個在之前的驗證設置中被認為是成功的患者，或者真的取決於臨床醫生，看看他們的患者在先前對IVIg 的反應如何他們可以勾選該框並將其放在 VYVGART Hytrulo 上。非常感謝。

Yeah, thanks for the question. So we're really pleased with where we've landed with our payer policies. I'd say they're generally favorable. And I think that they generally do have some IVIg or IG prior experience in general. And what that means is that it's not -- that there has to be some documentation of prior IVIg or subcutaneous IG use. But as you say, there's not a documentation of, for example, response rate or [I-RODS] or any of those measures that need to be used. So it is favorable.

是的，謝謝你的提問。因此，我們對付款人政策所取得的進展感到非常滿意。我想說他們總體上是有利的。我認為他們通常確實有一些 IVIg 或 IG 先前經驗。這意味著並非必須有一些先前使用 IVIg 或皮下 IG 的記錄。但正如您所說，沒有關於回應率或 [I-RODS] 或任何需要使用的措施的文件。所以是有利的。

It's also generally prior IVIg or subcutaneous IG in a lengthy period of time, sometimes through the patient's entire treatment cycle. So the policies are favorable, and we're pleased with where they're at.

通常也是在很長一段時間內（有時是患者的整個治療週期）之前接受過 IVIg 或皮下 IG 治療。所以政策是有利的，我們對它們的現狀感到滿意。

Alex Thompson, Stifel.

亞歷克斯湯普森，斯蒂菲爾。

Great. Thanks for taking my question and congrats on the quarter as well. I guess it's a follow up to the payer question. I guess, can you talk a little about your discussions around IG experience? And just given the broader population of ADHERE, do you expect to sort of be able to push past that in the future or is this sort of how you expect to see payer policies moving forward? Thanks.

偉大的。感謝您提出我的問題，也恭喜本季。我想這是對付款人問題的後續。我想，可以談談你們關於IG經驗的討論嗎？鑑於 ADHERE 的人口範圍更廣泛，您是否期望在未來能夠超越這一點，或者這就是您期望看到付款人政策向前發展的方式嗎？謝謝。

Yeah, Alex, I guess this is a variation of the same theme. So typically, payer policies position us behind IG, whether that's IV or subcu IG, which is not surprising given the price point.

是的，亞歷克斯，我想這是同一主題的變體。因此，通常情況下，付款人保單將我們置於 IG 之後，無論是 IV 還是 subcu IG，考慮到價格點，這並不奇怪。

The data which really resonates very well with payers is the region of function data. That's where they see value above and beyond the current therapies out there. And when we say that the payer policies are typically favorable, it basically means that there needs to be some sort of documentation at some point in the past where the patient had an insufficient response to IG, or and/or a tolerability issue.

真正引起付款人共鳴的數據是函數數據區域。這就是他們看到超越現有療法的價值的地方。當我們說付款人政策通常是有利的時，這基本上意味著在過去的某個時刻需要有某種文件記錄，表明患者對 IG 的反應不足，或和/或存在耐受性問題。

That's how the language reads. Some of these policies are actually visible publicly, but that's what it is. And we think that's not really going to be a practical handicap to the launch of the product.

這就是語言的讀法。其中一些政策實際上是公開可見的，但事實就是如此。我們認為這不會真正成為該產品推出的實際障礙。

Danielle Brill, Raymond James.

丹妮爾·布里爾，雷蒙德·詹姆斯。

Hi guys. Good morning. Congrats on the strong start of the launch. So on a percent of [TAM] basis, it looks like you captured more of the CIDP population right out of the gate than you even did with MG. Has this early strength impacted your thinking on the overall size of the opportunity? I mean to ask a little more directly, you said you're committed to treating 50,000 patients by 2030. Could you potentially get there with just MG and CIDP? Thank you.

嗨，大家好。早安.祝賀發射的良好開局。因此，以 [TAM] 的百分比為基礎，看起來您一開始就捕獲了比 MG 捕獲的更多 CIDP 人群。這種早期的優勢是否影響了您對機會整體規模的看法？我想問得更直接一些，您說您承諾到 2030 年治療 50,000 名患者。僅靠 MG 和 CIDP 就能實現這一目標嗎？謝謝。

Thank you, Daniel. This is an excellent question. And maybe, Karen, you would like to take it.

謝謝你，丹尼爾。這是一個很好的問題。也許，凱倫，你願意接受它。

Yes, certainly, I'll talk to this. So in terms of the CIDP, we are off to a strong start exactly as you say, and we're really pleased with that early signs being strong. The total patient population that we shared for CIDP on the addressable market is 12,000 patients and we're not changing that. We believe that that's still the patient population, that's the addressable market, and that's the -- those that are uncontrolled on IVIg.

是的，當然，我會談談這個。因此，就 CIDP 而言，正如您所說，我們有了一個良好的開端，我們對早期跡象的強勁感到非常高興。我們在目標市場上為 CIDP 共享的患者總數為 12,000 名患者，我們不會改變這一點。我們相信，這仍然是患者群體，這就是潛在市場，這就是那些在 IVIg 上不受控制的人。

We believe that between MG, CIDP, as well as the expansion indications that we have coming over the coming years, we're excited to look forward to the 50,000 patients in 2030. But that goes beyond MG and CIDP.

我們相信，結合 MG、CIDP 以及我們未來幾年的擴展適應症，我們很高興期待 2030 年將有 50,000 名患者。但這超出了 MG 和 CIDP 的範圍。

Akash Tewari, Jefferies.

阿卡什·特瓦里，杰弗里斯。

Hey, this is Amy on for Akash. Thanks for taking our question and congrats on the quarter. Is there a potential for you to combine your C2 with VYVGART and CIDP? And when can we start seeing -- when can you start these studies? And what could they show? And then also when can we get an update on your remaining Zai Lab trial in lupus nephritis? And what's your internal confidence in this? Thanks so much.

嘿，這是阿卡什的艾米。感謝您提出我們的問題並祝賀本季。您是否有可能將 C2 與 VYVGART 和 CIDP 結合？我們什麼時候可以開始看到──什麼時候可以開始這些研究？他們能展示什麼？那麼我們什麼時候可以得到 Zai Lab 狼瘡性腎炎剩餘試驗的最新資訊？您對此有何內在信心？非常感謝。

Yes, Amy. Thank you for the questions. That's probably always an opportunity to start combining certain therapies in severe autoimmunity, just like people are doing in cancer. For the moment, we really focused on launching efgartigimod which I think has shown unparalleled data in CIDP, just launching that product into the plan, which we just have been discussing. We do know that complement is in play probably as a result of autoantibodies driving that, and so we think empa deserves its own individual shot on goal in CIDP. So combo is a theoretical possibility, let's not take it off the table. But we have plenty of work set out for us for both VYVGART and empa.

是的，艾米。謝謝你的提問。這可能始終是一個開始結合某些療法來治療嚴重自體免疫疾病的機會，就像人們在癌症治療中所做的那樣。目前，我們真正專注於推出 efgartigimod，我認為它在 CIDP 中顯示了無與倫比的數據，只是將產品納入我們剛剛討論的計劃中。我們確實知道補體正在發揮作用，可能是自身抗體驅動的結果，因此我們認為 empa 值得在 CIDP 中擁有自己的個人射門機會。所以組合是一種理論上的可能性，我們不要把它排除在外。但我們為 VYVGART 和 empa 準備了大量的工作。

With regards to Zai Lab, remember they were doing two proof of concept trials to help us with the big volume of work we need to lift, and two kidney medications, membrane property and lupus nephritis. Both studies have been executed very well by Zai or being executed very well by Zai. So stay tuned when this data become public, we will definitely talk about it. So far, the trial is in rolling very well and we are very pleased with the quality which Zai has shown in its clinical trial execution across both indications.

關於再鼎醫藥，請記住他們正在進行兩項概念驗證試驗，以幫助我們完成需要完成的大量工作，以及兩種腎臟藥物，即膜特性和狼瘡性腎炎。這兩項研究都被宰相執行得很好，或者被宰相執行得很好。所以請繼續關注，當這個數據公開時，我們一定會談論它。到目前為止，試驗進展順利，我們對 Zai 在這兩個適應症的臨床試驗執行中所表現出的品質感到非常滿意。

Thank you for the question.

謝謝你的提問。

Yaron Werber, TD Cowen.

亞龍·韋伯，TD·考恩。

All right. Thanks, and congrats on a really terrific quarter. Maybe quick question. The myositis go/no go decision is coming up in three indications. You know, we all know that the IM&M passive transfer data was the sort of the best objective support to moving into Phase 3, but there's a lot of biological support for the other two. Maybe just how do you feel about all three indications? Thank you.

好的。謝謝，並祝賀這個季度非常出色。也許是一個快速的問題。肌炎是否繼續的決定將在三個適應症中做出。你知道，我們都知道 IM&M 被動傳輸資料是進入第三階段的最佳客觀支持，但其他兩個也有很多生物支持。也許您對這三個跡像有何看法？謝謝。

Yeah, thank you, Yaron, and thank you for the question. You're right to call out myositis because this is the last data card which we would like to turn before we leave a very productive and successful 2024. An equally strong biology conviction across the tree with a slight preference, indeed, as you call out for IM&M, and I think that it's convincing biology in place for all three of them.

是的，謝謝你，亞龍，也謝謝你提出這個問題。你說的肌炎是對的，因為這是我們在結束富有成效和成功的 2024 年之前想要翻開的最後一張數據卡。事實上，正如你呼籲的 IM&M 一樣，對整個樹的生物學信念也同樣強烈，但略有偏愛，我認為這對他們三個來說都是令人信服的生物學。

I think the fields in myositis is evolving, more and more are people classifying the subsets of myositis, not just based on clinical symptoms, but also serology, i.e. the presence of these pathogenic IgG autoantibodies in the serum of the patient. So I'm positive about all three based on what we know about the biology.

我認為肌炎領域正在不斷發展，越來越多的人對肌炎亞型進行分類，不僅根據臨床症狀，還根據血清學，即患者血清中是否存在這些致病性 IgG 自體抗體。因此，根據我們對生物學的了解，我對這三個方面都持正面態度。

There's always risk associated with the clinical experiment, and that's I think why we make a responsible decision to do that go/no go decision point whilst we are enrolling at risk, of course, the Phase 3 trial. So in practice, you know, what you will see us do is just make stop decisions in case they are warranted. But the Phase 3 is enrolling in all three subsets.

臨床實驗總是存在風險，這就是為什麼我們做出負責任的決定，在我們冒著風險入組（當然是第三階段試驗）的同時，做出進行/不進行的決策點。因此，在實踐中，您將看到我們所做的只是在有必要的情況下做出停止決定。但第三階段正在招募所有三個子集。

Thank you for the question.

謝謝你的提問。

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, and sorry if this was discussed already and we missed it, but I was curious to hear your thoughts on what you're seeing in terms of dosing frequency for the CIDP patients that have started therapy, especially for those that started in July and August. Thanks.

嗨，早安。感謝您提出我們的問題，如果我們已經討論過這個問題而我們錯過了，我們很抱歉，但我很想听聽您對已開始治療的 CIDP 患者的給藥頻率的看法，特別是對於那些從七月和八月開始。謝謝。

Yeah, thanks for the question. So it's a little too early for us to tell, the majority of patients are just getting started on therapy for CIDP. So and what we know is, as I mentioned earlier, that we have the open label extension study coming with the biweekly and the every three weeks dosing. So we really need to see how it plays out with real world utilization as we get more patients on therapy.

是的，謝謝你的提問。因此，現在判斷還為時過早，大多數患者剛開始接受 CIDP 治療。因此，正如我之前提到的，我們所知道的是，我們進行了開放標籤擴展研究，每兩週和每三週給藥一次。因此，隨著更多患者接受治療，我們確實需要看看它在現實世界中的應用效果如何。

Thank you, Karen. And Vikram, I think it's fair to assume in an early launch that typically patients will be dosed according to the label.

謝謝你，凱倫。Vikram，我認為在早期推出時可以公平地假設通常患者將根據標籤服用劑量。

Charles Pitman-King, Barclays.

查爾斯·皮特曼-金，巴克萊銀行。

Thanks very much for taking my questions. Charles Pitman-King from Barclays. And I'll also add my congratulations on the very strong quarter. Just following up on the dosing frequency, I was wondering if you could just reiterate to us your expected or your currently booked price for Hytrulo and CIDP. Originally in the $450,000, I believe was an expected balance between weekly and biweekly. Obviously, given the early launch is meant to be in line with the label, this suggests more weekly, so potentially a higher level of cost booked. So if you just kind of outline what your assumptions behind that are, and how that's progressing, that'd be great. Thank you.

非常感謝您回答我的問題。巴克萊銀行的查爾斯·皮特曼-金。我還要對這個非常強勁的季度表示祝賀。只是跟進給藥頻率，我想知道您是否可以向我們重申您對 Hytrulo 和 CIDP 的預期或當​​前預訂價格。我認為最初的 45 萬美元是每周和每兩週之間的預期平衡。顯然，考慮到早期發布是為了與標籤保持一致，這意味著每週都會發布更多內容，因此預訂的成本可能會更高。因此，如果您只是概述一下您背後的假設是什麼以及進展如何，那就太好了。謝謝。

Thank you. Thank you, Charles. It's Karl here. Yeah, the $450,000 is the number we came out at launch and just as a reminder how we got to that $450,000. It of course, it depends on the number of Hytrulo vials. So the utilization needs to be taken into consideration. So at the moment, we all know the study was done weekly, but in the real world, we will have to see how that play out. We have no new information to talk about. And then of course, you also have to talk about gross to net, but which is part of that $450,000. So I think it's too early to talk about, to give more information now.

謝謝。謝謝你，查爾斯。這是卡爾。是的，45 萬美元是我們在發佈時給出的數字，只是提醒我們如何達到 45 萬美元。當然，這取決於 Hytrulo 瓶子的數量。所以需要考慮利用率。所以目前，我們都知道這項研究是每週進行的，但在現實世界中，我們必須看看結果如何。我們沒有新的資訊可談。當然，你也必須談論總淨值，但這是 45 萬美元的一部分。所以我認為現在談論、提供更多資訊還為時過早。

Samantha Semenkow, Citi.

薩曼莎·塞門科，花旗銀行。

Hi, good morning. Thanks very much for taking the question and let me just add my congratulations on the strong quarter. Just turning back to the pipeline for membranous nephropathy, I guess maybe could you give a little bit of background on what drove the initial interest in this indication? And a little bit more color on your thoughts on the lack of a sufficient signal of this disease. Is there any read through to other indications in development? Thank you.

嗨，早安。非常感謝您提出問題，讓我對強勁的季度表示祝賀。回到膜性腎病變的研發管線，我想您能否介紹一下是什麼推動了人們對該適應症的最初興趣？關於這種疾病缺乏足夠訊號的想法，請多加一點色彩。是否有任何正在開發的其​​他適應症的通讀？謝謝。

Thank you for the question, Samantha. So first of all, there was conviction in MN based on the convincing biology that this is indeed a pathogenic antibody which is mediating the disease biology. We know the identity of the antibody. We know that the title correlates with disease severity and so on.

謝謝你的提問，薩曼莎。首先，基於令人信服的生物學原理，人們確信 MN 確實是一種介導疾病生物學的致病性抗體。我們知道抗體的身份。我們知道標題與疾病的嚴重程度等相關。

So typically, the facts which we like to see in order to build confidence. We were alerted by the DSMB throughout the study that basically there is no real signal here, and they asked us to reconsider the continuation of the study. That's where we decided to take a look at the data and make the decision.

通常，我們喜歡看到事實以建立信心。在整個研究過程中，DSMB 一直提醒我們，這裡基本上沒有真正的訊號，他們要求我們重新考慮是否繼續研究。這就是我們決定查看數據並做出決定的地方。

It comes a bit as a surprise. But that's why you do Phase 2 trials. I mean, you want to further validate the biology before you make a Phase 3 investment. It's actually perfectly serving the purpose. The only thing we can think of at the moment is that -- and that is specific for MN, these patients lose a ton of protein trough the kidneys. So these kidneys are extremely leaky.

這有點令人驚訝。但這就是您進行第二階段試驗的原因。我的意思是，在進行第三階段投資之前，您希望進一步驗證生物學。它實際上完美地達到了目的。目前我們唯一能想到的是——對於 MN 來說，這些患者透過腎臟損失了大量蛋白質。所以這些腎臟的滲漏非常嚴重。

And we're going to double click on the data set when it comes to exposure to the drug. But I would certainly not look at any read through to other indications where this is actually not the case. So stay tuned. We will be communicating more about it later. Thank you.

當涉及到藥物暴露時，我們將雙擊資料集。但我當然不會仔細閱讀其他跡象，但實際上並非如此。所以請繼續關注。稍後我們將對此進行更多溝通。謝謝。

Gavin Clark-Gartner, Evercore ISI.

Gavin Clark-Gartner，Evercore ISI。

Hey guys, congrats on all the progress. I was just wondering for the over 300 CIDP patients that were on treatment at the end of the quarter, are all these reimbursed patients or any of these patients on bridge or free drug supply still awaiting reimbursement approval?

嘿夥計們，祝賀所有的進步。我只是想知道，對於本季末正在接受治療的 300 多名 CIDP 患者，所有這些報銷患者或這些過渡或免費藥物供應的患者是否仍在等待報銷批准？

Gavin, thank you for your question. And they all reimbursed.

加文，謝謝你的問題。而且他們都報銷了。

Thank you.

謝謝。

Suzanne van Voorthuizen, Kempen.

蘇珊娜·範·沃特赫伊森，肯彭。

Hi, team, thanks for taking my question. It relates to pipeline news flow, I understand your guide on timelines when appropriate, but there's still a lot to unpack so to say. So can you frame a bit what we should expect for the coming 12 to 18 months? For example, for VYVGART, would be helpful to understand the sequence. We should expect the registration trials to read out data, and on the other hand, for empa and 119, a number of mid-stage trials have been ongoing for a bit. So yeah, can we expect the data reach ahead -- a year ahead for these pipeline assets? Thank you.

嗨，團隊，感謝您提出我的問題。它與管道新聞流有關，我理解您在適當的時候對時間表的指南，但可以說還有很多東西需要解開。那麼您能否概括一下我們對未來 12 至 18 個月的預期？例如，對於 VYVGART，將有助於理解序列。我們應該期望註冊試驗能夠讀出數據，另一方面，對於 empa 和 119，一些中期試驗已經進行了一段時間。那麼，是的，我們可以預期這些管道資產的數據會提前一年嗎？謝謝。

Yeah. Thank you, Suzanne, and thank you for being with us today. So typically, at the start of the new year, we would announce the clinical calendar for the year. So expect us to show up at JP Morgan with a more granular view on what 2025 will bring in terms of clinical milestones.

是的。謝謝你，蘇珊娜，也謝謝你今天和我們在一起。因此，通常在新年伊始，我們會宣布這一年的臨床日曆。因此，期待我們出現在摩根大通，更詳細地了解 2025 年將在臨床里程碑方面帶來什麼。

You're right to call out, there's going to be a lot of activity. If I do the math right, I think we will have between 5 and 10 Phase 3 trials only, and then of course, a whole slew of Phase 2 work and then a number of IND candidates getting into the clinic.

你喊出來是對的，將會有很多活動。如果我計算正確的話，我認為我們只會進行 5 到 10 項 3 期試驗，當然，還有大量的 2 期工作，然後一些 IND 候選人進入臨床。

So we for the moment focused on ending strong years, focusing on executing the last elements of the plan, including hopefully a strong Q4 and positive [MMN] data. And then on the podium in January, we will be talking to you about a detailed clinical calendar for 2025, okay? So stay tuned. Thank you.

因此，我們目前專注於結束強勁的年份，專注於執行計劃的最後要素，包括希望第四季度強勁和積極的 [MMN] 數據。然後在一月份的講台上，我們將與您討論 2025 年詳細的臨床日曆，好嗎？所以請繼續關注。謝謝。

Joel Beatty, Baird.

喬爾·比蒂，貝爾德。

Hi, congrats on the quarter. For the 300 patients who started in CIDP, were those patients that generally had formal payer policies in place, or were some of those, kind of one-offs without formal policies in place yet?

你好，恭喜本季。對於開始參加 CIDP 的 300 名患者來說，這些患者是否通常都有正式的付款人政策，或者其中一些患者是一次性的，尚未制定正式的政策？

Well, thanks for the question. It's a mix of patients that had policies in place and did not. Generally, for policies in place, their patients are able to get on therapy more quickly. But we have at this point in launch, we have a mix.

嗯，謝謝你的提問。其中既有製定政策的患者，也有沒有製定政策的患者。一般來說，對於適當的政策，患者能夠更快地接受治療。但目前我們已經推出了多種產品。

Joon Lee, Truist Securities.

李俊，Truist 證券公司。

Congrats on the strong quarter and thanks for taking our questions. Really curious to know your latest thoughts on the opportunity for ARGX-121 in IgA nephropathy given, you know, several competitor presentations at ASM last week. How do you think ARGX-121, the sweeping antibody can differentiate versus say those targeting CD28 or [bac and AP]. Thank you.

恭喜本季的強勁表現，並感謝您提出我們的問題。鑑於上週在 ASM 上的幾位競爭對手的演講，我真的很想知道您對 ARGX-121 在 IgA 腎病中的機會的最新想法。您認為 ARGX-121（掃除抗體）與針對 CD28 或標靶抗體的抗體有何不同？[bac 和 AP]。謝謝。

Yeah, thank you, Joon, for the question. I think what we see is very exciting. I mean, IgA nephropathy is a disease which is waiting to be unlocked by innovation, and you basically start to see the first generations of innovation coming into the space. But I think this is going to be a sizable opportunity. It's going to be an opportunity which will require multiple generations of innovation to really get there.

是的，謝謝你，瓊，提出這個問題。我認為我們所看到的非常令人興奮。我的意思是，IgA 腎病是一種等待創新來解鎖的疾病，你基本上開始看到第一代創新進入這個領域。但我認為這將是一個相當大的機會。這將是一個需要多代創新才能真正實現的機會。

I think what we have seen for the moment is encouraging data but clearly room for improvement. And that is I think the promise which is held by ARGX-121. I haven't seen any program which is so selective and precise in eliminating the bad guys and doing that with the speed and the depth of response which is just unparalleled by any of the data we have seen.

我認為我們目前看到的數據令人鼓舞，但顯然還有改進的空間。我認為這就是 ARGX-121 所做出的承諾。我還沒有見過任何程式能夠如此有選擇性和精確地消除壞人，並且其響應速度和深度是我們所見過的任何數據都無法比擬的。

So I think the design of the molecule is strong. Now we need to wait for the clinical experiment and see whether that strength of design translates into superior clinical efficacy. So I'm very excited about this molecule. Remember, it can play in more than one indication. IgA nephropathy is one which speaks to the imagination. But there are, of course, more indications on our table because we typically like to pursue pipelines in the product. Thanks for the question.

所以我認為這個分子的設計很強大。現在我們需要等待臨床實驗，看看這種設計優勢是否能轉化為卓越的臨床療效。所以我對這個分子非常興奮。請記住，它可以在不止一種指示中發揮作用。IgA 腎病是一種令人浮想聯翩的疾病。但當然，我們的桌子上還有更多的跡象，因為我們通常喜歡在產品中追求管道。謝謝你的提問。

Victor Floc'h, BNP Paribas.

維克多·弗洛克，法國巴黎銀行。

Hi, everyone. Thank you for taking my question. I do have a question on the pre-filled syringe opportunity. I mean, we've seen Hytrulo driving your line penetration in MG. So on paper PFS looks like a game changer for patients, but in practice, I was wondering if you are expecting it to be also a significant driver to the [gap] top line. And if I can squeeze just another one, I was wondering if you can give us a bit of visibility on when you will be able to launch the PFS ex-US? Thanks a lot.

大家好。謝謝你回答我的問題。我確實對預充填注射器機會有疑問。我的意思是，我們已經看到 Hytrulo 在 MG 中推動了您的線路滲透。因此，在紙面上，PFS 看起來像是患者的遊戲規則改變者，但在實踐中，我想知道您是否期望它也成為 [差距] 頂線的重要驅動力。如果我可以再擠出一個，我想知道您是否可以向我們透露您何時能夠在美國以外地區推出 PFS？多謝。

Thank you, Victor for the question. I think this is a question for Karen.

謝謝維克多的提問。我認為這是凱倫的問題。

Yeah, absolutely. Look, we're really excited for the April PDUFA date for PFS, and I think what it will do is continue the momentum that we've seen in MG and continue to sustain the launch in CIDP. So it really aligns with the strategy of providing not just the best efficacy and safety but also convenience for patients, particularly with our goal of self-administration, so that we can really compete very well for patients in both of these markets. And it aligns with our strategy of early aligned use in MG as well as broadening the prescriber base. So we're really looking forward to the April PDUFA date.

是的，絕對是。看，我們對 PFS 的 4 月份 PDUFA 日期感到非常興奮，我認為它將繼續我們在 MG 中看到的勢頭，並繼續維持 CIDP 的發布。因此，它確實符合不僅為患者提供最佳療效和安全性，而且為患者提供便利的策略，特別是符合我們自我給藥的目標，以便我們能夠真正在這兩個市場上為患者提供良好的競爭。它符合我們早期聯合使用 MG 以及擴大處方者基礎的策略。所以我們非常期待 4 月份的 PDUFA 日期。

And then do you want to just touch on when we expect to launch PFS ex-US? Second question.

那麼您想談談我們預計何時在美國以外地區推出 PFS 嗎？第二個問題。

Yes. So we have filings ongoing for the PFS ex-US, and they will follow over the coming months and year. Thank you, Beth.

是的。因此，我們正在為美國以外的 PFS 提交申請，這些申請將在未來幾個月和一年內跟進。謝謝你，貝絲。

Xian Deng, UBS.

鄧賢，瑞銀。

Hi. Thank you so much for taking my question. So I have a question on CIDP, please. I understand that 85% 90% of patients are actually coming from IVIg, but just wondering what is a typical patient portrait look like? So just wondering are these patients who really have very poor disease control with IVIg and they're switching? Or do you see actually patients with actually very good disease control, but they're switching for convenience? Thank you very much.

你好。非常感謝您回答我的問題。我有一個關於 CIDP 的問題，請問。我知道 85% 90% 的患者實際上來自 IVIg，但只是想知道典型的患者肖像是什麼樣的？所以我想知道這些患者是否真的透過 IVIg 疾病控制效果非常差並且正在轉換？還是你看到實際上疾病控制得很好的患者，但他們為了方便而改變？非常感謝。

I think the typical phenotype of the patients you would see so early in the launch are actually patients which have a real difficult time tolerating the drug or who are really weakening in between IVIg cycles or actually don't have full function. So I think this would be the two main drivers for these early patients coming on drug and the convenience of dosing does not go unnoticed.

我認為您在推出初期看到的患者的典型表型實際上是那些很難耐受藥物的患者，或者在 IVIg 週期之間真正虛弱的患者，或者實際上沒有完全功能的患者。因此，我認為這將是這些早期患者接受藥物治療的兩個主要驅動因素，並且給藥的便利性不會被忽視。

I mean, you need to imagine that a lot of CIDP patients who would be on IVIg would spend 1 to 2 days a month in an IV infusion chair, typically spending the bulk of the day in that chair. And then of course, a 30 to 90 second subcu-injection is very compelling. But I would say, right Karen, it's fair to say that it's mainly driven by insufficient response to Ig and or tolerability issues.

我的意思是，你需要想像一下，許多接受 IVIg 的 CIDP 患者每月有 1 到 2 天坐在 IV 輸液椅上，通常一天的大部分時間都坐在那張椅子上。當然，30 到 90 秒的皮下注射也非常引人注目。但我想說的是，凱倫，公平地說，這主要是由於對 Ig 和/或耐受性問題的反應不足所致。

Absolutely, and it's exactly what we would expect at this moment in launch.

當然，這正是我們在發佈時所期望的。

Thanks for the question.

謝謝你的提問。

Leland Gershell, Oppenheimer.

利蘭·格謝爾，奧本海默。

Okay, thanks, and my congrats on the quarter as well. Just teeing off the earlier comments on the decision to discontinue in MN. Is it fair to say that that was based primarily on biomarkers versus having to need to see a lack of clinical response? And maybe more broadly as we think about focusing R&D into the future, is it fair to say that argenx comply a fairly high bar with respect to go/no go decisions, given the breadth of opportunity, and as you look to achieve consistent profitability? Thanks.

好的，謝謝，我也恭喜本季。只是發表之前關於在明尼蘇達州停止生產的決定的評論。可以公平地說，這主要基於生物標記，而不是必須看到缺乏臨床反應嗎？也許更廣泛地說，當我們考慮將研發重點放在未來時，考慮到機會的廣泛性以及您希望實現一致的盈利能力，可以公平地說argenx 在進行/不進行決策方面遵守相當高的標準嗎？謝謝。

No, I like that question. I think your second point is a very important one. I mean, we're looking for indications where we can have a truly transformative effect, not a marginal effect but an incremental effect. It needs to be a game changing effect. And that's where we will basically put the mission of the company to work. And here I think it's very well known in MN that, you know, there are a couple of protein biomarkers in the urine which would basically be broadly accepted as proxies for clinical efficacy. So I was not close to the data. There was an (inaudible) in the company chaired by our Chief Medical Officer who was close to the data, but I believe it was based on an eGFR or a proteinuria marker. Thanks for the question.

不，我喜歡這個問題。我認為你的第二點非常重要。我的意思是，我們正在尋找能夠產生真正變革性效果的跡象，不是邊際效應，而是漸進效應。它需要具有改變遊戲規則的效果。這就是我們基本上將公司使命付諸實踐的地方。我認為明尼蘇達州眾所周知，尿液中存在一些蛋白質生物標記物，它們基本上被廣泛接受為臨床療效的代表。所以我對數據並不了解。公司中有一位（聽不清楚）由我們的首席醫療官擔任主席，他接近這些數據，但我相信它是基於 eGFR 或蛋白尿標記物。謝謝你的提問。

Andy Chen, Wolfe Research.

安迪陳，沃爾夫研究中心。

Hey, good morning. Thank you for taking the question. On your pipeline for both 213 and the IgA drug, can you remind us if the two drugs have been optimized into subcu formulation because the data so far looks like it's IV? And then also if you can quickly provide an update for the auto injector status, if you can. Thank you.

嘿，早安。感謝您提出問題。在你們的 213 和 IgA 藥物的研發管線中，你能否提醒我們這兩種藥物是否已優化為 subcu 製劑，因為到目前為止的數據看起來像是靜脈注射？如果可以的話，您是否可以快速提供自動注射器狀態的更新。謝謝。

Andy, thank you for the question. Obviously, the drugs we design are carrying the secure possibilities in them. So yes, we do have the subcutaneous product presentation in the plan for both molecules, and we strongly believe we can get there.

安迪，謝謝你的提問。顯然，我們設計的藥物蘊藏著安全的可能性。所以，是的，我們確實在這兩種分子的計劃中都有皮下產品介紹，我們堅信我們可以實現這一目標。

From an auto injector point of view, it's a bit premature for us to be public on timelines. We are fully focused on PFS. But I think you're right to call out that we are working in the background already very hard on that auto injectors where we said already a while ago that, you know, we have passed the prototyping phase, we are into the industrialization phase. So that that project is going forward at full speed. But we will be communicating about timelines when we get closer to a final product. Thanks for the question.

從自動注射器的角度來看，我們現在公開時間表還為時過早。我們完全專注於 PFS。但我認為你指出我們已經在自動注射器的後台非常努力地工作是正確的，我們不久前已經說過，你知道，我們已經通過了原型設計階段，我們正在進入工業化階段。因此該項目正在全速推進。但當我們接近最終產品時，我們將就時間表進行溝通。謝謝你的提問。

Rajan Sharma, Goldman Sachs.

拉詹·夏爾馬，高盛。

Hi, and thanks for taking the question. Just on the CIDP launch, obviously, you kind of talked to a number of patients on therapy after the first quarter relative to myasthenia gravis. Just thinking about kind of trajectory from here and run rate, would you kind of caution against utilizing that same run rate that we saw for VYVGART in myasthenia for CIDP?

您好，感謝您提出問題。顯然，就在 CIDP 啟動時，您與一些在第一季後接受重症肌無力治療的患者進行了交談。只是考慮這裡的軌跡和運行率，您是否會謹慎對待使用我們在肌無力中為 CIDP 看到的 VYVGART 相同的運行率？

Yeah, thanks for the question, and we're asking the same question. I think it's a good one. Since we're pleased with where we're at. But as I said earlier, I think it's important that we don't get ahead of ourselves.

是的，謝謝你的問題，我們也在問同樣的問題。我認為這是一件好事。因為我們對目前的處境感到滿意。但正如我之前所說，我認為重要的是我們不要超前。

The market dynamics in CIDP are quite different than MG. So you'll recall it's a switch market. Whereas MG was an add on market, the competitive set is different, the patients are different and also our data is different. So look, in particular, what I think is important to take into account is that 67% response rate in ADHERE. Let's see how that plays out in the real world. And it's still early days in terms of the competition and seeing VYVGART out only for one quarter, let's also see what the response is there.

CIDP 的市場動態與 MG 有很大不同。所以你會記得這是一個開關市場。雖然 MG 是市場上的附加產品，但競爭環境不同，患者不同，我們的數據也不同。因此，我認為特別需要考慮的是 ADHERE 中 67% 的回覆率。讓我們看看這在現實世界中是如何發生的。就比賽而言，現在還處於早期階段，VYVGART 只出局了四分之一，讓我們看看大家的反應如何。

Thomas Smith, Leerink Partners.

托馬斯史密斯，Leerink 合夥人。

Hey, good morning. Thanks for taking our questions, and let me add my congrats on the strong quarter. We saw you had a very strong presence at the recent AANEM meeting and there were a ton of also quite a number of competitor data sets there in MG. I was wondering if you could just comment on how you're thinking about some of these emerging targets like CD19 and MG.

嘿，早安。感謝您提出我們的問題，並讓我對強勁的季度表示祝賀。我們看到您在最近的 AANEM 會議上表現非常出色，並且 MG 中也有大量競爭對手的資料集。我想知道您是否可以評論一下您對 CD19 和 MG 等新興目標的看法。

And then separately, I wanted to ask about thyroid eye disease. It sounds like the Phase 3 is progressing as planned. But I was wondering if you could comment on enrollment. Just remind us what your expectations are for the Phase 3 topline readout. Thanks very much.

另外，我想問甲狀腺眼疾。聽起來第三階段正在按計劃進行。但我想知道你是否可以對入學發表評論。請提醒我們您對第三階段的頂線讀數有何期望。非常感謝。

Yes, Thomas, thank you for the question. I think you're right to call out our leadership at AANEM. I'm very proud of the continuous stream of data which we continue to produce as a market leader and the first in class agent. We continue to see in the real world that we have got a very high bar.

是的，托馬斯，謝謝你的提問。我認為您對 AANEM 領導力的評價是正確的。我對我們作為市場領導者和一流代理商不斷產生的持續數據流感到非常自豪。我們在現實世界中繼續看到我們的標準非常高。

If we talk about more than 50% MSC, if we talk about more than 80,000 patient years' worth of safety data and then the absence of REMS programs, vaccination requirements, [lapse], no albumin issue, and then of course, from a convenience point of view, we're the only one showing such a spectrum of dosing possibilities, which really allows you to tailor to the individual needs of the patient. So we've put the bar high.

如果我們談論超過 50% 的 MSC，如果我們談論超過 80,000 個患者年的安全數據，然後缺乏 REMS 計劃、疫苗接種要求、[失效]、沒有白蛋白問題，然後當然，來自從便利的角度來看，我們是唯一一家展示如此一系列劑量可能性的公司，這確實可以讓您根據患者的個人需求進行客製化。所以我們把標準設得很高。

We see competitors coming into the space, but I haven't seen any particular data set which is actually coming close to the high bar, which we have set. We also continue to evolve and lead the space, you know, by moving upstream in the treatment paradigm. I think we have shown infective steroid tapering data.

我們看到競爭對手進入這個領域，但我還沒有看到任何特定的資料集實際上接近我們設定的高標準。你知道，我們也透過向治療範式的上游發展，繼續發展並引領這個領域。我認為我們已經展示了感染性類固醇逐漸減少的數據。

I'm very proud of the fact that after one year, 25% of patients are off steroids. That's a big deal. And more than 40% are at 5 milligram or lower a day. That again is a paradigm shift in the treatment of myasthenia. So our conclusion of all the new data coming to the spaces, we've put the bar very high, and we continue to shift that bar. Thank you for the question.

一年後，25% 的患者不再使用類固醇，對此我感到非常自豪。這是一件大事。超過 40% 的人每天的攝取量為 5 毫克或更低。這又是肌無力治療模式的轉變。因此，我們對所有進入該領域的新數據得出的結論是，我們將標準設置得非常高，並且我們將繼續改變該標準。謝謝你的提問。

Yatin Suneja, Guggenheim.

亞汀‧蘇內賈 (Yatin Suneja)，古根漢。

Hi, this is [Thelma] for Yatin. Congrats for the quarter, and thanks for taking our question. So for the Sjogren program, can you please clarify if you have reached alignment with the FDA on the pivotal program? And is there any color you can share on trial design and statistical plan? Thank you.

大家好，我是雅汀的[Thelma]。恭喜本季度，感謝您提出我們的問題。那麼對於 Sjogren 計劃，您能否澄清一下您是否已與 FDA 就關鍵計劃達成協議？您在試驗設計和統計計劃方面有什麼可以分享的嗎？謝謝。

Thank you for the question. I think we're on track to start the study before the end of the year. I think that's the key message for our investment audience. We did have the appropriate FDA interactions. So we were aligned and we have calibrated study design and endpoints. So we're actually in operationalization [modes] and we are on track to start the study before the end of the year.

謝謝你的提問。我認為我們預計在今年年底前開始這項研究。我認為這是向我們的投資受眾傳遞的關鍵訊息。我們確實與 FDA 進行了適當的互動。因此，我們達成了一致，並校準了研究設計和終點。所以我們實際上處於可操作[模式]，並且我們預計在今年年底之前開始研究。

By the way, on TED, there's a question I forgot to answer a minute ago. You're right. Everything is on plan and on track, but too early, of course to talk about completion dates for both TED and Sjogren's. Thank you for the question.

順便說一句，在 TED 上，有一個問題我剛剛忘了回答。你說得對。一切都在計劃之中併步入正軌，但談論 TED 和 Sjogren 的完成日期當然還為時過早。謝謝你的提問。

Simon Baker, Redburn Atlantic. Simon, your line is open.

西蒙貝克，雷德本大西洋月刊。西蒙，您的線路已開通。

Douglas Tsao, H. C. Wainwright.

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

Hi, good morning. Thanks for taking the questions. I'm just curious on the commentary regarding the CIDP launch leading to new prescribers in MG. I'm just curious in terms of the profile for some of those new prescribers who are now starting. Were they just not -- not have big practices in MG? Were they just not familiar with the product? And just do you have any color in terms of why, just given the such early success you had in MG, why this was the catalyst for them? Thank you very much, and congrats on the results.

嗨，早安。感謝您提出問題。我只是對 CIDP 的推出導致 MG 出現新處方者的評論感到好奇。我只是對一些現在開始的新處方醫生的概況感到好奇。難道他們只是沒有在 MG 方面進行大規模實踐嗎？他們只是不熟悉該產品嗎？考慮到您在 MG 取得的如此早期的成功，您有什麼看法可以解釋為什麼這是他們的催化劑嗎？非常感謝，並對結果表示祝賀。

Yeah. Thank you for the question. Look, I would say you will recall that when we were characterizing the CIDP opportunity, there's a lot more CIDP patients that are out with community neurologists than there are MG patients. However, there are MG patients also with community neurologists.

是的。謝謝你的提問。你看，我想說你會記得，當我們描述 CIDP 機會時，與社區神經科醫生就診的 CIDP 患者比 MG 患者多得多。然而，也有 MG 患者接受社區神經科醫師的治療。

So we broadened our target list in line with the CIDP launch. And that is reflected by the field force expansion that we also invested in in preparation for the CIDP launch. So with that field expansion and the target expansion, we're reaching new neurologists, some of these are new to VYVGART, and some of them as you heard have started with the CIDP and then expanded their prescribing to MG.

因此，我們根據 CIDP 的啟動擴大了目標清單。這反映在我們為準備 CIDP 啟動而投資的現場人員擴張上。因此，隨著領域的擴展和目標的擴展，我們正在接觸新的神經科醫生，其中一些是VYVGART 的新手，正如您所聽到的，其中一些已經開始使用CIDP，然後將他們的處方擴展到MG 。

David Seynnaeve, Petercam.

大衛‧塞納維，彼得卡姆。

Hey, good afternoon. Regarding the CIDP trial with empa, it's related to one of the earlier questions asked. Can you say a few words on your decision to go immediately into a registrational study? What your conviction is based on? And maybe what exactly you expect from the outcome of this study, keeping in mind the data you've generated with (inaudible). Thank you.

嘿，下午好。關於 CIDP 與 empa 的試驗，它與先前提出的問題之一有關。您能談談您立即進行註冊研究的決定嗎？你的信念是基於什麼？也許您對這項研究的結果有何期望，請記住您產生的數據（聽不清楚）。謝謝。

Thank you, David for the question. Look, there remains work to be done in CIDP. No one has shown a higher response rate than we did with VYVGART and in the trial. If you're fair, there's actually a 70% response rate in stage A. Key question is what happened to the 30% patients who did not respond in that first 12 weeks?

謝謝大衛提出問題。看，CIDP 仍有工作要做。沒有人比我們在 VYVGART 和試驗中表現出更高的反應率。公平地說，A 階段實際上有 70% 的緩解率。

We have also seen the data from Sanofi's program underlining the role of complements, clearly complement being recruited by the disease-causing antibodies.

我們也看到賽諾菲計畫的數據強調了補體的作用，顯然補體是由致病抗體招募的。

And based on the inhouse ex-vivo data, the data from the field, we think it is warranted to launch empa straight into Phase 3, especially given the regain of function data we have seen in MMN, we think of MMN as the little sister indication of CIDP. So if you look at the totality of data, we did not want to lose any time in terms of Phase 2 work. We think it's warranted to go forward into Phase 3. So we're very excited about the opportunity and remember there's a ton of work to be done still in CIDP. Thank you for the question.

基於內部的體外數據和現場數據，我們認為有必要將 empa 直接啟動到第 3 階段，特別是考慮到我們在 MMN 中看到的功能數據的恢復，我們認為 MMN 是它的小妹妹CIDP 的指示。因此，如果您查看全部數據，我們不想在第二階段的工作上浪費任何時間。我們認為有必要進入第三階段。因此，我們對這個機會感到非常興奮，並記住 CIDP 仍有大量工作要做。謝謝你的提問。

Emmanuel Papadakis, Deutsche Bank.

伊曼紐爾‧帕帕達基斯，德意志銀行。

Thank you for taking the question. Maybe just follow on CIDP launch. Given you're starting to get a bit of feel for the market in the US, perhaps you could give us your best current estimate of the absolute number of CIDP patients in the US that are either refractory or unsatisfied from a convenience perspective with their current Ig treatment. And maybe just in a quick addition, reimbursement timelines in Europe CIDP. Could that start to contribute next year or is that more of a 2026 then? Thank you.

感謝您提出問題。也許只需關注 CIDP 的發布即可。鑑於您開始對美國市場有了一定的了解，也許您可以向我們提供您目前對美國CIDP 患者絕對數量的最佳估計，這些患者要么是難治性的，要么是從方便的角度來看對目前的情況不滿意。也許只是快速補充一下歐洲 CIDP 的報銷時間表。這會在明年開始發揮作用嗎？謝謝。

Yes, thanks for the two questions. So we have sized the addressable market for CIDP as 12,000 patients that we believe that are on therapy but have residual symptoms or are unsatisfied with their current therapy. So that's the addressable market that we see. In terms of ex-US, the CIDP filings are ongoing, obviously, pricing and reimbursement takes a little bit longer. But as soon as we have those approvals in hand, we'll be working diligently and quickly to get access for patients across the globe.

是的，謝謝你的兩個問題。因此，我們將 CIDP 的目標市場定為 12,000 名患者，我們認為這些患者正在接受治療，但有殘留症狀或對目前治療不滿意。這就是我們看到的潛在市場。就美國以外地區而言，CIDP 備案正在進行中，顯然定價和報銷需要更長的時間。但一旦獲得這些批准，我們就會勤奮、迅速地為全球患者提供使用服務。

Simon Baker, Redburn Atlantic.f

西蒙貝克，Redburn Atlantic.f

Thank you very much for taking my question twice. A question on VYVGART, and I wonder if you could give us any qualitative commentary on rebating and formula negotiation trends into 2025. And how's the addition of CIDP as an indication affected those negotiations meaningfully at all at this stage? Thanks so much.

非常感謝您兩次回答我的問題。關於 VYVGART 的問題，我想知道您是否能為我們提供有關 2025 年回扣和公式談判趨勢的定性評論。CIDP 的加入對現階段的談判有何影響？非常感謝。

Simon, thank you. It's Karl here. Thank you for the question. What I can say is where we are today, gross to net continues to be around 12%. That's public, that's in our financials. We don't see a deterioration there.

西蒙，謝謝你。這是卡爾。謝謝你的提問。我能說的是我們今天的處境，毛淨值仍然在 12% 左右。這是公開的，在我們的財務中。我們沒有看到那裡出現惡化。

I think that's what you can expect for the rest of the year. For 2025, and with prefilled syringe, all of those variables, I think it's a little bit too early to talk about that, and that can be part of a later discussion. Thank you for the question.

我認為這就是你對今年剩餘時間的預期。對於 2025 年，對於預充式註射器，所有這些變量，我認為現在談論這個還為時過早，這可以成為以後討論的一部分。謝謝你的提問。

And this concludes our question-and-answer session and does conclude today's conference call. We thank you for your participation today. You may now disconnect.

我們的問答環節到此結束，今天的電話會議也到此結束。我們感謝您今天的參與。您現在可以斷開連線。