argenx SE (ARGX) 2025 Q2 法說會逐字稿

Good morning. My name is Rob, and I will be your conference operator today. I would like to welcome everyone to the call. (Operator Instructions) I'd now like to introduce Beth DelGiacco, Vice President, Corporate Communications and Investor Relations. You may begin your call.

早安.我叫羅布，今天我將擔任您的會議主持人。我歡迎大家參加電話會議。（操作員指示）現在我想介紹企業傳播與投資人關係副總裁 Beth DelGiacco。您可以開始通話了。

Thank you. A press release was issued earlier today with our half-year 2025 financial results and second-quarter business update. This can be found on our website along with the presentation for today's webcast. Before we begin, on slide 2, I'd like to remind you that forward-looking statements may be presented during this call. These may include statements about our future expectations, clinical developments, regulatory timelines, the potential success of our product candidates, financial projections and upcoming milestones.

謝謝。今天稍早發布了一份新聞稿，其中包含我們 2025 年上半年的財務業績和第二季的業務更新。您可在我們的網站上找到該內容以及今天的網路廣播的簡報。在我們開始之前，在第 2 張投影片上，我想提醒您，本次電話會議期間可能會出現前瞻性陳述。這些可能包括有關我們未來預期、臨床發展、監管時間表、我們候選產品的潛在成功、財務預測和即將到來的里程碑的聲明。

Actual results may differ materially from those indicated by these statements. Argenx is not under any obligation to update statements regarding the future or to conform those statements in relation to actual results unless required by law. 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. Luc Truyen, Chief Medical Officer, will be available during the Q&A.

實際結果可能與這些聲明所示的結果有重大差異。除非法律要求，否則 Argenx 沒有義務更新有關未來的聲明或使這些聲明與實際結果相符。今天與我一起參加電話會議的還有執行長 Tim Van Hauwermeiren、財務長 Karl Gubitz 和營運長 Karen Massey。首席醫療官 Luc Truyen 將在問答環節出席。

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 number 3. Vision 2030 is our road map for long-term value creation, and I'm proud to say that we are exactly where we set out to be. Over the past 12 months, VYVGART has achieved exceptional year-over-year growth of 97% across all of its approved indications. We've also initiated multiple registrational trials in large market opportunities like Sjögren's, myositis and TED and advanced four new molecules into our pipeline.

謝謝你，貝絲，歡迎大家。我將從第三張投影片開始。2030願景是我們長期價值創造的路線圖，我很自豪地說，我們正朝著既定目標前進。在過去的 12 個月中，VYVGART 在其所有核准適應症上都實現了 97% 的同比增長。我們也在乾燥症、肌炎和 TED 等大型市場機會中啟動了多項註冊試驗，並將四種新分子推進到我們的產品線中。

This sets us up to create significant growth with 10 labeled indications and a robust late-stage pipeline by 2030. While we are building for the long term, we are also delivering today. 15,000 patients globally are now being treated with VYVGART, including 2,500 CIDP patients just one year into the launch. We are seeing growth across all indications, formulations and regions. And as expected, our prefilled syringe is already driving new patient starts and new prescriber demand.

這使得我們能夠在 2030 年前憑藉 10 個已標記適應症和強大的後期研發管線實現顯著成長。我們在進行長期建設的同時，也致力於實現目標。目前，全球有 15,000 名患者正在接受 VYVGART 治療，其中包括推出僅一年後的 2,500 名 CIDP 患者。我們看到所有適應症、配方和地區都在成長。正如預期的那樣，我們的預充式註射器已經推動了新患者的開始使用和新處方者的需求。

This momentum is a direct result of our team's extraordinary execution, and I want to thank them for advancing innovation that truly matters to patients. Karen will share more later in the call on our commercial performance and the path to reach 50,000 patients by 2030. Where I want to focus today is on the opportunity we have to expand VYVGART's broad potential and advance our pipeline of first-in-class assets.

這種勢頭直接得益於我們團隊的非凡執行力，我要感謝他們推動對患者真正重要的創新。Karen 稍後將在電話會議上分享更多有關我們的商業表現以及到 2030 年覆蓋 50,000 名患者的計劃。今天我想重點談談我們擴大 VYVGART 的廣泛潛力和推進我們一流資產管道的機會。

Slide 4. We have the opportunity to create significant value in the near term with our three Phase 3 pipeline assets. We are building momentum with efgartigimod in therapeutic areas beyond neurology. We presented our Phase 2 proof-of-concept results in myositis and Sjögren's for the first time at EULAR and the reception from the rheumatology community reminds me of the early enthusiasm we saw from neurologists when we first unveiled our MG data.

幻燈片 4。我們有機會利用我們的三期管道資產在短期內創造巨大的價值。我們正在利用 efgartigimod 在神經病學以外的治療領域取得進展。我們首次在歐洲風濕病研究年會 (EULAR) 上展示了針對肌炎和乾燥綜合徵的 2 期概念驗證結果，風濕病學界的反響讓我想起了我們首次公佈 MG 數據時神經病學家所表現出的熱情。

Rheumatologists are beginning to see what a targeted approach like efgartigimod could mean for their patients, raising the treatment bar beyond symptom management to a sustained functional improvement. In the myositis study, efgartigimod delivered significant improvement in muscle strength as measured by the TIS, which clinically mirrors what we saw preclinically in our mouse-specific transfer models.

風濕病學家開始認識到像 efgartigimod 這樣的針對性治療方法對他們的患者意味著什麼，將治療標準從症狀管理提升到持續的功能改善。在肌肉炎研究中，efgartigimod 顯著提高了肌肉強度（以 TIS 測量），這在臨床上反映了我們在小鼠特異性轉移模型中臨床前所看到的情況。

In the Sjögren's study, we observed meaningful improvement in systemic disease activity. Empasiprubart is also advancing in two registrational head-to-head studies versus IVIg in MMN and CIDP. Our decision to run head-to-head studies illustrates our conviction that empasiprubart has the potential to disrupt these markets. The Phase 2 ADA results in MMN support this and recently gained significant attention from treating neurologists at PNS in May.

在乾燥症的研究中，我們觀察到全身性疾病活動有了顯著改善。Empasiprubart 在兩項針對 MMN 和 CIDP 的 IVIg 註冊頭對頭研究中也取得了進展。我們決定進行面對面的研究，這表明我們堅信 empasiprubart 有可能顛覆這些市場。MMN 的第 2 階段 ADA 結果支持這一點，並且最近在 5 月引起了 PNS 治療神經科醫生的極大關注。

The data point that resonated most comes from the Patient Global Impression of Change Scale, where over 94% of treated patients felt better on empa than their best on IVIg, indicating empa could provide a transformative benefit. For CIDP, we are seeing a significant demand from the community from VYVGART Hytrulo, indicating that there is still a need for more innovation.

最能引起共鳴的數據點來自患者整體變化印象量表，其中超過 94% 的接受治療的患者使用 empa 後感覺比使用 IVIg 後感覺更好，這表明 empa 可以帶來變革性的好處。對於 CIDP，我們看到社群對 VYVGART Hytrulo 的需求很大，這表明仍需要更多創新。

We're committed to making the broadest impact by advancing two distinct mechanisms of action with VYVGART and empasiprubart. ARGX-119 is our third and most recent molecule to enter a registrational study following positive proof of biology data in CMS. The discovery and development of ARGX-119 exemplifies our innovation model well. We collaborated with the world's leading experts to design an antibody that activates MuSK in a way that stabilizes and potentially enhances the neuromuscular junction.

我們致力於透過推動 VYVGART 和 empasiprubart 兩種不同的作用機制來產生最廣泛的影響。ARGX-119 是我們第三個也是最近一個在 CMS 中獲得生物學數據積極證明後進入註冊研究的分子。ARGX-119的發現和開發很好地體現了我們的創新模式。我們與世界頂尖專家合作設計了一種能夠激活 MuSK 的抗體，可以穩定並潛在地增強神經肌肉接頭。

In our 16-patient Phase 1 CMS study, we observed consistent functional improvements across multiple endpoints. And this is just the beginning for ARGX-119. We have identified several opportunities across neurology for this agonistic antibody.

在我們的 16 名患者第 1 期 CMS 研究中，我們觀察到多個終點的一致功能改善。而這對 ARGX-119 來說只是一個開始。我們已經在神經病學領域發現了這種激動劑抗體的幾種應用機會。

Slide 5. Our immunology innovation platform continues to be a powerful engine for long-term growth. We are rapidly advancing four new molecules, including our IL-6 inhibitor, a second FcRn blocker and an IgA targeting antibody, all of which are now in Phase 1 studies. These programs are part of our broader portfolio of over 20 active IIP programs, each targeting areas of high unmet need. Our approach starts with identifying novel first-in-class immunology targets and building molecules that address them.

幻燈片 5。我們的免疫學創新平台持續成為長期成長的強大引擎。我們正在快速推進四種新分子，包括我們的 IL-6 抑制劑、第二種 FcRn 阻斷劑和一種 IgA 靶向抗體，所有這些現在都處於第一階段研究中。這些項目是我們更廣泛的超過 20 個活躍的 IIP 項目組合的一部分，每個項目都針對未滿足需求較高的領域。我們的方法首先是確定新的一流免疫學目標並建立針對這些目標的分子。

As part of our investment in our IIP, we are also expanding our toolbox to optimize our molecules to be best-in-class. We recently announced a collaboration with Unnatural Products to gain access to their AI-driven microcycle discovery platform, enabling the development of potent, selective and orally available peptides against targets that we select. This collaboration both expands our capabilities beyond antibodies and reinforces our commitment to continue to innovate on the patient experience.

作為對 IIP 投資的一部分，我們也正在擴展我們的工具箱，以優化我們的分子，使其達到同類最佳水平。我們最近宣布與 Unnatural Products 合作，以獲得其人工智慧驅動的微循環發現平台，從而能夠針對我們選擇的目標開發強效、選擇性和口服勝肽。此次合作不僅擴展了我們在抗體之外的能力，也加強了我們持續創新患者體驗的承諾。

I will now turn the call over to Karl to discuss our strong financial position, which remains a key lever for us in achieving our long-term growth vision to scale efficiently and prioritize our investment in innovation.

現在，我將把電話轉給卡爾，討論我們強大的財務狀況，這仍然是我們實現長期成長願景的關鍵槓桿，可以有效擴大規模並優先考慮對創新的投資。

Thank you, Tim. Slide 6. The second quarter 2025 financial results are detailed in this morning's press release. Total operating income in the second quarter was $967 million. This reflects $949 million in product net sales and $19 million in other operating income.

謝謝你，提姆。幻燈片 6。2025 年第二季的財務業績已在今天上午的新聞稿中詳細說明。第二季總營業收入為9.67億美元。這反映了 9.49 億美元的產品淨銷售額和 1,900 萬美元的其他營業收入。

We are very proud of a 97% growth we have been able to deliver since this time last year, representing the significant unmet need that exists in MG and CIDP and the transformative outcomes VYVGART can offer to patients.

我們為自去年同期以來實現的 97% 的成長感到非常自豪，這代表了 MG 和 CIDP 中存在的巨大未滿足需求以及 VYVGART 可以為患者提供的變革性成果。

On a quarter-over-quarter basis, we delivered 19% or $158 million in product net sales growth in the second quarter compared to first quarter of this year. If you look at the breakdown by region, product net sales were $802 million in the US, $52 million in Japan, $83 million across our rest of world markets, including Europe, Canada and our partner markets and $12 million for product supply to Zai Lab in China.

與今年第一季相比，我們第二季的產品淨銷售額較上季成長 19%，即 1.58 億美元。若按地區細分，產品淨銷售額在美國為 8.02 億美元，在日本為 5,200 萬美元，在包括歐洲、加拿大和我們的合作夥伴市場在內的世界其他市場為 8,300 萬美元，向中國再鼎醫藥供應產品為 1,200 萬美元。

We are happy to share that all global markets grew in the second quarter with the exception of our supply to China, which you'll remember is not reflective of demand and depends on when we ship within the quarter. We continue to expand our patient reach in our non-US markets and the overall contribution of these regions now represents more than 15% of our global product net sales.

我們很高興地告訴大家，除了對中國的供應外，第二季度所有全球市場都實現了增長，但您會記得，中國的供應並不反映需求，而是取決於我們在本季度的發貨時間。我們持續擴大美國以外市場的患者覆蓋範圍，這些地區的整體貢獻目前占我們全球產品淨銷售額的 15% 以上。

In the US specifically, we delivered 18% quarter-over-quarter growth, which reflects strong growth in both gMG and CIDP across all three presentations, VYVGART IV, subcu vial and the subcu prefilled syringe. We made the investment to move quickly with PFS because we know it will be a long-term growth enabler for all current and future indications. We are already seeing this play out in the near term and PFS has increased patient demand for VYVGART one quarter into its launch.

具體來說，在美國，我們實現了 18% 的環比增長，這反映了三種劑型（VYVGART IV、皮下小瓶和皮下預充式註射器）的 gMG 和 CIDP 均實現了強勁增長。我們投資 PFS 並迅速採取行動，因為我們知道它將成為所有當前和未來適應症的長期成長動力。我們已經看到這一趨勢在短期內顯現，而 PFS 在 VYVGART 推出一個季度後就增加了患者對它的需求。

With the introduction of PFS and the changing dynamics associated with Medicare Part D redesign, there was an increase in gross to net, which we anticipated. Gross to net went from 12% at the end of 2024 to approximately 20% at the end of the second quarter. Importantly, the net revenue contribution for a gMG patient and a CIDP patient continues to be consistent with our previous guidance.

隨著 PFS 的引入以及與 Medicare Part D 重新設計相關的動態變化，總額與淨額有所增加，這是我們預料到的。毛利率與淨利率比率從 2024 年底的 12% 上升至第二季末的約 20%。重要的是，gMG 患者和 CIDP 患者的淨收入貢獻繼續與我們先前的指導保持一致。

This means that going forward, even with the increased gross to net adjustments, growth will be driven by our ability to broaden our patient reach within the MG and CIDP markets and into new patient populations, PFS will help us to achieve this growth.

這意味著，即使總收入與淨收入的調整增加，未來成長仍將取決於我們在 MG 和 CIDP 市場中擴大患者覆蓋範圍以及拓展新患者群體的能力，而 PFS 將幫助我們實現這一增長。

Next slide. Total operating expenses in the second quarter are $766 million, an increase of $98 million compared with Q1. This includes a $49 million increase in SG&A and a $19 million increase in R&D, all of which reflects our commitment to deliver on our innovation mission in a disciplined way. Cost of sales for the quarter is $111 million, which brings our year-to-date gross margin to 11%.

下一張投影片。第二季總營運費用為 7.66 億美元，較第一季增加 9,800 萬美元。其中包括 4,900 萬美元的銷售、一般及行政費用增加和 1,900 萬美元的研發費用增加，所有這些都反映了我們以嚴謹的方式履行創新使命的承諾。本季銷售成本為 1.11 億美元，這使我們年初至今的毛利率達到 11%。

We continue to make important investments into our global supply chain. Our expansion strategy includes our commitment to manufacture in a region for that region and specifically to grow our capacity in the US R&D and SG&A expenses for Q2 were $328 million and $325 million, respectively, leading to an operating profit of $201 million for the period.

我們持續對全球供應鏈進行重要投資。我們的擴張策略包括致力於在某個地區為該地區進行生產，特別是擴大我們在美國的產能。第二季的研發和銷售、一般及行政費用分別為 3.28 億美元和 3.25 億美元，導致當期營業利潤為 2.01 億美元。

The quarterly financial income is $38 million, and we recorded $49 million of exchange gains, mainly resulting from our non-US dollar-denominated cash balances. The year-to-date effective tax rate is 15%. After tax, the profit for the quarter is $245 million and for the year-to-date is $415 million.

季度財務收入為 3,800 萬美元，我們記錄了 4,900 萬美元的匯兌收益，主要來自我們的非美元計價現金餘額。年初至今的有效稅率為15%。稅後，本季利潤為 2.45 億美元，年初至今利潤為 4.15 億美元。

Our cash balance at the end of the quarter, represented by cash, cash equivalents and current financial assets, is $3.9 billion. This is up from $3.4 billion as of the beginning of the year, mainly driven by net cash flow from operating activities of $0.4 billion for the first half of the year.

本季末的現金餘額（包括現金、現金等價物和流動金融資產）為 39 億美元。這一數字高於年初的 34 億美元，主要得益於上半年 4 億美元的營運活動淨現金流。

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

現在我將把電話轉給凱倫，她將提供有關商業方面的詳細資訊。

Thank you, Karl. Slide 8. At argenx, everything we do begins with a deep commitment to understanding and meeting patient needs. VYVGART continues to be a transformative medicine, raising the bar for patients of what they can achieve from their treatment.

謝謝你，卡爾。幻燈片 8。在 argenx，我們所做的一切都始於對理解和滿足患者需求的深切承諾。VYVGART 繼續成為一種變革性的藥物，提高了患者從治療中可以獲得的效果的標準。

In MG, we have the highest rate of minimum symptom expression across any treatment. And in CIDP, we're seeing real-world results that mirror the ADHERE data, including around functional improvement. This value proposition for patients supports our position as the leading branded biologic in MG in an increasingly competitive environment and that's exactly where we hope to go in CIDP as well. Before diving into the success of the quarter, I want to discuss the real-life impact of our treatment.

在重症肌無力症 (MG) 中，我們的最低症狀表現率是所有治療方法中最高的。在 CIDP 中，我們看到了與 ADHERE 數據相符的真實結果，包括功能改進。這項針對患者的價值主張支持了我們在日益激烈的競爭環境中作為 MG 領域領先品牌生物製劑的地位，這也正是我們希望在 CIDP 領域實現的目標。在深入探討本季的成功之前，我想先討論一下我們的治療對現實生活的影響。

Lynn, a biomedical engineer and marathon runner, was first diagnosed with CIDP when she was training for an Ironman and noticed a strange tingling in her fingers. Her CIDP unfortunately progressed, notwithstanding treatment with IVIg and other therapies. She maintained an active lifestyle and has been vocal about the challenges that come with managing her CIDP.

林恩是一名生物醫學工程師和馬拉松運動員，她在參加鐵人三項訓練時首次被診斷出患有 CIDP，當時她注意到她的手指有一種奇怪的刺痛感。不幸的是，儘管接受了 IVIg 和其他療法的治療，她的 CIDP 病情仍然惡化。她保持著積極的生活方式，並直言不諱地談論管理 CIDP 所面臨的挑戰。

Recently, she switched to VYVGART Hytrulo prefilled syringe, which she cited as an absolute game-changer for her treatment experience. She also shared that she's seen an overall improvement in her quality of life. Recently, for the first time in eight years, she didn't plan around a hospital schedule while traveling outside the US for three weeks. And while every patient's experience is different, this is just one of the many inspiring stories we've heard throughout this launch.

最近，她改用 VYVGART Hytrulo 預充填式註射器，她稱這絕對改變了她的治療體驗。她也表示，她的生活品質有了全面改善。最近，她八年來第一次沒有按照醫院的日程安排在美國境外旅行了三週。雖然每個病人的經歷都不同，但這只是我們在這次發布會上聽到的眾多鼓舞人心的故事之一。

Next slide. The team delivered another phenomenal quarter, and we saw VYVGART growing across all indications, formulations and regions. In the US, the introduction of the prefilled syringe led to increased demand for VYVGART with more patients initiating treatment across all VYVGART product presentations than we've seen in prior quarters. 50% of PFS patients were brand new to VYVGART with the other switching from Hytrulo vial or VYVGART IV. We also see the PFS expanding our prescriber base, which ultimately opens up our ability to reach new patients.

下一張投影片。團隊又一個季度表現出色，我們看到 VYVGART 在所有適應症、配方和地區都實現了成長。在美國，預充式註射器的推出導致對 VYVGART 的需求增加，與前幾季相比，更多患者開始使用 VYVGART 產品進行治療。 50% 的 PFS 患者是首次使用 VYVGART，其他患者則是從 Hytrulo 小瓶或 VYVGART IV 轉換。我們也看到 PFS 擴大了我們的處方者基礎，這最終增強了我們接觸新患者的能力。

Over 1,000 physicians have written a PFS prescription in the first quarter of launch with around 15% being first-time prescribers of any VYVGART product presentation. As with our prior launches, early momentum has been enabled by our ability to secure access quickly with favorable policies. As of this week, we have secured policies representing 70% of commercial lives.

在推出的第一季度，已有超過 1,000 名醫生開出了 PFS 處方，其中約 15％ 是首次開立 VYVGART 產品處方。與我們之前的發布一樣，早期的勢頭得益於我們能夠透過優惠政策快速獲得存取權限。截至本週，我們已獲得代表 70% 商業壽命的政策。

Let's now look at the growth dynamics for MG and CIDP, both of which meaningfully contributed to the quarter's performance and have near-term expansion opportunities. Slide 10. VYVGART continues to gain momentum in MG. It has now been 14 quarters since we launched VYVGART in MG and 14 quarters of consistent growth. Even so, we believe we are still at the early stages of unlocking the full opportunity, which we estimate to be 60,000 patients in the US. First priority is to shift the treatment paradigm, moving the goalpost for patients and resetting expectations of what a treatment can do.

現在讓我們來看看 MG 和 CIDP 的成長動態，它們都對本季的業績做出了重大貢獻，並且具有近期的擴張機會。幻燈片 10。VYVGART 在 MG 中繼續獲得發展勢頭。自從我們在 MG 推出 VYVGART 以來，已經過了 14 個季度，並且實現了 14 個季度的持續成長。即便如此，我們相信我們仍處於釋放全部機會的早期階段，我們估計美國有 60,000 名患者。首要任務是轉變治療模式，改變患者的目標並重新設定對治療效果的期望。

It's no longer that being controlled is being out of the hospital or being reliant on high doses of broad immunosuppressants. It's about being symptom-free and getting back to the activities you love. This shift is already underway and 60% of new patients to VYVGART come from oral, reflecting the value VYVGART delivers through its consistent safety and efficacy.

得到控制不再意味著出院或依賴高劑量的廣譜免疫抑制劑。這是為了擺脫症狀並恢復您喜歡的活動。這一轉變已經在進行中，60% 的 VYVGART 新患者來自口服，這反映了 VYVGART 透過其一致的安全性和有效性所提供的價值。

Second, we know that branded biologics still represent only 10% of the MG market today. We see this expanding as more innovation enters the market, all of which serves to raise treatment expectations for patients. We're focused on maintaining our position as the fastest-growing biologic in this evolving landscape.

其次，我們知道品牌生物製劑目前仍僅佔 MG 市場的 10%。隨著越來越多的創新進入市場，我們看到這種趨勢正在擴大，所有這些都有助於提高患者的治療期望。我們致力於在這個不斷變化的環境中保持我們作為成長最快的生物製劑的地位。

The launch of the prefilled syringe will be critical to achieving both of these goals. The PFS is expanding access to new MG patient segments, particularly those we couldn't reach with HCP administration as the only option. It's also emerging as a key differentiator to help us reach that additional 25,000 patients as the total biologic adoption grows. Lastly, we're also advancing registrational trials in seronegative and ocular MG, which together represent 18,000 patients of our total addressable market, and it supports our goal of VYVGART having the broadest MG label.

預充式註射器的推出對於實現這兩個目標都至關重要。PFS 正在擴大對新的 MG 患者群體的覆蓋範圍，特別是那些我們無法透過 HCP 管理作為唯一選擇覆蓋的患者群體。隨著生物製劑整體採用率的提高，它也逐漸成為幫助我們涵蓋另外 25,000 名患者的關鍵差異化因素。最後，我們也正在推進血清陰性和眼部 MG 的註冊試驗，這代表了我們整個目標市場的 18,000 名患者，它支持我們讓 VYVGART 擁有最廣泛的 MG 標籤的目標。

Slide 11. Turning to CIDP. We continue to see consistent growth across all key patient and prescriber metrics, the sign of a very successful launch. As of the end of June, over 2,500 patients have been treated globally with VYVGART Hytrulo, most of which are coming from the US. The launch in Japan and Germany are also off to a fast start.

幻燈片 11。轉向 CIDP。我們繼續看到所有關鍵患者和處方者指標的持續增長，這是一個非常成功的發布的標誌。截至 6 月底，全球已有超過 2,500 名患者接受 VYVGART Hytrulo 治療，其中大多數來自美國。日本和德國的上市也進展順利。

This momentum is driven by the clear and unmet need, and meaningful outcomes driven by the safety and efficacy we've seen with VYVGART Hytrulo in the real world and now the added convenience of our prefilled syringe.

這種動力是由明確且未滿足的需求所驅動，而有意義的結果則是由我們在現實世界中看到的 VYVGART Hytrulo 的安全性和有效性以及現在我們的預充式註射器的額外便利所驅動。

Importantly, we see significant room to continue to grow within our initial 12,000 patient population, and we believe the strong value proposition of VYVGART Hytrulo will support continued expansion even beyond that over time. We see tremendous opportunity ahead in MG and CIDP. We're just getting started.

重要的是，我們看到在最初的 12,000 名患者群體中還有很大的增長空間，並且我們相信 VYVGART Hytrulo 的強大價值主張將支持其隨著時間的推移繼續擴張。我們看到 MG 和 CIDP 未來存在著巨大的機會。我們才剛開始。

These indications alone give us a strong foundation for continued growth, and we're applying the same playbook as we expand into new diseases. We're focusing on what matters most, generating meaningful data for patients, moving rapidly and staying ahead of the competition through innovation.

這些跡象為我們持續成長奠定了堅實的基礎，我們在拓展新疾病治療領域時也採用了同樣的策略。我們專注於最重要的事情，為患者產生有意義的數據，快速行動並透過創新保持競爭優勢。

And with that, I'll now turn the call back to Tim.

說完這些，我現在將電話轉回給提姆。

Thank you, Karen. We are executing across the business to transform the treatment landscape for patients with autoimmune diseases. What we have achieved in MG and CIDP alone reflects the significant value we have already unlocked and the substantial growth still ahead. We are excited to build on our proven ability to translate innovation into commercial success as we enter new markets and the opportunity in front of us is expansive.

謝謝你，凱倫。我們正在整個業務範圍內採取行動，改變自體免疫疾病患者的治療狀況。僅在 MG 和 CIDP 上，我們所取得的成就就反映了我們已經釋放的巨大價值以及未來的巨大成長。隨著我們進入新市場並面臨廣泛的機遇，我們很高興能夠利用我們已證實的能力將創新轉化為商業成功。

Over the next 18 months, we expect data from six Phase 3 and six Phase 2 trials across our pipeline, each with the potential to expand our reach into new patient populations and unlock addressable markets well beyond where we are today. As we scale for the long term, we remain deeply committed to creating lasting value for our shareholders, our partners and most importantly, the patients we serve.

在接下來的 18 個月中，我們預計將從我們的整個產品線中獲得 6 個 3 期和 6 個 2 期試驗的數據，每個試驗都有可能將我們的覆蓋範圍擴大到新的患者群體，並開拓遠遠超出我們目前水平的潛在市場。在我們長期擴張的過程中，我們仍然堅定地致力於為我們的股東、合作夥伴以及最重要的，我們服務的患者創造持久的價值。

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

接線員，接下來我們將開始回答問題。

(Operator Instructions)

（操作員指示）

Alex Thompson, Stifel.

亞歷克斯·湯普森（Alex Thompson），Stifel。

Great. Good morning and thank you for taking my questions. I guess, really, how have your cycles per year in MG evolved as you generated additional chronic dosing data from AdDAP-NXT and with neurologists getting more comfortable with this idea of chronic FcRn administration? I guess really what I'm asking is one of the drivers of a higher gross to net here outside of the Part D exposure, a higher gross price per patient in MG followed by greater discounting towards a consistent net price? Thanks

偉大的。早上好，感謝您回答我的問題。我想，隨著您從 AdDAP-NXT 中獲得了額外的慢性給藥數據，並且神經科醫生對慢性 FcRn 給藥的想法越來越適應，您每年的 MG 週期是如何演變的？我猜我真正想問的是，除了 D 部分風險之外，導致總價與淨價上升的驅動因素之一是重症肌無力患者人均總價更高，隨後折扣力度更大，以達到穩定的淨價？謝謝

Thank you, Alex, for joining us on the call today. I would like to kick it off with Karl's answer to the second part of your question. And then I really like where you're going, and I would like to give that question -- part one of the question to Karen. Karl?

感謝亞歷克斯今天參加我們的電話會議。我想以卡爾對你問題第二部的回答開始。然後我真的很喜歡你要說的話，我想把這個問題——問題的第一部分交給凱倫。卡爾？

Thank you, Alex. The net revenue per patient for MG and CIDP remains consistent even with a higher gross to net. There are many variables that go into the net revenue per patient. It includes product mix, list price, gross to net utilization and adherence.

謝謝你，亞歷克斯。即使總收入與淨收入之比較高，MG 和 CIDP 的每位患者淨收入仍保持一致。影響每位患者淨收入的變數有很多。它包括產品組合、標價、總利用率與淨利用率以及依從性。

It is fair to assume that the combination of these variables offsets the impact of gross to net. And then also for the sake of clarity, we want to confirm that we have not taken any price increase during 2025 in the US. Over to you, Karen.

可以合理地假設這些變數的組合抵消了總額對淨額的影響。然後，為了清楚起見，我們想確認在 2025 年期間美國不會提高任何價格。交給你了，凱倫。

Thanks, Karl. Yes, and thanks for pointing out. I mean, we had a really strong quarter in MG, and that was actually driven by patient adds is what I would suggest. But we continue to guide to five cycles per year on average for an MG patient. But what you're right to call out is that there is a potential for higher utilization and adherence with Hytrulo and particularly with PFS because of the convenience, you can imagine, for patients, the convenience of a PFS for self-injection.

謝謝，卡爾。是的，感謝您的指出。我的意思是，我們在 MG 的一個季度表現非常強勁，我認為這實際上是由患者增加所推動的。但我們仍持續指導重症肌無力患者平均每年進行五個週期的治療。但您說得對，Hytrulo 和 PFS 具有更高的利用率和依從性的潛力，因為對於患者來說，您可以想像 PFS 可以方便地進行自我注射。

The other big driver here and what you can imagine is that once a patient experiences MSE with VYVGART, they want to stay in that minimum symptom expression. That's the advantage of individualized dosing, right? They can pick their dosing that keeps them in MSE. And that's that positive experience that is keeping patients on VYVGART. So those factors combined are really what led us to delivering what is remarkable. I think it's 14 quarters straight of growth with MG.

另一個重要驅動因素以及您可以想像的是，一旦患者透過 VYVGART 體驗到 MSE，他們就希望保持最低限度的症狀表現。這就是個體化用藥的優勢，對嗎？他們可以選擇適合自己的劑量以保持 MSE。正是這種正面的體驗讓患者繼續使用 VYVGART。所以，正是這些因素的結合才真正促使我們取得了非凡的成就。我認為 MG 已經連續 14 個季度實現成長。

Great. Thank you.

偉大的。謝謝。

Your next question comes from the line of Tazeen Ahmad -- bear with me moment.

您的下一個問題來自 Tazeen Ahmad——請耐心等待片刻。

Derek Archila, Wells Fargo.

富國銀行的德里克‧阿奇拉 (Derek Archila)。

Hey. Good morning and congrats on the update here and thank you for taking my question. So just one on the breakdown of the PFS switches between Hytrulo and IV. I know you said that 60% of PFS patients were new, leaving 40% from switches, but what's that breakdown look like? Thanks.

嘿。早安，恭喜您的更新，感謝您回答我的問題。因此，只需對 Hytrulo 和 IV 之間的 PFS 切換進行故障分析即可。我知道您說過 60% 的 PFS 患者是新患者，剩下 40% 是轉換患者，但這種細分情況是如何的呢？謝謝。

Derek, thanks for joining us on the call today, and thank you for this question, which is really focusing how are we growing the market with PFS instead of just switching an existing market, right, Karen?

德里克，感謝您今天參加我們的電話會議，也感謝您提出這個問題，這個問題真正關注的是我們如何透過 PFS 來擴大市場，而不是僅僅轉換現有市場，對吧，凱倫？

Yeah, absolutely. So we don't provide the split by product presentation, but we've shared in previous quarters a few facts that are important. Hytrulo is driving the majority of the growth for VYVGART. And as you rightly called out, 50% of prefilled syringe patients are new to VYVGART. So our strategy with bringing these innovations to market in both MG and CIDP is that we're looking to expand the market and improve our differentiation in increasingly competitive markets.

是的，絕對是如此。因此，我們不提供按產品分類的介紹，但我們在前幾個季度分享了一些重要的事實。Hytrulo 推動了 VYVGART 的大部分成長。正如您正確指出的那樣，50% 的預充式註射器患者都是第一次使用 VYVGART。因此，我們將這些創新推向 MG 和 CIDP 市場的策略是，我們希望擴大市場並在競爭日益激烈的市場中提高我們的差異化。

And that's exactly what we're seeing, the prefilled syringe is delivering is that market growth -- market expansion and product growth. So you can expect that to continue, I would say, through the end of the year. Thanks for the question.

這正是我們所看到的，預充式註射器帶來的是市場成長－市場擴張和產品成長。所以我想說，你可以預期這種情況會持續到今年年底。謝謝你的提問。

Akash Tewari, Jefferies.

Akash Tewari，傑富瑞。

Hey. Thanks so much. So our high-level math suggests your gMG patient adds are really meaningfully growing. I think it went from 800 to something over 1,500 in Q2. How much of this was the prefilled syringe allowing you to unlock new patients in an earlier line setting, and it doesn't seem like you're necessarily cannibalizing from the IV.

嘿。非常感謝。因此，我們的高級數學計算表明，gMG 患者的數量確實在顯著增長。我認為第二季的數量從 800 增加到了 1,500 多。其中有多少是預充式註射器，可以讓您在早期的線路設定中解鎖新患者，而且看起來您不一定是從 IV 中蠶食的。

And really, what's the correct run rate assumption going forward? And maybe just secondly, on your seronegative trial, can you talk about your confidence on that study reading out well? And why your primary endpoint for that trial is absolute MG-ADL drop versus the MG responder endpoint you've used in the past? Thank you.

那麼，未來正確的運行率假設是什麼？其次，關於您的血清陰性試驗，您能否談談您對該研究結果的信心？為什麼該試驗的主要終點是絕對 MG-ADL 下降，而不是您過去使用的 MG 反應終點？謝謝。

Yeah. Thank you, Akash. And we have the benefit of having our Chief Medical Officer, here today who is on the call, Luc. So Luc, we will give question two to you about the seronegative trial, but let's kick it off with you, Karen.

是的。謝謝你，阿卡什。我們很榮幸今天能有我們的首席醫療官 Luc 出席此次電話會議。那麼 Luc，我們將向您提出有關血清陰性試驗的第二個問題，但讓我們從您開始，Karen。

Yes. I think you rightly call out, we had a very strong quarter in MG, and that was fueled by prefilled syringe as we expected, expanding the market. Now prefilled syringe allowed us to grow the prescriber base. We had 1,000 prescribers for prefilled syringe in the quarter, 150 of those are new to VYVGART. And that's exactly what we thought would happen. So we're really pleased with the launch of prefilled syringe. It's delivering on what we thought it would deliver.

是的。我認為您說得對，我們在 MG 方面本季表現非常強勁，這得益於預充式註射器的推動，正如我們預期的那樣，它擴大了市場。現在預充式註射器使我們能夠擴大處方者基礎。本季我們有 1,000 名預充式註射器處方者，其中 150 名是 VYVGART 的新處方者。這正是我們所想會發生的。因此，我們對預充式註射器的推出感到非常高興。它實現了我們期望它能實現的目標。

In parallel to that, we are continuing to see growth in our IV business in MG. And that's because there is a place in the market where certain prescribers and patients prefer the IV option, and that's in line with our strategy that we want to meet the doctors and the patients where they are.

同時，我們的MG IV業務也持續成長。這是因為市場上某些處方者和患者更喜歡靜脈注射選項，這符合我們希望在醫生和患者所在地與他們會面的策略。

We want to provide the best efficacy and safety, and we know we have that with VYVGART, and we have multiple options for product presentations so that we can continue to really expand the market in MG, start to move into those earlier lines of treatment. And as the number 1 biologic and the fastest-growing biologic in the market, we're seeing exactly that play out in the market.

我們希望提供最佳的療效和安全性，我們知道 VYVGART 可以滿足這一需求，而且我們在產品展示方面有多種選擇，因此我們可以繼續真正擴大 MG 市場，並開始進入早期治療領域。作為市場上排名第一和成長最快的生物製劑，我們看到了這種現像在市場上的體現。

Luc, I'll hand it over to you.

盧克，我把它交給你了。

Thanks, Karen, and thanks for the question. We're very excited for the upcoming results. So directly related to the change of primary endpoints. So we changed to a change from baseline in MG-ADL because compared to a responder kind of definition and therefore, dichotomy, you retain more information and therefore, intrinsically, it would increase the power.

謝謝，凱倫，謝謝你的提問。我們對即將公佈的結果感到非常興奮。因此與主要終點的變化直接相關。因此，我們改變了 MG-ADL 的基線，因為與反應者類型的定義和二分法相比，您可以保留更多信息，因此從本質上講，它會增加力量。

And then secondly, we were also able to negotiate with the agency that based on all the data we already had on seronegatives that we could put the required p-value to be reached at 0.1. So those are two things that will help. And then what we also added was a better diagnostic accuracy ascertainment at the beginning to make sure we had the right patients in.

其次，我們也能與該機構協商，根據我們現有的所有血清陰性數據，我們可以將所需的p值設為0.1。所以，這兩點會有所幫助。然後，我們還在開始時增加了更好的診斷準確性確定，以確保我們找到合適的患者。

Yeah. Thank you, Luc. So to wrap it up, our confidence level is high, but guys, it's still a clinical trial, which has intrinsic risks. Thanks for the question.

是的。謝謝你，盧克。總而言之，我們的信心水平很高，但是夥計們，這仍然是一項臨床試驗，具有內在風險。謝謝你的提問。

Tazeen Ahmad, Bank of America.

美國銀行的塔津·艾哈邁德（Tazeen Ahmad）。

Hi, guys. Maybe my phone is just as excited about your beat as I am. I wanted to maybe ask two questions. Can you talk about expectations for increasing competition as the year progresses? Specifically, we've been getting a lot of questions about UPLIZNA. How are you thinking about the dynamic for that? And then secondly, can you just talk about the drop-off rate from treatment for patients? Maybe let's start with gMG because it's been launched longer.

嗨，大家好。也許我的手機和我一樣對你的節拍感到興奮。我想問兩個問題。您能談談對未來競爭日益激烈的預期嗎？具體來說，我們收到了很多有關 UPLIZNA 的問題。您如何看待這種動態？其次，您能談談患者的治療退出率嗎？也許我們可以從 gMG 開始，因為它推出的時間更長。

Yeah. Thank you, Tazeen, for joining us. And Karen, I think these are two excellent questions for you. Why don't we maybe kick it off with our views on the competitive dynamics of our space?

是的。感謝 Tazeen 加入我們。凱倫，我認為這兩個問題問得非常好。我們為什麼不從對我們所在領域競爭動態的看法開始討論呢？

Yeah, absolutely. As you say, Tazeen, the competitive dynamics or the competition is certainly heating up, and I would say as the leading biologic and the fastest-growing biologic, our task is to continue to raise the bar on what patients and prescribers can expect in MG. And we're doing exactly that. So whether you look at our MSE rate, our rapid and sustained efficacy, over 10,000 patient years of safety, and we've just talked about all of our product presentations, I think we set the bar very high in MG.

是的，絕對是如此。正如您所說，Tazeen，競爭態勢或競爭肯定正在升溫，我想說，作為領先的生物製劑和增長最快的生物製劑，我們的任務是繼續提高患者和處方者對 MG 的期望值。而我們正是這麼做的。因此，無論您看我們的 MSE 率、我們的快速和持續功效、超過 10,000 名患者年的安全性，以及我們剛剛討論過的所有產品介紹，我認為我們在 MG 方面設定的標準非常高。

Having said that, and we've said this before, we welcome innovation to MG market, to the CIDP market. Innovation is great for patients. And we believe that we are very well positioned to continue to lead and to continue to be the fastest-growing biologic within that expanding biologic market in MG.

話雖如此，我們之前也說過，我們歡迎 MG 市場和 CIDP 市場的創新。創新對於患者來說非常有益。我們相信，我們完全有能力繼續保持領先地位，並繼續成為 MG 不斷擴大的生物製劑市場中成長最快的生物製劑。

In terms of your second question on the discontinuation rate, I wasn't sure if it was for MG or CIDP or both, but I can say for both MG and CIDP, the discontinuation rate is in line with expectations, what you would expect for a chronic medicine. And in particular, as we talked about earlier, I think in MG, what we're seeing is once patients get into that MSE, they want to optimize their dose, and they want to stay on VYVGART.

關於您的第二個問題，即停藥率，我不確定是針對 MG 還是 CIDP，還是兩者兼而有之，但我可以說，對於 MG 和 CIDP，停藥率都符合預期，這與您對慢性病藥物的預期相符。特別是，正如我們之前所討論的，我認為在 MG 中，我們看到的是，一旦患者進入 MSE，他們就希望優化劑量，並且希望繼續使用 VYVGART。

With CIDP, we're seeing the majority of patients on a weekly. They're staying with weekly, but we do expect that some patients will start to switch to biweekly and our discontinuation rate is in line with what you'd expect for chronic medicine.

透過 CIDP，我們每週都會接待大多數患者。他們仍然堅持每週服用一次，但我們確實預計一些患者將開始改為每兩週服用一次，而且我們的停藥率與慢性病藥物的預期一致。

Thanks for the question.

謝謝你的提問。

Rajan Sharma, Goldman Sachs.

高盛的拉詹·夏爾馬。

Hi. This is Max for Rojan. So our question is, so based on the patient numbers you disclosed in the release, it looks like the number of patients on VYVGART for CIDP almost doubled from the end of January to the end of June. Is that a good proxy for the growth rate during the remainder of the year?

你好。這是 Max，代表 Rojan。所以我們的問題是，根據您在新聞稿中披露的患者人數，看起來從 1 月底到 6 月底，VYVGART 上 CIDP 的患者人數幾乎翻了一番。這是今年剩餘時間成長率的良好代表嗎？

Yeah. So thanks for the question. We're really pleased with that 2,500 patient number. And I think what you can expect is that the growth will continue through the end of the year. I do want to remind you that 2,500 patients is global. And in particular, we had a launch in Japan in the beginning of the year.

是的。感謝您的提問。我們對 2,500 名患者的數量感到非常高興。我認為你可以預期這種增長將持續到今年年底。我確實想提醒你，2,500 名患者是全球性的。特別是，我們在今年年初就在日本推出了該產品。

We recently had the EMA approval. So we've also launched in Germany. And I can tell you that both of those markets are off to a very fast start similar to what we saw in the US. And I think what those fast starts across all markets demonstrate is that patients and providers have been waiting for innovation to come to the CIDP market, and they're excited for VYVGART.

我們最近獲得了 EMA 的批准。因此我們也在德國推出了該產品。我可以告訴你們，這兩個市場都起步非常快，與我們在美國看到的情況類似。我認為所有市場快速起步表明患者和供應商一直在等待創新進入 CIDP 市場，他們對 VYVGART 感到興奮。

Thanks for the question.

謝謝你的提問。

James Gordon, JPMorgan.

摩根大通的詹姆斯戈登。

Hello. James Gordon, JPMorgan. Thanks for taking the questions. First question is on the CIDP launch. So I think President, you pointed us to just being reimbursed 12,000 US CIDP patients who failed Ig. But what are you seeing there?

你好。摩根大通的詹姆斯戈登。感謝您回答這些問題。第一個問題是關於 CIDP 啟動的。所以我認為總統先生，您指出我們只需向 12,000 名 Ig 失敗的美國 CIDP 患者報銷。但是你在那裡看到了什麼？

And how might it change? We did a survey that said or respondents said that actually quite a lot of the use was already in Ig-naive CIDP patients and the growth is going to increase -- or uptake was going to be growing almost as quickly in the naive patients as the experienced patients.

又將如何改變呢？我們做了一項調查，結果顯示或受訪者表示，實際上相當多的使用已經在未接受免疫球蛋白治療的 CIDP 患者中實現，並且增長速度將會增加 - 或者說，未接受免疫球蛋白治療的患者中的吸收量增長速度幾乎與接受免疫球蛋白治療的患者中的吸收量一樣快。

So are you seeing any of that? How might that change? How are you shape? Are you going to get some more data in CIDP to try and get use earlier or just it takes time? And then just a follow-up, which was gross margin assumptions. So I had heard some concerns that gross margin was going to contract as a result of IRA and effectively the discount you have to give and paying to Halozyme.

那你看到這些了嗎？這種情況將如何改變？你身體怎麼樣？您是否會在 CIDP 中取得更多資料以嘗試儘早使用，還是只是需要一些時間？然後是後續問題，即毛利率假設。因此，我聽到一些擔憂，即由於 IRA 以及實際上必須給予和支付給 Halozyme 的折扣，毛利率將會下降。

But then I also saw some comments in Zai Lab, which seem to suggest a very significant reduction in production costs for VYVGART, and that presumably will help your COGS ratio a lot. So what's the gross margin outlook going forward?

但後來我也看到了 Zai Lab 的一些評論，這些評論似乎表明 VYVGART 的生產成本大幅降低，這大概會對你們的 COGS 比率有很大幫助。那麼未來的毛利率前景如何呢？

Maybe, Karl, you start with the question two on cost of goods and the impact on gross margin. And then Karen, we can bring it back to you to where we are on the adoption curve in CIDP and how we see that evolve, right? Karl?

卡爾，也許你可以從第二個問題開始，也就是商品成本及其對毛利率的影響。然後 Karen，我們可以向您介紹我們在 CIDP 的採用曲線上所處的位置以及我們如何看待它的發展，對嗎？卡爾？

Yeah. Thank you, James. Thank you for the question. The gross margin on a year-to-date basis is currently at around 11%. Going forward, we expect that number to remain around 11%. Two offsetting factors going into that. We continue to drive down the cost of sales with our CDMOs as we move to larger sites, bespoke sites with higher yields and economies of scale.

是的。謝謝你，詹姆斯。謝謝你的提問。年初至今的毛利率目前約為11%。展望未來，我們預計該數字將保持在 11% 左右。有兩個抵消因素。隨著我們轉向更大的站點、具有更高收益和規模經濟的客製化站點，我們繼續降低 CDMO 的銷售成本。

And that is the reference I think Zai is probably referring to. Also in cost of sales is the royalty we're paying to Halozyme. And with Hytrulo becoming a bigger share of the business, and remember, we're paying royalties only on Hytrulo, not on IV, you would expect the royalty number to increase over time. So the decreasing standard cost of sales plus the increasing royalties should largely offset. And therefore, we expect the cost-of-sales percentage to be around 11%.

我認為 Zai 可能指的就是這個。銷售成本中還包括我們向 Halozyme 支付的特許權使用費。隨著 Hytrulo 在我們業務中所佔的份額越來越大，請記住，我們只為 Hytrulo 支付版稅，而不是為 IV 支付版稅，您可以預期版稅金額會隨著時間的推移而增加。因此，標準銷售成本的下降加上特許權使用費的增加應該會在很大程度上抵消。因此，我們預計銷售成本百分比將在 11% 左右。

Thank you for the question.

謝謝你的提問。

Yeah. Thank you. And in terms of the CIDP patient dynamics, so we're one year into launch. We're seeing strong growth and patient adds. We are still seeing in our data that 85% to 90% of the patients are coming from IVIg switches. And you'll recall, that's what we expected, and that's how we define that 12,000 TAM or addressable market as patients that are uncontrolled on their current medicines, the majority of which is IVIg.

是的。謝謝。就 CIDP 患者動態而言，我們已經推出一年了。我們看到了強勁的成長和患者的增加。我們的數據仍然顯示，85% 至 90% 的患者來自 IVIg 轉換。您會記得，這正是我們的預期，這就是我們如何將 12,000 TAM 或可尋址市場定義為無法控制當前藥物的患者，其中大多數是 IVIg。

So we are still seeing that that's where the majority of our source of business is. That could expand over time, but I would caution that we are very early on the launch curve. We're just one year into the launch. So we have a lot of growth ahead of us and a long way to go even in those 12,000 uncontrolled patients.

因此，我們仍然看到我們大部分的業務來源地在那裡。這可能會隨著時間的推移而擴大，但我要提醒的是，我們正處於發布曲線的早期階段。我們推出產品才一年時間。因此，我們還有很長的路要走，即使在那 12,000 名不受控制的患者中，我們也還有很大的發展空間。

To your question about when would we expect to start to get into earlier line patients or some naive patients, we do see some already. Our label enables it. So we don't need to do an additional study or change our label. Rather, the key here is making sure that we get the payer access in place. And obviously, HCP and patient experience is going to be critical as well. But I think you're pointing out the most important fact, which is we are very early on the growth curve in CIDP despite the strong uptake.

對於您提出的問題，即我們何時可以開始治療早期患者或一些初治患者，我們確實已經看到了一些。我們的標籤可以實現這一點。所以我們不需要做額外的研究或更改我們的標籤。相反，這裡的關鍵是確保我們能夠獲得付款人的存取權限。顯然，HCP 和患者的體驗也至關重要。但我認為您指出了最重要的事實，那就是儘管 CIDP 的成長勢頭強勁，但我們仍處於成長曲線的早期階段。

Thanks for the question.

謝謝你的提問。

(Operator Instructions)

（操作員指示）

Yatin Suneja, Guggenheim.

古根漢美術館的亞廷·蘇內賈（Yatin Suneja）。

Hey, guys. Thank you for taking my question. Just a quick one on gross to net as a clarification. So for this quarter, it was 20%. Could you comment on how do you think it is going to evolve for the second half of this year and then as we go into next year? Yeah, Thank you.

嘿，大家好。感謝您回答我的問題。簡單澄清一下總額與淨額的關係。本季這一比例為 20%。您能否評論一下您認為今年下半年以及明年的情況將如何發展？是的，謝謝。

Yatin, thank you for the question. Yeah, the gross to net increased to 20%, and that is a year-to-date number. It's not referring to the second quarter. So that's an 8% increase from the end of last year to the middle of the year. We talked about the key drivers of gross to net, so I won't repeat that.

Yatin，謝謝你的提問。是的，毛利與淨利比增加到了 20%，這是年初至今的數字。這並不是指第二季。因此，從去年年底到今年年中，這一數字成長了 8%。我們討論了毛利潤與淨利潤之間的關鍵驅動因素，因此我就不再重複了。

But I think what's really important is that the bulk of the increase is now behind us. Going forward, we will see increases in gross to net, but it will be smaller increases driven by the product mix, i.e., if PFS becomes a bigger share of a business, which it will, you're going to see gross to net creeping up.

但我認為真正重要的是，大部分成長已經過去。展望未來，我們將看到毛利與淨利之間的比率增加，但受產品組合推動的增幅會較小，也就是說，如果 PFS 在業務中所佔的份額變大（事實確實如此），你就會看到毛利與淨利之間的比率緩慢上升。

But the important thing and what we want to emphasize is that the net revenue per patient has not changed. And as I mentioned, at least for the foreseeable future, we're not expecting that to change. PFS comes with unique dynamics, but ultimately, it's expanding the market, resulting in incremental patients, which will drive revenues.

但重要的是，我們要強調的是，每位患者的淨收入並沒有改變。正如我所提到的，至少在可預見的未來，我們預計這種情況不會改變。PFS 具有獨特的動力，但最終，它將擴大市場，從而增加患者數量，從而推動收入成長。

Thank you for the question.

謝謝你的提問。

Yaron Werber, TD Cowen.

Yaron Werber，TD Cowen。

Great. Congrats on a great quarter. Two quick interrelated questions. Maybe just the first one, give us an update on the IV to subcu switch for VYVGART that you're seeing so far? And then secondly, we noticed that new ENERGIZE Phase 3 for empa head-to-head against placebo. So this is not an IVIg switch. There's almost like a Part A and then a Part B in the Phase 3 in adults with CIDP. Can you talk about the trial design and the strategy? Thank you.

偉大的。恭喜本季取得優異成績。兩個相互關聯的問題。也許只是第一個，請向我們提供您目前看到的 VYVGART 的 IV 到 subcu 開關的更新資訊？其次，我們注意到，新的 ENERGIZE Phase 3 與安慰劑有正面交鋒。所以這不是 IVIg 開關。對於患有 CIDP 的成年人來說，第 3 階段幾乎就像是先有 A 部分，然後有 B 部分。能談談試驗設計和策略嗎？謝謝。

Yeah. Thank you, Yaron, and thanks for joining us today in the call. Maybe, Karen, you want to talk a little bit about the IV to subcu switch. It's not really a switch dynamic we're driving. And then Luc, I would like to call on you to explain a little bit about empa strategy and the high-level features of this placebo-controlled second trial, okay? Karen?

是的。謝謝你，亞倫，謝謝你今天參加我們的電話會議。也許，凱倫，你想談談靜脈注射到皮下注射的轉換。我們真正推動的並不是轉變動力。然後，Luc，我想請你稍微解釋一下 empa 策略以及這個安慰劑對照的第二次試驗的高級特徵，好嗎？卡倫？

Yeah. Happy to take the question. So as Tim already flagged, our strategy with a prefilled syringe or subcutaneous is not a switch strategy. It's a market expansion strategy, and we're seeing that play out in the market. In fact, we're also seeing -- and recall that IV is only indicated for MG. What we're seeing is continued growth in the IV business.

是的。很高興回答這個問題。正如蒂姆已經指出的那樣，我們使用預充式註射器或皮下注射的策略並不是轉換策略。這是一種市場擴張策略，我們正在看到它在市場上發揮作用。事實上，我們也看到——回想一下，IV 僅適用於 MG。我們看到的是靜脈注射業務的持續成長。

So -- and we expect that, that will continue and that we will continue to see a substantial IV business in MG. And the reason for that is, again, the prescriber preference as well as patient preference, there is a portion of patients and prescribers who prefer that product presentation, and we expect that to continue to grow over the coming years. And Luc, if I can hand it over to you for the ENERGIZE question.

所以 — — 我們預計這種情況將會持續下去，並且我們將繼續看到 MG 的大量 IV 業務。原因在於，再次強調，處方醫生和患者的偏好，有一部分患者和處方醫生更喜歡這種產品展示方式，我們預計未來幾年這種趨勢將繼續增長。路克，我可以把有關 ENERGIZE 的問題交給你嗎？

Yes, it's energizing. So the approach we're taking here is that we learned a lot, and we've been innovators now in the CIDP field, first bringing efgartigimod forward, which shows you can have up to a 70% response, which would indicate an IGC dependency, but a third didn't respond.

是的，它充滿活力。因此，我們在這裡採取的方法是，我們學到了很多東西，我們現在一直是 CIDP 領域的創新者，首先提出了 efgartigimod，它顯示您可以獲得高達 70% 的回應，這表明存在 IGC 依賴性，但三分之一的人沒有回應。

And that then brings us to the question, okay, we are not satisfied. And then we saw the results of MMN with empa, which were quite spectacular, and we started thinking, could we not continue our journey and develop solutions for CIDP patients by also introducing empasiprubart. Once we've made that decision, the first thing is the recognition IVIg is, of course, widely used and could we be an improvement over IVIg. But that is not the only approach.

這就引出了一個問題：好吧，我們並不滿意。然後我們看到了 MMN 與 empa 的聯合治療效果，效果非常驚人，我們開始思考，我們能否繼續我們的旅程，並透過引入 empasiprubart 來為 CIDP 患者開發解決方案。一旦我們做出了這個決定，首先要考慮的就是 IVIg 的認可度，當然，它的使用範圍很廣，我們能否對 IVIg 做出改進。但這不是唯一的方法。

And that's why we also felt to kind of broaden that ability to get a signaling patient that we should look at patients that are either refractory or naive and look at the power of empasiprubart in that study. Study is pretty simple. It's a 24-week study against a placebo.

這就是為什麼我們也覺得應該擴大這種獲得訊號患者的能力，我們應該觀察那些難治性或初治性患者，並在該研究中觀察 empasiprubart 的功效。學習很簡單。這是一項為期 24 週的安慰劑研究。

Thank you, Luc. And we believe CIDP will become an increasingly competitive space. And therefore, the clinical development strategy, which you see emerge on clinicaltrials.gov, is basically there to give empa the best possible positioning in that exciting marketplace.

謝謝你，盧克。我們相信 CIDP 將成為一個競爭日益激烈的領域。因此，您在 clinicaltrials.gov 上看到的臨床開發策略基本上是為了讓 empa 在這個令人興奮的市場中獲得最佳定位。

Thanks for the questions.

感謝您的提問。

Sean Laaman, Morgan Stanley.

摩根士丹利的肖恩拉曼。

Thank you, operator and good morning, good afternoon, Tim and team. Thanks for taking my questions. Thinking strategically, I mean, you guys are now in a new era of profitability. I think cash flow will increase to cash was almost $0.5 billion for the six-month period. And on the other hand, you've got a number of clinical trials sort of coming up. I'm just wondering, how do you see the business evolving from here? What do you think of margin? And what's the balance sheet strategy going forward?

謝謝接線員，早上好，下午好，提姆和團隊。感謝您回答我的問題。從策略角度考慮，我的意思是，你們現在正處於一個獲利的新時代。我認為現金流將增加到六個月內現金近 5 億美元。另一方面，一系列臨床試驗即將到來。我只是想知道，您認為今後業務將如何發展？您如何看待保證金？未來的資產負債表策略是什麼？

Yeah. We're not going to comment on margins, and it's not a goal for us to strive for certain ratios and margins as an innovator in our spaces. But let us try to answer the question from a capital allocation point of view, right, Karl, because we are accumulating cash. Maybe you want to comment on this?

是的。我們不會對利潤率發表評論，作為我們領域的創新者，追求一定的比例和利潤率也不是我們的目標。但讓我們試著從資本配置的角度來回答這個問題，對吧，卡爾，因為我們正在累積現金。也許您想對此發表評論？

Yes. We have a really strong balance sheet, $3.9 billion. We added $500 million, $0.5 billion in the year, but also from an operating cash flow of $400 million in the year, which is really good. I think also as a CFO, I'm really proud to say that the revenue growth is outpacing the OpEx growth. But that resulting in incremental profit quarter-over-quarter.

是的。我們的資產負債表非常強勁，達到 39 億美元。我們增加了 5 億美元，今年的銷售額為 5 億美元，而且今年的營運現金流也達到了 4 億美元，這確實很好。我認為，作為一名財務官，我很自豪地說收入成長超過了營運支出成長。但這導致利潤逐季增加。

But that in itself is not the goal here. We are focusing on clinical catalysts and revenue growth. Our capital allocation priorities has always been clear. Priority number 1 is to deliver on the promise of VYVGART. There's still a lot of work to do, many indications and studies. Following that is empa and the rest of the pipeline. We've talked about it earlier, lots of exciting assets in science, and we want to invest there.

但這本身並不是這裡的目標。我們專注於臨床催化劑和收入成長。我們的資本配置重點一直很明確。首要任務是兌現 VYVGART 的承諾。還有很多工作要做，還有很多指示和研究。接下來是 empa 和其餘管道。我們之前已經討論過，科學領域有很多令人興奮的資產，我們想在那裡投資。

Third, what I want to highlight is the investment in our supply chain. The decisions we've made years ago bring us to a position today that we can say we have enough inventory and supply for all scenarios. And we need to make those same decisions for tomorrow, and that requires a lot of capital.

第三，我想強調的是我們對供應鏈的投資。我們多年前做出的決定使我們今天可以說我們擁有足夠的庫存和供應來應對所有情況。我們需要為明天做出同樣的決定，而這需要大量的資金。

Fourth is where we're starting to think about business development. The company has always looked outside for innovative biology. Typically, we found it in academic centers, but we're now moving to a stage where we can use the strength of our balance sheet to also look at other biotechs. And fifth, of course, we will get to a stage where we're going to return cash to shareholders, but that is not really part of the discussion today.

第四，我們開始考慮業務發展。該公司一直在外部尋找創新生物學。通常，我們在學術中心發現它，但現在我們正進入一個階段，我們可以利用我們的資產負債表的優勢來專注於其他生物技術。第五，當然，我們將進入向股東返還現金的階段，但這並不是今天要討論的內容。

So thank you very much for the question.

非常感謝您的提問。

Thomas Smith, Leerink Partners.

Leerink Partners 的 Thomas Smith。

Hey, guys. Good morning. Thanks for taking the questions and congrats on the really strong quarter. Just on CIDP, I was wondering if you could comment and maybe provide some updated thoughts on the FDA's FAERS update from June. Is there any visibility on where FDA is with their analyses or expectations on the timeline of resolution? And then just curious if there's any feedback you're hearing from prescribers in the field on this, any kind of evolution in thinking about how they're approaching switching from IVIg. Thanks so much.

嘿，大家好。早安.感謝您回答這些問題，並祝賀本季業績表現強勁。就在 CIDP 上，我想知道您是否可以發表評論，並就 FDA 6 月份的 FAERS 更新提供一些最新想法。FDA 對解決時間表的分析或預期如何？我只是好奇您是否聽過該領域處方人員對此的任何反饋，他們在如何從 IVIg 轉換方面有什麼想法？非常感謝。

Yeah. And I'm going to give the floor to Luc to briefly comment on this fares question. And then maybe, Karen, time for you to echo the voice of the field, right, the marketplace. Luc, why don't you kick it off?

是的。我將請呂克就票價問題作簡短評論。那麼，凱倫，也許是時候讓你回應這個領域、對，市場的聲音了。盧克，你為什麼不開始呢？

Yeah. And thanks for the question and allowing to give some context to this. We first talk about FAERS itself. So that's an important tool in the safety monitoring established in 2004, but it comes with many limitations. So it is actually a building repository or database with inputs from sponsors, providers and patients. But one thing that is missing in there is denominators.

是的。感謝您提出這個問題並允許我對此提供一些背景資訊。我們首先討論FAERS本身。這是 2004 年建立的安全監控中的重要工具，但它也有很多限制。因此，它實際上是一個包含贊助商、供應商和患者輸入的建築存儲庫或資料庫。但其中缺少一個東西，那就是分母。

And it is, therefore, not really useful to really assign causality or definitely not make a benefit-risk statement. And the reason I'm saying that is that a fast increase in exposure could lead to an increasing reporting. And that is maybe one of the reasons here given, as you saw, the success we're having in CIDP. Now having said that, we, of course, are monitoring this ourselves and are in conversations with the agency as per normal procedures.

因此，真正確定因果關係或絕對不做利益風險陳述並沒有真正的用處。我這樣說的原因是，曝光度的快速增加可能會導致報道的增加。如您所見，這也許就是我們在 CIDP 取得成功的原因之一。話雖如此，我們當然也在親自監控此事，並按照正常程序與該機構進行對話。

And in that sense, we have to note that this NIS, as it's called, was issued as a potential safety signal that requires monitoring. Now NIS have two levels: one, important potential, which has a much shorter window of evaluation or potential, which typically has a 12-month evaluation period. And at the end of that, in many cases, nothing happens or the monitoring period is extended.

從這個意義上講，我們必須注意到，這個所謂的 NIS 是作為需要監控的潛在安全訊號發布的。現在 NIS 有兩個層級：一是重要潛力，其評估窗口或潛力要短得多，通常有 12 個月的評估期。最後，在很多情況下，什麼事也沒有發生，或是監測期延長。

But of course, we can never exclude that we have to have a dialogue with the agency about a label change. But at this point, we don't have sight on that. Given the rate we observe ourselves as being a sponsor of less than 2% of these sort of events with over 2,500 patients exposed, we feel that the current benefit-risk ratio on CIDP is maintained.

但當然，我們絕不能排除我們必須與該機構就標籤更改進行對話。但目前我們還沒有看到這一點。鑑於我們觀察到自己是此類活動中不到 2% 的贊助商，並且有超過 2,500 名患者受到影響，我們認為 CIDP 的當前收益風險比是可以維持的。

Thank you, Karl (sic - Luc) And just to add to that, in terms of the feedback from prescribers, I would say the early experience that we hear about from prescribers is very positive. And we continually hear that, that real-world experience mirrors what we saw in ADHERE in the clinical trial.

謝謝你，卡爾（原文如此 - 盧克）另外，就處方者的反饋而言，我想說，我們從處方者那裡聽到的早期經驗是非常積極的。我們不斷聽到說，現實世界的經驗反映了我們在臨床試驗中看到的 ADHERE 情況。

The other positive signal that we see is that we are investing in patient activations and many patients are going in to ask their neurologists about VYVGART for CIDP. And what we're seeing is a very high grant rate when the patients ask. And what that means and what that demonstrates is that neurologists have confidence and belief in VYVGART in both the efficacy and the safety profile of the medicine. And that's obviously translating into the strong patient growth numbers that we're seeing in the quarter as well. So I think really positive early experience.

我們看到的另一個積極信號是，我們正在投資於患者激活，許多患者正在向他們的神經科醫生詢問有關用於 CIDP 的 VYVGART 的資訊。我們看到的是，當患者提出要求時，批准率非常高。這意味著並表明神經科醫生對 VYVGART 藥物的功效和安全性充滿信心和信心。這顯然也轉化為我們在本季看到的強勁的患者成長數量。所以我認為早期的經驗確實是正面的。

Thanks for the question.

謝謝你的提問。

Myles Minter, William Blair.

邁爾斯·明特、威廉·布萊爾。

Hey. Congrats on the quarter. Thanks for the question. It's following up on this actually. I think at AAN, you disclosed the 1,316 patients as of January 31 on Hytrulo CIDP, the worsening rate was 3.3%. It's pretty low now that you've got greater than 2,500 patients on therapy. Can you update us on that rate there? Or do you plan to update us? And then the second question is just on the Phase 4 IPIg to efgartigimod switch study in CIDP. Are we still expecting data for that this year?

嘿。恭喜本季取得佳績。謝謝你的提問。它實際上正在跟進此事。我認為在 AAN，您披露了截至 1 月 31 日使用 Hytrulo CIDP 的 1,316 名患者，惡化率為 3.3%。目前接受治療的患者人數已超過 2,500 人，這個數字相當低。您能向我們更新那裡的利率嗎？或者您打算向我們更新資訊嗎？第二個問題是關於 CIDP 中的第 4 階段 IPIg 至 efgartigimod 轉換研究。我們今年還期待這方面的數據嗎？

Yeah, Myles, I'm going to give you a very brief answer. So we are monitoring all AEs, of course, in the real world. This specific AE of severe CIDP worsening is actually not going up. It's rather stable or going down. It is a very small number. And I want to remind the audience that in the CIDP setting specifically, any therapeutic switch you would consider as a physician carries the risk of CIDP worsening. This is simply a known fact in the space.

是的，邁爾斯，我會給你一個非常簡短的答案。當然，我們在現實世界中監控所有的不良事件。這種嚴重 CIDP 惡化的特定 AE 實際上並沒有增加。它相當穩定或正在下降。這是一個非常小的數字。我想提醒觀眾，特別是在 CIDP 環境中，身為醫生，您考慮的任何治療轉換都存在 CIDP 惡化的風險。這只是該領域已知的事實。

What I do want to call out is the transformative benefit which we see for VYVGART in so many patients. The amount of patient anecdotes which are reaching us every week in terms of improvement in functionality, I think, is just impressive.

我確實想指出的是，我們看到 VYVGART 在許多患者身上帶來了變革性的益處。我認為，每週我們收到的有關功能改善的患者軼事數量令人印象深刻。

And to conclude, from where we sit, we don't see a real benefit-risk change. The switch study, I think, is well on track. It's enrolling, and we will keep you updated when the study progresses, finalizes. And as you can expect, data will be reported at the clinical conference.

總而言之，從我們目前的情況來看，我們沒有看到真正的收益風險變化。我認為轉換研究進展順利。它正在招生，當研究進展和結束時，我們會及時通知您。正如您所料，數據將在臨床會議上報告。

Thanks for the question.

謝謝你的提問。

Samantha Semenkow, Citi.

Samantha Semenkow 的花旗銀行。

Hi, good morning. Thanks very much for taking the questions. Just one on the pipeline for me. I'm wondering if you could just share some context on your decision to advance the clinical development of ARGX-119. Just the data you've seen so far in CMS, does it increase your enthusiasm as well for ALS and SMA? And just more broadly, I'm wondering what the overall market opportunity across indications for 119 is that you're envisioning? Thank you very much.

嗨，早安。非常感謝您回答這些問題。對我來說，管道上只有一個。我想知道您是否可以分享一些關於推進 ARGX-119 臨床開發的決定的背景。您目前在 CMS 中看到的數據是否也增加了您對 ALS 和 SMA 的熱情？更廣泛地說，我想知道您所設想的 119 各個適應症的整體市場機會是什麼？非常感謝。

Hey, Samantha, thank you for the question on 119, our latest kid on the block. And we have the benefit of having Luc here. So Luc, could you contextualize the go decision for CMS, please? And what is your view on potential read-through on other indications? And then I will briefly summarize how we look at the totality of the opportunity, okay?

嘿，薩曼莎，謝謝你在 119 號節目中提問，他是我們最新的嘉賓。我們有幸有 Luc 來到這裡。那麼 Luc，您能否為 CMS 詳細闡述這個決策？您對其他適應症的潛在解讀有何看法？然後我將簡要總結我們如何看待整個機會，好嗎？

Yes, certainly. So we chose indication CMS, which is an ultra-rare indication because that for us will be the best in human proof of the biology at work of an agonistic antibody for a mask. And therefore, we designed a small also driven by the availability of patients, but highly densely monitored with readouts and also building intra-patient dose escalation to come to the answer is the proof of biology.

是的，當然。因此，我們選擇了 CMS 這一適應症，這是一種極其罕見的適應症，因為對我們來說，這將是人類對口罩激動劑抗體的生物學作用的最佳證明。因此，我們設計了一個小型的、同樣受患者可用性驅動的、但高度密集地監測讀數的裝置，並且還建立了患者內部劑量遞增裝置，以得出答案，這是生物學的證明。

And so the basis for our decision is that we did find this. We found that on a clinically relevant endpoint correlated with a digital endpoint and also with a clinician-observed strength in the leg. And so the endpoints are 6 minutes walk test, a digital measure of cadence and strength on the MG that we have that signature with an increasing ability to walk longer distances in these patients.

因此，我們做出這項決定的依據是我們確實發現了這一點。我們發現，臨床相關終點與數字終點以及臨床醫生觀察到的腿部力量有關。因此，終點是 6 分鐘步行測試，這是 MG 上節奏和強度的數字測量，我們發現該特徵表明這些患者行走更長距離的能力不斷增強。

And that really made us say we have proof of biology, and we're going to continue the path in CMS as far as we can. With respect to read-through to the other indications, there should be some correlation, but there are two different things here. One is that the diseases that we're testing, ALS and SMA are, of course, complex with different biology. So we should not simply assume that there's a 100% read-through. And that's why we designed ALS development with a POC testing multiple doses.

這確實讓我們說我們有生物學證據，我們將盡可能地繼續在 CMS 中前進。對於其他適應症的解讀，應該會有一定的關聯，但這裡有兩件不同的事情。一是我們正在測試的疾病，ALS 和 SMA 當然很複雜，生物學特性也不同。所以我們不應該簡單地假設閱讀率是 100%。這就是我們設計 ALS 開發時採用 POC 測試多劑量的原因。

SMA is currently being designed.

SMA 目前正在設計中。

Thank you, Luc. So if we zoom out on 119, it's a typical argenx molecule or program. It comes straight from the innovation playbook, right? So a novel target where we collaborate with the world experts, a hell of an antibody, which we made and then the potential pipeline in a product because we're now already in three indications, CMS, ALS, SMA.

謝謝你，盧克。因此，如果我們縮小 119，它就是一個典型的 argenx 分子或程序。它直接來自創新劇本，對嗎？因此，我們與世界專家合作開發了一個新目標，製造了一個非常有效的抗體，然後將其作為產品的潛在管道，因為我們現在已經有三種適應症，即 CMS、ALS 和 SMA。

But just think about a molecule which has the potential to rejuvenate the neuromuscular junction that has exciting potential across a number of nerve regeneration indications, but also muscle diseases. So stay tuned. We're still assessing further potential in more indications to come. And thanks for the question on 119.

但試想一下，一種具有恢復神經肌肉接頭潛力的分子，它不僅對多種神經再生指徵有令人興奮的潛力，而且對肌肉疾病也有潛力。敬請關注。我們仍在評估未來更多跡象的進一步潛力。感謝您在 119 號問題上提出的問題。

Gavin Clark-Gartner, Evercore.

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

Hey, guys. Congrats on the great progress. So this week, AstraZeneca noted that they expect 40% of MG patients to be on self-administered therapies by 2030. I'm curious if this aligns with your market research and very early PFS experience seen to date. Thanks.

嘿，大家好。祝賀你取得巨大進步。因此本週，阿斯特捷利康指出，他們預計到 2030 年 40% 的 MG 患者將接受自我治療。我很好奇這是否與您迄今為止的市場研究和早期的 PFS 經驗相符。謝謝。

Yeah. Thank you, Gavin. And it's not to comment on market research results from colleagues in the field. I think what we tend to agree is that self-administration is important for patients, as we are actually already evidencing today. And remember the R&D Day of last July, where we basically increased our expectations for the total 10 in MG.

是的。謝謝你，加文。這並不是對該領域同事的市場研究結果進行評論。我認為我們傾向於同意的是，自我管理對患者很重要，正如我們今天實際上已經證明的那樣。還記得去年 7 月的研發日，我們基本上提高了對 MG 總數 10 的預期。

PFS and self-administration actually is going right after that box, which we showed of 23,000 extra patients, which we added in addition to the initial 17,000, which we had at the start of the MG launch. So very, very important. But we cannot comment on these numbers specifically.

PFS 和自我管理實際上緊隨其後，我們展示了 23,000 名額外患者，這些患者是我們在 MG 推出之初的 17,000 名患者的基礎上添加的。非常非常重要。但我們無法具體評論這些數字。

Thanks for the question.

謝謝你的提問。

Andy Chen, Wolfe Research.

安迪‧陳 (Andy Chen)，沃爾夫研究公司 (Wolfe Research)。

Hi, this is Emma on for Andy. Thanks for taking our questions. Congrats on the strong quarter. In the press release, it's mentioned that you're still in the early stages of MG and CIDP launches. We're just curious how you guys know the launch is still in early stages. Is it because prescriber coverage is still a small percent? Or are new prescriptions still rising? Or are new numbers still just far away from theoretical TAM? Thank you.

大家好，我是 Emma，為 Andy 服務。感謝您回答我們的問題。恭喜本季業績強勁。新聞稿中提到，你們仍處於 MG 和 CIDP 發布的早期階段。我們只是好奇你們如何知道發布仍處於早期階段。是因為處方覆蓋率仍然很小嗎？還是新處方仍在增加？還是新的數字與理論 TAM 還相差甚遠？謝謝。

Yeah. Thanks for the question. And I think it's all of those factors. So let's take it step by step. So first, in MG, I mean, we're 14 quarters in. But what we continue to see is that strong quarter-over-quarter patient growth, as you said. And I think we're fueling that growth with new innovations, for example, bringing a prefilled syringe to the market.

是的。謝謝你的提問。我認為所有這些因素都有。讓我們一步一步來。首先，在 MG，我的意思是，我們已經有 14 個季度了。但正如您所說，我們繼續看到患者數量逐季強勁增長。我認為我們正在透過新的創新來推動這種成長，例如，將預充式註射器推向市場。

And as Tim just talked through, we see that the biologics market -- share of market will grow in MG. We estimate that it will grow by about 25,000 patients. We also continue to invest in VYVGART and getting the broadest label for VYVGART. So we have our seronegative and our ocular MG studies. And that means that the total addressable market in MG ends up being around 60,000 patients. So that's where we get to. We're still -- despite being 14 quarters in with continuous growth, we're still early in the market opportunity for MG.

正如提姆剛才所說，我們看到生物製劑市場－重症肌無力的市佔率將會成長。我們估計患者人數將增加約25,000人。我們也持續對VYVGART進行投資，並為VYVGART取得最廣泛的標籤。因此，我們有血清陰性和眼部 MG 研究。這意味著 MG 的總目標市場最終將達到約 60,000 名患者。這就是我們要討論的。儘管已經連續 14 個季度保持成長，但我們仍處於 MG 市場機會的早期階段。

Likewise, with CIDP, we're only one year into launch with CIDP, and we're seeing continued strong uptake in the market. We've said that the TAM there is 12,000 patients, so we still have a way to grow. And I think over time and certainly over the long term, you can start to imagine that with more innovation coming to market, we just talked about empasiprubart potentially for CIDP, you can start to see that, that market will start to grow beyond the 12,000 as well over the long term.

同樣，我們推出 CIDP 僅一年時間，就看到市場持續強勁成長。我們說過，那裡的總患者人數為 12,000 名，因此我們仍有成長空間。我認為隨著時間的推移，當然從長遠來看，您可以開始想像，隨著更多創新進入市場，我們剛才談到 empasiprubart 對 CIDP 的潛在影響，您可以開始看到，從長遠來看，該市場也將開始增長到 12,000 以上。

So I think if you take a step back, when you look at our continued growth that we've delivered since the VYVGART launch and the fact that we have six Phase 3 studies reading out in the next 18 months, you can see that as a company, we're very much on the early side of the growth trajectory.

因此，我認為，如果你退一步來看，當你看到我們自 VYVGART 推出以來所取得的持續增長，以及我們在未來 18 個月內有六項 3 期研究成果的事實時，你就會發現，作為一家公司，我們正處於增長軌蹟的早期階段。

Thanks for the question.

謝謝你的提問。

Douglas Tsao, HC Wainwright.

Douglas Tsao、HC Wainwright。

Hi, everyone. Thanks for taking the questions and on all the progress. I'm just curious, in terms of the PFS, we've talked to some physicians who have said that they've had some challenge in terms of getting patients access to the product. I'm just curious, is that sort of one-offs? Or is there still a situation where perhaps demand is sort of exceeding the available supply?

大家好。感謝您回答這些問題並通報所有進度。我只是好奇，就 PFS 而言，我們與一些醫生進行了交談，他們說他們在讓患者獲得產品方面遇到了一些挑戰。我只是好奇，這是一次性的事嗎？或是否仍有需求超過供應的情況？

Yeah. Thanks for the question. Look, I think it's -- we're only one quarter into launch. And normally, with these -- into the launch of PFS, we always say that with any new launch, it takes about two quarters to get access in place. And I think we sometimes forget about that because our access team does such a great job and always beats expectations on that.

是的。謝謝你的提問。瞧，我認為──我們只進行了四分之一的發布。通常情況下，在推出 PFS 時，我們總是說，任何新產品的推出都需要大約兩個季度的時間才能獲得存取權限。我認為我們有時會忘記這一點，因為我們的訪問團隊做得非常出色，並且總是超出預期。

So of course, in early stages, there are going to be some questions like that or some feedback while we get access into place. What we see though is very -- is that we have 70% of commercial lives covered with prefilled syringe. And in general -- and we're adding to that all the time. In general, the feedback that we get from the field is that prescribers are very pleased with how quickly we're getting access for patients to prefilled syringe. I think that's reflected in the strong uptake.

因此，當然，在早期階段，當我們獲得存取權限時，會出現一些類似的問題或一些回饋。然而，我們看到的是——70% 的商業生活都覆蓋了預充式註射器。總的來說 — — 我們一直在不斷增加這一點。總體而言，我們從現場得到的回饋是，處方人員對於我們為患者提供預充式註射器的速度感到非常滿意。我認為這反映在強勁的接受度上。

And Douglas, if I can add, we definitely have enough inventory available. It's not driven by inventory.

道格拉斯，如果我可以補充的話，我們肯定有足夠的庫存。它不是由庫存驅動的。

Victor Floch, BNP Paribas.

法國巴黎銀行的維克多‧弗洛赫。

Thanks for taking my question. I have basically just one question on ITP, actually. I was wondering if you could update us on the feedback since launch. Because if I remember correctly, your ambition at the time was to position VYVGART as the first TPO-RA option basically as a fourth-line treatment. So I was basically wondering if the physician feedback so far in Japan basically supports this? And if by any chance, you could also share your market share in this market in Japan. Thanks so much.

感謝您回答我的問題。實際上，我對 ITP 基本上只有一個問題。我想知道您是否可以向我們更新自發布以來的反饋。因為如果我沒記錯的話，您當時的目標是將 VYVGART 定位為第一個 TPO-RA 選項，基本上作為四線治療。所以我基本上想知道到目前為止日本的醫生回饋是否基本上支持這一點？如果有機會的話，您也可以在日本的這個市場分享您的市場份額。非常感謝。

Yeah. Thank you, Victor, for the question on ITP. So the launch in Japan for ITP is actually going well. And what we find exciting is to see that in the real world, the clinical data are actually perfectly merit. So we see about a 50% response rate. The drug is landing first in the last line of ITP patients after they fail steroids, IVIg and TPOs. So very refractory patient population and still a very nice 50% response rate.

是的。感謝 Victor 提出有關 ITP 的問題。因此，ITP 在日本的推出進展順利。令人興奮的是，我們發現在現實世界中，臨床數據實際上是完全有價值的。因此我們看到的回應率大約是 50%。在類固醇、IVIg 和 TPO 治療失敗後，該藥物將成為 ITP 患者的最後一線治療藥物。因此，對於非常難治的患者群體，仍然有非常好的 50% 的反應率。

And if patients respond, just like similar in the clinical trial, they respond very quickly, and the safety profile of the product is also differentiating. I mean physicians badly need a fast-acting safe drug. So I think the drug is landing very well. Uptake is nice, and I think we will be gradually moving our way up in that treatment paradigm. So all in all, according to plan.

如果患者有反應，就像臨床試驗一樣，他們的反應非常快，而且產品的安全性也有所差異。我的意思是醫生迫切需要一種快速起效的安全藥物。所以我認為這種藥物效果很好。接受度很好，我認為我們將逐步在這種治療模式下進步。總而言之，一切都按照計畫進行。

Thanks for the question.

謝謝你的提問。

Charles Pitman-King, Barclays.

巴克萊銀行的查爾斯·皮特曼·金 (Charles Pitman-King)。

Hi, guys. Thanks for taking my questions. Just a final one, please, just on the kind of pricing dynamics. Just thinking about the Medicare process for argenx. I'm just wondering what potential quarterly fluctuation is there between assuming a Medicare discount and then kind of rightsizing it?

嗨，大家好。感謝您回答我的問題。最後，請問一下關於定價動態的問題。只是在思考 argenx 的醫療保險流程。我只是想知道，假設醫療保險折扣和適當調整其規模之間可能存在哪些季度波動？

What kind of visibility do you have to have confidence in your comments that the net price per patient is going to remain flat going forward as we think about trying to forecast your sales on a quarterly basis going forward and any potential fluctuations that might come as a result of that? Thank you.

當我們考慮嘗試預測未來季度的銷售額以及由此可能產生的任何波動時，您需要具備什麼樣的洞察力才能對您的評論充滿信心，即每位患者的淨價格未來將保持平穩？謝謝。

Yeah, Charles, let me kick it off and then hand over to Karl. But we will not get into the complexities of the US healthcare system in today's call. But I think what I want you all to remember from the call is the confirmation right, Karl, that the net contribution for an MG and CIDP patient is actually not changing. And if and when we think it's about to change, we will definitely flag it to this audience.

是的，查爾斯，讓我先開始，然後交給卡爾。但我們今天的電話會議不會討論美國醫療保健系統的複雜性。但我想讓大家記住的是，卡爾，通話中確認的是，MG 和 CIDP 患者的淨貢獻其實並沒有改變。如果我們認為它即將發生變化，我們一定會向觀眾發出警告。

Anything you would like to add?

您還有什麼要補充的嗎？

No. I think that's all. Thanks, Charles. Thanks for the question.

不。我想就這些了。謝謝，查爾斯。謝謝你的提問。

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

今天的電話會議到此結束。感謝您的參與，您現在可以斷開連接了。