argenx SE (ARGX) 2026 Q1 法說會逐字稿

Good morning. My name is David, and I'll be your conference operator today. I would like to welcome everyone to the call.

早安。我叫 David，今天將擔任本次會議的接線員。歡迎各位參加本次電話會議。

(Operator Instructions)

（接線員指示）

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

我想介紹企業事務副總裁 Beth DelGiacco。您現在可以開始會議。

Thank you. A press release was issued earlier today with our first-quarter 2026 financial results and business update. This can be found on our website along with the presentation for today's webcast.

謝謝。我們已於今日稍早發布新聞稿，內容包含 2026 年第一季財務結果與業務更新。您可在我們的網站上找到該新聞稿，以及今日網路直播的簡報資料。

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 development, regulatory time lines, the potential success of our product candidates, financial projections and upcoming milestones. 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.

在我們開始第 2 張投影片之前，我想提醒各位，本次電話會議中可能會提出前瞻性陳述。這些陳述可能包括我們對未來的預期、臨床開發、法規時程、產品候選藥物的潛在成功、財務預測以及即將到來的里程碑等。實際結果可能與這些陳述所示存在重大差異。除非法律要求，argenx 不負有更新與未來相關陳述或使該等陳述與實際結果相符之義務。

I'm very excited to be joined on the call today by Karen Massey, our new Chief Executive Officer. Today is her first official day on the job, marking a very important milestone in the story of argenx. We're also joined by Karl Gubitz, Chief Financial Officer; and Sandrine Piret-Gerard, Chief Commercialization Officer. I will now turn the call over to Karen.

我非常高興今天的電話會議邀請到我們的新任執行長 Karen Massey 一同參與。今天是她正式上任的第一天，這標誌著 argenx 發展歷程中的一個非常重要的里程碑。我們也邀請到財務長 Karl Gubitz，以及商業化長 Sandrine Piret-Gerard。接下來我將把電話會議交給 Karen。

Thanks, Beth, and welcome, everyone. I'll begin on slide 3. I want to start by saying what a privilege it is to step into the CEO role at this point in argenx journey. I joined the company three years ago for the culture, the science and the opportunity to help build a different kind of immunology innovation company alongside an exceptional team, one that scales innovation through collaboration, delivers meaningful impact for patients and creates lasting value for all stakeholders.

謝謝你，Beth，也歡迎各位。我將從第 3 張投影片開始。我想先說，在 argenx 旅程的此刻接任執行長一職，是一項莫大的榮幸。三年前我加入公司，是因為這裡的文化、科學，以及與一支卓越團隊並肩打造一種不同型態免疫學創新公司的機會——透過協作擴大創新規模、為病患帶來有意義的影響，並為所有利害關係人創造長期價值。

I'm excited to carry forward Vision 2030, our road map for growth and value creation at a moment when we have so much opportunity across the near, medium and long term. Near term, we see continued growth with VYVGART across both MG and CIDP. While we see the typical effect of seasonality, the underlying demand trends remain very strong and continue to point to a business that is both growing and expanding. I'm particularly encouraged by our new patient demand. Q1 was amongst our highest quarters ever.

我很期待在我們於短期、中期與長期都擁有大量機會的此刻，持續推進「願景 2030」（Vision 2030）——我們的成長與價值創造路線圖。短期而言，我們預期 VYVGART 在 MG 與 CIDP 兩個適應症上將持續成長。儘管我們看到典型的季節性影響，但基本需求趨勢仍然非常強勁，並持續指向一個同時在成長與擴張的業務。我尤其受到新病患需求的鼓舞。第一季是我們歷來表現最好的季度之一。

Our leadership in neuromuscular was on display at AAN last month, where consistently strong efficacy and safety data reinforced why physicians should treat broadly and earlier with VYVGART in both MG and CIDP. Importantly, that growing confidence amongst neurologists positions VYVGART well for the next phase of growth with potential expansion into seronegative, ocular and pediatric MG population based on the strength of ADAPT SERON, OCULUS and Jr.

我們在神經肌肉領域的領導地位於上月的 AAN 年會上充分展現；一致且強勁的療效與安全性數據，進一步強化了為何醫師應在 MG 與 CIDP 中更廣泛且更早使用 VYVGART 進行治療。重要的是，神經科醫師日益提升的信心，使 VYVGART 在下一階段成長中具備良好定位；基於 ADAPT SERON、OCULUS 與 Jr. 的強勁結果，VYVGART 有望擴展至血清陰性、眼肌型以及兒科 MG 族群。

When I consider the growth over the medium term, we see strong runway of launches ahead, reflecting the scale of our ambition and the depth of opportunity across our pipeline. We're advancing into rheumatology with readouts in autoimmune myositis and Sjogren's. And we expect the first readout and potential launch of our second medicine, empasiprubart in MMN.

就中期成長而言，我們看到未來仍有強勁的上市動能，反映出我們的雄心規模以及產品線（pipeline）中深厚的機會。我們正推進至風濕免疫領域，並將在自體免疫性肌炎與乾燥症（Sjogren's）取得讀出結果。同時，我們也預期在 MMN 上取得 empasiprubart 的首次讀出結果，並有機會推出我們的第二款藥物。

Lastly, we're investing in our future. We're building a pipeline by sourcing novel biology relevant in diseases of high unmet need, positioning us to sustain long-term growth and reach thousands more patients.

最後，我們正在投資未來。我們透過引進與高度未被滿足需求疾病相關的新穎生物學來建立產品線，使我們得以維持長期成長，並觸及更多、以千計的病患。

Slide 4. Our strategy is ambitious, but it's one we know how to execute. Vision 2030 is built on a proven immunology innovation model that has already delivered a strong foundation. Today, we have 5 clinical stage molecules that follow our model, first-in-class against a novel immune target and collectively addressing more than 15 distinct diseases. Empasiprubart is positioned to be the second medicine we bring to patients.

第 4 張投影片。我們的策略雄心勃勃，但也是我們知道如何落地執行的策略。「願景 2030」建立在一個已被驗證的免疫學創新模式之上，而該模式已奠定堅實基礎。目前我們有 5 個臨床階段分子遵循此模式：針對新穎免疫標的的同類首創（first-in-class），合計涵蓋超過 15 種不同疾病。Empasiprubart 有望成為我們帶給病患的第二款藥物。

Like VYVGART, we built this molecule very intentionally to target C2, leveraging novel biology insights from leading experts in complement biology. Through empasiprubart's development, we've also unraveled critical insights into MMN biology that position us to fully transform the treatment paradigm. The Phase III readout is expected in 4Q, bringing us closer to launching our second medicine.

如同 VYVGART，我們非常有目的性地打造此分子以鎖定 C2，並運用補體生物學領域頂尖專家的新穎生物學洞見。在 empasiprubart 的開發過程中，我們也解開了關於 MMN 生物學的關鍵洞見，使我們得以全面改變治療典範。第三期試驗讀出結果預計於第 4 季公布，讓我們更接近推出第二款藥物。

In MMN, we see a familiar opportunity, build and lead the category, much as we did with VYVGART and MG, transforming the entire disease space. In CIDP, we have the same ambition. We continue to grow our market share with VYVGART and are generating extensive real-world insights as patient experience increases.

在 MMN 上，我們看到一個熟悉的機會：建立並引領該類別，就如同我們以 VYVGART 在 MG 上所做的一樣，進而改變整個疾病領域。在 CIDP 上，我們也有同樣的企圖。我們持續以 VYVGART 提升市占率，並隨著病患使用經驗的累積，產生大量真實世界洞見。

And now by advancing a second targeted approach with empasiprubart, we will deepen our understanding of the underlying biology, helping us learn which patients benefit most from which treatment and positioning us to secure leadership in CIDP.

而現在，透過推進以 empasiprubart 為代表的第二種精準標靶策略，我們將加深對其基礎生物學的理解，協助我們辨識哪些病患最能從哪種治療中受益，並使我們有利於在 CIDP 中確立領導地位。

Slide 5. Across VYVGART and empasiprubart, we're solidifying our leadership in neuromuscular diseases and amongst the physician and patient communities. One of the most critical therapeutic area expansion for argenx is into rheumatology, and this starts with an important Phase III readout in autoimmune myositis in the third-quarter. This is a disease with significant unmet need, defined by progressive muscle weakness that makes basic daily activities really challenging, if not impossible. There are no approved treatments in necrotizing myositis.

第 5 張投影片。在 VYVGART 與 empasiprubart 兩項產品上，我們正在鞏固於神經肌肉疾病領域，以及在醫師與病患社群中的領導地位。對 argenx 而言，最關鍵的治療領域擴張之一是進入風濕免疫領域，而這將從第三季自體免疫性肌炎的一項重要第三期讀出結果開始。這是一種具有顯著未被滿足需求的疾病，其特徵為進行性肌肉無力，使基本日常活動變得非常困難，甚至不可能。在壞死性肌炎（necrotizing myositis）方面，目前尚無核准治療。

And in dermatomyositis, where serious symptoms can emerge in the muscle, in the skin and in other organ systems, the complexity of the disease means more innovation is needed. VYVGART has the potential to be the first truly targeted therapy in this indication, targeting autoantibodies against proteins that are highly expressed in the muscle. In the case of positive data, we plan to take an approach similar to what we've done with MG and CIDP because the myositis opportunity has similar attributes overlapping physician community, long established conventional treatment patterns and our goal to transform patient outcomes and become the number one prescribed biology.

而在皮肌炎（dermatomyositis）中，嚴重症狀可能出現在肌肉、皮膚以及其他器官系統；疾病的複雜性意味著需要更多創新。VYVGART 有潛力成為此適應症中第一個真正的精準標靶療法，鎖定針對在肌肉中高度表現蛋白的自體抗體。若數據為正面，我們計畫採取與 MG 與 CIDP 類似的策略，因為肌炎的機會具備相似特性：醫師社群高度重疊、既有傳統治療模式已長期建立，以及我們希望改變病患預後並成為處方量第一的生物製劑之目標。

Slide 6. We've significantly broadened the number of clinical candidates across our pipeline in the last 12 months, and you can expect to see this cadence continue. Adimanebart is in Phase III in CMS. ARGX-121 is in Phase II development in IgAN with more indications to follow. And our long-term FcRn portfolio is taking shape with ARGX-213 ready for Phase III and ARGX-124 to follow.

第 6 張投影片。在過去 12 個月中，我們已大幅擴增產品線中的臨床候選藥物數量，您也可以預期這樣的節奏將持續下去。Adimanebart 正在 CMS 的第三期試驗中。ARGX-121 正在 IgAN 的第二期開發中，後續還會拓展至更多適應症。而我們的長期 FcRn 產品組合也正在成形：ARGX-213 已準備進入第三期，ARGX-124 將緊接其後。

To expand our pipeline at a cadence that will sustain our long-term trajectory, we need to deliberately expand the ecosystem from which we source novel biology. We'll double down on collaborations and licensing agreements with academic institutions and biopharma companies alongside acquisitions and strategic investments, giving us the flexibility to engage in whatever form accelerates innovation into our pipeline. The establishment of our China entity supports this strategy, building on the clinical and commercial strength of our partnership with Zai Lab and embedding us into the local ecosystem to effectively access emerging innovation, accelerate evidence generation as part of our pipeline and product strategy and ultimately expand our impact in this region.

為了以可持續的節奏擴充產品線、支撐我們的長期成長軌跡，我們需要有意識地擴大取得新穎生物學的生態系來源。我們將加倍投入與學術機構及生技製藥公司的合作與授權協議，並結合併購與策略性投資，使我們能以任何可加速創新導入產品線的形式靈活參與。我們在中國設立實體支持此一策略：在與再鼎醫藥（Zai Lab）合作的臨床與商業優勢基礎上更進一步，並深度嵌入在地生態系，以有效取得新興創新、加速作為產品線與產品策略一部分的證據生成，並最終擴大我們在該地區的影響力。

As I step into this new role, I'm reminded of our purpose as a company, our patients. When we launched VYVGART fiveyears ago, we made a commitment to continue raising the standard of care for people living with myasthenia gravis. Today, with 17 consecutive quarters of growth, we continue to see that commitment translate into real-world impact in MG, in CIDP and in ITP. This is what gives me confidence in the opportunity ahead. We're deeply committed to our purpose, we're disciplined in our execution, and we'll be flexible in how we source innovation.

當我踏入這個新職務時，我再次想起我們作為一家公司的宗旨——我們的病患。五年前我們推出 VYVGART 時，我們承諾將持續提升重症肌無力患者的照護標準。今天，在連續 17 個季度成長之後，我們持續看到這份承諾在 MG、CIDP 與 ITP 上轉化為真實世界的影響。這正是讓我對前方機會充滿信心的原因。我們對使命深度投入、在執行上嚴謹自律，並且會在取得創新來源的方式上保持彈性。

With that, I'll turn the call over to Karl to walk through our financial performance and outlook.

接下來，我把電話交給 Karl，請他帶大家回顧我們的財務表現與展望。

Thank you, Karen. Slide 7. Product net sales for the first-quarter were $1.3 billion, representing 63% year-over-year growth. By region, we generated $1.1 billion in the US, $67 million in Japan, $112 million in the rest of the world and $12 million in product supplied to Zai Lab in China. The 1% quarter-over-quarter growth reflects the impact of Q1 seasonality in the US and is aligned with our expectations. Gross to net adjustments and net pricing in the US remained consistent with the prior quarter.

謝謝你，Karen。第 7 張投影片。第一季產品淨銷售額為 13 億美元，年增 63%。按地區來看，美國貢獻 11 億美元、日本 6,700 萬美元、世界其他地區 1.12 億美元，以及供應給中國再鼎醫藥（Zai Lab）的產品 1,200 萬美元。季增 1% 反映了美國第一季的季節性影響，並符合我們的預期。美國的毛利到淨利（gross-to-net）調整與淨定價水準與前一季一致。

Next slide, slide 8. Total operating expenses in the first-quarter were $919 million, representing a decrease of $36 million compared to the fourth-quarter of last year. Research and development expenses increased by $68 million quarter-over-quarter, principally driven by CMC investments to support the expected launch of a VYVGART subcu auto-injector in 2027, continued development of efgartigimod across several indications and advancing our pipeline assets. These strategic investments will deliver long-term growth through innovation in providing transformational outcomes to patients.

下一張投影片，第 8 張。第一季總營業費用為 9.19 億美元，較去年第四季減少 3,600 萬美元。研發費用季增 6,800 萬美元，主要由 CMC 投資所帶動，以支援預計於 2027 年推出的 VYVGART 皮下（subcu）自動注射器，同時也包括 efgartigimod 在多個適應症上的持續開發，以及推進我們的管線資產。這些策略性投資將透過創新、為病患帶來具變革性的治療成果，進而帶動長期成長。

SG&A decreased 17% quarter-over-quarter, reflecting higher discretionary expenses in the fourth-quarter of last year. We delivered an operating profit of $394 million in the first-quarter and operating margin of 30%, representing 183% year-over-year growth. With revenue growth exceeding operating expense growth, we are on a clear path of continued margin expansion, demonstrating the operating leverage we are building as VYVGART scales and we advance our pipeline.

SG&A 較前一季下降 17%，反映去年第四季較高的可自由裁量支出。第一季我們實現營業利益 3.94 億美元，營業利益率 30%，年增 183%。在營收成長超過營業費用成長的情況下，我們正走在持續擴大利潤率的明確道路上，展現出隨著 VYVGART 規模擴大及我們推進管線所建立的營運槓桿。

Tax for the quarter is in line with expectations at 14% of profit before tax. We ended the quarter with a cash balance of $4.9 billion, including cash, cash equivalents and current financial assets, an increase of more than $400 million from the beginning of the year. This strong financial position gives us significant flexibility as we deploy capital to fuel long-term growth.

本季稅負符合預期，為稅前利益的 14%。季末現金餘額為 49 億美元（包含現金、約當現金與流動金融資產），較年初增加超過 4 億美元。這個強勁的財務狀況，讓我們在部署資本以推動長期成長時具備高度彈性。

Our capital allocation priorities are clear: maximizing the VYVGART commercial opportunity, advancing our pipeline, strengthening our supply chain and pursuing business development opportunities to source the novel biology that will fuel our long-term growth. With our profitability profile, we are investing from a position of strength to deliver on Vision 2030.

我們的資本配置優先順序很明確：最大化 VYVGART 的商業機會、推進我們的研發管線、強化供應鏈，並尋求商務開發機會，以取得能驅動長期成長的新穎生物學創新來源。憑藉我們的獲利能力輪廓，我們將在優勢基礎上進行投資，以實現 Vision 2030。

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

現在我把電話交給 Sandrine，她將就商業面提供更詳細的說明。

Thank you, Karl. I'll begin on slide 9. Over the past several months, I have spent a lot of time with our field teams and meeting with physicians and patients. What stood out to me is how argenx translates differentiated science into execution in the market, from securing access to building physician confidence in VYVGART in MG, CIDP as well as in ITP in Japan and to delivering white glove patient support designed to help patients start and stay on therapy. This level of disciplined execution and building trust among our core stakeholders will be critical as we approach several important growth catalysts.

謝謝你，Karl。我將從第 9 張投影片開始。過去幾個月，我花了很多時間與我們的一線團隊相處，並與醫師及病患會面。最讓我印象深刻的是，argenx 如何把具差異化的科學轉化為市場上的執行力——從確保可近性、在 MG 與 CIDP 建立醫師對 VYVGART 的信心，以及在日本的 ITP 推動採用，到提供「白手套」等級的病患支持，協助病患開始治療並持續用藥。這種高度自律的執行力，以及在核心利害關係人之間建立信任，將在我們迎來多項重要成長催化劑之際至關重要。

Today, I'll focus on what's driving our performance and where we see the most meaningful opportunities to build from here under Vision 2030.

今天我將聚焦於推動我們表現的因素，以及在 Vision 2030 架構下，我們認為從此刻起最具意義、最值得加碼的機會所在。

Slide 10. We continue to deliver on our long-term growth strategy in 2026 with strong momentum across all indications and all regions. Our fundamentals are incredibly strong. Now four years into MG and two years into CIDP launch, we see no signs of demand slowing. In the first-quarter, new patient starts were amongst the highest since launch, and we continue to have an expanding prescriber base, reflecting increasing confidence in VYVGART. We now have over 5,000 neurologists prescribing VYVGART in the US.

第 10 張投影片。2026 年，我們持續落實長期成長策略，在所有適應症與所有地區都展現強勁動能。我們的基本面非常強勁。在 MG 上市已四年、CIDP 上市已兩年，我們未看到需求放緩的跡象。第一季新病患啟用人數為上市以來最高水準之一，同時處方醫師基礎持續擴大，反映醫師對 VYVGART 的信心提升。目前在美國已有超過 5,000 位神經科醫師開立 VYVGART。

Since launching in CIDP, we have effectively doubled our prescriber base and each incremental physician meaningfully expands the number of patients we can reach. We are also seeing a change in prescribing behavior. Across both MG and CIDP, we are seeing a clear shift towards earlier use of VYVGART as physicians gain confidence with what it can offer patients. In the US, our market research now shows that four out of five HCPs prefer to start with VYVGART as the first targeted biologic in gMG.

自 CIDP 上市以來，我們的處方醫師基礎已有效翻倍，而每新增一位醫師都能顯著擴大我們可觸及的病患數。我們也看到處方行為正在改變。在 MG 與 CIDP 兩個領域，我們清楚看到隨著醫師對 VYVGART 能為病患帶來的效益更有信心，處方正明顯轉向更早期使用。在美國，我們的市場研究目前顯示，五位 HCP 中有四位偏好在 gMG 中以 VYVGART 作為第一個標靶生物製劑的起始治療。

New patient demand continues to build across both indications, driving consistent quarter-over-quarter patient growth. The prefilled syringe has materially changed the demand of VYVGART. By giving patients and physicians more flexibility, it continues to drive earlier adoption across MG and CIDP with 68% of PFS patients being new to VYVGART since launch.

兩個適應症的新病患需求持續累積，帶動病患人數穩定的逐季成長。預充式注射器（PFS）已實質改變 VYVGART 的需求型態。透過為病患與醫師提供更大的彈性，它持續推動 MG 與 CIDP 的更早期採用；自 PFS 上市以來，PFS 病患中有 68% 為首次使用 VYVGART 的新病患。

Slide 11. VYVGART is the number one prescribed biologic in gMG, a market that continues to grow. We still believe the biggest opportunity is ahead of us, particularly given the nearly 80% of MG patients who are not yet on a biologic. We know the VYVGART profile is resonating with physicians in this population and more than 70% of patients starting VYVGART today come directly from oral therapies. This continues to be the focus of our strategy to extend our leadership by reaching patients earlier in their treatment journey and expanding our reach to broader MG populations.

第 11 張投影片。VYVGART 是 gMG 中處方量排名第一的生物製劑，而該市場仍在持續成長。我們仍然相信，最大的機會在前方，特別是考量到近 80% 的 MG 病患尚未使用生物製劑。我們知道 VYVGART 的產品特性在這個族群的醫師間引起共鳴，而目前開始使用 VYVGART 的病患中，超過 70% 是直接從口服療法轉換而來。這仍是我們策略的重點：在病患治療旅程更早期觸及他們，並將觸及範圍擴大到更廣泛的 MG 族群，以延伸我們的領導地位。

The data we presented at AAN served as an important driver of this strategy, and we were excited by the positive response from neurologists. VYVGART is delivering fast, deep and sustained efficacy and safety across MG patients. This supports our ambition for VYVGART to be the targeted treatment of choice, simplifying decision-making for the HCP and underscoring VYVGART's growth potential. I want to share one patient story that underscores why this matters.

我們在 AAN 發表的數據，成為推動此策略的重要驅動因素，而神經科醫師的正面回饋也讓我們備受鼓舞。VYVGART 在 MG 病患中展現快速、深度且持久的療效與安全性。這支持我們的企圖：讓 VYVGART 成為首選的標靶治療，簡化 HCP 的決策流程，並凸顯 VYVGART 的成長潛力。我想分享一則病患故事，說明為何這件事如此重要。

Pam first presented with blurred vision and muscle weakness around her eye, but her ocular symptoms were not taken seriously, and she was unable to access appropriate treatment. As her disease progressed, she ultimately generalized, suffered a serious fall and was hospitalized. When Pam later started VYVGART, the impact was meaningful, helping us regain both mobility and independence. Our experience is a powerful reminder that ocular MG is not an eye disease. It can be profoundly debilitating and disrupt independence in ways that are often underestimated.

Pam 起初出現視力模糊與眼周肌肉無力，但她的眼部症狀未被嚴肅看待，因此無法獲得適當治療。隨著疾病進展，她最終轉為全身型，遭遇一次嚴重跌倒並住院。Pam 後來開始使用 VYVGART 後，效果十分顯著，幫助她重新獲得行動能力與獨立性。這段經歷有力提醒我們：眼肌型 MG 並不是眼科疾病。它可能造成深度失能，並以常被低估的方式破壞個人獨立生活能力。

Our positive ocular MG data, which demonstrated consistent improvements in both total and double vision, bring us closer to extending innovation for these patients. We are now three days away from our May 10 PDUFA date for seronegative. So we are limited in what we can say at this point. A potential approval represents a meaningful opportunity to reach patients without detectable acetylcholine receptor antibodies up to 11,000 additional patients in the US Many of them have been left out of studies or lack access to targeted innovation, and this commitment is what drives us every day.

我們在眼肌型重症肌無力（ocular MG）的正向數據顯示，無論在總體症狀或複視方面皆有一致性改善，讓我們更接近把創新延伸至這些患者。距離我們針對血清陰性（seronegative）的 5 月 10 日 PDUFA 日期只剩三天。因此，目前我們能說的內容有所限制。潛在的核准代表一個重要機會，可觸及那些未檢出乙醯膽鹼受體抗體的患者，在美國最多可額外涵蓋 11,000 名患者。許多人一直被排除在研究之外，或缺乏取得精準創新的管道，而這份承諾正是每天驅動我們前進的力量。

Slide 12. Turning to CIDP. We continue to see consistently strong patient adds quarter-over-quarter with nearly 80% of new starts coming from direct switches from IVIg. As neurologists gain real-world experience and digest emerging data, they are becoming more thoughtful about when to introduce a targeted therapy like VYVGART and are increasingly considering earlier use. Physicians are citing VYVGART's functional outcomes as a key differentiator, particularly our grip strength data.

第 12 張投影片。接著談 CIDP。我們持續看到每季新增患者數穩健且一致地成長，近 80% 的新起始治療來自於由 IVIg 直接轉換。隨著神經科醫師累積真實世界使用經驗並消化新出爐的數據，他們對於何時導入像 VYVGART 這類標靶治療變得更審慎，也愈來愈多考慮更早使用。醫師指出 VYVGART 的功能性結局是關鍵差異化因素，特別是我們的握力數據。

When CIDP patients were followed from ADHERE to the open-label extension, mean grip strength continued to improve with weekly treatment up to 96 weeks. While neurologists shared that it is rare to see patients regain function after deterioration, which makes an observed recovery in grip strength especially compelling. We remain focused on understanding how best to serve the broader CIDP population and how we can shape the market for earlier use of VYVGART.

當 CIDP 患者從 ADHERE 研究追蹤至開放標籤延伸試驗時，平均握力在每週治療下持續改善，最長可達 96 週。神經科醫師也分享，患者在功能惡化後很少能再恢復功能，因此觀察到握力回復尤其具有說服力。我們仍專注於了解如何以最佳方式服務更廣泛的 CIDP 人群，以及我們如何塑造市場以推動更早使用 VYVGART。

We are steadily expanding within our initial 12,000 patient addressable market and are seeing our strategy to work increasingly beyond this. At AAN, we presented the post-hoc analysis of the ADHERE study showing 87.5% response among treatment-naive patients, which continue to support this effort.

我們在最初可觸及的 12,000 名患者市場中穩步擴張，並看到我們的策略在此範圍之外也愈來愈有效。在 AAN 上，我們發表了 ADHERE 研究的事後分析（post-hoc analysis），顯示未曾接受治療的患者反應率為 87.5%，持續支持這項努力。

As we look ahead, our focus is on repeating what has already proven successful. The commercialization playbook we built in MG and CIDP is repeatable, combining a patient-first access strategy, deep prescriber engagement and disciplined execution. This positions us well as VYVGART expands into new indications and our future medicines, including efgartigimod, move closer to launch. With that, I will now turn the call back over to Karen.

展望未來，我們的重點是複製已被證明成功的做法。我們在 MG 與 CIDP 建立的商業化作戰手冊可重複運用，結合以患者為先的可近性策略、對處方醫師的深度互動，以及嚴謹的執行。這讓我們在 VYVGART 擴展至新適應症，以及我們未來的藥物（包括 efgartigimod）更接近上市之際，具備良好定位。接下來我把電話交回給 Karen。

Thank you, Sandrine. Slide 13. Putting patients first is how we define success at argenx. I'm incredibly proud of what this team has accomplished, and I'm energized by what lies ahead. I'm grateful for the trust of our patients, our partners and our shareholders as we continue this journey together. Thank you. And with that, operator, we'll open the call up to questions.

謝謝你，Sandrine。第 13 張投影片。以患者為先，是我們在 argenx 定義成功的方式。我為這個團隊所完成的一切感到無比自豪，也對未來的道路充滿幹勁。感謝患者、合作夥伴與股東對我們的信任，讓我們能持續一起走在這段旅程上。謝謝。接下來，接線員，我們開放提問。

(Operator Instructions)

(接線員指示)

Yatin Suneja, Guggenheim Partners.

Yatin Suneja，Guggenheim Partners。

First, congratulations to Karen on the CFO confirmation. Perhaps two questions for me. So I would love to hear -- Karen, would love to hear your perspective on the outlook of argenx as you see it. Once you are fully settled in the role, do you anticipate or should we think about any sort of changes to the company's strategic direction? So that's one.

首先，恭喜 Karen 確認擔任 CFO。我這邊可能有兩個問題。我很想聽聽——Karen，我很想聽你對 argenx 未來展望的看法。等你完全進入狀況後，你是否預期，或我們是否應該考慮公司策略方向會有任何變動？這是第一個。

And then the second one is more around VYVGART. You do have two significant label expansion opportunities ahead of you. You probably will have the broadest label for MG across various subsets. How does this change the growth outlook as we head into second half? And how do you expect this differentiation to play out from a competition standpoint?

第二個問題比較聚焦在 VYVGART。你們接下來有兩個重大的適應症標籤擴張機會。你們可能會在 MG 的各個亞族群中擁有最廣的標籤。這會如何改變我們進入下半年時的成長展望？而從競爭角度，你預期這種差異化會如何展現？

(technical difficulty) I appreciate the congratulations on the new role. I'm really excited to be taking on this role at this time for the company. We're in such a position of strength with a really strong quarter and strong momentum, and there's so much opportunity and momentum ahead.

(技術問題) 感謝你對我新職務的祝賀。我非常興奮能在公司此刻接下這個角色。我們在這一季表現非常強勁、動能也很強，未來還有很多機會與動能。

So in terms of the question around strategic priorities, as I step into the role, my priorities are very clear. I mean, I was very much involved with Vision 2030, and there won't be a change in that strategy. For us, Vision 2030 is not just an aspiration, but it's a growth strategy. And so when you look at what we've laid out for that strategy, by the end of the decade, we want to have 2.5 more times patients -- 2.5 times patients on VYVGART. We want to have 3 times more indications approved and 5 molecules in late-stage development.

至於你提到的策略優先事項，當我接任這個角色時，我的優先事項非常清楚。我其實深度參與了 Vision 2030，因此這個策略不會改變。對我們而言，Vision 2030 不只是願景，而是一個成長策略。因此，當你看我們為該策略所規劃的內容，到本十年末，我們希望使用 VYVGART 的患者數增加到 2.5 倍——也就是 2.5 倍的患者數。我們希望核准的適應症數增加到 3 倍，並有 5 個分子進入後期開發。

And those five molecules in late-stage development really set us up for the next decade of growth. So there's no change to the strategy, and I'm incredibly optimistic about our future.

而這 5 個處於後期開發的分子，將為我們下一個十年的成長奠定基礎。因此策略不會改變，我對我們的未來非常樂觀。

In terms of your second question on label expansion, we've laid out a strategy in MG for continued growth by bringing continued innovation to the market. So we have our PDUFA date for seronegative in three days. Beyond that, we have ocular MG that we'll be filing quickly, and we're also looking at an expansion into the pediatric population in the future. So as you say, with this, we should in MG have the potential to have the broadest label and be able to reach the broader set of patients. And we see this as a huge differentiator in the market.

至於你第二個關於標籤擴張的問題，我們已在 MG 擬定持續成長策略，透過持續把創新帶到市場來推動成長。我們針對血清陰性（seronegative）的 PDUFA 日期在三天後。此外，我們也會很快遞交眼肌型 MG（ocular MG）的申請，並且未來也在評估擴展至兒科族群。因此如你所說，透過這些進展，我們在 MG 有機會擁有最廣的標籤，並能觸及更廣泛的患者族群。我們認為這將是市場上的重大差異化優勢。

What we're already seeing today is that physicians are choosing VYVGART four out of five times for their early line patients as their first biologic. So we see really strong momentum already and that growth strategy of expanding our label should continue that momentum through this year, but also into the future. Thanks for your questions.

我們目前已經看到的是，醫師在早期線別患者中，有五次裡有四次會選擇 VYVGART 作為他們的第一個生物製劑。因此我們已看到非常強的動能，而透過擴大標籤的成長策略，應可在今年延續這股動能，並延伸到未來。謝謝你的提問。

Tazeen Ahmed, Bank of America.

Tazeen Ahmed，美國銀行。

Okay. So Karen, there's a big second half of the year upcoming for the company with several data readouts. I did want to focus my question on myositis in particular. Can you clarify your view of the likelihood for success for each of the three subtypes that you're studying? In particular, do you feel like anyone is more likely to work than the others? I think some people recently have interpreted comments you've made to indicate that you might be less bullish on DM?

好的。Karen，公司下半年有幾項數據讀出，將是非常重要的下半年。我想把問題特別聚焦在肌炎（myositis）。你能否釐清你對正在研究的三個亞型各自成功機率的看法？特別是，你覺得是否有任何一個比其他更可能奏效？我想最近有些人把你先前的評論解讀為你對 DM 的看法可能沒那麼樂觀？

Thank you so much, Tazeen, and I appreciate the pipeline question early on in the earnings call. So yes, as you said, the second half of the year is exciting. Myositis is our entry into rheumatology. And we should remember this is a first-in-class opportunity. This is a white space opportunity.

非常感謝你，Tazeen，也謝謝你在法說會一開始就提到研發管線的問題。是的，如你所說，下半年令人振奮。肌炎是我們進入風濕免疫領域的切入點。而我們也應該記得，這是一個同類首創（first-in-class）的機會。這是一個尚未被充分開發的空白領域（white space）機會。

And we have a very thoughtfully designed trial that really lets us explore and understand across three different subtypes. Each of those subtypes is grounded in a very strong biology rationale. And you'll recall that we had Phase II data in these same three subtypes, and we did move forward into Phase III.

而我們設計了一項非常周全的試驗，讓我們能在三種不同亞型之間進行探索與理解。每一個亞型都有非常強的生物學機轉依據。你也會記得，我們在同樣這三個亞型上已有第二期數據，並且確實推進到第三期。

So what we see as a win is a positive study in meaning statistical significance on the primary endpoint. More broadly, I think what's important in myositis is that there's significant unmet need across myositis broadly. And we've been talking about IMNM quite a bit recently because we've had some learnings around IMNM.

所以我們所認定的勝利，是指研究結果為正向，也就是在主要終點上達到統計顯著性。更廣泛來看，我認為在肌炎領域重要的是，整體肌炎仍存在顯著未被滿足的醫療需求。而我們最近一直在談 IMNM，因為我們在 IMNM 方面獲得了一些新的洞見。

Specifically, as we've been learning about IMNM leading up to the readout and looking into the data in more detail, what you see is that because IMNM is -- those patients have no treatments available, it is severely underdiagnosed and undertreated. So we see that the TAM opportunity in IMNM is bigger than we previously thought. It's around 20,000 patients. That's more similar in size to a CIDP opportunity and with similar dosing.

具體而言，隨著我們在讀出前持續了解 IMNM、並更深入檢視數據，你會看到 IMNM 的情況是——這些患者沒有可用的治療，因此該疾病嚴重被低估診斷且治療不足。因此我們認為，IMNM 的 TAM（總可及市場）機會比我們先前想的更大。約有 20,000 名患者。其市場規模更接近 CIDP 的機會，且給藥方式也相近。

So that's why we've been focused on IMNM. But of course, the other subtypes, for example, DM also have significant unmet need and with limited treatment options. IVIg is the only treatment option available there. DM is a little bit more heterogeneous, but we still think that there's plenty of room for multiple mechanisms of action, and we think that VYVGART has a good value proposition in DM. So we're looking forward to the data readout in Q3.

這就是我們聚焦 IMNM 的原因。但當然，其他亞型，例如 DM，也同樣存在顯著未被滿足的需求，且治療選項有限。在那裡 IVIg 是唯一可用的治療選項。DM 的異質性稍高，但我們仍認為多種作用機轉仍有很大空間，且我們認為 VYVGART 在 DM 方面具有良好的價值主張。因此我們期待在第三季的數據讀出。

Derek Archila, Wells Fargo.

Derek Archila，富國銀行。

Let me add my congrats, Karen, to stepping into the CEO role. Just two quick questions. So first, just on 2Q '26 VYVGART step-up. Like in the last two years, we kind of see that move from 1Q growth to 2Q growth, and we see kind of a nice magnitude of step-up. So I guess, can you characterize what we should expect this year?

Karen，我也要恭喜你接任 CEO 一職。我有兩個很快的問題。第一個是關於 2026 年第二季 VYVGART 的成長跳升。過去兩年我們大致看到從第一季成長到第二季成長的轉換，而且跳升幅度不錯。所以我想問，你能否描述一下今年我們應該期待什麼？

And then just a follow-up to Tazeen's question on myositis. So my understanding is that it's not powered for the individual subtypes, but will you give any qualitative information on how the subtypes performed in the Phase III?

接著是延續 Tazeen 關於肌炎的問題。我的理解是，研究並未針對各個亞型個別設計統計檢定力，但你們是否會就第三期中各亞型的表現提供任何定性的資訊？

Thanks, Derek. Yes, let me hand it over first to Karl so that he can give you some comments on the momentum that we have heading into Q2. And then maybe, Beth, you can comment on the primary endpoint and communications.

謝謝你，Derek。是的，我先把問題交給 Karl，讓他就我們進入第二季時的動能做一些評論。然後也許 Beth，你可以就主要終點與對外溝通做些說明。

Thank you, Derek. Nice to hear from you. Every quarter has its different dynamics, of course. I think we need to focus on the underlying dynamics, which, as Karen already said and as we said earlier in the prepared remarks, which are very strong at the moment. Our full year expectations are unchanged.

謝謝你，Derek。很高興聽到你的聲音。當然，每一季都有不同的動態。我認為我們需要聚焦在基本面動態上；正如 Karen 已經提到、以及我們在先前的準備稿中所說，目前基本面非常強勁。我們對全年展望維持不變。

And the shape can look -- and you can look at the shape of the curve, which will be consistent with prior years. Thank you for the question, Derek.

而曲線的形狀可能會呈現——你也可以看曲線的形狀——將會與過去幾年一致。謝謝你的問題，Derek。

Derek, on the question about what we're going to share on the myositis readout, I think at this point, you know the style on which we communicate. Our plan is, of course, to give the outcome of the primary endpoint. The primary endpoint of the study is the mean TIS score, and that is taken at week 52. But we also understand the importance of contextualizing that subtype performance. How we do that and what that looks like will still to be seen.

Derek，關於肌炎讀出我們會分享什麼，我想此刻你也了解我們一貫的溝通風格。我們的計畫當然是公布主要終點的結果。本研究的主要終點是平均 TIS 分數，並在第 52 週評估。但我們也理解，將各亞型的表現放在脈絡中解讀的重要性。我們會如何呈現、以及呈現到什麼程度，仍有待觀察。

I think it's important to remember that because this is a new therapeutic space that we're entering, we will need to preserve some of that data for an upcoming medical meeting so that we can inform and generate enthusiasm among the rheumatology community.

我認為重要的是要記得，因為這是我們即將進入的一個新的治療領域，我們需要保留部分數據，留待即將到來的醫學會議發表，以便向風濕免疫科社群提供資訊並激發熱情。

James Gordon, Barclays.

James Gordon，巴克萊。

James Gordon of Barclays. I had a question on competition in MG. So I've had some questions from investors about competitors in the US, such as Amgen who have been talking about UPLIZNA, so CD19. They're talking about strong uptake in both bio-naive and switch patients even without step through. So are you seeing UPLIZNA being used much in MG?

我是巴克萊的 James Gordon。我有一個關於 MG 競爭態勢的問題。我收到一些投資人對美國競品的提問，例如安進（Amgen）一直在談 UPLIZNA，也就是 CD19。他們提到即使沒有 step through（逐步治療限制），在生物製劑初治與轉換患者中都有強勁的採用。所以你們是否看到 UPLIZNA 在 MG 中被大量使用？

And is it displacing in any patients that could use VYVGART? And also, I think that Amgen has said they're taking into a CIDP trials. So could that be a threat in addition to potentially C1s coming along? And then also from the competitive point of view, Regeneron, they've got cemdisiran, so also hitting C5, but also a potential MG approval later this year in Q4. Do you think that could impact VYVGART in MG?

它是否在某些患者上造成替代，影響原本可能使用 VYVGART 的人？另外，我想安進也表示他們正推進 CIDP 試驗。因此，除了可能出現的 C1s 之外，這是否也可能構成威脅？再從競爭角度，Regeneron 有 cemdisiran，同樣作用於 C5，且可能在今年第四季稍晚取得 MG 核准。你認為這會影響 VYVGART 在 MG 的表現嗎？

Yes. Thanks for the question, James. There's a lot in there. I'm actually going to hand it over to Sandrine. She's been spending a lot of time out in the field with customers and at AAN, and I think has probably recent experience to talk from.

是的。謝謝你的問題，James。裡面涵蓋很多面向。我其實要把問題交給 Sandrine。她最近花了很多時間在第一線與客戶互動，也參加了 AAN，我想她有最新的經驗可以分享。

Yes. Thank you. Thank you, Karen. Thank you, James. So indeed, competition has come. We have put MG on the map. So there are more and more players in there, and we see that as a good sign. It means there is a lot of potential in that market, but also more option and innovation for patients, which drives the size of the biologic market, which benefits all of us and especially VYVGART as four out of five providers really prefer VYVGART as the first biologic.

是的。謝謝。謝謝你，Karen。謝謝你，James。確實，競爭已經到來。我們把 MG 帶上了版圖。因此參與者越來越多，我們視之為正向訊號。這代表該市場潛力很大，同時也為患者帶來更多選擇與創新，進而推動生物製劑市場規模成長，讓我們所有人受益，尤其是 VYVGART，因為五位醫療提供者中有四位確實偏好以 VYVGART 作為第一個生物製劑。

Now going to your question specifically on some more recent entrants. So what is interesting to see is that most of the recent launches are really positioned towards the later lines. So more for refractory patients and after VYVGART. So we see some use but not really directly competing with us. Because what we are trying to do is to go in earlier lines because we know that 80% of the MG market is still not in the hands of biologics.

回到你特別提到的一些較新進入者。有趣的是，多數近期上市產品的定位其實偏向後線治療。也就是更針對難治型患者，且通常是在 VYVGART 之後。因此我們確實看到一些使用，但並未真正與我們直接競爭。因為我們的策略是往更前線推進，因為我們知道 MG 市場中仍有 80% 尚未由生物製劑所覆蓋。

And this is where we see a really big opportunity, and that's our strategy. So some use indeed, but more in later lines and refractory. So you also mentioned a recent competitor in CIDP. I mean, CIDP, as you know, we have been in the field for now two years. We have another product in development, empasiprubart in development.

而這正是我們看到的巨大機會，也是我們的策略。所以確實有一些使用，但更多是在後線與難治型。你也提到 CIDP 的近期競品。我的意思是，CIDP 如你所知，我們已在第一線耕耘兩年。我們也有另一個研發中的產品 empasiprubart。

So what we believe is that CIDP is a progressive disease, but very heterogeneous and there is space for multiple mechanism of action. And so with the current profile of VYVGART, we believe we have a very strong value proposition between the PFS, the efficacy, the safety, recent grip strength data where we showed sustained efficacy up to 96 weeks and then we just presented the data at AAN on earlier use where we did a post-hoc analysis.

因此我們的看法是，CIDP 是一種進行性疾病，但異質性很高，且多種作用機轉都有其空間。所以以 VYVGART 目前的產品特性而言，我們認為在 PFS、療效與安全性之間具備非常強的價值主張；此外，近期的握力數據顯示療效可持續至 96 週；而我們也剛在 AAN 發表了更早期使用的數據，當時我們做了事後（post-hoc）分析。

I don't know if you saw that one, where we showed that naive patients had 87.5% clinical efficacy. So we are really trying to extend here the overall market for VYVGART, and this is very great for CIDP patients. So now for the C5 question, I think Karen wanted to say something. So I'll hand it over back to Karen.

我不知道你是否看過那一段，我們展示了初治患者有 87.5% 的臨床療效。因此，我們確實是在努力擴大 VYVGART 的整體市場，而這對 CIDP 患者非常好。那麼現在回到 C5 的問題，我想 Karen 想說些什麼。所以我把時間交回給 Karen。

Thank you, Sandrine. I think that's great. And I think you're exactly on point. As we see new competitors coming into the market, C5s and others, they're mostly being used in the refractory space. So thanks for the question.

謝謝你，Sandrine。我覺得這很棒。而且我認為你說得非常到位。隨著我們看到新的競爭者進入市場，C5 類以及其他產品多半用在難治（refractory）領域。所以謝謝你的提問。

Alex Thompson, Stifel.

Alex Thompson，Stifel。

Congrats to Karen here. I was wondering sort of, to talk at a high level about your appetite for later-stage business development than what you've done historically? And maybe in the context of that, how you're thinking about your Forte equity investment?

恭喜 Karen。我想從較高層次請教一下：相較於你們過去的做法，你們對於更後期階段的商務開發（business development）的意願有多大？以及在這個脈絡下，你們如何看待對 Forte 的股權投資？

Yes. Thanks for the question. Yes, maybe to take a step back, we talked about our ambition earlier with Vision 2030. And over the long term, we want to become a leader in immunology. And what that means is that we're focused very much on building our pipeline.

是的。謝謝你的提問。是的，也許先退一步來看，我們先前談到 Vision 2030 的企圖心。從長期來看，我們希望成為免疫學領域的領導者。這代表我們非常專注於建立我們的研發管線。

In the past, we've been focused on building our pipeline through partnerships with academic institutions. You all know our Immunology Innovation Program where we've sought novel biology that can provide transformative outcomes for patients through those partnerships with academic institutions.

過去，我們主要透過與學術機構的合作來建立研發管線。各位都知道我們的免疫學創新計畫（Immunology Innovation Program），我們透過與學術機構的合作，尋找能為患者帶來變革性成果的新穎生物學（novel biology）。

As we've been developing a stronger cash balance and financial strength, what that allows us to do is broaden our lens a little bit and also look for other opportunities to source novel biology and where we can provide transformative outcomes. So your example that you're calling out of Forte is one example of that where we made a strategic investment in something that we see is novel biology and similar to what you also saw with Tensegrity earlier this year, where we're investing in options in novel biology.

隨著我們累積了更強的現金部位與財務實力，這讓我們能把視野再放寬一些，也去尋找其他取得新穎生物學、並能帶來變革性成果的機會。你提到的 Forte 就是一個例子：我們做了策略性投資，因為我們認為那是新穎生物學；而且也類似於你今年稍早看到的 Tensegrity，我們是在新穎生物學上投資選擇權（options）。

So you can expect to see more of this from us as we continue to build our pipeline and continue to leverage our balance sheet. We want to invest in our internal innovation pipeline and source novel biology from wherever we can find it. Thanks for the question.

因此，隨著我們持續建立研發管線並持續運用資產負債表，你可以預期我們會有更多這類動作。我們希望投資於內部創新管線，並從任何我們能找到的地方取得新穎生物學。謝謝你的提問。

Rajan Sharma, Goldman Sachs.

Rajan Sharma，高盛。

Maybe just on the topic of competition. We saw that J&J are running a head-to-head trial of ImAAVY versus VYVGART in myasthenia gravis, which could read out next year. I was just wondering if you could provide your perspectives on the trial design and expectations here. To what extent is that a risk to VYVGART or not conscious of the different formulation? And then a very quick follow-up for Karl.

想請教競爭相關的話題。我們看到 J&J 正在進行 ImAAVY 與 VYVGART 在重症肌無力（myasthenia gravis）的頭對頭試驗，可能明年讀出結果。我想請你分享對該試驗設計與預期的看法。這在多大程度上會對 VYVGART 構成風險，或者是否因為不同劑型而不太需要擔心？然後我有一個很快的追問要問 Karl。

I heard your comments on operating leverage. Should we expect to see this incrementally quarter-on-quarter as well as on an annual basis?

我聽到你對營運槓桿（operating leverage）的評論。我們是否也應該預期這會在每一季逐季改善，同時在年度基礎上也改善？

Yes. Thank you. I'll take your question on competition, and then I'll hand over to Karl for the question on operating leverage. Yes, look, I think Sandrine said it best, we put MG on the map and many competitors are following us, especially as a first-in-class FcRn. And I think what you see is that we've set the standard for efficacy in terms of MSE in MG and have the stronger safety profile and we have all of these innovations.

是的。謝謝。我先回答你關於競爭的問題，接著把營運槓桿的問題交給 Karl。是的，你看，我想 Sandrine 說得最好：我們把 MG 帶上了版圖，許多競爭者正在跟進，特別是作為同類首創（first-in-class）的 FcRn。而你看到的是，我們在 MG 的 MSE 療效方面樹立了標準，並且有更強的安全性概況，而且我們有所有這些創新。

We started with IV, we launched subcutaneous and then we most recently brought PFS to market. And in fact, PFS is driving the majority of our growth at the moment. And so when you think about that, I think the question to ask about some of these studies and these other competitors is what problem are they trying to solve? And I think what we're really focused on is how do we bring more value to more patients where there is unmet need.

我們從靜脈注射（IV）開始，推出了皮下注射（subcutaneous），而最近我們把 PFS 推向市場。事實上，PFS 目前正驅動我們大部分的成長。因此當你這樣思考時，我認為對於其中一些研究與其他競爭者，該問的問題是：他們想解決什麼問題？而我們真正專注的是：如何在仍有未被滿足需求的地方，為更多患者帶來更多價值。

And for us, that's focusing on PFS, which is unmatched as well as focusing on that strategy we were talking about earlier, which is broadening the population through seronegative, through ocular and in the future through pediatrics. So with that being said on competition, maybe, Karl, you can talk about our operating margin.

對我們而言，就是聚焦在同樣無可匹敵的 PFS，並且聚焦在我們先前談到的策略：透過血清陰性（seronegative）、透過眼肌型（ocular），以及未來透過兒科（pediatrics）來擴大族群。在競爭方面就先說到這裡；Karl，也許你可以談談我們的營業利益率（operating margin）。

Thank you. Thank you, Rajan, for the question. Yes, I want to start off by reminding we are on an innovation mission. The patient is your North Star. We have a unique opportunity now to invest in innovation and set the company up for the long run to build a long-term sustainable company. That is the capital allocation priorities of the company.

謝謝。謝謝你，Rajan，提問。是的，我想先提醒一下：我們正肩負創新使命。患者是你們的北極星。我們現在有一個獨特的機會去投資創新，並為公司長期發展做好布局，打造一家長期可持續的公司。這就是公司的資本配置優先順序。

But that said, yes, you are right. The very successful launch allows us to build a P&L where we already have a very good margin structure. Our gross margin is around 90%. Our operating margin is around 30%. We added $400 million of cash this quarter, getting us to $4.9 billion.

但話說回來，是的，你說得對。非常成功的上市推進，讓我們能建立一份損益表（P&L），而我們已經有非常好的利潤結構。我們的毛利率約 90%。我們的營業利益率約 30%。本季我們增加了 4 億美元現金，使我們的現金達到 49 億美元。

So yes, you can expect going forward to see margin expansion every quarter year-over-year, and we're going to continue to build on that. But that, of course, is not the objective at the moment, but I think we can do both. Thank you for the question.

所以是的，展望未來，你可以預期我們的利潤率會在每一季、以年對年（year-over-year）的基礎持續擴張，我們也會繼續在此基礎上累積。但當然，這目前不是我們的目標，不過我認為我們可以兩者兼顧。謝謝你的提問。

Allison Bratzel, Piper Sandler.

Allison Bratzel，Piper Sandler。

This is Ashleigh, on for Allison Bratzel. Congrats on the quarter and all the progress. So just from us, -- just curious to learn more about the go and no-go decision on the Phase II VARVARA trial in delayed graft function, which is expected midyear. What specific clinical or biomarker signals are you looking to see to justify advancing clinical development? And just more broadly speaking, how are you viewing the commercial opportunity in delayed graft function?

我是 Ashleigh，代替 Allison Bratzel 發言。恭喜本季表現以及所有進展。我們這邊想請教：關於延遲移植物功能（delayed graft function, DGF）的第二期 VARVARA 試驗，預計年中會有 go/no-go 決策；我們想更了解你們的判斷依據。你們具體在尋找哪些臨床或生物標記（biomarker）訊號，來支持推進後續臨床開發？以及更廣泛地說，你們如何看待 DGF 的商業機會？

Yes. Thanks for the question on DGF. As I said earlier, it's great to hear these questions on our pipeline. So DGF is an important indication for us for our second molecule, empasiprubart. And we're able to pursue DGF as an indication because of empasiprubart being a C2, so involved in both the classic and the lectin pathways in complement. So that's a differentiator for us.

是的。謝謝你關於 DGF 的提問。如我先前所說，很高興聽到大家對我們研發管線的問題。DGF 對我們第二個分子 empasiprubart 來說是一個重要適應症。我們之所以能把 DGF 作為適應症推進，是因為 empasiprubart 是 C2，參與補體（complement）的經典途徑與凝集素途徑。因此這是我們的差異化優勢。

In terms of the study, it is a Phase II study. So think about it as an exploratory study. And what we wanted to do was see the readout of the data for longer term. So we're following this data out to 52 weeks because in this patient population and what we hear from both patients as well as health care systems and providers is what they care about is the long-term outcomes. So as soon as we have that data, we'll be able to analyze it, and we'll have a go/no-go decision for Phase III. Thanks for the question.

就研究而言，這是一項第二期研究。所以可以把它視為探索性研究。我們想做的是觀察更長期的數據讀出。因此我們會追蹤到 52 週，因為在這個患者族群中，從患者以及醫療體系與醫療提供者那裡聽到的是：他們在意的是長期結果。一旦我們拿到那些數據，就能進行分析，並對第三期做出 go/no-go 決策。謝謝你的提問。

Daniel Brill, Truist Securities.

Daniel Brill，Truist Securities。

This is Alex, on for Daniel. Congrats on the quarter. Another question on myositis. On the placebo response, just curious if you could talk a little bit about what factors are known to drive the placebo response? And do you expect any difference in the three subgroups?

我是 Alex，代 Daniel 發言。恭喜本季表現。我還有一個關於肌炎（myositis）的问题。關於安慰劑反應，想請教您能否談談有哪些已知因素會驅動安慰劑反應？以及您是否預期三個亞組之間會有任何差異？

And then alternatively on the steroid taper, just curious what effects of the steroid tapering you're anticipating in the placebo response? And also, do you have any thoughts on why Roivant steroid tapering did not yield any decrease in the overall TIS endpoint in its placebo arm in its DM trial?

另外，關於類固醇減量（steroid taper），想請教您預期類固醇減量會對安慰劑反應產生哪些影響？以及，您對於 Roivant 在其 DM 試驗中，類固醇減量並未使其安慰劑組整體 TIS 終點下降，有什麼看法嗎？

Yes. Thanks for the question. So let me take them one by one. So first, when you think about placebo response, whether across immunology, you see this pretty consistently. We've seen it in MG, in CIDP, you see it in Sjogren's studies and similar in myositis.

是的。謝謝你的提問。我會逐一回答。首先，談到安慰劑反應，無論是在免疫學領域的各種研究中，你都會相當一致地看到這種現象。我們在 MG、在 CIDP 都看過；在乾燥症（Sjogren's）研究中也看得到；在肌炎中也類似。

So I think this is a pretty common phenomenon. And when you're developing an immunology like we are, then you start to get sort of good learnings around how to make sure you're minimizing that placebo response in the trial. And that can include things like training the sites to make sure that they understand how best to use the tools and the measures for the primary endpoint and that type of thing. So in this case, as you said, it's TIS. So I don't think there's anything special across the different subtypes or in myositis there.

所以我認為這是一個相當常見的現象。當你像我們一樣在開發免疫學藥物時，就會逐步累積一些經驗，了解如何在試驗中確保把安慰劑反應降到最低。這可能包括例如訓練各試驗中心，確保他們理解如何最佳地使用主要終點的工具與量測方式等。在這個案例中，如你所說，是 TIS。因此我不認為在不同亞型之間或在肌炎方面有什麼特別之處。

In terms of the steroid taper, we think this is actually a benefit of the design of our study because what we've been able to do is build it in late enough in the trial so that you have a systemic steroid tapering, which should actually unmask any placebo response and help us to show a clear benefit on active disease. So we see that -- the way that we've designed it is very elegant, and it should actually help us to uncover the benefit of VYVGART. In terms of Roivant, I would encourage you to ask them. I'm not sure about the details on their steroid taper. Thanks for the question.

至於類固醇減量，我們認為這其實是我們研究設計的一個優點，因為我們把它安排在試驗中相對後段的時間點，讓你有一個全身性類固醇減量，這應該能夠「揭露」任何安慰劑反應，並幫助我們在活動性疾病上顯示出明確的療效。所以我們認為——我們的設計方式非常精巧，實際上應該能幫助我們揭示 VYVGART 的效益。至於 Roivant，我建議你去問他們。我不太清楚他們類固醇減量的細節。謝謝你的提問。

Yaron Werber, TD Cowen.

Yaron Werber，TD Cowen。

Congrats, Karen, again. A couple of sort of interrelated questions. One, can you just give us a little bit of a sense? We're kind of thinking that CIDP is around 40% of sales right now, maybe kind of 37%. I don't know if I'm in the right ballpark.

Karen，再次恭喜。我有幾個彼此相關的问题。第一，能否請你稍微給我們一些概念？我們在想 CIDP 目前大約占銷售額的 40%，可能約 37%。我不確定這樣的估計是否在合理範圍內。

And then secondly, for the seronegative study, it was a very wide study with a p-value of 0.1 across all patients, which you hit successfully. And it looks like it was driven by the MuSK and LRP positive patients. In the seronegatives, technically, the endpoint wasn't met, but because just it came together literally at the end, there was a benefit throughout the period. Just trying to get your conviction that you can get a broad approval and not just in the autoantibody positive?

第二，關於血清陰性（seronegative）研究，這是一個涵蓋面很廣的研究，在所有患者中 p 值為 0.1，你們也成功達標。看起來結果是由 MuSK 與 LRP 陽性患者所驅動。在血清陰性患者中，嚴格來說終點沒有達成，但因為最後才剛好收斂，所以在整個期間其實都有看到效益。我想了解你們對於能取得廣泛核准（而不僅限於自體抗體陽性）的信心有多高？

Yes. Thanks for the questions. I'm going to hand it over to Karl to talk about CIDP and how the mix of business is evolving. And then I'll turn it over to Sandrine to talk about seronegative. I want to just make a point that's really important, though, related to the specific question you asked.

是的。謝謝你的提問。我先請 Karl 來談 CIDP 以及業務組合如何演變。接著我會請 Sandrine 來談血清陰性。不過我想先強調一點，這與你剛才問的具體問題非常重要。

We're three days from our PDUFA date. And so we have to be very careful in how we talk about this. So we're going to keep the discussion very general. And what I want Sandrine to talk about is, in general, how you see the seronegative opportunity in the case of approval so that we don't go into those details. But maybe we can talk about CIDP first.

我們距離 PDUFA 日期只剩三天。因此我們在談論方式上必須非常謹慎。所以我們會把討論維持在非常概括的層次。我希望 Sandrine 談的是：一般而言，在獲准的情況下，你如何看待血清陰性的機會，這樣我們就不會深入那些細節。不過也許我們可以先談 CIDP。

Yaron, thank you for the question. Yes, both CIDP and MG continue to be growth drivers for us. And of course, CIDP is mainly in the US now, but also in Japan and Germany, other markets we still have to launch. In terms of a percentage breakdown, I don't want to get into that, but I will say that MG is still the majority of our revenues and of course, the majority of our patients, but both still have a lot of growth in them. Thank you for the question. I'm handing over to Sandrine.

Yaron，謝謝你的提問。是的，CIDP 與 MG 仍然持續是我們的成長驅動力。當然，CIDP 目前主要在美國，但也在日本與德國；其他市場我們仍有待上市。至於百分比拆分，我不想深入談，但我可以說 MG 仍占我們營收的大宗，當然也占患者的大宗；不過兩者都仍有很大的成長空間。謝謝你的提問。我把時間交給 Sandrine。

So thank you for the question on seronegative. So like Karen said, I will stay very general because we are only three days away from the PDUFA date. So if approved in seronegative, as you mentioned, I mean, there is a pool of patients, which we assess at 11,000 patients that would cover potentially these three subtypes. And so as we are only three days away from PDUFA date, we are obviously ready to launch. I mean the good news is that we are already embedded into MG.

謝謝你關於血清陰性的提問。如 Karen 所說，因為距離 PDUFA 日期只剩三天，我會維持在非常概括的層次。如果血清陰性獲准，如你提到的，確實有一個患者池，我們評估約為 11,000 名患者，可能涵蓋這三個亞型。因此，既然距離 PDUFA 日期只剩三天，我們顯然已準備好上市。好消息是，我們已經深度布局於 MG 領域。

There is a big overlap of the prescriber base with more than 80% of the target we already did it that are actually -- that we cover seronegative patients. So we don't need to add any field force. The prescribers know extremely well VYVGART and the trust, their experience of VYVGART and its efficacy that is deep, fast and sustained. And then we had a good presentation at AAN with lots of questions.

處方醫師基礎有很大的重疊，我們已經觸及的目標中有超過 80%——也就是我們所覆蓋的——其實都會照護血清陰性患者。因此我們不需要增加任何外勤團隊。處方醫師對 VYVGART 非常熟悉，而且他們對 VYVGART 的信任、以及其療效的經驗——深度、快速且持久——都很扎實。此外，我們在 AAN 也有很好的發表，並收到很多問題。

So it shows that there is an interest really and that physicians and providers are really waiting for that. So I won't be able to go more into the details, but we should hear in the next few days.

所以這顯示確實有高度興趣，醫師與醫療提供者都在等待這個結果。我無法再更深入細節，但我們應該會在接下來幾天內得知消息。

Thomas Smith, Leerink Partners.

Thomas Smith，Leerink Partners。

Let me add my congrats to Karen on stepping into the CEO role here. It sounds like the VYVGART auto-injector continues to advance and you're guiding to launch in '27. Can you just provide an update on where you are with this formulation? What are the outstanding gating factors for bringing this to market? And how are you thinking about potential uptake in this form versus the prefilled syringe?

我也要恭喜 Karen 接任 CEO 這個職務。聽起來 VYVGART 自動注射器（auto-injector）持續推進，你們指引在 2027 年上市。能否請你更新一下目前這個劑型的進度？推向市場還有哪些尚待完成的關鍵門檻因素（gating factors）？以及你們如何看待這種形式相較於預充式注射器（prefilled syringe）的潛在採用情況？

And then if I could, just a clinical follow-up on efgartigimod in Graves' disease. We saw the Phase III design that you recently posted on clinicaltrials.gov. And I was wondering if you could comment on some of the design considerations for this multipart study? How are the patients being handled between Part A and Part B? And how are the background ATDs being managed? And maybe just higher level, like what are your expectations with respect to the primary endpoint registrational path?

另外如果可以的話，我想就 efgartigimod 在葛瑞夫茲病（Graves' disease）做一個臨床追問。我們看到你們最近在 clinicaltrials.gov 上公布的第三期設計。我想請你評論一下這個多部分（multipart）研究的一些設計考量？Part A 與 Part B 之間的患者如何處理？背景治療的抗甲狀腺藥物（ATDs）如何管理？以及更高層次地說，你們對主要終點與註冊性（registrational）路徑的預期是什麼？

Yes. Let me start with the auto-injector question. We're moving into manufacturing stage at the moment, as you say, to get ready for auto-injector launch in 2027. The key ungating factors are really just moving through those different gates of making sure that we're ready for production and approval. In terms of the opportunity that we see with auto-injector, what we saw with prefilled syringe is that it opened up a significantly larger patient population. And actually, and we said earlier, prefilled syringe is driving the majority of the growth for us today.

是的。我先從自動注射器的問題開始。正如你所說，我們目前正進入製造階段，為 2027 年自動注射器上市做準備。關鍵的解鎖因素其實就是依序通過不同的關卡，確保我們已準備好量產與取得核准。就我們看到的自動注射器機會而言，我們在預充式注射器上看到的是，它開啟了顯著更大的病患族群。而且其實，正如我們先前提到的，預充式注射器目前正驅動我們大部分的成長。

And so with auto-injector, I don't think it will be as much of a step forward as prefilled syringe. You'll recall that in the US, prefilled syringe moved us out of health care administration into at-home administration. But the auto-injector will be a step forward in terms of patient convenience and ease. So I think that's an important consideration for how we compete in MG.

因此就自動注射器而言，我不認為它會像預充式注射器那樣帶來那麼大的躍進。你會記得在美國，預充式注射器讓我們從醫療機構施打轉向居家施打。但自動注射器在病患便利性與易用性方面會是向前的一步。所以我認為，這是我們在 MG 領域競爭時一個重要的考量。

In terms of Graves' disease, clinicaltrials.gov covers all of the details that we want to disclose publicly. But I'm going to hand over to Beth to talk about some of the additional details.

關於葛瑞夫茲病（Graves' disease），clinicaltrials.gov 已涵蓋我們希望對外公開的所有細節。不過我會把時間交給 Beth，請她談一些額外的細節。

Yes. So we designed the Graves' study, taking into account, of course, precedent studies, and that was aligned with what the regulators wanted. So it's actually going to be two studies. You're going to see some kind of similar attributes of this study where we're actually dosing on top of antithyroid drugs. We're looking at patients at the end of the study who are euthyroid off antithyroids.

是的。因此我們在設計 Graves' 研究時，當然考量了既有的先例研究，並且與監管機關的期待一致。所以實際上會是兩項研究。你會看到一些與本研究相似的特徵，也就是我們會在抗甲狀腺藥物的基礎上加用給藥。我們觀察的是在研究結束時，停用抗甲狀腺藥後仍維持甲狀腺功能正常（euthyroid）的病患。

So you're going to see kind of a tapering off that. I think beyond that, we'll get into more details at a later date. But we're really excited about kicking this off. We're focused on moving enrollment along as quickly as possible. And yes, more to come at a later date.

因此你會看到某種逐步減量的安排。我想除此之外，我們會在之後的時間點再提供更多細節。但我們對於啟動這項研究感到非常興奮。我們專注於盡可能快速推進收案。是的，後續會在稍晚再更新更多內容。

Akash Tewari, Jefferies.

Akash Tewari，Jefferies。

Karen, congrats on the new role. Very well deserved. Just on your Phase III myositis trial design, will the FDA allow you to file on full data? Or does each subset need to be statistically significant for broad approval? And additionally, can you go over the rationale of your ADAPT Forward trials that look at VYVGART and empa in combination in gMG? What type of signal would you want to see to move that forward in larger studies?

Karen，恭喜你擔任新職務。實至名歸。關於你們第三期肌炎（myositis）試驗的設計，FDA 會允許你們以完整數據申請送件嗎？還是每個子族群都需要達到統計顯著，才能取得廣泛適應症核准？另外，你能否說明 ADAPT Forward 試驗的設計邏輯：在 gMG 中評估 VYVGART 與 empa 聯合使用？你們希望看到什麼樣的訊號，才會推進到更大型研究？

Yes. Thanks for the question. So in terms of myositis, look, the label that we get, that will be a review decision, and we'll have to see how the data unfolds and let the data speak in each of the subtypes. So we look forward to that data in Q3. I'm glad you asked about ADAPT Forward because it's a key part of our combination strategy that I think we're uniquely positioned as argenx to pursue in MG and also in some other of our indications like CIDP.

是的。謝謝你的提問。就肌炎而言，最終我們拿到的標籤會是審查端的決定；我們必須看數據如何展開，並讓各子型別的數據自己說話。因此我們期待在第三季看到這些數據。我很高興你問到 ADAPT Forward，因為它是我們聯合治療策略的關鍵一環；我認為以 argenx 的定位，我們在 MG 以及其他一些適應症（例如 CIDP）上具備獨特優勢可以推進。

So ADAPT Forward is a platform study. And what we're looking at is can we dramatically increase the efficacy and outcomes for patients in MG. So what we're looking at is VYVGART as the backbone of therapy and then looking at different combinations, for example, empasiprubart on top of VYVGART and can we increase the number of patients reaching MSE. Over time, we want to -- it's a platform trial.

因此 ADAPT Forward 是一項平台型研究。我們要看的，是能否大幅提升 MG 病患的療效與結局。我們的做法是以 VYVGART 作為治療骨幹，然後評估不同的組合，例如在 VYVGART 基礎上加上 empasiprubart，看看能否提高達到 MSE 的病患比例。隨著時間推進，我們希望——這是一個平台試驗。

So we will be adding different arms to the trial to explore different combinations of -- that we have in our pipeline so that we can explore which we would want to move forward in Phase III. Thanks for the question.

因此我們會在試驗中加入不同的治療組別，去探索我們研發管線中不同的聯合方案，從而評估哪些方案值得推進到第三期。謝謝你的提問。

Sean Laaman, Morgan Stanley.

Sean Laaman，Morgan Stanley。

Two questions. The first one is your recent data presented at AAN for VYVGART in treatment-naive CIDP patients. How do you see that evolving? Do you see VYVGART potentially moving up into the front line? I think there's a lot of focus on just the cost of drug, but considering you've got just a short injection with VYVGART versus all the [ plough ] that goes on with Ig administration, just to give your view there.

兩個問題。第一個是你們近期在 AAN 發表的 VYVGART 用於未接受治療（treatment-naive）的 CIDP 病患數據。你們認為這會如何發展？你們是否認為 VYVGART 有可能前移到一線治療？我知道大家很關注藥物成本，但考量到 VYVGART 只需要短時間注射，相較於 Ig 給藥所需的一堆流程與負擔，想請你分享你們的看法。

And Second question is just on the IMNM opportunity. And I think, Karen, you mentioned a CIDP-like opportunity. But just give us a bit more color on the accessibility of that patient base. Are they readily diagnosed? What do you have to do there to get into that market?

第二個問題是關於 IMNM 的機會。我想 Karen 你提到這是一個類似 CIDP 的機會。但能否再多提供一些關於該病患族群可及性的資訊？他們是否容易被診斷？你們需要做些什麼才能切入那個市場？

Yes. Thanks for the question. I'm going to hand over to Sandrine to talk about the CIDP data. She was at AAN, and then I'll come back to the question on IMNM.

是的。謝謝你的提問。我先把時間交給 Sandrine 談 CIDP 的數據。她人在 AAN，之後我再回來回答 IMNM 的問題。

Yes. So great question. And indeed, in my prepared remarks, I mentioned that post-hoc analysis because I think it's data what we have never presented before, showing that VYVGART can indeed have an impact -- a sustainable impact and fast impact and efficacy on naive patients, truly naive patients. So we are very excited by this data. A few analysts picked that up, and I think this is going to expand the possibilities for VYVGART because if you look at our label in the US, we actually could be used first line. So there is actually theoretically no barriers to using.

是的。這是個很好的問題。而且確實，在我事先準備的發言中，我提到了那個事後分析（post-hoc analysis），因為我認為那是我們以前從未呈現過的數據，顯示 VYVGART 的確能對初治病患——真正的初治病患——帶來影響：可持續的影響、快速的影響，以及療效。因此我們對這些數據感到非常興奮。有幾位分析師注意到了這點，我認為這將擴大 VYVGART 的可能性，因為如果你看我們在美國的標籤，其實可以作為一線使用。所以理論上，使用上其實沒有障礙。

And so in practice, we see two barriers for broader usage in first line. The first one is indeed getting physicians comfortable using it when patients are doing okay. And for that, it's very important to come with convincing data like the one in the post-hoc analysis, but also data like we are showing with the grip strength, where we show really very good efficacy on a sustained basis at 96 weeks.

因此在實務上，我們看到一線更廣泛使用有兩個障礙。第一個確實是讓醫師在病患狀況還算穩定時，願意放心使用。為此，提出像事後分析那樣具說服力的數據非常重要；同時也包括我們以握力（grip strength）呈現的數據，顯示在 96 週時仍能維持非常好的、持續性的療效。

And the second barrier we are seeing is indeed payers, and you mentioned that in your question. So having data like the one we had in the post-hoc analysis allows us to go back to payers and a discussion because ultimately, this is better for patients. So we believe this kind of data will help us move the needle step by step into broadening the market in first line for VYVGART. So I'll hand it back to Karen for your other question.

第二個障礙確實是付款方（payers），你在問題中也提到了。因此，像我們事後分析那樣的數據，讓我們能回到付款方那裡進行討論，因為歸根究柢，這對病患更好。所以我們相信，這類數據將幫助我們一步一步推動指標，擴大 VYVGART 在一線治療的市場。我把時間交回給 Karen 回答你另一個問題。

Thank you, Sandrine. Yes. And in relation to your question on IMNM being a CIDP-like opportunity, so the way that we see it is we size the addressable market as around 20,000 patients. But when you first look at the treated population of IMNM in claims data, it looks more like it's around 6,000 to 7,000 patients. So we think that those are quite easily accessible because there's no treatment options available at the moment.

謝謝你，Sandrine。是的。關於你提到 IMNM 是類似 CIDP 的機會，我們的看法是：我們估算可觸及市場大約是 20,000 名病患。但當你一開始從理賠（claims）數據去看 IMNM 的已治療族群時，看起來更像是約 6,000 到 7,000 名病患。因此我們認為這些病患其實相當容易觸及，因為目前並沒有可用的治療選項。

Beyond that, what we will need to invest in, in the case of positive data is really disease state education to increase awareness and increase diagnosis of this patient population. But what the opportunity that we have is that IMNM is frequently treated by neurologists, and there's quite a lot of overlap with those neurologists with our MG and our CIDP prescribers. So we see the opportunity to build this market in the same way that we've built the CIDP market, we've built the MG market. And I think we'll see, assuming positive data, the same outcome in IMNM. Thanks for your question.

除此之外，在數據為正的情況下，我們真正需要投入的是疾病狀態教育，以提升對這一患者族群的認知並提高診斷率。但我們所擁有的機會在於，IMNM 經常由神經科醫師治療，而這些神經科醫師與我們 MG 與 CIDP 的處方醫師有相當大的重疊。因此，我們看到有機會以我們建立 CIDP 市場、建立 MG 市場的同樣方式來打造這個市場。我認為在數據為正的前提下，我們也會在 IMNM 看到相同的結果。謝謝你的提問。

(Operator Instructions)

(接線員指示)

Gavin Clark-Gartner, Evercore.

Gavin Clark-Gartner，Evercore。

This is [ Yi Zhang ], on for Gavin. Congrats on the strong quarter. So one question about the next-generation molecule 213. We saw now it's become Phase I ready. Just wondering, can you share more details on the time line and how you are thinking about the indication selection?

我是[ Yi Zhang ]，代替 Gavin 發言。恭喜本季表現強勁。我有一個關於下一代分子 213 的問題。我們看到它現在已經達到第一期（Phase I）就緒。想請問你們能否分享更多時間表細節，以及你們如何思考適應症選擇？

And also for the Phase III, will it be a noninferiority study head-to-head against VYVGART or it's going to be a stand-alone placebo-controlled study?

另外，第三期（Phase III）會是與 VYVGART 正面對照的非劣性試驗，還是會做一個獨立的安慰劑對照試驗？

Yes. Thanks for the question. The strategy that we're laying out for FcRn is to continue our leadership for decades to come. And so as you mentioned, we have ARGX-213, which moves dosing to every four weeks and we also have ARGX-124, another next-generation asset in the FcRn space as well as our program focused on developing an oral FcRn.

是的。謝謝你的提問。我們為 FcRn 擬定的策略，是在未來數十年持續保持領導地位。因此如你所提到，我們有 ARGX-213，可將給藥頻率調整為每四週一次；同時我們也有 ARGX-124，這是 FcRn 領域的另一個下一代資產，以及我們專注於開發口服 FcRn 的計畫。

So we have a portfolio of options here for how we'll continue to build the FcRn space, explore the FcRn biology and deliver value to patients. So we aren't public on our clinical development plan for 213 yet, but it is Phase III ready and we'll be moving quickly. And we see a few different opportunities. One is to advance patient outcomes in the indications we already have approval for with a more convenient -- with 213 being a more convenient option. And we have the opportunity to expand the indications that we have FcRn approval in.

因此，我們在此有一個選項組合，將持續建構 FcRn 領域、探索 FcRn 生物學，並為患者帶來價值。目前我們尚未公開 213 的臨床開發計畫細節，但它已具備進入第三期（Phase III）的條件，我們會快速推進。我們也看到幾個不同的機會。其中之一，是在我們已獲核准的適應症中，以更便利的選項來改善患者結局——213 會是一個更便利的選擇。此外，我們也有機會擴大我們已取得 FcRn 核准的適應症範圍。

So continuing to expand the boundaries, if you will, of FcRn biology. So we're really excited about our full FcRn portfolio, and we think that it will be able to deliver growth for many years to come. Thanks for the question.

也就是說，持續拓展 FcRn 生物學的邊界。因此我們對完整的 FcRn 產品組合感到非常興奮，並認為它將能在未來多年帶來成長。謝謝你的提問。

Samantha Semenkow, Citi.

Samantha Semenkow，花旗（Citi）。

Jacob Mekhael, KBC Securities.

Jacob Mekhael，KBC Securities。

I have one maybe further down the line on the oral FcRn and your collaboration with UMP. Maybe if you can share a bit more on how that's evolving. And a follow-up on that, we see other disease areas that if you introduce an oral, that could expand the market. So I'm keen to hear your view on the potential impact of an oral FcRn on the overall biologics market in MG and CIDP. Could this lead to further market expansion? Or do you see it more as a tool to prevent the competition from taking existing share?

我有一個可能更長期的問題，關於口服 FcRn 以及你們與 UMP 的合作。如果可以的話，能否多分享一些這項合作目前的進展情況？以及基於此的追問：我們看到在其他疾病領域，如果引入口服劑型，可能會擴大市場。因此我很想聽聽你們對口服 FcRn 對 MG 與 CIDP 整體生物製劑市場潛在影響的看法。這是否可能帶來進一步的市場擴張？還是你們更把它視為防止競爭對手侵蝕既有市占的工具？

Yes. Thanks for the question on the oral. Our partnership with UMP is progressing incredibly well. They are great partners. And we've -- we're partnering with them on a number of targets and FcRn being the first one.

是的。謝謝你關於口服的提問。我們與 UMP 的合作進展非常順利。他們是很棒的合作夥伴。而且我們——正與他們在多個標的上合作，FcRn 是第一個。

And exactly, as you said, our strategy here is that we believe an oral FcRn can expand the market. You can imagine there are patients earlier in disease that would prefer to have an oral option rather than an injectable even if it is a PFS that we have with VYVGART. So the strategy here across all of our indications, and as I mentioned on the prior question, even potentially in new indications is that we'll be able to bring more convenient options to patients that continue to deliver the same efficacy and safety standard that we've seen with VYVGART. Thanks for the question.

而且正如你所說，我們在這裡的策略是：我們相信口服 FcRn 能夠擴大市場。你可以想像，有些疾病較早期的患者，即使我們有 VYVGART 的預充式注射器（PFS），他們仍可能更偏好口服選項而非注射。因此，我們在所有適應症上的策略——如我在上一個問題中提到的，甚至可能在新的適應症上——是能為患者帶來更便利的選擇，同時維持我們在 VYVGART 上所看到的同等療效與安全性標準。謝謝你的提問。

Victor Floch, BNP Paribas.

Victor Floch，法國巴黎銀行（BNP Paribas）。

Maybe just a quick follow-up on 213. And I mean, I was looking at the initiation of the Graves' disease Phase III. And I was just wondering how should we think in terms of additional VYVGART Phase III studies in new indication moving forward? Basically, should we assume that the Graves' disease program will be VYVGART's final Phase III for new indication and that any promising signal from the ongoing proof-of-concept studies will instead be pursued with the ARGX-213 due to IP consideration. So any comments on the life cycle management there would be helpful.

可能只是對 213 的一個快速追問。我看到你們啟動了葛瑞夫茲病（Graves' disease）的第三期（Phase III）。我想請問，未來我們應該如何看待 VYVGART 在新適應症上的額外第三期研究？基本上，我們是否應該假設葛瑞夫茲病計畫會是 VYVGART 在新適應症上的最後一個第三期（Phase III），而正在進行的概念驗證（proof-of-concept）研究若出現任何有前景的訊號，將因智慧財產權（IP）考量而改以 ARGX-213 推進？因此，若能就產品生命週期管理提供一些評論會很有幫助。

And maybe if I can just squeeze one on the on CDP. You've mentioned that the penetration of biologics in MG was around 20%. Can you share the same metrics for CDP? And can you discuss like the drivers that you have to further penetrate the market there?

另外如果我可以再塞一個關於 CDP 的問題。你們提到 MG 的生物製劑滲透率約為 20%。能否分享 CDP 的相同指標？以及能否談談你們在那裡進一步提升市場滲透率的驅動因素？

Yes. Let me take the first question, and I'll hand over the question on CIDP to Sandrine. So for Graves' disease, it's -- we're pursuing Graves as an indication for VYVGART. And I wouldn't assume that future indications that we name all are going to 213 or 124 or any of our future pipeline. We still see that we have a lot of runway with VYVGART and a strong development program with VYVGART.

好的。我先回答第一個問題，然後把 CIDP 的問題交給 Sandrine。關於葛瑞夫茲病，我們——正在以 VYVGART 推進葛瑞夫茲病這個適應症。我不會假設我們未來所命名的所有適應症都會轉到 213 或 124 或我們未來管線中的任何產品。我們仍然認為 VYVGART 還有很長的成長跑道，並且 VYVGART 的開發計畫也很強勁。

So we continue to invest and we'll continue to grow VYVGART. And in parallel, we'll be developing 213, 124 and the oral as well. As I mentioned earlier, we see those molecules as a real expansion opportunity for FcRn. But let me hand it over to Sandrine to talk about the CIDP opportunity and market growth opportunity.

因此我們會持續投資並持續擴大 VYVGART。同時，我們也會並行開發 213、124 以及口服方案。如我先前提到的，我們將這些分子視為 FcRn 的真正擴張機會。不過我先把話題交給 Sandrine，請她談談 CIDP 的機會以及市場成長機會。

Yes. And I like that you picked that up on the 80% in MG being still among orals, where there is a huge market opportunity. We see something similar with CIDP. So the total number of diagnosed patients for CIDP in the US is 42,000 patients and 24,000 of these patients are treated.

好的。我也很高興你注意到 MG 中仍有 80% 是口服藥物，這代表有巨大的市場機會。我們在 CIDP 也看到類似情況。在美國，CIDP 的確診患者總數為 42,000 人，其中有 24,000 人正在接受治療。

That means the remaining are not right now. They have been diagnosed, but they are not treated. Out of these 24,000 patients, 12,000 of them are on IVIg, but are not optimally treated. And these are the one we have been focusing on when we started launching because these are the ones that have a reason to switch to something better and that's why we feel that VYVGART is the answer.

這表示其餘患者目前並未接受治療。他們已被診斷出來，但尚未治療。在這 24,000 名患者中，有 12,000 人使用 IVIg，但治療並非最佳化。而這些正是我們在上市初期所聚焦的族群，因為他們有理由轉換到更好的治療，這也是我們認為 VYVGART 是答案的原因。

The other 12,000 that are treated and feel optimally treated with IVIg could also potentially switch to VYVGART because could benefit from efficacy like the grip strength data that we -- where we showed strong efficacy. So if you have to compare the 80:20 split of MG with what we see here in CIDP. I would say out of the 42,000, only 24 dozen are treated. The others are not. So there is room there.

另外那 12,000 名接受治療且覺得 IVIg 治療效果最佳的患者，也可能轉用 VYVGART，因為可能受益於我們所展示的療效，例如握力數據——我們在那裡顯示了強勁的療效。所以如果你必須把 MG 的 80:20 分布拿來和我們在 CIDP 這裡看到的情況比較。我會說在 42,000 人之中，只有 24 打正在接受治療。其他人沒有。所以那裡還有空間。

And then 12,000 feel optimally treated, but there is an opportunity for us to expand the market beyond what we already see today. So still a big potential beyond what we have focused on until now.

然後有 12,000 人覺得目前治療已達最佳，但我們仍有機會把市場擴大到超出我們今天所看到的規模。所以在我們迄今為止所聚焦的範圍之外，仍然有很大的潛力。

Myles Minte, William Blair.

Myles Minte，William Blair。

This is John, on for Myles. Wondering if you could talk a little bit about how you're viewing the evolving CIDP complement development landscape, especially as some of the early [ C1 ] inhibitor data has suggested a potentially best-in-class profile there? And as a follow-up, maybe if you could just talk a little bit about your views on knocking down both the lectin and classical pathway with C2 versus just knocking down the classical pathway?

我是 John，代替 Myles 發問。想請你談談你如何看待 CIDP 補體（complement）研發版圖的演進，特別是一些早期的 [ C1 ] 抑制劑數據顯示其可能具備同類最佳（best-in-class）的特徵？另外追問一下，也請你談談你對於用 C2 同時抑制凝集素（lectin）與經典（classical）途徑，與僅抑制經典途徑之間的看法？

Yes. Thanks for the question on empasiprubart. It's exciting to be bringing our second medicine potentially to market with our readout -- our first Phase III readout at the end of this year. Certainly, from my perspective, on the competitor data, I see this as a real confidence booster for why we're pursuing empasiprubart in CIDP. It demonstrates that they're in CIDP.

是的。謝謝你關於 empasiprubart 的提問。我們很興奮，可能在今年年底迎來第一次第三期讀出，並將我們的第二款藥物推向市場。當然，從我個人角度看，競品數據反而讓我更有信心，證明我們在 CIDP 追求 empasiprubart 的方向是對的。這顯示他們確實是在 CIDP 領域。

IgM is part of the driver of the disease and complement is at play. And so that reinforces why we think empasiprubart could have best-in-class potential in CIDP.

IgM 是疾病驅動因素的一部分，而補體也在其中發揮作用。因此，這也強化了我們認為 empasiprubart 在 CIDP 具有同類最佳潛力的判斷。

And I think from a company perspective, argenx is very well positioned in CIDP. There hasn't been innovation in CIDP in 30 years. The first innovation was VYVGART. And now as we develop that market, we also, in parallel, are developing empasiprubart.

而且我認為從公司角度來看，argenx 在 CIDP 領域的布局非常到位。CIDP 已經 30 年沒有創新。第一個創新是 VYVGART。而現在在我們開拓該市場的同時，也在並行推進 empasiprubart 的開發。

So I think what you'll see is that we have the opportunity between VYVGART and empasiprubart to really shape that market and transform the market. And I think it will look very different in the future from where it looks today as we really raise expectations of patients of what they can get from their medicine. So we're excited for that.

所以我認為你將會看到，我們有機會透過 VYVGART 與 empasiprubart 來真正塑造並改變這個市場。隨著我們真正提高患者對藥物可帶來效果的期待，未來的樣貌將會與今天非常不同。所以我們對此感到興奮。

In terms of the classic and the lectin pathway, the reason we chose C2 was very specific. And one of the reasons for that is because there are indications where lectin plays an important -- the lectin pathway plays an important role. I was talking about DGF earlier. And so we think that it gives us better pipeline and a product opportunity by targeting C2 to be able to get the efficacy benefit there, but also the safety benefit of leaving the alternate pathway intact. Thanks for the question.

至於經典途徑與凝集素途徑，我們選擇 C2 的原因非常明確。其中一個原因是，某些適應症中凝集素——凝集素途徑扮演重要角色。我先前提到過 DGF。因此我們認為，鎖定 C2 能帶來更好的產品機會與研發管線機會：既能取得療效上的收益，也能保留替代（alternate）途徑完整，從而帶來安全性上的收益。謝謝你的提問。

Samantha Semenkow, Citi.

Samantha Semenkow，花旗（Citi）。

Apologies for the technical difficulties. Just wanted to follow up on a couple of the previous questions on combination strategy for both CIDP and MG. How do you think about the market evolving? It seems combo therapies is a growing theme within I&I. And as you start to see some of that data in the platform trials that you talked about, Karen, how do you see the market evolving there and your opportunity to continue being a leader in both indications?

抱歉剛才有技術問題。我想追問前面幾個關於 CIDP 與 MG 的聯合用藥策略的問題。你如何看待市場的演變？看起來在 I&I 領域，聯合療法正成為一個日益增長的主題。而當你開始在你提到的平台試驗中看到一些數據時，Karen，你如何看待那裡市場的演變，以及你在兩個適應症中持續保持領導地位的機會？

Yes. Thanks for the question, and we had some technical difficulties earlier today as well. So no problem. In terms of our combination strategy, as you see -- as you said, you can see that this is emerging in I&I in a way that it's similar to how it did in oncology as well. So we're at the forefront of that, and we're driving innovation.

是的。謝謝你的提問，我們今天稍早也遇到一些技術問題。所以沒問題。就我們的聯合策略而言，正如你所說，你可以看到這在 I&I 領域正逐步浮現，類似於它在腫瘤學領域的發展方式。因此我們走在最前沿，並推動創新。

And I think we're very clear on what we want to achieve. The value proposition for combination therapy has to be that it substantially raises efficacy outcomes for patients. I think that's the bar that we need to see in combination therapy. And you have to be able to deliver that efficacy benefit without a safety trade-off.

而且我認為我們非常清楚想要達成什麼。聯合療法的價值主張必須是能為患者顯著提升療效結果。我認為這就是我們對聯合療法所要求的門檻。同時，你必須能在不犧牲安全性的前提下交付這樣的療效收益。

So that's what we'll be looking for in the platform studies with the different approaches, the different combinations that we'll be testing. And as I said earlier, the reason we did a platform study is that, that will allow us to test these quite quickly. And then when we see a signal, we'll be able to move quickly into Phase III with the goal that we always have as a company of elevating outcomes for patients. Thanks for the question.

因此，這就是我們在平台研究中、針對不同方法與不同組合進行測試時所要尋找的。而且如我先前所說，我們之所以做平台研究，是因為它能讓我們相當快速地測試這些方案。然後一旦看到訊號，我們就能迅速推進到第三期，目標始終如一：提升患者的治療結果。謝謝你的提問。

That's all the questions we have time for. I'd like to hand the call back over to Karen Massey for closing remarks.

我們能回答的問題就到這裡。我想把電話交回給 Karen Massey 做結語。

Thank you, everyone, for the questions and the great discussion, and we'll see you next quarter.

謝謝各位的提問與精彩的討論，我們下個季度見。

That does conclude our conference for today. Thank you for participating. You may now all disconnect.

今天的會議到此結束。感謝各位參與。各位現在可以斷線。