Viridian Therapeutics Inc (VRDN) 2024 Q1 法說會逐字稿

Welcome to the Veridian Therapeutics First Quarter 2024 a conference call. (Operator Instructions). As a reminder, this conference call is being recorded. I would now like to hand the call over to Ms. Louisa Stone, Manager of Investor Relations at. Viridian, Please go ahead.

歡迎參加 Veridian Therapeutics 2024 年第一季電話會議。（操作說明）再次提醒，本次電話會議正在錄音。現在我謹將電話交給投資人關係經理路易莎‧史東女士。薇麗迪安，請繼續。

Thank you, and welcome, everyone to our first quarter 2024 earnings conference call. The press release reporting our financial results and corporate updates as available on the Investors page of our corporate website at www.veridian therapeutics.com.

謝謝各位參加我們2024年第一季財報電話會議。新聞稿報告了我們的財務表現和公司最新動態，詳情請瀏覽我們公司網站 www.veridian therapeutics.com 的投資者頁面。

Joining me on the call this morning are Steve Mahoney, our President and CEO, Thomas Ciulla, our Chief Medical Officer, and Shan Wu, our Chief Business Officer.

今天早上和我一起參加電話會議的有我們的總裁兼執行長史蒂夫·馬奧尼、首席醫療官托馬斯·丘拉和首席商務官吳珊。

Before we begin, I would like to remind everyone that this conference call and webcast will contain forward looking statements regarding our financial outlook in addition to regulatory product development and commercialization plans and research activities. These statements are subject to risks and uncertainties that could cause actual results to materially differ from those forecasted. A description of these risks can be found in our most recent Form 10-Q and 10-K on file with the SEC.

在會議開始之前，我想提醒大家，本次電話會議和網路直播將包含有關我們財務前景、監管產品開發和商業化計劃以及研究活動的前瞻性聲明。這些聲明存在風險和不確定性，可能導致實際結果與預測結果有重大差異。有關這些風險的描述，請參閱我們向美國證券交易委員會提交的最新 10-Q 表格和 10-K 表格。

I'd now like to turn the call over to Steve Mahoney, our President and CEO.

現在我想把電話交給我們的總裁兼執行長史蒂夫·馬奧尼。

Thanks, Louisa, and welcome, everyone, to our first quarter earnings call. I'll start by giving a brief overview of Viridian, and then we'll get into more detail about our programs and recent progress. For those of you who are new to the Veridian story, our strategy is to identify market opportunities where there's a clear unmet need and where there's potential for us to develop differentiated products.

謝謝路易莎，歡迎各位參加我們第一季財報電話會議。我先簡要概述 Viridian，然後我們將更詳細地介紹我們的專案和最近的進展。對於那些還不了解 Veridian 的人來說，我們的策略是尋找市場機會，在這些市場中存在明顯的未滿足需求，並且我們有潛力開發差異化產品。

We then aim to engineer the best possible therapeutics and then move rapidly to advance our programs to patients turning to Slide 4. Slide 4 shows our pipeline, which includes both the thyroid eye disease or TED portfolio as well as an FcRn targeting autoimmune portfolio.

然後，我們的目標是研發出最好的治療方法，並迅速推進我們的項目，使患者能夠看到幻燈片 4。投影片 4 展示了我們的產品線，其中包括甲狀腺眼疾 (TED) 產品組合以及 FcRn 標靶自體免疫產品組合。

We have several exciting updates to provide across our pipeline today, which we'll get into next. We’re really excited to highlight for you today the significant progress that we’ve made across the business so far this year. Beginning with our 001 IV program, we are pleased to announce that THRIVE, our Phase 3 trial evaluating 001 in patients with active TED, completed enrollment in March.

今天我們將發布一系列令人興奮的最新進展，接下來我們將詳細介紹。今天我們非常高興地向大家重點介紹今年以來我們在各個業務領域的重大進展。從我們的 001 IV 計畫開始，我們很高興地宣布，評估 001 在活動性 TED 患者中療效的 3 期試驗 THRIVE 已於 3 月完成入組。

In fact, not only did THRIVE reach the target enrollment of 90 patients in mid-March, but we exceeded enrollment for a total of 113 patients due to strong patient demand at our clinical trial sites.

事實上，THRIVE 不僅在 3 月中旬達到了 90 名患者的入組目標，而且由於臨床試驗點的患者需求強勁，我們最終入組的患者總數達到了 113 名。

We expect to share top line results for THRIVE in September 2024. THRIVE-2, our Phase 3, trial initiating 001 IV in patients with chronic TED continues enrolling and is on track for top line data at the end of this year

我們預計 2024 年 9 月公佈 THRIVE 的主要表現。THRIVE-2 是我們的 III 期臨床試驗，旨在啟動 001 IV 治療慢性甲狀腺眼疾 (TED) 患者，目前該試驗仍在招募患者，預計將於今年年底公佈主要數據。

Lastly, for 001, we are announcing that we anticipate filing a BLA for the 001 program in the second half of 2025 for our subcutaneous 003 program, which we believe has the potential to be best-in-class. We recently completed a positive Type C meeting with the FDA and we are moving forward with our preparations for our pivotal program in line with our previous guidance.

最後，關於 001 項目，我們宣布預計將於 2025 年下半年為我們的皮下 003 項目提交 001 項目的生物製品許可申請 (BLA)，我們相信該項目有潛力成為同類最佳。我們最近與 FDA 完成了一次積極的 C 類會議，並且我們正在按照先前的指導方針推進關鍵項目的準備工作。

We will provide additional updates for the 003 program before we start that pivotal program, which remains on track for mid-year we are also progressing our FcRn portfolio as planned. We are aiming to file an IND for 006 by the end of this year and we plan to share 008 non-human primate data in the second half of 2024.

在啟動這項關鍵的 003 專案之前，我們將提供更多專案進度資訊。該專案仍按計劃於年中啟動，同時，我們的 FcRn 產品組合也在按計劃推進。我們的目標是在今年年底前提交 006 的 IND 申請，並計劃在 2024 年下半年分享 008 非人靈長類動物資料。

Lastly, we ended the quarter with $613 million in cash equivalents and short-term investments, and we maintain our cash runway into the second half of 2026 also as previously guided.

最後，本季末我們持有 6.13 億美元的現金等價物和短期投資，並且我們保持了先前預期的現金儲備，足以支撐到 2026 年下半年。

Now, I’d like to turn to our TED portfolio and talk more about the programs and our progress in a bit more detail. As, a reminder TED is an autoimmune condition characterized by inflammation and damage to the tissues around and behind the eyes.

現在，我想談談我們的 TED 專案組合，並更詳細地談談這些專案以及我們所取得的進展。提醒一下，TED 是一種自體免疫疾病，其特徵是眼睛周圍和後方的組織發炎和受損。

This leads to symptoms including proptosis or bulging of the eyes, redness, swelling, double vision and retraction to the eyelids. In severe cases, TED can be sight threatening with those symptoms as a backdrop, there is already a large market opportunity in TED that comes with global growth potential and an expansion that can come with better options for patients. There is an estimated 190,000 people in the US, alone who are living with moderate to severe TED.

這會導致眼球突出、發紅、腫脹、複視和眼瞼回縮等症狀。在嚴重的情況下，眼部甲狀腺功能減退症（TED）可能會威脅視力。在這些症狀的背景下，眼部甲狀腺功能減退症已經存在巨大的市場機會，具有全球成長潛力，隨著市場擴張，患者將有更好的選擇。據估計，光在美國就有 19 萬人患有中度至重度 TED。

These patients are only served by one marketed IGF-1R IV therapy currently, which generated approximately $1.8 billion in sales in just the US, alone in 2023. This approved therapy requires eight infusion every three weeks, which can be a significant burden for patients.

目前市面上只有一種 IGF-1R IV 療法可供這些患者使用，該療法僅在 2023 年就在美國創造了約 18 億美元的銷售額。這種核准的療法需要每三週進行八次輸液，這對患者來說可能是一個很大的負擔。

We see opportunity for us to provide differentiated options for TED patients with both our IV and subcu programs because TED is an autoimmune disease characterized by flaring symptoms, patients with moderate to severe symptoms struggle with quality-of-life issues that make it hard for them to drive, work and even sleep.

我們看到，我們有機會透過我們的靜脈注射和皮下注射方案為 TED 患者提供差異化的選擇，因為 TED 是一種自體免疫疾病，其特徵是症狀反覆發作，中度至重度症狀的患者會面臨生活品質問題，使他們難以開車、工作甚至睡覺。

Because it is a flared-based disease it is considered a new start market, which means that it doesn’t matter when a patient was diagnosed because you need to treat the flare or the onset of those symptoms this new start market also means that all patients experiencing symptoms will have the opportunity to choose from available treatment options with no chronic treatment to displace.

由於這是一種以發作為基礎的疾病，因此被視為一個新市場，這意味著患者何時被診斷出來並不重要，因為你需要治療的是發作或症狀的出現。這個新市場也意味著所有出現症狀的患者都將有機會從可用的治療方案中進行選擇，而無需取代慢性治療。

Once a flare is treated, patients do not remain on an anti-IGF-1R therapy. So, when a subsequent flare arises, physicians will have the opportunity to choose from available treatment options, including potential new options to manage these flares in their patients.

一旦病情發作得到控制，患者就不需要繼續接受抗IGF-1R治療。因此，當病情再次發作時，醫生將有機會從可用的治療方案中進行選擇，包括潛在的新方案來控制患者的病情發作。

This is a great position for our IV and subcu programs in TED because we believe that we are developing potential best-in-class therapies in a drug class that is shown to be highly effective in inhibiting IGF-1R and in treating TED.

對於我們的靜脈注射和皮下注射治療 TED 計畫來說，這是一個絕佳的機會，因為我們相信，我們正在開發一類藥物中潛在的最佳療法，該藥物已被證明在抑制 IGF-1R 和治療 TED 方面非常有效。

Turning to the specifics of our product candidates. Viridian is developing two anti-IGF-1R antibodies for TED. 001, which is delivered intravenously, and 003, which is delivered subcutaneously with the potential for self-administration. As you can see here, 003 and 001, have the same binding domain and we expect them to bind IGF-1R similarly.

接下來，我們來具體了解我們的候選產品。Viridian 正在開發兩種用於治療 TED 的抗 IGF-1R 抗體。 001 為靜脈注射，003 為皮下注射，並有可能實現患者自行給藥。正如您在這裡看到的，003 和 001 具有相同的結合域，我們預計它們將以類似的方式結合 IGF-1R。

They differ because 003 is engineered to have an extended half-life, which we have shown to be 40 to 50 days in healthy volunteers, which is 4 to 5 times that of 001 its parent molecule. With 001, we hope to have a fast to market differentiated IV therapy for patients with fewer doses and a shorter infusion time than the current standard of care. With 003, we hope to have we hope to develop a convenient, less frequent, low volume therapy that patients can self administer at home.

它們之間的差異在於 003 經過工程設計，具有更長的半衰期，我們已經證明，在健康志願者中，其半衰期為 40 至 50 天，是其母體分子 001 的 4 至 5 倍。我們希望透過 001，為患者提供一種快速上市的差異化靜脈輸液療法，與目前的標準療法相比，該療法劑量更少，輸液時間更短。我們希望透過 003 開發出一種方便、頻率較低、用量較少的療法，患者可以在家中自行進行治療。

Let’s review the 001 program, our progress and what makes us excited about the Phase 3 readout that we expect in September. On Slide 11, this is a reminder that we’ve already shown robust clinical activity with 001 after just two infusions in a Phase 2 clinical trial in active TED.

讓我們回顧一下 001 項目、我們的進展以及讓我們對 9 月即將公佈的第三階段結果感到興奮的原因。第 11 張投影片提醒我們，在針對活動性 TED 的 2 期臨床試驗中，僅兩次輸注 001 就已顯示出強大的臨床活性。

This robust activity is across all key areas of the disease proptosis or the bulging of the eyes, clinical activity score and diplopia or double vision we have added data from TEPEZZA clinical trials after two doses on this slide to show the data side by side while cross trial comparisons are difficult, we are encouraged by the clinical responses observed after just two doses of 001.

這種強勁的活性涵蓋了疾病的所有關鍵領域，包括眼球突出或眼球膨出、臨床活動評分和複視或重影。我們在這張投影片上加入了 TEPEZZA 臨床試驗中兩劑給藥後的數據，以便並排顯示數據。雖然跨試驗比較很困難，但我們對僅兩劑 001 後觀察到的臨床反應感到鼓舞。

On Slide 12, you can see that 001 was well tolerated in active TED patients with no serious adverse events, no infusion reactions, and no discontinuations. Similarly to active TED in patients with chronic TED just after just two infusions, 001 meaningfully reduced disease burden across each disease point as well.

從投影片 12 可以看出，001 在活躍的 TED 患者中耐受性良好，沒有發生嚴重不良事件、輸液反應和停藥。與慢性 TED 患者在接受兩次輸注後出現的活動性 TED 類似，001 也顯著降低了每個疾病點的疾病負擔。

On Slide 14, VRDN-001 was also well tolerated in chronic TED patients based on this Phase 2 data, we believe that the clinical regimen of VRDN-001 with fewer infusions, shorter infusion time and lower cumulative drug exposure has the potential to be a better choice for moderate to severe patients with TED.

根據第 2 期數據，VRDN-001 在慢性 TED 患者中也具有良好的耐受性（見投影片 14）。我們認為，VRDN-001 的臨床治療方案，輸注次數較少、輸注時間較短、累積藥物暴露量較低，有可能成為中度至重度 TED 患者的較佳選擇。

Now turning to our Phase 3 trial for VRDN-001, I would like to take a moment to thank all the patients and clinical trial site teams who have participated in our THRIVE trial we’re not done yet, and our achievements so far would not be possible without them. As we announced today, we completed enrollment for THRIVE in March with 113 patients, which exceeded our enrollment target of 90 patients due to strong demand and interest at our clinical sites.

現在，我想談談 VRDN-001 的 3 期試驗。在此，我要感謝所有參與 THRIVE 試驗的患者和臨床試驗中心團隊——我們還沒有完成，沒有他們的支持，我們迄今為止的成就是不可能實現的。正如我們今天宣布的那樣，我們在 3 月完成了 THRIVE 的招募工作，共招募了 113 名患者，由於我們臨床中心的強勁需求和興趣，這超過了我們 90 名患者的招募目標。

About half of THRIVE patients were from the US, and the other half came from Europe we expect to provide top line results for this study in September of this year. THRIVE-2, our second pivotal study in TED is ongoing and on track for top line readout at the end of this year.

THRIVE 研究的患者約有一半來自美國，另一半來自歐洲。我們預計將於今年 9 月公佈這項研究的主要結果。THRIVE-2 是我們在 TED 領域的第二項關鍵研究，目前正在進行中，預計今年年底公佈主要結果。

In addition to THRIVE and THRIVE-2, we recently initiated STRIVE, which is a planned safety study. STRIVE is a study of VRDN-001 in TED patients to complete the sufficient safety database for BLA submission alongside the patient numbers from THRIVE and THRIVE-2.

除了 THRIVE 和 THRIVE-2 之外，我們最近還啟動了 STRIVE，這是一項計畫中的安全性研究。STRIVE 是一項針對 TED 患者的 VRDN-001 研究，旨在與 THRIVE 和 THRIVE-2 的患者數量一起，完善足夠的安全性資料庫，以便提交 BLA 申請。

In conclusion, with VRDN-001, we are developing a potentially differentiated IV therapy for patients with fewer doses and shorter infusion time than the current standard of care, while inhibiting the same IGF-1R target which has been shown clinically and commercially to be effective in treating TED.

總而言之，我們正在利用 VRDN-001 開發一種潛在的差異化靜脈輸液療法，與目前的標準療法相比，該療法劑量更少、輸注時間更短，同時抑制相同的 IGF-1R 靶點，該靶點已在臨床和商業上證明可有效治療 TED。

We are very excited about bringing forward VRDN-001, as a potential option for patients, which could mean significantly less drug for patients fewer visits than the infusion center, lower volumes and less infusion share time.

我們非常高興地推出 VRDN-001，作為患者的潛在選擇，這意味著患者所需的藥物量將大大減少，就診次數將比輸液中心少，輸液量將減少，輸液共享時間也將減少。

Our next program, subcutaneous VRDN-003 will take this differentiation even further with the possibility of lower frequency subcutaneous administrations and potential for at home self-administration using auto injectors. We know from market examples that a later entrance subcu therapy can convert meaningful portions of an existing IV market.

我們的下一個項目，皮下注射 VRDN-003，將進一步實現這種差異化，有可能降低皮下注射頻率，並有可能使用自動注射器在家中進行自我注射。我們從市場案例中得知，後期進入市場的皮下療法可以轉化現有靜脈輸液市場中相當一部分重要份額。

We’ve included two of those examples here. In each of the cases on this slide, [subcu] offerings grew the overall market size of the class, in addition to quickly commanding a significant share of the IV markets and keep in mind that these subcu examples have the same or more frequent dosing than their IV counterparts.

我們在此列舉了其中兩個例子。在本幻燈片上的每個案例中，皮下注射產品都擴大了該類產品的整體市場規模，並且迅速佔據了靜脈注射市場的很大份額。請記住，這些皮下注射產品與靜脈注射產品的給藥頻率相同或更高。

This would not be the case with VRDN-003, which is designed to have potentially a best-in-class dosing profile. Also, it is important to point out in both examples that neither of these are in new start markets again the TED market is a new start market where we need to treat flaring disease or onset of symptoms as opposed to trying to convert patients from a long-term chronic therapy. With VRDN-003, we hope to provide patients with an anti-IGF-1R therapy that is a better option with respect to less overall drug exposure and more convenience.

VRDN-003 則不然，它的設計目標是擁有同類最佳的劑量特性。此外，需要指出的是，在這兩個例子中，它們都不屬於新市場。 TED 市場是一個新市場，我們需要治療疾病的急性發作或症狀的出現，而不是試圖將患者從長期慢性治療中轉化過來。我們希望透過 VRDN-003 為患者提供一種抗 IGF-1R 療法，這種療法在減少藥物暴露總量和提高便利性方面是更好的選擇。

On Slide 19, we show the complete data set from our Phase 1 healthy volunteer study of VRDN-003 to assess PK and PD the update here is the inclusion of the last cohort, cohort 5, where participants receive two doses of 003 28 days apart. The data confirms the differentiated PK and PD for VRDN-003 seen in the first four cohorts with an extended half life of 40 to 50 days and sustained increased levels of the PD biomarker IGF-1.

在第 19 張投影片中，我們展示了 VRDN-003 的 1 期健康志願者研究的完整數據集，以評估其藥物動力學和藥效學。這裡的更新是納入了最後一個隊列，即第 5 隊列，其中參與者在 28 天後接受兩劑 003。數據證實了前四個隊列中觀察到的 VRDN-003 的差異化 PK 和 PD，其半衰期延長至 40 至 50 天，且 PD 生物標記 IGF-1 的水平持續升高。

On Slide 20, you can see that the subcutaneous VRDN-003 was well tolerated in the Phase 1 study, including in the latest cohort 5 with no serious adverse events or discontinuations related to treatment, and observed adverse events were generally grade 1 and mild. As we shared previously, our pharmacokinetic modeling for VRDN-003 that showed that or predicts that three potential dosing regimens are available to us.

從投影片 20 可以看出，皮下注射 VRDN-003 在 1 期研究中耐受性良好，包括最新的第 5 組，沒有發生與治療相關的嚴重不良事件或中止治療，觀察到的不良事件通常為 1 級且程度較輕。正如我們之前分享的那樣，我們對 VRDN-003 的藥物動力學模型顯示或預測，我們有三種潛在的給藥方案可供選擇。

VRDN-003, every eight weeks, every four weeks and every two weeks could achieve or exceed the exposure levels of VRDN-001 that we saw in the active and chronic studies that were correlated to robust clinical activity for our Phase 2 clinical trials in TED.

VRDN-003 每八週、每四周和每兩週可達到或超過我們在活躍和慢性研究中看到的 VRDN-001 暴露水平，這些暴露水平與我們在 TED 的 2 期臨床試驗中的強勁臨床活性相關。

This gives us a lot of optionality as we move towards our pivotal studies for VRDN-003 and importantly, gives us the potential to develop for patients a best-in-class low volume subcu delivery option we are pleased to announce today that we have completed our Type C meeting with the FDA, and we are on track to initiate pivotal clinical trials for the VRDN-003 program. We will share more details on the pivotal trial design before we start those studies.

這為我們推進 VRDN-003 的關鍵性研究提供了很大的選擇餘地，更重要的是，它使我們有可能為患者開發出一流的低容量皮下給藥方案。我們很高興地宣布，我們今天已經完成了與 FDA 的 C 類會議，並且我們正按計劃啟動 VRDN-003 項目的關鍵性臨床試驗。在開始這些研究之前，我們將分享更多關於關鍵性試驗設計的細節。

Now turning to our FcRn portfolio. On Slide 24, in addition to TED and consistent with our development strategy, we are developing an exciting portfolio of potential best-in-class FcRn inhibitors to address the unmet needs of patients living with autoantibody mediated autoimmune diseases. FcRn inhibitors represent a large market opportunity.

現在來看看我們的FcRn投資組合。在第 24 張投影片中，除了 TED 之外，並且與我們的發展策略一致，我們正在開發一系列令人興奮的、潛在的同類最佳的 FcRn 抑制劑，以滿足患有自身抗體介導的自身免疫性疾病的患者的未滿足需求。FcRn抑制劑代表著巨大的市場機會。

The first FcRn inhibitor, [Efgart or Vyvgart] is approved for myasthenia gravis and is in registration for CIPD and it’s already annualizing to over $1 billion in annual sales. Myasthenia gravis alone is a large market with projected sales of over $4 billion annually by 2028.

第一個 FcRn 抑制劑 [Efgart 或 Vyvgart] 已獲準用於治療重症肌無力，目前正在註冊用於治療 CIPD，其年銷售額已超過 10 億美元。光是重症肌無力就是一個龐大的市場，預計到 2028 年，其年銷售額將超過 40 億美元。

In addition to myasthenia gravis, as you can see from the slide, there are additional sizable autoimmune indications that would meaningfully add to the FcRn opportunity.

除了重症肌無力之外，正如你從幻燈片中看到的那樣，還有其他一些相當大的自體免疫疾病適應症，這些適應症將顯著增加 FcRn 的應用機會。

Our FcRn franchise includes two assets, VRDN-006 and VRDN-008. With VRDN-006, we are excited to have the only other FcRn targeting Fc fragment in development other than efgartigimod. Argenx has shown that its Fc fragment achieves substantial efficacy while sparing an effect on albumin or LDL and shows better tolerability than the full length antibodies. We are on track to submit an IND for VRDN-006 by the end of this year and look forward to sharing more about the program in the future.

我們的 FcRn 特許經營權包括兩項資產，VRDN-006 和 VRDN-008。VRDN-006 是除 efgartigimod 之外，唯一正在開發的靶向 FcRn 的 Fc 片段，我們為此感到非常興奮。Argenx 已證明，其 Fc 片段在不影響白蛋白或 LDL 的情況下，可達到顯著療效，並且比全長抗體具有更好的耐受性。我們正按計劃於今年年底前提交 VRDN-006 的 IND 申請，並期待在未來分享更多關於該計劃的資訊。

Next on the right is VRDN-008. Our protein engineering efforts identified a molecule derived from Fc fragments that both extended the half-life and generated meaningfully deeper IgG reductions in animal models.

右邊下一個是 VRDN-008。我們的蛋白質工程研究發現了一種源自 Fc 片段的分子，既延長了半衰期，又在動物模型中產生了更顯著的 IgG 減少作用。

We believe VRDN-008 is a potential best-in-class extended half-life molecule targeting FcRn with the potential to more durably suppress IgG we are on track to provide VRDN-008 non-human primate data in the second half of this year as guided and we are excited to bring forward this portfolio of next generation FcRns to potentially offer patients a more convenient dosing profile compared to current weekly IV or subcu infusions.

我們相信 VRDN-008 是一種潛在的同類最佳的長半衰期分子，針對 FcRn，具有更持久抑制 IgG 的潛力。我們正按計畫在今年下半年提供 VRDN-008 非人靈長類動物數據。我們很高興推出這新一代 FcRn 產品組合，與目前每週一次的靜脈或皮下輸注相比，有望為患者提供更方便的給藥方案。

In addition, by aiming to improve the duration and depth of IgG suppression with VRDN-008, we hope to offer a best-in-class option for patients. Our team is executing. We have made excellent progress across the company in the first quarter of the year, and we look forward to continuing that momentum through the rest of the year to deliver on our multiple upcoming catalysts.

此外，我們希望透過 VRDN-008 提高 IgG 抑制的持續時間和深度，為患者提供一流的治療選擇。我們的團隊正在執行任務。今年第一季度，公司各方面都取得了顯著進展，我們期待在今年餘下的時間裡繼續保持這種勢頭，以實現我們即將推出的多個催化劑目標。

As I mentioned previously, we plan to report THRIVE top line in September and THRIVE-2 top line is on track for the end of the year. The FcRn programs are also proceeding as expected. It has been my pleasure today to provide these exciting updates across our portfolio and in particular for VRDN-001 and VRDN-003, reflecting the work that we’ve completed during this quarter.

正如我之前提到的，我們計劃在 9 月公佈 THRIVE 的營收數據，而 THRIVE-2 的營收數據預計將在年底前公佈。FcRn項目也如預期進行。今天我很高興為大家帶來我們產品組合的這些令人興奮的最新進展，特別是 VRDN-001 和 VRDN-003 的進展，這反映了我們在本季度完成的工作。

This progress and our recent achievements reflect our team’s ability to execute, and we are well positioned to continue to work and deliver on our exciting upcoming catalysts last but not least, we remain well capitalized, ending the quarter was $613 million and the runway is into the second half of 2026.

這項進展和我們最近的成就反映了我們團隊的執行能力，我們已做好充分準備，繼續推進並實現我們令人興奮的即將到來的催化劑項目。最後但同樣重要的是，我們資金充足，本季末資金為 6.13 億美元，足以支撐到 2026 年下半年。

So, with that, I’ll ask the operator to open the call for questions. Operator?

那麼，接下來我將請接線生開啟提問環節。操作員？

(Operator Instructions)

（操作說明）

Laura Chico from Wedbush Securities.

來自 Wedbush Securities 的 Laura Chico。

Good morning, and thanks very much for taking my question and congrats on the progress here. I guess I have two questions for you. First, with respect to the THRIVE data that's coming in September, I'm wondering if you can help kind of help us frame what success looks like on the efficacy side? But then also with respect to reduced drug exposure, how would you think this might impact the rate of hearing impairment events that you might see in trials?

早安，非常感謝您回答我的問題，也恭喜您在這裡的進展。我想問你兩個問題。首先，關於九月即將公佈的 THRIVE 數據，我想知道您能否幫助我們定義一下，在療效方面，成功是什麼樣的？但是，就減少藥物暴露而言，您認為這會對試驗中可能出現的聽力障礙事件的發生率產生怎樣的影響？

And then secondarily, just related to strive, I'm wondering if you could talk a little bit more about the inclusion of the active control? Thanks.

其次，關於 Strive，我想請您再詳細談談主動控制的加入？謝謝。

Yeah, sure. Thanks, Laura, for the question. In terms of what is good look like for THRIVE efficacy, as we’ve stated previously we think a profile that looks like TEPEZZA is similar to TEPEZZA would be a really good place for us to land.

當然可以。謝謝勞拉的提問。就 THRIVE 功效而言，正如我們之前所說，我們認為與 TEPEZZA 相似的特徵對我們來說將是一個非常好的落腳點。

So, with respect to hearing, yes, certainly, I think what we’re looking for in the same vein on efficacy for safety, getting a similar profile on the safety, because the safety profile for TEPEZZA is good, it’s benign, and you referenced hearing in particular.

所以，關於聽力方面，是的，當然，我認為我們尋求的也是同樣的有效性和安全性，以獲得類似的安全性特徵，因為 TEPEZZA 的安全性特徵很好，是良性的，而且你特別提到了聽力。

To the extent, the lower exposures improve upon that, that would be great. To the extent, it’s Cmax driven, we obviously have a lower Cmax just by virtue of the volume that we deliver versus TEPEZZA. So that could possibly be helpful. We’ll have to see. But I think in terms of what good looks like, we’d love to see a similar profile.

如果降低曝光量能改善這種情況，那就太好了。就 Cmax 而言，由於我們的產量比 TEPEZZA 低，顯然我們的 Cmax 也更低。這或許會有幫助。我們拭目以待。但我認為，就優秀標準而言，我們希望看到類似的形象。

With respect to your question on STRIVE, yes, I mean, look, the STRIVE is simply to complete the safety database, which is just normal blocking and tackling for a BLA submission. So, nothing there unusual on the active control arm. It’s just you got to run a well-controlled study.

關於你提出的 STRIVE 問題，是的，我的意思是，你看，STRIVE 只是為了完善安全資料庫，這只是 BLA 提交的正常步驟。所以，主動控制臂上沒有什麼異常狀況。你只需要進行一項控制良好的研究。

And so, we have the option of an active control arm of 3 mg/kg. It randomizes three to one, so the numbers will be heavily weighted towards the 10 mg, kg again, it’s all for the safety side of it.

因此，我們可以選擇 3 mg/kg 的活性對照組。它以三比一的比例隨機分配，因此數字會嚴重偏向 10 毫克/公斤，這都是為了安全起見。

So, I hope that answers the question.

希望這能解答你的疑問。

That does. Thank you.

確實如此。謝謝。

Alex Thompson with Stifel.

Alex Thompson 與 Stifel 合作。

Thanks for taking our questions. I guess I wanted to drill in a little bit more on safety in particular. I guess when you when you look back at the past, the safety profile from the Phase 3, versus the more recent chronic TED study, is it do you feel like the chronic TED study might represent a better if you ran it, as I say today with more focused hearing measuring that's more of a bar for safety? Or how are you thinking about like what to passive safety actually looks like today versus when those Phase 3, were started? And then for the top line for oh one in Phase 3, do you expect to be able to share any data beyond 15-weeks as part of that, either for safety or efficacy? Thanks.

謝謝您回答我們的問題。我想重點再深入探討一下安全方面的問題。我想，當你回顧過去，比較 3 期臨床試驗的安全性能和最近的慢性 TED 研究時，你是否覺得，如果像我今天所說的那樣，採用更集中的聽力測量方法，慢性 TED 研究可能會代表更好的安全標準？或者，您認為現今的被動安全與第三階段開始時相比，實際上體現了哪些變化？那麼，對於 oh one 在 3 期臨床試驗中的主要進展，您是否預計能夠分享超過 15 週的任何數據，無論是安全性還是有效性？謝謝。

Well, I can take the second one first, Alex. No, top line is top line. So, it’ll be a readout at the 15-week end point. With respect to your question on what would it sounds like you’re asking what is TEPEZZA post the clinical trials and what that real world experience is. Yes, that is all part of it. Like, we’re trying to understand that as well. I mean, I know Amgen is trying to understand that.

好吧，我可以先做第二個，亞歷克斯。不，第一行就是第一行。所以，這將是15週結束時的讀數。關於您提出的「TEPEZZA 在臨床試驗之後是什麼狀態，以及在現實世界中的經驗如何」這個問題。是的，這一切都是其中的一部分。我們也試著理解這一點。我的意思是，我知道安進公司正在努力了解這一點。

The physician community, the patient community, they’re all trying to understand that and maybe, Tom Ciulla, do you want to just explain how we’re approaching our reporting of AEs in the same way that depends a bit.

醫生群體、患者群體，他們都在努力理解這一點，湯姆·丘拉，你能否解釋一下我們是如何以同樣的方式報告不良事件的？這取決於具體情況。

So, Alex, as you know, the field is evolving that, as you alluded to, the updated guidance from the FDA, which led to a label change for a temporal, as you know, and in current clinical practice, physicians are assessing patients hearing at baseline during and after treatment.

所以，Alex，正如你所知，這個領域正在不斷發展，正如你提到的，FDA 更新了指導方針，導致暫時性藥物的標籤發生了變化，正如你所知，在目前的臨床實踐中，醫生們正在評估患者在治療期間和治療後的基線聽力。

So, we are recording adverse events using better returns. and as you know, this is just a standard way of recording patient reported changes in their health, including hearing.

因此，我們正在使用更好的回報來記錄不良事件。如您所知，這只是記錄患者自述健康狀況變化（包括聽力變化）的標準方法。

This is standard for any clinical trial, including TEPEZZA in their pivotal trials. We’re also assessing audiometry as is done in clinical practice at baseline and pre-specified points. So, we’re essentially doing what’s done currently in the evolving clinical practice.

這是所有臨床試驗的標準做法，包括 TEPEZZA 的關鍵性試驗。我們也按照臨床實務中的方法，在基線和預先指定的時間點評估聽力測試結果。所以，我們本質上是在做目前不斷發展的臨床實務中正在做的事情。

Great.

偉大的。

Michael Yee with Jefferies.

Michael Yee，來自 Jefferies。

Hello. Okay, great. I guess, we can hear you. So two questions. First was, on the ongoing THRIVE study, can you talk a little bit about how you can control for the hearing impairment and hearing loss events? I know that if you actually go look back at some of the TEPEZZA post marketing studies, that there’s some commentary and analysis around how patients have some of this hearing loss already, and there’s factors already going on with somebody’s TED patients in the background.

你好。好的，太好了。我想，我們能聽到你說話。所以，我有兩個問題。首先，關於正在進行的 THRIVE 研究，您能否談談如何控制聽力障礙和聽力損失事件？我知道，如果你回顧一下 TEPEZZA 的一些上市後研究，你會發現其中有一些評論和分析指出，患者本身就存在一些聽力損失，而且在某些 TED 患者中也存在一些潛在的健康問題。

And so just trying to think about how you can screen and protect for that. And think about that as you go through your Phase 3, and then on the subcu plans for Phase 3, I think you said that you met with the FDA and you’re planning to start Phase 3. Can you just talk a little bit about how that meeting went? And I know there were some uncertainties about going directly into Phase 3, so just talk a little bit about your confidence there or anything else that you need to do in order to start the Phase 3 for subcu. Thank you.

所以，我一直在思考如何篩檢和防範。在您進行第三階段試驗時，請考慮這一點。關於第三階段的皮下注射計劃，我想您說過您與FDA會面，並計劃開始第三階段試驗。能簡單談談那次會議的情況嗎？我知道直接進入第三階段存在一些不確定性，所以請您談談您對此的信心，或者為了開始皮下注射的第三階段，您還需要做哪些其他事情。謝謝。

Yeah, great. Thanks, Mike let me take the second one first and then I’ll ask Tom Ciulla to weigh in on the baseline hearing question that you had first. So, with respect to the VRDN-003 program, excuse me, we did have a positive meeting. We have not received the minutes yet.

好的，太棒了。謝謝，麥克，讓我先回答第二個問題，然後我會請湯姆·丘拉就你最初提出的基線聽力問題發表意見。所以，關於 VRDN-003 項目，恕我直言，我們確實進行了一次積極的會議。我們尚未收到會議記錄。

So, we had a positive meeting, and we are reiterating our guidance that we are going to start a pivotal program mid-year 20 this year. So, we will provide a lot more detail once we get on the other side of minutes, but before we start the study.

所以，我們進行了一次積極的會議，我們重申我們的指導方針，即我們將在今年年中啟動一項關鍵計劃。所以，在幾分鐘之後，但在開始研究之前，我們將提供更多細節。

So more to come but to answer your question, positive meeting, we feel good about our reiterating our guidance on starting that pivotal program. So we’re pretty excited there. With respect to the THRIVE and the baseline hearing,

後續還有更多消息，但回答你的問題，這是一次積極的會議，我們很高興能夠重申我們對啟動這項關鍵計劃的指導意見。所以我們對此感到非常興奮。關於THRIVE和基線聽力，

I’ll ask Tom Ciulla to jump in there, please.

請湯姆·丘拉加入進來。

Thanks, Michael. So as we said in the previous answer, adverse events in the studies reported via the measure of terms with standard methodology for reporting patient changes in their health, including hearing. And this was done in the TEPEZZA trials.

謝謝你，麥可。正如我們在先前的回答中所說，研究中報告的不良事件是透過標準方法對患者健康狀況（包括聽力）變化進行測量而得出的。而這正是在 TEPEZZA 審判中所做的。

As, I mentioned, we’re also using audiometry for monitoring and that’s consistent with the current clinical practice and FDA guidance. We do have an exclusion criteria for hearing loss at baseline, you can see that exclusion on clinicaltrials.gov.

正如我之前提到的，我們也使用聽力測試進行監測，這符合目前的臨床實踐和 FDA 指南。我們對基線聽力損失有排除標準，您可以在 clinicaltrials.gov 上查看該排除標準。

Okay, thank you.

好的，謝謝。

Gavin Clark-Gartner from Evercore.

來自 Evercore 的 Gavin Clark-Gartner。

Good morning and thanks for taking my questions. Just had two. First, on the Type C meeting for VRDN-003, does the FDA want to see any dose ranging work in TED patients as part of that pivotal? Or do you believe you can start dosing immediately in like a blinded pivotal portion of the trial?

早上好，感謝您回答我的問題。剛吃了兩個。首先，關於 VRDN-003 的 C 類會議，FDA 是否希望看到在 TED 患者中進行任何劑量範圍研究，作為這項關鍵性研究的一部分？或者您認為可以在試驗的盲法關鍵階段立即開始給藥嗎？

Yeah. So, thanks, Gavin. Like I said, give us a little bit more time. We’d like to see the minutes, but just take comfort from the fact that we are we feel positive coming out of that meeting and that we are starting our pivotal program, which is what we’ve been what we were guiding to previously, but we’ve now had that meeting. So, we feel good about where we’re going. But give us a little bit more time and we’ll be able to break down those details for you. that's kind of where we are, and we feel good.

是的。謝謝你，加文。正如我所說，請再給我們一點時間。我們想看看會議紀要，但令人欣慰的是，我們對這次會議感到積極，並且我們正在啟動我們的關鍵計劃，這正是我們之前一直努力的方向，而現在我們已經召開了這次會議。所以，我們對未來的發展方向感到滿意。但請給我們一些時間，我們會為您詳細解釋這些細節。目前情況就是這樣，我們感覺不錯。

Sounds good. We will, await more details. And are you able to provide any details on how the THRIVE baseline characteristics compared to TEPEZZA’s Phase 3?

聽起來不錯。我們將等待更多細節。您能否提供一些關於 THRIVE 基線特徵與 TEPEZZA 第三階段特徵比較的詳細資訊？

Yes, that’s another one, Gavin. I mean, it’s a great question. I totally appreciate it. It’s just that we’re just not there yet. We don’t have all that information for baseline THRIVE. We just completed enrollment, and so it’s going to take us a bit to just get all that together. So more to come on that one as well.

是的，還有另一個，加文。我的意思是，這是一個很好的問題。我非常感謝。只是我們還沒達到那個目標。我們沒有關於 THRIVE 基線的所有資訊。我們剛完成註冊，所以需要一些時間來整理所有資料。所以關於這一點，我們之後還會有更多消息。

Makes sense. Thank you.

有道理。謝謝。

[Rami Katkhuda] from LifeSci Capital

[Rami Katkhuda] 來自 LifeSci Capital

Hey guys, congrats on all the progress and thanks for taking my questions as well. You touched on the significant ex-U.S. market opportunity in TED. I guess, are you planning to file for approval of VRDN-001 in the U.S. and Europe in parallel once all the data is in hand? And how large of an opportunity could that ultimately represent?

各位，恭喜你們取得的所有進展，也感謝你們回答我的問題。您在TED大會上談到了美國以外市場的巨大機會。我猜，一旦所有數據都掌握在手，你們是不是打算同時在美國和歐洲申請VRDN-001的上市許可？這最終可能代表著多大的機會？

Yes. I think the epidemiology in Europe is very similar, so we know that to be the case, similar to the US, that is. With respect to our ex-US, plans, again, that’s something we’ll probably talk a bit more about later. We are as you can imagine, we are absolutely thinking about all that and the best approaches in these different geographies, even beyond Europe. So that’s all in the works. It will have more to say at a later time.

是的。我認為歐洲的流行病學情況非常相似，我們知道情況確實如此，與美國的情況類似。至於我們與美國以外的國家的計劃，我們以後可能會再詳細討論一下。正如您所想，我們正在認真考慮所有這些因素，以及在這些不同地區（甚至歐洲以外）的最佳方法。以上就是所有正在進行中的工作。稍後會有更多消息。

Got it. Sounds good.

知道了。聽起來不錯。

Derek Archila with Wells Fargo.

Derek Archila，來自富國銀行。

Hey, this is Adam on for Derek. Thanks for taking our questions today. I guess just a couple questions on the timeline really. So given THRIVE is still reading out like mid-2024-ish and THRIVE-2 reading out end of year. What factors are driving a second half 2025 BLA submission in TED? Is this related to the STRIVE study then? And in that sense, is an interim cut, would that be sufficient from STRIVE for a BLA submission or does the whole STRIVE trial need to be completed to support the BLA submission?

大家好，我是亞當，代德里克報道。感謝您今天回答我們的問題。我其實想問幾個關於時間安排的問題。所以，鑑於 THRIVE 預測結果預計在 2024 年年中左右發布，而 THRIVE-2 預測結果預計在年底發布。哪些因素促使人們在 TED 大會上提交 2025 年下半年的 BLA 報告？那麼這與 STRIVE 研究有關嗎？從這個意義上講，STRIVE 的中期試驗結果是否足以支持 BLA 申請，還是需要完成整個 STRIVE 試驗才能支持 BLA 申請？

Great. Thanks, Adam. Appreciate that question. Yeah, so what's driving the timeline is that remember So, we've talked about thrive to that a top-line readout at the end of the year. So, by definition, it’s not the completed study, right? So, we have to let that we have a follow-up period. There’s a total of the 52-week follow-up period, but only 37 weeks post the last dose.

偉大的。謝謝你，亞當。感謝您的提問。是的，所以推動這一時間表的因素是，記住，我們之前討論過，要在年底公佈一個總體數據。所以，根據定義，它還不是完整的研究，對吧？所以，我們需要留一段後續觀察期。總共有 52 週的追蹤期，但最後一次給藥後只有 37 週。

So there’s a follow-up period that is have to be taken into account and it’s primarily THRIVE-2 that’s driving the timeline. It really doesn’t. We’re not expecting STRIVE to have an impact there, that STRIVE should fit squarely within that timeline.

因此，後續階段必須考慮在內，而 THRIVE-2 主要是決定時間表的因素。並非如此。我們並不指望 STRIVE 會在那裡產生影響，STRIVE 應該完全符合該時間表。

And so, we feel that that’s probably the driver and we don’t need all of STRIVE. What you see there is that on the [ct.gov,] you see 212 patients that’s the maximum number that we’ll need.

因此，我們認為這可能是驅動因素，我們不需要 STRIVE 的全部內容。您可以看到，在 [ct.gov] 上，有 212 名患者，這是我們需要的最大人數。

We can do a data cut as soon as we reach the requisite number for the safety database. And there is a there are moving parts that go with that in terms of you can have dropout rates in your THRIVE and THRIVE-2.

一旦達到安全資料庫所需的資料量，我們就可以進行資料截斷。而且，THRIVE 和 THRIVE-2 中還存在一些變動因素，例如輟學率。

So, we kind of over engineer or over set up the STRIVE study, but we can do a data cut when we hit that threshold and again, all of this is really typical you have to have safety database that accompanies your BLA submission. So, it all kind of is normal blocking and tackling from our perspective.

所以，我們對 STRIVE 研究進行了過度設計或過度設置，但當達到該閾值時，我們可以進行資料截斷。而且，所有這些都非常典型，你必須有一個安全資料庫來配合你的 BLA 提交。所以，從我們的角度來看，這一切都只是正常的阻擋和擒抱而已。

But just got to take into account that THRIVE-2 is just a top line readout. So there’s more to do after a top line readout, which drives the second half 2025 finally.

但要注意的是，THRIVE-2 只是一個整體讀數。因此，在公佈整體數據之後還有更多工作要做，這將最終決定 2025 年下半年的走向。

Got you. That makes sense. And I guess, can you provide any numbers on what that threshold is and then just kind of following-up on the STRIVE study too? Is this the safety database you expect to leverage then with any potential regulatory path for VRDN-003? Thanks.

抓到你了。這很有道理。我想問一下，您能否提供一些關於該閾值的具體數據，以及STRIVE研究的後續進展？那麼，您打算利用這個安全資料庫來制定 VRDN-003 的任何潛在監管路徑嗎？謝謝。

Yeah, so 003, as we’ve talked about previously is a different molecular entity, so it will have its own path. So, the answer to that question is no. On the first question, the threshold is the threshold again, like I said, 212 is the max number. We’re not we’ll see where we end up.

是的，正如我們之前討論過的，003 是一個不同的分子實體，所以它將有自己的路徑。所以，這個問題的答案是否定的。關於第一個問題，閾值還是閾值，就像我說的，212 是最大值。我們不……我們走著瞧吧。

The total number is 300, but that includes THRIVE and THRIVE-2 active patients on [10 mg/kg]. So, we’re not anticipating needing the entire STRIVE study. And again, most importantly, STRIVE is not expected to drive the timeline for BLA submission. It’s more the THRIVE-2.

總數為 300 人，其中包括 THRIVE 和 THRIVE-2 的活躍患者。[10 毫克/公斤]。因此，我們預計不需要整個 STRIVE 研究。再次強調，最重要的是，STRIVE 預計不會影響 BLA 提交的時間表。它更像是 THRIVE-2。

It's more the drive to do that.

更重要的是做這件事的動力。

Thank you.

謝謝。

Your next question comes from the line of Gregory Renza with RBC.

你的下一個問題來自 Gregory Renza 和 RBC 的報道。

So, I'll turn it over to Tom in a second. But yes, I think if you could, you could see that we enrolled not only did we enroll thrive on time. We exceeded enrollments. We had really strong patient demand to drive that all within the month of it were all within the month of March.

好了，我馬上把麥克風交給湯姆。但是，是的，我想如果你能看到的話，你會發現我們不僅按時入學，而且還取得了成功。我們的招生人數超出了預期。我們有非常強勁的病患需求，推動了所有相關業務在三月的發展。

And I think that's a I think that should be a clear sign to the world that there are lots of there are lots of patients out there with TED that want to access IGF-1R therapy. So, that's a really good sign for US.

我認為這應該向世界發出一個明確的信號，那就是有很多患有 TED 的患者想要接受 IGF-1R 療法。所以，這對我們來說是個非常好的跡象。

Don't forget also that we had roughly half of the patients were enrolled in the US. I know that was a question mark for people. I think we've definitively answered that the US, is a very we've got the opportunity within the US, and then the other half in Europe where they have to be, as I mentioned, the epidemiology is the same. So I think that might be just a general answer.

也別忘了，我們大約有一半的患者是在美國招募的。我知道這對很多人來說是個問號。我認為我們已經明確回答了這個問題：美國的情況非常好，我們在美國境內擁有這樣的機會，而歐洲的另一半，正如我所提到的，他們的流行病學情況也是一樣的。所以我覺得這可能只是一個籠統的答案。

Tom, to do you want to talk about how competition for trials is shaking out?

湯姆，你想談談試鏡競爭的情況如何嗎？

Yeah, Sure. Yes. Thanks for your question, Gregory. I’m out in the field a lot talking to investigators and KOLs both in the US, and ex-US, and I can tell you there’s a lot of excitement about our portfolio as you know, our Phase 2 trial showed really promising results, and that’s driven a lot of interest, hence the over enrollment that Steve referenced.

好的，當然可以。是的。謝謝你的提問，格雷戈里。我經常外出與美國及其他地區的調查人員和關鍵意見領袖交流，我可以告訴你，大家對我們的產品組合感到非常興奮，正如你所知，我們的二期試驗顯示出了非常有希望的結果，這引起了很多人的興趣，因此才有了史蒂夫提到的超額招募。

Also, I can tell you that with respect to STRIVE, we have an active control. There is no placebo, and we think that’s going to drive enrollment there. So I think overall, just lots of positivity around our TED portfolio in our trials. I really can’t comment on competitors, but what I can say is just lots of excitement and enthusiasm around our portfolio.

另外，我可以告訴你們，關於 STRIVE，我們擁有積極的控制權。沒有安慰劑，我們認為這將推動那裡的報名人數。所以我覺得整體來說，我們在 TED 專案組合的測試中獲得了很多正面的回饋。我真的無法評論競爭對手，但我可以說的是，大家對我們的產品組合感到非常興奮和熱情。

Great. Thanks, guys.

偉大的。謝謝各位。

Julian Harrison with BTIG.

Julian Harrison 與 BTIG 合作。

Hi, good morning. Thank you for taking my question. I understand FcRn is kind of in the background this year, but I’m wondering if there’s maybe an IGF-1R to FcRn sequence and TED, that could be worthwhile to study in the future? Or are you mainly interested in FcRn opportunities beyond TED at this point?

您好，早安。感謝您回答我的問題。我知道今年 FcRn 的研究比較少，但我很好奇是否存在 IGF-1R 到 FcRn 的序列以及 TED，這在未來可能值得研究？或者，您目前主要對TED以外的FcRn機會感興趣嗎？

Yeah, it’s the latter, Julian. Yes, we’re we would we do so for TED patients, we firmly believe that IGF-1R is the key to that disease or the heart of that disease that’s where the cell signaling is taking place. You’ve got to hit that receptor in order to disrupt that. And so FcRns, the IL-6s, the other modalities are not or other mechanisms, we don’t feel are on target for moderate to severe TED patients. So for us, the IGF-1R is the key to TED. So FcRn will take that in different places, as we alluded to in the deck.

是的，是後者，朱利安。是的，我們之所以這樣做，是因為我們對 TED 患者堅信 IGF-1R 是疾病的關鍵或核心，細胞訊號傳導就發生在這裡。你必須擊中那個受體才能幹擾它。因此，FcRns、IL-6s 和其他療法或其他機制，我們認為對於中度至重度 TED 患者來說並非標靶。所以對我們來說，IGF-1R 是 TED 的關鍵。所以 FcRn 會在不同的地方發揮作用，正如我們在牌組中提到的那樣。

Thank you.

謝謝。

Trevor Allred from Oppenheimer.

來自奧本海默公司的特雷弗·奧爾雷德。

Hey guys, good morning. Thanks for taking the question. So with TEPEZZA sales trending down slightly, can you give us some perspective on why you think the new start market there appears to be somewhat stagnant and how you see this as a potential opportunity for you?

嘿，各位，早安。感謝您回答這個問題。鑑於 TEPEZZA 的銷售額略有下降，您能否談談您認為當地新創企業市場為何似乎有些停滯不前，以及您如何看待這其中的潛在機會？

Yeah, I mean, I think it’s hard for us to comment on Amgen sales. I think they are – they did report on their call last week. They did report year-over-year growth, which is the first time they’ve done that since the announcement of the merger. So we see that as a really good sign. Amgen was also really confident on their call that they believe that the market continues to be underpenetrated, which we agree with, and they believe that it’s going to continue to grow.

是的，我的意思是，我覺得我們很難對安進的銷售情況發表評論。我認為他們是……他們上週確實匯報了他們的通話情況。他們確實實現了同比增長，這是自宣布合併以來他們首次實現同比增長。所以我們認為這是一個非常好的跡象。安進在電話會議上也非常自信地表示，他們認為市場滲透率仍然很低，我們也同意這一點，他們相信市場將繼續成長。

Growth areas don’t forget, growth areas also include the other geographies, the introduction of [subcu]. And so they’ve now they’ve filed in Japan and Europe, which is good as they’re continuing to kind of blaze that trail for us. And don’t forget, even in the backdrop of all of this, they did close to $1.8 billion, or close to $2 billion in sales in 2023 in the backdrop of all of this as a first entrant.

成長區域別忘了，成長區域也包括其他地區，引進[亞囊]。所以他們現在已經在日本和歐洲提交了申請，這很好，因為他們正在繼續為我們開闢這條道路。別忘了，即使在所有這些背景下，作為第一個進入市場的公司，他們在 2023 年的銷售額也接近 18 億美元，或接近 20 億美元。

So, again, we feel that there’s plenty of room to run in TED, not only in the US., but elsewhere as well. And then we think subcu, particularly our subcu, which we think is potentially going to be best-in-class, where we can have patients that can access it just by delivery at home and they can self-administer at home.

所以，我們再次認為，TED 有很大的發展空間，不只在美國，在其他地方也是如此。然後我們認為皮下注射，特別是我們的皮下注射，我們認為它有可能成為同類最佳，患者可以透過送貨上門的方式獲得它，並且可以在家中自行注射。

We think that’s a game changer for TED patients, and I think the physician community agrees with us on that. So yes, we’re not particularly worried about IGF-1R being the right place for TED patients, and I think Amgen is going to prove that as well.

我們認為這對 TED 患者來說是一個顛覆性的改變，我認為醫生界也同意我們的觀點。所以，是的，我們並不特別擔心 IGF-1R 是否是 TED 患者的合適靶點，我認為安進公司也會證明這一點。

Okay. Great. That’s super helpful. And I guess, could you give us some perspective on when we might expect to see initial FcRn data with it – with the IND coming in the year, maybe second half to mid-year 2025?

好的。偉大的。這太有幫助了。那麼，您能否給我們一些關於何時能看到 FcRn 初步數據的看法呢？隨著 IND 在今年提交，或許在 2025 年下半年到年中？

Yeah, in our deck, you can see that we’ve got some healthy volunteer data that’s pegged to the second half of 2025 for that 006 program. It’s a little ways out. So we’ll look to see if we can pull that timeline in, but we feel, we’re on track for that [IND]. And then we’ll get the healthy volunteer study going. And so, we’ll get some data there.

是的，在我們的簡報中，您可以看到我們有一些健康的志工數據，這些數據與 2025 年下半年的 006 計畫相關。有點遠。所以我們會看看能否按時完成，但我們覺得，目前一切都在按計劃進行。[IND].然後我們就可以啟動健康志願者研究了。所以，我們會在那裡獲取一些數據。

I think don’t forget the 008 non-human primate data that has in other FcRns that has proven to be pretty translatable. So we’re pretty excited to see that. We saw great humanized mice data for 008, but obviously that’s mice data. We want to see the NHPs.

我認為不要忘記 008 非人靈長類動物數據，這些數據在其他 FcRn 中已被證明具有相當強的可轉化性。所以我們對此感到非常興奮。我們看到了 008 的優秀人源化小鼠數據，但顯然那是小鼠數據。我們想看看非人靈長類動物。

We’ll get that in the second half of this year. So we think that’s actually a relatively important thing for us to get. So we’re looking forward to FcRn moving forward. We got a lot to do there.

我們將在今年下半年實現這一目標。所以我們認為，這對我們來說其實是一件相對重要的事情。所以我們很期待FcRn接下來的發展。我們有很多事情要做。

Okay. Great. Thanks, Steve.

好的。偉大的。謝謝你，史蒂夫。

I'm sorry, at this time, we have reached the conclusion of the question-and-answer session. I would now like to turn the call back to Varian's President and CEO, Steve Mahoney, for closing remarks.

抱歉，問答環節到此結束。現在我想把電話轉回給瓦里安公司總裁兼執行長史蒂夫·馬奧尼，請他作總結發言。

Great. Thank you, operator, and thanks, everyone, for joining the call this morning and we've made a lot of progress, and we are executing. We're delivering on what we've what we've said, and that's important. We know that's important to use well. So thank you for your participation today, and we look forward to talking to you in the future.

偉大的。謝謝接線員，也謝謝大家今天早上參加電話會議，我們取得了很大進展，並且正在執行中。我們說到做到，這很重要。我們知道好好利用這一點很重要。感謝您今天的參與，我們期待未來與您再次交流。

This concludes today's conference call. You may disconnect your lines. Thank you for participating.

今天的電話會議到此結束。您可以斷開線路。感謝您的參與。