Scholar Rock Holding Corporation (SRRK) 2026 Q2 法說會逐字稿

Good morning. I'm Laura Ekas, Vice President of Investor Relations at Scholar Rock. With me today are David Hallal, Board Chair and Chief Executive Officer; Akshay Vaishnaw, President of R&D; Keith Woods, Chief Operating Officer; and Vikas Sinha, Chief Financial Officer.

早安。我是 Scholar Rock 投資人關係副總裁 Laura Ekas。今天與我一同出席的有董事會主席兼執行長 David Hallal；研發總裁 Akshay Vaishnaw；營運長 Keith Woods；以及財務長 Vikas Sinha。

During today's call, David will provide introductory remarks and a business update. Akshay will review our R&D progress. Keith will provide an update on our commercial readiness activities, and ViKas will provide a financial update. We will then open the call for questions.

在今天的電話會議中，David 將提供開場致詞與業務更新。Akshay 將回顧我們的研發進展。Keith 將就我們的商業化就緒活動提供最新進度，而 Vikas 將提供財務更新。之後我們將開放提問。

Before we begin, I'd like to remind you that during this call, we will be making various statements about Scholar Rock's expectations, plans, and prospects that constitute forward-looking statements for the purposes of safe harbor provisions under the Private Securities Litigation Reform Act of 1995.

在開始之前，我想提醒各位，在本次電話會議中，我們將就 Scholar Rock 的預期、計畫與前景發表各項陳述，這些陳述構成《1995 年私人證券訴訟改革法》安全港條款所定義的前瞻性陳述。

Any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any future date. I encourage you to go to the Investors and Media section of our website for our most up-to-date SEC statements and filings.

任何前瞻性陳述僅代表我們截至今日的觀點，不應被依賴為代表我們在任何未來日期的觀點。我鼓勵各位前往我們網站的「投資人與媒體」專區，查閱我們最新的 SEC 聲明與申報文件。

With that, I'd like to turn the call over to David. David?

接下來，我想把電話交給 David。David？

Thank you, Laura, and good morning. Thanks to everyone for joining our second-quarter earnings call. Today, Scholar Rock is operating from a position of strength as we enter a defining period for our company and for the SMA community.

謝謝你，Laura，早安。感謝各位參加我們第二季財報電話會議。今天，Scholar Rock 正以穩健的優勢邁入公司與 SMA 社群的關鍵時期。

Across the business, we are executing with focus, discipline and urgency as we drive toward the US launch of Apitegromab this quarter, advance our Apitegromab MAA in Europe to approval, and prepare for launch in Germany, build momentum across our world-leading anti-myostatin platform, and maintain the financial strength to support our ambitions.

在整體業務上，我們以專注、紀律與緊迫感執行各項工作，推進 Apitegromab 本季在美國上市、推動 Apitegromab 在歐洲的 MAA 取得核准並為德國上市做準備、在我們世界領先的抗肌生成抑制素（anti-myostatin）平台上持續累積動能，並維持財務實力以支持我們的抱負。

Most importantly, we are on the threshold of bringing the world's first muscle-targeted therapy to children and adults living with SMA as we advance Apitegromab through the final stages of the FDA regulatory process.

最重要的是，隨著我們推進 Apitegromab 進入 FDA 法規審查流程的最後階段，我們正站在把全球第一個以肌肉為標的的療法帶給 SMA 兒童與成人患者的門檻上。

Let me now provide additional detail on the ongoing review of our Apitegromab BLA in the US. As a reminder, the sole approvability issue for Apitegromab noted in the complete response letter that we received on our priority review PDUFA date last September was related to observations identified at a routine general site inspection of the Catalent Indiana fill-finish facility owned and operated by Novo Nordisk.

接下來我將就我們在美國 Apitegromab BLA 的持續審查提供更多細節。提醒各位，我們在去年 9 月優先審查 PDUFA 日期收到的完整回覆函（CRL）中，針對 Apitegromab 的唯一可核准性（approvability）問題，與 Novo Nordisk 所擁有並營運的 Catalent Indiana 充填與包裝（fill-finish）廠在例行一般性場址稽查中所發現的觀察事項相關。

Since our constructive and collaborative Type A meeting in November, the cadence of activities has reflected the shared understanding between us and the agency of the high unmet need in the SMA community and a shared sense of urgency to bring Apitegromab to children and adults with SMA as rapidly as possible.

自我們在 11 月舉行具建設性且合作的 Type A 會議以來，各項活動的節奏反映出我們與主管機關對 SMA 社群高度未被滿足需求的共同理解，以及共同的緊迫感：盡可能快速地將 Apitegromab 帶給 SMA 的兒童與成人。

We are grateful for the agency's sustained level of engagement and for our ongoing dialogue, including our March 3 Type C meeting where we discussed the accelerated progress that we had made at our second fill-finish facility, and the agreed upon data package to facilitate the FDA review of the second fill-finish facility.

我們感謝主管機關持續投入的參與程度，以及我們持續的對話，包括 3 月 3 日的 Type C 會議；在該會議中，我們討論了第二座充填與包裝廠所取得的加速進展，以及為促進 FDA 審查第二座充填與包裝廠而一致同意的資料套件。

In alignment with FDA guidance from this discussion, we submitted the Apitegromab BLA on March 30 with two fill-finish facilities, both Catalent Indiana and our second fill-finish facility. Our BLA was accepted in April with a PDUFA date of September 30. Agency review of our application is progressing well.

依據該次討論所提供的 FDA 指引，我們於 3 月 30 日提交 Apitegromab BLA，納入兩座充填與包裝廠：Catalent Indiana 以及我們的第二座充填與包裝廠。我們的 BLA 於 4 月獲受理，PDUFA 日期為 9 月 30 日。主管機關對我們申請案的審查進展順利。

Importantly, we have significant optionality with two independent paths to approval, either through Catalent Indiana or through our second fill-finish facility or both, whichever is determined to be the most rapid. As it relates to Catalent Indiana, the FDA inspection classification following the April 2026 general site inspection is pending. We continue to be very pleased by the progress made at our second fill-finish facility.

重要的是，我們擁有顯著的彈性，具備兩條彼此獨立的核准路徑：可透過 Catalent Indiana、或透過我們的第二座充填與包裝廠、或兩者同時進行，以被認定為最快速的方式為準。就 Catalent Indiana 而言，FDA 在 2026 年 4 月一般性場址稽查後的稽查分類仍待定。我們對第二座充填與包裝廠所取得的進展仍感到非常滿意。

Importantly, the data package for the FDA's review of the second facility has been submitted and the review is progressing. Underscoring our operational excellence at our second site, we now have more vials available from this facility than we do from Catalent Indiana. Notably, vials from both Catalent Indiana and our second facility are now on site at our third-party provider awaiting packaging and labeling upon approval.

同樣重要的是，供 FDA 審查第二座廠的資料套件已提交，審查正在進行中。凸顯我們在第二座場址的卓越營運表現，我們目前由該廠可供使用的藥瓶數量已多於 Catalent Indiana。值得注意的是，來自 Catalent Indiana 與第二座廠的藥瓶目前都已送達我們的第三方供應商場址，待核准後即可進行包裝與貼標。

As we near the final step in the US regulatory process, we are well aware that patients are awaiting the world's first muscle-targeted therapy for this devastating disease.

在我們接近美國法規流程的最後一步之際，我們深知患者正殷切等待這項針對此一毀滅性疾病的全球首個以肌肉為標的的療法。

Turning now to our commercial readiness in the US, our team continues to advance our launch preparations across all key functions, ensuring we are prepared to support patients, caregivers, and prescribers from day one. Our team is ready to launch Apitegromab at any time prior to and including our September 30 PDUFA date. Keith will discuss our commercial preparations in greater detail shortly.

接著談到我們在美國的商業化就緒情況，我們的團隊持續在所有關鍵職能上推進上市準備，確保從第一天起就能支援患者、照護者與處方醫師。在 9 月 30 日 PDUFA 日期之前（含當日）的任何時間，我們的團隊都已準備好推出 Apitegromab。稍後 Keith 將更詳細說明我們的商業化準備工作。

In addition to the US, we continue to look forward to serving children and adults living with SMA in Europe. I would now like to provide an update on where we stand in the European regulatory process. The Apitegromab MAA includes only Catalent Indiana fill-finish facility, and the EMA is awaiting the FDA inspection classification for that facility.

除美國之外，我們也持續期待能在歐洲服務 SMA 的兒童與成人患者。我現在想就我們在歐洲法規流程的進展提供更新。Apitegromab 的 MAA 目前僅包含 Catalent Indiana 充填與包裝廠，而 EMA 正在等待該廠的 FDA 稽查分類結果。

In parallel, we are engaging with European regulators with regards to potential inclusion of our second fill-finish facility in the Apitegromab application. Importantly, this facility has had recent successful site inspections by the FDA and the EMA. We are grateful for the EMA's continued level of engagement, and we look forward to providing updated guidance on the potential timing of a CHMP opinion upon alignment with the European regulators.

同時，我們也正就可能將第二座充填與包裝廠納入 Apitegromab 申請案一事，與歐洲主管機關進行溝通。重要的是，該廠近期已成功通過 FDA 與 EMA 的場址稽查。我們感謝 EMA 持續投入的參與程度，並期待在與歐洲主管機關達成一致後，就 CHMP 意見可能的時程提供更新指引。

Turning to our launch preparations in Europe, we are executing our commercial playbook, building momentum with launch readiness activities and engaging with the SMA community. We are planning for an initial launch in Germany with additional countries and regions to follow as we build out our planned 50-country operating platform.

談到我們在歐洲的上市準備，我們正執行商業化作戰手冊，透過上市就緒活動建立動能，並與 SMA 社群互動。我們規劃先在德國進行初始上市，並在我們建置預計涵蓋 50 個國家的營運平台過程中，陸續擴展至其他國家與地區。

We know it is not a matter of if, but when Apitegromab will be approved for children and adults with SMA in both the US and Europe, and we continue to work with urgency to reach the 35,000 people with SMA around the world who have received an SMN-targeted therapy.

我們知道問題不在於 Apitegromab 是否會在美國與歐洲獲准用於 SMA 兒童與成人，而在於何時獲准；我們也持續以緊迫感推進工作，以觸及全球約 35,000 名已接受 SMN 標的治療的 SMA 患者。

Turning now to our world-leading anti-myostatin pipeline. We continue to make meaningful progress with our key clinical programs. We have robust enrollment in our Phase 2 OPAL study evaluating Apitegromab in infants and toddlers with SMA. We initiated our randomized Phase 2 FORGE study in patients with FSHD.

接著談到我們世界領先的抗肌生成抑制素（anti-myostatin）產品線。我們在關鍵臨床計畫上持續取得具意義的進展。我們在第 2 期 OPAL 研究中（評估 Apitegromab 用於 SMA 嬰幼兒）招募情況強勁。我們已在 FSHD 患者中啟動隨機分派的第 2 期 FORGE 研究。

We are ready to engage with US and European regulators on the development path of our high concentration subcutaneous formulation of Apitegromab, which we will do once we have regulatory approvals. And enrollment and dosing is proceeding very well in our Phase 1 healthy volunteer study for SRK-439, our novel high-potency anti-myostatin antibody. Akshay will discuss these programs in greater detail shortly.

在取得法規核准後，我們已準備好就 Apitegromab 高濃度皮下注射劑型的開發路徑，與美國及歐洲主管機關展開交流。此外，我們針對 SRK-439（我們新型高效力抗肌生成抑制素抗體）的第 1 期健康受試者研究，招募與給藥進展非常順利。稍後 Akshay 將更詳細討論這些計畫。

Turning now to the balance sheet, we were very pleased to have ended the second quarter of 2026 with $492 million in cash, cash equivalents, and marketable securities. This cash balance includes net proceeds of $63 million from our ATM program during the second quarter. Vikas will provide more details later in the call.

接著談到資產負債表，我們很高興在 2026 年第二季末擁有 4.92 億美元的現金、約當現金與有價證券。此現金餘額包含我們在第二季透過 ATM 計畫取得的 6,300 萬美元淨收益。Vikas 將在稍後的電話會議中提供更多細節。

In June, we had the opportunity to be with the SMA physician and patient community at the Cure SMA Annual Meeting in Orlando. I was able to sit down with several SMA treating physicians and with a number of patients and their families. And during our time with them, we heard some very moving stories about the impact Apitegromab has had on children and adults who are participating in our Onyx and EAP programs.

6月，我們有機會在奧蘭多舉行的 Cure SMA 年會上，與 SMA 醫師與病友社群相聚。我得以與數位治療 SMA 的醫師，以及多位病友與其家屬坐下來交流。在與他們相處的過程中，我們聽到一些非常動人的故事，談到 Apitegromab 對參與我們 Onyx 與 EAP 計畫的兒童與成人所帶來的影響。

Our team at Scholar Rock is so inspired by these patients and by their families, and we look forward to ushering in the next phase of innovation for this community.

Scholar Rock 的團隊深受這些病友及其家屬的啟發，我們也期待為這個社群開啟下一階段的創新。

With that, I'll now turn the call over to Akshay for a closer look at our R&D initiatives. Akshay.

接下來，我將把電話會議交給 Akshay，請他更深入介紹我們的研發（R&D）計畫。Akshay。

Thank you, David, and good morning, everybody. As David shared, we're very pleased that the Apitegromab BLA continues to progress through FDA review with two independent paths to approval, and we remain on track for a decision by the September 30 PDUFA date.

謝謝你，David，各位早安。如 David 所分享，我們很高興 Apitegromab 的 BLA 仍持續在 FDA 審查流程中推進，且有兩條彼此獨立的核准途徑；我們仍按計畫在 9 月 30 日的 PDUFA 目標日期前等待決定。

The FDA inspection classification for Catalent Indiana is pending. We had anticipated the classification in late July within 90 days following inspection completion based on the agency's guidelines. We remain engaged with the FDA and will provide updates as appropriate.

Catalent Indiana 的 FDA 查廠分類結果仍待定。依據主管機關指引，我們原先預期在查廠完成後 90 天內、約於 7 月下旬取得分類結果。我們仍與 FDA 保持密切互動，並將在適當時機提供最新進展。

As it relates to the second fill-finish facility, we're very pleased to report that all necessary data have now been submitted to FDA and the agency's review of those data is progressing well. We're gratified by the agency's continued support since the CRL last September from the constructive and collaborative in-person Type A meeting in November to the early March Type C meeting.

至於第二個充填與包裝（fill-finish）廠，我們很高興報告所有必要資料現已提交 FDA，且主管機關對這些資料的審查進展良好。自去年 9 月收到 CRL 以來，主管機關持續給予支持，從 11 月具建設性且協作的實體 Type A 會議，到 3 月上旬的 Type C 會議，我們對此深感欣慰。

Throughout, the agency has appreciated the high met need in the SMA community, and we look forward to the final steps in the process.

在整個過程中，主管機關理解 SMA 社群高度未被滿足的醫療需求，我們也期待流程中的最後幾個步驟。

Turning now to Europe, we're pleased with the EMA's review of the Apitegromab marketing authorization application and with their continued level of engagement. As we have previously noted, approval in Europe is dependent on FDA clearance of the Catalent Indiana facility, which is currently the sole fill-finish site included in our MAA.

接著談到歐洲，我們對 EMA 對 Apitegromab 上市許可申請（MAA）的審查，以及其持續投入的互動程度感到滿意。如先前所述，歐洲的核准取決於 FDA 對 Catalent Indiana 廠的放行；該廠目前是我們 MAA 中唯一納入的充填與包裝據點。

The EMA continues to await the FDA's inspection classification for this facility. Additionally, we're engaging with the EMA regarding the process to include our second fill-finish facility in our Apitegromab application. Importantly, this facility is in good standing with European regulators. We will provide an update on timing upon alignment with the EMA.

EMA 仍在等待 FDA 對該廠的查廠分類結果。此外，我們也正與 EMA 討論將第二個充填與包裝廠納入 Apitegromab 申請案的流程。重要的是，該廠在歐洲監管機關方面的合規狀態良好。待與 EMA 就時程達成一致後，我們將提供時間點更新。

Turning to our pipeline, let me start with the Phase 2 OPAL trial. We continue to have robust enrollment in the study, which is evaluating Apitegromab in infants and toddlers with SMA under the age of two. As a reminder, this trial is enrolling participants who have been treated with an SMN1-targeted gene therapy or who are receiving ongoing treatment with an SMN-2 targeted treatment.

接著談我們的產品線（pipeline），先從第 2 期 OPAL 試驗開始。該研究持續維持強勁的收案，評估 Apitegromab 用於 2 歲以下 SMA 嬰幼兒。提醒各位，本試驗收納的受試者包括已接受 SMN1 標的基因治療者，或正在接受 SMN-2 標的治療的持續治療者。

This study is important because it is anticipated to expand the impact of Apitegromab to the full spectrum of patients, including those treated with Zolgensma. Notably, the rate at which the study is enrolling underscores the significant unmet need and the potential for Apitegromab in the youngest of SMA patients.

這項研究很重要，因為預期可將 Apitegromab 的影響擴展至完整的病患光譜，包括接受 Zolgensma 治療者。值得注意的是，研究的收案速度凸顯了顯著的未被滿足需求，以及 Apitegromab 在最年幼 SMA 病患中的潛力。

Setting now to our next indication for Apitegromab, Facioscapulohumeral Muscular Dystrophy, or FSHD. FSHD is a rare devastating neuromuscular disease. It is one of the most prevalent inherited muscular dystrophies, and there are no approved therapies to date. We've prioritized FSHD as the next indication for Apitegromab for three key reasons.

現在轉到 Apitegromab 的下一個適應症：顏面肩胛肱型肌肉失養症（Facioscapulohumeral Muscular Dystrophy，FSHD）。FSHD 是一種罕見且具毀滅性的神經肌肉疾病。它是最常見的遺傳性肌肉失養症之一，且迄今尚無核准療法。我們將 FSHD 列為 Apitegromab 的下一個適應症，主要基於三個關鍵原因。

First, the significant unmet need since approximately 20% of patients become wheelchair dependent. Second, the compelling preclinical data from the gold-standard FLExDUX4 mouse model that provides mechanistic rationale for Apitegromab in FSHD. and finally, data from randomized studies in FSHD which suggest muscle mass can increase and has the capacity to show functional benefit.

第一，未被滿足的需求顯著，約有 20% 的病患最終需依賴輪椅。第二，來自黃金標準 FLExDUX4 小鼠模型的具說服力臨床前數據，為 Apitegromab 用於 FSHD 提供機轉上的合理性。以及最後，FSHD 的隨機研究數據顯示肌肉量可增加，且有能力呈現功能性獲益。

For example, in studies of either rigorous physical therapy or treatment with anabolic agents, patients with FSHD demonstrated increases in lean mass and muscle function. These data suggest that Apitegromab as a monotherapy may have the potential to bring important benefit to FSHD patients.

例如，在嚴格的物理治療或使用合成代謝藥物的研究中，FSHD 病患展現了瘦體重與肌肉功能的提升。這些數據顯示，Apitegromab 作為單一療法可能有潛力為 FSHD 病患帶來重要獲益。

We're very pleased to announce today that we've initiated our Phase 2 study called FORGE, which is a randomized, double-blind, placebo-controlled trial with a sample size of 60 patients. We're also advancing two additional therapeutic programs in our world-leading anti-myostatin pipeline, a high-concentration subcutaneous formulation of Apitegromab and SRK-439.

我們很高興在今天宣布，我們已啟動名為 FORGE 的第 2 期研究；這是一項隨機、雙盲、安慰劑對照試驗，樣本數為 60 名病患。我們也正推進在全球領先的抗肌生成抑制素（anti-myostatin）產品線中的另外兩個治療計畫：Apitegromab 的高濃度皮下劑型，以及 SRK-439。

In our subcutaneous Apitegromab program, we showed some very exciting data in January from a Phase 1 study which demonstrated that subcutaneous Apitegromab appears to have favorable bioavailability and the pharmacodynamic profile comparable to IV administration. Additional development activities are ongoing, and we continue to plan for engagement with US and European regulators later this year following approval of Apitegromab.

在 Apitegromab 皮下給藥計畫方面，我們於 1 月公布了第 1 期研究的一些令人振奮的數據，顯示皮下 Apitegromab 似乎具有良好的生體可用率，且其藥效動力學（pharmacodynamic）特徵與靜脈注射（IV）給藥相當。其他開發工作仍在進行中；在 Apitegromab 獲准後，我們仍計畫於今年稍晚與美國及歐洲監管機關進行互動。

Turning now to SRK-439, our high-potency, high-affinity subcutaneously administered myostatin inhibitor. We're very excited about this program, and dosing in our phase one healthy volunteer study is progressing well. We expect to have top-line data from the study later this year.

接著談 SRK-439，這是我們高效價、高親和力、以皮下方式給藥的肌生成抑制素抑制劑。我們對此計畫感到非常振奮，目前在第 1 期健康受試者研究中的給藥進展順利。我們預期將於今年稍晚取得該研究的主要結果（top-line data）。

In closing, we're executing with urgency to bring Apitegromab to children and adults with SMA, whilst in parallel working to maximize our efforts. Maximize our impact for patients with our world-leading anti-myostatin pipeline across a range of rare, devastating neuromuscular diseases.

最後總結，我們正以高度急迫性推進，將 Apitegromab 帶給 SMA 的兒童與成人，同時也並行努力以最大化我們的投入。透過我們全球領先的抗肌生成抑制素產品線，在多種罕見且具毀滅性的神經肌肉疾病中，最大化我們對病患的影響。

I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith?

接下來我將把電話會議交給 Keith，請他說明我們的商業上市準備。Keith？

Thanks, Akshay, and good morning, everyone. As David noted, with the potential FDA approval of Apitegromab for children and adults with SMA by September 30, our US commercial organization is launch-ready across all key functions, and we are prepared to support patients, caregivers, and prescribers from day one.

謝謝你，Akshay，各位早安。如 David 所提到，若 Apitegromab 於 9 月 30 日前有望獲 FDA 核准用於 SMA 兒童與成人，我們的美國商業團隊已在所有關鍵職能上完成上市準備，並已準備好自第一天起支持病患、照護者與開立處方的醫師。

Given the significant unmet need in SMA, we have moved with urgency to build our commercial operations to ensure that patients who can benefit from Apitegromab will have broad and reliable access to Apitegromab. In the US, despite approximately 78% of children and adults living with SMA receiving an SMN-targeted therapy, 95% of patients continue to experience persistent and progressive muscle atrophy that limits both function and independence.

鑑於 SMA 顯著的未被滿足需求，我們以高度急迫性建立商業營運，以確保可從 Apitegromab 受益的病患能廣泛且穩定地取得 Apitegromab。在美國，儘管約 78% 的 SMA 兒童與成人正在接受 SMN 標的治療，仍有 95% 的病患持續出現持久且進行性的肌肉萎縮，限制其功能與獨立性。

As further evidence of the unmet medical need, data shared with us by Cure SMA show that an estimated one-third of people living with SMA in the US have received two or more SMN-targeted treatments, either sequentially or in combination. This data again underscores the significant opportunity we have with Apitegromab, the world's first muscle-targeted therapy for children and adults with SMA.

作為未被滿足醫療需求的進一步證據，Cure SMA 與我們分享的數據顯示，估計美國約三分之一的 SMA 病友曾接受兩種或以上的 SMN 標的治療，可能是序貫使用或合併使用。這些數據再次凸顯 Apitegromab 的重大機會：這是全球首個以肌肉為標的、用於 SMA 兒童與成人的治療。

Since our last earnings call, our US field team continues to broaden their reach, focusing on disease education and awareness around the unmet medical need while also reinforcing a broader understanding of SMA as a disease that consists of both the motor neuron and the muscle, the principal organ impacted by the disease.

自上次財報電話會議以來，我們的美國外勤團隊持續擴大觸及範圍，聚焦於疾病教育與對未被滿足醫療需求的認知提升，同時也強化對 SMA 的更全面理解：這是一種同時涉及運動神經元與肌肉（亦為主要受影響器官）的疾病。

We are also expanding our reach and frequency across approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams. Through these engagements, our field team is establishing case flows on a center-by-center basis to ensure that, upon approval, we are well positioned to support the SMA treatment centers once Apitegromab treatment decision has been made.

我們也正在擴大在約 140 家 SMA 治療中心、2,600 位開立處方的醫師及其多專科照護團隊中的觸及範圍與互動頻率。透過這些互動，我們的外勤團隊正以「逐中心」的方式建立個案流量，以確保在獲得核准後，一旦做出 Apitegromab 的治療決策，我們能處於有利位置，支援各 SMA 治療中心。

This past quarter, we have also strengthened our Scholar Rock Supports patient services program. The Scholar Rock Supports team is fully trained and prepared to provide comprehensive, individualized support to patients and caregivers at launch. Eligible patients and their families will be able to access this program to understand insurance coverage, identify available financial and co-pay assistance, and navigate treatment logistics.

在上一季，我們也強化了 Scholar Rock Supports 病患服務計畫。Scholar Rock Supports 團隊已完成全面訓練，並已準備在上市時為病患與照護者提供完整、個人化的支援。符合資格的病患及其家屬將可使用此計畫，以了解保險給付範圍、找出可用的財務與自付額（co-pay）補助，並協助處理治療相關的流程與後勤安排。

Turning now to patient engagement. Our connections with the SMA community remain strong. This past June, we had a significant presence at the Cure SMA Annual Meeting in Orlando. Scholar Rock served as a presenting sponsor of the meeting, and throughout the week, our teams engaged with healthcare professionals and members of the SMA patient community.

接下來談病患互動。我們與 SMA 社群的連結依然緊密。今年 6 月，我們在奧蘭多舉行的 Cure SMA 年會上有相當顯著的參與。Scholar Rock 擔任本次會議的發表贊助商（presenting sponsor），並在整週期間，我們的團隊與醫療專業人士及 SMA 病患社群成員進行交流。

I was very pleased that the Scholar Rock Symposium for Healthcare Professionals entitled Expert Perspective on the Evolving Management of Spinal Muscular Atrophy was one of the most attended expert sessions during the meeting. Equally, our patient symposium, MUSCLE: There's More to the Story in SMA, was attended by hundreds of SMA patients, caregivers, and families, and during this session, we sought their perspective on needs and priorities for people living with SMA.

我非常高興，面向醫療專業人士的 Scholar Rock 專題研討會「Expert Perspective on the Evolving Management of Spinal Muscular Atrophy（脊髓性肌肉萎縮症管理演進的專家觀點）」是會議期間出席人數最多的專家場次之一。同樣地，我們的病患研討會「MUSCLE: There's More to the Story in SMA（肌肉：SMA 的故事不只如此）」也吸引了數百位 SMA 病患、照護者與家屬參與；在該場次中，我們徵詢他們對 SMA 患者需求與優先事項的看法。

Every interaction we had during this meeting reinforces our determination and further strengthens our commitment to serve patients and families.

我們在此次會議中的每一次互動，都更加堅定我們的決心，並進一步強化我們服務病患與家庭的承諾。

Turning to US reimbursement, our market access team continues to advance discussions with national and key regional payers, as well as Medicare and Medicaid, with the goal of achieving broad reimbursement for eligible patients after approval.

談到美國給付（reimbursement），我們的市場准入團隊持續推進與全國性及主要區域性保險支付方（payers），以及 Medicare 與 Medicaid 的討論，目標是在核准後為符合資格的病患取得廣泛的給付。

Given the significant scope of our efforts and progress we've made in the past several months, we are ready and well-positioned to successfully support Apitegromab in the US immediately upon FDA approval.

鑑於我們在過去數月投入的工作規模與所取得的進展，我們已準備就緒，並處於有利位置，可在 FDA 核准後立即在美國成功支援 Apitegromab。

Turning now to Europe, we are advancing our launch preparations with a particular focus on Germany as we work with the EMA on the next steps for our application. Our team in Germany is using this additional time to execute the same launch readiness playbook that we have successfully deployed in the US over the last several months. This includes broadening and deepening of relationships with key SMA treatment centers and potential prescribers.

接著談歐洲，我們正推進上市準備，並特別聚焦德國，同時與 EMA 就我們申請案的後續步驟合作。我們在德國的團隊正利用這段額外時間，執行與我們過去數月在美國成功部署的相同上市就緒作戰手冊（launch readiness playbook）。其中包括擴大並深化與關鍵 SMA 治療中心及潛在處方醫師的關係。

In parallel, we are engaging with SMA advocates across Europe, participating in educational programs at various congresses and symposia hosted by patient advocacy organizations. As it relates to reimbursement and patient access, following European Commission approval of Apitegromab, we will be prepared to rapidly advance reimbursement submissions in Germany and other key markets.

同時，我們也與歐洲各地的 SMA 倡議者互動，參與由病患倡議組織主辦、在各類學術大會與研討會中的教育計畫。在給付與病患可近性方面，於歐盟執委會核准 Apitegromab 後，我們將準備迅速在德國及其他關鍵市場推進給付申請。

In addition, we are advancing our distributor relationships to extend the commercial reach of Apitegromab across multiple additional countries.

此外，我們也在推進與經銷商的合作關係，以將 Apitegromab 的商業觸及範圍延伸至更多其他國家。

In closing, we are fully prepared for a successful US launch immediately upon FDA approval, while advancing our launch preparations in Europe and working to establish our 50-country operating platform with the ambition of reaching the estimated 35,000 patients living with SMA worldwide who have received an SMN-targeted therapy.

最後，我們已完全準備就緒，可在 FDA 核准後立即於美國成功上市；同時也在推進歐洲的上市準備，並致力建立我們覆蓋 50 個國家的營運平台，目標是觸及全球估計約 35,000 名已接受 SMN 標靶治療、且罹患 SMA 的病患。

We are ready to usher in the next phase of innovation for children and adults with SMA, one patient, one caregiver, and one family at a time.

我們已準備好為 SMA 兒童與成人開啟下一階段的創新——一次幫助一位病患、一位照護者、一個家庭。

With that, I'll turn the call over to Vikas for a review of our financial performance. Vikas?

接下來，我把電話交給 Vikas，請他回顧我們的財務表現。Vikas？

Thank you, Keith. As we have shared previously, our financial objectives for 2026 remain focused on. Supporting our commercial build to deliver a strong Apitegromab launch, funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle space and continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value.

謝謝你，Keith。如同我們先前分享的，我們 2026 年的財務目標仍聚焦於：支持我們的商業化建置，以推動強勁的 Apitegromab 上市；資助研發活動以推進我們的產品線並擴大我們在肌生成抑制素（myostatin）與肌肉領域的領導地位；以及持續評估能強化資產負債表、並支持長期股東價值的機會。

Consistent with these priorities, I'd like to briefly review our second quarter financial results. For the second quarter, we reported $108.9 million in operating expenses, which included $19.7 million in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were $89.2 million.

依循這些優先事項，我想簡要回顧我們第二季的財務結果。第二季我們的營業費用為 1.089 億美元，其中包含 1,970 萬美元的非現金股票基礎薪酬。扣除股票基礎薪酬後，營業費用為 8,920 萬美元。

As we continue preparing for the anticipated launch of Apitegromab, we have strategically increased our commercial investments while keeping our overall operating expenses at the levels generally consistent with the second quarter of 2025. This disciplined approach to capital allocation has enabled us to advance launch readiness while continuing to invest in our key R&D programs and strengthening our global supply chain.

隨著我們持續為預期的 Apitegromab 上市做準備，我們在維持整體營業費用大致與 2025 年第二季一致的同時，策略性地提高了商業化投資。這種嚴謹的資本配置方式，使我們得以推進上市就緒工作，同時持續投資於關鍵研發計畫並強化我們的全球供應鏈。

Turning to our balance sheet, we ended the second quarter with $492 million in cash equivalents, and marketable securities. During the quarter, we further strengthened our cash position with $63 million in net proceeds from our ATM program.

談到資產負債表，我們在第二季末的現金及約當現金與有價證券為 4.92 億美元。本季期間，我們也透過 ATM 計畫取得 6,300 萬美元的淨募資款，進一步強化了現金部位。

Looking ahead, our FDA approval of Apitegromab, we will have an option to draw down an additional $150 million from our existing debt facility, and we plan to monetize a priority review voucher to further strengthen our balance sheet.

展望未來，在 Apitegromab 獲 FDA 核准後，我們將可選擇自現有債務融資額度再提取 1.5 億美元，並計畫將一張優先審查憑證（priority review voucher）變現，以進一步強化資產負債表。

We continue to operate with a disciplined financial plan, and our investment priorities remain focused on our Apitegromab commercial launch readiness in the US and Europe, strengthening our supply chain to support our expanding pipeline and anticipated global commercial demand for Apitegromab over time, and advancing our highly innovative clinical programs that Akshay discussed earlier in the call.

我們持續以嚴謹的財務計畫營運，而我們的投資優先順序仍聚焦於：在美國與歐洲的 Apitegromab 商業上市就緒、強化供應鏈以支援我們擴展中的產品線及未來 Apitegromab 預期的全球商業需求，以及推進 Akshay 先前在電話會議中提到的高度創新臨床計畫。

With that, I'll turn the call back to David. David?

接下來，我把電話交回給 David。David？

Thanks, Vikas. As we look ahead, Scholar Rock is entering one of the most important chapters in our history from a position of strength. With 55 days until our PDUFA date, we have the team, the financial foundation and the operational readiness to execute with confidence. Across the organization, our teams are prepared, energized and focused on what comes next: bringing forward the world's first muscle-targeted therapy for children and adults with SMA.

謝謝你，Vikas。展望未來，Scholar Rock 正以強勁的態勢邁入公司歷史上最重要的篇章之一。距離我們的 PDUFA 日期還有 55 天，我們具備團隊、財務基礎與營運就緒度，能夠自信地執行。在整個組織中，我們的團隊已準備就緒、充滿動能並專注於下一步：推動全球首個以肌肉為標的、用於 SMA 兒童與成人的治療。

We are moving into these final 55 days with urgency, discipline and a deep sense of responsibility to the SMA community. We know what is at stake, we know what is possible, and we are ready to deliver. As we close, we are mindful that August is SMA Awareness Month. This is an important opportunity to recognize the strength and resilience of the individuals living with SMA, their families, and the advocacy organizations that work tirelessly on their behalf.

在這最後的 55 天裡，我們將以緊迫感、紀律與對 SMA 社群深切的責任感向前推進。我們知道利害關係所在，我們知道可能達成的成果，而我們已準備好交付。在結語之前，我們也留意到 8 月是 SMA 認知月（SMA Awareness Month）。這是一個重要的機會，讓我們向 SMA 患者、其家人，以及不懈為他們努力的倡議組織所展現的力量與韌性致敬。

We are grateful to the patients, caregivers, healthcare professionals, and advocates, whose partnership continues to advance awareness, earlier diagnosis, and access to care. This month reinforces our commitment to the SMA community and our focus on delivering meaningful innovation that can make a lasting difference for people living with this rare and devastating disease. We look forward to updating you on our continued progress.

我們感謝病患、照護者、醫療專業人士與倡議者的支持與合作，這樣的夥伴關係持續推動認知提升、更早診斷與照護可近性。本月也再次強化我們對 SMA 社群的承諾，以及我們專注於提供具實質意義的創新，為這種罕見且具毀滅性的疾病患者帶來長久改變。我們期待向各位更新我們持續取得的進展。

And with that, we'll now open the line for questions. Operator?

接下來，我們將開放提問。接線員？

(Operator Instructions)

（接線員指示）

Eric Schmidt, Cantor.

Eric Schmidt，Cantor。

My question is on Apitegromab second fill-finish provider. How confident are you that this facility alone, independent of Catalan can support approval by the PDUFA? And can you share any anecdotes from your FDA interactions to support that they're making progress in the review of the package you submitted?

我的問題是關於 Apitegromab 的第二家充填與包裝（fill-finish）供應商。你們對於僅靠這個廠房、在不依賴 Catalan 的情況下，能在 PDUFA 前支援核准的把握有多大？另外，你們能否分享一些與 FDA 互動的具體情況，以佐證他們正在推進對你們所提交資料套件的審查？

Yeah, as we noted, really dating back to that March 3 Type C meeting, we were really gratified as we were updating the FDA on the rapid and meaningful progress we were making in our second facility that really together we agreed that resubmitting our BLA with two fill-finish facilities and actually having a plan to enable an Apitegromab approval with the fastest path.

是的，正如我們提到的，回溯到 3 月 3 日的 Type C 會議，當我們向 FDA 更新我們在第二座廠房所取得的快速且具實質意義的進展時，我們確實感到非常欣慰；我們雙方也一致同意，重新提交包含兩個充填-封裝（fill-finish）廠的 BLA，並且實際上制定一個計畫，以最快路徑促成 Apitegromab 的核准。

Whether or not it be Catalent Indiana, the second fill-finish facility or both, we were gratified that there was a complete alignment between us and the agency that that was the best approach. As we've noted on the call, the team here at Scholar Rock and the second facility, we're really proud of their efforts to now have more vials of Apitegromab ready for launch from this facility than we do from Catalent Indiana.

無論是 Catalent Indiana、第二個充填-封裝廠，或兩者並行，我們都很欣慰我們與主管機關之間完全一致認為這是最佳做法。正如我們在電話會議中提到的，Scholar Rock 這裡的團隊以及第二座廠房的團隊，我們都為他們的努力感到非常自豪；目前從這座廠房為上市準備好的 Apitegromab 小瓶數量，已經多於 Catalent Indiana。

And frankly than we did from Catalent Indiana at the time of our last PDUFA date, I think underscores how well we are working together. The review, I think, as both Akshay and I noted, is progressing well. The FDA has all of the data that we agreed upon during our Type C meeting. That was going to enable the review and eventual approval from that second facility, so we're pleased with that. And as we're noting, we're corresponding with the FDA on that.

坦白說，也比我們在上一次 PDUFA 日期時從 Catalent Indiana 所準備的還多，我認為這凸顯了我們合作得有多好。審查進度，我想如 Akshay 和我都提到的，進展順利。FDA 已取得我們在 Type C 會議中同意提供的所有資料。這些資料將使第二座廠房的審查以及最終核准得以進行，因此我們對此感到滿意。而且如我們所說，我們也正就此與 FDA 保持往來溝通。

I would also note a couple of other facts that I think are important here. That second facility has had successful recent site inspections by FDA and EMA. None of the approvals from this facility in the last 12 months have required a PAI or a PLI. And I think underscoring the performance of that facility during that time, the site underwent multiple routine GMP, general site inspections by EMA and FDA. So we feel really good about the position that we are in.

我也想補充幾個我認為在此很重要的事實。第二座廠房近期已成功通過 FDA 與 EMA 的場址稽查。該廠在過去 12 個月內的核准案，沒有任何一項需要 PAI 或 PLI。而且為了凸顯該廠在這段期間的表現，該場址也接受了 EMA 與 FDA 多次例行 GMP、一般場址稽查。因此我們對目前所處的位置感到非常有信心。

And then I might close by saying we contemplated the, well, what if we remove one of the facilities from the application? Could it impact our timeline at all? And that was contemplated and discussed between us and the agency. And based upon our discussions with the agency dating back to those March discussions, we do not expect that removing a facility from the application would have any impact on our ongoing review timeline at all.

最後我想說，我們也曾考量過：嗯，如果我們把其中一個廠房從申請案中移除會怎麼樣？是否會對我們的時程造成任何影響？這點我們與主管機關已經思考並討論過。根據我們與主管機關自 3 月討論以來的交流，我們不預期從申請案中移除一個廠房會對目前進行中的審查時程造成任何影響。

So Keith and team are ready to launch at any time between now and up to our September 30 PDUFA date. I'm certainly gratified by Scholar Rock's technical operations and quality team. They have stood up this second fill-finish facility faster than almost any example we can find in the industry.

因此 Keith 和團隊已準備好在現在到 9 月 30 日 PDUFA 日期之間的任何時間點啟動上市。我也確實對 Scholar Rock 的技術營運與品質團隊感到欣慰。他們建立第二個充填-封裝廠的速度，幾乎快過我們在產業中能找到的任何案例。

And they now have more Apitegromab vials that will be commercially available from this second facility, and they are at our labeling and packaging site awaiting approval. We're super excited. So we'll continue to keep you all updated over these last 55 days, but we feel like we're in a really good position. Thank you.

而且他們現在已備妥更多將由第二座廠房供應、可供商業化的 Apitegromab 小瓶，並已送達我們的標示與包裝場址，正等待核准。我們非常興奮。因此在接下來最後 55 天我們會持續向各位更新，但我們覺得目前處於非常有利的位置。謝謝。

Krypta Devarakonda, Truist Securities.

Krypta Devarakonda，Truist Securities。

I have a follow-up question on Eric's question regarding the second site. Is the -- David, you just mentioned that you can drop one of the sites at any time and it won't delay, but I was wondering if Catalent remains classified as OAI, is there a deadline or a date for administratively withdrawing that site so it doesn't trigger any delay for your PDUFA? Thank you.

我想就 Eric 關於第二個場址的問題追問一下。David，你剛提到你們可以在任何時間點撤掉其中一個場址而不會延遲；但我想問的是，如果 Catalent 仍被歸類為 OAI，是否有一個期限或日期需要在行政上撤回該場址，以免對你們的 PDUFA 造成任何延遲觸發？謝謝。

We are in correspondence with the agency. We're both well aware that the inspection classification is still pending from the April re-inspection. As Akshay noted, it's now drifted a bit beyond the 90-day guidance period, but it is only a guidance period from the FDA that they would classify an inspection. And look, we're very direct with one another about when we would reach that step, should we need to reach that step.

我們正與主管機關保持往來溝通。我們雙方都很清楚，4 月複查（re-inspection）後的稽查分類仍在待定中。如 Akshay 所說，目前已稍微超過 90 天的指引期間，但那只是 FDA 對於稽查分類時程的指引，而非硬性規定。而且你看，我們彼此之間對於若需要走到那一步、何時會走到那一步，都非常直接坦率地溝通。

In your example that you provided, let's just say the inspection classification reveals that there's no change to the current OAI classification. We've discussed with the agency, it would be a relatively simple step to notify them on their signal that we are removing that site, and the review would progress with that second facility.

以你提出的例子來說，假設稽查分類結果顯示目前的 OAI 分類沒有改變。我們已與主管機關討論過，屆時只要在他們發出訊號後通知他們我們將移除該場址，這會是一個相對簡單的步驟，而審查將以第二座廠房繼續推進。

And again, with our alignment with the FDA, we would expect no impact to the ongoing timeline. So that's where we are. I think Akshay and I are both heartened by the fact that we have ongoing open correspondence with the FDA, and the review is progressing well.

再者，基於我們與 FDA 的一致共識，我們預期對正在進行的時程不會有任何影響。所以目前就是這樣的狀態。我想 Akshay 和我都因為我們與 FDA 持續、開放的往來溝通而感到振奮，而且審查進展良好。

Tessa Romero, JPMorgan.

Tessa Romero，JPMorgan。

So just to double click here on some of these earlier comments, what are the specific items procedurally from now to September 30 that still need to be ticked off to allow for an approval? I think Akshay used the words final steps. And just to set the record straight, is there any reason to believe that the FDA will not be able to complete this review by September 30?

我想再更深入確認一下先前的一些評論，從現在到 9 月 30 日，程序上還有哪些具體事項需要完成，才能促成核准？我記得 Akshay 用了「最後步驟」這個詞。另外，為了釐清紀錄，是否有任何理由認為 FDA 無法在 9 月 30 日前完成這次審查？

Yeah, I'll just hand it over to Akshay but just underscore that last question. There really is no reason to believe with the work that's been done at the second facility and where we believe the FDA is in terms of reviewing the application, which as you recall, right, our resubmitted BLA really only included the new information from the second facility and the updated safety database. There's no reason to believe the FDA can't get their work done in these next 55 days. Akshay?

是的，我先把問題交給 Akshay，但我想先強調你最後那個問題。就第二座廠房已完成的工作，以及我們認為 FDA 在審查申請案方面所處的位置而言，確實沒有任何理由相信他們無法在接下來 55 天內完成工作；如你所記得的，我們重新提交的 BLA 其實只包含第二座廠房的新資訊以及更新後的安全性資料庫。沒有理由相信 FDA 無法在接下來 55 天內完成他們的工作。Akshay？

Yeah, I would just reconfirm that test. So starting last November when we had a face-to-face meeting with the FDA with all relevant parties and in March, the FDA guided that Catalent is the lead site in the MAA, but the second site would be welcome because it gives them greater ability to help us get this drug approved in a compliant manner to patients in a high unmet need setting.

是的，我會再確認一次。從去年 11 月我們與 FDA 進行面對面會議、所有相關方都在場開始，以及在 3 月時，FDA 指引指出 Catalent 是 MAA 的主導場址，但也歡迎第二個場址，因為這能讓他們有更大的能力，以合規的方式協助我們讓這款藥物核准，提供給高度未被滿足需求的病患。

So at the March juncture, we said we're ready to submit a second fill-finish site. They welcome that. We've talked through the process of how to get to submission and then the finish line with the PDUFA date, and we are exactly on track with all of that. Their review of the second fill site is progressing well.

因此在 3 月那個時間點，我們表示已準備好提交第二個充填-封裝場址。他們對此表示歡迎。我們也已把從如何走到提交、再到以 PDUFA 日期為終點的整個流程都談清楚，而我們在所有事項上都完全按計畫進行。他們對第二個充填場址的審查進展順利。

And as David said, we see no reason why we can't get to the PDUFA date with this drug approved before or at that time. And so it certainly feels on track. And we're very grateful to the agency's guidance and the expeditious manner in which they've been working with us and the true engagement and partnership.

而且如 David 所說，我們看不出有任何理由無法在 PDUFA 日期之前或當日讓這款藥物獲得核准。因此整體確實感覺一切都在軌道上。我們也非常感謝主管機關的指引，以及他們以迅速的方式與我們合作，並展現真正的投入與夥伴關係。

Michael Yee, UBS.

Michael Yee，UBS。

We were just wondering, do you have any color or any feedback from the FDA on that later-than-expected reclassification of the Catalent site, given the decision was sort of expected by the end of July? If you just have any feedback, you could pass along.

我們想請教，你們是否能提供一些背景或 FDA 的任何回饋，關於 Catalent 場址重新分類（reclassification）比預期更晚的情況？因為原本似乎預期在 7 月底前會有決定。如果你們有任何回饋可以分享的話。

Yeah, the only thing that we would note is obviously, the inspection report is public as are the 483 observations in that inspection report. We are well aware that Novo Nordisk provided a pretty robust response as they had the option to do within 15 days of that inspection. So there was a lot there for the FDA to review.

是的，我們唯一想補充的是，很明顯地，稽查報告是公開的，該稽查報告中的 483 觀察事項也同樣是公開的。我們也很清楚 Novo Nordisk 在該次稽查後 15 天內依其可行選項提交了相當完整的回覆。因此 FDA 需要審閱的內容相當多。

And I would just note that that guidance period is a guidance period. We're aware that sometimes the FDA does take a bit more time than that 90-day period. And as we noted, we're awaiting that. I'm sure our friends at Novo Nordisk are awaiting that inspection classification.

我只想補充說明，那個指引期間就是指引期間。我們也知道，有時 FDA 的確會比那個 90 天期間花更久的時間。如同我們所提到的，我們正在等待該結果。我相信我們在 Novo Nordisk 的朋友也在等待那份稽查分類結果。

While in parallel, the EMA is awaiting that classification, we're also engaging with our European regulators on the inclusion of our second fill-finish facility. So there really is nothing more to it other than the fact that it does happen. The 90 days are not a statutory requirement. The FDA literally has provided that as guidance. There's probably a lot there that the FDA is reviewing and we await, like everybody else, the pending inspection classification.

同時，EMA 也在等待該分類結果之際，我們也正與歐洲監管機構就納入我們第二座充填與成品製造（fill-finish）設施進行溝通。所以除此之外其實沒有更多含意，這種情況確實會發生。90 天並非法律規定的要求。FDA 只是把它作為指引提供。FDA 可能正在審查很多事項，而我們也和大家一樣，等待尚未出爐的稽查分類結果。

But I bring it back to this, both in US and Europe, we're continuing to move forward with this meaningful progress that we've made with our second fill-finish facility. Really wanted to make sure that we had a belt and suspenders approach to serving children and adults living with SMA and their families. And we feel like we're in a position of strength as we move forward and we await like everybody else, that inspection classification.

但我想回到重點：無論在美國或歐洲，我們都在持續推進第二座充填與成品製造設施所取得的重大進展。我們確實希望採取「雙重保險」的方式，來服務罹患 SMA 的兒童與成人及其家庭。我們認為在持續推進的同時，我們處於有利位置，並且也和大家一樣等待那份稽查分類結果。

Cory Kasimov, Evercore.

Cory Kasimov，Evercore。

I guess, I'll shift gears a little bit here and want to ask about your national and regional payer discussions. I'm curious how they're framing Apitegromab in step-edit terms. Are you seeing any payers signal that they'll impose time limits or require a re-review of benefit after a shorter initial authorization? Thank you.

我想我稍微換個話題，想請教你們與全國性及區域性支付方（payer）的討論。我很好奇他們在「階梯式用藥管理」（step-edit）的脈絡下，如何定位 Apitegromab。你們是否看到任何支付方暗示會設定時間限制，或在較短的初始核准後要求重新審查療效/給付？謝謝。

Thanks, Cory. I'm going to hand that over to Keith for his comments. I would just note as a headline, as Keith is prepared to answer that. We do believe this robust clinical development program that we've been running for, seven-plus years, and again, the fact that we met the highest bar, which was the Hammersmith Motor Function Scale and SMA, with a Statsig result from our pivotal SAPPHIRE trial, the robustness of that data sets up very well.

謝謝你，Cory。我把這題交給 Keith 來回應。在 Keith 準備回答之前，我先用一句話概括：我們確實相信，我們已執行超過七年的強健臨床開發計畫，再加上我們在關鍵性 SAPPHIRE 試驗中達到最高門檻——也就是 SMA 的 Hammersmith 運動功能量表（Hammersmith Motor Function Scale），且取得具統計顯著性（Statsig）的結果——這些強而有力的數據基礎非常有利。

As you know, I'll just underscore it for everybody listening in. I know you know it quite well, Cory. Patients were randomized who were on ongoing SMN-targeted therapy to receive either placebo or Apitegromab. So, they were on these ongoing therapies. And again, to hit that highest bar of the Hammersmith Motor Function Scale and SMA at Statsig with a .019 p-value.

如各位所知，我也再為所有正在收聽的人強調一次。我知道你很清楚，Cory。受試者是在持續接受 SMN 標靶治療的情況下被隨機分派，接受安慰劑或 Apitegromab。也就是說，他們都在既有治療上。而且再次強調，我們在 SMA 的 Hammersmith 運動功能量表這個最高門檻上達到統計顯著，p 值為 0.019。

We feel with a very low number of patients, we think sets up very well for those. A national and regional payer discussion. Keith?

我們認為在受試者人數非常少的情況下仍能達成這樣的結果，對於全國性與區域性支付方的討論非常有利。Keith？

Yeah, thanks, David. Look, as I stated in the prepared remarks, the team has been working and really extending not only our reach with these various payers, whether it's the commercial payers or government payers, but also the quality of the meetings by bringing in members from our medical team to discuss the robust clinical data package from our Apitegromab studies, and ultimately with the goal of making Apitegromab available for the broadest possible audience of children and adults living with SMA.

好的，謝謝你，David。如同我在事先準備的發言中提到的，團隊一直在努力，不僅擴大我們與各類支付方的接觸範圍——不論是商業保險支付方或政府支付方——也透過邀請醫療團隊成員參與，提升會議品質，來討論我們 Apitegromab 研究所累積的強健臨床數據套件；最終目標是讓 Apitegromab 能提供給最廣泛的 SMA 兒童與成人族群。

So as I've stated before, the real goal with these meetings is to be able to create policies as rapid as possible and policies that we believe will align more with the potential label, the FDA label, and less with an inclusion/exclusion criteria from our SAPPHIRE study.

因此，如我先前所說，這些會議的真正目標，是能夠制定盡可能快速的給付政策，並且我們相信這些政策會更貼近未來可能的標示（label）、也就是 FDA 核准標示，而不是更貼近我們 SAPPHIRE 研究的納入/排除條件。

With all that being said, if you take a look at the data on treatment for SMA, so I'm talking about the three SMN-targeted therapies that are available, almost 100% of them have a prior authorization. So I fully expect that you'll see that Apitegromab will have a prior authorization even when we have favorable policies that are constructed.

在此基礎上，如果你看 SMA 的治療數據——我指的是目前可用的三種 SMN 標靶治療——幾乎 100% 都需要事前授權（prior authorization）。所以我完全預期，即使我們建立了有利的給付政策，Apitegromab 也會需要事前授權。

Amy Li, Jefferies.

Amy Li，Jefferies。

I just wanted to put a finer point on the second fill-finish facility. You mentioned that you submitted data that the FDA requested the Type C, which sounds encouraging, but could you give us a sense of what was included in that data package? Are stability runs and release testing for this facility fully complete, and you would consider the facility launch ready? And do you expect any additional clearance from the FDA? Thanks so much.

我想更精準地追問第二座充填與成品製造設施。你提到你們提交了 FDA 在 Type C 會議中要求的資料，聽起來很令人鼓舞，但你們能否讓我們了解該資料套件包含哪些內容？該設施的穩定性試驗批次（stability runs）與放行檢測（release testing）是否已全部完成，你們是否認為該設施已具備上市啟動（launch ready）條件？另外，你們是否預期還需要 FDA 的任何額外放行/核准？非常感謝。

Yeah, I mean, what was inclusive and what we agreed upon was obviously the data that we generated from engineering runs, PPQ runs. The FDA has the full package in hand where we know their review of that is progressing well. We're in correspondence with them on that. Everything is straightforward and perfunctory.

是的，我的意思是，所涵蓋的內容以及我們同意的範圍，顯然包括我們從工程批次（engineering runs）、PPQ 批次所產生的資料。FDA 已完整收到整套資料，我們知道他們的審查進展良好。我們也就此與他們保持往來溝通。一切都很直接、也屬例行程序。

And there is, I guess, just to underscore the question, there is no more testing that is required for that for the product that has been viled at that facility at all. And again, as noted, that product is at our third-party labeling and packaging facility awaiting approval to support the launch.

另外，我想再呼應你的問題重點：對於在該設施完成分裝（vialed）的產品，完全不需要再做任何額外檢測。而且如先前所述，該產品目前已在我們的第三方貼標與包裝廠，等待核准以支援上市。

And of course, in a belt and suspenders approach, we have vials from Catalent Indiana at the packaging facility as well. So we're in a really good position, we're gratified to have reached an agreement with the FDA in March at the Type C meeting on what was required.

當然，採取「雙重保險」的做法，我們也在包裝廠備有來自 Catalent Indiana 的瓶裝產品。因此我們處於非常好的位置；我們也很欣慰在 3 月的 Type C 會議上與 FDA 就所需項目達成一致。

I would note that what was required was delivered to the FDA in a very timely fashion, because you don't only agree on what is submitted, but when it would be submitted within your framework of your PDUFA date, and we felt like we delivered that in a very timely fashion.

我也要指出，所要求的內容我們已非常及時地交付給 FDA，因為你們不僅會就提交什麼達成一致，也會在 PDUFA 日期的框架下就何時提交達成一致，而我們認為我們的提交非常及時。

And we're looking forward to these next 55 days to get through the final step and eventually launch Apitegromab. So thank you very much for your question.

我們也期待接下來這 55 天能完成最後一步，並最終推出 Apitegromab。非常感謝你的提問。

Gary Nachman, Canaccord.

Gary Nachman，Canaccord。

So as you've been preparing for a while now with the commercial team, any other initiatives you need to put in place between now and approval, or is it just really waiting for the final label? And what's the low-hanging fruit to go after with SMA patients where there could be a fair amount of pent-up demand for Apitegromab? And how long do you think it will take to get those patients on board? Thanks.

既然你們商業團隊已準備一段時間了，在核准前到現在之間，還有其他需要落地的措施嗎？還是其實就是等待最終標示？另外，在 SMA 病患中，你們認為最容易先切入的「低垂果實」是哪些族群——也就是對 Apitegromab 可能存在相當程度的累積需求（pent-up demand）的族群？你們認為要花多久時間才能讓這些病患開始用藥？謝謝。

Yeah, I'll start, and Keith will get in. But I think the -- as I mentioned during the call, Gary, we spend a lot of time with the community, patients, their families, the advocacy groups, the healthcare providers. And our ambition is that any patient living with SMA that can benefit from Apitegromab should have access to Apitegromab.

好的，我先回答，然後 Keith 也會補充。不過我想——如同我在電話會議中提到的，Gary，我們花了很多時間與社群互動，包括病患、家屬、倡議團體以及醫療照護提供者。我們的企圖心是：任何罹患 SMA 且能從 Apitegromab 受益的病患，都應該能取得 Apitegromab。

And so, we really do look at this holistically across the 35,000 patients globally that have received an SMN-targeted therapy, and we believe that we can really offer meaningful benefits to them. So we do think about it very holistically.

因此，我們確實以更全面的角度來看待這件事：全球約 35,000 名已接受 SMN 標靶治療的病患，我們相信我們能為他們帶來有意義的效益。所以我們確實是以非常整體的方式來思考。

Keith can share with you some of the dynamics at launch, but I think our general view is it could vary patient by patient, family by family, physician by physician in terms of their thinking about commencing treatment. Keith?

Keith 可以和你分享上市時的一些動態，但我想我們的整體看法是：在是否開始治療的思考上，可能會因病患而異、因家庭而異、也因醫師而異。Keith？

Yeah, thanks, David. I guess, first of all, what else is there to do? The point I want to make really clear is we are ready if we were to get the call tomorrow. The team is chomping at the bit to really get out there and launch this product. With that being said, there's always additional work that we can do.

是的，謝謝，David。我想，首先，還能做什麼呢？我想非常清楚地表達的是，如果明天接到通知，我們已經準備好了。團隊早就摩拳擦掌，真的很想走出去把這個產品推出上市。話雖如此，我們總是還有一些額外的工作可以做。

One of the main things that's taking place that I mentioned is really working with the various centers so that we can be prepared with these treatment centers on a case-by-case basis to work through the process of enrolling patients into our Scholar Rock Supports program.

我提到正在進行的其中一項主要工作，是與各個中心密切合作，讓我們能夠針對每一家治療中心逐案做好準備，推進將病患納入我們的 Scholar Rock Supports 計畫的流程。

We cannot begin to do any of this until after we have FDA approval. So we are doing a lot of dry run work here so that we can have a seamless and flawless execution as we take patients from being prescribed a product to ultimately being able to receive the product.

在獲得 FDA 核准之前，我們無法開始做任何這些事情。因此我們正在做大量的演練工作，確保在把病患從「開立處方」一路銜接到「最終能夠拿到產品」時，能夠順暢且無瑕地執行。

Where do you think the pent-up demand is? I've shared before on previous calls. We do have an early access program. Those will be the first patients that we will be focused on converting from early access product over to commercial product. That being said, we don't have full line of sight into what insurance coverage these early access patients have, so we're going to have to be going through that process and enrolling them in our program.

你認為被壓抑的需求會在哪裡？我在之前的電話會議上也分享過。我們確實有一個早期使用計畫。這些將會是我們首先聚焦、要把早期使用產品轉換為商業化產品的第一批病患。不過，我們並沒有完全掌握這些早期使用病患的保險給付情況，因此我們必須走完相關流程，並將他們納入我們的計畫。

The next will be our open-label extension patients, our ONYX patients. I want to remind you that they will have to go to a closeout visit of that study before they can even convert over to commercial drug, but we will begin to work them through the process of our Scholar Rock Supports program.

接下來會是我們的開放標籤延伸試驗病患，也就是 ONYX 病患。我想提醒各位，他們必須先完成該研究的結案訪視（closeout visit），之後才能轉換到商業化藥物，但我們會開始協助他們走 Scholar Rock Supports 計畫的流程。

So when you ask how long will it take? It will take time, mostly because you're going to see a prior auth with all of our patients that are going to be prescribed Apitegromab. The majority of them, you are most likely going to receive a denial for various reasons, whether it's a J-code or a policy not created or for some other reason. That will then send us into an appeal process, which can sometimes take some time.

所以當你問需要多久？這會需要時間，主要是因為所有將被處方 Apitegromab 的病患，你都會看到需要事前授權（prior auth）。其中大多數很可能會因各種原因收到拒付（denial），不論是因為沒有 J-code、或保單政策尚未建立、或其他原因。接著就會進入申訴（appeal）流程，而這有時會花上一段時間。

So I think that there will be certain patients that will have coverage that will allow them to go on sooner rather than others. But what I've typically seen other launches is during this first six months of launch, while we will be without a J-code. The time -- the average time from prescription to a patient actually being able to get infused is greater than 60 days.

因此我認為，會有部分病患因為已有給付而能更快開始用藥，其他人則會慢一些。但我通常在其他產品上市時看到的是，在上市的前六個月、也就是我們仍沒有 J-code 的期間，從開立處方到病患實際能夠接受輸注（infused）的平均時間會超過 60 天。

Marc Frahm, TD Cowen.

Marc Frahm，TD Cowen。

Maybe just -- I mean, it seems like the medical review is kind of done on both sides of the Atlantic. So maybe you can speak to the labeling discussions. And do you expect a largely identical label or do you think there are maybe important differences between the US and European label? And then I'll probably have a follow-up.

也許就——我的意思是，看起來大西洋兩岸的醫學審查都差不多完成了。所以也許你可以談談標示（labeling）的討論。你預期標示會大致相同嗎？還是你認為美國與歐洲的標示可能會有一些重要差異？然後我可能會再追問一題。

Yes, no, it's an important question. As you know, Akshay and I both addressed that we felt like we were in a really good spot at the end of the last review period with the FDA, and we're going to be picking it up more. We picked it up in the BLA right where we last left off, and then the comparisons between the two comments.

是的，這是個重要的問題。如你所知，Akshay 和我都提到，我們覺得在上一個與 FDA 的審查週期結束時，我們處於非常好的位置，而我們將會再把它接續推進。我們在 BLA 中就是從上次停下來的地方接著做，然後再比較兩邊的意見。

Yeah, I would just say we're very happy and comfortable with the way the dialogue has gone through the medical review. And obviously, it's not for us to comment on the final label, but we are certainly grateful for the engagement and the very constructive conversation. So we look forward to getting this drug approved in US and Europe, and I think we'll be comfortable with how to get it to the right patients.

是的，我只想說，我們對醫學審查過程中的對話進展感到非常滿意且安心。當然，最終標示不是由我們來評論，但我們確實非常感謝對方的投入，以及非常具建設性的討論。因此我們期待這個藥物能在美國與歐洲獲得核准，我想我們也會對如何把它提供給合適的病患感到有把握。

Yeah, and Marc, I think due to the great work by Akshay and the entire team. I think at the end of the day, we ask ourselves, are we going to have an opportunity to serve a meaningful number of patients living with SMA in the US and Europe? And again, eventually, our ambition is to reach patients in 50 countries around the world.

是的，而且 Marc，我想這要歸功於 Akshay 和整個團隊的出色工作。我想最終我們會問自己：我們是否有機會在美國與歐洲為相當數量、罹患 SMA 的病患提供服務？而且同樣地，最終我們的企圖心是觸及全球 50 個國家的病患。

And I think that Akshay has put us in a really good and the entire team, Jing and the entire team has put us in a really good position to be able to do that. But Akshay is right, until we have final USPIs and SmPCs. I think we'll comment on that when it's the right time. And hopefully, that time is coming in the coming days.

我認為 Akshay 讓我們處於非常好的位置，而整個團隊、Jing 以及整個團隊也讓我們處於非常好的位置，能夠做到這一點。但 Akshay 說得對，在我們拿到最終的 USPI 與 SmPC 之前，我想我們會在適當的時機再做評論。也希望那個時機會在接下來幾天內到來。

Okay. That's helpful. And then just on the CMC side, the EMA clearly seems to be essentially deferring to the FDA on the Catalent Indiana facility. Do you expect them to ultimately act similarly with the second facility, or is there something about the either that facility itself or the flexibility that the FDA granted you in terms of CMC requirements there that might lead the EMA to be more proactive itself in making a decision?

好的。這很有幫助。另外在 CMC 方面，EMA 顯然似乎基本上是在 Catalent 印第安納廠這件事上，實質上採取依循 FDA 的做法。你預期他們最終在第二個廠也會採取類似作法嗎？或者該廠本身、或 FDA 在那裡就 CMC 要求所給予的彈性，是否可能讓 EMA 更主動自行做出決定？

It's a very thoughtful question, Marc. Akshay?

這是個非常周到的問題，Marc。Akshay？

Yes, I mean, in our prepared remarks, we emphasized that second finished facility is in very good standing with regulators. And we're delighted that that's in progress with the FDA. Now, with respect to the EMA, obviously, any approval for a Apitegromab that involves the second facility will help. And we are right now heavily engaged with the CHMP to work on the progress of the EMA to completion and how we incorporate the second finished facility, if necessary.

是的，我的意思是，在我們事先準備的發言中，我們強調第二個成品（finished）廠在監管單位那裡的狀態非常良好。而且我們很高興 FDA 那邊的流程正在推進。至於 EMA，當然，任何涉及第二個廠的 Apitegromab 核准都會有幫助。我們目前也正與 CHMP 深度互動，推進 EMA 走到完成，並在必要時把第二個成品廠納入其中。

Kyle Pippito, Wolfe Research.

Kyle Pippito，Wolfe Research。

One for Europe, if the FDA maintains the OAI classification for Catalent, can you walk us through your potential timeline to get the second fill-finish facility into the MAA. And your thoughts on the earliest projected timeline for European approval? Thank you.

關於歐洲的問題：如果 FDA 維持對 Catalent 的 OAI 分類，你能否帶我們了解一下，將第二個充填-成品（fill-finish）廠納入 MAA 的可能時間表？以及你對歐洲最早可能核准時間的看法？謝謝。

Thank you very much. And as we noted during the call, while EMA and we await the classification decision from the FDA, in parallel, we're having the dialogue. EMA is very well aware of where we are with the second fill-finish facility. Akshay?

非常感謝。如同我們在電話會議中提到的，當 EMA 與我們在等待 FDA 的分類決定時，我們也同步在進行對話。EMA 非常清楚我們在第二個充填-成品廠方面的進度。Akshay？

Yeah, and as that dialogue is ongoing, I don't want to second guess what the final advice will be vis-a-vis necessity or the mechanism by which we incorporate the second fill-finish facility. But suffice it to say that the engagement and flexibility that both FDA and CHMP have shown us is very gratifying to us. They appreciate the unmet need and they're working with us. So we will be guided by them. There's a long game conversation and hopefully soon in due course we will update everything.

是的，隨著對話持續進行，我不想去臆測最終建議會是什麼——不論是關於是否必要，或是我們將第二個充填-成品廠納入的機制。但可以說的是，FDA 與 CHMP 所展現的投入與彈性，讓我們非常欣慰。他們理解未被滿足的醫療需求，並且正與我們合作。因此我們會依循他們的指引。這是一場長期的對話，希望不久之後、在適當時點，我們會更新所有進展。

I think just as a capper, I think what Akshay and I are really gratified just as we've experienced with the FDA, we're just gratified by the receptivity and the dialogue with EMA. But again, we'll await the specific approach pending the inspection classification. And of course, in parallel, our discussions on the second facility.

我想作為總結，正如我們與 FDA 的經驗一樣，Akshay 和我都對 EMA 的接受度與對話感到非常欣慰。但同樣地，我們會在等待稽查分類結果後，再採取具體作法。當然，同時我們也在推進關於第二個廠的討論。

Etzer Darout, Barclays.

Etzer Darout，巴克萊。

For FSHD on the clinical trial site, I know you guys mentioned that you're looking to focus in slightly less severe patients. And you're listing the participation requirement is a 1.5 to 3 on a Ricci scale on a 0 to 5 scale. Just want to verify that you're using a modified scale there because I think the Ricci scale is 0 to 10. And could you give some color as to what percentage of the FSHD population falls within that scale range?

關於臨床試驗網站上的 FSHD，我知道你們提到你們希望把重點放在病情稍微不那麼嚴重的患者。而你們列出的參與要求是在 0 到 5 的量表上，Ricci 量表分數為 1.5 到 3。我只是想確認你們使用的是修正版量表，因為我認為 Ricci 量表是 0 到 10。另外你們能否補充說明一下，FSHD 人群中大約有多少比例落在這個分數區間？

Yeah, we're using the same Ricci scaling system or scoring system that the Roche folks have used. And I think just want to confirm what you said that we're indeed focusing on patients with a Ricci score of 1.5 to 3 because once you get beyond a score of 3, we know from an FSHD database we have access to and I don't know whether Roche had it or not. But we know that by MRI, many muscle groups. Including the quads, which was their primary endpoint, begin to show a significant fat infiltration and fibrosis.

是的，我們使用的是與 Roche 團隊所使用相同的 Ricci 分級系統或評分系統。我想確認你剛才說的，我們確實聚焦於 Ricci 分數 1.5 到 3 的患者，因為一旦超過 3 分，根據我們可取得的 FSHD 資料庫（我不確定 Roche 是否也有），但我們知道從 MRI 來看，許多肌群包括股四頭肌（他們的主要終點），開始出現顯著的脂肪浸潤與纖維化。

So focusing on patients with the higher scores, I think, is a tough ask. And so, you need some muscle preserve so that the anti-myostatin mechanism can act on it to boost muscle mass and hopefully function too. Also, in terms of functionality, you'll note that their inclusion criteria included a 10-meter walk test between four to 12 seconds. So the upper bound is 12, whilst we've stipulated that the upper bound for our 10-meter walk is less than five.

因此我認為把重點放在更高分數的患者，是一個很難的要求。所以你需要保留一定的肌肉，讓抗肌生成素（anti-myostatin）的機制能作用其上，以增加肌肉量，並希望也能改善功能。另外在功能性方面，你會注意到他們的納入標準包含 10 公尺步行測試介於 4 到 12 秒。所以他們的上限是 12 秒，而我們規定 10 公尺步行的上限是小於 5 秒。

So we're certainly in that mild to moderate group of patients whilst they were in the moderate to severe. As to the exact numbers of patients. This is, the commonest muscular dystrophy, or there's at least a very significant five-digit number of patients around the world to help, and I think we're comfortable that we will help many patients should this drug ultimately be approved in that space. So I'm not concerned about that, and of course.

所以我們確實是在輕度到中度的患者族群，而他們是在中度到重度。至於患者的確切人數。這是最常見的肌肉萎縮症之一，或者至少全球有相當可觀的五位數患者人數可供協助；我想我們有信心，若這款藥物最終在該領域獲批，我們將能幫助許多患者。所以我不擔心這點，當然。

As we know, in all these rare diseases, as soon as the therapeutic appears, the rate of diagnosis improves and the rate of access to therapies improves, and patients start getting put on therapy earlier and earlier in the course of their disease.

如我們所知，在所有這些罕見疾病中，一旦出現治療方法，診斷率就會提高、取得治療的比例也會提升，患者也會在疾病進程更早期就開始接受治療。

So we're looking forward, as we announced today, to getting momentum going now in the study. The study is initiated. And we're excited to do this study where we have wonderful mouse data and where we think there's a robust rationale with our drug.

因此我們期待，如同我們今天宣布的，現在就讓研究開始加速推進。研究已啟動。我們很興奮能進行這項研究；我們有很棒的小鼠數據，也認為我們的藥物具備強而有力的研發依據。

Basma Radwan, Leerink Partners.

Basma Radwan，Leerink Partners。

Could you please share your perspective on the general and recent updates regarding the ILEA high-dose prefilled syringe? Specifically, management indicated that it plans to add a third plant on the regulatory package to enhance the likelihood of approval. How do you interpret that decision, given that the situation is very similar to yours with regard to Catalent involvement? Do you think it suggests that Catalent problems may be still outstanding? That's it for us. Thank you.

能否請你分享你對 ILEA 高劑量預充式注射器整體以及近期更新的看法？具體而言，管理層表示計畫在申報資料中加入第三家工廠，以提高獲批的可能性。鑑於在 Catalent 參與方面的情況與你們非常相似，你如何解讀這個決定？你是否認為這暗示 Catalent 的問題可能仍未解決？我們的問題就這些。謝謝。

Well, we know, first of all, I can't really comment on Regeneron other than to say I think we have a very different situation because our second fill-finish facility is different than their second fill-finish facility in this case, from our understanding. So that would just be a bright line.

嗯，首先我們知道，我其實無法評論 Regeneron，只能說我認為我們的情況非常不同，因為就我們的理解，在這個案例中，我們的第二家充填/成品製造（fill-finish）設施與他們的第二家充填/成品製造設施不同。所以這會是一條明確的分界線。

The similarity is that we both have Catalent Indiana in our applications. So I really can't comment on their commentary beyond the fact that I would note that our second fill-finish facility is in good standing with the FDA and EMA. They've had several general site inspections by FDA and EMA in 2025 and 2026. Their last 12 months of approvals for products there, and they have like nearly three dozen or more commercially available products from that facility.

相似之處在於，我們雙方的申請案都包含 Catalent Indiana。因此除了指出我們的第二家充填/成品製造設施在 FDA 與 EMA 方面狀態良好之外，我無法對他們的說法多作評論。該設施在 2025 與 2026 年接受過 FDA 與 EMA 的多次一般性場址稽查。過去 12 個月內該廠也有產品獲批，而且該設施有將近三打或更多已商業上市的產品。

But in the last 12 months, all approvals from that fill-finished facility, the PLI, PAIs have been waived by the agency. So I would just comment that, yes, as we've noted, the Catalent Indiana inspection classification is still pending. So that could be one thing that what you're referring to means. But they're very different situations in that we have a different second fill-finish facility.

但在過去 12 個月中，該成品製造設施的所有核准案，機構都豁免了 PLI、PAI。所以我只能評論說，是的，如同我們先前提到的，Catalent Indiana 的稽查分類仍在等待中。因此你所提到的意思，可能就是指這一點。但兩者情況非常不同，因為我們有不同的第二家充填/成品製造設施。

Geoff Meacham, Citigroup.

Geoff Meacham，花旗集團。

David, in line with your belts and suspenders comment, does the second facility provide a fully independent approval path, or are there any elements of the filing that are still dependent on Catalent Indiana?

David，呼應你「雙重保險」的說法，第二家設施是否提供一條完全獨立的核准路徑，還是申報中仍有任何部分依賴 Catalent Indiana？

And then second question, maybe for Akshay, what regulatory work will be required to get subcutaneous Apitegromab into the next pivotal development? I just wanted to maybe go over that. Thank you.

第二個問題可能給 Akshay：要讓皮下（subcutaneous）Apitegromab 進入下一個關鍵性（pivotal）開發，還需要做哪些法規工作？我只是想把這點再梳理一下。謝謝。

Actually, you want to take both? Yeah, thanks, Geoff. So on the first question, the second fill/finish provides a fully independent path. And the details of that were discussed in that March meeting we mentioned in the prepared remarks. We feel good about that. And I think that sort of speaks to David's belt-and-suspenders comments.

其實你要兩個都回答嗎？好的，謝謝你，Geoff。第一個問題，第二家充填/成品製造提供一條完全獨立的路徑。相關細節我們在先前準備稿中提到的 3 月會議裡已討論過。我們對此感到放心。我想這也呼應了 David 所說的「雙重保險」。

So that's great, and vis-a-vis the subcutaneous Apitegromab, just to refresh folks, in January, we shared data showing the wonderful sort of PK/PD profile of sub-Q Apitegromab, which makes all this feasible, and we have prepared a briefing document that we will be ready to send very soon after the approval of Apitegromab, so that we can engage with the regulators and begin that next important phase of development to bring a sub-Q option for patients.

很好。至於皮下 Apitegromab，先幫大家回顧一下：在 1 月，我們分享了數據，顯示皮下 Apitegromab 具有很出色的 PK/PD 特徵，使得這一切可行；我們也已準備好一份簡報文件，將在 Apitegromab 獲批後不久就能送出，以便與監管機構展開互動，並開始下一個重要的開發階段，為患者提供皮下給藥選項。

Now, that involves a dialogue with the FDA because for different drugs, different paths have been adopted. There's one view of the world that says it can be a PK/PD path matching the PK/PD criteria seen with Rab IV, the level of myostatin suppression and saturation and so forth. And the other extreme is you need to do more substantive development work. You mentioned pivotal.

接下來需要與 FDA 對話，因為不同藥物採用的路徑不同。其中一種觀點認為，可以走 PK/PD 路徑，去匹配 Rab 靜脈注射（IV）所見的 PK/PD 標準，例如肌生成素抑制與飽和的程度等等。另一個極端則是需要做更實質的開發工作。你提到了關鍵性試驗。

We are now finalizing the briefing documents to present the path forward that we think is reasonable, but we obviously need to engage with regulators to get their guidance. But as soon as we've done that, we will then provide an update in due course as to the path forwards because that will ultimately influence the timeline of getting subcutaneous Apitegromab to patients.

我們目前正在完成簡報文件，提出我們認為合理的後續路徑，但顯然需要與監管機構溝通以取得其指引。一旦完成，我們就會在適當時點更新後續路徑，因為這最終會影響皮下 Apitegromab 提供給患者的時間表。

Thank you. And this does conclude today's programming. Thank you so much for joining. You may now disconnect.

謝謝。今天的節目到此結束。非常感謝各位參與。您現在可以斷線。