Scholar Rock Holding Corp (SRRK) 2025 Q3 法說會逐字稿

Good morning, ladies and gentlemen, and welcome to Scholar Rock's third quarter 2025 conference call. (Operator Instructions) This call is being recorded on Friday, November 14, 2025.

各位女士、先生，早安，歡迎參加 Scholar Rock 2025 年第三季電話會議。（操作員指示）本次通話於2025年11月14日星期五進行錄音。

I would now like to turn the conference over to Scholar Rock. Please go ahead.

現在我將把會議交給 Scholar Rock 主持。請繼續。

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

早安.我是 Laura Ekas，Scholar Rock 的投資者關係副總裁。今天陪同我出席的有：董事長兼執行長大衛·哈拉爾；研發總裁阿克謝·瓦伊什納；營運長基斯·伍德；以及財務長維卡斯·辛哈。

For those of you participating via conference call, the accompanying slides can be accessed in the Events section on the Investors page of our website. During today's call is outlined on slide 2, 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.

對於透過電話會議參與的各位，可以在我們網站「投資者」頁面的「活動」部分存取相關幻燈片。今天的電話會議議程概述請見投影片 2，David 將作開場白並介紹業務最新進展。Akshay將檢視我們的研發進度。Keith 將介紹我們的商業準備活動的最新進展，Vikas 將介紹財務方面的最新進展。接下來我們將開放提問環節。

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 the 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、計劃和前景發表各種聲明，這些聲明構成 1995 年《私人證券訴訟改革法案》安全港條款所指的前瞻性聲明。

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 & 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，不應被視為代表我們截至未來任何日期的觀點。我建議您造訪我們網站的「投資者與媒體」版塊，查看我們最新的美國證券交易委員會聲明和文件。

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

接下來，我想把電話交給大衛。大衛？

Thank you, Laura, and good morning. Thanks to everyone for joining our third quarter earnings call today. In April, when I was appointed CEO after eight years in the Board Chairman role, and on the same day, we brought in Akshay Keep MCOs. We were confident that Scholar Rock positioned to be the next great global biotech powerhouse. We based this on several factors.

謝謝你，蘿拉，早安。感謝各位今天參加我們的第三季財報電話會議。今年四月，在我擔任董事會主席八年後被任命為首席執行官，就在同一天，我們引進了 Akshay Keep MCOs。我們當時很有信心，Scholar Rock 將成為下一個全球生技巨頭。我們是根據以下幾個因素做出決定的。

First, our conviction that the global opportunity with apitegromab in SMA alone offers the potential for many years of sustainable growth that will power our company through the end of this decade and into the next.

首先，我們堅信，光是 SMA 領域 apitegromab 的全球機會就蘊含著多年永續成長的潛力，這將推動我們公司在本十年末及下一個十年中持續發展。

Second, as world leaders in myostatin biology, our ability to deliver transformative therapies to patients suffering with additional rare, severe and debilitating neuromuscular disorders.

其次，作為肌肉生長抑制素生物學領域的全球領導者，我們有能力為患有其他罕見、嚴重和衰弱性神經肌肉疾病的患者提供變革性療法。

And third, leveraging our innovative platform to advance our novel subcutaneously administered myostatin inhibitor, SRK-439. When we joined Scholar Rock, the most significant milestone ahead was the September 22, PDUFA date for apitogramab in SMA, which had been granted priority review.

第三，利用我們的創新平台來推進我們新型皮下注射的肌肉生長抑制素抑制劑 SRK-439 的研發。當我們加入 Scholar Rock 時，最重要的里程碑是 9 月 22 日 SMA 的 apitogramab 的 PDUFA 日期，該藥物已獲得優先審查。

Our BLA was supported by robust data demonstrating apitegromab's efficacy and safety for children and adults living with SMA. Based upon our 188-patient prospective, randomized, double-blind, placebo-controlled multinational Phase III trial.

我們的生物製品許可申請得到了強有力的數據支持，這些數據證明了apitegromab對患有SMA的兒童和成人的療效和安全性。基於我們所進行的188位患者的前瞻性、隨機、雙盲、安慰劑對照的多國III期試驗。

This trial showed a statistically significant and clinically meaningful benefit in motor function as measured by the gold standard Hammersmith motor function scale for SMA. While we were disappointed to receive a complete response letter on September 22, we were pleased that the strength of our Phase III data was reflected in the FDA's review of our BLA.

該試驗表明，在運動功能方面，採用 SMA 黃金標準 Hammersmith 運動功能量表進行測量，結果顯示具有統計意義和臨床意義的改善。雖然我們對 9 月 22 日收到的完整回覆函感到失望，但我們很高興 FDA 對我們生物製品許可申請的審查反映了我們 III 期數據的實力。

And that the sole approvability issue referenced in the CRL was the status of our third-party fill, finish facility in Bloomington, Indiana, which is owned by Novo Nordisk. We know that it is not a matter of if but when apitegromab will be approved in the US for patients living with SMA. We are emboldened by the commitment we have made to the more than 35,000 patients globally living with SMA who have received an SMN targeted therapy.

CRL 中提到的唯一可批准性問題是我們位於印第安納州布盧明頓的第三方填充、包裝設施的狀態，該設施由諾和諾德公司擁有。我們知道，阿匹替格羅單抗在美國獲準用於治療脊髓性肌肉萎縮症（SMA）患者只是時間問題，而不是會不會核准的問題。我們為全球超過 35,000 名接受 SMN 標靶治療的 SMA 患者所作出的承諾而倍受鼓舞。

We are working expeditiously to deliver on our ambition that globally any patient with SMA who can benefit from apitegromab should have access to apitegromab. And now more than ever, we are confident in the significant opportunity that we have ahead of us to serve the SMA community as we work with the termination to bring this important medicine to children and adults with SMA. This is indeed what we know well and what we do well.

我們正在加緊努力，以實現我們的願景，在全球範圍內，任何能夠從阿匹替格羅單抗中獲益的 SMA 患者都應該能夠獲得阿匹替格羅單抗。現在，我們比以往任何時候都更有信心，我們面前有一個重要的機會，那就是為 SMA 群體服務，我們將努力促成這一重要舉措，為患有 SMA 的兒童和成人帶來這種重要的藥物。這正是我們擅長且精通的領域。

I would like to now provide a regulatory update on apitegromab. We had our Type A meeting with the FDA on Wednesday. We are grateful to the agency for their full participation, particularly in the context of a government shutdown. The meeting was in person and included the relevant leaders and decision-makers from the agency, including the neurology division and the office of compliance. Our team was joined by Kenneth Hobby, President of Cure SMA and representatives from Novo Nordisk.

現在我想就 apitegromab 的監管情況提供最新資訊。我們週三與FDA舉行了A類會議。我們感謝該機構的全力配合，尤其是在政府停擺的情況下。會議以面對面的形式舉行，與會者包括該機構的相關領導和決策者，包括神經科和合規辦公室的領導和決策者。我們的團隊與 Cure SMA 主席 Kenneth Hobby 和 Novo Nordisk 的代表一起進行了交流。

We were encouraged by the meeting -- it was constructive and collaborative. It was clear that there is a shared understanding of the high unmet need for the SMA community and a shared sense of urgency to bring apitegromab to children and adults with this disease. Novo Nordisk detailed the progress they have made in implementing their remediation plan at the Bloomington facility and affirmed that they expect the facility to be ready for reinspection by the end of this year.

這次會議令我們深受鼓舞——會議富有建設性，合作性強。很明顯，大家對 SMA 群體存在著巨大的未滿足需求有著共同的認識，並且都迫切希望將 apitegromab 帶給患有這種疾病的兒童和成人。諾和諾德詳細介紹了他們在布魯明頓工廠實施整改計畫所取得的進展，並確認他們預計該工廠將在今年年底前準備好接受重新檢查。

We discussed the path forward and await the final minutes of the meeting. We will continue to work closely with the FDA and anticipate resubmitting the BLA and US launch following approval of apitegromab for children and adults with SMA in 2026.

我們討論了未來的發展方向，並等待會議的最後總結。我們將繼續與 FDA 密切合作，並預計在 2026 年 apitegromab 獲準用於治療 SMA 兒童和成人後，重新提交 BLA 和美國上市申請。

I'd like to now turn to adding redundancy to our supply chain. When Novo Nordisk purchased the Bloomington site in December of 2024, they plan to internalize the plant for their own products. In light of that, Scholar Rock implemented a plan to add an additional US-based fill, finish facility.

接下來，我想談談如何為我們的供應鏈增加冗餘。2024 年 12 月，諾和諾德收購了布魯明頓工廠，計劃將工廠改造成自有產品生產基地。有鑑於此，Scholar Rock 實施了一項計劃，在美國增設一家灌裝和成品加工廠。

Now with the OAI classification, Scholar Rock has accelerated our timeline for an additional biller. We have selected a world-class commercial facility that has a proven track record and has successfully completed recent site inspections, including with the FDA and EMA. As you know, one of the bottlenecks to rapidly adding a new vialer is securing commercial capacity. This can be a lengthy process. Importantly, we have secured commercial capacity commencing in the first quarter of 2026, and tech transfer is now underway.

現在有了 OAI 分類，Scholar Rock 加快了我們新增計費器的進度。我們選擇了一家世界一流的商業設施，該設施擁有良好的業績記錄，並已成功完成近期的現場檢查，包括 FDA 和 EMA 的檢查。如您所知，快速增加新藥瓶的瓶頸之一是確保商業產能。這可能是個漫長的過程。重要的是，我們已確保從 2026 年第一季開始的商業產能，技術轉移目前正在進行中。

We anticipate submitting an sBLA for this facility later in 2026. In summary, we will continue to work with urgency to bring this important medicine to the SMA community. We look forward to providing clarity on resubmission timelines as soon as we are able.

我們預計將於 2026 年稍後為該設施提交 sBLA 申請。總之，我們將繼續全力以赴，盡快將這種重要的藥物帶給 SMA 患者族群。我們期待盡快就重新提交的時間表提供明確的資訊。

In addition to the large opportunity we have to serve children and adults with SMA, we continue to strategically advance our pipeline. This includes the Phase II OVAL study progressing apitegromab in a second rare debilitating neuromuscular disorder as well as advancing SRK-439 into the clinic.

除了我們為患有脊髓性肌肉萎縮症 (SMA) 的兒童和成人提供服務的巨大機會外，我們還將繼續策略性地推進我們的人才儲備。這包括推進 apitegromab 在第二種罕見的衰弱性神經肌肉疾病的 II 期 OVAL 研究，以及推進 SRK-439 進入臨床階段。

Akshay will provide additional detail on these activities shortly. Importantly, to reach our ambitions, I am pleased to opportunistically strengthened our balance sheet during the third quarter, and we continue to operate with a tight financial plan. which Vikas will discuss later in the call. This plan is aligned to thoughtful strategic investments to drive long-term value creation.

阿克謝稍後將提供有關這些活動的更多細節。重要的是，為了實現我們的目標，我很高興在第三季抓住機遇，加強了我們的資產負債表，我們將繼續嚴格執行財務計劃。 Vikas 稍後將在電話會議中對此進行討論。該計劃與深思熟慮的策略投資一致，旨在推動長期價值創造。

We remain confident in the strength of our strategy, the grid of our team and the transformative potential of apitegromab and our pipeline. The regulatory challenges we face today are temporary, but the opportunities ahead to serve patients are extraordinary.

我們對我們的策略實力、團隊架構以及apitegromab和我們的研發管線的變革潛力仍然充滿信心。我們今天面臨的監管挑戰是暫時的，但未來服務病患的機會卻是巨大的。

With that, I'll turn the call over to Akshay to provide more detailed update on our R&D progress. Akshay?

接下來，我將把電話交給 Akshay，讓他詳細介紹我們的研發進度。阿克謝？

Thank you, David, and good morning, everybody. As David noted, we continue to work with urgency to bring a bit of amount to children and adults with SMA as quickly as possible. SMA is a rare severe neuromuscular disease resulting in irreversible loss of muscular and progressive muscle wasting that diminishes the independence of both children and adults. Apitegromab has the potential to reverse the trajectory of SMA from a loss of motor function to a game of mode.

謝謝你，大衛，大家早安。正如大衛所指出的，我們正繼續加緊努力，盡快為患有 SMA 的兒童和成人提供一些幫助。SMA 是一種罕見的嚴重神經肌肉疾病，會導致肌肉不可逆的喪失和進行性肌肉萎縮，從而降低兒童和成人的獨立生活能力。Apitegromab 有可能逆轉 SMA 的病程，使其從運動功能喪失轉變為遊戲模式。

As demonstrated in the Phase III SAPPHIRE study, underscoring the importance of the potential benefit of this therapeutic. I'd now like to turn to Wednesday's Type A meeting. I was pleased to lead our team at that meeting for Peter. A Baleno, the meeting was in person and included the relevant leaders and decision-makers from the agency, including the neurology division and the Oxotecompliance. Team was joined by Kenneth Hobby, President of Cure SMA and representatives from Novo Nordisk.

如 III 期 SAPPHIRE 研究所示，凸顯了此療法的潛在益處的重要性。現在我想談談週三的A型人會議。我很榮幸能代表彼得帶領我們的團隊參加那次會議。巴萊諾表示，會議是面對面進行的，與會者包括該機構的相關領導和決策者，包括神經科和 Oxotecompliance 部門。Cure SMA 主席 Kenneth Hobby 和 Novo Nordisk 的代表也加入了團隊。

The meeting was constructive and collaborative. We reviewed the comprehensive data from the item apitegromab development program, including the Phase II TOPAS study, which demonstrated that delayed treatment results in suboptimal motor function outcomes. PAUSE These data underscore the impact of delayed treatment and the urgency to make apitegromab available to the SMA community.

會議富有建設性，合作性強。我們回顧了 apitegromab 計畫開發計畫的綜合數據，包括 II 期 TOPAS 研究，該研究表明延遲治療會導致運動功能結果不理想。暫停 這些數據凸顯了延遲治療的影響，以及讓 SMA 患者群體獲得 apitegromab 的迫切性。

At the time of meeting, it was clear that the CRL we received on September 22, was based solely on the need of the Bloomington facility to be in compliance with CGMP or current good manufacturing practice regulations. During the meeting, never there, there's detail the progress they have made in implementing a robust remediation plan at the Lovington facility.

會議召開時，我們清楚地認識到，9 月 22 日收到的 CRL 完全是基於布魯明頓工廠需要符合 CGMP 或現行良好生產規範法規這一原因。在會議期間，從未有人詳細介紹過他們在洛文頓工廠實施強有力的補救計劃所取得的進展。

Novo Nordics also shared with the FDA that did expect the facility to be ready for the inspection by the end of the year. We remain in close coordination with Neonode as we await the minutes from the Type A meeting. After Novo's completion of remediation of the Bloomington facility and a site inspection by the FDA we anticipate recommission of the BLA and US launch following approval of apitegromab in 2026.

諾和諾德公司也向美國食品藥物管理局表示，預計該工廠將在年底前做好接受檢查的準備。在等待A類會議紀要的同時，我們將繼續與Neonode保持密切協調。在諾和諾德完成布魯明頓工廠的整改以及FDA的現​​場檢查後，我們預計在2026年apitegromab獲得批准後，將重新啟動BLA並在美國上市。

As part of our long-term growth plans to serve patients around the world with apitegromab we're also accelerating time lines to bring a second fill-finish facility online. This process requires rigorous validation and regulatory approval to ensure the same quality, safety, and efficacy of the drug product.

作為我們長期發展計畫的一部分，為了向世界各地的患者提供 apitegromab，我們也正在加快第二個填充包裝設施的上線速度。該過程需要嚴格的驗證和監管批准，以確保藥品的品質、安全性和有效性。

Importantly, we have secured commercial capacity commencing in the first quarter of 2026 and anticipate submitting an sBLA for the second facility later in 2026. Outside of the US, we continue to expect a decision from the EMA on our apitegromab Marketing Alteration Application, or MAA, near the middle of next year.

重要的是，我們已確保從 2026 年第一季開始的商業產能，並預計將於 2026 年稍後為第二個設施提交 sBLA。在美國以外，我們仍然預計歐洲藥品管理局 (EMA) 將於明年年中左右對我們的 apitegromab 上市變更申請 (MAA) 做出決定。

Further to our commitment to a broad SMA community we announced today that we've initiated dosing in our Phase II OPL trial evaluating apitegromab in intent and probes under the age of two. The cloud is enrolling participants have been treated with an SMN1 pilot gene therapy or who are receiving treatment with an approved SMN2-targeted therapy.

為了進一步履行我們對廣大 SMA 患者群體的承諾，我們今天宣布，我們已啟動 II 期 OPL 試驗的給藥，該試驗旨在評估 apitegromab 在兩歲以下兒童中的應用和療效。該雲端平台正在招募已接受 SMN1 試驗基因療法治療或正在接受已批准的 SMN2 標靶療法治療的參與者。

It is designed to investigate two different doses of apitegromab for a duration of 48 weeks and will assess PK, PD, efficacy and safety. In the open study, early intervention with apitegromab could support muscle during the critical earning development phase, complementing Mentaterapy that aim to preserve merge neurals.

該研究旨在調查兩種不同劑量的阿匹替格羅單抗，療程為 48 週，並將評估其藥物動力學、藥效學、療效和安全性。在開放研究中，早期使用 apitegromab 進行幹預可以在關鍵的學習發展階段支持肌肉，補充旨在保護融合神經元的 Mentaterapy。

Emerging muscle growth when merchants muscles are still forming a bit of a apitegromab has a unique opportunity to improve motor outcomes in young children with SMA. Beyond SMA, we're on track to initiate clinical development activities for apitegromab in a second neuromuscular disorder by year-end. We plan to provide additional information on the disease and the clinical development strategy in early 2026.

當肌肉還在發育初期，肌肉生長就已經開始，而阿匹替格羅單抗為改善 SMA 兒童的運動能力提供了獨特的機會。除了 SMA 之外，我們預計在年底前啟動 apitegromab 治療第二種神經肌肉疾病的臨床開發活動。我們計劃在 2026 年初提供有關該疾病和臨床開發策略的更多資訊。

And finally, we continue to advance our world-leading Antimo staffing platform beyond apitegromab. The FDA has cleared the IND for SRK 149, and we're on track to initiate a Phase I study in healthy volunteers before the end of this year. This program is built on the validated approach that delivered apitegromab.

最後，我們將繼續推進我們世界領先的 Antimo 人員配備平台，使其超越 apitegromab。FDA 已批准 SRK 149 的 IND 申請，我們預計在今年年底前啟動針對健康志願者的 I 期研究。此方案建立在已驗證的阿匹替格羅單抗給藥方法之上。

Specifically, 149 was designed to be an innovative, subcutaneously administered myostatin inhibitor binding to both pro and latent myostatin with high affinity and selectivity. Based on prepoceldata, 39 has the potential to potently inhibit my staffing and increased muscle mass. We expect to have data from the south portion of the Phase I study in 2026.

具體來說，149 被設計成一種創新的皮下注射肌肉生長抑制素抑制劑，能夠以高親和力和選擇性與前體肌肉生長抑制素和潛在肌肉生長抑制素結合。根據 prepoceldata，39 有可能有效抑制我的肌肉生長和肌肉量增加。我們預計將於 2026 年獲得第一階段研究南部部分的數據。

In summary, our focus remains on bringing apitegromab to patients in two patients and investing with financial discipline to deliver on the promise of our broader pipeline. The strength of our data and the momentum across our programs gives us confidence in the impact we can deliver.

總而言之，我們的重點仍然是為兩名患者帶來 apitegromab，並以財務紀律進行投資，以實現我們更廣泛的研發管線的承諾。我們強大的數據和各項計劃的良好勢頭，讓我們對能夠產生的影響充滿信心。

Now at this point, I'll turn the call over to Keith to discuss our commercial launch strategy and planning. Keith?

現在，我將把電話交給基思，讓他來討論我們的商業發布策略和計劃。基思？

Thanks, Akshay. The SMA community is demanding more. Even with currently available treatments, they need a treatment that directly addresses progressive muscle wasting. Apitegromab demonstrated that ability in our Phase III SAPPHIRE study, and we will be ready to deliver apitegromab to the SMA community upon approval. This is not a matter of if, but when.

謝謝你，阿克謝。SMA 患者群體提出了更高的要求。即使採用目前已有的治療方法，他們仍需要一種能夠直接解決進行性肌肉萎縮問題的治療方法。Apitegromab 在我們的 III 期 SAPPHIRE 研究中證明了這種能力，一旦獲得批准，我們將準備好向 SMA 患者群體提供 apitegromab。這並非是否會發生的問題，而是何時發生的問題。

Our understanding for the demand of opidagrumab and our confidence in its potential to address the unmet need for children and adults with SMA continues to strengthen. As we look at SMA globally, nearly a decade following the launch of the first SMN targeted therapy, the demand for treatment continues to grow. After the first three quarters of 2025, annual revenue for current SMA treatments are trending to approximately $5 billion globally with the continued growth of SMN targeted therapies the need for the world's first muscle-targeted therapy is greater than it has ever been before.

我們對 opidagrumab 的需求的理解以及我們對其滿足 SMA 兒童和成人未滿足需求的潛力的信心不斷增強。從全球視野來看，在首個SMN標靶療法推出近十年後，SMA的治療需求仍在持續成長。2025 年前三個季度之後，目前 SMA 治療的年收入在全球將達到約 50 億美元。隨著 SMN 標靶療法的持續增長，對世界上首個肌肉標靶療法的需求比以往任何時候都更加迫切。

Our small, lean and highly experienced US customer-facing team is active in the field and we are using this additional time to enhance our engagement activities and to strengthen our performance against key prelaunch readiness metrics.

我們規模小、精幹且經驗豐富的美國客戶服務團隊活躍在一線，我們正在利用這段額外的時間來加強我們的互動活動，並提高我們在關鍵的發布前準備指標方面的表現。

As a reminder, we are just under four months in to our pre-commercial field deployment, whereas most biotech companies typically benefit from a longer runway prior to approval. Nationwide, there are approximately 140 SMA treatment centers and more than 2,600 SMA prescribing physicians. With this additional time, we are working to both broaden and deepen our engagement with these potential prescribing physicians.

提醒大家，我們目前處於商業化前現場部署階段，至今還不到四個月，而大多數生技公司在獲得批准前通常會有更長的準備時間。全國約有 140 個 SMA 治療中心和 2600 多名 SMA 處方醫生。利用這段額外的時間，我們正在努力擴大和深化與這些潛在處方醫生的聯繫。

However, an SMA patient is not just treated by one of these physicians but by a broader cross-specialty SMA treatment team. This team can include physical therapy, homonology, orthopedics and more. This additional time is enabling us to better understand the patient journey and the roles of the SMA treatment team in each of these 140 treatment centers and how they influence patient care.

然而，SMA 患者並非僅由其中一位醫生治療，而是由更廣泛的跨學科 SMA 治療團隊進行治療。這個團隊可以包括物理治療、順勢療法、骨科等等。這段額外的時間使我們能夠更好地了解患者的治療歷程，以及 SMA 治療團隊在這 140 個治療中心中的角色，以及他們如何影響患者的治療。

Additionally, our market access team is expanding their focus beyond that of national payers to also include top regional payers. This builds on our ambition that any patient with SMA who can benefit from apitegromab should have access to apitegromab.

此外，我們的市場准入團隊正在擴大其關注範圍，從全國性支付方擴展到包括主要的區域性支付方。這體現了我們的願景，任何能從阿匹替格羅單抗治療中獲益的 SMA 患者都應該有機會獲得阿匹替格羅單抗。

Furthermore, our unwavering commitment to the SMA patient community continues by a partnership at a local and national events and to educate on the importance of targeting muscle. We are deepening our collaboration with the accuracy groups, and we are also building lasting relationships, one patient, one caregiver, one family at a time. In Europe, our efforts continue to drive SMA education and awareness, laying the groundwork to ensure we reach patients efficiently across key markets.

此外，我們對 SMA 患者群體的堅定承諾仍在繼續，我們透過參與地方和全國性活動以及進行肌肉靶向治療重要性教育活動來實現這一目標。我們正在深化與精準醫療團隊的合作，我們正在與每位病患、每位照護人員、每個家庭建立持久的關係。在歐洲，我們繼續努力推動 SMA 的教育和宣傳，為確保我們能夠有效率地接觸到主要市場的患者奠定基礎。

Our opportunity to serve patients around the world in SMA is significant. There are an estimated 35,000 people with SMA who have received an SMN targeted therapy and who could be eligible for treatment with apitegromab -- we are making strategic disciplined investments in our launch infrastructure, and we will be ready to execute rapidly once apitegromab is approved.

我們有機會為世界各地的脊髓性肌肉萎縮症（SMA）患者提供服務，這意義重大。據估計，有 35,000 名患有 SMA 的患者接受了 SMN 標靶治療，他們可能符合接受 apitegromab 治療的條件——我們正在對我們的上市基礎設施進行戰略性的、有紀律的投資，一旦 apitegromab 獲得批准，我們將做好迅速執行的準備。

In short, we are ready the strategy is clear. The team is in place and our commitment to the SMA community has never been stronger.

總之，我們已經準備就緒，戰略很明確。團隊已經到位，我們對脊髓性肌肉萎縮症 (SMA) 患者群體的承諾從未如此堅定。

Now I will turn the call over to Vikas. Vikas?

現在我將把電話交給維卡斯。維卡斯？

Thank you, Keith. Our overarching objectives are to fund our R&D activities to expand our leadership in the myostatin and muscle space to support a strong commercial launch and to extend our runway to meet our eventual timelines for apitegromab approval. In line with these objectives, I'm pleased to provide our third quarter financial results and to discuss our approach to managing our cash runway and investment prioritization moving forward.

謝謝你，基斯。我們的總體目標是為研發活動提供資金，以擴大我們在肌肉生長抑制素和肌肉領域的領先地位，從而支持強勁的商業上市，並延長我們的資金儲備，以滿足我們最終獲得 apitegromab 批准的時間表。為了實現這些目標，我很高興向大家介紹我們第三季的財務業績，並討論我們未來在現金流管理和投資優先事項方面的方法。

Turning first to our third quarter results. We ended the third quarter with $369.6 million in cash and cash equivalents. For the quarter, we reported $103 million in operating expenses which includes $18.3 million in noncash stock-based compensation. Excluding stock-based compensation, operating expenses were $85.3 million, which reflects ongoing investments in infrastructure to support apitegromab regulatory approval, commercial readiness, and our clinical pipeline.

首先來看我們第三季的業績。第三季末，我們持有現金及現金等價物3.696億美元。本季度，我們報告的營運支出為 1.03 億美元，其中包括 1,830 萬美元的非現金股票選擇權費用。不包括股票選擇權費用，營運支出為 8,530 萬美元，這反映了為支持 apitegromab 的監管批准、商業準備和我們的臨床研發管線而對基礎設施進行的持續投資。

During the third quarter, we strengthened our balance sheet, adding $141.7 million. This cash came from two sources. First, we executed our ATM and sold approximately 2.8 million shares, which resulted in net proceeds of $91.7 million.

第三季度，我們加強了資產負債表，增加了 1.417 億美元。這筆資金來自兩個來源。首先，我們執行了自動增發計劃，出售了約 280 萬股股票，淨收益為 9,170 萬美元。

And second, we drew down $50 million from our existing debt facility. As we await apitegromab approval, we continue to operate with a tight financial plan focused on potful capital allocation to advance our clinical pipeline and strategic investments to support commercial readiness.

其次，我們從現有債務融資中提取了 5,000 萬美元。在等待 apitegromab 批准的同時，我們繼續執行嚴格的財務計劃，重點在於充足的資金配置，以推進我們的臨床研發管線和策略性投資，從而支持商業化準備。

Accordingly, we have adjusted our go-forward operating plan. We have deferred investments across a number of areas, including new hiring, launch expenses that are gated to approval, certain R&D activities, including a third indication for apitegromab and other discretionary spend.

因此，我們調整了未來的營運計劃。我們推遲了多個領域的投資，包括新員工招募、需要獲得批准才能進行的上市費用、某些研發活動（包括 apitegromab 的第三個適應症）以及其他可自由支配的支出。

Now I'll turn to the six prioritized investments we are making. The acceleration of a second fill, finish facility for apitegromab SME commercial launch readiness, Onex apitegromab extension study, the Phase II OPL study, the second indication for apitegromab and the commencement of SRK-439's clinical development.

現在我將介紹我們正在進行的六項優先投資。加快建造第二個填充、完成設施，為 apitegromab SME 商業上市做好準備；開展 Onex apitegromab 擴展研究、II 期 OPL 研究、apitegromab 的第二個適應症以及啟動 SRK-439 的臨床開發。

Turning to our balance sheet. Our current cash balance is $369.6 million, which we expect to be augmented by approximately $60 million in cash from the exercise of outstanding common warrants by year-end. With this, we expect our cash to be sufficient to fund operations into 2027. This cash runway has conservative assumptions and does not reflect any upside from potential sales of apitegromab or a priority review voucher.

接下來來看看我們的資產負債表。我們目前的現金餘額為 3.696 億美元，預計到年底，透過行使未行使的普通認股權證，現金餘額將增加約 6,000 萬美元。有了這些資金，我們預計我們的現金足以支持營運到 2027 年。該現金儲備基於保守假設，並未反映出阿匹格羅單抗潛在銷售或優先審評券帶來的任何收益。

To further strengthen our balance sheet, we intend to expand our credit facility while preserving our nondilutio financing options. We will provide further clarity on this as well as our anticipated operating expenses for 2026 during our fourth quarter earnings call. Scholar Rock continues to operate from a position of financial strength with a disciplined approach to capital allocation and a clear focus on supporting our strategic priorities.

為了進一步加強我們的資產負債表，我們打算擴大信貸額度，同時保留我們的非稀釋性融資選擇。我們將在第四季財報電話會議上對此以及我們預計的 2026 年營運支出作進一步說明。Scholar Rock 憑藉著雄厚的財務實力，採取嚴謹的資本配置方法，並明確專注於支持我們的策略重點，繼續穩步發展。

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

這樣，我就把電話轉回給大衛了。大衛？

Thanks, Vikas. In closing, Scholar Rock remains focused on near-term execution while building with financial discipline for the future. Our conviction, apitegromab and in our broader strategy is stronger than ever, and we are moving with urgency and purpose to deliver meaningful impact for patients. Our priorities are clear. execute with urgency to bring apitegromab, the world's first and only muscle targeted treatment that improves motor function to children and adults living with SMA as rapidly as possible.

謝謝你，維卡斯。最後，Scholar Rock 將繼續專注於近期業績，同時以嚴謹的財務紀律為未來發展奠定基礎。我們對阿匹格羅單抗以及我們更廣泛的策略的信念比以往任何時候都更加堅定，我們正以緊迫感和目標感採取行動，為患者帶來有意義的影響。我們的首要任務很明確：以最快的速度將全球首個也是唯一一個能夠改善脊髓性肌肉萎縮症（SMA）兒童和成人運動功能的肌肉標靶治療藥物apitegrob帶給他們。

Advanced epitogramab development activities in the second rare debilitating neuromuscular disease, and that will be followed by additional indications where we can have a transformative impact for patients. We want to progress SRK 439 into the clinic and continue to invest in our future with discipline to support these high-value initiatives.

針對第二種罕見的致殘性神經肌肉疾病，我們進行了先進的 epitogramab 開發活動，隨後將拓展到其他適應症，以便為患者帶來變革性的影響。我們希望推進 SRK 439 進入臨床階段，並繼續以嚴謹的態度投資未來，以支持這些高價值的措施。

Before I close, I want to share my sincere appreciation for Cure SMA and the SMA patient community. Over these past weeks, I have had the opportunity to meet with many individuals and families living with SMA. And the words of support that have been shared with us and with me directly have been tremendously meaningful as we work harder, better, and faster to bring this impactful medicine to those who can benefit.

在結束之前，我想表達我對 Cure SMA 和 SMA 患者群體的誠摯感謝。在過去的幾周里，我有機會與許多患有脊髓性肌肉萎縮症 (SMA) 的個人和家庭見面。在我們更努力、更有效率、更迅速地將這種有影響力的藥物帶給需要的人的過程中，大家給予我們的支持和鼓勵意義非凡。

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

接下來，我們將開放提問環節。操作員？

(Operator Instructions)

（操作說明）

Mani Faroohar, Leerink Partners.

Mani Faroohar，Leerink Partners。

Congrats on the progress through what's been obviously a choppy period for everyone in the government. I think a couple of quick questions. I know I'm violating one question. One, in terms of thinking about further financing opportunities to top up the tank as necessary, how do you think about debt versus royalty equity? Like how do you think about relative cost of capital and what's the most appropriate use once you get to a launch.

恭喜你們在政府經歷這段顯然充滿波折的時期後所取得的進展。我想問幾個簡單的問題。我知道我違反了一個問題。第一，在考慮必要時進一步融資以補充資金時，您如何看待債務與特許權使用費股權？例如，你如何考慮資金的相對成本，以及一旦產品上市，最適合的用途是什麼。

And then another commercial question. In the early days of launch, it is probable that you will be transitioning from one facility to another. To what extent does that introduce any operational risk going from products from one to so to another? And how can that be addressed ahead of time by you guys now?

然後又問了一個商業問題。在專案啟動初期，您很可能需要從一個設施過渡到另一個設施。產品從一種到另一種的轉變，在多大程度上會引入任何營運風險？你們現在該如何提前解決這個問題呢？

Thanks, Mani. Why don't we take the first question first with the cost on the financing options and then I'll come back on the redundancy of supply chain with Phil finish. Vikas?

謝謝你，馬尼。我們先來回答第一個問題，也就是融資方案的成本，然後再由我和菲爾一起討論供應鏈冗餘的問題。維卡斯？

Yes. Thank you, David. Man, our first objective here is to bridge the financing until the approval. And the first path to go from the lowest cost of capital is to take additional -- extend our loan facility a little bit more. We are in discussions with that.

是的。謝謝你，大衛。老兄，我們目前的首要目標是籌集到資金，直到獲得批准。而從最低資本成本入手的第一條途徑是增加－稍微延長我們的貸款期限。我們正在與相關方面進行磋商。

That will be our first opportunity. Royalty probably comes next, and it goes too long, and then we have to take a little bit of equity, that will be the last and the most expensive one, which we are trying to avoid at any cost is trying to get it as more non-dilutive first. Does that answer your question?

那將是我們的第一個機會。接下來可能是版稅，而且時間太長，然後我們不得不接受一些股權，這將是最後也是最昂貴的，我們無論如何都要避免這種情況，而是首先努力使其更具非稀釋性。這樣回答了你的問題嗎？

Yes. So a follow-up. That would imply that, relatively speaking, we should expect you guys to wind down use ATM much less going forward. Like how does that fit into the strategy?

是的。所以，有個後續問題。這意味著，相對而言，我們應該預期你們今後會大幅減少使用 ATM 機。這如何融入戰略中？

Yes. Obviously, our first objective is to work with the loan facility and expand upon that. And ATMs are put in place just to take some small augmentation of the capital at an opportunistic view. And we did take it down in the last quarter. Because we are only $50 million loan facility available.

是的。顯然，我們的首要目標是利用貸款機制並在此基礎上拓展。設定自動櫃員機只是為了以投機取巧的方式攫取一些額外的資金。我們在上個季度成功將其擊敗。因為我們只有5000萬美元的貸款額度。

We're expanding that loan facility as we discussed. And as soon as we have the new facility in place, will share it with all of you.

正如我們之前討論的那樣，我們正在擴大貸款額度。新設施一建成，我們就會和大家分享。

And then, Mani, regarding the second vial, a couple of bottlenecks I've shared with you and others in the past. One of the big ones I highlighted in the call was obviously finding a commercial line that is available, that is -- got the ideal configuration for our vial -- and our team under the leadership of Lisa Wyman, our Chief Tech and Quality Officer just did an extraordinary job in accelerating our second Vilar progress to secure commercial capacity in Q1 and commenced tech transfer in lightning speed since that middle of October time line when the OAI hit, we hope that, that was really important.

還有，Mani，關於第二個小瓶，我之前已經和你以及其他人分享過一些瓶頸問題。我在電話會議中重點強調的一點顯然是找到一條可用的商業生產線，也就是說，找到了適合我們小瓶的理想配置。在首席技術和品質長 Lisa Wyman 的領導下，我們的團隊出色地加快了第二代 Vilar 的研發進度，確保了第一季的商業產能，並且自 10 月中旬 OAI 生效以來，以閃電般的速度開始了技術轉移。我們希望這一點真的非常重要。

Now to get there and to get there quickly, you want to change as little as possible in your second vial as in your primary vialer, whether or not it's vial configuration, analytical testing, like -- so that actually helps you with speed as well.

為了盡快達到目標，你需要在第二個樣品瓶中盡可能少地改變主樣品瓶，無論是樣品瓶配置、分析測試等等——這實際上也有助於提高速度。

And those are the things that we'll be focused on. And then the impact in the marketplace really should be almost seamless whether or not we are distributing our apitegromab from the Bloomington facility or the new second vialer, it should really be quite seamless operationally to the marketplace, and we would expect it to be that way.

這就是我們將要關注的重點。那麼，無論我們是從布魯明頓工廠還是從新的第二瓶中分發 apitegromab，對市場的影響都應該幾乎是無縫的，從運營上到市場，都應該非常無縫，我們也希望情況確實如此。

Eric Schmidt, Cantor.

埃里克·施密特，坎托爾。

Congrats on the progress as well. David and team, for those of us who've kind of been reading the gory play by play around the Catalent facility in Bloomington and know some of the prior history and all the past issues. How do you -- how do you how to provide confidence that this remediation effort is on good footing and that the inspection will prove positive? And then maybe secondarily, do you expect that to be a Class I or Class II acceptance for the resubmission.

恭喜你的進展。對於我們這些一直在關注 Catalent 位於布魯明頓的工廠發生的血腥事件，並且了解一些先前歷史和所有過去問題的人來說，David 和他的團隊。你如何——你如何才能讓人相信這項補救工作進展順利，並且檢查結果會是正面的？其次，您認為重新提交的申請會被評為一級還是二級接受？

Thanks, Eric. And no doubt, there is a history in the facility. We think the history is really anchored around the quality system, the quality culture, and the facility. I think importantly, it largely links back to ownership that did not include sort of the steady hand of Novo Nordisk and their commitment to quality and compliance. And so one of the things that I've been saying often, and certainly, the gory details are gory, right?

謝謝你，埃里克。毫無疑問，這座建築有著悠久的歷史。我們認為，公司的歷史真正根植於品質體系、品質文化和工廠。我認為，更重要的是，這在很大程度上與所有權有關，因為所有權中沒有諾和諾德這樣的穩健領導者，也沒有他們對品質和合規性的承諾。所以，我常說的一件事是，當然，血腥的細節確實很血腥，對吧？

We got the observations, and we notified you all of those observations back on August 6, our last earnings call, which feels like a lifetime away now. And then we've kind of been riding through the CRL and the OAI. But what we have had a front row seat to is the collaboration with Novo, the commitment from the top of the organization, the changes in the staff that they are making.

我們收到了這些觀察結果，並在 8 月 6 日的上次財報電話會議上通知了大家，現在感覺好像已經是很久以前的事了。然後我們就一路經過了 CRL 和 OAI。但我們有幸近距離見證了與 Novo 的合作、公司高層的承諾以及他們正在進行的員工變動。

The integration of the Novo quality system into that facility and then the substantial progress that they've been making on a robust remediation plan, which as they noted to the FDA on Wednesday, they feel like there -- the facility is going to be reinspection ready by the end of this year.

諾和諾德的品質系統已融入該工廠，而且他們在強有力的整改計劃方面取得了實質進展。正如他們週三向FDA指出的那樣，他們感覺工廠將在今年年底前做好重新檢查的準備。

We don't think that Novo takes that lightly. We think that they are going through a series of internal exercises to make sure that they are reinspection ready. We would imagine that they'll continue to communicate with the FDA and gather feedback on what might be missing from their remediation plan that they would then need to tweak before any reinspection would take place, but we are surely been pleased with the seriousness and the urgency that from the top of that organization right through that facility, they are taking the remediation plan.

我們認為諾和諾德不會輕視這件事。我們認為他們正在進行一系列內部演練，以確保他們已做好重新檢查的準備。我們認為他們會繼續與 FDA 溝通，收集回饋意見，以了解他們的整改計畫中可能缺少哪些內容，以便在重新檢查之前進行調整。但我們非常高興地看到，從組織的最高層到整個工廠，他們都認真對待整改計劃，並展現出緊迫感。

Regarding Class I or Class II, I'll turn that over to Akshay for his thoughts as he was presiding over our team in person in Befesa, Wednesday, and the team just did a fantastic job. Akshay?

關於一級還是二級，我會把這個問題交給 Akshay 來回答，因為他週三在 Befesa 親自主持了我們團隊的會議，團隊的表現非常出色。阿克謝？

Thanks, David. Eric, I just want to reiterate that it was a very constructive and collaborative meeting. And I think the agency, as you might expect, shares, the need for urgency as we all work together to try and get apitegromab to patients. So it's not for us obviously to second gas table to be Class 1 or Class 2. But we were very heartened by the comments they made and the approach they committed to committed to help detect the license patients.

謝謝你，大衛。艾瑞克，我只想重申，這是一次非常有建設性和合作性的會議。正如你所料，我認為該機構也認同這種緊迫性，因為我們都在共同努力，爭取讓患者能夠盡快獲得阿匹替格羅單抗。所以，顯然我們無權決定天然氣表是 1 類還是 2 類。但他們發表的評論以及他們承諾幫助發現持證患者的做法，讓我們深受鼓舞。

So we need to work with Novo to get their work done. Let's wish them the best get to slight reinspected, resubmit the BLA and I'm confident the agency is going to act with earns and commitment to this community of patients we have always shown when it comes to us.

所以我們需要與Novo合作，才能讓他們完成工作。讓我們祝福他們一切順利，能夠進行輕微的複檢，重新提交生物製品許可申請（BLA），我相信該機構會像我們一直以來對待患者群體那樣，以應有的信譽和承諾來對待他們。

Tessa Romero, JPMorgan.

Tessa Romero，摩根大通。

David and team, thanks so much for the question here. So to be clear, the BLA that you plan to submit in 2026 for pmab will include Catalent as your primary fill finish and you plan to file the SBLA for the additional sale finish facility later in 2026 following the potential approval of the BLA. Why is that the right path versus using an additional fill, finish only?

David 和團隊，非常感謝你們提出這個問題。明確地說，您計劃在 2026 年提交的 pmab 的 BLA 將包括 Catalent 作為您的主要灌裝完成機構，並且您計劃在 BLA 可能獲得批准後，於 2026 年晚些時候提交額外的銷售完成機構的 SBLA。為什麼這種方法比只進行額外填充和收尾處理好？

And then a follow-up is just on the EMA review. How is that going with respect to manufacturing-related items.

然後，後續報導將針對EMA審查。製造業相關專案進度如何？

Thanks, Jess. Yes. I mean I think given where we are, given the tone and tenor of the meeting that Akshay presided over this week and again, the progress that Novo has been making which really enabled them to communicate to the FDA that they are on track to be reinspection ready by the end of this year. We just think that that is the absolute right path for us. We would expect that our BLA would be resubmitted with Catalent as our primary filler.

謝謝你，傑西。是的。我的意思是，我認為鑑於我們目前的處境，鑑於 Akshay 本週主持的會議的基調和氛圍，以及 Novo 取得的進展，這使得他們能夠向 FDA 傳達，他們正按計劃在今年年底前做好重新檢查的準備。我們只是認為這才是對我們來說絕對正確的道路。我們預計，我們將以 Catalent 作為主要填充劑重新提交我們的生物製品許可申請 (BLA)。

And we would expect a second pillar to be added to our file, which was frankly always going to be our plan anyway given the fact that Novo wants that facility for internal purposes. And so that is the path that we are following, obviously, everything that we are doing to accelerate our second vialer is a great insurance policy for us no matter what would happen and I was really gratified by our team's efforts over the course of just the past month with the major progress that they have made to secure commercial capacity at a second boiler and already have tech transfer underway, and of course, we'll be expecting the commencement of our commercial capacity to be leveraged beginning in Q1 of 2026. Regarding the EMA, I'd love to have Akshay comment on that. Obviously, quality and compliance is important to all regulators, including them.

我們預計會在我們的文件中增加第二個支柱，坦白說，考慮到 Novo 希望將該設施用於內部用途，這本來就是我們的計劃。所以，這就是我們正在遵循的道路。顯然，我們為加速第二個小瓶生產所做的一切，無論發生什麼，都是我們的一個很好的保險措施。在過去一個月裡，我們的團隊取得了重大進展，確保了第二個鍋爐的商業產能，並且技術轉移已經開始，我對此感到非常欣慰。當然，我們預計我們的商業產能將於 2026 年第一季開始投入使用。關於EMA，我很想聽聽Akshay對此發表一些評論。顯然，品質和合規性對所有監管機構（包括他們自己）都很重要。

Indeed. And just to review the status of the MAA, the question, and answers, I go back and forth have proceeded well. So the review continues in exactly the time frame you'd expect. And as we guided on the full comment, we expect a decision by the middle of next year.

的確。為了回顧 MAA 的現狀，問題和答案，我反覆討論，進展順利。所以，審查工作仍在預期的時間範圍內持續進行。正如我們在完整評論中所述，我們預計將在明年年中做出決定。

Now vis-a-vis the Kaplan manufacturing status and the EMA, there is a mutual recognition procedure. And so there is an interdependency. And I think we obviously agree with that. Though I would point out that everything we're doing for the 2026 resubmission, we Novo and all our averages from the 2026 resubmission of our BLA in the US launch following approval by the FDA is in line with supporting our MAA.

目前，關於 Kaplan 的生產狀況和 EMA，有一個相互認可程序。因此，它們之間存在著相互依存的關係。我想我們顯然都同意這一點。不過我想指出的是，我們為 2026 年重新提交申請所做的一切，以及我們 Novo 和我們在 2026 年重新提交的 BLA 申請的所有平均值，在獲得 FDA 批准後，都將支持我們的 MAA。

So there's not much more I can say right now, and obviously, we'll keep you updated. But let's stay, on track and as we proceed we hope that support the proposal.

所以現在我能說的就這麼多了，當然，我們隨時都會向大家更新最新消息。但讓我們繼續保持正軌，我們希望在推進過程中能夠得到大家的支持。

Tazeen Ahmad, Bank of America.

塔津·艾哈邁德，美國銀行。

Thanks for the detailed update. So can I ask when is the latest that you can have this reinspection for Catalent to be completed and given the green light in order to meet your expectations for a 2026 launch? And then just to play the scenarios for a second. And for whatever reason, the second inspection for Catalent doesn't resolve all issues. How quickly could you pivot to make any application with your second sale finish?

感謝您提供的詳細資訊。那麼，請問貴公司最遲何時能夠完成對 Catalent 的重新檢查並獲得批准，以滿足貴公司 2026 年的發布預期？然後就簡單地模擬一下這些場景。不知何故，對 Catalent 的第二次檢查並沒有解決所有問題。你能否迅速調整策略，利用第二次銷售完成的機會提出任何申請？

And how would that impact your timelines for 2026. Like would you be able to switch that BLA filing to a BLA filing and keep the timeline the same as you just mentioned?

那這將如何影響你們2026年的時間安排？例如，您能否將該 BLA 申請更改為 BLA 申請，並保持您剛才提到的時間表不變？

Yes. Thanks, Tazeen. It's again, a good question. Getting back to what we're targeting back to Eric's point, there's a history at the facility. It was under prior ownership.

是的。謝謝你，塔津。這又是一個好問題。回到我們討論的重點，回到艾瑞克剛才提到的，這個設施有著一段歷史。它之前屬於前任所有者。

They've had a few difficult inspections that have led to Form 483s and in this case, some repeat observations. So I understand and we understand that everybody could share some level of concern and, or skepticism that just getting a reinspection is not the objective. It's a successful reinspection.

他們經歷了幾次困難的檢查，導致出現了 483 表格，而且這次還有一些重複的觀察結果。所以我理解，我們也理解，每個人都可能對僅僅進行複檢並不是最終目的感到擔憂或懷疑。這是一次成功的複檢。

And we share with Novo Nordisk that is the objective. And to really put their own team through not only the remediation plan, but rigorous exercises to be reinspection ready, and we know that they are doing that. Related to your point about what if it doesn't resolve all issues, I think there's two ways to look at this to see there still observations in the facility and -- but yet to those observations warrant or not sort of a reclassification of the facility because that's really what we're playing for, a reclassification from OAI to either VAI or NAI.

我們和諾和諾德公司都認同這個目標。而且他們確實讓自己的團隊不僅執行了整改計劃，還進行了嚴格的演練，以做好重新檢查的準備，我們知道他們正在這樣做。關於你提到的“如果它不能解決所有問題怎麼辦”，我認為有兩種看待這個問題的方式：一方面，該設施仍然存在一些觀察結果，但這些觀察結果是否足以證明需要對該設施進行重新分類，因為這才是我們真正想要達到的目標，即從 OAI 重新分類為 VAI 或 NAI。

And for that, certainly, that is what the objective is. I think regarding your timeline, I think a reinspection could technically go pretty well into 2026, and we would still be within a frame of our guidance of resubmitting our BLA and then the US launch upon approval. We're obviously pleased that the tone and tenor of our Type meeting led by Akshay with the FDA with all the key decision makers, all the key groups. I think was constructive.

而這，當然，正是我們的目標。關於您的時間表，我認為重新檢查理論上可以持續到 2026 年，我們仍然會在重新提交生物製品許可申請 (BLA) 並在獲得批准後在美國上市的指導方針範圍內。我們顯然很高興看到由 Akshay 領導的與 FDA 的所有關鍵決策者和所有關鍵團體舉行的類型會議的基調和氛圍。我認為這很有建設性。

It was collaborative. And there really was a shared understanding of the unmet need and a shared understanding that urgency is necessary to serve a very important patient population. And so we're hoping that all of the steps that would be required that gets us to a reinspection would be done in an expeditious way within the regulatory framework that exists and that Novo will do their part. We don't think that they take lightly indicating to the FDA that they will be reinspection ready by the end of this year.

這是合作完成的。大家確實都認同存在未被滿足的需求，也認同必須盡快為非常重要的病患群體提供服務。因此，我們希望所有必要的步驟，以便我們能夠進行重新檢查，都能在現有的監管框架內迅速完成，並且希望諾和諾德能夠盡到自己的責任。我們認為，他們不會輕率地向美國食品藥物管理局表示，他們將在今年年底前做好重新檢查的準備。

We don't think that that's a low bar. We think they're holding themselves. Given the commitment to quality and compliance in the culture of Novo Nordisk, we think they say that with a pretty high hurdle in mind. But back to your question about should like a media right hit that facility. In other words, should the inspection not go well.

我們認為這個標準不算低。我們認為他們還在克制自己。鑑於諾和諾德公司文化中對品質和合規性的承諾，我們認為他們這麼說時心中設定的門檻相當高。但回到你關於媒體是否應該介入該設施的問題。換句話說，如果檢查不順利的話。

Then what role would the second Vilar play? Well, everything that we've done to accelerate that second vilar would be obviously extremely important for us in terms of should we need to pivot, and it's not an addition of this vial on an SBLA, but it's actually our primary resubmission strategy.

那麼第二個維拉爾會扮演什麼角色呢？顯然，我們為加速第二個小瓶的研發所做的一切，對於我們可能需要調整策略的情況來說都極為重要。這並不是在 SBLA 上增加一個小瓶，而是我們主要的重新提交策略。

There are a number of ways that the FDA, and we expect they've done this in the past. And given the shared urgency would understand some level of potential pathways to expedite adding a second biller as your first bilar in the form of a BLA. And everything that we're doing to expedite this process, we think, will aid us in case the impact of the inspection is not what we all expect it to be, which is a successful reinspection.

美國食品藥物管理局（FDA）有很多方法可以做到這一點，我們相信他們過去也曾經這樣做過。鑑於雙方的緊迫性，可以理解透過 BLA 的形式加快添加第二個計費者作為第一個計費者的潛在途徑。我們認為，我們為加快這一進程所做的一切，都將對我們有所幫助，以防檢查的影響不如我們所有人預期的那樣，即重新檢查取得成功。

And as we continue to work with that second biller, we can provide further guidance to you as we progress from tech transfer, which is now underway directly into the filling lines that we will be executing in Q1 and Q2, and we'll provide those updates over time.

隨著我們繼續與第二家結算商合作，我們將從技術轉移（目前正在進行中）直接過渡到我們將在第一季和第二季執行的填充生產線，並隨時向您提供進一步的指導，我們將隨著時間的推移提供這些更新。

Srikripa Devarakonda, Truist Securities.

Sriripa Devarakonda，Truist 證券公司。

Congratulations on all the progress. Thanks for all the details. So in terms of time lines for resubmission of BLA, I feel like we're all asking the same question, but is the plan to wait for the reinspection and for the OAI to be resolved before you submit the BLA and I understand that Novo has said that they're going to be inspection ready by year-end, but could Noble request an inspection and finally, would you be able to address whether Noble hired any outside consultants to help with this process?

祝賀你們取得的所有進展。謝謝你提供的所有細節。關於重新提交BLA的時間安排，我覺得我們都在問同一個問題，但計劃是等到複檢和OAI問題解決後再提交BLA嗎？我知道Novo說過他們會在年底前做好檢查準備，但Noble可以申請檢查嗎？最後，您能否說明Noble是否聘請了任何外部顧問來協助這個過程？

Yes. Maybe related to the timelines on resubmission. I think that Akshay can comment on our thinking and recognizing that with the collaboration with the FDA and the shared urgency, it's a little dynamic. We don't have our Type A meeting minutes yet, but Akshay can share at least our go-forward plan with respect to that.

是的。或許與重新提交的時間安排有關。我認為 Akshay 可以對我們的想法發表評論，並認識到，由於與 FDA 的合作以及共同的緊迫性，情況變得有點動態。我們還沒有A類會議的會議記錄，但Akshay至少可以分享我們在這方面的後續計劃。

Yes. I mean I think base case Chris safe to say that the reaction will have to account would submit after that. But as David said, it is a dynamic situation, and we'll do everything possible to we submit in a fashion to expedite the approval of this growth, which patients need so badly. And we were hardened by the in support from the agency during our tie -- so a lot is going to happen in the coming weeks and early part of next year, and we look forward to resonate.

是的。我的意思是，我認為克里斯的基本情況可以肯定地說，反應必須考慮到這一點，之後他會提交。但正如大衛所說，情況瞬息萬變，我們將盡一切可能提交申請，以加快這項患者急需的新技術的審批。在合作期間，我們得到了機構的支持，這讓我們更加堅定了決心——因此，在接下來的幾周和明年年初，將會發生很多事情，我們期待著產生共鳴。

Well, for sure, Kripa, given that, again, it's reiterated our sole and primary issue is the classification of this facility and their state of compliance. I think it's a safe assumption that we'd like to see that clear. And be ready to go immediately with a resubmission. That's sort of our go-forward plan at this point.

當然，Kripa，鑑於這一點，我們再次重申了我們唯一和主要的問題，即該設施的分類及其合規狀況。我認為，我們希望看到這一點得到澄清，這是一個合理的假設。做好立即重新提交的準備。這就是我們目前的下一步計劃。

And I think it's worth adding, David, that resubmission is really competitive the initial BLA remission neediest much more contained at around just the safety update and the CMC aspects of the file. So it's very -- we're ready to file that resubmission at very short notice.

大衛，我認為值得補充一點，重新提交的競爭非常激烈，最初的 BLA 豁免申請主要集中在安全性更新和 CMC 方面。所以，我們隨時準備在很短的時間內重新提交申請。

Crick, related to the Cnova request of reinspection. I think in a way, they're signaling that they're ready. We would imagine that Novo and the agency still has some wood to chop. Just how do we feel about the remediation plan? Is there anything left before reinspection needs to be done?

Crick，與 Cnova 的重新檢查請求有關。我認為從某種意義上說，他們是在發出準備就緒的訊號。我們認為 Novo 和這家機構還有一些工作要做。我們對補救計畫有何感想？在需要重新檢查之前，還有什麼需要注意的嗎？

I would expect that to be happening, okay? That would be an expectation. But in a way, they put themselves on notice with the FDA that they stand ready to be reinspected toward the end of this year. And we think that, that is really, really important. But as you know, this reinspection will not be announced.

我覺得這種情況應該會發生，好嗎？那是一種預期。但從某種意義上說，他們也向美國食品藥物管理局發出了信號，表明他們已做好準備，在今年年底接受重新檢查。我們認為，這一點真的非常非常重要。但如您所知，這次複檢不會對外公佈。

It would be like your typical unannounced inspection. And so that you can put yourself on notice and communicate with the office of compliance that your reinspection ready. As they notified at our Type A meeting at the end of this year, but we would expect the FDA to when they do reinspect the facility, it would likely be an unannounced inspection.

這就像是一次典型的突擊檢查。這樣您就可以通知合規辦公室，告知他們您的複檢已準備就緒。正如他們在今年年底的 A 類會議上通知我們的那樣，但我們預計 FDA 在重新檢查該設施時，很可能是突擊檢查。

And then your final question about like third parties, I think Novo is really looking very broadly, and they have been working with outside experts and helping them through all of these things, including the remediation plan and the progress with the remediation plan, and we have been pleased with the level of quality and urgency that they are applying to this remediation plan, and we're thankful to them for that.

至於你最後一個關於第三方的問題，我認為 Novo 的考慮範圍非常廣泛，他們一直在與外部專家合作，幫助他們處理所有這些事情，包括補救計劃和補救計劃的進展。我們對他們在這個補救計劃中展現的品質和緊迫性感到滿意，並對此表示感謝。

Etzer Darout, Barclays.

埃策爾·達魯特，巴克萊銀行。

Just because investors have sort of been circling this September 2026 date in terms of sort of timing for a potential approval David. Maybe if you could us understand what could -- maybe the PA minutes unveiled to you on the type of resubmission that you have to make, maybe their time lines around the decision once you have filed -- refiled the BLA?

戴維，投資人一直將 2026 年 9 月這個日期視為潛在批准的時間點。如果您能讓我們了解一些情況，或許就能幫到您——比如，PA會議記錄中向您透露的關於您必須重新提交的材料類型，或者您提交BLA申請後他們的決定時間表？

Yes. Thanks, Azur. It's a great question. And again, something that I think Akshay wanted to have some robust conversations with the agency on our Type A meeting, we're obviously not we don't have meeting minutes in hand, and we certainly want to allow the agency to do their work. But Akshay can comment on how we're thinking about the resubmission timing, and again, whether or not it would be Class I or Class II.

是的。謝謝，Azur。這是一個很好的問題。再次強調，我認為 Akshay 希望在我們的 A 類會議上與代理商進行一些深入的對話，但顯然我們沒有會議記錄，我們當然希望讓代理商進行他們的工作。但 Akshay 可以就我們如何考慮重新提交的時間發表意見，以及它究竟是 I 類還是 II 類。

Yes, as far as the minutes are concerned, I think it's always good to get the minutes in Haneda wrest that we're conveying to you this morning that they're documented in the minute or the progress that Adnovohas made of commitment that everyone is showing to the one of the math, Nova's comment about being ready for reinspection.

是的，就會議記錄而言，我認為最好還是把今天早上我們傳達給你們的羽田摔跤會議記錄下來，這些記錄都記錄在會議紀要中，或者記錄了阿諾沃所做的承諾，每個人都對數學方面所做出的承諾，以及諾瓦關於準備好接受複檢的評論。

And everything we just said so far -- so we await those minutes, and we're confident that they'll reflect what we're conveying this morning. Now as far as the recommission is concerned, we've just discussed that with the last question and, of course, we'll need to be as soon as the inflation is done or earlier, if possible. But we'll be guided by the agency to all of that. And we'll also be given on the review timeline.

我們剛才所說的一切——所以我們等待會議紀要，我們相信紀要會反映我們今天早上所傳達的內容。至於重新撥款的問題，我們剛才在上一個問題中已經討論過了，當然，我們需要在通貨膨脹結束後儘快完成，如果可能的話，最好是更早。但我們會聽從機構的指導來完成所有這些工作。我們也會收到審核時間表。

Now the minutes we get won't sell out the nature of the review timelines for the resubmission. That's not their fact if they wait recommission of the BLA before that. One thing I can tell you is that David emphasized the term and tenor of the meeting that there was support to active open to get on all parts of an pet agency to get this drug to patients as soon as possible.

現在我們獲得的幾分鐘時間不會洩露重新提交的審查時間表的性質。如果他們等到BLA重新成立後再做決定，那他們的說法就不成立了。我可以告訴你們的是，大衛強調了會議的措辭和基調，即支持積極開放地參與寵物機構的各個環節，以便盡快將這種藥物送到患者手中。

Mark Frahm, Cowen.

馬克‧弗拉姆，考恩。

And all the detailed disclosures around this meeting. Maybe in that light, just as you move forward and Nova hopefully is, in fact, in position to be reinspected. Just what do you expect to be able to disclose and kind of on what timeline, particularly given that it isn't even your facility directly, but it is a partner. Will you be able to disclose right when it gets inspected not until maybe some 433 are received? Just what are the disclosure plans there?

以及有關此次會議的所有詳細披露資訊。或許從這個角度來看，就像你繼續前進一樣，而 Nova 實際上也做好了重新檢查的準備。您預計能夠披露哪些信息，以及大概在什麼時間表上能夠披露，尤其考慮到這甚至不是您直接擁有的設施，而是合作夥伴的設施。你們能否在收到檢查報告後立即披露，而不是等到收到一些 433 表格後再披露？具體來說，那裡的資訊揭露計劃是什麼？

Thanks, Mark. I think our disclosure plans will really kind of look in the mirror and focus on us and the things that are material to us that we think are important this year. Obviously, the reinspection timeline when it happens, the outcome of it is really important. So I think we want to be open to sharing the important information with you. Obviously, the way this would work is inspection takes a week to a couple of weeks.

謝謝你，馬克。我認為我們的資訊揭露計畫將真正審視自身，並專注於我們認為今年重要的實質事項。顯然，複檢的時間安排以及複檢結果都非常重要。所以我覺得我們應該樂於與大家分享這些重要資訊。顯然，這種方式的運作方式是檢查需要一周到兩週的時間。

There's generally a closeout meeting. At that closeout meeting there's generally some kind of preliminary assessment when of Form 483. As we've noted in the past, I think, again, it's hard to believe when we first disclosed these Form 483 observations was only last quarter because it feels like for me, it's been a long time. But as you know, a Form 483 usually travels 75% of the time with any inspection. But of course, we wouldn't expect a Form 483 to result in an OAI most of the times.

通常會有一個總結會議。在結業會議上，通常會對 483 表格進行某種初步評估。正如我們過去所指出的那樣，我認為，很難相信我們首次披露這些 483 表格觀察結果只是在上個季度，因為對我來說，感覺已經過去了很長時間。但如您所知，在任何檢查中，483 表格通常有 75% 的機率會隨檢查一起提交。當然，我們通常不會期望 483 表格會導致 OAI。

And I think that's what started all of us. But I think that all along Novo has been approaching this very aggressively. So I think we'll just maintain as we have open lines of communication with you all when we have important information to share, we'll certainly do that. And I think what we've done in the past is even if it wasn't something for us, what we did learn of, let's just say, the classification of the facility as we did just last month in October. We tried to get out in front of that and disclose that and have some dialogue with you all on what that meant.

我想這就是我們所有人的起點。但我認為，諾和銳一直以來都採取了非常積極主動的態度來應對這個問題。所以，我認為我們會繼續保持與大家的溝通管道暢通，當有重要訊息要分享時，我們一定會這樣做。我認為我們過去所做的，即使那件事與我們無關，我們也了解到，比如說，該設施的分類，就像我們上個月（10 月）所做的那樣。我們試圖搶先一步，公開此事，並與大家就其意義進行一些對話。

And we'll continue to make a commitment to do the same here as we continue on this journey to an eventual resubmission and US launch upon approval.

我們將繼續致力於這樣做，因為我們將繼續努力，最終在獲得批准後重新提交申請並在美國發布。

Okay. That's helpful. And then maybe just on the idea of waiting for the reinspection to kind of happen in the reclassification before filing. But also in your prior answer is, you noted this would be kind of like an unannounced reinspection once they've communicated that they really are in position to be ready. But kind of a forcing mechanism to that at some level, it could be a submission of a BLA from anyone using this facility.

好的。那很有幫助。然後，或許可以考慮在重新分類過程中等待重新檢查結果出來後再提交申請。但你在先前的回答中也提到，這有點像是在他們表示已經做好充分準備之後進行的一次不事先通知的重新檢查。但在某種程度上，這是一種強制機制，任何使用該設施的人都可能需要提交生物製品許可申請 (BLA)。

So maybe is there some value of maybe filing ahead to kind of try to force the time line on the inspection -- or is your expectation that there are just so many other products flowing through this facility that that's kind of going to happen on its own without you guys being the forcing.

所以，提前提交申請或許能起到一定的推動作用，促使檢查按時進行——或者你們是否認為，由於該工廠有太多其他產品在流通，檢查自然會進行，無需你們主動推動。

Well, Mark, a couple of the things you said are really important. One is that we've heard this too, right? I mean the thing that creates urgency are pending applications. And right now, we don't have a pending application. We have a pending resubmission.

馬克，你說的有幾件事真的很重要。一是我們也聽過這種說法，對吧？我的意思是，真正造成緊迫感的是待處理的申請。目前，我們沒有待處理的申請。我們有一份待重新提交的申請。

At the same time, I would note that we were generally pleased with how constructive and collaborative, the in-person Type A meeting was and that there was this shared understanding of the unmet need and shared urgency. So while we're not on file, I would say a lot of the things, and you're absolutely right on your last point, that there are other pending applications at that site, and that can serve us well.

同時，我想指出，我們總體上對面對面的 A 類會議的建設性和協作性感到滿意，並且大家對未滿足的需求和緊迫性有了共同的理解。雖然我們的申請還沒有存檔，但我想說的是，您最後一點說得完全正確，該網站還有其他待處理的申請，這對我們很有幫助。

We do think our Type A meeting serves as a really good central point of highlighting while we're not on file, there is real urgency here for a community that is desperately wanting to benefit from the world's first and only muscle-directed therapy.

我們認為，我們的 A 型會議是一個很好的中心點，它強調了雖然我們沒有正式登記，但對於一個迫切希望從世界上第一個也是唯一一個肌肉定向療法中受益的群體來說，這裡確實存在著緊迫性。

And so we have to continue to work with the agency, be collaborative with them. Find everyone's right footing on what the right thing to do is, and that is really our go-forward plan. And we think we've built sort of a foundation and framework with the agency frankly, all the way through the initial priority review period up until September 22 and even through Wednesday, a really strong foundation for collaboration for us to work together to resolve this issue.

因此，我們必須繼續與該機構合作，與他們攜手共進。找到每個人對正確做法的正確立場，這才是我們真正的前進計畫。坦白說，我們認為我們已經與該機構建立了一種基礎和框架，從最初的優先審查期一直到 9 月 22 日，甚至到週三，這為我們共同解決這個問題奠定了非常堅實的合作基礎。

Yes. Thanks, and I just want to reiterate the importance of that collaborative approach. And you mentioned forcing functions as the by resubmitting ahead of the reinspection. I don't know that, that's why we want to be working closely with the FDA and be guided by them now. As we said, it's a dynamic situation then in the bite us.

是的。謝謝，我只想再次強調這種協作方式的重要性。您提到可以透過在重新檢查之前重新提交來強制執行某些功能。我不知道，所以我們現在想與FDA密切合作，並接受他們的指導。正如我們所說，這是一個瞬息萬變的局面，它會反過來咬我們一口。

So they support any kind of resolution in a particular time frame in and around the resection. We will, of course -- we are ready, and we can resubmit very efficiently -- but it's not about forcefution. We have to collaborate with the FDA.

因此，他們支持在切除手術前後特定時間範圍內的任何形式的解決方案。我們當然會——我們已經準備好了，而且我們可以非常有效率地重新提交——但這並不是強迫行為。我們必須與美國食品藥物管理局（FDA）合作。

Evan Seigerman, BMO Capital Markets.

Evan Seigerman，BMO資本市場。

With the delays for apitegromab. -- can you talk more about what your sales or market research team's efforts are two of patients ahead of the launch. You mentioned efforts to work with centers of excellence to understand the patient journey better, but do you feel you are developing a more robust number of patients which you could target for therapy following approval, potentially leading to a little bit of a faster uptake than people were probably initially expecting.

由於apitegromab的上市時間有所延遲，您能否詳細介紹您的銷售或市場研究團隊在產品上市前為兩名患者所做的努力？您提到正在努力與卓越中心合作，以更好地了解患者的治療歷程，但您是否覺得在獲得批准後，您可以針對更多患者進行治療，從而有可能比人們最初預期的更快地推廣治療？

Yes. It's a great question. Keith was highlighting it earlier in the call, and I'll turn it over to him for further comments on launch prep.

是的。這是一個很好的問題。Keith 在之前的通話中重點提到了這一點，我將把發言權交給他，讓他對發布準備工作做進一步評論。

Yes. Thanks for the question. I guess I'd say first of all, when the September 22 date occurred, that was after two months of the team being able to spend time out in the field. And now with the extended time, we're getting to not just visit with the physicians, but we're also getting to meet the SMA treatment teams and getting that full feel. And what is that additionally, not just the SMA treatment teams, we're spending more time with patient advocacy events and getting to speak with patients and their families.

是的。謝謝你的提問。我想說的第一點是，9 月 22 日那天，球隊已經進行了兩個月的戶外訓練。現在有了延長的時間，我們不僅可以拜訪醫生，還可以與 SMA 治療團隊見面，以獲得更全面的了解。此外，不僅是 SMA 治療團隊，我們也投入更多時間參與患者權益倡議活動，並有機會與患者及其家人交談。

And I can tell you what we're hearing is that there's a clear understanding of the unmet medical need and the approach of attacking this disease from a dual modality no longer just the motor neuron but also directly targeting the muscle, and this is being well accepted as we get the opportunity to meet with more people in the community. You add to the fact of the safety profile that apitegromab has demonstrated.

我可以告訴大家，我們聽到的回饋是，人們對未滿足的醫療需求有了清晰的認識，並且認識到應該採用雙管齊下的方法來治療這種疾病，不再僅僅針對運動神經元，而是直接針對肌肉。隨著我們有機會與社區中更多的人見面，這種方法也得到了廣泛的接受。您補充了apitegromab已展現的安全性。

And quite frankly, all of our studies, not just our SMA studies, but if you take a look at Embrace, that was also all adults all treated with 10-milligram per kilogram and exceptional safety results. I guess I would end with the fact that at the end of the day, we're offering the world's first muscle-targeted therapy, and in the event, if you have a choice to either be in a situation of having experienced muscle loss or the potential for muscle gain, why wouldn't you want to use a pitot.

坦白說，我們所有的研究，不只是SMA研究，如果你看看Embrace研究，所有受試者都是成年人，都接受了每公斤10毫克的治療，並且取得了非常好的安全效果。我想最後總結一下，歸根結底，我們提供的是世界上第一個針對肌肉的療法，而且，如果你可以選擇經歷肌肉流失還是有可能獲得肌肉增長，為什麼不選擇使用皮託管呢？

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

今天的電話會議到此結束。感謝您的參與。您現在可以斷開連線了。