Fulcrum Therapeutics Inc (FULC) 2024 Q2 法說會逐字稿

Good morning and welcome to Fulcrum's Therapeutics second quarter 2024 financial results and business update conference call. (Operator Instructions). This call is being webcast live and can be accessed on the Investors section of Fulcrum's website at www.fulcrumtx.com, and is being recorded.

早安，歡迎參加 Fulcrum Therapeutics 2024 年第二季財務業績和業務更新電話會議。（操作員說明）。本次電話會議正在進行網路直播，可透過 Fulcrum 網站 www.fulcrumtx.com 的投資者部分進行觀看，並且正在錄製中。

Please be reminded that remarks made during this call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.

請注意，本次電話會議期間發表的言論可能包含 1995 年《私人證券訴訟改革法案》含義內的前瞻性陳述。

These may include statements about the company's future expectations and plans, clinical development timelines and financial projections. While these forward-looking statements represent Fulcrum's view as of today, this should not be relied upon as representing the company's views in the future. Fulcrum may update these statements in the future but is not taking on an obligation to do so.

其中可能包括有關公司未來預期和計劃、臨床開發時間表和財務預測的聲明。雖然這些前瞻性陳述代表了 Fulcrum 目前的觀點，但不應將其視為代表公司未來的觀點。Fulcrum 可能會在未來更新這些聲明，但不承擔這樣做的義務。

Please refer to Fulcrum's most recent filings with the Securities and Exchange Commission for a discussion of certain risks and uncertainties associated with the company's business.

請參閱 Fulcrum 最近向美國證券交易委員會提交的文件，以了解與該公司業務相關的某些風險和不確定性的討論。

Leading the call today will be Alex Sapir, CEO and President of Fulcrum. Joining Alex on the call are Alan Musso, Chief Financial Officer; Dr. Pat Horn, Chief Medical Officer; and Dr. Iain Fraser, Senior Vice President of Development. After providing updates, on our key programs. There will be a brief Q&A in which Alex, Alan, Pat and Iain will be available to answer your questions.

今天的電話會議將由 Fulcrum 執行長兼總裁 Alex Sapir 主持。與 Alex 一起參加電話會議的還有財務長 Alan Musso；帕特‧霍恩博士，首席醫療官；以及開發資深副總裁 Iain Fraser 博士。提供關於我們的關鍵計劃的更新後。Alex、Alan、Pat 和 Iain 將進行簡短的問答，回答您的問題。

With that, it's my pleasure to turn the call over to Alex.

至此，我很高興將電話轉給亞歷克斯。

That's great. Thank you, Lisa, and good morning, everyone, and thanks to all of you for joining us for our second quarter conference call. We've organized today's call to provide you with updates on recent progress and upcoming milestones for our two clinical-stage assets, losmapimod and pociredir. And after a brief introduction, we'll segue into our pipeline, I'll then ask Alan to review the financials. And finally, we'll end by taking your questions.

那太棒了。謝謝麗莎，大家早安，謝謝大家參加我們的第二季電話會議。我們組織了今天的電話會議，為您提供有關我們兩種臨床階段資產 losmapimod 和 pociredir 的最新進展和即將到來的里程碑的最新資訊。在簡短的介紹之後，我們將繼續討論我們的管道，然後我會請艾倫審查財務狀況。最後，我們將回答大家的問題。

I am happy to report that we are on track to report top-line data for the Phase 3 REACH trial of losmapimod by the end of October compared to our previous guidance of the fourth quarter.

我很高興地報告，與我們先前第四季的指導相比，我們預計在 10 月底之前報告 losmapimod 第三階段 REACH 試驗的頂線數據。

As we advance toward this important inflection point, we continue to build out the team and add talent as we prepare for the potential NDA filing and the US commercial launch of losmapimod. In parallel, we are working with Sanofi in preparation for regulatory filings and the launch of losmapimod outside of the United States.

隨著我們邁向這一重要的轉折點，我們將繼續組建團隊並增加人才，為潛在的 NDA 申請和 Losmapimod 在美國的商業上市做準備。同時，我們正在與賽諾菲合作，準備監管備案以及在美國境外推出洛斯馬莫德。

As a reminder, in May of this year, we announced our collaboration and license agreement with Sanofi for the development and commercialization of losmapimod for facioscapulohumeral muscular dystrophy, or FSHD for short, for all territories outside of the US.

提醒一下，今年 5 月，我們宣布與賽諾菲就用於治療面肩肱型肌肉營養不良症（簡稱 FSHD）的 losmapimod 的開發和商業化達成合作和許可協議，適用於美國以外的所有地區。

We believe we selected the best possible partner for losmapimod as this collaboration combines Fulcrum's expertise in FSHD with Sanofi's deep regulatory development and commercial capabilities in neuromuscular markets around the world. Together, we look forward to delivering on our shared commitment to address the high unmet need of patients in the FSHD community.

我們相信，我們為 losmapimod 選擇了最佳的合作夥伴，因為此次合作結合了 Fulcrum 在 FSHD 方面的專業知識與賽諾菲在全球神經肌肉市場的深入監管開發和商業能力。我們期待共同兌現我們的共同承諾，解決 FSHD 社區患者未滿足的高度需求。

Let me spend a bit more time on losmapimod, which as many of, is an oral small molecule selective T-38 alpha-beta MAP kinase inhibitor that inhibits DUX4 expression and many of its downstream transcripts and thus prevents muscle cell death in patients with FSHD.

讓我多花一點時間來談談洛斯馬莫德，它是一種口服小分子選擇性T-38 α-β MAP 激酶抑製劑，可抑制DUX4 表達及其許多下游轉錄物，從而防止FSHD 患者的肌肉細胞死亡。

An estimated 30,000 patients in the US have FSHD, which is characterized by a slow but relentless loss of muscle function year-after-year resulting in significant impairment of upper extremity muscle function and mobility.

據估計，美國有 3 萬名 FSHD 患者，其特徵是肌肉功能年復一年緩慢但持續喪失，導致上肢肌肉功能和活動能力顯著受損。

And while there is a degree of heterogeneity in the onset and disease progression of FSHD, this relentless loss of muscle function means that approximately 20% of patients become wheelchair-bound. I think it's important to remind everyone that there are currently no approved therapies for FSHD and no drugs used off-label to help these patients.

雖然 FSHD 的發病和疾病進展存在一定程度的異質性，但這種肌肉功能的持續喪失意味著大約 20% 的患者需要坐輪椅。我認為重要的是要提醒大家，目前尚無批准的 FSHD 治療方法，也沒有超說明書使用的藥物來幫助這些患者。

Now let's turn our attention to our Phase 3 registrational trial called REACH, which I spoke about earlier. As a reminder, Reach is a 48-week Phase 3 trial intended to be registration enabling both here in the US and in ex-US geographies. In September of last year, we completed enrollment in REACH with a total of 260 patients.

現在讓我們將注意力轉向我之前談到的名為 REACH 的第三階段註冊試驗。提醒一下，Reach 是一項為期 48 週的 3 期試驗，旨在在美國境內和美國以外地區進行註冊。去年9月，我們完成了REACH入組，共有260名患者。

As presented at the 31st Annual FSHD Society International Research Congress in June of this year, baseline characteristics of the REACH study population are similar to those of our Phase 2 ReDUX4 study population.

正如今年 6 月在第 31 屆年度 FSHD 協會國際研究大會上提出的那樣，REACH 研究人群的基線特徵與我們的 2 期 ReDUX4 研究人群的基線特徵相似。

Building on the encouraging clinical benefit and favorable tolerability observed in our ReDUX4 trial, the primary endpoint for the REACH study is the change from baseline in the relative surface area or RSA, which is a quantitative assessment of reachable workspace and has been shown to correlate with disease severity and progression.

基於我們在ReDUX4 試驗中觀察到的令人鼓舞的臨床效益和良好的耐受性，REACH 研究的主要終點是相對表面積或RSA 相對基線的變化，這是對可到達工作空間的定量評估，並已被證明與疾病的嚴重程度和進展。

RSA is a measure of upper extremity range of motion and muscle function that specifically evaluate shoulder and arm mobility using 3D motion sensor technology and is indicative of the ability to perform activities of daily living.

RSA 是上肢運動範圍和肌肉功能的衡量標準，專門使用 3D 運動感測器技術評估肩部和手臂的活動能力，並顯示執行日常生活活動的能力。

Based on collaborative interactions with the clinical outcomes assessment, or COA Group at FDA, we are further assessing the extent to which a specific change in the RSA score is meaningful to patients. I'm pleased to report that we are on track to complete the activities agreed upon with the FDA at the time of reporting the REACH top-line data.

基於與臨床結果評估或 FDA 的 COA 小組的協作互動，我們正在進一步評估 RSA 評分的特定變化對患者有意義的程度。我很高興地報告，我們正在按計劃完成在報告 REACH 頂線數據時與 FDA 商定的活動。

Additional key secondary endpoints in REACH include muscle fat infiltration or MFI, which is a marker of disease pathology measured by whole body MRI, shoulder dynamometry and Patient Global Impression of Change or PGIC, for short.

REACH 中的其他關鍵次要終點包括肌肉脂肪浸潤或 MFI，它是透過全身 MRI、肩部測力和患者整體變化印象（簡稱 PGIC）測量的疾病病理學標誌。

We believe that the implementation of self-reported quality-of-life measures and health care utilization questionnaires will help inform our payer strategy as we begin preparing for a commercial launch to deliver the first FSHD treatment for patients.

我們相信，在我們開始準備商業啟動以向患者提供首個 FSHD 治療時，實施自我報告的生活品質衡量標準和醫療保健利用調查問卷將有助於為我們的付款人策略提供資訊。

We are now progressing towards the completion of the 48-week treatment phase of the trial for all enrolled patients. As of June 30, 2024, of the 234 out of the 260 who completed the 48-week treatment phase, 232 of those or 232 of those patients or 98% chose to enroll in Part B, the open-label extension of the study in which all patients receive drug.

我們現在正在努力完成所有入組患者為期 48 週的試驗治療階段。截至 2024 年 6 月 30 日，在 260 名完成 48 週治療階段的 234 名患者中，其中 232 名或其中 232 名患者或 98% 選擇參加 B 部分，即該研究的開放標籤擴展所有患者均接受藥物治療。

This very high percentage of patients opting to move into the open-label phase is similar to what was observed in our Phase 2 clinical trial, and we believe is indicative of the high unmet need for patients with FSHD. Again, we are on track to report top-line data by the end of October, which will bring us one step closer to delivering the first-ever FDA-approved therapy for FSHD patients.

選擇進入開放標籤階段的患者比例非常高，這與我們在第 2 期臨床試驗中觀察到的情況相似，我們認為這表明 FSHD 患者的需求未被滿足。同樣，我們預計在 10 月底之前報告主要數據，這將使我們距離為 FSHD 患者提供首個 FDA 批准的治療方法又近了一步。

Now let's talk a little bit about pociredir, which is our oral HBF inducer for the potential treatment of patients with sickle cell disease. The elevation of HbF or fetal hemoglobin is a clinically validated therapeutic rationale for sickle cell disease, a lifelong inherited blood disorder that severely impairs quality of life for approximately 100,000 people in the US and approximately 4.4 million people worldwide, making sickle cell disease one of the most prevalent nonmalignant hematologic diseases.

現在讓我們來談談 pociredir，它是我們的口服 HBF 誘導劑，用於治療鐮狀細胞疾病患者。HbF 或胎兒血紅蛋白升高是鐮狀細胞疾病的臨床驗證治療原理，鐮狀細胞疾病是一種終生遺傳性血液疾病，嚴重損害美國約10 萬人和全球約440 萬人的生活質量，使鐮狀細胞疾病成為最常見的疾病之一。

Historically, the standard treatment for sickle cell disease has included blood transfusion, pain medications and hydroxyurea that focus only on symptom relief. While exciting scientific progress has enabled the advancement and more recently, the approval of gene editing therapeutic approaches, we believe there remains a high unmet need for an oral therapeutic option that is broadly protective of sickle cell disease symptomatology.

從歷史上看，鐮狀細胞疾病的標準治療包括輸血、止痛藥和羥基脲，僅著重於緩解症狀。雖然令人興奮的科學進步推動了基因編輯治療方法的進步，並且最近獲得了批准，但我們認為，對廣泛保護鐮狀細胞疾病症狀的口服治療方案的需求仍然很高，未得到滿足。

As a first-in-class oral small molecule HbF inducer we believe pociredir has the potential to address this unmet need.

作為一流的口服小分子 HbF 誘導劑，我們相信 pociredir 有潛力解決這個未滿足的需求。

Turning now to our Phase 1b clinical trial, the PIONEER trial, we continue to make progress. Given that many of the sites we are newly activating are academic sites here in the US as well as outside the US, both of which have long site activation lead times, together with our narrower inclusion-exclusion criteria, it is taking longer than initially expected. We expect to have study data to share with everyone in 2025.

現在轉向我們的 1b 期臨床試驗，即 PIONEER 試驗，我們繼續取得進展。鑑於我們新激活的許多網站都是美國境內以及美國境外的學術網站，這兩個網站的激活週期都較長，再加上我們的納入排除標準較窄，因此所需時間比最初預期的要長。我們預計到 2025 年會有研究數據與大家分享。

As a reminder, Cohort 3 of the Phase 1b trial will evaluate pociredir at the 12 milligram once daily dose, with a dosing duration of three months followed by Cohort 4 at the 20-milligram once-daily dose also for three months. Both cohorts are expected to enroll approximately 10 patients each.

提醒一下，1b 期試驗的第 3 組將評估 12 毫克每日一次劑量的 pociredir，給藥持續時間為三個月，隨後第 4 組的 20 毫克每日一次劑量也持續三個月。兩個隊列預計各招募約 10 名患者。

We look forward to building on the encouraging clinical data obtained prior to the clinical hold which demonstrated that pociredir increased total fetal hemoglobin of a magnitude that could translate into a meaningful improvement in disease severity.

我們期待在臨床擱置之前獲得的令人鼓舞的臨床數據的基礎上，這些數據表明 pociredir 增加了胎兒總血紅蛋白的數量，可以轉化為疾病嚴重程度的有意義的改善。

These interim results of the Phase 1b trial involving 16 patients were recently reported in June at the EHA conference in Madrid and were very well received by the medical community. We believe that pociredir as an oral HbF inducer has the potential to provide a differentiated therapeutic option for people living with sickle cell disease, and addressing the significant unmet need in the sickle cell disease community remains a key priority for us.

這些涉及 16 名患者的 1b 期試驗的中期結果最近於 6 月在馬德里舉行的 EHA 會議上報告，並受到醫學界的好評。我們相信，pociredir 作為口服 HbF 誘導劑有可能為鐮狀細胞疾病患者提供差異化的治療選擇，而解決鐮狀細胞疾病領域未滿足的重大需求仍然是我們的首要任務。

So, with that update on the business, let me now turn it over to our Chief Financial Officer, Alan Musso, to run through the financials. Alan, over to you.

因此，在了解業務最新情況後，現在讓我將其交給我們的財務長艾倫·穆索 (Alan Musso)，他負責處理財務狀況。艾倫，交給你了。

Thanks, Alex. I'll now go over our results for the second quarter ended June 30, 2024. Let me start with our cash position. As of June 30, 2024, cash, cash equivalents and marketable securities were $273.8 million as compared to $236.2 million as of December 31, 2023.

謝謝，亞歷克斯。現在我將回顧截至 2024 年 6 月 30 日的第二季業績。讓我從我們的現金狀況開始。截至2024年6月30日，現金、現金等價物及有價證券為2.738億美元，截至2023年12月31日為2.362億美元。

The increase in our cash position is due to the $80 million upfront payment received from Sanofi in the second quarter of 2024, partially offset by cash used to fund our operating activities in the first half of the year.

我們現金部位的增加是由於 2024 年第二季從賽諾菲收到的 8,000 萬美元預付款，部分被用於為我們上半年營運活動提供資金的現金所抵銷。

Collaboration revenue was $80 million for the second quarter of 2024 compared to $0.9 million for the second quarter of 2023. The increase of $79.1 million was primarily due to recognition of the $80 million upfront license payment received from Sanofi during the second quarter of 2024.

2024 年第二季的協作收入為 8,000 萬美元，而 2023 年第二季的協作收入為 90 萬美元。增加 7,910 萬美元主要是因為確認了 2024 年第二季從賽諾菲收到的 8,000 萬美元預付款。

Our research and development expenses were $17.3 million for the second quarter of 2024 compared to $17.8 million for the second quarter of 2023. The decrease of $0.5 million was primarily due to the losmapimod global development cost-sharing reimbursement from Sanofi, partially offset by increased costs related to the advancement of the REACH trial.

2024 年第二季我們的研發費用為 1,730 萬美元，而 2023 年第二季為 1,780 萬美元。減少 50 萬美元主要是由於賽諾菲對 Losmapimod 全球開發費用分攤費用的報銷，但部分被與 REACH 試驗進展相關的費用增加所抵消。

The general and administrative expenses were $10.2 million for the second quarter of 2024 compared to $10.3 million for the second quarter of 2023 and the $0.1 million decrease was primarily due to lower equity compensation costs. For the second quarter of 2024, we had net income of $55.4 million compared to a net loss of $23.8 million for the second quarter of 2023.

2024 年第二季的一般和管理費用為 1,020 萬美元，而 2023 年第二季為 1,030 萬美元，減少 10 萬美元主要是由於股權補償成本降低。2024 年第二季度，我們的淨利潤為 5,540 萬美元，而 2023 年第二季的淨虧損為 2,380 萬美元。

For the foreseeable future, excluding the potential for future milestone payments under our Sanofi collaboration, we expect to be in a loss position, including for the year ended December 31, 2024.

在可預見的未來，排除我們與賽諾菲合作下未來里程碑付款的可能性，我們預計將處於虧損狀態，包括截至 2024 年 12 月 31 日的年度。

And finally, turning to our cash runway guidance. Based on our current operating plans, we continue to expect that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating requirements into 2027.

最後，轉向我們的現金跑道指南。根據我們目前的營運計劃，我們繼續預期現有的現金、現金等價物和有價證券將足以滿足我們到 2027 年的營運需求。

And with that, let me turn the call back over to Alex.

接下來，讓我把電話轉回給亞歷克斯。

Great. Thanks so much, Alan. So, with that overview of the business and the financials, Lisa, let's go ahead and open it up for questions.

偉大的。非常感謝，艾倫。那麼，麗莎，在對業務和財務狀況進行了概述之後，讓我們繼續提出問題。

(Operator Instructions)

（操作員說明）

Corinne Jenkins of Goldman Sachs.

高盛的科琳·詹金斯。

Maybe first for the work that you're doing to validate reachable workspace for the agency, has the agency specified like specific metrics that are most important or the magnitude of changes like the change or the benefit they'd like to see? Or are they just asking for like general proof that the reachable workspace is beneficial? And then are there multiple analyses that they requested them? Do they all sort of have to go in the same direction? And then my other question in terms of the cash runway guidance you just provided into '27, what specific like clinical milestones and then commercial activity?

也許首先對於您正在為驗證機構的可達工作空間所做的工作，機構是否指定了最重要的特定指標或變​​化的幅度（例如他們希望看到的變化或好處）？或者他們只是要求提供可到達的工作空間是有益的一般證據？然後他們要求進行多項分析嗎？他們都必須朝著同一個方向前進嗎？然後我的另一個問題是關於您剛剛提供的 27 年現金跑道指導，具體是什麼，例如臨床里程碑和商業活動？

Yes. It's great. Thanks, Corinne, and thanks for the question. And if there are any more follow-up questions, maybe just speak up a little bit. You were a little bit difficult to hear. I think in terms of your questions around regional workspace and some of the work we're doing to prove out the clinical meaningfulness of that work I'll turn that one over to Iain, and then I'll ask Alan to provide an update on your question around the financials. So, Iain?

是的。這很棒。謝謝科琳，也謝謝你的提問。如果還有其他後續問題，或許可以說出來。你聽得有點困難。我認為，就您關於區域工作空間的問題以及我們正在做的一些工作來證明這項工作的臨床意義而言，我會將其轉交給伊恩，然後我會請艾倫提供最新情況你關於財務的問題。那麼，伊恩？

Yes, sure. Thanks, Corinne. So, since the regional workspace has not previously been used for any approvals, there are no preestablished criteria. And so there has not been a numerical criterion put forward by the agencies for this.

是的，當然。謝謝，科琳娜。因此，由於區域工作空間之前尚未用於任何審批，因此沒有預先制定的標準。因此，各機構還沒有為此提出一個數字標準。

And the work we are doing is in order to evaluate that across a range of different approaches leading towards an understanding of the meaningful score difference, which is the FDA term for a change within a given patient that is considered meaningful.

我們正在做的工作是為了透過一系列不同的方法進行評估，從而理解有意義的評分差異，這是 FDA 術語，指的是特定患者體內被認為有意義的變化。

So, as I say, there is no prespecified numerical value for that and the work coming out of the results coming out of the work that we are doing will inform that.

因此，正如我所說，沒有預先指定的數值，我們正在進行的工作的結果將告訴我們這一點。

And then, Alan, maybe toward the second question that Corinne had.

然後，艾倫，也許是針對科琳提出的第二個問題。

Yes, sure, Corinne. The cash runway guidance that we're giving that does anticipate a full sort of success scenario. So, it funds the sort of further development of filing and commercialization of losmapimod. It also funds the completion of the ongoing pociredir trial as well as the follow-up trial that we anticipate, and funds the preclinical work that we have ongoing for which we expect that we'll have new products entering into the clinic from that work. So, it's sort of all in what we anticipate with the current portfolio of continued advancement.

是的，當然，科琳娜。我們給出的現金跑道指導確實預見了全面的成功場景。因此，它為洛斯馬莫德的申請和商業化的進一步發展提供資金。它還資助完成正在進行的 pociredir 試驗以及我們預期的後續試驗，並資助我們正在進行的臨床前工作，我們預計我們將有新產品進入臨床。因此，這就是我們對目前持續進步組合的預期。

Kristen Kluska of Cantor Fitzgerald.

坎托·菲茨杰拉德的克里斯汀·克魯斯卡。

Good morning everybody. Thanks for all the transparency and extra guidance today. So, on reachable workspace, we've been getting a lot of questions just because another company also had data on this. And to your point, given it really is a new endpoint hasn't been previously used for approval. Wondering now that we have two data sets supporting the endpoint if this helps, in your opinion, derisk this endpoint for this disease?

大家早安。感謝今天的所有透明度和額外指導。因此，在可達工作空間方面，我們收到了很多問題，只是因為另一家公司也有這方面的數據。就您的觀點而言，鑑於它確實是一個以前未用於批准的新端點。現在想知道我們有兩個支持該終點的數據集，您認為這是否有助於降低該疾病終點的風險？

Yes. That's great. Kristen, thanks for the question. I think to answer that, I'll turn it over again to Iain.

是的。那太棒了。克里斯汀，謝謝你的提問。我想回答這個問題，我會再把它交給伊恩。

Yes. Thanks, Kristen. I think it is encouraging for us to see that others are using this particular endpoint in FSHD, we certainly had hints from the agency that, that's the case that they're hearing about this as we engage with them around our discussions around reachable workspace.

是的。謝謝，克里斯汀。我認為看到其他人在FSHD 中使用這個特定端點對我們來說是令人鼓舞的，我們當然從該機構得到了暗示，當我們與他們圍繞可達工作空間進行討論時，他們正在聽到這一點。

And I think it also reinforces the point that we've made previously that we haven't been suggested an alternative primary endpoint in FSHD. So, I think we're overall encouraged by those reports that have been coming out.

我認為這也強化了我們先前提出的觀點，我們尚未建議 FSHD 的替代主要終點。因此，我認為我們對這些已經發布的報告總體感到鼓舞。

Yes. And the thing I would add, Kristen, this is Alex, is and you had mentioned some other data that had recently been published or reported out. I assume you're referring to the AVIDITY data.

是的。我要補充的是，克里斯汀，這是亞歷克斯，你提到了最近發布或報告的一些其他數據。我假設您指的是 AVIDITY 資料。

I think what the AVIDITY data does is it certainly validates not only the DUX4 pathway, but more specifically to your question, reachable workspace as the clinical endpoint, we believe that many of the registrational trials that will come after us will be required as part of their primary endpoint.

我認為 AVIDITY 數據的作用肯定不僅驗證了 DUX4 途徑，更具體地說，對於您的問題，可到達的工作空間作為臨床終點，我們相信，在我們之後進行的許多註冊試驗將需要作為他們的主要終點。

Okay. Appreciate that. And then assuming that the trial is successful, when you plan to meet with the FDA, can you just remind us, first, what the safety data set is including from the previous company that had studies in other indications. And then second, how much open-label extension data you're going to have from Phase 2 at that point to add on to the pool of evidence? Thanks so much, again.

好的。很欣賞這一點。然後假設試驗成功，當您計劃與 FDA 會面時，您能否提醒我們，首先，安全資料集包括先前進行其他適應症研究的公司的內容。其次，屆時您將從第二階段獲得多少開放標籤擴展資料來添加到證據池中？非常感謝，再次。

Thanks, Kristen. Maybe I'll take the first question, and I'll turn the second question over to Iain. Yes, I think that is really one of the truly unique things about losmapimod. I don't know if this is unprecedented, but we will be filing our new drug application with a patient safety database that includes over 3,600 patients.

謝謝，克里斯汀。也許我會回答第一個問題，然後我會將第二個問題交給伊恩。是的，我認為這確實是洛斯馬莫德真正獨特的事情之一。我不知道這是否是史無前例的，但我們將透過包含 3,600 多名患者的病患安全資料庫提交新藥申請。

So, I think for a rare disease such as FSHD with a prevalence of 30,000, have 3,600 patients in the patient safety database is quite remarkable. And I think one of the encouraging signs is the drug does have a fairly sort of unremarkable AE profile, and we've demonstrated that in our ReDUX4 study and expect to replicate that in our REACH study. And then, Alan sorry, Iain, maybe to Kristen's second question.

因此，我認為對於像 FSHD 這樣的罕見疾病，盛行率高達 30,000 例，患者安全資料庫中有 3,600 名患者是相當了不起的。我認為令人鼓舞的跡象之一是該藥物確實具有相當不顯著的 AE 特徵，我們已經在 ReDUX4 研究中證明了這一點，並期望在我們的 REACH 研究中複製這一點。然後，艾倫，對不起，伊恩，也許是克莉絲汀的第二個問題。

Yes. So, we'll have data from the ReDUX4 Phase 2 study, remember that enrolled 80 patients, and there's been a high rate of retention in the open-label extension of that study now up to exceeding three years of exposure in some of those patients, the ones that were enrolled at the very earliest in that study. We have about 11 patients in a separate open-label study in Europe that also has a prolonged duration of treatment. And then in the REACH study itself, there are 260 patients.

是的。因此，我們將獲得 ReDUX4 2 期研究的數據，請記住，該研究招募了 80 名患者，並且該研究的開放標籤擴展的保留率很高，目前其中一些患者的暴露時間已超過三年，最早參加研究的人。我們在歐洲進行了一項單獨的開放標籤研究，約有 11 名患者接受長期治療。REACH 研究本身有 260 名患者。

And as Alex mentioned in the original remarks, a very high proportion of those are electing to rollover into the open-label extension. And so, they will continue to experience exposure to losmapimod as we move towards filing. So, there is not only a large safety database from other indications that preceded the work that we had done, but also a large and growing safety database for this relatively a rare disease.

正如 Alex 在最初的評論中提到的，其中很大一部分人選擇轉入開放標籤擴充。因此，當我們準備提交申請時，他們將繼續接觸洛斯馬莫德。因此，不僅有一個來自我們所做工作之前的其他適應症的大型安全資料庫，而且還有一個針對這種相對罕見的疾病的大型且不斷增長的安全資料庫。

Joseph Schwartz of Leerink Partners.

Leerink Partners 的 Joseph Schwartz。

Thanks for the update sorry. So, I was wondering a couple of things on pociredir first and then losmapimod. Can you talk a little bit more about why it's taking longer to proceed with the Cohort 3 group of patients for pociredir, is it the IRB level? Is it enrollment? Can you give us any clear progress update? And maybe talk about what initiatives you have to accelerate things? And then I have a question on losmapimod?

感謝您的更新抱歉。所以，我首先想知道關於 pociredir 的一些事情，然後是 losmapimod。您能否多談談為什麼第 3 組患者需要更長的時間才能進行 pociredir，是 IRB 等級嗎？是招生嗎？您能給我們任何明確的進展嗎？或許可以談談你有哪些舉措加速事情的發展？然後我有一個關於losmapimod 的問題？

Sure. That's great, Joe. Thanks for the question. This is Alex. I'll take the first one and then depending on what the second question is, we'll figure out who we can triage that one, two. Yes. So, as I mentioned, and maybe I'll give a little bit more color the seven or eight sites that were involved in the PIONEER trial prior to the initiation of the clinical hold, which happened, I think, in February of 2023.

當然。太好了，喬。謝謝你的提問。這是亞歷克斯。我將回答第一個問題，然後根據第二個問題是什麼，我們將找出可以對這個問題進行分類的人。是的。因此，正如我所提到的，也許我會對在臨床暫停開始之前參與 PIONEER 試驗的七八個地點進行更多介紹，我認為臨床暫停發生在 2023 年 2 月。

Most of those sites tended to treat younger patients that didn't have the disease severity that was matching our new inclusion/exclusion criteria that we reached agreement on with the agency in order to get off clinical hold.

大多數這些網站傾向於治療較年輕的患者，這些患者的疾病嚴重程度不符合我們與該機構達成一致的新納入/排除標準，以便擺脫臨床擱置。

So many of those existing sites would have been terrific to reactivate it would have taken several months to get the new protocol through the IRB, all the contracting had been done. But because what we were hearing from those PIs is that they did not have those patients essentially, we had to activate all new sites both in the US as well as in countries outside of the US, specifically in Africa.

如此多的現有站點如果能夠重新啟動就非常好，因此需要幾個月的時間才能透過 IRB 獲得新協議，所有合約都已完成。但因為我們從這些 PI 那裡聽到的是，他們基本上沒有這些患者，所以我們必須啟動美國以及美國以外國家（特別是非洲）的所有新地點。

Our goal is still to our goal is still to activate about 15 to, call it, 17, 18 sites in the US and a handful of sites outside of the US So, we should have 20 sites, which should be more than adequate to enroll these 20 patients in these next 2 cohorts.

我們的目標仍然是在美國激活大約 15 個到 17、18 個站點以及美國境外的少數站點因此，我們應該有 20 個站點，這對於註冊來說應該綽綽有餘接下來 2 個隊列中的 20 名患者。

So, I think it's really just it's this long lead time of activating these sites, many of which are academic sites. And as many of you know, it can take up to nine months from contracting to IRB approval, all of the back and forth at the sites to get these sites activated again at these major academic institutions.

所以，我認為這實際上只是激活這些網站的準備時間很長，其中許多是學術網站。正如你們許多人所知，從簽約到 IRB 批准可能需要長達九個月的時間，所有這些網站都在這些主要學術機構的網站上來回。

In terms of what we're doing to try to speed that up, I think we're trying to do I think we're doing a couple of things. Number one, when a contract provision comes in, we're typically returning that around in about 24 to 48 hours.

就我們正在努力加快速度而言，我認為我們正在努力做幾件事。第一，當合約條款生效時，我們通常會在大約 24 到 48 小時內退還。

So, the people in our legal group knows that PIONEER is the top priority and anything that comes in from PIONEER gets put to the top of the pile. Unfortunately, we're sort of at the whim of the large institutional bureaucracy and unfortunately, things just take time.

因此，我們法律團隊的人員知道 PIONEER 是重中之重，任何來自 PIONEER 的產品都會被放在最上面。不幸的是，我們有點受大型機構官僚機構的影響，不幸的是，事情只是需要時間。

But I will say that of the sites that we have activated, and we have activated a number of sites, they continue to be very encouraged of the transformational potential that pociredir could bring to their patient population.

但我要說的是，在我們已經啟動的站點中，我們已經啟動了許多站點，他們仍然對 pociredir 可以為他們的患者群體帶來的變革潛力感到非常鼓舞。

There is a relatively small number of patients, somewhere between 7.5% to 10% of that 100,000 that I mentioned that do meet our inclusion/exclusion criteria. So, call it about 10,000 patients. But we believe that's more than an adequate number to be able to recruit these 20 patients. And as I mentioned, we will expect to have data that we can share with everybody sometime in 2025. And maybe turning to your losmapimod question?

確實符合我們的納入/排除標準的患者數量相對較少，在我提到的 100,000 名患者中，只有 7.5% 到 10% 之間。所以，稱之為大約 10,000 名患者。但我們相信這個數字足以招募這 20 名患者。正如我所提到的，我們預計將在 2025 年的某個時候獲得可以與所有人分享的數據。也許轉向你的losmapimod 問題？

Yes. That was helpful color on pociredir. So as far as losmapimod goes, I was wondering if you could talk about the output that will emerge from the work define the minimally clinically relevant difference on the RWS. Is this just one single number? Is it a range which might provide more context? And how do you feel about the ability of losmapimod to provide a benefit, which exceeds whatever you define as the MCD?

是的。這是 pociredir 上有用的顏色。因此，就 losmapimod 而言，我想知道您是否可以談談工作中產生的輸出，定義了 RWS 上最小的臨床相關差異。這只是一個數字嗎？它是一個可以提供更多背景資訊的範圍嗎？您對洛斯馬莫德提供的益處（超出您對 MCD 的定義）的能力有何看法？

Sure. Thanks, Joe. I think to answer that, let me turn that one over to Iain as well.

當然。謝謝，喬。我想回答這個問題，讓我也把這個問題交給伊恩。

Yes. Thanks, Joe. And just to clarify, I think the number that in the FDA terminology is the meaningful score difference. And I think the important thing to emphasize about that is that, that's not a mean score for the overall population that needs to be exceeded, but that's the score for a within patient change. So that's understanding what a change in the reachable workspace score is for an individual patient.

是的。謝謝，喬。澄清一下，我認為 FDA 術語中的數字是有意義的分數差異。我認為需要強調的重要一點是，這不是需要超過的總體人群的平均分數，而是患者內部變化的分數。因此，這就是了解對於單一患者而言，可達工作空間分數的變化是什麼。

And so, what that's most likely to be reflected in looking at the clinical trial data is proportions of patients individual patients that exceed that meaningful score difference. So, I think that's just conceptually a way of thinking about it. That's an important piece of it. And that's really the focus of this. There might well be a range of these depending on the outputs of that work, and we'll report that at the time of the top-line data.

因此，在查看臨床試驗數據時最有可能反映出的是超過有意義的評分差異的患者個體的比例。所以，我認為這只是概念上的一種思考方式。這是其中很重要的一部分。這確實是本文的重點。根據這項工作的產出，很可能會有一系列的結果，我們將在發布頂線數據時報告這一點。

Dae Gon Ha of Stifel.

Stifel 的 Dae Gon Ha。

Good morning. Thanks for taking my question. I'll stick my two questions with the losmapimod side of things. Specifically, on powering, so maybe a question for Iain. So, the first question is, you previously talked about powering for REACH as having benefited from the over enrollment. I think it was 96% to show 10% placebo-adjusted RSA. Just to clarify, was this just for the FSHD type one or inclusive of type two? And now that you do have the type 2 baseline characteristics disclosed, like what would that powering be, if any, different from the original one?

早安.感謝您提出我的問題。我將把我的兩個問題與洛斯馬莫德方面聯繫起來。具體來說，關於供電，所以可能是伊恩的問題。所以，第一個問題是，您之前談到為 REACH 提供支援是從過度註冊中受益的。我認為 96% 的人顯示 10% 的安慰劑調整 RSA。澄清一下，這只是針對 FSHD 第一類還是包括第二類？既然您確實已經披露了 2 型基線特徵，那麼這種動力與原始動力有何不同（如果有的話）？

And then second question on powering as well. Just curious, have you guys done additional sensitivity analysis around sort of the evolving resolve natural history data? And what might that mean for the powering of REACH? Thanks so much.

然後是關於供電的第二個問題。只是好奇，你們是否對不斷演變的解析自然史資料進行了額外的敏感度分析？這對於 REACH 的推動意味著什麼？非常感謝。

That's great. Thanks, Dae Gon, and thanks for the question. Yes, Iain, do you want to take that one?

那太棒了。謝謝大坤，也謝謝你的提問。是的，伊恩，你想買那個嗎？

Yes. Dae Gon, as I think we've articulated before, the powering for REACH was based on specifically the FSHD 1 patient population. And that's because the data that we used for the powering was from ReDUX4 and that study enrolled only FSHD type 1. And so that was the approach that was taken even though the primary endpoint for the REACH trial will include not only the type 1s but also the type 2s.

是的。Dae Gon，如我先前所闡明的，REACH 的支持特別基於 FSHD 1 患者群體。這是因為我們用於支援的數據來自 ReDUX4，而該研究僅招募了 FSHD 1 型。因此，儘管 REACH 試驗的主要終點不僅包括 1 類，還包括 2 類，但仍採取了這種方法。

And so, the powering initially, the powering was based on 210 FSHD type 1 patients, and that was the powering at 93%. And the expectation was that we would, in addition, have 20 FSHD type 2 patients who pushed the total up to 230.

因此，最初的供電是基於 210 名 FSHD 1 型患者，即 93% 的供電。我們預計還會有 20 名 FSHD 2 型患者，從而使總數達到 230 名。

The over-enrollment pushed the total enrollment from 230 to 260, and the type 1s went from 210 to 242. And so that's really the comparison for the powering was the 210 to 242 type 1s moving it from 93% to 96%.

超額招生使總入學人數從230人增加到260人，1類從210人增加到242人。因此，這實際上是 210 到 242 類型 1 的動力比較，將其從 93% 提高到 96%。

As you've indicated, we have the baseline characteristics. We know there are 18 FSHD type 2 patients. And we also know that the randomization is stratified for that. So, the expectation is that there will be nine and nine in each of the groups, placebo and active.

正如您所指出的，我們有基線特徵。我們知道有 18 位 FSHD 2 型患者。我們也知道隨機化是為此分層的。因此，預計每組（安慰劑組和活性組）將有九個和九個。

Great. Thanks again for the question, Dae Gon. Lisa?

偉大的。再次感謝大坤的提問。麗莎？

And our next question will be coming from the line of Gregory Renza.

我們的下一個問題將來自格雷戈里·倫扎（Gregory Renza）。

Yes. Alex and team, congrats on the progress. We're looking forward to the data in October. Alex, just on losmapimod, as you touched a bit about upon payer engagement, maybe just give us a glimpse of what that engagement will and has been looking like what do you see as the potential tailwinds from the potential data package and losmapimod's profile? Maybe what do you foresee as some of the greatest challenges when it comes to establishing the value proposition and assuming that the data cards flip as you foresee?

是的。亞歷克斯和他的團隊，祝賀你們的進展。我們期待十月份的數據。亞歷克斯，就 losmapimod 而言，當您談到付款人參與度時，也許只是讓我們了解一下這種參與度將如何，以及您認為潛在數據包和 losmapimod 資料中的潛在推動因素是什麼？在建立價值主張並假設數據卡如您所預見的那樣翻轉時，您認為最大的挑戰是什麼？

Yes. Great. Great question, and thanks for that. Yes, I think so, we've done some payer research following the results of the ReDUX4 study results. And what we heard from the payer community, and again, we probably went out and talked at the time to about 15 or so payers.

是的。偉大的。很好的問題，謝謝。是的，我認為是的，我們根據 ReDUX4 研究結果進行了一些付款人研究。我們從付款人社區聽到的消息，我們當時可能出去與大約 15 個左右的付款人進行了交談。

The majority of those were US-based payers. And I think what we heard across the board is it will be difficult to restrict access to this drug when approved for patients given the fact that there are clearly nothing available and nothing used off-label to help these patients with FSHD.

其中大多數是美國的付款人。我認為我們普遍聽到的是，當這種藥物被批准用於患者時，將很難限制其使用，因為顯然沒有任何可用的藥物，也沒有任何超適應症藥物可以幫助這些 FSHD 患者。

I think the other thing that we heard from that payer research when probing a bit on what they expected the pricing to look like for the drug, and we have not given any specific guidance around pricing, but what we clearly heard back from the payers is they would expect this to look very similar to other rare disease drugs that have been priced in the, call it, hundreds of thousands of dollars every year per patient.

我認為我們從付款人研究中聽到的另一件事是，在探討他們對藥物定價的預期時，我們沒有就定價給出任何具體指導，但我們從付款人那裡清楚地聽到的反饋是他們預計這看起來與其他罕見疾病藥物非常相似，這些藥物的定價為每位患者每年數十萬美元。

I think it's also important to remind people that patients that will start on this drug, we assume will more than likely stay on this drug for very, very long periods of time. And we've been seeing that now greater than 85% of patients in the ReDUX4 study are still on drug after a three-year period of time.

我認為提醒人們也很重要，我們認為開始服用這種藥物的患者很可能會繼續服用這種藥物非常非常長的一段時間。我們發現，現在 ReDUX4 研究中超過 85% 的患者在三年後仍在服用藥物。

So, once we get the REACH trial results, Greg, we'll go ahead and do more significant payer research. But to your question around the tailwind, and we spent a lot of time thinking about this. I think that one of the biggest tailwinds we'll have with payers is the requirement for a confirmed genetic test at the time or prior to the approval of the drug.

因此，格雷格，一旦我們獲得 REACH 試驗結果，我們將繼續進行更重要的付款人研究。但對於你關於順風的問題，我們花了很多時間思考這個問題。我認為我們對付款人最大的推動力之一是要求在藥物批准時或之前進行確認的基因測試。

And what we know right now from the work that we've done is only about 20% to 30% of patients are actually receiving a confirmed genetic test. So, if every payer we're operating under the assumption that payers will require that confirmed genetic test before approving the drug.

目前我們從所做的工作中了解到，只有大約 20% 到 30% 的患者實際上接受了確認的基因檢測。因此，如果我們的每個付款人都假設付款人在批准藥物之前需要經過確認的基因測試。

And so, we're doing a lot of work now. And there's a lot of examples out there of other rare diseases in which payers have required genetic tests. So, this is not a new concept. We're essentially sort of replicating the playbook of many other rare disease products that have come before us.

所以，我們現在正在做很多工作。還有很多其他罕見疾病的例子，付款人需要進行基因檢測。所以，這並不是一個新概念。我們本質上是在複製之前出現的許多其他罕見疾病產品的劇本。

But we will make sure at the time of launch that a lack of or we will make sure that genetic testing does not become an impediment to access and whether that's through partnership that we do with the FSHD society or whether we do it directly and provide genetic testing free of charge to patient. We're working through that right now to make sure at the time of launch, that does not become the tailwind that you mentioned.

但我們將在啟動時確保基因檢測的缺乏或我們將確保基因檢測不會成為獲取的障礙，無論是透過我們與 FSHD 協會的合作還是我們直接進行並提供基因檢測免費為患者進行檢測。我們現在正在解決這個問題，以確保在發佈時，這不會成為您提到的順風車。

That's helpful. Maybe related and as Alan has provided some detail on your runway. Just how are you coordinating the urgency about the planning for building a commercial organization when it comes to the leadership in such a capability and slotting that with respect to the data coming?

這很有幫助。也許相關，因為艾倫提供了一些關於你的跑道的細節。當涉及到這種能力的領導力並將其與即將到來的數據相結合時，您如何協調建立商業組織的規劃的緊迫性？

Yes, great question, Greg, and thanks for asking it. Yes. So, we are we have begun building out our commercial organization. I would expect that sometime in the third quarter of this year, we will announce the appointment of our Chief Commercial Officer.

是的，很好的問題，格雷格，感謝您提出這個問題。是的。所以，我們已經開始建立我們的商業組織。我預計我們將在今年第三季的某個時候宣布任命首席商務官。

And one of the things that I said to this individual, while I was interviewing with them is we will make sure that we properly resource this launch. We will make sure that if it's a question of do we fund it or do not fund it, we will always err on, we should fund it instead of not funding because we have to make sure that this is properly resourced. And as Alan mentioned in one of the earlier questions, the runway guidance that we have provided assumes a launch that we have very properly resourced beginning in 2026. Is that fair, Alan?

在採訪他們時，我對這個人說的一件事是，我們將確保為這次發布提供適當的資源。我們將確保，如果是我們資助還是不資助的問題，我們總是會犯錯誤，我們應該資助而不是不資助，因為我們必須確保有適當的資源。正如艾倫在之前的一個問題中提到的，我們提供的跑道指南假設我們從 2026 年開始就已經為發射提供了非常適當的資源。這樣公平嗎，艾倫？

Matthew Biegler of Op. Co.

作品的馬修·比格勒。公司。

We were curious if in your discussions with the FDA they'd ever asked for more data around the losmapimod's mechanism of action. As you mentioned, AVIDITY had some biomarker DUX4 reduction. clinically. I know preclinically you showed that, but you hadn't been able to clinically.

我們很好奇，在您與 FDA 的討論中，他們是否曾要求提供更多有關洛斯馬莫德作用機制的數據。正如您所提到的，AVIDITY 減少了一些生物標記 DUX4。臨床上。我知道你在臨床前已經證明了這一點，但你在臨床上卻未能做到。

So, I'm just kind of curious if you'd be open to rerunning archived biopsies from ReDUX using maybe a more sensitive assay if the FDA wanted you to? Or if not, if you think, really, at this point, the FDA is only concerned with validating the RWS tool?

所以，我只是有點好奇，如果 FDA 希望您這樣做，您是否願意使用更靈敏的檢測方法重新運行 ReDUX 中存檔的活檢？或者如果不是，您認為 FDA 目前只關心驗證 RWS 工具嗎？

Yes. Thanks, Matt. Great question. And let me turn that over to Iain as well.

是的。謝謝，馬特。很好的問題。讓我也把這個問題轉交給伊恩。

Matt, thanks. The issue of going back to biomarkers has not been something that's come up in any of discussions with the agency. The focus, I think, has very much been on the endpoints, given that it's a functional endpoint and the secondary endpoint similarly, are functional or related to muscle health. So, we haven't up to this point, received any specific requests around that. So, we don't have any specific plans to do so.

馬特，謝謝。在與該機構的任何討論中都沒有提到回歸生物標誌物的問題。我認為，重點主要集中在終點上，因為它是一個功能性終點，而次要終點同樣是功能性的或與肌肉健康相關的。因此，到目前為止，我們還沒有收到任何與此相關的具體請求。因此，我們沒有任何具體計劃。

The evidence that we have both in the original discovery of losmapimod as well as in the characterization of it was done in muscle cells derived from patients with FSHD, and you can perform the characterizations of the effect on DUX4 and all the downstream transcript pretty convincingly in that in vitro system where you don't have competition from other cell types and issues related to the biopsy and so on. And I think those data are very robust and show the mechanism of action on of losmapimod on the reductions of DUX4 and the downstream transcripts.

我們在洛斯馬莫德的最初發現及其表徵中所擁有的證據是在 FSHD 患者的肌肉細胞中完成的，您可以非常令人信服地表徵對 DUX4 和所有下游轉錄本的影響在體外系統中，您沒有來自其他細胞類型的競爭，也沒有與活檢等相關的問題。我認為這些數據非常可靠，並顯示了 Losmapimod 對 DUX4 和下游轉錄本減少的作用機制。

Okay. That makes sense. And maybe just squeeze one on the REACH 3 guidance. Is this a revision or just more fine-tuning from the prior guidance, which was year-end? And if it's a revision, kind of what are some of the factors driving that fit faster cadence going from year-end to now end of October.

好的。這是有道理的。也許只需在 REACH 3 指南中擠一擠即可。這是修訂版還是只是對去年年底的先前指導進行了更多微調？如果是修訂，那麼有哪些因素推動了從年底到十月底的更快節奏。

Yes, Matt, this is Alex. Yes. No, it's nothing more than simply fine-tuning. We as we're getting closer and we're seeing the last handful of patients having their last visit, we feel very confident in terms of our ability to report out the top line results by the end of October.

是的，馬特，這是亞歷克斯。是的。不，無非就是簡單的微調而已。隨著時間越來越近，我們看到最後幾名患者正在進行最後一次就診，我們對在 10 月底之前報告主要結果的能力非常有信心。

(Operator Instructions).

（操作員說明）。

There are no more questions in the queue. I would like to turn the call back to Alex for closing remarks. Please go ahead.

隊列中沒有更多問題。我想將電話轉回給亞歷克斯，讓他發表結束語。請繼續。

That's great. Thanks so much, Lisa. And again, thanks, everybody, for joining. Thanks to everyone for your interest in all the great things we're doing here at Fulcrum. And I guess before we conclude, I want to remind everyone that we continue to make progress with our Phase 1 PIONEER trial of pociredir, and are on track to report top-line data for the Phase 3 REACH trial by the end of October of this year.

那太棒了。非常感謝，麗莎。再次感謝大家的加入。感謝大家對我們在 Fulcrum 所做的所有偉大事情的關注。我想在我們結束之前，我想提醒大家，我們的 pociredir 第一階段 PIONEER 試驗繼續取得進展，並預計在今年 10 月底之前報告第三階段 REACH 試驗的主要數據年。

In parallel, we continue to prepare for the potential NDA filing and commercial launch of losmapimod in the US With our cash runway into 2027, we believe we are well positioned to execute on our corporate objectives and look forward to building on this momentum in the months and years ahead.

同時，我們繼續為洛斯馬莫德在美國潛在的NDA 備案和商業上市做準備。內繼續鞏固這一勢頭以及未來幾年。

And as I always like to do, before we jump off the call, I would be remiss if I did not extend my sincere appreciation and gratitude to my fellow Fulcrum teammates, to the physicians we work with to advance our clinical studies, to our partners at the FSHD society and finally and most importantly, to the patients and family and their families around the world. Without you and your commitment to become involved in clinical trials, none of what we do would be possible.

正如我一直喜歡做的那樣，在結束通話之前，如果我沒有向我的 Fulcrum 隊友、與我們一起推進臨床研究的醫生、我們的合作夥伴致以誠摯的讚賞和感謝，那我就失職了FSHD 協會，最後也是最重要的，是世界各地的患者及其家人和他們的家人。如果沒有您和您參與臨床試驗的承諾，我們所做的一切都是不可能的。

Thanks again to everyone who joined the call this morning. Please stay safe and healthy. Thanks so much.

再次感謝今天早上參加電話會議的所有人。請保持安全和健康。非常感謝。

Thank you for today's conference call. You all may disconnect.

感謝您參加今天的電話會議。你們都可以斷開連線。