Brainstorm Cell Therapeutics Inc (BCLI) 2023 Q1 法說會逐字稿

Greetings, and welcome to the BrainStorm Cell Therapeutics first quarter 2023 earnings call. (Operator Instructions) As a reminder, this call is being recorded.

大家好，歡迎參加 BrainStorm Cell Therapeutics 2023 年第一季度財報電話會議。 （操作員說明）謹此提醒，此通話正在錄音。

I would now like to introduce your host for today's call, Michael Wood of LifeSci Advisors. Mr. Wood, you may begin.

現在我想介紹一下今天電話會議的主持人，LifeSci Advisors 的邁克爾·伍德 (Michael Wood)。伍德先生，您可以開始了。

Well, good morning and thank you for joining us. Earlier today, BrainStorm issued a press release with its financial results for the first quarter of 2023, including a corporate update.

嗯，早上好，感謝您加入我們。今天早些時候，BrainStorm 發布了一份新聞稿，公佈了 2023 年第一季度的財務業績，其中包括公司最新情況。

Before passing it over to the company management for prepared remarks, I want to remind listeners that this conference call will contain numerous statements, descriptions, forecasts, and projections regarding BrainStorm Cell Therapeutics and its potential future business operations and performance; statements regarding the market potential for the treatment of neurodegenerative diseases such as ALS, sufficiency of the company's existing capital resources for continuing operations in 2023 and beyond; the safety and clinical effectiveness of the NurOwn technology platform; clinical trials of NurOwn and related clinical development programs; and the company's ability to develop strategic collaborations and partnerships to support its business planning efforts.

在將其交給公司管理層進行準備好的發言之前，我想提醒聽眾，本次電話會議將包含大量有關 BrainStorm Cell Therapeutics 及其未來潛在業務運營和業績的聲明、描述、預測和預測；關於治療 ALS 等神經退行性疾​​病的市場潛力、公司現有資本資源是否足以在 2023 年及以後持續運營的聲明； NurOwn 技術平台的安全性和臨床有效性； NurOwn 的臨床試驗及相關臨床開發項目；以及公司發展戰略合作和夥伴關係以支持其業務規劃工作的能力。

Forward-looking statements are subject to numerous risks and uncertainties, many of which are beyond BrainStorm's control, including the risks and uncertainties described from time to time in the company's SEC filings. The company's results may differ materially from those projected on today's call, and the company undertakes no obligation to publicly update any forward-looking statements.

前瞻性陳述受到眾多風險和不確定性的影響，其中許多風險和不確定性超出了 BrainStorm 的控制範圍，包括該公司在 SEC 備案文件中不時描述的風險和不確定性。該公司的業績可能與今天電話會議中預測的結果存在重大差異，並且該公司不承擔公開更新任何前瞻性聲明的義務。

Joining us on the call this morning be Mr. Chaim Lebovits, President and CEO of BrainStorm; Dr. Stacy Lindborg, Co-Chief Executive Officer; and Alla Patlis, Interim Chief Financial Officer. In addition, the company's Executive VP and Chief Medical Officer, Dr. Kirk Taylor, is also on the call and will be available to answer your questions during the Q&A session.

今天早上加入我們電話會議的是 BrainStorm 總裁兼首席執行官 Chaim Lebovits 先生； Stacy Lindborg 博士，聯席首席執行官；臨時首席財務官 Alla Patlis。此外，公司執行副總裁兼首席醫療官 Kirk Taylor 博士也將接聽電話，並在問答環節回答您的問題。

So with that, I'd like to turn the call over to Mr. Lebovits. Please go ahead.

因此，我想將電話轉給萊博維茨先生。請繼續。

Thank you, Michael. Good morning, and thank you to all of who have joined us to discuss our Q1 2023 financial results and recent progress.

謝謝你，邁克爾。早上好，感謝所有與我們一起討論 2023 年第一季度財務業績和最新進展的人。

The most important recent development for BrainStorm was that the FDA notified us that there will be an advisory committee to discuss our BLA. We'll announce the date as soon as we know it.

BrainStorm 最近最重要的進展是 FDA 通知我們將成立一個諮詢委員會來討論我們的 BLA。我們一知道日期就會宣布。

We remain confident that holding an ADCOM will be the fastest and most appropriate way to fulfill our regulatory obligations and commitment to patients. It will ensure a full and publicly accessible discussion that includes FDA reviewers, company's experts, investigators, who conducted the clinical program, and input from all relevant stakeholders in the ALS community based on their respective expertise, experience, and point of view.

我們仍然相信，持有 ADCOM 將是履行我們的監管義務和對患者的承諾的最快、最適當的方式。它將確保進行全面且公開的討論，其中包括 FDA 審查員、公司專家、實施臨床計劃的研究人員，以及 ALS 社區中所有相關利益相關者根據各自的專業知識、經驗和觀點提出的意見。

BrainStorm firmly believes our data support regulatory approval of NurOwn. A thorough analysis of NurOwn Phase 3 data show evidence of clinically meaningful effectiveness, and we are further encouraged by the strong and consistent biomarker data, which are predictive -- sorry, we had a glitch, which are predictive of clinical response to the trial span pathways and important to ALS, neuroinflammation, neurodegeneration, neuroprotection, and allowing with NurOwn mechanism of actions.

BrainStorm 堅信我們的數據支持 NurOwn 的監管批准。對 NurOwn 3 期數據的徹底分析顯示了具有臨床意義的有效性的證據，強大且一致的生物標誌物數據進一步鼓舞了我們，這些數據具有預測性——抱歉，我們遇到了一個小問題，這些數據可以預測試驗跨度的臨床反應通路對 ALS、神經炎症、神經變性、神經保護很重要，並允許 NurOwn 作用機制。

I'll now turn the call over to Dr. Stacy Lindborg for a more detailed review of the NurOwn's dataset and highlight of our actions to support the BLA. This morning we are fortunate to be sitting in the same room, myself with Dr. Lindborg, and with Dr. Kirk Taylor.

現在，我將把電話轉給 Stacy Lindborg 博士，以便對 NurOwn 的數據集進行更詳細的審查，並重點介紹我們支持 BLA 的行動。今天早上，我們很幸運能夠與 Lindborg 博士和 Kirk Taylor 博士坐在同一個房間。

Thank you. Stacy, it's yours.

謝謝。史黛西，這是你的了。

Thank you, Chaim. As Chaim just highlighted, one of the reasons an ADCOM remains a critical step towards our goal of fulfilling our commitment to ALS patients relates to the complex scientific issues that need to be discussed and addressed. We have assembled a robust data set that will benefit from a deep and thoughtful discussion.

謝謝你，查姆。正如 Chaim 剛剛強調的那樣，ADCOM 仍然是我們實現對 ALS 患者承諾的關鍵一步的原因之一與需要討論和解決的複雜科學問題有關。我們已經收集了一個強大的數據集，該數據集將受益於深入而深思熟慮的討論。

The process for approval of any BLA will be determined primarily by the body of evidence generated. And on this front, we firmly believe that NurOwn's data package is strong enough to support regulatory approval. We are grateful to the FDA for the opportunity to review the full body of clinical evidence with all key stakeholders in the open and transparent setting provided by an ADCOM.

任何 BLA 的批准流程將主要由生成的證據決定。在這方面，我們堅信 NurOwn 的數據包足以支持監管部門的批准。我們感謝 FDA 給我們機會在 ADCOM 提供的公開透明的環境中與所有主要利益相關者一起審查全部臨床證據。

Since we first announced the top-line data from NurOwn's Phase 3 trial, we have conducted a number of additional focused analyses. These analyses provide valuable insights into the trial outcomes and have helped us better understand NurOwn's treatment effect.

自從我們首次公佈 NurOwn 第三階段試驗的主要數據以來，我們進行了許多額外的重點分析。這些分析為試驗結果提供了寶貴的見解，並幫助我們更好地了解 NurOwn 的治療效果。

As disclosed previously, the Phase 3 trial did not reach statistical significance on the primary and secondary endpoints. However, in a pre-specified group of participants with less advanced disease at baseline, there was a clinically meaningful treatment response on the primary and secondary endpoints with NurOwn compared to placebo.

正如之前所披露的，3 期試驗的主要和次要終點並未達到統計學顯著性。然而，在預先指定的基線時疾病進展程度較低的參與者組中，與安慰劑相比，NurOwn 在主要和次要終點上出現了具有臨床意義的治療反應。

On the secondary endpoint, which was favored by the FDA, the average change from baseline to week 28 in the ALS functional rating scale. The treatment difference was statistically significant at the level of P equal to 0.05. These findings are important as looking at this pre-specified subgroup enabled us to focus on data that is not as impacted by the floor effect in the scale.

FDA 青睞的次要終點是 ALS 功能評級量表從基線到第 28 週的平均變化。在 P 等於 0.05 的水平上，治療差異具有統計學顯著性。這些發現很重要，因為查看這個預先指定的子組使我們能夠專注於不受規模下限效應影響的數據。

Furthermore, post hoc sensitivity analyses of participants across baseline thresholds of 27 to 35 on the same secondary endpoint showed that NurOwn treated participants retain on average 2 points of function more compared to those who were on placebo. This is a clinically meaningful result, which represents important preservation of function and quality of life for people living with ALS, as well as their loved ones.

此外，對同一次要終點的基線閾值 27 至 35 的參與者進行事後敏感性分析表明，與服用安慰劑的參與者相比，接受 NurOwn 治療的參與者平均保留了 2 個功能點。這是一個具有臨床意義的結果，代表了 ALS 患者及其親人功能和生活質量的重要保留。

Additional post hoc analyses which account for the floor effect in different ways have been presented at scientific meetings, and each have demonstrated similar findings and an important treatment effect with NurOwn across study endpoints. All of these -- one of these analyses presented this year, in fact, very recently at the Muscular Dystrophy Association Clinical and Scientific Conference, focused on participants with no evidence of a floor effect at baseline.

其他以不同方式解釋地板效應的事後分析已在科學會議上提出，每項分析都證明了類似的發現以及 NurOwn 在整個研究終點上的重要治療效果。所有這些——事實上，今年最近在肌營養不良協會臨床和科學會議上提出的其中一項分析，重點關注基線時沒有證據表明存在底線效應的參與者。

The defining feature of this analyses was the ability for the scale to measure ongoing decline in all scale items at baseline, rather than focusing on participants' baseline values. This analyses hits at the heart of the problem based on the trial, which is a scale measurement issue. And the results from these analyses, first they included more than half of the participants in the trial, and these participants had baseline scores ranging from 25 up through 46.

該分析的決定性特徵是量表能夠衡量基線上所有量表項目的持續下降，而不是關注參與者的基線值。根據試驗分析，這擊中了問題的核心，即尺度測量問題。這些分析的結果是，首先他們納入了一半以上的試驗參與者，這些參與者的基線分數從 25 到 46 不等。

On the primary endpoint, the clinical response analyses. We observed a difference of 18% between treatments, with a 41 response rate with NurOwn and a 23% response rate and a p-value of 0.35.

在主要終點上，進行臨床反應分析。我們觀察到治療之間存在 18% 的差異，NurOwn 的緩解率為 41，緩解率為 23%，p 值為 0.35。

On the secondary endpoint, average change from baseline to week 28. NurOwn treated participants retained on average 2.3 points of function compared to placebo, again, with a significant p-value of 0.04.

在次要終點上，從基線到第 28 週的平均變化。與安慰劑相比，NurOwn 治療的參與者平均保留了 2.3 個功能點，同樣，顯著 p 值為 0.04。

The last piece of evidence that I'll comment on relates to our strong and consistent biomarker data generated in our Phase 3 trial. NurOwn's clinical program has the distinction of being the largest CSF biomarker study ever conducted in ALS. We identified biomarkers relevant to ALS pathology and rigorously analyzed them resulting in three important observations.

我要評論的最後一個證據與我們在第三階段試驗中生成的強大且一致的生物標誌物數據有關。 NurOwn 的臨床項目是有史以來針對 ALS 進行的最大規模的腦脊液生物標誌物研究。我們確定了與 ALS 病理學相關的生物標誌物，並對它們進行了嚴格分析，得出了三個重要的觀察結果。

Number one, treatment with NurOwn impacts three pathways important to ALS. Across these pathways, NurOwn treated participants have significant increases in markers of neuroprotection and decreases in markers of neuroinflammation and neurodegeneration over time compared to placebo. This means we achieved target engagement and confirmed the mechanism of action of NurOwn in Phase 3.

第一，NurOwn 治療會影響 ALS 的三個重要途徑。在這些途徑中，與安慰劑相比，隨著時間的推移，接受 NurOwn 治療的參與者的神經保護標記物顯著增加，而神經炎症和神經退行性變標記物隨著時間的推移而減少。這意味著我們在第三階段實現了目標參與並確認了 NurOwn 的作用機制。

Number two, we observed biological activity with NurOwn across these pathways, important to ALS in all NurOwn participants, including those with advanced ALS where the ALS functional rating scale demonstrated measurement challenges.

第二，我們觀察了 NurOwn 在這些途徑中的生物活性，這對所有 NurOwn 參與者的 ALS 很重要，包括那些患有高級 ALS 的人，其中 ALS 功能評級量表顯示了測量挑戰。

Number three, statistical modeling identified CSF biomarkers predictive of clinical outcomes observed in the Phase 2 trial following treatment with NurOwn using baseline and data following treatment with baseline with NurOwn. This was not true for placebo.

第三，統計模型使用基線和 NurOwn 基線治療後的數據確定了 CSF 生物標誌物，可預測 NurOwn 治療後 2 期試驗中觀察到的臨床結果。對於安慰劑來說情況並非如此。

So in summary, we can objectively show that the ALS functional rating scale was unable to measure ongoing decline in participants with the most advanced ALS who were enrolled in the trial, a unique sample of trial participants relative to other approved products.

總而言之，我們可以客觀地表明，ALS 功能評級量表無法衡量參加試驗的最晚期 ALS 參與者的持續下降，這是相對於其他批准產品的獨特的試驗參與者樣本。

Based on the data I've just walked you through, we have clinical and biomarker data that demonstrate evidence of significantly better outcomes in participants treated with NurOwn. And we have amassed efficacy and safety data that we believe supports a positive benefit risk evaluation of NurOwn. Lastly, we continue in our efforts to engage with neurologists, other important scientists, and with the patient advocacy community, as we continue through the regulatory process.

根據我剛剛向您介紹的數據，我們擁有臨床和生物標誌物數據，這些數據證明接受 NurOwn 治療的參與者的結果明顯更好。我們積累了療效和安全性數據，我們相信這些數據支持對 NurOwn 進行積極的效益風險評估。最後，在監管過程中，我們將繼續努力與神經科醫生、其他重要科學家以及患者權益團體進行接觸。

During the first quarter, I had the opportunity to present on NurOwn at the MDA Clinical and Scientific Meeting and at the Annual -- California Annual ALS Research Summit. We also continue to receive requests from organizations dedicated to ALS, requesting we present our data as they prepare to participate in our upcoming advisory committee meeting. I shared these examples so that you know that we are doing everything we can to engage with different parts of the ALS ecosystem.

在第一季度，我有機會在 MDA 臨床和科學會議以及加州年度 ALS 研究峰會上介紹 NurOwn。我們還不斷收到致力於 ALS 的組織的請求，要求我們在他們準備參加即將舉行的諮詢委員會會議時提供我們的數據。我分享這些例子是為了讓您知道我們正在盡一切努力與 ALS 生態系統的不同部分進行互動。

Now, I'll turn the call back to Chaim for some additional comments.

現在，我將把電話轉回 Chaim，徵求一些補充意見。

Thank you, Stacy. Based on our confidence in NurOwn's clinical data and ALS and the urgency at which broad access to new ALS therapies is needed, we recently began a targeted capability build to expand our medical, regulatory, and advocacy teams in preparation for anticipated growth. We want to be able to move quickly through the coming months, so that if we are successful in achieving approval for NurOwn, the wait for patients and families to gain access will be as short as possible.

謝謝你，史黛西。基於我們對 NurOwn 的臨床數據和 ALS 的信心以及需要廣泛獲得新的 ALS 療法的緊迫性，我們最近開始有針對性的能力建設，以擴大我們的醫療、監管和宣傳團隊，為預期的增長做好準備。我們希望能夠在未來幾個月內迅速採取行動，這樣，如果我們成功獲得 NurOwn 的批准，患者和家屬獲得准入的等待時間將盡可能短。

On May 1, we appointed Dr. Kirk Taylor, as Executive Vice President and Chief Medical Officer. Kirk has more than 26 years of experience in global drug development programs from Phase 1 through post-approval studies and across multiple therapeutic areas including neurology and rare diseases. He's joining BrainStorm from EMD Serono, where he's a Senior VP, North American Medical Affairs. He led the efforts of the medical team there and played an instrumental role in the launch of three new treatments.

5 月 1 日，我們任命 Kirk Taylor 博士為執行副總裁兼首席醫療官。 Kirk 在全球藥物開發項目（從一期到批准後研究）以及神經病學和罕見疾病等多個治療領域擁有超過 26 年的經驗。他在加入 BrainStorm 之前曾在 EMD Serono 擔任北美醫療事務部高級副總裁。他領導了當地醫療團隊的工作，並在三種新療法的推出中發揮了重要作用。

At BrainStorm, Kirk will lead the global medical affairs function and launch activities including planned product launches, post approval commercialization efforts, and deepening relationships with the medical community. We're very pleased to have someone with Kirk's capabilities and experience joining the team. I want to take this opportunity to formally welcome him to BrainStorm. He's joining us here on the call today for the Q&A session.

在 BrainStorm，Kirk 將領導全球醫療事務職能和發布活動，包括計劃的產品發布、批准後商業化工作以及深化與醫學界的關係。我們很高興擁有柯克這樣的能力和經驗的人加入團隊。我想藉此機會正式歡迎他加入頭腦風暴。他今天將參加我們的電話會議，參加問答環節。

There are also two additional hires I want to highlight. The first is the recent appointment of Antonio Trejo as VP Regulatory Affairs, and Robin Wallace as Vice President Global Clinical Operations. Each of them bring approximately 25 years of relevant experience.

我還想強調另外兩名員工。第一個是最近任命 Antonio Trejo 為監管事務副總裁，Robin Wallace 為全球臨床運營副總裁。他們每個人都擁有大約 25 年的相關經驗。

Antonio is recognized specifically for global regulatory expertise in specialty products, including in the areas of CNS, oncology, and rare diseases. He has worked with the regulatory agencies in the US, Canada, Japan and Israel, [in addition] to continue to countries across Latin America.

Antonio 因其在特殊產品（包括中樞神經系統、腫瘤學和罕見疾病領域）的全球監管專業知識而受到特別認可。他曾與美國、加拿大、日本和以色列的監管機構合作，[此外]還繼續與拉丁美洲國家合作。

Robin has a demonstrated track record of clinical operations leadership across drug, biologic, and device programs, both at large companies including Novartis, Merck, and Amgen, as well as smaller biotech companies. I'm excited to expand our team with these talented individuals and know they share our excitement around NurOwn's prospects. We believe having them in their new roles will prepare BrainStorm for the exciting future ahead.

Robin 在藥物、生物製劑和設備項目的臨床運營領導方面擁有良好的記錄，無論是在諾華 (Novartis)、默克 (Merck) 和安進 (Amgen) 等大型公司，還是小型生物技術公司。我很高興能夠與這些才華橫溢的個人一起擴大我們的團隊，並且知道他們與我們一樣對 NurOwn 的前景感到興奮。我們相信，讓他們擔任新角色將為 BrainStorm 做好準備，迎接令人興奮的未來。

Finally, there's one other topic I want to cover briefly and that is the granting by the FDA of accelerated approval of tofersen for the treatment of ALS in April. This is the second new drug to be approved for ALS in the space for seven months, the other being Amylyx's RELYVRIO in 2022.

最後，我想簡單介紹一下另一個主題，那就是 FDA 在 4 月份加速批准 tofersen 用於治療 ALS。這是七個月內第二個獲批用於 ALS 的新藥，另一個是 Amylyx 於 2022 年批准的 RELYVRIO。

We're obviously watching these developments with great interest and applaud the efforts of both sponsoring companies to bring new treatments to the ALS community. We're hardened by the regulatory flexibility that the FDA had shown by approving both of these new products for the treatment of ALS.

顯然，我們正以極大的興趣關注這些進展，並對兩家贊助公司為 ALS 社區帶來新療法的努力表示讚賞。 FDA 批准這兩種治療 ALS 的新產品，顯示出監管的靈活性，這讓我們更加堅定。

I'll now turn the call to Alla to discuss our financials. Alla?

我現在將打電話給阿拉，討論我們的財務狀況。阿拉？

Thank you, Chaim. It is my pleasure now to walk you through our first quarter financial results.

謝謝你，查姆。現在我很高興向您介紹我們第一季度的財務業績。

BrainStorm cash, cash equivalents, and short-term bank deposits were approximately $2.2 million as of March 31, 2023, this compares to $3 million on December 31, 2022. Our research and development expenditures for the three months ended March 31, 2023 and 2022 were approximately $2.9 million and $2.6 million, respectively.

截至2023年3月31日，BrainStorm現金、現金等價物和短期銀行存款約為220萬美元，而2022年12月31日為300萬美元。截至2023年3月31日和2022年3月31日止三個月的研發支出分別約為290萬美元和260萬美元。

General and administrative expenses for the three months ended March 31, 2023 and 2022, respectively, were $2.2 million and $2.9 million. Net loss for the three months ended March 31, 2023 was $5.1 million, or $0.14 per share, as compared to net loss of approximately $5.4 million, or $0.15 per share, for the three months ended March 31, 2022.

截至2023年3月31日和2022年3月31日的三個月的一般和管理費用分別為220萬美元和290萬美元。截至2023年3月31日止三個月的淨虧損為510萬美元，即每股0.14美元，而截至2022年3月31日止三個月的淨虧損約為540萬美元，即每股0.15美元。

I'll turn it back to Chaim to close the call.

我會將其轉回 Chaim 以結束通話。

Thank you, Alla. Michael, please if you can read the Q&A. And after you read the questions and we will give answers, we will then open -- the operator, Jenny, will then open the call for anyone on the line having additional questions. Thank you. Michael?

謝謝你，阿拉。邁克爾，請您閱讀問答。在您閱讀完問題並且我們將給出答案後，我們將接通——接線員珍妮將接通線路上任何有其他問題的人的電話。謝謝。邁克爾？

Yes. First question is do you have a firm date for the upcoming advisory committee?

是的。第一個問題是，即將召開的諮詢委員會有確定的日期嗎？

No, we don't yet have a firm date. And once we will have, we will share that with you.

不，我們還沒有確定的日期。一旦我們實現了，我們將與您分享。

Okay, thanks. Next question is regarding the Phase 3 study. Why did BrainStorm designed the Phase 3 to allow participants with advanced ALS unless the inability to measure their progress during the study?

好的謝謝。下一個問題是關於第三階段研究。為什麼 BrainStorm 設計第 3 階段時允許患有晚期 ALS 的參與者除非無法在研究期間衡量他們的進展？

Stacy?

史黛西？

Yeah. We've reflected on this question internally in addition with our esteemed clinicians who were PIs for the trial. The Phase 3 study design was being conceived in 2016 and was submitted to the FDA as part of our R&D in 2017. And the reality is the floor effect level associated with the ALS functional rating scale was not understood well by the industry at that time.

是的。我們在內部以及與作為該試驗的 PI 的尊敬的臨床醫生一起思考了這個問題。 3 期研究設計於 2016 年構思，並於 2017 年作為我們研發的一部分提交給 FDA。現實情況是，當時業界並沒有很好地理解與 ALS 功能評級量表相關的地板效應水平。

In fact, even in the early days following the database slot when we were first understanding that the scale was not able to measure the decline in participants with the most advanced disease, when we looked in the literature to document these phenomena, specifically with the ALS functional rating scale, we hardly could find any articles addressing it explicitly.

事實上，即使在數據庫發布後的早期，當我們第一次了解到該量表無法衡量患有最晚期疾病的參與者的下降時，當我們查閱文獻記錄這些現象時，特別是 ALS功能評級量表，我們幾乎找不到任何明確討論它的文章。

We found articles that referenced an insensitivity at the lower end of the scale, but no one had ever had a large enough sample of participants with advanced ALS such that the floor confounded treatment estimates. So the level wasn't established. We know this is no longer the case. In fact, every trial being designed today is taking this into account. And even ongoing trials are amending their analyses plans to include analyses to minimize the floor effect.

我們發現一些文章提到了量表低端的不敏感性，但沒有人擁有足夠大的晚期 ALS 參與者樣本，以至於下限混淆了治療估計。所以等級沒有建立。我們知道情況已不再如此。事實上，今天設計的每項試驗都考慮到了這一點。甚至正在進行的試驗也在修改他們的分析計劃，以包括盡量減少地板效應的分析。

In R&D, as a biotech industry, we know it's not for the faint of heart and it requires the ability to constantly learn. It's not unusual to learn something new in a trial, and the steps you need to go through when you do are well understood.

在研發方面，作為生物技術行業，我們知道這不適合膽小的人，它需要不斷學習的能力。在試驗中學習新東西並不罕見，而且您需要執行的步驟也很容易理解。

First, you need to carry out all analyses as originally described and share them openly. Second, you define what was observed in the trial that was not anticipated. And third, you produce analyses that appropriately address for what was observed. We've done each of these steps. And I guess in some sense, our learning -- the fact that our learning can very objectively be measured and displayed is a blessing.

首先，您需要按照最初描述的方式進行所有分析並公開分享。其次，您定義試驗中觀察到的未預期的內容。第三，您進行適當地解決觀察到的問題的分析。我們已經完成了每個步驟。我想從某種意義上說，我們的學習——我們的學習可以非常客觀地衡量和展示這一事實是一種祝福。

At the end of the day, what we are facing is a scale measurement problem, which we can address and bring forward evidence of a positive benefit risk evaluation of NurOwn that we believe warrants approval.

歸根結底，我們面臨的是規模衡量問題，我們可以解決這個問題，並提出我們認為值得批准的 NurOwn 積極效益風險評估的證據。

Thank you so much. Michael?

太感謝了。邁克爾？

Next question. Does BrainStorm believe that it must have a biomarker analyses published in order to be successful at the ADCOM? And then there's a related question. You've been talking about the new biomarker manuscript for a long time now. What have been the challenges again that's published?

下一個問題。 BrainStorm 是否認為必鬚髮表生物標誌物分析才能在 ADCOM 上取得成功？然後還有一個相關的問題。您已經談論新的生物標誌物手稿很長時間了。再次發布的挑戰是什麼？

Thank you. Stacy?

謝謝。史黛西？

Do we believe it needs to be published, to be successful in ADCOM? No, we don't believe this is necessary. But we do think that the more data we publish, the more informed the community will be. From a status perspective, the manuscript is in the hands of all the authors as we speak for a final review and then it will be submitted.

我們是否認為它需要發布才能在 ADCOM 中取得成功？不，我們認為沒有必要。但我們確實認為，我們發布的數據越多，社區的信息就會越豐富。從狀態的角度來看，稿件是在所有作者手中，我們會進行最終審查，然後將其提交。

What I can tell you is that it's a rigorous paper through which we've generated valuable insights into the pathology of ALS that will aid in the development of future therapies, and we're optimistic it will be published in advance with ADCOM. But obviously, this is out of our control.

我可以告訴您的是，這是一篇嚴謹的論文，我們通過它對 ALS 的病理學產生了寶貴的見解，這將有助於未來療法的開發，我們樂觀地認為它將提前與 ADCOM 一起發表。但顯然，這是我們無法控制的。

Thank you.

謝謝。

Our next question, are you willing to partner with a large cap biopharmaceutical company to accelerate clinical trials for ALS and for other indications?

我們的下一個問題是，您是否願意與一家大型生物製藥公司合作，加速 ALS 和其他適應症的臨床試驗？

Well, we're always willing to entertain partnerships that are in the best interest of the company and further our ability to accelerate development of treatments of patients in need for sure.

嗯，我們總是願意建立符合公司最大利益的合作夥伴關係，並進一步增強我們加速開發有需要的患者的治療方法的能力。

Thanks. One more question. This is actually for Dr. Kirk Taylor. This investor said he was pleased to read the press release announcing Kirk's hiring as Chief Medical Officer. Kirk, can you share some perspective on exactly what motivated you to join BrainStorm?

謝謝。還有一個問題。這實際上是給柯克·泰勒博士的。這位投資者表示，他很高興閱讀宣布任命柯克為首席醫療官的新聞稿。 Kirk，您能分享一下您加入 BrainStorm 的具體動機嗎？

Thank you, Michael. Appreciate it. Yes, the unmet medical need and the possibility of bringing a new treatment to the ALS community were key motivators for me to join BrainStorm. Also, I'm honored to join the world-class team of researchers and biotech professionals that Chaim and Stacy have assembled. We're onto great things. Thank you.

謝謝你，邁克爾。欣賞它。是的，未滿足的醫療需求以及為 ALS 社區帶來新療法的可能性是我加入 BrainStorm 的關鍵動力。此外，我很榮幸能夠加入 Chaim 和 Stacy 組建的世界一流的研究人員和生物技術專業人士團隊。我們正在做偉大的事情。謝謝。

Thanks Kirk. That concludes the inbound questions.

謝謝柯克。入站問題到此結束。

Thank you so much, Michael. Jenny, would you open the call for questions and also advise participants on the call how they can ask?

非常感謝你，邁克爾。珍妮，您可以開始提問並建議電話參與者如何提問嗎？

(Operator Instructions) David Bautz, Zacks Small-Cap.

（操作員說明）David Bautz，Zacks Small-Cap。

Hey, good morning, everybody.

嘿，大家早上好。

Good morning.

早上好。

Quickly on the ADCOM and the potential date. How much lead times does the FDA usually give between announcing when the meeting is going to be and when it actually occurs?

快速了解 ADCOM 和潛在日期。 FDA 在宣布會議召開時間和實際召開會議之間通常需要多長時間？

David, it's a very good question. And through this, I like to take this opportunity to dive in a little bit more into this. Many are asking this from us offline, not actually for this call only. So the FDA has a right to give this ADCOM between -- until -- it has to be before the PDUFA date, obviously. And we don't know yet that this will be priority review or not a priority review.

大衛，這是一個非常好的問題。通過這個，我想藉此機會更深入地探討這一點。許多人在線下向我們詢問這個問題，而不僅僅是為了這次電話會議。因此，FDA 有權在——直到——之間提供 ADCOM，顯然，它必須在 PDUFA 日期之前。我們還不知道這將是優先審查還是不是優先審查。

We do understand that when -- the process we went through that it may be somewhere in the middle. I can tell you that the FDA is actively working on ADCOM date, they are discussing with us dates. So it's happening. It's just we're waiting to have the final confirmative date. And once we have that, we'll announce that. So I can tell you it's going to be in the next few months. It's quite clear.

我們確實明白，當我們經歷的過程中，它可能處於中間的某個位置。我可以告訴你，FDA 正在積極制定 ADCOM 日期，他們正在與我們討論日期。所以它正在發生。我們只是在等待最終確認日期。一旦我們有了這個，我們就會宣布。所以我可以告訴你這將在接下來的幾個月內發生。很清楚。

Okay. Now, at the ADCOM, there's always an open public hearing where essentially anyone can come, and I'll definitely say, testify? Does the company have any control over who gets to speak at that portion of the meeting?

好的。現在，在 ADCOM，總是會舉行公開聽證會，基本上任何人都可以參加，我肯定會說，作證？公司是否可以控制誰可以在會議的該部分發言？

Well, usually no. Everyone has a right to register for that meeting and speak.

嗯，通常不會。每個人都有權註冊參加該會議並發言。

Okay.

好的。

I'm sure you're familiar with that, David.

我相信你對此很熟悉，大衛。

Okay. And lastly, what is the company's strategy for advancing NurOwn in other jurisdictions? And do you think that another clinical trial is going to be necessary to get approval outside the US?

好的。最後，公司在其他司法管轄區推進 NurOwn 的戰略是什麼？您認為是否需要進行另一項臨床試驗才能在美國境外獲得批准？

Well, we look all the time at other jurisdictions. We now have an add-on in the team as you've heard. We have Antonio and he's also really working on that as well. He has a lot of experience, but to be very clear, we're very focused now on the advisory committee meeting here. We're going to come as strong as we can, and we anticipate getting an approval here albeit with a confirmatory trial probably. But we'll be able to discuss that in a later time.

嗯，我們一直在關注其他司法管轄區。正如您所聽說的，我們現在團隊中有一個附加組件。我們有安東尼奧，他也正在為此努力。他擁有豐富的經驗，但需要明確的是，我們現在非常關注這裡的諮詢委員會會議。我們將盡我們所能，我們預計會在這裡獲得批准，儘管可能需要進行驗證性試驗。但我們稍後可以討論這個問題。

Okay, sounds good. Thanks for taking my questions.

好的聽起來不錯。感謝您回答我的問題。

Thank you so much, David.

非常感謝你，大衛。

[Dan Michael], Private Investor.

[丹·邁克爾]，私人投資者。

Can you address your finances with only $2.2 million of cash left at the end of March? You should be out of money by about now.

3 月底只剩下 220 萬美元現金，你能解決財務問題嗎？你現在應該沒錢了。

Well, we are not. Just if you can look at the end of December, we also had a similar number. We were able to bring in through some block deals, institutional investors, and they give us support. We just don't want to use the ATM at these prices. We're waiting for an opportunity where we will use the ATM.

好吧，我們不是。如果你看看 12 月底，我們也有類似的數字。我們能夠通過一些大宗交易引入機構投資者，他們給了我們支持。我們只是不想以這些價格使用 ATM。我們正在等待使用 ATM 的機會。

We have $100 million facility with Ray J and Leerink. And we're just waiting opportunistically how to use that -- the time being (multiple speakers)

我們與 Ray J 和 Leerink 擁有 1 億美元的資金。我們只是在機會主義地等待如何使用它——暫時（多個發言者）

So at the moment you're using debt?

那麼目前您正在使用債務嗎？

No, we didn't use debt. We had institutional blocks coming in and we did -- through the ATM do a block share.

不，我們沒有使用債務。我們有機構區塊進來，我們確實通過 ATM 進行了區塊共享。

Okay. Thank you.

好的。謝謝。

Yeah. And we have zero debt, no convertible, no debt on our books. Thank you, sure.

是的。我們的債務為零，沒有可轉換債券，賬面上沒有債務。謝謝你，當然。

(Operator Instructions) Okay. We don't appear to have any more questions. I'm going to hand back over to --

（操作員指示）好的。我們似乎沒有更多問題了。我將把事情交還給——

Maybe wait one more second, Jenny.

也許再等一下，珍妮。

Oh, yeah. Okay. No problem. [Richard Robbins], Private Investor.

哦耶。好的。沒問題。 [理查德·羅賓斯]，私人投資者。

Good morning. I realized that the focus is on the upcoming ADCOM. But in your press release this morning, you indicated that you're working on next-generation drugs. Could you please expand on that?

早上好。我意識到重點是即將推出的 ADCOM。但在今天早上的新聞稿中，您表示您正在研究下一代藥物。您能詳細說明一下嗎？

Definitely. So our research and development team are working on different products and on different diseases. As you all know, we have a pipeline even for NurOwn, outside of ALS for MS, Parkinson's, Huntington's, and other diseases. At the same time, we have an access on product, which are working for other diseases as well. We will announce once we have that, but of course, we have a huge R&D team working on that.

確實。因此，我們的研發團隊正在研究不同的產品和不同的疾病。眾所周知，除了 ALS 之外，我們甚至還有針對 NurOwn 的產品線，用於治療多發性硬化症、帕金森病、亨廷頓舞蹈症和其他疾病。同時，我們還可以獲得對其他疾病也有效的產品。一旦確定，我們就會宣布，當然，我們有一個龐大的研發團隊致力於此。

Thank you.

謝謝。

Very welcome.

非常歡迎。

(Operator Instructions) It don't appear to have anybody else join the queue, Chaim.

（操作員指示）Chaim，似乎沒有其他人加入隊列。

No problem. Thank you very much, Jenny, for this call. Thanks everyone for being with us this morning. We are geared and fired up for the ADCOM and we'll see you on the next Q, hopefully, with very good news for you guys. Thank you very much.

沒問題。珍妮，非常感謝您打來這個電話。感謝大家今天早上和我們在一起。我們已為 ADCOM 做好準備並充滿熱情，我們將在下一個問題中與您見面，希望能為大家帶來好消息。非常感謝。

Thank you, everybody. This does conclude today's conference call. You may disconnect your lines at this time and have a wonderful day. Thank you for your participation.

謝謝大家。今天的電話會議到此結束。此時您可以斷開線路並度過美好的一天。感謝您的參與。