Brainstorm Cell Therapeutics Inc (BCLI) 2023 Q3 法說會逐字稿

Greetings, and welcome to the BrainStorm Cell Therapeutics Third Quarter 2023 Earnings Call. (Operator Instructions)

您好，歡迎參加 BrainStorm Cell Therapeutics 2023 年第三季財報電話會議。 （操作員說明）

As a reminder, this call is being recorded. And I would now like to introduce your host for today's call, Michael Wood of LifeSci Advisors. Mr. Wood, you may begin.

提醒一下，此通話正在錄音。現在我想介紹今天電話會議的主持人，LifeSci Advisors 的 Michael Wood。伍德先生，您可以開始了。

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

早安，感謝大家加入我們。今天早些時候，BrainStorm 發布了一份新聞稿，公佈了 2023 年第三季的財務業績，其中包括公司最新情況。

Before passing it off to the company management for prepared remarks, I'd like to remind listeners on 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 disorders, such as ALS, the sufficiency of the company's existing capital resources for continuing operations in 2023 and beyond, the safety and clinical effectiveness of NurOwn technology platform, clinical trials of NurOwn and related clinical development programs, as well as the company's ability to develop strategic collaborations and partnerships to support its business planning efforts.

在將其傳遞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，以及有關治療ALS 等神經退化性疾病的市場潛力、公司現有資本資源是否足以在2023 年及以後持續運作、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，以及公司發展策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，包括該公司不時在 SEC 文件中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有重大差異，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 today morning will be Mr. Chaim Lebovits, President and CEO of BrainStorm; Dr. Stacy Lindborg, Co-Chief Executive Officer; and Alla Patlis, Interim Chief Financial Officer.

BrainStorm 總裁兼執行長 Chaim Lebovits 先生今天上午將加入我們的電話會議。 Stacy Lindborg 博士，聯合執行長；臨時財務長 Alla Patlis。

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

現在我想把電話轉給萊博維茨先生。請繼續，查姆。

Thank you, Michael. Thanks to all who have joined us to discuss our Q3 2023 financial results and ongoing progress. We are committed to our goal of making NurOwn available to the ALS community. Our priority now is to work with the FDA and do everything in our power to align in a path forward.

謝謝你，麥可。感謝所有與我們一起討論 2023 年第三季財務業績和持續進展的人。我們致力於實現為 ALS 社區提供 NurOwn 的目標。我們現在的首要任務是與 FDA 合作，盡我們所能，協調前進的道路。

As announced previously, we made the decision to withdraw the BLA for NurOwn from FDA review. This decision was made following the outcome of the Advisory Committee that took place to review NurOwn in September. This action was coordinated with the FDA and is used by the FDA as a withdrawal without prejudice. The next important step will be for us to conduct the meeting with the FDA. We submitted the official request just a few days ago. We will use the opportunity to discuss next steps, including the design for confirmatory Phase 3b study in ALS which we intend to conduct at such meeting.

正如先前所宣布的，我們決定從 FDA 審查中撤回 NurOwn 的 BLA。這項決定是根據諮詢委員會 9 月審查 NurOwn 的結果做出的。這項行動是與FDA協調的，並被FDA視為無偏見的撤回。下一個重要步驟是我們與 FDA 舉行會議。我們幾天前才提交了正式請求。我們將利用這個機會討論後續步驟，包括我們打算在此類會議上進行的 ALS 驗證性 3b 期研究的設計。

As part of our regulatory strategy, we intend to seek a Special Protocol Assessment with the FDA before continuing with the planned registration trial. The SPA indicates agreement with the agency and the adequacy and the ability of critical elements of the trial design, including items such as patient entry criteria, endpoints, and planned analyses. We believe that having a SPA in place will derisk the regulatory aspect of our ALS program. We believe the process will likely take one or two meetings with the FDA; we will provide an update once we have clarity on the path forward.

作為我們監管策略的一部分，我們打算在繼續計劃的註冊試驗之前尋求 FDA 的特別方案評估。 SPA 顯示與該機構的協議以及試驗設計關鍵要素的充分性和能力，包括患者進入標準、終點和計劃分析等項目。我們相信，制定 SPA 將會消除我們 ALS 計劃的監管方面的風險。我們相信這一過程可能需要與 FDA 舉行一兩次會議；一旦我們明確了前進的道路，我們將提供最新資訊。

In parallel with these regulatory activities, our management team and Board are getting clearer thoughts how to finance the company and ensure we have the resources to fund the planned safety trial and position ourselves for success in the future. We're actively exploring various options to raise capital, including non-dilutive grants, as well as capitalizing on certain non-core assets, such as our exosome technology.

在進行這些監管活動的同時，我們的管理團隊和董事會正在更加清晰地思考如何為公司提供資金，並確保我們有資源為計劃中的安全試驗提供資金，並為未來的成功做好準備。我們正在積極探索各種籌集資金的選擇，包括非稀釋性贈款，以及利用某些非核心資產，例如我們的外泌體技術。

In October, we announced a strategic realignment to prioritize and conserve resources. This involved a number of steps, including streamlining our clean room operations as well as a targeted reduction in headcount of approximately 30%. Importantly, those positions that are most critical to managing the planned Phase 3b trial and regulatory submission will be retained. We estimate that overall, the strategic realignment will cut total resource consumption by approximately 50%. Many of us, including all senior management, took a 30% cut in their wages; as for myself, I took over a 90% cut for now. These were difficult but necessary decisions.

十月份，我們宣布了一項策略調整，以優先考慮並節省資源。這涉及許多步驟，包括簡化我們的無塵室運作以及有針對性地減少約 30% 的員工人數。重要的是，那些對管理計劃中的 3b 期試驗和監管提交最關鍵的職位將被保留。我們預計，整體而言，策略調整將使總資源消耗減少約50%。我們很多人，包括所有高階管理人員，薪水都減少了 30%；至於我自己，我暫時接受了90%的削減。這些是困難但必要的決定。

We're very thankful for the hard work and commitment of all our colleagues at BrainStorm for their contributions in advancing NurOwn. I'll now turn the call over to Dr. Stacy Lindborg for additional comments.

我們非常感謝 BrainStorm 所有同事的辛勤工作和承諾，感謝他們為推進 NurOwn 所做的貢獻。我現在將把電話轉給 Stacy Lindborg 博士以徵求更多意見。

Thank you, Chaim. There remains an urgent need for new treatments for ALS, treatments that are safe and effective. Every day, patients and their families confront the harrowing reality of this terrible disease. We've assembled a considerable body of data on NurOwn in ALS participants and we continue to believe that if approved, this product has the potential to be a valuable new treatment option for people living with this disease.

謝謝你，查姆。仍然迫切需要新的 ALS 治療方法，即安全有效的治療方法。每天，患者及其家人都會面對這種可怕疾病的悲慘現實。我們已經收集了有關 NurOwn 在 ALS 參與者中的大量數據，我們仍然相信，如果獲得批准，該產品有可能成為患有這種疾病的人的一種有價值的新治療選擇。

We are motivated by the courage of ALS patients and we're driven by the opportunity to bring a product to market that we believe can improve their lives. We look forward to continuing to engage with the FDA and conducting a confirmatory Phase 3b trial as efficiently as possible. Chaim?

ALS 患者的勇氣激勵我們，將我們相信可以改善他們生活的產品推向市場的機會也激勵著我們。我們期待繼續與 FDA 合作並盡可能高效地進行驗證性 3b 期試驗。柴姆？

Thank you so much, Stacy. I'll not now ask Alla to discuss our financials. Alla?

非常感謝你，史黛西。我現在不會要求阿拉討論我們的財務狀況。阿拉？

Thank you, Chaim. Cash and cash equivalents and short-term bank deposits were approximately $1.4 million as of September 30, 2023, compared to $3 million as of December 31, 2022. In July 2023, the company raised net proceeds of approximately $7 million in the registered direct offering.

謝謝你，查姆。截至2023 年9 月30 日，現金和現金等價物以及短期銀行存款約為140 萬美元，而截至2022 年12 月31 日為300 萬美元。2023 年7 月，該公司在註冊直接發行中籌集了約700 萬美元的淨收益。

Research and development expenses for the three months ended September 30, 2023, and September 30, 2022, were approximately $3.3 million and $3.8 million, respectively. General and administrative expenses for the three months ended September 30, 2023, and September 30, 2022, were approximately $2.7 million and $3.1 million, respectively.

截至2023年9月30日和2022年9月30日的三個月的研發費用分別約為330萬美元和380萬美元。截至2023年9月30日和2022年9月30日的三個月的一般和管理費用分別約為270萬美元和310萬美元。

Net loss for the three months ended September 30, 2023, was approximately $1.2 million or $0.03 per share as compared to a net loss of approximately $6.9 million or $0.19 per share for the three months ended September 30, 2022.

截至2023年9月30日止三個月的淨虧損約為120萬美元或每股0.03美元，而截至2022年9月30日止三個月的淨虧損約為690萬美元或每股0.19美元。

I'll turn it back to Chaim.

我會把它轉回給Chaim。

Thank you very much, Alla. Michael Wood from LifeSci will now read some questions we have received from investors. Michael. Michael are you there?

非常感謝你，阿拉。 LifeSci 的邁克爾伍德現在將閱讀我們從投資者那裡收到的一些問題。麥可.麥可你在嗎？

Sorry, I was on mute. Yes. Thank you, Chaim.

抱歉，我當時處於靜音狀態。是的。謝謝你，查姆。

Is it possible that even with the ADCOM decision around NurOwn, the FDA could grant limited approval for early stage ALS patients where there has been an observable positive response?

即使 ADCOM 就 NurOwn 作出決定，FDA 是否有可能對已經出現明顯陽性反應的早期 ALS 患者給予有限的批准？

Stacy, do you want to take this?

史黛西，你想要這個嗎？

Sure. We firmly believe that generating additional data in the form of the Phase 3b trial that we've been discussing is the only path forward that will gain regulatory approval of NurOwn. We would have never withdrawn the BLA if we weren't convinced that we would not be successful with the current data alone. Therefore, it's in our best interest to move forward as quickly as we can to get the trial started.

當然。我們堅信，以我們一直在討論的 3b 期試驗的形式產生額外數據是獲得 NurOwn 監管部門批准的唯一途徑。如果我們不確信僅靠當前數據無法取得成功，我們就永遠不會撤回 BLA。因此，盡快開始試驗符合我們的最大利益。

Thanks, Stacy. Next question, how long before you know more about your funding plans? And as a follow up to that, when are you hoping to start the Phase 3b? And are you waiting for funding or grants to come in before you can commence the study?

謝謝，史黛西。下一個問題，您需要多長時間才能了解更多有關您的融資計劃？作為後續行動，您希望何時開始 3b 階段？您是否正在等待資金或補助金的到來才能開始研究？

Thank you. We're working with banks who are being successful, including very recently, raising funds for biotech companies like BrainStorm and they are confident in our ability to raise the funds needed to run this trial. We will first work to secure a bridge to ensure stability as a company as we work in parallel to secure funding for the Phase 3b trial. As we shared in our prepared remarks, we are exploring a variety of avenues including partnerships and grants. Although there's always the chance of a large infuse in the funds, we expect to raise money for the trial in stages. We also do not intend to wait to start the trial until we have raised the full amount needed. We'll be thoughtful about starting the trial, but patients deserve more urgency than this.

謝謝。我們正在與一些成功的銀行合作，包括最近為 BrainStorm 等生技公司籌集資金，他們對我們籌集這項試驗所需資金的能力充滿信心。我們將首先努力建立一座橋樑，以確保公司的穩定性，同時我們將同時努力確保 3b 階段試驗的資金。正如我們在準備好的演講中所分享的那樣，我們正在探索各種途徑，包括合作夥伴關係和贈款。儘管總有可能獲得大量資金注入，但我們希望分階段為試驗籌集資金。我們也不打算等到籌到所需的全部資金後才開始試驗。我們會深思熟慮地開始試驗，但患者應該比這更緊迫。

Thank you. Next question is you working on the new trial parameters with the FDA?

謝謝。下一個問題是您與 FDA 一起研究新的試驗參數嗎？

Stacy?

史黛西？

Thank you, that's correct. As I mentioned in the prepared remarks, we've requested an expedited face-to-face meeting very recently and this meeting will allow us to discuss the next steps for NurOwn which will focus on the core design elements of the Phase 3b trial that we've been working on with a scientific advisory panel and a panel of people from the ALS community including those living with the disease. We intend to seek a special protocol assessment from the FDA, which, as many of you will know, is an opportunity to reach an agreement with the FDA before the study starts on the design of a clinical trial. And this trial, of course, is intended to serve as the primary basis for an efficacy claim and a BLA. We view this as beneficial and a valuable tool to accelerate the Phase 3b trial for NurOwn and potentially derisk the regulatory aspects of the program.

謝謝，這是正確的。正如我在準備好的發言中提到的，我們最近要求召開一次加急面對面會議，這次會議將使我們能夠討論 NurOwn 的後續步驟，重點是我們所研究的 3b 期試驗的核心設計元素。一直在與科學顧問小組和ALS 社區（包括患有該疾病的患者）組成的小組合作。我們打算尋求 FDA 的特殊方案評估，正如你們許多人所知，這是在臨床試驗設計研究開始之前與 FDA 達成協議的機會。當然，這項試驗旨在作為功效聲明和 BLA 的主要基礎。我們認為這是加速 NurOwn 3b 期試驗的有益且有價值的工具，並可能消除該專案的監管方面的風險。

Thank you. And one final question, why don't you partner with big pharma?

謝謝。最後一個問題，為什麼不與大型製藥公司合作？

Thank you. So, we are seeking to partner. In fact, we're in contact with a few companies. Although ALS is a relatively rare disease, the commercial opportunity is still compelling. And we know there's interest from companies who would like to have a successful product in cellular and to be able to serve patients. You have to remember though that cell therapy is still an emerging modality, especially in neurodegeneration. There are very few approved cell therapy products and R&D products are largely focused on oncology and genetic disease. Presumably, a major approval in the software space will bring investment and until then, some will remain cautious.

謝謝。因此，我們正在尋求合作夥伴。事實上，我們正​​在與幾家公司聯繫。儘管 ALS 是一種相對罕見的疾病，但商業機會仍然引人注目。我們知道一些公司對此很感興趣，他們希望擁有成功的蜂窩產品並能夠為患者提供服務。但您必須記住，細胞療法仍然是一種新興的治療方式，尤其是在神經退化性疾病方面。核准的細胞治療產品很少，研發產品主要集中在腫瘤學和遺傳疾病領域。據推測，軟體領域的重大批准將帶來投資，在此之前，有些人將保持謹慎。

As you may know, it took many years to develop CAR T-cell therapy for various cancer treatments and innovation came from within smaller companies before developing partnerships. Just a few examples, Kite Pharma went alone until after approval and then was bought out by Gilead. Juno Pharma did the same and was also acquired after FDA approval by Celgene, while Bluebird wasn't acquired and remains independent today.

如您所知，開發用於各種癌症治療的 CAR T 細胞療法花了很多年的時間，在建立合作夥伴關係之前，創新來自於較小的公司內部。舉幾個例子，Kite Pharma 一直單打獨鬥，直到獲得批准後才被吉利德收購。 Juno Pharma 也做了同樣的事情，並在 FDA 批准後被 Celgene 收購，而 Bluebird 沒有被收購，至今仍保持獨立。

We have a derisked Phase 3 asset which will have an attractive upside for the right company. So, we will pursue a partnership as a promising avenue. NurOwn as a technology represents a strategic approach, and therefore we would welcome a partnership. But until that comes, we're willing to do all we can to proceed with the funds we are able to raise.

我們擁有已消除風險的第三階段資產，這對合適的公司來說將具有有吸引力的優勢。因此，我們將尋求合作夥伴關係作為一條有前途的途徑。 NurOwn 作為一項技術代表了一種策略方法，因此我們歡迎建立合作夥伴關係。但在那之前，我們願意盡一切努力繼續籌集我們所能籌集到的資金。

[Jenny], would you like to open the call for any additional questions?

[Jenny]，您想打開電話詢問其他問題嗎？

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

當然。 （操作員說明）David Bautz，Zacks 小型股研究公司。

Hi. Good morning, everyone. Thanks for the update this morning. So, Chaim or Stacey, I was wondering if you could talk a little bit more about the SPA. And basically, what differentiates that from saying, quote-unquote, coming to an agreement with the FDA about a trial design?

你好。大家，早安。感謝您今天早上的更新。 Chaim 或 Stacey，我想知道您是否可以多談談 SPA。基本上，這與說「引用-不引用」、與 FDA 就試驗設計達成協議有什麼區別？

Stacy?

史黛西？

Yes. David, I think that the SPA pathway and the opportunity to formally recognize and use a pathway that the FDA offers really just gives the platform to come together and to align, but to have a formal agreement that's documented and I think it's a constructive way to approach this and it's something that I think will be positive for BrainStorm but also really ensure that the discussion and the work that we do ahead ultimately is it nets out with the successful trial really what we expect, which would be an approval. So, it really is about a formal engagement and interaction and it's a pathway that's well established and leveraged.

是的。大衛，我認為 SPA 途徑以及正式認可和使用 FDA 提供的途徑的機會實際上只是提供了聚集和協調的平台，但有一個記錄在案的正式協議，我認為這是一種建設性的方式我認為這對腦力激盪來說是積極的，但也真正確保我們未來所做的討論和工作最終能夠實現我們所期望的成功試驗，這將是一個批准。因此，這確實是一種正式的參與和互動，並且是一條成熟且有效利用的途徑。

Okay. And as far as -- go ahead, sorry.

好的。至於——請繼續，抱歉。

Hello, I was just going to add on that. Of course, I just want to emphasize that FDA is discussing with us an expedited pathway for SPA -- so that's just additional answer to your question. What we're looking for expedited SPA, as you know, if those companies that are able to have a SPA with FDA, there's a far higher chance for approval at the end of the day. But many choose not to do that because usually it can take a year or two years just to get to an agreed upon SPA. This pathway is talking about an expedited SPA process. We will be looking forward for that.

你好，我只是想補充一下。當然，我只是想強調 FDA 正在與我們討論 SPA 的快速途徑——所以這只是對你的問題的補充答案。我們正在尋求加速 SPA，如您所知，如果那些能夠與 FDA 簽訂 SPA 的公司，最終獲得批准的機會要高得多。但許多人選擇不這樣做，因為通常需要一年或兩年的時間才能達成一致的 SPA。此途徑討論的是加速 SPA 流程。我們將對此充滿期待。

Go on and give me another question please.

請繼續問我另一個問題。

Yes. Actually, I was going to key off of what you just said, and basically kind of looking at the timeline. So normally that process would be a year or two, you're looking at less than a year probably then for an expedited SPA?

是的。事實上，我本來打算結束你剛才所說的話，基本上看看時間表。通常情況下，這個過程需要一兩年的時間，那麼您可能會考慮不到一年的時間來進行加急 SPA？

Well, I don't want to put any words in FDA quotes on here, so I want to be very cautious. But I do see a first meeting very fast. And I think after that meeting, we will be -- we'll share that by the way. We'll share what happened at the meetings with the investor community, because this is our number one priority just now to see if we can come to an agreed upon SPA. And we'll learn from this meeting. That should be very soon. And we'll be announcing when that meeting is.

好吧，我不想在這裡引用 FDA 的任何文字，所以我想非常謹慎。但我確實很快就看到了第一次會議。我認為在那次會議之後，我們將順便分享這一點。我們將與投資者社群分享會議上發生的事情，因為這是我們現在的首要任務，看看我們是否可以達成一致的 SPA。我們將從這次會議中學習。那應該很快了。我們將宣布會議的召開時間。

Okay. Now I understand you probably won't be able to get into specifics, but do you foresee the primary outcome for the next Phase 3 trial being the same as it was for the previous one?

好的。現在我知道您可能無法透露具體細節，但您是否預期下一個三期試驗的主要結果與上一個試驗相同？

You know, we're going to have a discussion with the agency at that meeting. So, let's keep it for that. We will share once those meetings are done, and we come to an agreement, we'll share what the trial was like.

你知道，我們將在這次會議上與該機構進行討論。所以，讓我們保留它。一旦這些會議結束，我們將分享審判的情況，並達成協議。

Okay, all right. Thanks for taking the questions.

好吧，好吧。感謝您提出問題。

Sure.

當然。

William Barr, Tedium Adventures.

威廉‧巴爾，《乏味的冒險》。

Thank you. Thank you, Chaim. Thank you, Stacey. And thanks to everyone at BrainStorm. We've been following and we've been investors in the company for many years. So, my question is, why is this time going to be different? There's like those of us who know you guys have been fighting, how hard you've been working, and all of us -- many of us are really so beyond grateful for everything that you've done and continue to do for ALS patients.

謝謝。謝謝你，查姆。謝謝你，史黛西。感謝 BrainStorm 的所有人。我們多年來一直在關注該公司，並且一直是該公司的投資者。所以，我的問題是，為什麼這次會有所不同？就像我們這些人一樣，知道你們一直在奮鬥，知道你們一直在努力工作，我們所有人——我們中的許多人真的非常感謝你們為 ALS 患者所做的和繼續做的一切。

We're finally starting to see that the Amylyx treatment is maybe not what some people thought it was. And we're finally starting to see that some ALS chapters are maybe recognizing the issues also and they're starting to break away. We all saw what happened to the ADCOM and many of us believe that it was extraordinarily unfair. A lot of investors want BrainStorm to sue the FDA. And instead, you guys are basically embracing the FDA. And we know you want a treatment for ALS as quickly as possible. And we know that you guys have really done everything in your power to try to effectuate that. So why is this time going to be different?

我們終於開始看到 Amylyx 治療方法可能不像某些人想像的那樣。我們終於開始看到一些 ALS 分會可能也意識到了這些問題，並且開始脫離。我們都看到了 ADCOM 所發生的事情，我們中的許多人都認為這是極其不公平的。許多投資者希望 BrainStorm 起訴 FDA。相反，你們基本上都在擁抱 FDA。我們知道您希望盡快治療 ALS。我們知道，你們確實已經竭盡全力來實現這一目標。那麼為什麼這次會有所不同呢？

Thank you. It's a very good question. So first, allow me -- I don't want to comment on other treatments or other organizations. We are looking to have everyone supporting us, all the organizations and all the advocates. And we're going to work on that as well. We're going to reach out once we have a trial design and share it with them, listen to their inputs going forward.

謝謝。這是一個非常好的問題。首先，請允許我——我不想評論其他治療方法或其他組織。我們希望每個人、所有組織和所有倡議者都支持我們。我們也將致力於此。一旦我們有了試驗設計並與他們分享，我們就會聯繫他們，並聽取他們的意見。

What I think will be different? You have heard in our opening statements, we're trying to come to an agreement with this fund and I want to share with you it is proposed by the FDA to do that, to have an expedited SPA. So, then we should see how they will treat us different going forward. And we should acknowledge that even though some see the ADCOM as being overly negative to NurOwn -- what we are being explained and I want to be cautious what I'm saying that -- but many don't disagree with what we are saying. They just feel we need to have more data to prove that.

我認為會有什麼不同？您在我們的開場白中聽說過，我們正在努力與該基金達成協議，我想與您分享 FDA 提議這樣做，即加快 SPA。因此，我們應該看看他們今後將如何對待我們。我們應該承認，儘管有些人認為 ADCOM 對 NurOwn 太消極——我們正在解釋的內容，我想謹慎地說——但許多人並不反對我們所說的話。他們只是覺得我們需要更多數據來證明這一點。

So therefore, they want to align on an agreed upon trial to provide that data. And therefore, that discussion together with them is very important to see what are they seeking in addition to what we provided? So, we should align on what should be provided, what should be sufficient for an approval? That's what a SPA means. So going into that meeting, I think we are going to get an expedited meeting. We are getting the indication from agency that they want to have this meeting very fast. So, they're showing interest and supporting us being able to have another trial very soon to talk operational. I'll ask Stacy also to comment. Stacy?

因此，他們希望就一項商定的試驗進行協調以提供該數據。因此，與他們一起討論非常重要，看看除了我們提供的之外，他們還尋求什麼？因此，我們應該就應該提供什麼、什麼足以獲得批准進行協調？這就是 SPA 的意思。因此，在會議開始時，我認為我們將召開加急會議。我們從機構得到指示，他們希望盡快召開這次會議。因此，他們表現出了興趣並支持我們能夠很快進行另一次試驗以討論可操作性。我也會請史黛西發表評論。史黛西？

Yes. William, thank you for the comment and your question. And I think when we step back and we look at what we have before us, certainly when we brought forward our product, we believe and we still believe in our data and we believe that there was a discussion that should happen around the efficacy and safety and the appropriateness, given regulatory flexibility. But at the end of the day, when we look forward, one of the strongest positions and the things that we can do is we can execute on a trial design, leveraging the learnings that have occurred and we can read out a trial that meets its endpoints.

是的。威廉，謝謝您的評論和問題。我認為，當我們退後一步，看看我們面前的情況時，當然，當我們提出我們的產品時，我們相信並且仍然相信我們的數據，我們相信應該圍繞功效和安全性進行討論以及考慮到監管靈活性的適當性。但最終，當我們展望未來時，最有力的立場和我們能做的事情之一就是我們可以執行試驗設計，利用已經發生的經驗教訓，我們可以讀出符合其要求的試驗。端點。

And so what you see us doing is really doubling down. We understand our product. We have a lot of information that we believe gives us a lot of confidence in our ability to deliver this trial. And so, what we're focused on is bringing forward a product that's straightforward in this next trial that makes the regulatory process certainly more conventional and the next time around, certainly much more streamlined and smoother. So that's what I think of when I think about what's different next time. And drug development is incredibly complicated. We always learn things in our endeavors when we're tackling very tough diseases. And I think in the setting of this last Phase 3 program, certainly there were things that the community did not appreciate and did not understand. And we now have those learnings that we take forward to this next trial design.

所以你看到我們所做的確實是加倍努力。我們了解我們的產品。我們擁有大量訊息，我們相信這些資訊讓我們對開展這項試驗的能力充滿信心。因此，我們的重點是在下一次試驗中推出一種簡單的產品，使監管流程肯定更加傳統，並且在下一次試驗中肯定會更加簡化和順利。所以當我思考下次有什麼不同時，我就是這麼想的。藥物開發極為複雜。當我們在處理非常棘手的疾病時，我們總是能在努力中學到東西。我認為在最後一個第三階段計劃的背景下，肯定有一些社區不欣賞和不理解的事情。我們現在已經掌握了這些知識，並將其運用到下一個試驗設計中。

Thank you very much. For those of us that have ALS patients in our lives, have lost family and friends, I just wanted to say thank you to both of you and thank you for fighting the good fight for us.

非常感謝。對於我們這些生活中患有 ALS 患者、失去家人和朋友的人，我只想向你們倆表示感謝，並感謝你們為我們而戰。

No, thank you for your ongoing support many years, really appreciate that.

不，感謝您多年來的持續支持，真的很感激。

Thank you very much.

非常感謝。

Next question please, Jenny. We have time for one more.

請下一個問題，珍妮。我們還有時間再來一場。

Daniel Walker, private investor.

丹尼爾·沃克，私人投資者。

On manufacturing, you mentioned on the recent BrainStorm town hall meeting that BrainStorm has successfully manufactured 500 potent products. But I think there have been some questions about BrainStorm's ability to manufacture. Can you just comment on that? And then also moving forward, how will the clean rooms be managed? Will it be Catalent? Will it be the academic centers? Maybe you could just comment a little bit on that.

關於製造，您在最近的 BrainStorm 市政廳會議上提到，BrainStorm 已成功製造了 500 種強效產品。但我認為人們對 BrainStorm 的製造能力有一些疑問。你能對此發表評論嗎？接下來，無塵室將如何管理？會是康泰倫特嗎？是學術中心嗎？也許你可以對此發表一點評論。

That's it. Thank you very much, Daniel, for this question. So, we did have quite a few centers where we really produced as you know for the Phase 2 and Phase 3 trials given the academic centers and the commercial centers are only for the EAP.

就是這樣。非常感謝丹尼爾提出這個問題。因此，我們確實有許多中心，如您所知，我們確實為第二階段和第三階段試驗進行了生產，因為學術中心和商業中心僅適用於 EAP。

Going forward, it's first a financial question. The academic centers are cheaper than the commercial center to operate. And on the other hand, you do want to have a site that once you're successful with the trial, you should have a commercial site ready. So, we're going to balance that timing wise. Maybe we'll start with academic and then for the second part of the trial do a commercial site so we can gain from the cheaper cost earlier and then simultaneously be able to have the commercial site ready once the trial is done. Does that answer your question?

展望未來，首先是財務問題。學術中心的營運成本比商業中心便宜。另一方面，您確實希望擁有一個網站，一旦您試用成功，您就應該準備好一個商業網站。因此，我們將明智地平衡時間。也許我們會從學術開始，然後在試驗的第二部分建立一個商業網站，這樣我們就可以更早地從更便宜的成本中獲益，然後在試驗完成後同時能夠準備好商業網站。這是否回答你的問題？

That does. And I guess with the SPA, how do you ensure that this go around, because the FDA was quite critical of the manufacturing at the ADCOM. How do you ensure moving forward, though, that there is alignment, not only on the SPA but also in addition to the manufacturing so that at the time you resubmit the BLA, they feel confident in the manufacturing because obviously there's the difference between manufacturing at the trial level compared to actually at the time of commercialization?

確實如此。我想對於 SPA，你如何確保這種情況順利進行，因為 FDA 對 ADCOM 的生產非常挑剔。不過，如何確保向前推進，不僅在 SPA 上，而且在製造方面也保持一致，以便在您重新提交 BLA 時，他們對製造充滿信心，因為顯然，製造之間存在差異試驗水平與商業化時的實際水平相比？

Thank you. That's very helpful. You clarified the question even more. So, the answer is, FDA agreed to have discussions with us while we do the trial on the same patients to come to alignment. And as I've said previously, we have come to alignment on many of the same issues, if not most, but some of them it takes longer to do those qualification tests, which we are continuing to do those that are critical. But yes, FDA will have with us separate conversations with the same team and separately from the clinical team.

謝謝。這非常有幫助。你把問題說得更清楚了。所以，答案是，FDA 同意在我們對相同患者進行試驗時與我們進行討論，以達成一致。正如我之前所說，我們在許多相同的問題上（如果不是大多數）達成了一致，但其中一些問題需要更長的時間來進行這些資格測試，而我們將繼續進行那些至關重要的測試。但是，FDA 將會與我們的同一團隊和臨床團隊分別進行單獨的對話。

Excellent. Thank you so much. And then just lastly, I was wondering if you could just expand on the exosome technology. I know you're working on capitalizing that. Can you or Stacy just comment very briefly on what is that technology? And why that might be interesting and compelling to pharma or biotech?

出色的。太感謝了。最後，我想知道你是否可以擴展外泌體技術。我知道你正在努力利用這一點。您或史黛西能否簡單地評論一下這項技術是什麼？為什麼這對製藥或生技公司來說可能是有趣且有吸引力的？

Yes. You mentioned Stacy, so I'll ask Stacy to talk to that, of course. And if I am needed, I'll add on. Stacy?

是的。你提到了史黛西，所以我當然會請史黛西談談這個問題。如果需要的話我會補充。史黛西？

Sure. Daniel, this is an additional technology that we have been developing and certainly have thought of as an interesting and exciting part of our portfolio. And what's interesting about exosome is, of course, that it allows us to move into and explore different diseases that can carry cargo and can penetrate different parts of human bodies, and therefore brings different advantages. But we've done a number of preclinical studies, some of which we've published. And we think there's a lot of excitement in this technology platform, which again offers a wide array of diseases that there's a great unmet need and that these nanoscale particles really would be able to open possible administration options that would be very effective for treatment.

當然。丹尼爾，這是我們一直在開發的附加技術，當然也被認為是我們產品組合中有趣且令人興奮的部分。當然，外泌體的有趣之處在於，它讓我們能夠進入和探索不同的疾病，這些疾病可以攜帶貨物，並且可以滲透到人體的不同部位，因此帶來不同的優勢。但我們已經做了許多臨床前研究，其中一些已經發表。我們認為這個技術平台有很多令人興奮的地方，它再次提供了多種疾病的巨大未滿足的需求，而這些奈米級顆粒確實能夠打開可能的治療選擇，這對治療非常有效。

Excellent. Thank you both so much. I really appreciate it.

出色的。非常感謝你們倆。對此，我真的非常感激。

Thank you.

謝謝。

Thank you very much. That appears to be the end of our question-and-answer session. I will now hand back over to Chaim for closing comments.

非常感謝。我們的問答環節似乎就這樣結束了。我現在將交回 Chaim 進行總結評論。

Thank you very much and have a wonderful day. Thank you.

非常感謝您，祝您有個愉快的一天。謝謝。

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

謝謝大家。今天的會議到此結束。此時您可以斷開電話線並度過美好的一天。感謝您的參與。

Thank you, Jenny. Bye.

謝謝你，珍妮。再見。

Thank you.

謝謝。