Cellectis SA (CLLS) 2025 Q2 法說會逐字稿

Good day, everyone, and welcome to today's Cellectis second quarter 2025 earnings conference call. (Operator Instructions) Please note this call may be recorded, and I'll be standing by should you need any assistance.

大家好，歡迎參加今天的 Cellectis 2025 年第二季財報電話會議。（操作員指示）請注意，此通話可能會被錄音，如果您需要任何幫助，我會隨時待命。

It is now my pleasure to turn the conference over to Arthur Stril, Interim Chief Financial Officer. Please go ahead.

現在我很高興將會議交給臨時財務長 Arthur Stril。請繼續。

Good morning, and welcome, everyone, to Cellectis' second quarter 2025 business update and financial results conference call. Joining me on the call today are Dr. Andre Choulika, our Chief Executive Officer; and Dr. Adrian Kilcoyne, our Chief Medical Officer.

早安，歡迎大家參加 Cellectis 2025 年第二季業務更新與財務業績電話會議。今天與我一起參加電話會議的還有我們的執行長 Andre Choulika 博士和我們的首席醫療官 Adrian Kilcoyne 博士。

Yesterday evening, Cellectis issued a 6-K and press release reporting our financial statements for the six month period ended June 30, 2025, and a business update. The report and press release are available on our website at cellectis.com.

昨天晚上，Cellectis 發布了一份 6-K 報告和新聞稿，報告了截至 2025 年 6 月 30 日的六個月財務報表和業務更新。報告和新聞稿可在我們的網站 cellectis.com 上查閱。

As a reminder, we will make statements regarding Cellectis' financial outlook, including the presentation of our BALLI-01 and NatHaLi-01 clinical trials, the timing and ability to progress our clinical trial into a later phase, the progress of our R&D activities under the AstraZeneca partnership, the timing and outcome of our arbitration with Servier, and sufficiency of cash to fund operations.

提醒一下，我們將對 Cellectis 的財務前景發表聲明，包括我們的 BALLI-01 和 NatHaLi-01 臨床試驗的介紹、將我們的臨床試驗推進到後期階段的時間和能力、我們與阿斯特捷利康合作下的研發活動的進展、我們與 Servier 仲裁的時間和結果，以及資助的現金充足性。

These forward statements, which are based on our management's current expectations and assumptions and on information currently available to management, including information provided or otherwise publicly reported by our licensed partners, are subject to risk and uncertainties that may cause actual results to differ from those forecasted. A description of these risks can be found in our most recent Form 20-f filed with the Security Exchange Commission, SEC, and the financial report, including the management report, for the year ended on December 30, 2024, and subsequent filings Cellectis makes for the SEC from time to time.

這些前瞻性聲明基於我們管理層當前的預期和假設以及管理層當前可獲得的信息，包括我們的授權合作夥伴提供或以其他方式公開報告的信息，存在風險和不確定性，可能導致實際結果與預測結果不同。這些風險的描述可以在我們向美國證券交易委員會 (SEC) 提交的最新 20-f 表格、截至 2024 年 12 月 30 日的財務報告（包括管理報告）以及 Cellectis 不時向 SEC 提交的後續文件中找到。

I would now like to turn the call over to Andre.

現在我想把電話轉給安德烈。

Thank you, Arthur. Good morning, and thank you, everyone, for joining us today. I'd like to begin this call with an important announcement. On October 16, 2025, Cellectis will be hosting an Investors R&D Day in New York City. Our leadership team, along with key opinion leaders, will present the Phase 1 data set and late-stage development strategy for lasme-cel UCART22 in relapsed or refractory acute lymphoblastic leukemia. And we'll share insights on the company's vision and differentiated capabilities.

謝謝你，亞瑟。早安，感謝大家今天加入我們。我想以一項重要公告來開始這通通話。2025 年 10 月 16 日，Cellectis 將在紐約市舉辦投資者研發日。我們的領導團隊將與關鍵意見領袖一起展示 lasme-cel UCART22 治療復發或難治性急性淋巴性白血病的第 1 期資料集和後期開發策略。我們將分享有關公司願景和差異化能力的見解。

Cellectis Investors R&D Day is scheduled as an in-person only event. However, the recording of the event will be made available in the following days after the event.

Cellectis 投資者研發日計畫作為僅限現場參加的活動。不過，活動的錄音將在活動結束後的幾天內提供。

Despite the challenges of the biotech markets, our teams have remained focused on advancing research and developing solutions for patients with unmet medical needs. In July 2025, Cellectis is completed end of phase one discussion with both the United States Food and Drug Administration and the European Medicine Agency. We're excited to prepare for the initiation of the pivotal Phase 2 trial for lasme-cel UCART22 in relapsed or refractory acute lymphoblastic leukemia in the second half of this year.

儘管生物技術市場面臨挑戰，我們的團隊仍專注於推進研究並為未滿足醫療需求的患者開發解決方案。2025 年 7 月，Cellectis 完成與美國食品藥物管理局和歐洲藥品管理局的第一階段討論。我們很高興為今年下半年啟動 lasme-cel UCART22 治療復發或難治性急性淋巴性白血病的關鍵性 2 期試驗做好準備。

Regarding the NatHaLi-01 study, which is assessing eti-cel product, UCART20x22, in relaxed or refractory, non-Hodgkin lymphoma, Cellectis anticipates presenting data from Phase 1 and outlining its latest development strategy late 2025.

關於 NatHaLi-01 研究，該研究正在評估 eti-cel 產品 UCART20x22 在緩解性或難治性非何杰金氏淋巴瘤中的療效，Cellectis 預計將在 2025 年底提供第 1 階段的數據並概述其最新的發展策略。

On the partnership front, research and development activities are ongoing under the three cell and gene therapy programs under our joint research and collaboration agreement with AstraZeneca: one allergenic CAR-T for hematological malignancies, one allergenic CAR-T for solid tumors, and one in vivo gene therapy for genetic disorder.

在合作方面，我們與阿斯特捷利康簽訂的聯合研究與合作協議下的三個細胞和基因治療項目正在進行研發活動：一個用於治療血液系統惡性腫瘤的過敏性 CAR-T 計畫、一個用於治療實體瘤的過敏性 CAR-T 計畫、以及一個用於治療遺傳疾病的體內基因治療計畫。

Regarding our licensing agreement involving Servier and sublicensee Allogene, and following Servier's decision in September 2022 to seize the development of the licensed CD19 products, we've initiated an arbitration before the Paris Mediation and Arbitration Center to protect our interests. We're asking the tribunals to terminate the agreement with Servier and to award fair compensation for the losses incurred due to the lack of development of the licensed products and the payment of the milestone, which we consider due under the agreement. The arbitral decision is expected to be rendered on December 15, 2025, or before.

關於我們與施維雅及其分許可人 Allogene 之間的許可協議，以及在施維雅於 2022 年 9 月決定停止開發許可的 CD19 產品後，我們已向巴黎調解仲裁中心提起仲裁，以保護我們的利益。我們要求法院終止與施維雅的協議，並對因許可產品開發不足而造成的損失以及我們認為根據協議應得的里程碑付款給予公平的賠償。仲裁裁決預計於2025年12月15日或之前作出。

Earlier this quarter, Cellectis announced that during its Annual Shareholders Meeting, Mr. Andre Muller has been appointed as a member of the company's Board of Directors. I'm pleased to welcome Andre at Cellectis Board. His extensive experience will be an invaluable asset to the company.

本季度早些時候，Cellectis 宣佈在其年度股東大會上，Andre Muller 先生已被任命為公司董事會成員。我很高興歡迎 Andre 加入 Cellectis 董事會。他豐富的經驗將成為公司的寶貴資產。

We would also like to express our gratitude to Mr. Pierre Bastid and Mr. Axel-Sven Malkomes, who have terminated the directorship, for their esteemed commitment to Cellectis. It has been a huge honor and a pleasure for us all to work with them during their term. Their contribution over the past years has been exceptional, and their precious support has greatly contributed to the advancement of the company's strategy.

我們也要向已終止董事職務的 Pierre Bastid 先生和 Axel-Sven Malkomes 先生表示感謝，感謝他們對 Cellectis 的崇高承諾。在他們的任期內與他們共事是我們莫大的榮幸和快樂。他們多年來的貢獻非常卓越，他們的寶貴支持為公司策略的推進做出了巨大貢獻。

With that, I would like to turn the call over to Dr. Adrian Kilcoyne, our Chief Medical Officer, who will give an overview of our clinical trials. Adrian, please go ahead.

接下來，我想將電話轉給我們的首席醫療官 Adrian Kilcoyne 博士，他將概述我們的臨床試驗。阿德里安，請繼續。

Thank you, Andre. As Andre mentioned, Cellectis continues to focus its development efforts on the BALLI-01 and NatHaLi-01 studies. Recruitment to the dose escalation component of the Phase 1 BALLI-01 study, which is evaluating UCART22 in relapsed refractory B cell acute lymphoblastic leukemia, has been completed. This study addresses an important unmet need for patients who have relapsed following multiple prior lines of therapy, including a CD19 bispecific or autologous CAR-T and a few, if any, other treatment options. The Phase 1 data set has been shared with both FDA and EMA as part of the end of Phase 1 and scientific advice meetings.

謝謝你，安德烈。正如安德烈所提到的，Cellectis 繼續將其開發工作重點放在 BALLI-01 和 NatHaLi-01 研究上。第一階段 BALLI-01 研究的劑量遞增部分的招募已經完成，該研究正在評估 UCART22 在復發難治性 B 細胞急性淋巴性白血病的療效。這項研究解決了那些在接受過多種療法（包括 CD19 雙特異性或自體 CAR-T 以及一些（如果有的話）其他治療方案）後復發的患者的一個重要的未滿足需求。作為第一階段結束和科學建議會議的一部分，第一階段數據集已與 FDA 和 EMA 共享。

Following productive interactions with both agencies, we now have a clear path to commence our pivotal Phase 2 study later this year. We have set up additional trial sites in order to accelerate accrual into the Phase 2 study, and we will continue to focus on expanding sites in the United States and Europe, including the United Kingdom.

在與兩個機構進行富有成效的互動之後，我們現在有了一個明確的方向，可以在今年稍後開始我們關鍵的第二階段研究。我們已經建立了額外的試驗地點，以加速進入第二階段的研究，我們將繼續專注於擴大美國和歐洲（包括英國）的試驗地點。

We anticipate having sites open for recruitment into our Phase 2 study by the end of the year. We are also planning to publicly share the full Phase 1 dose escalation data set during our R&D day as highlighted by Andre earlier. Additional data from the Phase 1 study has also been submitted for consideration of presentation at the ASH Annual Conference in the fourth quarter.

我們預計今年年底前將開放站點招募第二階段研究的參與者。正如安德烈之前所強調的那樣，我們還計劃在研發日期間公開分享完整的第一階段劑量遞增資料集。第一階段研究的附加資料也已提交，以供考慮在第四季度的 ASH 年度會議上展示。

We also continue to enroll in the NatHaLi-01 study of our dual CAR-T asset, UCART20x22 in relapsed or refractory non-Hodgkin's lymphoma. This study is addressing an important unmet needs for patients who have relapsed following previous lines of therapy, including when available, an autologous CD19 CAR-T. Data from this program has also been submitted for presentation at the ASH Annual Conference in the fourth quarter. We are expanding our clinical trial sites to accelerate recruitment, and we hope to transition to Phase 2 preparation in 2026.

我們也繼續參與我們的雙 CAR-T 資產 UCART20x22 在復發或難治性非何杰金氏淋巴瘤中的 NatHaLi-01 研究。這項研究正在解決接受過先前療法（包括自體 CD19 CAR-T 療法）後復發的患者的一個重要的未滿足需求。該計劃的數據也已提交給第四季的 ASH 年度會議進行展示。我們正在擴大臨床試驗地點以加快招募速度，並希望在 2026 年過渡到第二階段的準備工作。

With that, I would like to hand the call over to Arthur Stril, selected as Chief Financial Officer and Chief Business Officer, for an overview of our financials for the second quarter of 2025. Arthur, please go ahead.

接下來，我想將電話交給被任命為財務長兼首席商務官的 Arthur Stril，以概述我們 2025 年第二季的財務狀況。亞瑟，請繼續。

Thank you, Adrian. We are pleased about the progress of our wholly owned product candidates, lasme-cel and eti-cel, as well as over the three cell and gene therapy programs in partnership with AstraZeneca. In this context, we are excited to be hosting an R&D Day on October 16 to focus on the Phase 2 data set and late-stage development strategy of lasme-cel as we prepare to launch a pivotal Phase 2 in H2 2025. We also expect to provide an update on eti-cel by the end of the year.

謝謝你，艾德里安。我們對我們的全資候選產品 lasme-cel 和 eti-cel 以及與阿斯特捷利康合作的三個細胞和基因治療計畫的進展感到高興。在此背景下，我們很高興於 10 月 16 日舉辦研發日，重點關注 lasme-cel 的第 2 階段資料集和後期開發策略，為 2025 年下半年啟動關鍵的第 2 階段做準備。我們也預計將在今年年底前提供 eti-cel 的最新消息。

Finally, the arbitral decision in our arbitration with Servier is expected to be rendered on or before December 15, 2025. Importantly, we are well positioned financially to execute on our pipeline as our cash, cash equivalents, and fixed-term deposits as of June 30, 2025, remain sufficient to fund our operations into H2 2027.

最後，我們與施維雅的仲裁預計將於 2025 年 12 月 15 日或之前作出裁決。重要的是，我們在財務上處於有利地位，可以執行我們的管道，因為截至 2025 年 6 月 30 日，我們的現金、現金等價物和定期存款仍然足以為我們到 2027 年下半年的營運提供資金。

Our cash, cash equivalents, restricted cash, and fixed-term deposits classified as current and non-current financial assets as of June 30, 2025, amounts to $230 million compared to $264 million as of December 31, 2024. This $33.2 million decrease is mainly due to $13.4 million of cash in from our revenue and $5.1 million of interest income, offset by cash payments from Cellectis to suppliers of $23.2 million; Cellectis' wages, bonuses, and social expenses paid of $23.6 million; the payments of lease debt of $5.4 million; and the repayment of the PGE loan of $2.6 million. You are invited to refer to our press release for figures related to consolidated net loss attributable to shareholders of Cellectis for the six months ended June 30, 2025.

截至 2025 年 6 月 30 日，我們的現金、現金等價物、受限現金和定期存款（歸類為流動和非流動金融資產）總額為 2.3 億美元，而截至 2024 年 12 月 31 日為 2.64 億美元。這筆 3,320 萬美元的減少主要是由於我們收入中的 1,340 萬美元現金和 510 萬美元利息收入，但被 Cellectis 向供應商支付的 2,320 萬美元現金、Cellectis 支付的 2,360 萬美元工資、獎金和社會費用、540 萬美元租賃債務以及支付的 2,360 萬美元工資、獎金和社會費用、540 萬美元的租賃債務以及支付 260 萬美元的貸款。請您參閱我們的新聞稿，以了解截至 2025 年 6 月 30 日的六個月內歸屬於 Cellectis 股東的合併淨虧損相關數據。

We're very much looking forward to welcoming you at our Investor R&D Day as well as to providing further updates later this year.

我們非常期待在投資者研發日上歡迎您的到來，並在今年稍後提供進一步的更新。

And now I would like to turn the call over to Andre for closing remarks.

現在我想把電話交給安德烈做結束語。

Thank you, Arthur. To close out this call, I would like to reiterate how excited we are to have one of our first products moving into Phase 2 pivotal trial powered registration and confident about the continued progress of our ongoing clinical trials in hematological malignancies, as well as how excited we are about our strategic collaboration with AstraZeneca.

謝謝你，亞瑟。在結束本次電話會議之前，我想重申一下，我們對我們的首批產品之一進入第二階段關鍵試驗註冊感到非常興奮，並且對我們在血液系統惡性腫瘤領域正在進行的臨床試驗的持續進展充滿信心，以及我們對與阿斯利康的戰略合作感到非常興奮。

At Cellectis, we strongly believe that our product candidates, our technologies, and our in-house manufacturing capabilities will lead us and our partners to paradigm shift for patients with hard-to-treat cancer and genetic disorders, positioning us at the forefront of this promising medical and scientific field.

在 Cellectis，我們堅信我們的候選產品、我們的技術和我們的內部製造能力將引領我們和我們的合作夥伴為患有難治性癌症和遺傳疾病的患者帶來範式轉變，使我們處於這個充滿希望的醫學和科學領域的前沿。

With that, I'd like to open the call for Q&A.

現在，我想開始問答環節。

(Operator Instructions) Gina Wang, Barclays.

（操作員指示）巴克萊銀行的吉娜·王 (Gina Wang)。

Thank you for taking my questions, and congrats on the progress. So I have two questions. One is regarding the Servier or arbitration decision by December 15. Could you contextualize the different scenarios and your likely actions?

感謝您回答我的問題，並祝賀您的進展。我有兩個問題。一個是關於 12 月 15 日之前 Servier 或仲裁的決定。您能否將不同的場景和您可能採取的行動具體化？

The second question is regarding the lasme-cel. You already met with both FDA and EMA regarding the pivotal Phase 2 trial design. Could you share a bit high-level thoughts with us? What could be the path forward there?

第二個問題是關於 lasme-cel 的。您已經與 FDA 和 EMA 會面討論關鍵的第 2 階段試驗設計。能與我們分享一些高層次的想法嗎？那裡的前進之路是什麼？

Hi, Gina. This is Andrea speaking. I'd like to answer the first question. It's complicated to answer your question for the simple reason that, I guess, that probably they're still thinking on the way that things are going to go. And I don't want to draw here any kind of scenario, knowing that any kind of scenario can happen from one side or another. And I personally hope that we're going to prevail in this arbitration, it means that we're going to get back our CD19 products and that we're going to have the compensation for like the loss incurred by the non-development by Servier.

你好，吉娜。我是安德里亞。我想回答第一個問題。回答你的問題很複雜，原因很簡單，我猜，他們可能仍在思考事情的發展方向。我不想在這裡描繪任何情景，因為我知道任何情景都可能從某一方面發生。我個人希望我們能夠在這次仲裁中獲勝，這意味著我們將拿回我們的 CD19 產品，並且我們將獲得因施維雅未開發而造成的損失的補償。

However, it's very difficult to forecast exactly what could be the decision at the end in this type of arbitration. So I would like to keep the door open as much as possible for any kind of scenarios. But we believe that we're totally right in our position knowing that there's nothing has been happening so far.

然而，很難準確預測此類仲裁的最終結果。因此，我希望盡可能為各種情況保持開放態度。但我們相信，我們的立場是完全正確的，因為到目前為止還沒有發生任何事情。

A question mark on -- if I start like putting some scenario, it means like I already thought about the fact that we can have some back position, but we don't have any back position. And for the question for like the interaction with the FDA and EMA, I would like to defer this to Adrian. Adrian?

一個問號——如果我開始提出一些場景，那就意味著我已經想到了我們可以有一些後備位置的事實，但我們沒有任何後備位置。至於與 FDA 和 EMA 互動的問題，我想將其交給 Adrian 來回答。阿德里安？

Yes. Thanks, Andre. Great question. Yes, we've interacted with both EMA and FDA. And just in the spirit of transparency, the EMA, it was a written feedback. They felt our submission was clear and therefore, they could give us very clear guidance on that and a very productive feedback that was.

是的。謝謝，安德烈。好問題。是的，我們與 EMA 和 FDA 都有互動。本著透明的精神，EMA 給出了書面回饋。他們認為我們的意見很明確，因此他們可以給我們非常明確的指導和非常有益的回饋。

The FDA was a face-to-face meeting. And well, I can't go into the details, but certainly at the R&D Day on October 16, we will share a lot more detail around the study design, et cetera. But a few top-level take homes is we got clear agreement on end points. We had -- there was no concerns raised around our statistical plan. There was no issues raised about the size of the database we would have as we go to BLA with -- based on what we had submitted.

FDA 是一次面對面的會議。嗯，我不能透露細節，但在 10 月 16 日的研發日上，我們肯定會分享更多有關研究設計等方面的細節。但一些高層的收穫是，我們在終點上達成了明確的一致。我們的統計計劃沒有引起任何擔憂。根據我們提交的內容，我們在提交 BLA 時，對於資料庫的大小沒有任何疑問。

So overall, in our view, there is a very clear path forward for our Phase 2 program. So again, I'd reiterate, very productive meetings with both regulatory authorities who are generally aligned in their feedback, which is always a bonus, I feel.

因此，總體而言，我們認為我們的第二階段計劃的前進道路非常明確。因此，我再次重申，與兩個監管機構的會談非常富有成效，他們的回饋基本上一致，我認為這總是一個額外的好處。

Thank you.

謝謝。

Kelly Shi, Jefferies

Kelly Shi，傑富瑞

Hi. This is Anqi Yu on behalf of Kelly Shi from Jefferies. Thank you for taking our questions. I have two questions. For the R&D Day, what data points should we expect? And supposing we don't have the trial design at this point, you will disclose on the R&D Day, then how do you consider the dynamic in the FDA that could potentially have any impacts on the pivotal trials? Thank you.

你好。我是 Jefferies 的 Kelly Shi 代表 Anqi Yu。感謝您回答我們的問題。我有兩個問題。對於研發日，我們應該期待哪些數據點？假設我們目前還沒有試驗設計，您將在研發日揭露，那麼您如何看待 FDA 的動態可能會對關鍵試驗產生影響？謝謝。

Good morning. This is Arthur. I will start on the R&D Day and then let Adrian add any points and also on the FDA question. So the purpose of the R&D Day is really twofold. We want to be presenting the full Phase 1 data set for lasme-cel that will include all patients that have been dosed so far and a particular focus on the patients at the recommended Phase 2 dose, and that will be safety, efficacy, and durability data set.

早安.這是亞瑟。我將從研發日開始，然後讓 Adrian 補充任何要點以及 FDA 問題。因此，研發日的目的其實有雙重。我們希望展示 lasme-cel 的完整第 1 階段資料集，其中將包括迄今為止已接受劑量治療的所有患者，並特別關注接受建議第 2 階段劑量治療的患者，這將是安全性、有效性和耐久性資料集。

And the second point is, we want to present what we call the late-stage development strategy, which indeed will include the Phase 2 design, the patient population, and then provide some color on the path to BLA. So it will be both a look into the path that all the Phase 1 set, but also look into the future as to the plans for the product all the way to commercialization.

第二點是，我們想要提出所謂的後期開發策略，它確實將包括第 2 階段的設計、病患群體，然後為 BLA 的道路提供一些色彩。因此，它不僅會審視第一階段設定的所有路徑，還會展望產品直至商業化的未來計畫。

And then, I'll hand it over to Adrian, if you want to add any color and address the FDA question.

然後，如果您想添加任何細節並解決 FDA 問題，我會將其交給 Adrian。

Yeah. I mean, I think, if I can answer with a question. Are there any barriers that became evident in our interactions with either regulatory authorities to progressing to Phase 2 and pivotal? Absolutely not. I think the regulatory authorities, both of them acknowledged the high unmet need that exists within the space, we're going -- positioning that product. But also, I think it was very clear from the tone of these meetings that they're broadly supportive of what we're doing.

是的。我的意思是，我想，如果我可以用一個問題來回答的話。在我們與監管機構的互動中，是否有任何阻礙我們進入第二階段和關鍵階段的障礙？絕對不是。我認為監管機構都承認該領域存在大量未滿足的需求，我們將定位該產品。但同時，我認為從這些會議的基調中可以清楚地看出，他們普遍支持我們所做的事情。

So again, to get alignment on endpoints, to get alignment broadly on the study design as we had presented it to them and patient numbers, overall, we don't see any significant roadblocks. So again, alignment between ourselves and the regulatory authorities, and again, as Arthur said, we will be presenting the study design in detail, discussing through those endpoints.

因此，再次強調，為了在終點上取得一致，為了在研究設計上取得廣泛一致，正如我們向他們展示的那樣，以及患者人數，總體而言，我們沒有看到任何重大障礙。因此，再次強調，我們與監管機構之間要保持一致，正如亞瑟所說，我們將詳細介紹研究設計，並透過這些終點進行討論。

But I think, again, I would restate that the registration path for this product seems relatively clear. And of course, everything is dependent on ultimately Phase 2 data.

但我想，我再次重申，該產品的註冊路徑似乎相對清晰。當然，一切最終都取決於第二階段的數據。

Great. Thank you.

偉大的。謝謝。

Jack Allen, Baird.

傑克艾倫，貝爾德。

Great. Thanks so much for taking the questions, and congratulations on progress. I guess maybe I'll start with one on UCART22 and the upcoming R&D Day. I wanted to ask what -- how the team is thinking about the bar for success in relapsed or refractory. I know there's some data about best response out there and also some autologous CD19 CAR-Ts. But as you move toward the pivotal study, what are you looking at as the bar for success?

偉大的。非常感謝您回答這些問題，並祝賀您取得進展。我想也許我會從 UCART22 和即將到來的研發日開始。我想問一下——團隊如何看待復發或難治性治療的成功標準。我知道有一些關於最佳反應的數據以及一些自體 CD19 CAR-T。但是，當您進行關鍵研究時，您認為成功的標準是什麼？

And what kind of expectations do you have for durability of response from UCART22? And how much follow-up should we expect on Phase 1 patients when we get that data update in the back half of this year and then, I guess, in mid-October? And then I have a quick follow-up as well.

您對 UCART22 的反應持久性有何期望？當我們在今年下半年以及十月中旬獲得數據更新時，我們應該對第一階段患者進行多少後續？然後我也會進行快速的跟進。

Thanks, Jack, for the great questions. Adrian, do you want to take this?

謝謝傑克提出的這些精彩問題。阿德里安，你想拿這個嗎？

Sure. So there's many questions in there. Suffice to say, again, more detail on the endpoints, the timing of those endpoints will be shared in detail during the October 16 R&D Day. But durability of response in the allogeneic setting is really important. And on sharing the data with both regulatory authorities, they are very clear on the approach we're taking will give you the adequate data in order to support a registration, assuming the data is positive based on our -- what we're showing.

當然。這裡面有很多問題。再次強調，有關端點的更多細節以及這些端點的時間將在 10 月 16 日研發日期間詳細分享。但在同種異體環境中反應的持久性確實很重要。在與兩個監管機構共享數據時，他們非常清楚我們採取的方法將為您提供足夠的數據以支持註冊，假設根據我們所展示的數據是積極的。

Of course, this is also, there will be a longer-term follow-up in all these. You know we're committed to a 15-year follow-up with these patients. But also within the trial, we will have a longer survival. It's a long overall survival follow-up for a number of years. But that is not part of the primary analysis. So the primary analysis, as we will share, is a much more short-term surrogate endpoint, which, again, has been agreed with the regulatory authorities.

當然這也是，這一切都會有更長期的後續。您知道我們致力於對這些患者進行 15 年的追蹤。但在試驗期間，我們的生存期也會更長。這是一項為期數年的長期整體存活追蹤。但這不是主要分析的一部分。因此，正如我們將要分享的，主要分析是一個更短期的替代終點，這一點也已得到監管機構的同意。

Yeah, that's very helpful. And then maybe for Adrian, again, with ALLO, correct it, as he sees fit. I'd love to hear any high-level thoughts you have on the recent decision by Allogene to move away from the CD52 lymphodepletion. How are you thinking about that as it relates to your programs moving forward? And would you anticipate including alemtuzumab in a potential pivotal study of UCART22?

是的，這非常有幫助。然後，也許對於 Adrian 來說，可以再次使用 ALLO 按照他認為合適的方式進行修正。我很想聽聽您對 Allogene 最近決定放棄 CD52 淋巴球清除療法的看法。您如何看待這與您今後的計劃有關？您是否預期會將阿崙單抗納入 UCART22 的潛在關鍵研究中？

Well, I can start on that, Arthur and Andre. Yeah, we've been following the Allogene story very closely. We believe that alemtuzumab is really important as part of the lymphodepletion regimen. And we want to be really cautious in how we interpret our approach in the context of Allogene's approach. I want to stress that these are very different positionings of the products. We're talking very late-stage ALL, and we're talking very late stage NHL compared to the Allogene approach is much earlier.

好吧，亞瑟和安德烈，我可以從這個開始。是的，我們一直在密切關注 Allogene 的故事。我們相信阿崙單抗作為淋巴球清除療法的一部分確實非常重要。我們希望在 Allogene 方法的背景下謹慎地解釋我們的方法。我想強調的是，這些產品的定位非常不同。我們討論的是晚期 ALL，我們討論的是晚期 NHL，相較之下異基因治療方法要早得多。

While we -- and we also don't know very much around the pharmacokinetics of the ALLO product. But it appears to be at a much higher dose than we gave within our clinical trials. And of course, the third thing, which is really, really important is that everything we do is based on risk benefits. So the risk-benefit assessment within the Allogene program is very different to the risk-benefit assessment we see in our programs.

同時，我們對 ALLO 產品的藥物動力學也了解不多。但它的劑量似乎比我們在臨床試驗中給出的劑量高得多。當然，第三件事，也是非常非常重要的一點，就是我們所做的一切都是基於風險利益。因此，Allogene 計劃中的風險效益評估與我們在我們的計劃中看到的風險效益評估非常不同。

So all in all, very difficult. I think it would be unfair to draw any comparisons between these programs. We are fairly confident about our risk-benefit assessment across our programs with alemtuzumab. And we believe that adds a critical improvement in responses, which we're fairly confident in our approach moving forward.

總而言之，非常困難。我認為對這些項目進行比較是不公平的。我們對阿崙單抗專案的風險效益評估非常有信心。我們相信這對回應產生重大改善，我們對我們未來的方法充滿信心。

Yeah, and if I can add something, I think, Adrian, you're spot on obviously on the on the dose, but it's also important to remember that these are different products. And I think our strategy has always been from the get-go to secure direct access to alemtuzumab, which is something that we've done with no fee a few years back, so we know we have access to actual alemtuzumab.

是的，如果我可以補充一點的話，我想，阿德里安，你顯然在劑量方面說得很對，但同樣重要的是要記住這些是不同的產品。我認為我們的策略從一開始就是確保直接獲得阿崙單抗，幾年前我們已經免費實現了這一點，因此我們知道我們可以獲得真正的阿崙單抗。

ALLO-647 is a different product. And honestly, we're not exactly aware as to how it compares, what are the glycosylation patterns, what is the structure of the antibody. And so it's very difficult to compare apples to oranges. And we're very pleased to be moving forward with actual alemtuzumab.

ALLO-647 是一種不同的產品。老實說，我們並不完全清楚它是如何比較的，糖基化模式是什麼樣的，抗體的結構是什麼樣的。因此，很難將蘋果和橘子進行比較。我們非常高興能夠推進阿崙單抗的實際研究。

Great, thank you so much. I guess just maybe one last one on the topic, any high-level thoughts on ways you can mitigate potential infection risk in your study? I know the CD52 lymphodepletion can lead to a longer depletion of T cells. Have you given any further thought on the protocols you can put in place to mitigate infection risk?

太好了，非常感謝。我想也許關於這個主題還有最後一個問題，您對如何在研究中減輕潛在的感染風險有什麼高層次的想法嗎？我知道 CD52 淋巴球耗竭會導致 T 細胞更長的耗竭。您是否進一步考慮過可以採取哪些措施來降低感染風險？

Yeah. I mean, it's part of all our trials. We have a mandatory prophylaxis. So that's already built in. We have significant risk mitigation strategies already built into our trials. So yeah, I think we've already addressed much of that. But of course, we remain vigilant.

是的。我的意思是，這是我們所有試驗的一部分。我們有強制性預防措施。所以這已經是內建的了。我們的試驗中已經納入了重要的風險緩解策略。是的，我認為我們已經解決了大部分問題。但當然，我們仍保持警戒。

Got it. Great. Thank you so much for taking the questions, and congratulations again on the progress.

知道了。偉大的。非常感謝您回答這些問題，並再次祝賀您的進展。

Yanan Zhu, Wells Fargo Securities.

朱亞南，富國證券。

Great. Thanks for taking our questions. Just maybe a follow-up on the FDA discussion for the BALL program. Wondering -- I know you will provide more details at the R&D Day. Wondering if the population for the pivotal trial, is that specific population like post CD19 CAR-T? Or is that a more broader population?

偉大的。感謝您回答我們的問題。也許只是對 FDA 關於 BALL 計劃討論的後續。想知道—我知道您將在研發日提供更多細節。想知道關鍵試驗的族群是否是像 CD19 CAR-T 後的特定族群？或者說是更廣泛的人群？

And also in terms of the patient number, could we look to the most recent CD19 autologous CAR-T programs for BALL for the rough range? Or could the trial be smaller than that? Thanks.

另外，就病患數量而言，我們能否查看最新的 CD19 自體 CAR-T 計畫的 BALL 大致範圍？或者試驗規模可以更小嗎？謝謝。

Yeah. Good questions. So as you're probably aware, we're looking at a very late-stage treatment. But we're going for a fairly broad patient population in terms of age cutoff. And again, we'll share the details with that, we would anticipate there will be a significant number of lines of therapy for most patients in our clinical trial. And that will include, obviously, CD19, CARs, et cetera. So you'll see detailed breakdown of this at the R&D Day. So I would encourage you to come along.

是的。好問題。所以您可能知道，我們正在研究一種非常晚期的治療方法。但就年齡界線而言，我們針對的患者族群相當廣泛。再次，我們將分享詳細信息，我們預計在我們的臨床試驗中，大多數患者都會有大量的治療方法。顯然，這將包括 CD19、CAR 等等。因此，您將在研發日看到這方面的詳細分析。所以我鼓勵你也來。

In terms of the number of patients, I think the number of patients in our trial is driven by two things. One is the powering of our trials. And the two is the requirement of the safety database, which often takes precedence. So I think, your suggestion that, looking at other autologous CD19, CARs in this space will give you, while not entirely accurate, it will give you a ballpark as to what kind of numbers we need to have in these kind of trials. And it's driven by the size of the safety database rather than the assumed statistical powering.

就患者數量而言，我認為我們試驗的患者數量由兩件事決定。一是為我們的試驗提供動力。二是資料庫安全的要求，往往是優先考慮的。因此我認為，您建議查看該領域的其他自體 CD19 CAR，雖然不是完全準確，但它可以為您提供一個大概的信息，讓我們知道在這種試驗中我們需要什麼樣的數字。它是由安全資料庫的大小而不是假設的統計能力所驅動的。

Great. That's super helpful. And then, as a follow-up for the Servier program arbitration discussion. I was just wondering, could you give us a sense of the size of the milestone payments that potentially could be awarded at that point? Thank you.

偉大的。這非常有幫助。然後，作為 Servier 計劃仲裁討論的後續行動。我只是想知道，您能否告訴我們屆時可能授予的里程碑付款的金額？謝謝。

Yeah. Thanks, Yanan. Unfortunately, as this is an ongoing legal matter, we're not going to be able to give more details on this. Sorry.

是的。謝謝，亞南。不幸的是，由於這是一個正在進行的法律問題，我們無法提供更多細節。對不起。

No problem. Thanks for taking the question, and congrats on the progress.

沒問題。感謝您提出這個問題，並祝賀您的進展。

Sebastiaan van der Schoot, Campton.

塞巴斯蒂安·范德斯庫特，坎普頓。

Hi. Congrats on the progress. Thank you for taking my questions. Just two from my side. The first one is a follow-up on Allogene decision to not move forward with the anti-CD2 or CD52 antibody. Can you maybe give some insight whether there's been any feedback from the regulators from inclusion of the anti-CD52 in your treatment regimen? And do you expect that there might be another conversation with the FDA after these filings from Allogene? And then I have a follow-up.

你好。恭喜你取得進展。感謝您回答我的問題。我這邊只有兩個。第一個是 Allogene 決定不再繼續推進抗 CD2 或 CD52 抗體的後續行動。您能否透露一下，在您的治療方案中加入抗 CD52 後，監管機構是否有任何回饋？您是否預計在 Allogene 提交這些文件後可能會與 FDA 進行另一次對話？然後我有一個後續問題。

Thanks, Sebastiaan. Adrian, do you want to take this one?

謝謝，塞巴斯蒂安。阿德里安，你想拿這個嗎？

Sebastiaan, I just want to make sure I've understood the question. Could you just repeat it?

塞巴斯蒂安，我只是想確保我理解了這個問題。你能再說一次嗎？

Sure. So it comes down to whether you expect that this decision from Allogene that came after your conversation with the regulator, but that can still influence the design of the pivotal study with the use of the anti-CD52 antibody.

當然。因此，這取決於您是否預期 Allogene 會在您與監管機構溝通後做出此決定，但這仍然會影響使用抗 CD52 抗體的關鍵研究的設計。

No. Honestly, we don't believe so. Again, we have an established safety profile and an established risk-benefit assessment. As Arthur already stated, the Allogene product is different. And if we assume they may be even similar, our dosing levels are significantly lower than we see with the Allogene product.

不。說實話，我們不這麼認為。再次強調，我們已經建立了安全概況和風險效益評估。正如 Arthur 所說，Allogene 產品有所不同。如果我們假設它們可能相似，我們的劑量水平明顯低於 Allogene 產品。

And again, the regulatory authorities have reviewed our full in the Phase 1 package, including detailed look at the safety profile. So I don't see that there will be -- we don't foresee any changes based on what we've seen with Allogene.

再次強調，監管機構已經審查了我們第一階段的全部方案，包括對安全性的詳細審查。因此我認為不會有——根據我們在 Allogene 上看到的情況，我們預計不會有任何變化。

Got it. Thank you. Very clear. And then maybe regarding the cash runway and cash position. Could you indicate whether that incorporates the entire completion of the study for lasme-cel?

知道了。謝謝。非常清楚。然後也許涉及現金流和現金狀況。您能否指出這是否包括對 lasme-cel 的整個研究？

Yeah, great question. So the cash runway into H2 2027 does include pivotal studies. Actually, we've made assumptions both for lasme-cel and for eti-cel. So all the costs are fully loaded in that front.

是的，很好的問題。因此，2027 年下半年的現金流量確實包括關鍵研究。實際上，我們對 lasme-cel 和 eti-cel 都做出了假設。因此，所有成本都已全部計入其中。

We've also been very prudent, as we've always done on cash in from milestones and non-dilutive funding. So this one have been probabilized, so there is potential for upside there if they do materialize. And we will provide the full detailed timelines of the Phase 2 at the R&D Day. But to answer your question, yes, the runway does include the pivotal studies.

我們也一直非常謹慎，就像我們一直對里程碑和非稀釋性融資的現金投入一樣。因此，這個已經是機率性的，如果它們真的實現，那麼就有上漲的潛力。我們將在研發日提供第二階段的完整詳細時間表。但要回答你的問題，是的，跑道確實包括關鍵研究。

Great. And then maybe a last one, it's on the data set for eti-cel by year end. Can you just give some color on the size of that data set? Will it be similar in patient size? I understand that there will not be enough follow-up, but in terms of patient size, will be similar to the lasme-cel disclosure back in October.

偉大的。然後也許是最後一個，它是關於年底 eti-cel 的資料集。您能否提供一些有關該資料集大小的詳細資訊？患者的體型會相似嗎？我知道後續追蹤不會足夠，但就病患規模而言，將與 10 月的 lasme-cel 揭露情況類似。

Yeah. I'll take that, Arthur. So obviously, lasme-cel is a much more -- it's early candidate with much more patients within the Phase 1 program than eti-cel. So you will be anticipating smaller patient numbers. And again, we will -- you'll see based on our submissions to ASH, you'll see much more of that data then.

是的。我接受，亞瑟。因此，顯然，lasme-cel 是一種更早期的候選藥物，其第一階段計劃中的患者數量比 eti-cel 多得多。因此，預計患者數量將會減少。再說一次，根據我們向 ASH 提交的內容，您將會看到更多此類資料。

Great. Thank you so much.

偉大的。太感謝了。

Salveen Richter, Goldman Sachs.

薩爾文·里克特，高盛。

Hey, good morning. This is Mark on for Salveen. Thank you so much for taking our question, and congrats on the quarter. I have, I guess, a follow-up on eti-cel. There's been a lot of news from autologous dual-targeting CAR-Ts recently. What are your thoughts? How do you view that data? Is there any read-through to eti-cel? And sort of beyond the allogeneic autologous, sort of, difference? How do you think eti-cel is differentiated in the dual targeting space? Thanks.

嘿，早安。我是 Salveen 的 Mark。非常感謝您回答我們的問題，並祝賀本季度取得的成績。我想，我對 eti-cel 有一個後續行動。最近有很多關於自體雙靶向 CAR-T 的新聞。您的想法是什麼？您如何看待這些數據？有沒有 eti-cel 的通讀？這是否超越了同種異體和自體之間的某種差異？您認為 eti-cel 在雙重目標領域有何不同？謝謝。

Yeah. It's a great question. We believe we've got a very well differentiated product with eti-cel. We believe our positioning is very clear. It's a later line diffuse large B-cell lymphoma, most likely, certainly within the non-Hodgkin lymphoma space. We think our -- and again, when you see what's presented at ASH, it will be assuming it's accepted. You'll see a clear strategy, which I think differentiates the product significantly from the current batch of autologous products. So I can't really say much more until we -- you see what we will be hopefully presenting at ASH.

是的。這是一個很好的問題。我們相信 eti-cel 是一款極具差異化的產品。我們相信我們的定位非常明確。它是一種晚期瀰漫性大 B 細胞淋巴瘤，很有可能，肯定屬於非何杰金氏淋巴瘤範圍。我們認為——再說一次，當您看到 ASH 上展示的內容時，就會假設它已被接受。您會看到一個清晰的策略，我認為這使該產品與當前一批自體產品有顯著區別。因此，在我們了解我們將在 ASH 上展示什麼之前，我真的不能說太多。

If I can add -- and thanks, Mark, for the great question. I think the -- I'm sorry, I'm going to state the obvious, but the big important piece about eti-cel is it does not target 19. A lot of the dual targeting data we've seen is like 19, 20, 19, 22. So I think it's great, but it's yet again hitting 19. So the primary competitors to 19, 20 or 19, 22 are going to be the approved autologous 19 and potentially some of the ALLO-19.

如果我可以補充的話——感謝馬克提出的這個好問題。我認為——抱歉，我要說的是顯而易見的事情，但 eti-cel 最重要的一點是它不是針對 19 歲。我們看到的許多雙重目標數據是 19、20、19、22。所以我認為這很棒，但它再次達到 19。因此，19、20 或 19、22 的主要競爭對手將是已批准的自體 19 以及潛在的部分 ALLO-19。

I think where eti-cel is pretty unique to our knowledge at this stage is that it is a 20 and 22. And so it will be particularly relevant to physicians who have hit 19 once and will then want to -- or twice and will want to alternate the targets.

我認為，就我們目前所知，eti-cel 非常獨特，因為它是 20 和 22。因此，它對於那些已經達到 19 個目標一次並希望再次達到或兩次並希望交替目標的醫生來說尤其重要。

I think the other important thing to remember is, right now, 19 is firmly entrenched in the second line. This is primarily YESCARTA and BREYANZI. If Allogene is successful with this first-line consolidation approach, to which, by the way, we have a vested interest in. There is a potential for 19 to come already up to the first-line consolidation. And then there will be a very, very strong need to hit orthogonal targets.

我認為要記住的另一件重要的事情是，現在，19 已經牢牢佔據了第二線。這主要是 YESCARTA 和 BREYANZI。如果 Allogene 能夠成功採用這種一線鞏固方法，順便說一句，我們對此有既得利益。19 有可能已經達到第一線整合的水準。然後，就會非常非常強烈地需要達到正交目標。

So coming at it with an off-the-shelf alternative that does not require an additional round of leukapheresis, harvesting of the patient, slots, et cetera. So pure off-the-shelf alternative that does not target 19, I think in our universe of NHL and LBCL, is pretty differentiated and unique.

因此，採用現成的替代方案，不需要額外的白血球分離術、採集病患樣本、插槽等。因此，我認為，在 NHL 和 LBCL 領域中，不針對 19 的純現成替代品是相當有區別且獨特的。

That makes sense. Thank you.

這很有道理。謝謝。

Thank you. It appears we have no further questions at this time. I would now like to turn the program back over to our presenters for any additional or closing remarks.

謝謝。目前看來我們沒有其他問題。現在，我想將節目交還給主持人，請他們發表任何補充或結束語。

Well, I would like to thank you all. And we definitely -- there is a very rich second half this year for Cellectis. So please stay tuned, and we hope that you will be all at our R&D Day event on October 16 and probably wait to what's going to happen for Cellectis by the end of the year. So a lot of like rich event things that will come up.

好吧，我想感謝大家。毫無疑問，今年下半年對 Cellectis 來說將是一個豐收的半年。所以請繼續關注，我們希望你們都能參加我們 10 月 16 日的研發日活動，並期待了解 Cellectis 在今年年底會發生什麼。因此，許多類似豐富的活動將會出現。

Thank you very much for your attention, and have a good day.

非常感謝您的關注，祝您有美好的一天。

Thank you, ladies and gentlemen. This concludes today's event. You may now disconnect.

謝謝各位，女士們、先生們。今天的活動到此結束。您現在可以斷開連線。