Marker Therapeutics Inc (MRKR) 2021 Q1 法說會逐字稿

Hello, and welcome to the Marker Therapeutics First Quarter 2021 Operating and Financial Results Conference Call and Webcast. (Operator Instructions) As a reminder, this conference is being recorded.

您好，歡迎參加 Marker Therapeutics 2021 年第一季營運和財務業績電話會議和網路廣播。 （操作員指示）提醒一下，本次會議正在錄音。

It's now my pleasure to turn the call over to CFO, Tony Kim. Please go ahead, sir.

現在我很高興將電話轉給財務長 Tony Kim。先生，請繼續。

Thanks, and welcome, everyone. The press release reporting our financial results is available in the News section of our corporate website at www.markertherapeutics.com. As a reminder, we will be making forward-looking statements regarding our financial outlook in addition to regulatory, product development and commercialization plans and research activities. These statements are subject to risks and uncertainties that may cause actual results to materially differ from those forecasted. A description of these risks can be found in our most recent Form 10-Q and 10-K on file with the SEC.

謝謝大家，歡迎大家。報告我們財務表現的新聞稿可在我們公司網站 www.markertherapeutics.com 的新聞部分查閱。提醒一下，除了監管、產品開發和商業化計劃以及研究活動之外，我們還將對我們的財務前景做出前瞻性陳述。這些聲明受風險和不確定性的影響，可能導致實際結果與預測結果有重大差異。這些風險的描述可以在我們向美國證券交易委員會提交的最新 10-Q 表和 10-K 表中找到。

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

現在我想把電話轉給彼得。

Thanks, Tony. Good afternoon, and thank you for joining us today. I'm pleased to say that we're off to a productive start to the year. During the first quarter, we completed a $56.5 million public offering of common stock that will support the continued growth of our pipeline, and we're making steady progress on both the clinical and the manufacturing fronts. In March, we treated the first patient with MT-401 in the safety lead-in portion of our Phase II trial in post-transplant acute myeloid leukemia, or AML. This is a significant milestone for us as it is our first company-sponsored trial with MultiTAA therapy, but also a critical first step towards developing what we believe could be a potentially transformative therapy for these patients who, at present, have only a 25% chance for surviving 5 years.

謝謝，托尼。下午好，感謝您今天加入我們。我很高興地說，我們今年的開局非常有成效。在第一季度，我們完成了 5,650 萬美元的普通股公開發行，這將支持我們產品線的持續成長，並且我們在臨床和製造方面都取得了穩步進展。今年 3 月，我們在移植後急性髓性白血病 (AML) II 期試驗的安全導入階段中使用 MT-401 治療了第一位患者。這對我們來說是一個重要的里程碑，因為這是我們首次由公司贊助的 MultiTAA 療法試驗，也是朝著開發一種我們認為可能對這些患者俱有潛在變革性的療法邁出的關鍵的第一步，目前這些患者 5 年生存率只有 25%。

We currently have several sites open and enrolling, and our clinical team is hard at work getting additional clinical sites online. Our Chief Medical Officer, Dr. Mythili Koneru will provide further details surrounding our AML trial in just a moment, and we look forward to taking your questions at the end of today's call. In parallel with the clinical developments, we continue to optimize the MT-401 cell therapy manufacturing process as we prepare to operationalize our new in-house manufacturing facility in the first half of this year.

我們目前已開放多個站點並正在招募，我們的臨床團隊正在努力讓更多臨床站點上線。我們的首席醫療官 Mythili Koneru 博士將立即提供有關我們的 AML 試驗的更多詳細信息，我們期待在今天的電話會議結束時回答您的問題。在臨床開發的同時，我們繼續優化 MT-401 細胞療法製造工藝，準備在今年上半年投入營運新的內部製造工廠。

In brief, we're excited to explore how these modifications can be applied across our MultiTAA therapies and could potentially result in an increase in the number of T cells available for patient administration, amongst other benefits. Our Chief Development Officer, Dr. Juan Vera has joined us today to provide a lot of details about the important process improvements that we've implemented. We look forward to completing the technology transfer from Baylor College of Medicine and manufacturing in-house all of the study drug for our AML trial and future trials.

簡而言之，我們很高興探索如何將這些修改應用於我們的 MultiTAA 療法，並可能增加可供患者管理的 T 細胞數量，以及其他益處。我們的首席開發長 J​​uan Vera 博士今天加入我們，提供有關我們實施的重要流程改進的大量詳細資訊。我們期待完成貝勒醫學院的技術轉讓，並在內部生產用於我們的 AML 試驗和未來試驗的所有研究藥物。

This year, our primary focus is on completing treatment of the patients in the safety leading portion of our AML study with MT-401 and enrolling patients in the Phase II portion of the trial. As you may recall, our cell therapy was designed to address the shortcomings of current treatments while maintaining patient safety. Many cell therapies in development today are pursuing single or even dual targets. However, this approach has demonstrated little -- limited improvement in patient outcomes.

今年，我們的主要重點是使用 MT-401 完成 AML 研究安全領先部分患者的治療，並招募患者參加試驗的 II 期部分。您可能還記得，我們​​的細胞療法旨在解決目前治療的缺點，同時確保患者的安全。當今正在開發的許多細胞療法都追求單一甚至雙重目標。然而，這種方法對患者治療效果的改善有限。

By contrast, we believe that our multi-antigen approach has the potential to reduce a lasting antitumor effect by allowing the patient's own T cells to expand and kill cancer cells alongside our therapy. By targeting multiple antigens and epitopes present within the tumor, we believe that our MultiTAA T cell therapy can effectively address the tumor head of the geneity, while recruiting the endogenous immunity that amplify the immune response through epitope spreading.

相較之下，我們相信，我們的多抗原方法有可能透過允許患者自身的 T 細胞擴增並在我們的治療過程中殺死癌細胞來降低持久的抗腫瘤作用。透過針對腫瘤內存在的多種抗原和表位，我們相信我們的 MultiTAA T 細胞療法可以有效地解決腫瘤的遺傳性頭部問題，同時募集內源性免疫，透過表位擴散來放大免疫反應。

At this time, I'd like to hand over the call to Dr. Mythili Koneru, our Chief Medical Officer, to review details of our Phase II trial and our progress to date. Mythili?

現在，我想將電話交給我們的首席醫療官 Mythili Koneru 博士，以審查我們第二階段試驗的細節以及迄今為止的進展。神話？

Thank you, Peter. As you just heard, this has been an important quarter for us as we dose the first patient within the safety leading portion of our Phase II trial in post-transplant AML. Just as a reminder, we plan to enroll approximately 6 patients in this portion of the trial. 3 patients will be dosed with MT-401, our lead product candidate manufactured with a legacy reagent, which was used in the Phase I trial conducted by our partners at the Baylor College of Medicine. The remaining 3 patients will be dosed with study drugs manufactured using a new reagent from an alternative supplier.

謝謝你，彼得。正如您剛才聽到的，這對我們來說是一個重要的季度，因為我們在移植後 AML II 期試驗的安全領先部分中為第一位患者進行了給藥。提醒一下，我們計劃在這部分試驗中招募約 6 名患者。 3 名患者將接受 MT-401 治療，這是我們採用傳統試劑製造的主要候選產品，該試劑已在貝勒醫學院的合作夥伴進行的第一階段試驗中使用。其餘 3 名患者將接受使用其他供應商提供的新試劑生產的試驗藥物。

Our clinical operations team has made considerable progress on getting sites open and have currently 9 sites activated. Additionally, we plan on opening approximately 20 sites in total for the Phase II portion of the study and anticipate being able to treat the first patient in the main portion of the protocol in Q3 of this year. MT-401, which was granted orphan drug designation in post-transplant AML has been well tolerated in an ongoing Phase I clinical trial conducted by our academic collaborators at Baylor College of Medicine in the setting.

我們的臨床營運團隊在開放站點方面取得了長足的進展，目前已啟動 9 個站點。此外，我們計劃為該研究的第二階段部分總共開放約 20 個站點，並預計能夠在今年第三季度治療該方案主要部分中的第一位患者。 MT-401 被授予移植後 AML 孤兒藥稱號，並且在我們貝勒醫學院的學術合作夥伴正在進行的 I 期臨床試驗中表現出良好的耐受性。

Overall, results showed that MT-401 was well tolerated with no incidence of cytokine release syndrome, neurotoxicity or grade 3 to 4 GvHD in the post allogeneic setting and demonstrated an antitumor effect with significant in vivo expansion of T cells. [Embrace], as reported in a recent publication by Lulla at all in December 2020, 11 of the 17 patients in the adjuvant disease setting dosed with MultiTAA-specific T cell therapy after receiving an allogeneic stem cell transplant never relapsed. Median leukemia free survival, or LFS is not reached at a median follow-up of 1.9 years.

總體而言，結果顯示 MT-401 耐受性良好，在同種異體移植後未發生細胞激​​素釋放症候群、神經毒性或 3 至 4 級 GvHD，並表現出抗腫瘤作用，體內 T 細胞顯著擴增。 [Embrace]，正如 Lulla at all 在 2020 年 12 月發表的最新文章中所報道的，在接受同種異體幹細胞移植後接受 MultiTAA 特異性 T 細胞療法治療的 17 名輔助疾病患者中，有 11 名從未復發。在中位追蹤時間 1.9 年內未達到無白血病存活中位數時間 (LFS)。

With 11 of the 15 patients remaining alive, estimated 2 years overall survival of 77% at a median follow-up of 1.9 years post infusion, which compares favorably with transplant outcomes for risk mashed AML MDS patients post-transplant. Additionally, 8 patients were treated for active disease that was resistant to salvage therapy post-transplant with the median of 5 prior lines of therapy. The range was 4 to 10. 2 of the 8 patients achieved objective responses with 1 complete response and 1 partial response, with 6 patients continuing with stable disease, some of which had reduction in tumor burden. Now based on these results, we are optimistic about MT-401's potential in this patient population.

15 名患者中仍有 11 名存活，輸注後平均追蹤 1.9 年，預計 2 年總體存活率為 77%，與移植後高風險 AML MDS 患者的移植結果相比，該結果更為有利。此外，8 名患者因對移植後挽救療法有抗藥性而接受了治療，平均接受過 5 種療法。範圍是 4 到 10。8 名患者中有 2 名達到客觀反應，其中 1 名完全反應，1 名部分反應，6 名患者病情繼續穩定，其中一些患者的腫瘤負擔有所減輕。現在基於這些結果，我們對 MT-401 在該患者群體中的潛力持樂觀態度。

To briefly recap, this multicenter AML study will be evaluating clinical efficacy of our product in patients with AML in both adjuvant and active disease settings, following an allogeneic stem cell transplant. In the adjuvant setting, approximately 120 patients will be randomized 1:1 to either MT-401 at 90 days post-transplant first standard of care observation, while about 40 patients with active disease will receive MT-401 as part of the single-arm group.

簡單回顧一下，這項多中心 AML 研究將評估我們的產品對異基因幹細胞移植後輔助和活動性疾病環境中的 AML 患者的臨床療效。在輔助治療中，約 120 名患者將在移植後 90 天首次標準護理觀察時以 1:1 的比例隨機分配接受 MT-401 治療，而約 40 名患有活動性疾病的患者將作為單組接受 MT-401 治療。

The primary objectives of the trial are to evaluate relapse-free survival in the adjuvant group and determine the complete remission rate and duration of complete remission in active disease patients. Additional objectives include for the adjuvant group: overall survival; and graft-versus-host disease relapse-free survival; while additional objectives for the active disease group include: overall response rate; duration of response; progression-free survival; and overall survival.

該試驗的主要目的是評估輔助組的無復發生存期，並確定活動性疾病患者的完全緩解率和完全緩解持續時間。輔助治療組的其他目標包括：整體存活期；以及移植物抗宿主疾病無復發生存率；而針對活躍疾病組的其他目標包括：整體反應率；反應持續時間；無惡化存活期；和整體存活率。

And with that, I'd like to hand the call over to Dr. Juan Vera, our Chief Development Officer.

現在，我想將電話交給我們的首席開發長胡安·維拉博士。

Thank you, Mythili. In parallel to the progress we have made on the clinical front, we continue to work on simplifying and streamline our manufacturing process while improving the T cell phenotype and antigen specificity of the final drug product. We believe these improvements may have an impact on the clinical performance of the drug product in our Phase II clinical study. These manufacturer optimizations fall under 2 major categories: technical and biological improvements. At present, we have incorporated several technical improvements, including: first, a 50% reduction in the manufacturing time, resulting in a 16 days manufacturing process, decreasing in this manner, the vein-to-vein time while improving the manufacturer throughput.

謝謝你，Mythili。在臨床方面取得進展的同時，我們繼續致力於簡化和精簡我們的製造流程，同時改善最終藥物產品的 T 細胞表型和抗原特異性。我們相信這些改進可能會對我們 II 期臨床研究中的藥品臨床表現產生影響。這些製造商的最佳化分為兩大類：技術和生物改進。目前，我們已經採用了多項技術改進，包括：首先，製造時間縮短了 50%，製造過程只需 16 天，從而減少了靜脈到靜脈的時間，同時提高了製造商的產量。

Second, a decrease in the number of technical interventions by approximately 95%, reducing in this way, the risk of contaminations. Third, an improved manufacturing process, which will reduce the risk of manufacturing failures. And 4 and final, despite the reduction in the manufacturing time, we are able to produce sufficient cell numbers to patients in the current clinical study. In addition to this technical improvements, we are now capable of producing a final drug product with a more favorable cell phenotype, upgraded magnitude of antigen specificity and a broader targeted recognition profile. We believe the combination of this technical and biological improvements might result in a clinical benefit in the upcoming study in AML. Importantly, these CMC changes have already been approved by the FDA and are currently implemented in the safety leading portion of the clinical study.

其次，技術介入次數減少約 95%，進而降低污染風險。第三，改良製造工藝，降低製造失敗的風險。最後，儘管製造時間縮短，但我們仍然能夠在目前的臨床研究中為患者提供足夠的細胞數量。除了這些技術改進之外，我們現在還能夠生產具有更有利的細胞表型、更高的抗原特異性和更廣泛的靶向識別特性的最終藥物產品。我們相信，這種技術和生物學改進的結合可能會在即將進行的 AML 研究中帶來臨床益處。重要的是，這些CMC變更已經獲得FDA批准，目前正在臨床研究的安全主導部分實施。

With that, I will turn the call back to Tony, our Chief Financial Officer to review the financials. Tony?

說完這些，我將把電話轉回給我們的財務長托尼來審查財務狀況。托尼？

Thanks, Juan. We ended the first quarter with $64.5 million in cash and cash equivalents. We expect that our current cash balance will support operations into the first quarter of 2023. Net loss for the quarter ended March 31, 2021, was $8.8 million compared to a net loss of $6.5 million for the quarter ended March 31, 2020. Research and development costs during the 3 months ended March 31, 2021, was $5.6 million compared to $3.8 million during the 3 months ended March 31, 2020. The increase of $1.8 million was primarily attributable to increases in headcount-related expenses and infrastructure expenses due to growth of research and development operations. General and administrative expenses were $3.1 million during the 3 months ended March 31, 2021, compared to $2.8 million during the 3 months ended March 31, 2020.

謝謝，胡安。第一季結束時，我們的現金和現金等價物為 6,450 萬美元。我們預計目前的現金餘額將支援到 2023 年第一季的營運。截至 2021 年 3 月 31 日的季度淨虧損為 880 萬美元，而截至 2020 年 3 月 31 日的季度淨虧損為 650 萬美元。截至 2021 年 3 月 31 日的三個月的研發成本為 560 萬美元，而截至 2020 年 3 月 31 日的三個月的研發成本為 380 萬美元。增加 180 萬美元主要歸因於研發業務成長導致的員工相關費用和基礎設施費用增加。截至 2021 年 3 月 31 日的 3 個月期間，一般及行政費用為 310 萬美元，而截至 2020 年 3 月 31 日的 3 個月期間為 280 萬美元。

At this time, we'd like to open the call up to questions. Operator?

現在，我們想開始回答問題。操作員？

(Operator Instructions) Our first question today is coming from Joe Catanzaro from Piper Sandler.

（操作員指示）我們今天的第一個問題來自 Piper Sandler 的 Joe Catanzaro。

Maybe just 2 for me. So with regards to the safety lead-in in those 6 patients, just wondering if we should expect any disclosures from you once that part of the trial has been completed? And if so, what should we -- what could we expect to hear out of those 6 patients?

對我來說可能只要 2 個。因此，關於這 6 名患者的安全引入情況，我只是想知道，一旦試驗的這一部分完成，我們是否應該期待您披露任何資訊？如果是這樣，我們應該——我們希望從這 6 名患者那裡聽到什麼消息？

Sure, Joe. That's a great question. Let me turn that over to Dr. Mythili Koneru, who can talk about the safety lead-in.

當然，喬。這是一個很好的問題。讓我把這個問題交給 Mythili Koneru 博士，他可以談談安全引導。

Yes. Thank you for your question. Regarding the safety lead-in, 6 patients, the primary objective is obviously safety. So we'll be looking specifically at dose-limiting toxicities. In terms of timing, as we've mentioned, we are looking on track to completing the safety lead-in for the 6 patients mid this year and to open up the main portion of the Phase II. We -- you may anticipate a potential announcement for when the main 2 -- main portion of the Phase II is opened potentially. And then the safety lead-in for the dose-limiting toxicity has been cleared safely.

是的。感謝您的提問。對於安全導入，6名患者，首要目標顯然是安全。因此我們將特別關注劑量限制毒性。就時間安排而言，正如我們所提到的，我們預計在今年年中完成 6 名患者的安全導入，並開啟第二階段的主要部分。我們—您可能會期待有關第二階段主要部分何時開放的潛在公告。然後劑量限制毒性的安全引入就被安全地清除了。

Okay. Got it. That's helpful. And then maybe quickly, my second question. Peter, you noted that the optimized manufacturing process generates a greater quantity of T cells. Is there any possibility or thinking that the main portion of the Phase II could use higher doses of 401 than what's being used during the safety lead-in? Or should we just be thinking more along the lines of improved T cell phenotype and antigen specificity?

好的。知道了。這很有幫助。然後也許很快，我的第二個問題。彼得，您指出優化的製造流程可以產生更多的 T 細胞。是否有可能或想法，第二階段的主要部分可能會使用比安全導入期間使用的劑量更高的 401？或者我們應該更多地考慮改善 T 細胞表型和抗原特異性？

Thanks, Joe. That's a great question. I think that we are very optimistic about what effects we might see from the improvement in the manufacturing process. But let me turn that question over to Juan, who is closest to the CMC and process improvements that we've implemented.

謝謝，喬。這是一個很好的問題。我認為我們對製造流程改進可能帶來的效果非常樂觀。但是，讓我把這個問題交給 Juan，他最了解 CMC 和我們實施的流程改善。

Sure. Yes. We actually feel very comfortable with the improvement that we have seen in the final product in term of the analytical comparison, right? And I'm referring specifically to the comparison in regards to the antigen specificity and the cell phenotype. Both we have shown that the current -- the new process is able to yield a product of greater analytical characteristics. So from that, I think that the product that will be manufactured for the ongoing study should yield a product of biological characteristics that would be superior to what we initially tested in the Baylor studies.

當然。是的。從分析比較來看，我們對最終產品的改進感到非常滿意，對嗎？我特別指的是抗原特異性和細胞表型的比較。我們已經證明，目前的新製程能夠生產出具有更佳分析特性的產品。因此，從這一點來看，我認為正在進行的研究所生產的產品應該會產生一種生物學特性的產品，這種產品將優於我們最初在貝勒研究中測試的產品。

In regards to the question on the cell dose, this new manufacturing process opened that opportunity, right? I think that now being able to simplify the manufacturing process and also being able to increase the overall cell numbers will give us the opportunity to explore even higher sales. With that, maybe, I don't know if Mythili wants to comment in term of the prospects of doing a higher cell dose in the clinical setting? My?

關於細胞劑量的問題，這個新的製造過程開啟了這個機會，對嗎？我認為現在能夠簡化製造流程並能夠增加整體電池數量將使我們有機會探索更高的銷售。就此而言，也許，我不知道 Mythili 是否想就臨床環境中進行更高細胞劑量的前景發表評論？我的？

Thank you, Juan. The Phase I AML and [ESA] that was done at Baylor College of Medicine has been exploring higher doses and shown them to be safe. So there is an opportunity for us to increase the dose in the main portion of the stage 2 based on that data and to do so without necessarily doing any additional safety readings per se.

謝謝你，胡安。貝勒醫學院進行的第一階段 AML 和 [ESA] 一直在探索更高的劑量並證明其是安全的。因此，我們有機會根據該數據增加第 2 階段主要部分的劑量，而不必進行任何額外的安全讀數。

Our next question today is coming from Kristen Kluska from Cantor Fitzgerald.

今天的下一個問題來自 Cantor Fitzgerald 的 Kristen Kluska。

And I like the new look of your website. I wanted to first ask, with all of these manufacturing, technical and biological improvements that you laid out, how are you thinking about how this could impact the cost and time savings on a larger scale? And then do you believe that this process could be utilized long term? Or will you continue to look at other items?

我喜歡您網站的新外觀。我想先問一下，對於您所列出的所有這些製造、技術和生物學改進，您認為這些改進將如何在更大範圍內影響成本和時間的節省？那麼您是否相信這個過程可以長期使用？或者您會繼續關注其他商品嗎？

Thanks for the question, Kristen. Let me turn that over to Juan.

謝謝你的提問，克里斯汀。讓我把這個交給胡安。

Thank you. That's an excellent question. We -- although we don't necessarily highlight that aspect, I think that the new simplified manufacturing process should have a significant impact in term of yield in some more economic final product that mainly result from a reduction in the overall sort of culture tone and the overall simplification and decreasing the number of interventions. So without a doubt, that is going to have a positive impact in the cost of the final drug product, right? And -- but I have to highlight that we are aware that there's still aspect that can be further improved in the manufacturing process.

謝謝。這是一個很好的問題。我們——儘管我們不一定會強調這一點，但我認為新的簡化製造流程應該對一些更經濟的最終產品的產量產生重大影響，這主要源於整體文化基調的減少和整體的簡化以及乾預次數的減少。所以毫無疑問，這將對最終藥品的成本產生正面影響，對嗎？但我必須強調，我們意識到製造過程中仍有一些方面可以進一步改進。

I believe what we have here is something that is a very solid and -- a very solid manufacturing process that will be suitable for even future commercialization. However, it gives a very strong foundations from this process to be optimized and to have a close system and to incorporate certain elements that will allow optimization of this process to simplify and speed up some of the components itself in the manufacturing. So to summarize, I think that I agree that with your statement that this simplification has an impact also in the economics, not just in the biology. And I think that this basically now has a very strong basis for future areas of improvement that I will consider at this point will be minimal, but nevertheless significant.

我相信我們現在擁有的是一種非常可靠的製造工藝，甚至適合未來的商業化。然而，它為優化這一流程奠定了非常堅實的基礎，並形成了一個封閉的系統，並結合了某些元素，從而可以優化這一流程，簡化和加快製造過程中某些組件本身的速度。總而言之，我認為我同意你的說法，這種簡化不僅對生物學有影響，而且對經濟學也有影響。我認為這基本上為未來的改進奠定了非常堅實的基礎，我認為目前的改進雖然很小，但意義重大。

And yesterday, you had a poster presentation with some comments from Dr. Smith at ASGCT with ABB, evaluating the potentials of the robotics implementation. So I wanted to ask, based on these early findings, if you could discuss if you're looking to further expand on this collaboration? And what any next step might look like?

昨天，你們觀看了海報展示，其中史密斯博士與 ABB 一起在 ASGCT 發表了一些評論，評估了機器人技術實施的潛力。因此，我想問一下，基於這些早期發現，您是否可以討論是否希望進一步擴大這種合作？下一步會怎樣？

I think we're very excited about the collaboration with ABB. I think that what we're finding is that with the implementation of robotic technology, we can improve the consistency of the manufacturing process, which is extraordinarily important in cell therapies. But Juan, I think that your comments here would be valuable.

我認為我們對與 ABB 的合作感到非常興奮。我認為我們發現，透過機器人技術的實施，我們可以提高製造過程的一致性，這對於細胞療法來說極為重要。但是胡安，我認為你在這裡的評論很有價值。

Sure. Thank you, Peter. I completely agree. Without a doubt, an area of high variability currently in the field associated with this generation of these patient-specific products is still rely on the operator, right? They definitely an inherent variability from this statin material. But I think that by being able to incorporate our robotic process, you're basically removing in the future a potential unknown, right, which basically is the addition of variability from the operator itself.

當然。謝謝你，彼得。我完全同意。毫無疑問，目前與這一代針對特定患者的產品相關的領域中高度可變的領域仍然依賴於操作員，對嗎？它們肯定具有這種他汀類藥物的固有變異性。但我認為，透過融入我們的機器人流程，你基本上可以消除未來潛在的未知數，對吧，這基本上就是操作員本身增加的可變性。

So I think that this is something that, as Peter mentioned, we're really excited about the collaboration with ABB, and we're looking forward to continue working towards the integration of a robotic process in the manufacture of MT-401. And we believe that this is something that could really transform a process and making it more suitable for future commercialization. So we're really excited about the line of work.

所以我認為，正如 Peter 所提到的，我們對與 ABB 的合作感到非常興奮，我們期待繼續致力於將機器人流程融入 MT-401 的製造中。我們相信這可以真正改變流程並使其更適合未來的商業化。所以我們對這項工作感到非常興奮。

And then the last question I have is that while I know you're focused on AML, could you talk about how you're thinking about any next potential indications to bring in-house based on the BCM proof of concept? And maybe specifically, what are going to be some of the key decision criteria?

然後我的最後一個問題是，雖然我知道您專注於 AML，但您能否談談您如何考慮基於 BCM 概念驗證在內部引入的下一個潛在跡象？具體來說，一些關鍵的決策標準是什麼？

Mythili? I think that this is appropriate for you.

神話？我認為這對你來說很合適。

Thank you, Peter. Thanks for the question. Yes, absolutely. I mean, I think you're right in the sense that we have been working very closely with Baylor and to follow and understand the Phase I data very closely so that we can appreciate where the opportunity lies in terms of unmet need for a patient population and where the data is really taking us. Obviously, the pancreatic cancer data was presented last year at ASCO and continues to look promising. So I think this being an allogeneic IND for the AML, we're obviously looking at other indications in the autologous program to move forward, including pancreas and potentially other indications. It's really trying to see where the data leads us and where the unmet need is and finding the appropriate time to do so.

謝謝你，彼得。謝謝你的提問。是的，絕對是。我的意思是，我認為你是對的，因為我們一直與貝勒密切合作，密切關注和了解第一階段的數據，以便我們能夠了解患者群體未滿足的需求方面的機會在哪裡，以及數據真正將帶我們去哪裡。顯然，胰腺癌數據去年在 ASCO 上公佈，並且看起來仍然很有希望。因此，我認為這是 AML 的異基因 IND，我們顯然正在研究自體計畫中的其他適應症，包括胰臟和其他潛在適應症。它實際上是試圖了解數據將把我們引向何方以及未滿足的需求在哪裡，並找到適當的時間來這樣做。

Yes. Kristen, obviously, we think that the lymphoma data is quite striking. And we believe strongly that the pancreatic results are continuing to accrue evidence that the MultiTAA therapy is driving a meaningful therapeutic benefit for those patients with pancreatic adenocarcinoma. However, I would ask you to just bear with us because at this point, I don't think that we have -- we've officially announced further plans beyond our AML study. And that is currently where our focus is, is driving enrollment in that study.

是的。克里斯汀，顯然，我們認為淋巴瘤數據非常驚人。我們堅信，胰腺研究結果正在不斷累積證據，證明 MultiTAA 療法正在為胰腺腺癌患者帶來有意義的治療益處。但是，我請您耐心等待，因為目前，我認為我們還沒有正式宣布反洗錢研究之外的進一步計劃。這就是我們目前的重點，即推動該研究的招生。

Our next question today is coming from Matt Biegler from Oppenheimer.

我們今天的下一個問題來自奧本海默公司的 Matt Biegler。

Yes. I'll tag on to that last one. Peter, can you give us an update on where Baylor is in their own AML trial? I think My mentioned, the trial was testing a higher dose of MultiTAA. Any idea when we might see updated data from that trial or any of the other Baylor trials for that matter?

是的。我將標記最後一個。彼得，您能向我們介紹一下貝勒大學 AML 試驗的最新進展嗎？我認為我提到過，試驗正在測試更高劑量的 MultiTAA。您知道我們什麼時候可以看到該試驗或任何其他貝勒試驗的更新數據嗎？

Yes, that's a great question, Matt. Thanks for the question. Let me turn it over to Mythili, who coordinates and talks to Dr. Premal Lulla, our primary investigator at Baylor on a fairly consistent basis for the latest update on the dose escalation portion of the Baylor Phase I trial.

是的，這是一個很好的問題，馬特。謝謝你的提問。讓我把這個任務交給 Mythili，他負責協調並與貝勒大學的主要研究員 Premal Lulla 博士進行溝通，以了解貝勒大學 I 期試驗劑量遞增部分的最新進展。

Thanks, Matt, for your question. So if you look at the recent publication of -- from that group from Dr. Premal Lulla on the Phase I AML MDS study, there was some data included on some of the higher dose levels in that paper. So I think they are nearly complete with that last dose level. So that paper, I think, more or less, is up to date on the current status on the 2 additional dose cohort. And I think it's a very close completion of that last cohort with the highest dose.

謝謝馬特的提問。因此，如果您看 Premal Lulla 博士團隊最近發表的關於 I 期 AML MDS 研究的論文，您會發現該論文中包含了一些有關某些較高劑量水平的數據。所以我認為最後的劑量水平已經基本完成了。因此，我認為該論文或多或少更新了 2 個額外劑量組的當前狀態。我認為最後一批最高劑量的疫苗接種已經非常接近完成了。

I'm sorry, Matt. My understanding is that Baylor was set to complete the last dose as -- the last patient in the dose escalation phase, the dose level 5 last year, but due to disruptions because of COVID. They're still in the process of finding a patient to replace the patient that they had originally planned to treat, but we're unable to because of coronavirus.

對不起，馬特。我的理解是，貝勒原本計劃完成最後一劑——作為劑量遞增階段的最後一名患者，即去年的劑量等級 5，但由於 COVID 而中斷。他們仍在尋找一名患者來取代他們原計劃治療的患者，但由於冠狀病毒，我們無法做到這一點。

Yes. That makes sense. I'll have to check out that publication. I had a quick follow-up on manufacturing. Just any plans to present side-by-side comparisons of the drug product manufactured with the new approach versus the old Baylor approach?

是的。這很有道理。我得去看看那份出版物。我對製造業進行了快速跟進。是否有計劃對採用新方法和舊貝勒方法生產的藥品進行並排比較？

Yes. Let me direct that question to Juan.

是的。讓我直接向胡安提出這個問題。

Thank you. Yes, that's actually a good point. We feel really proud on the progress we have made on the manufacturing front. And actually, we're presenting a small snip of that information on the ISCT meeting and ASCGT. So the poster is really focused on how we can best improve the manufacture of MT-401, where we disclose the collaboration with ABB incorporating a robotic process. However, I encourage you to look at the results where we actually disclosed some information in the first part of the work, which is geared towards simplifying the manufacturing process. And there, you will be able to see the impact from a biological standpoint when we compare the old and the new manufacturing process.

謝謝。是的，這確實是一個很好的觀點。我們對在製造業的進步感到非常自豪。實際上，我們在 ISCT 會議和 ASCGT 上展示了一小部分資訊。因此，海報真正關注的是我們如何最好地改進 MT-401 的製造，其中我們揭露了與 ABB 合作採用機器人流程的情況。不過，我鼓勵您查看結果，我們實際上在工作的第一部分披露了一些信息，旨在簡化製造過程。當我們比較新舊製造流程時，您將能夠從生物學的角度看到其影響。

Your next question is coming from Tony Butler from ROTH Capital.

您的下一個問題來自 ROTH Capital 的 Tony Butler。

Yes. Peter or Mythili, you made a reference to the first 6 patients, in which case you would look at the lead-in portion of the study, in which you would look for some side effects that may occur from one -- from one group versus the other group, one of the 3 patients versus the other 3 patients. And the question is, is there any other biological data that you need to feed to the FDA to actually demonstrate that there's really no difference between the 2 particular reagents that are being utilized? And effectively, 3 patients from 1 reagent versus 3 patients from the second reagent are literally identical. And I'm curious what that biological data might be.

是的。 Peter 或 Mythili，您提到了前 6 位患者，在這種情況下，您會查看研究的導入部分，在該部分中，您會尋找可能發生的一些副作用——一組與另一組之間、3 位患者中的一位與另外 3 位患者之間。問題是，是否需要向 FDA 提供其他生物學數據來證明所使用的兩種特定試劑之間確實沒有差異？實際上，使用第一種試劑檢測的 3 名患者與使用第二種試劑檢測的 3 名患者實際上是完全相同的。我很好奇這些生物數據是什麼。

Sure, Tony. Let me turn the question over to Mythili, but let me start by clarifying something, which is that the patients in the safety lead-in are being primarily monitored for safety. That is that none of them has dose-limiting toxicities. And that is the primary endpoint for these 6 patients in the safety lead-in. But from a practical standpoint, we won't be doing anything meaningfully different with these patients and we will be for the patients that will be enrolled in the official Phase II. So with respect to efficacy, if we see efficacy from these patients, we will plan to continue to monitor and report them just the same way that we would the Phase II patients. But Mythili, why don't I turn it over to you for the technical answer to Tony's question?

當然，托尼。讓我把這個問題交給 Mythili，但首先讓我澄清一點，那就是安全引導區的病人主要受到安全監控。也就是說，它們都沒有劑量限制性毒性。這就是安全導入階段這 6 位病患的主要終點。但從實際角度來看，我們不會對這些患者採取任何有意義的不同措施，我們只會針對參加正式 II 期臨床試驗的患者採取這些措施。因此，就療效而言，如果我們看到這些患者的療效，我們將計劃繼續以與 II 期患者相同的方式監測和報告他們。但是 Mythili，我為什麼不把 Tony 的問題的技術答案交給你呢？

Yes. Thank you for your question, Tony. And Peter is exactly right. We're really only focused on the dose-limiting toxicities that will be reported to the FDA, if any issues arise. We don't anticipate any differences in terms of the safety profile between the 2 cohorts. And beyond that, there's really nothing additional that we need to report to the FDA. So in terms of other biological data, that's already in terms of the reagents, manufacturing and similarities, all of that has already been provided. So it's really just this last piece of safety data that will be provided before opening up the main portion of the Phase II.

是的。謝謝你的提問，托尼。彼得說得完全正確。我們實際上只關注劑量限制毒性，如果出現任何問題，我們會向 FDA 報告。我們預計這兩個群體的安全性不會有任何差異。除此之外，我們真的沒有什麼需要向 FDA 報告的了。因此，就其他生物數據而言，包括試劑、製造和相似性，所有這些都已經提供了。因此，這實際上只是在第二階段主要部分開放之前提供的最後一部分安全資料。

Thanks, Mythili. Yes, Tony, that's a distinction that is sometimes lost and that the safety lead-in is really only intended to ensure that there are no DLTs from the product, but the issue of comparability has already been settled with the agency.

謝謝，Mythili。是的，托尼，這種區別有時會被忽略，安全引入實際上只是為了確保產品沒有 DLT，但可比性問題已經與該機構解決了。

We've reached end of our question-and-answer session. I'd like to turn the floor back over to Peter for you further closing comments.

我們的問答環節已經結束。我想把發言權交還給彼得，請他進一步發表結束語。

Thank you all for joining us today here on our first quarter earnings call. We appreciate your support, and we hope that you all stay safe and well during this pandemic. Have a nice evening.

感謝大家今天參加我們的第一季財報電話會議。我們感謝您的支持，並希望大家在這次疫情期間都能平安健康。祝你今晚愉快。

Thank you. That does conclude today's teleconference and webcast. You may disconnect your line at this time, and have a wonderful day. We thank you for your participation today.

謝謝。今天的電話會議和網路直播到此結束。此時您可以斷開線路，祝您有美好的一天。我們感謝您今天的參與。