Gamida Cell Ltd (GMDA) 2022 Q4 法說會逐字稿

Ladies and gentlemen, thank you for standing by. Welcome to the Gamida Cell's conference call for the full-year 2022 financial results. My name is Catherine, and I'll be your operator for today's call. Please be advised that this call is being recorded at Gamida Cell's request. I would now like to introduce your host for today, [Mike Koskowski] of Gamida Cell corporate communications. Please go ahead.

女士們，先生們，謝謝你們的支持。歡迎參加 Gamida Cell 的 2022 年全年財務業績電話會議。我叫凱瑟琳，今天的電話我是你的接線員。請注意，此次通話是應 Gamida Cell 的要求進行錄音的。我現在想介紹一下今天的主持人，Gamida Cell 公司通訊部的 [Mike Koskowski]。請繼續。

Thank you, Catherine, and good morning, everyone. Welcome to today's call, during which we will provide an update on the company and review our financial results for the full year of 2022. Earlier this morning, we issued a press release summarizing our financial results and providing a business update, which is available on our website at www.gamida-cell.com.

謝謝你，凱瑟琳，大家早上好。歡迎來到今天的電話會議，在此期間我們將提供公司的最新情況並審查我們 2022 年全年的財務業績。今天早上早些時候，我們發布了一份新聞稿，總結了我們的財務業績並提供了業務更新，可在我們的網站 www.gamida-cell.com。

Here with me on our call today are Abbey Jenkins, President and Chief Executive Officer; Ronit Simantov, Chief Medical Officer and Scientific Officer; Michele Korfin, Chief Operating Officer and Chief Commercial Officer; and Shai Lankry, Chief Financial Officer.

今天和我一起來的有總裁兼首席執行官 Abbey Jenkins； Ronit Simantov，首席醫療官兼科學官； Michele Korfin，首席運營官兼首席商務官；和首席財務官 Shai Lankry。

During this call, we may make forward-looking statements about our future expectations and plans, including with respect of the timing, and initiation, and progress of, and data reported from the preclinical and clinical trials of our product candidates; regulatory filings, including the review of the BLA for omidubicel by the FDA; commercialization planning efforts for potentially life-saving or curative, therapeutic, and commercial potential of Gamida Cell's product candidates, including GDA-201 and omidubicel; and our expectations regarding our projected cash, cash equivalents, and investments to be used for operating activities.

在此電話會議期間，我們可能會就我們未來的期望和計劃做出前瞻性陳述，包括關於我們候選產品的臨床前和臨床試驗的時間、啟動、進展和數據報告；監管文件，包括 FDA 對 omidubicel 的 BLA 審查； Gamida Cell 候選產品（包括 GDA-201 和 omidubicel）的潛在挽救生命或治療、治療和商業潛力的商業化規劃工作；以及我們對用於經營活動的預計現金、現金等價物和投資的預期。

Our actual results may differ materially from what we project today due to a number of important factors -- the scope, progress, and expansion of our clinical trials and impacts to the cost thereof; scientific, clinical, regulatory, and technical developments; those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics; and then the endeavor of building a business around such product candidates; as well as those considerations described in the risk factors sections of our most recent quarterly report on Form 10-Q and other filings that we make with the SEC from time to time.

由於許多重要因素——我們的臨床試驗的範圍、進展和擴展及其對成本的影響；我們的實際結果可能與我們今天的預測存在重大差異；科學、臨床、監管和技術發展；開發和商業化可安全有效地用作人類治療藥物的候選產品過程中固有的那些；然後努力圍繞這些候選產品建立業務；以及我們最近關於 10-Q 表的季度報告和我們不時向美國證券交易委員會提交的其他文件的風險因素部分中描述的那些考慮因素。

These forward-looking statements represent our views only as of today. And we caution you that we may not update them in the future, whether as a result of new information or future events, except as required by applicable law.

這些前瞻性陳述僅代表我們截至今天的觀點。我們提醒您，我們可能不會在未來更新它們，無論是由於新信息還是未來事件，除非適用法律要求。

Now, I'll turn the call over to our President and CEO, Abbey Jenkins.

現在，我將把電話轉給我們的總裁兼首席執行官 Abbey Jenkins。

Thank you, Mike and everyone who is joining us this morning. I want to start this call by grounding us in Gamida Cell's mission. When I joined the company last September, I was drawn to a commitment to discovering, developing, and delivering advanced cell therapies that offer patients with cancer hope for cures.

謝謝邁克和今天早上加入我們的每一個人。我想通過讓我們紮根於 Gamida Cell 的使命來開始這個電話會議。當我去年 9 月加入公司時，我被吸引到致力於發現、開發和提供先進的細胞療法，為癌症患者提供治癒的希望。

In our industry, particularly when we are focused on talking about our earning, we talk extensively about our science, potential indications, manufacturing, go-to-market plans, and projected revenues. As we do that, it's good to remember the North Star of unmet patient needs. Gamida Cell was founded to pursue those needs, and it elected to do so in one of the most challenging areas of medicine and science, stem cell transplant.

在我們的行業中，尤其是當我們專注於談論我們的收入時，我們會廣泛談論我們的科學、潛在適應症、製造、上市計劃和預計收入。當我們這樣做時，最好記住未滿足患者需求的北極星。 Gamida Cell 的成立就是為了滿足這些需求，它選擇在醫學和科學最具挑戰性的領域之一——幹細胞移植中實現這一目標。

We believe we have the opportunity to potentially save lives with our science. And so we aim to keep our focus squarely on our goal, as we make decisions about how to move forward as a company. Our belief has been and remains that Gamida Cell represents a compelling investment thesis. We have a robust short-term catalyst in the pending FDA approval of omidubicel, our lead product candidate; and great long-term potential in our pipeline of natural killer cell therapies, including clinical stage GDA-201 and our three engineered preclinical NK cell therapy candidates.

我們相信我們有機會用我們的科學拯救生命。因此，在我們決定如何作為一家公司向前發展時，我們的目標是將注意力集中在我們的目標上。我們一直堅信 Gamida Cell 代表了一個引人注目的投資主題。在我們的主要候選產品 omidubicel 即將獲得 FDA 批准時，我們擁有強大的短期催化劑；以及我們的自然殺傷細胞療法管道的巨大長期潛力，包括臨床階段的 GDA-201 和我們的三個工程臨床前 NK 細胞療法候選人。

In recent weeks, we had positive data presented on both omidubicel and GDA-201 at the Transplantation and Cellular Therapy, or TCT, meeting showing why we believe we are on the right path to bring meaningful innovation to patients with hematologic malignancies. Our intrinsic NK cell therapies, which are derived from healthy human donors are differentiated from other NK approaches and have data showing that they may not only stimulate direct cytotoxic effects that may trigger a response from the adaptive immune system which is very promising.

最近幾週，我們在移植和細胞治療 (TCT) 會議上展示了關於 omidubicel 和 GDA-201 的積極數據，這表明我們為什麼相信我們走在為血液系統惡性腫瘤患者帶來有意義的創新的正確道路上。我們的內在 NK 細胞療法源自健康的人類供體，與其他 NK 方法不同，並且有數據表明它們不僅可以刺激直接的細胞毒性作用，還可以觸發適應性免疫系統的反應，這是非常有前途的。

Unfortunately, we are in an extremely challenging economic environment. In our last earnings call, we guided a cash runway to the middle of 2023, and thus need to ensure that while we continue to pursue fundraising in support of bringing omidubicel to patients if approved, we also reduce expenses to preserve our cash runway. Therefore, today, we are taking decisive action with a strategic restructuring to prioritize our organizational efforts and resources around the commercial launch of omidubicel.

不幸的是，我們正處於一個極具挑戰性的經濟環境中。在我們上次的財報電話會議上，我們將現金跑道引導至 2023 年年中，因此需要確保在我們繼續籌款以支持將 omidubicel 帶給患者（如果獲得批准）的同時，我們還減少開支以維持我們的現金跑道。因此，今天，我們正在採取戰略重組的果斷行動，以圍繞 omidubicel 的商業發布優先考慮我們的組織工作和資源。

We are doing it because omidubicel has significant clinical evidence that it may increase access and improve outcomes for patients in need of an allogeneic stem cell transplant. If approved, omidubicel will be the first advanced cell therapy for stem cell transplant and may mark the only hope for the approximately [1,200] patients that go on transplants in each year, including those who are ethnically or racially diverse.

我們這樣做是因為 omidubicel 有重要的臨床證據表明它可以增加需要同種異體幹細胞移植的患者的機會並改善結果。如果獲得批准，omidubicel 將成為第一個用於乾細胞移植的先進細胞療法，並且可能標誌著每年大約 [1,200] 名接受移植的患者的唯一希望，其中包括那些不同種族或種族的患者。

The safety profile of omidubicel in our Phase 3 clinical study was consistent with the expected toxicities of an allogeneic stem cell transplant following conditioning therapy. And there was no increase in adverse events, serious adverse events, or infusion reactions in the omidubicel arm compared to control.

在我們的 3 期臨床研究中，omidubicel 的安全性與調節療法後同種異體幹細胞移植的預期毒性一致。與對照組相比，omidubicel 組的不良事件、嚴重不良事件或輸液反應沒有增加。

We had productive interactions with the FDA, including a recently completed late-cycle meeting earlier this quarter and a pre-licensing inspection of our manufacturing facility in the fourth quarter of 2022, with no 483 observations to date, significantly derisking our path to approval. We've had positive interactions with patients, transplanters, and advocacy leaders in the stem cell transplant space, encouraging us that they are looking forward to having the opportunity to have omidubicel as a new alternate donor source.

我們與 FDA 進行了富有成效的互動，包括本季度早些時候最近完成的周期後期會議以及 2022 年第四季度對我們的製造工廠進行的許可前檢查，迄今為止沒有 483 項觀察結果，這大大降低了我們獲得批准的途徑。我們與乾細胞移植領域的患者、移植者和倡導領袖進行了積極的互動，這讓我們感到鼓舞，他們期待有機會將 omidubicel 作為新的替代供體來源。

We believe omidubicel can fulfill our mission of having a meaningful, potentially life-saving, impact on patients. With today's announcement that we are strategically restructuring the organization to dedicate the vast majority of our resources to bring omidubicel to patients in the US, we've made the difficult decision to discontinue development of our early pipeline candidates, GDA-301, GDA-501, and GDA-601. We will, however, continue enrollment in our Phase 1 clinical trial of GDA-201.

我們相信 omidubicel 可以完成我們的使命，即對患者產生有意義的、可能挽救生命的影響。隨著今天宣布我們正在對組織進行戰略重組，以將我們的絕大部分資源用於為美國患者提供奧米比賽，我們做出了艱難的決定，即停止開發我們的早期管道候選藥物 GDA-301、GDA-501和 GDA-601。但是，我們將繼續參加 GDA-201 的 1 期臨床試驗。

Let me be clear, we believe in the scientific and therapeutic potential of these candidates, in fact, now, more than ever, based on the data presented at the recent medical meetings. These changes are being made solely for economic reasons.

讓我明確一點，我們相信這些候選人的科學和治療潛力，事實上，根據最近醫學會議上提供的數據，現在比以往任何時候都更加相信。這些變化完全是出於經濟原因。

Further to these changes, we will implement a headcount reduction of 17% beginning this week and extending into the second quarter, with the majority of impacted headcount tied to the discontinuation of our NK preclinical pipeline development. We will also close our operations in Jerusalem and consolidate Israeli operations into our state-of-the-art manufacturing site in Kiryat Gat. Finally, we will slow our ramp of hiring and expenses related to the launch of omidubicel.

除了這些變化，我們將從本週開始裁員 17%，並延續到第二季度，其中大部分受影響的員工與我們停止 NK 臨床前管道開發有關。我們還將關閉我們在耶路撒冷的業務，並將以色列的業務整合到我們位於 Kiryat Gat 的最先進的製造基地。最後，我們將放慢與推出 omidubicel 相關的招聘和支出。

These measures, collectively, will extend our cash runway through the third quarter. We believe these changes were make Gamida Cell a more focused, prioritized, and attractive investment opportunity and create a path for near-term value creation with the potential approval and launch of omidubicel. While we will be focused on bringing omidubicel to patients in the US if approved in May, we will also explore strategic options, including potential US and global partnerships that may enable us to do so with a more significant level of investment than our current resources allow.

這些措施共同將我們的現金跑道延長至第三季度。我們認為，這些變化使 Gamida Cell 成為一個更加專注、優先和有吸引力的投資機會，並通過 omidubicel 的潛在批准和推出為近期價值創造創造了途徑。如果在 5 月獲得批准，我們將專注於將 omidubicel 帶給美國的患者，同時我們還將探索戰略選擇，包括潛在的美國和全球合作夥伴關係，這可能使我們能夠以比我們當前資源允許的更大水平的投資來實現這一目標.

This has been an extremely difficult decision. Our teams have worked hard on the discovery work to support these NK programs and prepare for the launch of omidubicel in a more robust manner. We are encouraged by the initial clinical efficacy and safety profile of GDA-201 and our preclinical pipeline, but we must take these steps to sustain the company and position ourselves to bring omidubicel to the patients who need it.

這是一個極其艱難的決定。我們的團隊一直致力於發現工作，以支持這些 NK 計劃，並以更穩健的方式為 omidubicel 的推出做準備。我們對 GDA-201 的初步臨床療效和安全性以及我們的臨床前管道感到鼓舞，但我們必須採取這些步驟來維持公司並定位自己，將 omidubicel 帶給需要它的患者。

Finally, last week, we announced the appointment of Shawn Cline Tomasello as Chairwoman of our Board of Directors. Shawn, who has been on our Board since 2019, is a highly respected and seasoned biotech executive with tremendous experience in all dimensions of our industry, including corporate strategy and commercialization of innovative hematology, oncology, and cell therapy products.

最後，上週，我們宣布任命 Shawn Cline Tomasello 為董事會主席。肖恩自 2019 年以來一直在我們的董事會任職，是一位備受尊敬且經驗豐富的生物技術高管，在我們行業的各個方面都擁有豐富的經驗，包括公司戰略和創新血液學、腫瘤學和細胞治療產品的商業化。

On behalf of the Board and the entire company, we thank Robert Blum, our previous chairman, for his dedicated service over the past five years and look forward to Shawn's leadership as we approach our May 1 PDUFA date. These changes, both the strategic restructuring of the company and the evolution of the Board, support our transformation from a clinical-stage to commercial-stage company.

我們代表董事會和整個公司，感謝我們的前任董事長羅伯特·布魯姆在過去五年中的熱忱服務，並期待在我們接近 5 月 1 日 PDUFA 日期時肖恩的領導。這些變化，包括公司的戰略重組和董事會的演變，支持我們從臨床階段公司向商業階段公司的轉型。

Now, I'll turn the turn the call over to Ronit to take us through key data supporting omidubicel as we prepare for approval and commercialization. Ronit?

現在，我將把電話轉給 Ronit，在我們準備批准和商業化時，帶我們了解支持 omidubicel 的關鍵數據。羅尼特？

Thanks, Abbey, and good morning, everyone. Thank you for joining us on our call this morning. In light of our approaching PDUFA date and the strategic restructuring outlined by Abbey, I'll focus on the latest regulatory and clinical updates on omidubicel before Michele discusses our commercialization plan and manufacturing progress.

謝謝，修道院，大家早上好。感謝您今天早上加入我們的電話會議。鑑於我們即將到來的 PDUFA 日期和 Abbey 概述的戰略重組，在 Michele 討論我們的商業化計劃和製造進展之前，我將重點關注 omidubicel 的最新監管和臨床更新。

First, as Abbey mentioned, we held our late-cycle meeting with the FDA as planned in the first quarter. Our discussions with the agency continue to be productive as we head toward our PDUFA date of May 1. We do not anticipate an advisory committee, and there have been no significant safety or efficacy issues to date.

首先，正如 Abbey 提到的，我們在第一季度按計劃與 FDA 舉行了後期會議。在我們接近 5 月 1 日的 PDUFA 日期時，我們與該機構的討論繼續富有成效。我們預計不會有諮詢委員會，並且迄今為止沒有重大的安全性或有效性問題。

As a reminder, the study to support the regulatory submission was a successful Phase 3 global randomized study that met its primary and all secondary endpoints. The study randomized 125 patients aged 12 to 65 with high-risk hematologic malignancy who needed an allogeneic stem cell transplant and had no readily available matched donor.

提醒一下，支持監管提交的研究是一項成功的第 3 階段全球隨機研究，達到了其主要和所有次要終點。該研究將 125 名 12 至 65 歲的高危血液惡性腫瘤患者隨機分組，這些患者需要異基因幹細胞移植並且沒有現成的匹配供體。

The study demonstrated a median time to neutrophil engraftment in the as treated per protocol population of 10 days for patients transplanted with omidubicel compared to 20 days for the comparative group transplanted with standard cord blood. These results were both statistically significant and clinically meaningful given the importance of neutrophil engraftment for the key milestones in the recovery of patients who have undergone stem cell transplant.

該研究表明，接受 omidubicel 移植的患者按照方案治療的中性粒細胞植入的中位時間為 10 天，而接受標準臍帶血移植的對照組為 20 天。鑑於中性粒細胞植入對於接受干細胞移植的患者康復的關鍵里程碑的重要性，這些結果既具有統計學意義又具有臨床意義。

Subsequent data has also shown reduced healthcare resource utilization and improved patient-reported outcomes with omidubicel. We have had two recent data presentations on omidubicel; a key medical meeting; a podium presentation at the American Society of Hematology meeting in December, as discussed on our last earnings call; and an oral presentation at TCT, as referenced by Abbey, in February.

隨後的數據還顯示，使用奧米比賽可降低醫療保健資源利用率並改善患者報告的結果。我們最近有兩個關於 omidubicel 的數據介紹；一次重要的醫學會議；正如我們在上次財報電話會議上討論的那樣，在 12 月的美國血液學會會議上發表演講；以及 2 月份在 TCT 上的口頭報告，正如 Abbey 所引用的那樣。

In our presentation at ASH, we compared the results of our Phase 3 trial with results from existing donor sources, matched unrelated donors, mismatched unrelated donors, and haploidentical donors, using data reported to CIBMTR. After adjusting for baseline variable, the results showed that omidubicel was associated with a more rapid rate of neutrophil engraftment, with a median of 10 days for omidubicel versus 15 to 20 days for the other graft forces.

在我們在 ASH 的演講中，我們使用報告給 CIBMTR 的數據，將我們的 3 期試驗結果與現有供體來源、匹配的無關供體、不匹配的無關供體和半相合供體的結果進行了比較。調整基線變量後，結果顯示奧米比賽與更快的中性粒細胞植入率相關，奧米比賽的中位植入時間為 10 天，而其他移植力為 15 至 20 天。

There were comparable rate of grades 3 to 4 acute graft versus host disease, or GVHD, and of chronic GVHD, as well as comparable overall survival. These results suggest that omidubicel may be an effective and important new graft source option, making allogeneic stem cell transplants available to more patients.

3 至 4 級急性移植物抗宿主病 (GVHD) 和慢性 GVHD 的發生率相當，總生存率也相當。這些結果表明，omidubicel 可能是一種有效且重要的新移植物來源選擇，使同種異體幹細胞移植可用於更多患者。

In the omidubicel presentation in February at TCT, we presented new data characterizing peripheral blood lymphocytes measured in correlation with time to neutrophil and platelet engraftment in omidubicel cell transplanted and standard cord blood transplanted patients. These data showed that, at seven days post transplant, omidubicel transplanted patients showed a statistically significant correlation between CD3 and CD4 positive T-cell count and time to neutrophil engraftment.

在 2 月 TCT 的 omidubicel 演示中，我們提供了新數據，這些數據表徵了在 omidubicel 細胞移植和標準臍帶血移植患者中測量的與中性粒細胞和血小板植入時間相關的外周血淋巴細胞。這些數據表明，在移植後第 7 天，omidubicel 移植患者在 CD3 和 CD4 陽性 T 細胞計數與中性粒細胞植入時間之間顯示出統計學上顯著的相關性。

Patients transplanted with standard cord blood showed no such correlations at day seven post transplant and only began to show immune recovery starting at 14 days. These data support past findings that omidubicel stimulates faster immune response in standard cord blood, which may be a contributing mechanism resulting in a lower incidence of serious bacterial, fungal, and viral infections in omidubicel transplanted patients.

移植標準臍帶血的患者在移植後第 7 天沒有顯示出這種相關性，並且在第 14 天才開始顯示免疫恢復。這些數據支持過去的發現，即 omidubicel 在標準臍帶血中刺激更快的免疫反應，這可能是導致 omidubicel 移植患者嚴重細菌、真菌和病毒感染髮生率較低的一種機制。

We also have a publication in press in the journal of Transplantation and Cellular Therapy now available online, reporting on long-term follow-up of patients transplanted with omidubicel across five clinical trials. The analysis showed a three-year overall survival of 62.5% and disease-free survival of 54%. With up to 10 years of follow-up, omidubicel showed durable hematopoiesis.

我們還在《移植和細胞療法》雜誌上發表了一篇出版刊物，現已在線發布，報告了在五項臨床試驗中接受奧米比賽移植的患者的長期隨訪情況。分析顯示三年總生存率為 62.5%，無病生存率為 54%。經過長達 10 年的隨訪，omidubicel 顯示出持久的造血作用。

We are very excited by these data showing the long-term safety and durability of omidubicel. The data in these presentations and publications continue to support the clinical (technical difficulty) that we prepare to bring a potentially curative therapy to patients (technical difficulty)

我們對這些顯示 omidubicel 的長期安全性和耐用性的數據感到非常興奮。這些演示文稿和出版物中的數據繼續支持我們準備為患者帶來潛在治愈性療法（技術難度）的臨床（技術難度）

With that, I will turn the call over to Michele, who will provide an update on our plans to launch omidubicel in the US market upon potential FDA approval. Michele?

有了這個，我將把電話轉給 Michele，他將提供我們在 FDA 可能批准後在美國市場推出 omidubicel 的計劃的最新情況。米歇爾？

Thank you, Ronit, and good morning, everyone. I want to provide an update on where we are in terms of our launch plans for omidubicel in the US market. As Ronit shared, omidubicel continues to amass a body of evidence that shows it may be an effective and important new donor source option.

謝謝你，Ronit，大家早上好。我想提供有關我們在美國市場推出 omidubicel 計劃的最新情況。正如 Ronit 分享的那樣，omidubicel 繼續收集大量證據，表明它可能是一種有效且重要的新供體來源選擇。

We continue to have positive interactions with transplanters, transplant center teams, and payers as we prepare for launch. For transplanters, omidubicel's clinical outcomes, including rapid time to neutrophil engraftment, durable response, and quality of life, are appealing and a new donor source option. As we approach our May 1 PDUFA date, we continue to work toward maximizing a positive patient and transplant center experience when using omidubicel as the donor source of choice.

在我們準備推出時，我們將繼續與移植者、移植中心團隊和付款人進行積極的互動。對於移植者而言，omidubicel 的臨床結果（包括中性粒細胞植入的快速時間、持久的反應和生活質量）很有吸引力，並且是一種新的供體來源選擇。隨著 5 月 1 日 PDUFA 日期的臨近，我們將繼續努力在使用 omidubicel 作為首選供體來源時最大限度地提高患者和移植中心的積極體驗。

From a commercial standpoint, our research shows an unmet need for a new donor source for allogeneic stem cell transplant that potentially increases patient access to transplant and improves outcomes over existing donor sources. From an access standpoint, about 1,200 patients each year are eligible for stem cell transplant but do not receive one because they cannot find a donor. Omidubicel's less stringent matching criteria offers hope for those patients that they will be able to find a donor source.

從商業的角度來看，我們的研究表明，對用於同種異體幹細胞移植的新供體來源的需求未得到滿足，這可能會增加患者獲得移植的機會，並改善現有供體來源的結果。從准入的角度來看，每年約有 1,200 名患者有資格進行幹細胞移植，但由於找不到供體而未接受。 Omidubicel 不太嚴格的匹配標準為那些能夠找到供體來源的患者帶來了希望。

We know this is, at least in part, a health disparities issue, with patients who are White or Caucasian, having a higher chance of finding a match. For example, patients who are White have a 79% chance of finding a match in the donor registry, while patients who are Black or African-American have just a 29% chance. And this is according to Be The Match.

我們知道，至少在某種程度上，這是一個健康差異問題，白人或高加索人患者更有可能找到匹配對象。例如，白人患者有 79% 的機會在供體登記處找到匹配，而黑人或非裔美國人患者只有 29% 的機會。這是根據 Be The Match 的說法。

Our Phase 3 study has over 40% of patients enrolled who are racially and ethnically diverse. This demonstrates both the unmet need for non-Caucasian patients and also the ability for omidubicel to address this unmet need. In addition, there are opportunities to improve outcomes by using omidubicel when it addresses limitations or mitigates risks of other donor sources.

我們的 3 期研究有超過 40% 的患者參加，他們具有不同的種族和民族特徵。這表明非白種人患者的未滿足需求以及奧米比賽解決這一未滿足需求的能力。此外，在解決限製或減輕其他捐助者來源的風險時，有機會通過使用 omidubicel 來改善結果。

These include -- so first off, omidubicel has demonstrated statistically significant faster time to neutrophil engraftment versus standard cord blood, which currently represents about 5% of the donor sources -- and this was in our Phase 3 study -- and statistically significant faster time to neutrophil engraftment versus other donor types, which was presented in the ASH real-world evidence analysis that Ronit previously mentioned. Secondly, omidubicel offers a faster time from donor source identification to transplant as compared to matched unrelated donors and mismatched unrelated donors, with availability from omidubicel being approximately 30 days from start of manufacturing as compared to approximately two to three months required to align an unrelated donor to a patient.

其中包括 - 首先，omidubicel 已經證明與標準臍帶血相比，中性粒細胞植入的時間在統計學上顯著更快，標準臍帶血目前約佔供體來源的 5% - 這在我們的第 3 階段研究中 - 並且在統計學上顯著更快中性粒細胞植入與其他供體類型的比較，這在 Ronit 之前提到的 ASH 真實世界證據分析中有所介紹。其次，與匹配的無關供體和不匹配的無關供體相比，omidubicel 提供了更快的從供體來源識別到移植的時間，從製造開始大約 30 天就可以從 omidubicel 獲得，而匹配無關供體需要大約兩到三個月給病人。

Matched unrelated donors constitute about 43% of stem cell transplants, and mismatched unrelated donors are about 7%. And finally, compared to haploidentical donors, which comprise about 24% of transplants, omidubicel offers a faster time to neutrophil engraftment, as presented in our oral presentation at ASH. And omidubicel did not require post-transplant cyclophosphamide, or PTCy, in our Phase 3 study.

匹配的無關供體約佔幹細胞移植的 43%，不匹配的無關供體約佔 7%。最後，與約佔移植物 24% 的半相合供體相比，omidubicel 提供了更快的中性粒細胞植入時間，正如我們在 ASH 上的口頭報告中所展示的那樣。在我們的 3 期研究中，omidubicel 不需要移植後環磷酰胺或 PTCy。

PTCy helps to mitigate graft versus host disease, although cyclophosphamide is cardio toxic. So it could present risks and/or adverse events for patients. In regard to manufacturing, our state-of-the-art manufacturing facility in Kiryat Gat in Israel is already producing omidubicel for clinical study patients and is ready for commercial production.

PTCy 有助於減輕移植物抗宿主病，儘管環磷酰胺具有心臟毒性。因此，它可能會給患者帶來風險和/或不良事件。在製造方面，我們位於以色列 Kiryat Gat 的最先進的製造工廠已經在為臨床研究患者生產 omidubicel，並準備好進行商業生產。

We're showing the ability to deliver omidubicel back to a transplant center within approximately 30 (technical difficulty) and we have completed our Israeli Ministry of Health and FDA pre-licensing inspections with no 483 observations to date. This facility is also modular, so we can add additional cores for additional capacity as demand grows.

我們展示了在大約 30 分鐘（技術難度）內將 omidubicel 送回移植中心的能力，並且我們已經完成了以色列衛生部和 FDA 的許可前檢查，迄今為止沒有 483 次觀察。該設施也是模塊化的，因此我們可以隨著需求的增長添加額外的核心以獲得額外的容量。

We have a key partnership with Be The Match. They are very committed to partnering with us to introduce this new advanced cell therapy. Transplant centers are experienced that using the Be The Match resources to identify donor sources. We partnered with Be The Match for our Phase 3 study, and we have an established partnership with them, following potential FDA approval, to support transplant center access to omidubicel.

我們與 Be The Match 建立了重要的合作夥伴關係。他們非常致力於與我們合作，推出這種新的先進細胞療法。移植中心在使用 Be The Match 資源確定供體來源方面經驗豐富。我們與 Be The Match 合作進行了我們的 3 期研究，並且在獲得 FDA 的潛在批准後，我們與他們建立了合作夥伴關係，以支持移植中心獲得 omidubicel。

Upon approval, we are ready to deliver omidubicel to transplant centers. One of the most important things we must do with onboard transplant centers is to ensure they have the necessary procedures and logistics to deliver a cell therapy like omidubicel to patients in need. We are finding strong interest from transplant centers, including centers that were not participants in our clinical trials.

經批准後，我們準備將 omidubicel 交付給移植中心。我們必須對船上移植中心做的最重要的事情之一是確保他們擁有必要的程序和後勤保障，以向有需要的患者提供像 omidubicel 這樣的細胞療法。我們發現移植中心表現出濃厚的興趣，包括未參與我們臨床試驗的中心。

The strategic restructuring Abbey described at the beginning of the call aims to expand the team responsible for the launch of omidubicel, although slowing the ramp of hiring and expense as compared to our plans earlier this year. The vast majority of our resources will be focused on omidubicel and supporting this launch. The keys to launch success will be the onboarding of transplant centers and ensuring a positive experience for patients and care teams.

Abbey 在電話會議開始時描述的戰略重組旨在擴大負責推出 omidubicel 的團隊，儘管與我們今年早些時候的計劃相比，招聘和費用的增長有所放緩。我們的絕大部分資源將集中在 omidubicel 上並支持此次發布。啟動成功的關鍵將是移植中心的入職並確保為患者和護理團隊提供積極的體驗。

In the US, 70 transplant centers performed 80% of all transplants. We had initially anticipated our early launch sites would be focused on clinical trial sites. We are now encouraged by positive feedback coming from top transplant centers that were not part of our clinical trials.

在美國，70 個移植中心進行了 80% 的移植手術。我們最初預計我們的早期啟動站點將專注於臨床試驗站點。我們現在對來自頂級移植中心的積極反饋感到鼓舞，這些中心不屬於我們的臨床試驗。

So based on the level of resourcing we have available, we are preparing approximately 10 to 15 of those top 70 centers for the initial launch. And we are optimistic this could scale up once additional resources are available. We have the key objective of assuring a positive patient experience, including essential programs such as education and training sessions and process and logistic reviews in place.

因此，根據我們可用的資源水平，我們正在準備 70 個頂級中心中的大約 10 到 15 個用於初始啟動。我們樂觀地認為，一旦有更多資源可用，這可能會擴大。我們的主要目標是確保積極的患者體驗，包括必要的計劃，例如教育和培訓課程以及適當的流程和後勤審查。

Although omidubicel has a less stringent matching criteria than other sources, there's still a matching requirement. So we must work with centers to assure appropriate chain of identity and chain of custody. In addition, at the time of potential FDA approval, we will have our patient support systems in place through Gamida Cell Assist to facilitate access.

儘管 omidubicel 的匹配標準不如其他來源嚴格，但仍然存在匹配要求。因此，我們必須與中心合作，以確保適當的身份鍊和監管鏈。此外，在可能獲得 FDA 批准時，我們將通過 Gamida Cell Assist 建立我們的患者支持系統，以促進訪問。

In regards to patient access, we've already met with US payers, including payers that cover more than 90% of commercially covered lives. We also have an established ICD-10 PCS code, which has already been granted by CMS. Payers have indicated they anticipate covering a one-time therapy with curative intent upon FDA approval. And we also have discussed path to reimbursement with both commercial payers and CMS.

關於患者准入，我們已經與美國付款人會面，包括覆蓋超過 90% 商業承保生命的付款人。我們還有一個已建立的 ICD-10 PCS 代碼，該代碼已被 CMS 授予。付款人表示，他們預計在獲得 FDA 批准後，將涵蓋具有治愈目的的一次性治療。我們還與商業付款人和 CMS 討論了報銷途徑。

Over the last few years, we have conducted extensive market insight gathering, including four independent studies with consistent findings on the potential for omidubicel. With the combination of increased access and improved outcomes, we believe omidubicel has the potential to capture 20% to 25% of the addressable patient population at peak share, which would equate to 2,000 to 2,500 patients per year in the US.

在過去的幾年裡，我們進行了廣泛的市場洞察收集，包括四項獨立研究，這些研究對 omidubicel 的潛力有一致的發現。隨著可及性的增加和結果的改善相結合，我們認為 omidubicel 有可能在峰值份額時捕獲 20% 至 25% 的可尋址患者群體，這相當於美國每年 2,000 至 2,500 名患者。

We expect our uptake to come through the combination of market expansion and share shift. The market expansion will come from those approximately 1,200 patients a year we estimate go on transplant because they cannot find a match. And the share shift potential has been very consistent in our market insight studies focused mainly on the strength of our clinical data and our 30-day turnaround time.

我們預計我們的吸收將通過市場擴張和份額轉移相結合來實現。市場擴張將來自我們估計每年約有 1,200 名患者因找不到匹配而接受移植。在我們的市場洞察研究中，份額轉移的潛力一直非常一致，主要關注我們臨床數據的強度和我們 30 天的周轉時間。

Although we anticipate share capture from all donor sources, we anticipate the early stages of the launch will begin from standard cord and mismatched unrelated donor. And once transplanters gain experience, they will consider omidubicel in lieu of haplo and other sources.

儘管我們預計會從所有捐助者來源獲取份額，但我們預計啟動的早期階段將從標準臍帶和不匹配的無關捐助者開始。一旦移植者獲得經驗，他們就會考慮用 omidubicel 代替 haplo 和其他來源。

Again, back to our mission. This company was built to deliver potentially curative therapies to patients. We have a compelling therapy in omidubicel that, if approved, may increase access and improve outcomes, addressing critical unmet needs. We are now putting virtually everything we have behind this launch with the expectation that this could have a meaningful and positive impact on patients' lives.

再次回到我們的使命。這家公司的建立是為了向患者提供潛在的治療方法。我們在 omidubicel 中有一種令人信服的療法，如果獲得批准，它可能會增加可及性並改善結果，從而解決關鍵的未滿足需求。現在，我們將幾乎所有的一切都投入到此次發布中，期望這能對患者的生活產生有意義和積極的影響。

From a manufacturing, account management, and market access perspective, we are in a position to launch. We are very excited at the prospect of launching omidubicel in the US And through increased investment and/or strategic partnership, we have the potential to reach more transplant centers and patients at a faster pace.

從製造、客戶管理和市場准入的角度來看，我們有能力推出。我們對在美國推出 omidubicel 的前景感到非常興奮，並且通過增加投資和/或戰略合作夥伴關係，我們有可能以更快的速度接觸到更多的移植中心和患者。

I will now turn the call over to Shai to review our financial results.

我現在將電話轉給 Shai 以審查我們的財務業績。

Thank you, Michele, and good morning, everyone. Today, we will summarize our financial results for 2022. As Abbey mentioned earlier, today's strategic restructuring addresses a number of needs and reflect the more focused use of our resources, prioritizing the launch of omidubicel, if approved, and extending our cash runway through Q3.

謝謝你，米歇爾，大家早上好。今天，我們將總結我們 2022 年的財務業績。正如 Abbey 之前提到的，今天的戰略重組解決了許多需求，並反映了我們更集中地使用我們的資源，優先推出 omidubicel（如果獲得批准），並將我們的現金跑道延長至第三季度.

As of December 31, 2022, our total cash position, including the recent $25 million convertible loan with [COVID] Highbridge in December, was approximately $64.7 million compared to $95.9 million as of December 31, 2021.

截至 2022 年 12 月 31 日，我們的總現金頭寸約為 6,470 萬美元，而截至 2021 年 12 月 31 日為 9,590 萬美元，包括最近在 12 月向 [COVID] Highbridge 提供的 2,500 萬美元可轉換貸款。

Research and development expenses for 2022 were $42.7 million compared to $50.2 million in 2021. The decrease was mainly due to a $9.6 million decrease in clinical and operational activities related to the conclusion of our Phase 3, offset by an increase of $1.1 million in T&E and other expenses, as well as $1 million decrease in Israel Innovation Authority Grant.

2022 年的研發費用為 4270 萬美元，而 2021 年為 5020 萬美元。減少的主要原因是與我們的第三階段結束相關的臨床和運營活動減少了 960 萬美元，被 T&E 增加 110 萬美元和其他費用，以及以色列創新局撥款減少 100 萬美元。

Commercial expenses for the year were $12.9 million compared to $20 million in prior year. The decrease was primarily due to $8.2 million decrease in launch readiness activities, offset by an increase of $1.1 million in headcount-related expenses.

與上一年的 2000 萬美元相比，本年度的商業費用為 1290 萬美元。減少的主要原因是發射準備活動減少了 820 萬美元，被與人事相關的費用增加 110 萬美元所抵消。

General and administrative expenses for 2022 were $19.4 million compared to $17 million for 2020. The increase was mainly driven by an increase of $1.4 million attributed to our corporate headquarters and headcount-related expenses as well as a $1 million increase in professional services.

2022 年的一般和行政費用為 1,940 萬美元，而 2020 年為 1,700 萬美元。增加的主要原因是我們公司總部和人員相關費用增加了 140 萬美元，以及專業服務增加了 100 萬美元。

Finance expenses, net, were $4.4 million in 2022 compared to $2.6 million in 2021. The increase was primarily due to expenses relating to the Highbridge convertible loan, we signed back in December.

財務費用淨額在 2022 年為 440 萬美元，而 2021 年為 260 萬美元。增加的主要原因是與我們在 12 月簽署的 Highbridge 可轉換貸款相關的費用。

Net loss for the year of $79.4 million is compared to a net loss of $89.8 million in 2021. As I mentioned before, the actions we are announcing today will extend our cash runway through Q3. This cash runway guidance is based on our current operational plan and excludes any additional funding that may be received for business development activities that may be undertaken.

與 2021 年的淨虧損 8980 萬美元相比，今年的淨虧損為 7940 萬美元。正如我之前提到的，我們今天宣布的行動將把我們的現金跑道延長到第三季度。該現金跑道指南基於我們當前的運營計劃，不包括可能為可能開展的業務發展活動而收到的任何額外資金。

With that, I will turn the call back over to Abbey..

有了這個，我會把電話轉回修道院..

Thank you, Shai. Before I turn the call back over to Catherine for questions, I want to bring us back around to the beginning and summarize some key points from our discussion today. We believe in bringing potentially curative advanced cell therapies to patients. That's what Gamida Cell was founded to; it's our mission.

謝謝你，夏伊。在我將電話轉回給凱瑟琳提問之前，我想讓我們回到開頭並總結我們今天討論的一些要點。我們相信為患者帶來具有潛在治愈性的先進細胞療法。這就是 Gamida Cell 成立的目的；這是我們的使命。

As we were on the precipice of bringing omidubicel to market, we are ready. We are ready from a manufacturing and regulatory perspective. We had productive interactions with regulators and have completed both a pre-license inspection and late cycle meeting as we head toward our PDUFA date of May 1, which is significantly derisking our path to approval.

當我們即將將 omidubicel 推向市場時，我們已經做好了準備。我們從製造和監管的角度準備好了。我們與監管機構進行了富有成效的互動，並在我們接近 5 月 1 日的 PDUFA 日期時完成了許可前檢查和後期週期會議，這大大降低了我們獲得批准的風險。

We are ready from a commercial and medical perspective. We have strong market insights and a clear strategy that is focused on onboarding transplant centers and ensuring a positive experience for patients and transplant center teams.

我們從商業和醫療的角度準備好了。我們擁有強大的市場洞察力和明確的戰略，專注於入職移植中心並確保為患者和移植中心團隊提供積極的體驗。

We are ready as an organization. We are making the difficult decisions through the strategic restructuring of the company that will enable us to launch this therapy successfully. Our launch will ramp more slowly than we were planning at the end -- at the beginning of 2022 -- at the end of 2022, but we are committed to ensuring this meaningful therapy gets to patients and that transplant centers have a positive onboarding experience. While we are doing everything we can do to ensure a successful US launch, we will be exploring strategic options, including potential US and global partnerships, to enable a more robust commercial effort.

作為一個組織，我們已準備就緒。我們正在通過公司的戰略重組做出艱難的決定，這將使我們能夠成功推出這種療法。我們的啟動速度將比我們計劃在 2022 年底——2022 年初——2022 年底的計劃慢，但我們致力於確保這種有意義的治療能惠及患者，並確保移植中心擁有積極的入職體驗。在我們竭盡全力確保在美國成功推出的同時，我們將探索戰略選擇，包括潛在的美國和全球合作夥伴關係，以實現更強大的商業努力。

Omidubicel, if approved, has the potential to increase access and improve outcomes. It can make a meaningful difference in the lives of people who need a potentially curative therapy. This will be our primary focus as we go forward, and we are ready.

如果獲得批准，Omidubicel 有可能增加可及性並改善結果。它可以對需要潛在治愈療法的人的生活產生有意義的影響。這將是我們前進的主要重點，我們已做好準備。

Now, let's open the call for questions. Catherine, over to you.

現在，讓我們開始提問。凱瑟琳，交給你了。

Thank you. (Operator Instructions) Edward Tenthoff, Piper Sandler.

謝謝。 （操作員說明）Edward Tenthoff，Piper Sandler。

Great. Thank you very much. I'm sorry for the difficult decisions with respect to the headcount. But I know the focus on omidubicel will pay off. I was pleased to hear about the inspections. Can you give us a sense of what happens if you get the green light on May 1?

偉大的。非常感謝。我很抱歉在員工人數方面做出了艱難的決定。但我知道對 omidubicel 的關注會有所回報。我很高興聽到檢查的消息。你能告訴我們如果你在 5 月 1 日獲得批准會發生什麼嗎？

How quickly do you anticipate starting to convert over these 10 to 15 initial centers? Are they ready to go? Have they given any insight into how they anticipate onboarding and utilizing omidubicel? Thanks so much.

您預計開始轉換這 10 到 15 個初始中心的速度有多快？他們準備好了嗎？他們是否對他們如何預期入職和使用 omidubicel 給出了任何見解？非常感謝。

Hi. So good morning. It's Michelle. So I just want to be -- I just want to make sure I heard you well. The question was converting centers over upon FDA approval.

你好。早上好。是米歇爾。所以我只想——我只是想確保我聽到你的聲音。問題是在 FDA 批准後轉換中心。

No. So I know you said that you have the initial 10 to 15 that you'll be focused on, the first 10 or 15 centers. I'm wondering what this launch, in the early days, could look like in terms of -- have they stated how they anticipate incorporating omidubicel into their practice?

不，所以我知道你說過你有最初的 10 到 15 個你會關注的，前 10 或 15 個中心。我想知道在早期，這次發布會是什麼樣子——他們是否說明了他們預期如何將 omidubicel 納入他們的實踐？

Yeah. No, thank you. I appreciate that.

是的。不，謝謝。我很感激。

So let me -- I'll answer your question. I do want to thank you for recognizing the pre-licensing inspection. We were excited by that also. Certainly, a lot of focus has gone into building that facility and getting that facility ready for BLA acceptance and also getting ready for the inspection. So thank you for recognizing that.

所以讓我——我會回答你的問題。我確實要感謝您對許可前檢查的認可。我們也為此感到興奮。當然，很多重點都放在建設該設施上，讓該設施為 BLA 驗收做好準備，並為檢查做好準備。所以謝謝你認識到這一點。

In regards to the centers -- so based on our initial dialogue with centers, as I indicated, we anticipate approximately 10 to 15 centers onboarded by the end of 2023. The feedback from the centers has been consistent with the market insight studies.

關於中心——正如我所指出的，根據我們與中心的初步對話，我們預計到 2023 年底將有大約 10 到 15 個中心上線。這些中心的反饋與市場洞察研究一致。

When our medical team asked the centers for unmet needs, it's focused on those two key categories. It's those patients who are currently eligible for transplant and cannot find an appropriate donor and then also the opportunity to use omidubicel in lieu of current donor sources based on our clinical outcomes, our 30-day turnaround time that we have demonstrated both in our Phase 3 study but also in our EAP.

當我們的醫療團隊向中心詢問未滿足的需求時，他們主要關注這兩個關鍵類別。這是那些目前有資格接受移植但找不到合適的供體的患者，然後還有機會根據我們的臨床結果使用 omidubicel 代替當前的供體來源，我們在第 3 階段中證明了我們的 30 天周轉時間學習，但也在我們的 EAP 中。

In partnership with the discussions with the centers, we've also talked extensively to US payers, both on the government side and on the commercial side. We're very encouraged by that feedback. And we've made sure that as we're meeting with those 10 to 15 centers that we anticipate being onboarded this year, we also understand their payer mix and make sure we're proactively engaging with those payers too.

在與這些中心進行討論的過程中，我們還與政府和商業方面的美國付款人進行了廣泛的交談。我們對這些反饋感到非常鼓舞。我們已經確保，當我們與預計今年加入的 10 到 15 個中心會面時，我們也了解他們的付款人組合，並確保我們也積極與這些付款人接觸。

Great. That's helpful. Well, fingers crossed and looking forward to the launch and talking soon about that.

偉大的。這很有幫助。好吧，祈禱吧，期待發射並很快談論它。

Perfect. Thank you, Ted.

完美的。謝謝你，泰德。

Jon Miller, Evercore ISI.

喬恩·米勒，Evercore ISI。

Hey, guys. Thanks so much for taking my question. And again, I'm also sorry for the tough decisions that you've had to make, but looking forward to the potential approval. I'd love to ask more about the commercial partnership. So do you expect that the PDUFA or the approval is a gating factor on getting a commercial partnership ready to go?

大家好。非常感謝您提出我的問題。再次，對於您必須做出的艱難決定，我也感到抱歉，但期待潛在的批准。我想問更多關於商業夥伴關係的問題。那麼，您是否認為 PDUFA 或批准是準備好商業合作夥伴關係的一個門控因素？

And then within that, if you're ready for launch and you've got the manufacturing all set up and you've got your initial centers set up, what exactly are you looking for in a potential partner out besides cash, obviously? And what do you expect the role of the partner to be in a potential launch?

然後在其中，如果你準備好發布並且你已經建立了所有製造並且你已經建立了你的初始中心，那麼除了現金之外，你究竟在尋找潛在的合作夥伴是什麼？您希望合作夥伴在潛在發布中扮演什麼角色？

Sure. Jon, I'll start. And then Michelle, you can chime in. So I would say we -- I wouldn't call the PDUFA date a gating item. I think the fact that omidubicel is quite a meaningful therapy and as we're nearing our PDUFA date, we've been noticed, and we -- that certainly generated some interest.

當然。喬恩，我要開始了。然後米歇爾，你可以插話。所以我想說我們 - 我不會將 PDUFA 日期稱為門控項目。我認為 omidubicel 是一種非常有意義的療法，而且隨著我們接近 PDUFA 日期，我們已經受到關注，而且我們——這當然引起了一些興趣。

So we are in the early stages of having some conversations, but I don't -- I think obviously, it will be an important milestone in the process, but not the gating matter. Michele, over to you for the second part of the question.

所以我們正處於進行一些對話的早期階段，但我沒有 - 我認為顯然，這將是該過程中的一個重要里程碑，但不是門控問題。米歇爾，請你回答問題的第二部分。

Yeah, thank you, and good morning, John. Thank you for the question. So in regards to what we're looking for in a partner, as you alluded to, the additional support from the capital side is certainly critical. The other area is infrastructure. There's -- although we have a clear launch strategy and we've begun our launch execution, there are still aspects of our infrastructure we haven't built out yet due to resource constraints. So that's something that we would look from a potential partner.

是的，謝謝你，早上好，約翰。感謝你的提問。所以關於我們在尋找合作夥伴方面，正如你提到的，來自資本方面的額外支持當然是至關重要的。另一個領域是基礎設施。有——儘管我們有一個明確的發布戰略並且我們已經開始執行我們的發布，但由於資源限制，我們的基礎設施的某些方面仍然沒有構建。所以這就是我們從潛在合作夥伴那裡看到的東西。

What I could say, John, is the team we have hired so far for the launch execution, both in regards to the commercial individuals, the medical individuals, and manufacturing, are outstanding individuals, incredibly experienced. It's just a matter of adding additional infrastructure to support them and then adding additional resources to be able to maximize the full launch.

約翰，我能說的是，到目前為止，我們為啟動執行聘請的團隊，無論是在商業人員、醫療人員還是製造方面，都是傑出的人員，經驗豐富。只需要添加額外的基礎設施來支持它們，然後添加額外的資源就能夠最大限度地全面啟動。

And one thing I would add, John, is that the BD efforts are in parallel to our ongoing fundraising activities. While this expense reduction is extending us through the third quarter, we know that it will be important to explore all sources, BD being one of them as fundraising opportunities. Because our number one priority is to make sure we can get this product to as many transplant centers and as many patients as needed as quickly as possible.

約翰，我要補充的一件事是，BD 的努力與我們正在進行的籌款活動是並行的。雖然這種費用減少將我們延長到第三季度，但我們知道探索所有來源很重要，BD 是其中之一，作為籌款機會。因為我們的首要任務是確保我們能夠盡快將此產品送到盡可能多的移植中心和盡可能多的患者。

Thanks. That makes sense. One final one, then, for me. Obviously, you say you're continuing on with the GDA-201 Phase 1. Can you characterize what the ongoing spend is for that program specifically and when the next potential for data release from that is?

謝謝。這就說得通了。最後一個，那麼，對我來說。顯然，您說您正在繼續 GDA-201 第 1 階段。您能否具體描述該計劃的持續支出是多少，以及下一次發布數據的可能性是什麼時候？

Hey, Jon. So I'll -- This is Shai; I'll address your first question. So we do not comment on specific, I would say, plan for GDA or omi. I can tell you, the vast majority, as we mentioned in our prepared remarks, goes to omidubicel. And as for the NK, specific, GDA-201, our cash guidance, including taking this program all the way through the end of Phase 1 of the first cohort, that it will happen probably this year or early next year.

嘿，喬恩。所以我會——我是 Shai；我會回答你的第一個問題。所以我們不評論具體的，我會說，GDA 或 omi 的計劃。我可以告訴你，正如我們在準備好的評論中提到的，絕大多數都使用了 omidubicel。至於 NK，具體來說，GDA-201，我們的現金指導，包括將這個項目一直進行到第一批第一階段結束，它可能會在今年或明年初發生。

And I'll chime in, Jon. This is Ronit. So this is the Phase 1 dose escalation study. And the study is designed to enroll patients that are separated by the DLT observation period of 28 days. So it's a pretty slow pace of enrollment, and the costs are estimated to be minimal compared to the other things that we need to do.

我會插話，喬恩。這是羅尼特。所以這是第一階段劑量遞增研究。該研究旨在招募被 28 天 DLT 觀察期分開的患者。所以這是一個相當緩慢的註冊速度，與我們需要做的其他事情相比，成本估計是最低的。

And so priority for us was continuing to treat those patients and understanding more about the safety and efficacy of GDA-201 in that patient cohort. So we will, based on the design of the study, have some information probably at the end of the year or very beginning of next year that we can share.

因此，我們的首要任務是繼續治療這些患者，並更多地了解 GDA-201 在該患者隊列中的安全性和有效性。因此，根據研究的設計，我們可能會在今年年底或明年年初獲得一些我們可以分享的信息。

Thanks so much.

非常感謝。

Thank you. (Operator Instructions) Mark Breidenbach, Oppenheimer.

謝謝。 （操作員說明）Mark Breidenbach，Oppenheimer。

Hey. Good morning, guys. Thanks for taking the questions. Just a couple of really quick ones from me. First of all, I'm wondering how large of a commercial team and field force do you think you need to cover the 10 to 15 sites that you'll be targeting in 2023? And are all of those personnel already onboarded at this point?

嘿。早上好傢伙。感謝您提出問題。我只是幾個非常快速的。首先，我想知道您認為您需要多少規模的商業團隊和現場工作人員才能覆蓋您將在 2023 年定位的 10 到 15 個站點？此時所有這些人員都已經入職了嗎？

And then the second question is just on the late-cycle meeting that you recently had with the FDA. I was hoping maybe you could just comment on key learnings or takeaways from that interaction. Thanks again for taking the questions.

然後第二個問題是關於你最近與 FDA 舉行的後期會議。我希望也許你可以評論一下從那次互動中學到的關鍵知識或收穫。再次感謝您提出問題。

Excellent. Thank you, Mark. I'll start with the personnel. And then I'll turn to Ronit for late-cycle meeting. So in regard to personnel, I'll talk about a few key categories. First off, we do have our full-market account -- I'm sorry, our full-market access team needed for launch in place. These were very critical hires that have been working closely with the payers in addition to the operations teams in the transplant centers.

出色的。謝謝你，馬克。我將從人員開始。然後我將轉向 Ronit 進行後期會議。那麼在人員方面，我會講幾個關鍵的類別。首先，我們確實有我們的全市場賬戶——對不起，我們的全市場准入團隊需要到位才能啟動。這些都是非常重要的僱員，除了移植中心的運營團隊外，他們還與付款人密切合作。

We've also -- we have our head of marketing and account management onboard; and she's done an outstanding job in addition to our initial regional account directors. So that will be the team on the commercial side, working to onboard the 10 to 15 of transplant centers.

我們還有——我們有營銷和客戶管理負責人；除了我們最初的區域客戶總監之外，她還完成了出色的工作。因此，這將是商業方面的團隊，致力於加入 10 到 15 個移植中心。

In addition, Ronit has hired her medical affairs and MSL lead. And they've hired some MSLs to -- so our intention, as stated earlier, is to ramp up over time, being mindful of the cash runway. And we'll look to add more personnel as resources increase. The ultimate goal, as we've guided in the past, is to get to approximately 24 commercial account managers and 12 MSLs.

此外，Ronit 還聘請了她的醫療事務和 MSL 負責人。他們已經聘請了一些 MSLs 來——所以我們的意圖，如前所述，是隨著時間的推移而增加，同時注意現金跑道。隨著資源的增加，我們將尋求增加更多人員。正如我們過去指導的那樣，最終目標是獲得大約 24 名商業客戶經理和 12 名 MSL。

I'll just touch briefly -- not field focus, but our operations team at our facility in Israel is in place, as is our quality teams. So that's staffed appropriately for launch.

我只是簡單地談一談——不是現場重點，但我們在以色列工廠的運營團隊已經到位，我們的質量團隊也是如此。因此，為啟動配備了適當的人員。

I can take the late-cycle meeting question, Mark. This is Ronit. So as you've heard before, our interactions with FDA have been continuous since the acceptance of the BLA. And we've had dialogue with them that's been quite productive all throughout.

我可以回答後期會議的問題，馬克。這是羅尼特。因此，正如您之前聽說的那樣，自 BLA 被接受以來，我們與 FDA 的互動一直在持續。我們與他們進行了自始至終富有成效的對話。

The late-cycle meeting itself which took place during the first quarter was extremely productive, and there were no surprises there. We continue to understand that they will not -- there are no safety or efficacy issues that are raised at this point that we're aware of and we're feeling quite positive moving forward towards our PDUFA date.

在第一季度舉行的後期會議本身非常富有成效，沒有任何意外。我們繼續了解到他們不會——目前沒有我們意識到的安全或有效性問題，我們對 PDUFA 日期的進展感到非常積極。

Okay. Thanks so much.

好的。非常感謝。

Gil Blum, Needham & Company.

Gil Blum, Needham & Company。

Hi. This is Rohit on for Phil. Thanks for taking our questions. In terms of the pipeline, do you still plan to continue the development of 301, 501, and 601 down the line when the financing is secured? Thanks,

你好。這是菲爾的羅希特。感謝您回答我們的問題。管線方面，融資到位後，是否還打算繼續下線301、501、601的開發？謝謝，

This is Ronit. Was it not Gil? I didn't hear.

這是羅尼特。不是吉爾嗎？我沒聽見。

Roman (sic - Rohit) (multiple speakers)

羅馬語（原文如此 - Rohit）（多個揚聲器）

Oh, Rohit. Thank you. So in terms of the NK cell therapy pipeline, we very much believe in the potential of our NK pipeline and its differentiation from other NKs. And already, have shown some quite interesting in vitro and in vivo data that shows the potential of these cells to have rapid and intense cytotoxic activity. So overall, it's very promising.

哦，羅希特。謝謝。因此，就 NK 細胞治療管道而言，我們非常相信我們的 NK 管道的潛力及其與其他 NK 的區別。並且已經顯示了一些非常有趣的體外和體內數據，這些數據顯示了這些細胞具有快速和強烈的細胞毒活性的潛力。所以總的來說，這是非常有前途的。

However, at this time, we're not in an economic position to continue to advance that pipeline. We will maintain the IP to those assets, and we'll certainly consider financial solutions to allow the development of those assets at some point.

但是，目前，我們沒有經濟能力繼續推進該管道。我們將維護這些資產的知識產權，我們當然會考慮財務解決方案以允許在某個時候開發這些資產。

Thank you.

謝謝。

Jason Butler, JMP Securities.

Jason Butler，JMP 證券。

Hi. Thanks for taking the questions. Can you give us any more color on how you selected the target 10 to 15 institutions in terms of willingness to be an early adopter and potential reimbursement access versus the number of potential patients at those centers? And then second to that, can you just talk to us about how you think about expanding beyond that initial 10 to 15 and when that could happen? Thanks.

你好。感謝您提出問題。您能否根據成為早期採用者的意願和潛在的報銷渠道以及這些中心的潛在患者數量，進一步說明您是如何選擇 10 到 15 家目標機構的？其次，您能否與我們談談您如何考慮將最初的 10 人擴展到 15 人以及何時可以實現？謝謝。

Yeah, no. Thank you, Jason. Thank you for the question. So in regards to the selection of the 10 to 15 -- so as I mentioned in my prepared remarks, these are centers that are within the top 70 centers. And those top 70 make up 80% of the transplant. These are centers that have expressed interest in being onboarded and have demonstrated the ability to be onboarded fairly soon after potential FDA approval.

是的，不。謝謝你，傑森。感謝你的提問。因此，關於 10 到 15 名的選擇——正如我在準備好的發言中提到的，這些是排名前 70 名的中心。前 70 名佔移植的 80%。這些中心已表示有興趣加入，並已證明有能力在獲得 FDA 潛在批准後很快加入。

These are centers -- some of them were part of our clinical study, some were not. And we're very encouraged by the feedback from those centers. The centers were comfortable with the payer mix. And those centers have already met with the payers that would be working closely with those centers. So those were some of the aspects that led us to target those 10 to 15 centers.

這些是中心——其中一些是我們臨床研究的一部分，一些不是。這些中心的反饋讓我們深受鼓舞。這些中心對付款人組合感到滿意。這些中心已經與將與這些中心密切合作的付款人會面。所以這些是導致我們瞄準這 10 到 15 個中心的一些方面。

What I would say in regards to why it's 10 to 15, we want to be mindful of the resources that we currently have. And as additional resources come in, either capital or potential support from a partner, we would look then to increase the number of centers beyond that 10 to 15.

關於為什麼是 10 到 15，我想說的是，我們要注意我們目前擁有的資源。隨著額外資源的加入，無論是資金還是合作夥伴的潛在支持，我們都會考慮將中心數量增加到 10 個以上，增加到 15 個。

We've had a very engaging dialogue with a majority of the top centers throughout the US, and we're encouraged by the feedback. So as I mentioned, we'll start with the 10 to 15 as our target for this year, and then look to increase that as additional resources come in.

我們與美國大多數頂級中心進行了非常有吸引力的對話，我們對反饋感到鼓舞。因此，正如我提到的，我們將從 10 到 15 個作為我們今年的目標，然後隨著額外資源的進入而增加這個目標。

Thank you.

謝謝。

Vernon Bernadino, H.C. Wainright.

弗農貝納迪諾，H.C.溫賴特。

Hi, everyone. Thanks for taking my question. And I'm sorry to hear about the difficult decisions, but congrats on the progress you've made. It's been great so far. So definitely looking forward to the launch. Most of my questions have already been answered. But one question I was wondering is, with the headcount reduction of 17%, does that includes some of the hires you had made so far? Or is that basically across the board and core decision regarding a straight headcount reduction in the firm?

大家好。感謝您提出我的問題。我很遺憾聽到艱難的決定，但祝賀你取得的進步。到目前為止一切都很好。所以絕對期待發布。我的大部分問題已經得到解答。但我想知道的一個問題是，由於裁員 17%，這是否包括您迄今為止招聘的一些員工？或者這基本上是關於公司直接裁員的全面和核心決策？

Sure. Thanks, Vernon. The 17% reduction in headcount is mainly related to employees who are working on the NK pipeline -- the early NK pipeline. So with that discontinuation, we're ramping down on the employees who were working on those programs, primarily.

當然。謝謝，弗農。裁員 17% 主要與從事 NK 管道工作的員工有關——早期的 NK 管道。因此，隨著這種停產，我們主要減少了從事這些項目的員工。

Okay. And can you describe how much of the closed down operations in Jerusalem will have an effect?

好的。你能描述一下在耶路撒冷關閉的業務有多少會產生影響嗎？

Hi, Vernon. This is Shai.

嗨，弗農。這是夏。

Hi, Shai.

嗨，夏伊。

So as we mentioned in our prepared remarks, through this strategic, I'd say, change, we needed cash to the middle of the year. And with those changes, we extend our cash runway by another -- which, the way we see it, this is one element of the restructuring. The other one, I do believe that the make -- the changes we are making today will make Gamida Cell more focused, prioritized, and attractive investment opportunity with a very clear near-term value creation point.

因此，正如我們在準備好的發言中提到的那樣，通過這一戰略，我想說，改變，我們需要現金到年中。通過這些變化，我們將現金跑道延長了另一條——在我們看來，這是重組的一個要素。另一個，我確實相信，我們今天所做的改變將使 Gamida Cell 更加專注、優先和有吸引力的投資機會，具有非常明確的近期價值創造點。

In terms of your specific one on the Jerusalem side, quote, unquote, again, we do not comment on very specific, I would say, element of the business plan. But the vast majority of our spending will go to omidubicel, whereas the Jerusalem side is more dedicated to the R&D.

就您在耶路撒冷方面的具體內容而言，無論是引用還是取消引用，我們都不會對業務計劃中非常具體的元素發表評論。但我們的絕大部分支出將用於 omidubicel，而耶路撒冷方面則更專注於研發。

And can you remind us again on how much of a grant from the Israeli government you get for operations in Israel?

您能否再次提醒我們您從以色列政府那裡獲得了多少用於在以色列開展業務的贈款？

So we are keeping the operation in Israel. We do have two sites in Israel. One site is Kiryat Gat, which is the lead manufacturing site. And this is the main, I would say, purpose of all our refund that we received from the Israeli Innovation Authority. So there is no issue in terms of grants from the Israeli business. We continue with the R&D specifically to omidubicel and the manufacturing and all the IP related. Overall today, it's approximately $50 million we received in the last 20 years.

因此，我們將繼續在以色列開展業務。我們在以色列有兩個站點。一個地點是 Kiryat Gat，這是主要的製造地點。我想說，這是我們從以色列創新局收到的所有退款的主要目的。因此，在以色列企業的贈款方面沒有問題。我們繼續專門針對 omidubicel 和製造以及所有相關 IP 的研發。總的來說，我們在過去 20 年收到的收入約為 5000 萬美元。

Thank you very much. That information is very helpful. And I'm looking forward to launch. And best of luck.

非常感謝。這些信息非常有幫助。我期待著發布。祝你好運。

Thanks, Vernon.

謝謝，弗農。

Thank you. And I'm showing no further questions in the queue. I'd like to turn it back to Abbey Jenkins for closing remarks.

謝謝。我不會在隊列中顯示更多問題。我想把它轉回 Abbey Jenkins 作結束語。

Thank you, Catherine. I want to reiterate that the changes we've announced today will make Gamida Cell a more focused, prioritized, and attractive investment opportunity and create a path for near-term value creation with the potential approval and launch of omidubicel. And to be clear, we do plan to launch immediately following approval if granted on May 1.

謝謝你，凱瑟琳。我想重申，我們今天宣布的變化將使 Gamida Cell 成為一個更加專注、優先和有吸引力的投資機會，並通過可能批准和推出 omidubicel 為近期價值創造創造途徑。需要明確的是，如果 5 月 1 日獲得批准，我們確實計劃在獲得批准後立即啟動。

Our leadership team will be available after the call if there are any opportunities for follow-up discussions. We'll keep you current on all of our developments. And we thank you again for your interest and support of Gamida Cell. Thank you, everyone, for joining us on today's call.

如果有機會進行後續討論，我們的領導團隊將在電話會議後提供服務。我們會讓您了解我們所有的最新進展。我們再次感謝您對 Gamida Cell 的關注和支持。謝謝大家參加今天的電話會議。

This concludes today's conference call. Thank you for participating. You may now disconnect.

今天的電話會議到此結束。感謝您的參與。您現在可以斷開連接。