bluebird bio Inc (BLUE) 2024 Q3 法說會逐字稿

Good morning, ladies and gentlemen. Thank you for standing by. Welcome to bluebird bio's third quarter 2024 results conference call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. (Operator Instructions) Please note that today's conference is being recorded.

女士們、先生們，早安。感謝您的支持。歡迎參加 Bluebird Bio 2024 年第三季業績電話會議。此時，所有參與者都處於只聽模式。演講者演講結束後，將有問答環節。（操作員指示）請注意，今天的會議正在錄音。

I'll now hand the conference over to your host, Courtney O'Leary, Director of Investor Relations. Please go ahead.

現在我將會議交給主持人、投資者關係總監 Courtney O'Leary。請繼續。

Good morning, everyone, and thank you for joining our third quarter 2024 results call today. My name is Courtney O'Leary, Director of Investor Relations at bluebird bio.

大家早安，感謝您今天參加我們 2024 年第三季業績電話會議。我叫 Courtney O'Leary，是 Bluebird Bio 的投資人關係總監。

Before we begin, let me review our safe harbor statement. Today's discussion contains statements that are forward-looking under the Private Securities Litigation Reform Act of 1995, including expectations regarding our future financial results and financial position in addition to statements of the company's plans, expectations or intentions regarding regulatory progress, commercialization plans and business operations.

在我們開始之前，讓我先回顧一下我們的安全港聲明。今天的討論包含根據 1995 年《私人證券訴訟改革法》做出的前瞻性陳述，包括對我們未來財務業績和財務狀況的預期，以及有關監管進展、商業化計劃和業務運營的公司計劃、預期或意圖的陳述。

Such statements are based on current expectations and assumptions that are subject to risks and uncertainties and involve a number of risk factors that could cause actual results to differ materially from projected results. A description of these risks is contained in our filings with the SEC, which are available on the Investor Relations section of our website, www.bluebirdbio.com.

此類聲明基於目前的預期和假設，受風險和不確定性的影響，並涉及多種可能導致實際結果與預測結果有重大差異的風險因素。這些風險的描述包含在我們向美國證券交易委員會提交的文件中，可在我們網站 www.bluebirdbio.com 的投資者關係部分查閱。

With that, I will turn it over to bluebird bio's CEO, Andrew Obenshain.

說完這些，我將把麥克風交給 bluebird bio 的執行長 Andrew Obenshain。

Thank you, Courtney, and thank you everyone for joining our call this morning. As a gene therapy pioneer and the only standalone commercial-stage gene therapy company in the market today, we have built what we believe to be an unparalleled foundation over the past two years since our first FDA approval with a robust qualified treatment center network, proven access and reimbursement and demonstrated demand for our therapies from both patients and from providers. Today, we are seeing the results of those efforts.

謝謝你，考特尼，也謝謝大家今天早上參加我們的電話會議。作為基因治療的先驅和當今市場上唯一一家獨立的商業階段基因治療公司，自首次獲得 FDA 批准以來的兩年間，我們建立了自認為無與倫比的基礎，包括強大的合格治療中心網絡、經過驗證的可及性和報銷，以及患者和提供者對我們療法的明顯需求。今天，我們看到了這些努力的成果。

Since we reported our Q2 earnings in August, we have more than doubled patient starts from 27 to 57 across our portfolio. We initiated steps to reduce our cash operating expenses by 20% in Q3 2025, and we've laid out a roadmap to financial stability and cash flow breakeven in the second half of next year should we accomplish our goal of securing additional cash resources.

自從我們八月報告第二季度收益以來，我們整個投資組合中的患者數量增加了一倍多，從 27 人增加到 57 人。我們已採取措施，在 2025 年第三季將現金營運支出減少 20%，並且我們已經制定了在明年下半年實現財務穩定和現金流收支平衡的路線圖，以完成獲得額外現金資源的目標。

On today's call, Tom Klima, Chief Commercial and Operating Officer, will provide updates on the commercial launches of LYFGENIA, ZYNTEGLO and SKYSONA. Then, bluebird's CFO, James Sterling, will provide an update and some comments on our recent proxy vote before opening the call for Q&A.

在今天的電話會議上，首席商務和營運長 Tom Klima 將提供有關 LYFGENIA、ZYNTEGLO 和 SKYSONA 商業發布的最新情況。然後，bluebird 的財務長 James Sterling 將在開始問答環節之前，就我們最近的代理投票提供最新情況並發表一些評論。

I will now hand the call over to Tom.

我現在將電話交給湯姆。

Thanks, Andrew, and good morning, everyone. We continue to see strong growth in our commercial launches in the third quarter with clear and established paths to access for our therapies and an industry-leading QTC network. As Andrew noted, we more than doubled patient starts since our Q2 call to 57 from 27 in mid-August. Today, we announced that 74 patient starts have been completed or scheduled to date in 2024 across our portfolio.

謝謝，安德魯，大家早安。我們的商業發佈在第三季度繼續保持強勁增長，我們的治療方法擁有清晰而成熟的獲取途徑，並且擁有行業領先的 QTC 網路。正如安德魯所指出的，自第二季以來，我們接診的患者數量增加了一倍多，從 8 月中旬的 27 名增加到 57 名。今天，我們宣布，截至 2024 年，我們投資組合中已有 74 名患者完成或計劃開始接受治療。

Of that 74, 57 patient starts have been completed with an additional 17 starts scheduled through the rest of the year. And we continue to see strong and sustained demand into the future with 30 patient starts already scheduled in 2025. This trajectory continues to support the potential path to cash flow breakeven in the second half of 2025 as those starts convert to deliveries and infusions.

在這 74 例患者中，已經完成 57 例手術，另有 17 例手術計劃在今年剩餘時間內開始。我們將繼續看到未來強勁而持續的需求，2025 年已經安排了 30 名患者開始接受治療。隨著這些開工轉化為交付和注入，這一軌跡將繼續支持 2025 年下半年實現現金流收支平衡的潛在途徑。

Remember, as we have said, it takes approximately two quarters between a patient start and drug product delivery. We are particularly encouraged that because of our extensive QTC network, patients have broad access to our therapies.

請記住，正如我們所說的，從患者開始治療到藥品交付大約需要兩個季度。我們特別感到鼓舞的是，由於我們擁有廣泛的 QTC 網絡，患者可以廣泛地接受我們的治療。

To date, patients have initiated or enrolled for treatment across more than 30 unique QTCs. You will recall that bluebird has more than 70 total QTCs offering LYFGENIA and ZYNTEGLO in the US, highlighting the significant upside potential to come as the remaining centers in our network start their first patient. We have the manufacturing capacity we need today to achieve our cash flow breakeven point, and we have plans to double capacity for LYFGENIA in 2026 based on anticipated demand. This follows a similar launch dynamic as ZYNTEGLO where we scaled capacity commensurate with demand.

迄今為止，患者已在 30 多個獨特的 QTC 中發起或報名接受治療。您會記得，bluebird 在美國共有超過 70 個 QTC 提供 LYFGENIA 和 ZYNTEGLO，這凸顯了隨著我們網路中剩餘的中心開始接收第一位患者，未來的巨大上行潛力。我們目前擁有實現現金流量損益平衡點所需的製造能力，並且我們計劃根據預期需求在 2026 年將 LYFGENIA 的產能翻倍。這與 ZYNTEGLO 的發布動態類似，我們根據需求擴大容量。

Now, moving to access and reimbursement. Our goal has always been timely, equitable access to our therapies. We are extremely encouraged by the speed with which both commercial and many government payers are approving pathways to patient access.

現在，轉向訪問和報銷。我們的目標一直是及時、公平地獲得我們的治療。我們對商業和許多政府付款人批准患者使用途徑的速度感到非常鼓舞。

Focusing on LYFGENIA, more than half of all states have affirmed coverage through a preferred drug list or published coverage criteria. Additionally, nearly 50% of Medicaid-insured individuals with sickle cell disease in the US live in a state that has already completed prior authorization approval for the use of LYFGENIA for at least one patient.

重點關注 LYFGENIA，超過一半的州已通過首選藥物清單或公佈的承保標準確認了承保範圍。此外，美國近 50% 的享有醫療補助的鐮狀細胞疾病患者所居住的州已獲得對至少一名患者使用 LYFGENIA 的預先授權批准。

Commercially, multiple outcomes-based agreements have been published and/or in place for LYFGENIA, representing more than 200 million US lives. And timely access to LYFGENIA, ZYNTEGLO and SKYSONA has continued with zero ultimate denials to date for any of our therapies across both Medicaid and commercial.

從商業角度來看，LYFGENIA 已發布和/或實施了多項基於成果的協議，涉及超過 2 億美國人的生命。並且，人們繼續及時獲得 LYFGENIA、ZYNTEGLO 和 SKYSONA 的治療，迄今為止，我們在醫療補助和商業方面的所有療法均未遭到最終拒絕。

Now, I'd like to turn the call over to James to speak to our financials.

現在，我想讓詹姆斯談談我們的財務狀況。

Thank you, Tom, and good morning, everyone. I'm pleased this morning to confirm that we are now back on track with normal reporting timelines following the restatement of our financials earlier this year.

謝謝你，湯姆，大家早安。今天早上我很高興地確認，繼今年稍早重新申報財務狀況後，我們現在已經恢復正常的申報時間表。

In the third quarter, we reported $10.6 million in total revenue, down from $16.1 million in Q2. As we guided on our last earnings call, we anticipated this dip in Q3 revenues due to variation in manufacturing timelines. We expect revenue will rebound nicely in the fourth quarter with net revenue of at least $25 million as more patients are infused.

第三季度，我們報告的總收入為 1,060 萬美元，低於第二季的 1,610 萬美元。正如我們在上次收益電話會議上所指出的那樣，我們預計第三季收入將因製造時間表的變化而下降。我們預計，隨著更多患者接受治療，第四季收入將強勁反彈，淨收入將達到至少 2,500 萬美元。

Importantly, in the third quarter, we recognized our first LYFGENIA revenue, following the completion of the first LYFGENIA infusion well within our target timeline. As previously guided, in 2024, we continue to anticipate gross to net discounts in the range of 20% to 25% with fluctuations based on product and payer mix as well as utilization of our outcomes-based agreements.

重要的是，在第三季度，我們實現了第一筆 LYFGENIA 收入，因為我們在目標時間內完成了第一次 LYFGENIA 注入。根據先前的指導，到 2024 年，我們將繼續預計總折扣與淨折扣將在 20% 至 25% 之間，波動取決於產品和付款人組合以及基於成果的協議的利用情況。

On the cost side, SG&A expense was generally consistent with the prior-year period, while R&D expense was down $36.1 million year-over-year as previous R&D expenses were shifted to inventory and cost of products revenue.

成本方面，銷售、一般及行政開支與去年同期基本持平，而研發開支同比下降 3,610 萬美元，因為先前的研發開支轉移到庫存和產品成本收入。

As of September 30, 2024, we had $118.7 million of cash on hand, which is inclusive of $48 million in restricted cash. As we announced on our call in late September, we've implemented significant changes to optimize our cost structure.

截至 2024 年 9 月 30 日，我們持有 1.187 億美元現金，其中包括 4,800 萬美元的受限現金。正如我們在九月下旬的電話會議上所宣布的那樣，我們已經實施了重大變革以優化我們的成本結構。

These changes are anticipated to result in a 20% reduction in cash operating expenses when fully realized in Q3 of 2025. And we continue to anticipate quarterly cash flow breakeven in the second half of 2025, assuming we scale to approximately 40 drug product deliveries per quarter and obtain additional cash resources to extend our runway.

預計這些變化將在 2025 年第三季全面實現，導致現金營運費用減少 20%。我們繼續預計 2025 年下半年季度現金流將實現收支平衡，假設我們每季的藥品交付規模擴大到約 40 種，並獲得額外的現金資源來延長我們的跑道。

As Tom noted, we already have 30 patients scheduled for cell collection in 2025, and we feel confident that we remain on pace with previous guidance of approximately 40 deliveries per quarter in the second half of next year.

正如湯姆所說，我們已經安排了 30 名患者在 2025 年進行細胞採集，並且我們有信心在明年下半年繼續保持之前的指導速度，即每季度進行約 40 次採集。

We are engaging collaboratively with Hercules as we work to secure adequate cash runway to obtain additional financing and reach cash flow breakeven in the second half of next year. Based on our current forecasts, which assumes continued cost saving initiatives, successfully renegotiating key contracts and continued collaborative engagement from Hercules, we expect our existing cash and cash equivalents will enable us to fund our operations into the first quarter of 2025.

我們正在與 Hercules 合作，努力確保有足夠的現金流來獲得額外融資，並在明年下半年實現現金流收支平衡。根據我們目前的預測，假設繼續採取成本節約舉措、成功重新談判關鍵合約以及繼續與 Hercules 合作，我們預計現有的現金和現金等價物將使我們能夠為到 2025 年第一季的營運提供資金。

I wanted to provide a bit of color on our proxy vote last week. As you may have seen, we adjourned our meeting until December 4 to solicit additional votes to obtain approval of Proposal 4 related to the reverse stock split.

我想就我們上週的代理投票提供一些細節。正如您可能已經看到的，我們將會議休會至 12 月 4 日，以徵求更多投票，以獲得與反向股票分割相關的提案 4 的批准。

A vote in favor of Proposal 4 would enable us to regain compliance with Nasdaq's minimum bid price and increase the number of shares on a relative basis that we are authorized to issue, a necessary step to enable flexibility with our financing options. We are very pleased with the votes we've received so far and want to encourage every stockholder to vote in favor of Proposal 4. If any of our shareholders have questions about this or would like to discuss it further, please email us at investor@bluebirdbio.com.

支持提案 4 的投票將使我們能夠重新遵守納斯達克的最低買入價，並相對增加我們被授權發行的股票數量，這是實現我們融資選擇靈活性的必要步驟。我們對迄今為止收到的投票感到非常滿意，並希望鼓勵每位股東投票支持提案 4。如果我們的任何股東對此有疑問或希望進一步討論，請發送電子郵件至 investor@bluebirdbio.com。

With that, I will turn it back to Andrew.

說完這些，我就把話題轉回給安德魯。

Thanks, James. As we highlighted today, we are now in a period of accelerated growth, and we have visibility to cash flow breakeven in the second half of next year as we work to obtain additional sources of capital and execute on our launches across our validated commercial gene therapy platform. We look forward to continuing to update the investor community on our progress as we move towards changing first tens of hundreds of patients' lives.

謝謝，詹姆斯。正如我們今天所強調的那樣，我們目前正處於加速成長時期，隨著我們努力獲取額外的資金來源並執行我們經過驗證的商業基因治療平台的發布，我們可以在明年下半年實現現金流收支平衡。我們期待繼續向投資者社群通報我們在改變數萬名患者生活的過程中取得的進展。

With that, we'd like to open it up for questions. Operator?

現在，我們願意開始回答問題。操作員？

(Operator Instructions) Jack Allen, Baird.

（操作員指示）傑克艾倫，貝爾德。

Great. Thanks for taking the questions, and congrats to the team on the progress made over the quarter. I guess, my first one is on the revenues in the third quarter. It seems like there's an incremental stepdown as compared to revenues announced in the second quarter.

偉大的。感謝您回答這些問題，並祝賀團隊在本季的進展。我想，我的第一個問題是關於第三季的收入。與第二季公佈的收入相比，似乎出現了逐步下降的情況。

And I wanted to ask, what are you seeing as it relates to dynamics around administration of the cells after you deliver them to the treatment centers? Do you still see 100% pull-through in that regard, or are you seeing any kind of dropout from cell manufacturing to administration of cells? And then, I have a quick follow-up as well.

我想問一下，在將細胞送到治療中心後，您觀察到與細胞管理相關的動態是什麼？在這方面您是否仍然看到 100% 的拉動，或者您是否看到從細胞製造到細胞管理的任何形式的中斷？然後，我也會進行快速的跟進。

Yeah. Good morning, Jack. Thanks for the question. I'm going to hand it to Tom to respond.

是的。早上好，傑克。謝謝你的提問。我將把它交給湯姆來回應。

Hey, Jack. Good morning. Yeah, we still see 100% pull-through once we deliver cells to a patient being treated. As you know, there's just variability I think in terms of patient scheduling. And then, once we deliver the final drug product to the QTC, it's in the QTC's hand. So, it's a little bit out of our control once we deliver it back, but we -- once we deliver it back, we've seen 100% pull-through.

嘿，傑克。早安.是的，一旦我們將細胞輸送到接受治療的患者體內，我們仍然看到 100% 的成功率。如您所知，我認為患者安排方面存在差異。然後，一旦我們將最終的藥品交付給 QTC，它就在 QTC 手中。所以，一旦我們將其送回，情況就有點不受我們控制了，但是，一旦我們將其送回，我們就看到了 100% 的成功。

Got it. Great. And then, I guess, maybe I'll just dive in a little bit more. Are you seeing 100% manufacturing success rate? And then, my follow-up was also, on the 2025 scheduling, any color as it relates to how far out you're scheduling slots into 2025 of those 30 slots?

知道了。偉大的。然後，我想，也許我會深入探討。您看到 100% 的製造成功率嗎？然後，我的後續問題是，關於 2025 年的時間安排，是否有任何顏色與您在 30 個時段中將時段安排到 2025 年有多遠有關？

Yeah. Let me take the first one, and then I'll hand it -- the second one to Tom. So no, we do see -- recollections are a natural part of the process, right? So we do sometimes have to collect more than once for a patient. That is not a manufacturing failure. That is a normal part of the process. And we've -- and once a patient starts the process, nearly a 100% of those patients go on to an eventual delivery.

是的。讓我拿第一個，然後我將第二個交給湯姆。所以，我們確實看到了——回憶是這個過程的自然組成部分，對嗎？因此，有時我們確實需要為同一患者採集多次血液。這並不是製造失敗。這是過程的正常部分。一旦患者開始這個過程，幾乎 100％ 的患者都會最終分娩。

Now, Tom, go ahead.

現在，湯姆，繼續。

Yeah. Just to reiterate, although there's variability in terms of scheduling when the patients are being scheduled, we're seeing patients being scheduled one to two months out in many cases, and we're really pleased that we have now over 30 patients scheduled in 2025. But as Andrew mentioned, there's just some variability and recollection as part of the process, but we do not see patients drop out because of that.

是的。需要重申的是，儘管患者的預約時間存在差異，但我們發現許多患者的預約時間都提前了一到兩個月，我們很高興現在已有超過 30 名患者在 2025 年接受預約。但正如安德魯所提到的，整個過程會有一些變化和回憶，但我們並沒有看到患者因此而退出治療。

Gena Wang, Barclays.

巴克萊銀行的 Gena Wang。

Thank you for taking my questions. Maybe two. Regarding the 30 patients already scheduled in 2025, are these mainly in the first quarter? And then, for the LYFGENIA, cumulative -- the 17 patients that completed cell collection, what is the average cycle, cell collection cycle for these patients?

感謝您回答我的問題。或許是兩個。至於2025年已經預約的30名患者，這些主要是在第一季嗎？然後，對於 LYFGENIA，累計——完成細胞採集的 17 名患者，這些患者的平均週期、細胞採集週期是多少？

And quickly regarding the cash gap, you do have one-quarter, second quarter '25, cash gap. What could be the strategy in addition to Hercules you could cover this one-quarter cash gap?

就現金缺口而言，2025 年第一季和第二季確實存在現金缺口。除了大力士 (Hercules) 之外，還有什麼策略可以彌補這一季度的現金缺口？

Good morning, Gena. Thanks for the question. So I'll take the question about -- we'll take the question a little bit out of order. I'll answer the questions about the number of cycles for LYFGENIA. I'll hand it to Tom to talk about kind of how those patients are being scheduled out next year, and then go to James for the cash.

早安，吉娜。謝謝你的提問。因此我將回答這個問題——我們將稍微不按順序地回答這個問題。我將回答有關 LYFGENIA 的周期數的問題。我會把錢交給湯姆，讓他談談這些病人明年的安排，然後再去找詹姆斯要錢。

In terms of the number of cycles in the clinical trials for LYFGENIA, we said that 85% of patients either got done in about one or two cycles. We anticipate similar results for the commercial setting. The end is still relatively small, so I can't really comment on that yet.

就 LYFGENIA 臨床試驗的週期數而言，我們表示 85% 的患者在大約一到兩個週期內完成治療。我們預期商業環境中也會有類似的結果。結局仍然相對較小，所以我還無法對此發表評論。

But maybe Tom, if you could comment on whether those patients to be scheduled in first quarter?

但是湯姆，你能否評論一下這些病人是否會在第一季安排？

Yeah. Hey, Gena, good morning. We're really pleased with the sustained demand that we're seeing and we're really excited that 30 patients are already scheduled for next year. The vast majority of those are in Q1, although some are into Q2 as well. And we're really not commenting on the breakdown between LYFGENIA and ZYNTEGLO, but just an exciting marker of the demand that continues to exist.

是的。嘿，吉娜，早安。我們對持續的需求感到非常高興，我們也很高興明年已經有 30 位患者預約就診。其中絕大多數處於第一季度，但也有一些處於第二季度。我們實際上並不是在評論 LYFGENIA 和 ZYNTEGLO 之間的分歧，而只是評論持續存在的需求的一個令人興奮的標誌。

Right. And then, James, if you could comment on the cash gap and plans to fill that?

正確的。然後，詹姆斯，您能評論一下現金缺口以及填補計劃嗎？

Yeah. Hi, Gena. And you asked about Hercules, in particular. So, Hercules has been a great partner for us through this and we're in regular contact with them. It'd be premature for me to expand on specifics, nature of those conversations, but strategy to extend the runway include renegotiating key contracts and other cost initiatives, and our partnership with Hercules is key in helping move those forward.

是的。你好，吉娜。您特別問到了大力士 (Hercules)。所以，Hercules 一直是我們很好的合作夥伴，我們一直與他們保持聯繫。現在就詳細闡述這些對話的具體內容和性質還為時過早，但延長跑道的策略包括重新談判關鍵合約和其他成本舉措，而我們與 Hercules 的合作是推動這些舉措的關鍵。

Jason Gerberry, Bank of America.

美國銀行的傑森·格貝利（Jason Gerberry）。

On the scheduled new start disclosure, just wondering what's your internal assumption on conversion rate of that actually being -- [coming] a patient that gets their cells collected?

關於預定的新開始披露，我只是想知道您對實際轉化率的內部假設是什麼 - [即將到來的] 患者收集了他們的細胞？

And, I guess, the other question that I had was just with the new starts and the scheduled new starts for LYFGENIA specifically, are these concentrated in a few centers, or do you feel like the -- where the start activity is occurring is pretty broadly dispersed amongst centers so far?

而且，我想問的另一個問題是，關於 LYFGENIA 的新啟動和計劃中的新啟動，這些是否集中在幾個中心，或者您是否覺得——到目前為止，啟動活動發生的地方在各個中心之間分佈得相當廣泛？

Go ahead, Tom.

繼續吧，湯姆。

Yeah. Hey, good morning, Jason. In terms of a patient being scheduled and converting to a start, obviously, we don't want to get too excited about small ends, but so far it's very high. It's virtually -- it's essentially, 100% are converting to a start. The only variable is just the time. In some cases, they reschedule because of an event, but they 100% of the time have come back on the schedule, so that the conversion rate is high and we're pleased to see the excitement for gene therapy.

是的。嘿，早上好，傑森。對於病人的安排和轉為開始治療而言，顯然，我們不想對小結局太過興奮，但到目前為止，這個數字還很高。事實上 — — 本質上，100％ 都已轉變為開始。唯一的變數只是時間。在某些情況下，他們會因為某個事件而重新安排時間，但他們 100％ 的時間都會按照計劃重新開始，因此轉換率很高，我們很高興看到基因治療的興奮之情。

As far as -- and remind me the second part of the question?

至於──請提醒我問題的第二部分？

Is it broad based for LYFGENIA?

LYFGENIA 的基礎是否廣泛？

Yeah. So I think it's exciting because we have over 70 QTCs and we said approximately 30 QTCs have initiated for one of our therapies, which is pretty well spread out, but I think the most exciting part of that is we have about 40 QTCs that are looking to start their first patients. So, I think it's a nice indicator of growth ahead.

是的。所以我認為這是令人興奮的，因為我們有超過 70 個 QTC，我們說大約有 30 個 QTC 已經開始接受我們的一種療法，分佈相當廣泛，但我認為其中最令人興奮的部分是我們有大約 40 個 QTC 正在等待開始治療他們的第一批患者。所以，我認為這是未來成長的一個很好的指標。

Mani Foroohar, Leerink Partners.

Mani Foroohar，Leerink Partners。

Hi, good morning. This is CJ on for Mani. Thanks for taking my question. Mine is just regarding, you previously shared that time from cell collection to completion of manufacturing and testing was taking like a month longer than expected. Have you figured how to accelerate this process? If so, when you expect to fully realize this? Thank you.

嗨，早安。這是 CJ 為 Mani 表演的。感謝您回答我的問題。我只是擔心，您之前分享過，從細胞收集到完成製造和測試的時間比預期的要長一個月。您想知道如何加速這一進程嗎？如果是的話，您預計什麼時候能夠完全實現這一目標？謝謝。

Thanks for the question. I'm not sure that we've actually reported that before. We are seeing pretty consistent timelines or some variability. We'd say 70, 90 days for ZYNTEGLO, 90 to 105 days for LYFGENIA. There's a bell curve around that. So some are slower, some are faster. But in general, we've been very pleased with the quality of our manufacturing, the timeliness.

謝謝你的提問。我不確定我們之前是否真的報道過此事。我們看到了相當一致的時間表或一些變化。我們認為 ZYNTEGLO 的治療週期為 70 至 90 天，LYFGENIA 的治療週期為 90 至 105 天。那裡有一個鐘形曲線。所以有的慢，有的快。但總的來說，我們對我們的製造品質和及時性感到非常滿意。

I'll point out that for LYFGENIA, that first patient that we actually delivered, we actually delivered that within that -- slightly less than that timeframe for LYFGENIA. So in general, we're very pleased with our manufacturing.

我要指出的是，對於 LYFGENIA 來說，我們實際接生的第一位患者是在那個時間範圍內接生的——比 LYFGENIA 的時間範圍略短一些。總的來說，我們對我們的製造非常滿意。

Okay. Thank you. And if I could quickly add another one, like, to what degree is your manufacturing capacity limiting your ability to ramp patient starts and to achieve that 40 drug delivery per quarter that you're in -- by second half next year?

好的。謝謝。如果我可以快速添加另一個問題，例如，您的製造能力在多大程度上限制了您增加患者開始治療的能力，以及到明年下半年實現每季度 40 次藥物輸送的能力？

Yeah. So, our current capacity is adequate to get to the projections that we've been talking about and able to support us getting to 40 drug product deliveries per quarter, and ultimately, the cash flow breakeven that we've been talking about.

是的。因此，我們目前的產能足以實現我們一直在談論的預測，並能夠支持我們每季交付 40 種藥品，並最終實現我們一直在談論的現金流收支平衡。

We are working on expanding our capacity for LYFGENIA. We announced that we would double capacity in 2026 and that's based on our belief that the demand will continue to grow for LYFGENIA in sickle cell disease, but right now, we have adequate manufacturing capacity to hit our projections.

我們正在致力於擴大 LYFGENIA 的產能。我們宣布將在 2026 年將產能翻一番，這是基於我們對鐮狀細胞疾病對 LYFGENIA 的需求將持續增長的信念，但目前，我們有足夠的生產能力來達到我們的預測。

Salveen Richter, Goldman Sachs.

高盛的薩爾文·里希特（Salveen Richter）。

Hi. This is Lydia on for Salveen. Thanks so much for taking our question. I guess just going off the last one, can you speak to what drives your confidence in scaling to that 40 product deliveries per quarter by the second half of 2025? And how you expect this to evolve and ramp over the next few quarters? Thanks so much.

你好。這是 Salveen 的 Lydia。非常感謝您回答我們的問題。我想從最後一個問題出發，您能談談是什麼讓您有信心到 2025 年下半年將產品交付量擴大到每季 40 件嗎？您預計這一趨勢在接下來的幾季將如何發展和提升？非常感謝。

Go ahead, Tom.

繼續吧，湯姆。

Yeah, sure. Good morning, Lydia. It's really a couple of things. Number one, we continue to see strong and steady demand for ZYNTEGLO. We did expand our capacity this year for our manufacturing capacity at Lonza for ZYNTEGLO and SKYSONA, and we continue to see strong growth there and demand is not slowing down.

是的，當然。早安，莉迪亞。這確實是幾件事。首先，我們繼續看到對 ZYNTEGLO 的強勁而穩定的需求。我們今年確實擴大了 Lonza 的 ZYNTEGLO 和 SKYSONA 的生產能力，並且我們繼續看到那裡的強勁增長，需求並沒有放緩。

Number two, when you look at the ramp for LYFGENIA, we always said that it would ramp in the second half of the year and continue into 2025. As we look at more than the 30 patients that are already scheduled for next year and as you look at our QTC network of over 70 QTCs, 40 of which are looking to start their first patient, we see nothing but strong signals of demand in the market. So we're pleased with our growth, but more importantly there's a lot of unmet need in people living with sickle cell disease.

第二，當你觀察 LYFGENIA 的成長時，我們總是說它會在今年下半年成長並持續到 2025 年。當我們查看已經預約明年治療的 30 多名患者，以及查看我們的 QTC 網路（其中 40 個 QTC 正在等待治療他們的第一位患者）時，我們看到市場需求訊號強烈。因此，我們對我們的成長感到滿意，但更重要的是，鐮狀細胞疾病患者還有很多未滿足的需求。

Eric Schmidt, Cantor Fitzgerald.

艾瑞克·施密特、康托·費茲傑拉。

Good morning, and thanks for taking my question. One for James on the shareholder vote that went against you at the last stage. Can you speak to how many shares are currently authorized to be issued and how many additional shares you're asking for?

早上好，感謝您回答我的問題。給詹姆斯的一封信，關於股東在最後階段對你不利的投票。您能否說一下目前授權發行多少股以及您要求發行多少額外股份？

Go ahead, James.

繼續吧，詹姆斯。

So, about 194 million shares -- sorry, authorized to issue about 35 million shares available right now, to issue under current authority.

因此，大約有 1.94 億股 - 抱歉，目前授權發行約 3500 萬股，按照現有權力發行。

And the request for how many? Yeah.

請求數量是多少？是的。

So the request came in the form of reverse stock split, which has the effect of increasing the number of authorized shares on a relative basis. I don't have the number in front of me --

因此，請求以反向股票分割的形式提出，其效果是相對增加授權股份數量。我面前沒有號碼--

And that must depend on -- so the Board will be authorized to do a stock split in the range of 15 to 20. It depends on how -- what number that Board chooses. [Is it] 193 million or 197 million outstanding will shrink down, and therefore, that remaining 35 million will be issuable, and all this will be depend on what -- how that stock shrinks down.

這必須取決於——因此董事會將被授權進行 15 到 20 範圍內的股票分割。這取決於董事會選擇什麼數字。 1.93 億或 1.97 億的流通量將會減少，因此，剩餘的 3,500 萬將可以發行，而這一切都取決於存量如何減少。

Okay. 35 million will be fixed?

好的。 3500萬能修嗎？

Yeah.

是的。

And then, maybe one quick one for Tom. In terms of dropouts along the process, can you speak to how many total patients you've lost from the time of scheduling collection all the way through?

然後，也許對湯姆來說還有一個快速的問題。關於過程中的退出情況，您能否說一下從安排收集患者到整個過程中總共失去了多少名患者？

Go ahead, Tom.

繼續吧，湯姆。

Yeah. So it's a good question, Eric. So we're actually seeing a higher-than-anticipated pull-through rate. What we're seeing is if a patient comes off the schedule for any reason, they ultimately end up, for the most part, back on the schedule. So we're not seeing a high dropout rate.

是的。所以這是個好問題，艾瑞克。因此我們實際上看到的拉動率高於預期。我們看到的情況是，如果病人因為任何原因沒有按照計畫就診，那麼最終他們大多都會回到計畫中。因此我們的退學率並不高。

And I'll give you some examples. We did have 14 patients this year who came off the schedule for different reasons. Actually seven of them were hurricane related. They lived in an area that was impacted by hurricane. So they came off the schedule, but so far all of those patients have come back on the schedule. So, again, it's small ends, so it's early to give out those predictors, but we're really pleased with the pull-through rate that we're seeing.

我給你舉幾個例子。今年我們確實有 14 位患者因為不同原因而未能準時就診。事實上其中七起與颶風有關。他們居住在受颶風影響的地區。因此他們沒有按照計劃接受治療，但到目前為止，所有這些病人都已經恢復了計劃。因此，再說一次，這只是小規模的，所以現在給出這些預測值還為時過早，但我們對看到的拉動率感到非常滿意。

And then, if I can comment on the -- from collection to actually delivery, we have -- that is, once patient gets collected, that is essentially 100%. The exception would be that for occasionally for a sick patient versus SKYSONA, where we need to accelerate the process and not do a recollection, we might give that product for free for SKYSONA only. And so that would be the only example of a patient not converting to revenue.

然後，如果我可以評論一下——從收集到實際交付，我們有——也就是說，一旦病人被收集起來，這基本上是 100%。例外情況是，偶爾對於使用 SKYSONA 的病人，我們需要加速治療過程而不是進行回憶，我們可能會免費為 SKYSONA 提供該產品。所以這將是患者未轉化為收入的唯一例子。

Luca Issi, RBC Capital.

加拿大皇家銀行資本 (RBC Capital) 的 Luca Issi。

This is Lisa on for Luca. Just one on the gross margin. The cost of goods continue to remain higher than the revenue coming in. So wondering if you can help us understand which component is driving the high cost of goods? Is this the lentivirus production or the release assays? And what levers can you pull to reduce the expense here?

這是 Lisa 為 Luca 表演的。毛利率只有一個。商品成本仍然高於收入。所以想知道您是否可以幫助我們了解哪個因素導致了商品成本高漲？這是慢病毒生產還是釋放試驗？您可以採取什麼手段來降低此處的開支？

Go ahead, James.

繼續吧，詹姆斯。

So, it's the manufacturing for the most part, and they are high fixed costs associated with leases with our manufacturing contract providers and FTEs associated with the work. And so you're seeing high costs and negative margins at the moment as a result of the relatively low volumes in these early quarters of commercialization. We do expect that to change meaningfully as volumes increase now, including going positive in '25 on the gross profit. So there is, of course, high cost associated with the vector manufacturing as well like most of the manufacturing and testing.

因此，這在很大程度上是製造業，而且與我們的製造合約供應商的租賃以及與工作相關的全職員工相關的固定成本很高。因此，由於商業化初期產量相對較低，目前成本較高且利潤為負。我們確實預計，隨著銷量的增加，這種情況會發生重大變化，包括 25 年毛利將變為正值。因此，與大多數製造和測試一樣，向量製造的成本當然也很高。

Got it. And then, just a follow-up. Is it -- if there are more sickle cell patients getting treated with LYFGENIA, could the cost of goods come down? If I recall, I believe LYFGENIA uses a slightly different manufacturing protocol versus ZYNTEGLO. So any color here would be helpful. Thanks.

知道了。然後，只是後續行動。如果有較多的鐮狀細胞疾病患者接受 LYFGENIA 治療，藥品成本是否會下降？如果我沒記錯的話，我相信 LYFGENIA 使用的製造協議與 ZYNTEGLO 略有不同。所以這裡的任何顏色都會有幫助。謝謝。

Yeah, that's correct. Our suspension vector with LYFGENIA is lower cost than ZYNTEGLO's. So, there is an advantage there, and we expect to be able to adopt those savings over to ZYNTEGLO as we switch that.

是的，正確。我們的 LYFGENIA 懸浮載體成本比 ZYNTEGLO 的低。因此，這是一個優勢，我們希望在轉換過程中能夠將這些節省的資金轉移到 ZYNTEGLO 上。

Sami Corwin, William Blair.

薩米·科溫、威廉·布萊爾。

Good morning. Thank you for taking my question. So, last year, you saw a dip in revenue in Q4 due to some patients delaying their infusions until after the holidays. So I guess I was curious how much line of sight you have into infusion timing and what gives you confidence in hitting that $25 million in Q4?

早安.感謝您回答我的問題。因此，去年第四季的收入有所下降，因為一些患者將輸液推遲到假期後。所以我很好奇您對輸液時間的關注程度如何，以及是什麼讓您有信心在第四季達到 2500 萬美元的目標？

And then a second question is that how you kind of expect access and revenue to change with CMS's gene and cell therapy access model being rolled out to the states next year? And if that might supersede any of the negotiations you already have in place? Thank you.

第二個問題是，隨著 CMS 的基因和細胞治療模式明年在各州推廣，您預計治療管道和收入將如何變化？這是否會取代您已進行的任何談判？謝謝。

Go ahead, Tom.

繼續吧，湯姆。

Yeah, obviously, we're tracking very closely the number of infusions that have already happened in Q4 along with the infusions that are scheduled right now. That gives us the confidence in getting to the $25 million that we're projecting.

是的，顯然，我們正在密切關注第四季度已經發生的注入次數以及目前安排的注入次數。這使我們有信心實現預計的 2500 萬美元目標。

And then, Sami, the second part of your question was -- can you repeat that for us?

然後，薩米，你的問題的第二部分是──你能為我們重複一遍嗎？

Yeah. How you expect access and revenue to change with CMS's gene cell therapy access model being rolled out to the states next year? And if that might supersede any negotiations you already have in place?

是的。隨著 CMS 的基因細胞治療模式明年在各州推廣，您預計治療機會和收入將如何變化？這是否會取代您已經進行的任何談判？

Yeah. So we are -- I mean, we are -- we said before, we are collaborating with CMS on the CMMI program. We would expect to hear sometime in early December about that. That is a national program where they would provide a framework that states could access. We, of course, already have direct access with the states and have been having conversations with them as well. So it is more an accelerator, I would call it, than anything one replacing the other.

是的。所以我們 — — 我的意思是，我們 — — 我們之前說過，我們正在與 CMS 合作開展 CMMI 計劃。我們預計將在 12 月初聽到有關該消息。這是一個國家計劃，他們將提供一個各州都可以訪問的框架。當然，我們已經與各州建立了直接聯繫，也一直在與他們進行對話。因此，我認為它更像是一種加速器，而不是任何可以取代其他東西的東西。

Thank you. And I'm showing no further questions at this time. I will now turn the call back over to Mr. Andrew Obenshain for any closing remarks.

謝謝。我現在沒有其他問題了。現在我將把電話轉回給安德魯·奧本沙因先生，請他做最後發言。

So thank you very much everyone for joining the call today, and we look forward to updating you in the first quarter next year.

非常感謝大家今天的電話會議，我們期待明年第一季向大家報告最新情況。

Ladies and gentlemen, that does conclude our conference for today. Thank you for your participation, and you may now disconnect.

女士們、先生們，今天的會議到此結束。感謝您的參與，您現在可以斷開連接。