BioXcel Therapeutics Inc (BTAI) 2023 Q3 法說會逐字稿

Good morning, and welcome to BioXcel Therapeutics' Second Quarter 2023 Financial Results Conference Call. At this time, all participants are in a listen-only mode. (Operator Instructions) After the speakers' presentation, there will be a question-and-answer session. (Operator Instructions)

早安，歡迎參加 BioXcel Therapeutics 2023 年第二季財務業績電話會議。此時，所有參與者都處於只聽模式。 （操作員說明） 演講者演講結束後，將進行問答環節。 （操作員說明）

Just to remind everyone, certain matters discussed in today's conference call and/or answers that may be given to questions asked are forward-looking statements that are subject to risks and uncertainties related to future events and/or the future financial or business performance of the company. Actual results could differ materially from those anticipated in these forward-looking statements.

只是提醒大家，今天的電話會議中討論的某些事項和/或可能對提出的問題作出的回答屬於前瞻性陳述，這些陳述受到與未來事件和/或未來財務或業務績效相關的風險和不確定性的影響。公司。實際結果可能與這些前瞻性陳述中的預期有重大差異。

Risk factors that may affect future results are detailed in the company's annual report on Form 10-Q for the quarter ended March 31, 2023, which can be found at www.bioxceltheapeutics.com, or on www.sec.gov, and which will be updated in its quarterly report on Form 10-Q for the quarter ended June 30, 2023. As a reminder, today's conference is being recorded.

可能影響未來業績的風險因素詳見公司截至 2023 年 3 月 31 日的季度 10-Q 表格年度報告，該報告可在 www.bioxceltheapeutics.com 或 www.sec.gov 上找到，該報告將在截至2023 年6 月30 日的季度的10-Q 表格季度報告中進行更新。謹此提醒，我們正在錄製今天的會議。

Joining us on today's call are Dr. Vimal Mehta, Chief Executive Officer; Richard Steinhart, Chief Financial Officer; Joining them for participation in Q&A session are - Matt Wiley, Chief Commercial Officer; Dr. Rob Risinger, Chief Medical Officer of Neuroscience; Dr. Vince O'Neill, Chief R&D Officer of OnkosXcel Therapeutics; and Dr. Frank Yocca, Chief Scientific Officer.

加入我們今天電話會議的還有執行長 Vimal Mehta 博士；理查德‧史坦哈特，財務長；與他們一起參加問答環節的有：商務長 Matt Wiley； Rob Risinger 博士，神經科學首席醫學官； Vince O'Neill 博士，OnkosXcel Therapeutics 首席研發長；和首席科學官 Frank Yocca 博士。

It is now my pleasure to turn the call over to Dr. Mehta.

現在我很高興將電話轉給梅塔醫生。

Thank you, operator. Good morning, and thank you for joining us. Today I begin with several exciting and highly anticipated updates on our recent development with our two late-stage clinical program for BXCL501 TRANQUILITY and SERENITY III.In addition, I will cover important news about our financing plans with Oaktree Capital Management and Qatar Investment Authority.

謝謝你，接線生。早安，感謝您加入我們。今天，我首先介紹有關BXCL501 TRANQUILITY 和SERENITY III 兩個後期臨床項目的最新進展的一些令人興奮且備受期待的更新。此外，我還將報道有關我們與橡樹資本管理公司和卡塔爾投資局的融資計劃的重要新聞。

After this, I will touch briefly on update for our regarding the commercial activities and oncological benefit. Rich will then cover the financial results for the third quarter. let me begin with TRANQUILITY and SERENITY III programs. I am very pleased to highlight the tremendous progress that we have made with these late-stage clinical programs, which we believe represent significant value-creating catalysts.

之後，我將簡要介紹我們有關商業活動和腫瘤學益處的最新情況。里奇隨後將報導第三季的財務表現。讓我從 TRANQUILITY 和 SERENITY III 計劃開始。我非常高興地強調我們在這些後期臨床項目上取得的巨大進展，我們相信這些進展代表了重要的價值創造催化劑。

In both cases, we completed a productive meeting with the FDA regarding to portfolio development that we are aligned with the FDA's recommendation regarding a Phase 3 trial in the at-home setting for TRANQUILITY program as confirmed in the meeting minutes.

在這兩種情況下，我們與 FDA 就產品組合開發進行了富有成效的會議，會議記錄中確認，我們與 FDA 關於 TRANQUILITY 計劃家庭 3 期試驗的建議保持一致。

And our development path SERENITY III is based on the feedback we received at last week's meeting with the FDA subject to receipt of final meeting minutes we expect in December. Going into the TRANQUILITY in more detail, we are aligned with the FDA's recommendation regarding an additional Phase 2 trial in the adjuvant setting for TRANQUILITY as a potential path to sNDA submission.

我們的 SERENITY III 開發路徑是基於我們在上週與 FDA 舉行的會議上收到的回饋，並預計在 12 月收到最終會議紀錄。更詳細地討論 TRANQUILITY，我們同意 FDA 關於在 TRANQUILITY 輔助環境中進行額外 2 期試驗的建議，作為提交 sNDA 的潛在途徑。

This important development follows our positive pivotal TRANQUILITY II study results. In June, we completed independent third party TRANQUILITY II trial audit in October and receipt of our FDA meeting minutes just this month.

這項重要進展是在我們積極的關鍵 TRANQUILITY II 研究結果之後取得的。 6 月，我們於 10 月完成了獨立第三方 TRANQUILITY II 試驗審核，並於本月收到了 FDA 會議紀錄。

Specifically, we plan to conduct a Phase 3 clinical trial of BXCL501 in that home setting for the acute treatment of agitation associated with Alzheimer's disease. This potential market opportunity could include acute treatment of agitation across the full spectrum of Alzheimer's and related dementia and severity of agitation across all care settings.

具體來說，我們計劃在家庭環境中進行 BXCL501 的 3 期臨床試驗，用於急性治療與阿茲海默症相關的躁動。這個潛在的市場機會可能包括對所有阿茲海默症和相關癡呆症以及所有護理環境中躁動嚴重程度的急性治療。

We believe we are well positioned to bring BXCL501 to this very large and underserved market, which represents a unique opportunity for which there are no FDA-approved drugs. Why the agency has approved the chronic agitation treatment. We believe we are breaking new ground for this important unmet medical need.

我們相信，我們有能力將 BXCL501 引入這個非常大且服務不足的市場，這代表著一個獨特的機會，目前還沒有 FDA 批准的藥物。為什麼該機構批准了慢性躁動治療。我們相信，我們正在為這重要的未滿足的醫療需求開闢新天地。

We are pleased that as a result of our updated development plan that TRANQUILITY trial is no longer required as part of an sNDA submission. We expect this will lead us to redeploy resources and focus on the recently agreed at Homebase trial for the TRANQUILITY program.

我們很高興地看到，由於我們更新了開發計劃，TRANQUILITY 試驗不再需要作為 sNDA 提交的一部分。我們預計這將導致我們重新部署資源，並專注於最近在 Homebase 商定的 TRANQUILITY 計劃試驗。

In summary we believe we are now poised to advance the program as efficiently and cost effectively. Our confidence is further reinforced by the positive findings of an independent third party audit of data integrity at the single and TRANQUILITY II trial site. We have previously identified. This audit found no evidence of misconduct or for our beyond the instant previously reported. In addition, no findings was identify that impact data integrated. We look forward to finalizing our study protocol and moving this forward.

總而言之，我們相信我們現在已準備好高效且具有成本效益地推進該計劃。獨立第三方對單一試驗場和 TRANQUILITY II 試驗場的資料完整性審計的正面結果進一步增強了我們的信心。我們之前已經確定了。這次審計沒有發現任何不當行為的證據，也沒有發現我們先前報告的不當行為的證據。此外，沒有發現任何影響數據整合的發現。我們期待最終確定我們的研究方案並繼續前進。

Let me now turn to SERENITY III program, which is also making steady progress. As a reminder, here, we are evaluating the potential at home use of BXCL501 for agitation associated with bipolar disorder and schizophrenia.

現在讓我談談 SERENITY III 計劃，該計劃也在穩步取得進展。提醒一下，我們正在評估 BXCL501 在家中用於治療與躁鬱症和精神分裂症相關的躁動的潛力。

We've got an approved indication from government. Last week, we completed a productive meeting with the FDA and expect to receive meeting minutes in the first half of December. However, I can share now that based on preliminary FDA feedback, we plan to conduct an additional Phase 3 trial evaluating the safety of the 120 microgram dose to treat agitation in the home setting associated with bipolar and schizophrenia.

我們已經得到政府批准的指示。上週，我們與 FDA 完成了一次富有成效的會議，預計在 12 月上半月收到會議紀要。然而，我現在可以分享的是，根據 FDA 的初步回饋，我們計劃進行一項額外的 3 期試驗，評估 120 微克劑量治療家庭環境中與雙相情感障礙和精神分裂症相關的躁動的安全性。

We'll provide more details on this program as we advance for now, we are pleased that we have multiple opportunities to bring BXCL501 to a much larger number of patients in the home setting. This is the expand portion of our land and expand strategy. In conjunction with the clinical development. While we are focused on enhancing our operational and financial flexibility.

隨著目前進展，我們將提供有關該計劃的更多詳細信息，我們很高興有多次機會將 BXCL501 帶給更多家庭患者。這是我們的土地擴張部分和擴張策略。結合臨床發展。我們專注於提高營運和財務靈活性。

As reported today, we have entered into a binding term sheet with the Oaktree Capital and Qatar Investment Authority to amend our existing financing agreements, subject to final documentation, we have agreed to revise down that will increase our potential to access additional tranches of capital and have agreed to revise the revenue covenants to extend the compliance requirements. And covenant levels to align with our current projections following our business reprioritization.

正如今天報導的那樣，我們已與橡樹資本和卡達投資局簽訂了一份具有約束力的條款清單，以修改我們現有的融資協議，根據最終文件，我們已同意進行向下修改，這將增加我們獲得更多資本和資金的潛力。已同意修改收入契約以擴大合規要求。在我們重新調整業務優先順序後，契約水準與我們目前的預測保持一致。

We believe this is very positive news for the company, and this is an integral part of our overall financing strategy. We are grateful to our strategic financial partner for their ongoing support. safely.

我們相信這對公司來說是非常正面的消息，也是我們整體融資策略不可或缺的一部分。我們感謝我們的策略金融合作夥伴的持續支持。安全。

Briefly, we have focus commercial efforts to provide access for the government to our current customers and deploy direct contracting with hospital systems. The recent issuance of a [J code] by CMS is expected to streamline the reimbursement process across commercial and government payers. We hope this will help us continue to establish brand equity in economy and act as a bridge to the larger potential at-home market opportunities.

簡而言之，我們的重點是商業努力，為政府提供與我們目前客戶的接觸，並部署與醫院系統的直接合約。 CMS 最近發布的 [J 代碼] 預計將簡化商業和政府付款人的報銷流程。我們希望這將有助於我們繼續在經濟中建立品牌資產，並成為通往更大潛在國內市場機會的橋樑。

Additionally, if [currently] patent protection has been extended through 2043 with two new Orange Book-listed US patents.

此外，如果[目前]專利保護已延長至 2043 年，有兩項新的橙皮書列出的美國專利。

Before wrapping up, I would like to highlight that we are pleased with the continued progress of NIDA-funded Trial of BXCL501 for potential treatment of opioid use disorder being conducted by Columbia University.

在結束之前，我想強調的是，我們對哥倫比亞大學正在進行的 NIDA 資助的 BXCL501 潛在治療阿片類藥物使用障礙試驗的持續進展感到高興。

This may offer an important opportunity to address fentanyl combined with Allogene in what has been called an emerging threat by the White House Office of National Drug Control Policy. I'm also energized by our emerging neuroscience clinical development program that are the result of our unique use of artificial intelligence platform to drive drug innovation. We look forward to sharing more information about our opioid use disorder program and exciting pipeline in an R&D event that we plan to host next month.

這可能為解決芬太尼與 Allogene 聯合使用被白宮國家藥物管制政策辦公室所謂的新威脅提供了一個重要的機會。我還對我們新興的神經科學臨床開發計劃充滿活力，這是我們獨特地利用人工智慧平台來推動藥物創新的結果。我們期待在我們計劃於下個月舉辦的研發活動中分享有關我們的阿片類藥物使用障礙計劃和令人興奮的管道的更多資訊。

Look for more details soon.

盡快查找更多詳細資訊。

In addition, we were pleased with the recent positive survival data reported for BXCL501 in our open-label Phase two trial in patients with metastatic castration-resistant prostate cancer and in patients with small cell neuroendocrine prostate cancer.

此外，我們對最近在轉移性去勢抵抗性前列腺癌患者和小細胞神經內分泌前列腺癌患者的開放標籤二期試驗中報告的 BXCL501 的積極生存數據感到滿意。

With these data in hand, we are focusing on various strategic options for oncolytic. I'd like to end by thanking all of our colleagues for their dedication to our mission of bringing transformative medicines to patients is ultimately their tireless work that drives our census.

有了這些數據，我們將專注於溶瘤的各種戰略選擇。最後，我要感謝我們所有的同事，他們致力於為患者提供變革性藥物，最終推動我們的人口普查。

I will now turn the call over to Rich, who will review our third quarter financial results, Rich?

我現在將電話轉給里奇，他將審查我們第三季的財務業績，里奇？

Thank you Vimal. The quarter was indeed a transfer of a transformative time for the company, and I'm pleased to review our financial results for the third quarter of 2023. Net revenue of the company was approximately $341,000 for the quarter. Research and development expenses were $19.6 million for the third quarter of 2023 compared to $22.1 million for the same period in 2022.

謝謝維馬爾。該季度確實是公司轉型時期的轉變，我很高興回顧我們 2023 年第三季的財務業績。該季度公司的淨收入約為 341,000 美元。 2023 年第三季的研發費用為 1,960 萬美元，而 2022 年第三季的研發費用為 2,210 萬美元。

The decreased expenses were primarily attributable to a decrease in costs associated with the BXCL501 SERENITY III and the TRANQUILITY II clinical trials. Selling, general and administrative expenses were $24.3 million for the third quarter of 2023 compared to $17.1 million for the same quarter in 2022. The increased expenses were primarily attributable to an increase in one-time legal and professional fees, costs associated with the oncolytic sale potential public offering, as well as in personnel and related costs to support commercialization of the Algami in the United States prior to our reprioritization.

費用減少主要歸因於 BXCL501 SERENITY III 和 TRANQUILITY II 臨床試驗相關成本的減少。 2023 年第三季的銷售、一般和管理費用為 2,430 萬美元，而 2022 年第三季為 1,710 萬美元。費用增加的主要原因是一次性法律和專業費用以及與溶瘤藥物銷售相關的費用增加潛在的公開發行，以及在我們重新確定優先順序之前支持Algami 在美國商業化的人員和相關成本。

BioXcel Therapeutics had a net loss of $50.5 million for the third quarter of 2023 compared to a net loss of $41.8 million for the same period in 2022. The company used approximately $37.6 million in operating cash during the third quarter. Cash and cash equivalents totaled $90 million. As of September 30, 2023, company estimates that its current cash and cash equivalents will last through mid 2024. The estimated cash runway does not include potential additional capital that may become available under the amendments to the strategic financing agreements or resulting from any potential financing activities that we may undertake.

BioXcel Therapeutics 2023年第三季淨虧損為5,050萬美元，而2022年同期淨虧損為4,180萬美元。該公司第三季使用了約3,760萬美元的營運現金。現金及現金等價物總計 9,000 萬美元。截至 2023 年 9 月 30 日，公司估計其當前現金和現金等價物將持續到 2024 年中期。估計的現金跑道不包括根據戰略融資協議修正案或任何潛在融資可能產生的潛在額外資本我們可能進行的活動。

Now I'd like to turn the call back to Vimal.

現在我想把電話轉回維馬爾。

Thank you Rich we would now like to open the call for questions.

謝謝里奇，我們現在想開始提問。

Operator?

操作員？

Thank you.

謝謝。

(Operator Instructions)

（操作員說明）

Greg Harrison with Bank of America.

美國銀行的格雷格·哈里森。

Good morning, guys. Thanks for the update. And for taking the question with respect to the 100 patient trial, can you give us some more color on the TRANQUILITY development strategy as of going for a broad label initially? And then what are the criteria for success in this trial and what would a positive trial look like?

早上好傢伙。感謝更新。對於 100 名患者試驗的問題，您能否給我們更多關於 TRANQUILITY 開發策略的信息，就像最初尋求廣泛的標籤一樣？那麼這次試驗成功的標準是什麼？積極的試驗是什麼樣的呢？

Good morning, Greg. This is Vimal. We were very pleased that we have alignment on recommendation from the FDA to conduct a home setting time. As you know, home setting out most of the patients in Alzheimer's do live in their homes. I think and this was a very important development after we observed in TRANQUILITY III, that number of episodes that were required were like no more chronic in nature. So we don't need to conduct TRANQUILITY III anymore.

早安，格雷格。這是維馬爾。我們很高興我們能夠按照 FDA 的建議進行家庭設定時間。如您所知，大多數阿茲海默症患者確實住在自己的家中。我認為，這是我們在《寧靜 III》中觀察到的一個非常重要的發展，所需的發作次數本質上不再是慢性的。所以我們不需要再進行TRANQUILITY III了。

So under the circumstances, this is our best case scenario to move this drug and provide access to this medication to broadest possible patient population. So that's very exciting. And in terms of the clinical trial program, I will pass it on to Rob to describe what this program will entail at. Just remember, we just receive the meeting minutes from the FDA and we are in the process of developing the clinical protocol protocol right now.

因此，在這種情況下，這是我們轉移這種藥物並向盡可能廣泛的患者群體提供這種藥物的最佳情況。所以這非常令人興奮。至於臨床試驗計劃，我會將其轉交給 Rob 來描述該計劃的內容。請記住，我們剛剛收到 FDA 的會議紀要，我們現在正在製定臨床方案。

Rob

搶

So let me just highlight that we're really excited that we now agree with the FDA's proposed design will be a double-blind placebo-controlled study, primarily safety, and that includes describing clinical benefit as assessed by family or caregivers. We're actually working on the protocol and we expect to get back to you with them. Were there facts about that protocol?

因此，我要強調的是，我們非常興奮，我們現在同意 FDA 提議的設計將是一項雙盲安慰劑對照研究，主要是安全性，其中包括描述由家庭或照護者評估的臨床益處。我們實際上正在製定協議，希望盡快與您聯繫。有關於該協議的事實嗎？

Got it. And can you clarify whether you're able to use all of the data collected from TRANQUILITY II as part of their package.

知道了。您能否澄清一下您是否能夠使用從 TRANQUILITY II 收集的所有資料作為其軟體包的一部分。

Company believe after receiving our independent audit and call the information we have that we will be able to use that TRANQUILITY II data that we announced in June.

公司相信，在收到我們的獨立審計並致電我們掌握的資訊後，我們將能夠使用我們在 6 月宣布的 TRANQUILITY II 數據。

Great.Thanks for taking the questions.

太好了。感謝您提出問題。

Thanks.

謝謝。

Rag Selvaraju, H.C. Wainwright.

塞爾瓦拉朱 (Rag Selvaraju)，H.C.溫賴特。

Hi. Thanks for taking my questions. I was wondering, first of all, if you could provide us with some additional context and clarity on what you expect to be the ultimate path forward for fiber I in chronic agitation and within what time line you expect to pursue this? And if we should be thinking about this as being something that you would look to target formally once you have and approval of the label in Alzheimer's associated acute agitation.

你好。感謝您回答我的問題。首先，我想知道您是否能為我們提供一些額外的背景資訊並明確說明您期望纖維 I 在慢性躁動方面的最終前進道路，以及您期望在什麼時間內實現這一目標？如果我們應該考慮到這一點，那麼一旦您獲得並批准了阿茲海默症相關急性躁動的標籤，您就會正式瞄準這一目標。

That's a great question, Ram. When we had the conversation with the FDA on TRANQUILITYIII, considering there are so many number of episodes. And we did those Fiber One multiple times to to take those agitation episodes. So there was a discussion that would there be a possibility that Fiber I may be useful or could treat or could be developed as a chronic treatment?

這是一個很好的問題，拉姆。當我們與 FDA 就《TRANQUILITYIII》進行對話時，考慮到劇集數量如此之多。我們多次進行了“纖維一號”，以應對那些激動的情況。因此，有人討論纖維 I 是否有可能有用或可以治療或可以開發為慢性治療方法？

We have done chronic dosing, as you know, in the healthy volunteers for our MDD program. And we have data, but we have no data right now that it can treat chronic agitation. We will work with the agency to developed a path of explore the path. Currently, our laser focus is on acute treatment of agitation in a home setting so that we can expand our use of this drug in Alzheimer's related agitation. So that that path exists for us and we will have the opportunity to explore. But at this point in time, the strategy will be to deploy all resources and focus in completion of that trial in a home setting.

如您所知，我們在 MDD 計畫中對健康志願者進行了長期給藥。我們有數據，但目前還沒有數據顯示它可以治療慢性躁動。我們將與該機構共同製定一條探索路徑。目前，我們的重點是家庭環境中躁動的急性治療，以便我們可以擴大該藥物在阿茲海默症相關躁動中的使用。因此，這條道路對我們來說是存在的，我們將有機會探索。但目前的策略是部署所有資源並專注於在家庭環境中完成該試驗。

Okay. And then secondly, with respect to the Opioid Use disorder indication, do you have any additional information you can provide to us regarding the potential size of this market opportunity and maybe give us a sense of how you would expect 501 to potentially be deployed if it were to be approved on.

好的。其次，關於阿片類藥物使用障礙的適應症，您是否可以向我們提供有關該市場機會的潛在規模的任何其他信息，也許可以讓我們了解如果它出現，您預計如何部署 501待批准。

So as you note on this program is funded by NIDA. It's a huge, huge on national emerging track and the investigators we are working. They are wildly known investigators in this area and in the ongoing trial Phase 2 trial upon outcome of that trial, we will know what the path forward will be. But in terms of the market opportunity. I will pass it on to Matt if he can provide any color on this.

正如您所注意到的，該計劃是由 NIDA 資助的。這是一個巨大的國家新興軌道和我們正在工作的調查人員。他們是該領域廣為人知的研究人員，在正在進行的第二階段試驗中，根據該試驗的結果，我們將知道前進的道路是什麼。但就市場機會而言。如果馬特能提供任何相關信息，我會將其轉告給他。

Well, Rag I will give us additional data as we get closer to data readout. But we do know that the fentanyl plus sales, our phenomenon is an emerging threat.

好吧，當我們接近數據讀出時，Rag I 將為我們提供更多數據。但我們確實知道芬太尼加銷售，我們的現像是一個新興的威脅。

It's growing. And so there's not really great epidemiology yet on and we do know that certain cities like Philadelphia, where the state of Connecticut, for instance, has significant problems with this emerging threat. And it's certainly gotten the attention of the White House. We've got others we've got to do.

它正在增長。因此，目前還沒有真正的流行病學研究，但我們確實知道某些城市，例如費城，例如康乃狄克州，在這種新出現的威脅方面存在嚴重問題。這當然引起了白宮的注意。我們還有其他事情要做。

And lastly, if you can maybe comment on, you know, obviously, there's been some recent data that looks pretty positive for OnkosXcel. So can you give us a sense of, you know, on a qualitative basis and to what extent the availability of this clinical data might make it easier for you to either identify some kind of strategic partnership for seven oh one or indeed consummate spin out of OnkosXcel sell into a separate entity.

最後，如果您可以發表評論，您知道，顯然，最近的一些數據看起來對 OnkosXcel 非常積極。那麼，您能否讓我們了解一下，您知道，在定性的基礎上，以及這些臨床數據的可用性在多大程度上可以使您更容易地確定七對一的某種戰略合作夥伴關係，或者實際上是完美的分拆OnkosXcel 出售給一個單獨的實體。

With the data in hand and you saw the data from the two trials, I think that's very reinforcing about the 7.1 potential. Vince, you want to take the question?

有了手頭上的數據，並且您看到了兩次試驗的數據，我認為這非常增強了 7.1 的潛力。文斯，你想回答這個問題嗎？

Yes, I monitor. I would just add to that, that the really the complete data package that we have, as you said, including survival, but also biomarker work is exactly the data potential partners would really want and need to see to form an assessment. So now that we have that in hand, I can tell you we are actively having those conversations.

是的，我監控。我想補充一點，正如您所說，我們擁有的真正完整的數據包，包括生存率，還有生物標誌物工作，正是潛在合作夥伴真正想要和需要查看以進行評估的數據。現在我們已經掌握了這些訊息，我可以告訴你我們正在積極地進行這些對話。

Thank you.

謝謝。

Sumant Kulkarni with Canaccord Genuity.

Sumant Kulkarni 與 Canaccord Genuity。

Good morning. Thanks for taking my questions and all the updates. So on your alignment with the FDA, did you specifically ask the agency about your ability to file on the basis of the TRANQUILITY II trial alone and asked another way, I guess, why did the company not choose to go with a more limited sNDA initially with the potential to expand the label further at some time? Or was that not possible because you're still interested establish a safety profile and order patiets?

早安.感謝您提出我的問題和所有更新。那麼，關於您與 FDA 的合作，您是否專門詢問過該機構您是否有能力僅根據 TRANQUILITY II 試驗進行提交，並以另一種方式詢問，我想，為什麼該公司最初不選擇採用更有限的 sNDA是否有可能在某個時候進一步擴大品牌規模？或者這是不可能的，因為您仍然有興趣建立安全檔案並訂購病人？

Sumant with the agency when we met, we had all the conversation around the TRANQUILITY II program, what will allow us to file an sNDA in terms of our strategy, we adopted this strategy. We want to have the broadest access to this data to the patient. We know 80% of the patients live in a home setting, which is home and ALF.

當我們與該機構見面時，我們圍繞 TRANQUILITY II 計劃進行了所有對話，這將使我們能夠根據我們的策略提交 sNDA，我們採用了這一戰略。我們希望患者能夠最廣泛地存取這些數據。我們知道 80% 的患者生活在家庭環境中，即家庭和 ALF。

We have data in the airlines and to be able to get to the 8% patients, we needed to demonstrate safety and collect data on efficacy in a home setting. And that was the optimal choice by the company working with the agency that this will allow a broader access to the patients and also for their caregivers.

我們在航空公司有數據，為了能夠接觸到 8% 的患者，我們需要證明安全性並收集家庭環境中的療效數據。這是該公司與該機構合作的最佳選擇，因為這將使患者及其照護者能夠更廣泛地接觸患者。

One of the biggest reasons for these patients who go from a home to ALF or to nursing home is, as you know, in Alzheimer's patient is agitation. So we thought this is the best case scenario that we conduct a study in a home setting and get the best possible label we can in this patient population where, as you know, there are no drugs approved and that are not to our knowledge, no drugs under development also for acute treatment of agitation. So we have a very unique position.

如您所知，這些患者從家中前往 ALF 或療養院的最大原因之一是阿茲海默症患者的躁動。因此，我們認為這是最好的情況，我們在家庭環境中進行研究，並在這個患者群體中獲得盡可能最好的標籤，正如你所知，沒有藥物獲得批准，而且據我們所知，沒有藥物正在開發的藥物也用於急性躁動的治療。所以我們擁有非常獨特的地位。

Got it. And then is there a specific limit to the number of episodes that you can treat the four week period in this new trial to be considered an acute treatment? And how confident are you that the new at-home trial will not end up in a TRANQUILITY III like outcome where AD agitation is not really acute, chronic.

知道了。那麼，在這項新試驗中，四個星期內可以治療的被視為急性治療的發作次數是否有具體限制？您對新的家庭試驗不會產生類似 TRANQUILITY III 那樣的結果（AD 躁動並非真正急性、慢性）的結果有多大信心？

We are not aware of any guidelines like migraine where you have 15 episodes and it's acute and after 15 is considered as chronic, does that the path we will be working with the FDA? What is the definition of a acute and episodic, but as Rob said, we are developing a protocol and he will be providing you the details that what our inclusion criteria will be for a acute episodic treatment.

我們不知道有任何指南，例如偏頭痛，發作 15 次後為急性，15 次後被視為慢性，這是否是我們與 FDA 合作的途徑？急性和偶發性的定義是什麼，但正如 Rob 所說，我們正在製定一項方案，他將為您提供有關急性偶發性治療的納入標準的詳細資訊。

And we will be discussing more on this one. But to answer your question, there is not a clear guideline set up or a path about that or they know their trade, their inflammation will define that if you have X number of episodes is acute and if you have a number of episodes, it's gone.

我們將對此進行更多討論。但要回答你的問題，沒有明確的指導方針或相關路徑，或者他們知道自己的行業，他們的發炎將定義如果你有X次發作是急性的，如果你有多次發作，它就消失了。

And then the last question before I jump back in the queue, is on the 73 program you now are allowed to go home with a 1,000 microgram dose. Could you just give us some color on what led did that and the discussions with the FDA around that, given it's higher than what you had contemplated in the past?

然後，在我回到隊列之前，最後一個問題是關於 73 計劃，您現在可以攜帶 1,000 微克劑量回家。鑑於它比您過去預期的要高，您能否給我們介紹一下是什麼導致了這一結果，以及與 FDA 圍繞此進行的討論？

Yes. So when we had meeting on the city and the drill program, we had multiple choices for the doses. As you know, one 20 microgram is already approved dose for Aegon NV and now on company estimates that it has been given to at least more than 10,000 people, which is 120 and higher doses 180 and there is a lot of data generated which provide the confidence to the company as well as to the agency that this could be doors we should evaluate in a home setting.

是的。因此，當我們就城市和演習計劃召開會議時，我們對劑量有多種選擇。如您所知，1 20 微克已經是 Aegon NV 批准的劑量，現在公司估計該劑量已用於至少 10,000 多人，即 120 和更高劑量 180，並且生成的大量數據提供了讓公司和機構相信這可能是我們應該在家庭環境中評估的門。

We also had a choice to evaluate 80 where you understand and know that we had done PK/PD modeling based on our 60 microgram dose and we could have chosen the 80 microgram dose. Dose flexibility exists. The reason we are choosing one, 20 already a lot of safety data on 120 efficacy is already established. We need to evaluate primarily that safety in a home setting, and that will allow us to capture about 23 million episode that happened in a home setting and extend it beyond the 16 million episode that's in the hospital.

我們也可以選擇評估 80，您了解並知道我們已經根據 60 微克劑量進行了 PK/PD 建模，並且我們可以選擇 80 微克劑量。存在劑量彈性。我們選擇其中一個的原因是，20 已經有很多關於 120 功效的安全數據已經確定。我們首先需要評估家庭環境中的安全性，這將使我們能夠捕捉家庭環境中發生的大約 2300 萬個事件，並將其擴展到醫院中發生的 1600 萬個事件之外。

So it was very synergistic. Same does that give one in a hospital setting, if it can go in a home setting. And if patients in our homes adding need any more like no medication, they can always get 180 in the hospitals. So it's very, very synergistic. And as part of the reason we have chosen one 24 evaluating in a home setting.

所以這是非常協同的。如果它可以在家庭環境中使用，那麼在醫院環境中也可以提供相同的效果。如果我們家裡的病人需要更多的藥物，例如不需要藥物，他們總是可以在醫院得到 180。所以這是非常非常協同的。作為我們選擇 24 小時在家庭環境中進行評估的部分原因。

Thank you.

謝謝。

Colin Bristow with UBS.

瑞銀集團的柯林布里斯托。

Good morning and thanks for the update, mainly just a point of clarification on the path forward in Alzheimer's agitation. I think I heard you say the company believes the TRANQUILITY II data can be used, but what did FDA specifically say about the miscibility of the data? Or will this still remain a review issue?

早安，感謝您的更新，主要是對阿茲海默症躁動的前進道路進行澄清。我想我聽到你說該公司相信 TRANQUILITY II 數據可以使用，但是 FDA 對於數據的可混合性具體說了些什麼？或者這仍然是一個審查問題？

And then on the additional study. Can you just know from the time lines of properties we're going to this is looking to be sort of 2025 readout and then certainly on the cash runway.

然後再進行額外的學習。你能從我們打算的房產時間線上知道，這將是 2025 年的讀數，然後肯定會出現在現金跑道上。

And then on the cash runway, you're locked in Q2 you said your cash will get into mid '24. You've maintained that language. Is that simply because the updated agreement is finalized. More detail would be really helpful. Thanks.

然後在現金跑道上，你被鎖在第二季度，你說過你的現金將進入 24 年中期。你一直保持著這種語言。這只是因為更新後的協議已經敲定嗎？更多細節將會非常有幫助。謝謝。

Sure, Colin, this is Vimal. Coming back to your question about specifically about the TRANQUILITY II data. . We have no reason to believe or we have not any discussion which tells that TRANQUILITY data is not usable.

當然，科林，這是維馬爾。回到你關於 TRANQUILITY II 資料的問題。 。我們沒有理由相信，或者我們沒有任何討論表明 TRANQUILITY 資料不可用。

As you know, once you do submission of your sNDA, FDA does his assessment and that will continue to be the case for any sNDA. And in terms of our discussion, we have no reason to believe that. And as we said, company believes this data is usable based on our own assessment as well as on the independent audit that was recently concluded.

如您所知，一旦您提交了 sNDA，FDA 就會進行評估，並且任何 sNDA 都將繼續如此。就我們的討論而言，我們沒有理由相信這一點。正如我們所說，根據我們自己的評估以及最近的獨立審計結果，公司相信這些數據是有用的。

On your second question was what about the additional study then the readout will be there. As you notice that we have about a 100 patient study to conduct it in a home setting. We are developing the protocol. We are designing how many sites will be required to conduct the study. Only thing I can mention it is that conducting a trial in home setting is going to be a lot more easy than conducting a trial like TRANQUILITY II and III.

關於你的第二個問題，額外的研究怎麼樣，然後讀數就會在那裡。正如您所注意到的，我們有大約 100 名患者在家庭環境中進行的研究。我們正在開發該協議。我們正在設計進行這項研究需要多少個站點。我唯一可以提到的是，在家庭環境中進行試驗將比進行 TRANQUILITY II 和 III 等試驗容易得多。

The reason for that is in TRANQUILITY II entry. You have to helicopter in that CRO to make that assessment for the efficacy. And as we mentioned, we are trying to evaluate the safety and we'll continue to collect caregiver assessment of the efficacy.

原因就在 TRANQUILITY II 條目中。您必須直接前往 CRO 進行療效評估。正如我們所提到的，我們正在嘗試評估安全性，並將繼續收集護理人員對功效的評估。

So this time, we expect our burn to be relatively relatively easier in that setting, but we have not done a trial in a home setting. So we are very diligently working with our CRO, defining the protocol, getting that alignment on the protocol and very soon, we will be able to come back and say when we plan to initiate that trial when the first patient will be dosed when recruitment will be completed and how long will it take and when the value inflection catalyst will be there for the Alzheimer's related agitation program.

所以這次，我們預計在這種環境下我們的燒傷會相對容易一些，但我們還沒有在家庭環境中進行試驗。因此，我們正在與我們的CRO 非常努力地合作，定義方案，使方案與方案保持一致，很快，我們將能夠回來並說明我們計劃何時啟動該試驗，何時對第一個患者進行給藥，何時招募完成，需要多長時間，以及阿茲海默症相關鼓動計畫的價值拐點催化劑何時出現。

Third question is related to the Oak Tree. Recently we had concluding a binding term sheet with Opiant, Qatar Investment Authority. We are very grateful that they are very supportive. They have belief in BXCL501 and like us like we believe in it and now having a very clear path for Alzheimer's related agitation and also a path for expanding the label for [Regal may] in a home setting, which is the three program we bought.

第三個問題與橡樹有關。最近，我們與卡達投資局 Opiant 簽訂了一份具有約束力的條款清單。我們非常感謝他們的大力支持。他們對BXCL501 有信心，就像我們一樣相信它，現在有一條非常明確的治療阿茲海默症相關躁動的途徑，也有一條在家庭環境中擴大[Regal may] 標籤的途徑，這是我們購買的三個項目。

And based on our recent reprioritization of our commercial efforts and and in the organization, this agreement our tons needed to be amended. We are very pleased to report today. They have been agreed upon under the binding term sheet, which will be documented very soon. And I will pass it on to Richard so that he can outline what is the value for the organization and why this was needed.

根據我們最近對商業努力和組織的優先順序的重新安排，我們需要對這份協議進行修改。我們今天非常高興地報告。它們已在具有約束力的條款清單中達成一致，並將很快記錄下來。我會將其轉交給理查德，以便他概述該組織的價值以及為什麼需要這樣做。

Thanks. It's Richard Steinhart. So we've done a couple of things here column that make it attractive to us. We moved out the revenue covenants that would have started to impact our cash flow early next year for about a year. So that saves us into some significant cash payments that may have been required under the original deal. And as Vimal said, the new covenants and the new revenue targets really align with our reprioritization and our new budgets. So it gives us a lot more flexibility in terms of operation for us, and we renegotiated the tranches that may allow us to take down additional capital over the next few months.

謝謝。我是理查德·史坦哈特。因此，我們在本專欄中做了一些使其對我們有吸引力的事情。我們將明年初開始影響我們現金流的收入契約取消了大約一年。因此，這使我們節省了原始交易中可能需要的大量現金付款。正如維馬爾所說，新的契約和新的收入目標確實與我們的優先順序調整和新預算相符。因此，它為我們的營運提供了更大的靈活性，並且我們重新談判了可能允許我們在未來幾個月內獲得額外資本的部分。

Very helpful. Thank you.

很有幫助。謝謝。

Robyn Karnauskas, Truist Securities

Robyn Karnauskas，Truist 證券

Good morning and thanks for taking my questions. And I guess starting with the first one, I think before you said like the FDA like to up the dose in half for these asthma trials in what kind of conversations did you have that using the 60 milligram for and 60 microgram for the for the for the asthma setting for TRANQUILITY.

早安，感謝您提出我的問題。我想從第一個開始，我想在您說 FDA 喜歡將這些氣喘試驗的劑量增加一半之前，您進行過什麼樣的對話，使用 60 毫克和 60 微克用於治療TRANQUILITY 的氣喘設定。

Second question is at home. Can you update us on your thoughts on the number of potential doses that are in the market are episodes that you're going for at home and in the hospital setting or assisted living setting, like what is now the number that we should be thinking about? And then I have a follow-up.

第二個問題是在家。您能否告訴我們您對市場上潛在劑量數量的最新想法，這些劑量是您在家中、醫院環境或輔助生活環境中購買的劑量，例如我們現在應該考慮的數量？然後我有一個後續行動。

So I'm just trying to understand Robyn, your first question. Your first question is about the dose being used in the Alzheimer's related agitation, 60 microgram in a home setting. I just wanted to make sure that I understood.

所以我只是想了解羅賓，你的第一個問題。你的第一個問題是關於阿茲海默症相關躁動症所使用的劑量，家庭環境中為 60 微克。我只是想確保我理解了。

Yes. I mentioned before you talked about how the FDA always wants to cut the dose when they go at home. And that's what you did with serenity and lower and what gives you confidence that you're using it and we wanted to have a high dose for and your trial that you did in this low data centers.

是的。我之前提到過，FDA 總是想在他們回家時減少劑量。這就是您在平靜和較低的情況下所做的事情，這讓您對自己正在使用它充滿信心，我們希望您在這個低數據中心進行的試驗有高劑量。

And as you know, in our assisted living TRANQUILITY II program, we tested two doses, 40 micrograms and 60 micrograms. 60 microgram was statistically significant and it was well tolerated and we have a very clear safety profile established. And in TRANQUILITY I also, we had the data for the 60 microgram. So that does we want to test in a home setting because it's clearly establish data efficacy and safety profile to drug.

如您所知，在我們的輔助生活 TRANQUILITY II 計劃中，我們測試了兩種劑量：40 微克和 60 微克。 60 微克具有統計意義，且耐受性良好，我們建立了非常明確的安全概況。在 TRANQUILITY I 中，我們也有 60 微克的資料。因此，我們希望在家庭環境中進行測試，因為它清楚地建立了藥物的數據功效和安全性概況。

Rob would like to add anything?

羅布想補充什麼嗎？

Yeah the enhancements to the FDA, you know, south of 60 microgram had efficacy as demonstrated in TRANQUILITY II, and therefore, that's the dose to test at home. The safety profile of 60 was consistent with being able to be dosed at home. And so we believe a successful at-home trial will be demonstrating safety consistent with what we shown on met in the TRANQUILITY II study.

是的，FDA 的改進，你知道，在 TRANQUILITY II 中證明了 60 微克以下的劑量具有功效，因此，這是在家測試的劑量。 60 的安全性與在家中服用劑量一致。因此，我們相信成功的家庭試驗將證明安全性與我們在 TRANQUILITY II 研究中所顯示的結果一致。

In terms of the number of episodes, we continue because there is no drug approved. We are what we believe one of the leaders in acute treatment of agitation in Alzheimer's related agitation so we continue to do a lot of work internally to understand the opportunity. I will invite Matt to provide a little color what our understanding is on the number of episodes Sure.

就發作次數而言，我們繼續，因為沒有藥物被批准。我們相信，我們是阿茲海默症相關躁動急性治療領域的領導者之一，因此我們繼續在內部做大量工作來了解這個機會。我會邀請馬特提供一些我們對劇集數量的理解。

Good morning, Robyn. As Phil said earlier, better than 80% of patients with Alzheimer's dementia in an at-home setting. This is where the unmet need is potentially the greatest antipsychotics are not typically used in this population are due to the side effects and benzodiazepines are not an optimal choice. So typically what's used for these patients is some type of soothing technique and these are relatively ineffective. And so we believe that the opportunity is tremendous. What we've seen in our market research is that on average, the number of episodes per month for these patients in the at-home setting is six. So the opportunity out there is pretty tremendous.

早安，羅賓。正如菲爾之前所說，超過 80% 的阿茲海默症癡呆症患者是在家中患上癡呆症的。這就是未滿足的需求可能是最大的抗精神病藥物通常不用於該族群的地方，因為副作用和苯二氮平類藥物不是最佳選擇。因此，通常對這些患者使用某種類型的舒緩技術，但這些技術相對無效。因此我們相信機會是巨大的。我們在市場研究中發現，這些患者在家中平均每月發作六次。所以那裡的機會是相當巨大的。

Would you get other variations like patients that are not completely diagnose a lot of patients are seem to have Alzheimer's, but maybe they have other kinds of dementia with that is something I read in your press release like assumed Alzheimer's that that might be upside to the opportunity.

您是否會得到其他變化，例如未完全診斷的患者，許多患者似乎患有阿茲海默症，但也許他們患有其他類型的癡呆症，這是我在您的新聞稿中讀到的內容，例如假設的阿茲海默症，這可能對機會。

And then on the financing question, maybe talk a little bit about how you're thinking about Pryor, focusing on future development for Serenity once you get the final minutes back versus TRANQUILITY versus, say, monetizing 701 like I guess you have to prioritize one given your cash position. How are you thinking about?

然後在融資問題上，也許可以談談你對Pryor 的看法，一旦你回到最後幾分鐘，專注於Serenity 的未來發展，與TRANQUILITY 相比，比方說，透過701 貨幣化，我猜你必須優先考慮一個鑑於您的現金狀況。你想怎麼樣？

Yes. And regarding the prioritization on, I think good news is that we have full clarity on both program, a program and from our two recent FDA meeting. So we have both options at our disposal to bring this drug into the home setting, TRANQUILITY, considering a very large opportunity, it makes sense to prioritize TRANQUILITY program, and that is the meeting we had in October and we are more advanced in our protocol development and like taking next steps forward with the TRANQUILITY program.

是的。關於優先順序，我認為好消息是我們對這兩個計劃、一個計劃以及我們最近兩次 FDA 會議的情況都有充分的了解。因此，我們有兩種選擇可以將這種藥物引入家庭環境，TRANQUILITY，考慮到一個非常大的機會，優先考慮 TRANQUILITY 計劃是有意義的，這就是我們 10 月份舉行的會議，我們的方案更加先進發展，並喜歡透過TRANQUILITY 計劃向前邁出下一步。

Coming back related to your question about the financing arm, we believe that we are blessed that we have multiple options for financing. In addition to equity financing on the recently, I was able to revise our tons with our strategic partner to build financial flexibility in addition, we have opportunity to be able to partner 501 program.

回到你關於融資部門的問題，我們相信我們很幸運，我們有多種融資選擇。除了最近的股權融資之外，我還能夠與我們的戰略合作夥伴一起修改我們的噸位以建立財務靈活性此外，我們還有機會能夠與 501 計劃合作。

Now we have a full clarity in Alzheimer's agitation ex US That opportunity. We have not explored because right now, we were waiting for clarity on these two programs. And in addition, as you mentioned, and Vince also mentioned that we are focusing on monetization of OnkosXcel. So depending on the business need. We can leverage one of these on options to us to extend our cash runway and get to the clinical meaningful clinical milestones for the TRANQUILITY program followed by the trinity.

現在我們完全清楚了美國以外的阿茲海默症風潮的機會。我們還沒有進行探索，因為現在我們正在等待這兩個計劃的澄清。此外，正如您所提到的，Vince 也提到我們正在專注於 OnkosXcel 的貨幣化。所以要看業務需要。我們可以利用其中一個選項來擴展我們的現金跑道，並為 TRANQUILITY 計劃達到臨床上有意義的臨床里程碑，然後是三位一體。

And so Robyn, this is Matt. I'll just take on your question about the potential label for our presumed Alzheimer's dementia. And of course, these are things that we will test in the market to see how the market will react to them. But my initial reaction is that payers who might otherwise have a prior authorization due to a confirmed diagnosis might not be able to leverage a prior authorization in that way. That's number one.

羅賓，這是馬特。我將回答你關於我們推測的阿茲海默症的潛在標籤的問題。當然，這些都是我們將在市場上測試的東西，看看市場將如何反應。但我的最初反應是，原本可能因確診而獲得事先授權的付款人可能無法以這種方式利用事先授權。這是第一名。

Number two is that what we saw in our market research on episodes, one of the things we did collect is the number of episodes per month prior to the definitive diagnosis of Alzheimer's dementia, and that was three per month. So we do know that agitation exists prior to and may actually lead to the definitive diagnosis.

第二，我們在對發作的市場研究中看到，我們收集的資訊之一是在最終診斷出阿茲海默症之前每月發作的次數，即每月 3 次。所以我們確實知道躁動早於並且實際上可能導致明確的診斷。

Great. Thank you.

偉大的。謝謝。

Yatin Suneja with Guggenheim.

亞汀蘇內賈 (Yatin Suneja) 與古根漢。

Good morning. This is Dan on for Yatin. Thanks for the update. Could you please clarify what are the safety long-term requirements for submission for the pancreatic program? And then in the next, frankly, looking at the are you planning to use that clinical site which are awaiting register misconduct? Thank you.

早安.這是雅汀的丹。感謝更新。您能否澄清一下提交胰臟計畫的安全性長期要求是什麼？接下來，坦白說，看看您是否計劃使用正在等待註冊不當行為的臨床網站？謝謝。

Regarding the long-term safety, as we have outlined that, that is a will continue to be a topic of discussion with the FDA. Our drug is acute treatment of agitation is episodic in nature, depending on what is acute and episodic and what is chronic those discussion will continue. So it's a topic that we based on our frequency of agitation we will discuss with the agency in terms of the site.

關於長期安全性，正如我們所概述的那樣，這將繼續成為 FDA 討論的話題。我們的藥物對激動的急性治療本質上是偶發性的，這取決於什麼是急性和偶發性以及什麼是慢性，這些討論將繼續。因此，我們將根據我們的鼓動頻率與該機構在網站方面討論這個主題。

We are not planning to use on that side, which you mentioned, and that was specifically site design for the ALFs set and now as we mentioned that we are moving forward with the at-home setting. So we'll be using the new sites next month.

我們不打算在這一側使用，正如您所提到的，那是專門為 ALF 場景設計的，現在正如我們所提到的，我們正在推進家庭設置。因此，我們將在下個月使用新網站。

Thank you then.

那謝謝你了。

Graig Suvannavejh, Mizuho Securities USA

Graig Suvannavejh，美國瑞穗證券

Thanks for taking my questions. And a couple if I could. Just with respect to the comments around long-term safety requirements, I'm just wondering in support have an sNDA in the AD agitation setting, what is your current expectation or and what you'll need?

感謝您回答我的問題。如果可以的話，還有一對。關於長期安全要求的評論，我只是想知道在 AD 鼓動環境中支持 sNDA，您當前的期望是什麼或您需要什麼？

I guess, and the question is beyond the 100 patient four week study, is there a current thinking that you will need additional long-term safety in order to support and sNDA momentum.

我想，問題超出了 100 名患者為期 4 週的研究範圍，目前是否認為您需要額外的長期安全性才能支持 sNDA 勢頭。

A follow-up question, if I could. Just on the current OpEx. I think it came in significantly higher than we were expecting. And given the current turnover, wondering how we should anticipate OpEx to evolve in the fourth quarter and the first half of next year thing.

如果可以的話，有一個後續問題。就目前的營運支出而言。我認為它的表現明顯高於我們的預期。考慮到目前的營業額，我們想知道第四季和明年上半年的營運支出將如何發展。

Greg, regarding the long-term safety, as I mentioned, this will be a continued topic of discussion. It will happen between now when we are initiating the home setting trial as well as at the pre-NDA meeting. There is not a drug that has been approved, which is acute treatment for agitation in Alzheimer's and is the episodic in nature. So that package will be discussed with the agency, and we'll continue to update like where we are on those discussions. Currently, we are focused on the study for that home setting a pivotal trial that we have agreed with the agency.

格雷格，關於長期安全性，正如我所提到的，這將是一個持續討論的話題。這將在我們開始家庭環境試驗以及 NDA 前會議期間發生。目前還沒有一種藥物已被批准用於治療阿茲海默症躁動症，並且本質上是間歇性的。因此，我們將與該機構討論該方案，我們將繼續更新這些討論的進展。目前，我們正專注於對該房屋的研究，並已與該機構達成一致，設定了一項關鍵試驗。

In terms of the cash run rate, I will pass it on to Richard to provide color on is what our guidance is.

就現金運行率而言，我會將其傳遞給理查德，以提供我們的指導意見。

So good morning, Greg, how are you? The results of the reprioritization will begin to impact the fourth quarter and then certainly into next year. So we will see our burn rate start to decrease. In addition, there were a couple of one-time charges in this quarter that will be repeated moving forward. So overall, we expect the burn rate to decrease next quarter and then continue into next year.

早安，格雷格，你好嗎？重新調整優先順序的結果將開始影響第四季度，然後肯定會影響明年。所以我們會看到我們的燒錢率開始下降。此外，本季還有幾項一次性費用將在今後重複出現。因此，總體而言，我們預計下個季度的燒錢率將下降，然後持續到明年。

Great. And if I could ask maybe just one follow-on J code. How how should we expect that how that might impact the trajectory of account sales.

偉大的。如果我可以問也許只是一個後續的 J 代碼。我們應該如何預期這將如何影響客戶銷售的軌跡。

And so so great. First of all, we were we were very pleased with CMS' decision to issue a permanent J-code. We do believe that this will neutralize any economic concerns that hospitals and clinics might have in putting a gummy either on formulary or providing broader use within the hospital clinic. So we look at this as a positive certainly our corporate account director team has been getting positive feedback from from either key hospitals or systems that they've been in contact with. So we feel very good about this development and do think that it alleviates of one of the barriers to two increased use.

太棒了。首先，我們對 CMS 發布永久 J 程式碼的決定感到非常高興。我們確實相信，這將消除醫院和診所將軟糖列入處方或在醫院診所內提供更廣泛使用時可能產生的任何經濟擔憂。因此，我們認為這是積極的，我們的企業客戶總監團隊已經從他們接觸過的主要醫院或系統獲得了積極的回饋。因此，我們對這一發展感到非常滿意，並且確實認為它減輕了兩次增加使用的障礙之一。

Thank you.

謝謝。

Corinne Jenkins, Goldman Sachs.

科琳·詹金斯，高盛。

I guess just a couple from us. When do you anticipate that you're going to be able to finalize these protocols and initiate the at-home study for TRANQUILITY? And based on one of your prior answers, will these all be new trial sites that you need to enroll?

我想我們當中只有幾個人。您預計什麼時候能夠完成這些協議並啟動 TRANQUILITY 的家庭研究？根據您之前的答案之一，這些都是您需要註冊的新試用網站嗎？

And then, on the TRANQUILITY point, you guided, I think, for 100 patients in that study, but it comes that you've yet to determine the primary endpoint that you'll be evaluating. So how did you come up with that guidance number of fusions required and could it evolve as you determine that critical?

然後，在 TRANQUILITY 點上，我認為您在該研究中為 100 名患者提供了指導，但您尚未確定要評估的主要終點。那麼，您是如何得出所需的聚變指導數量的？它是否可以隨著您確定的關鍵程度而變化？

So in terms of the protocol, it's under development. We had a meeting with the FDA last month. So we were expecting the meeting minutes to confirm our understanding what protocol is in progress. We are finalizing the protocol once we had input from our all our experts are at board, then we will submit a protocol to that.

所以就協議而言，它正在開發中。上個月我們與 FDA 舉行了一次會議。因此，我們期待會議紀要能夠確認我們對正在進行的協議的理解。一旦我們收到所有專家的意見，我們就會最終確定協議，然後我們將提交一份協議。

And after the protocol has been submitted, as you know, we will be in a position and within a short time after that to be able to initiate that trial. So I will and we will provide the guidance on when we think the trial can be initiated and what when first patient will be dosed. Also, we are in the process of finding how many trial sites we will open and what the recruitment rate will look like.

如您所知，在提交方案後，我們將能夠在短時間內啟動該試驗。因此，我將提供有關何時可以啟動試驗以及何時對第一位患者給藥的指導。此外，我們正在研究將開設多少個試用站點以及招募率如何。

Coming back to your question about the 100 number that was as you saw that it is designed for a safety and to collect efficacy data. So FDA and has felt that that number will be sufficient to add to our current data set we have in with Fiber I in that early patient population. So those were the drivers and decision. We ended up making what needs to be shown in our homes. I think what patient number will be relevant and what the success criteria will be in terms of demonstrating the safety.

回到你關於 100 這個數字的問題，正如你所看到的，它是為了安全性和收集功效數據而設計的。因此 FDA 認為這個數字足以添加到我們目前在早期患者群體中使用纖維 I 的數據集。這些就是驅動因素和決定。我們最終製作了需要在家中展示的東西。我認為相關的患者數量以及證明安全性的成功標準是什麼。

Rob you'd like to add anything?

羅布，你想補充什麼嗎？

Just that the protocol in development is really focused on both feasible and rapid generation. And I recognize this is a pivotal trial, but the primary aim is described safety and only about 100 patients. And they are being treated as needed with the 60 microgram dose or placebo. And so it's designed to generate placebo-controlled safety data on adverse events for the FDA to review with respect to including these in a potential labeling at home. So we're not able to say when the results will be available. However, we'll share more facts once the protocol is finalized. And of course, we expect that we'll announce when in the enrollment begins.

只是開發中的協議確實專注於可行和快速生成。我認識到這是一項關鍵試驗，但主要目的是描述安全性，並且僅涉及約 100 名患者。他們根據需要接受 60 微克劑量或安慰劑的治療。因此，它的目的是產生有關不良事件的安慰劑對照安全數據，供 FDA 審查，以將這些數據納入潛在的家庭標籤中。因此，我們無法透露何時可以得到結果。然而，一旦協議最終確定，我們將分享更多事實。當然，我們希望我們會宣布報名開始的時間。

Okay. And then as you think about this extended agreement with Oaktree and QIA., how do you think about are we taking on additional debt versus and taking capital through the equity merger?

好的。然後，當您考慮與橡樹資本和 QIA 達成的延長協議時，您如何看待我們是否承擔額外債務以及透過股權合併獲取資本？

I think always it's a delicate balance based on the business need say your current cash position, the options you have at your disposal.

我認為這始終是基於業務需求的微妙平衡，例如您當前的現金狀況和您可以使用的選擇。

The good news is that we have both equity as well potential that option in addition to as I mentioned previously, our partnering, which can be outside the US for Alzheimer's related agitation because it's opportunity in US and outside is really large. In addition, as we mentioned that we have started now more a concerted effort for the Amkor's access. So we'll leverage these assets to develop our financing strategy that is create best value for our shareholders.

好消息是，除了我之前提到的我們的合作關係之外，我們既有股權也有潛力，可以在美國以外的地區進行與阿茲海默症相關的宣傳，因為它在美國和國外的機會非常大。此外，正如我們所提到的，我們現在已經開始為 Amkor 的訪問做出更多協調一致的努力。因此，我們將利用這些資產來制定我們的融資策略，為股東創造最大價值。

Yes. Thank you.

是的。謝謝。

Sumant Kulkarni with Canaccord Genuity.

Sumant Kulkarni 與 Canaccord Genuity。

You have to so you mentioned it's easier to conduct a trial at home because of the lack of I think you used the term helicopter in the CRO, but would the burden of ensuring safety in the patient at home not be greater relative to a more marked monitored setting and how real time will feed back to the company be based on safety based events in the new Phase 3 trial.

你必須這樣做，所以你提到，由於缺乏，在家裡進行試驗更容易，我認為你在CRO 中使用了“直升機”一詞，但相對於更明顯的情況，確保患者在家安全的負擔不會更大嗎？受監控的設定以及如何即時回饋給公司將基於新的第 3 階段試驗中基於安全的事件。

So AMF setting, as you know, it is a nonmedical setting where we have conducted TRANQUILITY II and II. So the only reason you had to handicap that in a CRO is to measure the efficacy, like, you know, back measurement and safety profile. We now have in elderly patients in TRANQUILITY II and II and we will be evaluating now in the home setting. So safety will be measured as for any other drug like that is being tested in a home setting.

所以，如您所知，AMF 設置是一種非醫療設置，我們已在其中進行了 TRANQUILITY II 和 II。因此，你必須在 CRO 中限制這一點的唯一原因是衡量療效，例如，你知道，回溯測量和安全性概況。我們現在在 TRANQUILITY II 和 II 中治療老年患者，我們現在將在家庭環境中進行評估。因此，將像在家庭環境中測試的任何其他類似藥物一樣對安全性進行衡量。

And then in terms of the efficacy, it will be more collecting the efficacy data, whether there were the agitation that the patient will come given more by your caregiver. And we are developing the protocol and how we will measure that. But safety is because we have now established 60 microgram efficacy in ALF and that that was primarily the reason to come up with a design in a home setting to expand the patient access to this drug to the patient population if we get approved.

然後就療效而言，將更多地收集療效數據，是否有患者會受到您的照護人員的更多幹擾。我們正在製定協議以及我們將如何衡量它。但安全性是因為我們現在已經確定了 60 微克對 ALF 的療效，這也是我們在家庭環境中提出一種設計的主要原因，如果我們獲得批准，可以將這種藥物的患者範圍擴大到患者群體。

And then we understand that the FDA could only opine on the data that you have in hand with TRANQUILITY II as part of a review, but did did the agency specifically say that only this one additional trial would be required to submit an sNDA or you won't require more efficacy trials.

然後我們了解到，作為審查的一部分，FDA 只能對您掌握的 TRANQUILITY II 數據發表意見，但該機構是否明確表示，僅需要這一項額外試驗來提交 sNDA，否則您將獲勝不需要更多的功效試驗。

I think this is our alignment, and this is the recommendation of the FDA that it is a 100 patient home setting trial with the efficacy assessment as well as collecting a safety assessment and collecting efficacy using a caregiver because that is the best possible design executable in a home setting some. So that's our clear understanding and company believe that there will be one more trial that we have outlined today will be required for a potential submission of this sNDA.

我認為這是我們的一致性，這是 FDA 的建議，這是一項 100 名患者家庭環境試驗，包括功效評估、安全評估和使用護理人員收集功效，因為這是最佳的可行設計家中設置一些。因此，這是我們的明確理解，公司相信，可能需要提交我們今天概述的另一項試驗才能提交此 sNDA。

And last one, I'll squeeze one in is this trial going to have a Part one In part two like SERENITY trial?

最後一個，我要塞進去一個，這個試驗是否會有像 SERENITY 試驗一樣的第一部分和第二部分？

No, this will not have Part one and Part two because as you know, 60 microgram has efficacy has been established, we believe, in TRANQUILITY I and then further confirmed in our TRANQUILITY II trial. So I in SERENITY, we were trying to determine a lower dose than the approved dose and time to see it will be efficacious and safe. And that's part of the reason it was designed as a two-part study and TRANQUILITY, there was no need to design that as a two-part story.

不，這不會有第一部分和第二部分，因為如您所知，我們相信，60 微克的功效已在 TRANQUILITY I 中得到證實，然後在我們的 TRANQUILITY II 試驗中得到進一步證實。因此，我在 SERENITY 中嘗試確定低於批准劑量的劑量和時間，以確保其有效且安全。這就是它被設計為兩部分研究和《寧靜》的部分原因，沒有必要將其設計為兩部分故事。

Thanks for the clarification.

感謝您的澄清。

Thank you. We have reached the end of our question-and-answer session. I would now like to turn the floor back over to Dr. Mehta for closing remarks.

謝謝。我們的問答環節已經結束。現在我想請梅塔博士發表閉幕詞。

Thank you, everyone, for joining us today and for your continued interest in BioXcel Therapeutics. Have a great day. Thank you.

感謝大家今天加入我們並感謝您對 BioXcel Therapeutics 的持續關注。祝你有美好的一天。謝謝。

This does conclude today's teleconference. We appreciate your participation. You may disconnect your lines at this time and enjoy the rest of your day.

今天的電話會議到此結束。我們感謝您的參與。此時您可以斷開線路並享受剩下的一天。