BioXcel Therapeutics Inc (BTAI) 2024 Q3 法說會逐字稿

Good morning, and welcome to the BioXcel Therapeutics Third-Quarter 2024 Earnings Conference Call. At this time, all participants are in a listen-only mode. (Operator Instructions) After the formal remarks, there will be a question-and-answer session. (Operator Instructions)

早安，歡迎參加 BioXcel Therapeutics 2024 年第三季財報電話會議。此時，所有參與者都處於只聽模式。 （操作員說明） 正式發言後，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。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 could affect future results are detailed in the company's quarterly report on Form 10-Q for the quarterly period ended June 30, 2024, which can be found at www.bioxceltherapeutics.com or on www.sec.gov and which will be updated in its quarterly report on Form 10-Q for the quarterly period ended September 30, 2024.

可能影響未來業績的風險因素詳見公司截至 2024 年 6 月 30 日的季度報告 10-Q 表格，該報告可在 www.bioxceltherapeutics.com 或 www.sec.gov 上找到，並將在更新了截至2024年9 月30 日的季度報告中的10-Q 表格。

As a reminder, today's call is being recorded. Speaking on today's call are Dr. Vimal Mehta, Chief Executive Officer; and Richard Steinhart, Chief Financial Officer. They will be joining the Q&A session by Dr. Vince O'Neill, Chief of Product Development and Medical Officer; Dr. Frank Yocca, Chief Scientific Officer; and Dr. Rob Risinger, Chief Medical Officer of Neuroscience.

提醒一下，今天的通話正在錄音。執行長 Vimal Mehta 博士在今天的電話會議上發言。和首席財務官理查德斯坦哈特。他們將參加由產品開發主管兼醫療官 Vince O'Neill 博士主持的問答環節； Frank Yocca 博士，首席科學官； Rob Risinger 博士，神經科學首席醫學官。

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

現在我很高興將電話轉給梅塔醫生。請繼續。

Thank you, operator. Good morning, everyone, and thank you for joining us. This past Tuesday, we announced important milestones with our two pivotal Phase 3 trials with BXCL501. These trials are for agitation associated with bipolar disorder or schizophrenia in the home setting and for agitation associated with Alzheimer's dementia.

謝謝你，接線生。大家早安，感謝您加入我們。上週二，我們宣布了 BXCL501 的兩項關鍵 3 期試驗的重要里程碑。這些試驗針對家庭環境中與躁鬱症或精神分裂症相關的躁動以及與阿茲海默症相關的躁動。

We believe BXCL501 could potentially be a much needed acute treatment option for the millions of patients, who do not have FDA approved therapy for these challenging conditions. Our goal is to transform the treatment paradigm.

我們相信 BXCL501 可能成為數百萬患者急需的急性治療選擇，這些患者沒有獲得 FDA 批准的針對這些挑戰性病症的治療方法。我們的目標是改變治療模式。

Starting with our SERENITY at home trial to evaluate BXCL501 for acute treatment of bipolar or schizophrenia-related agitation, we believe this indication represents a near-term growth opportunity and is our immediate focus area.

從我們的 SERENITY 家庭試驗開始，評估 BXCL501 對雙相情感障礙或精神分裂症相關躁動的急性治療，我們相信這一適應症代表了近期的成長機會，也是我們當前的重點領域。

We designed this study leveraging our substantial clinical experience with BXCL501 conducting 10 double-blinded placebo control trials. We are particularly excited to advance this trial, because it marks the first evaluation of BXCL501 in the home setting.

我們利用 BXCL501 進行 10 項雙盲安慰劑對照試驗的豐富臨床經驗設計了這項研究。我們對推進這項試驗感到特別興奮，因為它標誌著 BXCL501 在家庭環境中的首次評估。

The first patient was randomized late last week and we look forward to advancing enrollment. Trial duration is expected to be nine to 12 months from the September initiation as we previously communicated. We are also continuing our planning for our TRANQUILITY In-Care trial to evaluate BXCL501 as a potential acute treatment for Alzheimer's related agitation. This represents a much larger, longer term growth opportunity.

上週晚些時候，第一位患者被隨機分配，我們期待提前入組。正如我們之前溝通的那樣，從 9 月開始，試用期預計為 9 至 12 個月。我們也持續規劃 TRANQUILITY In-Care 試驗，以評估 BXCL501 作為阿茲海默症相關躁動的潛在急性治療方法。這代表著一個更大、更長期的成長機會。

We recently received feedback from the FDA on our proposed protocol for the trial, which will be our second pivotal Phase 3 trial for this condition and which largely mirrors our TRANQUILITY 2 Phase 3 study. In addition to acute agitation, we are pleased with the potential chronic treatment opportunities with BXCL501 through externally funded studies currently being conducted by leading academic research institutions.

我們最近收到了 FDA 對我們建議的試驗方案的回饋，這將是我們針對這種情況的第二個關鍵 3 期試驗，很大程度上反映了我們的 TRANQUILITY 2 3 期研究。除了急性躁動之外，我們對 BXCL501 透過領先學術研究機構目前正在進行的外部資助研究的潛在慢性治療機會感到高興。

We recently announced a Department of Defense grant to fund a Phase 2a efficacy and safety study of BXCL501 for acute stress disorder at the University of North Carolina. This marks the second externally funded stress-related trial of BXCL501. Just to remind everyone, we discovered BXCL501 using our AI platform and are pleased to see further validation of its broad therapeutic potential.

我們最近宣布獲得國防部撥款，資助北卡羅來納大學進行的 BXCL501 治療急性壓力症候群的 2a 期療效和安全性研究。這標誌著 BXCL501 的第二次外部資助的壓力相關試驗。只是提醒大家，我們使用我們的 AI 平台發現了 BXCL501，並很高興看到其廣泛治療潛力的進一步驗證。

While focusing on clinical development, we are continuing to maintain our approved product, IGALMI, in the market with minimal resources. We are committed to making this drug available to patients, while maintaining our brand equity. To support our trials with the goal of reaching data readout, we are working to strengthen our balance sheet. At the same time, we continue evaluating strategic financing alternatives.

在專注於臨床開發的同時，我們繼續以最少的資源在市場上維持我們已批准的產品 IGALMI。我們致力於向患者提供這種藥物，同時保持我們的品牌資產。為了支持我們的試驗以達到數據讀出的目標，我們正在努力加強我們的資產負債表。同時，我們持續評估策略融資方案。

In summary, we are pleased with the progress of our SERENITY and TRANQUILITY programs. We believe we have attractive opportunities to address substantial unmet medical needs, while expanding the market potential for BXCL501. This belief is reinforced by a growing intellectual property portfolio that provides long-term patent protection from which we aim to build a leadership position in the treatment of agitation.

總之，我們對 SERENITY 和 TRANQUILITY 計劃的進展感到滿意。我們相信，我們有誘人的機會來解決大量未滿足的醫療需求，同時擴大 BXCL501 的市場潛力。不斷增長的智慧財產權組合強化了這一信念，這些智慧財產權組合提供長期專利保護，我們的目標是在躁動治療領域建立領先地位。

I would now like to turn the call over to Rich, who will review our financial results for the third quarter of 2024. Rich?

我現在想將電話轉給 Rich，他將審查我們 2024 年第三季的財務表現。

Thank you, Vimal. Net revenue for IGALMI was $214,000 for the third quarter of 2024, compared to $341,000 for the same period in 2023. The decrease in sales was primarily due to the timing of reorders from existing customers.

謝謝你，維馬爾。 IGALMI 2024 年第三季的淨收入為 214,000 美元，而 2023 年同期為 341,000 美元。

However, for context, it's important to point out that net revenue of $1.9 million for the nine months ended September 30, 2024, represents an 89% increase from $1 million in the same period in 2023. The increase in new customer acquisitions and increased sales activity reflects rising utilization.

然而，就背景而言，需要指出的是，截至2024 年9 月30 日的九個月淨收入為190 萬美元，比2023 年同期的100 萬美元增長了89%。額的增加活動反映出利用率的上升。

Cost of goods sold for the three months ended September 30, 2024 and 2023 were $1.2 million $512,000 respectively. Cost of goods sold is related to the cost to produce, package, and deliver IGALMI to customers, as well as costs related to excess or obsolete inventory.

截至 2024 年 9 月 30 日和 2023 年的三個月銷售成本分別為 120 萬美元 512,000 美元。銷售成本與生產、包裝和向客戶交付 IGALMI 的成本以及與過剩或過時庫存相關的成本有關。

The increase in cost of goods sold for the three months ended September 30, 2024, is a result of higher non-cash charges for reserves for excess or obsolete inventory, compared to the same period in 2023. Charges for reserves for excess or obsolete inventory were $1.2 million and $495,000 for the three months ended September 30, 2024 and 2023 respectively.

截至 2024 年 9 月 30 日的三個月銷售成本增加，是由於與 2023 年同期相比，過剩或過時庫存儲備的非現金費用較高。和2023年9月30日的三個月分別為120萬美元和495,000美元。

Research and development expenses were $5.1 million for the third quarter of the 2024, compared to $19.6 million for the same period in 2023. The decreased expenses were primarily due to decreased clinical trial expenses, professional fees, as well as personnel and related costs resulting from the company's reprioritization in August of 2023 and reduction in force in May 2024.

2024 年第三季的研發費用為 510 萬美元，而 2023 年同期為 1,960 萬美元。順序，並於2024 年5 月減少力量。

Selling, general, and administrative expenses were $7.7 million for the third quarter of 2024, compared to $24.3 million for the same period in 2023. The lower expenses were primarily due to decreased personnel and related costs, resulting from the company's reprioritization in August 2023 and further reduction in force in May 2024, as well as decreased professional fees and commercial expenses.

2024 年第三季的銷售、一般和管理費用為 770 萬美元，而 2023 年同期為 2,430 萬美元。並減少專業費用和商業費用。

BioXcel Therapeutics had a net loss of $13.7 million for the third quarter of 2024, compared to a net loss of $50.5 million for the same period in 2023. Company used $16.3 million in operating cash during the third quarter of 2024. Cash and cash equivalents totaled $40.4 million as of September 30, 2024.

BioXcel Therapeutics 2024年第三季的淨虧損為1,370萬美元，而2023年同期的淨虧損為5,050萬美元。年9 月30 日為4040 萬美元。

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. Operator?

謝謝你，里奇。我們現在要開始提問。操作員？

(Operator Instructions)

（操作員說明）

Alec Stranahan, Bank of America.

亞歷克·斯特拉納漢，美國銀行。

Okay, great. Hey guys, thanks for taking our questions. Just two quick ones from us. I guess first, how has the launch of the SERENITY study gone so far? Maybe walk us through the process of getting the first patient on the study and whether you expect the rate of enrollment could be similar or maybe accelerate for the other 200 or so patients on the study? And then I've got a follow-up.

好的，太好了。嘿夥計們，感謝您提出我們的問題。我們只有兩個快的。我想首先，SERENITY 研究的啟動到目前為止進展如何？也許可以引導我們完成讓第一個患者參與研究的過程，以及您是否預計其他 200 名左右患者參與研究的入組率可能會相似或可能會加快？然後我有後續行動。

Sure. Good morning, Alec. This is Vince here. So I'll start that and then I'll hand over to Rob to give a bit more detail if he feels I'm missing anything. So essentially, as you know, the study has kicked off, the first patient randomized relatively recently. We're in the ramp up phase right now. That will be totally typical for a study like this. I will mention we're coming into the holiday season, so that would typically involve a little bit of a slowdown at the sites, but then we would typically see a rebound in the January timeframe. So this is our approach to the study and what we've seen so far is absolutely standard.

當然。早安，亞歷克。這是文斯。所以我會開始，然後我會交給羅布（Rob），如果他覺得我遺漏了什麼，我會提供更多細節。所以本質上，如你所知，這項研究已經開始，第一位患者是最近隨機分組的。我們現在正處於加速階段。對於這樣的研究來說，這將是完全典型的。我要提到的是，我們即將進入假期季節，因此網站通常會出現一些放緩，但隨後我們通常會在一月份的時間範圍內看到反彈。這就是我們的研究方法，到目前為止我們所看到的絕對是標準的。

I'll just mention lastly, we have a large number of sites, so 200 patients and I think the final number is 26 active sites. So that's a large number of sites for the number of patients or target enrollments. So we're very comfortable with the timeline and I'll just state it again, anticipated nine to 12 months.

最後我要提一下，我們有大量的站點，所以有 200 名患者，我認為最終的數量是 26 個活躍站點。因此，對於患者數量或目標註冊人數而言，網站數量很大。因此，我們對時間表非常滿意，我再次聲明一下，預計為 9 到 12 個月。

Anything to add to that, Rob?

羅布，還有什麼要補充的嗎？

No, we're right on track and the ramp up is occurring at the moment. So we're tracking and rolling this out across multiple sites and coordinating all of these activities, so it's truly integrated across sites.

不，我們正走在正軌上，目前正在加速推進。因此，我們正在跨多個站點追蹤和推出這一點，並協調所有這些活動，以便它真正跨站點整合。

Okay, that makes sense. And then on the randomization, could you just remind us if rescue medications are allowed, especially thinking about the placebo arm and whether this might actually be a helpful data point that you may be measuring over the 12-week study period? Thanks.

好吧，這是有道理的。然後關於隨機化，您能否提醒我們是否允許使用救援藥物，特別是考慮安慰劑組，以及這是否實際上是您在 12 週研究期間測量的有用數據點？謝謝。

Yes, rescue medications are allowed in the trial. Patients may receive a rescue if necessary. And as you said, half of, literally one-half of the patients, 100 versus 100, will be receiving placebo. So it's a sort of safety measure. And yes, we are tracking that as a sort of surrogate of inefficacy, we'll call it, an indicator of inefficacy for those patients on placebo.

是的，試驗中允許使用救援藥物。如有必要，患者可以接受搶救。正如你所說，一半，實際上是一半的患者，100 對 100，將接受安慰劑。所以這是一種安全措施。是的，我們正在追蹤它，作為一種無效的替代指標，我們稱之為安慰劑治療患者無效的指標。

Got it. Thanks for the color.

知道了。謝謝你的顏色。

(Operator Instructions)

（操作員說明）

Graig Suvannavejh, Mizuho Securities.

Graig Suvannavejh，瑞穗證券。

Hey, good morning. Thanks for taking my questions. I've got two; first, just I might have missed this earlier, but on the TRANQUILITY trial protocol, what was the specific feedback that you got from FDA? If you could provide any color on that?

嘿，早安。感謝您回答我的問題。我有兩個；首先，我可能之前錯過了這一點，但是關於 TRANQUILITY 試驗方案，您從 FDA 得到的具體回饋是什麼？如果你能提供任何顏色嗎？

And then secondly, was just hoping if you could provide an update on where you are with the cash runway and optionality or options that you are considering with regards to funding the company? Thanks.

其次，我只是希望您能否提供有關現金跑道以及您正在考慮為公司提供資金的選擇的最新情況？謝謝。

Sure. Good morning, Greg. This is Vince. I'll take the first part of that question. So, I mean, we don't typically give blow-by-blow details of FDA comments. What I can say, though, very comfortable in saying this, the feedback was direct, actionable, and really very concise. So, we're actually very happy with the feedback we received. Did you want to handle second part, Rich?

當然。早安，格雷格。這是文斯。我將回答這個問題的第一部分。所以，我的意思是，我們通常不會提供 FDA 評論的詳細細節。不過，我可以說的是，我很舒服地說出這一點，回饋是直接的、可操作的，而且非常簡潔。所以，我們實際上對收到的回饋感到非常滿意。里奇，你想拍第二部嗎？

I will handle it. Greg, did you have any more feedback you needed on the FDA feedback because we have already outlined that what that trial design is and what are the key elements of the design of the trial. Is there anything else we can embellish for you or that's good?

我會處理的。 Greg，您對 FDA 的回饋還有什麼需要的嗎？還有什麼我們可以為您修飾或有什麼好處嗎？

Thank you very much. That's very good.

非常感謝。那很好。

Sure, sure. Regarding the strengthening our balance sheet, as in my prepared remark, we are working on all options that we have previously outlined. And we work with our strategic partner also, so that we can get to the data readout. And that's the company's key focus, how to get to the data readout, and strengthen the balance sheet.

當然，當然。關於加強我們的資產負債表，正如我準備好的發言中所述，我們正在研究之前概述的所有選項。我們也與我們的策略合作夥伴合作，以便我們能夠讀取資料。這是公司的重點，如何讀取數據並加強資產負債表。

Maybe just a follow-up here with the trial underway of sorts. Can you provide color as to whether the current financing or cash to have is efficient to take the trial to completion again my apologies if I made [mistake] before?

也許這只是正在進行的審判的後續行動。您能否提供目前的融資或現金是否足以再次完成試驗的信息，如果我之前犯了[錯誤]，我深表歉意？

We have not provided any guidance Graig, but we initiated the trial with a view that we want to get to the data readout and work with all stakeholders, so that we can get to the data readout, which is not that far off from where we are, and we initiated this trial in September, and our guidance is nine to 12 months.

我們沒有提供任何指導 Graig，但我們啟動試驗的目的是希望能夠讀取數據並與所有利益相關者合作，以便我們能夠讀取數據，這離我們的目標並不遙遠。啟動了這項試驗，我們的指導期限是9 到12 個月。

Sumant Kulkarni, Canaccord Genuity

蘇曼特‧庫爾卡尼 (Sumant Kulkarni)，Canaccord Genuity

Good morning. Thanks for taking my questions. I have two: one financial and one trial related. The financial one is how would you characterize your latest negotiations with your large investors on your ability to keep financial covenants at bay?

早安.感謝您回答我的問題。我有兩份：一份是財務相關的，一份是與審判相關的。財務問題是，您如何描述與大型投資者就您遵守財務契約的能力進行的最新談判？

And second, how important is the successful completion of SERENITY At-Home to the company in terms of that trial's ability to potentially unlock partnership opportunities for the larger at-home agitation market?

其次，就該試驗能否為更大的家庭攪拌市場釋放合作機會而言，成功完成 SERENITY At-Home 對公司有多重要？

Hi, good morning, Suman. This is Vimal. As I mentioned that we are continuing speaking with all our stakeholders, including our strategic partners. And as you know, they've been very collaborative, and we are very pleased with that. We are continuing on that path and focus continues to be that we can strengthen the balance sheet at the same time we can get to the data readout.

嗨，早上好，蘇曼。這是維馬爾。正如我所提到的，我們正在繼續與所有利害關係人進行對話，包括我們的策略合作夥伴。如您所知，他們一直非常合作，我們對此感到非常滿意。我們將繼續沿著這條道路前進，重點仍然是我們可以在獲得數據讀數的同時加強資產負債表。

And for the SERENITY, coming back to your question regarding the SERENITY At-Home partnership, now if we think about the company, we are way advanced than where we were last year. We already have SERENITY At-Home trial in progress and we're expecting the data readout like you know next year.

對於 SERENITY，回到你關於 SERENITY At-Home 合作夥伴關係的問題，現在如果我們考慮一下公司，我們比去年先進得多。我們已經進行了 SERENITY At-Home 試驗，預計明年會出現如您所知的數據讀數。

The TRANQUILITY In-Care which is our second Phase 3 trial, which mirrors the TRANQUILITY 2, where we had positive data in efficacy and safety, so that is ready to start.

TRANQUILITY In-Care 是我們的第二個 3 期試驗，它與 TRANQUILITY 2 相似，我們在有效性和安全性方面獲得了積極的數據，因此已經準備好開始。

So company from a clinical trial perspective and moving these trial forward, which can be huge value drivers for all stakeholders like particularly patients, caregivers, health care providers and all our other stakeholders. We are pursuing every path that will allow us to maximize the value for our shareholders and that continues to be the focus of the company.

因此，公司從臨床試驗的角度出發並推動這些試驗，這對所有利害關係人（尤其是患者、照護者、醫療保健提供者和所有其他利害關係人）來說可能是巨大的價值驅動力。我們正在追求每一條能夠為股東創造最大價值的道路，這仍然是公司關注的焦點。

Samir Devani, Rx Security.

Samir Devani，Rx 安全。

Hi, guys. Thanks for taking my questions. I guess the first one is just on, I think last time we spoke, you mentioned that the TRANQUILITY In-Care trial would have a cohort of 20 patients in the At-Home setting. So I just wanted to confirm following the FDA feedback whether that was still the case. I guess that's question one.

嗨，大家好。感謝您回答我的問題。我想第一個剛開始，我想上次我們交談時，您提到 TRANQUILITY In-Care 試驗將在家庭環境中進行 20 名患者的隊列。所以我只是想根據 FDA 的回饋確認情況是否仍然如此。我想這是問題一。

Yes, hi there. This is Vince here. So yes, at the end of the day after discussion internally, we took that pilot out of the protocol. So currently that's not part.

是的，你好。這是文斯。所以，是的，在內部討論結束後，我們將該試點計畫從協議中刪除了。所以目前這還不是一部分。

Okay. And then I guess the second question is just really on BXCL701 and whether you've had made any progress in terms of options for progressing that product? Thanks very much.

好的。然後我想第二個問題實際上是關於 BXCL701 的，以及您在改進產品的選項方面是否取得了任何進展？非常感謝。

Sure, so I can give the following update. So we're still in the process, I think as I may be mentioned on the last quarterly, probably a longer process and at least I personally had anticipated, but what it's still within that process working with our firm, who has expertise in this area with potential partners, diligence, room activities, et cetera. So it's still an ongoing process.

當然，所以我可以提供以下更新。因此，我們仍在這個過程中，我想正如我在上個季度可能提到的那樣，可能是一個更長的過程，至少我個人已經預料到了，但在這個過程中仍然與我們公司合作，我們在這方面擁有專業知識有潛在合作夥伴的區域、勤奮、房間活動等等。所以這仍然是一個持續的過程。

Great, and maybe if I could just sneak one more in. In terms of the stock write-down, could you just confirm that, or give us an indication of what sort of inventory you're still holding and what may be at risk going forward, thanks.

太好了，也許我可以再偷偷放進一份。

Sure, this is Rich, thanks. I think we're in pretty good shape now with that write-down. We should have enough product to sustain us for the next couple of years. And remember, I think this is a non-cash charge, and if sales pick up, we'll have plenty of inventory to sell with this. It's just looking at where we are today and projecting out. So I think we're in pretty good shape with inventory.

當然，這是里奇，謝謝。我認為，透過減記，我們現在的狀況非常好。我們應該有足夠的產品來維持未來幾年的生活。請記住，我認為這是一項非現金費用，如果銷量回升，我們將有大量庫存可供出售。它只是著眼於我們今天所處的位置並進行預測。所以我認為我們的庫存狀況非常好。

Thanks, Richard.

謝謝，理查。

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

謝謝。我們的問答環節已經結束。我想請大家再發表進一步的結論。

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

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。

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

謝謝。今天的電話會議和網路廣播到此結束。此時您可以斷開線路並度過美好的一天。我們感謝您今天的參與。