BioXcel Therapeutics Inc (BTAI) 2024 Q2 法說會逐字稿

Good morning and welcome to the BioXcel Therapeutics second quarter 2024 earnings conference call. At this time, all participants are in a listen only mode. (Operator Instructions) Just to remind everyone, certain matters discussed in today's conference call and our 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.

早安，歡迎參加 BioXcel Therapeutics 2024 年第二季財報電話會議。此時，所有參與者都處於只聽模式。 （操作員說明）謹提醒大家，今天的電話會議中討論的某些事項以及我們可能對提出的問題做出的回答均為前瞻性陳述，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的。

Actual results could differ materially from those anticipated in these forward-looking statements. Risk factors that may affect future results are detailed in the company's Annual Report on Form 10-K for the year ended December 31, 2023, which can be found at www.biocceltherapeutics.com, or on www.sec.gov, and which will be updated in its Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2024. As a reminder, today's call is being recorded.

實際結果可能與這些前瞻性陳述中的預期有重大差異。可能影響未來業績的風險因素詳見該公司截至 2023 年 12 月 31 日止年度的 10-K 表格年度報告，該報告可在 www.biocceltherapeutics.com 或 www.sec.gov 上找到，該報告將在將在截至2024 年6 月30 日的季度報告中更新10-Q 表格。

Speaking on today's call are Dr. Vimal Mehta, Chief Executive Officer; Dr. Vince, O'Neill, Chief of Product Development and Medical Officer; and Richard Steinhart, Chief Financial Officer. They will be joined in the question-and-answer session by Dr. Frank Yocca, Chief Scientific Officer; Matt Wiley, Chief Commercial Officer; and Dr. Rob Risinger, Chief Medical Officer of Neuroscience. It is now my pleasure to turn the call over to Dr. Mehta. Please go ahead.

執行長 Vimal Mehta 博士在今天的電話會議上發言。 Vince, O'Neill 博士，產品開發主管兼醫療官；和首席財務官理查德斯坦哈特。首席科學官 Frank Yocca 博士將與他們一起參加問答環節；馬特威利 (Matt Wiley)，商務長； Rob Risinger 博士，神經科學首席醫學官。現在我很高興將電話轉給梅塔醫生。請繼續。

Thank you, operator. Good morning, and thank you for joining us today. At BioXcel Therapeutics, we are focused on expanding our lead asset BXCL501 into the home setting for bipolar and schizophrenia related agitation and into a new indication for Alzheimer's related agitation. We are extremely passionate and motivated to bring this much-needed therapeutic option to millions of patients who are struggling with episodes of agitation.

謝謝你，接線生。早安，感謝您今天加入我們。在 BioXcel Therapeutics，我們致力於將我們的主導資產 BXCL501 擴展到治療雙相情感障礙和精神分裂症相關躁動的家庭環境，以及治療阿茲海默症相關躁動的新適應症。我們非常熱情和積極地為數百萬患有躁動發作的患者提供這種急需的治療選擇。

We believe BXCL501 offers a compelling value proposition, and we look forward to advancing the development of our two clinical programs. At this time, we are delighted to reach an important juncture with the SERENITY program as we are finalizing development plans for BXCL501 in the at home setting, where the majority of agitation episodes occur.

我們相信 BXCL501 提供了令人信服的價值主張，我們期待著推進我們兩個臨床項目的開發。目前，我們很高興達到 SERENITY 計劃的一個重要節點，因為我們正在家庭環境中最終確定 BXCL501 的開發計劃，而大多數躁動發作都是在家庭環境中發生的。

We truly believe that this is a much-needed treatment option for patients, caregivers and healthcare providers as there are no FDA-approved therapies for agitation in the home setting. The SERENITY At-Home trial could represent a near-term value-creation opportunity to potentially expand beyond the institutional setting where IGALMI is currently marketed.

我們堅信，這是患者、照護者和醫療保健提供者急需的治療選擇，因為目前還沒有 FDA 批准的家庭躁動治療方法。 SERENITY At-Home 試驗可能代表一個近期價值創造機會，有可能擴展到 IGALMI 目前行銷的機構環境之外。

Our TRANQUILITY program plans with BXCL501 for Alzheimer's associated agitation are also progressing. This program could address a significant unmet medical need, and we are pleased to have received breakthrough therapy designation from the FDA for this indication.

我們針對阿茲海默症相關躁動症的 BXCL501 TRANQUILITY 計畫也正在取得進展。該計劃可以解決重大的未滿足的醫療需求，我們很高興獲得 FDA 針對該適應症的突破性治療指定。

We believe this program could represent a larger, longer term growth opportunity to potentially address an estimated 100 million annual episodes of Alzheimer's associated agitation. Vince will share updates on the TRANQUILITY and IGALMI program shortly.

我們相信，該計劃可能代表一個更大、更長期的成長機會，有可能解決每年約 1 億例阿茲海默症相關躁動的問題。 Vince 很快就會分享 TRANQUILITY 和 IGALMI 計劃的最新情況。

Turning to IGALMI, we have seen growing adoption among psychiatric care clinic and behavioral health facility. This market segment represents a community adjacent opportunity characterized by strong product freight and lower administrative barriers.

談到 IGALMI，我們發現精神科護理診所和行為健康機構越來越多地採用 IGALMI。該細分市場代表了社區鄰近的機會，其特點是產品貨運強勁且行政壁壘較低。

Given our small field commercial footprint, we believe targeting this market sector positions IGALMI for continued growth and scalability. This could also serve as a bridge to the home setting to the at-home setting, if approved. We believe this approach coupled with substantial patent protection that extends to 2043 could enable us to fully realize the potential of IGALMI.

鑑於我們的現場商業足跡較小，我們相信瞄準該市場領域將使 IGALMI 能夠實現持續成長和可擴展性。如果獲得批准，這也可以作為從家庭環境到家庭環境的橋樑。我們相信，這種方法加上持續到 2043 年的大量專利保護可以使我們充分發揮 IGALMI 的潛力。

Finally, we remain focused on strengthening our balance sheet to support our late-stage clinical trials with the goal of reaching data readouts. We are also evaluating strategic financing alternatives such as royalty monetization, OnkosXcel monetization as well as strategic partnerships.

最後，我們仍然專注於加強我們的資產負債表，以支持我們的後期臨床試驗，以實現數據讀出的目標。我們也正在評估策略性融資替代方案，例如版稅貨幣化、OnkosXcel 貨幣化以及策略合作夥伴關係。

In summary, plans for our core clinical programs are advancing, our market access strategy with IGALMI is progressing, and we are pleased with growing patent portfolio.

總而言之，我們的核心臨床項目計劃正在推進，我們與 IGALMI 的市場准入策略正在取得進展，我們對不斷增長的專利組合感到高興。

With that, I will turn the call over to Vince.

這樣，我會將電話轉給文斯。

Thank you, Vimal. So as Vimal announced, we're pleased to be preparing for initiation of our SERENITY At-Home trial, which as a reminder, is designed to be a double blind placebo controlled multi-center study to evaluate the safety and efficacy of the 120 microgram dose of BXCL501 over a 12-week period. The primary objective will be safety with efficacy measures as exploratory endpoints.

謝謝你，維馬爾。因此，正如 Vimal 所宣布的那樣，我們很高興準備啟動 SERENITY At-Home 試驗，提醒一下，該試驗旨在成為一項雙盲安慰劑對照多中心研究，以評估 120 微克的安全性和有效性12 週內BXCL501 的劑量。主要目標是安全性和功效測量作為探索性終點。

We recently received feedback on the trial protocol from the FDA. We've completed the CMC work we believe is sufficient to support the use of child proof pouches in the home setting and have finally defined the final statement of work in clinical site selection with our CRO, all preparing us for trial initiation.

我們最近收到了 FDA 對試驗方案的回饋。我們已經完成了 CMC 工作，我們相信這足以支持在家庭環境中使用兒童安全袋，並最終與我們的 CRO 一起確定了臨床地點選擇的最終工作聲明，所有這些都為我們啟動試驗做好了準備。

Plans for our TRANQUILITY program are also advancing with a focus on our tranquility In-Care trial. The proposed trial design is substantially similar to TRANQUILITY II. As a reminder, TRANQUILITY In-Care is a double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of a 60 microgram dose of 501 over a 12-week period. The primary endpoint is expected to be the change in PEC score at two hours post first stores. The TRANQUILITY In-Care protocol is in final form, and we're planning to submit it to the agency.

我們的 TRANQUILITY 計劃也在推進，重點是我們的 TRANQUILITY In-Care 試驗。建議的試驗設計與 TRANQUILITY II 基本相似。提醒一下，TRANQUILITY In-Care 是一項雙盲、安慰劑對照、多中心研究，旨在評估 60 微克劑量的 501 在 12 週內的有效性和安全性。主要終點預計是首次儲存後兩小時 PEC 評分的變化。 TRANQUILITY In-Care 協議已定稿，我們計劃將其提交給該機構。

Turning to our PMR study with IGALMI, we were pleased to have reported positive top line results, evaluating PRN or as needed treatment of IGALMI. The study achieved its objective and demonstrated no evidence of tachyphylaxis tolerance or withdrawal with the 180 microgram dose, the highest approved dose.

談到我們與 IGALMI 進行的 PMR 研究，我們很高興報告了積極的頂線結果，評估了 PRN 或根據需要進行 IGALMI 治療。該研究實現了其目標，並且沒有證據表明 180 微克劑量（批准的最高劑量）會導致快速耐受或戒斷。

Although the study was not statistically powered to evaluate repeat dose efficacy, a reduction in agitation was observed for each episode occurring during the seven-day study period and no serious adverse events were reported following treatment.

儘管該研究沒有統計學意義來評估重複劑量的療效，但在 7 天的研究期間觀察到每次發作的躁動有所減少，且治療後沒有報告嚴重的不良事件。

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

我現在想把電話轉給里奇，他將審查我們第二季的財務表現。

Thank you. Vince. Net revenue from of IGALMI was $1.1 million for the second quarter of 2024, compared to $457,000 for the same period in 2023. This represented 141% increase. Sequential quarterly revenue increased 90% in Q2 2024 from the first quarter of 2024. The growth in revenue for both periods was primarily driven by an increase in contracting with psychiatric care clinics and behavioral health facilities.

謝謝。文斯. IGALMI 2024 年第二季的淨收入為 110 萬美元，而 2023 年同期為 457,000 美元。 2024 年第二季的季度收入較 2024 年第一季成長了 90%。

Research and development expenses were $8 million for the second quarter of 2024, compared to $27 million for the same period in 2023. The decreased expenses were primarily attributable to the wind-down of the SERENITY III and TRANQUILITY II and TRANQUILITY III trials as well as decreased professional fees, personnel and related costs.

2024 年第二季的研發費用為 800 萬美元，而 2023 年同期為 2,700 萬美元。

Selling, general and administrative expenses were $9.5 million for the second quarter of 2024, compared to $25.9 million for the same period in 2023. The expense reduction was primarily attributable to a decrease in personnel and costs associated with the commercialization of IGALMI compared to the second quarter of 2023, driven by our reprioritization.

2024 年第二季的銷售、一般和管理費用為 950 萬美元，而 2023 年同期為 2590 萬美元。在我們重新調整優先順序的推動下，我們將在2023 年第四季實現這一目標。

BioXcel Therapeutics had a net loss of $8.3 million for the second quarter of 2024, compared to a net loss of $53.5 million for the same period in 2023. In the second quarter of 2024, the loss from operations of $17.3 million was offset by an unrealized gains related to derivative liabilities. Company used $23.2 million in operating cash during the second quarter of 2024. Cash and cash equivalents totaled $56.3 million as of June 30, 2024.

BioXcel Therapeutics 2024 年第二季的淨虧損為 830 萬美元，而 2023 年同期的淨虧損為 5,350 萬美元。相關的收益。 2024 年第二季度，公司使用了 2,320 萬美元的營運現金。

Finally, to enable us to deliver on our strategic clinical priorities that Vimal and Vince have described earlier, we are continuing to optimize operational efficiencies across our business.

最後，為了使我們能夠實現 Vimal 和 Vince 先前描述的策略性臨床優先事項，我們將繼續優化整個業務的營運效率。

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？

Thank you. (Operator Instructions) Sumant Kulkarni, Canaccord Genuity.

謝謝。 （操作員說明）Sumant Kulkarni，Canaccord Genuity。

I have three fairly quick ones. On TRANQUILITY In-Care, at what point do you expect to be able to let us know when data might be available? And on that trial, are there any new variables that might make the trial more or less expensive relative to how you characterized the cost previously?

我有三個相當快的。在 TRANQUILITY In-Care 上，您希望什麼時候能讓我們知道資料何時可用？在該試驗中，是否有任何新的變數可能會使試驗相對於您先前描述的成本變得更昂貴或更便宜？

This is Vince. So for TRANQUILITY In-Care, today we're not in a position to provide specific timing. To your second question in terms of cost, we don't obviously typically give specific cost guidance. But we see no reason to have changed our view on the cost of the study recently. It's essentially as previously thought or planned.

這是文斯。因此，對於 TRANQUILITY In-Care，今天我們無法提供具體的時間安排。關於成本方面的第二個問題，我們通常不會給出特定的成本指引。但我們認為沒有理由改變我們最近對這項研究成本的看法。這基本上是按照先前的想法或計劃進行的。

Got it. And then my third one is on SERENITY At-Home, how aligned would you say you are with the FDA on that trial and is the child proof packaging the key part of enabling that trial to proceed?

知道了。然後我的第三個是關於 SERENITY At-Home，您認為您在該試驗中與 FDA 的一致程度如何？

So I would say we are very aligned with the FDA. As I mentioned in prepared remarks, we did receive feedback from the FDA that has been incorporated into the protocol. Also, as mentioned, CNC work is complete, statement of work with the CRO has also been defined. So essentially the make ready work for that study is complete.

所以我想說我們與 FDA 非常一致。正如我在準備好的評論中提到的，我們確實收到了 FDA 的回饋，這些回饋已納入協議中。此外，如前所述，CNC 工作已完成，CRO 的工作說明書也已定義。因此，該研究的準備工作基本上已經完成。

And I'll squeeze one last one in. Can you talk about any potential areas of discussion that you might have with the FDA on the specific TRANQUILITY In-Care protocol?

我將插入最後一項。

I think one point I'll make is, obviously, TRANQUILITY versus SERENITY is an efficacy primary endpoint study. It's obviously a little more complicated, a little longer in duration and actually a little bit more expensive. We know that the FDA is very interested in repeat dose efficacy and I think that will be the one point that we want to get right going forward with that protocol.

我想我要指出的一點是，顯然，「寧靜」與「寧靜」是一項功效主要終點研究。它顯然更複雜一些，持續時間更長一些，而且實際上更昂貴一些。我們知道 FDA 對重複劑量療效非常感興趣，我認為這將是我們希望在該協議中得到正確進展的一點。

Alec Stranahan, Bank of America.

亞歷克·斯特拉納漢，美國銀行。

Good morning. This is Mary Kate on for Alec this morning. And we were just wondering, what are your expectations for the pace of trial execution for the Phase 3 trials upon initiation, maybe specifically SERENITY here? And maybe could you add some additional color on the potential market opportunity for the At-Home population here as well?

早安.這是瑪麗凱特今天早上為亞歷克做的。我們只是想知道，您對第三階段試驗啟動後的試驗執行速度有何期望，也許特別是這裡的「寧靜」？也許您還可以為這裡的居家人群的潛在市場機會添加一些額外的色彩嗎？

This is Vince. Just to paraphrase your first question, essentially, you're asking about the timing of the SERENITY At-Home study, correct?

這是文斯。只是為了解釋你的第一個問題，本質上，你是在問「SERENITY At-Home」研究的時間安排，對嗎？

Yeah. And how long it could potentially take in terms of enrollment once you guys initiate the trial?

是的。一旦你們開始試驗，註冊可能需要多長時間？

Sure. So again, not in a position to give precise timelines today, but I'll actually just repeat what I said a moment ago, and that is that SERENITY -- of the two studies, SERENITY is the less complex study, therefore the faster study and the less expensive study.

當然。再說一遍，今天我無法給出準確的時間表，但我實際上只是重複我剛才所說的話，那就是「寧靜」——在這兩項研究中，「寧靜」是不太複雜的研究，因此研究速度更快以及較便宜的學習。

Yeah. Mary Kate, this Matt. I'll talk a little bit about the market opportunity. So there are 23 million episodes in the at-home market. And what we found in market research is that patients who experience agitation with bipolar schizophrenia indicated that they would use the BXCL501 in 80% of their episodes, oftentimes at the very beginning or even before those episodes started. So in the prodromal phase of the episode.

是的。瑪麗凱特，這個馬特。我會談談市場機會。所以家庭市場有2300萬集。我們在市場研究中發現，患有雙相精神分裂症的躁動患者表示，他們會在 80% 的發作中使用 BXCL501，通常是在發作開始時甚至發作之前。所以在發作的前驅階段。

So the patients are motivated and rarely we could imagine taking some of the 23 million episodes adding to that as we draw some of those episodes out of the emergency department in the hospital.

因此，患者的動機很高，當我們從醫院急診室抽出一些病例時，我們幾乎無法想像從 2300 萬例病例中取出一些病例會增加這種情況。

Graig Suvannavejh, Mizuho.

Graig Suvannavejh，瑞穗。

Hi. This is Avantika on for Graig. I just had a couple of questions. The first is what is the company's current cash runway? And the second is for both the SERENITY and TRANQUILITY studies, are you still planning to finance it yourself or are you thinking of partnering with someone to actually run the trials? And the last is what is the -- what are the gating factors for actually initiating the trials now that you have -- you've discussed the protocol with the FDA, specifically for SERENITY?

你好。這是 Graig 的 Avantika。我只是有幾個問題。首先是公司目前的現金跑道是什麼？第二個是對於 SERENITY 和 TRANQUILITY 研究，您是否仍計劃自己資助，還是考慮實際與某人合作進行試驗？最後一個問題是－既然你已經與 FDA 討論了這個方案，特別是 SERENITY 的方案，那麼實際啟動試驗的限制因素是什麼？

Hi, this is Rich. On the cash runway, we really haven't changed our guidance on cash runway at all. So it remains similar to what we had talked about in the past.

嗨，這是里奇。在現金跑道上，我們實際上根本沒有改變對現金跑道的指導。所以它仍然與我們過去討論過的相似。

Good morning, Avantika, this is Vimal. Regarding your question on the financing, our fundraising strategy is based on funding both trial through various alternatives, as I mentioned. So we are exploring all those opportunities in parallel.

早安，阿凡提卡，我是維馬爾。關於你關於融資的問題，正如我所提到的，我們的籌款策略是透過各種替代方案為這兩項試驗提供資金。因此，我們正在同時探索所有這些機會。

As you said, both trials are at a point where we already have a feedback and SERENITY At-Home can begin and shortly, and we are finalizing our plans for the TRANQUILITY In-Care, which Vince already mentioned. So both are strategic priorities and we are moving them along. And as I mentioned in my prepared remarks, we are looking at strategic partnerships as well as a part of this excercise.

正如您所說，這兩項試驗都處於我們已經收到反饋的階段，SERENITY At-Home 很快就可以開始，我們正在敲定 TRANQUILITY In-Care 的計劃，文斯已經提到過這一點。因此，兩者都是策略重點，我們正在推動它們。正如我在準備好的發言中提到的，我們正在考慮策略夥伴關係以及本次演習的一部分。

Ram Selvaraju, H.C. Wainwright.

塞爾瓦拉朱 (Ram Selvaraju)，H.C.溫賴特。

Thanks so much for taking my questions. Three quick ones here. Firstly, just a question on the commercial side. Can you comment on any favorable tailwinds that you see the product experiencing at this juncture and what impact that might have on the cadence of revenue growth and prescription uptake over the course of the second half of 2024?

非常感謝您回答我的問題。這裡三個快點。首先，只是商業方面的問題。您能否評論一下您認為該產品目前遇到的任何有利因素，以及這可能對 2024 年下半年收入增長和處方使用的節奏產生什麼影響？

Secondly, with respect to OnkosXcel, can you provide any update with respect to potential spin out of that entity or other ways in which you could conceivably monetize BXCL701? And then lastly, if you could perhaps comment on your plans for the neurology pipeline beyond BXCL501 and to what extent that might potentially provide you with additional sources of non-dilutive funding?

其次，關於 OnkosXcel，您能否提供有關該實體的潛在分拆或其他可以透過 BXCL701 貨幣化的方式的最新資訊？最後，您是否可以評論一下 BXCL501 以外的神經學管道計劃，以及這可能在多大程度上為您提供額外的非稀釋性資金來源？

So this is Matt. To answer your question about tailwinds, one of the things that we've observed over the last three quarters is an increase in the interest from these community behavioral health centers. There are about 36,000 of these in the United States.

這就是馬特。為了回答您關於順風的問題，我們在過去三個季度觀察到的事情之一是這些社區行為健康中心的興趣增加。美國大約有 36,000 個這樣的人。

And we know from previous market research that psychiatrists have the highest intent to use the product of all the constituents in a hospital setting. So we think that that translates nicely to this community mental health clinic type setting.

我們從先前的市場研究中得知，精神科醫師最有意願在醫院環境中使用所有成分的產品。因此，我們認為這很好地適用於這種社區心理健康診所類型的設置。

We also, based on our experience, we know that the process for acquiring IGALMI does not require the type of administrative hurdles they typically see in a hospital IDN setting. So we feel like this is an opportunity that is near term low hanging fruit, and we'll continue to progress on that over the balance of the year and into next year as well.

此外，根據我們的經驗，我們知道收購 IGALMI 的過程不需要通常在醫院 IDN 環境中看到的行政障礙。因此，我們認為這是一個近期容易實現的機會，我們將在今年剩餘時間和明年繼續在這一方面取得進展。

This is Vince. Just to reiterate what you have said, we are obviously committed to monetizing 701. So we have initiated partner outreach through a retained firm, a firm that specializes in this area and that's in addition to assessing -- formally assessing the commercial value or indications. So we're pleased with progress so far, and we'll update you in June.

這是文斯。重申你所說的，我們顯然致力於將701 貨幣化。適應症。因此，我們對迄今為止的進展感到滿意，我們將在 6 月向您通報最新情況。

Good morning, Ram. This is Vimal. Regarding your third question, plan for our neuroscience pipeline. Currently, as I mentioned, we are very focused on moving two late-stage pivotal trials, but we will continue to look at it, what are the best options to progress the pipeline which is 502, 503 either through a partnership mechanism or any other ways to move those program forward.

早安，拉姆。這是維馬爾。關於你的第三個問題，規劃我們的神經科學管道。目前，正如我所提到的，我們非常專注於推進兩項後期關鍵試驗，但我們將繼續關注，透過合作機製或任何其他方式推進 502、503 管道的最佳選擇是什麼推動這些計劃的方法。

Thank you. I would like to turn the floor over to Dr. Mehta for closing remarks.

謝謝。我想請梅塔博士致閉幕詞。

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

謝謝。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。

This concludes today's teleconference. You may disconnect your lines at this time, and thank you for your participation.

今天的電話會議到此結束。此時您可以斷開線路，感謝您的參與。