Lumos Pharma Inc (LUMO) 2024 Q2 法說會逐字稿

Good afternoon, and welcome to Lumos Pharma's second quarter 2024 financial results and clinical programs update call.

下午好，歡迎參加 Lumos Pharma 2024 年第二季財務表現及臨床專案更新電話會議。

(Operator Instructions) As a reminder, this conference call is being recorded.

（操作員說明）謹此提醒，本次電話會議正在錄音。

I will now turn the call over to Lisa Miller, Vice President of Investor Relations.

我現在將把電話轉給投資者關係副總裁麗莎·米勒 (Lisa Miller)。

Thank you, operator.

謝謝你，接線生。

Before we proceed with the call, I would like to remind everyone that certain statements made during this call are forward-looking statements under US federal securities laws.

在我們進行電話會議之前，我想提醒大家，根據美國聯邦證券法，本次電話會議中所做的某些陳述屬於前瞻性陳述。

These statements are subject to risks and uncertainties that could cause actual results to differ materially from historical experience or present expectations.

這些陳述存在風險和不確定性，可能導致實際結果與歷史經驗或當前預期有重大差異。

Additional information concerning factors that could cause actual results to differ is contained in our periodic reports filed with the SEC.

有關可能導致實際結果不同的因素的更多資​​訊包含在我們向 SEC 提交的定期報告中。

The forward-looking statements made during this call can speak only as of the date hereof and the company undertakes no obligation to update or revise the forward-looking statements.

本次電話會議中所做的前瞻性陳述僅適用於本次電話會議之日，本公司不承擔更新或修改前瞻性陳述的義務。

Information presented on this call is contained in the press release we issued this afternoon and in our Form 10-Q and 8-K, which may be accessed from the Investors page of the Lumos Pharma website.

本次電話會議中提供的資訊包含在我們今天下午發布的新聞稿以及表格 10-Q 和 8-K 中，可從 Lumos Pharma 網站的投資者頁面存取這些資訊。

Speaking on today's call will be Rick Hawkins, CEO and Chairman; John McKew, President and Chief Scientific Officer; and Lori Lawley, Chief Financial Officer.

執行長兼董事長 Rick Hawkins 將在今天的電話會議上發言。 John McKew，總裁兼首席科學官；財務長洛里·勞利 (Lori Lawley)。

Dr. Duke Pitukcheewanont our Chief Medical Officer, will also join the call for the question-and-answer section.

我們的首席醫療官 Duke Pitukcheew 博士也將加入問答環節。

It's now my pleasure to turn the call over to Rick for our opening remarks.

現在我很高興將電話轉給里克，讓他致開幕詞。

Thank you, Lisa, and good afternoon, everyone.

謝謝麗莎，大家下午好。

I'm pleased to be speaking with you today to provide an update on our progress advancing LUM-201 as the first oral therapeutic for moderate pediatric growth hormone deficiency or PGHD.

我很高興今天與您交談，向您介紹我們將 LUM-201 作為第一個口服治療中度小兒生長激素缺乏症或 PGHD 的口服療法的最新進展。

During the second quarter and recent weeks, we have made significant progress in advancing our plans for a Phase 3 pivotal trial of LUM-20 in PGHD.

在第二季和最近幾週，我們在推進 LUM-20 治療 PGHD 第三階段關鍵試驗的計劃方面取得了重大進展。

Following very positive and productive End-of-Phase 2 Meeting with the FDA.

在與 FDA 進行了非常積極且富有成效的第二階段結束會議之後。

We have nearly finalized our proposal for a Phase 3 double blinded placebo controlled clinical trial with a 2:1 randomization and approximately 150 patients.

我們幾乎已經完成了 3 期雙盲安慰劑對照臨床試驗的提案，該試驗採用 2:1 隨機分組，受試者為約 150 名患者。

The FDA's input has been instrumental in shaping our proposed trial design acknowledging LUM-201 unique mechanism as a growth hormone secretagogue and recognizing that a placebo-controlled clinical trial is an appropriate approach for Phase 3.

FDA 的意見有助於制定我們提出的試驗設計，承認 LUM-201 作為生長激素促分泌劑的獨特機制，並認識到安慰劑對照臨床試驗是第 3 階段的適當方法。

We expect to finalize the design details in the fourth quarter.

我們預計將在第四季度完成設計細節。

Prior to our Meeting with the FDA, we had not developed plans for manufacturing of placebo to match our patented form of LUM-201.

在與 FDA 會面之前，我們尚未制定與我們的 LUM-201 專利形式相符的安慰劑生產計劃。

Now with the decision to move forward with a placebo-controlled Phase 3 trial, additional time is required to complete the manufacturing process and create the placebo matched materials necessary for use in the study.

現在決定推進安慰劑對照的第三階段試驗，需要額外的時間來完成製造過程並創建研究中使用所需的安慰劑匹配材料。

Consequently, extending our intended timeline to initiate Phase 3 in the second quarter of 2025 and this extension also allows all of us to continue managing our cash resources prudently and extend our cash runway.

因此，將我們的預期時間表延長到 2025 年第二季啟動第三階段，這項延期也使我們所有人能夠繼續審慎管理我們的現金資源並延長我們的現金跑道。

Additionally, in today's press release, we announced that we have engaged Piper Sandler to explore all strategic opportunities to maximize shareholder value and advance LUM-201 platform.

此外，在今天的新聞稿中，我們宣布已聘請 Piper Sandler 探索所有策略機會，以最大限度地提高股東價值並推進 LUM-201 平台。

Our regular ongoing business development activities have generated significant interest in the global potential for LUM-201 in multiple markets.

我們定期進行的業務開發活動引起了人們對 LUM-201 在多個市場的全球潛力的濃厚興趣。

Given this positive feedback we feel it's the right time to formally engaged Piper Sandler to ensure we're thoroughly exploring every potential transaction and opportunity that serves our shareholders best interest.

鑑於這一積極回饋，我們認為現在是正式聘請 Piper Sandler 的最佳時機，以確保我們徹底探索每項潛在交易和機會，為股東的最佳利益服務。

We're confident in LUM-201 potential as the first oral therapeutic in the expanding $5 billion global growth hormone market we're going to continue to focus our efforts on optimizing value for all of our stakeholders.

我們對 LUM-201 作為第一個口服治療藥物在不斷擴大的 50 億美元全球生長激素市場中的潛力充滿信心，我們將繼續集中精力為所有利益相關者優化價值。

In a moment, John and Lori are going to provide more details on this and other measures we're implementing to ensure we are well prepared to successfully initiate this trial next year.

稍後，約翰和洛里將提供有關此措施以及我們正在實施的其他措施的更多詳細信息，以確保我們為明年成功啟動這項試驗做好充分準備。

In the second quarter, we're pleased to see new analyses of data from our OraGrowtH trials presented at end of 2024.

在第二季度，我們很高興看到對 2024 年底 OraGrowtH 試驗數據的新分析。

The data emphasize the significance of the pulsatility pattern and growth hormone secretion from the growth response from LUM-201.

這些數據強調了 LUM-201 生長反應中的搏動模式和生長激素分泌的重要性。

They further illustrate them to a once unique ability to restore natural pulsatile growth hormone secretion, achieving similar to injectable recombinant growth hormone was significantly less exposure to circulating growth hormone.

他們進一步說明了他們曾經獨特的恢復自然脈動生長激素分泌的能力，實現了類似於注射重組生長激素的顯著更少的循環生長激素暴露。

These findings strongly support our planned placebo-controlled Phase 3 trial of LUM-201 in moderate PGHD.

這些發現強烈支持我們計劃在中度 PGHD 中進行 LUM-201 安慰劑對照 3 期試驗。

And top line results from the OraGrowtH212 trials were presented at three major endocrinology conferences in May this year.

OraGrowtH212 試驗的主要結果已於今年 5 月在三個主要內分泌學會議上公佈。

At the Pediatric Endocrine Society Meeting in Chicago, Dr. Andrew Dauber highlighted data showing that LUM-201 dose of 1.6 mgs per kg day achieved annualized height velocity comparable to daily injectable growth hormone and PGHD with a promising safety profile.

在芝加哥舉行的兒科內分泌學會會議上，Andrew Dauber 博士強調了數據顯示，每天每公斤 1.6 毫克的 LUM-201 劑量所達到的年化身高速度與每日註射生長激素和 PGHD 相當，且安全性良好。

Meanwhile, at the Growth Hormone Research Society and the European Congress of Endocrinology, Dr. Peter Clayton presented detailed analysis of LUM-201 effectiveness in restoring growth hormone secretion and increasing annualized height velocity at moderate PGHD patients.

同時，在生長激素研究學會和歐洲內分泌學大會上，Peter Clayton 博士詳細分析了 LUM-201 在恢復中度 PGHD 患者生長激素分泌和增加年身高增長速度方面的有效性。

The evolving data from all of those trials continue to attract significant interest from the Global Endocrinology Community, and we're encouraged by the growing enthusiasm from experts eager to participate in our Phase 3 trial as we finalize our plans.

所有這些試驗不斷變化的數據繼續引起全球內分泌學界的極大興趣，在我們最終確定計劃時，渴望參與我們的第三階段試驗的專家們的熱情日益高漲，這讓我們感到鼓舞。

Now I'm going to turn it over to John McKew for more details on our engagement with the FDA and our Phase 3 plan.

現在我將把它交給 John McKew，以了解有關我們與 FDA 合作以及我們的第 3 階段計劃的更多詳細資訊。

John?

約翰？

Thanks, Rick, and good morning and good afternoon, everyone.

謝謝瑞克，大家早安，下午好。

As Rick mentioned, following our productive end of Phase 2 Meeting with the FDA, we have been diligently designing a placebo-controlled Phase 3 registrational trial based on the FDA's recommendations and insights from our regulatory consultants, key opinion leaders and statisticians.

正如Rick 所提到的，在我們與FDA 的第二階段會議富有成效地結束之後，我們一直在根據FDA 的建議以及我們的監管顧問、關鍵意見領袖和統計學家的見解，努力設計安慰劑對照的第三階段註冊試驗。

We have crafted a trial design aimed at maximizing the likelihood of success.

我們精心設計了一項試驗設計，旨在最大限度地提高成功的可能性。

This trial will involve approximately 150 patients at over 80 global centers of a 2:1 randomization ratio of LUM-201 to placebo.

該試驗將涉及全球 80 多個中心的約 150 名患者，LUM-201 與安慰劑的隨機比例為 2:1。

One group will receive LUM-201 for 12 months.

一組將接受 LUM-201 治療 12 個月。

While the other will the other group will start with a placebo for six months and then crossover to LUM-201 for the remaining six months.

另一組將從安慰劑開始服用六個月，然後在剩下的六個月內交叉使用 LUM-201。

All participants must meet our 10 positive criteria during the screening process before randomization.

所有參與者在隨機分組前的篩選過程中都必須符合我們的 10 項積極標準。

This Phase 3 trial aims to achieve two key strategic objectives.

第三階段試驗旨在實現兩個關鍵戰略目標。

First is to provide the FDA with comprehensive data from a blinded controlled trial to support the valuation approval LUM-201.

首先是向 FDA 提供盲對照試驗的全面數據，以支持 LUM-201 的估值批准。

And the second is to ensure that all participants received LUM-201 at some point during the study enhancing its appeal to both patients and physicians for enrollment.

其次是確保所有參與者在研究期間的某個時間點接受 LUM-201，以增強其對患者和醫生入組的吸引力。

The study will have two co-primary endpoints.

該研究將有兩個共同主要終點。

The first would be to demonstrate the superiority of LUM-201 and annualized height velocity compared to the growth on placebo.

首先是證明 LUM-201 和年身高成長速度與安慰劑相比的優越性。

Second, co-primary would be to ensure that the AHV of LUM-201 has a lower bound of the 95% confidence interval above 6.7 centimeters per year, which is the minimal clinically meaningful AHV agreed upon with the FDA.

其次，共同的首要任務是確保 LUM-201 的 AHV 的 95% 置信區間下限高於每年 6.7 厘米，這是 FDA 同意的具有臨床意義的最小 AHV。

Following the 12-month treatment period, all participants will have the option to transition into a long-term safety extension receiving long-term treatment for up to three years.

12 個月的治療期結束後，所有參與者都可以選擇過渡到長期安全延期，接受長達三年的長期治療。

We believe this extension will be a compelling feature for enrollment and will help fulfill FDA's requirements for longer-term exposure.

我們相信這項擴展將成為一個引人注目的註冊功能，並將有助於滿足 FDA 對長期暴露的要求。

We are confident that the trial is sufficiently powered to achieve the two co-primary endpoints.

我們相信該試驗有足夠的能力實現兩個共同主要終點。

We are currently finalizing the design proposal and expect to secure final agreement with the FDA in the fourth quarter.

我們目前正在敲定設計方案，並預計在第四季與 FDA 達成最終協議。

A key component of the trial was the manufacturing of the placebo material, which we began after end of Phase 2 Meeting with the FDA.

該試驗的關鍵組成部分是安慰劑材料的生產，我們在與 FDA 的第二階段會議結束後開始生產。

As Rick mentioned, the placebo needs to be a capsule filled with many tablets that exactly match the presentation of active LUM-201 capsules.

正如 Rick 所提到的，安慰劑需要是一個膠囊，裡面裝滿了與活性 LUM-201 膠囊的外觀完全匹配的許多藥片。

Additionally, we must establish all standard stability data for this newly created placebo.

此外，我們必須為這種新創建的安慰劑建立所有標準穩定性數據。

Although we began work on these elements promptly producing the initial batches that met the specifications took longer than anticipated.

儘管我們開始研究這些元件，但迅速生產出符合規格的第一批產品所花費的時間比預期的要長。

Consequently, we chose to postpone other preparatory steps for trial initiations to manage our cash reserves effectively.

因此，我們選擇推遲試驗啟動的其他準備步驟，以有效管理我們的現金儲備。

We believe this is a prudent use of resources and expect to be fully prepared to launch Phase 3 in the second quarter of 2025.

我們相信這是對資源的審慎利用，並預計將做好充分準備，在 2025 年第二季啟動第三階段。

With a careful attention given to the trial design, we are confident in our ability to enroll patients in this pivotal trial in a timely manner.

透過對試驗設計的仔細關注，我們對及時招募患者參加這項關鍵試驗的能力充滿信心。

With that I'll turn it over to Lori for a review of our financial results for the second quarter.

接下來，我將把它交給洛里，讓他審查我們第二季的財務表現。

Thank you, John.

謝謝你，約翰。

We ended the quarter on June 30, 2024, with cash, cash equivalents and short-term investments totaling $16.8 million as compared to $36 million on December 31, 2023.

截至 2024 年 6 月 30 日，本季結束時，我們的現金、現金等價物及短期投資總額為 1,680 萬美元，而 2023 年 12 月 31 日為 3,600 萬美元。

As Rick and John have discussed, we are managing our cash resources conservatively by extending the initiation date of our Phase 3 trial into the second quarter of 2025.

正如 Rick 和 John 所討論的，我們透過將第 3 階段試驗的啟動日期延長至 2025 年第二季來保守地管理我們的現金資源。

We have also extended our cash runway through into the first quarter of next year.

我們也將現金跑道延長至明年第一季。

We will continue to manage our resources carefully and work with Piper Sandler to explore our opportunities to ensure we are well prepared to launch the trial effectively when the time is right.

我們將繼續謹慎管理我們的資源，並與 Piper Sandler 合作探索我們的機會，以確保我們做好充分準備，在適當的時候有效地啟動試驗。

Research and development expenses for the quarter were $4.6 million, a decrease of $1.4 million for the quarter ended June 30, 2024, compared to the same period in 2023, primarily due to decreases of $1.1 million in contract manufacturing expenses $0.3 million in personnel related expenses and $0.2 million in clinical trial expenses, offset by an increase of $0.2 million in consulting expenses.

該季度的研發費用為460 萬美元，截至2024 年6 月30 日的季度與2023 年同期相比減少了140 萬美元，主要是由於合約製造費用減少了110 萬美元，人事相關費用減少了30 萬美元20 萬美元的臨床試驗費用，被增加的 20 萬美元的諮詢費用所抵銷。

General and administrative expenses for the quarter were $3.7 million, a decrease of $0.5 million compared to the same period in 2023, primarily due to decreases of $0.2 million in personnel-related expenses, $0.1 million in travel, $0.1 million in consulting and $0.1 million in other expenses.

本季一般及行政費用為370 萬美元，較2023 年同期減少50 萬美元，主要是由於人事相關費用減少20 萬美元、差旅費用減少10 萬美元、諮詢費用減少10 萬美元、管理費用減少10 萬美元。

Net loss for the quarter ended June 30, 2024, was $7.6 million compared to a net loss of $8.9 million for the same period in 2023.

截至 2024 年 6 月 30 日的季度淨虧損為 760 萬美元，而 2023 年同期淨虧損為 890 萬美元。

Lumos Pharma ended Q2 2024 with 8,123,186 shares outstanding.

截至 2024 年第二季末，Lumos Pharma 已發行股票為 8,123,186 股。

With that, I will now turn it over to Rick for his closing remarks.

現在我將把會議交給 Rick 作結束語。

Thank you, Lori and John.

謝謝你們，洛瑞和約翰。

And as we mentioned at the start of the call, it's been an exciting and productive period for Lumos.

正如我們在電話會議開始時所提到的，對於 Lumos 來說，這是一個令人興奮且富有成效的時期。

Again, we're thrilled by the outcome of the end-of-Phase 2 Meeting with the FDA.

我們再次對與 FDA 的第二階段會議結束的結果感到興奮。

In recognition that LUM-201 is not another growth hormone mimetic, the FDA's guidance supporting a placebo-controlled trial, we believe to significantly de-risk our program.

認識到 LUM-201 不是另一種生長激素模擬物，FDA 的指導支持安慰劑對照試驗，我們相信可以顯著降低我們計畫的風險。

We're progressing toward initiating a Phase 3 registrational trial and achieving our goal of establishing LUM-201 as the first oral therapeutic capable of transforming the global growth hormone market, which has been dominated by injectable products for nearly 40 years.

我們正在努力啟動 3 期註冊試驗，並實現將 LUM-201 打造成第一個能夠改變全球生長激素市場的口服治療藥物的目標，近 40 年來，全球生長激素市場一直由注射產品主導。

We anticipate some very exciting developments throughout this year and look forward to keeping you informed about our progress.

我們預計今年會出現一些非常令人興奮的進展，並期待讓您隨時了解我們的進展。

Thank you all very much.

非常感謝大家。

And operator, we're ready to take questions.

接線員，我們準備好回答問題。

We will now begin the question-and-answer session.

我們現在開始問答環節。

(Operator Instructions)

（操作員說明）

Catherine Novack, Jones Trading.

凱瑟琳·諾瓦克，瓊斯貿易公司。

Hi, good afternoon, guys.

嗨，大家下午好。

It's exciting that you're able to get the Phase 3 trial design nail down with the FDA.

令人興奮的是，您能夠與 FDA 確定 3 期試驗設計。

But I wanted to ask about your expectations for enrollment dynamics.

但我想問一下您對招生動態的期望。

How long is it going to take for you to enroll 150 patients study?

你們招募 150 位患者的研究需要多久？

Given that is a 12-month study, should we not be expecting data until late 2026, 2027 whenever we're going to see updates from this?

鑑於這是一項為期 12 個月的研究，我們是否應該等到 2026 年底、2027 年才能看到更新數據？

So Duke, will you answer that question.

杜克，你能回答這個問題嗎？

And recall Duke is our Chief Medical Officer, and he's a pediatric endocrinologist?

還記得杜克是我們的首席醫療官，他是兒科內分泌學家嗎？

Go ahead, Duke.

繼續吧，杜克。

Thank you.

謝謝。

Thank you, Catherine.

謝謝你，凱瑟琳。

That's a good question, right?

這是個好問題，對吧？

So when we have the trial decide placebo-controlled trial.

因此，當我們進行試驗時，我們決定安慰劑對照試驗。

We have been looking to diligently about where to site might be.

我們一直在努力尋找選址地點。

So this type of placebo-controlled trial, we wanted to make sure now to meet the time line 15 to 18 months like what we plan, we do believe, and we're confident we can get that passed in depth duration complete.

因此，這種類型的安慰劑對照試驗，我們希望確保現在能夠滿足 15 至 18 個月的時間線，就像我們計劃的那樣，我們確實相信，並且我們有信心能夠在持續時間內完成該試驗。

What we plan to do is that we tried to enroll the patient different continent, especially we go including Latin America and Asia and South Eastern Europe.

我們計劃要做的是，我們嘗試將患者納入不同大陸，特別是我們去的地區包括拉丁美洲、亞洲和東南歐。

In those areas we have limited access to growth hormone.

在這些地區，我們獲得生長激素的機會有限。

And we do believe that by include some of those areas, we will be able to enroll those patients.

我們確實相信，透過納入其中一些領域，我們將能夠招募這些患者。

Not to mention when we announced the placebo-controlled trial, the last earnings call, we have influx of interest of the physician who interest to be part of our Phase 3.

更不用說當我們宣布安慰劑對照試驗（最後一次財報電話會議）時，有大量醫生對我們感興趣，他們有興趣參與我們的第三階段。

We have more than site that we need so we'd expect that 150 subjects 80 to 100 patients.

我們有超過我們需要的站點，因此我們預計有 150 名受試者和 80 到 100 名患者。

And we do believe that with a 15 to 18-month enrollment, we can achieve that pretty easily.

我們確實相信，透過 15 到 18 個月的註冊，我們可以輕鬆實現這一目標。

Got it.

知道了。

Is that?

是嗎？

And what is your other question?

你的另一個問題是什麼？

Okay.

好的。

And then sort of a strategic thought, assuming you are able to secure additional financing, are there other opportunity you could explore in the meantime to compensate for a data catalyst for data shadow from PGHD?

然後是一種策略思考，假設您能夠獲得額外的融資，那麼您是否可以同時探索其他機會來補償 PGHD 資料影子的資料催化劑？

Well, Catherine, we are completely focused on PGHD, obviously, yes, there are other indications that are that are out there for us explore, but we're going to do a prudent use of our capital to focus on where we're going to get across the finish line first.

好吧，凱瑟琳，我們完全專注於 PGHD，顯然，是的，還有其他跡象可供我們探索，但我們將謹慎使用我們的資本，專注於我們要去的地方首先衝過終點線。

And I don't know, Lori, if you have any additional thing you want to add to that?

我不知道，Lori，你還有什麼要補充的嗎？

No, I think you covered it well, Rick.

不，我認為你講得很好，瑞克。

I think as we suggested, our goal is to manage cash conservatively and ensure that we are and extending our runway to support operations and evaluate all opportunities at hand.

我認為正如我們所建議的，我們的目標是保守地管理現金，並確保我們能夠擴展我們的跑道以支持營運並評估手頭上的所有機會。

Got it.

知道了。

Thanks, guys.

謝謝，夥計們。

Thank you, Catherine.

謝謝你，凱瑟琳。

Leland Gershell, Oppenheimer.

利蘭·格謝爾，奧本海默。

Hey, good afternoon.

嘿，下午好。

Thanks for taking my questions.

感謝您回答我的問題。

Just wanted to ask, Rick, it sounded from your commentary around the potential goals with the strategic opportunities that you could be looking at, but maybe ex-US deals for LUM-201 and then initial monies from those deals could help fund your registration trial.

只是想問一下，Rick，從您對潛在目標和您可能關注的戰略機會的評論來看，也許 LUM-201 在美國以外的交易以及這些交易中的初始資金可以幫助您資助您的註冊試驗。

Is that a reasonable scenario?

這是一個合理的場景嗎？

Thanks.

謝謝。

It's reasonable, but you have to remember, Leland, on a regular basis, our business development folks have been generating a significant amount of interest on global potential of LUM-201 in multiple markets.

這是合理的，但你必須記住，Leland，我們的業務開發人員經常對 LUM-201 在多個市場的全球潛力產生極大的興趣。

And we've gotten a lot of positive feedback.

我們收到了很多正面的回饋。

And as a result, we felt it's probably the right time to engage an investment banker to make sure that we explore every one of these opportunities and any kind of potential transaction or opportunity that really serves all of our shareholders best.

因此，我們認為現在可能是聘請投資銀行家的最佳時機，以確保我們探索每個機會以及真正為我們所有股東提供最佳服務的任何潛在交易或機會。

So as a result, I think, it's easy to say we got a lot of different directions we can go.

因此，我認為，很容易說我們有很多不同的方向可以走。

We're in active discussions with not just investors, but as you pointed out, strategics have been interested for quite some time, both for maybe either global and also their regional players.

我們不僅與投資者進行積極的討論，而且正如您所指出的，戰略長期以來一直對全球和地區參與者感興趣。

But we're going to really choose the right deal or combination of deals that provides us the highest value to our shareholders at the lowest cost of capital that we can.

但我們將真正選擇正確的交易或交易組合，以盡可能最低的資本成本為我們的股東提供最高的價值。

Now I can tell you we're as a Phase 3 ready asset in a $5 billion market that we offer some really significant advantages, not just the fact oral delivery, but unique mechanism of action.

現在我可以告訴你，我們作為 50 億美元市場中的第三階段資產，我們提供了一些非常重要的優勢，不僅是口服給藥，還有獨特的作用機制。

I think we're feeling pretty good about our position right now.

我認為我們現在對自己的處境感覺很好。

Okay.

好的。

Thanks.

謝謝。

That's helpful.

這很有幫助。

And then just wanted to ask, I believe you have there's an ongoing study on the academic side in non-alcoholic fatty liver?

然後我想問一下，我相信您在非酒精性脂肪肝的學術方面正在進行一項研究嗎？

Just wanted to see if there's any update you can provide there.

只是想看看您是否可以提供任何更新。

Thank you.

謝謝。

Not much of an update except to say that the study is progressing in nonalcoholic fatty liver disease with our clinician at MGH.

沒什麼更新，只是說我們麻省總醫院的臨床醫生在非酒精性脂肪肝方面的研究正在取得進展。

And as you know, I think we have a potential in the cardiometabolic space as a combination product.

如您所知，我認為我們作為組合產品在心臟代謝領域具有潛力。

And we have some very interesting data that tells us we should spend more time there.

我們有一些非常有趣的數據告訴我們應該花更多時間在那裡。

And I think this study will also help us get to the next stage.

我認為這項研究也將幫助我們進入下一階段。

Great, terrific.

太棒了，太棒了。

Thanks very much for the added color.

非常感謝添加的顏色。

Charles Duncan, Cantor Fitzgerald.

查爾斯鄧肯，坎托菲茨杰拉德。

Good afternoon, Rick and team.

下午好，瑞克和團隊。

Thanks for taking our question.

感謝您提出我們的問題。

And let's see, I guess, congratulations on the recent end of Phase 2 Meeting.

我想，讓我們祝賀第二階段會議最近結束。

I guess we've talked about that a little bit in the past, but I wanted to ask you a couple of more questions about that.

我想我們過去已經討論過這個問題，但我想再問你幾個問題。

Do you plan or are you contemplating an interim review to look at conditional power?

您是否計劃或正在考慮進行臨時審查以了解有條件的權力？

Is there any natural cut point in which you may take a look at the patients enrolled and what kind of rates you're seeing in terms of annual height change, even just on a blinded basis?

是否存在任何自然的切入點，您可以在其中查看入組的患者以及您所看到的年度身高變化率，即使只是在盲法的基礎上？

Is there any news flow during the conduct of the trial that you could point to?

您可以指出審判過程中是否有任何新聞動態？

John, you want to answer that question?

約翰，你想回答這個問題嗎？

Hi Charles, we do not plan to take an interim peak.

嗨，查爾斯，我們不打算採取臨時高峰。

I'm not a great idea for the Phase 3 study even in a blinded sense.

即使在盲目的意義上，我也不是第三階段研究的好主意。

So we do, we will keep the data as far as we can going forward.

所以我們會盡量保留資料。

Appreciate the rigor.

欣賞嚴謹性。

And the other question that I had with regard to operationalizing that protocol, I again what you're saying in terms of manufacturing the placebo because that was a new and, in many ways, positive twist as to the plan.

關於實施該協議的另一個問題，我再次重申您在製造安慰劑方面所說的話，因為這是一個新的，並且在許多方面對計劃來說是積極的轉變。

But I'm kind of wondering between now and second quarter, what are some of the rates limiting steps?

但我有點想知道從現在到第二季度，有哪些利率限制措施？

Is it possible that your second quarter goal is not June, it's maybe April or help us understand the path to getting this trial underway?

您的第二季目標是否有可能不是 6 月，而是 4 月，或幫助我們了解進行此試驗的路徑？

Yes.

是的。

John, I want you to answer that question too.

約翰，我也希望你回答這個問題。

So while we are finalizing the protocol with the FDA, we are doing quite a bit of pre-work.

因此，在我們與 FDA 敲定協議的同時，我們正在做大量的前期工作。

Right now we are looking at sites, identifying investigators, identifying the best regions to work at, finalizing our placebo work, right.

現在我們正在尋找地點，確定研究人員，確定最佳工作區域，最終確定我們的安慰劑工作，對吧。

And as we transition to having an FDA approved protocol towards the end of the year in Q4, then we start the regulatory reach out for all the ex-US regulators.

當我們在第四季年底過渡到 FDA 批准的協議時，我們開始對所有前美國監管機構進行監管。

We go to the EMA, we go to some of the other regions.

我們去了 EMA，我們去了其他一些地區。

And then that starts the process of us negotiating contracts and budgets with each one of the 80-plus sites that we intend to have in the Phase 3 trial.

然後我們就開始與我們打算在第三階段試驗中擁有的 80 多個站點中的每一個站點談判合約和預算。

So I think there really is quite a bit of work for us to do after the FDA finalizes the design with us that will take us that period of time.

因此，我認為在 FDA 與我們最終確定設計之後，我們確實還有很多工作要做，這將花費我們一段時間。

Q2 is three months long, so we'll be in that window.

第二季有三個月的時間，所以我們將處於這個窗口期。

I don't have any more precise estimates for each of ours.

我對我們每個人都沒有更精確的估計。

Okay.

好的。

And then with regard to the FDA finalizing the protocol if you will, as you just mentioned, are there any outstanding issues?

那麼，關於 FDA 最終確定協議的問題，正如您剛才提到的，是否有任何懸而未決的問題？

Do you see this as I can't chart thing or you do believe there are points of call it debate you really want to get their input at this point, do you have full alignment?

你是否認為這是因為我無法繪製圖表，或者你確實相信有一些所謂的辯論點，你真的想在這一點上得到他們的意見，你有完全一致嗎？

Please continue, John.

請繼續，約翰。

Yeah, I think there's points of clarification on the specifics of the design.

是的，我認為設計細節有一些需要澄清的地方。

And remember, we have to proceed with the FDA's time lines for back-and-forth interactions.

請記住，我們必須按照 FDA 的時間表進行來回互動。

So that's really where we are right now.

這就是我們現在的處境。

Okay.

好的。

My last question is perhaps for Lori.

我的最後一個問題也許是問洛瑞的。

I'm wondering if you've been able to put pen to paper.

我想知道你是否能夠用筆寫在紙上。

I imagine you have been able to subsequent to the end of Phase 2 Meeting to kind of plan the budget for this trial.

我想您已經能夠在第二階段會議結束後規劃本次試驗的預算。

And I'm wondering where you're landing in terms of all in capital or plus cash needed to at least complete enrollment?

我想知道您的全部資本或至少完成註冊所需的現金在哪裡？

Go head Lori.

洛瑞，去吧。

Sure.

當然。

So if you recall, Charles, and great to talk with you today.

如果您還記得的話，查爾斯，很高興今天與您交談。

We previously have said and we've continued to say about $85 million to $100 million will support operations through 2026.

我們先前曾表示並將繼續表示將提供約 8,500 萬至 1 億美元的資金支持直至 2026 年的營運。

And so that has been the goal in terms of the capital we're looking to bring into finance the Phase 3 and to initiate the Phase 3 trial.

因此，這就是我們尋求為第三階段提供資金並啟動第三階段試驗的資本目標。

As we initiate and finalize the plans with the FDA, we'll issue additional guidance around timelines from an enrollment perspective and we may update those numbers as we finalize that trial design.

當我們與 FDA 啟動並最終確定計劃時，我們將從註冊角度發布有關時間表的額外指導，並且我們可能會在最終確定試驗設計時更新這些數字。

Okay.

好的。

That's great.

那太棒了。

No, I didn't recall that.

不，我不記得了。

So I appreciate you reminding us.

所以我很感謝你提醒我們。

So thanks for taking my questions.

謝謝你回答我的問題。

Thank you, Charles.

謝謝你，查爾斯。

Thank you.

謝謝。

Yasmeen Rahimi, Piper Sandler.

亞斯明·拉希米，派珀·桑德勒。

Hey, Good Afternoon, team.

嘿，下午好，團隊。

Thanks for taking our questions.

感謝您回答我們的問題。

First, could you remind us of the rationale for the formulation change from Phase 2 to Phase 3 on.

首先，您能否提醒我們將配方從第 2 階段改為第 3 階段的理由。

Do you need to do any additional bioequivalent studies with the mini tablets?

您是否需要對迷你片劑進行任何其他生物等效性研究？

And secondly, could you provide a little more color on what types of potential strategic opportunities are considering at this junction?

其次，您能否進一步說明目前正在考慮哪些類型的潛在策略機會？

Thank you.

謝謝。

I'll let you go first, John, in terms of bioequivalence or any other types of studies.

約翰，在生物等效性或任何其他類型的研究方面，我會讓你先走。

Yes.

是的。

So the rationale for that change in formulation was really threefold.

因此，改變表述的理由其實有三。

So, we want it to come.

所以，我們希望它到來。

If you remember, we are dosing by weight across a wide rate range with an oral product.

如果您還記得的話，我們口服產品的給藥劑量範圍很廣，以體重計算。

And so switching to this mini tab and the capsule allows us to make three different dosage strengths by just varying the number of many of the same, many tablets inside a capsule that allows us to have a much tighter and dose variance across that large weight range.

因此，改用這種迷你片劑和膠囊使我們能夠透過改變膠囊內許多相同片劑的數量來製造三種不同的劑量強度，從而使我們能夠在如此大的體重範圍內獲得更嚴格的劑量差異。

And it also gives us the opportunity for several different routes of administration, the larger kids and the older kids who are able to swallow a capsule or able to just take their capsules, the younger children might have a harder time and open the capsule up and take a mini tablet or even a mini tablet in soft fruit like period banana that will ease kind of the treatment burden across that whole.

它也為我們提供了幾種不同給藥途徑的機會，較大的孩子和較大的孩子能夠吞嚥膠囊或只能服用膠囊，較小的孩子可能會更困難，打開膠囊並服用服用迷你片劑，甚至是軟質水果（如香蕉）的迷你片劑，可以減輕整個治療的負擔。

It's range that we're talking about in our trial and commercially.

這是我們在試驗和商業中討論的範圍。

And yes, there would be a bridging PK study that we have already completed.

是的，我們已經完成了一項橋接 PK 研究。

So to answer your second part of that question is, yeah, you can imagine.

所以回答這個問題的第二部分是，是的，你可以想像。

I mean, our BD folks have done a great job and outreach to all the markets.

我的意思是，我們的 BD 人員做得非常出色，並且擴展到所有市場。

And they've really generated considerable interest and we've had ongoing discussions.

他們確實引起了相當大的興趣，我們一直在進行討論。

Once again, we can't be specific about those discussions, but I'll say that at least that they're not only strategic and strategic markets, but even beyond.

再說一次，我們不能具體說明這些討論，但我會說，至少它們不僅是策略和策略市場，甚至超越。

So both global and regional type of players who are interested.

因此全球和區域類型的玩家都感興趣。

We're going to be very careful and look at all the possibilities and potential deals that are on the table and the combination of whether it be financing or strategic is really going to be an interesting exercise over the next coming weeks and months.

我們將非常小心，審視所有擺在桌面上的可能性和潛在交易，無論是融資還是策略的結合，在接下來的幾周和幾個月裡確實將是一項有趣的活動。

I think we'll leave it at that.

我想我們就這樣吧。

Alright.

好吧。

Thank you very much.

非常感謝。

That's very helpful.

這非常有幫助。

This concludes our question and answer session as well as the conference.

我們的問答環節和會議到此結束。

Thank you for attending today's presentation.

感謝您參加今天的演講。

You may now disconnect.

您現在可以斷開連線。