Lumos Pharma Inc (LUMO) 2023 Q4 法說會逐字稿

Good afternoon, and welcome to Lumos Pharma 2023 fourth quarter and year end conference call.

下午好，歡迎參加 Lumos Pharma 2023 年第四季和年終電話會議。

Currently, all participants are in a listen-only mode.

目前，所有參與者都處於僅監聽模式。

Later we will conduct a question and answer session and instructions will follow at that time.

稍後我們將進行問答環節，屆時將提供說明。

As a reminder, this conference call is being recorded.

謹此提醒，本次電話會議正在錄音中。

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)。

Please go ahead.

請繼續。

Thank you, operator.

謝謝你，接線生。

Before we proceed with this call, I'd like to remind everyone that certain statements made during this call are forward-looking statements under U.S. federal securities laws.

在我們繼續這次電話會議之前，我想提醒大家，本次電話會議中所做的某些陳述屬於美國聯邦證券法規定的前瞻性陳述。

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。

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

本次電話會議期間所做的前瞻性陳述僅代表截至本新聞稿發布之日的情況，本公司不承擔更新或修改前瞻性陳述的義務。

Information information presented on this call is contained in the press release we issued this afternoon and in our Form 10 K, which may be accessed from the Investors page of the company's 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和 10 K 表格中，您可以從公司網站的投資者頁面訪問該表格。

Speaking on today's call will be Rick Hopkins, CEO and Chairman, John McHugh, our President and Chief Scientific Officer, Dr. Tuan, Chief Medical Officer, and Lori Lilly, our Chief Financial Officer.

執行長兼董事長 Rick Hopkins、總裁兼首席科學官 John McHugh、首席醫療官 Tuan 博士和財務長 Lori Lilly 將在今天的電話會議上發表演說。

Following our prepared remarks, the management team will be available for a question and answer session.

在我們準備好的發言之後，管理團隊將進行問答環節。

I will now turn the call over to Rick.

我現在將把電話轉給里克。

Thank you, Lisa, and good afternoon, everyone.

謝謝麗莎，大家下午好。

After the market closed today, we issued a press release announcing our results for 2023.

今天收盤後，我們發布了新聞稿，宣布 2023 年業績。

I'm pleased to report that 2023 was a successful year for Lumos, led by our announcement of positive top line results from our oral growth to 10 and will grow to 12 trials alone to a one in pediatric growth hormone deficiency.

我很高興地向大家報告，2023 年對Lumos 來說是成功的一年，我們宣布口腔生長試驗取得了積極的頂線結果，其中包括10 項試驗，並將增加到12 項，其中一項是關於兒科生長激素缺乏症的試驗。

On today's call, I will briefly summarize our trial results and other progress made in 2023.

在今天的電話會議上，我將簡要地總結我們的試驗結果以及 2023 年的其他進展。

Can recap highlights for recent weeks in May, our plans for 2024, and we'll be happy to answer your questions.

您可以回顧一下 5 月最近幾週的亮點以及我們 2024 年的計劃，我們很樂意回答您的問題。

Before I begin, I'd like to take a moment to congratulate Dr. percent Duke Project ONE and on his recent promotion to the role of Chief Medical Officer for Lumos.

在開始之前，我想花一點時間祝賀 Percent Duke Project ONE 博士，並祝賀他最近晉升為 Lumos 首席醫療官。

Dr. Duke's exceptional credentials are widely acknowledged, given his renowned expertise in growth disorders and his current leadership at the human growth foundation among various other distinguished roles and his team career since joining us in 2022, doctor, do it played a pivotal role in steering our overall trials to fruition, providing invaluable guidance, leveraging has influential network for enrollment and significantly contributing to analysis of trial data as our new CMO, Dr. group will continue to advocate for exploration of withdrawal one within the PTSD clinical community, a mission that has already garnered remarkable interest from pediatric endocrinologist globally, thanks to this advocacy efforts.

鑑於杜克博士在生長障礙方面享有盛譽的專業知識以及他目前在人類生長基金會的領導地位以及其他各種傑出的角色，以及他自2022 年加入我們以來的團隊職業生涯，杜克博士的卓越資歷得到了廣泛認可，博士，這在指導方面發揮了關鍵作用嗎？我們的整體試驗取得了成果，提供了寶貴的指導，利用了有影響力的入組網絡，並為試驗數據分析做出了重大貢獻，作為我們的新任首席行銷官，博士小組將繼續倡導在PTSD 臨床社區內探索撤回治療，這一使命由於這一宣傳工作，它已經引起了全球兒科內分泌學家的極大興趣。

Furthermore, Dr. Duke will be an integral part of shaping the trial design for a pivotal into one trial in PGHD details of which we're going to delve into later during this call.

此外，Duke 博士將成為塑造 PGHD 一項關鍵試驗的試驗設計的不可或缺的一部分，我們將在稍後的電話會議中深入探討該細節。

Dr. Dupont, we joined the call today for our Q&A session.

杜邦博士，我們今天參加了問答環節的電話會議。

So please join me in congratulating him on his well-deserved promotion.

因此，請和我一起祝賀他當之無愧的晉升。

Let's now revisit the key highlights of the results we disclosed last November.

現在讓我們回顧一下我們去年 11 月披露的業績的主要亮點。

As many of you are aware of them to a one has successfully met all primary and secondary endpoints in both worldwide trials, demonstrating clear proof of concept for its efficacy as an oral alternative to daily and weekly injectables for patients with PGHD results from our grow to 10 demonstrated that to 1.6 megs per kick dose of to tool Once achieved, annualized height velocities were a series of 8.2 centimeters a year, six months and 8 centimeters a year at 12 months, aligning consistently with historical growth rates for the moderate PGHD population.

正如你們中的許多人都知道的那樣，其中一個在兩項全球試驗中成功達到了所有主要和次要終點，證明了其作為口服替代品每日和每週注射劑對PGHD 患者的功效，從我們的成長到圖10 表明，一旦達到每次踢1.6 兆兆的劑量，年化身高增長速度為每年8.2 厘米、6 個月和12 個月時每年8 厘米，與中度PGHD 人群的歷史增長率一致。

The difference in HV. six and 12 months between the optimal 1.6 megs per kg room to a one dose.

高壓的差異。 6 個月到 12 個月之間，最佳劑量為每公斤 1.6 毫克到一劑。

And the recombinant growth hormone comparator arm fell within the noninferiority margin of less than two centimeters a year, and that's a criterion recently used for FDA approval.

重組生長激素比較臂的非劣效性幅度每年不到兩厘米，這是 FDA 最近用於批准的標準。

Although our growth to 10 was not specifically designed to establish noninferiority, we are pleased to note that the growth outcomes observed in this study are in line with the non-inferiority thresholds seen in recent successful FDA applications.

儘管我們成長到 10 並不是專門為了確定非劣效性而設計的，但我們很高興地註意到，本研究中觀察到的成長結果與最近成功的 FDA 申請中看到的非劣效閾值一致。

These findings enable us to design a successful Phase three trial to show non-inferiority against the daily injectable growth hormone comparator arm.

這些發現使我們能夠設計成功的第三階段試驗，以顯示相對於每日註射生長激素比較組的非劣效性。

Now the preliminary 24 months ago to a one data we presented in November, we've gathered from a subset of the world growth to tend to a patient or participants who met the protocol requirements for an extension beyond 12 months, combined with or over 12 subjects.

現在是24 個月前我們在11 月提供的一項數據的初步數據，我們從世界增長的一個子集中收集了數據，以照顧符合協議要求的患者或參與者，延長超過12 個月，並結合或超過12 個月。科目。

The data revealed a sustained effect showing a minimal decrease of approximately 6% in annualized height velocity from year one to year two as opposed to a significant decline of approximately 20% reported for daily common and growth hormone in moderate PGHD patients.

數據顯示，中度 PGHD 患者的年化身高速度從第一年到第二年的年化身高速度略有下降約 6%，而據報道，每日普通激素和生長激素顯著下降約 20%。

We'll go to Kent successfully achieved its prespecified primary endpoint, validating our predictive enrichment marker or pen test, while also meeting the secondary endpoint by demonstrating a 100% reproducibility of PAM positive classification.

我們將前往肯特成功實現其預先指定的主要終點，驗證我們的預測富集標記或筆測試，同時透過展示 PAM 陽性分類的 100% 再現性來滿足次要終點。

Additional data from the ORBIT two trial demonstrated that only 20% growth hormone concentration was needed to achieve comparable annualized height velocity that suggests that move to a one is more efficient than exogenous growth hormone to promote growth.

ORBIT 兩項試驗的其他數據表明，只需 20% 的生長激素濃度即可達到可比較的年化身高速度，這表明使用 1 比外源生長激素更能有效促進生長。

These data confirm the importance of Bloom tool one's unique mechanism of action to restore the natural foods, the physiology of pulsatile gross growth hormone secretion.

這些數據證實了Bloom工具獨特的作用機制對於恢復天然食物、脈動性生長激素分泌的生理機能的重要性。

Our data from both trials provided a clear safety profile for investigational tool one following the release of top line results in November, ongoing data collection and analysis have continued.

繼 11 月發布頂線結果後，我們的兩項試驗數據為研究工具一提供了明確的安全性概況，持續的數據收集和分析仍在繼續。

We anticipate presenting comprehensive 12-month annualized height velocity data from the oral growth to 10 trial in the second quarter of this year, most likely at a major medical conference.

我們預計在今年第二季（最有可能在一次重要的醫學會議上）提供從口腔生長到 10 項試驗的全面的 12 個月年化身高速度數據。

This dataset will include 12 month HV. data on all enrolled patients from zero to 10 trial.

該數據集將包括 12 個月的 HV。從 0 到 10 次試驗的所有入組患者的數據。

Additional 24 month data beyond the dataset we announced in November will also be available.

除了我們 11 月宣布的數據集之外，還將提供額外的 24 個月數據。

We expect to present these data in additional analysis at several meetings throughout 2024 to capitalize on the growing interest and excitement in the pediatric endocrine community about moving to a one as potentially the first oral therapeutic in this population.

我們預計將在 2024 年的幾次會議上進行額外分析，以利用兒科內分泌界對將藥物作為該人群中第一種口服治療藥物的興趣和興奮不斷增長的機會。

Following the announcement of our top line results, we've been diligent conducting additional analyses in preparation for an end-of-Phase two meeting with the FDA scheduled for the second quarter.

在公佈我們的主要結果後，我們一直在努力進行額外的分析，為定於第二季與 FDA 舉行的第二階段結束會議做準備。

Our data package for this meeting is comprehensive, featuring a larger dataset compared to other growth hormone counterparts at this developmental St. These data show them to one induces annualized height velocity is consistent with historical levels for moderate PGHD on recombinant growth hormone data also demonstrate that oral Bloom to a one produces a robust and enduring response at 1.6 mix per kick dose, making use of our predictive enrichment marker or payment strategy for selecting suitable moderate PGHD patients, thereby further derisking our Phase three program.

我們本次會議的數據包非常全面，與該發育聖地的其他生長激素同行相比，具有更大的數據集。這些數據表明，重組生長激素數據的年化身高速度與中度PGHD 的歷史水平一致，也顯示口服 Bloom 在每劑量 1.6 混合時產生強烈且持久的反應，利用我們的預測富集標記或支付策略來選擇合適的中度 PGHD 患者，從而進一步降低我們第三階段計劃的風險。

As a result, we have posted in the Phase two meeting with a high level of confidence and long before we announced top line results from our oral drug trials.

因此，早在我們宣布口服藥物試驗的主要結果之前，我們就充滿信心地在第二階段會議上發布了資訊。

We've been actively involved in advanced planning for a pivotal Phase three trial following our Phase two meeting with the FDA we will finalize the ultimate design of the trial, which we anticipate initiating in the fourth quarter of 2024.

在與 FDA 舉行第二階段會議後，我們一直積極參與關鍵第三階段試驗的高級規劃，我們將最終確定試驗的最終設計，預計於 2024 年第四季啟動。

We believe that this trial's results will support a new drug application for limb tool, one in the PJSD. indication with data maternity or Wally, our CFO, for a brief overview of our financial results.

我們相信，這項試驗的結果將支持肢體工具的新藥物應用，即 PJSD 中的一種。請與 data maternity 或我們的財務長 Wally 聯繫，以了解我們的財務表現的簡要概述。

Thanks, Lumos Pharma ended the year December 31st, 2023, with cash, cash equivalents and short-term investments totaling $36.1 million compared to $67.4 million on December 31st, 2022 due cash on hand as of December 31st, 2023, is expected to support operations through the third quarter of 2024, inclusive of our Phase three preparations and other operational activities, initiation of our Phase three clinical trial by end of 2024, subject to securing financing in the near term for research and development expenses were $22.1 million, an increase of $4.2 million for the year ended December 31st, 2023 compared to the same period in 2022 primarily due to increases of $3.3 million in clinical trial expenses, $0.9 million in contract manufacturing expenses, $0.2 million in consulting expenses and $0.2 million in other expenses, partially offset by a $0.4 million decrease in personnel-related expenses.

謝謝，截至2023 年12 月31 日，Lumos Pharma 的現金、現金等價物和短期投資總額為3,610 萬美元，而2022 年12 月31 日為6,740 萬美元，截至2023 年12 月31 日，手頭現金預計將支援營運截至2024 年第三季度，包括我們的第三期準備工作和其他營運活動，到2024 年底啟動我們的第三期臨床試驗（以近期獲得融資為條件），研發費用為2,210 萬美元，增加了截至2023年12月31日的年度與2022年同期相比增加了420萬美元，主要是由於臨床試驗費用增加了330萬美元，合約製造費用增加了90萬美元，諮詢費用增加了20萬美元，其他費用增加了20萬美元，部分抵銷人事相關費用減少 40 萬美元。

General and administrative expenses were $16.6 million, an increase of $0.9 million for the year ended December 31st, 2023 compared to the same period in 2022, primarily due to increases of $0.5 million in personnel-related expenses, $0.4 million in royalty expenses paid to the Public Health Agency of Canada and $0.1 million in travel expenses, partially offset by a $0.1 million decrease in other expenses.

截至2023 年12 月31 日的年度，一般及行政費用為1,660 萬美元，較2022 年同期增加90 萬美元，主要是由於人事相關費用增加50 萬美元，支付給公司的特許權使用費增加40 萬美元。加拿大公共衛生署和 10 萬加元的差旅費用，部分被其他費用減少 10 萬加元所抵消。

Net loss for the year ended December 31st, 2023 was $34 million compared to a net loss of $31.1 million for the same period in 2022.

截至 2023 年 12 月 31 日止年度的淨虧損為 3,400 萬美元，而 2022 年同期的淨虧損為 3,110 萬美元。

We ended Q4 2023 with 8,102,555 shares outstanding.

截至 2023 年第四季度，我們已發行 8,102,555 股股票。

And with that, I will turn it back to Rick for his closing remarks.

接下來，我將把它轉回給里克，讓他作結束語。

Very short and to conclude, we're absolutely thrilled by the progress of our program, David and by the excitement among the investment community.

非常簡短的結論是，大衛，我們對我們計劃的進展以及投資界的興奮感到非常興奮。

Regarding the potential for the first oral therapy for PGHD, we've been on the podium presenting data on numerous endocrine conferences over the past two years.

關於第一個口服治療 PGHD 的潛力，我們在過去兩年的許多內分泌會議上發表了數據。

Have seen the overwhelming acceptance of and excitement for our oral loom tool, one for confident in the distinct advantages offered by them to a one for patients with moderate PGHD and enthusiastic about advancing to the next developmental stage for this asset.

我們看到了人們對我們的口腔織布機工具的壓倒性接受和興奮，人們對它們為中度 PGHD 患者提供的獨特優勢充滿信心，並熱衷於將該資產推進到下一個發展階段。

We firmly believe that the dataset we are submitting to the FDA for upcoming into Phase two meeting provides ample support for a pivotal trial linked to a one at the 1.6 megs per kg dose, affirming its potential as a viable alternative to injectable products for appropriately selected moderate PGHD patients, and we eagerly anticipate sharing further insights from our growth trials throughout this year and keeping you abreast of our progress.

我們堅信，我們向FDA 提交的即將進入第二階段會議的數據集為一項與1.6 兆每公斤劑量的關鍵試驗相關的關鍵試驗提供了充分的支持，證實了其作為適當選擇的注射產品的可行替代品的潛力中度 PGHD 患者，我們熱切期待分享我們今年生長試驗的更多見解，並讓您及時了解我們的進展。

So thank you for your attention, everyone.

謝謝大家的關注。

And operator, we can now open this up for questions.

接線員，我們現在可以開放提問。

Team.

團隊。

Ladies and gentlemen, to ask a question, you will need to press star one one on your telephone and wait planning to be announced to withdraw your question, simply press star one.

女士們，先生們，要提問，您需要在電話上按星一一，然後等待宣布撤回您的問題，只需按星一即可。

Once again, please stand by while we compile the Q&A roster.

再次請大家耐心等待，我們正在整理問答名單。

Yes.

是的。

And our first question coming from the line of Charles Duncan, Cantor you lend itself.

我們的第一個問題來自查爾斯鄧肯（Charles Duncan），坎托（Cantor），你自然而然。

Yes.

是的。

Good afternoon, Rick and team.

下午好，瑞克和團隊。

Congrats on the progress last year.

祝賀去年的進步。

Looking forward to hearing more updates here in the near term.

期待在短期內聽到更多更新。

Thanks for taking our question.

感謝您提出我們的問題。

I had a key question about the end-of-Phase two meeting with agency.

我有一個關於與機構的第二階段結束會議的關鍵問題。

Is it actually scheduled and can you provide us some insight on what the key question is, is it is it really something that needs to be answered?

它實際上是預定的嗎？您能否向我們提供一些關於關鍵問題的見解，這真的是需要回答的問題嗎？

Or do you feel like this is more a point of execution in terms of going forward?

還是你覺得這更像是未來的執行點？

And then when would you anticipate being able to share the outcome, would it be post the meeting minutes or do you think it will be pretty straightforward and you'll share with us shortly after the meeting?

然後您預計什麼時候能夠分享結果，是發布會議記錄還是您認為這會非常簡單，您會在會議後不久與我們分享？

I'm going to ask a good question, Charles, and thank you for.

我要問一個好問題，查爾斯，謝謝你。

I'm going to turn that question over to John McCue.

我要把這個問題轉給約翰·麥庫。

Yes.

是的。

Thanks, Chuck.

謝謝，查克。

And Charles.

還有查爾斯。

I appreciate the question.

我很欣賞這個問題。

So before we started our Phase three study, we really would like we use this end of Phase two meeting to take everything we've learned from Phase two data that we've read out and we've put that into our Phase three protocol.

因此，在我們開始第三階段研究之前，我們真的希望利用第二階段會議結束時的時間，從我們讀出的第二階段數據中學到的一切，並將其納入我們的第三階段協議中。

And we want to walk away from that meeting with agreement on the specifics of that protocol.

我們希望在會議結束時就該協議的具體細節達成一致。

So I think it's a very important time for us to talk through any questions the agency has on the data as well as come to agreement on how on the path to move forward.

因此，我認為現在是我們討論該機構對數據的任何問題並就如何前進的道路達成一致的非常重要的時刻。

So it is will be a very it will be a good step forward for us when we get through that.

因此，當我們度過難關時，這對我們來說將是向前邁出的一大步。

And we have agreement on the Phase three protocol.

我們就第三階段協議達成了一致。

So we expect to communicate that information the outcome so that when when we hear back from them and the FDA probably in the meeting minutes, right?

因此，我們希望傳達這些訊息和結果，以便當我們收到他們和 FDA 的回應時可能會在會議記錄中，對嗎？

So we said that kind of timeframe was in Q2 of this year.

所以我們說這樣的時間範圍是在今年第二季。

Okay.

好的。

And can you tell them I might add is that there are a lot of historical precedence here.

你能告訴他們嗎？我可能要補充的是，這裡有很多歷史先例。

We're we feel confident about this meeting in a really an outstanding briefing book.

我們對這次會議充滿信心，這是一本非常出色的簡報。

I think that historically to these studies have been 12 months non-inferiority trials against an active comparator on the non-inferiority margin historically has been 1.8 to 2 centimeters.

我認為，從歷史上看，這些研究針對主動比較者進行了 12 個月的非劣效性試驗，歷史上的非劣效性幅度為 1.8 至 2 公分。

And um, we also expect to have a randomization to the 1.6 mix per take a day or two to one randomization to growth hormone.

嗯，我們還期望每天或兩天對 1.6 種混合進行隨機化，對生長激素進行隨機化。

I think most of these states have been about 200 subjects, and we expect that a similar design.

我認為這些狀態中的大多數已經有大約 200 個科目，我們期望有類似的設計。

And also, of course, we um, we validated our PEM strategy.

當然，我們驗證了我們的 PEM 策略。

I think we de-risk our program consisted of pretty considerably given the fact that we can select patients, we believe will be our drug will be effective in that as some moderate growth hormone deficient patients.

我認為我們的計劃包括相當大的風險，因為我們可以選擇患者，我們相信我們的藥物對於一些中度生長激素缺乏的患者將是有效的。

So we're pretty confident about our meeting with the our community meeting with the FDA.

因此，我們對與 FDA 的社區會議非常有信心。

It's helpful.

這很有幫助。

If I could just ask a follow-on question to that.

如果我能問後續問題就好了。

Those two observations.

這兩個觀察結果。

Rick, would you anticipate the pen strategy to help you inside and conduct a smaller sample size or conduct one about that size, would you know, perhaps greater confidence in that symptom tonight?

里克，您是否期望筆策略可以幫助您內部進行較小的樣本量或進行大約該大小的樣本，您知道嗎，也許今晚對這種症狀更有信心？

John, do you want to go there.

約翰，你想去那裡嗎？

Yes.

是的。

So I think the how we view the Pentacel that's going to kind of raise our efficiency rate, we're going to be able to bring in subjects that are likely responders to our molecule.

所以我認為我們如何看待 Pentacel 會提高我們的效率，我們將能夠引入可能對我們的分子有反應的受試者。

So I don't think it's going to reduce that size that will make it a more efficient trial at site.

因此，我認為縮小規模不會使其成為更有效的現場試驗。

Okay.

好的。

First question in terms of business development activity at one time.

關於業務發展活動的第一個問題。

You talked about possibly ex U.S. partnering.

您談到了可能與美國前的合作夥伴關係。

I guess I'm wondering if you have any further thoughts on that.

我想我想知道你對此是否有任何進一步的想法。

And then I'll hop back in the queue.

然後我會跳回隊列中。

Thanks.

謝謝。

Yes, thanks for that question, Charles.

是的，謝謝你的提問，查爾斯。

And assure I think that you can imagine as the first oral product in this very large global market, there are certain ex U.S. regional markets that in companies that have a job, we completed an outreach to us or vice versa, and we continue with those discussions and at the appropriate time, I think that we will partner with anyone who's going to go to.

並保證我認為你可以想像作為這個非常大的全球市場中的第一個口服產品，有某些美國以外的區域市場，在有工作的公司中，我們完成了對我們的外展，反之亦然，我們繼續這些討論並在適當的時候，我認為我們將與任何要去的人合作。

We believe it's going to be a good partner in those markets.

我們相信它將成為這些市場的良好合作夥伴。

Obviously, this will allow us to bring in some non-dilutive money.

顯然，這將使我們能夠帶來一些非稀釋資金。

And I think that's one of the goals that we would have for ourselves.

我認為這是我們為自己設定的目標之一。

Okay.

好的。

Thanks for taking the questions.

感謝您提出問題。

Yes, thank you.

是的，謝謝。

And our next question coming from the line of fill-in Gershell with Oppenheimer.

我們的下一個問題來自格謝爾與奧本海默的補充。

Yolanda Shelton.

約蘭達·謝爾頓。

Thank you, Rick and team for the update from just a question from from me.

感謝 Rick 和團隊根據我的問題提供的最新資訊。

With respect to additional indication potential for two oh one as we think about use of growth hormone products today and indications such as Turners and other such disorders.

當我們考慮當今生長激素產品的使用以及諸如特納氏症和其他此類疾病的適應症時，就兩對一的額外適應症潛力而言。

Just wondering if you could comment on any plans down the road to explore to a one's potential to have to be approved for those additional label indications.

只是想知道您是否可以評論任何計劃，以探索一個人必須獲得批准才能獲得這些附加標籤適應症的潛力。

Thank you.

謝謝。

And John, I'm going to let you start with that as an answer and thank you for the question.

約翰，我將讓你以此作為答案，並感謝你提出的問題。

Li-lan.

麗蘭.

We spent a lot of time lead and thinking through them the best opportunities to advance lumateperone and pass PGHDPJC. is a great opportunity, I think for us to show the effect of the molecule on restoring natural pulsatility and we can take that same mechanism and philosophy.

我們花了很多時間領導並思考推進 Lumateperone 並通過 PGHDPJC 的最佳機會。這是一個很好的機會，我認為我們可以展示分子對恢復自然脈動的影響，我們可以採用相同的機制和概念。

It applies to many of the other 11 indications that are approved for home for injectable growth hormone.

它適用於批准用於家庭注射生長激素的其他 11 種適應症中的許多適應症。

And so I think we were getting to the point where we have the data set where we can start to to think about how to move forward.

所以我認為我們已經有了數據集，我們可以開始思考如何前進。

But we have not yet made any decisions on the exact next steps that we're going to take.

但我們尚未就下一步具體採取的步驟做出任何決定。

But we have I think the data and I've done the background research on each one of those indications to make it make some good decisions shortly.

但我認為我們有數據，並且我已經對每個跡象進行了背景研究，以使其盡快做出一些好的決定。

And we will also recover some recall, we've got a politic pilot program underway in a broader cardio-metabolic opportunity of nonalcoholic fatty liver disease at MGH now as an investigator initiated trial.

我們還將恢復一些記憶，我們正在麻省總醫院開展一項針對非酒精性脂肪肝疾病更廣泛的心臟代謝機會的政治試點計劃，目前一名研究人員已啟動試驗。

But this investigator did demonstrate with a in a recombinant growth hormone trial in this indication, some pretty significant reductions in liver fat in this population.

但這位研究人員確實透過重組生長激素試驗證明了該族群的肝臟脂肪顯著減少。

So we're pretty excited to have and to work with this investigator.

因此，我們很高興能有這位調查員並與他合作。

I think it's a little premature right now to talk about anything beyond that, but we're always looking for ways to increase shareholder value with additional indications.

我認為現在談論除此之外的任何事情還為時過早，但我們一直在尋找透過其他跡象來增加股東價值的方法。

Got it, Rick, Rick.

明白了，瑞克，瑞克。

So at this point, we don't we don't have a view on when she may have data on her her study of Tier one, correct.

因此，在這一點上，我們不知道她何時可以獲得第一層學習的數據，正確的。

I know I know she doesn't have the trial fully enrolled yet, and it's a six month trial.

我知道她還沒有完全註冊試驗，這是一個為期六個月的試驗。

So we haven't really talked about that yet in the marketplace.

所以我們還沒有在市場上真正討論過這個問題。

Okay.

好的。

So stay tuned and look forward to hearing updates following your afternoon and thank you.

因此，請繼續關注並期待在下午之後聽到最新消息，謝謝。

Yes.

是的。

Thank you to our next question coming from the line of Ed White, H.C. Wainwright.

感謝 Ed White, H.C. 提出我們的下一個問題。溫賴特。

Your line is open.

您的線路已開通。

Hi.

你好。

Thanks for taking my questions.

感謝您回答我的問題。

So how should we be thinking about the Phase three start in the fourth quarter, or is that just when you will be in opening sites?

那麼，我們該如何考慮第四季開始的第三階段，或者說這正是你們開放工地的時間？

Or do you intend or do you think that you can actually treat patients in during the fourth quarter.

或者您是否打算或認為您可以在第四季度真正治療患者。

Okay.

好的。

Thank you for that question.

謝謝你提出這個問題。

And do will you answer that for us?

您願意為我們回答嗎？

I think you're on mute.

我認為你處於靜音狀態。

Thank you.

謝謝。

Yes, sorry, about that, I think gives us very good crushing rate.

是的，抱歉，關於這一點，我認為給我們帶來了非常好的破碎率。

As of now, we do diligence, like John mentioned, right, we already have a plan for the Phase three plan.

現在我們盡職盡責，就像John提到的，對吧，我們已經有了第三階段的計劃。

We get ready to really discuss with the FDA when we have planned.

當我們計劃好後，我們就準備好與 FDA 進行真正的討論。

In the meantime, we do a prep work what we need to get done in regards to have a time line that would be proposed rates.

同時，我們做了一些準備工作，以確定建議費率的時間表。

So we think that by the end of this year, we'd be able to be able to screen the subject and get, you know, the time line enrolled like what we proposed in the past.

因此，我們認為，到今年年底，我們將能夠篩選該主題，並獲得像我們過去提議的那樣的註冊時間線。

And I feel very optimistic because, as Rick mentioned earlier, that we have overwhelming requests from the KOLs around the world because they learn about our Phase two results.

我感到非常樂觀，因為正如 Rick 之前提到的，我們收到了來自世界各地 KOL 的大量請求，因為他們了解了我們的第二階段結果。

And they do believe that this can be a great potentially the new drug therapy for their patient, the first oral therapy.

他們確實相信，這對他們的患者來說是一種具有巨大潛力的新藥物療法，也是第一種口服療法。

So we do believe that you know, a lot investigator and actually some of those participated in our Phase two trial and some news to loom tool one.

所以我們確實相信你知道，很多研究人員，實際上其中一些人參與了我們的第二階段試驗，還有一些關於工具一的消息。

However, you know, with their request of participation, we do believe the time line one we get these are we up and running and we have a final, you know, site identify I think that the rules are going to go very quickly.

然而，你知道，根據他們的參與請求，我們確實相信我們得到的時間表是我們啟動並運行的，我們有一個最終的，你知道的，網站標識，我認為規則將很快實施。

And as we proposed in the plan in the past.

正如我們過去在計劃中提出的。

And as you know, obviously we have to we have to finalize the Phase three protocol.

如您所知，顯然我們必須完成第三階段協議。

We're obviously working with our vendor partners very actively.

顯然，我們正在非常積極地與供應商合作夥伴合作。

It's good to be on.

能上場真是太好了。

We've been active in all of the podium at all these of endocrine meetings around the world, and we've contacted and direct contact with was really experienced investigators.

我們一直活躍在世界各地所有內分泌會議的所有講台上，並且我們已經與真正經驗豐富的研究人員進行了接觸和直接接觸。

As a result.

因此。

There's a there's a lot more work that has to be done in terms of qualifying and activating the sites.

在資格和激活站點方面還有很多工作要做。

And of course, we have our Phase three drug product manufactured and ready to go.

當然，我們已經製造出第三階段藥品並準備就緒。

So we will have a ready go.

所以我們會做好準備。

And so all the preparation that needs to be done, we're doing right now and we hope to have that deal plenty of patients screened in the study for the end of the year.

因此，我們現在正在做所有需要做的準備工作，我們希望在今年年底的研究中對大量患者進行篩檢。

Okay.

好的。

Thanks, Rick, for taking my question.

謝謝瑞克回答我的問題。

Sure.

當然。

And our next question coming from the line of Catherine Novack with Jones Research.

我們的下一個問題來自瓊斯研究中心的凱瑟琳·諾瓦克。

Your line is open.

您的線路已開通。

Oh, hi, good afternoon, guys.

哦，嗨，大家下午好。

Just one question.

只有一個問題。

Can you remind us of what percent of the OraGrowtH patients enrolled were boys versus girls.

您能否提醒我們一下，登記的 OraGrowtH 患者中男孩和女孩的百分比分別是多少？

And there's a difference between how these two populations might be managed by pediatric endocrinologists in a sense that there and could potentially encourage patients who might not choose to have a daily injection trial and oral growth hormone therapy instead.

從某種意義上說，兒科內分泌學家對這兩個人群的管理方式存在差異，這可能會鼓勵那些可能不選擇每日註射試驗和口服生長激素治療的患者。

And Duke once you answer that question.

一旦你回答了這個問題，杜克就會。

Thanks.

謝謝。

Appreciate the question, Katherine.

感謝這個問題，凱瑟琳。

Yes, thank you, Catherine.

是的，謝謝你，凱瑟琳。

Decimate.

消滅。

Good questions.

好問題。

All applies very, you know, kind of unique, Ray, as you know, when you look at most of Phase three trial, especially long acting Ascendis, Novo Pfizer, majority of those subjects enrolled in a trial of male predominant right in our study is pretty much that, you know, mail a little bit more than female.

所有這些都適用，你知道，有點獨特，雷，正如你所知，當你看到大部分第三階段試驗時，特別是長效Ascendis、諾和輝瑞(Novo Pfizer)，大多數受試者參加了我們研究中男性主導的試驗你知道，郵件比女性多一點。

That's not that choose different, which is very, very interesting.

這並不是說選擇不同，這是非常非常有趣的。

I think when we go back to the first global Phase three trial, you can see that, you know, the number of countries going to increase the number of cycles increase because you know the time why we want to enroll.

我認為，當我們回到第一個全球第三階段試驗時，您可以看到，要增加週期數的國家數量會增加，因為您知道我們想要註冊的時間。

We have a goal that, you know, we want to complete enrollment within 15 to 18 months, which is again, this goal, it's actually that genome better than the goal that those previous Phase three trial have been running.

我們有一個目標，你知道，我們希望在 15 到 18 個月內完成註冊，這又是這個目標，實際上基因組比之前進行的第三階段試驗的目標更好。

Part of it is that, you know, that's during COVID.

部分原因是，你知道，那是在新冠疫情期間。

And now we don't have COVID and not to mention that we do not expect a lot of competitor to run the Phase three trial.

現在我們沒有新冠病毒，更不用說我們預計不會有許多競爭對手進行第三階段試驗。

Right.

正確的。

And in good regards to do you know, the oral, I think deck too, to enroll this subject, I heard loud and clear when they start to face true tried to increase enrollment in a timely manner.

向你致以良好的問候，你知道嗎，口頭的，我認為也是為了招收這個科目，當他們開始面對真正的嘗試及時增加招生時，我聽到了響亮而清晰的聲音。

We realize that a lot of subject really interested to participate in these oral argument or one trial?

我們意識到很多受試者真的有興趣參加這些口頭辯論或一場審判嗎？

Again, you know, I don't think that we expect any, but this issue of you know, give the patient, an ROE, especially most of them would like to be able to be on this drug and especially during the clinical trial because it would take years for us to really get the drug approved.

再說一次，你知道，我認為我們不會期望任何結果，但是你知道的這個問題，給患​​者一個 ROE，特別是他們中的大多數人希望能夠使用這種藥物，特別是在臨床試驗期間，因為我們需要數年時間才能真正使該藥物獲得批准。

I hope that that answers the question.

我希望這能回答問題。

Yes.

是的。

Yes, that's very helpful.

是的，這非常有幫助。

And actually, just one more on the end-of-Phase two meeting we've heard over and over from KOLs that growth beyond 12 months is really the most important aspect to them.

事實上，在第二階段結束會議上，我們一遍又一遍地從 KOL 那裡聽到，超過 12 個月的成長對他們來說確實是最重要的方面。

And are these data going to be a part of your discussion with FDA and how important is that to the for the Phase three trial design for that?

這些數據是否會成為您與 FDA 討論的一部分？這對於第三階段試驗設計有多重要？

That is a very good question.

這是一個很好的問題。

Katherine, among ask John to answer.

凱瑟琳等人請約翰回答。

Yes.

是的。

Thank you, Katherine.

謝謝你，凱瑟琳。

So I think the data that we've shown about the small subset of kids that we have on 24 months on growth has been very telling the restoration of normal growth hormone pulsatility, normal IGF-1 levels and FeNO more natural growth.

因此，我認為我們展示的關於一小部分兒童 24 個月生長情況的數據非常能說明正常生長激素脈動、正常 IGF-1 水平和 FeNO 恢復的正常生長情況。

That results from that has led to very consistent and durable growth comparing year to year one, right, we lose only a small tiny bit 6% of our HV. compared to a much larger drop-off that you see with daily injectable growth hormone.

由此產生的結果是，與第一年相比，我們實現了非常一致和持久的成長，對吧，我們只損失了 6% 的 HV。與每天注射生長激素時看到的更大的下降相比。

So that is a really important piece of data that come.

所以這是一個非常重要的數據。

We certainly will chat with the FDA about.

我們當然會與 FDA 討論。

And we do anticipate much, as Rick said earlier, standard Phase three trial design of 12 months.

正如 Rick 之前所說，我們確實對為期 12 個月的標準三期試驗設計抱有很大期望。

But because we have a number of subjects already that have gone past but 12 month timeframe.

但因為我們已經有許多科目已經過了 12 個月的時間範圍。

We're moving subjects come from our 24-month loan, our growth to 10 study into a long-term safety extension.

我們正在將 24 個月的貸款、我們的研究成長到 10 個月的研究對象轉移到長期安全擴展。

We will have quite a lot of longer-term data by the time we get to Phase three and get to an NDA.

當我們進入第三階段並達成保密協議時，我們將獲得大量長期數據。

So I think that data will be very helpful in addition to what we see in the Phase three 12 one study that Rick described.

因此，我認為除了我們在 Rick 描述的第三階段 12 一研究中看到的數據之外，這些數據也將非常有幫助。

So it is very important and it will be a very nice package of dermal data that Brian.

所以這非常重要，布萊恩說這將是一個非常好的皮膚數據包。

Great that that's very helpful.

太好了，這非常有幫助。

Thanks.

謝謝。

Thanks a lot, guys.

非常感謝，夥計們。

Thanks, Ken.

謝謝，肯。

And I'm showing no further questions in queue at this time.

目前我沒有在隊列中顯示任何其他問題。

Ladies and gentlemen, this concludes the Lumos Pharma Fourth Quarter 2023 earnings call.

女士們先生們，Lumos Pharma 2023 年第四季財報電話會議到此結束。

Thank you and Advocat.

謝謝你和倡議者。