Praxis Precision Medicines Inc (PRAX) 2024 Q3 法說會逐字稿

Good day and thank you for standing by. Welcome to the Practice Precision Medicines third quarter, 2024 corporate update. (Operator Instructions) I would now like to hand the conference over to your speaker today, Dan Ferry from Lifesci.

您好，感謝您的支持。歡迎閱讀《精準醫療實務》2024 年第三季公司更新。（操作員指示）現在，我想將會議交給今天的發言人，來自 Lifesci 的 Dan Ferry。

Good morning and welcome to the Practice Precision Medicines third quarter, 2024 financial results and business update conference call.

早安，歡迎參加 Practice Precision Medicines 2024 年第三季財務業績和業務更新電話會議。

This call is being webcast live and can be accessed on the investors section of the Practice website at www. Practice medicines.com.

本次電話會議將進行網路直播，您可以透過 Practice 網站 www. 的投資者專區觀看。實踐醫學.com。

Please note that remarks made during this call may contain forward-looking statements within the meeting of the Private Securities Litigation Reform Act of 1,995.

請注意，本次電話會議中的言論可能包含 1995 年《私人證券訴訟改革法案》範圍內的前瞻性陳述。

These may include statements about the company's future expectations and plans, clinical development timelines and financial projections.

這些可能包括有關公司未來預期和計劃、臨床開發時間表和財務預測的聲明。

While these forward-looking statements represent practices view as of today, they should not be relied upon as representing the company's views in the future. Practice may update these statements in the future but is not taking on an obligation to do so.

雖然這些前瞻性陳述代表了今天的實踐觀點，但不應將其視為代表公司未來的觀點。實踐中可能會在未來更新這些聲明，但沒有承擔這樣做的義務。

Please refer to practices. Most recent filings with the securities and exchange commission for discussion of certain risks and uncertainties associated with the company's business during the call. Today are Marcio Souza, President and Chief Executive Officer of Practice and Tim Kelly, Chief Financial Officer. After providing updates on our key programs, there will be a brief question and answer session with that. It's my pleasure to turn the call over to Marcio.

請參考做法。最近向美國證券交易委員會提交的文件，用於在電話會議中討論與公司業務相關的某些風險和不確定性。今天出席的是 Practice 總裁兼執行長 Marcio Souza 和財務長 Tim Kelly。在提供我們的主要專案的最新進展後，將會有一個簡短的問答環節。我很高興將電話轉給馬西奧。

Thank you, good morning and welcome to the Practice third quarter, 2024 conference call. This best squad to remain laser focused on advancing our pipeline as we gear up for next year to have four programs in registration totaling to a substantial multi billion dollar opportunity.

謝謝，早安，歡迎參加 2024 年第三季實務電話會議。這支最優秀的團隊將繼續專注於推進我們的產品線，為明年註冊四個項目做好準備，這些項目總額將達到數十億美元。

The phase three study in essential tremor essential three for lead program, Ulis account mind continues to progress. Well, we have confirmed all aspects of the internet analysis and are now updating the plans to have the results in Q1 2025.

特發性震顫的第三期臨床研究正在進行中，Ulis 博士表示，這項研究仍在持續進展。好吧，我們已經確認了互聯網分析的所有方面，現在正在更新計劃，以便在 2025 年第一季獲得結果。

Both studies are well powered and controlled for success because there's a range of outcomes for each study as well as the interim analysis. In the coming months, we decided that we will only share an update on timing for both study one and study two. Once we have evaluated the recommendation from the interim review board for the interim analysis in Q3. We're very excited to report the positive top line results for another asset in our pipeline Vormatrigine in the phase two NB trial in CN two A and CN eight A G.

這兩項研究都具有良好的效力和控制力，因為每項研究以及中期分析都有一系列結果。在接下來的幾個月裡，我們決定只分享第一項研究和第二項研究的時間更新。一旦我們評估了中期審查委員會對第三季中期分析的建議。我們非常高興地報告，我們產品線中的另一項資產 Vormatrigine 在 CN two A 和 CN eight A G 的二期 NB 試驗中取得了積極的頂線結果。

In the 15 patient study liturgy demonstrated an impressive 46% reduction in motor seizures versus placebo with third of the patients achieving an unprecedented seizure free status based on those results, we initiated a second registrational cohort of the study which has already started screening patients just weeks after completion of the prior cohorts in common Vormatrigine or metro previously known as practice 6 to 8 is starting out of the gate. Strong in all areas of our Comprehensive Energy Clinical Program.

在對 15 名患者進行的研究中，禮儀證明與安慰劑相比，運動性癲癇發作減少了 46%，令人印象深刻，其中三分之一的患者達到了前所未有的無癲癇發作狀態。基於這些結果，我們啟動了該研究的第二個註冊隊列，該隊列已在完成前幾組常見的 Vormatrigine 或 metro（以前稱為實踐 6 至 8）後僅幾週就開始對患者進行篩選。我們綜合能源臨床計劃的所有領域都表現出色。

The innovative observational study and power, a first of its kind in collaboration with the epilepsy study consortium launched in the third quarter in this short period of time, attracted the interest of over 1,000 patients who registered in the study.

這項創新的觀察性研究和成果是與第三季短時間內啟動的癲癇研究聯盟合作進行的首項此類研究，吸引了超過 1,000 名註冊參與研究的患者的興趣。

We expect the key learnings for empower to impact the entire energy program.

我們期望授權的關鍵經驗能夠影響整個能源計劃。

The phase two radiant and the phase 23 power one trials are on track for top line results next year, rounding out our portfolio as a nurse and begin dosing patients in Brazil in the second quarter for the EMBRAVE study. And we continue to engage with regulatory agencies in Europe and in the us to finalize the development plans in a two gain of function patients with our strong balance sheet, we continue to be fully funded as we pursue our vision to deliver precision therapies for patients with CNS disorders.

第二階段輻射試驗和第 23 階段動力一試驗預計在明年取得頂線結果，完善我們作為護理師的投資組合，並將於第二季度開始在巴西為患者進行 EMBRAVE 研究給藥。我們將繼續與歐洲和美國的監管機構合作，最終確定兩項功能獲得患者的開發計劃，憑藉我們強大的資產負債表，我們將繼續獲得充足的資金，以追求為中樞神經系統疾病患者提供精準治療的願景。

Let me now focus some more on Alexa, our innovative essential three program in et is the biggest and most comprehensive program conducted. Today.

現在讓我更專注於 Alexa，這是我們在 et 中創新的三大基本計劃，也是迄今為止最大、最全面的計劃。今天。

We began recruiting for the two phase three studies just about one year ago and have seen tens of thousands of patients interested in participating.

大約一年前，我們開始招募兩個第三階段研究的患者，並且已經看到數以萬計的患者有興趣參與。

This vibrant participation highlights the significance and met needs for the millions of patients with essential tremor in their physicians and caregivers who are seeking a therapy that will allow patients to perform daily activities without impairments.

這種積極的參與凸顯了數百萬特發性震顫患者對他們的醫生和護理人員的重要性和滿足的需求，他們正在尋求一種可以讓患者在不受損害的情況下進行日常活動的治療方法。

The need for treatment and essential tremor continues to be more defined as we advance this program in a survey we conducted with over 400 patients up to 77% of the respondents said they do not feel their et symptoms are managed with current treatments.

隨著我們推進該計劃，對治療和特發性震顫的需求變得越來越明確，我們對 400 多名患者進行了一項調查，高達 77% 的受訪者表示，他們認為目前的治療無法控制他們的特發性震顫症狀。

In a separate survey we conducted with 150 treating physicians. They share that 85% of their visits with 50 patients are focused on looking for treatment. Clearly, there is an incredible need here and we look forward to shortly completing the essential three study with the goal of bringing an option to the market as a quick refresher. The essential three program has two simultaneous phase three studies being run concurrently. 31 is a 12 weeks two arm placebo controlled parallel group study and study two is a 12 week randomized study. Both studies used as primary assessment, the change in the modified activity of daily living and they are both run entirely decentralized as in the patient's home rather than at a clinical site.

在另一項調查中，我們對 150 名主治醫生進行了調查。他們表示，在 50 名患者的問診中，85% 都是為了尋求治療。顯然，這裡存在著巨大的需求，我們期待很快完成三項基本研究，目的是為市場提供一種選擇，作為快速複習。基本三期項目有兩個同時進行的第三階段研究。研究 31 是一項為期 12 週的雙組安慰劑對照平行組研究，研究 2 是一項為期 12 週的隨機研究。這兩項研究均以日常生活活動改變作為主要評估，並且都是完全分散進行的，例如在患者家中而不是在臨床現場進行。

We share in our last quarterly call that we decided to trigger a pre plans analysis. When 50 to 75% of the patients have completed the 12 weeks that you want, the analysis will inform us whether we should continue the study throughout completion. If the primary end point is met, to consider seizing the study or to consider enrolling additional patients to ensure it's sufficiently powered for success.

我們在上次季度電話會議上表示，我們決定啟動預先規劃分析。當 50% 到 75% 的患者完成所需的 12 週治療後，分析結果將告知我們是否應該繼續完成研究。如果達到主要終點，則考慮繼續研究或考慮招募更多患者，以確保其具有足夠的成功動機。

Based on the expectation for the sufficient number of patients who complete the study cleaning of the data execution of the statistical testing and analysis by an independent boards and our internal operations. As well as considering the operational impacts in the study. Completion of 32 we will be finalizing the internet analysis in the first quarter of 2025.

基於對完成研究的足夠數量的患者的預期，數據清理由獨立委員會和我們的內部操作執行統計測試和分析。並在研究中考慮營運影響。完成 32 項後，我們將在 2025 年第一季完成網路分析。

Given the range of outcomes, we will not speculate on scenarios or timing for results of 31 and 32 until we hear from the internet and review boards at which time we will be better informed to provide an update regardless preparations continue to file the ND A as expected in 2025.

鑑於結果範圍廣泛，我們不會對 31 和 32 的結果情景或時間進行推測，直到我們從互聯網和審查委員會得到消息，屆時我們將獲得更好的信息來提供更新，無論準備工作是否繼續按預期在 2025 年提交 ND A。

Now, moving on to our highly differentiated epilepsy portfolio Vormatrigine previously known as plastic to A is a next generation functionally selective small molecule being developed as a once daily oral treatment for adults with epilepsy.

現在，我們來談談我們高度差異化的癲癇藥物組合，沃馬三嗪（以前稱為可塑性 A）是一種下一代功能選擇性小分子，正在開發作為成人癲癇患者的每日一次口服治療藥物。

We know that treatment options for common sis are lacking in both efficacy and tolerability. And we believe the profile emerging with romaine will provide a highly differentiated paradigm shifting way to treat this disease.

我們知道，常見 sis 的治療方案在療效和耐受性方面均有所欠缺。我們相信，長葉萵苣的出現將為治療這種疾病提供高度差異化的範式轉移方法。

Last quarter, we introduced our broad energy clinical program for Vormatrigine inn focal and generalized epilepsy.

上個季度，我們推出了局部性和全身性癲癇的沃馬三嗪廣泛的能量臨床計劃。

And I'm glad to share that the ambitious multi study goal we aim to achieve are advancing. Well.

我很高興地告訴大家，我們致力於實現的雄心勃勃的多項研究目標正在不斷推進。出色地。

Energy is comprised of four studies aiming to build a strong base of patients for our trial while generating multiple data points over the next 18 months to support the differentiated profile of our metro gene.

Energy 由四項研究組成，旨在為我們的試驗建立強大的患者基礎，同時在未來 18 個月內產生多個數據點，以支持我們的都市基因的差異化特徵。

Three trials of energy are to evaluate the efficacy and safety.

三次能量試驗是為了評估療效和安全性。

The first of these is radius, an open label study that will enroll patients with either focal or generalized epilepsy who receive me for eight weeks with a safe follow up of two weeks.

第一項是橈骨研究，這是一項開放標籤研究，將招募患有局部或全身性癲癇的患者，他們接受我的治療八週，並進行兩週的安全追蹤。

We are on track to deliver on topline results in the first half of 2025 which should help us better understand the effectiveness levels of our metro and its pharmacology in the patient population.

我們預計在 2025 年上半年實現頂線結果，這將有助於我們更好地了解我們的地鐵及其藥理學在患者群體中的有效性水平。

The power one and power two studies are 12 week phase 23 studies in patients with focal seizures. Power one is underway and we anticipate top line results towards the end of 2025 we will slightly stagger the initiation of power two to begin recruiting in the first half of 2025 the combined studies are expected to enroll approximately 500 patients globally as we consider other areas where Vormatrigine can play an important role. It's clear that it's activity in NAV 1.7 and NAV 1.8 coupled with fast acting pharmacology and safe profile could play an important role in pain management.

第一階段研究和第二階段研究是針對局部癲癇患者進行的 12 週的 23 期研究。Power One 正在進行中，我們預計在 2025 年底會獲得頂線結果，我們將稍微推遲 Power Two 的啟動時間，在 2025 年上半年開始招募患者，預計合併後的研究將在全球招募約 500 名患者，同時我們正在考慮 Vormatrigine 可以發揮重要作用的其他領域。很明顯，它在 NAV 1.7 和 NAV 1.8 中的活性加上快速起效的藥理學和安全特性可以在疼痛管理中發揮重要作用。

We are concluding our assessment about the potential role of our Vormatrigine in pain and we'll be sharing more in the near future.

我們正在對沃馬三嗪在緩解疼痛方面的潛在作用進行評估，並將在不久的將來分享更多資訊。

Now, turning to Relutrigine a functioning state modulator that is formulated for pediatric use in these a group of severe epilepsies, caties by developmental delays with early onset with CN two A and SN eight A being one of the most severe and refractory forms of these.

現在，我們來談談 Relutrigine，這是一種功能狀態調節劑，專為兒科使用而設計，用於治療一組嚴重的癲癇症，這些癲癇症由早發性發育遲緩引起，其中 CN 2A 和 SN 8A 是最嚴重和最難治的形式之一。

And we're currently there is no approved treatment as a reminder, Relutrigine has orphan and rare pediatric designation for these two indications.

需要提醒的是，目前尚無批准的治療方法，Relutrigine 針對這兩種適應症具有孤兒藥和罕見兒科藥物資格。

We're thrilled and humbled to share the unparalleled results we observed in phase two involve trial cohort one in SN two A and eight a last quarter where lit demonstrated a number of impressive and unprecedented data points.

我們非常興奮和榮幸地與大家分享我們在第二階段觀察到的無與倫比的結果，該結果涉及上個季度 SN 2A 和 SN 8a 中的試驗隊列一，其中展示了許多令人印象深刻且前所未有的數據點。

This two arm study was run over 16 weeks with 44 week periods. Patients in the placebo arm were admins placebo for one period and were littering for the other three periods and neither the patients or investigators were aware which period was on placebo.

這項雙組研究共進行了 16 週，每期 44 週。安慰劑組的患者在一個時期內接受安慰劑管理，在另外三個時期內亂丟垃圾，患者和研究人員都不知道哪個時期接受安慰劑管理。

15 patients completed the study and patients had the option to continue to an open labor extension. After the 16 weeks, a robust 46% placebo just a reduction in motor seizures over the period was observed with 33% or five out of 15 patients achieving seizure free status that notably was never seen before in this severe patient population.

15 名患者完成了研究，患者可以選擇繼續進行開放式分娩延長。16 週後，安慰劑組觀察到運動性癲癇發作減少 46%，其中 33% 或 15 名患者中有 5 名達到無癲癇發作狀態，這在這一嚴重患者群體中是從未見過的。

In addition, we saw a disease modifying impact noted in the study by both caregivers and clinicians with leveraging leading to meaningful improvements in overall well being of patients in areas of seizure severity and intensity alertness and other important measures.

此外，我們在研究中看到了護理人員和臨床醫生都注意到的疾病改變影響，這種影響在癲癇發作嚴重程度和強度警覺性以及其他重要指標方面顯著改善了患者的整體健康狀況。

It is also very impressive and encouraging finding given not only the severity of the disease but also the lack of improvement in these areas with currently available treatments.

考慮到疾病的嚴重性以及目前的治療方法無法改善這些方面，這項發現也令人印象深刻且令人鼓舞。

Lastly, theine was generally well tolerated with no drug related series of events or those reductions required during the study.

最後，茶鹼通常耐受性良好，沒有發生與藥物相關的一系列事件或在研究期間需要減少劑量。

These results further set up RLI as a potential first and best in class treatment. And following the successful proof of concept, we initiated screening for cohort two of the study which aims to enroll 80 patients and has been receiving interest from physicians and caregivers moving us closer to our goal of bringing a potential precision therapy for those severe patients.

這些結果進一步證明 RLI 是潛在的同類首創和最佳治療方法。在概念驗證成功之後，我們啟動了第二組研究的篩選，旨在招募 80 名患者，並得到了醫生和護理人員的關注，使我們更接近為重症患者提供潛在精準治療的目標。

In addition, across all these which affected nearly 200,000 people in the US. 70 to 80% of the patients are currently on a surge channel block.

此外，在美國，有近 20 萬人受到影響，其中 70% 至 80% 的患者目前處於激增通道阻斷狀態。

When we see the data from Relutrigine, which use a more target approach on the south channel mechanism. Of action. We believe there is a broader potential for Relutrigine across ogs.

當我們看到來自 Relutrigine 的數據時，它對南通道機制採用了更有針對性的方法。行動的。我們相信 Relutrigine 在 OGS 領域有更廣泛的潛力。

With that in mind, we're already diligently working with the regulatory agencies to finalize the Emerald Study Protocol for all G. We expect to finalize by the end of this quarter and initiate in 2025.

考慮到這一點，我們已經在與監管機構密切合作，以最終確定所有 G 的 Emerald 研究協議。我們預計將在本季末完成並於 2025 年開始。

We're very excited by both the potential and the progress of our so channel modulator for metro gene androgen.

我們對用於地鐵基因雄激素的 so 通道調節劑的潛力和進展感到非常興奮。

And there's a lot more to come in 2025.

2025 年還會發生更多的事情。

Running out our clinical epilepsy program is our first AO as a nursing designed to selectively decrease the expression of the SN two A gene and directly target the underlying cause. In early on that seizures in CN two a last quarter, we continue part A of the EMBRAVE Protocol in Brazil. This part of this study will provide important control data, examine the safety and effectiveness of nursing in a very severe disease population.

進行臨床癲癇治療計劃是我們作為護理人員實施的第一個 AO，旨在選擇性地降低 SN 兩個 A 基因的表達並直接針對根本原因。在上個季度早些時候 CN 的兩次緝獲行動中，我們繼續在巴西執行 EMBRAVE 協議的 A 部分。本研究的這一部分將提供重要的對照數據，檢驗極重症族群照護的安全性和有效性。

This continues to be an exciting time for practice. In 2024 has been a transformative year. Looking ahead to 2025 we have a number of inflection points and you remain the rigorous focus on execution.

這仍然是一個令人興奮的練習時刻。2024年是變革的一年。展望 2025 年，我們面臨許多轉折點，而您仍將嚴格關注執行。

We look forward to our potential. First of many ND A submissions in 2025 with that in mind. Let me now turn the call over to our Chief Financial Officer, Tim Kelly Tim.

我們期待我們的潛力。考慮到這一點，這是 2025 年眾多 ND A 提交中的第一個。現在，我將電話轉給我們的財務長 Tim Kelly Tim。

Thanks Mario and good morning everybody and thank you for joining today's call. I'll provide a quick summary on our third quarter. Financials. In Q3, our operating expenses were $57.1 million but $41.9 million of that R&D and the remaining $15.3 million for GN A and reflects an increased amount of clinical activity in our movement disorder and epilepsy programs.

謝謝馬裡奧，大家早安，謝謝你們參加今天的電話會議。我將對我們的第三季做一個簡要總結。財務。在第三季度，我們的營運費用為 5,710 萬美元，但其中 4,190 萬美元用於研發，剩餘的 1,530 萬美元用於 GN A，這反映了我們運動障礙和癲癇計畫的臨床活動增加。

During the third quarter, Prax has spent $27.7 million in operating cash similar to the second quarter of 2024 and it reflects our focus on working capital.

第三季度，Prax 的營運現金支出為 2,770 萬美元，與 2024 年第二季相似，這反映了我們對營運資金的關注。

We ended Q3 with $411.2 million in cash, cash equivalents and marketable securities which compares to $81.3 million of cash at December 31st 2023. With the increase primarily due to the net proceeds from practices follow on public offerings. Earlier this year, our cash supports the runway into 2027 and it includes funding all of the programs that Marcio discussed today through their readouts. Now, I'll pass it over to you Marcio.

截至第三季末，我們的現金、現金等價物和有價證券為 4.112 億美元，而 2023 年 12 月 31 日的現金為 8,130 萬美元。成長的主要原因是後續公開發行股票所獲得的淨收益。今年早些時候，我們的資金支持了 2027 年的跑道建設，其中包括資助 Marcio 今天透過讀數討論的所有項目。現在，我將把它交給你，馬西奧。

Thank you, Tim.

謝謝你，提姆。

Now when I open the call for Q&A operator.

現在，當我打開問答接線員的電話時。

Thank you (Operator Instructions)

謝謝（操作員指示）

Our first question comes from Ritu Baral with TD Cowen. When you may proceed.

我們的第一個問題來自 TD Cowen 的 Ritu Baral。何時可以繼續。

Good morning guys. Thanks for taking the question. A couple questions on retro gene 550 for tee specifically, as you think about the 80 patients in the expanded cohort for 80 patient, expanded cohort sufficient for registration. Will the enrollment criteria for that 80 patients be any different than the original 15 patients? And it, so do you expect it to result in any or prospectively expected to result in any changes to Fathy or safety?

大家早安。感謝您回答這個問題。具體來說，關於 T 恤的逆轉錄基因 550，有幾個問題，當您考慮擴展隊列中的 80 名患者時，80 名患者足以進行註冊。這 80 名患者的入選標準與最初的 15 名患者有何不同？那麼，您是否預期它會導致或預期會導致 Fathy 或安全性發生任何變化？

And then the second part of that question is you mentioned that you are seeking alignment with regulators in the first half. Can you talk to maybe any more specifics around that timing? How you might expect cohort to change based on feedback and any specifics on what you're asking on? Emerald? Thanks.

然後問題的第二部分是您提到您正在尋求與監管機構在上半年達成協議。您能談談更多關於該時間的具體細節嗎？根據回饋以及您所詢問的具體內容，您預計群組將如何變化？翠？謝謝。

Sounds good. Thanks Rich for, for the question. So on the first one, for the 80 additional patients that are enrolling on the second cohort on the, on the study right now. So number one, like there are active patients. So we we've been screening those, those patients in and getting them into the study, which is very good news in our view, the the major difference I would call on this on this is that it's actually the start dose for the patients randomized to drug.

聽起來不錯。感謝 Rich 提出這個問題。因此，就第一項研究而言，目前第二組還有 80 名患者正在招募中。首先，有活躍的病人。因此，我們一直在篩選這些患者並讓他們參與研究，這在我們看來是個非常好的消息，我認為的主要區別在於，這實際上是隨機分配藥物的患者的起始劑量。

So starting on the previous study was that half a milligram per kilogram per day. And this one is 1 mg per kilogram per day. So it's straight up into the one we believe to be the most efficacious. So we believe the impact is going to be on that. It's just a fast or faster. May I say effect in terms of like separation in a deeper effect, possibly maintaining or even expanding the number of seizure free days and number of patients there no real major like change on the inclusion criteria. So, from a patient population perspective, we're not expecting to see a different one.

因此，先前的研究開始於每天每公斤半毫克。這是每天每公斤1毫克。因此，我們認為它是最有效的。因此我們相信其影響將會是這樣的。只是快一點或更快一點而已。我可以說，在更深層的影響方面，類似分離的影響可能會維持甚至擴大無癲癇發作天數和患者人數，但納入標準並沒有真正的重大變化。因此，從患者群體的角度來看，我們並不期望看到不同的情況。

And then on the, on.

然後繼續。

The timing for, for Emerald. So we do have a protocol, we are aligning on specifics there. I would say it's a little bit more. Maybe traditional is what I would call. We're expecting to run like a parallel, a group of 1 to 112 weeks. What we are aligning is really the inclusion of those patients. So our view and our position right now is that we can phenotypically define patients with, with the independently of their genotypical like geology.

對 Emerald 來說，時機已到。所以我們確實有一個協議，我們正在就具體細節進行協調。我想說還多一點。我可能稱之為傳統。我們期望像平行運行一樣，運行 1 到 112 週的一組。我們真正要做的就是將這些病人納入其中。因此，我們現在的觀點和立場是，我們可以從表現型定義患者，而不受其基因型（如地質特徵）的影響。

And for as long as they have no sensitivity, the mechanism, number one and two seizure burden that are consistent with what we believe we can play a high impact that should be sufficient. So just double checking this like a number of like small details in terms of how to randomize and size and, and things like that, which should be done by, by the very end of the year and then we're going to be able to operationalize by the very beginning of next year.

只要它們沒有敏感性，機制、第一次和第二次癲癇發作負擔與我們認為的一致，我們就能發揮很大的影響，這就足夠了。因此，只需再次檢查一些關於如何隨機化和大小等細節的小問題，這些都應該在今年年底之前完成，然後我們將能夠在明年年初開始運作。

Got it. So I want to clarify, you are going to genotype these patients, but all they need to have is a genetic mechanism that's rational for good.

知道了。所以我想澄清一下，你要對這些病人進行基因分型，但他們所需要的只是一個合理的遺傳機制。

Yes it is Right.

是的，沒錯。

Okay. Got it. Thank.

好的。知道了。感謝。

Thank you.

謝謝。

Our next question comes from Yasmeen Rahimi with Piper Sandler Companies. You may proceed.

下一個問題來自 Piper Sandler Companies 的 Yasmeen Rahimi。您可以繼續。

Good morning team. Thank you so much for all the thoughtful comments. A few questions on the interim analysis. I think investors were just wondering I think the interim was expected to happen end of year, maybe just some color around why it got a little bit moved into one Q2025.

大家早安。非常感謝您所有的深思熟慮的評論。關於中期分析的幾個問題。我認為投資者只是想知道，我認為中期業績預計會在年底發生，也許只是關於為什麼它會稍微推遲到 2025 年季度的一些解釋。

And then it appears based on the remarks you've made that we're study two will not read out before the interim or at the interim. We'll get an insight about one and two. I just want to make sure just to go ahead and if you could just walk us through sort of the disclosures around both of the studies that, that could be helpful.

然後根據您所說的，我們似乎不會在中期之前或中期宣讀第二項研究。我們將深入了解一和二。我只是想確保繼續進行，如果您可以向我們介紹這兩項研究的披露內容，那可能會有所幫助。

And then last question is I think you guys noted that upon the outcome of the data in A T, you would reinitiate a Parkinson's disease program in 2025. Could you maybe comment on like the success in A T would move you into Parkinson's or what would that be a phase two study? Phase three study? Any color around that would be helpful and I'll jump back into the queue.

最後一個問題是，我認為你們已經注意到，根據 A T 數據的結果，你們將在 2025 年重新啟動帕金森氏症計畫。您能否評論一下 A T 的成功是否會使您進入帕金森氏症研究領域，或者這將是第二階段的研究？第三階段研究？任何顏色都會有幫助，我會跳回隊列。

Sounds good. Thanks, Yeah. So, start on the interim, right? So I'll, .

聽起來不錯。謝謝，是的。那麼，從過渡期開始吧？所以我會。

Start.

開始。

By saying we're very confident on, on the execution for, for the interim. I like every aspect that our monitoring and every discussion just increase our confidence on a successful like execution. Of course, it is biased towards our success to begin with and making sure we wrap up the, the program like concomitantly to slash shortly thereafter to the and then, and that was the, I would say the main driver here, right? So what what we're trying to do to take a step back if to deliver a successful program that we can file an ND A and can be incredibly clear the efficacy, the safety of helix aide hydrochlorate for this patient.

我們對臨時的執行情況非常有信心。我喜歡我們的監控和每次討論的每一個方面，它們都增加了我們對成功執行的信心。當然，這首先對我們的成功有偏見，並確保我們完成該計劃，同時隨後不久削減，然後，這就是我想說的主要驅動力，對吧？因此，我們正在嘗試退一步，以提供一個成功的計劃，我們可以提交 ND A，並且可以非常清楚地了解 helix aide 鹽酸鹽對該患者的療效和安全性。

What when you look into that, that there was a number of things that were either bumping up in terms of availability of ID MC members, the their ability to conduct the analysis, cleaning of the data. And I would argue the most important factor here is the influence on study two. That is the second part of your question, right? In the eventuality, which is a quite possibility that the internet analysis work exactly as we expect so very positive. We wanted to make sure the result of study study two is about the same time too shortly there after number one but two, that there is no influence and by influence, I mean, negative influence on study to read out. So when you're looking into as a program, it made sense for us to to make this, which in our view is a small and slight chain. That is safeguarding the overall like positive results in the in our view of the combined studies, right? 3,132 it's combination and the package for for the ND A. So that that is the main rationale on our end, everything is progressing brilliantly so far.

當你研究這個問題時，你會發現，在 ID MC 成員的可用性、進行分析和清理資料的能力方面，有很多事情都在增加。我認為這裡最重要的因素是對第二項研究的影響。這是你問題的第二部分，對嗎？最終，互聯網分析很有可能按照我們預期的那樣進行，因此非常積極。我們希望確保第二項研究的結果與第一項研究的結果大致相同，但第二項研究的結果不會對研究產生影響，我所說的影響是指對研究的負面影響。因此，當您將其視為一個程序時，我們這樣做是有意義的，在我們看來，這是一個小而輕微的鏈條。從我們的觀點來看，這可以保證綜合研究結果整體上取得正面成果，對嗎？3,132 是 ND A 的組合和包裝。因此，這是我們這邊的主要理由，到目前為止，一切都很順利。

Then on the, on the Parkinson's disease study, I think that as our content grows into the outcome on essential tr we need to really be ready to the expansion, right? Like time is an incredibly important asset on this business. We want to make sure that the an indication of value for ourselves for potential strategics and so on and so forth. I get off the ground. We had, I got some feedback from the ft a last time in terms of what they would like to see on AP this study. So we have a very good idea to design a phase two study in, Parkinson's that would significantly advance this program as well. So we're just restarting that in terms of the planning. So we are ready at that time to, kick off and, and restart. So we have a portfolio of indications and forex instead of just one.

然後，關於帕金森氏症的研究，我認為隨著我們的內容逐漸發展成為基本治療的結果，我們需要真正為擴展做好準備，對嗎？就像時間對這個產業來說是一項極為重要的資產。我們希望確保這能為我們自身的潛在策略等提供價值指示。我從地上下來。我們上次從《金融時報》獲得了一些回饋，關於他們希望在 AP 這項研究中看到什麼。因此，我們有一個很好的想法，設計一項針對帕金森氏症的第二階段研究，這也將顯著地推進這個計畫。因此，我們只是從規劃角度重新開始。因此，我們已做好準備，開始並重新開始。因此，我們擁有一系列指標和外匯，而不只是一種。

Great. Thank you. And I'll jump back in the queue.

偉大的。謝謝。我將重新回到隊列中。

Thank you.

謝謝。

Our next question comes from Joon Lee with Truist Securities. He may proceed.

我們的下一個問題來自 Truist Securities 的 Joon Lee。他可以繼續。

Hey, thanks for the updates guys. Just a quick clarification. Will you be including Lentis Gesto in the broader DEE study? Because there's, you know, genetic basis for L gaso and you seem to want to stick to epilepsy based on your response to the question. And I have a follow up.

嘿，謝謝大家的更新。只需簡單澄清一下。您會將 Lentis Gesto 納入更廣泛的 DEE 研究嗎？因為您知道，L gaso 有遺傳基礎，而且根據您對這個問題的回答，您似乎想堅持認為是癲癇。我還有一個後續行動。

Sounds good.

聽起來不錯。

So we will include LG patients on this who attempt to, the answer again to, reach you. Before we are providing and attempting to collect as much demo type information. Sometimes as you know, they're going to be Phenotypically Defined, clinically defined and not have like a final diagnosed there. But we believe that as these patients are right now, when you're looking to actually data on the literature on claims data for projects, one of the highest use of the actual channel mechanism with one of the highest issues in terms of tolerability, which we think is a, is a sweet spot for for our drugs. So we as we will be doing, but at the same time, that is part of like the entire discussion about inclusion criteria and measuring is the discussion we're having right now.

因此，我們將包括那些試圖再次向您提供答案的 LG 患者。之前我們提供並嘗試收集盡可能多的演示類型資訊。有時如您所知，它們將被表型定義、臨床定義，但不會得到最終的診斷。但我們相信，就這些患者而言，當您查看項目索賠數據文獻中的實際數據時，您會發現實際渠道機制的使用率最高，而耐受性方面的問題也最多，我們認為這是我們藥物的最佳選擇。所以我們會這樣做，但同時，這也是關於納入標準和衡量的整個討論的一部分，也是我們現在正在進行的討論。

Great. So it's a true the E study?

偉大的。那麼這是一項真正的 E 研究嗎？

Great. You know, for the interim analysis for essential program just wanted to clarify that you when you say refer to interim analysis, you're referring to the study one interim. So your withdrawal trial topline will depend on the interim from the parallel comparator trial. Is that correct? And then is, yeah.

偉大的。您知道，對於基本計劃的中期分析，我只是想澄清一下，當您說參考中期分析時，您指的是研究一項中期分析。因此，您的戒斷試驗頂線將取決於平行比較試驗的中期結果。對嗎？然後就是，是的。

Yeah. So, and, then number part part two is that or is there no inferiority analysis baked into that interim analysis?

是的。那麼，第二部分是這樣的，或者說中期分析沒有包含劣勢分析嗎？

Yeah. So the the interim analysis is on study one only as you mentioned, right? We, we don't believe that would be necessary or appropriate for, for an internal study too. The basically there's an alignment in terms of like database lock planning between the multiple events that we're talking here. So that is could be somewhat of an influence because those studies are recruited concomitantly, right? As you know, are from the same pool of patients and they are randomized to these studies. So that's why there could be some influence on depending on the outcome of the, of the internal as well. We will be reading out study to of course shortly thereafter on the inter that is easier one could argue study to monitor based on like event rates and, and things like that. So quite bullish about that to begin with.

是的。所以正如您所說，中期分析僅針對第一項研究，對嗎？我們，我們不認為這對內部研究是必要或適當的。基本上，我們在此討論的多個事件之間的資料庫鎖定規劃是一致的。所以這可能會有一定影響，因為這些研究是同時進行的，對嗎？如您所知，這些研究對象均來自同一群患者，並且是隨機分配到這些研究中的。所以這就是為什麼內部的結果也會受到一定的影響。當然，我們很快就會讀出研究成果，人們可以爭辯說，基於類似事件發生率等進行監測的研究更容易。所以一開始就對此相當樂觀。

Now, what was the second part of your question? About the inferiority or sorry.

現在，你的問題的第二部分是什麼？關於自卑或抱歉。

So that is a futility margin on the, on the lower end of the conditional power as its standards, right on, on interim like that. So fairly standard in terms of the, of the bottom there, a very wide, I would say a size information zone. Because that's why the reason why they study or the that is well designed to begin with. And on the other hand, as well, which should be considered that is it's tough for overwhelming efficacy as well. You were wanted to balance all of that, of course, from an information fraction perspective and from a spending perspective and in the overall execution to drive to the successful outcome.

因此，這是在有條件權力的下端作為其標準的無效邊際，就在臨時的範圍內。因此，就底部而言，這是一個相當標準的非常寬的尺寸資訊區。因為這就是他們學習的原因，或者說從一開始就精心設計的原因。另一方面，也應該考慮到，其難以達到壓倒性的功效。當然，您需要從資訊分數的角度、支出的角度以及整體執行的角度來平衡所有這些，以取得成功的結果。

Right? And then last question, you know, as we look to the, you know, very likely approval of sugen from vtex in January, it's actually impressive that it even works at all because it only targets one of the three voltage gated sodium channels in the peripheral nervous system. So it's actually interesting that you're advanced looking at your, your formal in in pain as well, which targets two out of the three pain receptors or not receptors, but voltage gated channels, any anecdotal evidence of pain reduction from your phase one or any other.

正確的？最後一個問題，您知道，當我們展望 1 月份 Vtex 極有可能批准 Sugen 時，它竟然能起作用，這確實令人印象深刻，因為它只針對外周神經系統中三個電壓門控鈉通道中的一個。因此，實際上很有趣的是，您正在先進地研究您的正式疼痛問題，它針對的是三個疼痛受體中的兩個或不是受體，而是電壓門控通道，您在第一階段或任何其他階段是否有任何減輕疼痛的軼事證據。

Thank you.

謝謝。

Yeah. So the, the like we're.

是的。所以，就像我們一樣。

Super excited about this as well, we've been looking to for a while. It, it's not something that made sense from a priority execution, capital location beforehand for us. But now we believe it does, we do have a very strong pre clinical evidence on

對此我們也非常興奮，我們已經期待了好一陣子了。對我們來說，從優先執行、事先確定資本位置的角度來看，這不是一件有意義的事。但現在我們相信它確實如此，我們確實有非常有力的臨床前證據

in pain models and in general like a very potent inhibitor of 17 and 18, as you know that mechanism and the duality of the mechanism is quite important in paying generally acute and sub chronic and chronic pain.

在疼痛模型中，一般來說，17 和 18 是一種非常有效的抑制劑，如您所知，這種機制和機制的二元性對於治療一般急性、亞慢性和慢性疼痛非常重要。

We thought that, yeah, we are, we're excited with what you're seeing and, and we think that what's most appropriate for us is just to finalize everything look into from a competitive standpoint, as well, make sure that would be competitive and then talk about our plan early in the year with all of you.

我們認為，是的，我們對您所看到的一切感到興奮，並且我們認為對我們來說最合適的就是從競爭的角度最終確定一切，確保具有競爭力，然後在年初與大家討論我們的計劃。

Thanks for all your answers.

感謝您的所有回答。

Of course. Thank you.

當然。謝謝。

Thank you.

謝謝。

Our next question comes from Francois Brisebois with Oppenheimer, you may proceed.

我們的下一個問題來自 Oppenheimer 的 Francois Brisebois，您可以繼續。

Hi, thanks for the questions and thanks for kind of going through the, the potential scenarios here and the complexity and the dependence between the or the the impact of study one on study two. So, but in terms of, you know, what to share on the interim, is it, you know, could it go into data or that's actually a really good case scenario where we stop the study because things are working out or is it more, you know, it, can we be assured that the actual top line of study one will be after study two that might come short after the interim. Look just any any help there understanding the timeline of the top line versus the interim for study one.

你好，謝謝你的提問，也謝謝你講解這裡的潛在場景以及研究一和研究二之間的複雜性和依賴性。所以，但是就您知道在此期間要分享什麼而言，您知道，它是否可以轉化為數據，或者這實際上是一個很好的案例場景，我們停止研究，因為事情正在進展，或者更重要的是，您知道，我們是否可以確保第一項研究的實際頂線將在第二項研究之後，而第二項研究可能會在中期之後出現不足。看看是否有任何幫助可以理解研究一的頂線與中期的時間線。

Yeah, sounds good. And so the like I, I think the scenarios like if I think about boot camps here, so one a as you mentioned, the potential to to stop like for, for overall Masco that obviously is not the base case, let's just play that out. And then both studies would be having the results at the same time, right? Like study one and study two at that point in time, which obviously would be a quite positive complete package. And, and so on. I think that is now an opportunity as well to increase the size of study one. And, and on that case, the conversation we'll be having as we're moving that one forward, we're increasing the size. And for the study two, we should have the results like very quickly as well.

是的，聽起來不錯。所以，我認為如果我考慮這裡的新兵訓練營，那麼正如你提到的那樣，對於整體 Masco 來說，停止的可能性顯然不是基本情況，讓我們來演繹一下。那麼兩項研究就會同時得出結果，對嗎？就像當時的研究一和研究二一樣，這顯然是一個非常積極的完整方案。等等。我認為現在這也是擴大研究規模的機會。並且，就此案而言，隨著我們推進這一案子，我們將進行討論，擴大其規模。對於第二項研究，我們也應該很快就會得到結果。

So that's when it associates the two that is the highest a priority probability that would happen is, is on that just because the range of the conditional power is the largest or the widest on that song. So I think that's the two, I would say we should plan the most around with the first one having the highest impact, right? In terms of like operationally making sure ready to like wrap it up the the other two studies and so on. So that's what we, one of the considerations and how, how we look into being ready for for the interim.

因此，當它將兩者關聯起來時，發生優先順序最高的機率是，僅僅是因為該歌曲的條件功率範圍最大或最寬。所以我認為這兩個方面，我想我們應該圍繞第一個方面製定最多的計劃，其中第一個方面的影響最大，對嗎？從操作角度來看，確保準備好完成另外兩項研究等等。這就是我們的考慮之一，以及我們如何為過渡做好準備。

Okay. Thank you. And then on the de commercial front, there's a lot of different ways to look at this market. Can you help us understand, you know US, X US, how you think about the commercial potential here?

好的。謝謝。從商業角度來看，有很多不同的方式來看待這個市場。您能幫助我們了解一下，您了解美國，X 美國，您如何看待這裡的商業潛力？

Yeah, the vast majority of the business and I would say from a dollar value perspective is in the United States, our modeling shows around 70% in, in the US. And then the about 30% outside of the US. For, for our pick. We're talking about a multi billion dollar pick here in GS, right? We just completed yet like another refinement of the epidemiology in the US and, and looking into the utilization of the mechanism, limitations and so on and it's a little bit shy of 200,000 patients in the US. When you consider that even relatively small market share gets you quite important figures in terms of like the potential big revenue. And then the outside of the US becomes a little bit more.

是的，從美元價值的角度來看，絕大多數業務都在美國，我們的模型顯示約 70% 的業務在美國。其中約 30% 在美國境外。對於，對於我們的選擇。我們正在談論 GS 中的數十億美元的選擇，對嗎？我們剛完成了美國流行病學的另一個細化，並研究了機制的利用、局限性等，美國的患者人數略低於 20 萬人。如果你考慮到即使相對較小的市佔率也能為你帶來相當重要的數據，例如潛在的巨額收入。然後美國以外的範圍就變得稍微大一些了。

I'm going to call opportunistic from business perspective, obviously important to have access to patients as well, but not as important to get the drug off the ground and, and get quite meaningful revenues. So about two thirds in the west, third outside of the west. That's how we would be modeling.

我從商業角度來看待機會主義，顯然接觸患者也很重要，但讓藥物起步並獲得相當可觀的收入並不那麼重要。大約三分之二在西部，三分之一在西部以外。這就是我們的建模方式。

Thank you.

謝謝。

Thank you.

謝謝。

Thank you.

謝謝。

Our next question comes from Yatin Suneja with Guggenheim. You may proceed.

我們的下一個問題來自古根漢的 Yatin Suneja。您可以繼續。

Hey, guys, thank you for taking my question. Just a couple for me as well. Mostly on the ET side, could you provide us where you are, at least on the enrollment from like how many patients have been enrolled in study one and two if you could and then, you know, we understand this. In turn, could you also talk about and maybe put some numbers around the possible sample size adjustment ranges based on the pre specified plan? Like how long would that take at the maximum if, you decide to increase the size and, and let's say you go with the maximum of patients that are allowed, how long will that will that take, will that take?

嘿，夥計們，謝謝你們回答我的問題。對我來說也只有幾個。主要是在 ET 方面，您能否向我們提供您的現狀，至少是招募情況，例如，如果可以的話，有多少患者參加了第一項和第二項研究，然後，您知道，我們了解這一點。反過來，您能否也談談並根據預先指定的計劃給出一些可能的樣本大小調整範圍的數字？例如，如果您決定增加規模，並且假設您接受允許的最大患者人數，那麼最多需要多長時間？

Yeah, absolutely. So, I'll, give as much as we feel that we could give right now in order like to preserve all the optionality for us. So the the current study states I would say of patients, right? The way we look into is not on the top of the screening, but on patients being randomized per week. So when you look into what we can maintain constantly, is anywhere between 2030 patients per week, randomized new patient. So, if you fast forward to potential scenario here.

是的，絕對是如此。因此，我會盡我們所能，為我們保留所有的選擇權。所以我想說目前的研究顯示患者的情況是這樣的，對嗎？我們研究的方式不是基於篩檢本身，而是基於每週隨機分配的患者。因此，當您研究我們可以持續維持的情況時，每周可以隨機接收 2030 名新患者。因此，如果您快進到這裡潛在的場景。

So I'm going to use like two scenarios where I can increase of 100 increase of 200 patients. You can see that that could be achieved in like anywhere between like 3 to 6 weeks of ization. Of course, you need 12 weeks after that to completion of the study, but it is very fast in terms of accruing new patients to the study if needed.

因此我將使用兩種場景，其中我可以增加 100 名或 200 名患者。您可以看到，這可以在 3 到 6 週的時間內實現。當然，之後還需要 12 週的時間才能完成研究，但如果需要的話，在增加新患者參與研究方面速度非常快。

Thank you.

謝謝。

Thank you.

謝謝。

Our next question comes from Douglas Tsao with H.C. Wainwright & Co., LLC. You may proceed.

下一個問題來自 H.C. Wainwright & Co., LLC 的 Douglas Tsao。您可以繼續。

Hi, good morning. Thanks for taking the questions. Just to confirm what you just said. So I think you indicated 30 patients. Is that enrolled and randomized or is that simply enrolled into the study in the scenario that you expand the patient population for essential?

嗨，早安。感謝您回答這些問題。只是為了確認你剛才說的話。所以我認為您指的是 30 名患者。這是招募和隨機的嗎？還是在擴大必需患者群體的情況下簡單地將其招募到研究中？

So that is the one we very, very comfortable from our like base of our organization that we can achieve very, I would say relatively easily. I'm not going to say easily because there's a lot that goes on on this randomized patients in a given week.

因此，從我們組織的基礎來看，我們非常放心，我們可以非常、相對輕鬆地實現這一目標。我不會輕易說，因為在特定的一週內，這些隨機患者身上會發生很多事情。

Okay, great. That's helpful. And then just if you could provide a little more color in terms of moving from trigen into these pain indications and what type of work you're going to be doing or need to do before coming out with a more sort of expanse or sort of coming forward with the full development plan.

好的，太好了。這很有幫助。然後，如果您可以提供更多關於從 trigen 轉向這些疼痛適應症的詳細信息，以及在提出更廣泛的範圍或提出完整的開發計劃之前您將要做或需要做哪些類型的工作。

So, we're working.

所以，我們正在工作。

And, and I'll say thanks for others working on this space. Right. There's a, there's a lot that's been done on the last few years here as well. And when you look into the, what, what it's important for us, a couple of things, right? So one is the pharmacology itself, right? Like, can we have the relief of like kindergarten to CX and to relationships with cyex, like quickly enough for certain types of pain? What is the ideal types of pain? What is our confidence in terms of pre clinically and potentially early clinical data, biomarker data, et cetera? That could get us to a point that we're incredibly confident as we were in las for when we like moving into a focal to have a similar package.

並且，我要感謝在這個領域工作的其他人。正確的。過去幾年這裡也做了很多事。當你研究什麼時，對我們來說什麼是重要的，有幾件事，對嗎？那麼一個是藥理學本身，對嗎？例如，我們能否快速緩解從幼兒園到 CX 以及與 cyex 的關係中某些類型的疼痛？理想的疼痛類型是什麼？我們對臨床前和潛在早期臨床數據、生物標記數據等的信心如何？這可以讓我們達到一種非常自信的程度，就像我們在拉斯維加斯一樣，當我們想進入一個焦點並擁有類似的包裹時。

I would say and, then like really understanding the impact that we're talking about here. And I think differently from other things being developed, both central and peripheral aspects of the disease. So how much more can we expect from this mechanism? What is again, ideal education, ideal study to show that and cost in that study and things like that. So that's the work is being done literally as we speak, a lot more to be finalized between now and the end of the year. And I think we're going to be in a very good position early in the year to showcase that to all of you.

我想說，然後真正理解我們在這裡談論的影響。我認為這種疾病與其他正在開發的事物不同，既包括疾病的核心方面，也包括疾病的外圍方面。那麼，我們對這機制還能期待多少呢？再說一遍，理想的教育、理想的學習，以及學習的代價等等。所以這就是我們所說的正在進行的工作，從現在到年底還有很多工作要完成。我認為我們將在今年年初處於非常有利的位置來向大家展示這一點。

Okay, great. And then just one more on (Elsa Urson), I think with last quarter, you indicated that the first patient was being enrolled in sort of the Global Registration study. But I think today you indicated that they're sort of being added to Embra. And I just want to understand if there's been any change as you think forward about the global registration program for that drug.

好的，太好了。然後再問一個問題（Elsa Urson），我想在上個季度，您表示第一位患者正在參加某種全球註冊研究。但我認為今天您表示它們正在添加到 Embra 中。我只是想了解一下，在考慮該藥物的全球註冊計劃時是否有任何變化。

Yeah. So no, that that's a very important aspect as well. So the way we've been looking into I know since that two fold here, so one we consider to be important to explore particularly the safety at a different exposure levels. And that's a lot of it's being done in Brazil right now on the second cohort is I want you to rerandomize like patient cohort to drug or placebo or to drug or sham. On that case, we're exploring a range of those sequentially increasing the dose on those patients. We're doing all the other assessments as well as you can imagine.

是的。所以，這也是一個非常重要的面向。因此，我知道我們一直在研究這兩種方法，因此我們認為特別重要的是探索不同暴露水平下的安全性。目前巴西正在對第二組患者進行大量研究，我希望將患者群體重新隨機分配到藥物組或安慰劑組、藥物組或假治療組。在這種情況下，我們正在探索一系列依序增加這些患者劑量的方法。正如您所想像的，我們正在進行所有其他評估。

So it was important for us to actually keep that separated from what we believe to be a very good long term ecas exposure. Those that is the 1 mg for a month for for the global study. We have very good alignment already in general what we is needed there. But we do have still a meeting pending this year with the FDA on finalizing which was likely a change on their end on, on the our expectations from, from when the meeting will appear. So nothing problematic there, but it's kind kind of drove the fact that we need just a little bit more time to make sure we have confidence on the final protocol so we can initiate it. So that is the, that's the bottom line for that one.

因此，對我們來說，真正將其與我們認為非常好的長期 ecas 風險分開是非常重要的。對於全球研究來說，這是每月 1 毫克的劑量。總體而言，我們已經很好地了解了那裡需要什麼。但是我們今年仍需與 FDA 舉行一次會議來最終確定會議時間，這很可能是他們對我們對會議召開時間的期望的一個變化。所以那裡沒有什麼問題，但這在某種程度上意味著我們需要多一點時間來確保我們對最終協議有信心，這樣我們就可以啟動它。這就是，這就是那件事的底線。

Okay. And so with the patients being studied in Brazil, now, are you, you're looking at some additional doses? And if the result, I mean, is there a scenario, I know you've been very confident in the 1 mg per K dose? I mean, is there a chance that if if results warrant that you would potentially look at higher doses in the US and European registrational studies?

好的。那麼，對於正在巴西進行研究的患者，現在，您是否正在考慮一些額外的劑量？如果結果是這樣，我的意思是，是否存在一種情況，我知道您對每公斤 1 毫克的劑量非常有信心？我的意思是，如果結果證明有理由相信，您是否有可能在美國和歐洲的註冊研究中考慮更高的劑量？

Yeah, we must follow the science, right? So what we've seen so far on the patients in the US, which are continuing taking the drug and on the patient in Europe and in Australia that that actually took higher dose than in the US. It's not meaningfully different in terms of to control gains on like development milestones and, and, and things like that.

是的，我們必須遵循科學，對嗎？到目前為止，我們看到美國的患者繼續服用該藥物，而歐洲和澳洲的患者服用的劑量實際上比美國患者服用的劑量更高。在控制開發里程碑等收益方面，並沒有什麼實質的差異。

Now we gotta remain open to, to the possibility that yes, it's going to be meaningfully different fact or whatever. And if that's the case, we would be in a position to complement the the global study, either in its open label phase right after the control phase or even in a different cohort, if that is warranted. So will be driven by the, the results that we see here. But the time lines in terms of like the original four patients that they're going to be those that are perfectly aligned with the enrollment timelines we have for the global study. So it shouldn't be a conflict in terms of how to get to the best possible result for those patients?

現在我們必須保持開放的態度，接受這樣的可能性：是的，這將是一個有意義的不同事實或其他什麼。如果是這樣的話，我們將能夠補充這項全球研究，無論是在對照階段之後的開放標籤階段，還是在不同的隊列中，如果有必要的話。因此將受到我們在此看到的結果的驅動。但就最初的四名患者而言，他們的時間表將與我們為全球研究制定的入組時間表完全一致。那麼，如何為這些患者獲得最佳治療結果不應該有衝突嗎？

Okay, great. Thank you very much.

好的，太好了。非常感謝。

Thank you.

謝謝。

Our next question comes from Kambiz Yazdi with Jefferies. You may proceed.

我們的下一個問題來自 Jefferies 的 Kambiz Yazdi。您可以繼續。

Morning team a couple of questions for me. The placebo response was pretty well controlled in essential one. Essential three has a more innovative decentralized design. What steps have you taken to control placebo response and study one in essential three?

早安，團隊，請問我幾個問題。安慰劑反應在基本療法中得到了很好的控制。Essential Three 具有更創新的分散式設計。您採取了哪些步驟來控制安慰劑反應並研究其中三個基本步驟？

And then as a second and a set of questions maybe on verme gene. What is sodium channel blockers demonstrated historically in PGTC seizures? And what would a registrational program consist of in a generalized epilepsy? Thank you.

然後第二個問題和一系列問題可能與verme基因有關。鈉通道阻斷劑在 PGTC 癲癇發作的病史表現如何？那麼，全身性癲癇的註冊計畫包括哪些內容呢？謝謝。

Sure. Thanks came. So, placebo was already pretty well through, as you said, on on essential one. When you're looking to essential three, there was one aspect that, we wanted to add to further control that. So, what are you seeing? Is There is a I would say.

當然。謝恩來了。因此，正如您所說，安慰劑在基本作用上已經發揮了很好的作用。當您查看三個基本要素時，有一個方面我們想添加以進一步控制它。那麼，你看到了什麼？有嗎？我想說的是。

Slight but important, change on patients that are less stable at baseline. And what I mean by that is, there were, pre, screening, assessments or sorry, a pre mation assessment on study, on essential one in a baseline assessment. So when you look into those patients that vary more in between.

對於基線較不穩定的患者來說，變化很小但很重要。我的意思是，有預先篩選評估，或抱歉，有研究的預先資訊評估，基線評估中必不可少的評估。因此，當您觀察這些患者時，會發現他們之間的差異更大。

Those They tended to be a little bit higher on placebo. Now, that study was, again, at the end of the day, it is going to be like four times smaller than essential three. So we wanted to ask the question whether or not the influence is going to be similar, right? So we chose to actually add a maximum variability parameter between those visits and to formally have visits the pre organization one. We believe because you can monitor our patients for all the study and we know how long it takes for the drug to start working that we're very successful on on controlling that on making sure that the, the patients that are similar to the what we want from you.

那些他們對安慰劑的依賴程度往往更高一些。現在，這項研究最終會比基本三項研究小四倍。所以我們想問影響是否會相似，對嗎？因此，我們選擇在這些訪問之間添加一個最大可變性參數，並正式將訪問設為組織前的訪問。我們相信，因為您可以在整個研究中監控我們的患者，我們知道藥物需要多長時間才能開始起效，所以我們能夠非常成功地控制這一點，確保患者與我們對您的期望相似。

One, right? We don't want to depart from the cohorts on one, but we want to make sure we tighten potentially variability that is not due to drug effect, but rather to placebo effects. So we're very, very confident on the measures that we put in place to to control placebo there.

一個吧？我們不想偏離某一群體，但我們希望確保我們能夠縮小潛在的變異性，這種變異性不是由於藥物作用而是由於安慰劑效應。因此，我們對控制安慰劑所採取的措施非常有信心。

And then on your second question on generalized by the primary or not. It's mixed the the results historically. And I think partially because that isn't really being a sergeant channel blocker that is very selective. And that really works on as these neurons are like firing a lot and, and expanding a lot in terms of like the loss of going through. I'm going to call on the on the action potentials that can really block the pathological events but not the physiological for this patient. That is a very good evidence with more selective or partially more selective drugs.

然後關於你的第二個問題，是否由主要因素來概括。從歷史上看，結果是好壞參半。我認為部分原因是，這並不是一個真正具有選擇性的中士頻道阻斷者。這確實有效，因為這些神經元就像在經歷損失時大量發射和擴張一樣。我將調用能夠真正阻止病理事件但不能阻止該患者生理事件的動作電位。這對於更具選擇性或部分更具選擇性的藥物來說是一個很好的證據。

So we are interesting enough, I would say anecdotally, the conversations that we've been having like a lot of the conversations that get off the ground and I get excited. A lot of people are equally or more excited actually about the generalized. What was a little bit surprising to me that there is so much excitement on generalized with, with romaine.

所以我們很有趣，我想說的是，我們進行的對話就像很多已經開始的對話一樣，我感到很興奮。事實上，很多人對於普遍化的東西同樣或更興奮。令我有點驚訝的是，人們對長葉萵苣有如此濃厚的興趣。

And I believe it's because that is a huge amenity there. And obviously, those seizures are incredibly important as well when they happen from the severity and potential impact and in terms of fatality as well for, for this patient. So we, we're going to see soon enough, I would say from radiance, what kind of impact we can have there and radiance going to serve as a, as a kind of springboard for us. And to design. What we would expect to be a registrational phase study for, for generalized as well?

我相信這是因為那裡有龐大的便利設施。顯然，這些癲癇發作對該患者來說也非常重要，因為它具有嚴重程度、潛在影響以及致命性。因此，我想說，我們很快就會看到，從光輝來看，我們能在那裡產生什麼樣的影響，而光輝將成為我們的一種跳板。並進行設計。我們期望註冊階段的研究針對什麼，以及針對普遍性的研究？

Great. Thank you.

偉大的。謝謝。

Thank you.

謝謝。

Our next question comes from Ami Fadia with Needham. You may proceed.

我們的下一個問題來自 Needham 的 Ami Fadia。您可以繼續。

Hi, good morning. Thanks for all the updates. A couple of quick questions for me. Firstly, on Alexa, can you comment on the enrollment and and how what percent of patients have completed randomization relative to the target enrollment for the two studies? And was there a slowdown in the enrollment rate that caused the shift in the timeline?

嗨，早安。感謝所有的更新。我有幾個簡單的問題。首先，在 Alexa 上，您能否評論一下招募情況，以及相對於這兩項研究的目標招募情況，有多少百分比的患者完成了隨機化？入學率的下降是否導致了時間表的轉變？

Maybe I'll ask my next question after you.

也許我會在你之後問下一個問題。

This one. Sure. So I mean, we not going to talk about that right now as you mentioned, right on the, on the prepared remarks and in the press release. But what, what I can tell you that is no, is slow down on what we expected from randomization or from patients on screening. I think we continue and we, we saw historically, we expected to continue to see if we decide to increase the, size of the study in Q1, a fairly robust number of patients coming through. I think we intentionally being managing that. So we can get like the best possible outcome for, for the studies that are two positive studies at like right after the entrance. So it's been a lot on us and, and of course, the gathering of patients and things like that, which influence but not meaningful.

這個。當然。所以我的意思是，正如你在準備好的演講稿和新聞稿中提到的那樣，我們現在不會談論這個問題。但是，我可以告訴你的是，我們對隨機化或篩選患者的預期並沒有放慢。我認為我們會繼續，而且從歷史上看，我們預計如果我們決定在第一季擴大研究規模，將會有相當多的患者參與。我認為我們有意管理這一點。因此，我們可以獲得最好的結果，對於入學後立即進行的兩項積極研究。所以，我們承受了很大的壓力，當然，還有病人的聚集等等，這些都有影響，但沒有什麼意義。

Got it. Okay. And then on Vormatrigine in the RADIANT study, can you talk about how many patients you're targeting or, or sort of the mix of patients between focal and generalized and, and how that might inform your plans to develop it further in generalized epilepsy?

知道了。好的。然後，關於 RADIANT 研究中沃馬三嗪的療效，您能否談談您的目標患者數量，或者局部性癲癇和全身性癲癇患者的組合情況，以及這可能如何影響您在全身性癲癇中進一步開發該藥物的計劃？

Yeah. So the the goal is to have 50 patients on a what, what we are seeing again on the early days. But what we're seeing is about like the expected 30% generalized 70% focal interest for like in general, particularly like on, on the site we targeted here, we were, I'll say are relatively selective in terms of the number of sites because that is again a huge interest and we didn't want to completely, I'm going to say lose control of the number of patients and get significantly more than we expect here.

是的。因此，我們的目標是讓 50 名患者接受我們早期看到的情況。但我們看到的是，總體而言，預期的 30% 廣義 70% 的焦點興趣，特別是在我們在此定位的站點上，我會說我們在站點數量方面是相對有選擇性的，因為這又是一個巨大的興趣，我們不想完全，我想說的是失去對患者數量的控制，並得到比我們預期更多的患者。

So we kept that a little bit tight in terms of the 50 or so patients that, that we expect to enroll that should be sufficient as we go like across. And it's an open label study, right? So we're continuously seeing and adapt as needed in, in terms of maybe reducing or increasing the num the total number of patients based on the overall interest. But 3,070 from generalized and focus is, is what we're going to expect to see at the end.

因此，我們對預計招募的患者人數控制得比較嚴格，大約只有 50 名，這應該就夠了。這是一項開放標籤研究，對嗎？因此，我們會根據整體情況不斷觀察並根據需要進行調整，例如減少或增加患者總數。但從整體和重點來看，3,070 是我們最終預計看到的。

Understood. Maybe just a last question for me as you design the MD E study, what assumptions would you be making with regards to regulatory performance in this patient population relative to the data that you saw, you know, in sen two A&A?

明白了。也許這只是我的最後一個問題，當您設計 MD E 研究時，相對於您在 sen two A&A 中看到的數據，您會對該患者群體的監管表現做出哪些假設？

Yeah. So arguably CN two NH A were the hardest of those conditions to treat when you, when you look across the board. Yeah, there are a few allergies that are incredibly difficult to treat, but those were definitely incredibly hard to see reduction to see your freedom mortality in Chile, for example, is like six times higher than right. So in, in infancy, so this is a complete like higher bar. So we expect FS are above the, the ay levels. They were saying of course, not powering the study for that. We're being a little bit more conservative, but I don't think it's unreasonable to assume that this would be best in class for this patients.

是的。因此，從整體來看，可以說 CN 和 NH A 是這些疾病中最難治療的。是的，有些過敏症非常難以治療，但這些過敏症的死亡率確實很難降低，例如，智利的死亡率比右邊高出六倍。所以在嬰兒期，這是一個完全更高的標準。因此我們預期 FS 將高於 ay 水準。他們當然說不會為此進行研究。我們的態度比較保守，但我認為，認為這對這些患者來說是最好的治療方法並不為過。

Thank you.

謝謝。

Thank you.

謝謝。

Thank you.

謝謝。

Our next question comes from Joel Beatty with baird. You may proceed.

我們的下一個問題來自 Baird 的 Joel Beatty。您可以繼續。

Hey, thanks for the updates. The first one is on (eex callide) For how if the interim is successful, could we get final results at the same time that we learned the interim is successful? Or will there inherently be some amount of time between those two events?

嘿，謝謝你的更新。第一個是關於（eex callide）如果中期成功，我們能否在得知中期成功的同時獲得最終結果？或者這兩件事之間必然會存在一定的時間間隔？

Yeah. No, Joe, like thanks for that. If in the return, it's, successful. So it's designed for an equivocal accs it's cross we will have results at the same time.

是的。不，喬，謝謝你。如果返回，則表示成功。因此，它是為模棱兩可的 accs 而設計的，透過交叉我們將同時得到結果。

Thanks and then in, DEEs, should we think about routine as kind of just working in, in patients who are already responsive to calcium channel blockers? Or could you talk about the potential to work beyond the patients who are responsive to those agents?

謝謝，那麼，DEE，我們是否應該將常規治療視為對已經對鈣通道阻斷劑有反應的患者的一種治療方式？或者您能否談談針對那些對這些藥物有反應的患者以外的患者進行研究的潛力？

Yeah. So, it's quite interesting. I think that is like when you're looking to pre clinically and, and all the work we did, it does not look like it could or should be restrictive to patients who had prior response or that are expected to. If you look into all the work we did with the two compounds published a little back. The the results on actual the dynamics of the neurons among the animal model, for example, into the syndrome are quite as the als but unexpected meaning are significantly better than you would expect on that, which is there is a bias towards maybe some first generation. So channel blockers wouldn't work on that population, right? So clearly not the case here.

是的。所以，這很有趣。我認為這就像當您在進行臨床前研究時，以及我們所做的所有工作時，它看起來似乎不能或不應該限制那些之前有反應或預期有反應的患者。如果你看一下我們之前發表的關於這兩種化合物的所有研究成果。例如，關於動物模型中神經元動態的實際結果進入綜合症，與 als 相當，但出乎意料的是，其意義明顯優於您預期的，這可能對第一代有偏見。那麼通道阻斷劑對於該族群不起作用，對嗎？顯然這裡的情況並非如此。

And then there's a few other ideas that would a priori expect to have AFAC that we're seeing AFAC on prepl models. And, and therefore, we should expect to have as well when a seizure is happening, right? If we could observe like the neuron, so channels are going crazy and that in a, in a very late term way to, to describe it. So stopping that activity is a very, very important for seizure control, independently of the the etiology of the seizure or a general even or origin in the brain anatomically. So we expect quite wide range here of Effy I think what we have to ask the question as well is how consistent is the seizure? Because we want to count them similarly, right? We're going to do one study. It can't be for example, some patients have clusters, others don't have clusters, so we shouldn't putting them together. So that's more of a restriction than anything else.

然後還有一些其他的想法，這些想法先驗地期望有 AFAC，我們在 prepl 模型上看到了 AFAC。因此，當癲癇發作時我們也應該預料到這種情況，對嗎？如果我們可以像觀察神經元一樣觀察，那麼通道就會變得瘋狂，並且以一種非常後期的方式來描述它。因此，停止該活動對於控制癲癇發作非常非常重要，無論癲癇發作的病因或大腦解剖學上的一般事件或起源如何。因此，我們期望 Effy 的範圍相當廣泛，我認為我們也必須問的問題是癲癇發作的一致性如何？因為我們想以類似的方式計算它們，對嗎？我們將進行一項研究。例如，有些病人有聚集性，有些病人沒有，所以我們不應該把他們放在一起。所以這比其他任何事情都更具限制性。

Thank you.

謝謝。

Sure.

當然。

Thank you. And as a reminder to ask a question, please press star 11 on your telephone.

謝謝。提醒一下，如果您想提問，請在電話上按星號 11。

Our next question comes from Laura Chico with Web Bush Securities. You may proceed.

我們的下一個問題來自 Web Bush Securities 的 Laura Chico。您可以繼續。

Hey, good morning very much. Thanks for taking the question. Two clarification questions for me first on the interim for let's call them. I I understand that the timing for the interim has extended to the first quarter 25. What I'm what I'm trying to clarify though is does it, does the actual at which the interim is executed, is that also changing? And I guess maybe asking it differently is if this was expected to be conducted at 50% for example, does this now shift to 60%? Ho hopefully that makes sense. And then I have a follow up.

嘿，早安。感謝您回答這個問題。首先我要先澄清兩個關於過渡期的問題。我了解中期期限已延長至第一季 25。但我想澄清的是，臨時措施的實際執行時間是否也在改變？我想也許換個角度問，如果預計以 50% 的比例進行，現在是否會變成 60%？希望這是有意義的。然後我有一個後續行動。

Now, it makes a lot of sense. Thanks for, for the question are.

現在，這很有意義。謝謝您的提問。

Now. So the the range that we gave for the information fraction, right? That if I understand correctly, that your question was between 50% and, and 75%. So the the range for, for the information does not change. Of course, it is a range because like depends on the exact day of the data transfer and the calculations by the AD MC. But we did not change that.

現在。那麼我們給的資訊分數的範圍是這樣的，對嗎？如果我理解正確的話，您的問題應該是在 50% 到 75% 之間。因此資訊的範圍不會改變。當然，這是一個範圍，因為取決於資料傳輸的特定日期和 AD MC 的計算。但我們並沒有改變這一點。

Okay. That's helpful. And then I guess I understand your comments also about with respect to study, conduct and integrity and the ramifications to study too. My understanding was that these are conducted under the same protocol. So I'm trying to understand the the separation of the release of results. And have you had any feedback from FDA on how they would like to see result communication? Thanks.

好的。這很有幫助。然後我想我也理解了您關於學習、行為和誠信以及學習的影響的評論。我的理解是，這些都是按照相同的協議進行的。所以我試著去理解結果發布的分離。您是否收到 FDA 的回饋，關於他們希望如何進行結果溝通？謝謝。

Yeah. So the last part is easy. So we did not and I don't think they often opine on how results should be communicated. They did though review the data, as I said is a plan which analyzed these studies separately. And and then there's another yet another analysis plan just for the internal analysis that that was revealed and we received it back many months ago from, from the FDA of that. So call on that. So the the influences last operation on the I like definition of operation, meaning you're absolutely correct. The, the studies are patients are randomized to one study or to the other.

是的。所以最後一部分很簡單。所以我們沒有，而且我也不認為他們經常對如何傳達結果發表意見。他們確實審查了數據，正如我所說，這是一個分別分析這些研究的計劃。然後還有另一個分析計劃，僅用於已披露的內部分析，幾個月前我們就從 FDA 收到了該計劃的回應。因此呼籲這一點。因此，我喜歡操作的定義，這意味著你是完全正確的。在這些研究中，患者被隨機分配到一項研究或另一項研究。

It's more on the influence of a potential impression of failure by patients who are on study tube as they finalize the study by a potential needles. Since they wouldn't know their own study tube or study one. So we didn't to unduly influence or create a quasi placebo effects by potentially in the positive case. To be perfectly H1st, here, creating a potential impact on, on what we expect to be applied successfully study. That is a study too. So maybe overly cautious on our end. But again, trying to safeguard the integrity of the entire program.

這更多的是受到患者在透過潛在針頭完成研究時對研究管的潛在失敗印象的影響。因為他們不知道自己的學習管或學習管。因此，我們不會透過潛在的正面案例來過度影響或創造準安慰劑效應。為了完美地實現 H1st，這裡，對我們期望成功應用的研究產生潛在的影響。這也是一項研究。所以我們可能過於謹慎了。但再次強調，我們試圖維護整個計畫的完整性。

Okay. That's helpful Marcio. And maybe the last question for me, I, I think I missed this and I know you commented that earlier, but could you expand a little bit more on the rationale that we start in the Parkinson's indication? I guess I'm, I'm trying to better understand what would be the mechanistic read through from the ET studies to Parkinson's. Thanks very much.

好的。這很有幫助，馬西奧。也許對我來說這是最後一個問題，我想我錯過了這一點，我知道您之前評論過這一點，但是您能否進一步闡述一下我們從帕金森症指徵開始的理由？我想我正在嘗試更好地理解從外星人研究到帕金森氏症的機械解讀。非常感謝。

Yeah. No, no, absolutely. So, I think the from a scientific ration I was very strong to begin with in Parkinson's right where the last time when we actually stopped the, the Parkinson's study had very little to do with our expectation in terms of results for that study and a lot to do with availability of capital. I think as we fast forward that we have significantly more safety data. Of course, we are excited. And, and we expect the study one and study two to be positive for essential timer. The data from a scientific standpoint did not get weak where they got stronger from a Parkinson's rationale.

是的。不，不，絕對不是。因此，我認為從科學的角度出發，我對帕金森氏症的研究從一開始就非常堅定，上次我們真正停止研究時，帕金森氏症研究與我們對該研究結果的預期關係不大，而與資金的可用性有很大關係。我認為，隨著我們快速前進，我們將擁有更多的安全資料。當然，我們很興奮。並且，我們預期研究一和研究二對於基本計時器來說是正面的。從科學角度來看，數據並沒有變弱，而從帕金森氏症的理論角度來看，數據卻變得更強了。

So why you not going to be like using like significant amount of capital to actually re initiate before the results? We want to be ready at the time of the essential three results to reit so maybe that's more of the message there than to have a delay and not necessarily use the mechanism. I think there is a far that.

那麼，為什麼你不打算在結果出來之前投入大量資金來重新啟動呢？我們希望在獲得三個關鍵結果時做好準備，以便重新進行審查，因此這也許更能傳達訊息，而不是延遲並且不一定使用該機制。我認為還有很長的路要走。

Is both go to market strategy here where while movement disorder specialists only have about 5 to 10% of the patients, those five to 10% of the patients co exist a lot more with Parkinson's patients. So that is a kind of a penetration of the market strategy that that is important and the other is just the engagement in general opinion. Leaders. Of course, it goes without saying that we think we can have a meaningful impact on, on parking some patients as well.

兩者都是市場策略，雖然運動障礙專家只接觸大約 5% 到 10% 的患者，但這 5% 到 10% 的患者與帕金森氏症患者共存的幾率要大得多。因此，這是一種重要的市場策略滲透，另一種只是公眾輿論的參與。領導者。當然，毋庸置疑，我們認為我們也可以對一些病人的停車問題產生有意義的影響。

Thanks very much.

非常感謝。

Thank you. I would now like to turn the call back over to Marcio Souza for any closing remarks.

謝謝。現在我想將電話轉回給馬西奧·索薩 (Marcio Souza) 來做結束語。

Thank you so much. I really appreciate Everyone joining the call and, and stay with us as the, as the company continued to to evolve quite positively was a lot went on on the last 10 months and nine months if comes departure, in terms of the transformation of the company for what is going to be next year for registrational programs and, and potentially one MD A submitted mid year or so for account model as we continue to progress, millions of patients will be positively impacted by those drugs and, and billions of dollars and in value will be created to all of us shareholders. So we'd appreciate it looking forward to providing more updates in the near future. And I'm sure we're going to be in touch. Thank you so much.

太感謝了。我非常感謝大家參加電話會議，並請大家繼續關注我們，因為公司在過去的 10 個月和 9 個月裡繼續保持著相當積極的發展，在明年的註冊計劃轉型方面發生了很多事情，並且可能會有一位 MD A 在年中左右提交帳戶模型，隨著我們繼續進步，數百萬患者將受到這些藥物的積極影響，並將為我們所有價值。因此，我們非常感激並期待在不久的將來提供更多更新。我確信我們會保持聯繫。太感謝了。

Thank you. This concludes the conference. Thank you for your participation. You may now disconnect.

謝謝。會議到此結束。感謝您的參與。您現在可以斷開連線。