Anavex Life Sciences Corp (AVXL) 2025 Q3 法說會逐字稿

Good morning, and welcome to the Anavex Life Sciences Fiscal 2025 third-quarter conference call. My name is Clint Tomlinson, and I will be your host for today's call. (Event Instructions). Please note, this conference is being recorded, and the call will be available on Anavex's website at www.anavex.com later today.

早安，歡迎參加 Anavex Life Sciences 2025 財年第三季電話會議。我叫克林特‧湯姆林森，我將擔任今天電話會議的主持人。（活動說明）。請注意，本次會議正在錄製中，通話內容將於今天稍晚在 Anavex 的網站 www.anavex.com 上發布。

With us today is Dr. Christopher Missling, President and Chief Executive Officer; and Sandra Boenisch, Principal Financial Officer.

今天與我們在一起的是總裁兼執行長 Christopher Missling 博士和財務長 Sandra Boenisch。

Before we begin, please note that this conference call -- the company will make some projections and forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. We encourage you to review the company's filings with the SEC, and this includes, without limitation, the company's Forms 10-K and 10-Q, which identify specific factors that may cause actual results or events to differ materially from those described in these forward-looking statements.

在我們開始之前，請注意，本次電話會議—公司將做出一些預測和前瞻性陳述。這些聲明僅是基於當前資訊和預期的預測，涉及許多風險和不確定性。我們鼓勵您查看公司向美國證券交易委員會提交的文件，其中包括但不限於公司的 10-K 表格和 10-Q 表格，這些表格確定了可能導致實際結果或事件與這些前瞻性陳述中描述的結果或事件存在重大差異的具體因素。

These factors may include, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights.

這些因素可能包括但不限於潛在產品的開發和/或商業化固有的風險、臨床試驗或監管批准結果的不確定性、獲得未來資本的需要和能力以及智慧財產權的維護。

With that, I'd like to turn the call over to Dr. Missling.

說完這些，我想把電話轉給 Missling 博士。

Thank you, Clint, and good morning, everyone. Thank you for being with us today to review our most recently reported financial results and to provide our quarterly business update.

謝謝你，克林特，大家早安。感謝您今天與我們一起審查我們最近報告的財務結果並提供我們的季度業務更新。

Our development of noninvasive targeted upstream compounds continues to advance, particularly in the context of Alzheimer's disease and schizophrenia. Clinical feedback highlights the importance of orally administered therapies that are both accessible and effective. At the recent Alzheimer's Association International Conference, AAIC 2025, we presented open-label extension data for blarcamesine, which demonstrated continued clinically meaningful benefit in early-stage Alzheimer's patients, further validating its therapeutic potential.

我們對非侵入性靶向上游化合物的開發正在不斷推進，特別是在阿茲海默症和精神分裂症方面。臨床回饋強調了方便且有效的口服療法的重要性。在最近舉行的阿茲海默症協會國際會議 AAIC 2025 上，我們展示了 blarcamesine 的開放標籤擴展數據，該數據證明了其對早期阿茲海默症患者俱有持續的臨床意義的益處，進一步驗證了其治療潛力。

In June 2025, a survey of Alzheimer's disease stakeholders from European Union member states on current unmet needs in Alzheimer care was conducted. There is a clear acknowledgment that oral therapies would, quote, facilitate things, quote, for many countries and be much more accessible for the respective health care systems, potentially requiring less extensive monitoring and complex administration compared to injectable monoclonal antibodies. This modality difference is seen as a key factor in potential broader market penetration.

2025 年 6 月，對歐盟成員國的阿茲海默症利害關係人進行了一項關於阿茲海默症護理中當前未滿足的需求的調查。人們清楚地認識到，口服療法將為許多國家帶來便利，並且更容易被各自的醫療保健系統所接受，與注射單株抗體相比，可能需要更少的廣泛監測和複雜的管理。這種模式差異被視為潛在更廣泛市場滲透的關鍵因素。

At the end of July, Anavex was honored to be a part of the program at the 2025 Alzheimer's Association International Conference, AAIC, in Toronto. The sharing of knowledge at these central events is important to help advance dementia science to better support the millions of individuals, families, and communities impacted by Alzheimer's disease.

7 月底，Anavex 很榮幸成為多倫多 2025 年阿茲海默症協會國際會議 (AAIC) 計畫的一部分。在這些核心活動中分享知識對於推動癡呆症科學發展至關重要，有助於更好地支持受阿茲海默症影響的數百萬個人、家庭和社區。

At the AAIC 2025 conference, we were pleased to present the latest findings for blarcamesine. The data were presented by Marwan Sabbagh, Professor of Neurology and Chairman of the Advisory Board of Anavex.

在 AAIC 2025 會議上，我們很高興展示了有關 blarcamesine 的最新研究成果。這些數據由神經病學教授兼 Anavex 顧問委員會主席 Marwan Sabbagh 提供。

The data showed that blarcamesine-treated patients continue to accrue benefit through up to four years as measured by the prespecified clinical endpoints, ADAS-Cog13 and ADCS-ADL, respectively. Further presentations at the AAIC 2025 conference featured prespecified precision medicine Phase 2b/3 48-week ANAVEX 2-73-AD-004 double-blind clinical trial data on blarcamesine, confirming the upstream mechanism of blarcamesine restoring impaired autophagy as an early event preceding amyloid-beta and tau.

數據顯示，接受 blarcamesine 治療的患者可繼續獲得長達四年的益處，這分別以預先指定的臨床終點 ADAS-Cog13 和 ADCS-ADL 來衡量。AAIC 2025 會議上的進一步演示展示了預先指定的精準醫療 2b/3 期 48 週 ANAVEX 2-73-AD-004 雙盲臨床試驗數據，該試驗數據針對 blarcamesine，證實了 blarcamesine 恢復受損自噬的上游機制是澱粉樣蛋白-β 和 tau 之前的早期事件。

And now I would like to direct the call to Sandra Boenisch, Principal Financial Officer of Anavex, for a financial summary of the recently reported quarter.

現在，我想請 Anavex 財務長 Sandra Boenisch 提供最近報告的季度財務摘要。

Thank you so much, Christopher, and good morning to everyone on the call. I'm pleased to share with you today our third-quarter financial results for our 2025 fiscal year.

非常感謝你，克里斯托弗，大家早安。我很高興今天與大家分享我們 2025 財年第三季的財務表現。

Our cash position on June 30 was $101.2 million, and we had no debt. During the quarter, we utilized cash and cash equivalents of $12.5 million in operating activities after taking into account changes in noncash working capital accounts. And as of the quarter end, we anticipate at the current adjusted cash utilization rate and range, an approximate runway of more than three years.

截至 6 月 30 日，我們的現金狀況為 1.012 億美元，並且沒有債務。在本季度，考慮到非現金營運資本帳戶的變化後，我們在經營活動中使用了 1,250 萬美元的現金和現金等價物。截至季末，我們預計按照目前調整後的現金利用率和範圍，預計運行時間將超過三年。

Our research and development expenses for the quarter were $10 million as compared to $11.8 million for the comparable quarter of last year. General and administrative expenses were $4.5 million as compared to $2.8 million for the comparable quarter of last year.

本季我們的研發費用為 1,000 萬美元，而去年同期為 1,180 萬美元。一般及行政開支為 450 萬美元，去年同期為 280 萬美元。

Compared to the same quarter of fiscal 2024, an increase in noncash compensation charges was offset by a decrease in overall cash operating expenses due to the completion of a large manufacturing campaign of blarcamesine to support execution and potential commercial readiness as we advance our therapeutic pipeline.

與 2024 財年同期相比，非現金薪酬費用的增加被整體現金運營費用的減少所抵消，這是由於完成了大規模的 blarcamesine 製造活動以支持執行和潛在的商業準備，因為我們推進了治療管道。

And lastly, we reported a net loss of $13.2 million for the quarter or $0.16 per share. Thank you, and back to you, Christopher.

最後，我們報告本季淨虧損 1,320 萬美元，即每股 0.16 美元。謝謝你，克里斯托弗，我們再見。

Thank you, Sandra. In summary, we are focused on continuing to advance our precision medicine compounds. We're excited to be potentially making a difference for individuals suffering from these diseases by presenting a scalable treatment alternative alongside the ease of oral administration. I would now like to turn the call back to Clint for Q&A.

謝謝你，桑德拉。總之，我們專注於繼續推進我們的精準醫療化合物。我們很高興能夠透過提供一種可擴展的治療方案以及便捷的口服給藥方式，為患有這些疾病的個人帶來改變。現在我想把電話轉回給克林特進行問答。

Soumit Roy, Jones Research.

蘇米特·羅伊（Soumit Roy），瓊斯研究公司。

Christopher, quick question on the -- congrats on the four-year data. Trying to understand the graph itself, could you help us explain if the delayed start patients, those were the ones from the placebo arm or the randomized trial?

克里斯多福，快速提問一下──恭喜四年的數據。試著理解圖表本身，您能否幫助我們解釋延遲開始治療的患者是來自安慰劑組還是隨機試驗的患者？

And just the nature of the curve between the Cog13 readout and the ADL, the Cog13 doesn't separate until 96 weeks, that's like two years versus ADL. Is there any specific thing that's going on there?

就 Cog13 讀數和 ADL 之間的曲線性質而言，Cog13 直到 96 週才分離，這就像兩年與 ADL 的對比。那裡發生了什麼具體的事情嗎？

So it's a good question. So you're referring to the four-year open-label extension data?

所以這是一個好問題。所以您指的是四年開放標籤擴充資料？

Yes.

是的。

Yes. So let me quickly explain again what is done. The patients are randomized at the beginning on the trial to either receiving placebo or active arm. Those patients who then finish the 48 weeks will get blarcamesine, all of them.

是的。因此，讓我再次快速解釋一下所做的事情。試驗開始時，患者被隨機分配接受安慰劑組或活性組。所有完成 48 週治療的患者都將接受 Blarcamesine 治療。

So those patients who started blarcamesine, but they were blinded to it, they didn't know, also stay blinded that when they receive blarcamesine if they were receiving blarcamesine in the previous 48 weeks. And they are called the continued blarcamesine or early start group.

因此，那些開始服用布拉卡美辛但對此一無所知的患者，他們不知道，如果他們在過去 48 週內接受過布拉卡美辛治療，那麼當他們接受布拉卡美辛治療時，他們也同樣不知道。他們被稱為繼續 blarcamesine 或早期開始組。

Those patients who now -- after they had placebo because they were randomized to placebo in the first 48 weeks, they now receive also blarcamesine. And what we now look at is the trajectory of the two arms, the early start group, which had blarcamesine since day one and those what we call late start group, which had blarcamesine after the placebo-controlled part.

現在服用安慰劑的患者，因為他們在前 48 週被隨機分配服用安慰劑，所以現在也服用了 blarcamesine。我們現在要看的是兩組的發展軌跡，早期開始組從第一天開始就使用布拉卡美辛，而我們所說的晚期開始組在安慰劑對照部分之後使用布拉卡美辛。

And what we find is that those patients who received the drug later, after placebo first, they do not catch up to the benefit of those patients who had blarcamesine from day one in the previous 48 weeks. And that indicates that if you have Alzheimer's disease, you want to be not getting it too late because you will not get the full benefit of the drug. And the same applies for both cognition, ADAS-Cog13, and activities of daily living or function, ADCS-ADL.

我們發現，那些先服用安慰劑後才開始服用該藥物的患者，並沒有趕上前 48 週從第一天開始服用 Blarcamesine 的患者所獲得的益處。這表明，如果您患有阿茲海默症，您最好不要太晚就醫，因為這樣您將無法充分享受藥物的益處。這同樣適用於認知（ADAS-Cog13）和日常生活活動或功能（ADCS-ADL）。

What we noticed was because of COVID, though, there was not a perfect transition from the end of the trial of 48 weeks into the open-label extension because sites were shut down. And so the patient were just barely able to measure the last measure of 48 weeks, but the open label was not accessible until, in some cases, a year later. But those patients eventually then joined.

然而，我們注意到，由於 COVID，從 48 週試驗結束到開放標籤延期並沒有實現完美的過渡，因為試驗點被關閉了。因此，患者只能勉強測量 48 週的最後測量值，但在某些情況下，直到一年後才能獲得開放標籤。但這些病人最終還是加入了。

And what we found was that we could basically separate two groups, those patients, which were not impacted by COVID, so to speak, by this shutdown trial sites, and those received the drug right away in the active arm specifically. And those had the best performance among all candidates. And those patients who got the drug after a longer pause or drug holiday, we call it also or interruption, they did not benefit as much even if they had previously the drug in the active arm in the placebo-controlled part.

我們發現，我們基本上可以分為兩組：一組患者沒有受到 COVID 的影響，也就是說，沒有受到此次關閉試驗地點的影響；另一組患者則立即在活性組中接受了藥物治療。他們是所有候選人中表現最好的。而那些在較長時間的停藥或藥物假期（我們也稱之為中斷）後服用該藥物的患者，即使他們之前在安慰劑對照組的有效組中服用過該藥物，他們也沒有獲得太多的益處。

So the message is here, the takeaway is twofold. First of all, what I already stated, you want to take the drug as soon as possible once you have an indication and diagnosis of Alzheimer's disease. And secondly, once you start taking blarcamesine, you want to continuously taking it and not interrupting for too long because that would also be not a perfect outcome to keep the cognition and function consistently better.

所以，這裡的資訊和收穫是雙重的。首先，我已經說過，一旦出現阿茲海默症的跡象和診斷，您就需要盡快服用該藥物。其次，一旦開始服用 Blarcamesine，您就需要持續服用，不要停藥太久，因為這也不是保持認知和功能持續改善的理想結果。

And to answer your question about this difference between ADAS-Cog13 and ADL, I think because of these ADAS-Cog13 is more sensitive to immediate actions and ADL has a bit of maybe a latency. The ADL seems to be more smooth in their trajectory than the ADAS-Cog13. So the ADAS-Cog13 is just more sensitive possibly to these changes, which I just described.

回答您關於 ADAS-Cog13 和 ADL 之間的差異的問題，我認為由於這些原因，ADAS-Cog13 對即時操作更敏感，而 ADL 可能有一點延遲。ADL 的軌跡似乎比 ADAS-Cog13 更平滑。因此，ADAS-Cog13 可能對我剛才所描述的這些變化更加敏感。

And the description in the conference in the graph on the slide shows clearly that those patients who were not interrupted or had a short interruption, they, in the ADAS-Cog13, they had a clearly better outcome in the active arm than those who had interruption, what I just basically said a minute ago. I trust that helps to explain the difference.

會議中幻燈片圖表中的描述清楚地表明，在 ADAS-Cog13 中，那些未中斷或短暫中斷的患者在活動組中的結果明顯優於中斷的患者，我剛才基本上已經說過了。我相信這有助於解釋差異。

No, that was super helpful. Were the patients at the beginning of the open-label extension, were they restaged? Were they still mild stage patients or some of them progressed to moderate?

不，這非常有幫助。患者是否處於開放標籤擴展的開始階段，是否進行了重新分期？他們還是輕度患者嗎？或者其中一些已經發展為中度患者？

So certainly, some have advanced, especially the placebo ones have advanced, but we kept all the patients, which was voluntarily, in the trial, irrespective of how they advanced or if they had advanced to more severe form of dementia. So both were -- all were allowed to continue and the majority did.

因此，當然，有些人的病情已經惡化，特別是服用安慰劑的患者，但我們讓所有患者自願參與試驗，無論他們病情如何惡化，或者是否已經發展為更嚴重的癡呆症。因此，所有人都被允許繼續，而且大多數人也確實這樣做了。

But they were not restaged for to assess if they are still mild AD or moderate?

但他們沒有重新進行分期來評估他們是否仍然是輕度 AD 還是中度 AD？

There was no need to because they were eligible to continue to stay on study drug. So irrespective of the staging. Does it make sense? So it's not taking away the ability to continue to stay on the study drug.

沒有必要，因為他們有資格繼續服用研究藥物。因此，無論舞台如何。這有意義嗎？因此，這並不會剝奪繼續服用研究藥物的能力。

No, I was wondering if your drug could even be applicable to the moderate stage patients?

不，我想知道您的藥物是否適用於中期患者？

So we have seen that actually in the Phase 2bs -- Phase 2a study, which was published 2020, that patients with mild to moderate, so more advanced stage, also benefited from blarcamesine. So in a way, we have confirmed a broader therapeutic window for not only for early Alzheimer's disease, but also for mild to moderate.

因此，我們實際上在 2020 年發表的 2bs 期 - 2a 期研究中看到，輕度至中度（即更晚期）的患者也受益於 blarcamesine。因此，從某種意義上說，我們不僅為早期阿茲海默症確認了更廣泛的治療窗口，而且為輕度至中度阿茲海默症也確認了更廣泛的治療窗口。

One last question. Any guidance on the EMA review or commentary back (multiple speakers) --

最後一個問題。關於 EMA 審查或評論的任何指導（多位發言者）——

So we stated that we would not provide comments until the final feedback or review is completed, and we stick to that, but we are excited about the progress.

因此，我們表示，在最終反饋或審查完成之前我們不會提供評論，我們堅持這一點，但我們對進展感到興奮。

Is that a 10-month review? Could you guide us -- help us with understanding the timeline? Filed in November last year?

這是 10 個月的審查嗎？您能指導我們－幫助我們理解時間軸嗎？去年十一月提交的？

So it was filed in November last year. It was accepted in December last year. There's a plus, minus time frame and depends also a little bit on variables, which are sometimes not -- cannot be anticipated. So we estimate that first quarter of next year, we should be able to provide feedback about the feedback from the EMA, first quarter next year.

因此它是在去年 11 月提交的。它於去年 12 月被接受。有一個加號和減號的時間框架，也取決於一些變量，這些變量有時是無法預料的。因此，我們估計明年第一季度，我們應該能夠提供有關 EMA 回饋的回饋，明年第一季。

The next questions will come from Tom Bishop. Tom, you're connected, I think you just need to unmute. Tom looks like having some issues. So I will go to the next person who is Jesse Silveira, and he is from Spirit of the Coast Analytics. I just need you to unmute, Jesse.

接下來的問題來自湯姆畢曉普 (Tom Bishop)。湯姆，您已連接，我認為您只需要取消靜音。湯姆看起來遇到了一些問題。我要聯絡下一位嘉賓，他是 Jesse Silveira，他來自 Spirit of the Coast Analytics。我只需要你取消靜音，傑西。

Can you hear me all right?

你聽見我說話嗎？

Yes. Great.

是的。偉大的。

All right. Clint, Dr. Missling. This is Jesse Silveira with Spirit of the Coast Analytics. We are an independent biotech intelligence group. I wanted to congratulate the Anavex team actually with your newly released data from AAIC.

好的。克林特，米斯林博士。我是 Spirit of the Coast Analytics 的 Jesse Silveira。我們是一家獨立的生物技術情報集團。我實際上想祝賀 Anavex 團隊發布了來自 AAIC 的新數據。

We were exceedingly impressed by the 19.5 months saved by patients. And actually, when benchmarked against Leqembi and Kisunla, the treatment duration to time saved ratio appears really favorable. In fact, at least, according to my group's analysis, it appears to be approximately 76% and 58%, respectively.

患者節省了 19.5 個月的時間，這讓我們印象非常深刻。實際上，與 Leqembi 和 Kisunla 相比，治療時間與節省時間的比率看起來確實很有利。事實上，至少根據我的團隊的分析，這個比例分別約為 76% 和 58%。

I did have a few questions to start. I was wondering, Dr. Missling, theoretically, could CRISPR technology be used to correct SIGMAR1 genotype, so turning mutation gene back to wild-type, in this case, to make blarcamesine and 3-71 more widely efficacious and even potentially increasing the market size. Is that something that could theoretically be done?

我確實有幾個問題要問。我想知道，Missling 博士，理論上，是否可以使用 CRISPR 技術來糾正 SIGMAR1 基因型，從而將突變基因恢復為野生型，在這種情況下，可以使 blarcamesine 和 3-71 更廣泛地發揮作用，甚至可能增加市場規模。從理論上來說，這是可以做到的嗎？

Thank you for the question. I think in theory, it does. The good news is that CRISPR technology is advancing rapidly, and it's very much utilized in oncology, which we also follow very closely. But the good thing is about the blarcamesine application is that the most patients, the vast majority has a very functional and wild-type, fully functional SIGMAR1 gene and other genes.

謝謝你的提問。我認為從理論上來說確實如此。好消息是，CRISPR 技術正在迅速發展，並且在腫瘤學領域得到了廣泛的應用，我們也密切關注。但關於 blarcamesine 應用的好處是，大多數患者，絕大多數都具有非常功能性的野生型、功能齊全的 SIGMAR1 基因和其他基因。

So there is really like the benefit of most patients are -- you don't have to apply anything complicated here to begin with. And let's get that first out there and there's always an ability to further improve from there for those who have a mutation. And a mutation might not be the perfect response to blarcamesine, but still better than placebo. So we should basically allow this to proceed.

因此，這對大多數患者來說確實是一個好處——你一開始就不需要應用任何複雜的方法。讓我們先解決這個問題，對於那些有突變的人來說，總有能力從那裡進一步改進。雖然突變可能不是對 blarcamesine 的完美反應，但仍然比安慰劑要好。所以我們基本上應該允許此事繼續進行。

Okay. Great. Additionally, can you tell us more about any Alzheimer's prevention planning? There appears to be an emphasis on that with -- some of the new slides that you put in the corporate slide deck, some emerging preclinical work, and the 2b/3 delayed start analysis. Are you actually looking to potentially run a preventative trial or a prophylactic trial in the future?

好的。偉大的。此外，您能否告訴我們更多有關阿茲海默症預防計畫的資訊？似乎重點強調了這一點——您在公司幻燈片中放入的一些新幻燈片、一些新興的臨床前工作以及 2b/3 延遲啟動分析。您是否真的希望在未來進行預防性試驗或預防性試驗？

We actually do. We had provided an update recently that there was the chance of -- in animals to prevent the onset of the disease of dementia in animals when they were pretreated with blarcamesine. And those animals who were not pretreated or with placebo, they developed the cognitive dementia in a water maze when they got injected with toxic and better fragments.

我們確實這麼做了。我們最近提供了最新消息，當動物接受布拉卡美辛預處理時，有機會預防動物患上癡呆症。而那些沒有接受過預先治療或安慰劑的動物，在被注射了有毒和更好的碎片後，在水迷宮中患上了認知癡呆症。

And I would also recommend for you to keep an eye on a lookout on a publication, which is peer-reviewed, will address that in more specific detail. So as a consequence of that, we stated that we would plan such a study.

我還建議您留意經過同行評審的出版物，它將更詳細地討論這個問題。因此，我們表示將計劃進行這樣一項研究。

The question is only when we are able to execute this because it will be, of course, a very long study, and that requires more resources. And so we want to first do this step-by-step, by bringing the drug first to market for patients.

問題只是我們何時能夠執行這項任務，因為這當然將是一項非常漫長的研究，需要更多的資源。因此，我們希望首先逐步實現這一目標，首先將藥物推向市場供患者使用。

Okay. That makes sense. And potentially, you would require a partner for that, potentially. And for my final question, we've observed from public lobbying disclosure filings that Anavex has retained Forbes state partners for government relations and lobbying services.

好的。這很有道理。並且，你可能需要一個合作夥伴。我的最後一個問題是，我們從公開遊說披露文件中觀察到，Anavex 已聘請福布斯州合作夥伴提供政府關係和遊說服務。

We also saw a social media post from Congressman, Henry Cuellar back in May, acknowledging a meeting with Anavex. And this seems to signal a concerted effort to engage with policymakers.

我們還看到了國會議員亨利·奎利亞爾 (Henry Cuellar) 五月在社交媒體上發布的一篇帖子，承認與 Anavex 舉行了會面。這似乎顯示我們正齊心協力與政策制定者合作。

Given the critical role of the FDA and other government bodies in the regulatory process for your drug candidates, could you provide some color on the specific strategic objectives of these engagements? And what are your key policy and regulatory goals that you believe this partnership will help you achieve, specifically, are there any particular policy discussions or regulatory frameworks you are tracking closely that could impact your commercialization and reimbursement?

鑑於 FDA 和其他政府機構在你們候選藥物監管過程中發揮的關鍵作用，您能否詳細介紹一下這些合作的具體策略目標？您認為此次合作將幫助您實現哪些關鍵政策和監管目標？具體來說，您正在密切關注的某些特定政策討論或監管框架是否會影響您的商業化和報銷？

We just want to make aware and raise awareness, and it's very commonly done by many, if not all, companies, such activities. And it's really raising the awareness in helping our legislative side to emphasize and provide funding and attention to patients with this terrible disease.

我們只是想讓人們意識到這一點並提高人們的認識，許多公司（如果不是全部的話）都普遍採取這樣的活動。這確實提高了人們的認識，幫助我們的立法部門重視並為患有這種可怕疾病的患者提供資金和關注。

And we think that it's important to always keep them up to date, and they welcome that very much because they received information from these interactions. And the area, as you know, is very dynamic as you can see that the Alzheimer's conference recently provided many new features of several drug updates. And so that is a requirement which we like to participate in, in the education of policymakers about the need and unmet need of patients with dementia and Alzheimer's disease specifically.

我們認為始終讓他們了解最新情況非常重要，他們也非常歡迎這一點，因為他們從這些互動中獲得了資訊。如您所知，這個領域非常活躍，正如您所看到的，阿茲海默症會議最近提供了幾種藥物更新的許多新特點。因此，我們願意參與其中，對政策制定者進行教育，讓他們了解癡呆症和阿茲海默症患者的需求和未滿足的需求。

Sure. That makes sense. And I assume that includes the SIGMAR1 Europe group as well, so teaching regulators and clinicians in Europe as well about SIGMAR1. And I guess to close out my segment here is along those lines, I'm assuming that you have heard of -- and you may have mentioned actually previously, but I assume that you've heard of the new accelerated voucher at the FDA. Is that something that Anavex is interested in and looking into?

當然。這很有道理。我認為這也包括 SIGMAR1 歐洲小組，因此也向歐洲的監管者和臨床醫生傳授有關 SIGMAR1 的知識。我想，我在這裡結束演講的思路是這樣的，我假設你已經聽說過——你可能之前提到過，但我認為你已經聽說過 FDA 的新加速券。這是 Anavex 感興趣並正在研究的事情嗎？

I would say that definitely, yes. I think every company with -- who has a program, which deserves attention and acceleration, especially if it's an unmet need, would very welcome such a program and so do we. So we very much welcome this program, and we look forward to the implementation of it.

我肯定會說是的。我認為，每家擁有值得關注和加速的項目的公司，特別是如果這是一個尚未滿足的需求，都會非常歡迎這樣的項目，我們也是如此。因此我們非常歡迎這個計劃，並且期待它的實施。

I'm sorry, just to finish, would you say that the chances of you acquiring that voucher is dependent on whether or not you receive an approval with the EMA? Or do you not really have an opinion on that?

抱歉，最後，您是否認為您獲得該憑證的機會取決於您是否獲得 EMA 的批准？或者你對此真的沒有什麼看法？

I think that's independent of that. I don't think it has any correlation.

我認為這與此無關。我認為它沒有任何關聯。

Okay. I'm going to try Tom Bishop again. Tom, if you could unmute.

好的。我將再次嘗試聯繫湯姆畢曉普 (Tom Bishop)。湯姆，你能取消靜音嗎？

Can you hear me?

你聽得到我嗎？

Yes. We're good.

是的。我們很好。

As near as I know, the company has only 3-71 in a clinical trial right now. Is that right?

據我所知，該公司目前只有 3-71 處於臨床試驗階段。是嗎？

That's correct. We have in compassionate use, though, we have ANAVEX 2-73 or blarcamesine in Alzheimer's disease right now ongoing.

沒錯。不過，出於同情用藥，我們目前正在使用 ANAVEX 2-73 或 blarcamesine 治療阿茲海默症。

So are all people that were in a prior trial allowed to stay on it basically, even if it's an OLE officially, but like all those (multiple speakers) --

那麼，所有參加過先前審判的人是否都可以繼續留在原地，即使它正式成為 OLE，但就像所有這些（多位發言者）——

Yes, so we have started the trial in Australia, and those patients were the first one to finish the trial, including the open-label extension study, and those were the ones who asked for a continuation. And that started also with the Phase 2a study in Australia.

是的，我們已經在澳洲開始了試驗，這些患者是第一批完成試驗的患者，包括開放標籤擴展研究，他們也是要求繼續試驗的患者。這也是從澳洲的 2a 期研究開始的。

So Australia has, right now, patients up to nine years, including the Phase 2a patient population. Some of them continued to take the drug every day since 2014.

因此，澳洲目前有年齡長達 9 歲的患者，包括第 2a 期患者族群。其中一些人自 2014 年以來每天繼續服用該藥物。

But those on the OLE are also still on it?

但是 OLE 上的那些也還在上面嗎？

The ones in Australia, correct. (multiple speakers) in Australia, yes.

澳洲的那些，正確。 （多位發言者）是的，在澳洲。

No, I mean the full OLE.

不，我指的是完整的 OLE。

Not all of them, only those in Australia continued because the other study participants finished after the open-label extension.

並非所有參與者都繼續了研究，只有澳洲的參與者繼續了研究，因為其他研究參與者在開放標籤擴展後就完成了研究。

Okay. So I noticed that R&D spending is still like $10 million. And so I'm wondering what -- where that's all going, what -- whether you could run down what people are working on kind of puts some meat on that bone and go down through the pipeline in that regard, Parkinson's, Rett, schizophrenia, Fragile X, that would be very helpful to see what's (multiple speakers) --

好的。所以我注意到研發支出仍有 1000 萬美元左右。所以我想知道這一切會走向何方，你是否可以了解人們正在研究什麼，為研究提供一些實質性的內容，並深入了解帕金森氏症、雷特氏症、精神分裂症、脆性 X 綜合徵等相關疾病的進展，這將非常有助於了解（多位發言者）——

Right. Part of this is going into the preparation for manufacturing. So we have a larger amount for the manufacturing of blarcamesine for the CMC. And the preparation of the trials, which we say -- we said we would anticipate to start, that is a Parkinson's disease study. This is a Fragile X study in another rare disease. All of these are also preparation expenses included in this R&D quarter outcome.

正確的。其中一部分用於製造準備。因此，我們有大量用於 CMC 的 blarcamesine 製造。我們說的試驗準備——我們說我們預計將開始，這是一項帕金森氏症研究。這是另一種罕見疾病的脆性 X 染色體研究。所有這些也都是包含在本季研發成果中的準備費用。

Yes, now it's been quite a while since that Parkinson's -- the last Parkinson's study ended. And I'm just wondering what's holding that back or whether you're waiting for EMA results or whatever?

是的，距離上次帕金森氏症研究結束已經有一段時間了。我只是想知道是什麼阻礙了這一點，或者您是否在等待 EMA 結果或其他什麼？

No, it's more like the Parkinson's area has gone through a very dynamic shift in understanding of the disease. And given our recent precision medicine analysis finding in Alzheimer, we want to really increase the chance of success of this Parkinson's trial as well. And one of the things which makes it challenging in Parkinson's disease is that L-dopa is a very good drug and patients with Parkinson do get L-Dopa.

不，這更像是帕金森氏症區域對疾病的理解發生了非常大的轉變。鑑於我們最近在阿茲海默症方面的精準醫療分析結果，我們也希望真正增加這項帕金森氏症試驗的成功機會。治療帕金森氏症的挑戰之一在於左旋多巴是一種非常好的藥物，帕金森氏症患者確實可以服用左旋多巴。

So you have to understand that if you get L-Dopa, and change the dose in the middle of the trial, those patients are not anymore eligible to be included in the analysis. So you lose power and you have to adjust for that.

所以你必須明白，如果你服用左旋多巴，並在試驗中途改變劑量，那麼這些患者就不再有資格納入分析。所以你會失去力量，你必須適應這一點。

So we have to find a way to avoid that to happen. So we try to find the best way to design the study, so it's becoming -- increase the chance of success. And that's what is the reason why we are making this thorough and not jumping right into it.

所以我們必須找到一種方法來避免這種情況發生。因此，我們嘗試找到設計研究的最佳方法，以增加成功的機會。這就是我們要徹底解決這個問題而不是直接進入這個問題的原因。

Okay. And Rett?

好的。還有雷特？

Rett is -- we're really excited about the Rett program, what we've done so far. I think once we have more clarity on the submission with Alzheimer, we would look at that again eventually.

雷特是——我們對雷特計劃以及我們迄今為止所做的一切感到非常興奮。我認為，一旦我們對阿茲海默症的提交有了更清晰的認識，我們最終會再次審視它。

Okay. And regarding the EMA decision, will there be an equivalent of like an FDA advisory committee opinion before that EMA advisory committee, so to speak?

好的。關於 EMA 的決定，在 EMA 諮詢委員會面前是否會有相當於 FDA 諮詢委員會的意見？

I think it works a bit different. The EMA makes a decision based on all participating countries in Europe, which are 27. So everybody has a vote, and that's how they are making the CHMP recommendation and the European Union parliament then either adopts it or changes the view on that. Mostly, they adopt it. So that is how the assessment is done in the European filing.

我認為它的工作原理有點不同。EMA 根據歐洲所有參與國（共 27 個）做出決定。因此每個人都有投票權，這就是他們提出 CHMP 建議的方式，然後歐盟議會要么採納該建議，要么改變對此的看法。大多數情況下，他們會採用它。這就是歐洲申請的評估方式。

Okay. So putting it another way, will we get some preliminary information from the EMA up or down before the final EMA decision is rendered?

好的。換句話說，在做出最終 EMA 決定之前，我們能否從 EMA 上漲或下跌中獲得一些初步資訊？

So I think the final decision will be rendered only after the CHMP provides their recommendation to the EMA. And then the EMA or the European Union makes the recommendation or the approval. So that is -- there is no interim or whatever. So there will be, as I pointed out before, probably first quarter of next year, a result.

因此我認為只有在 CHMP 向 EMA 提供建議後才能做出最終決定。然後 EMA 或歐盟會提出建議或批准。所以，也就是說，不存在任何臨時措施或其他措施。因此，正如我之前指出的，可能明年第一季就會有結果。

Okay. Can you share with us the state-of-the-art of how you'll go forward with selling blarcamesine if it gets approval, for example, hired sales force, a pharma partner, or any interest in somebody acquiring you, some marriage proposals?

好的。您能否與我們分享如果獲得批准，您將如何繼續銷售 blarcamesine 的最新進展，例如，聘請銷售人員、製藥合作夥伴，或者有人有興趣收購您，或者提出一些求婚？

So all options are open. The -- there's definitely an unmet need to treat patients with an oral therapeutic intervention in Europe that applies also for the rest of the world. And so there are several companies which we have started the dialogue with about marketing the drug in Europe.

因此所有選項都是開放的。歐洲的患者確實存在未滿足的口腔治療介入需求，這也適用於世界其他地區。因此，我們已經與幾家公司開始就在歐洲銷售該藥物進行對話。

But also we have a plan and a proposal ready to -- if we think that the shareholder would be better served with marketing the drug in Europe alone. So we are also able to do that if that turns out to be more favorable for shareholders or resulting in a higher shareholder value creation, but we have the options open until we get there.

但如果我們認為僅在歐洲銷售該藥物對股東更有利，我們也已經準備好了計劃和提案。因此，如果事實證明這對股東更有利或能為股東創造更高的價值，我們也可以這樣做，但在實現這一目標之前，我們還有其他選擇。

Okay. And with regards to non-cash compensation expenses, which you signaled out as having gone up. And I guess this is a question for Sandra, but is that increase in large part a function of that line item going up when the stock price goes up? I'm not sure of the formula.

好的。關於非現金薪酬費用，您指出其有所增加。我想這是桑德拉要問的問題，但這種增長是否在很大程度上是由於股價上漲時該項目也隨之上漲？我不確定這個公式。

If the stock price is higher when they're granted, then it does impact the value and make it a higher value, yes. It's also a function of how long the vesting period is and if new awards were granted.

如果授予時股票價格較高，那麼它確實會影響價值並使其價值更高，是的。它也取決於歸屬期的長短以及是否授予了新的獎勵。

I do have a question from Raghuram from H.C. Wainwright. He's having trouble with the connection. So I'll ask the question for him. Christopher, from Raghuram, what are likely to be the most important countries in Europe from a commercial standpoint for blarcamesine?

我確實有一個來自 H.C. Wainwright 的 Raghuram 的問題。他的連接出了問題。所以我會替他問這個問題。來自 Raghuram 的 Christopher，從商業角度來看，歐洲哪些國家可能是 blarcamesine 最重要的國家？

I think I would call out the big three: Germany, France, Italy, and then UK is not European anymore, but those are the countries I would think are the largest one to be focusing on.

我想我會說出三大國家：德國、法國、義大利，然後英國不再是歐洲國家，但我認為這些才是需要重點關注的最大國家。

Okay. A second question from him. Does the potential advent of anti-amyloid antibodies with significantly lower risk of ARIA reduce the need for a safe oral option that does not require MRI-based monitoring?

好的。他提出了第二個問題。抗澱粉樣蛋白抗體的潛在出現是否會顯著降低 ARIA 風險，從而減少對不需要 MRI 監測的安全口服藥物的需求？

I think the survey we received just last month, two months ago, was that there's really like a propensity for the inability to utilize injectable drugs for various reasons. It's not in the DNA, so to speak, of the GPs, general practitioners or neurologists in Europe to administer injectable drugs.

我認為我們上個月（兩個月前）收到的調查顯示，由於各種原因，人們確實傾向於無法使用注射藥物。可以這麼說，歐洲的全科醫師、全科醫師或神經科醫師的 DNA 中並沒有註射藥物的天賦。

It is, for that reason, a very high bar for penetration. And there's an extremely high preference for that reason to offer a oral solution like blarcamesine, which is expected to have, for that reason, a much more significant penetration.

因此，它的滲透門檻非常高。因此，人們非常傾向於提供像 Blarcamesine 這樣的口服溶液，預計這種溶液的滲透性會更強。

Okay. Great. And then his last question is, can you give any insight into when additional orphan indications for blarcamesine may be disclosed?

好的。偉大的。他的最後一個問題是，您能否透露何時可以披露 Blarcamesine 的其他孤兒藥適應症？

So we are preparing and planning a study in another rare disease orphan designation, and we will disclose it once we're getting there. But it's a very exciting indication with high unmet need. So we're very excited about that.

因此，我們正在準備和計劃另一項罕見疾病孤兒病研究，一旦完成，我們就會公佈。但這是一個非常令人興奮的跡象，但仍有大量未滿足的需求。所以我們對此感到非常興奮。

Okay. Great. I may have a follow-up question from Soumit.

好的。偉大的。我可能還有來自 Soumit 的後續問題。

A bit on the commercialization effort. Curious if you can provide us some kind of timeline when you make the decision whether to go solo or make the partnership because we are probably inside the six-month period of potential EMA decision?

稍微談一下商業化努力。好奇的是，當您決定單獨行動還是合作時，您是否可以提供一些時間表，因為我們可能處於 EMA 潛在決策的六個月內？

Yes. So if you look at the historical data of collaborations upfront amount and milestone, there is, of course, a higher shareholder value achieved if you are able to partner something once you have already approval or once you are on market already. That also has been often the case that acquisition or a partnering took place after the company went and marketed itself.

是的。因此，如果您查看合作前期金額和里程碑的歷史數據，那麼如果您能夠在獲得批准或進入市場後與某項產品進行合作，那麼當然可以獲得更高的股東價值。公司在進行自我行銷後，經常會發生收購或合作的情況。

So that would be increased the chances of increasing the value for shareholders, and that's what we are after ultimately. So I would say that is the best way to answer this question.

這樣就增加了增加股東價值的機會，這也是我們最終追求的目標。所以我認為這是回答這個問題的最好方式。

Have you filed in the UK? Because that's not under EMA.

您在英國有提交過申請嗎？因為那不屬於 EMA。

Yes, we are in the planning of doing that. So we mentioned that maybe a quarter or so ago that we are planning to also reach out to other jurisdictions. So this is in the making.

是的，我們正在計劃這麼做。因此，我們大約在一個季度前提到，我們計劃接觸其他司法管轄區。所以這正在醞釀中。

Okay. And one last one is with the EMA, if you can share with us, was there any back and forth between the EMA and their questions that caused the clock to stop after 120 days or 160 days, 181 days? And if CHMP is involved already in the -- for the oral explanation?

好的。最後一個問題是 EMA，如果您可以與我們分享的話，EMA 和他們的問題之間是否存在來回的爭論，導致時鐘在 120 天或 160 天或 181 天後停止？如果 CHMP 已經參與其中——進行口頭解釋？

So the procedure is very standard. There is nothing unusual in the process, and we are abiding by it. So everything is proceeding as standard in the process -- in the review process.

所以程序非常標準。整個過程沒有什麼不尋常的，我們都在遵守。因此，在審查過程中，一切都按標準進行。

So that is the last -- hang on one second. Yes, that's the last question, Dr. Missling. I'll turn it back over to you.

這是最後一個——等一下。是的，這是最後一個問題，米斯林博士。我會把它交還給你。

Thank you very much. In closing, so we'd like to continue to focus on execution and potential commercial readiness as we advance our therapeutic pipeline to potentially improve patients' lives living with these devastating conditions. I'd like to thank you, and good morning to everybody again.

非常感謝。最後，我們希望繼續專注於執行和潛在的商業準備，同時推進我們的治療管道，以潛在地改善患有這些毀滅性疾病的患者的生活。謝謝大家，再次向大家問好。

Thank you, ladies and gentlemen. That will conclude today's conference call. Thank you for participating. You may now disconnect.

謝謝各位，女士們、先生們。今天的電話會議到此結束。感謝您的參與。您現在可以斷開連線。