Belite Bio Inc (BLTE) 2025 Q3 法說會逐字稿

Ladies and gentlemen, thank you for joining us, and welcome to the Belite Bio third-quarter 2025 earnings call. (Operator Instructions)

女士們、先生們，感謝各位的到來，歡迎參加 Belite Bio 2025 年第三季財報電話會議。（操作說明）

I will now hand the call over to Julie Fallon. Please go ahead.

現在我將把電話交給朱莉法倫。請繼續。

Good afternoon, everyone. Thank you for joining us. On the call today are Dr. Tom Lin, Chairman and CEO of Belite Bio; Dr. Hendrik Scholl, Chief Medical Officer; Dr. Nathan Mata, Chief Scientific Officer; and Hao-Yuan Chuang, Chief Financial Officer.

大家下午好。感謝您的參與。今天參加電話會議的有：Belite Bio 董事長兼執行長 Tom Lin 博士；首席醫療官 Hendrik Scholl 博士；首席科學官 Nathan Mata 博士；以及首席財務官 Hao-Yuan Chuang。

Before we begin, let me point out that we will be making forward-looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and actual results may differ materially. We encourage you to consult the risk factors discussed in our SEC filings for additional detail.

在開始之前，我想指出，我們將做出一些基於我們當前預期和信念的前瞻性陳述。這些聲明存在一定的風險和不確定性，實際結果可能與聲明內容有重大差異。我們建議您查閱我們在提交給美國證券交易委員會的文件中討論的風險因素，以了解更多詳情。

And now, I'll turn the call over to Dr. Lin. Dr. Lin?

現在，我將把電話交給林醫師。林博士？

Thank you for joining today's call to discuss our third-quarter 2025 financial results. I'd like to start immediately by highlighting our recent progress. For GA, we completed the enrollment of the Phase 3 PHOENIX trial with 530 subjects.

感謝您參加今天舉行的電話會議，共同討論我們2025年第三季的財務表現。首先，我想重點介紹一下我們最近的進展。對於 GA，我們完成了 PHOENIX 3 期試驗的招募，共有 530 名受試者。

For Stargardt's disease, we have completed the Phase 3 DRAGON trial, and we now look forward to reporting the final top-line data by end of this month. The DRAGON 2 trial have enrolled approximately 35 subjects of our targeted enrollment of approximately 60 subjects, including 10 Japanese subjects. The data from Japanese subjects is intended to expedite a new drug application in Japan. We have been in close communications with Japan's PMDA and Sakigake-designated concierge to ensure that our JNDA is submitted as one of the first countries for market authorization.

針對 Stargardt 病，我們已經完成了 3 期 DRAGON 試驗，現在我們期待在本月底公佈最終的主要數據。DRAGON 2 試驗已招募約 35 名受試者，目標招募人數約 60 名受試者，其中包括 10 名日本受試者。來自日本受試者的數據旨在加快日本新藥申請流程。我們一直與日本藥品和醫療器材管理局 (PMDA) 和 Sakigake 指定的聯絡人保持密切溝通，以確保我們的 JNDA 申請能夠成為首批獲得市場授權的國家之一。

We also recently received positive feedback from regulatory authorities. Specifically, China's NMPA agreed to accept NDA for priority review based on the interim analysis results from the Phase 3 DRAGON trial.

我們最近也收到了監管機構的正面回饋。具體而言，中國國家藥品監督管理局根據 DRAGON 3 期試驗的中期分析結果，同意接受 NDA 進行優先審查。

Additionally, the UK's MHRA agreed to accept conditional marketing authorization application also based on DRAGON's interim analysis results. With the consistent feedback from major regulatory agencies across the world, we are encouraged that the DRAGON trial provides a strong foundation for global submissions and potential approvals.

此外，英國藥品和保健產品監管署 (MHRA) 也同意接受基於 DRAGON 中期分析結果的有條件上市許可申請。鑑於來自世界各地主要監管機構的一致回饋，我們感到鼓舞的是，DRAGON 試驗為全球申報和潛在批准奠定了堅實的基礎。

Lastly, we have completed a $50 million registered direct offering and an upsized $125 million private placement with leading healthcare investors with the potential for an additional $165 million upon full warrant exercise. This investment puts us in a very good position to advance and prepare for tinlarebant's commercialization.

最後，我們完成了 5,000 萬美元的註冊直接發行和 1.25 億美元的私募配售，投資方為領先的醫療保健投資者，如果認股權證全部行使，還有可能額外獲得 1.65 億美元。這項投資使我們處於非常有利的地位，可以推進 tinlarebant 的商業化進程並做好準備。

I'll now turn over the presentation to Hao-Yuan. Hao-Yuan?

現在我將把演講交給郝元。浩源？

Thank you, Tom. For Q3 2025, we had R&D expenses of $10.3 million compared to $6.8 million for the same period last year. The increase was mainly due to expenses related to the DRAGON trial and the PHOENIX trial, partially offsetting by the Australian R&D tax incentive program. It was also due to an increase in share-based compensation expenses.

謝謝你，湯姆。2025 年第三季度，我們的研發支出為 1,030 萬美元，而去年同期為 680 萬美元。成長主要歸因於 DRAGON 試驗和 PHOENIX 試驗的相關費用，部分被澳洲研發稅收優惠計畫所抵銷。這也是由於股權激勵支出增加所致。

Regarding G&A expenses, we had G&A expenses of $12.7 million compared to $2.9 million for the same period last year. The increase was primarily driven by an increase in share-based compensation expenses from the new grant of equity incentive plan this year, which has become higher as our share price and exercise price increased.

關於一般及行政費用，我們的一般及行政費用為 1,270 萬美元，而去年同期為 290 萬美元。此次成長主要是因為今年新授予的股權激勵計畫導致的股份支付費用增加，而隨著我們的股價和行使價格上漲，股份支付費用也隨之增加。

Overall, we had a net loss of $21.7 million compared to a net loss of $8.7 million for the same period last year. It is important to note that, as I said, the majority of our expense increase came from the share-based compensation, which was about $12.9 million and was not cash related.

整體而言，我們淨虧損 2,170 萬美元，而去年同期淨虧損為 870 萬美元。值得注意的是，正如我所說，我們的大部分支出成長來自股權激勵，約 1,290 萬美元，而且與現金無關。

Our total operating cash outflow for the third quarter was approximately $9.3 million, similar to $8.6 million in the second quarter.

第三季我們的經營現金流出總額約 930 萬美元，與第二季的 860 萬美元相近。

Moving to the balance sheet. As Tom shared, we were pleased to complete a registered direct offering and a significant pipe with gross proceeds of total $140 million with potential for up to additional $165 million of our full warrant exercise. With that, at the end of Q3, we had $275.6 million in cash, liquidity from time deposit and US treasury bills.

接下來查看資產負債表。正如湯姆所說，我們很高興完成了註冊直接發行和一筆重要的管道交易，總收益達 1.4 億美元，並且有可能透過行使全部認股權證獲得高達 1.65 億美元的額外收益。因此，截至第三季末，我們擁有 2.756 億美元的現金、定期存款和美國國債流動性。

We have made significant progress toward our key milestone year to date. We sincerely appreciate the continued trust and support from our shareholders. Our balance sheet remains strong and is expected to provide sufficient funding to support our clinical trials and preparation for commercialization. We are well-positioned to achieve our future objectives.

今年迄今為止，我們在實現關鍵里程碑目標方面取得了顯著進展。我們衷心感謝股東們一直以來的信任與支持。我們的資產負債表依然穩健，預計將提供充足的資金來支持我們的臨床試驗和商業化準備。我們已做好充分準備，實現未來的目標。

With that, I'll turn the call back to the operator for Q&A. Operator?

這樣，我就把電話轉回接線生進行問答環節。操作員？

(Operator Instructions) Yi Chen, H.C. Wainwright.

（操作說明）Yi Chen，H.C. Wainwright。

Thank you for taking my questions. So can you tell us whether you have submitted the application to the regulatory agency in China and UK? And if not, when do you plan to do so?

謝謝您回答我的問題。那麼，您能否告知我們您是否已向中國和英國的監管機構提交了申請？如果沒有，您計劃何時這樣做？

No, we have not. We plan to submit our first half next year. As you can see, there's a couple of regulatory agencies that has given us the green light to submit, whether it be based on interim analysis or waiting for the final report for the DRAGON study to come out. We want to maintain the consistent data package across all regulatory agencies. And therefore, the timeline for that will be first half of 2026.

不，我們沒有。我們計劃明年提交上半年的成績。如您所見，已有幾家監管機構批准我們提交申請，無論是基於中期分析還是等待 DRAGON 研究的最終報告發布。我們希望在所有監管機構之間保持資料包的一致性。因此，該方案的實施時間將在 2026 年上半年。

Got it. and can you also provide us with the current amount of shares outstanding after the most recent announcement.

明白了。另外，能否提供一下最近一次公告發布後目前的流通股數？

Hao-Yuan, this probably a question for you.

昊遠，這個問題或許該問你。

Yeah, I think somewhere like the total outstanding shares, I think list on the recent S3. I think somewhere like $35 million.

是的，我想應該在最近的 S3 報告中列出的總流通股數量之類的數據。我覺得大概是3500萬美元左右。

Bruce Jackson, Benchmark.

Bruce Jackson，基準。

Hi, good afternoon. Thank you for taking my questions. Following up on the last question about the international submissions. When do you think you might be submitting the application in Japan?

您好，下午好。謝謝您回答我的問題。關於上一個關於國際投稿的問題，我再補充一下。你預計什麼時候會在日本遞交申請？

Actually, we are still in discussion with Japan on how the -- basically, the different modules that we need to submit with Japan. We're still going through this with Japan. So the expected timeline will be first half. But given that we have several countries that we need to prioritize, we still haven't had a -- we still need to wait until the data comes out and then prioritize which countries will be submitted first.

實際上，我們仍在與日本方面討論——基本上，就是我們需要向日本提交的不同模組。我們與日本的關係仍在發展中。因此，預計時間表將安排在上半年。但鑑於我們有幾個國家需要優先考慮，我們還沒有——我們仍然需要等到數據出來，然後確定哪些國家要優先提交。

Certainly, Japan is up there. But having three, four countries submitted at once, we definitely wouldn't have the bandwidth to handle all those submissions and getting questions from regulatory agencies across several regions. So we still haven't had the list of where to prioritize first, but certainly, FDA is one of the first authorities that we need to submit first.

當然，日本也名列前茅。但如果三、四個國家同時提交申請，我們肯定沒有足夠的人力來處理所有這些申請，也無法回答來自多個地區監管機構的問題。所以，我們還沒有確定優先順序，但可以肯定的是，FDA 是我們需要先提交審批的機構之一。

So yeah. So I don't know if that answers your question, but at this point, we've got several that we had a green light to submit, including Japan.

是的。所以我不知道這是否回答了你的問題，但目前我們已經有好幾個項目獲準提交，其中包括日本。

Yeah. Okay. Then turning over to the PHOENIX trial. Are you going to have an interim analysis that is structured in a way that was similar to the DRAGON trial. So we're going to be testing for either futility or for adequacy of the sample size?

是的。好的。然後轉到 PHOENIX 審判。你們是否會進行一項類似 DRAGON 試驗的結構化的中期分析？所以我們要檢驗的是研究的無效性還是樣本數是否足夠？

Yes, we do. So at this point, we have an interim analysis planned for next year, probably around second half next year. But regarding the -- how we're going to go about that, I probably will refer to Nathan. Do you have a better idea on the structure of the interim analysis?

是的，我們有。因此，目前我們計劃在明年進行一次中期分析，大概在明年下半年左右。但關於——我們將如何著手這件事，我可能會諮詢內森。您對中期分析的架構有更清楚的思路嗎？

Very likely, it will be a sample size re-estimation as we did for DRAGON. Same sort of scenario where we set up a conditional window specifically refer to as a promising zone to look for efficacy trends within that window to determine whether or not we can supplement the sample size with additional subjects.

很可能，我們將像對 DRAGON 一樣重新估計樣本量。同樣的情況是，我們設置一個條件窗口，專門稱為有希望的區域，以在該窗口內尋找療效趨勢，以確定我們是否可以增加受試者的樣本量。

Okay. Super. Then last question for me. The SG&A levels have been moving around a little bit with all the milestone payments. What should we be assuming as kind of like the baseline level for SG&A expenses going forward?

好的。極好的。最後一個問題。由於各項里程碑付款的完成，銷售、一般及行政費用水準略有波動。我們應該假設未來銷售、管理及行政費用的基準水準為何？

Hao?

郝？

Well, Bruce, that's a good one, but it's also a little bit hard to estimate at this point of time. As you can see, we are starting to prepare for commercialization. So we're expanding the team now. So we don't have a clear -- and also many of that will involve some ESOP as well. And ESOP has become a big moving factor based on the share price on the actual expenses being recognized on the income statement.

嗯，布魯斯，這個問題問得好，但目前還很難估計。如您所見，我們正在開始為商業化做準備。所以我們現在正在擴充團隊。所以我們還沒有明確的答案──而且其中許多方案還會牽涉到員工持股計畫（ESOP）。員工持股計畫 (ESOP) 已成為影響股價的重要因素，其依據是損益表中確認的實際費用。

So we have a better understanding about the cash flow. But for the income statement itself, the G&A, it's a little bit hard to estimate a correct number given it was so much related to the valuation of the ESOP.

這樣我們對現金流有了更深入的了解。但就損益表本身而言，即一般及行政費用，由於它與員工持股計畫的估值密切相關，因此很難估算出正確的數字。

(Operator Instructions) Michael Okunewitch, Maxim.

（操作說明）Michael Okunewitch，Maxim。

Hey, guys. Thank you so much for taking my questions today. I just wanted to ask a couple of questions on your commercial preparations. In particular, what steps are you taking right now to prepare for a potential approval and launch? And then how are you prioritizing different regions since the DRAGON trial should serve for several different geographies?

嘿，夥計們。非常感謝您今天回答我的問題。我只是想問幾個關於你們商業準備方面的問題。具體來說，您目前正在採取哪些步驟來為可能的批准和上市做準備？那麼，由於 DRAGON 試驗應該涵蓋多個不同的地理區域，你們是如何確定不同區域的優先順序的呢？

Sure. Hao, do you want to give more details on this?

當然。郝，你想詳細說說這件事嗎？

Sure. Well, apparently US is the focus given the potential size, but we are applying for NDA in all the regions. Probably US or some smaller single market such as Japan will be relatively easy for us to focus on. And we remain open to seek cooperation and partnership for all the other regions. But for now, I think we're targeting on those markets that we think will be easier to take the handle by ourselves.

當然。顯然，鑑於其潛在規模，美國是重點關注對象，但我們正在所有地區申請保密協議。或許美國或像日本這樣較小的單一市場對我們來說會相對容易一些。我們仍然願意與其他所有地區尋求合作與夥伴關係。但就目前而言，我認為我們的目標是那些我們認為更容易自主掌控的市場。

And do you have a sense of how large of a sales force you would need for the US?

您大概知道在美國需要多大規模的銷售團隊嗎？

Sure. We probably will start with 20 people and then maybe up to 40 people.

當然。我們可能先從 20 人開始，然後可能會增加到 40 人。

And then one last for me, and I'll hop back into the queue. Just given that you have raised this additional $125 million and you have a pretty strong cash position, are you anticipating that your current cash should be sufficient for the commercial preparation and launch of tinlarebant?

最後我還要再來一個，然後我就重新排隊。鑑於您已經籌集了額外的 1.25 億美元，並且您的現金狀況相當強勁，您是否預計您目前的現金足以進行 tinlarebant 的商業準備和上市？

Well, that's a good question. So we estimate it could be probably about $200 million to commercialize Stargardt in the US. That's how we designed the recent transaction with additional cash coming from the warrant. So potentially, yes, we think we should have enough. But of course, that is just estimation.

嗯，這確實是個好問題。因此，我們估計在美國實現 Stargardt 商業化可能需要約 2 億美元。這就是我們設計最近這筆交易的方式，額外的現金將來自認股權證。所以，從理論上講，是的，我們認為我們應該有足夠的資源。當然，這只是估計。

(Operator Instructions) Marc Goodman, Leerink.

（操作說明）Marc Goodman，Leerink。

Yeah. Can you confirm the UK basically ask for the same interim information that you sent to the US FDA that got you the breakthrough designation. I mean is all of this the same exact information and in China as well, did they get anything different? Everybody got the same information?

是的。你能確認一下，英國方面是否也要求提供與你之前向美國FDA提交的、獲得突破性療法認定時相同的臨時資訊嗎？我的意思是，這些資訊都完全一樣嗎？在中國，他們得到的資訊有什麼不同嗎？每個人都得到了相同的訊息嗎？

Yeah. So everyone will present the same information. I'll let Hendrik answer those questions since he presented to them. Hendrik?

是的。這樣一來，每個人都會提供相同的資訊。既然亨德里克已經向他們做了報告，那就讓他來回答這些問題吧。亨德里克？

Yeah. Thank you, Tom. Yeah, Marc, this was the same set of information. The type of presentation was different. It was an in-person presentation in Beijing to the NMPA, including a large panel of experts from China. While for the UK, as an example, this was an online meeting with the agency. But the data set that was the basis for the presentation and the discussion was exactly the same.

是的。謝謝你，湯姆。是的，馬克，這是同一套訊息。展示方式有所不同。這是在北京向國家藥品管理局進行的現場匯報，其中包括來自中國的眾多專家。以英國為例，這是與該機構的線上會議。但作為演示和討論基礎的資料集卻完全相同。

There are no further questions at this time. This concludes today's call. Thank you for attending. You may now disconnect.

目前沒有其他問題了。今天的電話會議到此結束。謝謝各位的出席。您現在可以斷開連線了。