Belite Bio, Inc (BLTE) 2026 Q2 法說會逐字稿

Ladies and gentlemen, thank you for joining us, and welcome to the Belite Bio second-quarter 2026 earnings call. (Operator Instructions)

各位女士、先生，感謝您加入我們，歡迎參加 Belite Bio 2026 年第二季財報電話會議。(接線員指示)

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

我現在把會議交給 Julie Fallon。請開始。

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.

感謝各位加入我們。今天參與電話會議的有 Belite Bio 董事長兼執行長 Tom Lin 醫師；首席醫療長 Hendrik Scholl 醫師；首席科學長 Nathan Mata 醫師；以及財務長莊皓元。

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. Additionally, today, we will be discussing certain non-GAAP financial measures. Reconciliations to the most directly comparable GAAP measures are provided in the press release we issued today.

在開始之前，我先提醒各位，我們將發表以前瞻性陳述為主的內容，這些陳述係基於我們目前的預期與信念。此類陳述存在若干風險與不確定性，實際結果可能出現重大差異。我們建議您參閱我們向美國證券交易委員會（SEC）提交之文件中所討論的風險因素，以取得更多細節。此外，今天我們也將討論若干非 GAAP 財務衡量指標。與最直接可比之 GAAP 指標的調節表已載於我們今日發布的新聞稿中。

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

接下來我把電話交給 Lin 醫師。Lin 醫師？

Thank you, Julie. Good afternoon, everyone. Thank you for joining our second-quarter 2026 financial results and corporate update call. The first half of this year has been both exciting and deeply productive for Belite Bio. As we rapidly approach a potential regulatory approval of Tinlarebant for Stargardt disease in the US.

謝謝你，Julie。各位下午好。感謝各位參加我們 2026 年第二季財務結果與公司最新進展電話會議。今年上半年對 Belite Bio 而言既令人振奮，也極具生產力。我們正快速邁向 Tinlarebant 在美國用於 Stargardt 病的潛在監管核准。

We are very pleased to announce that the FDA has accepted our new drug application for Tinlarebant with priority review and establishing a PDUFA date of February 12, 2027. We believe this reflects the strength, consistency, and depth of clinical data generated across our development program.

我們非常高興宣布，FDA 已受理我們針對 Tinlarebant 的新藥申請（NDA），並給予優先審查，同時訂定 PDUFA 日期為 2027 年 2 月 12 日。我們相信，這反映了我們整體開發計畫所產生臨床數據的強度、一致性與深度。

In parallel with our pre-commercial preparations, we remain highly engaged with the medical and patient communities. The enthusiasm we are seeing underscores the profound need for a new treatment paradigm in Stargardt disease. This quarter, we presented our Phase 3DRAGON study results at four medical conferences across four countries, including the recent American Society of Retina Specialists, ASRS, Annual Meeting.

在進行商業化前置準備的同時，我們也持續與醫療界與病友社群保持高度互動。我們所看到的熱忱凸顯了 Stargardt 病在新治療典範上的迫切需求。本季我們在四個國家的四場醫學會議上發表了第三期 DRAGON 研究結果，其中包括近期的美國視網膜專科醫師學會（ASRS）年會。

At ASRS, we presented new secondary endpoint data, demonstrating subjects treated with Tinlarebant showed a hold to slightly decrease qAF values decreased by approximately 2% at month 25 compared to baseline. In contrast, subjects in the placebo group exhibited an approximately 20% increase in qAF values over the same period.

在 ASRS 上，我們發表了新的次要終點數據，顯示接受 Tinlarebant 治療的受試者 qAF 值維持不變至略為下降，於第 25 個月相較基線約下降 2%。相較之下，安慰劑組受試者在同一期間 qAF 值約增加 20%。

Quantitative autofluorescence or qAF is a marker of toxic bisretinoid accumulation, a key driver of retinal degeneration in Stargardt disease. The prevention or reduction of qAF strongly aligns with Tinlarebant mechanism of action, reinforcing its potential to hold or slow lesion growth.

定量自體螢光（quantitative autofluorescence，qAF）是有毒雙視黃質（bisretinoid）累積的指標，而這是 Stargardt 病視網膜退化的關鍵驅動因素。預防或降低 qAF 與 Tinlarebant 的作用機轉高度一致，進一步支持其有望維持或減緩病灶增長。

Looking ahead, we remain confident in our data, our science, and the transformative potential of Tinlarebant for patients living with Stargardt disease. We look forward to providing further updates as they become available.

展望未來，我們對自身數據、科學基礎，以及 Tinlarebant 為 Stargardt 病患者帶來變革性潛力仍充滿信心。我們也期待在有更多進展時提供進一步更新。

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

接下來我把簡報交給皓元，請他說明財務狀況。Hao？

Thank you, Tom. We have had a strong first half of the year and continue to execute well against our plan. Let me recap our financial statements. For the second quarter of 2026, our R&D expenses were $18.2 million compared to $11 million for the same period in 2025. The increase was primarily due to a royalty payment for additional milestone achieved under the license agreement.

謝謝你，Tom。我們今年上半年表現強勁，並持續依計畫良好執行。我先回顧我們的財務報表。2026 年第二季，我們的研發費用為 1,820 萬美元，較 2025 年同期的 1,100 萬美元增加。增加主要是因為在授權協議下達成額外里程碑而支付的權利金款項。

On a non-GAAP basis, excluding share-based compensation expenses, R&D expenses for second quarter were $17.2 million compared to $8.6 million in the second quarter of 2025. SG&A expenses in Q2 was $16.7 million compared to $6.5 million for the same period in 2025. The increase was primarily due to increase in professional service fee, wages and salary resulting from our team expansions.

以非 GAAP 基礎計算（排除股份基礎給付費用），第二季研發費用為 1,720 萬美元，較 2025 年第二季的 860 萬美元增加。第二季銷售、一般及行政（SG&A）費用為 1,670 萬美元，較 2025 年同期的 650 萬美元增加。增加主要來自專業服務費、薪資與工資上升，係因團隊擴編所致。

On a non-GAAP basis, SG&A expenses for the second quarter were $10.9 million compared to $1.3 million in 2025 second quarter. The GAAP net loss in the second quarter was $28.4 million compared to $16.3 million in the same quarter in 2025. On a non-GAAP basis, we reported a net loss of $21.6 million for the second quarter compared to $8.7 million in 2025 same quarter.

以非 GAAP 基礎計算，第二季 SG&A 費用為 1,090 萬美元，較 2025 年第二季的 130 萬美元增加。第二季 GAAP 淨損為 2,840 萬美元，較 2025 年同季的 1,630 萬美元增加。以非 GAAP 基礎計算，我們第二季淨損為 2,160 萬美元，較 2025 年同季的 870 萬美元增加。

We ended the quarter with $780 million in cash, cash equivalents, and US treasury bills. Overall, our balance sheet remains very strong, and we are extremely well funded into the future with a cash runway to commercialize Tinlarebant following a potential regulatory approval and to continue to advance our pipelines.

本季末，我們持有的現金、約當現金及美國國庫券為 7.80 億美元。整體而言，我們的資產負債表仍非常穩健，資金極為充裕；我們的現金續航力足以在 Tinlarebant 潛在獲得監管核准後推動商業化，並持續推進我們的產品線。

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

接下來我把電話交回接線員，進行問答。接線員？

(Operator Instructions)

(接線員指示)

Judah Frommer, Morgan Stanley.

Judah Frommer，摩根士丹利。

Yeah. Hi, guys. Congrats on the progress and thanks for taking the questions. A couple from us. I guess with the NDA accepted now, what are your thoughts on the role that DRAGON II can play for the US filing and/or regulatory process? Any incremental interaction with FDA that would shed light on what that trial could be potentially utilized for in the US?

是的。嗨，各位。恭喜進展順利，也謝謝你們回答問題。我們這邊有幾個問題。我想既然 NDA 現在已被受理，你們如何看待 DRAGON II 在美國申報和／或法規審查流程中可能扮演的角色？是否會與 FDA 有任何額外互動，能讓我們更了解該試驗在美國可能被如何運用？

And then, latest thinking on going lower in age going into peds for Tinlarebant, do you have trial plans to move the label below 12 years old in the near term? Thank you.

另外，關於 Tinlarebant 在兒科往更低年齡層延伸的最新想法，你們近期是否有試驗計畫，將適應症標籤往 12 歲以下推進？謝謝。

Thanks. Good questions. For the DRAGON II, I think at this stage, it's still pretty much a Japan study for the PMDA. Right now, we don't think -- we don't believe that the DRAGON II will contribute to the NDA process. As for the pediatric study, we do have plans, and I'll let Hendrik shed more light on the details of that study.

謝謝。問題很好。關於 DRAGON II，我認為在這個階段，它基本上仍然是一項提供給日本 PMDA 的日本研究。目前我們不認為——我們不相信 DRAGON II 會對 NDA 流程有所貢獻。至於兒科研究，我們確實有計畫，我會請 Hendrik 進一步說明該研究的細節。

Yeah, happy to. Thank you, Tom. So we are initiating a PIP study, a pediatric study in London, where we will investigate Tinlarebant in patients of the age 3 to 11, and this will be the basis to inform regulatory processes for patients that are younger than 12 years old.

好的，很樂意。謝謝你，Tom。我們正在啟動一項 PIP 研究，也就是在倫敦進行的兒科研究，將在 3 到 11 歲的患者中評估 Tinlarebant，這將作為支持 12 歲以下患者法規申請流程的基礎。

Marc Goodman, Leerink.

Marc Goodman，Leerink。

Yeah, hi. Could you tell us how much the royalty payment was, the one-timer that's within R&D? Second question, just tell us what you're thinking with respect to European filing? And then third, have you done any claims database analysis to figure out like exactly the number of patients that are in the United States that have actually under the claims database? Thanks.

是的，你好。你們能否告訴我們那筆一次性的權利金支付金額是多少（列在研發費用裡的那筆）？第二個問題，想請你們談談對歐洲申報的想法？第三，你們是否做過任何理賠資料庫（claims database）分析，以釐清美國在理賠資料庫中實際有多少患者？謝謝。

Hao, do you want to take this, given that it's the royalty payment?

Hao，這題你要不要回答？因為是權利金支付的問題。

Yep. Well, the first one is related to the completion of the Phase 3 study. And I can also take the third question. We will -- as we said on the press release, we do plan to host a Commercial Day event, it's going to be virtual in September, and we'll disclose about the numbers that we have surveyed about the question you just asked.

好。第一題與第三期研究完成有關。我也可以回答第三題。我們——如同新聞稿所說，我們計畫舉辦一場 Commercial Day 活動，將在 9 月以線上形式進行，屆時會揭露我們針對你剛才提到的問題所調查到的相關數據。

How much was the royalty payment?

那權利金支付金額是多少？

No, we cannot disclose that, Columbia asked us to keep that as a confidential, but yeah, it's related to the Phase 3 completion.

不，我們無法揭露。Columbia 要求我們將其保密；但沒錯，這與第三期完成有關。

Okay. And then just thoughts on European filing?

好的。那對歐洲申報的想法呢？

Okay. So I can take that. So right now, we are focused on the FDA with the PDUFA date in February 12. So that's our top priority, we'll be highly focused in the next six months on getting the drug approved.

好的。我來回答。目前我們的重點放在 FDA，PDUFA 日期是 2 月 12 日。這是我們的首要優先事項，接下來六個月我們會高度聚焦在讓藥物獲得核准。

So the European filing will probably be sometime after the FDA approval, we want to align everything with the FDA, the approval and all that. And there will be the consistent message and communications with the regulatory authorities outside of the US given what we discussed with the FDA and the approval and then there will be our strategy for ongoing regulatory filings.

因此，歐洲申報可能會在 FDA 核准之後的某個時間點進行；我們希望一切都與 FDA 對齊，包括核准等事項。並且，基於我們與 FDA 討論的內容與核准情況，對美國以外的法規主管機關也會有一致的訊息與溝通，之後也會有我們持續推進各地法規申報的策略。

Tazeen Ahmad, Bank of America.

Tazeen Ahmad，美國銀行。

In terms of manufacturing, have you stated where your manufacturing site is and whether or not that facility has completed an FDA inspection recently? Or is that going to be part of the requirement to get approval?

在製造方面，你們是否已說明製造廠址在哪裡？以及該設施最近是否已完成 FDA 稽查？或者那會是取得核准所需的一部分要求？

And then secondly, I just wanted to get your latest thoughts on the possibility of an AdCom just given the consolidated time that the FDA would have to review. When do you think is the latest realistically that you would be told if the agency decided to hold on? Thanks.

第二，我想了解你們對於是否可能召開 AdCom（諮詢委員會會議）的最新看法，考量到 FDA 審查時程相對緊湊。你們認為最晚在什麼時間點，才會被告知若主管機關決定要召開？謝謝。

So there's a few questions there. So I'll answer the first one, and I probably have to get you to repeat the last two, three questions. So the first one, we do have a CDMO in the US. These are all the -- but we're not at the privileged to review right now the names of the CDMOs, but these are all big names in the field -- in the industry. So we have ex US and then a US-based CDMO for that.

這裡有幾個問題。我先回答第一個，後面兩三個可能要請你再重複一次。第一個，我們在美國有一家 CDMO。這些——但我們目前不方便披露 CDMO 的名稱，不過它們都是這個領域——這個產業裡的大廠。所以我們在美國以外以及美國本土都有 CDMO。

So I hope that answers your question. What's the second and third question?

希望這回答了你的問題。第二和第三個問題是什麼？

It was more about the FDA. And given a consolidated timeline for review, what is your thought about having an AdCom as the agency talked about that? And realistically, when is the latest they could tell you if they were going to give you an AdCom?

主要是關於 FDA。在審查時程較為緊湊的情況下，你們對於可能召開 AdCom 有什麼看法？主管機關有談到這件事嗎？以及實務上最晚他們可能在什麼時候告訴你們，是否會安排 AdCom？

So right now, we don't believe there has AdCom being planned, but that doesn't mean that further down the line, the FDA would want to use AdCom, so nothing on that right now. So I would say that once we have more updates further down the line, then we'll probably review that -- update that at a more appropriate time. But at this stage, we just received the acceptance, so we don't have any further details on that.

目前我們不認為有規劃 AdCom，但這不代表之後 FDA 不會想要使用 AdCom，所以目前沒有這方面的資訊。我會說，等到後續有更多更新時，我們會在更適當的時間點再檢視——並更新這件事。但在這個階段，我們才剛收到受理通知，所以沒有更多細節。

Steve Seedhouse, Cantor.

Steve Seedhouse，Cantor。

Great. Thanks so much. Congrats on the NDA filing acceptance in the US. I was hoping you could just confirm or clarify that you expect a priority review voucher if you receive approval and if so, if you'd look to auction that just for the purposes of us modeling cash runway?

太好了。非常感謝。恭喜你們在美國的 NDA 申請獲受理。我想請你們確認或釐清一下：如果你們獲得核准，是否預期會拿到優先審查券（priority review voucher）？若是的話，你們是否會考慮把它拿去拍賣，以便我們在模型中估算現金跑道？

Hao, do you want to have the cash runway? You want to answer this?

Hao，你要談一下現金跑道嗎？你想回答這題嗎？

Well, yeah, we do expect that if we receive approval, we should get the priority review voucher just because we do have the rare pediatric disease designation. We have not decided whether we're going to sell it or we're going to use it. So we will confirm that later, but we continue to monitor the market and our own pipeline, et cetera.

嗯，是的，我們確實預期如果獲得核准，我們應該會拿到優先審查券，因為我們有罕見兒科疾病（rare pediatric disease）資格認定。我們尚未決定要出售它還是自行使用。所以我們之後會再確認，但我們會持續關注市場以及我們自身的產品線等。

Okay. Thanks for that. And then I also was hoping you could just provide an update on the geographic atrophy trial, whether you're still planning an interim readout later this year and what the precise timing of an update from that interim analysis might be? Thank you.

好的。謝謝你的說明。另外我也想請你們更新一下地理性萎縮（geographic atrophy）試驗的進度：你們是否仍計畫在今年稍晚進行期中讀出？以及該期中分析更新的確切時間點大概會是何時？謝謝。

Sure, I can answer this question. But isn't that the question regarding the cash runway?

當然，我可以回答這個問題。但這不是關於現金跑道的問題嗎？

I was just interested in the voucher pediatric voucher for our own modeling purposes, but however do you want to answer it. Thank you.

我只是為了我們自己的建模目的，想了解那張兒科優先審查券（pediatric voucher），不過你們想怎麼回答都可以。謝謝。

All right. Thanks. So the GA interim analysis falls during the busiest time with interacting with the FDA. So with the PDUFA date in mid-February, I would expect the busiest time to be in December and January 2027. So with that time line, our top priority is with the FDA approval. So I suspect that with the interim analysis for the GA will probably be sometime first quarter next year, probably after February.

好的。謝謝。GA 的期中分析時間點會落在與 FDA 互動最繁忙的時期。由於 PDUFA 日期在 2 月中旬，我預期最忙的時間會是在 2027 年 12 月與 2027 年 1 月。因此在這個時間表下，我們的首要任務是取得 FDA 核准。所以我推測 GA 的期中分析可能會在明年第一季的某個時間點，可能在 2 月之後。

Graig Suvannavejh, Mizuho. (Operator Instructions) And we'll move on to the next question for now.

Graig Suvannavejh，Mizuho。（接線員指示）我們先進行下一個問題。

Yi Chen, H.C. Wainwright.

Yi Chen，H.C. Wainwright。

Thank you for taking my questions. Just to clarify, has the FDA clearly indicated that the label will include patients over the age of 20 years old? Is that correct?

謝謝讓我提問。我想確認一下：FDA 是否已明確表示標籤會涵蓋 20 歲以上的患者？是這樣嗎？

So right now, there haven't been any discussion on the label yet. I believe there will come sometime data in the process, in the review process. But at this stage, given the data and all that, we expect that we would be able to get the full label or the more broader label.

目前為止，還沒有就標籤進行任何討論。我相信在審查流程的某個階段會開始討論。但在現階段，基於現有數據等，我們預期能取得完整標籤或更廣泛的標籤。

I'll ask Hendrik to give more expert advice on this. Hendrik?

我請 Hendrik 以更專業的角度補充說明。Hendrik？

Yeah, I'm happy to. And I think it's important to understand that lesion growth is not dramatically different across different age groups. That was shown in the ProgStar study, we have essentially the same progression rate of patients any age underneath 18, and 18 to 50, and patients 50 plus, so slightly larger but still a similar progression rate when we look at DDAF progression.

好的，我很樂意。我認為重要的是要理解：不同年齡族群之間的病灶生長並沒有顯著差異。ProgStar 研究已顯示，18 歲以下、18 到 50 歲，以及 50 歲以上患者的進展速率基本相同；50 歲以上略大一些，但當我們看 DDAF 進展時，仍是相近的進展速率。

Given that the underlying cause of the disease, ABCA4 dysfunction, is exactly the same, I would see no reason why the label would not include patients older than 20. But I think it's important that we do not really want to comment on potential label while the NDA is under review.

鑑於疾病的根本原因——ABCA4 功能失常——完全相同，我看不出標籤不涵蓋 20 歲以上患者的理由。但我認為重要的是，在 NDA 審查期間，我們不太希望評論潛在的標籤內容。

Got it. Do you currently have data regarding how many more percentage of patients are compliant with the dosing regimen after 24 months?

了解。你們目前是否有數據顯示，在 24 個月後，有多少比例的患者仍遵從（compliant）給藥方案？

Sure. Nathan, do you want to answer this question?

當然。Nathan，你想回答這個問題嗎？

I'm sorry, could you repeat the question? Sorry, I think my volume --

不好意思，你可以重複一下問題嗎？抱歉，我的音量--

What percentage of patients are -- have been compliant with the dosing regimen after 24 months?

在 24 個月後，有多少比例的患者——仍然遵從給藥方案？

In the GA study?

是在 GA 研究嗎？

No, no. In the Stargardt side.

不是，不是。是在 Stargardt 那邊。

In excess of 90%.

超過 90%。

Okay. And my last question is what's your estimate timeline for submission in Japan?

好的。我最後一個問題是：你們預估在日本的送件時間表是什麼？

Japan concurrently is happening at the same time. So given the Sakigake designation, it will probably be around three months after FDA approval, they want to approve the drug in Japan. So it's happening as we speak with the FDA submission and the PMDA submission is in parallel.

日本那邊是同步進行的。因此，考量 Sakigake 認定，可能會在 FDA 核准後約三個月左右，他們希望在日本核准該藥。所以目前正同步進行；我們向 FDA 的送件與向 PMDA 的送件是並行的。

Got it. Thank you very much.

了解。非常感謝。

(Operator Instructions) And I see no further questions at this time. This concludes today's call. Thank you for attending. You may now disconnect.

（接線員指示）我目前沒有看到其他問題。今天的電話會議到此結束。感謝各位參與。您現在可以掛線。