Belite Bio (BLTE) 2026 年第 2 季法說會：Tinlarebant FDA 審查與 7.8 億美元現金

重點總覽

• 美國 FDA 已受理 Belite Bio 用於治療史塔格氏症 (Stargardt disease) 之 Tinlarebant 的新藥申請 (NDA)，並給予優先審查，設定 PDUFA 目標行動日為 2027 年 2 月 12 日。
• 第三期 DRAGON 試驗數據顯示，接受 Tinlarebant 治療的患者在第 25 個月時，定量自體螢光 (qAF) 較基準線下降約 2%，而對照組 (安慰劑組) 則增加約 20%。
• 2026 財年第二季研發費用自去年同期的 1,100 萬美元增加至 1,820 萬美元，主因與完成第三期臨床試驗相關的權利金支付。
• GAAP 淨虧損自 2025 財年第二季的 1,630 萬美元擴大至 2,840 萬美元；Non-GAAP 淨虧損則自 870 萬美元增加至 2,160 萬美元。
• 截至本季末，Belite Bio 擁有的現金、現金等價物及美國國庫券共計 7.8 億美元。管理層表示，這為潛在獲准後的 Tinlarebant 商業化及推進產品線提供了充足資金。
• 該公司正優先推進美國的審查。管理層預計在獲得 FDA 潛在核准後提交歐洲申請，而日本 PMDA 的審查程序則同步進行中。

關鍵財務數據

業務與營運表現

Tinlarebant 仍是 Belite Bio 2026 財年第二季法說會的核心焦點。FDA 已受理史塔格氏症的 NDA 申請並給予優先審查，指定 PDUFA 目標行動日為 2027 年 2 月 12 日。

Belite Bio 在四個國家的四場醫學會議上發表了第三期 DRAGON 試驗結果。在美國視網膜專家協會 (ASRS) 年會上，該公司報告接受 Tinlarebant 治療的患者 qAF 僅輕微下降，在第 25 個月時較基準線下降約 2%；而同期對照組患者則增加約 20%。

管理層指出，qAF 是有毒雙維生素 A 類化合物 (bisretinoid) 累積的指標，而該物質正是導致史塔格氏症視網膜退化的主因。公司表示，此結果符合 Tinlarebant 的作用機制及其減緩病灶擴大的潛力。

據管理層透露，史塔格氏症計畫在 24 個月後的用藥遵囑性 (compliance) 仍維持在 90% 以上。

DRAGON II 仍主要是針對日本 PMDA 的研究，管理層目前預期該研究不會對美國 NDA 審查提供數據支援。此外，Belite Bio 正於倫敦啟動一項兒童臨床研究，以支援 12 歲以下患者的法規審查程序。

管理層指引

管理層表示，未來六個月的首要任務是取得美國的藥證核准。公司預計在獲得 FDA 潛在核准後推進歐洲的上市申請，以便在不同司法管轄區之間維持法規策略與溝通的一致性。

日本的法規審查程序正與美國同步推進。基於獲得「先驅審查指定制度」(Sakigake) 資格，管理層表示，日本的核准可能會在 FDA 潛在獲准後的約三個月內達成。

Belite Bio 目前預計其地圖狀萎縮 (geographic atrophy) 研究的中期分析將於 2027 年第一季進行，可能安排在 2 月之後，因為 2026 年 12 月與 2027 年 1 月預計將是與 FDA 互動最頻繁的時期。

若 Tinlarebant 獲得核准，管理層預計該公司將根據其罕見兒科疾病認定獲得一張優先審查憑證 (PRV)。Belite Bio 尚未決定是出售還是自行使用該憑證。

風險與觀察重點

• FDA 的核准仍取決於 2027 年 2 月 12 日 PDUFA 目標行動日之前的持續審查程序。
• 管理層表示，目前未安排諮詢委員會會議，但 FDA 仍可能在審查後期提出要求。
• 關於藥品標籤 (適應症範圍) 的討論尚未開始。管理層根據現有數據預期將獲得更廣泛的適應症標籤，但拒絕在 NDA 審查期間對潛在的最終標籤發表評論。
• 歐洲申請時程、日本核准時間以及地圖狀萎縮中期分析的時程，仍取決於 FDA 審查進度及美國的潛在核准。

分析師問答亮點

分析師關注的重點包括 DRAGON II 的角色、兒童藥物開發、法規時程排序、委託製造、潛在適應症標籤範圍以及資金規劃。管理層重申 DRAGON II 主要針對日本市場，預計不會支援美國的 NDA 審查程序。

在適應症標籤範圍方面，公司表示 FDA 尚未與其討論標籤細節。醫療長 Hendrik Scholl 指出，不同年齡層的病灶進展速率大致相似，且背後的 ABCA4 基因功能障礙相同，但他強調公司不會在審查期間預測最終的標籤內容。

Belite Bio 確認其同時與美國國內及海外的委託開發暨製造服務公司 (CDMO) 合作。該公司在電話會議中未提供關於這些設施的更多細節。

管理層亦表示，計劃於 9 月舉辦線上商業日 (Commercial Day)，屆時打算公布其美國市場調查中關於患者人數的調查結果。

法說會完整逐字稿

完整財報電話會議逐字稿

管理層陳述

Operator

Ladies and gentlemen, thank you for joining us, and welcome to the Belite Bio Second Quarter 2026 Earnings Call. [Operator Instructions].

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

Julie Fallon

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.

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.

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

Yu-Hsin Lin

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 U.S.

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.

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 III DRAGON study results at 4 medical conferences across 4 countries, including the recent American Society of Retina Specialists, ASRS, Annual Meeting. 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.

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. 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.

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

Hao-Yuan Chuang

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. 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.

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. We ended the quarter with $780 million in cash, cash equivalents and U.S. 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.

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

Operator

[Operator Instructions]. First question comes from the line of Judah Frommer with Morgan Stanley.

分析師問答

Judah Frommer

Congrats on the progress. 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 U.S. 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 U.S? 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?

Yu-Hsin Lin

Thanks. Good questions. For the DRAGON 2, 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.

Hendrik Scholl

Yes, 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 BH3311 and this will be the basis to inform regulatory processes for patients that are younger than 12 years old.

Operator

And your next question Marc Goodman with Leerink.

Marc Goodman

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?

Yu-Hsin Lin

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

Hao-Yuan Chuang

Yes. Well, the first one is related to the completion of the Phase III 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.

Marc Goodman

How much was the royalty payment?

Hao-Yuan Chuang

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

Marc Goodman

Okay. And then just thoughts on European filing?

Yu-Hsin Lin

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 6 months on getting the drug approved. 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 U.S. given what we discussed with the FDA and the approval and then there will be our strategy for ongoing regulatory filings.

Operator

And your next question comes from Tazeen Ahmad with Bank of America.

Tazeen Ahmad

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? 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.

Yu-Hsin Lin

There's a few questions there. So I'll answer the first one, and I probably have to get you to repeat the last 2, 3 questions. So the first one, we do have a CDMO in the U.S. These are all the -- 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 U.S. and then a U.S.-based CDMO for that. So I hope that answers your question.

What's the second and third question?

Tazeen Ahmad

It was more about the FDA. And given a consolidated time line 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?

Yu-Hsin Lin

So right now, we don't believe there has AdCom been 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.

Operator

And your next question comes from the line of Steve Seedhouse with Cantor.

Steven Seedhouse

Great. Thanks so much. Congrats on the NDA filing acceptance in the U.S. 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?

Yu-Hsin Lin

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

Hao-Yuan Chuang

Well, yes, 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.

Steven Seedhouse

Okay. 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?

Yu-Hsin Lin

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

Steven Seedhouse

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

Yu-Hsin Lin

All right. So the GA interim analysis fall 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.

Operator

Your next question comes from the line of Graig Suvannavejh with Mizuho. [Operator Instructions].

And we'll move on to the next question for now. Next question comes from Yi Chen with H.C. Wainwright.

Yi Chen

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

Yu-Hsin Lin

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

Hendrik Scholl

Yes, 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 under the 18, and 18 to 50, and patients 50 plus, so slightly larger but still a similar progression rate when we look at DDAF progression. Given that the underlying cause of the disease, namely 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.

Yi Chen

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

Yu-Hsin Lin

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

Nathan L. Mata

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

Yi Chen

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

Nathan L. Mata

In the GA study?

Yu-Hsin Lin

In the Stargardt side.

Nathan L. Mata

In excess of 90%.

Yi Chen

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

Yu-Hsin Lin

Japan concurrently is happening at the same time. So given the Sakigake designation, it will probably be around 3 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.

Yi Chen

Got it. Thank you very much.

Operator

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