贝利特生物 (BLTE) 2026财年第二季度业绩电话会：Tinlarebant FDA审评与7.8亿美元现金

核心要点

• FDA已受理Belite Bio针对施格兰氏病（Stargardt disease）提交的Tinlarebant新药上市申请（NDA）并给予优先审评，指定的PDUFA目标行动日期为2027年2月12日。
• III期DRAGON临床试验数据显示，接受Tinlarebant治疗的患者在第25个月时，定量自发荧光（qAF）较基线下降了约2%，而安慰剂组则上升了约20%。
• 2026财年第二季度研发费用从上年同期的1100万美元增至1820万美元，主要由于与完成III期研究相关的特许权使用费支出。
• 2025财年第二季度GAAP净亏损从1630万美元扩大至2840万美元。Non-GAAP净亏损从870万美元增至2160万美元。
• 截至本季度末，Belite Bio拥有的现金、现金等价物及美国国债总额为7.8亿美元。管理层表示，这为Tinlarebant在获得潜在批准后的商业化以及推进后续管线提供了资金保障。
• 该公司正优先推进美国的审评。管理层预计将在获得FDA潜在批准后提交欧洲上市申请，而日本PMDA的审评程序正在同步推进。

关键财务数据

业务与运营表现

Tinlarebant仍是Belite Bio 2026财年第二季度业绩电话会议的核心焦点。FDA已受理针对施格兰氏病的NDA申请并给予优先审评，并指定了2027年2月12日的PDUFA目标行动日期。

Belite Bio在四个国家的四场医学会议上展示了III期DRAGON临床试验的结果。在美国视网膜专家学会年会上，该公司报告称，接受Tinlarebant治疗的患者在第25个月时，qAF受到抑制并小幅下降，较基线下降约2%；而同期安慰剂组患者则增加了约20%。

管理层将qAF描述为有毒双类维生素A累积的标志物，后者是导致施格兰氏病视网膜变性的驱动因素。该公司表示，该结果与Tinlarebant的作用机制及其减缓病变扩大的潜力相符。

管理层表示，施格兰氏病项目中，用药24个月后的合规率仍保持在90%以上。

DRAGON II仍主要是面向日本PMDA的研究。管理层目前预计其不会支持美国的NDA审评。Belite Bio还在伦敦启动了一项儿童研究，为12岁以下患者的监管审批流程提供信息支持。

管理层指引

管理层表示，在未来六个月内获得美国批准是其首要任务。公司预计将在获得FDA潜在批准后推进欧洲的申请，以便在各监管辖区之间保持监管策略与沟通的一致性。

日本的监管审批程序正与美国的审评同步推进。基于获得的Sakigake（先驱导向）认定，管理层表示，日本的批准可能会在FDA获得潜在批准后的三个月左右实现。

Belite Bio目前预计其地图状萎缩研究的中期分析将在2027年第一季度发布，很可能会在2月之后，因为预计2026年12月和2027年1月将是与FDA互动最频繁的时期。

若Tinlarebant获得批准，管理层预计该公司将基于其罕见儿童疾病认定获得一张优先审评券。Belite Bio尚未决定是出售还是使用该审评券。

风险与关注要点

• FDA的批准仍取决于2027年2月12日PDUFA日期之前的持续审评进程。
• 管理层表示目前未计划召开咨询委员会会议，但在审评后期FDA仍可能要求召开。
• 关于标签（适应症范围）的讨论尚未开展。管理层基于现有数据预计将获得更广泛的标签，但拒绝在NDA审评期间对潜在的最终标签发表评论。
• 欧洲申请的时间、日本的批准以及地图状萎缩中期分析的时程安排，仍取决于FDA的审评进程及美国潜在的批准情况。

分析师问答环节亮点

分析师关注的焦点集中在DRAGON II的作用、儿童药物开发、监管顺序、生产制造、潜在标签范围及资金规划。管理层重申，DRAGON II主要针对日本，预计不会用于支持美国的NDA进程。

关于标签范围，公司表示FDA尚未对其进行讨论。首席医学官Hendrik Scholl指出，不同年龄组的病变进展率大致相似，且潜在的ABCA4功能障碍也是相同的，但他强调公司不会在审评期间预测最终的标签内容。

Belite Bio确认正与美国本土及境外（ex-U.S.）的合同开发与生产组织（CDMO）合作。公司在电话会议中未透露有关设施的更多细节。

管理层还表示，计划在9月举办一场线上商业日活动，届时拟公布其在美国市场调研中关于患者数量的研究发现。

业绩电话会议完整文字记录

完整财报电话会议逐字稿

管理层陈述

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.

分析师问答

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.