ベライト・バイオ（BLTE）2026年第2四半期決算説明会：ティンラレバントのFDA審査と7億8000万ドルの手元資金

主なポイント

• FDAはスターガルト病治療薬ティンラレバントに関するBelite Bioの新薬承認申請（NDA）を優先審査対象として受理し、PDUFA期日を2027年2月12日に設定しました。
• 第III相DRAGON試験のデータでは、ティンラレバント投与群の25か月時点における定量性自発蛍光（qAF）がベースラインから約2%減少したのに対し、プラセボ群では約20%増加しました。
• 2026年第2四半期の研究開発費は、主に第III相試験の完了に伴うロイヤリティ支払いの影響により、前年同期の1,100万ドルから1,820万ドルに増加しました。
• GAAPベースの純損失は、2025年第2四半期の1,630万ドルから2,840万ドルに拡大しました。Non-GAAPベースの純損失は870万ドルから2,160万ドルに拡大しました。
• Belite Bioの当四半期末における現金、現金同等物および米国短期国債の残高は7億8,000万ドルとなりました。経営陣は、これにより承認後のティンラレバントの商業化およびパイプライン推進に向けた資金が確保されると述べています。
• 同社は米国での審査を最優先しています。経営陣は、FDAでの承認可能性を踏まえ、その後に欧州での申請を行う見込みである一方、日本のPMDAにおける手続きは並行して進めていると説明しています。

主な財務データ

事業および業績の動向

ティンラレバントは引き続きBelite Bioの2026年第2四半期決算説明会における中心的な議題となりました。FDAはスターガルト病治療薬のNDAを優先審査対象として受理し、PDUFA期日を2027年2月12日に設定しました。

Belite Bioは4カ国で開催された4つの医学会で第III相DRAGON試験の結果を発表しました。米国網膜学会（ASRS）年次総会において、同社はティンラレバント投与群の25か月時点におけるqAF低下がわずかに留まり、ベースライン比で約2%低下したと発表しました。一方、プラセボ群では同期間中に約20%の増加を記録しました。

経営陣は、qAFをスターガルト病における網膜変性の要因となる有毒なビスレチノイドの蓄積を示すマーカーとして説明しました。同社はこの結果について、ティンラレバントの作用機序および病変の拡大を遅らせる可能性と一致していると述べています。

経営陣によると、スターガルト病プログラムにおける24か月後の服薬コンプライアンスは90%超を維持しました。

DRAGON II試験は引き続き主にPMDA（日本の規制当局）に向けた試験として位置づけられています。経営陣は現時点で、本試験が米国のNDA審査に寄与するとは見込んでいません。また、Belite Bioは12歳未満の患者に対する規制手続きの参考とするため、ロンドンで小児臨床試験を開始しています。

経営陣の見通し

経営陣は、今後6か月間の最優先事項は米国での承認取得であると述べています。管轄区域間での規制戦略および協議の整合性を図るため、FDA承認の可能性を踏まえた上で欧州での申請を推進する計画です。

日本での規制手続きは米国の審査と並行して進められています。先駆的医薬品指定に基づき、経営陣は日本の承認がFDA承認の約3か月後に行われる可能性があると述べています。

Belite Bioは現在、地図状萎縮（Geographic Atrophy）試験の中間解析結果の発表を2027年第1四半期（2月以降の見込み）と想定しています。2026年12月と2027年1月はFDAとの協議が最も多忙を極める時期になると予想されるためです。

ティンラレバントが承認された場合、経営陣は希少小児疾患指定に基づき、同社が優先審査バウチャーを取得すると見込んでいます。Belite Bioはこのバウチャーを売却するか自社で利用するかについての決定はまだ下していません。

リスクと注視点

• FDAの承認は、2027年2月12日のPDUFA期日に向けた現在進行中の審査プロセスに依存しています。
• 経営陣によると、現時点で諮問委員会の開催計画はありませんが、審査が進む中でFDAから開催を要請される可能性は残されています。
• 適応ラベルに関する協議はまだ行われていません。経営陣は利用可能なデータに基づき広範な適応ラベルを想定していますが、NDA審査中であるため、最終的な適応ラベルの可能性についてのコメントは控えました。
• 欧州での申請、日本での承認、および地図状萎縮試験の中間解析の時期は、依然としてFDAの審査プロセスおよび米国での承認の成否に依存しています。

アナリスト質疑応答の要点

アナリストの質問は、DRAGON IIの役割、小児向け開発、規制手続きの順序、製造、適応ラベルの範囲、および資金計画に集中しました。経営陣は、DRAGON IIは主に日本を対象としたものであり、米国のNDA手続きをサポートするものではないと改めて説明しました。

適応ラベルの範囲について、同社はFDAとまだ協議を行っていないと述べました。最高医学責任者（CMO）のヘンドリック・ショール氏は、病変の進行率は年齢層を問わずおおむね類似しており、根本にあるABCA4の機能障害も同様であると指摘しましたが、審査中に最終的な適応ラベルを予測することはないと強調しました。

Belite Bioは、米国内外の医薬品受託開発製造機関（CDMO）と提携していることを確認しました。決算説明会中、同社は施設に関するこれ以上の詳細は明らかにしませんでした。

経営陣はまた、9月にバーチャル形式でコマーシャル・デイを開催し、米国での市場調査に基づく患者数に関する調査結果を発表する予定であると述べました。

決算説明会文字起こし全文

決算説明会の完全なトランスクリプト

経営陣による説明

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.

質疑応答

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.