Belite Bio (BLTE) Earnings Call Q2 2026: FDA-Prüfung von Tinlarebant und 780 Mio. USD Barmittel

Wichtigste Erkenntnisse

• Die FDA hat den Zulassungsantrag (NDA) von Belite Bio für Tinlarebant zur Behandlung des Morbus Stargardt im Rahmen eines bevorzugten Prüfungsverfahrens angenommen und das PDUFA-Datum auf den 12. Februar 2027 festgelegt.
• Die Daten der Phase-III-Studie DRAGON zeigten, dass die quantitative Autofluoreszenz (qAF) bei mit Tinlarebant behandelten Patienten in Monat 25 um etwa 2 % gegenüber dem Ausgangswert sank, verglichen mit einem Anstieg von rund 20 % in der Placebogruppe.
• Im zweiten Quartal 2026 stiegen die Aufwendungen für Forschung und Entwicklung auf 18,2 Mio. US-Dollar gegenüber 11,0 Mio. US-Dollar im Vorjahr, was in erster Linie auf eine Lizenzgebührenzahlung im Zusammenhang mit dem Abschluss der Phase-III-Studie zurückzuführen ist.
• Der Nettoverlust nach GAAP weitete sich von 16,3 Mio. US-Dollar im zweiten Quartal 2025 auf 28,4 Mio. US-Dollar aus. Der Non-GAAP-Nettoverlust stieg von 8,7 Mio. auf 21,6 Mio. US-Dollar.
• Belite Bio beendete das Quartal mit liquiden Mitteln, Zahlungsmitteläquivalenten und US-Schatzwechseln in Höhe von 780 Mio. US-Dollar. Laut Management sichert dies die Finanzierung für die Kommerzialisierung von Tinlarebant nach einer potenziellen Zulassung und treibt die Entwicklung der Pipeline voran.
• Das Unternehmen räumt dem US-Prüfungsverfahren Priorität ein. Das Management rechnet mit einem Antrag auf Zulassung in Europa nach einer potenziellen FDA-Zulassung, während das PMDA-Verfahren in Japan parallel verläuft.

Wichtige Finanzdaten

Geschäftliche und operative Entwicklung

Tinlarebant stand weiterhin im Mittelpunkt der Telefonkonferenz von Belite Bio zu den Ergebnissen des zweiten Quartals 2026. Die FDA nahm den Zulassungsantrag (NDA) für Morbus Stargardt im Rahmen eines bevorzugten Prüfungsverfahrens an und legte das PDUFA-Datum auf den 12. Februar 2027 fest.

Belite Bio präsentierte die Ergebnisse der Phase-III-Studie DRAGON auf vier medizinischen Kongressen in vier Ländern. Auf der Jahrestagung der American Society of Retina Specialists berichtete das Unternehmen, dass die qAF bei den mit Tinlarebant behandelten Patienten nur leicht zurückging und in Monat 25 um etwa 2 % unter dem Ausgangswert lag. Bei den Patienten der Placebogruppe wurde im selben Zeitraum ein Anstieg von rund 20 % verzeichnet.

Das Management beschrieb die qAF als Marker für die Ansammlung toxischer Bisretinoide, die ein Treibsatz für die Netzhautdegeneration bei Morbus Stargardt ist. Das Unternehmen erklärte, dieses Ergebnis stehe im Einklang mit dem Wirkmechanismus von Tinlarebant und dessen Potenzial, das Läsionswachstum zu verlangsamen.

Die Therapietreue bei der Dosierung im Stargardt-Programm lag nach Angaben des Managements auch nach 24 Monaten weiterhin bei über 90 %.

DRAGON II bleibt primär eine auf Japan ausgerichtete Studie für die PMDA. Das Management geht derzeit nicht davon aus, dass sie zur US-NDA-Prüfung beitragen wird. Zudem leitet Belite Bio eine pädiatrische Studie in London ein, um die Zulassungsverfahren für Patienten unter 12 Jahren zu unterstützen.

Prognose des Managements

Das Management erklärte, der Erhalt der US-Zulassung sei in den nächsten sechs Monaten die oberste Priorität. Das Unternehmen rechnet damit, nach einer potenziellen FDA-Zulassung einen Zulassungsantrag in Europa einzureichen, damit seine regulatorische Strategie und Kommunikation über die verschiedenen Jurisdiktionen hinweg abgestimmt sind.

Das japanische Zulassungsverfahren verläuft parallel zur US-Prüfung. Auf Basis des Sakigake-Status erklärte das Management, dass eine japanische Zulassung etwa drei Monate nach einer potenziellen FDA-Zulassung erfolgen könnte.

Belite Bio erwartet die Zwischenanalyse für seine Studie zur geographischen Atrophie nun im ersten Quartal 2027, voraussichtlich nach Februar, da für Dezember 2026 und Januar 2027 die zeitintensivsten Interaktionen mit der FDA erwartet werden.

Sollte Tinlarebant zugelassen werden, geht das Management davon aus, dass das Unternehmen aufgrund des Status für seltene pädiatrische Erkrankungen einen Priority Review Voucher erhält. Belite Bio hat noch nicht entschieden, ob der Voucher verkauft oder selbst genutzt wird.

Risiken und wichtige Aspekte

• Die FDA-Zulassung bleibt vom laufenden Prüfungsverfahren vor dem PDUFA-Datum am 12. Februar 2027 abhängig.
• Das Management gab an, dass derzeit keine Sitzung des Beratungsausschusses geplant ist, die FDA ein solches Treffen im späteren Verlauf der Prüfung jedoch noch anfordern könnte.
• Gespräche über die genaue Indikation (Label) haben noch nicht stattgefunden. Das Management erwartet auf Basis der verfügbaren Daten eine breitere Indikation, lehnte es jedoch ab, sich während der laufenden NDA-Prüfung zum potenziellen endgültigen Label zu äußern.
• Der Zeitplan für den europäischen Antrag, die japanische Zulassung und die Zwischenanalyse zur geographischen Atrophie bleibt abhängig vom FDA-Verfahren und einer potenziellen US-Zulassung.

Wichtigste Punkte der Fragerunde mit Analysten

Die Analysten konzentrierten sich auf die Rolle von DRAGON II, die Entwicklung im pädiatrischen Bereich, die Abfolge der Zulassungsschritte, die Herstellung, den potenziellen Umfang der Indikation und die Liquiditätsplanung. Das Management bekräftigte, dass DRAGON II hauptsächlich für Japan vorgesehen ist und voraussichtlich nicht zur Unterstützung des US-NDA-Verfahrens dient.

Zum Indikationsumfang teilte das Unternehmen mit, dass die FDA diesen noch nicht besprochen hat. Chief Medical Officer Hendrik Scholl merkte an, dass die Läsionsprogressionsraten über die Altersgruppen hinweg weitgehend ähnlich seien und die zugrunde liegende ABCA4-Dysfunktion identisch sei, betonte jedoch, dass das Unternehmen während der Prüfung keine Prognosen zum endgültigen Label abgeben werde.

Belite Bio bestätigte, dass das Unternehmen sowohl mit Auftragsentwicklern und -herstellern (CDMOs) in den USA als auch außerhalb der USA zusammenarbeitet. Nähere Einzelheiten zu den Produktionsstätten nannte das Unternehmen während der Konferenz nicht.

Das Management gab zudem bekannt, im September einen virtuellen Commercial Day veranstalten zu wollen, auf dem es Ergebnisse zu Patientenzahlen aus seiner US-Marktforschung präsentieren möchte.

Vollständiges Transkript der Telefonkonferenz

Vollständiges Transkript der Telefonkonferenz

Ausführungen des Managements

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.

Fragen und Antworten

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.