벨라이트 바이오(BLTE) 2026년 2분기 실적 발표 콘퍼런스 콜: 틴라레반트 FDA 심사 및 7억 8,000만 달러 현금

핵심 요약

• 미국 식품의약국(FDA)이 스타가르트병 치료제 틴라레반트(Tinlarebant)에 대한 벨라이트 바이오(Belite Bio)의 신약허가신청(NDA)을 우선심사 대상으로 승인했으며, 처방약 유저피법(PDUFA) 목표일을 2027년 2월 12일로 지정했다.
• 임상 3상 DRAGON 연구 데이터에 따르면, 틴라레반트 투여 환자의 정량적 자가형광(qAF) 수치는 25개월 차에 기저치 대비 약 2% 감소한 반면, 위약 대조군은 약 20% 증가한 것으로 나타났다.
• 2026년 2분기 R&D 비용은 임상 3상 완료에 따른 로열티 지급의 영향으로 전년 동기의 1,100만 달러에서 1,820만 달러로 증가했다.
• GAAP 기준 순손실은 2025년 2분기 1,630만 달러에서 2,840만 달러로 확대됐다. 비GAAP(Non-GAAP) 기준 순손실은 870만 달러에서 2,160만 달러로 늘어났다.
• 벨라이트 바이오는 현금 및 현금성 자산, 미국 국채 7억 8,000만 달러를 보유하며 이번 분기를 마감했다. 경영진은 이 자금이 향후 승인 시 틴라레반트 상업화 및 파이프라인 진전을 위한 재원이 될 것이라고 밝혔다.
• 회사 측은 미국 내 심사를 최우선으로 진행하고 있다. 경영진은 FDA 승인 가능성 이후 유럽 내 허가를 신청할 계획이며, 일본 의약품의료기기종합기구(PMDA)의 심사 절차는 병행하여 진행 중이라고 설명했다.

주요 재무 데이터

사업 및 영업 실적

틴라레반트는 벨라이트 바이오의 2026년 2분기 실적 발표의 핵심 관심사였다. FDA는 스타가르트병 치료제 NDA를 우선심사 대상으로 받아들였으며 PDUFA 목표일을 2027년 2월 12일로 지정했다.

벨라이트 바이오는 4개국 4개 의학 학술대회에서 임상 3상 DRAGON 결과를 발표했다. 미국망막학회(ASRS) 연례 학술대회에서 회사 측은 틴라레반트 투여 환자의 qAF 수치가 25개월 차에 기저치 대비 약 2% 감소하며 미미한 감소세에 그쳤다고 발표했다. 같은 기간 위약 대조군 환자는 약 20% 증가를 기록했다.

경영진은 qAF를 스타가르트병의 망막 변성을 유발하는 독성 비스레티노이드 축적의 지표로 설명했다. 회사 측은 이번 결과가 틴라레반트의 작용 기전 및 병변 진행을 지연시킬 가능성과 일치한다고 밝혔다.

경영진에 따르면 스타가르트병 프로그램의 복약 순응도는 24개월 후에도 90% 이상을 유지했다.

DRAGON II 임상은 여전히 PMDA 승인을 위한 일본 중심 연구로 남아 있다. 경영진은 현재 해당 임상이 미국 NDA 심사에 기여할 것으로 보지 않고 있다. 한편 벨라이트 바이오는 12세 미만 환자에 대한 규제 절차에 대비하기 위해 런던에서 소아 임상 연구를 시작하고 있다.

경영진 가이던스

경영진은 향후 6개월간 미국 승인 획득이 최우선 과제라고 밝혔다. 회사는 각 국가별 규제 전략 및 소통을 조율할 수 있도록 FDA 승인 가능성 이후 유럽 내 허가 신청을 추진할 것으로 예상하고 있다.

일본의 규제 절차는 미국의 심사와 병행하여 진행 중이다. 사키가케 지정을 바탕으로, 경영진은 FDA 승인이 이뤄질 경우 약 3개월 후 일본 내 승인이 이뤄질 수 있다고 밝혔다.

벨라이트 바이오는 2026년 12월과 2027년 1월이 FDA와의 상호작용이 가장 활발한 시기가 될 것으로 예상됨에 따라, 지도상 위축증 연구의 중간 분석 시점을 2027년 1분기(2월 이후 가능성 높음)로 예상하고 있다.

틴라레반트가 승인될 경우, 경영진은 희귀 소아질환 지정을 바탕으로 우선심사 바우처를 받을 것으로 기대하고 있다. 벨라이트 바이오는 해당 바우처를 매각할지 직접 사용할지 여부를 아직 결정하지 않았다.

리스크 및 주시할 점

• FDA 승인은 2027년 2월 12일 PDUFA 목표일에 앞서 진행 중인 심사 절차에 따라 달라질 수 있다.
• 경영진은 현재 자문위원회 회의가 계획되어 있지 않으나, FDA가 심사 후반에 회의를 요청할 가능성은 여전히 존재한다고 언급했다.
• 적응증 및 처방 정보(라벨) 논의는 아직 이뤄지지 않았다. 경영진은 확보된 데이터를 바탕으로 보다 광범위한 라벨을 기대하고 있으나, NDA가 심사 중인 상황에서 최종 라벨의 범위에 대한 언급은 말을 아꼈다.
• 유럽 허가 신청, 일본 승인 및 지도상 위축증 중간 분석 시점은 FDA 심사 절차 및 미국 승인 여부에 달려 있다.

애널리스트 Q&A 하이라이트

애널리스트들은 DRAGON II의 역할, 소아 임상 개발, 규제 승인 순서, 위탁생산, 잠재적 라벨 범위 및 자금 계획에 집중했다. 경영진은 DRAGON II가 주로 일본을 타깃으로 하며 미국 NDA 절차를 지원할 것으로 예상되지 않는다는 입장을 재확인했다.

라벨 범위와 관련하여 회사 측은 FDA와 아직 라벨에 관한 논의를 진행하지 않았다고 밝혔다. 헨드릭 숄(Hendrik Scholl) 최고의료책임자(CMO)는 연령대별 병변 진행 속도가 전반적으로 유사하고 근본적인 ABCA4 기능 장애가 동일하다는 점을 지적하면서도, 심사 진행 중 최종 라벨을 예측하지는 않겠다고 강조했다.

벨라이트 바이오는 미국 내외의 위탁개발생산(CDMO) 업체들과 협력하고 있음을 확인했다. 회사는 실적 발표 통화 중 생산 시설에 대한 구체적인 세부 사항은 밝히지 않았다.

경영진은 또한 9월에 버추얼 커머셜 데이(Commercial Day)를 개최하여 미국 시장 조사에서 얻은 환자 수 데이터를 공유할 계획이라고 덧붙였다.

실적 발표 녹취록 전문

전체 실적 발표 컨퍼런스 콜 녹취록

경영진 발표

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.

질의응답

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.