BLTE - NASDAQ

Ladies and gentlemen, thank you for joining us, and welcome to the Belite Bio first quarter 2026 earnings call. After today's prepared remarks, we will host a Question-and-Answer session. If you would like to ask a question, please raise your hand. If you have dialed into today's call, please press star nine to raise your hand and star six to unmute. I will now hand the conference over to Julie Fallon. Please go ahead.

Good afternoon, everyone. 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. 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. Now I'll turn the call over to Dr. Lin. Dr. Lin?

Thank you, Julie. Good afternoon. Thank you for joining our first quarter 2026 financial results and updates. We have made exciting progress so far this year. We have received our phase III clinical study report in Q1, and without delay, in April, we initiated our NDA rolling submission to the FDA for Stargardt disease. We're on track to complete the submission by the second quarter of this year. As we approach the completion of the rolling submission, we're also preparing for our commercial launch. We have hired all commercial leadership positions and continue to build out our teams in sales, market access, and medical affairs. We're also building out our commercial infrastructure, as well as engaging with the retinal community to raise awareness of Stargardt disease. We are focused on preparing for a strong launch and looking forward to sharing more on our commercial planning in the future.

In line with that commitment to bring Tinlarebant to patients around the world, this last quarter, we also announced that we have completed enrollment in our phase II/III DRAGON II clinical trial evaluating Tinlarebant in Stargardt disease. This trial enrolled 73 adolescents and adult subjects aged 12 to 20 years from Japan, U.S., and U.K. This is a registration-enabling study to pursue approval in Japan. This is shaping up to be a pivotal year for Belite Bio as we begin our transition to a commercial stage company. We look forward to providing further updates on our work bringing therapies for retinal degenerative disease and certain unmet medical needs. I'll now turn over the presentation to Hao-Yuan to discuss the financials. Hao-Yuan?

Thank you, Tom. In Q1 2026, our R&D expenses were $15.7 million compared to $9.4 million in Q1 2025. The increase was mainly driven by higher spending on the DRAGON II trial, increased API and drug product manufacturing expenses, and higher consultant and professional service fee. On a non-GAAP basis, excluding share-based compensation expenses, R&D expenses in Q1 2026 were $13.8 million compared to $7.4 million in Q1 2025. SG&A expenses in Q1 2026 were $17 million compared to $6.1 million in Q1 2025. The increase in SG&A expenses were primarily due to increase in share-based compensation expenses, professional service fees, and wages and salaries resulting from our team expansion. On a non-GAAP basis, excluding share-based compensation expenses, the SG&A expenses in Q1 2026 were $5.7 million compared to $1.5 million in Q1 2025.

GAAP net loss for the quarter was $26.9 million compared to $14.3 million in the same period last year. On a non-GAAP basis, excluding share-based compensation expenses, net loss was $13.7 million in Q1 2026 compared to $7.6 million in Q1 2025. Despite the increased investment in R&D and SG&A, our balance sheet remains very strong. Specifically, with proceeds from ESOP and warrant exercise, we ended Q1 with $799 million in cash equivalent, and U.S. Treasury bills, a higher balance than at the end of 2025. This strong cash position gives us ample capital to execute on our goals, including finalizing our NDA application, preparing for the commercialization in Stargardt disease, and completing our ongoing clinical trials. With that, I'll now turn the call back to the operator for Q&A. Operator?

We will now begin the Question-and-Answer session. If you would like to ask a question, please raise your hand now. If you have dialed in to today's call please press star and nine to raise your hand, star six to unmute. Please stand by as we compile the Q&A roster. Your first question comes from the line of Judah Frommer with Morgan Stanley. Your line is open. Please go ahead.

Yeah. Hi, guys. Thanks for taking the question, and congrats on all the progress here. On DRAGON II, what confidence do you have based on communication with FDA that readout will not be necessary for an approval decision in the U.S.? I guess on the flip side of that, if FDA does imply that they would like to see DRAGON II results, what are the chances that that is confirmatory, and how could that play into timelines? Thank you.

Thanks, Judah. That's a great question. We had several meetings with the FDA, including a meeting with the FDA to discuss the strong positive data at interim analysis. It's the FDA's recommendation that we complete the DRAGON II study at two years with a possible path to one single study approval based on the robustness of our data.

Sorry, Tom, I think you mean DRAGON I, not DRAGON II.

Yes, DRAGON II. Sorry about that. We don't believe that the DRAGON II data would be applicable to our FDA filings. Even if there's a slight chance of that happening, we could always have the DRAGON II data available, at least the interim part of that to still serve as a confirmatory evidence. The DRAGON II is mostly for Japan regulatory requirements.

Okay.

I hope I answered your questions.

Yeah. No, that's great. Then just maybe touching on building out the commercial infrastructure, what are your latest thoughts on how targeted the commercial team or the field sales force team could be here, just given how concentrated the patient population is and where they're seen by centers of excellence? Thank you.

Hao, you are probably better to answer this.

Well, we do expect that we're going to have two teams. One, for the diagnostic promotion, to bring more disease awareness and awareness to genetic testing, to make sure that it's an easy reach out for the patient to be diagnosed and get that testing confirmed. Also have another team more focused on promoting the drug. In total, we're thinking about 30 to maybe 40 total team members for that regard. We do know that there are many retina specialists that already have a database of Stargardt patient confirmed with genetic testing. We're doing a lot of survey right now. We do expect to get the market an update about what we know, what we're going to be doing, hopefully in September, so you have a better idea about the whole plan and what is already confirmed out there.

Long story short, we do see that there are many patients are very incentivized to this treatment and continue to be follow up with their physicians. We'll be focused on retina specialist communities, the patient advocacy group, to better understand their needs and also, of course, the general ophthalmologist and probably the low vision optometrist community as well.

Great. Thank you.

Your next question comes from the line of Marc Goodman with Leerink. Your line is open. Please go ahead. A reminder that you may need to unmute locally.

Hey, guys. How are you? geographic atrophy, could you just talk about how you're thinking about this right now and timing of the interim and what happens if the GA indication ends up looking really, really strong?

Thanks, Marc. For GA, right now we are focused on getting the FDA approval for Stargardt disease. We are aiming for the interim for GA around end of the year. Right now, we don't know what's the data going to be like. If it's a strong positive data, then it's a good problem to have. At this time, we don't know what the data looks like, so we haven't given it much thought in terms of strategy anyway. I don't think I have an answer for you now. Probably near the time when we have the interim, we probably have a better idea.

Right. Okay. You will have a sense of that, right? I mean.

Yeah. We're aiming for end of the year, but it all depends on the coordination and getting the data ready with the CROs and all that. It's a much, much more bigger data than the Stargardt disease. Logistic-wise, I think it's a bit more complicated, but we aim for end of the year.

Yeah. Then just back on Stargardt, what's the timeline for Japan again? How is that looking?

I think Japan, given that we have Sakigake designation, the pioneer designation, I think the PMDA is aiming for approval within three months of the FDA approval.

Got it. Thank you.

We're looking on track for that as well.

Thank you.

Your next question comes from the line of Steve Seedhouse with Cantor. Your line is open. Please go ahead.

Great. Thanks, thanks for the color on the commercial preparations in the U.S. I actually just wanted to ask about on the other side, ex-U.S., particularly in Europe, sort of how you're thinking about filing timeline, Launch strategy, partnering strategy, if relevant. Would love your current thinking on the ex-U.S. opportunity.

Sure. Again, right now we are focusing on the FDA approval. Within the submission timeline, the six-month review period, we are expecting to have some questions from the FDA. We don't want to overstretch ourselves and file in different jurisdictions while we're focusing on the FDA. Our filing strategy is that the FDA forms the basis of our submission and rest of the world will be consistent with the FDA filing. The timeline will be based on what the responses from the FDA. At this point, again, the timeline, we'll need to update you on that. The FDA will serve as our priority.

Okay, terrific. I just want to follow up on the GA analysis around year-end as well. Is this the type of situation where you would share data in any scenario, resize the study, stop the study either for efficacy or futility? Can you just talk about maybe just some of the possible scenarios or that analysis?

This is just assuming what I think the possible scenario is probably resizing the study. The data will show us what the sample size is going to be after that interim. Again, this will be a data-driven decision and strategy.

Okay, thank you very much.

Steve, if I can make an additional comment. We're definitely trying everything that we can do to try to bring this treatment to all the patients around the world, both on GA and Stargardt disease. Like Tom said, some of these will be data-driven, and we did recognize that Stargardt disease in the U.S. will be our first focus. We're continuing to monitor all the other development and definitely try to bring the treatment to all the patients as soon as we can.

Thank you.

Your next question comes from the line of Graig Suvannavejh with Mizuho.

Hey, it's Graig. Thanks so much for taking my questions. Congrats on the progress. I had two questions, if I could. One, it's been some time now since you've had the data in-hand. Have you done any additional testing, market research-wise with payers in terms of potential pricing bands that would be acceptable? What are your latest thoughts on potential pricing? Second, fully appreciating that you are ramping up your pre-commercial activities, can you give a sense of what the level of awareness is of Tinlarebant right now with the prescribing community and whether, once you get to a place of launch, how much education will be needed? Thanks.

I'll ask Hendrik to discuss on the data part that you mentioned. Hao, maybe you want to comment on the commercial side of this question?

Sure. Hendrik, you want to go ahead first?

The IRD and retinal specialist community is a very well-defined community that meets regularly at ARVO, ASRS, and the American Academy meeting. People know about Belite Bio and Tinlarebant. We can certainly improve on that because our interviews with retinal specialists have shown that they are enthusiastic about the prospect of a first treatment ever for this so far untreatable disease. Plus, the convenience of this being an oral treatment. We know that the rate of retinal specialists that have in-depth knowledge about Tinlarebant and the DRAGON trial needs to be improved. We clearly know that. We will be present at the American Society of Retina Specialists meeting in Montreal in July, and we will be at the Retina Society meeting in Los Angeles in September.

We will be at the American Academy of Ophthalmology meeting in October, and we have presentations at all of those meetings. This will be major opportunities to educate the community about this forthcoming treatment for Stargardt disease. Yes, we are actively pursuing that.

Sorry, that was a long question. What was the second half of the question regarding the pricing and all that?

Yeah, I just wanted to get a sense of whether you, now that you've had the phase III data in hand, whether you've been able to do any additional payer market research in terms of how you're thinking about pricing.

Oh, got it. Hao, did you get that?

Yeah, I did. Yeah, Graig, so we have done several pricing projects so far. Far the payers have been super supportive of the price range that we're thinking about, and they definitely recognize the strong unmet need being a first treatment for Stargardt. I think we appreciate the payers have been showing a lot of support on this. It's still too early to really set the price. I think we talk about if people want to know maybe a reference price, we think that the average orphan drug price in the U.S. around $350,000, that's a fair reference price. Maybe up to $500,000. That would be the range that you'll consider to compare with some of the analogs out there. We haven't really set the price. It's still early.

We do see that this is a range that should be a fair assumptions. Okay, thank you.

Just a reminder that if you would like to ask a question, you can use the raise hand function, or if you have dialed into today's call, you can press star nine to raise your hand, star six to unmute. Your next question comes from the line of Yi Chen with H.C. Wainwright. Your line is open. Please go ahead.

Thank you for taking my questions. Assuming that you get FDA approval in early 2027, can you tell us how quickly you can launch the drug, whether your manufacturing facility is in alignment with that timing? More importantly, can you provide us with a rough estimate as to how many patients could you reasonably expect to receive the Tinlarebant treatment in 2027? Thank you.

Hao, you want to carry all of this question as well?

Yes. Thank you, Yi. Well, this is a small market drug, the manufacturing is not that complicated. Packaging, delivering, all relatively easy compared with most other drugs. We do expect that we should be fairly quickly be able to launch right upon approval. We are getting all the supply chain and the manufacturing ready right now. In terms of the number of patients at the first year, I think, like I said earlier, we would like to do more survey and maybe give the market a good throughout survey and numbers probably in September on the commercial day event. We're doing everything we can to try to find all this potential database and doing all the surveys and all the so-called medical affair tasks to make sure we warm up the community.

I think we cannot provide a specific guidance on today's call yet.

Thanks. A quick question on the operating expenses. I noticed the first quarter numbers are meaningfully higher compared to fourth quarter last year. Shall we expect that the operating expenses to continuously increase as you approach the FDA decision?

Well, it's a fair scenario as you get ready for launch. There is huge team expansion. Last year we were somewhere close to 30 team members now. We're now close somewhere like 90, right? We are expanding the team fast and also doing all these activity that we talk about. We don't expect that expense will go up too much. It's a fair assumption that it will go up while we go towards commercialization. Compared to last quarter, that's really not a fair assumption because that was when we just started some of the preparation work. Like I said, in the presentation, we're sitting on close to $800 million cash. We're in a very, very comfortable cash position. To launch Stargardt in the U.S., you probably look forward probably $300 million.

Our existing pipeline, as we talked about before, we expect the budget will be about $150 million for next three years. In total, we're talking about $450 million at most of the budget, while we're sitting on $800 million. We think we are very comfortable on cash, and this is going to be a good investment to be made to make sure that we get all the awareness out there and try to help the patient as fast, as broad as we can.

Got it. Thank you.

Thank you.