Belite Bio, Inc

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, 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.

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 are 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 filed our commercial leadership positions and continue to build up 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 past quarter, we also announced that we have completed enrollment in our Phase II/III DRAGON II clinical trial, evaluating Tinlarebant to Stargardt disease. This trial enrolled 73 adolescents and adult subjects age 12 to 20 years from Japan, United States, 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 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 diseases and significant unmet medical needs. I will 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, we proceed from ESOP and warrant exercise. We ended Q1 with $799 million in cash, 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?

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.

And then 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 time lines?

Thanks, Judah. So that's a great question.

So we had several meetings with the FDA, including a meeting with the FDA to discuss the strong positive data at interim analysis. And it's the FDA's recommendation that we complete the DRAGON II study at 2 years with a possible path to one single-study approval based on the robustness of our data.

And obviously...

I think you mean DRAGON I or DRAGON II?

Yes, DRAGON I, So we don't believe that the DRAGON II data would be applicable to our FDA filings. But 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 serve as a confirmatory evidence.

But the DRAGON II is mostly for Japan regulatory requirements. I hope that answers your questions.

That's great. And 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?

Hao, do you want to probably better to answer this?

Yes. Well, we do expect that we're going to have 2 teams, one for the diagnostic promotion to bring more disease awareness and awareness to genetic testing to make sure that it is an easy kind of reach-out for the patient to be diagnosed and get that testing confirmed.

And we 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 disease patients confirmed with genetic testing. We're doing a lot of survey right now.

We do expect to give 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.

But 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. So we'll be focused on the retina specialist committee -- community, the patient advocacy group to better understand the needs, and also, of course, the general ophthalmologists and probably the low-vision optometrist community as well.

Geographic atrophy, can 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. So 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.

And if it's a strong-positive data, then it's a good problem to have. But at this time, we don't know what the data looks like.

So we haven't given that much thought in terms of strategy anyway. So I don't think I'll answer for you now, probably nearer the time when we have the interim, we probably have a better idea.

Right. Okay.

So you will have a sense of that, right? I mean...

Yes. So we're aiming for end of the year, but it all depends on the coordination and getting the data ready with the CROs.

It's a much, much more bigger data than the Stargardt disease. So logistic-wise, I think it's a bit more complicated, but we are aiming for the end of the year.

Yes. And then just back on Stargardt, what's the time line for Japan again?

How...

I think Japan, given that we have Sakigake Designation, the Pioneer designation, I think the approval -- the PMDA is aiming for approval within 3 months of the FDA approval. So we're looking on track for that as well.

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, how you're thinking about filing time line, launch strategy, partnering strategy, if relevant.

Would love your current thinking on the ex-U.S. opportunity.

Sure, sure. So again, right now, we are focusing on the FDA approval.

So within the submission time line, the 6 months will be period. We are expecting to have some questions from the FDA.

So we don't want to overstretch ourselves and file in different jurisdictions while we're focusing on the FDA. So 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.

So the time line will be based on what the responses from the FDA. So at this point, again, the time line, we'll need to update you on that.

So the FDA will serve as our priority.

Okay. Terrific.

And I just want to follow up on the GA analysis around year-end as well. Is this a type of situation where you would share data in any scenario, resize the study, stop the study either for efficacy or futility?

Like can you just talk about maybe just some of the possible scenarios for that analysis?

So, this is just assuming what I think the possible scenario is probably resizing the study. So the data will show us what the sample size is going to be after that interim.

So again, this will be a data-driven decision and strategy.

Steve, if I can make an additional comment. We definitely are trying everything that we can do to try to bring this treatment to all the patients around the world, both on GA and Stargardt.

But 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, but 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.

It's Graig. Congrats on the progress.

I have 2 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?

And then second, fully appreciating that you are ramping up your pre-commercial activities. Can you give a sense of what the level of awareness of Tinlarebant right now within the prescribing community?

And whether once you get to a place of launch, how much education will be needed?

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

Sure. Hendrik, do you want to go ahead first?

I can start, certainly. So I mean, the IRD and retina 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.

But we know that the rate of retina 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. We will be at the Retina Society Meeting in Los Angeles in September.

We will be at the American Academy Meeting in October, and we have presentations at all of those meetings. So this will be major opportunities to educate the community about this forthcoming treatment for Stargardt disease.

But yes, we are actively pursuing that.

That was the last question. So what was the second half of the question regarding the pricing and all that?

Yes. 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?

Got it. Hao, did you get that?

So yes, Graig, we have done several pricing projects so far. So far, the payers has been super supportive of the price range that we're thinking about.

And they definitely recognize the strong unmet need being the first treatment for Stargardt. So I think we appreciate the payers has been showing a lot of support on this.

And it's still too early to really set the price. But 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 will be the range that you'll consider compared with some of the analog out there. But we haven't really set the price.

It's still early, but we do see that this is a range that should be a fair assumption.

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? And 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?

Hao, you want to take this question?

Yes. Yes.

So thank you, Lin. Well, this is a small-molecule drug.

So, the manufacturing is not that complicated, and the packaging, delivering are all relatively easy compared with most of the other drugs. So we do expect that we should be fairly quickly be able to launch right upon the approval.

We are getting all the supply chain and the manufacturing ready right now. Yes, the -- 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 get the market a good throughout kind of a survey and numbers probably in September on the Commercial Day event.

So 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 Affairs task to make sure we warm up the community. But I think we cannot provide a specific guidance on today's call yet.

And 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?

Yes. Well, it's a fair scenario as you get ready for launch.

There's huge team expansion. And we -- last year, we were somewhere close to like 30 team members now.

We're now close to somewhere like 90, right? So we are expanding the team fast and also do all this activity that we talked about.

So we don't expect that expense will go up too much, but it's a fair assumption that it will go up while we go towards commercialization. And compared to last quarter, that's really not a fair assumption because that was when we just started some of the preparation work.

But like I said in the presentation, we're sitting on close to $800 million cash. So we're in a very, very comfortable cash position.

To launch Stargardt disease in the U.S., we probably look forward probably USD 300 million. And our existing pipeline, as we talked about before, we expect the budget will be about $150 million for next 3 years.

So in total, we're talking about $450 million at most of the budget, while we're sitting on $800 million. So, 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.