BLTE - NASDAQ

Ladies and gentlemen, thank you for joining us, and welcome to the Belite Bio Fourth Quarter and Fiscal Year-End 2025 Earnings Call. [Operator Instructions] I will now hand the conference over to Sophie Hunt. 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, Belite Bio's 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 issued earlier today. And now I'll turn the call over to Hao. Hao?

Thank you for joining today's call to discuss our fourth quarter and full year 2025 financial results. 2025 was a year of significant progress for us as we achieved several key milestones. We look forward to a truly transformative year in 2026 as we position Tinlarebant to potentially become the first ever approved therapy for people living with Stargardt disease, the devastating eye disease that usually begins in childhood or young adulthood and leads to progressive vision loss and then legal blindness in almost all cases. Today, I'll provide a recap of our 2025 achievement, key milestone for 2026 and financial results. Starting with 2025 achievement, of course, the most significant achievement was the announcement of our top line results for the Phase III pivotal DRAGON trial in December. We're very excited to share that the trial met its primary efficacy endpoint, demonstrating statistically significance and clinically meaningful 36% reduction in the growth rate of upper lesion, measured by definitely decreased autofluorescence by fundus autofluorescence imaging compared with placebo. These results position us well for engagement with the regulatory authorities as we see a path to commercialization in Stargardt disease. In the DRAGON II study, we reached the target number of 60 subjects in January. As of February 27, we had enrolled 72 subjects as subjects who had passed the screening before, the registration closed, can still be admitted to the trial. We expect the final number of subjects enrolled to be between 72 and 75. We also completed enrollment in the Phase III PHOENIX trial in GA with 530 subjects. Finally, we completed a $402 million public offering with over allotment fully exercised by the underwriter in Q4. Importantly, the net proceeds went from this along with other raises comparing the year, restricting us extremely well to support commercialization preparation for Stargardt disease, development and expansion of pipelines and general corporate purposes. Now moving to 2026. As I said, this will be a transformative year for Belite. The top priority in our planned NDA submission to the FDA in the second quarter of 2026. And with our NDA submission planned, we have also kicked off our commercialization preparation work for Stargardt disease. I'm pleased to share that we have hired all of the key leadership positions and are now in the process of building our organization in sales, market access, medical affairs, marketing, regulatory and operations, et cetera. It's a busy but exciting time for us, and we look forward to sharing more as we progress with our launch preparation works. Last but not least, I'll now close with the financial recap. For the fourth quarter, R&D expenses were $14.6 million compared to $7.3 million in Q4 2024. The increase was primarily due to the first expenses related to the DRAGON II trial. Second, we received a lower Australian R&D tax incentive in Q4, 2025 as such incentive was received in Q3 2025 versus last year it was received in Q4 2024. And third, API manufacturing expenses. On a non-GAAP basis, which excludes share-based compensation expenses, R&D expenses for the fourth quarter was $12.2 million compared to $5.7 million for the same period in 2024. We believe this non-GAAP basis provides a better picture of our operating expenses since our share-based compensation expenses is heavily driven by achieving the volume milestone and the volatility of our own stock price and a comparable company stock price using the valuation. SG&A expenses were $13.5 million compared to $4.2 million in Q4 2024. The increase was primarily due to increase in share-based compensation expenses and professional service fees. As we achieved development milestones and started to prepare for filing and commercialization. On a non-GAAP basis, SG&A expenses for the fourth quarter was $4.2 million compared to $1.5 million in Q4 2024. Overall, the fourth quarter, we reported a net loss of $25.3 million compared to $10.1 million in Q4 2024. On a non-GAAP basis, we reported a net loss of $13.6 million for the fourth quarter compared to $5.9 million for Q4 2024. For the full year, R&D expenses were $45.4 million compared to $29.9 million for the full year 2024. The full year increase was primarily due to; first, expenses related to PHOENIX trial; second, share-based compensation expenses; and third, API manufacturing expenses, partially offset by the royalty payment recognized in 2024. On a non-GAAP basis, excluding share-based compensation expenses, the R&D expenses were -- for the full year was $36.2 million compared to $26.2 million for the same period in 2024. SG&A expenses were $38.9 million compared to $10.1 million in 2024. The increase was primarily due to increase in share-based compensation expenses and professional service fee. As we achieved development milestone and started to prepare for filing and commercialization. On a non-GAAP basis, SG&A expenses were -- for the full year were $9.1 million compared to $4.8 million in 2024. For the full year, we reported a net loss of $77.6 million compared to a net loss of $36.1 million in 2024. On a non-GAAP basis, net loss was $38.7 million compared to a non-GAAP net loss of $27.2 million in 2024. Moving to the balance sheet. As I said, we had a successful year of fundraising through underwritten public offering to registered direct offering and a significant pipe. We're very grateful to our shareholders for their strong support. As a result, we closed the year with $772.6 million in cash, cash equivalent, U.S. treasury bills and notes as compared with $145.2 million at the end of 2024. Our balance sheet remains strong, and we are well positioned to deliver our near and long-term objectives, including the commercial launch for Stargardt disease. With that, I'll turn the call back to the operator for Q&A.

[Operator Instructions] Your first question comes from the line of Judah Frommer with Morgan Stanley.

Just a couple of questions for us. I guess on the NDA submission, are you still thinking about that being a rolling submission? And what role would DRAGON II play within that submission process, I would -- maybe in the U.S. and other geographies as well. And then I guess just given the cash balance that you've amassed here, can you help us with the uses of cash between getting through the remaining Stargardt trials, getting through GA and commercialization and anything else we should be thinking about?

Okay. I'll answer the first question regarding the NDA. So it will be a rolling submission. We are on track for the NDA submission in Q2. We're expecting the CSR to finalize this month. And once that's finalized, we are ready to submit pretty soon. What's the next? DRAGON II. Yes, so the DRAGON II will be for Japan only because the Japanese authorities would like to see the data of Japanese patients, so that's strictly for the Japan. And the commercialization and the budget, I think it was the other question, I'll refer that to Hao. Hao?

Yes, so for the next three years, we expect existing pipeline, including the NDA submission, all of those, what we call that like R&D kind of related activity will cost us about $150 million. And for the commercialization itself for the next 3 years is probably somewhere between $200 million to $250 million.

Your next question comes from the line of Tazeen Ahmad with Bank of America.

Can you just give us a little bit of guidance on how we should be thinking about pricing given the profile of the drug and given the undermet need, we'd be curious to maybe get a sense of a range of what would be appropriate to be considering here? And then can you just remind us what are the key gating items left before you submit the NDA in the second quarter?

Hao, do you want to take this one as well?

Sure. Well, for the pricing, apparently, it's still early for us to set a price. But I think we have been seeing that the average rare disease drug price in the U.S. being somewhere about $350,000. And we do think it's fair to say that we expect ourselves can be doing better than that, but still early to really set a price.

Okay. And then on...

Yes, what was the other question?

Yes, what are the gating factors left before you submit for approval in 2Q?

I guess we have everything ready. So we're just waiting for the clinical study report. So as we speak, we are on track.

Your next question comes from the line of Marc Goodman with Leerink. Your next question comes from the line of [ Timur Ivannikov ] with Cantor.

This is [ Timur Ivannikov ] for Steve Seedhouse. So our question is about the timing of your potential launch. So assuming you have an NDA filing in the second quarter, do you have initial expectations on the launch timing? And then I think you were talking about maybe 25 field reps. But how quickly after the approval do you think you can launch? And how do you assess the difficulty of this launch maybe to other rare diseases or other retinal disease?

Hao, do you want to take this one as well?

Sure, sure. Well, so we expect we probably will launch by Q1 2027. The sales team, as you said, we expect that we have probably a team more focused on genetic testing, which will be one of the key factors to get the patient confirmed. The second team will be more about the drug -- about the brand. So total somewhere like 25 to 30, we think is a fair assumption at launch. Potentially, after 2 years of launch, you may expand that team further as you want to get to every corner in the U.S. Yes. So I think being able to launch by Q1 2027 is our goal. And to your question about the challenges, we think compared to other disease, given there's no treatment for Stargardt disease, this should be a fairly straightforward drug. The difficulty will really be getting patients, getting the physicians to be aware this treatment is available and then shorten the time it takes for people to get the genetic testing done and get their insurance coverage. I think that -- these will be the few execution kind of test that we will be focused on. But I wouldn't see those are like challenges for us.

Hao, maybe we could get Hendrik to also add more color to this question, given that he is prescribing himself. He looks after these Stargardt patients, and he knows the whole clinical landscape very well. So Hendrik, do you want to add anything? Any details?

Yes. Thank you, Tom, but I would like to confirm what Hao-Yuan just said and pointed out, it's a fact that many patients are lined up in large databases. Many of Stargardt patients because it includes genetic testing to make the diagnosis are being seen in large centers, including large academic centers and such centers typically have database of patients where they also include the genotype of these patients. So these patients, therefore, are immediately available because they are known to the centers and patients can be contacted by treating physicians if the patient him or herself would not seek clinical care immediately. So I believe because this is a monogenic disease, there's an extra opportunity to get to patients very quickly.

Your next question comes from the line of Marc Goodman with Leerink.

Yes. Sorry about the confusion. Can you talk about your filing plans OUS? And then secondly, what are your latest thoughts on the timing of an interim look for the GA work you're doing?

Thanks, Marc. So you're saying that the timing of ex-U.S. NDA submissions or the U.S.?

Yes, yes, OUS. Exactly, ex-U.S.

Okay. So the -- we want to set the priority of the FDA on U.S. We want to put all resources to make sure that we are successful with the NDA in the U.S. So everything outside of the U.S. will build on to that. And this requires discussions with the regulatory authorities in different regions to see what type of timing that we're expecting, or they're expecting. So this will be an update which regions they will prioritize after the U.S. So we are in constant communications with the EMA, the PMDA and other authorities as well. So we want to keep the U.S. -- keep all the bandwidth on the U.S. FDA given that we expect there's going to be a lot of questions. So we don't want to dilute our resources at this point by spreading it to -- spread out and then submission -- submitting it on too many regions. Does that answers your question?

Correct.

What was the other one?

The interim look for the geographic atrophy. Just curious what your latest thoughts are?

Yes. So right now, we are probably expecting that would be somewhere second half of the year. We haven't actually looked at it yet because we are prioritizing everything on launching Tinlarebant for Stargardt. So we will have a further update for that, probably in the next quarter.

Your next question comes from the line of Yi Chen with H.C. Wainwright.

This is [ Eduardo ] on for Yi. Just following up on the geographic atrophy trial. Do you have any idea of what level of lesion growth inhibition you're targeting to consider that trial as success in that broad population. And then also if you had any comments on capital allocation for the LBS-009, and how you prioritize that, and when you expect to maybe move into a Phase I study and if you have any details on the specific liver indication as a primary lead.

So I'll get Hendrik to answer on the GA one. I'll start with the 009. Right now, there's no plans for 009 yet. So again, we're prioritizing everything on Tinlarebant and be a successful launch in the U.S. first. All the others will fall and will prioritize after that. Hendrik?

And I'm happy. Thank you, Tom. I'm very happy to take the question on what's the threshold that would make treatment of GA success with our oral compound. When you think about OAKS, DERBY and GALE, [ the 2 ] injectables Syfovre and Izervay they found efficacy signals of 13%, 21% and 14% in their registration trials. Given that these are injectables that need to be injected essentially monthly for the rest of the life of patients affected by GA. We feel that if we reach that threshold, then it is already a success. Having said that, I mean, we are more ambitious given what we found in Stargardt disease, 36%, we feel that reaching 13%, 21%, 14% so roughly about -- something between 15% and 20% could absolutely be possible, and we would like to go beyond that. But again, since our compound is an oral compound, if we reach the same threshold, we will be the standard of care because it will be a very hard sell for patients to tell them to come in for injections every month if there is an oral treatment available.

Your next question comes from the line of Boris Peaker with Titan.

Congrats on the progress. Just maybe we'll start with Stargardt. Do you anticipate the label to become a broad Stargardt label for all patients? Or would it -- you think potentially be restricted to patients ages maybe 12 to 20, similar to the pivotal study.

I'll refer this to Nathan and of course, Hendrik to add more details as well. Nathan?

Nathan, here, the CSO. So we've had that discussion with FDA, and we've made the argument that basically it's the same disease, whether it's affecting children or adults, and they concurred. There's no evidence to suggest that these patient populations would be any different. Of course, Hendrik knows from the ProgStar data that the lesion growth profiles are not dramatically different between children and adults. So yes, we'll be pressing for the full label from -- for subjects 12 and older because, again, it's the same disease, same genetic sort of dysfunction that leads to the dysfunction of the same protein. So again, spectrum of the same disease across different populations.

Got it. And other just to follow up on -- go ahead. Sorry.

No, I just wanted to add that it's all about the generalizability of the data, right? And there has really been such an easy case to convince the regulator, this is the same disease. And we included adult subjects 18 to 20 years, but we also included adolescents, as you know, right? But if there is a patient affected at aged 22, 28, 32 with biallelic mutations in ABCA4, why would that be considered a different disease? Why would somebody believe there would be no efficacy if you treat later because, and Nathan pointed it out, the ProgStar study has shown that progression rates amongst different age groups, 12 to 18, 18 to 50 and beyond 50 were essentially similar.

Got it. And just another follow-up on Stargardt. I understand your initial emphasis is obviously going to be on the U.S. market. But I'm just curious for the ex U.S. opportunity, how important is visual acuity, I guess, for approval and potentially for just reimbursement and justifying pricing?

Hendrik, do you want to take this as well?

Certainly. I mean, to be clear, visual acuity is important for every regulator, right? It's just how realistic is it that any given trial in Stargardt disease would find a visual acuity efficacy signal, right? When you look at the ProgStar data and an average visual acuity loss of 0.55 letters per year, but life expectancy of 60 to 80 years after the first diagnosis. That means that it's simply impossible even if you have a treatment that arrests the progression to find an efficacy signal then visual acuity is the primary outcome measure. If you arrest progression and the progression is 1.1 letters in 2 years, that would be the difference that you would target, but everybody knows that there's a 15 letter threshold set by the FDA to be clinically meaningful. And the intersession variability of visual acuity measurements in a population of macular degeneration patients such as Stargardt is 8 letters. So meaning that visual acuity as an outcome measure is an unrealistic target. But DDAF, which is our primary endpoint has been shown in cross-sectional correlations in the ProgStar study to be highly significantly correlated with visual acuity loss. It just means that you have to treat for a while until eventually you will see a visual acuity benefit.

[Operator Instructions] Your next question comes from the line of Bruce Jackson with Benchmark.

So in terms of the commercialization strategy in the United States, you've chosen to go direct, have you given any thought to what your international commercialization strategy might look like?

Yes, of course. So right now, we are open. We're very flexible on that. We do have multinational pharmaceutical companies wanting to partner or license. Right now, that's still open. We believe right now, we -- at least our regulatory submission pathway is pretty straightforward for all regulatory authorities. So we believe we can add more value, at least starting from the FDA, once we get the approval, we'll see how it goes in other regions. But we believe that we have a very straightforward approval path for all other regions as well. So it depends on what kind of reasonable deals or deals that we think was a good partnership after the FDA -- after we get FDA approval.

Okay. Great. And then if I could just get a follow-up on the ex-U.S. regulatory strategy. You've got quite a bit going on this year. Do you intend to seek further approvals in Europe? And when might those get submitted? And that's...

So the FDA being on top of our priority. And then second, I would say the EMA and probably next to it will be Japan as well. And then followed by China and all other regions.

Your final question will be from the line of Michael Okunewitch with Maxim.

Congrats on all the great progress. I guess, I'd like to see if you could help me understand just how well understood the true prevalence of Stargardt diseases given there have been no approved therapies. Do you expect that having something available could help build awareness and uncover additional undiagnosed patients?

Hendrik, can we throw this question to you?

I'm happy to answer the question. So the answer is absolutely, absolutely. If there is a treatment, and we have seen that about a decade ago for patients affected by biallelic mutations in RPE65 to be treated with Luxturna, the first gene therapy for that condition, absolutely led to a whole wave of patients that have been undiagnosed before to be diagnosed. And that includes a proper diagnosis clinically and genetic testing. In Stargardt disease, the symptoms are more straightforward than in RPE65. It's a much more diffuse disease affecting night vision in the periphery. In Stargardt disease, central vision is affected. Patients seek clinical care, but we will need a genetic diagnosis in order to treat patients. What is the true prevalence of Stargardt disease? In the past, for rare diseases, it was very difficult to find out what the actual prevalence is. It's only known in the Beaver Dam eye study, Blue Mountain eye study, Rotterdam eye study, what the prevalent eye diseases are. But there's new opportunity since about a decade or so to study genetic databases, knowing about the mutations in the target gene and the penetration rate. And this allows us to estimate and taking into account the race mix in the United States that we need to consider about 53,000 patients being affected by ABCA4-mutated retinal disease, including Stargardt disease. So I think that it's a realistic number now, which is firmly based on genetic databases that are available for populations of European descent, East Asian descent and African descent.

Nathan, I believe you've published on this a few times. Anything you want to add?

No, no. I think Hendrik covered it very nicely. Yes, we did publish a review article recently, capping the prevalence of Stargardt disease, looking at it geographically across the world. And you can really look for that paper. It's published under my name and Hendrik's name just recently. But yes, so 53,000 in the United States and ex U.S., of course, more than that globally. So -- and again, the genetics really tells us what the prevalence are. That's what the data are based upon in terms of the publication that we recently submitted -- recently got accepted.

And then just one more as a follow-up, if you don't mind. I wanted to see, do you expect that there would be any value in looking into patients younger than 12 years old? And are there any plans for this expansion?

Yes. Let me just take that real quick. So we do have an approved pediatric investigational plan with EMA, which we plan to initiate in April of this year. So that's coming up very soon. That is a 2-year study looking at safety and efficacy in children 3 to 11 years of age. So we'll have to wait to see what the safety and efficacy data look like at the end of the 2-year study. But certainly, we do have plans to establish safety and efficacy in patients younger than 12.

And Hendrik, I believe that you answered the same question as well in one of the medical conferences just a month ago.

Yes, indeed. And we feel that although in DRAGON patients already had significantly lost vision on average, we feel that patients before losing significant vision will strongly benefit from Tinlarebant treatment. And that would typically be relatively young patients. So we feel that we absolutely must expand into the pediatric population. And as Nathan pointed out, it will be based on our findings in our pediatric study that we will start in the second quarter of this year.