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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.

There are no further questions at this time. This concludes today's call. Thank you for attending. You may now disconnect.

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.

There are no further questions at this time. This concludes today's call. Thank you for attending. You may now disconnect.