Q4 2025 Belite Bio Inc Earnings Call

March 2, 2026

Speaker #1: Ladies and gentlemen, thank you for joining us, and welcome to the Belite Bio fourth quarter and fiscal year-end 2025 earnings call. After today's prepared remarks, we will host a question-and-answer session.

Operator: Ladies and gentlemen, thank you for joining us. Welcome to the Belite Bio Q4 and fiscal year end 2025 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 9 to raise your hand and star 6 to unmute. I will now hand the conference over to Sophie Hunt. Please go ahead.

Speaker #1: If you would like to ask a question, please raise your hand. If you have dialed into today's call, please press star 9 to raise your hand and star 6 to unmute.

Speaker #1: I will now hand the conference over to Sophie Hunt. Please go ahead.

Speaker #2: Good afternoon, everyone. Thank you for joining us. On the call today are Dr. Tom Lynn, 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 Officer.

Tom Lin: 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. Now I'll turn the call over to Hao. Hao?

Sophie Hunt: 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. Now I'll turn the call over to Hao. Hao?

Speaker #2: 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.

Speaker #2: 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.

Speaker #2: 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.

Speaker #2: Hao?

Speaker #3: 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.

Hao-Yuan Chuang: Thank you for joining today's call to discuss our Q4 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 to potentially become the first ever approved therapy for people living with Stargardt disease, a 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 result for the Phase 3 DRAGON trial in December.

Speaker #3: We look forward to a truly transformative year in 2026 as we position China to potentially become the first-ever approved therapy for people living with SARS disease.

Speaker #3: The devastating eye disease that usually begins in childhood or young adulthood leads to progressive vision loss and then legal blindness in almost all cases.

Speaker #3: Today, I'll provide a recap of our 2025 achievements, key milestones for 2026, and financial results. Starting with 2025 achievement, of course, the most significant achievement was the announcement of our top-line result for the phase III free throw dragon trial in December.

Speaker #3: We're very excited to share that the trial met its primary efficacy endpoint, demonstrating statistically significant, and clinically meaningful 36% reduction in the growth rate of ocular lesions.

Hao-Yuan Chuang: We're very excited to share that the trial met its primary endpoint, demonstrating statistically significant and clinically meaningful 36% reduction in the growth rate of 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 27 February, we had enrolled 72 subjects. A subject 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 3 PHOENIX trial in GA with 1 to 30 subjects.

Speaker #3: Measured by definitively 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 SARS disease.

Speaker #3: In our 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.

Speaker #3: We expect the final number of subjects enrolled to be between 72 and 75. We also completed the enrollment in the phase III Phoenix trial in GA with founder 30 subjects.

Speaker #3: Finally, we completed a 402 million public offering with overall allotment fully exercised by the underwriter in Q4. Importantly, the net proceeds from this along with other raises completed in the year were suggesting us extremely well to support commercialization preparation for SARS disease.

Hao-Yuan Chuang: Finally, we completed a $402 million public offering with over allotment fully exercised by the underwriter in Q4. Importantly, the net proceed from this, along with other raises completed in the year, positions us extremely well to support commercialization preparation for Stalerzi, development and expansion of pipelines, and general corporate purpose. 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 Q2 2026. With our NDA submission planned, we have also kicked off our commercialization preparation work for Stalerzi. I'm pleased to share that we have hired all of the key leadership positions. We are now in the process of building our organization in sales, market access, medical affair, marketing, regulatory, and operations, et cetera.

Speaker #3: 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 is our planned MDS submission to the FDA in the second quarter of 2026.

Speaker #3: And with our MDS submission planned, we have also kicked off our commercialization preparation work for SARS disease. I'm pleased to share that we have higher all of the key leadership positions right now in the process of building our organization in sales, market access, medical affairs, marketing, regulatory, and operations, etc.

Speaker #3: 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 a financial recap.

Hao-Yuan Chuang: 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 a financial recap. For Q4, R&D expenses were $14.6 million compared to $7.3 million in Q4 2024. The increase was primarily due to, 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. Third, API manufacturing expenses. On a non-GAAP basis, which exclude share-based compensation expenses, R&D expenses for Q4 was $12.2 million, compared to $5.7 million for the same period in 2024.

Speaker #3: 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 first expenses related to the DRAGON II trial.

Speaker #3: Second, we received a lower Australian R&D tax incentive in Q4 2025 as such an incentive was received in Q3 2025 versus last year it was received in Q4 2024.

Speaker #3: And third, API manufacturing expenses. On a non-GAAP basis, which excludes share-based compensation expenses, R&D expenses for the fourth quarter were $12.2 million, compared to $5.7 million for the same period in 2024.

Speaker #3: We believe this non-GAAP basis provides a better picture about operating expenses since our share-based compensation expenses are heavily driven by achieving development milestones and the volatility of our own stock price and the comparable company stock price using the valuation.

Hao-Yuan Chuang: We believe this non-GAAP basis provide a better picture about operating expenses since our share-based compensation expenses is heavily driven by achieving development milestone and the volatility of our own stock price and the 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 services as we achieved development milestone and started to prepare for commercialization and filing. On a non-GAAP basis, SG&A expenses for Q4 was $4.2 million compared to $1.5 million in Q4 2024. Overall, Q4, we report a net loss of $25.3 million compared to $10.1 million in Q4 2024.

Speaker #3: G&A expenses were $13.5 million, compared to $4.2 million in Q4 2024. The increase was primarily due to increasing share-based compensation expenses and professional service fees.

Speaker #3: As we achieved development milestones and started to prepare for commercialization and filing. On a non-GAAP basis, as GNA expenses for the fourth quarter was $4.2 million, compared to $1.5 million in Q4 2024.

Speaker #3: Overall, the fourth quarter, we report a net loss of $25.3 million, compared to $10.1 million in Q4 2024. On a non-GAAP basis, we report a net loss of $13.6 million.

Hao-Yuan Chuang: On a non-GAAP basis, we report net loss of $13.6 million for Q4 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 the 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.

Speaker #3: For the fourth quarter, compared to $5.9 million for Q4 2024. For the full year, R&D expenses were $14.4 million, compared to $29.9 million. For the full year 2024, the full year increase was primarily due to, first, expenses related to the Phoenix trial; second, share-based compensation expenses; and third, API manufacturing expenses.

Speaker #3: Partially offset by the royalty payment recognized in 2024. On a non-GAAP basis, excluding share-based compensation expenses, the R&D expenses for the full year were $36.2 million, compared to $26.2 million for the same period in 2024.

Speaker #3: As GNA expenses were $38.9 million, compared to $10.1 million in 2024, the increase was primarily due to increasing share-based compensation expenses and professional service fees.

Hao-Yuan Chuang: The increase was primarily due to increase in share-based compensation expenses and professional services 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, $9.1 million compared to $4.8 million in 2024. For the full year, we report 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, two registered direct offerings, and a significant pipe. We're very grateful to our shareholders for their strong support.

Speaker #3: As we achieved development milestones and started to prepare for filing and commercialization. On a non-GAAP basis, as GNA expenses were for the full year were $9.1 million, compared to $4.8 million in 2024.

Speaker #3: For the full year, we report 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.

Speaker #3: Moving to the balance sheet, as I said, we had a successful year of fundraising, so underwritten public offering, two registered direct offerings, and a significant PIE.

Speaker #3: 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 equivalents, U.S. Treasury bills and notes, as compared with $145.2 million at the end of 2024.

Hao-Yuan Chuang: As a result, we closed the year with $772.6 million in 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 Stalerzi. With that, I'll turn the call back to the operator for Q&A.

Speaker #3: Our balance sheet remains strong, and we are well positioned to deliver our near- and long-term objectives, including the commercial launch for SARS disease. With that, I'll turn the call back to the operator for Q&A.

Speaker #1: 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 into today's call, please press *9 to raise your hand and *6 to unmute.

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 into today's call, please press star nine to raise your hand and star six to unmute. Please stand by while 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.

Speaker #1: Please stand by while we compile the Q&A roster. Your first question comes from the line of Judah Frommer with Morgan Stanley. Your line is open.

Speaker #1: Please go ahead.

Speaker #3: Yeah, hi guys. Thanks for the update. Just a couple of questions from us. I guess on the NDA submission, are you still thinking about that being a rolling submission?

Hao-Yuan Chuang: Yeah. Hi, guys. Thanks for the update. Just a couple questions for us. I guess on the NDA submission, are you still thinking about that being a rolling submission? What role would DRAGON II?

Speaker #3: And what role would dragon II play within that submission process? I would maybe in the US 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 STARGARD trials, getting through GA, and commercialization, and anything else we should be thinking about?

Judah Frommer: Play within that submission process, I would maybe in the US and other geographies as well. 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? Thank you.

Speaker #3: Thank you.

Speaker #4: 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.

Tom Lin: Okay. I'll answer the first question regarding the NDA. 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. Once that's finalized, we are ready to submit pretty soon. What's the next one? The DRAGON II. Yes. The DRAGON II will be for Japan only because of the Japanese authorities would like to see the data on Japanese patients. That's strictly for Japan only. The commercialization and the budget, I think, was the other question. I'll refer that to Hao. Hao?

Speaker #4: And once that's finalized, we are ready to submit pretty soon. What's the next trend—Dragon II? Yes. So the Dragon II will be for Japan only.

Speaker #4: And because of the Japanese authorities would like to see the data on Japanese patients. So that's strictly for Japan only. And the commercialization and the budget, I think it was the other question.

Speaker #4: I'll refer that to how.

Hao-Yuan Chuang: Yep. For the next three years, we expect the existing pipeline, you know, including the NDA submission, all of those, what we call that, like R&D kinda related activity, will cost us about $150 million. For the commercialization itself for the next three years is probably somewhere between $200 to $250 million.

Speaker #3: Yep. So for the next three years, we expect the existing pipeline including the NDA submission, all of those what we call that R&D kind of related activity or causes about $150 million.

Speaker #3: And for the commercialization itself over the next three years, it's probably somewhere between $200 million and $250 million.

Speaker #4: Great. Thank you.

Judah Frommer: Great. Thank you.

Speaker #1: Your next question comes from the line of Taseen Ahmad with Bank of America. Your line is open. Please go ahead.

Operator: Your next question comes from the line of Tazeen Ahmad with Bank of America. Your line is open. Please go ahead.

Speaker #5: Okay, great. Good afternoon. Thanks for taking my questions. Can you just give us a little bit of guidance on how we should be thinking about pricing?

Tazeen Ahmad: Okay, great. Good afternoon. Thanks for taking my questions. 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 unmet need, we'd be curious to maybe get a sense of a range of what would be appropriate to be considering here. Can you just remind us what are the key gating items left before you submit the NDA in Q2? Thank you.

Speaker #5: Given the profile of the drug and given the undermed need, we'd be curious to maybe get a sense of a range of what would be appropriate to be considering here.

Speaker #5: And then can you just remind us what are the key gating items left before you submit the NDA and the second quarter? Thank you.

Speaker #4: How you want to take this one as well?

Tom Lin: Hao, you wanna take this one as well?

Speaker #3: 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 rate of DD drug price in the US being somewhere about $350,000.

Hao-Yuan Chuang: Sure. Well, for the pricing, you know, apparently still early for us to set a price. I think, you know, we have been seeing that the average rare disease drug pricing in the US being somewhere about $350,000. 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.

Speaker #3: 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.

Tom Lin: one more-.

Speaker #4: So, what was the other question?

Tom Lin: one more-.

Tazeen Ahmad: Okay, great. Then on-

Tom Lin: Do you have a question?

Speaker #5: Yeah. What are the gating factors left before you submit for approval in Q2?

Tazeen Ahmad: Yeah. What are the gating factors left before you submit for approval in Q2?

Tom Lin: I guess we have everything ready. We're just waiting for the clinical study report. As we speak, we are on track.

Speaker #4: I guess we have everything ready. So we're just waiting for the clinical study report. So as we speak, we are on track.

Tom Lin: I guess we have everything ready. We're just waiting for the clinical study report. As we speak, we are on track.

Speaker #5: Okay. Great. Thank you.

Tazeen Ahmad: Okay, great. Thank you.

Speaker #1: Your next question comes from the line of Mark Goodman with Liebrink. Your line is open. Please go ahead. Mark, as a reminder, kindly unmute yourself by pressing star 6.

Operator: Your next question comes from the line of Marc Goodman with Leerink. Your line is open. Please go ahead. Marc, as a reminder, kindly unmute yourself by pressing star six. Moving on, your next question comes from the line of Timur Ivannikov with Cantor. Your line is open. Please go ahead.

Speaker #1: Moving on. Your next question comes from the line of Timur Ivanikov with Kantor. Your line is open. Please go ahead.

Timur Ivannikov: Yes, thank you. This is Timur Ivannikov on for Steve Seedhouse. Our question is about the timing of your potential launch. Assuming, assuming you have an NDA filing in Q2, do you have initial expectations on the launch timing? I think you were talking about maybe 25 field reps, but how quickly after the approval do you think you can launch? How do you assess the difficulty of this launch maybe to other rare diseases or other retinal disease? Thank you.

Speaker #4: Yes. Thank you. This is Timur Ivanikov on 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?

Speaker #4: 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?

Speaker #4: Thank you.

Speaker #6: How you want to take this one as well?

Tom Lin: Hao, you wanna take this one as well?

Speaker #3: Sure. Sure. Well, so we expect we're probably going to 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.

Hao-Yuan Chuang: Sure. Sure. Well, we expect we'll probably launch by Q1 2027. The sales team, as you said, we expect that we have probably a team more, you know, focused on genetic testing, which will be, you know, one of the key factors to get the patient confirmed. The second team will be more about the drug, about the brand. Total somewhere like 25 to 30, we think it's a fair assumption at launch. Potentially after two years of the launch, you may expand that team further as you know, wanna get to every corner in the US. I think being able to launch by Q1 2027 is our goal.

Speaker #3: The second team will be more about the drug, about the brand. So total, somewhere like 25 to 30 with a fair assumption at launch. Potentially, after two years of launch, you may expand that team further as you want to get to every corner in the US.

Speaker #3: Yeah, 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 diseases, given there's no treatment for Stargardt disease, this should be a fairly straightforward drug.

Hao-Yuan Chuang: To your question about the, you know, 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 in getting patients, getting, the, you know, physicians, be aware this treatment is available. Then, you know, shorten the time it takes for people to get the genetic testing done and get their insurance coverage. I think that piece will be the few execution kind of tasks that we will be focused on. I wouldn't say those are like challenges for us.

Speaker #3: The difficulty will really be getting patients, getting the physicians to be aware that this treatment is available, and then shortening the time it takes for people to get the genetic testing done and get the insurance coverage.

Speaker #3: I think these will be the few execution kind of attacks that we will be focused on. But I wouldn't see those are challenges for us.

Speaker #4: So, how maybe we could get Hendrik to also add more color to this question, given that he is a prescriber himself. He looks after these STARGARDT patients, and he knows the whole clinical landscape very well.

Tom Lin: Hao, maybe we could get Hendrik to also add more color to this question, given that he's a prescriber himself. He looks after these Stargardt patients, and he knows the whole clinical landscape very well. Hendrik, you wanna add anything, any details?

Speaker #4: So, Hendrik, do you want to add anything—any details?

Speaker #3: Yes, thank you, Tom. But I would like to confirm what we just said and pointed out. It’s a fact that many patients are lined up in large databases.

Hendrik Scholl: Yes. Thank you, Tom, but I would like to confirm what Howard 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. Such centers typically have database of patients where they also include the genotype of these patients. 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. I believe because this is a monogenic disease, there's an extra opportunity to get to patients very quickly.

Speaker #3: Many of STARGARD 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 databases of patients where they also include the genotype of these patients.

Speaker #3: 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.

Speaker #3: So I believe, because this is a more genetic disease, there's an extra opportunity to get to patients very quickly.

Speaker #4: Okay. Thank you very much.

Tom Lin: Okay. Thank you very much.

Speaker #1: Your next question comes from the line of Mark Goodman with Liebrink. Your line is open. Please go ahead.

Operator: Your next question comes from the line of Marc Goodman with Leerink. Your line is open. Please go ahead.

Speaker #4: Yeah, sorry about the confusion, guys. 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?

Marc Goodman: Yeah. Sorry about the confusion, guys. Can you talk about your filing plans OUS? Secondly, what are your latest thoughts on the timing of an interim look for the GA work you're doing? Thanks.

Speaker #4: Thanks.

Speaker #6: Thanks, Mark. So you're saying that the timing of XUS and the estimations or the US alone?

Tom Lin: Thanks, Marc. You're saying that the timing of ex-US NDA submissions or the US label?

Marc Goodman: Yes. Yes. OUS. Exactly. Ex-US.

Speaker #4: Yes. Yes. OUS. Exactly. XUS.

Speaker #6: Oh, XUS. Sorry. Okay. So, we want to set the priority of the FDA US. We want to pull all the resources to make sure that we are successful with the NDA in the US.

Tom Lin: Oh, ex-US. Sorry. Okay. We want to set the priority of the FDA or US. We wanna pool resources to make sure that we're successful with the NDA in the US. Everything outside of the US will build onto that. This requires discussions with the regulatory authorities in different regions to see what type of timing that we're expecting or they're expecting. This will be an update which regions that we'll prioritize after the US. We are in constant communications with the EMA, the PMDA, and other authorities as well.

Speaker #6: So everything outside of the US will build onto 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.

Speaker #6: So, this will be an update on which regions we'll prioritize after the US. We are in constant communication with the EMA, the PMDA, and other authorities as well.

Speaker #6: So we want to keep the US keep all the bandwidth on the US FDA given that we expect this is going to be a lot of questions so we don't want to dilute all our resources at this point by spreading it to spread it out and then submitting it on too many regions.

Tom Lin: We wanna keep the US, keep all the bandwidth on the US FDA, given that, you know, we expect there's gonna be a lot of questions. We don't wanna dilute all our resources at this point by spreading it too out, spread out and then submitting it on too many regions for the answers and questions.

Speaker #6: Are there answers to questions?

Speaker #4: Okay.

Marc Goodman: Okay.

Tom Lin: What was the other one?

Speaker #6: What was the other one?

Tom Lin: What was the other one?

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

Speaker #4: The interim look for the geographic atrophy. Just curious what your latest thoughts are.

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

Speaker #6: Yeah. So right now, we're probably expecting that would be somewhere second half of the year. We haven't actually looked at it yet because we're prioritizing everything on launching 10/11 for STARGARD.

Tom Lin: Yeah. Right now we are probably expecting that will 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. We will have a further update for that probably the next quarter.

Speaker #6: So we will have a further update for that, probably in the next quarter.

Speaker #4: Thanks.

Marc Goodman: Thanks.

Speaker #1: Your next question comes from the line of Yi Chen with HC Wainwright. Your line is open. Please go ahead.

Operator: Your next question comes from the line of Yi Chen with H.C. Wainwright & Co. Your line is open. Please go ahead.

Operator: Your next question comes from the line of Yi Chen with H.C. Wainwright. Your line is open. Please go ahead.

Speaker #6: Hi, 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 a success in that broad population?

Eduardo Garcia: Hi, 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 a success in that broad population? 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 1 study, and if you have any details on the specific liver indication as a primary lead.

Speaker #6: 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 one study.

Speaker #6: And if you have any details on a specific liver indication as a primary lead. So I'll get Hendrik to answer on the GA one.

Tom Lin: I'll get Henrik to answer on the GA one. I'll start with the 009. Right now there's no plans for 009 yet. Again, we're prioritizing everything on tadalafil and be a successful launch in the US first. All the others will follow, and we'll prioritize after that. Henrik.

Speaker #6: I'll start with the 009. Right now, there's no plans for 009 yet. So again, we'll prioritizing everything on 10/11 and be successful launch in the US first.

Speaker #6: All the others will fall and we'll prioritize after that.

Speaker #4: And I'm happier. Thank you, Tom. I'm very happy to take the question on what's the threshold that would make a treatment of GA success with our oral compound.

Hendrik Scholl: I'm happy. Yeah. Thank you, Tom. I'm very happy to take the question on what's the threshold that would make a treatment of GA success with our oral compound. When you think about OAKS, DERBY, and GATHER2, the injectable, so 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.

Speaker #4: When you think about oaks, derby, and geather too, the injectable, so Cefova and Esove, they found efficacy signals of 13, 21, and 14 percent in their registration trials.

Speaker #4: 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.

Speaker #4: Having said that, I mean, we are more ambitious. Given what we found in STARGARD disease, 36%, we feel that reaching 13, 21, 14 percent, so roughly what something between 15 and 20 percent should absolutely be possible.

Hendrik Scholl: Given what we found in Stargardt disease, 36%, we feel that, reaching 13%, 21%, 14%, so roughly what, something between 15% and 20% could absolutely be possible, and we would like to go beyond that. 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.

Speaker #4: 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.

Speaker #4: To tell them to come in for injections every month if there is an oral treatment available. Got it. Thanks so much for taking the questions.

Eduardo Garcia: Got it. Thanks so much for taking the questions.

Speaker #1: Your next question comes from the line of Boris Peaker with Titan. Your line is open. Please go ahead.

Operator: Your next question comes from the line of Boris Peaker with Titan. Your line is open. Please go ahead.

Speaker #5: Great. Thank you very much for taking my question. Congrats on the progress. I guess maybe we'll start with STARGARD. Do you anticipate the label to become a broad STARGARD label for all patients, or would you think potentially be restricted to patients ages maybe 12 to 20, similar to the Pivotal study?

Boris Peaker: Great. Thank you very much for taking my question, and congrats on the progress. Guess 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?

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

Speaker #6: I'll refer this to Nathan and, of course, Hendrik to add more details as well. Nathan?

Nathan Mata: Yeah. Nathan here, the CSO. 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. 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. Again, spectrum of the same disease across different populations.

Speaker #3: Yeah. 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.

Speaker #3: 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.

Speaker #3: 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 dysfunction of the same protein.

Speaker #3: So again, spectrum of the same disease across different populations.

Speaker #5: Got it. And another just to follow up on oh, go ahead. Sorry.

Boris Peaker: Got it. another just to follow up on... Oh, go ahead. Sorry.

Speaker #4: No, I just wanted to add that it's all about the generalizability of the data, right? And there has rarely been such an easy case to convince the regulator this is the same disease.

Hendrik Scholl: No, I just wanted to add that it's all about the generalizability of the data, right? There has rarely been such an easy case to convince the regulator this is the same disease. We included adult subjects 80 to 20 years, but we also included adolescents, as you know, right? If there is a patient affected at age 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 Nathan pointed it out, the ProgStar study has shown that progression rates amongst different age groups, 12 to 18 to 50 and beyond 50, were essentially similar.

Speaker #4: 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 age 20, 22, 28, 32, with biallelic mutations in ABCO4, why would that be considered a different disease?

Speaker #4: Why would somebody believe there would be no efficacy if you treat later? Because, and Nathan pointed that 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.

Speaker #5: Got it. And just another follow-up on STARGARD. Now, I understand your initial emphasis is obviously going to be on the US market, but I'm just curious—for the ex-US opportunity, how important is visual acuity?

Boris Peaker: Got it. Just another follow-up on Stargardt. Now, I understand your initial emphasis is obviously going to be on the US market, but I'm just curious, for the ex-US opportunity, how important is visual acuity, I guess, for approval and potentially for just reimbursement and justifying pricing?

Speaker #5: I guess for approval and potentially for just reimbursement, and justifying pricing.

Speaker #6: Hendrik, do you want to take this as well?

Tom Lin: Hendrik, do you want to take this as well?

Hendrik Scholl: Yes. 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, even if you have a treatment that arrests the progression, to find an efficacy signal when 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.

Speaker #4: 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 STARGARD disease would find visual acuity efficacy signal, right?

Speaker #4: 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 even if you have a treatment that arrests the progression, to find an efficacy signal when visual acuity is the primary outcome measure.

Speaker #4: If you arrest progression and the progression is 1.1 letters in two 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.

Hendrik Scholl: Everybody knows that there's a 15 letter threshold set by the FDA to be clinically meaningful. The intersession variability of visual acuity measurements in a population of macular deterioration patients such as Stargardt disease is eight letters. Meaning that visual acuity as an outcome measure is an unrealistic target. 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.

Speaker #4: And the inter-session variability of visual acuity measurements in a population of markedly degenerated patients, such as Stargardt, is 8 letters. So, meaning that visual acuity as an outcome measure is an unrealistic target.

Speaker #4: 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.

Speaker #4: It just means that you have to treat for a while until, eventually, you will see a visual acuity benefit.

Speaker #5: Got it. Thank you very much for taking my questions.

Boris Peaker: Got it. Thank you very much for taking my questions.

Speaker #4: Certainly.

Hendrik Scholl: Certainly.

Speaker #1: As a reminder, if you would like to ask a question, please raise your hand now. If you have dialed into today's call, please press *9 to raise your hand and *6 to unmute.

Operator: As a reminder, if you would like to ask a question, please raise your hand now. If you have dialed into today's call, please press star nine to raise your hand and star six to unmute. Your next question comes from the line of Bruce Jackson with Benchmark. Your line is open. Please go ahead.

Speaker #1: Your next question comes from the line of Bruce Jackson with Benchmark. Your line is open. Please go ahead.

Speaker #6: Hi. Good afternoon. 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?

Bruce Jackson: Hi. good afternoon. 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?

Speaker #3: Yeah, of course. So right now, we're open we're pretty flexible that we do have multinational pharmaceutical companies wanting to partner or license right now.

Tom Lin: Yeah, of course. Right now we're open. We're pretty flexible 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, seems pretty straightforward for all regulatory authorities. 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. We believe that we have a very straightforward approval path for all other regions as well. It depends on what kind of reasonable deals or deals that we think was a good partnership after the FDA, at the approval.

Speaker #3: That's still open. But we believe right now, at least, our regulatory submission pathway seems pretty straightforward for all regulatory authorities. So we believe we could add more value, at least starting from the FDA, once we get the approval.

Speaker #3: 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.

Speaker #3: So it depends on what kind of reasonable deals, or deals that we think are a good partnership, after the FDA—after we get an FDA approval.

Speaker #6: Okay, great. And then if I could just get a follow-up on the ex-US regulatory strategy—you've got quite a bit going on this year.

Bruce Jackson: Okay, great. If I could just get a follow-up on the ex-US 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? That's it for me. Thank you.

Speaker #6: Do you intend to seek further approvals in Europe and when might those get submitted? And that's it for me.

Tom Lin: Yes, of course. The FDA being top of our priority, and then second, I would say the EMA. Probably next to it will be Japan as well, and then followed by China and a lot of the regions.

Speaker #3: Yes, of course. So the FDA has been top of our priority and then second, I would say, the EMA. And probably next to it would be Japan as well.

Speaker #3: And then followed by China and all other regions.

Speaker #6: Got it. Thank you.

Hao-Yuan Chuang: Got it. Thank you.

Speaker #1: Your final question will be from the line of Michael Akunowicz with Maxim. Your line is open. Please go ahead.

Operator: Your final question will be from the line of Michael Okunewitch with Maxim. Your line is open. Please go ahead.

Speaker #4: Hey there. Thanks for taking my questions today and 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 STARGARD disease is given there have been no approved therapies.

Michael Okunewitch: Hey there. Thanks for taking my questions today and 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 disease is, given there have been no approved therapies. Do you expect that having something available could help build awareness and uncover additional undiagnosed patients?

Speaker #4: Do you expect that having something available could help build awareness and uncover additional undiagnosed patients?

Speaker #6: Hendrik, can we fill this question for you?

Tom Lin: Hendrik, can we put this question to you?

Speaker #4: Yeah, 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.

Hendrik Scholl: Yeah. I'm happy to answer the question. 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. 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?

Speaker #4: 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.

Speaker #4: In STARGARD disease, the symptoms are more straightforward than in RPE65. It's a much more diffuse disease affecting night vision in the periphery. In STARGARD disease, central vision is affected.

Speaker #4: Patients seek clinical care but we will need a genetic diagnosis in order to treat patients. What is the true prevalence of STARGARD disease in the past for rare diseases?

Hendrik Scholl: 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 Mountains Eye Study, Rotterdam Study, what the prevalent eye diseases are. 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. 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.

Speaker #4: It was very difficult to find out what the actual prevalence is. It's only known in the Beaverton eye study Blue Mountains eye study, Rotterdam eye study what the prevalent eye diseases are.

Speaker #4: 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 the and taking into account the race mix in the United States that we need to consider about 53,000 patients being affected by ABC04 mutated retinal disease, including STARGARD disease.

Hendrik Scholl: 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.

Speaker #4: 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.

Tom Lin: Nathan, I believe you've published on this, a few times. Anything you wanna add?

Speaker #6: Nathan, I believe you've published on this a few times. Anything you want to add?

Speaker #3: No, no. I think Hendrik covered it very nicely. Yes, we did publish a review article recently, tapping the prevalence of Stargardt disease, looking at it geographically across the world.

Nathan Mata: 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. You can look for that paper. It's published under my name and Hendrik's name just recently. Yeah, 53,000 in the United States and ex-US, of course, more than that, globally. Again, the genetics really tells us what the prevalence are, and that's what the data are based upon in terms of the publication that we recently submitted, that recently got accepted. Thank you.

Speaker #3: And you can look for that paper. It's published under my name and Hendrik's name—just recently. But yes, so 53,000 United States, and ex-US, of course, more than that globally.

Speaker #3: So, and again, the genetics really tells us what the prevalence are. And that's what the data are based upon in terms of the publication that we recently submitted—that recently got accepted, thank you.

Michael Okunewitch: Thank you. 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? Are there any plans for this expansion?

Speaker #4: Thank you. 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?

Speaker #4: And are there any plans for this expansion?

Speaker #3: So yeah, let me just take that real quick. So, we do have an approved pediatric investigational plan with the EMA, which we plan to initiate in April of this year.

Nathan Mata: The short answer.

Michael Okunewitch: Definitely. Yeah.

Nathan Mata: Yeah, let me just take that real quick. We do have an approved Paediatric Investigation Plan with EMA, which we plan to initiate in April of this year. That's coming up very soon. That is a two-year study, looking at safety and efficacy in children three to 11 years of age. We'll have to wait to see what the safety and efficacy data look like at the end of the two-year study. Certainly, we do have plans to establish safety and efficacy in patients younger than 12.

Speaker #3: So that's coming up very soon. That is a two-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 two-year study.

Speaker #3: But certainly, we do have plans to establish safety and efficacy in patients younger than 12.

Speaker #6: And Hendrik, I believe that you answered the same question as well at one of the medical conferences just a month ago.

Tom Lin: Hendrik, I believe that you answered the same question as well at one of the medical conferences just a month ago.

Speaker #4: Yeah. 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 Tinlaraban treatment.

Hendrik Scholl: Yeah, indeed. 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. We feel that we absolutely must expand into the pediatric population. As Nathan pointed out, it will be based on our findings in our pediatric study that we will start in the Q2 of this year.

Speaker #4: 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.

Speaker #6: Thank you very much.

Michael Okunewitch: Thank you very much.

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

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