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. 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 nine to unmute. 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, 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?
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, 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 III pivotal DRAGON trial in December.
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 atrophy lesion, measured by definitely decreased autofluorescence by fundus autofluorescence imaging compared with placebo. This result positioned 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. 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 III PHOENIX trial in GA with 430 subjects.
We completed a $402 million public offering with over allotment fully exercised by the underwriter in Q4. The net proceed from this, along with other raises completed in the year, positions us extremely well to support commercialization preparation for Stargardt disease, development and expansion of pipelines, and general corporate purposes.
Moving to 2026. As I said, this will be a transformative year for Belite Bio. The top priority is our planned NDA submission to the FDA in the second quarter of 2026. 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. We are now in the process of building our organization in sales, market access, medical affair, 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 a 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, first, expenses related to the DRAGON II trial.
Second, we received a lower Australian R&D tax incentive in Q4 2025, and 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 the fourth quarter was $12.2 million compared to $5.7 million for the same period in 2024.
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 service fee as we achieved development milestone and started to prepare for commercialization and filing. 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 report a net loss of $25.3 million compared to $10.1 million in Q4 2024.
On a non-GAAP basis, we report 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, $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, 2 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 equivalent, US 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 objective, including the commercial launch for Stargardt disease. With that, I'll turn the call back to the operator for Q&A.
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 Guber with Morgan Stanley. Your line is open. Please go ahead.
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 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.
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 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?
Yep. For the next three years, we expect the existing pipeline, you know, including the NDA submission and 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 million-$250 million.
Great. Thank you.
Your next question comes from the line of Tazeen Ahmad with Bank of America. Your line is open. Please go ahead.
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. Then can you just remind us what are the key gating items left before you submit the NDA in the second quarter? Thank you.
Hao, you wanna take this one as well?
Sure. Well, for the pricing, you know, apparently still early for us to set a price. But I think, you know, we have been seeing that the average rare disease drug price in the US being somewhere about $350,000. We do think it's fair to say that we expect our sale can be doing better than that, but still early to really set a price.
Okay, Gary.
Was there another question?
Yeah. What are the gaining factors left before you submit for approval in 2Q?
I guess we have everything ready. We're just waiting for the clinical study report. As we speak, we're on track.
Okay, great. Thank you.
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 star. Moving on, your next question comes from the line of Timur Ivannikov with Cantor. Your line is open. Please go ahead.
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 the second quarter, do you have initial expectations on the launch timing? Then 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 diseases? Thank you.
Hao, you wanna take this one as well?
Sure, sure. Well, we expect we probably will 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 25-30, 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 U.S. Yeah, I think, being able to launch by Q1 2027 is our goal.
To your question about the, you know, challenges, we think, compared with other disease, given there's no treatment for Stargardt disease, this should be a fairly straightforward drug. The difficulty will really be, you know, getting patients, getting, the, you know, physicians, be aware of 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 this will be the few execution kind of a task that we will be focused on. I wouldn't say those are like challenges for us.
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?
Thank you, Tom, I would like to confirm what Hao 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.
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.
Okay. Thank you very much.
Your next question comes from the line of Marc Goodman with Leerink Partners. Your line is open. Please go ahead.
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.
Thanks, Marc. You're saying that the timing of ex-US, NDA submissions or the US alone?
Yes. Yes. OUS. Exactly. Ex-US.
ex-U.S. Sorry. Okay. We want to set the priority with the FDA or U.S. We wanna pool resources to make sure that we are successful with the NDA in the U.S. Everything outside of U.S. 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 U.S. We are in constant communications with the EMA, the PMDA, and other authorities as well.
We wanna keep the U.S., keep all the bandwidth on the U.S. 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 out and then submitting it on too many regions.
Okay.
Hopefully that answers your questions.
Okay.
What was the other one?
The interim look for the Geographic Atrophy. Just curious what your latest thoughts are?
Right now we're probably expecting that will be somewhere second half of the year. We haven't actually looked at it yet because we're prioritizing everything on launching Tinlarebant for Stargardt. We will have a further update for that probably the next quarter.
Thanks.
Your next question comes from the line of Yi Chen with H.C. Wainwright. Your line is open. Please go ahead.
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 I study, and if you have any details on the specific liver indication as a primary lead.
I'll get Hendrik to answer on the GA one. I'll start with the LBS-009. Right now there's no plans for LBS-009 yet. Again, we're prioritizing everything on Tinlarebant, and be successful launch in the U.S. first. All the others will follow, and we'll prioritize after that.
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 a 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.
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. 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.
Got it. Thanks so much for taking the questions.
Your next question comes from the line of Boris Peaker with Titan. Your line is open. Please go ahead.
Great. Thank you very much for taking my question. Congrats on 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?
I'll refer this to Nathan and, of course, Hendrik to add more details as well. Nathan?
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.
Got it. Another-
If I could.
Just to follow up on... Oh, go ahead. Sorry.
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 18 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? 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.
Got it. Just another follow-up on Stargardt. I understand your initial emphasis is obviously gonna 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?
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.
Life expectancy of 60 y ears - 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 when visual acuity is the primary outcome measure. If you arrest progression, and the progression is 1.1 letters in two years, that would be the difference that you would target. Everybody knows that there's a 15-letter threshold set by the FDA to be clinically meaningful.
The intercession variability of visual acuity measurements in a population of macular deterioration patients such as Stargardt 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.
Got it. Thank you very much for taking my questions.
Certainly.
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.
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?
Yeah, of course. Right now we're open, we're pretty flexible that we do have multi-additional 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 with 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 is a good partnership after the FDA, after we get a FDA approval.
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.
Yes, of course. The FDA being top of our priority, second, I would say the EMA, probably next to it will be Japan as well, followed by China and all other regions.
Got it. Thank you.
Your final question will be from the line of Michael Okunewitch with Maxim Group. Your line is open. Please go ahead.
Hey there. Thank you 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?
Hendrik, can I refer this question to you?
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 the limitations 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 and 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 Bielefeld 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. 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 wanna add?
No, no. I think Hendrik covered it very nicely. Yes, we did a published 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. Yeah, 53,000 in U.S. and ex-U.S. 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.
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?
The short answer
Definitely. Yeah.
Let me just take that real quick. We do have an approved pediatric investigational 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.
Hendrik, I believe that, you answered the same question as well at one of, the medical conferences just a month ago.
Yeah, indeed. We feel that, although in DRAGON patients already had significantly lost vision on average, we feel that our 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 second quarter of this year.
Thank you very much.
There are no further questions at this time. This concludes today's call. Thank you for attending. You may now disconnect.