BLTE - NASDAQ

Ladies and gentlemen, thank you for joining us, and welcome to the Belite Bio Second Quarter 2025 Earnings Call. [Operator Instructions] I will now hand the call 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. Nathan Mata, Chief Scientific Officer; Dr. Hendrik Scholl, Chief Medical Officer; and Hao-Yuan Chuang, Chief Financial Officer. Before we begin, let me point out that we will be making forward-looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and actual results may differ materially. We encourage you to consult the risk factors discussed in our SEC filings for additional detail. Now I'll turn the call over to Dr. Lin.

Thank you for joining today's call to discuss our second quarter 2025 financial results. We have made excellent progress in the quarter towards advancing Tinlarebant narrowband in patients living with Stargardt's disease and geographic atrophy. For those who are new to our story, Tinlarebant is a first-in-class oral therapy intended to reduce the accumulation of toxic vitamin A byproducts, which is implicated in the progression of retinal lesions in patients with Stargardt disease and geographic atrophy. We believe this approach will be effective in slowing or halting lesion growth, which would ultimately preserve vision. To give you some perspective on the importance of this potential therapy, Tinlarebant has been granted breakthrough therapy, rare pediatric disease and Fast Track designations in the U.S. and Pioneer designation in Japan. They've also been granted orphan drug designation in the U.S., Europe and Japan. We believe this speaks to the significant unmet need for both indications as currently, there is no approved treatment for Stargardt disease and no approved oral treatment for geographic atrophy. As importantly, we are uniquely positioned to address these unmet needs as we are already in global Phase III trials for both indications. So with that, let me provide a high-level overview of the recent progress we have made. We have 2 studies underway with Tinlarebant in patients living with Stargardt disease. These are the Phase III DRAGON trial and the Phase II/III DRAGON II trial. As part of the Phase III DRAGON trial, we were pleased to announce earlier in the year that the Data Safety Monitoring Board had completed its interim analysis and recommended that the trial proceed without a sample size increase or modifications. They also recommended that we submit the data for further regulatory review for drug approval. As a result, the FDA granted Tinlarebant Breakthrough Therapy designation, and we remain on track to complete the study in Q4 this year. The DRAGON II trial also continues to be on track. We have enrolled 17 of our targeted enrollment of approximately 60 subjects, including about 10 Japanese subjects. The data from the Japanese subjects is intended to expedite a new drug application in Japan, where we have already been granted a Pioneer Drug Designation. In geographic atrophy, I'm pleased to share that we recently completed enrollment of our global Phase III study with 529 subjects enrolled. We recently also raised $15 million in gross proceeds in a registered direct offering on August 8. To summarize, we made excellent progress against our key milestones in the first half of the year. We are looking to carry this momentum into the second half of the year. Our balance sheet is also strong with 4 years of cash runway. We remain well positioned to advance Tinlarebant as potentially the first oral treatment for people living with degenerative retinal disease. I'll now turn over the presentation to Hao-Yuan. Hao-Yuan?

Thank you, Tom. For Q2 2025, we had R&D expenses of $11 million compared to $9.1 million for the same period last year. The increase was mainly due to higher pass-through expenses related to the PHOENIX trial and manufacturing expenses, which was partially offset by lower DRAGON trial expenses and development milestone payment for the completion of the Phase II trial in 2024. Also due to an increase in share-based compensation expenses. Regarding G&A expenses, we had G&A expenses of $6.5 million compared to $1.4 million for the same period last year. The increase was mainly due to an increase in share-based compensation expenses. Overall, we had a net loss of $16.3 million compared to a net loss of $9.5 million for the same period last year. One thing to note is that as the majority of the increase of the expenses came from the share-based compensation, which was about $7.6 million and was not cash related. The operating cash outflow was only about $8.6 million. At end of Q2, we had $149.2 million in cash, liquidity fund, time deposit and U.S. treasury bill. We also raised $15 million in gross proceeds in a registered direct offering on August 8. We still expect 4 years of cash runway without considering the commercialization costs and expect to be able to complete all 3 Phase III trials with our current cash. Thank you. Back to you, operator.

[Operator Instructions] Your first question comes from the line of Boris Peaker with Titan Partners.

Congratulations on the progress. I guess my first question is, what is the status of the FDA discussion regarding the interim data that -- from the DRAGON trial? Just trying to get a sense if that could potentially be a trigger for filing BLA. And if it's not -- NDA in this case, sorry. And if it's not, what data would you think you'd need to actually get from DRAGON 2 study, 1-year follow-up, 2-year follow-ups, how long prior to an NDA filing?

Thank you. So I can take this one. So we've met with -- so we filed the breakthrough based on the IA data. We've met with the FDA. And so given the IA criteria was met when all subjects achieved 12 months on study, majority of subjects have already reached 15 month's. So I mean, we could try for accelerated approval, but that would still require a confirmatory follow-up study. So the FDA recommended that we complete the 24-month study, which we are about to complete coming up in September -- September, October. And so we will submit that final data, and there will possibly be a single study approval based on the robust statistical significance.

Got you. So just to confirm, so there could be a possibility just filing on DRAGON I and using its basis -- and then driving DRAGON II. And when would you actually know that if DRAGON I in and of itself is sufficient for approval?

So I think based on the FDA's recommendation that the p-value would be robust enough, and we believe that we're on track to meet the criteria.

Got you. Okay. I appreciate that. Maybe if I could squeeze in just one question on GA. Now that the PHOENIX trial has completed enrollment, can you discuss if there's going to be an interim analysis, let's say, a year from now or so or any other timing around the data readouts?

Yes, sure. So there will be an IA for GA. I think based on the overall regulatory strategy right now, I don't think I have the time line with me, but it will be somewhere between halfway during the -- similarly to the Stargardt. I just don't have the details, but it will be halfway mark.

Your next question comes from the line of Bruce Jackson with Benchmark.

So you got the breakthrough therapy designation. And have you discussed with the FDA yet if there's a route to accelerated approval?

Yes. Yes. So there was a route for accelerated approval, but the FDA, they recommended since that we are -- we have a majority of the patients completing the 15 month's time point already. So it will be long to reach the 24 months. And then given that the accelerated approval will still require a second study, they recommended for a single study. I mean, the recommended for single study path with robust statistical significance.

Okay. And then my second question is about educating the medical community about your drug. So are there any upcoming presentations or data readouts that we can look to near term, not just the completion or the follow-up analysis as of the study, anything on an interim basis.

Well, I guess that our time line is very tight that we are expecting. So we ran the time line with the FDA and they acknowledge that we'll be submitting around first half, first half of next year. And they want us to remain confidential on revealing any efficacy data. So we were planning on presenting the IA data at AAO, which is roughly around about mid-October. But given that we'll be submitting very soon after that. So the FDA would prefer that we stay confidential on the data.

Bruce, I think I can add to that. So once we have the final 24-month data, we will announce that. But you wouldn't need to wait until the submission to see the data. But before that, we will not reveal interim data.

Your next question comes from Jennifer Kim with Cantor.

All right. Maybe 2. To start with DRAGON, can you remind us what the exact timing of data will be after the trial finishes in the fourth quarter? And then how and what data will be communicated and what you're hoping to show in terms of efficacy to support filing? Maybe we can start.

Nathan, probably best that you have answer this question.

Yes, as you know, we're looking at the change of atrophic lesion growth, right? So lesion growth rate over time in all subjects at all available time points. So we're looking for significance between placebo and treatment in terms of changing the trajectory of lesion growth. So that's what we expect to show the agency is a statistically significant difference in lesion growth rates. And that is what you need for approval. It's the same endpoint that was used in geographic atrophy to get the 2 intravitreal drugs approved. So it's basically the same metric.

Okay. And then my second question is just for PHOENIX. What is the latest dropout rate you've seen to date? Is it still in the 20% range? And what can we expect in terms of the depth of data in an interim analysis?

Well within our expected range. The depth of the interim, we have not finalized the interim design yet. So we will announce that once we know.

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

So for the DRAGON II trial, what is the current estimate time line for reaching the target enrollment of 60 patients. Is the current enrollment speed meeting your expectation?

Yes, I can take that question. So we initially launched DRAGON I -- sorry, DRAGON II in Japan. And now we are in the process of expanding into additional countries, which we expect will accelerate enrollment. We strategically timed the DRAGON to not compete with DRAGON I so that we can speed up the completion of DRAGON I. And of course, in the case if the DSMB recommends further sample size addition, then that wouldn't slow down the completion of DRAGON I. But since that the DSMB recommended that we wouldn't need to add every sample size. So we're kind of speeding up DRAGON II. So we kind of purposely staggered DRAGON II to not compete with DRAGON I. So even though it's slower than what we expected, but that is because of that DRAGON II will compete with DRAGON I. So I think we are still on time. I think the expectation of completing the enrollment probably will take, I would say...

End of this year.

Yes, end of this year.

Understood. And I noticed that your operating expenses continue to rise in the second quarter. Can you talk about the primary drivers for operating expenses and whether this kind of level of expensing is sustainable as you approach key clinical milestones later this year.

Well, first of all, the operating expenses does include a majority of the expenses come from the share-based compensation. So those are not cash related. And it's a little bit hard to really have a so-called precise forecast about that because they are purely based on the valuation of the options and the probability of reaching those milestones. Many of our ESOP is going to be related for development milestone. So it's purely based on the probability instead of time. You only get those listed as after we reach those milestones. So if you remove those, then the cash outflow is actually pretty close to the Q1 2025. This year and next year, we do expect to have higher current cash burn given that we do expect to reach several milestones on both through the both -- through all 3 Phase III studies. So this year and next year, we probably expect to have cash outflow of around $40 million to $45 million for 2 years, this and next year. And then it will dial down a lot starting from 3 years from now. So that's why we still expect 4 years of cash flow runway.

[Operator Instructions] Your next question comes from the line of Michael Okunewitch with Maxim Group.

Congrats on all the great progress. So I just wanted to follow up a little bit on the single pivotal design, in particular, if under the current FDA because there have been a lot of changes over the past several months, if you received any communication from the current admin that confirms that single pivotal for full approval, perhaps around when you got breakthrough designation.

I think we have presumable -- with the FDA after submitting the data, which we got breakthrough status. I think the path forward is very clear for us and very straightforward for a single study, if we met the statistical significance, robust statistical significance that is p of less than 0.01, then we have a single study approval path.

All right. And then just a follow-up. Do you have a sense of what sort of scale of a commercial force you would need to actually bring this to market, just given that you do have 4 years of runway, and it seems like you're pretty well positioned to reach approval here?

The current cash burn that we forecast for 4 years runway does not include the full skill set of the commercialization costs, which we are in progress of preparing that. So we will build the budget once we confirm.

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

So I guess what I want to know is we get the data released in this press release that you were talking about late this year, early next year. We get good news. How soon after will we be able to file? What are the gating issues between that press release and the filing, specifically talk about CMC as well as long-term safety data.

So I can answer this question. So the first one, long-term safety data, I believe we will need a safety data of 300, I believe we've acquired it. So Hendrik, can you help me out on the GA study, we will have enough safety subjects, right?

Exactly, Tom, right? So the requirement is that we would have 300 subjects that would be 12 months on the drug. And including our PHOENIX trial, we easily will have that by the first quarter of next year.

Sorry, Marc, what was your other question?

So basically, you could use the safety from both studies you're saying to include into that. That's why you're there. And the other was the CMC.

So we have ongoing discussion with the CMC with the FDA. So, so far, we are on track. So it will probably be another discussion with the FDA before we submit.

But just from -- where are we on that? And just where is the whole clock.

We are on the registration batch that's required for NDA.