Hello, and thank you for joining us to discuss Belite Bio's Second Quarter 2024 Financial Results. Joining the call today are Dr. Tom Lin, Chairman and CEO of Belite Bio; Dr. Nathan Mata, Chief Scientific Officer; and Hao-Yuan Chuang, Chief Financial Officer. Before we begin, let me point out that we will be making forward-looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and actual results may differ materially. We encourage you to consult the risk factors discussed in our SEC filings for additional detail. Please note that you can submit questions throughout the call by clicking on the Q&A box at the bottom of your screen, and we will respond to questions following our prepared remarks. Now I'll turn the call over to Dr. Lin.

Thanks, Julie. Thank you, everyone, for joining our second quarter 2024 earnings call. We had another strong quarter advancing our Tinlarebant trials, and I'm pleased with our progress in the year-to-date. Let me start off with our accomplishments during the second quarter by the overview of our programs. We had a productive quarter marked by several advancements in our programs. We have initiated the Phase Ib and Phase 2/3 trial of Tinlarebant in adolescent stargardt patients, also known as a DRAGON II study. And we have already completed enrollment for Phase Ib portion with 6 subjects in Japan. We've also received Sakigake designation in Japan, which means Pioneer Drug Designation in English. Such designation has only been granted to 27 drugs in its initiation in 2015, and Tinlarebant is the first ophthalmic drug to receive Sakigake designation, which is a testament to the groundbreaking potential of this drug and the unmet need it tends to address the people living with stargardt disease. We've made progress in our pivotal global Phase 3 trial of Tinlarebant in geographic atrophy subject as well, now as the Phoenix study and have already enrolled nearly 200 subjects to upfront -- until now. In addition, we've also raised $25 million from the registered direct offering in April here for strengthening our balance sheet. The Phase III DRAGON I study is fully enrolled with estimated interim readouts by Q4 2024 or early 2025 at the latest. As we enter the second half of the year, we are well positioned to execute on key milestones, and we look forward to sharing interim analysis from - initial interim analysis from our pivotal Phase III DRAGON study in the fourth quarter. Next slide, please. Okay. So for those of you, they were new to the story, that's the story. Tinlarebant…

Yes. Thank you very much, Tom. So what I'd like to share with you is the data from our 2-year open-label Phase II study in adolescent Stargardt subjects. This study was, as I mentioned, a 2-year study, 13- enrolled subjects from Taiwan and Australia. What a lot of people don't understand about Stargardt disease is there are over 1,500 known mutations that are associated with the disease. Not all of them are known to be pathogenic. In fact, many are mild to benign. So one of the analyses we did initially was to determine the genetic composition in our cohort. And we actually gave the genetic data to one of the premier preeminent genetics in Stargardt's disease in the world, Dr. Rando Allikmets at Columbia University. He evaluated our genetic data and determined that 11 of 13 subjects in our cohort had severe biallelic mutations, which we predict pathogenicity. And in those two, where there was a moderate allele [ph] in these 2 subjects, in vitro testing actually showed that these were pathogenic alleles. So our entire cohort really has severe pathogenic mutations that were predicted to progress very rapidly through the disease course. An independent assessment of the genetic severity is provided by something called the CAD score. That sounds for combined annotation dependent depletion score. It tells you the degree of severity of a particular genetic variant or genetic mutation. Scores above 20 are predicted to be among the 1% most deleterious. And every single one of our subjects with the section of 3 and 5 had these CAD scores above 20. So we have two independent confirmations on the severity of the genotypes of these kids. And despite the severity of these genotypes, we had five subjects, which represents 42% of the cohort that never developed atrophic…

Thank you, Nathan. So in Q2 2024, we had R&D expenses of $9.1 million compared to $5.5 million for the same period in 2023. The increase was primarily due to increase in expenses related to the milestone payment to Colombia for the completion of the Phase II study and share-based compensation. On G&A expenses, in Q2 2024, G&A expenses were $1.4 million, basically the same as Q2 2023. On net loss, we had a net loss of $9.5 million in Q2 2024 compared to $6.8 million for the same period in 2023. Regarding cash, we have cash deposit in U.S. treasury bills of total $112 million. We still expect around 3 years of cash runway. Thank you. Back to you, Tom.

Thanks, Hao-Yuan. To summarize, we had a strong start to the first half of the year and continue to make meaningful strides in advancing Tinlarebant clinical trials for Stargardt disease and geographic atrophy across several countries. We're also proud that we have received Sakigake designation in Japan, which we believe is a testament to the groundbreaking potential of this drug and the unmet - unmet need as there is no currently - no treatment for Stargardt disease. We're also in a strong financial position with $110 million in cash and cash equivalents. As we enter in the second half of the year, we are well positioned to execute on key milestones and look forward to conducting period of Phase 3 interim analysis from our DRAGON study in the fourth quarter. Finally, we look forward to seeing some of you next week at the H.C. Wainwright 4th Annual Ophthalmology Conference and hope you join our presentation on August 15. Please also note that in September, we will also be attending conferences with H.C. Wainwright, Cantor, Deutsche Bank and hope to see some of you there. Thanks again for joining this call, and now we open the call for questions.

Our first question comes from Marc Goodman with Leerink Partners. Marc, your line is now open.

Hi, good afternoon. This is Basma on for Marc. Our question is on DRAGON II. Could you provide color about this trial and what's the goal of this trial exactly does? Statistical significance needs to be achieved with this trial yes or no? And do you think the powering is enough? And do you also need to run another safety, long-term safety trial in these global sites as well for the submission OUS or only the DRAGON II will be enough and safety database in the US will be sufficient? Thank you. That's it.

Nathan, do you want to take this?

Yes, I'm happy to take that, Basma. So thank you for the question. Regarding statistical significance, yes, of course, we'll have to -- we're powering for statistical significance at the 2-year time point, that will have to be achieved in order to essentially meet that requirement for showing efficacy. Conditional power will not be a problem because in this particular study, we're actually randomizing 1:1 versus 2:1 in our original DRAGON study. So in the original DRAGON study drag in the study, there's roughly about 35 placebo subjects and the remainder of the 104 are the Tinlarebant-treated subjects, whereas in the DRAGON II study, there's an equal distribution in the randomization. So that helps the conditional power. And no, I don't believe we'll have to do -- I believe that you asked about an open-label extension study to evaluate safety based upon what the PDMA has told us in Japan is that they would only require the 2-year safety data from the required Japanese subjects that will be enrolled in Dragon II, which is a minimum of 9 Japanese subjects, which is exactly what we're complying with. So I hope that addresses all the questions, Basma?

Yes. Thank you. Very helpful.

Sure.

Our next question comes from Jennifer Kim with Cantor. Jennifer, in is now open.

Jennifer, I think, you are mute.

Oh, hi. Can you hear me now?

Yes.

Okay. Great. Thanks for taking my questions. Maybe to start off with DRAGON 1. With the interim analysis later this year, can you just remind us what you're thinking of in terms of the format of that update and what level of update we - of detail we should expect? And then my second question is maybe following up on the last question on Dragon II. You've completed the Phase Ib portion. Any thoughts on when we could see initial data from this trial? Is it going to be the 2-year data? Or can we see an interim update? Thanks.

Thanks, Jen. So the FDA guidance that we are not allowed to review any data. In fact, I mean, only the DSMB would have that knowledge, simply because the study -- the treatment is ongoing and we don't want to bias the data in any sorts. And the same question, Nathan, do you want to take it?

Regarding Dragon II?

Yes.

Yes. Sorry, Jennifer, what was the portion on Dragon II?

The interim data as well...

Yes. So the Phase Ib -- so they're sort of tied together, right? This is a PK/PD study originally just designed to make sure that the 5 milligram dose that we're using in all of our other trials achieves the same pharmacokinetic and pharmacodynamic response in Japanese subjects, which we have every confidence that it will. We won't be disclosing that data until we get to the end of the efficacy portion of the Phase II/III portion of the study, which, of course, is 2 years later. So we'll allow all that data to be disseminated once we have all of the top line 2-year data, which would, of course, include the PK/PD which basically dose efficacy or dose finding study.

Thanks. That's helpful. If I could squeeze in one more question. Just on PHOENIX, could you confirm the timing of enrollment completion for PHOENIX? And what kind of 1-year interim look should we expect for that trial? Thank you.

Yes. So we're targeting an enrollment size of 429 subjects. We're about at the 200 mark right now. So roughly about halfway in. Based upon our run rate, we expect to close that enrollment by the end of Q1 of 2025. In terms of data coming out of that study, you said the interim data, we haven't really decided what we'll be doing in terms of the interim analysis. In fact, we haven't quite developed or lease completely fleshed out the SAP, the statistical analysis plan for that study. So sort of put a tack on that one, you can get back to us later when we have a little bit more information for you.

Our third question comes from H.C. Wainwright, Yi Chen. Your line is now open

Thank you for taking my questions. My first question is just to confirm that both data from DRAGON I and DRAGON II trial are needed to submit to PMDA under the Sakigake designation correct?

Yes. That's correct.

And the same data set will be submitted to the US and European regulatory agencies as well. But under the Sakigake designation, do you think there's a chance that Japan could approve the drug faster or first before FDA and EMA?

Good question. So based on the information, Japan will want to be the first country to approve this drug. So a lot of discussion still needs to -- we still need to discuss with the PMDA regarding when they want to see the data. So once the study for the DRAGON, we will still need to communicate with the PMDA regarding when would they want to see the DRAGON II database.

Well, Yi Chen, I want to clarify that. So the Japan FDA does not require us to complete the entire DRAGON II for Japan [ph] submission. I think the requirement is that we complete DRAGON I, and we complete those 9 patients from Japan in the DRAGON II, then we can submit. We don't need to wait for the other U.S. and U.K. patients to complete their DRAGON II because the key point for them is that they just want to see how Japanese subjects are doing in this study. So they think DRAGON I is enough, and we just need to -- and we decided to be able to carve out those Japanese data for submission for Japan.

Thank you very much for clarification. And my follow-up question is regarding ABCA4 mutation, is that something being measured in the DRAGON I and DRAGON II trials? And how shall we interpret the Phase II observation regarding the mutation, those 5 patients into our potential projection of the DRAGON I and DRAGON 2 readout?

Nathan?

I'll take that. So of course, we do genetic analysis, genotyping on all subjects. They are required to have both a clinical and molecular confirmation of Stargardt disease. So that will be done regardless of which study we're looking at, we'll do that in all of our Stargardt trials. In terms of the -- sorry, sorry, Yi Chen, I lost track of the other part of your question.

I mean the results you observed from the 5 subjects in the Phase II trial, how should we translate that into interpreting the Phase 3 data?

That's really difficult because I think I mentioned before, there's over 1500 known mutations in Stargardt's disease. It's very difficult to sort of gene match everyone to sort of evaluate, so for instance, in those 5 subjects that never evolved an instant atrophic lesion, they had severe genotypes. But there are other severe genotypes would probably behave similarly, but they would be different, right? Still severe, but none of the same genetic locus. So it's very difficult to sort of take those -- the genotypes from those 5 subjects that didn't basically span any disease and sort of infer what would happen in the larger patient population has different genetic mutations, because again, there's such a varied genotype among these patients. You can see in our cohort just randomly our 13 subjects. They all had severe pathogenic mutation. So our thinking is that if they have a genotype that has a mutation and they have the pathology, this is going to be a severe disease, very quickly progressing disease. And so we're actually having that validation at screening. So we'll know with some measure of confidence whether or not our patients have pathological gene mutations or not. So we're not necessarily trying to match what we saw in those 5 subjects with what is happening in the other DRAGON studies. But it is important to note just generally speaking, those pathologic mutations that lead to disease have been sort of neutralized with our treatment. So we hope to see that, again, of course, with other severe genotypes. That's our hope.

Thank you.

Our next question comes from Bruce Jackson with Benchmark. Bruce, your line is now open.

Hi. Can you hear me okay?

Yes.

Okay. Super. With the presentation of the interim analysis for DRAGON, is that going to coincide with the medical meeting?

I don't believe medical meeting, do you refer our AAO?

Possibly AAO, yes.

Yes. No, no, it's not. So the interim analysis will be somewhere around, I think, end of the year. And -- but that's tricky time during Christmas, so we have to get the DSMB, the timing and them when they can review the data. So it may just run out into January 2025, but we are expecting end of the year to come back then interim analysis. And again, per FDA guidance, we are not allowed to review any data to the public, not to bias with data while the trip [ph] is going.

Okay. Great. And then with the R&D expense, you had the milestone payment this quarter. Can you just remind me what the base rate of R&D is for the next couple of quarters? And how the milestone timing for the remainder of the year and 2025 might play out?

So basically, well, this quarter, we have a higher expense because of that milestone payment. For the rest, I would expect to be probably the same between probably $7 million to $8 million a quarter. So for the entire 2024 is probably will be still around 30 to 35. Next year, 2025, it may be slightly higher, given that we expect some milestones to be achieved from the PHOENIX study. So I would say probably next year, it will be around 35 to 40. So I think for this year, next year until 2026, these three years will be pretty similar in terms of expense, around that 35 each expenses per year given we're running the PHOENIX study for these three years.

Okay, great. And congratulations on all the progress. Thank you.

Thank you so much, Bruce.

Please let me know if there are any additional questions.

No, I don't have any on the written question here.

Great. This concludes our Q&A portion of the call. I will now turn it back to Tom Lin for closing remarks.

Thanks. So thank you, everyone, for attending our earnings call for this quarter. We will certainly update the -- once we have conferences at AAO, we'll be presenting at AAO. And again, we will update all the events that's coming up for the upcoming - as time comes. Thank you, everyone, and we'll keep you updated.