Belite Bio, Inc

Hello, and thank you for joining us to discuss BeLiveBio's first quarter 2024 financial results. Joining the call today are Dr. Tom Lin, Chairman and CEO of BeLiveBio, Dr. Nathan Mata, Chief Scientific Officer, and Haowen Zhang, 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 today by clicking on the Q&A box at the bottom of your screen, and we'll respond to questions following our prepared remarks. Now I'll turn the call over to Dr. Lin.

Thank you, Judy. Thank you, everyone, for joining our reporting for the first quarter. I'm Tom Lin, CEO of Be Like Bio. Joining me is our CSO, Nathan Mata, and CFO, Hao Yuan. I'd like to start off with an overview. Teleriband is a novel once-a-day oral tablet designed to bind to a certain retinal binding protein, known as RPP4, as a means to specifically reduce retinal delivery to the eye. This approach is intended to slow or stop the formation of the toxic retinol derived by products which are generated in the visual cycle and are implicated in progression of Stargardt's disease and geographic atrophy. BeliveBio believes that early intervention directed at emerging retinal pathology, which is not mediated by inflammation, would be the best approach to potentially slow disease progression in Stargardt's disease and GA. There is still a significant unmet need for both indications, as currently there is no approved treatments for Stuller's disease, and there are currently no approved oral treatments for GA. And we are already in global phase three trials for both indications. So far, we have been granted fast-track designation, rare pediatric disease designation, and orphan drug designation in U.S., EU, and Japan. We have several patent families and with composition of matter patents lasting until 2040s. And with patent term extension and new patents to be filed, we will have patent protection past the 2040s. For starter indication, at Arvo last week, we presented further positive findings and treatment results from our end of phase two results, which our CSO will be presenting. The phase three is already fully enrolled. and estimated interim results by end of 2024 or early 2025. We've also initiated a phase two study in , which will recruit Japanese patients that is required for NDA in Japan. For GA, dry AMD indication, we currently have about 100 subjects enrolled in our phase three in GA. And with this, I would like to pass it on to our CSO to give the clinical and scientific update. Nathan?

Thank you, Tom. And thank you, everyone, for attending. What we'd like to share with you today are some new analyses that we have from our open-label phase 2 study in adolescent Stargardt disease. This trial enrolled 13 adolescent Stargardt subjects aged 12 to 18 years of age, and it was a two-year study in which patients took oral 10-larabant 5 milligrams daily. The first analysis I want to show you is a genetic analysis of all of our patients in study. This has not previously been reported. You'll see for each subject there are two entries. That's because there's an allele for each ABCA4 mutation, and so we're looking at two of them. And you can see 11 of 12, 11 of 13 subjects have severe biallelic mutations. This analysis, by the way, was conducted by Dr. Randall Alekmits, who is the identifier of the Stargardt gene, the ABCA4 gene. So the point here is that our cohort is very, very prominently affected with the severe mutations. There are only two subjects, subjects three and five, who have a moderate allele of ABC4 on their mutations. It's important to note the way these genes are classified is through a score called the combined annotated dependent depletion score. In this analysis, scores above 20 are predicted to be among the 1% most deleterious. And you can see all of our subjects, with the exception of those alleles on subjects three and five, have scores well above 20. So this is a very severely affected cohort. The next analysis I'd like to show you is that Of these subjects, five of them, subjects 1, 3, 4, 12, and 13, never spawned in atrophic lesion. This is important because in this particular study, subjects came in with only autofluorescent lesions at baseline. So we were monitoring the transition of the autofluorescent lesion to the atrophic lesion type. And then once the atrophic lesion formed, we measured the growth rate of that lesion. And we previously reported a remarkably reduced growth rate in the overall population. Now we're showing you in those subjects that did not grow in atrophic lesion over 24 months, they had very severe mutations. The absence of lesion growth in these subjects could not be attributed to benign or mild mutations. The next analysis is a very important one because it shows us that Four subjects have the exact same allelic mutation. Subjects nine and 10 are siblings. They are two brothers. You can see there they have the exact same mutations across alleles. And subjects 12, 13 are brother, sister. They also have the very same mutations. The reason this is important is because other investigators have suggested that identical genetic mutations predict identical disease course. I can tell you right now that is not what we're seeing in the phase two study. And I'll show you some of that information right now. Here we have the visual acuity analysis of all subjects before they came in to the study, and they all have different disease duration. What we did is we looked at subjects that had bilateral vision loss, that is vision loss in each eye, over the period of time between the time they were diagnosed and the time before they came into the study. And what we found is there were six subjects who had a very severe bilateral vision loss of 10 letters per eye, a mean annual loss of 10 letters per eye. That's significant because, well, for two reasons. It tells us that autofluorescent lesions in the fovea do, in fact, cause visual acuity loss. Remember, these subjects have not grown atrophic lesions yet. The other thing that's important is if you look at the sibling subjects again, 9, 10, and 12, and 13, you can see their visual acuity loss is very different. Subjects 9 and 10, again, are their two brothers. They have similar disease duration, but subject 10 lost vision, whereas his brother, subject 9, did not. Same thing for subjects 12 and 13. Subject 12 has an eight year of disease duration whereas the sister has only two years, yet the sister with only two year disease duration lost significant letters whereas the brother did not. So these data tell us in fact that identical genetic mutations do not necessarily predict identical disease progression as so far as it pertains to visual acuity loss. This is an analysis of the visual acuity of all subjects on the left-hand side, and then those six subjects shown on the right. I want to first say that in all subjects over 24 months, the mean loss was only five letters. That is, over a whole period of 24 months, all subjects lost only a mean of five letters over 24 months. So that's roughly 2.5 letters per year. Now, if we just focus on the six subjects that came in losing 10 letters per year, we now see over 24 months, they've only lost about 3.8 letters, which is roughly about two letters per year. So we've taken the significant 10 letter per year loss down to two letters per year. That is a substantial preservation of visual acuity in these subjects. The next analysis I'd like to show you is the lesion growth analysis. And it's important to note that these autofluorescent lesions convert to atrophic lesions. And a visual of that is shown on the graphic on the upper right-hand side. This is the actual lesion growth data from Subject 10. You can see at baseline there's this very large autofluorescent lesion, which is referred to as questionably decreased autofluorescence, or QDAF. over time atrophic lesions will be spawned within the autofluorescent lesions. The autofluorescent lesions are referred to as definitely decreased autofluorescent lesions. So here we're looking at the transition of the autofluorescent lesion to the atrophic lesion type, and we're seeing if there's a proportionality between the decrease of the autofluorescence and the increase of the atrophic lesion size. And that graphic is shown on the lower right-hand side. As I said before, 5 of 12 subjects, 42%, never grew an atrophic lesion. We're not including subject 7 in the analysis because that subject was lost to follow-up at month 12. But in those subjects that did grow atrophic lesions, you can see a very clear correlation between the decrease of the autofluorescent area and the increase of the atrophic lesion area, showing that the atrophic lesion does grow certainly within the autofluorescent zone, but nowhere else. So we have confined the lesion growth to within this zone, and we know that lesion growth from prior analyses is significantly lower than in natural history. There's only one subject, subject five, that grew an atrophic lesion outside of the initially identified autofluorescent area. And we believe that could be due to reading error of the image software, which I'll get to right now. We typically use, and most people use, a software called the Heidelberg Region Finding Software. This is a software that accompanies the imaging camera that allows ophthalmologists to grade lesions in the back of the eye, retinal lesions. The problem with this software is that it is affected by human error. So humans have to actually look at the lesions and demarcate the zone of atrophy. And sometimes readers don't agree. So you can have a case where you have a difference of opinion and it has to go to an arbitrator. So this can be a very time consuming and error prone process, but this is what is being used today. Our reading center has developed a new algorithm for reading lesions, which does not rely on subjective reader bias. In fact, it uses a mathematical classification of lesions based on the proximity of the lesion and the density to that of the optic disc, as well as healthy retinal tissue. And it focuses just on the six millimeter macular area, which is the most important to look at when you're talking about visual acuity loss. And as I said before, it's independent of reader bias. So it's looking at just pixel densities across the lesion area. And really all the reader is doing is getting the data back from the software and reporting whatever the computer algorithm provided. So I want to show you a reanalysis of our data using this particular image grading software. What we found, in fact, was that if we looked within the macular area using this new grading algorithm, we find that there's 12 eyes of eight subjects that did in fact have macular lesion involvement at baseline. And we monitored the lesion growth of the macular lesion of the patients over time. And you can see that on the left-hand side. The solid lines and dots show you the actual data from the lesion growth, and the dotted line shows you a third-order polynomial fitted through the data so you can see the trend line. You can see it's very clear. The lesions do grow from baseline out to about month 16, but then starting from month 16 to month 24, there was absolutely no further growth of the lesion. And you can see on the right-hand side the percentage involvement of the atrophy within that 6-millimeter macular zone. The 100% would indicate that all 6 millimeters of that zone are occupied by atrophy. As you can see here, our subjects are showing no more than a 7% encroachment of the atrophic lesion into the macula, and it's static from 16 to 24 months. So we're very pleased to report this data, and it is consistent with the stabilization of visual acuity that I showed you earlier. Finally, I want to talk a little bit about our phase three study in geographic atrophy. It's important to note that the phase three trial design in Stargardt disease and the phase three trial design in Stargardt's are very, very similar. They have the same dose, They have the same duration, two years, with the same interim analysis. There's the same two-to-one randomization. And of course, we're looking at all the same safety and efficacy assessments that we're looking at to judge atrophic lesion growth and adverse events. There's only two differences in these studies. First, the indication, GA instead of Stargardt's. And then secondly, a higher predicted enrollment population for the GA study to reflect the higher prevalence of the disease in the population. So with that, I'll turn it over to Haoyun to discuss financials. Thank you.