Belite Bio, Inc Price & Performance (BLTE)

Thank you for joining today's call to discuss our fourth quarter and full year 2024 financial results. 2024 was an exciting year for Belite. As we continue to make strong progress towards advancing tenerbent in patients living with Stargardt's disease and geographic atrophy. For those who are new to our story, Tenereben is a first in class oral therapy intended to reduce the accumulation of toxic vitamin a byproducts, which have been implicated in the progression of retinal lesions in patients with Stalin's disease and geographic atrophy. We believe this approach will be effective in slowing or halting lesion growth. Which would ultimately preserve vision. It is important to note that our approach focuses on early intervention of emerging retinal pathology that is not mediated by inflammation. We believe that this may be the best approach to potentially slow the progression of stava in GA. Give you some perspective on the importance of this potential therapy, Tenereben has been granted rare pediatric disease and fast track designations in The US and pioneer drug designation in Japan. It has also been granted orphan drug designation in The US, Europe, and Japan. We believe this speaks to the significant unmet need for both indications.

There's currently there is no approved treatment for Stalin's disease and no approved oral treatment for GA. And more importantly, we are uniquely positioned as we are already in global phase three trials for both indications. So with that, let me provide a high level overview of the recent progress we have made. Have two studies on the underway with teneleven in patients living with Stagler's disease. These are the phase three Dragon trial and the phase two three Dragon two trial. As part of the Phase III DRAGON trial, we recently announced that the Data Safety Monitoring Board has completed its interim analysis is based on all subjects having completed the one year assessment period. The DSMB recommended that the trial proceed without sample size increase or modifications. So, essentially, maintaining the sample size at hundred and four subjects. Addition, they recommend we submit the data for further regulatory review for drug approval. With the DSMB's review done, completion of the trial is on track for end of this year. The Dragon two trial continues to progress rapidly. We have enrolled 11 of our targeted enrollment of approximately 60 subjects including about 10 Japanese subjects. Data from the Japanese subject is intended to expedite a new drug application in Japan.

Which we have already been granted a pioneer drug designation. In GA, also continue to progress in our global phase three PHOENIX trial which has already enrolled over 400 subjects to date. We expect to increase this number of subjects to be enrolled in trial from approximately 430 subjects to 500 subjects. As we have been making good progress on our subject enrollment. To summarize, the excellent progress in our phase three trials and the promising interim results from phase three SATA study and a four year cash runway, we we remain well positioned in advancing Tendera band as potentially the first oral treatment for people living with degenerative retinal diseases. I'll now turn over the presentation to Nathan. Nathan? Please. Thank you, Tom. Here, we have an overview of our trial designs in Stargardt disease. As Tom mentioned, there are two Phase III trials that we're currently involved in. The first is called Dragon. It's a hundred and four subjects in that trial. The other trial is called Dragon two. There are 60 subjects in that trial. Can see the first, three rows here. This is the areas where these two trials are different. Otherwise, the trials are designed identically.

So there's a difference in the number of sample sizes I just said. A difference in the geography, the Dragon is a global study. Dragon two is focused on geographies in Japan, US, and UK. DRIGON-one study because of the larger sample size is a two:one randomization favoring tinlerovan, whereas the DRAGON-two trial has a one:one randomization with its 60 subjects. Otherwise, the trials are designed identically. And it's important to note that the endpoint for drug approval in Stargardt disease and GA is slowing the growth of atrophic lesions. And at the bottom, can see the key inclusion criteria for subjects involved in DRAGON and DRAGON two.

Here you see the demographics and baseline characteristics for the adolescent subjects involved in the DRAGON-one trial. As I mentioned, there were 104 subjects You can see the mean age is 15.4 years. So these are school age children. They have the average height and weight of children that age. On the right hand side, you see the breakout for male and female. Roughly 60% male and 40% female in the study population. Just below that, you see the race distribution heavily favored towards the Asian population we did heavily heavily recruit in China. So we have approximately 56% of our Asian population representing the study. About 37% being Caucasian and European and North American, and various other categories, approximately seven to 8%. Here's an overview of the interim analysis conclusions As Tom mentioned, the study DRAGON-one included a sample size estimation in which if the DSMB saw a trend towards efficacy at the middle of the study, then they would allow us to include up to additional 30 more patients to maintain that trend towards the end of study so that we could ensure a statistical significant difference by end of study. But in fact, the DSMB looked at the interim analysis, they felt there was no modification of the study required and that we should continue this study without a sample size increase.

I should remind you that the dosage that these children were getting in the DRAGON one and DRAGON two studies is five milligrams daily. This dose has been very well tolerated and deemed safe, a very very nice safety profile. Also important to note that at the time of the interim analysis, where approximately half of the subjects had already completed two years of dosing, The withdrawal rate was nine point six percent, which is of one hundred and four subjects. And the withdrawal rate due to ocular adverse events was only three point eight percent, that's four of one hundred and four subjects. Visual acuity was stabilized in majority of subjects with a mean change of baseline of less than three letters, so very well stabilized under both standard and low luminance throughout the two year study. But perhaps the most important, finding that the, DSMB provided for us was what's provided at the bottom there, comments, as Tom mentioned, they recommended us to submit the data for further regulatory review for drug approval, indicating perhaps an efficacy signal. And here are the safety data from the DRAGON-one trial. These are the treatment emergent adverse events. We fully anticipate to see two adverse events in terms of the drug related ocular event One is a form of chromatopsia called xanthopsia.

This is a yellow hue of color which appears in the visual field Typically upon waking, when light essentially drives this this this visual AE. This is a transient AE. It's last seconds to minutes. And no one dropped out of study because of from atopsia or xanthopsia. Delayed documentation is the other ocular e that we anticipate based upon the mechanism of Tenelera event action. This is the opposite of chromatopsia in which going into darkness, patients have a longer time to accommodate to dim light settings This can last two to three times longer than normal, perhaps somewhere between sixteen to twenty minutes. Again, it's reported as mild. It's transient. And this is not synonymous with night blindness or nyctilopia because these subjects will eventually get back their dark adapted sensitivity. And you can see the distribution on the right hand side in terms of the number of subjects and percentage in the patient population. The night vision impairment is a more severe exacerbation of delayed dark adaptation in which the delay may be longer than twenty minutes. You can see that occurred in fifteen subjects, approximately fourteen percent of the population. Headache was another AE that we found in approximately seven to eight percent of the population This can happen when subjects strain to use their visual acuity while experiencing these these AEs.

But importantly, there was no clinically significant findings in relation to vital signs to exams, cardiac health, or organ function, and the only systemic drug related AE was acne, which teenage kids can be prone to, especially when there's less vitamin a in the skin. But otherwise, an overall very, very well acceptable safety profile. So here we see the visual acuity data from the Dragon one study. This is the two year data. Looking at a visual acuity under both standard and low luminance. We see overall stabilized visual acuity. But for a clinical perspective, let's bring in CMO, doctor Hendrik Scholl, for his opinion. Hendrik?

Thank you, Nathan. When considering the clinical relevance of the finding, we have to take into account that when we measure visual acuity, the intersession variability in normal subjects is two letters on an EDTRS chart. In patients with macular degeneration, it's up to five letters. And that means that the variability that we see on the left for best corrected visual acuity and on the right for low illuminance visual acuity is within the standard variability that we find in such patients. Still, when we look at the left and the development of best corrected visual acuity and knowing the two thirds of the subjects are under tilarybent treatment is very reassuring that was essentially no loss at all of best practice visual acuity letters on a standard EDTRS chart. So with that, I hand it over back to Nathan.

Thank you, Andrew. Here is our overview of the trial design in geographic atrophy. This is our phase three trial called PHOENIX. As Tom mentioned, we're we're gonna recruit up to approximately 400 subjects Right now to date, we're right at about four hundreds. We got about a hundred more to go. We expect to close that enrollment by end of Q2 of this year. This, of course, is a global study, double blind. Same randomization as we had in DRAGON-one, two to one favoring to larabet, It's a two years treatment duration. And of course, just like in Stargardt's, we're looking for the slowing of atrophic lesion growth as the primary endpoint for drug approval. But, of course, we're also looking at BCVA, retinal anatomy by STOCT, and retinal sensitivity by microperimetry. And like the Stargardt Dragon one study, we will have an interim analysis at one year. With that, I think I'll throw it to Hao Yen for the financial results. Thank you.

Thank you, Nathan.

In 2024, we had R and D expenses of 29,900,000.0 compared to $28,800,000 in 2023. The increase in R and D expenses was primarily due to an increase in royalty payments the completion of the Phase two trial and an increase in share based compensation granted in the Q3 of twenty twenty four. On G and A expenses, in 2024, G and A expenses were 10,100,000.0 compared to $6,800,000 in 2023. The increase was primarily driven by an increase in share based compensation granted in the Q3 of twenty twenty four. On net loss, we had a net loss of $36,100,000 in 2024 compared to 31,600,000.0 in 2023. In terms of cash, we have $31,700,000 in cash and $113,500,000 in investment, end of twenty twenty four. As compared with 88,200,000.0 by end of twenty twenty three. The investments were in liquidity fund in time deposit and US treasury bills. One thing to know is that the net cash outflow for operating activities was 29,200,000.0 in 2024. Similar to the cash outflow of $29,800,000 in 2023. We also raised $15,000,000 in gross profit [indiscernible] In the registered direct offering in February 2025. Still expect four years cash runway without considering the cost front, second GA Phase three study.

Thank you. Back to you, operator.

We will now begin the question and answer session. Please limit yourself to one question and one follow-up. 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. Your first question comes from the line of Mark Goodman with Leerink. Your line is open. Please go ahead.

Moving along.

Your next question comes from the line of Jennifer Kim with Cantor. Your line is open. Please go ahead. Hi, thanks for taking my questions and congrats on the progress.

Maybe on my first question, starting with Stargardt. So on the DSMB's recent recommendation, you've said that you plan to reach out and I guess seek harmonization across some ex U. S. Regulatory authorities. Could you outline the potential outcomes from those interactions, either positive or negative? Thanks, Jennifer. I could take that.

So the DSMB, has recommended, the interim results be reviewed by regulatory agencies for drug approval, as you pointed out there. We believe this is very positive outcome, and, we'll be following DSL DSMB's recommendations to request, regulatory review and see whether [indiscernible] So we believe that certainly believe that the regulatory agencies will probably align with the DSMB recommendation because it's not every day that, they see they make this kind of recommendations during interim. But if not the regular if not they don't see it that way, then we'll just move on and carry carry on with the study.

K. And then maybe turning to 500 patients?

Well, we're we're, if you getting so this GA study has been, moving on very smoothly. We want to take this opportunity to enroll more subjects to further boost our chances of success. So based on this current enrollment rate, we should still be able to complete within our expected timeline, which is q three, this year. And the cost will still remain very feasible, based on the current recruitment rate.

Okay. If I could squeeze one more question with GA. Can you just remind me, what is your latest thinking on what the interim analysis will entail and how that sort of feeds into the decision to start a second trial.

You mean for the PHOENIX?

Interim?

For PHOENIX. Yeah.

Well, we certainly believe that [indiscernible] if we get any positive signals from the IF or PHOENIX, then we would speed up, and expedite our our second phase, second phase three trial for PHOENIX. So so for GA. Sorry.

Thank you. Your next question comes from the line of Mark Goodman with Leerink. Mark, as a reminder, please unmute yourself. Your line is now open. Please go ahead.

Hi, good afternoon. Can you hear me okay now?

Yes.

Okay. Hi, this is Basma on for Mark. Thank you for taking our questions. Our first question is about Stargardt disease.

You gonna be able when you meet with the authorities to regarding regulatory clarity for the submission, will you be able to also get confirmation regarding the potential for a broad label, given that the study population so far is only adolescents? And our second question, again, could you give us an update about the current discontinuation rates in the GA trial, the PHOENIX trial? Thank you.

Sure.

So I'll take the second question. And I'll leave the first one for Henrik. So, the dropout rate for Phoenix right now is approximately about twenty percent. It is it is with you it is very common, because majority of the subjects enrolled in in the GA trial are elderly population. So for previous studies, we've we've seen a dropout rate of about more than three percent. In fact, the dual rate of vitamin a study that was just recently, presented, at JPMorgan, The dropout rate is, more than thirty percent and certainly more for it makes myxistat and and the anti complement study. And so, I'll let Hendrik, I'll let Henrik to to confirm if we for the dropout rates for the for the beta complement studies and as well as answer your questions on the start up or label.

Henrik?

Yeah. And I'm I'm happy to. Thank you, Tom. So what we have seen in natural history studies is that in patients that have an early onset of disease, the disease is generally more severe. And shows a faster progression. The PROXA study has shown that subjects with younger age and early onset still show a relatively similar progression rate compared to other subjects that were older in the PROXA study. Not very old, obviously, but still is still a juvenile macular dystrophy. But would be adults. So we believe that the threshold actually, to get something approved for a pediatric population would be much much higher. And we feel that if we can show efficacy in a adolescent population, it should be relatively straightforward to get the drug approved for adults as well.

And the Henry, can you also mention about the dropout rate for, the and that complements as well as for the mixer set? So in in the end, yeah, in the anti complement studies, dropout rate was in the order of magnitude to twenty to thirty percent. I believe in, in the study with a mixed ostat, which, has quite extensive side effects. The dropout rate was was even higher I would actually hand over to to take the lead Martha, who actually knows a lot about that specific drug and its effect in geographic atrophy.

Yeah. It was a little over forty percent.

Yeah.

Thank you.

That's thank you. That's very helpful.

Your next question comes from the line of Yi Chen with HCW. Your line is open. Please go ahead.

Just to clarify, the adolescent drug disease patients enrolled currently into the Dragon trial, What, they represent what percentage of the Struggle disease patients diagnosed in the real world.

And, Rick, I believe there's a question for you as well.

I'd be happy to take that question. So the typical start up patient would would notice first symptoms in the second decade of life. We we see patients that have a very early onset as early as five years, and then they are patients that are later in adulthood develop the first symptoms. But as Carl Stargardt described the disease in nineteen o nine, right, This is a juvenile macular dystrophy, and it typically starts between 10 and 20 years of age with first symptoms. Given that our study population actually includes subjects between 12 and 20 years old. We feel that this is very representative and would show an overlap if we look at all Stargardt patients that that, that that would schedule a visit in clinic, I I would I would believe that that would represent two thirds of start up patients that I would see, for example, in my clinic.

So you would expect potential approval in the future for all Stuttgart disease patients in that age range, right? Not necessarily meeting those meeting the enrollment criteria in the current trial, correct?

So if I understood the the question correctly, the question is if we show a fixed The future the future prescription label is not restricted to the patient groups you are currently enrolling into a pivotal trial.

Exactly. The answer is no. And there would be no reason why he would not prescribe the drug to a, let's say, an adult patient that that developed the first symptoms at age 30. And and and and still shows progression of these DDF lesions, which is typical also for adult patients.

But do you expect any potential limitation on the payer side for reimbursement if the label is broader than the patients in currently enrolled in the drug and trial. I think we will definitely talk to the regulator [indiscernible] try to get the the label for the dials, which we think is doable. And I would probably just do a PK study to prove that it's it works the same on the on the Dallas patients.

Okay. Thank you.

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

Hi. Good afternoon. Thanks for taking the questions. First, a housekeeping question about the the capital raise you did for 15,000,000.

Have has anyone exercised the warrants yet attached to that?

Not yet.

Okay.

And then in February, you said that the your CRO is gonna be handling some of the regulatory process for you.

With the the data for Dragon. So could you just give us a little bit of color on where they are with that process right now, what the next step might be?

Yeah. Thanks thanks, Bruce. So so we have two or three different CRs representing us, for different, jurisdictions. And, certainly, they have a a procedure different procedure or or and and certainly different templates for, submitting these kinds of, regulatory submissions. So right now, it's in in in good hands, and and they are basically submitting as we speak.

Okay.

Alright. Great. That's it for me. Thank you.

Your next question comes from the line of Michael Okunowicz with Maxim. Your line is open. Please go ahead.

Hi, guys. Thanks for taking the questions.

Just first, what kind of difference in lesion growth between placebo and treatment is is the Dragon study powered for?

Nathan, do wanna answer this one?

Yeah. It's powered for a forty percent treat forty percent treatment effect with 80% power to detect that that effect at at the second year.

Okay. Great. Thank you, and congrats on all the progress. Thank you.

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