Belite Bio Q1 2025 Earnings Call Transcript

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May 14, 2025

There are 11 speakers on the call.

Operator

Hello and thank you for joining us to discuss BeLight Bio's first quarter twenty twenty

Speaker 1

five financial results. Joining the call today are Doctor. Tom Lin, Chairman and CEO of BeLight Bio Doctor. Hendrik Scholl, Chief Medical Officer Doctor. Nathan Mata, Chief Scientific Officer and Haoyang Zhang, Chief Financial Officer.

Speaker 1

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 Doctor. Lin.

Speaker 1

Thank

Speaker 2

you

Speaker 3

for joining today's call to discuss our first quarter twenty twenty five financial results. We continue to make good progress in this quarter towards advancing Tenereband in patients living with Stalin's disease and geographic atrophy. For those who are new to our story, tenerbin is a first in class oral therapy intended to reduce accumulation of toxic vitamin A byproducts, which is 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. To 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 designations in Japan.

Speaker 3

It has also been granted orphan drug designation in The US, Europe and Japan. We believe this speaks to this significant unmet need for both indications as currently there is no approved treatment for Stallard's disease and no approved oral treatment for geographic atrophy. 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've had made. We have two studies underway with tenerbent in patients living with Stargardt disease.

Speaker 3

These are the Phase three DRAGON trial and the Phase twothree DRAGON two trial. As part of the Phase three DRAGON trial, we recently announced that the Data Safety Monitoring Board has completed its interim analysis, which is based on all subjects having completed the one year assessment period. The DSMB recommended that the trial proceed without simple size increase or modifications. So essentially maintain the sample size at 104 subjects. In addition, they recommended that we submit the data for further regulatory review for drug approval.

Speaker 3

With the DSMB's review done, completion of our trial is on track for end of this year. The Dragon two trial continues to progress rapidly. We have enrolled 16 of our targeted enrollment of approximately 60 subjects including about 10 Japanese subjects. Data from a Japanese subject is intended to expedite a new drug application in Japan to which we have already been granted the pioneer drug designation. In GA, we also continue to make progress in our clinical global Phase three PHOENIX trial, which has already enrolled four sixty four subjects to date.

Speaker 3

We expect PHOENIX trial to be fully enrolled for 500 subjects in Q3 this year. To summarize, with the excellent progress in our Phase three trials and promising interim results from Phase three Stargardt study, 02/1935 is off to a great start from a clinical perspective and our balance sheet is also strong with a full year cash runway. We remain well positioned in advancing telnet band as potentially the first oral treatment for people living with degenerative retinal disease. I'd like to now turn over the presentation to Nathan.

Speaker 4

Thank you, Tom. So here I'll talk about the DRAGON clinical trials, both the DRAGON-one and DRAGON-two, as well as the interim analysis from the DRAGON-one study, as Tom just mentioned. Here's an overview of the clinical trial designs in the Phase III trials for DRAGON and DRAGON-two. These trials are designed nearly identically. There's only three differences in the trial designs, and those are highlighted in the top three rows.

Speaker 4

The first being the number of subjects, 104 in DRAGON versus 60 in DRAGON two. The global nature of the Phase three design for Dragon versus a more localized geography for Dragon two in Japan, U. S. And UK. And the randomization is two to one in Dragon versus one to one in Dragon two because of the difference in the sample sizes.

Speaker 4

Other than that, all of the other parameters of the study are identical. And of course, the endpoint is exactly the same, that is slowing the growth of the atrophic lesions as measured by FAF photography. This is the FDA accepted endpoint. The thing I should mention is that these studies both include the same dose, which is a five milligram daily dose that reduces retinal binding protein four to about 80% below the baseline value. And at the very bottom there, can see the key inclusion criteria.

Speaker 4

All subjects in the DRAGON studies are 12 to 20 years old, and they have clinically and medically confirmed diagnosis of Stargardt disease. Importantly, about the DRAGON trial, as Tom mentioned, there was an interim analysis. This was a prespecified sample size re estimation in which the DSMB would take an unmasked look at the study data to determine whether or not there was a trend for efficacy in a so called promising zone. This is a statistically identified window of conditional power that tells us there's a trend for efficacy. If in fact there were a trend for efficacy noted by the DSMB, we would then be allowed to add 30 additional subjects to the study that would preserve and enrich the observation made at the interim so that we would have a better chance of seeing a statistically significant difference at the end of the second year.

Speaker 4

That analysis, the interim analysis, was triggered when the last patient had met his or her twelve month visit. The DSMB then took a look at the data and they decided there was no modification of the study that would be required and that we continue the study without a sample size increase. So that told us that we were not in this conditional zone of power called the promising zone. We were either on the opposite side of that, which would be futile, or we're on the very positive side, which would be the overly efficacious side or unexpected efficacy. And in fact, we knew that we're probably very efficacious because the DSMB did provide an additional comment that they recommend we submit the data for further regulatory review for drug approval.

Speaker 4

That comment would not have been made if in fact we were on the futile side of that promising zone. We feel very optimistic and encouraged about this outcome. Importantly, at the time of the interim analysis, the overall withdrawal rate was less than ten percent. So only ten of a hundred and four subjects withdrew. Of course, we don't know the breakout between placebo and active treatment because, again, this is blended data.

Speaker 4

But to have less than a ten percent dropout when the majority of the study data has been analyzed is quite significant. And importantly, the withdrawal due to ocular adverse events was only three point eight percent. So only four of a hundred and four subjects withdrawing because of ocular AEs. This is particularly important because the nature of our MOA would predict that there would be ocular adverse events. We know now from these study data that is very well tolerated and, of course, of mild and transient.

Speaker 4

That's very important. And then finally, when we look at visual acuity, find that visual acuity was stabilized in a majority of subjects with a mean change from baseline of less than three letters under both standard and low luminous throughout the two year study. Here's the breakdown of the treatment emergent adverse events in the DRAGON trial. It's important to note that systemically there was only one drug related adverse event and that was acne. And this can happen in teens and preteens when vitamin a is diminished in the skin because vitamin a does help clean pores.

Speaker 4

So if we're diminishing it, perhaps pores are not so clean, but it's a very mild AE to have systemically. Other than that, clinically significant findings in relation to vital signs were nothing in relation to physical exams, cardiac health, or organ function. What you see on the table are the outcomes from the two ocular AEs that we expected, xanthopsia and delayed dark adaptation. As mentioned, these were reported as mild and, of course, transient. You can see on the right hand side the frequency and number of patients presenting with those AEs.

Speaker 4

The night vision impairment you see there, which is also mild, is a more severe exacerbation of delayed dark adaptation. We had that in fifteen reports of it. And then, of course, we have a non ocular headache in some subjects, which, of course, can be manifest when subjects strain to use their visual acuity while experiencing these ocular AEs. But overall, a very, very well tolerated and safe profile from an adverse event perspective. With respect to the visual acuity, I mentioned it was stabilization throughout the study trial.

Speaker 4

Since we have our CMO, doctor Hendrik Scholl, on the line, I'd like to get his clinical opinion on the BCVA data. Doctor Scholl?

Speaker 5

Thank you, Nathan. The chart shows the EDTRS score in the study eye in orange and the fellow eye in gray over twenty four months. This is blended data, meaning the telariband group and the placebo group are shown as one group. The early treatment diabetic retinopathy study, or briefly ETDRS visual acuity testing, is a standardized method used to measure visual acuity and is the gold standard in clinical trials and research settings. The EDTRS chart includes 14 lines with five letters per line and the maximum score on EDTRS visual acuity testing is 100.

Speaker 5

There is significant intercession variability of EDTRS visual acuity measurements in normal subjects, test retest variability typically falls within about five letters. And in individuals with macular degeneration, variability tends to be greater, namely about eight letters. What we see on the chart is that there was not even a single letter loss on average. Clearly, this allows to conclude that there was no significant loss of visual acuity, and it is fair to say there was stabilization of visual acuity in the DRAGON trial. I hand back over to Nathan.

Speaker 4

Thank you, Hendrik. So moving forward now, a few words on our Phase III trial in geographic atrophy, which is called PHOENIX. This is the overview of the trial design of PHOENIX. You can see on the right hand side the various criteria for the study. This study is enrolling up to 500 subjects.

Speaker 4

It's a global study. The design is essentially the same as that for the Stargardt trials. In fact, the dose is exactly the same, five milligrams daily. It produces the same pharmacodynamic effect in older healthy adults as it does in young adolescent children, so we can use the same dose. It has the same endpoint.

Speaker 4

There's going to be the same trial duration. We will also be including an interim analysis. The timing and specifications of that analysis have not been worked out, but that will be conducted. And of course, that trial is still enrolling. We expect to close that enrollment sometime this summer, as again, as I said, up to 500 subjects.

Speaker 4

So with that, I'll turn it over to Hao Yen for the financial results.

Speaker 6

Thank you, Nathan. For Q1 twenty twenty five, we had R and D expenses of $9,400,000 compared to $6,800,000 for the same period last year. The increase was primarily attributable to the share based compensation and the study higher clinical trial expenses related to the PHOENIX trial. Regarding G and A expenses, it was 6,100,000 compared to $1,600,000 for the same period last The increase was also due to share based compensation grade. Overall, we had a net loss of $14,300,000 compared to a net loss of $7,900,000 for the same period last year.

Speaker 6

One thing to note is that as the majority of the increase of the expenses came from the share based compensation, which was about $6,700,000 and was not cash related, the operating cash outflow was only about $8,300,000 As we raised $15,000,000 through the registered direct offering in February and received about $5,600,000 from employee stock option exercise, we had a cash increase of $12,300,000 for the quarter and leave us with $157,400,000 in cash, liquidity funds, time deposit and U. S. Treasury bills as end of Q1. We still expect four years of cash runway and we expect to be able to complete all of our current clinical trials with this current cash. Thank you.

Speaker 6

Back to you operator.

Operator

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

Speaker 2

This is Basma on for Mark. Thank you for taking our question. Could you please our first question is about the PHOENIX trial. Could you provide us a data about the discontinuation rates and how the enrollment is ongoing in this trial? And the second question we have, it's about any update on the regulatory meetings for the endpoints for, I'm sorry, for the trial requirements for, for Stargardt disease.

Speaker 2

You mentioned that you were supposed to we're working on you're planning to meet up with us, the FDA, and other regulatory agencies, to finalize the plan, the development plan. That's it for us. Thank you so much.

Speaker 3

Well, thank you. So I'll I'll take those questions. So the first question is regarding the Phoenix dropout rates. Is that right?

Speaker 2

Yes. Correct.

Speaker 3

So, the dropout rate is approximately around about twenty percent. This is way below, what's been reported in previous studies, such as duravelumab vilin a phase three, that was reported, I think, earlier this year. There's a dropout of about thirty percent, and I believe that in mixtecet and other anti complement inhibitors in GA, they were much, much more, over thirty to fifty percent, I believe. Henrik, could you shed some light on that?

Speaker 5

Yes. That is correct. I mean, it's not surprising that the injectables come with a higher risk and a higher dropout rate, and amixostat led to severe, night blindness, and there was also a higher, dropout rate in, in the trial using amixostat as a visual cycle modulator.

Speaker 3

Yep. Yep. So what's what's most surprising is that even the deuterated vitamin a had a more than thirty percent dropout rate, so we're way below that. So that's pretty encouraging considering that we're conducting the trial in very elderly patients. I think the average is around about 80 year old.

Speaker 3

So, compliance is is an issue, but we still have it within that twenty percent. So so we're doing pretty well. And then the second question was the Stargardt IA that we'll be discussing that with regulators. Is that correct, if I remember correctly?

Speaker 2

Yes. Correct.

Speaker 3

Yeah. So so we we have we have been scheduling meetings with with the regulators, and we are doing so right now. We will be meeting a few of those in we've scheduled some meetings in in the short term. We'll be updating, that as we go along. So right now, we don't have much to report back on.

Speaker 2

Got it. Thank you so much.

Operator

Your next question comes from the line of Jennifer Kim with Cantor. Your line is open. Please go ahead.

Speaker 7

Hi, thanks for taking my questions. Maybe to start off in Stargardt, can you just talk about the recent interactions you've had with the FDA and any thoughts on maybe any perceived regulatory risk given the changes at the agency? And then my second question is, with the Phase III data, I think it's coming in early twenty twenty six, can you just remind us what the goalpost is on efficacy and safety?

Speaker 3

So I'll take the first one. So we haven't met up with the FDA yet. We'll be meeting up with them soon, that that's been scheduled. And then what's the other

Speaker 7

question? If you have any thoughts on, I guess, regulatory risk, given specifically the the changes that have been going on at the agency?

Speaker 3

No. I I think we don't have, any risk given that, you know, the data speaks for itself. So even I mean, even though there's there's some changes within the FDA, I don't think that's gonna affect us, especially, I think, the division that that we've met up with. Well, the the the division that that's been, giving us guidance throughout the whole study, especially during the phase three, most of them are still there. So I don't think there's any issues with the, personnel change within the FDA.

Speaker 7

Okay. And then can I ask on the goalpost for the phase three the twenty four month phase three data?

Speaker 3

Oh, I'll tell you what, for for the Dragon. I think it will complete by q three this year. Nathan, do you wanna shed some light on that to confirm?

Speaker 4

Yeah. I think Jennifer may be asking about what exactly we're we're looking for in terms of efficacy and safety. Correct, Jennifer?

Speaker 3

Oh, okay. Yeah. So, Nathan, you wanna answer this then?

Speaker 4

I was just gonna mention that this study is powered to detect a thirty five percent treatment effect between placebo and active. So that's what we're looking for. I mean, you know, sometimes you get there, sometimes you don't. But based upon what the DSMB looked at and and told us in terms of safety and efficacy at the interim, which, by the way, as I mentioned, the majority of data were actually available at that time, we're expecting that we'll be getting very close to our anticipated treatment effect size of thirty to thirty five percent. Again, that's what the study is powered for.

Speaker 4

In terms of safety, I think the, outlook is very positive given, again, what we see at the interim, less than a ten percent overall withdrawal rate and three point eight percent with respect to ocular AEs. Again, because the majority of the study data had already been evaluated by the time of the interim analysis, roughly 70 to 75% of the data was evaluated. I don't think that's gonna change very much by the end of the year, so I expect those dropout rates to be fairly consistent. And as I said before, you never know about efficacy, but based upon what the DSMB saw at the interim, I'm expecting that we'll get very close to our anticipated treatment effect size.

Speaker 7

Got it. That's helpful. Thanks, guys.

Operator

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

Speaker 8

Hi. Thank you for taking my questions. In view of president Trump's recent policy of most favored nation drug pricing, could you comment on your potential strategy strategy regarding approval on launching of the drug in ex US territories, particularly Japan, and whether that will affect your US drug pricing and market prospects? Thank you.

Speaker 3

Yes. Thanks for that. Well, first of all, since Tenerevan has not launched yet, it's not gonna affect us immediately. We are still observing, how this is going to impact the industry as a whole, and then what type of drugs are affected, orphan drugs, rare pediatric drugs, etcetera. Right now, we are still monitoring what's going on, so we don't have a immediate answer for that.

Speaker 3

I don't think anyone has immediate answer for that, but we're still monitoring how it goes.

Speaker 8

Got it. Thank you. And a quick question on the financial side. Will the operating expenses continue to rise during the remainder of 2025 based on the level recorded in the first quarter?

Speaker 3

Yes. Hal, take this?

Speaker 6

Yes. I think it will be about, slightly higher than the q one. We did, I think we did guide the market at this year and the next year. We expect it to be higher expenses given that most of the milestone of the studies, all three studies expect to be reached in the next, one, two years. Yeah.

Speaker 6

So the expenses will be higher this and next year, but, moving forward, you will come down, back to the previous level.

Speaker 8

Okay. Thank you.

Speaker 6

Thank you.

Operator

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

Speaker 9

Hi. Good afternoon, and thanks for taking my questions. Can you hear me okay? Okay. Super.

Speaker 3

Perfect. Yep.

Speaker 9

Just to follow-up on that last question. We had the increase in stock comp during the quarter. Is that also expected to be a little bit higher going forward?

Speaker 6

Well, so, it will really depends on the allocation, of the of the expenses. So, you know, you will have part of our, ESOP will be only vested based on so called, the involvement milestone. So it is a little bit hard to say what exactly when it will be it will be allocated at. And for the those that are gonna be best based on time, it will be allocated, you know, by by the by the period of the duration of the option. So it's a little bit hard to really, you know, give you a figure.

Speaker 6

But I think, given, we did have some of the ease up that is happening that quarter, so, I don't expect it's gonna be that high, moving forward, this year.

Speaker 9

Okay. Great. And then last question for me is on manufacturing. Obviously, there's a push to move manufacturing to The United States for pharmaceuticals. How do you, how are you set up right now in terms of inventory, and how is your supply chain structured geographically?

Speaker 3

Oh, yep. Good question. So tenerbent is manufactured in The US, as well as in other geographies, so, tariffs is not going to affect us any any way.

Speaker 9

Okay. Super. Alright. Thank you very much.

Speaker 3

Thank you. Your

Operator

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

Speaker 10

Thank you. Thanks for taking my questions today, guys. So I I guess the first question I would like to ask is just if you could provide any more detail on the timing or any of the conditions that you would need to meet for that interim in g geographic atrophy.

Speaker 3

So we believe that we've, so speaking on behalf of the MAS team, and it's probably just me and Henrik, we are meeting with the regulators. We believe that what we got what it takes to get us over the line. But until we've made up having a official response from them, we're not gonna disclose anything as of now.

Speaker 10

Alright. Fair enough. And then are are there any additional sample size reestimations for the, for the PHOENIX study, or is that 500 patient count final as it looks like we're gonna wrap up enrollment in the next couple of months?

Speaker 3

Yep. So we're expecting to wrap up enrollment because, the enrollment is going very smoothly as of, now, we're making good progress. We don't believe that we would need, extra additional subjects. We're just editing it on, just in case because we are, having smooth enrollments, then why not just enroll more that to we enroll more subjects to boost up our success. Nathan, do we have anything to add on?

Speaker 4

No. No. In fact, right now, there's no plan to do a sample size re estimation in Phoenix. As Tom said, we're sort of hedging our bets now that enrollment is going smoothly to enroll as many subjects as possible at the very start so that we won't have to do a sample size re estimation at the interim.

Speaker 10

Alright. Thank you very much.

Speaker 4

Thank you, Michael.

Operator

There are no further questions, so this concludes today's call. Thank you for

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