Q1 2025 Belite Bio Inc Earnings Call
Ladies and gentlemen, thank you for joining us and welcome to the be light bio first quarter 2025 earnings call.
After today's prepared remarks, we will host a question and answer session. If you would like to ask a question. Please raise your hand, if you have dialed in to today's call. Please press star nine to raise your hand and star sixth on mute.
Speaker Change: I will now hand, the conference over to Julie Fallon. Please go ahead.
Julie Fallon: Hello, and thank you for joining us to discuss <unk> first quarter 2025 financial results joining.
Dr Tomlin: Joining the call today are Dr Tomlin, Chairman and CEO would be like bio Dr. Hendrick Shaw Chief Medical Officer.
Speaker Change: Dr Leath and matter Chief Scientific officer.
Speaker Change: And how young Zhang Chief Financial Officer.
Speaker Change: Before we begin let me point out that we will be making forward looking statements that are based on our current expectations and beliefs.
Speaker Change: These statements are subject to certain risks and uncertainties and actual results may differ materially.
Speaker Change: Encourage you to consult the risk factors discussed in our SEC filings for additional detail.
Now I'll turn the call over to Dr. Lynn.
Dr. Lynn: Thank you for joining today's call to discuss our first quarter 2025 financial results. We continued to make good progress in this quarter towards advancing to narrowband and patients living with startups disease in geographic atrophy for.
Dr. Lynn: So those who are new to our story to lab and ease of fluids in class oral therapy intended to reduce the accumulation of toxic vitamin a byproduct, which is implicated in the progression of <unk> in patients with established disease in geographic atrophy. We believe this approach will be effective in slowing or halting lesion growth.
Dr. Lynn: Which would ultimately preserve vision to.
Dr. Lynn: To give you some perspective on the quarters of this potential therapy to lab and has been granted rare pediatric disease and fast track designations in the U S and pioneer drug designations in Japan. It has also been granted orphan drug designation in the U S Europe and Japan.
Dr. Lynn: We believe this speaks to this significant unmet need for both indications is currently days no approved treatment for <unk> disease, and not approved oral treatment for geographic atrophy.
Dr. Lynn: And more importantly, we are uniquely positioned as we are already in a global phase III trials for both indications.
Dr. Lynn: So with that let me provide a high level overview of the recent progress we've had great.
Dr. Lynn: We have two studies under way within their brand in patients living with targets disease. These are the phase III <unk> trial and the phase two three creative to trial.
Dr. Lynn: As part of the Phase III, Greg and <unk>, 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.
Dr. Lynn: D F N b recommended that the crowd proceed without sympathize increase or modifications. So essentially maintained your sample size 804 subjects.
Dr. Lynn: In addition, they recommended that we submit that data for further regulatory review for drug approval.
Dr. Lynn: With the Dsw's review done completion of the trial is on track for end of this year.
Dr. Lynn: The Dragon two trial continues to progress rapidly we have enrolled 16 of our positive enrolment of approximately 60 subjects, including about 10 Japanese subjects.
Dr. Lynn: Data from the Japanese subjects is intended to expedite a new drug application in Japan to which we have already been granted upon the objectives emission.
Dr. Lynn: N G E. We also continue to make progress in our clinical global Phase III Phoenix trial.
Dr. Lynn: Which has already enrolled 464 subjects to date, we expect Phoenix trial to be fully enrolled 500 subjects in Q3 this year.
Dr. Lynn: To summarize with the excellent progress in our phase III trials and promising interim results from phase II <unk> study <unk>.
Dr. Lynn: <unk> five 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 eventing to lab and as potentially the first of all the treatment for people living with deteriorating retinal disease.
Dr. Lynn: Did now turn over the presentation to Nathan.
Nathan: Thank you Tom.
Nathan: So here I'll talk about the Dragon clinical trials, both are dragging one and dragged into as well as the interim analysis from the Dragon. One study as Tom just mentioned here is an overview of the clinical trial designs as the phase III trials for Dragon and dragging to these trials are designed nearly identical theres only three differences in the trial designs and those are highlighted in the top three rows.
Nathan: The first being the number of subjects 104 in Dragon versus 60 in Dragon to the global nature of the phase III design for Dragon a versus a more localized geography for Dragon two in Japan U S and U K and the randomization is two to one in dragon versus one to one and dragged into because of the difference in the sample sizes other than that all of the.
Nathan: Other parameters of the study identical and of course, the endpoint is exactly the same that is slowing the growth of the atrophic lesions as measured by FAA photography. This is the FDA accepted endpoint. The other 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%.
Nathan: Below the baseline value and at the very bottom that you can see the key inclusion criteria all subject to the Dragon studies are 12 to 20 years old and they have clinically and locally confirmed diagnosis of starboard disease importantly, about the Dragon trial as Tom mentioned, there was an interim analysis. This was a pre specified sample size re estimation in which.
Speaker Change: The DSM B would take an unmatched look at this study data to determine whether or not there was a trend for efficacy in a so-called promising. So 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 D. S. M. B. We would then be allowed to add 30 additional <unk>.
Speaker Change: 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 Change: That analysis the interim analysis was triggered when the last patient had met his or her 12 months visit the.
Speaker Change: The DSM V. Then took a look at the data and they decided there was no modification of the study that would be required and that will continue the study without a sample size increase so that told us that we were not in this conditional zone of power called a promising zone, we were either on the opposite side of that which would be futile or you're on the very positive side, which would be the overly efficacious.
Speaker Change: Cider unexpected efficacy and in fact, we knew that were probably very efficacious because of DSM. We did provide additional comment that they recommend we submit the data for further regulatory review for drug approval that comment would not have been made if in fact, we were on a futile side of that promising results, we feel very optimistic and encouraged about this outcome.
Speaker Change: Fortunately at the time of the interim analysis. The overall withdrawal rate was less than 10% only 10 of 104 subjects withdrew of course, we don't know the breakout between placebo and active treatment. Because again. This is blended data but to have less than a 10% drop out when the majority of the study data has been analyzed is quite significant and importantly, there was drawn.
Speaker Change: Due to ocular adverse events was only three 8%. So only four of 104 subjects withdrawing because of Aqua. He's this is particularly important because the nature of our MAA 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 that's very important.
Speaker Change: And then finally, when we look at visual acuity, we find that visual acuity was stabilized and a majority of subjects with a mean change from baseline of less than three letters under both standard and low luminance throughout the two year study.
Speaker Change: 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 teams and pre teams win vitamin a is diminished in the skin because by the way does help clean paws, so for diminishing it perhaps for the not so clean but its a very <unk>.
Speaker Change: While the AE to have systemically other than that clinically significant findings or at least a while says we're nothing in relation to physical exams cardiac health or organ function, what you'll see on the table are the outcomes from the two offer is that we expected xanthopsia and delayed documentation as mentioned these were reported as mild and of course transit you can see on the right hand side the frequency of <unk>.
Speaker Change: <unk> of patients presenting with those Aes. The night vision impairment you see there which is also mild is a more severe exacerbation of delayed bucket of patients. We had that in 15 reports of it and then of course, we have a non ocular E headache in some subjects, which of course can be manifest when subjects screened and user visual acuity, while experiencing these ocular aes, but over.
Speaker Change: We're all a very very well tolerated and safe profile from an adverse event perspective with respect to the visual acuity I mentioned there was stabilization throughout the study trial since we have our CMO Dr. Hinrich show on the line I'd like to get his clinical opinion on the PCA data potential.
Speaker Change: Thank you Nathan the chart shows the ETR S letter score in the study eye in Orange and the fellow eye and Gray over 24 months. This blended data, meaning that <unk> in the placebo group are shown as one group.
Speaker Change: The early treatment diabetic retinopathy study I'll briefly E. T D. R. S mental acuity testing as a standardized method used to measure visual acuity and as the gold standard in clinical trials and research settings.
Speaker Change: You did hear US solve includes 14 lines with five letters align in the Mexico score on EDI terrorists missile acuity testing its 100.
Speaker Change: They are significant intercession variability of Etfs as an acuity measurements enormous subjects test retest variability typically falls within about five let us in.
Speaker Change: And in individuals with macro degeneration variability tends to be greater namely about eight letters what we see on the chart is that it was not even a single letter loss on average clearly this allow us to conclude that there was no significant loss of visual acuity and it is fair to say that there was stabilization of visual acuity.
Speaker Change: In the Dragon trial.
Nathan: I hand back over to Nathan.
Nathan: Thank you Ginger.
Nathan: 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. It's a global study. The design is essentially the same as that for the start of our trials in fact.
Nathan: 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 there's going to be the same trial duration will also be including an interim analysis, the timing and specifications of that analysis have not been worked out but.
Nathan: 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, so with that I'll turn it over to Hal yen for the financial results.
Hal: Thank you Nathan.
Speaker Change: For Q1, 'twenty time side, we had R&D expenses of $9 4 million compared to $6 8 million for the same period last year.
Nathan: The increase was primarily attributable to the share based compensation and its body higher clinical trial expenses related to the Phoenix trial.
Nathan: Regarding G&A expenses U S $6 1 million compared to a $1 6 million for the same here last year. The increase was also due to the share based compensation grid.
Speaker Change: Paul We had a net loss of $14 3 million compared to a net loss of $7 9 million for the MTR last year.
Nathan: 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 7 million and was not cash related operating cash outflow was only about $8 3 million.
Nathan: As we raise 15 million due to a registered direct offering in February and received about $5 6 million from employee stock option exercise.
Nathan: Had a cash increase of $12 3 million for the quarter.
Nathan: And leave us with $157 4 million in cash liquidity ponds time deposit and U S Treasury bills as in Q1.
Nathan: We still expect four years of cash runway and we expect to be able to hear companies auto dollar or in clinical trials with this current cash. Thank you.
Nathan: Back to you operator.
Speaker Change: 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.
Speaker Change: You have dialed into todays call. Please press star nine to raise your hand and star sixth on mute. Please standby, while we compile the Q&A roster.
Speaker Change: Your first question comes from the line of Marc Goodman with Leerink Partners. Your line is open. Please go ahead.
Speaker Change: This is <unk> on for Mike. Thank you for taking our question could.
Speaker Change: Could you. Please our first question is about the Phoenix trial could you provide us some data about the discontinuation rates and <unk>.
Speaker Change: How the enrolment is ongoing in this trial.
Speaker Change: And the second question rehab.
Speaker Change: We have eight about any update on the regulatory meetings.
Speaker Change: For the end points for I'm, sorry for them try requirements.
Speaker Change: For Targa disease, you mentioned that you were supposed to be working on and planning to meet up with us to FDA and other regulatory agencies.
Speaker Change: To finalize the plan the development plan does it does thank you so much.
Speaker Change: Well thank you.
Speaker Change: So I'll take those questions. So the first question is regarding the Phoenix dropout rates is that right.
Speaker Change: Yes, correct. So the dropout rate is approximately around about 20%. This is way below what's been reported in previous studies.
Speaker Change: Such as.
Speaker Change: The Bureau voted rather in a phase III that was reported I think earlier. This year. There is a drop out about 30% and I believe that it makes is dead and other anti complement inhibitors in G. A they have a much much more over 30% to 50% I believe.
Henry: Henry equity shed some light on that.
Speaker Change: Yes that is correct I mean, it's not surprising that the injectables come with a higher risk and higher dropout rate and it makes us that's led to severe.
Speaker Change: A night blindness, and there was also a higher dropout rate in our in the triad and using it mixes such as the middle cycle modulator.
Speaker Change: Yep Yep, so what what's most of surprising is that even a dealer rated vitamin E had a bone and 30% dropout rate. So we are way below that so that's pretty encouraging considering that weird conducted the trial in very elderly patients I think the average user Rabbi 80 year old so compliance.
Speaker Change: <unk> is an issue, but we still have it within that 20%. So so we're doing pretty well.
Speaker Change: And then the second question was.
Speaker Change: The style that I E that we'll be discussing that work with regulators is that correct. If I remember correctly, yes.
Speaker Change: Yes, correct, yes.
Speaker Change: Yes so.
Speaker Change: So we have we have.
Speaker Change: Scheduling meetings with the regulators and.
Speaker Change: And we are doing so right now we will be meeting a few of those.
Speaker Change: We schedule some meetings in may 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 Change: Got it thank you so much.
Speaker Change: Yes.
Speaker Change: Your next question comes from the line of Jennifer Kim with Cantor. Your line is open. Please go ahead.
Jennifer Kim: Hi, Thanks for taking my questions maybe to start off and Star Guard can you just talk about the recent interactions you've had with the FDA and any thoughts on maybe any proceed regulatory risks given the changes at the agency.
Speaker Change: 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 Change: So I'll take the first one so we have met up with the FDA, yet that will be media blip. There soon so that that's been scheduled.
Speaker Change: And then what's the other question.
Speaker Change: If you have any thoughts on I guess regulatory risk given specifically about the changes that have been going on with agency <unk>.
Speaker Change: No I think we don't have.
Speaker Change: And the risk given that you know the data speaks for itself.
Speaker Change: So either I mean, even though days days.
Speaker Change: Subsidy changes within the FDA I don't think that's going to affect us.
Speaker Change: Especially I think that the vision that.
Speaker Change: We have made out with.
Speaker Change: Well the division that that's been key.
Speaker Change: <unk> given us guidance throughout the whole study.
Speaker Change: Especially during the phase III most of them must have a bad so I don't think there's any issues with the personnel change with you there.
Speaker Change: Okay, and then can I ask on the goalpost for the phase III, the 24 month phase III data.
Oh I'll tell you what to look for the Dragon I think it will complete by Q3.
Speaker Change: This year.
Speaker Change: Yes.
Nathan you want to shed some light on that confirmed I think Jennifer may be asking about but what exactly where we're looking for in terms of efficacy and safety correct Jennifer.
Jennifer Kim: Oh, okay.
Speaker Change: Yeah.
Speaker Change: So.
Speaker Change: Nathan you want to answer this but I was just going to mention that the study is powered to detect a 35% treatment effect between placebo and active so that's what we're looking for I mean, you know sometimes you get there is sometimes you don't but based upon what the D&B I looked at him and told US in terms of the safety and efficacy at the interim which by the way as I mentioned the majority of data we're actually avail.
Speaker Change: At that time, we're expecting that we'll be getting very close to our anticipated.
Speaker Change: The treatment effect size of 30% to 35% again, that's what the study is powered for in terms of safety I think the.
Speaker Change: Outlook is very positive given again, what we see at the interim less than a 10% overall withdrawal rate and three 8% 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 going to change very much by the end of the year. So I expect those.
Speaker Change: Dropout rates to be fairly consistent and as I said before you never know about efficacy, but based upon what the DSV saw at the interim unexpected that we will get very close to our anticipated the treatment effect size.
Speaker Change: Got it that's helpful. Thanks, guys.
Speaker Change: Your next question comes from the line of E. Chen with H C. W. Your line is open. Please go ahead.
E. Chen: Alright, Thank you for taking my questions.
E. Chen: In view of our President Trump's recent policy of most favored nation drug pricing could you comment on your potential strategy strategy regarding approval on launch of the drug ex U S territories, particularly Japan, and whether that will affect your U S.
E. Chen: Pricing and market prospects.
Speaker Change: Yes, thanks for that.
Speaker Change: Well first of all Canada to narrow body has not launched yet it's not going to affect US immediately we're still observing how that is going to impact the industry as a whole.
Speaker Change: And what type of drugs are affected woven drives rare pediatric graft et cetera, right now we're still monitoring what's going on so we don't have a immediate answer for that I don't think anyone is a immediate exit for that but we're still monitoring out windows.
Speaker Change: Got it thank you and a quick question on the financial side, our royalty operating expenses continued to rise during the remainder of 2025 based on the level recorded in the first quarter.
David: Yeah David.
David: Does that mean, you'll be abele are slightly higher than in Q1, we did a I think we did guide the market at this year into next year, we expect it to be a higher expenses given that most of the milestone of the studies all three studies and expect to be reached in the next one two years.
Speaker Change: Yeah. So the expenses will be higher this and next year, but moving forward you will come down back to the previous level.
David: Okay. Thank you.
David: <unk>.
Speaker Change: Your next question comes from the line of Bruce Jackson with Benchmark. Your line is open. Please go ahead.
Bruce Jackson: Hi, good afternoon, and thanks for taking my questions can you hear me okay.
Speaker Change: Okay.
Speaker Change: Perfect.
Speaker Change: Just a follow up on that last question, we had the increase in stock comp during the quarter or is that also expected to be a little bit higher going forward.
Speaker Change: Well, so it will really depend on the allocation of the operating expenses. So.
Speaker Change: You know you will have.
Speaker Change: Part of our ethos.
Speaker Change: The only benefit based on so called Hall.
Speaker Change: They bought about miles.
Speaker Change: So you said.
Speaker Change: And then it'll be hard to say what exactly when it will be you will be allocated out and those that are going to be best debate. All the time, you wont be allocated by by the by the period of the duration of the options. So it's a little bit hard to you.
Speaker Change: Really you would give you a figure but I think given we did have some of the aesop that is happening that order. So I don't expect that to be the high moving forward.
Speaker Change: This year.
Speaker Change: Okay, Great and then.
Speaker Change: Last question for me is on manufacturing, obviously, there's a push to move manufacturing to the United States for Pharmaceuticals, how do you.
Speaker Change: How are you set up right now in terms of inventory.
How is your supply chain structure and geographically.
Speaker Change: Oh, Yeah. Good question. So <unk> is manufactured in the U S. S Y as in other geographies so Terry.
Speaker Change: <unk> is not going to affect us any anyway.
Speaker Change: Okay Super Alright, Thank you very much.
Speaker Change: Thank you. Your next question comes from the line of Michael Okuda, which with Maxim. Your line is open. Please go away. Please go ahead.
Speaker Change: Okay. Thank you thank.
Michael Okuda: Thanks for taking my questions today.
Michael Okuda: So I guess the first question I would like to ask is if you can provide any more detail on the timing or any of that.
Michael Okuda: Assume that you would need to need or that in Germany in June and geographic atrophy.
Michael Okuda: So we believe that we.
Michael Okuda: So speaking on behalf of.
Michael Okuda: The mass teammates, probably just me and hen Greek.
We are meeting with the regulators, we believe that what we got what it takes to get us over the line, but until we ended up having.
Michael Okuda: Have any official response from them, we're not going to disclose anything as of now.
Michael Okuda: Alright fair enough.
Michael Okuda: And then.
Michael Okuda: Are there any additional sample size re estimations for the.
Speaker Change: The Phoenix study or is that five on your patient count finalize it looks like we're going to wrap up in enrollment in the next couple of months.
Speaker Change: Yep, So we expect them to rapidly enrollment because.
Speaker Change: That enrollment is going very smoothly S O.
Speaker Change: Now, we're making good progress we don't believe that we would need extra.
Speaker Change: Additional subjects.
Speaker Change: Which is editing it.
Speaker Change: Just in case, because we have of having smoothed enrollments than when it is.
Speaker Change: Enrolled more than two we enrolled more subjects to boost up our success.
Speaker Change: Nathan do it have anything to add on.
Speaker Change: 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 Change: Alright, Thank you very much thank you Michael.
Speaker Change: There are no further questions. So this concludes today's call.
Speaker Change: Okay.
Speaker Change: Okay.
Speaker Change: Sure.