Q2 2025 Belite Bio Inc Earnings Call
Speaker #1: US and Pioneer Designation in an. There's also been granted orphan drug designation in the US, Europe, and Japan. We believe this speaks to the significant unmet need for both indications, as currently there is no approved treatment for Stargardt's disease and no approved oral treatment for geographic atrophy.
Speaker #1: As importantly, we are uniquely positioned to address these unmet needs, as we are already in global Phase 3 trials for both indications. So with that, let me provide a high-level overview of the recent progress we have made.
Speaker #1: We have two studies underway with Teneriband in patients living with Stargardt's disease. These are the Phase 3 Dragon Trial and the Phase 2 Dragon 2 Trial.
Speaker #1: As part of the phase three dragon trial, we were pleased to announce earlier in the year that the data safety monitoring board had completed its interim analysis and recommended that the trial proceed without a sample size increase or modifications.
Speaker #1: They also recommended that we submit the data for further regulatory review for drug approval. As a result, the FDA granted Teneriband breakthrough therapy designation, and we remain on track to complete the study in Q4 this year.
Speaker #1: The Dragon Two trial also continues to be on track. We have enrolled 17 of our targeted enrollment of approximately 60 subjects, including about 10 Japanese subjects.
Speaker #1: The data from the Japanese subjects is intended to expedite a new drug application in Japan where we have already been granted a pioneer drug designation.
Speaker #1: In geographic atrophy, I'm pleased to share that we recently completed enrollment of our global phase three study with 529 subjects enrolled. We've recently also raised $15 million in gross proceeds in a registered direct offering on August the 8th.
Speaker #1: To summarize, we made excellent progress against our key milestones in the first half of the year. We are looking to carry this momentum into the second half of the year.
Speaker #1: Our balance sheet also strong with four years of cash runway. We remain well positioned to advance Teneriband as potentially the first oral treatment for people living with degenerative retinal disease.
Speaker #1: I'll now turn over the presentation to Hao-Yuan. Hao-Yuan?
Speaker #2: Thank you, Tom. For Q2 2025, we had R&D expenses of $11 million, compared to $9.1 million for the same period last year. The increase was mainly due to higher pathway expenses related to the clinical trial and manufacturing expenses.
Speaker #2: Which was partially offset by lower dragon trial expenses and development milestone payment for the completion of the phase two trial in 2024. Also, due to an rease in share-based compensation expenses.
Speaker #2: Regarding GNA expenses, we had GNA expenses of $6.5 million compared to $1.4 million for the same period last year. The increase was mainly due to an increase in share-based compensation expenses.
Speaker #2: Overall, we had a net loss of $16.3 million compared to a net loss of $9.5 million for the same period last year. One thing to note is that the majority of the increase in expenses came from share-based compensation.
Speaker #2: Which was about $7.6 million and was not cash-related. The operating cash outflow was only about $8.6 million. As of the end of Q2, we had $149.2 million in cash.
Speaker #2: Liquidity fund time deposit and U.S. Treasury bill. We also raised $15 million in gross proceeds in a registered direct offering on August 8th. We still expect four years of cash runway without considering the commercialization costs and anticipate being able to complete all three Phase 3 trials with our current cash.
Speaker #2: Thank you. Back to you, operator.
Speaker #3: Thank you. 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 #3: If you have dialed into today's call, please press star nine to raise your hand and star six to unmute. Please stand by while we compile the Q&A roster.
Speaker #3: You are first. Question comes from the line of Boris Peaker with Titan Partners. Please go ahead.
Speaker #4: Can you guys hear me? I just want to confirm. The line is live.
Speaker #5: Yes, we can hear you.
Speaker #4: Congratulations on the progress. I guess my first question is, what is the status of the FDA discussion regarding the interim data that you have from the Dragon trial?
Speaker #4: Just trying to get a sense if that could potentially be a trigger for filing BLA and if it's not, NDA in case, sorry, and if it's not, you know, what data would you think you'd ed to actually get from dragon two study one-year follow-up, two-year follow-ups?
Speaker #4: How long prior to an NDA filing?
Speaker #5: Thank you. So we can take this one. So we've met with the FDA, and we filed the breakthrough designation based on the IA data.
Speaker #5: And so, given the IA criteria was met when all subjects achieved 12 months on the study, the majority of subjects have already reached 15 months.
Speaker #5: So, I am, we could try for accelerated approval, but that would still require a confirmatory follow-up study. So the FDA recommended that we complete the 24-month study, which we're about to complete.
Speaker #5: Coming up in September, September, October. And so we will submit that final data and that will possibly be a single study approval based on the robust statistical significance.
Speaker #4: Gotcha. So just to confirm, this could be a possibility just filing on Dragon One and using that as an IS, and then Dragon Two.
Speaker #4: And when would you actually know that if Dragon One, in and of itself, is sufficient for approval?
Speaker #5: So I think based on the FDA's recommendation, that the P-value would be robust enough and we believe that we are on track to meet the criteria.
Speaker #4: Gotcha. Okay. I appreciate that. be if I could squeeze in just one question on GA. Now that
Speaker #5: Sure, sure.
Speaker #4: the Phoenix trial is completed enrollment, can you discuss if there's going be an interim analysis? Let's say, ou know, a year from now or so or, you know, any other timing around the data readouts?
Speaker #5: Yeah, sure. So there will be a IA for GA. I think based on the overall regulatory strategy right now, I don't think I have a timeline with me, but it will be something, it will be somewhere in between, halfway during the similarly to the Stargardt I just don't have the details, but it will be halfway mark.
Speaker #4: Gotcha. Well, I appreciate you taking my questions, and congrats on the progress again.
Speaker #5: Thank you so much.
Speaker #3: You are next question comes from the line of Bruce Jackson with Benchmark. Please go head.
Speaker #6: Hi. Thank you for taking my questions. Can you hear me okay?
Speaker #5: Yeah, yeah.
Speaker #6: Okay, super. So you got the Breakthrough Therapy designation, and have you discussed with the FDA yet if there's a route to accelerated approval?
Speaker #5: Oh, yeah. Yes. So there was a route for accelerated approval, but the FDA recommended that we have a majority of the patients completing the 15 months of time point already.
Speaker #5: So it wouldn't be long to reach the 24 months. Given that the accelerated approval will still require a second study, they recommended a single study.
Speaker #5: I mean, they recommended a single study path with robust statistical significance.
Speaker #6: Okay. And then my second question is about educating the medical community about your drugs. So are there any upcoming presentations or data readouts that we can look to?
Speaker #6: Near term, not just the completion or the follow-up analysis of the study, anything on an interim basis.
Speaker #5: Well, I guess that our timeline is very tight. That we are expecting so we ran the timeline with the FDA and they acknowledged that we'll submitting around the first quarter, first half, sorry, first half of next year.
Speaker #5: And they want us to remain confidential on revealing any efficacy data. So, we were planning on presenting the IA data and AAO, which is roughly around late October.
Speaker #5: But given that we'll be submitting very soon after that, the FDA would prefer that we stay confidential on the data.
Speaker #6: Okay, Bruce.
Speaker #2: I think I can add that. So once we have the final 24-month data, we will announce that. But so you wouldn't need to wait until the submission.
Speaker #2: To see the data. But before that, we will not reveal interim data.
Speaker #6: Okay, that's it for me. Thank you.
Speaker #2: Thank you.
Speaker #3: You are next question comes from Jennifer Kim with Cantor. Please go head.
Speaker #7: Hi, thanks for taking my questions. Can you hear all?
Speaker #5: Yes.
Speaker #7: right, maybe two to start with dragon. Can you remind us what the exact timing of data will be after the trial finishes in the fourth quarter?
Speaker #7: And then how and what data will be communicated, and what you're hoping to show in terms of efficacy to support filing? Maybe we can start there.
Speaker #5: Later, probably best that you have answered this question.
Speaker #2: You want me to.
Speaker #6: As you know, we're looking at the change of atrophic lesion growth, right? So lesion growth rate over time. And all subjects are all available time points.
Speaker #6: So we're looking for statistical significance between placebo and treatment in terms of changing the trajectory of lesion growth. So that's what we expect to show the agency is a statistically significant difference in lesion growth rates.
Speaker #6: And that is what you need for approval. It's the same endpoint that was used in geographic atrophy to get the two intravitreal drugs approved.
Speaker #6: So it's basically the same metric.
Speaker #7: Okay. And then my second question is just for Phoenix. What is the latest dropout rate you've seen to date? Is it still in the 20% range?
Speaker #7: And what can we expect in terms of the depths of data in an interim analysis?
Speaker #2: Well, I feel within our expected range. The depth of the interim, we have not finalized the interim design yet. So we'll announce that once we know.
Speaker #7: Thank you.
Speaker #3: You are next question comes from the line of Yu-Hsin with HC Wainwright. Please go ahead.
Speaker #8: Oh, thank you for taking my estions. So for the dragon two trial, what is the current estimate timeline for reaching the target enrollments of 60 patients?
Speaker #8: Is the current enrollment speed meeting your expectations?
Speaker #5: Yeah, I can take that question. So we initially launched dragon one. I'm sorry, dragon two in Japan. And now we are in the process of expanding to into additional countries.
Speaker #5: Which we expect will accelerate enrollment. We strategically timed the dragon two, not to compete with dragon one so that we can speed up the completion of dragon one.
Speaker #5: And of course, in the case if the DSMB recommends further sample size, addition, then that wouldn't slow down the completion dragon one. But since that the DSMB, that recommended that we wouldn't need to add any sample size.
Speaker #5: So we're kind of speeding up dragon two. So we're kind purposely staggered dragon two to not compete with dragon one. So even though it's slower than what we expected, but that is because of that dragon two wouldn't compete with dragon one.
Speaker #5: So I think we are still on time. I think the expectation of completing the enrollment probably will take, I would say.
Speaker #2: End of this year.
Speaker #5: Yeah, end of this year. Yeah.
Speaker #6: Understood. Thank you. And I noticed that your operating expenses continue to rise in the second quarter. Can you talk about the primary drivers for operating expenses and whether the, you ow, these kind of level of expensing is sustainable as you approach key clinical milestones later this year?
Speaker #2: Yeah, sure. Well, first of all, you ow the operating expenses does include a majority of the expenses come from the ship-based compensation. So those are not cash ated.
Speaker #2: And it's a little bit hard to really have a have a so-called precise forecast about that because they are purely based on evaluation of, you know, options and the probability of reaching those milestones.
Speaker #2: Many of our ESOP is going to be related for if development milestone. So it will purely based on the probability. So instead of time, you only get those vested as long as after we reach those milestones.
Speaker #2: So if you remove those, then the cash outflow is actually pretty close to the Q1, 2025. This year, and next year, we do expect to have higher cash burn given that we do expect to reach several milestones on both through the not both of those.
Speaker #2: So all three phase three studies. So this year and next year, we probably expect to have cash outflow around 40 to 45 million. For two years, this and next year.
Speaker #2: And then you will dial down a lot of starting from three years from now. So that's why we still expect four years of cash runway.
Speaker #6: Got it. Thank you.
Speaker #2: Thank you.
Speaker #3: A reminder that if you would like to ask a question, please raise your hand now. Your next question comes from the line of Michael Okonowicz with Maxim Group.
Speaker #3: Please go head.
Speaker #8: Hey, guys. Thank you so much for taking my questions today. Congrats on all the great progress.
Speaker #5: Thank you.
Speaker #6: So I just wanted to follow up little bit on the single pivotal design. In particular, if under the current FDA, because there have been a lot of changes over the past several months, if you received any communications from the current admin that confirms that single pivotal for full approval, perhaps around when you got breakthrough designation.
Speaker #5: I think we have. So we met with the FDA after submitting the IA data, which we call breakthrough status. I think the path forward is very clear for us and very straightforward.
Speaker #5: For a single study, if we met the statistical significance, robust statistical significance, that is P of less than 0.01, then we have a single study approval path.
Speaker #6: All right. Thank you. And then just a follow-up. Do you have a sense of what sort of scale of a commercial force you would need to actually bring this to market?
Speaker #6: Just given that you do have four years of runway and it seems like you're pretty well positioned to reach approval here.
Speaker #2: Well, the current cash burn that we forecast for four years runway has not included the full skill set of the commercialization costs. Which, you know, we are in progress of preparing that.
Speaker #2: So we will be able to budget once we confirm.
Speaker #6: All right. Thank you very much for taking my questions. And once in, congrats on all the great progress.
Speaker #5: Thank you very much.
Speaker #3: You are next question comes from the line of Mark Goodman with Lierink Partners. Please go head.
Speaker #9: Hi. Can ou hear me there?
Speaker #5: Yeah. Yes.
Speaker #9: Hey, guys. So I guess what I want to know is we get the data release in this press release that ou were talking about late this year, early next year.
Speaker #9: We get good news. How soon after will we be able to file? What are the gating issues between that press release and the filing specifically talk about CMC as well as long-term safety data?
Speaker #5: So I can answer this question. So the first one, long-term safety data, I think we will need a safety data of 300. I believe we've acquired it.
Speaker #5: So, Hendrik, can you help me out on the GA study? We will have enough safety subjects, right?
Speaker #9: Exactly, Tom, right? So the requirement is that we would have 300 subjects that would be 12 months on the drug. And including our Phoenix trial, we easily will have that by the first quarter of next year.
Speaker #5: Sorry, Mark, what was your other question?
Speaker #9: So basically, you could the safety from both studies you're saying to include into that. That's why ou're there. And the other was the CMC.
Speaker #5: Oh, so we have ongoing discussion with the CMC with the FDA. So so far, are on track. So it will probably be another discussion with the FDA before we submit.
Speaker #9: Which is from my where are on that? And just, you know, where's the whole process?
Speaker #5: We are on the registration batch. That's required for NDA.
Speaker #9: Right. Okay. Thank you.
Speaker #5: Thanks, Mark.