Q4 2024 Belite Bio Inc Earnings Call
[music].
Unknown Executive: Ladies and gentlemen, thank you for joining us and welcome to the Belite Bio fourth quarter and full year 2024 earnings conference 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.
Ladies and gentlemen.
Thank you for joining us and welcome to the be light bio fourth quarter and full year 'twenty 'twenty four earnings conference call. After today's prepared remarks, we will host a question and answer session.
Speaker Change: If you would like to ask a question. Please raise your hand, if you have dialed into todays call. Please press star nine to raise your hand and star six two on mute I will now hand, the conference over to Julie Fallon. Please go ahead.
Unknown Executive: If you have dialed into today's call, please press star 9 to raise your hand and star 6 to unmute.
Unknown Executive: I will now hand the conference over to Julie Fallon. Please go ahead.
Unknown Executive: Hello and thank you for joining us to discuss Belite Bio's fourth quarter and full year 2024 financial results. Joining the call today are Dr. Tom Lin, Chairman and CEO of Belite Bio, Dr. Hendrik Scholl, Chief Medical Officer. Dr. Nathan Mata, Chief Scientific Officer, and Haowen Zhang, Chief Financial Officer.
Julie Fallon: Hello, and thank you for joining us to discuss <unk> fourth quarter and full year 2024 financial results.
Joining the call today are Dr Tomlin, Chairman and CEO of <unk> bio Dr. Henry <unk>, Chief Medical Officer.
Speaker Change: Dr. Nathan Madden, Chief Scientific officer, and how long Shadow Chief Financial Officer.
Unknown Executive: 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.
Julie Fallon: Before we begin let me point out that we will be making forward looking statements that are based on our current expectations and beliefs.
Julie Fallon: These statements are subject to certain risks and uncertainties and actual results may differ materially.
Julie Fallon: We encourage you to consult the risk factors discussed in our SEC filings for additional detail.
Tom Lin: Now I'll turn the call over to Dr. Lin. 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 Tenerbean inpatients living with Stargardt's disease and geographic atrophy. For those who are new to our story, Tenerabend 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 Starler's disease and geographic atrophy. We believe this approach will be effective in slowing or halting lesion growth, which would ultimately preserve vision.
Lynn: Now I'll turn the call over to Dr. Lynn.
Thank you for joining today's call to discuss our fourth quarter and full year 2020 full financial results.
Lynn: Turning to April was an exciting year for <unk> as we continue to make strong progress towards advancing to narrow band in patients living with diverse disease and geographic atrophy.
Lynn: For those who are new to our story to nearby you say first in class oral therapy intended to reduce the <unk>.
Lynn: Cumulation of coffee product made by products, which have been implicated in the progression operator lesions in patients with established disease and geographic atrophy.
Lynn: We believe this approach will be effective in slowing or halting lesion growth, which would ultimately preserve vision.
Tom Lin: 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 Stargardt MGA.
Lynn: It is important to note that our approach focuses on early intervention of emerging grateful pathology based not mediated inflammation. We believe that this may be the best approach to potentially slow the progression of Saba and gea.
Tom Lin: to give you some perspective on the importance of this potential therapy. The lab band 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 US, Europe, and Japan. We believe this speaks to the significant unmet need for both indications. As currently, there is no approved treatment for Starlass 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.
Lynn: To give you some perspective on the importance of this potential therapy.
Lynn: <unk> has been granted rare pediatric disease and fast track designations in the U S and pioneer drug designation Japan.
Lynn: He has also been granted orphan drug designation in the U S Europe and Japan.
Lynn: We believe this speaks to the significant unmet need for both indications as kind of the days no approved treatment for <unk> disease, and non approved oral treatment for <unk>.
Lynn: And more importantly, we are uniquely positioned as we operated in global phase III trials for both indications.
Tom Lin: So with that, let me provide a high level overview of the recent progress we have made. We have two studies underway with delivered in patients living with Stargazer disease. These are the phase three dragon trial and the phase two, three dragon two trial. As part of the Phase 3 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 sample size increase or modifications. So essentially maintaining the sample size and having it for subtraction.
Lynn: So with that let me provide a high level overview of the recent progress we've had made.
Lynn: We have two studies underway with <unk> in patients living with saga disease. These are the phase III pivotal trial and the phase III three cricket to trough.
Lynn: As part of the Phase III trial, we recently announced that the data safety monitoring Board is company that you'd see German assays, which is based on all subjects have completed the one year assessment period.
Lynn: The D. S. M. B recommended that the trial proceed without sample size increase for modifications, so essentially maintaining the sample size at Henry for subjects.
Tom Lin: In addition, they recommend we submit the data for further regulatory review for drug approval. With the DSMB's review done, completion of trial is on track for end of this year. The DRAGON 2 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 subjects is intended to expedite a new drug application in Japan to which we have already been granted a pioneer drug designation. In GA, we also continue to progress in our Clinical Global Phase 3 Phoenix trial, which has already enrolled over 400 subjects today.
Lynn: In addition, they recommend we submit the data for the regulatory review for drug approval.
Lynn: Looking at the Smbs redo Devin completion of our trial is on track for end of this year.
Greg two trial continues to progress rapidly we have enrolled 11 of our targeted enrollment of approximately 60 subjects, including about 10 Japanese subjects.
Lynn: Data from the Japanese subjects is intended to expedite a new drug application in Japan to which we have already been granted at pioneer drug designation.
Lynn: In G. Eight we also continue to progress in our clinical global Phase III Phoenix trial, which has already enrolled over 400 subjects to date.
Tom Lin: We expect to increase the number of subjects to be enrolled in Phoenix trial from approximately 430 subjects to 500 subjects, as we have been making good progress on our subject enrollment.
Lynn: We expect to increase this number of subjects to be enrolled in Phoenix fell from approximately 480 subjects to 500 subjects as we have been making good progress on our subject enrolment to.
Tom Lin: to summarize with the excellent progress in our phase 3 trials. and the promising interim results from phase three stardust study and a four-year catch-run way, we remain well positioned in advancing TenderaBand as potentially the first oral treatment for people living with degenerative retinal disease.
Lynn: To summarize with the excellent progress in our phase III trials.
Lynn: The promising interim results from phase III Valor study at a four year cash runway, we remain well position in eventing till they're banned as potentially the first oral treatment for people living with degenerative retinal diseases.
Nathan Mata: 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's disease. As Tom mentioned, there are two phase three trials that we're currently involved in. The first is called DRAGON. It's 104 subjects in that trial. The other trial is called DRAGON 2. There are 60 subjects in that trial. You 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 size, as I just said.
I will now turn over the presentation to Nathan Nathan Please.
Thank you Tom.
Nathan Nathan: Here, we have an overview of our trial designs and started disease as Tom mentioned there are two phase III trials that we're currently involved in the first is called Dragon. It's 104 subjects in that trial. The other trial is called Dragon Twos 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 or <unk>.
Nathan Nathan: Signed identically so theres a difference in the number of sample size as I just said a difference in the geography. The Dragon is a global study Dragon two is focused on geographies in Japan U S and U K.
Nathan Mata: A difference in the geography. The DRAGON is a global study. DRAGON 2 is focused on geographies in Japan, U.S. and U.K. The DRAGON 1 study, because of the larger sample size, has a two to one randomization, Bayrington Larabat, whereas the DRAGON 2 trial has a one to one randomization with its 60 subjects. Otherwise, the trials are designed identically.
Nathan Nathan: The Dragon one study because of the larger sample sizes of two to one randomization favoring Tulare bet, whereas the Dragon two trial has a one to one randomization, we hit 60 subjects otherwise the trials are designed identically. It's important to note that the endpoint for drug approval is target disease N. G. E is slowing the growth of atrophic lesions and at the bottom you can see the key inclusion.
Nathan Mata: It's important to note that the endpoint for drug approval in Stargardt's disease and GA is slowing the growth of atrophic lesions. And at the bottom, you can see the key inclusion criteria for subjects involved in DRAGON and DRAGON 2. Here you see the demographics and baseline characteristics for the adolescent subjects involved in the DRAG-IN-1 trial. As I mentioned, there are 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.
Nathan Nathan: For subjects involved and Dragon and Dragon too.
Nathan Nathan: Here, you see the demographics and baseline characteristics for the adolescent subjects involved as a drag in one trial as I mentioned were 104 subjects you can see the mean age was $15. Four 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% fee.
Nathan Nathan: Mail in the study population and just below that you'll see the race distribution heavily favored towards the Asian population, because we did have headwinds heavily recruit in China. So we have approximately 56% of our Asian population, representing the study about a 37% being Caucasian in European and North American and various other categories approximately seven to eight <unk>.
Nathan Mata: And just below that, you see the race distribution, heavily favored towards the Asian population because we did 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 7-8%. Here's an overview of the interim analysis conclusions. As Tom mentioned, the study, DRAGON1, included a sample size re-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.
Nathan Nathan: <unk>.
Nathan Nathan: Here's an overview of the interim analysis conclusions as Tom mentioned the study Dragon. One included a sample size re estimation in which if the D. S. M. B saw a trend towards efficacy at the middle of this study then they would allow US to include up to additional 30 more patients to maintain that trend towards the end of the study so that we could ensure.
Nathan Nathan: A statistical significant difference by end of study, but in fact, the deals can be looked at the interim analysis. They felt there was no modification of the study required and that we should continue the study without a sample size increase I should remind you that the dosage that these are children were getting in the Dragon one in Dragon. Two studies is five milligrams daily this dose has been very well tolerated.
Nathan Mata: But in fact, when the DSMB looked at the interim analysis, they felt there was no modification of the study required and that we should continue the study without a sample size increase. I should remind you that the dosage that these children were getting in the DRAGON1 and DRAGON2 studies is 5 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 were approximately half of the subjects had already completed two years of dosing. The withdrawal rate was 9.6%, which is 10 of 104 subjects.
Nathan Nathan: And Dean save a very very nice safety profile also important to note 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 6%, which is 10 of 104 subjects and the withdrawal rate due to ocular adverse events was only three 8% that's four of 104 subjects.
Nathan Mata: And the withdrawal rate due to ocular adverse events was only 3.8%. That's 4 of 104 subjects. Visual acuity was stabilized in the 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 are additional comments, as Tom mentioned. They recommended us to submit the data for further regulatory review for drug approval, indicating perhaps an efficacy segment.
Nathan Nathan: Visual acuity was stabilize the majority of subjects with a mean change of baseline of less than three letters, so very well stabilize under both standard and low luminance threw out the two year study are perhaps the most important finding that the DSM V provider for US was what's provided at the bottom there additional comments as Tom mentioned, they recommended us to submit the data for further.
Speaker Change: Terry will review for drug approval, indicating perhaps an efficacy signal.
Nathan Mata: And here are the safety data from the DRAGON1 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 visual AE. This is a transient AE. It's last seconds to minutes, and no one dropped out of study because of chromatopsia or xanthopsia. Delayed dark adaptation is the other ocular AE that we anticipate based upon the mechanism of 10-layer event action.
Speaker Change: 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.
Speaker Change: Essentially drives this this of this of visual AE. This is a transient AE its last seconds to minutes and no. One dropped out of study because of Chromatopsia or xanthopsia delay documentation is the other oxalate E that we anticipate based upon the mechanism of <unk> action. This is the opposite of Chromatopsia in which going into darkness.
Nathan Mata: 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 16 to 20 minutes. Again, it's reported as mild, it's transient, and this is not synonymous with night blindness or nyctalopia 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 20 minutes.
Speaker Change: Patients have a longer time to accommodate to dim light setting. This can last two to three times longer than normal perhaps somewhere between 16 to 20 minutes again, it's reported as mild it's transient and this is not synonymous with night blindness or nyctalopia. Because these subjects will eventually get back their dark adapted sensitivity and you can see the distribution on the right hand side.
Speaker Change: In terms of the number of subjects in percentage in the patient population. The night vision impairment is a more severe exacerbation of delayed documentation and which of the delay may be as longer than 20 minutes. You can see that occurred in 15 subjects approximately 14% of the population and headache was another AE that we found in approximately 78% of the population this can happen when subjects.
Nathan Mata: You can see that occurred in 15 subjects, approximately 14% of the population. And headache was another AE that we found in approximately 7% to 8% of the population. This can happen when subjects strain to use their visual acuity while experiencing these AEs. But importantly, there was no clinically significant findings in relation to vital signs, physical 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.
Speaker Change: Strained to use their visual acuity, while experiencing these these aes, but importantly, there was no clinically significant findings in relation to vital signs symbols Ams cardiac health of 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 and the skin, but otherwise and overall very very well acceptable Cedric.
Speaker Change: Profile.
Nathan Mata: So here we see the visual acuity data from the DRAGON1 study. This is a two-year data. We're looking at a visual acuity under both standard and low luminance. We see overall stabilized visual acuity.
Speaker Change: So here, we see the visual acuity data from the Dragon. One study those are the two year data, we're looking at a visual acuity under both standard and low luminance, we see overall stabilized visual acuity before a clinical perspective, let's bring in our CMO. Dr entered Shaw for his opinion.
Hendrik Scholl: But for a clinical perspective, let's bring in our CMO, Dr. Hendrik Scholl, for his opinion. 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 EDGRS chart, and in patients with macular degeneration is 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 luminance visual acuity, is within the standard variability that you find in such patients.
Speaker Change: Thank you Nathan when considering the clinical relevance of the finding we have to take into account that can be measured in acuity. The intercession variability enrollment subjects us to let us on them either terrorists chart and.
Speaker Change: And in patients Smith magnitude generation is 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 alumina is it'll acuity is within the standard variability that defined in such patients.
Hendrik Scholl: Still, when we look at the left and the development of best corrected visual acuity and knowing that two-thirds of the subjects are under telerogant treatment, it's very reassuring that there was essentially no loss at all of best corrected visual acuity letters on a standard EGDRS chart.
Speaker Change: Still when we look at the left and the development of best corrected visual acuity and knowing that two thirds of the subjects are armed and narrow than treatment is very reassuring.
Speaker Change: St was essentially no loss at all of best practices and acuity letters on extended any theories chart.
Nathan Mata: So with that I hand it over back to Nathan. Thank you, Andrew. Here is our overview of the trial design in geographic attributes in our phase three trial called Phoenix. As Tom mentioned, we're going to recruit up to approximately 400 subjects. Right now, to date, we're right at about 400, so we've got about 100 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 1, two to one favoring to Larabet. It's a two years treatment duration.
Nathan: So with that I'll hand, it over back to Nathan.
Nathan: Thank you Andrew.
Nathan: Here is our overview of the trial design in geographic atrophy is as our phase III trial called Phoenix as Tom mentioned, where we're going to recruit up to approximately 400 subjects right now to date, we're right at about four hundreds we got about 100 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.
Nathan: We hadn't dragon one two to one favoring to layer of it. It's a two years' treatment duration and of course, just like it starts we're looking towards the slowing of atrophic lesion growth as the primary endpoint for drug approval, but of course, we're also looking at D. C. D E. A retinal anatomy by S. T. A C T and retinal sensitivity by micro Perimetry and light the starboard Dragon one study we will have in.
Nathan Mata: 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 BCDA, retinal anatomy by STLCT and retinal sensitivity by microperimetry. And like the Stargardt Dragon 1 study, we will have an interim analysis at one year.
Nathan: The interim analysis at one year.
Haowen Zhang: With that, I think I'll throw it to Haoyan for the financial results. Thank you. Thank you, Nathan. In 2024, we had R&D expenses of $29.9 million compared to $28.8 million in 2023. The increase in R&D expenses was primarily due to an increase in royalty payments for the completion of the Phase 2 trial and an increase in share-based compensation granted in the Q3 of 2024. On G&A expenses, in 2024, G&A expenses were $10.1 million compared to $6.8 million in 2023. The increase was primarily driven by an increase in share-based compensation granted in Q3 of 2024. On net loss, we had a net loss of $36.1 million in 2024 compared to $31.6 million in 2020.
Speaker Change: With that I think I'll throw it to <unk> for the financial results. Thank you.
Nathan: Thank you Nathan.
Nathan: In 2024, we had R&D expenses of $29 9 million compared to $28 8 million in 2023. The increase in R&D expenses was primarily due to an increase in royalty payments on a completion of the phase two trial.
Nathan: And an increase in share based compensation granted in Q3 of 'twenty Honey hole.
Nathan: On G&A expenses in 'twenty 'twenty, four G&A expenses were $10 1 million compared to $6 8 million in 2023.
Nathan: Increase was primarily driven by an increase in share based compensation granted in Q3 of 2024.
Nathan: Nella he had a net loss of $36 1 million in 2024 compared to $31 6 million in 2019.
Haowen Zhang: In terms of cash, we have $31.7 million in cash and $113.5 million in investment by end of 2024, as compared with $88.2 million by end of 2022. The investments were in liquidity funds, in-time deposits, and U.S. Treasury. One thing to note is that the net cash outflow for operating activities was $29.2 million in 2024. Similar to the cash outflow of $29.8 million in 2020. We also raised $15 million in gross profit proceeds in a registered direct offering in February 2025. We still expect four years cash runway without considering the cost from a second GFA3 study.
Nathan: In terms of cash we had $31 7 million in cash and Hungarian $13 5 million in investment by end of 2024 as compared with $88 2 million by end of 'twenty three.
Nathan: The next thing we're in the economy fun in time deposit and U S Treasury bills.
One thing to note is that the net cash outflow for operating activities was $29 2 million in 'twenty Honey fall.
Nathan: Similar to the cash outflow of $29 8 million in high 93.
Nathan: We also raised $15 million in gross proceeds in a registered direct offering in February 2025.
Nathan: We can expect for years cash runway mcgough containing our cost on a second G. A phase III study.
Haowen Zhang: Thank you.
Unknown Executive: Back to you, operator.
Nathan: <unk>.
Speaker Change: Back to you operator.
Unknown Executive: 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 star 9 to raise your hand and star 6 to unmute. Please stand by while we compile the Q&A roster.
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.
Jennifer Kim: Your first question comes from the line of Mark Goodman with Lee Rink. Your line is open. Please go ahead.
Speaker Change: Your first question comes from the line of Marc Goodman with Leerink. Your line is open. Please go ahead.
Jennifer Kim: Moving along, your next question comes from the line of Jennifer Kim with Kantor. Your line is open, please go ahead. Hi, thanks for taking my questions and congrats on the progress.
Speaker Change: Moving along your next question comes from the line of Jan.
Speaker Change: Mr. Kim with Cantor. Your line is open. Please go ahead.
Speaker Change: Hi, Thanks for taking my questions and congrats on the progress maybe on my first question, starting with Stark art. So honesty F. N. B's recent recommendation you 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.
Jennifer Kim: 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.
Speaker Change: Positive or negative.
Speaker Change: Thanks, Jonathan I can take that.
Tom Lin: I can take that. So the DSMB has recommended the entry results to be reviewed by regulatory agencies for drug approval, as you pointed out there. We believe this is very positive outcome and will be following DSMB's recommendations to request regulatory review and see whether the agency is confirmed with DSMB's recommendation. So we believe that, certainly believe that the regulatory agencies will probably align with the DSMB's recommendation because it's not every day that they make this kind of recommendations during interim, but if not, if they don't see it that way, then we'll just move on and carry on with the study.
Speaker Change: So the D. S. M. B has recommended that UK results to be reviewed by our regulatory agencies for drug approval as you pointed out there.
Speaker Change: We believe this is very positive outcome and how we'll be follow ABS MBS recommendations to request regulatory review it as he waited.
Speaker Change: Jay This is consumer P is a b's recommendation.
Speaker Change: So we believe there certainly believed by the regulatory agencies, who probably align with Ppas and B recommendation.
Speaker Change: Issue, because it's not every day that.
Speaker Change: They see they would make this kind of recommendation theory interim.
Speaker Change: But he's not the regular it is.
Speaker Change: If.
Speaker Change: He's a dosia their way, David where just.
Speaker Change: Move on and to carry on with the study.
Tom Lin: Okay, and then maybe turning to GA, what drove the decision to increase the sample size to 500 patients? Well, we're getting so the 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 Q3 this year.
Speaker Change: Okay, and then maybe turning to G. A what drove the decision to increase the sample size to 500 patients.
Speaker Change: Well, where we're at.
Speaker Change: Getting this study has been moving along very smoothly.
Speaker Change: We want to take this opportunity to roll more subjects to further boost our chances of success.
Speaker Change: So based on this current enrollment rate, we should still be able to compete with any I expected timeline, which is Q3 this year and the costumer still we'd be very feasible based on.
Tom Lin: And the cost will still remain very feasible based on the time we 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 if the if we get any positive signals from the IA for Phoenix, then we would speed up and expedite our second phase, second phase three trial for for GHA.
Speaker Change: Perm recruitment rate.
Speaker Change: Okay, if I could squeeze one more question with G. A can you just remind me.
Speaker Change: What is your latest thinking on.
Speaker Change: What.
Speaker Change: The interim analysis will entail and how that sort of feeds into the decision to start a second trial.
Speaker Change: You mean for the Phoenix being driven by Phoenix Yeah.
Speaker Change: Well, we certainly believe that if the if we get any positive signals from the iPhone.
Speaker Change: <unk> for Phoenix, then we would speed up and expedite Bal our second phase the second phase III trial for Phoenix.
Speaker Change: So it's now 48 sorry.
Speaker Change: Okay.
Basma Ibrahim: Your next question comes from the line of Mark Goodman with Lyrinc. 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.
Marc Goodman: Your next question comes from the line of Marc Goodman with Leerink Mark as a reminder, please on mute yourself. Your line is now open. Please go ahead.
Speaker Change: Hi, Good afternoon can you hear me Okay now.
Basma Ibrahim: Hi, this is Basma on for Mark. Thank you for taking our questions. Our first question is about Stargardt disease. Are you going to be able, when you meet with the authorities 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, can you give us an update about the current discontinuation rates in the GA trial, the Phoenix trial? Thank you. Sure.
Speaker Change: Okay. Hi, this is <unk> on for Mike. Thank you for taking our questions. Our first question, it's about Sagar disease.
Speaker Change: Are you going to be able when you meet with the authorities to regarding regulatory clarity for the submission would you be able to also get confirmation regarding the potential for a broad label.
Speaker Change: Given that the study population sapphires only adolescence.
Speaker Change: Second question again could you give us an update about that current discontinuation rates.
And the GE a child of Phoenix trial. Thank you.
Tom Lin: So I'll take the second question and I'll leave the first one for Hendrik. So the dropout rate for Phoenix right now is approximately about 20%. It is very common because the majority of the subjects enrolled in the GEA trial are elderly population. So for previous studies, we've seen the dropout rate about more than 3%. In fact, the dual rate of vitamin A study that was just recently presented at J.P. Morgan, the dropout rate is more than 30% and certainly more for emyxostat and the anti-complement study. And so I'll let Hendrik confirm the dropout rate for the anti-complement studies and as well as your questions on the startup or label.
Speaker Change: Sure.
Speaker Change: So I'll take the <unk> the.
Speaker Change: The second question I'll leave the first one for Henri So a.
Speaker Change: The dropout rate for Phoenix right now is approximately about 20%.
Speaker Change: It is it is.
Speaker Change: It is very common because.
Speaker Change: Majority of these subjects enrolled in the G. A trauma elderly population so.
Speaker Change: So for previous studies, we've seen a dropout rate of about one 3% in fact, the euro rate of vitamin a study.
Speaker Change: They were just recently presented at J P. Morgan the dropout rate is more than 30% and certainly more pool. It makes the state and.
Speaker Change: And that complement study in aerosol.
Hendrik: I'll, let hendrik.
Speaker Change: Thanks.
Speaker Change: Thanks Henry.
Speaker Change: Two.
Speaker Change: Comfort me period before the dropout rates for the prerecorded Pinnacle movement studies and as well as answer your questions on the startup or enable Henry.
Hendrik Scholl: Hendrik? Yeah, 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 a younger age and early onset still show a relatively similar progression rate compared to other subjects that were older in the proxor study, not very old, obviously, but still, it's still a juvenile maculatistrophy, but would be adults. So we believe that the threshold actually, to get something approved for a pediatric population would be much, much can show efficacy in our adolescent population.
Speaker Change: I'm happy to thank you Tom.
Speaker Change: While the Athena natural history studies is that.
Speaker Change: In patients that have an early onset of disease. The disease is generally more severe and show the path of progression the process that he has shown that sub.
Speaker Change: Subjects with.
Speaker Change: Younger age and early onset.
Speaker Change: Still show a relatively similar progression rate compared to other subject that we are older and the proxy that is not very old obviously, but still since it's still a juvenile macular dystrophy, but would be adults. So we believe that the threshold actually to get something approved for a pediatric population with.
Speaker Change: Would be much much higher and we feel that if the potential efficacy in our adolescent population it should be relatively straightforward to get the drug approved for adults as well.
Tom Lin: It should be relatively straightforward to get the drug approved for adults as well. and Hendrik King also mentioned about the dropout rate for the ad hoc compliments as well as for the mixed So in the end, in the anti-complement Dr. Michael Okunewitch, Yi Chen, Tom Lin, Nathan Mata, Zafar Aziz, Hendrik Scholl, Belite It was a little over 40%. Thank you. That's very helpful.
Speaker Change: And the indication yourself.
Speaker Change: You mentioned about the dropout rate for <unk> and that compliments as weisberg Immix is dead.
Speaker Change: So within in.
Speaker Change: In the anti complement.
Speaker Change: Ah studies.
Speaker Change: Baud rate was in the order of magnitude of 20% to 30% I believe in our in the study with a mix of start which has quite extensive side effects that dropout rate was even higher and would actually hand over to <unk>, Marta who actually knows a lot about.
Marta: That specific drug and its effect in geographic atrophy.
Speaker Change: Little over 40%.
Marta: Thank you thank.
Speaker Change: Thank you that's very helpful.
Yi Chen: Your next question comes from the line of Yi Chen with HCW. Your line is open. Please go ahead. And thank you for taking my question.
Speaker Change: Your next question comes from the line of <unk> Chen with H C. W. Your line is open. Please go ahead.
Alright, Thank you for taking my question.
Speaker Change: To clarify.
Hendrik Scholl: Just to clarify, the adolescent stroke or disease patients enrolled currently into the Dragon 12, what they represent, what percentage of the stroke or disease patients diagnosed in the real world? Hendrik, I believe there's a question for you as well. I'd be happy to take that question. So the typical Stargardt patient would notice first symptoms in the second decade of life. We see patients that have a very early onset, as early as five years, and then there are patients that are later in adulthood develop the first symptoms. But as Carl Stargardt described the disease in 1909, right, this is a juvenile maculodystrophy, 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 would schedule a visit in clinic, I would I would believe that that would represent two thirds of SACA patients that I would see, for example, in my clinic.
Speaker Change: The adolescent.
Speaker Change: The lessons from her disease patients being robbed currently each of the directors Carl what.
Speaker Change: <unk> represents what percentage of the starboard disease.
Speaker Change: <unk> diagnosed in the real world.
Speaker Change: Andrey believers critically as well.
Speaker Change: I'd be happy to take that question. So the typical startup patient wood wood.
Speaker Change: <unk> noted first symptoms in the second decade of life via D. C patients that have early very early onset as early as five years and then they are Ah patients that are later in adulthood developed the first symptoms, but S called Star Golf described the disease and 19 on I am right. This is the juvenile.
Speaker Change: Macular dystrophy and it typically starts between 10 and 20 years of age with her symptoms.
Speaker Change: Given that our study population actually include subjects between 12, and 20 years old we feel that it is very representative and would show an overlap. If you look at all startup patients that did it.
Speaker Change: That would.
Speaker Change: Schedule, a visit and clinic I would I would believe it.
Speaker Change: That would represent two thirds of startup patients that I would see for example in my clinic.
Hendrik Scholl: So you would expect potential approval in the future for all Stargardt disease patients in that age range, right, not necessarily meeting those, meeting the enrollment criteria in the current trial. If I understood the question correctly, the question is, if we show a future prescription label is not restricted to the patient groups you are currently enrolling into a pivotal drug? Exactly. The answer is no. And there would be no reason why we would not prescribe that drug to let's say an adult patient that developed the first symptoms at age 30 and still shows progression of these DDAF lesions, which is typical also for adult patients.
Speaker Change: So you would expect potential approval in the future or stop or disease patients in that age range already meeting those.
Speaker Change: Meeting the enrolment criteria your current trial.
Speaker Change: Yes.
Speaker Change: So if I understood the question correctly.
Speaker Change: This is vishal.
Speaker Change: The future the future prescription label is not restricted to the patient groups and are currently enrolling each where people are expecting the answer is no and there would be no reason why it would not prescribed the drug to earn let's say an adult patient that that developed the first symptoms at age 30, and and and and still shows pro.
Speaker Change: Question of these D D F lesions, which is typical also for adult patients.
Hendrik Scholl: But do you expect any potential limitation on the payer side for reimbursement if the label is broader than the patients currently enrolled in the Dragon Trust? Well, I think we will definitely possibly regulate to try to get the label for the adults, which we think is doable. And we'll probably just do a PK study to prove that it works the same on the adults.
Speaker Change: But do you.
Speaker Change: Any potential limitation on the payer side for reimbursement.
Speaker Change: The label is broader than the patients currently enrolled in the Dragon trial.
Speaker Change: Well I think we will definitely partner regulator.
Speaker Change: Two.
Speaker Change: Try to get a label for adults, which we think is doable.
And I would probably just do a PK study to prove out is that.
Speaker Change: It walks sustained wanda on the adult patients.
Hendrik Scholl: Thank you.
Speaker Change: Okay. Thank you.
Speaker Change: Okay.
Bruce Jackson: 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.
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 the questions.
Bruce Jackson: First, a housekeeping question about the capital raise you did for $15 million. Has anyone exercised the warrants yet attached to that? Not yet. Okay.
Bruce Jackson: First a housekeeping question about the the capital raise you did for $15 million.
Bruce Jackson: Has anyone exercised the warrants yet attached to that.
Bruce Jackson: Not yet.
Bruce Jackson: Okay.
Tom Lin: And then in February, you said that your CRO is going to be handling some of the regulatory process for you with 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 procedure, different procedure or and certainly different templates for submitting these kinds of regulatory submissions. So right now, it's in good hands and they're basically submitting as we speak.
Bruce Jackson: And then.
In February you said that your cero as can be handling some of the regulatory process for you.
Bruce Jackson: With the.
Bruce Jackson: The data for Dragon.
Speaker Change: 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.
Speaker Change: Yeah. Thanks, Thanks, Bruce so so we have a.
Two or three different geos, representing us for different jurisdictions.
And certainly they have a a procedure different procedure or is serving different attempted for.
Speaker Change: So based on these kinds of regulatory.
Regulatory submissions.
Speaker Change: So right now it's in good hands and there.
Speaker Change: Basically submitting as we speak.
Tom Lin: and Pear. All right, great.
Okay.
Bruce Jackson: That's it for me. Thank you.
Speaker Change: All right great. That's it for me thank you.
Michael Okunewitch: Your next question comes from the line of Michael Okunewitch with Maxim.
Speaker Change: Your next.
Speaker Change: Question comes from the line of Michael <unk> with Maxim. Your line is open. Please go ahead.
Nathan Mata: 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 the DRAGON study powered for? Nathan, do you want to answer this one? Yeah, it's powered for 40% is 40% treatment effect with 80% power to detect that that effect at at the second year.
Speaker Change: Hi, guys. Thanks for taking the questions just first what kind of difference in lesion growth between placebo and treatment as is the Dragon study powered for.
Nathan: Nathan to answer this one.
Nathan: Yes, it's powered for a 40% fee is 40% treatment effect with 80% power to detect that effect at the second year.
Nathan Mata: Okay, great. Thank you and congrats on all the progress.
Speaker Change: Okay, great. Thank you and congrats on all the balances.
Unknown Executive: Thank you.
Nathan: Okay.
Unknown Executive: There are no further questions at this time.
Nathan: There are no further questions at this time. This concludes today's call. Thank you for attending you may now disconnect.
Unknown Executive: This concludes today's call. Thank you for attending.
Unknown Executive: You may now disconnect.