Belite Bio, Inc (BLTE) Earnings Call Transcript & Summary

Ladies and gentlemen, thank you for joining us, and welcome to the Belite Bio 2026 Commercial Day. [Operator Instructions]. I will now hand the conference over to Julie Fallon. Please go ahead.

Good morning, everyone, and welcome to the Belite Bio Commercial Day. 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. Our goals for today are to provide you with an overview of the current diagnosis path and treatment paradigm for Stargardt disease Type 1. Stargardt impacts people's day-to-day lives, the significant unmet need for treatment and Blight Bio's commercial preparedness strategy. We have a full agenda. So with that, let me turn the call over to Dr. Lin, Belite Bio's Chairman and CEO, for opening remarks. Dr. Lin?

Good morning, everyone, and thank you for joining us for Belite Bio's Commercial Day. We are pleased to have the opportunity to share how we are preparing for what we believe could be a transformational next chapter for our company and for patients living with starless disease type 1. Brio, mission is to preserve vision and pioneer degenerative retinal disease treatments with fuel or notices today. We are approaching 1 of the most significant milestones in Belite Bio history. As we recently announced, FDA has accepted our NDA for tender narrow band in Stargardt disease Type 1. The FDA also granted priority review and assigned a PDUFA date of February 12, 2027. We are very encouraged by this milestone and believe the FDA's NDA acceptance with priority review reflects the immediate need for an approved treatment for status disease. Furthermore, we believe this reinforces the quality and depth of the data that we have generated throughout this drug development program. If approved, the Tinlarebant will be the first and only approved treatment for status disease type 1. Approval would also market evolution into a commercial state biotech company and allow us to begin delivering on the vision that has guided this organization since its founding. While the NDA is under review, our focus remains on ensuring we are ready to deliver for patients, physicians and the broader retail community. We have been building the capabilities needed to support the successful U.S. launch. -- from commercial infrastructure and market preparation to the physician education and patient support planning. All the goal of being ready to serve patients quickly. We have assembled a highly experienced team with deep expertise in rare disease of the margin and commercialization. Joining us today are Belite Bio's Chief Medical Officer, Dr. Henrik Scholl, Chief Commercial Officer, Keri Kilpatrick; and Chief Financial Officer, Hao-Yuan Chuang. Our objective is to give you an opportunity to hear from experts and get a clear view of the work we have done to support commercial readiness. -- and why we believe the Billa team is well positioned to execute successfully. Also with us today, you'll hear from Dr. Michel Malades who will provide an overview of Tenant and the unmet medical need in stages disease. Dr. Paul Bernstein, who will discuss the current treatment landscape and the opportunity to define the standard of care. And from the Belite team, Dr. Henrik Scholl will provide an overview of the global stack-up opportunity. and Kelly CoPatrick will discuss our U.S. commercial launch strategy. We'll also have time at the end for Q&A. But first, we are honored to work up Tracy and Haber who are both living with Stargardt disease Type 1 to share their personal journey. Hearing their story reminds self why this work matters and reinforces our commitment to bringing meaningful innovation to patients. Thank you again for joining us, and I will now turn it over to Tracy and Heber.

I began to not be able to have enough light in an already room I was beginning to make a lot of typographical errors at my workplace and my assignments took a lot longer. But for me, the most impacting realization was when I began to get into car axis. And it did not down me that it was my vision. I always assumed it to be the ag driver. But my last car accident really woke me up and that's when it decided to go get my eyes checked.

When I was reading the hurt all I could see was the letter E, the biggest letter on the chart and he -- the doctor was saying how I only was doing that to good appear of glasses since my little brother Gotapeer? And I mean, I was years old, I didn't understand what full vision meant in being able to see that meant. And so he cut my prescription in house. And from there were the glasses and 1 of my teachers went up to my mom and was concerned that I had a learning disability because I was falling behind in reading and that I'm still looking very close to my computer. And so that is what really made my mom was like, okay, some things Ron here. She's not reading well. She's not pulling basketball well. We think she's a learning disability. -- does all these little things started to add up even though I had a peer of glasses. So that really was a huge point where I diagnosed.

So the honest -- when I was explained what Stargardt was and what to expect, honestly, I wasn't listening. I still had vision. I couldn't believe that this was happening, and I went on with my day to day -- but I had to learn from myself and my own experiences as to what was happening with my eyes. And that brought up so many unwanted feelings for me. I did not want to believe what had been shared with me. I had still had vision enough to still drive. I could still use my phone without a problem. But within a year's time, or even longer than that 2 years, I'll even say, I began to notice that the vision was getting worse because I couldn't see it faces clear anymore. -- and reading my phone was more challenging. So that's pretty much, I think, the time span that it took for me to accept what is happening to me.

I was sitting there 9 years old, and I remember this so clearly, I was dancing in the doctor's office, sending a random singing the doctor came in and they looked at us and they were like you were going to have to pull have out of private school, and she will never be able to drive a car. And that is the only memory I have from being diagnosed in 90 years old of Stargardt disease is being told some of the works news of my life. I want to flow with my 2 cousins and just not being able to like drive knowing when I turn '16, I wouldn't be behind a car. And that's all a thought about with my eye disease, I didn't understand what it was no 1 explained to me what Stargardt was, just the things that I couldn't do. love reading. I can't wait a people back book, and that's really hit me hard. I think this past year, I've always been a huge reader. I have a Kindle, I listen to audio books, but there's nothing like turning a page of a buck driving is a huge one, and it probably will always be, I don't know what my future is going to look like, especially with wanting to be a mother with taking my kids to school or taking my kids to practice, just having those like little things I know why I won't be able to do with my kidos, I would definitely see a huge one is traveling. I love traveling, but I can't travel by myself yet. Airports scare me and they are very visually overstimulating -- and I have a hard time navigating them.

Stargardt disease impacts my life from the time I get out of my bet, took a time that I get back in the bed. And living with this vision impairment for me is a daily challenge, game dress identifying colors in clothes has become a challenge for me, preparing meals, organizing the spices making sure that it will calm myself while preparing the vegetables is a challenge. Reading expiration dates on boxes difficult, impossible for me, navigating outdoors extremely scary unsettling, not being able to know which how to go through in the grocery store, confusing for me. Overwhelming at the checkout counter not being able to know which aisle to go to when it's being cold, sickle purchase my groceries, challenging, getting in an open ride scary, not knowing if the driver takes a difficult amount that I'm unfamiliar with. Uncertainly, socializing with friends that are cited, not worrying if they are going to know how to take care of me, how to guide me while we go out. Ordering food from the menu how to identify it, where to find the food on my plate not knowing if I'm picking up for pools of air or not and learning how to get to the restaurant out there, feeling comfortable while they share pictures on their phones, with me. I think that overall is all challenged, but I've learned to make modifications. I've learned to ask for help. I've definitely learned to educate people on how I need their help and then where I don't need their help because Stargardt disease is a disease where your central vision is impacted, but the peripheral vision, my peripheral vision is good enough for me to be able to identify a house from a car. However, I would not be able to tell if there was someone sitting in that car or if some one is at the window of their house.

I would say it definitely takes a little bit of a mental drain on me sometimes just because I'm 18, and that's something I've struggled with is like going out with friends and like not knowing all the faces that are there because I can't recognize them that I need them to talk for me to be able to recognize their voices. And so -- that's definitely been something I've personally struggled with a lot is heating yet with friends and not knowing who is all there because of my vision. Yes, I've definitely struggled with asking for a combination just because sometimes they get tired of it and they just want to live a normal life. But I know I have to do it to be safe. And that's something that I struggle with, and my mom has to speak up for me sometimes like even cutting my food in restaurants in like a dam restaurant has always been a struggle for me -- so it always navigate towards finger food, but then when we man knows I want to seek, she'll order me and cut it up for me. So just like little things really suck sometimes, but the people as for myself make it better.

I think another thing for me is I miss seeing faces. I've learned to adjust to having a conversation with someone and enjoying that conversation. But in the back of my head is always that I wish I could see them. The most harder game for me is having conversations with my daughter, loads together. We have a great time, but not being able to see her face, it hurts me so much. So I think that to get that back or another one reading a book in bed, how I missed that. I miss that while on driving. But just being able to sit in the bed with a cup of tea and bringing a good book. That's something that I missed. I appreciate oratory versions of reading, but using my physical eyes for the things that I enjoy doing like crafting or doing a shadow on my eyes miss so much being Tracy, being me, it's taken away some things that I really enjoy. My hope for the future for people that are living with Star Guide is that they never give up on themselves. Even if they have to change some of their goals or their dreams that they remain steadfast to who they are and to be open to learning how to live life differently, learning to love themselves only to trust themselves and then learn to believe in themselves and not being afraid to ask for help, not being changed or angry when they make mistakes, never giving up and then also never giving up on a hope that there will, once they be a cure for us.

As you've just heard, consistent with my experience in managing patients, Stargardt disease has a profound impact on individuals living with this inherited retinal disease. It's the most common macular dystrophy. The color fundus photograph shown here shows the classic findings of retinal atrophy centrally with surrounding yellow white funders Flex within the posterior pole due to the accumulation of lipofuscin. It's caused by disease-causing variants in the gene ABCA for which encodes a key component of the visual cycle, also known as the vitamin A recycling pathway. ABCA4 is required for removal of vitamin A byproducts from the disk membranes of photoreceptors after photo bleaching of visual pigment. When this is malfunctioning, these byproducts are trapped within the photroceptor outer segments as the RPE phagocytoses the outer segments on a daily basis, these byproducts eventually accumulate within the RPE and react to form A2, a dime of vitamin A, which eventually leads to the generation of lipofuscin and related toxic bisretinoids, which results in RPE and photoreceptor dysfunction and death and thereby progressive loss of retinal structure and function over time. Presentation is very broad from early childhood to late adulthood. Stargardt disease, also known as ABCA4 associated retinal dystrophy is a rare and progressive eye disease, leading to legal blindness in almost all cases, there is no approved treatment for Stargardt disease. The pathophysiology of Stargardt disease is well understood, and the key symptoms include a gradual loss of central vision in both eyes, often starting in childhood or early adult and can include blurred vision or distorted central vision, central blind spots, photophobia or light sensitivity and delayed dark adaptation. Impaired color vision is also common as is difficulty with detailed tasks. Often peripheral vision remains relatively preserved. This has an impact on activities of daily living, including challenges with reading, using screens and recognizing faces, more often than not, there's an inability to drive. It can affect mobility, and has social, emotional and psychological impacts, including a greater degree of anxiety and depression and has impacts on career and FX finances and productivity. ProgStar, the progression of Stargardt disease was a landmark international multicenter natural history study designed to characterize the progression of ABCA4 associated Stargardt disease and to establish robust structural and functional outcome measures for future interventional clinical trials by both respectively and retrospectively following patients with standardized assessments, including Fundus Autofluorescence, spectral domain OCT, microperimetry and visual acuity, the study defined disease progression, quantified lesion growth and identified sensitive endpoints such as definitely decreased autofluorescence, DDAF. Moorfields Hospital was one of the leading international study centers, and I had the privilege to serve as 1 of the PIs and contributors to the ProgStar Study Group. -- using standardized funders or depressants imaging, ProgStar demonstrated that DDF definitely decreased autoforescence, represents well demarcated areas of complete RPE loss, and enlarges at a predictable rate, making it a robust primary structural endpoint. It also defined questionably decreased autofluorescence, QDAF as regions of partial autofluorescence reduction that eventually evolve into DDAF and thus provides an earlier marker of progression, as shown in the representative of cereal outearns images above. By combining DDAFand QDAF, as a total decrease or autofluorescence, which captures the full extent of RPE impairment and offers a sensitive, comprehensive metric across a wider disease spectrum. The ProgStar study confirmed that these autofluorescence measures are highly reproducible, correlate with functional decline and are suitable for detecting progression over 12 to 24 months. As a result, DDAF, CDAF and total DAF have become the standard structural outcome measures for interventional trials in Stargardt disease. In ProgStar report 17 it was shown that the mean DDF growth rate in the ProgStar cohort over 24 months was 0.74 millimeters squared per year. DDF growth as an outcome measure has been accepted by the FDA and as an approvable endpoint in treatment trials for Stargardt and was the primary endpoint in the pivotal DRAGON trial. In the prospective cohort of 434 Stargardt patients in the ProgStar study. The overall rate of best corrected visual acuity loss was 0.55 letters per year over 2 years. If we look specifically at those with the baseline BCVA between 2070 and 2200 which is shown in the lower left spaghetti plot and matches the Dragon cohort, visual acuity declined at a rate of 0.6 letters per year. If we now move to the DRAGON trial itself, this was a Phase III multicenter, randomized, double-masked, placebo-controlled study to evaluate the safety and efficacy of Tinlarebant in the treatment of Stargardt disease in adolescent subjects. The key inclusion criteria included a clinical diagnosis of Stargardt disease, age between 12 and 20 years, at least 1 disease-causing variant in the ABCA4 gene an atrophic lesion size based on DDF within 3 disk areas and a BCVA of 2,200 or better. Subjects were randomized to either oral Tinlarebant at 5 milligrams per day or placebo in a 2:1 ratio. The primary efficacy endpoint was the annualized rate of lesion growth in the aggregate area have definitely decreased autofluorescence from baseline. As measured by outprices imaging at month 25. The key secondary endpoint was the annualized rate of lesion growth in total area of decreased autofluorescence. Other secondary measures included visual acuity, photoreceptor morphology assessed by OCT and pharmacodynamics, namely the change in retinal binding protein 4 RP4 levels from baseline to end of study. Exploratory endpoints include change in retinal sensitivity by microperimetry. This slide shows the results for the biomarker in the systemic circulation, namely percent change from baseline in BPI. The goal of the pharmacodynamic effect was to reach at least 70% reduction in RBP 4. This graph shows that daily dosing of 5 milligrams per day with Tinlarebant, led to a sustained 80% reduction of RPP 4 with very little variability. And RPP-4levels returned to close to the original baseline value by 28 days after treatment with Tinlarebant was discontinued. This shows the primary endpoint of the DRAGON trial, namely the change from baseline in definitely decreased autofluorescence, total area in the study eye, which was both highly statistically significant and clinically meaningful. Applying an unstructured covariance matrix, the treatment effect was 35.7% compared to placebo and yielded a p-value of 0.0033. -- with the first order autoregressive covariance matrix, the treatment effect size was similar at 35.4% and with a p-value less than 0.0001. DDF lesion growth was slowed down to 0.38 millimeter squared per year while a progression of 0.59 millimeters squared per year was measured in the placebo group. To put this into context, in the ProgStar study, as mentioned earlier, the mean DDF lesion growth was 0.74 millimeter squared per year, so almost double. DAF, the sum of DDAF and QDAF represents the full extent of reduced autofluorescence and thereby captures both established atrophy and surrounding areas of earlier retinal pigment epthelime dysfunction. The ProgStar study demonstrated that CDAF converts the DDF over time, supporting its interpretation as an earlier stage of lesion evolution. Consequently, DAF provides a more comprehensive assessment of disease extent than DDF alone and has been established as one of the most sensitive structural measures of disease progression for [indiscernible] studies and interventional clinical trials. As disease progresses, enlargement at DAF is biologically expected to precede subsequent deterioration of retinal function and visual acuity, Consistent with the known sequence of RPE degeneration photoreceptor loss and functional decline. DAF was the key secondary endpoint in the DRAGON trial. It was found that Tinlarebant slowed DAF lesion growth by 33.7% compared to placebo and reach statistical significance. Best corrected vision acuity in the study eye did not show any significant change over the course of 2 years, neither for the Tinlarebant nor the placebo group. This is entirely expected based on natural history. Mean visual acuity at baseline in the Talara bank group was 39.9 ETDRS letters and most 39.7% at the end of study. Similarly, visual acuity was 39.4% for the placebo group at baseline and 40 letters at end of study. An ETDRS letter score of 39 to 43 letters corresponds to 2160 snow and visual acuity. The test retest variability for ETDRS change scores in Stargardt disease yields a repeatability coefficient of approximately 8 letters. So observed changes of 5 to 10 letters are frequently indistinguishable from measurement noise. The observed minor change in average visual acuity over 2 years is in line with the naturality of Stargardt disease, and was observed in the ProgStar study. Importantly, Tinlarebant maintained a well-tolerated safety profile over the 2-year course of treatment. The table here shows systemic safety and tolerability, namely the number of subjects who experienced at least 1 nonocular treatment-emergent adverse event. A total of 6 serious AEs were reported in the study. All events were nonocular with 4 assessed as unrelated and 2 assessors unlikely related to the study treatment. The most reported nonocular AEs were nasopharyngitis, headache and acne. Most events were mild and resolved during the study period. Regarding ocular safety and tolerability. Xanthopsia, which is a temporary yellowish division and delayed dark adaptation, meaning it takes a little longer for the eyes to adjust when moving from light to dark, with the most common drug-related atomic adverse events. The majority of Xanthopsia delayed dark adaptation and night vision impairment were mild. And of note, most resolved while on treatment. There were no serious ocular TEAEs, 4 TAEs led to study drug discontinuation and 2 led to study discontinuation. My experience is very much in keeping with this data that these effects were generally mild and did not represent a concern for the majority of patients. While visual acuity changes in Stargard cannot be detected in short follow-up periods and all relatively small data sets, when using large data sets, such as ProgStar an association of DDAF and visual acuity can be shown in a secondary cross-sectional data analysis using data from 301 ProgStar participants, which was 566 study eyes, it was shown that for QDAF lesions, every 1 millimeter square larger QDAF area was associated with 3.3 letters worse visual acuity for DDAF lesions and lesion area less than 11 millimeters squared, every 1 millimeter larger DDAF area was associated with 1.5 letters worse visual acuity. The DDAF lesions and again, lesion area less than 11 millimeters squared, every 1 millimeter larger DAF area was associated with 0.9 letters worth of visual acuity. In another secondary cross-section analysis of 64 study eyes of 36 ProgStar study participants aged 10 to 18 years, so adolescents, BCVA showed a linear correlation with DDAF lesion area. An increase of 1 millimeter squared in DDAF size was associated with a decline of 0.97 EGR letters in BCVA. Using Micro perimetry, a test that measures how paint a spot of light the retina can still detect, a healthy retina typically scores around 30 to 33 decibels, whereas retinal areas affected by DDAF in the DRAGON trial averaged only 4 decibels. This represents an approximately 400-fold loss of light sensitivity demonstrating that DDAF identifies retinal tissue with profound functional impairment, essentially little to no useful vision. This Kaplan-Meier curve demonstrates the slow, but inevitable loss of visual acuity in Stargardt disease. The slide shows data from a retrospective single-center study of 361 patients with Stargardt disease as the Department of Ophthalmology and Visual Science at the University of Illinois in Chicago. This represents the rationale for a timely intervention to allow for preservation of photoreceptors for the many decades of life remaining for patients affected by Stargardt disease. So in conclusion, I have no doubt that Tinlarebant has the potential to be the first-ever approved treatment for Stargardt disease. It's the first oral therapy in a retinal degenerative disease to demonstrate a clinically meaningful slowdown of neurodegeneration. There's an impressive 36% reduction in DDAF lesion growth rate, representing a statistically and clinically meaningful treatment effect in Stargardt disease. It was well tolerated with a good safety profile, it addresses the root pathogenic mechanism, offering disease-modifying approach where no approved therapies currently exist. Given the mechanism of action and inevitable progressive nature of the disease, Tinlarebant has broad applicability across disease stages from early ABCA4 mediated disease to more advanced atrophy. From a personal point of view, this data far exceeds what I dare to hope for in treating this devastating retinal condition that can affect patients of all ages. To have been involved in the DRAGON trial is a career highlight and I have no doubt at all that Tinlarebant should be approved globally and be made available at the earliest opportunity for patients with ABCA4 retinal dystrophy. Thank you for your attention.

Hello, I'm Dr. Paul Bernstein from the Marine Center of the University of Utah. I'm an inherited retina disease specialist who sees many patients with Stargardt disease, and I'm pleased to represent Belite Bio and talk with you today about the clinical journey of patients with Stargardt disease. The current started disease patient journey involves multiple specialists. The most common presentation of Stargardt disease is in childhood or early adulthood. Initially, symptoms can be settled. These could be poor vision or difficulty reading or doing poorly in school or they may be picked up on vision screening examinations. This will typically lead to referral to an eye care specialist, a general ophthalmologist or an optometrist, who will probably be somewhat familiar with Stargardt disease, having learned about it in their training and seeing patients with this. And they will do an eye examination and basic imaging. -- and that will show signs of changes in the macula consistent with Stargardt disease. Typically, these patients are then referred on to an inherited retina disease specialist if one is available. These specialists can then do sophisticated imaging to stage the disease and to give counseling to the patient. And then there is usually a referral for genetic testing and counseling with a genetic counselor. This allows for transformation because some diseases can simulate Stargardt disease, these are called phenocopies, but not actually be the more common ABCA for genes that accounts for 95% of Stargardt disease. This can then lead to disease management, which we will talk about in the next slide. When a retina specialist receives a new referral for a patient with Stargardt disease, there are various tests that we do to confirm the diagnosis and to understand what stage the disease is in and to then be able to give prognosis for the patient and treatment options. These include basic visual function tests such as best corrected visual acuity, microperimetry and color vision testing. Then the patient will undergo retinal imaging such as fundus photography, optical coherence tomography and fundus autofluorescence. These show typical findings of Stargardt disease, such as Flex or central atrophy, then the patient is referred for genetic testing. We do a large genetic panel usually that covers hundreds of different genes that can cause inherited retina diseases, but these genetic tests concentrate on looking for ABCA4 the gene responsible for Stargardt 1 disease. This is a very large gene and there are many different mutations that can be found in this gene. But genetic testing is very good. And typically, we are successful in finding one or both mutations in this gene, if a patient actually has start our disease. Genetic testing is critical because the presence of other diseases can simulate Stargardt disease, and confirmation has also been very important in various studies to establish treatments for this disease. Once a patient is diagnosed with Stargardt disease, they typically have regular follow-ups, maybe once a year with the inherited retina disease specialist to do functional tests and imaging to monitor progression of the disease. -- and document any visual decline and to counsel the patients and their families on their progress and prognosis. They can also give updates to the family of new treatments that have become available. As families and patients adapt to the disease, frequency decreases over time, but they still often want to touch base with their inherited retina disease specialists every few years. Overall, retina specialists are very familiar with Stargardt disease and comfortable with genetic testing, but general ophthalmologists, the ones who first see the disease have more limited knowledge on Stargardt disease and genetic testing, and that is why inherited retina disease specialty is so important. Imaging is very important for following Stargardt disease and imaging changes can often correlate with visual decline as the disease progresses. In the early stages, shown in the first column here, there are plastic Flex seen as these yellow spots in this picture of a patient's retina and in the macular area of a patient with Stargardt disease. These can sometimes be subtle and hard to see on just a clinical examination. But when we do specialized imaging such as fundus autofluorescence, these flex light up and show us bright bike dots that are classic signs of Stargardt disease. We also do optical coherence tomography or cross sections of the retina. And using OCT, we can see thinning of the retina here that is characteristic of Stargardt disease. With time, these flex can increase in number and then there can be the beginnings of atrophy in the center part of the retina, and this correlates often with visual decline. Shown here in Panel D, you can see an irregular kidney shaped area where photoreceptor cells and the supporting cells called the retinal pigment epithelium have died away, this area would lead to loss of vision in the corresponding spot of the visual field. And if it involves very center will start to lead to loss of central vision, making driving, reading and recognizing faces difficult. Again, this can be subtle, but is very well seen in fundus autofluorescence images as a darker area that's is easily recognized by retina specialist. Concurrently, optical coherence tomography shows even more thinning and loss of critical structures in the outer retina. In Panel G, which would be a very advanced case of the disease, the macula is almost -- is very heavily affected, and visual acuity would be expected to be more. This eye would have worse vision than 2,200. And if this were the case in both eyes, the patient would be legally blind, but possibly still able to read some print if it is very large. Unfortunately, in a few rare cases, there can be complete atrophy of the retina. These would be associated with severe mutations in the ABCA4 gene, and this patient would have very poor vision. This is not the ultimate end for many patients with Stargardt disease. Many of those patients end up with 2,200 vision and still some preservation, although they are visually impaired. Stargardt disease is challenging because as we have understood the pathogenesis of the disease and the genetics, treatments have not caught up as well. Today, there is no approved disease-modifying therapy. And we currently have to give supportive recommendations to the patient. This can include recommending UV blocking glasses and sunglasses to avoid excessive light and sun exposure to the retina. We know based on the mechanism of the disease that excess in vitamin A can be a problem, and we do not recommend use of high-dose vitamin A supplements. There has to be counseling on lifestyle and career choices. Patients need to understand that someday, they may not be able to drive and that they may have difficulty reading unless they have abilities to make print larger. And these low vision ads can be very helpful to the patients but are still not optimal for them. For patients and their caregivers, there is an unmet need. We need to help them to function and be able to read. There's a loss of independence, including inability to drive once they become visually impaired or legally blind, this can have a profound impact for a lifetime and there are other ongoing social challenges of not being able to see as well as their peers. So we are helping patients adapt to vision loss, but it does not change the course of the disease. We are leaving patients and caregivers to navigate a profound burden largely alone. But we are hopeful as new treatments come online that these supportive cares will be less and less needed as vision is preserved for longer and longer in their lives. As an inherited retina disease specialist who has taken care of Stargardt patients for more than 31 years, these are very exciting times. Stargardt disease has been a condition where until now, I have been only able to offer counseling and supportive care. If treatments are approved in the near future, this will allow us to offer more options for patients that may help preserve vision for longer and will allow less loss of independence and longer time with good vision throughout their life. Thank you.

I'm Hendrik Scholl, the Chief Medical Officer of Belite Bio. Being a retinal specialist myself, I have cared for patients with stage disease for more than 2 decades. And I've seen firsthand what the progressive loss of vision means for their lives. Having also spent many years studying the natural history of this disease, we have waited a very long time for a treatment that could change its course. Today, it feels as though we may finally be approaching that moment. I would like to step back and ask a fundamental question. How important is vision to people? Ultimately, -- the value of a therapy is determined not only by clinical trial results, but by the difference it makes in people's lives. To explore this, researchers and former colleagues of mine at Johns Hopkins Medicine, conducted a nationwide survey of more than 2,000 U.S. adults, representing the general population. Participants were asked to rank a range of serious health conditions according to which they would consider the worst to experience. The results are remarkable. Blindness ranked among the most field health conditions ahead of heart disease, loss of a limb, deafness, arthritis and HIV and essentially alongside cancer and Alzheimer's disease. Importantly, this pattern was remarkably consistent across all major ethnic groups. This tells us that preserving vision is not simply another medical objective. It protects one of the aspects of health that people value most. The next question is why blinded is perceived as so devastating. The same study asked participants which health outcome would have the greatest impact on their everyday lives. Here, the message is even clearer. Nearly half of all respondents identified loss of eyesight as the condition that would have the greatest impact on day-to-day life, well ahead of losing memory, a limb, speech or hearing. This finding reflects something that clinicians see every day. Vision is central to independence, mobility, communication, education, employment and social interaction. When Vision is progressively lost as in stage disease, the consequences extend far beyond the eye itself. That is why therapies capable of preserving vision have the potential to create meaningful value by slowing disease progression and helping patients maintain independence. As the most common juvenile macular degeneration, stage disease deprives patients of vision during education, career development and family life, creating a profound lifelong burden. I have seen these patients for more than 2 decades, starting in Germany, then the U.K., then the U.S. and now in Switzerland. This slide shows a video derived from a sequence of autofluorescence images over a period of 4 years in a patient affected by stage disease. He was a patient of mine at the Wilmar Eye Institute at Trans Hopkins in the years 2010 to 2016 and also participated in the ProgStar study. What we can see in the image is a central area of abnormal order presence. There are some white is spots, and these are caused by toxic bisretinoid accumulation and there is a round area of strongly decreased signal where the auto retina degenerated in such areas that we call definitely decrease or depresses or DDAS, the patient can't see any more. There was a continuous growth of this area over time and the patient lost visual acuity from 2040 to 2080 over 4 years. Reading ability for newspaper print is typically lost at a visual acuity of 2050 or lower. The continuous progression seen in this patient illustrates but there is no stage at which stage disease becomes biologically inactive. -- every year of untreated progression results in irreversible retinal cell loss. Confidently, a therapy capable of slowing this process would be expected to provide benefit throughout the disease continuum supporting treatment as soon as the diagnosis is established and continued irrespective of disease stage. Stargardt disease is classified as a rare disease, but it's important to put that into perspective. Rare does not necessarily mean uncommon. Based on current genetic epidemiology, we estimate that approximately 47,000 to 59,000 people in the United States are living with stage disease it similarly substantial patient populations across Europe and China. I would like to close with one simple message. This slide puts Stargardt disease into perspective. although it is classified as a rare disease, ABC F4 associated stage disease represents one of the largest genetically defined patient populations in medicine. Importantly, ABCA4 is also the single most prevalent disease gene in inherited retinal diseases worldwide, as shown on the upper right, making it the largest genetically defined target in this field. Patients generally have a normal life expectancy. So the number of people living with the disease continues to accumulate over time. Yet today, there is no approved disease-modifying therapy. This combination is compelling a highly validated genetic target, the largest opportunity in inherited retinal disease, a substantial addressable patient population and significant unmet medical need. We believe Belite Bio is uniquely positioned to establish the first disease-modifying treatment for stage disease and to become the leader in this important therapeutic area. Now I'd like to turn to our Chief Commercial Officer, Kelly Kilpatrick, to talk about our commercial plans.

Thank you very much, Hendrik. Good morning. I'm Kelly Kilpatrick, Chief Commercial Officer here at Belite Bio. As you've heard, Stargardt disease Type 1 represents a significant unmet need and opportunity. Our commercial team is focused on readiness and enabling the successful U.S. launch of Tinlarebant so that we can help as many patients as possible. I will take you through our market research, focus areas and why we believe our team is well positioned to execute. We believe the U.S. market opportunity is significant. -- and we are encouraged by the potential to bring forward a first-in-class treatment to patients who have been waiting for a treatment option. Stargardt disease Type 1 is the most common inherited retinal disease. Based on current estimates and through our work with Varana Health and the IRIS Registry, it is estimated there are approximately 53,000 people in the United States living with Stargardt Disease Type 1. And roughly 20,000 having been clinically diagnosed today. Of those 20,000, we believe about 50% have received a confirmatory genetic test, and this will be important for payer coverage. Importantly, we view the currently diagnosed and genetically confirmed population as a starting point. As awareness increases and a potential treatment becomes available, we believe there is an opportunity to bring even more patients into the diagnostic and testing pathway. As we prepare for a potential approval, our focus is clear. ensuring we're ready to execute a disciplined launch and shaping market conditions that will be critical for driving adoption. I will now walk you through how we are preparing our commercial organization. We've built a commercial organization designed to execute once approved. Our team brings deep expertise in rare disease, ophthalmology and commercialization. Together, we bring collective experience across more than 50 rare disease product launches. Our team is centered on the patient journey, ensuring we engage the right stakeholders at the right time from diagnosis through treatment. Our sales team is focused on retina specialists who will be the center of treatment. Complementing that effort, our diagnostic specialists provide unbranded education on genetic testing for inherited retinal disorders and educating physicians on available testing options. Our marketing team is focused on disease awareness today and will transition to branded education and demand generation across health care professionals and patient communities following approval. On the market access side, we're focused on payer coverage, reimbursement and patient support services. Our commercial operations team brings expertise in analytics, systems and field support to enable execution across the organization. Our full team collaborates compliantly with medical affairs and patient advocacy. Taken together, we've built an integrated organization aligned around a single objective, engaging the stakeholders who matter most. This positions us for a successful launch once approved. Our commercial strategy is grounded in extensive qualitative and quantitative market research. We have conducted interviews with payers, patients and HCPs across the United States. Given the high unmet need in Stargardt disease and since there's no approved therapies today, -- this creates a meaningful first-mover opportunity for Tinlarebant. The market research also tells us Stargardt disease and genetic testing awareness is already high among retinal specialists. There is an opportunity in the broader ophthalmology community where targeted education can help shorten the diagnostic journey. In addition, we are hearing encouraging feedback from the medical community about Tinlarebant's profile. And our focus is translating that enthusiasm into adoption. Also, while retina specialists are expected to be the primary prescribers there may be an opportunity for ophthalmologists to support these patients seeking treatment as well. Finally, our research helps us understand where patients can encounter some friction along the journey, particularly reaching the appropriate specialist in obtaining genetic confirmation. These are identifiable, addressable steps, and they directly inform where we are focusing our commercial preparation. Overall, our research reinforces 3 things: one, a significant unmet need; two, a treatment option physicians can believe in; and three, a clear road map for where we focus our efforts. Informed by our research and experience, we have identified 4 key focus areas that will be critical to success. These focus areas are designed around the steps required to move a patient from identification through treatment and long-term support. Ahead of approval, our focus is on increasing awareness of Stargardt disease through our medical team and educating the field on IRD genetic testing options. This includes advancing disease education and awareness of the unmet need, while partnering with organizations to extend our outreach. The second area of focus is access. We're working to ensure optimal market access to Tinlarebant's for patients. Our strategy is focused on the high unmet need, broad formulary coverage, pricing and affordability, seamless patient provider support along with a well-defined distribution model to address U.S. demand. Post approval, we ship to launch execution. Our goal is to drive adoption and establish Tinlarebant as the standard of care for Stargardt disease Type 1. This would be the first and only approved once-daily oral therapy. Lastly, but importantly is the patient experience. We're designing a comprehensive patient support program to simplify onboarding, support affordability and help patients stay on therapy over the long term. Together, these 4 focus areas create a clear readiness and launch road map for Belite. Now I'm going to cover each of the focus areas in a little more detail. Awareness is about ensuring the market understands Stargardt disease, its biology and the significant unmet need. Our key audience includes health care providers, payers, patients and caregivers. For health care providers, it's about increasing disease awareness and education around genetic testing in inherited retinal disease. For payers, it's ensuring they understand the burden of disease well before formulary coverage decisions are made. With that foundational understanding established, we then share compliant preapproval clinical information with formulary decision-makers. And for patients and caregivers, it's about building an informed and engaged community. We're executing an unbranded disease education campaign amplified by partnerships with patient advocacy organizations like prevent blindness and FFB, the foundation fighting blindness. These efforts support better disease understanding, increased recognition of the unmet need and a market that is prepared for a new treatment option prior to launch. Awareness also has a second piece, and it is genetic testing. Genetic confirmation is an important part of the pathway and the testing and environment has evolved tremendously over the years. Today, there are multiple testing avenues available to physicians and patients with several ways to collect the sample. These options also create more affordable opportunities for HCPs and patients who are seeking genetic testing. Our diagnostic specialists are focused on helping physicians understand the options for patients suspected of having an inherited retinal disorder and helping them understand the process. In a market where there has been significant unmet need it is understandable there has been less urgency to pursue confirmatory testing. Our objective is to educate physicians on genetic testing and to incorporate that into patient care. As we shared earlier, our research shows payers also recognize there is a high unmet need in Stargardt disease. We're highly focused on the pharmacy benefit managers. Given there is no Stargardt specific ICD-10 code yet, we are working with payers to help them understand their Stargardt patient population. The team's concentration will be to ensure that payer formulary criteria is written to the FDA label. We anticipate payers will require a confirmatory ABCA4 genetic test. This is consistent with other genetic diseases, and it connects to the genetic testing education we are conducting with HCPs. Overall, we're engaging payers early to minimize barriers at launch. Post approval, our focus shifts to driving adoption, activation and patient support. In regards to driving adoption and activation, our efforts are built on 2 promotional engines, personal and nonpersonal. Our targeting is data-driven. Within this priority HCP universe, personal promotion with field-based and remote territory business managers will focus on top priority HCPs who we expect will be the highest prescribers. Nonpersonal promotion will allow us to reach the entire priority HCP universe shown here through omnichannel marketing including educational websites, such as disease state and a branded website post approval. Other examples are paid social efforts and a paid search like Google and AI. The field focus will be with the top centers and key HCPs, and everyone else is reached through nonpersonal efforts. The patient experience is a critical focus area because our goal is to make this journey from prescription to treatment as seamless as possible for both patients and prescribers. Belite is committed to supporting affordability for eligible patients, and we are designing specific programs to help support our patient population. In addition, we're building a comprehensive patient services model with a specialty pharmacy partner who has distinct expertise in ophthalmology in rare disease. All of this is designed to support patients who are on therapy throughout the treatment journey from onboarding through continuity of care. Importantly, we are being mindful of the unique needs of a visually impaired patient and looking for ways to make receiving and staying on therapy as easy as possible. One example is that eligible patients will be able to receive Tinlarebant directly at their home, reducing the need for additional trips to a pharmacy and removing a potential logistical barrier to treatment. We also believe a strong specialty pharmacy partner and patient support model can play an important role in adherence. Because Tinlarebant is intended to be a daily treatment maintaining compliance will be critical to success. The patient support team will proactively help identify and address potential barriers throughout the patient journey. For example, if a patient's insurance changes, they can help navigate potential access considerations and work to minimize any disruption in care. The goal is a seamless experience to help eligible patients start therapy efficiently, navigate access and other considerations and remain on treatment. Our overall approach reflects Belite's values and commitments to the Stargardt community. And importantly, we also believe it enables successful commercialization. Here at Belite, we have been preparing for this moment, and we are prepared to launch. We have an opportunity to bring a transformative treatment option to star our community and meet the needs of a patient population that has been underserved for way too long. As you heard today, Stargardt disease is the most common inherited retinal disorder and the global market opportunity is significant. In the U.S. alone, it is estimated there are approximately 20,000 patients who have received a clinical diagnosis, giving us a strong starting point. The impact of Stargardt disease is profound and bringing forward an oral treatment option has the potential to meaningfully impact the quality of life for people living with this condition. We want to establish a new standard of care with Tinlarebant. As we approach PDUFA and the next milestones, we are confident we have brought together a highly capable organization and built a strategic plan to enable a successful U.S. launch. Our commercial infrastructure, market access strategy targeting approach and patient support model are designed to create a successful launch and ensure we can reach the patients, physicians and stakeholders who will drive adoption. And in closing, I want to thank Tracy and Heva for sharing their stories with us. Hearing from them is what motivates all of us at Belite to make a difference. And thank you, Dr. Hendrik Scholl, our Chief Medical Officer; and our 2 guests, Dr. Michel Michaelides and Dr. Paul Bernstein for their expertise. As we look forward, we will continue to engage across the Stargardt community as these insights are vital to our work. We are very excited about what lies ahead and committed to a high standard of execution. We've done the market research, the strategy is in place and the team is ready to deliver for patients. Thank you again for joining. This concludes our presentation. We will now open it up for a question-and-answer session.

[Operator Instructions]. Your first question comes from the line of Judah Frommer with Morgan Stanley.

It was very helpful as we head into potential approval here. A couple of questions for us that I guess both the doctors and the company could address. I guess, first, on expectations for breadth of use across ages and stages of Stargardt disease. Do you think Tinlarebant could be used fairly broadly across patient subpopulations. Do you think it could be used in patients with more advanced vision loss would those patients see progression? And how long do you think patients might be on drug?

Hendrik do you want to take that question?

Certainly. So we have to be clear, the clinical inclusion criteria that shows in the DRAGON trial because patients needed to have a baseline DD flation that was evaluable in order to be able to measure lesion growth during the course of the study period. So beyond best corrected acuity, of 2200, which corresponds to legal blindness as defined in the U.S. there exists a significant amount of remaining vision that is useful to patients. And research shows that the lesion growth profiles are not dramatically different between children and adults, and there is no evidence to suggest that there would be a treatment difference between those 2 patient populations. So for those reasons, we believe there could be a potential of clinical benefit for those who have more advanced vision loss, but I would also like to invite Dr. Bernstein to maybe add additional considerations about patients that are more advanced.

Yes. It is well understood that this disease progresses even past legal blindness as Dr. Scholl has said, there is strong interest. The mechanism is still the same, and there is -- no reason to think that continued use of this drug would not slow down the -- continue to slow down the disease and have clinically meaningful results for the patients. So I would anticipate that this would be used in all stages of the disease and not be limited to just the medium stages that were targeted in the study.

Thank you Bernstein, I would like to get to the other question, the second question that is Judah I younderstood correctly. The question is how long would patients be expected to remain on treatment. So again, right, Dragon provides 2 years of treatment data. This is the limitation. It's a fairly long trial, but it's still only 2 years. the approved label and physician judgment in the future will determine use in duration. But we would anticipate long-term usage for obvious reasons. It's also important to understand what happens after treatment stocks. So in our clinical trial, RP4 remained suppressed during dosing and return towards baseline after treatment ended, which is consistent with the reversible pharmacodynamic effect. So again, right, we would anticipate long-term treatment -- and again, I would like to invite Dr. Bernstein to add additional thoughts what he anticipates is going to happen when Tinlarebant becomes available for Stargardt patients.

Yes. I think the patients understand and have seen the progression of the disease as they usually have started with good vision in their younger years and have noted the relentless progression associated with ABCA4 disease. I think they will be very motivated based on the results of the DRAGON study to stay on this and to try to preserve as much vision as they have for a long period of time. So I think there will be many, many years of use of this drug in all patients that are eligible for it.

Great. And maybe just a follow-up on the market research efforts. I guess kind of more broadly, what has physician interest look like in terms of potential interest for prescribing Tinlarebant in the commercial setting? What can you tell us about both level of interest and maybe potential penetration within particular practices based on the research the company has done.

Sure. I'll take that question. As we talked about, we believe Stargardt disease represents a significant unmet need that clearly came through with our market research not only with HCPs, but also with payers. We're encouraged by the FDA's acceptance of our NDA with priority review. In regards to eligibility, as you heard earlier, we estimate there's approximately 53,000 patients in the U.S. living with Stargardt disease. With roughly 20,000 clinically diagnosed today, approximately 50% of them have received confirmatory genomic tests. The market research also states that our inherited retinal specialists and retina specialists in general, very familiar with Stargardt disease and genetic testing. They're encouraged by the profile of Tinlarebant that they've seen. But I think the important point here is the diagnosed and confirmed population, we recognize that as a starting point. And in addition, the market research that has also indicated because there isn't an approved therapy, there can be less urgency to genetically test, but we believe bringing a new treatment available can help improve the testing rates. I think it will depend prescribing habits will change over time depending on what the final label and payer balance turn out to be.