Ladies and gentlemen, thank you for joining us and welcome to the Belite Bio 2026 Commercial Day. After today's prepared remarks, we will host a question and answer session. If you would like to ask a question, please raise your hand. If you have dialed into today's call, please press star nine to raise your hand and star six to unmute. 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, how Stargardt impacts people's day-to-day lives, the significant unmet need for treatment, and Belite 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 Stargardt disease type 1. At Belite Bio, our mission is to preserve vision and pioneer degenerative retinal disease treatments where few or none exist today. We are approaching one of the most significant milestones in Belite Bio's history. As we recently announced, FDA has accepted our NDA for tinlarebant 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 Stargardt disease. Furthermore, we believe this reinforces the quality and depth of the data that we have generated throughout this drug development program. If approved, tinlarebant will be the first and only approved treatment for Stargardt disease type 1. Approval would also mark Belite's evolution into a commercial-stage 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 retinal community. We have been building the capabilities needed to support a successful U.S. launch. From commercial infrastructure and market preparation to 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, ophthalmology, and commercialization. Joining us today are Belite Bio's Chief Medical Officer, Dr. Hendrik Scholl, Chief Commercial Officer, Kelly 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 Belite team is well-positioned to execute successfully. Also with us today, you'll hear from Dr. Michel Michaelides, who will provide an overview of tinlarebant and the unmet medical need in Stargardt disease. Dr. Paul Bernstein, who will discuss the current treatment landscape and the opportunity to define the standard of care. From the Belite team, Dr. Hendrik Scholl will provide an overview of the global Stargardt opportunity, and Kelly Kilpatrick will discuss our U.S. commercial launch strategy. We'll also have time at the end for Q&A. First, we are honored to welcome Tracy and [Hava], who are both living with Stargardt disease type 1, to share their personal journey. Hearing their story reminds us 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 [Hava]. I began to not be able to have enough light in an already lit 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 accidents. It did not dawn on me that it was my vision. I always assumed it to be the other driver. But my last car accident really woke me up, and that's when I decided to go get my eyes checked. When I was reading the chart, all I could see was the letter E, the biggest letter on the chart. The doctor was saying how I only was doing that to get a pair of glasses since my little brother got a pair. I was eight years old. I didn't understand what full vision meant and being able to see that meant. So he cut my prescription in half. From there, I wore the glasses, and one 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 was still looking very close to my computer. So that is what really made my mom was like, "Okay, something's wrong here. She's not- She's not reading well, she's not playing basketball well. They think she has a learning disability. Just all these little things started to add up, even though I had a pair of glasses. That really was a huge point where I was diagnosed. To be honest, when I was explained what Stargardt disease 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 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, two years, I'll even say, I began to notice that the vision was getting worse, because I couldn't see faces clear anymore, and reading my phone was more challenging. That's pretty much, I think, the time span that it took for me to accept what has happened to me. I was sitting there, nine years old, and I remember this so clearly. I was dancing in the doctor's office, singing a random song, and the doctor came in, and they looked at us, and they were like, "You're going to have to pull [Hava] 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 at nine years old with Stargardt disease, is being told some of the worst news of my life. I went to school with my two cousins, and just not being able to drive, knowing when I turned 16, I wouldn't be behind a car. And that's all I thought about with my eye disease. I didn't understand what it was. No one explained to me what Stargardt was, just the things that I couldn't do. I love reading. I can't read a paperback 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 audiobooks, but there's nothing like turning a page of a book. 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 little things I know I won't be able to do with my kiddos. I would definitely say 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 bed to the time that I get back in the bed. Living with this vision impairment, for me, is a daily challenge. Getting dressed, identifying colors in the clothes has become a challenge for me. Preparing meals, organizing the spices, making sure that I don't harm 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 aisle 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 called to go purchase my groceries, challenging. Getting in an Uber ride, scary. Not knowing if the driver takes a different route that I'm unfamiliar with, unsettling. Socializing with friends that are sighted, 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 forkfuls of air or not, and learning how to get to the restroom out there. Feeling comfortable while they share pictures on their phones with me I think that overall it's all a challenge, 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 someone's at the window of that house. I would say it definitely takes a little bit of a mental drain on me sometimes just because I'm 18. That's one thing I've struggled with, is going out with friends and 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. That's definitely been something I've personally struggled with a lot, is hanging out with friends and not knowing who's all there because of my vision. I've definitely struggled with asking for accommodations just because sometimes I get tired of it, and I just want to live a normal life. 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. Even cutting my food in restaurants, in a dim restaurant, has always been a struggle for me, so I always navigate towards finger food. When my mom knows I want a steak, she'll order me it and cut it up for me. Just little things really suck sometimes, but the people I surround myself with 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. In the back of my head, it's always that I wish I could see them. The most heart-jerking for me is having conversations with my daughter, Laura, together. We have a great time, but not being able to see her face, it hurts me so much. I think that if I could get that back, or another one, reading a book in bed. How I miss that. I miss that more than driving. Just being able to sit in the bed with a cup of tea and reading a good old book, that's something that I miss. I appreciate auditory versions of reading, but using my physical eyes for the things that I enjoy doing, like crafting or doing eye shadow on my eyes, miss it 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 Stargardt 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, learning to trust themselves, and then learning to believe in themselves. Not being afraid to ask for help, not being ashamed or angry when they make mistakes. Never giving up, and then also never giving up on a hope that there will one day 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 fundus flecks within the posterior pole due to the accumulation of lipofuscin. It's caused by disease-causing variants in the gene ABCA4, 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 disc membranes of photoreceptors after photobleaching of visual pigment. When this is malfunctioning, these byproducts are trapped within the photoreceptor outer segments. As the RPE phagocytosis the outer segments on a daily basis, these byproducts eventually accumulate within the RPE and react to form A2E, a dimer of vitamin A, which eventually leads to the generation of lipofuscin and related toxic bisretinoids, which result 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 adulthood, 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 affects 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 Eye Hospital was one of the leading international study centers, and I had the privilege to serve as one of the PIs and contributors to the ProgStar study group. Using standardized fundus autofluorescence imaging, ProgStar demonstrated that DDAF, definitely decreased autofluorescence, 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 serial autofluorescence images above. By combining DDAF and QDAF, one has a total decreased autofluorescence, DAF, 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-based measures are highly reproducible, correlate with functional decline, and are suitable for detecting progression over 12 to 24 months. As a result, DDAF, QDAF, 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 DDAF growth rate in the ProgStar cohort over 24 months was 0.74 mm sq per year. DDAF growth as an outcome measure has been accepted by the FDA 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 two years. If we look specifically at those with a baseline BCVA between 20/70 and 20/200, 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. 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 one disease-causing variant in the ABCA4 gene, an atrophic lesion size based on DDAF within three disc areas, and a BCVA of 20/200 or better. Subjects were randomized to either oral tinlarebant at 5 mg per day or placebo in a 2:1 ratio. The primary efficacy endpoint was the annualized rate of lesion growth in the aggregate area of definitely decreased autofluorescence from baseline, as measured by autofluorescence 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 retinol- binding protein 4, RBP4, levels from baseline to end of study. Exploratory endpoints included change in retinal sensitivity by microperimetry. This slide shows the results for the biomarker in the systemic circulation, namely percent change from baseline in RBP4. The goal of the pharmacodynamic effect was to reach at least 70% reduction in RBP4. This graph shows that daily dosing of 5 mg per day with tinlarebant led to a sustained 80% reduction of RBP4 with very little variability. RBP4 levels 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 a first-order autoregressive covariance matrix, the treatment effect size was similar at 35.4% with a p-value less than 0.0001. DDAF lesion growth was slowed down to 0.38 mm sq per year, while a progression of 0.59 mm sq per year was measured in the placebo group. To put this into context, in the ProgStar study, as mentioned earlier, the mean DDAF lesion growth was 0.74 mm sq 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 epithelium dysfunction. The ProgStar study demonstrated that QDAF converts to DDAF over time, supporting its interpretation as an earlier stage of lesion evolution. Consequently, DAF provides a more comprehensive assessment of disease extent than DDAF alone and has been established as one of the most sensitive structural measures of disease progression for natural history studies and interventional clinical trials. As disease progresses, enlargement of 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 reached statistical significance. Best corrected visual acuity in the study eye did not show any significant change over the course of two years, neither for the tinlarebant nor the placebo group. This is entirely expected based on natural history. Mean visual acuity at baseline in the tinlarebant group was 39.9 ETDRS letters and was 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 - 43 letters corresponds to 20/16 Snellen visual acuity. The test-retest variability for ETDRS change scores in Stargardt disease yields a repeatability coefficient of approximately eight letters. So observed changes of 5- 10 letters are frequently indistinguishable from measurement noise. The observed minor change in average visual acuity over two years is in line with the natural history of Stargardt disease and was observed in the ProgStar study. Importantly, tinlarebant maintained a well-tolerated safety profile over the two-year course of treatment. The table here shows systemic safety and tolerability, namely the number of subjects who experienced at least one non-ocular treatment-emerging adverse event. A total of six serious AEs were reported in the study. All events were non-ocular, with four assessed as unrelated and two assessed as unlikely related to the study treatment. The most reported non-ocular 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 tint to vision, and delayed dark adaptation, meaning it takes a little longer for the eyes to adjust when moving from light to dark, were the most common drug-related ophthalmic 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. Four TAEs led to study drug discontinuation and two 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 Stargardt cannot be detected in short follow-up periods and/or 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 mm sq larger QDAF area was associated with 3.3 letters worse visual acuity. For DDAF lesions and lesion area less than 11 mm sq, every 1 mm larger DDAF area was associated with 1.5 letters worse visual acuity. For DAF lesions, and again, lesion area less than 11 mm sq, every 1 mm larger DAF area was associated with 0.9 letters worse visual acuity. In another secondary cross-sectional analysis of 64 study eyes of 36 ProgStar study participants aged 10 - 18 years, so adolescents, BCVA showed a linear correlation with DDAF lesion area. An increase of 1 mm sq in DDAF size was associated with a decline of 0.97 ETDRS letters in BCVA. Using microperimetry, a test that measures how faint a spot of light the retina can still detect, a healthy retina typically scores around 30- 33 dB, whereas retinal areas affected by DDAF in the DRAGON trial averaged only 4 dB. 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 at the Department of Ophthalmology and Visual Sciences 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. 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 dared 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 Moran Eye Center of the University of Utah. I'm an Inherited Retinal 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 Stargardt disease patient journey involves multiple specialists. The most common presentation of Stargardt disease is in childhood or early adulthood. Initially, symptoms can be subtle. 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 seen patients with this. 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 retinal disease specialist if one is available. These specialists can then do sophisticated imaging to stage the disease and to give counseling to the patient. Then there is usually a referral for genetic testing and counseling with a genetic counselor. This allows for confirmation because some diseases can simulate Stargardt disease, these are called phenocopies, but not actually be the more common ABCA4 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 retinal 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 flecks 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 retinal 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. Genetic testing is very good, and typically, we are successful in finding one or both mutations in this gene if a patient actually has Stargardt 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 specialist every few years. Overall, retina specialists are very familiar with Stargardt disease and comfortable with genetic testing. General ophthalmologists, the ones who first see the disease, have more limited knowledge of 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 classic flecks seen as these yellowish 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. When we do specialized imaging, such as fundus autofluorescence, these flecks light up and show as bright white dots that are classic signs of Stargardt disease. We also do optical coherence tomography or cross-sections of the retina. Using OCT, we can see thinning of the retina here that is characteristic of Stargardt disease. With time, these flecks 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 the 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 is easily recognized by a 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 very heavily affected, and visual acuity would be expected to be poor. This eye would have worse vision than 20/200, 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 20/200 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 excessive 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. These low vision aids 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 is 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. 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. 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 Retinal 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 Stargardt disease for more than two decades, and I have 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 feared 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 blindness 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 Stargardt 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, Stargardt 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 two 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 four years in a patient affected by Stargardt disease. He was a patient of mine at the Wilmer Eye Institute at Johns 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 autofluorescence. There are some whitish spots, and these are caused by toxic bisretinoid accumulation. There is a round area of strongly decreased signal where the outer retina degenerated. In such areas that we call definitely decreased autofluorescence, or DDAF, the patient can't see anymore. There was a continuous growth of this area over time, and the patient lost visual acuity from 20/40 to 20/80 over four years. Reading ability for newspaper print is typically lost at a visual acuity of 20/50 or lower. The continuous progression seen in this patient illustrates why there is no stage at which Stargardt disease becomes biologically inactive. Every year of untreated progression results in irreversible retinal cell loss. Consequently, a therapy capable of slowing this process would be expected to provide benefit throughout the disease continuum, supporting treatment as soon as a diagnosis is established and continued irrespective of disease stage. Stargardt disease is classified as a rare disease, but it is important to put that into perspective. Rare does not necessarily mean uncommon. Based on current genetic epidemiology, we estimate that approximately 47,000- 59,000 people in the United States are living with Stargardt disease, with 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, ABCA4-associated Stargardt 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 in 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 Stargardt disease, and to become the leader in this important therapeutic area. Now I like to turn to our Chief Commercial Officer, Kelly Kilpatrick, to talk about our commercial plans. Thank you very much, Hendrik. Good morning. I am Kelly Kilpatrick, Chief Commercial Officer here at Belite Bio. As you have 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 Verana 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 healthcare 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 and obtaining genetic confirmation. These are identifiable, addressable steps, and they directly inform where we are focusing our commercial preparation. Overall, our research reinforces three things. One, a significant unmet need. Two, a treatment option physicians can believe in. And three, a clear roadmap for where we focus our efforts. Informed by our research and experience, we have identified four 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 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 shift 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 four focus areas create a clear readiness and launch roadmap 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 healthcare providers, payers, patients, and caregivers. For healthcare providers, it's about increasing disease awareness and education around genetic testing and 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 pre-approval clinical information with formulary decision makers. 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 environment has evolved tremendously over the years. Today, there are multiple testing avenues available to physicians and patients with several ways to collect a 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 two promotional engines, personal and non-personal. 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. Non-personal promotion will allow us to reach the entire priority HCP universe shown here through omni-channel 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 non-personal 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 are building a comprehensive patient services model with a specialty pharmacy partner who has distinct expertise in ophthalmology and 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. 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 the Stargardt 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. In closing, I want to thank Tracy and [Hava] for sharing their stories with us. Hearing from them is what motivates all of us at Belite to make a difference. Thank you, Dr. Hendrik Scholl, our Chief Medical Officer, and our two 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. We will now begin the question and answer session. If you would like to ask a question, please raise your hand now. If you have dialed into today's call, please press star nine to raise your hand and star six to unmute. Please stand by while we compile the Q&A roster. Your first question comes from the line of Judah Frommer with Morgan Stanley. Your line is open. Please go ahead. Hi. Good morning, everyone. Thanks for putting on this event. It was very helpful as we head into potential approval here. A couple 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? How long do you think patients might be on drug? Thanks. Hendrik, you want to take that question? Certainly. We have to be clear, the clinical inclusion criteria were chosen in the DRAGON trial because patients needed to have a baseline DDAF lesion that was evaluable in order to be able to measure lesion growth during the course of the study period. Beyond best corrected visual acuity of 20/200, which corresponds to legal blindness as defined in the U.S., there exists a significant amount of remaining vision that is useful to patients. Research shows that 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 two patient populations. For those reasons, we believe there could be a potential of clinical benefit for those who have more advanced vision loss. 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 continue to slow down the disease and have clinically meaningful results for the patients. 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. Great. Thanks. Thank you, Dr. Bernstein. I would like to get to the other question, the second question that is, Judah, if I understood correctly. The question is how long would patients be expected to remain on treatment? Right. DRAGON provides two years of treatment data. That is the limitation. It's a fairly long trial, but it's still only two years. The approved label and physician judgment in the future will determine use and duration. But we would anticipate long-term usage for obvious reasons. It's also important to understand what happens after treatment stops. So in our clinical trial, RBP4 remained suppressed during dosing and returned toward baseline after treatment ended, which is consistent with the reversible pharmacodynamic effect. 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 as long a 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. Maybe just to follow up on the market research efforts, I guess kind of more broadly, what has physician interest looked 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's done? Thanks. 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 genetic tests. The market research also states that our inherited retinal specialists and retina specialists in general are very familiar with Stargardt disease and genetic testing. They're encouraged by the profile of tinlarebant that they've seen. I think the important point here is the diagnosed and confirmed population, we recognize that as a starting point. In addition, the market research that has also indicated, because there isn't an approved therapy, there can be less urgency to genetically test. 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 policies turn out to be. Kelly- Thanks. Maybe we have this great opportunity to have two of the most famous physicians treating this disease in the world, based in the U.S. and in the U.K. Maybe they could comment also on their enthusiasm and what they have heard, because they are extremely well-connected. Absolutely. Okay, I would be happy to go first. There is strong pent-up interest in finally having a treatment for this disease. It is very familiar to retina specialists, inherited retina disease specialists, and it has been very frustrating that we have watched these patients through our practices and through the many years we have been in practice to see them progressively lose vision. It is very exciting to finally have a treatment that may be available soon. The patients likewise are very motivated and interested. They, in this age of social media, especially in the target range for many of these patients with Stargardt disease, are very informed. I am certain are very excited about these opportunities. Thank you, Paul. From a U.K. perspective, I am anticipating we are going to get overwhelmed with interest and excitement about starting to take this therapy to extend useful vision for as long as possible. Certainly, patients are contacting us wanting to come into clinic sooner, chasing us to find out when it will become available. If it becomes available in the U.S. first, could they get access to it through the U.S. and any contacts there? The patient organizations are incredibly sensitized and enthusiastic about this. Stargardt's Connected, which is a well-established Stargardt patient group, is planning to showcase the potential benefits in tinlarebant. I have got no doubt this is going to be very well received by the patients. As Paul was saying, our colleagues, our retina specialists and IRD specialists are very much looking forward to the opportunity to finally be able to prescribe something that could change our patients' lives. Thank you. Your next question comes from the line of Tazeen Ahmad with Bank of America. As a reminder, please press star 6 to unmute. Your line is open. Please go ahead. Good morning. Thanks so much for making this presentation, and thanks for taking my questions. For the company, I wanted to get your thoughts about the pace with which this launch will progress. So, let's say that you get your on-time approval in February. Are you able to start marketing the products here in the U.S. right away? Taking into account, of course, the lack of treatments available and that the obviousness of patients wanting to get on therapy, just based on negotiations that you're going to have to make with the payers, how long initially do you think it's going to be before you're able to have doctors write a script and have a patient be commercially reimbursed? How much of that time do you think Belite might have to assist patients with reimbursement before you get full coverage? For the physicians on the call, I wanted to ask your thoughts about the adverse event of delayed dark adaptation and the night vision impairment. What, in your view, is the seriousness, if you will, of this event? Is this going to be in any way a detractor, maybe not for you, but for patients from wanting to take this treatment? Thanks. Thanks, Tazeen. I'll take the first two parts of that three-part question in regards to the commercial launch. We expect we'll have at least 50 customer-facing team members in the field post-approval. We believe we have a strong launch team built on individuals with rare disease and launch experience. If necessary, we could scale further based on the business needs. But in regards to how quickly we can do that, we're committed to a timely and effective launch. We remain focused on all of our preparation activities. As you've heard, we've completed extensive market research with payers, providers, and patients. We've identified four core pillars around awareness and genetic testing, access, adoption, and the patient experience. We feel we are very well positioned to execute, if approved, in a very quick manner. In regards to payers, which is a very good question, obviously they're really important to our launch. The feedback that we heard in our research indicates that payers generally recognize the significant unmet need of Stargardt disease, and we're preparing appropriate pre-approval engagement to establish the disease in clinical context. It's too early, I would say, to predict specific coverage policies before approval and the final label. But we are working with our medical teams, with our national account team, preparing compliant pre-approval engagement with all the formulary decision makers to establish the burden of disease, the relevant clinical context before coverage decisions are made. In parallel, we're building the reimbursement and patient support infrastructure needed for launch. Our conversations are ongoing now, and they will continue, and we'll continue to educate them on tinlarebant, the benefits of tinlarebant, working with our medical affairs team. As their medical policies are created, we will work with them on the criteria along with our approval process with especially pharmacy. In regards to- I'm happy to take the third question, right on a very important question to see on the side effect profile. tinlarebant was generally very well-tolerated in the DRAGON trial. Most ocular and non-ocular treatment-emergent adverse events were mild, and the majority resolved while patients remained on the study, and there were no serious ocular adverse events. The principal drug-related ocular events that you mentioned, right, xanthopsia or chromatopsia and delayed dark adaptation. These are anticipated. They are mechanism-based effects of producing retinal delivery to the eye rather than unexpected off-target safety signal. We lead with expectation setting for patients and families, explaining the potential temporary color shift and slower adjustment to darkness. Advising patients to avoid driving or other low-light activities while symptomatic, and making clear when they should contact their physician. Based on the two-year exposure that we have in DRAGON, the absence of serious ocular events, and the limited number of treatment-related discontinuation, we view the profile as manageable for a chronic oral therapy. But I would like to invite Dr. Michaelides to weigh in from a medical perspective because he is the champion in recruitment in DRAGON-I and also DRAGON-II, and is actually overseeing a very large cohort of Stargardt patients on the drug. Thank you, Hendrik. Yes. As Hendrik said, we've been very lucky in London to be able to participate in both the DRAGON trial that's been showcased during this discussion, as well as the follow-up DRAGON II. We've had more than 40, 4-0, patients who've been fortunate enough to participate in this interventional trial. My experience has been that, as has been said with the data as a whole, that the effects are generally mild and haven't really represented a concern for the majority of patients. In fact, a significant number of the patients in DRAGON II were patients from DRAGON. I do not anticipate these expected side effects that patient can be educated about will represent any significant issues when tinlarebant is approved. Your next question comes from the line of Steve Seedhouse with Cantor. Your line is open. Please go ahead. Yeah, great. Thanks so much. I just wanted to follow up on the, maybe for the expert physicians on the call, including Dr. Michaelides, just given his experience prescribing tinlarebant and DRAGON. For the company, I guess. On the tolerability discussion, I was wondering if there are any sort of concerns or expectations about long-term systemic issues or things you'd want to monitor, just with respect to vitamin A trafficking or metabolism, beyond obviously the ocular things that were discussed. Then for the company, curious about what the patient journey is today, what they actually need to do to obtain a genetic confirmation of Stargardt, and just if there are any barriers or challenges in that process, like costs or anything else that you might have to mitigate as a company when you launch tinlarebant. Thank you. Dr. Michaelides or Dr. Bernstein, do you want to start on the tolerability, and then I can address the genetic testing. I am happy to make a start. Regarding the specific question, we did not see any notable systemic side effects. As people hopefully have appreciated, this is actually reducing vitamin A delivery to the eye alone. It is causing a relative deficiency of vitamin A in the eye only, nowhere else in the body. There is no data suggesting that a long-term relative deficiency of vitamin A in the eye will cause any issues. Time will tell as with any drug, of course, but to date, I do not anticipate any other concerns. Yes, I would agree. I think there will, of course, be surveillance by physicians and the company for any ocular or any other side effects that might be attributed to lower vitamin A. But I think beyond just usual monitoring, the patient's health, ocular and systemic, is all that we would expect. Maybe a small comment. There is a study what would happen if you completely abolish RBP4. It is super rare, but you can inherit mutations for the gene for RBP4 itself, leaving absolutely no RBP4 in the circulation. I have seen those two sisters personally when I was a resident in Tübingen, Germany at the time, in the university eye hospital, where the mutations in the gene was discovered at the time. These two sisters, I believe 15 and 13 years old, meanwhile they are in their 30s, are completely healthy. They have no systemic problems. I think it is like an experiment, what would happen if you abolish RBP4, not just reducing it by 80%. I think this is a very reassuring finding out of the literature. Maybe I turn over to Kelly. I think the question was also about the patient journey. Yeah. Thank you, Hendrik. I think the question was in regards to genetic testing. As we talked about, we have four key focus areas, and genetic testing under awareness is our number one focus area, and genetic confirmation is an important part of the diagnostic pathway. The testing environment has evolved meaningfully over the years. We believe the IRD testing environment is much more accessible today than it has been historically. There are multiple testing and sample collection options available for HCPs or patients. If an HCP would like to order a test or a patient would like to be tested, there are multiple options available. I do think you mentioned a question around cost. Fortunately, there are two sponsored or supported programs available to patients that are either low cost or no-cost testing options, depending on the patient's insurance. These programs make access to testing much easier and reduce the financial burden on patients suffering from IRDs. I would also include a commercial lab performing sequencing for an IRD panel. Typically, it only takes three to four weeks from the time the sample is received until the ordering HCP gets the results. So it's much quicker than it had been in the years past. However, we do recognize there is an opportunity for additional education for HCPs, particularly outside the IRD specialist community, and that's where our diagnostic specialists are focused on helping these doctors understand those options and providing education on incorporating testing into patient care for those that want to order a test or patients that want to be tested. I think I talked about this in my presentation, but we do believe having a new treatment available for these patients can help improve diagnostic testing rates. Increasing awareness around genetic testing continues to be one of our main focus areas, and there are multiple options, low cost, no cost, and a quick turnaround time, I think are the key takeaway messages. We do not see genetic testing as a bottleneck towards the patient journey. It's the option of the patient or the HCP to order if they so choose. It's a pretty straightforward process in order to receive a genetic test today. Please note that we are approaching the end of our allotted time for today's conference call, and this will be the last question. Your final question comes from the line of Marc Goodman with Leerink. Your line is open. Please go ahead. Hi. Good morning, everyone. This is Alyssa on for Marc. To follow up on the genetic testing discussion, I was wondering if, just to clarify, general ophthalmologists are able to order these tests as well? What about optometrists if they so choose? Then also, if you're successful in raising awareness and getting genetic testing to the forefront, do you imagine that this could allow patients to receive tinlarebant earlier on in the disease course than the patients that were enrolled in the DRAGON studies? Thank you. Yeah, sure. Happy to answer that question. We expect retina and IRD specialists to be the prescribers early on. Again, it will depend on our final label and the payer policies that are out there. However, retina specialists, ophthalmologists, and optometrists, as you had mentioned, will have a role to play, and we view the broader testing community as an advantage for a focused rare disease launch. Especially up front, ophthalmologists will play an important role in identifying and referring and supporting these patients. I will say prescribing patterns will change over time. It obviously will initiate with retina specialists. But again, depending on our final label and how the payer policies are written, will determine if there is an opportunity for ophthalmology to also participate in the prescribing of tinlarebant. But at this point, it is too early to say. I can comment on the second question, and this is applicability of tinlarebant in cases where, let us say symptoms or clinical signs of the disease are not very advanced or early and a genetic diagnosis being established. This happens. It is not frequently, but it happens because it runs in families and I am still seeing patients one day a week, and there are families that come with three children, and maybe two or three are affected. So you have the oldest has the more advanced disease, and the youngest may have a very early disease, but you establish the diagnosis through genetic testing. Then it is certainly in the responsibility of the healthcare provider to prescribe tinlarebant in order to save vision and save full receptors in that specific situation. Again, I would like to draw on the experience of Dr. Michaelides, who is also a pediatric ophthalmologist, essentially a pediatric ophthalmologist because he sees many very young patients with Stargardt disease in London. Maybe he can share with us his approach and what he believes will be the future in prescribing tinlarebant in such early cases. A 100% delighted to. I agree with you a 100%, Hendrik. There is no doubt that we will want to intervene as early as possible, given that we are looking to slow progression. So obviously the earlier we intervene, the greater impact we are going to have. As you say, I equally have a similar experience of siblings being diagnosed. We can do that readily with OCT. We have been blessed with fantastic access to genetic testing in the U.K. and a lot of Western Europe. So, we have more than 1,000 patients with Stargardt disease that are molecularly confirmed. Many patients who still are very early and have been picked up almost pre-symptomatically, as you say. I would certainly be hoping I would be able to offer that to patients. I certainly know the families want to access it as early as they can. This concludes today's call. Thank you for attending. You may now disconnect.