Ocugen, Inc. — Call Transcript 2026

Hi. Good morning, everyone, and welcome to the Ocugen session. Ocugen is in the gene therapy world, not for systemic diseases, but for retinal diseases, which is very localized and gives the company a very different risk profile. We're happy to have CEO Shankar Musunuri here with us today, who will go through the late-stage pipeline. As you know, gene therapy for inherited retinal diseases were among the first to be approved, so there's a lot of strong history and understanding there. Shankar, let me give you the floor, and you can cover the story briefly, and then we'll launch into Q&A. Great. Thank you, Annabelle. Thank you for having me today. You'll see it on this slide, we're really trying to bring this cutting-edge technology to the market and also work harder to provide market access for patients who need them globally. Significant unmet medical need. When you look into vision loss diseases, across the board, we are working on majority of those diseases which exist today with significant unmet medical needs, starting with dry age-related macular degeneration. The late stage of it is called geographic atrophy. About 10% of AMD patients have GA. That means they have central vision blurriness when you are 60 or 65, and it's a very debilitating disease. Currently, there are two approved products in the U.S. There's nothing across the board. We are planning to get into phase III shortly with that program with a global trial so that we have hope for these patients. It's 2 million to 3 million patients in U.S. and EU together. The second program, retinitis pigmentosa, is a big inherited retinal disease. Defects in over 100 genes can cause it, and there's a first product, as Annabelle stated, Luxturna, got approved 2017. It covers only one out of those 100-plus genes. Taking a traditional gene therapy, you need 100-plus products. However, with our gene therapy, we're able to encompass all these patients with one product. That's 300,000 patients in U.S. and EU alone. The next one, Stargardt disease, there's nothing for these patients globally, and we are trying to address 100,000 patients in U.S. and EU. All these diseases are debilitating, that eventually, most of these patients do become legally blind. What we are trying to do is bring first-in-class gene therapies for these patients, potentially with one-time treatments for life. We're going after massive diseases. Unlike traditional gene therapy starting 200 or 2,000 patients, we're going after hundreds of thousands to millions. In this one, there's a direct comparison of conventional gene therapy versus modifiers. I'll target a few things in there. One of them, if you take it like mutation-specific, something like retinitis pigmentosa, defects in over 100 genes can cause it. You need more than 100 products. However, since we use master regulator modified genes, can target the entire disease with a single product, therefore, our one product can target that. Diseases such as geographic atrophy have many complex pathways, and our genes can address and regulate all those pathways and also bring healthy environment for cells to survive. Because of that, we're able to target complex diseases and diseases which are inherited in nature with a single product. We have many accelerated pathways with our products, with FDA as well as EMA. Before I go into our pipeline where we are, I just want to distinguish why our gene therapy is very distinct and different everything else out there. If you take any standard therapies, typically what you're doing is, at the symptom level, you're intervening and you're slowing down disease progression. If you take the traditional gene therapy, you assume the network is okay, but network is not okay. Genes don't work in isolation. They work as a network. When they have a defective gene, the defective gene dysfunction causes network to suffer the toxic effects of it. That's why if you give functioning gene for traditional gene therapy, depending on when you give that, it may have different impact outcomes. What we need to think about, genes don't work in isolation. How do you bring the homeostasis the entire network? That's what our genes are able to do. In case of RP, Stargardt, it's a rare disease and inherited in nature. GA is age-related. In all these cases, our genes have ability to reset the homeostasis, work on the entire network, and also create a healthy environment for retinal cells to survive. Because of that, we're able to target large diseases with a single product. We have three programs currently, two already in phase III, and one of them is about to get there. OCU400 targeting retinitis pigmentosa. Its enrollment completed this year. Top-line results are expected in first quarter next year. Because of the RMAT designation, we're eligible for rolling submission. We're going to initiate this year, third quarter, and as soon as the top-line results come in, within few weeks, we'll drop the clinical module that will start the six-month accelerated clock for potential approval in late next year. Second program, Stargardt disease, OCU410ST. It's going through phase II/III registration trial. We are anticipating interim outcome analysis for any size adjustment in third quarter of this year that will minimize any risk to phase III clinical trial. If everything is on track per plan, we should be able to have top-line results in second quarter and then file the BLA right after that. Both retinitis pigmentosa Stargardt programs, we have alignment with EMA. The single study we're doing in U.S. is good enough to file market authorizations in E.U. The last program, OCU410, we recently released very promising phase II data. We are working with EMA and FDA for a global clinical trial with two key endpoints. One lesion as a primary. Secondary is ellipsoid zone, which correlates to visual function. We're anticipating alignment by third quarter, so we can initiate the phase III, and that will be targeted for BLA approval in 2028. In a nutshell, we are targeting with our genes, major unmet medical needs today exist globally for three major vision loss diseases, retinitis pigmentosa, Stargardt, geographic atrophy, three BLAs by 2028. That's our target. Thank you. Got it. Let me just ask a couple questions regarding these programs. You mentioned you're a modifier. You're modifying the master regulator pathways. What specifically are you providing to that environment? Or is it a protein that is being expressed that you're providing, and is it different between 400 and 410? Just give us a sense of what exactly it is that is changing in that microenvironment. Yeah. If you take something like retinitis pigmentosa, what we found out, irrespective of what genetic defect you have, and whenever you have any genetic defect, you look at the key transcription factors which are important for key functions in retina, such as cell development, metabolism, inflammation, to cell survival. Whenever you have any defect, it could be PDE6B or rhodopsin, we found a link, and the key transcription factors responsible for key functions are depressed. We found a missing link. That's NR2E3. That's also depressed. When you upregulate and all these key transcription factors, they upregulate and reset the homeostasis, and then it creates a healthy environment for cells to survive. Those things, I think we modeled in those Nature publications, looking at pharmacodynamic effects and survival of the cells and the effects. Today, we see those things in human patients. If you see some of our subjects, not only they're able to slow down the disease progression, some of them it's stalling the disease progression, stopping it, and some of them it's reversing it. Similarly, if you take another gene, RORA, which targets Stargardt as well as AMD, a dry age-related macular degeneration, geographic atrophy. In both these cases, one of them is inherited disease, another one is age-related. You got four key pathways which cause degeneration in these patients, oxidative stress, lipid metabolism, inflammation, and complement system. Current therapies for GA, it target only one pathway complement system. That's why if you can regulate all these pathways, and then you can get optimum results. That's exactly RORA gene does. It regulates all these four pathways, and it also resets the homeostasis and also create healthy environment for retinal cells, photoreceptors, as well as RPE cells to survive. Because of that, we're able to, again, in Stargardt patients, and we have data now in some patients up to two years, not only it's slowing down disease progression, some of them actually it's reversing the disease. They can see better, not just stopping where they are. I think because of their nature, how our modifier genes are, we call them master regulators work. They work on the entire network and create a healthy environment for retinal cells to survive. Why this is a powerful concept? Our retinal cells are non-dividing cells. If we can do something to reset them and create a healthy environment, potentially we should get the impact rest of your life. Got it. When you are approaching these diseases, and let's start with RP since it's your latest stage, what stage of disease are you targeting? Is it early stage before they start losing their vision, or have they lost their vision or getting vision restored? Can you just help us understand what that ideal patient population is that would better respond to an environment improvement versus the actual gene improvement? Yeah. Absolutely. If you take RP patients, if you look at our phase III clinical trial, it's early to advanced stage. Obviously, you need to have some photoreceptors there for it to work. Because we have to go directly into retina and inject, and that's where you want the targeted gene expression to get the maximum effect. In these patients, as long as you have some vision left, you cannot give it to people who are legally blind. You need to have some retina left, early to advanced stage, and pediatric to adult. It encompasses entire population of RP. Obviously, if you go back and look at the data, there are facts. There are many, over 15 companies tried after Luxturna with the traditional gene therapies to develop therapies. Where are they today? There's only one company doing in phase III. The traditional gene therapy, it's important. If you give early on, you may have a better effect because they're not really doing anything with the network effect. Since our genes can regulate the network, we're able to see benefits patients across from early stage to advanced stage or middle of the disease. All of them are getting benefit. Once again, the benefit may vary where you are and depending on the genetic mutation and type. I think you saw some of the videos on our website there. One of those patients is actually 60 plus. Obviously he lost most of his peripheral vision. He has central vision left, and now he's getting the peripheral vision back with our therapy. That's pretty advanced stage, I can call it, and he's still be able to show the reversal. That was in Stargardt's or in GA? That's in a GA. Sorry, it's retinitis pigmentosa, not GA. Yeah. Okay. Yeah. All right. Are all the proteins that you're expressing between 400 and 410, they're not the same? They're targeting different microenvironments, correct? Yeah. In 400 with RP, we use NR2E3, that's our modified gene. With the Stargardt and GA, we use RORA. That's our modified gene delivered through AAV vector. Okay, got it. I guess when there have been so many failures in retinitis pigmentosa, how did the medical community get comfortable with this one? What are some of the findings you had from the earlier studies, and what can you share with us today about that? Once again, some of them, there are two reasons, right? One is a technical reason, and the other one is financial. When you're developing a biotech, any novel therapies. I think majority of them had mixed results in early-stage clinical trials, and even some of them had good signals. At the end of the day, Luxturna was a great technical success. At the end of the day, commercial viability was questionable, just like many gene therapy products in the marketplace. Unfortunately, we're in the field where when you have a novel technology transformative medicine, people expect significant revenues and commercial success. Some of these therapies, they struggled even though they had good, probably promising, okay data. At the end of the day, you only have that genetic mutation of 50 or 100 patients. It's very difficult to get capital resources to continue those clinical trials. I think majority of those trials, some of them struggled, they failed. Some of them had okay data, they didn't proceed further. Only one company right now is in phase III for XLRP. That's the late stage. Okay. That covers probably north of 5% of entire RP population. Got it. All right. Can you, just to give us an idea, are you slowing progression of RP? Are you restoring vision? What should we expect of this upcoming data? What is your actual endpoint? Is it slowing progression or is it actual restoration of vision? Obviously, what you're looking at is, whenever you're doing clinical trials for the RP, we're using mobility test, which is already approved in the Luxturna, a similar one. Obviously, we worked with FDA. We made it more specific and sensitive. We actually validated it in our phase III with actual patients, very robust. Here what you're measuring is you have a treatment group and untreated control group, right, assessor-blinded study. If you're able to show improvement in the mobility test, which reflects your quality-of-life improvement, like if I go to a restaurant in a dull light before therapy, I'm unable to go sit in my chair. I need somebody's help to hold my hand and take me. After therapy, that means I'm performing better in dull light, improve my quality of life. Now I can do it myself in a restaurant dull-light setting. I can go sit in my chair. That's an improvement. This mobility test actually simulates from bright light to dull moonlight. Our goal is to show the improvement after the treatment compared to untreated control group. That's a quantitative measure. In case of other therapies like Stargardt and GA, you have central vision, lesions or blurriness. What you're trying to do is you're trying to compare the growth of that lesion between treatment and untreated group. If you're slowing it down compared to untreated group, you're measuring it and statistical superiority based on the design, that means you've got a successful outcome. It's benefiting patients. In all these cases, obviously, as I mentioned before, you don't need to show reversal of the disease to get drugs approved because many of the drugs in the market today, if you look at it, majority of them, very close to probably more than 90%, and they're all slowing down disease progression compared to control arm. In our case, if the patients are able to stall the disease, that's good. If they're able to reverse in some cases I mentioned, that's monumental and it's really truly transformative. Obviously, in the clinical trial, we try to minimize risk as Ocugen. You take endpoints which are already approved by FDA because that has a probability of success is high and low risk, clinical trial design perspective. You can modify them as needed, but it's good to always take the precedence, right? You don't have to prove this endpoint works. That's what we are doing for all our clinical. We take something which already approved and then implementing what else is needed around it. Great. I guess, as you might have mentioned, there are several RP gene therapies that are in development right now. You could potentially be the first to market as a gene agnostic approach. Does this put you in a position to be standard of care? How intense is demand? How important is it to be first? What is your ideal patient here? Obviously, since you're gene agnostic, you could potentially affect everyone. How do you think about Your product versus some other approaches that are being developed right now. The only one which is in phase III is XLRP from another biotech company. Obviously, in our clinical trial, if you look at it, we had more than 25 genetic mutations covered. We covered all the major prevalence mutations, which are like, if you think something has 3% or 4% of RP, like XLRP, rhodopsin, PDE6B, Ushers, we covered major mutations. Our goal is to get a broad RP indication, and sometimes, some of the patients, it's very difficult to genotype them. If clinically, if they're phenotyped, they have RP, they can be used. We have expanded access program, very rare. We did it because of empathy for our patients, and they don't want to wait. Either they don't qualify for the clinical trial, or they don't want to wait for two years for the product to come out. We opened it up, and then several patients got expanded access. That's very broad. That's like syndromic, non-syndromic or clinically, if you're diagnosed, you can get it. Our goal is to get the broad indication irrespective of what their genetic mutations show then. We're not worried about somebody's coming with a single mutation-based gene therapies. As I mentioned before, those gene therapies, remember, the defect is there from birth. How the dysfunctioning gene or the protein expression is causing the damage to your network, people need to seriously look into that. Genes don't work in isolation. Those gene therapies, you have to be careful when you're intervening with the patients. If they do it early on, scientifically, they should get better results. Whereas we can take care of all the patients. That's our goal. Ideally, any therapy, you want to give it to any patient, you want to intervene earlier than later, right? If the patients who are in the late stage, we cannot deny them, because we're able to show in our clinical trials even the later-stage patients are benefiting. For them, it's all relative, right, Annabelle? Yeah. If you think, if I'm able to keep my central vision, that's like one of our patients says, I'll be good. If I can just hang on to what I have because I learned how to live with this. I don't want to go further down. That's our goal. We don't want to deny anyone. We want to have broad RP indications, take care of all the patients globally. Can you remind us how big your actual trial is and how many patients were able to be enrolled in the expanded access? Our actual trial is 140 patients. It's 2:1 ratio. That means a treated group has two to one. You take around 95 in treated and rest of them in untreated group, control. That's the design we had, which you had 140 patients. How many have been able to get access to the expanded access? Expanded access program, yeah. Expanded access program, we didn't publicly disclose the numbers. That protocol can go up to 75 patients. Can that be included in your BLA package at all? Obviously, we have to monitor those patients. The approval is based on the clinical trial, the phase III. This can be added as additional data, obviously. Just like phase I/II, all the data will be submitted for safety or any signals. We'll definitely summarize all that. Got it. With the phase III that are coming out in first quarter, you can already submit a rolling BLA. What is it that you can start with in terms of the submission? Yes. I think that's an advantage for the company and also the agency, right? We're able to submit a non-clinical module, and we're on target with our PPQ runs successful, and therefore, we'll be able to drop the CMC section this year. That's a big lift from any biotech company perspective. We also minimized any risk to our two phase III for RP and Stargardt. We introduced actually two commercial scale lots in our phase III to minimize any risk to CMC, where we are very robust in our CMC process. Those two sections can be dropped in this year. That way, as soon as the clinical data comes out in first quarter, once the top line comes in, within weeks, we can drop the clinical module. That's the plan for rolling submission. Great. Just wanted to move to Stargardt's really quickly. Obviously, that's another genetic disease. The approach here, this is a much more homogeneous group, and your endpoints are a little bit different. You're trying to change the progression of the lesion. Can you actually reverse the lesion, or once they have a lesion, those cells are completely dead? What is the approach here? You're just trying to slow the progression of lesion expansion, correct? I think in the clinical trial, all our clinical trials are one year because they've seen an unmet medical need. Other companies may be doing two-year, three-year trials. If you're able to show treatment benefit in one year in your unmet medical need, obviously agencies, FDA may support it, right? They don't want to keep doing two or three-year trials. In this one year, what is the measurable effect it can show compared to untreated arm? That's a lesion. Based on the disease progression, we can show that. You're slowing down the growth of the lesion compared to untreated control. Of course, you'll have other measures, but we don't have visual acuity and ups or down. The nature of the disease progression is slow. Obviously, we pick the primary endpoint, which is important for approval is lesion. If you are seeing other effects, what I was talking about, some of the patient videos, it takes time for them to show that like second year, third year, these patients are improving further and further, not just slowing down lesion. They're able to see clearly, their vision is coming back. Somebody may be able to see the faces very clearly, which was lagging before. That's more than just controlling the growth of the lesion. As I mentioned, those things we are not measuring in the clinical trial. Obviously, like all our gene therapies, we have to monitor them for safety for five years, every year on annual basis. When they come to doctor's office, some of these measures we observe. Obviously, with all our BLA filings, if we have early-stage clinical trial data, phase I or phase I/II, and what other data we have, if we have three-year data like we released for RP, or four years, we'll put that. All the data will be there in our BLA submission. Okay, great. You have an interim analysis coming up for the phase II/III. How is it powered, and what are you looking for in your decision tree? What are the possibilities here? Just maybe you can talk about that a little bit. Our trials are powered in about 90%, or in some cases, RP has 95% or more in GA. However, what we're looking for is, it's really important. I mean, it's really fortunate to have this adaptive design. During phase III clinical trial, when you're doing the registration trial, you're able to, not like a broad look, but under very strict guidance of data monitoring committee. They can take a look because in phase I you have a control line. In phase II/III, registration trial, you've got actual true control, untreated control. You're really getting actual data compared to that, and now you have ability to adjust. What are the potential outcomes? They look at it and say, based on predictive analytics, at 50% reaching eight months, or in 12 months it's going to hit it, no change needed. You need to size up. That way, if you size it up, add more patients, or in some cases, if you add another time point from 12-16 months, that may also help based on predictive analytics. All these outcome analysis or predictive analytics is based to minimize the risk, further risk for the ongoing phase III with the true control data. When you have that option, it's really very good gift for any company to minimize any phase III clinical trial risk during phase III clinical trial. If in this interim analysis you're given the green light to continue, those 50% of the patients you're seeing separation, you don't need to size up. Will the FDA seek perhaps an additional study of more patients because perhaps the 50% of that trial was not enough? Will you continue to enroll that trial based on 140, or will you stop right there and try to file? No, I think RP, we don't have interim analysis with ending, like all the patients completing one year. Stargardt. Oh, sorry. Stargardt is only 50% to eight months. Yeah. Our endpoint is actually one year. We continue. Yeah. Even though the data comes out good, we continue, and we close it out at 12 months. In geographic atrophy, obviously, that's a package we submitted to FDA and EMA, 300-patient trial, and that one has a different adaptive design. When 150 patients reach one year, that trial has a very good power. Even at 150 patients, I think for the primary endpoint lesion, I think our power is more than 90%. That one has an opportunity. If it hits it, there may be a potential to talk to the agencies and say, continue to collect the data, because it's a large disease and you need 300 patient data minimum for safety reasons. That's how we are using adaptive design to further de-risk our clinical trial. Also, create an upside scenario where, oh, can we file it sooner than later? That's great. In the five seconds that we have left, unfortunately, we're out of time, but can you tell us your cash position and where that takes you to? Yeah. We recently raised a gross $130 million with convert and with all the net and everything proceeds. Our cash runway gets into 2028. What does it mean? That will allow us to file two BLAs next year and also initiate the GA phase III clinical trial to create a tremendous upbeat for the company and inflection points. Obviously, a lot of things are going to be opened up. Stargardt also has a rare pediatric designation, and when you get approval in early 2028 based on our plans and the PRV, it could be worth $150 million-$200 million. Great. Obviously, we are in a very good cash position. My team is doing a fantastic job executing. In 2022, we started the first patient dosed in our gene therapy trial. Today, we lead ophthalmology gene therapies because of the breadth and the depth we cover with our gene therapy is so high. Large populations, significant unmet medical needs, this cash runway will help us to really focus on programs, what we are good at, and executing, and move all three programs to where they belong. Great. Thank you so much for the time. I appreciate the overview. Thank you for having me. Excellent

Speaker 1: Hi. Good morning, everyone, and welcome to the Ocugen session. Ocugen is in the gene therapy world, not for systemic diseases, but for retinal diseases, which is very localized and gives the company a very different risk profile. We're happy to have CEO Shankar Musunuri here with us today, who will go through the late-stage pipeline. As you know, gene therapy for inherited retinal diseases were among the first to be approved, so there's a lot of strong history and understanding there. Shankar, let me give you the floor, and you can cover the story briefly, and then we'll launch into Q&A. Hi. hi Good morning, everyone, and welcome to the Ocugen session. good morning everyone and welcome to the ocugen session Ocugen is in the gene therapy world, not for systemic diseases, but for retinal diseases, which is very localized and gives the company a very different risk profile. ocugen is in the gene therapy world not for systemic diseases but for retinal diseases which is very localized and gives the company a very different risk profile We're happy to have CEO Shankar Musunuri here with us today, who will go through the late-stage pipeline. we're happy to have ceo shankar musunuri here with us today who will go through the late-stage pipeline As you know, gene therapy for inherited retinal diseases were among the first to be approved, so there's a lot of strong history and understanding there. as you know gene therapy for inherited retinal diseases were among the first to be approved so there's a lot of strong history and understanding there Shankar, let me give you the floor, and you can cover the story briefly, and then we'll launch into Q&A. shankar let me give you the floor and you can cover the story briefly and then we'll launch into q&a

Speaker 2: Great. Thank you, Annabelle. Thank you for having me today. You'll see it on this slide, we're really trying to bring this cutting-edge technology to the market and also work harder to provide market access for patients who need them globally. Significant unmet medical need. When you look into vision loss diseases, across the board, we are working on majority of those diseases which exist today with significant unmet medical needs, starting with dry age-related macular degeneration. The late stage of it is called geographic atrophy. About 10% of AMD patients have GA. That means they have central vision blurriness when you are 60 or 65, and it's a very debilitating disease. Currently, there are two approved products in the U.S. There's nothing across the board. Great. great Thank you, Annabelle. thank you annabelle Thank you for having me today. thank you for having me today You'll see it on this slide, we're really trying to bring this cutting-edge technology to the market and also work harder to provide market access for patients who need them globally. you'll see it on this slide we're really trying to bring this cutting-edge technology to the market and also work harder to provide market access for patients who need them globally Significant unmet medical need. significant unmet medical need When you look into vision loss diseases, across the board, we are working on majority of those diseases which exist today with significant unmet medical needs, starting with dry age-related macular degeneration. when you look into vision loss diseases across the board we are working on majority of those diseases which exist today with significant unmet medical needs starting with dry age-related macular degeneration The late stage of it is called geographic atrophy. the late stage of it is called geographic atrophy About 10% of AMD patients have GA. about 10% of amd patients have ga That means they have central vision blurriness when you are 60 or 65, and it's a very debilitating disease. that means they have central vision blurriness when you are 60 or 65 and it's a very debilitating disease Currently, there are two approved products in the U.S. currently there are two approved products in the u.s There's nothing across the board. there's nothing across the board We are planning to get into phase III shortly with that program with a global trial so that we have hope for these patients. It's 2 million to 3 million patients in U.S. and EU together. The second program, retinitis pigmentosa, is a big inherited retinal disease. Defects in over 100 genes can cause it, and there's a first product, as Annabelle stated, Luxturna, got approved 2017. It covers only one out of those 100-plus genes. Taking a traditional gene therapy, you need 100-plus products. However, with our gene therapy, we're able to encompass all these patients with one product. That's 300,000 patients in U.S. and EU alone. The next one, Stargardt disease, there's nothing for these patients globally, and we are trying to address 100,000 patients in U.S. and EU. All these diseases are debilitating, that eventually, most of these patients do become legally blind. We are planning to get into phase III shortly with that program with a global trial so that we have hope for these patients. we are planning to get into phase iii shortly with that program with a global trial so that we have hope for these patients It's 2 million to 3 million patients in U.S. and EU together. it's 2 million to 3 million patients in u.s and eu together The second program, retinitis pigmentosa, is a big inherited retinal disease. the second program retinitis pigmentosa is a big inherited retinal disease Defects in over 100 genes can cause it, and there's a first product, as Annabelle stated, Luxturna, got approved 2017. defects in over 100 genes can cause it and there's a first product as annabelle stated luxturna got approved 2017 It covers only one out of those 100-plus genes. it covers only one out of those 100-plus genes Taking a traditional gene therapy, you need 100-plus products. taking a traditional gene therapy you need 100-plus products However, with our gene therapy, we're able to encompass all these patients with one product. however with our gene therapy we're able to encompass all these patients with one product That's 300,000 patients in U.S. and EU alone. that's 300,000 patients in u.s and eu alone The next one, Stargardt disease, there's nothing for these patients globally, and we are trying to address 100,000 patients in U.S. and EU. the next one stargardt disease there's nothing for these patients globally and we are trying to address 100,000 patients in u.s and eu All these diseases are debilitating, that eventually, most of these patients do become legally blind. all these diseases are debilitating that eventually most of these patients do become legally blind What we are trying to do is bring first-in-class gene therapies for these patients, potentially with one-time treatments for life. We're going after massive diseases. Unlike traditional gene therapy starting 200 or 2,000 patients, we're going after hundreds of thousands to millions. In this one, there's a direct comparison of conventional gene therapy versus modifiers. I'll target a few things in there. One of them, if you take it like mutation-specific, something like retinitis pigmentosa, defects in over 100 genes can cause it. You need more than 100 products. However, since we use master regulator modified genes, can target the entire disease with a single product, therefore, our one product can target that. Diseases such as geographic atrophy have many complex pathways, and our genes can address and regulate all those pathways and also bring healthy environment for cells to survive. What we are trying to do is bring first-in-class gene therapies for these patients, potentially with one-time treatments for life. what we are trying to do is bring first-in-class gene therapies for these patients potentially with one-time treatments for life We're going after massive diseases. we're going after massive diseases Unlike traditional gene therapy starting 200 or 2,000 patients, we're going after hundreds of thousands to millions. unlike traditional gene therapy starting 200 or 2,000 patients we're going after hundreds of thousands to millions In this one, there's a direct comparison of conventional gene therapy versus modifiers. in this one there's a direct comparison of conventional gene therapy versus modifiers I'll target a few things in there. i'll target a few things in there One of them, if you take it like mutation-specific, something like retinitis pigmentosa, defects in over 100 genes can cause it. one of them if you take it like mutation-specific something like retinitis pigmentosa defects in over 100 genes can cause it You need more than 100 products. you need more than 100 products However, since we use master regulator modified genes, can target the entire disease with a single product, therefore, our one product can target that. however since we use master regulator modified genes can target the entire disease with a single product therefore our one product can target that Diseases such as geographic atrophy have many complex pathways, and our genes can address and regulate all those pathways and also bring healthy environment for cells to survive. diseases such as geographic atrophy have many complex pathways and our genes can address and regulate all those pathways and also bring healthy environment for cells to survive Because of that, we're able to target complex diseases and diseases which are inherited in nature with a single product. We have many accelerated pathways with our products, with FDA as well as EMA. Before I go into our pipeline where we are, I just want to distinguish why our gene therapy is very distinct and different everything else out there. If you take any standard therapies, typically what you're doing is, at the symptom level, you're intervening and you're slowing down disease progression. If you take the traditional gene therapy, you assume the network is okay, but network is not okay. Genes don't work in isolation. They work as a network. When they have a defective gene, the defective gene dysfunction causes network to suffer the toxic effects of it. Because of that, we're able to target complex diseases and diseases which are inherited in nature with a single product. because of that we're able to target complex diseases and diseases which are inherited in nature with a single product We have many accelerated pathways with our products, with FDA as well as EMA. we have many accelerated pathways with our products with fda as well as ema Before I go into our pipeline where we are, I just want to distinguish why our gene therapy is very distinct and different everything else out there. before i go into our pipeline where we are i just want to distinguish why our gene therapy is very distinct and different everything else out there If you take any standard therapies, typically what you're doing is, at the symptom level, you're intervening and you're slowing down disease progression. if you take any standard therapies typically what you're doing is at the symptom level you're intervening and you're slowing down disease progression If you take the traditional gene therapy, you assume the network is okay, but network is not okay. if you take the traditional gene therapy you assume the network is okay but network is not okay Genes don't work in isolation. genes don't work in isolation They work as a network. they work as a network When they have a defective gene, the defective gene dysfunction causes network to suffer the toxic effects of it. when they have a defective gene the defective gene dysfunction causes network to suffer the toxic effects of it That's why if you give functioning gene for traditional gene therapy, depending on when you give that, it may have different impact outcomes. What we need to think about, genes don't work in isolation. How do you bring the homeostasis the entire network? That's what our genes are able to do. In case of RP, Stargardt, it's a rare disease and inherited in nature. GA is age-related. In all these cases, our genes have ability to reset the homeostasis, work on the entire network, and also create a healthy environment for retinal cells to survive. Because of that, we're able to target large diseases with a single product. We have three programs currently, two already in phase III, and one of them is about to get there. OCU400 targeting retinitis pigmentosa. Its enrollment completed this year. Top-line results are expected in first quarter next year. That's why if you give functioning gene for traditional gene therapy, depending on when you give that, it may have different impact outcomes. that's why if you give functioning gene for traditional gene therapy depending on when you give that it may have different impact outcomes What we need to think about, genes don't work in isolation. what we need to think about genes don't work in isolation How do you bring the homeostasis the entire network? how do you bring the homeostasis the entire network That's what our genes are able to do. that's what our genes are able to do In case of RP, Stargardt, it's a rare disease and inherited in nature. in case of rp stargardt it's a rare disease and inherited in nature GA is age-related. ga is age-related In all these cases, our genes have ability to reset the homeostasis, work on the entire network, and also create a healthy environment for retinal cells to survive. in all these cases our genes have ability to reset the homeostasis work on the entire network and also create a healthy environment for retinal cells to survive Because of that, we're able to target large diseases with a single product. because of that we're able to target large diseases with a single product We have three programs currently, two already in phase III, and one of them is about to get there. we have three programs currently two already in phase iii and one of them is about to get there OCU400 targeting retinitis pigmentosa. ocu400 targeting retinitis pigmentosa Its enrollment completed this year. its enrollment completed this year Top-line results are expected in first quarter next year. top-line results are expected in first quarter next year Because of the RMAT designation, we're eligible for rolling submission. We're going to initiate this year, third quarter, and as soon as the top-line results come in, within few weeks, we'll drop the clinical module that will start the six-month accelerated clock for potential approval in late next year. Second program, Stargardt disease, OCU410ST. It's going through phase II/III registration trial. We are anticipating interim outcome analysis for any size adjustment in third quarter of this year that will minimize any risk to phase III clinical trial. If everything is on track per plan, we should be able to have top-line results in second quarter and then file the BLA right after that. Both retinitis pigmentosa Stargardt programs, we have alignment with EMA. The single study we're doing in U.S. is good enough to file market authorizations in E.U. Because of the RMAT designation, we're eligible for rolling submission. because of the rmat designation we're eligible for rolling submission We're going to initiate this year, third quarter, and as soon as the top-line results come in, within few weeks, we'll drop the clinical module that will start the six-month accelerated clock for potential approval in late next year. we're going to initiate this year third quarter and as soon as the top-line results come in within few weeks we'll drop the clinical module that will start the six-month accelerated clock for potential approval in late next year Second program, Stargardt disease, OCU410ST. second program stargardt disease ocu410st It's going through phase II/III registration trial. it's going through phase ii/iii registration trial We are anticipating interim outcome analysis for any size adjustment in third quarter of this year that will minimize any risk to phase III clinical trial. we are anticipating interim outcome analysis for any size adjustment in third quarter of this year that will minimize any risk to phase iii clinical trial If everything is on track per plan, we should be able to have top-line results in second quarter and then file the BLA right after that. if everything is on track per plan we should be able to have top-line results in second quarter and then file the bla right after that Both retinitis pigmentosa Stargardt programs, we have alignment with EMA. both retinitis pigmentosa stargardt programs we have alignment with ema The single study we're doing in U.S. is good enough to file market authorizations in E.U. the single study we're doing in u.s is good enough to file market authorizations in e.u The last program, OCU410, we recently released very promising phase II data. We are working with EMA and FDA for a global clinical trial with two key endpoints. One lesion as a primary. Secondary is ellipsoid zone, which correlates to visual function. We're anticipating alignment by third quarter, so we can initiate the phase III, and that will be targeted for BLA approval in 2028. In a nutshell, we are targeting with our genes, major unmet medical needs today exist globally for three major vision loss diseases, retinitis pigmentosa, Stargardt, geographic atrophy, three BLAs by 2028. That's our target. Thank you. The last program, OCU410, we recently released very promising phase II data. the last program ocu410 we recently released very promising phase ii data We are working with EMA and FDA for a global clinical trial with two key endpoints. we are working with ema and fda for a global clinical trial with two key endpoints One lesion as a primary. one lesion as a primary Secondary is ellipsoid zone, which correlates to visual function. secondary is ellipsoid zone which correlates to visual function We're anticipating alignment by third quarter, so we can initiate the phase III, and that will be targeted for BLA approval in 2028. we're anticipating alignment by third quarter so we can initiate the phase iii and that will be targeted for bla approval in 2028 In a nutshell, we are targeting with our genes, major unmet medical needs today exist globally for three major vision loss diseases, retinitis pigmentosa, Stargardt, geographic atrophy, three BLAs by 2028. in a nutshell we are targeting with our genes major unmet medical needs today exist globally for three major vision loss diseases retinitis pigmentosa stargardt geographic atrophy three blas by 2028 That's our target. that's our target Thank you. thank you

Speaker 1: Got it. Let me just ask a couple questions regarding these programs. You mentioned you're a modifier. You're modifying the master regulator pathways. What specifically are you providing to that environment? Or is it a protein that is being expressed that you're providing, and is it different between 400 and 410? Just give us a sense of what exactly it is that is changing in that microenvironment. Got it. got it Let me just ask a couple questions regarding these programs. let me just ask a couple questions regarding these programs You mentioned you're a modifier. you mentioned you're a modifier You're modifying the master regulator pathways. you're modifying the master regulator pathways What specifically are you providing to that environment? what specifically are you providing to that environment Or is it a protein that is being expressed that you're providing, and is it different between 400 and 410? or is it a protein that is being expressed that you're providing and is it different between 400 and 410 Just give us a sense of what exactly it is that is changing in that microenvironment. just give us a sense of what exactly it is that is changing in that microenvironment

Speaker 2: Yeah. If you take something like retinitis pigmentosa, what we found out, irrespective of what genetic defect you have, and whenever you have any genetic defect, you look at the key transcription factors which are important for key functions in retina, such as cell development, metabolism, inflammation, to cell survival. Whenever you have any defect, it could be PDE6B or rhodopsin, we found a link, and the key transcription factors responsible for key functions are depressed. We found a missing link. That's NR2E3. That's also depressed. When you upregulate and all these key transcription factors, they upregulate and reset the homeostasis, and then it creates a healthy environment for cells to survive. Those things, I think we modeled in those Nature publications, looking at pharmacodynamic effects and survival of the cells and the effects. Today, we see those things in human patients. Yeah. yeah If you take something like retinitis pigmentosa, what we found out, irrespective of what genetic defect you have, and whenever you have any genetic defect, you look at the key transcription factors which are important for key functions in retina, such as cell development, metabolism, inflammation, to cell survival. if you take something like retinitis pigmentosa what we found out irrespective of what genetic defect you have and whenever you have any genetic defect you look at the key transcription factors which are important for key functions in retina such as cell development metabolism inflammation to cell survival Whenever you have any defect, it could be PDE6B or rhodopsin, we found a link, and the key transcription factors responsible for key functions are depressed. whenever you have any defect it could be pde6b or rhodopsin we found a link and the key transcription factors responsible for key functions are depressed We found a missing link. we found a missing link That's NR2E3. that's nr2e3 That's also depressed. that's also depressed When you upregulate and all these key transcription factors, they upregulate and reset the homeostasis, and then it creates a healthy environment for cells to survive. when you upregulate and all these key transcription factors they upregulate and reset the homeostasis and then it creates a healthy environment for cells to survive Those things, I think we modeled in those Nature publications, looking at pharmacodynamic effects and survival of the cells and the effects. those things i think we modeled in those nature publications looking at pharmacodynamic effects and survival of the cells and the effects Today, we see those things in human patients. today we see those things in human patients If you see some of our subjects, not only they're able to slow down the disease progression, some of them it's stalling the disease progression, stopping it, and some of them it's reversing it. Similarly, if you take another gene, RORA, which targets Stargardt as well as AMD, a dry age-related macular degeneration, geographic atrophy. In both these cases, one of them is inherited disease, another one is age-related. You got four key pathways which cause degeneration in these patients, oxidative stress, lipid metabolism, inflammation, and complement system. Current therapies for GA, it target only one pathway complement system. That's why if you can regulate all these pathways, and then you can get optimum results. That's exactly RORA gene does. If you see some of our subjects, not only they're able to slow down the disease progression, some of them it's stalling the disease progression, stopping it, and some of them it's reversing it. if you see some of our subjects not only they're able to slow down the disease progression some of them it's stalling the disease progression stopping it and some of them it's reversing it Similarly, if you take another gene, RORA, which targets Stargardt as well as AMD, a dry age-related macular degeneration, geographic atrophy. similarly if you take another gene rora which targets stargardt as well as amd a dry age-related macular degeneration geographic atrophy In both these cases, one of them is inherited disease, another one is age-related. in both these cases one of them is inherited disease another one is age-related You got four key pathways which cause degeneration in these patients, oxidative stress, lipid metabolism, inflammation, and complement system. you got four key pathways which cause degeneration in these patients oxidative stress lipid metabolism inflammation and complement system Current therapies for GA, it target only one pathway complement system. current therapies for ga it target only one pathway complement system That's why if you can regulate all these pathways, and then you can get optimum results. that's why if you can regulate all these pathways and then you can get optimum results That's exactly RORA gene does. that's exactly rora gene does It regulates all these four pathways, and it also resets the homeostasis and also create healthy environment for retinal cells, photoreceptors, as well as RPE cells to survive. Because of that, we're able to, again, in Stargardt patients, and we have data now in some patients up to two years, not only it's slowing down disease progression, some of them actually it's reversing the disease. They can see better, not just stopping where they are. I think because of their nature, how our modifier genes are, we call them master regulators work. They work on the entire network and create a healthy environment for retinal cells to survive. Why this is a powerful concept? Our retinal cells are non-dividing cells. If we can do something to reset them and create a healthy environment, potentially we should get the impact rest of your life. It regulates all these four pathways, and it also resets the homeostasis and also create healthy environment for retinal cells, photoreceptors, as well as RPE cells to survive. it regulates all these four pathways and it also resets the homeostasis and also create healthy environment for retinal cells photoreceptors as well as rpe cells to survive Because of that, we're able to, again, in Stargardt patients, and we have data now in some patients up to two years, not only it's slowing down disease progression, some of them actually it's reversing the disease. because of that we're able to again in stargardt patients and we have data now in some patients up to two years not only it's slowing down disease progression some of them actually it's reversing the disease They can see better, not just stopping where they are. they can see better not just stopping where they are I think because of their nature, how our modifier genes are, we call them master regulators work. i think because of their nature how our modifier genes are we call them master regulators work They work on the entire network and create a healthy environment for retinal cells to survive. they work on the entire network and create a healthy environment for retinal cells to survive Why this is a powerful concept? why this is a powerful concept Our retinal cells are non-dividing cells. our retinal cells are non-dividing cells If we can do something to reset them and create a healthy environment, potentially we should get the impact rest of your life. if we can do something to reset them and create a healthy environment potentially we should get the impact rest of your life

Speaker 1: Got it. When you are approaching these diseases, and let's start with RP since it's your latest stage, what stage of disease are you targeting? Is it early stage before they start losing their vision, or have they lost their vision or getting vision restored? Can you just help us understand what that ideal patient population is that would better respond to an environment improvement versus the actual gene improvement? Got it. got it When you are approaching these diseases, and let's start with RP since it's your latest stage, what stage of disease are you targeting? when you are approaching these diseases and let's start with rp since it's your latest stage what stage of disease are you targeting Is it early stage before they start losing their vision, or have they lost their vision or getting vision restored? is it early stage before they start losing their vision or have they lost their vision or getting vision restored Can you just help us understand what that ideal patient population is that would better respond to an environment improvement versus the actual gene improvement? can you just help us understand what that ideal patient population is that would better respond to an environment improvement versus the actual gene improvement

Speaker 2: Yeah. Absolutely. If you take RP patients, if you look at our phase III clinical trial, it's early to advanced stage. Obviously, you need to have some photoreceptors there for it to work. Because we have to go directly into retina and inject, and that's where you want the targeted gene expression to get the maximum effect. In these patients, as long as you have some vision left, you cannot give it to people who are legally blind. You need to have some retina left, early to advanced stage, and pediatric to adult. It encompasses entire population of RP. Obviously, if you go back and look at the data, there are facts. There are many, over 15 companies tried after Luxturna with the traditional gene therapies to develop therapies. Where are they today? There's only one company doing in phase III. Yeah. yeah Absolutely. absolutely If you take RP patients, if you look at our phase III clinical trial, it's early to advanced stage. if you take rp patients if you look at our phase iii clinical trial it's early to advanced stage Obviously, you need to have some photoreceptors there for it to work. obviously you need to have some photoreceptors there for it to work Because we have to go directly into retina and inject, and that's where you want the targeted gene expression to get the maximum effect. because we have to go directly into retina and inject and that's where you want the targeted gene expression to get the maximum effect In these patients, as long as you have some vision left, you cannot give it to people who are legally blind. in these patients as long as you have some vision left you cannot give it to people who are legally blind You need to have some retina left, early to advanced stage, and pediatric to adult. you need to have some retina left early to advanced stage and pediatric to adult It encompasses entire population of RP. it encompasses entire population of rp Obviously, if you go back and look at the data, there are facts. obviously if you go back and look at the data there are facts There are many, over 15 companies tried after Luxturna with the traditional gene therapies to develop therapies. there are many over 15 companies tried after luxturna with the traditional gene therapies to develop therapies Where are they today? where are they today There's only one company doing in phase III. there's only one company doing in phase iii The traditional gene therapy, it's important. If you give early on, you may have a better effect because they're not really doing anything with the network effect. Since our genes can regulate the network, we're able to see benefits patients across from early stage to advanced stage or middle of the disease. All of them are getting benefit. Once again, the benefit may vary where you are and depending on the genetic mutation and type. I think you saw some of the videos on our website there. One of those patients is actually 60 plus. Obviously he lost most of his peripheral vision. He has central vision left, and now he's getting the peripheral vision back with our therapy. That's pretty advanced stage, I can call it, and he's still be able to show the reversal. The traditional gene therapy, it's important. the traditional gene therapy it's important If you give early on, you may have a better effect because they're not really doing anything with the network effect. if you give early on you may have a better effect because they're not really doing anything with the network effect Since our genes can regulate the network, we're able to see benefits patients across from early stage to advanced stage or middle of the disease. since our genes can regulate the network we're able to see benefits patients across from early stage to advanced stage or middle of the disease All of them are getting benefit. all of them are getting benefit Once again, the benefit may vary where you are and depending on the genetic mutation and type. once again the benefit may vary where you are and depending on the genetic mutation and type I think you saw some of the videos on our website there. i think you saw some of the videos on our website there One of those patients is actually 60 plus. one of those patients is actually 60 plus Obviously he lost most of his peripheral vision. obviously he lost most of his peripheral vision He has central vision left, and now he's getting the peripheral vision back with our therapy. he has central vision left and now he's getting the peripheral vision back with our therapy That's pretty advanced stage, I can call it, and he's still be able to show the reversal. that's pretty advanced stage i can call it and he's still be able to show the reversal

Speaker 1: That was in Stargardt's or in GA? That was in Stargardt's or in GA? that was in stargardt's or in ga

Speaker 2: That's in a GA. Sorry, it's retinitis pigmentosa, not GA. Yeah. That's in a GA. that's in a ga Sorry, it's retinitis pigmentosa, not GA. sorry it's retinitis pigmentosa not ga Yeah. yeah

Speaker 1: Okay. Okay. okay

Speaker 2: Yeah. Yeah. yeah

Speaker 1: All right. Are all the proteins that you're expressing between 400 and 410, they're not the same? They're targeting different microenvironments, correct? All right. all right Are all the proteins that you're expressing between 400 and 410, they're not the same? are all the proteins that you're expressing between 400 and 410 they're not the same They're targeting different microenvironments, correct? they're targeting different microenvironments correct

Speaker 2: Yeah. In 400 with RP, we use NR2E3, that's our modified gene. With the Stargardt and GA, we use RORA. That's our modified gene delivered through AAV vector. Yeah. yeah In 400 with RP, we use NR2E3, that's our modified gene. in 400 with rp we use nr2e3 that's our modified gene With the Stargardt and GA, we use RORA. with the stargardt and ga we use rora That's our modified gene delivered through AAV vector. that's our modified gene delivered through aav vector

Speaker 1: Okay, got it. I guess when there have been so many failures in retinitis pigmentosa, how did the medical community get comfortable with this one? What are some of the findings you had from the earlier studies, and what can you share with us today about that? Okay, got it. okay got it I guess when there have been so many failures in retinitis pigmentosa, how did the medical community get comfortable with this one? i guess when there have been so many failures in retinitis pigmentosa how did the medical community get comfortable with this one What are some of the findings you had from the earlier studies, and what can you share with us today about that? what are some of the findings you had from the earlier studies and what can you share with us today about that

Speaker 2: Once again, some of them, there are two reasons, right? One is a technical reason, and the other one is financial. When you're developing a biotech, any novel therapies. I think majority of them had mixed results in early-stage clinical trials, and even some of them had good signals. At the end of the day, Luxturna was a great technical success. At the end of the day, commercial viability was questionable, just like many gene therapy products in the marketplace. Unfortunately, we're in the field where when you have a novel technology transformative medicine, people expect significant revenues and commercial success. Some of these therapies, they struggled even though they had good, probably promising, okay data. At the end of the day, you only have that genetic mutation of 50 or 100 patients. It's very difficult to get capital resources to continue those clinical trials. Once again, some of them, there are two reasons, right? once again some of them there are two reasons right One is a technical reason, and the other one is financial. one is a technical reason and the other one is financial When you're developing a biotech, any novel therapies. when you're developing a biotech any novel therapies I think majority of them had mixed results in early-stage clinical trials, and even some of them had good signals. i think majority of them had mixed results in early-stage clinical trials and even some of them had good signals At the end of the day, Luxturna was a great technical success. at the end of the day luxturna was a great technical success At the end of the day, commercial viability was questionable, just like many gene therapy products in the marketplace. at the end of the day commercial viability was questionable just like many gene therapy products in the marketplace Unfortunately, we're in the field where when you have a novel technology transformative medicine, people expect significant revenues and commercial success. unfortunately we're in the field where when you have a novel technology transformative medicine people expect significant revenues and commercial success Some of these therapies, they struggled even though they had good, probably promising, okay data. some of these therapies they struggled even though they had good probably promising okay data At the end of the day, you only have that genetic mutation of 50 or 100 patients. at the end of the day you only have that genetic mutation of 50 or 100 patients It's very difficult to get capital resources to continue those clinical trials. it's very difficult to get capital resources to continue those clinical trials I think majority of those trials, some of them struggled, they failed. Some of them had okay data, they didn't proceed further. Only one company right now is in phase III for XLRP. That's the late stage. I think majority of those trials, some of them struggled, they failed. i think majority of those trials some of them struggled they failed Some of them had okay data, they didn't proceed further. some of them had okay data they didn't proceed further Only one company right now is in phase III for XLRP. only one company right now is in phase iii for xlrp That's the late stage. that's the late stage

Speaker 1: Okay. Okay. okay

Speaker 2: That covers probably north of 5% of entire RP population. That covers probably north of 5% of entire RP population. that covers probably north of 5% of entire rp population

Speaker 1: Got it. All right. Can you, just to give us an idea, are you slowing progression of RP? Are you restoring vision? What should we expect of this upcoming data? What is your actual endpoint? Is it slowing progression or is it actual restoration of vision? Got it. got it All right. all right Can you, just to give us an idea, are you slowing progression of RP? can you just to give us an idea are you slowing progression of rp Are you restoring vision? are you restoring vision What should we expect of this upcoming data? what should we expect of this upcoming data What is your actual endpoint? what is your actual endpoint Is it slowing progression or is it actual restoration of vision? is it slowing progression or is it actual restoration of vision

Speaker 2: Obviously, what you're looking at is, whenever you're doing clinical trials for the RP, we're using mobility test, which is already approved in the Luxturna, a similar one. Obviously, we worked with FDA. We made it more specific and sensitive. We actually validated it in our phase III with actual patients, very robust. Here what you're measuring is you have a treatment group and untreated control group, right, assessor-blinded study. If you're able to show improvement in the mobility test, which reflects your quality-of-life improvement, like if I go to a restaurant in a dull light before therapy, I'm unable to go sit in my chair. I need somebody's help to hold my hand and take me. After therapy, that means I'm performing better in dull light, improve my quality of life. Now I can do it myself in a restaurant dull-light setting. Obviously, what you're looking at is, whenever you're doing clinical trials for the RP, we're using mobility test, which is already approved in the Luxturna, a similar one. obviously what you're looking at is whenever you're doing clinical trials for the rp we're using mobility test which is already approved in the luxturna a similar one Obviously, we worked with FDA. obviously we worked with fda We made it more specific and sensitive. we made it more specific and sensitive We actually validated it in our phase III with actual patients, very robust. we actually validated it in our phase iii with actual patients very robust Here what you're measuring is you have a treatment group and untreated control group, right, assessor-blinded study. here what you're measuring is you have a treatment group and untreated control group right assessor-blinded study If you're able to show improvement in the mobility test, which reflects your quality-of-life improvement, like if I go to a restaurant in a dull light before therapy, I'm unable to go sit in my chair. if you're able to show improvement in the mobility test which reflects your quality-of-life improvement like if i go to a restaurant in a dull light before therapy i'm unable to go sit in my chair I need somebody's help to hold my hand and take me. i need somebody's help to hold my hand and take me After therapy, that means I'm performing better in dull light, improve my quality of life. after therapy that means i'm performing better in dull light improve my quality of life Now I can do it myself in a restaurant dull-light setting. now i can do it myself in a restaurant dull-light setting I can go sit in my chair. That's an improvement. This mobility test actually simulates from bright light to dull moonlight. Our goal is to show the improvement after the treatment compared to untreated control group. That's a quantitative measure. In case of other therapies like Stargardt and GA, you have central vision, lesions or blurriness. What you're trying to do is you're trying to compare the growth of that lesion between treatment and untreated group. If you're slowing it down compared to untreated group, you're measuring it and statistical superiority based on the design, that means you've got a successful outcome. It's benefiting patients. I can go sit in my chair. i can go sit in my chair That's an improvement. that's an improvement This mobility test actually simulates from bright light to dull moonlight. this mobility test actually simulates from bright light to dull moonlight Our goal is to show the improvement after the treatment compared to untreated control group. our goal is to show the improvement after the treatment compared to untreated control group That's a quantitative measure. that's a quantitative measure In case of other therapies like Stargardt and GA, you have central vision, lesions or blurriness. in case of other therapies like stargardt and ga you have central vision lesions or blurriness What you're trying to do is you're trying to compare the growth of that lesion between treatment and untreated group. what you're trying to do is you're trying to compare the growth of that lesion between treatment and untreated group If you're slowing it down compared to untreated group, you're measuring it and statistical superiority based on the design, that means you've got a successful outcome. if you're slowing it down compared to untreated group you're measuring it and statistical superiority based on the design that means you've got a successful outcome It's benefiting patients. it's benefiting patients In all these cases, obviously, as I mentioned before, you don't need to show reversal of the disease to get drugs approved because many of the drugs in the market today, if you look at it, majority of them, very close to probably more than 90%, and they're all slowing down disease progression compared to control arm. In our case, if the patients are able to stall the disease, that's good. If they're able to reverse in some cases I mentioned, that's monumental and it's really truly transformative. Obviously, in the clinical trial, we try to minimize risk as Ocugen. You take endpoints which are already approved by FDA because that has a probability of success is high and low risk, clinical trial design perspective. You can modify them as needed, but it's good to always take the precedence, right? In all these cases, obviously, as I mentioned before, you don't need to show reversal of the disease to get drugs approved because many of the drugs in the market today, if you look at it, majority of them, very close to probably more than 90%, and they're all slowing down disease progression compared to control arm. in all these cases obviously as i mentioned before you don't need to show reversal of the disease to get drugs approved because many of the drugs in the market today if you look at it majority of them very close to probably more than 90% and they're all slowing down disease progression compared to control arm In our case, if the patients are able to stall the disease, that's good. in our case if the patients are able to stall the disease that's good If they're able to reverse in some cases I mentioned, that's monumental and it's really truly transformative. if they're able to reverse in some cases i mentioned that's monumental and it's really truly transformative Obviously, in the clinical trial, we try to minimize risk as Ocugen. obviously in the clinical trial we try to minimize risk as ocugen You take endpoints which are already approved by FDA because that has a probability of success is high and low risk, clinical trial design perspective. you take endpoints which are already approved by fda because that has a probability of success is high and low risk clinical trial design perspective You can modify them as needed, but it's good to always take the precedence, right? you can modify them as needed but it's good to always take the precedence right You don't have to prove this endpoint works. You don't have to prove this endpoint works. you don't have to prove this endpoint works That's what we are doing for all our clinical. We take something which already approved and then implementing what else is needed around it. That's what we are doing for all our clinical. that's what we are doing for all our clinical We take something which already approved and then implementing what else is needed around it. we take something which already approved and then implementing what else is needed around it

Speaker 1: Great. I guess, as you might have mentioned, there are several RP gene therapies that are in development right now. You could potentially be the first to market as a gene agnostic approach. Does this put you in a position to be standard of care? How intense is demand? How important is it to be first? What is your ideal patient here? Obviously, since you're gene agnostic, you could potentially affect everyone. How do you think about Your product versus some other approaches that are being developed right now. Great. great I guess, as you might have mentioned, there are several RP gene therapies that are in development right now. i guess as you might have mentioned there are several rp gene therapies that are in development right now You could potentially be the first to market as a gene agnostic approach. you could potentially be the first to market as a gene agnostic approach Does this put you in a position to be standard of care? does this put you in a position to be standard of care How intense is demand? how intense is demand How important is it to be first? how important is it to be first What is your ideal patient here? what is your ideal patient here Obviously, since you're gene agnostic, you could potentially affect everyone. obviously since you're gene agnostic you could potentially affect everyone How do you think about Your product versus some other approaches that are being developed right now. how do you think about your product versus some other approaches that are being developed right now

Speaker 2: The only one which is in phase III is XLRP from another biotech company. Obviously, in our clinical trial, if you look at it, we had more than 25 genetic mutations covered. We covered all the major prevalence mutations, which are like, if you think something has 3% or 4% of RP, like XLRP, rhodopsin, PDE6B, Ushers, we covered major mutations. Our goal is to get a broad RP indication, and sometimes, some of the patients, it's very difficult to genotype them. If clinically, if they're phenotyped, they have RP, they can be used. We have expanded access program, very rare. We did it because of empathy for our patients, and they don't want to wait. Either they don't qualify for the clinical trial, or they don't want to wait for two years for the product to come out. The only one which is in phase III is XLRP from another biotech company. the only one which is in phase iii is xlrp from another biotech company Obviously, in our clinical trial, if you look at it, we had more than 25 genetic mutations covered. obviously in our clinical trial if you look at it we had more than 25 genetic mutations covered We covered all the major prevalence mutations, which are like, if you think something has 3% or 4% of RP, like XLRP, rhodopsin, PDE6B, Ushers, we covered major mutations. we covered all the major prevalence mutations which are like if you think something has 3% or 4% of rp like xlrp rhodopsin pde6b ushers we covered major mutations Our goal is to get a broad RP indication, and sometimes, some of the patients, it's very difficult to genotype them. our goal is to get a broad rp indication and sometimes some of the patients it's very difficult to genotype them If clinically, if they're phenotyped, they have RP, they can be used. if clinically if they're phenotyped they have rp they can be used We have expanded access program, very rare. we have expanded access program very rare We did it because of empathy for our patients, and they don't want to wait. we did it because of empathy for our patients and they don't want to wait Either they don't qualify for the clinical trial, or they don't want to wait for two years for the product to come out. either they don't qualify for the clinical trial or they don't want to wait for two years for the product to come out We opened it up, and then several patients got expanded access. That's very broad. That's like syndromic, non-syndromic or clinically, if you're diagnosed, you can get it. Our goal is to get the broad indication irrespective We opened it up, and then several patients got expanded access. we opened it up and then several patients got expanded access That's very broad. that's very broad That's like syndromic, non-syndromic or clinically, if you're diagnosed, you can get it. that's like syndromic non-syndromic or clinically if you're diagnosed you can get it Our goal is to get the broad indication irrespective our goal is to get the broad indication irrespective of what their genetic mutations show then. We're not worried about somebody's coming with a single mutation-based gene therapies. As I mentioned before, those gene therapies, remember, the defect is there from birth. of what their genetic mutations show then. of what their genetic mutations show then We're not worried about somebody's coming with a single mutation-based gene therapies. we're not worried about somebody's coming with a single mutation-based gene therapies As I mentioned before, those gene therapies, remember, the defect is there from birth. as i mentioned before those gene therapies remember the defect is there from birth How the dysfunctioning gene or the protein expression is causing the damage to your network, people need to seriously look into that. Genes don't work in isolation. Those gene therapies, you have to be careful when you're intervening with the patients. If they do it early on, scientifically, they should get better results. Whereas we can take care of all the patients. That's our goal. Ideally, any therapy, you want to give it to any patient, you want to intervene earlier than later, right? If the patients who are in the late stage, we cannot deny them, because we're able to show in our clinical trials even the later-stage patients are benefiting. For them, it's all relative, right, Annabelle? How the dysfunctioning gene or the protein expression is causing the damage to your network, people need to seriously look into that. how the dysfunctioning gene or the protein expression is causing the damage to your network people need to seriously look into that Genes don't work in isolation. genes don't work in isolation Those gene therapies, you have to be careful when you're intervening with the patients. those gene therapies you have to be careful when you're intervening with the patients If they do it early on, scientifically, they should get better results. if they do it early on scientifically they should get better results Whereas we can take care of all the patients. whereas we can take care of all the patients That's our goal. that's our goal Ideally, any therapy, you want to give it to any patient, you want to intervene earlier than later, right? ideally any therapy you want to give it to any patient you want to intervene earlier than later right If the patients who are in the late stage, we cannot deny them, because we're able to show in our clinical trials even the later-stage patients are benefiting. if the patients who are in the late stage we cannot deny them because we're able to show in our clinical trials even the later-stage patients are benefiting For them, it's all relative, right, Annabelle? for them it's all relative right annabelle

Speaker 1: Yeah. Yeah. yeah

Speaker 2: If you think, if I'm able to keep my central vision, that's like one of our patients says, I'll be good. If I can just hang on to what I have because I learned how to live with this. I don't want to go further down. That's our goal. We don't want to deny anyone. We want to have broad RP indications, take care of all the patients globally. If you think, if I'm able to keep my central vision, that's like one of our patients says, I'll be good. if you think if i'm able to keep my central vision that's like one of our patients says i'll be good If I can just hang on to what I have because I learned how to live with this. if i can just hang on to what i have because i learned how to live with this I don't want to go further down. i don't want to go further down That's our goal. that's our goal We don't want to deny anyone. we don't want to deny anyone We want to have broad RP indications, take care of all the patients globally. we want to have broad rp indications take care of all the patients globally

Speaker 1: Can you remind us how big your actual trial is and how many patients were able to be enrolled in the expanded access? Can you remind us how big your actual trial is and how many patients were able to be enrolled in the expanded access? can you remind us how big your actual trial is and how many patients were able to be enrolled in the expanded access

Speaker 2: Our actual trial is 140 patients. Our actual trial is 140 patients. our actual trial is 140 patients It's 2:1 ratio. That means a treated group has two to one. You take around 95 in treated and rest of them in untreated group, control. That's the design we had, which you had 140 patients. It's 2: 1 ratio. it's 2 1 ratio That means a treated group has two to one. that means a treated group has two to one You take around 95 in treated and rest of them in untreated group, control. you take around 95 in treated and rest of them in untreated group control That's the design we had, which you had 140 patients. that's the design we had which you had 140 patients

Speaker 1: How many have been able to get access to the expanded access? How many have been able to get access to the expanded access? how many have been able to get access to the expanded access

Speaker 2: Expanded access program, yeah. Expanded access program, we didn't publicly disclose the numbers. That protocol can go up to 75 patients. Expanded access program, yeah. expanded access program yeah Expanded access program, we didn't publicly disclose the numbers. expanded access program we didn't publicly disclose the numbers That protocol can go up to 75 patients. that protocol can go up to 75 patients

Speaker 1: Can that be included in your BLA package at all? Can that be included in your BLA package at all? can that be included in your bla package at all

Speaker 2: Obviously, we have to monitor those patients. The approval is based on the clinical trial, the phase III. This can be added as additional data, obviously. Just like phase I/II, all the data will be submitted for safety or any signals. We'll definitely summarize all that. Obviously, we have to monitor those patients. obviously we have to monitor those patients The approval is based on the clinical trial, the phase III. the approval is based on the clinical trial the phase iii This can be added as additional data, obviously. this can be added as additional data obviously Just like phase I/ II, all the data will be submitted for safety or any signals. just like phase i/ ii all the data will be submitted for safety or any signals We'll definitely summarize all that. we'll definitely summarize all that

Speaker 1: Got it. With the phase III that are coming out in first quarter, you can already submit a rolling BLA. What is it that you can start with in terms of the submission? Got it. got it With the phase III that are coming out in first quarter, you can already submit a rolling BLA. with the phase iii that are coming out in first quarter you can already submit a rolling bla What is it that you can start with in terms of the submission? what is it that you can start with in terms of the submission

Speaker 2: Yes. I think that's an advantage for the company and also the agency, right? We're able to submit a non-clinical module, and we're on target with our PPQ runs successful, and therefore, we'll be able to drop the CMC section this year. That's a big lift from any biotech company perspective. Yes. yes I think that's an advantage for the company and also the agency, right? i think that's an advantage for the company and also the agency right We're able to submit a non-clinical module, and we're on target with our PPQ runs successful, and therefore, we'll be able to drop the CMC section this year. we're able to submit a non-clinical module and we're on target with our ppq runs successful and therefore we'll be able to drop the cmc section this year That's a big lift from any biotech company perspective. that's a big lift from any biotech company perspective We also minimized any risk to our two phase III for RP and Stargardt. We also minimized any risk to our two phase III for RP and Stargardt. we also minimized any risk to our two phase iii for rp and stargardt We introduced actually two commercial scale lots in our phase III to minimize any risk to CMC, where we are very robust in our CMC process. Those two sections can be dropped in this year. That way, as soon as the clinical data comes out in first quarter, once the top line comes in, within weeks, we can drop the clinical module. That's the plan for rolling submission. We introduced actually two commercial scale lots in our phase III to minimize any risk to CMC, where we are very robust in our CMC process. we introduced actually two commercial scale lots in our phase iii to minimize any risk to cmc where we are very robust in our cmc process Those two sections can be dropped in this year. those two sections can be dropped in this year That way, as soon as the clinical data comes out in first quarter, once the top line comes in, within weeks, we can drop the clinical module. that way as soon as the clinical data comes out in first quarter once the top line comes in within weeks we can drop the clinical module That's the plan for rolling submission. that's the plan for rolling submission

Speaker 1: Great. Just wanted to move to Stargardt's really quickly. Obviously, that's another genetic disease. The approach here, this is a much more homogeneous group, and your endpoints are a little bit different. You're trying to change the progression of the lesion. Can you actually reverse the lesion, or once they have a lesion, those cells are completely dead? What is the approach here? You're just trying to slow the progression of lesion expansion, correct? Great. great Just wanted to move to Stargardt's really quickly. just wanted to move to stargardt's really quickly Obviously, that's another genetic disease. obviously that's another genetic disease The approach here, this is a much more homogeneous group, and your endpoints are a little bit different. the approach here this is a much more homogeneous group and your endpoints are a little bit different You're trying to change the progression of the lesion. you're trying to change the progression of the lesion Can you actually reverse the lesion, or once they have a lesion, those cells are completely dead? can you actually reverse the lesion or once they have a lesion those cells are completely dead What is the approach here? what is the approach here You're just trying to slow the progression of lesion expansion, correct? you're just trying to slow the progression of lesion expansion correct

Speaker 2: I think in the clinical trial, all our clinical trials are one year because they've seen an unmet medical need. Other companies may be doing two-year, three-year trials. If you're able to show treatment benefit in one year in your unmet medical need, obviously agencies, FDA may support it, right? They don't want to keep doing two or three-year trials. In this one year, what is the measurable effect it can show compared to untreated arm? That's a lesion. Based on the disease progression, we can show that. You're slowing down the growth of the lesion compared to untreated control. Of course, you'll have other measures, but we don't have visual acuity and ups or down. The nature of the disease progression is slow. Obviously, we pick the primary endpoint, which is important for approval is lesion. I think in the clinical trial, all our clinical trials are one year because they've seen an unmet medical need. i think in the clinical trial all our clinical trials are one year because they've seen an unmet medical need Other companies may be doing two-year, three-year trials. other companies may be doing two-year three-year trials If you're able to show treatment benefit in one year in your unmet medical need, obviously agencies, FDA may support it, right? if you're able to show treatment benefit in one year in your unmet medical need obviously agencies fda may support it right They don't want to keep doing two or three-year trials. they don't want to keep doing two or three-year trials In this one year, what is the measurable effect it can show compared to untreated arm? in this one year what is the measurable effect it can show compared to untreated arm That's a lesion. that's a lesion Based on the disease progression, we can show that. based on the disease progression we can show that You're slowing down the growth of the lesion compared to untreated control. you're slowing down the growth of the lesion compared to untreated control Of course, you'll have other measures, but we don't have visual acuity and ups or down. of course you'll have other measures but we don't have visual acuity and ups or down The nature of the disease progression is slow. the nature of the disease progression is slow Obviously, we pick the primary endpoint, which is important for approval is lesion. obviously we pick the primary endpoint which is important for approval is lesion If you are seeing other effects, what I was talking about, some of the patient videos, it takes time for them to show that like second year, third year, these patients are improving further and further, not just slowing down If you are seeing other effects, what I was talking about, some of the patient videos, it takes time for them to show that like second year, third year, these patients are improving further and further, not just slowing down if you are seeing other effects what i was talking about some of the patient videos it takes time for them to show that like second year third year these patients are improving further and further not just slowing down lesion. They're able to see clearly, their vision is coming back. Somebody may be able to see the faces very clearly, which was lagging before. That's more than just controlling the growth of the lesion. As I mentioned, those things we are not measuring in the clinical trial. Obviously, like all our gene therapies, we have to monitor them for safety for five years, every year on annual basis. When they come to doctor's office, some of these measures we observe. Obviously, with all our BLA filings, if we have early-stage clinical trial data, phase I or phase I/II, and what other data we have, if we have three-year data like we released for RP, or four years, we'll put that. All the data will be there in our BLA submission. lesion. lesion They're able to see clearly, their vision is coming back. they're able to see clearly their vision is coming back Somebody may be able to see the faces very clearly, which was lagging before. somebody may be able to see the faces very clearly which was lagging before That's more than just controlling the growth of the lesion. that's more than just controlling the growth of the lesion As I mentioned, those things we are not measuring in the clinical trial. as i mentioned those things we are not measuring in the clinical trial Obviously, like all our gene therapies, we have to monitor them for safety for five years, every year on annual basis. obviously like all our gene therapies we have to monitor them for safety for five years every year on annual basis When they come to doctor's office, some of these measures we observe. when they come to doctor's office some of these measures we observe Obviously, with all our BLA filings, if we have early-stage clinical trial data, phase I or phase I/II, and what other data we have, if we have three-year data like we released for RP, or four years, we'll put that. obviously with all our bla filings if we have early-stage clinical trial data phase i or phase i/ii and what other data we have if we have three-year data like we released for rp or four years we'll put that All the data will be there in our BLA submission. all the data will be there in our bla submission

Speaker 1: Okay, great. You have an interim analysis coming up for the phase II/III. How is it powered, and what are you looking for in your decision tree? What are the possibilities here? Just maybe you can talk about that a little bit. Okay, great. okay great You have an interim analysis coming up for the phase II/ III. you have an interim analysis coming up for the phase ii/ iii How is it powered, and what are you looking for in your decision tree? how is it powered and what are you looking for in your decision tree What are the possibilities here? what are the possibilities here Just maybe you can talk about that a little bit. just maybe you can talk about that a little bit

Speaker 2: Our trials are powered in about 90%, or in some cases, RP has 95% or more in GA. However, what we're looking for is, it's really important. I mean, it's really fortunate to have this adaptive design. During phase III clinical trial, when you're doing the registration trial, you're able to, not like a broad look, but under very strict guidance of data monitoring committee. They can take a look because in phase I you have a control line. In phase II/III, registration trial, you've got actual true control, untreated control. You're really getting actual data compared to that, and now you have ability to adjust. What are the potential outcomes? They look at it and say, based on predictive analytics, at 50% reaching eight months, or in 12 months it's going to hit it, no change needed. You need to size up. Our trials are powered in about 90%, or in some cases, RP has 95% or more in GA. our trials are powered in about 90% or in some cases rp has 95% or more in ga However, what we're looking for is, it's really important. however what we're looking for is it's really important I mean, it's really fortunate to have this adaptive design. i mean it's really fortunate to have this adaptive design During phase III clinical trial, when you're doing the registration trial, you're able to, not like a broad look, but under very strict guidance of data monitoring committee. during phase iii clinical trial when you're doing the registration trial you're able to not like a broad look but under very strict guidance of data monitoring committee They can take a look because in phase I you have a control line. they can take a look because in phase i you have a control line In phase II/III, registration trial, you've got actual true control, untreated control. in phase ii/iii registration trial you've got actual true control untreated control You're really getting actual data compared to that, and now you have ability to adjust. you're really getting actual data compared to that and now you have ability to adjust What are the potential outcomes? what are the potential outcomes They look at it and say, based on predictive analytics, at 50% reaching eight months, or in 12 months it's going to hit it, no change needed. they look at it and say based on predictive analytics at 50% reaching eight months or in 12 months it's going to hit it no change needed You need to size up. you need to size up That way, if you size it up, add more patients, or in some cases, if you add another time point from 12-16 months, that may also help based on predictive analytics. All these outcome analysis or predictive analytics is based to minimize the risk, further risk for the ongoing phase III with the true control data. When you have that option, it's really very good gift for any company to minimize any phase III clinical trial risk during phase III clinical trial. That way, if you size it up, add more patients, or in some cases, if you add another time point from 12-16 months, that may also help based on predictive analytics. that way if you size it up add more patients or in some cases if you add another time point from 12-16 months that may also help based on predictive analytics All these outcome analysis or predictive analytics is based to minimize the risk, further risk for the ongoing phase III with the true control data. all these outcome analysis or predictive analytics is based to minimize the risk further risk for the ongoing phase iii with the true control data When you have that option, it's really very good gift for any company to minimize any phase III clinical trial risk during phase III clinical trial. when you have that option it's really very good gift for any company to minimize any phase iii clinical trial risk during phase iii clinical trial

Speaker 1: If in this interim analysis you're given the green light to continue, those 50% of the patients you're seeing separation, you don't need to size up. Will the FDA seek perhaps an additional study of more patients because perhaps the 50% of that trial was not enough? Will you continue to enroll that trial based on 140, or will you stop right there and try to file? If in this interim analysis you're given the green light to continue, those 50% of the patients you're seeing separation, you don't need to size up. if in this interim analysis you're given the green light to continue those 50% of the patients you're seeing separation you don't need to size up Will the FDA seek perhaps an additional study of more patients because perhaps the 50% of that trial was not enough? will the fda seek perhaps an additional study of more patients because perhaps the 50% of that trial was not enough Will you continue to enroll that trial based on 140, or will you stop right there and try to file? will you continue to enroll that trial based on 140 or will you stop right there and try to file

Speaker 2: No, I think RP, we don't have interim analysis with ending, like all the patients completing one year. Stargardt. No, I think RP, we don't have interim analysis with ending, like all the patients completing one year. no i think rp we don't have interim analysis with ending like all the patients completing one year Stargardt. stargardt

Speaker 1: Oh, sorry. Oh, sorry. oh sorry

Speaker 2: Stargardt is only 50% to eight months. Stargardt is only 50% to eight months. stargardt is only 50% to eight months

Speaker 1: Yeah. Yeah. yeah

Speaker 2: Our endpoint is actually one year. We continue. Our endpoint is actually one year. our endpoint is actually one year We continue. we continue

Speaker 1: Yeah. Yeah. yeah

Speaker 2: Even though the data comes out good, we continue, and we close it out at 12 months. In geographic atrophy, obviously, that's a package we submitted to FDA and EMA, 300-patient trial, and that one has a different adaptive design. When 150 patients reach one year, that trial has a very good power. Even at 150 patients, I think for the primary endpoint lesion, I think our power is more than 90%. That one has an opportunity. If it hits it, there may be a potential to talk to the agencies and say, continue to collect the data, because it's a large disease and you need 300 patient data minimum for safety reasons. That's how we are using adaptive design to further de-risk our clinical trial. Also, create an upside scenario where, oh, can we file it sooner than later? Even though the data comes out good, we continue, and we close it out at 12 months. even though the data comes out good we continue and we close it out at 12 months In geographic atrophy, obviously, that's a package we submitted to FDA and EMA, 300-patient trial, and that one has a different adaptive design. in geographic atrophy obviously that's a package we submitted to fda and ema 300-patient trial and that one has a different adaptive design When 150 patients reach one year, that trial has a very good power. when 150 patients reach one year that trial has a very good power Even at 150 patients, I think for the primary endpoint lesion, I think our power is more than 90%. even at 150 patients i think for the primary endpoint lesion i think our power is more than 90% That one has an opportunity. that one has an opportunity If it hits it, there may be a potential to talk to the agencies and say, continue to collect the data, because it's a large disease and you need 300 patient data minimum for safety reasons. if it hits it there may be a potential to talk to the agencies and say continue to collect the data because it's a large disease and you need 300 patient data minimum for safety reasons That's how we are using adaptive design to further de-risk our clinical trial. that's how we are using adaptive design to further de-risk our clinical trial Also, create an upside scenario where, oh, can we file it sooner than later? also create an upside scenario where oh can we file it sooner than later

Speaker 1: That's great. In the five seconds that we have left, unfortunately, we're out of time, but can you tell us your cash position and where that takes you to? That's great. that's great In the five seconds that we have left, unfortunately, we're out of time, but can you tell us your cash position and where that takes you to? in the five seconds that we have left unfortunately we're out of time but can you tell us your cash position and where that takes you to

Speaker 2: Yeah. We recently raised a gross $130 million with convert and with all the net and everything proceeds. Our cash runway gets into 2028. What does it mean? That will allow us to file two BLAs next year and also initiate the GA phase III clinical trial to create a tremendous upbeat for the company and inflection points. Obviously, a lot of things are going to be opened up. Stargardt also has a rare pediatric designation, and when you get approval in early 2028 based on our plans and the PRV, it could be worth $150 million-$200 million. Yeah. yeah We recently raised a gross $130 million with convert and with all the net and everything proceeds. we recently raised a gross $130 million with convert and with all the net and everything proceeds Our cash runway gets into 2028. our cash runway gets into 2028 What does it mean? what does it mean That will allow us to file two BLAs next year and also initiate the GA phase III clinical trial to create a tremendous upbeat for the company and inflection points. that will allow us to file two blas next year and also initiate the ga phase iii clinical trial to create a tremendous upbeat for the company and inflection points Obviously, a lot of things are going to be opened up. obviously a lot of things are going to be opened up Stargardt also has a rare pediatric designation, and when you get approval in early 2028 based on our plans and the PRV, it could be worth $150 million-$200 million. stargardt also has a rare pediatric designation and when you get approval in early 2028 based on our plans and the prv it could be worth $150 million-$200 million

Speaker 1: Great. Great. great

Speaker 2: Obviously, we are in a very good cash position. My team is doing a fantastic job executing. In 2022, we started the first patient dosed in our gene therapy trial. Today, we lead ophthalmology gene therapies because of the breadth and the depth we cover with our gene therapy is so high. Large populations, significant unmet medical needs, this cash runway will help us to really focus on programs, what we are good at, and executing, and move all three programs to where they belong. Obviously, we are in a very good cash position. obviously we are in a very good cash position My team is doing a fantastic job executing. my team is doing a fantastic job executing In 2022, we started the first patient dosed in our gene therapy trial. in 2022 we started the first patient dosed in our gene therapy trial Today, we lead ophthalmology gene therapies because of the breadth and the depth we cover with our gene therapy is so high. today we lead ophthalmology gene therapies because of the breadth and the depth we cover with our gene therapy is so high Large populations, significant unmet medical needs, this cash runway will help us to really focus on programs, what we are good at, and executing, and move all three programs to where they belong. large populations significant unmet medical needs this cash runway will help us to really focus on programs what we are good at and executing and move all three programs to where they belong

Speaker 1: Great. Thank you so much for the time. I appreciate the overview. Great. great Thank you so much for the time. thank you so much for the time I appreciate the overview. i appreciate the overview

Speaker 2: Thank you for having me. Thank you for having me. thank you for having me

Speaker 1: Excellent Excellent excellent