Okay. Thank you. Good afternoon, and thanks for joining us to have a conversation with Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. Ocugen is a biotech company focused on gene therapies for blindness diseases. Unlike traditional gene therapies that correct a single mutation, Ocugen's modified gene therapy platform targets master regulatory genes that control multiple gene networks, supporting a gene agnostic approach across a broad range of mutations and across complex eye diseases. So that platform now supports three late-stage programs with three planned BLA submissions. The first of that, OCU400 in retinitis pigmentosa with top-line liMeliGhT data expected in the first quarter of 2027 and a rolling BLA tied to that data. OCU410ST is in Stargardt disease, with top-line from pivotal phase II/III GARDian3 trial expected in the second quarter of 2027 and a BLA to follow that. And in terms of OCU410, which is in geographic atrophy, which just started the study, the phase III study, ArMaDa3 trial, with a BLA targeted in 2028. To discuss these three BLA strategy and also the path all the way from here to 2028, let's get started with Shankar. Shankar, glad to see you. Thank you. And appreciate you for accepting our invitation and talking to our audience today. For those who are new to Ocugen, could you just frame for us the long-term strategy around these three planned BLA submissions that you have? And how sequenced are they in terms of operations, and what all needs to get done from here to the next 18 months? Yeah. Thank you for having me to tell the Ocugen story and where we are. Obviously, it's a big undertaking when you have three phase III programs and they target Inherited Retinal Diseases and AMD, which is dry AMD, which is much bigger than wet AMD, right? These three programs encompass majority of the vision loss diseases today, which have a significant unmet medical need. All our therapies are potentially one-time treatments for life. That's going to be a monumental difference in patients across the globe. In the next 18 months, as you stated, the top-line data from OCU400 targeting broad retinitis pigmentosa, early to advanced stage, pediatric to adult, it covers all the patients. RP can be caused by multiple mutations. Mutations in over 100 genes. The goal is to cover entire population. Between U.S. and E.U., we got about 300,000 patients. It's large. Our goal is to get the top-line, get ready for BLA submissions. One of the most important thing we learn from the industry, mistakes or positives and negatives, the negatives are CMC. Yeah. If you go back and look, at least in our team, I'm very proud of them, did a fantastic job, and they have successfully completed PPQ runs, process validations. We introduced two commercial scale lots in phase III. We don't take any risk from CMC perspective. Non-clinical and clinical, the CMC modules are ready to go. As soon as the clinical data comes out, we'll discuss with FDA, we'll try to file that ASAP. That'll also prepare us to follow that with the market authorization in Europe. Why? Because EMA waived clinical trials for both RP, OCU400, and Stargardt, OCU410ST. They don't need any additional work. The clinical trial in U.S. is good to file there. We'll follow through. What does it mean? I think next program is Stargardt in a few months later, if everything goes according to our plan. And again, we will get the top-line, we will file the filings in U.S. and EU. GA is a large disease burden. Thanks to FDA, from end of phase II meeting to dosing patients in phase III, record time. I think very proud of my team, within four months. Not only FDA reviewed it, they gave Regenerative Medicine Advanced Therapy designation, which is given based on the promising data in early stage and significant unmet medical need. There is a significant unmet medical need, even though there are a couple of products in the market. in U.S., which do not exist in EU. That program, the clinical trial, we assume about 9- 12 months of recruitment. By 2028, second half, it should be done. As soon as the top-line results come, we will file the BLA. We will be ready. The RMAT designation gives potential priority review. Instead of waiting for 10- 12 months, regular therapies, you can get an accelerated. That is a big plus for a large disease like that. What it means, number one, we need to be very good with our filings, getting them on time. There is an old saying, "Get it right first time." We want to do a good job. We want our team to do a good job so everything runs like a clock with FDA and EMA. Yeah so we get less questions. If a quality of filing is good, we can get through it. Number two, we need to prepare for commercialization. Obviously, you are seeing the deals we are announcing ex-U.S. Obviously, even though some of us came from large pharma, as a growing biotech, it is not practical to think we will go and launch everywhere. We need to be prepared. We will definitely focus on strategic partnerships outside of U.S. In U.S., first and foremost, there are multiple things we are doing even before top-line results come out, which we can do without spending a lot of capital. Looking into, number one, talking to payers, including CMS, lining the top and creating centers for excellence. Because vitrectomy, all our therapies go after subretinal injection. Even though all retinal specialists are trained, it is a small surgery. It is not rocket science. However, we did very well with our clinical trials. More than 300 patients dose. Among all our trials, we do not have any SAEs related to our product, the gene therapy. On the surgery side, we mastered something. We want to make that cookie cutter. We want to create centers for excellence for delivering those gene therapies so that not every specialist touches. Yeah. We had to create those. We are working behind-the-scenes work. Getting into patients registry. Then comes the hard part, getting into sales and marketing, all the strategic initiatives. Obviously, we are mindful of the capital. That work is going to start as soon as top line comes in. Then we are going to exponentially grow and ready for, at least in the U.S., potentially launch. Because we can create a lot of value for our shareholders if we launch on our own in U.S. and ex-U.S. That is the prep we are doing. Once you do the prep for IRD, like take RP, we got more than 100,000 patients in U.S. Then in 2028, 2029, GA comes, geographic atrophy. We are talking about 1 million-2 million patients in U.S. Then we are ready for it. Yeah. We can scale up in U.S. That is the plan for commercial. We are doing some work behind the scenes, which is not capital intensive. The capital intensive, we want to be careful. Once the top-line results come up, we will have a plan, we will execute it. Very good. The next question, I am not asking for a science lesson, but what is the rationale for modifier gene therapy, and why do you think that is different from other gene therapies that people are looking at? Yeah. There are two aspects of it. One is technical complexity. You take something like retinitis pigmentosa. Defects in over 100 genes can cause it. If you take traditional gene therapy, you need more than 100 products. That comes to number two. Commercially, they may not be viable. Gene therapies, when they started the first gene therapy, it was priced below $1 million. Then they went all the way to $4 million a patient. But where are these companies? Unfortunately, in our industry, it is very clear you can have a cutting-edge science. Gene therapies are transformative medicine, no question about it. However, commercially, if they are not viable, that means you get a very unique technology to the market. If you do not make blockbuster revenues in our field, market considers it a failure. The technology is not failing. They targeted very good targets. Some of them have 50 patients, 200 patients, even 2,000 patients. That is a big difference. Our gene therapies, either you take IRDs like RP or big disease like AMD. AMD has multiple problems. In a simple sense, it has multiple pathways. Yeah. Other modalities targeting one pathway at a time, you saw the results in the marketplace. The disease may be controlled a little bit, but may not be optimum for patients. If you control with a one-time therapy, you do not need multiple injections for patients, almost like six to nine per year. One injection, one and done for life. That is a big difference between other gene therapies and us. We are going after, first time, it is very disruptive. Not 200 patients, not 2,000 patients. Hundreds of thousands of patients, millions with a one-time potential treatment for life. That change is a big paradigm shift on the commercial potential for all these. We have a lot of flexibility on pricing, and however you cut it, there are significant revenue potential we can generate. Okay. Let us get to the little wrinkle that has happened, which I do not think is an issue, but we need to cover it. This is on the news that you put out from the IDMC, which happened on September 3rd. They did a pre-specified analysis of the 26 subjects who were treated with the OCU410ST. Can you walk us through what the DMC actually saw and what are they suggesting? Yeah. Let's talk about Stargardt before I go into that. It's a very complex disease. Mutations in our, it's a large gene, but we cover more than ABCA4. ABCA4 itself has 1,200 mutations. There is a lot of heterogeneity in patient population. Also, there is also lesion size. If you pick the therapy which is awaiting approval, the oral, they use a very narrow band. Our phase I data looks very promising. Some of the patients actually, the patient video, not only it slowed down the disease progression, actually it's improving. That's only possible with our gene therapies. Not only slowing down disease progression, it can stall it in some patients. In some patients, it can reverse. But to measure that, it takes time. We're already doing a lot. If you look at the entire spectrum of therapies out there, irrespective of what modality they used, RP, Stargardt, GA, nobody is doing 1 year of trials. Why is Ocugen doing it? Number one, there is a significant unmet medical need. There is a desperation for patients. Agencies understand it. They saw promising data in early-stage trials. They're allowing us to go into one-year trials. It's very efficient, so it can get to the product to the market sooner than later. Number two, Stargardt, obviously, we had limited data in phase I, which looks very promising. Based on that, we assigned a trial phase II. FDA allowed us to call it phase II-III, and it has got over 50 patients. Obviously, the goal was pre-specified DMC. It's a blinded review, so nobody gets to see it. It's a master review. Confidentially, a small team looks at it, and it's for any adjustments. Because it's a small trial and we have a limited population, RP at least we had larger patient population in phase I, too. GA had a lot more patients from genomic medicine perspective. So you have ability to adjust. But the other hand, one year itself, what we are doing is a shorter window. Right? But we had good data because we were able to show that most of the people in this field did two-year trials. Looking at eight months, there is a heterogeneity in the population lesion size. Unfortunately, as they stated, we don't have data. They looked at the partial data of the demographic. They looked at the full data set, and the lesion size didn't favor according to that report. We transparent way shared with the market. That's number one. When they looked at the total data set, they said it's going to be neutral. That way, remember, it's statistics, right? When you have a smaller data set, variability goes high. You go to a larger set, variability come down. Then you can see the differences. They suggested it's very clear the lesion differences also get neutralized. You won't see the difference. That's why it's meaningful to look at the data when your complete data set comes up either eight months or we can look at 12 months. The study is for 12 months. In a nutshell, what is the final recommendation? Continue. That's important for market to understand. We, in a transparent way, shared whatever DMC gave us so that we can explain. That's what we did. Whatever they said, the negative effect, we talked about it, but the important thing for investors is the study is continuing. All our studies have good promising data from early-stage trials. We'll continue. We also upgraded our team. We have a retinal specialist who's a CMO now. We upgraded, we hired more resources into that, and we're going to navigate. We know our gene therapies work. It's not just collecting quantitative data. Our patients speak for themselves, and they have testimonials coming from RP, coming from Stargardt. We are confident we are going to navigate, get all these therapies to the market in the next few years. Sometimes you get little bumps, and we have a team which can navigate that, and there's a significant unmet medical need, and FDA has seen the data, and they're very supportive in what we are doing. EMA is endorsing them. We'll navigate. This is a small blip. It's a little confusion in the marketplace. The bottom line is the study is continuing, and I think people have to patiently wait until top-line data comes out in one year. That one year itself is a shorter timeframe compared to any other therapies out there. All of them use two-year timeframe. Okay? If the time goes on, we can generate a lot more data, too. Turning on to OCU410 and GA molecule. You dosed your first patient on September 1st after that Type B in a phase II meeting in July after aligning with the FDA on the endpoints. What are the most important concessions or alignments that you got in that meeting, and how do you see that study, not only enrollment but also trying to stay on time? Yeah. Good points. The phase II data for the optimal dose looks very promising, and we had a good meeting with FDA. In fact, after many years, it's the first face-to-face meeting. They're meeting face to face, which is very. The whole group was there, very supportive. The clinical trial based on the size we saw, effect size, of course, we don't take the entire effect size. We had about 31% lesion reduction. That's primary endpoint in one year. Why that's important? Because again, it's a very efficient trial, one year. If I do two-year trials, can I look at visual acuity, something else? Sure. I can look at it. But one year, if you look at the disease progression based on natural history, we need to be practical. What endpoints make sense, quantitatively show the difference? Lesion made sense. FDA agreed with it. That's number one. Primary endpoint is set. We had a 31% decrease in phase II in the population. Also about 20% in the optimal dose, the lesion actually stabilized. It didn't grow. That's very important. Yeah. That's why our gene therapies have ability to do that, not only slow down, and some patients may stall. Some, if you wait for longer time, like two or three years, it may even reverse. Again, that's telling based on RP and Stargardt. What, number one, they agreed on endpoint. Number two, we built some more buffer for statistical analysis. We didn't take 31% we're going to hit. We took a smaller number than that. Then we came up with 237 patients. That gives us 95% power for primary endpoint. It's pretty big. Minimizing any risk, what we can do. The secondary endpoints are ellipsoid zone, which correlates to functional vision, and also low luminance visual acuity. There, obviously, they're not powered, but we need to look at some trends. That's all. That's a mandate, and that's what we're looking at. It's a pretty straightforward trial. Right now, obviously, my team did a fantastic job getting the sites up and running and started dosing. We're going to also, in addition to U.S. and Canada, look into Europe. It's a large disease burden. We're not going to get a waiver from EMA like RP and Stargardt. This is a big disease, and we are actually looking into European centers now. We're going to open European centers, get the buy-in from EMA for the same trial, and that will be part of the global clinical trial program, U.S., Canada, Europe. We're hopeful the recruitment may take nine months to 12 months, but by 2028, second half, we'll have the top-line results. The plan is expect to file the BLA in late 2028 sometime. That's the plan. That's going on track. We are, again, thankful to the FDA. Not only based on the data, they cleared our IND amendment very quickly to move into phase III. They also gave Regenerative Medicine Advanced Therapy designation, which is rare for large diseases. Typically, it's given to orphan. Why in this case? Two things. Promising data they have to see in early-stage clinical trials, clinical data. Number two, the disease has to have significant unmet medical need. This allows us to get a potential, as I mentioned before, priority review, six months approval clock instead of longer, 10- 12 months for regular therapies. The immediate catalyst is the first quarter 2027 phase III data from OCU400 for RP, which will be followed by a rolling BLA. Operationally, what has to go right between now and actually completion of that submission of the BLA? Operationally, as I mentioned, the two modules are already done, like non-clinical and CMC, and the focus is all on clinical. Make sure all these patients come back for their visits on time. Make sure everything is QC'd and all the things you do in our industry. All the patient data is in and data lock occurs on time. I think we have a good team now, and they're watching everything, so we don't miss anything. They're on target to get the top-line results out in the first quarter. As soon as they come out, FDA is willing to meet with us, and we'll have a pre-BLA meeting with them right after that and align a plan, and then we'll keep moving. When the data comes out in the first quarter, do you plan to wait to present it in a medical conference, or how will you telegraph that? Again, we have multiple elements we have to discuss. The most important thing is getting in alignment with FDA more than anything else. That's the most important for us. Obviously, they are our collaborators from a regulatory perspective, and first and foremost important thing for Ocugen is to work with FDA more than anything else. All those things will follow afterwards. Okay. That's what we are focused on. In the interest of time, you closed the second quarter with $130 million and also retired some debt. With that cash, what sort of a runway should we expect from them? We're going to extend the runway into 2028. Two of these programs are almost a little bit complete. The third one will be almost complete. Obviously, as a growing company, we have to continue to watch capital mobilization. The two things we are working on. One, obviously, we're working on potential partnership deals ex-U.S. We are going to watch and see how much of non-dilutive funding it brings in by the time top-line results come. There may be other avenues of supporting commercialization at that time. It doesn't have to be equity. There may be other instruments we can use, royalty, including others. Our goal is to minimize dilution to our existing shareholders, look into other opportunities, then if necessary, we can continue to raise equity too. That should extend our runway. We're always watching like any other growing biotech because we are also very mindful of dilution to our existing shareholders, and we'll carefully orchestrate and manage our capital mobilization. Thank you, Shankar. Thanks for taking time and being here. And good luck, I know there's a lot. Thank you. Thank you for having me.