All right, good morning, everyone. So we're a biotech company focused on cell and gene therapies and vaccines for public health. Forward-looking statement. We have three first-in-class platform technologies, starting with our modifier gene therapy, mostly focused on blindness diseases. The first program, OCU400, entered into phase III recently. This targets retinitis pigmentosa, which covers about 100 genes, single product. I'll go into more details a little later in the presentation. We have Regenerative Medicine Advanced Therapy designation from FDA, as well as orphan drug designations from FDA and EMA. T he second program, OCU410, which targets dry age-related macular degeneration, the late stage, is called geographic atrophy. Currently, it's going through phase 1/2 clinical trials. The next one, OCU410ST, which is targeting Stargardt disease. Stargardt disease is a big orphan disease, about 40,000 patients in the U.S. alone.
Currently, that's going through phase 1/2 this year. Coming to our another Regenerative Medicine Advanced Therapy platform, it's targeting cartilage repairs. This is first-in-class. It's a 3D technology. There is only one regenerative cell therapy product in the marketplace today, that has got a 2D technology. Currently, once again, this is, we have Regenerative Medicine Advanced Therapy designation, significant unmet medical need. We actually built our facilities in Malvern, Pennsylvania. This is a very complex science. So we can take your cartilage cells and what you can grow, we can grow them in a year. We can actually grow them in these tabletop bioreactors in 1-2 weeks. And QC release and test it and, you know, send it back to the surgeon in one to, about 6-8 weeks' time.
So currently, once again, we're going to initiate the phase III, you know, contingent upon funding because this requires a two- to three-year, it's a two-year trial. The next one is inhalation vaccine platform. It's first-in-class again. And we currently are collaborating with NIAID. And they're going to conduct our clinical trial this year. By mid this year, we're planning to file the IND. Then it'll go into clinical trial. So our goal is to really focus on our modifier gene therapy. That's the primary focus of the company. And other platform technologies will be very opportunistic for collaborations and everything else to move those programs forward and create value for our shareholders and patients. So today, I'm going to focus most of my talk on my modifier gene therapy platform.
So if you look at all the breakthroughs coming from 20th century to 21st century, starting with penicillin to recombinant DNA, in the 21st century, you got human genome fully, you know, sequenced and, gene and cell therapies. Then you got first mRNA vaccines. Several gene therapy products are getting approved in the last three years, especially. And we believe we have something, a breakthrough technology, which can make a big difference in patients' lives, you know. If you take a traditional gene therapy or, CRISPR, you can go only one gene at a time. And if you take something like heterogeneous disease, like retinitis pigmentosa, you can have 100 genes which can cause RP. And there's no way pharmaceutical companies are going to develop 100 products. That's where we come in. So the modifier gene , they can one product has potential to treat all those diseases.
Similarly, we can go after big diseases like dry age-related macular degeneration. 260 million patients globally struggle with it. Top 10% of those patients are late-stage Geographic Atrophy. So we have our modifier gene can target that. So therefore, we believe we can make a significant difference in impacting lives in future years. So what are modifier gene s? These are like master regulators in retina. And, they have ability to control, functional network from cell development to, inflammation, metabolism, to survival. They reset the homeostasis in retina, restore the function, and create a healthy environment for cells to survive. That's very important because when you have a defect, it's not just a defect. Epigenetics clearly shows there may be cascading effect. There may be other genes which are really important, and they're all depressed.
So what these genes do, they have ability to control the functional network and all underlying gene expressions. If the good genes are suppressed, they have ability to, you know, upregulate them and control the bad ones. So because of that, we have first program OCU400 targeting RP, which affects about 300,000 patients in the US and EU alone. EMA recently agreed that we can use US data. We don't need any additional trials, to apply for market authorization in Europe when you're done with the US. And OCU410, again, it targets dry AMD. It's a big disease burden in the US itself. About 10 million patients under dry AMD. 1 million of them struggle with late stage geographic atrophy. So when you take retinitis pigmentosa and Leber congenital amaurosis, LCA has about another 25 genes.
Between two of them, what the patients look for, most of these patients have loss of peripheral vision, night vision first, and then central vision left. Eventually, they lose that too. So whatever stage they are in, you know, if they have central vision left and they're still, you know, independent and semi-independent, they would like to, you know, stay that way. For them, preserving the vision is a big thing. And if there is a way to even, you know, improve and restore a little bit what they lost, that'll be really phenomenal for these patients. So when you're doing these clinical trials, we always try to see with the gene therapy, can we at least preserve what they have? And if we can improve, that'll be phenomenal.
And so where we are with our first program, once again, to recap, this is targeting retinitis pigmentosa and Leber congenital amaurosis. And they cover about 125 genes. With a single gene agnostic, modifier gene, we believe we can potentially treat all these patients. 1.6 million globally struggle with it. About 300,000 patients in the U.S. and EU alone. And so where we are, we have completed phase 1/2. Based on the data, we worked with FDA. We moved on to phase III clinical trial. So we're the first company to get a broad RP indication with a gene therapy to move into phase III. And there is currently only one product which is approved about 5 years ago. It's called Luxturna. And it treats one genetic disease out of those 125 genes.
Our goal is to, you know, complete this clinical trial and get ready for approvals in 2026. This is a summary of the Phase 1/2 clinical trials. You can see 89% of the patients either preserved or improved in the treated eye with one of the measures, either BCVA or low light visual acuity or mobility test. Mobility test is a functional test which we're moving forward into phase III. That's very relevant for these patients. That means your ability to walk through a maze with different light conditions. And, if I can go sit in my chair in a restaurant in a dull light without much support, that's a big improvement for these patients. And again, 75% of the patients have either preserved or improved, with the mobility test, in these studies.
About 80% of the patients, rhodopsin patients, rhodopsin covers about 10%-12% of RP. It's a big population. Some of it is autosomal dominant. Sometimes you have to be careful treating autosomal dominant genes with additional gene therapy. Sometimes you may get gain of function. We have shown in our clinical trials, a lot of those patients are rhodopsin patients. They did well. In fact, 80% of them have shown either improvement or preservation with the mobility test. So rhodopsin actually proves the gene is working as a gene agnostic because we are not giving rhodopsin to fix rhodopsin genetic defect. We are giving a modified gene NR2E3 and showing improvement in rhodopsin patients. So that itself demonstrates what we demonstrated in animal studies, the two Nature publications.
And now we are showing in human studies, you know, it's working the way it's supposed to work in a gene agnostic approach. So the phase III clinical trial, what we agreed with FDA, it's a two arms. One is gene, rhodopsin because it's 10%-12% of the population. Other arm is gene agnostic. We have multiple mutations. Anybody with RP, you know, early stage, including pediatric population 8+, mid stage or late stage, as long as you can meet the, you know, those two requirements, the BCVA and ability to improve two levels in the mobility test. That's important. So if somebody is on the edge, you know, they're not qualified for the test. And then they won't be in the trial. So as long as you qualify any RP patients get into trial.
So it's like both these arms are powered, more than 90%. And we also looked into responder analysis. You know, that's very important. So that means there is an untreated group, 2-to-1 ratio, 50 to 25 in each group. And if the untreated also need to have responders, you know, to compare, right? So if they don't have any responders, you don't need, you know, much response in the treated group. I mean, typically, the way we powered these studies is you're expecting 50% of the people respond in treated and 10% in untreated to cover risk. And, and so that's how the study is powered. But in the untreated, typically, with the sensitivity and specificity for this test is improved compared to, you know, what people have used in the past. That's why we call it LDNA, Luminance Dependent Navigation Assessment.
And if untreated shows, you know, no response, and typically you're not supposed to see it, untreated population, and you don't even need 50% respond, you can even go down to show the effectiveness. And that's how it's powered. So in a nutshell, it's a two-arm study, Rhodopsin and gene agnostic. We'll include all RP patients who meet the inclusion criteria. And it's a one-year duration. And now taking the data from our phase 1/2 Rhodopsin patients, who can meet this criteria? It's like a post hoc analysis because some of the patients in phase 1/2, they ended up actually on the edge, you know. And if you can look at that, you know, range levels, in phase 1/2, it only goes to 0.1. We went actually below that to 0.24. And FDA actually suggested you don't need to have uneven distribution of those baseline levels.
You can actually uniformly distribute. That's the blue levels at the bottom. See those nine levels? So this actually, it's good. It, it actually benefits. So that's why if you look at the post hoc analysis, those five patients who actually can show improvement because they're not on the edge, and if you they'll fit into phase III clinical trial. If you do the re-analysis, like those blue lines or bars are like a, you know, potential phase III patients who meet. And then based on the LDNA in phase III, the same patients with the orange, you know, bars, they're in phase 1/2 , you know. That's the mobility test we used. So typically, the phase III levels go up. That means, the mobility test is a little more, you know, evenly distributed. So you'll see a better response in phase III in a nutshell, okay?
So here it's showing 60% response rate looking at the post hoc analysis of phase 1/2, which is very exciting for us moving into phase III. So where are we today? Again, we initiated our phase III. We opened several centers. We're screening patients to dose shortly. We'll be dosing. And so our goal is to, it's a one-year duration. It'll take some time to recruit these patients. And, since we have RMAT designation, it's an accelerated approval. So we can start with the rolling submission sometime late next year. And, we'll finish the clinical parts, you know, early 2026. Once you're done with that, it's a six-month clock to get approval. And, we'll also do the parallel track with the EMA. We'll file the market authorization in parallel to BLA with FDA.
So our goal is to get this product approved for large populations in the US and EU first, and then, you know, including other global markets we'll start looking into. So switching gears, now I'll go to the big disease burden, dry age-related macular degeneration. Dry age-related macular degeneration is a tough disease. It's caused by four different pathways. Oxidative stress, as we all age, we have less antioxidants reaching into that part of the retina. And then because of that, the lipid metabolism issues, and they create aggregates. And then you have inflammatory response. Then you form complement system. So there are four different pathways, you know, oxidative stress, lipid metabolism, inflammation, complement system. There are two products approved last year. It's unmet medical need. And they both target complement system. There's a gene therapy, by one of the big pharma. They're also working on complement system.
But that's not the cause of the disease. The disease starts with oxidative stress and lipid metabolism, okay? So that's the difference. Our another modified gene RORA targets all those four pathways. And the current therapies, you need 6-12 injections, which is very difficult the rest of your life. And also, they have a moderate decrease in lesion growth in these patients, about 30% after 2-year treatment. And about 10%-12% are getting neovascular treatment with dry AMD. You're getting like wet AMD. So there is a significant issue there from, you know, adverse event profile perspective. So if you take that and you take, RORA, you know, or OCU410, it's a one-time potential treatment for life.
They have potential to stall, you know, and preserve what you have, not just continue to go down even after treatment, and also potential to even reverse because these genes are very powerful. So here, to demonstrate that, we have generated data from all those four pathways. What I'll do is I'll start from bottom right. That's an ABCA4 model. That's a defective ABCA4 defective gene. That's also applicable to Stargardt disease. That's what the defect is in Stargardt patients. So in this, defective gene, I mean, as I mentioned before, when you have any defect, other good genes are depressed. In this ABCA4, it's not the ABCA4 defect is causing CD59 suppression. So the green line in normal animals is a CD59 expression. In untreated, that line is missing. See that? You only see the blue.
When you treat it, CD59 expression comes up with the RORA gene. We are not giving CD59 gene, just like one of the big pharma is working on the clinical trial. They're using CD59. We are giving a modified gene RORA. So it modifies the expression of other genes in the network. That's what exactly it's demonstrating, okay? And so not only that, we looked into all our modified genes, NR2E3, NR1D1, and RORA. RORA has the most effectiveness in treating with antioxidative properties. So that's what you're going to see. And survival of the cells, under, you know, oxidative stress. And then on the left top, you see the drusen. The lipid metabolism causes aggregates. It's a drusen formation. And it's significantly reducing its after treatment. And the right top, you got anti-inflammatory properties. So this gene is very powerful.
It, you know, treats all those four pathways, which is really a fundamental issue with treating this disease, AMD. So the Stargardt is another disease, as I mentioned. So we are going through this program, that also has about 40,000 patients in the US alone, which is, currently no approved therapies. So with this, you know, these are the milestones for this year. Our goal is to really aggressively recruit patients in our phase III clinical trial, continue to provide updates to the market this year. And we're also going to come up with the preliminary efficacy and safety data from OCU410, which is geographic atrophy, big, big, disease. People are waiting for the data. And we're going to provide something this year in the second half. And similarly, from OCU410ST, also we'll provide, some preliminary results of safety and efficacy.
We're also evaluating partnership, and regional partnership, global partnership. Hopefully, this year, we'll be able to announce that. So with that said, I would like to play the patient video to close out. This is a patient after 1 year of treatment with Rhodopsin. And you're going to see the results. You know, he started getting it back, actually. He's getting his peripheral vision back. Very excited to show this and share how impactful this is. I was able to see right here this movement on my hand. I didn't used to see it. Now I can see the movement. I don't see it clearly, very clearly, but I can see a movement there. And that's the change. Sorry. Of my life, I knew about this illness. And now I have a hope. Since I was a little boy, I knew about this illness, retinitis pigmentosa.
But I never had a problem. I was about 43 years old when my condition started being worse little by little, taking part of my vision away from me. So what retinitis pigmentosa is actually a whole collection of diseases, each of them caused by different mutations. The most common course of the disease is to begin with night blindness. The second big change is they start losing side vision. 37 years in the postal service. My eye had to be in good shape to be able to do my job. At each of the stages, there's a loss, a sense of loss, and sort of a grieving process. Patients have to sort of move on and try to adjust to a new set of capabilities. And once they've done that, something else happens, and they have to adjust to that, you know.
And eventually, you know, I think the last part of the disease, when all that's left is sort of a central island, and that starts closing in, and they can actually tell from month to month, you know, that it's getting smaller. It's a tough psychological challenge. When you have this, and it goes little bit more, little bit more, little bit, you don't really realize the situation. They told me about Ocugen. I started the study. They did the surgery. I can see a little bit more in some areas that I didn't used to. You know, it was kind of dark, totally dark. For a lot of people, it's not much. But for me, you know, it's a big step. I don't have to be guided. I don't have to ask to be helped, to be independent in a way.
I'm so thankful for the company and for the doctors and researchers with this study. My hope was to stop the illness and get more than that, get information, educate yourself so you can help yourself and help others. This is once again a remarkable recovery from this patient. I mean, there are a lot of patients like this. For them, you know, what the message is, whatever he has left, if he can preserve that and show that independence, it's a big thing for him. That's why he's very, very excited about it. He thought he's going to lose whatever he has. He also says, actually, told us he can see cars in his, you know, rearview mirror sometimes in the peripheral vision when he's driving. That's remarkable, you know, because other than these patients, they keep losing it. Eventually, they'll become legally blind.
That's the fear they have. You know, for us, you know, it's an orphan disease, hundreds of thousands of patients, not a few hundred, a few thousand. And we think, you know, it's not a number for them. It's a whole family that gets impacted, not just the patient. So we are really honored to treat these patients. And we believe our therapies are going to be big, impactful globally in the coming years. Thank you.
Yeah. Sorry. You said that the drug is gene agnostic.
Yeah.
Can you describe a little bit about the mechanism, maybe how the two drugs compare, how similar is 400 to 410?
Yeah. Yeah. So the 400 you're targeting in retinitis pigmentosa, like Leber congenital amaurosis. So this is a group of inherited retinal diseases. It's not a single disease. That's why about 100-125 genes can cause this disease.
So if you take a traditional gene therapy approach or even CRISPR gene editing, you need, like, for each genetic defect, you need one product, right? So that's impossible to make those, especially when you got 100, 125 genes. So what these genes do, when you have what we found out, I mean, there are Nature publications, there are two publications, a lot of animal models, you can see it. So when you have a defective gene, even these modified genes are depressed. So when you give NR2E3, it resets the homeostasis and restores the function. That means not only by giving NR2E3, that expression, as soon as it comes up, it will start working on the entire network. And all other genes which are depressed, which are needed for the function, their expression levels will go up, okay? And then they'll restore the function.
And that means the defective gene, you're not really trying to fix or augment it, but other genes have the ability to take the function and also create a healthy environment for cells to survive. That's really important right now because these are non-dividing cells. What you have is what you have for life. And if you reset it and create an environment. So that's talking about that's why so one product like OCU400 is a potential treatment for all those patients with RP and LCA. That's why FDA allowed us to go in there. The second one is OCU410. So here, the RORA gene specifically, once again, these genes have the ability to reset the homeostasis, okay? So your function of lipid metabolism, oxidative stress, are not working well. That's why it caused these aggregates. They got, like, you know, inflammatory markers like cytokine.
Then it leads into AMD, wet and dry. And so if you go to the core of the problem, how do you reset it? That's what RORA gene does. It starts re-regulating and resets the whole regulatory pathways and makes sure everything is functioning. That's what RORA gene does and all these four pathways and go to the core of the disease. So that's why we believe it has the ability to preserve what you have and stop progression, the lesions. Data will tell in a few months and this year. And also, it has got the ability to even, you know, restore or, you know, restore part of the things you lost. Again, time will tell in clinical trials. But these genes are very powerful. And they also autoregulate. You don't need a lot of doses. We target, like, 10 to the 10 gene copies.
What we found in phase 1/2 for the first program, we didn't find much dose relationship. Even the low dose was effective. High dose is also very safe. So we believe you need a threshold for these genes to work. You don't have to give a lot. Once it reaches threshold, it'll self-regulate.