Conference Transcript - OCGN 2026-08-11

Whitney Ijim, Analyst — Canaccord

Excellent. All right. Thank you, everyone, for joining us this afternoon. My name is Whitney Ijim. I'm one of the biotech analysts here at Canaccord. And it's my pleasure to be joined by Ocugen this afternoon and the chairman, CEO, co-founder, Shankar Musmuk. So thank you so much for being here.

Shankar Musunuri, Chairman

Thank you for having me.

Whitney Ijim, Analyst — Canaccord

So starting with the high-level overview, sorry, and I've been told to talk to the microphone, so I'm going to face this way. What does the company look like today? And what are you hoping to build over the next five years?

Shankar Musunuri, Chairman

Great question. I think, I mean, we as oxygen today, I believe we lead ophthalmology gene therapies. Why? We have three programs in late stage. If you take the vision loss diseases today with significant unmet medical needs, IRDs, which are inherited diseases, retinitis pigmentosa and stargard, and on the age-related macular degeneration, dry AMD is much bigger, right? it's 90% of the AMD compared to wet AMD is 10%. And all these diseases have significant unmet medical needs. And these are blindness diseases today with very limited therapies or no therapies across the globe. And today I'm proud to say Ocigen is in late stages, all three of them. And we're targeting like two BLAs next year and for both retinitis pigmentosa in Stargard and AMD in 2028. So that's what Ocigen is. So if you're able to get the approvals in all the major markets, US and EU, including Japan, in the next three years and launch these therapies, I mean, that will be a monumental task because that can change the paradigm of all these unmet medical needs across the globe for blindness diseases. Millions of patients can get benefited. Today, as we are talking, a lot of the patients across the globe are becoming legally blind, right? So that's what Ocogen wants to do. Not only take these gene therapies in the next two years, work hard to file those BLAs or market authors in Europe across the globe, get them approvals, but we want to work even harder to provide market access to patients who need them globally. Not after five or ten years in a parallel process.

Whitney Ijim, Analyst — Canaccord

Okay, that's important too. And so for a lot of investors who think about gene therapy for eye diseases, they might be thinking about these like really niche-y gene-specific approaches that are like kind of slow and you've got to go multiple to get a larger opportunity. But you all are doing something different. You're going, you're thinking broader, I guess, with your gene modifier technology, which uses nuclear hormone receptors. So what is a nuclear hormone receptor? Where did the IP come from, how did you identify it, and why was it interesting to you?

Shankar Musunuri, Chairman

Nuclear hormone receptors, these are modifier genes. Simple concept is genes don't work in isolation. They work as a network. So when somebody has a genetic defect, it's there from birth. So you have the misfolded or toxic protein it's producing, right? And that has an impact on network of genes. So, there are genes which are transcription factors responsible in retina for key functions, phototransduction, cell development, metabolisms, cell survival. So some of these key functioning genes are impacted by defective genes, irrespective to what defect it is, right? And so the technology, it identified the missing link, you know. For something like retinitis pigmentosa, mostly a disease that impacts phototransduction pathways, there's a modifier gene, NRTV3, that's invented by Dr. Nina Hyatt-Harvard. And she invented these modifier genes. She was a genetics person. She worked on human genome before. She spent about 20 years inventing these genes. And the invention was, you know, you take RP, you take multiple mutations. Every time there's a mutation, she's tracking it back. Oh, these transcription factors are, you know, getting impacted. They provide key functions. And then, oh, NR2E3 is a link. How about you upregulate NR2E3? Everything comes to sync, you know, cellular human stasis. And then she also worked on RORA, which is another modifier gene, because when you take diseases such as Stargard or AMD, one is inherited, another one is age-related. But they both have same complex pathways for progression of the disease, right? Oxidative stress, lipid metabolism, inflammation complement system. And this other gene regulates all those pathways. It also creates a healthy environment for retinal cells, including RPA, to survive, which is very important in the case of this. RPA has to be protected. And so the technology came from Harvard, from Dr. Nina Heider's lab. She did the original work, and then, of course, Ocugen took it, and they exponentially grew that technology platform. And today, not only the IP covers the ability of our modifier genes in ophthalmology space, we also cover neurospace. That's the future.

Whitney Ijim, Analyst — Canaccord

Okay. Very helpful. So I'm going a little bit out of order here, and we're going to start with Ocufor10 in Starburst, please. And so, I guess, can you review the product here? You briefly alluded to kind of the transgene, but talk about the vector, the transgene, and how it's administered.

Shankar Musunuri, Chairman

Yeah. So, in Stargard and GA, geographic atrophy, which is a late stage of dry agilator macular degeneration, in both the cases, of course, you have the macular, central vision issues, you have lesion. There are complex pathways. And so if you take current therapies, the two products approved for GA in the U.S., they target complement system, right? And many companies worked on that. So if you take that pathway, that's a final frontier, you can call it. It's the last pathway. But the origination is caused by oxidative stress and drusen formation, which is lipid metabolism. Then you get inflammation. So a lot of these are important, too, very important. So, this RORA regulates all these pathways, and we have data in one of those nature publications. We demonstrated that. And so, not only it regulates all these pathways, it also has the ability to reset cellular homeostasis and create a healthy environment for these cells to survive. There are certain cells in our human system, right? You take our retinal cells, or you take like a neural, and some of them, and even cardiac, some of the key things we are looking at. And these are all non-dividing cells. And if you take these non-dividing cells, that's why as we age, we have issues, right? Everybody has these age-related diseases. But if there is anything we can trigger to reset them at the cellular level, that would be phenomenal, right? So that's what the genes do. you know your anti kind of aging kind of a trigger reset the cellular homeostasis and then you are creating a healthy environment for these cells to survive so that means at least you should not go down where you are that's a concept right if you in some patients if you're able to stop the progression that's great if you're able to reverse it that's even phenomenal and we're seeing that with both the genes in r2e3 some of the patients are improving further and further not only what they have. Some people who got loss of peripheral vision, they're getting it back. Some people have central vision issues, RP, they're getting it back. RP patients are getting central vision back. They're seeing much clearer now. So I think in all these cases, it's reversing it too. So that's a difference. I mean, any modality you take for GA, even for Stargard, you know, oral therapies, they may hit on that one pathway, oxidative stress. It may slow down disease progression, but those modalities cannot offer you stopping the progression or reversing it. It's only possible because this modified gene therapy platform has the ability to do that.

Whitney Ijim, Analyst — Canaccord

Okay, perfect. And just to dive into Stargard a little bit more, we talk about, I talk about Stargard first, we tend to focus on that one because there's a direct comp here. There's another company, different mechanism, a different route of administration, but targeting Stargard in the filed, it's like a six billion dollar company. So I think sometimes I bring up Stargard and you're a gene therapy company, so people will say, well, what niche of Stargard are they going What's the small subset of patients they're going after? Can you talk about that? Are there specific subsets or aspects of the disease or patients you're enrolling, or is this also a broad start?

Shankar Musunuri, Chairman

I mean, Stargard is a very complex disease. I mean, obviously, if you look at our phase three, we go from three-plus pediatric all the way to adult, early to advanced stage. Within that segment, I mean, of course, if you look at the oral therapy, you're talking about, I mean, the lesion size, too. I mean, they went on a very low lesion size, 7 and below. And so there is a difference. And also, their clinical trial focuses on age group of 12+. So there must be a reason for that, right? And so, I mean, obviously, agencies look at all these safety issues when you go to pediatric population. So they restrict up to a certain age group. And we were asked to actually go down to 3+, to cover the population. We also have a rare pediatric designation with our StarGuard. So the StarGuard, we have a combined, the study got, it's a U.S. study, but it got endorsed by EMA. They like the design. We did only small phase one because of unmet medical need and promising data we saw in phase one. FDA allowed us to convert our phase two into phase two, three total trial. And then EMA said, that's fine. You don't need any other clinical trials, right? So the clear difference, again, I'm going back. So they are focusing on oxidative path, right? So that modality, I'm not saying it's bad. It's one of the key factors. But you have to take it oral medication every day. And if it slows down a disease progression compared to control, that's fine. When you don't have anything, that's great. But you have to be compliant. You have to take it all your life, right? And whereas we come back and show after our therapy, because our modifier genes based on all the nature publications, all the data we're getting from all these programs over the years. And if you're able to not only slow down, in some patients it's stopping it. Some patients are divorcing it. And it's one and done. That's it. You take one injection back of the eye, you're done. So that's a huge difference. And also, depending on the effect, the efficacy and safety are very important when you launch any, let the data dictate. If the data comes out, all the things I'm talking about, and this is going to be a paradigm shift, it could create a gold standard of care where anybody else with other modalities will have a tough time crossing that. Right? I mean, so that's a differentiation. Asia. For sure, for sure.

Whitney Ijim, Analyst — Canaccord

Okay, perfect. So phase 2-3 running, as you said, a pivotal study with a mass interim coming up that could kind of help de-risk the ultimate outcome, and remind us when are we expecting top-line data from the final study?

Shankar Musunuri, Chairman

Yeah, the phase 2-3, we're anticipating second quarter of next year, the top-line results, and right after that, within a few months, we'll do the BLA filing and market authorization in parallel. That's the plan. So that means if everything goes according to the plan, sometime in 28, you should get approval and launch. Yep. Yep. Perfect.

Whitney Ijim, Analyst — Canaccord

Okay. And then same gene, but moving over to geographic atrophy. Briefly, I guess, can you talk about the differences between 410ST, the Stargard program, and 410 over GA? Are there meaningful differences between the constructs or is it a nomenclature term?

Shankar Musunuri, Chairman

I mean, the construct is the same. I mean, obviously, Stargard is a rare disease. It has its own path. The VGs, I think, the amount of gene therapy, the RORA we use, the concentration is different. It's a different dose. Stargard has a different dose than GA. And so that's the difference. I mean, obviously, they have their own regulatory path. One is a broad disease. The good thing is probably we're the first company to get regenerative medicine advanced therapy for AMD. It's a huge disease. And based on the promising data from phase two and unmet medical need, before FDA gave a green signal for us to go into phase three, same week they gave us RMAT designation, which will be very beneficial. Why? Because typically the pedophilates for any big therapies which are not orphan is 10 months. In reality, they take longer than 10 months because they're regular products right or not orphan significant so in this case having the tarmat is almost like a inclusive of you know fast track and breakthrough encompassing for gene therapies and it'll allow us to get a accelerated approval when you do the filing in six months which is very important right and also as you and there are a lot of other things benefits you get post marketing commitments to channeling with FDA during the phase three clinical trial you know anytime you want to have meetings because of unmet medical need or mad they jump on calls with you so they're supposed to collaborate with you to take this therapy to the market because of unmet medical need and the promise so I think those are all the elements we have in GA. So we are trying to embark on a, you know, phase three, about to get started this quarter. It's a single trial with, we already got green light from FDA. We're working with the MA, and hopefully in the next few weeks to months, we'll be able to align. So it'll be a single global trial.

Whitney Ijim, Analyst — Canaccord

Okay, perfect. We will be looking for updates on that. And last but not least, least as far as the product discussions go, is OCU400, which we could start with as well because that's the nearest term pivotal readout coming in the first quarter of next year. So for OCU400 and RP, what's different about the transgene here? You mentioned NR2E3. So why does that make more sense in RP versus ROR for the other two?

Shankar Musunuri, Chairman

So NR2E3 works mostly on the transduction pathways. And also when you take RP, you start with, you know, rods. And so I think we're regulating those transduction path, photostransduction is important for ARP patients because they lose the peripheral vision, night vision first, and the central vision. Whereas GA and Stargardt, it's almost like a macula. They've got central vision issues. So I think, so RORA is better regulating those pathways. So that's how we differentiated it. And so we have good evidence in those nature publications. They're very methodically done. Then we went to the clinic.

Whitney Ijim, Analyst — Canaccord

Yep. Okay. And on the clinic, can you talk about the key takeaways from the phase 1-2 study? And I guess what is new or different in the phase 3 design versus the phase 1-2? And, well, I'll stop there.

Shankar Musunuri, Chairman

I'll talk to you later. So the phase 1-2 study obviously is still a controlled study, but the contralateral is the control. we had 18 patients with multiple mutations and the rhodopsin is one of the big ones because typically most of the rhodopsin mutations are autosomal dominant that means if you try to give a traditional gene therapy you can get a gain of function which is actually a gain of dysfunction right because the the defective gene is so powerful they can turn the functioning gene signal into more negative right so that's why a lot of traditional companies didn't attempt because that could be safety impact i mean if you take entire rp rhodopsin is the biggest of all the mutations 10 to 12 percent of rp population so we took that tough population in phase one two we showed very promising results right and so if you take we also use the same similar i can't say the same mobility test it's a functional test because these patients lose peripheral vision, night vision, so if they can walk through a maze with obstacles, that's the first product got approved, using the same endpoint. With agencies, if you use the endpoints where they gave approvals, you have a low barrier to pass, right? So at least you're aligned, you're not trying to get a new endpoint and prove it's clinically meaningful and all that. So that's good. So we took that, and when we looked at the phase 1-2, obviously, sometimes you got a because it's a safety study you got a very high risk population too and some of them are on the almost like a ceiling effect you can call it on that mobility test there that means you know you're already we stopped at let's say at one lux level you're already at one lux level you may be improving as a patient you can come back and say oh I can see something in the peripheral but I can't test you on the primary end point right so we had patients like that and so if you take the demographic of patients will fit into phase three, ability to pass few levels. And phase three also went to 0.04 levels. We made it more specific and sensitive by working with FDA. They helped us out. It's a very good design. And so we had about 63% of the patients in phase one, two, with invisible for phase three, pass like two levels compared to untreated eye, which is zero. So that's a good result, right? Then, for designing phase three, obviously, we refined that test further, make it more sensitive and specific to, based on FDA input. And then, we decreased that buffer. We put some buffer in there, like, okay, if you have 50%, if you hit responders, and then, even though control population, like control-related light didn't hit, None of them actually have two levels. Okay, assume like they're 10% failure, right? Or 10% of control, if at all they reach. So based on that, we came up with 95% power reaching, passing two levels, responder rates, right? So in a real life, I mean, if control subjects don't pass, they have zero, you don't even need to get 45, 50%. Much lower number responder rate, it will pass. Why that's important? Laxana got approval with 30 patients, okay? And our phase one, we had 18. Our phase three has 140 patients with two-to-one ratio. And we had more than 30 mutations covered in the phase three, so most comprehensive, early to advanced stage, pediatric to adult, you know, most complex genetic medicine trial to date globally, period. All the genetic medicine FDA approved to date, they had less than 50 patients. That's it. So this is most comprehensive. And then in phase three, we took 30 patients. Laksana got approved with 30 patients, actual patients. We validated the course. It's very robust. The variability in the course is almost zero. That means a patient walks on a certain level multiple times, changes everything. It will only fit on that level. So I think it's very robust. So that's what we are using in phase three. So, I mean, those are all good elements of our clinical trial, which is validated course and also refined course by closely working with FDA and data supported by phase one, two, going into phase three. We built some buffer into that. We took all those precautions. In addition, we'll continue to monitor low luminance visual equity as a secondary. And there are some PGAs, patient quality, all those things we're collecting. LLVA is very important because this mobility test is very complex. Tomorrow, when you get the product approved, or even the current phase 1-2 patients, we track them for three years. We're going to have four years later next year before we do the VLA filing. It's very difficult to do mobility tests. It gets very complex. But on an annual basis, when they go to the doctor's office, low luminance visual equity LLVA is an easier test. It's more accurate and more sensitive for these patients, because most of these patients with these IRDs, even for dry AMD, the low-light visual equity is a first target. Definitely BCA will have an impact on the longer term, but LLVA is a good measure for all these patients. So it's easier to measure in the doctor's office. So that's a long-term measure. In fact, in RP, we showed most of these patients are about two-line improvement on the treated eyes using LLVA consistently, three years, which is good. We also show durability. And another thing with all our therapies, they're subretinal, right? It's a small retrectomy. They're going directly into the retina because you want to express the gene where the effect is needed. In RP as well as Stargard and GA, Stargard and GA, you can have multiple blebs, so you get a more surface area for gene expression, so you get the impact. And so all these targets we have going into I lost the thought. So with a going into RP with a phase 3 clinical trial, so LLVA becomes a long-term measure okay for payers that's what I want to bring it back because you want an easier measure that's why you're doing LLVA because mobility test is very complex okay for approval up to one year we'll monitor because all the gene therapy the reason I brought all the gene therapy programs we have obligation to agencies to monitor for safety for five years okay after one year is for approval but even phase one two will continue to monitor so we also treated more than 325 patients across all the clinical trials, including expanded access program for RP, but more than 50 patients in EAP. So a lot of data coming up, almost like more than 200 patients treated for RP. So we don't have any SAEs, serious adverse events related to our gene therapy in all these populations. So we have good safety database.

Whitney Ijim, Analyst — Canaccord

Right, right, strong safety database. Okay, that's super helpful. So phase three data coming in the first quarter, as I said. Talk to us about the BLA filing. I think you're on track to start that actually ahead of the data. Is that still the case?

Shankar Musunuri, Chairman

Yeah, yeah. So we, good point. A lot of gene therapy companies, I think CMC is very complex. So we are ahead of the curve. Most of the, all our phase three clinical trials, we did whatever is needed, you know, not taking a lot of favors. We introduced two commercial-scale lots for both RP and Stargard in our Phase III. And then we have successfully completed our PPQs, validation runs, needed for BLA submission for RP. And that same material, the supplies can be used for commercial. We already have launched supplies for RP. So we're in a good shape. So non-clinical and CMC sections are ready to go, the modules. So obviously, under RMAT, we have eligibility to file rolling submission. Obviously, we had to work with FDA, their timing, their clock, and their resources. So we agreed, and FDA suggested when the top line comes in, talk to us at the pre-BLA meeting. At that time, maybe we'll allow you the sections already. And so that will still give them several weeks' head start before we drop the clinical module. So I think that's where we left off. Yes, under RMET, you're eligible, but you still have to work with the agency. Gotcha, gotcha. Okay, so the target is first quarter top line, and within months after that, we'll file the BLA completion. But the ruling BLA submission doesn't have any impact on completing BLA filing approval clock. Approval clock starts with final BLA module. That's clinical. Six months from there. So if you do it in second quarter, fourth quarter is potential approval.

Whitney Ijim, Analyst — Canaccord

Right, right. Okay, got it. And in the last 15 seconds, you recently converted $130 million convertible note financing, extended the runway into 2028. We've kind of talked a little bit about that, but just quickly review, what are the key milestones that that capital gets you through, and how should investors think about spend, particularly as you start before 10 phase three?

Shankar Musunuri, Chairman

The good thing is the spend won't increase that much next year, because we've got two phase threes this year. We completed recruitment, and those things next year, they're completing them, right? So their spend is going to come down, and the GA is going to take over. So mostly our financials are going to stay the same. 130 should get us into 2028. And obviously, we announced another regional partnership. On the BD side, we're very active. I mean, U.S., we want to be very opportunistic because there's a lot of value in U.S. for our gene therapies. We'll work with payers. But ex-U.S., you know, even though a lot of us came from big pharma, we can do global commercialization. but, you know, it takes a lot of effort and time to establish infrastructure, we are evaluating partnerships. So based on how much of money we can raise non-deliative funding through partnerships, we're going to evaluate next year. And then if we need to rise, we want to be sensitive to our shareholders. And if we need any more equity rise, we'll rise it to support our commercialization in the U.S. So that's the plan.

Whitney Ijim, Analyst — Canaccord

Okay, perfect. Very helpful, very interesting story. So we are out of time, but thank you very much for that overview. and thank you everybody for coming to listen.

Shankar Musunuri, Chairman

Thank you.