Ocugen at H.C. Wainwright: Progress in Gene Therapy for Blindness

On Thursday, 25 September 2025, Ocugen Inc. (NASDAQ:OCGN) presented at the H.C. Wainwright – Biotech "On Tap" 2025 conference, highlighting its strategic focus on gene therapies for blindness diseases. The company outlined its plans to submit three Biologics License Applications (BLAs) within the next three years. While the conference underscored Ocugen's progress and optimism, it also noted the challenges of bringing these therapies to market.

Key Takeaways

• Ocugen plans to submit three BLAs by 2028, beginning with OCU400 for retinitis pigmentosa in 2026.
• The company is focusing on global market access for its gene therapies.
• Recent divestment of Neocort will allow Ocugen to concentrate on its core gene therapy programs.
• Ocugen's cash runway extends into 2027, supported by a recent $20 million investment.
• The Phase 3 Limelight trial for OCU400 is underway, with top-line data expected next year.

Financial Results

• Ocugen has secured a cash runway into 2027, aided by a $20 million investment and a potential $30 million option.
• The company's quarterly burn rate is about $12 million, totaling $50 million annually.
• The divestment of Neocort, valued at over $100 million, will help strengthen Ocugen's balance sheet.

Operational Updates

• OCU400 (Retinitis Pigmentosa):

- Phase 3 Limelight trial is enrolling 150 patients with top-line data expected in 2025.

- BLA submission is targeted for 2026, with EMA agreeing to accept US clinical data for European market authorization.

• OCU410 ST (Stargardt Disease):

- Phase 2/3 registration trial is underway, using an adaptive design to potentially accelerate approval.

- Twelve-month data shows a 48% reduction in lesion size from baseline.

• OCU410 (Geographic Atrophy):

- Phase 1 data indicates a 27% reduction in lesion growth from baseline at six months.

- Twelve-month data is anticipated in the first quarter of next year.

Future Outlook

• Ocugen is committed to submitting three BLAs by 2028, aiming for global market access.
• The company plans to continue developing its core gene therapy programs, including OCU400, OCU410 ST, and OCU410.
• Monetization of the Neocort stake is expected to further support these initiatives.

Q&A Highlights

• OCU400 Endpoint:

- Primary endpoint is a luminance-dependent navigation assessment, offering more sensitivity and specificity than previous tests.

• OCU410 ST Benchmarking:

- Success would mean replicating Phase 1 safety and tolerability, with superior lesion growth reduction compared to control.

• OCU410 (GA) Comparison:

- OCU410 shows superior lesion growth reduction compared to existing drugs, with a 27% reduction at six months.

Readers are encouraged to refer to the full transcript for more detailed insights.

Full transcript - H.C. Wainwright – Biotech "On Tap" 2025:

Unidentified speaker: We're good to go. Okay. Good afternoon, and thanks for joining us to have a conversation with Shankar Moussunari, CEO, and Huma Kumar, CFO I mean, CMO of Ocugen. Ocugen is a biotech company focused on discovery, development, and commercializing novel gene and cell therapies. The company's proprietary gene therapy platform has generated numerous clinical candidates with, potential for three BLA submissions in the next three years starting in 2026.

So the first BLA submission is targeted, in in 2026 for the with the lead clinical candidate, OCU 400, which is currently in phase three for treatment of retinitis pigmentosa or RP. The second drug is the is against Stargardt disease, which which which is, OCU four ten ST, and then the third one is against geo geographic atrophy or GA, which is o q four ten. That would that is targeted in 2028. So to discuss these three assets and this you know, did this company strategy itself, I invite Shankar and Huma to this conversation. So glad to see you, boss.

Thank you. And thanks for accepting our invitation. So, Shankar, just to start off, especially for those folks who are not who are new to Ocugen and how would what how would you help them understand what Ocugen stands for and especially some of the the technology that, you know, you folks have developed?

Shankar Moussunari, CEO, Ocugen: Yeah. First and foremost, we are focused on gene therapies targeting many blindness diseases. The three targets we have, retinitis pigmentosa, Stargardt, and chagrofic atrophy, which is part of dry age related macular degeneration. They should cover and compass majority of the blindness diseases or vision loss diseases significant unmet medical needs we have today. So our goal is to, as you stated, our team works very hard.

And in fact, we started dosing our first patients in 2022. And today, we are leading ophthalmology gene therapies because of the depth and the breadth of targets we have. And these three therapies, we are planning to have three BLAs in the next three years, and that should have a significant impact on these patients globally. So Ocugen's mission is to not only take these therapies to the market as we are planning for the next few years and also work even harder to provide market access to patients who need them globally.

Unidentified speaker: And then in terms of your, you know, the the platform itself, which is the modifier gene therapy platform, Huma, can you just describe for us a little bit about the technology itself?

Huma Kumar, CMO, Ocugen: So, our novel modifier gene therapy, platform, there are two products. It's OCU four hundred, and there is OCU four ten for geographic atrophy and for Stargardt as well. Let me start with OCU four hundred as our Limelight modified gene therapy, trial is up for the BLA next year in 2026 broad RP indication. What it is is that, we have only one approved product. I'll try to, give some, you know, background so that people are able to understand what we do and what's the relevance of that.

So Luxturna is only approved product, which has been covering only less than one percent of r b sixty five mutation of the entire RP landscape. Correct. We are the only, I should say, game in town for broad RP indication. Ninety eight to ninety nine percent of the patients could have a potential therapy in the next year or so. What does it mean is that we are resetting the the homeostasis homeostasis and and and and improving improving the outer nuclear layer.

What does it mean is if you look at the laptop, you shut it down and you just shut it you know, open it back on. It just resets everything. So for these patients, particularly, it's virtually, like, would say, impossible to have one product for more than 100 mutations that we have at this moment. So, our trial, Limelight, is covering not only clinical and or genetic diagnosis, but all more for syndromic and nonsyndromic forms as well. On the other hand, the second novel, modifier gene therapy that we have is four ten, which is a unique four way mechanism of action.

The proof products only cover complement system. We are covering all four, including not only complement system, oxidative stress, lipid metabolism, and reducing inflammation, which is right from the pathophysiology starts from the pathophysiology of the disease.

Unidentified speaker: Fantastic. So let's start with the OCU 400 because that's the key program, and it's just coming up, you know, to a BLA next year. If you can help us understand the design of the study and also, you know, the two patient populations that you are trying to address here?

Huma Kumar, CMO, Ocugen: So our Limelight trial, which is OCU 400, is phase three, and we have actually one fifty patients or subjects that we're going to be recruiting, seventy five in the rhodopsin, and seventy five in the gene agnostic arm, fifty being active, and twenty five being controlled. Our primary endpoint of the study is, to look at, the baseline change for the lux levels, and we're also looking at the treatment treated as well as the untreated kind of, like, you know, the difference between the lux level improvement. The secondary efficacy endpoints we're looking into is low luminance visual equity, and why we have chosen that is very consistent across all our gene therapy programs. In our phase one two, we have established not only our safety, tolerability, efficacy, but also durability at twenty four months.

Unidentified speaker: So when we start thinking about these two patient groups, do you expect the data to look different within the two groups, or is it just so that you can cover all of the RP?

Huma Kumar, CMO, Ocugen: So it's a great question, and I'm glad you have asked that. So our RP, Ocufour hundred is for early to advanced cases. So if you start looking at from 2017 to 2025, many products have failed still so far. And, also, there's only one approved product for r b sixty five. Correct.

So the pathophysiology can be a little bit different in terms of clinical symptoms and also reaching at the age maximum as 40, and their symptoms remain the same, but there is no difference. We are actually hope to all those patients with unmet medical need, whether it's rhodopsin, whether it's all gene agnostic, which covers all ninety five percent or more of the mutations, not only nationally, but also globally.

Shankar Moussunari, CEO, Ocugen: I think going back to the basics of science, what she explained. Right? So our genes go after fundamentally cellular homeostasis and resetting it

Unidentified speaker: Mhmm.

Shankar Moussunari, CEO, Ocugen: And creating a healthy environment for photoreceptors or retinal cells to survive because they're nondividing cells. That's the difference between our technology and everything else out there. So because of that, we target the entire disease. You take RP, target the entire disease. You take Stargardt, you got about twelve hundred mutations that can cause the disease from ABCA four.

If If you take genetic link, you need 1,200 products. So we go after the cellular resetting the homeostasis at cellular level. I think that's that's a very powerful concept. And what we are seeing in our clinical trials again and again, we have four publications in Nature demonstrating how these molecules or modified genes are working. And now we're replicating those results in human subjects consistently across all three programs.

Unidentified speaker: So since since, you start talking about the clinical data, can you highlight some of the data that you have seen so far in your prior trials so that gives, that gives confidence that the phase three should should should be positive?

Huma Kumar, CMO, Ocugen: So are you talking about, the four hundreds? 400, of course, twenty four months, durability, safety, and efficacy has been promising. We have till to date, no serious adverse events related to the investigational across all our programs such as inflammation, which is the hallmark of the gene therapy. So that is that is one aspect. The second is in terms of, our LLVA at twenty four months, we saw the durability, which is more sensitive and specific marker for, you know, visual equity.

And that means you are improving the quality of life of patients. And they're basically when you go to the optometrist or ophthalmologist, you see the vision chart. And if they're able to gain that, you know, with a p value of 0.025, it's clinically meaningful and statistically significant. So that's what we have seen across multiple mutations and over the period of two years.

Unidentified speaker: And then can we if we can spend a a couple minutes on your primary endpoint, which is the fifty two week change Mhmm. In the luminance dependent navigation assessment.

Huma Kumar, CMO, Ocugen: Yes.

Unidentified speaker: So what is this endpoint, and how should we think about that in terms of what sort of a change we should see between a treated patient versus a plus you know, if it is a placebo?

Huma Kumar, CMO, Ocugen: Okay. So we do have a control in this as 50 patients. And what we can see I'll I'll I'll tell you a little bit about luminance dependent navigation assessment is basically a mobility test, and it is derived basically, if you look at MLMT was the basis of approval for LUXTRANA as well. And but they were not sensitive and specific at that time. They had zero to six levels.

We have zero to nine levels, which means we have uniform correlation between the lux intensity and lux levels. It is more calibrated, sensitive, and specific because the heterogeneity of retinitis pigmentosa is more, and we're covering early to advanced cases. What does it mean is that fifty two weeks? Within a year from the baseline, we are looking at the improvement across all mutations, and that can be between the treated and the untreated control or the control that we are looking into. So that is that is what we have made this course very sensitive and specific and that we anticipate that that would be giving the improvement in those patients.

So there are two things. We are looking at improvement. We're also looking at the photoreceptors of these patients are present as a part of the inclusion criteria that we have. So I hope I answered your question.

Unidentified speaker: Yes. And so, Shankar, so as we started off this conversation, it's saying this program is built for a BLA in 2026. What's how is the progress going with the with this current study? And then when should we see some top line data so that, you know, you can still file in 2026? And would this dataset be good for filing in Europe as well?

Shankar Moussunari, CEO, Ocugen: Yeah. Two questions. First, 2026, obviously, it's SSR blinded study, so we cannot look at the data interim during the clinical trial. Number two, so the data will come out next year. And, obviously, the BLA, as soon as the data comes out, there are two things critical things you need for BLA.

One is the CMC. A lot of gene therapy companies struggle, which are doing really well with the quality of the product. And we're already on target to complete our PPQ runs in support of the BLA this year. So that's a big thing, which will be out of the way. So as soon as the data comes out, the top line results come out.

Within few weeks, we'll file the final section. Under RMAT designation, when you have regenerative medicine advanced therapy with FDA, you are eligible to file rolling submission. Mhmm. So we can start the submission anytime before the clinical data comes out, get get, you know, the nonclinical section, CMC sections in. As soon as the clinical comes out, it also gives the agency error of time alert, and they can start reviewing.

And as soon as the clinical data is dropped, that's where your six month clock starts. So we we can do that next year. The second thing is EMA. EMA agreed with our US design, and they said we don't need any clinical trials in Europe. So with this clinical data we have on this single trial in US, they agree we can file for submission for market authorization in Europe.

And so they started already allocating resources. They confirmed it will go through a centralized procedure sometime this year. So they're getting ready for that.

Unidentified speaker: Thanks. And then regarding the second program, which is the OCU four ten ST. So before we go into the program, what is Targardt disease? Because some folks probably don't know what it is. And, you know, in terms of the study itself, what needs to be completed, you know, in in terms of not only the study, but also if there's any additional work that needs to be done before you file the BLA?

Huma Kumar, CMO, Ocugen: So good questions. I'll try to make it simpler. Stargardt disease is also a form of inherited retinal disease. It is primarily the disease of the pediatric population because it gets manifested there. Majority of the patients do not get diagnosed because of, you know, the genetic component or the clinical piece associated with it.

But once they are diagnosed, it's in the advanced stage, which means they cannot differentiate between sharp objects. There's blurriness of patient, and they have no other approved product as of right now globally anywhere that could be the potential answer to, either reverse or stabilize whatever they have. What we are doing here is that there are ABCA four mutations that are responsible, as Shankar has mentioned, greater than 1,200 mutations. And, of course, the variation or the pathophysiology of the disease is the bull's eye lesion or the, lesion that we have to we have also used as an endpoint, in our phase two slash three right now as a reduction. Not only that, their quality of life in terms of functional vision, which is also low luminous visual equity, gets impacted as well.

So what does it mean for, the patient? They are going to get legally blind very soon even at the between the ages of 35 and 40. So our trial, and our product, OCU four ten s t, as I've said, the unique four way mechanism of action with the ABCA four model. What we are doing at this point is that we are conducting a phase two slash three registration trial that we are actually got win from the agency with an adaptive design, unique adaptive design. And the purpose of that adaptive design is to look at the sample size re estimation towards like, you know, when we have 16 treated and eight controlled subjects and that's true control.

And that will actually dictate us for further actions such as we can even go for approval ahead of time. This is also shaving off two to three years of the framework regulatory process that is very cumbersome and it means that the potential hope for the patients globally as well and nationally. In terms of the design, we have fifty one patients overall, thirty four in the treatment, and seventeen in the control. And it is also for early to late stages of Stargardt disease.

Shankar Moussunari, CEO, Ocugen: And pediatric to adult.

Unidentified speaker: Yeah. Yes. Yeah. Since there is nothing to compare against in the sense there's no drug or anything else, so what would you deem as a positive when the data comes out, and how should we benchmark it so that, you know, people like me can understand whether this is something that can get through the regulators and be meaningful in the market?

Huma Kumar, CMO, Ocugen: The first and the foremost thing that everybody asks is if, you know, how we have conducted our phase one. We have not only demonstrated safety and tolerability, no serious adverse events related to the product, no adverse events of special interest. So that's actually pretty good. Established our maximum tolerable dose in that way. Phase two slash three, we have been consistently releasing our data, and particularly for twelve months as well where it says a reduction of 48% from baseline, which is pretty remarkable for that timeline, which is and and these are as as we have mentioned early to advanced cases and the pediatric population three years of age and above.

So I hopeful and the data is trending in the right direction, and we are looking into lesion size reduction, which eventually would be which is the hallmark of the pathophysiology of the disease.

Shankar Moussunari, CEO, Ocugen: So that's the primary endpoint. So if you if you show statistical superiority, the degeneration and or the lesion growth, you're slowing it down compared to control untreated arm. So I think, as she stated, we have very promising data in phase one if you replicate it and if we show the, you know, statistical superiority to control group. And then the secondary endpoint, visual equity, we're also monitoring. So

Huma Kumar, CMO, Ocugen: So I forgot to add one thing. Thank you, Shankar. A single subretinal injection, one and done treatment. Yeah. That's what it is.

Unidentified speaker: Yeah. So in in terms of the GA, the third indication, obviously, are couple of approved drugs out there. So when you start comparing OCU410 for this indication, how should we think about the data that would that would come out at the end of phase three versus what people have seen with with the true approved drugs?

Huma Kumar, CMO, Ocugen: That's a great question. So first of all, I'll talk about safety. It seems like, you know, it is given, but for geographic atrophy as this is a disease population, we are looking at 50 years of age and above. Right? And they were comorbid conditions like diabetes, cardiovascular, all of the above.

Safety, in in the proof products, there were cases of CNV, endophthalmitis, and if you look at the package insert, that was a big talk of town. We have not reported any serious adverse event related to the investigational product, which is Ocufor ten g a and adverse events of special interest. So that is the first hallmark we are looking in as a first competitor. Second is the from the efficacy perspective, of course, structural growth reduction was was something that they looked into, which was not really statistically significant or clinically meaningful, but there was unmet need there. That's that's what it was.

What we have been consistently looking in our phase one is and phase two, we have released the data at six months for 27% reduction from the baseline, which means there is a true control, high dose and medium dose and a control, which means there is one:one:one randomization and that along with Phase I data consistently showing improvement in the LLVA. So LLVA is something that also is a hallmark of this four ten GA program and you know there is no approved product in GA and they are looking into the functional parameters. So we are covering it at all fronts. We are looking at structural. We are also looking at functional.

So I think this would be an amalgamation of both. We have not come up to an agreement as of right now. The data from Phase two will dictate that. But I think we know that we are going to address structural as well as

Shankar Moussunari, CEO, Ocugen: And functional.

Huma Kumar, CMO, Ocugen: And functional. Yeah.

Shankar Moussunari, CEO, Ocugen: So I think yeah. That's it. So the two products have structural Yeah. And and and and then safety. And and we have structural as well as functional, and it's one and done and stuff every month or every other month.

Correct.

Unidentified speaker: In terms of the data, at least from the phase one study, I believe we have seen up to six months at this point. So what additional data could we see from that study, especially for the the GA product?

Huma Kumar, CMO, Ocugen: So phase one we have twelve months. It's actually six months for phase two. And that's what we are going to like, you know, continuously, you know, talk about we'll we'll consistently talk about. Safety is has all been already been established about efficacy parameters such as lesion growth reduction and LLB as as well. And if you look at that, this is one of the key quest questions I have been asked that, you know, what's the comparator?

So if you look at the Pexitoco plan studies in OAX and Derby trials, there is like even at twenty four months, there is only reduction of 16 to 18% versus the preservation of retinal tissue that we have seen 27%. We also have seen 0.31 versus what they had even we out, you know, at three months or six months, we were way above the curve. And if you look at the twenty seven percent at six months is is more as compared to twenty four months that Pexitocoplan had at six and eight and and eighteen months. So I think I think we are trending in the right direction, and this is clinically meaningful and statistically significant.

Shankar Moussunari, CEO, Ocugen: And and so we're also going to because the functional endpoint, typically, structural, you'll start seeing the effect with gene therapies sooner. Functionally, it takes time. I think by nine to twelve months, just like we saw in phase one study, by the time we get the twelve months data in the first quarter next year, that should also show, as she stated, LLVA trends. And and if they continue like the we are seeing in phase one, think that'll give us a very good indication getting into phase three. I mean, with gene therapies, the data we released in about 25 patients after six months is remarkable.

Unidentified speaker: So I I know we are running out of time, but it'll be a remiss if we don't talk about your divestment, right, of the neocard asset. So in general, you know, what's your commentary on on the divestment itself? And what how should it help your balance sheet?

Shankar Moussunari, CEO, Ocugen: Absolutely. Yeah. Currently, we have I mean, obviously, our focus for the last few years is gene therapies. I mean, that's why, as I stated, 2022 started dosing first patient. Today, have three programs covering the breadth of many blindness diseases.

So we really want to focus on gene therapies and do and become a global leader. Therefore, the amazing technology we have on cell therapy side for cartilage repairs, articular cartilage defects, and many more technologies we developed. And we got a RMAT designation, and it's a we changed the design, put it in a best place for success. And so we're not doing justice to the patients. There's so much of unmet medical need in orthopedic space.

Regenerative medicine is just about to start. So we felt it's better we spin it off, and so they have a focused team really taking it to the patients. From our balance sheet perspective, overnight, I don't think market really is not deeply looking at it. We created it's public information, more than a 100,000,000 value for Neocort and our orthopedic regenerative cell therapy assets. We priced it.

Our combined company will be valued at 150,000,000 with a reverse merger with Charisma. That means they got a 15,000,000 valuation. In parallel to that, we're raising money. So the Ocugen with investors, they'll hold majority of the stake. Ocugen will be a significant component of that.

And so that means we'll continue to support them as they need. And as they move forward with their phase three clinical trial, their market comparables are there. There is one regenerative cell therapy company. They're almost trading at eight to 10 multiples of their revenues. So it's just a matter of time.

What it means, oxygen has significant stake, and that means our shareholder value will grow. It's like think like an investment in an asset. If that asset value grows, you know, that'll add it to our balance sheet. We can anytime monetize that too. So that are all going to help with ArQagen to put more resources into our gene therapies.

So we hit those three BLAs in next three years.

Unidentified speaker: So as a last question, what's what's your what's the strength of your balance sheet at this point in terms of cash, and what sort of a runway could you get?

Shankar Moussunari, CEO, Ocugen: Yep. I mean, Ocugen, we're very efficiently run if you compare to lot of biotechs out there in our space. So we have about 12,000,000 a quarter or 50,000,000. That's our runway with three programs. So based on that, recently, we got a $20,000,000 injection into our balance sheet from a very large firm.

And and so they also have a very creative way of potentially another 30,000,000 option when the price hits certain target. And so with the 30, we should extend our runway into 2027.

Unidentified speaker: Okay. Perfect. Thank you. Thanks for your time, and good luck.

Shankar Moussunari, CEO, Ocugen: Thank you for having us. Yes, sir.

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