Ocugen, Inc.

For OCU410 and geographic atrophy, we continue to advance toward Phase 3 development, following encouraging Phase 2 data, with the program expected to move through Phase 3 enrollment toward BLA submission by 2028. Taken together, these milestones reflect a disciplined, multi-program strategy designed to create value through a series of increasingly important clinical and regulatory readouts. I'll now turn the call over to Rita to provide an update on our financial results for the quarter ended March 31, 2026. Rita?

Thank you, Shankar. Good morning, everyone. Total operating expenses for the three months ended March 31, 2026 were $19.4 million and included research and development expenses of $11.3 million and general and administrative expenses of $8.1 million. compared to total operating expenses for the three months ended March 31, 2025 of $16 million that included research and development expenses of $9.5 million and general and administrative expenses of $6.5 million. Ocugen reported a $0.06 net loss per common share for the three months ended March 31, 2026 compared to a $0.05 net loss per common share for the three months ended March 31, 2025. The company's cash, cash equivalents, and restricted cash totaled $32.2 million as of March 31, 2026, compared to $18.9 million as of March 31, 2025. The company received $37.5 million in gross proceeds inclusive of $15 million due to exercise warrants in the first quarter of 2026. With the recent offering, the company is expected to have cash, cash equivalents, and restricted cash of $112.1 million at closing, which includes the Avenue debt payoff and expects to extend cash runway into 2028. The company had 338.3 million shares of common stock outstanding as of March 31st, 2026. That concludes my financial update. Shankar, back to you.

Thank you, Rita. The first quarter of 2026 was a quarter defined by execution. We delivered positive 12-month phase 2 data for ARCA 410 NGA, completed enrollment and dosing in the Guardian 3 trial well ahead of schedule, and continued advancing ARCA 400 towards its rolling BLA submission later this year. Looking ahead, the remainder of 2026 is poised to be consequential with multiple meaningful inflection points. We expect interim outcome analysis from Guardian III in the third quarter, regulatory alignment with FDA and EMA on the OCU-410 phase III design in the third quarter, and the initiation of our first BLA submission for OCU-400 also in the third quarter. Each of these milestones bring us a step closer to delivering on our commitment of three BLAs by 2028. I want to thank our employees, investigators, and patients who have trusted us with their participation and our shareholders for continued belief in our mission to advance Cures for Blindness. We'll now open the call for questions.

Operator?

At this time, I would like to remind everyone in order to ask a question, please press star then the number one on your telephone keypad. We will pause for just a moment to compile the Q&A roster. Your first question comes from the line of Leland Gerschel from Oppenheimer. Please go ahead.

Hey, AccuGent team. This is Jason on for Leland. It seems like it's gearing up to be an exciting year for Accu 400 and the Retina 8, this pigmentosa. And with that, could you give us a sense of what's being submitted for the rolling BLA versus the full BLA next year? And How else are you thinking about the pre-commercialization activities going into 2027 launch? Thank you.

Yeah, yeah. So the rolling submission, as we stated, will start with non-clinical section module. And also, we're completing our PPQ lots from CMC manufacturing perspective. That module will also be submitted this year. And next year, as soon as the top line results come in, within weeks after that, the final clinical module will be submitted. And that will trigger a P2P data of six months' clock, expected to get the approval in the fourth quarter and launch. From commercial perspective, there are multiple things we are doing. As we mentioned before, we're working on one thing, on the pricing, the government, CMS. And second step is, of course, as a company, we have established very standard way of treating these patients with our surgery procedure, which we use in clinical trials to minimize any risk to the patient. So we want to really identify the centers for excellence where our gene therapy can be administered. So that's what we're working on with the second step. And the third step is, of course, getting up for sales and marketing and really commercial plans. I think we're working on the strategy. And typically, you start that work a year before. So later part of the year, getting into the early next year, that's when we'll start bulk of the commercial work.

Sounds great. Thank you again. Thank you.

Your next question comes from the line of Michael Arconovich from Maxim Group. Please go ahead. Thank you for taking my questions today. Good morning, everyone, and congrats on all the great progress you've been making. Thank you. I guess just to start off, I'd like to see, given how quickly the Stargardt program has been moving, how close together are you expecting an RP and a Stargardt approval could occur? And then are there any additional commercial considerations you're evaluating with the possibility that you will be kind of ramping both of these launches contemporaneously?

No, good question. I mean, if everything goes according to the plan, they could be within six months of each other. Obviously, from launch or commercial perspective, again, we're targeting the same Centers for Excellence for Gene Therapy Administration. The same surgeons are going to administer for administering for RP. So, In fact, actually, it helps us as a company, economies of scale, and how we are setting it up for RP, and that will always help with Stargardt disease. So when Stargardt is going to come later, within six months, we should be in a great shape from a commercial perspective and launch perspective.

All right. And then how much overlap is there between the – the Limelight and the Guardian studies? Are they largely at the same centers, or if you take them combined, do they reach a broader swath of the overall market? Go ahead, Hema.

Good morning. This is Hema Kumar. I'm going to take this question.

Good question. So both represent inherited retinal diseases, of course, arachnidase, pigmentosa. You know, we have almost the same centers. There is a single subretinal injection. There is an overlap. However, there is no approved product for Stargard, and we do have a huge unmet medical need in terms of RPA as well. So the centers are almost the same, and also, as Shankar has mentioned, centers for excellence. There is an increased demand for, as we have now closed the enrollment, for both the programs to go as fast as we could per the protocol. And there will be some overlap, but there are quite a few more centers that we have identified as well.

So the current clinical centers, Michael, just to answer, close it out, are not good enough for commercial. They're good. but we need a lot more for commercialization.

Of course. Thank you very much. I appreciate the additional clarity. And once again, congrats on all the progress.

Thank you.

Your next question comes from the line of Robert Lebuyer from Noble Capital Markets. Please go ahead.

Good morning and congratulations on all of the progress that you've been making. My question has to do with the marketing and your mentions of centers for excellence. My understanding was that the product is something that can be administered by any ophthalmologist and anyone who treats patients and is very easy to fit into the current practice. So I was wondering about the centers of excellence and how you're going to launch the product if it's going to be a broad launch or focused on specific areas or treatment centers in the marketplace?

Yeah, I mean, good question. I mean, obviously, this vitrectomy, it's not a big surgery. I think any of our 2,500 trained, well-trained retinal surgeons in our country can do that. However, You know, it's a one-and-done treatment. It's a one-time administration for life. So we want to make sure whatever center they're going to participate in the commercial and that they get trained on the same surgical manual we've been using in the clinical trials, which has been worked successfully. So that's the goal. I mean, once again, I just want to clarify this is not a, you know, complex surgery. And any of those surgeons, the 2,500 smart retinal surgeons can do this. But as a company, we just want to make sure we train a group of people. I mean, whatever centers we need for commercialization, it could be 100, it could be 200. We want to make sure those centers are very well trained and so they follow the same procedure across the board for consistency and patient safety.

Okay. And, you know, just my understanding was that anyone who's a retina surgeon can administer... without any special training. Is that correct or just what is the difference between the clinical trial and actual practice?

I can answer that, Robert. Good to hear from you.

Yes, it's a vitrectomy, which is pretty common part of standard of care. This is not a new treatment or anything that they have not done. What Shankar is mentioning here is actually every product has its specifications. So when we are launching the centers of excellence, that would be the main centers that could further train down the next few centers like down there. So the initial launch, definitely we have all across. They're covering majority of the centers. And this is not a new treatment, new procedure, or single subretinal injection that we are giving. However, when we define centers of excellence, that's always like the initial ones that take the burden off, like for the first launch and then go towards the next. So yes, you do not need any special training. This is pretty much standard of care. And we have vitro retinal surgeons that are pretty good in doing this. The only difference is this. There is, of course, with any product, you come up with the guidelines, and that's what they have to do.

And, Robert, I think this is the first time, remember, we're not talking about going after 500 patients or 1,000 patients. We're going to go after hundreds of thousands of patients with our therapies. So there is a whole payer system in how you direct the patients. It's a new paradigm oxygen has to establish. It's nothing like anybody has done before. I just wanted to make it very clear in gene therapy space.

Yes, okay, that's a very good point. Okay, thank you. Your next question comes from the line of Whitney Eja from Canaccord Genuity. Please go ahead.

Hey guys, congrats on all the progress this quarter. First question, I guess, headed into the interim for the Stargardt study in the third quarter. Can you remind us, first of all, the powering of that study, like what was assumed, and then what should we all be expecting to see in that readout? And then, sorry, third part of this question is just what is the range of outcomes based on that data for the study moving forward?

Yeah, I think, Whitney, so this is outcome analysis.

It's not really giving an interim analysis like you expect in a primary endpoint and secondary endpoint analysis. So this is done under strict guidance of data monitoring committee. So why do you do adaptive design to minimize any further risk to phase 3 clinical trial? In phase 3 clinical trial, we have a true control arm, untreated arm, which will be compared with the treatment arm. And so the DMC looks at predictive analytics and see that study is designed for 12 months. And at eight months, when 50% of the patients are completed, they're going to look at it compared to control arm. Are we going to meet the success? And if there is nothing to be changed, you continue. That's outcome number one. No changes. Top line results come out in second quarter of next year. BLA will be filed a few weeks after that. And then the clock starts for a pre-do for date. The outcome number two, I mean, we did recruit additional patients in the trial. I mean, obviously, anticipation of dropouts and all that. But if the analytics show you need to increase number by certain number, we have to, again, recruit. I mean, that may have, you know, that means you have to monitor those patients for additional one year. So that has impact on the top line results in the filing. And during that time frame, we're also, based on the discussions we had in the agency in the past, we have two options. Either you increase the size And we can also look into adding an additional time point. From 12 months, you also can add 16 months time point. You can also look into predictive analytics and see if you're going to meet the criteria of making a success if you extend it to 16 months. So those are the two options we have. Obviously, the DMC with the blinded staff oxygen, they're going to look at it carefully and present this option to agency and get the buy-in from FDA, which they have recommended these two options in the past. And then once the outcome is finalized with the agency's concurrence, we're going to let the markets know. So just to sum it up, one of the outcomes is there's no change. We're on track. Everything is looking good. Another outcome is, yeah, there is some delay, but the delay also minimizes any risk to the clinical trial we're ingesting. So there's a delay of, you know, six months. So those are the outcomes we'll discuss with the market.

Got it. Okay, that's really helpful. And then going back to the powering question, have you disclosed the powering of the study on the primary endpoints?

Go ahead.

Yes. Yes. So basically, we have for the stronger disease, 51 subjects, 34 treatment, 17 control and adaptive design would be for 24, 16 treatment, eight control. And yes, it's adequately powered around 90 percent or more. And we are going to look at the powering once again, once the adaptive analysis interim outcome is going to be there. However, we are sufficiently powered, keeping in mind the prevalence as well as the no approved product right now in market. So the interim outcome would be either sample size re-estimation or no.

Got it. Okay. That's helpful. And then... Just to double check, in terms of the range of outcomes, there's no, I guess, upside scenario, right? Just to make sure we're fully thinking through all the scenarios. Best case scenario is things are on track. There's not like a best case, oh, we're going to end early or something like that, right?

Yeah, that's right.

Yeah, I think, I mean, unless, yeah, I mean, Whitney, if you look at many products which got approvals under, you know, some of them got approvals with 30 patients, right? So with orphan diseases, I mean, obviously, as Huma stated, it's 50% reaching eight months. Eight months, I think, we have to also be practical. I mean, if it's one year, it's a different story. For gene therapies, for us, modified genes to start working, you know, minimum you need like six months to show something. And at one year time frame, you really reach the effect size and everything else. So, obviously, again, the data will tell and we'll wait. For orphan diseases, you're right. If there's a significant unmet medical need, even sometimes at the interim, if you really hit it out of the park, agencies will consider that. But I also want to be practical about it. I think eight months is for adjustment, but typically it's good to look at it one year. One year itself, if you look at all our clinical trials compared to other things going on in the industry across all our three programs, we are doing one-year trials compared to anybody out there. Most of the people do two-year trials because we're able to show effect size, treatment effect, and benefit in one year.

Yes, that makes perfect sense. Okay. And then just moving on, a cash question. What does the new guidance into 2028 assume in terms of GA Phase III spend?

Yeah, it's included within the GA. The GA Phase III spend is included in the new cash runway into Q1 or into 2028. So, yeah, we do anticipate being able to cover the expenses for that trial.

Okay, got it. And I guess could you – I know the conversations with the agency are ongoing, but what was assumed in terms of size of that study just for cash reasons or any design characteristics you can talk about at this point just for, again, from a cash – estimation perspective?

It's a 300 patients global trial in the US, EU, and Canada together. And most of the patients are majority will be in the US because we have the existing centers. And so it's two to one ratio, 200 in treatment and 100 in untreated control. It's powered at over 95% for the primary endpoint and 92% for easy ellipsoid zone secondary endpoint. And it has an adaptive design that when 150 patients reach one year, we can take a look.

Got it. Okay, perfect. That's it for me. Thanks so much.

Your next question comes from the line of Ramakant Swayampakula from HC Wainwright. Please go ahead.

Thank you. This is RK from HC Wainwright. Good morning, Shankar and team. A lot of my questions have been answered, but I have a couple of them. On the OCU 400, you know, where you're planning to start the rolling VLA in third quarter, can you confirm your PPQ runs would be completed, you know, in time, like by end of second quarter or so, so that you can initiate your rolling VLA?

Yeah, absolutely. We're on target to complete them this quarter.

Okay, perfect. And support the rolling bill submission.

Okay, great.

Rita, in terms of expenses, we have seen G&A and R&D expenses go up this quarter, which is understandable. But how should we think about this going forward, especially into 2027, as you're preparing, you know, for commercialization? And relatedly, you know, on the BD side of things, what progress has been made, especially for 410 in terms of ex-US licensing?

Yeah, thank you for your questions. So first of all, just to kind of address the, you know, the spend that you're seeing, you have to consider part of it is due to timing and then also related to, you know, us exceeding some of our programmatic milestones, right? So if you're looking at, you know, ACCU 410ST and ACCU 410GA, we accelerated those timelines. And so when you think about, you know, us completing our enrollment for ACCU 400 and ACCU 410ST, the completion of that enrollment in Q1 will now enable us to kind of ramp down the clinical spend going into the balance of the year. So we feel really confidently about, you know, the anticipated spend that we had in 2026 going into 2027. And so we're, you know, averaging around $50 to $60 million per year from a spend perspective, which is why we believe that our cash runway is into 2028. And then going to the business development, we are, you know, actively evaluating various BD deals for XUS for both OCU 400 and OCU 410ST. You know, we have term sheets that we're looking at. So, you know, there's a lot of work going on, just as Shankar said, that Avi is doing, who leads our business development team, to ensure that, you know, we are evaluating those alternatives and then making the best decision for Ocugen and for our shareholders. And then lastly, from a commercialization perspective for Accu410 GA, we are still looking to commercialize that with a partner. Although, you know, as we talked about the spend associated with the clinical trials, we, you know, we have that incorporated into our runway.

Thank you. One last question. Shankar, I know in the past we have talked a little bit about payers and how to get payers. agreed to the pricing that you would come up with. Any commentary, you know, especially from recent conversations from your payers as you're getting closer, you know, to commercialization, especially with the price tag that you are thinking for some of your drugs?

Yeah, I mean, obviously, there is a publication in Retina, I mean, that talks about potential pharmacoeconomic model justifying one to two million, somewhere in the range price tag. And also there is a publication from New England Journal of Medicine. And this is, again, we're happy to state CMS and CMMI, they're looking into, you know, what is the next iteration of sickle cell model they created. And the publication clearly goes into pay over time and subscription models, which can help with the budgetary constraints we have. How can we work? So everything is very creative, RK, as I mentioned before, from launch, you know, and the treatment centers too, and how the CMMI is looking. I'm very pleased to say, obviously, these are one-time treatments, right? So we need to think about some creative ways. How can we work with payers and make sure, especially if CMS, the government is spending a lot of money for orphan diseases or diseases like GA, which will have most of the patients are above 60, And you're going to get a bigger chunk of economy and budget from CMS. So we need to really think into all those options, how we can provide market access to more patients who need them. So that NJM article coming out of CMS is a good one. And it goes into, okay, if the price tag is high, you pay your time. Because if you say your therapy is one-time treatment for life, you stand by it. And so those are very good models coming out. I mean, it's a very creative thinking, and we are aligned with that strategy.

Perfect. Thank you. Thanks for taking all my questions.

Your next question comes from the line of Daniel Gataulin from Chargent. Please go ahead.

Yes. Hi. Good morning, guys. Thank you for taking my question. I have a more general question on easy preservation. It appears to be emerging as an important endpoint. So I wanted to ask in your conversations with the regulators, what would you say their most recent position is on the importance of easy preservation? And two, are there any differences in how U.S. and EU regulators are thinking about easy preservation? Thank you.

Daniel, good question. We just submitted our meeting request to both FDA and EMA. Obviously, I will let you know by early third quarter the input and alignment with them. I mean, obviously, you saw GA trial, what we publicly stated. Our primary endpoint is lesion because that's an approved endpoint in two commercial products in the U.S. And the secondary endpoint is we are proposing is ellipsoid zone. because it correlates to visual function. We believe that should satisfy them, you regulators. And obviously, we're going to wait until we complete all the meetings, everything buttoned up and aligned, then we let the markets know.

Thank you. That will conclude our question and answer session.

Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.