Ocugen, Inc.

Good morning and welcome to Occugen's second quarter 2036 financial results and business update. All participants are in listen-only mode. Following the speaker's commentary, there will be a question-and-answer session. I will now turn the call over to Chris Clark, Occugen's head of communications. You may begin.

Thank you, operator, and good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri. Occugen, Chairman, CEO, and Co-Founder, who will provide a business update and an overview of our clinical and operational progress. Rita Johnson-Greene, our Chief Financial Officer, is also on the call to provide a financial update for the quarter ended June 30th, 2026. Abhi Gupta, Executive Vice President of Commercial and Business Development, and Dr. Mohamed Genead, who joined Occugen as Chief Medical Officer in June, will be available to answer questions following the presentation. This morning we issued a press release covering our business and operational highlights for the second quarter of 2026. We encourage listeners to review the press release, which is available on our website at ocugen.com. A replay of this call, along with the accompanying slide presentation, will be available on the investors section of the Ocugen website. Please note that certain statements made during today's discussion may be forward-looking in nature. including those related to our clinical development pipeline, regulatory timelines, commercialization strategy, and financial information, and our anticipated cash runway. These statements reflect management's current expectations and are inherently subject to risk, uncertainties, and assumptions that may cause actual results to differ materially from those expressed or implied. We encourage you to review our filings with the Securities and Exchange Commission including the risk factors detailed therein for a more comprehensive understanding of these potential risks. Finally, OxyGEN's quarterly report on Form 10-Q covering the second quarter of 2026 will be filed today. I will now turn the call over to Dr. Musunuri.

Thank you, Chris, and good morning, everyone. The second quarter was a defining one for OxyGEN. The FDA cleared our phase three trial for Oxy410 To initiate dosing in geographic atrophy patients and granted RMAP designation for the program, we signed a binding term sheet with Roots Pharmaceutical to negotiate an exclusive license for Acu400 in retinitis pigmentosa across the Middle East and North Africa, MENA region. And from the closing of 130 million convertible notes financing, we extended our cash runway into 2028, now able to support all three of our late-stage programs. Before I walk through the quarter, I want to step back because Ocugen's potential is worth putting into context. For more than a decade, gene therapy in ophthalmology has been confined to a single gene, a single mutation, and a single small patient population. Our modified gene therapy platform takes a fundamentally different approach. Rather than targeting individual mutations, it is designed to address the root cause of complex retinal diseases by modulating master regulators, nuclear hormone receptors that govern multiple gene networks. The platform is gene agnostic, inherently multifactorial, and designed to deliver a durable benefit from a single, one-time subretinal injection. What this means, in practice, is that Opigen is not building three separate drugs. We're advancing one platform across three late-stage programs, each targeting a major cause of blindness for which patients today have either no approved treatment whatsoever or therapies that demand chronic injections and carry meaningful safety burdens. Retinitis pigmentosa, or RP, Stargardt disease, angiogastrophy, or GA, together affect approximately 3 million people across the United States and Europe, a combined patient population, and a commercial opportunity for larger than anything currently served by approved gene therapies and ophthalmology. Across our pipeline, spanning phase one through phase three, we have treated more than 325 patients, including EAP, through multiple doses and indications, and we have not observed a drug-related serious adverse event. We remain on track to file three BLH by 2028. This positions the first half of 2027 as a catalyst-rich window for ArcGEN with the top-line data for ARCA 400 and ARCA 410ST and our planned BLA submissions following over a short period. Let me walk you through how each program is advancing. Then I will hand over the call to Rita for financials. Starting with ARCA 410 for GA, a secondary to dry age-related macular degeneration, or dry AMD, GA represents our largest commercial opportunity. with approximately 2 to 3 million patients in the U.S. and Europe combined. There are currently no approved treatments for GA in Europe. Current approved therapies in the U.S. target only one complement pathway and require frequent intravitreal injections, which has been associated with treatment discontinuation in clinical practice. GA is a multifactorial disease driven by four distinct pathways that contribute to the progressive degeneration of the macula. and Drusen, inflammation, oxidative stress, and complement or activation. The currently approved therapies in the U.S. address only one of these four pathways, the complement system, which is partly why they have been unable to demonstrate meaningful functional outcomes for patients. Ocuportin operates differently. By delivering RORA, a nuclear hormone receptor that acts as a master regulator of retinal homeostasis. Ocu410 is designed to address all four disease pathways simultaneously with a single subretinal injection, has the potential to redefine the standard of care in this indication. We recently received FDA clearance for Ocu410 phase three registrational trial for GA. The trial, Armada 3, is planned to be a global study of approximately 237 subjects. using an adaptive design powered at 95% for the primary endpoint with the BLA and market authorization application filings targeted for 2028. We plan to initiate phase three by September 2026. This design is anchored by positive 12-month data from our phase two armada trial. At the optimal dose, OQ410 delivered a statistically significant 31% reduction in GA lesion growth within the patient population of lesion size 2.5 millimeter square and 17.5 millimeter square. The criteria to be used in our phase three portal trial versus control, approximately twice the benefit of approved complement inhibitors and from a single injection. We also saw a 27% preservation of the ellipsoid zone within the same patient population and no drug-related serious adverse events reported to date. Importantly, these phase two data help support the FDA's decision to grant ORMAD designation for OQ410. Turning to OQ410-ST for Stargardt disease, Stargardt is a pediatric onset retinal disorder affecting approximately 100,000 patients in the U.S. and Europe and roughly one million people globally. There are no approved therapies available for these patients today. RQ410-ST is designed to address over 1,200 pathogenic mutations in the ABCA4 gene with a single onetime treatment. On April 1st, we announced the completion of enrollment and dosing in our Phase 2-3 Guardian 3 Total Confirmatory Trial enrolling 63 participants. We expect the interim outcome decision for the first 50% of subjects at eight months in the third quarter of 2026, and top-line Phase 2-3 data in the second quarter of 2027, with our BLA submission to follow mid-2027. Moving to Octave 400 for RP, the Phase 3 Limelight Trial is the first and largest genetic medicine registration trial for broad RP, spanning more than 30 genetic mutations. Approximately 300,000 people in the US and Europe are living with RP, which is caused by mutations in more than 100 genes. The only approved gene therapy for RP today targets a single gene, RPE65, which accounts for less than 2% of all RP cases. RQ400 is designed to provide a therapeutic option for all RP patients, and that is a fundamentally different commercial opportunity. Enrollment in the limelight is complete. with 140 patients randomized two to one treated versus control across the row and gene agnostic comps spanning more than 30 genetic mutations associated with early to late stage RP, including pediatrics. The breadth of the population intended to validate the gene agnostic mechanism of action of our novel modified gene therapy platform. The primary endpoint is 12 months change in visual function assessed by Luminance Dependent Navigation Assessment, or LDNA. Subjects are followed for one year, post-dosing, for the primary endpoint analysis. Top-line Phase III data is expected in the first quarter of 2027, advancing ARCHIV 400 to a potential approval in the fourth quarter of 2027. FDA feedback confirmed that the path to ruling-based submission remains tied to top-line data expected in the first quarter of 2027. On the manufacturing side, our process performance qualification, PPQs, badges are complete, supporting BLA and commercial launch supplies. Brand planning and marketing initiatives led by Abhi Gupta, our EVP of commercial and business development, continue to scale in preparation for launch. We also advanced our global commercialization strategy for Accu 400 during the quarter. In July, We signed a binding term sheet with Root Pharmaceutical and its strategic partner, Eldao International Holdings, for exclusive rights to OQ400 in the Middle East and North Africa. We're active on the BD front to find other global partners for regional commercialization partnerships where RPE is most prevalent. Here is a snapshot of the market opportunity across our three late-stage development programs. While Occu410 for GA represents our largest commercial opportunity, we believe all three programs have the potential to generate significant revenue while addressing areas of substantial unmet medical need. As we continue advancing our pipeline, we're also building the foundational commercial capabilities to support future global access. Our efforts are focused on five key areas. were in discussions with CMS and payers to establish early market access and reimbursement strategies. Second, we continue to identify and evaluate specialized centers of excellence with expertise in subretinal surgical procedures that could support future treatment delivery. Third, we are mapping the patient journey from diagnosis through treatment and long-term follow-up with the goal of facilitating a seamless experience for patients, caregivers, and healthcare providers. Fourth, we are assessing manufacturing, supply chain and distribution requirements to help ensure operational readiness. Finally, we are beginning to build out our commercial infrastructure, including our marketing and sales capabilities as we ramp up for launch. With that, I'll turn the call over to Rita for the financial update. Rita.

Thank you, Shankar. Good morning, everyone. Total operating expenses for the three months ended June 30, 2026 for $17.9 million and included research and development expenses of $10.7 million and general and administrative expenses of $7.2 million. This compares to total operating expenses for the three months ended June 30, 2025 of $15.2 million, which included research and development expenses of $8.4 million and general and administrative expenses of $6.8 million. Total operating expenses for the six months ended June 30, 2026 were $37.3 million. and included research and development expenses of $21.9 million and general and administrative expenses of $15.4 million. This compares to the total operating expenses for the six months ended June 30, 2025 of $31.2 million, which included research and development expenses of $17.9 million and general and administrative expenses of $13.2 million. Occitan reported a 7 cent net loss per common share for the three months ended June 30th, 2026, compared to a 5 cent net loss per common share for the three months ended June 30th, 2025. On our capital position, following the closing of the $130 million convertible notes financing, The company's cash, cash equivalents, and restricted cash totaled $100.4 million as of June 30, 2026, extending our cash runway into 2028. The company had 339 million shares of common stock outstanding as of June 30, 2026. That concludes my financial update. Shankar, back to you.

Thank you, Rita. The second quarter was a quarter of execution. The remainder of 2026 is poised to be impactful. We expect the ARQ 410-ST interim outcome decision in the third quarter, and we expect to initiate the ARQ 410 phase three trial in this quarter. Looking to 2027, we expect top line data from both ARQ 400 and ARQ 410-ST in the first half of the year, followed by our planned BLA submissions. Each of these milestones brings us a step closer to delivering on our commitment to three BLAs by 2028, offering potentially life-altering improvement to patients coping with blindness-causing diseases. I want to thank our investigators and patients who have trusted us with their participation and our shareholders for their continued belief in our mission to advance cures for blindness. We'll now open the call for questions.

Thank you. Ladies and gentlemen, we will now begin the question and answer session. And at this time, I would like to remind everyone, in order to ask a question, please press star, followed by the number one on your telephone keypad. And if you would like to withdraw your question, simply press star one again. Again, if you would like to ask a question, press star one on your telephone keypad. Our first question comes from the line of Michael Okunwich with Maxim Group. Please go ahead.

Hey there, thank you so much for taking my questions and congrats on all the great progress. So I wanted to ask, you now have a handful of international partnerships, which makes OCU 400 a truly international program at this point. So I just wanted to see if you could share the regulatory plans in particular for ex-US jurisdictions, what's required there, and how those timelines could vary versus your BLA path.

Michael, what we have with RQ400, we got alignment from EMA in addition to FDA with the same that single trial we're doing in U.S. is good for approvals. And across the globe for orphan gene therapies, typically they get approval based on U.S. approval. So everything will be linked to our U.S. FDA approval in MENA and other regions.

All right, thank you. And then I wanted to see also if you could just highlight some of the key differences in the trial design between Armada 3 and the Phase 2 Armada trial.

Let our CMO, Dr. Genead, answer that.

Thank you, Michael. Regarding Armada 3, which is our global Phase 2 trial for GA, which we just got the approval from FDA, you know, just recently to be initiated this quarter, The phase three trial designed for Armada 3 will be one treatment arm with OQ410 versus a control will be two to one randomization allocation. And that data will follow each subject up to 12 months. And this is where we're going to be looking at the primary efficacy endpoint plus other key functional endpoint. The Armada 1, the earlier phase 1, 2 GA trials was similar. on the efficacy. So we should expect, you know, similar outcome here. We're going to look, the numbers obviously is different. We're going to be enrolling in Armada 3, close to 237 subjects in 2-1 allocation. It's going to be global. We're going to go ex-US. We're going to go to Europe and other territorial part in the world. But the primary endpoint will be very similar. So we should expect to see similar trend what we saw from Armada 1, the phase 1, 2 AJA trial.

Thank you. And then just one last one for me before I hop back into the queue. So it looks like in Stargardt, there is a chance that we'll have an approved therapy sometime around when you'll be completing your own BLA filing. So it'll be a chronic therapy versus a one-time. But I wanted to ask how important the pricing on other therapies, since we don't have any pricing comps, would be to inform your own pricing strategy and if there's any way that we can think about how to translate pricing between a chronic ongoing therapy and a one-time therapy.

Yeah, good question, Michael. I think the way you look at it is our treatments are one-and-done treatments potentially, so that will have a different pricing structure than ongoing chronic therapies. Number two, everything will be dictated by data. And if you have a safe One-time treatment, I think our gene therapies, once again, you know, we're still collecting data, as you can see in some of the patients in RP, and as they approach like second year, third year, they're improving further. So the current therapies, if the oral therapy comes to the market, what patients are going to look for is a therapy just reducing the degeneration of the disease are in some patients either stalling it, is it has potential to reverse it in some patients. At least, you know, with our modified gene therapy in some of the patients, we're seeing all those trends. So that could be a big differentiating factor. And also, as you know, Stargardt impacts a lot of pediatric patients. And the current clinical trial they're conducting focuses on 12 plus. And our clinical trial focuses on three plus. So there are a lot of differentiators. So whenever we come for pricing, because of the differentiated disruptive technology platform we have, and obviously everybody will focus on safety, efficacy, and one and done treatment, there'll be more compliant for anyone. So I think all those factors will be rolled in. So I don't think we'll be truly comparing any pricing what the other chronic therapies are doing. If you have a me too products, the answer is yes. But if you have truly differentiated disruptive technology, just completely different, it'll come, we can price it on its own merits.

All right. Thank you. I appreciate the additional callers.

Our next question comes from the line with the IHM Mechanical Genuity. Please go ahead.

Hey, guys. My congrats on all the progress as well. Just to keep going on the Stargardt discussion, Shankar, since you mentioned it, can you talk about a little bit more, I guess, around the TPP here and the potential to show kind of reversal of disease and improvement in visual acuity? Is that something that is reasonable to expect given the duration of follow-up in the ongoing Phase 2-3 study? And I guess, if so, is there anything that was done in terms of entry criteria to maybe enrich for that outcome as far as patient baseline characteristics?

I will ask Dr. Genead to talk a little bit about baseline characteristics, then I'll answer the other question. Go ahead. Thank you, Shankar.

Hi, Whitney. Yes, happy to answer. So our population was definitely broader than other competitors. Just to highlight, first, we included, you know, patients from early to late stage Stargardt disease. That's number one. And Shankar just mentioned, too, we included subjects, you know, are younger than, you know, young adults. We included, you know, subjects, you know, three plus years of age. So that's a very broad population. As you know, for Stargardt, The earlier the better, especially if it's a progressive retina degeneration disease. The lesion size we also included in our trial, the Phase 2-3 Guardian trial, was more broader than what we saw with others. And our lesion size can include smaller lesions and also larger lesions. So we have a broad spectrum, and that's also going to be aligned with our early, late stage strategy for the disease. We already included some of the subjects in our Phase 2-3 trials. So we will be excited to see the data. In addition to the gene mutation, specifically, we include all the variants and all the other specific mutations included in the ABCA4-related retinopathy. So it includes SORGET and others as well. So this is also on the disease indication overall. Based on your point about the functional, I think this is going to be critical. So we saw from our Phase 1 data, that we just published at the Eye Nature, you know, early in the year, we saw, you know, very clear, you know, structure, slowing in the structure of progression in those patients. And also we saw functional benefit in those patients. And as you remember, as you know, in Stargardt disease, The first target is to hold that progression, to stop losing more retinal structure and function, which we achieved in our prior trial. The second goal, which will be the upside here and the ultimate goal, to reverse that tide, try to improve on the disease outcome. And we saw that in our phase one, two. We saw some of the patients did improve in visual function. The gain was Six letters close to one line between the treated versus untreated eyes. So we felt also very excited about the functional gain in the patient population. So that's kind of where we think, you know, the big differentiation, the broader application of our molecule.

Whitney, just to clarify, the primary endpoint, because it's a one-year trial, it's not a two-year trial, it's still a lesion. Then there are secondary visual function we'll be monitoring. In addition to that, at the time of filing, we continue to monitor our early-stage phase one patients, and so we'll have long-term data in those patients, too.

Got it. Really helpful. And then just last question, and maybe, Rita, this one's for you. Can you help us understand how you're thinking about cash, given the exciting progress with the GA study and the ability to start that study in September, I think you said? If there is a need to kind of pull levers to extend cash runway further, how should we think about maybe the startup of GA versus commercial prep for RP or Stargardt and just kind of how you guys are thinking about those different levers if needed? Thanks.

Yeah, thank you, Whitney. So first of all, I mean, our primary goal is to make sure that we are, you know, minimizing shareholder dilution, but evaluating opportunities in order to raise capital, just as you said, in order to bring these novel products to patients. So just first of all, we have cash runway into 2028. And so I just want to remind everyone of that, which gives us the confidence to execute our, you know, our clinical state, our late stage products that we have, and then progress to BLA submission for both OCU 410 and OCU 410ST in 2027, with the potential to commercialize OCU 400 by the end of the year in 2027. We do have some additional levers that we can pull. One, you know, we have the PRV for OCU410ST given the RPD designation that we have. And so, of course, we have the ability to sell that for somewhere between, you know, 100 to 200, even prior to approval. And that's something that we are evaluating. We also have various business development deals that we are looking at, you know, from a Globalization perspective, we're looking at XUS for both OCU 400, OCU 410ST, and even GA, right, just depending upon what that term sheet looks like. So always looking for, you know, potential deals that we can make in order to, you know, again, just minimize that dilution. We also have the Janus Henderson warrants, right? There's another 10 million warrants at $1.50 strike price, which could bring in another $15 million. And those warrants expire in August of 2027. And then, of course, we anticipate a special meeting in September of this year in order to increase authorized shares, which will give us the ability to raise additional equity if we decide to do so. So, again, you know, just looking at both non-dilutive as well as dilutive options in order to make sure that we are able to bring these amazing and novel products to patients as well as looking at maximizing shareholder value.

Very helpful. Thank you.

Our next question comes from Delano Charles Wallace with H.D. Wigwright. Please go ahead.

Hi, this is Charles from HC Wainwright. I'm for RK. Thanks for taking my question. Maybe a question on the Armada 3 design. So it seems like based on the prior earnings call, the study has been a little bit resized. I think previously you said it would be about 300 patients, and now it's 237 patients. So I was just curious if this was something the FDA specifically asked for, or if this was something you proposed, and then also if the assumptions change based on effect size, variability, dropout, or the narrower lesion size compared to the Phase II.

Yeah, Dr. Vinit.

Thank you, Charles. Yes, we had a discussion with the agency, the FDA, so all this being aligned and discussed, with the FDA. But to answer your questions specifically, it was based on all the sample size estimation and also the power calculation we did. So the estimate you are citing, the 300, was based on estimate. But when we saw the effect size based on our Armada 1, the Phase 1-2 trial, as we discussed today, we saw the 31% reduction in the medium dose, the optimal dose, which is the one we are taking forward. When we did our calculation based on that, We saw, you know, the 237 total population to be enrolled will give us 95% power in our pivotal trial. All these pieces have been discussed with the agency. Obviously, it's based on the rate of change, the slope analysis for the primary efficacy. So the effect size, you know, based on what we saw from earlier trial was very positive and was strong enough that we end up, you know, with 237. Two to one randomization, as we mentioned earlier, 158 in the treatment arm and 79 in the control arm. So all this has been discussed and aligned, and as we announced today, we got the clearance from the FDA to initiate our phase three trial in the next few weeks.

Thank you. Very helpful. And then I guess for the rolling submission, so I think Originally, the guidance was to submit in the third quarter, and now I believe it's the first quarter after the limelight data. I guess my question is, what kind of changed between submitting the non-clinical module earlier compared to after the top-line data, the limelight?

Charles, I think from our perspective already, I think we're doing very well with our PPQs, as we've mentioned. A lot of gene therapy companies stuck with CMC. We're ahead of the game. We used to commercial scale lots in phase three. We completed our PPQs on time. We got non-clinical at PPQ or done so. We have CMC non-clinical ready to go. I mean, obviously, this is where we have to work with the agency when they're comfortable. And that's the timeline they gave us. And we're going to be fine with that. The reason is, I just want to clarify, it's good to have Rolling Submission that gives a head start for agency, okay? It's for their own benefit. And if they want to wait until next year, I mean, we are ready to file it as soon as the top line comes for the pre-BLA meeting. We may still give them a head start of maybe a month or two months before we drop the clinical section. So, however, I just want to clarify, until the final BLA is completed with the clinical section, the PDUFA date, the accelerated clock of six months doesn't start. I just want to clarify that. So once again, this is a collaboration between the sponsor and the agency. In this case, of course, we respect their decision, whatever they are, because they have a lot of programs, a lot of workload. Whatever the reasons are, we are fine with it. I think we're ready from our perspective, and we'll work with them closely in a collaborative way. And whenever we have a top line, we'll be ready to fight it. So it doesn't change any, yeah, filing clock, as we mentioned before, second quarter, complete the BLA filing, anticipated approval in fourth quarter, six months accelerated clock. Very helpful. Thanks for taking both questions.

Once again, if you would like to ask a question, please press star followed by the number one on your telephone keypad. Our next question comes from the line of Robert Leboyer with Double Capital Markets. Please go ahead.

Good morning and congratulations on the progress. Just to follow up on that last question, my understanding was that the BLA submission will be completed in early 2027 when the clinical module is filed. That's when you get the PDUFA date and the approval launches based on that. But you also have rolling submission and have the option of filing the CMC and the other non-clinical modules before that. Is that still your plan?

Yes. Yes, Robert, absolutely. Because based on agency's suggestion and recommendation, as soon as the top line comes out, we'll have a pre-BLA meeting. Right after that, we can file the two modules, non-clinical and CMC modules. So that will still give them a head start. Then as soon as the clinical module is done, when you file it, the period of update starts. So that's basically our plan is to file that in second quarter. So six months' clock should be fourth quarter, approval clock.

OK, terrific. Thank you for that.

And at this time, we have no further questions. I would like to turn the call back over to the oxygen team for closing remarks.

Thank you all for attending today's webcast. Really appreciate. All our investors, shareholders, patients, providers, thank you.

This concludes today's conference call. You may now disconnect.