OCGN - NASDAQ

Good morning, and welcome to Ocugen's Third Quarter 2025 Financial Results and Business Update. Please note that this call is being recorded at this time. [Operator Instructions] I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin.

Thank you, operator. And good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri, Ocugen's Chairman, CEO and Co-Founder, who will provide a business update and an overview of our clinical and operational progress. Ramesh Ramachandran, our Chief Accounting Officer, is also on the call to provide a financial update for the quarter ended September 30, 2025. Dr. Huma Qamar, Chief Medical Officer, will be available to answer questions following the presentation. This morning, we issued a press release detailing associated business and operational highlights for the third quarter of 2025. We encourage listeners to review the press release, which is available on our website at ocugen.com. This call is being recorded and a replay with the accompanying slide presentation will be available on the Investors section of the Ocugen website for approximately 45 days. This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements include, but are not limited to, statements regarding our clinical development activities and related anticipated timelines. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission, the SEC, including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this presentation speaks only as of the date of this presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events or otherwise after the date of this presentation. Finally, Ocugen's quarterly report on Form 10-Q covering the third quarter of 2025 will be filed today. I will now turn the call over to Dr. Musunuri.

Thank you, Tiffany. Thank you all for joining us today. I'm pleased to share an update on our modifier gene therapy platform and would like to recognize that in just over 3 years, we brought our lead candidate, OCU400, from initial Phase 1/2 dosing to nearing Phase 3 enrollment completion. The OCU410ST Phase 2/3 pivotal confirmatory trial is following close behind, and we are on track to complete enrollment in the first quarter of 2026, lining up for our planned biological licensing application, BLA submission, in the first half of 2027 for OCU410ST. This rapid progress is somewhat unheard of in the industry and not only reinforces our commitment to file 3 BLAs in the next 3 years, but it also brings us closer to addressing the incredible unmet medical needs that exist for patients facing vision loss. While all 3 programs are moving along on schedule, we received additional, positive news in the third quarter that the Committee for Medicinal Products for Human Use, CHMP, of the European Medicines Agency confirmed the acceptability of a single U.S.-based trial for submission of MAA in Europe for OCU410ST. This alignment allows us to maintain the same timeline and budget efficiencies in Europe as we have with the OCU400 pivotal trials. To fund clinical trial progress, we continue to pursue opportunities to increase our working capital and in August, closed the registered direct offering with Janus Henderson. The gross proceeds were approximately $20 million, which we anticipate will extend our runway through the second quarter of 2026, and we will receive $30 million of additional gross proceeds if the warrants are exercised in full, extending our runway into '27. The OCU400 Phase 3 liMeLiGhT clinical trial remains on track for BLA and MAA submissions in 2026. It is the only broad retinitis pigmentosa RP gene-agnostic trial to address multiple genetic mutations with a single therapeutic approach. And it's important to note that this is the largest known Phase 3 orphan, gene therapy trial. There are approximately 300,000 people in the U.S. and Europe combined living with RP, which affects more than 100 genes. Ocugen's gene-agnostic approach has the potential to treat multiple gene mutations associated with RP with a single, one-time, subretinal injection. Currently, the only approved gene therapy for RP targets a single gene RPE65, which accounts for 1% to 2% of RP patient population. This product achieved peak sales of $52 million in 2023 with a patient population of approximately 2,000. We believe OCU400 has far greater commercial potential as it is intended to provide a therapeutic option for the remaining 98% to 99% of RP patients. We anticipate commercialization in 2027. Process validation and manufacturing activities are progressing well in support of the BLA. Brand planning and marketing initiatives led by Abhi Gupta, our EVP of Commercial and Business Development, are scaling up as well. We will begin rolling submission of the OCU400 BLA in the first half of 2026 and release Phase 3 top line data in the fourth quarter of 2026, in line with our commitments. As we prepare for what will ultimately be a global rollout for OCU400, we're pursuing regional partnerships that preserve Ocugen's rights to larger geographies to maximize total patient reach while also generating return for our shareholders. In September, we announced an exclusive licensing agreement with Kwangdong Pharmaceutical Company Limited for the rights to OCU400 in South Korea. Under the agreement, the company will receive up to $7.5 million in upfront and development milestone payments plus sales milestones of $1.5 million for every $15 million of sales in South Korea, projected to reach $180 million or more in the first 10 years of commercialization. We will also earn a 25% royalty on net sales generated by Kwangdong and will be responsible for manufacturing and supplying OCU400. There are an estimated 7,000 individuals in the Republic of Korea with RP, which represents approximately 7% of the U.S. market. OCU400 provides the opportunity for our partner to help thousands of patients facing vision loss. Upon regulatory approval of OCU400 in Korea, we believe Kwangdong will become a leader in the field of ophthalmic gene therapy in South Korea. Now let's move on to OCU410ST. OCU410ST has the potential to target over 1,200 pathogen mutations in the ABCA4 gene associated with Stargardt disease and other ABCA4-related retinopathies with a single, onetime, subretinal injection. As I mentioned earlier, enrollment in the Phase 2/3 GARDian3 clinical trial is ahead of schedule. The strong response underscores the significant, unmet medical need among Stargardt patients who currently have no approved treatment options available. Stargardt disease affects approximately 100,000 people in the U.S. and Europe combined and approximately one million people globally. With CHMP acceptance of U.S. trial data for the MAA submission, we'll maximize resources and streamline development efforts with the goal of bringing OCU410ST to patients in Europe sooner than originally anticipated. The 12-month data from all available Phase 1 subjects showed highly encouraging results with a 48.2% reduction in lesion growth and a meaningful online 6-letter gain in visual acuity in evaluable treated eyes compared with untreated eyes. All treated eyes also demonstrated stabilization or improvement in visual function, highlighting a consistent and tangible therapeutic benefit. Interim data from ongoing Phase 2/3 study is expected mid-2026, further advancing our goal of bringing OCU410ST to patients in need. Finally, OCU410 is specifically designed to address multiple pathways implicated in the pathogenesis of dry age-related macular degeneration and offers a promising advantage for current treatment options that target only one pathway, the complement system, which does not fully address the disease progression and underlying causes of vision loss. Currently approved treatment options require frequent intravitreal injections about 6 to 12 doses per year and are accompanied by various safety risks. For example, roughly 12% of patients develop wet AMD following treatment. With approximately 2 million to 3 million geographic atrophy patients in the U.S. and Europe combined, OCU410 represents a significant market opportunity. Current therapies have notable limitations, and there are no treatments approved for GA in Europe, as existing FDA-approved options failed to demonstrate meaningful functional outcomes. OCU410 is therefore well positioned to address this critical, unmet, medical need. At 12 months, available subjects in the Phase 1 study showed a 23% reduction in lesion growth, along with a 2-line or 10-letter stabilization or gain with visual equity in treated eyes. Preliminary results from 6-month interim analysis demonstrated a 27% reduction in lesion growth and preservation of retinal tissue in the treated eyes when compared to untreated control eyes. This reduction is over twice that observed with currently approved, intravitreal therapies at 6 months, monthly and every other month PEG citicoline injections, which showed only 13% and 12% reductions, respectively, highlighting OCU410's potential to provide a significant and meaningful therapeutic benefit to patients with a onetime treatment. In addition to the greater lesion reduction, a single subretinal injection of OCU410 demonstrates greater efficacy in preserving retinal tissue surrounding GA lesions compared with monthly and every other month PEG citicoline treatments. We plan to provide full 12-month data from the Phase 2 study, including both structural and functional outcomes, in the first quarter of 2026 and anticipate initiating the Phase 3 study next year. I will now turn the call over to Ramesh Ramachandran to provide an update on our financial results for the quarter ended September 30, 2025.

Thank you, Shankar. The company's cash, cash equivalents and restricted cash totaled $32.9 million as of September 30, 2025, compared to $58.8 million as of December 31, 2024. With the recent $20 million financing in the third quarter, we believe our current cash position provides sufficient runway to operate through the second quarter of 2026. Total operating expenses for the 3 months ended September 30, 2025, were $19.4 million and included research and development expenses of $11.2 million; and general and administrative expenses of $8.2 million. This compares to total operating expenses for the 3 months ended September 30, 2024, of $14.4 million that included research and development expenses of $8.1 million; and general and administrative expenses of $6.3 million. That concludes my update for the quarter, Tiffany, back to you.

Thank you, Ramesh. We will now open the call for questions. Operator?

Your first question comes from the line of Michael Okunewitch with Maxim Group.

Congratulations on all the progress. So, I guess to just start out, I'd like to ask a little bit about OCU400 and in particular the timing of the BLA completion. You mentioned that you are going to be starting that in the first half of 2026, enrolling BLA. But then how quickly do you believe that you can turn around the pivotal data over to the FDA and complete that filing?

As soon as it in, we're nearing completion as we stated. And we'll have resources ready to turn around in weeks.

All right. That's great to hear. And then in terms of your manufacturing, are there any other items that you need to do to get ready for commercial manufacturing before you can start submitting that and start the rolling BLA process?

Yes, good question. Our PPQ, our process validations runs, are going very well. They're on target. In fact, all the material we're making in support of the registration, they can be commercialized. So, we will have lots made ready to go.

And then just one more for me, and I'll hop back into the queue. For OCU410ST, with the upcoming interim readout, what endpoints are you expecting to release? Or is that going to be an internal DSMB review?

I mean for endpoint for the Phase 2, yes, it's already in the clinical protocol. We are looking at lesion growth compared to untreated control group. We do have untreated control group. And the secondary, we're looking at visual equity.

But I'm specifically referring to the interim release that you're expecting midyear 2026. Will that be publicly released or is that going to be internal?

That's a good point. Yes, there will be limited information publicly. The DMC looks at it, and then we'll be informing the agency. However, we will give some indications about the clinical trial.

I appreciate the update. It looks like there's a lot to look forward to Ocugen.

Your next question comes from the line of Boris Peaker with Titan Partners.

A couple of questions for me. Maybe let's start with the OCU400. Can you just remind us exactly what the statistical kind of design of the liMeLiGhT study is, what the assumptions were? And I'm just curious if you've had kind of recent discussion with the FDA to make sure that it's still acceptable. And I think this question comes in the context of what we've seen just recently with uniQure, where it sounds like the FDA may have moved the goalpost a little bit in their gene therapy. So, I think I just want to get a sense to make sure that similar dynamic doesn't happen here.

Yes. Good question. I'll start, then Huma will chime in. So first and foremost, I want to distinguish all our clinical trials, including our Phase 2 GA trials, we have a control arm within the study. Typically, FDA -- that has been a tradition. Most of the clinical trials, FDA really looks for control within the study, not using some external controls or very rarely natural history. So I think I really want to differentiate that from what you mentioned about uniQure. So, we do have our OCU400, OCU410ST total trials, including even OCU410 GA clinical trial, we have untreated control subjects in the clinical trial. So that's what we're using to compare. And Huma, go ahead on the clinical trial design.

Yes. Thank you, Shankar. So, the clinical trial design is we have 150 subjects that we are planning to enroll for the study, 100 in the treatment and 50 in the control, 75 in the rhodopsin and 75 in the gene-agnostic arm with 50 being active and 25 in the control. We have 97% power for this study. We have assumed 50% and 10% treatment effect in the treated and the untreated eyes. And there is a 2:1 randomization, which means there is -- we are treating both the eyes as they meet the inclusion/exclusion criteria with the worst eye being dosed first. This is the only trial that is globally available with clinical and/or genetic diagnosis of RP with syndromic and covering non-syndromic forms.

And maybe a question on OCU200. You mentioned the milestone that we'll see completion of enrollment by the end of the year in Phase 1. Can you comment when we'll see the data? And what would that initial data include?

So we are on track for our OCU200 enrollment for our Phase 1. Our periodic safety updates are there. There are no serious adverse events related to the product listed as of right now. Yes, in the beginning of next year, we will be providing some more updates on the safety and efficacy of OCU200 as soon as it becomes available. But as this is a Phase 1 first-in-human, there are no serious adverse events related to the investigational product reported as of right now.

And how many patients will you have in that initial update?

So, we have a dose-ranging, dose escalation portion of the study. So, that would be almost 9 to 12 subjects.

Your next question comes from the line of Swayampakula Ramakanth with H.C. Wainwright.

So, a couple of quick questions. The first one on OCU400, as you're nearing to the point where you're putting not only your application together, but also thinking about commercialization, what sort of investments are you making within the commercial infrastructure so that you're allocating appropriately within your budget for pre-commercial activities? Yes, let me start with that.

Yes. I think, I mean, based on 2027 launch next year, we will be ramping up slowly for U.S. commercialization. Obviously, our goal is to continue to look for partnerships just like we announced one in South Korea and other regional partnerships in Europe, Japan and elsewhere. But U.S., we are looking still into because it's a good market. We have a good handle. We created a lot of the centers for excellence, which will be part of the commercialization for subretinal delivery. Therefore, we will have more information provided next year, but we'll slowly ramp up. We'll be allocating a carefully limited budget starting with. But we're looking into other revenues to beef that up next year.

Okay. And then on the ST, with the comment that it's reaching 50% enrollment on the GARDian3 trial, what's the cadence of enrollment there? Just trying to understand how the enrollment is going. Are there any geographies where you're seeing less of an enrollment? How many patients you're screening so that you get the enrollment moving?

Yes. I'll let Huma take that.

So, this trial includes 51 subjects, 34 in the treatment, 17 in the control. We are almost 50% and above in our enrollment. We have completed that, and we are on track. So, there are no geographical restrictions here. Actually, I would say, this is basically the disease of the pediatric population, and we are covering early to late stages of Stargardt, also 3 years of age and above. That's why we are pretty confident we are going to meet the enrollment goal. In fact, we do have almost half of what we have enrolled more than that in the screening queue. So, we are on track for completion of our enrollment. But there is no geographic restrictions. There are almost 15 centers that we have equally covered across in the U.S. and all of them have the screening metrics for those patients as well. So, we are on track.

Shankar, if I may, can I ask one quick one on OCU400 itself? Having looked at the data from the earlier studies and the patient characteristics, have you folks identified any of the patients? Obviously, there are some who are high responders and some who are non-responders. In general, how are you thinking about what the real potential is for OCU400? And what could your commercial strategy be so that you try to get the highest adoption that you could get?

So RK, I mean, our design clearly outlines based on the strong scientific basis, we are targeting entire RP from early-stage to advanced pediatric to adult, and that's the coverage. And I mean, we are including in our Phase 3 clinical trial all major components of RP. That means some of the mutations with high prevalence rhodopsin or XLRP, [ RUSHERS ] and PDE6B, all those mutations are included with good representation. So our goal is to look at the data holistic and get the broad RP indication, so that we provide a treatment potentially for all RP patients, not restricted to specific genotype.

Your next question comes from the line of Robert LeBoyer with NOBLE Capital.

Just as a follow-up from some of the previous questions, my understanding of OCU400 is that it's a regulatory gene that affects the entire gene network downstream, restoring homeostasis. So, early-stage, late-stage, child, adult, specific mutations really wouldn't matter because it's affecting the whole downstream pathway that leads to blindness. So please let me know if that is the correct way to think about it.

Yes, absolutely, Robert. You stated it.

Okay. And secondly, the OCU410 in GA is also ongoing. And could you just go over some of the endpoints and what to expect in that trial?

Yes. Huma?

So, we have completed our ArMaDa enrollment Phase 1/2. The Phase 1 was a typical dose-ranging, dose escalation, 3 plus 3 design, 9 subjects there. And also we had a Phase 2, where we had a high dose, medium dose and a controlled, which means that we enrolled 17 subjects in high dose, 17 in the medium dose and 17 in the controlled. Also in terms of the endpoints in Phase 2/3, which is important, it's Stargardt atrophy lesion growth reduction from baseline and also looking at low luminance visual equity over the period of time as well, which is functional we have been consistently releasing the data on that, and we are on track for following up those patients until early part of next year.

So there is a 1-year time point, Robert, to clarify. Our GA trial is a 1-year trial.

Your next question comes from the line of Elemer Piros with Lucid Capital Markets.

So, Shankar, you talked about OCU410ST and your agreement -- OCU410ST, your agreement with the European agency that the U.S. trial would be sufficient. Do you have a similar agreement related to the RP program as well?

Yes. We have a similar agreement with [indiscernible], with EMA, on RP program.

Okay. And what would be the regulatory path in South Korea for OCU400?

Yes. Currently, it looks like South Korea and the rest of the world, typically for orphan gene therapies, they are following FDA closely. And once we get FDA approval, based on the FDA approval, we get approvals in those countries. So most of these countries don't need any additional clinical trials.

I understand. And one housekeeping question, how much of the $7.5 million or up to $7.5 million that you received from Kwangdong is included in the cash balance that you reported?

The $7.5 million is not included in the cash burn at this point of time. It is going to be in future. So that's not part of our cash burn at this point of time.

And could we project some into the fourth quarter in terms of the initial payment?

The $1 million, which we received from Kwangdong, that is already in the cash projection, but nothing more than that.

Okay, $1 million. Okay. And lastly, could you talk about your manufacturing capacity, Shankar, especially when we think about much larger indications such as OCU410 for geographic atrophy?

Yes. The manufacturing capacity, we have ex-U.S. partner with plenty of capacity. I mean, we have our own facility in our backyard in Malvern, Pennsylvania, and that facility will be ready. Our plan is to get that ready by 2027. And when we get the first approval, it's called a prior approval supplement with the U.S. FDA at the site. And our goal in future is to produce all U.S. supply from our U.S. manufacturing site.

But you mentioned also that you have an ex-U.S. partner as well, manufacturing partner.

Yes. That's right. Yes, currently. And we have plenty of capacity, global capacity.

Final question comes from the line of Daniil Gataulin with Chardan. Please go ahead.

First on OCU400, the trial, as we know, you have 2 arms, one with RHO mutations, one with RP gene-agnostic mutations. For the gene-agnostic arm, are you disclosing how many different mutations are included there? And in terms of the enrollment, did one arm see a more robust enrollment than the other?

Go ahead.

So actually, it's an assessor-blinded study. So, at this point, we cannot disclose. Gene agnostic means that all the major mutations will be covered, which is more than 95% geographically look at it in U.S. and globally. So we are going to cover that in the gene agnostic and all forms of rhodopsin are going to be in the rhodopsin arm. So that is the first one. And yes, there is a robust enrollment randomization that we are proceeding with. As we stated, clinical and/or genetic diagnosis, syndromic, as well as non-syndromic forms.

In terms of prep for BLA filing for OCU400, what are the outstanding CMC -- or what is the outstanding CMC work that needs to be completed before you file?

Yes. With the RMAT designation, Daniil, we have opportunity to initiate rolling submission, as we stated, in the first half of next year. And so you can submit nonclinical and CMC sections. So, as far as CMC is concerned, we have to complete our process validation runs for drug substance and drug product, and it's progressing really well, and we're on target. So, those sections will be submitted before we submit the clinical section. That will be the last one late next year. So somewhere in the middle of next year, somewhere we'll be submitting the manufacturing section. We'll start with nonclinical, followed by CMC, followed by clinical.

And one last question for LCA. Is that on hold for now or is that completely off or out of your pipeline? Or what are your plans for LCA?

From a market perspective, Daniil, it's very small. What we'll do is -- right now, it's not in our plan. And obviously, once the RP gets into the market, we can always look into that for Phase 4.

This concludes the Q&A portion. I will now turn the call back over to Chairman, CEO and Co-Founder, Dr. Shankar Musunuri. Please go ahead.

Thank you, operator. The third quarter was marked with important clinical progress, strategic business development and essential financing accomplishments. We are poised to close 2025 on a strong note and look forward to early 2026 catalysts that will further advance Ocugen's position as a biotechnology leader in gene therapy for blindness disease. Have a great day. Thank you.