Ocugen, Inc.

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. All participant lines are in a listen only mode. Following the speaker's commentary, there will be a question and answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's head of corporate communications. You may begin.

Thank you, operator. Good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri, OccuGen'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 30th, 2025. Dr. Huma Kumar, chief medical officer, will be available to answer questions following the presentations. 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 Occugen.com. This call is being recorded, and a replay with the accompanying slide presentation will be available on the investor section of the Occugen 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, potentials, 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 activity 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 speak 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, Ogden's Court of the Report on Form 10-Q, covering the third quarter of 2025, will be filed today. I will now turn the call over to Dr. Musuneri.

Thank you, Tiffany. Thank you all for joining us today. I'm pleased to share an update on our modified gene therapy platform, and would like to recognize that in just over three years, we brought our lead candidate, OQ400, from initial Phase I-II dosing to nearing Phase 3 enrollment completion. The RQ4-10ST Phase 2-3 pivotal confirmatory trial is following close behind, and we're 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 RQ4-10ST. This rapid progress is somewhat unheard of in industry. and not only reinforces our commitment to file three BLAs in the next three years, but it also brings us closer to addressing the incredible unmet medical needs that exist for patients facing vision loss. While all three programs are moving along on schedule, we received additional positive news in the third quarter that the Committee for Medicinal Products for Human Use, THMP, 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 project efficiencies in Europe as we have with the OCU400 total trials. To fund total trial progress, we continue to pursue opportunities to increase our working capital And in August, closed the registered direct offering to Janice 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 OCU 400 Phase III Limelight Clinical Trial remains on track. for BLA and MAI submissions in 2026. This 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 three orphan gene therapy trial. There are approximately 300,000 people in the US and Europe combined living with RP. which affects more than 100 genes. Opigen'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, RP65, 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 Aukey 400 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 market initiatives led by Abhi Gupta, our EVP of commercial and business development are scaling up as well. We will begin rolling submission of the RQ400 BLA in the first half of 2026 and release phase three 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 RQ400 by 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 Kwong Dong 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 Kwong Dong and will be responsible for manufacturing and supplying Accu 400. There are an estimated 7,000 individuals in the Republic of Korea with RP, which represents approximately 7% of the U.S. market. OQ400 provides the opportunity for our partner to help thousands of patients facing vision loss. Upon regulatory approval of OQ400 in Korea, we believe Hwang Dong will become a leader in the field of ophthalmic gene therapy in South Korea. Now, let's move on to Ocuportin ST. Ocuportin ST has the potential to target all 1200 pathogens mutations in the ABCA4 gene associated with Stargardt disease and other ABCA4 related retinopathies with a single one-time subretinal injection. As I mentioned earlier, Enrollment in the Phase 2-3 Guardian 3 clinical trial is ahead of schedule. The strong response underscores the significant unmet medical needs among Stargardt patients who currently have no approved treatment options available. Stargardt disease affects approximately 100,000 people in the US and Europe combined, and approximately 1 million people globally. With CHMP acceptance of US trial data, for the NAA submission will maximize resources and streamline development efforts with the goal of bringing Occu410-ST 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 six-letter gain in visual equity in available 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 RQ410ST to patients in need. Finally, Ocu410 is specifically designed to address multiple pathways implicated in the pathogenesis of dry edulated 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 intraorbital injections. about six to 12 doses per year and are accompanied by various safety risks. For example, roughly 12% of patients developed with AMD following treatment. With approximately two to three million geography 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 fail to demonstrate meaningful functional outcomes. OCU410 is therefore well-positioned to address this critical medical need. At 12 months, available subjects in the Phase 1 study showed a 23% reduction in lesion growth, along with a two-line or 10-letter stabilization or GAID with visual equity in treated eyes. Preliminary results from six-month interim analysis demonstrated a 27% reduction in lesion growth and preservation of retinal tissue in the treated eyes when compared to untreated controlled eyes. This reduction is or twice that observed with currently approved intravitreal therapies at six months monthly and every other month PEC Cytocoplan 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 one-time treatment. In addition to the greater lesion production, a single subretinal injection of Ocu410 demonstrates greater efficacy in preserving retinal tissue surrounding GA lesions compared with monthly and every other month type statistical plan treatments. We plan to provide full 12-month data from the phase two study, including both structural and functional outcomes in the first quarter of 2026 and anticipate initiating the phase three 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 30th, 2025.

Thank you, Shankar. The company's cash, cash equivalents, and restricted cash total $32.9 million as of September 30th, 2025, compared to $58.8 million as of December 31st, 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 three months ended September 30th, 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 with total operating expenses for the three months under 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?

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

Hey, guys. Thank you so much for taking my questions today. Congratulations on all the progress.

Thank you, Michael.

So I guess to just start out, I'd like to ask a little bit about OCU 400, and in particular, the timing of the BLA completion. You mentioned that you're going to be starting that in the first half of 26, 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 gets in.

I think, I mean, we're nearing completion as we stated. And so we will have resources ready to turn around in weeks.

All right. It'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?

Yeah, or good question. Our PPQ, our process validation runs are doing 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 large-made ready to go. All right.

Thank you. And then just one more from me, and I'll hop back into the queue. For 410ST, 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 two, yes, it's already in the clinical protocol. We're 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 mid-year 26. Will that be publicly released or is that going to be internal?

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

All right. Thank you very much. I appreciate the update. It looks like there's a lot to look forward to at AcuGen. Thank you.

Your next question comes from the line of Boris Peeker with Titan Partners. Please go ahead.

Good morning. A couple questions from me. Maybe let's start with the 400. 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. I think this question comes in a context of what we've seen just recently with Unicure, where it sounds like the FDA may have moved the goalpost a little bit in their gene therapy. So I think we just want to get a sense to make sure that a similar dynamic doesn't happen here.

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 II 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 UNICUR. So we do have our ARCU-400, ARCU-410ST, total trials, including even ARCU 410 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.

Yeah, 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 two-in-one randomization, which means 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.

Got it. Great. That's very helpful. Maybe a question on IQ200. You mentioned the milestone that we'll see completion of enrollment by the end of the year in phase one. Can you comment when we'll see the data and what would that initial data include?

Yeah, go ahead. So we are on track for our OCU 200 enrollment for our phase one. 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 OCU 200 as soon as it becomes available. But as this is a phase one for student-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.

Great. Thank you very much for taking my questions.

Your next question comes from the line of Soyambakula Ramankar with HC Wainwright. Please go ahead.

Thank you. Good morning, Shankar and team. So a couple of quick questions. The first one on NAWQ 400. As, you know, as you're nearing to the point where you're putting not only your application together but also thinking about commercialization, You know, what sort of investments are you making within the commercial infrastructure so that, you know, you're allocating appropriately within your budget for pre-commercial activities? Yeah, let me start with that.

Yeah, good morning, Yarkin. Thank you. Yeah, 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 these centers for excellence, which will be part of the commercialization for sub-retinal deliveries. Therefore, we will have more information provided next year, but we'll slowly ramp up. We'll be allocating carefully limited budget starting with, but we're looking into other revenues to beef that up next year.

Okay, and then on the AST, you know, with the comment that, you know, it's reaching 50% enrollment on the Guardian 3 trial, What's the cadence of enrollment there? Are you having... Just trying to understand how the enrollment is going. Is there any geographic... Are there any geographies where you're seeing less of an enrollment? How many patients you need to... You're screening so that you get the enrollment moving.

Yeah, I'll let Huma take that.

Huma, go ahead. Thank you, Shankar. 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 three 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, you know, the screening metrics for those patients as well. So we are on track.

Shankar, if I may, can I ask one quick one on RQ400 itself? You know, are there, having looked at the data from the earlier studies on 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 ARCA-400? And, you know, what could your commercial strategy be so that, you know, you try to get the highest adoption that you could get?

Yeah. 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 III clinical trial, all major components of RP. I mean, that means some of the mutations with high prevalence, like 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 the treatment potentially for all RP patients, not

restricted to specific genotype. Thank you. Yeah, go ahead. Okay, please go ahead. No, no, thanks. That's what I said. Thanks for taking my question.

Your next question comes from the line of Robert Leboyer with Noble Capital. Please go ahead.

Good morning. 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.

Yeah, 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, Timon.

yes yes so um hi robert so we have completed our armada enrollment uh phase one uh slash two the phase one was a typical dose ranging dose escalation three plus three design nine subjects there and also we had a phase two where we had a high dose medium dose and a control which means that we enrolled 17 subjects in high dose 17 in the medium dose and 17 in the control. Also in terms of the endpoints in phase two slash three, which is important, it's such graphic atrophy lesion growth reduction for 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 that's a one-year time point, Robert, to clarify.

Although our GA trial is a one-year trial. Okay, great. Thank you very much for that detail. You're welcome.

Your next question comes from the line of Elmer Piros with Lucid Capital and Markets. Please go ahead.

Yes, good morning. So, Shankar, you talked about OQ410-ST and your agreement. No, I think, yes, OQ410-ST, your agreement with the European agency that the US trial would be sufficient. Do you have a similar agreement related to the RP program as well?

Yes.

Yeah.

We have similar agreement with the EU. Okay.

And what would be the regulatory path in South Korea for 400?

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

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

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

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

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

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

Yeah, the manufacturing capacity, we have an ex-US partner with plenty of capacity. And the goal is to, I mean, we have our own facility in our backyard in Melbourne, 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 prior approval supplement with the US FDA. We have to add the site. And our goal in future is to produce all US supply from our US manufacturing site.

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

Yes, that's right. Yeah, currently, yeah. And we have plenty of capacity, global capacity.

Okay, thank you so much.

Final question comes from the line of Daniel Catalan with Chardon. Please go ahead.

Yes, hi, good morning, guys. Thank you for taking my questions. I have a couple here. First, on OCU-400, the trial... As we know, you have two arms, one with thron 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 I can. Thank you. Thanks for the question. So actually, it's an assessor blinded studies. At this point, we can not disclose gene agnostic means that all the major mutations will be covered, which is more than ninety five percent geographically look at it in US and globally. So we are going to cover that in the gene diagnostic and all forms of rhodopsin are going to be in the rhodopsinome. 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.

Okay, got it. In terms of PrEP for BLA filing for OCU400, What are the outstanding CMC, or what is the outstanding CMC work that is, that needs to be completed before you file?

Yeah, with the automatic designation, Daniel, we have opportunity to initiate rolling submission, as we stated, in the first half of next year. And so you can submit non-clinical and CMC sections. So as far as CMC is concerned, we had 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 non-clinical, followed by CMC, followed by clinical.

Okay, got it. Understand. 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?

Yeah, that's a, from market perspective, then it's very small. What we'll do is that's 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.

Okay. Got it. All right. Thank you very much for taking my questions.

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

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

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