Ocugen, Inc. (OCGN) Q3 2026 Earnings Call Transcript

Good morning, and welcome to Ocugent's third quarter 2026 financial results and business update. All participants are currently 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, Ocugent's VP of Communications. You may begin.

Thank you, Operator, and good morning, everyone. Joining me on today's call on webcast is Dr. Shankar Musonori, Ockenden Chairman, CEO, and Co-Founder, who will provide a business update and an overview of our clinical and operational progress. Rita Johnson-Green, our Chief Financial Officer, is also on the call to provide a financial update for the quarter ended September 30, 2026. Dr. Mohamed Ghanid, our Chief Medical Officer, Dr. Arun Upadhyay, our Chief Scientific Officer, and Abe Gupta, Executive Vice President of Commercial and Business Development, will be available to answer questions following the presentation. This morning, we issued a press release covering our business and operational highlights for the third 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 slide presentation, will be available on the Investors section of the Ocugen website. Before we begin, 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, international expansion, and financial information, and our anticipated cash runway. These statements reflect management's current expectations and are inherently subject to risks, uncertainties, and assumptions that may cause actual results to materially differ 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, Ocugen's quarterly report on Form 10-Q, covering the third quarter of 2026, will be filed today. I will now turn the call over to Dr. Musonori.

Thank you, Chris, and good morning, everyone. The third quarter was a quarter of execution for Archigen. We now have all three of our late-stage modified gene therapy programs in Phase III. We advanced our business development strategy, and we established a presence in Europe to support pre-commercial activities. With three phase three programs underway, we're executing against a clear strategy to advance our pipeline and build our global presence through strategic partnerships to bring our therapies to patients around the world. Our modified gene therapy platform remains the foundation of everything we do. Rather than targeting a single mutation, it is designed to address the underlying biology of complex retinal disease by restoring balance across multiple gene networks. The platform is gene agnostic. It is multifactorial, and it is designed to deliver durable benefit from a single, one-time injection. That approach is what allows us to advance one platform across three major causes of blindness. Geographic Atrophy, Retinitis Pigmentosa, and Stargardt Disease. We remain on track to 5.3 BLS by 2028, and our next major milestone is clear. OCU400 is expected to be the first of our Phase III programs to read out, with top-line data anticipated in the first quarter of 2027. Let me walk you through the progress made across each program this quarter. I will begin OCU-400 in RP, followed by OCU-410 in Stargard disease, and OCU-410 in GA before handing the call to Rita for the financial update. In OCU-400 for RP, this was an especially productive quarter for the program with progress on clinical, regulatory, and commercial firms. On the clinical side, we completed enrollment in our Phase III line-wide trial with 140 patients. Top-line data from the trial are expected in the first quarter of 2027, followed by our planned BLA submission. Our process performance qualification batches are complete, which supports both our BLA and the commercial line supply of OCU-400. On the commercial side, in October, we completed an exclusive OCU-400 license deal for the Middle East and North Africa with Roots Pharmaceutical. Finalizing the binding term sheet we announced in July, under the agreement, Octogen is eligible for up to $4 million in combined upfront and potential initial development milestone payments, up to $255 million in potential sales milestones, and a 22% royalty on net sales, and we will manufacture commercial supply. This is our second reason of RQ400 license following our agreement with Kwon Dong Pharmaceutical in South Korea. On the regulatory and access side, RQ400 received provisional approval and priority designation in the Bahamas under its Longevity and Regenerative Therapies Act or LATO. We have executed an agreement with a local partner, the Global Alliance for Regenerative Medicine. To coordinate and manage patient care, and we are progressing site readiness and preparation for full lot of approval. We're advancing toward potential patient enrollment as early as year end. This pathway is a meaningful step toward expanding access for RP patients while potentially generating commercial revenues. Taken together, the Access Pathway in the Bahamas and the Roots License Agreement reflected deliberate strategy. We intend to bring OCU400 to patients in the United States to extend its reach internationally to regional partnerships that add commercial scale without requiring us to build our own infrastructure in every market. As we bring that international reach, we remain focused on the clinical development pathway that will underpin our efforts to bring OCU400 to patients in major markets. Our Phase I-II experience also provides important context for the Phase III limelight study. In a post hoc analysis of seven Phase I-II subjects meeting Phase III eligibility criteria, Here, five of seven treatise are 71.4%. are projected to meet the Phase III responder threshold of at least a two-level improvement on the LDNA scale from baseline at Week 52. LDNA measures functional vision with a wider range and finer resolution than the mobility test used in Phase I-II, addressing the ceiling effect seen in the study. Our previously reported three-year follow-up adds the longer-term perspective. Seven out of eight valuable consented-treated subjects, or 88%, showed improvement or preservation of LLVA compared with untreated eyes at year three, with no serious adverse events related to OCU-400 reported through three years. Separately, our phase 3 power analysis shows that even at hypothetical treatment responder rate of just 20%, Compared with 1% in untreated controls, the model estimates 93.2% power to detect a difference on the primary endpoint. We believe these data support both the design of limelight and the potential of OCU400, and we look forward to top-line results in the first quarter of 2027. Turning to OCU410ST for Stargardt disease, a pediatric onset retinal disorder for which there are no approved therapies. We continue to advance our pivotal phase 2-3 guardian trial for protocol, and we anticipate top-line data in the second quarter of 2027 with the BLA submission to follow. Finally, Acuporten GA, an advanced form of dry agulated macular degeneration, and our largest commercial opportunity. This quarter marked an important transition for the program into phase 3. In September, we announced the first patient dosed in Armada III, our global Phase III clinical trial. We plan to complete trial enrollment next year with a target BLA filing in 2028. Armada III is a 237-subject study that randomizes patients two to one to a single subretinal injection of RQ410 Not Untreated Control across the United States, Canada, and Europe. The primary endpoint is GA lesion growth over 12 months. This is the first pivotal gene therapy trial in geographic atrophy. Today's approved GA therapies target a single pathway and require ongoing introductory reactions as often as monthly. Ocu410 is designed to address multiple pathways underlying the disease with a single, one-time treatment. We believe that combination of mechanism and durability gives Ocu410 a clear, competitive advantage. Here is a snapshot of the market opportunity across our three late-stage development programs. With OCU-410 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. With that, I will turn the call over to Rita for the financial update. Rita?

Thank you, Shankar. Good morning, everyone. Our cash, cash equivalents, restricted cash, and short-term investments totaled $84.6 million as of September 30, 2026. We expect this balance to support our cash runway into 2028, which funds two of our three late-stage programs through their anticipated BLA filings. We had 339.3 million shares of Common Stock Outstanding as of September 30, 2026. Total operating expenses for the three months ended September 30, 2026, were $19.7 million and included research and development expenses of $12.5 million and general and administrative expenses of $7.2 million. This compared to total operating expenses for the three months ended September 30, 2025, of $19.4 million, which included research and development expenses of $11.1 million and general and administrative expenses of $8.2 million. Ocugen reported a $0.01 net loss per common share for the three months ended September 30, 2026, compared to a $0.07 net loss per common share for the three months ended September 30, 2025. Total operating expenses for the nine months ended September 30, 2026, were $57 million, and included research and development expenses of $34.4 million, and general and administrative expenses of $22.6 million. Compared to total operating expenses for the nine months ended September 30, 2025 of $50.5 million that included research and development expenses of $29.1 million and general and administrative expenses of $24.4 million. Ocugen reported a $0.14 net loss per common share for the nine months ended September 30, 2026, compared to a $0.17 net loss per common share for the nine months ended September 30, 2025. That concludes my financial update. Sankar, back to you.

Thank you, Rita. The progress we've made this quarter reinforces our confidence in the paths ahead. We moved RQ410 into Phase III with the first patient dosed in RMRF III. We completed our regional licensing agreement for RQ400 in the Middle East and North Africa. We opened a pathway to treat RQ patients in the Bahamas, and we established a European presence to support our development and commercialization for that reason. Each of these modes supports the same objective, which is advancing three late-stage gene therapy programs toward approval and bringing them to patients around the world. Looking ahead, our focus is firmly on execution. The most important near-term catalyst is top-line data from RQ400 in the first quarter of 2027, followed by our planned BLA submission. We then expect top-line from RQ410ST in the second quarter of 2027. We remain on track to deliver three BLAs for the next three years. I want to thank our employees, our investigators, and the patients who have placed their trust in us, as well as our shareholders for their continued support of our mission to advance Cures for Blindness. We will now open the call for questions. Operator?

At this time, I would like to remind everyone in order to ask a question, 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 Swayam Pakula Ramakhan with H.E. Winride. Your line is open.

Thank you. This is R.K. from H.E. Winride. Good morning, Shankar and Rita. And the rest of the team. Just wanted to understand a little bit about the ST program, the Stargardt program, Shankar. You know, in September, you had the DMC recommendation come to you. And when will the full cohort actually complete, you know, the follow-up period, the eight-month follow-up period? And beyond that, you know, what sort of data do you need to make your next decision? And also, I think you're still planning to file in the second quarter 27. So what else needs to get resolved by then?

Yeah. Thank you. Okay. Good morning. Yeah, the protocol, it's targeted for 12 months read in second quarter. I'll let our CMO, Dr. Ganeet, answer the questions. Go ahead. Thank you, Shankar.

Good morning, RK. So, as we subside in the protocol, this interim analysis was pre-subsified in the original protocol we discussed with the agency, and it was a part of our adaptive design to look at any adjustment need during, you know, the trial, conduction, and the DMC was assembled, as you're aware, and they recommended to continue to start without modification. And so, us. As a company, all the team are still blinded. We are continuing the study until month 12, and we're looking forward to releasing the data by second half or second quarter next year in 2027. As you are aware, too, about this internal analysis, it was done in a small cohort of patients in the study. It was, you know, an early time point, the month 8 versus month 12 as a primary. As you know, for Stargate disease as a disease, it is a slowly progressive retina-generated disease, so we wouldn't expect to see too much at month 8 versus month 12. In addition, as you know, with gene therapy product, the suppression of the transgene and the product take time. And then what we are trying to do here, we're looking at the data at month 12 per, you know, the original design on the pre-existing protocol.

Okay, thank you. So if I understand this right, so I know I misspoke. I said second quarter 27 filing. The readout is the second quarter, just as you said. So once you have all that data. You anticipate, you know, meeting with the FDA before moving forward, or, you know, what other steps, you know, would you be taking once you get that 12-month data point, just as you suggested?

Yeah. Yeah, I think, yeah, that's the standard operating procedure, RK. When you get the top-line data, you need to discuss with agency first. Then you need to, if the data falls the way you anticipate, following the protocol and SAP analysis, what we agreed with the FDA, obviously, then you move on to the next step, you know, have a pre-BLA meeting and move on to filing the BLA.

Fantastic. Then regarding the LARTA approval in Bahamas, first of all, I think it's a good opportunity for you folks to get something commercial and also see how patients react or adopt in real world, which is fantastic. So when you say by year end, by the end of this year, what would we see, you know, in terms of is it just going to be full approval and potentially somebody lining up to have their initial surgery or you just will have the full approval and the hospital will be trying to get somebody in, get a surgery done? Yeah, in the new, in 2007, I mean.

Yeah. So, R.K., I let, you know, we have our commercial head here. I think I'll tell you my answer.

So, right, R.K., nice to hear from you. So, the LARDA program in Bahamas really represents something really innovative across our industry. And, yes, in addition, right now we have a conditional provisional approval, but by the end of the year, we can expect a full approval. We don't necessarily share forecasts on patients, especially for EAP, but stay tuned, and we will be sharing more catalysts on that specific program.

Perfect. Thank you. Thanks for taking my questions. I'll step back.

Your next question comes from the line of Robert Le Boyer with Nabu. Your line is open.

Good morning, everyone, and congratulations on the progress during the quarter. My question only has to do with the 410 program, and good news that you initiated patient treatment during the quarter, and my understanding is that it's a two-to-one randomization. But I don't have anything about any kind of interim analysis. Is there anything planned like that?

Yeah. Robert, this is a one-year trial. That means after recruitment, we're going to monitor patients for one year, looking for primary and secondary analysis, and currently they're not planning.

Okay. Are there any milestones for recruitment that you're planning to announce, or is it just when you complete the full enrollment?

I mean, our goal is to complete enrollment in 2027 in line for BLA filing in 2028.

Okay, great. So everything's on track. Sounds good. Okay. Thank you very much. Thank you, Robert.

Your next question comes from the line of Michael Oconiewicz with Maxim Group. Your line is open.

Hi. Thank you, guys, for taking my question. Congrats on all the progress this quarter. Thank you. I'd like to just talk a little bit more about the upcoming DMC review in Stargard and provide some context on what the issues raised at the prior one were. Did this have anything to do with the magnitude of treatment effect over the time period? Was it fluctuation in functional measures over that period with a smaller population? Or was it statistical noise at baseline that kind of led them to say, we want to see the full picture before making a decision?

Yeah, thank you for the question. So, it's all of the above, but let me give you more specifics. Let's just go back to the disease progression, because this is important to understand. Stargate, as you know, it's one of those slowly progressive retina-generated diseases, and it takes time to see any delta, and eight months was quite early time point. This is number one. Number two, if you remember the interim analysis, also we look at only a snapshot of certain patients, only not half, even less than half of the patient in that trial. So the small n definitely will determine this. The other big piece was in this analysis, the heterogeneity we saw at the baseline of this patient. Remember, still less than half of the patient enrolled in the trial, and the baseline characteristic was just heterogeneous. It was quite, you know, variable from small to large, so we cannot conclude, we cannot claim anything as well. Lastly, and I want to also reiterate this part. This was not an efficacy analysis. It was a part of the adaptive design to look at any need to do any adjustment on the trial design, increase duration, increase the number of patients, and the DMC recommended to continue without any modification. And for us, as a company, we will continue to follow the patient per the existing protocol until month 12, and then we'll look at the data in the full set of the patient at month 12.

And just how confident are you that the N equals 63 total size of the study is large enough to kind of ameliorate any of those baseline differences?

Yeah, good point. Based on the prior data from the phase 1-2, we saw, you know, treatment effect, you know, small number of patients was N of 9. We saw the 54% reduction or slower lesion growth in the treated eyes versus the untreated. Also, we saw functional benefit But on the visual acuity. Back to your question about, you know, the NF63, you know, it will be the full set of patients. The study was powered, based on the earlier assumption, to show such significant months 12, was, you know, close to 90% powering. And also, we should see, you know, like more hemogenous population, the baseline characteristics should be, you know, balanced between the control and treated. That will also will drive, you know, a different value. In addition, we have a longer time point, right? Month 12 versus month 8, and we saw the delta in our early study at month 12 versus month 8.

All right. Thank you. And then just one more before I hop back into the queue, I want to switch gears and talk a little bit about the Bahamas program, and in particular, what the payment model looks like under LARDA, and who is the target patient for that? Would that be patients that live locally in the Bahamas? Would it be people flying in for potential medical tourism to get early access to the treatment? We can just provide a bit more color on what that looks like.

Yeah, I'll let Ami answer that.

Go ahead.

Yeah, so I think first on the program is open globally. And yes, it's looking at the Bahama population, but much more broader. So it is truly like a global program. And so secondly, the way that the model works in terms of pricing is something that's consistent with the way that the Bahama system is set up. And so it is a little bit more towards commercial than it is towards clinical. And that's something specific that the Bahama regulatory body enables Ocogen to do.

So, Michael, just to recap, anybody globally who is eligible and they can go get the treatment at this stage under this approval.

All right. Would pricing match what you would be targeting for U.S. or international pricing, or is this more of an exploring program? That's correct. It would be. Yeah, it would be.

It would be matching U.S. price.

Well, thank you very much. I appreciate the additional clarity.

Thank you. Your next question comes from the line of Fallamer Pyrrhus with Lucid Capital Markets. Your line is open.

Yes, good morning. Just one more follow-up on the Bahamas treatment availability. How would patients learn about that this is available? In the Bahamas for them, what sort of marketing effort if you are thinking of to undertake?

Yes, as an Ocugen, we don't market EAPs, but we are working with a local vendor that's responsible for all patient-related activities. And so they would be the one who are educating not only patients, but KOLs, including the community about this, you know, world's first option for a global EAP.

Okay. And coming back to the interim analysis, was the DMC equipped with trying to model potential outcome? If they were to normalize the baseline characteristics to be even between the two arms.

So, DMC, as you know, it's an advisory body, and it's also an independent, unblinded body assigned to the study. The DMC has a very clear period of protocol assignment. They were looking at part of the adaptive design. They're looking at three outcomes. Again, it's not an efficacy outcome. It's just part of the adaptive design for simple science, recalculation, or adjustment, if needed. They had access to the unblinded data. They looked at all the efficacy, including, you know, the primary, the lesion size, and all the other functional, including also the genetic correlation. So they were equipped, you know, to make a decision based, you know, on data. As we said before, you know, and this is important, again, to reiterate, the sample size was small, early time point, and also So, um, um, We had some heterogeneity in the baseline characteristics in that interim analysis. The good news is also, you know, remember they had, you know, opportunity, you know, to do different outcomes, they continue the study without modification because they believe, you know, with all full sets of patients at month 12, the heterogeneity and all this baseline, you know, like heterogeneity will be normalized at month 12. And this is what we also believe based on our data and also patient, you know, selection in the trial.

And so you can confirm that, since you have all the baseline characteristics of the patients, that the full data set is going to be well-balanced between the two treatment arms?

That's correct.

Okay. Okay. Thank you very much. That's all what I had.

Your next question comes from the line of Leila and Gershaw with Oppenheimer & Co. Your line is open.

All right. Thanks for taking our questions. I just wanted to ask, I think it was asked earlier on the StarGuard, the eight-month analysis. So just to be clear, will we... Will you, the company, have input from the DMC on their full analysis of the enrollment at eight months, and could that impact the study? I just wasn't clear. I know that was asked in an earlier question, but it wasn't clear your answer. If you could just inform there. Thank you.

No, it's a pleasure, and no, so the DMC is independent, but they have access to the unblinded data. We don't. The company, the study is still blinded, and all the team or running the study is still blinded. If you're asking, you know, will the DMC will do another announcement at eight months with a full data set? No. We are continuing to study until month 12, and this is where the time is going to look at all the data from the trial as a company by month 12. And this is part of the design of the study. This is a pre-existing protocol. This is why we communicate to the agency. And we believe the reason why, because eight months is an early time point, and we have a small number of patients. So we want to see the full set of patients at month 12 per the existing protocol. Yeah, I know. That's very helpful.

Thanks for clarifying.

And I guess a question in terms of the final data when you have them. If you could just remind us what you expect to see based on performance in the past with 410ST, what the size of lesion reduction you'd expect? You know, versus comparator, and given this is a dynamic space, kind of to be competitive here, what size reduction would you want to see given the other therapies being developed in this space? Thank you.

So, the primary analysis for this study was based on the atrophic lesion size change over time until month 12, and the study was far close to 90% to look at that delta. The delta you're talking about, close to 25% delta. If you remember, you know, the phase 1-2 data we shared earlier or we discussed earlier, we showed close to 54% reduction on the lesion size, but this was an early study. It was also a small number of patients, and that was our benchmark, but we went more conservative, and we talked about 25% delta. Between the treated and the controlled eyes to show statistical significance. That's on the lesion size growth. Also, we are also looking at other functional in point, including the PCVA and other functional in point in the study. And we saw earlier in our phase 1-2 functional benefit as well from baseline to month 12 up to 6-large gain in the treated eye versus the controlled or untreated eye. So, we believe, based on this earlier data, we should also see, you know, similar in our trend on the structural and functional improvement in these subjects.

That's very helpful. Thank you.

Your next question comes from the line of Whitney Aijem with Kanakorijen with you. Your line is open.

Hey, guys. Thanks for taking the question. On OCU400 and the powering, thank you for walking through that. I guess, can you remind us from the Phase 1-2 experience, first of all, how were you defining responder in the Phase 1-2 as it relates to the mobility testing, and what are the differences between how you're defining it there versus the Phase 3? And then remind us what was the responder rate, particularly in the row population in Phase 1-2, and remind us what percent of the Phase 3 are row patients. That's part one, and then I have a follow-up. Sorry for so many questions.

Hi, Whitney. Thank you for your question. You know, regarding the Phase 1-2, what we saw from the Phase 1-2 in the RP, the Ocu-400 program, this was in of a teen subject. I remember we did some, you know, subgroup announcement. What we saw based on the mobility test, and we used, you know, the same test, you know, externally used, you know, for their approval, which we call the multiple luminous mobility test, MLMT. What we saw in that study, in the treated eye who received Ocu400, we saw 43% of the eyes did improve to lux level on the mobility test, which is the approvable endpoint for RP, versus 0% of the control untreated eyes did show that. And then when we projected this, To our phase three, you know, mobility test, which we call the LDNA, which is very similar to the LMT, but it's a broader range, 10 lux level versus six, but using the same methodology, the two lux level improvement. And our projection was, on that time, we went from 42 up to, 43 up to 71%. improvement in the treated eye versus 0% in the control. So when we built our phase three assumption, and it was based on a very conservative analysis, we didn't say, you know, we're going to go with 71% improvement. We said the treated eye will, the assumption was 50% improvement in the treated eye versus 10% control. And remember, in the phase one, two, the control was 0%. So if you went with The 50% treated versus the 10% controlled, the delta is 40%, that will give us 99% powering in the study. Okay? And if you look at this, you know, in the other way, right, if you look at the half, you know, full or half empty, right, even if the treated eye shows only 20% treatment effect, which is not our assumptions, right, this is not our assumption, but if you went to 20% and the control did behave exactly the way it did behave in the phase 1-2, which is like close to 0%, given 1%, we're still hitting 93% power. So, what we're saying here, in a different way, we have a very high confidence, you know, with OCU400, especially the study, is well-powered to show statistical significance at month 12, using the two LuxWeb improvements, the same one, LuxTern, used for their approval.

Can you answer your question with me?

That's really helpful.

Yes, that's really helpful. And then just on the row bit, am I remembering correctly that on the phase one, two, the responder rate in the row subgroup was higher, or either way, I guess, can you remind us what percent of patients in the pivotal are row patients?

Yeah, so for the pivotal trial, the limelight, you know, just to remind, it's 140 subject was enrolled, randomized to 221, 92 was treated, and 48 was untreated, or the control arm. The redopsin, we have, you know, like, we have redopsin patient in the phase 3 trials, but the majority will be in non-redopsin, you know, we call it gene agnostic. But we have a very large number of patients, you know, in the phase 3 trials who have redopsin mutation. And you're correct. We saw, you know, a very positive trend in the reduction of the population in the Phase I-II. So we are expecting to replicate, you know, if not better, in our Phase III trials.

Got it. Excuse me. All right, that's helpful. And then this last question is just on timing. I believe you announced completion of enrollment in the study in the beginning of March. Can you just help us understand, like, how quickly after enrollment was that final patient treated and just trying to get and how long you think it might take to kind of organize, clean up the database, et cetera, before you can lock it and announce top line?

I mean, as we stated before, Whitney, we're ready to do all that. That's why we're doing a lot of work behind the scenes. As we announced before, first quarter, by end of first quarter, we'll be able to announce that top line. Got it. Okay. Thanks for the question.

Thank you. I will now turn the call back over to Shankar Muzunuri for closing remarks.

Thank you. Thank you for attending another good Progressive Learnings call today. Thanks to all our shareholders, patients, all our employees for supporting Ocigen. Thank you.

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