OCGN Q3 2026 earnings call transcript, November 4, 2026

Thank you for listening.

Good morning, and welcome to OCUGN'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, OCUGN'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, OCUGN Chief. 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 Upadai, 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 OCGN 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, OCGEN'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 OCGEN. 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 III 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 onetime injection. That approach is what allows us to advance one platform across three major causes of blindness. Geotrophic atrophy recognizes pigmentosa and Stargard disease.

We remain on track to file three BLAs by 2028 and our next major milestone is clear. OCU400 is expected to be the first of our phase three 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 OCU400 in RP, followed by OCU410 in Stargard disease and OCU410 in GA before handing the call to Rita for the financial update. In OCU400 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 three limelight 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 badges are complete, which supports both our BLA and the commercial line supply of OCU400. On the commercial side, in October, we completed an exclusive OCU400 license deal for the Middle East and Africa with Roots Pharmaceutical, finalizing the binding term sheet we announced in July. Under the agreement, OCGN 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 OCGN 400 license following our agreement with Guangdong Pharmaceutical in South Korea. On the regulatory and access side, OCGN 400 received provisional approval and priority designation in the Bahamas under its Longevity and Regenerative Therapies Act, or LARTA. We have executed an agreement with a local partner, the Global Alliance for Regenerative Medicine, to qualify for 100. coordinate and manage patient care. And we are progressing site readiness and preparation for full LARTA 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 from the Bahamas and the Roots License Agreement reflected deliberate strategy.

We intend to bring OCU-400 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 OCU-400 to patients in major markets. Our phase one two experience also provides important context for the phase three limelight study. In a post-hoc analysis of seven phase one two subjects meeting phase three eligibility criteria, five of seven treated eyes or 71.4% are projected to meet the phase three responder threshold of at least a two level improvement on the LD RNA scale from baseline at week 52. LD RNA measures functional vision with a wider range and final resolution than the mobility test used in phase one two, addressing the ceiling effect seen in the study. Our previously reported three year follow up adds a longer term perspective. Seven out of eight valuable consented treated subjects or 88% showed improvement or preservation of LLVA compared to with untreated eyes at year three, with no serious adverse events related to OCU-400 reported through three years. Separately, our phase three 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 RQ400, and we look forward to top line results in the first quarter of 2027. Turning to RQ410ST for Stargardt disease, a pediatric onset retinal disorder for which there are no approved therapies. We continue to advance our pivotal phase two, three guardian trial for protocol, and we anticipate top line data in the second quarter of 2027. with a 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 three. In September, we announced the first patient dosed in Armada III, our global phase three clinical trial. We plan to complete trial enrollment next year with a target B. LA filing in 2028. Armada III is a 237 subject study that randomizes patients two to one to a single subretinal injection of RQ410 or 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 injections as often as monthly. RQ410 is designed to address multiple pathways underlying the disease with a single, one-time treatment. We believe that combination of mechanism and durability gives RQ410 a clear, competitive advantage. Here is a snapshot of the market opportunity across our three late-stage development programs.

With RQ410 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 84.4%. Six million dollars as of September 30th, 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 30th, 2026. Total operating expenses for the three months ended September 30th, 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 30th, 2025, of $19.4 million, which included research and development expenses of $11.1 million, and general and administrative expenses of $19.4 million.

OCGN reported a $0.01 net loss per common share for the three months ended September 30th, 2026, compared to a $0.07 net loss per common share for the three months ended September 30th, 2025. Total operating expenses for the nine months ended September 30th, 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 30th, 2025, of $50.5 million, that included research and development expenses of $29.1 million, and general and administrative expenses of $24.4 million. reported a $0.14 net loss per common share for the nine months ended September 30th, 2026, compared to a $0.17 net loss per common share for the nine months ended September 30th, 2025. That concludes my financial update. Sankar, back to you. Thank you, Rita. The progress we made this quarter reinforces our confidence in the paths ahead. We moved Occuporten into Phase 3. With the first patient dosed in RMLF3, we completed our regional licensing agreement for RQ400 in the Middle East and North Africa. We opened a pathway to treat RQ patients in Bahamas, and we established a European presence to support our development and commercialization from that reason. Each of these moves 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 Ramakal.

Yeah, thank you, R.K. Good morning. Yeah, the protocol, it started for a 12-month read in the second quarter. I'll let our CMO, Dr. Ganeet, answer the questions. Go ahead. Thank you, Shankar. Good morning, R.K. So as we subside in the protocol, this interim analysis was a pre-subsified in the original protocol. We discussed with the agency, and it was a part of our adaptive design to look at the in-adjustment need during the trial, conduction. And the DMC was assembled, as you're aware, and they recommended to continue to study without modification. 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 release 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 at the primary. And as you know, for a target disease as a disease, it is a slowly progressive retina-generative 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, go ahead. 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... If not, you need to... 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 finding the BLA. Fantastic. Then regarding the LARTA approval in Bahamas, first of all, it's a good... I think it's a good opportunity for you folks to get something commercial and also see how they... We know that an important thing is to answer them, and maybe we'll leave a few more debats and you can ask them to change them. So, let's try it in. If it's the first step. How patients react or adopt in real world, which is fantastic. So, when you say by your 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 in the new...in 37th, I mean. Yeah. So, Arke, I let...you know, we have our commercial head here. I think I'll tell you my answer. So, right, Arke, 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. Okay. Thank you. Thanks. So I'm taking my questions. I'll step back.

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

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... 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 internal analysis, also we look at only snapshots of certain patients, only not half, even less than half of the patient in that trial. So the small end definitely will determine this outcome. The other big... 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 patients? find 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 on the visual acuity. Back to your question about, you know, the N of 63, you know, it will be the full set of patients. The study was powered. Based on the earlier assumption, to show significant at month 12, it was, you know, close to 90% powering. And also, we will should see, you know, like more hemogenous population. The baseline correct risk 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. In particular, what the payment model looks like for 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 could just provide a bit more color on what that looks like. Yeah, let me 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 OCGN to do.

So, Michael, just to recap, anybody globally who is eligible, 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 explore? If 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.

Your next question.

This 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?

this different outcome, 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 So, thank you. well-balanced between the two treatment orders. That's correct. Okay. Okay. Thank you very much. That's all what I had.

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

Hi, thanks for taking our questions. I just wanted to ask, I think it was asked earlier on the StarGuard, the eight-month analysis, if we... 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 will that, 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 a dependent body. They have access to... The unblinded data, we don't. As a 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 analysis at eight months with a full data set? No. We are continuing the 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 will be communicated 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. Did you answer your question? Okay. 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 410.

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, you know, other therapies being developed in this space? Thank you. Yeah. So the primary analysis for this study was based on the atrophic lesion size change over time to month 12. And the study was far close to 90% to look at that delta. 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. Also, we are also looking at other functional endpoints, including the PCVA and other functional endpoints in the study. And we saw earlier in our phase 1, 2, functional benefit as well from baseline to month 12, up to 6-liter gain in the treated eye versus controlled or untreated eye. So we believe, based on this earlier data, we should also see, you know, similarly in our trend on the structural and functional improvement in those subjects. That's really helpful. Thank you.

Your next question comes from the line of Whitney Aijem with Canikor Genwty. Your line is open.

from 43 up to 71% improvement in the treated eye versus 0% in the control. So when we built our phase three assumption, Whitney, 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 a 50% treated versus a 10% control, 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, a different way, we have a very high confidence, you know, with OCU-400, especially the study is well powered to show statistical significance at month 12. Using the two LuxWeb improvements, the same one looks trying to use for their approval. Did I answer your question? 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 row, the row subgroup was higher? And if, 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 LAMLITE, you know, just to remind, it's 140 subject was enrolled, randomized to 2 to 1, 92 was treated and 48. was untreated or the control arm. The redoxin, we have, you know, like, we have reproducing patients in the phase 3 trials, but the majority will be in non-redoxin, 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 redoxin mutation. And you're correct, we saw, you know, a very positive trend in the redoxin subpopulation in the phase 1-2. So we are expecting to replicate, you know, if not better in our phase 3 trials.

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 have 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 earnings call today. Thanks to all our shareholders, patients, all our employees for supporting OCGN. Thank you.

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