Ocugen (OCGN) Q3 2026 Earnings Call Transcript

DATE

Wed, Nov 04, 2026

CALL PARTICIPANTS

• Chairman, Chief Executive Officer, and Co-Founder - Shankar Musunuri
• Chief Financial Officer - Rita Johnson-Greene
• Chief Medical Officer - Mohamed Genead
• Chief Scientific Officer - Arun Upadhyay
• Executive Vice President of Commercial and Business Development - Abhi Gupta
• Vice President of Communications - Chris Clark

TAKEAWAYS

• Cash and Investments -- $84.6 million, providing a runway into 2028 and funding two late-stage programs through BLA filings.
• Net Loss per Share -- $0.01, representing a reduction from a $0.07 loss due to changes in the fair value of derivative liabilities.
• Research and Development Expenses -- $12.5 million, increasing from $11.1 million to support three active Phase III programs.
• General and Administrative Expenses -- $7.2 million, decreasing from $8.2 million reflecting management's disciplined approach.
• MENA Licensing Agreement -- Up to $4 million in upfront and development milestones, plus up to $255 million in sales milestones and a 22% royalty on net sales.
• OCU400 Enrollment -- 140 patients, completing the target for the Phase III liMeliGhT trial in retinitis pigmentosa.
• OCU400 Top-line Data -- Expected in the first quarter of 2027, serving as the first Phase III readout for the modifier gene therapy platform.
• OCU410ST Top-line Data -- Expected in the second quarter of 2027, following the completion of the Phase II/III GARDian3 trial.
• ArMaDa3 Trial Initiation -- 240 subjects, dosing the first patient in a global Phase III study for geographic atrophy.
• OCU400 Phase I/II Projection -- 71.4% responder rate, based on a post-hoc analysis of subjects meeting Phase III eligibility criteria.
• Long-term OCU400 Data -- 88% improvement or preservation, observed in subjects compared with untreated eyes at the three-year follow-up.
• liMeliGhT Statistical Power -- 93.2%, estimated even at a hypothetical 20% responder rate compared with 1% in controls.
• Stargardt Trial Sample Size -- 63 subjects, powered to detect a 25% delta in lesion growth over 12 months.
• Common Stock Outstanding -- 339.3 million shares, as of Sept. 30, 2026.
• 9-Month R&D Spending -- $34.4 million, up from $29.1 million driven by clinical trial acceleration.
• Collaborative Revenue -- $1.95 million, generated during the third quarter.
• Total Assets -- $108.1 million, compared to $43.5 million at the end of 2025.
• Convertible Notes -- $83.1 million, recorded as a current liability on the balance sheet.
• Bahamas Approval -- Provisional, allowing for potential patient enrollment and commercial revenue as early as year-end 2026.
• GA BLA Submission Target -- 2028, following the completion of the ArMaDa3 Phase III trial.

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RISKS

• Chief Medical Officer Genead warned that the baseline characteristic for the Stargardt interim analysis was just heterogeneous and variable from small to large, preventing management from claiming specific efficacy at that stage.

SUMMARY

Ocugen, Inc. (NASDAQ:OCGN) reported it has transitioned into a late-stage clinical organization with three active Phase III modifier gene therapy programs for retinitis pigmentosa, Stargardt disease, and geographic atrophy. Management reported a strategic focus on global expansion through regional licensing agreements, including a new partnership for the Middle East and North Africa market and an established affiliate in Europe. The company confirmed it remains on track to file three Biologics License Applications by 2028, supported by a cash runway extending into that same year. Management stated that its gene-agnostic platform is designed to address complex retinal diseases by restoring balance across gene networks rather than targeting individual mutations.

• Chief Executive Officer Musunuri stated, "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."
• Management established a European presence in Amsterdam to support pre-commercial activities and future marketing authorization submissions.
• The company completed Process Performance Qualification batches for OCU400, which supports both the upcoming BLA submission and commercial launch supply.
• The Bahamas pathway under the Longevity and Regenerative Therapies Act provides a global early access opportunity for retinitis pigmentosa patients.
• Chief Medical Officer Genead noted that the GARDian3 trial for Stargardt disease will continue without modification following a Data Monitoring Committee recommendation.
• The OCU410 program for geographic atrophy randomizes patients two to one to a single subretinal injection versus an untreated control group.

INDUSTRY GLOSSARY

• BCVA: Best Corrected Visual Acuity, a standard measurement of the best vision a person can achieve with corrective lenses.
• BLA: Biologics License Application, a request for permission to introduce a biologic product into interstate commerce.
• GA: Geographic Atrophy, an advanced form of dry age-related macular degeneration.
• LARTA: Longevity and Regenerative Therapies Act, a regulatory framework in The Bahamas for advanced medical treatments.
• LDNA: Luminance Dependent Navigation Assessment, a clinical endpoint measuring functional vision in various lighting conditions.
• RP: Retinitis Pigmentosa, a group of rare genetic disorders that involve a breakdown and loss of cells in the retina.
• PPQ: Process Performance Qualification, a stage in manufacturing that confirms the process can consistently produce quality products.

Full Conference Call Transcript

Operator: Good morning, and welcome to Ocugen'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, Ocugen's VP of Communications. You may begin.

Chris Clark: 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. Rita Johnson-Greene, our Chief Financial Officer, is also on the call to provide a financial update for the quarter ended September 30th, 2026. Dr. Mohamed Genead, our Chief Medical Officer, Dr. Arun Upadhyay, our Chief Scientific Officer, and Abhi 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. Musunuri.

Shankar Musunuri: Thank you, Chris, and good morning, everyone. The third quarter was a quarter of execution for Ocugen. We now have all three of our late-stage modifier 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 modifier 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 file three BLAs 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 OCU400 in RP, followed by OCU410ST in Stargardt 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 fronts. On the clinical side, we completed enrollment in our phase III 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 batches are complete, which supports both our BLA and the commercial launch supply of OCU400.

On the commercial side, in October, we completed an exclusive OCU400 license deal for the Middle East and North Africa with Roots Pharmaceutical, finalizing the binding term sheet we announced in July. Under the agreement, Ocugen 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 regional OCU400 license, following our agreement with Kwangdong Pharmaceutical in South Korea. On the regulatory and access side, OCU400 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 coordinate and manage patient care, and we are progressing site readiness in preparation for full LARTA approval. We are 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 reflect a deliberate strategy. We intend to bring OCU400 to patients in the United States to extend its reach internationally through regional partnerships that add commercial scale without requiring us to build our own infrastructure in every market.

As we build 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, five of seven treated eyes, or 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 that study.

Our previously reported three-year follow-up adds a longer-term perspective. Seven out of eight evaluable 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 OCU400 reported through three years. Separately, our phase III 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 II/III GARDian3 trial per protocol, and we anticipate top-line data in the second quarter of 2027, with a BLA submission to follow. Finally, OCU410 for GA, an advanced form of dry age-related macular degeneration and our largest commercial opportunity. This quarter marked an important transition for the program into phase III. In September, we announced the first patient dosed in ArMaDa3, our global phase III clinical trial. We plan to complete trial enrollment next year, with a target BLA filing in 2028.

ArMaDa3 is a 237-subject study that randomizes patients two to one to a single subretinal injection of OCU410 or to 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 intravitreal injections 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.

While OCU410 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?

Rita Johnson-Greene: Thank you, Shankar. Good morning, everyone. Our cash equivalents, restricted cash, and short-term investments totaled $84.6 million 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 $8.2 million. Ocugen 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. Ocugen 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. Shankar, back to you.

Shankar Musunuri: Thank you, Rita. The progress we made this quarter reinforces our confidence in the path ahead. We moved OCU410 into phase III with the first patient dosed in ArMaDa3. We completed our regional licensing agreement for OCU400 in the Middle East and North Africa. We opened a pathway to treat RP patients in Bahamas, and we established a European presence to support our development and commercialization from that region. 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 OCU400 in the first quarter of 2027, followed by our planned BLA submission. We then expect top line from OCU410ST the second quarter of 2027. We remain on track to deliver three BLAs over 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?

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 Swayampakula Ramakanth with H.C. Wainwright. Your line is open.

Swayampakula Ramakanth: Thank you. This is RK from H.C. Wainwright. Good morning, Shankar, Rita, and the rest of the team. Just wanted to understand a little bit about the ST program, the Stargardt program, Shankar. In September, you had the DMC recommendation come to you, and when will the full cohort actually complete the follow-up period, the eight-month follow-up period? Beyond that, what sort of data do you need to make your next decision? Also, I think you are still planning to file in the second quarter of 2027. So, what else needs to get resolved by then?

Shankar Musunuri: Yeah. Thank you, RK. Good morning. The protocol, it is targeted for 12 months read in second quarter. I will let our CMO, Dr. Genead, answer the questions. Go ahead.

Mohamed Genead: Thank you, Shankar. Good morning, RK. So, as we specified in the protocol, this interim analysis was pre-specified 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 the trial conduction. The DMC was assembled, as you are aware, and they recommended to continue the study without modification. So us, as a company, all the team are still blinded. We are continuing the study till month 12, and we are looking forward to release the data by second half or second quarter next year in 2027.

As you are aware too, about this interim analysis was done in a small cohort of patient in the study. It was an early time point, the month eight versus month 12 as a primary. As you know, for Stargardt disease, the disease is a slowly progressive retinal degenerative disease, so we wouldn't expect to see too much at month eight versus month 12. In addition, as you know, with gene therapy product, the expression of the transgene on the product take time. What we are trying to do here, we're looking at the data at month 12 per the original design on the preexisting protocol.

Swayampakula Ramakanth: Okay, thank you. If I understand this right, I know I misspoke. I said second quarter 2027 filing. The readout is the second quarter, just as you said. Once you have all that data, you anticipate meeting with the FDA before moving forward, or what other steps would you be taking once you get that 12-month data point, just as you suggested?

Shankar Musunuri: Yeah. Go ahead, Shankar. I think, yeah, that's the standard operating procedure, RK. When we 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. Have a pre-BLA meeting and move on to filing the BLA.

Swayampakula Ramakanth: 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. When you say by year end, by the end of this year, what would we see in terms of, is it just going to be full approval and potentially somebody lining up to have their initial surgery, or you will just will have the full approval and the hospital will be trying to get somebody in, get a surgery done–

Shankar Musunuri: Yeah.

Swayampakula Ramakanth: –in the new, in 2027, I mean.

Shankar Musunuri: Yeah. RK, I let, we have our Commercial Head here, Abhi, let him answer.

Abhi Gupta: RK, nice to hear from you. The LARTA program in Bahamas really represents something really innovative across our industry. 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 catalyst on that specific program.

Swayampakula Ramakanth: Okay. Thanks for taking my questions. I'll step back.

Operator: Your next question comes from the line of Robert LeBoyer with Noble. Your line is open.

Robert LeBoyer: Good morning, everyone, and congratulations on the progress during the quarter. My question only has to do with the OCU410 program, and good news that you initiated patient treatment during the quarter. My understanding is it's a 2:1 randomization, but I don't have anything about any kind of interim analysis. Is there anything planned like that?

Shankar Musunuri: 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, we're not planning interim.

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

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

Robert LeBoyer: Okay, great. Everything's on track.

Shankar Musunuri: Yes.

Robert LeBoyer: Sounds good. Okay, thank you very much.

Shankar Musunuri: Thank you, Robert.

Operator: Your next question comes from the line of Michael Okunewitch with Maxim Group. Your line is open.

Michael Okunewitch: Hi, thank you guys for taking my question. Congrats on all the progress this quarter.

Shankar Musunuri: Thank you.

Michael Okunewitch: I'd like to see you just talk a little bit more about the upcoming DMC review in Stargardt and provide some context on what the issues raised at the prior one. 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 the statistical noise at baseline that kind of led them to say, "We want to see the full picture before making a decision"?

Mohamed Genead: Yeah, thank you for the question. It is all of the above, but let me give you more specific. Let us just go back to the disease progression, because this is important to understand. Stargardt, as you know, it is one of those slowly progressive retinal degenerative 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 looked at only 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 outcome.

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 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 if there 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.

For us, as a company, we will continue to follow the patient per the existing protocol till month 12, and then we will look at the data and the full set of the patient at month 12.

Michael Okunewitch: Just how confident are you that the N equals 63 total size of the study is large enough to ameliorate any of those baseline differences?

Mohamed Genead: Yeah. Good point. Based on the prior data from the phase I/II, we saw treatment effect, small number of patient was N of nine. 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 the N of 63, it will be the full set of patient. The study was powered based on the early assumption to show such significant month 12. It was close to 90% powering. Also, we should see more homogenous population. The baseline characteristic should be balanced between the control and treated. That will also will drive a different value.

In addition, we have a longer time point, right? Month 12 versus month eight, and we saw the delta in our early study at month 12 versus month eight.

Michael Okunewitch: All right, thank you. 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 LARTA, 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? Could you just provide a bit more color on what that looks like?

Shankar Musunuri: Yeah, I'll let Abhi answer that. Go ahead.

Abhi Gupta: Yeah. So I think first on, the program is open globally. Yes, it's looking at the Bahama population, but much more broader. So it is truly like a global program. 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. 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 Ocugen to do.

Shankar Musunuri: Michael-

Abhi Gupta: Right.

Shankar Musunuri: –just to recap, anybody globally who is eligible, and they can go get the treatment at this stage under this approval.

Michael Okunewitch: All right. Would pricing match what you would be targeting for U.S. or international pricing, or is this more of an exploratory program?

Abhi Gupta: That is correct. It would be. Yeah, it would be.

Michael Okunewitch: Okay.

Shankar Musunuri: It would be matching U.S. price. Yeah.

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

Shankar Musunuri: Thank you.

Operator: Your next question comes from the line of Elemer Piros with Lucid Capital Markets. Your line is open.

Elemer Piros: 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?

Abhi Gupta: Yeah. As Ocugen, we don't market EAPs, but we are working with a local vendor that's responsible for all patient-related activities. They would be the one who are educating not only patients, but KOLs, including the community about this world's first option for a global EAP.

Elemer Piros: Okay. 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?

Mohamed Genead: DMC, as you know, it's an advisory body, and it's also independent, unblinded body assigned to the study. The DMC had a very clear per the protocol assignment. They were looking at, as part of the adaptive design, they were looking at three outcome. Again, it's not an efficacy outcome, it's just a part of the adaptive design for sample size recalculation or adjustment if needed. They had access to the unblinded data. They look at all the efficacy, including the primary, the lesion size, and all the other functional, including also the genetic correlation. So they were equipped to make a decision based on data.

As we said before, and this is important again to reiterate, the sample size was small, early time point, and also we had some heterogeneity at the baseline characteristics in that interim analysis. The good news also, remember they had opportunity now to do different outcome. They continue the study without modification because they believe with all full set of patient at month 12, the heterogeneity and all this baseline heterogeneity will be normalized at month 12. This is what we also we believe based on our data and also patient selection in the trial.

Elemer Piros: You can confirm that since you have all the baseline characteristics of the patients, that the full dataset is going to be well-balanced between the two treatment arms?

Mohamed Genead: That is correct.

Elemer Piros: Okay. Okay. Thank you very much. That is all what I had.

Operator: Your next question comes from the line of Leland Gershell with Oppenheimer & Co. Your line is open.

Leland Gershell: All right. Thanks for taking our questions. Just want to ask, I think it was asked earlier on the Stargardt, the eight-month analysis. Just to be clear, would 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–

Mohamed Genead: Go ahead.

Leland Gershell: –inform there. Thank you.

Mohamed Genead: No, it's a pleasure. No. The DMC is independent body. They have access to the unblinded data. We don't. The company study is still blinded, and all the team running the study is still blinded. If you're asking, will the DMC do another analysis at eight months with a full dataset? No. We are continuing the study till month 12, and this is where the time we're going to look at all the data from the trial as a company by month 12. This is part of the design of the study. This is a preexisting protocol. This is what we communicate to the agency.

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 I answer your question?

Leland Gershell: That's great. Okay. Yeah, that's very helpful. Thanks for clarifying. Just 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 OCU410ST, what the size of lesion reduction you'd expect versus comparator, and given this is a dynamic space, to be competitive here, what size reduction would you want to see, given the other therapies being developed in the space? Thank you.

Mohamed Genead: Yeah. The primary analysis for this study was based on the atrophic lesion size change over time till month 12. The study was powered close to 90% to look at that delta. The delta you're talking about, close to 25% delta. If remember, the phase I/II 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 patient, and it was our benchmark. But we went more conservative, and we talked about 25% delta between the treated and the control eyes to show statistical significance. That's on the lesion size growth.

We're also looking at other functional endpoint, including the BCVA and other functional endpoint in the study. We saw earlier in our phase I/II functional benefit as well from baseline to month 12, up to six letter gain in the treated eye versus the contralateral untreated eye. We believe, based on this earlier data, we should also see similar trend on the structure and functional improvement in these subjects.

Leland Gershell: That's very helpful. Thank you.

Operator: Your next question comes from the line of Whitney Ijem with Canaccord Genuity. Your line is open.

Whitney Ijem: 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 I/II experience, first of all, how were you defining responder in the phase I/II as it relates to the mobility testing, and what are the differences between how you're defining it there versus the phase III? Then remind us what was the responder rate, particularly in the RHO population in phase I/II, and remind us what percent of the phase III are RHO patients. That's part one, and then I have a follow-up. Sorry for so many questions.

Mohamed Genead: Yeah. Hi, Whitney. Thank you for your question. Regarding the phase I/II, what we saw from the phase I/II in the RP, the OCU400 program, this was N of 18 subject. Remember, we did some subgroup analysis. But what we saw based on the mobility test, and we used the same test externally used for their approval, which we called the Multi-Luminance Mobility Test, MLMT. What we saw in that study, in the treated eye who received OCU400, we saw 43% of the eyes did improve two lux level on the mobility test, which is the approval endpoint for RP, versus 0% of the control untreated eyes did show that.

When we projected this to our phase III mobility test, which we call the LDNA, which is very similar to the MLMT, but it's a broader range, 10 lux level versus six, but using the same methodology, the two lux level improvement. Our projection was, on that time, we went from 43% up to 71% improvement in the treated eye versus 0% in the control. So when we built our phase III assumption, Whitney, it was based on a very conservative analysis. We didn't say, "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. Remember, in the phase I/II, 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? If you look at this in the other way, right, if you look at the half 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 behaved in the phase I/II, which is close to 0%, give them 1%, we're still hitting 93% power.

So what we're saying here, a different way, we have a very high confidence with OCU400, especially the study is well-powered to show statistical significance at month 12. Using the two lux level improvement, the same one externally used for their approval. Did I answer your question, Whitney?

Whitney Ijem: Got it. That's really helpful. Yes, that's really helpful. Just on the RHO bit, am I remembering correctly that on the phase I/II, the responder rate in the RHO subgroup was higher? Either way, I guess, can you remind us what percent of patients in the pivotal are RHO patients?

Mohamed Genead: Yeah. So for the pivotal trial, the liMeliGhT, just to remind, it's 140 subject was enrolled, randomized to 2:1, 92 was treated, and 48 was untreated or the control arm. The rhodopsin we have, we have rhodopsin patient in the phase III trials, but the majority will be non-rhodopsin. We call it gene agnostic. But we have a very large number of patients in the phase III trials who have rhodopsin mutation. You're correct, we saw a very positive trend in the rhodopsin subpopulation in the phase I/II. So we are expecting to replicate, if not better, in our phase III trials.

Whitney Ijem: Got it. Excuse me. All right, that's helpful. Then, just 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 how quickly after enrollment was that final patient treated, 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?

Shankar Musunuri: 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.

Whitney Ijem: Got it. Okay. Thanks for the question.

Shankar Musunuri: Thank you.

Operator: I will now turn the call back over to Dr. Shankar Musunuri for closing remarks.

Shankar Musunuri: Thank you. Thank you for attending another good progressive earnings call today. Thanks to all our shareholders, patients, all our employees for supporting Ocugen. Thank you.

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

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