Ocugen - Earnings Call - Q3 2025
November 5, 2025
Executive Summary
- Q3 2025 advanced Ocugen’s late‑stage gene therapy portfolio: OCU410ST reached ~50% enrollment with EMA acceptance of a single U.S. trial for MAA, while OCU400 Phase 3 enrollment nears completion and rolling BLA submission is targeted for 1H 2026.
- Financial runway extended: $20M financing closed; cash/restricted cash was $32.9M at 9/30/25, supporting operations into 2Q 2026; potential $30M additional gross proceeds if warrants are fully exercised.
- Q3 results: revenue $1.752M, net loss $(20.051)M, and net loss/share $(0.07); OpEx rose to $19.4M (R&D $11.2M, G&A $8.2M), reflecting program progression and pre‑commercial build‑out.
- Business development catalyst: exclusive OCU400 license in South Korea with up to $7.5M upfront/near‑term milestones, $1.5M sales milestones per $15M, and 25% royalty; Ocugen to supply product.
What Went Well and What Went Wrong
What Went Well
- EMA endorsed single U.S. trial for OCU410ST MAA, simplifying EU pathway and preserving timeline/budget; enrollment ~50% with completion targeted in 1Q 2026.
- OCU400 Phase 3 nearing enrollment completion; rolling BLA to begin 1H 2026 and Phase 3 top‑line targeted for 4Q 2026. Quote: “We will begin rolling submission of the OCU400 BLA in the first half of 2026”.
- Manufacturing readiness improved. Quote: “Our PPQ… process validation runs are going very well… lots made ready to go”.
What Went Wrong
- Loss widened: Q3 net loss $(20.051)M vs $(12.970)M YoY; OpEx increased to $19.4M from $14.4M YoY as programs scale.
- Limited transparency on OCU410ST interim readout (mid‑2026) due to DSMB/agency process; management expects only “limited information publicly,” potentially reducing near‑term data visibility.
- Funding dependence remains: while runway extends into 2Q 2026, commercialization and Phase 3 initiations will require continued financing/partnering; warrants exercise is contingent.
Transcript
Operator (participant)
Good morning and welcome to Ocugen's Third Quarter 2025 Financial Results and Business Update. Please note that this call is being recorded at this time. All participant lines are in a listen-only mode. Following the speaker's commentary, there will be a question-and-answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin.
Tiffany Hamilton (Head of Corporate Communications)
Thank you, operator. Good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri, Ocugen's Chairman, CEO, and Co-Founder, who will provide a business update and an overview of our clinical and operational progress. Ramesh Ramachandran, our Chief Accounting Officer, is also on the call to provide a financial update for the quarter ended September 30, 2025. Dr. Huma Qamar, Chief Medical Officer, will be available to answer questions following the presentation. This morning, we issued a press release detailing associated business and operational highlights for the third quarter of 2025. We encourage listeners to review the press release, which is available on our website at ocugen.com. This call is being recorded, and a replay with the accompanying slide presentation will be available on the Investor section of the Ocugen website for approximately 45 days.
This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements include, but are not limited to, statements regarding our clinical development activities and related anticipated timelines. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations.
These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission, the SEC, including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this presentation speak only as of the date of this presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events, or otherwise, after the date of this presentation. Finally, Ocugen's quarterly report on Form 10-Q covering the third quarter of 2025 will be filed today. I will now turn the call over to Dr. Musunuri.
Shankar Musunuri (Chairman, CEO and Co-Founder)
Thank you, Tiffany. Thank you all for joining us today. I'm pleased to share an update on our modified gene therapy platform, and we'd like to recognize that in just over three years, we brought our lead candidate, OCU400, from initial Phase 1/2 dosing to nearing Phase 3 enrollment completion. The OCU410ST Phase 2/3 pivotal confirmatory trial is following close behind, and we're on track to complete enrollment in the first quarter of 2026, lining up for our planned biological licensing application, BLA submission, in the first half of 2027 for OCU410ST. This rapid progress is somewhat unheard of in industry and not only reinforces our commitment to file three BLAs in the next three years, but it also brings us closer to addressing the incredible unmet medical needs that exist for patients facing vision loss.
While all three programs are moving along on schedule, we received additional positive news in the third quarter that the Committee for Medicinal Products for Human Use, CHMP, of the European Medicines Agency confirmed the acceptability of a single U.S.-based trial for submission of MAA in Europe for OCU410ST. This alignment allows us to maintain the same timeline and budget efficiencies in Europe as we have with the OCU400 total trials. To fund clinical trial progress, we continue to pursue opportunities to increase our working capital and, in August, closed the registered direct offering with Janus Henderson. The gross proceeds were approximately $20 million, which we anticipate will extend our runway through the second quarter of 2026, and we will receive $30 million of additional gross proceeds if the warrants are exercised in full, extending our runway into 2027.
OCU400 Phase 3 liMeliGhT clinical trial remains on track for BLA and MAA submissions in 2026. It has the only broad retinitis pigmentosa, RP gene-agnostic trial to address multiple genetic mutations with a single therapeutic approach. It is important to note that this is the largest known Phase 3 orphan gene therapy trial. There are approximately 300,000 people in the U.S. and Europe combined living with RP, which affects more than 100 genes. Ocugen's gene-agnostic approach has the potential to treat multiple gene mutations associated with RP with a single one-time subretinal injection. Currently, the only approved gene therapy for RP targets a single gene, RPE65, which accounts for 1%-2% of the RP patient population. This product achieved peak sales of $52 million in 2023 with a patient population of approximately 2,000. We believe OCU400 has far greater commercial potential.
As it is intended to provide a therapeutic option for the remaining 98%-99% of RP patients. We anticipate commercialization in 2027. Process validation and manufacturing activities are progressing well in support of the BLA. Brand planning and marketing initiatives led by Abhi Gupta, our EVP of Commercial and Business Development, are scaling up as well. We will begin rolling submission of the OCU400 BLA in the first half of 2026. We will release Phase 3 top-line data in the fourth quarter of 2026 in line with our commitments. As we prepare for what will ultimately be a global rollout for OCU400, we are pursuing regional partnerships that preserve Ocugen's rights to larger geographies to maximize total patient reach while also generating return for our shareholders. In September, we announced an exclusive licensing agreement with Kwangdong Pharmaceutical Company Limited for the rights to OCU400 in South Korea.
Under the agreement, the company will receive up to $7.5 million in upfront and development milestone payments plus sales milestones of $1.5 million for every $15 million of sales in South Korea, projected to reach $180 million or more in the first 10 years of commercialization. We will also earn a 25% royalty on net sales generated by Kwangdong and will be responsible for manufacturing and supplying OCU400. There are an estimated 7,000 individuals in the Republic of Korea with RP, which represents approximately 7% of the U.S. market. OCU400 provides the opportunity for our partner to help thousands of patients facing vision loss. Upon regulatory approval of OCU400 in Korea, we believe Kwangdong will become a leader in the field of ophthalmic gene therapy in South Korea. Now, let's move on to OCU410ST.
OCU410ST has the potential to target over 1,200 pathogenic mutations in the ABCA4 gene associated with Stargardt disease and other ABCA4-related retinopathies with a single one-time subretinal injection. As I mentioned earlier, enrollment in the Phase 2/3 GARDian3 clinical trial is ahead of schedule. The strong response underscores the significant unmet medical needs among Stargardt patients who currently have no approved treatment options available. Stargardt disease affects approximately 100,000 people in the U.S. and Europe combined and approximately 1 million people globally. With CHMP acceptance of U.S. trial data for the MAA submission, we'll maximize resources and streamline development efforts with the goal of bringing OCU410ST to patients in Europe sooner than originally anticipated.
The 12-month data from all available phase one subjects showed highly encouraging results, with a 48.2% reduction in lesion growth and a meaningful one-line six-letter gain in visual acuity in available treated eyes compared with untreated eyes. All treated eyes also demonstrated stabilization or improvement in visual function, highlighting a consistent and tangible therapeutic benefit. Interim data from ongoing Phase 2/3 study is expected mid-2026, further advancing our goal of bringing OCU410ST to patients in need. Finally, OCU410 is specifically designed to address multiple pathways implicated in the pathogenesis of dry age-related macular degeneration and offers a promising advantage for current treatment options that target only one pathway, the complement system, which does not fully address the disease progression and underlying causes of vision loss. Currently approved treatment options require frequent intra-arterial injections, about 6-12 doses per year, and are accompanied by various safety risks.
For example, roughly 12% of patients develop wet AMD following treatment. With approximately 2 million-3 million geographic atrophy patients in the U.S. and Europe combined, OCU410 represents a significant market opportunity. Current therapies have notable limitations, and there are no treatments approved for GA in Europe, as existing FDA-approved options fail to demonstrate meaningful functional outcomes. OCU410 is therefore well-positioned to address this critical unmet medical need. At 12 months, available subjects in the Phase 1 study showed a 23% reduction in lesion growth, along with a two-line or 10-letter stabilization or gain in visual acuity in treated eyes. Preliminary results from six-month interim analysis demonstrated a 27% reduction in lesion growth and preservation of retinal tissue in the treated eyes when compared to untreated control eyes. This reduction is over twice that observed with currently approved intra-arterial therapies at six months, monthly, and every other month.
PEG cetacoplan injections, which showed only 13% and 12% reductions respectively, highlighting OCU410's potential to provide a significant and meaningful therapeutic benefit to patients with one-time treatment. In addition to the greater lesion reduction, a single subretinal injection of OCU410 demonstrates greater efficacy in preserving retinal tissue surrounding GA lesions compared with monthly and every other month PEG cetacoplan treatments. We plan to provide full 12-month data from the Phase 2 study, including both structural and functional outcomes in the first quarter of 2026, and anticipate initiating the Phase 3 study next year. I will now turn the call over to Ramesh Ramachandran to provide an update on our financial results for the quarter ended September 30th, 2025.
Ramesh Ramachandran (Chief Accounting Officer)
Thank you, Shankar. The company's cash, cash equivalents, and restricted cash totaled $32.9 million as of September 30th, 2025, compared to $58.8 million as of December 31st, 2024.
With the recent $20 million financing in the third quarter, we believe our current cash position provides sufficient runway to operate through the second quarter of 2026. Total operating expenses for the three months ended September 30th, 2025, were $19.4 million and included research and development expenses of $11.2 million and general and administrative expenses of $8.2 million. This compares to total operating expenses for the three months ended September 30th, 2024, of $14.4 million that included research and development expenses of $8.1 million and general and administrative expenses of $6.3 million. That concludes my update for the quarter. Tiffany, back to you.
Tiffany Hamilton (Head of Corporate Communications)
Thank you, Ramesh. We will now open the call for questions. Operator?
Operator (participant)
At this time, I would like to remind everyone, in order to ask a question, please press star, then the number one on your telephone keypad.
We'll pause for just a moment to compile the Q&A roster. Your first question comes from the line of Michael Okunewitch with Maxim Group. Please go ahead.
Michael Okunewitch (Senior Analyst of Biotechnology)
Hey, guys. Thank you so much for taking my questions today. Congratulations on all the progress.
Shankar Musunuri (Chairman, CEO and Co-Founder)
Thank you, Michael.
Michael Okunewitch (Senior Analyst of Biotechnology)
I guess to just start out, I'd like to ask a little bit about OCU400 and, in particular, the timing of the BLA completion. You mentioned that you're going to be starting that in the first half of 2026, the rolling BLA. Then how quickly do you believe that you can turn around the pivotal data over to the FDA and complete that filing?
Shankar Musunuri (Chairman, CEO and Co-Founder)
As soon as it cuts in. I think, I mean, we're nearing completion, as we stated. We will have resources ready to turn around in weeks.
Michael Okunewitch (Senior Analyst of Biotechnology)
All right. That's great to hear.
In terms of your manufacturing, are there any other items that you need to do to get ready for commercial manufacturing before you can start submitting that and start the rolling BLA process?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. Good question. Our PPQ, our process validation runs are going very well. They're on target. In fact, all the material we're making in support of the registration, they can be commercialized. So we will have lots made ready to go.
Michael Okunewitch (Senior Analyst of Biotechnology)
All right. Thank you. And then just one more from me, and I'll hop back into the queue. For 410ST, with the upcoming interim readout, what endpoints are you expecting to release? Or is that going to be an internal DSMB review?
Shankar Musunuri (Chairman, CEO and Co-Founder)
I mean, for endpoint for the Phase 2? Yes. It's already in the clinical protocol. We're looking at lesion growth compared to untreated control group. We do have untreated control group.
The secondary, we're looking at visual acuity.
Michael Okunewitch (Senior Analyst of Biotechnology)
I'm specifically referring to the interim release that you're expecting mid-year 2026. Will that be publicly released, or is that going to be internal?
Shankar Musunuri (Chairman, CEO and Co-Founder)
That's a good point. Yeah. There'll be limited information publicly. The DMC looks at it, and then we'll be informing the agency. However, we will give you some indications about the clinical trial.
Michael Okunewitch (Senior Analyst of Biotechnology)
All right. Thank you very much. I appreciate the update. It looks like there's a lot to look forward to at Ocugen.
Shankar Musunuri (Chairman, CEO and Co-Founder)
Thank you.
Operator (participant)
Your next question comes from the line of Boris Peaker with Titan Partners. Please go ahead.
Boris Peaker (Managing Director)
Good morning. A couple of questions for me. Maybe let's start with the 400. Can you just remind us exactly what the statistical kind of design of the liMeLiGhT study is, what the assumptions were?
I'm just curious if you've had kind of recent discussion with the FDA to make sure that it's still acceptable. I think this question comes in a context of what we've seen just recently with uniQure, where it sounds like the FDA may have moved the goalpost a little bit in their gene therapy. I think we just want to get a sense to make sure that a similar dynamic doesn't happen here.
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. Good question. I'll start, then Huma will chime in. First and foremost, I want to distinguish all our clinical trials, including our Phase 2 GA trials. We have a control arm within the study. Typically, FDA, that has been a tradition. Most of the clinical trials, FDA really looks for control within the study, not using some external controls or very rarely natural history.
I think I really want to differentiate that from what you mentioned about uniQure. We do have our OCU400, OCU410ST. Total trials, including even the OCU410 GA clinical trial, we have untreated control subjects in the clinical trial. That is what we are using to compare. Huma, go ahead on the clinical trial design.
Huma Qamar (Chief Medical Officer)
Yeah. Thank you, Shankar. The clinical trial design is we have 150 subjects that we are planning to enroll for the study, 100 in the treatment and 50 in the control, 75 in the rhodopsin, and 75 in the gene-agnostic arm, with 50 being active and 25 in the control. We have 97% power for this study. We have assumed 50% and 10% treatment effect in the treated and the untreated eyes. There is a two-in-one randomization, which means there is.
We are treating both the eyes as they meet the inclusion-exclusion criteria, with the worst eye being dosed first. This is the only trial that is globally available with clinical and/or genetic diagnosis of RP with syndromic and covering non-syndromic forms.
Boris Peaker (Managing Director)
Got it. That's very helpful. Maybe a question on OCU200. You mentioned a milestone that we'll see completion of enrollment by the end of the year in Phase 1. Can you comment when we'll see the data and what would that initial data include?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. You're good.
Huma Qamar (Chief Medical Officer)
Yes. We are on track for our OCU200 enrollment for our Phase 1. Our periodic safety updates are there. There are no serious adverse events related to the product listed as of right now.
Yes, in the beginning of next year, we will be providing some more updates on the safety and efficacy of OCU200 as soon as it becomes available. As this is a Phase 1, first in human, there are no serious adverse events related to the investigational product reported as of right now.
Boris Peaker (Managing Director)
How many patients will you have in that initial update?
Huma Qamar (Chief Medical Officer)
We have a dose-ranging, dose-escalation portion of the study. That would be almost 9-12 subjects.
Boris Peaker (Managing Director)
Great. Thank you very much for taking my questions.
Operator (participant)
Your next question comes from the line of Swayampakula Ramakanth with H.C. Wainwright. Please go ahead.
Swayampakula Ramakanth (Managing Director and Senior Equity Analyst)
Thank you. Good morning, Shankar and team. A couple of quick questions. The first one on OCU400. As you're nearing to the point where.
You're putting not only your application together but also thinking about commercialization, what sort of investments are you making within the commercial infrastructure so that you're allocating appropriately within your budget for pre-commercial activities? Yeah. Let me start with that.
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. Good morning, RK. Thank you. Yeah. I mean, based on 2027 launch, next year, we will be ramping up slowly for U.S. commercialization. Obviously, our goal is to continue to look for partnerships. Just like we announced one in South Korea and other regional partnerships in Europe, Japan, and elsewhere. U.S., we are looking still into because it's a good market and we have good handle. We created a lot of these centers for excellence, which will be part of the commercialization for subretinal delivery. Therefore, we will have more information provided next year, but we'll slowly ramp up. We'll be allocating carefully limited budget starting with.
We're looking into other venues to beef that up next year.
Swayampakula Ramakanth (Managing Director and Senior Equity Analyst)
Okay. On the ST, with the comment that it's reaching 50% enrollment on the GARDian3 trial, what's the cadence of enrollment there? Are you having, just trying to understand how the enrollment is going. Are there any geographies where you're seeing less of an enrollment? If so, how many patients you need to, you're screening so that you get the enrollment moving?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. I'll let Uma take that. Huma, go ahead.
Huma Qamar (Chief Medical Officer)
Thank you. Thank you, Shankar. Okay. This trial includes 51 subjects, 34 in the treatment, 17 in the control. We are almost 50% and above in our enrollment. We have completed that, and we are on track. There are no geographical restrictions here. Actually, I would say.
This is basically the disease of the pediatric population, and we are covering early to late stages of Stargardt, also three years of age and above. That's why we are pretty confident we are going to meet the enrollment goal. In fact, we do have almost half of what we have enrolled, more than that, in the screening queue. We are on track for completion of our enrollment. There are no geographic restrictions. There are almost 15 centers that we have equally covered across in the U.S., and all of them have the screening metrics for those patients as well. We are on track.
Swayampakula Ramakanth (Managing Director and Senior Equity Analyst)
Shankar, if I may, can I ask one quick one on OCU400 itself? Having looked at the data from the earlier studies, on the patient characteristics, have you folks identified any of the patients?
Obviously, there are some who are high responders and some who are non-responders. In general, how are you thinking about what the real potential is for OCU400? And what could your commercial strategy be so that you try to get the highest adoption that you could get?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. So, RK, I mean, our design clearly outlines based on the strong scientific basis. We're targeting entire RP from early stage to advanced, pediatric to adult. And that's the coverage. I mean, we are including in our Phase 3 clinical trial all major components of RP. That means some of the mutations with high prevalence, like rhodopsin or XLRP, RUSHERS, and PDE6B. All those mutations are included with good representation. Our goal is to look at the data holistic and get the broad RP indication so that we provide treatment potentially for all RP patients, not.
Restricted to specific genotype.
Swayampakula Ramakanth (Managing Director and Senior Equity Analyst)
Thank you.
Shankar Musunuri (Chairman, CEO and Co-Founder)
The market will be wide open. Yeah. Go ahead. Okay. Please go ahead.
Swayampakula Ramakanth (Managing Director and Senior Equity Analyst)
No, no. Thanks. That's what I said. Thanks for taking my question.
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. Thanks. Yeah. Yep.
Operator (participant)
Your next question comes from the line of Robert LeBoyer with Noble Capital. Please go ahead.
Robert LeBoyer (Senior Life Sciences Analyst)
Good morning. Just as a follow-up from some of the previous questions, my understanding of OCU400 is that it's a regulatory gene that affects the entire gene network, downstream restoring homeostasis. Early stage, late stage, child, adult, specific mutations really wouldn't matter because it's affecting the whole downstream pathway that leads to blindness. Please let me know if that is the correct way to think about it.
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. Absolutely, Robert. You stated it.
Robert LeBoyer (Senior Life Sciences Analyst)
Okay. Secondly, the OCU410 in GA is also ongoing.
Could you just go over some of the endpoints and what to expect in that trial?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yes. Huma?
Huma Qamar (Chief Medical Officer)
Yes. Yes. Hi, Robert. We have completed our ArMaDa enrollment Phase 1/2. The Phase 1 was a typical dose-ranging, dose-escalation, 3+3 design, nine subjects there. Also, we had Phase 2 where we had a high dose, medium dose, and a control, which means that we enrolled 17 subjects in high dose, 17 in the medium dose, and 17 in the control. In terms of the endpoints in Phase 2/3, which is important, it is a geographic atrophy lesion growth reduction from baseline and also looking at low luminance visual acuity over the period of time as well, which is functional. We have been consistently releasing the data on that, and we are on track for following up those patients till early part of next year.
Shankar Musunuri (Chairman, CEO and Co-Founder)
That's a one-year time point, Robert, to clarify. Our GA trial is a one-year trial.
Robert LeBoyer (Senior Life Sciences Analyst)
Okay. Great. Thank you very much for that detail.
Shankar Musunuri (Chairman, CEO and Co-Founder)
You're welcome.
Operator (participant)
Your next question comes from the line of Elemer Piros with Lucid Capital Markets. Please go ahead.
Elemer Piros (Managing Director)
Yes. Good morning. Shankar, you talked about OCU410ST and your agreement. No, I think it, yes, OCU410ST, your agreement with the European agency that the U.S. trial would be sufficient. Do you have a similar agreement related to the RP program as well?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yes. Yeah. We have a similar agreement with EMA on the RP program too. Yeah.
Elemer Piros (Managing Director)
Okay. What would be the regulatory path in South Korea for 400?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. Currently, it looks like South Korea and the rest of the world, typically for orphan gene therapies, they're following FDA closely.
Once you get FDA approval, based on the FDA approval, you get approvals in those countries. Most of these countries do not need any additional clinical trials.
Elemer Piros (Managing Director)
Yeah. I understand. One housekeeping question. How much of the $7.5 million or up to $7.5 million that you received from Kwangdong is included in the cash balance that you reported?
Ramesh Ramachandran (Chief Accounting Officer)
The $7.5 million is not included in the cash balance at this point of time. It is going to be in the future. That is not part of our cash balance at this point of time.
Elemer Piros (Managing Director)
Could they project some into the fourth quarter in terms of the initial payment?
Ramesh Ramachandran (Chief Accounting Officer)
The $1 million which we received from Kwangdong, that is already in the cash projection, but nothing more than that.
Elemer Piros (Managing Director)
Okay. $1 million. Okay.
Lastly, could you talk about your manufacturing capacity, Shankar, especially when we think about much larger indications such as OCU410 for geographic atrophy?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. The manufacturing capacity, we have an ex-U.S. partner with plenty of capacity. The goal is to, I mean, we have our own facility in our backyard in Malvern, Pennsylvania. That facility will be ready. Our plan is to get that ready by 2027. When we get the first approval, it's called a prior approval supplement with the U.S. FDA. We have to add the site. Our goal in the future is to produce all U.S. supply from our U.S. manufacturing site.
Elemer Piros (Managing Director)
You mentioned also that you have an ex-U.S. partner as well, manufacturing partner?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yes. That's right. Yeah. Currently, yeah. We have plenty of capacity, global capacity.
Elemer Piros (Managing Director)
Okay. Thank you so much.
Operator (participant)
Final question comes from the line of Daniil Gataulin with Chardan. Please go ahead.
Daniil Gataulin (Senior Research Analyst)
Yes. Hi. Good morning, guys. Thank you for taking my questions. I have a couple here. First, on OCU400. The trial. As we know, you have two arms, one with RHO mutations, one with RP gene-agnostic mutations. For the gene-agnostic arm, are you disclosing how many different mutations are included there? In terms of the enrollment, did one arm see a more robust enrollment than the other?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Go ahead.
Huma Qamar (Chief Medical Officer)
Thank you. Thanks for the question. Actually, it's an assessor-blinded study. At this point, we cannot disclose. Gene-agnostic means that all the major mutations will be covered, which is more than 95% geographically looked at in the U.S. and globally.
We are going to cover that in the gene-agnostic, and all forms of rhodopsin are going to be in the rhodopsin arm. That is the first one. Yes, there is a robust enrollment randomization that we are proceeding with, as we stated, clinical and/or genetic diagnosis, syndromic as well as non-syndromic forms.
Daniil Gataulin (Senior Research Analyst)
Okay. Got it. In terms of prep for BLA filing for OCU400, what are the outstanding CMC, or what is the outstanding CMC work that needs to be completed before you file?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. With the RMAT designation. Daniil, we have an opportunity to initiate rolling submission, as we stated, in the first half of next year. You can submit non-clinical and CMC sections. As far as CMC is concerned, we have to complete our process validation runs for drug substance and drug product. It is progressing really well.
We're on target. Those sections will be submitted before we submit the clinical section. That will be the last one late next year. Somewhere in the middle of next year, we'll be submitting the manufacturing section. We'll start with non-clinical, followed by CMC, followed by clinical.
Daniil Gataulin (Senior Research Analyst)
Okay. Got it. Understand. One last question for LCA. Is that on hold for now, or is that completely off or out of your pipeline, or what are your plans for LCA?
Shankar Musunuri (Chairman, CEO and Co-Founder)
Yeah. From a market perspective, it's very small. What we'll do is, right now, it's not in our plan. Obviously, once the RP gets into the market, we can always look into that for Phase 4.
Daniil Gataulin (Senior Research Analyst)
Okay. Got it. All right. Thank you very much for taking my questions.
Operator (participant)
This concludes the Q&A portion.
I will now turn the call back over to Chairman, CEO, and Co-Founder, Dr. Shankar Musunuri. Please go ahead.
Shankar Musunuri (Chairman, CEO and Co-Founder)
Thank you, Operator. The third quarter was marked with important clinical progress, strategic business development, and essential financing accomplishments. We're poised to close 2025 on a strong note and look forward to early 2026 catalysts that will further advance Ocugen's position as a biotechnology leader in gene therapy for blindness diseases. Have a great day. Thank you.
Operator (participant)
Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.