Ocugen Q4 2025 Earnings Report

Ocugen EPS Results

• Actual EPS: -$0.06
• Consensus EPS: -$0.06
• Beat/Miss: Met Expectations
• One Year Ago EPS: N/A

Ocugen Revenue Results

• Actual Revenue: ($0.19) million
• Expected Revenue: $0.86 million
• Beat/Miss: Missed by -$1.05 million
• YoY Revenue Growth: N/A

Ocugen Announcement Details

• Quarter: Q4 2025
• Date: 3/4/2026
• Time: Before Market Opens
• Conference Call Date: Wednesday, March 4, 2026
• Conference Call Time: 8:30AM ET

Ocugen (NASDAQ:OCGN) Inc is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing gene therapies to treat rare inherited retinal diseases, as well as vaccines designed to address unmet needs in infectious diseases. Headquartered in Malvern, Pennsylvania, the company applies its proprietary gene therapy platform to create novel treatments aimed at preserving and restoring vision, while leveraging strategic partnerships to broaden its vaccine pipeline.

In its gene therapy portfolio, Ocugen is advancing multiple programs targeting retinal disorders. Lead candidates include OCU400 for retinitis pigmentosa and OCU410 for wet age-related macular degeneration, each engineered to deliver functional genetic material to retinal cells. The company’s approach is rooted in adeno-associated viral (AAV) vector technology, which has the potential to enable single-administration treatments and durable therapeutic benefits for patients facing progressive vision loss.

On the vaccine front, Ocugen entered into a collaboration with India-based Bharat Biotech in mid-2020 to co-develop and commercialize the COVID-19 vaccine Covaxin in the United States and Canada. This partnership illustrates Ocugen’s ability to partner with established developers to bring critical vaccine candidates into North American regulatory pathways. The company is also exploring additional vaccine opportunities that may leverage its manufacturing and distribution networks.

Founded in 2014, Ocugen is led by President and Chief Executive Officer Dr. Shankar Musunuri, who co-founded the company with the goal of addressing conditions with limited or no treatment options. Under his leadership and that of an experienced management team, Ocugen continues to advance its pipeline through clinical trials and regulatory interactions, with a mission to deliver innovative therapies for patients worldwide.

Ocugen Q4 2025 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: Enrollment is complete for the OCU400 Phase 3 liMeliGhT trial (140 patients); top-line 12‑month data are expected in Q1 2027 and a rolling BLA submission is planned to begin in Q3 2026 with commercialization targeted in 2027.
• Positive Sentiment: Interim Phase 2 data for OCU410 in geographic atrophy showed a 46% reduction in lesion growth at 12 months (P=0.015) and a 50% responder rate, with full Phase 2 data due this month and Phase 3 initiation planned for 2026.
• Positive Sentiment: The Stargardt program (OCU410ST) remains ahead of schedule, benefited from a peer‑reviewed Phase 1 publication and CHMP confirmation that the single U.S. trial can support an EMA application, with pivotal topline data expected in 2Q 2027.
• Negative Sentiment: Cash on hand extends runway only into Q4 2026 after a recent $22.5M raise, with an extension to Q2 2027 contingent on full exercise of $30M of warrants, indicating near‑term financing risk before commercialization.
• Positive Sentiment: Management and business development were strengthened in 2025 (new commercial, finance, and operations hires), plus a regional licensing deal for OCU400 in Korea and the creation of the Ocucelix subsidiary to pursue non‑core assets, supporting commercialization and value‑creation plans.

Presentation

[Operator]

Good morning, and welcome to Ocugen's fourth quarter and full year 2025 financial results and business update. All participant lines are currently in listen-only mode. Following the speakers' 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 at Ocugen]

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 and full year ended December 31st, 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 fourth quarter and full year 2025. 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 accompanying slide presentation, will be available on the investor section of the Ocugen website.

[Tiffany Hamilton, Head of Corporate Communications at Ocugen]

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, 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 outcomes to differ materially from those expressed or implied. We encourage you to review our filings with Securities and Exchange Commission, including the risk factors detailed therein, for a more comprehensive understanding of these potential risks. Finally, Ocugen's annual report on Form 10-K, covering the full year 2025, will be filed today. I will now turn the call over to Dr. Musunuri.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Thank you, Tiffany. Thank you all for joining us today. I'm pleased to share an update on what was a transformative year for Ocugen. Considerable development across all of our modifier gene therapy programs, including licensing and financing agreements to strengthen our financial position and meaningful appointments to our leadership team made in 2025 a year of real momentum for Ocugen. We are now poised to leverage upcoming catalysts and advance the business as we near the first of our three BLA filings. I'm proud of what this team has accomplished, and I'm confident that with a full bench of experienced leadership across the organization, we have the resources and the know-how to drive Ocugen's transition into a commercial stage company. Let me walk you through each program, starting with OCU400 and retinitis pigmentosa, which I will refer to as RP going forward.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

It is important to note that the phase III liMeliGhT clinical trial is the only broad RP gene agnostic trial and the largest known phase III orphan gene therapy trial. Approximately 300,000 people in the U.S. and Europe are living with RP is caused by mutations in more than 100 genes. OCU400 is designed as a modifier gene therapy utilizing NR2E3, a central transcriptional regulator of retinal specific pathways, to address multiple genetic mutations with a single one-time treatment. The only approved gene therapy for RP today targets a single gene, RPE65, which accounts for just 1%-2% of the total RP patient population. We believe OCU400 has significantly wider commercial potential as it is intended to provide a therapeutic option for 98%-99% of all RP patients.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

I'm pleased to report that enrollment is now complete for the OCU400 phase III liMeliGhT trial. As a one-year clinical trial, top-line data will be available in the first quarter of 2027. These data are anticipated to support the Biologics License Application, BLA, filing for OCU400 and potential approval in 2027. The liMeliGhT clinical trial enrolled 140 patients who were randomized two to one into the treatment group and untreated control group across mutations, including RHO and gene-agnostic arms. The gene-agnostic arm includes many genetic mutations, including those most prevalent: TULP1, CRX, XLRS, USH2A, XLRP, and PDE6B. The target population included patients with early to late stage disease among a broad RP population, including pediatrics. The primary endpoint is 12-month change in visual function assessed by LDNA, Luminance Dependent Navigation Assessment, with improvement in lux level from baseline to 12 months.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

We also released positive long-term three-year phase I/II data for OCU400 that builds on our prior two-year results. The data demonstrates sustained, clinically meaningful approximately two-line LLVA gain, reinforcing durable gene agnostic benefit. OCU400 maintained a favorable durability, safety, and tolerability profile with the no new treatment-related serious adverse events or adverse events of interest emerged. With enrollment complete and these strong long-term data in hand, we are on track to begin the rolling BLA submission in the 3rd quarter of 2026. Process validation and manufacturing activities are progressing well in support of the timeline. Plant planning and marketing initiatives are scaling up as well. We anticipate commercialization in 2027 in line with our commitments.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

As we prepare for what will ultimately be global rollout of OCU400, we are pursuing regional partnerships that preserve Ocugen's right to larger geographies while also generating near-term value for our shareholders. In 2025, we executed our first regional licensing agreement with Kwangdong Pharmaceutical Company Limited for the exclusive Korean rights OCU400. With upfront fees and near-term development milestone payments along with royalties, this was a valuable collaboration for Ocugen and a critical step in the company's business development strategy. There are an estimated 7,000 individuals in the Republic of Korea with RP, equal to approximately 7% of addressable U.S. RP market. This approach allows us to maximize total patient reach while retaining full commercial rights in the U.S. and Europe. Now, let's move on to OCU410ST for Stargardt disease.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

OCU410ST holds the potential to target over 1,200 pathogenic mutations in the ABCA4 gene associated with the Stargardt disease and other ABCA4-related retinopathies with a single one-time treatment. Stargardt disease affects approximately 100,000 patients in the U.S. and Europe combined, and approximately one million people globally with no approved treatment options available. The phase II/III GARDian3 pivotal confirmatory trial remains ahead of schedule. We anticipate top line phase II/III data in the 2Q 2027, followed by the BLA submission. In January, we announced the peer-reviewed publication of our phase I GARDian trial results in Eye, which supports the favorable safety, tolerability, and efficacy profile of OCU410ST and its potential to provide clinically meaningful functional and structural benefits in Stargardt patients. This independent validation further strengthens the scientific foundation supporting the ongoing pivotal trial.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Importantly, the Committee for Medicinal Products for Human Use, CHMP, of the European Medicines Agency confirmed that data from our single U.S.-based trial can also support an EMA application. This alignment allows us to maintain the same timeline and budget efficiencies in Europe as we have with this OCU400 pivotal trial, streamlining our development efforts and bringing OCU410ST to patients in Europe sooner than originally anticipated. The program has also received Rare Pediatric Disease Designation, further strengthening its regulatory positioning. I would like to explain Ellipsoid Zone, EZ analysis in greater detail, as this is now an exploratory endpoint for both the GARDian3 and ArMaDa clinical trials. The Ellipsoid Zone is a hyperreflective band representing photoreceptor inner and outer layer segmentation. It indicates photoreceptor health and is a biomarker for photoreceptor structural integrity and metabolic health.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

EZ disruption precedes RPE loss and visible atrophy in geographic atrophy and Stargardt disease. EZ measurement is important because it provides early and sensitive detection. EZ changes occur before visible RPE atrophy expansion in GA and Stargardt progression. It also enables earlier intervention and more sensitive treatment effect detection in GA and Stargardt. Finally, EZ correlates to earlier functional therapeutic benefit with an effect as early as one year compared to other measures such as visual acuity with clinically meaningful effect at two years or more. Since all of our clinical trials aim to demonstrate benefit at one year, and we are targeting significant unmet medical needs, EZ is a relevant measure to show functional outcome in these trials. As EZ analysis has been established as a clinically relevant endpoint for dry AMD clinical trials, it was critical to incorporate this measure for both our Stargardt and GA trials.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

As shown in this bar graph, change from baseline at 12 months in treated fellow eyes across doses, excluding two subjects lost to follow-up and one subject with retinal detachment, demonstrated mean of 116% in lesion reduction in available treated eyes relative to untreated eyes. Specifically, 50% of OCU410ST treated eyes achieved EZ preservation exceeding expected disease decline or atrophy progression at 12 months. This change from baseline structural preservation on spectral domain OCT quantified as 116% lesion reduction and ellipsoid zone integrity highlights meaningful photoreceptor protection and functional therapeutic benefit in Stargardt disease, underscoring the key differentiator of modifier gene therapy. Let's turn to OCU410 in GA secondary to late stage dry AMD.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

With approximately 2 million-3 million GA patients in the U.S. and Europe combined, OCU410 represents a significant market opportunity. OCU410 is specifically designed to address multiple pathways implicated in the pathogenesis of dry age-related macular degeneration and offers a promising advantage over current treatment options that target only one pathway, the complement system. Currently approved treatment options require frequent intravitreal injections, about 6-12 doses per year, and are accompanied by various safety risks. For example, roughly 12% of patients develop vitritis following treatment. There are no treatment approved for GA in Europe, and existing FDA-approved options have failed to demonstrate meaningful functional outcomes. OCU410 is therefore well-positioned to address this critical unmet need. In January, we announced positive preliminary 12-month data from approximately 50% of patients evaluated to date in the phase II ArMaDa clinical trial evaluating OCU410.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

The key findings were compelling. We observed a 46% reduction in lesion growth at 12 months across the medium and high-dose groups combined versus control, with statistical significance at P of 0.015 in a cohort of 23 patients. We also saw a 50% responder rate, with patients achieving greater than 50% lesion size reduction versus control. To put this in context, currently marketed products have demonstrated only a 22% lesion reduction at two years. At one year, OCU410 is already delivering more than double the benefit seen with existing therapies at twice the time. A subgroup analysis of patients with baseline GA size of 7.5 mm square or greater, representing advanced atrophy, demonstrated a 57% reduction in lesion growth in treated eyes for the medium dose and a 56% reduction for the high dose compared with control.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

This suggests OCU410 may be even more effective in patients with substantial disease burden. The dataset also included encouraging 12-month phase I findings, where OCU410-treated eyes demonstrated 60% slower loss of the ellipsoid zone or EZ compared to untreated fellow eyes. The 60% reduction in EZ loss rate indicates that OCU410 treatment is substantially slowing the rate of photoreceptor degeneration compared to the natural history observed in the untreated fellow eyes. We look forward to reporting the complete dataset from the OCU410 phase II ArMaDa trial this month and anticipate initiating phase III in 2026. Let me also provide a brief update on our other programs. For OCU200, no serious adverse events or adverse events related to OCU200 have been reported to date across the phase I dose escalation cohorts, and trial enrollment is expected to be completed in the first quarter of 2026.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Regarding our inhaled vaccine candidate, OCU500, NIAID intends to initiate the phase I clinical trial in the second quarter of 2026. Finally, we created Ocucelix as a wholly-owned subsidiary for our regenerative cell therapy assets, including NeoCart, with the goal to be independent through financing that will maximize value for Ocugen shareholders and patients. We will provide further details as the process progresses. Across the portfolio, 2026 represent multiple defined inflection points. These include completion of enrollment for OCU410ST in early 2026, full phase II data for OCU410 this month, interim pivotal data for OCU410ST in the third quarter, initiation of phase III for OCU410 in 2026, and start of rolling BLA submission for OCU400 in the third quarter. Each of these milestones builds towards longer-term regulatory and commercialization objectives and reinforces our commitment to file three BLAs in the next three years.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Operationally, we also strengthened our executive leadership team with several appointments, including Abhi Gupta to Executive Vice President, Commercial and Business Development, bringing more than 20 years of experience across commercial strategy, gene therapy, and corporate development in the biopharmaceutical industry. Recently, Rita Johnson-Greene was named Chief Financial Officer. Rita's experience in financial strategy and capital planning supports our continued focus on disciplined resource allocation as our programs advance toward late-stage development and potential commercialization. Just this week, Paul Slade joined us as Executive Vice President, Operations. Paul has more than 20 years of leadership experience in biologics and cell and gene therapy technical operations. He joins from Bristol Myers Squibb, where for over 16 years, he held leadership roles in manufacturing launch, scale-up, orchestration of reliable global supply chains with a CAR T focus for the last five years.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

He will lead operations to strengthen execution and support the company's transition toward regulatory approvals and commercialization. I will now turn the call over to Rita Johnson-Greene to provide an update on our financial results for the quarter and full year ended December 31st, 2025. Rita.

[Rita Johnson-Greene, CFO at Ocugen]

Thank you, Sankar. I am thrilled to join the Ocugen team and support the company through its imminent transitions into a commercial enterprise. Starting with our fourth quarter results, research and development expenses for the three months ended December 31st, 2025 were $10.7 million compared to $8.3 million for the three months ended December 31st, 2024. General and administrative expenses for the three months ended December 31st, 2025 were $6.1 million compared to $6.3 million for the three months ended December 31st, 2024. Ocugen reported a $0.06 net loss per common share for the three months ended December 31st, 2025 compared to a $0.05 net loss per common share for the three months ended December 31st, 2024.

[Rita Johnson-Greene, CFO at Ocugen]

For the full year ended December 31st, 2025, research and development expenses were $39.8 million compared to $32.1 million for the full year ended December 31st, 2024. General and administrative expenses were $27.6 million compared to $26.7 million for the prior year. Ocugen reported a $0.23 net loss per common share for the year ended December 31st, 2025 compared to a $0.20 net loss per common share for the year ended December 31st, 2024. Our current cash and cash equivalents extend our runway into the fourth quarter of 2026. This includes the recent raise of $22.5 million through an underwritten registered direct offering of common stock led by RTW Investments.

[Rita Johnson-Greene, CFO at Ocugen]

In addition, if the $30 million in warrants from the prior Janus Henderson raise are exercised in full, it will extend cash runway into the second quarter of 2027. That concludes my financial update. Shankar, back to you.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Thank you, Rita. We'll now open the call for questions. Operator?

[Operator]

Thank you. If you'd like to ask a question, please press star and one on your telephone keypad to raise your hand and enter the queue. If you'd like to withdraw your question or your question has been answered, please press star and one again. We will be taking our first question from Michael Okunewitch from Maxim Group. Please go ahead.

[Michael Okunewitch, Senior Research Analyst at Maxim Group]

Hey, guys. Thank you so much for taking my questions today. Congrats on all the great progress you've made. I guess to start off, just given that it is a 12-month primary endpoint for the liMeliGhT study, how confident are you in the ability to turn around the data from that, from when you hit on that top-line endpoint to actually releasing the top-line data within first quarter 2027?

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Huma?

[Huma Qamar, CMO at Ocugen]

Thank you for the question. We are very confident that we will be able to hit our timeline.

[Michael Okunewitch, Senior Research Analyst at Maxim Group]

All right. Just for that endpoint, could you just remind us some of the modifications that you made for that particular navigation assessment course and why you decided to go with a primary metric for RP?

[Huma Qamar, CMO at Ocugen]

In terms of the mobility test that we are using proprietary to Ocugen, that's Luminance Dependent Navigation Assessment. It's the mobility test that was approved as the primary endpoint for Luxturna, that was called MLMT at that time. That was only designed for RPE65 mutation that covers only 1%-2% of the RP landscape. This is a very sensitive and specific test. As you can see that this is the broad RP indication trial covering all clinical, syndromic, non-syndromic. All the genetic mutations that cause RP are included. This has uniform lux levels and intensity and lux levels from 0-9, and that has the ability to capture the change in real time, which is from the baseline up to 52 weeks. This was also aligned with FDA.

[Huma Qamar, CMO at Ocugen]

It's a validated, test as well as, this was approved by FDA and the only test that can capture the real change with functional outcome, improving the functional outcome or demonstrating the functional outcome in these mutations. Just to let you know, we are the only trial globally, that is covering all the majority of the gene agnostic mutations as we have covered this morning in one of our slides as well. Thank you.

[Michael Okunewitch, Senior Research Analyst at Maxim Group]

Thank you. Certainly very helpful. Last one before I jump back into the queue. For the Stargardt program, it's looking like there could be an approval from another company for a chronic therapy by the time you file for OCU410ST. I wanted to know how this might impact the opportunity or pricing potential and if there's any reason that OCU410ST couldn't be complementary with other therapies as they come to market.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Yeah, I'll take that. There are other therapies out there. Obviously what we have shown, if you look at the data we published in Eye, in one year, again, I wanted to restate all our trials, we're able to show treatment benefit in one year, unlike other trials out there, two years or more. The data we showed in one year is compelling. It looks superior. Also, our goal is to show also functional benefit. With the gene therapy, we're targeting the major pathways which are complex in Stargardt and GA. With the RORA gene. Also we have ability to reset the homeostasis and bring, you know, make sure we create a healthy environment for retinal cells to survive. That's very important factor. We're not just trying to slow down the disease progression.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

We're working on the, in our genes have ability to control the entire network. There is a big difference. Also this is one and done. If you have a one-and-done therapy, this will set up the standard of care. We're not worried about other therapies if they come to the market first. It's good. Those therapies will educate the market, and they'll create a market education and everything else. We will come back and then we may be, you know, behind them, but it's okay because what we believe, we are going to set the standard of care for Stargardt patients globally. Huma?

[Huma Qamar, CMO at Ocugen]

Yes. Thank you, Shankar. Very well said. That, I would like to add that this is the trial that we are also having the population 3 years of age and above, versus the other trials that are very limited in the age population. Also, the inclusion/exclusion criteria is very globally representing. Other than that, this is the OCU410ST is targeting early to advanced cases of Stargardt disease. If you look at in the comparison, the safety and tolerability and efficacy that we have seen in terms of lesion growth reduction and also the functional and structural outcomes has been trending in the right direction and promising from the clinical standpoint as well.

[Michael Okunewitch, Senior Research Analyst at Maxim Group]

Thank you. It's certainly an exciting time for the space and looking forward to any further updates that you have.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Thank you.

[Huma Qamar, CMO at Ocugen]

Down there.

[Operator]

Thank you. Our next question comes from the line of Boris Peaker from Titan Partners. Please go ahead.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Boris?

[Operator]

Mr. Peaker, you might be on mute.

[Boris Peaker, Managing Director and Senior Equity Research Analyst at Titan Partners Group]

Hear me? Sorry.

[Operator]

Um.

[Boris Peaker, Managing Director and Senior Equity Research Analyst at Titan Partners Group]

Perfect. for the OCU400, for the rolling BLA, when would we get the FDA feedback on your CMC part of the filing?

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Typically, the CMC will be, also we're planning to file this year. I mean, FDA has right to, you know, request comments before or they will wait for entire section to be filed. Even though they're internally reviewing, you may not expect anything before the actual final clinical module is filed.

[Boris Peaker, Managing Director and Senior Equity Research Analyst at Titan Partners Group]

Got it. You know, speaking of the FDA, have you discussed the Ellipsoid Zone as an endpoint with the agency? I'm just curious what their thoughts about it as maybe, you know, kind of a secondary endpoint. Is it something that could be incorporated into a label claim? Would that make any kind of a difference from the commercial perspective? Just kind of general thoughts on that endpoint.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Ellipsoid Zone, I mean, obviously, as we stated before, all our clinical trials, we're trying to show a benefit because diseases we are targeting have significant unmet medical needs. More delays and doing longer trials will take not only the resources from our perspective, it's not doing benefit to the patients. If you're able to show a benefit using primary endpoints, what we picked, which are acceptable, obviously, the EZ will be a secondary and some other analysis just to support further that, you know, demonstrating this is showing a good functional outcome or related to functional outcome. That's important. If you do longer trials like two or three years, sure, we can look into multiple options. Obviously, the agency's perspective from FDA's perspective, they really focus on primary endpoint. If you hit the primary endpoint, you'll get the approval.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

If you hit the secondary, yes, you can include it in the product insert and the label. However, remember, all our clinical trials, we have obligation to continue them for five years for safety monitoring. That means one year data is needed for filing. After that, second year, third year, fourth year, and fifth year, we monitor the patients. Even we'll continue to monitor them with the secondary endpoints. At any point, the data is looking good, we can always add it to the label. From FDA's perspective, you have to hit the primary endpoint to get the product approved. Secondary is not necessary for approval. I mean, if you hit it's good. You can put it in the label.

[Boris Peaker, Managing Director and Senior Equity Research Analyst at Titan Partners Group]

Got it. I just want to understand also, have you spoken to docs? Like, what's the commercial value? Let's say you could get an Ellipsoid Zone label claim, you know, obviously not the primary endpoint but still mentioned as positive in the label. Would that really make a difference? Is this something that the docs actually care about, or is it just kind of scientifically nice and a curiosity more than anything else?

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Yeah. Go ahead, Huma.

[Huma Qamar, CMO at Ocugen]

Yeah. Boris, this is Huma. In terms of your question, with MD alignment, yes, all the protocols are approved with exploratory secondary endpoints. Yes, in terms of EZ, it's the new hot topic for the clinicians, in terms of functional outcomes and structural integrity for photoreceptor and retinal pigment epithelium. This is where actually FDA is leaning a lot, based on, you know, these particular conditions such as Stargardt disease and geographic atrophy secondary to age-related macular degeneration. In fact, there has been a buying in consensus from the IRD physicians as well as the geographic atrophy, AMD, surgeons as well. There is a real benefit to it, not only from the structural perspective, but also from functional.

[Huma Qamar, CMO at Ocugen]

Yes, this is now being taken not only, you know, nationally in U.S., but also from Europe as well. Of course, there is a clinically meaningfulness in terms of functional outcome for EZ. That's what we are seeing, that, you know, that could have a potential meaningful information when we are going to file our claim commercially.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Also geographic atrophy phase III in Stargardt why we're going to look at entire phase II data set this month, then we're going to propose the endpoints with FDA and EMA. We do have an opportunity to introduce EZ as a secondary endpoint if the data is trending the way we anticipate.

[Michael Okunewitch, Senior Research Analyst at Maxim Group]

Great. Thank you very much for taking my questions.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Thank you.

[Operator]

Thank you. Our next question comes from the line of Swayampakula Ramakanth from H.C. Wainwright. Please go ahead.

[Swayampakula Ramakanth, Managing Director and Senior Healthcare Analyst at H.C. Wainwright & Co.]

Thank you. Good morning, Shankar, Rita, and Huma. A couple of questions from me. Looking into the OCU410 program, you know, in the phase II study, the medium dose showed a, you know, 54% reduction versus the high dose which showed a 36% reduction. I'm just trying to understand, you know, when as you go into your phase III study, what is going to impact your decision for dose selection? And also, do you think that between these doses, you are actually seeing some sort of a plateau effect in the transgene expression?

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

RK, good morning. Yeah. I think the data we released, obviously, the high dose had less numbers in there. I would wait until we get the complete data set this month to make any inference. Obviously, you know, agency's perspective, if a lower dose is showing equal or better effect, I mean, you take that into phase III. That's a standard practice. I suggest to now we wait. Typically, what we look for in our genes and what we have seen in our RP studies too, typically these genes require a threshold. Once you hit the threshold, we didn't see any dose response. We're going to evaluate carefully once we get the full data set.

[Swayampakula Ramakanth, Managing Director and Senior Healthcare Analyst at H.C. Wainwright & Co.]

Okay. Thanks for that. you know, in the subpopulations where the baseline lesions were greater than, equal to or greater than 7.5 mm square, you saw a 57% reduction in the lesion growth. As you get into the phase III study, you know, would you have any restrictions in terms of the size of the lesions? Or do you plan to use the same criteria as in phase II, which was the all comers?

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

That's a good question. Yes. This is why we do phase II, right? We're going to carefully evaluate and we go from, what is it, 2.5 to 22. We're going to evaluate and see. Because on the lower side, I mean, as you know, analytically, you'll have more variability. Of course, we're going to look at, you know, where the average patients fall, even though in the large trials people have done way a lot of data. We're going to look at all those metrics and see what is the right group to go into the phase III.

[Swayampakula Ramakanth, Managing Director and Senior Healthcare Analyst at H.C. Wainwright & Co.]

All right. The last question from me is on the ST for, OCU410ST program, where, you know, you're expecting to get the enrollment done this quarter and put up some interim data in Q3. In that data set, you know, what are we really looking for which can give us some indication of how the 2027 BLA filing is gonna go, especially I'm thinking about the signals on either on the structural side of things or on the functional side of things and where, what do you weigh more and how should we be thinking when the data comes out?

[Huma Qamar, CMO at Ocugen]

RK, good morning. Thanks for your question. In terms of the masked interim analysis that's coming, later part of the year will be for 24 subjects, 16 treatment and eight in the control. This is the adaptive design that's a unique approach we have taken. What are the, what kind of data points we're going to present as we have presented this morning as well, of course, the primary endpoint, the lesion growth reduction, as well as structural as well as functional, which is, the visual acuity. Not last but not the least, of course, we are looking into the Ellipsoid Zone, which is the functional outcome, which is very unique. That data was very well received from Stargardt perspective, that we have recently presented at one of the conferences as well.

[Huma Qamar, CMO at Ocugen]

Yeah, we are going to look all of that, and of course, safety and tolerability will be there as well. As of right now, it is trending in the right direction, and this is what we are looking into to release masked interim analysis for those subjects later part in the year.

[Swayampakula Ramakanth, Managing Director and Senior Healthcare Analyst at H.C. Wainwright & Co.]

Perfect. Thank you very much. Thanks for taking all my questions.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Thank you.

[Operator]

Thank you. Our next question comes from the line of Elemer Piros from Lucid Capital Markets. Please go ahead.

[Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets]

Yes, good morning. I'd like to ask a question about the primary measure visual function in the RP study. What would be a clinically meaningful improvement? Where do you draw the threshold for that?

[Huma Qamar, CMO at Ocugen]

Thanks for your question. Basically, as we said that it's a change in lux level improvement from baseline. As you know, there is a lot of mutations we are looking into. Technically, 1 lux level and more because it's a validated protocol, LDNA and Luminance Dependent Navigation Assessment. That's what we are aiming for, and that's what our analysis is going to be based off of. As I've said earlier as well, there is a lot of heterogeneity with clinical diagnosis, syndromic, non-syndromic forms. Of course, the clinically meaningfulness is greater than or equal to 1 lux level.

[Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets]

One lux.

[Huma Qamar, CMO at Ocugen]

Depending on. Yes. Greater than or equal to 1 lux.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

I think Elemer Piros, as we, Huma Qamar has stated, we validated this course during phase III with the real patients, and, the course looks very robust. Based on our KOL input, they're very extremely happy with this.

[Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets]

Yeah. Thank you. What are some of the secondary endpoints that you will also look at to support the primary?

[Huma Qamar, CMO at Ocugen]

Of course, the secondary endpoints are, of course, on the visual acuity, low luminance visual acuity, and also, you know, the patient-reported outcome scores. We've been looking into it, and that is actually very well, you know, agreed and aligned upon with the FDA.

[Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets]

One last question. Are both eyes treated, if you could remind us, in the treatment arm?

[Huma Qamar, CMO at Ocugen]

Yes, that is correct.

[Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets]

Yeah. Okay.

[Huma Qamar, CMO at Ocugen]

Yes. If they meet the inclusion/exclusion criteria, both the eyes are treated. It's a two into one randomization, a single subretinal injection, and the control group will have a crossover after one year.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Yeah. The study eye is the worst eye for analytic analysis. Yes, exactly.

[Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets]

You would compare the worst eye to the control group and the worst eye in that group?

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Yeah. It's so the study eye, yeah. Study eye is compared with the control group. Yeah.

[Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets]

Yeah.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

From the analysis primary.

[Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets]

Thank you so much.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Yeah.

[Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets]

Thank you, Shankar. Thanks, Huma.

[Operator]

Thank you. Our next question comes from the line of Daniil Gataulin from Chardan. Please go ahead.

[Analyst at Chardan]

Hi, this is Steven on for Daniil. Thanks for taking my question. For dry AMD, you mentioned the 50% responder rate. Were there any underlying characteristics that made a patient more likely to be a responder?

[Huma Qamar, CMO at Ocugen]

Are you talking about the 410 GA?

[Analyst at Chardan]

Yes.

[Huma Qamar, CMO at Ocugen]

Yes. In terms of that, the responder rate. By the way, we have the inclusion/exclusion criteria, which was very well uniform across the groups. It was, you know, the baseline characteristics were that, there was a mean age that we were looking into. Of course, the GA gets diagnosed at a certain age. Of course, 70 in the mid-seventies was the mean age. We were also looking at, you know, the lesion size, which actually is pretty much well worse with the OAKS and DERBY trials, and Apellis has got approval on it, was 7.5 mm squared. That was the mean as well, up to 8.03.

[Huma Qamar, CMO at Ocugen]

In terms of the baseline characteristics, the responders basically responded, you know, on the medium dose as well as on the high dose as well. There was not really any other unique criteria that we would say at this point till we get our final, you know, clinical study report at that point. At this point, it seemed like it was uniform across all dose groups.

[Analyst at Chardan]

Got it. Thank you.

[Operator]

Thank you. This concludes the Q&A portion. I will now turn the call back over to Chairman, CEO, and Co-founder, Dr. Shankar Musunuri.

[Shankar Musunuri, Chairman, CEO, and Co-founder at Ocugen]

Thank you, operator. 2025 was marked by important clinical progress, strategic business development, and essential financing accomplishments across the organization. We're entering 2026 with a strong momentum and a clear line of sight to multiple catalysts that will further advance Ocugen's position as a biotechnology leader in gene therapy for blindness diseases. We expect to deliver full phase II data for OCU410 this month, complete enrollment for OCU410ST, initiate phase III for OCU410 in geographic atrophy, and begin our rolling BLA submission for OCU400. I want to thank our employees, investigators, patients, and shareholders for their continued support. We look forward to updating you on our progress. Have a great day.

[Operator]

The meeting is now concluded. Thank you all for joining. You may now disconnect.

Participants

Executives

• Huma Qamar, CMO
• Rita Johnson-Greene, CFO
• Shankar Musunuri, Chairman, CEO, and Co-founder
• Tiffany Hamilton, Head of Corporate Communications

Analysts

• Boris Peaker, Managing Director and Senior Equity Research Analyst at Titan Partners Group
• Elemer Piros, Managing Director and Research Analyst at Lucid Capital Markets
• Michael Okunewitch, Senior Research Analyst at Maxim Group
• Swayampakula Ramakanth, Managing Director and Senior Healthcare Analyst at H.C. Wainwright & Co.
• Analyst at Chardan