Ocugen Q2 2026 Earnings Report

Ocugen EPS Results

• Actual EPS: -$0.07
• Consensus EPS: -$0.05
• Beat/Miss: Missed by -$0.02
• One Year Ago EPS: N/A

Ocugen Revenue Results

• Actual Revenue: $1.49 million
• Expected Revenue: $0.89 million
• Beat/Miss: Beat by +$599.00 thousand
• YoY Revenue Growth: N/A

Ocugen Announcement Details

• Quarter: Q2 2026
• Date: 8/6/2026
• Time: Before Market Opens
• Conference Call Date: Thursday, August 6, 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 Q2 2026 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: OCU410 received FDA clearance to begin its Phase III ARMADA-3 trial in geographic atrophy, along with RMAT designation. The approximately 237-patient global study is expected to start by September 2026, supported by Phase II data showing a 31% reduction in lesion growth at the optimal dose.
• Positive Sentiment: Ocugen expects several major clinical catalysts, including an interim decision for OCU410ST in Stargardt disease in the third quarter of 2026 and top-line data for OCU400 and OCU410ST in the first half of 2027. Management continues to target three BLA submissions by 2028.
• Positive Sentiment: Enrollment is complete in the 140-patient Phase III liMeliGhT trial for OCU400 in broad retinitis pigmentosa, with top-line data expected in the first quarter of 2027. The company also completed manufacturing qualification batches intended to support potential BLA filings and commercial supply.
• Positive Sentiment: Ocugen closed a $130 million convertible notes financing, reporting $100.4 million in cash, cash equivalents, and restricted cash at June 30, which management says extends its runway into 2028. It also signed a binding term sheet granting Roots Pharmaceutical and Al-Dhow International exclusive OCU400 rights in the Middle East and North Africa.
• Negative Sentiment: Operating expenses increased to $17.9 million in the quarter from $15.2 million a year earlier, while quarterly net loss widened to $0.07 per share from $0.05. Management is evaluating additional financing, warrant exercises, business-development transactions, and a potential share authorization increase, creating possible future dilution.

Presentation

[Operator]

Good morning, welcome to Ocugen's second quarter 2026 financial results and business update. All participants are in listen-only mode. Following the speakers' commentary, there will be a question and answer session. I will now turn the call over to Chris Clark, Ocugen's Head of Communications. You may begin.

[Chris Clark, Head of Corporate Communications at Ocugen]

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. Rita Johnson-Greene, our Chief Financial Officer, is also on the call to provide a financial update for the quarter ended June 30th, 2026. Abhi Gupta, Executive Vice President of Commercial and Business Development, and Dr. Mohammed Genead, who joined Ocugen as Chief Medical Officer in June, will be available to answer questions following the presentation. This morning we issued a press release covering our business and operational highlights for the second 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 accompanying slide presentation, will be available on the investors section of the Ocugen website.

[Chris Clark, Head of Corporate Communications at Ocugen]

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, 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 differ materially 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 second quarter of 2026, 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, Chris, good morning, everyone. The second quarter was a defining one for Ocugen. The FDA cleared our phase III trial for OCU410 to initiate dosing in geographic atrophy patients and granted RMAT designation for the program. We signed a binding term sheet with Roots Pharmaceutical to negotiate an exclusive license for OCU400 in retinitis pigmentosa across the Middle East and North Africa, MENA region. From the closing of a $130 million convertible notes financing, we extended our cash runway into 2028, now able to support all three of our late-stage programs. Before I walk through the quarter, I want to step back because Ocugen's potential is worth putting into context. For more than a decade, gene therapy and ophthalmology has been confined to a single gene, a single mutation, and a single small patient population. Our modified gene therapy platform takes a fundamentally different approach.

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

Rather than targeting individual mutations, it is designed to address the root cause of complex retinal diseases by modulating master regulators, nuclear hormone receptors that govern multiple gene networks. The platform is gene agnostic, inherently multifactorial, and designed to deliver a durable benefit from a single one-time subretinal injection. What this means in practice is that Ocugen is not building three separate drugs. We are advancing one platform across three late-stage programs, each targeting a major cause of blindness for which patients today have either no approved treatment whatsoever or therapies that demand chronic injections and carry meaningful safety burdens. Retinitis pigmentosa, or RP, Stargardt disease, and geographic atrophy, or GA, together affect approximately 3 million people across the U.S. and Europe, a combined patient population, and the commercial opportunity for larger than anything currently served by approved gene therapies in ophthalmology.

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

Across our pipeline, spanning phase I through phase III, we have treated more than 325 patients, including EAP, through multiple doses and indications, and we have not observed a drug-related serious adverse event. We remain on track to file three BLAs by 2028. This positions the first half of 2027 as a catalyst reach window for Ocugen with the top-line data for OCU400 and OCU410ST and our planned BLA submissions following over a short period. Let me walk you through how each program is advancing. I will hand over the call to Rita for financials. Starting with OCU410 for GA, secondary to dry age-related macular degeneration, or dry AMD, GA represents our largest commercial opportunity with approximately 2 million-3 million patients in the U.S. and Europe combined. There are currently no approved treatments for GA in Europe.

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

Current approved therapies in the U.S. target only one complement pathway and require frequent intravitreal injections, which has been associated with treatment discontinuation in clinical practice. GA is a multifactorial disease driven by four distinct pathways that contribute to the progressive degeneration of the macula. Drusen, inflammation, oxidative stress, and complement or activation. The currently approved therapies in the U.S. address only one of these four pathways, the complement system, which is partly why they have been unable to demonstrate meaningful functional outcomes for patients. OCU410 operates differently by delivering RORA, a nuclear hormone receptor that acts as a master regulator of retinal homeostasis. OCU410 is designed to address all 4 disease pathways simultaneously with a single subretinal injection, has the potential to redefine the standard of care in this indication. We recently received FDA clearance for OCU410 phase III registrational trial for GA.

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

The trial, ArMaDa3, is planned to be a global study of approximately 237 subjects, using an adaptive design powered at 95% for the primary endpoint, with the BLA and market authorization application filings targeted for 2028. We plan to initiate phase III by September 2026. This design is anchored by positive 12-month data from our phase II ArMaDa trial. At the optimal dose, OCU410 delivered a statistically significant 31% reduction in GA lesion growth within the patient population of lesion size 2.5 millimeters square and 17.5 millimeters square. The criteria to be used in our phase III pivotal trial versus control, approximately twice the benefit of approved complement inhibitors and from a single injection. We also saw a 27% preservation of the ellipsoid zone within the same patient population, and no drug-related serious adverse events reported to date.

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

Importantly, this phase II data helps support the FDA's decision to grant RMAT designation for OCU410. Turning to OCU410ST for Stargardt disease. Stargardt is a pediatric-onset retinal disorder affecting approximately 100,000 patients in the U.S. and Europe, and roughly 1 million people globally. There are no approved therapies available for these patients today. OCU410ST is designed to address over 1,200 pathogenic mutations in the ABCA4 gene with a single one-time treatment. On April 1st, we announced the completion of enrollment and dosing in our phase II/III GUARDIAN3 pivotal confirmatory trial, enrolling 63 participants. We expect the interim outcome decision for the first 50% of subjects at eight months in the third quarter of 2026, and top-line phase II/III data in the second quarter of 2027, with our BLA submission to follow mid-2027. Moving to OCU400 for RP.

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

The phase III liMeliGhT trial is the first and largest genetic medicine registration trial for broad RP, spanning more than 30 genetic mutations. Approximately 300,000 people in the U.S. and Europe are living with RP, which is caused by mutations in more than 100 genes. The only approved gene therapy for RP today targets a single gene, RPE65, which accounts for less than 2% of all RP cases. OCU400 is designed to provide a therapeutic option for all RP patients, and that is a fundamentally different commercial opportunity. Enrollment in the liMeliGhT is complete, with 140 patients randomized two to one, treated versus control across the RHO and gene-agnostic arms, spanning more than 30 genetic mutations associated with early to late-stage RP, including pediatrics. The breadth of the population intended to validate the gene-agnostic mechanism of action of our novel modified gene therapy platform.

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

The primary endpoint is 12-month change in visual function assessed by luminance dependent navigation assessment, or LDNA. Subjects are followed for one year post-dosing for the primary endpoint analysis. Top-line phase III data is expected in the first quarter of 2027, advancing OCU400 to a potential approval in the fourth quarter of 2027. FDA feedback confirmed that the path to rolling BLA submission remains tied to top-line data expected in the first quarter of 2027. On the manufacturing side, our process performance qualification, PPQs, batches are complete, supporting BLA and commercial launch supplies. Brand planning and marketing initiatives led by Abhi Gupta, our EVP of Commercial and Business Development, continue to scale in preparation for launch. We also advanced our global commercialization strategy for OCU400 during the quarter.

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

In July, we signed a binding term sheet with Roots Pharmaceutical and its strategic partner, Al-Dhow International Holding, for exclusive rights to OCU400 in the Middle East and North Africa. We're active on the BD front to find other global partners for regional commercialization partnerships where RP is most prevalent. 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. As we continue advancing our pipeline, we're also building the foundational commercial capabilities to support future global access. Our efforts are focused on five key areas. First, we're in discussions with CMS and payers to establish early market access and reimbursement strategies.

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

Second, we continue to identify and evaluate specialized centers of excellence with expertise in subretinal surgical procedures that could support future treatment delivery. Third, we are mapping the patient journey from diagnosis through treatment and long-term follow-up with the goal of facilitating a seamless experience for patients, caregivers, and healthcare providers. Fourth, we are assessing manufacturing, supply chain, and distribution requirements to help ensure operational readiness. Finally, we are beginning to build out our commercial infrastructure, including our marketing and sales capabilities, as we ramp up for launch. With that, I'll turn the call over to Rita for the financial update. Rita?

[Rita Johnson-Greene, CFO at Ocugen]

Thank you, Shankar. Good morning, everyone. Total operating expenses for the three months ended June 30, 2026, were $17.9 million and included research and development expenses of $10.7 million and general and administrative expenses of $7.2 million. This compares to total operating expenses for the three months ended June 30th, 2025 of $15.2 million, which included research and development expenses of $8.4 million and general and administrative expenses of $6.8 million. Total operating expenses for the six months ended June 30th, 2026 were $37.3 million and included research and development expenses of $21.9 million and general and administrative expenses of $15.4 million. This compares to the total operating expenses for the six months ended June 30th, 2025 of $31.2 million, which included research and development expenses of $17.9 million and general and administrative expenses of $13.2 million.

[Rita Johnson-Greene, CFO at Ocugen]

Ocugen reported a $0.07 net loss per common share for the three months ended June 30th, 2026, compared to a $0.05 net loss per common share for the three months ended June 30th, 2025. On our capital position, following the closing of the $130 million convertible note financing, the company's cash equivalents, and restricted cash totaled $100.4 million as of June 30th, 2026, extending our cash runway into 2028. The company has 339 million shares of common stock outstanding as of June 30th, 2026. That concludes my financial update. Shankar, back to you.

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

Thank you, Rita. The second quarter was a quarter of execution. The remainder of 2026 is poised to be impactful. We expect the OCU410ST interim outcome decision in the third quarter. We expect to initiate the OCU410 phase III trial in this quarter. Looking to 2027, we expect top-line data from both OCU400 and OCU410ST in the first half of the year, followed by our planned BLA submissions. Each of these milestones brings us a step closer to delivering on our commitment to three BLAs by 2028, offering potentially life-altering improvement to patients coping with blindness causing diseases. I want to thank our investigators and patients who have trusted us with their participation and our shareholders for their continued belief in our mission to advance cures for blindness. We'll now open the call for questions. Operator?

[Operator]

Thank you. Ladies and gentlemen, we will now begin the question and answer session. At this time, I would like to remind everyone in order to ask a question, please press star followed by the number oneon your telephone keypad. If you would like to withdraw your question, simply press star one again. If you would like to ask a question, press star one on your telephone keypad. Our first question comes from the line of Michael Okunewitch with Maxim Group. Please go ahead.

[Michael Okunewitch, Analyst at Maxim Group]

Hey there. Thank you so much for taking my questions. Congrats on all the great progress.

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

Thank you, Michael.

[Michael Okunewitch, Analyst at Maxim Group]

I wanted to ask, you now have a handful of international partnerships, which makes OCU400 a truly international program at this point. I just wanted to see if you could share the regulatory plans in particular for ex-U.S. jurisdictions, what's required there, and how those timelines could vary versus your BLA path.

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

Michael, what we have with OCU400, we got alignment from EMA in addition to FDA with the same, that single trial we're doing in U.S. is good for approvals. Across the globe for orphan gene therapies, typically, they get approval based on U.S. approval. Everything will be linked to our U.S. FDA approval in MENA and other regions.

[Michael Okunewitch, Analyst at Maxim Group]

All right. Thank you. I wanted to see if also if you could just highlight some of the key differences in the trial design between ArMaDa-3 and the phase II ArMaDa-1 trial.

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

I let our CMO, Dr. Genead, answer that.

[Mohammed Genead, Chief Medical Officer at Ocugen]

Thank you, Michael. Regarding ArMaDa-3, which is our global, phase III trial for GA, which we just got the approval from FDA just recently to be initiated this quarter. The phase III trial design for ArMaDa-3 will be one treatment arm with OCU410 versus a control, will be 2:1 randomization allocation. The idea is to follow each subject up to 12-months. This way we're going to be looking at the primary efficacy endpoint plus other key functional endpoint. The ArMaDa-1, the earlier phase I/II GA trials was similar on the efficacy. We should expect similar outcome here. We're going to look. The numbers obviously are different. We're going to be enrolling in our ArMaDa-3 close to 237 subjects in 2:1 allocation. It's going to be global. We're going to go ex U.S.

[Mohammed Genead, Chief Medical Officer at Ocugen]

We're going to go to Europe and other territorial part in the world. The primary endpoint will be very similar, so we should expect to see similar trend what we saw from ArMaDa-1, the phase I/II GA trial.

[Michael Okunewitch, Analyst at Maxim Group]

All right. Thank you. Just one last one from me before I hop back into the queue. It looks like in Stargardt, there is a chance that we'll have an approved therapy sometime around when you'll be completing your own BLA filing. It'll be a chronic therapy versus a one-time. I wanted to ask how important the pricing on other therapies, since we don't have any pricing comps, would be to inform your own pricing strategy, and if there's any way that we can think about how to translate pricing between a chronic ongoing therapy and a one-time therapy.

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

Yeah. Good question, Michael. I think the way you look at it is our treatments are one and done treatments potentially, so that'll have a different pricing structure than ongoing chronic therapies. Number two, everything will be dictated by data. If you have a safe one-time treatment, I think our gene therapies, once again, and we're still collecting data, as you can see in some of the patients in RP, and as they approach second year, third year, they're improving further. The current therapies, if the oral therapy comes to the market, what patients, providers are going to look for is the therapy just reducing the degeneration of the disease? Or in some patients, is it stalling it? Is it has potential to reverse it in some patients? At least with our modified gene therapy, in some of the patients, we're seeing all those trends.

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

That could be a big differentiating factor. Also, as you know, Stargardt impacts a lot of pediatric patients. In their current clinical trial they're conducting focuses on 12+, and our clinical trial focuses on 3+. There are a lot of differentiators. Whenever we come for pricing, because of the differentiated disruptive technology platform we have, and obviously everybody will focus on safety, efficacy, and one and done treatment, that'll be more compelling for anyone. I think all those factors will be rolled in. I don't think we'll be truly comparing any pricing what the other chronic therapies are doing. If you have a me-too products, the answer is yes. If you have truly a differentiated and disruptive technology, just completely different, we can price it on its own merits.

[Michael Okunewitch, Analyst at Maxim Group]

All right. Thank you. I appreciate the additional color.

[Operator]

Our next question comes from the line of Whitney Ijem with Canaccord Genuity. Please go ahead.

[Whitney Ijem, Analyst at Canaccord Genuity]

Hey, guys. My congrats on all the progress as well. Just to keep going on the Stargardt discussion, Shankar, since you mentioned it, can you talk about a little bit more, I guess, around the TPP here and the potential to show kind of reversal of disease and improvement in visual acuity? Is that something that is reasonable to expect given the duration of follow-up in the ongoing phase II/III study? I guess if so, is there anything that was done in terms of entry criteria to maybe enrich for that outcome as far as patient baseline characteristics?

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

I will ask Dr. Genead to talk a little bit about baseline characteristics, then I'll answer the other question. Go ahead.

[Mohammed Genead, Chief Medical Officer at Ocugen]

Thank you, Shankar. Hi, Whitney. Yes, happy to answer. Our population was definitely broader than other competitor. Just to highlight, first, we included patients from early to late stage Stargardt disease. That's number one. As Shankar just mentioned, two, we included subjects younger than young adults. We included subjects three plus years of age. That's a very broad population. As you know, for Stargardt, the earlier, the better, especially it's a progressive retinal degeneration disease. The lesion size we also included in our trial, the phase II/III GUARDIAN3 trial, was more broader than what we saw with others, and our lesion size can include smaller lesion, also larger lesion. We have a broad spectrum, and that's also going to be aligned with our early late stage strategy for the disease.

[Mohammed Genead, Chief Medical Officer at Ocugen]

We already included some of the subject in our phase II, III trials, we will be excited to see the data. In addition to the gene mutation, specifically, we include all the variants and all the other specific mutation included in the ABCA4 related retinopathy. It includes Stargardt and others as well. This is also on the disease indication as well, overall. Based on your point about the functional, I think this is going to be critical. We saw from our phase I data that we just published at the Eye Nature early in the year. We saw a very clear slowing in the structure progression in those patients, and also we saw functional benefit in those patients.

[Mohammed Genead, Chief Medical Officer at Ocugen]

As you remember, as you know, in Stargardt disease, the first target is to hold that progression, to stop losing more retinal structure and function, which we achieved in our prior trial. The second goal, which will be the upside here, and the ultimate goal, to reverse that tide. Try to improve on the progress or improve on the disease outcome. We saw that in our phase I, II. We saw some of the patients did improve in visual function. The gain was six letters, close to one line between the treated versus the untreated eye. We felt also very excited about the functional gain in those patient population. That's kind of where we think the big differentiation, the broader application of our molecule.

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

Whitney, just to clarify the primary endpoint, because it's a one year trial, it's not a two year trial, it's still lesion. There are secondary visual function we'll be monitoring. In addition to that, at the time of filing, we continue to monitor our early-stage phase I patients, we'll have long-term data in those patients, too.

[Whitney Ijem, Analyst at Canaccord Genuity]

Got it. Really helpful. Just last question, and maybe Rita, this one's for you. Can you help us understand how you're thinking about cash, given the exciting progress with the GA study and the ability to start that study in September, I think you said? If there is a need to pull levers to extend cash runway further, how should we think about maybe the startup of GA versus commercial prep for RP or Stargardt, and just how you guys are thinking about those different levers if needed? Thanks.

[Rita Johnson-Greene, CFO at Ocugen]

Yeah. Thank you, Whitney. First of all, our primary goal is to make sure that we are minimizing shareholder dilution, evaluating our opportunities in order to raise capital, just as you said, in order to bring these novel products to patients. Just first of all, we have cash runway into 2028. I just want to remind everyone of that, which gives us the confidence to execute our clinical late-stage products that we have and then progress to BLA submission for both OCU410 and OCU410ST in 2027, with the potential to commercialize OCU400 by the end of the year in 2027. We do have some additional levers that we can pull. One, we have the PRV for OCU410ST, given the RPD designation that we have.

[Rita Johnson-Greene, CFO at Ocugen]

Of course, we have the ability to sell that for somewhere between $100-$200, even prior to approval, and that's something that we are evaluating. We also have various business development deals that we are looking at from a globalization perspective. We're looking at ex-U.S. for both OCU400, OCU410ST, and even GA, right? Just depending upon what that term sheet looks like. Always looking for potential deals that we can make in order to, again, just minimize that dilution. We also have the Janus Henderson warrants, right? There's another 10 million warrants at a $1.50 strike price, which could bring in another $15 million, and those warrants expire in August of 2027.

[Rita Johnson-Greene, CFO at Ocugen]

Of course, we anticipate a special meeting in September of this year in order to increase authorized shares, which will give us the ability to raise additional equity if we decide to do so. Again, just looking at both non-dilutive as well as dilutive options in order to make sure that we are able to bring these amazing and novel products to patients, as well as looking at maximizing shareholder value.

[Whitney Ijem, Analyst at Canaccord Genuity]

Very helpful. Thank you.

[Operator]

Our next question comes from the line of Charles Wallace with H.C. Wainwright. Please go ahead.

[Charles Wallace, Analyst at H.C. Wainwright]

Hi. This is Charles from H.C. Wainwright on for RK. Thanks for taking my question. Maybe a question on the ArMaDa-3 design. It seems like based on the prior earnings call, the study has been a little bit resized. I think previously you said it would be about 300 patients, and now it's 237 patients. I was just curious if this was something the FDA specifically asked for or if this was something you proposed. And then also if the assumptions changed based on effect size, variability, dropout, or the narrower lesion size compared to the phase II.

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

Yeah. Dr. Genead?

[Mohammed Genead, Chief Medical Officer at Ocugen]

Thank you, Charles. Yes. We had a discussion with the agency, the FDA. All this being aligned and discussed with the FDA. To answer your question specifically, it was based on the sample size estimation and also the power calculation we did. The estimate you are citing, the 300, was based on estimate. When we saw the effect size based on our ArMaDa-1, the phase I/II trial, as we discussed today, we saw the 31% reduction in the medium dose, the optimal dose, which is the one we are taking forward. When we did our calculation based on that, we saw the 237 total population to be enrolled will give us 95% power in our pivotal trial. All these pieces have been discussed with the agency. Obviously, it is based on the rate of change, the slope analysis for the primary efficacy.

[Mohammed Genead, Chief Medical Officer at Ocugen]

The effect size, based on what we saw from earlier trial, was very positive and was strong enough that we end up with 237. 2:1 randomization, as we mentioned earlier, 158 in the treatment arm and 79 in the control arm. All this has been discussed and aligned, and as we announced today, we got the clearance from the FDA to initiate our phase II trial in the next few weeks.

[Charles Wallace, Analyst at H.C. Wainwright]

Thank you. Very helpful. I guess for the rolling submission, I think originally the guidance was to submit in the third quarter, and now I believe it is the first quarter after the liMeliGhT data. I guess my question is, what kind of changed between submitting the non-clinical module earlier compared to after the top line data, the liMeliGhT?

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

Charles, I think from our perspective, we are ready. I think we are doing very well with our PPQs, as we have mentioned. A lot of gene therapy companies stuck with CMC. We are ahead of the game. We used two commercial-scale lots in our phase III. We completed our PPQs on time. We got non-clinical and PPQ are done, so we have CMC non-clinical ready to go.

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

Obviously, this is where we had to work with the agency whenever they are comfortable, and that is the timeline they gave us, and we are going to be fine with that. The reason is, I just want to clarify, it is good to have rolling submission that gives a head start for agency. Okay? It is for their own benefit. If they want to wait until next year, we are ready to file it as soon as the top line comes from the pre-BLA meeting.

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

We may still give them a head start of maybe a month or two months before we drop the clinical section. However, I just want to clarify, until the final BLA is completed with the clinical section, the PDUFA date, the accelerated clock of six months doesn't start. I just want to clarify that. Once again, this is a collaboration between the sponsor and the agency. In this case, of course, we respect their decision, whatever they are, because they have a lot of programs and a lot of workload. Whatever the reasons are, we're fine with it. I think we're ready from our perspective, and we'll work with them closely in a collaborative way. Whenever we have a top line, we'll be ready to file it.

[Charles Wallace, Analyst at H.C. Wainwright]

Great. Very helpful.

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

It doesn't change any filing clock as we mentioned before. Second quarter, complete the BLA filing. Anticipated approval in fourth quarter. Six months accelerated clock.

[Charles Wallace, Analyst at H.C. Wainwright]

Very helpful. Thanks for taking both questions.

[Operator]

Once again, if you would like to ask a question, please press star followed by the number one on your telephone keypad. Our next question comes from the line of Robert LeBoyer with Noble Capital Markets. Please go ahead.

[Robert LeBoyer, Senior Equity Research Analyst at Noble Capital Markets]

Good morning. Congratulations on the progress. Just to follow up on that last question. My understanding was that the BLA submission will be completed in early 2027 when the clinical module is filed. That's when you get the PDUFA date and the approval launch is based on that. You also have rolling submission and have the option of filing the CMC and the other non-clinical modules before that. Is that still your plan?

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

Yes, Robert. Absolutely. Based on agency's suggestion and recommendation, as soon as the top line comes out, we'll have a pre-BLA meeting. Right after that, we can file the two modules, non-clinical and CMC modules. That will still give them a head start. As soon as the clinical module is done, when you file it, the PDUFA date starts. That's basically our plan is to file that in the second quarter. Six months clock should be fourth quarter, approval clock.

[Robert LeBoyer, Senior Equity Research Analyst at Noble Capital Markets]

Okay, terrific. Thank you for that.

[Operator]

At this time, we have no further questions. I would like to turn the call back over to the Ocugen team for closing remarks.

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

Thank you all for attending today's webcast. Really appreciate all our investors, shareholders, patients, providers. Thank you.

[Operator]

This concludes today's conference call. You may now disconnect. Have a good day.

Participants

Executives

• Chris Clark, Head of Corporate Communications
• Shankar Musunuri, Chairman, CEO, and Co-Founder
• Rita Johnson-Greene, CFO
• Mohammed Genead, Chief Medical Officer

Analysts

• Michael Okunewitch, Analyst at Maxim Group
• Whitney Ijem, Analyst at Canaccord Genuity
• Charles Wallace, Analyst at H.C. Wainwright
• Robert LeBoyer, Senior Equity Research Analyst at Noble Capital Markets