OCGN Q1 2026 Earnings Call
Operator (AI Assigned): Good morning. Welcome to Ocugen Q1 2026 Financial Result and Business Update. All participants line are currently in listen-only mode. Following the speaker commentary, there will be a question-and answer-session. I will now turn the call over to Tiffany Hamilton, Ocugen Head of Corporate Communications.
Tiffany Hamilton (Head of Corporate Communications): Thank you, Operator, and good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri, Ocugen's Chairman, CEO, and Co-founder, who will provide a business update and an overview of our clinical and operational progress. Rita Johnson-Greene, our Chief Financial Officer, is also on the call to provide a financial update for the quarter ended 31 March 2026. Dr. Huma Qamar, Chief Medical Officer, will be available to answer questions following the presentation.
Tiffany Hamilton (Head of Corporate Communications): This morning, we issued a press release covering our business and operational highlights for Q1 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 investor section of the Ocugen website.
Tiffany Hamilton (Head of Corporate Communications): 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 pipelines, 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 results to differ materially from those expressed or implied.
Tiffany Hamilton (Head of Corporate Communications): 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 Q1 2026 will be filed today. I will now turn the call over to Dr. Musunuri.
Shankar Musunuri (Chairman, CEO, and Co-founder): Thank you, Tiffany, and good morning, everyone. Before I walk through the quarter, I want to discuss the $115 million offering of convertible senior notes that we announced yesterday. With the recent offering, the company is expected to have cash equivalents and restricted cash of $112.1 million at closing, which includes the Avenue debt payoff. The company will use the remaining net proceeds for general corporate purposes and expects to extend cash runway into 2028.
Shankar Musunuri (Chairman, CEO, and Co-founder): The offering is expected to close on 7 May 2026, subject to customary closing conditions, and includes an option to retire the debt with a cash payment. If the remaining Jones Henderson warrants are exercised, the company will receive an additional $15 million in gross proceeds, increasing expected cash equivalent and restricted cash to $127.1 million. Now, I would like to step back because Ocugen's potential is worth putting into context.
Shankar Musunuri (Chairman, CEO, and Co-founder): 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. 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 entire gene networks.
Shankar Musunuri (Chairman, CEO, and Co-founder): The platform is gene agnostic, inherently multifactorial, and designed to deliver durable benefit from a single one-time subretinal injection. What this means in practice is that Ocugen is not building three separate drugs. We're 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.
Shankar Musunuri (Chairman, CEO, and Co-founder): Retinitis pigmentosa or RP, Stargardt disease, and geographic atrophy or GA together affect approximately 3 million people across the United States and Europe, a combined patient population and a commercial opportunity far larger than anything currently served by approved gene therapies in ophthalmology.
Shankar Musunuri (Chairman, CEO, and Co-founder): Across our pipeline, spanning phase 1 through phase 3, we have treated more than 250 patients across multiple doses and indications, and we have not observed a drug-related serious adverse events. In our most recent readout, OCU410 demonstrated approximately twice the treatment benefit over currently approved therapies in GA, delivered at one-time injection. We remain on track to file 3 BLAs over next 3 years by 2028. First of those, OCU400 for RP, will begin rolling submission in Q3 of this year.
Shankar Musunuri (Chairman, CEO, and Co-founder): This positions H1 2027 as a catalyst-rich window for Ocugen with Phase 3 top-line data for OCU400, top-line Phase 2/3 data for OCU410ST, and BLA submissions all expected to converge over a short period. In the first months of 2026, we completed enrollment in 2 of our late-stage programs, delivered positive Phase 2 top-line data in a third, and are diligently working toward initiating our first BLA submission later this year. Let me walk you through how each program is advancing. Starting with OCU400 for RP.
Shankar Musunuri (Chairman, CEO, and Co-founder): The phase 3 liMeliGhT clinical trial is the only broad gene agnostic RP trial and the largest known phase 3 orphan gene therapy trial in the field. Approximately 300,000 people in the US 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 just 1% to 2% of all RP cases.
Shankar Musunuri (Chairman, CEO, and Co-founder): OCU400 is designed to provide a therapeutic option for the remaining 98% to 99% of RP patients, and that is a fundamentally different commercial opportunity. Enrollment in liMeliGhT is now complete, with 140 patients randomized 2 to 1 across the rho and gene agnostic arms covering over 25 genetic mutations associated with early to advanced stage RP, including pediatrics. The breadth of population is intended to validate the gene agnostic mechanism of action of our novel modifier gene therapy platform.
Shankar Musunuri (Chairman, CEO, and Co-founder): The primary endpoint is 12-month change in visual function assessed by luminance dependent navigation assessment or LDNA. We plan to initiate the rolling BLA submission for OCU400 in Q3 2026 and complete BLA submission by Q2 2027. Phase 3 top-line data is expected in Q1 2027, with a potential FDA approval targeted for Q4 2027. On the manufacturing side, process performance qualifications, PPQ batches completion is on track for Q2 2026.
Shankar Musunuri (Chairman, CEO, and Co-founder): Brand planning and marketing initiatives led by Abhi Gupta, our EVP of Commercial and Business Development, continue to scale in preparation for launch. OCU400 continues to demonstrate encouraging long-term durability with the 3-year data supporting sustained improvement in visual function compared with untreated eyes.
Shankar Musunuri (Chairman, CEO, and Co-founder): In the Phase 1/2 study, the treatment effect was maintained over time across evaluable subjects with the clinically meaningful mean changes in LLVA observed at years 1, 2, and 3 in both the multiple mutation and rho subgroups. Importantly, these results suggest the benefit is not limited to a single genetic subtype, reinforcing the program's gene agnostic mechanism of action. From a safety perspective, OCU400 continues to show a favorable profile with no serious adverse events reported as being related to treatment.
Shankar Musunuri (Chairman, CEO, and Co-founder): At the 3-year time point, 88% of treated evaluable subjects demonstrated either improvement or preservation in visual function relative to untreated eyes, highlighting both the durability and consistency of the response. Taken together, these data support the potential for OCU400 to deliver sustained clinical benefit over time in retinitis pigmentosa while maintaining a strong safety profile. Turning to OCU410ST for Stargardt disease.
Shankar Musunuri (Chairman, CEO, and Co-founder): Stargardt disease is a pediatric-onset retinal disorder affecting approximately 100,000 patients in the US and Europe, and roughly 1 million 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 1 April, we announced the completion of enrollment and dosing in our Phase 2/3 GARDian3 pivotal confirmatory trial, enrolling 63 participants in less than 9 months, well ahead of the originally planned timeline.
Shankar Musunuri (Chairman, CEO, and Co-founder): That pace reflects both the depth of unmet need in Stargardt disease and the exceptional engagement of our investigators and patient community. The interim analysis is planned for Q3 2026, with the top-line Phase 2/3 data expected in Q2 2027, and BLA submission to follow by mid-2027. OCU410ST continues to demonstrate a favorable safety and tolerability profile, with no product-related serious adverse events reported to date. Turning now to OCU410 for GA. Late-stage dry age-related macular degeneration.
Shankar Musunuri (Chairman, CEO, and Co-founder): GA represents our largest commercial opportunity, with approximately 2 to 3 million patients in the United States and Europe combined. There are currently no approved treatments for GA in Europe. In the United States, approved therapies require 6 to 12 intravitreal injections per year indefinitely, carry meaningful safety risks, including conversion to wet AMD in roughly 12% of treated patients, and face real-world dropout rates of nearly 40%.
Shankar Musunuri (Chairman, CEO, and Co-founder): GA is a multifactorial disease driven by four distinct pathways that contribute to the progressive degeneration of the macula, lipid deposits, drusen, chronic inflammation, oxidative stress, and complement activation. The currently approved therapies in the US 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.
Shankar Musunuri (Chairman, CEO, and Co-founder): By delivering RORA, a nuclear hormone receptor that acts as a master regulator of retinal homeostasis, OCU410 is designed to address all four disease pathways simultaneously with a single subretinal injection, has the potential to redefine the standard of care in this indication. In March, we reported positive top-line 12-month data from our Phase 2 ArMaDa clinical trial.
Shankar Musunuri (Chairman, CEO, and Co-founder): The study enrolled 51 patients aged 50 years and older with the GA lesions in the foveal or non-foveal region, randomized one to one to one to receive a single subretinal administration of OCU410 at the medium dose, high dose, or no treatment in the control group. The optimal dose, which is the medium dose, demonstrated a 31% reduction in lesion growth relative to control at 12 months, with a P value of less than 0.05.
Shankar Musunuri (Chairman, CEO, and Co-founder): To put this in context, currently approved intravitreal therapies have shown approximately 15% reduction at 12 months for avacincaptad pegol and 22% reduction at 24 months for pegcetacoplan. OCU410 administered as a single one-time injection has shown the potential for an approximately 2-fold treatment benefit relative to approved therapies that require continuous chronic dosing regimens. Across the treated population, 55% of treated subjects demonstrated a 30% or greater reduction in lesion size relative to control.
Shankar Musunuri (Chairman, CEO, and Co-founder): We also observed a 27% slower rate of ellipsoid zone loss, which is structural biomarker that correlates with preservation of photoreceptor integrity and visual function. On safety, OCU410 continues to demonstrate a safe and tolerable profile with no serious adverse events, no adverse events of special interest related to OCU410 reported to date. We are now incorporating these results into an optimized Phase 3 trial design, including a targeted GA lesion size window and an adaptive design powered at greater than 95%.
Shankar Musunuri (Chairman, CEO, and Co-founder): We are now on track to meet with FDA and EMA to align on the Phase 3 study design and reach regulatory agreement by Q3 2026, with potential BLA filing by 2028. As a one-time treatment for life, OCU410 has the potential to eliminate the chronic treatment burden and patient fatigue associated with currently approved therapies and to offer a lasting solution for the 2 to 3 million patients in the US and Europe living with GA. Let me briefly update you on other programs. For OCU200, we completed phase I clinical trial enrollment in Q1 2026.
Shankar Musunuri (Chairman, CEO, and Co-founder): No serious adverse events or adverse events related to OCU200 have been reported to date. On OCU500, our first-in-class inhaled mucosal COVID-19 vaccine candidate designed to be administered via inhalation and intranasal delivery, NIAID intends to initiate the OCU500 phase I clinical trial in Q2 2026. Today, we want to highlight the meaningful progress across our retinal gene therapy pipeline and the important milestones we expect over the next several quarters.
Shankar Musunuri (Chairman, CEO, and Co-founder): For OCU400 in retinitis pigmentosa, we remain on track to begin a rolling BLA submission in 2026, with Phase 3 top-line data expected in 2027, and the potential for BLA and launch thereafter. For OCU410ST in Stargardt disease, we have completed dosing ahead of schedule in our pivotal Phase 2/3 GARDian3 trial, with interim analysis expected in the Q3 of 2026 and top-line results anticipated in the Q2 of 2027, followed by a planned BLA submission.
Shankar Musunuri (Chairman, CEO, and Co-founder): For OCU410 in geographic atrophy, we continue to advance toward Phase 3 development following encouraging Phase 2 data, with the program expected to move through Phase 3 enrollment toward BLA submission by 2028. Taken together, these milestones reflect a disciplined multi-program strategy designed to create value through a series of increasingly important clinical and regulatory readouts. I'll now turn the call over to Rita to provide an update on our financial results for the quarter ended 31 March 2026. Rita?
Rita Johnson-Greene (CFO): Thank you, Shankar. Good morning, everyone. Total operating expenses for the 3 months ended 31 March 2026 were $19.4 million and included research and development expenses of $11.3 million and general and administrative expenses of $8.1 million, compared to total operating expenses for the 3 months ended 31 March 2025 of $16 million that included research and development expenses of $9.5 million and general and administrative expenses of $6.5 million.
Rita Johnson-Greene (CFO): Ocugen reported a $0.06 net loss per common share for the 3 months ended 31 March 2026, compared to a $0.05 net loss per common share for the 3 months ended 31 March 2025. The company's cash equivalents, and restricted cash totaled $32.2 million as of 31 March 2026, compared to $18.9 million as of 31 March 2025. The company received $37.5 million in gross proceeds, inclusive of $15 million due to exercised warrants in Q1 2026.
Rita Johnson-Greene (CFO): With the recent offering, the company is expected to have cash equivalents, and restricted cash of $112.1 million at closing, which includes the Avenue debt payoff and expects to extend cash runway into 2028. The company had 338.3 million shares of common stock outstanding as of 31 March 2026. That concludes my financial update. Shankar, back to you.
Shankar Musunuri (Chairman, CEO, and Co-founder): Thank you, Rita. The Q1 2026 was a quarter defined by execution. We delivered positive 12-month phase 2 data for OCU410 in GA, completed enrollment and dosing in the GARDian3 trial well ahead of schedule, and continued advancing OCU400 towards its rolling BLA submission later this year. Looking ahead, the remainder of 2026 is poised to be consequential with multiple meaningful inflection points.
Shankar Musunuri (Chairman, CEO, and Co-founder): We expect interim outcome analysis from GARDian3 in Q3, regulatory alignment with FDA and EMA on the OCU410 phase 3 design in Q3, and the initiation of our first BLA submission for OCU400 also in Q3. Each of these milestones bring us a step closer to delivering on our commitment of 3 BLAs by 2028. I want to thank our employees, investigators, and patients who have trusted us with their participation, and our shareholders for continued belief in our mission to advance cures for blindness. We'll now open the call for questions. Operator?
Operator (AI Assigned): 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 will pause for just a moment to compile the Q&A roster. Your first questions comes from the line of Leland Gershell from Oppenheimer. Please go ahead.
[Analyst] (Oppenheimer) (Analyst): Hey, Ocugen team. This is Jason on for Leland. It seems like it's gearing up to be an exciting year for OCU400 and the retinitis pigmentosa. With that, could you give us a sense of what's being submitted for the rolling BLA versus the full BLA next year? How else are you thinking about the pre-commercialization activities going into 2027 launch? Thank you.
Shankar Musunuri (Chairman, CEO, and Co-founder): Yeah. Yep. The rolling submission, as we stated, will start with non-clinical section module. Also, we're completing our PPQ lots from CMC manufacturing perspective. That module will also be submitted this year. Next year, as soon as the top-line results come in, within weeks after that, the final clinical module will be submitted, and that will trigger a PDUFA date of 6 months clock, expected to get the approval in the Q4 and launch. From commercial perspective, there are multiple things we are doing.
Shankar Musunuri (Chairman, CEO, and Co-founder): As we mentioned before, we're working on one thing on the pricing with the government CMS. Second step is, of course, as a company, we have established very standard way of treating these patients with our surgery procedure, which we use in clinical trials, to minimize any risk to the patient. We want to really identify the centers for excellence where our gene therapy can be administered. That's what we're working on as the second step. The third step is, of course, getting up for sales and marketing and really commercial plans.
Shankar Musunuri (Chairman, CEO, and Co-founder): I think we're working on the strategy. Typically you start that work year before. Later part of the year, getting into the early next year, that's when we'll start bulk of the commercial work.
[Analyst] (Oppenheimer) (Analyst): Sounds great. Thank you again.
Shankar Musunuri (Chairman, CEO, and Co-founder): Thank you.
Operator (AI Assigned): Your next questions comes from the line of Michael Okunewitch from Maxim Group. Please go ahead.
Michael Okunewitch (Senior Biotechnology Analyst): Hey there. Thank you for taking my questions today. Good morning, everyone, and congrats on all the great progress you've been making.
Shankar Musunuri (Chairman, CEO, and Co-founder): Thank you.
Michael Okunewitch (Senior Biotechnology Analyst): I guess just to start off, I'd like to see, given how quickly the Stargardt program has been moving, how close together are you expecting an RP and a Stargardt approval could occur? Are there any additional commercial considerations you're evaluating with the possibility that you will be kind of ramping both of these launches contemporaneously?
Shankar Musunuri (Chairman, CEO, and Co-founder): No, good question. I mean, if everything goes according to the plan, they could be within 6 months of each other. obviously, from launch or commercial perspective, again, we're targeting the same centers for excellence for gene therapy administration. The same surgeons who are administering for RP. In fact, actually, it helps us as a company, economies of scale, and how we are setting it up for RP, and that'll always help with Stargardt disease.
Shankar Musunuri (Chairman, CEO, and Co-founder): When Stargardt, you know, is going to come later within 6 months, we should be in a great shape, from commercial perspective and launch perspective.
Michael Okunewitch (Senior Biotechnology Analyst): All right. How much overlap is there between the. The liMeliGhT and the GARDian3 studies, are they largely at the same centers, or if you take them combined, do they reach a broader swath of the overall market?
Shankar Musunuri (Chairman, CEO, and Co-founder): Go ahead, Huma.
Huma Qamar (Chief Medical Officer): Good morning. This is Huma Qamar. I'm going to take this question. Good question. Both represent inherited retinal diseases, of course, retinitis pigmentosa. You know, we have almost the same centers. There is a single subretinal injection. There is an overlap. However, there is no approved product for Stargardt, and we do have a huge unmet medical need in terms of RP as well. The centers are almost the same, and also, as Shankar has mentioned, centers for excellence.
Huma Qamar (Chief Medical Officer): There is an increased demand for as we have now closed the enrollment for both the programs to go as fast as we could per the protocol. You know, there will be some overlap, but there are quite a few more centers that we have identified as well.
Shankar Musunuri (Chairman, CEO, and Co-founder): The current clinical centers, Michael, just to answer, close it out, are not good enough for commercial. They're good, but we need a lot more for commercialization.
Michael Okunewitch (Senior Biotechnology Analyst): Yep. Of course. Thank you very much. I appreciate the additional clarity. Once again, congrats on all the progress.
Shankar Musunuri (Chairman, CEO, and Co-founder): Thank you.
Operator (AI Assigned): Your next questions comes from the line of Robert LeBoyer from Noble Capital Markets. Please go ahead.
Robert LeBoyer (Senior Biotechnology Analyst): Good morning, and congratulations on all of the progress that you've been making. My question has to do with the marketing and your mentions of centers for excellence. My understanding was that the product is something that can be administered by any ophthalmologist and anyone who treats patients and is very easy to fit into the current practice.
Robert LeBoyer (Senior Biotechnology Analyst): I was wondering about the centers of excellence and how you're gonna launch the product, if it's going to be a broad launch or focused on specific areas or treatment centers in the marketplace?
Shankar Musunuri (Chairman, CEO, and Co-founder): Yeah. I mean, good question. I mean, obviously, this vitrectomy, it's not a big surgery. I think any of our 2,400 trained, well-trained retinal surgeons in our country can do that. However, you know, it's a one and done treatment. It's a one-time administration for life. We want to make sure whatever center they're going to participate in the commercial and that they get trained on the same surgical manual we have been using in the clinical trials, which has been worked successfully. That's the goal.
Shankar Musunuri (Chairman, CEO, and Co-founder): I mean, once again, I just want to clarify, this is not a, you know, complex surgery, and any of those surgeons, the 2,400 smart retinal surgeons can do this. As a company, we just want to make sure we train a group of people. I mean, whatever centers we need for commercialization, it could be 100, it could be 200, we want to make sure those centers are very well-trained, and so they follow the same procedure across the board for consistency and patient safety.
Robert LeBoyer (Senior Biotechnology Analyst): Okay. You know, just my understanding was that anyone who's a retinal surgeon can administer OCU400 without any special training. Is that correct? You know, just what is the difference between the clinical trial and actual practice?
Huma Qamar (Chief Medical Officer): I can answer that, Robert. Good to hear from you. Yes, it's a vitrectomy, which is pretty common. It's part of standard of care. This is not a new treatment or anything that they have not done. What Shankar is mentioning here is actually every product has its specifications. When we are launching the centers of excellence, that would be the main centers that could further train down the next few centers, like down there. The initial launch, definitely we have all across. They're covering majority of the centers.
Huma Qamar (Chief Medical Officer): This is not a new treatment, new procedure, or single subretinal injection that we are giving. When we define centers of excellence, that's always like the initial ones that take the burden off, like, you know, for the first launch and then go towards the next. Yes, you do not need any special training. This is pretty much standard of care. We have retro retinal surgeons that are pretty good in doing this. The only difference is this. There is, of course, with any product, you come up with the guidelines, and that's what they have to do.
Shankar Musunuri (Chairman, CEO, and Co-founder): Robert, I think this is the first time. Remember, we're not talking about going after 500 patients or 1,000 patients. We're going to go after hundreds of thousands of patients with our therapies. There is a whole payer system and how you direct the patients. It's a new paradigm Ocugen has to establish. It's nothing like anybody has done before. I just wanted to make it very clear in gene therapy space.
Robert LeBoyer (Senior Biotechnology Analyst): Yes, okay. That's a very good point. Okay, thank you.
Operator (AI Assigned): Your next questions comes from the line of Whitney Ijem from Canaccord Genuity. Please go ahead.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Hey, guys. Congrats on all the progress this quarter. First question, I guess, headed into the interim for the Stargardt study in Q3, can you remind us, first of all, the powering of that study? Like, what was assumed? What should we all be expecting to see in that readout? Sorry, third part of this question is just what is the range of outcomes based on that data for the study moving forward?
Shankar Musunuri (Chairman, CEO, and Co-founder): Yeah. I think, Whitney. This is outcome analysis. It's not really giving any interim analysis like we expect in our primary endpoint and secondary endpoint analysis. This is done under strict guidance of data monitoring committee. Why do you do adaptive design? To minimize any further risk to phase 3 clinical trial. In phase 3 clinical trial, we have a true control arm, untreated arm, which will be compared with the treatment arm. The DMC looks at predictive analytics and see that study is designed for 12 months.
Shankar Musunuri (Chairman, CEO, and Co-founder): At 8 months, when 50% of the patients have completed, they're going to look at it compared to control arm. Are we going to meet the success? If there is nothing to be changed, you continue. That's outcome number 1. No changes. Top-line results come out in Q2 of next year. BLA will be filed few weeks after that. The clock starts for PDUFA date. The outcome number 2, I mean, we did recruit additional patients in the trial.
Shankar Musunuri (Chairman, CEO, and Co-founder): I mean, obviously anticipation of dropouts and all that. If the analytics show you need to increase number by a certain number, we have to again recruit. I mean, You know, that means you have to monitor those patients for additional 1 year. That has impact on the top-line results in the filing. During that timeframe, we're also, based on the discussions we had in the agency in the past, we have 2 options. Either you increase the size, and we can also look into adding an additional time point.
Shankar Musunuri (Chairman, CEO, and Co-founder): From 12 months, you also can add 16 months time point. You can also look into predictive analytics and see if you're going to meet the criteria of making a success if you extend it to 16 months. Those are the 2 options we have. Obviously, the DMC with the blinded staff at Ocugen, they're going to look at it carefully and present this option to agency and get the buy-in from FDA, which they have recommended these 2 options in the past. Once the outcome is finalized by, with agency's concurrence, we're going to let the markets know.
Shankar Musunuri (Chairman, CEO, and Co-founder): Just to sum it up, 1 of the outcomes is there's no change. We're on track. Everything is looking good. Another outcome is there is some delay, but the delay also minimizes any risk to the clinical trial we're adjusting. There's a delay of, you know, 6 months. Those are the outcomes we'll discuss with the market.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Got it. Okay, that's really helpful. Can, going back to the just, powering question, what have you disclosed the powering of the study, on the primary endpoint?
Shankar Musunuri (Chairman, CEO, and Co-founder): Go ahead, Huma.
Huma Qamar (Chief Medical Officer): Yes. Yes. Basically, we have for the Stargardt disease, 51 subjects, 34 treatment, 17 control. Adaptive design would be for a 24/16 treatment, 8 control. Yes, it's adequately powered around 90% or more. We are going to look at the powering once again, once the adaptive, you know, analysis interim outcome is going to be there. However, we are sufficiently powered, keeping in mind the prevalence as well as the no approved product right now in market. The interim outcome would be either sample size re-estimation or no.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Got it. Okay. That's helpful. Then, just to double-check, in terms of the range of outcomes, there's no, I guess, upside scenario, right? Just to make sure we're fully thinking through all the scenarios. Best case scenario is things are on track. There's not, like a best case, Oh, we're gonna end early, or something like that, right?
Shankar Musunuri (Chairman, CEO, and Co-founder): Yeah, that's right.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Okay.
Shankar Musunuri (Chairman, CEO, and Co-founder): Yeah, I think, I mean, unless. Yeah. I mean, Whitney, if you look at many products which got approvals under, you know, some of them got approvals with 30 patients, right? With orphan diseases, I mean, obviously, with the, as Huma stated, it's 50% reaching 8 months. 8 months, I think we have to be also be practical. I mean, if it's a 1 year, it's a different story. For gene therapies, for us, modified genes to start working, you know, minimum you need, like, 6 months to show something.
Shankar Musunuri (Chairman, CEO, and Co-founder): At 1 year timeframe, you really, reach the effect size and everything else. Obviously, again, the data will tell, and we'll wait. For orphan diseases, you're right. If there's a significant unmet medical need, even the sometimes in the interim, if you really hit it out of the park, agencies will consider that. I also want to be practical about it. I think 8 months is for adjustment, but typically it's good to look at it 1 year.
Shankar Musunuri (Chairman, CEO, and Co-founder): 1 year itself, if you look at all our clinical trials compared to, you know, other things going on in the industry across all our 3 programs, we are doing 1-year trials compared to anybody out there. Most of the people do 2-year trials because we're able to show effect size, treatment effect, and benefit in 1 year.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Yep. That makes perfect sense. Okay. Just moving on, a cash question. What does the new guidance into 2028 assume in terms of GA Phase 3 spend?
Rita Johnson-Greene (CFO): Yeah. It's included within the GA. The GA Phase 3 spend is included in the new cash runway into Q1 or into 2028. Yeah, we do anticipate being able to cover the expenses for that trial.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Okay. Got it. I guess could you, I know the conversations with the agency are ongoing, but what was assumed in terms of size of that study just for cash reasons or any design characteristics you can talk about at this point, just for, again, from a cash estimation perspective?
Shankar Musunuri (Chairman, CEO, and Co-founder): It's a 300 patients global trial in US, EU, and Canada together.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Got it.
Shankar Musunuri (Chairman, CEO, and Co-founder): Most of the patients or majority will be in US because we have the existing centers. It's 2-to-1 ratio. 200 in treatment and 100 in untreated control. It's powered at over 95%.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Yeah.
Shankar Musunuri (Chairman, CEO, and Co-founder): for the primary endpoint.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Got it.
Shankar Musunuri (Chairman, CEO, and Co-founder): 92% for easy ellipsoid zone secondary endpoint. It has adaptive design. At 150 patients reach 1 year, we can take a look.
Whitney Ijem (Managing Director and Senior Biotechnology Analyst): Got it. Okay, perfect. That's it for me. Thanks so much.
Operator (AI Assigned): Your next questions comes from the line of Swayampakula Ramakanth from H.C. Wainwright. Please go ahead.
Swayampakula Ramakanth (Managing Director and Senior Healthcare Analyst): Thank you. This is RK from H.C. Wainwright. Good morning, Shankar and team. A lot of my questions have been answered, but I have a couple of them. On the OCU400, you know, where you're planning to start the rolling BLA in Q3, can you confirm your PPQ runs would be completed, you know, in time, like, by end of Q2 or so that you can initiate your rolling BLA?
Shankar Musunuri (Chairman, CEO, and Co-founder): Yeah, absolutely. We're on target to complete them this quarter.
Swayampakula Ramakanth (Managing Director and Senior Healthcare Analyst): Okay. Perfect.
Shankar Musunuri (Chairman, CEO, and Co-founder): Yep, and support the rolling BLA submission.
Swayampakula Ramakanth (Managing Director and Senior Healthcare Analyst): Okay, great. Rita, in terms of expenses, we have seen G&A and R&D expenses go up this quarter, which is understandable. How should we think about this going forward, especially into 2027, as you're preparing for commercialization? Relatedly, on the BD side of things, what progress has been made especially for OCU400 and OCU410 in terms of ex-US licensing?
Rita Johnson-Greene (CFO): Yeah. Thank you for your questions. First of all, just to kind of address the, you know, the spend that you're seeing, you have to consider part of it is due to timing, and then also related to, you know, us exceeding some of our programmatic milestones, right? If you're looking at, you know, OCU410ST and OCU410, we accelerated those timelines.
Rita Johnson-Greene (CFO): When you think about, you know, us completing our enrollment for OCU400 and OCU410ST, the completion of that enrollment in Q1 will now enable us to kind of ramp down the clinical spend going into the balance of the year. We feel really confidently about, you know, the anticipated spend that we have in 2026 going into 2027. We're, you know, averaging around $50 to 60 million per year from a spend perspective, which is why we believe that our cash runway is into 2028.
Rita Johnson-Greene (CFO): Then going to the business development, we are, you know, actively evaluating various BD deals for ex-US for both OCU400 and OCU410ST. You know, we have term sheets that we're looking at, so, you know, there's a lot of work going on, just as Shankar said, that Abhi is doing, who leads our business development team, to ensure that, you know, we are evaluating those alternatives and then making the best decision for Ocugen and for our shareholders.
Rita Johnson-Greene (CFO): Lastly, from a commercialization perspective for OCU410 GA, we are still looking to commercialize that with a partner. Although, you know, as we talked about the spend associated with the clinical trials, we, you know, we have that incorporated into our runway.
Swayampakula Ramakanth (Managing Director and Senior Healthcare Analyst): Thank you. One last question. Shankar, I know in the past we have talked a little bit about payers and how to get payers agreed to the pricing that you would come up with. Any commentary, you know, especially from recent conversations from your payers as you're getting closer, you know, to commercialization, especially with the price tag that you are thinking for some of your drugs?
Shankar Musunuri (Chairman, CEO, and Co-founder): Yeah. I mean, obviously, there is a publication in Retina, I mean, that talks about potential pharmacoeconomic model, justifying $1 to 2 million, somewhere in the range price tag. There is a publication from New England Journal of Medicine, this is again, we're happy to state, CMS and CMMI, they're looking into, you know, what is the next iteration of sickle cell model they created. The publication clearly goes into payer time and subscription models, which can help with the budgetary constraints we have.
Shankar Musunuri (Chairman, CEO, and Co-founder): How can we work? Everything is very creative, RK, as I mentioned before, from launch, you know, I mean, treatment centers to, and how the CMMI is looking. I'm very pleased to say obviously, these are one-time treatments, right? We need to think about some creative ways. How can we work with payers, and make sure, especially if CMS, the government is spending a lot of money for orphan diseases or diseases like GA, which will help. Most of the patients are above 60, and they're going to get a bigger chunk of economy and budget from CMS.
Shankar Musunuri (Chairman, CEO, and Co-founder): We need to really think into all those options, how we can provide market access to more patients and who need them. Then, that NEJM article coming out of CMS is a good one. It goes into, okay, if the price tag is high, you pay over time, because if you say your therapy is one-time treatment for life, you stand by it. Those are very good models coming out. I mean, it's a very creative thinking, and we are aligned with that strategy.
Swayampakula Ramakanth (Managing Director and Senior Healthcare Analyst): Perfect. Thank you. Thanks for taking all my questions.
Operator (AI Assigned): Your next questions comes from the line of Daniil Gataulin from Chardan. Please go ahead.
Daniil Gataulin (Senior Equity Research Analyst): Yes. Hi, good morning, guys. Thank you for taking my question. I have a more general question on ellipsoid zone preservation. It appears to be emerging as an important endpoint. Wanted to ask, in your conversations with the regulators, what would you say their most recent position is on the importance of ellipsoid zone preservation? 2, are there any differences in how US and EU regulators are thinking about ellipsoid zone preservation? Thank you.
Shankar Musunuri (Chairman, CEO, and Co-founder): Daniel, good question. We just submitted our meeting request to both FDA and EMA. Obviously, I will let you know by early Q3, the input and alignment with them. I mean, obviously you saw GA trial, what we publicly stated. Our primary endpoint is lesion because that's an approved endpoint in two commercial products in US. The secondary endpoint is we are proposing is ellipsoid zone because it correlates to visual function. We believe that should satisfy them, EU regulators.
Shankar Musunuri (Chairman, CEO, and Co-founder): Obviously, we're going to wait until we complete all the meetings, everything buttoned up and aligned, then we'll let the markets know.
Daniil Gataulin (Senior Equity Research Analyst): Got it. Thank you.
Operator (AI Assigned): That will conclude our question and answer session. Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.
