管理層發言
Good morning, and welcome to Ocugen's Second Quarter 2026 Financial Results and Business Update. I will now turn the call over to Chris Clark, Ocugen's Head of Communications. You may begin.
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 June 30, 2026; Abhi Gupta, Executive Vice President of Commercial and Business Development; and Dr. Mohamed 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.
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 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.
Thank you, Chris, and good morning, everyone. The second quarter was a defining one for Ocugen. The FDA cleared our Phase 3 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. And from the closing of $130 million convertible notes financing, we extended our cash runway into 2028, now able to support all 3 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 in 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 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 3 separate drugs. We're advancing 1 platform across 3 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 United States and Europe, a combined patient population, and the commercial opportunity far larger than anything currently served by approved gene therapies in ophthalmology.
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 3 BLAs by 2028. This positions the first half of 2027 as a catalyst-rich 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. Then I will hand over the call to Rita for financials. Starting with OCU410 for GA, a secondary to dry age-related macular degeneration or dry AMD. GA represents our largest commercial opportunity with approximately 2 million to 3 million patients in the U.S. and Europe combined. There are currently no approved treatments for GA in Europe. 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 4 distinct pathways that contribute to the progressive degeneration of the macula, drusen, inflammation, oxidative stress and complement RORA activation. The currently approved therapies in the U.S. address only 1 of these 4 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. 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 Marketing 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 millimeter square and 17.5 millimeter square. The criteria to be used in our Phase III pivotal trials 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. Importantly, these Phase II data help 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 1, we announced the completion of enrollment and dosing in our Phase II/III GARDian3 pivotal confirmatory trial, enrolling 63 participants. We expect the interim outcome decision for the first 50% of subjects at 8 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. 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 2:1 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 modifier gene therapy platform. The primary endpoint is 12-month change in visual function assessed by Luminance Dependent Navigation Assessment, or LDNA. Subjects are followed for 1 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, PPQ, 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. 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 are 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 all 3 late-stage development programs. While OCU410 for GA represents our largest commercial opportunity, we believe all 3 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 5 key areas. First, we're in discussions with CMS and peers to establish early market access and reimbursement strategies. 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?
Thank you, Shankar. Good morning, everyone. Total operating expenses for the 3 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 3 months ended June 30, 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 6 months ended June 30, 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 6 months ended June 30, 2025, of $31.2 million, which included research and development expenses of $17.9 million, and general and administrative expenses of $13.2 million.
Ocugen reported a $0.07 net loss per common share for the 3 months ended June 30, 2026, compared to a $0.05 net loss per common share for the 3 months ended June 30, 2025. On our capital position, following the closing of the $130 million convertible notes financing, the company's cash, cash equivalents, and restricted cash totaled $100.4 million as of June 30, 2026, extending our cash runway into 2028. The company has 339 million shares of common stock outstanding as of June 30, 2026. That concludes my financial update. Shankar, back to you.
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, and 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 3 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?
分析師問答
Our first question comes from the line of Michael Okunewitch with Maxim Group.
Congrats on all the great progress. So, I wanted to ask, you now have a handful of international partnerships, which makes OCU400 a truly international program at this point. So 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?
Michael, what we have with OCU400 is alignment from the EMA in addition to the FDA that the single trial we're doing in the U.S. is sufficient for approvals. Globally, orphan gene therapies typically receive approval based on the U.S. approval, so approvals in MENA and other regions will be linked to our U.S. FDA approval.
All right. And then I wanted to see also if you could just highlight some of the key differences in the trial design between ArMaDa3 and the Phase II ArMaDa trial?
I will let our CMO, Dr. Genead, answer that.
Thank you, Michael. Regarding ArMaDa3, our global Phase III trial for GA, we recently received FDA approval to initiate it this quarter. The Phase III trial will have one treatment arm with OCU410 versus a control with a 2:1 randomization allocation, and subjects will be followed for up to 12 months. We will assess the primary efficacy endpoint plus other key functional endpoints. ArMaDa1, the earlier Phase I/II GA trial, showed similar efficacy, so we should expect a similar outcome here. We'll examine the numbers, which will obviously differ. ArMaDa3 will enroll approximately 237 subjects in a 2:1 allocation. It will be global, conducted outside the U.S. in Europe and other regions. The primary endpoint will be very similar, so we should expect trends similar to those seen in ArMaDa1, the Phase I/IIa GA trial.
All right. And then just one last one from me before I hop back into the queue. So 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. So it will be a chronic therapy versus a one-time. But 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?
Yes. Good question, Michael. I think the way you should look at it is that our treatments are potentially one-and-done, so they will have a different pricing structure than ongoing chronic therapies. Second, everything will be dictated by data. If you have a safe one-time treatment, for example our gene therapies, we are still collecting data; in some RP patients, as they enter the second and third years they are improving further. If an oral chronic therapy comes to market, patients and providers will look to see whether the therapy simply reduces degeneration, whether it stalls progression, or whether it has the potential to reverse disease in some patients. With our modified gene therapy we are seeing all of those trends in some patients, which could be a major differentiator. Stargardt also affects many pediatric patients. The current clinical trial focuses on patients 12 and older, while our trial includes patients aged 3 and up.
There are therefore many differentiators. When it comes to pricing, because of our differentiated, disruptive platform, and because everyone will focus on safety, efficacy, and the one-and-done nature of treatment, those factors will be taken into account. I do not think we will be strictly comparing our pricing to what other chronic therapies are doing. If a product is a me-too, then yes, but if you have a truly disruptive technology that is completely different, it should be priced on its own merits.
Our next question comes from the line of Whitney Ijem with Canaccord Genuity.
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? And 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?
I will ask Dr. Genead to talk a little bit about baseline characteristics, then I'll answer the other question.
Thank you, Shankar. Whitney, yes, happy to answer. Our population was definitely broader than other competitors. First, we included patients from early to late-stage Stargardt disease. As Shankar mentioned, we also included younger subjects; we enrolled individuals 3 years of age and older, so this is a very broad population. For Stargardt, the earlier the better, especially for a progressive retinal degeneration disease. The lesion size criteria in our Phase II/III GARDian trial were broader than what we’ve seen elsewhere, encompassing both smaller and larger lesions. That aligns with our early-to-late-stage strategy for the disease. We have already enrolled some subjects in our Phase II/III trials and are excited to see the data. In terms of genetics, we included all variants and all specific mutations associated with ABCA4-related retinopathy, so the indication covers Stargardt and other ABCA4-related conditions.
Regarding functional outcomes, our Phase I data published early in the year showed a very clear slowing of structural progression in treated patients, and we also observed functional benefit. In Stargardt disease, the first objective is to halt progression and prevent further loss of retinal structure and function, which we achieved in our prior trial. The next, more ambitious goal is to reverse that progression and improve outcomes, and in our Phase I/II we saw some patients improve in visual function, with gains around six letters, close to one line, comparing treated versus untreated eyes. That is where we believe the major differentiation lies: the broader application of our molecule.
Whitney, just to clarify, the primary endpoint, because it's a 1-year trial, it's not a 2-year trial, it's still a lesion. Then 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, and so we'll have long-term data in those patients still.
Got it. Really helpful. And then just last question, and maybe Rita, this one is for you. Just 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 kind of pull levers to extend the cash runway further, how should we think about maybe the startup of GA versus commercial prep for RP or Stargardt, and how you guys are thinking about those different levers if needed?
Yes. Thank you, Whitney. So, first of all, our primary goal is to to make sure that we are minimizing shareholder dilution while evaluating opportunities to raise capital, as you said, to bring these novel products to patients. We have cash runway into 2028, and I want to remind everyone of that, which gives us the confidence to execute our clinical-stage and late-stage products and then progress to BLA submission for both OCU410 and OCU410ST in 2027, with the potential to commercialize OCU400 by the end of 2027. We do have additional levers we can pull. One is the PRV for OCU410ST given the RPD designation. We have the ability to sell that for somewhere between $100 million and $200 million, even prior to approval, and that's something we are evaluating. We are also looking at various business development deals from a globalization perspective, ex-U.S. for OCU400, OCU410ST, and even GA, depending upon what the term sheet looks like.
We're always looking for potential deals to minimize dilution. We also have the Janus Henderson warrants — another 10 million warrants at a $1.50 strike price, which could bring in another $15 million, and those warrants expire in August 2027. We anticipate a special meeting in September to increase authorized shares, which will give us the ability to raise additional equity if we decide to do so. So again, we're looking at both non-dilutive and dilutive options to make sure we can bring these novel products to patients while maximizing shareholder value.
Our next question comes from the line of Charles Wallace with H.C. Wainwright.
This is Charles from H.C. Wainwright, calling for RK. I have a question about the ArMaDa3 design. It appears that, based on the prior earnings call, the study has been slightly resized. Previously you said it would be about 300 patients and now it's 237. Was this change requested specifically by the FDA, or did you propose it? Also, do the assumptions change regarding effect size, variability, dropout, or the narrower lesion size compared to Phase II?
Yes, Dr. Genead?
Thank you, Charles. Yes, we had a discussion with the agency, the FDA. So all this being aligned and discussed with the FDA. But to answer your question specifically, it was based on the sample size estimation and also the power calculation we did. So the estimate you're citing, the 300, was based on estimate. But when we saw the effect size based on our ArMaDa1, the Phase I/II trial, as we discussed today, we saw the 31% reduction in the median 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's based on the rate of change, the slope analysis for the primary efficacy. So, 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. So all this has been discussed and aligned, and as we announced today, we got the clearance from the FDA to initiate our Phase III trial in the next few weeks.
Very helpful. And then I guess for the rolling submission: I think originally the guidance was to submit in the third quarter, and now I believe it's the first quarter after the liMeliGhT data. I guess my question is, what changed between submitting the non-clinical module earlier compared to after the top-line data of the liMeliGhT?
Charles, I think from our perspective, we're ready. I mean, I think we are doing very well with our PPQs as we've mentioned. There are a lot of gene therapy companies stuck with CMC. We're ahead of the game. We used to 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. I mean, obviously, this is where we have to work with agency when they're comfortable. And that's the timeline they gave us, and we're going to be fine with that. The reason is, I just want to clarify, it's good to have rolling submission that gives a head start for agency, okay? It's for their own benefit. And if they want to wait until next year, I mean, we are ready to file it as soon as the top line comes for the pre-BLA meeting. We may still give them a head start of maybe a month or 2 months before we drop the clinical section.
So however, I just want to clarify, until the final BLA is completed with the clinical section, the PDUFA date, the accelerated clock of 6 months doesn't start. I just want to clarify that. So 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 are fine with it. I think we're ready from our perspective, and we will work with them closely in collaborative way. And whenever we have a top line, we'll be ready to file it.
Great, very helpful.
So it doesn't change any filing clock. As we mentioned before, we expect to complete the BLA filing in the second quarter, with anticipated approval in the fourth quarter under the six-month accelerated clock.
Our next question comes from the line of Robert LeBoyer with NOBLE Capital Markets.
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 launches based on that. But 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?
Yes, Robert, absolutely. Because 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 2 modules, non-clinical and CMC modules. So that will still give them a head start. And as soon as the clinical module is done when you file it, the PDUFA date starts. So that's basically our plan is to file that in second quarter, so 6 months' clock should be fourth quarter, approval clock.
And at this time, we have no further questions. I would like to turn the call back over to the Ocugen team for closing remarks.
Thank you all for attending today's webcast. Really appreciate all our investors, shareholders, patients, providers. Thank you.
This concludes today's conference call. You may now disconnect. Have a good day.