Ocugen, Inc. Q1 FY2025 Earnings Call

Good morning, and welcome to Ocugen's First Quarter 2025 financial results and business update. Please note that this call is being recorded at this time. All participant lines 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 Tiffany Hamilton, Ocugen's 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. Ramesh Ramachandran, our Chief Accounting Officer, is also on the call to provide a financial update for the quarter ended March 31, 2025. Dr. Huma Qamar, our Chief Medical Officer, will be available to answer questions following the presentation. This morning, we issued a press release detailing associated business and operational highlights for the first quarter of 2025. We encourage listeners to review the press release, which is available on our website at ocugen.com. This call is being recorded, and a replay with the accompanying slide presentation will be available on the Investors section of our website for approximately 45 days. This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believe, potential, propose, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, or should, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements include, but are not limited to, statements regarding our clinical development activities and related anticipated timelines. These statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission, including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this presentation speak only to the date of the presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events, or otherwise, after the date of this presentation. Finally, Ocugen's quarterly report on Form 10Q covering the first quarter of 2025 was filed today. I will now turn the call to Dr. Musunuri.

Thank you, Tiffany, and thank you all for joining us today. As detailed in our press release, we are excited to discuss the substantial progress of our game-changing modifier gene therapy platform. All three gene therapies are advancing through the clinic, and we're on track to meet our goal of three biologics license application/market authorization application filings in the next three years. Modifier gene therapies offer potential cures for life. Unlike traditional gene therapies or gene editing, modifier gene therapies have the ability to regulate gene networks, reset homeostasis, restore the functional network, and create a healthy environment for retinal cells to survive. Since photoreceptors in retinal cells are non-dividing, creating a healthy environment is a powerful concept, and cells can potentially survive for life with this therapy. Today, based on the unique mechanism of action, these therapies target diseases affecting large patient populations globally—millions—rather than a very small group of hundreds to a few thousand patients as with the traditional gene therapies in the market today. Positive clinical data continues to support the multi-layered gene therapy approach. We started off the year with two-year safety and efficacy data from the Phase 1/2 OCU400 clinical trial that further supports the gene-agnostic mechanism of action of OCU400, a broad RP treatment not restricted to specific mutations with durability. It was especially gratifying to establish alignment with the FDA for the OCU410ST Phase 2/3 pivotal confirmatory trial, which can be the basis of a BLA submission in 2027. Accelerating the clinical timeline of OCU410ST will save significant costs and potentially address disease burden even sooner than anticipated. Finally, dosing was completed ahead of schedule in the Phase 2 portion of the OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy late-stage dry AMD. We are planning to initiate the Phase 3 clinical trial in 2026, on target with a BLA submission in 2028. I will now provide additional details around the program updates. OCU400 has the potential to treat approximately 300,000 RP patients in the US and EU and 1.6 million patients globally using the gene-agnostic approach delivered via a single subretinal injection to target all 100 genes associated with RP. A traditional gene therapy approach would require developing 100 different products to address all 100 genes, which is not commercially viable. More significantly, recent news affirms that the traditional gene therapy approach is ineffective in achieving clinically meaningful outcomes for treating RP. The positive two-year long-term safety and efficacy data from the Phase 1/2 clinical trial of OCU400 for RP demonstrates durability and statistically significant improvement in the visual function LLVA in all evaluable treated subjects at two years when compared to untreated eyes. Additionally, 100%, or 10/10, of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes. The Phase 3 study spanning one year will enroll 150 participants divided into two study cohorts: 75 participants with the raw gene mutations and 75 participants who are gene agnostic. In each arm, participants will be randomized in a 2:1 ratio to receive either treatment or remain in an untreated control group, respectively. The OCU400 Phase 3 broad clinical trial is open to all eligible RP patients, early to advanced stage RP, including pediatric subjects age five and older, regardless of gene mutation, syndromic or non-syndromic forms of RP. Two key parts of our potential BLA/MAA filings next year are on schedule. The first is manufacturing, and we are on track to complete process validations this year in support of registration. This material can also be used for commercial supply in 2027. The second part, the Phase 3 clinical trial, is progressing well. Just this week, the EMA granted eligibility to submit our OCU400 MAA via centralized procedure as an ATMP based on the current study design and statistical analysis plan. This is a significant project milestone for OCU400. The EMA has appointed a project lead to support any queries related to the application, and six months prior to the MAA submission, the rapporteur and co-rapporteur will be appointed to support the application. This eligibility grant is a testament to the EMA's recognition of the potential of OCU400 to address a serious unmet medical need in Europe. There is currently no approved treatment option for Stargardt disease that encompasses more than 1,200 mutations and affects 100,000 people in the US and EU and 1 million globally. The FDA's decision to approve a Phase 2/3 trial registration underscores the potential of OCU410ST to meet this critical unmet medical need, which has never been explored in clinical trials for Stargardt disease. Data from the OCU410ST Phase 1 GUARDIAN clinical trial has shown significant improvements in both structural and functional outcomes. Additionally, OCU410ST has consistently demonstrated a very favorable safety and tolerability profile. In BCVA, treated eyes demonstrate a two-line or 10-letter gain in visual equity compared to untreated eyes and a statistically significant improvement with a P-value of 0.02 in visual function compared to untreated LOIs. In the latest data, atrophic lesions grew slower by 54% at six months and by 103% at 12 months in evaluable treated eyes compared to untreated eyes. The Phase 2/3 pivotal confirmatory clinical trial, which includes an adaptive design with an interim MAST analysis at eight months, will include 51 subjects, 34 of whom will receive a single subretinal injection of OCU410ST in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. The primary endpoint in the clinical trial is change in atrophic lesion size. Secondary endpoints include visual acuity as measured by test-corrected visual acuity, BCVA, and LLVA compared to untreated controls. One year data will be utilized for the BLA filing. OCU410, specifically designed to address multiple pathways implicated in the pathogenesis of dry AMD, offers a distinct advantage over current treatments that target only one cause of GA, require multiple injections per year, and are accompanied by various safety concerns. Our goal with OCU410 is to provide a comprehensive and durable solution, a potential one-time therapy for life for the 2 million to 3 million people in the US and Europe and 8 million globally suffering from GA. In February, dosing was completed in the Phase 2 portion of the OCU410 Phase 1/2 ArMaDa clinical trial for GA. In evaluable subjects, OCU410 12-month data demonstrates a four-line or 23-letter gain in visual acuity. There was a 41% slower GA lesion growth in treated eyes versus untreated fellow eyes after a single injection. Furthermore, a 12-month OCU410 treatment preserves more retinal tissue around the GA lesions of treated eyes compared to commercially available products given monthly or every other month. In the Phase 2 study, the safety and efficacy of OCU410 in patients with GA secondary to dry AMD will be assessed. 51 patients will be randomized 1:1 into either of two treatment groups, medium or high dose, or a control group. In the treatment group, subjects will receive a single subretinal 200 microliter administration of medium dose or high dose while the control remains untreated. The Data and Safety Monitoring Board has evaluated safety in all 51 patients from the Phase 2 clinical trial. To date, there have been no serious adverse events related to OCU410. Phase 2 interim results are expected in the fall of this year. I would also like to provide a summary of programs outside of our first-in-class modifier gene therapy platform. Earlier this year, the first patient was dosed in the Phase 1 clinical trial for OCU200, the company's biologic product candidate for diabetic macular edema. We are currently dosing the second cohort based on the safety data from the first cohort. Ocugen plans to complete the Phase 1 clinical trial in the second half of 2025. The company intends to initiate the Phase 3 trial for NeoCart, contingent on adequate availability of funding and based on the potential of a future partnership. Lastly, the investigational new drug application is in effect for OCU500, and the National Institute of Allergy and Infectious Diseases intends to initiate a Phase 1 clinical trial in the second quarter of 2025. Ocugen is continuing discussions with the relevant government agencies as well as strategic partners regarding developmental funding for its vaccine technology for flu. With that, I will now turn the call over to our Chief Accounting Officer, Ramesh Ramachandran, to provide an update on our financial results for the first quarter ended March 31, 2025.

Thank you, Shankar. Our research and development expenses for the quarter ended March 31, 2025, were $9.5 million compared to $6.8 million for the first quarter of 2024. General and administrative expenses for the quarter ended March 31, 2025, were $6.5 million compared to $6.4 million during the same period in 2024. Net loss was approximately $15.3 million or $0.05 net loss per share for the quarter ended March 31, 2025, compared to a net loss of approximately $11.9 million or $0.05 net loss per share for the first quarter of 2024. Our cash and restricted cash totaled $38.1 million as of March 31, 2025, compared to $58.8 million as of December 31, 2024. The company expects that its cash and restricted cash will provide the company cash runway into the first quarter of 2026. As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital. That concludes my update for the quarter. Tiffany, back to you.

Thank you, Ramesh. We will now open the call for questions. Operator?

Thank you. We will now begin the question and answer session. And your first question comes from the line of Michael Okunewitch with Maxim Group. Your line is open.

Thank you so much for taking my questions today. Congratulations on all the progress. So I think just first off, in terms of the liMeliGhT study, where are you at enrollment? And when do you have to complete that to reach your filing targets?

So for our liMeliGhT OCU400 GA 1 Phase 3 trial, we are on track for enrollment as planned for the first half of 2025. And we will be on track for our BLA submission a year from now in terms of clinical.

Just as we approach the completion of that first pivotal study, I'd like to see if you could just expand a little bit on manufacturing capabilities and then what you would have to build or expand to support the filing commercialization stage of this product?

Michael, I'll answer the first part, then our CSO, Dr. Arun Upadhyay, is on the call too. He can explain what our manufacturing plans are. So for BLA submission, we have to complete process validations at the commercial scale, which we are on target this year. And so that's what is needed. And obviously, we have plenty of capacity for commercial launch of this product outside the US. We also built our own facilities here in Malvern, Pennsylvania. Once we commercially launch the product in 2027, shortly after that, within two years, our goal is to get the second site in the US ready for commercial supplies. Arun, do you want to go into more details on this year's plans?

Yeah, thanks, Shankar. So as you stated, we are on track to complete the process validation activity and get ready for BLA submission next year. For the initial commercial launch, we'll be using our partner in CanSinoBio for the OCU400 project. But our plan is that subsequently, we are going to bring back the manufacturing, we'll do the tech transfer and bring the manufacturing in-house in our Malvern GMP manufacturing facility, which we completed construction last year. Subsequently, we plan to release the product from our second site in the US. So that's our strategy to support the future product lines.

And then just one more for me and I'll hop back into the queue. Could you give us an idea of when we could expect to see the next update on the OCU200 program? I know it's not core, but DME has been an exciting space.

Yes. I think the clinical trial we are planning to complete in the later part of this year. So our goal is to provide a clinical update, including preliminary efficacy and safety before the end of the year.

Next question comes from the line of Robert LeBoyer with Noble. Your line is open.

Good morning, everybody. I had a clarification on some of the prepared remarks regarding Europe and the regulation there. My understanding was that these remarks referred to 410ST and the clinical trial going on. Am I correct on that?

No, your question Robert related to 410ST. I mean the morning we actually talked during this. We have a recent communication from EMA regarding OCU400 just to clarify. And so for submission of the MAA and that's the path, and it's going well. That's what we talked about. As far as OCU410ST is concerned, the clinical trial we designed with the FDA, which will be a registration trial that we are going to embark on shortly. We are discussing that strategy with EMA. We don't have the final answer yet. And obviously, since this is a much-needed product with significant unmet medical need, just as with RP and Stargardt disease, there are no approved treatments in EU. We are hoping they will align with the FDA clinical trial design and also agree. Again, we are in negotiations with EMA. When we have an answer from them, we'll let the markets know.

Okay, great. And just separately, you had mentioned the influenza trial with NIAID and considering what's been going on with HHS and the FDA and the NIH in the past two or three months, with the overhaul going on at those agencies. Have you had any changes in the relationship or with contact with people, or any insights you can give as to what the agency is thinking going forward? I know that some of the companies that were developing COVID vaccines had grants revoked and canceled. So I was wondering if you could give us any insight as to what's going on in the divisions that you interact with?

Yes, Robert, great question. This is related to our COVID vaccine targeting intranasal versus inhalation routes. This clinical trial, NIAID is still on target to support it. In fact, I will ask Dr. Upadhyay, our CSO, to comment more on this. He is in direct contact with the NIAID. Go ahead.

Thank you, Shankar. I think we don't see any impact on us related to the changes you just mentioned that are happening at the government end. So we are constantly interacting with NIAID, and we are collaboratively working with them to finalize the clinical study plan as well as support the required material to initiate the Phase 1 of the study. So we are on track, and as Shankar mentioned, we are hoping to initiate the Phase 1 study in the second quarter this year. Our relationship has been great. I would say that it has even improved. Our engagement with NIAID has increased significantly to initiate the study.

Next question comes from the line of Swayampakula Ramakanth with H.C. Wainwright. Your line is open.

Thank you. Good morning, Shankar and team. A couple of quick questions on the liMeliGhT study. Does the Phase 3 study have an interim look? And if so, what's the general timing that we should expect that to be?

There is no interim look in the study. Once the enrollment is complete, we'll be giving periodic updates on the safety for 30 subjects each that we are following for the liMeliGhT study. However, the full data would be available once the CSR is final. As this is a blinded study, we cannot release the data before, yes.

A quick clarification on the design, because maybe I misheard it. I thought the randomization for the liMeliGhT study was 2:1.

Yes.

Okay. Because I thought I heard it as 1:1 based on Shankar’s comments, but I probably got it wrong.

RK, just a clarification, the 1:1:1 is for the currently ongoing clinical trial for OCU410 targeting dry age-related macular degeneration. So they have a medium dose, high dose, and control in that Phase 2.

One last question from me is on the three modified gene therapy programs. What clinical data updates would we see in 2025? And just trying to see if we can get any idea of how strong the data is going to look in all of the pivotal programs as we move forward?

I think from OCU400's perspective, as Huma stated, it's a blinded Phase 3 trial. You'll get periodic updates on how the trial is progressing. Other than that, the data won't come out until next year until the trial is completely done. The second program, OCU410 ArMaDa trial for dry AMD, we are expecting interim results sometime this fall. OCU410ST, obviously, the registration trial is going to start shortly in the next couple of months. We are going to provide when we have complete 12 months data from Phase 1 on this study. In addition, OCU200, the Phase 1 trial should be complete late this year, and we'll provide the data from the trial too.

Next question comes from the line of an indiscernible source.

For OCU400 for RP, to file in Europe, do any of the patients need to be treated in Europe, or is the data from the US and Canada sufficient?

So as we have stated in the past as well, no additional trial is required in Europe. If we get the approval in the US based on our primary endpoint and the study design, then US and Canada is sufficient. In fact, the US is sufficient for our approval in Europe. So no additional trials are required. This trial has sufficient representation for building mutations as it's the only broad RP indication gene agnostic trial globally.

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

Thank you, operator. Our efforts in the first quarter of the year evidenced the importance of our gene therapy programs and the significance of upcoming milestones. We remain steadfast in our mission to provide a one-time therapy for life to address considerable unmet medical needs that exist for millions of patients facing the terrifying prospect of losing their vision, and we look forward to what this quarter and the rest of the year holds for the company, our people, patients, and shareholders. Thank you.

This concludes today's conference call. Thank you all for joining, and you may now disconnect.