Ocugen - Earnings Call - Q1 2025

Executive Summary

• Q1 2025: Revenue was $1.48M, net loss was $(15.35)M or $(0.05) per share; operating expenses were $16.0M; cash and restricted cash were $38.1M with runway into Q1 2026.
• Pipeline momentum: EMA granted eligibility to submit an OCU400 MAA via the centralized procedure (ATMP), OCU410ST received FDA alignment for a Phase 2/3 pivotal confirmatory trial (target BLA 2027), and OCU410 Phase 2 dosing completed ahead of schedule (target Phase 3 in 2026; BLA 2028).
• Estimates context: Wall Street consensus estimates for Q1 2025 EPS and revenue were not available via S&P Global; therefore no beat/miss assessment versus estimates is possible (S&P Global; no consensus available) [functions.GetEstimates].
• Near-term catalysts: OCU400 Phase 3 enrollment completion remains on track for 1H 2025 with filings targeted mid-2026; OCU410ST Phase 2/3 initiation mid-2025; OCU410 (ArMaDa) Phase 2 interim data expected in fall 2025; OCU500 Phase 1 (NIAID-sponsored) initiation targeted for Q2 2025.

What Went Well and What Went Wrong

What Went Well

• Regulatory de-risking for OCU400: “EMA granted eligibility to submit our OCU400 MAA via centralized procedure as an ATMP,” which management called “a significant project milestone” supporting 2026 filings.
• Portfolio execution across three gene therapies: “All three gene therapies are advancing through the clinic, and we're on track to meet our goal of three BLAs/MAAs in the next three years” (OCU400 ’26, OCU410ST ’27, OCU410 ’28).
• Safety/tolerability updates: OCU410 Phase 2 DSMB review with “no serious adverse events related to OCU410,” and OCU200 Phase 1 DSMB greenlight to continue (favorable safety signal).

What Went Wrong

• Cash draw and higher R&D as programs advance: Cash declined to $38.1M from $58.8M QoQ; R&D increased to $9.5M from $6.8M YoY, lifting total OpEx to $16.0M (vs. $13.2M YoY).
• Limited financial leverage from revenue: Revenue remained de minimis ($1.48M), with loss from operations $(14.50)M; net loss per share unchanged at $(0.05), reflecting pre-commercial stage and modest collaboration revenue.
• Data visibility: No interim look for the blinded OCU400 Phase 3 trial; EMA alignment for OCU410ST clinical design still pending, tempering near-term data catalysts and European clarity for Stargardt.

Transcript

Operator (participant)

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 speaker's commentary, there will be a question-and-answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin.

Tiffany Hamilton (Head of Corporate Communications)

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 investor 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, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, 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. Such 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 filing with the Securities and Exchange Commission, the SEC, 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.

Shankar Musunuri (Chairman, CEO and Co-founder)

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 are 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 and 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 with a large patient population globally, millions, instead of 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 affirm the modifier gene therapy approach. We started off the year with a two-year safety and efficacy data from the phase I/II OCU-400 clinical trial that further supports the gene-agnostic mechanism of action of OCU-400, a broad RP treatment not restricted to specific mutations with durability. It was especially gratifying to reach an alignment with the FDA for the OCU-410ST phase II/III parenteral confirmatory trial, which can be the basis of a BLA submission in 2027. Accelerating the clinical timeline of OCU-410ST will save significant costs and potentially address disease burden even sooner than anticipated. Finally, dosing was complete.

We are out of schedule in the phase II portion of the OCU-410 phase I/II RMRO clinical trial for geographic atrophy, late-stage dry AMD. We are planning to initiate the phase III clinical trial in 2026 and target with a BLA submission in 2028. I will now provide additional details around the program updates. OCU-400 has the potential to treat approximately 300,000 RP patients in the U.S. and EU and 1.6 million patients globally using a gene-agnostic approach delivered via 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 a 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 I-II clinical trial of OCU-400 for RP demonstrates a durable and statistically significant, with a p-value of 0.005, improvement in the visual function (LLVA) in all evaluable treated subjects at two years when compared to untreated eyes. Additionally, 100%, 10 out of 10 of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes. The phase III study, spanning one year, will enroll 150 participants divided into two study comps: 75 participants with the row gene mutations and 75 participants who are gene-agnostic. In each, participants will be randomized in a two-to-one ratio to receive either treatment or remain in an untreated control group, respectively.

The OCU-400 phase III limelight clinical trial is open to all eligible RP patients, only to advanced stage RP, including pediatric subjects age five plus, regardless of gene mutation, syndromic, and 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 tracking 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 III clinical trial, is progressing well. Just this week, EMA granted eligibility to submit our OCU-400 MAA via a centralized procedure as an ATMP based on the current study design and statistical analysis plan. This is a significant project milestone for OCU-400.

EMA has appointed a project lead to support any queries related to the application, and six months prior to MAA submission, a rapporteur and a co-rapporteur will be appointed to support the application. This eligibility grant is a testament to EMA's recognition of the potential of OCU-400 to address 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 U.S. and EU and 1 million globally. The FDA's decision to approve a phase II-III trial for registration underscores the potential of OCU-410ST to meet this critical unmet medical need and has never been explored in clinical trials for Stargardt disease. Data from the OCU-410ST phase I Guardian clinical trial has shown significant improvements in both structural and functional outcomes. Additionally, OCU-410ST has consistently demonstrated a very favorable safety and tolerability profile.

In BCVA, treated eyes demonstrate a two-line or 10-letter gain in the visual acuity compared to untreated eyes and a statistically significant p-value of 0.02 improvement in visual function when compared to untreated fellow eyes. In the latest data, atrophic lesions grew slower by 54% at six months and by 103% at 12 months in evaluable treated eyes when compared to untreated eyes. The phase II-III pivotal confirmatory clinical trial, which includes an adaptive design with an interim mass analysis at eight months, will randomize 51 subjects, 34 of whom will receive a single subretinal injection of OCU-410ST in the eye with worst 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 best-corrected visual acuity, BCVA, and LLVA compared to untreated controls.

One-year data will be utilized for the BLA filing. OCU-410, 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 OCU-410 is to provide a comprehensive and durable solution, a potential one-time therapy for life for the 2-3 million people in the U.S. and Europe and 8 million globally suffering GA. In February, dosing was complete in the phase II portion of the OCU-410 phase I-II RMRO clinical trial for GA. In evaluable subjects, OCU-410 12-month data demonstrates a four-line or 23-letter gain in the visual acuity. There was 41% slower GA lesion growth in treated eyes versus untreated fellow eyes after a single injection.

Furthermore, at 12 months, OCU-410 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 II study, the safety and efficacy of OCU-410 in patients with GA secondary to dry AMD will be assessed. Fifty-one patients were randomized 1-1-1 into either of two treatment groups, medium or high dose, or a control group. In the treatment group, subjects received a single subretinal 200 microliter administration of medium dose or high dose, while the control remained untreated. The Data and Safety Monitoring Board has evaluated safety in all 51 patients from the phase II clinical trial. Today, there have been no serious adverse events related to OCU-410. Phase II 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 I clinical trial for OCU-200, the company's biologic product candidate for diabetic macular edema. We are currently dosing the second cohort based on first cohort safety data. Ocugen plans to complete the phase I clinical trial in the second half of 2025. The company intends to initiate the phase III trial for NEOCAR, contingent on adequate availability of funding and/or based on the potential of a future partnership. Lastly, the investigational new drug application is in effect for OCU-500, and the National Institute of Allergy and Infectious Diseases intends to initiate a phase I 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. Ramesh.

Ramesh Ramachandran (Chief Accounting Officer)

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 total $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.

Tiffany Hamilton (Head of Corporate Communications)

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

Operator (participant)

Thank you. We will now begin the question and answer session. If you would like to ask a question, please press star one on your telephone keypad to raise your hand and join the queue. To withdraw your question, simply press star one again.

If you are called upon to ask your question and are listening via loudspeaker on your device, please pick up your handset and ensure that your phone is not on mute when asking your question. Again, press star one to join the queue. Your first question comes from the line of Michael Okunewitch with Maxim Group. Your line is open.

Michael Okunewitch (Senior Biotechnology Analyst)

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

Huma Qamar (Chief Medical Officer)

Good morning. This is Huma Qamar, same Ocugen. Thanks for your question.

For our limelight OCU-400 301 phase III 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. Thank you.

Michael Okunewitch (Senior Biotechnology Analyst)

All right. Thanks for that answer. 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 out and expand to support the filing and commercialization stage of this product.

Shankar Musunuri (Chairman, CEO and Co-founder)

Yeah. 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. For BLA submission, we have to complete process validations at the commercial scale, which we are on target this year.

That is what is needed. Obviously, we have plenty of capacity for commercial launch of this product, XUS. 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 U.S. and get it ready for launching our commercial supplies. Arun, you want to go into more details on this year's plans?

Arun Upadhyay (CSO)

Yeah. Thanks, Shankar. As you stated, we are on track to complete the process validation activity and get ready for BLA submission next year. For initial commercial launch, we will be using our partner, Consigno Bio, for the OCU-400 project. Our plan is, 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 have completed construction last year. Subsequently, we plan to release the product from our second site in the U.S., that's our strategy to support the future commercialization.

Michael Okunewitch (Senior Biotechnology Analyst)

All right. Thank you. Just one more from 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 OCU-200 program? I know it's not core, but DME has been an exciting space.

Shankar Musunuri (Chairman, CEO and Co-founder)

Yeah. I think the clinical trial, we're planning to complete the later part of this year. Our goal is, before the end of the year, we'll provide a clinical update, including preliminary efficacy and safety.

Michael Okunewitch (Senior Biotechnology Analyst)

All right. Thank you so much. Once again, congrats on all the progress.

Shankar Musunuri (Chairman, CEO and Co-founder)

Thank you.

Operator (participant)

Next question comes from the line of Robert LeBailleur with Nobel. Your line is open.

Robert LeBailleur (Analyst)

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 410-ST and the clinical trial going on. Am I correct on that?

Shankar Musunuri (Chairman, CEO and Co-founder)

Your question, Robert, related to 410-ST, yeah. I mean, the morning we actually talked during this, we have a recent communication from EMA on OCU-400, just to clarify. For submission of the MAA, and that's the path, and it's going well. That's what we talked about. As far as OCU-410ST is concerned, the clinical trial we designed with FDA, which will be a registration trial, which we are going to embark on shortly. We are discussing that strategy with the EMA. We do not have the final answer yet.

Obviously, see, this is a much-needed product, significant unmet medical need, just as RP, Stargardt disease. There's no approved treatment in the EU. We are hoping they will align with FDA clinical trial design and also agree. Again, we are in negotiations with the EMA. When we have an answer from them, we'll let the markets know.

Robert LeBailleur (Analyst)

Okay. Great. 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 and the overhaul going on with 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.

Shankar Musunuri (Chairman, CEO and Co-founder)

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's in direct contact with NIAID. Go ahead.

Arun Upadhyay (CSO)

Thank you, Shankar. Yes. I think we don't see any impact on us related to the changes you just mentioned happening at the government end. We are constantly in interaction with NIAID, and we are collaboratively working with them to finalize the clinical study plan as well as supporting the required material to initiate the phase I study. We are on track.

As Shankar mentioned, we are hoping to initiate the phase I study in the second quarter this year. We are on target for that. Our relationship has been great. I would say I think it has improved even, actually. Our engagement with NIAID has increased significantly to initiate the study.

Robert LeBailleur (Analyst)

Okay. Great. Thank you very much.

Operator (participant)

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

Ramakanth Swayampakula (Managing Director)

Thank you. Good morning, Shankar and team. A couple of quick questions on the limelight study. Does the phase III study have an interim look? If so, what's the general timing that we should expect that to be?

Huma Qamar (Chief Medical Officer)

This is Huma. Thanks for the question. There is no interim look in the study.

Once the enrollment is complete and we'll be giving periodic updates, there are 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. Thank you.

Ramakanth Swayampakula (Managing Director)

Thank you for that, Huma. A quick clarification on the design, because I was—maybe I misheard it. I thought the randomization for the limelight study was 2:1, but.

Huma Qamar (Chief Medical Officer)

Yes.

Ramakanth Swayampakula (Managing Director)

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

Shankar Musunuri (Chairman, CEO and Co-founder)

Arun K, just a clarification. The 1:1:1 is for the currently ongoing clinical trial for OCU-410 targeting dry age-related macular degeneration. They have medium dose, high dose, and control in that phase II.

Ramakanth Swayampakula (Managing Director)

Perfect. Perfect.

One last question from me is on the three modifier gene therapy programs, what clinical data updates would we see in 2025? I am 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.

Shankar Musunuri (Chairman, CEO and Co-founder)

I think from OCU-400 perspective, as Huma stated, it is a blinded phase III trial. You will get periodic updates on how the trial is progressing. Other than that, the data will not come out until next year, until the trial is completely done. The second program, OCU-410, Armarra trial for dry AMD, and we are expecting interim results sometime this fall. OCU-410ST, obviously, the registration trial is going to start shortly in the next couple of months. We are going to provide when we have a complete 12-month data from phase I on this study.

In addition, OCU-200, the phase I trial should be complete late this year, and we'll provide the data from the trial too.

Ramakanth Swayampakula (Managing Director)

Perfect. Thanks. Thanks for taking all my questions.

Shankar Musunuri (Chairman, CEO and Co-founder)

Thank you.

Operator (participant)

Next question comes from the line of Daniil V. Gataulin, Chardan Capital Markets. Your line is open.

Good morning. This is Stephen on for Daniil. Thanks for taking my question. For OCU-400 for RP to file in Europe, do any of the patients need to be treated in Europe, or is the data from the U.S. and Canada going to be sufficient?

Tiffany Hamilton (Head of Corporate Communications)

I'll take that question. That's a great question, actually. As we have stated in the past as well, no additional trial is required in Europe. If we get the approval in the U.S. based on our primary endpoint and the study design, U.S. and Canada is sufficient.

In fact, U.S. is sufficient for our approval in Europe. So no additional trials are required. This trial has sufficient representation for global mutations as it's the only broad RP indication gene agnostic trial globally.

Great. Thank you.

Operator (participant)

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.

Shankar Musunuri (Chairman, CEO and Co-founder)

Thank you, Operator. Our efforts in the first quarter of the year evidence 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 look forward to what this quarter and the rest of the year holds for the company, our people, patients, and shareholders. Thank you.

Tiffany Hamilton (Head of Corporate Communications)

Thanks again, everyone. Goodbye.

Shankar Musunuri (Chairman, CEO and Co-founder)

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