Ocugen, Inc. Price & Performance (OCGN)

Thank you, operator, and good morning, everyone. Joining me on today's call and webcast is Doctor. Shankar Musanuri, AlcuDent'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, will provide more detail on our financial results and Doctor. Huma Kumar, 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 fourth quarter and full year of 2024. 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 the Ocugen website for approximately forty five 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, propose, 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 preclinical and clinical development activities and related anticipated development 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 filings 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 as of the date of this 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 annual report on Form 10 K covering 2024 will be filed today. I will now turn the call over to Doctor. Musonuri.

Thank you, Tiffany, and thank you all for joining us today. We are eager to share the ongoing progress of our novel modifier gene therapy platform across all three clinical programs. It was especially exciting to announce last week that we reached an alignment with the FDA to move forward with a Phase twothree pivotal confirmatory trial for RQ410ST BLA targeting Stargardt disease, making it possible to potentially expedite our clinical development timeline by two to three years, which is expected to save significant costs in addressing disease burden even sooner than anticipated. This important news also brings us closer to our goal of three potential BLAs in the next three years, AKU400 in 2026, AKU410ST in 2027 and AKU410 in 2028. We know this is a bold ambition, but I'm confident that we have the strategic and scientific expertise along with an unrelenting commitment to patients to deliver on our commitment. During 2024, we continuously advanced our programs in line with enrollment and dosing timelines and are continuing to drive the product pipeline forward in 2025. Through our development, we are providing data to validate our revolutionary platform. To support our efforts in the clinic, we secured $65,000,000 in equity and debt financings in the second half of twenty twenty four that extends cash runway into the first quarter of twenty twenty six.

Let's discuss ARQ400, our lead candidate in more detail. Retinitis pigmentosa affects three hundred thousand people in The U. S. And EU combined and one point six million globally and is associated with mutations in more than one hundred genes. With only one product on the market that addresses one percent to two percent of patient population, you can see the ability for ARQ400 to meet a tremendous unmet medical need and potentially capture all the market share through its gene agnostic mechanism of action. In February, the European Commission provided a positive opinion from the European Medicines Agency's EMA Committee for Advanced Therapies for OQ400 Advanced Therapy Medicinal Product, ATMP classification. ATMP classification is granted to medicines that can offer groundbreaking opportunities for the treatment of disease and accelerates the regulatory review timeline of this potential on time gene therapy for life. Additionally, this classification allows Oncogen to interact with EMA more frequently for scientific advice and protocol assistance. In January, we announced positive two year safety and efficacy data from the ARQ400 Phase III clinical trial that demonstrated clinically meaningful improvement of two line gain, 10 letters on the ET DRS chart in low luminescence visual equity LLBA in treated eyes when compared to untreated fellow eyes.

This treatment effect was statistically significant with a P value of 0.005 in all subjects regardless of mutation at two years demonstrating the long term durability for IQ 400. These two year LLVA findings, which are the most sensitive measure of visual function, are consistent with the results observed at one year. The Phase III study spanning one year will enroll 150 participants divided into two study arms, 75 participants with the raw gene mutations and seventy five participants who are gene agnostic. In each arm, participants will be randomized in a two:one ratio to receive either treatment, which is 2.5x1010 VGi of RQ400 or remain in an untreated control group respectively. We're actively enrolling patients in The U. S. And Canada in the Phase III Limelight clinical trial of ARQ400 and intend to complete enrollment in the first half of twenty twenty five to remain on track to meet BLA and MAA filings targets mid-twenty twenty six. Next up is Ocuford10ST. With no approved treatments, options available for patients with Stargardt disease, one hundred thousand patients in The U. S. And Europe combined are desperate for an answer. AQ400410ST with a single subretinal injection has potential to treat Stargardt and all ABCA4 associated retinopathies and in the fourth quarter received orphan medicinal product designation from the EMA for the treatment of ABCA4 associated retinopathies.

Earlier this week, we announced that our AQ410ST also received ATMP classification along with our AQ410, which is a critical step to potentially address these severely unmet medical needs in the very near future. Six month data from Phase one of the ARQ410ST CARDIAN trial demonstrated clinically meaningful two line 10 letter improvement in visual function measured by best corrected visual equity, BCVA, which is statistically significant with a p value of 0.02 in treated eyes. 100% of available treated eyes demonstrated stabilization or improvement in visual function. There was 52% slower atrophic lesion growth in ARQ410T treated eyes versus untreated fellow eyes after single injection at six months in seven patients and 103% slower atrophic lesion growth in treated eyes versus untreated fellow eyes at twelve months in two patients. Ocuphoten ST maintains a favorable safety and tolerability profile with no serious adverse events, including no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endo thalamicis or choroidal neovascularization and no adverse events of special interest. The Phase twothree pivotal confirmatory trial of Ocu410ST will randomize 51 subjects, thirty four of whom will receive a single subretinal two hundred microliter injection of OQ410ST at a concentration of 1.5 times 10 to the 11 vector genomes, VG per ml, in the eye with the worst visual equity and 17 of whom can serve an untreated controls.

The primary endpoint in the clinical trial is change in atrophic lesion size. The secondary endpoint include visual equity as measured by best corrected visual equity and LLVA compared to untreated controls. One year data will be utilized for the BLA filing. We plan to initiate the Phase twothree study mid-twenty twenty five and are targeting BLA submission by 2027. Now let's move on to our developments in AQ-four ten, which is specifically designed to address multiple pathways implicated in the pathogenesis of dry adulated macular degeneration, DAMD, and offer a distinct advantage to our current treatment options that target only one pathway, a complement system. Currently FDA approved treatment options require frequent intravitreal injections about six to twelve doses per year and are accompanied by various safety considerations. For example, roughly twelve percent of patients developed wet macular degeneration following treatment. It is also important to note there are no approved therapies for geographic atrophy, GA, in Europe. ONCURE410 has the potential to regulate all four pathways related to disease progression lipid metabolism, inflammation, oxidative stress and activation of the complement system, thereby addressing the underlying causes of this disease. Approximately two million to three million patients in U. S. And EU and eight million patients globally suffer from GA, advanced form of EAMD.

Preliminary nine month data of Ocufortin showed clinically meaningful two line or 10 letter improvement in visual function, LLBA and treated eyes compared to untreated eyes in the Phase one portion of the trial. Subjects showed considerably slower lesion growth 44% from baseline in treated eyes versus untreated fellow eyes at nine months and follow-up data from the Phase one study. Preservation of retinal tissue at nine months at our GA lesions of treated eyes with a single injection of ARQ410 in Phase I compared favorably to published data on a leading FDA approved complement inhibitor given monthly or every other month at the same time points. In the Phase II study, the safety and efficacy of AQ-four ten in patients with GA secondary to DAMD will be assessed. Fifty one patients were randomized twelve thousand one hundred and twenty one into either of two treatment groups, medium or high dose, but a control group. In the treatment group, subjects receive a single subretinal two hundred microliter administration of five x ten ten vector genomes or Vgsml, which is a medium dose, or one point five x ten eleven Vgsml high dose, while the control group remained untreated. This week, the DSMB convened and reviewed the safety and tolerability profile of an additional 15 subjects from the Phase two portion of the study.

No serious adverse events related to OQ-four ten have been reported to date in all 60 subjects including Phase one. Unlike currently available treatments for GA, there were no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endometrial mitis or choroidal neovascularization and no adverse events of special interest. Interim clinical data from the ARMADA clinical trial will be available in the second half of twenty twenty five. This data will help us design a future pivotal confirmatory Phase three study planned for 2026 and enable our potential BLA and MAA filings as soon as 2028. Given the multifunctional effect of our modified gene therapy, the profound unmet medical need, limited treatment options and the fact that it is designed as a one and done treatment, we believe ARQ410 can be a potential gold standard for treating GA worldwide. Lastly, I would like to call attention to our biologic candidate and inhalation vaccines platform. ARQ200 moved into the clinic and patients are currently being dosed in Phase one clinical trial for diabetic macular edema, DME. ARKU200 has the potential to change the treatment landscape for DME, diabetic retinopathy and macular degeneration, wet AMD, with its unique mechanism of action, binding the active component, thromstatin, to integrin receptors that play a crucial role in disease pathogenesis and holds the promise to benefit all DMA patients, including the thirty percent to forty percent of patients who do not respond to current anti VEGF therapies.

The ARQ200 Phase I clinical trial is a multicenter open label dose escalation study to assess drug safety via intravitreal injection in three cohorts. Low dose, zero point zero two five mg, medium dose zero point zero five mg and high dose zero point one mg. All subjects will receive a total of two intravital injections of ORQ-two hundred, six weeks apart. Patient follow-up will take place up to three months after the last injection. Approximately twelve million people in The United States, One Hundred And Thirty Million people worldwide are affected by DME, Doctor or wet AMD. The investigational new drug IND application for OQ500, the company's inhaled mucosal vaccine for COVID-nineteen was cleared by the FDA. The National Institute of Allergy and Infectious Diseases, NIAID, part of the National Institute of Health is expected to sponsor and conduct the Phase one trial to assess the safety, tolerability and immunogenicity for AQ-four hundred administered via two different routes, inhalation into the lungs and intranasally as a spray. The Phase one trial will enroll 80 adult subjects aged 18 to 64 years. 40 subjects will be assigned to the low dose group and 40 subjects will be assigned to the high dose group.

Within each group, 20 subjects will receive the inhalation form of the vaccine and the other 20 subjects will receive the intranasal form. The primary aim of the study is to determine safety, while secondary and exploratory endpoints include antibody production, systemic as well as mucosal and the number of breakthrough COVID-nineteen infections. OcQ500 is based on a novel chimpanzee adenovacter CHAD36 technology. Earlier clinical studies to prevent COVID-nineteen that employed a similar technology administered by inhalation demonstrated increased Mucosal and systemic antibodies and a durable immune response up to one year using one fifth the dose compared to the same vaccine administered intramuscular. The Phase I clinical trial is anticipated to start in the second quarter of twenty twenty five. I'll now turn the call over to Ramesh Ramachandran to provide the financial update. Ramesh? Thank you, Shankar, and good morning, everyone.

I will now provide an overview of the key financial results for the fourth quarter and full year of 2024. Our research and development expenses for the quarter ended 12/31/2024, were $8,300,000 compared to $7,800,000 for the fourth quarter of twenty twenty three. For the full year ended 12/31/2024, research and development expenses were $32,100,000 compared to $39,600,000 for the year ended 12/31/2023. General and administrative expenses for the fourth quarter ended 12/31/2024 were $6,300,000 compared to $5,200,000 for the fourth quarter of twenty twenty three. General and administrative expenses for the year ended 12/31/2024 were $26,700,000 compared to $32,000,000 for the year ended 12/31/2023. Net loss was approximately $13,900,000 or $0.05 net loss per share for the quarter ended 12/31/2024, compared to a net loss of approximately $1.11 or $0.04 per share net loss for the fourth quarter twenty twenty three. Full year net loss was $54,100,000 or $0.2 net loss per share compared to a net loss of $63,100,000 for the full year 2023 or $0.26 net loss per share. Our cash and restricted cash totaled $58,800,000 as of 12/31/2024, compared to $39,500,000 as of year ended 12/31/2023. We expect that our cash and restricted cash will enable us to fund operations into the first quarter of twenty twenty six.

As always, we are proactively exploring shareholder friendly opportunities to increase our working capital, including partnerships that will drive long term strategy for our scientific platforms. That concludes my update for the quarter. Tiffany, back to you. Thank you, Ramesh. We will now open the call for questions. Operator?

We will now begin the question and answer Our first question comes from the line of Michael Pritovich from Maxim Group. Please go ahead.

Hi there. Thank you guys so much for taking my questions today. Congrats on all the good progress. Thank you.

So I guess, first off, thinking about a more of a housekeeping question. When thinking about your runway, does this factor in for potentially newly launching studies like the, OCTU410ST Phase twothree?

Yes, Michael. It's already budgeted.

All right.

And then I guess in terms of the DME program, right, do you have an idea of when we could expect to see data start to emerge from that Phase one? And then what sort of efficacy endpoints are being evaluated just given that it is a Phase one study?

Good morning. I'll ask Kumar to address this.

Good morning. Thank you for the question. Actually, we are looking at the safety and efficacy report of ARQ200 towards the end of this year. And as we are assessing the safety of unilateral intravartial administration of ARQ200, we're also looking at the exploratory endpoints of BCVA and the dose response of ORQ-two hundred. However, we are also looking at the secondary endpoints of ORQ-two hundred antibody formation and PK of ORQ-two hundred as well.

Okay. Thank you. And then just one more from me and I'll hop back into the queue. Just for AKU-five hundred, right, I know this really hasn't been much of a core program lately, but just perhaps have you heard anything regarding funding availability for that Phase one, just given that the recent uncertainty around funding we've heard of at NIH?

Yes, Michael. The NIAID had meetings with us after our IND approval and they're still stating they're on track to initiate the Phase one.

All right. Thank you very much for the additional clarity. And once again, congrats on all the progress.

Thank you.

Our next question comes from the line of Swayampakula Ramakanth from H. C. Wainwright. Please go ahead.

Thank you. Good morning, Shankar, Umer and Tiffany. Couple of quick questions from me. So Shankar, you certainly have made an aggressive target for your team of filing three BLAs starting next year. So what gives you and your team confidence that you could achieve this? And as investors, what should we be watching out to see your progress towards this goal?

Yes. Good morning. Okay, good question. I mean, investors need to look at our track record. We started most of the gene therapy programs getting into the clinic late twenty twenty one, twenty twenty two. And today we are in the beginning of 2025 and we have all three programs running from all cylinders. So our track record speaks for itself. And so the goal is, once again, thanks to FDA for allowing us to knock out the Phase III and ability to convert existing Phase II clinical trial into Phase IIIII confirmatory CUTTL trial for BLA for Stargardt disease. It's a significant unmet medical need and that naturally lines up. I mean, so we are estimating if we start the trial mid this year about nine months of recruitment and then it's a one year follow-up that will put us into '27 and so that should be reasonably targeted for BLA. Similarly, getting into ARQ-four ten, we recently announced a month ago that our recruitment is completed in our Phase II clinical trial for ARQIFORTAM targeting geographic atrophy. So that means our Phase II will be completed by early next year. Our interim data, which is coming out in the second half of this year, will allow us to start having conversations and discussions with FDA as well as EMA on Phase III clinical design.

So we are hopeful to initiate that next year. Once again that will have a one year duration and GA is relatively easier to recruit compared to orphan diseases such as RP and Stargardt and we are inundated with patients during our Phase II clinical trial where so many patients reaching out to us. Therefore, that gives us confidence we can complete that clinical trial including recruitment. If you start in 2026, we can relatively get it done by 2028 and that can be lined up for BLA. And so that's why I think these timelines are reasonable. So starting next year, RP retinitis pigment is ARQ400 and 2027 is going to be ARKU4 tends to target disease and 2028 we're targeting BLA for ARKU4 and geographic atrophy.

Perfect. Thank you for that. So we are just talking about four ten for GA. So we know that there are two approved therapies for this indication. So given that, how easy would it be to convince both physicians and patients to initiate therapy on OQ410 as considering the price point difference?

Go ahead, Jima.

So RK, thank you for the question. I'll provide the clinical aspect and the price point. Shankar can provide the input. So in terms of one of the things I wanted to mention here is that we completed recruitment ahead of time for GA because there was a huge number of requests from the patients who have already got SYFYRAY and ISORRAY. In fact, they were on the waiting list and we had a certain number to recruit for our Phase three. In terms of whatever the approved therapies right now are there, particularly with competitors, the first and the foremost in terms of the clinicians, the safety and tolerability profile of those products are concerning and twelve percent of the patients are progressing to the wet AMD. In terms of the safety and tolerability profile of Occhio four ten, that gives us extreme confidence that there were no serious adverse events like CMV and ischemic optic neuropathy and ophthalmitus and vasculitis, which are the hallmarks of currently the approved products. Also our protocol had included the washout period for those two approved products as well for a three month period. So in terms of recruitment and in terms of the safety and tolerability and efficacy profile, we are not only seeing differences or improvements in the structural as well as the functional outcomes.

And in Europe, there is no approved products. So most majority of the physicians because of the compliance issues of six to 12 injections per year and safety considerations are not really prescribing. And also the patients are reluctant to do that. As you see, the age being onset is 60 years of age and older. And in terms of the price point, I would let Shankar add his thoughts here.

Thank you, Kumar. So RK from the price point perspective, as we stated, U. S. Itself has more than million patients with a late CAMD which is geographic atrophy and most of these patients get potentially funded by CMS. So we need to start working with them. Obviously, as an organization, we are very mindful. We're watching other gene therapies, how they are getting priced. And unfortunately in the marketplace, there are gene therapy products either to date, either they're targeting very complex diseases or they're going after ultra rare diseases or they're going after diseases that already have a solution in the marketplace, which is not an unmet medical need. It's using scientific platform AAV vector to deliver something to replace like one and done instead of taking multiple injections of biologicals or certain treatments. So definitely we need to consider all the price points and when you have a huge population like this, number one, the pricing should be reasonable and it's a one and done treatment. So even the patients are mostly in 60s or 70s, they still have many, many years of quality of life years remaining for them. So we'll do the appropriate pharmacoeconomic analysis and we will price it fairly.

So that our goal is to make sure the payers can reimburse it and the patients who really need the product, they get it. Our goal is to provide market access. We're going to work on every effort in our perspective to make sure our patients get our product, we need them.

Thank you. Thank you for that. So last question from me on OCA-four hundred, with the Phase III program with the Phase III study in progress, what additional data should we expect from the Phase onetwo study between now and filing of your BLA next year?

So as we have recently updated on our durability profile as well as safety. So safety will continue to be there. That's a commitment that we have made. So also on the efficacy functional endpoints, we will continue to report recently announced at the two year durability that's important. Okay. The LLVA data we recently announced at the two year durability that's important, not only from payer perspective too. So we'll definitely have three year data at the time of filing next year.

Perfect. Thanks for taking all my questions.

Thank you.

Question comes from the line of Robert Laboyer from Noble Capital Markets. Please go ahead. Good morning and congratulations on all the progress. My question has to do with OCU-five hundred and whether there will be any grant revenue to the company associated with the Phase one trial?

Yes. As we stated publicly, NIAID is sponsoring this program and we have completed our obligations from company perspective. We are responsible for doing all the preclinical work and manufacturing and filing the IND, getting it approved and clear to FDA and then transferred to them. So we've done our part and they're supposed to fund the Phase one clinical program and take it to the next level.

Okay. Will the funding be recorded as revenue by the company or will this just be something where you turn it over to the agency and they run the trial?

Yes, we'll be turning over to the agency there on the trial.

Our next question comes from the line of Danilo Gotland from Chardan. Please go ahead. Hey, good morning guys. Congrats on all the progress. A couple of questions on four ten ST. First, what is your manufacturing strategy for four ten ST and do you plan on using the commercial grade product for the Phase twothree study?

Good question, Daniel. We already made at a commercial scale introduced into our Phase one and the same scale would be used for Phase twothree. We'll follow the similar pattern like we're doing for RP. For RP, we're introducing two commercial scale lots in our pivotal trial. We'll do the same thing for SD program, which is consistent and the FDA agreed with our strategy and we're moving forward with that.

Okay, got it. And in terms of the sites for this study, are you looking at both The US and ex U. S. Sites? And what are proportion of each if you're using both?

There is no ex U. S. Sites except for Canada, of course. I mean, we do have sites set up in Canada for retinitis pigmentosa. If necessary we'll activate those sites for Stargardt. But based on the patient population, we only need 51 patients. I think, Huma and our team are comfortable that they can get those patients in U. S. Very quickly.

Got it. Okay. Thank you very much for taking the question.

Thank you.

This concludes the Q and A portion. I will now turn the call back over to Chairman, CEO and Co Founder, Doctor. Shankar Musanuri.

Thank you, operator. We appreciate the continued interest and involvement of our key stakeholders as we move forward with our transformative initiatives. We look forward to a year of significant catalysis ahead as we establish Arcogen as the pioneering biotechnology leader in gene therapies for blindness diseases. Have a great