We're going to get going with our next company presenter at the B of A Annual Healthcare Conference, and I'm pleased to be introducing Oculis and Sylvia Cheung, CFO. Sylvia has some slides that she's going to run through, so I'll hand it over to you, Sylvia.
Thank you. Thank you, everyone, for joining. Thanks, Jason, for the invitation and the opportunity to present. I'll be sharing with you an overview of Oculis' portfolio, but before that, this is the standard safe harbor statement, cautionary notes on forward-looking statements that I'll be making during this call. Oculis is a global biopharmaceutical company focused on innovations in the ophthalmology and neuro-ophthalmology space. We are global. We're dual-listed. We have three core assets, and within each of the assets, we have multiple indications. Today, I'll be covering the lead indications in each of these core assets. The next slide will cover what those are. We have a strong balance sheet, no debt. We reported $206 million of cash, cash equivalent, at the end of March, and the runway is into early 2028.
Management teams are experienced and with a track record of success, as well as supported by international leading healthcare investors. Here, on this slide, slide four, it shows the three core assets and their lead indications. All these assets are first in class, addressing large critical medical needs. OCS-01, it's a proprietary technology. It's called OptiReach. It's a formulation of high-concentration dexamethasone. It's unique, and it's first of its kind because it's an eye drop that reaches to the back of the eye. The initial indication that we are aiming for is for diabetic macular edema. It's a topical treatment to allow early intervention and for patients with an inadequate response to the current standard of care. I'll go into a little bit more about the treatment paradigm and how OCS-01 fits into that.
You can see at the bottom of the slide, the graphical illustration of the visual impairment or disturbances that the patients experience with each of these three indications. For the second asset on the screen here, it's Privosegtor. It's actually our newest asset. It's OCS-05. It is a neuroprotective peptide small molecule. It's a novel neuroprotective candidate promoting neuronal and axonal survival. The implication and the application of this asset is quite broad. I'll go through some of the indications that we will be targeting. Initially, it's on acute optic neuritis, and we announced a very positive readout in the beginning of this year. Lecaminlimab is OCS-02. It's a topical eye drop and antibody fragment technology of TNF alpha inhibitor. We are targeting dry eye as the initial indication for study. This particular asset has anti-inflammatory and anti-necrosis mode of action.
Our aim is quite unique for dry eye. We're targeting genotype-based development, drive precision medicine. I'll cover what that means and also the most recent data from OCS-02. First, let's go over OCS-01, OptiReach, for diabetic macular edema. It's a large and growing market, as we know, but the market is not very well served. Less than 60% of patients are diagnosed, and within the diagnosed group, 44% are treated. The current available approved treatments are all invasive, meaning that they're either injectables or implants. This means that it's a very high burden of treatment with low patient compliance, which results in, as you can see on the chart, a very small segment of the patient is treated. How will OCS-01 address this? Before I get into that, first of all, the diagnosis is through OCT, very simple.
They go to the doctors, they get a scan, they're diagnosed with OTC. Now, today, 56% of patients diagnosed don't have any treatment. The first thing they will ask when they're diagnosed is, are there any drops? Is there a pill? The answer is no and no. 44% of patients are treated once diagnosed, but the issue there is 40% of those treated patients are not receiving adequate response. And adequate response means five letters of improvement, meaning if you go to the eye doctor, you have these eye charts with letters and rows. They're not seeing, they're not getting five letters improvement, meaning one line on the eye chart. Where can OCS-01 come into play here? First of all, it's very versatile. It can cover the whole continuum of care.
Secondly, in the segments where there are no competition, no available treatment or inadequate treatment, OCS-01 can fit in, meaning on the early intervention side, this can be a first-line treatment for patients who currently don't have any pre-invasive treatment available to them. On the back end, for patients who are not receiving adequate response, this particular product, OCS-01, can be used as a combination with anti-VEGF or used standalone. The versatility of the product is very attractive. We are also going to be placed in the market targeting the segments of the market where there is no current competition. We estimate that the total U.S. addressable patient population is 1.3 million, which is a very substantial market. The company has a very good clinical profile on this candidate, positive and consistent good results across four clinical studies.
I'll here show you data from the last clinical study, which is a phase III study. It's the stage one phase III study of OCS-01. First of all, the letter gain was 7.2 letters in BCVA, best corrected visual acuity, versus baseline at week six and increasing to 7.6 at week 12, which is the end of the maintenance period. Second is a percentage of patients with greater than 15 letters was at 25% at the end of the loading dose, week six, and increased to close to 30%, or in this case, to be specific, 27.4% at the end of the maintenance period, week 12. Further, when you look at CST, which is the measurement of the retinal thickness, we also saw very strong statistical significance at week six, maintaining up to week 12 at the end of the maintenance period.
When you look at the patients by naive versus previously treated, we can see that the patients in both groups show positive results with the treatment of OCS-01. From a safety standpoint, very well tolerated with no unexpected AEs. Overall, the results were very, very encouraging, and we were very pleased with the outcome and made the decision to move into stage two of the phase III study. The design is exactly the same. The only two differences are one is the patient size. In the stage two study, we have over 800 patients in two trials, 400 each. The duration of the study is 52 weeks. The previous stage one study was 12 weeks. We're very pleased to have announced the completion of enrollment, really very rapid, which speaks to how patients and physicians really like the product.
We were able to complete the over 800 patients for the two studies and over 119 sites were activated. Rapid enrollment, full enrollment completed in the beginning of this year, and we are looking to announce phase III results in Q2 of next year. Shortly thereafter, we'll be looking to submit our new drug application with the FDA. Now, coming back to DME patient treatment paradigm, you can see here that our goal is to have OCS-01 be the first in line, first-line treatment in the early intervention segment, and then as a new treatment option, whether it be standalone or combination in the diagnosed and treated, both are white spots where no competition or available treatments are currently available to patients.
The total diagnosed population for DME is roughly about 1.8 million, and only 0.5 million are currently treated with well-controlled results, which leaves about 1.3 million of patients as our addressable market. Substantial market. In summary, we can say that it is a large market, large addressable population, robust data from the historical studies. We de-risked the phase III program, and we recently completed the enrollment of stage two and are looking to announce readout in Q2 of next year. Near-term catalysts include the data readout in Q2 of 2026, as well as NDA filing in the second half of 2026. Privosegtor, this is an exciting development. This asset actually allows the company to transform and also allow us to evolve our pipeline from a pure ophthalmology company to neuro-ophthalmology. This particular asset has potential neurology applications as well.
Let me first share with you what Privosegtor is. It is a neuroprotective candidate activating neurotrophic signaling pathways. It is a small molecule peptide. It is an activator instead of a blocker, which is important. We selected this by a high-throughput screening for neurotrophic and neuroprotective properties. We have done many animal models, including glaucoma, multiple sclerosis, and acute optic neuritis. Acute optic neuritis is the indication that we selected for the phase II study, and I will share with you the outcome of that study. This particular asset activates SGK, which activates FOXO3, which is known to help neuronal and axonal survival. It triggers multiple beneficial effects, including apoptosis, oxidation, and inflammation. Why was acute optic neuritis selected? First of all, before I get into that, I just want to mention from a patient prevalence standpoint, it is estimated at roughly about 65,000 between U.S.
Europe, roughly about 50/50 switch. The indication has an orphan drug designation. For Privosegtor, this particular indication was selected not only because there is no approved therapy, but also because it is a very pure model in terms of showing neuroprotective benefits. Now, the typical patient is a female in her 30s, active and childbearing age, and suffers from inflammation of optic nerves, severe ocular pain, visual disturbances, reduced color perception, and so on. The retinal ganglion cells are in distress and die. Here you can see that from a visual standpoint, normal vision, as you can see, is the colored picture. Patients in the acute AON episode lose contrast, they lose the color vision, and the low contrast vision also significantly deteriorates in addition to having severe ocular pain.
Before I get into the data, I just want to share with you the measurements, biomarker measurements. How do we measure efficacy? It's actually quite simple. It's done through a commonly used imaging test called OCT, which I'm sure you're familiar with. There are two measurements that we measure. One is the ganglion cell inner plexiform layer, or the GCIPL layer. This is the layer that is used to measure the status of the axon. On this slide, you can see that in the middle there, the top layer with yellow and orange and red layer is RNFL. The layer below it is GCIPL, and the GCIPL is the layer that measures the thickness of the ganglion cell. The RNFL is the retinal nerve fiber layer, and that particular layer gives us the status of the axon.
The top layer is the health of the axon, and the second layer below it is the health of the retinal ganglion cell. From a functional standpoint, we measure LCVA, low contrast visual acuity, which is an approvable endpoint by the FDA, which is important because patients with acute optic neuritis have very poor low contrast from a vision standpoint. Here is the summary of our phase II study. It is a multicenter double mask study. Treatment is five days, five consecutive days of IV injection. The current standard of care is steroid IV for five days as well. Our active arm is basically Privosegtor OCS-05 plus steroid compared with the control arm, which is placebo plus steroid, five days consecutive and six months evaluation period.
The good news to report here is we saw very positive results from the acuity study, both from an anatomy and biology standpoint, and from a vision improvement standpoint, we also saw great improvement. Safety is clean. You can see the summary at the bottom. Let me walk you through the outcome. Patients with Privosegtor in a Privosegtor arm, we saw a 43% improvement in thickness of GCIPL. Again, this is the measurement for retinal ganglion cells. It means that patients receiving Privosegtor can protect and preserve their retinal ganglion cells 43% better than patients without Privosegtor. That improvement continues to maintain to month six. In addition, RNFL, this is the measurement of axon, and we also saw 30% of improvement for patients receiving Privosegtor at month six.
In terms of visual improvement, this is very exciting because we saw that patients in the Privosegtor arm actually saw 18 letters betterment comparing with patients in the control group. That improvement continues and maintains through month six. We further looked into the data and looked at patients that are with multiple sclerosis, patients without multiple sclerosis, and patients with HCVA below and over 60. What we saw is there are consistent benefits across the different patient groups and across the different time points. We are very encouraged by this outcome. All measurements are pointing in the same direction, showing that retinal ganglion cells and axons are being preserved for survival. What's even more exciting is in Privosegtor arm, we saw that something that's called a Neuro filament. We saw a very good outcome from that. Neuro filament is found in cytoplasm.
It forms the neuro cytoskeleton of the neuron. When neurons are suffering, they're released into the blood. By the way, this is an approvable endpoint. What we see here is a very nice curve where patients receiving OCS-05 are having less neuro filament being released into the blood, which suggests that they have less ganglion cells and axons dying. Again, across all endpoints, GCIPL, RNFL, LCVA, Neuro filament data, we saw that they're consistently pointing to one direction, meaning axons and neurons are being saved. With the very positive results, we are moving full steam ahead into registrational study on AON, the acute optic neuritis, which was the dataset that I just shared with you. In addition, based on pathophysiology assessment, as well as the mode of administration assessment, we are going to add two additional indications to study. One is acute relapses in MS.
The other one is NAION. From multiple sclerosis, I don't need to get into a lot of details. It's the most common CNS condition affecting young adults. Here you can see that the world prevalence is 2.8 million, and in the U.S., about 850,000. It's a very large and growing disease. Currently, there are no current treatments that are slowing the disease progression. There isn't a product from a neuroprotection standpoint that can aid MS. What we're aiming to do is to target acute relapses of MS, which is very similar to AON, the indication that I just mentioned to you before. Our goal is to use Privosegtor to treat acute relapses because MS acute relapses is very similar to AON.
What we would like to do is, you can see here, utilizing the treatment, the hypothesis that we have is that Privosegtor's neuroprotection and axon protection will reduce the relapse-associated worsening, thereby giving benefits to patients and reducing the possibility of having permanent disability from relapses of MS. The next indication that we're looking to study is NAION. This is a disease where there is no approved treatment. The indication in the U.S. is roughly about 20,000-30,000 patients. It is an orphan drug. Very similarly, in this case, there is rapid vision loss, including visual field defect, potential permanent vision loss in many patients, and over 60% of patients have significant visual impairment. Our aim with Privosegtor is twofold.
With acute optic neuritis, we are meeting with the FDA in Q3 to talk about the registrational study based on the positive phase II results that I shared with you earlier. We will start phase II and phase III registrational study in 2026. On NAION and multiple sclerosis relapse, we will be discussing with the FDA in the second half of this year and completing potential IND readiness activities and be in a position to file NDA IND submission in 2026. For Lecaminlimab, which is OCS-02, it is a topical product. It is a novel anti-TNF eye drop for ocular inflammation. Initially, we are studying it for dry eye disease. This particular asset is a proven mode of action. It has a very good enhanced ocular penetration to the eye.
It also has a genetic biomarker and a precision medicine approach, which we are pursuing based on the previous results as well as interactions with the FDA in terms of registrational trial design. Dry eye, as we all know, is a large population disease, but it is also very unsatisfied or patients that are largely unsatisfied. Only 13% of patients getting prescriptions are experiencing lasting relief and are renewing their prescription. Typically, patients would come into the doctor with a bag of treatments and, on a trial and error basis, see what works. Oftentimes, products are not working, slow to have drug effect, and also discomfort in terms of staining and pain. Our OCS-02 is very unique in terms of its properties. Let me go through with you. The last study that we did was a phase II-B study.
The aim of that study was to evaluate Lecaminlimab in the treatment of signs for dry eye. The 2nd aim was to see if Lecaminlimab in subjects with this TNFR1 genotype works well and works better in population without this genotype. 3rd is to select the efficacy endpoint for signs. The good news is we achieved all objectives for this study. We selected inferior corneal staining as the sign efficacy endpoint for phase III or for registrational study, which we have discussed with the FDA. Here you can see that the outcome from the last study for full population, it was negative 0.12. The population with the TNFR1 genotype is close to 0.6, so multiple folds better than the full population. What's interesting to note is Xiidra, for example, an approved product, also approved based on inferior corneal staining.
Our outcome in the full population is better than their best outcome. Our outcome in the genotype is 6x better than the best of Xiidra's efficacy outcome from their phase III study. You can see that the onset is very rapid. Across multiple efficacy endpoints that we studied, the TNFR1 genotype consistently showed much more, much better profound responses to Lecaminlimab, which gave us the confidence of pursuing a genetic biomarker-based precision medicine approach. Safety profile, pristine, nothing to note. The product is really unique, a new mode of action, rapid onset, as quick as 15 days or roughly two weeks, better than the months, two to three months from existing products. Very good tolerability and drop comfort versus existing products in the market have discomfort, staining, and pain as side effects.
The ability to predict response because we know the response and the effect is predictable, a simple saliva test can identify patients with TNFR1. Very easy to administer a test. The outcome is seven times and five times better on symptoms and signs. Very remarkable and profound effect. This is the design of our phase II and phase III study, which was aligned with the FDA based on our meeting with them in this past quarter. We are looking to enroll roughly about 160 patients. 2/3 of them would be patients that are positive with TNFR1. The primary endpoint, because it is a symptom study, will be looking at global ocular discomfort for the genotype positive patient. The secondary endpoint will be looking at all patient population.
In conclusion, for Lecaminlimab, it's a very large disease population, 10 million estimated to have moderate to severe dry eye. Our genotype population is roughly 20%, which means that our addressable patient population is 2 million, a very large group. We can own that population given the profile of the product. Robust data from previous studies. The near-term milestones include starting the phase II and phase III study later this year with top-line results in the second half of next year. In conclusion, because of the positive clinical trials, the science and the development that we achieved, we've been able to evolve and really transform our product portfolio from ophthalmology into neuro-ophthalmology.
Our focus right now is to advance OCS-01 in DME, to start the Lecaminlimab personalized medicine trial for dry eye, which is a phase II and phase III study, and to go full steam ahead with acute optic neuritis based on the very positive results that I shared with you earlier. In addition, we're looking to expand OCS-05 into NAION and multiple sclerosis relapses. What I shared with you on Privosegtor is just the tip of the iceberg. There are multiple additional ophthalmology and neuro-ophthalmology indications that Privosegtor can potentially serve. We look forward to sharing more information on that in the coming days in future announcements. In summary, Oculis, even though we're a small biopharmaceutical company, we've made a lot of progress. We have a diverse and highly differentiated product portfolio, late stage in terms of OCS-01 with two indications in phase III.
We also have multiple catalysts coming up in the next few months and quarter. All of these assets are first in class with extremely robust historical clinical data and are positioned to serve markets or segments of the markets where there is no competition and in great need for innovation and products like OCS-01, OCS-05, and OCS-02. With that, I would like to thank you for joining the presentation. Jason, if there are any questions that you would like to ask or anything that you would like to supplement?
Sure. Maybe just in terms of the lead opportunity, diabetic macular edema.
Yeah.
How you're thinking about sort of where you focus your promotional efforts if the therapy is approved, do you think this is going to be driven by the retinal surgeon who's doing a lot of the intravitreal injections?
Do you see it potentially being used more upstream of that if you get to the watch and wait patient? Maybe just piece together that commercial opportunity a little bit.
Yeah, absolutely. Happy to do that. The good news is the diagnosed population today is 1.8 million. It is a large population, which means that physicians have their names and know where they are. A lot of them are not treated. Our focused approach is initially to go to the retinal specialists because they are already treating patients with anti-VEGF. The need there is really patients who are not getting the adequate response. They now have a tool that can help. Patients who are getting anti-VEGF, there is a compliance issue. Clinical settings require 10-11 or so visits. They are only coming in three to four.
In between those visits, physicians have told us that they would actually welcome a topical eye drop that can prolong the efficacy. A patient after anti-VEGF injection can be sent home with a topical to bridge in between the visits. That is where the retinal specialists have told us our product plays really well in their space. There are roughly about 5,000, so not a large prescriber base for the retinal specialist, very focused, and we will initially start there. Beyond that, we will be looking into the earlier segment where the general ophthalmologists are serving. That is roughly about 15,000 general ophthalmologists in the U.S. There today, when patients get diagnosed in a general ophthalmologist's office, they do not have anything, right?
When OCS-01 is approved, if they're diagnosed, they have an option right there and then that's off the shelf that doctors can offer them. I was told by one of the general ophthalmologists that a patient came in and they were supposed to have a cataract surgery, but when they did the OCT scan, they saw that there is DME. They had to put the cataract surgery on hold to get DME treated first before the cataract surgery. The only thing they can do is today refer them to retinal specialists. When OCS-01 is available, that picture changes dramatically because something is available and efficacious and safe for those patients in earliest stage. What we'll do is very focused, starting with retinal specialists and then go to general ophthalmologists.
Okay.
Yep. All right. Sounds good. Thank you so much.
Thank you.