Hello everyone, and thank you for joining the H.C. Wainwright's Fifth Annual Ophthalmology Conference. My name is Kyle Murie, and I'm an analyst on the Corporate Access team. H.C. Wainwright is a full-service investment bank dedicated to providing corporate finance, strategic advisory, and related services to public and private companies across multiple sectors and regions. We have a total of 19 publishing senior analysts and over 650 companies covered across all sectors. If you'd like more information, please check out our website, hcwco.com. With that said, have a productive and enjoyable day. I'd like to introduce our presenter, Sylvia Cheung, who is CFO of Oculis . Thank you.
Thank you, Kyle, and thank you, H.C. Wainwright, for the opportunity to present Oculis today. Before we start, I would like to just give a quick reminder of the safe harbor statement related to forward-looking statements that I'll be making during this presentation. Oculis is a global biopharma dually listed company on NASDAQ. We have a portfolio of innovative ophthalmology and neuro-ophthalmology candidates targeting significant market opportunities. We have three core assets: OCS-01, or OPTIREACH, which is a formulation of high-concentration dexamethasone eye drop, aiming to become the first non-invasive treatment option for diabetic macular edema. It's currently in phase III, with readout anticipated in Q2 of 2026. Privosegtor, or OCS-05, is a first-in-class neuroprotective candidate with potential ophthalmology, neuro-ophthalmology, and potential opportunities even beyond that. It's currently in development in acute optic neuritis, and it's being investigated in NAION and MS relapses.
Licaminlimab, or OCS-02, is a novel topical anti-TNF candidate with a genotype-based development to drive a precision medicine approach in dry eye disease. We're starting phase II/III in the second half of this year, with readout anticipated by the end of next year. We have a strong balance sheet, no debt, and a cash runway into early 2028. Here we have a pictorial or graphical illustration of our three core assets, as well as the three lead indications for those assets. Down in the bottom, you can see the visual disturbances or impairments as a result of the diseases: diabetic macular edema, acute optic neuritis, and dry eye disease. This presentation will be focusing on these lead indications within the three core asset families. First, OCS-01 and diabetic macular edema. As we know, DME is a large and growing market.
In 2020-2021, 37 million patients globally are impacted by DME, and it's expected to grow to above 50 million by 2045. The current treatments that are available or approved are all invasive in nature, meaning anti-VEGF injectables or steroid implants. The result is a high burden of treatment and low patient compliance. What we have is a market that currently has about 60% of patients diagnosed and about 40% undiagnosed. Within the 60% of the diagnosed group, only 44% is treated. The rest of the patients who are diagnosed are not treated. This is mainly the outcome of the invasive current available treatment options. Before we get into the product profiles and how it will be positioned in the market, let me just mention a couple of things. One is patients present themselves with DME symptoms through OCT imaging diagnosis.
As I mentioned before, 56% of the diagnosed patients currently are not receiving treatment. In this particular segment, OCS-01 aims to be the first-line treatment for those patients who need early intervention. As a matter of fact, the product is very versatile, and it can provide treatment alternatives across the whole continuum of DME care. In the diagnosed and treated segment, which is about 44% of the diagnosed patients, only 60% of the patients that are receiving anti-VEGF is getting adequate response, which is defined as one line or five letters. 40% of them are not getting adequate response. In this particular segment, OCS-01 again can act as a very efficacious and safe option and potentially also be in combination with anti-VEGF. As a result of the current needs in the market, both in early intervention and in the diagnosed population, we estimate that the U.S.
addressable patient population is 1.3 million. Oculis had completed four clinical trials very successfully in the past. The latest trial that we conducted with readout was from our phase III stage 1 of the DIAMOND program. There are five key takeaways. Let me walk through those with you. First is on gain in BCVA, best-corrected visual acuity. We saw that OCS-01 patients had 7.2 letter gains at week 6, which is the end of loading dose, and improved to 7.6 letters at the end of maintenance dose. Point number two is at the end of week 6, 25.3% of patients had 15 letters or more gain at the end of week 6, which improved to 27.4% at the end of week 12. Further, we also saw a rapid and sustained reduction in retinal edema already at week 12 and sustained through week 6 and week 12.
When we looked at patient population from a naive and previously treated segments, we saw that positive results were observed in both populations. Last but not the least, from a safety and comfort standpoint, the product was well tolerated with no unexpected adverse events and no evidence of cataract up to 12 weeks. IOP increase was consistent with literature. We also rapidly completed our phase III stage 2 program, activated 119 sites with over 800 patients already enrolled. We're on our way to collect the needed data, and the top-line results from our phase III study are anticipated in Q2 of 2026. As I mentioned before, there's about 1.8 million DME patients diagnosed, and of that, only half a million patients are treated and well controlled. That means that for Oculis, OCS-01 can serve the 1.3 million patients who are either not treated or treated but with inadequately controlled results.
This really presents a significant opportunity for Oculis with OPTIREACH, and it'll be able to expand the current market, which is valued at about $3 billion. In summary, DME is a large market, a growing patient population, and we have a large opportunity of providing solutions to 1.3 million patients who are currently either not treated or getting inadequate results from the current available products. Data is very robust from our phase III trial stage 1, and we're completing the stage 2 of the phase III, and we expect readout to be in Q2 of next year. If positive, NDA filing would be in the second half of 2026. Let's take a look at Privosegtor, and the lead indication there is acute optic neuritis. Privosegtor is a neuroprotective candidate activating neurotrophic signaling pathways that support neurons and axon survival and preservation.
It's a small molecule peptide that penetrates the blood retinal barrier, and in in vivo models, which we conducted, and these are in glaucoma, multiple sclerosis, and acute optic neuritis, the product showed it preserves, protects, or promotes survival of neurons and axons. It also triggers multiple beneficial effects on apoptosis, oxidation, and inflammation. Let's take a look at acute optic neuritis. For Privosegtor, acute optic neuritis, or AON, was chosen as an initial indication, not only because it has no approved neuroprotection treatment currently available. It also is a very pure model in terms of showing neuroprotective benefits. A typical patient is a female in her 30s, young, active, and child-bearing age group that suffers from inflammation of the optic nerve, severe ocular pain, visual disturbances, reduced color perception, and so on. AON is also one of the early onsets of multiple sclerosis. AON is an orphan indication.
It's estimated at about 65,000 patients in the U.S. and in Europe, roughly about 50/50 between the two geographies. Core symptoms are pain and loss of vision. Today, there are no approved products for AON from a neuroprotection standpoint. Physicians use a very high dose of steroids to reduce or shut down inflammation when the acute episodes start. Now, sadly, the prognosis is we end up with patients who could potentially improve high visual contrast, meaning HCVA, but the low visual contrast, LCVA, would not be back to normal, which is a large unmet need, and Privosegtor will be able to address in terms of low visual acuity as well as neuroprotection for patients who are in need. Before we get into the data from our latest trial, I would like to first mention the way that we measure efficacy for acute optic neuritis.
It's actually quite straightforward through OCT, which is a common non-invasive imaging test. As you know, OCT scans a few things. In this case, the ganglion cell inner plexiform layer, or GCIPL, and RNFL, which is the retinal nerve fiber layer. They're used to detect and monitor damages in the optic nerve and retina, particularly in conditions such as glaucoma. Here you can see in the middle, there is an orange and yellow layer that is RNFL thickness. It gives the status of the axon. The layer below it, which is green and yellow, is GCIPL, which gives the health of the retinal ganglion cells. From a functional standpoint for acute optic neuritis, we measure low visual acuity, LCVA, which is also an FDA-approvable endpoint. Here we have the acuity trial design. We announced the readout in the beginning of this year.
It was a phase II multicenter double-masked trial comparing OCS-05 plus steroid versus placebo plus steroid. The treatment period is five days, followed by a six-month evaluation period. The primary endpoint was cataract safety. As you know, OCS-05 is a new class and a new molecular entity, and it's typical to have a primary endpoint for safety. The secondary endpoints are focused on efficacy, imaging, and function. We're extremely pleased with the outcome of the acuity trial results. Privosegtor, or OCS-05, achieved primary safety endpoint and key secondary endpoints, showing functional visual improvement and neuroprotective anatomically and biologically as well. Let's take a look at the details. Here we have GCIPL thickness measurement, which gives us the health of the retinal ganglion cells. Patients in the Privosegtor arm who received the drug showed 43% improvement in GCIPL thickness at month three and maintained through month six.
Now let's take a look at RNFL. RNFL thickness measurement gives us the health of the axon. Patients with Privosegtor (OCS-05) treatment showed approximately 30% improvement in RNFL thickness through month six. Now on functional measurement, LCVA. Patients who were treated with Privosegtor achieved clinically meaningful improvement in visual function by 18 letters difference in month three and sustained at month six, which is extremely encouraging. Further, we dived into patient details by looking at MS patients versus non-MS patients, high visual acuity, or at 60 or lower than 60. No matter how we look at it, we saw Privosegtor bring in benefits at month three and sustained at month six, and the benefits are consistent across the groups. Another unique and exciting finding is neurofilament data. As you may know, neurofilament is found in the cytoplasm.
Neurofilament forms a neuronal cytoskeleton of the neuron, and then when the neurons are suffering, they're released into the blood or the cerebral spinal fluid. In fact, neurofilament is a regulatory endpoint as well. What we see here is a nice curve where patients who are treated with Privosegtor are having less neurofilament being released into the blood, which means that they have less retinal ganglion cells and axons dying. Since AON is an acute disease, patients will go back to normal after month six because the crisis is over. The data shown here gives us even more confidence, and taken in combination with the previous measurement that I show, that Privosegtor can act in a situation of neuron and axon injuries to protect neurons and axons.
In summary, the outcome was very positive for acute optic neuritis, and we're meeting with the FDA to discuss a full development program to support acute optic neuritis registrational plan. With a very positive result and the encouraging data, we're proceeding with AON development, and we are also adding two programs: acute relapses in MS and NAION to our portfolio. MS is the most common form of CNS conditions affecting young adults. The market is valued at above $20 billion, and we know there is a very high prevalence of patients, roughly about 2.8 million worldwide or 850,000 here in the U.S. It is characterized by relapses or attacks on the CNS, leading to inflammation, demyelination, and neurodegeneration. Existing therapies only slow the disease progression, while relapses continue to cause disabilities.
The goal Oculis would like to achieve with Privosegtor is to treat relapses and to reduce the relapse-associated worsening. MS relapse is very much like acute optic neuritis that I described before, and there is a need to protect axons and neurons which are suffering during the relapse acute periods. The estimated number of relapses in the U.S. is around 170,000, and the symptoms include loss of vision, like acute optic neuritis, and it can also have severe weakness or poor balance interfering with mobility. The next newly added indication is non-arteritic anterior ischemic optic neuropathy. It's a second indication that we're expanding in the Privosegtor family of portfolio of assets. It's a condition causing sudden vision loss, and it's also a rare disease with approximately 20,000- 30,000 patients in the U.S., mainly affecting patients over 50 years of age.
In this condition, retinal ganglion cells, axons, and optic nerve atrophies are caused by hypoperfusion. As we know it, over 60% of patients have significant visual impairments in the affected eye, and there's permanent visual damage in many patients. Again, no approved product and high unmet need. We're moving forward with our plans to discuss with the FDA on NAION , MS in addition to acute optic neuritis discussion, which I mentioned earlier. In summary, with the very positive results from Privosegtor phase II study, it unlocks a number of potential indications for us. It had broad potential in multiple neuro-ophthalmology and neurology indications where we are lacking neuroprotective therapies with high unmet need for neuroprotection.
The plan is to complete regulatory discussions in the second half of this year and on AON , move forward with our phase II/III trial in the beginning of next year, and potentially IND submissions for the two new indications in 2026. Next, we have Licaminlimab for dry eye disease. Licaminlimab is a novel anti-TNFα eye drop for ocular inflammation. It's specifically formulated for topical delivery. It's clinically proven. It enhances ocular penetration due to its small size. From a treatment standpoint, we also have a proprietary genetic biomarker, and for patients with a specific genotype TNFR1, we saw a profound response to OCS-02 or Licaminlimab, which I'll go through in the next few slides. First, looking at dry eye disease, I don't think I have to mention that it's a large and unsatisfied market. Only 13% of patients are experiencing lasting relief after 12 months of treatment.
As a matter of fact, 85%- 90% of patients discontinue their treatment within six months from the start of the treatment. The discontinuation and switching is a very common phenomenon that we see in dry eye disease because the current available products are not providing the needed efficacy. We have, for Licaminlimab, done three positive phase II trials. The summary of the phase II trials is as follows. There are three main points. First is we saw meaningful and rapid treatment effects in both signs and symptoms from the previous three phase II studies. We also saw more profound treatment effects in patients with TNFR1 genotype, and this represents about 20% of the U.S. general population. For that particular group of patients with TNFR1 gene positive, they have five times better efficacies in signs and seven times better efficacies in symptoms compared to the general population.
The product is also well tolerated with a drop-like comfort similar to artificial tear comfort level. The most recent phase II study is the RELIEF study. It's a phase II-B trial. The trial was conducted to address three objectives. The first was to evaluate the efficacy of Licaminlimab in the treatment of signs for dry eye. The second is to confirm the differentiated response to Licaminlimab in subjects with TNFR1 biomarker in the signs of dry eye disease. The third or the last is to select the primary sign efficacy endpoint for phase III trial and to inform the overall development plan. We're happy to report that all three objectives were achieved. We saw that the product worked very well in full population. In addition to that, it worked extremely well in patients with TNFR1 genotype. We also selected corneal staining as the sign endpoint for our phase III program.
Corneal staining, as you may know, is reflective of inflammation and apoptosis, which plays a crucial role in dry eye disease. Corneal staining, mainly the inferior part, is also the most commonly assessed sign in clinical practice as it can affect the quality of vision. Here you can see that the effects from Licaminlimab kick in really fast for TNFR1 positive patients, as quickly as day 15. From a safety standpoint, we saw low incidence of adverse events with Licaminlimab, actually similar to vehicle. No ocular SAEs are reported with Licaminlimab and no drug-related SAEs. Very positive safety outcome from the phase II-B trial on Licaminlimab for dry eye disease. The opportunity for Licaminlimab in dry eye disease is large. It's highly differentiated, marked by being a new mode of action and targeting both signs and symptoms.
Very rapid onset as early as week two, comparing current existing products in month three or four. Significant early onset and results can be seen with usage of OCS-02. Tolerability and drop comfort is also very high, and the ability to predict treatment is critical. This is a very unique differentiator from other products that are currently in the market. We saw five-fold higher response rate in signs and seven-fold higher symptom efficacy for patients who have TNFR1 genotype biomarker. In terms of the next study, which we actually discussed with the FDA and aligned on the trial design, we're looking to start the study later this year with results anticipated in late 2026. The trial is a phase II/III study. It's randomized, multiple center, and double masked. Total number of patients is roughly about 160, and the TNFR1 genotype patient will represent about two-thirds of the trial.
The primary endpoint is a global ocular discomfort score at day 29 in patients with TNFR1 genotype positive patients. The key secondary endpoint is global ocular discomfort score at day 29 in all patients. In summary, Licaminlimab addresses a large patient population who are in the moderate to severe group. This patient population is estimated at about 10 million in the U.S. who are patients with moderate to severe dry eye disease. Of this group, about 2 million are TNFR1 positive. A large group that Licaminlimab can really own because of the profound impact that we saw in previous trials for that genotype population. Data is robust based on the previous studies, and we're moving forward with the registrational phase II/III trial and aligned with the FDA already earlier in the year.
We plan to start the trial in the second half of this year with top-line results available by the second half of next year. In conclusion, Oculis is really delivering a very nice portfolio, well balanced with novel and highly differentiated products. The strategic evolution of the company had advanced from OCS-01 and OCS-02 on DME and dry eye into neuro-ophthalmology, initially starting with acute optic neuritis and expanding into two additional indications, NAION and MS relapses. Privosegtor has the potential to allow us to add multiple additional neuro-ophthalmology and neurology conditions. We're very pleased with the power of the portfolio and the science behind that will allow us to continue to add and create value for the company and for the shareholders.
In summary, we have a very rich set of catalysts coming up across multiple assets and indications. In the three core assets, OCS-01, OPTIREACH, the DME trial is well underway, and we anticipate top-line results from our phase III program in Q2 of 2026. If positive, we'll be submitting NDA to the FDA in the second half of next year. Dry eye and acute optic neuritis will be able to start our phase II/III trials in the upcoming quarters, and we'll be also looking to add development activities on NAION and MS relapses. We're very pleased with the development program as well as the upcoming catalysts, and we also have a very strong balance sheet to help fund the activities that we have planned and continue to execute and deliver on the milestones.
Thank you very much for your time and interest, and we look forward to updating you on our advancements.
Thank you, Sylvia. I just want to thank all of our attendees for taking part in what has been a very productive and informative series of presentations. We appreciate all your efforts and are very grateful for your presence at our conference this year. Thank you.