For this session, we'll have a presentation by Oculis. Oculis is a clinical stage ophthalmology company with a pipeline of candidates targeting diabetic macular edema, dry eye disease, and acute optic neuritis. Presenting for the company is Ms. Sylvia Cheung, Chief Financial Officer of Oculis.
Thank you. Good morning, everyone. Thank you for your patience, and thank you, Yi, for the introduction. Thanks to H.C. Wainwright for the opportunity to present to you Oculis. Before I begin, I'd like to remind you about the safe harbor statements related to forward-looking statements that I'll be making during today's presentation. Oculis is a global pharmaceutical company focused on innovations addressing ophthalmic and neuro-ophthalmic diseases. We are dual-listed on the U.S. and in Iceland. We have innovative portfolios based on three core assets: OCS-01, OCS-05, which is Privosegtor, and OCS-02, Licaminlimab, addressing significant market opportunities. OCS-01 is the first eye drop for the treatment of DME. OCS-05, Privosegtor, is a first-in-class neuroprotective candidate, which recently announced very positive phase two results, and we're currently advancing into three indications. I will go through what they are and the meaning of this transformative data. OCS-02 is Licaminlimab.
It is a novel topical anti-TNFα in genotype-based development, and the lead indication there is a personalized medicine for dry eye disease. We have a strong balance sheet, reported $206 million of cash on hand at the end of March, no debt, and runway is into early 2028. The company is led by an experienced management team with a track record of success and supported by leading international healthcare investors. Here are our three core assets: O-01, Privosegtor, and Licaminlimab, and their related leading assets in those three categories. Down at the bottom of the slide, you can see the visual issues that patients experience. Let me give you an overview of each of these leading assets. O-01 is a proprietary optic reach formulation of high-concentration Dexamethasone.
It is a topical treatment for DME to enable early intervention for patients who are not receiving treatments and for patients at the back of the treatment cycle who receive inadequate responses to the current standard of care. For Privosegtor, which currently the leading indication is acute optic neuritis, Privosegtor is a protective peptoid small molecule. It's a novel neuroprotective candidate promoting neuronal and axonal survival with broad potentials in neuro-ophthalmology and neurology. For Licaminlimab, our leading asset is dry eye. The leading indication is dry eye. It's a TNF blocker with anti-inflammatory and anti-necrosis mode of action in genotype-based development to drive personalized medicine. For this presentation, I'll be focusing on DME in OCS-01, acute optic neuritis in OCS-05, Privosegtor, and dry eye for Licaminlimab in OCS-02. Now, as we all know, DME is a large and growing market with untapped opportunities.
Today, the only available treatments are all invasive, meaning injectables or implants. These obviously come with a high burden of treatment as well as very low patient compliance. As a result, we only see 44% of the diagnosed population currently being treated, which left a vast majority of the population untreated. Within the treated group, there is also inadequate response. How is DME diagnosed? It's actually very simple: through OCT imaging. 56% of the diagnosed patients are in what we call the early intervention group, where there is no available treatment today. What they're experiencing is just observation. 44% of the diagnosed population currently is receiving diagnosis and treated, of which 40% is not receiving adequate response.
Where OCS-01 can come in, it's really to provide versatile treatment alternatives across the whole continuum of DME care, from early intervention all the way to diagnosed and treated population. Our aim is to be first line in early intervention and offer new treatment options in the diagnosed and treated population by either in combination with injectable or as a standalone option. The addressable market is very large. In the U.S., it is estimated at about 1.3 million. The company had positive and consistent results across four previously completed studies. What I would like to highlight for you is our phase III stage one study, which showed really strong results. Here are the five takeaways. First is we saw that patients treated with OCS-01 achieved 7.2 letter gain in BCVA at week six and increased to seven point six at week 12, which is the end of the maintenance period.
Second, when we look at the number of patients gaining 15 letters or more, we achieved 25.3% at the end of week six and improved to 27.4% at the end of week 12, the maintenance period. Moreover, from a CST or CMT standpoint, which is the measurement of retinal thickness, we saw very rapid and sustained reduction of retinal thickness. As a matter of fact, p-value at 0.001 at the end of week six and maintained up to week 12. When we looked at the efficacy between naive patients and previously treated patients, we saw positive results in both populations. From a safety standpoint, there are no SAEs reported that were deemed to study drug-related, and no evidence of cataract formation of up to 12 weeks, IOP increase consistent with letter chart. The product is very well tolerated with no unexpected AEs.
With the very positive results, we advanced into stage two of our phase III program. It's a randomized double-blind placebo-controlled study, 12-month trial with over 800 patients. The primary endpoint is change in BCVA ETDRS letter score at week 52. The key secondary endpoint is a percentage with over 15 letters gain at week 52. We rapidly enrolled over 800 patients. We just recently announced completion of patient enrollment, so 119 sites were activated, over 800 patients enrolled. We expect to announce top-line results from our phase III trials, both trials in Q2 of 2026. Here we're looking at the current and future DME patient management. As I mentioned before, in the early intervention segment, we're looking to be the first-line treatment for patients with visual acuity worsening and/or presence of center-involving edema.
For the diagnosed and treated patient, you may recall that 40% of them are not getting adequate responses to anti-VEGF. We can come in as a versatile solution to be used standalone or as a combination to help bridge for patients who are not coming as frequently as they should for anti-VEGFs. As a result, if you look at the 1.8 million patients in the U.S. who are diagnosed with DME, currently 500,000 are treated with well-controlled results, whereas 1.3 million are not. We are looking to address that 1.3 million population with OCS-01, which is the only topical that can get into the back of the eye to treat DME. First-line treatment for early intervention, versatility across the continuum of care from a treatment option standpoint. It also expands the potential prescriber base. Retina specialists at 3,000 in the U.S.
In the future, general ophthalmologists can also be prescribers. They are multiple folds, actually 15,000 of them here in the U.S. In conclusion, large market with 1.3 million large addressable population here in the U.S. phase III trials enrolled very rapidly, ongoing with enrollment completed and results expected in Q2 of 2026 with, if positive, NDA filing in the second half of next year. Now let me move on to Privosegtor for acute optic neuritis. Privosegtor is a novel asset for Oculis. The result is transformative, which we announced earlier this year. It's a neuroprotective candidate that activates neurotrophic signaling pathways supporting neuronal and axonal survival and preservation. As I mentioned before, it's a small molecule peptoid that penetrates the blood, brain, and retinal barrier. It activates SGK, thereby activating the FOXO3 pathway, which is known to be related to neuronal survival response.
It triggers multiple functional and beneficial effects on apoptosis, oxidation, and inflammation. What is Acute Optic Neuritis? It's an acute inflammation of the optic nerve that can lead to permanent visual damage. As you can see there, vision loss is particularly affecting color and contrast. The retinal ganglion cells and axons in the optic nerves are being insulted and have injuries. It is also directly linked to chronic conditions like multiple sclerosis and other autoimmune diseases. Before I get into the data results, I would like to first talk about AON, which currently has no approved products for a Neuroprotective Therapy. You can see from a visual standpoint, color and visual contrast are deeply impacted for those patients. From a measurement standpoint, they are actually quite easy. On the biological front, there are two measurement metrics. One is GCIPL. The other one is RNFL.
From a functional standpoint, it's LCVA, Low Contrast Visual Acuity. Now, RNFL is the top layer, if you can take a look at the middle of the graph with orange dots and the yellow background. It's a thickness measurement for the axon, whereas GCIPL is the thickness measurement for retinal ganglion cells. We completed our acuity phase two study for acute optic neuritis. The great news is we achieved primary safety endpoint and secondary endpoint showing functional improvement and neuroprotective benefits on vision, anatomy, and biology. Safety is good, as you can see at the bottom of this slide. Now, let me share with you the efficacy endpoints. Patients who received Privosegtor at the end of month three showed 43% less thickness reduction, which means protection of retinal ganglion cells from the treatment of Privosegtor, which got maintained to month six.
Secondly, on RNFL, patients receiving Privosegtor comparing to the placebo arm showed 30% improvement in less thickness decrease, which means the axons are protected as well. When we look at the functional efficacy endpoint, patients received Privosegtor showed 18 letters improvement versus the placebo arm at month three and maintained to month six. This is really great because there is a very significant improvement in low visual contrast visual improvement. We also took a deeper look into the patient populations. No matter how we look at it, whether our patients with MS or non-MS or AON patients with HCVA less than 60 or equal to or greater than 60, no matter how we look at it, we saw benefits across different groups and across different time points, which is extremely encouraging. More importantly, we saw that patients receiving Privosegtor arm achieved lower neurofilament release.
Neurofilament, as you know, are released into the cerebral spinal fluid when the axons and neurons are dying or are suffering from injury. In this case, you can see on the graph on the right-hand side, there is a very nice graph, the purple line, showing reduction in neurofilament released into CSF. Obviously, because it is an acute disease, AON is an acute disease, at the end of month six, when the acute trauma is behind, patients are more or less back to a more normal level. We were very encouraged and pleased with the outcome from the acute optic neuritis phase II study. As a result, we are moving full steam ahead on the investigational program for AON. Furthermore, we are adding two indications: acute relapses in multiple sclerosis and NAION are two additional indications we will be adding to OCS-05.
Now, multiple sclerosis, as you know, is the most common CNS condition affecting young adults. Very high prevalence, 2.8 million worldwide and 850,000 here in the U.S. Large and growing market driven by immunomodulators today. The existing therapies only slow down disease progression where relapses continue to cause disabilities. Relapsing remaining MS is characterized by acute attacks, which means relapses, and may cause permanent disabilities. Here in the U.S., it is roughly about 170,000 estimated per year. 85% of the patients would have the RMS at initial diagnosis. The current standard of care is steroid IV with incomplete recovery leading to neurodegeneration and permanent disability. What we are aiming to do is to leverage the science on Privosegtor by helping patients to address relapse-associated worsening, meaning lowering the relapse-associated worsening. You can see here, relapses will occur.
The aim for OCS-05 is to reduce the accumulation of disability by dealing with the relapses, similar to how we were able to achieve in the acute optic neuritis disease. What is NAION? It is a rare disease. No treatment is currently available. It has orphan indication. Here in the U.S., roughly about 20,000-30,000 patients, impacting patients over 50 years of age. Retinal ganglion cells, axons, and optic nerve atrophies are caused by hypoperfusion. It would lead to permanent, potentially permanent visual losses in many patients. As a matter of fact, over 60% of patients have significant visual impairments in the affected eye. Our aim is also to leverage the Privosegtor neuroprotective benefits to help patients with NAION. Our next steps are to meet with the FDA in the third quarter to discuss the registration study for acute optic neuritis.
From there, we'll advance into investigational trial preparation. We anticipate to start that in the first half of 2026. For NAION and MS relapses, the two newer indications that we are looking into, we are planning to complete some IND readiness activities and also speaking with the FDA in the second half of this year in terms of development pathway. We have potential IND submissions planned in 2026. Now, Licaminlimab is a novel asset. It's a topical biologic initially targeting dry eye. It's a clinically proven new MOA with potential transformative impact on ocular inflammation. Not only is it a proven MOA, it enhances ocular penetration. Furthermore, we have identified a genotype who super responds to OCS-02 or Licaminlimab.
Now, in the interest of time, I'll jump to phase II, the latest trial that we did, the outcome, as well as our next steps for this particular asset. When we designed the phase 2b trial, there were three objectives in mind. First is to evaluate Licaminlimab in the treatment of signs for dry eye disease. The second is to show that there is differentiation for Licaminlimab in subjects with TNFR1 genotype biomarker. Third is to select the right and the most beneficial endpoint from an efficacy standpoint for signs in order for us to move into registrational studies. The good news is we were able to identify inferior cornea staining, which is reflective of inflammation and apoptosis, which plays a crucial role in dry eye disease. It is also the most commonly assessed sign in clinical practice for dry eye.
What we saw is for inferior cornea staining, for full population, we achieved negative 0.12, which is better than the best outcome of Xiidra from a sign efficacy standpoint. For patients with TNFR1, we saw six times better than the Xiidra outcome, which is very positive for us. Not only is the efficacy very strong, it also kicks in really fast, week two. Across other signs efficacy measurements, we saw profound improvement for patients with TNFR1. Therefore, we are moving into our registrational study and expect to start that toward the second half of this year with readout in the second half of next year. In conclusion, I would like to share that the Oculis pipeline has transformed strategically over time. We expanded beyond ophthalmology into neuro-ophthalmology thanks to Privosegtor's great outcome, which substantially increased our addressable market.
Our portfolio is deep, innovative, highly differentiated, and targeting large unmet needs, including first topical for diabetic macular edema, first topical biological eye drop for dry eye disease, and first in class neuroprotective asset for acute optic neuritis moving into registrational study, as well as additional indications to capture. We are really well positioned to generate multiple value catalysts in the coming quarters. I am very happy to share the Oculis story with you and see if there are any questions that people may have. Very good. Thank you for joining us this morning.