All right, welcome everyone. We're going to get started here. So we are in track four, and I would like to welcome Melissa Tosca from Kiora Pharmaceuticals.
Thank you so much. It's great to be here today. I'm Melissa Tosca, the CFO for Kiora Pharmaceuticals. At Kiora, we are developing treatments for retinal diseases where there's significant unmet need. We're seeking to improve sight in patients with severe vision loss due to inherited or age-related diseases. The despair that's experienced by someone who's lost or is losing their vision is overwhelming. Sadly, with no treatments available currently, patients are told they must learn to cope with the inevitable progressive loss of their vision, ultimately leading to blindness. The graph shown here summarizes published data, which confirms this sense of despair and fear around vision loss.
The study participants ranked losing their eyesight to have the greatest effect on day-to-day life, seen in the top graph, and they ranked blindness as one of the worst conditions above Alzheimer's disease and even cancer, as you can see in the graph at the base of the slide. I'm inspired, I'm so inspired to be at Kiora, where we're striving to replace that despair with hope as we work to slow and restore vision loss. Our pipeline is comprised of two assets that have application across several retinal diseases. Starting with our 301 asset, we're using a small molecule photoswitch to confer light sensitivity. We started initially with a program in retinitis pigmentosa, and we plan to expand into other diseases as well, including choroideremia and Stargardt's disease.
We also have another asset known as KIO-104, which is a non-steroidal small molecule that's focused on helping patients with retinal inflammation. Let's jump into KIO-104 first. We know that swelling is caused by inflammation and can often be a downstream effect of many eye-related diseases or issues, including diabetic macular edema, retinal vein occlusion, cataract surgery, as well as posterior non-infectious uveitis. We've been able to leverage existing knowledge from other therapeutic areas within rheumatology and apply it to the eye, to specifically eye-related diseases that could also benefit. Looking at posterior non-infectious uveitis specifically, it's a group of disorders that are characterized by inflammation in the back of the eye. This inflammation is often chronic. It can flare up at any time, leading to visual impairment and ultimately vision loss.
Currently, the clinical symptoms are being treated by steroids, which are not ideal given they can cause adverse effects leading to cataracts, glaucoma, and ultimately blindness. There's estimated to be approximately 400 patients in the U.S. alone who suffer with this condition, so we feel this is a significant area of need and market opportunity. Our preclinical data has looked very promising, where we've been able to see positive effects indicating reduced inflammation as well as a reduced number of relapses. We've previously run a phase I/II study in posterior non-infectious uveitis. This was a first-in-human study, and it was a single intravitreal injection where really our primary objective was to ensure safety and tolerability while seeing an improvement in inflammation. In terms of results, importantly, we saw no serious adverse events.
There was excellent tolerability at all doses, and we saw that this drug remained within the ocular area it was injected, not passing systemically to peripheral areas. The results also showed meaningful improvement in visual acuity, highlighted in the chart on the left, which compares overall change in eye chart letters from baseline at increasing doses with measured improvement compared to Humira as well as steroids. We also saw improvement in swelling within the retina after a single intravitreal dose, which is highlighted in the images on the right, where you can see reduced swelling from baseline to day 28. As next steps, we're planning to begin a phase II trial in the first half of 2025. The K is not a typo. It is KLARITY for Kiora.
We're going to be looking at a few different disease states that all have macular edema, which is swelling in the central part of the retina, and then expanding that into a matched-controlled study where we'll be assessing multiple doses of the drug in a way to evaluate its potential ability to help reduce swelling and improve visual acuity for these patients. Let's go ahead and move on to KIO-301, our small molecule photoswitch. Within the retina, we have rods and cones, which are the photoreceptor cells that take light and convert it into electrical energy. That energy is transmitted into cells known as retinal ganglion cells, RGCs, which relay that signal to the brain. In many retinal diseases, the rods and cones die, and that ultimately is what leads to blindness.
However, since RGCs are cells that remain alive, we're able, through our small molecule, to target those cells and essentially turn them into light-sensitive cells. Our small molecule enters RGCs and alters the ion flow through a channel that causes the cell to be activated when light is present or deactivated when light goes away. We've completed extensive validation in preclinical models. In the cell models, we were able to see that when electrical energy was applied to treated cells, there was a noticeable response to light, which you can see as the signal is turned on and off, that wasn't seen in the untreated cells on the left. Additionally, in mouse models, we saw evidence of behavioral changes in the mice when they were exposed to light, where they ceased movement. As we know, mice are nocturnal animals, and generally, they're more active in darkness.
We saw no change in movement of untreated blind mice, so to speak, when exposed to light, but in treated mice, we saw normal movement in darkness and a decline in movement when they were exposed to light, indicating that the drug was having an effect on their ability to see light. The inherited retinal disease landscape is quite vast. However, the largest area of unmet need is in patients with a disease known as retinitis pigmentosa. On this basis, we've started with RP as our first disease indication and intend to expand into other disease areas as well. So a little bit about retinitis pigmentosa. It's actually a group of diseases that can be caused by over 150 known mutations in over 50 genes.
Given our small molecule is mutation-agnostic, we believe we can reach all of those patients, unlike a gene therapy that would be targeted to a specific gene or a specific mutation. Patients generally begin losing their vision in their upper teens to 20s and become legally blind by their mid-50s. Typically, they experience symptoms of night blindness or reduced peripheral vision, which gradually extends to the loss of their central vision as well. With no available treatments currently, patients are left hopeless. They are given a diagnosis that basically they should learn to cope with the inevitable loss of their vision. We've completed a phase I-B ABACUS study and released top-line data last November at the American Academy of Ophthalmology. This was a first-in-human study where we looked at different drug amounts among patients with different levels of disease progression, and specifically, we had two cohorts.
The first cohort was comprised of patients who had no light perception or bare light perception, and the second cohort was comprised of patients who could see hand motions or they could count fingers. Across both cohorts, patients were considered off-chart, meaning they couldn't read even the largest letter on an eye chart. We saw improvement in visual field among these patients, which you can see here in the graphs. We also saw an improvement in visual acuity using the Berkeley Rudimentary Vision Test, and one of the tests specifically was looking at light perception, essentially exposing patients to a series of illuminated letters presented on a screen from a rear projector. They were asked basically to indicate if light was present and also if they could identify the letter. From this, we saw an overall improvement in light perception, especially among the NLP no-light perception patients.
Some of these patients had not seen light prior to the study for many years, and they were indicating that they were seeing light, which, of course, was tremendous news. We also used a variety of mobility courses to test their functional vision. We were looking to test their ability to navigate around objects in a room, to identify the location of windows as well as doors. From this, we saw an overall improvement from baseline in the functional tests, but more importantly, we gained additional insights that we can apply in the functional vision tests for phase II. Another interesting aspect of this study was the functional MRI data that we obtained.
This technology allowed us to visually capture and quantify changes happening in the vision processing center of the brain throughout the treatment, noticing that at the four-week mark, the drug had essentially metabolized, and we had seen the decline of that activity. The data from this study shows, though, an increase of that activity, which you can see from the light-up areas in the earlier administration of the drug through its metabolism to week four. The patient testimonials from our phase I-B study have been tremendous. We have been able to hear direct feedback from the patients, which has indicated the positive ways that they've been impacted as a result of this study. These are just a few of the quotes from some of the patients in the study.
We didn't include videos for this presentation, but I encourage you to go to our website and watch the videos there because I think hearing it directly from a patient is incredibly inspiring. In terms of takeaways from the study, most importantly, there were no safety or tolerability concerns that were noted. We saw improvement in visual acuity, visual field, and functional vision, and last December, we had an IND meeting with the FDA where we were able to get important clarifications from regulators on the approvable outcome assessments to ensure alignment of our upcoming phase II trial design. On the heels of this phase I-B data, there was a lot of excitement, which is what led to us putting together a strategic partner with Théa Open Innovation for the global excluding Asia rights to KIO-301 for all inherited retinal diseases.
Upon closing, we received a sizable upfront payment of $16 million. In addition, Théa will reimburse all of the phase II trial costs, and they will conduct and fund the phase III trial. As we achieve various milestones, we'll receive additional non-dilutive capital of up to an additional $285 million, as well as tiered royalties upon commercialization that reach into the 20%. This was a very meaningful deal for the company that's provided us with significant non-dilutive capital that extends our cash runway beyond two years into 2027, excluding any additional milestones. This puts us in a great position relative to other microcap companies where we don't need to raise additional capital through dilutive equity financing. We've kicked off our phase II study preparations. This will be a larger study of approximately 36 patients, and it will include a control group.
The patients will receive three intravitreal injections spaced out six weeks apart, approximately. There will also be an open label extension whereby the control group will also be eligible to receive the drug if they choose. We've currently received a grant from the Choroideremia Research Foundation to fund validation of novel functional vision endpoints, and specifically, these are looking to measure the ability to identify objects, to navigate around objects, to identify light from different locations and objects, as well as doorways and windows. But these endpoints are really what will help us to assess whether there's overall functional vision improvement in the everyday life of these patients, which is essential for drug approval based on feedback that we've received in our early conversations with the FDA. I am so grateful to work alongside an incredible group of individuals.
Our leadership team is comprised of experienced biotech executives who bring extensive expertise as well as drug development experience. We have worked across all asset life cycles from development to commercialization, and we are truly passionate about making an impact in the lives of patients suffering with vision loss. We have several upcoming milestones over the next year, which we believe will provide multiple value inflection points. For KIO-301, today, we announced that we received IND approval from the regulatory agency in Australia to initiate the ABACUS-2 phase II clinical trial. In early Q1 of next year or sooner, we'll complete the endpoint validation of our functional vision assessments, and during the second half of next year, we plan to complete enrollment of ABACUS-2.
For 104, we're finalizing our non-clinical data package as we speak, which we plan to complete before year-end, and we plan to initiate our phase II clinical trial in the first half of next year for that program. In conclusion, we have innovative modalities, both of which are small molecules. We see a significant market opportunity for both of our assets. For KIO-301, there are approximately 100,000 patients in the U.S. with inherited retinal diseases, and looking at KIO-104, there are even larger disease populations we feel that can be reached. The partnership with Théa de-risked the 301 program while still allowing us to pursue partnership for the Asia rights, and importantly, it provides us with non-dilutive capital that enables us to continue development of 104, and for that program, we have the worldwide commercial rights.
We have a strong cash position of approximately $28 million currently, which provides runway extending into 2027. And with that, I want to thank you for your time today. It's been my pleasure to be here and share about the exciting work we're doing at Kiora. And I'm happy to take any questions if there are any. Yes.
Commercialization by your side. It's okay. Are you a contract manufacturer, or what is your goal to commercialize this product?
Sure. So for commercialization of KIO-301, we're going to be leaning on our partner, Théa. So they own essentially the commercial rights to KIO-301 for commercialization in both the U.S. and Europe. So we intend to see that program commercialized in the U.S. and Europe, and we'll be intending to receive a royalty of upwards of 20% on those net sales.
And then for 104, we have full rights to global commercialization. Granted, we are a small, lean, and nimble company, and so it's hard to say, "Yes, we will internally build out a commercial sales force and commercialize it ourselves," but that's what we continue to plan towards. And if we are successful through these trials, then that gives us the ability to make that determination of, "Do we move in that direction, or do we partner that program as well?"
Yeah, sorry, can you catch the low percentage you did on the first one?
I'm sorry, what was that? Oh, upwards to 20%.
301 deal. So you retain the U.S. rights?
So the question was for the 301 deal, did we retain the U.S. rights? No, we did not. We partnered 301, and essentially, Théa Open Innovation owns the commercial rights in the U.S.
For that program as well as in Europe. But we still own the commercial rights to Asia or the ability to partner in Asia separate from the partnership with Théa.
What's the next data for that?
The next data, yeah. So we just received the approval to initiate today, and so we intend to finalize those functional endpoints and expect to dose our first patient in early 2025. We expect to have full enrollment in the second half of 2025, and the trial will extend approximately a year to a year and a half. So a readout at the end of 2025 or into early 2026.
It's an open label trial, you said, right? That you were going to talk about some of what you're doing there? And RP?
Yes.
Is there anyone else doing? What is your competitive landscape aside from the gene therapy approaches that you said? Are you not doing?
The question was, in terms of competitive landscape, what else is out there? Currently, we're not aware of another company that's working on a small molecule. It doesn't mean that they're not in stealth mode and working on it without our awareness, but as it relates to a small molecule approach, we're not aware of another company working on that. We are aware of several companies that are working on gene therapies, and again, the difference there is that those would be specific to a single mutation or a specific mutation. We know there's a lot for these diseases where our small molecule is mutation- agnostic and could span across a variety of mutations and genes.