All right, hello everyone, and thank you for joining us throughout the day here at the Lytham Partners Fall 2024 Investor Conference. My name is Robert Blum, Managing Partner of Lytham Partners. During our next presentation here, we welcome Kiora Pharma, ticker symbol of KPRX on the Nasdaq, and joining us today from the company is the Chief Financial Officer, Melissa Tosca. Before I turn it over to Melissa, I want to remind everyone that management is available for one-on-one meetings throughout the conference here. If you've not already scheduled your one-on-one meeting and would like to do so, feel free to send me an email, that's Blum, B-L-U-M, @lythampartners.com, or you can visit the landing page for the website, which is lythampartners.com/fall2024. From there, you can click on the investor registration to make your one-on-one selection.
So with that, Melissa, the floor is all yours.
Thank you so much, Robert. It's great to be here today. Before we get started, I quickly need to make mention of the forward-looking nature of certain statements in this presentation. Simply just means that they're subject to change in the future. At Kiora, we're developing treatments for retinal diseases where there's significant unmet need. We seek 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, I can only imagine, overwhelming. Sadly, with no treatments currently available, patients are given the bleak instruction by their doctors that they must learn to cope with becoming blind. The graphs shown here summarize published data, which confirms the sense of despair and fear of vision loss.
The study participants ranked losing their eyesight to have the greatest effect on day-to-day life, and they ranked blindness as one of the worst conditions above even cancer. I am 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 disease indications. Starting with our KIO-301 asset, we're using a small molecule photo switch to confer light sensitivity. We've started initially with a program in retinitis pigmentosa, and we plan to expand into other diseases as well. 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. So let's jump into KIO-104 first.
We've been able to leverage existing knowledge from other therapeutic areas within rheumatology and apply it to eye-related diseases that could also benefit. We know that swelling is caused by inflammation and can often lead to 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, which is the first disease indication we're focused on. Posterior non-infectious uveitis is a group of disorders that are characterized by inflammation in the back of the eye. This inflammation is often chronic and can flare up at any time, leading to visual impairment and ultimately vision loss. Currently, the clinical symptoms are being treated by steroids, which can cause adverse effects, leading to cataracts and even glaucoma.
There's estimated to be approximately 400,000 patients in the U.S. alone with this condition, so we feel that this is a significant area of need, and it provides great market opportunity. We've previously run a phase I/II study in posterior non-infectious uveitis. This was a first-in-human study and was a single intravitreal injection, where our primary objective was to ensure safety and tolerability while seeing improvement in inflammation. In terms of the results, we saw no serious adverse effects. There was excellent tolerability at all doses, I'm sorry, and we saw that this drug remained within the ocular area it was injected in, and did not pass systemically through the blood to other peripheral areas.
The results also showed meaningful data improvement in visual acuity, highlighted in the chart on the left, as well as improvement in swelling within the retina after a single intravitreal dose. That's highlighted in the images on the right. As the next steps, we're planning to begin a phase II trial in the H1 of 2025 . 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 we'll be 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 move on to KIO-301, our small molecule photo switch. Retinitis pigmentosa is the first disease area we're focused on for KIO-301.
It's actually a group of diseases that can be caused by over a hundred and fifty known mutations in over fifty genes. And given our small molecule is mutation agnostic, we believe we can reach all of these patients, unlike a gene therapy that would be targeted to a specific gene or a mutation. Patients generally begin losing their vision in their upper teens, twenties, and become legally blind by their mid-fifties. Typically, they begin to 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, these are the patients that are given that hopeless diagnosis, that they should learn to cope with the inevitable loss of their vision. We've completed our phase Ib ABACUS study and released top-line data last November at the American Academy of Ophthalmology conference.
This was a first-in-human study where we looked at different drug amounts among patients with different levels of disease progression. Safety was of paramount importance, as always, and we had a host of efficacy endpoints. 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 count fingers. Across both cohorts, all the patients were considered off-chart, meaning they couldn't read even the largest letter on an eye chart. In terms of takeaways from the study, most importantly, there were no safety or tolerability concerns noted. We saw improvement in visual acuity, visual field, and functional vision.
In December, we had a preliminary IND meeting with the FDA, where we were able to get important clarifications from the regulators on the approvable outcome assessments to ensure alignment of our upcoming phase II trial design. Since then, we've had ongoing dialogue with those regulatory bodies. On the heels of this data, there was a lot of excitement, as you can imagine, which led to us putting together a strategic partnership with a French company known as Théa Open Innovation. This was for the global, excluding Asia, rights to KIO-301 for all inherited retinal diseases. Upon closing, we received a significant upfront payment of $16 million, and in addition, Théa will reimburse all phase II trial costs and will then conduct and fund the phase III trials.
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 into the 20%. This was a very meaningful deal for the company that has provided us with significant non-dilutive capital that extends our cash runway beyond two years into 2027. This puts us in a great position relative to other micro-cap companies, where we don't need to raise additional capital through dilutive equity financings. We've kicked off our phase II study preparations. This will be a larger study of approximately 36 patients and will include a control group. Across four visits, the patients will receive intravitreal injections that will be spaced out about three weeks apart. There will also be an open-label extension, where the control group will also be able to receive the drug, if they choose.
I am so grateful to work alongside an incredible group of individuals. Our leadership team is comprised of experienced executives who bring ophthalmology expertise, as well as drug development experience. We have worked across all asset life cycles, from development all the way to commercialization, and most importantly, we are truly passionate about making an impact in the lives of patients who are suffering with vision loss. We have several upcoming milestones over the next year. We believe all of these will provide multiple value inflection points. For KIO-301, specifically, before year-end, we'll initiate our ABACUS 2 phase II clinical trial, and then in Q1 of next year, we'll complete the endpoint validation for our functional vision assessments. During the H2 of next year, we plan to complete enrollment for the ABACUS 2.
For KIO-104, we're finalizing our non-clinical data package, which we hope and plan to have complete before year-end, and we'll be initiating our phase II clinical trial in the H1 of next year for that program. So certainly lots to be excited about related to these programs. In conclusion, we have innovative modalities, both of which are small molecules. We see significant market opportunity for both of our assets. Specifically for KIO-301, there are approximately a hundred thousand patients in the U.S. with inherited retinal diseases, and looking at KIO-104, there are even larger disease populations we believe we can reach.
The partnership with Théa de-risked the KIO-301 program, while still allowing us to pursue partnership for the Asia rights, and importantly, it provides us with that non-dilutive capital that enables us to continue the development of the KIO-104 program, for which we have the commercial worldwide rights. We have a strong cash position of approximately $28 million, which provides runway extending into 2027, and with that, I just want to thank you so much for your time today. It's been my pleasure to share about the exciting work we're doing at Kiora, and Robert, I'll pass it back to you to facilitate the Q&A.
Perfect. Thank you very much, Melissa, for that overview there. I have a few questions here. Earlier this year, you reported additional results from functional MRI scans of patients in ABACUS 1. What is that data telling you guys?
Sure. So the most recent data quantified activity in the brain following the treatment of KIO-301, and this functional MRI data demonstrated a significant increase in neural activity over baseline within the brain's visual processing center. The increase was time-dependent and demonstrated concordance with previously reported improvements in visual field, visual acuity, along with functional vision. Importantly, though, the data reinforces what we believe is the mechanism of action for KIO-301 that provides benefit and gives us confidence that the compound is doing what is intended.
... Okay, great. What's happening with regards to ABACUS 2?
Sure. So we are currently interacting with the European and U.S. regulatory agencies. Based on the feedback that we've received so far, both agencies are moving toward functional endpoints as the ultimate approval. This, from our perspective, is great news, given we've observed improvements across several functional endpoints in ABACUS 1, and, that's, you know, provided us with insights on how to refine and improve those assessments further in ABACUS 2, as well as any subsequent studies.
Help for people that may not understand, what do you mean with regards to functional endpoints?
Mm-hmm. Of course. So there are some clinical trial endpoints that assess underlying disease factors as a proxy for patient benefit, such as relapses in MS or lesion size in GA. We're seeing, however, that regulatory bodies are moving toward assessing how new therapies are impacting day-to-day living versus benefiting underlying biological effects. Simply put, is the patient feeling better, or do they have a higher quality of life?
I guess, as it relates to Kiora, you know, what do you think that this ultimately means for you guys? Sounds like you think it's beneficial.
Yeah. So, I mean, given our cash position, we're able to invest the time in completing the endpoint validation work for ABACUS 2. Once complete, you know, we look forward to dosing the first patients in that trial, and we'll be able to really generate the most meaningful data possible. And if the trial is successful, as we will, you know, certainly expect it to be, we're then positioned to conduct a single phase III to seek approval in both the U.S. and Europe.
Okay. All right, that's helpful. Shifting here to 104, you know, talk about, for investors, what makes this product unique?
The MOA for 104 has already been shown to work clinically and commercially. It's part of the same family of drugs as Aubagio, which is an oral medication for multiple sclerosis, and this drug has been used to benefit hundreds of thousands of patients worldwide already. We're looking to use our proprietary and even more potent compounds, which are in the same class as Aubagio, to apply it to the eye, and we feel it could address a significant need for a variety of inflammatory indications, both rare, like uveitis, as well as larger diseases like diabetic macular edema.
Okay. All right. Maybe the final question here. You mentioned you had cash into 2027. Obviously unique in today's environment here. I guess my question is, does this include any of the anticipated milestones that you talked about from the partnership?
I am happy to say no, it does not. So any milestone payments will be additive and will extend our runway even further. And what that means for Kiora and importantly for our shareholders, is that we have runway that brings us beyond our anticipated data readouts for two clinical trials.
Okay. All right, perfect. Well, let's. We'll go ahead and leave it there. Melissa, thank you very much for your time here today. I want to remind everyone just two quick items. Again, if you've not already scheduled your one-on-one with the management team here at Kiora, you can send me an email. Again, that's Blum, B-L-U-M, @lythampartners.com, or again, go to the landing page for the website, lythampartners.com/fall2024. From there, you can click on the Investor Registration button. And I would make this comment as well, if you would like to meet and today doesn't work, happy to look to coordinate something after the event as well. Again, we have additional presentations coming up. Again, visit the conference homepage for all the information. Again, Melissa, thank you so much for your time.
Really hope you enjoy the conference.
Thank you so much, Robert.