Lytham Spotlight Series | Kiora Pharmaceuticals (Nasdaq: KPRX)

Hinzugefügt: 2026-05-06

[00:00:00][music] [music] >> Hello everyone. Welcome back to the Lithium Spotlight series where we shine a light on public companies through insightful interviews and exclusive updates. My name is Robert Bloom, managing partner here at Lithium and your host.

[00:00:26]Today, we're excited to feature Keio Pharmaceuticals, a clinical stage biotech focused on retinal disease advancing small molecule therapies to address vision loss with two phase two programs underway. Keio 301 aimed at restoring functional vision in retinitis pigmentosa and Keio 104 targeting retinal inflammation and macular edema.

[00:00:54]301 is supported by strategic partnerships with Teijin Laboratories in Europe and Senju Pharmaceutical in Asia providing external validation, reducing development risk and establishing a path to future royalties.

[00:01:09]I'm joined by the company's chief executive officer to discuss recent developments including their latest Nature Medicine publication, progress with the 301 program and how the company is positioning itself both clinically and commercially within this emerging category.

[00:01:27]Before we begin, please note that this discussion may include forward-looking statements. We encourage you to review the disclaimers and disclosures available at the end of this video or in the description box below.

[00:01:40]With that said, let's get started.

[00:01:42]Brian, welcome.

[00:01:45]Thank you, Robert. Always a pleasure to to be a part of this. Fantastic. Uh You know, let's start off. What is the significance of the Nature Med publication that just came out and what does it change for investors?

[00:01:58]Yeah, so you mentioned the word validation in your intro there with respect to the partnerships that we have in place.

[00:02:05]And we view this publication obviously in what is considered to be a top-tier peer-reviewed journal as further validation into the work that we're doing and really the promise around Keio 301 in not only being a safe but potentially effective therapy in helping patients with mid-to-late stage retinitis pigmentosa regain vision that was unfortunately taken away from them.

[00:02:32]Yeah. Where is sort of the Keio 301 program today and what are the key next milestones?

[00:02:39]Yeah, so we are actively in collaboration with our partners, Laboratoire Théa, who you mentioned earlier as well as Senju Pharmaceutical enrolling a phase two randomized controlled multi-center study.

[00:02:53]That study is going to consist of about 36 patients that again we will be dosing repeatedly over about a 3 to 6-month period of time and looking to see if not only can we repeat some of the data that we generated in our first in man study, but how does it hold up against a control arm which is obviously critical as we develop drugs in the space.

[00:03:16]But then secondly, it's a really interesting approach here because what we are doing is giving vision back to patients who have lost it due to degeneration of photoreceptors, but vision is not exclusively in the eye.

[00:03:29]It's also in the brain and that's where we process what the eye is actually capturing and sending to the brain for vision.

[00:03:36]So the idea of doing repeat injections we hope to see that these patients not only will improve vision after their first injection, which is what we did see previously but the potential to learn how to use this new vision and actually get even better with their ability to see after repeat injections.

[00:03:59]Yeah. Can you explain what a photo switch is and and why this mechanism matters commercially?

[00:04:07]Certainly. So what we're doing is we're taking advantage of natural biology in the eye and if you think about how neurons usually fire it's through essentially depolarization and repolarization. In other words, they get activated so they signal and then they get deactivated so they stop signaling.

[00:04:27]What our molecule is capable of doing is actually lodging inside some of those specific ion channels that control that activation or deactivation and then turn those cells on in the presence of light and then turn them back off in the absence of light. And so you can repeat this back and forth or or think almost like a light switch or a photo switch turning on and off.

[00:04:52]And so mechanistically, that's how we see this actually playing out and there's multiple publications prior to this most recent Nature Medicine publication that explains that mechanism of action in in different experimental settings.

[00:05:07]Obviously, the nice part here is we are seeing consistency from all of that earlier work that we have done now translating into our phase 1b clinical study that was just published in Nature Medicine.

[00:05:20]So we're really excited to see that come to fruition and and keep going down this clinical development pathway.

[00:05:26]And then when we think further about it, there's a lot of potential utility beyond its own role as a vision restoring therapy for a patient. So what this molecule is not doing is it's not changing the underlying genetic defects that lead to this disease.

[00:05:45]And so there's certainly a world we could consider where a patient who unfortunately gets diagnosed with retinitis pigmentosa due to one of what what's known to be north of 150 different gene mutations today could get that gene therapy replacement to stop further degradation of their vision.

[00:06:04]But that's not going to give back the vision that was taken away from them.

[00:06:08]And that's where our molecule slots into play and actually gives that vision back at some level.

[00:06:15]Yeah. Expand on how Keio 301 sort of is positioned clinically and how does it compare to competing approaches?

[00:06:25]Yeah, so kind of building upon that last statement, right? Is we know that for instance, sticking with retinitis pigmentosa for a minute, north of 150 different known genes can cause mutations or have mutations that can cause the disease.

[00:06:38]Yet it's interesting how almost all of these disease have a very similar, if you will phenotype, what happens to the patient in terms of losing vision through the degeneration of their photoreceptors.

[00:06:52]And so we know that our molecule, based on how it works, could actually help any of these mutations. So we refer to it as essentially a gene mutation agnostic approach towards restoring vision. And when you think about it from a uh small molecule perspective we are one of the only ones that we're aware of at least that are actively pursuing an approach like this.

[00:07:19]We chatted a little bit earlier about uh partnerships, but maybe expand on and sort of your commercial partnership strategy, what it looks like and why it's strategically important.

[00:07:30]So after the readout of again our phase 1b study, which again things get a little bit delayed, but was just published in Nature Med um we had some relationships already that with some big pharma companies and one in particular really came in saying we want to partner with you on this even though we were having discussions with a lot of different companies at the time.

[00:07:51]And we decided to actually select uh Laboratoire Théa to actually partner on the further development as well as commercialization of the molecule.

[00:08:01]Théa for those that are not as familiar because they are a France a French-based company in Clermont-Ferrand. They're also a private company. So that's why from the Wall Street perspective, not a lot of investors maybe have heard of them or are familiar with them.

[00:08:16]But they are an ophthalmic specific company. They are one of the biggest uh private family-owned companies that is focused in eye care in Europe. They have recently surpassed over a billion dollars in annual sales with their current product portfolio and saw a ton of opportunity and excitement around our Keio 301 asset.

[00:08:39]And so that really is what drove the partnership there where the terms of the partnership are are absolutely out there for those to see at least at some level um but a very good deal for Keio as well as a very high revenue share, if you will, or or via a royalty stream that we will be recognizing through commercialization hopefully of the molecule after it gets registration in in the various territories. And then about a year later in further discussions with Senju Pharmaceutical, who's based in Osaka, Japan whereas Théa did not take any rights to the Asian territories Senju came in and we put an option agreement together that will be triggered at the end of the phase two study for them to go forward or not with respect to taking those commercial rights within most of the countries within Asia as well. And Senju, like Théa, is a privately owned ophthalmic focused company. So there's a lot of parallels between these two organizations and and groups that we are incredibly excited to be working with as we continue the advancement of the drug.

[00:09:53]All right, excellent. How should investors think about the market opportunity and sort of long-term economics of this program.

[00:10:03]So, I think very favorably. RP is an orphan disease. We do have orphan drug designations in key territories and we're continuing to pursue more and more on that front.

[00:10:13]And with these diseases, you always get the opportunity to go after more of these orphan type pricing and then therefore markets that that are quite attractive, especially with the types of deal terms that we have with both Thea and with Senju.

[00:10:27]So, we think that the markets are are substantial and if the drug continues to perform how it has previously, we don't think that we're just stopping at RP with our partnerships, but we think this could play in other inherited retinal diseases, things like choroideremia, uh Stargardt disease and and many, many more. And then also, again playing on its mechanism of action, even thinking about moving into more of the age-related degenerations of the retina, which are very large and established mass market indications with some very big companies, obviously generating substantial revenues off of their therapeutics.

[00:11:07]Good segue there. Beyond KIO-301, what else in the pipeline should investors be paying attention to? Yeah, so we have been talking about 301 for up until now, but we also have another very exciting molecule called 104 or KIO-104.

[00:11:22]This is a small molecule, uh similar in the sense of of a steroid, but we don't have the steroid adverse side effects, if you will, when used in the eye.

[00:11:35]And so, 104 is using a known mechanism of action called DH ODH inhibition, which I know can be a mouthful if you actually spell it out, it's even more so, but the molecule builds on a large body of data in inhibiting DH ODH that Sanofi, who I'm sure most of you are familiar with, had a franchise here with two drugs called Aubagio and Avara and Aubagio that were first-generation DH ODH inhibitors.

[00:12:05]These drugs were indicated for MS and RA, so playing on systemic autoimmune disease.

[00:12:12]We have essentially a third-generation, more potent molecule that we have really dedicated our efforts towards developing this within ophthalmology and within the back of the eye in particular, retinal inflammation.

[00:12:26]And so, we view this as a huge opportunity because a lot of retinal diseases have a big part of inflammation as a component of it. So, if you can suppress some of that inflammation, primarily the T cells that drive a lot of that activity, you have the ability to restore a lot of what these disease progressions potentially are leading towards, which is at the end of the day vision loss.

[00:12:48]Okay.

[00:12:49]Uh talk about the recent financing and, you know, why now?

[00:12:55]Yeah, it's a good question, um because we didn't have to do anything. Our balance sheet was very supportive of us getting through readouts for both of our phase two clinical trials that are actively enrolling. So, it's not like our backs were against the wall or there was some desperate play by the company, but we've been talking with a lot of top-tier uh healthcare-focused institutional investors and an opportunity came up to really cement one of those relationships and that was with Perceptive Advisors.

[00:13:26]And then also, in bringing in an existing investor, AT Capital One. So, two very top-tier investors.

[00:13:32]So, it was more of a strategic play to align more closely with some of these incredible incredible healthcare investors that we know take typically long approaches on stocks and look to help partner essentially with the company to build opportunities for both sides of the street, you know, for for the company as well as for the investors.

[00:13:54]So, we saw that as an opportunity and we're able to get a a small deal done, uh but but really one that we think is meaningful and aligns interests as we continue to think about how we grow Kiora as a company.

[00:14:11]All right. And as a final question here, what do you think investors are still misunderstanding about the story today?

[00:14:21]Uh well, first off, I don't think as many investors know about the story. So, I think that's probably step one is, you know, how do we make sure that people know who Kiora is and what we are actively doing.

[00:14:31]And we're a company that operates incredibly efficiently, but we have an incredible team that knows how to develop drugs.

[00:14:37]We have the ability to expand further into the commercialization of certain therapeutics when that time hopefully comes for this organization.

[00:14:45]We have an incredible investor base now that's completely aligned with us as we think about the future of the company and how we grow that. And we have runway. So, unlike a lot of other companies with similar market caps as we are, we have no financing overhangs that we have any concern around. So, we're going to get through inflection points, we're going to continue to operate efficiently, and I think our pipeline is going to provide a lot of promise as we continue to to bring these drugs into late-stage clinical trials and hopefully to the market.

[00:15:19]All right, very good. Well, Brian, we will leave it there. Thank you very much for that update. Uh thank you to all of our viewers for tuning in to another episode here of the Lytham Spotlight series. Uh if you haven't done so already, make sure to hit the uh subscribe button so you don't miss out on any future content. Uh and if you found today's episode insightful, hit that like button as well uh to let us know. So, Brian, thank you very very much again for your time today.

[00:15:44]Appreciate it. Robert, thanks for having me. All right, thanks everyone for watching.

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