All right, well, I think we'll just get going. Thank you very much for being here. So we're KALA BIO, and at Kala, what we're working on is a really high-value program for the PCED market. It's persistent corneal epithelial defect, and in 2024- No worries. Take... Don't be sorry. Take your time. We know Adam really well, so-
That's kind of secret in biotech.
Here it is. Here it is.
It's all right.
That's all right. A couple are coming in, so we'll just wait a second. Hello, and welcome. We are KALA BIO, and we are working this year on a phase II study for persistent corneal epithelial defects. What we're most excited about is that we truly do have one of those pipeline in a product applications that our secretome program enables, and we've started a phase II that is enrolling as we speak. We have really meaningful milestones coming up this year, so our phase II data, phase IIb data will read out, hopefully by the end of the year. That's our target. We, in addition to that, have a very experienced team. We've been well-funded.
We have cash that takes us well beyond that Phase IIb readout, and maybe the most exciting thing is that corneal epithelial defects is a massive market. There's one other product in that market. It's only been on the market for a few years, and it already exceeds $700 million. We believe we're able to make an even better product. It's a terrific product that we're competing with, but our goal is to make a product that's even better. Each one of our executives and our team members has well over 20 years of drug development experience.
Our CMO, who's sitting in the front there, Kim, has been working in ophthalmology for many years and has a huge track record of having products approved through the FDA. We as a team, actually earlier in Kala's history, have already gotten two products the whole way through FDA approval. We're very excited about our track record and, you know, this is what we've been working on for the last couple of years, is to advance programs into the clinic. We believe we have a really strong CMC and R&D engine. You can see the Phase II program, but we also have an opportunity to take that same product and begin development for a second indication in limbal stem cell deficiency.
Maybe one of the things that, you know, we're the most excited about is the chance to even treat some of these, rare back-of-the-eye indications, in retinal diseases, and that's an early-stage program that we've been advancing over the last two years. What do we do? Well, we believe we're really at the kinda tip of the spear with mesenchymal stem cells. The beauty of those, to say it simply, is that we get cell therapy without having to implant the cells and all of the issues that comes along with that.
We're able to very efficiently extract the secretomes that have the right kinds of agents to be able to treat, in particular, wounds and have that regenerative therapy value and efficacy in the marketplace in a product that, as you'll see in a few minutes, is very simple to manufacture and very simple to use. We're able to create wound healing and tissue repair effects, as well as have an anti-inflammatory and immunomodulatory effect. What we're doing with our core asset right now is to be able to treat a broad range of persistent corneal epithelial defects.
So if you just take a look here, when I talk about pipeline in a product, not only do we have these first couple of indications that we've talked about, but really there could be application around a wide range of uses, including corneal ulcers or chemical injury burns. In the back of the eye, some of these categories I'm sure are very familiar. Retinitis pigmentosa or RP, as it's called, Stargardt's disease. There's another company that has had some recent Stargardt's data and is really innovating, and we believe that our product could have applications in those areas as well. So our first product is KPI-012. It is human bone marrow-derived mesenchymal stem cell therapy, and, as I talked about, for persistent corneal epithelial defect.
I think really the core takeaway here is that we have a product that has all of those wound-healing elements that are wrapped together in a single package so that we can treat, we believe, the wide range of things that causes this disorder and have that multifactor efficacy. Also, because it comes from human-derived cells, we have a product that we've seen an excellent safety and tolerability profile from. We've seen no issues whatsoever in the first clinical trial we ran, as well as all of our preclinical models. The FDA granted us Orphan Drug and Fast Track designation. So in PCED, if you think about what's happening, the patient has this epithelial defect on the surface of the eye, and for many patients, that does heal.
But if it continues and it doesn't heal with traditional therapies, you could be leading towards something that could cause thinning or scarring of that corneal tissue. It can lead to infection or perforation and even permanent vision loss. So by the time that patient has been treated with, let's just say some OTC products or other conventional therapies, and that is not healing, the ophthalmologist corneal specialist is getting very concerned. In fact, a lot of these patients will go from an optometrist that they might go to first, to an ophthalmologist, and then very quickly to a corneal specialist. I probably should say that our previous products that we developed were also corneal-focused products, and we have outstanding relationships with the corneal specialists in the area.
But if you don't heal that and maintain that healing, you could end up with a really significant problem for the patients, and most of the patients have more than one underlying etiology. It's estimated that there's about 100,000 patients in the US and maybe as many as triple that if you add in the US and the rest of the world combined. We had conducted a Phase Ib study in patients where we saw rapid efficacy. We'll show you those data in a few minutes, and this market only has one other product in it right now. Again, that product has been very successful because of the unmet needs, but there's still a significant amount of room out there for another product, and physicians need multiple products typically to be able to treat and heal these patients.
Just to talk a little bit about the market, there's no single therapy today that addresses all of the different etiologies. There is a product out there, Oxervate, that is for neurotrophic keratitis, and that's one of the etiologies. About a third of what causes persistent corneal epithelial defects is neurotrophic keratitis. We feel that we can treat that as well as the other 2/3 of those etiologies. There's still a need for rapid and sustained wound healing. It can take a bit of time with some of the products that are on the marketplace right now. And to show you just how big of an unmet need there is, what often happens to these patients is their eyelids will be sewn shut. So imagine how uncomfortable and intrusive that can be for a patient when that's what you have to resort to.
It's one of the reasons that Oxervate has done so well and one of the reasons we believe we have such a big opportunity in front of us. There's also an unmet need for something that's well-tolerated. Some of the more invasive therapies are not very well-tolerated and are quite intrusive. But even the current product that's on the market has between 16% to 20% of patients that have a meaningful side effect that often causes them to have to stop the therapy, and that's really problematic for the patient and the doctor, especially when you know continue to consider that these patients have this defect that has maybe been going on for several weeks now, and the doctor will cure it as quickly as they can. The market's worth well over $1 billion in the US alone.
That main product, Oxervate, is on its way to over $700 million. And you know, we believe that a product that could be used across all the different causes for PCED could have a substantial market penetration. Here's just the data that supports how big their product has become, and congratulations to them. They've done a fantastic job. They have a great product. They're building out a market and you know, we're thrilled to have a chance to develop what we believe could be an even better product in this marketplace. So, what do you want? Well, when you get these erosions, there's multiple things that you need to heal the patient and certainly you know, growth factors are one of them.
But you also would love to have protease inhibitors and matrix proteins, and essentially, what's involved with refining the secretome is that we are producing a product that has all of those natural healing elements, and then we're delivering it in a concentration where we get that rapid and complete healing of that erosion. We did a phase Ib study. We studied 8 patients. What was really exciting and compelling to us was all of the patients had meaningful improvement, and 6 of the 8 patients were completely healed, and even 4 of those 6 patients healed after the first week of therapy. The other thing that's... You can really get your heads around, a lot of diseases, you can't see the healing happening. It might be pain relief or some other kind of biomarker. Here, we use an objective photograph of the eye.
In our study, it's read by a third-party reading center, and so we're really able to minimize investigator variability in assessing that efficacy outcome. If you take a look here, you can see that we did treat patients across the various causes or etiologies of persistent corneal epithelial defect. You see that rapid improvement and the complete healing in the vast majority of patients. Even in a patient that, you know, have had the disorder for many months, we were able to show improvement. We also got rapid pain relief very quickly in the trial. The vast majority of these patients are feeling that pain. Remember, they've got an erosion on their eyes, so it's super sensitive.
Even blinking or, you know, other materials that are touching the eye or the eye is getting wind, you really can feel this, and it is extremely uncomfortable. So to see the pain relief happen very quickly was a great sign of efficacy for us. We are progressing toward having our readout by the end of this year. That has been enabled by us getting our IND approved very late last year, getting the beginning of our trial with a safety cohort going in the beginning of 2023, and then our enrollment really started about mid-year last year, and our enrollment curves have been ramping up, and we feel good about delivering that data.
If the study is positive, we do believe that it could be the first of two pivotal trials that are needed, so that's really meaningful value creation for us. The trial design is that what we've done is we have about 90 patients that we're enrolling, 2 different doses plus vehicle. It's 8 weeks of treatment, plus 2-week and 6-month follow-up evaluations. We have about 40 sites in the US We are opening sites as well in South America to be able to give us the best read across regions and hopefully increase enrollment. And our primary endpoint is, as I mentioned before, complete healing and read by a third-party reading center, which really reduces variability.
I mentioned limbal stem cell deficiency, but this is another, product opportunity that has very little treatment modalities for it right now. It is another orphan or rare disease, and our current product could be used to treat, this, disorder. This is one where you actually have, you know, some tissue that is growing over the side or could be the corner of the eye, and we believe that, our product could be used here, and this is something that we would have the ability to, start this year should we choose to do so. Our manufacturing process is, very straightforward. We take that cell bank and, use a bioreactor production process. We harvest the secretome, and we're able to turn that final product into, a blow-fill-seal individual use, product, form.
That makes it super simple for the patient. There's no reconstitution or other multi-step processes that are needed, and we think that could be a great advantage for us. We've worked with the FDA on what the CQAs will look like, and we'll continue to work with them, but we've been able to make material reliably for the clinical trial and as we continue to advance that process with CMC. And as I mentioned earlier, we also think it could be used for Retinitis Pigmentosa or other potential back-of-the-eye indications. We are working on a product form that could be used to treat those disorders, and that just unlocks an entire additional set of value-creating disease opportunities for us. We are fortunate that it is both a biologic with 12 years of exclusivity, as well as the orphan exclusivity for 7 years.
We have very strong patents around how we make the product and that final product presentation. And again, as I mentioned, we have the US Fast Track designation, and that IP protection goes well beyond 2040. So I think I just summarize that we're really excited about the opportunity we have in front of us. This market has been well developed over the last couple of years by Oxervate and the Dompé team. They've done a terrific job. We believe we can be another modality that's added into that treatment armamentarium for patients and doctors, and we're excited to finish our trial and get the data out this year. So I'll stop there and see if there's any questions. Sure.
So, you talked about like the phase II design and changing things. You want to talk about your rationale, how much of those design, safety, and efficacy question, that kind of thing, and then help us understand the expectations?
Sure. You're right about what you said, and our CMO, Kim, is right here, so I'll have him answer.
Sure. A great question. Excuse me. In the initial trial, we used the 1-unit dose and saw very good efficacy. So we went to the phase IIb, which as discussed with FDA, would be a pivotal trial. And they, of course, as always, want to see some dose ranging as well. We saw 75% of the patients heal in the initial trial. And so going to higher dose, we may have the opportunity to heal even more of those patients. As we talked about, we're looking at a broad range of etiologies. So we wanted to do the dose range, and we wanted to maximize efficacy, so that's why we chose to add the other dose level to the ongoing trial. And your second question, I was-
Actual months versus...
We, to a certain extent, based our design on what Oxervate did, and that's available through FDA Summary Basis of Approval. They saw in their US phase III trial, 67% improvement in the Oxervate arm, their high dose... 65% in the high arm, 17% in the low arm. So, we anticipate that, as sort of a potential or even higher. You know, we're working with Oxervate, which is a great product, has a single growth factor, nerve growth factor. By the secretome approach, we have numerous molecules, growth factors that are known to be involved in the wound healing process. And that's why we feel that we're able and will be able to show efficacy across multiple underlying etiologies.
We do have neurotrophic agents, so we expect to see healing in the Neurotrophic Keratitis as well. So, you know, what we're targeting at the end of the year is to have really data on the primary and key secondary endpoints on, you know, the number of patients that completely healed, the rate of healing. We're measuring pain. Some of these patients have significant pain, and even vision. If this defect is in the center of the cornea, in the visual axis, you, of course, will have diminished vision. So, those are the key endpoints that we will be targeting to read out at the end of this trial.
We definitely designed it to be a first pivotal if positive, so we don't expect to have to change very much. Perhaps we would just do, you know, one dose potentially in the next study, but, you know, it remains to be seen till we get the data.
Can I ask a question?
Yep, sure.
How are you thinking about the population that you're going?
No, we have... And we've discussed this with the agency. We have allowed virtually all etiologies of PCED. There's one or two, like, you know, significant burns, where you lose all your limbal stem cells or those sorts of things, that the only way to heal those is surgery. But we have a very broad list of etiologies. So other than those really severe, we're not limiting it to any particular etiology. And the FDA has indicated that you're not going to have to show statistical significance for each etiology, but if you enroll all comers and have a broad range, then that will be sufficient for us to potentially grant an indication for a broad treatment of these persistent defects.
Okay. Well, thank you very much. Hope you enjoy the conference.