Good morning everyone, and thank you for joining the 2026 H.C. Wainwright 28th Annual Global Investment Conference. My name is Dr. Jayne Montgomery, an Associate Research Analyst at H.C. Wainwright, and I am happy today to introduce our presenter for this session, David Moss, CEO of INmune Bio.
Thank you, Dr. Montgomery, and thank you for having me. My name is David Moss. I am the CEO and Founder of INmune Bio. INmune Bio is focused on two late-stage platforms around inflammation and immunology. One of the things that we really focus on in those two areas is the fact that most treatments that treat inflammation are immunosuppressive. The two drug platforms we have today are very potent in treating inflammation and modulating inflammation, and they do it without immunosuppression. That is a very, very big deal. Our two platforms are CORDStrom and XPro. Many of you know XPro and have heard about it. It was the first to treat neuroinflammation as a driver of Alzheimer's disease. In June of last year, it did not hit its endpoint of cognition in a phase II randomized study.
There is only one study I will point out that did that, and that was Eli Lilly's donanemab, which was a large, almost phase III study. It did show very clear signals that there is a drug. It met endpoints from an efficacy standpoint and an effect size on two cognitive endpoints, p-tau, GFAP, two neurodegenerative endpoints on white and gray matter imaging, particularly white matter imaging. It actually had a strong p-value to show that it remyelinates. It also hit things like Neuropsychiatric Inventory. Those are things like sleep disorders, aggression, depression, and so on. This is a program where we met with the FDA at the end of phase II. They granted us Fast Track, and they gave us a clear path for registration phase II-B/III program. As many as you know, that is a very expensive proposition.
That program sits on hold currently while we move CORDStrom forward. CORDStrom is in an ultra-rare disease called recessive dystrophic EB, which I will talk about. It is in the process of submitting regulatory applications for approval, first in the U.K., which is going Q4 this year, and we expect approval sometime in Q2 of next year in the U.K. It will submit its accelerated approval and conditional marketing authorization approval in the U.S. and in Europe in Q1 of next year. If the program gets accelerated approval in the United States, it comes with a priority review voucher. Guess what we will do with the priority review voucher, is we will use that to help fund the phase II-B program in Alzheimer's. CORDStrom is what we are going to talk about today.
The trade name of the product is called Ebstrocel, and it is the first treatment that treats the systemic nature and then endpoints that are important to the patients in recessive dystrophic EB. Recessive dystrophic EB is a genetic disease. These children are born without Collagen VII, and they have chronic skin conditions that you see visibly externally on their skin, but it also happens internally to every organ in their body except for their brain. What you see externally is inside their mouth. It happens on their eyes, as you can see in this top picture. It is in their nose, behind their eyelids, down their esophagus, through their gastrointestinal tract. And they have these chronic wounds. The process is from 0 days to 10 years of age. The wounds heal faster. There is less fibrosis.
As they get 10- 20 years of age, the wounds take longer to heal. They are more fibrotic. Ultimately, when they get to their second or third decade of life, some of these wounds just do not heal, and they end up developing squamous cell carcinoma, and unfortunately, they pass. Most of these children do not make it past their third decade of life. What is interesting is that the treatments that are approved, there are three, are all topical in nature, and that is understood because most of these patients are treated by dermatologists. Dermatologists, when they look at these diseases, they see it as a topical disease. But if you look at the top complaints, and there were two studies that were done, this is one by DEBRA, and then there was a patient-focused drug development meeting. The patients do not complain about wounds.
They complain about itch or pain as their top two, and then many are gastrointestinal or psychiatric, mental health issues, not being able to sleep, not being able to take a bath, not being able to go to school, things like that. Wounds usually show up at number six or number seven. What happens in the disease is you get this inflammation- itch cycle. You could actually probably swap one or two back or forth, itch first, then inflammation or inflammation, then itch. They develop this scratching. If it is on their bare skin, they have very weak skin. It is called butterfly skin. It is very susceptible to peeling. They will create wounds. They will introduce bacteria. If they have existing wounds, they will create blisters and prevent wounds for healing. And this helps with prolonging the incidence of the disease.
And this, you get more inflammation and on and on and on you go. What Ebstrocel does is it reduces the itch and inflammation, and it introduces growth factors that change the immunology of the patient's macrophages at the wound, and it also introduces growth factors to improve wound healing. So it addresses the top complaints of these patients, which are itch and pain, and it reduces inflammation, and it helps with wound healing. Now, this is really that chart in a long bar format. If you reduce itch and scratching, you improve wounds over time, and you should reduce the incidence of fibrosis and ultimately squamous cell carcinoma. Now, no one is ever going to do this study because it takes so long, and I use the word should reduce.
But if you look at the quote at the very bottom here, this is from our lead PI, and she's one of the leading people who treats RDEB in the U.K. And her quote was, "Treatment with Ebstrocel in RDEB patients is likely to be disease-modifying, improving itch, skin healing, quality of life, and reducing the risk of squamous cell carcinoma later in life." Should reduce the risk later in life. This is not something we will test, but the clinicians know that by reducing the top important endpoints of itch and pain will make a difference in these patients. So this was the study that we ran. It was the largest blinded randomized trial ever run in recessive dystrophic EB. It had two groups.
It had a treated group that was then washed out and given placebo, and then it had group two, which had placebo and then was washed out and given drug. It's a double-blind crossover study. What's very interesting is that the patients in group one who received placebo had an ongoing effect of drug treatment and fared much better than the patients who got placebo in group two, who worsened and worsened and worsened until they got drug. So comparing placebo- to- placebo and drug treatment- to- drug treatment between groups one and group two is quite fascinating. This is the data. Early on in month three, there was a significant reduction in itch. We did not see improvements in wounds at this stage. That itch was maintained at six months and slightly improved, and then we started to see improvements in skin integrity. That's the iscorEB.
So what happens if you think about it is if you're not itching, it takes a while for you to pick that up in terms of your skin integrity. One of the most potent and powerful developments here was the patient-reported outcomes. As many as you know, in ultra-rare and rare diseases, patient-reported outcomes can be significant drivers in regulatory decisions. It's too small to read, but the patients, if you read some of it will tell you they had significant improvements in the ability to take a bath, the quality of the wounds, less pain, less itch, being able to go to school, do activities, and so on to that sort. There was a child that happened to do a report, an article at the BBC.
If you type in BBC and RDEB, it'll pop up, and he talks about these experiences of being able to go to the beach, kick a soccer ball, et c. So tremendous patient-reported outcomes. When we met with the MHRA, we filed a pre-marketing authorization application. They asked us to lead with the patient-reported outcomes first as part of our marketing authorization application. So this is itch. This is how it's measured. It's called the Itch Man Scale. This is a validated scale of itch. It has five different levels. It goes from 0- 4. All of us in this room are sitting at a 0. If you have several mosquito bites or a very bad case of psoriasis, you might be at a 1. All of our patients in the trial were either at a 2 or a 3.
2 is where the itch is so bad that it's hard to concentrate, hard to sit still. Three is where it's very difficult to even sleep, to concentrate, to be still, et cetera. One of the children described it as having RDEB as being bitten by a mosquito a thousand times a day. The itch is just a tremendous driver of the psychological impact of these children. Patients who took placebo had an increase in itch. They went from level 2 to level 3, and patients who took Ebstrocel had almost a 70% reduction, significant reduction moving from level 3 to level 2. What this resulted in is the green and the red bars are Ebstrocel at three and six months, and the black and the gray are your placebo.
What this resulted in was less inflammation in the joints, better feeling and able to use their hands, less pain in their joints, their eyes, their mouth. That results in better eating, doing tasks that all of us take for granted every day, like going to school, doing leisure activities, sleeping, just general activities. Significant improvement in the children's wellbeing. This was a single trial. It was the largest randomized trial that's ever been done in RDEB. If you look at the conditional framework in the U.K. and Europe, and it's very similar for the accelerated pathways in the United States, if you have a drug that you can show clear benefit risk profile, which clearly we talked about here, the drug's been given more than 200 administrations with no SAEs whatsoever.
If you have proof of concept and partial data, which is what we have in the trial, we have significant reductions in pain and itch and improvements in skin integrity and very strong patient-reported outcomes. It's an unmet need. There is nothing that treats the systemic nature of this disease, and there's nothing that treats itch or pain. Then you have substantial evidence expected. In other words, you have a confirmation, a phase III trial ongoing, which will open early next year. You have a really good framework and understanding for conditional approval. Most of our interactions have been with the MHRA. As I mentioned earlier, we submitted a pre-MAA package. We've also submitted the package for the open label phase III program. This trial that was run was aged six months to 16 years of age.
When we submitted the open label trial, they asked us to go from 0 days of age to 18 years of age, because they know that the program was safe. I'm not going to go through this in too much detail, but we know exactly how CORDStrom works. We can match the mechanism of action with how we reduce itch and pain and how we improve wound healing, and we can match it to the patient samples in the trial. Not only can we match the MOA in vivo to the patients, we also can match it in vitro in assays. When you look at approval, the regulatory agencies want to know, obviously, is the drug safe? Second thing they want to know is, does it meet the endpoints that are important to the disease?
Third thing they want to know is, can you manufacture the drug repeatably, reliably, and batch to batch. The last thing they want to know, ultimately, is can you match the MOA with the endpoints that are important to the disease? And that we can. I am not going to go through this in a lot of detail, but Ebstrocel on the far right, it releases things like IL-11, PGE2, IDO, and HGF hepatocyte growth factors. What happens is you take macrophages from an M1 to an M2 state, and when you do that, IL-13 is released. IL-11 and IL-13 change TH2 and TH1 cells and reduce IL-31, which is responsible for itch and pain. If you look in the green boxes here, these are the patient samples. By doing all of this, you see TNF, which is an inflammatory cytokine.
Patients who took placebo had an increase in inflammation. Patients who took Ebstrocel had a reduction in TNF. If you look at the middle box, that is IL-13. You want to see IL-13 released, which shows that you change macrophages from an M1 to an M2 state. Again, same thing here. You get a reduction in IL-13 in placebo and an increase in IL-13 with treatment. HGF, which is growth factors responsible to help with wound healing, same thing here. Reduction in HGF from placebo and an increase in HGF with treatment. This is what CORDStrom is. It is the most advanced mesenchymal stromal cell program that has ever been developed. It was developed as a drug from day zero. What we do is we screen donor cords for certain cytokine production, and we take the ones that we want to treat the disease that we are targeting.
We use very specific identity markers. We are able to combine and expand it, and this combination and identity process means that when we expand it, the batch that we make today is the same batch that we make tomorrow, five years, or 10 years from now. So you solve the heterogeneity problem and make a homogeneous product. Part of the problems with MSCs in the past has been heterogeneity. Where are they sourcing them from? They are not looking at the markers. When they go to expand, they get a different product every time. That has been solved with CORDStrom. It actually ends up being a platform because now you have a cell that you can tailor to different diseases, and you can also genetically modify it. A next generation version of CORDStrom will likely include Collagen VII to deliver Collagen VII systemically.
It is a closed loop manufacturing system that has been validated. Everything is done through bioreactors, which you can scale up. We are currently at a scale where we can produce more than we can treat patients in the U.K. All we need to do is just scale up our current bioreactors, but all of the fit, fill, finish, and storage is all closed. These are the milestones. Everything has been done up to the MAA submission to the EMA. Everything is very clear with the MHRA because we have already had a pre-MAA meeting which really identified all of the clinical and CMC issues, which are all addressable. So we feel very comfortable about conditional marketing authorization in the U.K. The BLA to the FDA and the MAA to the EMA will happen in Q1 of next year.
The follow-on trial will start in Q1 of next year. This is the competitive situation. There's three products approved. The real leader is VYJUVEK, Krystal. It is a topical gene therapy. It's a very effective product. Its only limitations are that it doesn't address itch pain. In fact, on the label, it's an increase in itch, which is part of wound healing. Obviously, you cannot use it systemically. It cannot be used for the wounds inside the mouth, the eyes, et c. The other limitations are that they're limited. You can only use about 2.5 credit card sizes of material on a wound before you move on to the next wound. As you saw in the pictures, these kids have wounds all over their arms, their legs, their back, their chest.
So it will take a while to be able to use it. ZEVASKYN is a per wound treatment. It's expensive. It does work well. If you have late-stage wounds that are not healing, it's a great product. FILSUVEZ is really a birch bark oil. It's not well-used. Most patients stop using it after a period of time. All three of these are topical in nature. I'll end with some quotes from some of the patients that were on our trial, but it's emotional. This is what gives us dedication to get this product to these patients. I can tell you that every single patient that was on our trial wants to stay on the product. They're going to enroll in the open label, and we have a lot that are waiting. With that, I'll end.
All right. Thank you so much, David. We have time for one question if anyone has anything pressing that they'd like to ask. No?