Welcome to L.A., everybody. Thanks to LD Micro. We've been working with them for many years, good friends here, and appreciate the invitation to talk about Arcturus to some new faces and to some familiar ones. The messenger RNA world has been through kind of a whirlwind over the last decade, even though it's been around for 30 years. Everyone in this room has been impacted by mRNA vaccines with the COVID pandemic. Arcturus is leading the next generation of mRNA medicines, though. As we walk through this presentation, I think it's important to understand how we're differentiated and where we are leading, and we'll kind of go through this. We do have an approved COVID vaccine.
While our competitors were vaccinating Europe and U.S., we were busy vaccinating Singapore and Japan and Vietnam. We do have an approved COVID vaccine in 32 countries, and it's called KOSTAIVE. We have a relationship with BARDA for pandemic flu, and we utilize our differentiating technologies in those programs. We're based in San Diego, for those that's just a couple hours south of here. If you're ever in the area, come by and say hi. We have approximately 100 employees and been around for about 12 years. What sets us apart from the field is We're one of the leaders or leaders in injectable or intravenously dosed mRNA, and especially for inhaled messenger RNA for cystic fibrosis.
Inhaling mRNA is an extremely difficult delivery challenge that we've worked through over the past decade. Hundreds of millions of dollars we've invested into this, and we'll give you an update today. I mentioned that we have differentiating technologies and know-how, especially with respect to delivery. Our delivery technology is different. For those that are familiar with RNA medicines and very successful RNA companies, the key to success is safe and effective delivery of those RNA molecules. The messenger RNA molecule is perfect. We just need to get it to where it needs to be safely and effectively, and we have LUNAR. That's a lipid nanoparticle delivery technology that's different. It's chemically different in multiple ways, and those differences are showcased in preclinical and now clinical data.
With respect to mRNA technology, at the top of this slide, on the vaccine portion of our business, we use self-amplifying mRNA, which is again a next-generation technology. It's about 30 times more efficient at expressing protein, it's a significant improvement over conventional mRNA. We also have significant know-how on how to make the mRNA. If you're new to the field and you wanna understand where to invest or where to what to closely track in the messenger RNA space, it's very important that you look at how their experience with manufacturing these types of therapeutics. mRNA medicines are not like our grandparents' aspirin. It's very sophisticated. You have a beautiful, large, highly negatively charged mRNA molecule that needs to be encapsulated in this, you know, sophisticated nanoparticle technology.
To manufacture that on scale, there's very few companies that know how to do that. We are fortunately one of them. Because of the pandemic, we got extraordinary funding to scale up our platform and technology capabilities. That's a point of differentiation as well. We can make these mRNA lipid nanoparticles on scale and that's a point of emphasis. It's an mRNA delivery and manufacturing know-how. We apply these to our flagship programs are on this summary slide here. We have an inhaled mRNA therapeutic for cystic fibrosis. This is a very exciting technology for us and exciting program because the lung is a valuable organ. If you're in the pharmaceutical industry, you know how valuable the lung is. People like to breathe.
We're the first, potentially the first, and we're leading with inhaled mRNA. If we're successful with CF, then that opens up an enormous white space commercial opportunity for us in the field of mRNA therapeutics. We're excited about that, and we have a phase II study that's ongoing right now. It's an open label study, we're going to be learning a lot if about our program throughout this year. The second program is the ornithine transcarbamylase deficiency program. This is a rare liver disease, I mentioned that we have the ability to deliver RNA to the liver, which is also a challenge. Unlike the vaccines, right? When you just inject the vaccine right into your arm, there's no delivery challenge there. You just stab the arm and push a button, right?
With respect to intravenous delivery of RNA to the liver, that's a more substantial challenge. You have to survive the bloodstream, you have to break through the holes of the liver called the fenestrae, you have to access the right cells, which are called hepatocytes, you have to get inside these cells and break out of the endosome. It's an extraordinary amount of science there, and we're one of the leaders there. This asset, this ornithine transcarbamylase deficiency asset is in the middle just finishing up our phase II study, and we have an EOP2 means end of phase II meeting in the second half of 2026, that's a near milestone for us as well.
Again, it represents a flagship opportunity for the platform. If this, so far it's safe and effective and working very well, and if that continues, then we have a lot of opportunity behind it. Now I'm going to summarize CF, where we are. Again, this is an inhaled, our, you know, messenger RNA therapeutic, and when the mRNA enters the lungs, it makes or expresses a brand-new CFTR protein. The most serious types of cystic fibrosis are people that are called Class I CF, and they're missing the protein entirely. There's nothing there to fix. There's other competitors in this space that are very successful with what are called CFTR modulators. That means they're fixing a broken transporter. That is not what we do. At Arcturus, we're in the business of making a brand-new one. These people have no drugs.
There's no, there's no standard of care that with therapeutics at all. We are, we're in phase II. There's a lot of attention on this program from the CF community and especially the Class I CF community, and we've established safety and tolerability in our studies at 5 mg , 10 mg , 15 mg . These are generous doses of mRNA. No one has previously achieved these levels of doses. One of the questions we get from investors all the time is, "How in the world are you getting those doses so high?" All previous attempts were dramatically lower doses, and it's because of those technologies that I reviewed previously. In order to dose mRNA high, you need to have a benign, relatively safe and biodegradable delivery technology that's chemically different from the older delivery technologies, and we've done that.
The impurities in the RNA. It's usually not the most exciting biotech story to talk about purification in mRNA, but it's a big deal in our space. The impurities in mRNA can be immunogenic. They cause an undesired immune responses and inflammation, inflammatory response. It's not good. We've optimized that over the past decade. Because we have a more pure mRNA, a completely different delivery technology, you combine that together, and you have, we've seen that it's safe and well-tolerated even up to 15 mg daily. I wanna emphasize some differences here too. It's not only administered daily, but we do so without any steroid treatment before, during, or after. Our competitors over the last couple decades always had to administer steroids before people inhale it to prevent these undesired inflammatory responses.
Before, during, and after, they've always had to do it. The FDA mandated it. With us, the FDA hasn't. Our safety profile is, knock on wood, wherever it is, it's very clean. We have to hope that that maintains, and we feel very good that this is a differentiating element of our technology. A different delivery technology, a different purification process to remove those impurities, and that allows us to dose it highly without steroids daily. Right now we're in phase II, and we're presently in a trial that we're enrolling up to 20 people. Instead of 28 days, we're gonna be extending the duration of treatment for 12 weeks. The CF Foundation is in support of this program. They've been a fantastic partner. They understand where every single Class I CF subject is in the U.S.
Europe. What a powerful partner that is. If this is successful, we have a very streamlined path to commercialization, with very limited budgets. They're a great partner. We look forward to working with them more so in the future. We have all the Rare Pediatric Disease Designations, the Orphan Drug Designations from the U.S. and Europe that allows us some sort of competitive patent-related exclusive benefits. The slide deck that I'm doing is available on our website, so I won't be going through every detail here. If our therapeutic works in Class I CF, then it's applicable to everybody. Imagine if you're someone with a broken CFTR, why not, instead of fixing it up, why not get a brand-new one with an mRNA treatment?
If it works, if our therapeutic works in Class I CF, then there's no reason we cannot franchise this into the entire CF community because I think everyone will want a brand-new CFTR protein in their lungs rather than a fixed-up one or a modulated one. We'll see. That's the overall opportunity is quite enormous. Presently, I think it's helpful for people to understand, if you have Class I CF, what do they do now? Well, you put on a vest. I don't know if you guys are familiar, if you've seen these vests, but they strap on a vest that shakes. It does this. They have to put it on, whether it's 30 minutes, an hour, two hours, and it just like it shakes them.
They're inhaling saline, saltwater solution, just trying to loosen things up because their lungs are so congested. It's a nested disease. It just builds and builds and builds until the subject can no longer breathe, and they pass. It's a horrible disease. Right now, all they've got is shaking and breathing in saltwater. We're, you know, we're hoping that we can be the first to really dramatically change the field for Class I subjects. You can see there's about 10,000 Class I CF patients globally. I mentioned that there's no really standard of care for them and the other highlights there. They're ineligible for CFTR modulators, which are very successful drugs. But they're not successful for Class I because they do not have the protein.
There's nothing there to fix. They're ineligible for those. You said that rapid adoption is expected too. I've talked with everyone. If this works, this will be taken up very quickly. Phase I was very successful. Phase I-B, we transitioned to people with CF, and we successfully dosed up, and we were doing some dose range finding studies. We got closer to locking in the dose. If you go right down to phase II, as we went through five milligrams and 10 mg and 15 mg, it was safe and well-tolerated. Now we've locked in the dose at 10 mg, and we're extending it to 12 weeks, and that's where we are. Here's some data I want to just stress.
Mucus plug, these CF patients, you know, the accumulation of mucus is how they die, right? They drown in their own mucus. If you can remove these mucus plugs, this is very important to the patient, their doctors, their families, and I wanna just show you a picture of what it looks like. These are the lungs. Let me just go to a picture of before and after. Let's see if I can use my laser. If you see on the left here, this is an 18-year-old female, and she has a life expectancy of about five years from today. She's very motivated, right? Already, she's getting accumulation of these, what's represented in red is the mucus plugs.
After 28 days of treatment, you can see a very clear resolution of those mucus plugs. This is very exciting data for these subjects. Now, even in more advanced patients, you can see this person is obviously more advanced, and they even have very large mucus plugs that are very difficult to correct. After 28 days, you can see that we're making headway there. This is a nested disease, and that's why it needs to be inhaled every day, and we're just peeling away this nest one inhalation at a time until hopefully, as we extend this treatment, we'll see this continue. If so, we're sitting on an extraordinary technology and extraordinary drug. Mucus plugs have been being corrected or being reduced. It's generally safe and well-tolerated.
We have meaningful trends of clinical activity that we're showing on these high-resolution CT scans that are showing some great progress. Why isn't our stock trading at billions right now? Why hasn't Wall Street responded to this extraordinary data that's never been generated in the field? Because you can't get a drug approved with pictures. You need to show lung function. The purpose of the study that we're doing right now is to evaluate two types of lung function, including FEV and LCI. FEV is when there's a straw, and you blow into a box. That's kind of like an 1800s technology. It's very old, very familiar, validated. LCI is lung clearance index. It's more sophisticated.
This is a study where you fill the lungs with 100% oxygen, and maybe some of you don't realize, but the air we're breathing right now is 20% oxygen, in case you need a reminder. If you fill your lungs with 100% oxygen and then you time how many cycles does it take to go to 20% oxygen with normal air, that lung clearance index, the number of cycles it takes to establish, you know, equilibrate your lungs, is a very sophisticated test. It's very sensitive. The good news is that it correlates nicely with mucus plug reduction.
This is the first time we're evaluating lung clearance index in this present study in addition to FEV, and we hope to get some good news as we continuously collect some data in phase II this year in this open label study. Again, there will be about 20 subjects enrolled, and they're inhaling 10 mg daily, not for 28 days like the pictures you just saw, but for 12 weeks. We extend the duration of that. Now on to OTC. Ornithine transcarbamylase deficiency is a rare liver disease. This is an ammonia-related disorder. For those that need a refresher on ammonia, that's Windex, right? If you don't clear After you eat a burger, I don't know if anybody had any protein yesterday, but those proteins are converted into ammonia, and you get rid of that.
The urea cycle in the liver converts that ammonia to urea. That's where you get urine, and you urinate it out. That's how we get rid of it. If your urea cycle is messed up, if you do not have ornithine transcarbamylase in there, that means ammonia levels rise, and they cross the blood-brain barrier, and that's very bad. We're in the business of, again, just like in the CF space, in this case, you have a messed up or missing protein in the liver. We go in, and we replace what's missing or dysfunctional and restore urea cycle activity. There's 10,000 subjects with this disease in U.S. and Europe. The present standard of care is ammonia scavengers. They're very expensive.
They have to pop these pills and drink a lot of water and urinate 10 times today to get this managed. It's very difficult to control the spikes of ammonia. Imagine if you're a parent and you have a child that eats the wrong sandwich at school. They need to be rushed to the hospital. You need to induce a coma, clean out the ammonia, and pray that when they wake up, they don't have neurological deficits. It's an extremely stressful disease, and rather than maintaining it with ammonia scavengers, why don't we just replace what's missing and fix or functionally cure the disease? That's what we're doing. In phase I, it was very safe. We dosed it up to 0.4 mg/kg there in 24 subjects.
phase I-B, we continued dosing up further to 0.5 mg/ kg in actual subjects, patients. We went and did two phase II studies in Europe and U.S., we got a lot of initial data that started us to get excited about this program. The phase II ongoing open label study is happening right now. We just have a very small number that we're completing. We talked with the FDA. We shared the data with them in a Type C meeting. This is very new. We shared that on our recent earnings call, they gave us very clear direction what we need to proceed to advance this further, not only with adults, but with the kids. The pediatric element of this population is the largest commercial opportunity in our view.
It was really good to hear from the FDA what we need to do. We have that end of phase II meeting at the end of this, or and we said the second half of this year, and we look forward to getting that done as soon as we can. This also has Orphan Drug Designation status in U.S. and Europe. This ongoing U.S. phase II study is evaluating at the bottom, you'll see 0.3 mg/kg and 0.5 mg/ kg. We're doing a study of five bi-weeks. Every couple of weeks we do an IV infusion, and we're looking at these biomarkers. There's a lot of biomarkers associated with the urea cycle. You can look at ammonia, you can look at glutamine, which is a wonderful biomarker.
If the ammonia gets converted to glutamine, it's also a problem because glutamine crosses the blood-brain barrier and then converts back to ammonia. Everyone wants to see glutamine and ammonia reduce. Here's some interim data. Glutamine's a great biomarker to track because even if you're on ammonia scavengers, you have problems with glutamine. You can see that, you know, before treatment on the left, you know, the dots are high. This is glutamine levels are very high. As you go through the treatment process, they lower into the normal area, which is fantastic news for these people. As you discontinue treatment, it goes back up. We see this consistently. We shared this data. We have additional data that we'll be sharing later this year.
We can track ureagenesis. There's wonderful new assays out there to look at the urea cycle itself, and we're starting to gain some traction there and understanding. We saw ammonia is stable and within the normal range as well. In summary, it's safe, it's well-tolerated. We're normalizing multiple biomarkers. We've got cool new, sophisticated ways to track the urea cycle and how it's working. We have this end of phase II meeting in the second half of this year. Vaccines, I'll just summarize quickly because I know there's a lot of fatigue in the COVID space. Self-amplifying mRNA generated the KOSTAIVE story, which went through discovery all the way to approvals at the end of this era with Japan and throughout Europe and the United Kingdom.
It has shown that our platform has been shown time and time again in multiple phase III studies to be superior to conventional mRNA, and not just by a little bit, by significant amounts. We have shown this over and over again, published it in premier journals like The Lancet. This is well established now. I just want to emphasize that we already have gone through this process and validated our platform. You can see this these phase III responses just to summarize, like this is conventional mRNA is lower, and then self-amplifying mRNA or the Arcturus platform is much higher, much longer lasting, much more antigens are being produced, a more durable immune response. We also have LUNAR-H5N1 is the bird flu.
Just to touch on that, the U.S. government is funding a whole bunch of programs. We believe we have the best vaccine in the world for pandemic flu. Let's hope we never talk about this again. If pandemic flu becomes an issue, it'll be about 20 times more deadly than COVID. Instead of 3% of the population dying, it'll be more like 50%-60%. If this ever becomes an issue, we have a very strong relationship with the government and they'll very likely be using our technology. This is our board of directors. In fact, Operation Warp Speed Chairman Moncef Slaoui, that might be a familiar name to some of you. He reported to Biden and Trump and helped us through and before, during, and after.
He was very excited to join our team as chairman because we're leading this next generation of mRNA. We've got the ex-president of Merck Vaccines, Jacques Cholat. We've got a whole bunch of fantastic board members that are supporting us in this effort. I'll pause there. Anyone have any questions? Go ahead.
What, I know I'm an old man, but what happened to phase III clinical trials? I mean, there's no mention of any phase III.
Oh, well, phase II. Yes. Well, because we're in the rare disease space, once you show safety, tolerability, and efficacy in a rare disease with no competition, and both our rare disease programs are like that, no to little competition. You have a conversation at the EOP2 meeting, they're gonna tell you what you need to do for the phase III, but it's very unlikely that it's an extraordinary trial. But we'll still have to do a phase III, the likelihood of success of that is very high, statistically speaking, if you show safety and tolerability and efficacy in a phase II study. Yeah.
Well, thank you so much. Any other questions can be taken outside.
Yeah, I'll take them outside. Thank you.