Okay. Good morning, everyone, and welcome to our next session. I am Sara Nik from H.C. Wainwright's Healthcare Research Team. It is my pleasure to introduce Arcturus Therapeutics, an mRNA medicines company developing therapeutics for rare diseases. Presenting today on behalf of the company is President and CEO, Joseph Payne. Please welcome me and Jo-
Yeah, thanks, Sara.
-Yeah. Go ahead, the floor is yours.
Rather than a fireside presentation today, we decided to go through a more formal slide deck. This is a corporate deck that is available on our website, but as we go through it, I will provide some context, maybe some information that is not necessarily detailed on the slides, and then we will have a Q&A session afterwards, and we can follow up. Arcturus is a messenger RNA medicines company. It differentiates itself from the other mRNA companies with our next-generation technologies.
We have a different delivery technology and capabilities in purifying mRNA and manufacturing a customized nebulizer for the inhaled treatments. As we go through, we hope that you see that we are a next generation mRNA medicines company with an extraordinary near-term and long-term opportunity for investors to pay attention to. We are based in San Diego. We have about 100 employees, so we are right on Science Center Drive there.
If you're ever in San Diego and want to stop by, it's a well-known area for biotechnology. San Diego has about 450 companies, and 100 of them have walls and mortar, and that includes us, and about 40 to 50 of those are publicly traded, and we sit in around the 15th in terms of a rank order with publicly traded biotech companies in San Diego. On the left, you see that we have an approved product, and we've recently regained control, strategic control, 100% control, of the KOSTAIVE asset. This is a COVID vaccine that's approved in 32 countries, including Japan, the U.K., and the European Union. We've also partnered with BARDA and will be able to speak. That data's going to be published. BARDA went out and looked at the bird flu aggressively, and they explored different technologies and different vaccines, including ours.
We'll have the opportunity to publish on that relatively soon, and we have a good relationship with them. On the right side, you see that we have a therapeutic franchise for ornithine transcarbamylase deficiency and CF. These are two rare disease assets that lead two platforms in the therapeutics arm of our company. Whether you're inhaling messenger RNA for cystic fibrosis as a flagship asset there, or injecting messenger RNA intravenously, our flagship program is ornithine transcarbamylase deficiency. We're partnered with the Cystic Fibrosis Foundation for our CF program, and we also engaged, signed a deal with Thermo Fisher. We've strengthened our manufacturing relationship in exchange for commercial manufacturing rights. They're helping us and supporting our CF program, and it could be significant if we make the decision to proceed further there into phase III. We have proprietary technologies.
Just want to emphasize that what differentiates us in the vaccine side is self-amplifying mRNA, and what differentiates us on the therapeutics arm of the company is our delivery technology. That is what is uniquely different. It's chemically different, it's biodegradable, it's non-accumulating, and these differentiations are helping to set us apart from a data and a commercial strategy going forward. Manufacturing know-how is a big deal. There's very few companies that have scaled, especially on commercial products, on mRNA. We're one of them, and if you go to self-amplifying mRNA, we are the only one, even though we're a relatively small company. We have a proprietary process that we use in GMP facilities to make self-amplifying mRNA products on scale, and that's very differentiating. Self-amplifying mRNA is large.
It's difficult to make, purify, formulate, and ship, and the logistics of shipping, but we've managed that successfully, and we're the first to do so with that process. I just want to emphasize the differentiation know-how there. A simple therapeutics pipeline. I know that we have a product in vaccines that provides stability to the organization. It provides a source of non-dilutive monies, which is great as a CEO, but the value, the true value being created at Arcturus is in the therapeutics branch, and we have a respiratory product and a hepatic or liver product. You can see that even though there's more than 100,000 global prevalence of CF, our initial focus is on Class I cystic fibrosis. About 10%-15% of the population doesn't respond effectively to modulators or other standards of care.
We're first movers in this field of Class I CF, which is an exciting opportunity for us commercially. On the ornithine transcarbamylase side, happy to report that we have a phase II data readout that we're going to be sharing later this month, so it's a very near-term milestone for us. It's not just a phase II data readout, but we've had a pair of Type C meetings for our OTC deficiency program this year. We've already indicated that it's been positive and productive, those meetings. But additional details, granularity on the regulatory path forward, we'll be able to share that later this month. In addition to a data readout and regulatory clarity, we're also providing a platform update. It's the cake, the icing, the cherry. It's a fulsome update, and we encourage investors to participate and pay attention to that communication later this month.
That's near term. Ornithine transcarbamylase is greater than 10,000 people, if you can Google that. Says the prevalence, and that prevalence is U.S. and Europe. So, there's potentially thousands of early adopters for this product if we're successful. This could be an extraordinary product for us. We're excited about its potential commercially. Beginning with our CF product, we have a key decision for the CF product in Q4. The reason we're guiding the go/no-go decision to proceed into phase III, that decision, if positive, if we proceed into phase III, that triggers a significant increase in contributions from the Thermo Fisher deal that we signed in July. So that's why we're providing guidance towards the decision. It's an open label study that's presently ongoing for our CF program.
You can see that as we provide a background, this is an inhaled messenger RNA therapeutic that makes or produces CFTR protein in the lungs. So we're not in the same business as other competitors in the CF space. Most CF companies are in the business of modulating a broken transporter. That is not what we do. We make a brand new one. So we're expressing a new CFTR protein in the lung that's very different from the field. It's been generally safe and well-tolerated in all studies, in phase I, phase I-B and phase II, even up to 28 daily days at 15 mg daily. So that's a significant accomplishment for inhaled RNA therapeutics. If you have been tracking this field, human beings do not like to inhale lipids and RNA. There's been extreme challenges in doing this with safety and tolerability over decades.
It doesn't matter what type of RNA, whether small, medium or large RNAs for different applications. There's been a lot of challenges for the field. There's been a lot of efforts because the lung is considered a trillion dollar organ. It is a very important part of our bodies, and so you can understand why these copious amounts of companies have been trying to accomplish this. But what is significant is that going up to the second bullet point is we've established safety and tolerability at 15 mg daily, and we did it without steroids before, during or after. We administered this drug in the home, self-administration, not in a clinic under the oversight of nurses and doctors. The FDA is allowing us to do this, so this is a very significantly differentiated platform.
And why am I emphasizing safety and tolerability is because Class I CF, guys, is a tough disease. It's a nested, messy disease in the lung, and you have to hit it daily. That's our view. You have to hit it daily, every day, inhale it, and chip away at this disease and resolve those mucus plugs, resolve the undesired inflammation, swelling, cirrhotic disease, right? Just heal the lung to allow other treatments or other things to work better. But we believe that this is the ideal approach for Class I CF. The Cystic Fibrosis Foundation has already committed $25 million. The reason we're emphasizing our relationship with the Cystic Fibrosis Foundation is because they've been very helpful in enrollment, in trials, in sites, and also, not just the cash, but the support of the foundation has been very important and meaningful to us.
And they know where all the Class I subject patients are. They have a nice database in the U.S. and Europe especially, so it's very helpful. We've received all the Rare Pediatric Disease Designations in Europe and U.S. That's also a good indication of potential regulatory progress. I mentioned earlier, but Thermo Fisher, if we make the decision to proceed into phase III, the financial contributions associated with that contract increase significantly, up to $40 million. I've already mentioned that the CF is a significant market opportunity, and if we can simply make new CFTR in their lungs, this would be functionally curing that local area of the lungs. It's not just modulating anything. It's functionally curing the local lung environment. So this could be a very meaningful drug, not only for Class I CF. If it works, it'll be applicable to everybody, potentially, in the field.
It's very franchisable, a very attractive commercial opportunity. In Class I CF, we mentioned there's approximately 10,000 or more Class I CF subjects globally. So there'd be thousands of early adopters to this technology if it became available. The summary, we've been in phase I, successfully phase I-B. We've already summarized at phase II on a previous slide. I want to focus on the bottom point of this slide, that we're presently in a 12-week open label study, enrolling up to 20 people in Class I, including in Israel and Turkey. Israel and Turkey have high prevalence of Class I CF. It's like 30%-40%. So there's access to a considerable number of Class I subjects.
We feel very confident on the cadence of enrollment to support this trial, and that's why we've been indicating that we should have sufficient data in Q4 of this year to make a decision. It's an open label study, so of course, the decision will come before the data is actually communicated publicly, whether it's a day away. We're collecting a lot of data in this trial. There's two lung function measurements we're collecting for FEV and LCI, and also we're collecting multiple quality of life measures. These are validated surveys that the FDA appreciates and takes into serious consideration when they're approving a drug. Also we're taking beautiful pictures, high-res CT scans before and after treatment. So it's a considerable amount of data that's being collected, and we'll be able to compare this data to a normative study that's being collected by the Cystic Fibrosis Foundation.
The CF Foundation is doing a natural history-like study in hundreds of Class I subjects. They are collecting LCI and FEV functional data in just the natural course of life for these Class I subjects. We will be able to compare our study and our data to the normative study, and that is another advantage. The next update for the normative REACH study by the CF Foundation is in October in Atlanta at the annual conference there. In terms of some interim data, we have already shared, after two cohorts, we shared some data, and then we did Cohort 3, and now we are presently in Cohort 4. Just to refresh what we saw in the second cohort, we saw mucus plug reduction. These pictures, before and after on the left, you see in the lower register, mucus plugs getting resolved just after 28 days.
In more advanced patients, see where someone has considerable mucus plugs, even in the lower register again, larger mucus plugs are getting resolved just after 28 days. We are presently in a much longer study, so we are very interested to see the before and after pictures of these mucus plugs being resolved. No one has ever shown this data before. It is very encouraging, especially to the subjects. Imagine a subject, their family seeing their mucus plug shrinking. A picture is very powerful. But for regulatory agencies, they need to see this translate into lung function improvements. Our desire, our plan here is with this present fourth cohort, this 12-week study, that we will be able to see continued improvement in mucus plug reduction, which will translate. The desire here is that translates into lung function improvements.
There are two different lung function improvements, FEV, which is a very old technology. It is from the 1800s. You blow through a straw into a box, and they determine lung volumes there. Then there is a more sophisticated passive maneuver called lung clearance index, where you fill the lungs with a gas that can be then easily detected over time as you just breathe normally. It determines how many cycles of breathing in and out it takes for you to equilibrate your lungs. Both of these are very important to the FDA. They are both approvable endpoints, the lung function assays. They are both being included in the normative study, the REACH study by the CF Foundation. So we will be able to compare to that as well. Nice summary data we have collected so far.
Of course, the Cohort 3 and Cohort 4, we have not shared any data yet. The decision for that is in Q4. We are presently enrolling, if you go to the bottom, up to 20 again, 10 milligrams. It is an open label study. We have modified the protocol to strengthen the baseline. We had problems in the first three cohorts with some wobbly baseline participants. That is no longer a concern. That is a prerequisite to have a normalized baseline before they participate in the study. We think we have strengthened the statistical meaningfulness or opportunity to establish more meaningful data in this fourth cohort as well. Now going on to LUNAR-OTC. Previously, we used to call this the dark horse of the company. Now I think we will see how the market perceives the data forthcoming later this month.
But we do intend to provide a phase II data readout in the U.S. and Europe, looking at multiple biomarkers and safety and tolerability. I think it's time for this program to become more visible. It's sandwiched in between our commercial enterprise vaccines, which is stable and revenue producing. Then you have the CF product, which has a significant commercial opportunity. Do not forget about this program. It's the number one urea cycle disorder. The present standard of care is ammonia scavengers. We are not in the business of addressing ammonia or the symptoms. We're functionally curing the disease by delivering a normal ornithine or by expressing or making a normal enzyme in the liver. Our approach is ideal for functionally curing this disease. We had successful in phase I and phase I-B.
We navigated our way through two phase II trials in the United Kingdom, European Union, and the United States. This data readout is coming in later this month. So it's very near term, and we've also received all the Fast Track designations for this program. On these slides are summarized what we're collecting, but it's several doses over a few months. Every other week, we administer this IV. We've narrowed the dose range down to 0.3 and 0.5, and we've completed all the dosing, and we're now just preparing to share the data. Just as a refresher, we have shared some interim data previously. So we have shown that glutamine is being normalized in these subjects. This is very exciting data for stable adults, because stable adults take all these ammonia scavengers to help control their ammonia, but their glutamine levels are still high.
If you functionally cure the disease, you not only normalize ammonia, but normalize glutamine. Glutamine is, when you have elevated ammonia in your body, that ammonia gets converted to glutamine before it's scavenged. Glutamine is a problem. It crosses the blood-brain barrier, releases ammonia there, and these patients still don't feel very well. Even though they're following the rules and the regimen and the diet and the pills, they still have elevated glutamine, and this is an important biomarker for stable adults. We've also increased ureagenesis. We have a nice assay for N15 ureagenesis that showcases improvements there. We maintain stability in ammonia within the normal range. This is more important to the younger patients because that's where there's more unmet medical need and severity and fatality or death. So this is very important to the younger population.
So we've already shown some interim data that's encouraging. Looking forward to sharing an update later this month on the complete data set and the regulatory path clarity from the two Type C meetings. As I mentioned, we're going to be providing an update to the platform as well. It's been a while since we've updated our capabilities within the intravenously dosed liver platform as a whole. Now going on to vaccines, and we're running out of time here, but I just want to emphasize that we did, going to the far right, then just in August, we regained control, global control, over KOSTAIVE. The market received this well.
It's nice to have control over an asset that's not only potentially revenue producing as a COVID vaccine, but we've already shown, we've already proven in multiple phase III studies that we've shown that we express more antibodies and longer lasting and broader antibodies than the present market leader in a very large vaccine market opportunity. Please remember that COVID, even if there's fatigue in this room and from investors about COVID, that the COVID vaccine opportunity is larger than shingles and RSV combined. It is a very large market opportunity, and we've already showcased that we have superiority over the market leader, and we have complete control over that asset. So stay tuned as we build that asset out commercially. It's going to be an interesting asset to follow. I mentioned it's already been published in multiple phase III studies.
You can see that we have more antibodies, longer lasting antibodies, broader. This implies a lot, right? It implies more protection, longer lasting protection, better protection. This is what people want to see, especially if you're elderly, the 65+ crowd. There was 30,000 deaths attributed to COVID last year. People are dying from this regularly. It's going to be a really bad COVID season this season if you look at it. So it's interesting. H5N1, I'm just touching on this. This is the bird flu program with BARDA. There's a publication that's going to be coming out on this. We look forward to people to look at that and you can compare it to other technologies because the government evaluated dozens of opportunities in vaccines against bird flu. We'll be able to publish on that. Then just mentioning our Arcturus board of directors.
Moncef is our board chair, and just a very sophisticated board to help us with this CF decision coming up in Q4 and to help guide the commercial success of the vaccine enterprise and everything we're doing. It's more than just the management team. We've got a great board. I'll pause there and we'll take some questions. Thank you for your time.