Thanks to all of you for joining us at the Global Bio-Innovation Forum during J.P. Morgan Week. I'm Steve Lo, and I'm the CEO of Vaxart, which is a clinical stage biotechnology company developing a range of oral recombinant pill vaccines based on our innovative delivery platform. Our stock is listed on OTC under the ticker VXRT. During today's discussion, we may make forward-looking statements, including statements about our future business strategies and operations, product development, and regulatory progress, including statements about ongoing and planned clinical trials. Actual results could materially differ from those discussed in these forward-looking statements due to a number of important factors, including uncertainty inherent in the clinical development and regulatory process and other risks associated in the risk factors section of Vaxart's most recently filed annual report on Form 10-K and also on other periodic reports filed with the SEC.
Vaxart undertakes no obligation to update any forward-looking statements after the date of this call. We are delighted to be a part of the Bio-Innovation Forum. There have been a lot of news about vaccines lately, but less conversations around innovation, and that's why we're excited to be here, because today we want to talk about innovation in vaccines. When you hear about vaccines, I'm sure the first thing that comes to your mind is standing in line, getting an injection, and we want to change that narrative. We, as a company, have lead candidates in Phase 2 for COVID-19, a norovirus vaccine candidate that is ready for Phase 2, along with influenza vaccine candidates. We also have a preclinical program exploring our technology as a therapeutic vaccine to treat HPV infection. We also have partnerships to accelerate our development and a very experienced team of deep industry expertise.
Joining me today are actually two of those members of the management team with this deep expertise: Dr. Sean Tucker, our Chief Scientific Officer, and Dr. James Cummings, our Chief Medical Officer. During today's discussion, Sean and James will review our underlying technology platform, the preclinical and clinical data supporting this platform, and multiple oral pill vaccine candidates, and our potential to improve access and acceptance of vaccines that can play an essential role in protecting public and individual health. We'll be sharing this information in a question-and-answer format, and I'll start with the question for Sean. Sean's our founder of Vaxart and has served as our Chief Scientific Officer since February of 2010.
He has a Bachelor of Science degree in Chemical Engineering from the University of Washington, a Master of Science in Chemical Engineering from the University of California, Berkeley, and a PhD in Immunology from the University of Washington. Sean, it's great to be working for a company that you founded. What was your motivation and objective in founding Vaxart?
Thanks, Steve, for the great introduction. Let me tell you a little bit about Vaxart. We started this company because what we wanted to do was have a different sort of approach from vaccination. We wanted the vaccine to come to the user rather than having to go get a shot, and I think the key from the standpoint of technology is we developed something that would be easy to give to people, in this case, tablets, because that's the simplest format you could possibly think of. No one needs training to figure out how to swallow a tablet. We all do that all the time. There's a lot of good technical reasons to develop this sort of technology. One, my background is in mucosal immunology.
And what we know is that injected vaccines are very good at making serum antibodies, but they're not always great from the standpoint of making antibodies in the nose, and the mouth, and the intestines, the places where your pathogens actually sort of show up and invade you. And there are some major advantages of making an antibody response or a T-cell response in these wet surfaces. For one, the type of antibody that's made is called IgA in the mucosal system, which is different than IgG. And IgA is an antibody just by the nature of having four binding sites versus two. It is much more effective from the standpoint of cross-protection and cross-reactivity. And I think that's important when you're talking about pathogens like flu and COVID, which are changing all the time.
The other thing, of course, is that, as I mentioned before, injected vaccines aren't known to make these sort of responses, but a mucosal vaccine can, and our vaccine makes antibody responses in the intestine. Of course, our tablets are swallowed. They're delivered to the intestine, but they also make it in the nose, and for things like COVID and flu, that is the first line of defense. The other thing I want to mention is that we wanted to make this, and again, my background originally was in chemical engineering, we wanted to make this very simple. We wanted to be able to go from one indication to the next very quickly without having to change the same manufacturing program over and over again with every new indication, and the technology works by copying and pasting in your gene of interest into this adenoviral vector.
And that vector is dried and tableted, and it allows it to go and work in your intestinal space. One of the key attributes of the technology is that when we make this adenovirus, it would get ignored because it's non-replicating. And when we put it into the intestinal space, we have this double-stranded RNA adjuvant that turns on the immune system and tells it to respond to something. And it responds just to the gene of interest. And whether that's a norovirus or a flu or COVID, it's really specific. And I think this is a key aspect of the technology, what we call the VAAST platform, is that you can use the same technology over and over again for all these different indications without having to start from scratch.
Thanks, Sean. I think we're always very excited about our technology and how you explained it. Certainly very helpful for the audience there. I'll turn the next question over to James. James is a board-certified infectious disease physician with a 26-year career in the U.S. Army and a proven track record in vaccine, drug, and diagnostics development. He served as the director of the Department of Defense's Global Emerging Infections Surveillance and Response System with viral surveillance laboratories in 71 countries. Wow. And also the director of translational medicine and regulated activities for the Walter Reed Army Institute of Research and a consultant to the Surgeon General for all medical research and development. And certainly, James, we're always delighted with your expertise, and especially with all the trials that we're running here.
But from your extensive expertise in infectious disease and vaccine development, what do you see are potential benefits of oral pill vaccines from a public health perspective?
Thanks, Steve. There's multiple benefits from an oral pill vaccine delivery platform. There's the potential for improved efficacy, and that's likely due to stimulation of both the mucosal as well as systemic immunity that Sean just went over versus what we see with most injectable vaccines, which is primarily just a systemic response. That wet surface interface, that's the point of invasion from those microbes, those infectious organisms coming into the body, so if you can stop it before it gets in, even better. There's a potential for improved safety. We have a very benign safety and tolerability profile in nearly 1,200 people across multiple vaccine candidates. Certainly, there's no injection site reactions because we don't inject this vaccine, but more importantly, when we look at this, our vaccine candidates have a safety profile that's very similar, if not the same, as placebo in the studies we've done to date.
The rapid development we've been able to achieve enables response to novel pathogens that crop up, but certainly also when variants of the current pathogens occur, whether it's in COVID or flu or what have you. From an access standpoint, I'm very excited about this platform for global health across all comers. It can be administered at home. There's no need for a medical professional, really, or to visit a clinic or pharmacy. There's no scheduling of appointments that has to be done or traveling to a site for a vaccination, which should make this approach much more convenient, and I think that's one of the key factors of people actually getting a vaccine. It simplifies distribution here in that there's no cold chain requirements. We have a very thermostable product, and also there's no red bag medical waste.
So there's no needles, syringes, et cetera, where we have to figure out how to dispose of the end products as well, and I think if you take a look at that whole milieu of the logistics, of the ease of administration, of the convenience factor, of the safety factor, and the mucosal immunity factor, which may have an impact not just on protective efficacy, but may decrease transmission, I think this has a chance to really change and increase vaccination rates writ large.
Yeah, that's really a good point there, James, and frankly, all good points that you make there. So yeah, I'll turn the next section over to talk a little bit about data. Sean, you're our founder, which really makes you our first scientist in the company as well, and so tell us about some of the preclinical data that Vaxart has that supports, let's say, potential safety and efficacy.
Yeah. Again, I've been doing this for a long time. We have lots of great preclinical data. And I think James really illustrated some really well the safety aspects of the vaccine. Again, in animals, sometimes this is hard to assess safety because animals, you can't ask them if they feel bad or not. But I do think that the data that we've accumulated in humans is very important for that. From the standpoint of efficacy, I'm going to tell you a little bit about a couple of different studies that we've done as examples of just how powerful this technology is. For one, we did this study in hamsters. It was published in Science Translational Medicine.
Effectively, we asked the question, if you have a mucosal response due to oral vaccination and you're making antibodies in the nose, does that have an impact on the ability of that vaccine to transmit to somebody else? For example, if I got vaccinated, but someone near me didn't, would that still allow my protective response to keep it from transmitting? We did this study in hamsters where we vaccinated either by oral vaccine or by protein vaccine or no vaccine into index animals, exposed them to a huge amount of COVID to cause breakthrough, and then put them near animals that had not been vaccinated. What we found. We blew air, again, from one cage to another, so they weren't touching.
And what we found is if you had either the injected vaccine or the oral vaccine, you could be protected pretty well from the standpoint of infection. But what was interesting is that if you had the oral Vaxart approach given to, in this case, hamsters, those hamsters were less likely to cause disease in the animals that are nearby, so they had less and less infection. And it basically showed that you could have dramatic impacts on transmission by essentially just making a vaccine that was given orally and made antibodies in the nose. And I think that was a wonderful study for us. On the avian flu side of things, I think we also had a very, very nice study where we showed that our vaccine that was a seasonal vaccine, quadrivalent, could protect against avian flu.
And I think, again, this has to do with the ability of our approach to make antibodies in the nose that are a little bit more cross-reactive. And I think those were major studies from the standpoint of just showing the potential of this platform, again, in preclinical models. But these are very nice accepted models from the standpoint of infectious disease.
Thanks, Sean. And what's great about Vaxart is we've taken the preclinical and we're in clinical trials, which, again, it has us as obviously a more advanced biotech company. And certainly, with James' extensive experience, we've been involved in quite a few clinical trials. So let me turn it over to you, James. Why don't you share with us some of the trials we're working on right now?
I sure will, and I'll go by some of the leaders in our portfolio because our approach is a platform approach. It's not just limited to one vaccine. We have a whole bunch, but I'll just hit the highlights for now. In our norovirus vaccine, we performed a challenge study where we actually immunized folks or gave them a placebo, and then we challenged them with norovirus and took a look at, did they become infected? Yes, no. Did they have symptomatic disease, and I can tell you from the challenge study, we showed that we had an impact and prevented infection in a good number of those folks. We had a study that was funded in part by the good people at the Gates Foundation where we looked at moms who had already delivered, lactating mothers who were breastfeeding.
We wanted to do a couple of things with this study for norovirus because this is a certain portion of the population that gets impacted by norovirus where we took a look to see, was it safe and well tolerated by the moms? It sure was. Did it give a robust immune response? It sure did. Also, we took a look at the level of the IgA in their breast milk. We saw a significant increase in their IgA, that immune response to norovirus. We went one step further. We took a look at the poopy diapers of the infants that those moms were breastfeeding. Sure enough, we saw some solid transfer of IgA from the moms to the infants. That was a pilot study done in South Africa by, I think, very good fodder for follow-on studies to come.
Within the norovirus portfolio, we also took a look at the older population. So along with testing vaccines in healthy 18-49-year-olds, we took a look at the population from 55 all the way up to 80, 80-plus. And we saw some very interesting things there. Certainly, it was safe. It was well tolerated. It provided a robust immune response. But there was no impact on immunosenescence or the impact of aging on vaccine response. We gave the same vaccine strength, et cetera, to the younger 18-49 folks as we did to the 55-80-year-old folks. And what we saw was very similar, if not the same, immune responses.
And that's very encouraging because it tells us that from our approach, one doesn't necessarily need to have to add additional amounts of vaccine or additional adjuvants, et cetera, that we get a solid performance of the vaccine throughout that 18 and above age spectrum. Recently, we completed a head-to-head study where we looked at our first and our second-generation constructs of the norovirus vaccine. And we were able to show, although it was a rather smaller study, we were able to show statistically that the new constructs gave an even more robust immune response to those who participated in that study. So we're moving ahead with our second-generation construct of our norovirus vaccine based on very solid data moving forward. Another item in our portfolio is, of course, the COVID-19 vaccine program.
We've already published and discussed our phase one results where we had very robust mucosal and also T-cell immune responses to our oral tableted COVID-19 vaccine. Then for flu, this goes back to 2017, 2018, thereabouts. It's published in Lancet ID. We did a head-to-head study looking at our influenza vaccine compared to an injected vaccine. It was Fluzone that was compared to at the time, probably the most popular injected flu vaccine around. We had some pretty good outcomes there. We showed that our vaccine candidate reduced symptomatic infection by 39% compared to placebo. The Fluzone decreased symptomatic infection by about 27%, so a little less than we did. We had an improved reduction in symptomatic disease. When it came to looking at infection, we showed a 47% decrease in infection where Fluzone showed a 43% decrease in infection compared to placebo.
So statistically the same, numerically superior. And again, a very safe, clean safety profile. And as I mentioned, those results were published as we put all the results in the peer-reviewed literature, present them at conferences. And that's in Lancet ID. And that's just the top three in our portfolio right now, Steve.
Sure, James. Yeah, and thanks for the history and the background on the results from the trials in the past. I know that as a company, we have certainly an important trial going on, as you mentioned. And I'm sure the audience is thinking, what should we expect in 2026 on certainly that trial and anything else? James, I'll turn it back to you if there's some updates you can provide in terms of what's in store for the future in 2026.
I can't tell you what the data is going to be, although I can tell you what the data is we're expecting. For the COVID-19 program, we had a sentinel safety group for that 400 folks, 200 receiving a licensed approved mRNA vaccine versus our product, which is pill-based. It's not injected. It's not mRNA. We took a look at those groups over a 12-month period, primarily for safety, but we collected immunogenicity. We collected efficacy data as well. We should have preliminary top-line results by sometime in Q1 of 2026. I layer that right behind that will come our 5,000-subject data, looking at the KP.2 variant, the 2,500 and 2,500 or so cohort of an approved mRNA vaccine injection versus our oral pill, not mRNA. We should have that in late 2026.
So I think we have two very compelling inflection points coming up that I'm pretty excited about. In terms of norovirus, if I can say with the portfolio format, we had, as I said, some very strong data come out of that head-to-head study looking at the first-generation versus second-generation constructs. And we're teed up to execute a Phase 2b safety and immunogenicity study that could potentially begin sometime in 2026, as early as that. And then for influenza, we continue development of our seasonal and our avian influenza programs, again, bolstered by the very positive results we've had in the past.
Looking at the strength of this program, not just in the accessibility and the ability to deploy these vaccines rapidly, but the turnaround time of once a variant or strain is recognized of being able to turn that around into a vaccine that can be distributed to the population in a very easy way.
Great. Thanks, James. Yeah, since we're nearing the end of our discussion, I'll summarize a few items just to remind the audience. Since this is a Bio-Innovation Forum, hopefully what we've shared with you is that there is still innovation going on in vaccines, and as Vaxart, we're proud to be working on this innovation. As both Sean and James have shared with all of you, we have not only the convenience of the oral pill vaccine candidates, but also there's some good scientific rationale that supports why we certainly have a belief that we'll be successful with our upcoming trial results, and on top of that, we are very excited about what 2026 is going to bring us, as James had outlined, and if you have more questions or you want more information on Vaxart, please go to our website at www.vaxart.com.
It has even more of the corporate presentation graphics that highlight some of the science that we've been working on, as well as many of the articles referenced that James and Sean have talked about, and with that, before we end, first of all, I want to say thank you again for the opportunity to present at this forum. I'll turn it over to our founder, Sean, for a few closing remarks.
Yeah, I think from our standpoint, I'm proud of the fact that we're not developing just another needle stick. We're developing something that would be different from the standpoint of the vaccines. And it completely could revolutionize the entire industry because of these tablets that everybody knows how to take, the fact that they're room temperature stable. In theory, we should be able to send them to everybody's house. We could send them by DoorDash rather than having you go to the pharmacy to go get them. And I think from that standpoint, this has the potential impact that could be revolutionary in terms of the vaccine space. And I think that from my standpoint, this is why I'm really proud to be part of Vaxart and have started this company.
Thank you, everybody. I appreciate your time and listening in today.