All right. Good afternoon, everyone. Welcome to Oppenheimer's 36th Annual Healthcare Life Sciences Conference. My name is Andreas Argyrides. I'm one of the senior biotech analysts at Oppenheimer, and today I have the pleasure to be joined by Aquestive CEO Dan Barber and CFO Ernie Toth. Aquestive develops orally administered products to deliver complex molecules, alternatives to invasive and inconvenient standard of care therapies. They are advancing Anaphylm, a sublingual formulation of epinephrine for the treatment of severe allergic reaction. Great to have you today, Dan and Ernie. With that, I'll let you take it away with the presentation, if we have time, we'll jump into Q&A after.
Thanks, Andreas. Thank you for having us today. Thank you to you and the team at Oppenheimer for the opportunity, and I also appreciate all of you who are listening. I appreciate your time today as we walk through our story. Let me just start by, of course, reminding you of our safe harbor language. You can find this presentation on our website and read it in its entirety if you so choose to. For those of you who may not be familiar with our story, we are a publicly traded company, of course. We are focused on our drug delivery technology. Our technology, in particular, is oral thin film, and I'll spend a decent amount of time today talking about that technology, as well as some of the future technologies that we have in our company.
Our mission, as you would expect, is to take existing molecules that we believe are sub-optimized, use our technologies, our drug delivery technologies, and provide meaningful improvements to patients. We are an established company. We've been around for almost 20 years, and over those 20 years, we have brought six products through the FDA, and we've had 2.5 billion doses that we have delivered to patients around the world, in fact, on six continents across those products. The one we're most well known for is a product called Suboxone, which we make for Indivior. That product is in the opioid addiction space and has been on the market since 2010. As you may be aware, if you are familiar with our story, we do have a product launch coming up.
We actually have two product launches coming up, but one product launch in particular that we'll focus on. We are U.S.-based. We do our own manufacturing in Indiana, and, in terms of size, we're around 150 employees. I talked a little bit about our oral thin film technology. We partner that technology with something we call Adrenoverse. An Adrenoverse is actually our ability to take a drug, epinephrine, and deliver it into the body with various different release profiles. If you think about epinephrine, which is obviously not only natural in our bodies, but has been synthesized as a drug since around 1900, epinephrine has a couple of shortcomings. One, it has a very short half-life, so 5 minutes or less.
Two, it's very hard to control, three, it's really hard to gain absorption of epinephrine. What our prodrug platform allows us to do is take epinephrine and with different functional groups designed for different purposes, create different absorption and release profiles of epinephrine. We have an intellectual property suite around that technology. Well, I'll talk about our lead asset today. We do believe there are long-term capabilities with that platform to go into not only the allergy space, where we're focused today, but also the dermatology space. In addition to our oral PharmFilm technology, Adrenoverse is key to our future. Now, in terms of our existing products, what you see on this slide are a few examples of the products we make.
I spoke about Suboxone before, but we also make Emylif in Europe, which is an ALS drug, Sympazan here in the U.S., where we've partnered that with another company, one that I'm really excited about, which is Ondif, which we actually make for the Brazilian market and continues to grow year-over-year for the last several years, both for us as a company as well as our partner. When I look at the nausea space in this technology, in particular, it's very interesting, one that has done very well. From a pipeline perspective, once I get through the opening comments on the company, I'll spend almost all of my time on Anaphylm. From a pipeline perspective, we have three programs. Anaphylm, epinephrine sublingual film, is the primary program.
That is the one that is focused on severe allergic reactions, including anaphylaxis, and we'll get into that in a few minutes. We do also have Libervant, which is our diazepam buccal film for acute repetitive seizures. That's actually tentatively approved by the FDA already. We're waiting for a block from a competitor's orphan drug exclusivity to expire, which will actually expire in January of next year. Libervant here in the U.S. will come to market soon, and outside of the U.S., we've already partnered with a company called Pharmanovia to bring that to. I believe at this point there's actually almost 20 countries that they have filed in.
The third product we have is AQST108 epinephrine topical gel, which is in phase I, so we've gotten through preclinical and into the clinic, and that is for alopecia areata. We believe this showcases the versatility of our Adrenoverse platform, which I spoke about before, and shows the potential with not only Anaphylm, but with things to come in future years. I'm incredibly lucky to work with a great team of people. What you see on this page is our leadership team, as many of you know, especially of those of you who are experienced investors, having the right team is everything.
We are fortunate to have Sherry Korczynski, who led the EpiPen efforts at Mylan for a number of years, Matthew Davis, who is a seasoned Chief Science and Chief Development Officer, who comes out of much larger companies, and most recently, Dr. Matt Greenhawt, who was Distinguished Clinician of the Year for AAAAI, the allergy conference that is actually coming up this weekend. Very lucky to have a great team. I should also mention that I'm joined by Ernie Toth, our CFO, who's on the call with me today. I'll walk through this piece, but when we get into Q&A, I'm sure Ernie may have a few more comments on this.
We have worked hard over the last almost 2-3 years, to create the right capital for our company to execute on our strategy. We did a raise in the first half of 2024 that started to bring capital into the company, and most recently, in August of last year, we did a very high-quality raise where we were able to bring over $80 million of cash into the organization at the time, along with a compelling deal with RTW to revenue share a strip of Anaphylm once we come to market.
Between those 2 efforts and the cash we already have on hand, we believe we're well-positioned for not only getting through the process we need to go back through with the FDA, but also into the launch process that will come up after that. Let me dive a little deeper into Anaphylm, our lead asset, and the first thing you need to know is how the product works. I actually have an example of the product here with me today. It's a demo film, if you can see this, if I put it here in front of my body. What we believe our product will be able to provide to people, the first thing is the ease of use.
You can carry it on the back of your phone, which, anyone who's interacted with me before knows I show this all the time. It obviously is not only needle-free, but it's not a medical device, so you don't have any of the complexities of a medical device. You simply tear the pouch open, pull out the film, which you can see right here, I'll put it closer to the camera, and then put it under your tongue, where it turns from this film into a gel in a matter of seconds as your saliva hydrates the film and quickly starts to absorb. That leads to the third point, which is the product is designed to work quickly. We actually have, we believe, we...
products in this category, we have the fastest T-max that I've seen, which is at 12 minutes in our studies. The comparator in our studies, which was the auto-injector, is at 20 minutes. Speed is really important in this space, and what you see here is what's happening in the body, or the likelihood of an occurrence being more extreme if you do not treat early on. Each minute that goes by as your body is having a severe allergic reaction, the odds of needing greater medical intervention grow substantially. With our product, we believe people will take it early because they'll have it with them, and it's easy to take, and it will obviously have the fast, rapid absorption that we talked about before. The market size does continue to grow.
Last year, the scripts were just under $5 million here in the U.S. All of the figures you see here are U.S. only. We do anticipate that growth continuing in 2025, in fact, or the actuals for 2025 being over $5 million. When I look at the last couple of quarters for this market, the growth has actually accelerated, which is to be expected as new entrants are promoting the need for products in the space. Right now, the market is largely auto-injector. Over 90% of the market, as far as we can tell from the data we have, is still in the auto-injector realm. We believe that as our product comes to market, we'll be able to impact those auto-injectors and move them over to film.
The remainder of the market is nasal spray at this point. When you think about the auto-injector, another reason that our product becomes important is when you look at the behaviors of people in this space, and I'll point back to a story I've told before. DoorDash of all people did a survey on how often people will forget something and then go back to their house. What they found is that 40% of people forget something at least 3 to 4 times a month. You get in your car, you drive down the street, and you realize, "Ugh, I forgot XYZ," and you have to go back and get it.
We know that a lot of us, half of us, are forgetting something, at least multiple times a month, and the rest of us are probably forgetting something one to two times a month. Another survey that was done actually in the allergy space asked carriers of the EpiPen how often they would go home to get their EpiPen if they forgot it, and they were out or they were in their car, and 0% said they would turn around, go home, and get their auto injector. You have, through DoorDash, knowledge that we all forget things which we inherently knew, and you have through the allergy survey I referenced, knowledge that people will not go back if they forget their EpiPen.
It's really important that people have their product with them at all times, since that barrier, that behavioral barrier exists of the inconvenience of carrying it, the forgetfulness, the fear of having to use it, and of course, the complacency of not wanting to remember that you have something that could be life-threatening and always have that top of mind. When we look at our product, we believe that ease of carry is an incredibly transformational element of our story. I'll skip over this because I talked about this before. I do wanna also hit on this particular survey we did, and some of you who know me may have heard me talk about this before.
We've done plenty of surveys, like all companies do, but typically when you do a survey, it's a phone survey or maybe it's an online survey. We took our surveys one step further, and we actually sent to people mock-ups of the auto injector, the nasal spray, and the film. We sent the physical product to their house, and we then asked them, as you see in the data here, to open up the package, look at the offering, and then tell us which one they would like to carry. Out of fifty, I think it was 52 people, 52 or 53 people, we found that only one person preferred the nasal, and only one person said, "Well, I don't know whether I want the film or the nasal.
I want them equally." Everyone else in the survey wanted the film. What that tells us is, as we have suspected, seeing is believing with our story and getting out in front of the doctor, in front of the patient, making sure they can actually see our product and understand our product, will drive not only use of the product market share, but also having people carry the product and have it with them when they need it. In terms of launch readiness, we continue to have our commercial team in-house ready to go. We've kept on not only Sherry Korczynski, who I talked about before, but all of her immediate staff underneath. When it comes time to ramp up our commercial team once again, which is just a matter of months, we're ready to go.
Let me actually skip forward and talk a little bit about the complete response letter we just received from the FDA. We had a PDUFA date on our Anaphylm product, which was January 31st. On January 30th or 31st, we received from the FDA a complete response letter, and that will require us to do additional human factors work, which you can see on this milestone slide is in Q1 and Q2 of this year, as well as an additional PK study, which, as you also see, is largely in Q2 of this year. We are quickly working through the human factors and clinical PK study that need to be conducted. We've guided publicly that we will resubmit in Q3 of this year.
It is a six-month review clock with the FDA, depending on when we file in Q3, we will have our approval. We would expect to have our approval in Q1 of 2027. We'll obviously provide more guidance on our activities and what we've been up to in our earnings call next week. I do want to just spend a minute or two on the other elements inside of our organization. We do have, as I mentioned before, a pipeline product called AQST108, which is our topical gel of epinephrine. It is also a prodrug. It is a different prodrug than the one we're using for Anaphylm, the prodrug with AQST108 is designed to release differently. With Anaphylm, we want to absorb quickly, cleave off the prodrug quickly, and have that instantaneous action in the bloodstream.
108, which is local only, so it's not a systemic product, we want to gain absorption and then have a slow release over a number of hours. The prodrug we're using in this case, in AQST108, allows us to do that. That continuous release of epinephrine in the damaged tissue, we believe will allow for a restoration of immune privilege. We're in phase I on this study right now, on this program right now, and we'll expect to have more to say in the next quarter or 2. In terms of the opportunity around the indication, which is alopecia areata, we do believe t hat there's a significant opportunity right now when patients begin to.
have alopecia areata, they will start out with a steroid, which generally doesn't work overly well. The next step is to move into taking a JAK inhibitor, which is an incredible leap from a steroid to a JAK inhibitor. We believe we have a spot right in between those two, where we can be relevant and helpful to the patient community, and that's where we'll focus. We also believe by doing that, we get away from the black box warnings that are associated with the JAK inhibitors. Here you can see a little bit on the study I talked about before. The phase I study is in a number of subjects, who have alopecia, and they're dosed once a day, and at the end of the study, we'll look at local tolerability as well as PK and PD impact.
In terms of the milestones, I should have mentioned before, we did open our IND back in Q4. The phase I study, we'll have an update on next week, you can see the additional work that we plan on doing in 2026. With that is the story of Aquestive at this time. With that, I'll open it up to Andreas and any questions people may have.
Yeah, for sure. I think obviously, you know, you guys got a platform, a successful platform. You mentioned the pipeline here, but a lot of focus in the near term is definitely going to be on Anaphylm. Maybe, and you provided some really cool tidbits on the, on the survey and all that. Maybe we can get your take on market dynamics. What do you... You know, we have neffy that's on the market right now. We've had some pretty good initial uptake for in terms of, like, market share in the auto injector market. They're, you know, trying to continue to grow that. There's some, you know, strong projections there on our end as well. We hope the market continues to grow.
I think I asked this question a bunch, but is this market, um, is growth in the market going to be largely driven by awareness, or is there awareness now? Is it more of changing, um, habits and comfort around a novel product outside of the auto injector, which has been so firmly entrenched for decades? How are you guys thinking about this, and then when, you know, when approved, you know, what does that mean for your expectations on the launch?
Yeah, no. I, first, let me say, I think it's a great time for patients to have multiple options coming to market of different delivery systems or other than an autoinjector in a space that, Andreas Argyrides, as you put, for decades, that's all you had. I do think what you're seeing with the nasal spray, and we'll see what they have to say in their upcoming earnings call, but I think it's a significant lift to change behavior. neffy being the first one there, has had to do all of it so far. Obviously, when we come to market, we'd like to think we'll be helping also to change behaviors in the market.
My personal belief continues to be that three to five years down the line, there won't be a lot of people taking auto injectors. I think it will move over to these newer products because they are easier to use, they are less invasive. In our case, I talked through our advantages. I think that as patient awareness continues to grow and as HCP comfort continues to grow, there will be a broader move over to alternate delivery. Of course, I do believe that will be a battle of patient preference and marketing and all of those things, of whether people choose a nasal spray or a film.
That makes total sense. Real world data is also going to be a part of improving that comfort around a novel product, I guess.
Correct.
Let me ask, you know, because I think it's top of mind for investors as well, and you did a good job explaining it, but maybe just how I'm trying to use my words carefully in terms of trivial, but, like, how smooth, I guess, is the are these requirements that you need to provide to the FDA with the PK study and the human factor studies? Maybe just a little bit more color, if you will, just to give us a sense of, you know, your level of confidence of completing that successfully.
Sure. Well, it's all relative, right? Compared to the clinical questions that we've been sharing with the investor community for a couple of years, that the FDA had, these are very straightforward things that we need to do. When we look at the mountains we've climbed to get to where we are, those were all way more significant than what is left. The important part is we don't have approval today. Until the day we have approval, our entire focus, whether it's easy or hard, will be on making sure we completely execute on the work that needs to be do, the work that needs to be done. The human factors validation study that we need to do and the PK study that we need to do are things we know how to do really well.
Let's get it done, let's execute, make sure it's clean, get to the resubmission, and then, of course, we'll continue to communicate to everyone.
Okay. To be clear, these are not, new types of studies. Studies that you've done before, that you just got to replicate with a few little tweaks and touches there per the FDA's request.
That's right. That would be a simpler way to say it. These are things we've done, we know how to do it, and just make sure we do it the right way.
Perfect. Fantastic. One more question, just on the 6-month timeframe, is that something that was communicated to you with the FDA, or is that standard in terms of from the PDUFA file? Yeah.
It is standard. There's 2 types of reviews when you resubmit. One is a 2-month review, which generally doesn't have new clinical data, so if you changed some basic thing in your CMC or something like that. Then there's the Type 2, which is a 6-month review, which is generally where you have clinical data. We have clinical data. We would expect them the FDA to put us into that Type 2 category. Having said that, we will advocate for ourselves. We will be out in front of it and talking to them and saying, "We're not sending you a lot of data.
Six months seems like a long time to review a human factors validation study and one PK study. It will be completely up to the FDA whether they move faster than the six months, but it is definitely plausible that they could.
Okay, fantastic. Ernie, I don't want to leave you entirely and just have you just staring at the screen. Maybe just give us. I know you guys provided a slide on the financial picture. Also, you know, there, I think there was a mention of the RTW optionality here. Just thinking about cash runway launch, you know, money for the launch, all that, maybe a quick word or two from you would be good.
Sure. Thanks, Andreas. We're close to our earnings call, so we're not going to be able to give any formal guidance. What I can reiterate is what we said earlier this year. We ended 2025 with a little over $121 million. That provides us a comfortable cash balance to get through this year, to deal with what we now know of all the issues with the CRL, the remediation that needs to be done. It also supports the commercial staff that we're keeping on board to prepare for a launch next year. As we said, we would end 2026 with a healthy cash balance, significant cash balance, I use the exact words, that would get us into 2027 to prepare for launch.
That does not even include any of the additional funding from RTW on an approval and accessing that line or any other funds that would come from out-licensing any products. Just a reminder, we still own all the rights to Anaphylm, so they are available for us to monetize, should we decide to do so. In addition, we have Libervant in the U.S. that comes off ODE next year that would be available for us to license. We feel that we have the cash on hand, plus things, options available to us to support a launch upon approval by the FDA.
Fantastic. You guys have had a lot of things to be excited about. You're in a good position financially, so we'll look forward to... You absolutely, you guys got to execute until Q3, and then we'll hopefully, you know, the clock will start ticking then, on our way to approval.
That's right.
All right. Appreciate it, guys. This has been great. I know we are at time. Again, thanks for the insights, the presentation. I always like to get the full, the full picture of the company versus just always focused on, what investors want with Anaphylm. Kudos to you guys, we'll continue the conversation. Appreciate it.
Thanks a lot. Take care.
All right. You got it. With that, we'll conclude.