Make them ready whenever.
Awesome.
Is this working? Afternoon, everyone. Thanks for joining us at the Leerink Partners Global Healthcare Conference. My name is Nick Gasek. I'm one of the VPs in Equity Research here at Leerink. Pleasure to have Aquestive Therapeutics here with us today, joined by CEO Dan Barber, CFO Ernie Toth. Great to see you both.
Thanks for having us. Nice to be here today.
Great to have you. I guess to kick it off, I think we can start with some bigger picture questions and then maybe dig a little deeper into the story from there. I guess for investors who are new to Aquestive, how would you summarize the company's core focus areas in 2025, particularly with respect to Anaphylm and Libervant?
Sure. I mean, you said it perfectly, Nick. First of all, thank you to you and the team here at Leerink for having us. Always a pleasure to be here. In terms of our core areas of focus in 2025, Anaphylm, which is our Epinephrine Sublingual Film, is absolutely the priority. We have to get through the finishing of our filing, which will happen in the next few weeks. We started that last week. We need to get our acceptance, which would happen in the second quarter. We may or may not have an AdCom in the second half of the year, making sure that goes well and then getting to approval. That is just on the development side. We also need to be ready for launch. We are doing a lot of work around that as well.
The other piece that maybe people are less familiar with is we do want to expand our product into international markets. We will start the filing process in Europe, U.K., Canada once we have our Anaphylm U.S. FDA application into the FDA. Anaphylm is clearly the center of our focus. The other area that is emerging, and you'll hear us talk more about as the year goes on, is our AQST-108 epinephrine topical gel product. The prodrug epinephrine platform that we're using for Anaphylm, we believe can be used for a lot of products. With AQST-108, we're taking that same great technology that our science team has developed and putting it to work in a topical gel for alopecia areata. We're excited about that product.
We'll have it into our phase II-A this year and hopefully have some data by the end of the year. Those two things are clearly the focus for this year.
Sounds great. Maybe focusing a little bit on Anaphylm, could you talk a little bit about the value proposition here and severe allergy and anaphylaxis? I guess where do you think this product could ultimately fit in the treatment landscape?
Sure. I appreciate that question because as far as we're aware, we're the only non-device that's in development. We do believe that when you look at the patient group that requires or needs a rescue product, which by the way is really underserved right now, we think we service all of the patients in that world. Why? Because our product not only is an oral product, but it can be carried on the back of your phone. If you can see, I don't know if the camera can pick this up or not, if you can see the yellow on the back of my phone, that's actually our product. When you look at what's going on in the patient world, patients aren't carrying their product. When someone has a severe allergic reaction, they're very diligent about carrying their rescue product for about a year.
The second year, they're somewhat diligent about carrying their product. By the third year, they're not carrying it. Providing a product that is an extension of you, is an extension of the way you live, which we all carry our phones, we think is a really important way to reach to all of those patients.
Got it. No, that makes a lot of sense. I guess thinking about the current epinephrine landscape, what do you think some of the drivers are commercially or, I guess, from areas of unmet need? Because you're really grilled upon what we currently see with the auto injectors.
Yeah. It's interesting. I think we're, if I step away from just wearing my Aquestive hat for a second and I look at the space and all of the different companies that are in this space, I think we're having the wrong conversation. The mortality rate in this space has not moved in the last 40 years. Think about that. The auto injectors came on the market, now most recently the nasal sprays, yet the same number of people are dying every year from anaphylaxis. That tells you that something's wrong. When you dig deeper into, well, what's wrong, there's two things you find out. One is the population of people who have a prescription or have been given a prescription and didn't fill it because they didn't think it was important.
There is a population of people who were not even aware they had an allergen, and so they had nothing with them. I think the awareness part is huge here. Not just us, but the other companies in this space need to work hard to make sure that the population that should be carrying a rescue product is carrying a rescue product. I think not only will that hopefully ultimately impact the mortality rate, but by nature it will expand the market.
Got it. No, I think that makes plenty of sense. I guess if Anaphylm was approved, it would probably be the second no-needle epinephrine product on the market. I'm curious, in your view, what types of patients would be drawn to this type of profile over what's out there now? Who do you think the early adopters would be?
Sure. No. Traditionally, Nick, this is something that you and I have talked about before, right? Traditionally, when a product is first to market, the ones behind it do not get as much of a market share because you are talking about molecules. If I pick on the migraine space, if you think of triptans, sumatriptan, Imitrex was the first one to market, got the biggest market share. Then there were six more triptans that came, and they all came in pill form. There were seven pills you could choose from. It is hard for patients or healthcare providers as well to differentiate in that environment. What we are talking about here, though, is drug delivery. It is a delivery device.
I think every year for patients, when they come to the physician and they have that opportunity to choose between an auto injector, a nasal spray, and a film, we'll have that opportunity every time to win over new patients. Being a first mover is, of course, an advantage, but we don't think in a delivery environment that we're at a major disadvantage over time, over three to five years, being the second to market.
Got it. I guess maybe switching back to the filing, I guess, could you give us a status update where that's at? I know you mentioned that you've started the filing. When do you expect to complete that? I wanted to ask you about the pediatric data.
Sure. Absolutely. We started our filing last week. Some people call it a rolling submission. There are modules in the NDA, as you know. We put in the module three, which is the CMC module. By the end of this month, we will have all of our modules in, which starts the clock for the FDA. When the clock starts at the end of this month, there will be a 10-month review cycle, which would put our potential approval date somewhere in late January or early February. The FDA has a 60-day period to decide whether they are accepting the NDA for review or not. They have 14 more days before they send you a letter. You will hear people talk about a 74-day letter.
After the end of this month, 74 days later, we'll get our letter accepting the NDA, and then that will take us to the approval period in late January, early February.
Got it. It sounds like things are moving along nicely.
Yeah. Yeah. No, we're very happy.
That's great. Maybe coming back to the pediatric study, you mentioned the top line data was in line with your expectations. What does that mean? What role is this data going to play in the filing overall? Where do you think things land?
Sure. Yeah. This data is important to make sure we get our label all the way down to seven years. It's based on weight, so it'll be 30 kg, which is roughly a seven-year-old. Without this data, we would be from 17 years of age and up. It's important for making sure the label is complete that this data goes into our NDA. In terms of what the data looked like or what we were looking for, it was a very simple study. It's a single-dose study where the pediatric subject comes in, they're dosed with our product, we pull blood samples for a couple of hours, and they go home. When you look at the data, it's just a line, which is kind of a boring thing to look at.
What you want to be able to do is bridge that to your adult data. So when we say it's in line with expectations, what we're really saying is it looked exactly like we expected it to look, and it ties to the adult data, and there's no outliers or issues we have to deal with.
Got it. I guess do you have plans to disclose that data down the line in terms of patient numbers and things like that? How should investors think about that?
Yes. We absolutely will disclose the data. What we have to decide is what conference or poster or presentation format to do that in, but we absolutely will give that data to the public.
Got it. I think that would be very helpful. You have been messaging, you are preparing for a potential adcom in the fall. I am curious how that is going and what topics do you think the FDA would want to dig into at a possible meeting?
Yeah. No, the AdCom is not only for us, but also on the FDA side, it's a lot of work. For us, it's an opportunity to tell our story. We have already hired what's called a regulatory communications firm. They're already putting together the team in our company as well as KOLs who would present our data. We'll start that process of making sure we're ready to tell our story the right way if there were to be an advisory committee. In terms of what the FDA would ask, and just I'll digress for one second just so people understand, the way it works is the FDA would come up with a series of questions, three or four, that they ask to their standing advisory committee for that area. We would expect those questions to, one, be the utility of film in this space.
Does the committee see utility in the film? We think we win that. The second and third are usually around your application. In our case, we do know that with our product, when you take a second dose, there are what we call outliers where they get higher amounts of epinephrine than the known EpiPen label. What that means is you, meaning Aquestive, we need to prove safety on those outliers, which we think we have a compelling case for. The second place we would expect them to talk is on the tail of our curve, somewhere around 45 or 60 minutes, we know that we dip below the comparison to the manual IM. That is another spot where the FDA will likely have questions.
Got it. That makes a ton of sense. Just thinking about other aspects of the profile from a PK/PD standpoint, is there anything in the data that really stands out to you so far relative to the competitors or maybe even the auto injectors so far?
Yeah. We really believe that our profile is the best profile for a patient going through a severe allergic reaction. Nick, if you remember last week with earnings, I used the phrase, it's just common sense. Think about what's happening in a subject or a patient, excuse me, when they're having an allergic reaction. First, your blood pressure is dropping. Second, histamine is, well, first I should say histamine is circulating through your body at a high level. Second, your blood pressure is dropping. Third, you may have some constriction in your airway. Bad things are happening, and you're having a lot of anxiety about how do I get out of this moment. What do you want in that moment? You want a lot of epinephrine. You want it to go away.
When you look at those first 15 or 30 minutes, which, as I mentioned before, is when most fatalities occur, our curve looks very much like the auto injector curve. We are very comparable in those first 15 or 30 minutes. That's where we think the life-saving attributes of the product really come through. When we think about the competitive advantage of our product, that curve to me is a really important part of the story.
For sure. I've gotten this question from investors previously. It's Anaphylm, it's an oral film. You're supposed to put it under your tongue. What happens if I swallow it? Does it still work?
Yeah. We actually did a study where, and that was as the FDA asked us to build out our complete package for our filing, we had to do studies where it was with different liquids, so hot liquid, cold liquid, different pH levels. We had people eat a peanut sandwich and then take our product. One of the things they asked us to do is have people swallow the film instead of having it go under the tongue and dissolve. What we find is the Cmax level, so the peak concentration level, actually is still relatively high. I want to say I could be a little off in these numbers, but it still got to about 250 pg /mL . What we do see is that the Tmax goes out to about 25 minutes. We go from 12 to 25.
If you look at the other products in the market, their Tmaxs are already 20-25 minutes. We know that when people swallow the film, they're still getting a significant dose of epinephrine.
For sure. I guess maybe looking back at the conference, Quad AI last weekend, you had some new data there. Anything you were particularly excited about on those new data sets? There was quite a bit there.
No, thank you for that question. We had a full team in San Diego last weekend, and I know you had a chance to look at what the team was presenting. We presented four posters. Those posters were across two really critical areas for our company. First was we did a study in the fall. We called our OASIS study where we exposed people to a known allergen. If you're allergic to pineapple, let's say, we exposed you to pineapple. We allowed symptoms to occur, so edema, scratchy throat, whatever. There were systemic symptoms as well. We dosed our product. What we saw is in a median time of five minutes, all symptoms are gone. We know that our OASIS study shows our product works on localized edema very fast.
We know the OAS patients we were using, also 10% of the time it develops into anaphylaxis. That OASIS data we think is very powerful. When we have the opportunity to talk with physicians, that will be a differentiator for us that no one else has. The other thing we showed in a couple of our posters was our excursion data. When you think about, and I have to take it out again.
You got to do it.
Like a proud parent, right? When you think about our product, it not only fits on the back of your phone, but you're going to be at the soccer field. You're going to be on a ski mountain. You're going to be wherever you are over the course of a year with your product. We have created the excursion data to show what happens. Let's start with the easy side, the cold side. If you are below the standard storing conditions, which for almost every drug on the planet is, except for ones that need to be refrigerated, I think 68 to 77 degrees Fahrenheit, you can go to minus 10 degrees Fahrenheit if you want with our product and still use it. You cannot freeze. Our product does not freeze. There's not enough water in it for it to freeze.
That alone we think is a distinct advantage. If you go to the high side, where you are in high temperatures, we tested our product at 158 degrees Fahrenheit and held stability for, I think it was a month, or it was either a week or a month of time, which, by the way, you and I could not survive at 158 degrees Fahrenheit. We know that our product can be put into extreme temperatures and still be durable. I should mention you can actually submerge in water for a week if you feel like it as well. We think it is very durable for people in the real world. We think that is also part of the story.
Got it. I guess just assuming this product is stored at room temperature, what's typical shelf life? How does it compare to the auto injectors and other competitors?
Sure. Yeah. We filed with 24 months of shelf life. So we'll come to market with 24 months. The auto injectors, it's hard to tell, but the consensus I've seen is they're somewhere around 18 months. The nasal spray product, I believe the adult is at 30 months and the pediatrics at 24 months. Look, from our perspective, people renew their prescriptions on an annual basis. If I think about my wife and our children, every July or August, you're into the doctor's office and you're saying, what do I need for the school year? We think the important thing is to make sure that the product has a shelf life that is well beyond that yearly renewal time period.
Absolutely. Maybe looking ahead, assuming Anaphylm's approved potential launch in early 2026, what are you doing to prepare? How many reps do you think you're going to have at launch? How are you going to drive awareness maybe ahead of that launch?
Sure. Sure. We've done a lot of work building out the internal team already. I'm excited to say we have not only an SVP of Commercial who, by the way, used to run the EpiPen franchise at Mylan. I'm very excited that she's part of the team. We have a VP of Sales. We have a VP of Market Access. We have a marketing team that's in place. That kind of base infrastructure, we have. They've done a phenomenal job of already building the advertising campaigns and the things that we would do from a marketing perspective. On the Medical Affairs side, we also have a Medical Affairs leader as well as some people around that person who are driving poster presence, conference presence, making sure we have publications.
I believe the number of conferences that we're attending this year for Anaphylm is something like 22. Being out in the world on awareness right now is absolutely job number one.
For sure. I guess thinking about prescribers, any specialties you're planning on targeting initially? Do you think some specialties would be more receptive to an oral film versus the auto injectors or nasal? How are you thinking about targeting the prescriber base?
Sure. Yeah. When you break apart the prescriber base, clearly allergists and pediatricians are the place to start. You're not going to go to kind of the general GP world until you've really built your brand and saturated those call points. We'll start with the allergists with big practices who see a lot of people, and we'll work our way down from there. When you look at this space, while there are a large number of physicians who prescribe an EpiPen, the allergists really are at the center of that universe. If you just think of you and I, if you were diagnosed with a need to carry an EpiPen, you would likely have that done by an allergist. You'd go back to your GP after that. That allergy call point is the central place to start.
For sure. I guess with a competitor that recently launched, any learnings you could take away from, I guess, the early days of that launch? How are you personally thinking about maybe advertising promotion around your product if and when it's approved?
Sure. I think of it in three ways. One, I continue to believe awareness is paramount in this space. Awareness is one of those places that the different competitors should be able to work together because people are still dying from anaphylaxis. There clearly is a need for more people to be carrying whatever product. We should all be working together to make sure that awareness is as big as possible so that we actually create the outcome that is good for patients. The other two things that are important, one, we have seen with the product that's out there now that some physicians want to see clinical efficacy because even the EpiPen did not have any efficacy studies performed in order to be approved. That leaves physicians in a spot where they usually have proof and they do not have proof.
With our product, we will definitely come forward with our OASIS data and use that as a surrogate for efficacy where we show them, hey, while we couldn't run a clinical efficacy study because it wouldn't be safe to the patient, we have this great OASIS data that shows you how quickly the product is working the way it's supposed to work. The third thing that we, of course, have been watching is the payer. The always famous payer universe. That was one of the reasons we just brought in an in-house market access person is we need to be out in the payer world telling our story now. Some of the payer problems are just inevitable. There will be NDC blocks. There will be plans that are just not friendly to new products.
The more effort and work and time you spend with payers, you will eventually get them to the right place. As we've watched Anaphylm or as we've watched our competitor and their experience, we want to be a little bit more on the ball and learn from their experience as Anaphylm comes to market.
Got it. That makes a ton of sense. I guess it might be a good time to pause, see if any questions from the audience. I can keep rolling. I want to make sure we have enough time to cover everything. But Libervant, I'm curious, what's your longer-term outlook for this product? I know it's a commercial product right now. It's revenue-generating. How do you envision this fitting into the acute repetitive seizure landscape longer term?
Yeah. My first thing is I just feel bad for Libervant. I feel bad for the patients who use Libervant. I mean, these are two- to five-year-olds who, and think about it. If you're a parent who has a two- to five-year-old that's just been diagnosed with epilepsy and put on a maintenance medication, and then you're told, and by the way, if the maintenance medication doesn't work, use this rectal gel. I mean, that's a terrible situation to be in as a parent. We have a product that is currently on the market that they can use instead of the rectal gel. For those who may not know, there is a court case involved in Libervant where potentially we may not be able to always have the product available to two- to five-year-olds.
I feel for these children who are on this product and need to keep having it with them. There's that in the short term. In the long term, as of 2027, the orphan drug block that we're under expires and we'll be able to provide the product to all age groups. At that time, we will happily do that. We do believe Libervant continues to be a high-value and a high-value proposition product. We will in 2027, whether it's through our own efforts or working with someone else, make sure they have access to the product.
Got it. I guess maybe thinking ahead to 2027, how are you thinking about label expansion into that older than 12 population? And how large do you think this opportunity could really be?
Sure. Yeah. Libervant, it's funny. If you had asked me that a couple of years ago, I would have said it's a big opportunity for the company. When I look at Anaphylm and I look at AQST-108 and our topical gel for alopecia areata, and I look at vitiligo, perhaps right behind that, and then perhaps other places we use that prodrug epinephrine platform, those are big opportunities. With Libervant, the important thing is to make sure we get it to patients. Whether it is something that creates tremendous value for the company or not, I think it may be overshadowed by these other bigger opportunities over time.
Definitely. I wanted to ask you about AQST-108 and alopecia areata. I guess you're going to be starting a phase II-A in the second quarter. I guess what should investors be watching from an efficacy standpoint in that study? Is there a bar you're looking to beat or meet? How should we think about that?
Yeah. We have a phase II-A with 36 subjects. We do not have a placebo arm in it, which typically with a phase II-A, you would. The reason we have decided not to do that is because in this space, you track something called your SALT score. Alopecia areata, you get these blotchy losses of hair. Physicians will give you a score on hair loss based on different quadrants on your head. In a study, you check the change in SALT score. We feel that the science lines up very nicely here that we can restore immune privilege for people who have lost it in the immune response, which is attacking their body by tampering that immune response down with epinephrine.
When we think about a SALT score over a 12-week period, we think we'll see a substantial change in that SALT score. We feel strongly enough about the science and the change that we can create that we think any placebo drift that could be in there by not having a placebo arm will be more than carried by that change. That is why we went with a simpler design that we think will give a very nice reading at 12 weeks.
Sure. Sure. I guess thinking about 108, the topical gel profile, what features do you think would be attractive to patients in this space? What do you think is really going to drive adoption relative to what's currently available on the market? JAKs and topical steroids, et cetera.
Yeah. I think people, and I actually happen to know somebody, a pediatric person who has alopecia areata. It's a very hard thing to deal with. If you think about parents who are dealing with a child who has to go to school and deal with all of the stigma around alopecia areata, solving that quickly is important. Going to a JAK inhibitor where it is a systemic product, it has known significant side effects, it has a black box warning, that's using a sledgehammer to solve that problem. If we can create a localized epinephrine that stops the immune issue that's going on, allow hair growth to happen, especially in that pediatric space, we think that's a winner. We're excited for that, and we can't wait for the data to come out.
Got it. We'll look forward to that. Maybe a question for Ernie. I guess how should we think about cash runway, use of cash over the next couple of quarters? How are you allocating resources across the different programs?
As we just announced last week, we have $93 million of pro forma cash after accounting for our year-end balance and some recent raises we did through our ATM facility to bring in two high-quality institutional healthcare investors. We see that as providing runway through everything this year that we're dealing with. Whether it's the filing of the NDA, preparing for an AdCom, the 108 study that you just asked, Dan, about, all the pre-commercial work of preparing for a launch next year, Sherry and her team being out there at the advocacy groups, doing all the payer work. We're ready to launch next year upon approval. It gets us into 2026, hopefully an approval, early first quarter, and the initial stages of a launch.
Got it. That's very helpful. I know we're coming up on time here, but I want to ask a bigger picture question to close out. Maybe how are you thinking about potential partnerships either for Anaphylm or Libervant or maybe even 108? Is there any aspects you would be looking for in a potential partner? Is this something? Are these programs something you want to be advancing alone all the way? Or how should investors think about that angle?
Sure. From an Anaphylm perspective, our focus is on maximizing the value for our stakeholders. We know this product has tremendous value, not only from an investor and a company standpoint, but from a patient standpoint. We will only do something if it maximizes our reach. In the meantime, we are preparing and are ready and will launch on our own if we can make sure that we reach the people who need this product. In terms of longer term and the other programs, I think what you'll see us do over the years to come is make sure we focus in an area where we can leverage our knowledge and our ability and where we're the best people for that spot. You won't see us spread across a bunch of different areas that do not add up to something that works together.
That's the work that we'll have to do as Anaphylm comes to market.
Got it. Look forward to that. I think that's all the time we have today. Thanks for joining us and enjoy the rest of the conference.
Thanks, Nick. Appreciate it.
Thank you.