Okay, great. Well, hey everybody. Thanks for joining us for our Leerink Global Healthcare Conference. I'm Roanna Ruiz, one of the senior biotech analysts here at Leerink, and it's my great pleasure to introduce the Aquestive team. With me I have Dan Barber, CEO, Matt Greenhawt, CMO, and Ernie Toth, CFO. Thanks so much for joining us.
Thanks.
Thanks for having us. Great to be here.
Yeah, definitely. I'll kick it off with a big picture question before diving into the details, and for anybody who's new or revisiting the Aquestive story, could you just recap the company's core strategic focus areas? You know, what are your goals for this year and beyond, and thinking about Anaphylm?
Sure. No, Roanna I appreciate you asking that question because it gives me the opportunity to remind people while Anaphylm is the center of our attention and our resource work right now, and the story that we'll spend most of our time on today, we are a bigger company than just Anaphylm. We have two technologies that we have in-house and we have worked on for years. We actually have six FDA approvals we've gotten over the years. As we look forward beyond just Anaphylm, our newer technology, which we call Adrenoverse, which is taking epinephrine and turning it into a prodrug, has a lot of opportunity, we believe, beyond just Anaphylm and into other indications.
When people learn about our company, while they will first see Anaphylm, they should realize we have a revenue stream from the things we've done in the past, and we have a pipeline that we believe will be valuable with our other technologies.
Yep. Sounds good. Starting with some of the fresher news for Anaphylm, because I do get a lot of questions about the lead program there, thinking about the recent CRL and how far you've come from that point, can you talk about, you know, next steps, timelines, just help refresh everybody, like where things stand now and what you're looking forward to.
Sure. Well, the phrase I'll harp on for a second is complete response letter. One of the questions we get from people is, what does a CRL mean? What it means is it is the complete response from the FDA after reviewing your entire application. What you look for in your CRL are what are the deficiencies after that complete response and what are not shown as deficiencies. What we really like about the package that came back in the CRL, the silver lining, so to speak, is, the clinical part of our package had no deficiencies. In the complete response, there are zero clinical deficiencies from the discussions we'd had with the FDA.
Our focus right now on the CRL, and I'm sure we'll get into the details, really is on executing on the things that were in the CRL. We believe we can do that rapidly. We believe we can resubmit by Q3 of this year, which would lead to an approval, early in 2027.
Yeah. Sounds great. Framing the CRL as a bit of a roadmap going forward, how are you thinking about the FDA feedback up to this point in terms of some of the questions they've been asking, and in terms of timelines and executing on those two studies that were in the CRL, how are you going to ensure that you meet your goals and timings there?
Sure. Well, I mentioned before we have six approvals over the years, oftentimes. Look, the FDA is full of people who are trying to do a good job and make sure they make the right decisions. Oftentimes the correspondence you get from the FDA is a little bit confusing, and sometimes you feel like you need a decoder ring to understand exactly what they're saying. With this CRL, it's the exact opposite. It couldn't be clearer what we need to do in order to remediate the deficiencies in the current CRL. The roadmap which we've given to all of you is very straightforward, we're focused on executing on it. In terms of the studies we have to do, there are two studies. There's a human factors validation study, which we will redo. We've already.
We already have our protocol, we already have our CRO, we already have our time slot for running the study, and then there's a PK study, which we have the same thing, protocol done, CRO assigned, dates set. We feel very confident that we can get through the timeline we've put out. We have submitted our Type A meeting request to the FDA. We'll have our meeting in the next few weeks, and we'll be off to the races.
Nice. Sounds great. I'm gonna double-click on that because I do get questions about both those studies that you're gonna execute on. Maybe starting on the human factors side. What instruction changes or questions are you digging into to address the FDA's questions there, and what's your confidence going in?
Sure. Well, I'll start with the confidence. We feel very confident. We think that the changes that are necessary are straightforward. I will say I do believe they could have been addressed in the original approval process. I think timeline-wise on the FDA side, it appears it just didn't work out for them to get to us soon enough to have that conversation. The first thing is opening the package. Our a package in the previous human factors validation study, you had to fold the top and then tear, and the FDA was concerned that could be difficult for some people, so we got rid of the fold. Now you just tear.
The other thing that came out in the CRL is we had a number of people, I believe it was 19 out of 166, who put it on top of the tongue instead of under the tongue. In the original human factors validation study, if you can imagine the foil, it didn't have any instructions or a picture on it. The new one, which for those in the room will be able to see, we put a picture. That shows, well, here's where you put the film. And then the third thing was around tolerability, where we had very few people who that was an issue on. I believe it was four.
In terms of tolerability, which goes to making sure you not only put the product in your mouth, but keep it in your mouth, we have added to our instruction base and we'll also provide more education and demo films in the physician's office so that people are familiar with the product. From our perspective, all of these are very solvable and we think we have the right path.
Yep. Looking to the other side of the package in terms of PK. What are you thinking about, looking into there and how do you think that could translate into possible labeling, you know, changes, things like that?
Sure. There's potentially five arms in the PK study. Three of them we have done multiple times. One is an IM. I can't even count at this point how many times we've done an IM arm. One is our product given by a clinician, same thing. Another is self-administration, which we've done, now we'll have more data. Those three we do believe are approvability gates, and we would expect them to look exactly like they've looked the previous times we've done them. The other two arms do speak to when people don't apply the product the right way, and we think the FDA is we don't see those as approvability arms, we see those as arms where it's informational for the FDA. What does happen when you put it on top of the tongue?
What does happen when you chew it? We already have top of the tongue data, we would expect it to look similar. The answer is, from a PK perspective, you have a lower Cmax and a longer Tmax, but still an efficacious dose. I know we'll get farther in the conversation and Matt will give some perspectives, we do believe that pharmacodynamic measures, even on top of the tongue, are robust.
Got it. I wanted to zoom out a little bit. You have these studies that you're working on, potential incremental label changes coming through from there. Like, where do you see Anaphylm's ultimate label landing, you know, with this additional work that you're doing? Just help frame it for if everything goes well, what would be your base case? Yeah.
I think in terms of let's start with the downside. Will we have things on our label that are difficult or problematic? I don't see that. We got labeling instructions in our CRL. They're all straightforward. We're happy to agree to them. While you never want a CRL, there is an opportunity on the upside in my mind with the work that we're doing, where now we can provide more characterization, potentially right in our label, that as we look at the market and as we watch what happens with the competitive set, may give us an opportunity to put some information in there that before we wouldn't have thought of.
We don't see a lot of downside, to the label getting, I'll call it junked up with things, but we do see a potential upside for us.
Yep. I hear you. I did wanna ask, you know, it sounds like you're very confident going in. You set a goal in terms of possible resubmission and approval for Anaphylm. I guess, what's your conviction in hitting those timelines? How smooth is it to execute on these additional studies and things like that?
Sure. We have high conviction. I would say barring a curveball out of left field that nobody is looking for or sees, we know the Type A, we know the slots for our studies, we know the work that has to go in. We have a great development team that's been through the wars and knows how to handle these things. I see no reason right now why we wouldn't be on schedule.
Yep. Then a twist question that I've got from some investors is, you know, what's the likelihood that, let's say, you submit your new package to the FDA, et cetera, that you could be reviewed faster than expected and get earlier approval?
Oh, come on. That's Of course, we're gonna get approved early, right? No. manage the things you can control and work on the things you can't, right? With the FDA, obviously it's up to them how long they take to review the application. It is a six-month clock. What I would say is a couple of things. One, there is precedence in this space. Our competitor did also receive a CRL, and instead of going the six months, was approved in four months in their window, despite me telling you that wouldn't happen and you said it would, and you were right.
I think with this FDA, if you are clear with what your objectives are, you're engaged with them, and you're aligned with what they're trying to accomplish, there's more opportunity for success on both sides. I do think we stand a chance of beating the clock, and we'll definitely, as soon as we've put the application in, make sure we spend the time on that.
Yep. I hear you. Thinking about the sort of commercially, let's say everything goes well, Anaphylm gets approved. How are you thinking about the broader category validation? You have a competitor ahead of you sort of building the market, and then what do you plan to message when if and when Anaphylm eventually launches?
Sure. Well, our messaging. I think my mic is off. It's fine for everyone in the room, but I don't know if this is that's a problem.
Still hear me or?
Sure.
Okay. go ahead.
Okay. There we go. It's back on, right? Okay, great. Your question was, where will we position ourselves in the market, right?
Yep.
Our messaging is, again, really clear, and also, I think, not dissimilar from the other alt product that's out there, which is it's easy to carry, easy to use, and works fast. Patients will have the opportunity to then decide, well, which of the products that are available fits that those measures better than the other, right? We do know in our survey work that when we you've heard me say this, seeing is believing, that when we put the auto-injector, the nasal spray, and the film in front of people and say, "Which one do you want?" The film does really well.
Yep, I hear you. just thinking ahead, in terms of peak penetration p ossible peak sales for Anaphylm, how are you thinking about that?
Sure. Well, look, I'm the CEO of Aquestive. I want it to be as high as possible, right? I think that let's step back and look at the overall market. If you take the industry players, myself, the other companies out of the mix, and you just ask allergists or experts in this space, "What's going to happen in the next three to five years?" I've yet to find someone who says the auto-injector is going to stay dominant. I do think that the market is going to move and is moving to alternate delivery. The question becomes: What is the mix between that alternate delivery? I personally believe that if we execute well, we stand a really good chance of being favored in a lot of scenarios, and that bodes well to, hopefully, a very high market share.
Yep. That makes sense. Another question that I've gotten often is just thinking about the back-to-school surge for epinephrine products. How do you think that could layer into the launch for Anaphylm going forward?
Sure. I'll turn it over to Matt in a second here because he definitely has more experience in the office than I do. What I would say before I hand it to him is I think our timing is really good because we, in theory, if approved, on the schedule that we expect, will be approved at this time next year, which will give us some months to prepare and be relevant in that time zone. Matt, maybe you could talk about the experience there.
Yeah. The, a lot of it goes back to shorter half-lives on devices, so if you're giving something for school, you obviously want it to last the whole school year. There are action plans as well that we usually prescribe at the same time, so the schools really wanted everything sort of June 1st or later, which sort of sets up this time period. Also, you know, think about as a parent when it's easier to bring your kid in the summer.
We sort of do have these surges, but that doesn't mean that we're not prescribing at other periods of time. Now, with products that have longer half-life, sort of that acute period where you have to get it sort of the summer of or whatnot, there's a little bit less reliance on that, so. You know, that's still a very important historical time. you know, we have patients that come in and get epinephrine prescribed all times of year.
Yep, I hear you. Tagging onto that, your competitors run into a couple bottlenecks in terms of trying to break into the back-to-school surge, et cetera. What are you learning from that experience and any sort of strategies that you wanna use for Anaphylm going forward?
Yeah, and I think the adding to how Matt framed it, the first is the ability to open up that window. If it doesn't have to be this narrow four or eight or 12-week window because your product offers a different way of renewing, we think that is an area that can be helpful. I think there is a part of this that. Look, we're not gonna change the behaviors of moms with middle school kids, right? They are going to follow what is known in their world, part of this is also just making sure you have the muscle and the presence and the process to make sure you can be helpful and insert yourself in that timeframe.
Yep. I hear you. I'll give you a bit of a break, Dan. I'll throw a couple questions to Matt. In terms of medical affairs perspective, how are you thinking about just leveraging the real world evidence so far for no-needle epinephrine and some of the data so far for Anaphylm in terms of messaging to physicians and detailing?
You know, the experience has been interesting. You know, for a long time we didn't have a choice. We had what the technology offered, which was an auto-injector and evolved out of atropine years ago, you know? It's nice to see this evolve. It's nice to see some of the issues of the absorbency be solved with different carriers that allow sort of the space to evolve. The real world evidence continues to build. I think more physicians are becoming familiar with it, feeling comfortable prescribing it, that's always going to, you know, look good. Obviously, you know, the data are exactly what you would expect, it's underwhelming because of course it works. Of course it works fast. That's, you know, these questions. The 505(b)(2) pathway works.
The FDA wouldn't use that if it didn't. You know, some of the initial inertia to like, "Oh, will it work or whatnot?" I think, you know, the allergy community is past that. It's moving in and getting it approved in, what, prior arts or whatnot. You know, what I like about sort of the needle-free space is obviously, you know, every child and when you do a challenge, a food challenge or, you know, have immunotherapy reaction or whatnot, even adults, nobody likes being injected. It's a, you know, it's sort of a necessary evil. Now we have these alternatives that are going to meet more preference of what somebody might want for optimal delivery. If, you know, this had been there to begin with, I think you never would've seen an auto-injector evolve.
You know, we wanna increase awareness that you have alternatives. We wanna highlight our data to show that this is something that we're confident should work very well clinically and deliver everything that you would expect with epinephrine. Really just spread the word and that's, I think the task over the next couple of months here.
I hear you. As you're going forward with that, wanted to dig in and think about what are some of the themes coming through when you're speaking to allergists and starting to message the story about Anaphylm? Is the conversation going towards more the no-needle route of administration? Is it about onset of efficacy? What sort of bubbles up to the top as what they're asking about?
I think all of that. Can you take out your phone for a second?
Oh, sure.
Let's start with this. Can you show the back? Where is it? It's on you, right? This is the biggest obstacle as a prescriber and what keeps me up at night, you know, is my patient going to have their product with them when they need it? The answer is, before, I sure hope so, but data shows probably not, and if you do have it with you, are you even going to use it? There are plenty of data that show it's delayed, you wait, you sit in the parking lot of the things like you don't wanna know how the sausage is made on this one. It's frightening as a prescriber. You know, you spend all this time in a visit prepping somebody to use it, when to use it, and then they just don't execute.
A product that you can have with you, tucked behind your phone, in your pocket or whatnot, drastically increases the probability that you're going to use it. You take away the needle, and you just put it in your mouth, under your tongue, you're enhancing all the features of why somebody would wanna use it and reducing sort of any negative experience with it. All the allergists, every physician, every prescriber knows this, and I think it's just sort of making people aware that you have this option. You take some of the PK and PD properties of this drug, and, I mean, this thing performs so well in terms of what you're comparing it to, really robust levels. It gets in fast. The pharmacodynamic response, which, you know, means your heart rate, your blood pressure, superb.
It does everything that you'd want it to do. You want that to happen fast. You want that to rise as quickly as possible because you're treating shock, right? If you look at some of the way that these historically have performed, you gotta wonder, "Oh, boy, this it works, but can that be potentially enhanced?" I think that that's what our opportunity is here.
Yeah. That's super interesting. Another question I've gotten often is: How are you thinking about the low-hanging fruit patients that may get Anaphylm first if everything goes well? Who would be picked out by physicians as the ones that really should get Anaphylm first, assuming it's out of the gate available commercially?
It's an interesting question. I've actually never looked at it like that. I mean, any patient who has a risk of an allergic disease where there's potential for anaphylaxis, that's first. You know, it could be the peanut allergic kid that you see at 10 or the venom allergic adult, the beekeeper who just refuses to give up the hobby and is stung frequently. Really anybody, the whole target audience, anybody with a risk for anaphylaxis would be first. I think something like this is just, it's so easy to carry. It's gonna prove ideally easy to open, I mean, it's solving all of the issues, all of the inertia that we've seen clinically. You know, it can't come fast enough.
There's a really good potential to sorta turn around a lot of the morbidity that we see with anaphylaxis outcomes resulting from simply human behavior, sort of not doing what you're sort of instructed to do when you need to. Again, if we can simplify this, I would be very surprised if everything doesn't improve in that aspect.
Yeah.
if you're okay, I do wanna put a spin on that.
Okay. Sure. Go for it.
I think, Matt, from a medical perspective, put it perfectly of course. Well, any patient is a candidate. From a who are the first movers that we expect, our research right now shows that, especially in that teenage to college age, think of 15 to 30 as kind of the fastest prime movers or people who will adopt quickly. I think about my son, who is 19 and at college. As a parent who's 10 hours away from your child, that's a natural spot to want to make sure that there's a higher level of compliance. That will probably be the first place we see movement, we'll expand to there. To Matt's point, ultimately, this is for everyone.
Yep.
I mean, I would agree with that. That age in particular is something that, you know, typically you're looking at like a food allergic patient or something like that. Allergists are very afraid of sort of, you think about all the risks that my 21 year old daughter might be taking on a, you know, without food allergy. Add in that additional medical risk. To have that in. Every kid has their phone with them. The ability to have that behind their phone, it's going to put the medicine at least potentially in the right place when they do take risks that we as parents really can't control.
Don't wanna know about.
Yeah, definitely.
Yeah. I'm not, I'm not gonna touch the topic. Yes, that's super helpful. One last commercial question before I ask a couple different ones. Could you refresh us on your commercial launch plans, number of reps, things like that advertising, promotion? Like, just level set, like where do you think you're gonna go with the launch with Anaphylm?
Sure. Well, first we're giving Sherry Korczynski, our Chief Commercial Officer, a very short break, so that's why she's not with us today, is she rests up before the next push as we look towards our next target launch date. As we look at the market and we watch it evolve, we do think that more reps will not only increase our ability to see more physicians, but as you heard Sherry talk about last week, frequency with the competitive landscape is important. We wanna make sure that it's not just awareness, but it's action on top of awareness that we're able to drive through launch, and we do think that going from 50 to 75 reps will do that. We still don't see DTC as a place for us in particular to focus.
We do like that there's DTC going on in the space, and actually, if you look at Q4 in 2025 in totality, the overall market grew nicely. In Q4, the market grew by 9% year-over-year. Auto-injectors on their own grew by 5%. The DTC work that's going on is actually expanding the auto-injector world, and we believe we'll also benefit from that when it's our turn.
Yep. I hear you. I'll throw a question to Ernie as well. Could you just remind us or frame, like, how are you thinking about cash position now? Recently had some updates with the RTW financing as well, could you just emphasize, you know, what's been updated recently? How does that excite you going forward and, you know, thinking about cash position with Anaphylm launching soon?
Well, I think one thing that's important Roan , that the company is probably better capitalized today than it ever has been. Certainly ending 2025 with $120 million in the bank provides us certainly the runway to see through all the remediation efforts, to resubmit with the FDA, keep our core commercial team intact and give them another year, you know, nine months to a year to prepare to launch. That's important. You know, with the capital raise that we did in August, of which RTW was a lead investor, and also the revenue interest financing that they provided was an important part of that, and an important part of our capital structure going forward.
We did announce last week that they had extended the timeline for that. We now have until June 30, 2027. We think that's more than enough time for to get Anaphylm through the process and hopefully approved by the FDA. It provides us the launch, a part of the launch financing, for Anaphylm. It was also, you know, important to note as part of that, they also invested more money in the company. We appreciate their support. I think it's a great vote of confidence in the company. Let's also remember this, that, you know, we've given cash runway. They said we would end 2026 with $70 million. That doesn't include any of the additional proceeds from RTW if approved. As a reminder, we still own all of the rights to Anaphylm.
If we were to out-license for Europe, that would bring in additional cash. Let's not forget Libervant, our other product that comes off of ODE early next year, that we still have the U.S. rights to, that could provide us additional capital. We feel we're in a very good place to get through this year and support the launch of Anaphylm, if approved.
Yeah. Great. That's a good segue in terms of thinking bigger picture pipeline, thinking about Anaphylm. You still own all the rights for Anaphylm, like partnership, possible BD. How are you thinking about those themes going forward?
Yes. Right. No, no. Look, I think Ernie framed it perfectly. The company's better financed than ever before, which leads to us being better positioned than ever before. Obviously on the BD side, which Ernie's heavily involved in, we think it'll be a busy year. From a pipeline perspective as well, I think you'll see us continue to make pipeline moves to make sure that not only do we have Anaphylm coming to the market, but we have more things behind it for people to get excited about.
Yep. Got it. Got it. In the last couple minutes, I'll ask another big picture question.
Sure.
What do you think is most underappreciated by the Street in terms of the Aquestive story? Is there anything coming up that could possibly surprise investors?
Yeah, I think that there's maybe the right way to put it, I don't know that anyone will be surprised by this.
Okay. Yeah.
There's a lot of cynicism in our space, right, about the ability to launch, the ability to get through the complexities of all the things around you. I think the opportunity for Aquestive in a launch scenario is to surprise to the upside. I think it's so easy to look at the barriers which are real and are significant and think that it's always going to suppress what happens. There's an element of Anaphylm that just captures people's imagination, and I see it all the time when I talk with my family and friends. When you show them what you're working on, their faces light up. I've been at Aquestive almost 20 years. We've worked on I don't know how many products. I've never had anyone's face light up on any of the other products.
I think there's something here that if we execute well and our commercial team can really capture through marketing, we could really surprise people.
Yeah. That sounds great. Looking forward to more updates. I think we're at time now, so I'll just thank you again for coming.