Aquestive Therapeutics at Citizens JMP: Strategic Moves in Allergy Treatment

Aquestive Therapeutics (NASDAQ:AQST) presented its strategic priorities at The Citizens JMP Life Sciences Conference 2025 on Thursday, 08 May 2025. The company, led by CEO Dan Barber, focused on ANIFILM, its oral film epinephrine formulation for severe allergic reactions. While preparing for FDA approval, Aquestive emphasized both the challenges and opportunities in expanding the market for non-injectable epinephrine options.

Key Takeaways

• Aquestive’s NDA for ANIFILM was submitted to the FDA, with acceptance expected in June.
• The company anticipates a potential AdCom by late October and a PDUFA date in early February.
• ANIFILM shows a two-to-one preference over existing medical devices in trials.
• Aquestive is leveraging existing contracts and engaging with payers for a smooth commercial launch.
• The U.S. epinephrine market, valued at $1 billion, could grow to $2-3 billion with increased awareness.

Financial Results

• Aquestive plans to provide a detailed financial update in their upcoming earnings call.
• The company reports a healthy financial position with multiple funding options.
• The U.S. market for epinephrine is currently around 5 million doses annually, valued at $1 billion, with potential growth to $2-3 billion.

Operational Updates

• ANIFILM’s NDA submission is a key milestone, with FDA acceptance anticipated in June.
• Aquestive is preparing for a potential AdCom, focusing on the product’s PK curve and OASIS study results.
• Manufacturing flexibility is ensured by controlling its supply chain, mitigating disruption risks.
• Pre-commercial activities include engaging with medical conferences and key opinion leaders (KOLs).

Future Outlook

• Aquestive is prioritizing a smooth FDA approval process and preparing for a first-quarter launch next year.
• The company is targeting high-decile allergists to drive ANIFILM adoption.
• Market expansion is expected as non-injectable options gain popularity, with the potential for the market to double in size.

Q&A Highlights

• Aquestive is eager for an AdCom to showcase their program and gain a positive vote from MDs.
• The company aims to include their PK curve and OASIS study findings on the product label.
• The OASIS study demonstrated rapid symptom relief within five minutes of administering ANIFILM.

For further details, please refer to the full transcript below.

Full transcript - The Citizens JMP Life Sciences Conference 2025:

Jason, Citizens: Thank you everybody. Good morning and welcome to the Citizens Life Science Conference. Really excited to be joined next by Aquestive Therapeutics. Aquestive is a company that has, successfully leveraged its oral film technology through multiple approved products. Our focus right now, the company’s focus is, ANNAFILM, which is an oral film, epinephrine formulation for severe allergy.

Company recently submitted the NDA, so so, you know, a a lot going on there and preparing for launch too. So we’re joined today by Dan Barber, the company’s CEO. Dan, welcome.

Dan Barber, CEO, Aquestive Therapeutics: Thank

Jason, Citizens: you. Maybe just ask you to give a couple intro comments and we’ll jump into some Q and A.

Dan Barber, CEO, Aquestive Therapeutics: Sure. First, Jason, thank you to you and the team at Citizens for inviting us back again this year. I was saying to you before, this is my fourth year in a row, which I feel like in this industry is a is a nice long run, so I’ll I’ll take it. Look. This is in my mind, I’ve been at a quest of a long time, the most exciting time we’ve had as a company.

We just filed with the FDA our ANIFILM application which is an epinephrine product which would be used for anaphylaxis and severe allergic reactions. So that is something that was monumental for the team and we’re incredibly excited about where that takes us. As we look forward there’s two things that we are highly focused on. One is obviously getting ANIFILM through the FDA process as smoothly as possible, and two of course is commercial, commercial, commercial. There’s a lot of focus right now on all the pre commercial activities and where we’re going.

So we’re 15% of the way through the clock with the FDA, goes by fast, and we want to be ready.

Jason, Citizens: So let’s just start there. You submitted the NDA just a few weeks ago. We’re waiting to hear confirmation of the PDUFA date. Just remind us of the timelines there and I’ll ask but at a very high level how have the interactions been from FDA so far during the review?

Dan Barber, CEO, Aquestive Therapeutics: Loaded question in today’s world, right? So first on timelines, pretty standard timelines. We would expect to receive our acceptance letter from the FDA in June so we’re getting pretty close to when that should come through. We would expect if we do have an advisory committee meeting which the FDA said we may have one, We would expect that to occur in call it mid October to Thanksgiving, somewhere in that range. And then of course our PDUFA date which will get assigned when we get our acceptance letter in June, we expect that to be the January, early February.

So those are kind of the three milestones to point to. In terms of our interactions with the FDA, business as usual. What I would say is we are very aware as we look around at the problems that the FDA is facing around us, but so far we have not experienced that.

Jason, Citizens: And then guess another loaded question, how are you thinking about an AdCom?

Dan Barber, CEO, Aquestive Therapeutics: Well for us we really want an AdCom, right, which is maybe kind of an odd thing to say for a company but we want our moment to really highlight our program, get the awareness up, have an advisory committee of MDs that is put together by the FDA give that positive vote for our program. So we’re hopeful that AdCom does happen. We’re preparing like one will where we have our KOLs lined up. We’re spending time even on slides. But it is it is questionable in my mind whether with what the FDA is going through whether they’ll be able to put an AdCom together so we’ll see.

Jason, Citizens: And I think the other positive point there is we have a recent example of an AdCom for the nasal spray and we know exactly what FDA cared about it was speed of onset and that’s something that we can talk about the product profile later but you know exactly what the focus would be for FDA going into this.

Dan Barber, CEO, Aquestive Therapeutics: We think we do. When I look back, so we have six approvals that we’ve gotten through the FDA over the years and when I look at all of those approvals and I look at what we’ve done for this package this is by far the most interactions we’ve had with the FDA prior to a submission and by far the biggest package that we’ve put together. In fact I think we’ve put together the biggest epinephrine package anyone on this planet’s ever done. So we feel like we’ve done all the right work, we feel like we’ve had all the right conversations and now it’s time for the FDA to do its work. So we’ll dig in more into the development program, the data, but just at a high level when you think about product label, product profile, what are the key attributes of ANIFILM that you really would want to highlight?

Sure, sure. So the way I think about it is I would separate two things, One, what’s on the label and two, how we’ll position the product maybe is the way to put it. So the label, one of the most critical things we want to see on the label is our PK curve and we think our product really really shines when it comes to PK curve. As you know Jason, our uptake on the front of the curve those first fifteen minutes we match the auto injector. So that being in the label we think is important and that will be a big selling point for physicians.

The other two things that we expect to have in the label but we’ll see as we go through the process, One would be information on our OASIS study which is a study we did where we exposed people to an allergen and then gave our product so we have good results to show from that. And then the third area we’ve done a lot of work is in the storage conditions. So our product which we believe is a product that can be taken with you wherever you are in life, you can take it to the top of a ski slope, you can dunk it in water, you can leave it on the seat of your car in Phoenix, We think having the storage conditions on the label would be really helpful as well.

Jason, Citizens: Got it, fantastic. Alright, let’s talk about the commercial prep that you’re doing. So where are you today, what are the priorities? You said it was obviously a core focus for the next few months.

Dan Barber, CEO, Aquestive Therapeutics: Yep. So we usually have usually when we’re at your conference we’ve just had earnings. This year it’s a little backwards for us so we’re having this conference then we’ll have earnings next week. So in our earnings call next week we’ll walk through extensively what we’ve done from a commercial standpoint but what I can share today that we’ve already said publicly is that awareness is a huge focus right now. So we’re very very active in the community, we’re at all the medical conferences, we’re dealing with all the KOLs, we’re putting out posters and presentations.

So all of that work is foundational awareness work. And what we find with that awareness work is as we’re out in the community, in our company we’ve started to say seeing is believing. Right? So it’s this idea that when people actually see the product and they experience it and they have to, you know I have to pull out my phone when they see that you can actually carry the product on the back of your phone and that our storage conditions allow that, we really see people’s eyes light up. So when I tie our work and the seeing is believing that we see to our preference data, so our preference data shows a two to one preference for our product over the medical devices, that makes me very excited for once we have the opportunity to launch the product.

Jason, Citizens: So let’s talk about the data. You mentioned before that early part of the curve, the PK curve especially looks very similar to the auto injectors. Just walk us through that data set, the pivotal PK bioequivalence study.

Dan Barber, CEO, Aquestive Therapeutics: Sure, sure. So with all of the pieces and parts of our program or anyone’s program sometimes people forget the core, the fundamental, right? So the fundamental part of our pivotal program follows the FDA’s guidelines on how to get a comparable product approved. And there’s two elements. One is Cmax, right?

So do you match on the peak amounts you give? And the other is AUC, is your exposure the same as the other product? And in both of those instances in our pivotal study our Cmax actually is right between the two auto injectors. So forget the I’m we’re right between the auto injectors. On partial AUC out to sixty minutes which would be the other important measure we’re right between the manual I’m and the auto injector.

So on the two core pieces for approval we hit our marks perfectly. And the other obviously important thing from a pivotal study perspective is that we’re safe and well tolerated. So one thing I know has been discussed is that as you look at the thirty to sixty minute time frame, the

Jason, Citizens: tail drops off faster than other products. So that’s something that you’ve talked about with FDA, you’ve addressed, you have data to speak to. So can you talk about just how you think about that tail then the repeat dosing work you’ve done?

Dan Barber, CEO, Aquestive Therapeutics: Sure. So the first important thing to realize is that’s actually not an element of approval, That’s a, I’ll call it an ad hoc question from the FDA. So what the pharmacokinetics at the FDA has asked is okay if I look at just the forty five minute mark, just the fifty five minute mark, and I look at your geometric mean values they’re lower than the other products. How should I think about that? Right, so what we’ve said to them is a couple of things.

One we’ve said, well if you don’t look at just mean, you actually look at the data set, all of our points are within the data set of the other products. The second important point is obviously in this disease state, all the action is in the first thirty minutes. You want to shut down that mast cell degranulation and that’s what our curve does. So at forty five to sixty minutes you should be past the issues that you had during your severe allergic reaction. And then the final point which is probably the most obvious of the three is if you’re still having symptoms at forty five minutes that’s what the second one’s for.

You mentioned the OASIS study, think

Jason, Citizens: this is a really important study. Can you walk us through A) what the study was and B) why you conducted it and then we’ll come back and talk about what you actually saw.

Dan Barber, CEO, Aquestive Therapeutics: Sure, yeah. So the FDA originally wanted us to show some level of what happens with edema or any issues that may be in your mouth at the time of a reaction. So we actually first proposed that we inject people with histamine, cause a reaction and then dose our product. The FDA came back to us and said that seems a little complicated why don’t you just expose people to a known allergen. So in our study we exposed people to something that they were known to have an allergic reaction to and we did it twice.

So the first time we did it without giving our product and the second time we had them come back and we had them be exposed to the allergen. We waited fifteen minutes and then we gave our product and we compared what the change was between the two. When our product is not given we see edema, we see all the signs of an allergic reaction and that lasts for well past an hour. When we expose them to an allergen and then we give our product, within five minutes, the median time to all symptom relief was five minutes and within ten minutes all angioedema is gone. So we showed a clear benefit that not only we can show to the FDA but going back to your label question, when that’s in our label and we get to talk about that with physicians we think that is an important element especially when we look at our competitor who’s recently launched which is great by the way for patients.

We know one of the barriers they’ve pointed to is clinical evidence. So we have that clinical evidence and that study is a really important one for us.

Jason, Citizens: And just the last point which I think is funny because it’s part of the reason you started the study was you also showed the PK curve has no there’s no impact on the PK curve with or without edema. That’s right. Maybe let’s just talk about the competitive product for a second so an Intranasal was launched several months ago what’s the feedback you hear from physicians both in terms of their awareness and willingness to explore using the product?

Dan Barber, CEO, Aquestive Therapeutics: Yeah, so I think this is a groundbreaking time for this space. The auto injector, the EpiPen and then the subsequent auto injectors that have come through since then has really been the only thing people could use for forty or fifty years. Right? So the fact that now we’re starting to have these other alternate ways of delivering the product is not only incredible for patients but it’s going to allow a space that really hasn’t gotten a lot of attention or positive attention I should say in recent years to really expand to where it belongs. So what we really are excited about when we look at what our competitor is doing, think the awareness is really important.

And the expansion of the market we already see happening, so this first quarter this year was 5% growth over last year’s first quarter and we expect the market to double over the next few years so right now it’s at about five million doses a year we would expect that to get to 10 as these efforts take hold. So clearly physicians are used to

Jason, Citizens: injectable products, how willing do you think they are to try something different?

Dan Barber, CEO, Aquestive Therapeutics: Yeah I think it’s a spectrum. Think it’s like most products in life, there is definitely a pent up demand by patients, caregivers, healthcare providers who understand the problem in the space that people don’t carry their product, they start to become less sensitive to the issue they may have and now they are in a position of danger if they are exposed to the allergen. So I think that group is very, very open to the products that are coming out. As you go across the spectrum there of course will be physicians who, and they play an important role in the industry, who will say show me, right? They want to see the other physicians have success with their patients first before they adopt a new product.

So I think going back to the awareness idea, I think the best thing that companies like us can do to really get that spectrum to move in the right direction is make sure we put all of our information out, give them the opportunity to read our data, see how we have positioned our product and also see how patients react to the product. Are there things that you can do post approval to continue to build that clinical experience set for physicians? Sure, sure. And you see with the nasal spray that they did have a phase four commitment that involves use in the office. I think using any rescue product in an office setting, so as most of you are probably aware, a lot of allergists have people come in for food allergy tests or other tests and sometimes there is a reaction and they do have to give epinephrine.

Usually in a clinical setting that’s where the manual injection is used because it’s cheap But if you can create a study or a use profile where physicians are able to use your product in their clinic that can drive awareness and comfort with the product. So whether it’s a phase four commitment from the FDA or just good practice, that’s a great way to get people comfortable with your product. You mentioned the market’s growing.

Jason, Citizens: Can you just give us a little, a few metrics around market size today?

Dan Barber, CEO, Aquestive Therapeutics: Sure, yeah. Well as I mentioned before, right now our data suggests The US market is around five million doses which puts it at about a billion dollar marketplace. We believe, and if you look at prevalence, the number of people who should carry a rescue product of some sort is of course much much higher. The advocacy groups that talk about the need will put the number around 40,000,000. So you’ve got 5,000,000 rescue products being prescriptions a year versus 40,000,000 people who should be.

Now we all know from the difference between prescriptions and prevalence you’re never going to get to forty million but in our mind we think with the efforts that are going on, the awareness, the better products, the willingness of people to carry things that are more an extension of who they are than something that has to be carried in a bag, we do think that 5,000,000 goes to 10. So a billion dollar market goes to 2,000,000,000. Our competitor, and I hope they’re right, has pegged the growth at $14,000,000 a year so that would put it at almost $3,000,000,000 So we’ll see. Somewhere between 2,000,000,000 and $3,000,000,000 is probably where the market lands.

Jason, Citizens: Okay. And then what does the market research say that non injectables could become as a proportion of that market? Yeah.

Dan Barber, CEO, Aquestive Therapeutics: Our surveys and like most companies in our situation we’ve done a lot of surveys. Our surveys suggest that while the auto injector use won’t disappear, there will always be someone or some group of people who want the auto injector, it will become a minor part of the market. There’s no active awareness of the product, it is an older technology so we would expect that to become, pick whatever you want, a small percentage. And we do believe that the other new technologies, so the nasal spray right now and our product will take up the rest of the market. Obviously, we love our product.

Our data suggests that we’ll have more than the other product, but obviously time will tell on that front.

Jason, Citizens: And I I think it’s just we talked about this before. We can debate all day long which technology gets slightly more or less used but non injectables will be a meaningful part of the market so that that’s what people really should should think about in terms of the the opportunity.

Dan Barber, CEO, Aquestive Therapeutics: I don’t see how it won’t dominate the market. Just don’t see how it won’t.

Jason, Citizens: Another part of this is the payers. Mhmm. So what’s the work you’ve done so far? What are you hearing from them? And obviously you’re not going to guide to a price now but just how do you think about broadly pricing strategy?

Dan Barber, CEO, Aquestive Therapeutics: Sure. Yeah. Well we’re obviously watching very closely what’s going on with payers and with the launch of another product but there’s only so much you can really do, right? So from our perspective the really important point with payers is that everyone has access to patients. So we would expect in a well organized marketplace that as our product comes out, the current products, any products behind us, that the payers treat those equally and allow them to have access as long as people aren’t playing pricing games, right?

So that is our hope for this space so that the patient can benefit. The real work that we’re doing right now is also on awareness. You’re allowed when you’re a certain period of time ahead of launch you’re allowed to interact with payers. We’re doing that on a daily basis. And we actually, thanks to Libervant, which unfortunately in the courts we did not win in the courts, but thanks to Libervant we do have all of our distribution contracts and a lot of our payer contracts set up.

So those contracts are still live, they’re still active, and they’ll stay live, and that’s going to save us months and months of energy and startup time as we get to our launch.

Jason, Citizens: Got it. Yep. Commercial footprint. Uh-huh. Can you talk about the let me let me ask this in a different way.

So so just give us an understanding of the current cash position, cash runway, and in the context of especially in the market today, cash is a really important thing to be confident about. How do you think about the appropriate launch commitment?

Dan Barber, CEO, Aquestive Therapeutics: Sure, yeah. So next week at our earnings we’ll have more of a cash update But what what I would say broadly, what what would be comments I’ve already said before is when you look at the ways to fund a company, we are in a position where we have a variety of ways to do that. And that’s really important. And and anyone who’s followed our story knows that Ernie Toth and Ernie, our CFO is here with me today as well. We have over the years had to face times, especially several years ago, where we didn’t have a lot of Where it was you had to make this work or you had to get an equity raise done.

Where we sit right now, especially compared to a lot of companies around us, the options we have are actually really healthy. So we can partner, we can look to external markets to bring in money, we have debt facilities that we can employ if we wanted to, we can look to the equity markets if we have to where they become healthier, but all of those options together creates a basket that makes us comfortable that we have a healthy path forward. So I feel for the companies around us as we were talking before because I remember what that feels like but thankfully for us we’re not in that situation right now.

Jason, Citizens: So when you when you look at the nasal spray competitor where is the product getting used? What’s the kind of number of physicians and the profile of physicians that you think are an ideal target to think about early during a launch?

Dan Barber, CEO, Aquestive Therapeutics: Yeah. Well from our perspective the way we think about it, there’s no way other than focusing on the allergist and the high decile allergist. Right? It may seem like a very simple approach because it is, right? So for the first whatever period of time making sure in those high decile allergists that our voice is heard, that our market share is healthy, that our adoption rate is good, that’s going to be the core focus.

And then as you get past that launch period of time and you start to look to be broader, we’ll look to other places.

Jason, Citizens: Maybe just one more. Again, understanding you’re not giving guidance, if you get approval, you know, let’s just say towards the middle of the first quarter of next year, how do you think about the timing of the launch and how quickly you can start to get adoption?

Dan Barber, CEO, Aquestive Therapeutics: We’ll be ready. We will be ready. So we’re already hiring the key people as I’ve said before. We’re working with the payers as we talked about. We will have the infrastructure in place.

We will day one be, and I’m not saying product will be in the market day one, but day one we will be locked and loaded and ready to go.

Jason, Citizens: And this is often a question which companies coming to market with their first products have to contend with. You’re not coming to market with your first product. Just from a manufacturing perspective, where are you today and talk about supply chain.

Dan Barber, CEO, Aquestive Therapeutics: Yeah, no, and that’s one of the really powerful parts of the way our company is set up is we control our supply chain. We make our own product. So making our own product allows us a lot of flexibility as we go through that final component with the FDA where you’re working on labeling and what’s the final version going to look like. A lot of companies have to wait a long period of time because of a change right at the end of the approval process So that from my perspective is really important. We have looked at the disruption that’s in the supply chain from everything that’s going on from our perspective there’s nothing that we’re concerned about so that’s a really powerful position to be in for us.

Jason, Citizens: I’ll ask one more high level question. As you think about the next, you know, really important twelve months here, where do you spend your time? What’s the most important thing for you and the company to focus on?

Dan Barber, CEO, Aquestive Therapeutics: ANIFILM, ANIFILM, ANIFILM. This is we are a year ago when I was talking to you, we were talking about other elements of the company, which are still important and still there. But we’re in the zone right now where launching ANIFILM is everyone’s first conversation of the day and everyone’s last conversation of the day. Fantastic Dan, really appreciate you being here. Thank you for having us.

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