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Jefferies Global Healthcare Conference 2025

Jun 4, 2025

Richard Lowenthal
President and CEO, ARS Pharmaceuticals

Thank you very much. I'll present the beginning, and I'll invite Eric Karas, our Chief Commercial Officer, up for the commercial part of the presentation so he can present that part. I'm very happy to be here and present. Next slide. Oh, I'm clicking, or they didn't give me that. Okay. Okay, there we go. Forward-looking statement. This year has been a very active year for us. This is our first full year of launch of neffy, which is the first needle-free epinephrine product that is now moving very quickly. We're approved in type 1 allergic reactions, including anaphylaxis. We have both a 1 mg and 2 mg product now approved on the market for ages 4 and 15 kilos or above. We are also working on a product that will be even a lower dose in the future.

It might be a couple of years from now, but we'll have a product for children under 15 kg as well. We've had really strong executions to date. We've had a good first quarter for second full quarter of the year or of the launch, $7.8 million in net sales. We're just gaining traction. We just launched the 1 mg, so that's additive, about 23% of the market. We're really picking up very quickly on sales moving into what is a peak summer season. We believe this is a—and we'll talk a little bit more about the market and the population. It's a multi-billion dollar market size. Currently, the generic market is about $1 billion, but at our net, it should be about $1.7 billion. We believe the market can grow.

I'll talk a little bit more about this easily into a $3 billion market, but even potential to grow beyond that. We have NCAE-like IP protection. We have patents out to 2039. We have two major patent families. One is an NCE-like patent family based on the technology we use for absorption. It's the absorption enhancer plus epinephrine. The other is a patent family based on the ability to administer epinephrine at a low dose, which is very, very important given the toxicity of high-dose epinephrine. In addition, we believe that we're very well capitalized for this launch. At the end of the first quarter, we still have $275 million in cash. We believe that we have sufficient cash to get to cash neutral later next year. When you think about anaphylaxis, it's accompanied by some fairly severe symptoms, of course.

The major symptoms when you're having an anaphylactic reaction include urticaria and angioedema. Roughly, most patients will have urticaria and angioedema where they're having skin rash, hives, facial edema, tongue swelling, lip swelling. They also have difficulty breathing. About half of patients have bronchospasms. There are also gastrointestinal problems. About 40% of people have severe gastrointestinal reaction. They have cramping, pain, vomiting, which are all signs of anaphylaxis. In any patient, you could have a subset of these symptoms. Really, nasal is—what did I do? Sorry. Nasal is actually one of the best routes of administration next to injection. Obviously, people don't like to inject themselves. As the next best route of administration, we believe nasal makes the most sense. The reason is you avoid the angioedema. You're spraying directly into the nose. It's a passive absorption like NARCAN.

You don't need to breathe. The bronchospasm doesn't interfere with dosing. Vomiting or gastrointestinal issues would also not interfere with dosing intranasally versus putting something in your mouth. We believe that this is a very—really almost an ideal way to administer a drug for this type of indication. Why is nasal important in this indication? Why is it really an unmet medical need as recognized by regulatory authorities? First of all, for the injection products that have been on the market, they've worked fine. They've been on the market for many, many years, over 35 years, and they're very effective. People don't like to carry them. Carriage is very important. You have to have the epinephrine with you. You don't know when you're going to get exposed to an antigen in most cases.

You need to have the epinephrine with you at all times. Carrying the product is very, very important. In the case of the auto-injectors, it's well documented that people carry it less than 50% of the time. Even when they carry it, they usually only carry one of the devices. They do not carry two. You need a second dose about 10% of the time. It is very important to carry two. neffy is much easier to carry. We're showing one of our options to carry it here. You can do other things, but this is an option that we offer to carry in a carry case. I have mine here. It fits very easily in your pocket, and it's relatively small. It's like half the size of an iPhone.

Very easy to carry, which is one of the problems that we're trying to solve for. More importantly, people don't like to inject themselves, and they don't like to inject their kids. Literature shows that about between 25% and 60% of the time, but roughly on average, half the time, people actually don't administer the auto-injector when they're supposed to or when they need it. They literally will go to the emergency room with the auto-injector and show up in the emergency room for treatment, but they have not injected themselves with epinephrine. In addition, people delay. Even when they do inject themselves, they wait. They wait till it's serious enough and wait till they really need to inject themselves. That's the wrong paradigm. That's not what doctors want them to be doing.

The ideal way to dose epinephrine is immediately at the first signs of symptoms you're administering epinephrine. That's the most effective time to administer when the symptoms are still just starting, and you can stop them very quickly and avoid later comorbidities, other adverse events that occur from the reaction itself. Having a needle-free option that's not intimidating, it's very easy to use, very easy to administer is a real benefit to the patient population who are needle-phobic, which is a large percentage of this patient population does not want to inject and hesitates to inject. Finally, the auto-injectors have issues with reliability. The new rule of FDA, which came out after most of the auto-injectors were approved, is that you should have reliability that the device would deliver an effective dose 99.999% of the time.

That means less than 1 in 100,000 could potentially not deliver an effective dose. neffy meets that criteria. It's the same device that's used for NARCAN. NARCAN has been well proven to be equally reliable. So both products are pretty much equal. And this product is super easy to use. It requires no training. NARCAN , as you know, is OTC, so no training required. In addition, we have better stability with neffy. Our formulation is designed to be a little bit more stable. In particular, at high temperature, we have a much greater advantage at high temperature. Our expiration is about 30 months, and our temperature stability is up to 122 degrees Fahrenheit. We actually stored it for three months at 122 degrees Fahrenheit, and it's stable for that period of time.

When we look at the population, just to give you an idea of how we're seeing the entire market, there are about 40 million people in the United States that have type 1 allergic reactions, either to food or venom or drugs. In our assessment, there's about 20 million that could benefit, that are severe and could benefit from epinephrine. They probably should be carrying epinephrine. Of them, there's about 6.5 million that are prescribed epinephrine. Not all of them fill their prescriptions, but 6.5 that are prescribed epinephrine. Most of those are with allergists and pediatricians. There's 13.5 million people that are typically not with pediatricians or allergists, only about 2%. They are not prescribed epinephrine in general. They have not received a prescription. With the people that do get prescriptions, the 6.5, 3.2 million fill it regularly. 3.3 million do not.

There's a large, about 50% of prescriptions do not get filled in this space. A lot of the time, it's because of the injection. Patients don't want it. The doctor prescribes it anyway because the doctor wants to cover any liability they have. If you're severe, they want to prescribe epinephrine. Even if you tell them, "I'm not going to fill it," a lot of doctors tell us they prescribe anyway because they want to make sure they have it on record that they gave you the prescription. We believe that the 3.2 million population that currently has auto-injectors, that we can convert a large percentage of those. We also believe that a large percentage of the 3.3 million would be more amenable to getting neffy because a lot of them are not filling their prescriptions because they don't want an auto-injector.

They don't want to have to carry it or they allow their prescription to lapse. Between the two of these, we believe that there's easily a market size of potentially $3 billion at our net. In addition, we believe that some percentage of these patients out there that are not aware of the need for epinephrine, they're not visiting the right doctors. A lot of them went to an emergency room, were diagnosed in an emergency room, and left and never got medical help. We believe a lot of those patients are also potential to bring into the market and to really expand this market beyond that. Beyond this, there's also institutional use for epinephrine, which is not well adopted with auto-injectors because of the need for training and the risk of the injection device. With neffy, there's no risk.

There's no risk of self-injury with the device. And it's much easier to use. You can imagine that neffy can be used in public places, in restaurants, other public by first responders that are not allowed to use injection, or even in airplanes and other public transportation. We believe that there's even a bigger market for neffy in the future in those types of areas. We've been very successful at partnering neffy outside the United States. ARS is focused on the U.S. population, U.S. market. ALK is our partner in Europe, U.K., and Canada. They also have other territories around the world that we license to ALK-Abelló. Some of them are sitting in the room. They're doing a very good job. We expect launch of the first neffy, EURneffy it's called, in Europe.

EUR neffy, we expect to be launched possibly later this month or early July in Germany. We're also expecting approval very shortly in the U.K., which is one of the largest markets in Europe. Germany and the U.K. are the two large markets, and then expanding out from there. We've also filed in Canada and expect approval later this year in Canada as well. In addition, China has been submitted. It's based on the U.S. approval. That also, we expect to see approval in China later this year. There is no auto-injector approved in China, by the way, so there is no community-used epinephrine today in China. neffy will be the first community-used epinephrine available in China. Again, in China, our understanding from some of the history of this is that the needle was always an issue for the Chinese regulatory authorities.

They've never approved an out-of-hospital use epinephrine product with a needle. Japan is also pending. We anticipate approval as well in Japan later this year. It's a big year for neffy worldwide. Australia is licensed to CSL, largest pharmaceutical company in Australia. They expect approval possible later this year, but probably sometime first half of next year in Australia. Eric, you want to—yeah.

Eric Karas
Chief Commercial Officer, ARS Pharmaceuticals

First, Rich and I appreciate everyone being here. We know that we're competing with the FDA commissioner. That really makes us feel special that you chose to spend some time with us. I'm going to go through just a commercial update. I think everyone knows that we're at month nine. When I think about a launch, it's not only the first six months of your launch, but it's really kind of phases.

This next six months is going to be critical for us. We have a lot of catalysts, and we're going to go through this in a very exciting time for the brand and the company. When we think about kind of commercialization right now and beginning this year as we put together a strategic plan, there's key areas that we are focused on. The first one, a lot of this is obvious, is we really want to make sure that we're driving adoption and we're educating prescribers. We want to make sure that they have confidence in the product. We want to make sure that they have trust in the product. The next thing that we wanted to do is we wanted to make sure that we remove barriers for physicians and patients in terms of achieving access.

At this point, we have 92% coverage in the commercial space. I'll get into a little more detail. The nice thing about that too is when we look at our contracts, we're maintaining a gross to net, so retention of greater than 50%. That's really, really strong for a product like this that's going into obviously a generic market. As we look at kind of now into the second half of this year, we want to engage consumers more because we know from our market research when a customer sees an ad for neffy or information about neffy, there's a high, high likelihood that they're going to go to their doctor and they're going to ask for it.

When we look at our market research from a physician perspective, we know that a majority of the time when a patient asks, a physician will fill the prescription. I'll get into a little more detail there as well. Rich mentioned, and I'll speak to this, but we recently announced a co-promotion in the pediatric space, and I'll get into the details there. A lot of catalysts, a lot of tailwinds here as we go into the second half of the year. Specifically, when we look at targeted prescribers, our sales force, we've got 95 sales reps. We have 11 managers, and we have a virtual team of 10. We've been focused on about 12,000 physicians, which account for about 45% of the epinephrine prescriptions that are currently written. These are all the allergists. These are your high deciles, eights, nines, and tens.

We have engaged over 10,000 physicians. Launched to date, about 5,500 of those physicians have prescribed neffy. We are continuing to drive share. We also want to drive broad utilization amongst the prescriber base as well. As I mentioned, about a month ago, we announced the co-promotion with ALK. This allows us to get to another 8,000 pediatricians. It allows us to increase the footprint of our sales team to now cover 20,000 physicians overall, but 55% of the market. This is an ideal time because when you think about everything that is happening with 1 mg availability about a month ago, our coverage continues to strengthen quarter- over- quarter. We are going into the back-to-school season, which the next four months starting in June account for about 45% of the overall business.

Launching the DTC, this is a really great time to kind of expand our footprint. We are very excited about that. That team was actually trained the last two weeks. I know most of the team is in the field this week. They are starting to make an impact immediately. On the other side of the slide here, I talked a little bit about building trust and confidence. This is a program that we launched with. It is called neffy Experience. We know that doctors said, "Listen, I have these oral food challenge clinics. Sometimes a patient may have a reaction where I need to use epinephrine. It would be great if I could use neffy in those clinics." We provide doctors with product just for the clinic. These are not samples to give out to patients. This is working out nicely.

We have about 2,500 physicians that are enrolled in this program. We did a survey recently. So we've got data on about 600 patients, which is very strong. In the second half of this year, we're looking to get more of that data out, whether it's being published. We're going to run another survey because we're extending this to the 1 mg that we have available as well. Most likely, we'll have a poster at the college meeting in November. There are other case studies that we've also gotten back from doctors where patients have had an experience in the office. We had one patient that was unconscious. They were given neffy within a couple of minutes. They were revived, and they were better.

We want to make sure that we get that information out into the literature because we do know, again, that builds that trust and confidence. We want doctors to say to patients when they say, "Hey, how do you know? Have you seen this work?" "Yep, I've seen it firsthand in my clinic," or, "There's a lot of literature now documenting that." That's part of that strategy of really building that trust and confidence amongst physicians. Now, when we look at kind of uptake for this year, we're seeing continued trends in market research. Kind of on the left-hand of the slide here, this is information that we've gotten in some of our market research. This one was back in December. Some of the common things that you would expect in a launch as a potential barrier for a physician.

This is nothing unusual that I've dealt with, not dealt with in other launches and other products I've worked on. Cost and coverage is about 40% of the time. They want to get clinical experience. As I mentioned, and I'll get into this in a little bit, our coverage quarter- over- quarter has increased. We're in a really good spot right now with commercial coverage. We're continuing to reduce those barriers. Clinical experience. Doctors are getting more and more experience through our experience program. We're also doing a lot through education at conferences. We're doing peer-to-peer education too, where most of our promotional speakers are part of that experience program so they can kind of share firsthand experience, what they've seen in their clinic with doctors attending those programs.

Now, we wanted to also do a baseline ATU that we did in March before we started our DTC campaign because it is geared and targeted towards we have a component towards healthcare providers as well. It was interesting, really favorable numbers here. Two out of three physicians said they would offer neffy to their patients today. We know when a patient asks for neffy, 9 out of 10 times, the doctor will prescribe it. We also asked physicians of the patients currently that have an auto-injector, what percentage of them a year from now will be your preference share? That was at 51%. These are very encouraging numbers. We are continuing to trend in that direction with the performance that we have seen over the last couple of months.

Now, specifically on coverage, one thing I'll highlight first on the left-hand of the slide there is that we have a Co-Pay program that buys our Co-Pay down to $25. This has been very successful because we really wanted to take cost off the table. And this really can help a majority of patients out there. Right now, when a patient, the average for a filled generic auto-injector is about $42. So again, this program has been really received quite well from the advocacy community, from patients and physicians. But as we were talking about coverage, right now on the commercial side, which is about 62% of the overall business in the epinephrine market right now, we have 92% commercial coverage. And if I break that down, it's really three components. It's coverage with no PA.

It's coverage where you have to submit a PA, but there's a high likelihood that you would get that approved. There are restrictions. If you break that down, it's 57% coverage with no PA, 35% with a PA, and only about 8% where it's restricted. Our representatives now are going in and having conversations with healthcare providers and saying, "Hey, if you write five prescriptions this week, on average, three are going to be covered. Two are going to need a PA." The PA is pretty simple in this category as well. We have best practices that we've really been able to gather over the last couple of months in the early days of launch here that the representatives have those best practices and are able to share that with their healthcare providers and the personnel in those offices that handle the PAs.

We are seeing a success rate of PAs of about 65%. We think we can continue to grow that number, but very encouraged by the commercial coverage at this point. Now, the other component of this, it's kind of convoluted and complex with the PBMs, but above them, there are GPOs. With all the GPOs, Ascent, MSR, and Zinc, we do have contracts in place. If you look at Ascent, 89% of their payers are covering the product. With MSR, it's 88%. With Zinc, it's 97%. Our contracts, when we negotiate, we're only offering rebates where the product is covered without any type of PA or any type of restriction. The nice thing about our contracts too is we're able to, and this is trending, we're slightly above this, but we'll be above 50% in terms of gross to net retention for our contracts.

Again, really in a strong position from a payer and contracting standpoint. Now, the last couple of slides that I'll hit here before I turn it back over to Rich is consumer awareness. In the plan originally, we were going to do a DTC campaign late this year and probably kind of launch it more in January of 2026. As we looked at the other strategic imperatives that we had of driving adoption and hitting access, we really felt back in December like we should accelerate this. We actually filmed the commercial back in April. We launched the DTC campaign about two weeks ago. We also did a baseline ATU because we wanted to see what is consumer awareness at this point.

We're going to measure this on a monthly basis to really see the impact of our DTC campaign because we want to know specifically, did you see it? What did you do? Did you make an appointment? Did you ask for a prescription? Did you get a prescription? What we found, though, in this study most recently is only about 16% of patients unaided were aware of neffy. That is on the low side, but it creates a huge opportunity for us and really, again, supports the value and the opportunity we see with DTC. We also know that 81% of caregivers are likely to ask their healthcare provider about neffy after learning about it. A little bit of exposure to the product profile information, and that's what we're doing with the campaign.

Richard Lowenthal
President and CEO, ARS Pharmaceuticals

We see a pretty significant opportunity here with parents and caregivers to go in and ask for this product. I mentioned the last stat already, 9 out of 10 doctors will fill this when a patient asks for it. Here is the campaign. I do not know, by a show of hands, have people seen it? Okay. Only about a third. I got to do a better job, Rich, of getting it out there. Yeah. The campaign is Hello neffy, Goodbye Needles. I will tell you, back in probably January, we tested for creative concepts. We did it with consumers, parents, patients, physicians. This concept, Head and Shoulders, scored the best. I mean, second and third was not even close.

Really, what this is about is saying goodbye to all the challenges, saying goodbye to anxiety, saying goodbye to fear, saying goodbye to hesitation, and hello to a product that you can incorporate into your life that's easy. When you talk to patients, they want freedom. When you talk to parents, they want peace of mind. That's what neffy provides to them. We set this up in a way where it really showcases everyday people integrating it into their lives. There's a young lady there. She's out to dinner in one of the scenes with friends, a college student, a middle school student in the morning getting ready for school, and then a birthday party. All around community and food, right? You shouldn't be afraid. You shouldn't have that anxiety, right? You can easily be prepared with neffy. Again, Hello neffy, Goodbye Needles.

We also have a jingle on this. I do not know if, again, the folks that saw this tested extremely well. It is that song that you often hear at sporting events of, "Na na na na, hey, hey, hey, goodbye." It is a great song. It is very catchy. I am making Rich smile up here. That is great. That really tested well too because when people heard that, they remembered it, right? It really kind of triggered them to say, "You know what? I want to go and do something. This is going to help me or it is going to help my child." The investment here is pretty significant. From May to the end of this year, we are going to invest about $45 million.

This is going to be multi-channel over connected TV, broadcast TV, social media, traditional things that you see online with search and banner ads, print, point of care at pharmacy, also at doctors' offices too. We'll have wallboards, and we have them up already, exam room, the monitors with information about the product and the commercial. In the early days here, let me just show a couple of numbers. We launched this May 15th. I can tell you on YouTube, we have the video. We have Google Ads driving to the content. I checked putting these slides together last week. We had a million views in just around 17 days, which is really, really strong. When we look at that information too, a view is only counted when a consumer watches it for 80% or more. If they click through after five seconds, it doesn't count.

We're able to track these patients too. Where do they go? Do they go to our website? Do they click on something that's considered a high-value action? We're able to reserve ads up to them as well. Another big component of this is connected TV, online TV. Everyone's familiar with those platforms. In the first two weeks, we delivered 8 million views impressions to households that kind of match our indexing for our target. We did a lookalike model of, "Okay, let's take a family that has children that are affected, middle-income kind of commercial insurance, and then we apply that to the general population." We're able to kind of target our efforts on those households.

We really, really wanted to get to those families, those moms that are the decision-maker because over the next couple of months, as they're going into their summer appointment before school starts in September, the mom is going to be the one, in a lot of cases, making that decision. Website traffic is up, broadcast as well. We've started advertising on CNBC, Fox News. We're going to be expanding that onto broadcast television, other cable networks. This past weekend, we actually sponsored the National Spelling Bee. We had sponsorship on the Countdown Clocks. We had our commercial. We got over 11 million impressions in just about a week. From a social media perspective too, we have influencers out there that are creating content for us. We're using them to really kind of amplify what we're doing from a DTC perspective.

Get information out there in the brand, but have other folks carry that message to their followers as well. This is just the start. This is two and a half weeks. This will continue, and it'll continue to build over the next couple of weeks as we expand out into other places on connected TV and traditional broadcast and cable. I think with that, I'm going to turn it back over to Rich.

We'll try to finish up quickly because we're out of time. As you could see, I think we have got a lot of tailwinds going into the peak season of the year because summertime is where a lot of the children are going back to get their prescriptions for the year, going back to their annual appointments with their allergists. It's really a peak time of the year.

We have our 1 mg that's launched now and available. That adds about 23% to the market size for us because that's about what it represents as far as the number of scripts. Our broad direct-to-consumer campaign is starting, and we're hoping that you'll start seeing much more of that over the next couple of months and leading into the summer peak season. Again, commercial coverage is important. We're still expanding that. We're trying to work through those companies still requiring prior authorization and those companies, a handful of companies, but those companies that are still blocking and requiring medical exceptions, which we don't want to see. Expanding our U.S. sales force via our partnership with ALK, as we've discussed.

We believe that really coming into this period right now, it's a very critical period for us where we believe that we can not only increase our market share significantly, but going into the peak season really drive revenue and drive prescriptions through the summer period. In addition, we are still working very hard on our urticaria program. Just to give you a snapshot of that, this is data from our phase I study. It shows initial phase II study really was in patients, placebo-controlled, but in an in-house, in-clinic study where we really showed very rapid resolution of urticaria symptoms. The target here is really on flares. It's not on chronic treatment. We're not talking about treating people every day with epinephrine. We're talking about when they're on antihistamine or on XOLAIR, they have an exacerbation, a serious flare, they call it. This is a very, very serious outbreak of urticaria. It's very itchy, hives. A lot of people go to the emergency room because of this to get IV antihistamine or steroids. Epinephrine, especially nasal epinephrine, is exceptionally rapid at resolving these symptoms. We're now initiating our phase II-B study, a controlled phase II-B study in an outpatient setting. That will test neffy in an outpatient setting with self-assessment by the patients. It will be a three-way randomized crossover study. Everybody will take either two doses of epinephrine nasal spray or placebo. They'll be compared not only to cross groups, but within themselves. It's a very powerful study. Everybody treats themselves three times, and you could really see a difference. We expect to see results early next year and then start a phase III

We think this is a potential $2 billion-$3 billion additional market in this area. Really, we think that we've had a very strong launch so far. This is a very critical year for us to really accelerate adoption of neffy and really drive the launch into this summer and into the next year. In 2026, we just believe we will continue to grow neffy into a market leader. Obviously, we believe that we have potential for multi-billion dollar peak sales with this product going into the future years.

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