So, good afternoon, everyone, and thanks for joining the ARS Pharmaceuticals presentation. I've got Justin Chakma here, who's Chief Business Officer of ARS, and you and Kathy Scott, CFO. And you have a recently approved new nasal product for epinephrine. So maybe you can go ahead and give us the presentation, and we'll leave some time for Q&A.
Sure, great. Yeah, thanks, Annabel. So, good afternoon, everyone. Again, Justin Chakma, Chief Business Officer at ARS Pharmaceuticals. We're focused on developing or commercializing now neffy, which is the first and only needle-free epinephrine product approved for the treatment of severe allergies. I'll be making forward-looking statements, so please refer to our latest 10-Q that we filed last week with the SEC for further details. We're focused exclusively on commercializing neffy. So, neffy is a nasal spray that delivers systemic levels of epinephrine comparable to an injection. neffy was approved in August by both the FDA and EMA, and we're about one and a half months into a very exciting launch here in the United States.
We believe neffy's unique profile has the ability to, one, get rid of the needle-related safety issues that are associated with autoinjectors, and also reduce the anxiety and hesitation that leads to delays in treatment and even patients refusing to fill the prescriptions for epinephrine autoinjectors. We think that the unique profile allows us to unlock a multi-billion-dollar opportunity in the United States, especially given our long period of expected exclusivity. We have seven Orange Book-listed patents, composition of matter, and method of treatment that expire 14 years from now, so in 2038, and we're also very well-resourced with a pro forma cash position of about $350 million U.S. dollars, including the $145 million upfront ex-U.S. licensing deal that we just announced with ALK last week.
The fundamental clinical problem that neffy solves is delays in treatment with epinephrine, which are statistically associated with worse clinical outcomes, worse vital signs, increased risk of biphasic reaction, increased risk of emergency room visits, hospitalizations, as well as increased risk of fatality. And 80%-90% of the patients that currently even fill their epinephrine prescription are either non-compliant or dissatisfied. They're not carrying their device. They're not using the device or delaying using the device. And neffy solves all these issues with its small, needle-free, easy-to-use, reliable format that takes away all the anxiety and hesitation, giving patients peace of mind to be able to dose epinephrine at the first sign of an allergic reaction. We believe neffy's profile unlocks a multi-billion dollar US market opportunity that's currently unavailable to the currently approved injectable devices.
There are currently about 20 million patients that were diagnosed with severe allergies in the last three years per IQVIA claims data. We believe all of these are eligible candidates for epinephrine, but only 3.2 million of those patients actually fill their prescriptions. And as I mentioned, 80%-90% of those are dissatisfied or non-compliant. In addition, there are an additional 3.3 million patients that received a prescription in the last three years, but either didn't fill or didn't refill their prescription, largely due to device limitations. The top three reasons are needle size, portability, and of course, neffy solves all three of those. And so that represents a potential doubling of today's market opportunity with injectables. All these 6.5 million patients that are prescribed epinephrine, the vast majority of them rather, are seen by allergists and pediatricians that our sales force is directly calling upon.
There's an additional 13.5 million patients that have been diagnosed with severe allergies in the last three years but haven't received an epinephrine prescription. And we know that the vast majority of those are not with allergists and not with pediatricians. And so these are with doctors that are not as well-educated about treating severe allergies or treating anaphylaxis. And so we'll be reaching out to those patients and those doctors who are currently relying on antigen avoidance or emergency room visits or incorrect use of over-the-counter antihistamines through a couple of methods: continuing medical education, disease awareness campaigns, as well as direct-to-consumer marketing. And all three of these segments say that they've got more devices relative to injection. And the reason for that is because of its small and needle-free, easy-to-use format. It fits into patients' lifestyles better.
And so they're more willing to carry it if they're more willing to use it. They're more willing to leave it in more places, especially with additional caregivers if they have children that have allergies, with a babysitter or a grandma whom they may not have previously felt comfortable with leaving an autoinjector with. You might wonder what neffy looks like. So actually, here it is. It fits easily in your pocket. We're offering it in a carrying case, so you can carry two sprayers per treatment guidelines, because about 10% of the time you need a second dose, irrespective of the epinephrine device, in order to get resolution of allergic symptoms. It's needle-free, obviously. It doesn't require any priming. You simply insert and press, and you get drug almost instantaneously into the systemic circulation.
The magic of what makes neffy possible is a novel absorption enhancer called Intravail. Intravail allows neffy to deliver systemic epinephrine levels at a safe, low dose. The 2 milligrams is equal to 0.3 milligrams injection. Importantly, within the safe therapeutic window established by the exposures of the approved injection products. They give neffy a single dose or a repeat dose during 10% of the time that you need a second dose, or during an allergic reaction when you might have increased mucosal permeability that causes increased absorption of epinephrine. All of the exposures of neffy are within the range of the approved injection products. It's, again, established to be very kind of safe.
In addition to being able to deliver epinephrine systemically, neffy also, importantly, does so without any meaningful pain or irritation, which we've measured by formal scales in our clinical studies. It's important because half of this population is a pediatric population. So kind of tolerability, palatability are kind of very important considerations. These unique attributes of Intravail are what actually give rise to our very strong intellectual property position, the composition, matter, and method of treatment patents that protect nasal sprays that deliver low safe doses of epinephrine, like neffy. The unique profile of neffy was recently recognized by a significant partnership that we signed with global allergy leader ALK. ALK is the largest pure-play allergy player in the world, with a market cap of almost $5 billion and north of $800 million in sales, mostly in Europe.
And we've licensed them the rights to commercialize neffy in Europe, Canada, and other unpartnered regions. ALK shares our conviction of neffy's potential. They publicly have guided to peak sales in anaphylaxis alone in these regions of about $425 million U.S. dollars. And so that represents more than a doubling or tripling of the current sales of epinephrine injection devices in these licensed regions. By partnering with the rest of the world, this allows ARS to exclusively focus on commercializing the U.S. market, which today represents about 80% of the global net sales of the epinephrine autoinjectors, and likely north of 90% of the actual cash flows. And that skewing of value towards the United States is going to be even more skewed for neffy, or even more pronounced for neffy, rather, because of our expected reimbursement and pricing.
And the $145 million upfront gives us significant flexibility to invest into the U.S. opportunity and really accelerate the adoption of neffy. We're also making good progress on the clinical and regulatory aspects of neffy, with the one milligram dose for the 15- 30 kg children recently accepted for priority review by the FDA, with a PDUFA date of March 6, 2025. And so that lines up nicely for back-to-school season with both the one milligram and two milligram doses available, also all dosage forms for children, and as well entering a time when we expect to have a majority of commercial coverage in place. We also have additional regulatory filings and commercial launches in the coming months, some of which trigger additional near-term milestones that will further bolster our balance sheet.
We'll be initiating a Phase IIb study in chronic spontaneous urticaria patients that are currently being treated with antihistamines or are still experiencing acute exacerbations that neffy might be able to treat. We believe that this follow-on indication has the potential to be a blockbuster indication on its own right. Turning now to updates on our launch and commercialization efforts. What we're seeing early on the launch is actually kind of very promising. The mix of patients that we're seeing early in the launch is actually very consistent with our market research. The mix is well-balanced between patients that are currently filling their epinephrine autoinjector scripts but are dissatisfied or non-compliant, as well as patients that have either refused to fill or refused to refill their prescription.
And so I think seeing this kind of good representation of all the different patient segments this early in the launch bodes well for the long-term market potential of neffy. Our market research also indicated that over time that neffy would represent a vast majority of HCP-initiated prescribing of epinephrine. And to this end, we've seen very strong interest with hundreds of physicians and allergists at the recent American College of Allergy, Asthma and Immunology Conference in Boston attending neffy-related presentations. And so the reception has been very, very positive from the physician community, consistent with the market research results that we shared previously.
To build on this early promise, our first kind of near-term commercial strategic imperative is to ensure and promote awareness and education of neffy among physicians using our sales force so that they completely understand the neffy data and, importantly, feel comfortable prescribing neffy, especially if asked unprompted by a patient for neffy. Our sales force has been out in the field for a little more than a month. They visited about 5,700 of the 12,500 targets, and about a third of the visited doctors have already prescribed neffy. And so I think that's highly encouraging to us this early in the launch, only about five or six weeks in. This strong interest from the healthcare providers is also reflected in now more than 1,200 healthcare provider offices that are now participating in and received drugs for participating in the neffy Experience Program.
And this is a program that allows healthcare providers to get first-hand experience trying neffy as rescue therapy for anaphylaxis symptoms that might develop during allergy challenge in the clinic. And so in addition to the robust PK/PD data of neffy, having this first-hand clinical experience may allow physicians to tell their patients that they've tried neffy first-hand, that they've seen their work the same as injection, and that may also further accelerate adoption. And so in addition to the HCP visits, as well as the neffy Experience Program, we also have underway a wide suite of continuing medical education, speaker programs, and peer-to-peer education programs to ensure that we have the complete surround sound to help keep neffy top of mind for these healthcare providers. Our second near-term commercial strategic imperative is to ensure broad market access for neffy.
While we've seen excellent success rates in first-pass prior authorization approvals facilitated largely by our support services program, neffy Connect, I mean, the ultimate goal is to have a seamless patient experience without any additional paperwork required on the part of either the HCP or the patient. The patient can simply walk into their retail pharmacy, pay $25 or less copay, and get their neffy. We're well on track to meet our commercial coverage goals, which is north of 60% by month six and north of 80% by month 12. We have contracting underway with all the key payers, including the three largest ones, and we expect initial coverage decisions by year-end.
Over the coming months, once our HCPs have been visited by our sales reps, we have the 1,000+ HCP offices that have gotten first-hand neffy experience under their belt, and we have a seamless insurance experience for the vast majority of patients with commercial coverage. We're going to press the go button on our consumer marketing efforts, and that's going to begin with first celebrity-driven public service announcements in partnership with FARE, which is the largest food allergy advocacy group in the United States, followed shortly afterwards by our own celebrity-driven direct-to-consumer marketing campaigns, and our almost $350 million pro forma cash balance gives us significant flexibility in terms of investing into these consumer marketing efforts. We know that when a patient asks for neffy, 99% of the time the doctor will prescribe neffy if the patient asks for it.
But we want that patient experience to be seamless, both from a healthcare provider point of view, so that the physician understands the neffy data, maybe even has first-hand experience using neffy through neffy Experience, but also from an insurance point of view, again, so that once a physician puts their prescription through the EMR, that the patient can simply go to the retail pharmacy without additional paperwork and pay $25 or less to get their neffy. And this will be the case for a majority of patients with commercial coverage in the next few months, starting in the middle of the first half of 2025. So we're highly encouraged by the trajectory of the neffy launch so far, which is actually in track or in line with our own internal forecasts that track towards neffy capturing a lion's share of this multi-billion dollar U.S. market opportunity.
Over the coming months, we're excited to share additional updates, especially as we fully embrace and activate the consumer-driven demand that we anticipate for neffy, especially going into the peak summer season, where we'll have both the one milligram and two milligram doses commercially available for children of all ages. We'll have commercial coverage in place for a majority of patients, and our consumer marketing efforts will be in full steam, and so we look forward to sharing additional updates on the neffy launch as we progress, and we're happy to take any questions.
I've got a couple, actually. Related to coverage, do you have any anticipated gross to nets that you're assuming and how that's going to evolve over time, and any kind of, I think you have unrestricted access there, so they don't have to step through any kind of epinephrine prescription before they get to the neffy.
Now, Kathy, do you want to take that?
Sure. So gross to net, we are targeting 50%. All of our payer contract negotiations to date have indicated that we will be successful in that target. So I think that's reasonable to be modeling that from a financial point of view. And your second question was?
Related to any need, I think you have unrestricted access there, so you don't have any step there.
No setback. We're currently working through prior authorizations because not all payers are currently covering that, but those will decline over time as we get our payer coverage.
Okay. And can you talk a little bit about your patient assistance program and who that is geared to? Do you expect it to be for just during this period of time, or is there always going to be some kind of patient assistance program for the cash pay business?
We expect to have the PAP in place throughout the duration unless something changes. It would be targeted towards folks that need financial assistance and cannot afford our product because we want it to get in the hands of everyone that needs it. In terms of volume, we expect that to be kind of in the single digit of our total units that are sold.
Okay. Great. And then can you talk about IP a little bit and the competitive landscape? There's, I guess, a couple others that are developing inhaled epinephrine as well. So I just want to understand the competitive landscape for you.
Sure. Justin, you want to take that one?
Yeah, sure. So just to talk about IP first, so our IP covers composition of matter, which is the combination of epinephrine plus Intravail, which is the novel absorption enhancer that I mentioned. And so that blocks kind of any generic up until 2038. And we have a second layer of patents that are related to method of treatment, so treatment of type 1 allergic reactions, including anaphylaxis. And those cover other nasal sprays that are what we consider to be low dose, right? So neffy is two milligrams, that's considered to be kind of low dose. And the reason why low dose is important is because there's a therapeutic window for epinephrine. I think four milligrams by injection is considered to be the minimally lethal dose in the literature. And so it's cardiotoxic if you kind of go too high.
And so we think that that poses a kind of important regulatory barrier or the combination, the patent plus the kind of fact that there's a therapeutic window, and we were the first to be able to show that with nasal spray, you get injection-like absorption with a low dose, that that, again, poses a kind of structural barrier to potential competitors since our IP blocks other low-dose formulations. And there's not really any reason from a risk-benefit point of view for the FDA or a regulator to approve a higher dose epinephrine product. Regulators usually don't do that. And so we feel pretty, again, get very comfortable with kind of the competitive situation with respect to nasal sprays. And then there are other nasal sprays kind of in development that, using different technologies, powders, and whatnot, that are able to achieve lower dose, not as low as neffy formulations.
But the problem with all those nasal sprays is that neffy's adverse event profile is very benign, 9.7% rate of mild nasal discomfort, again, not correlated with pain at all, just some people don't like having something up in their nose, 6% rate of mild headache, again, makes sense given that you have increased systolic blood pressure as a physiological mechanism for epinephrine. And so I think that from a tolerability point of view, I mean, that's something for this pediatric population that neffy is served. We have not seen a safety profile that comes anywhere close to neffy. And so I think that from a competitive point of view, I think the bar is quite high in terms of what neffy sets.
Okay. So when you think about this type of administration, how, I mean, right now we saw the numbers on the population. Can you talk about how that might expand prescribing? Do you see this enlarging the market, or just are you going pretty much after only that population that's being prescribed right now?
Yeah. So as I mentioned, we're already. There's about half the population that is visiting the doctors we're calling upon. And the half of the population that is getting the doctors telling them, like, "Go get an epinephrine prescription," they're not filling for a variety of reasons, right? Largely, as I mentioned, due to needle size, portability, some cost issues. They're not filling the prescription. And so we're already directly calling on those doctors that are prescribing to those patients. And so those patients, when we have done market research surveys and we ask them, "Hey, neffy is now available as a new treatment option," right? The vast majority of them, 70%-ish, say that they would go and ask the neffy or ask the doctor about neffy, or they'd go and fill the prescription.
And so just by virtue of kind of neffy being out there and not having to target or kind of go into that broader population that our reps are currently not calling upon, we should see some sort of, we expect to see some sort of market expansion with respect to the patients actually choosing to fill their epinephrine prescription, but now with neffy, whereas they would not do that with EpiPen. And I think that, as I showed with the numbers, that's about a doubling of the current number of kind of addressable patients. And we're already seeing that early in the launch, right?
That there's a kind of nice balance between kind of patients that are already filling their scripts and people in scripts but don't like the EpiPen, so they're switching, as well as patients that are not filling or refusing to fill the EpiPen scripts, now saying that, "Now neffy's available. I'll actually carry this. I'll actually use this, so I'm going to fill this prescription." And so I think we already are seeing kind of early signs after only six weeks in, right? But just in terms of the patient segments that are being represented amongst the patients that are currently getting neffy, you're already seeing that expansion segment coming into play a bit.
Okay. Can you talk about the infrastructure that you have, the sales support required for a launch in this space?
Yeah. So our sales force, which is our sales organization, is about 118, which includes the reps, virtual reps, ASMs. And that gets to about half of the total prescriptions in the United States that are being prescribed by doctors for epinephrine. There's sort of a longer tail of kind of primary care doctors and other specialists that prescribe epinephrine. And we won't be calling on those doctors directly. I mean, that's not an efficient way of reaching those doctors. We'll be getting to those doctors through, again, continuing medical education, through direct-to-consumer advertising on the patient side of things, as well as disease awareness campaigns. Opportunistically, maybe over time, we may kind of gradually expand our sales force slightly, but it's sort of a very long tail. And so there's sort of very high kind of, if you will, diminishing returns.
We believe that there's more efficient ways to reach out to that kind of broader segment of patients and doctors.
Okay. Got it.
I put up one slide earlier. If I saw it correctly, it said 66% of the doctors would recommend neffy. What is the reason for not? Is there any specific reason for them to shy away from it?
So I think that, so you're referring to this middle figure here, right? 66% of the time. So you can think of that as kind of like share, right? So 66% of, if you will, patients, they would like, I mean, so I think that doctors see that, and it's a couple of things. So one is that there's certain doctors that, as in any market, they're kind of fast adopters, right? Slower adopters and very, very slow adopters, right? Because they're 10%. And we see the same segmentation in our physician research. And so there's some doctors that are saying that, like, I'll wait for my peers for a few years, like 10%, to see how this plays out before I switch. So that's kind of factored into that.
And then if there's doctors that have patients that they see are kind of comfortable, which they are, right? 15%-20% of the patients are fine with EpiPen, carrying it, using it, right? Why rock the boat, right? They're already compliant, so there's no need to kind of encourage a switch. And so I think those two segments is what you're seeing in kind of that 30%ish that's being reflected there. But I think that this is the HCP side of things. And when you go back to the patient side of things, right, from a patient perspective, like the demand is much, much higher. And just because the physicians don't know, necessarily know that their patient is not carrying, not using, right?
They just prescribed epinephrine, and they may not even know whether the patient fills or refills their prescription, which is why there's this kind of half that is sort of out there without a prescription, even though the doctor told them to get one.
Okay. Can you talk about the cost relative to the EpiPen? Is there any kind of cost pushback on that at all?
Do you want to cover that, Kathy?
We have a cash program currently of $199. So if you don't have insurance or if you're not covered yet, you can walk in with a prescription, pay $199. If you do have insurance coverage, we have a copay buy-down program, so we'll buy it down to $25 maximum is the maximum copay. Generic autoinjectors, the average copay is actually $40. So we're trying to be a lower-cost alternative. So besides all the actual other benefits, we're trying to make it very economical.
Great, and I don't suppose you have provided any guidance for first-year sales at all?
Not at this time. We're pretty early. We do expect to do that over time, but I think that a lot of people are just tracking IQVIA data and extrapolating out what they think the curve looks like.
Okay. Great.