Eton Pharmaceuticals, Inc. (ETON)
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The Citizens JMP Life Sciences Conference

May 13, 2024

Sean Brynjelsen
CEO, Eton Pharmaceuticals

Let's hit. All right, well, thank you. I'd like to thank JMP for the invitation to be here today, and exciting to speak about what we do. So let's get into it. We'll skip this guy. The company is a rare disease-focused company, and by that I mean really ultra-rare disease. The definition of rare disease by the FDA is 200,000 patients and below. We typically focus on 10,000 patients and below. So the reason for that is a couple of fold. One is that nobody else is doing it. Second reason is there's over 5,000 rare diseases with little to no therapies. And we find that the FDA is pretty good. We can work together with them. They want to get these diseases addressed. One of our products has only 80 patients. It's one of our most profitable products.

So you might think, well, not a lot of patients, not a lot of profit. It's actually the opposite. Lower patients, more profit, more revenue. And it works really well for a small company with a very low SG&A footprint. So we can acquire or develop an ultra-rare disease product, launch it, and field it with five salespeople, typically, because there are very few doctors that are treating ultra-rare diseases, so you kind of know where they are. So that's point one. Point two, we're commercial. We've got five commercial products, cash flow positive. We're going to be profitable by the end of the year. Very little debt. I don't like debt. I don't like issuing equity. I like to be profitable. It's funny, when we had no commercial products, our stock was trading at 10.

Now that we're making money and we're generating real revenue and solid, we're solid as a company, the stock goes the opposite way, but we'll get back. I think it's going to do quite well in the months and quarters to come. I'll speak a little bit about that later. Three late-stage pipeline candidates. One of them is our biggest product in our company's history, our ET-400, which I'll speak to, and is a product we're super excited about. Our short-term goal is to hit $100 million in sales. We did a little over $30 million last year. We're going to do a lot more this year, and next year should be really a nice breakout year for us. Then the other aspect of this is how choppy is the revenue? Do they go up? Do they go down?

Well, we go up 13 straight quarters of sequential revenue growth. And Alkindi Sprinkle will continue to add patients. It's growing consistently, but albeit slowly. However, the product we believe in as a precision dose supplement and therapy for patients that suffer from adrenocortical insufficiency. And then lastly, I think I said just a moment ago, I don't like debt, and I don't like issuing equity. We have a strong financial position, $16.7 million. It's actually going to be a little higher than that here coming up in the next coming weeks. But you get the idea. We're a company that is really built in a fiscally responsible way. We don't believe in taking excessive risks. And we'd rather do more products, a little bit less risky products, late-stage products, and products where we can see a revenue or profitability in the months to come.

All right, so what is ultra-rare? I touched on it on the last slide, but as I said, there's over 5,000 rare diseases, and FDA's definition of 200,000 and below, most of those are ultra-rare. That's a big point. We also like to go where there's a meaningful impact. And so if a disease is not being treated and we can provide the first or perhaps the second-line treatment for a given ultra-rare disease, well, guess what? Insurance reimbursement's not an issue. Especially when it's a life-threatening disease, you're not going to be dealing with issues of I mean, obviously, there's always some, but in the grand scheme of things, insurance reimbursement is a very minor part or concern or risk factor for the company.

The targeted commercialization of small patient populations is that we can use a team of 12 people, and they may be able to promote products for adrenocortical insufficiency. They could promote products for metabolic genetics. There's only and then eventually, as you get more and more products, you'll split the team. But you really are able to cross-promote products. And then the limited competition. You look at companies our size, well, the products we pursue are not interesting, really, to larger rare disease companies like Horizon or Recordati or some Pfizer products and even a variety of other players who are mostly focused on high-value one-off rare disease products. We're a dedicated company to rare disease, and I'm very excited about that. We do have you see in that box there, all of those are commercial except for ET-600.

That will be filed shortly, and that will be commercial within the next 12 months or so. The filing and the studies are going swimmingly well, and I think you'll see that product hit the market, which treats a disease called diabetes insipidus. So then let's go to the next slide here. Got to watch my time. So a little bit on our commercialization strategy. Strategy is designed to serve the unique needs of ultra-rare patient populations. We've broken out the country in the following segments that you can see color-coordinated. We have really made an effort to hire folks that are already in rare disease, already in that have the relationship. So if you look at a product like carglumic acid, that's technically a generic of a brand product called Carbaglu. And we have a lion's share of the market, mainly because of the relationships.

The fact that we go to these physicians and can speak to them and physicians know the sales rep is tremendous. And so that helps. The second is the advisory board on key opinion leaders. So we have a big focus on finding the main docs in these spaces that will champion your product, and then others will follow. So we do have KOLs. We do have advisory boards. We really pull out all the stops in terms of getting the key opinion leaders involved. And then our Eton Cares program is really. I haven't seen anything like it out there in industry. I've been doing this for more than 25 years, amazingly. And the fact is, if a patient gets a script, we ship. We don't go through the process. Big Pharma will say, if you can't afford your drug, we're going to help you to get your drug.

You jump through hoops. You fill out paperwork. You have interviews. You go through a long process. You might get your drug in a couple of months. You get our drug like the next day. We literally will ship. My goal here is, obviously, to run a fiscally, financially fit company, but the big goal, the main goal, is to get products to patients in need. So I was reading something recently, and it had to do with setting the right goal, which will drive the company and be to success. And there was a little example they used in this particular story. I won't go into all of the details, but they had two companies. One had set the goal to make money, and the other set the goal to produce the best product possible for their patients. And you can guess which one actually was successful.

So we realized our goal has to be patient-focused. We need to focus on getting as many rare disease orphan drug products to patients as possible, and the rest of it will take care of itself. We manage our business very carefully, so I don't want you to think otherwise. But we also have a very patient focus. We want to make sure we're making a difference in this world, and that drives everything we do, creates a tremendous amount of cohesion on the team. We have one of the lowest turnover rates I've ever seen, a couple in like seven years. That's pretty amazing. We focus on a concentrated prescriber base. A small number of specialists allows us for very efficient operations. The 12-person sales team is the right size today, but it may not be in the months to come as we do additional acquisitions.

We could expand that a little bit. But the takeaway on that point is that as we grow revenues, you're not going to see a commensurate growth with SG&A. The SG&A will grow slower than the revenues, and so you'll see additional profitability coming through that. And obviously, as a rare disease company, we use a specialty pharmacy to provide the wraparound services that patients expect. I talked about a little bit on our revenue, but this is good. We are going to increase that slope, though, in the coming quarters. You're going to see continued growth to the end of this year. I mentioned we're cash flow positive. We'll be GAAP accounting profitable by the end of the year. And we don't need to do anything rash. We don't need to take in a lot of debt. We don't need to go and raise equity.

It feels good to be in that position where you can make the right decisions. You're not making decisions for the short term. So what is going to grow us for the future? Well, three things. One, existing product line. We add patients consistently. We just launched PKU GOLIKE, which I'll talk about in a minute, PKU GOLIKE, which is, we think, a great product for the metabolic genetics space. New product launches. ET-400 will be our biggest product launch in our company's history. We're getting ready. That's been filed. We will launch that in the first quarter of next year. And that product will be priced very similarly to Alkindi Sprinkle . And so we think that patients will have optionality to use either product. We find out a very high discontinuation rate on Alkindi Sprinkle .

And so due to the texture, these are little granules that a child puts in their mouth. If any of you have children, you know that the texture issue, it's not always about taste, but the texture is not right, doesn't work well. The liquid version, which tastes great, great as far as medicine goes, let's just be clear on that. Maybe I should say it tastes a little bit like a cherry cough syrup. That's not so bad compared to the sprinkles. And when you're talking about an infant of one to two, the preferred dosage format is going to always be the liquid, whether it's liquid Tylenol or whatever it may be. Product acquisitions and licensing. David, our head of business over here, and myself are involved in doing a lot of M&A and licensing transactions. Virtually all of our products were either acquired or licensed.

We have a couple like ET-400 we did in-house. But the takeaway on that is we are actively engaged. We just bid $46 million about two weeks ago on a product coming out of bankruptcy called Zokinvy. We were able to line up that financing in seven days. So using a combination of cash on hand, a little bit of debt, a little bit of equity, but someone else came and wanted to pay more. And we didn't want the winner's curse. Well, you win the deal, but did you really want to win the deal at that price? So we'll be disciplined, and we're happy to walk away from a transaction because we don't need to do a transaction to be successful. We can be successful with what we already have on our plate. And then path to 10, I want to do 10 commercial products by 2025.

It might be slightly into 2026. We've got 5 down. We've got two more well on their way, ET-400, ET-600. That'll happen in the next 12 months. Those two will launch. Then ZENEO. I don't really count that as part of the 10. I think we really have to do three product acquisitions. The ZENEO autoinjector, which is like the EpiPen for hydrocortisone, that is a very large market, and it could make more money than all of these other ones combined. That ZENEO, though, it looks like it's going to be a 2026 launch. And that's a big one. So for the sake of time here, I'm going to only touch briefly on why Alkindi Sprinkle was even a product.

So imagine you have a one-year-old who suffers from adrenocortical insufficiency, and then the doctor says, "Well, we don't have any medicine for babies, but we do have the adult tablet. So why don't you crush that up, weigh it out, or go to a compounding pharmacist and go get a suspension? We'll figure out a way." So they were splitting pills, crushing pills, compounding. None of those are really optimum, especially when you need to get the right dose every day. If you're a diabetes patient, you know that you got to have the right amount of insulin. Too much, no good. Too little, no good. Has to be the right amount. The difference in this case is that the bad side effects would accumulate over a period of years, which would typically be like short stature. It would be precocious puberty.

It would be a lot of other types of side effects, which I won't go into, but there's more than a dozen. And so getting the right dose day in and day out, your child can grow to be pretty normal, really, with no discernible issues. But when you don't do that day in and day out, that's a problem. But doctors will typically overdose because underdosing has even more serious side effects. So they'll overdose the product, and that's what they did for years, and that child would grow short of the finish line in terms of their potential height. So this product is a standard in many European countries. We brought it into the U.S. We launched it. There's 5,000 addressable patients. We have less than 10% of that for a variety of reasons. The main is that people want the compounded suspension.

So that brought us to ET-400. Doctors said, "Look, why can't you guys come out with a liquid version of this? It would be so much easier." Pfizer tried it. They failed. They launched it in the 1990s. They pulled it off the market. They had stability issues. Hydrocortisone is not stable in water. And so I'm a scientist by background, just so everybody knows. I developed products for half of my career for Baxter and was now Pfizer. And I met with our team, and I'm like, "We got to be able to stabilize hydrocortisone in water." So through some internal ideas, we were able to come up with a viable formulation on ET-400 that's room temperature stable, tastes great, again, for medicine. It is a product that I'm very proud about, and we have filed this. We expect to launch in early 2025.

You think about the products that are sold for about $35,000-$40,000 a year per patient. We have less than 10% of the 5,000 addressable patients. We think we can get several thousand patients on ET-400 in short order. So that's a product that could really blow past the $50 million of combined sales that I've mentioned on previous calls. And we do have patents and all of that, so. Okay. PKU GOLIKE. This is a product I originally wasn't sure if I wanted to do, but it was such a good fit with our sales team. So we sell nitisinone, carglumic ccid, and betaine. That's all for metabolic geneticists. This product is ideal. So imagine you take a protein bar, a regular one, and you suffer from phenylketonuria, which is a disease that you cannot process the amino acid phenylalanine. There's 20 amino acids out there.

Phenylalanine is one of them. These patients cannot process phenylalanine at very low levels. So they need protein without that particular amino acid. And that's really hard to get. You're not going to get any kind of standard protein mixture, steaks, or anything like that. Everything has all of them. So PKU GOLIKE has all the amino acids you would need in a protein bar or a protein shake, but without phenylalanine. So that's why this is the only way that these patients can really get their protein needs met are through these types of products. You've got large companies like Abbott Nutrition that also has a product on this. The market's much larger than $100 million, and we're having a modest goal of 10%. I think that's modest because I've tasted the other products. This tastes great.

It was designed by a doctor out of Italy whose son suffers from this disease, which made it personal for him, and he wanted to make the best product possible. We've had tremendous feedback on this launch. I think it's going to do, and I think I said on the call, we should be able to approach $5 million in sales next year on this product. We'll see. I think the following year, it would not surprise me if we did $10 million. Picking up a product for basically $2 million is a pretty good payback. Then our metabolic genetics portfolio, these three are generics, but in the world of rare disease, you have to detail generic products. Pricing doesn't work. Contracting doesn't work. Insurance companies don't manage these little rare disease drugs, especially at this level.

So we found the detailing really to be the key to success. We're over 50% market share in carglumic. We have a significant market share in betaine, and we're gaining market share on nitisinone by just being in the offices talking, and prescribers are switching to our products. Plus, our Eton Cares program is well received. I never want a doctor to complain that their patient couldn't get insurance reimbursement. So we will, again, just ship. Ship. We'll worry about that later. We deal with the insurance companies after the fact, not before the fact. So ET-600 is a product we developed for a disease called diabetes insipidus. This was, again, a doctor's request. This condition impacts less than 5,000 pediatric patients in the United States. For those of you who are not aware of diabetes insipidus, it has nothing to do with diabetes. It's actually a frequent urination.

So a child might go, who has this disease, may go to the bathroom 20 times more than a regular child. So what this product does is it resets it, helps the child to have more reduced, vastly reduced needs to go use to urinate. The problem is the children are having trouble absorbing the water into their bodies, so it's just going straight through. So this drug basically allows that. And the problem is it's available as a pill, but the pill is not being well received, and there's a lot of noncompliance with little kids. So what we thought is, let's do the and then the doctors were trying to make their own custom suspensions. Let's just go ahead and leverage our formula we did for ET-400. This is also a water-sensitive drug. And we came up with this product, this ET-600. The NDA is literally done.

We just have to complete the bioequivalency study, which we expect to have done in the second half of the year. We already did a small bioequivalency study, so we know it's bioequivalent. That's not a question the product will do. I think the product will probably do like $20 million a year in sales. Our ZENEO hydrocortisone autoinjector, I'm going to say a couple of words on this and then move on. It's just like the EpiPen, but for hydrocortisone. If you don't take your Alkindi, you don't take your ET-400, you'll pass out. The mom then will have to take the child to the hospital, and the hospital will use this little kit here on the left. There are some parents that have that kit at home, but it's complicated. You have to reconstitute it. It's a freeze-dried product.

There's a number of steps in order to administer it. You got to make sure there's no air bubbles. You got to make sure you swabbed everything so it's sterile. It's a process. And if your child is dying on the floor, it's hard to keep your mind straight to do all of that. The autoinjector will give freedom of movement because you can carry that anywhere. I've seen it. I have it, and I've used it, not on myself, but a dummy version. It's like two. You click and hit. Click and hit. It's really, really a beautiful device. So that product, we hope to have filed next year, and we're making our batches this year. We'll run basically a bioequivalency study between these two, so no clinicals, maybe a human factors study.

Then we expect at least 240,000 units, which is what the Solu-Cortef has sold in the market today. So at our price point of about $400-$500 per unit, that gives us a $100-$200 million opportunity. But we think those 240,000 units will go vastly up because now parents will have the option to buy this EpiPen-like autoinjector, where if you look at today in the hospital market, you can buy a bottle of a little vial of epinephrine for under $10, but nobody does that. Everybody buys the $400 EpiPen because what are they going to do with a little vial of epinephrine at their house? They're not going to know what to do in most cases. Obviously, there's exceptions.

So we think a lot of the hospital market, which is in the millions, actually, there's millions of vials sold in the hospital market, will shift over to the retail, and we'll be able to I mean, it could be amazing. I don't even want to guess what kind of money we could see on that. All right. I've got a little less than a minute. Investment summary. We have a solid portfolio, commercial products. We have products in late-stage development. We have products with the FDA under review. Launches are happening. Next year is going to be an absolute breakout year. We've proven we can do this over a longer period of time. We continue to grow revenues quarter-over-quarter. We're very well capitalized to fund growth, positive cash flow, looking to profitability in the fourth quarter.

And then our near-term catalysts will be the PKU GOLIKE, which will add nice revenue, and then the potential approval of ET-400 and ET-600 in the next 12 months and active product acquisitions. So with that, that concludes my presentation. Thank you, everyone.

Speaker 2

I guess maybe just a quick question, Sean, for me.

Thanks. Just a quick question for me. You talked about, obviously, the focus on bringing in additional products. Obviously, your priority is ultra-rare disease. Other than fitting into the leverage that you have today, what are the key things that you look for in new product acquisition, potential acquisitions?

Sean Brynjelsen
CEO, Eton Pharmaceuticals

Yeah. Late-stage acquisitions we can get done for under $40 million. We look for, obviously, a rare disease. So an ultra-rare disease would be the best spot. I would say those are the three broad strokes on what we look for, not therapeutic, because we can deploy a sales team, and with that, whatever ultra-rare disease product comes into our lab. So that's also a unique aspect. We're not stuck in pediatric endocrinology. Any other questions? No.

Speaker 2

I'll just throw in one more. When you think about the size of the commercial infrastructure today and the line of sight that you have to that 2025, how much bigger does that salesforce or overall commercial infrastructure need to go?

Sean Brynjelsen
CEO, Eton Pharmaceuticals

I think as we get closer to launching ET-400, we'll add another four to five, which will take us in that 18-20-person salesforce. That should be good for the following year. Then we'll judge based on acquisitions if we're going to grow that beyond it. But you'll still always see a low SG&A footprint on it.

Speaker 2

Sean, thank you very much. Appreciate it.

Sean Brynjelsen
CEO, Eton Pharmaceuticals

Thank you.

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