ETON - NASDAQ

Thank you for standing by. My name is Alex, and I will be your conference operator for today. At this time, I would like to welcome everyone to the Eton Pharmaceuticals Q2 2025 Earnings Call. [Operator Instructions] Thank you. I would now like to turn the call over to Mr. David Krempa. Please go ahead.

Thank you, operator. Good afternoon, everyone, and welcome to Eton's Second Quarter 2025 Conference Call. This afternoon, we issued a press release that outlines the topics we plan to discuss on today's call. The release is available on our website, etonpharma.com. Joining me on our call today, we have Sean Brynjelsen, our CEO; James Gruber, our CFO; and Ipek Trinkaus, our Chief Commercial Officer. In addition to taking live questions on the call today, we will be answering questions that are e-mailed to us. Investors can send their questions to [email protected]. Before we begin, I would like to remind everyone that remarks made during today's call may contain forward-looking statements and involves risks and uncertainties that could cause actual results to differ materially from those contained in these forward-looking statements. Please see the forward-looking statements disclaimer in our earnings release and the risk factors in the company's filings with the SEC. Now, I will turn the call over to our CEO, Sean Brynjelsen.

Thank you, David. Good afternoon, everyone, and thank you for joining us today. I'm excited to be discussing our results, and it was another record quarter for the company. It is truly the most exciting time in our history. We entered 2025 with strong momentum from the growth of our existing portfolio, plus the closing of some value-creating acquisitions in December. The momentum carried into the second quarter in the business continues to fire on all cylinders. From our stellar commercial execution to the advancement of our pipeline programs from our development and regulatory functions, I could not be happier with the performance from the team this year. While we knew that 2025 was going to be a transformational time for Eton with 3 product launches planned, I am pleased to say it is going even better than we expected. The second quarter was particularly productive with a number of critical achievements, including the ongoing relaunches of Increlex and Galzin, which resulted in record product sales and more than 100% revenue growth year-over-year. FDA approval of Khindivi and the launch within days of the approval, marking our eighth ultra-rare disease product [indiscernible] The NDA submission for ET-600, another high-value pediatric endocrinology product candidate, which was subsequently accepted for review and assigned a February 2026 PDUFA date. This gives us another near-term product launch in the first quarter of 2026. And we delivered on the bottom line, generating $8 million of cash flow from operations and $3.1 million of adjusted EBITDA, highlighting our commitment to profitability and providing us with additional capital for future acquisitions. Turning to the results. Revenue was $18.9 million in the quarter, a remarkable 108% over the prior year period. We've now grown product revenue for 18 straight quarters every quarter since the launch of Alkindi Sprinkle in 2020. The increase was driven from broad performance across our portfolio, but the biggest contributors were continued growth in Alkindi Sprinkle and the addition of revenue from Increlex and Galzin. When we hosted our Investor Day in March, we provided guidance that we expected to exit 2025 at an $80 million revenue run rate. Based on the outperformance in the first half of this year, we now expect to reach the $80 million run rate in the third quarter, a full 3 months ahead of schedule. Turning to product-specific commentary. I will start with Increlex. Increlex has continued to exceed expectations, and we could not be happier with this acquisition. As a reminder, when we acquired the product in late December 2024, it had only 67 active patients. Our goal was to reach 100 active patients by the end of 2025. I'm pleased to share that we reached that goal a couple of weeks ago, 5 months ahead of schedule. This achievement did not come easy. The product had been flat or declining for many years prior to our acquisition, and it has taken a lot of hard work from our entire organization to reverse the trend. Since our January relaunch, we have made significant investments to improve education and awareness among the community. Our team had a major presence at the key endocrinology conferences this year, including Pediatric Endocrinology Nursing Society, the Pediatric Endocrinology Society and most recently, the Endocrinology Society Annual Meeting. During these conferences, we held multiple advisory boards, participated in product symposia, hosted exhibit booths, presented a new scientific poster featuring real-world registry and held hundreds of meetings with leading pediatric endocrinologists. We believe Eton is uniquely positioned to be able to drive such immediate Increlex adoption because of our deep relationships with the pediatric endocrinology community. These strong relationships should also benefit our launches of Khindivi and soon ET-600. I'm very happy with the initial Increlex results, but our work is not done. We believe severe primary IGF-1 deficiency is still significantly undiagnosed, and we have more to do on the awareness and education front. While the rate of future additions is not likely to match what we saw in the first 6 months of ownership, we remain confident that a large opportunity remains. Our new goal is to reach 110 patients by the end of this year. And over the next few years, we hope to be able to return to the level of 185 patients that were once on treatment. Reaching that 185 long-term goal would result in roughly $50 million to $60 million of annual sales for the company. In addition, we see an even greater opportunity for long-term growth through the harmonization of the U.S. and EU definitions of severe primary IGF-1 deficiency. As discussed previously, the EU currently has a broader definition. In the U.S., patient's IGF level must be at least 3 standard deviations below the median. While in Europe, the level needs to be only approximately 2 standard deviations below. Increlex's previous owner has been running a patient registry in Europe, which has collected data from hundreds of patients over the past [indiscernible] years. To us, this real-world data clearly demonstrates that the product is safe and effective for patients whose IGF levels are between -2 and -3 standard deviations. We recently approached the FDA to discuss harmonizing the definition between the 2 regions. The agency communicated to us that some type of follow-on clinical study would likely be necessary. As such, they requested that we put together a protocol design and request a meeting. We are in the process of working with external consultants to draft the design that is achievable and meets the FDA requirements. We intend to submit a treatment IND for an open-label study where we would enroll patients with IGF levels between -2 and -3 standard deviations. While it's too early for us to speculate on the precise time or cost of such a study, we believe the large commercial opportunity would more than justify any reasonable amount of time and capital invested into such a study. As a reminder, we currently estimate that there are approximately 200 patients in the U.S. that meet the -3 standard deviation criteria, but believe more than 1,000 would meet the broader -2 standard deviation criteria. If successful, that would grow potential market from around $60 million a year to nearly $300 million per year. We will keep you updated as our discussions with the FDA progress. Turning now to our adrenal insufficiency franchise of Alkindi Sprinkle and Khindivi. We were excited to receive FDA approval of Khindivi on May 28th. As the only FDA-approved oral solution of hydrocortisone, Khindivi fulfills a critical unmet need by allowing simple and accurate dosing tailored to each patient's needs. It does not require refrigeration, mixing or shaking and eliminates the need to split or crush tablets, which can lead to inaccurate dosing. Khindivi was approved for pediatric patients 5 years of age and older. While we and our toxicologists believe the data shows that the product is safe for infants as young as 1 month, the FDA had reservations due to a limited amount of safety data around how 3 of our inactive ingredients are metabolized when used in combination. Because of this, the FDA ultimately approved the product with a label of patients aged 5 and above. Roughly 60% of our Alkindi patients are 4 and under, so we expect this age limit will be a near-term hindrance to the Khindivi launch trajectory. However, we have a plan in place that should make it a short-lived headwind. We know there is a huge unmet need for this product among infants and toddlers, and we remain committed to addressing it. Once feedback was received on our label, we immediately went to work on a revised formulation that drastically reduces the amount of these 3 excipients. We've already manufactured registration batches of the revised formulation and have a pre-submission meeting with the FDA scheduled for September. Our current plan is to submit a prior approval supplement in the first half of 2026, which could allow for an approval and a broadened label by the end of 2026. Despite this initial headwind, we've already seen adoption among patients and have received favorable feedback from families and caregivers. Many physicians have told us they plan to switch all their patients, 5 and up, that were previously on compounded products. These switches have been occurring as patients have their regularly scheduled checkups. Now in its fifth year, Alkindi Sprinkle continues to deliver robust growth with no signs of slowing down. In fact, the first half of 2025 generated more new Alkindi scripts than any other 6-month period in the product's history. Adding Khindivi to the mix should allow us to accelerate that growth. Our combined adrenal insufficiency franchise recently eclipsed 500 active patients, and we remain confident that we can reach $50 million of combined sales in the coming years, which would be approximately 1,000 active patients. Ultimately, we expect to reach much higher levels than that once the Khindivi label is expanded. Another important value driver for us for [ sure ] is Galzin. With the acquisition of Galzin, we saw another opportunity to add significant value and improve access for patients with Wilson disease. After the acquisition, our first priority was to solve Galzin's historic access and affordability issues. Before Eton acquired Galzin, very few pharmacies actually stocked the drug. Supplies were occasionally out of stock. No patient support services were in place to help with insurance paperwork, and there was no financial support. So even if patients were able to get the product, many of them faced very expensive out-of-pocket costs. As a result, we believe that the majority of patients in the U.S. that were on zinc therapy were actually taking an inferior non-FDA-approved over-the-counter zinc gluconate supplement. For years, doctors have been hesitant to prescribe Galzin because of these access issues. They knew there was a good chance they would get a call back from a patient saying the co-pay was too expensive or their pharmacy did not stock it or they needed support with insurance paperwork. As a result, many doctors had turned to recommending supplement products strictly because access was easier. We knew we could solve these issues with our best-in-class patient support program, dedicated rare disease specialists and extensive education and awareness campaign. Galzin is now available exclusively through the Eton Cares patient support program, which offers $0 co-pay for qualified patients, 24/7 customer support, allowing every patient who wants Galzin to get it regardless of financial circumstances. Now the message is clear to physicians. If they prescribe Galzin for their Wilson patients, Eton Cares will make sure the patient receives the product. They are now prescribing Galzin with this peace of mind. In addition, under diagnosis has also been a major problem with Wilson disease. The majority of patients aren't diagnosed until adulthood when symptoms begin to present themselves after decades of copper buildup. The delay in diagnosis leads to worse outcomes, including neurological damage and liver failure. Wilson disease is believed to impact approximately 10,000 people in the United States, but only about 20% are diagnosed and on therapy. We expect to see a long-term tailwind as increased genetic testing and better screening leads to higher rates of diagnosis and more patients starting preventive zinc therapy. As I mentioned earlier, our Galzin launch is off to a strong start, and we expect the trajectory to continue. I believe the majority of existing Galzin users have now been converted to our product. We're aiming to reach 200 active patients by the end of this year, setting the foundation for continued growth in the years ahead. In addition to removing barriers to access, we think we can do even more to improve the lives of Wilson disease patients. Currently, Galzin must be taken 3 times per day, and patients must fast before and after taking the medication. This is a burdensome process for patients and leads to poor compliance. To address these issues, we're developing ET-700, an extended-release version of Galzin. Our team initiated the program prior to the acquisition of Galzin and have now filed a patent on our proprietary formulation. During the second quarter, we held a meeting with the FDA to discuss the clinical pathway for ET-700. We view the outcome of this meeting positively since clarity on the study program and clinical requirements was achieved. Based on the FDA feedback, our plan is to initiate a proof-of-concept study near the end of this year, followed by a dose-ranging and pivotal Phase III study. ET-700 represents another very large market opportunity for Eton. With this product, we believe we would capture a majority of the estimated 800 U.S. patients on some form of zinc therapy and generate more than $100 million in annual revenue. During the quarter, we continued to make progress with our other pipeline product candidates, the most notable of which is ET-600. Last month, the FDA announced that it accepted our ET-600 NDA and assigned our target action date of February 25, 2026. In the last few weeks, we were also awarded a second patent for the product, which grants us protection through 2044. As a reminder, ET-600 is our proprietary oral solution of desmopressin under development for the treatment of diabetes insipidus. Right now, desmopressin is approved in tablet, nasal and injectable forms, none of which allow for the small, precise and titratable doses needed for younger patients. Many pediatric patients use unapproved compounded liquid suspensions or are forced to cut tablets. If approved, ET-600 would be the only oral liquid option on the market, addressing a significant unmet need we've identified within the pediatric endocrinology community. We hosted an ET-600 advisory board with key opinion leaders last month at the ENDO conference, and the feedback was overwhelmingly positive. The health care community is anxious to see the product on the market and our commercial launch activities are underway in anticipation of a potential Q1 2026 launch. Given our strong presence in pediatric endocrinology with existing promotion of Alkindi Sprinkle, Khindivi and Increlex, we expect to be able to hit the ground running with a strong launch in 2026. The potential ET-600 launch should bolster our already strong near-term growth prospects, and we will continue to further turbocharge our growth with additional product acquisitions. Opportunistic business development transactions have been central to our historic growth, and we remain on the hunt for new product acquisitions. We are doing so from an attractive position of strength, both financially and operationally. Eton has more than $30 million of cash on hand and great access to additional capital, if necessary, to fund any acquisitions or transactions that fit our strategy. Given the robust growth outlook of our existing product and pipeline, we are under no pressure to chase acquisitions, however. We will remain disciplined as we pursue approved or late-stage ultra-rare disease products that are strategically and financially attractive. While I am proud of the record second quarter sales and the massive revenue growth, I am even prouder of our ability to do so profitably. As you know, we pride ourselves on running a fiscally responsible business and are not interested in growing revenue if it does not lead to profitability. This quarter, we have started to show early signs of the immense earnings power of our business. We generated $8 million of operating cash flow and delivered strong adjusted EBITDA and non-GAAP earnings per share. We've now made the necessary SG&A investments to support our broader portfolio and larger revenue base. So we expect to continue to see meaningful margin expansion, as expenses remain relatively flat, while revenue grows in the coming quarters. Great companies are not built overnight, and it has taken many years of dedication from our team to put us in a position we are in today. I am thankful for all of their hard work and incredibly impressed by the team's ability to execute and outperform across all facets of the business. The position we are in is truly unique with 8 approved products, 3 in-process product launches with long runways for growth, another potential product launch in the first quarter of 2026, multiple label expansion opportunities and a pipeline full of innovative candidates that are progressing towards market, the future has never been brighter for Eton. With that, I'll hand it over to James, our Chief Financial Officer, to discuss the financials. James?

Thank you, Sean. Turning to our financial results. Our second quarter revenue was $18.9 million compared to $9.1 million in the second quarter of 2024, an increase of 108% and was comprised entirely of product sales. The growth was driven primarily by increased sales of Alkindi as well as the addition of Increlex and Galzin, which were not included in the prior year period. As Sean mentioned, we now expect to achieve an $80 million annual revenue run rate in the third quarter of 2025, one quarter ahead of previous projections. While we expect sequential revenue growth to continue, it will not be at as rapid of a pace as was seen from Q1 to Q2. The second quarter benefited from the first full quarter of Increlex revenue under Eton distribution, and at increased patient levels, so quarterly growth was more pronounced than it will be in the coming quarters. Gross profit for the quarter was $11.9 million compared with $5.6 million in the prior year period due primarily to increased product sales. Adjusted gross profit, which excludes the impact of acquired inventory step-up adjustments and an intangible amortization was $14.1 million or 75% of total revenue versus $5.9 million of adjusted gross profit or 65% of total revenue in the prior year period. The improved adjusted gross margin profile was driven by continued growth of higher-margin Alkindi Sprinkle and the addition of higher- margin Increlex revenue. Our adjusted gross margin profile is expected to decline in the second half of this year as we transition to a new international distribution model for ex-U.S. Increlex. While the transfer price at which we supply product for ex U.S. markets is materially dilutive to corporate gross margin, we continue to expect to report full year 2025 adjusted gross margin of approximately 70% and for long- term adjusted gross margin to reach 75% by 2028. R&D expenses for the quarter were $3.7 million compared with $3.0 million in the prior year period. The second quarter of 2025 included a $2.2 million NDA application fee related to the ET-600 submission and a $0.5 million licensing payment for Amglidia. General and administrative expenses for the quarter were $9.7 million compared with $5.6 million in the prior year period. The increase was primarily driven by the expansion of our sales force, marketing costs associated with 3 product launches and increased compensation and benefit expenses associated with additional corporate headcount to support our growing portfolio. On an adjusted basis, which removes the impact of share-based compensation, transaction-related costs and other onetime expenses, G&A expense was $7.6 million compared to $4.9 million in the prior year period. We do not plan to make any additional significant investments into SG&A this year, and much of our planned 2025 marketing spend was heavily weighted in the first half of the year with our product launch efforts. So we expect adjusted G&A spending to remain flat or slightly decline for the remainder of 2025. We believe that the investments we've made over the past several quarters will support much higher revenue levels than where we are today. Adjusted EBITDA for the second quarter of 2025 was $3.1 million compared to negative $1.6 million in the second quarter of 2024. Total company net loss was $2.6 million for the quarter compared to a net loss of $3.0 million in the prior year period. Net loss per basic and diluted share was $0.10 during the quarter compared to a net loss per basic and diluted share of $0.12 in the prior year period. On a non-GAAP basis, we reported net income of $1.5 million for the second quarter of 2025 compared to a net loss of $1.9 million in the prior year period, and diluted earnings per share of $0.03 for the second quarter of 2025 compared to a loss of $0.08 per basic and diluted share in the prior year period. Eton finished the second quarter with $25.4 million of cash on hand, and we generated $8 million of operating cash flow during the quarter. A $4.6 million payment from Esteve for the international rights to Increlex was received shortly after the end of Q2. So the company's cash balance as of today stands at more than $30 million. This concludes our remarks on second quarter results. And with that, we'll turn it over to the operator for Q&A.

[Operator Instructions] And your first question comes from the line of Chase Knickerbocker with Craig-Hallum.

Congrats on the quarter. Sean, I just -- maybe to start on Khindivi. Can you kind of talk through the momentum that you've seen so far in the early stages of that launch and kind of what you're hearing from clinicians on that [ restricted ] label? And if we think about over the remainder of the year as far as what's possible from a volume's perspective, any way for us to kind of think about kind of patient growth for that franchise along with Alkindi, kind of in the remainder of the year?

Sure. Thanks, Chase, for the question. We have not broken out Khindivi and Alkindi into separate buckets. I can say that some physicians have already instructed their offices to only -- for anybody of 5 and above that is on liquid, they will completely convert to -- I mean, to Khindivi. And we'll continue -- we've been adding patients on a weekly basis. That rate combined with Alkindi Sprinkle, we're pleased with. We think that we're going to exceed expectations on those 2 products as a combined, and we see we're very clearly on track to do more than $50 million. The real opportunity though, of course, will be when we can get the label expanded to include the 4 and below. That's where we really think the population and the majority of the sales will go. But yes, I don't want to break out Khindivi and Alkindi yet, but I can tell you that the launch is moving along. It's not going in -- unexpected. We get a majority or a very large proportion of the 5 and above. And we're not seeing a lot of conversions from going off of Alkindi and going on to Khindivi. So right now, it seems to be more additive than anything else. We were, for a bit there, weren't sure if we would see a lot of conversions from Alkindi to Khindivi, but that has not been the case.

Helpful color, Sean. And maybe on Increlex, can you share a little bit more on kind of what kind of trial design they were kind of discussing with you in that FDA meeting? I mean, was it something where they referred to kind of the original trial for Increlex? Was it something that -- something a little bit smaller and quicker would be sufficient? Just any general thoughts that came out of that meeting would be helpful to kind of think about the path forward for that.

We believe we'll open up an IND where we'll be enrolling patients in between the -2 and the -3 standard deviations and that, that will be -- we'll be able to then go to doctors and tell them, look, if your patient isn't -- is -2, for example, where we have an open trial that patient can receive product under the IND. And our hope is that we can then place the product with that patient and still get reimbursed for it. So it would be an ongoing trial. Then at some point we would compile all that data and then put it in front of the agency. So it really depends on if the agency allows us to take that role. I'm kind of optimistic they will because it's already being used that way in Europe.

Got it. And as far as the kind of expectations for Increlex from here, obviously, we've had this big [ bolus ] initially upon launch. I mean what's the right way to think about it both in the remainder of the year and kind of if we look out, I mean, how quickly do you think we can get to this 185 patients? Is it something where it will be meaningfully slower now? Or is it something where we can still add 20, 30, 40 patients next year? Just trying to get a sense of how you're thinking about it. [Audio Gap]

Your next question comes from the line of Madison El-Saadi with B. Riley Securities.

Congrats on really a great quarter. I guess 2 questions from me. Maybe could you help us understand where the beat came from across Increlex, Galzin, and then Alkindi, Khindivi? It sounds like you really had outperformance across the board. But just wondering really if one drove it? And if you would -- if that's the expectation for second half, which product could really drive second half?

Madison, this is James. Sean and David got cut off. Chase at Craig-Hallum, we'll get back to you on your question as well. But Madison, we still don't disclose product-specific revenue just from a competitive standpoint. But per the -- all of the comments on Increlex exceeding expectations from a patient on therapy and an initial ramp, the beat is definitely heavily weighted toward Increlex, but we saw a growth in Alkindi and Galzin as well, but Increlex definitely led the way.

Got it. And then -- and maybe this is for Sean or David, whenever they get back.

Yes, we just got reconnected. Can you hear us?

Yes. Yes, we can. Just in time. Yes. So, I guess regarding the FDA feedback, did they specify the 2 to 3 standard deviation range? And did they give any rationale for requesting this trial? I'm just wondering if you feel like you gained the clarity from them on really what the regulatory vehicle is going to be going forward? If it's going to be a typical kind of label update? Or is it going to be a prior approval submission?

Yes. It will be a open IND is what we would like to do. And what that means is we could go to doctors today and the -- our sales reps should be able to say, if your patient is -2, which is the European standard, we have this open IND, you can enroll them in this also, and we would -- we hope we would get reimbursed for that, but it would be something that we could go and tell doctors to bring patients in. And we'd collect that data over a period of time and then provide that to the Agency. But that is the European label. So that's -- I don't see any reason why we couldn't do that. When we went to the Agency, we told them that's what we wanted to do. They said, come back to us with some sort of a proposal where we can -- you can collect some U.S. clinical data. And right now, our consultants are saying that they're confident that, that would be acceptable to the Agency. But we'll provide another update later this year, and we'll be able to tell you more in the quarters to come. I expect to get agreement on this though, in the fourth quarter, and then we would start right away with enrolling patients.

Your next question, we have again with Mr. Chase Knickerbocker with Craig-Hallum.

Sorry, just one quick follow-up, maybe for James. Just on kind of back half ramp. Sorry if I missed this in your prepared remarks, but if I think about the back half ramp for OpEx, here the onetime items in R&D, I mean, any commentary as far as how we should think about the back half ramp in OpEx?

Yes. With R&D, that will definitely -- we have the big filing fee, $2 plus million filing fee in Q2 as well as $0.5 million licensing payment for Amglidia. So those obviously won't reoccur. And then from an SG&A standpoint, the big item that will change from H1 to H2 with the 3 product launches pretty much behind us at this point, that will be a pretty significant ramp down. So we don't anticipate while the -- we did go up a little bit on an adjusted basis from 7.3% to 7.6%, we expect to be at or below those levels second half of the year.

And we have the last question comes from the line of Swayampakula Ramakanth with H.C. Wainwright.

Just to talk a little bit about the Khindivi launch. You're stating that there are about 60% of the patients are age 4 and older. So --

Four and below, I'm sorry --

Sorry, 4 and below. Sorry, 4 and below. Yes. I'm reading wrong from my notes. So in terms of getting this new formulation and to do the study, is this going to be -- how big of a study is that going to be? And in terms of timing, in general, how long does these sort of studies take?

There's no study. We had to lower the excipient concentration. So we did that. We have to submit in, we believe, the first quarter, and then we hope to have approval of the wider label by the end of next year. So this is very quick. It was a slight modification of our existing formula, which was really easy to do.

Okay. And then -- so let's say you get that all taken care of by the end of next year. So what sort of an additional patient number can you have -- can this drug be offered?

Well, by itself, I mean, there's over 1,000 patients taking the oral suspension today. So, how quick that would ramp up? That's a good question. I guess we'll have to see. But there's no doubt in my mind. Once we have that one and combined with Alkindi, we'll exceed 1,500 patients, probably 2,000 patients on product. That seems very achievable.