Liquidia Corporation

Chief Medical Officer, Dr. Rajiv Sagar, Chief Operating Officer and CFO, Michael Cassetta, Chief Commercial Officer, Scott Mumaw, and General Counsel, Rusty Schoenler. Before we begin, please note that today's conference call will contain forward-looking statements, including those statements regarding future results, unaudited and forward-looking financial information, as well as the company's future performance and or achievement. These statements are subject to known and unknown risks and uncertainties, which may cause our actual results or performance to be materially different from any future results or performance expressed or implied on this call. For additional information, including a detailed discussion of our risk factors, please refer to the company's documents filed with the Securities and Exchange Commission, which can be accessed on our website. I would now like to turn the call over to Roger for our prepared remarks, after which he will open the call for your questions.

Good morning, everyone, and thank you for joining us today. In a little more than two weeks, May 24th to be precise, we will have reached the PDUFA goal date for Liquidia's first internally developed commercial product, Utrepia. an investigational inhaled dry powder formulation of tropostinil for the treatment of pulmonary arterial hypertension, or PAH, and pulmonary hypertension associated with interstitial lung disease, or PHILD. With all eyes on the future, we are going to keep our prepared remarks focused on a few key areas. First, I'd like to take a moment to highlight last week's news regarding the decision rendered by the District Court dismissing United Therapeutics' cross-claim that sought to challenge our amendment to the new drug application for eutropia. which added the treatment of PHILD to Utrecht's proposed label. While United Therapeutics has the right to appeal the court's ruling, we are pleased with the court's decision to dismiss this cross-claim, specifically holding that United Therapeutics failed to establish standing. We are proud to say there continue to be no legal barriers barring Utrecht's potential final approval following the expiration of gating regulatory exclusivity on May 23rd, 2025. With this favorable ruling in hand, we are doubling down on our preparations for the potential launch of Utrepia, with a laser focus on five key strategic areas. First, we're developing what we believe is a best-in-class product profile for Utrepia. As we've said before, Utrepia's tolerability, titratability, ease of use, and future labeling speaks to the fact that it offers a differentiated product profile. We continue to further characterize these clear benefits in the company's open-label assent study in PHILD patients. As communicated in this morning's press release, cohort A of the assent study is now fully enrolled with more than 50 patients. The interim data has shown us that the dosing and tolerability profile in the first 20 patients to complete eight weeks of treatment was consistent with observations made in the INSPIRE study of PAH patients. Thus far, PHILD patients in the assent study have been able to titrate to doses that are three times higher than the labeled target dose of nebulized tibiasis. These patients have also shown positive trends on exploratory measures of efficacy, including six-minute walk distance. Additional data from the ASCENT study will be presented during two poster sessions at the American Thoracic Society's International Conference in San Francisco on May 20th. As part of the ASCENT study, we will begin cohort B, a directed transition study in the coming months where we will take patients unsatisfied with the clinical attributes of Tyveso and Tyveso-DPI and transition them to Eutrevia. The goal of this study will be to directly compare the differences and potential benefits that Eutrevia presents, both in PAH and PHILD patients. You'll hear more regarding this study in the months ahead. Next, we are fully prepared to go to market with a competitive share of voice. We have 50 sales reps in the field who have been on board for about 18 months and a companion medical affairs team, all with an impressive level of rare disease experience and most with pH experience. These groups have been actively surveilling both the major centers of excellence as well as the community prescriber base in preparation for Utrecht's potential launch. Our third strategic area of focus is our preparation to launch a full suite of patient support services, which we have meticulously put into place. Physicians and patients should expect no differences in support with Eutrepia, whether starting inhaled Trapasil for the first time or transitioning from incumbent inhaled products. Fourth, we've continued to focus on ensuring robust product availability. Mike and his team at Liquidia have prepared to put product in the channel in only two to three weeks after Eutrepia's potential approval. And finally, the fifth strategic area of focus that will help us ensure success is broad payer access. Over the last several years, we have developed strong relationships with payers who understand the differentiated product profile that Eutrepia can offer to patients. We remain confident that patients will have access to Eutrepia within a short time after launch. With all these in play, we feel well prepared to launch Eutrepia into the marketplace once approved, and we look forward to fulfilling our promise to provide patients with PAH and PHILD, a much-needed and potentially best-in-class therapeutic alternative. I'll now turn the call over to Mike to provide an overview of our first quarter 2025 financials. Mike?

Thank you, Roger, and good morning, everyone. Turning to our first quarter 2025 financial results, which can be found in the press release, you will see that revenue was $3.1 million for the three months ended March 31, 2025. compared to $3 million for the three months ended March 31st, 2024, revenue related primarily to the promotion agreement. The increase of $0.1 million was primarily due to the impact of unfavorable growth to net returns adjustments recorded in the prior year, offset by lower sales volumes in the current year. Cost of revenue was $1.5 million for each of the three months ended March 31st, 2025 and 2024. Cost of revenue related to the promotion agreement as noted above. Research and development expenses were $7 million for the three months ended March 31st, 2025, compared to $10.1 million for the three months ended March 31st, 2024. The decrease of $3.1 million, or 31%, was primarily due to a $3.6 million decrease in personnel expenses, including stock-based compensation, due to a shift from activities related to research and development to preparation for the potential commercialization of EUTREPIA. These decreases were offset by a $1.7 million increase in clinical expenses related to our L606 program and a $0.4 million decrease in expenses related to our EUTREPIA research and development activities. General and administrative expenses were $30.1 million for the three months ended March 31, 2025, compared to $20.2 million for the three months ended March 31, 2024. The increase of $9.9 million, or 48%, was primarily due to an $8.1 million increase in personnel expenses, including stock-based compensation, driven by higher headcount and a shift from activities related to research and development to preparation for the potential commercialization of EUTREPIA, a $0.6 million increase in legal fees related to our ongoing EUTREPIA-related litigation, and a $0.6 million increase in facilities and infrastructure expenses. We incurred a net loss for the quarter-ended March 31, 2025, of $38.4 million. or 45 cents per basic and diluted share, compared to a net loss of $30.1 million, or 40 cents per basic and diluted share, for the three months ended March 31st, 2024. Included in the comparative results we have presented is an immaterial revision of other income, as previously reported for fiscal year 2024. This revision is a technical, non-cash accounting adjustment related to gain and loss recognized when we made amendments to our HCR facility made in 2024. Additional details are included in the Form 8K we filed this morning. I would now like to turn the call back over to Roger.

Thank you, Mike. In summary, we continue to drive forward the right set of strategies to support the successful potential launch of Eutrepia in the coming weeks, and we look forward to providing both physicians and patients with what we believe could soon become the prostacyclin of first choice. I would now like to open the call to questions. Operator, first question, please.

Thank you. At this time, we will conduct the question and answer session. As a reminder, to ask a question, you will need to press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. Our first question comes from the line of Julian Harrison of VTIG. Your line is now open.

Hi, congrats on all the progress and thank you for taking my questions. I'm wondering if you believe United has any injunctive value left on the cross claim that was dismissed last week. Also curious if you had any reaction to the dismissal without prejudice versus with prejudice, given the judge's expressed views on standing in the opinion memorandum.

Good morning, Julian. Good to hear from you. Rusty, if you wouldn't mind answering Julian's two questions.

Sure. Julian, thanks for the question. So let me, I mean, over a couple things i mean one what what's absolutely clear is that there's currently no proceeding um actively ongoing in which united therapeutics is seeking to enjoin either the fda from giving us approval for eutrapia or seeking um any sort of uh temporary restraining order or preliminary injunction to prevent our launch of eutrapia upon approval um that's number one number two um The dismissal was without prejudice. Again, keep in mind it was a motion to dismiss, so the judge was not addressing the full merits of the case. This was a question of whether their complaint had even set forth grounds upon which the court could even entertain their lawsuit. What that does mean is they can try to repurpose or repassion the argument in the future, but I think what's clear from the way in which he approached the opinion is They can't come back with the exact same argument they did before. I think they still have significant standing issues trying to link the bundling guidance to a 30-month stay. And keep in mind, again, this is all just at the motion to dismiss phase. They still would have to then show that they're correct on the merits as well. As far as other things that United Therapeutics might do, which I think your question alluded to, You know, that's really not for us to say. You know, we don't know what lengths they're going to go to try to deny PH and PHLV patients access to an alternative therapy. What I can say, though, is, you know, we'll be ready, you know, whatever form they pursue something, if they pursue anything from here on out, we'll be ready to address it. Thanks.

Excellent. Very helpful. Thank you, and congrats again. Thank you, Julian.

Next question, please.

Thank you. Our next question comes from the line of Kambiz Yazdi of Jefferies. Your line is now open. Kambiz, do we have you here?

Sorry. Morning, team. Thank you so much for the questions and very exciting times for the company. Maybe a little bit on the, you know, the forward-looking transition studies Any kind of thoughts on what may be interesting patient baseline characteristics that you may be looking to enroll in that study and then would love to get an update on L606 as well.

Great. So, both of those questions fall into Rajiv Sagar's court. So, Rajiv, do you mind answering those?

Yeah, I can be. Thanks for the question. So, the first question was regarding the upcoming transition study. This study is specifically evaluating the transition from Tyveso, either nebulized and preferably dry powder inhaler, to eutropia. These patients will be specifically in the category of PHLD. And that's very purposeful. As you know, for NDA filing in the INSPIRE study, we already showed that we were able to successfully transition patients with group 1 PAH from nebulized Tyveso directly to Eutropia. And now we're trying to do the same thing in the PHLD population. So first of all, these patients will be on background Tyveso. The majority of them are on dry powder inhaler. These patients will have PHLD. And very similar to cohort A in part of the SENS study that we're presenting at ATS, We're going to be looking for the ability to, we're going to be looking at safety. So can we safely transition number two? Can we transition them and then continue to showcase our product profile, which we believe is going to be beneficial in terms of our ability to up titrate that patient, maintain their clinical stability, or even improve them. So we'll be looking at exploratory efficacy modalities, inclusive of, of course, six-minute walk and other quality of life indicators. So we look forward to showcasing that study and initiating that in the next few months. And your second question, Kambiz, is that on L606?

Yeah, just an update there.

Yeah, thank you. So, you know, we're working diligently to initiate this global study. Just to recap, this is the liposomal sustained release formulation of Triprostol that we're going to be delivering twice a day. Just to recap, this is a global study with more than 300 patients and more than 20 companies, and we anticipate that we'll be initiating this study by year's end. Thank you.

Thank you. Our next question comes from the line of Greg Harrison of Scotia Bank. Your line is now open.

Hey, good morning, guys. Congrats on the progress, and thanks for taking the question. So, we've seen the competitors struggle to convert patients to their dry powder formulation, and their nebulized formulation has persisted longer than thought initially. Could you speak to the patient perspective on switching to dry powder formulation and and whether you think Utrepia will have a stronger case for keeping patients on a DPI formulation than we've seen with Tybaso, and how do you think the assent data will help with this argument?

Yeah, good morning, Greg. Thanks for the question. So I'll start with an answer, and then Rajiv, if you'll give your thoughts as well, I think that'd be helpful. So, you know, again, I think you are right. It's surprising that there remain residual nebulized Tybaso patient base. I think it's about 31% of the scripts to date. when all thoughts, including the competitor's thoughts initially, were that all patients would transition to the DPI for portability reasons alone. So that hasn't happened, and the question is why. We think it's formulation-driven and that their formulation is part of an aggregated polydispersed formulation on a FTKP backbone, really, and a high resistance device and low flow really doesn't deliver the drug to the lower airway, which is the site of action. So that's completely the opposite of what a print-enabled eutropia is going to achieve, and which is what we've shown in the ASCENT study, that patients can tolerate the drug well, they can get to very high doses, and they can get there quickly. So those two points are important in the sense that Patients want to feel better. They want to do it on a convenient and portable therapeutic modality. And we think eutropia is clearly defined as a differentiated and a better opportunity to do just that. So I think what we'll do in the marketplace first is go after new patients to let physicians experience the benefits of eutropia firsthand. And then once they're comfortable with that, then we'll see if they will transition, particularly their nebulized patients, who are going to be looking for an alternative, more portable therapy. And then also, I think it becomes a question of would you even start Tyveso DPI? Or if you're on Tyveso DPI, would you want to transition? And as Rajiv just said in the previous question, we're going to have data directing to the specifics of how you transition and the benefits of doing the same. So we're really excited about all of the market opportunity. But I do agree that the nebulized Tyveso retained commercial share is at risk and we're still going to go after it in time. So, Rajiv, do you have any thoughts?

Yeah, thanks, Roger. I think, again, just highlighting, I think we believe in our scientific formulation and our hypothesis that the print formulation with the low flow resistance device is actually what these patients need. I think from my perspective, I think what's really unique is two things. Back when we did the INSPIRE study in Group 1 PAH, it took a while for patients to titrate up eutropia to get to doses of therapeutic zones. I think what we're seeing now, a few years later, now that we did the first cohort study that we now have fully enrolled, and we're going to showcase that data at ATS, is that not only can we titrate doses to above the sort of traditional 9 to 12 breaths, you know, we're dosing these to, you know, at least two-fold levels higher than what traditionally is given by Tyvesa. But we can do it in a matter of just, you know, several weeks, not, you know, over a course of a year. And I think that's very important because these patients are extremely sick. They are susceptible to clinical worsening. And finally, inherently, they just have terrible coughs. And one thing I think to your question is that the current incumbents dry powder formulation, I think one of its major limitations is its inability to titrate to the appropriate doses to match the clinical severity of the patient, in part, due to cough. And I think one of the things that we are going to continue to highlight, especially at ATS, is just the tolerability profile of eutropia seems to be well received by the patients and the practitioners that are participating in the study. So I think using that signal And re-showing that in the transition study, I think, would be well received by the scientific community.

Great. Thank you, Rajeev. I think your comments speak to the snippets of, you know, why we're so excited about the pending launch. Operator, next question, please.

Thank you. Our next question comes from the line of Corey Jubinville of LifeSci Capital. Your line is now open.

Thanks for taking our questions, and congrats on the progress. So, you mentioned earlier in the prepared remarks that you're building out this prescriber support team, and as we speak to KOLs, in addition to efficacy and tolerability, it seems that ease of prescribing also appears to be a major component to their prescription habits. Could you just provide a little bit more detail as to what that support team might look like in their activities? Specifically, what might you be doing that improves the prescriber experience over what's out there presently and how that compares to potential competitors? And, you know, I guess on the patient side, in terms of early patient access, do you have any details about what a potential bridging program might look like if that's in the plans for patients?

Yeah, thanks for the question, Corey. So we're benefited by having our Chief Commercial Officer, Scott Muma, on the call. So, Scott, if you wouldn't mind addressing the question.

Yes, sure. Good morning. So, you know, the specifics I think we'll sort of share as we get through and pass approval around the patient support program. But what I would say is, you know, our team has over a decade, most folks have over a decade of experience with prostenol in the various forms, working with especially pharmacies, working with this patient population. And we have a very good understanding of what the needs of the HCPs and the patients are. So we've built out a program that we think in all respects will be as good as or better than what's on the market right now in terms of the way that these patients have been cared for. We completely understand the point that you raised around being able to start these patients is what's very important. to early success, and we're going to make sure that we have everything in place to make sure that happens. And we look forward to sharing that with you hopefully after the end of May.

Great. Thank you, Scott.

Thank you. Our next question comes from the line of Jason Garbury of B of A. Your line is now open.

Hey guys, this is Chi for Jason. Thanks for taking our question. So you have some data presentation at the ATS meeting in a couple of weeks. I'm curious if you can give us an early flavor of these presentations, what's your expectations, and when might we get the full 48-week data from the ASCEND study? Thanks so much.

Great. Since, Rajiv, you're the leading architect of the cohort I studied, you could talk to that question.

Yeah, thanks for that question. I think at this point, you know, obviously we're under embargo to go into the actual details of the study, but essentially we're going to be presenting three posters. Two are specifically related to the Ascent study, which we will showcase the first 20 patients that were treated for eight weeks. Remember, these are patients with newly diagnosed PHLD that are now taking eutrepia. So we'll showcase a few things, the baseline demographics. We will detail out the tolerability profile of these patients as well as the dosing characteristics that we're seeing. So that's the first thing. The second thing is that we'll finally showcase some exploratory endpoints within the first two posters. The first one being, of course, what happens to their exercise tolerability, which is defined by six-minute walk distance compared to baseline through eight weeks. And the other thing is we're going to showcase a novel endpoint that's known as cardiac effort, and that will be presented by Dr. Dan LaChante at the University of Rochester. Again, sort of understanding why potentially these patients are showing, you know, improvements in their exercise capacity and what are the potential reasons for that and how eutropia is modifying its performance on the right ventricle. The third poster will be presented. looking at transitioning a patient that was participating in the INSPIRE study who had acutely worsened and was hospitalized, placed on intravenous treprosinal and transitioned to LIQ861 or eutropia, and just highlighting that safety, that ability to transition patients from parental therapy back to LIQ861 in that study. All in all, we're very excited to, again, showcase some of these, our product profiles at ATS.

Thank you, Rajiv. Obviously, the timing of that data is quite exciting, and the fact that it will be presented literally within a week of potential approval. Operator, next question. Thank you.

Thank you. Our next question comes from the line of Sergey Bellinger of Needham. Your line is now open.

Hi, good morning. A couple of commercial questions. The first one maybe for Roger and Scott, can you just describe the level of awareness of eutropia and its differentiating attributes in the group of physicians you'll be targeting upon launch? And secondly, do you expect that there could be some warehouse demand for this product or this is a group of physicians that will likely want to run their own

um internal evaluation before really ramping up usage of utopia thanks yeah thank you thanks for the question sir scott maybe you could opine on this sure um so on the awareness question um obviously you know prior to launch we're very limited uh in you know what we can communicate perspective in fact nothing so but we're looking forward to the launch and i can guarantee you that we will be loud The awareness will go up extremely quickly. There is background awareness due to the medical information we've shared, et cetera. But once the sales team gets out there, we have a full suite of marketing activities, electronic activities. Our goal is that every HCP who is involved in PH, whether that be PAH or PHLD, will be aware and will be able to try it very soon after launch. The second question was about warehousing. I think there might be some of that due to the tolerability issues in the conversation that came up earlier around the nebulized patients. Certainly, as Roger has said, I think even earlier in this call, we'll be focused on new patients because those are the patients that give the physician really the best opportunity to try the drug in sort of a clean way. Having said that, we have heard from a number of physicians that they have patients that have transitioned back to the nebulized version of Tyveso. And I think we all understand that that's not optimal from a convenience standpoint and a dosing standpoint. And so we will get some of those. We will work with those physicians to make sure that those are successes. But strategically, you know, the patients that we'll be going after first and foremost will be those patients that are new to prostacyclines.

Yeah, thanks. And I think, Serge, one way to look at this is that there's been almost three phases of how we integrate eutrepa into the standard of care, and I do think we can change the paradigm that currently exists and become the prostacyclin of first and best choice. So I think we'll do the new patient starts, then we'll do the transitions, and then, you know, if you just said what's the current inhaled tropostanol market, that's a $2 billion market opportunity and growing significantly still with the sort of white space that remains in PHILD. But then the other 2 billion today market opportunities, the oral prostacyclins, both of Travi and Arenatran, enjoy. And, you know, I don't think there's been any counter-detailing against those products to date, and we certainly are going to do that because they have significant off-target effects. There's quite some duress that the patients have to undergo to get to therapeutic doses and be maintained on those therapies that we think we can solve for because now for the first time, There's a readily titratable and durable inhaled prostacyclin called Utrepia. So those different promotional cadences will happen at different paces, but I think collectively we're gonna integrate ourselves across all of that. So when you look at what's the current market opportunity, it's really for a billion and growing. And I think there's opportunity for us to be very successful. We don't necessarily need to take incumbent share, but I think over time that will happen. Operator, we have your time. One more question if you have any. Thank you.

Our last question comes from the line of Ryan Deschner of Raymond James. Your line is now open.

Good morning. I'm curious on what you're anticipating in terms of the split between commercial and public payers in PAH and PILV. recently filed. Thank you.

Yeah, so Scott, if you'll answer the first part of that, and then Rusty, if you'll talk about the 494 litigation.

Yeah, from a pay rate standpoint, looking at the process cycling market and Tyvesia specifically, we think we'll probably have about 50% Medicare, about 35% commercial, maybe 10% Medicaid, and then 5% other, whether that be TRICARE, you know, DOD, et cetera. So that's We'll see when we get out there, but that seems to be what we're expecting.

Ryan, thanks for the question on the 494 lawsuit. We're not really going to comment much on that. Obviously, we filed the lawsuit. I think the complaint sets forth pretty clearly our thoughts as to the grounds on which we're proceeding with an infringement lawsuit. you know, that case is in the very early stages. So, again, can't really comment on timing and, you know, typical with past practice, won't comment, you know, publicly on legal theories or legal strategies we're going to pursue in the case.

Great. Thank you, Scott. Thank you, Ryan, for the question. So, with that, we'll close. And, you know, as you can see, we're very excited about actually matriculating our mission and vision of delivering a new treatment modality to patients with PH and PHLD. And hopefully as May 24th approaches, we'll be in touch and speaking with you all very soon. Thank you for your time this morning.

Thank you for your participation in today's conference. This does conclude the program. You may now disconnect.