Liquidia Corporation (LQDA) Earnings Call Transcript & Summary

Hello, everyone. My name is Ben Davis. I'm an associate with the JPMorgan Healthcare Investment Banking team, and I hope you're all enjoying your third day of the 2026 JPMorgan Healthcare Conference. It's my privilege to introduce Liquidia team. CEO, Roger Jeffs, will be presenting. Afterwards, he'll be joined by CFO, Michael Kaseta, on stage for a Q&A at the end.

Great. Thank you, Ben, and thank you for all of you here today. Also, thank you to those online who are listening in. I'm going to give the corporate overview really to give you a sense of kind of how we did in 2025. As a preview to that, we had a spectacular year, and also then give you a look into the future, kind of where we're headed above and beyond where we are today. Also, I want to say that I'm joined here today with Rusty Schundler, our General Counsel; Scott Moomaw, our Chief Commercial Officer; Jason Adair, our Chief Business Officer; and Rajeev Saggar, our Chief Medical Officer. Usual forward-looking statements, and I'll refer you to our SEC filings for the true risks and uncertainties. So for those of you who may be new to the story, Liquidia is a biopharmaceutical company driven by science and passion with patients as our North Star. And our purpose is to improve inhaled drug delivery to by employing proprietary and better formulations to focus on particularly the prostacyclin class of therapeutics to change and improve the standard of care for patients with high unmet need. What really centers us today in terms of success is the 3 Ps, the product, YUTREPIA, which is based on a proprietary PRINT platform. It was FDA approved in May '23 of '25 for PAH and PH-ILD, and it was launched and provided to patients within 1 week of approval, which at that point in time, we think was a record. We have a robust pipeline that supports and leverages the strength that we're building. L606 is a liposomal treprostinil inhaled suspension that provides extended release. We think, as I'll show you some data that will convince you, that it also has a potential for a better safety profile, which will allow us to dose to better effect. And it's also in a 2 times a day regimen versus current day therapies, which are 4 times a day. And it uses a rapid and portable modern-day [ mesgen ] nebulizer that's graft actuated. I'll update you today on progress. We have a number of new clinical studies that will fortify our position, which is what we feel with YUTREPIA is the leading and emerging leader in the inhaled treprostinil space. We're going to do studies that are going to treat inadequate responders to patients on other therapies, both inhaled Tyvaso as well as oral therapies like Uptravi and Orenitram, and explore the broader renaissance indications that are now available to us in IPF, PPF, PH-COPD and scleroderma-associated Raynaud phenomenon. So in terms of '25, how did we do? Again, it was an outstanding year for Liquidia. We achieved profitability in our very first quarter of launch, which I think puts us in very rare air for a biotech company in its first quarter of full sales. For the year, $148.3 million. And you can see that grew from third quarter '25 from $51.7 million to $90.1 million of net product sales, a 75% growth Q-over-Q. The result of that growth was that we provided $30 million plus to our bottom line in terms of positive cash flow and fortifying the balance sheet. Maybe spend a little bit of time again for those who may be newer to the story that PH and PH-ILD are massive market opportunities today and with room to grow. So if you look at on the left-hand panel, the prostacyclin market. This shows PAH and PH-ILD. There's inhaled $1.7 billion in annual revenue. Oral is almost $2 billion in annualized revenue and the parenteral IV subcu business is about $572 million. So in aggregate, $4.3 billion in net sales as it exists today. The treprostinil-based therapies represent about 62% of that or $2.7 billion. There still remain many, many more patients to be addressed, who have continuing unmet need. In PAH, and these are estimates, this is not meant to be particularly precise, but about 4,000 patients on inhaled, 10,000 patients on oral and 4,000 patients on IV subcu. PH-ILD, a much different story. A newer indication only first approved for inhaled treprostinil in 2021, but 6,000 patients on inhaled with, we feel 54,000 or more prevalent and untreated patients. So that is marginally penetrated. And these are numbers from before YUTREPIA launch with a lot of upside and room to penetrate and grow these markets and capture share within these markets. So here's our own product momentum, which, again, I think you'll view as outstanding and almost a linear escape velocity from day 1. So we launched in June and here's to 12/31. So launch to date, 2,800 new prescriptions or referrals, we'll call them both of that; about 2,300 new patient starts. And our conversion rate from referral to start is about 85% if you look at trailing average. That's unheard of for a new product launch where you face new-to-market blocks and other impediments in the early phases of launch. But you can see there's been a robust uptake for this therapy, even amongst the payers. So kind of a little bit of demographics on the right, where are these patients coming from? 3 out of 4 patients are starting YUTREPIA are new to prostacyclin therapy, so they've never had a background prostacyclin therapy. And 1 in 4 are transitioning from other prostacyclin therapies. So 25% transition, 75% new. So we think we are displacing other therapies in terms of being the prostacyclin of first choice. And obviously, 25% of our starts are representing that we're also taking share from existing therapies. One thing of note, we're not just taking it from the inhaled market in PAH. So in PAH only, there are oral therapy approvals, Orenitram and Uptravi. And you can see 30% of our 25% of switches is from the oral therapies. And this was a bit of a self-directed plan. It happened somewhat organically in the field. It wasn't something that we were pushing for in this initial period of launch. So very pleased that the product is so well received. It's being used beyond even where we're targeting our marketing efforts. So two things that will help us in the future. One is to drive depth of prescriptions and increase [ breadth ] of target prescribers. Most of our scripts today still remain in the PAH space versus PH-ILD. While there's a lot of room to grow in PH-ILD, prostacyclins have been used in PAH for over 30 years. So there's a lot of rapid adoption in the PAH community for a newer and better prostacyclin, and that's why you see the predominance of our scripts are coming from the PAH arena so far. But certainly, going forward and into the future, I think PH-ILD has a rapid chance to catch up. So if you see in the upper left-hand panel, most of the prescriptions are from the major centers, 24% are from the community, 76% from the centers. That's purposeful. We targeted the centers of excellence and the KOLs because we want to drive awareness among these big centers and have them be advocates for YUTREPIA. And I think as you've seen from the script and start rate, we're doing a very good job of that. Pulmonologists have written more prescriptions, not surprising, given it's an inhaled therapy. 63% of our scripts are from pulmonologists, about 30% from cardiologists, and the rest are from probably rheumatologists who treat scleroderma patients who have associated pulmonary hypertension. The interesting thing on depth is we're achieving not just breadth, so we have 750 prescribers launched to date. But within those -- that prescriber base, our average script rate is about 4 scripts per prescriber on average or 3.8 prescriptions per prescriber. We have 24% who've had actually had 5 or more -- 34%, 2 to 4 and 42% with 1. What we've seen is a bit of a, what we'll call it, a tipping point number of prescriptions. Once we can get the center to 3 to 5 scripts, they have a threshold moment where they then begin to see the true product differentiation. They see the expressed difference in tolerability and the ability to titrate and get to dose in effect, and they then tell us that they're going to port their entire patient base to YUTREPIA. So depth is critical among these major centers, and it's a focus of our commercial team in the new year. We are planning a number of additional high-impact studies to continue to build upon this product profile and its differentiated characteristics. And we do this without fear, and we're going to do it with a very purposeful manner. So in PH-ILD, last year at this conference, we spoke about the ASCENT data for the first time. That study remains ongoing. And what we've shown in that study, and I'll show you the data specifically in a minute, that it confirms the tolerability, titratability and 6-minute walk improvement as it relates to dose escalation in PH-ILD patients. That's a 52-week study. That study will terminate in Q1 of 2026, and we're going to present the 24-week data at the PVRI conference in January. The new studies for us are PH-ILD study. We're going to take patients on Tyvaso DPI and transition them directly to YUTREPIA. So that's a bold study. We'll take patients that are underserved on that product, so they can't get to dose, they can't get to effect. We'll move them to YUTREPIA, and I'm confident that we'll show that we can improve that patient's profile and response rate with YUTREPIA. The other study we'll do because as you heard, we're getting organic scripts from the oral transition -- the oral market. We're going to go ahead and do a study so that we have data to show the benefits of that and inform the community on how to best transition patients from oral to YUTREPIA. It's an open-label study. First patient in will be mid-'26. And again, same thing. We're going to take patients that are intolerant due to the significant GI side effects that those patients have, move them to YUTREPIA and show that we can benefit those patients as well. And I think once we have that data in hand, it will be the first time a company is counter detailed an oral and try to displace that share. And I think we can do that in a very effective way. The other study that's of interest is a PAH sotatercept combination study, which we will initiate in the second half of '26. That's an investigator-initiated study. There's emerging medical literature on the fact that there are highly complementary mechanisms between sotatercept and prostacyclins. So one thing that happens with sotatercept, very effective drug, patients are getting better. But one thing that happens in concert with sotatercept's administration on top of a prostacyclin is it's exacerbating the prostacyclin side effects that exist, so more headache, nausea and things like that. So those are systemic side effects. So -- and mechanistically, what that drug does is it reduces RV after load, decreases RV contractility, but does nothing to cardiac index or cardiac output. What prostacyclins will do is that they also can improve RV contractility, but we can improve the cardiac output. So we're an inotrope. So in addition to helping with the pressures, we can help with the function of the right ventricle. So these 2 drugs should work well together. What we have had occur in the field is when sotatercept has been started, those patients have improved to the point where patients and physicians wanted to remove the systemic prostacyclin, either oral or parenteral, because it was having exacerbating side effects. When they try to withdraw those therapies, they typically fail because, again, they need the contractility from the prostacyclin. What they have done is swapped out the oral or parenteral for YUTREPIA. So there's another opportunity. So across the full spectrum of oral inhaled and parenteral, we're trying to capture full share. And our marketing message, they're very simple. They're the 3 Ds. So it's enhanced deep lung delivery, ease of use with a low effort device and titration to higher therapeutic doses. Before we launched, these were aspirational, now the reality. So I think when you look across the 3 Ds, what the product is showing -- the product profile has shown is that we can deliver to the deep lung without exacerbation of cough because we don't deposit in the upper airway. We have an easy-to-use device with its low effort. It's a mono dispersed particle. We don't need any energy from the device to break it up, so it can be low resistance, and we can escalate these patients quite quickly to higher doses and to better effect. So the way we looked at accelerated dose titration again from the ASCENT study, which we've evolved since the first presentation of early data last year is looking at taking PH-ILD patients naive to prostacyclin and adding YUTREPIA. And just for -- to orient people on the left, the way that YUTREPIA is dosed is on breath equivalents to Tyvaso. We have 4 tablet strengths, 26.5, 53, 79.5 and 106. Basically, the way to think about that from a labeling standpoint is that's equivalent to 3, 6, 9 and 12 breath equivalents. And then we give the patients in this open-label ASCENT study dose over time. And you can see at week 24, our maximum dose was 424 micrograms, which is a 48 breath equivalent. Our median dose was 185.5 micrograms, which is a 3 to 4x higher comparable maintenance dose than what you see with Tyvaso. So the targeted Tyvaso dose is 9 to 12 breaths, and you can see we're extending well beyond that. And the advantage of that, it doesn't matter if you have an oral prostacyclin, an inhaled prostacyclin or parenteral prostacyclin, the higher the dose, the better the magnitude of effect. So for the first time, you're seeing an inhaled prostacyclin able to be dosed to higher levels and to better effect. So one of the things we were concerned about is as we escalate this dose 3 to 4x, do we then exacerbate cough in patients with PH-ILD? And the answer is no. So if you look at change from -- we use a simplified cough score to look at cough longitudinally. And if you compare from baseline to week 8, so we had gotten to 15 breath equivalents, no change, 1.3 to 1.3. Week 16, we've gotten to 18 breath equivalents, no change, actually a little bit down. And week 24, a similar picture, 21 breath equivalents and no change in the cough score. So what that's telling you PRINT works. The fact that we can make particles in the lower end of the respirable range and deliver them discretely to the lower airway is providing the benefit that we wanted. If you look on the right, again, we're now showing dose matters. Week 8, improvement of 21.5 meters, we increased the dose again, 31.5. Increased the dose again, and the outcome now is 41-meter change. So dose is doing exactly what you want in these patients. And we certainly think when we transition patients, for example, we'll be able to repeat this and dose those patients up. So we talked a little bit about broader indications, and this is truly a renaissance period for treprostinil, particularly by the inhalation route. So there was a successful study in IPF that read out quite favorably with very good improvement in forced vital capacity or FVC. We are going to do an open-label study with YUTREPIA in these patients, very similar to the ASCENT study, so that we can learn about does -- do the benefits that we've seen in PH and PH-ILD port to the IPF patients as well. We're confident that it will. In systemic scleroderma patients with Raynaud's phenomenon, that's a Phase II study that will start in the -- with first patient in late this year, really to look at the -- can we improve the digital vasculopathy that they experience with pain tingling and numbness in the fingers that can progress to ulcers and necrosis and amputation even. So a new arena for us, trying to get beyond just pulmonary diseases. And then in PH-COPD, we think there's tremendous opportunity if we select the right patient population, use YUTREPIA to its full benefit and move that product along. So what's nice about PH-COPD, it's a Phase III-ready program. We have work to do in terms of designing the protocol. We want to make sure that we get the right patients in the study to enrich the chance of success. But we look to start that study in 2027. So if you look at the benefit of opening of these new markets just in terms of the potential and numbers of patients that need inhaled prostacyclin treatment, this is what this slide attempts to address. So PH associated, we've talked about is about 18,000-plus patients today; PH-ILD, about 60,000 prevalent patients; scleroderma patients about 30,000; IPF patients, we estimate probably around 200,000 or more; and PH-COPD, about 300,000. So the market opportunities are below. And you can see if you just aggregate these up, and these are, again, rough estimates, but I think it doesn't take a lot to convince you that these are the opportunities in these large markets. They're approaching $20 billion in cumulative value. So one way that we're going to prepare for the newer markets is not just with YUTREPIA. So some of the studies that we're doing are to inform what we'll do next and what we'll then use is L606, which is our extended release liposomal formulation, which provides more consistent exposure over time, and I'll try to show you some of that here. So the left-hand panel looks at mean plasma concentration on the y-axis over time. This is Tyvaso in the blue. You give it 4 times a day. It has a 45-minute half-life. So you get a high peak to trough, but you get it 4 times a day. And then overnight from hour 16 to hour 24, you can see the drug is absent. L606 is shown below. This is an equivalent dose exposure, but given twice a day, and you can see that we lower the Cmax by sevenfold. Cmax is usually associated with the level and degree of adverse events. and then we'll dose it every 12 hours. And that's important because if you look to the right-hand curve, what we're trying to do is get as close as we can to a parenteral steady-state PK profile. And you can see with Tyvaso, hours 0 to 12. And L606, we get a similar exposure in total. And then from hours 12 to 24, you can see this is where the value of L606 will be. We'll provide an equivalent exposure in the 12- to 24-hour interval, which was in terms of comparison to 0 to 12 hours. So we won't be yo-yoing the dose and basically taking patients off drug every night. The advantage of that, obviously, is we want to try to keep these patients stable. So the other thing about L606, the data that we've been able to gain to date suggests that it could be the most well-tolerated inhaled treprostinil ever. So if you look at AE profile for cough, you can see 32.1% of patients had cough, but only 14% of that was deemed to be drug related. The typical cough incidence rate is -- or frequency is about 50%. So a reduced level of cough here and other things. So you're not seeing -- you're seeing very little systemic effects. But in particular, cough, which seems to be the hallmark problem, is mitigated. Also, because of that, a similar occurrence that we see with YUTREPIA in the sense that the median dose, these are patients studied over the period of a year, is about 19 breath equivalents, but we've gotten patients up to 378 micrograms, so up to -- beyond 25 breath equivalent. So if you look at the right-hand panel, a lot of patients are getting to higher doses with ease. So exactly what you'd want, but now in a twice-a-day format, not 4 times a day. And the advantage of that, again, we've studied in an open-label format. We already have data for patients over 48 weeks. The week 12 walk was a mean of 23.6, median of 17.2, week 48 median of 22.5 and mean of 29.4. So you can see a sustained benefit in walk, and these are patients transitioned from prior inhaled treprostinil use. And then the peak and trough, this is the critical point, like how did we do? Did we avoid any excursion in efficacy because we had a high peak to trough variance? And the answer is no, we did not. So at week 48, the trough was 24.3. And at week 48, the peak is 22.5. So exactly what you want to see in an extended release format. So under Rajeev's leadership, we are initiating the Re-Spire study. It's a Phase III multicenter, randomized, double-blind, placebo-controlled trial in about 350 patients using sites around the world. So more than 20 countries will participate. That study has been initiated, and the delivery device is a moderate -- when we -- it's a nebulizer because it's a suspension. But I want you to know that this is a modern-day breath actuated mesh nebulizer. This is not like the previous nebulizer that's available and was developed in the mid-2000s. This one is new. We use it. It's the FOX Mobile device, but there's experience with it, and it's CE marked and 510(k) ready. So at the end, to summarize, we also have the capital to do this work. So it's a lot of work, and we're trying to -- we're really trying to scale the company. We're scaling manufacturing. We're scaling our clinical programs. We're going to scale our sales force a little bit. So we ended Q4 -- 4Q '25 with $190.1 million in cash or cash equivalents. And our goal is to increase profitability quarter-over-quarter, and we certainly feel we'll be increasing our balance sheet quarter-over-quarter as well. So I thank you for your time and attention, and we'll open it up to questions.

So just a quick question to open up Q&A, was the rapid update of prescriptions and new starts surprising? And how do you see it continuing?

So I wouldn't say it was surprising because we understood well the deficits of the competitive therapy. We did have some aspirational goals in mind. And I think we've ASCENT showed us very clearly that we could -- in a patient population, the PH-ILD patients that have a predaliction for cough that we can minimize the impact of cough on dosing and therefore, the effect of the therapy. So we think we're pulling all the appropriate levers. We are more convenient, safer and more efficacious. And again, the only thing that's similar is it's 4 times a day. So I think those -- pulling those three levers the way we have has been impressive. I think there were some upside surprises. I think the oral transitions happening and as soon as they have, the fact that the addition of sotatercept is allowing deescalation or de-intensification of prostacyclin therapy off of these systemically tough products like parenteral or oral to YUTREPIA is critical. And if you look at the trajectory and pace, we've been consistent, sustained, and I call it the escape velocity because it's taken us to profitability. And if you just -- I think we -- it's not unreasonable, and it's our goal to be over $1 billion product in 2027.

So given that statement and the success so far, what would be your expectations for 2026 and 2027?

Yes. I won't speak specifically because I don't want to give you a forecast. But I think the bigger global statement is this can be a $1 billion product in PAH and PH-ILD, all things being equal as they are today. We just continue doing what we're doing. We get more prescriptions, we get more depth of prescriptions, displace oral. So I think one of the key areas for us, that's a $2 billion market today, not only to steal share, but as physicians look to add a prostacyclin, they now add YUTREPIA instead of an oral therapy, which they are doing, obviously now.

Congratulations on your success. How do you see the litigation? What's the status of that with United Therapeutics and how do you see that unfolding?

Yes, it's a focal point. Maybe, Mike, do you want to take that one?

Yes. Thanks for your question. The litigation where it stands right now is we had a trial in Delaware back last June. We went through an accelerated briefing period. We are, at this point, just waiting for the judge to rule. We are extremely confident in the arguments that we made. We strongly believe that we should win this case. We are prepared for any outcome. Our focus right now is really on the execution of everything Roger just talked about, the launch of YUTREPIA, initiating all these clinical programs and everything that goes with a successful launch. But we're prepared, we're confident, and we look forward to a ruling, hopefully very soon.

Yes. Great question.

So given the $30 million in positive cash flow, only 2 quarters after launch, which is quite impressive, how do you interpret that for what that may mean for 2026?

Yes. So I'll start and then Mike will turn to you. So again, we think we're going to have a -- we have a goal to increase profitability. And I think clearly, if we stay on the trajectory that we're on, and I don't see any impediment to not doing that, then I think we'll continue to build our balance sheet.

Yes. I mean I'd just say the fact that 7 months into launch, we're a cash flow generating company, I think, is a testament of our execution and also puts us in a position to be able to continue the successful launch. As Roger said, we can invest further into the launch by expanding our sales force. We can double our capacity to manufacture supply, which is our plan for 2026 to open a new facility in 2027, and then also start these multiple programs that are targeted to increasing the product profile of YUTREPIA, getting L606 into the clinic for our global Phase III and also looking at these new indications. So as Roger said, growing profitability while investing into this -- into all of these opportunity with the cash that we have on hand without the need for additional capital, I think, really is a testament to our success in 2025, and we look to continue that in '26 and beyond.

And I think, Ben, maybe I'll add. I think just to put this in perspective, how well we've done, the competitor said they have about 10,000 patients on an inhaled treprostinil. In 7 months, we've added 2,800. So we're at about 25% share in 7 months. They launched their nebulized drug in 2009 and their DBI in 2022. So in a very short period of time, we have rapidly captured a large share, particularly of new patient starts, and we've also transitioned some of the share. And I don't see any way -- any reason that wouldn't continue.

And when might you have the first look at the data coming from some of the new studies you've outlined?

Yes. And we tried to put a little bit of a soft timeline here. So we try not to be too descriptive. I think the goal is to get them started because they're open label, and we're going to take -- like we did in the ASCENT study, we took sort of snapshots of data at different periods of times, 8, 16, 24 weeks. We'll do that again here. So we could show data late this year. But again, just to soften it and just say sometime in '27 would be a reasonable expectation.

All right. Well, thank you very much for your time.

All right. Thank you, everyone.