All right. I'm Ben Burnett, biotech analyst at Wells Fargo. I'm pleased to be here with the Liquidia management team. We have Mike Kaseta, CFO, and Jason Adair, Chief Business Officer. Thank you all for doing this.
Hey, thanks for having us, Ben.
Morning, Ben.
Why don't we just kick this off and just start by giving us a quick overview of Liquidia and kind of the near-term sort of events that we should be focused on.
Yeah, Ben, really appreciate you having us. It is a really exciting time for Liquidia. Our first product launch, YUTREPIA, has gotten off to a very important start for us. Just in Q2 alone, we recorded $170 million of revenue, all while increasing our profitability quarter-on-quarter. We launched this product back in May of 2025. We have had a rapid uptake. We have been on a linear curve of patient additions, and feel that we are taking a disproportionate share of new patient starts in the space in both PAH and PH-ILD. We are very excited for where we are going.
This is a very important time for these patients that have suffered for a long time, to be able to have a product that has the tolerability profile, and ease of use profile that really helps, and ultimately allows us to look at other opportunities, where we are now pursuing our next generation product, L606, where we have launched our phase III placebo-controlled trial in PH-ILD, enrolled our first patient in Q2, and looking forward, where this time next year we will have between eight and 10 clinical trials in place across a variety of disease states, both open label phase IV studies and also potentially two additional registrational studies in PH-COPD and systemic sclerosis with Raynaud's phenomenon.
We have created a tremendous amount of value already, but we feel like we are really just scratching the surface here of being able to do much more while increasing sales of YUTREPIA, increasing profitability, and reinvesting part of that profitability back into our pipeline. It is a great time for Liquidia, and we are really looking forward to the future here.
Okay, fantastic. Why do not we start with YUTREPIA in the ongoing launch, and maybe we can kind of start on the adoption side. You have seen some strong adoption over the last couple of quarters. Going forward, where do you see that kind of adoption curve and ramp going?
Yeah. So like we said, every metric that we have tracked at the launch has been ultra successful in both PAH and PH-ILD. What we have seen since the launch, we have really seen an even split of scripts in both PAH and PH-ILD. We are seeing about 75% of our scripts are coming from naive patients, which is really market-growing scripts, which we are very excited about and 25% are coming from switch from other prostacyclins like TYVASO DPI, Remodulin and oral prostacyclins. As we look forward to the rest of the year and beyond, as I said, we are at $170 million of revenue in Q2. We have for the last three quarters, grown $40 million revenue each quarter. We see that continuing and our goal and our plan is to be on a billion-dollar run rate of $250 million by Q4 of 2025.
The opportunity in PH-ILD, I think we have talked about this time and again, that this is still a relatively under-penetrated disease state, that we believe is probably penetrated less than 20%. So there is still a tremendous amount of white space for us to pursue there. We have increased the size of our sales force in the last quarter by 33% to help penetrate that area, especially in the local community setting. But then in PAH, we think there is an equal opportunity through new patients, but also we are very interested in switches of oral prostacyclins. There is over 10,000 patients that are on oral prostacyclins currently. We feel the flexibility of the product profile of YUTREPIA is very conducive to switch those patients.
We are about to enroll an open label study in oral prostacyclin switch patients, which we are very excited about and hope to have data in the first half of 2027 on that. While the early launch has been extremely successful, we feel like there is still tremendous opportunity to grow and grow for the foreseeable future here.
Okay. That is great. Maybe could you talk about, now that you have multiple quarters kind of under the belt, what has been sort of the physician feedback and sort of why are you seeing kind of the adoption in those kind of segments that you saw, the new patients and the switches?
Yeah, I think for years especially in PH-ILD, only TYVASO and TYVASO DPI were available for patients. I think what YUTREPIA brings is a differentiated product profile that provides dosing flexibility to allow patients to titrate to higher doses. Our PRINT technology, which is really the cornerstone of what our differentiation is, where we manufacture particles of uniform size and shape that were specifically designed to bypass the back of the throat and the upper airways, to achieve deep lung deposition. Our ability to titrate to those high doses we feel is critically important and what we've seen, we had launched in 2024 a study called ASCENT, which was an open label study in PH-ILD, where the goal was to take PH-ILD patients and start them in YUTREPIA and try to titrate them to higher doses in a very short period of time.
What we saw in that was very telling. Our drug, the target dose of TYVASO has always been between nine and 12 breaths of TYVASO. What we were able to do with ASCENT is show that you can get to high doses in that relatively short amount of time. So we really measured at eight weeks, at 16 weeks, and 24 weeks. At eight weeks, we were able to get to the equivalent, on average, of 15 breaths of TYVASO, and achieve a 21-meter walk improvement. At 16 weeks, we were able to get to the equivalent of 18 breaths of TYVASO and saw a 31-meter improvement.
At 24 weeks, we were able to get to the equivalent of 21 breaths and see a 41-meter improvement, all while cough, which at the time of initiation was really deemed as more of a minor transient cough, that cough stayed the same, even though we were getting up to that 21 breath. So the feedback we're getting from doctors is really the consensus is unanimous here that we offer a profile that is easy to take, dry powder inhaled, and we're able to titrate to higher doses, which we think has been a welcome addition to the treatment paradigm for these patients in both PAH and PH-ILD.
Going back to some of those other comments, when you were talking about the forward-looking sort of growth rate, do you see that growth being different in PAH or PH-ILD? Maybe could you just talk about what is your current thinking on the commercial opportunity in those two categories?
Yeah, I think as I said earlier our split has remained extremely consistent since launch against PAH and PH-ILD. Now, if you look at the overall market, if you back out and you look at the overall market opportunity, I think if you look at PH-ILD, we've said we believe that addressable market could be 60,000 patients or more. As we've all learned, relatively under-penetrated. I think if you think about it long term in PH-ILD, the long-term opportunity in PH-ILD is definitely larger. We've said previously that we think that could be a $3 billion, $4 billion, $5 billion market opportunity, which was, again, part of the reason that we've increased the size of our sales force.
A lot of these patients are sitting in local community settings where the first product approved in PH-ILD was back in 2021, so it's still relatively under-diagnosed, working very closely with doctors to help them identify patients, diagnose patients, and ultimately either treat patients or refer those patients to the academic centers. If we think about long term, I think the PH-ILD opportunity is very large. The PAH opportunity is a different opportunity in the sense that it's a mature market. If you take the oral prostacyclin market, the inhaled prostacyclin market, and the parenteral market, that market is well over $3 billion right now. We believe that we have access to that entire market as we move forward. As I said, we're doing a study in oral switches.
We are going to do a study that we plan to kick off in 2027 that shows a patient that is on WINREVAIR that we can move from parenteral treatment onto YUTREPIA. These doctors, these physicians, these patients want to see data, and that's what we are pushing towards, to generate as much data as we can in both PAH and PH-ILD. While the long-term opportunity in PH-ILD, I think is probably larger, I think the short to medium-term opportunity in PAH is still massive and could easily be in excess of $2 billion.
Okay. That's awesome. When we think about the most important kind of patient segment, at least near term, is it the treatment treprostinil-naive category that's sort of driving the most growth?
Yeah. What I'd say is those prostacyclin-naive patients, which as I said, is representing about 75% of our new patient scripts, new patient starts, I believe that's going to be the larger opportunity. In PAH and especially in PH-ILD, these patients are churning a lot. A lot of new patients are coming into the system every year. Those scripts ultimately will be market-growing opportunities. I would expect as we move forward to maintain that ratio of 75/25. But the overall opportunity in PAH, in PH-ILD, naive switched patients, I think is going to allow us to sustain the growth that we've been on really since the launch.
Okay. Excellent. And maybe thinking about the sort of price side, net pricing kind of long term, where do you see those discounts leveling out?
Yeah. We've not disclosed our specific GTN. I think where we have been, we feel very comfortable where we are. One of our goals at launch was to make sure that patients have an opportunity to choose YUTREPIA if they want to. And in order to achieve that, we needed to make sure that we were not disadvantaged at all with payers. We have an amazing market access team, and what I'd say, where we sit here now in September of 2026 is we are at parity. We are not disadvantaged whatsoever. We're very comfortable with where we sit. We feel a combination of that along with what we feel is a better product profile really positions us well for future growth. What I would say as we move forward, we believe that from a pricing point of view, we feel very comfortable.
I think like most products, you could see a slight erosion in price as years go by, but I don't see anything significant and feel very confident with where we are.
Okay. Excellent. I think we've kind of been waiting on a day-by-day basis for this legal update for what feels like over a year now. I think it is over a year.
14 months.
But who's counting? Yeah. I understand this is a hard thing to speak to specifically, but what can you say about this? I guess in a lose scenario, how should we think about that?
What I would say is what we're focused on is the here and now, and focused on commercial execution, focused on our pipeline and all of the studies that we talked about earlier. We can only control what we can control, and right now we have our plates full with servicing these PAH and PH-ILD patients, which we will continue to do unless we're told otherwise. The situation on legal, like you said, the trial was in June of 2025. We said publicly this time last year on this stage with you that we are expecting a ruling anytime, and we're now sitting here a year later. We don't know when it's coming. What I will say, like any good management team, any good company, we will be prepared for any scenario that is put in front of us.
We believe that we should win this case, but there are no guarantees. Whatever that decision is, we will be prepared. We will be prepared to act quickly, because again, what is most important is that patients are treated fairly. We want to make sure that there is no disruption. Again, we believe we should win the case, but we will be prepared for any outcome and we will be prepared to act quickly.
Okay. Excellent. I think another interesting story that played out over time is we have seen this PH-ILD use case for treprostinil expand. I want to go through some of these different expansion opportunities one by one, but starting with sort of the IPF and the PPF opportunities. How do you see those markets and how do you get into those markets?
You are right, Ben. It is an exciting time. When we started this program in 2016, it was hard to convince any investors that inhaled treprostinil was going to matter, and luckily, we put YUTREPIA into the clinic, and we are now in the market. At the same time, the investment community has seen what the medical community sees, which is what drives the best use of treprostinil or prostacyclin is the local delivery, right? Can we get to higher exposures in the most tolerable way in the convenient manner? If you can optimize those first two for dose range and tolerability, there are new indications that can be available in the future. One of those is IPF or PPF, progressive pulmonary fibrosis. What we see in the data that has been published is clearly a signal that there is a benefit to patients.
If you go back to that equation about exposure and tolerability, YUTREPIA has shown that we can dose very high in a tolerable way. The next step for Liquidia is to do an open label study in PPF and IPF patients to look at that exact question. Like ASCENT, PH-ILD patients, which included IPF patients. Can we dose to higher levels? If we can we get better efficacy in a tolerable way? If we can show that in an open label study, then we can take that knowledge into our clinical development plan either for L606 or YUTREPIA. But we need to prove the hypothesis first that dose matters. We believe that it does, as it has in every other indication that it has been studied in.
We don't want to make any promises about what that looks like in the future, but you should know about Liquidia. We're always going to do the right science, right? We're going to make decisions that are informed by data, and we're looking forward to that data.
Okay, excellent. That study will give you information on both IPF and PPF.
Correct.
Okay, excellent. Then another study that I think you've talked about recently is in PH-COPD. Talk about that opportunity. I guess first of all, how big is that commercial opportunity?
Yeah. PH-COPD is very attractive. We know that it's been studied in the past with inhaled treprostinil. The challenge with COPD, millions of patients. If you have PH on top of the COPD, it can be lethal depending on the level of PH that you have. There is a GoDeep registry where there's information that indicates that if you have a PVR above 5, that your three-year survival rate is less than 20%. The challenge for the study is to define the level of COPD in a way that doesn't complicate the interpretation of the effect on PH. The balancing act is designing the inclusion and exclusion criteria appropriately, and our Chief Medical Officer, Dr. Rajeev Saggar, has been working on this extensively over the last year, and we feel really confident about the trial design that we're working on.
The intent is to start that trial in the end of 2027. It is a big lift going quickly. We do have the benefit of some learnings from the past study. Again, it is really defining the clinical trial appropriately. We believe in the mechanism. We know that inhaled treprostinil treats pulmonary hypertension. The question is how do you design a trial to give you the cleanest read on that signal? In terms of total population, as we think about defining it could be upwards of 300,000 patients in the U.S.
Okay. That is great perspective. I also wanted to ask about Raynaud's phenomenon associated with systemic sclerosis. What is the hypothesis there for treprostinil?
Again, we are benefiting from previous studies that have looked at prostacyclins in this disease, so specifically scleroderma, rare disease, patients with a tissue-based disease. We are looking at Raynaud's, which is one of the most common, I do not want to call it side effects, but one of the most common phenomenon that these patients have. Like 90% of scleroderma patients have secondary Raynaud's. The theory is that with a potent vasodilator, you can get to levels that can expand or make the blood flow better in the digits, especially when we think about the fingers.
The issue has been this exposure tolerability equation. We know that with inhaled treprostinil, we can get to high levels in a tolerable way. The study that we are going to kick off in the next month is going to be looking at that dose finding. Can we get to a dose where we believe that we can affect or drive exposure into the digits that have an effect potentially on the attack rate? When we move forward from that study, it will go directly into a pivotal. It is a quick study, so we are going to start it this year. The phase II dose finding will be done in the beginning of next year, and if it is positive and we have a good dose response, we will take that right into a pivotal study at the end of next year. It is a very quick study.
One of the reasons that we're confident is if you just look in Europe, infused iloprost, another potent prostacyclin analog, is currently used to treat patients with Raynaud's. But that's a very cumbersome and painful product to deliver. Again, we believe high dose exposure with YUTREPIA in a tolerable way might get us to similar levels that's more attractive for patients to dose in the U.S.
Okay. Excellent. So data, you're saying first half of next year or potentially next year?
The phase II study that we're going to kick off, we'll have that in the first half of next year.
Okay. Great. I want to spend a little bit of time also talking about L606. I guess, what is the advantage of L606 relative to YUTREPIA in your view?
Again, we go back to that kind of equation, exposure drives efficacy, tolerability drives durability, and then convenience drives compliance. When we found L606 from our partner, Formosa in Taiwan, what they were working on is the tolerability component, right? Because when you look at 30 years of development here, tolerability has driven a lot of the innovation. What we see that L606 brings from YUTREPIA is not the exposure. YUTREPIA is achieving very similar exposures to what L606 can do. But the tolerability profile of L606 is vastly improved. It's a liposome, so treprostinil, a known topical irritant, is protected in the inside of that liposome. As a patient inhales that twice a day with a nebulizer, it's really shielding the upper airway.
That tolerability profile showed up in our open label study in the U.S., where if you looked at cough, which is a signal that people pay attention to, at 48 weeks, we saw 14% of patients reporting a drug-related cough at levels that would be equivalent to the median dose was above 19 breaths per session comparable to TYVASO. I know I just threw some numbers out there.
Yeah. Walk us through that math. That's interesting.
Yeah. I know we've talked about this. With all of the competing products, it's starting to get hard to compare exposure and tolerability. The best way that we talk about it is comparable doses of TYVASO. TYVASO nebulized has been in the market for over 15 years. The medical community is thinking about dose titration as breaths per session, with the target of being nine to 12 breaths per session. When we look at YUTREPIA dosing and L606 dosing, we calculate what the comparable dose is relative to that standard. What we've seen in YUTREPIA and L606 is that we can achieve levels that are two and three times as high as that target TYVASO dose.
When you ask me a question of what is the median dose that we saw in our 48-week study of L606, the median dose was comparable to 19 breaths per session, four times a day. But we are doing that in two sessions, one in the morning and one at night. It is not just that the exposure is higher, it is that we are also smoothing out that exposure over 24 hours. If you believe that the parenteral is the most effective way of delivering a prostacyclin analog, then delivering that second dose right before the patient goes to sleep ensures that you are treating the disease while they sleep. You are also bringing Cmax down with two different doses and spreading out that AUC over 24 hours. We believe that that twice daily dosing with the liposome is the reason that we are seeing a better tolerability profile.
And we are encouraged that most patients could titrate to very high doses, and I think we actually had 21% of patients above a dose that would be comparable to 30 breaths per session of TYVASO nebulized.
Okay. That is super interesting. And I think you kind of spoke to this, but I guess what matters the most? Is it Cmax? Is it AUC? And I guess I am kind of asking this in the context of some of these kind of QD formulations that are in the works with a couple of competitors.
Again, when we think about parenteral, which was the first form, it was exposure over 24 hours that drove it. And everything since then has been this optimization question of tolerability and efficacy. We think it is AUC, and we are going to have to demonstrate that in a clinical study. That is our INSPIRE study, a placebo-controlled study. But we already know from our open label study that patients are effectively treating their disease with this twice daily exposure. That is with a seven times reduced Cmax when we compare it to the nine breath per session. When it comes to the other QD formulations, ultimately the clinical data will tell us what is best. But if you believe in the hypothesis that more continuous exposure over 24 hours is what can drive efficacy, then at least L606 has a chance to further improve efficacy.
We don't have to do that for approval. We just have to run a placebo-controlled study to show that we don't lose the efficacy that's known with the inhaled route. But is there a potential to improve it? We'll have to wait and see. We know that patients like our product since most patients have stayed on drug in that open label study.
To that point, can you talk about what is the right development path for L606?
We have the benefit of designing a program to take advantage of the decades of experience of prostacyclins with treprostinil and the different routes. So, when we went to the agency, we went with that full data package. So really what our design is now is to prove that we don't lose any efficacy in a twice-daily formulation since we have changed the PK. But the benefit of the history can all be applied in the package itself.
Okay. I guess, is it fair to assume that where we see YUTREPIA gaining traction and showing success, will L606 follow in those footsteps?
That's the right way to think about it. We believe in the inhaled route for prostacyclin, so wherever inhaled prostacyclin goes is an opportunity for L606. It becomes a sequencing issue for the business, as we think about what trials to do with YUTREPIA versus L606. Currently our focus is use L606, get it moving forward in PAH and PH-ILD. The INSPIRE study is a single study in PH-ILD, but the FDA has indicated we can get both indications with that study given the history that I mentioned. A lot of the other studies that we're kicking off are using YUTREPIA, because it's known, it's in the market, it has a lot more data. It's very easy for patients to take. It's dosed four times a day.
But that could be a potential benefit when we look at some of the things we're studying, like how do you transition patients from oral to inhaled? How do you transition a patient from IV treprostinil, who might be on sotatercept, to an inhaled formulation that's much more attractive for patients to take? We're going to look at real-time hemodynamics with some studies that we're planning. We're actually going to look at hemodynamics as we push the dose, because when you think about the last 15 years of history, where we haven't fully explored is if you can dose higher safely, what is the potential of this mechanism? It could be much broader. So that's why we're looking at things like PH-COPD, and we're going as fast as we can. Nothing's really stopping us, and with the financial picture that Mike mentioned, we're funding all this on our own.
I don't want to come back to that point, but just one more on just the development of L606. So you have a phase III study up and running now. Talk about the timelines there, and when can we expect to learn about that?
We believe we'll have the top-line data in 2029. As most phase III studies, it's driven by the enrollment rate, and it's a global study. So, we're going to be in more than 20 countries. It's roughly 350 patients in a one-to-one randomized controlled trial against placebo. We know there's some other trials out there that are competing, but there's a couple benefits that we think that we have. One, strong data coming from the 48-week study. That's the first. The second is the fact that by going global, we're really addressing a problem that has been left unaddressed with the past inhaled formulations. There's a lot of patients out there that still need to benefit from inhaled formulations, especially in PH-ILD. The last thing I would say is it is a nebulized product.
We are using a device that is very familiar to physicians outside the United States. The device that we are using is in partnership with a company called Vectura. That particular model, a version of that model was already used to treat PAH with iloprost. When we show up to a clinical site and say, "This is our product profile, this is our device," they have a very high degree in confidence that patients can use that device.
Okay. For both of these drugs that are based off treprostinil, I think there is a lot of focus just going back to IPF for just a moment. With TYVASO potentially going there, and could there potentially be an exclusivity around that asset? I guess, how do you think about navigating that scenario in IPF if that happens?
Yeah. It is a question that we are getting. We know that TYVASO, if approved, will have orphan drug designation, and that would come with seven years of exclusivity in the United States. We are going to let the clinical science dictate what our path is. The next step for us is to confirm our hypothesis that dose drives improved efficacy. First, we got to make sure it is tolerable, right? That is the main goal is in a IPF and PPF patient, if we push the dose to higher levels, can they tolerate it? We believe the answer is yes, because we are treating those patients already that happen to have PH-ILD.
If we are successful in showing that there is a dose response, I think that then becomes the question, Ben, is how do we use that information to think about the next study that we would do to either use it with YUTREPIA and figure out how to bring that to market with a label claim, or more likely is how do we put that into an L606 study, right? Because it is competitive. So, we are going to have to find a way to differentiate everything, not just based on the formulation, but if we can show that with our dosing, we can get to improved efficacy, that is pretty exciting.
Okay. Excellent. If there are any questions from the audience, just raise your hand and I will work it into the conversation. But Mike, I want to ask you, as you have expanded investment into the sales force and now as L606 is advancing and we see a range of other studies coming up on the pipeline, how do you think about just the business overall? Is the focus on just revenue growth, or is there an eye towards maintaining profitability?
Yeah. So, as I had said earlier, if you look at our Q2 results, we generated about $100 million of EBITDA, about $80 million of net income, and also we added $60 million of cash to our balance sheet. One of our foundational principles is as we grow YUTREPIA, obviously we want to grow the top line. We want to treat as many patients as we can. But we also want to grow profitability. So, while we are reinvesting, as you said, we are reinvesting back into our sales force. We are increasing our patient support services for YUTREPIA. We are clearly investing in R&D across all of these studies. The plan is to do that while increasing our profitability as sales increase. Just to give you an example, Roger has previously stated at this past earnings that we believe 2027 sales will be more than $1 billion.
We believe we can drop over 50% of that as additional cash on the balance sheet. So, for a company that is still relatively early in its commercial stage, to be able to generate that type of profitability and positive cash flow, I think really separates us from a lot of other companies and ultimately gives us the flexibility to where we believe additional investment is important or needed. We will not hesitate to do that, but we absolutely have an eye towards maintaining and increasing profitability quarter- on -quarter.
Okay. Excellent. Well, I think that is it for me. Anything to leave us with?
Listen first, just really appreciate you having us. We're very excited for where Liquidia is heading. We feel YUTREPIA is just scratching the surface of what we're capable of doing, and very excited about our pipeline as we move forward here. We'll have a lot of additional information as we move forward. As Jason said, I think we'll have a lot of data as we move into 2027 and beyond. As I said, we feel very confident in our ability to continue to grow on the same trajectory that we are on right now for YUTREPIA. Very excited and really appreciate you having us.
Okay. Thanks so much.
Thanks, Ben.
Thank you, Ben.