Liquidia Corporation

Good morning, and welcome, everyone, to the Liquidia Corporation Full Year 2024 Financial Results and Corporate Update Conference Call. My name is Shannon, and I will be your conference operator today. Currently, all participants are in a listen-only mode. Following the presentation, we will conduct a question-and-answer session. Instructions will be provided at that time for you to queue up for your questions. I would like to remind everyone that this conference call is being recorded. I will now hand the call over to Jason Adair, Chief Business Officer.

Thank you, Shannon. It's my pleasure to welcome everyone to the Liquidia Corporation Full Year 2024 Financial Results and Corporate Update Call. Joining the call today are Chief Executive Officer, Dr. Roger Jeffs, Chief Operating Officer and CFO, Michael Cassetta, Chief Medical Officer, Dr. Rajiv Sagar, Chief Commercial Officer, Scott Mumma, and General Counsel, Rusty Shumla. 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 achievements. 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. Roger?

Thank you, Jason. Good morning, everyone, and thank you for joining us today. We believe that 2025 has the potential to be a transformational year for the company as we further build upon the success of 2024, which resulted in the broadening of the tentatively approved label for Utrepia to include both the treatment of patients with pulmonary arterial hypertension, or PAH, and pulmonary hypertension associated with interstitial lung disease, or PHILD. There are four strategic imperatives that will drive our success and growth, both in the near and long term. These are seeking to obtain final approval and launching eutropia in both PH and PHILD as soon as possible after expiry of the gating clinical exclusivity on May 23rd, or just 65 days from now. Secondly, to continue to advance the clinical profile of Eutrepia to position it as not only the best-in-class inhaled prostacyclin, but also the first-in-choice prostacyclin for all patients in need of an oral or inhaled prostacyclin who remain underserved by current alternatives. Third, building upon our commercial and medical prowess to compete fiercely for capture of material and enduring market share in this multibillion-dollar and growing market segment. And finally, to further evolve our mission of being a leader in the pH space by continuing to leverage our development expertise to advance treatment options that have the potential to further improve the lives of patients, like our Phase III Ready, NextGen, sustained-release liposomal therapy, L606. I'd like to take a few minutes to expand on each of these strategic goals. With regard to the first imperative of seeking to launch Utrepia into the marketplace as soon as possible, I'm happy to say that based on the favorable legal decisions over the last few years, there are no legal barriers that currently impact the FDA's ability to issue final approval of eTrepia after May 23rd, when the exclusivity granted to our competitor will expire. It's also important to remember that when the FDA issues a tentative approval of an NDA, it means the NDA has met all requirements for approval, but cannot re-approve due to existing legal or regulatory barriers. In our case, the sole barrier identified by the FDA was the new clinical exclusivity granted to Tyvesa DBI, which expires on May 23, 2025. As directed in the tentative approval label, we plan to submit requests for final approval in the coming weeks. With regard to the second imperative of continuing to advance our studies to establish the clinical profile of eutropia, the medical community's interest in eutropia is increasing, with particular interest in the data being generated from our ASCENT trials. the open-label safety study of your TREVIA and PHIP patients. I'm happy to report, excuse me. Sorry, I have a call this morning. I'm happy to report that we're on track to complete enrollment in the coming weeks with 50 or more patients in total to be enrolled. We continue to be encouraged by the positive patient response to escalating doses of eutropia. As a reminder, at the JP Morgan Healthcare Conference in January, we showed the dosing and tolerability profile for the first 20 patients that completed eight weeks of treatment. We noted that the PHLV patients on eutropia were able to titrate to doses that are three times the comparable therapeutic target of nebulized tovesa. And today, we're happy to share some new information from the ASCEND study. We've also been looking at exploratory measures of efficacy One such measure is the change in six-minute walk-death test at week eight. We are pleased to report in this same 20-patient cohort, the mean change in baseline improved by 26.4 meters. While it is difficult to draw strong conclusions from cross-study comparisons, it is worth noting that in the phase three registrational study of nebulized toveso in PHIV patients, the INCRE study, active group patients had an observed mean improvement of six-minute walk distance of 15 meters at week 8 and 22 meters at week 16. We are highly encouraged by this early efficacy data from ASCEND as it demonstrates that eutrophia supports the key therapeutic attributes in PHLD we aspired for it, specifically being well-tolerated and amenable to rapid dose escalation to doses well beyond historical standards, leading to an accelerated therapeutic outcome that is in line or better than published results of existing therapies. And it should be noted that this data is especially compelling when compared to published data from Tyveso DPI and a like sample of treatment-like EHILD patients at the National Jewish Health Center that we have discussed previously, where 59% of patients discontinued therapy after a median time of only 40 days, with drug-related AEs, especially cough and clinical worsening, listed as the primary reasons for discontinuation of Tyveso DPI. We looked We look forward to highlighting a more robust data set from the ASCENT trial at the ATS International Conference in San Francisco this May. With regard to our third strategic imperative to build upon our commercial and medical prowess, we have built a commercial enterprise that we feel is best in class. Our team has been in place for over a year and a half and continues to support the use of troposomal injection while also reinforcing relationships with our healthcare providers and our understanding of the unmet needs of PH and PHLD patients throughout the country. We are prepared to provide a seamless service to patients and providers upon launch of eutropia and look forward to educating the PAH and PHLD communities on the favorable and potentially game-changing product attributes of eutropia. Lastly, as we look at our fourth imperative to innovate and develop better therapeutic options, we continue to advance L606, our sustained release liposomal formulation of troposinol, that provides more consistent 24-hour drug exposure in two 12-hour dosing segments, including during sleeping hours. We know that continuous infusion has shown the best efficacy with prostacyclin analogs, and we believe that the PK profile of L6S6 may provide the next closest non-invasive approach to maximizing the benefit from the more targeted inhaled route of administration. It is for all these reasons and with an eye towards our pending launch of Utopia in the coming months, that we decided to further strengthen our balance sheet, as announced yesterday, with an extension of our value partnership with health care royalty partners that Mike will speak about now, along with an overview of our 2024 financials.

Mike? Thank you, Roger, and good morning, everyone.

Before we review our full year 2024 financial results, I'm happy to highlight yesterday's announcement regarding expansion of our financing agreement with Healthcare Royalty Partners, which will provide liquidity up to an additional $100 million of financing in three tranches, including the $25 million tranche funded at closing. We are grateful for the trust, commitment, and confidence that Healthcare Royalty Partners has demonstrated over the years And we are optimistic that these proceeds and a successful launch of EUTREPIA following the expiration of exclusivity this May could lead to our reaching profitability without the need for additional capital. Turning to our full year 2024 financial results, which can be found in the press release, you will see that revenue was $14 million for the year ended December 31st, 2024, compared with $17.5 million for the year ended December 31st, 2023. Revenue related primarily to the promotion agreement. The decrease of $3.5 million was primarily due to lower sales quantities driven by limitations on the availability of pumps used to administer tropostomal injections subcutaneously. Sales quantities will continue to be impacted or at risk until alternative pumps are available. Cost of revenue was $5.9 million for the year ended December 31st, 2024, compared with $2.9 million for the year ended December 31st, 2023. Cost of revenue related to the promotion agreement as noted above. The increase from the prior year was primarily due to our Salesforce expansion during the fourth quarter of 2023. Research and development expenses were $47.8 million for the year ended December 31st, 2024, compared with $43.2 million for the year ended December 31st, 2023. The increase of 4.6 million or 11% was primarily due to One, a $6.1 million increase in expenses related to our L606 program. Two, a $5.3 million increase in expenses related to euthyrepia research and development activities, including the ASCEND trial. Three, a $5.1 million increase in personnel expenses, including stock-based compensation, related to increased headcounts. And four, a $3.5 million upfront license fee due to Pharmosa for the exclusive license in Europe to develop and commercialize L606 recorded during the year ended December 31st, 2024. Offset by one, $5.1 million in lower commercial manufacturing expenses reflecting the impact of expensing eutropia inventory costs in the prior year. And two, a $10 million upfront licensee due to Pharmosa for the exclusive license in North America to develop and commercialize L606 during the year ended December 31st, 2023. General administrative expenses were $81.6 million for the year ended December 31st, 2024, compared with $44.7 million for the year ended December 31st, 2023. The increase of $36.9 million, or 82%, was primarily due to one, A $19.7 million increase in personnel expenses, including stock-based compensation, driven by higher headcount and expansion of our sales force in the fourth quarter of 2023. Two, a $7.9 million increase in legal fees related to our ongoing EUTREPIA-related litigation. And three, a $6.8 million increase in commercial expenses in preparation for the potential commercialization of EUTREPIA. In summary, we incurred a net loss for the 12 months ended December 31st, 2024 of $130.4 million, or $1.66 per basic and diluted share, compared to a net loss of $78.5 million, or $1.21 per basic and diluted share, for the 12-month ended December 31st, 2023. With that, I would now like to turn the call back over to Roger.

Thank you, Mike. As you can see, we're well positioned clinically, commercially, and financially for the potential launch of our first product based on our proprietary print technology, which will serve as the growth engine for our continued evolution and inflection. I would now like to open the call for questions. Operator, first question, please.

Thank you. As a reminder, to ask a question, please press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again. Our first question comes from the line of Julian Harrison with BTIG. Your line is now open.

Hi, good morning. Thank you for taking my questions. Roger, you touched on this a little in your prepared remarks, but just wondering if you could talk more about the administrative sequence and timeline to convert eTrepia's tentative approvals in PAH and PHILD to full approvals.

Yeah, so as we said in the opening, thank you, Jillian, and great to talk to you. As we said in the opening, we plan to request final approval in the coming weeks. In the tentative approval label that we received in August, it was recommended and suggested that we apply for final approval either two or six months ahead of the action date, which would be May 23rd, given we view this as a Class 1 resubmission because no new data will be required for approval. We have been in concert with the agency working to deliver that letter around the March 24th timeframe so that we can be granted final approval around May 23rd. And Arno, that specifically addresses what you're seeking.

Yeah, absolutely. That's very helpful. Thank you. And then a follow-up, if I may, on L606, I'm curious what the physician feedback has been so far for a lower CMAX, broader AUC, longer half-life alternative to nebulized tibiaso. And then, sorry if I missed it, I'm wondering also when specifically you plan to initiate the phase three of the SPIRE trial.

Yeah, great. And we're fortunate to have Dr. Rajiv Sagar on the call today. So, Rajiv, if you wouldn't mind.

Yeah, thanks, Julian, for the question. So, first of all, I think, you know, the current open-label study is continues to gate very well. You know, we have long-term safety data that has accrued since the inception of this study. What clear feedback is, is twice-a-day dosing is an absolute game-changer for these patients when you inhale treprosanil. I think that's first and foremost. Second of all, I think that The use of the liposome itself has showcased what we believe highlights some impressive safety advantages. And finally, further reducing the C-max with the twice-a-day dosing profile and the sustained release formulation of the liposome itself. We have noted that systemic side effects continue also to be further abated, even beyond what we initially anticipated. So I think that combination of both of those are going to be highly encouraging for as we advance forward with the RESPIRE study. In regards to initiating the RESPIRE study, you know, we're working full steam ahead to prepare to initiate the study by year's end. You know, it's around March right now, and so we look forward to providing further information as we get closer to that timeframe near the end of the year.

Okay, great. Thank you again, and congrats on all the progress. Thank you, Julie.

Our next question comes from the line of Jason Gerberry with Bank of America. Your line is now open.

Hey, good morning. This is Bob and Patel on for Jason. Two questions from us. The first is, given the green space in PHILD, what's Liquidia's strategy to drive early market penetration for Eutropia? Can you share the market marketing differentiation that you plan to promote to doctors? And then, given the 505B2 pathway, should we expect any differences in the label for eTrepia versus Teveso DPI? I know in the past you've mentioned demonstrating titration to three times higher doses compared to Teveso DPI.

Yes, I'll answer the second question first, and then I'll ask Scott Muma, our chief commercial officer, if he would address the launch dynamics around pH, IOD, and differentiation that we intend to emphasize. So you're exactly correct, Bob, and I think the label, it is a 505B2, so we share obviously the indication claims and things like that, but where we differ from the label is in the dose titration table, because in our A study that we used to bridge, we showed that we could escalate the doses, as you mentioned, up to three times higher than the target therapeutic dose of the brand. So that will be in the label, and I think it's an important point of differentiation because it's print-enabled, using our proprietary print technology to drive the particles to the lower airway preferentially and avoid the toxicities associated with upper airway deposition. you know, it's going to help us drive a maximal outcome for these patients as they continue to progress, and therefore help us keep those patients on therapy longer is our presumption. So we think these are important and critical aspects. You know, if you look across any route of administration across the cycling, dose matters, and the ability to drive dose usually predicts the sort of therapeutic utility of that therapy, and that's why Tyveso has had limitations in dosing, and it's also why other drugs like Upravi and oral formulation, which is dose-limited and dose-sealing, also has limitations over the long and chronic treatment course of these patients. So lots of opportunity there from us from a labeling and dosing standpoint. So, Scott, if you could talk a little bit about kind of how we want to approach this market without giving away the shop too much.

Sure. Good morning. So it You know, if you think about it, we have the usual designation between centers and communities, and we're going – we are approaching and we will continue to approach both, especially after launch. And the situation in the centers is that the patients are really flowing in. There's an increasing number of patients flowing in as the awareness of PHILD increases, and we will obviously only add fuel to that fire. So we're in the centers, and we're going to make sure that we – ensure that Utrepia is the first choice of prostacyclin for those patients that have PHILD for a lot of the reasons that Roger just outlined and you've heard us talk about before. So, you know, we're going to harvest those patients immediately. The other thing we're doing is in the community, and there's many, many patients out there that doctors don't even know exist. They have ILD. The doctors aren't looking for PH. We need to help increase awareness. We need to help increase diagnosis. and then either get that doctor to prescribe or to refer that patient into one of the centers. And we've actually been doing that over the last six months or a year, going deeper and deeper into the community, educating on PHILD even before we get on the market because we know that that's only going to behoove us once we do get on the market because we do believe we have the superior product.

Great. Thank you so much.

Our next question comes from the line of Serge Bellinger with Needham. Your line is now open.

Hi, good morning. A couple commercially related questions, probably for Scott. Just can you remind us the breakdown between commercial and Medicare coverage for both PAH and PHILD? And then assuming we're going to get approval here in a couple months, can you talk about what you expect in terms of the ramp-up in coverage across both commercial and Medicare, I guess, over the next 12 months or so?

Yeah, great question. Great question, Serge. Scott?

Yeah, good morning, Serge. So, in terms of the breakdown, generally, think of it as Medicare is the biggest, so 50% Medicare, probably 30% to 40% commercial, and then the remaining is Medicare, other DOD, et cetera. In terms of the coverage over the first year, as you probably know, we're not commenting on our strategies. I will say that we have been working with the payers closely for quite some time, and we feel like we have great relationships with us. They have the desire to make sure that we maximize access, so we share that desire. And I think you'll see when we launch that we're very sound on that front.

Great. Thanks, Scott. Operator, next question.

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

Hi, good morning. Remind us what key readouts we should be focusing on for the assent study and what specifically we can expect in the data coming at ATS. I'm going to have a follow-up.

Yeah, thank you, Rajiv, if you wouldn't mind answering that one. Yeah, thanks, Ryan.

So, you know, as Roger talked about in the prepared remarks today, one of the highlights that we spoke about was the walk effect that was observed in the first 20 patients treated within eight weeks, which showed an observed mean improvement of around 26.4 meters. And I think that first and foremost highlights a few key processes first. that we can get patients to what we believe is even a new therapeutic goal that surpasses the traditional 9 to 12 breaths that have typically been sort of the ceiling effect of Tyveso since 2009. We've highlighted that we're able to achieve doses in the overwhelming majority of patients by week eight that's equivalent to greater than 50, greater or equal to 15 breaths by this time. So, So we can get patients up to what we believe is a very effective dose and even higher at an earlier time point and then allow further titration to further doses to stabilize the patient and plateau them out. So I think that's what's going to be quite encouraging. At ATS, we're going to showcase more regarding the data sets, regarding how we dose the patients. We're going to talk a little bit about our effects in terms of some quality of life questionnaires as well. And in the future, our plan is to, as we've talked about, the SENS study will be fully enrolled within the next few weeks here. So we're really excited about that. And then we do plan to prepare and submit for the final data set later in the year and present that at a major respiratory conference in the future.

Great. Thank you, Rajiv. That's helpful.

Thank you very much. And will you be doing additional hiring for the commercial field team after potential approval in both indications is confirmed? And what is the status of the current field team with regards to both hiring and training? Thank you.

Yeah, so Scott, if you wouldn't mind.

Yeah, sure. So the status, I'll start with that one. The status of the current sales team is that they're locked and loaded and ready. We've had the team on board for 14 months now, and these were experienced mostly pH, all rare disease folks to begin with. And so they've been out in the field strengthening relationships. They've been training, training, training. They are absolutely ready to go and very, very excited. On the future front, we actually just went through another exercise over the course of the last few months to take a look at our Salesforce sizing strategy. We think we're well-suited to launch, as I mentioned earlier. We will take another look at that once we get out and start to understand sort of the sensitivity factor for the number of reps, and then we'll look at potentially expanding, if that makes sense, in the future.

Another thing I would add, we are adequately sized for launch in May in both indications. And then as Scott said, obviously, as we continue to build and present the unique benefits of Eutropia, I think we want to articulate that even broader. So, you know, we could upsize our sales force probably in the 26 timeframe if necessary. Operator, is it another question?

Our next question comes from the line of Greg Harrison with Scotiabank. Your line is now open.

Hey, good morning, guys. Thanks for taking the questions. Wondering if you can comment on your updated cash runway following your recent financing and what assumptions go into that estimate that you could reach profitability with your current balance sheet. And then if you could also comment on which factors play into your decision on pricing, given your view of Utopia's profile relative to the competitor? Thanks.

Great. I think both of those questions would be well answered by Mike.

Yeah, Greg, great to talk to you again. As it relates to runway, as we said, you know, with this $100 million of additional financing from healthcare royalties, $25 million we received at closing, $50 million will be received upon the first commercial sale of Utrepia, and the final $25 million will be at mutual option once we reach a cumulative $100 million in net sales of Utrepia. I think it's pretty simple. We're very excited about the launch of Utrepia. you know, Roger said, and Scott has said, we are, you know, fully ready from a commercial readiness point of view, from a supply point of view, we feel very bullish about our ability to launch this successfully. And we feel that if, you know, assuming as, as Roger said, we file for full approval here coming up, we get full approval on or near the expiration of the exclusivity date. We feel confident if we, we hit our goals and hit our targets that we can be profitable on this current balance sheet. And, you know, we, And like I said, fully support the L6S6 program, all of our, you know, phase four studies we're doing on eutropia, while also the, you know, ongoing commercial readiness. So we're very excited. We're very confident. We're very happy to have a great partner, Lake Healthcare Royalties, you know, on this journey, and we look forward to launch. As to the second question, you know, we're not going to talk about pricing strategy. I think what I would say is, As we've demonstrated or continue to demonstrate and what we feel our early results on ASCENT only reinforce that, that we feel that we have a superior product profile. And based on that, we want to make sure that we are always balancing patient access while also fulfilling the value proposition that Utopia can bring. So we're not going to talk specifically about, you know, pricing strategies in totality, but I think we're very confident as we come to market that we will have the right strategy to make sure that patients have choice as we move forward.

Great. That's helpful. Thanks so much. Thanks for the question, Greg. Operator, next question.

Our next question comes from the line of Corey Juvenville with LifeSite Capital. Your line is now open.

Good morning, and thanks for taking our questions, and congrats on this new efficacy data with the science. Really exciting. You know, just kind of following the theme of more drug is better, I recognize that the sample size here is still relatively small, but have you had any type of dose response among patients? You know, are those who are reaching two to three-fold the Tyveso equivalent dose, achieving better outcomes than those in the study who might be, you know, kind of in the lower range equivalent to the existing standard of care. And this also might be a naive question, but is it possible to get these efficacy data on label, and how would you go about marketing this six-minute walk to prescribers post-approval?

Yeah, I'll take the second question, and then Rajiv can say what he can about the dose versus effect curve. So it's not, the data is, would not be available for indication claims per se because it's an open label safety study. I think it would be part of an annual update to the label once approved where we could update the clinical pharmacology section to describe the data both in terms of safety and potentially in terms of efficacy. But, again, that's an negotiation with the FDA about what we could then include in the clinical pharmacology section of the label. So I'll reserve sort of commitment on that, and we'll do the best we can to get it in there. But I think, you know, certainly we will abstract and then publish the data. And I think, as we're seeing, there's tremendous inbound interest based on what we're describing. And there's over 20 doctors doing this current assent study. Many of them are the seminal thought leaders, and I think the fact that they're doing the study and we're seeing the results that we're seeing is going to help highlight the data when they lead panel sessions, et cetera, because now they're seeing the true value of print as it relates to eutropia. And then I will just comment on those responses. before turning it over to Rajiv, it's very hard in a small sample of patients to show dose response, frankly. Plus, we're allowing patients to individually titrate to dose, so we're not sort of predefining what dose level patients should titrate to and then measuring that versus effect. So it's a continuum. So that's a difficult paradigm to show dose response in, but Rajiv, I don't know if you have additional comment there.

Yeah, Corey, I think a few things to highlight. First of all, the patients that we're rolling in assent are quite heterogeneous. We purposely designed the study to showcase safety and tolerability of eutropia. So we not only include patients with mild hemodynamic limitations with their pulmonary hypertension, but also patients with severe hemodynamic impairments with various levels of complexity with the interstitial lung disease and their respiratory physiological impairments. But I think despite that heterogeneity, I think what the data is showing, despite these small numbers, but even though the study continues to enroll and continue to highlight a few things, number one, clearly the low-resistance inhaler combined with our print formulation is absolutely allowing these patients to titrate eutropia to, again, to what I believe are actually new therapeutic levels. And that's very important because it's well known that prostanoid therapies are titratable. It is dose-related, so effects tend to improve as dose is escalated. The problem with current therapies on the market, including oral and systemic parenteral therapies, is that they're just significantly limited by terrible systemic side effects, especially in the oral therapies with the GI side effects and, of course, the parenterals with indwelling lines or subcutaneous complications. I think with the current inhaled therapies on the market today, we know that, at least in the INCREASE study, what was very interesting relative to the SENS study is, by eight weeks, only 50% of the patients in the tibetanibolide study for increase with PHLD were able to reach the 10 to 12 breath levels versus in the SENT study, despite the heterogeneity in the patient population, we're able to get patients, the overwhelming majority of patients, up to more than or equal to 15 breath equivalents. So, again, I think that what the SENT data is going to show at ATS and what KOLs are going to see is that eutropia is very well tolerated and titratable, and that they can customize to whatever needs their patients have with both PAH and PHLD in the very near future.

And, Corey, that's a great answer, Rajiv. Thank you. I think one way we're going to take on this dose issue directly, we're going to do a directed transition study from patients on Tyveso and Tyveso DPI that are underserved by those therapies, probably because of dose limitations. We'll transition them to eutrapia. improve their dose, and if we show, therefore, an improved outcome, we've shown not only is dose related to outcome, but we've also differentiated the products even in a different way and a helpful way for eutropia. So look for that study to start up in the near future as well. So operator, next question, please.

Thank you, and I'm currently showing no further questions at this time. I'd like to hand the call back over to Roger Jeffs for closing remarks.

Thank you very much. So we really appreciate everyone joining us today, and we look forward to speaking again soon. Bye-bye.

This concludes today's conference call. Thank you for your participation. You may now disconnect.