Good morning, and welcome everyone to the Liquidia Corporation Full-Year 2023 Financial Results and Corporate Update Conference Call. My name is Michelle, and I will be your conference operator today. [Operator instructions]. I would like to remind everyone that this conference call is being recorded. I would now like to hand the conference call over to Jason Adair, Chief Business Officer.

Thank you, Michelle. It's my pleasure to welcome everyone to Liquidia's full-year quarter 2023 financial results and corporate update call. Joining the call today are Chief Executive Officer, Dr. Roger Jeffs; Chief Operating Officer and CFO, Michael Kaseta; Chief Commercial Officer, Scott Moomaw, Chief Medical Officer, Dr. Rajeev Saggar, and General Counsel, Rusty Schundler. 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’ll open-up the call up for your questions. Roger?

Thank you, Jason. Good morning, everyone, and thank you for joining us today. While today's call is intended to review the company's accomplishments in the last year, we know that physicians, patients, and our investors are solely focused on one thing, the potential FDA approval in the coming weeks of YUTREPIA, our novel dry-powder formulation of Treprostinil. I'll ask Rusty to address the legal and regulatory path to approval in more detail shortly, but to put it simply, with the recent decisions by the federal circuit affirming the invalidity of the sole patent that is blocking our approval, the FDA should be able to grant approval for YUTREPIA after March 31, when regulatory exclusivity to treat PH-ILD with Tyvaso expires. A precise final approval date is hard to forecast, but we view the remaining steps as largely procedural. Final FDA approval has always been the goal, and we have never been closer or better prepared than today. Our commercial teams are in place and ready for launch. Our expanded field force has been raising the profile of Liquidia in their territories over the last three months. Our manufacturing team is preparing inventory in anticipation of a potential launch in both PAH and PH-ILD. Our R&D team continues to build clinical knowledge by studying YUTREPIA and PH-ILD patients in the open-label ASCENT trial, and our finance team has positioned the company with the resources and discipline required to execute a successful launch. We entered 2024 at a full sprint due to the resolve and execution of our team in 2023. Last year, everything grew in the right direction. Our confidence grew with legal wins. Our balance sheet grew with key transactions by marquee investors and insiders, and our pipeline grew with the in-license of L606, the most clinically advanced next-generation sustained-release…

Thank you, Roger. I'd like to group the recent litigation and regulatory activity into two buckets. First, those items on the critical path to YUTREPIA’s approval. Second, the recent attempts by United Therapeutics to assert new legal theories to block approval of YUTREPIA. All told, we see the increased and frantic litigation activity by United Therapeutics as perhaps a sign that even they believe that the legal impediments to final approval of YUTREPIA are nearing an end. In the first bucket I mentioned, as we have publicly stated previously, there are only two items on the critical path for YUTREPIA to be launched. First, Judge Andrews must lift the injunction he ordered in August 2022 based on his finding of infringement of the 793 patent, a patent that has subsequently been invalidated, a finding that was affirmed again yesterday when the Federal Circuit denied United Therapeutics’ request for a rehearing. And second, the regulatory exclusivity granted to Tyvaso for the PH-ILD indication must expire, which will occur on March 31. Once both of these have occurred, the FDA will have the ability to issue final approval for YUTREPIA for both the PAH and PH-ILD indications. We'll not speculate on the specific date when Judge Andrews will render his decision, but the matter has been fully briefed and could be decided at any time. In the second bucket, over the last several weeks, United Therapeutics has sought to add new impediments to FDA approval in our launch of YUTREPIA by seeking preliminary injunctions in multiple proceedings. However, as we have stated previously, in order to obtain any preliminary injunctions, the burden will be on United Therapeutics to convince the judge that among other things, they're likely to succeed on the merits in those actions. We believe that this burden will be…

Thank you, Rusty, and good morning, everyone. The company has never been in a stronger financial position than it is now, heading towards its first major product launch. The financial discipline we've shown to date has not only allowed us to fully engage in defending against the litigation campaign that has been directed against us, but has demonstrated to the savvy investors that we can meet and exceed expectations as we look to build value in the company without overspending or incurring significant dilutions. Turning to our full 2023 financial results, which can be found in the press release, you'll see that revenue was $17.5 million for the year in 2023, compared with $15.9 million in 2022, tied to our promotion agreement with Sandoz to commercialize Treprostinil injections. The increase of $1.6 million was primarily due to favorable growth to net chargeback rebate and managed care adjustments, offset by the impact of lower sales quantities as compared to the prior year. Cost of revenue was roughly the same for 2023 and 2022 at $2.9 million. Cost of revenue relates to the promotion agreement. Research and development expenses were $43.2 million in 2023, compared with $19.4 million in 2022. The increase of $23.8 million or 122% was primarily due to the $10 million upfront license fee payment to Pharmosa for the exclusive license to develop and commercialize L606 for North America, plus an additional $2.6 million in support of that program. Expenses related to our YUTREPIA program increased to $13 million from $6.7 million the year prior, primarily due to increased manufacturing of pre-launched commercial supply, and the startup of our ASCENT study. Personnel and consulting expenses, including stock-based compensation expense, also increased $5.1 million, primarily due to increased headcount to support the potential commercialization of YUTREPIA. General and administrative expenses were $44.7 million in 2023, compared with $32.4 million in 2022. The increase of $12.3 million or 38% was primarily due to a $9.8 million increase in personnel and consulting expenses, including stock-based compensation, partially driven by the expansion of our field force, and also a $1.4 million increase in commercial expenses in preparation for the potential commercialization of YUTREPIA. In summary, we incurred a net loss in 2023 of $78.5 million as compared to a net loss of $41 million in 2022. We ended the year with $83.7 million cash on hand then quickly added another $100 million in the first week of January, with a private placement of equity to a single investor, and a third advance from HealthCare Royalty under our agreement from January 2023. In summary, we are well positioned to achieve our corporate objectives in 2024. I would now like to turn the call back over to Roger.

Thank you, Mike. 2024 is shaping up to be the transformational gear at Liquidia. We are poised in the starting blocks, and as you and Rusty have both described, have fought earnestly to get where we are today. We look forward to proving ourselves in the market, but more importantly, easing the burden of patients suffering from these debilitating diseases. With that, I would now like to open our call up for questions. Operator, first question please.

Thank you. [Operator Instructions] And our first question is going to come from the line of Greg Harrison with Bank of America. Your line is open. Please go ahead.

Good morning. This is Mary Kate on for Greg. Thanks for taking our question. So, as you guys prepare for your commercial transition, do you see any differences in launch strategy for PAH compared to PH-ILD? Maybe why or why not, and do you anticipate equal interest in both indications? Thanks.

Good morning, Mary Kate. Thanks for the question. So, we're fortunate to have Scott Moomaw, our Chief Commercial officer on the phone. Scott, maybe if you would like to opine on that.

Yes. So, there's a little bit of difference in strategy. So, in PAH, as you know, there are many medications already available. So, it's going to be really demonstrating why our prostacyclin is the best alternative relative to the other prostacyclins for a lot of reasons that we can obviously get into, and we believe that we will be very successful in that space, getting earlier use of our inhaled prostacyclin YUTREPIA, because it is so convenient and because of the titration of dose. And so, in that space, we'll be targeting the big centers, the physicians that use prostacyclins already. We'll get some new physicians probably, but we'll focus on the targets that do use prostacyclins. In PH-ILD, as you know, this is a relatively untapped market. And so, the strategy there is going to be much more about educating on the prevalence of PH-ILD and then getting physicians to look for it and then getting them to treat it. And we'll be out in the community with community pulmonologists, help educating them that this condition exists and that it's deadly. And then we'll be educating them on YUTREPIA. And if they would be willing to use YUTREPIA, that's great. We will aid them in doing that. If they won't, then of course we would like them to refer that patient to a PAH center of excellence where it would be treated. So, I think that's a brief summary of how we'll approach the two markets.

Great. Thank you.

And maybe I'll just add a few comments. So, I think in the - it's kind of what I said in my opening statement, Mary Kate, that in PAH, we would like to be the first-in-choice prostacyclin. And the reason I think we can do that is because really with YUTREPIA and its ability to titrate to doses that are on order of threefold more than what was originally possible with Tyvaso, we've changed therapeutic index of that molecule, and that's all enabled by our PRINT technology. Why that's important is now we can deliver the drug for PAH patients to the site of action through the lung and avoid the significant off-target effects, which are really hampering for the oral therapies in particular. So, if you look at the op Uptravi, it starts at a 200 microgram dose and it's titratable up to a ceiling of 1,600 micrograms, but it has a ceiling. And that maintenance dose is determined by tolerability. It's indicated to delay disease progression and decrease the risk of hospitalization, but it's improvement on six-minute walk distance is modest, only 12 meters, and I believe that was not significant. The consequence of that therapy is 42% of those patients have diarrhea, 33% have nausea, and 18% have vomiting. So, significant off-target effects. And Orenitram is a very similar story. It's a used TID. It's titrated to effect. It's indicated to delay disease progression and improved six-minute walk distance. But in the largest study of that therapy in 690 patients, 69% of those patients had diarrhea, 40% had nausea, and 36% had vomiting, which clearly limits dosing. So, and in fact, UTHR has said lately that because it's so difficult to titrate, they're actually promoting titration via the parental route and then transition to oral. So, you can see this is burdensome and onerous. What YUTREPIA will then do is negate completely these off target effects to the GI tract and allow dose titration. So, again, we're going to look at the oral prostacyclin market as a significant market where we can gain share, and we'll do that sort of tactically after we position ourselves as the best-in-class inhaled prostacyclin, as Scott mentioned, for both PAH and PH-ILD. Operator, next question, please.

One moment for our next question. And our next question is going to come from the line of Julian Harrison with BTIG. Your line is open. Please go ahead.

Hi, good morning. Thank you for taking my question. Just to be clear, based on some of your prepared remarks, if you are forced to seek approval in PH-ILD via supplement instead of the current arrangement, your view is that filing a supplement with 327 patent now in the Orange Book should not trigger an automatic stay. Am I understanding that correctly?

Yes. thanks for the question, Julian. Good morning. I’ll ask Rusty to answer that, please.

Yes. So, let me clarify maybe a couple of things. So, first, I think our view is that the amendment being rejected and us having to file this by supplement is sort of the worst-case scenario. We think what the FDA did was absolutely right accepting our amendment. So, we don't think this will even come into play. But if we were required to come into a supplement, then that's exactly right. So, if you look at the statute and again, it's 21 USC 355 C3C, what it - it specifically defines those patents that can give rise to a 30-month stay. And critically, it says only those patents, and I'll quote it, before the date on which the application and then (excluding an amendment or supplement to the application was submitted). So, again, supplements are treated the exact same way as amendments for purposes of determining which patents can give rise to a 30-month stay. And so, even if we were required to file a supplement, the result would be no new 30-month stay.

Very helpful. Thank you.

Thank you, Jolene. Operator, next question please.

One moment. And our next question is going to come from the line of Serge Belanger with Needham. Your line is open. Please go ahead.

Hi, good morning. Thanks for taking my questions. I guess the first one, and apologies if I missed this in the prepared remarks, but has there been any additional interactions with the agency post the late January PDUFA date for the PH-ILD approval? Have they asked for additional info or given you any additional in information regarding their internal process for that potential approval? And then secondly, I guess for Rusty, maybe just talk about the Supreme Court decision to deny the Liquidia petition late last month, and just what it means to the overall legal proceedings. Thanks.

Thanks, Serge. Good morning. I'll break this into two parts. So, Rajeev oversees our regulatory group, so he can answer the first question regarding interactions with FDA. And then Rusty, if you'll answer the Supreme Court question. Rajeev?

Yes, thanks, Roger. Hi, Serge. Good morning. So, in regards to our - we continue to believe very strongly, as Rusty already alluded to, that the amendment we filed to add on the indication for PH-ILD, remains appropriate. And in line with our discussions with the FDA, as you know, the only stopping gap was really the clinical exclusivity, which if we had received previous approval, that would lead to a tentative approval. Now, we anticipate that date is shortly arriving on May 31 when the clinical exclusivity ends. And therefore, the entire package right now will lead to now a full approval for both indications for PAH and PH-ILD, and we remain confident in that matter. I'll turn it over to Rusty to answer your second question.

Serge, thanks for the question. So, the Supreme Court case really has no bearing. So, as a reminder, that case was our attempt to overturn the original Hatch-Waxman decision on the 793 patent and raise some arguments that we think were overlooked by the lower courts that there shouldn't have been a finding of infringement at all. Supreme Court didn't take up that appeal, but again, it all relates to the 793 patent, which separately has been invalidated at this point, and now affirms really twice by the federal circuit. So, with that decision from the federal circuit, the decision of the Supreme Court really doesn't bear on sort of how this is going to play out at all.

Okay, thank you.

Yes, maybe, Serge, I'll just add a little bit to Rajeev. So, I think, the one communications we've had, obviously we missed the January 24 PDUFA action date. And the reason for that has been communicated is that the FDA is awaiting for the injunction to be removed. So, as Rusty said, there's two things that need to happen principally for us to get full approval, which is the injunction removed and then the potentially the clinical exclusivity to expire at the end of March. So, as Rajeev said, we're looking now. The amendment was filed and asked for full approval even when we filed it in July for both PAH and PH-ILD. So, it's our expectation now that we'll skip the tentative approval phase and probably just go to a full approval after the March 31 clinical exclusivity expiration. Operator, next question, please.

One moment. And our next question is going to come from the line of Matt Kaplan with Ladenburg Thalmann. Your line is open. Please go ahead. Mr. Kaplan, your phone could be on mute.

Oh, thank you. Yep. Good morning. And Roger, just to follow up on that question in terms of - and thank you, Rusty, for the detailed play by play in terms of the moving parts here in the litigation. But in terms of the critical path and Judge Andrews lifting of the injunction, can you give us some more detail in terms of the moving parts there and how that portion of it will work? Obviously, the regulatory exclusivity expiration is just a date on the calendar, so that's easy.

Yes, and Rusty can fill in here, but again, we feel Judge Andrews actually has all he needs now, Matt, to remove the injunction. The December 20th affirmation by the federal circuit Court of Appeals should have given him the power to remove it. And I think he was just waiting to see the rehearing request denied, and then the mandate to issue, which will happen next week. I think next Tuesday is when it should issue. So, at that point, he'll be fully empowered to do what he needs to do. Whether or not - I think, Rusty, you can comment on how you see the pending PI, how that interplay may impact this.

Yes. So, Roger, on the existing injunction, what you laid out is exactly correct. Again, it's been fully briefed in front of Judge Andrews so that he has what he needs. We also yesterday supplemented what we had submitted to him to provide him the denial to rehear and request that was issued by the federal circuit. So, again, he has all the information in front of him. And obviously, they have the new case, the 327 patent case where they've also requested a preliminary junction. Those cases really don't relate to one another. They’re both in front of the same judge and obviously the patent system similar, but procedurally, the injunction that currently exists isn't tied to their request for a new injunction on the new patent. So, I don't think there's any interdependency there between the two actions,

And Matt, that's why we're not giving a specific day when we think the approval action will happen, but again, other than it'll be after March 31 when the exclusivity expires.

Right. Okay, great. And then shift gears a little bit in terms of L606. Can you give us some more details in terms of the regulatory pathway there? You described only needing one additional study for approval in both PAH and PH-ILD. Can you give us a sense in the terms of the timeline of that as well?

Yes, I'd love to. So, Rajeev's also overseeing that effort. So, Rajeev, if you wouldn't mind answering the question.

Yes, thank you, Matt, and good morning to you as well. So, as you alluded to, L606 is our liposomal formulation of sustained-release for Treprostinil that's going to be delivered twice a day with really a smart portable nebulizer. So, we're really excited about this program as it's sort of the leading effort to a Phase III program. The program is designed using a similar strategy like we have with YUTREPIA. So, this is a 505(b)(2) pathway with the label drug being Tyvaso. In our Type C discussions with FDA that had occurred back in December of 2023, once again, we had confirmation that a single placebo efficacy study with L606, would lead to approval for both indications for group one PIH, as well as group three PH-ILD. In that regard, as you stated, we specifically have chosen the indication for PH-ILD to take into a global large Phase III study. That study is gated to initiate sometime in the - near Q4 of 2024.

Great. Thank you, Rajeev. And I think the one thing to add to that is, I guess the other part of your question is, how long will that take? I think because there's such a scarcity of treatments for PH-ILD, the clinical trial, particularly when we do it in European centers for instance, has potential to enroll quite rapidly. I think the normal time course for the sample size we're contemplating would be two years or so, but I think maybe we can shorten that a bit. Then there's time to get through the six-month endpoint and then time to collect the data, submit, and review. So, I think just in broad brush strokes, we're looking at, from first patient in to an FDA decision, is probably in the three and a half to four-year arrangement, Matt. Next question, please, operator.

And one moment for our next question. Our next question is going to come from the line of Kambiz Yazdi with Jefferies. Your line is open. Please go ahead.

Morning team. With the filed motion for preliminary injunction with regards to 327, what would be the timelines associated with that? And then can you remind us the FDA's perspective of NDA reamendment versus NDA for indication expansion with regards to tentatively approved drugs? Do we have any precedence there? Thank you.

Yes, I think both of those questions are in Rusty's court. So, if you wouldn't mind, Rusty.

Sure. So, on the first question for the 327 patent, so there is a briefing schedule on that. United Therapeutics has filed their brief requesting - in support of their request for a preliminary injunction. Our response is currently due on April 5th. Their reply then would be due April 19. The thing I'd - and then from there, the court either schedules a hearing or not and makes a decision. The thing I'd remind you though is that, again, the default is that if there's no preliminary injunction in place, there's nothing that blocks us from moving forward, getting approval and launching. So, the burden is on United Therapeutics to get a preliminary injunction before that happens. So, we'll see if they try to accelerate the proceedings or what they try to do on that front, but that's the timeline that's currently in place on that. As far as the FDA position on amendments versus supplements, again, I think if you look at the FDA existing guidances, so there's a 2004 non-binding guidance, that is what United Therapeutics was pointing to, which they claim stands for the proposition that you can never add an amendment to a pending NDA - or I'm sorry, never add an indication to a pending NDA. However, the 2016 regulations and the cited 21 CFR 314.60 subsection F, expressly contemplates situations where new indications could be added to a pending NDA, including a 505(b)(2) NDA like ours. So, again, I think clearly the FDA is contemplating that there are at least circumstances where indications can be added to NDAs as evidenced by the regulations.

And if I may add, Kambiz, I think the guidance that United Therapeutics is pointing towards is the bundling guidance, but that's really more specific. If you're changing route dosage form or formulation and providing new data, that should be submitted separately, and it's a way to make sure that the agency gets their review fees. We've done none of that. So, it's the same route, same dosage form, same formulation and no new data. So, we think we're well within the statute that Rusty just described. Great. Operator?

And then I guess one other follow-up is on the ASCENT trial. What kind of patient populations are being studied and how is enrollment proceeding there?

Yes, great question. Appreciate that. So, Rajeev, if you wouldn't mind.

Yes, thanks, Kambiz. So, once again, the ASCENT study is something that we are extremely excited about. More importantly, this is a study that's absolutely needed in the literature and has actually been desired by the KOLs across the entire region of the United States, requesting that patients that have been recently diagnosed with PH-ILD that are naïve to any therapy, are then placed onto YUTREPIA, which really will highlight three pillars that we have continued to suggest that are very important to this patient profile. The first thing is tolerability and titratability. These things are going to be led by our PRINT technology and therefore our formulation. The combination of those two allows us to use a very low resistance off-the-shelf inhaler which has the simplicity that is needed for patients that have impairments in lung function, but can deliver the dose profiles that we believe are going to be required to not only achieve the minimum therapeutic goals of equivalency of 10 to 12 breaths four times a day of inhaled Treprostinil, but more importantly, lead to actually more improvements in clinical outcomes and efficacy standards that are used, such as walk distance and actual overall clinical outcomes. We're very encouraged right now by the current enrollment rates that we're seeing. As you know, we enrolled our first patient to the mid to end of December of 2023, and we anticipate that we'll complete enrollment up to 60 patients by the end of this year. So, we look forward to sharing some snapshots of that data in future meetings that are coming up shortly. Roger?

Thank you, Rajeev. And I think, Kambiz, it's a great question. I think the other thing again, why this data is important, and I think when you look at the data that's - some of the data that's been published on United Therapeutics is Tyvaso DPI, particularly the data out of the National Jewish Center, Colorado, you can see that there's been some difficulty with the DPI in at least their single center patient population, where there was about 60% of their patients dropped off between three and six months, whether or not they were naïve to prostacyclins or transitioned previously from nebulized. And I think the other thing that's interesting to us is that there's still a retained 40% population of nebulized patients. I think when UTHR launched a few years ago, the assumption was that they would convert that entire market quite quickly to Tyvaso DPI. So, that's not happened. So, the question is why, and we think it may be for the inability to dose those patients to good clinical effect, which we're trying to solve for with YUTREPIA. So, if this data bears out the way we think it will, then that will certainly auger that this is the best-in-class therapy and first-in-choice therapy. Next question, please.

I'm showing no further questions, and I'd like to hand the conference back over to Roger Jeffs for further remarks.

Great. Thanks, operator. So, with no further questions, again, we thank you for joining us today. My sincere hope is that the next time we address you on the earnings call, Liquidia will be providing to patients what we feel is the preferred product for inhaled Treprostinil, and it will come at a critical time as the market for inhaled Treprostinil rapidly expands. Thank you and have a good day.

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