This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.
spk07: 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 currently all participants are in a listen-only mode following the presentation we will conduct a question and answer session the instructions will be provided at the time for you to queue up for a question i would like to remind everyone that this conference is being recorded i would now like to hand the conference over to Jason Adair, Chief Business Officer.
spk25: Thank you, Michelle. It's my pleasure to welcome everyone to Liquidia's full year 2023 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 Commercial Officer Scott Mumaw, Chief Medical Officer Dr. Rajiv Sagar, 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 for your questions.
spk22: Roger?
spk18: Thank you, Jason. Good morning, everyone, and thank you for joining us today. Thanks. 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 Eutrepia, our novel dry powder formulation of Trepocino. I will 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 Eutrepia after March 31st, when regulatory exclusivity to treat PHLD with type A so 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 best – of in their territories over the last three months. Our manufacturing team is preparing inventory in anticipation of a potential launch in both PAH and PHILD. Our R&D team continues to build clinical knowledge by studying eutrophia in PHILD patients in the open-label assent 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 inhaled-through-prosinol program. Given the proximity of a potential launch, I'd like to spend a little time framing the potential market opportunity for eutrophia as we see it, both in PHILD and PAH. With regard to PHILD, patients. However, these estimates likely undervalue the total addressable population, since those calculations rely on historical publications before the field had effective tools to treat the disease and therefore reasons to diagnose the disease. With inhaled tryprosinol as the only approved mechanism to treat PHLD, the market penetration is still in its infancy. We believe there is significant growth in this market. Total inhaled tryprosinol revenues currently sit at about a 1.3 billion annual run rate in the U.S. alone. With regard to PAH, we also believe that eutrophia has potential for significant uptake. We view eutrophia as having the potential to not only be the best-in-class inhaled troposinol, given its dosing flexibility and ease of use, but also the first-in-choice prostacycline. Specifically, patients who previously considered the oral prostacyclines as their starting choice can now avoid the significant and limiting off-target GI toxicities associated with these drugs while still achieving therapeutic doses. Thus, combining current sales of oral prostacyclines of approximately 1.6 billion in pH and With the recently reported sales from inhaled riposte at all of $1.3 billion, the market opportunity for eutrapia could be approaching $3 billion and growing incrementally as the PHLD patients still remain largely untreated. With its unique and differentiated approach, it's exciting to think about the potential for eutrapia and the rapture value in both of these markets. At this time, I would like to ask Rusty to summarize the next steps towards final. Thank you.
spk05: 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 Utrepia's approval. Second, the recent attempts by United Therapeutics to assert new legal theories to block approval of Utrepia. 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 Utrepia 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 Utrepia 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 Tyveso for the PHILD indication must expire, which will occur on March 31st. Once both of these have occurred, the FDA will have the ability to issue final approval for Eutrepia for both the PAH and PHILD indications. We will 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 eTrepia 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 are likely to succeed on the merits in those actions. We believe that this burden will be a challenge for them to meet based on the laws and the facts that issued. The first request for preliminary injunction is directed to the second Hatch-Waxman lawsuit alleging infringement of the 327 patent in the treatment of PHLD. Issued after the Eutrapia NDA was submitted and after Liquida amended its NDA to add PHLD to the label for Eutrapia, the 327 patent covers the treatment of PHLD patients with Tybaso in accordance with the dosing regimen in the Tybaso label. As we have noted previously, There is considerable prior art that teaches the treatment of PHILD patients with Tyveso, including the 793 patent and multiple peer-reviewed publications in the decade prior to the priority date for the 327 patent that described positive results from treating PHILD patients with Tyveso. Courts have generally refrained from issuing preliminary injunctions in situations where there are substantial questions as to the validity of a patent. And in light of all the prior art here, we believe substantial questions of validity of the 327 patent exist. The second request for preliminary injunction is directed at United Therapeutics' recently filed suit against the FDA under the Administrative Procedures Act. Filed in the United States District Court for the District of Columbia, this suit alleges that the FDA mistakenly permitted Liquidea to amend the NDA for Eutrepia to add the PHLB indication. We have intervened in the case and are now a party to the case alongside the FDA. First and foremost, the FDA did in fact accept our amendment to the NDA for review, and we believe that the FDA's acceptance of our amendment for review was proper and in full accordance with current FDA regulations. United Therapeutics' argument that an amendment to add a new indication is improper is based primarily on a non-binding 2004 FDA guidance document, ignoring subsequent FDA regulations adopted in 2016 that expressly contemplate the possibility of adding new indications through an amendment. Secondly, even if United Therapeutics was correct that the amendment was improper, that would not mean that United Therapeutics would receive a new 30-month stay, as they have argued. For instance, even if the amendment was now rejected by the FDA, Liquidia could simply supplement its NDA after approval to add the PHILD indication. The exact same process used by United Therapeutics to add PHILD to the label for Tyveso. Critically, the statute expressly states that amendments and supplements are treated the exact same way in determining whether a patent can give rise to a 30-month stay, meaning that only those patents submitted to the Orange Book prior to the filing date of the original NDA, not the filing date of the amendment or supplement, can give rise to a 30-month stay. Briefing on the motion for preliminary injunction will be completed on March 18th, and a hearing will be held on March 29th. We look forward to this matter being addressed in short order. In summary, Liquidia sees the path to launching the atrophy in two straightforward actions, removal of the injunction and approval of the products. The rest is a last-ditch attempt by a competitor to make any and every argument they can to maintain their monopoly and deny patients access to a meaningful treatment option. We have long anticipated the possibility that United Therapeutics could engage in such a flurry of activity as we meet near clearance of the original Hatch Waxman litigation, and we are prepared to meet them head-on. With that, I will pass to Mike for a review of last year's financials.
spk27: 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 dilution. Turning to our full 2023 financial results, which can be found in the press release, you will 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 promotional injections. The increase of $1.6 million was primarily due to favorable growth in that 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 PhRMOSA 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 Utrepia 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 Utrepia. General and administrative expenses were $44.7 million in 2023, compared with $32.4 million in 2022. The increase $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 Utrepia. 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 and 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.
spk18: Thank you, Mike. 2024 is shaping up to be the transformational year at Equidia. 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.
spk07: Thank you. To ask a question at this time, please press star 1-1 on your telephone and you will hear an automated message advising you your hand is raised. To withdraw your question, please press star 1-1 again. One moment while we compile our Q&A roster. 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.
spk01: Good morning. This is Mary Kate on for Greg. Thanks for taking our questions. So as you guys prepare for your commercial transition, do you see any differences in launch strategy for PAH compared to PHILD? Maybe why or why not? And do you anticipate equal interest in both indications? Thanks.
spk18: Good morning, Mary Kate. Thanks for the question. So we're fortunate to have Scott Lumar, our chief commercial officer on the phone. Scott, maybe if you would like to opine on that.
spk15: Yeah, 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 eutrapia because it is so convenient and because of the titration 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 PHILD, 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 PHILD and then getting physicians to look for it and then getting them to treat it. And, you know, 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 eutropia. And if they would be willing to use eutropia, 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 pH center of excellence where it would be treated. So I think that's a brief summary of how we'll approach the two markets.
spk12: Great. Thank you.
spk18: And maybe I'll just add a few comments. So I think 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 for osteocycline. And the reason I think we can do that is because really with eutropia and its ability to titrate to doses that are, you know, on order of threefold more than what was originally possible with Tyveso, We've changed the 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 Optravi, 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 its 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 arenatram is a very similar story. It's a used TID. It's titrated to effect. It's indicated to delay disease progression and improve 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. And in fact, Uther has said lately that because it's so difficult to titrate, they are actually promoting titration therapy via the parenteral route and then transition to oral. So you can see this is burdensome and onerous. What eutropia will then do is negate completely these off-target effects to the GI tract and allow those titrations. 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 tactfully after we position ourselves as the best-in-class inhaled prostacyclin, as Scott mentioned, for both PAH and PHILD. Operator, next question, please.
spk06: One moment for our next question. And our next question is going to come from the line of Julian Harrison with BTIG.
spk07: Your line is open. Please go ahead.
spk14: 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 PHILD via supplement instead of the current arrangement, your view is that filing a supplement with the 327 patent now in the Orange Book should not trigger an automatic stay. Am I understanding that correctly?
spk18: Yes. Thanks for the question, Julian. Good morning. I'll ask for us to answer that, please.
spk05: Yes, so let me clarify maybe a couple things. So first, I think our view is that, you know, the amendment being rejected and us having to file this by supplement is sort of the worst-case scenario. You know, 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 and do a supplement, then that's exactly right. So if you look at the statute, again, it's 21 U.S.C. 355 C.3.C., you know, what it specifically defines, those patents that can give rise to a 30-month stay. And critically, it says, you know, only those patents, and I'll quote it, before the date on which the application, and then in parentheses, 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.
spk11: Very helpful. Thank you.
spk18: Thank you, Jillian. Operator, next question, please.
spk07: 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.
spk29: Hi, good morning. Thanks for taking my question. 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 PHILD approval. Have they asked for additional info or given you any additional 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 liquidity petition late last month. and just what it means to the overall legal proceedings. Thanks.
spk18: Thanks, Serge. Good morning. I'll break this into two parts. So Rajiv 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. Rajiv?
spk20: Yeah, thanks, Roger. Hi, Serge. Good morning. So in regards to our, you know, we continue to... believe very strongly, as Rusty already alluded to, that the amendment we filed to add on the indication for PHLD remains appropriate and in line with our discussions with the FDA. As you know, the only stopping gap was really the clinical exclusivity, which, you know, if we had received previous approval, that would lead to a tentative approval. Now we anticipate that date is shortly arriving on May 31st 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 PHLD, and we remain confident in that matter. I'll turn it over to Rusty to answer your second question.
spk05: Sir, thanks for the question. So, the Supreme Court case really has no bearing. 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, overlooked by the lower courts, that there shouldn't have been a finding of infringement at all. you know, 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, you know, 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.
spk18: Thank you. Yeah, maybe, Serge, I'll just add a little bit to Rajiv. So I think, um, You know, the one communications we've had, obviously we missed the January 24th 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 and then potentially the clinical exclusivity to expire at the end of March. So as Rajiv said, we're looking now. The amendment was filed and asked for full approval even when we filed it in July for both PH and PHLD, 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 31st clinical exclusivity expiration. Operator, next question, please.
spk07: One moment. And our next question is going to come from the line of Matt Kaplan with Lindenburg-Tholomon. Your line is open. Please go ahead.
spk22: Mr. Kaplan, your phone could be on mute.
spk19: 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.
spk18: Yeah, Rusty can fill in here, but, you know, again, we feel Judge Andrews actually has all he needs now, Matt, to remove the injunction. You know, the December 20th affirmation by the Federal Circuit Court of Appeals should have given them the power to remove it, and I think he was just waiting to see the hearing 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 this pending PI, how that interplay may impact this.
spk05: 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 of the rehearing request that was issued by the Federal Circuit. So again, he has all the information in front of him. Obviously, they have the new case, the 327 patent case, where they've also requested a preliminary injunction. Those cases really don't relate to one another. They're both in front of the same judge, and obviously the patent system is similar, but procedurally the injunction that currently exists isn't tied to their request for a new injunction on the new patent. I don't think there's any interdependency there between the two actions.
spk18: That's why we're not given a specific day when we think the approval action will happen, but Again, other than it'll be after March 31st when the exclusivity expires. Right, right. Okay, great.
spk19: 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 needing one additional study for approval in both PH and PHILT. Can you give us a sense in terms of the timeline of that as well?
spk18: Yeah, I'd love to. So Rajiv is also overseeing that effort. So Rajiv, if you wouldn't mind answering the question.
spk20: Yeah, thank you, Matt, and good morning to you as well. So as you alluded to, L606 is our liposomal formulation of sustained release for truprosanil 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 leading the effort to a Phase III program. The program is designed using a similar strategy like we have with Utrepia. So this is a 505B2 pathway with the label drug being Tyveso. 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 1 PAH as well as Group 3 PHLD. In that regard, as you stated, we specifically have chosen the indication for PHLD to take into a global large Phase III study. That study is gated to initiate sometime near Q4 of 2024.
spk18: Great. Thank you, Rajiv. And I think the one thing to add to that is, you know, I guess the other part of your question is how long will that take? You know, I think because there's such a scarcity of treatments for PHLD, 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 brushstrokes, we're looking at a From first patient in to an FDA decision is probably in the three and a half to four year range, Matt.
spk17: Next question, please, operator.
spk07: And one moment for our next question. Our next question is going to come from the line of Kamboos Yazdi with Jefferies. Your line is open. Please go ahead.
spk10: Morning, Payne. 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 re-amendment versus NDA for indication expansion with regards to tentatively approved drugs? Do we have any precedents there? Thank you.
spk18: Yes, I think both of those questions are in Rusty's court, so if you wouldn't mind, Rusty.
spk05: Sure. So on the first question for the 327 patent, so there is a briefing schedule on that. You know, United Therapeutics has filed their brief requesting in support of their request for a preliminary injunction. Our response is due, currently due on April 5th. Their reply then would be due April 19th. 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 and getting approval and launching. So the burden's 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 is pointing to, which they claim stands for the proposition that you can never add an amendment to a pending NDA. I'm sorry, never add an indication to a pending NDA. However, you know, the 2016 regulations and the site is 21 CFR 314.60 subsection F expressly contemplates situations where new indications could be added to a pending NDA, including a 505 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. you know, as evidenced by the regulations.
spk18: And if I may add, you know, Comby, 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 statutes that Rusty just described.
spk10: Great. Operator? I guess one other follow-up is on the assent trial. What kind of patient populations are being studied, and how is enrollment proceeding there?
spk18: Yeah, great question. We appreciate that.
spk20: So, Rajiv, if you wouldn't mind. Thanks, Kambiz. So once again, the SENS 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 PHLD that are naive to any therapy are then placed onto eutropia, which is 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 PRINC 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 treprosanil, 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 rate 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?
spk09: Thank you, Rajiv.
spk18: And I think it's a great question. I think the other thing, you know, again, why this data is important, I think when you look at the data that's, some of the data that's been published on United Therapeutics' TABEZA DPI, particularly the data out of the National Jewish Center in Colorado, you can see that there's been some difficulty with the TPI 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 naive 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 Uther launched a few years ago, the assumption was that they would convert that entire market quite quickly 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 eutrophia. So if this data bears out the way we think it will, then that will certainly augur that this is the best-in-class therapy and first-in-choice therapy. Next question, please.
spk07: I'm showing no further questions, and I'd like to hand the conference back over to Roger Jeffs for further remarks.
spk18: 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, Liquida will be providing to patients what we feel is the preferred product for inhaled tripostinol, and it will come at a critical time as the market for inhaled tripostinol rapidly expands. Thank you, and have a good day.
spk07: This concludes today's conference call. Thank you for participating. You may now disconnect. Thank you. Thank you. Thank you. Bye. you 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. Currently, all participants are in a listen-only mode. Following the presentation, we will conduct a question and answer session. The instructions will be provided at the time for you to queue up for a question. I would like to remind everyone that this conference is being recorded. I would now like to hand the conference over to Jason Adair, Chief Business Officer.
spk25: Thank you, Michelle. It's my pleasure to welcome everyone to Liquidia's full year 2023 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 Commercial Officer, Scott Mumaw, Chief Medical Officer, Dr. Rajiv Sagar, 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 for your questions.
spk18: 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 Eutrepia, our novel dry powder formulation of Trepocino. I will 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 Eutrepia after March 31st, when regulatory exclusivity to treat PHLD with Typeso 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 PHILD. Our R&D team continues to build clinical knowledge by studying eutrophia in PHILD patients in the open-label assent 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 inhaled-through-prosinol program. Given the proximity of a potential launch, I'd like to spend a little time framing the potential market opportunity for eUTREPIA as we see it, both in PHILD and PAH. With regard to PHILD, patients. However, these estimates likely undervalue the total addressable population since those calculations rely on historical publications before the field had effective tools to treat the disease and therefore reasons to diagnose the disease. With inhaled tryprosinol as the only approved mechanism to treat PHILD, the market penetration is still in its infancy. we believe there is significant growth in this market. Total inhaled tryprosinol revenues currently sit at about a 1.3 billion annual run rate in the U.S. alone. With regard to PAH, we also believe that eutrophia has potential for significant uptake. We view eutrophia as having the potential to not only be the best-in-class inhaled tryprosinol, given its dosing flexibility and ease of use, but also the first-in-choice process cycle. Specifically, patients who previously considered the oral prostacyclines as their starting choice can now avoid the significant and limiting off-target GI toxicities associated with these drugs while still achieving therapeutic doses. Thus, combining current sales of oral prostacyclines of approximately $1.6 billion in pH with the recently reported sales from inhaled reposadol of $1.3 billion, the market opportunity for uterative could be approaching $3 billion and growing incrementally as the PHILD patients still remain largely untreated. With its unique and differentiated approach, it's exciting to think about the potential for eutropia and the rapture value in both of these markets. At this time, I would like to ask Rusty to summarize the next steps towards final.
spk05: 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 Utrepia's approval. Second, the recent attempts by United Therapeutics to assert new legal theories to block approval of Utrepia. 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 Utrepia 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 Utrepia 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 Tyveso for the PHLD indication must expire, which will occur on March 31st. Once both of these have occurred, the FDA will have the ability to issue final approval for eutrepia for both the PAH and PHILD indications. We will 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 eutrepia 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 are likely to succeed on the merits in those actions. We believe that this burden will be a challenge for them to meet based on the laws and the facts at issue. The first request for preliminary injunction is directed to the second Hatch Waxman lawsuit alleging infringement of the 327 patent in the treatment of PHLD. Issued after the eutrepia NDA was submitted, and after Liquida amended its NDA to add PHLD to the label for eutrepia, the 327 patent covers the treatment of PHLD patients with Tybaso in accordance with the dosing regimen in the Tybaso label. As we have noted previously, there is considerable prior art that teaches the treatment of PHLD patients with Tybaso, including the 793 patent and multiple peer-reviewed publications in the decade prior to the priority date for the 327 patent that described positive results from treating PHILB patients with Tyveso. Courts have generally refrained from issuing preliminary injunctions in situations where there are substantial questions as to the validity of a patent. And in light of all the prior art here, we believe substantial questions of validity of the 327 patent exist. The second request for preliminary injunction is directed at United Therapeutics recently filed suit against the FDA under the Administrative Procedures Act. Filed in the United States District Court for the District of Columbia, this suit alleges that the FDA mistakenly permitted Liquidea to amend the NDA for Eutrepia to add the PHLB indication. We have intervened in the case and are now a party to the case alongside the FDA. First and foremost, the FDA did in fact accept our amendment to the NDA for review, and we believe that the FDA's acceptance of our amendment for review was proper and in full accordance with current FDA regulations. United Therapeutics' argument that an amendment to add a new indication is improper is based primarily on a non-binding 2004 FDA guidance document, ignoring subsequent FDA regulations adopted in 2016 that expressly contemplate the possibility of adding new indications through an amendment. Secondly, even if United Therapeutics was correct that the amendment was improper, That would not mean that United Therapeutics would receive a new 30-month stay, as they have argued. For instance, even if the amendment was now rejected by the FDA, Liquidia could simply supplement its NDA after approval to add the PHILD indication. The exact same process used by United Therapeutics to add PHILD to the label for Tyveso. Critically, the statute expressly states that amendments and supplements are treated the exact same way in determining whether a patent can give rise to a 30-month stay. meaning that only those patents submitted to the Orange Book prior to the filing date of the original NDA, not the filing date of the amendment or supplement, can give rise to a 30-month stay. Briefing on the motion for preliminary injunction will be completed on March 18th, and a hearing will be held on March 29th. We look forward to this matter being addressed in short order. In summary, Liquidia sees the path to launching the Atrapia in two straightforward actions, removal of the injunction and approval of the products. The rest is a last-ditch attempt by a competitor to make any and every argument they can to maintain their monopoly and deny patients access to a meaningful treatment option. We have long anticipated the possibility that United Therapeutics could engage in such a flurry of activity as we meet near clearance of the original Hatch Waxman litigation, and we are prepared to meet them head-on. With that, I will pass to Mike for a review of last year's financials.
spk27: 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 will 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 proportional injection. 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 PhRMOSA 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 EUTREPIA program increased to $13 million from $6.7 million the year prior, primarily due to increased manufacturing of prelaunched commercial supply and the startup of our ascent study. and consulting expenses, including stock-based compensation expense, also increased $5.1 million, primarily due to increased headcount to support the potential commercialization of Utrepia. 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 Utrepia. 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 and 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.
spk18: Thank you, Mike. 2024 is shaping up to be the transformational year at Laquidia. We are fourth 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.
spk07: Thank you. To ask a question at this time, please press star 11 on your telephone and you will hear an automated message advising you your hand is raised. To withdraw your question, please press star 11 again. One moment while we compile our Q&A roster. 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.
spk01: Good morning. This is Mary Kate. I'm 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 PHILD? Maybe why or why not? And do you anticipate equal interest in both indications? Thanks.
spk18: Good morning, Mary Kay. Thanks for the question. So we're fortunate to have Scott Mumar, our chief commercial officer, on the phone. Scott, maybe if you would like to opine on that.
spk15: Yeah, 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 eutrapia because it is so convenient and because of the titration 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. And PHILD, 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 PHILD and then getting physicians to look for it and then getting them to treat it. And, you know, 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 eutropia. And if they would be willing to use eutropia, 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 pH center of excellence where it would be treated. So I think that's a brief summary of how we'll approach the two markets.
spk12: Great. Thank you.
spk18: And maybe I'll just add a few comments. So I think 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 for osteocycline. And the reason I think we can do that is because really with eutropia and its ability to titrate to doses that are on order of threefold more than what was originally possible with Tyveso, We've changed the 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 Optravi, 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 its 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 arenatram is a very similar story. It's a used TID. It's titrated to effect. It's indicated to delay disease progression and improve 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. And in fact, Uther has said lately that because it's so difficult to titrate, they are actually promoting titration therapy via the parenteral route, and then transition to oral. So you can see this is burdensome and onerous. What eutropia will then do is negate completely these off-target effects to the GI tract and allow those titrations. 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 tactfully after we position ourselves as the best-in-class inhaled prostacyclin, as Scott mentioned, for both PAH and PHILD. Operator, next question, please.
spk06: One moment for our next question. And our next question is going to come from the line of Julian Harrison with BTIG.
spk07: Your line is open. Please go ahead.
spk14: 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 PHILD via supplement instead of the current arrangement, your view is that filing a supplement with the 327 patent now in the Orange Book should not trigger an automatic stay. Am I understanding that correctly?
spk18: Yes. Thanks for the question, Julian. Good morning. I suggest you answer that, please.
spk05: Yes, so let me clarify maybe a couple things. So first, I think our view is that, you know, the amendment being rejected and us having to file this by a supplement is sort of the worst-case scenario. You know, 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 and do a supplement, then that's exactly right. So if you look at the statute, again, it's 21 U.S.C. 355 C.3.C., you know, what it specifically defines, those patents that can give rise to a 30-month stay. And critically, it says, you know, only those patents, and I'll quote it, before the date on which the application, and then in parentheses, 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.
spk11: Very helpful. Thank you.
spk18: Thank you, Jillian. Operator, next question, please.
spk07: One moment. And our next question is going to come from the line of Serge Bellinger with Needham. Your line is open. Please go ahead.
spk29: Hi, good morning. Thanks for taking my question. 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 PHILD approval. Have they asked for additional info or given you any additional 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 liquidity petition late last month and just what it means to the overall legal proceedings. Thanks.
spk18: Thanks, Serge. Good morning. I'll break this into two parts. So Rajiv 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. Rajiv?
spk20: Yeah, thanks, Roger. Hi, Serge. Good morning. So in regards to our, you know, we continue to, you know, believe very strongly, as Rusty already alluded to, that the amendment we filed to add on the indication for PHLD 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 31st 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 PHILD, and we remain confident in that matter. I'll turn it over to Rusty to answer your second question.
spk05: Sir, thanks for the question. So, the Supreme Court case really has no bearing. 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. you know, 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, you know, 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.
spk18: Thank you. Yeah, maybe, Serge, I'll just add a little bit to Rajiv. So I think... You know, the one communications we've had, obviously we missed the January 24th 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 potential of the clinical exclusivity to expire at the end of March. So as Rajiv said, we're looking now. The amendment was filed and asked for full approval even when we filed it in July for both PH and PHLD. 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 31st clinical exclusivity expiration. Operator, next question, please.
spk07: Well, one moment. And our next question is going to come from the line of Matt Kaplan with Ladenburg-Tholomon. Your line is open. Please go ahead.
spk19: Oh, thank you. 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 exclusivity expiration is just a date on the calendar, so that's easy.
spk18: Yeah, 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 them the power to remove it, and I think he was just waiting to see the hearing request denied and then the mandate to issue, which will happen next week, I think next Tuesday is when it should issue. 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 this pending PI, how that interplay may impact this.
spk05: 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 of the rehearing request that was issued by the Federal Circuit. So again, he has all the information in front of him. Obviously, they have the new case, the 327 patent case, where they've also requested a preliminary injunction. You know, those cases really don't relate to one another. They're both in front of the same judge, and obviously the patent system is similar, but procedurally, you know, the injunction that currently exists isn't tied to their request for a new injunction on the new patent. So, you know, I don't think there's any interdependency there between the two actions.
spk18: And that's why we're not given a specific day when we think, you know, the approval action will happen, but Again, other than it'll be after March 31st when the exclusivity expires. Right, right. Okay, great.
spk19: 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 needing one additional study for approval in both PH and PHILT. Can you give us a sense in terms of the timeline of that as well?
spk18: Yeah, I'd love to. So Rajiv is also overseeing that effort. So Rajiv, if you wouldn't mind answering the question.
spk20: Yeah, thank you, Matt, and good morning to you as well. So as you alluded to, L606 is our liposomal formulation of sustained release for truprosanil 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 three program. The program is designed using a similar strategy like we have with Eucrepia. So this is a 505B2 pathway with the label drug being Tyveso. 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 1 PAH as well as Group 3 PHLD. In that regard, as you stated, we specifically have chosen the indication for PHLD to take into a global large Phase III study. That study is gated to initiate sometime near Q4 of 2024.
spk18: Great. Thank you, Rajiv. And I think the one thing to add to that is, you know, I guess the other part of your question is how long will that take? You know, I think because there's such a scarcity of treatments for PHLD, 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 brushstrokes, we're looking at a from first patient in to an FDA decision is probably in the three-and-a-half to four-year range, Matt.
spk17: Next question, please, operator.
spk07: And one moment for our next question. Our next question is going to come from the line of Kamboos Yazdi with Jefferies. Your line is open. Please go ahead.
spk10: Morning, Payne. 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 re-amendment versus NDA for indication expansion with regards to tentatively approved drugs? Do we have any precedents there? Thank you.
spk18: Yeah, I think both of those questions are in Rusty's court, so if you wouldn't mind, Rusty.
spk05: Sure. So on the first question for the 327 patent, so there is a briefing schedule on that. You know, United Therapeutics has filed their brief requesting in support of their request for a preliminary injunction. Our response is due, currently due on April 5th. Their reply then would be due April 19th. 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 and getting approval and launching. So the burden's 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 is pointing to, which they claim stands for the proposition that you can never add an amendment to a pending NDA. I'm sorry, never add an indication to a pending NDA. However, you know, the 2016 regulations and the site is 21 CFR 314.60 subsection expressly contemplates situations where new indications could be added to a pending NDA, including a 505 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. you know, as evidenced by the regulations.
spk18: And if I may add, you know, 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 dose form, same formulation, and no new data, so we think we're well within the statutes that Rusty just described.
spk13: Great.
spk10: Operator? I guess one other follow-up is on the assent trial. What kind of patient populations are being studied, and how is enrollment proceeding there?
spk18: Yeah, great question. We appreciate that. So, Rajiv, if you wouldn't mind.
spk20: Yeah, thanks, Kambiz. So once again, the SENS 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 PHLD that are naive to any therapy are then placed onto eutropia, which is 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 PRINC 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 treprosanil, 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 rate 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?
spk09: Thank you, Rajiv.
spk18: And I think it's a great question. I think the other thing, you know, again, why this data is important, I think when you look at the data that's, some of the data that's been published on United Therapeutics' Tabeza DPI, particularly the data out of the National Jewish Center in Colorado, you can see that there's been some difficulty with the TPI 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 naive 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 Uther launched a few years ago, the assumption was that they would convert that entire market quite quickly 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 eutropia. So if this data bears out the way we think it will, then that will certainly augur that this is the best-in-class therapy and first-in-choice therapy. Next question, please.
spk07: I'm showing no further questions, and I'd like to hand the conference back over to Roger Jeffs for further remarks.
spk18: 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, Liquida will be providing to patients what we feel is the preferred product for inhaled tripostinol, and it will come at a critical time as the market for inhaled tripostinol rapidly expands. Thank you, and have a good day.
spk07: This concludes today's conference call. Thank you for participating. You may now disconnect.
Disclaimer