Liquidia Corp Q4 FY2023 Earnings Call

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. 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 for your questions.

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 inhaled Treprostinil program. Given the proximity of a potential launch, I'd like to spend a little time framing the potential market opportunity for YUTREPIA as we see it, both in PH-ILD and PAH. With regard to PH-ILD, current treatment 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 Treprostinil as the only approved mechanism to treat PH-ILD, the market penetration is still in its infancy. We believe there is significant growth in this market. Total inhaled Treprostinil revenues currently sit at about a $1.3 billion annual run rate in the US alone. With regard to PAH, we also believe that YUTREPIA has potential for significant uptake. We view YUTREPIA as having the potential to not only be the best-in-class inhaled Treprostinil, given its dosing flexibility and ease of use, but also the first-in-choice prostacyclin. Specifically, patients who previously considered the oral prostacyclin 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 prostacyclins of approximately $1.6 billion in PAH with the recently reported sales from inhaled Treprostinil of $1.3 billion, the market opportunity for YUTREPIA could be approaching $3 billion, and growing incrementally as the PH-ILD patients remain largely untreated. With its unique and differentiated approach, there is potential for YUTREPIA to add significant value in both of these markets. At this time, I would like to ask Rusty to summarize the next steps towards final FDA approval.

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 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 and 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 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. Their first request for preliminary injunction is directed at the second Hatch-Waxman lawsuit alleging infringement of the 327 patent in the treatment of PH-ILD. Issued after the YUTREPIA NDA was submitted and after Liquidia amended its NDA to add PH-ILD to the label for YUTREPIA, the 327 patent covers the treatment of PH-ILD patients with Tyvaso in accordance with the dosing regimen in the Tyvaso label. As we have noted previously, there is considerable prior art that teaches the treatment of PH-ILD patients with Tyvaso, 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 PH-ILD patients with Tyvaso. The 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. A 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 Liquidia to amend the NDA for YUTREPIA to add the PH-ILD 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. 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 PH-ILD indication, the exact same process used by United Therapeutics to add PH-ILD to their label for Tyvaso. 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 18, and a hearing will be held on March 29. We look forward to this matter being addressed in short order. In summary, Liquidia sees the path to launching YUTREPIA in two straightforward actions: removal of the injunction and approval of the product. 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 near clearance of the original Hatch-Waxman litigation, and we are prepared to meet them head on.

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 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'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, which 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. 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.

Thank you, Mike. 2024 is shaping up to be a transformational year 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. Our first question comes from 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 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 line. Scott, maybe if you would like to opine on that.

Yes. So, there's a little bit of difference in strategy. 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. 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. 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. We'll be out in the community with community pulmonologists, helping educate them that this condition exists and that it's deadly. 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. In PAH, we would like to be the first-in-choice prostacyclin. The reason I think we can do that is because really with YUTREPIA and its ability to titrate to doses that are on the order of threefold more than what was originally possible with Tyvaso, we've changed the therapeutic index of that molecule. 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. If you look at the 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. The 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. Similarly, Orenitram has a very similar story, titrated to effect, indicated to delay disease progression and improved six-minute walk distance. 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. 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 transitioning to oral. YUTREPIA will negate these off-target effects to the GI tract and allow dose titration. We will be looking at the oral prostacyclin market as a significant market where we can gain share, tactically repositioning ourselves as the best-in-class inhaled prostacyclin, for both PAH and PH-ILD.

One moment for our next question. 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 the 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 a couple of things. 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 file a supplement, then that's exactly right. The statute specifically defines those patents that can give rise to a 30-month stay. The statute expressly states that amendments and supplements are treated the same, meaning that only those patents submitted to the Orange Book prior to the filing date of the original NDA can give rise to a 30-month stay. Therefore, even if we were required to file a supplement, the result would be no new 30-month stay.

Very helpful. Thank you.

Thank you, Julian. Operator, next question please.

One moment. 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 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. Rajeev oversees our regulatory group, so he can answer the first question regarding interactions with FDA. And then Rusty, if you'll address the Supreme Court question. Rajeev?

Yes, thanks, Roger. Hi, Serge. Good morning. We continue to believe very strongly, as Rusty already alluded to, that the amendment we filed to add the indication for PH-ILD remains appropriate. 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. We anticipate that date is shortly arriving on May 31 when the clinical exclusivity ends, leading to full approval for both indications for PAH and PH-ILD. We remain confident in that matter. I'll turn it over to Rusty to answer your second question.

Serge, thanks for the question. 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. The Supreme Court didn't take up that appeal, but again, it all relates to the 793 patent, which has been invalidated at this point and confirmed twice by the federal circuit. So, with that decision from the federal circuit, the Supreme Court decision doesn't impact how this is going to play out at all.

Yes, maybe, Serge, I'll just add a little bit to Rajeev. So, the one communication we've had is that we missed the January 24 PDUFA action date. The reason for that has been communicated as the FDA awaiting the removal of the injunction. As Rusty said, there are two things that need to happen principally for us to get full approval: the injunction removal and then the potential expiration of the clinical exclusivity at the end of March. We expect to skip the tentative approval phase and go directly to full approval after March 31.

One moment. Our next question is going to come from the line of Matt Kaplan with Ladenburg Thalmann. Your line is open. Please go ahead.

Good morning. Roger, just to follow up on that question 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. I think he was just waiting to see the rehearing request denied, and then the mandate to issue, which will happen next week. At that point, he'll be fully empowered to do what he needs to do. Rusty, you can comment on how you see the pending preliminary injunction request and its interplay with this.

On the existing injunction, what you laid out is exactly correct. It's been fully briefed in front of Judge Andrews so that he has what he needs. We also supplemented what we had submitted to him to provide him with the denial to rehear the request issued by the federal circuit. Again, he has all the information in front of him. They have the new case, the 327 patent case, where they've requested a preliminary junction. Those cases really don't relate to one another: they’re both before the same judge and are procedurally independent. We don't see any interdependency 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 it will be after March 31 when the exclusivity expires. Yes, I'd love to. Rajeev, if you wouldn’t mind answering the question about L606.

Yes, thank you, Matt, and good morning to you as well. L606 is our liposomal formulation of sustained-release Treprostinil, which will be delivered twice a day with a smart portable nebulizer. We're really excited about this program as it's the leading effort towards a Phase III program. Our strategy is similar to what we have with YUTREPIA. We had confirmation that a single placebo efficacy study with L606 would lead to approval for both indications, Group 1 PH and Group 3 PH-ILD. This study is set to initiate near Q4 of 2024.

Great. Thank you, Rajeev. The timeline from first patient in to an FDA decision is probably in the three and a half to four-year range, assuming we can obtain the required enrollment quickly. This is considering there's a scarcity of treatments for PH-ILD, which may enable rapid enrollment.

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

On the first question for the 327 patent, there is a briefing schedule on that. United Therapeutics has filed their brief requesting a preliminary injunction. Our response is currently due on April 5th, and their reply will be due by April 19. The court will then either schedule a hearing or not and make a decision. However, if there's no preliminary injunction in place, nothing blocks us from moving forward to get approval and launch. The burden is on United Therapeutics to secure a preliminary injunction before that happens. On the FDA position regarding amendments versus supplements, if you look at the FDA's existing guidance, there is a non-binding 2004 guidance that United Therapeutics refers to, claiming that you can never add an indication to a pending NDA. However, the 2016 regulations expressly contemplate situations where new indications could be added to a pending NDA, including a 505(b)(2) NDA like ours.

If you look at the guidance that United Therapeutics is referring to, it points to bundling guidance, which is more specific to changing the route, dosage form, or formulation and providing new data. We're not doing any of that; it's the same route, same dosage form, same formulation, and no new data. We believe we are well within the statute that Rusty described.

The ASCENT study is something that we are extremely excited about as it's something that's needed in the literature. It will include patients recently diagnosed with PH-ILD that are naïve to any therapy, which will highlight tolerability and titratability improved by our PRINT technology. The combination will allow the use of a low resistance inhaler to deliver dose profiles required to achieve adequate therapeutic goals, such as improved walk distance and overall clinical outcomes. Current enrollment rates are encouraging, and we expect to complete enrollment of 60 patients by the end of this year.

Kambiz, great question. When looking at published data on United Therapeutics' Tyvaso DPI, it's interesting that there were high dropout rates among patients who were naïve to prostacyclins. There's still a retained population of nebulized patients as well. If this data confirms our expectations, it will indicate we offer the best-in-class and first-in-choice therapy. Operator, please move to the next question.

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. With no further questions, we again 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 believe is the preferred product for inhaled Treprostinil 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.