Earnings Call Transcript - LQDA Q2 2023

Operator, Operator

Good morning, and welcome everyone to the Liquidia Corporation Second Quarter 2023 Financial Results and Corporate Update Conference Call. My name is Livia, and I will be your conference operator today. Currently, all participants are in a listen-only mode. Following the presentation, we will conduct a question-and-answer session. Instructions will be provided at that time for you to queue up for questions. I would like to remind everyone that this conference call is being recorded. I will now hand the conference call over to Jason Adair, Chief Business Officer.

Jason Adair, Chief Business Officer

Thank you, Livia. It's my pleasure to welcome everyone to Liquidia's second quarter 2023 financial results and corporate update conference call. Joining the call today are Chief Executive Officer, Dr. Roger Jeffs; Chief Financial Officer, Michael Kaseta; General Counsel, Rusty Schundler; and Chief Medical Officer, Dr. Rajeev Saggar. Before we begin, please note that today's conference call will contain forward-looking statements, including those statements regarding future results, unaudited and forward-looking financial information as well as the company's future performance and/or achievements. These statements are subject to known and unknown risks and uncertainties, which may cause our actual results or performance to be materially different from any future results or performance expressed or implied on this call. For additional information, including a detailed discussion of our risk factors, please refer to the company's documents filed with the Securities and Exchange Commission, which can be accessed on our website. I would now like to turn the call over to Roger for our prepared remarks, after which, he will open the call up for your questions.

Roger Jeffs, CEO

Good morning, everyone and thank you for joining us. In our opening remarks, today, we're going to take a very focused approach to address the issue that is most top of mind for our company, our employees, and our shareholders, specifically the path forward as we see it, to the successful resolution after litigation and launch of YUTREPIA for both PAH and PH-ILD. I will note, however that in addition to the significant legal derisk, which Rusty will talk about shortly, we also achieved other major and important milestones in the quarter, most notably the license of L606, a Phase 3 clinical program for the twice-daily liposomal formulation of inhaled treprostinil. That positions us with the best-in-class portfolio of inhaled treprostinil products to best address patient needs, not only today, but also in the future. As mentioned, the bulk of our prepared remarks will focus on the recent legal and regulatory actions related to the ongoing litigation. I have asked Rusty to elaborate on four specific points. First, the favorable affirmation by the Federal Circuit that we do not infringe any valid claims of the 066 patent. Second, our confidence that United's attempt to overturn the PTAB decision on the 793 patent will fail. Third, the positive impact in submitting an amendment to add the PH-ILD indication to YUTREPIA label. And lastly, our confidence in why we feel that the recently allowed patent claims to United Therapeutics related to the treatment of PH-ILD will not be an impediment to YUTREPIA.

Rusty Schundler, General Counsel

Thank you, Roger. As a reminder, Liquidia has been party to two separate appeal proceedings at the Federal Circuit that are relevant to the launch of YUTREPIA. Broadly speaking, the appeals relate to two patents asserted against Liquidia, the 066 patent, which describes a way of making and storing treprostinil, and the 793 patent, which describes the method of use to treat patients with pulmonary hypertension. Before walking through the recent decisions and activities, I would like to point out that our guidance over the last 12 months is still the same. We believe the ongoing litigation will be concluded between the end of 2023 and the middle of 2024, clearing the path to final approval and launch of YUTREPIA. The only thing that has changed in the last year is our increased confidence in our guidance with each legal decision. Now moving to the recent decision. On July 24th, the Federal Circuit affirmed the District Court's decision from last August in the Hatch-Waxman litigation. The outcome of the appeal was in line with our expectations, meaning the claims of the 066 patent were affirmed as obvious unpatentable and thus invalid, and that YUTREPIA does not infringe the single valid 066 patent claim that was asserted against us. The Federal Circuit also affirmed that YUTREPIA infringes the asserted claims of the 793 patent and that based solely on the arguments presented in the Hatch-Waxman litigation, the 793 patent is valid. However, the court also commented in the written decision that the court is aware that the Patent Trial and Appeal Board, or PTAB, has found all of the claims of the 793 patent to be unpatentable and that the PTAB decision is on appeal, which I will discuss shortly. As we've noted previously, should the PTAB decision be affirmed on appeal, the 793 patent would be completely invalidated and all previous rulings related to the alleged infringement of the 793 patent would be dissolved. Liquidia would then be free to seek final approval from the FDA for YUTREPIA. As next steps with respect to this Federal Circuit ruling, it is possible that one or both parties could seek a rehearing by this three-judge panel and/or a hearing en banc in front of the full Federal Circuit. One or both parties could also file for certiorari with the United States Supreme Court. However, we see nothing in the Federal Circuit's decision regarding the 066 patent that we believe is likely to lead to any further rehearing or certiorari being granted. Even if a rehearing or certiorari is granted, it is important to remember that all four judges who have ruled on the 066 patent between the District Court and Federal Circuit have found the 066 patent claims to be invalid or not infringed. Regardless, our ability to seek final approval for YUTREPIA is not contingent on the conclusion of rehearing or appeal of the affirmed Hatch-Waxman decision. The proceeding that is currently limiting our ability to seek final approval for YUTREPIA is United's appeal of the PTAB's decision which invalidates the 793 patent, which I mentioned briefly earlier. To summarize, all of the 793 patent claims have been ruled by the PTAB to be unpatentable. Their first ruling was in July 2022. The merits of Liquidia's arguments were further reinforced in February 2023 when the PTAB denied United's request for a rehearing and reaffirmed that all of the claims are obvious over publicly accessible prior art. In April, United appealed the PTAB's decision to the Federal Circuit and briefings should be completed in the fourth quarter of this year. Once briefing is completed, the Federal Circuit has ordered oral arguments to be scheduled on the next available date in its calendar, which we expect to be in the late fourth quarter of 2023 to early 2024. Once heard, the Federal Circuit could issue its ruling by one of two procedures. First, the court could issue a simple summary affirmance of the PTAB's decision within a few days after oral argument. Or second, the court could issue a full written opinion, in which case we would anticipate likely receiving the decision within a few months after oral argument, similar to the timing of the Hatch-Waxman appeal decision. We will not predict which of these decision passes is unlikely. However, whenever a favorable decision is issued, Liquidia will immediately seek final regulatory approval for YUTREPIA. With these timeframes in mind, we continue to believe that the ongoing litigation will be concluded sometime between late 2023 and early 2024. I'd like to turn now to the amended NDA that Liquidia submitted to request the addition of the PH-ILD indication to the proposed label for YUTREPIA. The amendment was filed on July 24, the same day that we received the decision in the Hatch-Waxman appeal. Due to the nature of the amendment, we were required to issue a second Paragraph IV notice that certified as of the date of the submission that the six patents listed for Tyvaso in the Orange Book are invalid and/or not infringed by YUTREPIA. Three of those patents, the 066, 901, and 793 patents, are the same three patents that have been litigated over the last several years and have been found to be invalid or not infringed by YUTREPIA. The other three patents in the Orange Book for Tyvaso are directed specifically to the nebulized delivery of treprostinil and are completely unrelated to YUTREPIA, and were not asserted against Liquidia in the original Hatch-Waxman litigation. Although, it is possible that United could file a new Hatch-Waxman lawsuit based on this amended NDA, the existing Federal Circuit decisions on these 066 and 901 patents and the future favorable affirmance of the PTAB's invalidation of the 793 patent would be binding once finalized on appeal. Under well-settled legal principles, United cannot maintain a second lawsuit for infringement of the same old patents against the same YUTREPIA products. Even if a new lawsuit is filed and a new 30-month stay at the FDA is triggered, that lawsuit would effectively end upon completion of the 793 appeal because all issues in the new lawsuit would have been decided and binding at that time. Thus, although it's possible that the amended NDA could trigger further litigation from United, we do not anticipate any material change to our timeline. Finally, I want to address the new patent claims allowed to United at the end of June, which covered the treatment of PH-ILD patients with inhaled treprostinil. We expect the patent will issue in the coming weeks and likely be added to the Orange Book for Tyvaso. Two main questions we have received have been A, how do these claims impact the FDA's approval of YUTREPIA for PH-ILD? And B, how could the USPTO grant these claims given the unpatentability of the 793 claims to treat patients with all forms of pulmonary hypertension? I will address each of these in turn. As the first question, it is important to note that because the new patent was not listed in the Orange Book at the time we submitted our NDA amendment, there will be no 30-month stay at the FDA that attaches to this new patent. While we expect United may file a lawsuit alleging that Liquidia infringes this new patent, we would not automatically be delayed in our ability to seek final approval for the PH-ILD indication. Instead, the burden would be on United to seek and prevail on obtaining a preliminary injunction. To do so, the burden would be on United to demonstrate among other things that they are substantially likely to prevail on the merits of the case. Historically, the courts have generally declined to grant preliminary injunctions in situations where there are substantial questions as to the validity of the patented issue. This brings us to the second question. How could the USPTO grant these claims given the unpatentability of the 793 claims to treat all forms of pulmonary hypertension? As you know, we cannot reveal the details of our legal positions. That being said, we strongly believe that this new patent will be found to be invalid because of substantial prior art that predates the priority date of this new patent application and fully anticipates all of these new patent claims. For example, the 793 patent itself, which was filed in 2007 and predates this new patent implication by more than 10 years, already covers and closes the same treatment of inhaled treprostinil to patients with all groups of pulmonary hypertension, including PH-ILD as United itself has argued in court. In addition, over the last 10 to 15 years, many physicians have conducted and published studies and analyses regarding the treatment of PH-ILD patients with treprostinil including inhaled treprostinil. In fact, our own Chief Medical Officer, Rajeev Saggar, explored these of treprostinil to treat PH-ILD patients almost 15 years ago, measuring the same basic endpoints that are identified in this new set of patent claims. A great many of these publications predate United's new patent application by a number of years and constitute prior art to the new patent. Ultimately, this new patent will likely be litigated, but it is fundamental to patent law that a patent that is not novel and covers methods of treatment that were already widely known will not be valid. Accordingly, we strongly believe this new patent will not affect Liquidia's ability to commercialize YUTREPIA. In summary, the merits of Liquidia's arguments remain sound and if affirmed will open the door to treating patients in the near future, and we do not view this new patent as having any impact on that result. I'll now pass the call on to Mike to briefly address our financial reporting.

Mike Kaseta, CFO

Thank you, Rusty and good morning, everyone. Our second quarter 2023 financial results can be found in the press release and the 10-Q filed this morning. Broadly speaking, the company continues to execute and manage its business activity with financial discipline in mind. We ended the second quarter with $88.2 million in cash, equating to a net burn of only $5.1 million over the first six months of this year. During the quarter, revenue from treprostinil injection increased $0.9 million compared to the same quarter last year due to favorable gross to net charge back and rebate adjustments, while cost of sales remained flat at $0.7 million. R&D expenses in the quarter increased $12.5 million compared to the second quarter of 2022, primarily due to the $10 million upfront payment tied to licensing North American rights to L606 from Pharmosa Biopharma and an expense which has been offset by the technical difficulty. Overall, the company remains well-positioned financially through the key value-creating milestones tied to the resolution of the litigation. We are preparing to launch YUTREPIA with speed, building a pipeline with new products and remaining opportunistic in our ability to create value going forward. With that, I'd like to now turn the call back over to Roger.

Roger Jeffs, CEO

Thank you, Mike and thank you, Rusty, for clearly articulating why the merits of our case give us great confidence. And importantly, while we anticipate our timeline for legal clarity to remain as we have been saying, specifically between the end of 2023 and of 2024. With that, I would now like to open the call for questions. Operator, first question, please.

Operator, Operator

Thank you. Our first question comes from Greg Harrison with Bank of America. Your line is open.

Mary Kate, Analyst

Good morning. This is Mary Kate on for Greg. Thank you so much for taking our question. I guess, looking at L606 here, where do you see this fitting into the treatment paradigm for PAH and PH-ILD? And do you think there are certain patients who will likely prefer this to a DPI? Thank you.

Roger Jeffs, CEO

Yeah. Thank you. Good morning. We appreciate the question. Rajeev, if you would, please answer that.

Rajeev Saggar, Chief Medical Officer

Thank you, Mary Kate. Good morning. Regarding L606, it is a liposomal formulation of treprostinil created for extended pharmacokinetic plasma levels over 12 hours. Because of its design, it achieves a significantly lower Cmax, approximately eight times lower than Tyvaso. This aspect is crucial as it helps reduce some of the main side effects associated with peak plasma levels seen with Tyvaso while maintaining a similar AUC. Essentially, this allows for consistent, stable 24-hour exposure with twice-daily dosing, compared to Tyvaso, which requires four doses a day and does not provide coverage during sleeping hours. We expect that as we progress through the clinical studies, L606 will be adopted as a prime choice for both PAH and PH-ILD due to these clinical advantages.

Roger Jeffs, CEO

Next question?

Operator, Operator

One moment for our next question. And our next question coming from the line of Julian Harrison with BTIG. Your line is open.

Julian Harrison, Analyst

Hi. Good morning. Thank you for taking my questions and congrats on all the progress. First, just to confirm some of your prepared remarks, United's new PH-ILD patent does not preclude your ability to seek final FDA approval for YUTREPIA and PH-ILD. Did I understand that correctly?

Roger Jeffs, CEO

Rusty, do you want to address that?

Rusty Schundler, General Counsel

Sure. So, I think that is correct, unless United were to obtain a preliminary injunction. So, I think, as I commented on previously, there would be no 30-month stay that would attach to this new patent. And so, we would not be automatically prevented from obtaining approval for PH-ILD. Instead, the burden would be on United to obtain a preliminary injunction. And for the reasons noted during the prepared remarks, we think they'll have a hurdle to overcome to obtain that preliminary injunction.

Julian Harrison, Analyst

Okay. Great. Thanks for clarifying that. And then, can you just remind us of your clinical development plan in PH-ILD? You don't need clinical data for approval here, but I'm curious what data points you think would be most helpful to characterize for the medical community and generally speaking, are you able to comment on the timeline there?

Roger Jeffs, CEO

Yeah. So, it's correct, Julian. I'll answer the first part. We do not need any additional data to add PH-ILD to the label. And Rajeev, if you want to talk about the Phase 4 type studies that we're doing, if you will, to better inform the community about the use of YUTREPIA in PH-ILD patients.

Rajeev Saggar, Chief Medical Officer

Thank you for the question. To reiterate what Roger mentioned, the guidance from the FDA indicates that we do not require any new clinical study to amend the application for PH-ILD. Regarding YUTREPIA, we believe the major unanswered question is the use of a dry powder formulation of treprostinil. For patients with PH-ILD, it's important to note that Tyvaso was originally approved using a nebulizer. Therefore, we think that conducting a prospective, open-label study will address these unanswered questions about the utility of YUTREPIA, especially with our low-resistance inhaler. This research will clarify several clinical aspects. We expect it to demonstrate our improved tolerability profile seen in our INSPIRE study in PAH, and we anticipate similar results here. Additionally, we want to emphasize our capability to titrate the drug to higher doses, which is essential for achieving better clinical outcomes, such as improved walking distance. We believe that these patients would benefit from a more portable device like YUTREPIA, and we expect these improvements will be positively received by the PAH community. As for the study, we plan to initiate it towards the end of 2023, specifically in the United States. Thank you.

Julian Harrison, Analyst

Great. Thank you.

Roger Jeffs, CEO

Thank you, Julian. Thank you, Rajeev. Next question, please operator.

Operator, Operator

Our next question coming from the line of Serge Belanger with Needham. Your line is open.

Serge Belanger, Analyst

Hi, good morning. Just a couple questions. I guess, the first one on the recently filed NDA amendment for the PH-ILD indication. Just wondering about the next steps, is it kind of the standard FDA acceptance within 60 days? And do you expect a tentative approval from the FDA? I think you've talked about a six-month review process.

Roger Jeffs, CEO

The 30-day clock before they will indicate to us whether it's a type one or type two submission. Type one would be granted a two-month review and a type two submission would be granted a six-month review. We feel that it could be a type one resubmission. But again, we'll just wait to see what the agency says because the 30 days will come up in mid-October. But the good news there is then we would get potentially tentative approval for PH-ILD as early as October, or as late as early 2024, which again, we would be prohibited from launching into that indication until the market exclusivity ends in March of 2024. But that's the basic timeline. I think the other thing to point out, Serge, is that the tentative approval for PAH remains, and this is just an amendment to that tentative approval for PAH seeking tentative approval at this time for PH-ILD.

Serge Belanger, Analyst

Okay. And just thinking ahead of YUTREPIA launch, do you see the opportunity here as a kind of a switch from Tyvaso or would it be more of new patient starts that would begin with YUTREPIA, and does that differ between PAH and PH-ILD?

Roger Jeffs, CEO

I think it's largely a new patient start paradigm. I think, the way we view this is we want doctors to use it a few times, get comfortable with its use in a patient, quote unquote, who is de novo to prostacyclins. And then once that comfort base exists, then potentially they would switch. But I think the real opportunity here as we see it is more in the de novo patient base. There's a lot of turnover here. As you know, this is an unrelenting disease. Patients come on and come off the drug, their drugs commonly over time. So, for us, it's more going after the de novo market. The market that's already on a process like and can be a little bit stickier. I think we've acknowledged that. So, it's less about switches. Now, having said that, I think if there's intolerance and as we hear in particular with PH-ILD for Tyvaso and Tyvaso DPI, for example, those patients I think would be readily accepting of a DPI formulation that perhaps may be more tolerable and more titratable. We will pursue the switches, but our primary focus will be on the de novo patient market. There are many patients being treated, and current reports suggest over 6,000 are on therapy, roughly split 50-50 between nebulized and DPI formulations. Additionally, once we demonstrate the use and effectiveness of YUTREPIA in pulmonary arterial hypertension, we will target its prostacyclin as our preferred option, as it is gentler to take, easily titratable in the YUTREPIA format, and could potentially replace oral prostacyclins such as Orenitram and UPTRAVI. Thank you for the question, Serge. Operator, please proceed to the next question.

Operator, Operator

Thank you. And our next question coming from the line of Kambiz Yazdi with Jefferies. Your line is open.

Kambiz Yazdi, Analyst

Hi, team. How should we think about OpEx moving forward with the YUTREPIA open-label study and then eventually the L606 Phase 3? Thank you.

Roger Jeffs, CEO

Yeah. That's a great question. I'll ask our CFO, Mike Kaseta, please.

Mike Kaseta, CFO

Good morning, Kambiz. Thank you for your question. As I mentioned earlier, we closed Q2 with around $88 million in cash. We are very confident in our capacity to navigate key events in 2024, which includes onboarding our expanded sales force in Q4 of 2023. Once we receive the go-ahead to proceed with the launch of YUTREPIA, we will be fully prepared and ready to start immediately. We have strong confidence in that aspect. Regarding L606, as I indicated, we made a $10 million upfront payment to Pharmosa. We anticipate that the majority of development expenses will occur as we progress through 2024, particularly in the latter part of that year, and as the Phase 3 advances. Ultimately, we are very confident in our position once we have the clarity to move forward.

Roger Jeffs, CEO

Thank you, Mike. Thanks for the question, Kambiz. Operator, next question please.

Operator, Operator

Thank you. And I see no further questions in the Q&A queue at this time. I'll now turn the call back over to you, Dr. Jeffs, for any closing remarks.

Roger Jeffs, CEO

Thank you, operator. So, with no further questions, again, I'd like to thank you for joining us today. We look forward to reporting on our continued progress in the coming quarters. Goodbye.

Operator, Operator

Ladies and gentlemen, that does conclude our conference for today. Thank you for your participation. You may now disconnect.