Liquidia Corporation (LQDA) Earnings Call Transcript & Summary

Hello, all. Thank you for joining us this morning. My name is Harry Pearson. I'm a member of JPMorgan's investment banking team. I'm pleased to welcome you all to Liquidia Corporation's presentation and Q&A. I'm joined by Dr. Roger Jeffs, the CEO; Mike Kaseta, CFO; and Rusty Schundler, General Counsel, who'll give a presentation followed by Q&A. Take it away.

Wonderful. Thank you, Harry. Everyone, can you hear me okay? Yes. So we're delighted to be presenting. This is our first podium presentation for Liquidia at JPM, and I think it signals an inflection moment for the company as a whole. I'm joined today by Mike Kaseta, our CFO; Rusty Schundler, our GC; have Rajeev Saggar, our CMO; and Scott Moomaw, our Chief Commercial Officer; and also I'd like to welcome the board members of Liquidia that are here today as well. These are the regular forward-looking statements. I encourage you to read those and look to our filings for certain risks and uncertainties related to the stock and things I may or may not say today. We are very excited about the acceleration and the progress that we're making at Liquidia at the company. Our mission is to develop best-in-class therapies for patients with pulmonary hypertension, and we feel we've made significant strides towards that mission statement. Strategically, we're positioned to drive significant market presence in a rapidly expanding PH market across our 3 verticals, what we call the 3Ps: PRINT platform technology, products, which includes the pending approvals for YUTREPIA; and our pipeline asset, L606, which we licensed in this past year from our partner, Pharmosa, which is a twice a day sustained release formulation. With regard to PRINT platform technology, and I want to spend a little bit of time here. Everything that we do in the pulmonary hypertension space is going to be accelerated by the way that we use our nanomolding technology to produce precise uniform particles of precise shape and composition. These particles, we can really fine-tune into the appropriate range of the -- appropriate segment of the respirable range to maximize deep lung delivery. YUTREPIA, as you may know, has PH approval, tentative approval for PAH, and for PH-ILD, we -- in 2 weeks from today, we have a PDUFA date for that, which we're also seeking tentative approval. That approval, if granted, will be gated by market exclusivity that United Therapeutics has that ends in March. We also have a treprostinil for injection. It's a generic form of Remodulin that we are the commercial partner with our partner, Sandoz, and they talked to you about the sustained release formulation. But those are the 3 verticals where we're trying to maximize our business platform. So what are the markets? And how do they look like? PH and PH-ILD are highly attractive markets, and I'll break down -- if you don't know what pulmonary hypertension is, it's increased pulmonary pressure from vasculopathy in PH or scarring of lung tissue in PH-ILD, results in right ventricular hypertrophy and eventually death for these patients. So severe rare disease with continuing unmet need. In WHO Group 1, there's 40,000 patients treated today roughly. 3-year survival is better. It's 75%. 5-year survival is about 50%. So still a lot to do there. 4 mechanisms of action, 14 branded therapeutics and over a $5 billion market opportunity if you just look at pulmonary hypertension alone. Of that, about $2.7 billion is in what we call prostacyclin or prostacyclin-mimetic components of that. That's the market we're going after, both what's inhaled, what's in oral, and then also maybe infringing on the Remodulin franchise by having a durable therapy like YUTREPIA. WHO Group 3 is a different beast. It's -- it was first approved in 2001, a study in WHO Group 3 patients with PH-ILD for nebulized TYVASO. It's PH associated with lung disease. In this instance, I'm showing that there's about 60,000 prevalent patients, which we can distill from academic research, syndicated academic research. 3-year survival is very morbid, 35%. 5-year survival is even worse, about 14%. Currently, the only available option to these patients is inhaled treprostinil both in the nebulized and DPI format that United Therapeutics offer. If you look on the right-hand curve, prior to 2021, the approval of PH-ILD, and 2022, the approval of the DPI format, TYVASO was fairly static, about $400 million per annum. And you can see it's inflected a significant amount of revenue growth. And at the end of the third quarter, they reported $1.3 billion run rate across the nebulized and DPI format. So those are the markets that we're going to enter into. If you look at that more granularly, here's their -- they broke it down for the first time. Rapidly increasing demand for inhaled treprostinil. Really, the value proposition, though, seems to be porting to the DPI formulation, which we have with YUTREPIA. So again, stagnant through '21, ILD came to market, and then in '22, the DPI. If you look at the growth, the green bar is the DPI. Now the DPI accounts for about 65% of the inhaled treprostinil revenue. Only 35% remains with nebulized TYVASO. You can see it's -- the DPI is cannibalizing nebulized share as well. So while all that looks good, and the CAGR is impressive, the real-world reality is a little bit different. So here's the -- and I'll try to make sure I get these numbers right. Here's a prospective study from the National Jewish Health center that was published or abstracted at the PHPN Symposia in September. National Jewish is one of the largest and best pulmonary centers in the world, so a very capable expert in the treatment of patients with PH-ILD. They treated 26 patients that were naive to prostacyclins. So never experienced prostacyclins, but their disease demanded increasing therapy, so they put them on inhaled treprostinil in the DPI format. They also started 22 patients that were transitioned from TYVASO, nebulized to TYVASO DPI. Of the 26 that started, 18 discontinued in the naive subset group, so 69%. Of that, the main reason was clinical worsening and cough. In the naive group or the transition group, 22 started, 11 DCed, or 50% discontinuation. So of the 18 DCs in the first group, 11 went back to nebulizer. Of the 11 DCs in the second group, 7 went back to nebulizer. That tells you it's not the drug. So they're going back from TYVASO DPI back to nebulized formulation. So why is that? I think it's because they couldn't deliver the dose appropriately to the patients and titrate the dose to effect. So that's the first real-world experience from a large prospective cohort of patients. The time on therapy on average was between 3 to 6 months, so it didn't take long for the therapy to fail. So that's the market opportunity. You see there's a preference for portability and DPI use versus nebulizer. But the sustainability of the therapy, at least the way this one center used it, was not that impressive. So we think YUTREPIA is going to provide a much more attractive option. And why is that? So it's the first and only product candidate using our PRINT technology. If you look on the left, we can reliably and with high fidelity print 1.3-micron MMAD particles. It's in a trefoil shape, which mimics a natural form of pollen, and it's free flowing. But what you also see here is no aggregation or clumping. The particles remain independent, which is critical. On the right, this is a publication from one of our scientists in-house from 2012, where we looked within the respirable range. The respirable range is 1 to 5 microns, and we looked to see if we formulated particles at 4.6 microns, or 1.3 microns, which is what YUTREPIA is, look at the different distribution. And you can see on the right that the 1.3 microns performs beautifully. It goes to the distal airways, fills the lungs and less deposition in the upper airway and throat. That's what you want when you have a dry powder formulation. We have -- again, the history of what we did. How did we get this drug approved? It was through a 505(b)(2) pathway. We showed comparable pharmacokinetics. We have a tablet formulation of 79.5 micrograms, which is comparable to 9 breaths of TYVASO, and everything now is viewed as breath equivalence, like how do you match the TYVASO nebulized breath equivalent. So 79.5 for us matches 9 breaths equivalent of TYVASO given 4 times a day. We studied 121 patients, both naive and/or transitioned from TYVASO, and we've been continuing to treat patients since 2017, now with the longest duration of therapy of 5.5 years. We have seeking -- we have a PH-ILD indication, which we filed for approval -- an amendment for approval over the summer. PDUFA date is January 24, so 2 weeks from today, we'll get an answer on that application, hopefully. And critically, that application requires no additional clinical work, although we're beginning to do clinic work in PH-ILD to better inform our dosing strategy. What we've learned, at least in PH patients, through this open-label extension study is that as patients progress over time, so here it's showing 2 months, 12 months, 24 months, the doses on the left and the comparable breaths of TYVASO is on the right. The target breath dose is that gray bar. 9 to 12 breaths, that's what's in the indication label for TYVASO and TYVASO DPI. You can see, over time, the blue bars' median dose that were at 15 breaths equivalents, and at 24 months, 51% of patients are above 132.5 or 15 breath equivalents of TYVASO with a max dose now at 30 breaths equivalents. This is completely changing the therapeutic profile of inhaled treprostinil. So these are at levels in terms of dose that have never been seen before. This is the clinical value and utility of YUTREPIA. So the 4 elements that are critical to our success and why this will drive significant share are that it's a portable therapy, it's tolerable, it's titratable and durable. So simple pocket-sized device, convenient storage. We use a low-resistance pocket-sized inhaler, one here. Proven device, both in COPD and asthma, that physicians and patients are going to be very comfortable with. It's a Plastiape device. You just twist it, open the capsule, hold the capsule, pierce it, close it and then 2 breaths, you're done. That's the therapy. Do that 4 times a day based on your dose. We've shown that it's tolerable, that's why we're achieving the breath equivalents that we're achieving. So with inhaled nebulized TYVASO, the ceiling -- the target dose is 9 to 12, but patients have really struggled, and I'll show you some data to get above that 9 to 12 breath threshold. And we do it with a low resistance DPI. And the reason we can use low resistance, and this is going to be important from a marketing standard, is because the particles are free-formed, monodispersed and not agglomerated. You saw the pictures. The competitive DPI molecule is not that. It's aggregated because it's spray-dried on to what's called an FDKP backbone. That was -- that's a co-opted technology from their inhaled insulin program. Because of that, it clumps. You need a high-resistance device to break that up, and then that takes more energy in a patient population where the #1 symptom is shortness of breath, so probably not ideal for what you're trying to achieve. We've shown it's titratable. Again, I showed you the curves where both the median dose and the max dose are increasing admirably over time. And then we're trying to prolong the treatment. Again, it will help from -- it will help the patients and will help from a revenue curve the longer we treat. So our goal with this type of profile is to be the best-in-class and first-in-choice prostacyclin. And what does that mean? So that means we're not just going to try to take the inhaled market. We're going to go after the oral market. So when a physician decides to add a prostacyclin in pulmonary arterial hypertension, they add YUTREPIA, and that will delay the time for the need for a more onerous parenteral therapy. So dose matters. And again, I think this is well published data, and it's across all routes, but I'll just show you specifically for inhaled treprostinil first in PH and then I'll show it too in PH-ILD. So this is a retrospective analysis of 5,000 patients, so a large survey sample, from between 2009 and 2018. And it showed that if you got to 12 or more breaths in this data cut versus 9 breaths, you had a delayed transition to IV or subcu therapy, greater persistence on therapy and slightly improved survival rates. At the bottom, though, you'll see only 25% of patients -- this is real-world data -- were titrated to greater or equal to 12 breaths 4 times a day at TYVASO. So with a nebulized formulation, it's been very hard to achieve satisfactory doses. That's the limitation of inhaled treprostinil. In PH-ILD, similar story. So this is from the registration study for which PH-ILD was approved for a nebulized TYVASO and then bridged for TYVASO DPI. This time, the data cuts are 9 breaths, so greater than or equal to 9 breaths. You can see on the left if you got to 9 breaths or more, more clinical improvement in the red, and less clinical worsening in the blue. On the primary outcome measure, 6-minute walk distance, same thing. Better outcome the higher the dose, particularly prominent as you got above into the 10 to 12 breath range. So we're excited about that, because dose matters, and we have the therapy that can deliver the highest and most effective dose, we believe. And that will be manifest in our product labeling. So if you look at the products, people say, well, how do you differentiate? We're going to differentiate on formulation. So the PRINT technology formulation allows the use of a low-resistance device, which is easy for the patient to use. So it's highly user-friendly. Our label will encompass these higher doses. So if you look down to the dose titration described in the label, this is from our original submission. It included doses up to 24 breath equivalents of TYVASO. The competitive molecule only has 11 to 12 breaths as their target indicated dose. So we'll differentiate on the most critical aspect of the utility of a prostacyclin, and an inhaled prostacyclin in particular, as you've seen, by dose, and we'll do it with a low-resistance device. Our product requires no refrigeration during the product lifetime and no loss of powder or required device orientation to deliver. So if you drop the device, no spillage. Theirs is more sensitive and could spill drug if dropped. I'll talk briefly about the legal, just to frame it. In the Q&A, Rusty is here, so he can answer questions on process. So we had a Federal Circuit Court decision on December 20th that affirmed the invalidity of the '793 patent that the PTAB decision. That decision will override the previous District Court decision. So now in aggregate, all 3 patents that were asserted against us that were Orange Book listed, we found no claim that's either valid or infringed. So we've passed the substantive bar of what we want to do, and we think this point forward for PAH, it's more of just a process issue, how do we -- to turn the tentative approval that we have to a final approval. For PH-ILD, there is a second Hatch-Waxman lawsuit that was filed. So when we filed the amendment, we had to recertify against the Orange Book-listed patents. '793 was still in play at that time. Obviously, that will go away with the recent ruling. And the '327 patent, which we think is really -- we don't believe will -- we know it won't cause a regulatory stay, so that won't trigger a regulatory stay because it was not Orange Book-listed at the time of our original NDA application. So it's more is what -- that would just be more of a legal case of not what that patent is, and we've press released on Monday what we think about that patent. The next step then from process, we're going to ask the District Court to set aside the existing injunction. In fact, we did that on December 26. We have about -- we have the briefing schedules ongoing by January 23, and Rusty can talk about the details. The case will be fully briefed and in Judge Andrew's hands to release the existing injunction, and then it's just determined by his own sort of schedule as to when that will happen. In parallel, we're already in dialogue with the FDA on figuring out how we move the tentative approval to final approval. So they're aware of the affirmation in the Federal Circuit, and we're trying to work with them now, especially with the pending tentative approval for ILD in play also. How do we get both done, and on what time line? So the other question is, are you -- when will you be ready to launch? We're ready now. So that's the good news. So we have 50 sales reps that we onboarded in October. They're very scientifically fluent and commercially capable. They have 10 years of rare disease experience on average, and about 60% to 70% of them have direct PAH experience, as you can see from companies at the bottom. If not, they're the superstars from the rare disease field and are used to these high-touch patients, and how to promote and sell into this space. As I said, we put them in the field early. That was a bit at risk, but we wanted them trained and ready to go. Scott's had his team ready to go since January 1. So again, ready today. We're just waiting on the final things to happen from a process standpoint. We also, in 2023, strengthened our pipeline. I won't spend a lot of time on this one as we begin to get more data, but if you look here, it's basically liposomal-encapsulated treprostinil. The liposomal encapsulation extends the product PK from 4 hours to 12 hours, so it sets up for a twice a day versus 4 times a day regimen. It has a 7x lower Cmax, which is good. So you've extended the PK curve. So having a lower Cmax should improve the tolerability profile, and we match on AUC to Infinity when you do the proper PK comparisons. We've got this licensed with very strong proof-of-concept data. So there was an existing open-label ongoing study. There's 20 of the 60 -- that study is enrolling now. There's -- 20 of those 60 patients are in. Some have gone past 1 year, some are already at the 25 to 27 breaths equivalents. So tolerable, titratable, durable. So the key hallmarks of YUTREPIA seem to be porting to the twice a day formulation. And when you think about YUTREPIA and DPIs versus nebulized, we did 4 things: improved portability, tolerability, titratability and durability. The one thing we didn't change was regimen, still 4 times a day. L606 changes that fifth paradigm. So that's the value proposition here. We're going to start that study -- those plans by the end of this year. It will go through its regular 18 to 24 months enrollment. There's a precedent pathway. We'll just do the same study that UTHR did for TYVASO nebulized, the INCREASE study. 6 months to get the patients through the study, 3 months to analyze, 1 year for review and approval. So we think roughly 4 years from start, we can get to go low-hanging fruit, just precedent knowledge that -- in PH, the inhaled treprostinil will work. We've just got a longer-acting formulation of that. So excited about that. And it also will be highly portable. It's using a modern breath-actuated nebulizer, much different than what the current inhaled treprostinil format looks like. We had an FDA Type C meeting in December. We got agreement with the agency that the comparable -- so we already -- the comparable bioavailability was done when we acquired the program. The ongoing open-label safety study I've talked about, which is informing dosing and how will we design the algorithm in that patient, and then there was agreement that a single placebo-controlled study in PH-ILD, which we'll do globally, will suffice for both PH-ILD and PAH approval. And the beauty of doing it in PH-ILD, there'll be no background therapy, so we just have to beat placebo, which should be a pretty low bar. And finally, very well capitalized. We've been very active in the past month. We've raised $126 million since reporting Q3 earnings. We closed the third quarter with $76.2 million in cash. We added $26 million in an equity raise, including the $11 million from insiders, some on our board; added $20 million from a HealthCare Royalty revenue interest financing agreement, just topped up on that; and then importantly for us, we raised $75 million via PIPE with a fund related to Patient Square Capital here in San Francisco. So we feel, and this is a critical aspect here, that if we're able to launch in both PAH and PH-ILD in April that this funding will bridge us to profitability. So we're ready to launch, and we look forward to questions as we try to rewrite the future of these patients with pulmonary hypertension. Thank you.

Great. Thank you, Roger. I think I might start off with one question, and then we'll hand it over to the audience. Could you expand a little bit more on the process to getting final approval and launch in PAH and then also in PH-ILD?

Yes. So I'll turn it over to Rusty because that's mostly in his -- the process of that is in his hands.

So as Roger pointed out earlier, we have 2 ongoing Hatch-Waxman lawsuits, and I'll touch on each of them. And the first one, which is really the one that's gating our ability to get to market, that was the original Hatch-Waxman lawsuit that they filed. They asserted 3 different patents against us. With the December Federal Circuit decision that we received, we now have Federal Circuit affirm decisions that we don't infringe any valid claims of any of those patents. So substantively, we feel like that first Hatch-Waxman lawsuit at this point is resolved. It's now process. So the process is we need to, one, go to the FDA and seek final approval; and two, we think we're going to have to get Judge Andrews to rescind and set aside the injunction that he issued at the end of the trial. Again, substantively, we don't think there's an issue. In fact, we had asked him for a stay of the injunction before the Federal Circuit decision, about a week before that decision came down. He denied the stay, but he denied it on grounds that he wasn't going to speculate as to what the Federal Circuit was going to do. At this point, there's no question what the Federal Circuit is going to do. They did it. He also said as part of that opinion that it was his view that if we did get an affirmance of the Federal Circuit, that we'd be able to launch our product. So we think he's already signaled what he thinks the answer is. Again, we just have to go through the process. So as Roger said, on December 26, we filed our opening brief asking him to set aside the injunction. Their response brief is due a week from yesterday, so next Tuesday. We'll get a reply the week after on January 23, and at that point, as Roger said, it's in the judge's hands. We anticipate we'll get a decision relatively quickly on that because we do think this is top of mind for them. The second lawsuit, we don't think is a gating issue for us on any of the indications. They again asserted the '793 patent, which with December's decision, we don't think is really an issue in that lawsuit at all anymore. In fact, we filed, as we announced on Monday, a motion to dismiss to dismiss all claims related to that patent. The other patent, the '327 patent, I think there are a couple of things that are important to point out about that. First, as Roger noted, there's no 30-month stay. A 30-month stay by statute, it has to be a patent that was submitted to the Orange Book before the original NDA was filed. There's no question the '327 patent was not submitted to the Orange Book before original NDA was filed. What that means is that United Therapeutics would have to affirmatively get the court to issue a preliminary injunction for them to be able to interfere at all with our ability to launch in PAH and PH-ILD. In order to do that, they've got to convince the judge that they're substantially likely to succeed on the merits. And as we've been saying, we pointed out this patent was coming last June. We've talked about it on our earnings calls. And as we said all along, we think there's substantial prior art before this patent, and we think there are going to be significant challenges to United Therapeutics upholding the validity of that patent. We started showing some of our work in what we filed on Monday, and we press released that, pointing out at least some of the prior art. But I'd note that's not all the prior art that's out there. This was a patent application they filed in 2020, meaning everything prior to 2020 is prior art. That includes the '793 patent, which covers almost the same thing. And the other thing to keep in mind is the '327 patent, what that's really covering is physicians treating PH-ILD patients with TYVASO in accordance with the TYVASO label. And doctors have been doing that for more than 10 years. Rajeev Saggar, our CMO, when he was treating patients, he was treating patients with TYVASO, PH-ILD patients with TYVASO. And so again, we don't think there's anything in that patent that's anything new, and certainly not new as of 2020. Again, we anticipate that they will seek a preliminary injunction, but we feel confident that we've got the prior art that we'll be ready for that assertion of a preliminary injunction.

And again, that's specific to PH-ILD, won't interfere with what we're doing at PAH in terms of launch.

Assuming no preliminary injunction...

Sorry, would you just mind, in the microphone?

Assuming no preliminary injunction, are you willing to launch at risk in 2024 in PH-ILD?

Yes, Rusty.

Yes. So what we said consistently is we'll make that decision at the time that we're preparing to launch. But if we look at and survey the patents that are out there at that time, and we don't think there are any valid patents that have been issued at that point in time, we're not going to hold back our launch for a patent that we think is invalid.

And then similarly, if there's some way you can't launch in PH-ILD, would you consider just launching in PH and then waiting until PH-ILD is resolved?

Yes. We're going to launch the product as soon as we're able. I mean our goal is to get this therapy to patients. We've been waiting a long time to do this. There's a demand curve that I think warrants that we get there and get there soon, and you can see potentially that the current existing therapies are problematic, that we can improve upon that. So as soon as we're able to launch, as I said, we're ready to launch. We have the sales team in force, sales aids, marketing materials, everything is ready to go as soon as we have freedom to operate.

I just had a question. What drove the timing of the ILD amendment? Why didn't you file earlier, I guess?

Some of it was just to see -- really nothing drove it. And again, what's really driving it is the back end market exclusivity expires in end of March. So we worked from that point backwards, and we wanted to have a decision in hand through, again, what's going to happen on the 24th, if the agency agrees, is they'll give us another tentative approval and add PH-ILD to the tentative approval indication. So it's really just trying to get ahead of that time line, nothing specific about trying to do anything clever.

All right. So it was just coincidence that it was close to the patent...

Like I said, we worked backwards.