Liquidia - Q4 2022

Transcript

Operator (participant)

Good morning, and welcome everyone to the Liquidia Corporation's full year 2022 financial results and corporate update conference call. My name is Chris and I'll 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 call over to Jason Adair, Senior Vice President, Corporate Development and Strategy. Sir, please go ahead.

Jason Adair (SVP, Corporate Development and Strategy)

Thank you, Chris. It's my pleasure to welcome everyone to Liquidia's full year 2022 financial results and corporate update conference call. Joining the call today are Chief Executive Officer Roger Jeffs, Chief Medical Officer Dr. Rajeev Saggar, Chief Financial Officer Michael Kaseta, 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.

The company will file its 10-K on Monday, March 20th. 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)

Thank you, Jason. Good morning, everyone, thank you for joining us. As I look back on 2022, my first year as CEO at Liquidia, I remain humbled to have joined an organization so poised for success. My initial attraction to join the company in an operational role was related to the potential of YUTREPIA to universally transform prostacyclin therapy from a high-burden treatment option to a low-burden option for patients. Specifically, four key attributes resonated loudly with me. YUTREPIA's tolerability, YUTREPIA's titratability, YUTREPIA's durability, and YUTREPIA's usability and portability. These four attributes continue to resonate with me and support my belief that YUTREPIA has the potential to be the prostacyclin therapy of first choice and best in class inhaled therapy for patients with either PH or PH-ILD.

The open-label extension data that continues to mature only further my belief in the commercial potential for YUTREPIA to participate significantly in what is now the fastest growing segment of the PH agent prostacyclin market. The other main excitement for joining the event strength of the entire organization and our internal capability to manufacture drug substance in-house. It also didn't hurt that we already had tentative approval and labeling in hand. In 2022, we made key strategic hires to strengthen our core capabilities. In concert with our legal success in 2022, Liquidia is now well positioned to maximize the commercial uptake of YUTREPIA at launch.

I'd like to now turn the call over to Rajeev Saggar, our CMO, and one of those key 2022 hires, to expand on why we are so excited about YUTREPIA's unique product profile and why we believe we are ideally positioned to provide a differentiated best-in-class option for patients. Rajeev.

Rajeev Saggar (Chief Medical Officer)

Thank you, Roger, and good morning, everyone. In recent months, our team has engaged with the medical community about certain topics related to YUTREPIA, such as its product profile, the benefits of low resistance dry powder inhaler device for patients, and our upcoming clinical plans. Today, I thought it would be helpful to briefly touch on these points. YUTREPIA was designed with a specific goal in mind: to deliver treprostinil to the deepest parts of the lungs across a wide range of doses and a broad range of patients with varying lung function. To achieve this objective, we combined the PRINT technology with the RS00 Plastiape dry powder inhaler. A simple, proven device used successfully by many tens of thousands of adults and children with breathing-related problems such as COPD and asthma.

To the first point of expanding the dose range, our clinical studies in pulmonary arterial hypertension or PAH prove that a 79.5 microgram dose of YUTREPIA is delivered at bioequivalent doses to nine breaths of nebulized Tyvaso. More importantly, YUTREPIA has been safely and conveniently titrated to doses comparable to 27 breaths of Tyvaso, a level rarely, if ever, achieved with a nebulizer. To the second point of very our low DPI proved easy to use without need for advanced technology to train patients, and further demonstrated it is robust enough to be used while coughing or positioning in a myriad of ways. In fact, given the device's proven track record with obstructive lung diseases, there's considerable interest among medical professionals to use YUTREPIA, particularly for patients with pulmonary hypertension associated with interstitial lung disease or PH-ILD, where lung restriction and impaired respiratory effort are common.

Put simply, uniform PRINT particles are in the ideal respirable range for deep lung delivery, providing consistent and delivery with a low device across a wide range of inspiratory efforts for PAH and PH-ILD patients. To further inform the use of YUTREPIA, we intend to clinical trial later this year that will generate data on how YUTREPIA may be best utilized in PH-ILD. We think an open-label study will greatly benefit our understanding of tolerability and the ability to titrate in this patient population. I'm excited as we move closer to the potential launch of YUTREPIA. I'd like now to turn the call over to Rusty for an update on the legal proceedings. Rusty?

Rusty Schundler (General Counsel and Corporate Secretary)

Thank you, Rajeev. As a reminder, the company has received rulings through proceedings in the court and in parallel inter partes review proceedings before the Patent Trial and Appeal Board, or PTAB, that all of the claims in the three patents asserted by United Therapeutics against the company are either invalid or not infringed by Liquidia. Over the last several months, we have seen further progress in our litigation to bring YUTREPIA to market. First, we are pleased with the PTAB's decision in February to reject United Therapeutics request for a rehearing of the '793 IPR. In its decision, the PTAB clarified the grounds upon which it found that all the claims in the '793 patent were unpatentable. United Therapeutics now has 63 days from the decision date of February 2 to file an appeal of the PTAB's decision.

Assuming UT files an appeal, which they have publicly stated they will, we would project that oral arguments could occur as early as late fourth quarter of 2023 or first quarter of 2024. We would then anticipate that a decision could be rendered by the court as early as a few days after oral argument if the court issues a summary affirmance, or within a few months after oral argument if a full written opinion is issued. Second, we are pleased with the progress in the appeal of the district court's decision in the Hatch-Waxman trial. Briefing in that appeal has now been completed, and the court is in the process of scheduling oral argument, which we expect to occur sometime in the second or third quarter of 2023.

As with the appeal of the '793 IPR, we would expect to receive a written decision of the court within a few months after oral argument. All of these appeals, we will not summarize our arguments here, but all briefings are of course available to the public through the court's PACER system. There may be opportunities to accelerate the timeline in one or both appeal proceedings, and we will seek opportunities to proceed through the appeals process as quickly as possible. It is notable that United Therapeutics did not appeal the Hatch-Waxman decision related to the '901 patent, that patent is no longer an impediment to our launch of YUTREPIA.

With the '901 patent having been dropped, we would now be able to seek final approval for YUTREPIA if the decision of the district court in the Hatch-Waxman litigation is affirmed on appeal with respect to the '066 patent, and either the district court's decision regarding the '793 patent is reversed on appeal or the PTAB's decision regarding the '793 patent is affirmed on appeal. In short, if the original decisions are affirmed on appeal, then we can seek final approval of YUTREPIA immediately. I will now pass the call on to Mike for an overview of our financial reporting. Mike?

Michael Kaseta (CFO)

Thank you, Rusty. Good morning, everyone. Before I address the results for the full year 2022, I wanted to briefly comment on the security of our funds and relationship to SVB, a bank with whom we've had a relationship for about two years. As previously disclosed, we repaid all debt owed to SVB back in January as part of the financing agreement with HealthCare Royalty Partners. We also have maintained all cash and cash equivalents at SVB, 99% of which was held in a BlackRock mutual fund, and the remainder of which was held in an operating account. On Tuesday this week, substantially all of our cash was transferred out of SVB to an accredited financial institution. We will continue to evaluate our cash management and investment policies in an effort to protect our capital from events similar to what occurred in the last week.

Turning to our full year 2022 financial results, which can be found in the press release issued today, you will see that revenue increased to $15.9 million for the year ended December 31, 2022, compared with $12.9 million for the prior year. The profit split percentage we received under our promotion agreement with Sandoz was 50% for the entire year, whereas in 2021, the profit split percentage decreased from 80%-50% as a result of achievement of predetermined cumulative sales thresholds. Revenue in 2022 is net of $2.7 million in amortization of the contract acquisition costs associated with the pro-promotion agreement.

Next, cost of revenue was $2.9 million for the full year 2021, compared with $3 million for the prior year. 2022 included a full year of Salesforce related costs, as well as amortization of the intangible asset associated with the promotion agreement. Research and development expenses in 2022, of $19.4 million for the full year, compared with $20.5 million the year prior. The decrease of $1.1 million or 5% was primarily due to a $0.9 million decrease in personnel, consulting, and stock-based compensation expenses. General and administrative expenses were $32.4 million for the full year of 2022, compared to $23.1 million for the prior year.

The increase of $9.3 million, or 40%, was primarily due to a $4.2 million increase in commercial, marketing, and personnel expenses in preparation for the potential commercialization of YUTREPIA and a $3.1 million increase in stock-based compensation expense driven by an option modification charge recorded in the first quarter of 2022. In summary, we have incurred a net loss of $41 million or $0.67 per basic and diluted share, compared to a net loss of $34.6 million or $0.70 per basic and diluted share for the year ended December 31st, 2021. Turning to our balance sheet, we ended 2022 with $93.3 million of cash on hand.

We further strengthened our access to capital in January through the revenue interest financing agreement with HealthCare Royalty for up to $100 million in four tranches. The first tranche of $32.5 million netted an approximate $10 million increase in cash after paying off the SVB debt facility. The remaining tranches are related to, one, clearance of the legal pathway, two, acquisition of an internal asset, and three, mutual agreement of the parties. I would now like to turn the call back over to Roger.

Roger Jeffs (CEO)

Thank you, Mike. Reflecting on the previous year, I can say with a 100% confidence that we're prepared to deliver the potential of our product and future patients. At this time, I would now like to open the questions. Operator, first question.

Operator (participant)

Thank you. Before that, as a reminder to ask a question, please press star one one on your phone and wait for your name to be announced. To withdraw your question, please press star one one again. Stand by as we compile the Q&A roster. One moment please for our first question. Our first question will come from Greg Harrison of Bank of America. Your line is open.

Gregory Harrison (Biotechnology Analyst)

Hey, good morning. Thanks for taking the question. As you start to get closer to launch, what feedback are you receiving from physicians or patients, regarding the differentiation between YUTREPIA and Tyvaso DPI and the demand for YUTREPIA when you come to market?

Roger Jeffs (CEO)

Yeah. Hi, good morning. Wonderful question. We're excited to answer. At this time, I'd like to pass that to Rajeev, maybe you can give some thoughts on conversations you've had with various KOLs and then your own reflections on what differentiates YUTREPIA potentially from other DPIs in the space.

Rajeev Saggar (Chief Medical Officer)

Thanks, Greg. Thanks for the opportunity to answer this question. First thing is that I think it's very important to understand that YUTREPIA has been studied in pulmonary hypertension patients in the INSPIRE database. We've been able to titrate successfully YUTREPIA to doses equivalent up to 27 breaths 4x a day of Tyvaso, which is incredible. I think that showcases, one, our tolerability, and two, our titratability. Specifically, where the concern with pulmonary hypertension associated with interstitial lung disease is of importance is because this population has not only pulmonary hypertension as a vascular injury, but they also have an interstitial disease which causes a limitation, of course, but vital capacity. We believe this is where YUTREPIA has the ability to shine, in part because of its low resistance device that we're using.

We believe this will allow the limitations of the patient's lung function to be better tolerable and suitable for a device in particular. To that end, you know, we are hearing that our device, at least in those practitioners who have used it in our INSPIRE registry, feel like it has similarities to the simplicity of a nebulizer, but of course, has all the benefits of a dry powder inhaler, such as portability. To this point, we think, Greg, it's very important that we study some of these product profiles of YUTREPIA, specifically in PH-ILD, where we believe this will have the highest utility and impact in. That's where we alluded to that we will move forward with an open-label study to really showcase and highlight, 1, the tolerability of YUTREPIA in this patient population.

As you know, there has not been an official study using a dry powder inhaler in PH-ILD, so we believe this is important for the community and KOLs to experience. Second, to showcase our ability not only in the tolerability, but as such, because of that improved tolerability, we should see titratability to higher doses in this patient population. We look forward to initiating the study by the end of the year.

Roger Jeffs (CEO)

Thank you. Thank you very much, Rajeev. Great answer. It really sort of solidifies why we're so excited about the product profile of YUTREPIA and our ability to capture a significant share of the market. Operator, next question, please.

Operator (participant)

Thank you. One moment, please, for our next question. Our next question will come from Kambiz Yazdi of Jefferies. Your line is open.

Kambiz Yazdi (Biotechnology Equity Research VP)

Morning, team. Can you provide any granularity on kind of the gating factors to potentially starting a PH-ILD study? In terms of what feedback have you received on DPIs causing coughing in PH-ILD? Thank you so much.

Roger Jeffs (CEO)

Good morning, Kambiz. Great to hear from you as well. In terms of gating factors to starting studies, really that's just our ability to get the protocol approved at ethics committees, CROs engaged, and really just CTM for the study supply. That's fairly simplistic. We're working on that now, and it's our intention to start those studies in the coming quarters. In terms of PHILD, I'll let Rajeev speak to that.

Rajeev Saggar (Chief Medical Officer)

Thanks, Kambiz. you know, I think, you know, what we're hearing is several things. One, I think patients without a doubt like the convenience of a dry powder inhaler. That's a fact. Two, you know, these patients that have pulmonary hypertension associated with interstitial lung disease

Roger Jeffs (CEO)

patients with.

Rajeev Saggar (Chief Medical Officer)

Operator.

Jason Adair (SVP, Corporate Development and Strategy)

Yeah. Hey, hey, Chris. This is Jason Adair. Rajeev, you cut out there, as you started to explain the PHILD. I'm not sure if you could repeat?

Rajeev Saggar (Chief Medical Officer)

Can you hear me now?

Jason Adair (SVP, Corporate Development and Strategy)

Yeah.

Rajeev Saggar (Chief Medical Officer)

Yeah. In particular, with PH-ILD, because of their inherent limitations with cough in general, we understand that there's a certain number of patients in this population that tend to have when exposed to a high resistance inhaler. You know, if this is done improperly, you know, that potentially can cause the patient to have additional cough and limitations when they're exposed to certain types of dry powder inhalers.

Roger Jeffs (CEO)

Okay. Thank you, Rajiv. I think the importance now of our own study approach in patients with PHILD, it could be possible that the not only the tolerability, but the rate of titration in that would be a little bit different from our oral hypertension. But we think given the profile of our product, we're well positioned to test that show the best. Operator, next question please.

Rajeev Saggar (Chief Medical Officer)

Thank you. One moment please for our next question. Our next question will come from Serge Belanger of Needham. Your line is open.

Serge Belanger (Managing Director and Senior Analyst)

Hi, good morning. It's just one question. I think Roger, you've highlighted over the last few quarters how YUTREPIA inhaler is a low resistance DPI inhaler and highlighted some of the benefits there. Maybe just talk about what are the benefits and how that inhaler differentiates the product from a Tyvaso DPI. Thank you.

Roger Jeffs (CEO)

Yeah. Yeah, that's a great question. I'll team up again with Rajeev to answer this question. I think a lot of our answer is gonna be predicated on the fact that it's formulation driven. It's actually the PRINT formulation of the YUTREPIA product that allows the use of low resistance device. Rajeev, maybe you can speak to kind of the, how the formulation leads to the use of device and what combination that benefit uniquely ports.

Rajeev Saggar (Chief Medical Officer)

Yes. Thanks for that question. You know, I think to highlight what Roger just noted. You know, the innovative technology allows for these drug particles that require almost no deagglomeration. What that means is that YUTREPIA does not have to overcome such barriers. The PRINT powder is already disaggregated to its own. The particles have already been sized to deposit deep in the lung, and a low resistance inhaler device is obviously the most suitable perfection for our PRINT technology. By already optimizing size and shape of these drug particles, the PRINT formulation essentially enables a more ideal dry powder experience across a broad range of inspiratory flow rates. This is why, you know, effectively, this principle set is probably one of the key reasons that there's growing excitement to bring YUTREPIA into the market, particularly into PHI.

Roger Jeffs (CEO)

Thank you, Rajeev. I think, again, it's the formulation that is really having the ability to use. It'll be differentiated to delivery platform. Certainly without the need to deagglomerate, which requires the higher resistance device, it's going to take more capacity to do that. We think that then can meet certain limitations there, a leading entry into the market. Operator, next question please.

Operator (participant)

Thank you. One moment for the next question. Our next question will come from Julian Harrison of BTIG. Your line is open.

Julian Harrison (Managing Director and Biotechnology Analyst)

Hi, good morning. Thank Thank you for taking my question. I'm wondering if sotatercept has any bearing on the DPI treprostinil market opportunity in your mind.

Roger Jeffs (CEO)

Yeah. Great question, Julian. Again, once more I'll tag team with Rajeev. I think first let's just say it's an exciting time in pulmonary hypertension research. New mechanisms, new modalities, new fits, particularly onto triple combination therapy is impressive. I think there's growing excitement, including our own around how we interplay with these new modalities. Having said that, I've been in this field for 30+ years, and every time approach to treatment has come in, there's been a presumption that it should replace previous methodologies or treatments. It's never, ever been borne out through these, including this part says here, this might become consequential and added to therapy. I see this study being, you know, the sotatercept was seen as add-on to other therapies. The best benefit is working in prostacyclins.

In those patients, the prostacyclin dose was held steady because they were testing a, you know, the test patient. I think it would be exciting now to see what happens with YUTREPIA and sotatercept in combination, where YUTREPIA could also be titrated with the patients specifically. Again, a lot of excitement. I feel like it really changed our opinion of the opportunities we see with YUTREPIA. We still think YUTREPIA future will be the first choice that, the option, people can capture significant share of a market which has plenty of opportunity for really another drug, but obviously other inhaled prostacyclins as well. That's what excited about process licenses. In my belief, it will remain today. They're the only drug that can be titrated to effect. Given that there's like recipe of it will be terrible.

Rajeev, do you have anything to add to that?

Rajeev Saggar (Chief Medical Officer)

No. I believe Roger said it excellently. I'll just add. Remember, one of the key issues here is also focused on not only pulmonary hypertension, but also specifically pulmonary hypertension associated with interstitial lung disease, where sotatercept has yet not just shown a benefit. The entirety of both populations will be important, and that's going to have to be driven to show our effectiveness in both stages. Thank you.

Roger Jeffs (CEO)

Great, Edward. Thank you. Operator, next question.

Operator (participant)

Thank you. One moment please for our next question. Our next question will come from Matt Kaplan of Ladenburg Thalmann. Your line is open.

Matthew Kaplan (Managing Director and Head of Healthcare Equity Research)

Thank you. Good morning. Just a couple of questions on following up on the PH-ILD opportunity. I guess, one, could you tell us a little bit about the approval pathway for PH-ILD? Secondly, with your planned open label study, what are some of the endpoints you're going to look at there? Is there opportunity to showcase perhaps potential improvement in efficacy due to the titratability?

Roger Jeffs (CEO)

Thanks, Matt. It's good to hear. I'll talk about pathways. As we've said before, we've confirmed with the FDA in writing that no additional studies are required for approval for PH-ILD patients. Having said that, obviously we can't seek approval until the market exclusivity expires in just four. Opportunity for that market post that date. That's the pathway. Some of kind of how we apply that, approval will depend on the resolution of the legal case and when we get approval for pulmonary arterial hypertension and move our tentative approval to full approval. In general, those are the parameters that are gating for PH-ILD. Maybe, Rajiv, if you could speak a little bit to the points of what we hope there.

Rajeev Saggar (Chief Medical Officer)

Yeah. Thanks for the question. First of all, as we just discussed on this call, this will be an open study specifically in PH-ILD. In that regard, one of the main focuses is to really understand the tolerability of YUTREPIA patient population. Remember, this is a broad range of patients with different types, heterogeneous phenotypes, lung disease. Really highlighting the experience in that broad range population and see if there's any particular population that stands out with the best response. The second thing is that we're going to be focusing titratability. We know that if patients achieve certain breath equivalent to titrate to have a better response. We want to see if, number one, we can absolutely reach those levels and more importantly, exceed those levels in a tolerable fashion.

This will translate into several quantitative that we can acquire, including typical data points such as improved walk capacity or 6-minute walk distance. We can also look at various effects not only on the right ventricle itself, using non-invasive parameters to but also changes in scoring of the actual fibrosis itself, again, using non-invasive imaging methods. More to come as the final protocol gets up.

Roger Jeffs (CEO)

Thank you, Rajeev.

Matthew Kaplan (Managing Director and Head of Healthcare Equity Research)

Thank you. That's very helpful. just one-.

Operator (participant)

I see there are no further questions. Sorry. I see there are no further questions in the queue. I would now like to turn the conference back to Roger Jeffs for closing remarks.

Roger Jeffs (CEO)

If we can let Matt back in.

Jason Adair (SVP, Corporate Development and Strategy)

Chris, Matt had one more question. Could you let him back in so he can ask it?

Operator (participant)

No problem. One moment, please.

Matthew Kaplan (Managing Director and Head of Healthcare Equity Research)

Can you hear me?

Operator (participant)

Yes. Mr. Kaplan, you are now able to ask your next question.

Matthew Kaplan (Managing Director and Head of Healthcare Equity Research)

Great. Just a quick question. In terms of with the moving parts associated with the Hatch-Waxman litigation and the PPA group, decisions, what's your current thoughts on the timing for full approval now?

Roger Jeffs (CEO)

Yeah, great question. Maybe I'll ask Rusty to opine on sort of how he sees the timeline for the legal situation playing out.

Rusty Schundler (General Counsel and Corporate Secretary)

Sure. Thanks for the question, Matt. You know, we really have two separate, you know, opportunities to get clear of the patents at this point, the appeal of the Hatch-Waxman decision and the appeal of the 793 IPR. Both those are on different timelines. First is the appeal of the Hatch-Waxman decision. As I noted earlier, briefing's now complete, and we're just waiting on the court to set in oral argument dates. We think that date will be sometime in the second quarter or third quarter of this year. Once oral argument's been held, we expect the decision could come as quickly as a few days after oral argument or it could take a couple of months, depending on whether we get summary affirmance or not.

If in that decision, the '066, the district court's decision regarding the '066 patent is upheld, and if the '793 decision of the district court is overturned, we would be able to proceed immediately to seek full approval after that. That would put it sometime in the, you know, second, third, and fourth quarter this year. Alternatively, if that appeal results in the '066 patent decision being upheld, and the '793 decision of the Hatch-Waxman, the district court being upheld, then we would have to wait for the IPR appeal to play out. As we noted, we expect that oral argument in that will likely be held sometime fourth quarter this year or first quarter next year, you know, or first half of next year.

Again, same thing, after that oral argument is, you know, we could get a decision as quickly as a few days afterwards or as long as a few months afterwards.

Roger Jeffs (CEO)

Thank you, Rusty. Very clear. Operator, next question if there are any.

Operator (participant)

Thank you. One moment please. I see no further questions at this time in the queue. I will turn the call back to Roger Jeffs for closing remarks.

Roger Jeffs (CEO)

Great. Firstly, let me say we appreciate everybody calling in and listening. I hope you share the excitement we have around our building momentum. We're really trying to build on the back of some very positive news with, as it related to the legal situation. As we continue to go to the market, you can hear our excitement around the product capabilities of YUTREPIA and how it could be introduced uniquely into the marketplace and benefit patients in a differentiated way. We thank you again for joining us, and we look forward to reporting on our continued progress in the coming quarters. Thank you, everyone. Bye-bye.

Operator (participant)

This concludes today's conference call. Thank you all for participating. You may now disconnect and have a pleasant day.