Liquidia Q4 2024 Earnings Call Transcript

Quartr
Provided by Quartr
March 19, 2025

Key Takeaways

  • After FDA granted a tentative label for EUTREPIA covering PAH and PH–ILD, Liquidia plans to request final approval within weeks of May 23, 2025—the expiry of competitor exclusivity.
  • Enrollment in the ASCENT trial is on track for 50+ patients, with initial data showing a 26.4 m mean improvement in six-minute walk distance at eight weeks—exceeding published results for nebulized Tyvaso DPI.
  • Liquidia’s 14-month-established commercial team is fully trained to launch EUTREPIA, focusing on increasing PH–ILD diagnosis and capturing market share in both specialized centers and community settings.
  • The company is advancing L-606, a twice-daily sustained-release liposomal treprostinil, designed for consistent 24-hour exposure and set to initiate Phase III by year-end.
  • A new up to $100 million financing arrangement with Healthcare Royalty Partners strengthens Liquidia’s balance sheet to support the EUTREPIA launch and aims to drive profitability post-launch.
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Earnings Conference Call
Liquidia Q4 2024
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Operator

Good morning, and welcome everyone to the Liquidia Corporation Full Year 2024 Financial Results and Corporate Update Conference Call. My name is Shannon, 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 your questions. I would like to remind everyone that this conference call is being recorded. I will now hand the call over to Jason Adair, Chief Business Officer.

Jason Adair
Jason Adair
Chief Business Officer at Liquidia

Thank you, Shannon. It's my pleasure to welcome everyone to the Liquidia Corporation Full Year 2024 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 Medical Officer Dr. Rajeev Saggar, Chief Commercial Officer Scott Moomaw, 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.

Jason Adair
Jason Adair
Chief Business Officer at Liquidia

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 for your questions. Roger.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Thank you, Jason. Good morning, everyone, and thank you for joining us today. We believe that 2025 has the potential to be a transformational year for the company as we further build upon the success of 2024, which resulted in the broadening of the tentatively approved label for YUTREPIA to include both the treatment of patients with pulmonary arterial hypertension, or PAH, and pulmonary hypertension associated with interstitial lung disease, or PH-ILD. There are four strategic imperatives that will drive our success and growth both in the near and long term. These are: seeking to obtain final approval and launching YUTREPIA in both PAH and PH-ILD as soon as possible after expiry of the gating clinical exclusivity on May 23rd, or just 65 days from now.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Secondly, to continue to advance the clinical profile of YUTREPIA to position it as not only the best in class inhaled prostacyclin but also the first in choice prostacyclin for all patients in need of an oral or inhaled prostacyclin who remain underserved by current alternatives. Third, building upon our commercial and medical prowess to compete fiercely for capture of material and enduring market share in this multi-billion dollar and growing market segment. Finally, to further evolve our mission of being a leader in the PAH space by continuing to leverage our development expertise to advance treatment options that have the potential to further improve the lives of patients like our phase III ready NextGen sustained-release liposomal therapy L606. I'd like to take a few minutes to expand on each of these strategic goals.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

With regard to the first imperative of seeking to launch YUTREPIA into the marketplace as soon as possible, I'm happy to say that based on the favorable legal decisions over the last few years, there are no legal barriers that currently impact the FDA's ability to issue final approval of YUTREPIA after May 23 when the exclusivity granted to our competitor will expire. It's also important to remember that when the FDA issues a tentative approval of an NDA, it means the NDA has met all requirements for approval but cannot approve due to existing legal or regulatory barriers. In our case, the sole barrier identified by the FDA was the new clinical investigation exclusivity granted to Tyvaso DPI, which expires on May 23, 2025. As directed in the tentative approval label, we plan to submit the request for final approval in the coming weeks.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

With regard to the second imperative of continuing to advance our studies to establish the clinical profile of YUTREPIA, the medical community's interest in YUTREPIA is increasing, with particular interest in the data being generated from our ASCEND trial, the open-label safety study of YUTREPIA in PH-ILD patients. I'm happy to report, sorry, having a cough this morning. I'm happy to report that we are on track to complete enrollment in the coming weeks, with 50 or more patients in total to be enrolled. We continue to be encouraged by the positive patient response to escalating doses of YUTREPIA. As a reminder, at the JPMorgan Healthcare Conference in January, we shared the dosing and tolerability profile for the first 20 patients that completed eight weeks of treatment.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

We noted that the PH-ILD patients on YUTREPIA were able to titrate to doses that are three times the comparable therapeutic target of nebulized Tyvaso. Today, we're happy to share some new information from the ASCEND study. We've also been looking at exploratory measures of efficacy. One such measure is the change in six-minute walk test at week eight. We are pleased to report in this same 20-patient cohort, the mean change from baseline improved by 26.4 meters. While it is difficult to draw strong conclusions from cross-study comparisons, it is worth noting that in the phase III registrational study of nebulized Tyvaso in PH-ILD patients, the INCREASE study, active group patients had an observed mean improvement of six-minute walk distance of 15 meters at week eight and 22 meters at week 16.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

We are highly encouraged by this early efficacy data from ASCEND, as it demonstrates that YUTREPIA supports the key therapeutic attributes in PH-ILD we aspired for, specifically being well tolerated and amenable to rapid dose escalation to doses well beyond historical standards, leading to an accelerated therapeutic outcome that is in line or better than published results of existing therapies. It should be noted that this data is especially compelling when compared to published data from Tyvaso DPI in a like sample of treatment, i.e., PH-ILD patients at the National Jewish Health Center that we have discussed previously, where 69% of patients discontinued therapy after a median time of only 40 days, with drug-related AEs, especially cough and clinical worsening, listed as the primary reasons for discontinuation of Tyvaso DPI.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

We look forward to highlighting a more robust data set from the ASCEND trial at the ATS International Conference in San Francisco this May. With regard to our third strategic imperative to build upon our commercial and medical prowess, we have built a commercial enterprise that we feel is best in class. Our team has been in place for over a year and a half and continues to support the use of treprostinil injection while also reinforcing relationships with our healthcare providers and our understanding of the unmet needs of PAH and PH-ILD patients throughout the country. We are prepared to provide a seamless service to patients and providers upon launch of YUTREPIA and look forward to educating the PAH and PH-ILD communities on the favorable and potentially game-changing product attributes of YUTREPIA.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Lastly, as we look at our fourth imperative to innovate and develop better therapeutic options, we continue to advance L606, our sustained-release liposomal formulation of treprostinil that provides more consistent 24-hour drug exposure in two 12-hour dosing segments, including during sleeping hours. We know that continuous infusion has shown the best efficacy with prostacyclin analogs, and we believe that the PK profile of L606 may provide the next closest non-invasive approach to maximizing the benefit from the more targeted inhaled route of administration. It is for all these reasons, and with an eye towards our pending launch of YUTREPIA in the coming months, that we decided to further strengthen our balance sheet, as announced yesterday, with an extension of our valued partnership with HealthCare Royalty Partners that Mike will speak about now, along with an overview of our 2024 financials. Mike.

Michael Kaseta
Michael Kaseta
COO and CFO at Liquidia

Thank you, Roger, and good morning, everyone. Before we review our full year 2024 financial results, I'm happy to highlight yesterday's announcement regarding expansion of our financing agreement with HealthCare Royalty Partners, which will provide Liquidia up to an additional $100 million of financing in three tranches, including the $25 million tranche funded at closing. We are grateful for the trust, commitment, and confidence that HealthCare Royalty Partners have demonstrated over the years, and we are optimistic that these proceeds and a successful launch of YUTREPIA following the expiration of exclusivity this May could lead to our reaching profitability without the need for additional capital. Turning to our full year 2024 financial results, which can be found in the press release, you will see that revenue was $14 million for the year ended December 31, 2024, compared with $17.5 million for the year ended December 31, 2023.

Michael Kaseta
Michael Kaseta
COO and CFO at Liquidia

Revenue related primarily to the promotion agreement. The decrease of $3.5 million was primarily due to lower sales quantities driven by limitations on the availability of pumps used to administer treprostinil injection subcutaneously. Sales quantities will continue to be impacted or at risk until alternative pumps are available. Cost of revenue was $5.9 million for the year ended December 31, 2024, compared with $2.9 million for the year ended December 31, 2023. Cost of revenue related to the promotion agreement, as noted above. The increase from the prior year was primarily due to our sales force expansion during the fourth quarter of 2023. Research and development expenses were $47.8 million for the year ended December 31, 2024, compared with $43.2 million for the year ended December 31, 2023.

Michael Kaseta
Michael Kaseta
COO and CFO at Liquidia

The increase of $4.6 million, or 11%, was primarily due to, one, a $6.1 million increase in expenses related to our L606 program; two, a $5.3 million increase in expenses related to YUTREPIA research and development activities, including the ASCEND trial; three, a $5.1 million increase in personnel expenses, including stock-based compensation related to increased headcount; and four, a $3.5 million upfront license fee due to Formosa for the exclusive license in Europe to develop and commercialize L606 recorded during the year ended December 31, 2024, offset by, one, $5.1 million in lower commercial manufacturing expenses reflecting the impact of expensing YUTREPIA inventory costs in the prior year; and two, a $10 million upfront license fee due to Formosa for the exclusive license in North America to develop and commercialize L606 during the year ended December 31, 2023.

Michael Kaseta
Michael Kaseta
COO and CFO at Liquidia

General administrative expenses were $81.6 million for the year ended December 31, 2024, compared with $44.7 million for the year ended December 31, 2023. The increase of $36.9 million, or 82%, was primarily due to, one, a $19.7 million increase in personnel expenses, including stock-based compensation driven by higher headcount and expansion of our sales force in the fourth quarter of 2023; two, a $7.9 million increase in legal fees related to our ongoing YUTREPIA-related litigation; and three, a $6.8 million increase in commercial expenses in preparation for the potential commercialization of YUTREPIA. In summary, we incurred a net loss for the 12 months ended December 31, 2024, of $130.4 million, or $1.66 per basic and diluted share, compared to a net loss of $78.5 million, or $1.21 per basic and diluted share for the 12 months ended December 31, 2023.

Michael Kaseta
Michael Kaseta
COO and CFO at Liquidia

With that, I would now like to turn the call back over to Roger.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Thank you, Mike. As you can see, we're well positioned clinically, commercially, and financially for the potential launch of our first product based on our proprietary PRINT technology, which will serve as the growth engine for our continued evolution and inflection. I would now like to open the call for questions. Operator, first question, please.

Operator

Thank you. As a reminder to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. Our first question comes from the line of Julian Harrison with BTIG. Your line is now open.

Julian Harrison
Julian Harrison
Managing Director at BTIG

Hi, good morning. Thank you for taking my questions. Roger, you touched on this a little in your prepared remarks, but just wondering if you could talk more about the administrative sequence and timeline to convert YUTREPIA's tentative approvals in PAH and PH-ILD to full approvals.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Yes. As we said in the opening, thank you, Julian. Great to talk to you. As we said in the opening, we plan to request final approval in the coming weeks. In the tentative approval label that we received in August, it was recommended and suggested that we apply for final approval either two or six months ahead of the action date, which would be May 23. Given we view this as a class one resubmission because no new data will be required for approval, we have been in concert with the agency working to deliver that letter around the March 24 timeframe so that we can be granted final approval around May 23. I do not know if that specifically addresses what you are seeking.

Julian Harrison
Julian Harrison
Managing Director at BTIG

Yeah, absolutely. That's very helpful. Thank you. A follow-up, if I may, on L606. I'm curious what the physician feedback has been so far for a lower CMAX, broader AUC, longer half-life alternative to nebulized Tyvaso. Sorry if I missed it. I'm wondering also when specifically you plan to initiate the phase III Respire trial.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Yeah, great. We are fortunate to have Dr. Rajeev Saggar on the call today. Rajeev, if you would not mind.

Rajeev Saggar
Rajeev Saggar
Chief Medical Officer at Liquidia

Yeah, thanks, Julian, for the question. First of all, I think the current open-label study continues to gate very well. We have long-term safety data that has accrued since the inception of the study. What clear feedback is, is twice-a-day dosing is an absolute game changer for these patients when you inhale treprostinil. I think that's first and foremost. Second of all, I think that the use of the liposome itself has showcased what we believe highlights some impressive safety advantages. Finally, further reducing the CMAX with the twice-a-day dosing profile and the sustained-release formulation of the liposome itself, we have noted that the systemic side effects continue also to be further abated, even beyond what we initially desired. I think the combination of both of those are going to be highly encouraging as we advance forward with the Respire study.

Rajeev Saggar
Rajeev Saggar
Chief Medical Officer at Liquidia

In regards to initiating the Respire study, we're working full steam ahead to prepare to initiate the study by year's end. It's around March right now, and so we look forward to providing further information as we get closer to that timeframe near the end of the year.

Julian Harrison
Julian Harrison
Managing Director at BTIG

Okay, great. Thank you again, and congrats on all the progress.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Thank you, Julian.

Operator

Our next question comes from the line of Jason Gerberry with Bank of America. Your line is now open.

Jason Gerberry
Jason Gerberry
Managing Director and Equity Research Analyst at Bank of America

Hey, good morning. This is Bhavan Patel on for Jason. Two questions from us. The first is, given the green space in PH-ILD, what's Liquidia's strategy to drive early market penetration for YUTREPIA? Can you share the marketing differentiation that you plan to promote to doctors? Given the 505(b)(2) pathway, should we expect any differences in the label for YUTREPIA versus Tyvaso DPI? I know in the past you've mentioned demonstrating titration to three times higher doses compared to Tyvaso DPI.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Yeah. I'll answer the second question first, and then I'll ask Scott Moomaw, our Chief Commercial Officer, if he would address the launch dynamics around PH-ILD and differentiation that we intend to emphasize. You're exactly correct, Bhavan. I think the label, it is a 505(b)(2), so we share obviously the indication claims and things like that. Where we differ from the label is in the dose titration table because in our study that we used to bridge, we showed that we could escalate the doses, as you mentioned, up to three times higher than the target therapeutic dose of the brand. That will be in the label, and I think it's an important point of differentiation because it's print-enabled using our proprietary PRINT technology to drive the particles to the lower airway preferentially and avoid the toxicities associated with upper airway deposition.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

It's going to help us drive a maximal outcome for these patients as they continue to progress, and therefore help us keep those patients on therapy longer is our presumption. We think these are important and critical aspects. If you look across any route of administration or prostacyclin, dose matters, and the ability to drive dose usually predicts the sort of therapeutic utility of that therapy. That's why Tyvaso, as labeled, has had limitations in dosing, and it's also why other drugs like Uptravi, an oral formulation, which is dose-limited and dose-ceiling, also has limitations over the long and chronic treatment course of these patients. Lots of opportunity there from us from a labeling and dosing standpoint. Scott, if you could talk a little bit about kind of how we want to approach this market without giving away the shop too much.

Scott Moomaw
Scott Moomaw
CCO at Liquidia

Sure. Good morning. If you think about it, we have the usual designation between centers and communities, and we are approaching, and we will continue to approach both, especially after launch. The situation in the centers is that the patients are really flowing in. There is an increasing number of patients flowing in as the awareness of PH-ILD increases, and we will obviously only add fuel to that fire. We are in the centers, and we are going to make sure that we ensure that YUTREPIA is the first choice prostacyclin for those patients that have PH-ILD for a lot of the reasons that Roger just outlined, and you have heard us talk about before. We are going to harvest those patients immediately. Doing is in the community, and there are many, many patients out there that doctors do not even know exist. They have ILD. The doctors are not looking for PH.

Scott Moomaw
Scott Moomaw
CCO at Liquidia

We need to help increase awareness. We need to help increase diagnosis, and then either get that doctor to prescribe or to refer that patient into one of the centers. We have actually been doing that over the last six months or a year, going deeper and deeper into the community, educating on PH-ILD even before we get on the market because we know that is only going to behoove us once we do get on the market because we do believe we have the superior product.

Julian Harrison
Julian Harrison
Managing Director at BTIG

Great. Thank you, Scott, so much.

Operator

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

Serge Belanger
Serge Belanger
Managing Director of BioPharma Equity Research at Needham

Hi, good morning. A couple of commercially related YUTREPIA questions, probably for Scott. Just can you remind us the breakdown between commercial and Medicare coverage for both PAH and PH-ILD? Assuming we're going to get approval here in a couple of months, can you talk about what you expect in terms of the ramp-up in coverage across both commercial and Medicare, I guess, over the next 12 months or so? Thanks.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Yeah, great question. Serge, Scott.

Scott Moomaw
Scott Moomaw
CCO at Liquidia

Yeah, good morning, Serge. In terms of the breakdown, generally, think of it as Medicare is the biggest. 50% Medicare, probably 30-40% commercial, and then the remaining is Medicare, other DOD, etc. In terms of the coverage over the first year, as you probably know, we're not commenting on our strategies. I will say that we have been working with the payers closely for quite some time, and we feel like we have great relationships with us. They have the desire to make sure that we maximize access, so we share that desire. I think you'll see when we launch that we're very sound on that front.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Great. Thanks, Scott. Operator, next question.

Operator

Our next question comes from the line of Ryan Deschner with Raymond James. Your line is now open.

Ryan Deschner
Ryan Deschner
Director of Biotechnology at Raymond James

Hi, good morning. Remind us what key readouts we should be focusing on for the ASCEND study and what specifically we can expect in the data coming at ATS, and then I'm going to follow up.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Yeah. Thank you, Ryan. Rajeev, if you wouldn't mind answering that one.

Rajeev Saggar
Rajeev Saggar
Chief Medical Officer at Liquidia

Yeah, thanks, Ryan. As Roger talked about in the prepared remarks today, one of the highlights that we spoke about was the walk effect that was observed in the first 20 patients treated within eight weeks, which showed an observed mean improvement of around 26.4 meters. I think that first and foremost highlights a few key processes. First, that we can get patients to what we believe is even a new therapeutic goal that surpasses the traditional 9-12 breaths that have typically been sort of the ceiling effect of Tyvaso since 2009. We've highlighted that we're able to achieve doses in the overwhelming majority of patients by week eight that's equivalent to greater than or equal to 15 breaths by this time.

Rajeev Saggar
Rajeev Saggar
Chief Medical Officer at Liquidia

We can get patients up to what we believe is a very effective dose and even higher at an earlier time point, and then allow further titration to further doses to stabilize the patient and plateau them out. I think that's what's going to be quite encouraging. At ATS, we're going to showcase more regarding the data sets, regarding how we dose the patients. We're going to talk a little bit about our effects in terms of some quality of life questionnaires as well. In the future, our plan is to, as we've talked about, the ASCEND study will be fully enrolled within the next few weeks here. We're really excited about that. We do plan to prepare and submit for the final data set later in the year and present that at a major respiratory conference in the future.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Great. Thank you, Rajeev.

Ryan Deschner
Ryan Deschner
Director of Biotechnology at Raymond James

That's helpful. Thank you very much. Will you be doing additional hiring for the commercial field team after potential approval in both indications is confirmed? What is the status of the current field team with regards to both hiring and training? Thank you.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Yeah. Scott, if you wouldn't mind.

Scott Moomaw
Scott Moomaw
CCO at Liquidia

Yeah, sure. I'll start with that one. The status of the current sales team is they're locked and loaded and ready. We've had the team on board for 14 months now, and these were experienced, mostly PH, all rare disease folks to begin with. They've been out in the field strengthening relationships. They've been training, training, training. They are absolutely ready to go and very, very excited. On the future front, we actually just went through another exercise over the course of the last few months to take a look at our salesforce sizing strategy. We think we're well-suited to launch, as I mentioned earlier. We will take another look at that once we get out and start to understand sort of the sensitivity factor for the number of reps, and then we'll look at potentially expanding, if that makes sense, in the future.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Another thing I would add is we are adequately sized for launch in both indications. As Scott said, obviously, as we continue to build and present the unique benefits of YUTREPIA, I think we want to articulate that even broader so we could upsize our salesforce probably in the 2026 timeframe if necessary. Operator, is it another question?

Operator

Our next question comes from the line of Greg Harrison with Scotiabank. Your line is now open.

Greg Harrison
Greg Harrison
Director at Scotiabank

Hey, good morning, guys. Thanks for taking the questions. Wondering if you can comment on your updated cash runway following your recent financing and what assumptions go into that estimate that you could reach profitability with your current balance sheet. If you could also comment on which factors play into your decision on pricing, given your view of YUTREPIA's profile relative to the competitor. Thanks.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Great. I think both of those questions would be well answered by Mike.

Scott Moomaw
Scott Moomaw
CCO at Liquidia

Yeah, Greg, great to talk to you again. As it relates to runway, as we said, with this $100 million of additional financing from HealthCare Royalty Partners, $25 million we receive at closing, $50 million will be received upon the first commercial sale of YUTREPIA, and the final $25 million will be at mutual option once we reach a cumulative $100 million in net sales of YUTREPIA. I think it's pretty simple. We're very excited about the launch of YUTREPIA. As Roger said and Scott have said, we are fully ready from a commercial readiness point of view.

Scott Moomaw
Scott Moomaw
CCO at Liquidia

From a supply point of view, we feel very bullish about our ability to launch this successfully, and we feel that if, assuming, as Roger said, we file for full approval here coming up, we get full approval on or near the expiration of the exclusivity date, we feel confident if we hit our goals and hit our targets that we can be profitable on this current balance sheet and, like I said, fully support the L606 program, all of our phase four studies we're doing on YUTREPIA, while also the ongoing commercial readiness. We are very excited. We are very confident. We are very happy to have a great partner, like HealthCare Royalty Partners, on this journey, and we look forward to launch. As to the second question, we are not going to talk about pricing strategy.

Scott Moomaw
Scott Moomaw
CCO at Liquidia

I think what I would say is, as we've demonstrated or continue to demonstrate and what we feel our early results on ASCEND only reinforce that, that we feel that we have a superior product profile. Based on that, we want to make sure that we are always balancing patient access while also fulfilling the value proposition that YUTREPIA can bring. We're not going to talk specifically about pricing strategies in totality, but I think we're very confident as we come to market that we will have the right strategy to make sure that patients have choice as we move forward.

Greg Harrison
Greg Harrison
Director at Scotiabank

Great. That's helpful. Thanks so much.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Thanks for the question, Greg. Operator, next question.

Operator

Our next question comes from the line of Cory Jubinville with LifeSci Capital. Your line is now open.

Cory Jubinville
Cory Jubinville
Managing Director and Equity Research Analyst at LifeSci Capital

Good morning, and thanks for taking our questions. Congrats on this new efficacy data with ASCEND. It's really exciting. Just kind of following the theme of more drug is better, I recognize that the sample size here is still relatively small, but have you had any type of dose response among patients? Are those who are reaching two to three-fold the Tyvaso equivalent dose achieving better outcomes than those in the study who might be kind of in the lower range equivalent to the existing standard of care? This also might be a naive question, but is it possible to get these efficacy data on label, and how would you go about marketing this six-minute walk to prescribers post-approval?

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Yeah, I'll take the second question, and then Rajeev can say what he can about the dose versus effect curve. The data would not be available for indication claims per se because it's an open label safety study. I think it would be part of an annual update to the label once approved where we could update the clinical pharmacology section to describe the data both in terms of safety and potentially in terms of efficacy. That is a negotiation with the FDA about what we could then include in the clinical pharmacology section of the label. I'll reserve sort of commitment on that, and we'll do the best we can to get it in there. I think certainly we will abstract and then publish the data. I think as we're seeing, there's tremendous inbound interest based on what we're describing.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

There are over 20 doctors doing this current ASCEND study. Many of them are the seminal thought leaders. I think the fact that they are doing the study and we are seeing the results that we are seeing is going to help highlight the data when they lead panel sessions, etc., because now they are seeing the true value of print as it relates to YUTREPIA. I will just comment on dose response before I turn it over to Rajeev. It is very hard in a small sample of patients to show dose response, frankly. Plus, we are allowing patients to individually titrate to dose, so we are not sort of predefining what dose level patients should titrate to and then measuring that versus effect. It is a continuum. That is a difficult paradigm to show dose response in. Rajeev, I do not know if you have additional comment there.

Rajeev Saggar
Rajeev Saggar
Chief Medical Officer at Liquidia

Yeah, Cory, I think just I think a few things to highlight. First of all, the patients that we're enrolling in ASCEND are quite heterogeneous. We purposely designed the study to showcase safety and tolerability of YUTREPIA. We not only include patients with mild hemodynamic limitations with their pulmonary hypertension, but also patients with severe hemodynamic impairments with various levels of complexity with the interstitial lung disease and their respiratory physiological impairments. I think despite that heterogeneity, I think what the data is showing, despite these small numbers, even though, as you know, the study continues to enroll and continue to highlight a few things, number one, clearly the low-resistance inhaler combined with our print formulation is absolutely allowing these patients to titrate YUTREPIA to, again, to what I believe are actually new therapeutic levels.

Rajeev Saggar
Rajeev Saggar
Chief Medical Officer at Liquidia

That is very important because it is well known that prostaglandin therapies are titratable. It is dose-related, so effects tend to improve as dose is escalated. The problem with current therapies on the market, including oral and systemic parenteral therapies, is that they are just significantly limited by terrible systemic side effects, especially in the oral therapies with the GI side effects and, of course, the parenterals with indwelling lines or subcutaneous complications.

Rajeev Saggar
Rajeev Saggar
Chief Medical Officer at Liquidia

I think with the current inhaled therapies on the market today, we know that at least in the INCREASE study, what was very interesting relative to the ASCEND study is by eight weeks, only 50% of the patients in the Tyvaso nebulized study for INCREASE with PH-ILD were able to reach the 10-12 breath levels versus in the ASCEND study, despite the heterogeneity in the patient population, we're able to get the overwhelming majority of patients up to more than or equal to 15 breath equivalents. I think that what the ASCEND data is going to show at ATS and what KOLs are going to see is that YUTREPIA is very well tolerated and titratable, and that they can customize to whatever needs their patients have with both PAH and PH-ILD in the very near future.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Cory, that's a great answer, Rajeev. Thank you. I think one way we're going to take on this dose issue directly is we're going to do a directed transition study from patients on Tyvaso and Tyvaso DPI that are underserved by those therapies, probably because of dose limitations. We'll transition them to YUTREPIA, improve their dose, and if we show, therefore, an improved outcome, we've shown not only is dose related to outcome, but we've also differentiated the products even in a different way and a helpful way for YUTREPIA. Look for that study to start up in the near future as well. Operator, next question, please.

Operator

Thank you. I am currently showing no further questions at this time. I would like to hand the call back over to Roger Jeffs for closing remarks.

Roger Jeffs
Roger Jeffs
CEO at Liquidia

Thank you very much. We really appreciate everyone joining us today, and we look forward to speaking again soon. Bye-bye.

Operator

This concludes today's conference call. Thank you for your participation. You may now disconnect.

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    • Greg Harrison
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