Liquidia Corporation

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.

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 Cassetta, Chief Medical Officer, Dr. Rajiv Sagar, Chief Commercial Officer, Scott Mumma, and General Counsel, Rusty Shumla. Before we begin, please note that today's conference call will contain forward-looking statements, including those statements regarding future results unaudited and forward-looking financial information, as well as the company's future performance and or achievements. These statements are subject to known and unknown risks and uncertainties, which may cause our actual results or performance to be materially different from any future results or performance expressed or implied on this call. For additional information, including a detailed discussion of our risk factors, please refer to the company's documents filed with the Securities and Exchange Commission, which can be accessed on our website. I would now like to turn the call over to Roger for our prepared remarks, after which he will open the call for your questions. Roger?

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 Utrepia to include both the treatment of patients with pulmonary arterial hypertension, or PAH, and pulmonary hypertension associated with interstitial lung disease, or PHILD. 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 eutropia in both PH and PHILD as soon as possible after expiry of the gating clinical exclusivity on May 23rd, or just 65 days from now. Secondly, to continue to advance the clinical profile of Eutrepia 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 multibillion-dollar and growing market segment. And finally, to further evolve our mission of being a leader in the pH 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. With regard to the first imperative of seeking to launch Utrepia 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 eTrepia after May 23rd, 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 re-approve due to existing legal or regulatory barriers. In our case, the sole barrier identified by the FDA was the new clinical exclusivity granted to Tyvesa DBI, which expires on May 23, 2025. As directed in the tentative approval label, we plan to submit requests for final approval in the coming weeks. With regard to the second imperative of continuing to advance our studies to establish the clinical profile of eutropia, the medical community's interest in eutropia is increasing, with particular interest in the data being generated from our ASCENT trials. the open-label safety study of your TREVIA and PHIP patients. I'm happy to report, excuse me. Sorry, I have a call this morning. I'm happy to report that we're 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 eutropia. As a reminder, at the JP Morgan Healthcare Conference in January, we showed the dosing and tolerability profile for the first 20 patients that completed eight weeks of treatment. We noted that the PHLV patients on eutropia were able to titrate to doses that are three times the comparable therapeutic target of nebulized tovesa. And 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-death test at week eight. We are pleased to report in this same 20-patient cohort, the mean change in 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 three registrational study of nebulized toveso in PHIV patients, the INCRE study, active group patients had an observed mean improvement of six-minute walk distance of 15 meters at week 8 and 22 meters at week 16. We are highly encouraged by this early efficacy data from ASCEND as it demonstrates that eutrophia supports the key therapeutic attributes in PHLD we aspired for it, 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. And it should be noted that this data is especially compelling when compared to published data from Tyveso DPI and a like sample of treatment-like EHILD patients at the National Jewish Health Center that we have discussed previously, where 59% 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 Tyveso DPI. We looked We look forward to highlighting a more robust data set from the ASCENT 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 troposomal injection while also reinforcing relationships with our healthcare providers and our understanding of the unmet needs of PH and PHLD patients throughout the country. We are prepared to provide a seamless service to patients and providers upon launch of eutropia and look forward to educating the PAH and PHLD communities on the favorable and potentially game-changing product attributes of eutropia. 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 troposinol, 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 L6S6 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 Utopia in the coming months, that we decided to further strengthen our balance sheet, as announced yesterday, with an extension of our value partnership with health care royalty partners that Mike will speak about now, along with an overview of our 2024 financials.