Core Viewpoint - Liquidia Corporation has received acceptance from the FDA for its New Drug Application resubmission for YUTREPIA™ (treprostinil) inhalation powder, aimed at treating pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) [1][2] Group 1: FDA Approval Process - The FDA confirmed that the resubmission was a complete, Class 1 response to the previous action letter issued on August 16, 2024, which granted tentative approval for YUTREPIA for both PAH and PH-ILD [1] - The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of May 24, 2025, for the final decision on the NDA [1] Group 2: Company Overview - Liquidia Corporation is focused on developing innovative therapies for patients with rare cardiopulmonary diseases, particularly in the area of pulmonary hypertension [2] - The company utilizes its proprietary PRINT® Technology to create its lead candidate, YUTREPIA™ (treprostinil) inhalation powder, which is an investigational drug for PAH and PH-ILD [2] - Liquidia is also developing L606, a sustained-release formulation of treprostinil, and currently markets generic Treprostinil Injection for PAH treatment [2]

Core Insights - Liquidia Corporation is presenting new data on LIQ861 (YUTREPIA™) at the ATS 2025 International Conference, focusing on safety, tolerability, and exploratory efficacy in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) [1][2] Group 1: Product Information - YUTREPIA is an investigational inhaled dry-powder formulation of treprostinil, designed for easy use with a low-effort device [3] - The FDA issued tentative approval for YUTREPIA for PAH and PH-ILD indications in August 2024 [3] - YUTREPIA was developed using Liquidia's PRINT technology, which allows for precise and uniform drug particle creation for enhanced lung deposition [3] Group 2: Clinical Studies - The ASCENT study is an open-label, prospective multicenter trial evaluating the safety and tolerability of dry powder inhaled treprostinil in PH-ILD patients [3] - The INSPIRE study demonstrated the long-term safety and tolerability of LIQ861 in a PAH patient transitioning from parenteral treprostinil [1] - Three posters will be presented at the ATS conference, detailing initial safety and exploratory efficacy data, changes in cardiac effort, and a case study of transitioning from parenteral treprostinil to LIQ861 [2] Group 3: Company Overview - Liquidia Corporation focuses on developing innovative therapies for rare cardiopulmonary diseases, particularly in pulmonary hypertension [4] - The company is also developing L606, a sustained-release formulation of treprostinil, and markets generic Treprostinil Injection for PAH treatment [4]

Financial Data and Key Metrics Changes - Revenue for the year ended December 31, 2024, was $14 million, a decrease from $17.5 million for the year ended December 31, 2023, primarily due to lower sales quantities related to the promotion agreement [21] - Cost of revenue increased to $5.9 million in 2024 from $2.9 million in 2023, attributed to sales force expansion [23] - Research and development expenses rose to $47.8 million in 2024 from $43.2 million in 2023, an increase of 11% driven by various program costs and personnel expenses [24] - General and administrative expenses surged to $81.6 million in 2024 from $44.7 million in 2023, an increase of 82% primarily due to higher personnel costs and legal fees [25] - The net loss for 2024 was $130.4 million or $1.66 per share, compared to a net loss of $78.5 million or $1.21 per share in 2023 [25] Business Line Data and Key Metrics Changes - The decrease in revenue was mainly due to limitations on the availability of pumps used for administering treprostinil injections [22] - The company is advancing the clinical profile of YUTREPIA, with positive patient responses reported in the ASCENT trial [12][15] Market Data and Key Metrics Changes - The company is preparing for the launch of YUTREPIA in both PAH and PH-ILD markets, which are expected to be significant growth areas [8][9] - The company has established a commercial enterprise to support the use of treprostinil injection and is focused on capturing market share in a growing market segment [16] Company Strategy and Development Direction - The company aims to obtain final approval and launch YUTREPIA as soon as possible after May 23, 2025, when exclusivity for a competitor expires [10] - The strategic imperatives include advancing the clinical profile of YUTREPIA, building commercial capabilities, and innovating new therapeutic options like L606 [9][17] - The company plans to leverage its proprietary PRINT technology to differentiate YUTREPIA from competitors [40] Management's Comments on Operating Environment and Future Outlook - Management believes 2025 could be transformational, with expectations for a successful launch of YUTREPIA [8] - The company is optimistic about reaching profitability without additional capital, contingent on the successful launch of YUTREPIA [20][21] - Management expressed confidence in their commercial readiness and relationships with payers to maximize access to YUTREPIA [49][66] Other Important Information - The company announced an expansion of its financing agreement with HealthCare Royalty Partners, providing up to an additional $100 million in financing [20] - The ASCENT trial is on track to complete enrollment with positive early efficacy data reported [12][15] Q&A Session Summary Question: What is the timeline for converting YUTREPIA's tentative approvals to full approvals? - Management plans to request final approval in the coming weeks, aiming for approval around May 23, 2025 [29] Question: What feedback has been received regarding L606? - Physician feedback indicates that twice-a-day dosing is a significant advantage, and the liposomal formulation shows impressive safety benefits [34] Question: What is Liquidia's strategy for early market penetration for YUTREPIA? - The company plans to ensure YUTREPIA is the first choice for patients with PH-ILD and increase awareness and diagnosis in the community [45] Question: What is the breakdown between commercial and Medicare coverage? - The coverage breakdown is approximately 50% Medicare, 30-40% commercial, with the remainder from other sources [48] Question: What key readouts should be focused on for the ASCENT study? - Key readouts include the six-minute walk test results, with an observed mean improvement of 26.4% meters reported [53] Question: Will there be additional hiring for the commercial field team? - The current sales team is fully prepared, and future hiring will be assessed based on market response post-launch [59][61] Question: What is the updated cash runway following recent financing? - The company is confident in its ability to reach profitability with the current balance sheet and financing arrangements [66][68]

Liquidia Corporation (NASDAQ:LQDA) Q4 2024 Earnings Conference Call March 19, 2025 8:30 AM ET Company Participants Jason Adair - Chief Business Officer Roger Jeffs - CEO Michael Kaseta - COO and CFO Rajeev Saggar - Chief Medical Officer Scott Moomaw - Chief Commercial Officer Rusty Schundler - General Counsel Conference Call Participants Julian Harrison - BTIG Serge Belanger - Needham Ryan Deschner - Raymond James Greg Harrison - Scotiabank Cory Jubinville - LifeSci Capital Operator Good morning. And welcom ...

Group 1 - Liquidia Technologies, Inc. reported a quarterly loss of $0.46 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.38, and compared to a loss of $0.42 per share a year ago, indicating an earnings surprise of -21.05% [1] - The company posted revenues of $2.92 million for the quarter ended December 2024, missing the Zacks Consensus Estimate by 43.11%, and this represents a decline from year-ago revenues of $4.53 million [2] - Over the last four quarters, Liquidia Technologies has not surpassed consensus EPS estimates and has topped consensus revenue estimates only once [2] Group 2 - Liquidia Technologies shares have increased by approximately 25.3% since the beginning of the year, contrasting with a decline of -4.5% in the S&P 500 [3] - The current consensus EPS estimate for the upcoming quarter is -$0.34 on revenues of $4.14 million, and for the current fiscal year, it is -$1.14 on revenues of $47.04 million [7] - The Medical - Biomedical and Genetics industry, to which Liquidia Technologies belongs, is currently ranked in the top 30% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8]

Financial Performance - The total reported net revenue of branded therapies targeting the prostacyclin pathway for PAH and PH-ILD in the U.S. was $4.1 billion in 2024[37]. - United Therapeutics' treprostinil-based products generated U.S. net revenue of $2.5 billion in 2024, with the Tyvaso franchise contributing $1.6 billion[37]. - Branded Remodulin generated U.S. revenue of approximately $464 million in 2024, down from $415 million in 2023, indicating a year-over-year decline of about 10.6%[51]. - The Tyvaso franchise's annual sales increased from $400 million in 2019 to $1.6 billion in 2024, correlating with the expansion into the PH-ILD indication[37]. Product Development and Approval - YUTREPIA received tentative FDA approval for both PAH and PH-ILD on August 16, 2024, with final approval delayed until May 23, 2025, due to regulatory exclusivity for Tyvaso DPI[25][44]. - The NDA for YUTREPIA was submitted in January 2020, and the FDA conducted pre-approval inspections in 2021[44]. - The ongoing open-label study for L606, a liposomal formulation of treprostinil, is evaluating its efficacy for PAH and PH-ILD[27]. - The upcoming global placebo-controlled efficacy study for L606 will include approximately 340 patients across more than 100 sites in at least 20 countries[68]. - The company plans to commercialize YUTREPIA in the U.S. market for PAH and PH-ILD, leveraging existing relationships with healthcare providers[74]. Technology and Innovation - YUTREPIA's formulation utilizes PRINT technology to enhance aerosolization and lung deposition, allowing for a low-effort delivery device[40]. - The proprietary PRINT technology allows for the engineering of drug particles that enhance pharmacological benefits, including prolonged duration of drug release and reduced adverse side effects[69]. - The L606 inhalation system is designed for rapid delivery using breath-actuated smart technology, allowing for a dose in less than 2 minutes[62]. - The L606 formulation demonstrated comparable bioavailability to nebulized Tyvaso, with a single dose of L606 (51 mcg) resulting in a significantly reduced peak plasma concentration approximately 7.3-fold lower than Tyvaso (54 mcg)[65]. - L606 is expected to reduce dosing frequency to twice daily, with extended plasma concentrations up to 12 hours after a single dose[66]. Regulatory Environment - The regulatory approval process for drug candidates in the United States requires substantial time and financial resources, with no guarantee of timely approvals[135]. - The FDA's Investigational New Drug application (IND) must be submitted before human clinical studies can begin, and it automatically becomes effective 30 days after receipt unless concerns are raised[137]. - Clinical studies are conducted in three phases: Phase 1 focuses on safety and dosage, Phase 2 evaluates efficacy and safety in a limited population, and Phase 3 assesses overall risk/benefit in a larger patient population[145]. - The submission of a New Drug Application (NDA) is subject to a substantial application user fee, with waivers available under certain conditions[148]. - The NDA review process includes an in-depth substantive review to determine safety and effectiveness, which may involve inspections of manufacturing facilities[153]. Competitive Landscape - The pharmaceutical industry is highly competitive, with major competitors having greater financial resources and faster marketing approval processes[110]. - Liquidia's product candidates, YUTREPIA and L606, will compete in the PAH and PH-ILD markets against existing therapies and new entrants[112]. - The company is pursuing both composition-of-matter patents and method-of-use patents for its product candidates[105]. Partnerships and Agreements - A license agreement with Pharmosa was established in June 2023, granting exclusive rights in North America for L606, with an upfront fee of $10 million and potential milestone payments of up to $37.75 million for clinical development[82][84]. - The company has a Promotion Agreement with Sandoz for Treprostinil Injection, which includes a profit-sharing structure where Liquidia PAH receives 50% of net profits up to $500 million and 75% for profits exceeding that amount[90]. - The company has entered into a Pump Development Agreement with Sandoz and Mainbridge for a new pump for subcutaneous administration of treprostinil, with shared development costs[89]. Compliance and Regulatory Risks - Compliance with healthcare laws and regulations may constrain the company's business and financial arrangements[216]. - The company faces risks related to government regulation, including anti-kickback and false claims laws, which may impact operations[217]. - The FDA may withdraw product approval if regulatory compliance is not maintained or if new safety issues arise post-marketing[194]. Market Dynamics and Pricing - Third-party payors significantly influence the coverage and reimbursement status of approved products, impacting commercialization[208]. - The U.S. government is increasingly interested in implementing cost containment programs, including price controls and restrictions on reimbursement[211]. - The Inflation Reduction Act of 2022 imposes a new corporate alternative minimum tax on corporations with average annual financial statement income exceeding $1 billion over a three-year period[214]. - Increased emphasis on managed care and cost containment measures in the U.S. is expected to continue, putting pressure on pharmaceutical pricing and coverage[215].

Financial Performance - Liquidia Corporation reported a net loss of $130.4 million or $1.66 per share for the year ended December 31, 2024, compared to a net loss of $78.5 million or $1.21 per share for the previous year, reflecting an increase in losses of 66.3%[14] - Revenue for the year was $14.0 million, down from $17.5 million in 2023, a decrease of 20% primarily due to lower sales quantities of Treprostinil Injection[9] - Total revenue for the year ended December 31, 2024, was $13,996,000, a decrease of 20.5% compared to $17,488,000 in 2023[24] - The net loss for the year ended December 31, 2024, was $130,394,000, compared to a net loss of $78,502,000 in 2023, representing a 66.3% increase in losses[24] - The net loss per common share, basic and diluted, was $1.66 in 2024, compared to $1.21 in 2023[24] Expenses - Research and development expenses rose to $47.8 million, an increase of 11% from $43.2 million in 2023, driven by higher costs associated with the L606 program and YUTREPIA research[11] - General and administrative expenses surged to $81.6 million, an increase of 82% from $44.7 million in 2023, largely due to higher personnel costs and legal fees related to ongoing litigation[12] - Total costs and expenses increased significantly to $135,290,000 in 2024, up 48.9% from $90,872,000 in 2023[24] - Research and development expenses rose to $47,842,000 in 2024, an increase of 10.5% from $43,242,000 in 2023[24] - General and administrative expenses surged to $81,569,000 in 2024, up 82.2% from $44,742,000 in 2023[24] Cash and Assets - Cash and cash equivalents increased to $176.5 million as of December 31, 2024, compared to $83.7 million at the end of 2023, representing a growth of 110.5%[8] - The accumulated deficit as of December 31, 2024, was $559,492,000, compared to $429,098,000 at the end of 2023[24] - Total assets grew to $230,313,000 in 2024, compared to $118,332,000 in 2023[24] - Total stockholders' equity increased to $77,275,000 in 2024, up from $47,293,000 in 2023[24] Regulatory and Clinical Developments - The FDA granted tentative approval for YUTREPIA on August 16, 2024, with potential for final approval after the expiration of regulatory exclusivity on May 23, 2025[3] - The ASCENT study for YUTREPIA is nearing enrollment completion, showing promising tolerability and titratability in patients with PH-ILD[4] - Liquidia is advancing clinical studies for L606, with ongoing safety studies in patients with PAH and PH-ILD, aiming for a pivotal study to support approval[5] - The company anticipates receiving $50 million upon the first commercial sale of YUTREPIA following FDA approval, contingent on no injunctions against commercialization[7] Financing - Liquidia has strengthened its financial position with an amendment to its financing agreement with HCRx, providing up to an additional $100 million in funding[7]

Core Insights - Liquidia Corporation reported financial results for the full year ended December 31, 2024, highlighting a strengthened financial position and progress towards the potential approval of YUTREPIA [1][2][8] Financial Performance - Cash and cash equivalents increased to $176.5 million as of December 31, 2024, compared to $83.7 million as of December 31, 2023 [7] - Revenue for the year was $14.0 million, a decrease from $17.5 million in 2023, primarily due to lower sales quantities of Treprostinil Injection [9] - Cost of revenue rose to $5.9 million from $2.9 million in the previous year, attributed to sales force expansion [10] - Research and development expenses increased by 11% to $47.8 million, driven by higher costs related to the L606 program and YUTREPIA activities [11] - General and administrative expenses surged by 82% to $81.6 million, mainly due to increased personnel costs and legal fees [12] - Net loss for the year was $130.4 million, or $1.66 per share, compared to a net loss of $78.5 million, or $1.21 per share, in 2023 [14] Corporate Developments - Liquidia amended its financing agreement with HCRx to secure up to an additional $100 million, with $25 million received at closing [6] - The FDA granted tentative approval for YUTREPIA for PAH and PH-ILD, with potential final approval expected after the expiration of regulatory exclusivity on May 23, 2025 [3][8] - The ASCENT study for YUTREPIA is nearing enrollment completion, showing promising tolerability and titratability in PH-ILD patients [4] - Clinical studies for L606, a sustained-release formulation of treprostinil, are ongoing, with a pivotal study planned to support approval for both PAH and PH-ILD [5][17] Market Context - YUTREPIA is positioned as a therapeutic alternative for patients with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) [2][15] - The prevalence of PAH in the U.S. is estimated at 45,000 patients, while PH-ILD affects over 60,000 patients, indicating a significant market opportunity for Liquidia's products [19][20]

Core Insights - Liquidia Corporation has entered into a sixth amendment with HealthCare Royalty to secure up to an additional $100 million in financing for the development of its therapies, particularly YUTREPIA™ [1][2] - The funding will be utilized for the ongoing commercial development of YUTREPIA for pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), as well as for other clinical trials and general corporate purposes [1][2] Financing Details - The agreement includes an initial $25 million at closing, with potential additional tranches of $50 million upon the first commercial sale of YUTREPIA after FDA approval, and $25 million upon reaching net sales exceeding $100 million by June 30, 2026 [2] - Payments to HealthCare Royalty are capped at 175% of the total amounts advanced, with minimum rates of return set at 16%, 13%, and 12% for the new tranches [3] Company Overview - Liquidia Corporation focuses on developing innovative therapies for rare cardiopulmonary diseases, with YUTREPIA being its lead candidate for treating PAH and PH-ILD [4] - The company is also working on L606, a sustained-release formulation of treprostinil, and markets a generic version of Treprostinil Injection for PAH treatment [4] HealthCare Royalty Overview - HealthCare Royalty is a prominent royalty acquisition company that has invested over $5 billion in more than 90 biopharmaceutical products [5]

Core Viewpoint - Liquidia Corporation has announced a sixth amendment to its agreement with HealthCare Royalty to secure up to an additional $100 million in financing to support the development of its product YUTREPIA™ and other corporate purposes [1][2]. Financing Details - The financing will be provided in three tranches: $25 million at closing, $50 million upon the first commercial sale of YUTREPIA after FDA approval, and $25 million upon mutual agreement after achieving net sales exceeding $100 million by June 30, 2026 [2]. - The total payments to HealthCare Royalty are capped at 175% of the amounts advanced, with minimum rates of return set at 16%, 13%, and 12% for the new tranches [3]. Product Development Focus - Liquidia is focused on the commercial development of YUTREPIA for treating pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) [1][4]. - The company is also pursuing clinical trials for pediatric patients and further evaluations for WHO Group 1 and Group 3 patients [1]. Management Insights - The Chief Financial Officer of Liquidia expressed optimism that the new financing, combined with a successful launch of YUTREPIA, could lead the company to profitability without needing additional capital [3]. - The CEO of HealthCare Royalty emphasized the potential of YUTREPIA to meet unmet medical needs in patients with PAH and PH-ILD [3]. Company Overview - Liquidia Corporation specializes in developing innovative therapies for rare cardiopulmonary diseases, with a focus on pulmonary hypertension and proprietary PRINT® Technology [4]. - The company also markets generic Treprostinil Injection for PAH treatment and is developing L606, a sustained-release formulation of treprostinil [4]. HealthCare Royalty Overview - HealthCare Royalty is a leading royalty acquisition company that has invested over $5 billion in more than 90 biopharmaceutical products [5].