Financial Data and Key Metrics Changes - Revenues for Q3 2025 were $17.2 million, a decrease of $24.5 million compared to Q3 2024, while revenues for the nine months ended September 30, 2025, were $74.8 million, down $54.7 million year-over-year [14] - Total operating expenses for Q3 2025 were $33.7 million, down from $52.4 million in Q3 2024, and for the nine months ended September 30, 2025, total operating expenses were $119.8 million compared to $191.8 million in the prior year [14][15] - The net loss for Q3 2025 was approximately $13.5 million or $0.49 per diluted share, compared to a net loss of $6.9 million or $0.26 per diluted share in Q3 2024 [16] Business Line Data and Key Metrics Changes - The ARCT-032 program for cystic fibrosis is progressing with a planned 12-week study to begin in the first half of 2026, following positive interim data from the ongoing phase 2 clinical trial [5][9] - The ARCT-810 program for ornithine transcarbamylase deficiency is preparing for regulatory meetings in 2026 to discuss pivotal trial strategies for both pediatric and adult populations [9][10] Market Data and Key Metrics Changes - The Costave COVID-19 vaccine program has faced delays in the BLA filing due to regulatory changes by the FDA, impacting commercial visibility in the U.S. [13] - The Japanese partner, Meiji Seika Pharma, has launched a two-dose vial of Costave updated for the JN.1 variant in Japan, marking the first distribution of this presentation [10] Company Strategy and Development Direction - The company is focused on advancing its mRNA therapeutics and vaccines pipeline, with a commitment to addressing the unmet medical needs in cystic fibrosis and OTC deficiency [9][18] - Cost reductions are being implemented to extend the runway for the cystic fibrosis and OTC programs, while expecting continued support from CSL for commercialization in Asia and Europe [13][17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing clinical trials and the potential for ARCT-032 to address significant unmet medical needs in cystic fibrosis [7][8] - The company remains in a strong financial position with a cash runway extended into 2028, allowing for the achievement of near-term milestones [17] Other Important Information - The company plans to add additional sites for the upcoming 12-week study to accelerate enrollment, particularly targeting countries with higher prevalence of class one CF patients [78] - The FDA's stance on CT imaging as a potential endpoint for approval is still under discussion, with management indicating that it may serve as a supportive endpoint rather than a primary one [54] Q&A Session Summary Question: Have you been able to do some PK/PD modeling for the third-dose cohort? - Management confirmed that the third cohort is being evaluated similarly to the first two cohorts, with additional data collection planned for the 12-week study [21] Question: What are the optionalities for the OTC pivotal programs? - Management indicated that separate discussions with regulatory agencies will be held for adult and pediatric populations, focusing on different biomarkers for each group [24] Question: Do you expect improvements in mucus over time in the CF study? - Management noted that mucus plugs form in smaller airways and that improvements are expected over time, with the 12-week study designed to assess this [28] Question: What metrics will drive the go/no-go decision for the 15-mg dose? - Management stated that safety and tolerability will be key metrics, along with observing a dose response in mucus plug reduction [37] Question: When should we expect to see data from the 15-mg cohort? - Data from the 15-mg cohort is expected in the first quarter of next year, with plans to transition quickly to the 12-week study thereafter [44] Question: What is the age range for the pediatric population in the OTC program? - Management indicated that the age cutoff for pediatric patients will be determined in discussions with regulatory agencies [80]