Financial Data and Key Metrics Changes - For the full year 2024, total revenue was $450.7 million, with Orlodeo contributing $437.7 million [29] - The company achieved a non-GAAP operating profit of $62.9 million, a significant increase from a non-GAAP operating loss of $48.1 million in 2023 [29] - Cash at the end of the year was approximately $343 million, with net cash utilization for the quarter at $8.4 million [30] Business Line Data and Key Metrics Changes - Orlodeo revenue grew by 34% year-over-year, reflecting strong execution by the commercial team [6][10] - In Q4, Orlodeo generated $124.2 million, with $17.2 million (13.9%) coming from ex-U.S. sales [29] - The company raised its Orlodeo revenue guidance for 2025 to between $535 million and $550 million [11] Market Data and Key Metrics Changes - The company noted a higher percentage of Medicare patients on Orlodeo can afford their co-payments due to the Inflation Reduction Act [10] - The paid patient rate across the entire patient base was 73.5%, with expectations to increase further [44][94] - The company expects to see continued strong demand in early 2025, consistent with the momentum from 2024 [10] Company Strategy and Development Direction - The company aims to file an NDA for Orlodeo for pediatric patients aged 2 to 11 this year [16][26] - The pipeline includes advancing BCX17725 for Netherton syndrome and ivorlstat for diabetic macular edema, with clinical data expected later this year [7][24] - The long-term revenue guidance includes a three-year CAGR of 20%, targeting over $750 million by 2027 and $1 billion by 2029 [30][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution and performance, stating that the momentum is expected to carry into 2025 and beyond [9] - The management highlighted the importance of the upcoming clinical data and the potential for BCX17725 to address an unmet need in Netherton syndrome [19][20] - The company anticipates positive and sustainable quarterly cash flow and EPS in the second half of 2025 [30] Other Important Information - The company is presenting real-world evidence at the Quad AI Conference, showcasing the effectiveness of Orlodeo in reducing attack rates for HAE patients [12] - The pediatric trial for Orlodeo showed that granules were safe and well-tolerated, with a high retention rate among participants [26][88] Q&A Session Summary Question: Timing for data from Netherton syndrome patients - Management indicated that data will be available later this year, with a focus on skin healing and KLK5 activity [35][38] Question: Proportion of paid patients and guidance for Orlodeo - The paid patient rate was 73.5% overall, with expectations for Medicare patients to increase significantly due to affordability improvements [44][94] Question: Tolerability of pediatric formulation compared to adults - The pediatric formulation showed good tolerability and compliance, similar to adult experiences [74] Question: International demand for Orlodeo - Demand in Europe, Canada, and Japan is growing, with confidence in Orlodeo's effectiveness leading to increased prescriptions [80][81] Question: Historical reauthorization rates - Approximately half of patients go through reauthorization in Q1, with improvements noted in the process over the years [104]