BioCryst Pharmaceuticals(BCRX)2025-02-24 17:17Financial Data and Key Metrics Changes - For Q4 2024, total revenue was 124.2 million [39] - Full year 2024 total revenue reached 437.7 million [39] - Non-GAAP operating profit was 48.1 million in 2023 [40] - Cash at the end of 2024 was approximately 8.4 million [41] Business Line Data and Key Metrics Changes - Orladeo revenue grew by 34% year-over-year, maintaining the same number of new patients as in its first year [9] - For Q4, 51.7 million (11.8%) [39] - The company raised Orladeo revenue guidance for 2025 to between 550 million [14] Market Data and Key Metrics Changes - 97% of surveyed US HAE treaters are considering prescribing Orladeo, with 59% extremely likely to prescribe it to more patients [17] - The company noted a significant increase in the percentage of Medicare patients able to afford co-payments due to the Inflation Reduction Act [13] Company Strategy and Development Direction - The company aims to file an NDA for Orladeo for pediatric use in children aged 2 to 11 this year [21] - BCX1775 for Netherton syndrome and Avoralstat for diabetic macular edema are advancing in clinical trials, with expectations for meaningful data readouts this year [21][28] - Long-term revenue guidance includes a three-year CAGR of 20%, targeting over 1 billion by 2029 [44] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution and performance across the company, highlighting a strong position for future growth [8] - The management team noted that the execution in 2024 has set the stage for positive and sustainable cash flow and EPS in the second half of 2025 [43] - There is optimism regarding the potential for BCX1775 to address an unmet need in Netherton syndrome, which is currently underdiagnosed [27] Other Important Information - The company is seeing strong commercial demand for RAPIVAB due to the prevalence of flu, despite some uncertainty around future government funding [42] - The pediatric oral granule formulation of Orladeo has shown promising results in trials, with high tolerability and retention rates [35][114] Q&A Session Summary Question: Can you narrow down when data from Netherton patients is expected? - Management indicated that data will be available later this year, with a focus on skin healing and KLK5 activity [51][52] Question: What is the current paid patient proportion for Orladeo? - The company reported a 73.5% paid rate across all patients, with about 20% of patients on Medicare [120] Question: How does the pediatric study's tolerability compare to adults? - The pediatric oral granules have shown good tolerability and compliance, similar to adult experiences [98] Question: What is the expected timeline for dosing Netherton syndrome patients? - Dosing for Netherton syndrome patients is expected to begin around mid-year, with data on skin activity anticipated by year-end [83] Question: What percentage of KLK5 suppression is needed for clinical benefit? - The company is looking for nearly complete suppression of KLK5 activity, although the exact percentage required for clinical benefit is not yet known [128]