Core Insights - BioCryst Pharmaceuticals announced the departure of Dr. Helen Thackray, the chief research and development officer, effective September 1, 2025, transitioning to an advisory role until the end of the year [1][2] - Dr. Thackray joined BioCryst in 2019 as a board member and became the chief research and development officer in 2021, being a finalist for the CEO succession process [2] - The CEO of BioCryst, Jon Stonehouse, expressed gratitude for Dr. Thackray's contributions, including the launch of a new protein therapeutics platform and advancements in various clinical programs [3] Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases [5] - The company utilizes structure-guided drug design to develop innovative oral small-molecule and protein therapeutics targeting challenging diseases [5] - BioCryst has commercialized ORLADEYO (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of therapies [5]

Financial Performance & Guidance - BioCryst's Q2 2025 was strong, driven by increased ORLADEYO demand[14] - ORLADEYO revenue is expected to meet the previous guidance range of $580-600 million for FY25, even excluding Q4 2025 EU revenue[16] - The company anticipates achieving full-year operating profit in 2026 and positive net income & cash flows in 2026+[58] - Cash, cash equivalents, restricted cash & investments totaled $287 million as of June 30, 2025[63] - The company paid down $50 million of its senior credit facility in July 2025[62] ORLADEYO & Market Dynamics - Market research indicates a growing patient preference for oral administration of HAE long-term prophylaxis (LTP) since 2023[17] - Monte Carlo simulation projects ORLADEYO reaching a steady state of over 2,000 patients in the US by 2028[25] - ORLADEYO revenue advances to royalty-free tier (>$550M)[61] - The blended royalty rate for ORLADEYO is expected to decline to approximately 4% at peak sales once OMERS reaches its cap[67] Pipeline Development - The PDUFA target date for the pediatric NDA for ORLADEYO is December 12, 2025[32] - An IND has been cleared by the FDA for BCX17725, a targeted KLK5 inhibitor for treating Netherton syndrome (NS)[37]

First Quarter 2025 Results Call Corporate Update & Financial Results May 5, 2025 CONFIDENTIAL & PROPRIETARY Forward-looking statements President and Chief Executive Officer ORLADEYO® update Charlie Gayer Chief Commercial Officer BioCryst's presentation contains forward-looking statements, including, but not limited to, statements regarding future results and forward-looking financial information, company performance, achievements, future market share or size, and expectations regarding pipeline development. ...

Core Insights - BioCryst Pharmaceuticals reported a strong Q1 2025 performance with ORLADEYO net revenue of $134.2 million, reflecting a 51% year-over-year increase [1][6] - The company has raised its full-year 2025 revenue guidance for ORLADEYO to between $580 million and $600 million, up from previous estimates [1][15] - BioCryst anticipates achieving profitability for the full year 2025, a year earlier than previously expected [1][16] Financial Performance - Total revenues for Q1 2025 reached $145.5 million, a 56.8% increase from $92.8 million in Q1 2024 [8] - Research and development expenses decreased to $37.3 million, down 19.8% from $46.5 million in Q1 2024 [9] - Selling, general and administrative expenses rose to $82.5 million, a 38.7% increase compared to $59.5 million in Q1 2024 [10] - Operating income for Q1 2025 was $21.2 million, compared to an operating loss of $14.5 million in Q1 2024 [11] Debt Management - The company paid down $75 million of its debt early in Q2 2025, which is expected to save approximately $23.5 million in interest over the loan's life [1][14] Product Development - BioCryst submitted a new drug application (NDA) for ORLADEYO oral granules for children aged 2-11 with hereditary angioedema (HAE) [1][5] - The company is progressing with clinical trials for BCX17725 for Netherton syndrome and avoralstat for diabetic macular edema (DME) [7][11] Market Dynamics - The percentage of ORLADEYO patients on paid drug increased to approximately 84%, up from 73.5% at the end of 2024, indicating strong market demand [6][4] - A recent survey indicated that 70% of U.S. HAE patients prefer oral prophylaxis therapy, up from 50% in 2023 [6]

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]