BioCryst Pharmaceuticals FY Conference Summary Company Overview - **Company**: BioCryst Pharmaceuticals (NasdaqGS: BCRX) - **Event**: FY Conference held on January 12, 2026 - **Key Speakers**: Charlie Gayer (CEO), Babar Ghias (CFO), Dr. Bill Sheridan (Chief Development Officer) Core Industry and Company Insights Industry: Rare Disease Pharmaceuticals - BioCryst focuses on developing treatments for rare diseases, particularly hereditary angioedema (HAE) Key Developments in 2025 - **ORLADEYO Growth**: Achieved the highest number of new patient prescriptions in the U.S. since its launch, with a paid rate improvement ending Q1 at 84% and the year at 81% compared to a long-term goal of 85% [3][4] - **European Business Sale**: Sold the European business for $250 million to streamline operations and focus on U.S. growth [3][4] - **Profitability**: Achieved profitability for the first time in company history, with expectations to maintain profitability moving forward [4] - **New Approval**: Received approval for ORLADEYO for children as young as two years old, expanding the market potential [4] Financial Performance - **2025 Preliminary Results**: - ORLADEYO revenue: $601 million, exceeding guidance of $590-$600 million - Ongoing business revenue (excluding European business): $563 million - Cash position: Nearly $340 million [5] - **2026 Revenue Guidance**: Anticipating ORLADEYO revenue of $625-$645 million and total revenue (including RAPIVAB) of $635-$660 million [5] Growth Strategy - **Three Pillars for Growth**: 1. **ORLADEYO**: Expected to reach $1 billion in peak sales by 2029 with a contribution margin over 80% [6] 2. **Novanavart**: Acquired through Astria Therapeutics, expected to enhance the portfolio with a differentiated injectable product [4][12] 3. **Netherton Syndrome Program**: Aiming to develop a transformative therapy for a high-need rare disease with no current treatments [20][21] Market Dynamics - **HAE Market**: Competitive landscape with 10-11 approved products, but ORLADEYO remains the only oral prophylaxis option, which is a significant differentiator [28][29] - **Patient Retention**: Approximately 50% of patients remain on ORLADEYO after five years, indicating strong efficacy and satisfaction [9] Future Outlook - **Revenue Growth**: Projected 15% year-over-year CAGR for ORLADEYO through 2029, with additional contributions from Novanavart and international markets [11][30] - **Acquisition of Astria**: Expected to close soon, enhancing BioCryst's capabilities in the HAE space [12][26] - **Netherton Syndrome**: Initial proof of concept trial expected to provide data by the end of 2026, with potential for pivotal study thereafter [26][43] R&D and Pipeline - **BCX 17725 for Netherton Syndrome**: Entering initial proof of concept trials, with a focus on safety and efficacy in patients [20][24] - **Avoralstat for Diabetic Macular Edema**: Currently in phase 1 study, with plans to out-license post-study completion [31][48] Conclusion - BioCryst Pharmaceuticals is positioned for significant growth in the rare disease market, with a strong focus on innovative therapies and strategic acquisitions. The company aims to leverage its successful commercialization strategies and maintain profitability while expanding its product offerings.

Financial Data and Key Metrics Changes - The company reported quarterly revenue of $145.5 million, with $134 million attributed to Orlodayo, marking a significant increase from the previous year [6][28] - Annual revenue guidance for Orlodayo has been raised to between $580 million and $600 million, reflecting a growth of 33% to 37% over last year [6][29] - Operating profit for Q1 2025 was $21.2 million, with net income reported as slightly positive [29] - Cash at the end of the quarter stood at $317 million, and the company reduced its outstanding debt by $75 million, bringing it down to $249 million [28][30] Business Line Data and Key Metrics Changes - The U.S. commercial team successfully transitioned patients from free drug to paid status, achieving a 10 percentage point increase in the paid patient rate, now at 84% [10][12] - The paid rate among Medicare patients improved significantly, with 89% now receiving paid therapy [93] - The gross to net ratio improved, with expectations to maintain it around 15% for the full year [14][51] Market Data and Key Metrics Changes - The company noted a strong demand for Orlodayo, with new prescriptions slightly exceeding the best quarter from the previous year [8][10] - The percentage of U.S. patients who strongly prefer oral prophylaxis increased to 70%, up from 51% in 2023 [10] - The company anticipates reaching $1 billion in global revenue by 2029, with a more profitable path than previously expected [13][64] Company Strategy and Development Direction - The company is focusing on expanding its pipeline, with significant milestones achieved, including the submission of a pediatric NDA for Orlodayo [16] - The company aims to capture more revenue from Orlodayo as the patient base continues to grow, with a long-term goal of 85% paid therapy [12][13] - The company is committed to disciplined capital allocation to create sustainable long-term revenue growth while reducing debt [30][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the sustainability of the paid rate improvement, viewing 84% as a new floor for future performance [118] - The company is optimistic about the impact of the Inflation Reduction Act on patient affordability and access to Orlodayo [11][57] - Management highlighted the importance of real-world evidence in convincing payers of the drug's efficacy, which has improved payer conversations [94] Other Important Information - The company is preparing for the launch of a pediatric formulation of Orlodayo, with expectations for rapid deployment following approval [106] - The company is also advancing its clinical pipeline, with a focus on rare diseases and the potential for transformative treatments [16][18] Q&A Session Summary Question: What is the potential impact of the pediatric launch on Orlodayo? - Management expects the pediatric launch to be a significant tailwind, with around 500 pediatric patients in the U.S. potentially appropriate for prophylactic therapy [35][36] Question: Can you elaborate on the improvements in getting patients onto paid drug? - The team made investments in the reauthorization process and improved their execution, leading to a higher paid rate [46][47] Question: What benchmarks are being set for the DME program? - The company is looking for a clear reduction in retinal edema as a benchmark for moving forward with the DME program [41][97] Question: How sustainable is the paid rate improvement? - Management believes the paid rate improvement is sustainable, with 84% being a new baseline for future performance [118] Question: What is the expected timeline for the pediatric formulation launch? - The company aims to minimize the lag time between approval and market availability, similar to previous launches [106][107]