BioCryst Pharmaceuticals (NasdaqGS:BCRX) FY Conference Transcript

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.