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.

Core Insights - Galapagos NV has announced changes to its Board of Directors to enhance governance and align with its strategic direction focused on business development and novel medicines [1][10] Board Composition Changes - Dr. Neil Johnston has been appointed as a Non-Executive Independent Director, and Devang Bhuva as a Non-Executive Non-Independent Director, both effective November 1, 2025 [2] - An additional Non-Executive Independent Director is expected to be appointed soon to ensure a balanced and diversified Board [2] Departures from the Board - Non-Executive Independent Directors Dr. Elisabeth Svanberg and Dr. Susanne Schaffert, along with Non-Executive Non-Independent Director Andrew Dickinson, will step down from their positions effective November 1, 2025 [3] Leadership Statements - Jérôme Contamine, Chair of the Board, expressed confidence that the new directors' financial leadership and strategic transaction experience will be crucial for the company's transformation and value creation [4] - CEO Henry Gosebruch emphasized the importance of these changes for steering the company towards sustainable growth and enhancing stakeholder value [4] Director Backgrounds - Dr. Neil Johnston has extensive experience in biopharma, previously serving as Executive Chair at Yellowstone Biosciences and holding senior roles at Novartis [5] - Devang Bhuva has a strong background in corporate development and investment banking, currently serving as Senior Vice President at Gilead [6]

Core Viewpoint - Viatris Inc. has acquired Aculys Pharma, enhancing its portfolio in the Central Nervous System (CNS) therapy area with exclusive rights to develop and commercialize pitolisant and Spydia in Japan, targeting significant unmet medical needs [1][4][8] Acquisition Details - The acquisition includes exclusive development and commercialization rights for pitolisant in Japan, which is on track for marketing approval by the end of 2025 for treating excessive daytime sleepiness (EDS) and cataplexy in narcolepsy patients [2][4] - Viatris also acquired exclusive rights for Spydia Nasal Spray in Japan and certain Asia-Pacific markets, which was approved in June 2025 for treating status epilepticus [3][10] Strategic Alignment - The acquisition leverages Viatris' existing commercial infrastructure and expertise in CNS, aligning with its strategy to target accretive regional business development opportunities [4][8] - Viatris aims to expand its innovative product portfolio in Japan, which includes other products under regulatory review or in pivotal Phase 3 trials [4][14] Financial Terms - The acquisition agreement includes an upfront payment to Aculys Pharma shareholders, with additional payments contingent on achieving specified regulatory and commercial milestones, along with royalties on net sales [5][14] Clinical Trial Results - Recent Phase 3 clinical trial results in Japanese patients for pitolisant showed significant improvement in EDS compared to placebo, with no serious adverse events reported [7][9][14] - The OSAS Phase 3 trial demonstrated statistically significant improvement in EDS for patients receiving pitolisant, consistent with global clinical study results [9][14] Product Information - Pitolisant is a selective/inverse agonist of the histamine H3 receptor, approved in multiple countries for narcolepsy and OSAS, with a strong safety profile [6][14] - Spydia Nasal Spray is the first intranasal anti-seizure medication approved in Japan for status epilepticus, marking a significant advancement in treatment options [10][11]