1. They are like a **Vertex Pharmaceuticals** for rare diseases, but also developing antiviral medicines.

2. Think of them as a specialized **Gilead Sciences** that also has a strong focus on developing oral drugs for rare diseases.

• RAPIVAB (peramivir injection): An intravenous neuraminidase inhibitor for the treatment of acute uncomplicated influenza.
• ORLADEYO: An oral serine protease inhibitor used to treat hereditary angioedema.
• BCX9930: An oral factor D inhibitor currently in Phase II clinical trials for complement-mediated diseases.
• BCX9250: An oral activin receptor-like kinase-2 inhibitor in Phase I clinical trials for fibrodysplasia ossificans progressiva.
• Galidesivir: A RNA dependent-RNA polymerase inhibitor in Phase I clinical trials for various RNA viruses, including Marburg, Yellow Fever, Ebola, and Zika.

BioCryst Pharmaceuticals (BCRX) sells primarily to other companies through collaboration and in-license relationships for the distribution and marketing of its pharmaceutical products in various regions. The major customer companies identified from the provided description are:

• Torii Pharmaceutical Co., Ltd. (TYO: 4551)
• Seqirus UK Limited (a subsidiary of CSL Limited, ASX: CSL)
• Shionogi & Co., Ltd. (TYO: 4507)
• Green Cross Corporation (KRX: 006280)
• Mundipharma International Holdings Limited (private company)

Charlie Gayer, President and Chief Executive Officer

Charlie Gayer was appointed President of BioCryst Pharmaceuticals in August 2025 and assumed the role of Chief Executive Officer on January 1, 2026. He also joined the Board of Directors in January 2026. Mr. Gayer joined BioCryst in 2015 as Vice President of Global Strategic Marketing and was promoted to Chief Commercial Officer in January 2020. Before joining BioCryst, he held several U.S. and global commercial leadership positions in rare disease categories at Talecris Biotherapeutics, Inc., where he led U.S. alpha-1 antitrypsin deficiency marketing and later European sales and marketing. After Talecris was acquired by Grifols, S.A., Mr. Gayer led the U.S. marketing team for the combined immune globulin product portfolio. Earlier in his career, he spent six years at GlaxoSmithKline in various marketing and sales roles. He began his career as a strategic consultant for biopharmaceutical companies and served as a business analyst at Genzyme Corporation for three years. Mr. Gayer earned his B.A. in politics from Princeton University and his M.B.A. from the Fuqua School of Business of Duke University.

Babar Ghias, Chief Financial Officer and Head of Corporate Development

Babar Ghias was appointed Chief Financial Officer and Head of Corporate Development at BioCryst Pharmaceuticals, effective July 7, 2025. Prior to joining BioCryst, Mr. Ghias served as CFO at AvenCell Therapeutics since 2022. He previously served as executive vice president of investments and portfolio management at Paragon Biosciences, a global biotech development firm. At Paragon, he launched four biotechnology companies focused on rare diseases. From 2014 to 2017, Mr. Ghias was CFO and head of corporate development at Marathon Pharmaceuticals, a specialty rare disease company, where he prepared the company for a commercial launch and led its successful sale. He has raised over $1 billion in capital for companies he has been involved with. Earlier in his career, for over a decade, he was an investment banker at Credit Suisse, where he provided strategic advice to clients and boards of directors in the healthcare and life sciences industries, closing over $80 billion in transactions. Mr. Ghias holds an M.B.A. with honors from Washington University in St. Louis and a B.S. in economics from Lahore University of Management Sciences in Pakistan.

William P. Sheridan, Chief Development Officer

Dr. William P. Sheridan became Chief Development Officer at BioCryst Pharmaceuticals on September 14, 2022, focusing on advancing the company's pipeline. He initially joined BioCryst in July 2008 as Chief Medical Officer. Before his tenure at BioCryst, Dr. Sheridan spent 15 years at Amgen, Inc., where he held various Vice President-level roles, including Vice President-North American Medical Affairs. At Amgen, he was instrumental in building the international medical affairs function and contributing to the successful launch of numerous compounds, such as Aranesp®, Enbrel®, Kineret®, Neulasta®, and Sensipar®. Prior to Amgen, he served as the Head of the Bone Marrow Transplant Service at Royal Melbourne Hospital. Dr. Sheridan earned his MB BS degree (MD equivalent) at the University of Melbourne in Victoria, Australia. He is a board-certified Fellow of the Royal Australasian College of Physicians, with a sub-specialty in medical oncology, and a Fellow of the American College of Physicians.

Ryan Arnold, Chief Medical Officer

Dr. Ryan Arnold was appointed Chief Medical Officer of BioCryst Pharmaceuticals on September 15, 2022. He joined BioCryst in March 2022 as Senior Vice President of Global Medical Affairs. Dr. Arnold has over 20 years of industry experience, with prior roles of increasing responsibility in medical affairs at various companies, including Amgen, Genzyme, Biogen, Sage Therapeutics, and Annexon Biosciences. Throughout his career, he has contributed to building and leading medical functions, supporting the development and launch of numerous specialty and rare disease products across neurology, hematology, immunology, oncology, and other areas. Dr. Arnold holds a Doctorate of Osteopathic Medicine from the Michigan State University College of Osteopathic Medicine and completed residency training that included clinical responsibilities in otolaryngology and facial plastic surgery.

Alane Barnes, Chief Legal Officer and Corporate Secretary

Alane Barnes serves as the Chief Legal Officer and Corporate Secretary for BioCryst Pharmaceuticals. She has been with BioCryst as General Counsel since June 2006 and as Corporate Secretary since December 2006. Ms. Barnes has been involved in company filings as recently as January 2026.

1. Competition for ORLADEYO: BioCryst's flagship product, ORLADEYO (berotralstat), an oral treatment for hereditary angioedema (HAE), faces significant and growing competition in the market. This competition comes from established injectable HAE therapies, such as Takeda's Takhzyro and CSL Behring's Haegarda, as well as emerging oral options like Pharvaris' deucrictibant and sebetralstat. This competitive landscape could exert pressure on ORLADEYO's market share, pricing, and overall sales growth, potentially impacting BioCryst's revenue and its trajectory towards sustained profitability. Some analyses suggest a potential reduction in ORLADEYO's market share in the coming years.
2. Clinical Development and Regulatory Risk for Pipeline Products: The future growth and diversification of BioCryst are heavily reliant on the successful development and regulatory approval of its pipeline candidates. The company has several drugs in early to mid-stage clinical trials, including BCX9930 (Phase II for complement-mediated diseases), BCX9250 (Phase I for fibrodysplasia ossificans progressiva), and Galidesivir (Phase I for various RNA viruses). Clinical trials inherently carry high risks of failure, and the inability to achieve positive results or secure necessary regulatory approvals for these programs would significantly hinder the company's long-term growth prospects. Even for ORLADEYO, delays or failures in obtaining approval for label expansions, such as for pediatric patients, can negatively affect growth projections.
3. Dependence on ORLADEYO and Challenges to Sustained Profitability: BioCryst's financial health and ability to achieve sustained profitability are substantially tied to the continued commercial success of ORLADEYO. While the company has shown progress towards profitability, achieving and maintaining consistent positive cash flow remains a challenge. If ORLADEYO's revenues do not meet expectations, or if research and development expenses for pipeline candidates or commercial costs for marketed products are higher than anticipated, BioCryst may need to seek additional funding, potentially leading to shareholder dilution.

BioCryst Pharmaceuticals' main products, ORLADEYO and peramivir injection, target significant addressable markets.

ORLADEYO (Hereditary Angioedema - HAE)

The global hereditary angioedema (HAE) therapeutics market was valued at approximately $4.1 billion in 2023 and is projected to reach $9.1 billion by 2032, exhibiting a compound annual growth rate (CAGR) of 9.2% from 2024 to 2032. Another estimate places the global HAE therapeutic market size at $4.25 billion in 2024, with an expectation to reach $8.29 billion by 2032, at a CAGR of 8.72% during the forecast period. The United States represents a substantial portion of this market, accounting for approximately 90% of the HAE market size, or around $2 billion in 2023. North America held a 46.8% share of the global HAE therapeutics market in 2023 and is anticipated to maintain its dominance. BioCryst Pharmaceuticals projects that ORLADEYO could achieve peak annual sales exceeding $1 billion.

Peramivir Injection (Influenza)

Peramivir is an intravenous neuraminidase inhibitor used for the treatment of acute uncomplicated influenza. The global peramivir market is projected to reach $10.55 billion by 2030, with a robust CAGR of 15.55% from 2025 to 2033. In 2024, peramivir accounted for 18% of global anti-influenza drug prescriptions, which totaled over 550 million. More broadly, the global influenza antiviral drugs market is projected to reach approximately $5.8 billion by 2033, growing at a CAGR of 4.5% from 2025 to 2033. The global neuraminidase inhibitors drug market, which includes peramivir, was valued at $5.1 billion in 2025 and is projected to reach $9.7 billion by 2035, growing at a CAGR of 6.8%. North America is expected to hold a significant share of this market, with a projected 39.9% by 2035.

BioCryst Pharmaceuticals (BCRX) is poised for future revenue growth over the next 2-3 years, driven by several key factors:

1. Continued Growth and Market Penetration of ORLADEYO in Adult Hereditary Angioedema (HAE) Patients: BioCryst anticipates sustained revenue increases from its flagship product, ORLADEYO. The company projects full-year 2026 ORLADEYO revenues to be between $625 million and $645 million, representing approximately 13% growth over 2025 revenues adjusted for Europe. This growth is fueled by increasing patient volume, strong patient retention rates (e.g., 61% of patients remaining on treatment at twelve months), and strategic pricing adjustments, including a 9% price increase in early January (2026) that is expected to result in a net 4.5% increase. BioCryst also has a long-term goal of ORLADEYO achieving $1 billion in annual sales by 2029.
2. Expansion into the Pediatric HAE Market with ORLADEYO Pellets: A significant driver of future revenue is the recent FDA approval in December 2025 for ORLADEYO oral pellets for HAE patients aged 2 to less than 12 years. This new formulation positions ORLADEYO as a likely sole oral prophylactic option for this pediatric population for several years, opening up a new and important market segment. Management has expressed strong confidence in the long-term revenue potential from this pediatric indication.
3. Advancement and Potential Launch of Navenibart for HAE: The acquisition of Astria Therapeutics in late 2025/early 2026 introduced Navenibart, a subcutaneously administered drug, into BioCryst's HAE pipeline. Navenibart is currently in Phase III clinical trials, with top-line data anticipated in early 2027 and a potential regulatory filing by the end of 2027. This asset is designed to be a differentiated injectable prophylaxis for HAE, offering the potential for less frequent dosing (every three or six months), and is expected to address an additional addressable market of over 5,000 patients, further strengthening BioCryst's HAE franchise.

Share Issuance

• BioCryst Pharmaceuticals registered 11,000,000 shares of common stock for issuance under its Stock Incentive Plan in 2025.
• The total outstanding shares for BioCryst Pharmaceuticals increased from 182 million to 210 million between 2021 and 2025.
• In Q4 2025, shares outstanding increased by 1.2% from the prior quarter, reaching 213 million shares.

Inbound Investments

• BioCryst Pharmaceuticals ended 2025 with $337.5 million in cash and investments.
• The company had access to a $400 million financing facility with Blackstone Life Sciences.

Outbound Investments

• BioCryst completed the acquisition of Astria Therapeutics, Inc. in late 2025/early 2026 to expand its Hereditary Angioedema (HAE) portfolio.
• In October 2025, BioCryst sold its European ORLADEYO business to Neopharmed Gentili S.p.A., generating $243.3 million in revenue.

Capital Expenditures

• Capital expenditures were reported as -$2.47 million in the last 12 months.
• In Q4 2025, capital expenditures were $802K, marking a decrease of 40.3% from the prior quarter.
• Increased research and development (R&D) costs are anticipated in 2026, primarily due to ongoing Phase III trials and BLA enabling CMC activities for Navenabart.