BioCryst Pharmaceuticals is like **Alexion, but for oral rare disease drugs.**

BioCryst Pharmaceuticals is like **Vertex, but focused on hereditary angioedema and related rare immunological disorders.**

• ORLADEYO (berotralstat): An oral medication approved for the prophylactic treatment of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
• RAPIVAB (peramivir injection): An intravenous antiviral drug approved for the treatment of acute uncomplicated influenza in adults and pediatric patients two years and older.
• BCX9930: An investigational oral Factor D inhibitor currently in clinical development for the treatment of complement-mediated diseases.

BioCryst Pharmaceuticals (BCRX) sells primarily to other companies, specifically a limited number of wholesale pharmaceutical distributors. These distributors then supply BioCryst's products to pharmacies, hospitals, and other healthcare providers, which ultimately dispense them to individual patients.

According to BioCryst's latest annual report (10-K), sales to its three largest wholesale distributors collectively accounted for approximately 99% of its gross product sales for the fiscal year ended December 31, 2023. These major customer companies are:

• Cencora, Inc. (COR)
• Cardinal Health, Inc. (CAH)
• McKesson Corporation (MCK)

Jon P. Stonehouse Chief Executive Officer; Director

Mr. Stonehouse has served as the Chief Executive Officer and Director of BioCryst Pharmaceuticals since 2007. He is set to retire as CEO on December 31, 2025, but will remain on the Board of Directors. Prior to BioCryst, he was Senior Vice President of Corporate Development at Merck KGaA, where his responsibilities included corporate mergers and acquisitions, global licensing and business development, and corporate strategy. During his tenure at Merck KGaA, he led the effort that resulted in the acquisition of Serono, S.A., which was then the largest biotechnology company in Europe. Mr. Stonehouse also held various positions at Astra Merck/AstraZeneca and began his pharmaceutical career as a sales representative at Merck & Co., Inc..

Babar Ghias Chief Financial Officer; Head - Corporate Development

Mr. Ghias was appointed Chief Financial Officer and Head of Corporate Development at BioCryst in 2025. He previously served as the Chief Financial Officer of AvenCell Therapeutics since 2022. He also held the role of Executive Vice President of Investments and Portfolio Management at Paragon Biosciences, a global biotech development firm, where he launched four biotechnology companies focused on rare diseases. From 2014 to 2017, he was the CFO and Head of Corporate Development at Marathon Pharmaceuticals, where he prepared the company for commercial launch and subsequently led its successful sale. Mr. Ghias has successfully raised over $1 billion in capital throughout his career for the companies he has been involved with. Earlier in his career, he spent over a decade as an investment banker at Credit Suisse, serving as a senior member of the mergers and acquisitions team and completing over $80 billion in transactions in the healthcare and life sciences industries.

Philip George Chief Strategy Officer

Mr. George has been the Chief Strategy Officer of BioCryst Pharmaceuticals since 2022. He brings over 30 years of experience in management consulting, primarily within the biopharmaceutical industry. His previous roles include leading the North American Pharmaceutical and Medical Products strategy group at Accenture (formerly Andersen Consulting). He has also worked as an independent consultant, focusing on early- to mid-stage biopharma companies.

Alane P. Barnes Chief Legal Officer; Secretary

Ms. Barnes has served as the Chief Legal Officer and Secretary of BioCryst Pharmaceuticals since 2018. Her prior positions within the company include Vice President, General Counsel, and Corporate Secretary. Before joining BioCryst, she served with the UAB Research Foundation. Ms. Barnes has a background in commercial litigation and healthcare law and was a Senior Licensing Associate at the University of Alabama at Birmingham. She is currently a board member of the Research Triangle Area Association of Corporate Counsel.

Charlie Gayer President And Chief Commercial Officer

Mr. Gayer holds the title of President and Chief Commercial Officer at BioCryst Pharmaceuticals. He is based in Durham, North Carolina.

The key risks to BioCryst Pharmaceuticals (BCRX) involve its financial health and liquidity, significant reliance on its primary product amid increasing competition, and the inherent volatility of its stock price.

1. Financial Health and Liquidity: BioCryst Pharmaceuticals has a history of operating losses and negative cash flows, with expectations for this to continue until revenues reach a sustainable level. The company's financial strength is considered poor, largely due to high debt levels and a low interest coverage ratio of 0.85, indicating that its earnings may not be sufficient to cover interest expenses, potentially necessitating further debt. An Altman Z-Score of -1.95 places the company in a financial distress zone, suggesting a potential risk of bankruptcy within the next two years. While the company projects a clear path to profitability with positive earnings per share and cash flows in the latter half of 2025, securing additional capital for future operations, drug development, and commercialization remains crucial.
2. Product Reliance and Market Competition for ORLADEYO: The company faces a significant risk due to its high reliance on a single product, ORLADEYO, for hereditary angioedema (HAE) treatment. The HAE market is becoming increasingly competitive, with other pharmaceutical companies actively developing and introducing alternative treatments, which could threaten ORLADEYO's market positioning and revenue sustainability. Recent reports indicate a slight decline in ORLADEYO's quarterly growth of new prescribers and anticipated further drops following the divestment of BioCryst's European business, contributing to a cautious outlook on future financial performance and market penetration. There is no assurance that the company's commercialization strategies for ORLADEYO will succeed or that the market will develop as projected.
3. Stock Price Volatility and Potential Dilution: BioCryst's stock price has historically been highly volatile, a common characteristic of biotechnology companies, and these fluctuations are often not directly tied to its financial results. This volatility could lead to a significant decline in investment value and has previously exposed companies to securities class action litigation. Furthermore, any future sales and issuances of additional securities by BioCryst could dilute the ownership interests of current stockholders and exert downward pressure on the stock price.

• Gene Therapies for Hereditary Angioedema (HAE): Companies like uniQure (with AMT-061) are developing gene therapies for HAE that aim to provide a long-term or potentially curative solution with a single administration. If successful, these therapies could fundamentally disrupt the market for chronic prophylactic treatments like BioCryst's ORLADEYO (berotralstat) by eliminating the need for ongoing medication. This represents a paradigm shift from continuous treatment to a potential one-time intervention, analogous to how new technologies can make existing solutions obsolete.
• Emergence of Competing Oral Plasma Kallikrein Inhibitors: ORLADEYO's primary competitive advantage is its oral administration, offering convenience over injectable HAE prophylactics. However, other companies, such as Protagonist Therapeutics with PTG-321, are developing their own oral plasma kallikrein inhibitors. Should a competing oral product demonstrate superior efficacy, safety, or convenience (e.g., less frequent dosing or better tolerability) in clinical trials, it could directly challenge ORLADEYO's market share and limit its growth potential.

BioCryst Pharmaceuticals' main products and their addressable markets are as follows:

• ORLADEYO (berotralstat): This oral therapy is for the prevention of hereditary angioedema (HAE) attacks. BioCryst Pharmaceuticals projects global peak sales for ORLADEYO to reach $1 billion. The company expects global net ORLADEYO revenue for the full year 2025 to be between $515 million and $535 million. ORLADEYO currently holds approximately 20% of the HAE market share, with projections indicating an increase to about 25% over the next three years.
• RAPIVAB (peramivir injection): An intravenous neuraminidase inhibitor for the treatment of influenza. The U.S. Department of Health and Human Services awarded BioCryst Pharmaceuticals a contract valued at up to $69 million for the procurement of up to 95,625 doses over a five-year period for the U.S. Strategic National Stockpile. This represents a U.S. government procurement market for RAPIVAB.

1. Continued Growth of ORLADEYO in Adult Hereditary Angioedema (HAE): ORLADEYO (berotralstat) is projected to continue its strong performance. BioCryst forecasts full-year 2025 global net ORLADEYO revenue to be between $590 million and $600 million. The drug demonstrated a 37% year-over-year revenue increase in the third quarter of 2025, driven by robust patient demand and consistent patient retention rates. The company anticipates ORLADEYO achieving $1 billion in peak revenue by 2029.
2. Expansion of ORLADEYO into the Pediatric HAE Market: BioCryst plans to expand ORLADEYO's market by introducing a granule formulation for children aged 2 to under 12. A new drug application (NDA) for this pediatric indication was submitted in 2025, with a PDUFA (Prescription Drug User Fee Act) date set for September 12, 2025. This expansion is expected to address a significant unmet medical need and further contribute to ORLADEYO's overall revenue growth.
3. Acquisition of Astria Therapeutics and Navenibart: BioCryst entered into a definitive agreement to acquire Astria Therapeutics, with the transaction anticipated to close in the first quarter of 2026. This acquisition is expected to bolster BioCryst's position in the HAE market and is projected to drive double-digit HAE revenue growth well into the 2030s through its product, Navenibart, after ORLADEYO revenue reaches a steady plateau.
4. Advancement of Pipeline Products, including BCX17725 for Netherton Syndrome: BioCryst is advancing its pipeline with programs targeting rare diseases. Notably, BCX17725, a KLK5 inhibitor for Netherton syndrome, is progressing in clinical development. An Investigational New Drug (IND) application for BCX17725 was open as of the first quarter of 2025, and promising data from this program is anticipated by the end of 2025. The progression of such pipeline assets could enhance the company's portfolio and market presence, potentially contributing to revenue growth in the later part of the 2-3 year timeframe.

Share Issuance

• In November 2021, BioCryst completed a Post IPO funding round, raising $350 million.
• As part of the October 2025 acquisition of Astria Therapeutics, BioCryst will issue stock, resulting in Astria stockholders owning approximately 15% of the proforma equity in the combined company.
• The number of shares outstanding for BioCryst Pharmaceuticals increased by 1.84% in one year as of the latest data.

Inbound Investments

• On September 30, 2024, Vanguard Group Inc acquired 1,537,103 shares of BioCryst Pharmaceuticals, increasing its stake to 9.81% through a purchase valued at $7.60 per share.
• BioCryst completed a Post IPO funding round on November 22, 2021, raising $350 million with participation from investors including Great Point Partners and Athyrium Capital Management.

Outbound Investments

• In October 2025, BioCryst announced a definitive agreement to acquire Astria Therapeutics for a mix of cash and stock, representing an implied aggregate equity value of approximately $920 million and an enterprise value of approximately $700 million.

Capital Expenditures

• In the last 12 months, capital expenditures were reported at -$2.34 million.
• The company anticipates achieving positive cash flow in the second half of 2025 and for the full year 2026, not including non-cash stock compensation.