BioCryst Pharmaceuticals (BCRX) investor relations material

Strategic vision and business development

• Aims to become a high-growth, innovative rare disease biotech with multiple marketed products by 2030, leveraging ORLADEYO's momentum and financial strength to diversify the portfolio.

• Recent divestiture of the European business improved profitability and enabled the acquisition of Astria Therapeutics, aligning with the goal of multiple products by 2030.

• Business development focuses on late-stage assets to minimize early development risk, with a preference for synergistic and complementary products.

• Maintains a disciplined approach to pipeline evolution, prioritizing quick decisions and focusing on areas with clear product differentiation.

• External business development and capital allocation, including potential share buybacks, are considered core to future growth, but risk will be managed conservatively in the near term.

Product portfolio and market positioning

• ORLADEYO, in its sixth year, continues to drive growth, with a target of $1 billion in peak sales by 2029, requiring an average of 150 net new patients annually.

• The company holds a unique position as the only oral therapy for HAE in pediatrics (ages two and up), with strong ongoing demand and new prescriber growth.

• Navenibart, a best-in-class injectable for HAE, is positioned as complementary to the oral product, targeting patients preferring less frequent dosing.

• Market research indicates significant demand for injectables with quarterly or semi-annual dosing, with navenibart aiming to be a game changer in this segment.

• The competitive landscape is expected to intensify with new oral entrants, but real-world data and long-term safety are seen as key differentiators.

Pipeline updates and clinical development

• Navenibart's phase III trial is the largest and longest in HAE, with BLA filing and top-line data expected at the end of next year; enrollment is on track to complete by mid-year.

• Phase II data for navenibart showed 90%-92% attack rate reduction, with durability and convenience as major advantages; regulatory benchmarks and real-world outcomes are emphasized.

• The Netherton syndrome program targets a severe, underdiagnosed rare derm condition, with proof-of-concept results expected by year-end; investment is measured until clinical activity is confirmed.

• The company is open to expanding into other rare derm or autoimmune indications, leveraging its established commercial infrastructure.

• Pipeline decisions are guided by clinical meaningfulness, patient need, and potential for rapid advancement if early signals are positive.