BioCryst Pharmaceuticals (BCRX) investor relations material

Key achievements and business transformation

• Achieved record ORLADEYO new patient prescriptions in the U.S. in 2025, with paid rate improvement to 81% by year-end and first full year of profitability.

• Sold European business for $250 million, streamlining operations and improving margins.

• Entered into a proposed $700 million acquisition of Astria Therapeutics, adding late-stage HAE asset navenibart.

• Secured pediatric approval for ORLADEYO, expanding the addressable market to children as young as two.

• Achieved profitability for the first time and expect to remain profitable going forward.

Financial performance and guidance

• 2025 preliminary ORLADEYO revenue reached $601 million, exceeding guidance, with $563 million excluding EU sales.

• Over 1,600 US patients on therapy at year-end 2025.

• 2026 ORLADEYO revenue guidance is $625–$645 million; total revenue including RAPIVAB projected at $635–$660 million.

• Base operating expenses (pre-Astria) expected at $380–$390 million, with an additional $70–$80 million post-Astria acquisition.

• Expected cash balance of $1 billion+ by 2029, supporting future growth opportunities.

Growth strategy and market outlook

• ORLADEYO expected to reach $1 billion peak sales in 2029, driven by U.S. growth, pediatric expansion, and modest international contributions.

• Contribution margin for ORLADEYO approaching 80%, expected to exceed 90% with navenibart addition.

• Three strategic growth pillars: internal R&D, externalizing non-core assets, and targeted business development.

• U.S. HAE prophylaxis market growing 3–5% annually, with 80% of patients on prophylaxis.

• Addressable HAE market expanding, with navenibart targeting 5,000+ patients and ORLADEYO predicted to serve 2,000+.