BioCryst Pharmaceuticals (BCRX) investor relations material

Company overview and product portfolio

• Focuses on rare disease commercialization with a differentiated, patient-focused approach.

• ORLADEYO is the only oral prophylactic therapy for HAE, generating $602 million in revenue last year.

• Pipeline includes late-stage navenibart for HAE (phase III, BLA filing planned for end of 2027) and early-stage BCX17725 for Netherton syndrome.

ORLADEYO performance and outlook

• Maintains full-year revenue guidance of $625M-$645M, with Q1 results in line with expectations.

• Consistent new patient demand, with more new starts last year than in the first year of launch.

• 82% of patients are reimbursed, with payer mix stable and gross-to-net tightening to 15%.

• Peak sales guidance of $1B (excluding EU), driven by 150+ net new US patients/year and pediatric expansion.

• Pediatric launch delayed by batch issue, but root cause identified and product expected soon.

Market dynamics and competition

• HAE market has 10 branded products; ORLADEYO growth comes from both switches and new starts.

• No contracting pressure; ORLADEYO is now the #2 prophylaxis product by prescription.

• Confident in $1B peak sales despite upcoming oral competitor (Pharvaris) and gene editing entrants (Intellia).

• Oral on-demand Ekterly has not impacted ORLADEYO's trajectory.