BioCryst Pharmaceuticals (BCRX) Morgan Stanley 24th Annual Global Healthcare Conference summary

Strategic vision and business evolution

• Transitioned from internal discovery to external innovation to expand rare disease portfolio and leverage profitability.

• Aims to have three marketed products and over $1 billion in revenue by 2030, with stable costs and growing profitability reinvested into pipeline expansion.

• Built a strong leadership team focused on external opportunities and risk-managed pipeline growth.

• Acquisition of Astria and navenibart exemplifies the shift to acquiring late-stage and earlier phase assets.

ORLADEYO franchise performance and market dynamics

• ORLADEYO remains the only oral prophylactic for HAE, driving strong demand and long-term growth nearly six years post-launch.

• Real-world evidence and physician experience reinforce confidence in efficacy and patient retention.

• Pediatric indication with a new formulation is exceeding expectations and may transform treatment for children.

• Prophylactic therapy now dominates HAE treatment, with 80% of patients on such regimens.

• Patient priorities are efficacy, safety, modality preference, and market access.

Competitive landscape and future outlook

• Recent competitor phase III data was anticipated and modeled; new oral therapies may slow growth but are unlikely to erode the established base.

• Patients satisfied with current oral therapy are unlikely to switch, while new entrants will compete for incident patients.

• Navenibart complements the portfolio by targeting injectable-preferring patients, offering less frequent dosing and strong efficacy.

• Pivotal ALPHA-ORBIT trial for navenibart is fully enrolled, with data expected in Q3 next year and high confidence in both dosing regimens.

• Commercial infrastructure for ORLADEYO is fully leveraged for navenibart, supporting operating leverage as revenue grows.