BioCryst Pharmaceuticals (BCRX) investor relations material

Business overview and financial position

• ORLADEYO revenue projected at $590–$600 million for the year, with a path to $1 billion peak sales by 2029, driven by strong US growth and upcoming pediatric indication approval.

• Achieved first year of profitability, a year ahead of schedule, and expects to maintain profitability even after the Astria acquisition.

• Divested EU business for $250 million, improving the balance sheet and enabling debt retirement.

• Expense guidance lowered to $430–$440 million, with further reductions expected post-EU divestiture and Astria integration.

• Operating profitability and margins projected to improve significantly through 2027 and beyond, with steady-state cost structure and synergies from Astria.

ORLADEYO commercial performance and strategy

• Maintains strong demand and patient retention despite new competitors, with consistent growth expected in Q4.

• US business outperforms initial expectations, offsetting loss of EU sales; pediatric indication (PDUFA date Dec 12) expected to further boost growth.

• Strategy to reach 8,000 untapped US patients includes expanding prescriber base and generating real-world evidence, especially for HAE with normal C1 inhibitor.

• Pediatric granule formulation to be priced equivalent to capsules, with high trial retention and favorable safety profile.

Pipeline and acquisition updates

• Astria acquisition on track for Q1 closing, bringing Nevenabart, a differentiated injectable for HAE, into the portfolio.

• Nevenabart targets 5,000+ injectable-experienced patients, leveraging existing commercial infrastructure for a cost-effective launch.

• Top-line pivotal data for Nevenabart expected Q1 2027, with launch anticipated in late 2028; EU commercialization likely via partnership.

• Netherton program expects initial data in Q1 and more robust data later in the year, aiming for pivotal study start in 2027.

• DME program to be spun out after initial nine-patient cohort, seeking a partner for further development.