BioCryst Pharmaceuticals (BCRX) Goldman Sachs 46th Annual Global Healthcare Conference summary

Strategic focus and commercial performance

• Focus on rare diseases with a disruptive oral therapy approach, expanding into protein therapeutics and injectables.

• ORLADEYO serves as the revenue engine, showing strong commercial growth and market disruption in HAE.

• Achieved accelerated path to profitability, expecting to be net income and cash flow positive for the full year.

• Paid patient rate for ORLADEYO reached 84%, with significant Medicare improvements due to policy changes.

• Minimal price increases and robust patient support services have driven sustained growth and payer confidence.

Market insights and patient/physician dynamics

• Real-world evidence demonstrates ORLADEYO's efficacy matches injectables, increasing physician confidence.

• Patient feedback highlights life-changing convenience, reducing disease burden and stress.

• Market research and specialty pharmacy data inform strategies to address friction points and optimize switching.

• Pediatric opportunity seen as significant, with easy-to-administer mini tabs and potential halo effect in families.

• Market share expansion driven by new patient growth, high paid conversion, and durable patient retention.

Pipeline development and clinical milestones

• Two key programs, BCX-17725 for Netherton syndrome and Avoralstat for DME, are advancing with data expected by year-end.

• BCX-17725 aims to address a severe unmet need in Netherton syndrome, with initial patient studies underway.

• Avoralstat leverages a novel delivery method for eye disease, targeting patients unresponsive to VEGF inhibitors.

• Both programs are designed for efficient capital use, with early clinical data to inform further investment.

• Potential market sizes for both indications could expand as diagnosis and awareness improve.