BioCryst Pharmaceuticals (BCRX) 2024 Wells Fargo Healthcare Conference summary

Commercial performance and market dynamics

• ORLADEYO, the first oral therapy for hereditary angioedema (HAE), has seen consistent patient growth since launch, with 2024 sales expected between $420 million and $435 million globally.

• Key growth drivers include patient and physician preference for oral therapy, strong efficacy, and increasing confidence in the drug.

• 52% of patients switched from injectable prophylaxis, 32% from on-demand therapy, and 17-18% were therapy-naive; retention rates have stabilized at 60% at 12 months.

• Market research indicates ORLADEYO is expected to become the most prescribed HAE drug over the next 12 months, with demand projected to continue for several years.

• Paid therapy rates reached 74.5% at the end of Q2, with a goal to reach 85%; among commercially insured patients, the paid rate was 82%.

Market opportunity and future outlook

• The HAE prophylaxis market has grown from 35-40% to 75% of diagnosed patients, with expectations to reach 85%.

• ORLADEYO holds about 15-17% market share in prophylaxis, with 50% share among new prophylaxis starts.

• The product is projected to reach $1 billion in annual sales, with $800 million from the US and $200 million from global markets by 2029.

• Ex-US launches are progressing, with Western Europe nearly fully launched and expansion underway in Canada, Japan, and other regions; global commercialization is expected to double country count in two years.

• ORLADEYO has IP protection until late 2039, supporting long-term revenue stability.

Pediatric and pipeline developments

• NDA filing for pediatric use (ages 2-11) is planned for 2025, targeting about 500 US patients; oral therapy is expected to drive rapid uptake and potentially higher penetration than in adults.

• Early treatment in children is anticipated to improve quality of life and may have a halo effect, increasing adoption among family members.

• The Netherton syndrome program (BCX17725) is entering human trials by year-end, with early patient data expected next year; the opportunity is estimated at several hundred million dollars annually.

• Avoralstat, a kallikrein inhibitor, will enter DME trials in early 2025, with proof-of-concept data possible by late 2025 or early 2026.

• Complement pathway programs (oral C5, C2 inhibitors, and a bifunctional inhibitor) are advancing toward lead identification, with clinical entry possible in 2026.