BIOCRYST PHARMACEUTICALS INC Q4 FY2024 earnings call

• Orladeo revenue grew 34% year-over-year in 2024, with strong momentum continuing into 2025. The commercial team's execution led to this growth.
• Clinical progress: Pediatric clinical trial data for Orladeo allows filing of NDA in 2025; BCX1775 advanced for Netherton syndrome; Avoralstat for DME to enter clinic in 2025.
• Financial performance: Achieved non-GAAP operating profit over $60 million in 2024, which was three times the planned amount, indicating strong execution.

For the full year 2024, total revenue was $450.7 million, with Orladeo contributing $437.7 million (97.1% of total revenue). In the fourth quarter of 2024, total revenue was $131.5 million, with Orladeo accounting for $124.2 million (94.4% of Q4 total revenue). Other programs like BCX1775 for Netherton syndrome and Avoralstat for DME are in development but not yet generating revenue.

• Raised Orladeo revenue guidance for 2025 to $535 million to $550 million based on Medicare patient reauthorization and demand. Total revenue guidance adjusted to $560 million to $575 million.
• Long-term goals: Aim for global revenue over $1 billion by 2029 with a 20% CAGR in revenue by 2027, and a 5% CAGR in operating expenses (excluding stock-based compensation) by 2027, aiming for positive and sustainable cash flow in the future.

• Uncertainty around future government funding for RAPIVAB.
• Reauthorization season dynamics affecting revenue recognition, including free drug distribution and gross-to-net issues.
• Regulatory and clinical trial risks for pipeline programs like BCX1775 and Avoralstat.

Q: Details on Netherton syndrome clinical data timing and positive data expectations.

A: Data from Netherton syndrome clinical trials is expected in 2024. Looking for KLK5 activity suppression in the skin and signs of skin healing. A few patients' data can indicate mechanism activity, with a potential short path to pivotal studies.

Q: Orladeo paid patients and Netherton skin healing timeline.

A: Medicare paid rate for Orladeo was around 55-56% at year-end 2024, expected to improve. Skin healing from Netherton syndrome treatment is expected in a few months due to the molecule's potency and affinity.

Q: Pediatric study tolerability, Orladeo attack rates, and guidance conviction.

A: Pediatric study of Orladeo in children with HAE showed granules are safe and well-tolerated, with early and sustained reduction in attack rates. Guidance raised due to Medicare patients moving to paid therapy and strong Q1 demand. Orladeo's attack rates are competitive with other therapies.

Q: Avoralstat TPP and Netherton patient identification.

A: Avoralstat's target product profile is an infrequent injection for diabetic macular edema. Netherton syndrome patients can be identified via the distinctive bamboo hair feature and genetic testing, with engagement with the patient community to aid identification