• ORLADEYO Revenue Growth: 34% year-over-year increase for 2024.
• Total Revenue for Q4 2024: $131.5 million.
• ORLADEYO Revenue for Q4 2024: $124.2 million.
• Total Revenue for Full Year 2024: $450.7 million.
• ORLADEYO Revenue for Full Year 2024: $437.7 million.
• Non-GAAP Operating Profit for 2024: Over $60 million.
• Operating Expenses for Q4 2024: Approximately $115 million (excluding non-cash stock compensation).
• Operating Expenses for Full Year 2024: $388 million (excluding non-cash stock compensation).
• Cash at Year-End 2024: Approximately $343 million.
• Net Cash Utilization for Q4 2024: $8.4 million.
• 2025 ORLADEYO Revenue Guidance: Raised to $535 million to $550 million.
• 2025 Total Revenue Guidance: Adjusted to $560 million to $575 million.

• Warning! GuruFocus has detected 7 Warning Signs with BCRX.

Release Date: February 24, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) reported a 34% year-over-year growth in ORLADEYO revenue for 2024, indicating strong market performance.
• The company achieved a non-GAAP operating profit of over $60 million, which was three times larger than planned, showcasing effective financial management.
• BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) is advancing its pipeline with promising clinical trials for BCX17725 for Netherton Syndrome and Avoralstat for Diabetic Macular Edema.
• The company is on track to file an NDA for ORLADEYO for pediatric use, expanding its market potential to children aged 2 to 11.
• BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) raised its ORLADEYO revenue guidance for 2025 to $535 million to $550 million, reflecting strong demand and favorable impacts from the Inflation Reduction Act for Medicare patients.

Negative Points

• The company faces uncertainty around future government funding for RAPIVAB, which could impact revenue streams.
• There is a potential headwind from foreign exchange rates, particularly affecting revenue from the European Union.
• BioCryst Pharmaceuticals Inc (NASDAQ:BCRX) anticipates flat to slightly down revenue in Q1 2025 due to the reauthorization season and gross-to-net impacts.
• The company acknowledges that the Netherton Syndrome market is underdiagnosed, which could pose challenges in patient identification and market penetration.
• Despite the positive outlook, the company remains cautious about the variability in ex-US demand and the impact of distributor shipment timing on quarterly results.

Q & A Highlights

Q: Can you provide more details on the expected timeline for Netherton Syndrome data and what constitutes positive data? A: Helen Thackray, Chief Research & Development Officer: We expect to have data this year. For Netherton Syndrome, we only need data from a small number of patients to confirm the mechanism. We're looking for the drug's effect on KLK5 activity in the skin, leading to healing. If successful, we could move quickly into pivotal studies, potentially with a single pivotal trial supported by this year's data.

Q: What is the current status of ORLADEYO's paid patient proportion, especially within Medicare? A: Charles Gayer, Chief Commercial Officer: We ended last year with 73.5% paid across our patient base. For Medicare, it was lower, around 55-56%. We expect this to improve significantly due to the Inflation Reduction Act, potentially reaching 80% by the end of the quarter.

Q: How does the pediatric formulation of ORLADEYO compare in terms of tolerability and regulatory expectations? A: Helen Thackray, Chief Research & Development Officer: The pediatric oral granules show good tolerability and similar efficacy to adults. Regulatory approval will follow a pediatric extrapolation approach, matching safety and exposure to extend labeling from adults to children. We are on track with the FDA and EMA for this process.

Q: What are the expectations for ORLADEYO's international demand in 2025, considering the Q4 uptick? A: Charles Gayer, Chief Commercial Officer: We see growing confidence in ORLADEYO internationally, with transformative confidence in markets like the UK and early confidence in Italy. Despite FX headwinds, we expect continued growth, although the percentage of ex-US revenue might be slightly deflated due to strong US growth.

Q: How is BioCryst addressing the underdiagnosis of Netherton Syndrome? A: Charles Gayer, Chief Commercial Officer: We are expanding our efforts to identify patients, focusing on distinctive features like bamboo hair. We plan to educate the market and engage with the patient community to improve diagnosis and awareness as we move into clinical trials.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.