BioCryst Pharmaceuticals, Inc. Q2 FY2025 earnings call

Key Points

• ORLADEYO had its best quarter since approval, with strong revenue and new patient demand. The commercial team's execution and market understanding contributed to sustainable growth.
• Signed a definitive agreement to sell the European business to Neopharmed Gentili, aiming to close in early October, which will strengthen the company's financial position, allowing paydown of term debt and increasing operating profit margin.
• Pipeline programs for Netherton syndrome and DME are on track to have data by the end of 2025.
• Strong financials: $45 million in cash generated in Q2, paid down $125 million in term debt, and expects to reach $700 million in cash by 2027, with plans to deploy capital into value-creating opportunities.

Total revenue for the second quarter was $163.4 million. ORLADEYO contributed $156.8 million, which is nearly 90% of the total revenue. ORLADEYO had 45% year-over-year revenue growth. In terms of revenue contribution, ORLADEYO was the dominant segment, driving the majority of the company's quarterly revenue.

Guidance

• Confident of reaching the upper half of the revenue guidance range of $580 million to $600 million for the full year, after excluding Q4 European revenue upon closing the sale in early October.
• Plans to provide more detailed guidance on the go-forward BioCryst business at the third quarter earnings call in November.

Risks

• Market competition: Potential new entrants in the HAE treatment space could impact ORLADEYO's market position.
• Regulatory uncertainties: Delays or issues in the approval process for pipeline programs like Netherton syndrome and DME could affect future growth.
• Dependence on ORLADEYO: Over-reliance on ORLADEYO's performance could pose risks if there are challenges with the product.

Q: Of the 45% year-over-year ORLADEYO net rev growth, how much was volume and how much was better paid rate or net price?

A: Mix of volume, gross to net improvement, more efficient paid shipments, lower discontinuations during the first half of 2025 compared to last year, and strong international results contributed to the growth.

Q: Any early indications of how June and July approvals in HAE are affecting ORLADEYO demand?

A: No significant impact seen yet, as physicians aren't waiting for new products and ORLADEYO remains differentiated with strong real-world evidence.

Q: Could you speak a little bit to persistency rates in relationship to other injectable prophylactic regimens?

A: 1-year persistence rate for ORLADEYO is statistically identical to TAKHZYRO and HAEGARDA, around 60%, with ORLADEYO numerically a bit better, showing it has the profile patients want.

Q: Any early indications of how the June and July approvals in HAE are affecting ORLADEYO demand?

A: No impact seen yet, as physicians aren't waiting for new products and ORLADEYO remains differentiated.

Q: What is the status of the pediatric PDUFA delay?

A: The FDA needed more time to review final reports, leading to a PDUFA date of December 12, and the company is on track for approval this year.

Q: What is your penetration rate into the addressable market today?

A: About 3,000 patients had tried ORLADEYO by the end of 2024, with roughly half still on therapy, and there's still significant upside growth remaining.

Q: How are you thinking about the on-demand launch of oral Ekterly and its impact on ORLADEYO?

A: Patients are excited about the all-oral combination, which could attract patients to ORLADEYO and improve patient retention, but prophy remains the backbone of therapy.

Q: How do you think about the deployment of capital and potential acquisitions?

A: The company is in a strong position to deploy capital in the rare disease space, looking to consolidate assets with high unmet needs and leveraging existing operational infrastructure.

Q: What is the typical second half push and pulls for ORLADEYO revenue?

A: Second half tends to be driven by new patients, with paid rate likely to go down slightly from the high watermark in Q2, but demand is expected to continue strong.

Q: On the pipeline programs, for Netherton what is the latest thoughts on the pivotal trial design and regulatory timeline?

A: The pivotal trial for Netherton syndrome will focus on patient-important endpoints like skin healing and itching, with a potential shorter path to registration due to unmet need.

Q: For the DME program, what is the bar for the retinal swelling reduction results expected at the end of this year?

A: The bar is to show similarity or noninferiority to existing anti-VEGF products in terms of best corrected visual acuity and change in central subfield thickness.