BIOCRYST PHARMACEUTICALS INC Q1 FY2025 earnings call

• ORLADEYO Performance: Q1 revenue was $134 million. The U.S. commercial team made significant progress in moving patients from free drug to paid, leading to raising ORLADEYO annual revenue guidance to $580 million to $600 million (33% to 37% growth over last year).
• Pipeline Updates: Submitted pediatric NDA for ORLADEYO to FDA with filings in other regions planned. Initiated patient enrollment for BCX17725 in Netherton syndrome and avoralstat in DME. BCX17725 is designed to target KLK5, with Phase 1 trial for Netherton syndrome underway.
• Financials: Strong financial position with $145.5M Q1 revenue. Operating expenses increased but driven by commercial growth. Expect full-year profitability this year, paid down $75M debt in April.

Total revenue for the first quarter was $145.5 million. Of this, $134.2 million came from ORLADEYO. Specifically, $120.2 million (89.5%) of ORLADEYO's total revenue was from the U.S. The U.S. commercial team saw a 10 percentage point jump in the rate of paid patients, with Medicare contributing about two-thirds of this improvement.

• Raised ORLADEYO annual revenue guidance to $580 million to $600 million (33% to 37% growth over last year).
• Expect to be profitable on a full-year basis this year, accelerating cash flow and profitability goal by a year.
• Pediatric launch of ORLADEYO expected to be a tailwind, though no specific revenue guidance yet for pediatrics.

• Risks associated with clinical trials in rare diseases, including potential challenges in enrolling patients.
• Uncertainties in the commercial rollout of pediatric ORLADEYO and the DME program's clinical data readout.

Q: About pediatric HAE patients on TAKHZYRO and ORLADEYO uptake.

A: Charlie Gayer said there are about 500 pediatric patients in the U.S. on TAKHZYRO, with at least 200 appropriate for prophylactic therapy, and ORLADEYO is expected to draw patients from switching injectable therapies and new starts.

Q: On DME program data.

A: Helen Thackray said they are looking for retinal edema reduction in DME patients as part of the clinical data readout this year.

Q: On paid rate improvement.

A: Charlie Gayer explained about two-thirds of the paid rate improvement was due to Medicare via the IRA, and the team's preparation contributed to the commercial side improvement.

Q: On Netherton syndrome trial.

A: Helen Thackray discussed the Phase 1 trial design for Netherton syndrome, looking for skin outcomes like drug presence in epidermal samples and clinical impact on itch score and physician assessments.

Q: On pediatric ORLADEYO launch.

A: Charlie Gayer and Jon Stonehouse talked about quick rollout potential and uptake dynamics, noting oral formulation is likely the natural choice for pediatric prophylaxis.

Q: On Netherton preclinical tox and efficacy metrics.

A: Helen Thackray discussed preclinical toxicology supporting the drug and clinical impact metrics like skin normalization and itch reduction to determine efficacy.