BioCryst Pharmaceuticals' Q4 2024: Contradictions in ORLADEYO Patients, Medicare Reauthorization, and APeX-P Opportunities

These are the key contradictions discussed in BioCryst Pharmaceuticals' latest 2024 Q4 earnings call, specifically including: ORLADEYO paid patient expectations, Medicare reauthorization predictions, and APeX-P regulatory and market opportunities.



ORLADEYO Revenue and Market Momentum:
- BioCryst reported revenue of $131.5 million for Q4, with $124.2 million from ORLADEYO, contributing to full-year revenue of $450.7 million with $437.7 million from ORLADEYO.
- ORLADEYO's revenue grew by 34% year-over-year, reflecting strong market momentum supported by Medicare coverage improvements due to the Inflation Reduction Act.

Clinical Program Advancements:
- BioCryst advanced BCX17725 for Netherton syndrome into the clinic and is progressing avoralstat for DME towards clinical trials.
- These advancements are aimed at serving under-met medical needs and aligning with the company's vision to bring life-changing treatments for patients.

Pediatric Clinical Trial Success:
- The APeX-P trial with the pediatric oral granular formulation of ORLADEYO demonstrated positive results with children achieving early and sustained reduction in monthly HAE attack rates.
- This success supports the submission of an NDA, which is on track for later this year, and highlights the need for pediatric oral prophylaxis options.

International Market Expansion:
- BioCryst saw increased ex U.S. revenue contribution, with 17.2 million or 13.9% of Q4 revenue and 51.7 million or 11.8% for the full year.
- The company expects continued growth in international markets, driven by increased confidence in ORLADEYO's efficacy and broader adoption of oral prophylaxis options.