Release Date: February 24, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
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.
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