Release Date: May 05, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript .
Positive Points
Negative Points
Q & A Highlights
Q : Can you provide insights into the potential pediatric launch for ORLADEYO and its impact on the market? A : Charles Gayer, Chief Commercial Officer, explained that there are approximately 500 pediatric HAE patients in the US, with at least 200 potentially suitable for prophylactic therapy. The introduction of an oral option like ORLADEYO could change the treatment landscape, as there is no preference for injectables among children. The expectation is that ORLADEYO will become the market leader due to its ease of administration compared to injectables.
Q : What are the expectations for the DME program's first readout, and what would be considered a successful outcome? A : Helen Thackray, Chief Research & Development Officer, stated that the focus is on safety, tolerability, and the effect on retinal thickness. The goal is to observe a reduction in retinal edema, with preclinical data suggesting a quick response. A significant reduction in edema would be a positive indicator for the program's success.
Q : How did BioCryst achieve such a significant improvement in the paid rate for ORLADEYO, and is this sustainable? A : Charles Gayer highlighted that two-thirds of the improvement was due to the Inflation Reduction Act, which helped Medicare patients afford their prescriptions. The commercial segment also saw improvements due to better preparation and real-world evidence supporting ORLADEYO's efficacy. The current paid rate is expected to be sustainable, with further incremental improvements anticipated.
Q : With profitability pulled forward, how does this impact BioCryst's investment strategy, particularly for the early-stage pipeline? A : Jon Stonehouse, CEO, noted that the programs are already fully funded to proceed as quickly as possible. The company is focused on cleaning up the balance sheet, as evidenced by the recent $75 million debt paydown, and will continue to look at similar opportunities as cash flow improves.
Q : What are the expectations for the Netherton syndrome program, and what are the key milestones? A : Helen Thackray explained that the program is designed to address the fundamental pathology of Netherton syndrome. The Phase 1 trial will assess drug levels in the epidermis and clinical impact, with initial data expected by year-end. The goal is to see normalization of the skin and reduction in itch, with potential dose escalation if necessary.
For the complete transcript of the earnings call, please refer to the full earnings call transcript .
This article first appeared on
GuruFocus
.