BioCryst Pharmaceuticals (BCRX) 24th Annual Needham Virtual Healthcare Conference summary

Business performance and product growth

• ORLADEYO sales exceeded $437 million last year, with over 36% year-over-year growth and a trajectory toward $1 billion peak sales by 2029, supported by strong IP protection through 2035 and 2039.

• Revenue acceleration was driven by increased physician and patient experience, more new prescriptions, and improved patient access to paid therapy, with overall paid rates expected to approach 80% after Medicare co-pay changes.

• Guidance for 2025 was raised to $535–$550 million due to strong demand and the positive impact of the Inflation Reduction Act on Medicare patient affordability.

• Retention rates remain high, with over 60% of patients staying on ORLADEYO for at least 12 months, and real-world evidence supports competitive efficacy and retention compared to other therapies.

• Ex-U.S. commercialization is expanding, with ORLADEYO available in over 30 countries and similar growth trends, though pricing is lower outside the U.S.

Market dynamics and competitive landscape

• ORLADEYO is now the de facto prophylactic treatment for newly diagnosed patients, with 52% of new starts switching from other prophylactic products and 32% from acute-only therapy.

• The company expects to maintain a 25% market share even as new oral competitors enter, supported by annual market simulations and strong patient/physician preference.

• New oral therapies are seen as beneficial for the market, potentially increasing adoption of all-oral HAE treatments.

• Messaging and targeting strategies have remained consistent, focusing on convenience, efficacy, and building real-world evidence to reinforce confidence among healthcare providers and patients.

• The commercial team has continuously refined processes, particularly in converting free drug to paid therapy, contributing to launch success.

Pipeline and R&D updates

• Two clinical programs are advancing, with patient data expected by year-end; the Netherton syndrome program aims to deliver a targeted, subcutaneous therapy with potential for functional cure and streamlined registration.

• The Netherton syndrome trial is in phase one, enrolling both healthy volunteers and patients, with interim data showing promising safety and efficacy; pivotal endpoints are being evaluated for future trials.

• Pediatric formulation of ORLADEYO has completed interim analysis, showing strong safety and efficacy, with plans to file for registration this year and target the under-12 patient segment.

• Avoralstat is entering clinical trials for diabetic macular edema, leveraging its safety profile and unique delivery method to address retinal vascular leakage.