BioCryst Pharmaceuticals (BCRX) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Company overview and current position

• Orladeyo, an oral prophylactic for HAE, is generating over $400 million in annual revenue and is on a path toward $1 billion, with a strong financial position and approaching profitability.

• The company is advancing its pipeline, including 17-725 for Netherton syndrome and avoralstat for DME, and is preparing to re-enter the clinic with new programs.

• Orladeyo is the only approved oral prophylactic for HAE, offering a unique position in a market that is shifting toward prophylaxis over on-demand treatment.

Orladeyo market dynamics and efficacy

• HAE market in the US is about 70% prophylactic, expected to rise to 80%+ as more patients prefer prevention over on-demand treatment.

• Orladeyo showed a 44% reduction in attacks in trials, with 50% of patients achieving 70%+ reduction and 20% achieving 90%+; real-world data shows up to 91% reduction over 96 weeks.

• Attack severity is reduced by 76% on Orladeyo, leading to less need for rescue medication and shorter attack duration.

• Retention rate is about 60%, similar to injectable prophylactics, with most discontinuations due to lack of efficacy rather than GI side effects.

• 52% of new users switched from other prophylactics, 32% from on-demand, and 17% are treatment-naive; market share is higher among treatment-naive patients.

Commercial strategy and physician adoption

• Physician confidence in Orladeyo is rising, with 52% of allergists/immunologists likely to prescribe it, up from 29% in early 2022/2023.

• Prescribing is balanced between large institutions and community practices, with both contributing significantly.

• Commercial efforts focus on efficacy and convenience, real-world evidence, and health economic data; plans include expanding to pediatric patients with an oral granule formulation.

• Pediatric application will be submitted in the US next year, targeting an addressable market of about 500 children, with potential for a halo effect in families.