BioCryst Pharmaceuticals (BCRX) Barclays 27th Annual Global Healthcare Conference summary

Financial and commercial outlook

• Achieved operating profit last year and expects to be EPS profitable by year-end, with full-year EPS profitability next year and expanding profits in subsequent years.

• ORLADEYO, the lead product for hereditary angioedema, is projected to generate $535–$550 million in revenue this year, with peak global sales targeted at $1 billion.

• To reach $800 million in US peak sales by 2029, the plan includes adding 200 net new patients annually, increasing the paid patient rate to 85%, and implementing modest price increases.

• Real-world data from over 800 patients show strong efficacy and long-term benefits, supporting confidence in continued commercial success despite upcoming competitors.

• The company is positioned to balance profitability with advancing its pipeline, aiming for a second commercially successful product.

Pipeline and R&D updates

• Two pipeline programs will enter the clinic this year, with initial patient data expected by year-end.

• BCX17725 targets KLK5 for Netherton syndrome, aiming to restore protein function and improve skin healing; preclinical knockout models support the approach.

• Phase 1 for BCX17725 will include both healthy volunteers and patients, focusing on safety, pharmacokinetics, and early clinical outcomes like skin healing within weeks.

• Data from a handful of patients could demonstrate efficacy quickly, with a bundled data update planned for late 2024; pivotal trial plans will follow regulatory interactions.

• Dosing frequency for BCX17725 is expected to be every one to two weeks, with adjustments based on early patient outcomes.

DME program and scientific rationale

• Avoralstat, previously unsuccessful as an oral HAE therapy due to poor solubility, is being repurposed for diabetic macular edema (DME) via suprachoroidal injection.

• Animal data show sustained retinal exposure and efficacy in reducing vascular leakage for up to 180 days, suggesting potential for once or twice yearly dosing.

• Plasma kallikrein inhibition by avoralstat demonstrated comparable reduction in retinal leakage to VEGF inhibitors in preclinical models.

• The DME program will assess changes in retinal edema and visual acuity after a single dose in patients, with outcomes tracked over several months.