Clinical DevelopmentThe company presented positive 48-week long-term data from the Phase 2 HELIOS trial, showing sustained improvement in key health metrics.
Market OpportunityThe medically important PBH population could exceed the commonly cited 167,000 patients, indicating a larger market opportunity for avexitide.
Product PricingAvexitide’s orphan and breakthrough therapy designations suggest it can command premium pricing, estimated at $144,000 per patient annually.