Clinical TrialsThe company presented positive 48-week long-term data from the Phase 2 HELIOS trial, showing sustained improvement in key health metrics.
Market OpportunityKOL discussions at ENDO, coupled with ASMBS survey data and Lee et al., suggest the medically important PBH population could exceed the commonly cited 167,000 patients, highlighting a significant market opportunity.
Pricing PotentialAvexitide’s orphan and breakthrough therapy designations suggest it can command premium pricing, estimated at $144,000 per patient annually.