Pipeline DiversificationAMX0114 represents a novel antisense approach in ALS, with early safety data expected to validate its mechanism and diversify Amylyx's pipeline.
Potential Market OpportunityEven with conservative patient estimates, the total market opportunity for a new therapy for PBH could easily exceed several billion dollars.
Regulatory DesignationsWith Breakthrough Therapy and Orphan designations, avexitide is positioned for premium pricing and rapid uptake if approved.