ENERGY 3 is a Phase 3 multicenter randomized (2:1) controlled open-label clinical trial designed to evaluate the efficacy and safety of BMN 401 in children ages 1-12 with ENPP1 deficiency. Change from baseline in PPi through week 52 and RGI-C global score at week 52 are co-primary endpoints in this study. Following discussions with health authorities, RGI-C was added as a co-primary endpoint to assess clinically meaningful functional improvement in children with ENPP1 deficiency.

Key secondary endpoints include change from baseline in Rickets Severity Score (RSS), change from baseline in growth Z-score (height/body length and weight), and measurements of BMN 401 serum concentration and enzyme activity. Enrollment in ENERGY 3 was completed in January 2025, with the inclusion of 27 pediatric participants.