Inozyme Pharma Announces Positive Interim Data for INZ-701 in Infants and Young Children with ENPP1 Deficiency and Key Program Updates

Core Insights - Inozyme Pharma announced positive interim results from its ENERGY 1 trial and Expanded Access Program (EAP) for INZ-701 in infants and young children with ENPP1 Deficiency, showing improvements in survival, heart function, and reductions in ectopic calcification and hypophosphatemia [1][4][7] - The company completed enrollment in the ENERGY 3 pivotal trial for pediatric patients with ENPP1 Deficiency and received regulatory guidance for the ASPIRE pivotal trial in children with ABCC6 Deficiency [5][6][10] Positive Interim Data from ENERGY 1 Trial and EAP - Interim data showed that 80% of infants treated with INZ-701 survived beyond their first year, compared to a historical survival rate of approximately 50% [7] - All surviving patients exhibited substantial reductions or stabilization of arterial calcifications, with some achieving complete resolution [7] - Improvements in left ventricular ejection fraction (LVEF) were noted in all surviving patients [7] - No radiographic evidence of rickets was observed in patients evaluated beyond one year of age, supported by stabilization or increases in serum phosphate levels [7] - INZ-701 demonstrated a favorable safety profile, with no serious treatment-related adverse events reported [7] Enrollment and Regulatory Progress - Enrollment in the ENERGY 3 pivotal trial is complete, with dosing expected to finish in January 2026 and topline data anticipated in early 2026 [6][9] - The trial's design includes a 2:1 randomized approach, providing over 90% power to detect meaningful differences in clinical endpoints [9] - Preliminary support from U.S. and EU regulators has been received for the ASPIRE trial, which aims to address severe complications of ABCC6 Deficiency in children [10][11] About ENPP1 and ABCC6 Deficiency - ENPP1 Deficiency is a rare disease affecting blood vessels, soft tissues, and bones, with a significant mortality rate in infants [13] - ABCC6 Deficiency similarly affects blood vessels and soft tissues, leading to severe complications in pediatric patients [14][15] - Both conditions currently lack approved therapies, highlighting a significant unmet medical need [13][15] About Inozyme Pharma - Inozyme Pharma is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for rare diseases affecting bone health and blood vessel function [16][17] - The lead candidate, INZ-701, is designed to increase levels of inorganic pyrophosphate (PPi) and adenosine, targeting multiple diseases caused by deficiencies in these molecules [17]