NCT07473973

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Inozyme Pharma · Industry
• Sex: All
• Age: 0 Years – 1 Year
• Healthy volunteers: Not accepted

Summary

The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.

Detailed description

ENPP1 Deficiency is an ultra-rare genetic disorder in which inactivating mutations in the ENPP1 gene lead to a deficiency in the ENPP1 enzyme. ENERGY 2 (Study INZ701-105) is a multicenter, single-arm, open-label Phase 3 study to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency. The study will consist of a Screening Period of up to 60 days, a Treatment Period of 52 weeks, a 52-week Extension Period, and an End of Treatment (EOT) Visit 30 days after the last dose of INZ-701.

Conditions

• Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
• Autosomal Recessive Hypophosphatemic Rickets
• Generalized Arterial Calcification of Infancy 1

Interventions

Type	Name	Description
DRUG	INZ-701	Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.

Timeline

• Start date: 2025-03-26
• Primary completion: 2028-09-11
• Completion: 2028-11-07
• First posted: 2026-03-16
• Last updated: 2026-03-18

Locations

8 sites across 8 countries: Brazil, France, Hungary, Italy, Saudi Arabia, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study