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

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

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.

Participants must meet all of the following:

Inclusion Criteria:

1. Infant aged ≤ 1 year at the time of enrollment.
2. Confirmed diagnosis of ENPP1 deficiency, based on genetic testing.
3. Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement).
4. Medically stable to participate in a 52-week treatment study.
5. Written informed consent provided by a parent or legal guardian.

Exclusion Criteria

Participants will not be eligible if any of the following apply:

1. Receiving end-of-life or hospice care.
2. Prior treatment with INZ-701, unless received through an approved expanded access program.
3. Concurrent participation in another interventional clinical trial.
4. Planned major surgery during the study period that would interfere with study participation.