INZ-701 enzyme replacement therapy is used to treat ENPP1 deficiency. Credit: Africa Studio/Shutterstock.com.

Inozyme Pharma has dosed the first infant in the Phase Ib ENERGY-1 clinical trial of INZ-701 enzyme replacement therapy to treat ENPP1 deficiency.

The open-label, single-arm study is designed to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), tolerability, and safety of the therapy in infants with ENPP1 deficiency.

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It intends to enrol up to eight infants aged between one and 12 months from various sites in Europe and the US.

Infants will be administered INZ-701 subcutaneously for 52 weeks. In an extension period beyond 52 weeks, patients may also receive INZ-701 dosages.

Doses of the therapy range from 0.2mg/kg once weekly to 0.6mg/kg twice a week, and may further increase based on the PK/PD and safety data of the INZ-701 study.

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Evaluation of exploratory biomarkers, cardiac function, functional performance, development, survival, growth, and plasma pyrophosphate (PPi) levels are additional outcome measures of the study.

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Inozyme Pharma senior vice-president and chief medical officer Kurt Gunter said: “Initiation of the ENERGY-1 trial in infants is an important milestone, as we continue to advance INZ-701 with the goal of improving the lives of patients with ENPP1 deficiency across all age groups.

“We are committed to a global programme to identify and treat all newborns with this condition.”

INZ-701 is a recombinant Fc fusion protein and is in development for treating rare disorders of the skeleton, soft tissue, and vasculature.

It is currently being evaluated in Phase I/II clinical trials to treat ABCC6 deficiency, along with ENPP1 deficiency.