Inozyme Pharma is speaking to the US Food and Drug Administration (FDA) about running a Phase III trial of INZ-701 in paediatric ATP binding cassette subfamily C member 6 (ABCC6) deficiency.

The trial will target a previously overlooked population of paediatric patients with ABCC6 deficiency at high risk of stroke said chief operating officer, Matt Winton PhD.

If given the green light from the FDA, the Phase III trial will likely begin in Q1 2025 and will enrol patients at sites in North America, Europe, Brazil, and Japan.

Winton told Clinical Trials Arena the company expects to be able to see benefit from INZ-701 in paediatric patients within one year to 18 months, with hopes that endpoints predictive of ischaemic stroke could provide a suitable basis for accelerated approval in the US and conditional marketing authorisation in the EU.

The company will also run a confirmatory study for two to three years for full approval of the candidate.

The company is also running a Phase III trial in paediatric ectonucleotide pyrophosphatase/phosphodiesterase-1 (ENPP1) deficiency (NCT06046820) and is initiating a Phase III trial of the candidate in infants with ENPP1 deficiency (NCT05734196).

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The company ran Phase I/II dose escalation trials of INZ-701 in adults with ABCC6 deficiency (NCT05030831) and ENPP1 deficiency (NCT04686175).

The Phase I/II trials met their endpoints with favourable safety, pharmacokinetics (PK) and pharmacodynamics (PD) as well as showing ability to raise inorganic pyrophosphate (PPi) which Winton said is a key biomarker of efficacy.

The company is also running a Phase Ib trial of the candidate in infants with ENPP1 deficiency.

INZ-701 is a novel enzyme replacement therapy (ERT) which replaces ENPP1 – an important regulator of calcification in the body.

GlobalData predicts global sales of INZ-701 to hit $591m in 2030.

GlobalData is the parent company of Clinical Trials Arena.

ABCC6 and ENPP1 deficiencies

ENPP1 and ABCC6 proteins play a prominent role in inhibiting ectopic calcification and arterial stenosis.

ABCC6 deficiency is a rare severe inherited disorder caused by mutations in the ABCC6 gene, which leads to low levels of PPi which is essential for preventing harmful soft tissue calcification and regulating bone mineralisation. There are currently no approved therapies for ABCC6 deficiency.

ENPP1 deficiency is a progressively debilitating condition of the vasculature, soft tissue, and skeleton for which there are also no approved therapies.