Inozyme reports topline data from ongoing Phase 1/2 trials of INZ-701

Inozyme Pharma announced positive topline pharmacokinetic, pharmacodynamic, and safety data from the ongoing Phase 1/2 clinical trials of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency. The Company also reported encouraging patient reported outcome data as measured by global impression of change in the ENPP1 Deficiency trial. Phase 1/2 Clinical Trial in Adults with ENPP1 Deficiency: Nine patients were initially enrolled in the ongoing Phase 1/2 clinical trial across three dose cohorts of INZ-701. Pharmacodynamic Data: Rapid, significant, and sustained increase in PPi was observed in all patients, with a target PPi threshold observed from the lowest dose of 0.2 mg/kg. Pharmacokinetic Data: INZ-701 activity in a dose proportional manner was observed. Long half-life of approximately 126 hours and drug accumulation as shown by a greater than dose proportional exposure suggests the potential for once weekly dosing. Safety Data: INZ-701 was generally well-tolerated and exhibited a favorable safety profile, with no serious or severe adverse events attributed to INZ-701 and no adverse events leading to study withdrawal. 3/9 patients experienced mild adverse events related to INZ-701. Emerging Exploratory Clinical Data: 6/8 patients showed concordant improvements on overall health on C-GIC and P-GIC; 5/8 patients showed "much improved" (+2) or "very much improved" (+3) on P-GIC; No patients showed a deterioration in overall health from baseline on C-GIC or P-GIC. Phase 1/2 Clinical Trial in Adults with ABCC6 Deficiency: Nine patients were initially enrolled in the ongoing Phase 1/2 trial across three dose cohorts of INZ-701. Pharmacodynamic Data: Rapid and significant increase in PPi was observed in all cohorts with a dose response observed. PPi showed sustained increase in the highest dose cohort to levels comparable to those observed in a study of healthy subjects, which showed PPi levels between 1002 nM and 2169 nM. Pharmacokinetic Data: INZ-701 activity in a greater than dose proportional manner was observed. Long half-life of approximately 126 hours. Safety Data: INZ-701 was generally well-tolerated and exhibited a favorable safety profile, with no serious or severe adverse events. All adverse events were mild to moderate in severity.