Inozyme Pharma Reports Encouraging Preliminary Results from Phase 1/2 INZ-701 Studies in Adults with PXE and ENPP1 Deficiencies

Inozyme Pharma, Inc., a clinical-stage biopharmaceutical company working on novel treatments for rare diseases associated with pathological mineralization and intimal proliferation, recently reported promising data from Phase 1/2 clinical trials of their drug INZ-701. This drug is intended for the treatment of ABCC6 Deficiency and ENPP1 Deficiency, conditions marked by abnormal calcium accumulation in the body.

The trials have shown that INZ-701 is safe and generates positive immunogenic responses. Patients participating in these studies displayed clinical improvements in areas such as cardiovascular health and visual function, along with overall positive patient-reported outcomes.

ABCC6 Deficiency, especially in early-onset forms, commonly leads to critical complications like stroke. The ongoing trials involving INZ-701 have included participants across various dosage groups and have continually monitored effects such as the stabilization of vascular pathology and improvements in visual function, even suggesting that such treatments could be extended to pediatric patients at risk of severe outcomes.

Additionally, data from patients with ENPP1 Deficiency suggest that weekly doses of INZ-701 can effectively maintain beneficial blood markers that correlate with disease progression. These findings support the potential of INZ-701 in regular clinical use and encourage further trials.

Inozyme Pharma plans to further explore INZ-701's applications, particularly aiming to initiate pivotal trials in pediatric patients with ABCC6 Deficiency by early 2025. This decision follows both natural history studies and responses observed in ongoing trials, highlighting the urgent need for effective treatments in preventing severe complications such as stroke and cardiovascular disease in young patients.

The company also shared insights on the safety and efficacy of different dosing schedules, confirming the general tolerance and sustained benefits observed in longer-term treatments involving home administration of the drug.

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