Inozyme Pharma Inc (NASDAQ: INZY) announced interim safety, pharmacokinetic (PK), pharmacodynamic (PD), and exploratory efficacy data from its ongoing Phase 1/2 trials of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency.
Meaningful reduction of fibroblast growth factor-23 (FGF-23) observed. Most patients with ENPP1 Deficiency have elevated levels of FGF-23, which leads to increased phosphate wasting and hypophosphatemia, a key driver of osteomalacia and rickets.
Serum phosphate (Pi) levels increased over time without phosphate and active vitamin D supplementation, which were withheld from patients during the study.
Also Read: Inozyme Outlines Plans For Pediatric Trial For ENPP1 Deficiency Treatment: Targets 2026 Commercial Launch For INZ-701.
A statistically significant correlation between the increase in plasma pyrophosphate (PPi) and the decrease in FGF-23 was observed at one week post-first dose.
Upward trends were observed in bone-specific alkaline phosphatase levels from baseline, which signal biological activity in bone tissue.
Concordant improvement in GIC scores was reported by patients (P-GIC) and clinicians (C-GIC), and no patient showed a deterioration from baseline.
Subgroup analysis of patients who presented with arthritis/arthralgia at baseline showed improvement in the 6-minute walk test and increased spine bone mineral density and bone mineral content.
Data suggests clinical benefits for ENPP1 Deficiency, including improvement in key biomarkers, patient-reported outcomes, and functional outcomes.
Price Action: INZY shares are up 10.60% at $4.77 on the last check Tuesday.
