- Inozyme Pharma Inc (NASDAQ:INZY) announced preliminary biomarker, safety, and pharmacokinetic (PK) data from the first three patients treated in the Phase 1 portion of its Phase 1/2 trial of INZ-701 in ENPP1 Deficiency.
- ENPP1 Deficiency manifests generalized arterial calcification of infancy (GACI), characterized by extensive vascular calcification and overgrowth of smooth muscle cells inside blood vessels.
- At the 0.2 mg/kg dose level of INZ-701, all three patients showed rapid, significant, and sustained increases in PPi levels, a key predictive biomarker of therapeutic benefit in ENPP1 Deficiency.
- The range of PPi levels across three patients at the screening was 132-333 nM.
- The range of PPi levels measured six hours after the first dose was 581-1239 nM, an approximately 4-fold mean increase from screening across the three patients.
- The mean PPi level across the three patients during the 32-day dose evaluation period was 1356 nM, an approximately 5-fold increase from screening.
- INZ-701 was generally well-tolerated, with no serious adverse events reported, and otherwise exhibited a favorable initial safety profile.
- Dosing is underway at the 0.6 mg/kg dose level of INZ-701 in the second cohort of the trial.
- Inozyme plans to report topline data from the ongoing trial in 2H of 2022.
- Price Action: INZY shares are up 14.90% at $4.33 during the market session on the last check Monday.
