Inozyme Pharma Post Encouraging Early Data From Genetic Disorder Trial

Inozyme Pharma Inc INZY has announced preliminary biomarker, safety, and pharmacokinetic (PK) data from the first three subjects in the Phase 1 portion of its ongoing Phase 1/2 trial of INZ-701 in ABCC6 Deficiency, which presents as pseudoxanthoma elasticum (PXE) in older individuals.
PXE is a progressive disorder characterized by the accumulation of deposits of calcium and other minerals (mineralization) in elastic fibers, a component of connective tissue.
At the 0.2 mg/kg dose level of INZ-701, all three subjects showed rapid and significant increases in PPi levels. In preclinical models, plasma pyrophosphate (PPi) was a key predictive biomarker of therapeutic benefit in ABCC6 Deficiency.
Among the three subjects, the range of peak PPi levels observed during the 32-day dose evaluation period was 2139-4090 nM.
Preliminary PK and INZ-701 enzymatic activity remained consistent with data previously reported from the ongoing Phase 1/2 trial of INZ-701 in subjects with ENPP1 Deficiency.
Following the planned review of these preliminary data by a data safety monitoring board (DSMB), Inozyme expects to initiate dosing in the next dose cohort (0.6 mg/kg) in Q3. Topline data from the Phase 1/2 trial in ABCC6 Deficiency is expected in Q1 of 2023.
Price Action: INZY shares are down 13.9% at $4.69 during the market session on the last check Wednesday.