Inozyme Pharma Post Encouraging Early Data From Genetic Disorder Trial

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.

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