- Data from a handful of patients in the phase 1 portion of a phase 1/2 trial showed that Inozyme Pharma's ( NASDAQ: INZY ) INZ-701 led to positive biomarker, safety, and pharmacokinetic results in adults with ABCC6 Deficiency .
- All three adults showed rapid and significant increases in levels of pyrophosphate (PPi), a calcification inhibitor, after receiving the drug at a dose of 0.2 mg/kg. The company said in preclinical studies, PPi was predictive biomarker of therapeutic benefit.
- There were no serious adverse events reported. A grade 1 event was reported, though it was not related to INZ-701.
- Following review by a data safety monitoring board, InoZyme ( INZY ) plans to begin dosing in the next 0.6 mg/kg cohort this quarter and report topline data from Phase 1/2 trial in Q1 2023.
- Read about Inozyme's ( INZY ) recent partnership with the Rady Children’s Institute for Genomic Medicine .
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Inozyme Pharma releases positive early-stage data on candidate for ABCC6 Deficiency