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Inozyme Pharma, Inc. (Nasdaq: INZY) is a clinical-stage biopharmaceutical company dedicated to developing novel therapeutics aimed at treating rare metabolic diseases. Focusing on disorders linked to paradoxical mineralization, Inozyme is pioneering solutions for conditions impacting vasculature, soft tissue, and skeletal health. Their lead drug, INZ-701, is an enzyme replacement therapy designed to correct defects in pathways involving ENPP1 and ABCC6 deficiencies.
INZ-701 showcases promise in treating diseases such as pseudoxanthoma elasticum (PXE) and generalized arterial calcification of infancy (GACI). This drug, currently in Phase 1/2 clinical trials, has demonstrated positive preliminary results, including safety, pharmacokinetic, and pharmacodynamic data, as well as trends toward clinical improvement. The trials indicate that INZ-701 increases plasma pyrophosphate (PPi) levels, a crucial marker for these conditions.
During the trials, INZ-701 was generally well tolerated with favorable safety profiles. Inozyme has undertaken comprehensive natural history studies to better understand the progression of ABCC6 Deficiency, especially in pediatric populations at high risk for severe complications like strokes.
In addition to their clinical advancements, Inozyme is committed to expanding its pipeline and therapeutic focus, planning pivotal trials for both pediatric and adult populations affected by these genetic disorders. The company also actively engages with patient communities and global health institutions to further research and raise awareness.
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