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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.
For real-time updates and detailed information, visit Inozyme Pharma or follow them on LinkedIn, X (formerly Twitter), and Facebook.
Inozyme Pharma (Nasdaq: INZY) announced that Yves Sabbagh, Ph.D., will present preliminary data from the ongoing Phase 1/2 clinical trial of INZ-701 in patients with ENPP1 Deficiency at the European Calcified Tissue Society Congress on April 17, 2023. The trial has shown positive preliminary pharmacokinetic, pharmacodynamic, and safety data, with ongoing dosing in the Phase 2 portion. The company plans to report interim data in Q3 2023 and add a new cohort for once-weekly dosing investigation.
ENPP1 Deficiency is a serious condition leading to high morbidity in infants and lacks approved therapies. The trial assesses INZ-701's safety and PK/PD profile, with exploratory endpoints focusing on calcification and functional outcomes.
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