Inozyme Pharma(US:INZY) Newsfilter·2024-05-23 12:30Core Viewpoint - Inozyme Pharma, Inc. is advancing its clinical-stage biopharmaceutical development with the presentation of topline data from ongoing Phase 1/2 clinical trials of INZ-701 for treating ENPP1 Deficiency and ABCC6 Deficiency at upcoming medical conferences [1] Group 1: Clinical Trials and Presentations - The European Calcified Tissue Society Congress (ECTS) 2024 will feature presentations on INZ-701's impact on bone and mineral metabolism biomarkers and clinical outcomes in adults with ENPP1 Deficiency [2][3] - The Endocrine Society's Annual Meeting (ENDO) 2024 will also include presentations on the safety and exploratory efficacy of INZ-701 in adults with ABCC6 Deficiency [3][4] Group 2: ENPP1 Deficiency Overview - ENPP1 Deficiency is a rare condition with a prevalence of approximately 1 in 64,000 pregnancies, leading to severe complications such as generalized arterial calcification of infancy and osteomalacia [4][5] - There are currently no approved therapies for ENPP1 Deficiency, highlighting the unmet medical need [4][5] Group 3: ABCC6 Deficiency Overview - ABCC6 Deficiency affects approximately 1 in 25,000 to 1 in 50,000 individuals and can lead to conditions like pseudoxanthoma elasticum, with no approved therapies available [7][8] - Pediatric patients may develop severe neurological and cardiovascular complications due to ongoing vascular calcification [7][8] Group 4: INZ-701 Development - INZ-701 is an enzyme replacement therapy designed to treat rare vascular and soft tissue disorders by metabolizing ATP to generate pyrophosphate (PPi), a natural inhibitor of mineralization [9][10] - The ongoing Phase 1/2 trials for both ENPP1 and ABCC6 Deficiencies primarily assess the safety, tolerability, and pharmacokinetic and pharmacodynamic profiles of INZ-701 [5][8]