Inozyme presents new data at ASBMR 2024 annual meeting

Inozyme Pharma is presenting four posters at the American Society for Bone and Mineral Research, ASBMR, 2024 Annual Meeting, including new data that demonstrate the progression and impact of rare bone and blood vessel diseases in children. Key findings of the retrospective analysis included: In the 84 patients with ENPP1 Deficiency, cardiovascular complications and arterial calcifications developed early in life, predominately within the first few months. A striking 88% of patients experienced ectopic calcifications, with 81% showing arterial calcification and 66.7% showing aortic calcification. Cardiovascular complications, including hypertension, heart valve defects and heart failure, were noted in 76% of patients. Among the 84-patient cohort, 51 were diagnosed with Generalized Arterial Calcification of Infancy alone and only 19 survived beyond infancy; 22 were diagnosed with GACI, survived beyond infancy and progressed to Autosomal Recessive Hypophosphatemic Rickets Type 2; and 11 patients presented initially with ARHR2. Musculoskeletal complications, including phosphate-wasting rickets, bowed extremities, and pain, were documented in all patients diagnosed with ARHR2, with 45% of ENPP1 Deficiency survivors showing symptoms by age 3 and 75% by age 11. The data highlight that 95% of surviving ENPP1 Deficiency patients are expected to experience cardiovascular, musculoskeletal, and other organ complications, underscoring the lifelong burden of the disease.