Inozyme Pharma is focused on developing treatments for rare and debilitating metabolic diseases, with an initial focus on disorders of pyrophosphate deficiency.
Pyrophosphate (PPi) is an important regulator of calcification in the body. Calcification is a complex process that requires both phosphate (PO4-3), and calcium (Ca+2). While phosphate is necessary for this process, PPi is a potent anti-mineralization factor that inhibits formation of potential disease-causing calcium deposits. A proper ratio of phosphate/PPi is maintained in healthy tissue to avoid over/under calcification. Consequently, abnormal PPi levels are associated with a variety of pathologies affecting bones, arteries, and other organs.
In certain rare, life-threatening and devastating genetic disorders such as Generalized Arterial Calcification of Infancy (GACI), Autosomal Recessive Hypophosphatemic Rickets type 2 (ARHR2), Pseudoxanthoma Elasticum (PXE) and Chronic Kidney Disease (CKD), PPi levels are below the normal physiological levels. This can lead to either over mineralization of soft tissues and/or under mineralization of bone.
Inozyme Pharma is developing an enzyme replacement therapy (INZ-701) designed to treat calcification disorders of the circulatory system, bone and kidney. In pre-clinical studies, the company’s experimental therapy has shown potential to generate plasma (PPi), and restore it to physiological levels and thereby correcting the underlying cause of these serious diseases for which there are no approved therapies.
Inozyme Pharma is preparing to test the safety and effectiveness of INZ-701 in infants with GACI. Given the broad therapeutic applicability of the program, the company may embark on testing the drug in other chronic diseases.
For links to relevant scientific abstracts/publications, please see below (subscriptions may be required).
New perspectives on rare connective tissue calcifying diseases
Can biological calcification occur in the presence of pyrophosphate?
Pyrophosphate: a key inhibitor of mineralization.
ENPP1-Fc prevents mortality and vascular calcifications in rodent model of generalized arterial calcification of infancy