• Company received $49 million dollar funding round in November of 2017
• INZ-701 received Orphan Drug Designation in both US and Europe

Inozyme Pharma (Inozyme) announced on 7/17/18 that both the Food and Drug Administration’s (FDA) Office of Orphan Products Development and the European Medicines Agency’s (EMA) Committee for Orphan Medical Products (COMP) granted Orphan Drug Designation to INZ-701 for the treatment of ENPP1 Deficiency. INZ-701, the Company’s lead therapeutic candidate, is in pre-clinical development for the treatment of patients with ENPP1 Deficiency, a serious and life-threatening calcification disorder that manifests as generalized arterial calcification of infancy (GACI) in infants and as autosomal recessive hypophosphatemic rickets type 2 (ARHR2) post-infancy.

“Orphan Drug Designation, both in the United States and the European Union, is an important regulatory milestone for Inozyme as we continue our quest to develop INZ-701 for patients with rare and life-threatening calcification disorders,” said Axel Bolte, co-founder and chief executive officer of Inozyme. “The dual designations from the FDA and EMA provide crucial momentum for INZ-701, putting us in an excellent position to rapidly advance the clinical development program for this novel enzyme replacement therapy.”

INZ-701 is an enzyme replacement therapy under development with the intention to be used for the treatment of calcification disorders of the circulatory system, bones, and kidneys. In pre-clinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys and normalizing bone.

In November of 2017, Inozyme received a $49 million funding round. The financing was led by Longitude Capital, and included participation from New Enterprise Associates (NEA), Novo Ventures and Sanofi Ventures.