Inozyme Raises $73 Million to Advance Pipeline Targeting Rare Diseases of Abnormal Mineralization

Rare Daily Staff

Inozyme Pharma, a clinical-stage rare disease company developing novel therapeutics for the treatment of abnormal mineralization, said it priced an underwritten offering of 16.3 million shares of its common stock at a price of $3.69 per share and, to certain investors in lieu of common stock, pre-funded warrants to purchase 3.5 million shares of common stock at a price of $3.6899 per pre-funded warrant, for aggregate gross proceeds of approximately $73 million, before deducting underwriting discounts and commissions and other offering expenses.

Inozyme Pharma is pursuing treatments to address the underlying causes of diseases of abnormal mineralization that impact the vasculature, soft tissue, and skeleton. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. The company’s lead candidate INZ-701 is an enzyme replacement therapy in phase 1/2 development for the treatment of the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

Genetic mutations resulting in the deficiency of the enzyme ENPP1 lead to severe disorders of calcification, including Generalized Arterial Calcification of Infancy (GACI) and autosomal recessive hypophosphatemic rickets type 2.

Mutations in the human ABCC6/MRP6 gene is a known cause of pseudoxanthoma elasticum, a heritable recessive disorder characterized by calcification of elastin fibers in connective tissue, including the heart, vasculature, skin, and eyes.

In preclinical studies, INZ-701 has shown potential to generate plasma pyrophosphate and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time normalizing bone mineralization.