Inozyme Pharma Announces FDA Fast Track for INZ-701 in ABCC6 Deficiency

BOSTON, July 02, 2024 – Inozyme Pharma, Inc. (Nasdaq: INZY), a biopharmaceutical firm focused on innovative treatments for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has awarded Fast Track designation to its drug candidate INZ-701 for addressing ABCC6 Deficiency.

Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme Pharma, emphasized the significance of this development. According to Treco, the FDA's Fast Track designation highlights the potential of INZ-701 to become a crucial therapy for those suffering from ABCC6 Deficiency, especially children who face heightened risks of severe medical conditions such as stroke and critical neurological and cardiovascular diseases. Treco expressed optimism about collaborating with regulatory bodies to finalize a pivotal study for pediatric patients suffering from ABCC6 Deficiency by the end of 2024.

The FDA's Fast Track designation aims to accelerate the development and review of new drugs designed to treat or prevent serious health conditions and meet unmet medical needs. The designation for INZ-701 is based on nonclinical pharmacology data and preliminary results from an ongoing Phase 1/2 trial involving adults with ABCC6 Deficiency. The Fast Track status will enable Inozyme Pharma to have more frequent interactions with the FDA and a quicker regulatory review process.

In April 2024, Inozyme Pharma revealed positive topline data from its ongoing Phase 1/2 trial of INZ-701 in adults with ABCC6 Deficiency. The trial showed improvements in vascular pathology, visual function, and patient-reported outcomes (PROs). Initial findings from natural history studies indicate that pediatric patients with ABCC6 Deficiency suffer from a significant disease burden, including a high incidence of major clinical events such as stroke, severe neurological disease, and severe cardiovascular disease occurring early in life.

ABCC6 Deficiency is a progressively debilitating condition affecting the vasculature and soft tissues, impacting approximately 1 in 25,000 to 1 in 50,000 individuals worldwide. Infants diagnosed with the condition often suffer from generalized arterial calcification of infancy (GACI Type 2), a condition that closely resembles GACI Type 1, the infant form of ENPP1 Deficiency. Children who survive their first year are at risk of developing serious neurological and cardiovascular diseases due to continued vascular calcification and stenosis. Older individuals with ABCC6 Deficiency often develop pseudoxanthoma elasticum (PXE), characterized by pathological mineralization in blood vessels and soft tissues, affecting the skin, eyes, and vascular system. Currently, there are no approved treatments for ABCC6 Deficiency.

INZ-701 is a recombinant Fc fusion protein that acts as an ENPP1 enzyme replacement therapy, designed to treat rare disorders affecting the vasculature, soft tissue, and skeleton. Preclinical studies have shown that INZ-701 has the potential to prevent pathological mineralization and intimal proliferation, which are significant factors in severe genetic disorders like ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. INZ-701 is currently in clinical development for treating ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis.

Inozyme Pharma, Inc. is a clinical-stage biopharmaceutical company that specializes in developing novel therapies for rare diseases affecting the vasculature, soft tissue, and skeleton. The company's lead product, INZ-701, aims to address pathological mineralization and intimal proliferation, which contribute to morbidity and mortality in severe diseases. INZ-701 is undergoing clinical trials for the treatment of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis.