Positive Topline Data for Inozyme Pharma

Clinical-stage rare disease biopharmaceutical company, Inozyme Pharma (Nasdaq: INZY) has reported positive topline data from its Phase ½ clinical trial evaluating INZ-701 as a potential treatment for ENPP1 Deficiency and ABCC6 Deficiency.

ENPP1 Deficiency is a progressive condition with no approved therapies. Approximately 50% of infants with ENPP1 Deficiency die within six months of birth. ABCC6 Deficiency is a severe, progressive rare disorder that leads to low levels of plasma pyrophosphate (PPi), which is essential for preventing harmful soft tissue calcification and regulating bone mineralization.

Highlights

• Inozyme’s clinical studies showed rapid, significant, and sustained increase in PPi.
• Encouraging patient-reported outcome data in all dose cohorts.
• INZ-701 was generally well-tolerated and exhibited a favorable safety profile in both trials.

Inozyme expects to achieve multiple key milestones in 2023, including the initiation of a pivotal trial of INZ-701 for pediatric patients with ENPP1 Deficiency.

For video

Inozyme Pharma (Nasdaq: INZY) has reported positive topline data from its Phase ½ clinical trial evaluating INZ-701 as a potential treatment for ENPP1 Deficiency and ABCC6 Deficiency.

The company expects to initiate a pivotal trial of INZ-701 for pediatric patients with ENPP1 Deficiency in 2023.