Inozyme Pharma (INZY) Piper Sandler 36th Annual Healthcare Conference summary

Company focus and disease overview

• Focuses on rare diseases affecting bone health and vascular function, specifically ENPP1 deficiency, ABCC6 deficiency, and calciphylaxis, all linked to dysregulation in the PPi adenosine pathway.

• ENPP1 and ABCC6 deficiencies present similarly in infants but are distinguished by genetic testing; both have high early mortality and no approved treatments.

• Calciphylaxis affects end-stage kidney disease patients, causing painful, life-threatening skin lesions with high mortality.

Scientific and clinical insights

• PPi (inorganic pyrophosphate) inhibits unwanted calcification, while adenosine prevents vascular stenosis; both are dysregulated in target diseases.

• ENPP1 deficiency leads to severe infant mortality due to vascular calcification, shifting to bone-related symptoms in survivors.

• Current therapies are only symptomatic, with little efficacy in altering disease progression.

Pipeline and clinical development

• Lead program INZ-701 is an enzyme replacement therapy showing rapid, sustained increases in PPi and normalization of key biomarkers in ENPP1 deficiency.

• Multiple trials (ENERGY-1, 2, 3) address different age groups and phenotypes, with endpoints tailored to survival in infants and bone health in children.

• ENERGY-3 phase 3 trial uses PPi as the primary endpoint in the US and co-primary with RGI-C in Europe, with studies powered for robust regulatory submission.