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

Company overview and disease focus

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

• ENPP1 and ABCC6 deficiencies are genetic, while calciphylaxis is associated with end-stage kidney disease.

• ENPP1 deficiency presents with severe infant mortality and progressive bone and vascular complications throughout life.

• Diagnosis relies on genetic testing, with no approved treatments currently available; management is symptomatic.

Pipeline and clinical development

• Lead program is in pivotal studies, with final phase 3 data expected by the end of next year; two additional programs are close behind.

• INZ-701, an ENPP1 enzyme replacement therapy, has shown rapid and sustained increases in plasma PPi and normalization of key biomarkers in adults.

• Multiple ENERGY trials target different age groups due to varying disease phenotypes and endpoints, aiming for a broad regulatory label.

• 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 on the secondary endpoint for regulatory robustness.

Regulatory strategy and trial design

• FDA accepted PPi as a primary endpoint for pediatric trials, while EMA requires a clinical endpoint as co-primary; both agencies have aligned on similar study designs.

• Infant trial (ENERGY-2) is ex-US due to FDA's requirement for a placebo-controlled design, considered unethical by advocacy groups and academic centers.

• Data from ENERGY-1, ENERGY-2, and pediatric/adult studies will be combined for regulatory submissions.