Inozyme Pharma (INZY) 2024 Wells Fargo Healthcare Conference summary

Company overview and asset focus

• Clinical-stage rare disease company targeting disorders of mineralization via the PPi-Adenosine pathway.

• Lead asset INZ-701 is an enzyme replacement therapy developed to address ENPP1 deficiency and expanded to ABCC6 deficiency and calciphylaxis.

• Proof of concept shown in animal models and humans for all three indications.

Patient identification and market opportunity

• Genetic prevalence of ENPP1 deficiency is estimated at 1 in 64,000 pregnancies, with over 1,000 patients identified globally.

• Patient identification efforts include global outreach, partnerships, and a new registry with GACI Global.

• Estimated 10,000 patients in key markets, with potential for expansion to symptomatic heterozygotes and related disorders.

• Newborn screening initiatives in the UK include ENPP1 and ABCC6 on the panel.

Clinical trial progress and data highlights

• Adult ENPP1 study showed INZ-701 is safe, well-tolerated, and rapidly increases PPi to normal levels, with improvements in bone biomarkers and six-minute walk test.

• ENERGY-3 pivotal pediatric trial is open-label, randomized, with PPi as the primary endpoint in the US and co-primary with RGI-C in the EU; data expected in the second half of 2025.

• ENERGY-1 infant study focuses on safety, dose selection, and early efficacy signals, with high mortality highlighting unmet need.