Inozyme Pharma (INZY) TD Cowen 45th Annual Healthcare Conference summary

Key program updates and clinical progress

• Lead asset INZ701 is a recombinant ENPP1 enzyme in development for ENPP1 and ABCC6 deficiencies, and calciphylaxis.

• Pivotal Energy 3 trial in pediatric ENPP1 deficiency is fully enrolled, with data expected in Q1 2026.

• Consistent biomarker and clinical improvements observed in adults and infants, including normalization of PPi and improved phosphate levels.

• Favorable safety profile in adults and infants, with manageable anti-drug antibodies in infants.

• Expanded access and global studies in infants, children, and adults will support regulatory submissions.

Market opportunity and patient identification

• Estimated 10,000 ENPP1 deficiency patients in North America, Brazil, Japan, and Europe.

• Over 670 clinically or genetically diagnosed patients identified, with an additional 860 identified via medical record searches.

• Ongoing efforts to increase diagnosis rates, including newborn screening in the UK and awareness campaigns in Japan.

• Rare disease pricing and market expansion could lead to blockbuster status.

• Registries and patient databases expected to grow as awareness and treatment availability increase.

Regulatory and commercialization strategy

• Energy 3 trial in children is the pivotal study for approval, supplemented by infant and adult data.

• Regulatory alignment in the US and Europe, with plans to file simultaneously in both regions.

• Pediatric study endpoints include plasma PPi change and rickets score; European regulators allow a relaxed statistical threshold.

• Long-term open-label extension planned for all pediatric participants post-52 weeks.

• Plans to expand indications to ABCC6 deficiency and calciphylaxis, with next-generation and gene therapy approaches in development.