Maze Therapeutics (MAZE) Citigroup’s Biopharma Back to School Summit 2026 summary

Company overview and strategic objectives

• Focused on developing precision medicines for kidney metabolic diseases, with three clinical programs in development, all based on human genetics and wholly owned.

• Lead program MZE829 targets APOL1-mediated kidney disease (AMKD), currently in phase II (HORIZON study), with pivotal trial initiation planned for H1 2027.

• MZE782, an SLC6A19 inhibitor, is in phase II for PKU and will enter phase II for CKD in H1 2025.

• All programs leverage the Compass discovery platform to develop small molecule therapies.

AMKD market opportunity and clinical insights

• AMKD primarily affects individuals of West African descent, with 6 million Americans carrying high-risk APOL1 variants; about 250,000 could benefit from APOL1 inhibitors.

• Disease awareness and testing are increasing, driven by ongoing clinical development and sponsor engagement.

• No approved treatments exist for AMKD, and the field is evolving with new clinical data and increased focus on genetic and environmental risk factors.

HORIZON phase II study and clinical data

• HORIZON is an open-label basket study testing MZE829 in three AMKD subtypes: FSGS, diabetic, and non-diabetic/non-FSGS.

• Initial data showed a 36% average UACR reduction across 12 patients, with 62% reduction in FSGS subgroup.

• Targeting 10–15 patients per segment for final data by end of 2024 or early 2025; pivotal trial design will be informed by these results and regulatory feedback.

• A 30% proteinuria reduction is considered clinically meaningful, though lower thresholds may be relevant for complex cases like diabetic AMKD.

• Potential for patient enrichment strategies based on responder analysis in future studies.