Maze Therapeutics (MAZE) investor relations material

Pipeline overview and upcoming catalysts

• Three clinical programs are in development: MZE829 for APOL1-mediated kidney disease (AMKD), MZE782 for PKU, and MZE782 for chronic kidney disease (CKD), all supported by a genetics-driven platform.

• MZE829 is in phase II (HORIZON study) with final data expected end of this year or early next; a pivotal program is planned for the first half of next year.

• MZE782 for PKU has just started a phase II trial (CIPheR), with top-line data expected in 2027.

• MZE782 for CKD is set to begin phase II in the first half of next year.

• All programs are small molecules with full rights retained.

HORIZON study insights and AMKD landscape

• Initial HORIZON data showed a 36% average uACR reduction and a 50% response rate across AMKD subgroups, with FSGS patients showing a 62% reduction.

• The study includes FSGS, diabetic, and hypertensive non-diabetic AMKD patients, aiming for 10–15 patients per subgroup in the final data.

• The 30% uACR reduction is considered a meaningful clinical benchmark, though lower reductions may be significant in diabetic cohorts.

• HORIZON design was intentionally broad to address the full spectrum of AMKD, reflecting biological rationale and unmet need.

• Recent protocol tweaks lowered the uACR enrollment threshold and capped FSGS enrollment to balance subgroups.

Competitive landscape and regulatory considerations

• There is significant unmet need in AMKD, with an estimated 250,000 U.S. patients potentially benefiting from APOL1 inhibitors.

• The field is awaiting recommendations from the PARSOL Shield initiative, which may define surrogate endpoints and accelerate development.

• The company is monitoring competitor trials (AMPLITUDE, Vertex) and expects a surge in clinical data in the next 12–18 months.

• Feedback from KOLs suggests a preference for broad pivotal trials with subgroup analyses rather than separate cohorts.

• A go-it-alone commercialization strategy is being considered, but partnerships remain an option.