Maze Therapeutics (MAZE) investor relations material

Pipeline overview and clinical progress

• Three clinical programs are in development: MZE829 (APOL1 inhibitor for AMKD), MZE782 (SLC6A19 inhibitor for PKU and CKD), all wholly owned and supported by a genetics-driven platform.

• MZE829 is in phase II (HORIZON study) for AMKD, showing a 36% average UACR reduction in 12 patients, with 62% reduction in FSGS patients.

• MZE782 is in phase II for PKU (CIPheR trial) and will start a phase II CKD trial in the first half of next year.

• HORIZON study is expanding enrollment, with data from 10-15 patients in each AMKD segment expected by end of this year or early next year.

• Adjustments to HORIZON include lowering UACR entry threshold and capping FSGS enrollment to balance patient segments.

Key clinical findings and strategy

• MZE829 demonstrated efficacy across AMKD subgroups (FSGS, diabetic, hypertensive non-diabetic), supporting broad potential.

• FSGS patients showed particularly deep responses, but the drug is expected to benefit all AMKD segments.

• Preclinical data suggest MZE829 may have a dual mechanism, potentially offering greater potency than competitors.

• Ongoing data collection will inform phase III design, with a pivotal trial targeted to start in the first half of 2027.

• Enrollment is expected to accelerate due to increased awareness and improved screening for APOL1 variants.

Regulatory and industry collaboration

• The PARASOL initiative is expected to provide recommendations on endpoints and trial design for AMKD, which will be incorporated as appropriate.

• Phase III design is under discussion, with a one-year accelerated approval endpoint as a base case, but final plans will depend on upcoming data and PARASOL guidance.

• The company is monitoring competitor trials and industry developments to inform its own strategy.