Maze Therapeutics (MAZE) Investor presentation summary

Strategic focus and pipeline overview

• Focused on developing novel small molecule precision medicines for kidney and metabolic diseases, leveraging human genetics for target validation and drug discovery.

• Lead programs include MZE829 for APOL1-mediated kidney disease (AMKD) and MZE782 for phenylketonuria (PKU) and chronic kidney disease (CKD).

• Expected cash runway extends into 2029, supported by $528 million in cash and equivalents as of March 2025.

MZE829 for APOL1-mediated kidney disease (AMKD)

• AMKD affects at least 250,000 patients in the U.S. with no approved therapies; patients experience earlier onset and rapid progression to end-stage kidney disease.

• MZE829 is a selective APOL1 inhibitor with a dual mechanism, blocking pore assembly and function to protect podocytes.

• Phase 2 HORIZON study showed a 36% mean reduction in proteinuria (uACR) across evaluable patients, with 49% reduction in non-diabetics and 62% in FSGS subset.

• MZE829 was well-tolerated with no serious adverse events; most treatment-related adverse events were mild or moderate.

• Pivotal trial initiation targeted for 1H 2027, with further data updates expected late 2026/early 2027.

MZE782 for phenylketonuria (PKU) and chronic kidney disease (CKD)

• MZE782 is an oral substrate reduction therapy designed to benefit the full spectrum of PKU patients, potentially reducing dietary burden and improving quality of life.

• Phase 1 data showed up to 42-fold increase in urinary phenylalanine excretion, supporting advancement to Phase 2 in PKU by mid-2026.

• Phase 2 PKU trial will assess safety, tolerability, and efficacy, with topline data expected in 2027.

• MZE782 also targets CKD, addressing gaps in current therapies, with Phase 2 initiation planned for 1H 2027.

• Preclinical and early clinical data support its potential to reduce proteinuria and slow CKD progression, both as monotherapy and add-on.