Maze Therapeutics (MAZE) Corporate 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).

• Pipeline includes additional research in metabolic diseases and a partnered program (MZE001/S606001) for Pompe disease.

• Expected cash runway extends into 2029, supported by recent equity, debt, and milestone payments.

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: blocks pore assembly and function, aiming to delay disease progression.

• 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 only mild or moderate adverse events and no serious adverse events reported.

• Next steps include pivotal program initiation in 2027, with additional data updates expected in late 2026/early 2027.

MZE782 for PKU and CKD

• PKU affects at least 60,000 patients in key geographies, with limited efficacy of current therapies and significant dietary burden.

• MZE782 is an oral substrate reduction therapy designed to benefit a broad spectrum of PKU patients and potentially liberalize diet requirements.

• Phase 1 data showed MZE782 was well-tolerated, with up to 42-fold increase in urinary phenylalanine excretion, supporting advancement to Phase 2.

• For CKD, MZE782 targets SLC6A19 to reduce proteinuria and slow disease progression, with potential as monotherapy or add-on to standard of care.

• Preclinical and early clinical data support its mechanism and potential efficacy; Phase 2 trials in PKU and CKD are expected to initiate in mid to late 2026.