Maze Therapeutics (MAZE) Goldman Sachs 47th Annual Global Healthcare Conference 2026 summary

Lead asset updates and clinical data

• MZE829 demonstrated strong safety, tolerability, and efficacy in the phase II HORIZON study, with up to 62% UACR reduction in FSGS patients and nearly 50% in non-diabetic cohorts, exceeding benchmarks from literature.

• Early signals of efficacy were observed in diabetic patients, with about 40% showing response despite prior treatments, supporting further data collection in this subgroup.

• Pivotal studies for MZE829 in non-diabetic patients are planned to start in the first half of 2027, with full HORIZON readout expected by end of 2026 or 2027.

• MZE782 phase I data showed safety, linear PK, and strong pharmacodynamic effects, with over 40-fold increase in urinary phenylalanine excretion, suggesting best-in-class potential for PKU.

• Phase II proof-of-concept study for MZE782 is underway, including monotherapy and combination with BH4, with data expected in 2027.

Competitive landscape and differentiation

• No approved therapies exist for APOL1 kidney disease; MZE829’s dual mechanism (blocking and disrupting APOL1 pore assembly) offers potency and differentiation.

• MZE829 achieved higher UACR reductions in heavily pre-treated patients compared to other agents, positioning it as potentially best-in-class.

• MZE782 is three to four times more potent than other oral substrate reducers in PKU, aiming to help more patients reach clinically meaningful plasma Phe reductions and potentially discontinue restrictive diets.

Regulatory and clinical development strategy

• Proteinuria reduction is a recognized approvable endpoint for FSGS; ongoing studies and the Parasol initiative may clarify regulatory paths for APOL1 and non-FSGS populations.

• For PKU, plasma phenylalanine reduction is the approvable endpoint; MZE782’s mechanism does not rely on residual enzyme activity, potentially broadening its applicability.

• CKD program leverages genetic and preclinical data showing renal protection via SLC6A19 inhibition, with proof-of-concept studies planned to start in 2027.