Maze Therapeutics Shares Positive Phase I Results for MZE782 in PKU and CKD

Maze Therapeutics reported promising data from its randomised, placebo-controlled, double-blind Phase I trial evaluating MZE782, an oral SLC6A19 inhibitor, for phenylketonuria (PKU) and chronic kidney disease (CKD). The study enrolled 112 healthy adults, divided into single ascending dose (SAD), multiple ascending dose (MAD), and food-effect cohorts. SAD doses ranged from 30mg to 960mg, while MAD doses (120mg–720mg) were administered once or twice daily for one week. The trial assessed safety, tolerability, pharmacokinetics, pharmacodynamics, and the impact of meals on drug absorption. Urinary phenylalanine (Phe) and glutamine (Gln) excretion served as biomarkers of SLC6A19 inhibition, while kidney function was monitored using estimated glomerular filtration rate (eGFR).

MZE782 demonstrated good tolerability, consistent absorption, six-hour peak concentration, and an 11-hour half-life. Increased urinary Phe and Gln confirmed target engagement. An initial eGFR decline reversed after dosing stopped. Maze recently launched the Phase II HORIZON study evaluating MZE829 in APOL1 kidney disease.

16-09-2025