Maze Therapeutics has announced positive results from the Phase 1 trial of MZE782, an oral SLC6A19 inhibitor designed to treat phenylketonuria (PKU) and chronic kidney disease (CKD). The randomized, double-blind, placebo-controlled study involved 112 healthy adult volunteers and evaluated single and multiple ascending doses of MZE782.
The trial demonstrated a dose-dependent increase in urinary phenylalanine excretion, with a 39-fold increase observed after a single 960 mg dose and a 42-fold increase after a 240 mg twice-daily regimen over seven days. Additionally, MZE782 produced a dose-dependent initial dip in estimated glomerular filtration rate (eGFR), similar to effects seen with SGLT2 inhibitors, suggesting potential kidney protective benefits.
The drug was well tolerated across all doses, with no serious adverse events reported. Mild treatment-related adverse events included headache and diarrhea, which did not lead to discontinuation.
Based on these findings, Maze plans to initiate Phase 2 trials of MZE782 in both PKU and CKD in 2026. The company views these results as a significant step forward in developing targeted therapies for these conditions.
