Maze Therapeutics, Inc. (NASDAQ:MAZE) on Thursday shared clinical results from the Phase 1 healthy volunteer study of MZE782, an oral small molecule targeting the solute transporter, SLC6A19.
MZE782 has the potential to be a best-in-class therapy for patients with phenylketonuria (PKU), an inherited metabolic disorder, and a first-in-class therapy for chronic kidney disease (CKD).
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Pharmacokinetics (PK)
MZE782 demonstrated a favorable plasma PK profile after single and multiple oral doses.
Oral administration was associated with consistent absorption, with a tmax of six hours and a half-life of 11 hours.
Exposure increased proportionally with dose, and steady-state was achieved by Day 3, supporting once- or twice-daily dosing regimen to be evaluated in Phase 2.
Neutral Amino Acid Excretion
MZE782 produced dose-dependent increases in 24-hour urinary excretion of the neutral amino acids Phe and Gln across both SAD and MAD cohorts, confirming target engagement and SLC6A19 inhibition.
A 39-fold increase in urinary Phe excretion over 24 hours was observed with a single dose of 960 mg of MZE782. A 42-fold increase in urinary Phe excretion over 24 hours on Day 7 was observed in the 240 mg twice-daily dose cohort.
A 55-fold increase in urinary Gln excretion over 24 hours was observed with a single dose of 960 mg of MZE782. A 68-fold increase in urinary Gln excretion over 24 hours on Day 7 was observed in the 240 mg twice-daily dose cohort.
Estimated Glomerular Filtration Rate (eGFR)
MZE782 demonstrated a dose-dependent initial eGFR dip over seven days of dosing that was similar in magnitude to what has been observed in patients initiating SGLT2 and RAAS inhibitors.
Next Steps
Maze plans to initiate two Phase 2 proof-of-concept trials of MZE782, evaluating plasma Phe reduction in PKU and proteinuria reduction in CKD in 2026.
MZE782 was well tolerated across all doses in all cohorts. There were no serious adverse events, no severe adverse events, and no treatment-related adverse events leading to discontinuation.
Maze Therapeutics announced an oversubscribed private placement of approximately $150 million.
The private placement will be for 4 million shares at $16.25 per share.
MAZE Price Action: Maze Therapeutics shares were up 52.15% at $24.37 at the time of publication on Thursday. The stock is trading at a new 52-week high, according to Benzinga Pro data.
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