Maze Therapeutics (NASDAQ: MAZE) presented updated clinical data across two programs at Citigroup's Biopharma Back to School Summit on Thursday.
MZE829, the company's lead asset, is an APOL1 inhibitor targeting APOL1-mediated kidney disease and focal segmental glomerulosclerosis (FSGS). Initial Phase II HORIZON data released in March showed an average 36% reduction in urine albumin-to-creatinine ratio across 12 evaluable patients. Half met the predefined response threshold of at least 30% reduction. In the FSGS subgroup, the average reduction was 62%, with three of four patients responding. Two of five diabetic AMKD patients achieved reductions of 35% and 47% on top of background GLP-1 and SGLT2 inhibitor therapies.
Maze is enrolling toward 10–15 evaluable patients per segment across FSGS, diabetic AMKD, and non-diabetic non-FSGS AMKD. A pivotal program for MZE829 is targeted for initiation in the first half of 2027.
"What APOL1 does is, when you have the high-risk variants, it punches holes in the podocytes of the kidney," said CFO Misbah Tahir. "MZE829 not only blocks those pores or holes that form, it also disrupts the assemblies of those pores in the first place."
About 6 million Americans carry two copies of the APOL1 high-risk variant; roughly 15–20% of them develop chronic kidney disease. Maze estimates an addressable U.S. patient pool of approximately 250,000.
Vertex Pharmaceuticals is advancing a competing APOL1 inhibitor, inaxaplin. Phase II AMPLITUDE data are expected in the fourth quarter.
MZE782, a SLC6A19 inhibitor for phenylketonuria and chronic kidney disease, entered Phase II testing last month. Phase I safety and tolerability results in more than 100 healthy volunteers showed a 42-fold increase in urinary phenylalanine excretion at the 240 mg twice-daily dose. That result exceeded a competitor's benchmark increase of roughly tenfold and produced no food effect or Hartnup-like signals. The company cited those findings as reason for the accelerated Phase II initiation.
"At our 240 BID dose, we actually achieved a 42-fold increase in urinary Phe excretion. Those results exceeded our expectations and certainly resulted in us moving as quickly as possible to get MZE782 into a phase II study," Tahir said.
The Phase II CIPheR study is dividing patients into three cohorts—120 mg BID monotherapy, 240 mg BID monotherapy, and 240 mg BID combined with a BH4 agent—with top-line data expected in 2027.
Maze noted that over roughly 4½ years about 25 patients' worth of clinical data has been disclosed by the company and competitors, adding, "that's going to change dramatically in the next 12 to 16 months."
Shares were quoted at around $24.78 after declining approximately 41% year to date. Maze's market capitalization stood at about $1.37 billion. The stock has traded between $15.70 and $53.65 over the past year. Wall Street coverage carries price targets of $46 to $110 with a consensus strong-buy rating. The company reported a current ratio above 18, maintained a cash position exceeding its debt, and posted a loss per share of $2.52 over the trailing twelve months through Q2 2026.
