Maze Therapeutics (MAZE) presented updated clinical data and program timelines at the H.C. Wainwright 28th Annual Global Investment Conference, highlighting Phase II results for its lead APOL1 inhibitor and encouraging biomarker activity for a second drug candidate aimed at phenylketonuria.
CFO Bhatnai told moderators that the company is building its programs independently rather than pursuing partnerships. "Go it alone is very much in the cards," he said, citing Maze's drug portfolio and its Compass platform.
MZE829, an APOL1 inhibitor designed to mimic a naturally occurring protective genetic variant, is being studied in the Phase II HORIZON trial for APOL1-mediated kidney disease. Initial data released in March 2024 showed an average urine albumin-to-creatinine ratio (UACR) reduction of 36% across all patients, with an overall response rate of 50% — defined as achieving at least a 30% UACR reduction. In the focal segmental glomerulosclerosis subgroup of four patients, the average UACR decline reached 62%, with three of four meeting the response threshold. Two patients in the diabetic kidney disease cohort — both on background SGLT2 inhibitors and GLP-1 agonists — saw UACR reductions of 35% and 47%. Bhatnai said the company plans to disclose 10 to 15 patients per segment across the HORIZON trial's three cohorts: FSGS, diabetic kidney disease, and hypertensive non-diabetic kidney disease.
The addressable market is substantial but currently unmet. Maze estimates 6 million U.S. patients carry two copies of the APOL1 high-risk variant, roughly 1 million of whom have chronic kidney disease and an estimated 250,000 could benefit from an APOL1 inhibitor. No treatments are currently approved for APOL1-mediated kidney disease.
MZE829's final major HORIZON data readout is expected by late 2024 or early 2025, with a pivotal-stage decision and program initiation planned for early 2025.
In parallel, Maze advanced its second candidate, MZE782, an inhibitor of the SLC6A19 neutral amino acid transporter. The Phase II CIPheR trial for phenylketonuria (PKU) launched last month, while a Phase II program in chronic kidney disease is slated to begin in the first half of 2025. Phase I data in more than 100 healthy volunteers showed MZE782 was well-tolerated and produced a 42-fold increase in urinary phenylalanine excretion at a 240 mg twice-daily dose, surpassing an internal target of a 10-fold increase. Top-line data from the CIPheR PKU trial is expected in 2027. PKU affects approximately 60,000 patients in key geographies, with roughly two-thirds either on a strict medical diet or receiving no treatment.
Maze's shares were referenced around $24.18 to $24.60, down roughly 51% over the trailing six months, with a market capitalization of $1.37 billion. The company reported $20 million in revenue over the past twelve months and holds a current ratio of 18.31, reflecting cash holdings that significantly exceed short-term obligations. Analyst price targets range from $46 to $110. Bhatnai described the next 12 to 18 months as a critical window during which multiple clinical catalysts will unfold and the breadth of program advancement will become clearer.
