Maze Therapeutics disclosed that its oral, dual‑mechanism APOL1 inhibitor, MZE829, achieved a 35.6% mean reduction in urinary albumin‑to‑creatinine ratio (uACR) at week 12 in the Phase 2 HORIZON trial. The basket‑design study enrolled 15 patients, with 12 evaluable for efficacy; 50% of those patients experienced a reduction of over 30% in uACR, a threshold associated with a 10‑year delay in progression to end‑stage kidney disease.
The drug’s performance varied across subgroups. In the focal segmental glomerulosclerosis (FSGS) cohort, MZE829 produced a 61.8% mean uACR reduction, while in non‑diabetic APOL1‑mediated kidney disease (AMKD) patients the mean reduction was 48.6%. No serious adverse events were reported; headache and diarrhea were the most common treatment‑related events.
Maze’s financial position supports continued development. As of December 31, 2025, the company held $360.0 million in cash, cash equivalents, and marketable securities, providing a runway into 2028 despite an EBITDA of –$132 million in the last twelve months. The company plans to advance MZE829 into a pivotal program and to initiate two Phase 2 trials of MZE782 in phenylketonuria and chronic kidney disease.
Management emphasized the significance of the data. "We are pleased to show initial promising proof‑of‑concept for MZE829, an oral precision medicine that was designed to treat the underlying cause of AMKD by uniquely inhibiting both pore formation and channel function in the podocyte," said Harold Bernstein, M.D., Ph.D., President of R&D and Chief Medical Officer. "Based on the data shown today, as well as genetics data derived through our Compass platform, we believe that MZE829's dual mechanism approach has the potential to address the unmet need in AMKD patients. We look forward to meeting with regulators and key scientific leaders to align on a pivotal program in patients with AMKD, and anticipate presenting HORIZON data at a future medical conference," he added. CEO Jason Coloma, Ph.D., noted, "We are proud of the progress that Maze achieved in 2025 and have already carried our strong record of execution into the new year, as evidenced by today's positive topline data from our Phase 2 HORIZON trial of MZE829 in broad AMKD."
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