Maze Therapeutics reported positive topline Phase 2 data for MZE829, an oral dual-mechanism APOL1 inhibitor, in patients with broad APOL1-mediated kidney disease (AMKD). In the company’s Horizon trial, Maze said the drug delivered a clinically meaningful average reduction in urine protein levels measured by uACR. Maze reported a 35.6% mean reduction in proteinuria at week 12 and noted efficacy subgroup signals, including a 61.8% reduction in patients with focal segmental glomerulosclerosis (FSGS) and a 48.6% reduction among AMKD patients without diabetes. The company said it will continue enrollment and plans to meet regulators as it prepares a pivotal program. Despite the positive result, Maze’s shares fell sharply, reflecting investor sensitivity to the competitive landscape—particularly Vertex Pharmaceuticals’ AMKD program targeting the same pathway. Analysts compared MZE829’s magnitude to Vertex’s inaxaplin results as expectations for a clear differentiation remain high. Maze also reiterated that no approved therapies specifically target AMKD, leaving room for a first disease-specific oral option if Phase 3 validates the proteinuria signals with clinical outcomes.
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