MZE782 is a potential first-in-class, oral, small molecule therapy targeting SLC6A19

Proof of mechanism data anticipated 2H 2025

SOUTH SAN FRANCISCO, CA, USA I October 01, 2024 I Maze Therapeutics, a clinical-stage biopharmaceutical company harnessing the power of human genetics to develop novel, small molecule precision medicines for patients living with common diseases, today announced that the first participants have been dosed in the company’s Phase 1 clinical trial evaluating MZE782 in healthy volunteers. MZE782 is a potentially first-in-class, oral, small molecule targeting the solute transporter, SLC6A19. MZE782 has the potential to initially address approximately five million of the approximately 37 million chronic kidney disease (CKD) patients in the United States who have inadequate responses to currently available CKD therapies.

“CKD is a serious and progressive disease that impacts millions of people in the United States, with standard therapies focusing on slowing disease progression, rather than targeting the underlying genetic drivers of disease,” said Harold Bernstein, M.D., Ph.D., president, R&D, and chief medical officer of Maze. “Using our Compass platform, we identified the solute transporter SLC6A19 as a promising treatment target to address a subset of patients with CKD and have designed MZE782 to phenocopy the kidney protective effects of SLC6A19 protective variants. MZE782 has the potential to be a first-in-class therapy to treat approximately five million patients that do not respond to currently available treatments. This is our third clinical program, and the initiation of this first-in-human clinical trial is an important step forward in understanding the therapeutic potential of MZE782. The trial is designed to not only evaluate its safety and tolerability, but also to provide pharmacokinetic and pharmacodynamic insights using a non-invasive biomarker intended to optimize the design of future clinical trials in patients. We expect to provide initial results of this clinical trial, including potential proof of mechanism utilizing these biomarkers, in the second half of 2025.”

The Phase 1 clinical trial is a randomized, double-blind, placebo-controlled single and multiple ascending dose study in healthy volunteers designed to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics as measured by urinary excretion of known SLC6A19 cargo, e.g., neutral amino acids.

Beyond its use as a potential standalone therapy, MZE782 may also provide a significant benefit to patients in combination with standard of care, including as a complementary treatment to current approved regimens, or as an alternative option for those patients who do not adequately respond to the current standard of care.

About Maze Therapeutics
Maze Therapeutics is a clinical-stage biopharmaceutical company harnessing the power of human genetics to develop novel, small molecule precision medicines for patients living with common diseases, with a focus on renal, cardiovascular and related metabolic, including obesity. The company is advancing a pipeline using its Compass platform, which provides insights into the genetic variants in disease and links them with the biological pathways that drive disease in specific patient groups. The company’s pipeline is led by two wholly owned lead programs, MZE829 and MZE782, each of which represents a novel precision medicine-based approach for chronic kidney disease. For more information, please visit mazetx.com, or follow us on LinkedIn and X (formerly Twitter).

SOURCE: Maze Therapeutics