Maze Therapeutics Announces Completion of Phase 1 First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease

MZE001 was Well-Tolerated and Reduced Glycogen Accumulation in Blood Cells in Healthy Volunteers, Supporting Advancement into Phase 2 Clinical Trial

Excerpt from the Press Release:

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced the completion of its first-in-human clinical trial designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and food effect of MZE001 in healthy volunteers. MZE001, an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup, is being evaluated for the potential oral treatment of patients with Pompe disease.

The double-blind, placebo-controlled, single and multiple ascending dose study enrolled 112 participants. Topline data demonstrated that administration of MZE001 was well-tolerated at single and multiple doses, with a PK profile supporting twice-daily dosing. Importantly, at exposures predicted to be clinically relevant, MZE001 reduced blood cell glycogen, a translatable biomarker for muscle glycogen, in line with preclinical observations. Based on these findings, Maze plans to initiate a Phase 2 clinical trial of MZE001 in 2023. Full results of the MZE001 Phase 1 trial will be presented at the 19^th annual WORLD Symposium, being held from February 22- 26, 2023.

“Through insights generated from our genetically driven Compass Platform, we’ve designed MZE001 to inhibit GYS1 and reduce skeletal and respiratory muscle glycogen synthesis and its subsequent accumulation in patients with Pompe disease,” said Harold Bernstein, M.D., Ph.D., president, research and development and chief medical officer of Maze.

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