MZE001 Was Well Tolerated and Reduced Glycogen Accumulation in Blood Cells in Healthy Volunteers

Phase 1 Results Support Advancement into Phase 2 Trial in Patients with Pompe Disease, Expected to Initiate in 2023

Data Presented at WORLDSymposium 2023

SOUTH SAN FRANCISCO, CA, USA I February 27, 2023 I Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced positive results from the company’s Phase 1 clinical trial of MZE001 in healthy volunteers. MZE001, an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen accumulation, is being evaluated for the potential treatment of patients with Pompe disease. MZE001 aims to address the underlying cause of disease progression through a mechanism complementary to current standard of care.

“We are highly encouraged by the clinical data for MZE001, which showcases its ability to potently and selectively inhibit GYS1, the enzyme that controls glycogen production, a key driver of Pompe disease,” said Harold Bernstein, M.D., Ph.D., president, research and development and chief medical officer of Maze. “We are pleased that these data mirror our preclinical findings and provide proof of mechanism for MZE001. While enzyme replacement therapy has brought significant benefit to patients, glycogen accumulation in muscle continues to allow the disease to progress, specifically impacting ambulation and respiratory function. By leveraging large sources of matched genetic and clinical data with our Compass platform, we were able to design MZE001 to address longstanding questions around safety and efficacy that previously precluded development of a substrate reduction therapy.”

The first-in-human, double-blind, placebo-controlled, single and multiple ascending dose clinical trial was designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and food effect of MZE001 in healthy volunteers, and enrolled 112 participants. In the trial, MZE001 was well tolerated at doses up to 720 mg twice daily. Response to MZE001 was evaluated in patients using a novel biomarker, peripheral blood mononuclear cell (PBMC) glycogen, and demonstrated exposure-dependent reductions in PBMC glycogen across dose levels 10 days after administration, confirming target engagement with GYS1. The full presentation of the findings can be found on the Maze website,

“MZE001 has the potential to be the first oral therapy for Pompe disease and is intended to be used as a monotherapy option and in combination with the current standard of care to potentially treat all patients with Pompe disease,” said Jason Coloma, Ph.D., chief executive officer of Maze. “We are committed to the efficient execution of this program so that we may reach patients in need as soon as possible and are finalizing plans to initiate our Phase 2 clinical program in patients in 2023.”

About Pompe Disease

Pompe disease is a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase (GAA), which lead to the buildup of glycogen in skeletal muscle, respiratory muscle and cardiac muscle tissues resulting in progressive weakness and respiratory compromise.

About MZE001

MZE001 is an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. GYS1 is an enzyme responsible for glycogen production. MZE001 is currently being evaluated as a potential oral treatment for patients with Pompe disease, as well as other glycogen storage disorders.

About Maze Therapeutics

Maze Therapeutics is a clinical-stage biopharmaceutical company applying advanced data science methods in tandem with a robust suite of research and development capabilities to advance a pipeline of novel precision medicines for patients with genetically defined diseases. Maze has developed the Maze CompassTM platform, a proprietary, purpose-built platform to understand and integrate the critical step of variant functionalization into each stage of genetic drug development. Maze combines human genetic data, functional genomic tools and data science technology to map novel connections between known genes and their influence on susceptibility, timing of onset and rate of disease progression. Using Compass, Maze is building a broad portfolio of wholly owned and partnered programs. Maze is based in South San Francisco. For more information, please visit, or follow us on LinkedIn and Twitter.

SOURCE: Maze Therapeutics