6 March 2023 - - US-based Maze Therapeutics, a company translating genetic insights into new precision medicines, has posted positive results from the company's Phase 1 clinical trial of MZE001 in healthy volunteers, the company said.

MZE001, an oral glycogen synthase 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.

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 glycogen, and demonstrated exposure-dependent reductions in PBMC glycogen across dose levels 10 days after administration, confirming target engagement with GYS1.

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

MZE001 is an oral glycogen synthase 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.

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 Compass 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.