Maze Therapeutics has commenced participant dosing in its Phase I trial of MZE001 in a bid to advance its Pompe disease program.

MZE001 is an oral glycogen synthase (GYS1) inhibitor. To address the Pompe disease, the oral inhibitor is designed to limit the disease-causing glycogen build-up.

It is being assessed for the potential oral treatment of late-onset Pompe disease patients. Pompe disease is a rare, inherited disorder.

The placebo-controlled, double-blind, single ascending dose and multiple ascending dose Phase I trial will assess MZE001’s tolerability, pharmacodynamics, pharmacokinetics, food effect, and safety in healthy volunteers.

Additionally, the target engagement pharmacodynamic biomarkers will be assessed in the trial for providing dose-dependent proof-of-mechanism data.

Maze Therapeutics chief medical officer Sarah Noonberg said: “The initiation of this study is a significant milestone for both Maze and the Pompe community as we advance into the clinic with a potentially disease-modifying treatment for patients.

“This rapid transition to a clinical-stage company represents the dedication by our team and underscores the broad potential of our Compass platform to accelerate the drug discovery and development process.

“This study brings us a step closer in our mission to harness the power of genetic insights and translate them into precision medicines for patients with serious diseases.”

The company stated that MZE001 treatment showed selective and potent inhibition of GYS1, which led to reduced accumulation of glycogen through a substrate reduction method, in preclinical disease models.

In multiple preclinical species, the treatment was well-tolerated with no on- or off-target toxicity observed.

Recently, the company presented the preclinical data at the 18th Annual WORLD Symposium that supports the MZE001 advancement.