Dosing has started for a new clinical trial investigating a potential new treatment for Pompe disease. The potential treatment, called MZE001, will first be tested in healthy volunteers.

The aim of the phase 1, double-blind, placebo-controlled, single ascending dose/multiple ascending dose and food effect study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of oral MZE001. The study is currently recruiting an estimated 80 participants, aged 18 to 50 years, at the Orange County Research Center in Tustin, California.

Participants will either receive the study drug or a placebo. The primary outcome measure of the study is the number of participants with adverse events as a measure of safety and tolerability, and the secondary outcome measure is pharmacokinetics following multiple doses of MZE001. The estimated completion date of the trial is December 30, 2022. 

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“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,” Sarah Noonberg, MD, PhD, chief medical officer of Maze and developer of MZE001 said in a press release.

MZE001 is a glycogen synthase inhibitor. It aims to limit the build-up of glycogen caused by the deficiency of the acid alpha-glucosidase enzyme in Pompe disease, which is normally responsible for breaking down glycogen.

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MZE001 has been shown to selectively inhibit glycogen synthase and reduce the accumulation of glycogen in preclinical models of Pompe disease. The potential treatment was well tolerated in several animal models, and no on- or off-target toxicities occurred. 

Currently, the only effective treatment option for Pompe disease is enzyme replacement therapy, where the body is provided with the deficient acid alpha-glucosidase enzyme. However, many patients develop an immune reaction against the enzyme, with time reducing the efficacy of this approach. MZE001 uses a substrate reduction approach, which could overcome this problem.


Maze Therapeutics announces phase 1 trial initiation evaluating MZE001 as a potential oral treatment for Pompe disease. News release. Maze Therapeutics; February 17, 2022.

A randomized phase 1 single and multiple ascending dose study of MZE001. February 21, 2022. Accessed February 22, 2022.