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.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

“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.