Maze Therapeutics announces Phase 1 trial initiation evaluating MZE001 as a potential oral treatment for Pompe Disease
New therapeutic is designed to inhibit the buildup of glycogen in patients with Pompe Disease
21 Feb 2022
Maze Therapeutics, a company translating genetic insights into new precision medicines, has announced the initiation of dosing in 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 buildup, is being evaluated for the potential oral treatment of patients with late-onset Pompe disease.
“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,” said Sarah Noonberg, M.D., Ph.D., chief medical officer of Maze. “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.”
Pompe disease is a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase (GAA), which leads to the buildup of glycogen in skeletal muscle, respiratory muscle, and cardiac muscle tissues resulting in progressive weakness and respiratory compromise. In preclinical disease models, treatment with MZE001 has demonstrated potent and selective inhibition of GYS1, leading to reduced accumulation of glycogen through a substrate reduction approach. Importantly, treatment in multiple preclinical species was generally well-tolerated with no on- or off-target toxicity observed. Maze recently presented preclinical data supporting the advancement of MZE001 at the 18th Annual WORLD Symposium.
The Phase 1 trial is a double-blind, placebo-controlled, single ascending dose and multiple ascending dose studies to assess the safety, tolerability, pharmacokinetics and pharmacodynamics, and food effect of MZE001 in healthy volunteers. In addition, target engagement pharmacodynamic biomarkers will be evaluated to provide dose-dependent proof-of-mechanism data.