Maze Therapeutics has recently completed a phase 1 first-in-human trial of MZE001, an oral glycogen synthase (GYS1) inhibitor being developed to treat Pompe disease, according to a press release.

The double-blind, placebo-controlled, single- and multiple-ascending dose study aimed to assess the safety, pharmacokinetics, and pharmacodynamics as well as the food effect of MZE001 in more than 100 healthy volunteers.

The study’s primary outcome measure was the number of participants that experienced adverse effects. Secondary outcome measures included maximum concentration following multiple doses of MZE001, the area under the curve following multiple doses, and the accumulation ratio following multiple doses.

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Participants were aged less than 55 years and were healthy, according to previous medical records, physical examination, and laboratory studies. All patients were able to conduct a treadmill test without cardiac abnormalities. Patients with a history of cancer, cardiac disease, kidney disease, hepatic disease, alcoholism, a smoking habit, or drug hypersensitivity were excluded from the study.

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Results demonstrated that MZE001 was safe and well tolerated at both single and multiple doses. The pharmacokinetic profile of the drug suggested that twice daily dosing was the optimal scheme.

Regarding efficacy, the trial showed that MZE001 was capable of reducing blood cell glycogen levels. Based on results from the phase 1 trial, the company plans on moving forward with a phase 2 trial in the near future.

“Accumulation in these important muscle groups has proven particularly resistant to treatment with intravenous enzyme replacement therapy, the current standard of care,” said Harold Bernstein, MD, PhD, president of research and development and chief medical officer of Maze. “These topline results give us confidence that GYS1 may be safely inhibited, supporting MZE001’s advancement as the potential first oral therapy, alone or in combination with enzyme replacement, to treat Pompe disease and possibly other glycogen storage disorders.”


Maze Therapeutics announces completion of phase 1 first-in-human trial evaluating MZE001 as a potential oral treatment for Pompe disease. News release. Maze Therapeutics; December 15, 2022.