" class="no-js "lang="en-US"> Maze Therapeutics Presents Potential Pompe Disease Treatment
Friday, April 12, 2024

Maze Therapeutics to Present Phase 1 Results from First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease

Maze Therapeutics, a company translating genetic insights into new precision medicines, has announced that the company will present data from its first-in-human clinical trial of MZE001 for Pompe disease in healthy volunteers at the WORLDSymposium 2023. In addition, the company will present a novel non-invasive biomarker for therapeutic intervention in Pompe disease and further preclinical work supporting the program’s advancement.

MZE001, an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen accumulation, is being evaluated for the potential treatment of patients with Pompe disease. The WORLDSymposium is being held February 22-26, 2023, in Orlando.

“We are looking forward to sharing the full data from our Phase 1 study of MZE001 at this year’s WORLDSymposium, which highlight its unique potential as a new oral substrate reduction therapy for patients living with Pompe disease,” said Harold Bernstein, president, research and development and chief medical officer of Maze.

“By leveraging our genetically driven Compass Platform, we have designed MZE001 to inhibit GYS1, an enzyme responsible for glycogen production, and address the underlying cause of disease progression through a mechanism complementary to standard of care. These exciting clinical data support the development of MZE001 as a potential monotherapy and combination therapy with standard of care, as well as our plans to advance MZE001 into a Phase 2 clinical trial in patients with Pompe disease.”

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