Maze Therapeutics to Present Phase 1 Results from First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease at the WORLDSymposium 2023
February 17, 2023SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced that the company will present data from its first-in-human clinical trial of MZE001 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, M.D., Ph.D., 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.”
Details of the presentations are as follows:
Oral Presentations
Title: Results from a first in human study of MZE001, an orally bioavailable inhibitor of glycogen synthase 1 and potential substrate reduction therapy for Pompe disease
Presenter: Julie Ullman
Date & Time: Sunday, February 26, 2023 at 9:00 a.m.
Title: Small molecule of glycogen synthase 1 restores autophagolysomal and metabolic pathway dysfunction in a mouse model of Pompe disease
Presenter: Yannan Xi
Date & Time: Saturday, February 25, 2023 at 1:00 p.m.
Poster Presentations
Title: Results from a first in human study of MZE001, an orally bioavailable inhibitor of glycogen synthase 1 and potential substrate reduction therapy for Pompe disease
Presenter: Julie Ullman
Date & Time: Saturday, February 25, 2023 at 4:00 p.m.
Title: Quantification of peripheral blood mononuclear cell (PBMC) glycogen as a novel biomarker for therapeutic intervention in Pompe disease
Presenter: Terrance Satterfield/Daniela Linzner
Date & Time: Saturday, February 25, 2023 at 4:00 p.m.
Title: Small molecule inhibition of glycogen synthase 1 restores autophagolysosomal and metabolic pathway dysfunction in a mouse model of Pompe disease
Presenter: Julie Ullman/Yannan Xi
Date & Time: Saturday, February 25, 2023 at 4:00 p.m.
About Pompe Disease
Pompe disease is a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase (GAA), which lead to the buildup of glycogen in skeletal muscle, respiratory muscle and cardiac muscle tissues resulting in progressive weakness and respiratory compromise.
About MZE001
MZE001 is an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. MZE001 is currently being evaluated for the potential oral treatment of patients with Pompe disease and potentially other glycogen storage disorders.
About Maze Therapeutics
Maze Therapeutics is a clinical-stage biopharmaceutical company applying advanced data science methods in tandem with a robust suite of research and development capabilities to advance a pipeline of novel precision medicines for patients with genetically defined diseases. Maze has developed the Maze CompassTM platform, a proprietary, purpose-built platform that combines human genetic data, functional genomic tools and data science technology to map novel connections between known genes and their influence on susceptibility, timing of onset and rate of disease progression. Using Compass, Maze is building a broad portfolio of wholly owned and partnered programs. Maze is based in South San Francisco. For more information, please visit mazetx.com, or follow us on LinkedIn and Twitter.
Contacts
Jillian Connell, Maze Therapeutics
[email protected]
650.850.5080
Media:
Dan Budwick, 1AB
[email protected]