Maze Therapeutics Presents New Preclinical Data Supporting Advancement of MZE001 as a Potential Treatment for Pompe Disease

Findings Highlight Potential of Oral Substrate Reduction Therapy to Lower Disease-Causing Glycogen Build-up as a Treatment for Pompe Disease

Data Support Advancement of MZE001 into Phase 1 Clinical Trial Expected to Start in First Half of 2022

Excerpt from the Press Release:

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced new preclinical data supporting the advancement of MZE001, which aims to address Pompe disease by reducing pathologic glycogen accumulation through the inhibition of muscle glycogen synthase (GYS1). Data are being presented during two oral presentations and two poster sessions at the 18th Annual WORLD Symposium being held February 7-11, 2022.

Pompe disease is a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase (GAA), an enzyme responsible for degrading lysosomal glycogen into glucose monomers. These mutations lead to the buildup of lysosomal glycogen, primarily in skeletal muscle, respiratory muscle and cardiac muscle tissues, leading to progressive weakness and respiratory compromise.

In several in vitro and in vivo analyses with GYS1 inhibitors presented and highlighted below, data demonstrated potent inhibition of GYS1, leading to reduced accumulation of glycogen through a substrate reduction approach. Importantly, GYS1 inhibition was generally well tolerated with no on-target or off-target toxicity observed.

“Pompe disease affects both people who are living with the disease, as well as their caregivers. Whilst enzyme replacement therapy has been lifesaving, disease progression, including skeletal muscle and respiratory muscle weakness still occurs in some patients,” said Priya Sunil Kishnani, M.D., Duke Department of Pediatrics, Duke University Medical School. “I am encouraged by the preclinical data from Maze’s program targeting glycogen synthesis, which could potentially offer a new and complementary treatment option for these patients.”

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