Therapy Areas: Cardiovascular
Maze Therapeutics Licenses MZE001 Pompe Disease Treatment to Sanofi
1 May 2023 - - US-based precision medicines developer Maze Therapeutics has signed an exclusive worldwide license agreement with France-based Sanofi (NASDAQ: SNY) for Maze's glycogen synthase 1 program, including clinical candidate MZE001, which is currently in development for the treatment of Pompe disease and other potential indications.

MZE001, designed and developed by Maze, is an oral GYS1 inhibitor that aims to address Pompe disease by limiting disease-causing glycogen accumulation.

Maze has advanced MZE001 through Phase 1 development, and recently reported results of its study in healthy volunteers.

In the first-in-human, double-blind, placebo-controlled, single and multiple ascending dose clinical trial, MZE001 was well-tolerated at doses up to 720 mg twice daily.

Response to MZE001 was evaluated in patients using a novel biomarker, peripheral blood mononuclear cell glycogen, and demonstrated exposure-dependent reductions in PBMC glycogen across dose levels 10 days after administration, confirming target engagement with GYS1.

These results were further confirmed in a muscle biopsy cohort, that showed equivalent reductions in muscle glycogen with MZE001.

Under the terms of the agreement, Maze will receive a USD 150m payment consisting of both upfront cash and future equity investment for the rights to further develop and commercialize MZE001, as well as an exclusive license to related GYS1-targeting back-up programs and intellectual property.

Maze will be eligible to receive up to an additional approximately USD600 m in potential development, regulatory and sales milestones, as well as meaningful royalties on sales if MZE001 is successfully commercialized.

Pompe disease is a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase, which lead to the buildup of glycogen in skeletal muscle, respiratory muscle and cardiac muscle tissues resulting in progressive weakness and respiratory compromise.

MZE001 is an investigational oral glycogen synthase inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. GYS1 is an enzyme responsible for glycogen production.

MZE001 is currently being evaluated as a potential oral treatment for patients with Pompe disease, as well as other glycogen storage disorders.

Maze Therapeutics is a biopharmaceutical with a focus on genetically informed therapies for common diseases such as chronic kidney disease.

Maze applies variant functionalization in tandem with advanced data science methods and a robust suite of research and development capabilities to advance a pipeline of novel precision medicines.

Maze has developed the Maze Compass Platform, a proprietary, purpose-built platform to understand and integrate the critical step of variant functionalization into each stage of drug development.

Utilizing the Maze Compass Platform, Maze is building a portfolio of wholly owned and partnered programs. Maze is based in South San Francisco.
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