Sanofi to license Pompe treatment from Maze

2023-05-01

临床1期引进/卖出基因疗法上市批准临床结果

Sanofi has signed a worldwide licensing deal with San Francisco-based Maze Therapeutics for Maze’s glycogen synthase 1 (GYS1) program, including its lead clinical candidate currently in development for the treatment of Pompe disease.

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 in a study of healthy volunteers. The drug is also being evaluated as a potential oral treatment for other glycogen storage disorders.

Under the terms of the deal, Maze will receive $150 million in 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 also be eligible for an additional $600 million in potential milestones.

For Sanofi, the deal adds another rare disease med to its pipeline. The drugmaker has two marketed enzyme replacement therapies in the Pompe space — Myozyme, approved in 2006 and its newer drug Nexviazyme, approved in 2021.

Although enzyme replacement therapies are currently the standard of care for Pompe, new gene therapies have the potential to offer more treatment options. Amicus' gene therapy, AT-GAA, is designed to treat the inherited muscle disorder by delivering a recombinant version of an enzyme that is usually deficient in Pompe patients. The therapy's review was pushed by the FDA last fall after ongoing COVID travel restrictions kept the agency from being able to carry out its facility inspections. The agency's decision is now expected around the third quarter of 2023.

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