Shionogi & Co., Ltd., a company based in Japan, and Maze Therapeutics, Inc., from California, have signed an agreement to develop and sell a new drug called MZE001. This drug could be the first pill taken by mouth to treat Pompe disease. MZE001 is a class of drugs called substrate reduction therapies.

Shionogi now has the exclusive rights to sell MZE001 around the world. They paid Maze $150 million upfront and will pay more based on how well the development and sales of the drug develop. MZE001 works by stopping an enzyme that helps make glycogen, potentially reducing its build-up in muscles. Maze feels that MZE001 is ready for a phase 2 clinical trial.

Both companies believe that MZE001 could be used on its own or with other existing treatments to better manage Pompe disease. The U.S. Food and Drug Administration has also recognized MZE001 as an “Orphan Drug,” which means it’s considered important for treating a rare disease and gets certain benefits, like tax breaks and a period of time where no similar drugs can compete against it.

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Tagged: business, substrate reduction therapy