Shionogi & Co., LTD., and Maze Therapeutics, Inc. have agreed to develop for market, MZE001, a new substrate reduction therapy drug that could be the first ever pill take orally to treat Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More.
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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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. 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 enzymeA protein that helps the body carry out chemical reactions. More that helps make glycogenA stored form of sugar used for energy. More, potentially reducing its build-up in muscles. Maze feels that MZE001 is ready for a phase 2 clinical trialA research study that tests new treatments or approaches in people. More.
Both companies believe that MZE001 could be used on its own or with other existing treatments to better manage Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. 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.
For more information: mazetx.com/maze-therapeutics-highlights-new-preclinical-data-supporting-advancement-of-apol1-inhibitor-candidate-toward-clinical-evaluation-for-apol1-kidney-disease-2-2-2-2/