The drug candidate MZ-101, recently highlighted by Drug Hunter, is an oral and selective small molecule GYS1 inhibitor that decreased the accumulation of glycogen in skeletal muscle in a mouse model of Pompe disease.
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The development of MZ-101 is a significant breakthrough in inhibiting glycogen synthase 1 (GYS1). It selectively targets GYS1, reducing muscle glycogen without affecting GYS2 for liver glycogen. In mouse models of Pompe disease, MZ-101 treatment reduced glycogen accumulation in skeletal muscle as effectively as traditional enzyme replacement therapy (ERT). These results may indicate its potential for the treatment of Pompe disease and other glycogen storage disorders.

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