MZ-101   Click here for help

GtoPdb Ligand ID: 13115

Synonyms: MZ101
Compound class: Synthetic organic
Comment: MZ-101 is a preclinical, small molecule, selective inhibitor of glycogen synthase 1 (GYS1; P13807) [1]. It was designed as a strategy for the treatment of glycogen storage disorders such as Pompe disease, via a mechanism that reduces glycogen synthesis and storage in muscle. Inhibition of GYS1 is proposed as an alternative/or adjunct to alpha glucosidase (GAA) enzyme replacement therapy for Pompe disease.
2D Structure
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Physico-chemical Properties
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Hydrogen bond acceptors 7
Hydrogen bond donors 2
Rotatable bonds 9
Topological polar surface area 86.16
Molecular weight 449.52
XLogP 2.91
No. Lipinski's rules broken 0
SMILES / InChI / InChIKey
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Canonical SMILES CC(C)C1=CC=C(C=C1)[C@H](C2=CC=CC=C2)NC(=O)[C@@H]3C[C@H](CN3C(=O)CC4=CN=NN4)F
Isomeric SMILES CC(C)C1=CC=C(C=C1)[C@@H](NC(=O)[C@@H]2C[C@@H](F)CN2C(=O)CC3=CN=NN3)C4=CC=CC=C4
InChI InChI=1S/C25H28FN5O2/c1-16(2)17-8-10-19(11-9-17)24(18-6-4-3-5-7-18)28-25(33)22-12-20(26)15-31(22)23(32)13-21-14-27-30-29-21/h3-11,14,16,20,22,24H,12-13,15H2,1-2H3,(H,28,33)(H,27,29,30)/t20-,22+,24+/m1/s1
InChI Key VIXMWFGMNPKXHI-SFLYRZDNSA-N
References
1. Ullman JC, Mellem KT, Xi Y, Ramanan V, Merritt H, Choy R, Gujral T, Young LEA, Blake K, Tep S et al.. (2024)
Small-molecule inhibition of glycogen synthase 1 for the treatment of Pompe disease and other glycogen storage disorders.
Sci Transl Med, 16 (730): eadf1691. [PMID:38232139]