Drug General Information
Drug ID
D0U3KP
Former ID
DIB013178
Drug Name
HT-100
Synonyms
Halofunginone
Drug Type
Small molecular drug
Indication Duchenne dystrophy [ICD-11: 8C70; ICD-10: G71.0] Phase 2 [1]
Company
Halo therapeutics
Structure
Download
2D MOL

3D MOL

Formula
C16H18Br2ClN3O3
Canonical SMILES
C1CC(C(NC1)CC(=O)CN2C=NC3=CC(=C(C=C3C2=O)Cl)Br)O.Br
InChI
1S/C16H17BrClN3O3.BrH/c17-11-6-13-10(5-12(11)18)16(24)21(8-20-13)7-9(22)4-14-15(23)2-1-3-19-14;/h5-6,8,14-15,19,23H,1-4,7H2;1H
InChIKey
SJUWEPZBTXEUMU-UHFFFAOYSA-N
PubChem Compound ID
PubChem Substance ID
Target and Pathway
Target(s) Bifunctional aminoacyl-tRNA synthetase (EPRS) Target Info Modulator [2]
BioCyc TRNA charging
KEGG Pathway Porphyrin and chlorophyll metabolism
Aminoacyl-tRNA biosynthesis
Metabolic pathways
Panther Pathway Heme biosynthesis
Pathwhiz Pathway Arginine and Proline Metabolism
WikiPathways Parkinsons Disease Pathway
tRNA Aminoacylation
References
REF 1 ClinicalTrials.gov (NCT02525302) HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02.
REF 2 Halofuginone and other febrifugine derivatives inhibit prolyl-tRNA synthetase. Nat Chem Biol. 2012 Feb 12;8(3):311-7.

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