General Information of Drug (ID: DM2QIN1)

Drug Name
HT-100 Drug Info
Synonyms Halofunginone
Indication
Disease Entry ICD 11 Status REF
Duchenne dystrophy 8C70 Phase 2 [1]
Cross-matching ID
PubChem CID
62894
CAS Number
CAS 55837-20-2
TTD Drug ID
DM2QIN1

Molecule(s) Related to This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Bifunctional aminoacyl-tRNA synthetase (EPRS) TTXYCDZ SYEP_HUMAN Modulator [2]

The Expression Level of Molecule(s) in Normal Tissue of Major ADME-Related Organs

Molecule Molecule Type Gene Name Liver Colon Kidney Small Intestine
Bifunctional aminoacyl-tRNA synthetase (EPRS) DTT EPRS 6.311 6.223 7.295 7.226
Molecule Expression Atlas in Normal Tissue of Major ADME-related organs

The Expression Level of Molecule(s) between Disease Section and Healthy Individual Tissue

The Studied Disease Duchenne dystrophy
ICD Disease Classification 8C70
Molecule Name Molecule Type Gene Name p-value Fold-Change Z-score
Bifunctional aminoacyl-tRNA synthetase (EPRS) DTT EPRS 1.16E-05 -0.56 -1.7
Molecular Expression Atlas between Disease Section and Healthy Individual Tissue

References

1 ClinicalTrials.gov (NCT02525302) HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02.
2 Halofuginone and other febrifugine derivatives inhibit prolyl-tRNA synthetase. Nat Chem Biol. 2012 Feb 12;8(3):311-7.