General Information of Drug (ID: DMCD6G1)

Drug Name
SMT-C1100 Drug Info
Synonyms
BMN-195; Utrophin modulators (Duchenne muscular dystrophy); VOXC-1100; Utrophin modulators (Duchennemuscular dystrophy), VASTox; Utorphin upregulators (Duchenne muscular dystrophy), VASTox/University of Oxford; Utrophin modulators (Duchenne muscular dystrophy), Summit/BioMarin; Utropine inducer (Duchenne muscular dystrophy), Summit/BioMarin
Indication
Disease Entry ICD 11 Status REF
Duchenne dystrophy 8C70 Phase 2 [1]
Cross-matching ID
PubChem CID
25109292
CAS Number
CAS 945531-77-1
TTD Drug ID
DMCD6G1

Molecule(s) Related to This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Utrophin (UTRN) TTNO1VA UTRO_HUMAN Regulator (upregulator) [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
Utrophin (UTRN) DTT UTRN 4.194 5.236 5.213 4.459
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
Utrophin (UTRN) DTT UTRN 2.98E-03 0.72 1.68
Molecular Expression Atlas between Disease Section and Healthy Individual Tissue

References

1 Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
2 Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers. J Clin Pharmacol.2015 Jun;55(6):698-707.