Details of the Drug

General Information of Drug (ID: DMCD6G1)

• Drug Name: SMT-C1100
• 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]

• Drug Type: Small molecular drug
• #Ro5 Violations (Lipinski): 0:
  Molecular Weight (mw); 337.4
  Logarithm of the Partition Coefficient (xlogp); 4.3
  Rotatable Bond Count (rotbonds); 3
  Hydrogen Bond Donor Count (hbonddonor); 0
  Hydrogen Bond Acceptor Count (hbondacc); 4
• Chemical Identifiers:
  Formula: C19H15NO3S
  IUPAC Name: 5-ethylsulfonyl-2-naphthalen-2-yl-1,3-benzoxazole
  Canonical SMILES: CCS(=O)(=O)C1=CC2=C(C=C1)OC(=N2)C3=CC4=CC=CC=C4C=C3
  InChI: InChI=1S/C19H15NO3S/c1-2-24(21,22)16-9-10-18-17(12-16)20-19(23-18)15-8-7-13-5-3-4-6-14(13)11-15/h3-12H,2H2,1H3
  InChIKey: KSGCNXAZROJSNW-UHFFFAOYSA-N
• Cross-matching ID:
  PubChem CID: 25109292
  CAS Number: 945531-77-1
  DrugBank ID: DB12888
  TTD ID: D0Y7YD

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Utrophin (UTRN)	TTNO1VA	UTRO_HUMAN	Regulator (upregulator)	[2]

Molecular Expression Atlas of This Drug

• 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

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.