EZUTROMID

Investigational

Details

Stereochemistry	ACHIRAL
Molecular Formula	C19H15NO3S
Molecular Weight	337.392
Optical Activity	NONE
Defined Stereocenters	0 / 0
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

CCS(=O)(=O)C1=CC2=C(OC(=N2)C3=CC=C4C=CC=CC4=C3)C=C1

InChI

InChIKey=KSGCNXAZROJSNW-UHFFFAOYSA-N

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

HIDE SMILES / InChI

Molecular Formula	C19H15NO3S
Molecular Weight	337.392
Charge	0
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Description
Sources: http://adisinsight.springer.com/drugs/800022951 | https://www.ncbi.nlm.nih.gov/pubmed/21573153 | https://www.ncbi.nlm.nih.gov/pubmed/25935002 | https://www.ncbi.nlm.nih.gov/pubmed/27055247 | https://clinicaltrials.gov/ct2/show/NCT02858362

Ezutromid (SMTC-1100) is a small molecule utrophin upregulator. Ezutromid was identified from an iterative analoging approach from initial hits identified using a human muscle-specific utrophin A promoter cell-based assay. It increases both utrophin RNA and protein resulting in a significant reduction in dystrophic symptoms and increased muscle function in dystrophin-deficient mdx mice ( a mouse model of Duchenne muscular dystrophy (DMD)). Ezutromid was deemed safe and well tolerated in a Phase 1a healthy volunteer study and successfully completed a Phase 1b study in DMD boys. Summit Therapeutics is developing Ezutromid for the treatment of Duchenne muscular dystrophy.

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Utrophin 1 Target ID: P46939 Gene ID: 7402.0 Gene Symbol: UTRN Target Organism: Homo sapiens (Human) Sources: https://www.ncbi.nlm.nih.gov/pubmed/21573153	Activator 1447

Conditions

Condition	Modality	Targets	Highest Phase	Product
Duchenne muscular dystrophy 18 Sources: http://adisinsight.springer.com/drugs/800022951 https://www.ncbi.nlm.nih.gov/pubmed/25935002	Primary		Phase II 1147	Unknown Approved Use Unknown

C_max

Value	Dose	Analyte	Population
122 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/25651188	200 mg/kg 2 times / day multiple, oral dose: 200 mg/kg route of administration: Oral experiment type: MULTIPLE co-administered:	EZUTROMID plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: HIGH-FAT
531 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/25651188	400 mg/kg single, oral dose: 400 mg/kg route of administration: Oral experiment type: SINGLE co-administered:	EZUTROMID plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: HIGH-FAT
80.6 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30650257	2500 mg single, oral dose: 2500 mg route of administration: Oral experiment type: SINGLE co-administered:	EZUTROMID plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: FED
65.7 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30650257	2500 mg 2 times / day multiple, oral dose: 2500 mg route of administration: Oral experiment type: MULTIPLE co-administered:	EZUTROMID plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED

AUC

Value	Dose	Analyte	Population
830 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/25651188	200 mg/kg 2 times / day multiple, oral dose: 200 mg/kg route of administration: Oral experiment type: MULTIPLE co-administered:	EZUTROMID plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: HIGH-FAT
4215 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/25651188	400 mg/kg single, oral dose: 400 mg/kg route of administration: Oral experiment type: SINGLE co-administered:	EZUTROMID plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: HIGH-FAT
672 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30650257	2500 mg single, oral dose: 2500 mg route of administration: Oral experiment type: SINGLE co-administered:	EZUTROMID plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: FED
551 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30650257	2500 mg 2 times / day multiple, oral dose: 2500 mg route of administration: Oral experiment type: MULTIPLE co-administered:	EZUTROMID plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED

T_1/2

Value	Dose	Analyte	Population
29 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/25651188	200 mg/kg 2 times / day multiple, oral dose: 200 mg/kg route of administration: Oral experiment type: MULTIPLE co-administered:	EZUTROMID plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: HIGH-FAT
9.8 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/25651188	400 mg/kg single, oral dose: 400 mg/kg route of administration: Oral experiment type: SINGLE co-administered:	EZUTROMID plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: HIGH-FAT
6.53 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30650257	2500 mg single, oral dose: 2500 mg route of administration: Oral experiment type: SINGLE co-administered:	EZUTROMID plasma	Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: FED

Doses

Dose	Population	Adverse events
400 mg/kg single, oral Highest studied dose Dose: 400 mg/kg Route: oral Route: single Dose: 400 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/25651188	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M Food Status: HIGH-FAT Sources: https://pubmed.ncbi.nlm.nih.gov/25651188	Sources: https://pubmed.ncbi.nlm.nih.gov/25651188
200 mg/kg 2 times / day multiple, oral Studied dose Dose: 200 mg/kg, 2 times / day Route: oral Route: multiple Dose: 200 mg/kg, 2 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/25651188	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M Food Status: HIGH-FAT Sources: https://pubmed.ncbi.nlm.nih.gov/25651188	Sources: https://pubmed.ncbi.nlm.nih.gov/25651188
2500 mg 2 times / day multiple, oral Studied dose Dose: 2500 mg, 2 times / day Route: oral Route: multiple Dose: 2500 mg, 2 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/30650257	unhealthy, CHILD Health Status: unhealthy Age Group: CHILD Sex: M+F Food Status: FED Sources: https://pubmed.ncbi.nlm.nih.gov/30650257	Sources: https://pubmed.ncbi.nlm.nih.gov/30650257

Sourcing

Vendor/Aggregator	ID	URL
1PlusChem LLC	1P01E9ZO	https://www.1pchem.com/search?query=1P01E9ZO
AChemBlock	M15367	http://www.achemblock.com/catalogsearch/result/?q=M15367
AstaTech	F20702	http://astatechinc.com/CPSResult.php?CRNO=F20702
Axon MedChem	2481	https://www.axonmedchem.com/product/2481
BLD Pharm	BD631052	http://www.bldpharm.com/search/Search.html?keyword=BD631052
Cayman Chemical	20309	http://www.caymanchem.com/app/template/Product.vm/catalog/20309
Chem Scene	CS-5710	http://www.chemscene.com/goproductList/searchProductList?productObj=CS-5710
ChemShuttle	186162	https://www.chemshuttle.com/catalogsearch/result/?q=186162
eMolecules	64114891	https://orderbb.emolecules.com/search/#?query=64114891
Excenen	EX-A2333	http://www.excenen.com/searchresultlist.php?id=EX-A2333
mcule	MCULE-2097183913	https://mcule.com/MCULE-2097183913/
MedChem Express	HY-17614	https://www.medchemexpress.com/search.html?q=HY-17614&ft=&fa=&fp=
Molnova	M16772	https://www.molnova.com/en/serch-list.html?keywords=M16772
MolPort	MolPort-042-665-876	https://www.molport.com/shop/molecule-link/MolPort-042-665-876
MuseChem	I005626	https://www.musechem.com/product/I005626.html
ShangHai Biochempartner	BCP18328	http://www.biochempartner.com/search_BCP18328
TargetMol	T3424	https://www.targetmol.com/search?keyword=T3424

PubMed

Title	Date	PubMed
Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy.	2016	27055247
Second-generation compound for the modulation of utrophin in the therapy of DMD.	2015-08-01	25935002
Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers.	2015-06	25651188
Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse.	2011-05-06	21573153

Patents

Sample Use Guides

In Vivo Use Guide

A multicenter, Phase 1 study (NCT02056808) enrolled 12 pediatric patients with Duchenne muscular dystrophy, divided equally into three groups (A-C). Group A were given 50 mg/kg on Days 1 and 11, and 50 mg/kg bid on Days 2 to 10. Group B and C received 100 mg/kg on Days 1 and 11; Group B and Group C were given 100 mg/kg bid and 100 mg/kg tid, respectively, on Days 2 to 10.

Route of Administration: Oral

In Vitro Use Guide

In vitro dosing of human myoblasts with Ezutromid (SMT C1100) leads to a 25% increase in utrophin mRNA when compared to vehicle-only dosing after three days of treatment. Treatment of human DMD cells with SMT C1100 lead to a 2-fold increase in utrophin protein levels at an optimal concentration of 0.3 µM after 3 days of treatment.

Substance Class	Chemical Created by `admin` on `Wed Apr 02 09:37:05 GMT 2025` Edited by `admin` on `Wed Apr 02 09:37:05 GMT 2025`
Record UNII	645R07LF0D
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

EZUTROMID

Official Name

English

ezutromid [INN]

Preferred Name

English

SMT-C1100

Code

English

BMN195

Code

English

5-(ETHYLSULFONYL)-2-(NAPHTHALEN-2-YL)BENZO(D)OXAZOLE

Systematic Name

English

Ezutromid [WHO-DD]

Common Name

English

5-(ETHANESULFONYL)-2-(NAPHTHALEN-2-YL)-1,3-BENZOXAZOLE

Systematic Name

English

SMTC-1100

Code

English

SMT C1100

Code

English

BENZOXAZOLE, 5-(ETHYLSULFONYL)-2-(2-NAPHTHALENYL)-

Systematic Name

English

BMN-195

Code

English

Classification Tree Code System Code

FDA ORPHAN DRUG

351111