ACEBILUSTAT

Details

Stereochemistry	ABSOLUTE
Molecular Formula	C29H27N3O4
Molecular Weight	481.5424
Optical Activity	UNSPECIFIED
Defined Stereocenters	2 / 2
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

OC(=O)C1=CC=C(CN2C[C@@H]3C[C@H]2CN3CC4=CC=C(OC5=CC=C(C=C5)C6=NC=CO6)C=C4)C=C1

InChI

InChIKey=GERJIEKMNDGSCS-DQEYMECFSA-N

InChI=1S/C29H27N3O4/c33-29(34)23-5-1-20(2-6-23)16-31-18-25-15-24(31)19-32(25)17-21-3-9-26(10-4-21)36-27-11-7-22(8-12-27)28-30-13-14-35-28/h1-14,24-25H,15-19H2,(H,33,34)/t24-,25-/m0/s1

HIDE SMILES / InChI

Molecular Formula	C29H27N3O4
Molecular Weight	481.5424
Charge	0
Count

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	2 / 2
E/Z Centers	0
Optical Activity	UNSPECIFIED

Description
Sources: https://www.ncbi.nlm.nih.gov/pubmed/27792868 | https://cysticfibrosisnewstoday.com/acebilustat/ | https://www.cff.org/Trials/Pipeline/details/108/Acebilustat-CTX-4430 | https://celtaxsys.com/pipeline/

Acebilustat (formerly CTX-4430) is an investigational oral drug under development by Celtaxsys. It is a potent inhibitor of the enzyme leukotriene A4 hydrolase (LTA4H), which catalyzes the rate‐limiting step in the formation of leukotriene B4 (LTB4), a potent chemoattractant and activator of inflammatory immune cells including neutrophils. Acebilustat is currently being tested in Phase 2 clinical trial as a modulator of inflammation in patients with cystic fibrosis (CF). The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan drug status to acebilustat as a treatment for cystic fibrosis.

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Leukotriene A4 hydrolase 4 Target ID: CHEMBL4618 Sources: https://adisinsight.springer.com/drugs/800035422 \| https://cysticfibrosisnewstoday.com/acebilustat-ctx-4430-cf-lung-inflammation/	Inhibitor 8297		64.0 nM [IC50]

PubMed

Title	Date	PubMed
Phase I Studies of Acebilustat: Biomarker Response and Safety in Patients with Cystic Fibrosis.	2017 Jan	27806191
Phase I Studies of Acebilustat: Pharmacokinetics, Pharmacodynamics, Food Effect, and CYP3A Induction.	2017 Jan	27792868
EMPIRE-CF: A phase II randomized placebo-controlled trial of once-daily, oral acebilustat in adult patients with cystic fibrosis - Study design and patient demographics.	2018 Sep	30056216

Sample Use Guides

In Vivo Use Guide

Seventeen patients with mild to moderate cystic fibrosis were enrolled and randomized into groups receiving placebo or doses of 50 mg or 100 mg acebilustat administered orally, once daily for 15 days. Sputum neutrophil counts were reduced by 65% over baseline values in patients treated with 100 mg acebilustat.

Route of Administration: Oral

In Vitro Use Guide

IC50 of acebilustat for inhibition of LTB4 production in human whole blood is 31 ng/ml, ∼0.064 uM.

Substance Class	Chemical Created by `admin` on `Sat Dec 16 10:51:01 GMT 2023` Edited by `admin` on `Sat Dec 16 10:51:01 GMT 2023`
Record UNII	J64RI4D29U
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

ACEBILUSTAT

Official Name

English

Acebilustat [WHO-DD]

Common Name

English

4-(((1S,4S)-5-((4-(4-(2-OXAZOLYL)PHENOXY)PHENYL)METHYL)-2,5-DIAZABICYCLO(2.2.1)HEPT-2-YL)METHYL)-BENZOIC ACID

Systematic Name

English

BENZOIC ACID, 4-(((1S,4S)-5-((4-(4-(2-OXAZOLYL)PHENOXY)PHENYL)METHYL)-2,5-DIAZABICYCLO(2.2.1)HEPT-2-YL)METHYL)-

Systematic Name

English

EP-501

Code

English

CTX-4430

Code

English

acebilustat [INN]

Common Name

English

Classification Tree Code System Code

EU-Orphan Drug

EU/3/14/1363