Details
Stereochemistry | ACHIRAL |
Molecular Formula | C31H42N2O3 |
Molecular Weight | 490.6768 |
Optical Activity | NONE |
Defined Stereocenters | 0 / 0 |
E/Z Centers | 6 |
Charge | 0 |
SHOW SMILES / InChI
SMILES
CC\C=C/C\C=C/C\C=C/C\C=C/C\C=C/C\C=C/CCC(=O)NCCNC(=O)C1=CC=CC=C1O
InChI
InChIKey=JQLBBYLGWHUHRW-KUBAVDMBSA-N
InChI=1S/C31H42N2O3/c1-2-3-4-5-6-7-8-9-10-11-12-13-14-15-16-17-18-19-20-25-30(35)32-26-27-33-31(36)28-23-21-22-24-29(28)34/h3-4,6-7,9-10,12-13,15-16,18-19,21-24,34H,2,5,8,11,14,17,20,25-27H2,1H3,(H,32,35)(H,33,36)/b4-3-,7-6-,10-9-,13-12-,16-15-,19-18-
CAT-1004 (Edasalonexent)is an orally administered small molecule designed to inhibit NF-κB, which is activated from infancy in Duchenne muscular dystrophy and is central to causing muscle damage and preventing muscle regeneration. Structurally CAT-1004 represents covalently links salicylic acid and docosahexaenoic acid -- two compounds known to inhibit NF-κB. . In a Phase 1 study in adults, NF-κB activity in peripheral mononuclear cells was inhibited following a single dose of edasalonexent but not by equimolar doses of salicylic acid and docosahexaenoic acid. Chronic activation of NF-κB is a key driver of muscle degeneration and suppression of muscle regeneration in Duchenne muscular dystrophy, which occurs early in the disease process and precedes loss of muscle function. Salicylic acid prevents NF-κB mediated muscle atrophy and decreases protein catabolism in muscle. Docosahexaenoic acid has been shown to upregulate anti-inflammatory pathways and suppress pro-inflammatory pathways via modulation of NF-κB activity. Edasalonexent is endocytosed and hydrolyzed by intracellular fatty acid amide hydrolase (FAAH) to release salicylic acid and DHA in the intracellular compartment, thus having a potential advantage of selectively delivering higher doses in target muscle cells where FAAH is abundant.
Approval Year
Sample Use Guides
In Vivo Use Guide
Sources: https://www.ncbi.nlm.nih.gov/pubmed/30452422
33, 67 and 100 mg/kg/day
Route of Administration:
Oral
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EU-Orphan Drug |
EU/3/15/1560
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FDA ORPHAN DRUG |
453714
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44626120
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C171665
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100000178258
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10223
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1204317-86-1
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AF3Z6434KS
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DB15010
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JK-104
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ACTIVE MOIETY