Acetylleucine

Acetylleucine is a modified amino acid used in the treatment of vertigo and cerebellar ataxia.

Acetylleucine

(S)-(-)-N-Acetyl-leucine
Names
IUPAC name
2-Acetamido-4-methylpentanoic acid
Other names
N-Acetylleucine; N-Acetyl-L-Leucine
Identifiers
3D model (JSmol)
3DMet
1724849 (S)-(-)
ChEBI
ChEMBL
ChemSpider
EC Number
  • Racemic: 202-734-9
985259 (S)-(-)
KEGG
MeSH acetylleucine
PubChem CID
UNII
  • InChI=1S/C8H15NO3/c1-5(2)4-7(8(11)12)9-6(3)10/h5,7H,4H2,1-3H3,(H,9,10)(H,11,12) Y
    Key: WXNXCEHXYPACJF-UHFFFAOYSA-N Y
SMILES
  • Racemic: CC(C)CC(NC(C)=O)C(O)=O
Properties
C8H15NO3
Molar mass 173.212 g·mol−1
Appearance White crystals
Melting point −115 to −113 °C; −175 to −172 °F; 158 to 160 K
log P −0.265
Acidity (pKa) 3.666
Basicity (pKb) 10.331
Pharmacology
N07CA04 (WHO)
Related compounds
Related compounds
ENU
Except where otherwise noted, data are given for materials in their standard state (at 25 °C [77 °F], 100 kPa).
Infobox references

Acetylleucine is also being developed as a possible treatment for several neurological disorders by IntraBio Inc. Clinical trials with acetylleucine for the treatment of three orphan, fatal, neurodegenerative disorders are underway: Niemann-Pick disease type C, GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases), and ataxia–telangiectasia. In 2020, IntraBio announced the successful multinational clinical trial results of the Niemann-Pick type C clinical trial. IntraBio is also investigating acetylleucine for the treatment of common inherited and acquired neurological diseases including Lewy body dementia, amyotrophic lateral sclerosis, restless legs syndrome, multiple sclerosis, and migraine Acetylleucine has received orphan drug designations from the U.S. Food & Drug Administration (FDA) and the European Commission.

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