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Title: Olesoxime  
Author: World Heritage Encyclopedia
Language: English
Subject: Pharmacology
Publisher: World Heritage Encyclopedia


Systematic (IUPAC) name
Clinical data
Legal status
CAS number
ATC code ?
Chemical data
Formula C27H45NO 
Mol. mass 399.65

Olesoxime (TRO19622) is the lead compound of Trophos' proprietary cholesterol-oxime compound family of mitochondrial pore modulators.[1][2]

It is a molecule that has a cholesterol-like structure and displays strong neuroprotective properties. TRO19622 is as effective as a cocktail of three neurotrophic factors in keeping motor neurons alive in culture.[3]

Preclinical studies have demonstrated that the compound promotes the function and survival of neurons and other cell types under disease-relevant stress conditions through interactions with the mitochondrial permeability transition pore (mPTP).[3]

A 2009–2011 phase 3 clinical trial of olesoxime in amyotrophic lateral sclerosis did not demonstrate a significant increase in survival versus placebo.[4]

The results of a 2011–2013 combined phase 2/3 trial of olesoxime in spinal muscular atrophy (SMA) indicated that the compound prevents deterioration of muscle function, suggesting that olesoxime might become the first drug approved to treat SMA.[5]


  1. ^ Martin LJ (August 2010). "Olesoxime, a cholesterol-like neuroprotectant for the potential treatment of amyotrophic lateral sclerosis". IDrugs 13 (8): 568–80.  
  2. ^ "Olesoxime". New Drugs Online Report. UK Medicines Information. 
  3. ^ a b Bordet T, Buisson B, Michaud M, Drouot C, Galéa P, Delaage P, Akentieva NP, Evers AS, Covey DF, Ostuni MA, Lacapère JJ, Massaad C, Schumacher M, Steidl EM, Maux D, Delaage M, Henderson CE, Pruss RM (2007). "Identification and Characterization of Cholest-4-en-3-one, Oxime (TRO19622), a Novel Drug Candidate for Amyotrophic Lateral Sclerosis". Journal of Pharmacology and Experimental Therapeutics 322 (2): 709–720.  
  4. ^ "Trophos announces results of phase 3 study of olesoxime in Amyotrophic Lateral Sclerosis". Press Release. Trophos. 2011-12-13. 
  5. ^ "Trophos announces top-line results of pivotal trial of olesoxime in spinal muscular atrophy". Press Release. Trophos. 2014-03-10. 

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