U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Details

Stereochemistry ABSOLUTE
Molecular Formula C27H45NO
Molecular Weight 399.6523
Optical Activity UNSPECIFIED
Defined Stereocenters 7 / 7
E/Z Centers 1
Charge 0

SHOW SMILES / InChI
Structure of OLESOXIME, E-

SMILES

[H][C@@]1(CC[C@@]2([H])[C@]3([H])CCC4=C\C(CC[C@]4(C)[C@@]3([H])CC[C@]12C)=N\O)[C@H](C)CCCC(C)C

InChI

InChIKey=QNTASHOAVRSLMD-SIWSWZRQSA-N
InChI=1S/C27H45NO/c1-18(2)7-6-8-19(3)23-11-12-24-22-10-9-20-17-21(28-29)13-15-26(20,4)25(22)14-16-27(23,24)5/h17-19,22-25,29H,6-16H2,1-5H3/b28-21+/t19-,22+,23-,24+,25+,26+,27-/m1/s1

HIDE SMILES / InChI

Molecular Formula C27H45NO
Molecular Weight 399.6523
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 7 / 7
E/Z Centers 1
Optical Activity UNSPECIFIED

Olesoxime (TRO19622) a small-molecule with a cholesterol-like structure has remarkable neuroprotective properties for motor neurons in cell culture and in rodents. The biopharmaceutical company Trophos initially developed this compound. This medicine is in phase II clinical trial in treating spinal muscular atrophy and in phase I for patients with stable relapsing remitting multiple sclerosis. This drug was also investigated in phase III clinical trial for amyotrophic lateral sclerosis, but it did not demonstrate a significant increase in survival versus placebo and that study was discontinued. Preclinical studies have demonstrated that the olesoxime promoted the function and survival of neurons and other cell types under disease-relevant stress conditions through interactions with the mitochondrial permeability transition pore (mPTP).

CNS Activity

Curator's Comment: Known to be CNS penetrant in mouse, rat. Human data not available

Approval Year

Targets

Targets

Primary TargetPharmacologyConditionPotency
Target ID: mitochondrial permeability transition pore
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Unknown

Approved Use

Unknown
Primary
Unknown

Approved Use

Unknown
Primary
Unknown

Approved Use

Unknown
PubMed

PubMed

TitleDatePubMed
Olesoxime (cholest-4-en-3-one, oxime): analgesic and neuroprotective effects in a rat model of painful peripheral neuropathy produced by the chemotherapeutic agent, paclitaxel.
2009 Dec 15
Patents

Patents

Sample Use Guides

Participants will receive 10 milligrams per kilogram (mg/kg) suspension once a day either orally or via a naso-gastric or gastrostomy tube with the main meal (preferably at the same time of the day)
Route of Administration: Oral
Neuroprotective dose-effect of olesoxime against motor neuron cell death was studied. Rat embryonic motor neurons were cultured in 96-well plates in the absence of trophic factors and with increasing concentrations of olesoxime (concentrations ranging from 0.1 to 10 µM), or with pure syn or anti forms of olesoxime. Motor neuron survival was measured after 3 days or 7 days in vitro by direct counting of live cells labeled with calcein-AM. Survival is expressed as a ratio of surviving cell (A-B) relative to positive controls treated with a cocktail of neurotrophic factors (NTFs) and negative controls that received DMSO alone (0.1%). Treatment with olesoxime produced a dose-dependent increase in cell survival with EC50 around 3 µM. Importantly, both syn and anti forms were equally potent.
Substance Class Chemical
Created
by admin
on Sat Dec 16 08:50:01 GMT 2023
Edited
by admin
on Sat Dec 16 08:50:01 GMT 2023
Record UNII
8V8EF6094N
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
OLESOXIME, E-
Common Name English
Code System Code Type Description
PUBCHEM
9930827
Created by admin on Sat Dec 16 08:50:01 GMT 2023 , Edited by admin on Sat Dec 16 08:50:01 GMT 2023
PRIMARY
CAS
66538-08-7
Created by admin on Sat Dec 16 08:50:01 GMT 2023 , Edited by admin on Sat Dec 16 08:50:01 GMT 2023
PRIMARY
FDA UNII
8V8EF6094N
Created by admin on Sat Dec 16 08:50:01 GMT 2023 , Edited by admin on Sat Dec 16 08:50:01 GMT 2023
PRIMARY