OLESOXIME

Description
Sources: https://www.ncbi.nlm.nih.gov/pubmed/28460889 | https://www.roche.com/media/store/releases/med-cor-2015-01-16.htm | https://clinicaltrials.gov/ct2/show/NCT01808885

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

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
mitochondrial permeability transition pore 3 Target ID: mitochondrial permeability transition pore Sources: https://www.ncbi.nlm.nih.gov/pubmed/20721828	Modulator 828

Conditions

Condition	Modality	Highest Phase	Product
Spinal muscular atrophy 5 Sources: https://www.ncbi.nlm.nih.gov/pubmed/28460889	Primary	Phase II 1132	Unknown Approved Use Unknown
Relapsing-remitting multiple sclerosis 10 Sources: https://clinicaltrials.gov/ct2/show/NCT01808885	Primary	Phase I 428	Unknown Approved Use Unknown
Amyotrophic lateral sclerosis 41 Sources: https://www.ncbi.nlm.nih.gov/pubmed/24447620	Primary	Phase III 656	Unknown Approved Use Unknown

Sourcing

Vendor/Aggregator	ID	URL
BLD Pharm	BD630551	http://www.bldpharm.com/search/Search.html?keyword=BD630551
Lab Network	LN01342007	https://labnetwork.com/frontend-app/p/#!/moleculedetails/LN01342007
MolPort	MolPort-046-426-194	https://www.molport.com/shop/molecule-link/MolPort-046-426-194
Molnova	M13512	https://www.molnova.com/en/serch-list.html?keywords=M13512
NCI Plated 2007	21311
TimTec	ST50984588	http://www.echemstore.com/catalogsearch/result/?q=ST50984588
eMolecules	106538472	https://orderbb.emolecules.com/search/#?query=106538472

PubMed

Title	Date	PubMed
Specific antinociceptive activity of cholest-4-en-3-one, oxime (TRO19622) in experimental models of painful diabetic and chemotherapy-induced neuropathy.	2008 Aug	18492948
Dysfunction of mitochondria and sarcoplasmic reticulum in the pathogenesis of collagen VI muscular dystrophies.	2008 Dec	19076452
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	19833436
Clinical trials in CNS--SMi's eighth annual conference.	2010 Feb	20127552

Patents

Sample Use Guides

In Vivo Use Guide

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

In Vitro Use Guide

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	Mixture 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	A6778U5IFY
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

OLESOXIME

Official Name

English

(EZ)-N-(CHOLEST-4-EN-3-YLIDENE)HYDROXYLAMINE

Common Name

English

TRO-19622

Code

English

olesoxime [INN]

Common Name

English

OLESOXIME [USAN]

Common Name

English

TRO19622

Code

English

Olesoxime [WHO-DD]

Common Name

English

NSC-21311

Code

English

CHOLEST-4-EN-3-ONE, OXIME

Common Name

English

4-CHOLEST-EN-3-ONE, OXIME

Common Name

English

Cholest-4-en-3-ylidenehydroxylamine

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

214305