Home | Clinical | New Drugs Online
NDO Logo

Home | Additional information is available to accredited, [registered, NHS users]

New Drugs Online Report for olesoxime
Generic Name:
Trade Name:  
Synonym: TRO19622, RG6083 
Entry Type: New molecular entity  
Development and Regulatory status
UK: Discontinued 
EU: Discontinued 
US: Discontinued 
UK launch Plans: Available only to registered users
Actual UK launch date:  
Apr 12: A news release in Dec 11 reporting disappointing results of PIII study implies that the development programme in AMS is unlikely to be continued, although the company will progess studies in Spinal Muscular Atrophy and multiple sclerosis [5] 
26/04/2012 12:00:53
Orphan drug status in EU and US [2]. 
02/04/2010 19:56:05
Mar 10: Key efficacy results from PIII study expected Q4 2011 (1) 
18/03/2010 08:19:06
Trial or other data
Dec 11: The PIII study failed to demonstrate a significant increase in survival with olesoxime (330mg daily) vs placebo in patients receiving riluzole. The company suggest that this is because the disease process is already so severe and rapidly progressing by the time of diagnosis that any further benefit of olesoxime over that of riluzole cannot be detected. The study was an 18-month randomized RCT in 512 patients diagnosed with ALS between 6 and 36 months before enrollment and receiving standard of care. It was undertaken in 15 centres in the EU as part of a 3-year collaborative project MitoTarget for which the European Commission has awarded a grant of nearly EUR 6 million.
Actelion has informed Trophos of its decision to not exercise its exclusive option under the July 2010 Acquisition Option Agreement [4] 
14/12/2011 22:02:16
Feb 11: NCT01285583 is a 15 month open-label safety extension study in 350 ALS patients on riluzole who took part in the TRO19622 CL E Q 1015-1 study. The dose is two capsules of olesoxime (330mg) once a day with the noon meal as add-on therapy to riluzole 50mg twice daily. The study started in Oct 10 and is due to complete Mar 12 [4]. 
21/02/2011 10:08:44
Jul 10: Actelion has signed an acquisition agreement to purchase Trophos SA within 2 months of Actelion´s receipt of the results of an ongoing PIII study with olesoxime (but not later than Dec 31, 2012). The purchase is partially contingent on market approval by the FDA as well as overall pipeline progression of other compounds [3].  
20/07/2010 20:44:47
Mar 10: The PIII study of olesoxime is part of a three year collaborative project named MitoTarget. The European Commission has awarded a grant of nearly EUR 6 million for MitoTarget which is being carried out by a consortium led by Trophos. As well as the clinical trial, MitoTarget aims to enlarge the understanding of mitochondrial dysfunction in neurodegenerative diseases and assess the therapeutic potential of Trophos´ novel proprietary class of mitochondrial pore modulator molecules in neurological diseases [2]. 
02/04/2010 19:55:39
Mar 10: Pivotal PIII study. 18-month randomized, parallel group, double-blind, placebo controlled trial evaluating the efficacy and safety of olesoxime 330mg od against placebo in patients (>500 enroled) diagnosed with ALS between 6 and 36 months before enrolment who are already treated with riluzole (50mg bd, both study arms). The study is being undertaken in 15 centres in France, Germany, UK, Belgium and Spain. The primary end-point of the study is the overall 18 month survival rate. Secondary end-points include the ALS Functional Rating Scale, time to assisted ventilation, vital capacity (a measure of respiratory function), Manual Muscular Testing and quality of life. (1) 
18/03/2010 08:22:05
Evidence Based Evaluations
NHSC  http://www.nhsc-healthhorizons.org.uk/files/downloads/1577/2034.e530e255.Olesoxime.pdf 
Available only to registered users
BNF Category:
Drugs used in essential tremor, chorea, tics, and related disorders (04.09.03)
Pharmacology: in the cholesterol-oxime compound family of mitochondrial pore modulators  
Epidemiology: ALS (Lou Gehrigs disease) is a progressive and fatal neurological disease affecting >100,000 people worldwide. There is no cure for ALS [2].  
Indication: Amyotrophic lateral sclerosis (motor neurone disease) 
Method(s) of Administration
Company Information
Name: Trophos SA 
US Name: Trophos SA 
Further Information
Anticipated commissioning route (England)
High cost drug list? Awaiting Update
Implications Available only to registered users