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Trophos' olesoxime selected by Thomson Reuters’ ‘Ones to Watch’ Report as a top five Phase III product

Olesoxime in ongoing pivotal efficacy study for amyotrophic lateral sclerosis.

Trophos SA, a clinical-stage pharmaceutical company developing therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, announced today that olesoxime, its lead compound, has been selected as one of the five most interesting products entering Phase III in the ‘Ones to Watch’ report. A Thomson Reuters quarterly review of the latest phase changes in the pharmaceutical pipeline, the ‘Ones to Watch’ is based on independent data and analysis from Thomson Pharma. For a copy of the full report with analysis, visit

Olesoxime is currently in an ongoing pivotal efficacy study in amyotrophic lateral sclerosis (ALS), which is supported by the EU MitoTarget project. Efficacy results are expected in mid-2011.

‘We are delighted that the promise of olesoxime has been recognised by the independent experts of Thomson Reuters,’ said Damian Marron, Chief Executive Officer of Trophos. ‘We believe olesoxime could be a valuable new medicine for the ALS community and we are looking forward to the results of the ongoing study, which we hope will demonstrate that promise and bring new hope to ALS patients.’

Olesoxime (TRO19622) is the lead compound of the Trophos' proprietary cholesterol-oxime compound family of mitochondrial pore modulators. Preclinical studies have demonstrated that the compounds promote the function and survival of neurons and other cell types under disease-relevant stress conditions through interactions with the mitochondrial permeability transition pore (mPTP), and olesoxime has been shown to be active in the SOD1 model of ALS [1].

Olesoxime has successfully completed Phase I studies in healthy volunteers and Phase Ib studies in ALS patients. These clinical trials demonstrated that the product is well tolerated and has an excellent safety profile. They also showed that once-a-day oral dosing achieves the predicted exposure level required for efficacy, based on preclinical models. Drug interaction studies with riluzole, the only registered treatment for ALS, showed no interaction of TRO19622 with riluzole pharmacokinetics.

The ongoing clinical study of olesoxime is part of a three-year collaborative project named MitoTarget. The European Commission has awarded a grant of nearly €6 million for MitoTarget, which will be carried out by a consortium led by Trophos. MitoTarget forms part of the Seventh Framework Programme of the European Community for Research, Technological Development and Demonstration Activities. In addition to 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.

ALS, more commonly known as Lou Gehrig's disease in the USA, is a progressive and fatal neurological disease that is estimated to affect more than 100,000 people worldwide. There is no cure for ALS. The only drug approved for ALS is riluzole (Sanofi-Aventis), which has been demonstrated to give a 2–3-month survival benefit to ALS patients.


1 Bordet, T. et al. (2007) Identification and characterization of Cholest-4-en-3-one, Oxime (TRO19622), a novel drug candidate for amyotrophic lateral sclerosis. J. Pharmacol. Exp. Ther. 322, 709–720


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