Trophos Announces Final Patient Completion of Pivotal Phase 3 Efficacy Study of Olesoxime in Amyotrophic Lateral Sclerosis (ALS, Or Lou Gehrig's Disease)

Published: Sep 22, 2011

Marseille, France, September 22, 2011 - Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, announces today that the last patient has now completed the major pivotal phase three efficacy study of olesoxime in Amyotrophic Lateral Sclerosis (ALS).

Over 500 patients were enrolled in the study and the last patient completed the prescribed study protocol procedures on 15th September. Work is now ongoing to finalise the analysis of the data. Efficacy and safety top-line results are expected in the fourth quarter of 2011.

The study is part of the EU FP7 three-year MitoTarget project (see below), comprising a pan-European consortium of leading experts in ALS and spearheaded by Trophos. The trial protocol has benefited from EMEA Scientific Advice procedure.

"We are very pleased with the speed and efficiency of the completion of the treatment of over 500 patients in this key clinical study of olesoxime in ALS. This reflects the motivation and hard work of all involved and the commitment of the patients, their carers and the teams at the clinical sites," said Jean-Louis Abitbol, chief medical officer at Trophos. "We are all looking forward with great anticipation to the results of the study, due before the end of the year.”

“ALS is a terrible and fatal underserved disease. More people die from ALS than multiple sclerosis (MS) each year. Patients urgently require new therapies that can prolong survival and improve the patient's respiratory and muscular functions,” said Damian Marron, CEO, Trophos. ”We believe olesoxime could be a valuable new medicine for the ALS community and we all hope that we may see a new chapter in the treatment of this devastating disease."

Trial design and end-points

This phase three study is an 18-month randomized, parallel group, double-blind trial evaluating the efficacy and safety of olesoxime against a placebo in patients diagnosed with ALS between six and thirty six months before enrolment who are already treated with riluzole. Olesoxime is dosed at 330 mg oral capsules once a day and riluzole is dosed in both arms at 50 mg twice a day. The study is being undertaken in 15 centers 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. Safety and tolerability is closely monitored and an independent Data Safety Monitoring Board is overseeing the trial.

The study is sponsored by Trophos and is being performed by a consortium of prominent European clinical investigators, all of whom have extensive prior experience conducting and collaborating in large multi-center clinical trials in ALS.


This clinical study of olesoxime is part of a three-year collaborative project named MitoTarget (Grant Agreement No: HEALTH-F2-2008-223388). The European Commission has awarded a grant of nearly EUR six million for MitoTarget. The project is being carried out by a pan-European consortium of experts led by Trophos. MitoTarget forms part of the Seventh Framework Programme of the European Community for Research, Technological Development and Demonstration Activities. As well as the clinical trial, MitoTarget aims to develop and increase 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 (see press release of December 18, 2008).

Orphan drug designations

Trophos has been granted orphan drug designation for olesoxime for the treatment of ALS and SMA (spinal muscular atrophy) by the U.S. Food and Drug Administration and 'Orphan Medicinal Product' designation for both ALS and SMA by the European Commission in the EU.

About olesoxime

Olesoxime (TRO19622) is the lead compound of 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); olesoxime has been shown to be active in the SOD1G93A mouse model of ALS (Bordet et al., JPET 322:709-720, 2007).

Olesoxime has successfully completed phase I studies in healthy volunteers and phase Ib studies in ALS patients. These previous 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 on riluzole pharmacokinetics.

Olesoxime is also in a pivotal efficacy and safety study in Spinal Muscular Atrophy (SMA), substantially funded by the AFM patient association (see release of October 15, 2010), and is in pre-clinical development for multiple sclerosis.

Information for physicians and patients

Trophos and the MitoTarget consortium have established a dedicated website for the MitoTarget project with a link from the Trophos website. The multilingual web-based resource aims to be the first point of call for all of those affected by ALS. This includes sufferers and all physicians, nurses and specialists, as well as members of the public with a friend or family member with the disease. The website contains detailed information on ALS and related subjects, and hosts a comprehensive range of links enabling all those affected to find further information and support.

About ALS (Amyotrophic Lateral Sclerosis):

ALS, more commonly known as Lou Gehrig's disease in the USA, is a progressive and fatal neurological disease that is estimated to affect over 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 two to three month survival benefit to ALS patients.

For more information about ALS, see

About Trophos:

Trophos is a clinical stage pharmaceutical company developing innovative therapeutics for indications with under-served needs in neurology and cardiology. The Company has a novel and proprietary cholesterol oxime based chemistry platform generating a pipeline of drug candidates. The lead product, olesoxime (TRO19622), is in phase three development for the orphan neurological diseases of ALS (as part an EU funded project, MitoTarget) and Spinal Muscular Atrophy (substantially funded by the AFM patient association). A second product, TRO40303, is in early clinical development to treat cardiac reperfusion injury (as part of an EU funded project, MitoCare). Trophos' mitochondrial pore modulator compounds enhance the function and survival of stressed cells via modulation of dysfunctional mitochondria through interactions at the permeability transition pore (mPTP). Recently published clinical studies support the therapeutic rationale for mitochondria targeted drugs, which Trophos is uniquely placed to exploit.

Trophos has two strategic partnership agreements with Actelion; an acquisition option agreement and a research collaboration agreement.

Trophos was founded in 1999, is based in Marseille, France and currently has 37 employees.

Mark Tidmarsh


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