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Trophos and the MitoTarget Consortium demonstrate potential of modulating mitochondrial dysfunction in the treatment of neurodegenerative diseases

"Mitochondrial dysfunction is increasingly recognized as a key target in developing effective treatments for neurodegenerative diseases. These first data coming from the various MitoTarget research program are very encouraging and will be complemented by a range of ongoing and planned studies during the remainder of the MitoTarget project. At the same time a key part of the MitoTarget project, the pivotal clinical study of Trophos’ mitochondrial pore modulator, olesoxime, in over 500 patients with Amyotrophic Lateral Sclerosis, is proceeding on track, with results expected in the fourth quarter of next year." Dr Rebecca Pruss, MitoTarget Project's Coordinator

Presentations at the Society for Neuroscience congress also focus on underlying mechanisms of neuronal cell metabolism and death



Marseille, France, November 18, 2010 - 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 Trophos and partners in the European Union funded MitoTarget consortium presented four posters at the Society for Neuroscience (SfN) Meeting, 13th – 17th November, in San Diego, USA. The data presented demonstrate the potential for treating a range of neurodegenerative diseases including Alzheimer’s Disease and Huntington’s Disease by targeting mitochondrial dysfunction and also focused on mechanisms involved in neuronal metabolism and death.

“We are very pleased to present with our partners this important work, conducted in collaboration with a distinguished international scientific consortium,” said Rebecca Pruss, CSO at Trophos. “Mitochondrial dysfunction is increasingly recognized as a key target in developing effective treatments for neurodegenerative diseases. These first data coming from the various MitoTarget research program are very encouraging and will be complemented by a range of ongoing and planned studies during the remainder of the MitoTarget project. At the same time a key part of the MitoTarget project, the pivotal clinical study of Trophos’ mitochondrial pore modulator, olesoxime, in over 500 patients with Amyotrophic Lateral Sclerosis, is proceeding on track, with results expected in the fourth quarter of next year.”

The four presentations at the SfN were:

  • Changed mitochondrial membrane properties in neurodegenerative diseases - A pharmacological target for Olesoxime, Eckmann et al. This focused on changes in mitochondrial membrane fluidity in aging and in models of neurodegeneration (Alzheimer’s disease and Huntington’s disease), which can influence mitochondrial fusion, fission and function, and that olesoxime reverses modifications in membrane fluidity and morphology in cells with Huntington’s Disease mutations or induced mitochondrial dysfunction
  • Mitochondrial morphology - A sensitive marker for mitochondria targeting drugs, Eckert et al. This describes how mitochondrial morphology perturbances can be a sensitive measure of stress-induced mitochondrial dysfunction and proposes that protection of the mitochondrial morphology represents an important strategy to investigate new drugs for the treatment of Alzheimer’s Disease
  • Calreticulin: A link between ER stress and death in SOD1-associated ALS motoneurons, Bernard et al. This describes fundamental research on possible mechanisms underlying motor neuron cell death in amyotrophic lateral sclerosis (ALS)
  • Primary cortical and striatal neurons use substrates other than glucose for energy production, Gouarné et al. This focuses on the ability of neurons to use glial-supplied substrates other than glucose to support oxidative phosphorylation, which is important when evaluating metabolic processes in neurodegeneration.


MitoTarget is a three year collaborative project started in February 2009 for which the European Commission has awarded a three year grant of nearly EUR 6 million. MitoTarget is being carried out by an international consortium of 17 partners comprising of leading Western European treatment and research centres, led by Trophos. MitoTarget forms part of the Seventh Framework Programme of the European Community for Research, Technological Development and Demonstration Activities and has two main goals: to investigate the efficacy and safety of Trophos compound olesoxime (TRO19622) in a pivotal clinical study in Amyotrophic Lateral Sclerosis (ALS) patients (see releases of May 9 2009 and March 17 2010) and to develop the understanding of mitochondrial dysfunction and therapeutic potential of a novel proprietary class of molecules in neurological diseases.

The work was supported by the European Union under the 7th Framework Programme for RTD - Project MitoTarget - Grant Agreement HEALTH-F2-2009-223388


About Trophos: www.trophos.com
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, with the lead product, olesoxime (TRO19622), in phase 3 development and a second product, TRO40303, in early clinical development. 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 Ltd; an acquisition option agreement and a research collaboration agreement.


Trophos was founded in 1999, is based in Marseille, France and currently has 29 employees.

 

For further information, please contact:
Andrew Lloyd & Associates
Andrew Lloyd / Neil Hunter
Tel: +44 1273 675100
allo@ala.com / neil@ala.com

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    • EUR 7 million grants boost Trophos cholesterol-oxime programs
    • Trophos and the MitoTarget Consortium demonstrate potential of modulating mitochondrial dysfunction in the treatment of neurodegenerative diseases
    • Actelion joins forces with Trophos to further advance olesoxime to the market once results of the MitoTarget clinical study are known.
    • Trophos announces results of phase 3 study of olesoxime in Amyotrophic Lateral Sclerosis
    • Disappointing results of olesoxime clinical trial announced
    • Trophos initiates pivotal efficacy study of olesoxime in Amyotrophic Lateral Sclerosis (Lou Gehrig's disease)
    • Trophos announces final patient completion of pivotal phase three efficacy study of olesoxime in Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease)
    • MitoTarget Launches New Online Resource for Physicians and Sufferers from Rare Illness ALS (also known as Lou Gehrig’s disease)
    • Trophos completes patients enrolment in ALS phase 3 trial
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The MitoTarget project is supported by the European Union under the 7th Framework Programme for RTD - Project MitoTarget - Grant Agreement HEALTH-F2-2008-223388

Seventh Framework Programme European Union