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Trophos and MS-Repair Partners Present Promising Data on Novel Approach in Multiple Sclerosis

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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, announced that Trophos and partners in the MS-Repair consortium delivered an oral presentation detailing Trophos’ novel approach in multiple sclerosis (MS) at the recent Society for Neuroscience (SfN) Meeting.

The data presented demonstrate that olesoxime, Trophos’ lead compound, is a promising candidate for neuroaxonal repair and remyelination in white matter diseases, notably multiple sclerosis. The MS-Repair project is supported by the French Agence Nationale pour la Recherche (ANR).

“Disease progression in MS may reflect chronic and progressive neurodegeneration while relapses only reflect acute focal inflammation of the CNS. Hence, despite recent advances in treating relapses, therapeutic strategies promoting remyelination and providing neuroprotection are now the key needs in MS,” said Rebecca Pruss, CSO at Trophos. “We are very pleased to present with our academic partners this important work, which demonstrates the potential for olesoxime to bring a new treatment approach in multiple sclerosis through neuronal protection and remyelination aimed at addressing the long term progression and disability associated with the disease.”

The oral presentation of results was:
Novel compounds to promote axon repair and remyelination in multiple sclerosis, Bordet et al. It showed that Olesoxime dose-dependently promoted oligodendrocyte maturation and myelination in several in vitro (oligodendrocyte progenitor cells, oligodendrocyte – neuron co-cultures, organotypic brain slice cultures) and two in vivo models (cuprizone and lysophosphatidyl choline induced demyelination).

The results were obtained by a collaboration supported by the ANR project MS-Repair awarded to Trophos and two academic partners in Marseille, Dr Pascale Durbec at the Institut de Biologie du Développement de Marseille Luminy CNRS – Université de la Méditerranée UMR6216 and Dr Angèle Viola at the Centre de Résonance Magnétique Biologique et Médicale CNRS – Université de la Méditerranée UMR6612, in February 2009.

Olesoxime (TRO19622) is the lead compound of Trophos' proprietary cholesterol-oxime compound family of mitochondrial pore modulators, developed for their ability to 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).

The data announced show that olesoxime also has the ability to promote remyelination in addition to providing neuroprotection. Olesoxime is currently in two pivotal clinical efficacy studies, the first  in over 500 patients with Amyotrophic Lateral Sclerosis with results expected in the fourth quarter of next year and the second a recently started pivotal clinical study in Spinal Muscular Atrophy.

“These data on olesoxime in MS highlight the further strong potential of Trophos’ cholesterol oxime mitochondrial pore modulators in neurodegenerative diseases. Multiple sclerosis is one of the more common neurodegenerative diseases with an estimated 1.5 - 2.5 million sufferers globally,” added Damian Marron, CEO of Trophos. “We will continue this work to provide a complete package of data that will clearly demonstrate the value and therapeutic positioning of our compounds in multiple sclerosis.”