Trophos presents data at the American Academy of Neurology on SMA and MS

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 has described the design of its pivotal clinical study of olesoxime in spinal muscular atrophy (SMA) via a poster presentation at this week’s 6th Annual American Academy of Neurology (AAN) meeting held in Honolulu, Hawaii, US, April 9 to 16, 2011. In addition at the conference, Trophos also delivered an oral presentation detailing Trophos’ novel approach in multiple sclerosis (MS). Both the paper and oral presentation were delivered at the conference on April 14.


The poster, on the pivotal efficacy study of olesoxime in the rare, neurodegenerative condition, SMA, demonstrates how the challenges of designing a robust study in an indication where there is no precedent were met by bringing together and working with key opinion leaders, patient and physician networks and regulators. The study commenced in October 2010 (see release of October 15 2010,]) and is currently recruiting patients. Efficacy results are expected in the second half of 2013. The study is substantially funded by Trophos’ partnership with the Association Française contre les Myopathies (AFM) (see release of 19 March 2009) and the trial protocol has benefited from the EMA Protocol Advice procedure.

In addition, the MS research data, delivered in an oral presentation, demonstrate that olesoxime, Trophos’ lead compound, is a promising candidate for neuroaxonal repair and remyelination in white matter diseases, notably multiple sclerosis. The data was generated with partners in the MS-Repair project, which is supported by the French Agence Nationale de la Recherche (ANR).

“These presentations highlight the rich potential of olesoxime and Trophos’ family of cholesterol oxime mitochondrial pore modulators in neurodegenerative conditions,” said Rebecca Pruss, CSO at Trophos. “SMA is a debilitating neuromuscular disease and there is an immense need for a treatment that can slow down or prevent the loss of muscle function in SMA patients. No specific treatment exists today. We are delighted to be working with the AFM and major clinical centers around Europe to undertake this important clinical trial.”

“It is now recognized that disease progression in MS may reflect chronic and progressive neurodegeneration while relapses only reflect acute focal inflammation of the CNS,” continued Dr Pruss. “Hence, despite recent advances in treating relapses, therapeutic strategies promoting remyelination and providing neuroprotection are now the key needs in MS. We are very pleased to present with our academic partners this important work that demonstrates the potential for olesoxime to bring a new treatment approach in multiple sclerosis through neuronal protection and remyelination. The aim of the work is to address the long term progression and disability associated with the disease.”

The poster presentation was:

Design of a Phase II Clinical Trial to Demonstrate Efficacy, Safety and Evaluate Biomarkers in Patients with Spinal Muscular Atrophy, Pruss et al. It described the process to design and launch a two-year, multi-centric, European trial with olesoxime in 3-25 year old patients with SMA.

The study is a 24-month randomized, parallel group, double-blind, placebo controlled trial comparing olesoxime against placebo in non-ambulant type 2 and type 3 SMA patients aged from three years upwards. Olesoxime is dosed at ten mg/kg/day and patients are randomised to receive olesoxime in a 2:1 ratio versus placebo. Around 160 patients are being recruited into the study in 23 centers in France, Italy, Germany, UK, Belgium, the Netherlands and Poland.

The primary end-point of the study is the change from baseline in the MFM functional scale. Secondary endpoints include the Hammersmith functional motor scale and electromyography (CMAP -Compound Muscle Action Potential – and MUNE – Motor Unit Number). There will be an interim analysis for efficacy and futility after one year. Safety and tolerability are closely monitored and an independent data safety monitoring board is overseeing the trial.

The oral presentation of results was:

Olesoxime promotes olligodendrocyte maturation and myelination and could be a promising complementary therapy for the treatment of multiple sclerosis, Pruss et al. It presented evidence 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 in February 2009 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.

About olesoxime

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 today 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 this year (see releases of May 9 2009 and March 17 2010) and the second a recently started pivotal clinical study in Spinal Muscular Atrophy.

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, with the lead product, olesoxime (TRO19622), in phase three 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 36 employees.