Trophos program boosted by EUR 1 million grant for olesoxime in progressive Multiple Sclerosis


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 the award of a EUR 1 million grant to support the Translate-MS-Repair project.

The French ‘Agence Nationale de la Recherche’ (ANR) awarded the second successive grant to Trophos to target Multiple Sclerosis with olesoxime. The funding will be provided under the Recherches Partenariales et Innovation Biomedicale 2012 program.

Translate-MS-Repair will be a 24 month project and will include a Phase Ib/IIa clinical study of olesoxime, Trophos’ lead compound, to target neurodegeneration that underlies long term progressive disability in multiple sclerosis.

The project will be operated by a consortium spearheaded by Trophos including leading experts in Marseille (AP-HM/CNRS and CEMEREM-CRMBM), Rennes (CHU de Rennes and INRIA VISAGES) and Reims (CHU de Reims).

The clinical trial, which will be performed in 3 centers in France, will be led by Dr. Jean Pelletier AP-HM/CNRS-CEMEREM. The clinical trial is designed to demonstrate the compatibility of olesoxime as a co-medication with existing treatments, the most frequent being beta interferon. The study will also assess the feasibility of non-conventional magnetic resonance imaging procedures in a multicenter trial. The clinical trial is designed to result in future large-scale clinical trials to assess efficacy of olesoxime to prevent progressive disability in MS patients.

“MS is the first cause of non-traumatic disability in young adults. While there are a number of effective treatments to control the relapsing inflammatory episodes, they have little effect on progressive disability in MS patients,” said Rebecca Pruss, CSO, Trophos. “MS is a chronic inflammatory disease leading to demyelination and axonal degeneration in the central nervous system. However, it is now also recognized that MS is a neuro-degenerative disease as well. There is a real unmet need for agents to promote neuroprotection and myelin repair for MS.”

“The second grant from the ANR to Trophos for our MS program is a recognition of the quality of Trophos’ ongoing activity in neurodegenerative diseases within the Trophos pipeline,” said Christine Placet, CEO, Trophos. “The ongoing success of olesoxime and the Trophos pipeline fits perfectly with Trophos strategy value creation.”

Translate-MS-Repair is a sequel to the successful ANR funded MS-Repair project lead by Trophos and performed in collaboration with Aix-Marseille University and CNRS partners, IBDML (UMR 6216) and CRMBM (UMR 6612). MS-Repair (ANR-08-BIOT-016-01) explored from 2008 to 2011 the activity of the molecule olesoxime in various in vitro and in vivo models of MS. This project showed that the molecule is not only neuroprotective, but it has the ability to promote the maturation of oligodendrocyte progenitor cells (OPCs) into myelinating oligodendrocytes (details in Magalon et al, Ann Neurol. 2012 Feb;71(2):213-26).

About Translate-MS-Repair

Translate-MS-Repair is a consortium spearheaded by Trophos with 3 clinical centers (Assistance Publique Hôpitaux de Marseille, Centre Hospitalier Universitaire de Rennes, Centre Hospitalier Universitaire de Riems) and two MRI specialist labs (Centre National de la Recherche Scientifique CEMEREM and INRIA VISAGES). The consortium members have been specifically selected to complete this study. The Phase 1b/2a study will be a randomized, placebo-controlled, double blind study to evaluate the effect of olesoxime in 30 relapsing remitting MS patients stably treated with beta interferon 1a.  A range of conventional and non-conventional MRI procedures will be performed at enrollment and after 6 months of treatment.  The primary outcome will be the change in relapse rate to assure that olesoxime treatment is compatible with interferon. The study will also assess the feasibility of performing non-conventional MRI including 23Na-MRI, diffusion tensor imaging, magnetic transfer resonance and magnetic resonance spectroscopy in a multicenter trial.

About Olesoxime

Olesoxime (previously known as TRO19622) is a mitochondrial targeted, cholesterol-like compound that is currently undergoing Phase 2 clinical evaluation for the treatment of spinal muscular atrophy. Olesoxime’s safety and tolerability has already been tested in 15 clinical trials involving 968 patients or healthy volunteers and proved to have an excellent safety profile.

The mechanism of action of olesoxime involves prevention of mitochondrial dysfunction and improved microtubule dynamics, both implicated in neuroprotection and oligodendrocyte maturation. Studies in preclinical models showing that olesoxime favors myelination were reported by Trophos and colleagues in Annals of Neurology (Magalon et al, Ann Neurol. 2012 Feb;71(2):213-26).

About Multiple Sclerosis

MS is a chronic inflammatory disease of the central nervous system leading to demyelination and axonal degeneration. When myelin is destroyed, it leaves random areas of scar tissue (sclerosis) which affect the ability of nerve cells to communicate with each other and results in the blocking of neurological transmissions leading to physical and cognitive disability. It is estimated that approximately 2 million people worldwide suffer from MS. Most of them are 20 – 40 years old with females outnumbering males by 2:1 ratio.  The majority of MS patients initially are diagnosed on the basis of recurring inflammatory episodes (relapsing remitting MS) but about 50 per cent of these patients will show progressive disability even in the absence of inflammatory relapses (secondary progressive MS) within 10 years of diagnosis, those figures raising to 90 per cent within 30 years. Others present with progressive neurological symptoms typical of MS without an episodic inflammatory history (primary progressive MS). Although there are several therapies approved for the treatment of RRMS, there are no marketed drugs to treat progressive MS.

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 II/III development for spinal muscular atrophy, an orphan neurological disease and, TRO40303, in Phase II clinical development, to treat cardiac reperfusion injury following angioplasty to treat an acute myocardial infarction; this study is part of an EC FP7 funded translational research project called MitoCare (HEALTH-F2-2010-261034). Trophos’ proprietary, cholesterol-oxime family of compounds enhance the function, and survival of stressed cells and favour neuronal and oligodendrocyte maturation.

Trophos was founded in 1999, is based in Marseille, France and currently has 26 employees.