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Trophos Initiates Pivotal Efficacy Study of Olesoxime in Spinal Muscular Atrophy

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Trophos SA has announced the initiation of the pivotal efficacy study of olesoxime in the rare, neurodegenerative condition, Spinal Muscular Atrophy (SMA). The study is substantially funded by Trophos’ partnership with the Association Française contre les Myopathies (AFM) (see release of 19 March 2009). The trial protocol has benefited from the EMA Protocol Advice procedure. Efficacy results are expected in 2013.

Spinal Muscular Atrophy is an autosomal recessive genetic disease that affects the motor neurons of the voluntary muscles that are used for activities such as crawling, walking, head and neck control, and swallowing. Approximately 1 in 6000 babies born are affected and about 1 in 40 people are genetic carriers. SMA patients are divided into four subtypes depending on disease onset and severity but all suffer from degeneration of motor neurons controlling voluntary muscles with proximal limb and trunk muscle weakness leading to respiratory distress and in the most severe cases, ultimately death.

“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, for whom no specific treatment exists today," said Christian Cottet, managing director of the AFM. “The AFM has been working with Trophos since 2001 and the initiation of this clinical study with olesoxime in SMA is the fruit of our long standing partnership. Thanks to the donations to the French Telethon, the AFM is supporting 36 clinical trials involving 30 different diseases. These trials accelerate the development of the therapies of tomorrow: gene therapy, stem cells, pharmacogenomics - for rare diseases and common diseases.”

Olesoxime (TRO19622) is the lead compound of the Trophos' proprietary cholesterol-oxime compound family of mitochondrial pore modulators. Preclinical studies have demonstrated that olesoxime promotes the function and survival of neurons and other cell types under disease-relevant stress conditions through interactions with the mitochondrial permeability transition pore (mPTP) and olesoxime has been shown to be active in the NSE-Cre F7/F7 model of SMA.

Olesoxime is currently undergoing a pivotal efficacy study in another rare motor neuron disease, Amyotrophic Lateral Sclerosis (ALS), which has recruited 512 patients and for which results are expected in Q4 2011. Olesoxime has successfully completed a phase 1b study in SMA patients, having previously completed phase I/Ib studies in healthy volunteers and ALS patients. These 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.

“Olesoxime has a promising profile as a potential treatment for SMA and we are hopeful the results of this study will demonstrate that promise, bringing a much needed treatment option and new hope to SMA patients and their families," added Jean-Louis Abitbol, Chief Medical Officer at Trophos. “We are delighted to be working with the AFM and major clinical centers around Europe to undertake this clinical trial.”

Trial design and end-points:

The study is a 24-month randomized, parallel group, double-blind, placebo controlled trial comparing olesoxime against placebo in non-ambulant SMA patients aged from 3 years upwards. Olesoxime will be dosed at 10 mg/kg/day and patients will be randomized to receive olesoxime in a 2:1 ratio versus placebo. It is planned that around 160 patients will be recruited into the study in approximately 20 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 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 will be closely monitored and an independent Data Safety Monitoring Board will oversee 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 SMA.