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Trophos Completes Pivotal Phase II/III Study of Olesoxime in SMA

Published: Friday, November 08, 2013
Last Updated: Friday, November 08, 2013
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The French patients’ association AFM-Telethon supported the study which evaluates the ability of olesoxime to preserve the neuromuscular function of SMA patients.

Trophos SA has completed its efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA).

Outcome data are expected to be available towards the end of 2013. If positive, Trophos will file olesoxime for market authorization in the EU and the US for the treatment of SMA with the aim of having the drug on the market by 2015.

The phase II/III pivotal study evaluated the efficacy and safety of Trophos’ olesoxime - a novel mitochondria pore modulator with neuroprotective and nerve repair properties.

Substantial funding for this study came via the partnership of Trophos with the Association Francaise contre les Myopathies (AFM-Telethon) (see press release of March 19, 2009).

The study enrolled 165 SMA patients in a 24-month randomized, parallel group, double-blind, placebo controlled trial comparing olesoxime against placebo in type II and non-ambulant type III SMA patients aged from three to twenty five years old.

Olesoxime is administered at the dose of 10 mg/kg/day using a specially developed liquid formulation. Patients were randomized to receive olesoxime in a 2:1 ratio versus placebo. This trial protocol and its data analysis plan went through the EMA protocol advice procedure.

The primary end-point of the study is the change from baseline in the Motor Function Measure (MFM) functional scale. Secondary end-points include the Hammersmith Functional Motor Scale, the respiratory function and electromyography (CMAP - Compound Muscle Action Potential and MUNE - Motor Unit Number) as well as measures of safety, tolerance and quality of life.

Trophos is also exploring changes in a panel of possible SMA biomarkers in collaboration with the Spinal Muscular Atrophy Foundation (

The study is sponsored by Trophos. The coordinating investigator is Dr. Enrico Bertini, a leading clinical expert with extensive knowledge on the pathophysiology of SMA. His expertise in the design and performance of clinical trials in SMA was vital for setting up the project. The study was conducted in 22 centers in France, Italy, Germany, UK, Belgium, the Netherlands and Poland by a network of investigators with extensive experience in treating SMA patients.

"This study will be a landmark for the SMA community. Olesoxime is a promising therapy for patients living with SMA. It gives hope that they will maintain function and autonomy,” said Dr Enrico Bertini. “The longitudinal data collected from 165 patients for two years comparing functional outcome measures with potentially predictive and innovative biomarkers will make a valuable contribution to future clinical trial designs for SMA or other neuromuscular diseases.”

"We are pleased to have been able to enrol 165 patients with this rare disease according to protocol within one year, without substantial withdrawal throughout the two year clinical trial," said Dr Wilfried Hauke, chief medical officer at Trophos. “As this was basically a clinical trial in children as young as three years old and adolescents, the exceptional motivation and commitment of the patients and their families as well as the 22 clinical teams was a key element in the successful completion of this clinical trial. We anticipate the results very shortly.”

"The partnership between Trophos and the AFM-Telethon that started in 2000 has realized a major step towards providing a possible therapy to improve the quality of life of SMA patients,” said Christian Cottet, CEO, AFM-Telethon. “This has been achieved thanks to the generous public donations that allow the AFM-Telethon to play an essential role in enabling companies like Trophos to discover and develop therapies for rare diseases.”

"Trophos and the AFM-Telethon have been working together for over a decade. This crucial clinical study is the latest step in our long-standing partnership," said Christine Placet, CEO, Trophos. "Assuming positive results in this clinical trial, Trophos is now in discussions with both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) to prepare for the next steps in bringing this drug to market as soon as possible."

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