Trophos SA, has announced the completion of patient enrolment in the pivotal efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA).
Over 160 patients have been recruited into the trial since its initiation in October 2010. 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 the second half of 2013.
"The completion of recruitment in this pivotal clinical study in only ten months given the rarity of SMA is a great achievement and a major step in the development of olesoxime as a potential treatment for SMA," said Jean-Louis Abitbol, chief medical officer at Trophos.
Abitbol continued, "SMA is a debilitating and disabling neuromuscular disease and there is a critical 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. Over 160 patients have been included in the study in only ten months, which reflects both the great commitment of patients and clinicians to find a treatment for SMA and the motivation and hard work of all involved. We anticipate the results of the trial in the second half of 2013 and hope this will be a historic moment for the medical community as well as those affected now and in the future.”
"Thanks to the donations to the French telethon, we have been supporting the development of olesoxime since the first screening up to and including the ongoing clinical phases. The recruitment in this important clinical study has just been completed with great efficiency and brings hope for a first potential treatment to SMA patients,” said Christian Cottet, CEO, AFM.
“Trophos and the AFM been working together for over a decade and this crucial clinical study with Trophos’ olesoxime in SMA is the fruit of our long standing partnership,” said Damian Marron, CEO, Trophos.
Marron continued, “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. This study as well as our soon to be completed pivotal study of olesoxime in amyotrophic lateral sclerosis (ALS) underlines Trophos’ commitment to developing breakthrough therapies for rare and serious neurodegenerative diseases.”
Trial design and end-points
The study is an 24-month randomized, parallel group, double-blind, placebo controlled trial comparing olesoxime against placebo in non-ambulant SMA patients aged from three to twenty five years old. Olesoxime is being dosed at ten mg/kg/day and patients are randomized to receive olesoxime in a 2:1 ratio versus placebo. The study is being conducted in over 160 patients in 24 centres in France, Italy, Germany, UK, Belgium, the Netherlands and Poland.
The primary end-point of the study is the change from baseline in the Motor Function Measure (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-centre clinical trials in SMA.