Olesoxime (TRO19622) is the lead compound of the Trophos’ proprietary cholesterol-oxime compound family of mitochondrial pore modulators.
The 24-month randomized, parallel group, double-blind, placebo controlled trial will compare olesoxime against placebo in non-ambulant SMA patients aged from three to 25 years old.
Olesoxime is being dosed at 10 mg/kg/day and patients are randomised 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.
The study is substantially funded by Trophos’ partnership with the Association Française contre les Myopathies (AFM).
Efficacy results are expected in the second half of 2013.
Trophos has been granted orphan drug designation for olesoxime for the treatment of SMA and amyotrophic lateral sclerosis (ALS) by the US Food and Drug Administration and ‘Orphan Medicinal Product’ designation for both ALS and SMA by the European Commission in the EU.