Trophos to file olesoxime after positive data in orphan indication

French pharma Trophos has reported positive top-line data from its pivotal Phase II/III study of olesoxime in spinal muscular atrophy (SMA), a product the company has resolutely backed despite past setbacks.

Olesoxime, a cholesterol-like compound that targets and preserves mitochondrial integrity and function in stressed cells, was shown to prevent loss of motor function (the primary endpoint) for two years compared with placebo