Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, has completed its efficacy study of Olesoxime in 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).
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.
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).
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.
Praying for the good result of Trophos SMA treatment exploration
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