Trophos Presents Pivotal Phase II/III Study Results on Olesoxime in Spinal Muscular Atrophy at The American Academy of Neurology Meeting

By Cure SMA | Published On August 26, 2014

Originally published on April 29, 2014

The 66th American Academy of Neurology (AAN) Meeting is being held this week in Philadelphia. Over 10,000 neurologists are in attendance. Trophos presented late breaking data on their pivotal phase II/III trial on olesoxime in Spinal Muscular Atrophy there.

Trophos is a clinical stage pharmaceutical company developing innovative therapeutics for indications with under-served needs in neurology and cardiology. The company has a novel and proprietary cholesterol-oxime based chemistry platform generating a pipeline of drug candidates. The lead product, olesoxime (TRO19622), is being developed for SMA and multiple sclerosis.

The company indicated that the data shows that patients treated with olesoxime were able to maintain motor function over the two-year period of the study and that typical health complications associated with SMA occurred less frequently than in patients treated with a placebo, leading to better well being.

The new data presented at AAN is from the recently completed international, double-blind, placebo-controlled study involving 165 type II and non-ambulatory type III SMA patients, ranging in age from 3 to 25 years old.

The results show that olesoxime treatment preserved motor function for two years using the Motor Function Measure scale (MFM) D1+D2 as the primary endpoint. The MFM is a standardized neuromuscular disease-specific functional scale. In contrast, patients in the placebo arm of the study experienced a loss of motor function starting from a mean score of 39 per cent at baseline to 37.1 per cent after two years. The mean loss of 1.9 points in motor function over the two-year study period confirms that the natural disease progression results in approximately 1 per cent per year loss of motor function in SMA patients.

“Analysis of the data from the pivotal trial of olesoxime provides further evidence of its neuroprotective effect, with a statistically significant impact on maintenance of motor function for the two years of the trial compared with placebo,” said Dr. Enrico Bertini, the principle investigator of the study. “Results from secondary endpoints were also promising. The olesoxime-treated group experienced fewer adverse events caused by the disease itself. SMA is a devastating condition which, even in its less severe forms, leads to progressive muscle wasting and the loss of mobility and motor function. Olesoxime has the potential to be the first ever treatment approved especially for SMA patients.”

Read FAQs on the Trophos drug or see our full 2014 SMA Drug Pipeline for more information.

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