SMA: Olesoxime Results Look Promising

Update (May 2, 2014): Trophos, developer of olesoxime to treat SMA, announced more detailed results from this trial at the spring 2014 annual meeting of the American Academy of Neurology and in an April 28, 2014, press release. The company said the data show that patients treated with olesoxime maintained motor function over the two-year period of the study and that typical health complications associated with SMA occurred less often in the olesoxime group than in the placebo group, leading to greater well-being. "The efficacy of olesoxime in patients with SMA demonstreated in this pivotal trial [now referred to as a phase 2-3]  is highly encouraging," said Christine Placet, chief executive officer at Trophos. "On the basis of these results we intend to file for approval in both the US and Europe as soon as possible."

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The experimental compound olesoxime (TRO19622), in development by French biotechnology company Trophos as a potential treatment for spinal muscular atrophy (SMA), has shown encouraging results in a phase 2 trial involving 165 children and young adults with this disorder.

In their March 10, 2014, press release, Trophos announced that olesoxime showed a "beneficial effect on the maintenance of motor function."

The drug, which targets the energy-producing parts of cells known as mitochondria, is designed to enhance the function and survival of cells under stress.

If approved in the near future, it could become the first commercially available drug developed specifically for SMA.

Olesoxime-treated participants didn't lose function

The olesoxime trial was conducted in seven European countries in patients with type 2 or type 3 SMA who were 3 to 25 years old. Participants were randomly assigned to receive either 10 milligrams per kilogram of body weight per day of olesoxime by mouth, or a look-alike, inactive medication (placebo).

Every three months for two years, participants were evaluated on standardized tests of motor function, as well as tests of electrical signals from nerves to muscles, tests of respiratory function, quality-of-life measures, incidence of typical SMA complications and safety outcomes.

Over the two-year course of the study, participants on the placebo showed progressive loss of motor function typical for SMA, while those on olesoxime showed preservation of motor function and fewer disease-related adverse events than the placebo group.

Trophos says it expects to publish more detailed results and to announce them at upcoming scientific conferences. "Our focus now is on the regulatory steps needed to bring this important product to patients as quickly as possible," said Enrico Bertini, principal investigator on this study and a pediatric neurologist at Bambino Gesu Hospital in Rome.

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