Trophos Announces Results of Phase III Study of Olesoxime in ALS

Trophos SA has announced the results from the phase III study of Trophos’ lead compound, olesoxime, in 512 patients with ALS. Olesoxime did not demonstrate a significant increase in survival versus placebo in patients receiving riluzole (Rilutek®).

A trend was seen on patients’ function as measured by the ALSFRS-R functional rating scale. Olesoxime was very well tolerated.

Following receipt of these results, Actelion has informed Trophos of its decision to not exercise its exclusive option under the July 2010 acquisition option agreement between Trophos and Actelion (see Actelion release of 13 December 2011).

Trophos continues its ongoing programs and has financing secured to at least the end of 2013. These programs include:

• An ongoing pivotal trial of olesoxime in Spinal Muscular Atrophy (SMA) with results due in the second half of 2013,
• An ongoing phase IIa proof-of-concept study of TRO40303 in cardiac ischemia-reperfusion injury (IRI), with results due before end 2012,
• A planned phase II proof-of-concept trial of olesoxime in Multiple Sclerosis (MS) disease progression (subject to securing outside funding),
• A research collaboration with Actelion to screen and characterize Actelion compounds using Trophos’ proprietary CNS assay technology.

The inability of olesoxime to show a greater effect on survival for ALS patients above that of riluzole in the phase III trial is most likely because the disease process is already so severe and rapidly progressing by the time of diagnosis that any further benefit of olesoxime over that of riluzole cannot be detected.

Indeed, it is known that in the most widely used ALS model, over 50 per cent of the motor neurons and neuromuscular connections have already been lost by the time the first symptoms appear.

Of Trophos’ other target indications for olesoxime, SMA type II and III, being studied in an ongoing trial, these differ in that disease progresses slowly over many years.

Progressive MS is again different as neuronal degeneration correlated with disease progression only sets in after symptoms appear.

“The results of this study in ALS are disappointing, above all for the ALS community, who urgently require new therapies that can prolong survival and improve function. We are genuinely proud to have worked closely with this community and our international partners in the MitoTarget project on this important and very well run study,” said Damian Marron, CEO, Trophos.

Marron continued, “We remain convinced of the promise of our cholesterol-oxime, mitochondrial pore modulator compounds. We have ensured that Trophos is financed until at least the end of 2013, so that we continue to move forward on our other programs, which address high medical need orphan or niche indications with no existing treatments.”