PhIII ALS failure costs Trophos a $250M buyout deal with Actelion

France's Trophos says that a Phase III study of its lead drug for ALS (Lou Gehrig's disease) failed to hit its primary endpoint, and the swing and a miss will cost its investors up to $250 million. That's the top amount Actelion had promised to pay for Trophos when it took out a buyout option for 10 million euros.

Trophos recruited 512 ALS patients for the study, but tersely noted that the patients taking olesoxime failed to demonstrate an improved survival rate compared to the riluzole/placebo arm. The biotech quickly concluded that the Phase III failure was probably due to the poor condition of patients once they're diagnosed. "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," the developer noted in its release. And Trophos went on to say that a separate study of the experimental drug in MS patients isn't likely to run into the same problem.

That wasn't good enough for Actelion, the hard-pressed Swiss biotech which inked a buyout option in the summer of 2010. In need of new products and pipeline wins, Actelion simultaneously announced that it is giving up its option. Late-stage development is a high-stakes game, and analysts have noted that the trend these days is to kill nonperforming pacts as quickly as possible rather than allow them to linger. Trophos, for its part, is turning to several other programs in the clinic.

"We remain convinced of the promise of our cholesterol-oxime, mitochondrial pore modulator compounds," said Trophos CEO Damian Marron. "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."

- get the press release from Trophos
- here's the release from Actelion

Special Report: Actelion - Biotech's Biggest Spenders 2011