Biogen ALS Failure Highlights Clinical Trial Frustrations

1/10/13Follow @cathyarnst

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companies are chasing after those same patients for their trials. Currently there are at least five drugs in Phase 2 trials for ALS; if each enrolled a few hundred patients, the available pool could quickly be depleted.

There is also a good news/bad news development in the treatment of the disease—steady improvement in the standard of care means that trial participants on placebo are doing better than expected, raising the bar for experimental drugs. “We have seen dramatic incremental improvements in patient care that have slowed down the disease in the last five years,” Perrin said. That means multi-year clinical trials may find that the level of improvement required by the time testing ends is higher than when the trial began.

That may have been part of the problem for olesoxime, developed by France’s Trophos. Trophos reported in December 2011 that olesoxime failed to show clinically significant improvement over a placebo in a Phase 3 trial of 512 patients in advanced stages of the disease. At this year’s Annual Symposium on Amyotrophic Later Sclerosis/Motor Neuron Disease, held in Chicago Dec. 5-7, the Parisian doctor who presented the results of the unsuccessful olesoxime study said that almost 70 percent of patients on placebo during the trial survived 18 months, compared with a 50 percent survival rate for patients in a 1996 ALS study, according to a report in the Alzheimer’s Research Forum.

The need for better treatments could not be more dire. ALS, often called Lou Gehrig’s disease after the revered New York Yankees first baseman who died from it in 1941, is a progressive neurological disease which attacks nerve cells that control muscle movement. Patients typically die within a few years of diagnosis. There is no known cause or cure, and the only drug on the market, Sanofi’s (NYSE: SNY) riluzole (Rilutek), was approved by the FDA seven years ago, and improves survival times by just a few months.

As for the other drugs in clinical trials, progress is a bit of a one step forward, two steps back dance. Neuraltus Pharmaceuticals of Palo Alto, CA, reported in October that its NP001, a drug that prompts certain immune system cells to protect rather than attack the central nervous system, failed to meet its clinical endpoints in a 136-patient Phase 2 trial. Nevertheless, the company said it will move … Next Page »

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