By Karen Bernstein

In the "Year of the Deal" - with its preoccupation on industry consolidation and focus on company survival - one hears not a lot lately about the original biotech goal of providing cures for untreatable diseases - the promise of "novel drugs for unmet needs."

The demise of another program in amyotrophic lateral sclerosis is a reminder of how difficult the promise will be to attain, and a caution that even if consolidation creates a more robust industry balance sheet, the ability to demonstrate clinical success may be the most unmet need of the biotechnology sector.

The Syntex/Synergen Neuroscience Joint Venture said last week that it is discontinuing its trials of recombinant CNTF in ALS, based on study results showing the biologic hasn't slowed the course of the disease. Syntex has been acquired by Roche Holding, while Synergen Inc. is now owned by Amgen Inc.

Second CNTF setback

The 570-patient study in mild to moderately ill patients found that CNTF didn't slow the progression of muscle weakness over a six-month period compared to placebo. Patients received subcutaneous injections of placebo or 0.5, 2 or 5 µg/kg of drug. In addition, preliminary safety data showed that undisclosed side effects were observed at a greater rate in patients receiving drug. The data will be presented at the May meeting of the American Academy of Neurology meeting in Seattle.

While the results haven't been fully disclosed, they appear to mirror the experience of Regeneron Pharmaceuticals Inc. (REGN) with its CNTF trial. That study was stopped last spring after patients in the treatment group experienced side effects including cough and weight loss that were associated with poor performance on the trial's primary endpoint of muscle strength.

Neither AMGN nor Syntex have made a final decision on CNTF's future, according to AMGN spokesperson David Kaye. "This trial is dead, discontinued," he said. "But given that there is no available treatment for ALS, there may be some other research strategy to explore."