When is a trial 'negative,' exactly?

Cytokinetics invented a drug to treat ALS, the rare and devastating muscle-wasting disease. It entered that drug in a 485-patient trial, testing whether it could improve ALS symptoms on three measures. In the end, the drug did not significantly beat placebo on any of them, which is commonly considered a failure.

But, as STAT’s Adam Feuerstein reports, Cytokinetics doesn’t see it that way. In an interview, CEO Robert Blum said there was “a consistent and dose-dependent effect that is clinically meaningful for ALS patients.” The trial’s lead investigator said the data were “among the most impressive” he’d ever seen in an ALS trial, and now Cytokinetics wants to start a Phase 3 study that would support FDA approval.

That might seem ill-advised considering that, again, the drug didn’t meet any of its goals, but Cytokinetics’ share price rose about 6% yesterday, so at least some people think it’s a good idea.

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