Forget the Hint of Efficacy

In drug development, a hint of efficacy is usually a lot worse than no efficacy at all. At least when a drug fails hard, it's easy to throw it on the shelf and focus on something else. When there's a hint of efficacy, drugmakers have to make the hard choice of exploring the hint, sinking more time and money into a drug that might not work, or end the development despite the small hope of potential.

Let's hope Eli Lilly (NYSE: LLY  ) doesn't make the wrong one.

On Friday, the pharma said its Alzheimer's disease drug, solanezumab, failed two phase 3 trials. Both trials measured cognitive and functional decline of the patients, and the drug failed to show an effect compared with placebo for both measurements in both clinical trials. It really doesn't get much worse than that.

Except, but, bar, save for, however -- insert your own glimmer of hope -- if you combine the two trials, solanezumab slowed the cognitive decline compared to placebo. If you just look at the patients with mild Alzheimer's disease, the pooled data shows statistical significance, but the pooled data for moderate Alzheimer's disease patients doesn't.

Lilly hasn't released the full data set yet, but I'm willing to bet if you stand on your head and read the data upside down, you can probably find something else interesting that solanezumab does. Cure arachnophobia in left-handed women with blonde hair, perhaps?

It's not quite that bad, because looking at efficacy in the pooled data was a pre-specified analysis, but it isn't going to get the drug approved, given that it failed the primary analysis of the individual trials. At the very best, Eli Lilly will have to run two more clinical trials, each one as large as the first two combined, in hopes of showing efficacy, and even then, the FDA would probably want to see improvement in patient function as well as a cognitive improvement.

If it takes 2,000 patients per trial to prove that a drug works, it's likely that it isn't doing all that much to help the patients. With large numbers, small decreases in cognitive or functional decline can be statistically significant, but that doesn't make them clinically meaningful.

Earlier this month, Pfizer (NYSE: PFE  ) , Johnson & Johnson (NYSE: JNJ  ) and Elan (NYSE: ELN  ) ditched their similar-acting Alzheimer's disease drug, bapineuzumab, after it failed a couple of clinical trials. Despite the hint of efficacy, Eli Lilly would be wise to do the same.

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  • Report this Comment On August 28, 2012, at 3:29 PM, chenac wrote:

    I'd wait to see the details of the study before suggesting that they drop the drug altogether. Their post hoc analysis of the study was pre-specified, not done specifically to generate significance in their results.

    Considering the sheer impact of AD on our society and with no disease modifying drugs available, I'd be willing to gamble on a drug that shows some promise. Again the devil is in the details.

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