After iffy phase 2, Allergan plows forward with phase 3 Botox depression study

Analysts are split on just how promising Allergan's phase 2 Botox depression data actually is, but the company believes the "pipeline in a drug" can deliver.

Allergan’s out with a mixed bag of phase 2 results for blockbuster Botox in depression. And while some analysts aren’t thrilled by the data, the company sees enough promise to move the therapy into a wide phase 3 study.

Going up against placebo, a 30-unit dose of the med significantly moved the needle on the Montgomery-Asberg Depression Rating Scale in adult women at weeks 3 and 9, posting changes of -4.2 and -3.6, respectively, Allergan said Wednesday. But at Week 6—the study’s primary endpoint—the 30-unit dose of Botox failed to post statistically significant results. And the 50-unit arm, as Evercore ISI analyst Umer Raffat put it, “did NOT work,” failing to top placebo in the efficacy department at the primary endpoint.

Allergan, though, was undeterred, announcing that it was “encouraged by these data” and planning to move forward with a phase 3 study, company R&D chief David Nicholson said in a statement.

Why? The way Wells Fargo analyst David Maris sees it, with the results showing “a positive trend at certain data points,” he thinks “one might reasonably expect a bigger study may show more clearly positive results.”

“We believe Allergan’s depth of experience with Botox and, separately, in depression, should provide an abundant opportunity to explore the potential of Botox in depression,” he added in a note to clients.

Raffat, however, was much more skeptical in his own investor note.  “My sense: Botox has some off-label sales in depression anyways ... perhaps they don’t wanna jinx that near-term ... and let a phase 3 go on for years to come,” he wrote. “ ... Maybe I am being too pessimistic here ... but the data don’t read good on this one.”

Of course, if Allergan can get it done in phase 3, there’s a large patient population waiting for new therapy options. There’s “a big need for different treatment modalities in depression,” a condition that affects millions of Americans, Nicholson said in an interview last year, pointing out that “any given antidepressant is effective in perhaps one-third of the patients who receive it.”

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Even if the Dublin drugmaker can land a depression approval for Botox—a product that already boasts 11 indications and has provided a “pipeline in a drug” for Allergan for years—the challenge won’t end there. Botox, unlike its depression rivals, is an injectable, rather than an oral therapy, and most psychiatrists aren’t exactly used to giving injections.

Allergan, though, isn’t worried. “We will need to educate the psychiatric community,” Nicholson acknowledged, but if Botox can prove its worth, “I have no doubt in my mind the psychiatric community will embrace it and will start giving in injections,” he said.