- Allergan reported Wednesday evening that its blockbuster wrinkle-reducer Botox (onabotulinumtoxin A) failed to show statistical significance in a mid-stage trial of patients major depressive disorder (MDD).
- The topline data from the Phase 2 trial compared a 30 unit dose and a 50 unit dose against a placebo for 24 weeks. The primary endpoint for the study was change from baseline at six weeks on the Montgomery-Asberg Depression Rating Scale (MADRS).
- Neither dose showed statistically significant improvement over placebo, but the lower dose did show "numerically superior" data.
Numerically superior is a term companies use to sugarcoat a lack of statistical significance, or failure, in a study. This often means the drug appeared to perform better than placebo, but not enough to reach the thresholds clinicians were testing for.
Despite that, Allergan has chosen to move the drug forward into late-stage testing, hoping a larger study — likely of only the lower dose — will yield that elusive statistical significance it missed in the Phase 2 trial.
"We are encouraged by these data and the potential impact on adults with major depressive disorder. Given our in-depth and extensive clinical trial experience in CNS including depression, we plan to move forward and develop a phase 3 program for a potential new treatment option for patients," said David Nicholson, chief R&D officer at Allergan.
Botox has long been the gift that keeps on giving for Allergan. Prior to its reverse-merger with Actavis, Botox was virtually the only money-making product the small dermatology company had.
Botox was initially approved as a wrinkle-reducer and used as a cosmetic dermatology product, but over the years, Allergan has worked to get the toxin approved for almost a dozen other indications, including migraines, excessive sweating and overactive bladder.
Botox currently stands as Allergan’s best-selling product, with sales of $2.8 billion worldwide in 2016 — bringing in more than a $1 billion more than its next best-selling product. Aside from the many approved indications for the drug, Botox is also often used off-label for depression.
Evercore ISI analyst Umer Raffat expressed skepticism about the move to bring Botox into Phase 3 after the unfortunate mid-stage data.
"Data are not good … although I'm not sure investors gave any credit to this program to begin with," wrote Raffat in a note to investors on April 5.
"My sense: Botox has some off-label sales in depression anyway … perhaps they don't wanna [sic] jinx that near-term … and let a ph 3 go on for years to come," he added.