From the new Endpoints site, here’s a roundup of the worst moments in the clinic for drug development in the first half of 2016. Several of them were covered at the time here as well, but there the whole list is worth a look, because it illustrates the many ways that big trouble can hit.
The basic story is The Drug Didn’t Work, as you’d figure, but there are plenty of variations available. Unexpected tox, higher-than-expected placebo response, and “technically worked but not well enough for anyone to ever buy any of it” are three classics, and no matter what the problem, it can always be exacerbated by overpromising beforehand. That’s a vice common to small companies, but it can happen to the bigger ones, too.
For example, one of the recent events on the list that I haven’t mentioned involves AbbVie’s purchase of Stemcentrx. I first wrote about them here – they’re the Peter Thiel-backed company that’s been going hard after cancer stem cells. Data for their lead program (the antibody-drug conjugate Rova-T) against non-small-cell lung cancer were released recently at the ASCO meeting, but the results were not very impressive (one month survival advantage at best). This was a small trial, and there were indications that Rova-T might be able to perform better, but considering that AbbVie paid nearly $6 billion upfront (with $4 billion in potential milestones), people were expecting a lot more than this.
There will be room for another such list by the end of the year, I’m sure. But it’s worth keeping in mind that clinical failures are (given the state of our knowledge) built into the system we have. We do a very ragged job of picking winners in this business, so if there aren’t a fair number of clinical wipeouts, it would probably mean that we’re not putting enough high-risk high-reward things into the clinic in the first place. (For that matter, even some of the putatively low-risk programs hit the banana peel). We’re a long way from being able to avoid nasty surprises in Phase II and Phase III, a fact that investors should always keep in mind.