I worked on and around aducanumab, an experimental treatment for Alzheimer’s, for years during my time at Biogen. I thought it was going to work. I wanted it to work — my father had died of Alzheimer’s disease. It was awful watching his mind disintegrate over the course of a decade, which made me even more hopeful for a positive outcome for aducanumab.
I had also worked on dexpramipexole, a molecule that Biogen and Knopp Biosciences were testing as a treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. I wanted it to work, too, since one of my close friends had ALS. He was taking part in the trial for the drug, which made me even more hopeful for a positive outcome.
But dexpramipexole didn’t work any better than the placebo it was being tested against. I cried the day I found out. I remember sitting in a bathroom stall in Biogen’s building in Weston, Mass., with tears dropping onto my gray business casual trousers, crushed by the knowledge that my friend wouldn’t be helped, nor would countless others.
That day, George Scangos, who was the CEO of Biogen at the time, called an all-company meeting just after lunch. He asked that everyone stop what they were doing and come to the auditorium to talk about dexpramipexole.
George wasn’t a natural public speaker, and at times it showed. On that day, though, he walked to the podium without a discernible shred of discomfort. He stood calmly in front of the company, investors, and the media and, with a presence I had never before seen, told the crowd in no uncertain terms that dexpramipexole did not work and would not be a treatment for ALS.
He said the results made him profoundly sad, but made it clear that Biogen would do no fishing for false signals in subtypes or subgroups that, in the picked-over carcass of the dataset, may have appeared to have responded to the drug.
He said it was important to have the integrity to admit failure when failure was clear and how it was our responsibility to respect patients and their families by not giving them false hope. He then shared his resolution that Biogen would not stop fighting ALS and the company would focus its capital, time, and effort on the next generation of therapies.
Biogen’s stock got hammered, dropping more than 8% that day. Yet I’d never been more proud than I was that day to work for George and for Biogen, having seen for years companies hem and haw at negative results and claim that if you squint and look sideways that the molecule really did work, hoping that investors would have mercy on the stock price — and in the process ignoring the responsibility to be honest and forthcoming to patients and their families.
When I learned last week that Biogen had completed a retrospective analysis of the aducanumab data — the same data that seven months earlier the company had said justified stopping work on the drug — I thought of my father, my friend, George Scangos, and people around the globe with Alzheimer’s, ALS, and other diseases for which there are no cures.
I hope that Biogen made a mistake in its futility analysis back in March. But if it didn’t, and this is another example of the American public watching big pharma trying to spin bad data, then I can’t help but wonder if Biogen, and perhaps the industry, has lost its way.
Ted Whitford is a veteran of the biotech industry who now privately invests in the health care market.