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.
How unfortunate that rather than being excited for patients who may need this therapy, and in the process allow regulators to decide on the integrity of the data and the unmet clinical need, you’ve chosen instead to write a misleading, libelous headline based on no evidence whatsoever and a dramatic narrative to accompany it. Biogen should be commended, not condoned.
This drug has given many of us hope. Many who were on it. I think they should look into what it has done for the liver. For it is not a bad thing. Can’t say anymore then that. Fingers crossed.
Sir, Though you tried your best to sound impartial, your negative thoughts clearly show in every word you wrote. If the drug doesn’t work, FDA won’t probably approve it and it will clearly fail, but dont waste your time writing these stupid articles.
I am a free lance writer for the investor website GuruFocus. I plan to include the comments on here made by Theo Yang and Dr. Mark Hertzman and would like to know their titles, affiliations and qualifications.
It was the fda stating the data in emerge and trend at those patients receiving high dose Aducanumab for long enough are a file-able data set. Not the company on its own. The Emerge data are clear. It would be wrong NOT to pursue the high dose pathway.
I was in the study and I fear the same. The way it was shut down in dark of night and then up doe fda? I also have marker and had the negative side effect of brain swelling.
I too am skeptical.
My wife was in one of their clinical trials. It failed to work with her. No improvement whatsoever. I do know that she was getting the active drug rather than a placebo.
That was when her MMSE was over 20. Now, 5 years later, it’s too late for anything to work.
I work for Abec (the people who built the stainless steel tanks and systems for Biogen Luterbach) and when I read the news about the Alzheimers drug failing it crushed me because my grandfather just recently died because of it.
It’s so obvious who here is an actual researcher.
Its not working because industry is targeting a symptom, A beta is a secondary effect to an underlying metabolic dysfunction, just as Tau protein is. You actually want to cure Alzheimers? Then target the reason for hypoglucose metabolism, an early clinical feature known for over 30 years to distinguish AD from other dementias. Dont target a secondary effect.
The ‘Time’ magazine article detailed the dose/duration of treatment of two similar cohorts. The expected dose/response relationship is confirmed. The treatment will be approved for early/moderate AD patients.
I, too, worked on this compound, but as an investigator in the field. My guess is what Biogen has learned is similar to what we learned, after years, working on Lilly’s similar compound: that a small group of people may respond. The important question is: can these people be predicted in advance?