CAMBRIDGE, Mass. — Biogen’s top scientist offered unflinching support Thursday for the efficacy of the company’s controversial Alzheimer’s drug called aducanumab, shrugging off outside skepticism and almost daring regulators not to approve it.
“I believe the drug works,” said Dr. Al Sandrock, Biogen’s head of research and development and chief medical officer, speaking at the STAT Summit. “I’m very excited about the prospect of getting the drug approved.”
In a statement emailed to STAT after this story ran, Biogen said, “Our focus is to follow the science. Biogen respects and appreciates the FDA review process.”
In October, Biogen shocked just about everyone when it said it was resurrecting aducanumab — and pursuing regulatory approval — based on a new and more positive analysis from two large clinical trials previously thought to be negative.
The crucially important details of this new aducanumab analysis have not yet been shared publicly, but that will change in early December when Biogen is scheduled to present the data at the Clinical Trials on Alzheimer’s Disease annual meeting.
What was expected to be a somewhat sleepy Alzheimer’s research meeting is now a red-hot ticket.
“I don’t think the field has ever seen data like this,” said Sandrock, sounding very much like a man who wants to prove Biogen’s critics wrong.
Dr. Michael Ehlers, Biogen’s previous research and development chief, left the company right before the aducanumab announcement. Some critics have pointed to Ehlers’ exit as a sign that there was internal disagreement about the strength of the aducanumab data.
Ehlers knew about the aducanumab results, “and I can tell you he was excited about it,” said Sandrock on Thursday. Ehlers’s decision to leave Biogen to join a venture capital firm was more about wanting to be “close to the science,” he added.
Biogen shared the aducanumab data with officials at the Food and Drug Administration during two face-to-face meetings held in June and October. After the second meeting, the FDA told Biogen that it was “reasonable” to file for aducanumab’s approval, Sandrock said.
Will the FDA approve aducanumab?
Sandrock didn’t offer a prediction, but if the FDA rejects the drug now or asks Biogen to conduct another large clinical trial to collect additional data, it would mean “lots more people” would get Alzheimer’s without a drug to help them.
“It’s in their hands now,” said Sandrock.
About the Author Reprints
Hello..
Which time this drug launched in medicine market..
May Allah accept us
..
This could be perfect for Biogen profits and terrible for patients.
My prediction:
FDA gets pressured to approve and Biogen gets to market an ineffective drug.
Competitors create me-too drugs that all fail in their trials (because the original compound doesn’t really work).
Biogen ends up with market exclusivity due to luck / Type I error.
Meanwhile scientists keep chasing the amyloid hypothesis in order to get a second drug with this mechanism on the market — a mechanism that never worked in the first place.
Potentially effect treatments never get explored.
I hope that even if FDA approves they require a confirmatory trial — much like FDA required AMAG to do a follow-up trial on Makena (which turned out not to work in Pre-term birth).
My wife has demita and would like to take this drug when it gets approved
I am 71 years old. I have recently been diagnosed with alzheimer’s disease after some years of MCI diagnosis. I would be only too pleased to try aducanadab treatment (or almost any other possible remedy) if I thought that there was any chance whatsoever that I could achieve a cure or even some respite term for this insidious disease.
The prognosis in this area is dire and I can only look forward with considerable despair! Please note! Do not delay! Those of us in this predicament cannot wait.
Yours faithfully
Anne Goodwin
I have lost 3 person and I am praying for my wife for this to work
I am willing to let her be a subject in any test to prove the value of any test that you would have. I pray every day for a cure to this nightmare.
how can I get this to help my wife??? please
Mike B
It seems warranted as solution to the possibly false hype to put Dr. Ehlers on the witness stand. It is just too unbelievable that at the time of expensive trials this “new finding” was overlooked or not considered. I side with the “false hope” stance for Alzheimer patients and their families. Dr. Sandrock’s blunt posturing is almost offensive but sure smells of big bribes being involved.
It is relatively common in the world of Alzheimer’s drug development for head research scientitsts to over promote their drug candidates. Al Sandrock clearly fits into this category.
The only charts Biogen has released so far are for aducanumab’s primary endpoint: CDR-SB scores.
https://investors.biogen.com/static-files/40565136-b61f-4473-9e58-9be769bbac6c (page 22).
At 78 weeks, patients taking aducanumab in the Emerge trial have declined by about 1 point whereas those in the Engage trial have declined by about 1.1 points which makes sense since the two trials were nearly identical. The apparent slower rate of decline in the Emerge aducanumab group versus the Engage aducanumab group is almost entirely due to differences in the rate of decline in the placebo groups in each trial (1.8 versus 1.5) This amounts to a .8 point less decline versus .4 point less decline. The former is just barely significant whereas the latter is not.
Anti-amyloid drugs such as aducanumab appear to have almost no effect on non-APOE4 carriers whereas they have some effect on APOE4 carriers. A person with one APOE4 gene declines about 14 percent more rapidly and a person with two copies of the APOE4 gene declines about 23 percent more rapidly in early Alzheimer’s disease than those without the gene. What anti-amyloid drugs do is to reduce the rate of decline for APOE4 carriers to a rate similar to that of non-carriers.
What the FDA should require from Biogen is like comparisons: non-carriers taking aducanumab versus non-carriers taking the placebo, individuals with one copy of the APOE4 gene taking aducanumab versus individuals with one copy of the gene taking the placebo, and individuals with two copies of the gene taking aducanumab versus individuals with two copies of the gene taking the placebo. In the end, only people with two copies of the APOE4 gene may benefit to any degree from the drug but they are also the group most likely to suffer from serious side effects due to the drug.
This is not a drug that will substantially slow down the rate of decline in people with Alzheimer’s disease.
Here an idea take a video camera and ask a couple of volunteers a couple of questions. If they are doing good post the video so we can all view the success. I think its all about stocks and money and a drug that is better than nothing. My opion
No. It worked.
How unbelievably cruel to with hold a medicine that will help Alzheimer’s patients and their families caring for them. Then why not just do away with all medicines.
Ms Sharp did you read Lane Simonian’s informed comment (with links) at the top of the page? What is truly cruel is to raise desperate people’s hopes in order to inflate stock prices, and then disappoint them and their families, after disrupting the difficult and crucial process of coming to terms with a terrible truth. The timing of Dr. Ehler’s departure from the company is ominous.
I agree with Isobel. Also, many studies are beginning to show that mind-body interventions are highly effective for Alzheimer’s – see the studies below. It is just that profit driven pharma always want to come up with drugs for EVERYTHING.
Last, N., et al. (2017). The effects of meditation on grey matter atrophy and neurodegeneration: A systematic review. Journal of Alzheimer’s Disease, 56(1), 275-286.
Quintana-Hernández, D. J. et al., Mindfulness in the maintenance of cognitive capacities in Alzheimer’s disease: a randomized clinical trial. J Alzheimers Dis. 50, 217-232 (2016).
Larouche, E., Hudon, C., & Goulet, S. (2015). Potential benefits of mindfulness-based interventions in mild cognitive impairment and Alzheimer’s disease: an interdisciplinary perspective. Behavioural Brain Research, 276, 199-212.
Lima, S., Garrett, C., Machado, J. C., Vilaça, M., & Pereira, M. G. (2019). Quality of life in patients with mild Alzheimer disease: the mediator role of mindfulness and spirituality. Aging & mental health, 1-8.
Russell-Williams, J., et al. (2018). Mindfulness and meditation: treating cognitive impairment and reducing stress in dementia. Reviews in the Neurosciences, 29(7), 791-804.