Scientists and analysts greeted yesterday’s announcement that Biogen is trying to resurrect aducanumab, its experimental Alzheimer’s drug, with caution and outright skepticism.
Minutes after Biogen released its stunning news, investment analysts pummelled the company’s executives on a conference call. And in an overview puplished by StatNews, one investment analyst likened Biogen’s move to “a game of corporate three-card monte.”
Last March, Biogen said that aducanumab had failed a “futility analysis” and abruptly cancelled two large studies. Now, however, Biogen said that its decision to seek FDA approval for the drug was based on further data analysis that showed that patients who received aducanumab “experienced significant benefits” in memory and general cognition.
“Patients also experienced benefits on activities of daily living including conducting personal finances, performing household chores such as cleaning, shopping and doing laundry and independently traveling out of the home,” the company said.
Dr. David Wolk, co-director of the University of Pennsylvania’s Memory Center, called the announcement “hopeful,” but quickly added that “there is much yet to learn.”
“While we are all happy that this may ultimately turn into a viable treatment, we should also be cautious until the research community and the FDA has an opportunity to review these data with the company,” Dr. Wolk said.
The Biogen announcement was indeed not thorough on data, a fact that caused some researchers skepticism.
In an interview, Dr. Vincent Mor, a professor of Health Services, Policy and Practice at Brown University, greeted the news of the announcement with skepticism, saying that it was likely to be “hugely controversial.”
“If the effect of a treatment is large, you tend to see it in the data as soon as you open up the data, as soon as you are unblinded,” Dr. Mor explained. “If it was ambiguous and now they think it goes in one direction as opposed to another direction, the effect was probably not large.”
“It’s probably nothing to write home about in the long run,” Dr. Mor concluded.
But given the enormous unmet need for an Alzheimer’s treatment, experts suggested that the FDA may be open to approve a drug that provided some benefit even if it is not a home run treatment or cure.
Evercore analyst Umer Raffat told StatNews that “it’s not inconceivable that FDA is open to this filing.” However, he added, “whether FDA approval means major commercial acceptance is a whole different debate.”
Biogen said it would release additional data at a major Alzheimer’s conference in December.
Aducanumab targets the build-up of amyloid in the brain, which has long been suspected as a cause of Alzheimer’s. If approved by the FDA, aducanumab would be the first treatment to demonstrate that removing amyloid from the brain impacts Alzheimer’s.
In the meantime, people living with Alzheimer’s and their families—particularly those who participated in the aducanumab trials—were left wondering about the future. Biogen has not communicated the news with research centers or patients.
The Penn Memory Center, which participated in the aducanumab trials, sent a note to patients saying that it “appreciates your patience as news continues to come forward regarding Biogen’s announcement” and promised to continue to provide updates “as we and the rest of the research community learn more over the coming months.”
At the Advanced Memory Research Institute (AMRI) of New Jersey, which had the largest contingent of people enrolled in the trials, researchers expressed “heartbreak” about the Biogen news.
“We were excited to hear the news of the FDA submission,” AMRI said in an email, “but we were also disappointed that the results could not have been found sooner so that the trials would not have to end.
“We had too many subjects, families, and staff who were heartbroken over this study’s termination,” AMRI continued. “We wish we could have spared that heartbreak, but we are hopeful that we may be able to offer them something positive in the year to come.”