It’s the “era of neuroscience”; an industry “turning point”; and “an amazing time”—superlatives were flowing freely as the 12th annual Clinical Trials on Alzheimer’s Disease congress (CTAD) kicked off in rainy San Diego late Wednesday afternoon.
The atmosphere felt thick with anticipation as attendees massed in the downtown hotel where the conference is taking place, less than a day away from a presentation of data from a late-stage trial of a drug that earlier this year appeared cosigned to that clichéd dustbin of history.
Disappointing results from Biogen’s Phase 3 trial of aducanamab—an amyloid-targeting drug developed based on the “amyloid hypothesis,” a theory positing that by reducing the protein, researchers could change the course of the disease—stopped the study in March. But in October, Biogen (NASDAQ: BIIB) floored the Alzheimer’s disease (AD) community by announcing that after looking deeper into the data and talking with the FDA about what it found, it planned to ask the agency to review the drug after all.
The company is set to reveal the data that led it to that conclusion at the conference 8 a.m. Thursday.
Stephen Salloway, the Martin M. Zucker Professor of Psychiatry and Human Behavior and professor of neurology at Brown University, opened his keynote address on how to interpret recent results in Alzheimer’s disease research with a joke nodding to the anticipatory fervor: “It’s been a real smooth ride this year, so it should be easy to make sense of what’s been going on.”
Biogen now has its chance to explain what’s been going on with aducanumab. If the data support an eventual approval, it would be the first new treatment OK’d by the FDA for the disease, which is the most common form of dementia, in 16 years, Salloway said.
Results reported in 2015 from a Phase 1b trial of aducanamab had an “exhilarating effect” on the field, Salloway recalled.
“Here we had a drug that targets plaque, that substantially lowers plaque even in a small Phase 1b trial, and it seemed to slow cognitive decline,” he said.
Results from the trial had an effect beyond the patients who received it and their caregivers, he said.
“This has help change the narrative and overcome fear and stigma, getting the message out of the hopeful nature of this potential breakthrough,” he said.
Those hopes were dashed this spring before being revived, shockingly, in fall.
“This is an unprecedented comeback,” Salloway said. “I don’t think I’ve recall seeing a drug that looked like it was headed to the dustbin coming back and going to the FDA in a short period of time for review.”
The data will shed light on how likely Biogen is to have a successful submission. Some industry followers have expressed skepticism as to the drug’s prospects. Only one of the two trials of the drug that the company analyzed met the primary endpoint, and it’s unclear why.
Even if it is OK’d, Salloway said the drug should be considered simply a “good first step” toward an eventual combination treatment that will markedly increase the quality of life for people with the disease.
He drew an analogy to the decades of work that led to HIV treatments that improved upon AZT, the first drug approved to treat the infection, noting the importance of research funding and broad public education in the development of better treatments in the form of combination therapies.
“This will not be the final treatment for Alzheimer’s disease, but this could give a foothold in treating the disease,” he said.
At the start of the conference, organizers recognized Zaven Khachaturian, president of Prevent Alzheimer’s Disease 2020, a senior science advisor to the Alzheimer’s Association, and editor-in-chief of Alzheimer’s & Dementia: Journal of the Alzheimer’s Association, for his foundational work in AD clinical trials.
Paul Aisen, director of the University of Southern California Alzheimer’s Therapeutic Research Institute and a San Diego-based member of the CTAD organizing committee, described Khachaturian as instrumental in the creation about 40 years ago of a national program, at the National Institute on Aging within the National Institutes of Health, aiming to treating the disease.
Khachaturian, in short remarks, urged continued scientific open-mindedness and rigor in pursuit of more effective treatments for Alzheimer’s and dementia, regardless of how the furor over aducanamab plays out.
“We’re lucky because now we have a coalition of advocacy groups such as the Alzheimer’s Association, academic groups, industry is very much engaged in it, Congress is very much engaged in it, and there is international interest,” he said. “Our obligation as a group is to deliver on that promise, and that requires doing better science than we have done, and doing science differently, and keeping our minds open to new ideas.”