Highly Anticipated Data On Would-Be Alzheimer’s Drug Raises Hope And Questions

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How does Biogen Chief Medical Officer Al Sandrock feel after the curtain was lifted on clinical trial data of the experimental Alzheimer’s drug his company is developing with Japanese drugmaker Eisai?

“It was a shot in the arm for me,” he says of the trial results, which showed BAN-2401 cleared amyloid plaques from the brains of people in the early stages of Alzheimer’s disease and reduced the cognitive decline they experienced. For him, it’s supporting the whole idea that targeting amyloid can help people with Alzheimer’s, repeating the experiment Biogen ran with its own amyloid-targeting experimental drug aducanumab and getting the same results.

“As a neurologist who used to see these patients and feel completely helpless, the idea that we’re getting close now to potentially getting the first disease-modifying therapy is a big deal for me,” he says. “That’s why I was so excited when I saw the BAN data.”

Sandrock is feeling good. But not all observers see the data as positively, and some large questions remain for further data analysis and future trials to answer.

One of the first questions asked after the data was presented at the Alzheimer’s Association International Conference in Chicago yesterday was whether the decision of a health authority (not the U.S. Food and Drug Administration) that patients carrying a variant of the APOE gene linked to earlier onset of Alzheimer’s disease could not be randomized into the highest dose group, 10mg/kg every two weeks, could have made that dose group look better than it should. “We argued against it vehemently, and results would suggest we might have been right,” said Eisai Chief Medical and Clinical Officer Lynn Kramer, who presented the data.

That disclosure was a “curveball,” EvercoreISI analyst Umer Raffat wrote in a debriefing note to clients, and on Biogen’s webcast for analysts he called it the biggest question about the trial. In further analysis, he makes the case that it doesn’t doom the trial results to be an artifact of the highest dose arm getting healthier patients. Sandrock said more analysis of the trial data to figure that out would come soon.

Brian Skorney, an analyst at R.W. Baird, is more skeptical. “The noise in this study is deafening,” he wrote in a research note. “We do not think this study provides good supportive evidence for aducanumab.”

“I think there are a lot of caveats with these data, and overinterpreting them and overhyping them is a mistake for our field,” said Reisa Sperling, director of the center for Alzheimer Research and Treatment at Brigham and Women’s Hospital. “But for me it’s evidence we’re on the right track, really really encouraging.”

Whether the slowed decline in cognition and function BAN-2401 appeared to confer in the trial is meaningful for patients is hard to say because doctors don’t have much experience with the main assessment the trial used, Sperling says. And it depends what stage of Alzheimer’s the patient is at. For those with mild dementia, the 30% reduction in decline the trial showed is “important but not good enough,” she says. In an earlier stage, where many of the patients in the BAN-2401 trial were, that effect looks better: “If you could give people three or five more years of independence, that’s a huge deal.”

Whether BAN-2401 makes it to patients remains to be seen, but Eisai USA CEO Ivan Cheung is “optimistic,” he said in an interview before the data presentation. “We will be very proactive with the FDA and other health authorities in finding the best path forward for BAN-2401,” he said. “Any innovation needs to become actual medicine in the hands of patients.”



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