Alzheimer’s fight all but a stalemate
Innovative drug trial shows promise, then disappoints.
A promising drug trial excites doctors and investors. The company’s stock soars. Then more data pours in, confusion grows, the shares crash and the questions start — will this Alzheimer’s drug fail like all the others?
That’s been the history of treatments for the neurodegenerative disease, which affects about 35 million people around the world. There’s no approved product that can slow or stop the ailment, and about 200 drugs have failed despite years and billions of dollars of effort.
A new clinical trial of a drug, called BAN2401, under development from Biogen and Eisai seemed like it might reverse that trend, after the companies said earlier this month that their study of the compound showed it slowed progression of the disease.
Instead, investors who follow the companies and doctors looking for a useful treatment have been left with more questions than answers, even after trial results released this week showed that the drug slowed the progression of the earliest stages of the disease by 30 percent. The findings added a glimmer of hope but left plenty of reasons for doubt.
The BAN2401 trial was unusual from the start.
Most drug studies assign fixed numbers of patients to each part of the trial. Some get a placebo. Others get a low dose of the drug, and the rest a higher dose. It’s a system to identify whether a drug is better than a sham treatment, and if so, what dose is best?
Eisai and Biogen did something different, hoping to save money and time. The trial added patients into whatever dose looked most promising as the trial proceeded. It was an idea meant to help them quickly find a dosage for a larger, final-stage trial without needing to enroll as many participants.
Researchers also used a novel measure of how fast a patient’s Alzheimer’s was progressing. The new method was designed to pick up the earliest hints of cognitive decline, a more sensitive tool than the blunter measurements used in past trials.
Even with the new methods, the trial failed to meet its primary goal. In December, after calculating 12 months of patient results, the drug hadn’t clearly slowed cognitive decline. While many studies would have stopped then, the trial was designed to continue so the company could reanalyze the data under a different method with six months’ more data.
When they did, the trial appeared to have turned from failure into success.
A day before the results for BAN2401 were unveiled to doctors and researchers gathered at a convention center in Chicago, an executive from Eisai promised certainty, something that failed trials from a host of other drugmakers hadn’t been able to offer.
“It will be clear,” said Lynn Kramer, Eisai’s chief medical officer for neurology. “You won’t have any ambiguity.” That wasn’t the case. One stock analyst, Robert W. Baird & Co.’s Brian Skorney, said there were “almost no reasonable conclusions to draw.” Another, Laura Chico of Raymond James Financial, called the results “head spinning.” Biogen and Eisai shares plunged.