Good afternoon, readers.
The biotech world went into a full-on frenzy Tuesday when drug giant Biogen dropped this whopper: The company is reviving its Alzheimer’s drug hopeful, aducanumab, after leaving it for dead all the way back in March. In fact, it’s marching forward with a Food and Drug Administration (FDA) application to approve the drug for certain patients facing cognitive decline.
It’s hard to overstate how unusual (and unexpected) this development is. Biogen had initially concluded, after some significant hype in initial trials, that aducanumab ultimately wouldn’t pass muster following something called a “futility analysis” of the experimental treatment.
Now, it’s arguing that futility analysis may prove, well, futile.
“Today is about hope and opportunity,” Biogen CEO Michel Vounatsos told Fortune in a phone interview. “The decision was part of a new analysis of the dataset. The ‘T’s were crossed and the ‘I’s were dotted.” (That sort of bullishness, at least on Tuesday, seemed to permeate the investor class, which sent Biogen shares soaring more than 26%—though the staying power remains to be seen.)
The procedural mishmash on this is fairly complicated. To put it in simple terms: There was still data coming in after aducanumab’s initial demise, and Biogen’s newfound optimism is based on a renewed analysis, of a subset of patients, who began receiving higher doses of the drug. That analysis suggests aducanumab can slow the rate of cognitive decline among certain Alzheimer’s patients.
Things get even more complicated, though. For instance, there were two late-stage aducanumab studies that led to this conclusion—one met its clinical goal outright, but the other did not. But even in that latter arm, Biogen says, certain patients saw a benefit after being switched to a higher dose.
“It was a gradual understanding,” said Biogen’s chief medical officer Alfred Sandrock in an interview.
The reactions to the news have ranged from legitimately optimistic to highly skeptical. Asked whether the announcement was unequivocally good news, bad news, or a mixed bag, Keith Fargo, director of scientific programs and outreach at the Alzheimer’s Association, told Fortune, “It’s… interesting good news.”
Fargo demurred on whether or not aducanumab will ultimately get across the FDA finish line given the highly unusual circumstances (he says he doesn’t recall another instance like this where a drug left for dead by a company is suddenly revived). But he says any progress on a treatment that attacks, not just the symptoms of Alzheimer’s, but the underlying causes of the disease, is worth cheering.
“We’re excited about the submission, we’re hopeful it will be approved if appropriate,” he said. “But the FDA will have many considerations to make.”
Some analysts, such as investment bank Evercore/ISI’s Umer Raffat, appeared to hedge on whether this will play out well in an investor note; the FDA could very well approve the treatment (as both Vounatsos and Sandrock have mentioned, the decision to submit for regulatory approval came after conversations with the agency). But the picture is far from clear.
Much more on this soon. Read on for the day’s news.
Sy Mukherjee, @the_sy_guy, sayak.mukherjee@fortune.com
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