(Bloomberg Opinion) -- Just eight months after it announced the failure of its lead Alzheimer’s drug, aducanumab, Biogen Inc. is attempting to bring it back.
The biotech giant announced Tuesday that it plans to submit the drug for FDA approval based on “a new analysis of a larger dataset,” which showed that the drug was able to slow mental decline in some patients after all. It’s an astonishing turnaround that sent the stock surging some 40% in early trading.
The drug represents a substantial commercial opportunity in a devastating and currently untreatable disease, and the Food and Drug Administration has been increasingly flexible on approving drugs in diseases with no good options. But let’s not get ahead of ourselves. Investors should treat this update with caution; instead, they appear to be overreacting. I’ll be surprised if this gambit works. The data is far from clear.
Biogen discontinued two studies of aducanumab in March based on what’s called a futility analysis, a pre-specified early look to see if a study is likely to succeed and worth continuing. It now thinks the initial review was incorrect after looking at new data from more patients. After adding those findings, the company says one of the two trials, called Emerge, showed the drug had a statistically significant impact in patients who took a high dose.
Even if Biogen manages to convince the agency that the director’s cut of its first trial is legitimate, the company may fall short. That’s because the FDA typically demands two successful trials for this type of drug, and the second trial, Engage, is still a clear failure. At the high-dose that Biogen is touting, patients who took aducanumab appeared to do worse than those who took a placebo on a key measure even with the new data. That’s tough to explain away, though Biogen is certainly trying. The company says more patients in Emerge were exposed to sufficient levels of the drug, and that data from a subset of Engage patients looks positive. But that second analysis appears to be from a small group of patients and is difficult to trust.
There are other red flags. Generally speaking, scientists aren’t fans of re-analyzing data. It inevitably injects bias into trials that are carefully designed to avoid it. If you interrogate any data set long enough, especially when you have an enormous financial incentive to find success, you’re likely to discover a positive interpretation. A few years back, Eli Lilly & Co. dug through two failed late-stage trials of a similar Alzheimer’s medicine and decided it only failed because the company had picked the wrong patients. It ran a new study based on that assumption, and it failed.
Biogen’s rescue mission is in part a different spin on the story; the company says its new data means that one of its trials should never have been labeled a failure in the first place. The attempt to put a positive spin on the second badly failed trial is the same sort of cherry-picking that gets drugmakers in trouble.
The world badly needs an Alzheimer’s drug, and the company’s engagement with the FDA may mean the agency is inclined to be open-minded. Biogen still has an uphill battle to prove that it actually has one.
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Max Nisen is a Bloomberg Opinion columnist covering biotech, pharma and health care. He previously wrote about management and corporate strategy for Quartz and Business Insider.
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