By Joseph Walker
Biogen Inc. plans to seek regulatory approval early next year
for an Alzheimer's disease drug that had been considered a lost
cause after the company pulled the plug on late-stage studies
because of disappointing results.
The surprising about-face, which Biogen made public on Tuesday,
came after the company took a closer look at study data that it
didn't have when it halted efforts in March. The new data indicated
the drug did work in patients who received the highest dose in one
of the studies.
The second look triggered conversations with regulators that
breathed new life into the drug, while raising the hopes of those
confronting Alzheimer's memory loss, reviving a long-running
hypothesis about its molecular roots and adding more than $13
billion to Biogen's market capitalization.
The Cambridge, Mass., biotechnology company had been discussing
its new data analyses with the U.S. Food and Drug Administration
since June, Biogen Chief Executive Michel Vounatsos said in an
interview. But it wasn't until the company's latest meeting with
the FDA on Monday that Biogen gained confidence to submit the drug
called aducanumab for approval, he said.
"Until yesterday, it was still inconclusive," Mr. Vounatsos
said.
An FDA spokesman declined to comment, citing confidentiality
provisions in federal law.
Shares in Biogen surged 26% to $281.87. When the company and
partner Eisai & Co. said in March they would terminate the
late-stage studies, Biogen lost about $18 billion in market value.
Even after Tuesday's surge, Biogen shares were down 6.3% since the
end of last year.
The company said it plans to seek approval for aducanumab in
early 2020 and would continue talks with regulatory authorities in
international markets, including Europe and Japan.
If approved, Biogen's drug would be the first to slow cognitive
decline in Alzheimer's patients, a milestone in long-running but
largely futile efforts to find a medicine that can treat the
memory-robbing disease.
Given the lack of treatments, patients and investors had been
excited by aducanumab's potential when it started two late-stage,
or Phase 3, trials to see whether it worked and should be
approved.
But in March, Biogen said it was discontinuing the studies early
after conducting a so-called futility analysis in which researchers
looking at interim results predict whether a study will fail.
That analysis was based on data from patients who had completed
18 months in the trials through December 2018, or 49% of patients
enrolled in the first trial and 57% of patients in the second
trial.
After discontinuing the trials, Biogen reanalyzed the data based
on additional data from patients who had completed the trial
between December and March.
The new data showed that the drug's highest dose succeeded in
the second trial in significantly slowing patients' cognitive
decline compared with placebo, Biogen said.
The first study was still considered a failure in the new
analysis, but its results looked more encouraging when looking only
at patients who took the highest dose for extended periods, the
company said.
P. Murali Doraiswamy, professor of psychiatry and behavioral
sciences at Duke University School of Medicine, said in an
interview that the new data Biogen released looked encouraging but
more detailed results are needed before drawing any definitive
conclusions whether aducanumab works.
Biogen's plan to use two discontinued studies for a new drug
approval is unusual, if not unprecedented, not least because making
public that the studies had failed could introduce bias into the
results from trial investigators, Dr. Doraiswamy said.
Still, "the FDA would be likely to give them the benefit of the
doubt" if the company can show the failed study is supportive of
the successful study, he said.
But whether regulators will clear the drug for sale, or whether
insurers will pay for it, is unclear.
For now, investors are betting that Biogen's consultations with
the FDA are a good indication the agency will overlook any flaws in
how the studies were conducted to approve the first new drug for a
devastating disease affecting millions of people in the U.S.
The surge in Biogen's share price on Tuesday suggests investors
see a 50% chance of the drug being approved, said Geoffrey Porges,
an SVB Leerink LLC analyst.
Based on the company's remarks about its talks with the FDA, Mr.
Porges pegs the odds of approval even higher, at two-thirds.
Still, Mr. Porges said he worries about the economic impact that
an approval could have on the U.S. health-care system. "The
breakthrough medicines that make a really big difference are
expensive enough," Mr. Porges said. "But here you could have
millions of patients using a medicine with what appears to be a
fairly modest effect, if any effect. Is that the type of thing we
should be bankrupting Medicaid for?"
Biogen's reversal is also likely to reignite debate over the
hypothesis that has informed much of the recent research and
investment into potential Alzheimer's drugs: that the buildup in
the brain of a sticky substance called Beta amyloid plays a pivotal
role in the disease.
Drugs like aducanumab target the sticky tangles with the goal of
slowing or halting the progression of the disorder. But a number of
drugs developed with the idea in mind have failed.
After Biogen discontinued the aducanumab trials in March, some
researchers began calling for a rethinking of the field's focus on
amyloid and the need to invest in new approaches. Now, the FDA
might have the final say. "If the FDA were to give its blessing"
and approve aducanumab, "that would validate the amyloid
hypothesis," Dr. Doraiswamy said.
Write to Joseph Walker at joseph.walker@wsj.com
(END) Dow Jones Newswires
October 22, 2019 19:26 ET (23:26 GMT)
Copyright (c) 2019 Dow Jones & Company, Inc.
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