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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 email@example.com
(END) Dow Jones Newswires
October 22, 2019 19:26 ET (23:26 GMT)
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