(Bloomberg) -- Biogen Inc. will ask U.S. drug regulators to approve the company’s experimental Alzheimer’s therapy, reviving the nearly abandoned treatment after a new analysis of more data from two failed clinical trials showed promising results.
The move by Biogen -- if successful -- would be the drug industry’s most dramatic reversal of fortune in history. The company said it made the decision after talking with the U.S. Food and Drug Administration and reviewing data showing the therapy helped Alzheimer’s patients’ cognitive function and their ability to perform basic tasks.
In March, the company said a major trial of the drug, aducanumab, had been a failure. That announcement sent Biogen’s shares down by more than 25% and raised questions about the firm’s future.
Investors erased most of those losses after the new announcement Tuesday, giving Biogen shares their biggest intraday gain on record. The stock jumped as much as 42% to $318 -- nearing where the stock traded before the March plunge.
The Cambridge, Massachusetts-based drugmaker said in a statement that it plans to send its application to the FDA early next year. It is developing the treatment in partnership with Japan’s Eisai Co.
Biogen Chief Executive Officer Michel Vounatsos said the company met with the FDA Monday and got the agency’s blessing to move ahead. “We got clear support from the FDA,” he said in a telephone interview. The company does not believe it needs to conduct additional trials of the drug before moving ahead.
The company also reported third-quarter financial results on Tuesday, though last quarter’s numbers will almost certainly be overwhelmed by investors’ review of a drug that, if it reaches the market, would upend the industry and reset Biogen’s future. Adjusted earnings were $9.17 per share, topping the $8.27 per share average of analysts’ estimates.
The Alzheimer’s results are certain to receive closer-than-usual scrutiny from regulators and investors, as well as rival drugmakers who have abandoned other related treatments after similar, earlier setbacks.
In the new analysis, Biogen looked at data from the two final-stage trials of more than 3,000 patients. A subset of patients treated with the highest dose of aducanumab had what the company said was a statistically significant slowing of decline of cognitive ability and basic activities of daily living.
The announcement shocked a medical field that’s grown used to failure after failure, and is skeptical that a previously unsuccessful trial could be the basis of drug that will make a meaningful difference for patients.
“Statistically significant does NOT guarantee clinically meaningful,” said Samuel Gandy, the associate director of the Mount Sinai Alzheimer’s Disease Research Center in New York. “I want to believe, but I’m not ready to suspend disbelief.”
That may not matter. There are no currently approved treatments to slow or stop Alzheimer’s, a neurodegenerative disease that is the most common cause of dementia in the U.S. and robs people of their ability to remember and to perform basic tasks. Biogen’s drug targets beta amyloid, a protein that forms clumps of plaque in the brain and is thought to be a cause of the disease.
Biogen’s drug reduced levels of the plaques, the company said. While other companies’ drugs have shown a similar effect on beta amyloid, they failed to provide better clinical results for patients. The drug does not appear to cure the disease, only to slow its progression.
The data giving the company hope is from a trial called EMERGE, which included 1,638 people with Alzheimer’s disease. The company made the initial decision to discontinue the trials based on results from December, but kept collecting information from patients and looking at the data. As more data rolled in, the company saw a positive signal from the group getting the highest dose of the drug. In a presentation to investors Tuesday, Biogen said the initial analysis had been incorrect.
“After consultation with the FDA, we believe that the totality of these data support a regulatory filing,” Biogen said in the presentation.
Alzheimer’s has led to failure after failure in the drug industry, often after large, costly efforts that showed glimmers of hope in early trials that ultimately led to disappointments in larger test. Any successful drug is likely to be a blockbuster, given the millions of potential patients and the lack of competition.
“So many companies ran away from this space,” Vounatsos said.
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