Biogen was also cagey about safety, saying only that the drug's adverse-event profile in the two trials was "consistent with previous studies of aducanumab", but provided no details for the newly enlarged high-dose groups.
The company said that the results of patients in the failed second trial who had received high doses also supported the findings of the first. About 5.8 million people in the United States now live with Alzheimer's, and that's projected to rise to almost 14 million by 2050. If aducanumab is ultimately approved it will be the first drug to reach the market to treat cognitive decline in the early stages of Alzheimer's disease.
Biogen said it had met with the Food and Drug Administration twice, once in June and again on Monday to discuss the data. I have always believed that once a drug looked like it worked and got across the finish line with the FDA, it would open the floodgates to make better versions.
The rise in the company's share price might be in part because the last time aducanumab was in the news, it was in March, when the company and development partner Eisai announced they were discontinuing its Phase III ENGAGE and EMERGE trials of the drug due to a futility analysis that indicated they would fail to meet their primary endpoints.
U.S. drug company Biogen has announced that it will soon seek regulatory approval in the country for a "groundbreaking" drug that could slow down Alzheimer's disease.
Based on the discussions with the FDA, Biogen plans to file a Biologics License Application (BLA) in early 2020 and will continue talking to authorities in global markets such as Europe and Japan.
Bart De Strooper, who directs the UK Dementia Research Institute at University College London, called the news "fantastic".
Michel Vounatsos, chief executive officer at Biogen, said: "With such a devastating disease that affects tens of millions worldwide, today's announcement is truly heartening in the fight against Alzheimer's". That's according to Biogen, the drug's creator, which has announced plans to seek regulatory approval of Aducanumab sometime early next year. The new analysis suggests that at the highest dose, the drug, which aims to help the body clear harmful plaques from the brain, demonstrated an effective impact on patients. The announcement came as a shock to investors, as the drug, aducanumab, had previously been reported as a failure. Current drugs only temporarily ease symptoms and do not slow the loss of memory and thinking skills. But the results are not published in a peer review and experts have many questions about whether aducanumab actually works, and if so, how.
Founded by caregivers, and focused on creating a world without Alzheimer's and all dementia, the Alzheimer's Association is an organization that puts the person with dementia and family members first. These studies are targeting a wide variety of known and potential new aspects of the disease, such as inflammation and other promising new targets for therapy.
Rebecca Edelmayer, a scientist at the Alzheimer's Association, said: "We're really encouraged by the information that [Biogen] has provided" and added it is the "largest benefit so far" for any experimental medicine.
Tsai went on to temper her praise with some words of caution, "But with aducanumab, when they halted the trial, they said there was no hope that this drug would ever succeed".