Biogen stunned investors and scientists alike today, announcing it will resurrect an Alzheimer's drug it had declared a failure in March; the company plans in early 2020 to ask the U.S. Food and Drug Administration for marketing approval of aducanumab, an antibody created to bind and eliminate the protein beta-amyloid in the brain.
Lab employees work at Biogen Idec in Cambridge, Mass., April 25, 2013.
Biogen's CEO Michel Vounatsos said they're hopeful this drug will reduce Alzheimer's effects on the brain. Previously, the pharmaceutical company had conducted studies showing that treatment with aducanumab reduced the amount of amyloid plaque in the brain.
According to the Centers for Disease Control, as many as 5 million Americans were living with Alzheimer's disease in 2014, a number projected to almost triple to 14 million by the year 2060. If approved, aducanumab would become the first therapy to reduce the clinical decline of Alzheimer's disease and would also be the first therapy to demonstrate that removing amyloid beta resulted in better clinical outcomes. That data came from two separate trials, dubbed EMERGE (which enrolled 1,638 patients) and ENGAGE (which included 1,647 patients). When the studies repeatedly failed, Budson and others were starting to wonder whether the strategy was helping patients at all.
AD is a progressive brain disorder that affects older people, leading to loss of memory, decline in functional ability and change in personality and behaviour.
Biogen gave a presentation on aducanumab today which indicated that the result of the futility analysis based on data available as of 26 December 2018 "was incorrect".More news: President Taps Gantz to form Coalition after Netanyahu Fails
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But Biogen said its trial data is strong enough to apply for medicine licences in the US, Europe and Japan early next year.
The company said it chose to revisit submitting the therapy for approval following a new analysis of the data from the failed trials and a series of discussions with the Food and Drug Administration.
Alzheimer's and other dementias challenge researchers to search for ways to diagnose, treat and eventually cure - if not prevent - the disease. Both were Phase III multicenter, randomized, double-blind, placebo-controlled, parallel-group studies testing the safety and efficacy of 18-month regimens of aducanumab at two different doses.
In March, biotech company Biogen halted two Phase III clinical trials of the antibody drug aducanumab after the analysis of preliminary data suggested it was destined for failure. Multiple factors contributed to the greater exposure to aducanumab in the new analysis of the larger dataset, including data on a greater number of patients, a longer average duration of exposure to high dose, the timing of protocol amendments that allowed a greater proportion of patients to receive high dose, and the timing and pre-specified criteria of the futility analysis.
"It's a tricky question" whether the results are truly meaningful for patients until more details are known, said Dr. Ronald Petersen, a Mayo Clinic dementia specialist who has consulted for Biogen on the studies.
The failure of treatment, which was widely expected to be the Biogen's next blockbuster treatment, spurred demands on Wall Street for it to spend more on product acquisitions at a time when its peers have splurged out billions of dollars on deals. Together, they tipped the scales to show that those higher doses led to a "significant reduction of clinical decline from baseline".