Biogen Stocks Rise Following FDA’s Inspiriting Comments on Alzheimer’s Drug, Aducanumab
By Daniel Ojeda, Ph.D.
On November 4th, the shares of Biogen Inc. and its partner Eisai Co Ltd jumped 40% after positive comments by staff from the US Food and Drug Administration (USFDA) ahead of a meeting where a decision regarding the recommendation of aducanumab’s approval will be made. The meeting is scheduled for this Friday, November 6th. If approved, it would mark a milestone for the treatment of Alzheimer’s disease. This announcement comes days after the European Medicines Agency (EMA) accepted aducanumab’s Marketing Authorization Application for review.
About Alzheimer’s Disease
Alzheimer’s Disease (AD) is a progressive neurodegenerative disorder that affects approximately 44 million people worldwide and around 5.5 million people in the USA. AD is the most common form of dementia, globally. It is characterized by aggregation of amyloid-beta (Aβ) peptides that form plaques and the formation of Tau protein-containing neurofibrillary tangles. This leads to progressive memory loss, difficulty concentrating and processing abstract concepts, eventually losing the ability to function independently. Although a small minority of cases are linked to specific genetic mutations, the exact factor(s) driving AD remains still not fully understood. Current research majorly focuses on Beta-amyloid and Tau, the two proteins believed to play a role in neural injury.
Treatment options for AD are minimal, with a failure rate of nearly 99%. Currently, there are two types of drugs available to treat the cognitive symptoms. The first type comprises cholinesterase inhibitors, such as Donepezil, Galantamine, and Rivastigmine, which increases the levels of a common neurotransmitter in the brain. The second type is Memantine, which regulates the activity of glutamate. However, no treatment is available that can slow down the progression of the disease, creating an unmet need.
Developed by Neurimmune and licensed by Biogen, aducanumab (BIIB037) is a monoclonal antibody that targets the beta-amyloid protein. The efficacy and safety of aducanumab were evaluated in two Phase 3 multicenter, randomized, double-blind, placebo-controlled, parallel-group clinical trials, EVOLVE, and EMERGE.
In early 2019, the company announced its decision to end the ongoing trials following a futility analysis conducted by an independent data monitoring committee. However, it made a U-turn several months later in October, when new analyses of the EMERGE trial data showed that aducanumab impacted the pathophysiology of the disease and slowed the cognitive and functional decline in patients with mild AD. Besides, a subset of patients from the ENGAGE study who received higher exposure to the drug support their findings from EMERGE.
Michel Vounatsos, CEO of Biogen, expressed enthusiasm late last week by saying, “Alzheimer’s disease has become a significant and growing burden for societies around the world, and we believe aducanumab represents the first breakthrough that can change the course of this devastating disease,”
Although there is a reason for investors to be excited about the news, there is still a chance that the FDA will recommend a third trial to confirm the efficacy of aducanumab fully. This recommendation would be based on the fact that EVOLVE clinical trial failed to show any benefits with aducanumab treatment, but the EMERGE trial demonstrated significant benefit.
The FDA staff said that data from one of the trials “provides the primary evidence of effectiveness as a robust and exceptionally persuasive study demonstrating” a clinically meaningful treatment effect. One statistical reviewer opined that a third study is needed to confirm aducanumab’s effectiveness. “There is no compelling substantial evidence of treatment effect or disease slowing,” FDA staffer Tristan Massie wrote in the review. If the agency recommends another Phase 3 clinical trial, it could take years for results to be available.
Editor: Rajaneesh K. Gopinath, Ph.D.
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